UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM10-Q
[Mark One]
| ☒ | QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the Quarterly Period Ended March 31, 20192020
OR
| ☐ | TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
Commission FileNo. 001-33057
CATALYST PHARMACEUTICALS, INC.
(Exact name of registrant as specified in its charter)
| Delaware | 76-0837053 | |
(State or other jurisdiction of incorporation or organization) | (IRS Employer Identification No.) | |
355 Alhambra Circle Suite 1250 Coral Gables, Florida | 33134 | |
| (Address of principal executive offices) | (Zip Code) | |
Registrant’s telephone number, including area code: (305)420-3200
Securities registered pursuant to Section 12(b) of the Act:
Title of Each Class | Ticker Symbol | Name of Exchange on Which Registered | ||
| Common Stock, par value $0.001 per share | CPRX | NASDAQ Capital Market |
Indicate by checkmark whether the 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 registrant was required to file such report(s), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of RegulationS-T during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, anon-accelerated filer, a smaller reporting company or an emerging growth company. See definitions of “accelerated filer”, “large accelerated filer”, “smaller reporting company” and “emerging growth company” in Rule12b-2 of the Exchange Act:
| Large accelerated filer | ☐ | Accelerated Filer | ☒ | |||
| Non-accelerated filer | ☐ | Smaller reporting company | ||||
| Emerging growth company | ☐ | |||||
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 pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule12b-2 of the Exchange Act). Yes ☐ No ☒
Securities registered pursuant to Section 12(b) of the Act:
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Indicate the number of shares outstanding of each of the issuer’s classes of common stock, as of the latest practicable date 102,854,257103,415,365 shares of common stock, $0.001 par value per share, were outstanding as of May 6, 2019.7, 2020.
CATALYST PHARMACEUTICALS, INC.
INDEX
INDEXPART I. FINANCIAL INFORMATION
| Item 1. | ||||||
Consolidated balance sheets at March 31, | 3 | |||||
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Item 2. | MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS | |||||
Item 3. | ||||||
Item 4. | ||||||
| PART II. OTHER INFORMATION | ||||||
Item 1. | ||||||
Item 1A. | ||||||
Item 2. | ||||||
Item 3. | ||||||
Item 4. | ||||||
Item 5. | ||||||
Item 6. | ||||||
CATALYST PHARMACEUTICALS, INC.
| March 31, | December 31, | March 31, | December 31, | |||||||||||||
| 2019 | 2018 | 2020 | 2019 | |||||||||||||
| (unaudited) | (unaudited) | |||||||||||||||
ASSETS | ||||||||||||||||
Current Assets: | ||||||||||||||||
Cash and cash equivalents | $ | 19,081,714 | $ | 16,559,400 | $ | 101,750,937 | $ | 89,511,710 | ||||||||
Short-term investments | 26,551,064 | 36,922,213 | — | 5,007,050 | ||||||||||||
Accounts receivable, net | 7,251,381 | — | 6,918,563 | 10,536,997 | ||||||||||||
Inventory | 96,587 | 56,012 | 2,211,338 | 1,956,792 | ||||||||||||
Prepaid expenses and other current assets | 1,881,266 | 1,649,781 | 6,062,715 | 4,351,074 | ||||||||||||
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Total current assets | 54,862,012 | 55,187,406 | 116,943,553 | 111,363,623 | ||||||||||||
Investments | 4,991,600 | 5,008,243 | ||||||||||||||
Operating leaseright-of-use asset | 1,074,020 | — | 730,284 | 793,252 | ||||||||||||
Property and equipment, net | 140,320 | 245,425 | 196,926 | 210,467 | ||||||||||||
Deposits | 8,888 | 8,888 | 8,888 | 8,888 | ||||||||||||
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Total assets | $ | 61,076,840 | $ | 60,449,962 | $ | 117,879,651 | $ | 112,376,230 | ||||||||
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LIABILITIES AND STOCKHOLDERS’ EQUITY | ||||||||||||||||
Current Liabilities: | ||||||||||||||||
Accounts payable | $ | 2,940,086 | $ | 2,337,367 | $ | 1,347,577 | $ | 4,117,447 | ||||||||
Accrued expenses and other liabilities | 6,086,029 | 7,173,987 | 16,287,936 | 19,981,295 | ||||||||||||
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Total current liabilities | 9,026,115 | 9,511,354 | 17,635,513 | 24,098,742 | ||||||||||||
Accrued expenses and other liabilities,non-current | — | 154,799 | ||||||||||||||
Operating lease liability, net of current portion | 875,098 | — | 568,421 | 647,532 | ||||||||||||
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Total liabilities | 9,901,213 | 9,666,153 | 18,203,934 | 24,746,274 | ||||||||||||
Commitments and contingencies | ||||||||||||||||
Stockholders’ equity: | ||||||||||||||||
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at March 31, 2019 and December 31, 2018 | — | — | ||||||||||||||
Common stock, $0.001 par value, 150,000,000 shares authorized; 102,804,257 shares and 102,739,257 shares issued and outstanding at March 31, 2019 and December 31, 2018, respectively | 102,804 | 102,739 | ||||||||||||||
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at March 31, 2020 and December 31, 2019 | — | — | ||||||||||||||
Common stock, $0.001 par value, 150,000,000 shares authorized; 103,408,699 shares and 103,397,033 shares issued and outstanding at March 31, 2020 and December 31, 2019, respectively | 103,409 | 103,397 | ||||||||||||||
Additionalpaid-in capital | 212,287,975 | 211,265,279 | 217,751,166 | 216,205,678 | ||||||||||||
Accumulated deficit | (161,208,464 | ) | (160,563,961 | ) | (118,262,609 | ) | (128,688,624 | ) | ||||||||
Accumulated other comprehensive loss | (6,688 | ) | (20,248 | ) | ||||||||||||
Accumulated other comprehensive income (loss) | 83,751 | 9,505 | ||||||||||||||
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Total stockholders’ equity | 51,175,627 | 50,783,809 | 99,675,717 | 87,629,956 | ||||||||||||
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Total liabilities and stockholders’ equity | $ | 61,076,840 | $ | 60,449,962 | $ | 117,879,651 | $ | 112,376,230 | ||||||||
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The accompanying notes are an integral part of these consolidated financial statements.
CATALYST PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSSINCOME (LOSS) (unaudited)
| For the Three Months Ended March 31, | For the Three Months Ended March 31, | |||||||||||||||
| 2019 | 2018 | 2020 | 2019 | |||||||||||||
Product revenue, net | $ | 12,448,438 | $ | — | $ | 29,136,472 | $ | 12,448,438 | ||||||||
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Operating costs and expenses: | ||||||||||||||||
Cost of sales | 1,711,788 | — | 4,150,866 | 1,711,788 | ||||||||||||
Research and development | 3,307,959 | 3,259,042 | 4,222,811 | 3,307,959 | ||||||||||||
Selling, general and administrative | 8,416,460 | 2,674,398 | 10,063,048 | 8,416,460 | ||||||||||||
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Total operating costs and expenses | 13,436,207 | 5,933,440 | 18,436,725 | 13,436,207 | ||||||||||||
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Loss from operations | (987,769 | ) | (5,933,440 | ) | ||||||||||||
Operating income (loss) | 10,699,747 | (987,769 | ) | |||||||||||||
Other income, net | 343,266 | 233,548 | 336,233 | 343,266 | ||||||||||||
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Loss before income taxes | (644,503 | ) | (5,699,892 | ) | ||||||||||||
Net income (loss) before income taxes | 11,035,980 | (644,503 | ) | |||||||||||||
Provision for income taxes | — | — | 609,965 | — | ||||||||||||
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Net loss | $ | (644,503 | ) | $ | (5,699,892 | ) | ||||||||||
Net income (loss) | $ | 10,426,015 | $ | (644,503 | ) | |||||||||||
Net income (loss) per share: | ||||||||||||||||
Basic | $ | 0.10 | $ | (0.01 | ) | |||||||||||
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Net loss per share – basic and diluted | $ | (0.01 | ) | $ | (0.06 | ) | ||||||||||
Diluted | $ | 0.10 | $ | (0.01 | ) | |||||||||||
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Weighted average shares outstanding – basic and diluted | 102,747,923 | 102,557,350 | ||||||||||||||
Weighted average shares outstanding: | ||||||||||||||||
Basic | 103,407,347 | 102,747,923 | ||||||||||||||
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Net loss | $ | (644,503 | ) | $ | (5,699,892 | ) | ||||||||||
Other comprehensive loss: | ||||||||||||||||
Diluted | 106,534,600 | 102,747,923 | ||||||||||||||
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Net income (loss) | $ | 10,426,015 | $ | (644,503 | ) | |||||||||||
Other comprehensive income (loss): | ||||||||||||||||
Unrealized gain (loss) onavailable-for-sale securities | 13,560 | (21,826 | ) | 74,246 | 13,560 | |||||||||||
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Comprehensive loss | $ | (630,943 | ) | $ | (5,721,718 | ) | ||||||||||
Comprehensive income (loss) | $ | 10,500,261 | $ | (630,943 | ) | |||||||||||
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The accompanying notes are an integral part of these consolidated financial statements.
CATALYST PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENT OF CHANGES IN STOCKHOLDERS’ EQUITY (unaudited)
For the three months ended March 31, 20192020 and 20182019
| Preferred Stock | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Gain (Loss) | Total | Common Stock | Additional | Accumulated Other | ||||||||||||||||||||||||||||||||||||||||||||
Balance at December 31, 2018 | $ | — | $ | 102,739 | $ | 211,265,279 | $ | (160,563,961 | ) | $ | (20,248 | ) | $ | 50,783,809 | ||||||||||||||||||||||||||||||||||||||
| Preferred Stock | Shares | Amount | Paid-in Capital | Accumulated Deficit | Comprehensive Gain (Loss) | Total | ||||||||||||||||||||||||||||||||||||||||||||||
Balance at December 31, 2019 | $ | — | 103,397,033 | $ | 103,397 | $ | 216,205,678 | $ | (128,688,624 | ) | $ | 9,505 | $ | 87,629,956 | ||||||||||||||||||||||||||||||||||||||
Issuance of stock options for services | — | — | 933,411 | — | — | 933,411 | — | — | — | 1,383,672 | — | — | 1,383,672 | |||||||||||||||||||||||||||||||||||||||
Exercise of stock options for common stock | — | 65 | 89,285 | — | — | 89,350 | — | 11,666 | 12 | 26,137 | — | — | 26,149 | |||||||||||||||||||||||||||||||||||||||
Amortization of restricted stock for services | — | — | — | 135,679 | — | — | 135,679 | |||||||||||||||||||||||||||||||||||||||||||||
Other comprehensive gain (loss) | — | — | — | — | 13,560 | 13,560 | — | — | — | — | — | 74,246 | 74,246 | |||||||||||||||||||||||||||||||||||||||
Net loss | — | — | — | (644,503 | ) | — | (644,503 | ) | ||||||||||||||||||||||||||||||||||||||||||||
Net income (loss) | — | — | — | — | 10,426,015 | — | 10,426,015 | |||||||||||||||||||||||||||||||||||||||||||||
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Balance at March 31, 2019 | $ | — | $ | 102,804 | $ | 212,287,975 | $ | (161,208,464 | ) | $ | (6,688 | ) | $ | 51,175,627 | ||||||||||||||||||||||||||||||||||||||
Balance at March 31, 2020 | $ | — | 103,408,699 | $ | 103,409 | $ | 217,751,166 | $ | (118,262,609 | ) | $ | 83,751 | $ | 99,675,717 | ||||||||||||||||||||||||||||||||||||||
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| Preferred Stock | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Gain (Loss) | Total | Common Stock | Additional | Accumulated Other | ||||||||||||||||||||||||||||||||||||||||||||
Balance at December 31, 2017 | $ | — | $ | 102,549 | $ | 207,421,710 | $ | (126,560,447 | ) | $ | — | $ | 80,963,812 | |||||||||||||||||||||||||||||||||||||||
| Preferred Stock | Shares | Amount | Paid-in Capital | Accumulated Deficit | Comprehensive Gain (Loss) | Total | ||||||||||||||||||||||||||||||||||||||||||||||
Balance at December 31, 2018 | $ | — | 102,739,257 | $ | 102,739 | $ | 211,265,279 | $ | (160,563,961 | ) | $ | (20,248 | ) | $ | 50,783,809 | |||||||||||||||||||||||||||||||||||||
Issuance of stock options for services | — | — | 971,340 | — | — | 971,340 | — | — | — | 933,411 | — | — | 933,411 | |||||||||||||||||||||||||||||||||||||||
Exercise of stock options for common stock | — | 37 | 32,995 | — | — | 33,032 | — | 65,000 | 65 | 89,285 | — | — | 89,350 | |||||||||||||||||||||||||||||||||||||||
Other comprehensive gain (loss) | — | — | — | — | (21,826 | ) | (21,826 | ) | — | — | — | — | — | 13,560 | 13,560 | |||||||||||||||||||||||||||||||||||||
Net loss | — | — | — | (5,699,892 | ) | — | (5,699,892 | ) | ||||||||||||||||||||||||||||||||||||||||||||
Net income (loss) | — | — | — | — | (644,503 | ) | — | (644,503 | ) | |||||||||||||||||||||||||||||||||||||||||||
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Balance at March 31, 2018 | $ | — | $ | 102,586 | $ | 208,426,045 | $ | (132,260,339 | ) | $ | (21,826 | ) | $ | 76,246,466 | ||||||||||||||||||||||||||||||||||||||
Balance at March 31, 2019 | $ | — | 102,804,257 | $ | 102,804 | $ | $212,287,975 | $ | (161,208,464 | ) | $ | (6,688 | ) | $ | 51,175,627 | |||||||||||||||||||||||||||||||||||||
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The accompanying notes are an integral part of these consolidated financial statements.
