Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2016 | Nov. 10, 2016 | |
Document And Entity Information [Abstract] | ||
Entity Registrant Name | ARCA Biopharma, Inc. | |
Entity Central Index Key | 907,654 | |
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2016 | |
Document Fiscal Period Focus | Q3 | |
Trading Symbol | ABIO | |
Document Fiscal Year Focus | 2,016 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Smaller Reporting Company | |
Entity Common Stock, Shares Outstanding | 9,082,366 |
BALANCE SHEETS (Unaudited)
BALANCE SHEETS (Unaudited) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Current assets: | ||
Cash and cash equivalents | $ 8,095 | $ 38,802 |
Marketable securities | 15,373 | |
Other current assets | 320 | 114 |
Total current assets | 23,788 | 38,916 |
Marketable securities | 3,967 | |
Property and equipment, net | 69 | 28 |
Other assets | 855 | 630 |
Total assets | 28,679 | 39,574 |
Current liabilities: | ||
Accounts payable | 1,141 | 364 |
Accrued compensation and employee benefits | 162 | 669 |
Accrued expenses and other liabilities | 853 | 471 |
Total current liabilities | 2,156 | 1,504 |
Deferred rent, net of current portion | 43 | |
Total liabilities | 2,199 | 1,504 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Common stock, $0.001 par value; 100 million shares authorized at September 30, 2016 and December 31, 2015; 9,080,703 and 9,051,217 shares issued and outstanding at September 30, 2016 and December 31, 2015, respectively | 9 | 9 |
Additional paid-in capital | 134,744 | 134,128 |
Accumulated other comprehensive income | (6) | |
Accumulated deficit | (108,267) | (96,067) |
Total stockholders’ equity | 26,480 | 38,070 |
Total liabilities and stockholders’ equity | $ 28,679 | $ 39,574 |
BALANCE SHEETS (Unaudited) (Par
BALANCE SHEETS (Unaudited) (Parenthetical) - $ / shares | Sep. 30, 2016 | Dec. 31, 2015 |
Statement Of Financial Position [Abstract] | ||
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 100,000,000 | 100,000,000 |
Common stock, shares issued | 9,080,703 | 9,051,217 |
Common stock, shares outstanding | 9,080,703 | 9,051,217 |
STATEMENTS OF OPERATIONS AND CO
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Costs and expenses: | ||||
Research and development | $ 3,720 | $ 1,716 | $ 9,227 | $ 5,170 |
General and administrative | 992 | 1,101 | 3,094 | 3,110 |
Total costs and expenses | 4,712 | 2,817 | 12,321 | 8,280 |
Loss from operations | (4,712) | (2,817) | (12,321) | (8,280) |
Interest and other income | 53 | 4 | 121 | 7 |
Interest expense | (1) | (4) | ||
Net loss | (4,659) | (2,814) | (12,200) | (8,277) |
Change in unrealized loss on marketable securities | (19) | (6) | ||
Comprehensive loss | $ (4,678) | $ (2,814) | $ (12,206) | $ (8,277) |
Net loss per share: | ||||
Basic and diluted | $ (0.51) | $ (0.31) | $ (1.35) | $ (1.54) |
Weighted average shares outstanding: | ||||
Basic and diluted | 9,068,376 | 9,034,016 | 9,062,516 | 5,358,629 |
STATEMENTS OF STOCKHOLDERS' EQU
STATEMENTS OF STOCKHOLDERS' EQUITY (Unaudited) - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-In Capital | Accumulated Other Comprehensive Income | Accumulated Deficit |
Beginning Balance, shares at Dec. 31, 2014 | 3,021,498 | ||||
Beginning Balance at Dec. 31, 2014 | $ 14,741 | $ 3 | $ 99,360 | $ (84,622) | |
Issuance of common stock for cash, net of offering costs, shares | 6,003,082 | ||||
Issuance of common stock for cash, net of offering costs | 34,175 | $ 6 | 34,169 | ||
Issuance of common stock upon vesting of Restricted Stock Units, shares | 26,669 | ||||
Issuance of common stock upon vesting of Restricted Stock Units, value | 0 | $ 0 | 0 | $ 0 | 0 |
Adjustment for fractional shares | (32) | ||||
Share-based compensation | 599 | 599 | |||
Net loss | $ (11,445) | (11,445) | |||
Ending Balance, shares at Dec. 31, 2015 | 9,051,217 | 9,051,217 | |||
Ending Balance at Dec. 31, 2015 | $ 38,070 | $ 9 | 134,128 | (96,067) | |
Issuance of common stock upon vesting of Restricted Stock Units, shares | 29,486 | ||||
Issuance of common stock upon vesting of Restricted Stock Units, value | 0 | $ 0 | 0 | 0 | 0 |
Share-based compensation | 605 | 605 | |||
Change in unrealized loss on marketable securities | (6) | (6) | |||
Other | 11 | 11 | |||
Net loss | $ (12,200) | (12,200) | |||
Ending Balance, shares at Sep. 30, 2016 | 9,080,703 | 9,080,703 | |||
Ending Balance at Sep. 30, 2016 | $ 26,480 | $ 9 | $ 134,744 | $ (6) | $ (108,267) |
STATEMENTS OF CASH FLOWS (Unaud
STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
Cash flows from operating activities: | ||
Net loss | $ (12,200) | $ (8,277) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation | 17 | 14 |
Amortization of other assets | 207 | 133 |
Amortization of premiums and discounts on marketable securities | 100 | |
Share-based compensation | 605 | 454 |
Change in operating assets and liabilities: | ||
Other current assets | (98) | 147 |
Other assets | (303) | (229) |
Accounts payable | 648 | (167) |
Accrued compensation and employee benefits | (507) | (215) |
Accrued expenses and other liabilities | 391 | 229 |
Other | (5) | (3) |
Net cash used in operating activities | (11,145) | (7,914) |
Cash flows from investing activities: | ||
Purchases of property and equipment | (8) | (9) |
Purchases of marketable securities | (20,504) | |
Proceeds from maturities of marketable securities | 950 | |
Net cash used in investing activities | (19,562) | (9) |
Cash flows from financing activities: | ||
Proceeds from the issuance of common stock | 37,000 | |
Common stock offering costs | (2,756) | |
Repayment of principal on vendor finance agreement | (193) | |
Net cash provided by financing activities | 34,051 | |
Net (decrease) increase in cash and cash equivalents | (30,707) | 26,128 |
Cash and cash equivalents, beginning of period | 38,802 | 15,354 |
Cash and cash equivalents, end of period | 8,095 | 41,482 |
Supplemental cash flow information: | ||
Interest paid | 4 | |
Supplemental disclosure of noncash investing and financing transactions: | ||
Common stock offering costs accrued but not yet paid | (11) | $ 69 |
Change in unrealized loss on marketable securities | (6) | |
Leasehold improvement lease incentive | $ 48 |
The Company and Summary of Sign
The Company and Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2016 | |
Accounting Policies [Abstract] | |
The Company and Summary of Significant Accounting Policies | (1) The Company and Summary of Significant Accounting Policies Description of Business ARCA biopharma, Inc., or the Company or ARCA, a Delaware corporation, is headquartered in Westminster, Colorado. The Company is a biopharmaceutical company principally focused on developing genetically-targeted therapies for cardiovascular diseases. The Company’s lead product candidate, Gencaro™ (bucindolol hydrochloride), is a pharmacologically unique beta-blocker and mild vasodilator that ARCA is evaluating in a clinical trial for the treatment of atrial fibrillation (AF) in patients with heart failure with reduced left ventricular ejection fraction (HFREF). The Company has identified common genetic variations in receptors in the cardiovascular system that it believes interact with Gencaro’s pharmacology and may predict patient response to the drug. The Company is testing this hypothesis in a Phase 2B/Phase 3 clinical trial of Gencaro, known as GENETIC-AF. The AF indication for Gencaro was chosen based on clinical data from a prior Phase 3 heart failure (HF) trial of Gencaro in 2,708 HF patients, or the BEST trial, which suggested that Gencaro may be successful in reducing or preventing AF. GENETIC-AF is a Phase 2B/Phase 3, multi-center, randomized, double-blind, adaptive design clinical trial comparing the safety and efficacy of Gencaro against an active