UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-Q
☒
For the quarterly period ended SeptemberJune 30, 20182019
OR
☐
For the transition period from to
Commission file number: 001-35890File No. 001‑35890
Millendo Therapeutics, Inc.
(Exact nameName of registrantRegistrant as specifiedSpecified in its charter)
Delaware | 45‑1472564 | ||
(State or Incorporation or | (I.R.S. Employer Identification No.) | ||
110 Miller Avenue, Suite 100 | 48104 | |
(Address of | (Zip Code) |
Registrant’s telephone number, including area code)code: (734) 845‑9000
301 North Main Street, Suite 100, Ann Arbor, Michigan 48104
(Former Name, Former Address and Former Fiscal Year, if Changed Since Last Report)
Securities registered pursuant to Section 12(b) of the Act:
Title of each class | Trading Symbol(s) | Name of each exchange on which registered |
Common Stock, $0.001 par value | MLND | The Nasdaq Capital Market, LLC |
Indicate by check mark whether the registrant:registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the Registrantregistrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405229.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company or an emerging growth company. See the definitions of “large accelerated filer,” “acceleratedaccelerated filer,” “smaller reporting company” and "emerging“emerging growth company"company” in Rule 12b-212b‑2 of the Exchange Act.
Large accelerated filer | ☐ | Accelerated filer | ☐ | |||
Non-accelerated filer | ☒ | Smaller reporting company | ☒ | |||
Emerging | ☐ |
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-212b‑2 of the Exchange Act). Yes
The number of November 6, 2018, there were 35,826,429 shares of the registrant’sRegistrant’s Common Stock, $0.001 par value $0.001 per share, outstanding.outstanding as of August 9, 2019 was 13,412,058.
2
| As of September 30, | As of December 31, | ||||||
| 2018 | 2017 | ||||||
| Assets | |||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 31,917 | $ | 15,703 | |||
| Short-term investments | 14,433 | 51,500 | |||||
| Short-term restricted cash | 789 | — | |||||
| Prepaid expenses and other current assets | 1,847 | 1,578 | |||||
| Total current assets | 48,986 | 68,781 | |||||
| Property and equipment, net | 108 | 3,113 | |||||
| Investment in joint venture | 140 | 146 | |||||
| Long-term restricted cash | — | 789 | |||||
| Other long-term assets | — | 24 | |||||
| Total assets | $ | 49,234 | $ | 72,853 | |||
| Liabilities and stockholders’ equity | |||||||
| Current liabilities: | |||||||
| Accounts payable | $ | 604 | $ | 2,242 | |||
| Accrued expenses and other current liabilities | 3,277 | 5,562 | |||||
| Total current liabilities | 3,881 | 7,804 | |||||
| Other non-current liabilities | 442 | 751 | |||||
| Total liabilities | 4,323 | 8,555 | |||||
| Commitments and contingencies (Note 10) | |||||||
| Stockholders’ equity: | |||||||
| Preferred stock, $0.001 par value; 5,000,000 shares authorized, no shares issued and outstanding | — | — | |||||
| Common stock, $0.001 par value; 100,000,000 shares authorized; 35,826,429 and 35,725,230 shares issued and outstanding at September 30, 2018 and December 31, 2017, respectively | 36 | 36 | |||||
| Additional paid-in capital | 367,110 | 365,769 | |||||
| Accumulated other comprehensive loss | (3 | ) | (27 | ) | |||
| Accumulated deficit | (322,232 | ) | (301,480 | ) | |||
| Total stockholders’ equity | 44,911 | 64,298 | |||||
| Total liabilities and stockholders’ equity | $ | 49,234 | $ | 72,853 | |||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
| 2018 | 2017 | 2018 | 2017 | ||||||||||||
| Revenues | $ | 55 | $ | 56 | $ | 203 | $ | 204 | |||||||
| Costs and expenses: | |||||||||||||||
| Costs of revenues | 44 | 29 | 210 | 572 | |||||||||||
| Research and development | 1,680 | 4,016 | 6,695 | 14,777 | |||||||||||
| Selling, general and administrative | 4,176 | 5,056 | 11,045 | 22,935 | |||||||||||
| Restructuring | 53 | 361 | 3,637 | 3,842 | |||||||||||
| Total costs and expenses | 5,953 | 9,462 | 21,587 | 42,126 | |||||||||||
| Loss from operations | (5,898 | ) | (9,406 | ) | (21,384 | ) | (41,922 | ) | |||||||
| Interest income, net | 228 | 193 | 643 | 560 | |||||||||||
| Other income (expense), net | (7 | ) | (3 | ) | (5 | ) | (37 | ) | |||||||
| Loss from equity method investment | (2 | ) | (140 | ) | (6 | ) | (1,015 | ) | |||||||
| Loss before income taxes | (5,679 | ) | (9,356 | ) | (20,752 | ) | (42,414 | ) | |||||||
| Income tax expense | — | 11 | — | 33 | |||||||||||
| Net loss | $ | (5,679 | ) | $ | (9,367 | ) | $ | (20,752 | ) | $ | (42,447 | ) | |||
| Net loss per share—basic and diluted | $ | (0.16 | ) | $ | (0.26 | ) | $ | (0.58 | ) | $ | (1.19 | ) | |||
| Weighted average number of shares used in net loss per share—basic and diluted | 35,790 | 35,687 | 35,759 | 35,664 | |||||||||||
| Net loss | $ | (5,679 | ) | $ | (9,367 | ) | $ | (20,752 | ) | $ | (42,447 | ) | |||
| Other comprehensive loss: | |||||||||||||||
| Unrealized gains on available-for-sale securities | 5 | 27 | 24 | 38 | |||||||||||
| Comprehensive loss | $ | (5,674 | ) | $ | (9,340 | ) | $ | (20,728 | ) | $ | (42,409 | ) | |||
| Nine Months Ended September 30, | |||||||
| 2018 | 2017 | ||||||
| Cash flows from operating activities: | |||||||
| Net loss | $ | (20,752 | ) | (42,447 | ) | ||
| Adjustments to reconcile net loss to net cash used in operating activities: | |||||||
| Depreciation and amortization | 633 | 1,311 | |||||
| Impairment of property and equipment related to restructuring | 2,203 | 250 | |||||
| Gain on sale of property, plant and equipment | (66 | ) | — | ||||
| Amortization of (discount) premium on debt securities | (176 | ) | 101 | ||||
| Stock-based compensation expense | 1,259 | 6,377 | |||||
| Issuance of common stock for director fees | 82 | 102 | |||||
| Net loss on equity method investment | 6 | 1,015 | |||||
| Changes in operating assets and liabilities: | |||||||
| Prepaid expenses and other assets | (473 | ) | 635 | ||||
| Other assets | 24 | — | |||||
| Accounts payable | (1,638 | ) | 517 | ||||
| Accrued expenses, deferred rent and other non-current liabilities | (2,594 | ) | (5,440 | ) | |||
| Net cash used in operating activities | (21,492 | ) | (37,579 | ) | |||
| Cash flows from investing activities: | |||||||
| Investment in joint venture | — | (550 | ) | ||||
| Purchases of plant and equipment | — | (112 | ) | ||||
| Maturities of short-term investments | 70,759 | 67,839 | |||||
| Proceeds from sale of property, plant and equipment | 438 | — | |||||
| Purchases of short-term investments | (33,491 | ) | (48,403 | ) | |||
| Net cash provided by investing activities | 37,706 | 18,774 | |||||
| Cash flows from financing activities: | |||||||
| Net cash provided by financing activities | — | — | |||||
| Net increase (decrease) in cash, cash equivalents and restricted cash | 16,214 | (18,805 | ) | ||||
| Cash, cash equivalents and restricted cash at beginning of period | 16,492 | 44,369 | |||||
| Cash, cash equivalents and restricted cash at end of period | $ | 32,706 | $ | 25,564 | |||
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
Unless the context suggests otherwise, references in this Quarterly Report on Form 10‑Q, or Quarterly Report, to amounts reported within the condensed consolidated balance sheets.
| As of September 30, | As of September 30, | ||||||
| 2018 | 2017 | ||||||
| Cash and cash equivalents | $ | 31,917 | $ | 24,775 | |||
| Restricted cash | 789 | 789 | |||||
| Total cash, cash equivalents and restricted cash | $ | 32,706 | $ | 25,564 | |||
This Quarterly Report on Form 10‑Q contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements reflect our plans, estimates and beliefs and include, but are not limited to, statements about our plans to develop and commercialize our product candidates; the progress and timing of our ongoing and planned clinical trials for our product candidates, including the timing of topline results from the Phase 2b portion of our Phase 2b/3 clinical trial of livoletide in Prader Willi syndrome (“PWS”) under which Millendo will merge with OvaSciencepatients and the timeline for our Phase 2b clinical study of nevanimibe in an all-stock transaction. A wholly owned subsidiary of OvaScience will merge withcongenital adrenal hyperplasia, the potential and into Millendo, with Millendo survivingtiming for a neurokinin 3 receptor antagonist (MLE-301) as a wholly owned subsidiarypotential treatment of OvaScience (the "Merger"). OvaSciencevasomotor symptoms to enter clinical trials; the potential advantages of our discontinuation of our Phase 2 clinical study of nevanimibe in Cushing's syndrome; the timing of and our ability to obtain and maintain regulatory approvals for our product candidates; and our estimates regarding future revenue, if any, future expenses, the funding of our operations, including whether our existing cash, cash equivalents, marketable securities, and restricted cash will be sufficient to fund our current operating plans into the fourth quarter of 2020 and through the topline results of the Phase 2b portion of our Phase 2b/3 clinical trial of livoletide in PWS patients, as well as our future capital requirements and needs for additional financing. These statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “anticipates,” “believes,” “could,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “would” and similar expressions intended to identify forward-looking statements. Forward-looking statements reflect our current views with respect to future events and are based on assumptions and subject to risks and uncertainties. Because of these risks and uncertainties, the forward-looking events and circumstances discussed in this report may not transpire. These risks and uncertainties include, but are not limited to, the risks included in this Quarterly Report on Form 10‑Q under Part II, Item 1A, “Risk Factors.”
Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent our estimates and assumptions only as of the date of this document. You should read this document with the understanding that our actual future results may be materially different from what we expect. Except as required by law, we do not undertake any obligation to update or revise any forward-looking statements contained in this report, whether as a result of new information, future events or otherwise.
3
PART I – Financial Information
MILLENDO THERAPEUTICS, INC.
(Unaudited)
(in thousands except share and per share amounts)
|
|
|
|
|
|
|
|
| June 30, |
| December 31, | ||
|
| 2019 |
| 2018 | ||
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
| $ | 55,274 |
| $ | 73,286 |
Short-term restricted cash |
|
| 699 |
|
| 45 |
Marketable securities |
|
| — |
|
| 4,385 |
Prepaid expenses and other current assets |
|
| 6,416 |
|
| 3,373 |
Refundable tax credit |
|
| 2,123 |
|
| 2,333 |
Total current assets |
|
| 64,512 |
|
| 83,422 |
Long-term restricted cash |
|
| 658 |
|
| 439 |
Operating lease right of use assets |
|
| 2,349 |
|
| — |
Other assets |
|
| 805 |
|
| 213 |
Total assets |
| $ | 68,324 |
| $ | 84,074 |
Liabilities and stockholders’ equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Current portion of debt |
| $ | 199 |
| $ | 189 |
Accounts payable |
|
| 3,091 |
|
| 1,998 |
Accrued expenses |
|
| 6,147 |
|
| 7,630 |
Operating lease liabilities — current |
|
| 1,492 |
|
| — |
Total current liabilities |
|
| 10,929 |
|
| 9,817 |
Debt, net of current portion |
|
| 278 |
|
| 383 |
Operating lease liabilities |
|
| 1,865 |
|
| — |
Other liabilities |
|
| 227 |
|
| 752 |
Total liabilities |
|
| 13,299 |
|
| 10,952 |
Commitments and contingencies (Note 6) |
|
|
|
|
|
|
Stockholders’ equity: |
|
|
|
|
|
|
Preferred stock, $0.001 par value: 5,000,000 shares authorized; no shares issued and outstanding |
|
| — |
|
| — |
Common stock, $0.001 par value: 100,000,000 shares authorized; 13,412,058 and 13,357,999 shares issued and outstanding at June 30, 2019 and December 31, 2018, respectively |
|
| 13 |
|
| 13 |
Additional paid-in capital |
|
| 237,136 |
|
| 234,876 |
Accumulated deficit |
|
| (184,323) |
|
| (164,086) |
Accumulated other comprehensive income |
|
| 140 |
|
| 148 |
Total stockholders’ equity attributable to Millendo Therapeutics, Inc. |
|
| 52,966 |
|
| 70,951 |
Equity attributable to noncontrolling interests |
|
| 2,059 |
|
| 2,171 |
Total stockholders’ equity |
|
| 55,025 |
|
| 73,122 |
Total liabilities and stockholders’ equity |
| $ | 68,324 |
| $ | 84,074 |
See accompanying notes to unaudited interim consolidated financial statements
4
MILLENDO THERAPEUTICS, INC.
Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)
(in thousands except share and per share amounts)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Three Months Ended |
| Six Months Ended | ||||||||
|
| June 30, |
| June 30, | ||||||||
|
| 2019 |
| 2018 |
| 2019 |
| 2018 | ||||
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
| $ | 5,981 |
| $ | 3,200 |
| $ | 12,185 |
| $ | 5,969 |
General and administrative |
|
| 4,179 |
|
| 1,786 |
|
| 8,632 |
|
| 3,405 |
Loss from operations |
|
| 10,160 |
|
| 4,986 |
|
| 20,817 |
|
| 9,374 |
Other expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Interest expense (income), net |
| $ | (313) |
| $ | (6) |
| $ | (628) |
| $ | (15) |
Other loss |
|
| 24 |
|
| 62 |
|
| 48 |
|
| 69 |
Net loss |
| $ | (9,871) |
| $ | (5,042) |
| $ | (20,237) |
| $ | (9,428) |
Net loss attributable to noncontrolling interest |
|
| — |
|
| 196 |
|
| — |
|
| 321 |
Net loss attributable to common stockholders |
| $ | (9,871) |
| $ | (4,846) |
| $ | (20,237) |
| $ | (9,107) |
Net loss per share of common stock, basic and diluted |
| $ | (0.74) |
| $ | (6.82) |
| $ | (1.51) |
| $ | (12.82) |
Weighted‑average shares of common stock outstanding, basic and diluted |
|
| 13,379,842 |
|
| 710,390 |
|
| 13,368,981 |
|
| 710,390 |
Other comprehensive income (loss): |
|
|
|
|
|
|
|
|
|
|
|
|
Foreign currency translation adjustment |
| $ | 1 |
| $ | 28 |
| $ | (8) |
| $ | 47 |
Comprehensive loss |
| $ | (9,870) |
| $ | (4,818) |
| $ | (20,245) |
| $ | (9,060) |
Comprehensive income attributable to noncontrolling interest |
| $ | — |
| $ | 4 |
| $ | — |
| $ | 7 |
Comprehensive loss attributable to Millendo Therapeutics, Inc. |
| $ | (9,870) |
| $ | (4,822) |
| $ | (20,245) |
| $ | (9,067) |
See accompanying notes to unaudited interim consolidated financial statements
5
MILLENDO THERAPEUTICS, INC.
Consolidated statements of convertible preferred stock, redeemable noncontrolling interests and stockholders’
(deficit) equity
(Unaudited)
(in thousands except share amounts)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Three Months Ended June 30, 2019 | |||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Total |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Stockholders’ |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| (Deficit) |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Equity |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
| Accumulated |
| attributable |
| Total Equity |
| Total | ||||
|
|
|
|
|
|
| Additional |
|
|
|
| Other |
| to Millendo |
| Attributable to |
| Stockholders’ | |||||
|
| Common Stock |
| Paid-in |
| Accumulated |
| Comprehensive |
| Therapeutics, |
| Noncontrolling |
| (Deficit) | |||||||||
|
| Shares |
| Amount |
| Capital |
| Deficit |
| Income |
| Inc. |
| Interests |
| Equity | |||||||
Balance at April 1, 2019 |
| 13,357,999 |
| $ | 13 |
| $ | 235,815 |
| $ | (174,452) |
| $ | 139 |
| $ | 61,515 |
| $ | 2,171 |
| $ | 63,686 |
Exercise of stock options |
| 45,947 |
|
| — |
|
| 166 |
|
| — |
|
| — |
|
| 166 |
|
| — |
|
| 166 |
Exercise of BSPCE warrants |
| 8,112 |
|
| — |
|
| 160 |
|
| — |
|
| — |
|
| 160 |
|
| (112) |
|
| 48 |
Stock-based compensation expense |
| — |
|
| — |
|
| 995 |
|
| — |
|
| — |
|
| 995 |
|
| — |
|
| 995 |
Foreign currency translation adjustment |
| — |
|
| — |
|
| — |
|
| — |
|
| 1 |
|
| 1 |
|
| — |
|
| 1 |
Net income (loss) |
| — |
|
| — |
|
| — |
|
| (9,871) |
|
| — |
|
| (9,871) |
|
| — |
|
| (9,871) |
Balance at June 30, 2019 |
| 13,412,058 |
| $ | 13 |
| $ | 237,136 |
| $ | (184,323) |
| $ | 140 |
| $ | 52,966 |
| $ | 2,059 |
| $ | 55,025 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Six Months Ended June 30, 2019 | |||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Total |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Stockholders’ |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| (Deficit) |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Equity |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
| Accumulated |
| attributable |
| Total Equity |
| Total | ||||
|
|
|
|
|
|
| Additional |
|
|
|
| Other |
| to Millendo |
| Attributable to |
| Stockholders’ | |||||
|
| Common Stock |
| Paid-in |
| Accumulated |
| Comprehensive |
| Therapeutics, |
| Noncontrolling |
| (Deficit) | |||||||||
|
| Shares |
| Amount |
| Capital |
| Deficit |
| Income |
| Inc. |
| Interests |
| Equity | |||||||
Balance at January 1, 2019 |
| 13,357,999 |
| $ | 13 |
| $ | 234,876 |
| $ | (164,086) |
| $ | 148 |
| $ | 70,951 |
| $ | 2,171 |
| $ | 73,122 |
Exercise of stock options |
| 45,947 |
|
| — |
|
| 166 |
|
| — |
|
| — |
|
| 166 |
|
| — |
|
| 166 |
Exercise of BSPCE warrants |
| 8,112 |
|
| — |
|
| 160 |
|
| — |
|
| — |
|
| 160 |
|
| (112) |
|
| 48 |
Stock-based compensation expense |
| — |
|
| — |
|
| 1,934 |
|
| — |
|
| — |
|
| 1,934 |
|
| — |
|
| 1,934 |
Foreign currency translation adjustment |
| — |
|
| — |
|
| — |
|
| — |
|
| (8) |
|
| (8) |
|
| — |
|
| (8) |
Net income (loss) |
| — |
|
| — |
|
| — |
|
| (20,237) |
|
| — |
|
| (20,237) |
|
| — |
|
| (20,237) |
Balance at June 30, 2019 |
| 13,412,058 |
| $ | 13 |
| $ | 237,136 |
| $ | (184,323) |
| $ | 140 |
| $ | 52,966 |
| $ | 2,059 |
| $ | 55,025 |
6
MILLENDO THERAPEUTICS, INC.
Consolidated statements of convertible preferred stock, redeemable noncontrolling interests and stockholders’
(deficit) equity (continued)
(Unaudited)
(in thousands except share amounts)
|
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|
|
| Three Months Ended June 30, 2018 | |||||||||||||||||||||||||||||||||||
|
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|
|
| Stockholders’ Equity (Deficit) | ||||||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Total |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Stockholders’ |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| (Deficit) |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Equity |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Accumulated |
| attributable |
| Total Equity |
| Total | ||||
|
| Convertible |
| Redeemable |
|
|
|
|
|
|
|
|
|
|
|
| Additional |
|
|
|
| Other |
| to Millendo |
| Attributable to |
| Stockholders’ | |||||||||
|
| Preferred Stock |
| Noncontrolling |
|
| Common Stock |
| Common-1 Stock |
| Paid-in |
| Accumulated |
| Comprehensive |
| Therapeutics, |
| Noncontrolling |
| (Deficit) | ||||||||||||||||
|
| Shares |
| Amount |
| Interests |
|
| Shares |
| Amount |
| Shares |
| Amount |
| Capital |
| Deficit |
| Income |
| Inc. |
| Interests |
| Equity | ||||||||||
Balance at April 1, 2018 |
| 90,848,515 |
| $ | 132,922 |
| $ | 10,462 |
|
| 246,347 |
| $ | — |
| 464,043 |
| $ | — |
| $ | 6,369 |
| $ | (141,155) |
| $ | 24 |
| $ | (134,762) |
| $ | 2,171 |
| $ | (132,591) |
Stock-based compensation expense |
| — |
|
| — |
|
| — |
|
| — |
|
| — |
| — |
|
| — |
|
| 159 |
|
| — |
|
| — |
|
| 159 |
|
| — |
|
| 159 |
Foreign currency translation adjustment |
| — |
|
| — |
|
| 4 |
|
| — |
|
| — |
| — |
|
| — |
|
| — |
|
| — |
|
| 24 |
|
| 24 |
|
| — |
|
| 24 |
Net income (loss) |
| — |
|
| — |
|
| (196) |
|
| — |
|
| — |
| — |
|
| — |
|
| — |
|
| (4,846) |
|
| — |
|
| (4,846) |
|
| — |
|
| (4,846) |
Balance at June 30, 2018 |
| 90,848,515 |
| $ | 132,922 |
| $ | 10,270 |
|
| 246,347 |
| $ | — |
| 464,043 |
| $ | — |
| $ | 6,528 |
| $ | (146,001) |
| $ | 48 |
| $ | (139,425) |
| $ | 2,171 |
| $ | (137,254) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Six Months Ended June 30, 2018 | |||||||||||||||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
| Stockholders’ Equity (Deficit) | ||||||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Total |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Stockholders’ |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| (Deficit) |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Equity |
|
|
|
|
|
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Accumulated |
| attributable |
| Total Equity |
| Total | ||||
|
| Convertible |
| Redeemable |
|
|
|
|
|
|
|
|
|
|
|
| Additional |
|
|
|
| Other |
| to Millendo |
| Attributable to |
| Stockholders’ | |||||||||
|
| Preferred Stock |
| Noncontrolling |
|
| Common Stock |
| Common-1 Stock |
| Paid-in |
| Accumulated |
| Comprehensive |
| Therapeutics, |
| Noncontrolling |
| (Deficit) | ||||||||||||||||
|
| Shares |
| Amount |
| Interests |
|
| Shares |
| Amount |
| Shares |
| Amount |
| Capital |
| Deficit |
| Income |
| Inc. |
| Interests |
| Equity | ||||||||||
Balance at January 1, 2018 |
| 90,848,515 |
| $ | 132,922 |
| $ | 10,584 |
|
| 246,347 |
| $ | — |
| 464,043 |
| $ | — |
| $ | 6,192 |
| $ | (136,894) |
| $ | 8 |
| $ | (130,694) |
| $ | 2,171 |
| $ | (128,523) |
Stock-based compensation expense |
| — |
|
| — |
|
| — |
|
| — |
|
| — |
| — |
|
| — |
|
| 336 |
|
| — |
|
| — |
|
| 336 |
|
| — |
|
| 336 |
Foreign currency translation adjustment |
| — |
|
| — |
|
| 7 |
|
| — |
|
| — |
| — |
|
| — |
|
| — |
|
| — |
|
| 40 |
|
| 40 |
|
| — |
|
| 40 |
Net income (loss) |
| — |
|
| — |
|
| (321) |
|
| — |
|
| — |
| — |
|
| — |
|
| — |
|
| (9,107) |
|
| — |
|
| (9,107) |
|
| — |
|
| (9,107) |
Balance at June 30, 2018 |
| 90,848,515 |
| $ | 132,922 |
| $ | 10,270 |
|
| 246,347 |
| $ | — |
| 464,043 |
| $ | — |
| $ | 6,528 |
| $ | (146,001) |
| $ | 48 |
| $ | (139,425) |
| $ | 2,171 |
| $ | (137,254) |
See accompanying notes to unaudited interim consolidated financial statements
7
MILLENDO THERAPEUTICS, INC.
Consolidated Statements of Cash Flows
(Unaudited)
(in thousands)
|
|
|
|
|
|
|
|
| Six Months Ended | ||||
|
| June 30, | ||||
|
| 2019 |
| 2018 | ||
Operating activities: |
|
|
|
|
|
|
Net loss |
| $ | (20,237) |
| $ | (9,428) |
Adjustments to reconcile net loss to net cash used in operating activities: |
|
|
|
|
|
|
Depreciation |
|
| 19 |
|
| 15 |
Stock‑based compensation expense |
|
| 1,934 |
|
| 336 |
Amortization of operating lease right of use assets |
|
| (206) |
|
| — |
Unrealized foreign currency loss |
|
| — |
|
| 61 |
Changes in operating assets and liabilities: |
|
|
|
|
|
|
Prepaid expenses and other current assets |
|
| (2,910) |
|
| (761) |
Other assets |
|
| 62 |
|
| — |
Accounts payable |
|
| 1,056 |
|
| (367) |
Accrued expenses and other liabilities |
|
| (892) |
|
| 446 |
Cash used in operating activities |
|
| (21,174) |
|
| (9,698) |
Investing activities: |
|
|
|
|
|
|
Purchase of property and equipment |
|
| (277) |
|
| (7) |
Proceeds from sale of marketable securities |
|
| 4,385 |
|
| — |
Net cash paid in Alizé asset purchase |
|
| — |
|
| (524) |
Cash provided by (used in) investing activities |
|
| 4,108 |
|
| (531) |
Financing activities: |
|
|
|
|
|
|
Repayment of debt |
|
| (90) |
|
| (84) |
Payment of financing costs |
|
| (162) |
|
| (482) |
Proceeds from option and BSPCE warrant exercises |
|
| 214 |
|
| — |
Cash used in financing activities |
|
| (38) |
|
| (566) |
Effect of foreign currency exchange rate changes on cash |
|
| (35) |
|
| (4) |
Net decrease in cash, cash equivalents and restricted cash |
|
| (17,139) |
|
| (10,799) |
Cash, cash equivalents and restricted cash at beginning of period |
|
| 73,770 |
|
| 17,623 |
Cash, cash equivalents and restricted cash at end of period |
| $ | 56,631 |
| $ | 6,824 |
Supplemental schedule of non‑cash investing and financing activities: |
|
|
|
|
|
|
Right of use assets acquired under operating leases |
| $ | 1,840 |
| $ | — |
Financing costs in accounts payable and accrued expenses |
| $ | 83 |
| $ | 159 |
See accompanying notes to unaudited interim consolidated financial statements
8
MILLENDO THERAPEUTICS, INC.
Notes to Unaudited Interim Consolidated Financial Statements
1. Organization and description of business
Description of Business
Millendo believe that the Merger, if finalized, will result inTherapeutics, Inc., a specialtyDelaware corporation, together with its subsidiaries, is a late‑stage biopharmaceutical company primarily focused on developing novel treatments for orphan endocrine diseases where current therapies do not exist or are insufficient. The transactionCompany is currently advancing three product candidates. The Company’s most advanced product candidate, livoletide (AZP‑531), is a potential treatment for Prader‑Willi syndrome (“PWS”), a rare and complex genetic endocrine disease characterized by hyperphagia, or insatiable hunger. The Company is also developing nevanimibe (ATR‑101) as a potential treatment for patients with classic congenital adrenal hyperplasia (“CAH”), a rare, monogenic adrenal disease that requires lifelong treatment with exogenous cortisol, often at high doses. The Company has added a new product candidate to its research and development pipeline: a neurokinin 3 receptor (NK3R) antagonist (MLE-301), as a potential treatment of vasomotor symptoms (“VMS”), defined as hot flashes and night sweats in menopausal women.
The Company has been investigating nevanimibe (ATR-101) as a potential treatment for patients with endogenous Cushing’s syndrome (“CS”), a rare endocrine disease characterized by excessive cortisol production from the adrenal glands. As a result of slower than anticipated enrollment in its CS Phase 2 clinical trial, the Company elected to discontinue this trial in August 2019, suspend development of nevanimibe for the treatment of CS, and focus its resources on other programs in its research and development pipeline.
The Company’s operations to date have focused on acquiring technology and assets, conducting preclinical studies and clinical trials, organization and staffing, business planning, and raising capital. The Company does not have any products approved by the Boards of Directors of both companies.for sale and has not generated any revenue from product sales. The Merger is expected to close in the fourth quarter of 2018,Company’s product candidates are subject to long development cycles and the Company may be unsuccessful in its efforts to develop, obtain regulatory approval of OvaScience shareholders at a special shareholder meeting to be held on December 4, 2018, as well as other customary conditions.for or market its product candidates.
The Company is subject to a number of risks similar to other life science companies, including, but not limited to, risks associated with clinical and preclinical development, the need to developobtain adequate additional funding for the ongoing and obtain marketing approval for certainplanned clinical development of our fertility treatments, competitors developing new technological innovations,its product candidates. Because of the need to successfully commercializenumerous risks and gain market acceptance of our fertility treatmentsuncertainties associated with pharmaceutical products and protection of proprietary technology, anddevelopment, the outcome of our exploration of strategic alternatives. If we do not successfully develop and commercialize any of our fertility treatments, we will beCompany is unable to accurately predict the timing or amount of funds required to complete development of its product candidates, and costs could exceed the Company’s expectations for a number of reasons, including reasons beyond the Company’s control.