CATALYST PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited)
| For the Three Months Ended March 31, | For the Three Months Ended March 31, | |||||||||||||||
| 2019 | 2018 | 2020 | 2019 | |||||||||||||
Operating Activities: | ||||||||||||||||
Net loss | $ | (644,503 | ) | $ | (5,699,892 | ) | ||||||||||
Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||||||||||
Net income (loss) | $ | 10,426,015 | $ | (644,503 | ) | |||||||||||
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: | ||||||||||||||||
Depreciation | 13,748 | 8,015 | 13,541 | 13,748 | ||||||||||||
Non-cash change in right-of-use asset | 59,621 | — | ||||||||||||||
Amortization ofright-of-use asset | 62,968 | 59,621 | ||||||||||||||
Stock-based compensation | 933,411 | 971,340 | 1,519,351 | 933,411 | ||||||||||||
Change in accrued interest and accretion of discount on investments | 81,296 | (53,674 | ) | |||||||||||||
(Increase) decrease in: | ||||||||||||||||
Accounts receivable, net | (7,251,381 | ) | — | 3,618,434 | (7,251,381 | ) | ||||||||||
Inventory | (40,575 | ) | — | (254,546 | ) | (40,575 | ) | |||||||||
Prepaid expenses and other current assets and deposits | (231,485 | ) | 180,916 | (1,711,641 | ) | (231,485 | ) | |||||||||
Increase (decrease) in: | ||||||||||||||||
Accounts payable | 602,719 | (1,140,552 | ) | (2,769,870 | ) | 602,719 | ||||||||||
Accrued expenses and other liabilities | (1,337,155 | ) | (401,277 | ) | (3,699,518 | ) | (1,337,155 | ) | ||||||||
Operating lease liability | (67,155 | ) | — | (72,952 | ) | (67,155 | ) | |||||||||
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Net cash used in operating activities | (7,962,755 | ) | (6,081,450 | ) | ||||||||||||
Net cash provided by (used in) operating activities | 7,213,078 | (8,016,429 | ) | |||||||||||||
Investing Activities: | ||||||||||||||||
Purchases of property and equipment | (5,633 | ) | — | — | (5,633 | ) | ||||||||||
Purchases of investments | (9,944,974 | ) | (31,847,693 | ) | — | (9,944,974 | ) | |||||||||
Proceeds from sales/maturities of investments | 20,346,326 | — | ||||||||||||||
Proceeds from maturities and sales of investments | 5,000,000 | 20,400,000 | ||||||||||||||
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Net cash provided by (used in) investing activities | 10,395,719 | (31,847,693 | ) | 5,000,000 | 10,449,393 | |||||||||||
Financing Activities: | ||||||||||||||||
Proceeds from exercise of stock options | 89,350 | 33,032 | 26,149 | 89,350 | ||||||||||||
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Net cash provided by (used in) financing activities | 89,350 | 33,032 | 26,149 | 89,350 | ||||||||||||
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Net increase (decrease) in cash and cash equivalents | 2,522,314 | (37,896,111 | ) | 12,239,227 | 2,522,314 | |||||||||||
Cash and cash equivalents - beginning of period | 16,559,400 | 57,496,702 | ||||||||||||||
Cash and cash equivalents—beginning of period | 89,511,710 | 16,559,400 | ||||||||||||||
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Cash and cash equivalents - end of period | $ | 19,081,714 | $ | 19,600,591 | ||||||||||||
Cash and cash equivalents—end of period | $ | 101,750,937 | $ | 19,081,714 | ||||||||||||
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Non-cash investing and financing activities: | ||||||||||||||||
Unrealized gain (loss) onavailable-for-sale securities | $ | 13,560 | $ | (21,826 | ) | $ | 74,246 | $ | 13,560 | |||||||
The accompanying notes are an integral part of these consolidated financial statements.
CATALYST PHARMACEUTICALS, INC.
NOTES TO UNAUDITED CONSOLIDATED FINANCIAL STATEMENTS
| 1. | Organization and Description of Business. |
Catalyst Pharmaceuticals, Inc. and subsidiary (collectively, the Company)“Company”) is a biopharmaceutical company focused on developing and commercializing innovatinginnovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), Anti-MuSK antibody positive myasthenia gravis(MuSK-MG), Congenital Myasthenic Syndromes (CMS), and Spinal Muscular Atrophy (SMA) Type 3.
On November 28, 2018, the U.S. Food and Drug Administration, or FDA, granted approval of Firdapse®Firdapse® for the treatment of adults with LEMS.LEMS (ages 17 and above). On January 15, 2019, the Company launched its first product, Firdapse®Firdapse®, in the United States for the treatment of adults with LEMS.
Since inception, the Company has devoted substantially all of its efforts to business planning, research and development, recruiting management and technical staff, acquiring operating assets, raising capital, and selling its product. The Company has incurred operating losses in each period from inception, through Marchand started reporting operating income during the year ended December 31, 2019. The Company has been able to fund its cash needs to date through several public and private offerings of its securities and from revenues from its product sales. See Note 12.11 (Stockholders’ Equity).
Capital Resources
While there can be no assurance, based on currently available information, the Company estimates that it currently has sufficient resources to support its operations for at least the next 12 months from the issuance date of this Form10-Q.
The Company may raise required funds in the future through public or private equity offerings, debt financings, corporate collaborations, governmental research grants or other means. The Company may also seek to raise new capital to fund additional product development efforts, even if it has sufficient funds for its planned operations. Any sale by the Company of additional equity or convertible debt securities could result in dilution to the Company’s current stockholders. There can be no assurance that any required additional funding will be available to the Company at all or available on terms acceptable to the Company. Further, to the extent that the Company raises additional funds through collaborative arrangements, it may be necessary to relinquish some rights to the Company’s drug candidates or grant sublicenses on terms that are not favorable to the Company. If the Company is not able to secure additional funding when needed, the Company may have to delay, reduce the scope of, or eliminate one or more research and development programs, which could have an adverse effect on the Company’s business.
Risks and Uncertainties
There are many uncertainties regarding the novel coronavirus(COVID-19) pandemic, and the Company is closely monitoring the impact of the pandemic on all aspects of its business, including how the pandemic is impacting its patients, employees, suppliers, vendors, business partners, clinical trials and distribution channels. The Company is unable to predict the impact thatCOVID-19 will have on its financial position and operating results in future periods due to numerous uncertainties. The Company will continue to assess the evolving impact of theCOVID-19 pandemic and make adjustments to its operations as necessary.
| 2. | Basis of Presentation and Significant Accounting Policies. |
| a. | INTERIM FINANCIAL STATEMENTS. The accompanying unaudited interim consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (U.S. GAAP), and pursuant to the rules and regulations of the Securities and Exchange Commission (SEC) for reporting of interim financial information. Pursuant to such rules and regulations, certain information and note disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been omitted. The consolidated balance sheet as of December 31, |
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
In the opinion of management, the accompanying unaudited interim consolidated financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these consolidated statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 20182019 included in the 20182019 Annual Report on Form10-K filed by the Company with the SEC. The results of operations for the three months ended March 31, 20192020 are not necessarily indicative of the results to be expected for any future period or for the full 20192020 fiscal year.
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| b. | PRINCIPLES OF CONSOLIDATION. The consolidated financial statements include the Company’s accounts and those of its wholly-owned subsidiary, Catalyst Pharmaceuticals Ireland, Ltd. (“Catalyst Ireland”). All intercompany accounts and transactions have been eliminated in consolidation. Catalyst Ireland was organized in 2017. |
| c. | USE OF ESTIMATES.The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. |
| d. | CASH AND CASH EQUIVALENTS. The Company considers all highly liquid instruments purchased with an original maturity of three months or less to be cash equivalents. Cash equivalents consist mainly of money market |
| e. | INVESTMENTS. The Company invests in high credit-quality funds in order to obtain higher yields on its cash available for investments. At March 31, |
Short-Term Bond Fund
The Company previously owned a short-term bond fund isthat was classified inas trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognizes realized gains and losses and unrealized gains and losses to earnings. At March 31, 20192020 and December 31, 2018, the only investment2019, there were no investments classified as trading securities, wasas the Company sold its interest in the short-term bond fund.fund in 2019. There were no sales of trading securities for the three months ended March 31, 2020. Realized losses on trading securities during the three months ended March 31, 2019 were $4,980. There were no sales on trading securities for the three months ended March 31, 2018. Unrealized gain (loss) on trading securities was $52,741$0 and ($58,861),$52,741, respectively, for the three months ended March 31, 20192020 and March 31, 2018,2019, and is included in other income, net in the accompanying consolidated statements of operations.
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
U.S. Treasuries
U.S. Treasuries are classified asavailable-for-sale securities. The Company classifiesavailable-for-sale securities with stated maturities of greater than three months and less than one year from the date of purchase as short-term investments.Available-for-sale securities with stated maturities greater than one year are classified asnon-current investments in the accompanyingits consolidated balance sheets. The Company recordsavailable-for-sale securities at fair value with unrealized gains and losses reported in accumulated other comprehensive income (loss) in stockholders’ equity. Realized gains and losses are included in other income, net and are derived using the specific identification method for determining the cost of securities sold. Interest income is recognized when earned and is included in other income, net in the consolidated statements of operations.operations and comprehensive income (loss). The Company recognizes a charge when the declines in the fair value below the amortized cost basis of itsavailable-for-sale securities are judged to be other-than-temporary. The Company considers various factors in determining whether to recognize an other-than-temporary charge, including whether the Company intends to sell the security or whether it is more likely than not that the Company would be required to sell the security before recovery of the amortized cost basis. The Company has not recorded any other-than-temporary impairment charges on itsavailable-for-sale securities. See Note 3.
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| f. | ACCOUNTS RECEIVABLE, NET. Accounts receivable |
| g. | INVENTORY. Inventories are stated at the lower of cost or net realizable value with cost determined under thefirst-in-first-out (FIFO) cost method. Inventories consist of raw materials and supplies, work in process and finished goods. Costs to be capitalized as inventories primarily include third party manufacturing costs |
Products that have been approved by the FDA or other regulatory authorities, such as Firdapse®Firdapse®, are also used in clinical programs to assess the safety and efficacy of the products for usage in treating diseases that have not been approved by the FDA or other regulatory authorities. The form of Firdapse®Firdapse® utilized for both commercial and clinical programs is identical and, as a result, the inventory has an “alternative future use” as defined in authoritative guidance. Raw materials and purchased drug product associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes and, therefore, does not have an “alternative future use”.
The Company evaluates for potential excess inventory by analyzing current and future product demand relative to the remaining product shelf life. The Company builds demand forecasts by considering factors such as, but not limited to, overall market potential, market share, market acceptance, and patient usage.
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
| h. | PREPAID EXPENSES AND OTHER CURRENT ASSETS. Prepaid expenses and other current assets consist primarily of prepaid research fees, prepaid insurance, prepaid commercialization expenses, prepaid |
| i. | FAIR VALUE OF FINANCIAL INSTRUMENTS.The Company’s financial instruments consist of cash and cash equivalents, |
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| j. | FAIR VALUE MEASUREMENTS.Current Financial Accounting Standards Board (FASB) fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity’s own assumptions that it believes market participants would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). |
Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability.
| Fair Value Measurements at Reporting Date Using | ||||||||||||||||
| Balances as of March 31, 2019 | Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) | Significant Other Observable Inputs (Level 2) | Significant Unobservable Inputs (Level 3) | |||||||||||||
Cash and cash equivalents: | ||||||||||||||||
Money market funds | $ | 15,137,632 | $ | 15,137,632 | $ | — | $ | — | ||||||||
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Short-term investments: | ||||||||||||||||
Short-term bond fund | $ | 16,571,264 | $ | 16,571,264 | $ | — | $ | — | ||||||||
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U.S. Treasuries | $ | 9,979,800 | $ | — | $ | 9,979,800 | $ | — | ||||||||
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Investments: | ||||||||||||||||
U.S. Treasuries | $ | 4,991,600 | $ | — | $ | 4,991,600 | $ | — | ||||||||
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| Balances as of December 31, 2018 | Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) | Significant Other Observable Inputs (Level 2) | Significant Unobservable Inputs (Level 3) | |||||||||||||
Cash and cash equivalents: | ||||||||||||||||
Money market funds | $ | 14,462,087 | $ | 14,462,087 | $ | — | $ | — | ||||||||
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Short-term investments: | ||||||||||||||||
Short-term bond fund | $ | 26,541,349 | $ | 26,541,349 | $ | — | $ | — | ||||||||
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U.S. Treasuries | $ | 10,380,864 | $ | — | $ | 10,380,864 | $ | — | ||||||||
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Investments: | ||||||||||||||||
U.S. Treasuries | $ | 5,008,243 | $ | — | $ | 5,008,243 | $ | — | ||||||||
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| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
| Fair Value Measurements at Reporting Date Using | ||||||||||||||||
| Balances as of March 31, 2020 | Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) | Significant Other Observable Inputs (Level 2) | Significant Unobservable Inputs (Level 3) | |||||||||||||
Cash and cash equivalents: | ||||||||||||||||
Money market funds | $ | 23,610,888 | $ | 23,610,888 | $ | — | $ | — | ||||||||
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U.S. Treasuries | $ | 69,997,200 | $ | — | $ | 69,997,200 | $ | — | ||||||||
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| Balances as of December 31, 2019 | Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) | Significant Other Observable Inputs (Level 2) | Significant Unobservable Inputs (Level 3) | |||||||||||||
Cash and cash equivalents: | ||||||||||||||||
Money market funds | $ | 23,963,617 | $ | 23,963,617 | $ | — | $ | — | ||||||||
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U.S. Treasuries | $ | 59,932,200 | $ | — | $ | 59,932,200 | $ | — | ||||||||
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Short-term investments: | ||||||||||||||||
U.S. Treasuries | $ | 5,007,050 | $ | — | $ | 5,007,050 | $ | — | ||||||||
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| k. | OPERATING LEASES. |
Operating lease ROU assets and operating lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term at commencement date. As the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of future payments. The operating lease ROU asset also includes any lease payments made and excludes lease incentives and initial direct costs incurred. The Company’s lease terms do not include options to extend or terminate the lease as it is not reasonably certain that it will exercise these options. Lease expense for minimum lease payments is recognized on a straight-line basis over the lease term. The Company has lease agreements with lease andnon-lease components, which are generally accounted for separately. Refer to Note 2.r. for discussion of adoption method.
| l. | REVENUE RECOGNITION. |
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to arrangements that meet the definition of a contract under Topic 606, including when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. For a complete discussion of accounting for product revenue, seeProduct Sales,Revenue, Net below.
The Company also generatesmay generate revenues from payments received under a collaborative agreement. Collaborative agreement payments may include nonrefundable fees at the inception of the agreements, milestone and event-based payments for specific achievements designated in the collaborative agreements, and/or royalties on sales of products resulting from a collaborative arrangement. For a complete discussion of accounting for collaborative arrangements, seeRevenues from Collaborative Arrangement below.
|
Product Sales,Revenue, Net:The Company sells Firdapse®Firdapse® to athe Customer (its exclusive distributor) who subsequently resells Firdapse®Firdapse® to both a small group of exclusive SPs who have exclusive contracts with the Company to distribute the Company’s products to patients and potentially to medical centers or hospitals on an emergency basis. In addition to the distribution agreementsagreement with its Customer, the Company enters into arrangements with health care providers and payors that provide for government mandatedgovernment-mandated and/or privately negotiated rebates, chargebacks, and discounts with respect to the purchase of the Company’s products.
The Company recognizes revenue on product sales when the Customer obtains control of the Company’s product, which occurs at a point in time (upon delivery). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 1015 and 6030 days.
Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods, and are recorded in cost of sales.
If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred, if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the three months ended March 31, 2019.2020.
As ofDuring the three months ended March 31, 2020 and 2019, all of the Company’s sales arewere to its distributor.Customer.
Reserves for Variable Consideration:Revenue from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established. Components of variable consideration include trade discounts and allowances, product returns, provider chargebacks and discounts, government rebates, and other incentives, such as voluntary patient assistance, and other allowances that are offered within contracts between the Company and its Customer, payors, and other indirect customers relating to the Company’s sale of its products. These reserves, as detailed below, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable (if the amount is payable to the Customer) or a current liability (if the amount is payable to a party other than a Customer)customer).
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the respective underlying contracts.
