Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2017 | Mar. 07, 2018 | Jun. 30, 2017 | |
Document and Entity Information | |||
Entity Registrant Name | CATABASIS PHARMACEUTICALS INC | ||
Entity Central Index Key | 1,454,789 | ||
Document Type | 10-K | ||
Document Period End Date | Dec. 31, 2017 | ||
Amendment Flag | false | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Public Float | $ 24,632,957 | ||
Entity Common Stock, Shares Outstanding | 29,035,502 | ||
Document Fiscal Year Focus | 2,017 | ||
Document Fiscal Period Focus | FY |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Current assets: | ||
Cash and cash equivalents | $ 16,369 | $ 23,596 |
Available-for-sale securities | 14,931 | |
Prepaid expenses and other current assets | 1,094 | 1,001 |
Total current assets | 17,463 | 39,528 |
Property and equipment, net | 321 | 568 |
Restricted cash | 113 | 113 |
Total assets | 17,897 | 40,209 |
Current liabilities: | ||
Accounts payable | 773 | 1,405 |
Accrued expenses | 2,432 | 3,677 |
Current portion of notes payable, net of discount | 2,479 | 3,243 |
Other liability | 332 | |
Total current liabilities | 6,016 | 8,325 |
Deferred rent, net of current portion | 89 | 53 |
Notes payable, net of current portion and discount | 2,479 | |
Other liability | 266 | |
Total liabilities | 6,105 | 11,123 |
Commitments (Note 9) | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value per share, 5,000,000 shares authorized and no shares issued and outstanding | ||
Common stock, $0.001 par value per share, 150,000,000 shares authorized; 23,645,247 and 18,817,572 shares issued and outstanding at December 31, 2017 and 2016, respectively | 24 | 19 |
Additional paid-in capital | 183,202 | 173,141 |
Accumulated other comprehensive loss | (4) | |
Accumulated deficit | (171,434) | (144,070) |
Total stockholders' equity | 11,792 | 29,086 |
Total liabilities and stockholders' equity | $ 17,897 | $ 40,209 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2017 | Dec. 31, 2016 |
Consolidated Balance Sheets | ||
Preferred stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred stock, authorized (in Shares) | 5,000,000 | 5,000,000 |
Preferred stock, issued (in shares) | 0 | 0 |
Preferred stock, outstanding (in shares) | 0 | 0 |
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, authorized (in shares) | 150,000,000 | 150,000,000 |
Common stock, issued (in shares) | 23,645,247 | 18,817,572 |
Common stock, outstanding (in shares) | 23,645,247 | 18,817,572 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Consolidated Statements of Operations | |||
Revenue | $ 500 | ||
Operating expenses: | |||
Research and development | 18,682 | $ 25,450 | $ 23,030 |
General and administrative | 8,912 | 10,108 | 8,629 |
Total operating expenses | 27,594 | 35,558 | 31,659 |
Loss from operations | (27,094) | (35,558) | (31,659) |
Other (expense) income: | |||
Interest expense | (462) | (837) | (978) |
Interest and investment income | 160 | 242 | |
Other income, net | 32 | 93 | 7 |
Total other expense, net | (270) | (502) | (971) |
Net loss | $ (27,364) | $ (36,060) | $ (32,630) |
Net loss per share - basic and diluted (in dollars per share) | $ (1.26) | $ (2.22) | $ (4.06) |
Weighted-average common shares outstanding used in net loss per share - basic and diluted (in shares) | 21,681,534 | 16,230,190 | 8,041,948 |
Consolidated Statements Compreh
Consolidated Statements Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Consolidated Statements Comprehensive Loss | |||
Net loss | $ (27,364) | $ (36,060) | $ (32,630) |
Other comprehensive income (loss): | |||
Gain (loss) on available-for-sale securities | 4 | (4) | |
Total other comprehensive income (loss): | 4 | (4) | |
Comprehensive loss | $ (27,360) | $ (36,064) | $ (32,630) |
Consolidated Statements of Conv
Consolidated Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit) - USD ($) $ in Thousands | Series A Convertible Preferred Stock | Series B Convertible Preferred Stock | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Loss | Total |
Balance at Dec. 31, 2014 | $ 47,898 | $ 32,248 | |||||
Balance (in shares) at Dec. 31, 2014 | 68,837,703 | 34,129,571 | |||||
Increase (decrease) in Convertible Preferred Stock | |||||||
Issuance of series B convertible preferred stock, net of issuance cost of $0.1 million | $ 12,331 | ||||||
Issuance of series B convertible preferred stock, net of issuance cost of $0.1 million (in shares) | 13,062,965 | ||||||
Conversion of convertible preferred stock into common stock | $ (47,898) | $ (44,579) | |||||
Conversion of convertible preferred stock into common stock (in shares) | (68,837,703) | (47,192,536) | |||||
Balance at Dec. 31, 2014 | $ 1 | $ 2,326 | $ (75,380) | $ (73,053) | |||
Balance (in shares) at Dec. 31, 2014 | 493,200 | ||||||
Increase (Decrease) in Stockholders' Equity (Deficit) | |||||||
Issuance of common stock from initial public offering/for registered direct and at-the-market offerings, net of issuance costs of $0.6 million, $1.1 million and $7.3 million for the years 2017, 2016 and 2015 respectively | $ 5 | 61,739 | 61,744 | ||||
Issuance of common stock from initial public offering/for registered direct and at-the-market offerings, net of issuance costs of $0.6 million, $1.1 million and $7.3 million for the years 2017, 2016 and 2015 respectively (in shares) | 5,750,000 | ||||||
Conversion of convertible preferred stock into common stock | $ 9 | 92,468 | 92,477 | ||||
Conversion of convertible preferred stock into common stock (in shares) | 9,029,549 | ||||||
Conversion of series B preferred stock warrants into warrants for the purchase of common stock | 206 | 206 | |||||
Proceeds from exercises of common stock options | 91 | 91 | |||||
Proceeds from exercises of common stock options (in shares) | 40,548 | ||||||
Stock-based compensation expense | 1,658 | 1,658 | |||||
Net loss | (32,630) | (32,630) | |||||
Balance at Dec. 31, 2015 | $ 15 | 158,488 | (108,010) | 50,493 | |||
Balance (in shares) at Dec. 31, 2015 | 15,313,297 | ||||||
Increase (Decrease) in Stockholders' Equity (Deficit) | |||||||
Issuance of common stock from initial public offering/for registered direct and at-the-market offerings, net of issuance costs of $0.6 million, $1.1 million and $7.3 million for the years 2017, 2016 and 2015 respectively | $ 3 | 11,991 | 11,994 | ||||