Merger with OvaScience
In December 2018, OvaScience, Inc., a Delaware corporation (“OvaScience”), now known as Millendo Therapeutics, Inc. (the “Company”), completed its merger (the “Merger”) with privately-held Millendo Therapeutics, Inc. (“Private Millendo”), in accordance with the terms of the Agreement and Plan of Merger and Reorganization, dated August 8, 2018, as amended on September 25, 2018 and November 1, 2018 (the “Merger Agreement”), whereby Orion Merger Sub, Inc., a Delaware corporation and wholly-owned subsidiary of OvaScience (the “Merger Sub”), merged with and into Private Millendo, with Private Millendo continuing as a wholly owned subsidiary of OvaScience.
Under the terms of the Merger Agreement, OvaScience issued shares of its common stock to Private Millendo’s stockholders, at an exchange ratio of 0.0744 shares of OvaScience common stock, for each share of Private Millendo common stock outstanding immediately prior to the Merger. OvaScience also assumed all of the stock options outstanding under the Private Millendo 2012 Equity Incentive Plan, as amended (the “Private Millendo Plan”), with such stock options henceforth representing the right to purchase a number of shares of OvaScience’s common stock equal to 0.0744 multiplied by the number of shares of Private Millendo common stock previously represented by such options.
9
The Company’s shares of common stock listed on The Nasdaq Capital Market, previously trading through the close of business on Friday, December 7, 2018 (the “Merger Date”) under the ticker symbol “OVAS,” commenced trading on The Nasdaq Capital Market, under the ticker symbol “MLND,” on Monday, December 10, 2018.
The Merger was accounted for as a reverse acquisition and recapitalization, with Private Millendo being treated as the accounting acquirer. As such, the results of operations and cash flows prior to the Merger Date, relate to Private Millendo and its subsidiaries. Subsequent to the Merger Date the information relates to the consolidated entities of Millendo Therapeutics, Inc. All share and per share amounts in the unaudited interim consolidated financial statements and related notes have been retroactively adjusted, where applicable, for all periods presented to give effect to the exchange ratio applied in connection with the Merger.
Liquidity
The Company has incurred net losses since inception and it expects to generate treatment revenue or achieve profitability.losses from operations for the foreseeable future primarily due to research and development costs for its potential product candidates. As of SeptemberJune 30, 2018, we2019, the Company had cash, cash equivalents, marketable securities and restricted cash of $56.6 million and an accumulated deficit of approximately $322.2$184.3 million.
In April 2019, the Company entered into an “at-the-market” (“ATM”) equity distribution agreement with Citigroup Global Markets Inc. acting as sole agent with an aggregate offering value of up to $50.0 million, which allows the Company to sell its common shares through the facilities of the Nasdaq Capital Market. Subject to the terms of the equity distribution agreement, the Company is able to determine, at its sole discretion, the timing and number of shares to be sold under this ATM facility. No common shares have incurred annual net operating lossesbeen issued to date under the Company’s ATM equity distribution agreement.
The Company will likely require additional capital in each year since our inception. We have generated limited treatment revenues relatedthe future through equity or debt financings, partnerships, collaborations, or other sources to our primary business purposecarry out the Company’s planned development activities. If additional capital is not secured when required, the Company may need to delay or curtail its operations until such funding is received. Various internal and have financed our operations primarily through private placementsexternal factors will affect whether and when the Company’s product candidates become approved drugs. The regulatory approval and market acceptance of our preferred stock, which was subsequently converted to common stock,the Company’s proposed future products (if any), length of time and public salescost of our common stockdeveloping and interest income earned oncommercializing these product candidates and/or failure of them at any stage of the drug approval process will materially affect the Company’s financial condition and future operations. The Company believes its cash, cash equivalents, marketable securities and short-term investments balances.
2.Basis of presentation and summary of significant accounting policies
Basis of presentation
The accompanying unaudited interim consolidated financial statements include the accounts of Millendo Therapeutics, Inc. and its subsidiaries, and all intercompany amounts have been eliminated. The unaudited condensedinterim consolidated financial statements have been prepared by us in accordanceconformity with U.S. generally accepted accounting principles generally accepted(“GAAP”). Any reference in these notes to applicable guidance is meant to refer to GAAP as found in the United StatesAccounting Standards Codification (“ASC”) and Accounting Standards Updates (“ASU”) of Americathe Financial Accounting Standards Board (“US GAAP”FASB”). These condensed
Unaudited interim financial statements
The Company has prepared the accompanying unaudited interim consolidated financial statements have been omitted. In the opinion of management, thebased on Securities and Exchange Commission (“SEC”) rules that permit reduced disclosure for interim periods. These unaudited interim consolidated financial statements reflectinclude, in the Company’s opinion, all adjustments, which with the exception of restructuring accruals described in Note 9, consistedconsisting only of normal and recurring adjustments that the Company considers necessary for thea fair presentation of ourits consolidated financial position at September 30, 2018,and results of our operations for these periods. The Company’s historical results are not necessarily indicative of the threeresults
10
to be expected in the future and nine months ended September 30, 2018 and 2017 and cash flows for the nine months ended September 30, 2018 and 2017.
The accompanying unaudited interim consolidated financial statements should be read in conjunction with the annual audited consolidated financial statements and related notes as of and for the year ended December 31, 2017, which are contained2018 included in ourthe Company’s Annual Report on Form 10-K for the year ended December 31, 2017 (“2017 Annual Report on Form 10-K”) that was10‑K filed with the SecuritiesSEC on April 1, 2019. Since the date of such financial statements, there have been no changes to the Company’s significant accounting policies except as follows:
Leases
In February 2016, the FASB issued ASU 2016‑02, Leases (Topic 842), which requires the Company as the lessee to recognize most leases on the balance sheet thereby resulting in the recognition of right of use assets and Exchange Commissionlease obligations for those leases currently classified as operating leases. ASU 2016‑02 became effective for the Company on January 1, 2019.
The Company adopted ASU 2016‑02 using a modified retrospective transition approach as of January 1, 2019 and will not restate its comparative period financial information for effects of the standard or make the new required lease disclosures for periods before the date of adoption. The Company has elected to adopt the package of transition practical expedients and, therefore, have not reassessed (1) whether existing or expired contracts contain a lease, (2) lease classification for existing or expired leases or (3) the accounting for initial direct costs that were previously capitalized. As a result of the adoption of ASC 842, the Company recognized an operating lease liability of $2.0 million based on the present value of the minimum rental payments of the leases and a corresponding operating lease right of use (“SEC”ROU”) asset of $0.9 million within the Company’s consolidated balance sheet. The Company’s ROU asset is exclusive of any early lease termination obligations whereby future contractual lease payments exceed estimated sublease income.
Under ASC 842, the Company determines if an arrangement is a lease at contract inception. A lease exists when a contract conveys to the customer the right to control the use of identified property, plant, or equipment for a period of time in exchange for consideration. The definition of a lease embodies two conditions: (1) there is an identified asset in the contract that is land or a depreciable asset (i.e., property, plant, and equipment), and (2) the customer has the right to control the use of the identified asset.
The lease liabilities are initially and subsequently measured at the present value of the unpaid lease payments at the lease commencement date. When readily determinable, the Company uses the implicit rate in determining the present value of lease payments. When leases do not provide an implicit rate, the Company uses its incremental borrowing rate based on March 15,the information available at the lease commencement date, including the lease term.
The ROU asset is initially measured at cost, which comprises the initial amount of the lease liability adjusted for lease payments made at or before the lease commencement date, plus any initial direct costs incurred less any lease incentives received. For operating leases, the ROU asset is subsequently measured throughout the lease term at the carrying amount of the lease liability, plus initial direct costs, plus (minus) any prepaid (accrued) lease payments, less the unamortized balance of lease incentives received. Lease expense for lease payments is recognized on a straight-line basis over the lease term.
The Company has noncancelable operating leases for office and laboratory space which have remaining lease terms between two and seven years. In connection with the Merger, the Company assumed a sublease agreement for office and laboratory space located in Waltham, Massachusetts. The term of the sublease expires in November 2020.In February 2019 and October 2018, the Company entered into two additional noncancelable operating leases for office space in Ann Arbor, Michigan for the Company’s headquarters; one that the Company took possession of in April 2019, and the other that the Company took possession of in July 2019, respectively. In April 2019, the Company entered into a lease agreement for office space in Lexington, Massachusetts to expand its operations.
As of June 30, 2019, the operating lease ROU asset and the operating lease liabilities were $2.3 million and $3.4 million, respectively. The discount rate used to account for the Company's operating leases under ASC 842 is the Company’s estimated incremental borrowing rate of 7.0%. The Company has options to extend certain of its leases for another five to nine years. These options to extend were not recognized as part of the Company’s measurement of the ROU assets
11
and operating lease liabilities for the three and six months ended June 30, 2019 nor were the future payments related to the lease the Company entered into in October 2018.
Rent expense related to the Company's operating leases was approximately $156,000 and $46,000 for the three months ended June 30, 2019 and 2018, respectively, and approximately $223,000 and $94,000 for the six months ended June 30, 2019 and 2018, respectively. Cash paid for amounts included in the measurement of the lease liabilities was approximately $0.4 million and $0.5 million and the Company received approximately $85,000 and $0.2 million in sublease payments related to its Waltham, Massachusetts lease during the three and six months ended June 30, 2019, respectively. The weighted average remaining term of the Company’s noncancelable operating leases is 3.49 years. Future minimum rental payments under the Company’s noncancelable operating leases at June 30, 2019 is as follows (amounts in thousands):
|
|
|
|
2019 |
| $ | 759 |
2020 |
|
| 1,448 |
2021 |
|
| 440 |
2022 |
|
| 451 |
2023 |
|
| 463 |
Thereafter |
|
| 196 |
Total |
|
| 3,757 |
Present Value Adjustment |
|
| (400) |
Lease liability at June 30, 2019 |
| $ | 3,357 |
Use of estimates and summary
The preparation of significant accounting policies
Restricted cash
Restricted cash relates to amounts used to secure the Company’s credit card facility balances held on deposit with major financial institutions, to collateralize a letter of Significant Accounting Policies,”credit in our 2017 Annual Report on Form 10-K.
Deferred offering costs
The Company capitalizes costs that are directly associated with in-process equity financings until such financings are consummated, at which time such costs are recorded against the gross proceeds from the applicable financing. If a financing is abandoned, deferred offering costs are expensed. As of June 30, 2019, deferred offering costs were $0.2 million.
Net loss per share
Basic loss per share of common stock is computed by dividing net loss attributable to common stockholders by the weighted‑average number of shares of common stock (including shares of common‑1 stock during the three and six months ended June 30, 2018) outstanding during each period. Diluted loss per share of common stock includes the effect, if any, from the potential exercise or conversion of securities, such as convertible preferred stock, preferred stock warrants, restricted stock, and stock options, which would result in the issuance of incremental shares of common stock. In computing the basic and diluted net loss per common share, are calculated by dividing net loss by the weighted weighted‑average number of shares outstanding during of common stock remains
12
the period. Potentiallysame for both calculations due to the fact that when a net loss exists, dilutive shares including outstanding stock options and unvested restricted stock units, are onlynot included in the calculation of diluted net loss per share when their effectas the impact is anti‑dilutive.
The amounts in the table below werefollowing potentially dilutive securities have been excluded from the calculationcomputation of diluted net loss per share due to their anti-dilutive effect (in thousands):
| As of September 30, | |||||
| 2018 | 2017 | ||||
| Outstanding stock options and restricted stock units | 5,160 | 6,468 | |||
|
|
|
|
|
|
| June 30, | ||
|
| 2019 |
| 2018 |
Stock options |
| 2,640,077 |
| 661,490 |
Convertible preferred stock |
| — |
| 6,759,109 |
Common stock warrants |
| 17,125 |
| — |
Preferred stock warrants |
| — |
| 17,125 |
BSA and BSPCE warrants |
| 148,607 |
| 156,719 |
|
| 2,805,809 |
| 7,594,443 |
Recent accounting pronouncements
In August 2018, the Financial Accounting Standards Board ("FASB")FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): 2018‑13, Disclosure Framework—Framework - Changes to the Disclosure Requirements for Fair Value Measurement.Measurement (Topic 820). ASU 2018-13 eliminates, adds and modifies2018‑13 resulted in certain disclosure requirements formodifications to fair value measurements as part of its disclosure framework project. This update is effective for annual and interim periods beginning after December 15, 2019 with early adoption permitted. We are currently assessing the impact ASU 2018-13 will have on our consolidated financial statements and footnotemeasurement disclosures, thereto.
In August 2016, the FASB issued ASU No. 2016-15, 2016‑15, Statement of Cash Flows (Topic 230): Classification of Certain Cash Receipts and Cash Payments. ASU 2016-15 requiresPayments, which makes eight targeted changes to how cash receipts and cash payments are presented and classified in the presentationstatement of debt prepayment or debt extinguishment costs, contingent consideration payments made after a business combination, proceeds from the settlement of insurance claims, proceeds from the settlement of corporate-owned life insurance policiescash flows. This standard was effective January 1, 2019 and distributions received from equity method investees. This update is effective for annual and interim periods beginning after December 15, 2017 usingrequired adoption on a retrospective transition methodbasis unless it was impracticable to each period presented. Weapply, in which case the Company would be required to apply the amendments prospectively as of the earliest date practicable. The Company adopted this standard as of January 1, 2018 with nowhich did not have a material impact on ourits consolidated financial statements.
In June 2016, the FASB issued ASU No. 2016-13, Measurement of2016‑13, Financial Instruments – Credit Losses on (Topic 326), which replaces the incurred loss impairment methodology in current GAAP with a methodology that reflects expected credit losses and requires consideration of a broader range of reasonable and supportable information to inform credit loss estimates. Additionally, ASU 2016‑13 requires a financial asset measured at amortized cost basis to be presented at the net amount expected to be collected through the use of an allowance of expected credit losses. In May 2019, the FASB issued ASU No. 2019-05, Financial Instruments - Credit Losses (Topic 326): Targeted Transition Relief, which amends ASU 2016-13 by providing entities with an option to irrevocably elect the impairment model by requiring entitiesfair value option to use a forward-looking approach basedbe applied on expected losses rather than incurred lossesan instrument-by-instrument basis for eligible financial instruments that are within the scope of Topic 326. The fair value option election does not apply to estimate credit losses on certain types of financial instruments. This may result in the earlier recognition of allowances for losses. The guidanceheld-to-maturity debt securities. ASU 2016‑13, as amended, is effective for fiscal years beginning after December 15, 2019, including interim periods within those fiscal years, with early adoption permitted. We are currently assessingand requires a modified retrospective approach. The Company is in the process of evaluating the impact ASU 2016-13 will haveof this new guidance on ourits consolidated financial statements and footnote disclosures thereto.
3. Fair value measurements
The following table presents the FASB issued ASU No. 2016-02, Leases, which is intended to increase transparency and comparability among organizations by recognizing leaseCompany’s assets and lease liabilities on the balance sheet and disclosing key information about leasing arrangements. Under ASU 2016-02, a lessee will be required to recognize assets and liabilities for both operating and financing leases with lease terms of more than 12 months. The amendment is effective for annual periods beginning after December 15, 2018, and interim periods within those annual periods. Early adoption is permitted. In July 2018, the FASB issued ASU 2018-11, Leases, to address two requirements of ASU 2016-02. ASU 2018-11 allows entities to recognize a cumulative-effect adjustment from the application of ASU 2016-02 to the opening balance of retained earnings in the period of adoption. In addition, ASU 2018-11 provides lessors with a practical expedient to not separate non-lease components from the associated lease component if certain conditions are met. We are currently assessing the impact ASU 2016-02 will have on our consolidated financial statements and footnote disclosures thereto.
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| June 30, 2019 | |||||||
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| (Level 1) |
| (Level 2) |
| (Level 3) | |||
Assets |
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|
|
|
|
|
|
|
Money market funds (included in cash and cash equivalents) |
| $ | 52,079 |
| $ | — |
| $ | — |
Marketable securities - U.S. government agency |
| $ | — |
| $ | — |
| $ | — |
Marketable securities - Corporate debt securities |
| $ | — |
| $ | — |
| $ | — |
13
| Description | Balance as of September 30, 2018 | Level 1 | Level 2 | Level 3 | ||||||||||||
| Assets: | ||||||||||||||||
| Cash and money market funds | $ | 31,917 | $ | 31,917 | $ | — | $ | — | ||||||||
| Corporate debt securities (including commercial paper) | 8,968 | — | 8,968 | — | ||||||||||||
| U.S. government securities | 5,465 | — | 5,465 | — | ||||||||||||
| Total | $ | 46,350 | $ | 31,917 | $ | 14,433 | $ | — | ||||||||
| Description | Balance as of December 31, 2017 | Level 1 | Level 2 | Level 3 | ||||||||||||
| Assets: | ||||||||||||||||
| Cash and money market funds | $ | 15,703 | $ | 15,703 | $ | — | $ | — | ||||||||
| Corporate debt securities (including commercial paper) | 35,531 | — | 35,531 | — | ||||||||||||
| U.S. government securities | 15,969 | — | 15,969 | — | ||||||||||||
| Total | $ | 67,203 | $ | 15,703 | $ | 51,500 | $ | — | ||||||||
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| December 31, 2018 | |||||||
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| (Level 1) |
| (Level 2) |
| (Level 3) | |||
Assets |
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|
|
|
|
|
|
Money market funds (included in cash and cash equivalents) |
| $ | 25,145 |
| $ | — |
| $ | — |
Marketable securities - U.S. government agency |
| $ | — |
| $ | 2,994 |
| $ | — |
Marketable securities - Corporate debt securities |
| $ | — |
| $ | 1,391 |
| $ | — |
Prior to the Merger in December 2018, the Company’s preferred stock warrants were liability classified and short-term investments
| September 30, 2018 | Amortized Cost | Gross Unrealized Gains | Gross Unrealized Losses | Fair Value | ||||||||||||
| Cash and money market funds | $ | 31,917 | $ | — | $ | — | $ | 31,917 | ||||||||
| Corporate debt | ||||||||||||||||
| Due in one year or less | 8,969 | — | (1 | ) | 8,968 | |||||||||||
| U.S. government securities | ||||||||||||||||
| Due in one year or less | 5,467 | — | (2 | ) | 5,465 | |||||||||||
| Total | $ | 46,353 | $ | — | $ | (3 | ) | $ | 46,350 | |||||||
| Reported as: | ||||||||||||||||
| Cash and cash equivalents | $ | 31,917 | $ | — | $ | — | $ | 31,917 | ||||||||
| Short-term investments | 14,436 | — | (3 | ) | 14,433 | |||||||||||
| Total | $ | 46,353 | $ | — | $ | (3 | ) | $ | 46,350 | |||||||
| December 31, 2017 | Amortized Cost | Gross Unrealized Gains | Gross Unrealized Losses | Fair Value | ||||||||||||
| Cash and money market funds | $ | 15,703 | $ | — | $ | — | $ | 15,703 | ||||||||
| Corporate debt | ||||||||||||||||
| Due in one year or less | 38,053 | — | (21 | ) | 38,032 | |||||||||||
| U.S. government securities | ||||||||||||||||
| Due in one year or less | 13,474 | — | (6 | ) | 13,468 | |||||||||||
| Total | $ | 67,230 | $ | — | $ | (27 | ) | $ | 67,203 | |||||||
| Reported as: | ||||||||||||||||
| Cash and cash equivalents | $ | 15,703 | $ | — | $ | — | $ | 15,703 | ||||||||
| Short-term investments | 51,527 | — | (27 | ) | 51,500 | |||||||||||
| Total | $ | 67,230 | $ | — | $ | (27 | ) | $ | 67,203 | |||||||
4. Accrued expenses
Accrued expenses consist of (amounts in an unrealized loss position for less than 12 months was $8.6 million and $22.9 million, respectively. At September 30, 2018, we did not hold any investments that have been in a continuous unrealized loss position for 12 months or longer.thousands):
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|
|
|
|
| June 30, |
| December 31, | |
|
| 2019 |
| 2018 | |
Compensation and related benefits | $ | 2,028 |
| $ | 3,537 |
Professional fees |
| 1,751 |
|
| 1,140 |
Preclinical and clinical costs |
| 2,002 |
|
| 1,811 |
Lease termination |
| — |
|
| 630 |
Other |
| 366 |
|
| 512 |
Total | $ | 6,147 |
| $ | 7,630 |
In connection with the adoption of ASC 842 on quantitative and qualitative factors, and we consideredJanuary 1, 2019, the decline in market value for the four debt securities in an unrealized loss positionlease termination balance as of December 31, 2018 was reclassified into the Company’s operating lease liability.
5. Debt
Bpifrance Reimbursable Advance
In December 2017, in connection with its acquisition of Alizé Pharma SAS (“Alizé”), the Company assumed €0.7 million of debt that Alizé had outstanding with Bpifrance Financing (“Bpifrance”). The original advance amount of €0.8 million (“the Bpifrance Advance”) was provided to Alizé as an innovation aid that required Alizé to carry out certain activities related to its livoletide clinical development program and incur a certain level of program expenditures. No interest is charged or accrued under the advance.
The Company is required to make quarterly principal payments, which began in December 2016 and continue through September 30, 2018,2021. The quarterly principal payments escalate over the repayment period beginning with €17,500 per quarter and increasing to be primarily attributable€50,000 through maturity. In addition to the then current economic and market conditions. We will likely notquarterly payments, beginning January 1, 2016, Bpifrance may require the Company to pay, by no later than March 31st of each year, a reimbursement annuity equal to 20% of the proceeds generated by the Company from license, assignment or use of livoletide. Under no circumstance, however, would the Company be required to sell these securities,reimburse to Bpifrance principal amounts greater than the original advance it received.
The Company is permitted to repay the Bpifrance Advance at any time, at which point it would be released from all commitments and doobligations under the Bpifrance Advance agreement. The Bpifrance Advance Agreement does not intend to sell these securities before the recovery of their amortized cost bases, which recovery is expected within the next 12 months. Based on our analysis, we do not consider these investments to be other-than-temporarily impaired as of September 30, 2018.
| As of September 30, | As of December 31, | ||||||
| 2018 | 2017 | ||||||
| Laboratory equipment | $ | 1,247 | $ | 3,480 | |||
| Furniture | 230 | 371 | |||||
| Computer equipment | 13 | 208 | |||||
| Leasehold improvements | 288 | 2,754 | |||||
| Total property and equipment, gross | 1,778 | 6,813 | |||||
| Less: accumulated depreciation and amortization | (1,670 | ) | (3,700 | ) | |||
| Total property and equipment, net | $ | 108 | $ | 3,113 | |||
| As of September 30, | As of December 31, | ||||||
| 2018 | 2017 | ||||||
| Compensation and related benefits, including severance | $ | 1,447 | $ | 2,215 | |||
| Development, site costs and contract manufacturing | — | 519 | |||||
| Legal, audit and tax services | 422 | 1,542 | |||||
| Consulting | 142 | 160 | |||||
| Deferred rent | 353 | 334 | |||||
| Other accrued expenses and other current liabilities | 913 | 792 | |||||
| $ | 3,277 | $ | 5,562 | ||||
| Shares | Weighted average exercise price per share | Weighted average remaining contractual term (years) | Aggregate intrinsic value (in thousands) | |||||||||
| Outstanding at December 31, 2017 | 5,745,815 | $ | 7.28 | 8.32 | $ | 43 | ||||||
| Granted | 1,251,877 | 0.96 | ||||||||||
| Forfeited/Canceled | (1,837,518 | ) | 5.87 | |||||||||
| Outstanding at September 30, 2018 | 5,160,174 | 6.23 | 7.80 | 1 | ||||||||
| Exercisable at September 30, 2018 | 2,577,812 | 10.61 | 6.76 | 1 | ||||||||
| Three months ended September 30, | Nine months ended September 30, | ||||||
| 2018 | 2017 | 2018 | 2017 | ||||
| Risk-free interest rate | 2.7% | 1.9% | 2.7% | 1.3% - 2.2% | |||
| Dividend yield | — | — | — | — | |||
| Volatility | 83% | 92% | 83% - 85% | 89% - 109% | |||
| Expected term (years) | 5.3 | 6.1 | 5.3 - 6.1 | 2.0 - 6.9 | |||
During the three and nine months ended September 30, 2018, we granted options to purchase 72,000 and 1,251,877 shares of our common stock to employees at a weighted average grant date fair value of $0.65 and $0.70 per share, respectively, and with a weighted average exercise price of $0.95 and $0.96 per share, respectively. During the three and nine months ended September 30, 2017, we granted options to purchase 192,500 and 4,207,856 shares of our common stock at weighted average grant date fair values of $1.05 and $1.14 per share, respectively, and with weighted average exercise prices of $1.40 and $1.51 per share, respectively.
14
| Accrued restructuring balance as of December 31, 2017 | $ | 403 | ||
| Plus: | ||||
| Severance | 1,377 | |||
| Other | 109 | |||
| Less: | ||||
| Payments | (1,842 | ) | ||
| Accrued restructuring balance as of September 30, 2018 | $ | 47 | ||
6. Litigation
Liabilities for loss contingencies arising from claims, assessments, litigation, fines, penalties, and contingencies
On November 9, 2016, a purported shareholder derivative action was filed in the Business Litigation Session of the Suffolk County Superior Court in the Commonwealth of Massachusetts (Cima v. Dipp et al., No. 16-3443-BLS1 (Mass.Sup.16‑3443‑BLS1 (Mass. Sup. Ct.)) against certain present and former officers and directors of OvaScience and one current director of the Company (a former director of OvaScience) and the CompanyOvaScience as a nominal defendant alleging breaches of fiduciary duty, unjust enrichment, abuse of control, gross mismanagement and corporate waste for purported actions related to the Company'sOvaScience’s January 2015 follow-on public offering. On February 22, 2017, the court approved the parties’ joint stipulation to stay all proceedings in the action until further notice. Following a status conference in December 2017, the stay was lifted. On January 23,25, 2018, at the parties’ request, the court entered a second order staying all proceedings in the action until further order of the court. We believeThe Company believes that the complaint is without merit and intendintends to defend against the litigation. There can be no assurance, however, that wethe Company will be successful. A resolution of this lawsuit adverse to OvaScience or the other defendants could have a material effect on OvaScience's consolidated financial position and results of operations in the period in which the lawsuit is resolved.At present, we arethe Company is unable to estimate potential losses, if any, related to the lawsuit.lawsuit
On March 24, 2017, a purported shareholder class action lawsuit was filed in the U.S. District Court for the District of Massachusetts (Dahhan v. OvaScience, Inc., et al., No.1:17-cv-10511-IT(D.No. 1:17‑cv‑10511‑IT (D. Mass.)) against the CompanyOvaScience and certain of our former officers and directors of OvaScience alleging violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 (the “Dahhan Action”"Dahhan Action"). On July 5, 2017, the Courtcourt entered an order approving the appointment of Freedman Family Investments LLC as lead plaintiff, the firm of Robins Geller Rudman & Dowd LLP as lead counsel and the Law Office of Alan L. Kovacs as local counsel. Plaintiff filed an amended complaint on August 25, 2017. The defendantsCompany filed a motion to dismiss the amended complaint, which the Courtcourt denied on July 31, 2018. On August 14, 2018, wethe Company answered the amended complaint. We believeThe parties presently are engaged in discovery. The Company believes that the amended complaint is without merit and intendintends to defend against the litigation. There can be no assurance, however, that wethe Company will be successful. A resolution of this lawsuit adverse to the Company or the other defendants could have a material effect on
On July 27, 2017, a purported shareholder derivative complaint was filed in the U.S. District Court for the District of Massachusetts (Chiu v. Dipp et al., No. 1:17-cv-11382-IT17‑cv‑11382‑IT (D. Mass.)) against certain of the Company's present and former directors and the CompanyOvaScience, as a nominal defendant, certain former officers and directors of OvaScience and one current director of the Company (a former director of OvaScience) alleging breach of fiduciary duty, unjust enrichment and violations of Section 14(a) of the Securities Exchange Act of 1934 alleging that compensation awarded to the director defendants was excessive and seeking redress for purported actions related to the Company’sOvaScience’s January 2015 follow-on public offering and other public statements. On September 26, 2017, the plaintiff filed an amended complaint which eliminated all claims regarding allegedly excessive director pay and additionally alleged claims of abuse of control and corporate waste. On October 27, 2017, the defendants filed a motion to dismiss the amended complaint. The court heard oral argument on the motion to dismiss on April 5, 2018. On April 13, 2018, the court granted the defendants’ motion to dismiss the amended complaint for failure to state a claim for relief under Section 14(a). The court also dismissed the plaintiffs’ pendent state law claims without prejudice, based on lack of subject matter jurisdiction. On April 25, 2018, the plaintiffs moved for leave to amend the complaint, and to stay this case pending the outcome of the Dahhan Action and the now-dismissed Westmoreland Federal Action. The defendants doCompany does not believe that the proposed amended complaint cures the defects in the current complaint, but informed plaintiffs’ counsel that, in the interest of judicial economy, the defendants would not oppose the proposed amendment if the court would consider staying the case pending the resolution of the Westmoreland Federal Action (now dismissed) and the pending Dahhan Action. On April 27, 2018, the court granted the plaintiffs’ motion for leave to amend the complaint and for a stay. On April 30, 2018, the plaintiffs filed their second amended complaint. On May 23, 2018, the Courtcourt entered an order staying this case pending the resolution of the Dahhan Action and the (now dismissed) Westmoreland Federal Action. We believeThe Company believes that the complaint is without merit and intendintends to defend against the litigation. There can be no assurance, however, that wethe Company will be successful. A resolution of this lawsuit adverse toAt present, the Company or the other defendants could have a material effect on our consolidated financial position and results of operations in the period in which the lawsuit is resolved. At present, we are unable to estimate potential losses, if any, related to the lawsuit.lawsuit
Between October 16, 2018 a Company stockholderand November 21, 2018, five putative class action lawsuits were filed a complaint in various federal District Courts against OvaScience, Inc. and the U.S. District Court for the DistrictOvaScience Board of Delaware (Directors related to OvaScience’s proposed merger with Millendo Therapeutics, Inc.: Cunningham v. Kroeger, et al., No. 1:18-cv-01595-CFC18‑cv‑01595 (D. Del.)) against the Company, its chief executive officer filed Oct. 16, 2018); Adlard v. OvaScience, Inc., et al., No. 1:18‑cv‑12332 (D. Mass. filed Nov. 6, 2018); Wheby v. OvaScience, Inc.,
15
et al., No. 1:18‑cv‑1811 (D. Del. filed Nov. 16, 2018); Cuenca Aubets v. OvaScience, Inc., et al., No. 1:18‑cv‑10882 (S.D.N.Y. filed Nov. 20, 2018); and the members of the Company’s Board of Directors, allegingKim v. OvaScience, Inc., et al., No. 1:18‑cv‑10939 (S.D.N.Y. filed Nov. 21, 2018). The Complaints each alleged violations of Section 14(a) of the Securities Exchange Act of 1934 and Rule 14a-914a‑9 promulgated thereunder, and as against the individual defendants, alleging violations of Section 20(a) of the Securities Exchange Act of 1934. The Cunningham plaintiff allegesalleged that the defendants made materially misleading disclosures in aOvaScience’s Form S-4S‑4 Registration Statement filed with the SEC on September 26, 2018 in connection with Company’somitted or misrepresented material information regarding OvaScience’s proposed merger with Millendo Therapeutics, Inc. (“Millendo”), by allegedly omitting material information concerning the sale process. The plaintiff seeks declaratoryAdlard, Wheby, Cuenca Aubets and injunctive relief to enjoin the proposed merger with Millendo, rescissory damages against the individual defendants, including pre-judgment and post-judgment interest, costs, and attorneys’ fees. We believeKim plaintiffs alleged that the complaint is without merit and intend to defend against the litigation. There can be no assurance, however, that we will be successful. At present, we are unable to estimate potential losses, if any, related to the lawsuit.