The amount of variable consideration which is included in the transaction price may be constrained, and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplated application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of March 31, 20192020 and, therefore, the transaction price was not reduced further during the three months ended March 31, 2019.2020. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known.
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Trade Discounts and Allowances:The Company provides its Customer with a discount that is explicitly stated in its contract and is recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, the Company receives sales order management, data and distribution services from the Customer. To the extent the services received are distinct from the sale of Firdapse®Firdapse® to the Customer, these payments are classified in selling, general and administrative expenses in the Company’s consolidated statement of operations and comprehensive loss.income (loss). However, if the Company has determined such services received to date are not distinct from the Company’s sale of products to the Customer, these payments have been recorded as a reduction of revenue within the consolidated statement of operations and comprehensive lossincome (loss) through March 31, 2019,2020, as well as a reduction to accounts receivables,receivable, net on the consolidated balance sheets.
FundedCo-pay Assistance Program: The Company contracts with a third-party to manage theco-pay assistance program intended to provide financial assistance to qualified insuredcommercially-insured patients. The calculation of the accrual forco-pay assistance is based on an estimate of claims and the cost per claim that we expectthe Company expects to receive associated with Firdapse®Firdapse® that has been recognized as revenue, but remains in the distribution channel at the end of each reporting period. These payments are considered payable to the customerCustomer and the related reserve is recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenseexpenses and other current liabilities in the consolidated balance sheets.
Product Returns:Consistent with industry practice, the Company offers the SPs and its distributor limited product return rights for damaged and expiring product, provided it is within a specified period around the product expiration date as set forth in the applicable individual distribution agreement. The Company estimates the amount of its product sales that may be returned by its Customer and records this estimate as a reduction of revenue in the period the related product revenue is recognized, as well as reductions to trade receivables, net on the condensed consolidated balance sheets.recognized. The Company currently estimates product return liabilities using available industry data and its own sales information, including its visibility into the inventory remaining in the distribution channel. The Company has an insignificant amount of returns to date and believes that returns of its products will continue to be minimal.
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
Provider Chargebacks and Discounts:Chargebacks for fees and discounts to providers represent the estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to the Customer who directly purchases the product from the Company. The Customer charges the Company for the difference between what they pay for the product and the ultimate selling price to the qualified healthcare providers. These reserves are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue, net and accounts receivables,receivable, net. Chargeback amounts are generally determined at the time of resale to the qualified healthcare provider by the Customer, and the Company generally issues credits for such amounts within a few weeks of the Customer’s notification to the Company of the resale. Reserves for chargebacks consist principally of chargebacks that the Customer has claimed, but for which the Company has not yet issued a credit.
Government Rebates:The Company is subject to discount obligations under state Medicaid programs and Medicare. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenses and other current liabilities on the consolidated balance sheets. For Medicare, the Company also estimates the number of patients in the prescription drug coverage gap for whom the Company will owe an additional liability under the Medicare Part D program. The Company’s liability for these rebates consists of invoices received for claims from prior quarters that have not been paid or for which an invoice has not yet been received, estimates of claims for the current quarter, and estimated future claims that will be made for product that has been recognized as revenue, but which remains in the distribution channel inventories at the end of each reporting period.
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Bridge and Patient Assistance Programs: The Company provides free Firdapse®Firdapse® to uninsured patients who satisfypre-established criteria for either the Bridge Program or the Patient Assistance Program. Patients who meet the Bridge Program eligibility criteria and are transitioning from investigational product while they are waiting for a coverage determination, or later, for patients whose access is threatened by the complications arising from a change of insurer may receive a temporary supply of free Firdapse®Firdapse® while the Company is determining the patient’s third-party insurance, prescription drug benefit or other third-party coverage for Firdapse®Firdapse®. The Patient Assistance Program provides free Firdapse®Firdapse® for longer periods of time for those who are uninsured or functionally uninsured with respect to Firdapse®Firdapse® because they are unable to obtain coverage from their payer despite having health insurance.insurance, to the extent allowed by applicable law. The Company does not recognize any revenue related to these free products and the associated costs are classified in selling, general and administrative expenses in the Company’s consolidated statements of operations and comprehensive loss.income (loss).
Revenues from Collaborative Arrangement:The Company has entered into a collaboration agreement for the further development and commercialization of generic Sabril®Sabril® (vigabatrin) tablets. Pursuant to the terms of this agreement, collaboratorscollaborator could be required to make various payments to the Company, including upfront license fees, milestone payments based on achievement of regulatory approvals, and royalties on sales of products resulting from the collaborative agreement.
Nonrefundable upfront license fees are recognized upon receipt as persuasive evidence of an arrangement exists, the price to the collaborator is fixed or determinable and collectability is reasonably assured. For the three months ended March 31, 2019 and March 31, 2018, no revenue was recognized.
Refer to Note 8, Collaborative Arrangement, for further discussion on the Company’s collaborative arrangement.
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The Company currently has a single product with limited commercial sales experience, which makes it difficult to evaluate its current business, predict its future prospects and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, Firdapse®, and expects Firdapse® to constitute virtually all of product revenue for the foreseeable future. The Company’s success depends on its ability to effectively commercialize Firdapse®.
The Company relies exclusively on third parties to formulate and manufacture Firdapse® and its drug candidates. The commercialization of Firdapse® and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and the commercialization of Firdapse®. If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of drugs.
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
The collaborative agreement provides for a milestone payment upon achievement of development and regulatory events. The Company accounts for milestone payments in accordance with the provisions of Accounting Standards Update (ASU)No. 2010-17, Revenue Recognition – Milestone Method (“Milestone Method of Accounting”). The Company recognizes consideration that is contingent upon the achievement of a milestone in its entirety as revenue in the period in which the milestone is achieved only if the milestone is substantive in its entirety. A milestone is considered substantive when it meets all of the following criteria:
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The consideration relates solely to past performance; and |
| 3. | The consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. |
A milestone is defined as an event (i) that can only be achieved based in whole or in part on either the entity’s performance or on the occurrence of a specific outcome resulting from the entity’s performance, (ii) for which there is substantive uncertainty at the date the arrangement is entered into that the event will be achieved, and (iii) that would result in additional payments being due to the vendor.
The Company believes that achievement of the milestone will be substantive and there will be no substantive uncertainty once the milestone is achieved.
Since the Company will receive royalty reports 60 days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter following the quarter in which the corresponding sales occurred. As of March 31, 2020 and 2019, there was no royalty revenue from sales of the collaborative product.
Refer to Note 7 (Collaborative Arrangement), for further discussion on the Company’s collaborative arrangement.
| m. | RESEARCH AND DEVELOPMENT. Costs incurred in connection with research and development activities are expensed as incurred. These costs consist of direct and indirect costs associated with specific projects, as well as fees paid to various entities that perform research related services for the Company. |
| n. | STOCK-BASED COMPENSATION.The Company recognizes expense in the consolidated statements of operations for the fair value of all stock-based payments to employees, directors and consultants, including grants of stock options and other share-based awards. For stock options, the Company uses the Black-Scholes option valuation model, the single-option award approach, and the straight-line attribution method. Using this approach, compensation cost is amortized on a straight-line basis over the vesting period of each respective stock option, generally one to |
| o. | CONCENTRATION OF RISK.The financial instruments that potentially subject the Company to concentration of credit risk are cash equivalents (i.e., money market funds), investments and accounts receivable, net. The Company places its cash and cash equivalents with high-credit quality financial institutions. These amounts at times may exceed federally insured limits. The Company has not experienced any credit losses in these accounts. |
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
The Company sells its product in the United States through an exclusive distributor (its Customer) to specialty pharmacies. Therefore, its distributor and specialty pharmacies account for all of its trade receivables and net product revenues. The creditworthiness of its Customer is continuously monitored, and the Company has internal policies regarding customer credit limits. The Company estimates an allowance for expected credit loss primarily based on the credit worthiness of its Customer, historical payment patterns, aging of receivable balances and general economic conditions.
The Company currently has a single product with limited commercial sales experience, which makes it difficult to evaluate its current business, predict its future prospects and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, Firdapse®, and expects Firdapse® to constitute virtually all of product revenue for the foreseeable future. The Company’s success depends on its ability to effectively commercialize Firdapse®.
The Company relies exclusively on third parties to formulate and manufacture Firdapse® and its drug candidates. The commercialization of Firdapse® and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and for the commercialization of Firdapse®. If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of its drugs.
| p. | ROYALTIES. Royalties incurred in connection with the Company’s license agreement, as disclosed in Note 9 (Agreements), are expensed to cost of sales as revenue from product sales is recognized. |
| q. | INCOME TAXES. The Company utilizes the asset and liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on differences between the financial reporting and tax basis of assets and liabilities and are measured using enacted tax rates and laws that will be in effect when the differences are expected to reverse. A valuation allowance is provided when it is more likely than not that some portion or all of a deferred tax asset will not be realized. |
The Company recognizes the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting themore-likely-than-not threshold, the amount recognized in the financial statements is the largest benefit that has a greater than 50 percent likelihood of being realized upon ultimate settlement with the relevant tax authority. The Company is subject to income taxes in the U.S. federal jurisdiction and various state jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for years before 2016. If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense.
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
On March 27, 2020, the Coronavirus Aid, Relief, and Economic Security (CARES) Act was signed into law making several changes to the Internal Revenue Code. The changes include, but are not limited to: increasing the limitation on the amount of deductible interest expense, allowing companies to carryback certain net operating losses, and increasing the amount of net operating loss carryforwards that corporations can use to offset taxable income.
The tax law changes in the CARES Act did not have a material impact on the Company’s income tax provision.
| r. | COMPREHENSIVE INCOME (LOSS). U.S. GAAP requires that all components of comprehensive income (loss) be reported in the financial statements in the period in which they are recognized. Comprehensive income (loss) is net income (loss), plus certain other items that are recorded directly into stockholders’ equity. The Company’s comprehensive |
NET |
| March 31, | ||||||||
| 2019 | 2018 | |||||||
Options to purchase common stock | 10,604,500 | 6,902,500 | ||||||
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Potential equivalent common stock excluded | 10,604,500 | 6,902,500 | ||||||
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PotentiallyDiluted net income (loss) per common share is computed by dividing net income (loss) by the weighted average number of common shares outstanding, increased by the assumed conversion of other potentially dilutive options to purchasesecurities during the period.
The following table reconciles basic and diluted weighted average common shares:
| For the Three Months Ended March 31, | ||||||||
| 2020 | 2019 | |||||||
Basic weighted average common shares outstanding | 103,407,347 | 102,747,923 | ||||||
Effect of dilutive securities: | ||||||||
Unvested restricted stock units subject to vesting requirements issued to employees andnon-employees | 352,500 | — | ||||||
Common stock issuable upon the exercise of stock options | 2,774,753 | — | ||||||
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Diluted weighted average common shares outstanding | 106,534,600 | 102,747,923 | ||||||
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Outstanding common stock equivalents totaling approximately 5.4 million, were excluded from the calculation of diluted net income (loss) per common share for the three months ended March 31, 2020 as oftheir effect would be anti-dilutive. For the three months ended March 31, 2019, approximately 10.6 million shares of outstanding stock options were excluded from the calculation of diluted net loss per common share because a net loss was reported in this period and 2018 had exercise prices ranging from $0.79 to $4.64.therefore their effect was anti-dilutive.
| t. | RECLASSIFICATIONS. Certain prior year amounts in the consolidated financial statements have been reclassified to conform to the current year presentation. |
| 2. | Basis of Presentation and Significant Accounting Policies (continued). |
RECENTLY ISSUED ACCOUNTING STANDARDS. In |
In June 2018, the FASB issued ASUNo. 2018-07,Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting that largely aligns the accounting for share-based payment awards issued to employees and nonemployees. Under this ASU, most of the guidance on such payments to nonemployees would be aligned with the requirements for share-based payments granted to employees. ASU2018-07 is effective for all entities for annual reporting periods beginning after December 15, 2018, including interim reporting periods within each annual reporting period, with early adoption permitted. The Company has adopted this standard as of January 1, 2019. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements.
In June 2016, the FASB issued ASUNo. 2016-13,Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments. The standard amends the impairment model by requiring entities to use a forward-looking approach based on expected losses to estimate credit losses for most financial assets and certain other instruments that aren’t measured at fair value through net income. Foravailable-for-sale debt securities, entities will be required to recognize an allowance for credit losses rather than a reduction in carrying value of the asset. Entities will no longer be permitted to consider the length of time that fair value has been less than amortized cost when evaluating when credit losses should be recognized. The Company plans to adoptadopted the new guidancestandard on January 1, 2020. The Company doesadoption of this standard did not anticipate the adoption will have a material impact on itsthe Company’s consolidated financial positionstatements.
In August 2018, the FASB issued ASU2018-15,Intangibles – Goodwill and Other –Internal-Use Software (Subtopic350-40), Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract. The amendments in this update align the requirements for capitalizing implementation costs incurred in a hosting arrangement that is a services contract with the requirements for capitalizing implementation costs incurred to develop or resultsobtaininternal-use software (and hosting arrangements that include aninternal-use software license). Accordingly, the amendments in this update require an entity in a hosting arrangement that is a service contract to follow the guidance in Subtopic350-40 to determine which implementation costs to capitalize as an asset related to the service contract and which costs to expense. The Company adopted the new standard on January 1, 2020 and applied prospectively to all implementation costs incurred after the date of operations.adoption. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements.
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| 3. | Investments. |
Available-for-sale investments by security type were as follows:
| Amortized cost | Gross Unrealized Gains | Gross Unrealized Losses | Estimated Fair Value | |||||||||||||
At March 31, 2019: | ||||||||||||||||
U.S. Treasuries - ST | $ | 9,979,760 | $ | 40 | $ | — | $ | 9,979,800 | ||||||||
U.S. Treasuries - LT | 4,998,328 | — | (6,728 | ) | 4,991,600 | |||||||||||
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Total | $ | 14,978,088 | $ | 40 | $ | (6,728 | ) | $ | 14,971,400 | |||||||
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At December 31, 2018: | ||||||||||||||||
U.S. Treasuries - ST | $ | 10,382,699 | $ | — | $ | (1,835 | ) | $ | 10,380,864 | |||||||
U.S. Treasuries - LT | 5,026,656 | — | (18,413 | ) | 5,008,243 | |||||||||||
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Total | $ | 15,409,355 | $ | — | $ | (20,248 | ) | $ | 15,389,107 | |||||||
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| Estimated Fair Value | Gross Unrealized Gains | Gross Unrealized Losses | Amortized Cost | |||||||||||||
At March 31, 2020: | ||||||||||||||||
U.S. Treasuries – Cash equivalents | $ | 69,997,200 | $ | 83,751 | $ | — | $ | 69,913,449 | ||||||||
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At December 31, 2019: | ||||||||||||||||
U.S. Treasuries – Cash equivalents | $ | 59,932,200 | $ | 2,042 | $ | — | $ | 59,930,158 | ||||||||
U.S. Treasuries – ST | 5,007,050 | 7,463 | — | 4,999,587 | ||||||||||||
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Total | $ | 64,939,250 | $ | 9,505 | $ | — | $ | 64,929,745 | ||||||||
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In accordance with FASB ASC Topic 320, “Investments – Debt and Equity Securities”, or ASC 320, the Company has classified its U.S. Treasuries asavailable-for-sale securities with secondary or resale markets, and, as such, they are reported at fair value with unrealized gains and losses included in comprehensive loss in stockholders’ equity and realized gain and losses, included in other income, net.
| 3. | Investments (continued). |
There were no realized gains or losses fromavailable-for-sale securities for the three months ended March 31, 20192020 or 2018.