Issuance of common stock from initial public offering/for registered direct and at-the-market offerings, net of issuance costs of $0.6 million, $1.1 million and $7.3 million for the years 2017, 2016 and 2015 respectively (in shares) | 3,243,015 | ||||||
Proceeds from exercises of common stock options and warrants | $ 1 | 502 | 503 | ||||
Proceeds from exercises of common stock options and warrants (in shares) | 261,260 | ||||||
Stock-based compensation expense | 2,160 | 2,160 | |||||
Unrealized losses on short-term investments | $ (4) | (4) | |||||
Net loss | (36,060) | (36,060) | |||||
Balance at Dec. 31, 2016 | $ 19 | 173,141 | (144,070) | (4) | $ 29,086 | ||
Balance (in shares) at Dec. 31, 2016 | 18,817,572 | 18,817,572 | |||||
Increase (Decrease) in Stockholders' Equity (Deficit) | |||||||
Issuance of common stock from initial public offering/for registered direct and at-the-market offerings, net of issuance costs of $0.6 million, $1.1 million and $7.3 million for the years 2017, 2016 and 2015 respectively | $ 5 | 8,031 | $ 8,036 | ||||
Issuance of common stock from initial public offering/for registered direct and at-the-market offerings, net of issuance costs of $0.6 million, $1.1 million and $7.3 million for the years 2017, 2016 and 2015 respectively (in shares) | 4,804,796 | ||||||
Proceeds from exercises of common stock options | 23 | $ 23 | |||||
Proceeds from exercises of common stock options (in shares) | 22,879 | 22,879 | |||||
Stock-based compensation expense | 2,007 | $ 2,007 | |||||
Realized gain on short-term investments | $ 4 | 4 | |||||
Net loss | (27,364) | (27,364) | |||||
Balance at Dec. 31, 2017 | $ 24 | $ 183,202 | $ (171,434) | $ 11,792 | |||
Balance (in shares) at Dec. 31, 2017 | 23,645,247 | 23,645,247 |
Consolidated Statements of Con7
Consolidated Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit) (Parenthetical) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Series B Convertible Preferred Stock | |||
Stock issuance costs | $ 0.1 | ||
Initial public offering | |||
Stock issuance costs | $ 0.6 | $ 1.1 | $ 7.3 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Operating activities | |||
Net loss | $ (27,364) | $ (36,060) | $ (32,630) |
Reconciliation of net loss to net cash used in operating activities: | |||
Depreciation and amortization | 304 | 395 | 202 |
Stock-based compensation expense | 2,007 | 2,160 | 1,658 |
Accretion of discount/premium on investment securities | 26 | 148 | |
Non-cash interest expense | 155 | 277 | 293 |
Gain on sale of fixed assets | (30) | (52) | |
Changes in assets and liabilities: | |||
Prepaid expenses and other current assets | (93) | (229) | (450) |
Other assets | 2 | ||
Accounts payable | (632) | 77 | 196 |
Accrued expenses | (1,215) | 418 | 954 |
Deferred rent | 6 | 8 | (18) |
Net cash used in operating activities | (26,836) | (32,858) | (29,793) |
Investing activities | |||
Purchases of available-for-sale securities | (45,539) | ||
Sales and maturities of available-for-sale securities | 14,910 | 30,456 | |
Purchases of property and equipment | (57) | (459) | (421) |
Sale of property and equipment | 30 | 52 | |
Net cash provided by (used in) investing activities | 14,883 | (15,490) | (421) |
Financing activities | |||
Proceeds from initial public offering, net of issuance costs | 61,744 | ||
Proceeds from issuance of preferred stock, net of issuance costs | 12,331 | ||
Proceeds from registered direct offering, net of issuance costs | 10,603 | ||
Proceeds from at-the-market offering, net of issuance costs | 8,036 | 1,391 | |
Proceeds from exercise of common stock options and warrants | 23 | 503 | 91 |
Proceeds from borrowing | 5,000 | ||
Payments on borrowing | (3,333) | (3,333) | (833) |
Debt issuance costs | (7) | ||
Net cash provided by financing activities | 4,726 | 9,164 | 78,326 |
Net (decrease) increase in cash and cash equivalents | (7,227) | (39,184) | 48,112 |
Cash and cash equivalents, beginning of period | 23,596 | 62,780 | 14,668 |
Cash and cash equivalents, end of period | 16,369 | 23,596 | 62,780 |
Supplemental disclosure of cash flow information | |||
Cash paid for interest | $ 327 | $ 582 | 684 |
Non-cash financing activities | |||
Warrants for the purchase of series B preferred stock issued in conjunction with credit facility | 107 | ||
Initial public offering costs in accounts payable and accrued liabilities | (492) | ||
Reclassification of deferred IPO costs from non-current assets to additional paid-in capital | 1,787 | ||
Reclassification of warrant liability to additional paid-in capital | $ 206 |
Organization and Operations
Organization and Operations | 12 Months Ended |
Dec. 31, 2017 | |
Organization and Operations | |
Organization and Operations | 1. Organization and Operations The Company Catabasis Pharmaceuticals, Inc. (the "Company") is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics based on the Company's proprietary Safely Metabolized And Rationally Targeted, or SMART, linker, or SMART Linker SM , drug discovery platform. The Company's SMART Linker technology platform enables the Company to engineer product candidates that can simultaneously modulate multiple targets in a disease. The Company's proprietary product candidates impact pathways that are central to diseases where efficacy may be optimized by a multiple target approach. The Company's primary focus is on treatments for rare diseases. The Company has applied its SMART Linker drug discovery platform to build an internal pipeline of product candidates for rare diseases and plans to pursue partnerships to develop additional product candidates. The Company was incorporated in the State of Delaware on June 26, 2008. Liquidity and Going Concern In August 2016, the Company entered into a sales agreement with Cowen and Company LLC, ("Cowen"), pursuant to which the Company could issue and sell shares of common stock for an aggregate maximum offering amount of $10.0 million under an at-the-market offering program (the "First ATM Program"). In March 2017, the Company reduced the maximum amount of the offering under the First ATM Program to $8.8 million, which was fully utilized as of September 30, 2017. In October 2017, the Company entered into a second sales agreement with Cowen pursuant to which the Company may issue and sell shares of common stock for an aggregate maximum offering amount of $10.0 million under a