Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2019 | Nov. 06, 2019 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2019 | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q3 | |
Trading Symbol | ARCT | |
Entity Registrant Name | Arcturus Therapeutics Holdings Inc. | |
Entity Central Index Key | 0001768224 | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Title of 12(b) Security | Common Stock, par value $0.001 per share | |
Security Exchange Name | NASDAQ | |
Entity File Number | 001-38942 | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 32-0595345 | |
Entity Address, Address Line One | 10628 Science Center Drive | |
Entity Address, Address Line Two | Suite 250 | |
Entity Address, City or Town | San Diego | |
Entity Address, State or Province | CA | |
Entity Address, Postal Zip Code | 92121 | |
City Area Code | 858 | |
Local Phone Number | 900-2660 | |
Entity Common Stock, Shares Outstanding | 15,125,293 | |
Document Quarterly Report | true | |
Document Transition Report | false |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 74,172 | $ 36,709 |
Accounts receivable | 2,625 | 4,481 |
Prepaid expenses and other current assets | 2,611 | 638 |
Total current assets | 79,408 | 41,828 |
Property and equipment, net | 2,073 | 1,975 |
Operating lease right-of-use asset, net | 5,324 | |
Equity-method investment | 303 | 288 |
Non-current restricted cash | 107 | 107 |
Total assets | 87,215 | 44,198 |
Current liabilities: | ||
Accounts payable | 4,013 | 2,398 |
Accrued liabilities | 6,676 | 3,907 |
Deferred revenue | 10,999 | 6,272 |
Total current liabilities | 21,688 | 12,577 |
Deferred revenue, net of current portion | 14,551 | 7,534 |
Long-term debt | 10,016 | 9,911 |
Operating lease liability, net of current portion | 5,065 | |
Deferred rent | 534 | |
Total liabilities | 51,320 | 30,556 |
Stockholders’ equity | ||
Common stock: $0.001 par value; 30,000 shares authorized; 15,126 issued and outstanding at September 30, 2019; NIS 0.07 par value; 30,000 shares authorized, 10,762 issued, 10,719 outstanding and 43 held in treasury at December 31, 2018 | 15 | 214 |
Additional paid-in capital | 96,559 | 58,302 |
Accumulated deficit | (60,679) | (44,874) |
Total stockholders’ equity | 35,895 | 13,642 |
Total liabilities and stockholders’ equity | $ 87,215 | $ 44,198 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) | Sep. 30, 2019$ / sharesshares | Dec. 31, 2018₪ / sharesshares |
Statement Of Financial Position [Abstract] | ||
Common stock, par value | (per share) | $ 0.001 | ₪ 0.07 |
Common stock, shares authorized | 30,000,000 | 30,000,000 |
Treasury shares | 43,000 | |
Common stock, shares issued | 15,126,000 | 10,762,000 |
Common stock, shares outstanding | 15,126,000 | 10,719,000 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Statement Of Income And Comprehensive Income [Abstract] | ||||
Collaboration revenue | $ 3,318 | $ 3,423 | $ 17,821 | $ 8,176 |
Operating expenses: | ||||
Research and development, net | 7,053 | 3,969 | 21,646 | 12,135 |
General and administrative | 3,881 | 3,810 | 10,871 | 17,141 |
Total operating expenses | 10,934 | 7,779 | 32,517 | 29,276 |
Loss from operations | (7,616) | (4,356) | (14,696) | (21,100) |
Gain (loss) from equity-method investment | 303 | (47) | 15 | (47) |
Finance (expense) income, net | (120) | 150 | (321) | 373 |
Net loss | $ (7,433) | $ (4,253) | $ (15,002) | $ (20,774) |
Net loss per share, basic and diluted | $ (0.56) | $ (0.42) | $ (1.18) | $ (2.07) |
Weighted-average shares outstanding, basic and diluted | 13,201 | 10,093 | 12,734 | 10,059 |
Comprehensive loss: | ||||
Net loss | $ (7,433) | $ (4,253) | $ (15,002) | $ (20,774) |
Unrealized gain on short-term investments | 2 | 7 | ||
Comprehensive loss | $ (7,433) | $ (4,251) | $ (15,002) | $ (20,767) |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) shares in Thousands, $ in Thousands | Total | Common Stock [Member] | Additional Paid-In Capital [Member] | Accumulated Other Comprehensive (Loss) Gain [Member] | Accumulated Deficit [Member] |
Balance at Dec. 31, 2017 | $ 33,794 | $ 212 | $ 56,674 | $ (3) | $ (23,089) |
Balance (in shares) at Dec. 31, 2017 | 10,699 | ||||
Net loss | (20,774) | (20,774) | |||
Unrealized gain on short-term investments | 7 | 7 | |||
Share-based compensation | 753 | 753 | |||
Issuance of common stock upon exercise of stock options | 371 | $ 2 | 369 | ||
Issuance of common stock upon exercise of stock option (in shares) | 62 | ||||
Balance at Sep. 30, 2018 | 14,151 | $ 214 | 57,796 | 4 | (43,863) |
Balance (in shares) at Sep. 30, 2018 | 10,761 | ||||
Balance at Jun. 30, 2018 | 17,795 | $ 214 | 57,189 | 2 | (39,610) |
Balance (in shares) at Jun. 30, 2018 | 10,748 | ||||
Net loss | (4,253) | (4,253) | |||
Unrealized gain on short-term investments | 2 | 2 | |||
Share-based compensation | 587 | 587 | |||
Issuance of common stock upon exercise of stock options | 20 | 20 | |||
Issuance of common stock upon exercise of stock option (in shares) | 13 | ||||
Balance at Sep. 30, 2018 | 14,151 | $ 214 | 57,796 | $ 4 | (43,863) |
Balance (in shares) at Sep. 30, 2018 | 10,761 | ||||
Balance at Dec. 31, 2018 | 13,642 | $ 214 | 58,302 | (44,874) | |
Balance (in shares) at Dec. 31, 2018 | 10,762 | ||||
Net loss | (15,002) | (15,002) | |||
Treasury stock (in shares) | (43) | ||||
Share-based compensation | 1,185 | 1,185 | |||
Redomiciliation share exchange | $ (203) | 203 | |||
Issuance of restricted common stock and option, net of issuance costs | 15,545 | $ 2 | 15,543 | ||
Issuance of restricted common stock and option, net of issuance costs (in shares) | 2,400 | ||||
Issuance of common stock, net of issuance costs | 21,278 | $ 2 | 21,276 | ||
Issuance of common stock, net of issuance costs (in shares) | 1,995 | ||||
Issuance of common stock upon exercise of stock options | 50 | 50 | |||
Issuance of common stock upon exercise of stock option (in shares) | 12 | ||||
Effect of adoption of ASU 2014-09 | (803) | (803) | |||
Balance at Sep. 30, 2019 | 35,895 | $ 15 | 96,559 | (60,679) | |
Balance (in shares) at Sep. 30, 2019 | 15,126 | ||||
Balance at Jun. 30, 2019 | 21,618 | $ 13 | 74,851 | (53,246) | |
Balance (in shares) at Jun. 30, 2019 | 13,120 | ||||
Net loss | (7,433) | (7,433) | |||
Share-based compensation | 383 | 383 | |||
Issuance of common stock, net of issuance costs | 21,278 | $ 2 | 21,276 | ||
Issuance of common stock, net of issuance costs (in shares) | 1,995 | ||||
Issuance of common stock upon exercise of stock options | 49 | 49 | |||
Issuance of common stock upon exercise of stock option (in shares) | 11 | ||||
Balance at Sep. 30, 2019 | $ 35,895 | $ 15 | $ 96,559 | $ (60,679) | |
Balance (in shares) at Sep. 30, 2019 | 15,126 |
CONDENSED CONSOLIDATED STATEM_3
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2019 | Sep. 30, 2018 | |
OPERATING ACTIVITIES: | ||
Net loss | $ (15,002) | $ (20,774) |
Adjustments to reconcile net loss to net cash provided by (used in) operating activities: | ||
Depreciation and amortization | 531 | 415 |
Amortization of right-of-use operating lease asset | 544 | |
Share-based compensation expense | 1,185 | 753 |
Non-cash interest expense | 105 | |
Gain (loss) from equity-method investment | 15 | (47) |
Changes in operating assets and liabilities | ||
Accounts receivable | 1,856 | (643) |
Prepaid expense and other assets | (1,973) | 611 |
Accounts payable | 1,489 | 938 |
Accrued liabilities | 1,432 | 9 |
Deferred revenue | 10,941 | 3,647 |
Net cash provided by (used in) operating activities | 1,093 | (14,997) |
INVESTING ACTIVITIES: | ||
Proceeds from maturities of short-term investments | 30,211 | |
Purchases of short-term investments | (9,093) | |
Acquisition of property and equipment | (503) | (1,253) |
Net cash (used in) provided by investing activities | (503) | 19,865 |
FINANCING ACTIVITIES: | ||
Proceeds from the issuance of restricted common stock and option, net of issuance costs | 15,545 | |
Proceeds from the issuance of common stock, net of issuance costs | 21,278 | |
Proceeds from exercise of stock options | 50 | 332 |
Net cash provided by financing activities | 36,873 | 332 |
NET INCREASE IN CASH, CASH EQUIVALENTS AND RESTRICTED CASH | 37,463 | 5,200 |
Cash, cash equivalents and restricted cash at beginning of the period | 36,816 | 25,238 |
Cash, cash equivalents and restricted cash at end of the period | 74,279 | 30,438 |
Supplemental disclosure of cash flow information | ||
Cash paid for interest | 508 | |
Non-cash investing activities | ||
Right-of-use asset obtained in exchange for lease liabilities | 5,868 | |
Sale of intangible assets for equity investment | 590 | |
Release of repurchase liability for restricted shares | 37 | |
Purchase of property and equipment in accounts payable | $ 126 | $ 177 |
Description of Business, Basis
Description of Business, Basis of Presentation and Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2019 | |
Accounting Policies [Abstract] | |
Description of Business, Basis of Presentation and Summary of Significant Accounting Policies | Note 1. Description of Business, Basis of Presentation and Summary of Significant Accounting Policies Description of Business Arcturus Therapeutics Holdings Inc. (the “Company”) is a RNA medicines company focused on significant opportunities in rare, liver, and respiratory diseases. Management believes that the Company’s key proprietary technology has the potential to address the major hurdles in RNA development, namely the effective and safe delivery of RNA therapeutics to disease-relevant target tissues. The financial statements for periods prior to June 17, 2019 Arcturus Therapeutics Ltd. June 17, 2019 Arcturus Therapeutics Ltd Basis of Presentation The accompanying condensed consolidated financial statements include the accounts of Arcturus Therapeutics Holdings Inc. and its subsidiaries and are unaudited. All intercompany accounts and transactions have been eliminated in consolidation. These condensed consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. In management’s opinion, the accompanying condensed consolidated financial statements reflect all adjustments, consisting of normal recurring adjustments, considered necessary for a fair presentation of the results for the interim periods presented. Interim financial results are not necessarily indicative of results anticipated for the full year. These condensed consolidated financial statements should be read in conjunction with the audited financial statements and footnotes included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018. These condensed consolidated financial statements are prepared in accordance with GAAP, which requires management to make estimates and assumptions regarding the valuation of certain debt and equity instruments, the disclosure of contingent assets and liabilities at the date of the financial statements and Liquidity The Company’s activities since inception have consisted principally of performing research and development activities, general and administration activities, and raising capital. The Company’s activities are subject to significant risks and uncertainties, including failing to secure additional funding before the Company achieves sustainable revenues and profit from operations. The Company is a pre-clinical bioscience company that is dependent on obtaining external equity and debt financings to fund its operations. Historically, the Company’s primary sources of financing have been through the sale of its securities, through issuance of debt and through collaboration agreements. The Company raised $10.0 million in gross proceeds from a long-term debt agreement entered into on October 12, 2018 with Western Alliance Bank (Note 5). On June 18, 2019, the Company entered into a Third Amendment to the Research Collaboration and License Agreement with Ultragenyx Pharmaceutical Inc. (“Ultragenyx”), from which the Company received $30.0 million. Furthermore, in separate offerings, the Company raised total net proceeds of $21.4 million during the third quarter of 2019 through the sale of equity securities. Research and development activities have required significant capital investment since the Company’s inception. The Company expects its operations to continue to require cash investment to pursue the Company’s research and development activities, including preclinical studies, formulation development, clinical trials and related drug manufacturing. The Company has experienced net losses since its inception and as of September 30, 2019 has an accumulated deficit of $60.7 million. Recent Developments Arcturus Therapeutics Ltd. (“Arcturus Israel”) completed the process to redomicile from an Israeli limited company to a Delaware corporation (the “Redomiciliation”). On February 11, 2019, Arcturus Israel filed an application with the Tel-Aviv District Court (the “Israeli Court”) to approve the convening of a general shareholders meeting of Arcturus Israel for the approval of the Redomiciliation pursuant to Sections 350 and 351 of the Israeli Companies Law (the “Companies Law”). Following the Israeli Court approval dated March 13, 2019, the Company filed with the Securities and Exchange Commission a registration statement on Form S-4 (the “Form S-4”), which included a joint proxy statement/prospectus for the convening of the general shareholder meeting, held on May 17, 2019, as described in the Form S-4. The general shareholders meeting resulted in the approval of the Redomiciliation, and Arcturus Israel then approached the Israeli Court and requested its approval of the Redomiciliation. In connection with the Redomiciliation, Arcturus Israel entered into a share exchange agreement (the “Exchange Agreement”) with a special-purpose company, Arcturus Therapeutics Holdings Inc., the reporting company and parent to Arcturus Therapeutics, Inc. (“Arcturus Sub”). In furtherance of the Redomiciliation, and pursuant to the terms of the Exchange Agreement, the holders of ordinary shares of Arcturus Israel as of a future record date and the holders of options to purchase ordinary shares of Arcturus Israel as of the same record date transferred their ordinary shares of Arcturus Israel and options to purchase ordinary shares