comparator, the beta-blocker Toprol XL (metoprolol succinate), that seeks to enroll a combined total of approximately 620 patients. Eligible patients will have HFREF, a history of paroxysmal AF (episodes lasting 7 days or less) or persistent AF (episodes lasting more than 7 days and less than 1 year) in the past 6 months, and the beta-1 389 arginine homozygous genotype that the Company believes responds most favorably to Gencaro. The primary endpoint of the study is time to first event of symptomatic AF/atrial flutter (AFL), or all-cause mortality. The GENETIC-AF Data and Safety Monitoring Board (DSMB) will conduct a pre-specified interim analysis of study endpoints for efficacy, safety and futility to recommend whether or not the trial should proceed to Phase 3. The DSMB will make its recommendation based on a predictive probability analysis of certain trial data after a sufficient number of patients have evaluable endpoint data. An enrolled patient has evaluable endpoint data either when they experience their first endpoint event, or after they complete the 24-week follow up period. The DSMB interim analysis will focus on analyses of the AF/AFL endpoints in the trial using both clinical-based intermittent monitoring and device-based continuous monitoring techniques. Based on the results of the interim analysis, the DSMB may recommend that the trial proceed to Phase 3, the trial be completed as a Phase 2B study, or termination of the trial due to futility. ARCA, in collaboration with the GENETIC-AF Steering Committee, will determine the next steps for the trial based on the DSMB recommendation from this interim analysis and on the Company’s available financing. Based on the current enrollment projections, the Company now expects to reach 150 patients enrolled in the trial during the first quarter of 2017. The Company projects that the outcome of the DSMB interim analysis and recommendation will be available in the third quarter of 2017. Should the DSMB recommend that the study continue to Phase 3, the trial would continue enrolling to a total of approximately 620 patients, subject to the Company obtaining sufficient financing to fund the Phase 3 portion of the trial. If the Company continues with the Phase 3 portion of the GENETIC-AF, it will need to raise additional capital to complete the Phase 3 portion of the GENETIC-AF clinical trial and submit for FDA approval. If the Company is unable to obtain additional funding or is unable to complete a strategic transaction, it may have to discontinue development activities on Gencaro or discontinue its operations. Liquidity and Going Concern The Company devotes substantially all of its efforts towards obtaining regulatory approval and raising capital necessary to fund its operations and it is subject to a number of risks associated with clinical research and development, including dependence on key individuals, the development of and regulatory approval of commercially viable products, the need to raise adequate additional financing necessary to fund the development and commercialization of its products, and competition from larger companies. The Company has not generated revenue to date and has incurred substantial losses and negative cash flows from operations since its inception. The Company has historically funded its operations through issuances of common and preferred stock. In June 2015, the Company raised approximately $34.2 million in net proceeds to provide additional funds for the Phase 2B/3 GENETIC-AF trial and the Company’s ongoing operations. The Company is enrolling patients in the Phase 2B portion of the GENETIC-AF trial, and the Company believes that its current cash and cash equivalents will be sufficient to fund its operations, at its projected cost structure, through at least the end of 2017. However, in light of the significant uncertainties regarding clinical development timelines and costs for developing drugs such as Gencaro, the Company expects to raise additional capital to finance the completion of GENETIC-AF and the Company’s future operations. If the Company is delayed in completing or is unable to complete additional funding and/or a strategic transaction, the Company may discontinue its development activities or operations. The Company’s liquidity, and its ability to raise additional capital or complete any strategic transaction, depends on a number of factors, including, but not limited to, the following: • progress of GENETIC-AF, including enrollment and any data that may become available; • the costs and timing for the GENETIC-AF clinical trial in order to gain possible FDA approval for Gencaro; • the market price of the Company’s stock and the availability and cost of additional equity capital; • the Company’s ability to retain the listing of its common stock on the Nasdaq Capital Market; • general economic and industry conditions affecting the availability and cost of capital; • the Company’s ability to control costs associated with its operations; • the costs of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; and • the terms and conditions of the Company’s existing collaborative and licensing agreements. The sale of additional equity or convertible debt securities would likely result in substantial additional dilution to the Company’s stockholders. If the Company raises additional funds through the incurrence of indebtedness, the obligations related to such indebtedness would be senior to rights of holders of the Company’s capital stock and could contain covenants that would restrict the Company’s operations. The Company also cannot predict what consideration might be available, if any, to the Company or its stockholders, in connection with any strategic transaction. Should strategic alternatives or additional capital not be available to the Company, or not be available on acceptable terms, the Company may be unable to realize value from its assets and discharge its liabilities in the normal course of business which may, among other alternatives, cause the Company to further delay, substantially reduce or discontinue operational activities to conserve its cash resources. There are significant uncertainties surrounding the clinical development timelines and costs and likely the need to raise a significant amount of capital in the future. These financial statements have been prepared with the assumption that the Company will continue as a going concern and will be able to realize its assets and discharge its liabilities in the normal course of business and do not include any adjustments to reflect the possible future effects on the recoverability and classification of assets or the amounts and classification of liabilities that may result from the inability of the Company to continue as a going concern. The Company may not be able to raise sufficient capital on acceptable terms, or at all, to continue development of Gencaro or to otherwise continue operations and may not be able to execute any strategic transaction. Basis of Presentation The accompanying unaudited financial statements of the Company were prepared in accordance with generally accepted accounting principles for interim financial information and instructions to Form 10-Q and Rule 10-01 of Regulation S-X. Accordingly, these financial statements do not include all of the information and footnotes required by accounting principles generally accepted in the United States of America (GAAP) for complete financial statements. In the opinion of management, these financial statements include all normal and recurring adjustments considered necessary for a fair presentation of these interim financial statements. The results of operations for the nine months ended September 30, 2016 are not necessarily indicative of