Certain U.S. Treasuries at March 31, 2019 had fair values greater or lower than their amortized costs and, therefore, contained unrealized gains (losses). Given that the Company has no intent to sell the U.S. Treasuries until a recovery of the fair value, which may be at maturity, and there are no current requirements to sell any of these securities, the2019.
The Company did not consider these investments to be other-than-temporarily impairedhold any securities in an unrealized position for more than 12 months as of March 31, 2019. The Company anticipates full recovery of amortized costs with respect to these investments at maturity. The duration of time the U.S. Treasuries have been in a continuous unrealized loss position as of March 31, 2019 was more than 12 months.2020.
The estimated fair values ofavailable-for-sale securities at March 31, 2019,2020, by contractual maturity, are summarized as follows:
| March 31, 2019 | ||||
Due in one year or less | $ | 9,979,800 | ||
Due after one year but within two years | 4,991,600 | |||
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| |||
| $ | 14,971,400 | |||
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| |||
| March 31, 2020 | ||||
Due in one year or less | $ | 69,997,200 | ||
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| 4. | Prepaid Expenses and Other Current Assets. |
Prepaid expenses and other current assets consist of the following:
| March 31, 2019 | December 31, 2018 | |||||||
Prepaid research fees | $ | 240,377 | $ | 358,209 | ||||
Prepaid insurance | 616,761 | 800,261 | ||||||
Prepaid commercialization fees | 430,573 | 17,030 | ||||||
Prepaid subscription fees | 287,760 | 170,552 | ||||||
Other | 305,795 | 303,729 | ||||||
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| |||||
Total prepaid expenses and other current assets | $ | 1,881,266 | $ | 1,649,781 | ||||
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| |||||
| March 31, 2020 | December 31, 2019 | |||||||
Prepaid manufacturing costs | $ | 3,471,078 | $ | 1,526,013 | ||||
Prepaid insurance | 995,146 | 1,263,129 | ||||||
Prepaid subscription fees | 492,716 | 501,251 | ||||||
Prepaid research fees | 490,102 | 481,057 | ||||||
Prepaid commercialization expenses | 287,083 | 62,959 | ||||||
Other | 326,590 | 516,665 | ||||||
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| |||||
Total prepaid expenses and other current assets | $ | 6,062,715 | $ | 4,351,074 | ||||
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| 5. | Operating Leases. |
The Company has operating lease agreements for its corporate office. The leases have free and escalating payment provisions. The leases have remaining lease terms of approximately 3.7 years, and include options to extend the leases for up to 1 year and options to terminate the lease within 1 year. There are no obligations under finance leases.
The components of lease expense were as follows:
| ||||
| March 31, 2020 | ||||
Operating lease cost | $ | 74,079 | ||
Supplemental cash flow information related to leases was as follows:
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| March 31, 2020 | ||||
Cash paid for amounts included in the measurement of lease liabilities: | ||||
Operating cash flows | $ | 84,061 | ||
Right-of-use assets obtained in exchange for lease obligations: | ||||
Operating leases | $ | 5,305 | ||
Supplemental balance sheet information related to leases was as follows:
| March 31, 2019 | March 31, 2020 | |||||||
Operating leaseright-of-use assets | $ | 1,074,020 | $ | 730,284 | ||||
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| |||||||
Other current liabilities | $ | 282,605 | $ | 306,677 | ||||
Operating lease liabilities | 875,098 | |||||||
Operating lease liabilities, net of current portion | 568,421 | |||||||
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| |||||||
Total operating lease liabilities | $ | 1,157,703 | $ | 875,098 | ||||
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| |||||||
Weighted Average Remaining Lease Term | 3.7 years | |||||||
Weighted Average Discount Rate | 4.81 | % | ||||||
Payments
| 5. | Operating Leases (continued). |
Weighted average remaining lease term | 2.7 years | |||
Weighted average discount rate | 4.81 | % |
Remaining payments of lease liabilities as of March 31, 20192020 were as follows:
2019 (remaining nine months) | $ | 248,108 | ||
2020 | 339,605 | |||
2021 | 349,788 | |||
2022 | 329,662 | |||
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| |||
Total lease payments | 1,267,163 | |||
Less imputed interest | (109,460 | ) | ||
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| |||
Total | $ | 1,157,703 | ||
|
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|
Property and equipment, net consists of the following:
| March 31, 2019 | December 31, 2018 | |||||||
Computer equipment | $ | 52,704 | $ | 52,704 | ||||
Furniture and equipment | 218,084 | 212,451 | ||||||
Leasehold improvements | — | 177,417 | ||||||
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| |||||
| 270,788 | 442,572 | |||||||
Less: Accumulated depreciation | (130,468 | ) | (197,147 | ) | ||||
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| |||||
Total property and equipment, net | $ | 140,320 | $ | 245,425 | ||||
|
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| |||||
Depreciation expense was $13,748 and $8,015, respectively, for the three-month periods ended March 31, 2019 and 2018.
2020 (remaining nine months) | $ | 255,544 | ||
2021 | 349,788 | |||
2022 | 329,662 | |||
|
| |||
Total lease payments | 934,994 | |||
Less imputed interest | (59,896 | ) | ||
|
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Total | $ | 875,098 | ||
|
|
Accrued Expenses and Other Liabilities. |
Accrued expenses and other liabilities consist of the following:
| March 31, 2019 | December 31, 2018 | March 31, 2020 | December 31, 2019 | |||||||||||||
Accrued preclinical and clinical trial expenses | $ | 943,742 | $ | 821,633 | $ | 940,963 | $ | 1,183,513 | ||||||||
Accrued professional fees | 1,651,181 | 1,311,061 | 3,309,351 | 1,241,526 | ||||||||||||
Accrued compensation and benefits | 764,362 | 1,941,449 | 1,814,396 | 3,064,645 | ||||||||||||
Accrued license fees | 1,573,642 | 3,000,000 | 3,945,991 | 8,751,991 | ||||||||||||
Lease liability | 282,605 | — | ||||||||||||||
Accrued purchases | 1,243,991 | 1,313,310 | ||||||||||||||
Accrued contributions | 1,135,000 | 1,535,000 | ||||||||||||||
Operating lease liability | 306,677 | 300,518 | ||||||||||||||
Accrued variable consideration | 704,834 | — | 1,308,344 | 884,764 | ||||||||||||
Deferred rent and lease incentive | — | 33,408 | ||||||||||||||
Income tax payable | 2,143,490 | 1,533,696 | ||||||||||||||
Other | 165,663 | 66,436 | 139,733 | 172,332 | ||||||||||||
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Current accrued expenses and other liabilities | 6,086,029 | 7,173,987 | 16,287,936 | 19,981,295 | ||||||||||||
Lease liability -non-current | 875,098 | — | ||||||||||||||
Deferred rent and lease incentive –non-current | — | 154,799 | ||||||||||||||
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| |||||||||||||||
Leaseliability—non-current | 568,421 | 647,532 | ||||||||||||||
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| |||||||||||||
Non-current accrued expenses and other liabilities | 875,098 | 154,799 | 568,421 | 647,532 | ||||||||||||
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| |||||||||||||
Total accrued expenses and other liabilities | $ | 6,961,127 | $ | 7,328,786 | $ | 16,856,357 | $ | 20,628,827 | ||||||||
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Collaborative Arrangement. |
In December 2018, the Company entered into a collaboration and license agreement (collaboration)(Collaboration) with Endo, International plc’s subsidiary, Endo Ventures Limited (EVL), for the further development and commercialization of generic Sabril®Sabril® (vigabatrin) tablets through Endo’s U.S. Generic Pharmaceuticals segment, doing business as Par Pharmaceutical.
EVL has assumedUnder the Collaboration, Endo assumes all development, manufacturing, clinical, regulatory, sales and marketing costs under the collaboration, while the Company is responsible for exercising commercially reasonable efforts to develop, or cause the development of, a final finished, stable dosage form of generic Sabril®Sabril® tablets.
Under the terms of the Collaboration, the Company has received anup-front payment, and will receive a milestone payments based on achievement of regulatory approvals,payment, and a sharing of defined net profits upon commercialization from EVLEndo consisting of amid-double digit percent of net sales of generic Sabril®Sabril®. The Company has also agreed to a sharing of certain development expenses. Unless terminated earlier in accordance with its terms, the collaboration continues in effect until the date that is ten years following the commercial launch. Forlaunch of the year ended December 31, 2018, a $500,000 upfront license fee was recognized. For the three-month period ending March 31, 2019, no collaborative arrangement revenue was recognized.product.
Collaborative Arrangement (continued). |
The collaborative agreement provides for a $2.0 million milestone payment on the commercial launch of the product by Par. As of March 31, 2020 and 2019, no milestone payments have been earned.
There were no revenues from collaborative arrangement for the three months ended March 31, 2020 and 2019. Total expenses incurred, net, in connection with the collaborative agreement for three months ended March 31, 2020 and March 31, 2019 were approximately $5,700 and $11,952, and have been included in research and development expenses in the accompanying consolidated statements of operations.
| 8. | Commitments and Contingencies. |
In 2018, the Company became aware that certain patents granted to Northwestern in 2018University (which patents have been licensed by Northwestern to a third-party)third party) for a new GABA aminotransferase inhibitor were deriveddeveloped fromCPP-115.CPP-115, which had previously been licensed to the Company by Northwestern. As a result, the Company terminated its license agreement with Northwestern for CPP-115, based on the Company’s position that Northwestern has violated the license agreement because of its failure to transfer these new patent rights to the Company as part of the existing license agreement. It is also the Company’s position that Northwestern’s publication of information about the new patents in violation of the license agreement has damaged the Company. On October 26, 2018, the Company terminated the license agreement forCPP-115and filed a claim for damages incommenced an arbitration proceeding against Northwestern seeking damages for Northwestern’salleged breaches of the license agreement.
On November 5, 2018, Shortly thereafter, Northwestern advisedfiled counterclaims against the Company that in its view, Northwestern has a right to terminate the license agreement with the Company because the Company has allegedly breached the license agreement by failing to pay certain milestones and by allegedly failing to use commercially reasonable efforts to develop and commercialize any products. Northwestern has also advised the Company that, in its view, the Company has engaged in wrongful conduct and communications with the third party that licensed the new patents from Northwestern, and that such communications have damaged Northwestern’s relationship with that third party. The Company disputes Northwestern’s allegations and is vigorously defending itself against claims that Northwestern has brought against it in the arbitration proceeding.action seeking damages for alleged breaches by the Company of the license agreement. On May 21, 2019, the Company entered into a settlement agreement with Northwestern that resolved all pending disputes between the parties with no admission of liability by either party, released all claims of liability or wrongdoing between the Company and Northwestern, and dismissed the pending arbitration. Under the settlement agreement, the Company received a $100,000 payment on May 21, 2019, which is reported as income in other income, net in the consolidated statement of operations. The Company is also entitled to receive certain contingent compensation that will be reported when and if received.
In May 2019, the FDA approved an NDA for Jacobus Pharmaceuticals for Ruzurgi®, their version of amifampridine (3,4-DAP), for the treatment of pediatric LEMS patients (ages 6 to under 17). The Company believes that Jacobus is offering Ruzurgi® at a lower price than the Company is offering Firdapse®. In addition, while the NDA for Ruzurgi® only covers pediatric patients, the Company believes Ruzurgi® is being prescribed off label to adult LEMS patients. If Jacobus is able to successfully sell Ruzurgi®off-label to adult LEMS patients, it could have a material adverse effect on the Company’s business, financial condition and results of operations.
The Company believes that the FDA’s approval of Ruzurgi® violated its statutory rights and was in multiple other respects arbitrary, capricious and contrary to law. As a result, in June 2019 the Company filed suit against the FDA and several related parties challenging this approval and related drug labeling. The Company’s complaint, which was filed in the federal district court for the Southern District of Florida, alleges that the FDA’s approval of Ruzurgi® violated multiple provisions of FDA regulations regarding labeling, resulting in misbranding in violation of the Federal Food, Drug, and Cosmetic Act (FDCA); violated its statutory rights to Orphan Drug Exclusivity and New Chemical Entity Exclusivity under the FDCA; and was in multiple other respects arbitrary, capricious, and contrary to law, in violation of the Administrative Procedure Act. Among other remedies, the suit seeks an order vacating the FDA’s approval of Ruzurgi®. Jacobus has intervened in the case. Each party has filed a cross motion for summary judgement. There can be no assurance as to the outcome of this lawsuit.
Additionally, from time to time the Company may become involved in legal proceedings arising in the ordinary course of business. Except as set forth above, the Company believes that there is no other litigation pending at this time that could have, individually or in the aggregate, a material adverse effect on its results of operations, financial condition or cash flows.
|
Agreements. |
| a. | LICENSE AGREEMENT WITH BIOMARIN (FIRDAPSE®). On October 26, 2012, the Company entered into a license agreement with BioMarin Pharmaceutical, Inc. (BioMarin) for the North American rights to |
On May 29, 2019, the Company entered into an amendment to its license agreement for Firdapse®. Under the amendment, the Company has expanded its commercial territory for Firdapse®, which originally was comprised of North America, to include Japan. Additionally, the Company has an option to further expand its territory under the license agreement to include most of Asia, as well as Central and South America, upon the achievement of certain milestones in Japan. Under the amendment, the Company will pay royalties on net sales in Japan of a similar percentage to the royalties that the Company is currently paying under its original license agreement for North America.
During January 2020, the Company was advised that BioMarin has transferred certain rights under the license agreement to SERB S.A.
| b. | AGREEMENTS FOR DRUG MANUFACTURING, DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES. The Company has entered into agreements with contract manufacturers for the manufacture of commercial drug and drug and study placebo for the Company’s trials and studies, with contract research organizations (CRO) to conduct and monitor the Company’s trials and studies and with various entities for laboratories and other testing related to the Company’s trials and studies. The contractual terms of the agreements vary, but most require certain advances as well as payments based on the achievement of milestones. Further, these agreements are cancellable at |
Income Taxes. |
The Company is subject to income taxes in the U.S. federal jurisdiction and various states jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for any years before 2015.2016. If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense.
The Company’s netCompany has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax asset has a 100% valuation allowance atassets. As of March 31, 20192020 and December 31, 2018 as2019, based on the Company’s history of operating losses, the Company believeshas concluded that it is more likely than not that the benefit of its deferred tax assetassets will not be realized.