second at-the-market offering program (the "Second ATM Program"). Cowen is not required to sell any specific amount, but acts as the Company's sales agent using commercially reasonable efforts consistent with its normal trading and sales practices. Shares sold pursuant to the sales agreement have been sold pursuant to a shelf registration statement, which became effective on July 19, 2016. The Company pays Cowen 3% of the gross proceeds from any common stock sold through these sales agreements. During the year ended December 31, 2017, the Company sold an aggregate of 4,804,796 shares of common stock pursuant to the First ATM Program, at an average price of $1.79 per share, for gross proceeds of $8.6 million, resulting in net proceeds of $8.0 million after deducting sales commissions and offering. As of December 31, 2017, $8.6 million of common stock remained available for sale under the Second ATM Program. Subsequent to December 31, 2017, the Company sold an aggregate of 5,390,255 shares of common stock pursuant to the Second ATM Program, at an average price of $1.59 per share, for gross proceeds of $8.6 million, representing the remainder of the common stock that could be sold under the Second ATM Program, resulting in net proceeds of $8.3 million after deducting sales commissions and offering expenses, thus the Second ATM Program has been fully utilized. During the year ended December 31, 2016, the Company sold an aggregate of 3,243,015 shares of common stock either through a direct offering or an at-the-market offering at an average offering price of $4.04 per share. The shares sold in the offering were sold pursuant to the Shelf Registration Statement. The Company received aggregate gross proceeds from the sales of $13.1 million, resulting in net proceeds of $12.0 million after deducting underwriting discounts and commissions and offering expenses. As of December 31, 2017, the Company had an accumulated deficit of $171.4 million. The Company has been primarily involved with research and development activities and has incurred operating losses and negative cash flows from operations since its inception. The Company is subject to a number of risks including, but not limited to, the need to obtain adequate additional funding, including the resources necessary to fund a global Phase 3 clinical trial of edasalonexent in DMD, which the Company plans to begin in the first half of 2018. The Company is currently exploring various avenues to secure capital to advance edasalonexent into a Phase 3 clinical trial, and its Phase 3 development activities will be dependent on securing adequate capital resources. The Company is also subject to a number of risks similar to other life science companies, including, but not limited to, successful discovery and development of its drug candidates, raising additional capital, development by its competitors of new technological innovations, protection of proprietary technology and regulatory approval and market acceptance of the Company's products. The Company anticipates that it will continue to incur significant operating losses for the next several years as it continues to develop its product candidates. As of December 31, 2017, the Company had available cash and cash equivalents of $16.4 million. The Company's current operating plan provides for cash to fund operations through September 2018. Based on the Company's available cash resources, the Company does not have sufficient cash on hand to support current operations for at least the next twelve months from the date of filing this Annual Report on Form 10-K. This condition requires substantial doubt about the Company's ability to continue as a going concern. The Company's plans to address this condition include seeking additional funds through equity or debt financings or through collaboration or licensing transactions. The Company may be unable to obtain equity or debt financings or enter into collaboration or licensing transactions and, if necessary, the Company will be required to implement cost reduction strategies, including ceasing or delaying development of the Company's product candidates. Accordingly, the Company has concluded that substantial doubt exists regarding the Company's ability to continue as a going concern for a period of at least twelve months from the date of issuance of these consolidated financial statements. The accompanying consolidated financial statements have been prepared on a going concern basis, which contemplates the realization of assets and satisfaction of liabilities in the ordinary course of business. The consolidated financial statements do not include any adjustments relating to the recoverability and classification of recorded asset amounts or the amounts and classification of liabilities that might result from the outcome of this uncertainty. The Company will require substantial additional capital to fund operations. The Company has not generated any product revenues and has financed its operations primarily through public offerings and private placements of its equity securities. There can be no assurance that the Company will be able to obtain additional debt or equity financing or generate product revenue or revenues from collaborative partners, on terms acceptable to the Company, on a timely basis or at all. The failure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company's business, results of operations, and financial condition. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2017 | |