of Arcturus Israel, respectively, to the Company and, in exchange thereof, received one share of common stock of the Company for each ordinary share of Arcturus Israel and an option to purchase one share of common stock of the Company in exchange for each ordinary share of Arcturus Israel underlying the existing option to be so exchanged, respectively. As a result of the Exchange Agreement, the par value of Arcturus Israel’s ordinary shares prior to the Redomiciliation is presented in New Israeli Shekel and the par value of the Company’s common stock subsequent to the Redomiciliation is presented in United States Dollars. As of June 17, 2019, the common stock of the Company is listed on the NASDAQ Stock Market LLC (“NASDAQ”). Upon consummation of the transactions contemplated by the Share Exchange Agreement, Arcturus Israel’s ordinary shares were delisted from trading on NASDAQ, and Arcturus Israel became a private company (as defined in the Companies Law) wholly-owned by the Company. Pursuant to the Exchange Agreement, on June 12, 2019 all of the shares of Arcturus Sub were distributed to the Company and Arcturus Sub became a wholly-owned and direct subsidiary of the Company. This distribution was completed in connection with a liquidation of Arcturus Israel which was formally initiated after the redomiciliation described above. On October 30, 2019, the Company amended its loan agreement with Western Alliance Bank. See “Note 5. Long-term debt with Western Alliance Bank” See “Note 6 Stockholders’ Equity – Registered Direct Offerings” Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision-maker in making decisions regarding resource allocation and assessing performance. The Company and its chief operating decision-maker view the Company’s operations and manage its business in one operating segment, which is the research and development of medical applications for the Company’s nucleic acid-focused technology. Revenue Recognition Effective January 1, 2019, the Company adopted Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers (Topic 606) , or Topic 606, The terms of the Company’s collaborative research and development agreements include license fees, upfront payments, milestone payments when and if certain research or technology transfer milestones are achieved, development milestones and reimbursement for research and development activities, option exercise fees, other contingent payments for the achievement of defined collaboration objectives and certain preclinical, clinical, regulatory and sales-based events, as well as royalties on sales of commercialized products. Arrangements that include upfront payments are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company performs obligations under these arrangements. The Company records research funding as accounts receivable when the right to consideration is unconditional. The event-based milestone payments represent variable consideration, and the Company uses the most likely amount method to estimate this variable consideration because the Company will either receive the milestone payment or will not, which makes the potential milestone payment a binary event. The most likely amount method requires the Company to determine the likelihood of earning the milestone payment. Given the high degree of uncertainty around achievement of these milestones, the Company determines the milestone amounts to be fully constrained and does not recognize revenue until the uncertainty associated with these payments is resolved. The Company will recognize revenue from sales-based royalty payments when or as the sales occur. The Company will re-evaluate the transaction price in each reporting period as uncertain events are resolved and other changes in circumstances occur. A performance obligation is a promise in a contract to transfer a distinct good or service to the collaborative partner and is the unit of account in Topic 606. A contract’s transaction price is allocated to each distinct performance obligation based on relative standalone selling price and recognized as revenue when, or as, the performance obligation is satisfied. Under Topic 606, the Company elected to use the practical expedients permitted related to adoption, which does not require the Company to disclose certain information regarding certain remaining performance obligations as of the end of the reporting period. Topic 606 is applicable for revenue recognized in accordance with the p See “Note 2, Collaboration Revenue” Leases In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842), which requires lessees to recognize most leases on the balance sheet as lease liabilities with corresponding right-of-use assets and to disclose key information about leasing arrangements. The Company adopted Topic 842 on its effective date in the first quarter of 2019 using a modified retrospective approach. The Company elected the available package of practical expedients upon adoption, which allowed it to carry forward historical assessments of whether existing agreements contained a lease and the classification of existing operating leases. The Company continues to report its financial position as of December 31, 2018 under the former lease accounting standard (Topic 840) in the condensed consolidated balance sheet The adoption impact was due to the recognition of an operating lease liability with a corresponding right-of-use asset based on the present value of remaining minimum lease payments. A reduction of the right-of-use asset was recorded to reflect the balance of the deferred rent obligation and there was no impact to retained earnings. Research and Development, Net Research and development costs are expensed as incurred. These expenses result from the Company’s independent research and development efforts as well as efforts associated with collaboration arrangements. Research and development costs include salaries and personnel-related costs, consulting fees, fees paid for contract research and manufacturing services, the costs of laboratory supplies, equipment and facilities, preclinical studies and other external costs are shown net of any grants. Cystic Fibrosis Foundation On August 1, 2019, the Company amended its Development Program Letter Agreement, dated May 16, 2017 and as amended July 13, 2018, with the Cystic Fibrosis Foundation (“CFF”). Pursuant to the amendment, (i) CFF will increase the amount it will award to advance LUNAR-CF to $15.0 million from approximately $3.2 million, (ii) the Company will provide $5.0 million in matching funds for remaining budgeted costs, (iii) the related disbursement schedule from CFF to Arcturus will be modified such that (a) $4.0 million will be disbursed upon execution of the CFF Amendment, (b) $2.0 million will be disbursed within 30 days of the first day of each of January, April, July and October 2020 upon Arcturus invoicing CFF to meet project goals, and (c) the last payment of $3.0 million less the prior award previously paid out, equaling approximately $2.3 million, will be disbursed upon Arcturus Sub invoicing CFF to meet good manufacturing practices and opening an Investigational New Drug (“IND”) application. The funds received from CFF will be recognized as contra research and development expense in proportion to the percentage covered by CFF of the overall budget. For the three months ended September 30, 2019, the Company recognized $0.7 million of contra expense with $3.3 million remaining in accrued expenses. Statement of Cash Flows The following table provides a reconciliation of cash and cash equivalents and restricted cash reported within the condensed consolidated balance sheet to the total of the same such amounts shown in the condensed consolidated statement of cash flows: (in thousands) September 30, 2019 September 30, 2018 Cash and cash equivalents $ 74,172 $ 30,331 Non-current restricted cash 107 107 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 74,279 $ 30,438 Net Loss per Share Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding for the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding for the period determined using the treas ury-stock method. Dilutive shares of common stock are comprised of stock options. No dividends were declared or paid during the reported periods. Recently Adopted Accounting Pronouncements Revenue from Contracts with Customers In May 2014, the Financial Accounting Standards Board (FASB) issued Topic 606, which supersedes nearly all existing revenue recognition guidance under GAAP. The FASB subsequently issued amendments to Topic 606 that have the same effective date and transition date. The Company adopted this new guidance, effective January 1, 2019, using the modified retrospective transition method, in which the standard is applied as of the date of initial adoption. The Company recorded the cumulative effect of initially applying the standard as an adjustment to the opening balance of accumulated deficit. The adoption of the new revenue recognition guidance resulted in an increase of $0.8 million to deferred revenue and an increase of $0.8 million to accumulated deficit as of January 1, 2019. The change in revenue was due to a change in how the Company accounts for changes in the measure of progress and changes to the transaction price and for the recognition of revenue. Under Topic 605, the Company accounted for changes to the measure of progress and changes to the transaction price prospectively. Topic 606 requires companies to account for a change to the measure of progress or a change to the transaction price as a cumulative catch-up in the period of change. There were no other impacts upon the adoption of Topic 606. The Company will apply the standard to all new contracts initiated on or after the effective date. Leases In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842) In July 2018, the FASB issued ASU 2018-11, Leases (Topic 842): Targeted Improvements provides entities an optional transition method to apply the new guidance as of the adoption date, rather than as of the earliest period presented. In transition, entities may also elect a package of practical expedients that must be applied in its entirety to all leases commencing before the effective date, unless the lease was modified, to not reassess (a) the existence of a lease, (b) lease classification or (c) determination of initial direct costs, which effectively allows entities to carryforward accounting conclusions under previous U.S. GAAP. The Company adopted ASU 2016-02, using the optional transition method and electing the package of practical expedients described above on January 1, 2019. Due to the adoption, the Company recognized a new lease liability on the Company’s consolidated balance sheet for its operating lease of office and lab space of $6.4 million on January 1, 2019, with a corresponding right-of-use asset of $5.9 million based on the present value of the remaining minimum lease payments. See Note 8 for further discussion. |
Collaboration Revenue
Collaboration Revenue | 9 Months Ended |
Sep. 30, 2019 | |
Collaboration Agreements [Abstract] | |
Collaboration Revenue | Note 2. Collaboration Revenue The Company has entered into license agreements and collaborative research and development arrangements with pharmaceutical and biotechnology companies. Under these arrangements, the Company is entitled to receive license fees, upfront payments, milestone payments when and if certain research or technology transfer milestones are achieved, development milestones, reimbursement for research and development activities, option exercise fees, other contingent payments for the achievement of defined collaboration objectives and certain preclinical, clinical, regulatory and sales-based events, as well as royalties on sales of commercialized products. The Company’s costs of performing these services are included within research and development expense. The Company’s milestone payments are typically defined by achievement of certain preclinical, clinical, and commercial success criteria. Preclinical milestones may include in vivo proof of concept in disease animal model(s), lead candidate identification, and completion of IND-enabling studies. Clinical milestones may include successful enrollment of the first or second patient in or completion of Phase I, II, and III clinical trials, and commercial revenue is often tiered based on net or aggregate sale amounts. The Company cannot guarantee the achievement of these milestones due to risks associated with preclinical and clinical activities required for development of nucleic acid medicine-based therapeutics. The following table presents changes during the nine months ended September 30, 2019 in the balances of contract assets, including receivables from collaborative partners, and contract liabilities, including deferred revenue, as compared to what was disclosed in the Company’s Annual Report. (in thousands) Contract Assets BALANCE - December 31, 2018 $ 4,480 Additions for revenue billings 16,524 Deductions for cash collections (18,379 ) BALANCE – September 30, 2019 $ 2,625 (in thousands) Contract Liabilities BALANCE - December 31, 2018 $ 13,806 Additions for advanced billings 29,136 Additions resulting from adoption of Topic 606 803 Deductions for promised goods/services provided in current period (18,195 ) BALANCE – September 30, 2019 $ 25,550 The following table summarizes the Company’s collaboration revenues for the periods indicated (in thousands). For the Three Months Ended September 30, For the Nine Months Ended September 30, (Dollars in thousands) 2019 2018 2019 2018 Collaboration Partner - Janssen $ 966 $ 291 $ 2,101 $ 638 Collaboration Partner - Ultragenyx 922 1,789 4,854 4,495 Collaboration Partner - Takeda 224 302 518 905 Collaboration Partner - CureVac 1,057 314 6,360 772 Other 149 727 3,988 1,366 Total collaboration revenue $ 3,318 $ 3,423 $ 17,821 $ 8,176 The following paragraphs provide information regarding the nature and purpose of the Company’s most significant collaboration arrangements. Collaboration Partner – Janssen In October 2017 the Company entered into a research collaboration and license