In addition to the matters described above, the Company may be a party to litigation and subject to claims incident to the ordinary course of business from time to time. Regardless of the outcome, litigation can have an adverse impact on the Company because of defense and settlement costs, diversion of management resources and other factors.
7. Stock‑based compensation
On June 11, 2019, the Company held its 2019 Annual Meeting of Stockholders (the “Annual Meeting”). At the Annual Meeting, the Company’s stockholders approved the Company’s 2019 Equity Incentive Plan (the “2019 Plan”) and the Company’s 2019 Employee Stock Purchase Plan (the “2019 ESPP,” and together with the 2019 Plan, the “Plans”). The 2019 Plan is the successor to the Private Millendo 2012 Stock Plan and the background process leadingOvaScience 2012 Stock Incentive Plan (each, as amended, the “Prior Plans”) and allows the Company to grant stock options, restricted stock unit awards and other awards at levels determined appropriate by the Company’s board of directors (the “Board”) or the compensation committee of the Board. No additional awards will be granted under either of the Prior Plans. The 2019 ESPP enables employees to purchase shares of the Company’s common stock through offerings of rights to purchase the Company’s common stock to all eligible employees. The Plans were adopted by the Company’s Board on April 29, 2019, subject to approval by the Company’s stockholders, and became effective with such stockholder approval on June 11, 2019. Outstanding awards under the Prior Plans continue to be subject to the terms and conditions of the Prior Plans.
The aggregate number of shares of our common stock that may be issued under the 2019 Plan will not exceed 2,919,872. The number of shares of our common stock reserved for issuance under the 2019 Plan will automatically increase on January 1 of each year, for a period of ten years, from January 1, 2020 continuing through January 1, 2029, by 4% of the total number of shares of our common stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares as may be determined by the Board.
The aggregate number of shares of our common stock that may be issued under the 2019 ESPP is 133,580 shares, plus the number of shares of our common stock that are automatically added on January 1st of each year for a period of up to ten years from January 1, 2020 continuing through January 1, 2029, by 1% of the proposed transaction,total number of shares of our common stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares as may be determined by the Board.
The Company measures employee and potential conflictsnonemployee stock‑based awards at grant‑date fair value and records compensation expense on a straight‑line basis over the vesting period of interest. The plaintiff seeks injunctive relief to enjoin the proposed merger with Millendo, declaratory relief, rescissory damages in the event that the merger with Millendo is consummated, costs, and attorneys’ fees. We believe that the complaint is without merit and intend to defend against the litigation. There can be no assurance, however, that we will be successful. At present, we are unable to estimate potential losses, if any, related to the lawsuit.award.
16
The Company recorded stock‑based compensation expense in the following expense categories of its accompanying consolidated statements of operations and comprehensive loss for the three months ended June 30, 2019 and 2018 and six months ended June 30, 2019 and 2018, respectively (amounts in thousands):
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|
| Three months |
| Six months | ||||||||
|
| ended |
| ended | ||||||||
|
| June 30, |
| June 30, | ||||||||
|
| 2019 |
| 2018 |
| 2019 |
| 2018 | ||||
Research and development |
| $ | 378 |
| $ | 67 |
| $ | 801 |
| $ | 138 |
General and administrative |
|
| 616 |
|
| 92 |
|
| 1,133 |
|
| 198 |
Total |
| $ | 994 |
| $ | 159 |
| $ | 1,934 |
| $ | 336 |
Stock options
Options issued under the 2019 Plan may have a contractual life of up to 10 years and may be exercisable in cash or as otherwise determined by the Board. Vesting generally occurs over a period of not greater than four years.
The following table summarizes the activity related to stock option grants to employees and nonemployees for the six months ended June 30, 2019:
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| Weighted |
| Weighted-average | |
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|
| average |
| remaining | |
|
|
|
| exercise price |
| contractual | |
|
| Shares |
| per share |
| life (years) | |
Outstanding at December 31, 2018 |
| 1,764,287 |
| $ | 26.81 |
| 8.0 |
Granted |
| 1,028,701 |
|
| 10.23 |
|
|
Exercised |
| (45,947) |
|
| 3.63 |
|
|
Forfeited |
| (106,964) |
|
| 86.07 |
|
|
Outstanding at June 30, 2019 |
| 2,640,077 |
| $ | 18.36 |
| 7.9 |
Vested and exercisable at June 30, 2019 |
| 893,208 |
| $ | 30.71 |
| 5.1 |
Vested and expected to vest at June 30, 2019 |
| 2,640,077 |
| $ | 18.36 |
| 7.9 |
As of June 30, 2019, the unrecognized compensation cost related to 1,746,869 unvested stock options expected to vest was $12.1 million. This unrecognized compensation will be recognized over an estimated weighted‑average amortization period of 3.1 years. The aggregate intrinsic value of options outstanding and options exercisable as of June 30, 2019 was $5.8 million and $3.6 million, respectively. The options granted during the three and six months ended June 30, 2019 had an estimated weighted average grant date fair value of $8.50 and $7.09, respectively. There were no options granted during the three or six months ended June 30, 2018. The grant date fair value of each option grant was estimated during the three and six months ended June 30, 2019 using the following assumptions within the Black‑Scholes option‑pricing model:
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| Three months ended |
| Six months ended |
| ||
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| June 30, |
| June 30, |
| ||
|
| 2019 |
| 2019 |
| ||
Expected term (in years) |
|
| 5.93 |
|
| 6.01 |
|
Expected volatility |
|
| 79 | % |
| 80 | % |
Risk-free interest rate |
|
| 1.91 | % |
| 2.35 | % |
Expected dividend yield |
|
| 0 | % |
| 0 | % |
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|
At the time of the Alizé acquisition in December 2017, Alizé had 6,219 non‑employee (BSA) warrants and 5,360 employee (BSPCE) warrants outstanding, which have weighted‑average exercise prices of €80.06 and €83.40, respectively. As of June 30, 2019, all BSAs and BSPCEs were vested. In June 2019, a total of 600 BSPCE warrants were exercised resulting in the issuance of 8,112 shares of the Company’s common stock. As of June 30, 2019, there were an aggregate of 148,607 shares of common stock issuable upon the exercise of the warrants with a weighted‑average exercise price of $6.90 per share. These instruments are included in the equity attributable to noncontrolling interests.
17
8. Subsequent events
Subsequent events were evaluated through the filing date of this Quarterly Report.
18
You should read the following discussion of our financial condition and results of operations in conjunction with our interim unaudited consolidated financial statements that involve risks and uncertainties. The statements containedthe notes thereto included elsewhere in this Quarterly Report on Form 10-Q that are not purely historical are forward-looking statements within the meaning of Section 27A of the Securities Act of 193310‑Q and Section 21E of the Securities Exchange Act of 1934. Without limiting the foregoing, the words “may,” “shall,” “will,” “should,” “could,” “expects,” “plans,” “intends,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” “target,” “goal”, “seek”, “likely,” “hope” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these words. All forward-lookingwith our annual audited consolidated financial statements included in this Quarterly Report on Form 10-Q are based on information available to us up to, and including, the date of this document, and we expressly disclaim any obligation to update any such forward-looking statements to reflect events or circumstances that arise after the date hereof. Our actual results could differ materially from those anticipated in these forward-looking statements as a result of certain important factors, including those set forth in this Item 2 — “Management’s Discussion and Analysis of Financial Condition and Results of Operations,” as well as under the heading “Risk Factors” contained in Item 1A of our Annual Report on Form 10-K10‑K for the year ended December 31, 20172018 as filed with the Securities and in Part II, Item 1A “Risk Factors” of this Quarterly Report.
Overview
We are a further restructuring of its organization and a workforce reduction of approximately 50%. On May 3, 2018, OvaScience announced that its Board of Directors had approved a corporate restructuring plan furthering its on-going efforts to effectively align its resources. On August 9, 2018, OvaScience announced that it had entered into a definitive agreement with Millendo Therapeutics, Inc. ("Millendo") under which Millendo will merge with OvaScience in an all-stock transaction. A wholly owned subsidiary of OvaScience will merge with and into Millendo, with Millendo surviving as a wholly owned subsidiary of OvaScience (the "Merger"). OvaScience and Millendo believe that the Merger will result in a specialtylate-stage biopharmaceutical company primarily focused on developing novel treatments for orphan endocrine diseases where current therapies do not exist or are insufficient. The transaction has been approvedWe are currently advancing three product candidates. Our most advanced product candidate, livoletide (AZP‑531), is a potential treatment for Prader-Willi syndrome (“PWS”), a rare and complex genetic endocrine disease characterized by the Boardshyperphagia, or insatiable hunger, that contributes to serious complications, a significant burden on patients and caregivers and early mortality. In a randomized, double-blind, placebo-controlled Phase 2 clinical trial in 47 patients with PWS, we observed that administration of Directors of both companies. The Merger is expected to closelivoletide once daily was associated with a clinically meaningful improvement in the fourth quarter of 2018, subject to the approval of OvaScience shareholders at a special shareholder meeting to be held on December 4, 2018, as well as other customary conditions.
| Three Months Ended, | 2018/ 2017 Comparison | Nine Months Ended | 2018 / 2017 Comparison | ||||||||||||||||||||||||||
| September 30, | Increase / (Decrease) | September 30, | Increase / (Decrease) | ||||||||||||||||||||||||||
| 2018 | 2017 | $ | % | 2018 | 2017 | $ | % | ||||||||||||||||||||||
| Revenues | $ | 55 | $ | 56 | $ | (1 | ) | (2 | )% | $ | 203 | $ | 204 | $ | (1 | ) | — | % | |||||||||||
| Costs of revenues | 44 | 29 | 15 | 52 | % | 210 | 572 | (362 | ) | (63 | )% | ||||||||||||||||||
| Research and development expenses | 1,680 | 4,016 | (2,336 | ) | (58 | )% | 6,695 | 14,777 | (8,082 | ) | (55 | )% | |||||||||||||||||
| Selling, general and administrative expenses | 4,176 | 5,056 | (880 | ) | (17 | )% | 11,045 | 22,935 | (11,890 | ) | (52 | )% | |||||||||||||||||
| Restructuring | 53 | 361 | (308 | ) | (85 | )% | 3,637 | 3,842 | (205 | ) | (5 | )% | |||||||||||||||||
| Interest income, net | 228 | 193 | 35 | 18 | % | 643 | 560 | 83 | 15 | % | |||||||||||||||||||
| Other income (expense), net | (7 | ) | (3 | ) | 4 | 133 | % | (5 | ) | (37 | ) | (32 | ) | (86 | )% | ||||||||||||||
| Loss from equity method investment | 2 | 140 | (138 | ) | (99 | )% | 6 | 1,015 | (1,009 | ) | (99 | )% | |||||||||||||||||
| Income tax expense | — | 11 | (11 | ) | (100 | )% | — | 33 | (33 | ) | (100 | )% | |||||||||||||||||
| Net loss | $ | 5,679 | $ | 9,367 | $ | (3,688 | ) | (39 | )% | $ | 20,752 | $ | 42,447 | $ | (21,695 | ) | (51 | )% | |||||||||||
We are developing nevanimibe (ATR-101) for the treatment of patients with classic congenital adrenal hyperplasia (“CAH”), a rare, monogenic adrenal disease that requires lifelong treatment with exogenous cortisol, often at high doses. These chronic high doses of cortisol can result in side effects that include diabetes, obesity, hypertension and psychological problems. When on suboptimal doses of cortisol, female CAH patients can experience hirsutism, infertility and menstrual irregularity, and male CAH patients can experience testicular atrophy, infertility and testicular tumors, making it difficult for physicians to appropriately treat CAH without causing adverse consequences. We reported results from our restructuring activities. Our costsPhase 2 clinical trial of revenues include the cost of processing patient tissue that corresponds to treatment revenues for the reporting period. Given our shiftnevanimibe in corporate prioritiespatients with CAH in March 2018 and focus on research and development, we expect cost of revenues to continue to decreaseinitiated a Phase 2b trial in the future.
We have also added a new product candidate to our research and development expense for the three months ended September 30, 2018 as compared to the three months ended September 30, 2017, from $4.0 million to $1.7 million was primarily attributable to:
Since inception, we have incurred significant operating losses and other costs primarily attributable to the decrease in our headcount as result of our corporate restructuring initiatives;negative operating cash flows and
Recent Developments
Livoletide (AZP-531) for the potential treatment for Prader-Willi syndrome (PWS)
In March 2019, we announced that we initiated a pivotal Phase 2b/3 clinical trial of livoletide in PWS patients, with topline results from the Phase 2b portion of the study expected in the first half of 2020. As of August 8, 2019, we had 26 clinical sites recruiting patients in the U.S., Europe and Australia. We also plan to submit a protocol amendment, allowing 4 to 7 year-olds to participate in the Phase 2b/3 clinical study. The planned protocol amendment will include
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supporting juvenile toxicity data to allow dosing down to 4 years of age to cover the entire PWS population. We have also initiated pre-clinical activities in support of the development of a multi-dose pen device to improve patient and caregiver convenience, patient compliance, and further simplify administration of livoletide. On July 29, 2019, the U.S. Food and Drug Administration, or FDA, designated as a Fast Track development program the investigation of livoletide for PWS.
Neurokinin 3 receptor (NK3R) antagonist for the treatment ofvasomotor symptoms (VMS)
We have added a new product candidate to our research and development pipeline: MLE-301, an NK3R antagonist, as a potential treatment of VMS. MLE-301 is a second generation NK3R antagonist, a target that Millendo and others have previously shown plays a key role in regulating the activity of KNDy (kisspeptin/NKB/dynorphin) neurons, which are overactive in menopausal women and play an important role in the generation of VMS. MLE-301 currently is in preclinical studies designed to enable first-in-human clinical studies, which we expect to initiate in 2020. We have a worldwide, exclusive license from F. Hoffmann-La Roche Ltd (“Roche Basel”) and Hoffman-La Roche Inc. (“Roche US”, and together with Roche Basel, “Roche” ), pursuant to a license agreement with Roche with an effective date of October 16, 2018 (the “Roche License Agreement”). As consideration for the rights granted to us under the Roche License Agreement, we have agreed to pay Roche an up-front payment, as well as additional milestone payments and royalties on sales of any MLE-301 products that we commercialize. Roche is affiliated with Roche Finance Ltd., which is a holder of more than 5% of our outstanding capital stock and which also employs Carole L. Nuechterlein, J.D., a member of our board of directors.
Nevanimibe for the treatment of classic congenital adrenal hyperplasia (CAH)
The nevanimibe Phase 2b CAH study began in the third quarter of 2018 and 2017,is ongoing. Preliminary data with a starting dose of 1000 mg BID (twice per day) has demonstrated lower tolerability than expected based on prior clinical experience with nevanimibe. Three of six subjects in the Phase 2b CAH study discontinued the study after beginning treatment as a result of non-serious adverse events, primarily of rash, dysuria, or diarrhea. Patients treated at 500 mg BID in previously completed studies tolerated nevanimibe well. We believe that dose escalation from a starting dose of 500 mg BID to higher planned doses over a longer period (16 weeks versus 12 weeks) may allow patients to better tolerate the therapy. We have submitted a protocol amendment to the appropriate regulatory authorities in all countries with active clinical trial sites to reflect this change in dosing and length of study treatment period. The study has resumed recruitment under the amended protocol.
Previous experience with nevanimibe includes a Phase 2 CAH study where patients started at 125 mg BID and dose escalated to 1000 mg BID, and a healthy volunteer drug-drug interaction study with nevanimibe at 500 mg BID for 11 days. In addition, the Phase 1 adrenocortical carcinoma (ACC) study dosed subjects over 2.5 times above the highest dose being explored in the Phase 2b CAH study.
We expect to provide an update on CAH study timelines in the second half of 2019.
Nevanimibe for the treatment of endogenous Cushing’s syndrome (CS)
We have been investigating nevanimibe (ATR-101) in a Phase 2 clinical trial as a potential treatment for patients with endogenous Cushing’s syndrome (“CS”), a rare endocrine disease characterized by excessive cortisol production from the adrenal glands. As a result of slower than anticipated enrollment in this Phase 2 clinical trial, we elected to discontinue this trial in August 2019, suspend development of nevanimibe for the treatment of CS, and focus our resources on other programs in our research and development pipeline. The costs associated with discontinuing our CS trial are not expected to be material.
Merger
On December 7, 2018, OvaScience, Inc., or OvaScience, now known as Millendo Therapeutics, Inc., completed its reverse merger or, the Merger, with what was then known as “Millendo Therapeutics, Inc.,” or Private Millendo, in accordance with the terms of the Agreement and Plan of Merger and Reorganization dated as of August 8, 2018, as amended on September 25, 2018 and November 1, 2018, or the Merger Agreement. OvaScience’s shares of common
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stock listed on The Nasdaq Capital Market, previously trading through the close of business on Friday, December 7, 2018 under the ticker symbol “OVAS,” commenced trading on The Nasdaq Capital Market, under the ticker symbol “MLND,” on Monday, December 10, 2018.
Immediately following the Merger, Private Millendo became a wholly-owned subsidiary of OvaScience. Upon consummation of the Merger, or the Closing, OvaScience adopted the business plan of Private Millendo and discontinued the pursuit of OvaScience’s business plan pre-Closing. The Merger was accounted for as a reverse recapitalization with Private Millendo as the accounting acquirer. On the Merger date, the primary pre-combination assets of OvaScience was cash, cash equivalents and marketable securities. At the time of the Merger, OvaScience had net assets of $38.0 million, which for both periods waswere comprised primarily of $0.6 millioncash, cash equivalents and marketable securities.
Components of interest income relatedOur Results of Operations
Research and development expense
Research and development expense consists primarily of costs incurred in connection with the development of our product candidates. We expense research and development costs as incurred. These expenses include:
· | personnel expenses, including salaries, benefits and stock-based compensation expense; |
· | costs of funding research performed by third parties, including pursuant to agreements with contract research organizations, or CROs, as well as investigative sites and consultants that conduct our preclinical studies and clinical trials; |
· | expenses incurred under agreements with contract manufacturing organizations, or CMOs, including manufacturing scale-up expenses and the cost of acquiring and manufacturing preclinical study and clinical trial materials; |
· | payments made under our third-party licensing agreements, other than amounts classified as acquired in-process research and development expenses; |
· | consultant fees and expenses associated with outsourced professional scientific development services; |
· | expenses for regulatory activities, including filing fees paid to regulatory agencies; and |
· | allocated expenses for facility costs, including rent, utilities, depreciation and maintenance. |
Milestone payment obligations incurred prior to short-term investments.regulatory approval of a product candidate, which are accrued when the event requiring payment of the milestone occurs are included in research and development expense.
We typically use our employee, consultant and infrastructure resources across our development programs. We track certain outsourced development costs by product candidate, but do not allocate all personnel costs or other internal costs to specific product candidates.
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The following table summarizes our OvaXon joint venture was de minimisresearch and development expenses by product candidate, personnel expense and other expenses for the three and ninesix months ended SeptemberJune 30, 2018. Loss from equity method investment from this joint venture was $0.1 million2019 and $1.0 million2018, respectively:
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| Three Months Ended |
| Six Months Ended | ||||||||
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| June 30, |
| June 30, | ||||||||
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| 2019 |
| 2018 |
| 2019 |
| 2018 | ||||
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| (dollars in thousands) |
| (dollars in thousands) | ||||||||
Nevanimibe expenses |
| $ | 910 |
| $ | 1,117 |
| $ | 1,728 |
| $ | 2,385 |
Livoletide expenses |
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| 3,028 |
|
| 954 |
|
| 6,031 |
|
| 1,392 |
MLE-301 expenses |
|
| 172 |
|
| — |
|
| 444 |
|
| — |
Personnel expenses |
|
| 1,646 |
|
| 997 |
|
| 3,525 |
|
| 1,926 |
Other expenses |
|
| 225 |
|
| 132 |
|
| 457 |
|
| 266 |
Total |
| $ | 5,981 |
| $ | 3,200 |
| $ | 12,185 |
| $ | 5,969 |
We expect our research and development expense will increase for the threeforeseeable future as we seek to advance development of our product candidates. The successful development of our product candidates is highly uncertain. At this time, we cannot reasonably estimate or know the nature, timing and nine months ended September 30, 2017.
| September 30, | December 31, | ||||||
| 2018 | 2017 | ||||||
| Cash, cash equivalents and short-term investments | $ | 46,350 | $ | 67,203 | |||
| Working capital | 45,105 | 60,977 | |||||
| Nine Months Ended September 30, | |||||||
| 2018 | 2017 | ||||||
| Cash (used in) provided by: | |||||||
| Operating activities | $ | (21,492 | ) | $ | (37,579 | ) | |
| Investing activities | 37,706 | 18,774 | |||||
| Capital expenditures (included in investing activities above) | — | (112 | ) | ||||
| Financing activities | — | — | |||||
· | the number of clinical sites included in the trials; |
· | the length of time required to enroll suitable patients; |
· | the number of patients that ultimately participate in the trials; |
· | the number of doses patients receive; |
· | the duration of patient follow-up and number of patient visits; |
· | the results of our clinical trials; |
· | the establishment of commercial manufacturing capabilities; |
· | the receipt of marketing approvals; and |
· | the commercialization of product candidates. |
We may never succeed in obtaining regulatory approval for livoletide, nevanimibe or any future product candidates we may develop. Product candidates in later stages of clinical development, like livoletide, generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials.
General and administrative expense
General and administrative expense consists primarily of personnel expenses, including salaries, benefits and stock-based compensation expense, for employees in executive, finance, accounting, business development, legal and commercializationhuman resource functions. General and administrative expense also includes corporate facility costs, including rent, utilities, depreciation and maintenance, not otherwise included in research and development expense, as well as legal fees related to intellectual property and corporate matters and fees for accounting, recruiting and consulting services.
We anticipate that our general and administrative expense will increase as a result of increased headcount, expanded infrastructure and higher accounting, legal, consulting and investor relations fees, as well as increased director and
22
officer insurance premiums, associated with being a public company. We also anticipate that our general and administrative expense will increase as we support additional clinical trials for livoletide and nevanimibe. In addition, if and when we believe that regulatory approval of livoletide or nevanimibe appears likely, we anticipate an increase in headcount and expense as a result of our fertility treatments,preparation for commercial operations.
Interest expense (income), net
Interest income represents amounts earned on our cash, cash equivalents, marketable securities and restricted cash balances.
Results of operations
Comparison of the three months ended June 30, 2019 and 2018
The following table summarizes our operating results for the periods indicated:
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Operating expenses: |
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Research and development |
| $ | 5,981 |
| $ | 3,200 |
| $ | 2,781 |
| 86.9 | % |
General and administrative |
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| 4,179 |
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| 1,786 |
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| 2,393 |
| 134.0 |
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Loss from operations |
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| 10,160 |
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| 4,986 |
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| 5,174 |
| 103.8 |
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Other expenses: |
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Interest expense (income), net |
|
| (313) |
|
| (6) |
|
| (307) |
| * |
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Other loss |
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| 24 |
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| 62 |
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| (38) |
| (61.3) |
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Net loss |
| $ | (9,871) |
| $ | (5,042) |
| $ | (4,829) |
| 95.8 | % |
*Not Meaningful
Research and development expense
Research and development expense increased by $2.8 million to $6.0 million for the three months ended June 30, 2019 from $3.2 million for the three months ended June 30, 2018. The following table summarizes our research and development expenses for the three months ended June 30, 2019 and 2018:
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| Three Months Ended |
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Preclinical and clinical development expense |
| $ | 4,112 |
| $ | 2,094 |
| $ | 2,018 |
| 96.4 | % |
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Compensation expense, other than stock-based compensation |
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| 1,268 |
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| 930 |
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| 338 |
| 36.3 |
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Stock-based compensation expense |
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| 378 |
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| 67 |
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| 311 |
| 464.2 |
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Other expenses |
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| 223 |
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| 109 |
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| 114 |
| 104.6 |
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Total research and development expense |
| $ | 5,981 |
| $ | 3,200 |
| $ | 2,781 |
| 86.9 | % |
|
The increase in total research and development expense is attributable to:
· | a $2.0 million increase in preclinical and clinical development expense mainly related to the development of livoletide; |
· | a $0.6 million increase in compensation and stock-based compensation expenses as a result of our increase in research and development headcount and additional options granted; and |
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· | a $0.1 million increase in other expenses due to facility and other overhead expenses. |
General and administrative expense
General and administrative expense increased by $2.4 million to $4.2 million for the three months ended June 30, 2019 from $1.8 million for the three months ended June 30, 2018. The increase was primarily due to a $1.4 million increase in compensation and stock-based compensation expense as a result of our increase in general and administrative headcount and changes to compensation arrangements, a $0.6 million increase in professional fees incurred primarily as a result of operating as a public company, and a $0.4 million increase in insurance, rent and facility related expenses. The increase in insurance is due to operating as a public company and the extentincrease in rent and facility related expenses is due to whichincreased headcount and additional leased office space.
Interest expense (income), net
Interest expense (income), net increased by $0.3 million to $0.3 million net interest income for the three months ended June 30, 2019 from net interest income of $6,000 for the three months ended June 30, 2018. The change was primarily due to higher interest income received as a result of larger cash, cash equivalent and marketable securities balances we may enter into collaborations with third partieshad immediately following the Merger.
Other loss
Other loss decreased by $38,000 to $24,000 for the three months ended June 30, 2019 from $62,000 for the three months ended June 30, 2018 due to lower foreign currency losses as a result of exchange rate fluctuations on transactions denominated in a currency other than our functional currency.
Comparison of the six months ended June 30, 2019 and 2018
The following table summarizes our operating results for the periods indicated:
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| 2018 |
| Change |
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Operating expenses: |
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Research and development |
| $ | 12,185 |
| $ | 5,969 |
| $ | 6,216 |
| 104.1 | % |
General and administrative |
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| 8,632 |
|
| 3,405 |
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| 5,227 |
| 153.5 |
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Loss from operations |
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| 20,817 |
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| 9,374 |
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| 11,443 |
| 122.1 |
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Other expenses: |
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Interest expense (income), net |
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| (628) |
|
| (15) |
|
| (613) |
| * |
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Other loss |
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| 48 |
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| 69 |
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| (21) |
| (30.4) |
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Net loss |
| $ | (20,237) |
| $ | (9,428) |
| $ | (10,809) |
| 114.6 | % |
*Not Meaningful
Research and development expense
Research and commercializationdevelopment expense increased by $6.2 million to $12.2 million for the six months ended June 30, 2019 from $6.0 million for the six months ended June 30, 2018. The following table summarizes our research and development expenses for the six months ended June 30, 2019 and 2018:
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| 2018 |
| Change |
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| (dollars in thousands) |
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Preclinical and clinical development expense |
| $ | 8,205 |
| $ | 3,800 |
| $ | 4,405 |
| 115.9 | % |
Compensation expense, other than stock-based compensation |
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| 2,724 |
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| 1,788 |
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| 936 |
| 52.3 |
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Stock-based compensation expense |
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| 801 |
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| 138 |
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| 663 |
| 480.4 |
|
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Other expenses |
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| 455 |
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| 243 |
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| 212 |
| 87.2 |
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Total research and development expense |
| $ | 12,185 |
| $ | 5,969 |
| $ | 6,216 |
| 104.1 | % |
The increase in total research and development expense is attributable to:
· | a $4.4 million increase in preclinical and clinical development expense mainly related to the development of livoletide; |
· | a $1.6 million increase in compensation and stock-based compensation expenses as a result of our increase in research and development headcount and additional options granted; and |
· | a $0.2 million increase in other expense due to an increase in facility and other overhead expenses. |
General and administrative expense
General and administrative expense increased by $5.2 million to $8.6 million for the six months ended June 30, 2019 from $3.4 million for the six months ended June 30, 2018. The increase was primarily due to a $2.2 million increase in compensation and stock-based compensation expense as a result of our fertility treatments,increase in general and administrative headcount and changes to compensation arrangements, a $2.0 million increase in professional fees incurred mainly related to being a publicly traded company, and a $1.0 million increase in insurance, rent and facility related expenses due to increased headcount and operating as a public company.