results expected for the full year ending December 31, 2016. The Company has generated no revenue to date and its activities have consisted of seeking regulatory approval, research and development, exploring strategic alternatives for further developing and commercializing Gencaro, and raising capital. These unaudited financial statements should be read in conjunction with the audited financial statements and footnotes thereto for the year ended December 31, 2015 included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission. Amounts presented are rounded to the nearest thousand, where indicated, except per share data and par values. Concentrations of Credit Risk Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash and cash equivalents. The Company has no off-balance-sheet concentrations of credit risk, such as foreign exchange contracts, option contracts, or foreign currency hedging arrangements. The Company maintains cash and cash equivalent balances in the form of bank demand deposits and money market fund accounts with financial institutions that management believes are creditworthy. Such balances may at times exceed the insured amount. Accrued Expenses As part of the process of preparing its financial statements, the Company is required to estimate accrued expenses. This process involves identifying services that third parties have performed on the Company’s behalf and estimating the level of service performed and the associated cost incurred for these services as of the balance sheet date. Examples of estimated accrued expenses include contract service fees, such as fees payable to contract manufacturers in connection with the production of materials related to the Company’s drug product, and professional service fees, such as attorneys, consultants, and clinical research organizations. The Company develops estimates of liabilities using its judgment based upon the facts and circumstances known at the time. Recent Accounting Pronouncements In August 2014, the Financial Accounting Standards Board (FASB) issued FASB Accounting Standards Update (ASU No. 2014-15), Presentation of Financial Statements – Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern. . In April 2015, the FASB issued Accounting Standards Update No. 2015-05: Intangibles - Goodwill and Other - Internal-Use Software (Subtopic 350-40): Customer's Accounting for Fees Paid in a Cloud Computing Arrangement In January 2016, FASB issued ASU No. 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) In March 2016, the FASB issued ASU No. 2016-09, Compensation - Stock Compensation: Improvements to Employee Share-Based Payment Accounting |
Net Loss Per Share
Net Loss Per Share | 9 Months Ended |
Sep. 30, 2016 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | (2) Net Loss Per Share The Company calculates basic earnings per share by dividing loss attributable to common stockholders by the weighted average common shares outstanding during the period, excluding common stock subject to vesting provisions. Diluted earnings per share is computed by dividing loss attributable to common stockholders by the weighted average number of common shares outstanding during the period increased to include, if dilutive, the number of additional common shares that would have been outstanding if the potential common shares had been issued. The Company’s potentially dilutive shares include stock options, restricted stock units and warrants for common stock . Because we reported a net loss for the three and nine months ended September 30, 2016 and 2015, all potentially dilutive shares of common stock have been excluded from the computation of the dilutive net loss per share for all periods presented. September 30, 2016 2015 Potentially dilutive securities, excluded: Outstanding stock options 584,865 170,545 Unvested restricted stock units 32,402 71,780 Warrants to purchase common stock 3,718,022 3,739,948 4,335,289 3,982,273 |
Marketable Securities and Fair
Marketable Securities and Fair Value Disclosures | 9 Months Ended |
Sep. 30, 2016 | |
Marketable Securities And Fair Value Disclosures [Abstract] | |
Marketable Securities and Fair Value Disclosures | (3) Marketable Securities and Fair Value Disclosures Marketable securities consisted of the following as of September 30, 2016 (in thousands): September 30, 2016 Gross Gross Amortized Unrealized Unrealized Fair Cost Gains Losses Value Short-term available-for-sale securities: Corporate bonds $ 14,982 $ 2 $ (11 ) $ 14,973 Commercial paper 400 — — 400 Total $ 15,382 $ 2 $ (11 ) $ 15,373 Long-term available-for-sale securities: Corporate bonds $ 3,964 $ 6 $ (3 ) $ 3,967 Total $ 3,964 $ 6 $ (3 ) $ 3,967 As of September 30, 2016, the amortized cost and estimated fair value of available-for-sale securities by contractual maturity were as follows (in thousands): Amortized Fair Cost Value Due in one year or less $ 15,382 $ 15,373 Due in one to two years 3,964 3,967 Total $ 19,346 $ 19,340 Fair value is defined as 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 (exit price). Inputs used to measure fair value are classified into the following hierarchy: • Level 1—Unadjusted quoted prices in active markets for identical assets or liabilities. The Company’s Level 1 assets consist of money market investments. The Company does not have any Level 1 liabilities. • Level 2—Unadjusted quoted prices in active markets for similar assets or liabilities; unadjusted quoted prices for identical or similar assets or liabilities in markets that are not active; or inputs other than quoted prices that are observable for the asset or liability. The Company’s Level 2 assets consist of corporate bonds and commercial paper securities. The Company does not have any Level 2 liabilities. • Level 3—Unobservable inputs for the asset or liability. The Company does not have any Level 3 assets or liabilities. The following table identifies the Company’s assets that were measured at fair value on a recurring basis (in thousands): Balance Level 1 Level 2 Level 3 September 30, 2016 Money market $ 8,026 $ 8,026 $ — $ — Corporate bonds 18,940 — 18,940 — Commercial paper 400 — 400 — Total $ 27,366 $ 8,026 $ 19,340 $ — December 31, 2015 Money market $ 38,728 $ 38,728 $ — $ — Total $ 38,728 $ 38,728 $ — $ — As of September 30, 2016 and December 31, 2015, the Company had $8.0 million and $38.7 million, respectively, of cash equivalents consisting of money market funds with maturities of 90 days or less. The Company has the ability to liquidate these investments without restriction. The Company determines fair value for these money market funds and equity securities with Level 1 inputs through quoted market prices. There were no transfers of assets between fair value hierarchy levels during the nine month period ended September 30, 2016. Fair Value of Other Financial Instruments The carrying amount of other financial instruments, including cash and accounts payable approximated fair value due to their short maturities. |
Property and Equipment
Property and Equipment | 9 Months Ended |
Sep. 30, 2016 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment | (4) Property and Equipment Property and equipment consist of the following (in thousands): Estimated Life September 30, 2016 December 31, 2015 Computer equipment 3 years $ 81 $ 90 Lab equipment 5 years 142 142 Furniture and fixtures 5 years 83 91 Computer software 3 years 84 84 Leasehold improvements Lesser of useful life or life of the lease 59 8 449 415 Accumulated depreciation and amortization (380 ) (387 ) Property and equipment, net $ 69 $ 28 For the nine months ended September 30, 2016 and 2015, depreciation and amortization expense was $17,000 and $14,000, respectively. |
Related Party Arrangements
Related Party Arrangements | 9 Months Ended |