Summary of Significant Accounting Policies | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation and Principles of Consolidation The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiary, Catabasis Securities Corporation. All intercompany balances and transactions have been eliminated in consolidation. These consolidated financial statements have been prepared in accordance with United States generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented. Use of Estimates The preparation of the Company's consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. Actual results could differ from such estimates. The Company utilizes certain estimates to record expenses relating to research and development contracts. These contract estimates, which are primarily related to the length each contract and the amount of service provided as of each measurement date, are determined by the Company based on input from internal project management, as well as from third-party service providers. Off-Balance Sheet Risk and Concentrations of Credit Risk The Company has no off-balance sheet risk, such as foreign exchange contracts, option contracts or other foreign hedging arrangements. Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and restricted cash. The primary objectives for the Company's investment portfolio are the preservation of capital and the maintenance of liquidity. The Company's investment policy includes guidelines on the quality of the institutions and financial instruments and defines allowable investments that the Company believes minimizes the exposure to concentration of credit risk. Cash and Cash Equivalents The Company considers highly liquid investments with a maturity of three months or less when purchased to be cash equivalents. Cash equivalents, which consist primarily of money market funds backed by U.S. government securities, are stated at fair value. Cash and cash equivalents consist of the following (in thousands): December 31, 2017 2016 Cash $ $ Money market fund ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Available-for-Sale Investments The Company classifies all short-term investments with a remaining maturity of greater than three months at the time of purchase as available-for-sale. Available-for-sale securities are recorded at fair value, with the unrealized gains and losses reported in other comprehensive loss. The amortized cost of debt securities is adjusted for the amortization of premiums and accretion of discounts to maturity. Such amortization is included in interest and investment income. Realized gains and losses, interest, dividends and declines in value judged to be other-than-temporary are included in interest and investment income. The cost of securities sold is based on the specific identification method for purposes of recording realized gains and losses. To determine whether an other-than-temporary impairment exists, the Company considers whether it has the ability and intent to hold the investment until a market price recovery, and whether evidence indicating the recoverability of the cost of the investment outweighs evidence to the contrary. Fair Value of Financial Instruments The fair value hierarchy prioritizes the inputs to valuation techniques used to measure fair value into three broad levels as follows: Level 1 inputs are quoted prices (unadjusted) in active markets for identical assets or liabilities; Level 2 inputs are inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly; and Level 3 inputs are unobservable inputs that reflect the Company's own assumptions about the assumptions market participants would use in pricing the asset or liability. Financial assets and liabilities are classified in their entirety based on the lowest level of input that is significant to the fair value measurement. The carrying amounts reflected in the balance sheets for cash equivalents, restricted cash, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values at December 31, 2017 and 2016, due to their short-term nature. There have been no changes to the valuation methods during the years ended December 31, 2017 and 2016. The Company evaluates transfers between levels at the end of each reporting period. There were no transfers of assets or liabilities between levels during the year ended December 31, 2017 and 2016. At December 31, 2017, the carrying value of the Company's debt approximated fair value, which was determined using Level 3 inputs, including a quoted interest rate. The Company's investment portfolio may include fixed income securities that do not always trade on a daily basis. As a result, the pricing services used by the Company apply other available information as applicable through processes such as benchmark yields, benchmarking of like securities, sector groupings and matrix pricing to prepare valuations. The Company validates the prices provided by its third party pricing services by obtaining market values from other pricing sources and analyzing pricing data in certain instances. The Company determines the fair value of available-for-sale securities (Note 4) using Level 2 inputs. Deferred Financing Costs Deferred financing costs include costs directly attributable to the Company's offerings of its equity securities and its debt financings. Costs attributable to equity offerings are charged against the proceeds of the offering once the offering is completed. Costs attributable to debt financings are deferred and amortized over the term of the debt using the effective interest rate method. Property and Equipment Property and equipment consist of laboratory equipment, computer equipment, leasehold improvements and furniture and fixtures. Property and equipment is stated at cost and depreciated using the straight-line method over the estimated useful lives of the respective assets. Costs of major additions and betterments are capitalized; maintenance and repairs, which do not improve or extend the life of the respective assets, are charged to expense as incurred. Upon retirement or sale, the cost of the disposed asset and the related accumulated depreciation are removed from the accounts and the resulting gain or loss is recognized. Impairment of Long-Lived Assets The Company continually evaluates whether events or circumstances have occurred that indicate that the estimated remaining useful life of its long-lived assets may warrant revision or that the carrying value of these assets may be impaired. The Company has not recognized any significant impairment charges from inception through December 31, 2017. Research and Development Expenses Research and development costs are expensed as incurred. Research and development costs include salaries and personnel-related costs, stock-based compensation, consulting fees, fees paid for contract research services, the costs of laboratory equipment and facilities and other external costs. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred. The deferred amounts are expensed as the related goods are delivered or the services are performed. Revenue Recognition Effective January 1, 2017, the Company adopted Accounting Standards Codification ("ASC"), Revenue from Contracts with Customers, ("ASC 606"). This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, collaboration arrangements and financial instruments. Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. In determining the appropriate amount of revenue to be recognized as the Company fulfills its obligations under the agreement, the Company performs the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The Company only applies the five-step model to contracts when it is probable that it will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations, and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. Stock-Based Compensation The Company accounts for its stock-based compensation awards in accordance with ASC Topic 718, Compensation—Stock Compensation ( "ASC 718"). ASC 718 requires all share-based payments to employees, including grants of employee stock options, to be recognized in the statements of operations based on their grant date fair values. For stock options granted to employees and to members of the board of directors for their services on the board of directors, the Company estimates the grant date fair value of each option award using the Black-Scholes option-pricing model. The use of the Black-Scholes option-pricing model requires management to make assumptions with respect to the expected term of the option, the expected volatility of the Common Stock consistent with the expected term of the option, risk-free interest rates and expected dividend yields of the Common Stock. For awards subject to service-based vesting conditions, the Company recognizes stock-based compensation expense equal to the grant date fair value of stock options on a straight-line basis over the requisite service period. The Company expenses restricted stock awards based on the fair value of the award on a straight-line basis over the associated service period of the award. Share-based payments issued to non-employees are recorded at their fair values, and are periodically revalued as the equity instruments vest and are recognized as expense over the related service period in accordance with the provisions of ASC Topic 505, Equity . For equity instruments granted to non-employees, the Company recognizes stock-based compensation expense on a straight-line basis. During the years ended December 31, 2017, 2016 and 2015, the Company recorded stock-based compensation expense for employee and non-employee stock options and restricted stock, which was allocated as follows in the statements of operations (in thousands): Year Ended December 31, 2017 2016 2015 Research and development $ $ $ General and administrative ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ No related tax benefits were recognized for the years ended December 31, 2017, 2016 and 2015. Grant Awards In the years ended December 31, 2017, 2016 and 2015, the Company received $124,000, $376,000, $100,000, respectively, in grants from the Muscular Dystrophy Association, Friedreich's Ataxia Research Alliance and Parent Project for Muscular Dystrophy. In the years ended December 31, 2017, 2016, 2015 the Company utilized $205,000, $224,000 and $100,000 of the grants received to offset related expenses incurred in the Company's statements of operations. Net Loss Per Share Basic net loss per share is calculated by dividing net loss by the weighted average shares outstanding during the period, without consideration for Common Stock equivalents. Diluted net loss per share is calculated by adjusting weighted average shares outstanding for the dilutive effect of Common Stock equivalents outstanding for the period, determined using the treasury-stock method. For purposes of the dilutive net loss per share calculation, Preferred Stock, stock options, warrants to purchase Common Stock and warrants to purchase Preferred Stock are considered to be Common Stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented. The following Common Stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect: Year Ended December 31, 2017 2016 2015 Stock options Common stock warrants ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Income Taxes The Company provides deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the Company's financial statement carrying amounts and the tax basis of assets and liabilities using enacted tax rates expected to be in effect in the years in which the differences are expected to reverse. A valuation allowance is provided to reduce the deferred tax assets to the amount that will more likely than not be realized. The Company accounts for uncertain tax positions in accordance with the provisions of ASC Topic 740 , Expenses—Income Taxes . When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. The Company did not have any significant uncertain tax positions for any periods presented. Segment Information Operating segments are identified as components of an enterprise for which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision making group, in making decisions on how to allocate resources and assess performance. The Company views its operations and manages its business in one operating segment. The Company operates in one geographic segment. Comprehensive Loss Comprehensive loss is defined as the change in equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. For the years ended December 31, 2017 and 2016, amounts in accumulated other comprehensive loss were comprised of unrealized gains and losses on available-for-sale securities. For the year ended December 31, 2015, comprehensive loss was equal to net loss. Recent Accounting Pronouncements—Adopted In May 2014, the Financial Accounting Standards Board ("FASB") issued ASU 2014-09, Revenue from Contracts with Customers , ("ASU 2014-09"). The standard replaced existing revenue recognition standards and significantly expanded the disclosure requirements for revenue arrangements. The Company adopted the standard on January 1, 2017 using the full retrospective method. The adoption of the new standard did not have an effect on previously issued consolidated financial statements as the Company had not recognized any revenue prior to 2017. In March 2016, the FASB issued ASU 2016-09, Improvements to Employee Share-Based Payment Accounting , ("ASU 2016-09"). This standard amended the existing guidance in an attempt to simplify several aspects