agreement with Janssen (the “2017 Agreement”). The 2017 Agreement allocated discovery, development, funding obligations, and ownership of related intellectual property among the Company and Janssen. The Company received an upfront payment of $7.7 million and may receive preclinical, development and sales milestone payments of $56.5 million, as well as royalty payments on any future licensed product sales. Janssen began reimbursing the Company for research costs during the first quarter of 2019 upon the completion of the first of three research periods. Janssen may also pay option exercise fees within the $1.0 million to $5.0 million range per target. Janssen will pay royalties on annual net sales of licensed products in the low to mid-single digits range, subject to reduction on a country-by-country and licensed-product-by-licensed-product basis and subject to certain events, such as expiration of program patents. In addition, the 2017 Agreement includes an exclusivity period. Accounting Analysis under ASC 606 In evaluating the 2017 Agreement in accordance with ASC Topic 606, the Company concluded that the contract counterparty, Janssen, is a customer. The Company identified the following promised goods/services as of the inception of the Agreement: (i) research services, (ii) license to use Arcturus technology and (iii) participation in the Joint Research Committee. The Company concluded that the promised goods/services are incapable of being distinct and consequently do not have any value on a standalone basis. Accordingly, they are determined to represent a single performance obligation. The Company concluded that Janssen’s options to select additional collaboration targets and to license rights to selected targets are not priced at a discount and therefore do not represent performance obligations for which the transaction price would be allocated. As of September 30, 2019, the remaining transaction price of $23.6 million, consisting of upfront consideration received and budgeted reimbursable out-of-pocket costs, is expected to be recognized using an input method over the remaining research period of 18 months. None of the development and commercialization milestones were included in the transaction price, as all milestone amounts were not estimated to be met, are outside the control of the Company and contingent upon success in future clinical trials and the collaborator’s efforts. Any consideration related to sales-based royalties will be recognized when the related sales occur, provided that the reported sales are reliably measurable, and the Company has no remaining promised goods/services, as such sales were determined to relate predominantly to the license granted to Janssen and therefore have also been excluded from the transaction price. For the three and nine months ended September 30, 2019, an adjustment of $0.3 million was made to the transaction price. Total deferred revenue as of September 30, 2019 and December 31, 2018 for Janssen was $6.1 million and $6.5 million, respectively. No transition adjustment was necessary upon adoption of Topic 606. Collaboration Partner – Ultragenyx In October 2015 the Company entered into a research collaboration and license agreement with Ultragenyx (the “Ultragenyx Agreement”), whereby Arcturus granted to Ultragenyx a co-exclusive license under Arcturus technology and shall be in effect only during the reserve target exclusivity term as discussed in the following paragraphs. This collaboration agreement was amended in 2017, 2018 and during the second quarter of 2019. During the initial phase of the collaboration, the Company will design and optimize therapeutics for certain rare disease targets. Ultragenyx has the option under the Ultragenyx Agreement to add additional rare disease targets during the collaborative development period. Additionally, during the collaborative development period, the Company will participate with Ultragenyx in a joint steering committee. The Ultragenyx Agreement also includes an initial exclusivity period with an option to extend this period. As part of the Ultragenyx Agreement and related amendments, Ultragenyx has paid $27.9 million in upfront fees, exclusivity extension fees and additional consideration. Ultragenyx also reimburses the Company for all internal and external development costs incurred, pursuant to the Ultragenyx Agreement, is required to make additional payments upon exercise of the Ultragenyx expansion option or exclusivity extension (if any) and if Ultragenyx achieves certain, clinical, regulatory and sales milestones, then the Company is eligible to receive royalty payments $0.5 million to $1.5 million. The current potential development, regulatory and commercial milestone payments for the existing development targets as of September 30, 2019 are $138.0 million. Ultragenyx will pay royalties as a single-digit percentage of net sales on a product-by-product and country-by-country basis during the applicable royalty term. As of September 30, 2019, the Company has not yet reached the clinical phase of the contract. On June 18, 2019, Arcturus and Ultragenyx amended the collaboration agreement for a third time (“Amendment 3”). As part of Amendment 3, the total number of targets was increased from 10 to 12, and reserve targets will be exclusively reserved for Ultragenyx with no fees for four years after execution of the amendment. An equity component was also added as part of Amendment 3 wherein Ultragenyx purchased 2.4 million shares of common stock at a premium price. Along with the equity purchase, Ultragenyx received the option to purchase 0.6 million additional shares of common stock at $16 per share within two years of executing the amendment (Note 6). The consideration received from Ultragenyx as a result of Amendment 3 was equal to $30.0 million and was comprised of a $24.0 million common stock purchase and a $6.0 million upfront payment. Specifically for Amendment 3, management determined the transaction price to be $14.4 million, comprised of $6.0 million from the upfront payment and $8.4 million from the premium paid by Ultragenyx for the purchase of Arcturus common stock. See further discussion below regarding determining the transaction price. Management determined the fair value of the premium received by using the opening stock price subsequent to execution of Amendment 3 and applying a lack of marketability discount as the shares received by Ultragenyx are restricted for two years. Accounting Analysis under ASC 606 In evaluating the Ultragenyx agreement in accordance with ASC Topic 606, the Company concluded that the contract counterparty, Ultragenyx, is a customer. The Company has identified the following promised goods/services as part of the initial agreement and subsequent amendments: (i) research services, (ii) license to use Arcturus technology, (iii) exclusivity and (iv) participation in the Joint Steering Committee. The Company concluded that the promised goods/services are incapable of being distinct and consequently do not have any value on a standalone basis. Accordingly, they are determined to represent a single performance obligation. The Company concluded that Ultragenyx’s options to extend exclusivity and options to select additional collaboration targets and to license rights to selected targets are not priced at a discount and therefore do not represent performance obligations for which the transaction price would be allocated. At September 30, 2019, the transaction price included the upfront consideration received, exclusivity extension payments and additional consideration received pursuant to Amendment 3. The Company recognizes the reimbursement of labor and expenses as costs are incurred and none of the development and commercialization milestones were included in the transaction price, as all milestone amounts were fully constrained. As part of its evaluation of the constraint, the Company considered numerous factors, including that the consideration is outside the control of the Company and contingent upon success in future clinical trials, approval from the Food and Drug Administration and the collaborator’s efforts. Any consideration related to sales-based royalties will be recognized when the related sales occur as they are constrained, provided that the reported sales are reliably measurable and the Company has no remaining promised goods/services, as such sales were determined to relate predominantly to the license granted to Ultragenyx and therefore have also been excluded from the transaction price. Amendment 3 was deemed a contract modification and accounted for as part of the original Ultragenyx Agreement and the Company recorded a cumulative catch-up adjustment of $1.1 million on the modification date. The transaction price will be recognized to revenue on a straight-line basis using an input method over the 4-year reserve target exclusivity period. The reserve target exclusivity period represents the timing over which promised goods/services Upon adoption of Topic 606, the Company reversed $0.8 million of previously recorded revenue related to Ultragenyx through an increase to deferred revenue and a decrease to beginning retained earnings. The adjustment was due to a change in the way the Company accounts for updates to the period over which revenue is recognized as well as accounting for adjustments to the transaction price. Under Topic 605, the Company accounted for these changes prospectively and under Topic 606 the Company accounts for the changes as a change in estimate recorded as a cumulative catch-up in the period in which the change occurred. Collaboration Partner – CureVac In January 2018, the Company entered into a Development and Option Agreement with CureVac, (the “Development and Option Agreement”). Under the terms of the Development and Option Agreement, the parties agreed to conduct joint preclinical development programs once CureVac makes a payment to pull down a target on the basis of which CureVac is granted options for taking a license on pre-agreed license terms to develop and commercialize certain products incorporating the Company’s patents and know-how related to delivery technology (the LUNAR® platform) (the “Arcturus Delivery Technology”), and CureVac patents and know-how related to mRNA technology. Subject to certain restrictions, the parties will have an undivided one-half interest in the patents and know-how developed jointly by the parties during the course of the Development and Option Agreement. Pursuant to the terms of the Development and Option Agreement, CureVac will have a number of target options to co-develop from a reserved target list to enter into licenses under the Arcturus Delivery Technology with respect to the development, manufacture and commercialization of licensed products (which can include products identified for development by the Company unless the Company is permitted by the terms of the Development and Option Agreement to place such products on a restricted list). A separate notice and fee will be required for each license agreement. If the target to which the license agreement relates is chosen by the parties for co-development under the Co-Development Agreement (which is defined below and discussed in the following paragraph) the license agreement will terminate as such programs will be covered under the Co-Development Agreement discussed below, and therefore CureVac will be given a credit for any exercise fees, milestone payments already paid and all other payments made in relation to the license agreement towards future such payments incurred with respect to future licenses under the Arcturus Delivery Technology. Prior to expiration of the initial term of 8 years (which was subsequently amended, as discussed below), the Agreement also includes an option to extend the term on an annual basis for up to 3 years and subject to payment by CureVac to Arcturus of a non-refundable annual extension fee. The agreement included potential milestone payments for selected targets from CureVac to the Company. The current potential milestone payment for the remaining target as of September 30, 2019 is $14.0 million for rare disease targets and $23.0 million for non-rare disease targets. CureVac will pay royalties as a percentage of net sales on a product-by-product and country-by-country basis during the applicable royalty term in the low single-digit range. As of September 30, 2019, the Company has not yet reached the clinical phase of the contract. Pursuant to a May 2018 amendment to the Development and Option Agreement (as amended and restated on September 28, 2018), the Company increased the number of targets available to CureVac under the Development and Option Agreement and agreed upon the license forms to be executed upon selection of the targets by CureVac. Concurrently with the Development and Option Agreement, the Company entered into a Co-Development and Co-Commercialization Agreement (the “Co-Development Agreement”) which the Company considered a combined contract with the Development and Option Agreement for purposes of revenue recognition. However, on February 11, 2019, the Company announced the termination of the obligations of CureVac for the preclinical development of ARCT-810, effective 180 days from February 5, 2019 and the re-assumption by the Company of the worldwide rights thereto. As a result, On July 26, 2019, the Company entered into an amendment (“CureVac Amendment”) to its Development and Option Agreement (as amended, the “Development and Option Agreement”), with CureVac, pursuant to which the Company and CureVac agreed to (i) shorten the time period during which CureVac may select potential targets to be licensed from the Company from eight years to four years, and (ii) reduce the overall number of maximum targets to be reserved and licensed. In connection with the CureVac Amendment, the Company and CureVac also entered into a Termination Agreement (the “Termination Agreement”) terminating the Co-Development Agreement between the Company and CureVac dated as of January 1, 2018. The Termination Agreement is effective as of July 26, 2019. Pursuant to the Termination