Sep. 30, 2016 | |
Related Party Transactions [Abstract] | |
Related Party Arrangements | (5) Related Party Arrangements Transactions with the Company’s President and Chief Executive Officer The Company has entered into unrestricted research grants with its President and Chief Executive Officer’s academic research laboratory at the University of Colorado. Funding of any unrestricted research grants is contingent upon the Company’s financial condition, and can be deferred or terminated at the Company’s discretion. Total expense under these arrangements for the nine months ended September 30, 2016 and 2015 was $329,000 and $277,000 respectively. |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2016 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | (6) Commitments and Contingencies The Company has or is subject to the following commitments and contingencies: Employment Agreements The Company maintains employment agreements with several key executive employees. The agreements may be terminated at any time by the Company with or without cause upon written notice to the employee, and entitle the employee to wages in lieu of notice for periods not exceeding one calendar year from the date of termination without cause or by the employee for good reason. Certain of these agreements also provide for payments to be made under certain conditions related to a change in control of the Company. Operating Lease On August 1, 2013 the Company entered into a lease agreement for approximately 5,300 square feet of office facilities in Westminster, Colorado which has served as the Company’s primary business office since October 1, 2013. Effective March 2, 2016, the lease was renewed for an additional 38 month term beginning October 1, 2016 and expiring on November 30, 2019. Below is a summary of the future minimum lease payments committed for the Company’s facility in Westminster, Colorado as of September 30, 2016 (in thousands): Remainder of 2016 $ 7 2017 83 2018 88 2019 83 Total future minimum lease payments $ 261 Rent expense for the nine months ended September 30, 2016 and 2015 was $62,000 and $60,000, respectively. Duke University In November 2013, the Company entered into a clinical research agreement with Duke University (Duke) to serve as the clinical research organization for the Company’s GENETIC-AF clinical study. Under the agreement the Company is responsible to pay Duke for their work managing certain aspects of the clinical study. Upon completion of the clinical study, the agreement will terminate. The agreement can be terminated earlier by the Company for any reason with 90 . Cardiovascular Pharmacology and Engineering Consultants, LLC ARCA has licensed worldwide rights to Gencaro, including all preclinical and clinical data from Cardiovascular Pharmacology and Engineering Consultants, LLC (CPEC), who has licensed rights in Gencaro from Bristol Myers Squib (BMS). CPEC is a licensing subsidiary of Indevus Pharmaceuticals Inc. (a wholly owned subsidiary of Endo Pharmaceuticals), holding ownership rights to certain clinical trial data of Gencaro. Under the terms of its license agreement with CPEC, the Company will incur milestone and royalty obligations upon the occurrence of certain events. If the FDA grants marketing approval for Gencaro, the license agreement states that the Company will owe CPEC a milestone payment of $8.0 million within six months after FDA approval. The license agreement states that a milestone payment of up to $5.0 million in the aggregate shall be paid upon regulatory marketing approval in Europe and Japan. The license agreement also states that the Company’s royalty obligation ranges from 12.5% to 25% of revenue from the related product based on achievement of specified product sales levels, including a 5% royalty that CPEC is obligated to pay under its original license agreement for Gencaro. The agreement states that the Company has the right to buy down the royalties to a range of 12.5% to 17% by making a payment to CPEC within six months of regulatory approval. |
Equity Financings and Warrants
Equity Financings and Warrants | 9 Months Ended |
Sep. 30, 2016 | |
Equity [Abstract] | |
Equity Financings and Warrants | (7) Equity Financings and Warrants 2015 Equity Financing Private Investment in Public Entity (PIPE) Transaction On June 16, 2015, the Company sold an aggregate of 6,003,082 security units, made up of an aggregate 6,003,082 shares of the Company’s common stock and warrants to purchase an aggregate of 2,401,233 shares of the Company’s common stock, at a purchase price of $6.1635 per unit, for aggregate net proceeds of approximately $34.2 million. The warrants became exercisable on December 13, 2015, expire on June 16, 2022, and have an exercise price of $6.1012 per share. The warrants provide for cashless exercise and settlement in unregistered shares if there is no effective registration statement registering, or the prospectus contained therein is not available for, the issuance of the shares of common stock underlying the warrants at the time of exercise. The Company’s Registration Statement on Form S-3 registering the shares of common stock, including the shares of common stock underlying the warrants, issued in the transaction for public resale was declared effective on July 20, 2015. Warrants Warrants to purchase shares of common stock were granted as part of various financing and business agreements. All outstanding warrants were recorded in additional paid-in capital at their estimated fair market value at the date of grant using a Black-Scholes option-pricing model. As of September 30, 2016, these warrants, by year of expiration, are summarized below: Year of Expiration Number of Warrants Weighted Average Exercise Price 2016 60,890 $ 87.39 2017 24,124 17.89 2018 963,153 11.77 2019 224,323 15.73 2020 44,299 15.96 2022 2,401,233 6.10 3,718,022 $ 9.67 |
Share-based Compensation
Share-based Compensation | 9 Months Ended |
Sep. 30, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Share-based Compensation | (8) Share-based Compensation For the three and nine month periods ended September 30, 2016 and 2015, the Company recognized the following non-cash, share-based compensation expense in the statements of operations (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Research and development $ 94 $ 42 $ 189 $ 132 General and administrative 173 107 416 322 Total $ 267 $ 149 $ 605 $ 454 Stock option transactions for the nine month period ended September 30, 2016 under the Company’s stock incentive plans were as follows: Number of Options Weighted Average Exercise Price Weighted Average Remaining Contractual Term (in years) Options outstanding at December 31, 2015 161,278 $ 18.30 6.97 Granted 442,300 3.31 Exercised — — Forfeited and cancelled (18,713 ) 28.39 Options outstanding at September 30, 2016 584,865 $ 6.64 9.02 Options exercisable at September 30, 2016 159,887 $ 14.55 7.46 Options vested and expected to vest 583,904 $ 6.65 9.02 Stock award transactions related to restricted stock units for the nine month period ended September 30, 2016 under the Company’s stock incentive plans were as follows: Number of Shares Weighted Average Grant Date Fair Value Restricted stock units outstanding at December 31, 2015 62,359 $ 8.36 Granted — — Vested and released (29,486 ) 8.78 Forfeited and cancelled (471 ) 4.69 Restricted stock units outstanding at September 30, 2016 32,402 $ 8.02 |
Income Taxes
Income Taxes | 9 Months Ended |