Stockholders’ Equity. |
2016 Shelf Registration StatementPreferred Stock
OnThe Company has 5,000,000 shares of authorized preferred stock, $0.001 par value per share, at March 31, 2020 and December 23, 2016, the31, 2019. No shares of preferred stock were outstanding at March 31, 2020 and December 31, 2019.
| 11. | Stockholders’ Equity (continued). |
Common Stock
The Company filed a shelf Registration Statement on FormS-3 (the 2016 Shelf Registration Statement) with the SEC to sell up to approximately $33.8 millionhas 150,000,000 shares of authorized common stock, par value $0.001 per share. At March 31, 2020 and December 31, 2019, 103,408,699 and 103,397,033 shares, respectively, of common stock. The 2016 Shelf Registration Statement (fileNo. 333-215315) was declared effective by the SECstock were issued and outstanding. Each holder of common stock is entitled to one vote of each share of common stock held of record on January 9, 2017. No sales have been conductedall matters on which stockholders generally are entitled to date under the 2016 Shelf Registration Statement.vote.
2017 Shelf Registration Statement
On July 12, 2017, the Company filed a universal shelf Registration Statement on FormS-3 (the 2017 Shelf Registration Statement) with the SEC to sell up to $150 million of common stock, preferred stock, warrants to purchase common stock, or debt securities (including debt securities that may be convertible or exchangeable for common stock or other securities), which securities may be offered separately or together in units or multiple series. The 2017 Shelf Registration Statement (fileNo. 333-219259) was declared effective by the SEC on July 26, 2017.
On November 28, 2017, the Company filed a prospectus supplement and offered for sale 16,428,572 shares of its common stock at a price of $3.50 per share in an underwritten public offering under the 2017 Shelf Registration. The Company received gross proceeds in the public offering of approximately $57.5 million before underwriting commission and incurred expenses of approximately $3.7 million.
At March 31, 2019,2020, there is approximately $92.5 million available for future sale under the 2017 Shelf Registration Statement.
Stock Compensation. |
For the three-month periods ended March 31, 20192020 and 2018,2019, the Company recorded stock-based compensation expense as follows:
| Three months ended March 31, | Three months ended March 31, | |||||||||||||||
| 2019 | 2018 | 2020 | 2019 | |||||||||||||
Research and development | $ | 287,721 | $ | 294,315 | $ | 418,053 | $ | 287,721 | ||||||||
Selling, general and administrative | 645,690 | 677,025 | 1,101,298 | 645,690 | ||||||||||||
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|
| |||||||||||||
Total stock-based compensation | $ | 933,411 | $ | 971,340 | $ | 1,519,351 | $ | 933,411 | ||||||||
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Stock Options
As of March 31, 2019,2020, there were outstanding stock options to purchase 10,604,50012,201,668 shares of common stock, of which stock options to purchase 5,259,1566,687,980 shares of common stock were exercisable as of March 31, 2019.2020.
During the three-month periods ended March 31, 20192020 and 2018,2019, the Company granted seven-year term options to purchase an aggregate of 207,000735,000 and 1,772,500207,000 shares, respectively, of the Company’s common stock to employees and directors. The Company recorded stock-based compensation related to stock options totaling $933,411$1,383,672 and $971,340,$933,411, respectively, during the three-month periods ended March 31, 20192020 and 2018.2019. During the three-month periods ended March 31, 2020 and 2019, respectively, 854,831 and 2018, respectively, 984,164 and 744,998 options vested.
During the three-month periods ended March 31, 20192020 and 2018,2019, options to purchase 65,00011,666 shares and 36,66665,000 shares, respectively, of the Company’s common stock were exercised, with proceeds of $89,350$26,149 and $33,032,$89,350 respectively, to the Company.
As of March 31, 2019,2020, there was approximately $8,071,744$10.4 million of unrecognized compensation expense related tonon-vested stock option awards granted under the 2014 and 2018 Stock Incentive Plans. The cost is expected to be recognized over a weighted average period of approximately 2.382.23 years.
Stock Compensation (continued). |
Restricted Stock Units
There were no grants of restricted stock units to employees or directors during the three-month periods ended March 31, 2020 and March 31, 2019. During the three-month period ended March 31, 2020, the Company recordednon-cash stock-based compensation expense related to restricted stock units totaling $135,679. No stock-based compensation related to restricted stocks was recorded during the three-month period ended March 31, 2019.
As of March 31, 2020, there was approximately $1.5 million of unrecognized compensation expense related tonon-vested restricted stock units granted under the 2018 Stock Incentive Plan. The cost is expected to be recognized over a weighted average period of approximately 2.67 years.
| 13. | Subsequent Events. |
TheOn May 7, 2020, the Company became aware earlier this week from press reports thatamended the FDA has approved an NDAlease for Jacobus Pharmaceuticals for their version of 3,4-DAP for pediatric LEMS patients. The Company is inits office facilities. Under the early stages of assessing both the impact of the FDA’s recent action on its business and its available options, and there can be no assurance as to the ultimate impact of the FDA’s recent action onamended lease, the Company’s business and future resultsleased space will increase from approximately 7,800 square feet of operations.space to approximately 10,700 square feet of space.
ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS |
Introduction
Management’s Discussion and Analysis of Financial Condition and Results of Operations (MD&A) is intended to provide an understanding of our financial condition, changes in financial condition and results of operations. The discussion and analysis is organized as follows:
| • | Overview.This section provides a general description of our business and information about |
| • | Basis of Presentation.This section provides information about key accounting estimates and policies that we followed in preparing our consolidated financial statements for |
| • | Critical Accounting Policies and |
| • | Results of |
| • | Liquidity and Capital |
| • | Caution Concerning Forward-Looking |
OverviewOVERVIEW
We are a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare, debilitating, chronic neuromuscular and neurological diseases. We are dedicated to making a meaningful impact on the lives of those suffering from rare diseases, and we believe in putting patients first in everything we do.
Impact of theCOVID-19 pandemic on our business
The recent outbreak of a novel strain of coronavirus(COVID-19) has resulted in extended shutdowns of many businesses, the curtailment of travel and large gatherings, and economic and labor instability in the United States and around the world.COVID-19 was declared a pandemic by the World Health Organization in March 2020, and on March 16, 2020, in light of worsening conditions as a result of the pandemic, we implemented a number of safety related initiatives among our employees, including a travel ban and a work from home policy for all employees. This included our customer-facing employees, who are working remotely and utilizing telephone andweb-based technologies to provide support to patients and their healthcare providers. Since many healthcare providers are delaying seeing patients other than those affected byCOVID-19, this has, starting recently, delayed the diagnosis of LEMS patients and their initiating therapy, which has slowed our efforts to locate new patients who could benefit from our therapy.
Our Firdapse® supply chain remains robust and thus far we have observed no disruptions in the production of Firdapse®. We reiterate that we are committed to providing patients with the ability to obtain an uninterrupted supply of Firdapse®, and we believe that we have an adequate supply of Firdapse® to address patients’ needs through at least June 2021. Further, we are advised by our U.S. manufacturing partners that they have implemented contingency plans to remain in operation. We are committed to meeting our “patients” needs for Firdapse® and believe that our supply chain will remain solid and uninterrupted through theCOVID-19 outbreak and beyond.
We were fortunate that all subjects were enrolled in the MuSK-MG trial before the full outbreak of theCOVID-19 health emergency. However, while all subject visits in our MuSK-MG trial have now been completed, delays in closing out trial sites and data-collection from the study will likely delay our ability to reporttop-line results from this trial until the third quarter of 2020. Further, while all of the subjects who have agreed to participate in our SMA Type 3proof-of-concept study have been identified, the last seven subjects will not be able to complete the trial until the trial sites that are conducting the study are permitted by regulatory authorities to resume clinical trial activities. While there can be no assurance, we now believe that our SMA Type 3 study will be completed later this year and that we will be in a position to reporttop-line results from this study by the end of 2020.
While we are doing our best to monitor and react to the impact of theCOVID-19 health crisis on our business, there can be no assurance as to the ultimate manner in which these recent events will impact our business and our results of operations.
Firdapse®
In October 2012, we licensed the North American rights to Firdapse®Firdapse®, a proprietary form of amifampridine phosphate, or chemically known as3,4-diaminopyridine phosphate, from BioMarin Pharmaceutical Inc. (BioMarin). phosphate. When we acquired the rights to the product, it had already been granted orphan drug designation by the FDAFood and Drug Administration (FDA) for the treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS),LEMS, a rare and sometimes fatal autoimmune disease characterized by muscle weakness. Additionally, in August 2013, we were granted “breakthrough therapy designation” by the United States Food & Drug Administration (FDA),FDA for Firdapse®Firdapse® for the treatment of LEMS. Further, the FDA has granted Orphan Drug Designation for Firdapse®Firdapse® for the treatment of Congenital Myasthenic Syndromes (CMS) and Myasthenia Gravis (MG).
On November 28, 2018, we received approval from the FDA for Firdapse®Firdapse® 10 mg tablets for the treatment of adults with LEMS.LEMS (age 17 and above). In January 2019, we launched Firdapse® in the United States, selling through a field force experienced in neurologic, central nervous system or rare disease products consisting at the time of approximately 20 field personnel, including sales (Regional Account Managers), patient assistance and insurance navigation support (Patient Access Liaisons), and payer reimbursement (National Account Managers) personnel. We also have a field-based force of six medical science liaisons who are helping educate the medical communities and patients about LEMS and about our ongoing clinical trial activities evaluating Firdapse®Firdapse® for other ultra-orphan, neuromuscular diseases. Finally, we are working with several rare disease advocacy organizations (including Global Genes, the National Organization for Rare Disorders (NORD), and the Myasthenia Gravis Foundation of America) to help increase awareness and level of support for patients living with LEMS, Anti-MuSK antibody positive myasthenia gravis, orMuSK-MG, CMS, and Spinal Muscular Atrophy (SMA) Type 3, and to provide education for the physicians who treat these rare diseases and the patients they treat.
In early 2020, we expanded our field sales group by almost one hundred percent and contracted with a rare-disease experienced inside sales agency. Through this recent expansion of our sales team, we hope to expand our sales efforts beyond the neuromuscular specialists who regularly treat LEMS patients to reach roughly 9,000 neurology and neuromuscular healthcare providers that may be treating an adult LEMS patient who can benefit from Firdapse®. We also recently launched ourno-cost LEMS voltage gated calcium channel (VGCC) antibody testing program (using a commercially available test approved by the FDA) for use by physicians who suspect their patient may have LEMS and wish to reach a definitive diagnosis.
Because of theCOVID-19 pandemic, we have recently implemented a number of safety related initiatives among our employees, including a travel ban and a work from home policy for all employees. This included our customer-facing employees, who are working remotely and utilizing telephone andweb-based technologies to provide support to patients and their healthcare providers. We are also continuing to expand our digital and social media activities in order to introduce our product to potential patients and their healthcare providers. Since many healthcare providers are delaying seeing patients other than those affected byCOVID-19, this has, starting recently, limited our ability to locate new patients who might benefit from our drug and slowed our efforts to increase our sales from prior periods.
We are supporting the distribution of Firdapse® through “Catalyst Pathways™”,” our personalized treatment support program. “Catalyst Pathways”Pathways™” is a single source for personalized treatment support, education and guidance through the challenging dosing and titration regimen to an effective therapeutic dose. It also includes distributing the drug through a very small group of exclusive specialty pharmacies (primarily AnovoRx), which is consistent with the way that most pharmaceutical products for ultra-orphan diseases are distributed and dispensed to patients. We believe that by using specialty pharmacies in this way, the difficult task of navigating the health care system is far better for the patient needing treatment for their rare disease and the health care community in general.
In order to help adult LEMS patients afford their medication, we, like other pharmaceutical companies which are marketing drugs for ultra-orphan conditions, have developed an array of financial assistance programs that are available to reduce patientco-pays and deductibles to a nominal affordable amount. For eligible patients with commercial coverage, aco-pay assistance program designed to keepout-of-pocket costs to $10 or lessnot more than $10.00 per month is available for all LEMS patients prescribed Firdapse®. We are also donating, and committing to continue to donate, money to qualified, independent charitable foundations dedicated to providing assistance to any U.S. LEMS patients in financial need. Subject to compliance with applicable regulatory requirements, our goal is that no LEMS patient is ever denied access to Firdapse® for financial reasons.
In May 2019, the FDA approved a New Drug Application (NDA) for Ruzurgi®, another version of amifampridine (3,4-DAP), for the treatment of pediatric LEMS patients (ages 6 to under 17). Based on publicly available information, we believe that Jacobus Pharmaceuticals is offering Ruzurgi® at a list price of $80 for each 10 mg tablet, and the Jacobus’ drug is approved up to a maximum daily dose of 100 mg. Based on this price, we believe that the cost for a patient taking a daily dose of 60 mg per day would be $175,200 annually and the cost for a patient taking a daily dose of 100 mg would be $292,000 annually. Both prices are lower than the list price for an equivalent amount of Firdapse®. In addition, while the NDA for Ruzurgi® only covers pediatric patients, we believe that Ruzurgi® is being prescribed off label to some number of adult LEMS patients. If Jacobus is able to successfully sell Ruzurgi®off-label to additional adult LEMS patients, it could have a material adverse effect on our business, financial condition and results of operations.
We became aware earlierbelieve that the FDA’s approval of Ruzurgi® violated our statutory rights and was in multiple other respects arbitrary, capricious and contrary to law. As a result, in June 2019 we filed suit against the FDA and several related parties challenging this week from press reportsapproval and related drug labeling. Our complaint, which was filed in the federal district court for the Southern District of Florida, alleges that the FDA’s approval of Ruzurgi® violated multiple provisions of FDA regulations regarding labeling, resulting in misbranding in violation of the Federal Food, Drug, and Cosmetic Act (FDCA); violated our statutory rights to Orphan Drug Exclusivity and New Chemical Entity Exclusivity under the FDCA; and was in multiple other respects arbitrary, capricious, and contrary to law, in violation of the Administrative Procedure Act. Among other remedies, the suit seeks an order setting aside the FDA’s approval of Ruzurgi®.
We have filed a motion for summary judgement in our case, and the FDA has approved an NDAfiled a cross motion for summary judgement. Further, Jacobus Pharmaceuticals for their version of 3,4-DAP for pediatric LEMS patients. We are continuing to focus our attention on our core mission of serving the needs of LEMS patients and on educating the medical community about this devastating disease. We are alsohas intervened in the early stages of assessing bothcase and has filed their own cross motion for summary judgement. Based on currently available information, we expect a decision in the impact of the FDA’s recent action on our business and our available options, and therecase sometime later this year. There can be no assurance as to the ultimate impactoutcome of this lawsuit, as to the FDA’s recent action ontiming of any decision, or as to the likelihood of an appeal if our business and future results of operations.suit is successful.