Interest expense (income), net
Interest expense (income), net increased by $0.6 million to $0.6 million net interest income for the six months ended June 30, 2019 from $15,000 net interest income for the six months ended June 30, 2018. The change was primarily due to higher interest income received as a result of larger cash, cash equivalent and marketable securities balances we had immediately following the Merger.
Other loss
Other loss decreased by $21,000 to $48,000 for the six months ended June 30, 2019 from $69,000 for the six months ended June 30, 2018 due to lower foreign currency losses as a result of exchange rate fluctuations on transactions denominated in a currency other than our functional currency.
Liquidity and Capital Resources
The following table sets forth the primary uses of cash and cash equivalents for the six months ended June 30, 2019 and 2018:
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| 2019 |
| 2018 | ||
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| (in thousands) | ||||
Net cash used in operating activities |
| $ | (21,174) |
| $ | (9,698) |
Net cash provided by (used in) investing activities |
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| 4,108 |
|
| (531) |
Net cash used in financing activities |
|
| (38) |
|
| (566) |
Effect of foreign currency exchange rate changes on cash |
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| (35) |
|
| (4) |
Net decrease in cash, cash equivalents and restricted cash |
| $ | (17,139) |
| $ | (10,799) |
25
Uses of funds
Operating activities
During the six months ended June 30, 2019, we used $21.2 million of cash to fund operating activities. During the six months ended June 30, 2019, cash used in operating activities reflected our net loss of $20.2 million and a net increase in operating assets and liabilities of $2.7 million, offset by non-cash charges of $1.7 million, principally related to stock-based compensation.
During the six months ended June 30, 2018, we used $9.7 million of cash to fund operating activities. Cash used in operating activities reflected our net loss of $9.4 million and a net increase in operating assets and liabilities of $0.7 million, offset by non-cash charges of $0.4 million, principally related to stock-based compensation.
Investing activities
During the six months ended June 30, 2019, we received $4.4 million in net proceeds from the sale of marketable securities and paid $0.3 million in purchases of property and equipment. During the six months ended June 30, 2018, we made remaining payments of $0.5 million in connection with the asset acquisition of Alizé.
Financing activities
During the six months ended June 30, 2019, we used cash of $0.1 million in principal loan repayments and $0.2 million in the payment of financing costs, offset by $0.2 million in proceeds from the exercise of options and warrants. During the six months ended June 30, 2018, we used cash of $0.1 million in principal loan repayments and $0.5 million in the payment of financing costs.
Funding requirements
We expect our expenses to increase in connection with our ongoing activities, particularly as we continue the research and development of, continue or initiate clinical trials of, and seek marketing approval for, our product candidates. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant commercialization expenses related to program sales, marketing, manufacturing and distribution to the extent that such sales, marketing and distribution are not the responsibility of potential collaborators. Furthermore, we expect to incur additional costs associated with operating as a public company. Accordingly, we will need to obtain substantial additional funding in connection with our continuing operations. If we are unable to estimateraise capital when needed or on attractive terms, we may would be forced to delay, reduce or eliminate our research and development programs or future commercialization efforts.
In April 2019, we entered into an "at-the-market", or ATM, equity distribution agreement with Citigroup Global Markets Inc. acting as sole agent with an aggregate offering value of up to $50.0 million, which allows us to sell our common shares through the amountsfacilities of increased capital outlaysthe Nasdaq Capital Market. Subject to the terms of the equity distribution agreement, we are able to determine, at our sole discretion, the timing and number of shares to be sold under this ATM facility. No common shares have been issued to date under our ATM equity distribution agreement.
As of June 30, 2019, we had cash, cash equivalents, marketable securities and restricted cash of $56.6 million, which we believe are sufficient to fund our planned operations into the fourth quarter of 2020. Our existing cash, cash equivalents, marketable securities and restricted cash are currently expected to be sufficient to fund our current operating expenses associated with completingplans through the developmenttopline results of the Phase 2b portion of our current treatments in development. livoletide pivotal Phase 2b/3 PWS study.
Our future capital requirements will depend on many factors, including:
· | the outcome of our Phase 2b CAH protocol amendment submissions to appropriate regulatory authorities and ethics committees, and the timing of responses to these submissions; |
· | the scope, progress, results and costs of preclinical studies and clinical trials; |
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· | the scope, prioritization and number of our research and development programs; |
· | the costs, timing and outcome of regulatory review of our product candidates; |
· | our ability to establish and maintain collaborations on favorable terms, if at all; |
· | the extent to which we are obligated to reimburse, or entitled to reimbursement of, clinical trial costs under collaboration agreements, if any; |
· | the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending intellectual property-related claims; |
· | the extent to which we acquire or in-license other product candidates and technologies; |
· | the costs of securing manufacturing arrangements for commercial production; and |
· | the costs of establishing or contracting for sales and marketing capabilities if we obtain regulatory approvals to market our product candidates. |
Identifying potential product candidates and conducting preclinical studies and trials;
Until such time, if ever, as we can generate sufficientsubstantial product revenues, from our fertility treatments to become profitable, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and licensing arrangements. We do not have any committed external source of funds. In addition, we may elect to raise additional funds even before we need them if the conditions for raising capital are favorable. To the extent that we raise additional capital through the sale of equity or convertible debt securities, theyour ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect theyour rights ofas a common stockholders.stockholder. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.
If we raise additional funds through additional collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or treatmentsproduct candidates or to grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our fertility treatmentproduct development or future commercialization efforts or grant rights to develop and market treatmentsproduct candidates that we would otherwise prefer to develop and market ourselves.
Contractual Obligations and Commitments
In February 2019, we entered into a five-year lease agreement for office space in Ann Arbor, Michigan commencing April 1, 2019 for our corporate headquarters. Annual rent payments range from $374,000 in the first year and escalate to $421,000 in the fifth year.
In April 2019, we entered into a lease agreement for office space in Lexington, Massachusetts to expand our operations. Monthly rental payments commenced in May 2019 and continue through the lease term, which is scheduled to end in September 2020. We have the right to terminate the lease at any time and upon prior written notice. We will make future rental payments of $74,000 and $96,000 in fiscal 2019 and 2020, respectively.
In May 2019, we funded an escrow arrangement totaling approximately $1.0 million in connection with a sublease agreement for office space located in Waltham, Massachusetts assumed in connection with the Merger. The escrowed amount represents the difference in the annual base rent payable under the sublease agreement and the annual base rent
payable under the overlease for the remaining term of the sublease agreement. The escrowed amount will be released on a monthly basis over the remaining lease term as the annual base rent payments are made by us.
As of June 30, 2019, there were no other material changes in commitments under contractual obligations, compared to the contractual obligations disclosed in our Annual Report.
Off-Balance Sheet Arrangements
We did not have during the periods presented, and we do not currently have, any off-balanceoff balance sheet arrangements as of June 30, 2019, as defined in Item 303(a)(4)(ii) of Regulation S-K.
Critical Accounting Policies
Other than as described under SEC rules.
Not required for trading or speculative purposes. We maintain our cash, cash equivalents and short-term investments with a high quality, accredited financial institution. Our marketable securities are subject to interest rate risk and will fall in value if market interest rates increase.
Evaluation of Disclosure Controls and Procedures.
Our management, with the participation of our principalChief Executive Officer (principal executive officerofficer) and principalChief Financial Officer (principal financial officer,officer), evaluated the effectiveness of our disclosure controls and procedures (as defined in the Securities Exchange Act of 1934 Rules 13a-15(e) or 15d-15(e)) as required by paragraph (b) of Exchange Act Rules 13a-15 or 15d-15, as of SeptemberJune 30, 2018. The term “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act, means controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is accumulated and communicated to the company’s management, including its principal executive and principal financial officers, as appropriate, to allow timely decisions regarding required disclosure. Management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures.2019. Based on the evaluation of our disclosure controls and procedures as of SeptemberJune 30, 2018,2019, our principal executive officerChief Executive Officer and principal financial officerChief Financial Officer concluded that, as of such date, our disclosure controls and procedures were effective at thea reasonable assurance level.
Changes in Internal Controls.
There were no changes in our internal control over financial reporting occurred during the fiscal quarter ended SeptemberJune 30, 20182019 that hashave materially affected, or isare reasonably likely to materially affect, our internal control over financial reporting.
Limitations on Controls
Our disclosure controls and procedures and internal control over financial reporting are designed to provide reasonable assurance of achieving the desired control objectives. Our management recognizes that any control system, no matter how well designed and operated, is based upon certain judgments and assumptions and cannot provide absolute assurance that its objectives will be met. Similarly, an evaluation of controls cannot provide absolute assurance that misstatements due to error or fraud will not occur or that all control issues and instances of fraud, if any, have been detected.
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Liabilities for loss contingencies arising from claims, assessments, litigation, fines, penalties, and plaintiffs filedother sources are recorded when it is probable that a First Amended Class Action Complaint on June 17, 2016. In October 2016, the Superior Court granted the motion to intervene of an additional plaintiff, Westmoreland County Employee Retirement System (“Westmoreland”). On November 7, 2017, the Superior Court denied the plaintiffs’ motion for class certification. On August 14, 2017, the defendants filed their motion for summary judgment against plaintiffs Heather Carlson, Cesar Castellanos, Philipp Hofmann, and Carlos Rivas, which the plaintiffs opposed. On November 21, 2017, the Superior Court allowed the defendants’ motion for summary judgment,liability has been incurred and the claims asserted by plaintiffs Heather Carlson, Cesar Castellanos, Philipp Hofmann, and Carlos Rivas in the consolidated actions were dismissed, leaving Westmoreland as the sole remaining plaintiff. On November 22, 2017, Westmoreland filed a putative class action complaint in the U.S. District Court for the District of Massachusetts against the same defendants alleging the same claims as were alleged in the state court case (Westmoreland County Employee Retirement System v. OvaScience, Inc., et al., No. 1:17-cv-12312-IT (D. Mass.)) (the “Westmoreland Federal Action”). On January 22, 2018, Westmoreland filed a motion to voluntarily dismiss the Superior Court action without prejudice. The defendants opposed that motion. Oral argument on Westmoreland’s motion for voluntary dismissal was held on April 3, 2018. On April 5, 2018, the Superior Court allowed Westmoreland’s motion for voluntary dismissal without prejudice. The Superior Court entered final judgment on April 18, 2018, dismissing Westmoreland’s claims without prejudice and dismissing the claims of plaintiffs Heather Carlson, Cesar Castellanos, Philipp Hofmann, and Carlos Rivas with prejudice. In the Westmoreland Federal Action, on August 24, 2018, Westmoreland filed a stipulation and proposed order, signed by counsel for all parties, voluntarily dismissing that action without prejudice (the “Westmoreland Stipulation”). Pursuant to the Westmoreland Stipulation, Westmoreland agreed that it will not re-file or reassert its claims arising under the Securities Act that were alleged in its complaint in the Westmoreland Federal Action and will pursue damages, if any, only as a member of the putative class in the Dahhan Action (described below). On August 28, 2018, the court in the Westmoreland Federal Action entered an order approving the Westmoreland Stipulation. Accordingly, the Westmoreland Federal Action has been dismissed.
On November 9, 2016, a purported shareholder derivative action was filed in the Business Litigation Session of the Suffolk County Superior Court in the Commonwealth of Massachusetts (Cima v. Dipp et al., No. 16-3443-BLS116‑3443‑BLS1 (Mass. Sup. Ct.)) against certain present and former officers and directors of OvaScience and one current director of the Company (a former director of OvaScience) and the CompanyOvaScience as a nominal defendant alleging breaches of fiduciary duty, unjust enrichment, abuse of control, gross mismanagement and corporate waste for purported actions related to the Company’sOvaScience’s January 2015 follow-on public offering. On February 22, 2017, the court approved the parties’ joint stipulation to stay all proceedings in the action until further notice. Following a status conference in December 2017, the stay was lifted. On January 23,25, 2018, at the parties’ request, the court entered a second order staying all proceedings in the action until further order of the court. The Company believes that the complaint is without merit and intends to defend against the litigation. There can be no assurance, however, that the Company will be successful. A resolution of this lawsuit adverse toAt present, the Company or the other defendants could have a material effect on our consolidated financial position and results of operations in the period in which the lawsuit is resolved. At present, we are unable to estimate potential losses, if any, related to the lawsuit.
On March 24, 2017, a purported shareholder class action lawsuit was filed in the U.S. District Court for the District of Massachusetts (Dahhan v. OvaScience, Inc., et al., No. 1:17-cv-10511-IT17‑cv‑10511‑IT (D. Mass.)) against the CompanyOvaScience and certain of our former officers and directors of OvaScience alleging violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 (the “Dahhan Action”"Dahhan Action"). On July 5, 2017, the Courtcourt entered an order approving the appointment of Freedman Family Investments LLC as lead plaintiff, the firm of Robins Geller Rudman & Dowd LLP as lead counsel and the Law Office of Alan L. Kovacs as local counsel. Plaintiff filed an amended complaint on August 25, 2017. The defendantsCompany filed a motion to dismiss the amended complaint, which the Courtcourt denied on July 31, 2018. On August 14, 2018, the Company answered the amended complaint. We believeThe parties presently are engaged in discovery. The Company believes that the amended complaint is without merit and intendintends to defend against the litigation. There can be no assurance, however, that wethe Company will be successful. A resolution of this lawsuit adverse to the Company or the other defendants could have a material effect on ourthe Company’s consolidated financial position and results of operations in the period in which the lawsuit is resolved.operations. At present, we arethe Company is unable to estimate potential losses, if any, related to the lawsuit.
On July 27, 2017, a purported shareholder derivative complaint was filed in the U.S. District Court for the District of Massachusetts (Chiu v. Dipp et al., No. 1:17-cv-11382-IT17‑cv‑11382‑IT (D. Mass.)) against certain of the Company's present and former directors and the CompanyOvaScience, as a nominal defendant, certain former officers and directors of OvaScience and one current director of the Company (a former director of OvaScience) alleging breach of fiduciary duty, unjust enrichment and violations of Section 14(a) of the Securities Exchange Act of 1934 alleging that compensation awarded to the director defendants was excessive and seeking redress for purported actions related to the Company’sOvaScience’s January 2015 follow-on public offering and other public statements. On
Between October 16, 2018 a Company stockholderand November 21, 2018, five putative class action lawsuits were filed a complaint in various federal District Courts against OvaScience, Inc. and the U.S. District Court for the DistrictOvaScience Board of Delaware (Directors related to OvaScience’s proposed
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merger with Millendo Therapeutics, Inc.: Cunningham v. Kroeger, et al.,No. 1:18-cv-01595-CFC18‑cv‑01595 (D. Del.)) against the Company, its chief executive officer filed Oct. 16, 2018); Adlard v. OvaScience, Inc., et al., No. 1:18‑cv‑12332 (D. Mass. filed Nov. 6, 2018); Wheby v. OvaScience, Inc., et al., No. 1:18‑cv‑1811 (D. Del. filed Nov. 16, 2018); Cuenca Aubets v. OvaScience, Inc., et al., No. 1:18‑cv‑10882 (S.D.N.Y. filed Nov. 20, 2018); and the members of the Company’s Board of Directors, allegingKim v. OvaScience, Inc., et al., No. 1:18‑cv‑10939 (S.D.N.Y. filed Nov. 21, 2018). The Complaints each alleged violations of Section 14(a) of the Securities Exchange Act of 1934 and Rule 14a-914a‑9 promulgated thereunder, and as against the individual defendants, alleging violations of Section 20(a) of the Securities Exchange Act of 1934. The Cunningham plaintiff allegesalleged that the defendants made materially misleading disclosures in aOvaScience’s Form S-4S‑4 Registration Statement filed with the SEC on September 26, 2018 in connection with Company’somitted or misrepresented material information regarding OvaScience’s proposed merger with Millendo Therapeutics, Inc. (“Millendo”), by allegedly omitting material information concerning the sale process. The plaintiff seeks declaratoryAdlard, Wheby, Cuenca Aubets and injunctive relief to enjoin the proposed merger with Millendo, rescissory damages against the individual defendants, including pre-judgment and post-judgment interest, costs and attorneys’ fees. We believeKim plaintiffs alleged that the complaint is without merit and intend to defend against the litigation. There can be no assurance, however, that we will be successful. At present, we are unable to estimate potential losses, if any, related to the lawsuit.
In addition to the matters described above, the Company may be a party to litigation and subject to claims incident to the ordinary course of business from time to time. Regardless of the outcome, litigation can have an adverse impact on the Company because of defense and settlement costs, diversion of management resources and other information set forth in this report, youfactors.
You should carefully consider the risk factors discussedrisks described below, as well as general economic and business risks and the other information in Part I, Item 1A “Risk Factors”this Quarterly Report on Form 10‑Q and in our Annual Report on Form 10-K for the year ended December 31, 2017. With the exception2018. The occurrence of any of the risk factorsevents or circumstances described below there hereor other adverse events could have been noa material changes inadverse effect on our business, results of operations and financial condition and could cause the trading price of our common stock to decline. Additional risks or additionsuncertainties not presently known to us or that we currently deem immaterial may also harm our business.
Risks Related to the risk factors included inReverse Merger
The risks arising with respect to the historic OvaScience business and operations may be different from what we anticipate, which could lead to significant, unexpected costs and liabilities and could materially and adversely affect our Annual Report on Form 10-Kbusiness going forward.
It is possible that we may not have fully anticipated the extent of the risks associated with the recent Reverse Merger we completed with OvaScience. After the Reverse Merger, OvaScience’s historic business was discontinued, but prior to the transaction OvaScience had a significant operating history. As a consequence, we may be subject to claims, demands for payment, regulatory issues, costs and liabilities that were not and are not currently expected or anticipated. Notwithstanding our exercise of due diligence pre-transaction and winding down of the OvaScience business post-transaction, the risks involved with taking over a business with a significant operating history and the costs and liabilities associated with these risks may be greater than we anticipate. We may not be able to contain or control the costs or liabilities associated with OvaScience’s historic business, which could materially and adversely affect our business, liquidity, capital resources or results of operation.
Risks Related to Our Financial Position and Need for Additional Capital
We have incurred significant operating losses since inception and anticipate that we will continue to incur substantial operating losses for the yearforeseeable future and may never achieve or maintain profitability.
Since inception, we have incurred significant operating losses and negative operating cash flows and there is no assurance that we will ever achieve or sustain profitability. Our net loss was $84.6 million and $27.2 million for the years ended December 31, 2017 and our Quarterly Reports on Form 10-Q2018, respectively, and $9.9 million and $20.2 million for the quartersthree and six
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months ended March 31, 2018 and June 30, 2018.2019, respectively. As of June 30, 2019, we had an accumulated deficit of $184.3 million. We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future. We have devoted substantially all of our efforts to the acquisition of and preclinical and clinical development of two of our product candidates, livoletide and nevanimibe, as well as to building our management team and infrastructure. It could be several years, if ever, before we have a commercialized product and our commercialized products, if any, may not be profitable. The net losses we incur may fluctuate significantly from quarter to quarter and year to year. We anticipate that our expenses will increase significantly in connection with our ongoing activities such as:
· | continuing the ongoing and planned clinical development of livoletide and nevanimibe; |
· | continuing the preclinical development and potential clinical development of MLE-301; |
· | initiating preclinical studies and clinical trials for any additional diseases for our current product candidates and any future product candidates that we may pursue; |
· | building a portfolio of product candidates through the acquisition or in-license of drugs or product candidates and technologies; |
· | developing, maintaining, expanding and protecting our intellectual property portfolio; |
· | manufacturing, or having manufactured, clinical and commercial supplies of our product candidates; |
· | seeking marketing approvals for our current and future product candidates that successfully complete clinical trials; |
· | establishing a sales, marketing and distribution infrastructure to commercialize any product candidate for which we may obtain marketing approval; |
· | hiring additional administrative, clinical, regulatory and scientific personnel; and |
· | incurring additional costs associated with operating as a public company. |
In order to become and remain profitable, we will need to develop and eventually commercialize, on our own or with collaborators, one or more product candidates with significant market potential. This will require us to be successful in a range of challenging activities, including completing clinical trials of livoletide, nevanimibe, and MLE-301, developing commercial scale manufacturing processes, obtaining marketing approval, manufacturing, marketing and selling any current and future product candidates for which we may obtain marketing approval, and satisfying any post-marketing requirements. We may never succeed in any or all of these activities and, even if we do, we may never generate revenue from product sales or achieve profitability.
Because of the numerous risks and uncertainties associated with pharmaceutical products and development, we are unable to accurately predict the timing or amount of increased expenses or when, or if, we will be able to achieve profitability. For example, enrollment for our Phase 2b CAH clinical trial remains paused in certain countries while appropriate regulatory authorities and ethics committees review our submissions for a protocol amendment. We are unable to predict the outcome of these submissions or the timing of responses to these submissions. If we are required by the U.S. Food and Drug Administration, or FDA, or other regulatory authorities such as the European Medicines Agency, or EMA, to amend existing studies or trials, to perform studies and trials in addition to those currently expected, or if there are any delays in the development or in the completion of any planned or future preclinical studies or clinical trials of our current or future product candidates, our expenses could increase and profitability could be further delayed.
Even if we do achieve profitability, we may not meet the continued listing standards of The Nasdaq Capital Market, which requirebe able to sustain or increase profitability on a minimum closing bid price of $1.00 per share.quarterly or annual basis. Our failure to meet Nasdaq’s continued listing standardsbecome and remain profitable would decrease our value and could impair our ability to raise capital, maintain our research and development efforts, expand our business or continue our operations. A decline in our value also could cause you to lose all or part of your investment.
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We have a limited operating history and have never generated any revenue from product sales, which may make it difficult to assess our future viability.
We are a clinical stage biopharmaceutical company with a limited operating history. Our operations to date, with respect to the development of our product candidates, have been limited to organizing and staffing the business, business planning, raising capital, acquiring our product candidates and other assets and conducting preclinical and clinical development of our product candidates. We have not yet demonstrated an ability to successfully complete clinical development of a product candidate, obtain marketing approval, manufacture a commercial-scale drug (or arrange for a third-party to do so on our behalf), or conduct sales and marketing activities necessary for successful commercialization. Consequently, our predictions about our future success or viability may not be as accurate as they could be if we had more experience developing product candidates.
Our ability to generate revenue from product sales and achieve profitability depends on our ability, alone or with any future collaborations, to successfully complete the development of, and obtain the regulatory approvals necessary to commercialize, livoletide, nevanimibe and any additional product candidates that we may pursue in the future. We do not anticipate generating revenue from product sales for the next several years, if ever. Our ability to generate revenue from product sales depends heavily on our or any future collaborators’ success in:
· | timely and successful completion of development activities of our current product candidates; |
· | obtaining and maintaining regulatory and marketing approvals for livoletide, nevanimibe and any future product candidates for which we successfully complete clinical trials; |
· | launching and commercializing any product candidates for which we obtain regulatory and marketing approval by establishing a sales force, marketing and distribution infrastructure or, alternatively, collaborating with a commercialization partner; |
· | qualifying for coverage and adequate reimbursement by government and third-party payors for our current or any future product candidates, if approved, both in the United States and internationally, and reaching acceptable agreements with such government and third-party payors on pricing terms; |
· | developing, validating and maintaining a commercially viable, sustainable, scalable, reproducible and transferable manufacturing process for livoletide, nevanimibe or any future product candidates that are compliant with current good manufacturing practices, or cGMP; |
· | establishing and maintaining supply and manufacturing relationships with third parties that can provide an adequate amount and quality of drugs and services to support our planned clinical development, as well as the market demand for livoletide, nevanimibe and any future product candidates, if approved; |
· | obtaining market acceptance, if and when approved, of livoletide, nevanimibe or any future product candidates as a viable treatment option by physicians, patients, third-party payors and others in the medical community; |
· | effectively addressing any competing technological and market developments; |
· | implementing additional internal systems and infrastructure, as needed; |
· | negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter, and performing our obligations pursuant to such arrangements; |
· | maintaining, protecting and expanding our portfolio of intellectual property rights, including patents, trade secrets and know-how; |
· | avoiding and defending against third-party interference or infringement claims; and |
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· | attracting, hiring and retaining qualified personnel. |
We will require additional capital to finance our operations, which may not be available on acceptable terms, if at all. Failure to obtain capital when needed may force us to delay, limit or terminate certain of our development programs, future commercialization efforts or other operations.
We expect our expenses to increase in connection with our ongoing activities, particularly as we continue to develop, and if approved, commercialize, livoletide, nevanimibe, and MLE-301. Additionally, if we obtain marketing approval for our product candidates, we expect to incur significant expenses related to manufacturing, marketing, sales and distribution. Furthermore, we expect to incur additional costs associated with operating as a public company.
As of June 30, 2019, our cash, cash equivalents, marketable securities and restricted cash were $56.6 million. Our existing cash, cash equivalents, marketable securities and restricted cash are currently expected to be sufficient to fund our current operating plans into the fourth quarter of 2020, which we expect will be sufficient to fund our operating plans through the topline results of the Phase 2b portion of our livoletide pivotal Phase 2b/3 PWS study. However, our operating plans may change as a result of many factors currently unknown to us, and we may need to seek additional funds sooner than planned, through public or private equity or debt financings, third-party funding, and marketing and distribution arrangements, as well as other collaborations, strategic alliances and licensing arrangements, or a combination of these approaches. In any event, we will require additional capital to pursue preclinical and clinical activities, regulatory approval and the commercialization of our current and future product candidates. Even if we believe we have sufficient capital for our current or future operating plans, we may seek additional capital if market conditions are favorable or if we have specific strategic considerations. If we elect to do so, additional capital may not be available to us on acceptable terms, if at all. Our ability to access additional capital, and as a result our operating results and liquidity needs, could be negatively affected by market fluctuations and economic downturn. Any additional capital raising efforts may divert our management from its day-to-day activities, which may adversely affect our ability to develop and commercialize our current and future product candidates.
Raising additional capital by issuing equity or debt securities may cause dilution to our existing stockholders, and raising funds through lending and licensing arrangements may restrict our operations or require us to relinquish proprietary rights.
Until such time as we can generate substantial revenue from product sales, if ever, we expect to finance our cash needs through a combination of equity and debt financings, strategic alliances and license and development agreements in connection with any future collaborations. To the extent that we raise additional capital by issuing equity securities, our existing stockholders’ ownership may experience substantial dilution, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a stockholder. Equity and debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as redeeming our shares, making investments, incurring additional debt, making capital expenditures or declaring dividends.
The incurrence of indebtedness could result in increased fixed payment obligations and we may be required to agree to certain restrictive covenants therein, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could adversely affect our ability to conduct our business.
If we raise additional capital through collaborations, strategic alliances or third-party licensing arrangements, we may have to relinquish valuable rights to our intellectual property, future revenue streams, research programs or product candidates, or grant licenses on terms that may not be favorable to us. If we are unable to raise additional capital through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts, or grant rights to develop and market product candidates that we would otherwise develop and market ourselves.
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We may be required to make payments under licenses applicable to livoletide, nevanimibe and MLE-301.
We have certain milestone and royalty payments related to livoletide, nevanimibe and MLE-301. We acquired worldwide, exclusive rights to nevanimibe pursuant to our license agreement with the delistingRegents of the University of Michigan, or the University of Michigan, entered into in June 2013, or the UM License Agreement. Under the terms of the UM License Agreement, we are obligated to make significant milestone and royalty payments in connection with the attainment of certain development steps and the sale of resulting products with respect to nevanimibe, as well as other material obligations. In addition, pursuant to an assignment agreement for certain patents and patent applications relating to livoletide, we are also required to pay royalties on commercial sales and licensing of livoletide to the assignors. We acquired worldwide, exclusive rights to MLE-301 pursuant to the Roche License Agreement. Under the terms of the Roche License Agreement, we are obligated to make significant milestone and royalty payments in connection with the attainment of certain development steps and the sale of resulting products with respect to MLE-301, as well as other material obligations. In addition, we are required to share a portion of any net proceeds received in connection with certain agreements that we may enter into with third parties to develop and commercialize MLE-301. If milestone or other non-royalty obligations become due, we may not have sufficient funds available to meet our obligations, which will materially adversely affect our business operations and financial condition. If milestone or other non-royalty obligations become due, we may not have sufficient funds available to meet our obligations, which will materially adversely affect our business operations and financial condition.
We may expend our limited resources to pursue a particular product candidate or disease and fail to capitalize on product candidates or diseases that may be more profitable or for which there is a greater likelihood of success.
Because we have limited financial and managerial resources, we focus on research programs and product candidates for specific indications. As a result, we may forego or delay pursuit of opportunities with respect to our own product candidates for additional indications and other product candidates or diseases that later prove to have greater commercial potential. Our resource allocation decisions may ultimately not result in successful clinical development programs and may cause us to fail to capitalize on other viable product candidates, commercial products or profitable market opportunities. In addition, our spending on current and future research and development programs and product candidates for specific indications may not yield any commercially viable products. For example, and as previously disclosed in our periodic reports in 2019, in our Phase 2 clinical trial for CS we have experienced slower than anticipated enrollment, which could make impractical further development of nevanimibe for the treatment of CS. As a result of the difficulty in enrolling this trial, we elected to discontinue this Phase 2 clinical trial in August 2019 and will suspend development of nevanimibe for the treatment of CS.
If we do not accurately evaluate the commercial potential or target market for a particular product candidate, we may relinquish valuable rights to that product candidate through sale, collaboration, licensing or other royalty arrangements in cases in which it would have been advantageous for us to retain sole development and commercialization rights.
Risks Related to Development and Commercialization
Our future success is dependent on the successful clinical development, regulatory approval and subsequent commercialization of livoletide, nevanimibe and any future product candidates. If we are not able to obtain the required regulatory approvals, we will not be able to commercialize our current or future product candidates and our ability to generate revenue will be adversely affected.