Sep. 30, 2016 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | (9) Income Taxes In accordance with GAAP, a valuation allowance should be provided if it is more likely than not that some or all of the Company’s deferred tax assets will not be realized. The Company’s ability to realize the benefit of its deferred tax assets will depend on the generation of future taxable income. Due to the uncertainty of future profitable operations and taxable income, the Company has recorded a full valuation allowance against its net deferred tax assets. The Company believes its tax filing positions and deductions related to tax periods subject to examination will be sustained upon audit and, therefore, has no reserve for uncertain tax positions. |
The Company and Summary of Si16
The Company and Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2016 | |
Accounting Policies [Abstract] | |
Description of Business | Description of Business ARCA biopharma, Inc., or the Company or ARCA, a Delaware corporation, is headquartered in Westminster, Colorado. The Company is a biopharmaceutical company principally focused on developing genetically-targeted therapies for cardiovascular diseases. The Company’s lead product candidate, Gencaro™ (bucindolol hydrochloride), is a pharmacologically unique beta-blocker and mild vasodilator that ARCA is evaluating in a clinical trial for the treatment of atrial fibrillation (AF) in patients with heart failure with reduced left ventricular ejection fraction (HFREF). The Company has identified common genetic variations in receptors in the cardiovascular system that it believes interact with Gencaro’s pharmacology and may predict patient response to the drug. The Company is testing this hypothesis in a Phase 2B/Phase 3 clinical trial of Gencaro, known as GENETIC-AF. The AF indication for Gencaro was chosen based on clinical data from a prior Phase 3 heart failure (HF) trial of Gencaro in 2,708 HF patients, or the BEST trial, which suggested that Gencaro may be successful in reducing or preventing AF. GENETIC-AF is a Phase 2B/Phase 3, multi-center, randomized, double-blind, adaptive design clinical trial comparing the safety and efficacy of Gencaro against an active comparator, the beta-blocker Toprol XL (metoprolol succinate), that seeks to enroll a combined total of approximately 620 patients. Eligible patients will have HFREF, a history of paroxysmal AF (episodes lasting 7 days or less) or persistent AF (episodes lasting more than 7 days and less than 1 year) in the past 6 months, and the beta-1 389 arginine homozygous genotype that the Company believes responds most favorably to Gencaro. The primary endpoint of the study is time to first event of symptomatic AF/atrial flutter (AFL), or all-cause mortality. The GENETIC-AF Data and Safety Monitoring Board (DSMB) will conduct a pre-specified interim analysis of study endpoints for efficacy, safety and futility to recommend whether or not the trial should proceed to Phase 3. The DSMB will make its recommendation based on a predictive probability analysis of certain trial data after a sufficient number of patients have evaluable endpoint data. An enrolled patient has evaluable endpoint data either when they experience their first endpoint event, or after they complete the 24-week follow up period. The DSMB interim analysis will focus on analyses of the AF/AFL endpoints in the trial using both clinical-based intermittent monitoring and device-based continuous monitoring techniques. Based on the results of the interim analysis, the DSMB may recommend that the trial proceed to Phase 3, the trial be completed as a Phase 2B study, or termination of the trial due to futility. ARCA, in collaboration with the GENETIC-AF Steering Committee, will determine the next steps for the trial based on the DSMB recommendation from this interim analysis and on the Company’s available financing. Based on the current enrollment projections, the Company now expects to reach 150 patients enrolled in the trial during the first quarter of 2017. The Company projects that the outcome of the DSMB interim analysis and recommendation will be available in the third quarter of 2017. Should the DSMB recommend that the study continue to Phase 3, the trial would continue enrolling to a total of approximately 620 patients, subject to the Company obtaining sufficient financing to fund the Phase 3 portion of the trial. If the Company continues with the Phase 3 portion of the GENETIC-AF, it will need to raise additional capital to complete the Phase 3 portion of the GENETIC-AF clinical trial and submit for FDA approval. If the Company is unable to obtain additional funding or is unable to complete a strategic transaction, it may have to discontinue development activities on Gencaro or discontinue its operations. |
Liquidity and Going Concern | Liquidity and Going Concern The Company devotes substantially all of its efforts towards obtaining regulatory approval and raising capital necessary to fund its operations and it is subject to a number of risks associated with clinical research and development, including dependence on key individuals, the development of and regulatory approval of commercially viable products, the need to raise adequate additional financing necessary to fund the development and commercialization of its products, and competition from larger companies. The Company has not generated revenue to date and has incurred substantial losses and negative cash flows from operations since its inception. The Company has historically funded its operations through issuances of common and preferred stock. In June 2015, the Company raised approximately $34.2 million in net proceeds to provide additional funds for the Phase 2B/3 GENETIC-AF trial and the Company’s ongoing operations. The Company is enrolling patients in the Phase 2B portion of the GENETIC-AF trial, and the Company believes that its current cash and cash equivalents will be sufficient to fund its operations, at its projected cost structure, through at least the end of 2017. However, in light of the significant uncertainties regarding clinical development timelines and costs for developing drugs such as Gencaro, the Company expects to raise additional capital to finance the completion of GENETIC-AF and the Company’s future operations. If the Company is delayed in completing or is unable to complete additional funding and/or a strategic transaction, the Company may discontinue its development activities or operations. The Company’s liquidity, and its ability to raise additional capital or complete any strategic transaction, depends on a number of factors, including, but not limited to, the following: • progress of GENETIC-AF, including enrollment and any data that may become available; • the costs and timing for the GENETIC-AF clinical trial in order to gain possible FDA approval for Gencaro; • the market price of the Company’s stock and the availability and cost of additional equity capital; • the Company’s ability to retain the listing of its common stock on the Nasdaq Capital Market; • general economic and industry conditions affecting the availability and cost of capital; • the Company’s ability to control costs associated with its operations; • the costs of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; and • the terms and conditions of the Company’s existing collaborative and licensing agreements. The sale of additional equity or convertible debt securities would likely result in substantial additional dilution to the Company’s stockholders. If the Company raises additional funds through the incurrence of indebtedness, the obligations related to such indebtedness would be senior to rights of holders of the Company’s capital stock and could contain covenants that would restrict the Company’s operations. The Company also cannot predict what consideration might be available, if any, to the Company or its stockholders, in connection with any strategic transaction. Should strategic alternatives or additional capital not be available to the