of accounting for employee share-based payment transactions, including the accounting for income taxes, forfeitures, and statutory tax withholding requirements, as well as classification in the statement of cash flows. The Company adopted the standard on January 1, 2017. The amended guidance eliminates the requirement that excess tax benefits be realized as a reduction in current taxes payable before the associated tax benefit can be recognized in additional paid-in capital. This created approximately $0.2 million of deferred tax assets relating to federal and state net operating losses that were fully offset by a corresponding increase in the valuation allowance. As a result, there was no cumulative effect adjustment to accumulated deficit. As part of the adoption of ASU 2016-09, the Company elected to record forfeitures as they occur, which did not have a material impact on the consolidated financial statements upon adoption. In May 2017, the FASB issued ASU 2017-09, Scope of Modification Accounting , ("ASU 2017-09"). This standard provides clarity on whether the change in existing terms and conditions of a share-based award require application of the guidance in ASC Topic 718, Compensation—Stock Compensation. The Company early adopted the standard in the period ending June 30, 2017. As of December 31, 2017, there was no material impact on the Company's consolidated financial statements, and any future impact from adoption will be dependent on future transactions involving modifications of share-based awards. Recent Accounting Pronouncements—Not Yet Adopted From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its financial position or results of operations upon adoption. In February 2016, the FASB issued ASU 2016-02, Leases , ("ASU 2016-02"). This standard amends the existing guidance to require lessees to present most leases on their balance sheets but recognize corresponding expenses on their statements of operations. It is effective for annual reporting periods beginning after December 15, 2018, but early adoption is permitted. The Company is currently evaluating the impact that this standard will have on its consolidated financial statements. |
Financial Instruments
Financial Instruments | 12 Months Ended |
Dec. 31, 2017 | |
Financial Instruments | |
Financial Instruments | 3. Financial Instruments The following tables present information about the Company's financial assets and liabilities that have been measured at fair value, and indicates the fair value hierarchy of the valuation inputs utilized to determine such fair value. Below is a summary of assets and liabilities measured at fair value on a recurring basis (in thousands): As of December 31, 2017 Quoted Prices Significant Significant Total Assets: Cash and cash equivalents: Money market funds $ $ — $ — $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total assets $ $ — $ — $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2016 Quoted Prices Significant Significant Total Assets: Cash and cash equivalents: Money market funds $ $ — $ — $ Available-for-sale securities: Corporate debt securities — — U.S. government-sponsored securities — — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total assets $ $ $ — $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2017 and December 31, 2016, the Company's cash equivalents consisted principally of money market funds, which approximated their fair value due to their short-term nature. |
Available-for-Sale Securities
Available-for-Sale Securities | 12 Months Ended |
Dec. 31, 2017 | |
Available-for-Sale Securities | |
Available-for-Sale Securities | 4. Available-for-Sale Securities As of December 31, 2017, the Company held no available-for-sale securities. The following table summarizes the available-for-sale securities held at December 31, 2016 (in thousands): Amortized Gross Gross Fair Value December 31, 2016 Corporate debt securities $ $ — $ ) $ U.S. government-sponsored securities — — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ — $ ) $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ The contractual maturities of all available-for-sale securities held at December 31, 2016 were one year or less. There were fifteen available-for-sale securities in an unrealized loss position at December 31, 2016, none of which had been in an unrealized loss position for more than 12 months. The aggregate fair value of these securities at December 31, 2016 was approximately $13.0 million. The Company did not hold any securities with other-than-temporary impairment at December 31, 2016. Gross realized gains and losses on the sales of available-for-sale securities are included in other income, net. Unrealized holding gains or losses for the period that have been included in accumulated other comprehensive income, as well as gains and losses reclassified out of accumulated other comprehensive income into other income, net were not material to the Company's condensed consolidated results of operations. The cost of securities sold or the amount reclassified out of the accumulated other comprehensive income into other income, net is based on the specific identification method for purposes of recording realized gains and losses. During the year ended December 31, 2016 the Company received $7.8 million in proceeds from sales of available-for-sale securities. There were no proceeds from sales of available-for-sale securities in the year ended December 31, 2017, as all proceeds related to maturities of underlying securities. The gains on proceeds from sales and maturities of available-for-sale securities were not material to the Company's condensed consolidated results of operations for the years ended December 31, 2017 and 2016. |
Restricted Cash
Restricted Cash | 12 Months Ended |
Dec. 31, 2017 | |
Restricted Cash | |
Restricted Cash | 5. Restricted Cash At December 31, 2017 and 2016, the Company had an outstanding letter of credit for $0.1 million as a security deposit for its operating lease agreement for office space (Note 9). The Company is required to maintain this deposit for the duration of the lease agreement. |
Property and Equipment
Property and Equipment | 12 Months Ended |
Dec. 31, 2017 | |
Property and Equipment | |