Agreement, CureVac agreed to make a one-time payment to Arcturus in the amount of $4.0 million. The payment was made in July 2019. Accounting Analysis under ASC 606 In evaluating the CureVac Development and Option Agreement and Co-Development Agreement in accordance with ASC Topic 606, the Company concluded that the contract counterparty, CureVac, is a customer. The Company has identified the following promised goods/services as part of the initial agreement with CureVac and subsequent amendments: (i) research services, (ii) license to use Arcturus technology, (iii) exclusivity and (iv) participation in the Joint Steering Committee. The Company concluded that the promised goods/services are incapable of being distinct At September 30, 2019, the transaction price included the $5.0 million upfront consideration received. The Company recognizes the reimbursement of labor and expenses as costs are incurred and none of the development and commercialization milestones were included in the transaction price, as all milestone amounts were fully constrained. As part of its evaluation of the constraint, the Company considered numerous factors, including that receipt of the milestones is outside the control of the Company and contingent upon success in future clinical trials and the collaborator’s efforts. Any consideration related to sales-based royalties will be recognized when the related sales occur as they are constrained, provided that the reported sales are reliably measurable and the Company has no remaining promised goods/services, as such sales were determined to relate predominantly to the license granted to CureVac and therefore have also been excluded from the transaction price. For the three and nine months ended September 30, 2019, no adjustments were made to the transaction price. The transaction price of $5.0 million was recorded as deferred revenue in the Company’s balance sheet upon receipt and is currently being recognized as revenue on a straight-line basis using an input method over the amended forty-six month contractual term as of September 30, 2019. As a result of Amendment 3, the Company recorded a cumulative catch up adjustment of $0.4 million on the modification date, July 26, 2019. Total deferred revenue as of September 30, 2019 and December 31, 2018 for CureVac was $3.4 million and $4.4 million, respectively. No adjustment was necessary upon adoption of Topic 606 Other Collaboration Revenue The $4.0 million of other collaboration revenue for the nine months ended September 30, 2019 was primarily related to the Research and Exclusive License Agreement with Synthetic Genomics, Inc. (“SGI”) into which the Company entered during October 2017. Under the agreement, the Company granted SGI an exclusive license for the Arcturus LMD Technology to research, develop and sell products for diseases excluding all respiratory disease viruses other than influenza. Revenue related to this agreement is made up of labor reimbursements and sublicense revenue. The Company recognized a sublicense revenue amount of $3.3 million for the nine months ended September 30, 2019 whereby SGI sublicensed to multiple parties. |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | Note 3. Fair Value Measurements The Company establishes the fair value of its assets and liabilities using the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. The Company establishes a fair value hierarchy based on the inputs used to measure fair value. The three levels of the fair value hierarchy are as follows: Level 1: Quoted prices in active markets for identical assets or liabilities. Level 2: Inputs, other than the quoted prices in active markets, that are observable either directly or indirectly. Level 3: Unobservable inputs in which little or no market data exists and are therefore determined using estimates and assumptions developed by the Company, which reflect those that a market participant would use. The carrying value of cash, restricted cash, accounts receivable, accounts payable, and accrued liabilities approximate their respective fair values due to their relative short maturities. The carrying amounts of long-term debt for the amount drawn on the Company’s debt facility approximates fair value as the interest rate is variable and reflects current market rates. As of September 30, 2019 and December 31, 2018, all assets measured at fair value on a recurring basis consisted of cash equivalents, money market funds, which were classified within Level 1 of the fair value hierarchy. The fair value of these financial instruments was measured based on quoted prices. |
Balance Sheet Details
Balance Sheet Details | 9 Months Ended |
Sep. 30, 2019 | |
Balance Sheet Related Disclosures [Abstract] | |
Balance Sheet Details | Note 4 Prepaid expenses and other current assets consisted of the following as of September 30, 2019 and December 31, 2018: (in thousands) September 30, 2019 December 31, 2018 Prepaid expenses $ 2,578 $ 546 Other current assets 33 92 Total $ 2,611 $ 638 Property and equipment, net for the same periods consisted of the following: (in thousands) September 30, 2019 December 31, 2018 Research equipment $ 3,235 $ 2,711 Computers and software 271 200 Office equipment and furniture 561 527 Leasehold improvements 34 34 Total 4,101 3,472 Less accumulated depreciation and amortization (2,028 ) (1,497 ) Property and equipment, net $ 2,073 $ 1,975 Depreciation and amortization expense was $0.2 million and $0.2 million Accrued liabilities consisted of the following as of September 30, 2019 and December 31, 2018: (in thousands) September 30, 2019 December 31, 2018 Accrued compensation $ 1,979 $ 974 Cystic Fibrosis Foundation Liability (Note 1) 3,336 — Refundable fees received — 2,259 Current portion of operating lease liability 801 — Other accrued liabilities 560 674 Total $ 6,676 $ 3,907 |
Debt
Debt | 9 Months Ended |
Sep. 30, 2019 | |
Debt Disclosure [Abstract] | |
Debt | Note 5. Debt Long-term debt with Western Alliance Bank On October 12, 2018, the Company entered into a Loan and Security Agreement with Western Alliance Bank (the “Bank”) The Company paid a loan origination fee of $128,000 which was recorded as a debt discount and is being accreted over the term of the Loan. In addition, the Company is required to pay a fee of $350,000 upon certain change of control events. Upon maturity or prepayment, the Company will be required to pay a 3% fee, or a 2% fee if the U.S. Food and Drug Administration accepts certain IND applications prior to maturity. Because acceptance of an IND is outside of the Company’s control, management estimated that the Company will be liable for a fee of 3% of the principal balance, or $300,000 upon repayment or maturity, and such fee is accreted to the debt balance using the effective interest method over the term of the Loan. The Loan is collateralized by all of the assets of the Company, excluding intellectual property, which is subject to a negative pledge. The Loan contains customary conditions of borrowing, events of default and covenants, including covenants that restrict the Company’s ability to dispose of assets, merge with or acquire other entities, incur indebtedness and make distributions to holders of the Company’s capital stock. In addition, the Company is required to maintain at least 50% of its deposit and investment accounts, or $20 million, whichever is lower, with Western Alliance Bank. The Loan also includes covenants which include the Company’s (1) nomination of a clinical candidate by December 31, 2018, which the Company is in compliance with, and (2) submission of a clinical candidate for IND application, made to the U.S. Food and Drug Administration by December 31, 2019 and have it approved by January 31, 2020, provided that, if the Company has received net cash proceeds from sale, on or after October 12, 2018, of the Company’s equity securities in an amount of not less than $15,000,000, then the IND submission date shall be extended to May 31, 2020 and the approval date shall be extended to June 30, 2020. As a result of the equity purchase by Ultragenyx of 2.4 million shares of common stock (previously discussed at Note 2), the IND submission date and approval date have been extended to May 31, 2020 and June 30, 2020, respectively. Should an event of default occur, including the occurrence of a material adverse effect, the Company could be liable for immediate repayment of all obligations under the Loan. As of September 30, 2019, the Company is in compliance with all covenants and conditions of the Loan. On October 30, 2019, Arcturus Therapeutics, Inc. (the “Borrower”), a wholly-owned subsidiary of the Company and the Bank entered into a Third Amendment (the “Third Amendment”) to the Loan and Security Agreement dated as of October 12, 2018 (as amended, the “Loan Agreement”). Pursuant to the amendment, the Bank agreed to make a term loan to the Company on October 30, 2019, in the amount of $15 million (the “Term Loan”). After repaying $10 million for the Loan the Company owed to the Bank, the resulting net increase in the indebtedness of the Company was $5 million. The Term Loan bears interest at a floating rate ranging from 1.25% to 2.75% above the prime rate. The amendment further provides that the Term Loan has a maturity date of October 30, 2023. The Company shall make monthly payments of interest only until the interest-only end date of April 1, 2021, which is subject to extension to October 1, 2021 upon the occurrence of an equity or expansion event and the absence of an event of default, and thereafter shall make monthly payments of principal and interest during a 30-month amortization period. The Term Loan may be prepaid in full at any time, provided that a prepayment fee is required to be paid by the Company upon prepayment. The prepayment fee ranges from 0.50% to 2.00% of the prepaid principal amount depending upon the date on which the prepayment is made. In connection with the Third Amendment, the Company guaranteed the Borrower’s obligations under the Loan Agreement and pledged substantially all of its assets as security under the Loan Agreement. The amendment also modified the Company’s covenants to extend the dates by which a selected IND must be submitted and by which the U.S. Food and Drug Administration or equivalent authority must accept the IND, and also required the Company to maintain the lesser of (i) all of its total consolidated and unrestricted cash in total deposits with the Bank or (ii) $15 million in total deposits with the Bank. |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 2019 | |
Stockholders Equity Note [Abstract] | |
Stockholders' Equity | Note 6. Stockholders’ Equity Registered Direct Offerings In July and September of 2019, the Company entered into engagement letters with H.C. Wainwright & Co., LLC (the “Placement Agent”) relating to registered direct offerings of common stock to certain institutional investors. Pursuant to each letter agreement, the Company agreed to pay the Placement Agent a cash fee of 6.0% of the gross proceeds from each offering and reimburse the cost of expenses. In connection therewith, on August 1, 2019, August 2, 2019 and September 26, 2019, the Company and certain Investors entered into securities purchase agreements relating to the issuance and sale of shares of common stock. The purchase price per share for each share offered in the offerings was $11.50. The aggregate gross proceeds of the offerings were $23.0 million for an aggregate of 1,995,653 shares of common stock. The net proceeds to the Company from the Offering, after deducting Placement Agent fees and the Company’s estimated offering expenses, were approximately $21.3 million. The Offerings closed on August 5, 2019 and September 30, 2019, respectively. The offer and sale of the common stock was registered under the Securities Act of 1933, as amended (the “Securities Act”), on the Company’s Registration Statement on Form S-3 (Registration No. 333-232281), previously filed with the Securities and Exchange Commission and declared effective on July 29, 2019. Equity Purchase Agreement On June 18, 2019, the Company entered into an Equity Purchase Agreement (the “Expanded Ultragenyx Agreement”) with Ultragenyx. Pursuant to the terms of the Expanded Ultragenyx Agreement, the Company sold an aggregate of 2,400,000 shares of common stock, par value $0.001 per share (“Common Stock”) at a price of $10.00 per share to Ultragenyx on June 19, 2019. Pursuant to the Expanded Ultragenyx Agreement, the Company also granted Ultragenyx a two-year option (the “Option”) to purchase up to 600,000 additional shares of Common Stock at a price of $16.00 per share. The Option to purchase additional shares of Common Stock may not be exercised if Ultragenyx’s ownership of the Company’s common stock would exceed 19.99% of the Company’s total shares outstanding following such exercise. The option was recorded as a component of stockholders’ equity within additional paid-in capital. Pursuant to the terms of the Expanded Ultragenyx Agreement, until the later of (i) the first anniversary of the closing date or (ii) the date on which Ultragenyx beneficially owns less than 8.0% of the total voting power of the Company, at each annual stockholders meeting or any stockholders meeting at which members of the board of directors (the “Board”) are to be elected, the Company must nominate one director designated by Ultragenyx (the “Ultragenyx Designee”). Additionally, the Ultragenyx Designee has the contractual right to be appointed to all Board committees (subject to applicable NASDAQ rules). Ultragenyx also has the right to have a designee attend Board meetings as a non-voting observer. In connection with the Expanded Ultragenyx Agreement, the Company and Ultragenyx entered into a Registration Rights Agreement (the “Registration Rights Agreement”). The Registration Rights Agreement requires the Company to file a registration statement providing for the resale of the shares within 180 days of June 18, 2019, and provides Ultragenyx with certain “piggy-back” registration rights with respect to registration statements that the Company may file. Restricted Common Shares In March 2013, the founders of the Company purchased 2,783,686 shares of common stock for $0.0068 per share. Of the shares purchased, 1,538,353 were subject to a repurchase option whereby the Company has an option for two months after date of termination of service to repurchase any or all of the unvested shares at the original purchase price per share. The repurchase option shall be deemed to be automatically exercised by the Company as of the end of the two-month period unless the Company notifies the purchaser that it does not intend to exercise its option. The shares will be vested (1) 25% after obtaining suitable siRNA license; (2) 25% after in vivo 622,667 Net Loss per Share Dilutive securities that were not included in the calculation of diluted net loss per share for the three and nine months ended September 30, 2019 as they were anti-dilutive totaled 166,921 and 96,615, respectively, and 98,000 and 91,000 for the three and nine months ended September 30, 2018, respectively. For the three and nine months ended September 30, 2019 and 2018, the calculation of the weighted-average number of shares outstanding excludes unvested restricted shares of common stock of 622,667. |