We do not have any drugs that have received regulatory approval and may never be able to develop marketable product candidates. We expect that a substantial portion of our common stock,efforts and expenses for the foreseeable future will be devoted to the clinical development of livoletide and nevanimibe, and as a result, our business currently depends heavily on the successful development, regulatory approval and commercialization of these product candidates. We cannot be certain that livoletide or nevanimibe will receive regulatory approval or be successfully commercialized even if we receive regulatory approval. The research, testing, manufacturing, safety, efficacy, labeling, approval, sale, marketing and distribution of our product candidates are, and will remain, subject to comprehensive regulation by the FDA and other regulatory agencies in the United States and similar foreign regulatory authorities. Failure to obtain regulatory approval for livoletide or nevanimibe in the United States or other jurisdictions will prevent us from commercializing and marketing livoletide or nevanimibe.
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The Phase 2b portion of our recently initiated Phase 2b/3 PWS trial may or may not be sufficient to support FDA approval depending on the data. Additionally, the FDA may require additional data (for example, in children) in order to support an NDA approval in PWS in the United States.
Even if we were to successfully obtain approval from the FDA and comparable foreign regulatory authorities for our product candidates, any approval might contain significant limitations related to use restrictions for specified age groups, warnings, precautions or contraindications, or may be subject to burdensome post-approval study or risk management requirements. If we are unable to obtain regulatory approval for our product candidates, or any approval contains significant limitations, on our own or with any future collaborators, we may not be able to obtain sufficient funding or generate sufficient revenue to continue the development of any other product candidate that we may in-license, develop or acquire in the future.
Furthermore, even if we obtain regulatory approval for livoletide or nevanimibe, we will still need to develop a commercial infrastructure, or otherwise develop relationships with collaborators to commercialize, establish a commercially viable pricing structure and obtain approval for adequate reimbursement from third-party and government payors. If we, or our collaborators, are unable to successfully commercialize livoletide or nevanimibe, we may not be able to generate sufficient revenue to continue our business.
Preclinical studies or earlier clinical trials are not necessarily predictive of future results and the results of our clinical trials may not support our livoletide or nevanimibe claims.
Our product candidates, livoletide and nevanimibe, are still in development and will require extensive clinical testing before we are prepared to submit an NDA or other similar application for regulatory approval. Enrollment for our Phase 2b CAH clinical trial remains paused in certain countries while appropriate regulatory authorities and ethics committees review our submissions for a protocol amendment. We are unable to predict the outcome of these submissions or the timing of responses to these submissions. We cannot predict with any certainty if or when we might submit an NDA for regulatory approval for livoletide or nevanimibe for the treatment of any indication or whether any such application will be approved by the relevant regulatory authority. Human clinical trials are very expensive and difficult to design and implement, in part because they are subject to rigorous regulatory requirements. For instance, the FDA or foreign regulatory authorities may not agree with our proposed endpoints for any clinical trials of livoletide or nevanimibe, even if validated in prior clinical trials of similar product candidates, which may delay the commencement of our future clinical trials. The FDA or foreign regulatory authorities may also not agree with our proposed trial designs or dosing regimens, which may likewise prevent or delay the commencement of our future clinical trials. The clinical trial process is also time-consuming. We estimate that clinical trials of livoletide and nevanimibe for each of the indications that we are pursuing will take the next several years to complete. Failure can occur at any stage of the trials, and we could encounter problems that cause us to abandon or repeat clinical trials. Further, we may encounter challenges in the clinical development of product candidates for reasons unrelated to the observed safety or efficacy of such product candidates in prior clinical trials. In addition, because we may at times pursue the treatment of multiple indications for a single product candidate, setbacks or failures in, or termination of, clinical development for one indication may have a negative impact on the clinical development for the treatment of other indications. For example, and as previously disclosed in our periodic reports in 2019, in our Phase 2 clinical trial for CS we have experienced slower than anticipated enrollment, which could make impractical further development of nevanimibe for the treatment of CS. As a result of the difficulty in enrolling this trial, we elected to discontinue this Phase 2 clinical trial in August 2019 and will suspend development of nevanimibe for the treatment of CS.
Success in preclinical testing and early clinical trials does not ensure that later and pivotal clinical trials will generate the same results, or otherwise provide adequate data to demonstrate the safety and efficacy of a product candidate. Frequently, product candidates that have shown promising results in early clinical trials have subsequently suffered significant setbacks in later or pivotal clinical trials. For example, we expended substantial time and resources on a previous product candidate, MLE4901, an NK3R antagonist, which we ceased developing in 2017 due to concerns relating to elevated liver enzymes observed in clinical trials.Our approach to targeting orphan endocrine diseases where current therapies do not exist or are insufficient, is novel and unproven, and as such, the cost and time needed to develop livoletide and nevanimibe is difficult to predict and our efforts may not be successful. If we do not observe favorable results in future or planned clinical trials of livoletide and nevanimibe, we may decide to delay or abandon development of livoletide and nevanimibe, which could harm our business, financial condition and results of operations. A number of
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companies in the biopharmaceutical industry have suffered significant setbacks in advanced clinical trials due to lack of efficacy or adverse safety profiles, notwithstanding promising results in earlier trials.
We may encounter substantial delays in our clinical trials or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.
Before obtaining marketing approval from regulatory authorities for the sale of livoletide, nevanimibe and any future product candidates, we must conduct extensive clinical trials to demonstrate the safety and efficacy of the product candidate for its intended indications. We cannot guarantee that any clinical trials will be conducted as planned or completed on schedule, if at all. For example, enrollment for our Phase 2b CAH clinical trial remains paused in certain countries while appropriate regulatory authorities and ethics committees review our submissions for a protocol amendment. We are unable to predict the outcome of these submissions or the timing of responses to these submissions. A failure of one or more clinical trials can occur at any stage of testing. Events that may prevent successful or timely completion of clinical development include:
· | failure to obtain regulatory approval to commence a trial; |
· | unforeseen safety issues; |
· | determination of dosing issues; |
· | lack of effectiveness during clinical trials; |
· | inability to reach agreement on acceptable terms with prospective contract research organizations, or CROs, and clinical trial sites; |
· | slower than expected rates of patient recruitment, failure to recruit adequate numbers of suitable patients to participate in our clinical trials or failure to maintain participation of recruited patients in clinical trials; |
· | failure to manufacture sufficient quantities of a product candidate for use in clinical trials; |
· | inability to monitor patients adequately during or after treatment; and |
· | inability or unwillingness of medical investigators to follow our clinical protocols. |
Further, we, the FDA, an institutional review board (“IRB”), or other regulatory authority may suspend our clinical trials at any time if it appears that we or our collaborators are failing to conduct a trial in accordance with regulatory requirements, including, for example, the FDA’s good clinical practice (“GCP”), regulations, that we are exposing participants to unacceptable health risks, or if the FDA or other regulatory authority, as the case may be, finds deficiencies in our investigational new drug (“IND”), application or other submissions, or the manner in which the clinical trials are conducted. Therefore, we cannot predict with any certainty the schedule for commencement and completion of future clinical trials. If we experience delays in the commencement or completion of our clinical trials, or if we terminate a clinical trial prior to completion, the commercial prospects of our current and future product candidates could be harmed, and our ability to generate revenue from our current or future product candidates, once approved, may be delayed or eliminated. In addition, any delays in our clinical trials could increase our costs, slow down the approval process and jeopardize our ability to commence product sales and generate revenue. Any of these occurrences may harm our business, financial condition and results of operations. In addition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates.
Moreover, principal investigators for our clinical trials may serve as our scientific advisors or consultants from time to time and receive compensation in connection with such services. We will be required to report these relationships to the FDA or other regulatory authorities as part of the drug approval process. The FDA or other regulatory authorities may conclude that a financial relationship between us and a principal investigator has created a conflict of interest or
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otherwise affected interpretation of the trial results. They may therefore question the integrity of the data generated at the applicable clinical trial site and the utility of the clinical trial itself may be jeopardized. This could result in a delay in approval, or rejection, of our marketing applications by the FDA or other regulatory authority, as the case may be, and may ultimately lead to the denial of marketing approval of one or more of our product candidates.
We have never obtained marketing approval for a product candidate and we may be unable to obtain, or may be delayed in obtaining, marketing approval for our current product candidates or any future product candidates that we may develop.
We have never obtained marketing approval for a product candidate. It is possible that the FDA may refuse to accept for substantive review any NDAs that we submit for our product candidates or may conclude after review of our data that our application is insufficient to obtain marketing approval of our product candidates. If the FDA does not accept or approve our NDAs for any of our product candidates, it may require that we conduct additional clinical trials, preclinical studies or manufacturing validation studies and submit that data before it will reconsider our applications. Depending on the extent of these or any other FDA-required trials or studies, approval of any NDA or application that we submit may be delayed by several years, or may require us to expend more resources than it has available. It is also possible that additional trials or studies, if performed and completed, may not be considered sufficient by the FDA to approve our NDAs.
Any delay in obtaining, or an inability to obtain, marketing approvals would prevent us from commercializing our product candidates, generating revenues and achieving and sustaining profitability. If any of these outcomes occurs, we may be forced to abandon our development efforts for our product candidates, which could significantly harm our business, prospects, operating results and financial condition.
Enrollment and retention of patients in clinical trials is a competitive, expensive and time-consuming process and could be made more difficult or rendered impossible by multiple factors outside our control.
Identifying and qualifying patients to participate in our clinical trials is critical to our success. We may encounter delays in enrolling, or be unable to enroll, a sufficient number of patients to complete any of our clinical trials, and even once enrolled we may be unable to retain a sufficient number of patients to complete any of our trials. Patient enrollment and retention in clinical trials, including our recently initiated Phase 2b/3 clinical trial of livoletide in PWS patients, depends on many factors, including: the size of the patient population, the nature of the trial protocol, our ability to recruit clinical trial investigators with the appropriate competencies and experience, the existing body of safety and efficacy data with respect to the study drug, the number and nature of competing treatments and ongoing clinical trials of competing drugs for the same disease, the proximity of patients to clinical sites and the eligibility criteria for the trials, our ability to obtain and maintain patient consents and the risk that patients enrolled in clinical trials will drop out of the trials before completion.
The competitive nature of clinical trials in the pharmaceutical and biotechnology industries may make it difficult for us to recruit a sufficient number of patients to complete any of our clinical trials, or may increase costs. We may not be able to initiate or continue to support clinical trials of our product candidates for one or more indications, or any future product candidates, if we are unable to locate and enroll a sufficient number of eligible participants in these trials as required by the FDA or other regulatory authorities. For example, and as previously disclosed in our periodic reports in 2019, in our Phase 2 clinical trial for CS we have experienced slower than anticipated enrollment, which could make impractical further development of nevanimibe for the treatment of CS. As a result of the difficulty in enrolling this trial, we elected to discontinue this Phase 2 clinical trial in August 2019 and will suspend development of nevanimibe for the treatment of CS. Even if we are able to enroll a sufficient number of patients in our clinical trials, if the pace of enrollment is slower than we expect, the development costs for our product candidates may increase and the completion of our trials may be delayed or our trials could become too expensive or impractical to complete.
Our ability to enroll and retain patients in clinical trials of livoletide may be adversely impacted by the fact that livoletide is administered by subcutaneous injection. Furthermore, any negative results we may report in clinical trials of our product candidates may make it difficult or impossible to recruit and retain patients in other clinical trials of those product candidates. Delays or failures in planned patient enrollment or retention may result in increased costs, program delays or both, which could have a harmful effect on our ability to develop livoletide and nevanimibe, or could render
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further development impossible. In addition, we may rely on CROs and clinical trial sites to ensure proper and timely conduct of our future clinical trials and, while we intend to enter into agreements governing their services, we will be limited in our ability to compel their actual performance.
Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit their commercial potential or result in significant negative consequences following any potential marketing approval.
During the conduct of clinical trials, clinical investigators monitor changes in patients’ health, including illnesses, injuries and discomforts. Often, it is not possible to determine whether or not the product candidate being investigated caused these conditions, and regulatory authorities may draw different conclusions or require additional testing to confirm these determinations if they occur. In addition, it is possible that as we test livoletide, nevanimibe or any other product candidate in larger, longer and more extensive clinical programs, or as use of these product candidates becomes more widespread if they receive regulatory approval, illnesses, injuries, discomforts and other adverse events that were observed in earlier trials, as well as conditions that did not occur or went undetected in previous trials, will be observed or reported by subjects. If clinical testing indicates that livoletide, nevanimibe or any future product candidate has side effects or causes serious or life-threatening side effects, we may need to change the design of ongoing clinical trials or adjust dosing levels in ongoing or future clinical trials, and the development of the product candidate may be delayed or terminated entirely. For example, in recent years clinical trials by other companies evaluating product candidates for treatment of PWS, which employed a different mechanism of action than livoletide, have resulted in serious adverse events, including patient deaths, and the eventual termination of the clinical trial and/or clinical development program. Further, if the product candidate has received regulatory approval, such approval may be revoked, which would materially harm our business, prospects, operating results and financial condition.
Moreover, if we elect or are required to modify, delay, suspend or terminate any clinical trial for our product candidates, the commercial prospects of our product candidates may be harmed and our ability to generate revenue through their sale may be delayed or eliminated. Any of these occurrences may harm our business, financial condition and prospects significantly.
We face substantial competition, and our operating results will suffer if we fail to compete effectively.
The commercialization of new drugs is competitive, and we may face worldwide competition from major pharmaceutical companies, specialty pharmaceutical companies, biotechnology companies and ultimately generic companies. Our competitors may develop or market therapies that are more effective, safer or less costly than any that we are commercializing, or may obtain regulatory or reimbursement approval for their therapies more rapidly than we may obtain approval for ours.
We are aware of a number of companies that are working to develop drugs that would compete, directly or indirectly, against livoletide for the treatment of PWS, nevanimibe for the treatment of classic congenital adrenal hyperplasia or CAH and MLE-301 for the treatment of vasomotor symptoms or VMS.
Soleno Therapeutics, Inc. is currently developing diazoxide choline controlled release, an ATP-sensitive potassium channel agonist, and Levo Therapeutics, Inc. is pursuing development of carbetocin, a long-acting analogue of oxytocin, for the treatment of PWS. Each of Saniona AB, GLWL Research Inc. and Insys Therapeutics, Inc. have also announced or initiated smaller trials in PWS for the treatment of hyperphagia. There are also a number of compounds in preclinical development.
We are aware of three other companies developing treatments for patients with CAH: Diurnal Group PLC is developing an exogenous cortisol treatment with a modified release intended to more closely match the physiological release profile of cortisol but recently announced a failed Phase 3 study and placed their U.S. development activities on hold. They intend to submit a Market Authorization Application to the EMA in the fourth quarter of 2019. Both Spruce Biosciences, Inc and Neurocrine Biosciences, Inc. are developing CRF1 antagonists and have completed Phase 2 studies in adults. Neurocrine has announced the initiation of a Phase 2 study in a pediatric population.
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We are aware of three competing NK3R antagonists currently in clinical development for VMS. Astellas is developing fezolinetant and is preparing for a Phase 3 trial, KaNDy Therapeutics is currently conducting a Phase 2b trial for NT-814 a dual NK1/3 receptor antagonist and Sojournix is currently studying SJX-653 in a Phase 1 trial. Also, Acer Therapeutics is licensing of osenetant, an NK3R antagonist, which has previously been in clinical studies.
Many of our existing or potential competitors may have substantially greater financial, technical and human resources than we do, and significantly greater experience in the discovery and development of product candidates, including in the recruitment of patients for clinical trials, as well as in obtaining regulatory approvals of those product candidates in the United States and in foreign countries. Our current and potential future competitors may also have significantly more experience commercializing drugs that have been approved for marketing. If we are not able to compete effectively against existing and potential competitors, our business and financial condition may be harmed.
Mergers and acquisitions in the pharmaceutical and biotechnology industries could result in even more resources being concentrated among a small number of our competitors. Competition may reduce the number and types of patients available to us to participate in clinical trials, because some patients who might have opted to enroll in our trials may instead opt to enroll in a trial being conducted by one of our competitors.
Competition may further increase as a result of advances in the commercial applicability of technologies and greater availability of capital for investment in these industries. Our competitors may succeed in developing, acquiring or licensing, on an exclusive basis, drugs that are more effective or less costly than any product candidate that we may develop.
Any inability to successfully complete clinical development of a product candidate could result in additional costs or impair or eliminate our ability to generate revenue from future sales of such product candidate, if approved, or from any regulatory and commercialization milestone with respect to such product candidate. In addition, if we make manufacturing or formulation changes to livoletide or nevanimibe, we may need to conduct additional testing to bridge our modified product candidates to earlier versions. Clinical trial delays could also shorten any periods during which we may have the exclusive right to commercialize livoletide or nevanimibe, or allow our competitors to bring comparable drugs to market before we do, which could impair our ability to successfully commercialize livoletide or nevanimibe, and may harm our business, financial condition and results of operations.
Established pharmaceutical and biotechnology companies may invest heavily to accelerate discovery and development of novel compounds or to in-license novel compounds that could make livoletide or nevanimibe less competitive. In addition, any new product that competes with an approved product must demonstrate compelling advantages in efficacy, convenience, tolerability and safety in order to overcome price competition and to be commercially successful. Accordingly, our competitors may succeed in obtaining patent protection, discovering, developing and receiving FDA or other regulatory authority approval, or commercializing drugs before we do, which would have an adverse impact on our business and results of operations.
The availability of our competitors’ products could limit the demand and the price we are able to charge for any product candidate we develop. The inability to compete with existing or subsequently introduced drugs would harm our business, prospects, financial condition and results of operations.
The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and even if we obtain approval for a product candidate in one country or jurisdiction, we may never obtain approval for, or commercialize, that product candidate in any other jurisdiction, which would limit our ability to realize our full market potential.
Prior to obtaining approval to commercialize a product candidate in any jurisdiction, we or our collaborators must demonstrate with substantial evidence from well-controlled clinical trials, and to the satisfaction of the FDA or foreign regulatory agencies, that such product candidates are safe and effective for their intended uses. Results from preclinical studies and clinical trials can be interpreted in different ways. Even if our product candidates meet their safety and efficacy endpoints in clinical trials, the FDA or foreign regulatory agencies may believe the clinical trials do not show the appropriate balance of safety and efficacy in the indication being sought or may interpret the data differently than we do, and deem the results insufficient to demonstrate the appropriate balance of safety and efficacy at the level required
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for product approval. Further, the regulatory authorities may not complete their review processes in a timely manner, or we may otherwise not be able to obtain regulatory approval. Additional delays may result if an FDA Advisory Committee or other regulatory authority recommends non-approval or restrictions on approval. In addition, we may experience delays or rejections based upon additional government regulation from future legislation or administrative action, or changes in regulatory authority policy during the period of product development, clinical trials and the review process.
Further, in order to market any products in any particular jurisdiction, we must establish and comply with numerous and varying regulatory requirements on a country-by-country basis regarding safety and efficacy. Approval by the FDA in the United States does not ensure approval by regulatory authorities in any other country or jurisdiction. In addition, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries, and regulatory approval in one country does not guarantee regulatory approval in any other country. Approval processes vary among countries and can involve additional product testing and validation and additional administrative review periods. Seeking foreign regulatory approval could result in difficulties and costs for us and require additional preclinical studies or clinical trials, which could be costly and time consuming. We do not have any product candidates approved for sale in any jurisdiction, including in international markets, and we do not have experience in obtaining regulatory approval in international markets. If we fail to comply with regulatory requirements in international markets or to obtain and maintain required approvals, or if regulatory approvals in international markets are delayed, our target market will be reduced and our ability to realize the full market potential of any product we develop will be unrealized.
The FDA or any foreign regulatory bodies can delay, limit or deny approval of our product candidates or require us to conduct additional preclinical or clinical testing or abandon a program for many reasons, including:
· | the FDA or the applicable foreign regulatory agency’s disagreement with the design or implementation of our clinical trials; |
· | negative or ambiguous results from our clinical trials or results that may not meet the level of statistical significance required by the FDA or comparable foreign regulatory agencies for approval; |
· | serious and unexpected drug-related side effects experienced by participants in our clinical trials or by individuals using drugs similar to our product candidates; |
· | our inability to demonstrate to the satisfaction of the FDA or the applicable foreign regulatory body that our product candidates are safe and effective for the proposed indication; |
· | the FDA’s or the applicable foreign regulatory agency’s disagreement with the interpretation of data from preclinical studies or clinical trials; |
· | our inability to demonstrate the clinical and other benefits of our product candidates outweigh any safety or other perceived risks; |
· | the FDA’s or the applicable foreign regulatory agency’s requirement for additional preclinical studies or clinical trials; |
· | the FDA’s or the applicable foreign regulatory agency’s disagreement regarding the formulation, labeling or the specifications of our product candidates; |
· | the FDA’s or the applicable foreign regulatory agency’s failure to approve the manufacturing processes or facilities of third-party manufacturers with which we contract, including failure of such manufacturers to pass the required pre-approval inspections; or |
· | the potential for approval policies or regulations of the FDA or the applicable foreign regulatory agencies to significantly change in a manner rendering our clinical data insufficient for approval. |
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Even if we eventually complete clinical testing and receive approval of an NDA or foreign marketing application for our product candidates, the FDA or the applicable foreign regulatory agency may grant approval contingent on the performance of costly additional clinical trials, including Phase 4 clinical trials, or the implementation of a Risk Evaluation and Mitigation Strategy, or REMS, which may be required to ensure safe use of the drug after approval. The FDA or the applicable foreign regulatory agency also may approve a product candidate for a more limited indication or patient population than we originally requested, and the FDA or applicable foreign regulatory agency may not approve the labeling that we believe is necessary or desirable for the successful commercialization of a product candidate. Any delay in obtaining, or inability to obtain, applicable regulatory approval would delay or prevent commercialization of that product candidate and would negatively impact our business and results of operations.
If we are not able to obtain orphan drug designations or exclusivity for any of our current or future product candidates for which we seek such designation, the potential profitability of any such product candidates could be limited.
Regulatory authorities in some jurisdictions, including the United States, may designate drugs for relatively small patient populations as orphan drugs. Under the Orphan Drug Act of 1983, the FDA may designate a product as an orphan drug if the treatment is intended to treat a rare disease or condition, which is generally defined as a patient population of fewer than 200,000 individuals in the United States. Generally, if a product with an orphan drug designation subsequently receives the first marketing approval for a disease for which it receives the designation, then the product is entitled to a period of marketing exclusivity that precludes the applicable regulatory authority from approving another marketing application for the same product for the same disease for the exclusivity period except in limited situations. For purposes of small molecule drugs, the FDA defines “same drug” as a drug that contains the same active moiety and is intended for the same use as the drug in question.
We have received orphan drug designation for livoletide from the FDA and EMA for the treatment of PWS. Nevanimibe has received orphan drug designation from the FDA and the EMA for the treatment of CAH. We may also seek orphan drug designation, where applicable, for our current product candidates in additional indications or for our future product candidates. However, obtaining an orphan drug designation can be difficult and we may not be successful in doing so for any of our current or future product candidates, in any applicable indication. Even if we were to obtain orphan drug designation for a product candidate, we may not obtain orphan exclusivity and that exclusivity may not effectively protect the product candidate from the competition of different products or drugs for the same condition, which could be approved during the exclusivity period. Additionally, after an orphan drug is approved, the FDA could subsequently approve another application for the same product for the same disease if the FDA concludes that the later product is shown to be safer, more effective or makes a major contribution to patient care. Orphan drug exclusive marketing rights in the United States also may be lost if the FDA later determines that the request for designation was materially defective, the prevalence of the orphan disease is found to increase such that the qualifying criterion is no longer met or if the manufacturer is unable to assure sufficient quantity of the product to meet the needs of patients with the rare disease or condition. The failure to obtain an orphan drug designation for any product candidates we may develop and seek it for, the inability to maintain that designation for the duration of the applicable period, or the inability to obtain or maintain orphan drug exclusivity could reduce our ability to make sufficient sales of the applicable product candidates to balance our expenses incurred to develop it, which would have a negative impact on our operational results and financial condition.
If we are not able to obtain required regulatory approvals, we will not be able to commercialize livoletide or nevanimibe, and our ability to generate revenue will be harmed.
Livoletide and nevanimibe and the activities associated with their development and commercialization, including their design, research, testing, manufacture, safety, efficacy, recordkeeping, labeling, packaging, storage, approval, advertising, promotion, sale and distribution, are subject to comprehensive regulation by the FDA and other regulatory agencies in the United States and by similar regulatory authorities outside the United States. Failure to obtain marketing approval for livoletide and nevanimibe or failure to meet post-marketing requirements will prevent us from commercializing them.
We have not yet received approval from regulatory authorities to market any product candidate in any jurisdiction, and it is possible that none of livoletide, nevanimibe or any future product candidates will ever obtain the appropriate
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regulatory approvals necessary for us to commence product sales. Neither we nor any future collaborator is permitted to market any of our product candidates in the United States until we receive regulatory approval of an NDA from the FDA.
The time required to obtain approval of an NDA by the FDA is unpredictable but typically takes many years following the commencement of clinical trials and depends upon numerous factors, including the substantial discretion of the regulatory authorities. Prior to submitting an NDA to the FDA or an equivalent application to other foreign regulatory authorities for approval of livoletide for the treatment of PWS and for approval of nevanimibe for the treatment of CAH, we will need to complete its currently planned registration clinical trials for each, and additional trials that the FDA may require us to complete.
Additionally, if the results of our clinical trials are inconclusive or if there are safety concerns or serious adverse events associated with livoletide or nevanimibe, we may:
· | be delayed in obtaining marketing approval for livoletide or nevanimibe, if at all; |
· | obtain approval for indications or patient populations that are not as broad as intended or desired; |
· | obtain approval with labeling that includes significant use or distribution restrictions or safety warnings; |
· | be subject to additional post-marketing testing requirements; |
· | be required to perform additional clinical trials to support approval or be subject to additional post-marketing testing requirements; |
· | have regulatory authorities withdraw, or suspend, their approval of the drug or impose restrictions on its distribution in the form of REMS; |
· | be subject to the addition of labeling statements, such as warnings or contraindications; |
· | be sued; or |
· | experience damage to our reputation. |
Furthermore, approval policies, regulations, or the type and amount of clinical data necessary to gain approval may change during the course of a product candidate’s clinical development and may vary among jurisdictions.
We may rely on third-party CROs and consultants to assist us in filing and supporting the applications necessary to gain marketing approvals. Securing marketing approval requires the submission of extensive preclinical and clinical data and supporting information to regulatory authorities for each disease to establish the safety and efficacy of livoletide, nevanimibe and any future product candidate for that disease. Securing marketing approval also requires the submission of information about the product manufacturing process to, and inspection of manufacturing facilities by, the regulatory authorities.
Even if we obtain regulatory approval for livoletide, nevanimibe or future product candidates, we will remain subject to ongoing regulatory oversight.
Even if we obtain any regulatory approval for livoletide, nevanimibe or future product candidates, the approved product will be subject to ongoing regulatory requirements for manufacturing, labeling, packaging, storage, advertising, promotion, sampling, record-keeping and submission of safety and other post-market information. For example, we must comply with the FDA’s advertising and promotion requirements, such as those related to direct-to-consumer advertising and the prohibition on promoting products for uses or in patient populations that are not described in the product’s approved labeling. In addition, any regulatory approvals that we receive for livoletide, nevanimibe or future product candidates may also be subject to REMS limitations on the approved indicated uses for which the drug may be marketed
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or to the conditions of approval, or contain requirements for potentially costly post-marketing testing, including Phase 4 clinical trials, and surveillance to monitor the quality, safety and efficacy of the drug.
In addition, drug manufacturers and their facilities are subject to payment of user fees and continual review and periodic inspections by the FDA and other regulatory authorities for compliance with cGMP requirements and adherence to commitments made in the NDA or foreign marketing application. If we, or a regulatory authority, discover previously unknown problems with a drug, such as adverse events of unanticipated severity or frequency, or problems with the facility where the drug is manufactured or disagrees with the promotion, marketing or labeling of that drug, a regulatory authority may impose restrictions relative to that drug, the manufacturing facility or us, including requiring recall or withdrawal of the drug from the market or suspension of manufacturing.
If we fail to comply with applicable regulatory requirements following approval of livoletide, nevanimibe or future product candidates, a regulatory authority may, among other things:
· | issue a warning letter asserting that we are in violation of the law; |
· | seek an injunction or impose administrative, civil or criminal penalties or monetary fines; |
· | suspend or withdraw regulatory approval; |
· | suspend any ongoing clinical trials; |
· | refuse to approve a pending NDA or comparable foreign marketing application (or any supplements thereto) submitted by us or our strategic partners; |
· | restrict the marketing or manufacturing of the drug; |
· | seize or detain the drug or otherwise require the withdrawal of the drug from the market; |
· | refuse to permit the import or export of product candidates; or |
· | refuse to allow us to enter into supply contracts, including government contracts. |
Any government investigation of alleged violations of law could require us to expend significant time and resources in response and could generate negative publicity. The occurrence of any event or penalty described above may inhibit our ability to commercialize livoletide and nevanimibe, and harm our business, financial condition and results of operations.
In addition, the FDA’s policies, and those of equivalent foreign regulatory agencies, may change and additional government regulations may be enacted that could suspend or restrict regulatory approval of livoletide and nevanimibe. We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained and we may not achieve or sustain profitability, which would harm our business, financial condition and results of operations.
Even if one of our product candidates receives marketing approval, it may fail to achieve market acceptance by physicians, patients, third-party payors or others in the medical community necessary for commercial success.