Property and Equipment | 6. Property and Equipment Property and equipment and related accumulated depreciation were as follows (in thousands): December 31, Estimated Useful Life (Years) 2017 2016 Lab equipment 3 $ $ Computer equipment 3 Furniture and fixtures 5 Leasehold improvements Lesser of useful life or remaining lease term ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Less accumulated depreciation and amortization ) ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total property and equipment, net $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Depreciation and amortization expense was $0.3 million, $0.4 million and $0.2 million for the years ended December 31, 2017, 2016 and 2015, respectively. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2017 | |
Accrued Expenses | |
Accrued Expenses | 7. Accrued Expenses Accrued expenses consisted of the following (in thousands): December 31, 2017 2016 Accrued compensation $ $ Accrued contracted research costs Accrued professional fees Accrued other ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Notes Payable
Notes Payable | 12 Months Ended |
Dec. 31, 2017 | |
Notes Payable | |
Notes Payable | 8. Notes Payable On August 27, 2014, the Company entered into a credit facility with MidCap Financial Trust, Flexpoint MCLS SPV LLC and Square 1 Bank, which was subsequently amended in March and December 2015 (as amended, the "Credit Facility"). The Credit Facility provided for initial borrowings of $5.0 million under a term loan ("Term Loan A") and additional borrowings of up to $20.0 million under other term loans, for a maximum of $25.0 million. On August 27, 2014, the Company received proceeds of $5.0 million from the issuance of promissory notes under Term Loan A. On March 31, 2015, the Company received proceeds of $5.0 million from the issuance of promissory notes under another term loan ("Term Loan B"). The remaining amounts available for borrowing under this arrangement expired unused as of July 31, 2015, leaving total borrowings under the Credit Facility at $10.0 million. All amounts outstanding under the Credit Facility are due on October 1, 2018 and are collateralized by substantially all of the Company's personal property, other than its intellectual property. Interest-only payments were due monthly on amounts outstanding under the Credit Facility until September 1, 2015 and, thereafter, interest and principal payments are due in 36 equal monthly installments from October 1, 2015 through September 1, 2018. Amounts due under the Credit Facility bear interest at an annual rate of 7.49%. In addition, a final payment equal to 3.48% of any amounts drawn under the Credit Facility is due upon the earlier of the maturity date, acceleration of the term loans or prepayment of all or part of the term loans. The final payment is being accrued as additional interest expense using the effective-interest method from the date of issuance through the maturity date, and is recorded within other current liabilities. In the event of prepayment, the Company is obligated to pay 1% to 3% of the amount of the outstanding principal depending upon the timing of the prepayment. As of December 31, 2017, the Company would be obligated to pay 1% of the amount of the outstanding principal in the event of a prepayment. The effective interest rate as of December 31, 2017 was 11.2%. In conjunction with Term Loan A, the Company issued warrants (the "2014 Warrants") to purchase 157,844 shares of series B convertible preferred stock. In conjunction with Term Loan B, the Company issued warrants (the "2015 Warrants") to purchase an additional 157,844 shares of series B convertible preferred stock. Upon the closing of the Company's initial public offering (the "IPO") in June 2015, the warrants to purchase shares of convertible preferred stock to the lenders were automatically converted into warrants to purchase an aggregate of 24,566 shares of Common Stock at an exercise price of $12.21 per share. The warrants were exercisable immediately and expire after seven-years. At issuance, the warrants were valued at $0.2 million using the Black-Scholes option-pricing model. They were recorded as debt discounts of $0.2 million and are being accreted as interest expense using the effective-interest method over the remaining term of the loans. There are no financial covenants associated with the Credit Facility; however, there are negative covenants restricting the Company's activities, including limitations on asset dispositions, mergers or acquisitions; encumbering or granting a security interest in its intellectual property; incurring indebtedness or liens; paying dividends; making certain investments; and entering into certain other business transactions. Upon the occurrence and continuation of an event of default, the lenders have the right to exercise certain remedies against the Company and the collateral securing the loans under the Credit Facility, including cash. Events of default include, among other things, failure to pay amounts due under the Credit Facility, insolvency, the occurrence of a material adverse event, which includes a material adverse change in the business, operations or conditions (financial or otherwise) of the Company or a material impairment of the prospect of repayment of any portion of the obligations, the occurrence of any default under certain other indebtedness and a final judgment against the Company in an amount greater than $250,000. The occurrence of a material adverse event could result in acceleration of the payment of the debt. At December 31, 2016, the Company concluded that the likelihood of the acceleration of the debt was remote, as a material adverse event had not occurred and was unlikely to occur and therefore the debt was classified in current and long-term liabilities based on scheduled principal payments. At December 31, 2017, the maturity date of the debt was within twelve months and therefore all debt was classified as current. Following the occurrence and during the continuance of an event of default, borrowings under the Credit Facility shall bear interest at a rate per annum, which is five hundred basis points, or 5.00%, above the rate that is otherwise applicable. The Company assessed all terms and features of the Credit Facility in order to identify any potential embedded features that would require bifurcation or any beneficial conversion features. As part of this analysis, the Company