Share-Based Compensation
Share-Based Compensation | 9 Months Ended |
Sep. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Share-Based Compensation | Note 7. Share-Based Compensation In August 2018, the Company adopted the 2018 Omnibus Equity Incentive Plan (“2018 Plan”). Under the 2018 Plan, the Company is authorized to issue up to a maximum of Stock Options Share-based compensation expenses included in the Company’s condensed statements of operations and comprehensive loss for the three and nine months ended September 30, 2019 and 2018 were: For the Three Months Ended September 30, For the Nine Months Ended September 30, (in thousands) 2019 2018 2019 2018 Research and development $ 158 $ 286 $ 466 $ 315 General and administrative 225 301 719 438 Total $ 383 $ 587 $ 1,185 $ 753 Share-based compensation expense for the three and nine months ended September 30, 2019 excludes expense related to options granted to the Chief Executive Officer and Chief Financial Officer as the option grants are subject to shareholder approval which was ratified at the Company’s annual meeting on October 25, 2019. |
Leases
Leases | 9 Months Ended |
Sep. 30, 2019 | |
Leases [Abstract] | |
Leases | Note 8. Leases In October 2017, the Company entered into a non-cancellable operating lease agreement for office space adjacent to its previously occupied headquarters. The commencement of the lease began in March 2018 and the lease extends for approximately 84 months from the commencement date with a remaining lease term of six years. Monthly rental payments are due under the lease and there are escalating rent payments during the term of the lease. The Company is also responsible for its proportional share of operating expenses of the building and common areas. In conjunction with the new lease, the Company received free rent for four months and received a tenant improvement allowance of $74,000. The lease may be extended for one five-year period at the then current market rate with annual escalations; however, the Company deemed the extension option not reasonably certain to be exercised and therefore excluded the option from the lease terms. The Company entered into an irrevocable standby letter of credit with the landlord for a security deposit of $96,000 upon executing the lease which is included (along with additional funds required to secure the letter of credit) in the balance of non-current restricted cash. Operating lease right-of-use asset and liability on the condensed consolidated balance sheets represent the present value of remaining lease payments over the remaining lease terms. The Company does not allocate lease payments to non-lease components; therefore, payments for common-area-maintenance and administrative services are not included in the operating lease right-of-use asset and liability. The Company uses its incremental borrowing rate to calculate the present value of the lease payments, as the implicit rate in the lease is not readily determinable. As of September 30, 2019, the payments of the operating lease liability were as follows: (in thousands) Remaining Lease Payments 2019 (remaining) $ 311 2020 1,272 2021 1,310 2022 1,349 2023 1,390 Thereafter 1,745 Total remaining lease payments 7,377 Less: imputed interest (1,511 ) Total operating lease liabilities $ 5,866 Weighted-average remaining lease term 6 years Weighted-average discount rate 8.4 % Operating lease costs consist of the fixed lease payments included in operating lease liability and are recorded on a straight-line basis over the lease terms. Operating lease costs were $0.3 million and $0.9 million for the three and nine months ended September 30, 2019. |
Related Party Transactions
Related Party Transactions | 9 Months Ended |
Sep. 30, 2019 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | Note 9. Related Party Transactions Ultragenyx On June 17, 2019, Arcturus and Ultragenyx executed Amendment 3. In addition, as a result of the Expanded Ultragenyx Agreement, Ultragenyx owns 15.9% of the outstanding common stock of the Company as of September 30, 2019. For the three and nine months ended September 30, 2019, the Company has recognized revenue of $0.9 million and $4.9 million, respectively, and for the three and nine months ended September 30, 2018, the Company recognized revenue of $1.8 million and $4.5 million. As of September 30, 2019 and 2018, the Company holds accounts receivable balances of negligible amounts. (Note 2) Providence In March 2016, the Company entered into a Research Collaboration and License Agreement with a related party, Providence, whose CEO and President is also a shareholder of the Company, to identify and optimize microRNA modulators or mimetics for the treatment of neoplastic diseases. In April 2017, the Providence Agreement was amended to include mRNA for the treatment of neoplastic disease. In July 2018, the Providence Agreement was amended and restated to cover brain neoplasms, breast neoplasms and ovarian neoplasms. Each party is responsible for their own research costs under the agreement, and Providence is responsible for all development costs through the completion of Phase 2 clinical trials. The Company is entitled to share in future product revenue of each product provided the Company shares in the product’s post Phase 2 costs. Separately, Providence has agreed to pay for FTEs at a specified rate. For the three and nine months ended September 30, 2019, the Company has recognized revenue of Equity-Method Investment In June 2018, the Company completed the sale of its intangible asset related to the ADAIR technology. Pursuant to the asset purchase agreement for ADAIR, the Company received a 30% ownership interest in the common stock of a privately held company in consideration for the sale of the ADAIR technology. As this ownership interest is greater than 20% and one executive of the Company holds a seat on the investee’s board of directors, the Company has the ability to exercise significant influence over the operating and financial policies of this investee; therefore, the Company accounts for this investment as an equity-method investment. The Company has no requirement to invest further in this private company. The Company has recorded $0.6 million of its share of losses of the equity method investee leaving no equity investment balance as of June 30, 2019. During the third quarter of 2019, the equity method investee issued shares of its common stock at a share price greater than the initial investment which resulted in the Company recording a gain in its equity method investment. The gain was partly offset by additional losses incurred by the equity method investee resulting in a net gain of $0.3 million recorded as of September 30, 2019. |
Subsequent Events
Subsequent Events | 9 Months Ended |
Sep. 30, 2019 | |
Subsequent Events [Abstract] | |
Subsequent Events | Note 10. Subsequent Events Amendment to Long-term debt with Western Alliance Bank See discussion of amendment at Note 5. |
Description of Business, Basi_2
Description of Business, Basis of Presentation and Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2019 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying condensed consolidated financial statements include the accounts of Arcturus Therapeutics Holdings Inc. and its subsidiaries and are unaudited. All intercompany accounts and transactions have been eliminated in consolidation. These condensed consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. In management’s opinion, the accompanying condensed consolidated financial statements reflect all adjustments, consisting of normal recurring adjustments, considered necessary for a fair presentation of the results for the interim periods presented. Interim financial results are not necessarily indicative of results anticipated for the full year. These condensed consolidated financial statements should be read in conjunction with the audited financial statements and footnotes included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018. These condensed consolidated financial statements are prepared in accordance with GAAP, which requires management to make estimates and assumptions regarding the valuation of certain debt and equity instruments, the disclosure of contingent assets and liabilities at the date of the financial statements and |
Liquidity | Liquidity The Company’s activities since inception have consisted principally of performing research and development activities, general and administration activities, and raising capital. The Company’s activities are subject to significant risks and uncertainties, including failing to secure additional funding before the Company achieves sustainable revenues and profit from operations. The Company is a pre-clinical bioscience company that is dependent on obtaining external equity and debt financings to fund its operations. Historically, the Company’s primary sources of financing have been through the sale of its securities, through issuance of debt and through collaboration agreements. The Company raised $10.0 million in gross proceeds from a long-term debt agreement entered into on October 12, 2018 with Western Alliance Bank (Note 5). On June 18, 2019, the Company entered into a Third Amendment to the Research Collaboration and License Agreement with Ultragenyx Pharmaceutical Inc. (“Ultragenyx”), from which the Company received $30.0 million. Furthermore, in separate offerings, the Company raised total net proceeds of $21.4 million during the third quarter of 2019 through the sale of equity securities. Research and development activities have required significant capital investment since the Company’s inception. The Company expects its operations to continue to require cash investment to pursue the Company’s research and development activities, including preclinical studies, formulation development, clinical trials and related drug manufacturing. The Company has experienced net losses since its inception and as of September 30, 2019 has an accumulated deficit of $60.7 million. |
Recent Developments | Recent Developments Arcturus Therapeutics Ltd. (“Arcturus Israel”) completed the process to redomicile from an Israeli limited company to a Delaware corporation (the “Redomiciliation”). On February 11, 2019, Arcturus Israel filed an application with the Tel-Aviv District Court (the “Israeli Court”) to approve the convening of a general shareholders meeting of Arcturus Israel for the approval of the Redomiciliation pursuant to Sections 350 and 351 of the Israeli Companies Law (the “Companies Law”). Following the Israeli Court approval dated March 13, 2019, the Company filed with the Securities and Exchange Commission a registration statement on Form S-4 (the “Form S-4”), which included a joint proxy statement/prospectus for the convening of the general shareholder meeting, held on May 17, 2019, as described in the Form S-4. The general shareholders meeting resulted in the approval of the Redomiciliation, and Arcturus Israel then approached the Israeli Court and requested its approval of the Redomiciliation. In connection with the Redomiciliation, Arcturus Israel entered into a share exchange agreement (the “Exchange Agreement”) with a special-purpose company, Arcturus Therapeutics Holdings Inc., the reporting company and parent to Arcturus Therapeutics, Inc. (“Arcturus Sub”). In furtherance of the Redomiciliation, and pursuant to the terms of the Exchange Agreement, the holders of ordinary shares of Arcturus Israel as of a future record date and the holders of options to purchase ordinary shares of Arcturus Israel as of the same record date transferred their ordinary shares of Arcturus Israel and options to purchase ordinary shares of Arcturus Israel, respectively, to the Company and, in exchange thereof, received one share of common stock of the Company for each ordinary share of Arcturus Israel and an option to purchase one share of common stock of the Company in exchange for each ordinary share of Arcturus Israel underlying the existing option to be so exchanged, respectively. As a result of the Exchange Agreement, the par value of Arcturus Israel’s ordinary shares prior to the Redomiciliation is presented in New Israeli Shekel and the par value of the Company’s common stock subsequent to the Redomiciliation is presented in United States Dollars. As of June 17, 2019, the common stock of the Company is listed on the NASDAQ Stock Market LLC (“NASDAQ”). Upon consummation of the transactions contemplated by the Share Exchange Agreement, Arcturus Israel’s ordinary shares were delisted from trading on NASDAQ, and Arcturus Israel became a private company (as defined in the Companies Law) wholly-owned by the Company. Pursuant to the Exchange Agreement, on June 12, 2019 all of the shares of Arcturus Sub were distributed to the Company and Arcturus Sub became a wholly-owned and direct subsidiary of the Company. This distribution was completed in connection with a liquidation of Arcturus Israel which was formally initiated after the redomiciliation described above. On October 30, 2019, the Company amended its loan agreement with Western Alliance Bank. See “Note 5. Long-term debt with Western Alliance Bank” See “Note 6 Stockholders’ Equity – Registered Direct Offerings” |