Company, or not be available on acceptable terms, the Company may be unable to realize value from its assets and discharge its liabilities in the normal course of business which may, among other alternatives, cause the Company to further delay, substantially reduce or discontinue operational activities to conserve its cash resources. There are significant uncertainties surrounding the clinical development timelines and costs and likely the need to raise a significant amount of capital in the future. These financial statements have been prepared with the assumption that the Company will continue as a going concern and will be able to realize its assets and discharge its liabilities in the normal course of business and do not include any adjustments to reflect the possible future effects on the recoverability and classification of assets or the amounts and classification of liabilities that may result from the inability of the Company to continue as a going concern. The Company may not be able to raise sufficient capital on acceptable terms, or at all, to continue development of Gencaro or to otherwise continue operations and may not be able to execute any strategic transaction. |
Basis of Presentation | Basis of Presentation The accompanying unaudited financial statements of the Company were prepared in accordance with generally accepted accounting principles for interim financial information and instructions to Form 10-Q and Rule 10-01 of Regulation S-X. Accordingly, these financial statements do not include all of the information and footnotes required by accounting principles generally accepted in the United States of America (GAAP) for complete financial statements. In the opinion of management, these financial statements include all normal and recurring adjustments considered necessary for a fair presentation of these interim financial statements. The results of operations for the nine months ended September 30, 2016 are not necessarily indicative of results expected for the full year ending December 31, 2016. The Company has generated no revenue to date and its activities have consisted of seeking regulatory approval, research and development, exploring strategic alternatives for further developing and commercializing Gencaro, and raising capital. These unaudited financial statements should be read in conjunction with the audited financial statements and footnotes thereto for the year ended December 31, 2015 included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission. Amounts presented are rounded to the nearest thousand, where indicated, except per share data and par values. |
Concentrations of Credit Risk | Concentrations of Credit Risk Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash and cash equivalents. The Company has no off-balance-sheet concentrations of credit risk, such as foreign exchange contracts, option contracts, or foreign currency hedging arrangements. The Company maintains cash and cash equivalent balances in the form of bank demand deposits and money market fund accounts with financial institutions that management believes are creditworthy. Such balances may at times exceed the insured amount. |
Accrued Expenses | Accrued Expenses As part of the process of preparing its financial statements, the Company is required to estimate accrued expenses. This process involves identifying services that third parties have performed on the Company’s behalf and estimating the level of service performed and the associated cost incurred for these services as of the balance sheet date. Examples of estimated accrued expenses include contract service fees, such as fees payable to contract manufacturers in connection with the production of materials related to the Company’s drug product, and professional service fees, such as attorneys, consultants, and clinical research organizations. The Company develops estimates of liabilities using its judgment based upon the facts and circumstances known at the time. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In August 2014, the Financial Accounting Standards Board (FASB) issued FASB Accounting Standards Update (ASU No. 2014-15), Presentation of Financial Statements – Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern. . In April 2015, the FASB issued Accounting Standards Update No. 2015-05: Intangibles - Goodwill and Other - Internal-Use Software (Subtopic 350-40): Customer's Accounting for Fees Paid in a Cloud Computing Arrangement In January 2016, FASB issued ASU No. 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) In March 2016, the FASB issued ASU No. 2016-09, Compensation - Stock Compensation: Improvements to Employee Share-Based Payment Accounting |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Earnings Per Share [Abstract] | |
Summary of Potentially Dilutive Shares of Common Stock | Because we reported a net loss for the three and nine months ended September 30, 2016 and 2015, all potentially dilutive shares of common stock have been excluded from the computation of the dilutive net loss per share for all periods presented. September 30, 2016 2015 Potentially dilutive securities, excluded: Outstanding stock options 584,865 170,545 Unvested restricted stock units 32,402 71,780 Warrants to purchase common stock 3,718,022 3,739,948 4,335,289 3,982,273 |
Marketable Securities and Fai18
Marketable Securities and Fair Value Disclosures (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Marketable Securities And Fair Value Disclosures [Abstract] | |
Summary of Marketable Securities | Marketable securities consisted of the following as of September 30, 2016 (in thousands): September 30, 2016 Gross Gross Amortized Unrealized Unrealized Fair Cost Gains Losses Value Short-term available-for-sale securities: Corporate bonds $ 14,982 $ 2 $ (11 ) $ 14,973 Commercial paper 400 — — 400 Total $ 15,382 $ 2 $ (11 ) $ 15,373 Long-term available-for-sale securities: Corporate bonds $ 3,964 $ 6 $ (3 ) $ 3,967 Total $ 3,964 $ 6 $ (3 ) $ 3,967 |
Summary of Amortized Cost and Estimated Fair Value of Available-for-Sale Securities by Contractual Maturity | As of September 30, 2016, the amortized cost and estimated fair value of available-for-sale securities by contractual maturity were as follows (in thousands): Amortized Fair Cost Value Due in one year or less $ 15,382 $ 15,373 Due in one to two years 3,964 3,967 Total $ 19,346 $ 19,340 |
Summary of Assets Measured at Fair Value on Recurring Basis | The following table identifies the Company’s assets that were measured at fair value on a recurring basis (in thousands): Balance Level 1 Level 2 Level 3 September 30, 2016 Money market $ 8,026 $ 8,026 $ — $ — Corporate bonds 18,940 — 18,940 — Commercial paper 400 — 400 — Total $ 27,366 $ 8,026 $ 19,340 $ — December 31, 2015 Money market $ 38,728 $ 38,728 $ — $ — Total $ 38,728 $ 38,728 $ — $ — |
Property and Equipment (Tables)
Property and Equipment (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Property Plant And Equipment [Abstract] | |
Summary of Property and Equipment | Property and equipment consist of the following (in thousands): Estimated Life September 30, 2016 December 31, 2015 Computer equipment 3 years $ 81 $ 90 Lab equipment 5 years 142 142 Furniture and fixtures 5 years 83 91 Computer software 3 years 84 84 Leasehold improvements Lesser of useful life or life of the lease 59 8 449 415 Accumulated depreciation and amortization (380 ) (387 ) Property and equipment, net $ 69 $ 28 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Commitments And Contingencies Disclosure [Abstract] | |
Future Minimum Lease Payments | Below is a summary of the future minimum lease payments committed for the Company’s facility in Westminster, Colorado as of September 30, 2016 (in thousands): Remainder of 2016 $ 7 2017 83 2018 88 2019 83 Total future minimum lease payments $ 261 |