We are currently conducting arecently completed the last subject visits in our Phase 3 clinical trial evaluating Firdapse® for the treatment of adults withMuSK-MGMuSK-MG. Our trial is a multi-site, international (United States, Italy and Serbia), double-blind, placebo-controlled, clinical trial being conducted under a Special Protocol Assessment (SPA) with the FDA. The trial is a multi-site, international (United States and Italy), double-blind, placebo-controlled, clinical trial that is targeted to enroll approximatelyenrolled more than 60 subjects diagnosedwith MuSK-MG. The trial willMuSK antibody positive patients. It also enroll up toenrolled more than 10 generalized myasthenia gravis patients who will bewere assessed with the same clinical endpoints butendpoints. However, achieving statistical significance in this subgroup of patients is not required and only summary statistics will be provided. We initiated this trial in January 2018 and are currently enrolling subjects. We currently expect toreport top-line results from this trial in the second half of 2019. Details of this trial are available on www.clinicaltrials.gov (NCT03304054).
We are currently conducting a Phase 3 clinical trial evaluating Firdapse® for the treatment of certain types of CMS. This trial, which will include approximately 23 adult and pediatric subjects, is being conducted at trial sites around the United States and Canada. Details of this trial are available onwww.clinicaltrials.gov (NCT02562066)(NCT03304054). Based on currently available information, we expect
We were fortunate that all subjects were enrolled in our MuSK-MG trial before the full outbreak of theCOVID-19 health emergency. However, while all subject visits in our MuSK-MG trial have now been completed, delays in closing out trial sites and data-collection from the trial will likely delay our ability to reporttop-line results from this trial inuntil the second halfthird quarter of 2019.2020.
Because
If our trial is successful, we intend to file a supplemental new drug application (sNDA) with the FDA has granted Orphan Drug Designation for Firdapse®for the treatment of patients with CMS and Myasthenia Gravis (MG), if we are the first to receive approvals in the future for Firdapse® for the treatment of CMS orMuSK-MG,MUSK-MG we would be eligible to receive seven years of marketing exclusivity for those indications added to our Firdapse® label, of which there can be no assurance.indication.
We are currently conducting aproof-of-concept clinical study evaluating Firdapse® as a symptomatic treatment for ambulatory patients with Spinal Muscular Atrophy (SMA) Type 3, ambulatory.3. The study, which is designed asbeing conducted at trial sites in Italy and Serbia, plans to evaluate approximately 12 subjects in a randomized (1:1), double-blind,2-period,2-treatment, crossover, outpatientproof-of-concept study to evaluateevaluating the safety, tolerability and potential efficacy of amifampridine in ambulatory patients diagnosed with SMA Type 3. TheDetails of this trial are available on www.clinicaltrials.gov (NCT03781479).
We have identified and recruited all of the subjects necessary to complete thisproof-of-concept study. However, the last seven subjects will not be able to complete the study is planneduntil the trial sites conducting the study are permitted to include approximately 12 patients,resume clinical trial activities. While there can be no assurance, we now believe that this study will be completed later this year and that we currently expectwill be in a position to reporttop-line results from this study inbefore the first halfend of 2020. Details of this trial are available onwww.clinicaltrials.gov (NCT03781479)
We also plan to begin studies in 2020 evaluating Firdapse® as a treatment for Kennedy’s Disease and Hereditary Neuropathy with liability to Pressure Palsies (HNPP). However, our plans for these studies have not yet been finalized and we do not yet know what form they will take or what timelines they will be on.
There can be no assurance that our currently ongoing trialsclinical programs evaluating Firdapse® for the treatment ofMuSK-MG, CMS, or SMA Type 3, or any trials we may undertake in the future to evaluate Firdapse® for the treatment of other rare neuromuscular diseases, will be successful. Further, there can be no assurance that we will ever be granted the right to commercialize Firdapse®Firdapse® for any of these additional indications.
Finally,We are also currently in the early stages of developing a long-acting formulation of amifampridine. We have retained a contractor which is currently assisting us in developing the formulation of the product. We currently anticipate that initial formulation candidates and their drug release and absorption properties should be determined during 2020. There can be no assurance we are taking stepswill be able to seek approval for Firdapse® in Canada. We also intend as a longer term strategy to seek tosuccessfully develop a sustained releaselong-acting formulation of amifampridine and that such formulation will ever be approved by the FDA for commercialization.
In October 2019, we submitted an NDS in Canada seeking approval of Firdapse® for the treatment of LEMS. Our application has been accepted for review and we have been granted a priority review. There can be no assurance that our application will be approved. In addition, since there is no orphan exclusivity in Canada, even if our NDS for Firdapse® is approved, there can be no assurance that any application for amifampridine filed by other parties will not be approved as well. As a result, even if our NDS is approved, we may face competition in Canada for LEMS patients.
In May 2019, we entered into an amendment to our license agreement for Firdapse®. Under the amendment, we have expanded our commercial territory for Firdapse®, which originally was comprised of North America, to include Japan. Additionally, we have an option to further expand our territory under the license agreement to include most of Asia, as well as Central and South America, upon the achievement of certain milestones in Japan. Under the amendment, we will pay royalties on net sales in Japan of a similar percentage to the royalties that we are currently paying under our original license agreement for North America.
We are currently in discussions with Japanese regulatory authorities to determine the type of clinical trial that will be required before we will be granted the right to file an application to commercialize Firdapse® in Japan. There can be no assurance that we will successfully obtain the right to commercialize Firdapse® in Japan.
All of our patent rights for Firdapse® are derived from our license agreement. Under the License Agreement, we licensed two pending patents and certain trademarks for Firdapse®. One of the licensed applications, U.S. App. No. 10/467,082 is abandoned as are its children (U.S. App. No. 14/085,017 and 14/818,848) such that we are no longer pursuing patent protection out of this family of applications. The second licensed patent application claims methods of administering Firdapse®. We recently received an office action from the United States Patent and Trademark Office responding to our second application, and we are in the process of responding to that office action. There can be successfulno assurance that our pending patent will be granted or as to the protection from competition that it will provide us if it is granted.
Further, there can be no assurance that we do not or will not infringe on patents held by third parties or that third parties in these efforts.the future will not claim that we have infringed on their patents. In the event that our products or technologies infringe or violate the patent or other proprietary rights of third parties, there is a possibility we may be prevented from pursuing product development, manufacturing or commercialization of our products that utilize such technologies until the underlying patent dispute is resolved. For example, there may be patents or patent applications held by others that contain claims that our products or operations might be determined to infringe or that may be broader than we believe them to be. Given the complexities and uncertainties of patent laws, there can be no assurance as to the impact that future patent claims against us may have on our business, financial condition, results of operations, or prospects.
Generic Sabril®
In September 2015, we announced the initiation of a project to develop generic versions of Sabril®(vigabatrin). Sabril®is marketed by Lundbeck Inc. in the United States in two dosage forms (powder sachets and tablets) for the treatment of infantile spasms and refractory complex partial seizures. Par Pharmaceutical brought the first generic version of the powder sachet to market, and, to date, several generic versions of the powder sachets have been approved. However, at this time, there is only one approved generic version of the tablets.
On December 18, 2018, we announced that we had signedentered into a definitive agreement with Endo International plc’s subsidiary, Endo Ventures Limited (“Endo”), for the further development and commercialization of generic Sabril®Sabril® tablets through Endo’s United States Generic Pharmaceuticals segment, Par Pharmaceutical. Pursuant to the agreement, in December 2018, we have received anup-front payment of $500,000, and$500,000. We will be entitled to receive a milestone payment of $2.0 million on the commercial launch of the product. Further, we will receive milestone payments based on achievement of regulatory approvals, and a sharing of defined net profits upon commercialization and we are obligated to share the costs of certain expenses for development.development expenses.
There can be no assurance that our collaboration with Endo for the development of generic Sabril® (vigabatrin) tablets will be successful and that if an ANDAabbreviated new drug application (ANDA) is approved for vigabatrin tablets in the future, that it will be profitable to us.
Available Capital Resources
At March 31, 2019,2020, we had cash and investments of approximately $50.6$101.8 million. Based on our current financial condition and forecasts of available cash, we believe that we have sufficient funds to support our operations for at least the next 12 months.months from the date of this Form10-Q. There can be no assurance that we will continue to be successful in commercializing Firdapse®Firdapse® or becomewill continue to be profitable or as to whetherand cash flow positive. Further, there can be no assurance that if we will requireneed additional funding in the future, (and whether any such required funding will be available).available to us. See “Liquidity and Capital Resources” below for further information on our liquidity and cash flow.
Basis of Presentation
Revenues.
At March 31, 20182020 we were a development stage company, as we had no revenues from product sales. At March 31, 2019 we have beguncontinued to generate revenues from product sales as we have begun to commercialize Firdapse®of Firdapse®. We expect these revenues to fluctuate in future periods based on our sales of Firdapse®Firdapse®. At March 31, 2020 and March 31, 2019, we did not generate revenues under our collaborative agreement with Endo. We expect our revenues from the collaborative agreement to fluctuate in future periods based on our collaborator’s ability to meet various regulatory milestones set forth in such agreement.
Cost of Sales.
Cost of sales consists of third-party manufacturing costs, freight, royalties, and indirect overhead costs associated with sales of Firdapse®Firdapse®. Cost of sales may also include period costs related to certain inventory manufacturing services, inventory adjustments charges, unabsorbed manufacturing and overhead costs, and manufacturing variances. Prior to FDA approval in November 2018, the cost of manufacturing Firdapse®Firdapse® was expensed, including ourbuild-up of anticipated launch product. This will cause thehas caused cost of sales to appear artificially low foras we consumed product manufactured prior to approval, and will continue to do so until we deplete such product and additional product is manufactured.
Research and Development Expenses.
Our research and development expenses consist of costs incurred for company-sponsored research and development activities, as well as support for selected investigator-sponsored research. The major components of research and development costs include preclinical study costs, clinical manufacturing costs, clinical study and trial expenses, insurance coverage for clinical trials, consulting, and other third-party costs, salaries and employee benefits,
stock-based compensation expense, supplies and materials, and allocations of various overhead costs related to our product development efforts. To date, all of our research and development resources have been devoted to the development of Firdapse®Firdapse®,CPP-109 (our version of vigabatrin), and formerlyCPP-115, and we currently expect that our future development costs will be attributable principally to the continued development of Firdapse®Firdapse®.
Our cost accruals for clinical studies and trials are based on estimates of the services received and efforts expended pursuant to contracts with numerous clinical study and trial sites and clinical research organizations (CROs). In the normal course of our business we contract with third parties to perform various clinical study and trial activities in thethe on-going development of potential products. The financial terms of these agreements are subject to negotiation and vary from contract to contract and may result in uneven payment flows. Payments under the contracts depend on factors such as the achievement of certain events or milestones, the successful enrollment of patients, the allocation of responsibilities among the parties to the agreement, and the completion of portions of the clinical study or trial or similar conditions. The objective of our accrual policy is to match the recording of expenses in our consolidated financial statements to the actual services received and efforts expended. As such, expense accruals related to preclinical and clinical studies or trials are recognized based on our estimate of the degree of completion of the event or events specified in the specific study or trial contract. We monitor service provider activities to the extent possible; however, if we underestimate activity levels associated with various studies or trials at a given point in time, we could be required to record significant additional research and development expenses in future periods. Preclinical and clinical study and trial activities require significantup-front expenditures. We anticipate paying significant portions of a study or trial’s cost before such begins,they begin, and incurring additional expenditures as the study or trial progresses and reaches certain milestones.
Selling, General and Administrative Expenses.
We had no product sales or selling expenses in the first quarter of 2018. During the first quarter of 2019, we have actively committed funds to developing our commercialization program for Firdapse®Firdapse® and we have continued to incur commercialization expenses, inclusive of sales, marketing, patient services, patient advocacy and other commercialization related expenses, as we have beguncontinued our sales program for Firdapse®Firdapse®.
Our general and administrative expenses consist primarily of salaries and personnel expenses for accounting, corporate, compliance, and administrative functions. Other costs include administrative facility costs, regulatory fees, insurance, cost for preparation for commercialization, and professional fees for legal, information technology, accounting, and consulting services.
Stock-Based Compensation.
We recognize expense for the fair value of all stock-based awards to employees, directors, and consultants in accordance with accounting principles generally accepted in the U.S. GAAP.(U.S. GAAP). For stock options, we use the Black-Scholes option valuation model in calculating the fair value of the awards.
Income Taxes.
Our effective income tax rate is the ratio of income tax expense (benefit) over our income (loss) before income taxes.
We have incurredare currently conducting a study of the availability for use of our net operating losses since inception. Our net deferred tax asset has a 100% valuation allowance asloss carryforwards and other credits under Section 382 of March 31, 2019 and December 31, 2018, as we believe it is more likely than not that the deferred tax asset will not be realized. If an ownership change, as defined under Internal Revenue Code, Section 382, occurs,and the results of this study could impact the amounts of net operating losses and other credits that we have available for use in future periods, and the timing of any of our carry-forward tax losses may be subject to limitation.
As required by ASC 740,Income Taxes, we would recognize the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting themore-likely-than-not threshold, the amount recognized in the financial statements is the largest benefit that has a greater than 50 percent likelihood of being realized upon ultimate settlement with the relevant tax authority.their use.
Recently Issued Accounting Standards.
For discussion of recently issued accounting standards, please see Note 2, “Basis of Presentation and Significant Accounting Policies,” in the interim consolidated financial statements included in this report.
Non-GAAP Financial Measures.
We prepare our consolidated financial statements and notes thereto which accompany this report in accordance with U.S. GAAP. To supplement our financial results presented on a U.S. GAAP basis, we may usenon-GAAP financial measures in our reports filed with the Commission and/or our communications with investors.Non-GAAP measures are provided as additional information and not as an alternative to our consolidated financial statements presented in accordance with GAAP. Ournon-GAAP financial measures are intended to enhance an overall understanding of our current financial performance. We believe that thenon-GAAP financial measures we present provide investors and prospective investors with an alternative method for assessing our operating results in a manner that we believe is focused on the performance of ongoing operations and provide a more consistent basis for comparison between periods.
Thenon-GAAP financial measure that we present excludes from the calculation of net income the non-cash expense associated with stock-based compensation. Further, we often reportnon-GAAP net income (loss) per share, which is calculated by dividingnon-GAAP net income (loss) by the weighted average common shares outstanding.
Anynon-GAAP financial measures that we report should not be considered in isolation or as a substitute for comparable U.S. GAAP accounting, and investors should read them in conjunction with our financial statements and notes thereto prepared in accordance with U.S. GAAP. Finally, thenon-GAAP measures of net income (loss) we may use may be different from, and not directly comparable to, similarly titled measures used by other companies.
Critical Accounting Policies and Estimates
Our discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which have been prepared in accordance with U.S. GAAP. The preparation of these consolidated financial statements requires us to make judgments, estimates, and assumptions that affect the reported amounts of assets and liabilities revenues, expenses and disclosuresthe disclosure of contingent assets and liabilities.liabilities at the date of the consolidated financial statements, as well as the reported revenues and expenses during the reporting periods. For a full discussion of our accounting policies, please refer to Note 2 on the Financial Statements included in our 20182019 Annual Report on Form10-K filed with the SEC. Our most critical accounting policies and estimates include: revenue recognition, leases, accounting for research and development expenses and stock-based compensation, measurement of fair value, income taxes, and reserves. We continually evaluate our judgments, estimates and assumptions. We base our estimates on the terms of underlying agreements, our expected course of development, historical experience and other factors that we believe are reasonable based on the circumstances, the results of which form our management’s basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates. There have been no material changes to our critical accounting policies and estimates from the information provided in Part II, Item 7,Management’s Discussion and Analysis of Financial Condition and Results of Operations included in our 20182019 Annual Report on Form10-K10-K. other than accounting policies for revenue recognition and leases as discussed in Note 2, “Basis of Presentation and Significant Accounting Policies,” in the interim consolidated financial statements included in this report.