Even if one of our product candidates receives marketing approval, it may fail to gain sufficient market acceptance by physicians, patients, third-party payors and others in the medical community. If any such product candidate does not achieve an adequate level of acceptance, we may not generate significant product revenue and may not become profitable. The degree of market acceptance of a product candidate, if approved for commercial sale, will depend on a number of factors, including but not limited to:
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· | the efficacy and potential advantages compared to alternative treatments; |
· | the success of our efforts to educate physicians, patients, third-party payors and others in the medical community on the benefits of our products; |
· | effectiveness of sales and marketing efforts; |
· | the cost of treatment in relation to alternative treatments, including any similar generic treatments; |
· | our ability to offer our drugs, once approved, for sale at competitive prices; |
· | the convenience and ease of administration compared to alternative treatments; |
· | the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; |
· | the strength of marketing and distribution support; |
· | the availability of third-party coverage and adequate reimbursement, and patients’ willingness to pay out-of-pocket in the absence third-party coverage or adequate reimbursement; |
· | the prevalence and severity of any side effects; and |
· | any restrictions on the use of our drugs, once approved, together with other medications |
If the market opportunities for our product candidates are smaller than we believe they are, our product revenues may be adversely affected and our business may suffer.
Our efforts to educate physicians, patients, third-party payors and others in the medical community on the benefits of our products, if and when approved, may require significant resources and may never be successful. Further, patient populations suffering from PWS and CAH, and other indications we may target in the future, are small and have not been established with precision. If the actual number of patients is smaller than we estimate for any disease that we are targeting, or if we cannot raise awareness of these diseases and diagnosis is not improved, our revenue and ability to achieve profitability may be adversely affected. For example, since the patient populations for PWS and CAH are small, the per-patient drug pricing must be high in order to recover our development and manufacturing costs, fund adequate patient support programs and achieve profitability. For PWS and CAH, then, we may not maintain or obtain sufficient sales volume at a price high enough to justify our product development efforts and our sales and marketing and manufacturing expenses. Because we expect sales of livoletide and nevanimibe, if approved, to generate substantially all of our product revenue for the foreseeable future, the failure of either of these product candidates to find market acceptance would harm our business.
If we are unable to establish sales, marketing and distribution capabilities, either on our own or in collaboration with third parties, we may not be successful in commercializing our product candidates, if approved.
We do not have any infrastructure for the sales, marketing or distribution of our products, and the cost of establishing and maintaining such an organization may exceed the cost-effectiveness of doing so. In order to market any product that may be approved, we must build our sales, distribution, marketing, managerial and other non-technical capabilities, or make arrangements with third parties to perform these services. There can be no assurance we will be able to do so in a cost-effective manner, on terms favorable to us, or at all.
While we may seek the aid of global or regional collaborators to provide additional resources for larger indications or to co-commercialize our product candidates in the European Union and certain other territories, we expect to build a focused sales, distribution and marketing infrastructure to market our product candidates in the United States itself, if approved. There are significant expenses and risks involved with establishing our own sales, marketing and distribution
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capabilities, including our ability to hire, retain and appropriately incentivize qualified individuals, generate sufficient sales leads, provide adequate training to sales and marketing personnel, and effectively manage a geographically dispersed sales and marketing team. Any failure or delay in the development of our internal sales, marketing and distribution capabilities could delay any product launch, which would adversely impact its commercialization.
Factors that may inhibit our efforts to commercialize our products on our own include:
· | our inability to recruit, train and retain adequate numbers of effective sales and marketing personnel; |
· | the inability of sales personnel to obtain access to educate adequate numbers of physicians as to the benefits or our drug products; |
· | the inability of reimbursement professionals to negotiate arrangements, for formulary access, reimbursement, and other acceptance by payors; |
· | restricted or closed distribution channels that make it difficult to distribute our products to segments of the patient population; |
· | the lack of complementary medicines to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines; and |
· | unforeseen costs and expenses associated with creating an independent sales and marketing organization. |
Further, we do not anticipate having the resources in the foreseeable future to allocate to the sales and marketing of our product candidates in certain markets overseas. Therefore, our future success will depend, in part, on our ability to enter into and maintain collaborative relationships for such capabilities, the collaborator’s strategic interest in a product and such collaborator’s ability to successfully market and sell the product. We intend to pursue collaborative arrangements regarding the sale and marketing of our product candidates, if approved, for certain markets overseas; however, we cannot assure you that we will be able to establish or maintain such collaborative arrangements, or if able to do so, that we will have effective sales forces. To the extent that we depend on third parties for marketing and distribution, any revenue we receive will depend upon the efforts of such third parties, and there can be no assurance that such efforts will be successful.
If we are unable to build our own sales force or negotiate a collaborative relationship for the commercialization of our product candidates, we may be forced to delay our potential commercialization or reduce the scope of our sales or marketing activities for them. If we elect to increase our expenditures to fund commercialization activities itself, we will need to obtain additional capital, which may not be available to us on acceptable terms, or at all. If we do not have sufficient funds, we will not be able to bring our product candidates to market or generate product revenue. We could enter into arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be ideal and we may be required to relinquish rights to our product candidates or otherwise agree to terms unfavorable to us, any of which may have an adverse effect on our business and results of operations.
If we are unable to establish adequate sales, marketing and distribution capabilities, either on our own or in collaboration with third parties, we will not be successful in commercializing our product candidates and may not become profitable. We will be competing with many companies that currently have extensive and well-funded sales and marketing operations. Without an internal team or the support of a third-party to perform sales and marketing functions, we may be unable to compete successfully against these more established companies.
Even if we obtain and maintain approval for our current and future product candidates from the FDA, we may nevertheless be unable to obtain approval for our product candidates outside of the United States, which would limit our market opportunities and could harm our business.
Approval of a product candidate in the United States by the FDA does not ensure approval of such product candidate by regulatory authorities in other countries or jurisdictions, and approval by one foreign regulatory authority does not
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ensure approval by regulatory authorities in other foreign countries or by the FDA. If approved, sales of livoletide, nevanimibe and any future product candidate outside of the United States will be subject to foreign regulatory requirements governing clinical trials and marketing approval. Even if the FDA grants marketing approval for a product candidate, comparable regulatory authorities of foreign countries also must approve the manufacturing and marketing of the product candidate in those countries. Approval procedures vary among jurisdictions and can involve requirements and administrative review periods different from, and more onerous than, those in the United States, including additional preclinical studies or clinical trials. In many countries outside the United States, a product candidate must be approved for reimbursement before it can be approved for sale in that country. In some cases, the price that we intend to charge for any product candidates, if approved, is also subject to approval. Obtaining approval for livoletide, nevanimibe or any future product candidate in the European Union from the European Commission following the opinion of the EMA, if we choose to submit a marketing authorization application there, would be a lengthy and expensive process. Even if a product candidate is approved, the FDA or the European Commission, as the case may be, may limit the indications for which the drug may be marketed, require extensive warnings on the drug labeling or require expensive and time-consuming additional clinical trials or reporting as conditions of approval. Obtaining foreign regulatory approvals and compliance with foreign regulatory requirements could result in significant delays, difficulties and costs for us and could delay or prevent the introduction of livoletide, nevanimibe or any future product candidate in certain countries.
Further, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries. Also, regulatory approval for livoletide, nevanimibe or any future product candidate may be withdrawn. If we fail to comply with the regulatory requirements, our target market will be reduced and our ability to realize the full market potential of livoletide, nevanimibe or any future product candidate will be negatively impacted, and our business, prospects, financial condition and results of operations could be harmed.
We are exposed to a variety of risks associated with our international operations.
Since the closing date of the Merger, we have been engaged in the process of winding up various subsidiaries of OvaScience, some or all of which are in foreign jurisdictions. We expect to incur additional costs to complete this process. Moreover, even if we successfully wind up these entities, we may be exposed to liability in these foreign jurisdictions as a result of their historical operations.
In addition, in December 2017, we acquired Alizé, a biopharmaceutical company based in Lyon, France. As of June 30, 2019, we had 30 employees located in the United States and 7 employees located in France. Our global operations expose us to numerous and sometimes conflicting legal, tax and regulatory requirements, and violations or unfavorable interpretation by the respective authorities of these regulations could harm our business. Risks associated with international operations include the following, and these risks may be more pronounced if we seek to commercialize livoletide, nevanimibe or any future product candidates outside of the United States:
· | different regulatory requirements for approval of therapies in foreign countries; |
· | reduced protection for intellectual property rights; |
· | unexpected changes in tariffs, trade barriers and regulatory requirements; |
· | economic weakness, including inflation, or political instability in particular foreign economies and markets; |
· | compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; |
· | foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country; |
· | foreign reimbursement, pricing and insurance regimes; |
· | workforce uncertainty in countries where labor unrest is more common than in the United States; |
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· | changes in diplomatic and trade relationships; |
· | anti-corruption laws, including the FCPA, and its equivalent in foreign jurisdictions, such as the UK Bribery Act; |
· | production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and |
· | business interruptions resulting from geopolitical actions, including war and terrorism or natural disasters including earthquakes, typhoons, floods and fires. |
In addition, there are complex regulatory, tax, labor, and other legal requirements imposed by both the European Union and many of the individual countries in and outside of Europe, with which we may need to comply. Many biopharmaceutical companies have found the process of marketing their own products in foreign countries to be very challenging.
Furthermore, in some countries, the pricing of prescription pharmaceuticals is subject to governmental control. In these countries, pricing negotiations with governmental authorities can take considerable time after the receipt of marketing approval for a product candidate. In addition, there can be considerable pressure by governments and other stakeholders on prices and reimbursement levels, including as part of cost containment measures. Political, economic and regulatory developments may further complicate pricing negotiations, and pricing negotiations may continue after coverage and reimbursement have been obtained. Reference pricing used by various countries and parallel distribution or arbitrage between low-priced and high-priced countries, can further reduce prices. To obtain reimbursement or pricing approval in some countries, we may be required to conduct a clinical trial that compares the cost-effectiveness of our product candidate to other available therapies, which is time-consuming and costly. If coverage and reimbursement of our product candidates are unavailable or limited in scope or amount, or if pricing is set at unsatisfactory levels, our business could be harmed.
Legal, political and economic uncertainty surrounding the planned exit of the U.K., from the European Union, or EU, may be a source of instability in international markets, create significant currency fluctuations, adversely affect our operations in the U.K. and pose additional risks to our business, revenue, financial condition, and results of operations.
On June 23, 2016, the U.K. held a referendum in which a majority of the eligible members of the electorate voted for the U.K. to leave the EU. The U.K.’s withdrawal from the EU is commonly referred to as Brexit. The lack of clarity over which EU laws and regulations will continue to be implemented in the U.K. after Brexit (including financial laws and regulations, tax and free trade agreements, intellectual property rights, data protection laws, supply chain logistics, environmental, health and safety laws and regulations, immigration laws and employment laws) may negatively impact foreign direct investment in the U.K., increase costs, depress economic activity and restrict access to capital. The uncertainty concerning the U.K.’s legal, political and economic relationship with the EU after Brexit may be a source of instability in the international markets, create significant currency fluctuations, and/or otherwise adversely affect trading agreements or similar cross-border co-operation arrangements (whether economic, tax, fiscal, legal, regulatory or otherwise) beyond the date of Brexit.
These developments, or the perception that any of them could occur, have had, and may continue to have, a significant adverse effect on global economic conditions and the stability of global financial markets, and could significantly reduce global market liquidity and limit the ability of key market participants to operate in certain financial markets. In particular, it could also lead to a period of considerable uncertainty in relation to the U.K. financial and banking markets, as well as on the regulatory process in Europe. Asset valuations, currency exchange rates and credit ratings may also be subject to increased market volatility. The long-term effects of Brexit will depend on any agreements (or lack thereof) between the U.K. and the EU and, in particular, any arrangements for the U.K. to retain access to EU markets either during a transitional period or more permanently.
Such a withdrawal from the EU is unprecedented, and it is unclear how the U.K.’s access to the European single market for goods, capital, services and labor within the EU, or single market, and the wider commercial, legal and regulatory
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environment, will impact us. We may also face new regulatory costs and challenges that could have an adverse effect on our operations. Depending on the terms of the U.K.’s withdrawal from the EU, the U.K. could lose the benefits of global trade agreements negotiated by the EU on behalf of its members, which may result in increased trade barriers that could make our doing business in the U.K. more difficult. Furthermore, there are likely to be changes to the way in which marketing approvals are granted in the U.K., which could add time and expense to the process by which our product candidates receive and maintain regulatory approval in the U.K. and across the EEA in future.
Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any product candidate that we may develop.
We face an inherent risk of product liability exposure related to the testing of our current and future product candidates, and may face an even greater risk if we commercialize any product candidate that it may develop. If we cannot successfully defend ourselves against claims that any such product candidates caused injuries, we could incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in:
· | decreased demand for any product candidate that we may develop; |
· | loss of revenue; |
· | substantial monetary awards to trial participants or patients; |
· | significant time and costs to defend the related litigation; |
· | withdrawal of clinical trial participants; |
· | the inability to commercialize any product candidate that it may develop; |
· | injury to our reputation and significant negative media attention; and |
· | increased marketing costs to attempt to overcome any injury to our reputation or negative media attention. |
In addition, we face an inherent risk of product liability exposure related to OvaScience’s prior use of fertility treatments in humans. Product liability claims involving OvaScience’s activities may be brought for significant amounts because OvaScience’s potential fertility treatments involved mothers and children. For example, it is possible that we will be subject to product liability claims that assert that OvaScience’s potential fertility treatments have caused birth defects in children or that such defects are inheritable. These claims could be made many years into the future based on effects that were not observed or observable at the time of birth. If we cannot successfully defend against claims that OvaScience’s potential fertility treatments caused injuries, we will incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in, among other things, significant costs to defend the related litigation; substantial monetary awards or payments to trial participants or patients; loss of revenue; and the diversion of management’s resources.
Although we maintain product liability insurance coverage, such insurance may not be adequate to cover all liabilities that we may incur. We anticipate that we will need to increase our insurance coverage each time we commence a clinical trial and if we successfully commercialize any product candidate. Insurance coverage is increasingly expensive. We may not be able to maintain insurance coverage at a reasonable cost or in an amount adequate to satisfy any liability that may arise.
If OvaScience failed to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
OvaScience is subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. OvaScience’s prior operations involved the use of hazardous and flammable materials, including chemicals and
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biological materials. OvaScience’s prior operations also produced hazardous waste products. OvaScience generally contracted with third parties for the disposal of these materials and wastes. In the event of contamination or injury resulting from OvaScience’s use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources. We also could incur significant costs associated with civil or criminal fines and penalties.
We do not maintain insurance for environmental liability or toxic tort claims that may be asserted against us in connection with OvaScience’s storage or disposal of biological, hazardous or radioactive materials.
Risks Related to Regulatory Compliance
Our current and future relationships with investigators, health care professionals, consultants, third-party payors and customers may be subject, directly or indirectly, to federal and state healthcare fraud and abuse laws, false claims laws, health information privacy and security laws, and other healthcare laws and regulations. If we are unable to comply, or have not fully complied, with such laws, we could face substantial penalties.
Our operations may be directly, or indirectly through our prescribers, customers and purchasers, subject to various federal and state fraud and abuse laws and regulations, including, without limitation, the federal Anti-Kickback Statute, the federal civil and criminal false claims laws and Physician Payments Sunshine Act and regulations. These laws may constrain our current and future business or financial arrangements and relationships through which we conduct our operations, including how we research, market, sell and distribute our products for which we obtain marketing approval. In addition, we may be subject to patient privacy laws by both the federal government and the states and other countries in which we conduct our business. The laws that will affect our operations include, but are not limited to:
· | the federal Anti-Kickback Statute, which prohibits, among other things, persons or entities from knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe or rebate), directly or indirectly, overtly or covertly, in cash or in kind, in return for the purchase, recommendation, leasing or furnishing of an item or service reimbursable under a federal healthcare program, such as the Medicare and Medicaid programs. This statute has been interpreted to apply to arrangements between pharmaceutical manufacturers on the one hand, and prescribers, purchasers, formulary managers, and others on the other hand. In addition, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act, or collectively PPACA, amended the intent requirement of the federal Anti-Kickback Statute, establishing that a person or entity no longer needs to have actual knowledge of this statute or specific intent to violate it in order to have committed a violation; |
· | federal civil and criminal false claims laws, including the federal civil False Claims Act, and civil monetary penalty laws which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment or approval from Medicare, Medicaid or other government payors that are false or fraudulent. PPACA provides, and recent government cases against pharmaceutical and medical device manufacturers support the view, that federal Anti-Kickback Statute violations and certain marketing practices, including off-label promotion, may implicate the federal civil False Claims Act; |
· | the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which created additional federal civil and criminal statutes that prohibit a person from knowingly and willfully executing a scheme or from making false or fraudulent statements to defraud any healthcare benefit program, regardless of the payor (e.g., public or private); |
· | HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, or HITECH, and their implementing regulations, which imposes certain requirements relating to the privacy, security and transmission of individually identifiable health information without appropriate authorization by entities subject to the rule, such as health plans, health care clearinghouses and certain health care providers, known as covered entities, and their business associates who create, use or disclose individually identifiable health information on their behalf; |
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· | federal transparency laws, including the federal Physician Payments Sunshine Act, that require certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually to CMS information related to: (i) payments or other “transfers of value” made to physicians and teaching hospitals and (ii) ownership and investment interests held by physicians and their immediate family members; |
· | state and foreign law equivalents of each of the above federal laws, such as state anti-kickback, self-referral, and false claims laws which may apply to our business practices, including but not limited to, research, distribution, sales and marketing arrangements as well as submitting claims involving healthcare items or services reimbursed by any third-party payor, including commercial insurers; state laws that require pharmaceutical manufacturers to comply with the industry’s voluntary compliance guidelines and the applicable compliance guidance promulgated by the federal government that otherwise restricts payments that may be made to healthcare providers; state laws that require pharmaceutical manufacturers to file reports with states regarding marketing information, such as the tracking and reporting of gifts, compensation and other remuneration and items of value provided to healthcare professionals and entities; state laws that require the reporting of information related to drug pricing; and state and local laws requiring the registration of pharmaceutical sales and medical representatives; and |
· | state and foreign laws that govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts. |
Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations will involve substantial costs. However, because of the breadth of these laws and the narrowness of the statutory exceptions and regulatory safe harbors available, it is possible that some of our business activities could be subject to challenge under one or more of such laws. If our operations are found to be in violation of any of the laws described above or any other government regulations that apply to us, we may be subject to penalties, including significant administrative, civil and criminal penalties, damages, fines, additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar agreement to resolve allegations of noncompliance with these laws, exclusion from participation in government health care programs, such as Medicare and Medicaid, disgorgement, contractual damages, reputational harm and the curtailment or restructuring of our operations, any of which could harm our ability to operate our business and our results of operations. Similar sanctions and penalties, as well as individual imprisonment, also can be imposed upon executive officers and employees, including criminal sanctions against executive officers under the so-called “responsible corporate officer” doctrine, even in situations where the executive officer did not intend to violate the law and was unaware of any wrongdoing.
The risk of us being found in violation of these laws is increased by the fact that many of them have not been fully interpreted by the regulatory authorities or the courts, and its provisions are open to a variety of interpretations. Any action against us for violation of these laws, even if we successfully defend against it, could cause us to incur significant legal expenses and divert our management’s attention from the operation of our business. The shifting compliance environment and the need to build and maintain a robust and expandable system to comply with multiple jurisdictions with different compliance and/or reporting requirements increases the possibility that a healthcare company such as we may run afoul of one or more of the requirements.
Coverage and adequate reimbursement may not be available for our current or future product candidates, which could make it difficult for us to sell them profitably, if approved.
Market acceptance and sales of any product candidates that we commercialize, if approved, will depend in part on the extent to which coverage and reimbursement for these drugs and related treatments will be available from third-party payors, including government health administration authorities and private health insurers. Third-party payors often rely upon Medicare coverage policy and payment limitations in setting their own coverage and reimbursement policies. However, decisions regarding the extent of coverage and amount of reimbursement to be provided for any product candidates that we develop will be made on a plan-by-plan basis. As a result, the coverage determination process is often a time-consuming and costly process that may require us to provide scientific and clinical support for the use of our products to each payor separately, with no assurance that coverage and adequate reimbursement will be applied
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consistently or obtained. One payor’s determination to provide coverage for a drug does not assure that other payors will also provide coverage, and adequate reimbursement, for the drug. Additionally, a third-party payor’s decision to provide coverage for a therapy does not imply that an adequate reimbursement rate will be approved. Each plan determines whether it will provide coverage for a therapy, what amount it will pay the manufacturer for the therapy, and on what tier of its formulary it will be placed. The position on a formulary generally determines the co-payment that a patient will need to make to obtain the therapy and can strongly influence the adoption of such therapy by patients and physicians. Patients who are prescribed treatments for their conditions and providers prescribing such services generally rely on third-party payors to reimburse all or part of the associated healthcare costs. Patients are unlikely to use our drugs unless coverage is provided and reimbursement is adequate to cover a significant portion of the cost of our drugs.
A primary trend in the U.S. healthcare industry and elsewhere is cost containment. Third-party payors have attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. We cannot be sure that coverage and reimbursement will be available for any drug that we commercialize and, if reimbursement is available, what the level of reimbursement will be. Inadequate coverage and reimbursement may impact the demand for, or the price of, any drug for which we obtain marketing approval. If coverage and reimbursement are not available, or are available only to limited levels, we may not be able to successfully commercialize livoletide, nevanimibe and any future product candidates that we develop.
Additionally, there have been a number of legislative and regulatory proposals to change the healthcare system in the United States and in some foreign jurisdictions that could affect our ability to sell any future product candidates profitably. These legislative and regulatory changes may negatively impact the coverage and available reimbursement for livoletide, nevanimibe and any future product candidates we may commercialize, following approval, if obtained.
Healthcare legislative reform measures may have a negative impact on our business and results of operations.
In the United States and some foreign jurisdictions, there have been, and continue to be, several legislative and regulatory changes and proposed changes regarding the healthcare system that could prevent or delay marketing approval of product candidates, restrict or regulate post-approval activities, and affect our ability to profitably sell any product candidates for which we obtain marketing approval.
In March 2010, PPACA was passed, which substantially changed the way healthcare is financed by both the government and private insurers, and significantly impacts the U.S. pharmaceutical industry. PPACA, among other things: (i) addresses a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected; (ii) increases the minimum Medicaid rebates owed by manufacturers under the Medicaid Drug Rebate Program and extends the rebate program to individuals enrolled in Medicaid managed care organizations; (iii) establishes annual fees and taxes on manufacturers of certain branded prescription drugs; (iv) expands the availability of lower pricing under the 340B drug pricing program by adding new entities to the program; and (v) establishes a new Medicare Part D coverage gap discount program, in which manufacturers must, as of January 1, 2019, agree to offer 70% point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D.
Since PPACA was enacted, the U.S. federal government also has announced delays in the implementation of key provisions of PPACA. Additionally, there have been judicial and Congressional challenges to certain aspects of PPACA, as well as efforts by the Trump administration to repeal or replace certain aspects of PPACA. Since January 2017, President Trump has signed two Executive Orders and other directives designed to delay the implementation of certain provisions of PPACA or otherwise circumvent some of the requirements for health insurance mandated by PPACA. Concurrently, Congress has considered legislation that would repeal or repeal and replace all or part of PPACA. While Congress has not passed comprehensive repeal legislation, two bills affecting the implementation of certain taxes under PPACA have been signed into law. The Tax Cuts and Jobs Act of 2017, or Tax Act, included a provision which repealed, effective January 1, 2019, the tax-based shared responsibility payment imposed by PPACA on certain individuals who fail to maintain qualifying health coverage for all or part of a year that is commonly referred to as the “individual mandate”. Additionally, on January 22, 2018, President Trump signed a continuing resolution on appropriations for fiscal year 2018 that delayed the implementation of certain PPACA-mandated fees, including the so-called “Cadillac” tax on certain high cost employer-sponsored insurance plans, the annual fee imposed on certain health
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insurance providers based on market share, and the medical device excise tax on non-exempt medical devices. Further, the Bipartisan Budget Act of 2018, among other things, amended the PPACA, effective January 1, 2019, to increase from 50 percent to 70 percent the point-of-sale discount that is owed by pharmaceutical manufacturers who participate in Medicare Part D and to close the coverage gap in most Medicare drug plans, commonly referred to as the “donut hole”. More recently, in December 2018 CMS published a new final rule permitting further collections and payments to and from certain PPACA qualified health plans and health insurance issuers under the PPACA risk adjustment program. On December 14, 2018, a Texas U.S. District Court Judge ruled that the PPACA is unconstitutional in its entirety because the “individual mandate” was repealed by Congress as part of the Tax Act. While the Texas U.S. District Court Judge, as well as the Trump administration and CMS, have stated that the ruling will have no immediate effect pending appeal of the decision, it is unclear how this decision, subsequent appeals, and other efforts to repeal and replace the PPACA will impact the PPACA. We continue to evaluate the potential impact of PPACA and its possible repeal or replacement on our business.
We expect that PPACA, as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and in additional downward pressure on the price that we are able to charge for any approved drug in the United States. For example, there have been several recent U.S. Congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform government program reimbursement methodologies for drugs. At the federal level, the Trump administration’s budget proposal for fiscal year 2019 contains further drug price control measures that could be enacted during the 2019 budget process or in other future legislation, including, for example, measures to permit Medicare Part D plans to negotiate the price of certain drugs under Medicare Part B, to allow some states to negotiate drug prices under Medicaid, and to eliminate cost sharing for generic drugs for low-income patients. Further, the Trump administration released a “Blueprint” to lower drug prices and reduce out of pocket costs of drugs that contains additional proposals to increase drug manufacturer competition, increase the negotiating power of certain federal healthcare programs, incentivize manufacturers to lower the list price of their products, and reduce the out of pocket costs of drug products paid by consumers. The Department of Health and Human Services, or HHS, has started soliciting feedback on some of these measures and, at the same, is immediately implementing others under its existing authority. For example, in May 2019, CMS issued a final rule to allow Medicare Advantage Plans the option of using step therapy for Part B drugs beginning on January 1, 2020. The final rule codified a CMS policy change that was effective January 1, 2019. Congress and the Trump administration have each indicated that it will continue to seek new legislative and/or administrative measures to control drug costs. At the state level, legislatures are increasingly passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, such measures are designed to encourage importation from other countries and bulk purchasing. Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability, or commercialize our drugs. We expect that additional state and federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in reduced demand for our product candidates or additional pricing pressures.
In addition, other legislative changes have been adopted since PPACA was enacted. These changes include aggregate reductions in Medicare payments to providers of 2% per fiscal year, which went into effect on April 1, 2013 and, following passage of the Bipartisan Budget Act of 2018, among other legislative amendments, will remain in effect through 2027 unless additional Congressional action is taken. In January 2013, President Obama signed into law the American Taxpayer Relief Act of 2012, which, among other things, further reduced Medicare payments to several types of providers and increased the statute of limitations period for the government to recover overpayments to providers from three to five years. These new laws may result in additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on our customers and, accordingly, our financial operations.
Additional changes that may affect our business include those governing enrollment in federal healthcare programs, reimbursement changes, rules regarding prescription drug benefits under the health insurance exchanges and fraud and abuse and enforcement. Continued implementation of PPACA and the passage of additional laws and regulations may result in the expansion of new programs such as Medicare payment for performance initiatives, and may impact existing
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government healthcare programs, such as by improving the physician quality reporting system and feedback program. For each state that does not choose to expand its Medicaid program, there likely will be fewer insured patients overall, which could impact the sales, business and financial condition of manufacturers of branded prescription drugs. Where patients receive insurance coverage under any of the new options made available through PPACA, the possibility exists that manufacturers may be required to pay Medicaid rebates on their resulting drug utilization, a decision that could impact manufacturer revenues.
Regulatory, legislative or self-regulatory/standard developments regarding privacy and data security matters could adversely affect our ability to conduct our business.
We are subject to and affected by laws, rules, regulations and industry standards related to data privacy and security, and restrictions or technological requirements regarding the collection, use, storage, security, retention or transfer of data. In the United States, the rules and regulations to which we may be subject include federal laws and regulations enforced by the Federal Trade Commission, the Department of Health & Human Services, and state privacy, data security, and breach notification laws, as well as regulator enforcement positions and expectations. Internationally, governments and agencies have adopted and could in the future adopt, modify, apply or enforce additional laws, policies, regulations, and standards covering privacy and data security that may apply to our business. New regulation or legislative actions regarding data privacy and security (together with applicable industry standards) may increase our costs of doing business. In addition to privacy and data security regulations currently in force in the jurisdictions where we operate, the European Union General Data Protection Regulation, or GDPR, went into effect in May 2018. The GDPR contains numerous requirements and changes from existing European Union, or EU, law, including more robust obligations on data processors and data controllers and heavier documentation requirements for data protection compliance programs. Specifically, the GDPR will introduce numerous privacy-related changes for companies operating in the EU, including greater control over personal data-by-data subjects (e.g., the “right to be forgotten”), increased data portability for EU consumers, data breach notification requirements, and increased fines. In particular, under the GDPR, fines of up to €20 million or up to 4% of the annual global revenue of the noncompliant company, whichever is greater, could be imposed for violations of certain of the GDPR’s requirements. The GDPR requirements apply not only to third-party transactions, but also to transfers of information between us and our subsidiaries, including employee information. However, despite our ongoing efforts to bring our practices into compliance before the effective date of the GDPR, we may not be successful either due to various factors within our control, such as limited financial or human resources, or other factors outside our control. It is also possible that local data protection authorities may have different interpretations of the GDPR, leading to potential inconsistencies amongst various EU member states. Any failure or alleged failure (including as a result of deficiencies in our policies, procedures, or measures relating to privacy, data security, marketing, or communications) by us to comply with laws, regulations, policies, legal or contractual obligations, industry standards, or regulatory guidance relating to privacy or data security, may result in governmental investigations and enforcement actions, litigation, fines and penalties, additional regulatory oversight and reporting obligations or adverse publicity. We expect that there will continue to be new proposed laws, regulations and industry standards relating to privacy and data protection in the United States, the European Union, and in other jurisdictions, and we cannot determine the impact such future laws, regulations and standards may have on our business.