Equity Financings and Warrants
Equity Financings and Warrants (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Equity [Abstract] | |
Summary of Warrants by Year of Expiration | As of September 30, 2016, these warrants, by year of expiration, are summarized below: Year of Expiration Number of Warrants Weighted Average Exercise Price 2016 60,890 $ 87.39 2017 24,124 17.89 2018 963,153 11.77 2019 224,323 15.73 2020 44,299 15.96 2022 2,401,233 6.10 3,718,022 $ 9.67 |
Share-based Compensation (Table
Share-based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Non-cash, Share-based Compensation Expense | For the three and nine month periods ended September 30, 2016 and 2015, the Company recognized the following non-cash, share-based compensation expense in the statements of operations (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Research and development $ 94 $ 42 $ 189 $ 132 General and administrative 173 107 416 322 Total $ 267 $ 149 $ 605 $ 454 |
Summary of Stock Option Activities | Stock option transactions for the nine month period ended September 30, 2016 under the Company’s stock incentive plans were as follows: Number of Options Weighted Average Exercise Price Weighted Average Remaining Contractual Term (in years) Options outstanding at December 31, 2015 161,278 $ 18.30 6.97 Granted 442,300 3.31 Exercised — — Forfeited and cancelled (18,713 ) 28.39 Options outstanding at September 30, 2016 584,865 $ 6.64 9.02 Options exercisable at September 30, 2016 159,887 $ 14.55 7.46 Options vested and expected to vest 583,904 $ 6.65 9.02 |
Summary of RSU Activity | Stock award transactions related to restricted stock units for the nine month period ended September 30, 2016 under the Company’s stock incentive plans were as follows: Number of Shares Weighted Average Grant Date Fair Value Restricted stock units outstanding at December 31, 2015 62,359 $ 8.36 Granted — — Vested and released (29,486 ) 8.78 Forfeited and cancelled (471 ) 4.69 Restricted stock units outstanding at September 30, 2016 32,402 $ 8.02 |
The Company and Summary of Si23
The Company and Summary of Significant Accounting Policies (Details Textual) | Jun. 16, 2015USD ($) | Mar. 31, 2017Patients | Sep. 30, 2016USD ($)Patients |
Company And Summary Of Significant Accounting Policies Table [Line Items] | |||
Revenues | $ | $ 0 | ||
Concentrations of credit risk | $ | $ 0 | ||
Private Investment In Public Entity Transaction | |||
Company And Summary Of Significant Accounting Policies Table [Line Items] | |||
Proceeds from the issuance of security units | $ | $ 34,200,000 | ||
Phase 3 heart failure (HF) trial of Gencaro | BEST Trial | |||
Company And Summary Of Significant Accounting Policies Table [Line Items] | |||
Number of patients | Patients | 2,708 | ||
Phase 2B/Phase 3 | GENETIC-AF Trial | |||
Company And Summary Of Significant Accounting Policies Table [Line Items] | |||
Number of patients company plans to enroll | Patients | 620 | ||
Trial endpoint, term | 168 days | ||
Phase 2B | GENETIC-AF Trial | Forecast | |||
Company And Summary Of Significant Accounting Policies Table [Line Items] | |||
Number of patients company plans to enroll | Patients | 150 |
Net Loss Per Share - Summary of
Net Loss Per Share - Summary of Potentially Dilutive Shares of Common Stock (Details) - shares | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share, Amount | 4,335,289 | 3,982,273 |
Outstanding stock options | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share, Amount | 584,865 | 170,545 |
Unvested restricted stock units | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share, Amount | 32,402 | 71,780 |
Warrants to purchase common stock | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share, Amount | 3,718,022 | 3,739,948 |
Marketable Securities and Fai25
Marketable Securities and Fair Value Disclosures - Summary of Marketable securities (Details) $ in Thousands | Sep. 30, 2016USD ($) |
Schedule of Available-for-sale Securities [Line Items] | |
Amortized Cost | $ 19,346 |
Fair Value | 19,340 |
Short-term available-for-sale securities | |
Schedule of Available-for-sale Securities [Line Items] | |
Amortized Cost | 15,382 |
Gross Unrealized Gains | 2 |
Gross Unrealized Losses | (11) |
Fair Value | 15,373 |
Short-term available-for-sale securities | Corporate bonds | |
Schedule of Available-for-sale Securities [Line Items] | |
Amortized Cost | 14,982 |
Gross Unrealized Gains | 2 |
Gross Unrealized Losses | (11) |
Fair Value | 14,973 |
Short-term available-for-sale securities | Commercial paper | |
Schedule of Available-for-sale Securities [Line Items] | |
Amortized Cost | 400 |
Fair Value | 400 |
Long-term available-for-sale securities | |
Schedule of Available-for-sale Securities [Line Items] | |
Amortized Cost | 3,964 |
Gross Unrealized Gains | 6 |
Gross Unrealized Losses | (3) |
Fair Value | 3,967 |
Long-term available-for-sale securities | Corporate bonds | |
Schedule of Available-for-sale Securities [Line Items] | |
Amortized Cost | 3,964 |
Gross Unrealized Gains | 6 |
Gross Unrealized Losses | (3) |
Fair Value | $ 3,967 |
Marketable Securities and Fai26
Marketable Securities and Fair Value Disclosures - Summary of Amortized Cost and Estimated Fair Value of Available-for-Sale Securities by Contractual Maturity (Details) $ in Thousands | Sep. 30, 2016USD ($) |
Investments Debt And Equity Securities [Abstract] | |
Due in one year or less, Amortized Cost | $ 15,382 |
Due in one to two years, Amortized Cost | 3,964 |
Amortized Cost | 19,346 |
Due in one year or less, Fair Value | 15,373 |
Due in one to two years, Fair Value | 3,967 |
Fair Value | $ 19,340 |
Marketable Securities and Fai27
Marketable Securities and Fair Value Disclosures - Summary of Assets Measured at Fair Value on Recurring Basis (Details) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | $ 27,366 | $ 38,728 |
Corporate bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 18,940 | |
Money market | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 8,026 | 38,728 |
Commercial paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 400 | |
Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 8,026 | 38,728 |
Level 1 | Money market | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 8,026 | $ 38,728 |
Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 19,340 | |
Level 2 | Corporate bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 18,940 | |
Level 2 | Commercial paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | $ 400 |
Marketable Securities and Fai28
Marketable Securities and Fair Value Disclosures (Details Textual) - USD ($) | 9 Months Ended | |
Sep. 30, 2016 | Dec. 31, 2015 | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Transfers of assets between fair value hierarchy levels | $ 0 | |
Fair Value Measurements, Recurring | Level 1 | Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents consisting of money market funds | $ 8,000,000 | $ 38,700,000 |
Property and Equipment - Summar
Property and Equipment - Summary of Property and Equipment (Details) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Dec. 31, 2015 | |
Property Plant And Equipment [Line Items] | ||
Property and Equipment, Gross | $ 449 | $ 415 |
Accumulated depreciation and amortization | (380) | (387) |
Property and equipment, net | $ 69 | 28 |
Computer Equipment | ||
Property Plant And Equipment [Line Items] | ||
Estimated Life | 3 years | |
Property and Equipment, Gross | $ 81 | 90 |
Lab Equipment | ||
Property Plant And Equipment [Line Items] | ||
Estimated Life | 5 years | |
Property and Equipment, Gross | $ 142 | 142 |
Furniture and Fixtures | ||
Property Plant And Equipment [Line Items] | ||
Estimated Life | 5 years | |