Segment Information | Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision-maker in making decisions regarding resource allocation and assessing performance. The Company and its chief operating decision-maker view the Company’s operations and manage its business in one operating segment, which is the research and development of medical applications for the Company’s nucleic acid-focused technology. |
Revenue Recognition | Revenue Recognition Effective January 1, 2019, the Company adopted Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers (Topic 606) , or Topic 606, The terms of the Company’s collaborative research and development agreements include license fees, upfront payments, milestone payments when and if certain research or technology transfer milestones are achieved, development milestones and reimbursement for research and development activities, option exercise fees, other contingent payments for the achievement of defined collaboration objectives and certain preclinical, clinical, regulatory and sales-based events, as well as royalties on sales of commercialized products. Arrangements that include upfront payments are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company performs obligations under these arrangements. The Company records research funding as accounts receivable when the right to consideration is unconditional. The event-based milestone payments represent variable consideration, and the Company uses the most likely amount method to estimate this variable consideration because the Company will either receive the milestone payment or will not, which makes the potential milestone payment a binary event. The most likely amount method requires the Company to determine the likelihood of earning the milestone payment. Given the high degree of uncertainty around achievement of these milestones, the Company determines the milestone amounts to be fully constrained and does not recognize revenue until the uncertainty associated with these payments is resolved. The Company will recognize revenue from sales-based royalty payments when or as the sales occur. The Company will re-evaluate the transaction price in each reporting period as uncertain events are resolved and other changes in circumstances occur. A performance obligation is a promise in a contract to transfer a distinct good or service to the collaborative partner and is the unit of account in Topic 606. A contract’s transaction price is allocated to each distinct performance obligation based on relative standalone selling price and recognized as revenue when, or as, the performance obligation is satisfied. Under Topic 606, the Company elected to use the practical expedients permitted related to adoption, which does not require the Company to disclose certain information regarding certain remaining performance obligations as of the end of the reporting period. Topic 606 is applicable for revenue recognized in accordance with the p See “Note 2, Collaboration Revenue” |
Leases | Leases In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842), which requires lessees to recognize most leases on the balance sheet as lease liabilities with corresponding right-of-use assets and to disclose key information about leasing arrangements. The Company adopted Topic 842 on its effective date in the first quarter of 2019 using a modified retrospective approach. The Company elected the available package of practical expedients upon adoption, which allowed it to carry forward historical assessments of whether existing agreements contained a lease and the classification of existing operating leases. The Company continues to report its financial position as of December 31, 2018 under the former lease accounting standard (Topic 840) in the condensed consolidated balance sheet The adoption impact was due to the recognition of an operating lease liability with a corresponding right-of-use asset based on the present value of remaining minimum lease payments. A reduction of the right-of-use asset was recorded to reflect the balance of the deferred rent obligation and there was no impact to retained earnings. |
Research and Development, net | Research and Development, Net Research and development costs are expensed as incurred. These expenses result from the Company’s independent research and development efforts as well as efforts associated with collaboration arrangements. Research and development costs include salaries and personnel-related costs, consulting fees, fees paid for contract research and manufacturing services, the costs of laboratory supplies, equipment and facilities, preclinical studies and other external costs are shown net of any grants. Cystic Fibrosis Foundation On August 1, 2019, the Company amended its Development Program Letter Agreement, dated May 16, 2017 and as amended July 13, 2018, with the Cystic Fibrosis Foundation (“CFF”). Pursuant to the amendment, (i) CFF will increase the amount it will award to advance LUNAR-CF to $15.0 million from approximately $3.2 million, (ii) the Company will provide $5.0 million in matching funds for remaining budgeted costs, (iii) the related disbursement schedule from CFF to Arcturus will be modified such that (a) $4.0 million will be disbursed upon execution of the CFF Amendment, (b) $2.0 million will be disbursed within 30 days of the first day of each of January, April, July and October 2020 upon Arcturus invoicing CFF to meet project goals, and (c) the last payment of $3.0 million less the prior award previously paid out, equaling approximately $2.3 million, will be disbursed upon Arcturus Sub invoicing CFF to meet good manufacturing practices and opening an Investigational New Drug (“IND”) application. The funds received from CFF will be recognized as contra research and development expense in proportion to the percentage covered by CFF of the overall budget. For the three months ended September 30, 2019, the Company recognized $0.7 million of contra expense with $3.3 million remaining in accrued expenses. |
Statement of Cash Flows | Statement of Cash Flows The following table provides a reconciliation of cash and cash equivalents and restricted cash reported within the condensed consolidated balance sheet to the total of the same such amounts shown in the condensed consolidated statement of cash flows: (in thousands) September 30, 2019 September 30, 2018 Cash and cash equivalents $ 74,172 $ 30,331 Non-current restricted cash 107 107 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 74,279 $ 30,438 |
Net Loss per Share | Net Loss per Share Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding for the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding for the period determined using the treas ury-stock method. Dilutive shares of common stock are comprised of stock options. No dividends were declared or paid during the reported periods. |
Recently Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements Revenue from Contracts with Customers In May 2014, the Financial Accounting Standards Board (FASB) issued Topic 606, which supersedes nearly all existing revenue recognition guidance under GAAP. The FASB subsequently issued amendments to Topic 606 that have the same effective date and transition date. The Company adopted this new guidance, effective January 1, 2019, using the modified retrospective transition method, in which the standard is applied as of the date of initial adoption. The Company recorded the cumulative effect of initially applying the standard as an adjustment to the opening balance of accumulated deficit. The adoption of the new revenue recognition guidance resulted in an increase of $0.8 million to deferred revenue and an increase of $0.8 million to accumulated deficit as of January 1, 2019. The change in revenue was due to a change in how the Company accounts for changes in the measure of progress and changes to the transaction price and for the recognition of revenue. Under Topic 605, the Company accounted for changes to the measure of progress and changes to the transaction price prospectively. Topic 606 requires companies to account for a change to the measure of progress or a change to the transaction price as a cumulative catch-up in the period of change. There were no other impacts upon the adoption of Topic 606. The Company will apply the standard to all new contracts initiated on or after the effective date. Leases In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842) In July 2018, the FASB issued ASU 2018-11, Leases (Topic 842): Targeted Improvements provides entities an optional transition method to apply the new guidance as of the adoption date, rather than as of the earliest period presented. In transition, entities may also elect a package of practical expedients that must be applied in its entirety to all leases commencing before the effective date, unless the lease was modified, to not reassess (a) the existence of a lease, (b) lease classification or (c) determination of initial direct costs, which effectively allows entities to carryforward accounting conclusions under previous U.S. GAAP. The Company adopted ASU 2016-02, using the optional transition method and electing the package of practical expedients described above on January 1, 2019. Due to the adoption, the Company recognized a new lease liability on the Company’s consolidated balance sheet for its operating lease of office and lab space of $6.4 million on January 1, 2019, with a corresponding right-of-use asset of $5.9 million based on the present value of the remaining minimum lease payments. See Note 8 for further discussion. |
Description of Business, Basi_3
Description of Business, Basis of Presentation and Summary of Significant Accounting Policies (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Accounting Policies [Abstract] | |
Schedule of Cash and Cash Equivalents and Restricted Cash | The following table provides a reconciliation of cash and cash equivalents and restricted cash reported within the condensed consolidated balance sheet to the total of the same such amounts shown in the condensed consolidated statement of cash flows: (in thousands) September 30, 2019 September 30, 2018 Cash and cash equivalents $ 74,172 $ 30,331 Non-current restricted cash 107 107 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 74,279 $ 30,438 |
Collaboration Revenue (Tables)
Collaboration Revenue (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Collaboration Agreements [Abstract] | |
Summary of Changes in Balances of Contract Assets and Contract Liability | The following table presents changes during the nine months ended September 30, 2019 in the balances of contract assets, including receivables from collaborative partners, and contract liabilities, including deferred revenue, as compared to what was disclosed in the Company’s Annual Report. (in thousands) Contract Assets BALANCE - December 31, 2018 $ 4,480 Additions for revenue billings 16,524 Deductions for cash collections (18,379 ) BALANCE – September 30, 2019 $ 2,625 (in thousands) Contract Liabilities BALANCE - December 31, 2018 $ 13,806 Additions for advanced billings 29,136 Additions resulting from adoption of Topic 606 803 Deductions for promised goods/services provided in current period (18,195 ) BALANCE – September 30, 2019 $ 25,550 |
Summary of Collaboration Revenue | The following table summarizes the Company’s collaboration revenues for the periods indicated (in thousands). For the Three Months Ended September 30, For the Nine Months Ended September 30, (Dollars in thousands) 2019 2018 2019 2018 Collaboration Partner - Janssen $ 966 $ 291 $ 2,101 $ 638 Collaboration Partner - Ultragenyx 922 1,789 4,854 4,495 Collaboration Partner - Takeda 224 302 518 905 Collaboration Partner - CureVac 1,057 314 6,360 772 Other 149 727 3,988 1,366 Total collaboration revenue $ 3,318 $ 3,423 $ 17,821 $ 8,176 |
Balance Sheet Details (Tables)
Balance Sheet Details (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Balance Sheet Related Disclosures [Abstract] | |
Schedule of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consisted of the following as of September 30, 2019 and December 31, 2018: (in thousands) September 30, 2019 December 31, 2018 Prepaid expenses $ 2,578 $ 546 Other current assets 33 92 Total $ 2,611 $ 638 |
Summary of Components of Property and Equipment, Net | Property and equipment, net for the same periods consisted of the following: (in thousands) September 30, 2019 December 31, 2018 Research equipment $ 3,235 $ 2,711 Computers and software 271 200 Office equipment and furniture 561 527 Leasehold improvements 34 34 Total 4,101 3,472 Less accumulated depreciation and amortization (2,028 ) (1,497 ) Property and equipment, net $ 2,073 $ 1,975 |
Schedule of Accrued Liabilities | Accrued liabilities consisted of the following as of September 30, 2019 and December 31, 2018: (in thousands) September 30, 2019 December 31, 2018 Accrued compensation $ 1,979 $ 974 Cystic Fibrosis Foundation Liability (Note 1) 3,336 — Refundable fees received — 2,259 Current portion of operating lease liability 801 — Other accrued liabilities 560 674 Total $ 6,676 $ 3,907 |
Share-Based Compensation (Table
Share-Based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Schedule of Share-based Compensation Expenses | Share-based compensation expenses included in the Company’s condensed statements of operations and comprehensive loss for the three and nine months ended September 30, 2019 and 2018 were: For the Three Months Ended September 30, For the Nine Months Ended September 30, (in thousands) 2019 2018 2019 2018 Research and development $ 158 $ 286 $ 466 $ 315 General and administrative 225 301 719 438 Total $ 383 $ 587 $ 1,185 $ 753 |
Leases (Tables)
Leases (Tables) | 9 Months Ended |
Sep. 30, 2019 | |
Leases [Abstract] | |