Future laws, regulations, standards and other obligations or any changed interpretation of existing laws or regulations could impair our ability to operate our business and negatively impact our results of operations.
Risks Related to Our Intellectual Property
We rely on the availability of licenses for intellectual property from third parties and these licenses may not be available to us on commercially reasonable terms, or at all.
We rely upon the UM License Agreement to certain patent rights and proprietary technology from the University of Michigan that are important or necessary to the development of nevanimibe. We rely upon the Roche License Agreement to certain patent rights and proprietary technology from Roche that are important or necessary to the development of MLE-301. As of June 30, 2019, with respect to nevanimibe patent rights, we owned two issued U.S. patents, two pending U.S. patent applications, and a number of patent applications in other jurisdictions, and we jointly owned, with the University of Michigan, three issued U.S. patents, one pending U.S. patent application, and a number of patent applications in other jurisdictions. In addition, as of June 30, 2019, with respect to livoletide patent rights, we
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owned four issued U.S. patents, one pending U.S. patent application, and a number of patents and pending patent applications in other jurisdictions. Finally, as of June 30, 2019, with respect to MLE-301 patent rights, we exclusively licensed from Roche, one issued U.S. patent and a number of patents and pending patent applications in other jurisdictions. There is no guarantee that any of the foregoing patent applications will result in issued patents, or that any current patents or patent applications, if issued, will include claims that are sufficiently broad to cover our product candidates or future products, or to provide meaningful protection from our competitors in all territories in which we may wish to develop or commercialize our products in the future. We will be able to protect our proprietary rights from unauthorized use by third parties only to the extent they are covered by valid and enforceable patents or are effectively maintained as trade secrets within our organization. If third parties disclose or misappropriate our proprietary rights, it may have a material adverse effect on our business.
The licenses granted under the UM License Agreement and Roche License Agreement, respectively, are revocable under certain circumstances including if we cease to do business, fail to make the payments due thereunder, commit a material breach of the agreement that is not cured within a certain time period after receiving written notice or fail to meet certain specified development and commercial timelines. In such an event, our ability to compete in the market for a particular drug indication may be diminished. Termination of the UM License Agreement or Roche License Agreement may result in us having to negotiate a new or reinstated agreement, which may not be available to us on equally favorable terms, or at all, which may mean we are unable to develop or commercialize nevanimibe or MLE-301, as applicable. Additionally, the UM License Agreement, Roche License Agreement and other licenses we may enter into in the future may not provide exclusive rights to use such intellectual property and technology at all, in all relevant fields of use and/or in all territories in which we may wish to develop or commercialize our product candidates in the future. As a result, we may not be able to prevent competitors from developing and commercializing competitive products, including in territories included in the UM License Agreement and Roche License Agreement.
Licenses to additional third-party patents and materials that may be required for our development programs may not be available in the future or may not be available on commercially reasonable terms, or at all, which could harm our business and financial condition.
Our intellectual property licenses and agreements with third parties may be subject to disagreements over contract interpretation, which could narrow the scope of our rights to the relevant intellectual property or technology or increase our financial or other obligations to our licensors.
We currently depend, and will continue to depend, on the UM License Agreement. In addition, pursuant to an assignment agreement for certain patents and patent applications relating to livoletide, we are also required to pay royalties on commercial sales and licensing of livoletide to the assignors. Further, the assignors under this assignment agreement have a right to repurchase the assigned intellectual property at a certain price in the event we do not, upon receiving notice, use reasonable efforts to develop, introduce for sale and promote products derived from the assigned intellectual property. Such reasonable efforts involve spending an annual amount of at least CDN$100,000 in research and development related to livoletide, actively pursuing the registration, licenses and permits necessary to market livoletide and actual commercialization of livoletide, if approved. We currently depend, and will continue to depend, on the Roche License Agreement. If Roche terminates the Roche License Agreement due to a breach of any of our material obligations under the Roche License Agreement or due to our insolvency, or if we terminate the Roche License Agreement without cause, the rights and licenses granted by Roche to us under the Roche License Agreement will terminate on the effective date of the termination. In such event, if Roche provides us with timely notice, and to the extent reasonably requested by Roche, we must transfer to Roche all regulatory filings and approvals, all final pre-clinical, non-clinical and clinical study reports and clinical study protocols, trademarks, and all data, including clinical data, materials and information, in our possession and control related to MLE-301 necessary or reasonably useful for Roche to continue to develop and commercialize MLE-301. Further, if the effective date of such a termination is after the first commercial sale of the first MLE-301 product, Roche shall have a worldwide, non-exclusive, sublicensable, transferable license to research, develop, manufacture, and sell MLE-301 compounds and products. In such event, Roche would pay to us royalty fees with respect to the sale of those compounds and products. Further development and commercialization of livoletide, nevanimibe and MLE-301 may, and development of any future product candidates may, require us to enter into additional license, assignment or collaboration agreements. The agreements under which we currently hold or license intellectual property or technology from third parties are complex, and certain provisions in such agreements may be susceptible to multiple interpretations. The resolution of any contract interpretation
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disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our financial or other obligations under the relevant agreement, either of which could have a material adverse effect on our business, financial condition, results of operations and prospects.
If any of our current or future licenses or agreements or material relationships or any in-licenses upon which our current or future licenses and intellectual property are based are terminated or breached, we may:
· | lose our rights to develop and market our current and any future product candidates; |
· | lose our rights to patent protection for our current or any future product candidates; |
· | experience significant delays in the development or commercialization of our current or any future product candidates; |
· | not be able to obtain any other licenses on acceptable terms, if at all; or |
· | incur liability for damages. |
These risks apply to any agreements that we may enter into in the future for livoletide, nevanimibe, MLE-301 or for any future product candidates. If we experience any of the foregoing, it would have a material adverse effect on our business, financial condition and results of operations.
If we fail to comply with our obligations in the agreements under which we hold or license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license and intellectual property rights that are important to our business.
Further, we cannot provide any assurances that third-party patents or other intellectual property rights do not exist, which might be enforced against our current product candidates, resulting in either an injunction prohibiting our manufacture or sales, or, with respect to our sales, an obligation on our part to pay royalties and/or other forms of compensation to third parties. Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our current licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates, which could have a material adverse effect on our business, prospects, financial condition and results of operations.
If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection obtained is not sufficiently broad, we may not be able to compete effectively in our markets.
We rely upon a combination of patents, trade secret protection and confidentiality agreements to protect the intellectual property related to our product candidates. Our success depends in large part on our ability to obtain and maintain patent protection in the United States and other countries with respect to our current and future product candidates in the United States and other countries in which we plan to develop and commercialize such product candidates. We seek to protect our proprietary position by filing patent applications in the United States and abroad related to our development programs and product candidates. The patent prosecution process is expensive and time-consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner.
Pursuant to the UM License Agreement, we obtained an exclusive, worldwide license to develop, manufacture and commercialize nevanimibe. However, the UM License Agreement permits the University of Michigan, and other non-profit research institutions which are granted such rights from the University of Michigan, to manufacture and research nevanimibe for internal research, public service and internal educational purposes, all of which could result in new patentable inventions concerning the manufacture or use of nevanimibe. In addition, pursuant to an assignment agreement for certain livoletide patents and patent applications, certain individuals at the Erasmus University Medical Center and the University of Turin were granted non-exclusive rights to use the assigned intellectual property for non-commercial research with our prior written consent, all of which could result in new patentable inventions concerning
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the manufacture or use of livoletide. Pursuant to the Roche License Agreement, we obtained an exclusive, worldwide license to develop, manufacture and commercialize MLE-301. However, the Roche License Agreement permits Roche to use MLE-301 for internal research purposes, which could result in new patentable inventions concerning the manufacture or use of MLE-301.
It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection. The patent applications that we own or in-license may fail to result in issued patents with claims that cover our current and future product candidates in the United States or in other foreign countries. There is no assurance that all of the potentially relevant prior art relating to our patents and patent applications has been found, which can invalidate a patent or prevent a patent from issuing from a pending patent application. Even if patents do successfully issue and even if such patents cover our current and future product candidates, third parties may challenge their validity, enforceability or scope, which may result in such patents being narrowed, invalidated or held unenforceable. Any successful opposition to these patents or any other patents owned by or licensed to us could deprive us of rights necessary for the successful commercialization of any product candidates that we may develop. Further, if we encounter delays in regulatory approvals, the period of time during which we could market a product candidate and companion diagnostic under patent protection could be reduced.
If the patent applications we hold or have in-licensed with respect to our development programs and product candidates fail to issue, if their breadth or strength of protection is threatened, or if they fail to provide meaningful exclusivity for our current and future product candidates, it could dissuade companies from collaborating with us to develop product candidates, and threaten our ability to commercialize future drugs. Any such outcome could have a material adverse effect on our business.
The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions and has in recent years been the subject of much litigation. In addition, the laws of foreign countries may not protect our rights to the same extent as the laws of the United States, or vice versa. For example, European patent law restricts the patentability of methods of treatment of the human body more than United States law does. Further, we may not be aware of all third-party intellectual property rights potentially relating to our product candidates. Publications of discoveries in scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically published 18 months after filing, or in some cases, not at all. Therefore, we cannot know with certainty whether we were the first to make the inventions claimed in our owned or licensed patents or pending patent applications, or that we were the first to file for patent protection of such inventions. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights are highly uncertain. Our pending and future patent applications may not result in patents being issued which protect our technology or product candidates, in whole or in part, or which effectively prevent others from commercializing competitive technologies and drugs. Changes in either the patent laws or interpretation of the patent laws in the United States and other countries may diminish the value of our patents or narrow the scope of our patent protection.
Recent patent reform legislation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents. On September 16, 2011, the Leahy-Smith America Invents Act, or the Leahy-Smith Act, was signed into law. The Leahy-Smith Act includes a number of significant changes to United States patent law. These include provisions that affect the way patent applications are prosecuted and may also affect patent litigation. The United States Patent and Trademark Office, or USPTO, recently developed new regulations and procedures to govern administration of the Leahy-Smith Act, and many of the substantive changes to patent law associated with the Leahy-Smith Act, and in particular, the first to file provisions, only became effective on March 16, 2013. Accordingly, it is not clear what, if any, impact the Leahy-Smith Act will have on the operation of our business. However, the Leahy-Smith Act and its implementation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents, all of which could have a material adverse effect on our business and financial condition. Any further changes in either the patent laws or interpretation of the patent laws in the United States and other countries may diminish the value of our patents and patent applications or narrow the scope of our potential patent protection.
Moreover, we may be subject to a third-party pre-issuance submission of prior art to the USPTO or become involved in opposition, derivation, reexamination, inter partes review, post-grant review or interference proceedings challenging our patent rights or the patent rights of others. An adverse determination in any such submission, proceeding or litigation
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could reduce the scope of, or invalidate, our patent rights, allow third parties to commercialize our technology or product candidates and compete directly with us, without payment to us, or result in our inability to manufacture or commercialize product candidates without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates.
The issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our owned and licensed patents may be challenged in the courts or patent offices in the United States and abroad. Such challenges may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and product candidates, or limit the duration of the patent protection of our technology and product candidates. Moreover, patents have a limited lifespan. In the United States, the natural expiration of a patent is generally 20 years from the earliest filing date of a non-provisional patent application. Various extensions may be available; however, the life of a patent, and the protection it affords, is limited. Without patent protection for our current or future product candidates, we may be open to competition from generic versions of such drugs. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, we owned and licensed patent portfolio may not provide it with sufficient rights to exclude others from commercializing drugs similar or identical to that of us.
We jointly own patents and patent applications with third parties. Our ability to exploit or enforce these patent rights, or to prevent the third-party from granting licenses to others with respect to these patent rights, may be limited in some circumstances.
We jointly own certain patents and patent applications with third parties. In the absence of an agreement with each co-owner of jointly owned patent rights, we will be subject to default rules pertaining to joint ownership. Some countries require the consent of all joint owners to exploit, license or assign jointly owned patents, and if we are unable to obtain that consent from the joint owners, we may be unable to exploit the invention or to license or assign our rights under these patents and patent applications in those countries. For example, we secured exclusive rights from the University of Michigan for certain patents and patent applications that they jointly own with us related to nevanimibe. Additionally, in the United States, each co-owner may be required to be joined as a party to any claim or action we may wish to bring to enforce these patent rights, which may limit our ability to pursue third-party infringement claims.
We have in-licensed patents and patent applications from third parties. Our ability to exploit or enforce these patent rights, or to prevent the third-party from granting licenses to others with respect to these patent rights, may be limited in some circumstances.
We have in-licensed certain patents and patent applications from third parties. In the absence of an agreement with each patent rights owner, we will be subject to default rules pertaining to ownership. Some countries require the consent of all owners to exploit, license or assign owned patents, and if we are unable to obtain that consent from the owners, we may be unable to exploit the invention or to license or assign our rights under these patents and patent applications in those countries. For example, we secured exclusive rights from Roche for certain patents and patent applications that they own related to MLE-301. Additionally, in the United States, each owner may be required to be joined as a party to any claim or action we may wish to bring to enforce these patent rights, which may limit our ability to pursue third-party infringement claims.
Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
Periodic maintenance fees, renewal fees, annuity fees and various other government fees on patents and/or applications will be due to be paid to the USPTO and various government patent agencies outside of the United States in several stages over the lifetime of our owned and licensed patents and/or applications and any patent rights it may own or license in the future. We rely on our outside counsel or our licensing partners to pay these fees due to non-U.S. patent agencies. The USPTO and various non-U.S. government patent agencies require compliance with several procedural,
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documentary, fee payment and other similar provisions during the patent application process. We employ reputable law firms and other professionals to help us comply and we are also dependent on our licensors to take the necessary action to comply with these requirements with respect to our licensed intellectual property. In many cases, an inadvertent lapse can be cured by payment of a late fee or by other means in accordance with the applicable rules.
There are situations, however, in which non-compliance can result in abandonment or lapse of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction.
In such an event, potential competitors might be able to enter the market and this circumstance would have a material adverse effect on our business.
Patent terms may be inadequate to protect our competitive position on our product candidates for an adequate amount of time.
Given the amount of time required for the development, testing and regulatory review of new product candidates such as livoletide, nevanimibe and MLE-301, patents protecting such candidates might expire before or shortly after such candidates are commercialized. We expect to seek extensions of patent terms in the United States and, if available, in other countries where we are prosecuting patents. In the United States, the Drug Price Competition and Patent Term Restoration Act of 1984 permits extension of the term of one U.S. patent that includes at least one claim covering the composition of matter of an FDA-approved drug, an FDA-approved method of treatment using the drug. The extended patent term cannot exceed the shorter of five years beyond the non-extended expiration of the patent or 14 years from the date of the FDA approval of the drug. However, the applicable authorities, including the FDA and the USPTO in the United States, and any equivalent regulatory authority in other countries, may not agree with our assessment of whether such extensions are available, and may refuse to grant extensions to our patents, or may grant more limited extensions than we request. Further, we may not elect to extend the most beneficial patent to us or the claims underlying the patent that we choose to extend could be invalidated. If any of the foregoing occurs, our competitors may be able to take advantage of our investment in development and clinical trials by referencing its clinical and preclinical data and launch their drug earlier than might otherwise be the case.
Intellectual property rights do not necessarily address all potential threats to our business.
The degree of future protection afforded by our intellectual property rights is uncertain because intellectual property rights have limitations, and may not adequately protect our business. The following examples are illustrative:
· | we or any strategic partners might not have been the first to make the inventions covered by the issued patents or pending patent applications that we own or control; |
· | we might not have been the first to file patent applications covering certain of our inventions; |
· | others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our intellectual property rights; |
· | it is possible that our pending patent applications will not lead to issued patents; |
· | issued patents that we own or control may not provide us with any competitive advantages, or may be held invalid or unenforceable as a result of legal challenges; |
· | our competitors might conduct research and development activities in the United States and other countries that provide a safe harbor from patent infringement claims for certain research and development activities, as well |
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as in countries where we do not have patent rights and then use the information learned from such activities to develop competitive drugs for sale in our major commercial markets; |
· | we may not develop additional proprietary technologies that are patentable; and |
· | the patents of others may have an adverse effect on our business. |
We do not have broad composition of matter patent protection with respect to nevanimibe.
We own certain patents and patent applications with claims directed to a form of nevanimibe and to specific methods of using nevanimibe and it expects to have marketing exclusivity from the FDA and EMA for a period of seven and ten years, respectively, because nevanimibe has not been approved in these markets. However, we do not have composition of matter protection in the United States and elsewhere broadly covering nevanimibe. We may be limited in our ability to list our patents in the FDA’s Orange Book if the form of the compound used is materially different from what is claimed in our patents, or if the use of its product, consistent with its FDA-approved label, would not fall within the scope of our patent claims. Also, our competitors may be able to offer and sell products so long as these competitors do not infringe any other patents that we (or third parties) hold, including patents with claims directed to the forms and manufacture of nevanimibe and/or method of use patents. In general, patents covering certain forms of a compound and method of use patents are more difficult to enforce than broad composition of matter patents because, for example, of the risks that the FDA may approve different forms of subject compounds or alternative uses of the subject compounds not covered by the method of use patents, and others may engage in off-label sale or use of the subject compounds. Physicians are permitted to prescribe an approved product for uses that are not described in the product’s labeling. Although off-label prescriptions may infringe its method of use patents, the practice is common across medical specialties and such infringement is difficult to prevent or prosecute. FDA approval of uses that are not covered by our patents would limit our ability to generate revenue from the sale of nevanimibe, if approved for commercial sale. Off-label sales would limit our ability to generate revenue from the sale of nevanimibe, if approved for commercial sale.
Third parties may initiate legal proceedings, which are expensive and time consuming, alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could have a material adverse impact on the success of our business.
Our commercial success depends, in part, upon our ability, and the ability of our future collaborators, to develop, manufacture, market and sell livoletide, nevanimibe, MLE-301 and any future product candidates and use our proprietary technologies without infringing the proprietary rights and intellectual property of third parties. The biotechnology and pharmaceutical industries are characterized by extensive and complex litigation regarding patents and other intellectual property rights. We may in the future become party to, or be threatened with, adversarial proceedings or litigation regarding intellectual property rights with respect to livoletide, nevanimibe, MLE-301 and any future product candidates and technology, including interference proceedings, post grant review and inter partes review before the USPTO. Third parties may assert infringement claims against us based on existing patents or patents that may be granted in the future, regardless of their merit. There is a risk that third parties may choose to engage in litigation with us to enforce or to otherwise assert their patent rights against us. Even if we believe such claims are without merit, a court of competent jurisdiction could hold that these third-party patents are valid, enforceable and infringed, which could have a material adverse effect on our ability to commercialize livoletide, nevanimibe, MLE-301 and any future product candidates. In order to successfully challenge the validity of any such U.S. patent in federal court, we would need to overcome a presumption of validity. As this burden is a high one requiring us to present clear and convincing evidence as to the invalidity of any such U.S. patent claim, there is no assurance that a court of competent jurisdiction would invalidate the claims of any such U.S. patent. If we are found to infringe a third-party’s valid and enforceable intellectual property rights, we could be required to obtain a license from such third-party to continue developing, manufacturing and marketing our product candidate and technology. However, we may not be able to obtain any required license on commercially reasonable terms or at all. Even if we were able to obtain a license, it could be non-exclusive, thereby giving our competitors and other third parties access to the same technologies licensed to us, and it could require us to make substantial licensing and royalty payments. We could be forced, including by court order, to cease developing, manufacturing and commercializing the infringing technology or product candidate. In addition, we could be found liable for monetary damages, including treble damages and attorneys’ fees, if we are found to have willfully infringed a patent or other intellectual property right. A finding of infringement could prevent us from manufacturing and
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commercializing livoletide, nevanimibe, MLE-301 or any future product candidates or force us to cease some or all of our business operations, which would have a material adverse effect on our business. Claims that we have misappropriated the confidential information or trade secrets of third parties could have a similar material adverse effect on our business. Even if we prevail in such infringement claims, patent litigation can be expensive and time consuming, which would harm our business, financial condition and results of operations.
We may become involved in lawsuits to protect or enforce our patents, the patents of our licensors or our other intellectual property rights, which could be expensive, time consuming and unsuccessful.
Competitors may infringe or otherwise violate our patents, the patents of our licensors or our other intellectual property rights. To counter infringement or unauthorized use, we may be required to file legal claims, which can be expensive and time-consuming. In addition, in an infringement proceeding, a court may decide that a patent of ours or our licensors is not valid or is unenforceable, or may refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question. An adverse result in any litigation or defense proceedings could put one or more of ours patents at risk of being invalidated or interpreted narrowly and could put our patent applications at risk of not issuing. The initiation of a claim against a third-party may also cause the third-party to bring counter claims against us such as claims asserting that our patents are invalid or unenforceable. In patent litigation in the United States, defendant counterclaims alleging invalidity or unenforceability are commonplace. Grounds for a validity challenge could be an alleged failure to meet any of several statutory requirements, including lack of novelty, obviousness, non-enablement or lack of statutory subject matter. Grounds for an unenforceability assertion could be an allegation that someone connected with prosecution of the patent withheld relevant material information from the USPTO, or made a materially misleading statement, during prosecution. Third parties may also raise similar validity claims before the USPTO in post-grant proceedings such as ex parte reexaminations, inter partes review, or post-grant review, or oppositions or similar proceedings outside the United States, in parallel with litigation or even outside the context of litigation. The outcome following legal assertions of invalidity and unenforceability is unpredictable. We cannot be certain that there is no invalidating prior art, of which we and the patent examiner were unaware during prosecution. For the patents and patent applications that we have licensed, we may have limited or no right to participate in the defense of any licensed patents against challenge by a third-party. If a defendant were to prevail on a legal assertion of invalidity or unenforceability, we would lose at least part, and perhaps all, of any future patent protection on our current or future product candidates. Such a loss of patent protection could have material adverse effect on our business.
We may not be able to prevent, alone or with our licensors, misappropriation of our intellectual property rights, particularly in countries where the laws may not protect those rights as fully as in the United States. Our business could be harmed if in litigation the prevailing party does not offer us a license on commercially reasonable terms. Any litigation or other proceedings to enforce our intellectual property rights may fail, and even if successful, may result in substantial costs and distract our management and other employees. Even if we prevail in such infringement claims, patent litigation can be expensive and time consuming, which would harm our business, financial condition and results of operations.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. There could also be public announcements of the results of hearings, motions or other interim proceedings or developments. If securities analysts or investors perceive these results to be negative, it could have an adverse effect on the price of our common stockstock.
Changes in U.S. patent law or the patent law of other countries or jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our product candidates.
The United States has recently enacted and implemented wide-ranging patent reform legislation. The U.S. Supreme Court has ruled on several patent cases in recent years, either narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in certain situations. In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once obtained. Depending on actions by the U.S. Congress, federal courts, USPTO, and the relevant law-making bodies in other countries, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce patents that we have licensed or that we might
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obtain in the future. Similarly, changes in patent law and regulations in other countries or jurisdictions or changes in the governmental bodies that enforce them or changes in how the relevant governmental authority enforces patent laws or regulations may weaken our ability to obtain new patents or to enforce patents that we have licensed or that we may obtain in the future.
We may not be able to protect our intellectual property rights throughout the world, which could have a material adverse effect on our business.
Filing, prosecuting and defending patents covering livoletide, nevanimibe, MLE-301 and any future product candidates throughout the world would be prohibitively expensive. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop our own drugs and, further, may export otherwise infringing drugs to territories where we may obtain patent protection, but where patent enforcement is not as strong as that in the United States. These drugs may compete with our drugs in jurisdictions where we do not have any issued or licensed patents and any future patent claims or other intellectual property rights may not be effective or sufficient to prevent them from so competing.
Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.
If we rely on third parties to manufacture and commercialize livoletide, nevanimibe, MLE-301 or any future product candidates, or if we collaborate with third parties for the development of livoletide, nevanimibe or any future product candidates, we must, at times, share trade secrets with them. We may also conduct joint research and development programs that may require us to share trade secrets under the terms of our research and development partnerships or similar agreements. We seek to protect our proprietary technology in part by entering into confidentiality agreements and, if applicable, material transfer agreements, consulting agreements or other similar agreements with our advisors, employees, third-party contractors and consultants prior to beginning research or disclosing proprietary information. These agreements typically limit the rights of the third parties to use or disclose our confidential information, including our trade secrets. Despite the contractual provisions employed when working with third parties, the need to share trade secrets and other confidential information increases the risk that such trade secrets become known by our competitors, are inadvertently incorporated into the technology of others, or are disclosed or used in violation of these agreements. Given that our proprietary position is based, in part, on our know-how and trade secrets, a competitor’s discovery of our trade secrets or other unauthorized use or disclosure could have an adverse effect on our business and results of operations.
In addition, these agreements typically restrict the ability of our advisors, employees, third-party contractors and consultants to publish data potentially relating to our trade secrets. Despite our efforts to protect our trade secrets, our competitors may discover our trade secrets, either through breach of our agreements with third parties, independent development or publication of information by any of third-party collaborators. A competitor’s discovery of our trade secrets would harm our business.
We may be subject to claims that our employees, consultants or independent contractors have wrongfully used or disclosed confidential information of their former employers or other third parties.
Certain of our employees, consultants or advisors are currently, or were previously, employed at universities or other biotechnology or pharmaceutical companies, including our competitors or potential competitors. Although we try to ensure that our employees, consultants and advisors do not use the proprietary information or know-how of others in their work for us, we may be subject to claims that these individuals or we have used or disclosed intellectual property, including trade secrets or other proprietary information, of any such individual’s current or former employer. Litigation may be necessary to defend against these claims. If we fail in defending any such claims, in addition to paying monetary damages we may lose valuable intellectual property rights or personnel. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management.
In addition, while it is our approach to require our employees and contractors who may be involved in the conception or development of intellectual property to execute agreements assigning such intellectual property to us, we may be unsuccessful in executing such an agreement with each party who, in fact, conceives or develops intellectual property
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that we regard as our own. The assignment of intellectual property rights may not be self-executing or the assignment agreements may be breached, and we may be forced to bring claims against third parties, or defend claims that they may bring against us, to determine the ownership of what we regard as our intellectual property.
Risks Related to Our Dependence on Third Parties
We do not have our own manufacturing capabilities and will rely on third parties to produce clinical and commercial supplies of livoletide and nevanimibe, and any future product candidate.
We have no experience in drug formulation or manufacturing and do not own or operate, and we do not expect to own or operate, facilities for product manufacturing, storage and distribution, or testing. We will rely on a contract manufacturing organization, or CMO, to produce additional livoletide active pharmaceutical ingredient, or API, for us for clinical use. We also currently rely on CMOs to produce nevanimibe for our clinical trials. Additionally, we rely on CMOs with respect to the manufacture of drug product for our clinical trials, including for filing and packaging. Any significant delay in the supply of a product candidate, or the raw material components thereof, for an ongoing clinical trial due to the need to replenish the supply or replace a third-party manufacturer could considerably delay completion of our clinical trials, product testing and potential regulatory approval of our product candidates. If we or our manufacturer are unable to purchase these raw materials after regulatory approval has been obtained for our product candidates, the commercial launch of our product candidates would be delayed or there would be a shortage in supply, which would impair our ability to generate revenue from the sale of our product candidates.
We will need to rely on third-party manufacturers to supply us with sufficient quantities of livoletide and nevanimibe to be used, if approved, for the commercialization of each. The facilities used by our contract manufacturers to manufacture our product candidates must be approved by the FDA pursuant to inspections that will be conducted after we submit our NDA to the FDA. We do not control the manufacturing process of, and are completely dependent on, our contract manufacturing partners for compliance with cGMP requirements for manufacture of drug products. If our contract manufacturers cannot successfully manufacture material that conforms to our specifications and the strict regulatory requirements of the FDA or others, they will not be able to secure or maintain regulatory approval for their manufacturing facilities. In addition, we have no control over the ability of our contract manufacturers to maintain adequate quality control, quality assurance and qualified personnel. If the FDA or a comparable foreign regulatory authority does not approve these facilities for the manufacture of our product candidates or if it withdraws any such approval in the future, we may need to find alternative manufacturing facilities, which would significantly impact our ability to develop, obtain regulatory approval for or market our product candidates, if approved. Further, our reliance on third-party manufacturers entails risks, to which we would not be subject if we manufactured product candidates ourselves, including:
· | inability to meet our product specifications and quality requirements consistently; |
· | delay or inability to procure or expand sufficient manufacturing capacity; |
· | issues related to scale-up of manufacturing; |
· | costs and validation of new equipment and facilities required for scale-up; |
· | failure to comply with cGMP and similar foreign standards; |
· | inability to negotiate manufacturing agreements with third parties under commercially reasonable terms; |
· | termination or nonrenewal of manufacturing agreements with third parties in a manner or at a time that is costly or damaging to us; |
· | reliance on a limited number of sources, and in some cases, single sources for product components; |
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· | lack of qualified backup suppliers for those materials that are currently purchased from a sole or single source supplier; |
· | operations of our third-party manufacturers or suppliers could be disrupted by conditions unrelated to our business or operations, including the bankruptcy of the manufacturer or supplier; |
· | inability to find replacement manufacturers or suppliers, if necessary, on terms favorable to us, in a timely manner, or at all; |
· | carrier disruptions or increased costs that are beyond our control; and |
· | failure to deliver our products under specified storage conditions and in a timely manner. |
Any of these events could lead to clinical trial delays, failure to obtain regulatory approval or impact our ability to successfully commercialize our products once approved. Some of these events could be the basis for FDA or other regulatory authority action, including injunction, recall, seizure, or total or partial suspension of production.