Property and Equipment, Gross | $ 83 | 91 |
Computer Software | ||
Property Plant And Equipment [Line Items] | ||
Estimated Life | 3 years | |
Property and Equipment, Gross | $ 84 | 84 |
Leasehold Improvements | ||
Property Plant And Equipment [Line Items] | ||
Estimated Life | Lesser of useful life or life of the lease | |
Property and Equipment, Gross | $ 59 | $ 8 |
Property and Equipment (Details
Property and Equipment (Details Textual) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
Property Plant And Equipment [Abstract] | ||
Depreciation and amortization expense | $ 17 | $ 14 |
Related Party Arrangements (Det
Related Party Arrangements (Details Textual) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
Chief Executive Officer | Research Grants | ||
Related Party Transaction [Line Items] | ||
Total expense | $ 329 | $ 277 |
Commitments and Contingencies32
Commitments and Contingencies (Details Textual) $ in Thousands | Mar. 02, 2016 | Sep. 30, 2016USD ($) | Sep. 30, 2015USD ($) | Aug. 01, 2013ft² |
Commitments And Contingencies [Line Items] | ||||
Area of office facilities taken on lease | ft² | 5,300 | |||
Additional operating lease term | 38 months | |||
Lease renewal start date | Oct. 1, 2016 | |||
Lease expiration date | Nov. 30, 2019 | |||
Rent expense | $ 62 | $ 60 | ||
Royalty buy down due period | 6 months | |||
Minimum | ||||
Commitments And Contingencies [Line Items] | ||||
Percentage of royalty obligation | 12.50% | |||
Percentage of range of royalties that can be bought down | 12.50% | |||
Maximum | ||||
Commitments And Contingencies [Line Items] | ||||
Percentage of royalty obligation | 25.00% | |||
Percentage of range of royalties that can be bought down | 17.00% | |||
Clinical Research Agreement with Duke University | ||||
Commitments And Contingencies [Line Items] | ||||
Agreement termination notice period | 90 days | |||
Upon Food and Drug Administration Approval | ||||
Commitments And Contingencies [Line Items] | ||||
Expected milestone payments due upon approval | $ 8,000 | |||
Upon Regulatory Marketing Approval | ||||
Commitments And Contingencies [Line Items] | ||||
Expected milestone payments due upon approval | $ 5,000 | |||
CPEC | ||||
Commitments And Contingencies [Line Items] | ||||
Percentage of royalty obligation | 5.00% |
Commitments and Contingencies -
Commitments and Contingencies - Future Minimum Lease Payments (Details) $ in Thousands | Sep. 30, 2016USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
Remainder of 2016 | $ 7 |
2,017 | 83 |
2,018 | 88 |
2,019 | 83 |
Total future minimum lease payments | $ 261 |
Equity Financings and Warrant34
Equity Financings and Warrants (Details Textual) - USD ($) $ / shares in Units, $ in Millions | Jun. 16, 2015 | Sep. 30, 2016 | Dec. 31, 2015 |
Equity Financing And Warrants [Line Items] | |||
Common stock, shares issued | 9,080,703 | 9,051,217 | |
Private Investment In Public Entity Transaction | |||
Equity Financing And Warrants [Line Items] | |||
Common stock, shares issued | 6,003,082 | ||
Warrants available to purchase additional commons shares | 2,401,233 | ||
Purchase price for common stock unit | $ 6.1635 | ||
Proceeds from the issuance of security units | $ 34.2 | ||
Warrants exercisable date | Dec. 13, 2015 | ||
Warrants were exercisable upon issuance, expiry date | Jun. 16, 2022 | ||
Warrant price per share | $ 6.1012 | ||
Private Investment In Public Entity Transaction | Security Units | |||
Equity Financing And Warrants [Line Items] | |||
Common stock, shares issued | 6,003,082 |
Equity Financings and Warrant35
Equity Financings and Warrants - Summary of Warrants by Year of Expiration (Details) | 9 Months Ended |
Sep. 30, 2016$ / sharesshares | |
Class Of Warrant Or Right [Line Items] | |
Number of Warrants | shares | 3,718,022 |
Weighted Average Exercise Price | $ / shares | $ 9.67 |
2,016 | |
Class Of Warrant Or Right [Line Items] | |
Year of Expiration | 2,016 |
Number of Warrants | shares | 60,890 |
Weighted Average Exercise Price | $ / shares | $ 87.39 |
2,017 | |
Class Of Warrant Or Right [Line Items] | |
Year of Expiration | 2,017 |
Number of Warrants | shares | 24,124 |
Weighted Average Exercise Price | $ / shares | $ 17.89 |
2,018 | |
Class Of Warrant Or Right [Line Items] | |
Year of Expiration | 2,018 |
Number of Warrants | shares | 963,153 |
Weighted Average Exercise Price | $ / shares | $ 11.77 |
2,019 | |
Class Of Warrant Or Right [Line Items] | |
Year of Expiration | 2,019 |
Number of Warrants | shares | 224,323 |
Weighted Average Exercise Price | $ / shares | $ 15.73 |
2,020 | |
Class Of Warrant Or Right [Line Items] | |
Year of Expiration | 2,020 |
Number of Warrants | shares | 44,299 |
Weighted Average Exercise Price | $ / shares | $ 15.96 |
2,022 | |
Class Of Warrant Or Right [Line Items] | |
Year of Expiration | 2,022 |
Number of Warrants | shares | 2,401,233 |
Weighted Average Exercise Price | $ / shares | $ 6.10 |
Share-based Compensation - Summ
Share-based Compensation - Summary of Non-cash, Share-based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Total expenses | $ 267 | $ 149 | $ 605 | $ 454 |
Research and development | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Total expenses | 94 | 42 | 189 | 132 |
General and administrative | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Total expenses | $ 173 | $ 107 | $ 416 | $ 322 |
Share-based Compensation - Su37
Share-based Compensation - Summary of Stock Option Activities (Details) - $ / shares | 9 Months Ended | 12 Months Ended |
Sep. 30, 2016 | Dec. 31, 2015 | |
Number of Stock Options | ||
Number of Options, Options outstanding, beginning of period | 161,278 | |
Number of Options, Granted | 442,300 | |
Number of Options, Forfeited and cancelled | (18,713) | |
Number of Options, Options outstanding, end of period | 584,865 | 161,278 |
Number of Options, Options exercisable, end of period | 159,887 | |
Number of Options, Options vested and expected to vest | 583,904 | |
Stock Options, Weighted Average Exercise Price | ||
Weighted Average Exercise Price, Options outstanding, beginning of period | $ 18.30 | |
Weighted Average Exercise Price, Granted | 3.31 | |
Weighted Average Exercise Price, Forfeited and cancelled | 28.39 | |
Weighted Average Exercise Price, Options outstanding, end of period | 6.64 | $ 18.30 |
Weighted Average Exercise Price, Options exercisable, end of period | 14.55 | |
Weighted Average Exercise Price, Options vested and expected to vest, end of period | $ 6.65 | |
Stock Options, Weighted Average Remaining Contractual Term | ||
Weighted Average Remaining Contractual Term (in years), Options outstanding | 9 years 7 days | 6 years 11 months 19 days |
Weighted Average Remaining Contractual Term (in years), Options exercisable | 7 years 5 months 16 days | |
Weighted Average Remaining Contractual Term (in years), Options vested and expected to vest | 9 years 7 days |
Share-based Compensation - Su38
Share-based Compensation - Summary of RSU Activity (Details) | 9 Months Ended |
Sep. 30, 2016$ / sharesshares | |
Restricted Stock Awards, Number of Shares | |
Restricted stock units outstanding, beginning of period | shares | 62,359 |
Vested and released | shares | (29,486) |
Forfeited and cancelled | shares | (471) |
Restricted stock units outstanding, end of period | shares | 32,402 |
Restricted Stock Awards, Weighted Average Grant Date Fair Value | |
Restricted stock units outstanding, beginning of period | $ / shares | $ 8.36 |
Vested and released | $ / shares | 8.78 |
Forfeited and cancelled | $ / shares | 4.69 |
Restricted stock units outstanding, end of period | $ / shares | $ 8.02 |
Income Taxes (Details Textual)
Income Taxes (Details Textual) | Sep. 30, 2016USD ($) |
Income Tax Disclosure [Abstract] | |
Reserve for uncertain tax positions | $ 0 |