Results of Operations
Revenues.
We had no revenues forFor the three-month periodperiods ended March 31, 2018. For the three-month period ended March 31,2020 and 2019, we recognized $29,136,472 and $12,448,438, respectively, in net revenue from product sales as we beganfrom Firdapse®. We had no revenues from our sales programcollaborative arrangement for Firdapse®.the three months ended March 31, 2020 and 2019.
Cost of Sales.
Cost of sales was approximately $4.2 million for the three months ended March 31, 2020 compared to approximately $1.7 million for the three months ended March 31, 2019 compared to $0 for the three months ended March 31, 2018. The increase in cost of sales was entirely attributable to the commercial launch of Firdapse® in January 2019. Cost of sales includesconsists principally of royalty payments which are based on net revenue as defined in the applicable license agreement. Further, cost of sales may be artificially low until we fully utilize product manufactured and recorded as expense prior to approval.FDA approval of Firdapse®.
Research and Development Expenses.
Research and development expenses for the three-month periods ended March 31, 20192020 and 20182019 were approximately $3.31$4.2 million and $3.26$3.3 million, respectively, and represented approximately 25%22.9% and 55%24.6% of total operating costs and expenses for the three-month periods ended March 31, 20192020 and 2018,2019, respectively. Research and development expenses for the three months ended March 31, 20192020 and 20182019 were as follows:
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| 2019 | 2018 | $ | % | 2020 | 2019 | $ | % | |||||||||||||||||||||||||
Research and development expenses | $ | 3,020,238 | $ | 2,964,727 | 55,511 | 1.9 | % | $ | 3,804,758 | $ | 3,020,238 | 784,520 | 26.0 | % | ||||||||||||||||||
Employee stock-based compensation | 287,721 | 294,315 | (6,594 | ) | (2.2 | %) | 418,053 | 287,721 | 130,332 | 45.3 | % | |||||||||||||||||||||
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Total research and development expenses | $ | 3,307,959 | $ | 3,259,042 | 48,917 | 1.5 | % | $ | 4,222,811 | $ | 3,307,959 | 914,852 | 27.7 | % | ||||||||||||||||||
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For the three months ended March 31, 2019,2020, research and development expenses increased $48,917approximately $0.9 million compared to the same period in 2018,2019, primarily attributable to the following:
increases in medical and regulatory affairs and quality assurance expenses and expenses from our ongoing clinical trials evaluating Firdapse® for the treatment ofMuSK-MG, CMS, and ourproof-of-concept trial evaluating Firdapse® for the treatment of SMA Type 3;
| • | increases in headcount, medical and regulatory affairs and quality assurance expenses and expenses from our ongoing clinical trials evaluating Firdapse® for the treatment ofMuSK-MG, and ourproof-of-concept trial evaluating Firdapse® for the treatment of SMA Type 3; and |
offset, in part, by a decreaseincrease of $6,594$130,332 in employee stock-based compensation which is non cashnon-cash and relates to the expense of stock options awards to certain employees.employees, due to increase in headcount.
We expect that research and development expenses will continue to be substantial in 20192020 as we continue our clinical trialsprogram evaluating Firdapse®Firdapse® for the treatment ofMuSK-MG, and CMS, continue ourproof-of-concept trial for SMA Type 3, continue our Expanded Access Program, take steps to develop a sustained release formulation of Firdapse®Firdapse®, begin to evaluate Firdapse® as a treatment for other neuromuscular diseases, and potentiallyassuming positive results from the trial, prepare ana sNDA for Firdapse®Firdapse® for the treatment ofMuSK-MGMuSK-MG. and/or CMS.
Selling, General and Administrative Expenses.
Selling, general and administrative expenses for the three months ended March 31, 20192020 and 20182019 were approximately $8.42$10.1 million and $2.67$8.4 million, respectively, and represented 63%54.6% and 45%62.6% of total operating costs and expenses for the three months ended March 31, 20192020 and 2018,2019, respectively. Selling, general and administrative expenses for the three months ended March 31, 20192020 and 20182019 were as follows:
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| 2019 | 2018 | $ | % | 2020 | 2019 | $ | % | |||||||||||||||||||||||||
Selling | $ | 5,103,908 | $ | 615,848 | 4,488,060 | 728.8 | % | $ | 5,804,145 | $ | 5,103,908 | 700,237 | 13.7 | % | ||||||||||||||||||
General and administrative | 2,666,862 | 1,381,525 | 1,285,337 | 93.0 | % | 3,157,605 | 2,666,862 | 490,743 | 18.4 | % | ||||||||||||||||||||||
Employee stock-based compensation | 645,690 | 677,025 | (31,335 | ) | (4.6 | %) | 1,101,298 | 645,690 | 455,608 | 70.6 | % | |||||||||||||||||||||
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Total selling, general and administrative expenses | $ | 8,416,460 | $ | 2,674,398 | 5,742,062 | 214.7 | % | $ | 10,063,048 | $ | 8,416,460 | 1,646,588 | 19.6 | % | ||||||||||||||||||
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For the three months ended March 31, 2019,2020, selling, general and administrative expenses increased approximately $5.74$1.6 million compared to the same period in 2018,2019, primarily attributable to the following:
increase of approximately $4.49$0.7 million in selling (commercialization) expenses, which consist primarily of commercial systems implementationthe costs hiringof our expansion of the sales force and supporting personnel, product launch costs, andthe cost of our market access and market research efforts. Commercialization expenses in 2018 werepre-commercial expenses before we began preparing our commercial program for Firdapse®;contracting with a rare-disease experienced inside sales agency;
| • | increase of approximately $0.5 million in general and administrative expenses, which is primarily due to the expansion of our operations and headcount to support our ongoing efforts to commercialize Firdapse®; and |
increase of approximately $1.29$0.5 million in general and administrative expenses primarily due to our efforts to expand our operations and headcount in connection with the commercialization of Firdapse®;
offset, in part, by a decrease of $31,335 in employee stock-based compensation which is non cash and relates to the expense of stock options awards to certain employees and directors.
We expect that selling, general and administrative costs, including commercialization costs,expenses will continue to increasebe substantial in 2019 compared with the selling, general and administrative costs incurred in 2018,future periods as we continue our efforts to sell Firdapse® and take steps that we hope will help us expand our sales activities for Firdapse®.business.
Stock-Based Compensation.
Total stock-based compensation for the three-month periods ended March 31, 2020 and 2019 were $1,519,351 and 2018 were $933,411, and $971,340, respectively. In the first quarter of 2018, employees and directors2020, grants were grantedprincipally for options relating to 2017 performance.2019year-end bonus awards. In the first quarter of 2019, most of the option grants were to new employees hired in connection with the launch of Firdapse®Firdapse®.
Other Income, Net.
We reported other income, net in all periods relating to our investment of funds received from offerings of our securities and product sales. TheFor the three months ended March 31, 2019, other income, net also included $100,000 received as part of the settlement agreement between us and Northwestern. Excluding the settlement income, the slight increase in other income, net for the three months ended March 31, 20192020 when compared to the same period in 20182019 is primarily due to higher invested balances, partly offset by lower yields on investments and higher invested balances, as we had revenues from product sales during 2019.investments. Other income, net, typically consists of interest income, dividend income and unrealized and realized gain (loss) on trading securities. These proceeds are used to fund our drug development activities and our operations. Substantially all such funds were invested in short-term interest-bearing obligations, short-term bond funds and U.S. Treasuries.
Income Taxes.
We have incurred net operating losses since inception. Forinception through the three-month periodsperiod ended March 31, 2019. Our effective income tax rate was 5.4% and 0.0% for the three months ended March 31, 2020 and 2019, respectively. Differences in the effective tax and 2018 wethe statutory federal income tax rate of 21% is driven by state income taxes and anticipated annual permanent differences, including orphan drug credit expense limitations and other items.
We had no uncertain tax positions as of March 31, 2020 and December 31, 2019. We have applied a 100%full valuation allowance againstfor our deferred tax asset as we currently believe that it is more likely than not that the deferred tax asset will not be realized.assets at March 31, 2020 and December 31, 2019.
Net Loss.Income (Loss).
Our net income was $10,426,015 for the three months ended March 31, 2020 ($0.10 per basic and diluted share) as compared to a net loss was $644,503 and $5,699,892, respectively,of ($644,503) for the three months ended March 31, 2019 and 2018 ($0.01 per basic and $0.06,diluted share).
Non-GAAP Net Income.
Ournon-GAAP net income, which excludes for the three months ended March 31, 2020 a $1,519,351 expense associated withnon-cash stock-based compensation was $11,945,366 ($0.12 and $0.11, respectively, per basic and diluted share). Ournon-GAAP net income for the three months ended March 31, 2019 was $288,908 ($0.00 per basic and diluted share), which excludesnon-cash stock-based compensation of $933,411.
Liquidity and Capital Resources
Since our inception, we have financed our operations primarily through severalmultiple public and private offering of our securities. However, duringsecurities and, since January 2019, we launched our initial product, Firdapse®, and began to receive fundsfrom revenues from product sales. At March 31, 2020, we had cash and cash equivalents and investments aggregating approximately $101.8 million and working capital of approximately $99.3 million. At December 31, 2019, we had cash and cash equivalents and investments aggregating $50.6approximately $94.5 million and working capital of $45.8approximately $87.3 million. At March 31, 2018, we had cash and cash equivalents and short-term investments aggregating $77.9 million and working capital of $76.2 million. At March 31, 2019,2020, substantially all of our cash and cash equivalents were deposited with one financial institution, and such balances were in excess of federally insured limits. Further, as of such date, substantially all such funds were invested in money markets and U.S. Treasuries.
We have to date incurred operating losses through the quarter ended March 31, 2019 and we may never become profitable. Further, wereported operating income for the first time during the three and six month periods ended June 30, 2019. We expect to continue to spend substantial dollars on our current and future drug development programs.
Based on forecasts of available cash, we believe that we have sufficient resources to support our currently anticipated operations for at least the next 12 months.months from the date of this report. There can be no assurance that we will ever becomeremain profitable and cash-flow positive or that we will be able to obtain any additional funding that we may require in the future.
WeIn the future, we may also require additional working capital to support our operations depending on our future success with Firdapse®Firdapse® sales and whether our results arecontinue to be profitable and cash flow positive. There can be no assurance as to the amount of any such funding that will be required forthesefor these purposes or whether any such funding will be available to us when it is required.
In that regard, our future funding requirements will depend on many factors, including:
the scope, rate of progress and cost of our clinical trials and other product development activities;
future clinical trial results;
the terms and timing of any collaborative, licensing and other arrangements that we may establish;
the cost and timing of regulatory approvals;
the cost and delays in product development as a result of any changes in regulatory oversight applicable to our products;
the cost and timing of establishing sales, marketing and distribution capabilities;
the level of revenues that we report from sales of Firdapse®;
| • | the level of revenues that we report from sales of Firdapse®; |
the effect of competition and market developments;
the cost of filing and potentially prosecuting, defending and enforcing any patent claims and other intellectual property rights; and
the extent to which we acquire or invest in other products.
We plan to raise additional funds that we may require in the future through public or private equity offerings, debt financings, corporate collaborations or other means. We also may seek governmental grants for a portion of the required funding for our clinical trials and preclinical trials. We may further seek to raise capital to fund additional product development efforts or product acquisitions, even if we have sufficient funds for our planned operations. Any sale by us of additional equity or convertible debt securities could result in dilution to our stockholders. There can be no assurance that any such required additional funding will be available to us at all or available on terms acceptable to us. Further, to the extent that we raise additional funds through collaborative arrangements, it may be necessary to relinquish some rights to our technologies or grant sublicenses on terms that are not favorable to us. If we are not able to secure additional funding when needed, we may have to delay, reduce the scope of or eliminate one or more research and development programs, which could have an adverse effect on our business.
On July 12, 2017, we filed a shelf registration statement with the SEC to sell up to $150 million of common stock, preferred stock, warrants to purchase common stock, debt securities and units consisting of one or more of such securities (the “2017 Shelf Registration Statement”). The 2017 Shelf Registration Statement (file no.333-219259) was declared effective by the SEC on July 26, 2017. We have completed one offering under the 2017 Shelf Registration Statement, raising net proceeds of approximately $53.8 million from the sale of 16,428,572 shares of our common stock on November 28, 2017.
On December 23, 2016, we filed a shelf registration statement with the SEC to sell up to $33.8 million of common stock (the “2016 Shelf Registration Statement”). This shelf registration statement was declared effective by the SEC on January 9, 2017. We have made no sales under the 2016 Shelf Registration Statement.
As of the date of this Form10-Q, the full amount of our 2016 Shelf Registration Statement and $92.5 million of our 2017 Shelf Registration Statement remains available for future sales.
Cash Flows.
Net cash used inprovided by (used in) operating activities was $7,962,755$7,213,078 and $6,081,450,($8,016,429), respectively, for the three-month periods ended March 31, 20192020 and 2018.2019. During the three months ended March 31, 2020 net cash provided by operating activities was primarily attributable to our net income of $10,426,015, decreases in accounts receivable of $3,618,434 and of $1,677,156 ofnon-cash expenses. This was partially offset by increases of $1,711,641 in prepaid expenses and other current andnon-current assets, $254,546 in inventory and decreases of $2,769,870 in accounts payable, $3,699,518 in accrued expenses and other liabilities and $72,952 in operating lease liability. During the three months ended March 31, 2019, net cash used in operating activities was primarily attributable to our net loss of $644,503, increases of $7,251,381 in accounts receivable, and of $231,485 in prepaid expenses and other current assets and deposits and $40,575 in inventory and decreases of $1,337,155$1,337,154 in accrued expenses and other liabilities, and $67,155$67,156 in operating lease liability. This was partially offset by increases of $602,719 in accounts payable, and $1,006,780$953,106 ofnon-cash expenses. During the three months ended March 31, 2018, net cash used in operating activities was primarily attributable to our net loss of $5,699,892, and decreases of $1,140,552 in accounts payable, and $401,277 in accrued expenses and other liabilities. This was partially offset by a $180,916 decrease in prepaid expenses and other current assets and deposits, and $979,355 of non-cash expenses. Such additionalnon-cash expenses consist of depreciation, amortization ofright-of-use asset, stock-based compensation expense, and change in right-of-use asset,accrued interest and stock-based compensation expense.accretion of discount in investments.
Net cash provided by investing activities was $10,395,719$5,000,000, for the three-month period ended March 31, 2020, consisting primarily of proceeds from maturities of investments. Net cash provided by investing activities was $10,449,393 for the three-month period ended March 31, 2019, consisting primarily of proceeds from sales and maturities of investments of $20,400,000, partially offset by purchases of investments of $9,944,974 and sales/maturities of investments of $20,346,326. Net cash used in investing activities was $31,847,693, for the three-month periods ended March 31, 2018, consisting primarily of purchases of short-term investments.$9,944,974.