Payments of Operating Lease Liability | As of September 30, 2019, the payments of the operating lease liability were as follows: (in thousands) Remaining Lease Payments 2019 (remaining) $ 311 2020 1,272 2021 1,310 2022 1,349 2023 1,390 Thereafter 1,745 Total remaining lease payments 7,377 Less: imputed interest (1,511 ) Total operating lease liabilities $ 5,866 Weighted-average remaining lease term 6 years Weighted-average discount rate 8.4 % |
Description of Business, Basi_4
Description of Business, Basis of Presentation and Summary of Significant Accounting Policies (Details Textual) | Jul. 13, 2019USD ($) | Jul. 12, 2019USD ($) | Jun. 18, 2019USD ($) | Jan. 01, 2019USD ($) | Oct. 12, 2018USD ($) | Sep. 30, 2019USD ($) | Sep. 30, 2019USD ($)Segment | Sep. 30, 2018USD ($) | Feb. 11, 2019shares | Dec. 31, 2018USD ($) |
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Total net proceeds through sale of equity securities | $ 21,400,000 | |||||||||
Accumulated deficit | (60,679,000) | $ (60,679,000) | $ (44,874,000) | |||||||
Number of ordinary share of company in exchange for each ordinary share | shares | 1 | |||||||||
Number of operating segment for research and development | Segment | 1 | |||||||||
Dividends declared or paid | $ 0 | $ 0 | ||||||||
Increase in deferred revenue due to adoption of new revenue recognition | 10,941,000 | $ 3,647,000 | ||||||||
Operating lease liability | 5,866,000 | 5,866,000 | ||||||||
Operating lease right-of-use asset | $ 5,324,000 | $ 5,324,000 | ||||||||
Office and Lab Space [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Operating lease liability | $ 6,400,000 | |||||||||
Operating lease right-of-use asset | 5,900,000 | |||||||||
ASC 606 [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Increase in deferred revenue due to adoption of new revenue recognition | 800,000 | |||||||||
Increase in accumulated deficit | $ 800,000 | |||||||||
Loan and Security Agreement [Member] | Western Alliance Bank [Member] | Term Loan [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Gross proceeds from long-term debt agreement | $ 10,000,000 | |||||||||
Research Collaboration and License Agreement [Member] | Ultragenyx [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Third amendment fee received | $ 30,000,000 | |||||||||
Underlying Agreement [Member] | Cystic Fibrosis Foundation [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Payments for matching funds for remaining budgeted costs | $ 5,000,000 | |||||||||
Disbursed amount upon execution of amendment | 4,000,000 | |||||||||
Final payment of disbursement amount | 3,000,000 | |||||||||
Disbursement payment upon achievement of required manufacturing practices and IND application | 2,300,000 | |||||||||
Contra expense included in research and development expense | 700,000 | |||||||||
Contra expense remaining amount included in accrued expenses | 3,300,000 | |||||||||
Underlying Agreement [Member] | Cystic Fibrosis Foundation [Member] | Disbursement Due January 2020 [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Disbursement amount payable upon achievement of project goal | 2,000,000 | |||||||||
Underlying Agreement [Member] | Cystic Fibrosis Foundation [Member] | Disbursement Due April 2020 [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Disbursement amount payable upon achievement of project goal | 2,000,000 | |||||||||
Underlying Agreement [Member] | Cystic Fibrosis Foundation [Member] | Disbursement Due July 2020 [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Disbursement amount payable upon achievement of project goal | 2,000,000 | |||||||||
Underlying Agreement [Member] | Cystic Fibrosis Foundation [Member] | Disbursement Due October 2020 [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Disbursement amount payable upon achievement of project goal | 2,000,000 | |||||||||
Underlying Agreement [Member] | Cystic Fibrosis Foundation [Member] | LUNAR-CF [Member] | ||||||||||
Summary Of Significant Accounting Policy [Line Items] | ||||||||||
Payments for advance | $ 15,000,000 | $ 3,200,000 |
Schedule of Cash and Cash Equiv
Schedule of Cash and Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Dec. 31, 2017 |
Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 74,172 | $ 36,709 | $ 30,331 | |
Non-current restricted cash | 107 | 107 | 107 | |
Total cash, cash equivalents and restricted cash shown in the statement of cash flows | $ 74,279 | $ 36,816 | $ 30,438 | $ 25,238 |
Collaboration Revenue - Summary
Collaboration Revenue - Summary of Changes in Balances of Contract Assets and Contract Liability (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2019USD ($) | |
Collaboration Agreements [Abstract] | |
Contract Assets, Balance | $ 4,480 |
Additions for revenue billings | 16,524 |
Contract Assets, Deductions for cash collections | (18,379) |
Contract Assets, Balance | 2,625 |
Contract Liabilities, Balance | 13,806 |
Contract Liabilities, Additions for advanced billings | 29,136 |
Additions resulting from adoption of Topic 606 | 803 |
Deductions for promised goods/services provided in current period | (18,195) |
Contract Liabilities, Balance | $ 25,550 |
Collaboration Revenue - Summa_2
Collaboration Revenue - Summary of Collaboration Revenues (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Total collaboration revenue | $ 3,318 | $ 3,423 | $ 17,821 | $ 8,176 |
Collaboration Partner - Janssen [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Total collaboration revenue | 966 | 291 | 2,101 | 638 |
Collaboration Partner - Ultragenyx [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Total collaboration revenue | 922 | 1,789 | 4,854 | 4,495 |
Collaboration Partner - Takeda [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Total collaboration revenue | 224 | 302 | 518 | 905 |
Collaboration Partner - CureVac [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Total collaboration revenue | 1,057 | 314 | 6,360 | 772 |
Other Collaboration [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Total collaboration revenue | $ 149 | $ 727 | $ 3,988 | $ 1,366 |
Collaboration Revenue (Details
Collaboration Revenue (Details Textual) | Jul. 26, 2019USD ($) | Jun. 18, 2019USD ($)Target$ / sharesshares | Feb. 11, 2019 | Jan. 01, 2019USD ($) | Sep. 30, 2019USD ($)shares | Sep. 30, 2018USD ($) | Sep. 30, 2019USD ($)shares | Sep. 30, 2018USD ($) | Dec. 31, 2017USD ($) | Jun. 16, 2019Target | Dec. 31, 2018USD ($)shares |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Deferred revenue | $ 25,550,000 | $ 25,550,000 | $ 13,806,000 | ||||||||
Purchase of common stock, shares | shares | 15,126,000 | 15,126,000 | 10,762,000 | ||||||||
Increase in deferred revenue due to adoption of new revenue recognition | $ 10,941,000 | $ 3,647,000 | |||||||||
Collaboration revenue | $ 3,318,000 | $ 3,423,000 | 17,821,000 | 8,176,000 | |||||||
ASC 606 [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Increase in deferred revenue due to adoption of new revenue recognition | $ 800,000 | ||||||||||
Collaboration Partner Janssen [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration revenue | 966,000 | 291,000 | 2,101,000 | 638,000 | |||||||
Collaboration Partner - Ultragenyx [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration revenue | 922,000 | 1,789,000 | 4,854,000 | 4,495,000 | |||||||
Collaboration Partner - CureVac [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration revenue | 1,057,000 | 314,000 | 6,360,000 | 772,000 | |||||||
Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner Janssen [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Upfront payment received | 7,700,000 | ||||||||||
Revenue recognition potential milestone revenue recognized | 56,500,000 | ||||||||||
Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner Janssen [Member] | ASC 606 [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Remaining performance obligation | 0 | 0 | |||||||||
Adjustments to transaction price | 300,000 | 300,000 | |||||||||
Deferred revenue | 6,100,000 | 6,100,000 | $ 6,500,000 | ||||||||
Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner - Ultragenyx [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Upfront payment received | $ 6,000,000 | 27,900,000 | |||||||||
Revenue recognition potential milestone revenue recognized | $ 138,000,000 | ||||||||||
Royalty payment term description | Ultragenyx will pay royalties as a single-digit percentage of net sales on a product-by-product and country-by-country basis during the applicable royalty term. | ||||||||||
Number of targets | Target | 12 | 10 | |||||||||
Purchase of common stock, shares | shares | 2,400,000 | ||||||||||
Purchase of additional shares of common stock | shares | 600,000 | ||||||||||
Purchase of additional shares of common stock price per share | $ / shares | $ 16 | ||||||||||
Consideration received | $ 30,000,000 | ||||||||||
Consideration received for common stock purchase | 24,000,000 | ||||||||||
Transaction price | 14,400,000 | ||||||||||
Premium paid for purchase of common stock | $ 8,400,000 | ||||||||||
Lack of marketability discount restricted period | 2 years | ||||||||||
Collaboration revenue | 900,000 | $ 1,800,000 | $ 4,900,000 | $ 4,500,000 | |||||||
Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner - Ultragenyx [Member] | ASC 606 [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Remaining performance obligation | 0 | 0 | |||||||||
Deferred revenue | 13,600,000 | $ 13,600,000 | 2,700,000 | ||||||||
Revenue, practical expedient description | The Company recognizes the reimbursement of labor and expenses as costs are incurred and none of the development and commercialization milestones were included in the transaction price, as all milestone amounts were fully constrained. | ||||||||||
Cumulative catch up adjustment | $ 1,100,000 | ||||||||||
Revenue recognition, reserve target exclusivity period | 4 years | ||||||||||
Increase in deferred revenue due to adoption of new revenue recognition | $ 800,000 | ||||||||||
Decrease in retained earnings due to adoption of new revenue recognition | $ 800,000 | ||||||||||
mRNA Technology [Member] | Collaboration Partner - CureVac [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Royalty payment term description | CureVac will pay royalties as a percentage of net sales on a product-by-product and country-by-country basis during the applicable royalty term in the low single-digit range | ||||||||||
Development and option agreement date | Jan. 31, 2018 | ||||||||||
Expiration of initial term | 8 years | ||||||||||
Option to extend intial term on an annual basis | 3 years | ||||||||||
mRNA Technology [Member] | Collaboration Partner - CureVac [Member] | Rare Disease Targets [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue recognition potential milestone revenue recognized | $ 14,000,000 | ||||||||||
mRNA Technology [Member] | Collaboration Partner - CureVac [Member] | Non-Rare Disease Targets [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue recognition potential milestone revenue recognized | 23,000,000 | ||||||||||
mRNA Technology [Member] | Collaboration Partner - CureVac [Member] | ASC 606 [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Remaining performance obligation | 0 | 0 | |||||||||
Adjustments to transaction price | 0 | 0 | |||||||||
Deferred revenue | $ 3,400,000 | 3,400,000 | $ 4,400,000 | ||||||||
Transaction price | $ 5,000,000 | ||||||||||
Revenue, practical expedient description | The Company recognizes the reimbursement of labor and expenses as costs are incurred and none of the development and commercialization milestones were included in the transaction price, as all milestone amounts were fully constrained. | ||||||||||
Upfront fee received | $ 5,000,000 | ||||||||||
Contractual term | 46 months | ||||||||||
Cumulative catch up adjustment | $ 400,000 | ||||||||||
mRNA Technology [Member] | Collaboration Partner Cure Vac Entered Into Co Development Agreement [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement termination date | Feb. 11, 2019 | ||||||||||
Percentage of global rights reassumed | 100.00% | ||||||||||
Termination Agreement [Member] | Collaboration Partner Cure Vac Entered Into Co Development Agreement [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement settlement obligation one time settled amount | $ 4,000,000 | ||||||||||
Research And Exclusive License Agreement [Member] | Synthetic Genomics, Inc [Member] | Other Collaboration Revenue [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration revenue | $ 4,000,000 | ||||||||||
Other collaboration agreement date | 2017-10 | ||||||||||
Sublicense revenue recognized | $ 3,300,000 | ||||||||||
Minimum [Member] | Collaboration Partner Cure Vac Entered Into Co Development Agreement [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Potential target license term | 4 years | ||||||||||
Minimum [Member] | Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner Janssen [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Option exercise revenue range per target | $ 1,000,000 | ||||||||||
Minimum [Member] | Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner - Ultragenyx [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Option exercise revenue range per target | 500,000 | ||||||||||
Maximum [Member] | Collaboration Partner Cure Vac Entered Into Co Development Agreement [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Potential target license term | 8 years | ||||||||||
Maximum [Member] | Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner Janssen [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Option exercise revenue range per target | $ 5,000,000 | ||||||||||
Maximum [Member] | Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner - Ultragenyx [Member] | |||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||
Option exercise revenue range per target | $ 1,500,000 |
Collaboration Revenue (Detail_2
Collaboration Revenue (Details Textual 1) - Research Collaboration And Exclusive License Agreement [Member] - Collaboration Partner Janssen [Member] - ASC 606 [Member] $ in Millions | Sep. 30, 2019USD ($) |
Revenue Remaining Performance Obligation Expected Timing Of Satisfaction [Line Items] | |