We may in the future enter into collaborations with third parties to develop our product candidates. If these collaborations are not successful, our business could be harmed.
We may enter into collaborations with third parties in the future. We may in the future determine to collaborate with other pharmaceutical and biotechnology companies for development and potential commercialization of our product candidates. These relationships, or those like them, may require us to incur non-recurring and other charges, increase our near- and long-term expenditures, issue securities that dilute our existing stockholders or disrupt our management and business. In addition, we could face significant competition in seeking appropriate collaborators and the negotiation process is time-consuming and complex. Our ability to reach a definitive collaboration agreement will depend, among other things, upon our assessment of the collaborator’s resources and expertise, the terms and conditions of the proposed collaboration and the proposed collaborator’s evaluation of several factors. If we license rights to our product candidates, we may not be able to realize the benefit of such transactions if we are unable to successfully integrate them with our existing operations and company culture.
If any such potential future collaborations do not result in the successful development and commercialization of product candidates, or if one of our future collaborators terminates its agreement with us, we may not receive any future research funding or milestone or royalty payments under the collaboration. If we do not receive the funding we expect under these agreements, the development of our product candidates could be delayed and we may need additional resources to develop our product candidates. In addition, if one of our future collaborators terminates its agreement with us, we may find it more difficult to attract new collaborators and the perception of us in the business and financial communities could be adversely affected. All of the risks relating to product development, regulatory approval and commercialization apply to the activities of our potential future collaborators.
We may not be successful in finding strategic collaborators for continuing development of livoletide or nevanimibe, or successfully commercializing or competing in the market for certain diseases.
We may seek to develop strategic partnerships for developing and commercializing livoletide or nevanimibe, due to capital costs required to develop the product candidate, manufacturing constraints or anticipated commercialization costs. We may not be successful in our efforts to establish such a strategic partnership or other alternative arrangements for livoletide or nevanimibe because our research and development pipeline may be insufficient or third parties may not view livoletide or nevanimibe as having the requisite potential to demonstrate safety and efficacy. In addition, we may be restricted under an existing collaboration agreement from entering into a future agreement with a potential collaborator. We cannot be certain that, following a strategic transaction or license, we will achieve an economic benefit that justifies such transaction.
If we are unable to reach agreements with suitable collaborators on a timely basis, on acceptable terms or at all, we may have to curtail the development of our product candidates, reduce or delay the development programs, delay potential commercialization, reduce the scope of any sales or marketing activities or increase our expenditures and undertake
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development or commercialization activities at our own expense. If we elect to fund development or commercialization activities on our own, we may need to obtain additional expertise and additional capital, which may not be available to us on acceptable terms or at all. If we fail to enter into collaborations and do not have sufficient funds or expertise to undertake the necessary development and commercialization activities, we may not be able to further develop livoletide or nevanimibe, which could harm our business, financial condition and results of operations.
We rely on third parties to conduct, supervise and monitor our clinical trials, and if those third parties perform in an unsatisfactory manner, it may harm our business.
We currently do not have the ability to independently conduct preclinical studies and clinical trials that comply with the regulatory requirements known as good laboratory practice, or GLP, or GCP, respectively. We also do not currently have the ability to independently conduct large clinical trials. We intend to rely on CROs and clinical trial sites to ensure the proper and timely conduct of our clinical trials, and we expect to have limited influence over their actual performance.
We intend to rely upon CROs to monitor and manage data for our clinical programs, as well as the execution of future preclinical studies. We expect to control only certain aspects of our CROs’ activities. Nevertheless, we will be responsible for ensuring that each of our studies or trials is conducted in accordance with the applicable protocol, legal, regulatory and scientific standards and our reliance on the CROs does not relieve us of our regulatory responsibilities.
We and our CROs will be required to comply with GLP and GCP, which are regulations and guidelines enforced by the FDA and are also required by the Competent Authorities of the Member States of the European Economic Area and comparable foreign regulatory authorities in the form of International Conference on Harmonization guidelines for any of our product candidates that are in preclinical and clinical development, respectively. The regulatory authorities enforce GCP through periodic inspections of trial sponsors, principal investigators and clinical trial sites. Although we rely on CROs to conduct any future GLP-compliant preclinical and preclinical studies and current or planned GCP-compliant clinical trials, we remain responsible for ensuring that each of our GLP preclinical studies and clinical trials is conducted in accordance with our investigational plan and protocol and applicable laws and regulations, and our reliance on the CROs does not relieve us of our regulatory responsibilities. If we or our CROs fail to comply with GCP, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications. Accordingly, if our CROs fail to comply with these regulations or fail to recruit a sufficient number of subjects, we may be required to repeat clinical trials, which would delay the regulatory approval process.
While we will have agreements governing their activities, our CROs are and will not be our employees, and we will not control whether or not they devote sufficient time and resources to our future clinical and preclinical programs. These CROs may also have relationships with other commercial entities, including our competitors, for whom they may also be conducting clinical trials, or other drug development activities which could harm our business. We face the risk of potential unauthorized disclosure or misappropriation of our intellectual property by CROs, which may reduce our trade secret protection and allow our potential competitors to access and exploit our proprietary technology. If our CROs do not successfully carry out their contractual duties or obligations, fail to meet expected deadlines, or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to its clinical protocols or regulatory requirements or for any other reasons, our clinical trials may be extended, delayed or terminated, and we may not be able to obtain regulatory approval for, or successfully commercialize any product candidate that we develop. As a result, our financial results and the commercial prospects for any product candidate that we develop would be harmed, our costs could increase, and our ability to generate revenue could be delayed.
If our relationships with these CROs terminate, we may not be able to enter into arrangements with alternative CROs or do so on commercially reasonable terms. Switching or adding additional CROs involves substantial cost and requires management time and focus. In addition, there is a natural transition period when a new CRO commences work. As a result, delays occur, which can negatively impact our ability to raise additional capital.
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with GLP or their contractual obligations to us for any reason, the negative impact on our business and financial condition could be more profound than if we relied on a greater number of CROs.
Risks Related to Our Business Operations, Employee Matters and Managing Growth
Recent acquisitions and potential future acquisitions could prove difficult to integrate, disrupt our business, dilute stockholder value and strain our resources.
We completed our acquisition of Alizé Pharma SAS, or Alizé, through which we acquired livoletide, our PWS product candidate, in December 2017. In the future, we may acquire additional companies, technologies or product candidates that we believe could complement or expand our business. Integrating the operations of acquired businesses successfully or otherwise realizing any of the anticipated benefits of acquisitions involves a number of potential challenges. The failure to meet these integration challenges could seriously harm our financial condition and results of operations. Realizing the benefits of acquisitions depends in part on the integration of operations and personnel. These integration activities are complex and time-consuming, and we may encounter unexpected difficulties or incur unexpected costs, including with respect to:
· | diversion of management attention from ongoing business concerns to integration matters; |
· | coordinating clinical and preclinical development plans; |
· | consolidating and rationalizing information technology and accounting platforms and administrative infrastructures; |
· | complexities associated with managing the geographic separation of the combined businesses and consolidating multiple physical locations; |
· | discontinuation of operations of OvaScience and contingent liabilities we assumed in connection with the Merger; |
· | reconciling different corporate cultures; and |
· | retaining scientific and other key employees. |
Acquired businesses may have liabilities, adverse operating issues or other matters of concern arise following the acquisition that we fail to discover through due diligence prior to the acquisition. Further, our acquisition targets may not have as robust internal controls over financial reporting as would be expected of a public company. Acquisitions may also result in the recording of goodwill and other intangible assets that are subject to potential impairment in the future that could harm our financial results. We may also become subject to new regulations as a result of an acquisition, including if we acquire operations in a country in which we do not already operate. If we fail to properly evaluate acquisitions or unanticipated issues arise following the acquisition, we may incur costs in excess of what we anticipate and may not otherwise achieve the anticipated benefits of any such acquisitions.
We are highly dependent on the services of our key executives and personnel, including Julia C. Owens, Ph.D., our chief executive officer, Louis Arcudi III, our chief financial officer, and Ryan Zeidan, Ph.D., our senior vice president of development, and if we are not able to retain these members of our management team or recruit and retain additional management, clinical and scientific personnel, our business will be harmed.
We are highly dependent on Drs. Owens and Zeidan and Mr. Arcudi. The employment agreements we have with these officers do not prevent such persons from terminating their employment with us at any time. The loss of the services of any of these persons could impede the achievement of our research, development and commercialization objectives.
In addition, we are dependent on our continued ability to attract, retain and motivate highly qualified additional management, clinical and scientific personnel. If we are not able to retain our management and to attract, on acceptable
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terms, additional qualified personnel necessary for the continued development of our business, we may not be able to regain compliancesustain our operations or grow.
We may not be able to attract or retain qualified personnel in the future due to the intense competition for qualified personnel among biotechnology, pharmaceutical and other businesses. Many of the other pharmaceutical companies that we compete against for qualified personnel and consultants have greater financial and other resources, different risk profiles, are located in geographies with a larger biotechnology industry presence and a longer history in the industry than we do. They also may provide more diverse opportunities and better chances for career advancement. Some of these characteristics may be more appealing to high-quality candidates and consultants than what we have to offer. If we are unable to continue to attract, retain and motivate high-quality personnel and consultants to accomplish our business objectives, the rate and success at which we can discover and develop product candidates and our business will be limited and we may experience constraints on our development objectives.
Our future performance will also depend, in part, on our ability to successfully integrate newly hired executive officers into our management team and our ability to develop an effective working relationship among senior management. Our failure to integrate these individuals and create effective working relationships among them and other members of management could result in inefficiencies in the development and commercialization of our product candidates, harming future regulatory approvals, sales of our product candidates and our results of operations. Additionally, we do not currently maintain “key person” life insurance on the lives of our executives or any of our employees.
We will need to expand our organization, and we may experience difficulties in managing this growth, which could disrupt our operations.
As of June 30, 2019, we had 37 employees, 35 of whom were full-time and two of whom were part-time employees. As our development and commercialization plans and strategies develop, we expect to need additional managerial, operational, sales, marketing, financial, legal and other resources. Our management may need to divert a disproportionate amount of our attention away from our day-to-day activities and devote a substantial amount of time to managing these growth activities. We may not be able to effectively manage the expansion of our operations, which may result in weaknesses in our infrastructure, operational inefficiencies, loss of business opportunities, loss of employees and reduced productivity among remaining employees. Our expected growth could require significant capital expenditures and may divert financial resources from other projects, such as the development of our current and future product candidates. If our management is unable to effectively manage our growth, our expenses may increase more than expected, our ability to generate and grow revenue could be reduced, and we may not be able to implement our business strategy. Our future financial performance and our ability to commercialize our product candidates, develop a scalable infrastructure and compete effectively will depend, in part, on our ability to effectively manage any future growth.
Our employees, independent contractors, principal investigators, consultants, commercial collaborators, service providers and other vendors may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could have an adverse effect on our results of operations.
We are exposed to the risk of fraud or other misconduct by our employees, principal investigators, consultants and commercial partners. Misconduct by these parties could include intentional failures to comply with FDA regulations or the regulations applicable in other jurisdictions, provide accurate information to the FDA and other regulatory authorities, comply with healthcare fraud and abuse laws and regulations in the United States and abroad, report financial information or data accurately or disclose unauthorized activities to us. In particular, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Such misconduct also could involve the improper use of information obtained in the course of clinical trials or interactions with the minimum bidFDA or other regulatory authorities, which could result in regulatory sanctions and cause serious harm to our reputation. It is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from government investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations. If any such actions are instituted against us and we are not successful in
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defending itself or asserting our rights, those actions could have a negative impact on our business, financial condition and results of operations, including the imposition of significant fines or other sanctions.
We may be delayed in our receipt of certain tax benefits that Alizé historically received as a French technology company.
As a French technology company, Alizé historically benefited from certain tax advantages, including the French research tax credit (credit d’impot recherche), or CIR. The CIR is a French tax credit aimed at stimulating research and development, and can offset French corporate income tax due. Alizé has historically received CIR reimbursements promptly following filing for such reimbursements with applicable French taxing authorities. During the year ended December 31, 2017, claims were made totaling $1.0 million, which we received in the first quarter of 2019. During the year ended December 31, 2018, claims were made totaling $1.4 million. Following our acquisition of Alizé, the combined business may no longer qualify as a French small or medium size enterprise, and, accordingly, the combined business may be subject to a three-year waiting period for reimbursement of CIRs, which could adversely affect the combined business’s results of operations and cash flows.
Our internal computer systems, or those of our collaborators or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our product development programs.
Our internal computer systems and those of our current and any future collaborators and other contractors or consultants are vulnerable to damage from computer viruses, unauthorized access, natural disasters, terrorism, war and telecommunication and electrical failures. While we are not aware of any such material system failure, accident or security breach to date, if such an event were to occur and cause interruptions in our operations, it could result in a material disruption of our development programs and our business operations, whether due to a loss of our trade secrets or other proprietary information or other similar disruptions. For example, the loss of clinical trial data from completed or future clinical trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent that any disruption or security breach were to result in a loss of, or damage to, our data or applications, or inappropriate disclosure of confidential or proprietary information, we could incur liability, our competitive position could be harmed and the further development and commercialization of our product candidates could be delayed.
We may be exposed to significant foreign exchange risk.
We incur portions of our expenses, and may in the future derive revenue, in currencies other than the U.S. dollar, in particular, the euro. As a result, we are exposed to foreign currency exchange risk as our results of operations and cash flows are subject to fluctuations in foreign currency exchange rates. We currently do not engage in hedging transactions to protect against uncertainty in future exchange rates between particular foreign currencies and the euro. Therefore, for example, an increase in the value of the euro against the U.S. dollar could be expected to have a negative impact on our operating expenses as euro denominated expenses, if any, would be translated into U.S. dollars at an increased value. We cannot predict the impact of foreign currency fluctuations, and foreign currency fluctuations in the future may adversely affect our financial condition, results of operations and cash flows.
Risks Related to Ownership of Our Common Stock and Our Status as a Public Company
The trading price requirementof the shares of our common stock may be volatile, and purchasers of our common stock could incur substantial losses.
Our stock price may be volatile. The stock market in general and the market for biopharmaceutical companies in particular have experienced extreme volatility that has often been unrelated to the operating performance of particular companies. As a result of this volatility, investors may not be able to sell their common stock at or above the price paid for the shares. The market price for our common stock may be influenced by many factors, including:
· | the commencement, enrollment or results of our clinical trials or changes in the development status of our product candidates; |
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· | any delay in our regulatory filings for any product candidate we may develop, and any adverse development or perceived adverse development with respect to the applicable regulatory authority’s review of such filings, including without limitation the FDA’s issuance of a “refusal to file” letter or a request for additional information; |
· | adverse results from, delays in or termination of clinical trials; |
· | adverse regulatory decisions, including failure to receive regulatory approval of our product candidates; |
· | unanticipated serious safety concerns related to the use of our product candidates; |
· | changes in financial estimates by us or by any securities analysts who might cover our stock; |
· | conditions or trends in our industry; |
· | changes in the structure of healthcare payment systems; |
· | changes in the market valuations of similar companies; |
· | stock market price and volume fluctuations of comparable companies and, in particular, those that operate in the biopharmaceutical industry; |
· | publication of research reports about us or our industry or positive or negative recommendations or withdrawal of research coverage by securities analysts; |
· | announcements by us or our competitors of significant acquisitions, strategic partnerships or divestitures; |
· | announcements of investigations or regulatory scrutiny of our operations or lawsuits filed against us; |
· | investors’ general perception of our company and our business; |
· | recruitment or departure of key personnel; |
· | overall performance of the equity markets; |
· | trading volume of our common stock; |
· | disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies; |
· | significant lawsuits, including patent or stockholder litigation; |
· | general political and economic conditions; and |
· | other events or factors, many of which are beyond our control. |
In addition, in the past, stockholders have initiated class action lawsuits against pharmaceutical and biotechnology companies following periods of volatility in the market prices of these companies’ stock. Such litigation, if instituted against us, could cause us to incur substantial costs and divert management’s attention and resources from our business.
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If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about us, our business or our market, our stock price and trading volume could decline.
The trading market for our common stock will be influenced by the research and reports that equity research analysts publish about us and our business. As a newly public company, we have only limited research coverage by equity research analysts. Equity research analysts may elect not to initiate or continue to provide research coverage of our common stock, and such lack of research coverage may adversely affect the market price of our common stock. Even if we continue to have equity research analyst coverage, we will not have any control over the analysts or the content and opinions included in their reports. The price of our stock could decline if one or more equity research analysts downgrade our stock or issue other unfavorable commentary or research. If one or more equity research analysts ceases coverage of our company or fails to publish reports on us regularly, demand for our stock could decrease, which in turn could cause our stock price or trading volume to decline.
Future sales of our common stock in the public market could cause our share price to decline.
Sales of a substantial number of shares of our common stock in the public market could occur at any time, subject to the restrictions and limitations described below. If our stockholders sell, or the market perceives that our stockholders intend to sell, substantial amounts of our common stock in the public market, the market price of our common stock could decline significantly and could impair our ability to raise capital through the sale of additional equity securities. We are unable to predict the effect that sales, particularly sales by our directors, executive officers, and significant stockholders, may have on the prevailing market price of our common stock. As of June 30, 2019, we had 13,412,058 shares of common stock outstanding. All of our outstanding shares of common stock are available for sale in the public market, subject only to the restrictions of Rule 144 under the Securities Act. In addition, the shares of common stock subject to outstanding options under our equity incentive plans and the shares reserved for future issuance under our equity incentive plans will become eligible for sale in the public market in the future, subject to certain legal and contractual limitations. In addition, certain holders of our common stock have the right, subject to various conditions and limitations, to request we include their shares of our common stock in registration statements we may file relating to our securities.
Provisions in our certificate of incorporation and by-laws and under Delaware law could make an acquisition of us, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management.
Provisions in our certificate of incorporation and by-laws may discourage, delay or prevent a merger, acquisition or other change in control of us that stockholders may consider favorable, including transactions in which our common stockholders might otherwise receive a premium price for their shares. These provisions could also limit the price that investors might be willing to pay in compliancethe future for shares of our common stock, thereby depressing the market price of our common stock. In addition, because our board of directors is responsible for appointing the members of our management team, these provisions may frustrate or prevent any attempts by our stockholders to replace or remove our current management by making it more difficult for stockholders to replace members of our board of directors. Among other things, these provisions:
· | establish a classified board of directors such that not all members of the board are elected at one time; |
· | allow the authorized number of our directors to be changed only by resolution of our board of directors; |
· | limit the manner in which stockholders can remove directors from the board; |
· | establish advance notice requirements for stockholder proposals that can be acted on at stockholder meetings and for nominations to our board of directors; |
· | limit who may call stockholder meetings; |
· | prohibit actions by our stockholders by written consent; |
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· | require that stockholder actions be effected at a duly called stockholders meeting; |
· | authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a “poison pill” that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not been approved by our board of directors; and |
· | require the approval of the holders of at least 75 percent of the votes that all our stockholders would be entitled to cast to amend or repeal certain provisions of our certificate of incorporation or by-laws. |
Moreover, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the Delaware General Corporation Law, which prohibits a person who owns 15 percent or more of our outstanding voting stock from merging or combining with us for a period of three years after the date of the transaction in which the person acquired 15 percent or more of our outstanding voting stock, unless the merger or combination is approved in a manner prescribed by the statute.
Concentration of ownership of our common stock among our existing executive officers, directors and principal stockholders may prevent our other Nasdaq listing criteria.
As of June 30, 2019, our executive officers, directors and current beneficial owners of 5% or more of our common stock and their respective affiliates, in the aggregate, beneficially own approximately 52% of our outstanding common stock. As a result, these persons, acting together, can significantly influence all matters requiring stockholder approval, including the election and removal of directors, any merger, consolidation, sale of all or substantially all of our assets, or other significant corporate transactions.
Some of these persons or entities may have interests different than yours. For example, because many of these stockholders purchased their shares at prices substantially below the current market price of our common stock and have held their shares for a longer period, they may be more interested in selling our company to an acquirer than other investors, or they may want us to pursue strategies that deviate from the interests of other stockholders.
We are at risk of securities class action and similar litigation.
In the eventpast, securities class action litigation has often been brought against a company following a decline in the market price of our securities. This risk is especially relevant for us because biopharmaceutical companies have experienced significant stock price volatility in recent years. We remain the subject of various securities class action lawsuits and shareholder derivative lawsuits that were filed against OvaScience and certain of its officer and directors, as described in more detail in Item 3, Legal Proceedings. These lawsuits, as well as any similar lawsuits initiated in the future, could result in substantial cost and a diversion of management’s attention and resources, which could harm our business.
If we fail to maintain proper and effective internal controls, our ability to produce accurate financial statements on a timely basis could be impaired.
We are subject to the reporting requirements of the Securities Exchange Act of 1934, as amended, or the Exchange Act, The Sarbanes-Oxley Act, the Dodd-Frank Wall Street Reform and Consumer Protection Act, or the Sarbanes-Oxley Act and the rules and regulations of the stock market on which our common stock is delisted fromlisted. The Sarbanes-Oxley Act requires, among other things, that we maintain effective disclosure controls and procedures and internal control over financial reporting. Neither our management nor our auditors have evaluated, assessed or attested to our internal control over financial reporting as is set forth in Item 308 of Regulation S-K promulgated under the Exchange Act, and Section 404 of the Sarbanes-Oxley Act as of December 31, 2018, the end of our last fiscal year. We were unable to conduct the required assessment primarily due to the Merger occurring in the fourth quarter of 2018 and the substantial change in operational focus, management and the internal control environment following the Merger. We intend to do our first internal control assessment as of December 31, 2019.
We may identify weaknesses in our system of internal financial and accounting controls and procedures that could result in a material misstatement of our financial statements. Our internal control over financial reporting will not prevent or detect all errors and all fraud. A control system, no matter how well designed and operated, can provide only reasonable,
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not absolute, assurance that the control system’s objectives will be met. Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that misstatements due to error or fraud will not occur or that all control issues and instances of fraud will be detected.
If we are not able to comply with the requirements of Section 404 of the Sarbanes-Oxley Act in a timely manner, or if we are unable to maintain proper and effective internal controls, we may not be able to produce timely and accurate financial statements. If that were to happen, the market price of our stock could decline and we could be subject to sanctions or investigations by the stock exchange on which our common stock is listed, the Securities and Exchange Commission, or SEC, or other regulatory authorities.
We expect to incur increased costs as a result of operating as a public company, and our management is required to devote substantial time to compliance with our public company responsibilities and corporate governance practices.
As a relatively new public company, we incur significant legal, accounting and other expenses that we did not incur as a private company. The Sarbanes-Oxley Act, the Dodd-Frank Wall Street Reform and Consumer Protection Act, the listing requirements of the Nasdaq Capital Market and other applicable securities rules and regulations impose various requirements on public companies. Our management and other personnel need to devote a substantial amount of time to compliance with these requirements. Moreover, these rules and regulations increase our legal and financial compliance costs and will make some activities more time-consuming and costly. For example, we expect that these rules and regulations may make it more difficult and more expensive for us to obtain directors’ and officers’ liability insurance, compared to when we were a private company, which could make it more difficult for us to attract and retain qualified members of our board of directors. We cannot predict or estimate the amount of additional costs we will continue to incur as a public company or the timing of such costs.
The recently passed comprehensive tax reform bill could adversely affect our business and financial condition.
On December 22, 2017, President Trump signed into law the Tax Act which significantly revises the Internal Revenue Code of 1986, as amended. The newly enacted federal income tax law, among other things, contains significant changes to corporate taxation, including reduction of the corporate tax rate from a top marginal rate of 35% to a flat rate of 21%, limitation of the tax deduction for interest expense to 30% of adjusted earnings (except for certain small businesses), effective for net operating losses incurred in taxable years beginning after December 31, 2017, limitation of the deduction for net operating losses to 80% of current year taxable income and elimination of net operating loss carrybacks, one time taxation of offshore earnings at reduced rates regardless of whether they are repatriated, elimination of U.S. tax on foreign earnings (subject to certain important exceptions), immediate deductions for certain new investments instead of deductions for depreciation expense over time, and modifying or repealing many business deductions and credits. Notwithstanding the reduction in the corporate income tax rate, the overall impact of the new federal tax law is uncertain and our business and financial condition could be adversely affected. In addition, it is uncertain how various states will respond to the newly enacted federal tax law. The impact of this tax reform on holders of our common stock wouldis also uncertain and could be adverse. We urge you to consult with your legal and tax advisors with respect to this legislation and the potential tax consequences of investing in or holding our common stock.
Our effective tax rate may fluctuate, and we may incur obligations in tax jurisdictions in excess of accrued amounts.
We are subject to rulestaxation in more than one tax jurisdiction. As a result, our effective tax rate is derived from a combination of applicable tax rates in the various places that impose additional sales practice requirementswe operate. In preparing our financial statements, we estimate the amount of tax that will become payable in each of such places. Nevertheless, our effective tax rate may be different than experienced in the past due to numerous factors, including passage of the newly enacted federal income tax law, changes in the mix of our profitability from jurisdiction to jurisdiction, the results of examinations and audits of our tax filings, our inability to secure or sustain acceptable agreements with tax authorities, changes in accounting for income taxes and changes in tax laws. Any of these factors could cause us to experience an effective tax rate significantly different from previous periods or our current expectations and may result in tax obligations in excess of amounts accrued in our financial statements.
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We might not be able to utilize a significant portion of our net operating loss carryforwards.
As of December 31, 2018, we had federal and state net operating loss carryforwards of $249.6 million and $249.2 million, respectively. The federal and state net operating loss carryforwards will begin to expire, if not utilized, by 2031. These net operating loss carryforwards could expire unused and be unavailable to offset future income tax liabilities. Under the newly enacted federal income tax law, federal net operating losses incurred in 2018 and in future years may be carried forward indefinitely, but the deductibility of such federal net operating losses is limited. It is uncertain how various states will respond to the newly enacted federal tax law. In addition, under Section 382 of the Internal Revenue Code of 1986, as amended, and corresponding provisions of state law, if a corporation undergoes an “ownership change,” which is generally defined as a greater than 50% change, by value, in its equity ownership over a three-year period, the corporation’s ability to use its pre-change net operating loss carryforwards and other pre-change tax attributes to offset its post-change income or taxes may be limited. We may experience ownership changes in the future as a result of subsequent shifts in our stock ownership, some of which may be outside of our control. If an ownership change has occurred or occurs in the future and our ability to use our net operating loss carryforwards is materially limited, it would harm our future operating results by effectively increasing our future tax obligations.
We do not anticipate paying any cash dividends on broker-dealers who sell our securities. The additional burdens imposed upon broker-dealers by these requirements could discourage broker-dealers from effecting transactionscommon stock in the foreseeable future.
You should not rely on an investment in our common stock. This would significantlystock to provide dividend income. We have not declared or paid cash dividends on our common stock to date. We currently intend to retain our future earnings, if any, to fund the development and negatively affectgrowth of our business. In addition, the abilityterms of investors to trade our securities and would significantly and negatively affect the value and liquidityany existing or future debt agreements may preclude us from paying dividends. As a result, capital appreciation, if any, of our common stock. These factors could contribute to lower prices and larger spreads instock will be your sole source of gain for the bid and ask prices forforeseeable future. Investors seeking cash dividends should not purchase our common stock. Delisting would prevent us from satisfying a closing condition for
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds
Recent Sales of Unregistered Securities
We did not sell any unregistered securities during the proposed Merger, and, in such event, Millendo may electthree months ended June 30, 2019.
Issuer Purchases of Equity Securities
We did not to consummaterepurchase any securities during the proposed Merger.three months ended June 30, 2019.
Item 3. Defaults upon Senior Securities
Not applicable.
Item 4. Mine Safety Disclosures
Not applicable.
Not applicable.
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The following exhibits are incorporated by reference or filed as part of this Quarterly Report on Form 10-Q are set forth in the following Exhibit Index.
Exhibit | |||
Number |
Description | |||
3.1 | |||
3.2 | |||
10.1*± | |||
10.2*± | |||
10.3*± | |||
10.4*^ | |||
10.5 | |||
10.6 ± | |||
10.7*± | |||
10.8*± |
| ||
10.9± | |||
31.1* | |||
31.2* | |||
32.1+ | |||
101.INS | |||
XBRL Instance Document | |||
101.SCH | XBRL Taxonomy Extension Schema Document | ||
101.CAL | XBRL Taxonomy Extension Calculation Linkbase Document | ||
101.DEF | XBRL Taxonomy Extension Definition Linkbase Document | ||
101.LAB | XBRL Taxonomy Extension Label Linkbase Document | ||
101.PRE | XBRL Taxonomy Extension Presentation Linkbase Document |
*Filed herewith.
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^Portions of this exhibit (indicated by asterisks) have been excluded because such information (i) is not material and (ii) would be competitively harmful if publicly disclosed.
± Indicates management contract or compensatory plan.
+This certification is being furnished solely to accompany this Quarterly Report on Form 10-Q pursuant to 18 U.S.C. Section 1350, and is not being filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liability of that section, nor shall it be deemed incorporated by reference into any filing of the registrant under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date hereof, regardless of any general incorporation language in such filing.
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Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrantRegistrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
MILLENDO THERAPEUTICS, INC. | |||||
By: | /s/ Julia C. Owens, Ph.D. | ||||
Julia C. Owens, Ph.D. | |||||
President and Chief Executive Officer (Principal Executive Officer) | |||||
By: | /s/ Louis Arcudi III | ||||
Louis Arcudi III | |||||
Chief Financial Officer (Principal Financial and Accounting | |||||
Date: August 12, 2019
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