Net cash provided by financing activities during the three-month periods ended March 31, 2020 and 2019 was $26,149 and 2018 was $89,350, and $33,032, respectively, consisting of proceeds from the exercise of options to purchase common stock.
Contractual Obligations.Obligations and Arrangements.
We have entered into the following contractual arrangements:
| • | Payments |
| • | Purchase commitments. We have entered into purchase commitments with our contract manufacturing organizations aggregating to approximately $950,000 per year. The agreements expire on various dates through 2024. |
| • | Employment |
| • | Lease for office |
Off-Balance Sheet Arrangements.
We currently have no debt or capitalfinance leases. We have operating leases for our office facilities. We do not have anyoff-balance sheet arrangements as such term is defined in rules promulgated by the SEC.
Caution Concerning Forward-Looking Statements
This Current Report on Form10-Q contains “forward-looking statements”, as that term is defined in the Private Securities Litigation Reform Act of 1995. These include statements regarding our expectations, beliefs, plans or objectives for future operations and anticipated results of operations. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. Without limiting the foregoing, “believes”, “anticipates”, “proposes”, “plans”, “expects”, “intends”, “may”, and other similar expressions are intended to identify forward-looking statements. Such statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or other achievements to be materially different from any future results, performanceperformances or achievements expressed or implied by such forward lookingforward-looking statements. Factors that might cause such differences include, but are not limited to, those discussed in the section entitled “Item 1A – Risk Factors” in our 2019 Annual Report on Form10-K.
The forward-looking statements made in this report are based on current expectationscontinued successful commercialization of Firdapse® and the development of additional indications for Firdapse® is highly uncertain. Factors that involve numerous risks and uncertainties, includingwill affect our success include the uncertainty of:
The impact of the effectrecent outbreak of a novel strain of coronavirus on our business and future results of operations arising fromor on the recent approval by the FDA of an NDA for Jacobus Pharmaceuticals for their version of 3,4-DAP for the treatment of pediatric LEMS patients;economy generally;
| • | Whether we will be able to continue successfully market Firdapse® while maintaining full compliance with applicable federal and state laws, rules and regulations; |
our estimates regarding anticipated capital requirements and our needs for additional financing in the future;
| • | Whether our estimates of the size of the market for Firdapse® for the treatment of Lambert-Eaton Myasthenic Syndrome (“LEMS”) will turn out to be accurate; |
the impact on Firdapse® of adverse changes in potential reimbursement and coverage policies from government and private payors such as Medicare, Medicaid, insurance companies, health maintenance organizations and other plan administrators, or the impact of pricing pressures enacted by industry organizations, the federal government or the government of any state, including as a result of increased scrutiny over pharmaceutical pricing or otherwise;
the impact on our business and results of operations of recent public statements by Senator Bernie Sanders and a vocal group of LEMS patients who object to our pricing of Firdapse®;
whether we will be able to successfully market Firdapse® while maintaining full compliance with applicable federal and state laws, rules and regulations;
whether our estimates of the size of the market for our drug candidates will turn out to be accurate;
whetherWhether we will be able to locate LEMS patients who are undiagnosed or are misdiagnosed with other diseases;
whetherWhether patients will discontinue from the use of our efforts to commercialize Firdapse® will be successful and, even if theydrug at rates that are successful, whetherhigher than historically experienced or are higher than we can become profitable;project;
whetherIf the average daily dose taken by patients changes over time, it could affect our results of operations;
| • | Whether Firdapse® patients can be successfully titrated to stable therapy; |
| • | Whether we can continue to market Firdapse® on a profitable and cash flow positive basis; |
Whether any guidance that we provide to the public market will turn out to be accurate;
Whether payors will continue to reimburse for our product at the price that we charge for the product;
The ability of our third-party suppliers and contract manufacturers to maintain compliance with current Good Manufacturing Practices (cGMP);
The ability of our distributor and the specialty pharmacies that distribute our product to maintain compliance with applicable law;
Our ability to maintain compliance with applicable rules relating to our patient assistance programs and our contributions to 501(c)(3) organizations that support LEMS patients;
| • | The scope of our intellectual property and the outcome of any future challenges or opposition to our intellectual property, and, conversely, whether any third-party intellectual property presents unanticipated obstacles for Firdapse®; |
| • | The effect on our business and future results of operations arising from the approval by the FDA of Ruzurgi® for the treatment of pediatric LEMS patients (ages 6 to under 17); |
| • | Whether our suit against the United States FDA seeking to vacate the FDA’s approval of Ruzurgi® will be successful; |
| • | Whether we can continue to compete successfully if the approval of Ruzurgi® is not overturned and Ruzurgi® continues to be prescribed foroff-label use in adult LEMS patients; |
| • | Whether, because of the lower price of Ruzurgi®, payers will require that patients tryoff-label Ruzurgi® first before they approve Firdapse® as a treatment for adult LEMS patients; |
| • | The impact on Firdapse® of adverse changes in potential reimbursement and coverage policies from government and private payors such as Medicare, Medicaid, insurance companies, health maintenance organizations and other plan administrators, or the impact of pricing pressures enacted by industry organization, the federal government or the government of any state, including as a result of increased scrutiny over pharmaceutical pricing or otherwise; |
| • | The impact on our business and results of operations of public statements by politicians and a vocal group of LEMS patients and doctors who object to our pricing of Firdapse®; |
Changes in the healthcare industry and the effect of political pressure from President Trump, Congress and/or medical professionals seeking to reduce prescription drug costs;
changesThe state of the economy generally and its impact on our business;
Changes to the healthcare industry occasioned by any future repeal and replacement of the Affordable Care Act, in laws relating to the pricing of drug products, or changes in the healthcare industry generally;
theThe scope, rate of progress and expense of our clinical trials and studies,pre-clinical studies,proof-of-concept studies, and our other drug development activities, and whether our trials and studies will be successful;
ourOur ability to complete our trials and studies on a timely basis and within the budgets we establish for such trials and studies;
whetherWhether the trials that we are currently undertaking to evaluate Firdapse® forrecent coronavirus outbreak will further affect the treatment of Anti-MuSK antibody positive myasthenia gravis(MuSK-MG), Congenital Myasthenic Syndromes (CMS), and Spinal Muscular Atrophy (SMA) Type 3, or any other trials that we undertake in the future, will be successful;
whether Firdapse® will ever be approved for the treatment ofMuSK-MG, CMS, SMA Type 3, or any other neuromuscular disease;
the resulttiming of our currently ongoing arbitration action with Northwestern University regardingclinical trials;
| • | Whether the trials that we are currently undertaking to evaluate Firdapse® for the treatment of Anti-MuSK antibody positive myasthenia gravis(MuSK-MG), and Spinal Muscular Atrophy (SMA) Type 3, or any other trials that we may undertake in the future, will be successful; |
Whether if our licenseMuSK-MG Phase 3 clinical trial is successful, the FDA will permit us to submit a supplemental new drug application (sNDA) forCPP-115;MuSK-MG without a second Phase 3 trial, and whether any such application will be accepted for filing (and even if accepted, whether such application will be approved);
| • | Whether Firdapse® will ever be approved for the treatment ofMuSK-MG, SMA Type 3, or any other neuromuscular disease; |
| • | Whether our NDS filing in Canada to commercialize Firdapse® in that jurisdiction will be approved and, even if approved for sale in Canada, whether we can successfully commercialize the product in Canada on a profitable basis; |
| • | The impact on sales of Firdapse® in the United States if an amifampridine product is purchased in Canada for use in the United States; |
| • | Whether we will be able to obtain approval to commercialize Firdapse® in Japan and what clinical trials will be required in Japan in order to obtain such marketing approval; |
| • | Whether we can successful develop, obtain approval of and successfully market a sustained release version of Firdapse®; |
| • | Whether our efforts to grow our business beyond Firdapse® through acquisitions of companies orin-licensing of product opportunities in the neuromuscular or neurology therapeutic areas will be successful; |
Whether we will have sufficient capital to finance any such acquisitions;
whetherWhether our version of generic vigabatrin tablets will ever be approved by the United States Food and Drug Administration (FDA);FDA;
evenEven if our version of vigabatrin tablets areis approved for commercialization, whether Endo Ventures/Par Pharmaceutical (our collaborator in this venture) will be successful in marketing the product; and
whether CatalystWhether we will earn milestone payments on approvalthe first commercial sale of an Abbreviated New Drug Application (ANDA) for generic vigabatrin tablets and royalties on sales of generic vigabatrin tablets;
the ability of our third-party suppliers and contract manufacturers to maintain compliance with current Good Manufacturing Practices (cGMP);
the ability of our distributor and the specialty pharmacies that distribute our product to maintain compliance with applicable law; and
our ability to maintain compliance with applicable rules relating to our patient assistance programs and our contributions to 501(c)(3) organizations that support LEMS patients.tablets.
Our current plans and objectives are based on assumptions relating to the commercialization of Firdapse®Firdapse® and the development of additional indications for Firdapse®Firdapse®. Although we believe that our assumptions are reasonable, any of our assumptions could prove inaccurate. In light of the significant uncertainties inherent in the forward-looking statements we have made herein, which reflect our views only as of the date of this report, you should not place undue reliance upon such statements. We undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
| ITEM 3. | QUANTITATIVE AND QUALITATIVE DISCLOSURE ABOUT MARKET RISK |
Not applicable.Market risk represents the risk of changes in the value of market risk-sensitive instruments caused by fluctuations in interest rates, foreign exchange rates and commodity prices. Changes in these factors could cause fluctuations in our results of operations and cash flows.
Our exposure to interest rate risk is currently confined to our cash and short-term investments that are from time to time invested in highly liquid money market funds and U.S. Treasuries. The primary objective of our investment activities is to preserve our capital to fund operations. We also seek to maximize income from our investments without assuming significant risk. We do not use derivative financial instruments in our investment portfolio. Our cash and investments policy emphasizes liquidity and preservation of principal over other portfolio considerations.
| ITEM 4. | CONTROLS AND PROCEDURES |
| a. | We have carried out an evaluation, under the supervision and with the participation of our management, including our principal executive officer and principal financial officer, of the effectiveness of the design and operation of our disclosure controls and procedures (as defined in Rules13a-15(e) and15d-15(e) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). Based on such evaluation, our principal executive officer and principal financial officer have concluded that as of March 31, |
| b. | During the three months ended March 31, |
| ITEM 1. | LEGAL PROCEEDINGS |
In 2018, we became awareRuzurgi®
We believe that certain patents grantedthe FDA’s approval of Ruzurgi® violated our statutory rights and was in multiple other respects arbitrary, capricious and contrary to Northwestern in 2018 (which patents have been licensed by Northwestern to a third-party) for a new GABA aminotransferase inhibitor were derived fromCPP-115.law. As a result, it is our positionin June 2019 we filed suit against the FDA and several related parties challenging this approval and related drug labeling. Our complaint, which was filed in the federal district court for the Southern District of Florida, alleges that Northwestern hasthe FDA’s approval of Ruzurgi® violated their license agreement with us forCPP-115 based on their failure to transfer these new patent rights to us as partmultiple provisions of the existing license agreement. It is also our position that Northwestern’s publication of information about the new patentsFDA regulations regarding labeling, resulting in misbranding in violation of the license agreement has damaged us. On October 26, 2018, we notified Northwestern that we were terminatingFederal Food, Drug, and Cosmetic Act (FDCA); violated our statutory rights to Orphan Drug Exclusivity and New Chemical Entity Exclusivity under the license agreement forCPP-115FDCA; and seeking damages for Northwestern’s breachwas in multiple other respects arbitrary, capricious, and contrary to law, in violation of the license agreement. Further, onAdministrative Procedure Act. Among other remedies, the same date, wesuit seeks an order vacating the FDA’s approval of Ruzurgi®.
We recently filed a claimmotion for damagessummary judgement in arbitration against Northwesternour case, and the FDA has filed a cross motion for Northwestern’s breaches of the license agreement.
On November 5, 2018, Northwestern advised ussummary judgement. Further, Jacobus has intervened in our case and filed their own cross-motion for summary judgement. Based on currently available information, we expect that in its view, Northwestern hasthere will be a right to terminate the license agreement with us because we allegedly breached the license agreement by failing to pay certain milestones and by allegedly failing to use commercially reasonable efforts to develop and commercialize any products. Northwestern has also advised us that, in its view, we have engaged in wrongful conduct and communications with the unrelated pharmaceutical company that licensed the new patents from Northwestern, and that such communications have damaged Northwestern’s relationship with that party. We dispute Northwestern’s allegations and intend to vigorously defend ourselves against claims that Northwestern has brought against usdecision in the arbitration proceedings.
The arbitration is currently pending and therecase sometime later this year. There can be no assurance as to the outcome of this matter.lawsuit, the timing of any decision, or the likelihood of an appeal if our suit is successful.
Additionally, fromOther Litigation
From time to time we may become involved in legal proceedings arising in the ordinary course of business. WeOther than as set forth above, we believe that there is no litigation pending at this time that could have, individually or in the aggregate, a material adverse effect on our results of operations, financial condition or cash flows.
| ITEM 1A. | RISK FACTORS |
There are many factors that affect our business, our financial condition, and the results of our operations. In addition to the information set forth in this quarterly report, you should carefully read and consider “Item 1A. Risk Factors” in Part I, and “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part II, of our 20182019 Annual Report on Form 10–K10-K filed with the SEC, which contain a description of significant factors that might cause our actual results of operations in future periods to differ materially from those currently expected or desired.
| ITEM 2. | UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS |
None
| ITEM 3. | DEFAULTS UPON SENIOR SECURITIES |
None
| ITEM 4. | MINE SAFETY DISCLOSURE |
Not applicable
| ITEM 5. | OTHER INFORMATION |
None
| 31.1 | Certification of Principal Executive Officer under Section 302 of the Sarbanes-Oxley Act of 2002 | |
| 31.2 | Certification of Principal Financial Officer under Section 302 of the Sarbanes-Oxley Act of 2002 | |
| 32.1 | Certification of Principal Executive Officer under Section 906 of the Sarbanes-Oxley Act of 2002 | |
| 32.2 | Certification of Principal Financial Officer under Section 906 of the Sarbanes-Oxley Act of 2002 | |
| 101.INS | XBRL Instance Document | |
| 101.SCH | XBRL Taxonomy Extension Schema | |
| 101.CAL | XBRL Taxonomy Extension Calculation Linkbase | |
| 101.DEF | XBRL Taxonomy Extension Definition Linkbase | |
| 101.LAB | XBRL Taxonomy Extension Label Linkbase | |
| 101.PRE | XBRL Taxonomy Extension Presentation Linkbase | |
Pursuant to the Securities Exchange Act of 1934, the Registrant has caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| Catalyst Pharmaceuticals, Inc. | ||
| By: | /s/ Alicia Grande | |
| Alicia Grande | ||
| Vice President, Treasurer and Chief Financial Officer | ||
Date: May 10, 201911, 2020
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