Remaining performance obligation | $ 0 |
Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date: 2019-07-01 | |
Revenue Remaining Performance Obligation Expected Timing Of Satisfaction [Line Items] | |
Remaining research period | 18 months |
Remaining performance obligation | $ 23.6 |
Balance Sheet Details - Schedul
Balance Sheet Details - Schedule of Prepaid Expenses and Other Current Assets (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Prepaid Expenses And Other Current Assets [Abstract] | ||
Prepaid expenses | $ 2,578 | $ 546 |
Other current assets | 33 | 92 |
Total | $ 2,611 | $ 638 |
Balance Sheet Details - Summary
Balance Sheet Details - Summary of Components of Property and Equipment, Net (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | $ 4,101 | $ 3,472 |
Less accumulated depreciation and amortization | (2,028) | (1,497) |
Property and equipment, net | 2,073 | 1,975 |
Research equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 3,235 | 2,711 |
Computer and software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 271 | 200 |
Office equipment and furniture [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 561 | 527 |
Leasehold improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | $ 34 | $ 34 |
Balance Sheet Details (Details
Balance Sheet Details (Details Textual) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Balance Sheet Related Disclosures [Abstract] | ||||
Depreciation and amortization | $ 200 | $ 200 | $ 531 | $ 415 |
Balance Sheet Details - Sched_2
Balance Sheet Details - Schedule of Accrued Liabilities (Details) - USD ($) $ in Thousands | Sep. 30, 2019 | Dec. 31, 2018 |
Accrued Liabilities Current [Abstract] | ||
Accrued compensation | $ 1,979 | $ 974 |
Cystic Fibrosis Foundation Liability (Note 1) | 3,336 | |
Refundable fees received | 2,259 | |
Current portion of operating lease liability | 801 | |
Other accrued liabilities | 560 | 674 |
Total | $ 6,676 | $ 3,907 |
Debt (Details Textual)
Debt (Details Textual) - USD ($) shares in Thousands | Oct. 30, 2019 | Oct. 12, 2018 | Sep. 30, 2019 | Sep. 30, 2019 |
Common Stock [Member] | ||||
Debt Instrument [Line Items] | ||||
Stock issued during period | 1,995 | 1,995 | ||
Loan and Security Agreement [Member] | Western Alliance Bank [Member] | Long-term Debt [Member] | ||||
Debt Instrument [Line Items] | ||||
Gross proceeds from long-term debt agreement | $ 10,000,000 | |||
Loan maturity date | Oct. 30, 2023 | Oct. 1, 2022 | ||
Interest of the prime rate plus | 1.25% | |||
Loan interest payment terms | The loan has an interest-only period of 19 months, which could be extended by an additional 6 months if certain conditions are met, followed by an amortization period of 30 months, or 24 months if the interest-only period is extended. | |||
Loan interest-only payment period | 19 months | |||
Loan interest-only payment period | 6 months | |||
Debt Instrument, amortization period | 30 months | 30 months | ||
Debt Instrument, amortization period | 24 months | |||
Loan origination fee paid | $ 128,000 | |||
Warrant fee payable | $ 350,000 | |||
Prepayment fee percentage | 3.00% | |||
Prepayment fee percentage | 2.00% | |||
Percentage of fee liable on principal balance | 3.00% | |||
Fee payable upon repayment | $ 300,000 | |||
Debt instrument, collateral | The Loan is collateralized by all of the assets of the Company, excluding intellectual property, which is subject to a negative pledge. The Loan contains customary conditions of borrowing, events of default and covenants, including covenants that restrict the Company’s ability to dispose of assets, merge with or acquire other entities, incur indebtedness and make distributions to holders of the Company’s capital stock. In addition, the Company is required to maintain at least 50% of its deposit and investment accounts, or $20 million, whichever is lower, with Western Alliance Bank. | |||
Debt instrument, collateral amount | 20,000,000 | |||
Available sale of equity securities | $ 15,000,000 | |||
Term loan | $ 15,000,000 | |||
Repayments of existing debt | 10,000,000 | |||
Net increase in indebtedness | $ 5,000,000 | |||
Loan interest-only payment extended maturity date | Oct. 1, 2021 | |||
Loan interest-only payment maturity date | Apr. 1, 2021 | |||
Loan and Security Agreement [Member] | Western Alliance Bank [Member] | Long-term Debt [Member] | Ultragenyx [Member] | Common Stock [Member] | ||||
Debt Instrument [Line Items] | ||||
Stock issued during period | 2,400 | |||
Loan and Security Agreement [Member] | Western Alliance Bank [Member] | Long-term Debt [Member] | Minimum [Member] | ||||
Debt Instrument [Line Items] | ||||
Interest of the prime rate plus | 1.25% | |||
Prepayment fee percentage | 0.50% | |||
Percentage required to be maintain in deposit and investment | 50.00% | |||
Loan and Security Agreement [Member] | Western Alliance Bank [Member] | Long-term Debt [Member] | Maximum [Member] | ||||
Debt Instrument [Line Items] | ||||
Interest of the prime rate plus | 2.75% | |||
Prepayment fee percentage | 2.00% | |||
Deposits | $ 15,000,000 |
Stockholders' Equity (Details T
Stockholders' Equity (Details Textual) | Sep. 26, 2019USD ($)$ / sharesshares | Jun. 19, 2019$ / sharesshares | Jun. 18, 2019 | Mar. 31, 2013$ / sharesshares | Sep. 30, 2019$ / sharesshares | Sep. 30, 2018shares | Sep. 30, 2019USD ($)$ / sharesshares | Sep. 30, 2018shares | Dec. 31, 2017USD ($)Milestoneshares | Dec. 31, 2018₪ / sharesshares | Dec. 31, 2016Milestone | Dec. 31, 2014shares |
Class Of Stock [Line Items] | ||||||||||||
Gross proceeds from offering | $ | $ 21,278,000 | |||||||||||
Common stock, shares issued | 15,126,000 | 15,126,000 | 10,762,000 | |||||||||
Common stock, par value | (per share) | $ 0.001 | $ 0.001 | ₪ 0.07 | |||||||||
Antidilutive securities excluded from computation of earnings per share | 166,921 | 98,000 | 96,615 | 91,000 | ||||||||
Unvested Restricted Ordinary Shares [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Antidilutive securities excluded from computation of earnings per share | 622,667 | 622,667 | 622,667 | 622,667 | ||||||||
Restricted Common Stock [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Stock issued during period | 2,783,686 | |||||||||||
Share price of stock issued | $ / shares | $ 0.0068 | |||||||||||
Shares subject to repurchase option | 1,538,353 | |||||||||||
Share based compensation arrangement by share based payment award period from termination date of employee or consultant for repurchase of shares | 2 months | |||||||||||
Number of milestones achieved | Milestone | 2 | 2 | ||||||||||
Common shares, unvested | 622,667 | 622,667 | 622,667 | 622,667 | 769,176 | |||||||
Accelerated ordinary shares vested | 146,510 | |||||||||||
Modification expense resulting from accelerated vesting of ordinary shares | $ | $ 1,495,000 | |||||||||||
Vested After Obtaining Suitable siRNA License [Member] | Restricted Common Stock [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Shares vesting percentage | 25.00% | |||||||||||
Vested After in Vivo Proof-of-concept Achieved [Member] | Restricted Common Stock [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Shares vesting percentage | 25.00% | |||||||||||
Vested After IND Application Completed [Member] | Restricted Common Stock [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Shares vesting percentage | 25.00% | |||||||||||
Vested After in Human Efficacy Achieved [Member] | Restricted Common Stock [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Shares vesting percentage | 25.00% | |||||||||||
Equity Purchase Agreement [Member] | Collaboration Partner - Expanded Ultragenyx [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Common stock purchase price per share | $ / shares | $ 10 | |||||||||||
Common stock, shares issued | 2,400,000 | |||||||||||
Common stock, par value | $ / shares | $ 0.001 | |||||||||||
Purchase of additional shares of common stock | 600,000 | |||||||||||
Purchase of additional shares of common stock price per share | $ / shares | $ 16 | |||||||||||
Equity Purchase Agreement [Member] | Collaboration Partner - Ultragenyx [Member] | Maximum [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Ownership interest of common stock | 8.00% | |||||||||||
Registration Rights Agreement | Collaboration Partner - Ultragenyx [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Percentage of minimum common stock may not exercised total shares outstanding | 19.99% | |||||||||||
Registration Rights Agreement | Collaboration Partner - Ultragenyx [Member] | Maximum [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Period to register sale of shares | 180 days | |||||||||||
H.C. Wainwright & Co., LLC [Member] | Securities Purchase Agreement [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Net proceeds from offering after deducting placement agent fees and offering expenses | $ | $ 21,300,000 | |||||||||||
Closing date of offering | Sep. 30, 2019 | |||||||||||
Common Stock [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Stock issued during period | 1,995,000 | 1,995,000 | ||||||||||
Common Stock [Member] | H.C. Wainwright & Co., LLC [Member] | Letter Agreement [Member] | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Percentage of cash fee on gross proceeds from offering | 6.00% | |||||||||||
Common Stock [Member] | H.C. Wainwright & Co., LLC [Member] | Securities Purchase Agreement [Member] | Investor | ||||||||||||
Class Of Stock [Line Items] | ||||||||||||
Common stock purchase price per share | $ / shares | $ 11.50 | |||||||||||
Gross proceeds from offering | $ | $ 23,000,000 | |||||||||||
Common stock, shares issued | 1,995,653 |
Share-Based Compensation (Detai
Share-Based Compensation (Details Textual) - shares | 1 Months Ended | ||
Jun. 30, 2019 | Sep. 30, 2019 | Aug. 31, 2018 | |
2018 Omnibus Equity Incentive Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Share-based compensation number of shares authorized | 1,100,000 | ||
Shares issued in period | 243,750 | ||
2018 Omnibus Equity Incentive Plan [Member] | Non-Executive Officer [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Shares issued in period | 130,000 | ||
2019 Omnibus Equity Incentive Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Share-based compensation number of shares authorized | 2,600,000 | ||
Common stock reserved for future issuance | 1,233,707 |
Share-Based Compensation (Det_2
Share-Based Compensation (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Share-based compensation expenses | $ 383 | $ 587 | $ 1,185 | $ 753 |
Research and Development [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Share-based compensation expenses | 158 | 286 | 466 | 315 |
General and Administrative [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Share-based compensation expenses | $ 225 | $ 301 | $ 719 | $ 438 |
Leases (Details Textual)
Leases (Details Textual) - USD ($) | 1 Months Ended | 3 Months Ended | 9 Months Ended |
Oct. 31, 2017 | Sep. 30, 2019 | Sep. 30, 2019 | |
Lessee Lease Description [Line Items] | |||
Operating lease, remaining lease term | 6 years | 6 years | |
Operating lease costs | $ 300,000 | $ 900,000 | |
October 2017 Lease Amendment [Member] | |||
Lessee Lease Description [Line Items] | |||
Operating lease extended additional term | 84 months | ||
Operating lease, remaining lease term | 6 years | ||
Lessee, operating lease, term of contract | 4 months | ||
Tenant improvement allowance | $ 74,000 | ||
Lessee, operating leases, option to extend | The lease may be extended for one five-year period at the then current market rate with annual escalations; | ||
Security deposit | $ 96,000 |
Leases - Payments of Operating
Leases - Payments of Operating Lease Liability (Details) $ in Thousands | Sep. 30, 2019USD ($) |
Leases [Abstract] | |
2019 (remaining) | $ 311 |
2020 | 1,272 |
2021 | 1,310 |
2022 | 1,349 |
2023 | 1,390 |
Thereafter | 1,745 |
Total remaining lease payments | 7,377 |
Less: imputed interest | (1,511) |
Total operating lease liabilities | $ 5,866 |
Weighted-average remaining lease term | 6 years |
Weighted-average discount rate | 8.40% |
Related Party Transactions - Ul
Related Party Transactions - Ultragenyx (Details Textual) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Related Party Transaction [Line Items] | ||||
Collaboration revenue | $ 3,318 | $ 3,423 | $ 17,821 | $ 8,176 |
Collaboration Partner - Ultragenyx [Member] | ||||
Related Party Transaction [Line Items] | ||||
Collaboration revenue | 922 | 1,789 | $ 4,854 | 4,495 |
Research Collaboration And Exclusive License Agreement [Member] | Collaboration Partner - Ultragenyx [Member] | ||||
Related Party Transaction [Line Items] | ||||
Ownership interest of common stock | 15.90% | |||
Collaboration revenue | $ 900 | $ 1,800 | $ 4,900 | $ 4,500 |
Related Party Transactions - Pr
Related Party Transactions - Providence (Details Textual) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2019 | Sep. 30, 2018 | Sep. 30, 2019 | Sep. 30, 2018 | |
Related Party Transaction [Line Items] | ||||
Total collaboration revenue | $ 3,318 | $ 3,423 | $ 17,821 | $ 8,176 |
Providence Therapeutics, Inc [Member] | Research Collaboration and License Agreement [Member] | ||||
Related Party Transaction [Line Items] | ||||
Total collaboration revenue | 100 | $ 400 | 400 | $ 500 |
Accounts receivable, balance amount | $ 400 | $ 400 |
Related Party Transactions - Eq
Related Party Transactions - Equity-Method Investment (Details Textual) - USD ($) | 3 Months Ended | 6 Months Ended | 9 Months Ended | ||||
Sep. 30, 2019 | Sep. 30, 2018 | Jun. 30, 2019 | Sep. 30, 2019 | Sep. 30, 2018 | Dec. 31, 2018 | Jun. 30, 2018 | |
Related Party Transaction [Line Items] | |||||||
Share of gain (losses) for investee | $ 303,000 | $ (47,000) | $ 15,000 | $ (47,000) | |||
Equity investments | $ 303,000 | $ 303,000 | $ 288,000 | ||||
ADAIR Technology [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Equity method ownership percentage | 30.00% | ||||||
Share of gain (losses) for investee | $ (600,000) | ||||||
Equity investments | $ 0 | ||||||
ADAIR Technology [Member] | Minimum [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Equity method ownership percentage | 20.00% |