assessed the economic characteristics and risks of the Credit Facility, including put and call features. The Company determined that all features of the Credit Facility were clearly and closely associated with a debt host and did not require bifurcation as a derivative liability, or the fair value of the feature was immaterial to the Company's financial statements. The Company reassesses the features on a quarterly basis to determine if they require separate accounting. Estimated future principal payments at December 31, 2017 are as follows (in thousands): Year Ending December 31, Amount 2018 ​ ​ ​ ​ ​ Total $ Less: discount for warrants and costs paid to lenders ) Less: current portion ) ​ ​ ​ ​ ​ Note payable, net of current portion and discount $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ During the years ended December 31, 2017, 2016 and 2015, the Company recognized $0.5 million, $0.8 million and $1.0 million, respectively, of interest expense related to the Credit Facility. |
Commitments
Commitments | 12 Months Ended |
Dec. 31, 2017 | |
Commitments | |
Commitments | 9. Commitments In November 2010, the Company entered into an operating lease for office and laboratory space, which has been amended multiple times. Based on the latest amendment, the lease agreement includes escalating rent payments and is effective through June 30, 2020. The Company is recognizing rent expense on a straight-line basis over the lease term. Future minimum payments required under the non-cancelable operating lease as of December 31, 2017 are summarized as follows (in thousands): Period Ending December 31, Amount 2018 2019 2020 ​ ​ ​ ​ ​ Total minimum lease payments $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Rent expense for the years ended December 31, 2017, 2016 and 2015 was $1.3 million, $0.9 million and $0.8 million, respectively. |
Revenue Recognition
Revenue Recognition | 12 Months Ended |
Dec. 31, 2017 | |
Revenue Recognition | |
Revenue Recognition | 10. Revenue Recognition In August 2017, the Company entered into an option agreement ("Option Agreement") with an unaffiliated party ("Recipient"), which is within the scope of ASC 606. Under the terms of the Option Agreement, the Company agreed to provide compound material for certain of their product candidates and the right for the Recipient to use the material to perform research during the term of the Option Agreement. The Option Agreement included a non-refundable up-front payment of $250 thousand. The term of the Option Agreement commenced in August 2017 and could be extended upon additional payments of $250 thousand made by the Recipient, at its option, at the beginning of each of the next two quarterly periods. In October 2017, the Company received an additional $250 thousand payment to extend the term for an additional quarterly period. In December, 2017, the Recipient terminated the agreement. In the future, the Company will seek to generate additional revenue primarily from a combination of product sales and collaborations with strategic partners. During the year ended December 31, 2017, the Company recognized $500 thousand in revenue related to the Option Agreement. |
Preferred Stock
Preferred Stock | 12 Months Ended |
Dec. 31, 2017 | |
Preferred Stock | |
Preferred Stock | 11. Preferred Stock Convertible Preferred Stock In March, 2015, the Company issued 13,062,965 shares of Series B Preferred Stock at $0.9503 per share, and received net proceeds of $12.3 million. Prior to the IPO, the holders of the Company's convertible Preferred Stock had certain voting and dividend rights, as well as liquidation preferences and conversion privileges. All rights, preferences, and privileges associated with the convertible Preferred Stock were terminated at the time of the Company's IPO in conjunction with the conversion of all outstanding shares of convertible Preferred Stock into shares of Common Stock. Upon the closing of the Company's IPO on June 30, 2015, all outstanding shares of the Company's Preferred Stock were automatically converted into 9,029,549 shares of Common Stock. As of December 31, 2016, the Company had 5,000,000 shares of Preferred Stock authorized for issuance, $0.001 par value per share, with none issued or outstanding. Preferred Stock As of December 31, 2017, the Company had 5,000,000 shares of preferred stock authorized for issuance, $0.001 par value per share, with none issued or outstanding. Preferred stock may be issued from time to time in one or more series, each series to have such terms as stated or expressed in the resolutions providing for the issue of such series adopted by the board of directors of the Company. Preferred stock which may be redeemed, purchased or acquired by the Company may be reissued except as otherwise provided by law. |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2017 | |
Common Stock | |
Common Stock | 12. Common Stock As of December 31, 2017, the Company had 150,000,000 shares of Common Stock authorized for issuance, $0.001 par value per share, with 23,645,247 shares issued and outstanding. The voting, dividend and liquidation rights of holders of Common Stock are subject to and qualified by the rights, powers and preferences of the holders of any outstanding Preferred Stock. The Company's Common Stock has the following characteristics: Voting The holders of Common Stock are entitled to one vote for each share of Common Stock held at all meetings of stockholders and written actions in lieu of meetings. Dividends The holders of Common Stock are entitled to receive dividends, if and when declared by the board of directors. Cash dividends may not be declared or paid to holders of Common Stock until paid on each series of outstanding Preferred Stock in accordance with their respective terms. No dividends have been declared or paid from the Company's inception through December 31, 2017. Liquidation In the event of any voluntary or involuntary liquidation, dissolution or winding up of the Company, the holders of Common Stock are entitled to share ratably in the Company's assets available for distribution to stockholders, subject to any preferential or other rights of any then-outstanding Preferred Stock. Reserved for Future Issuance The Company has reserved for future issuance the following shares of Common Stock: As of December 31, 2017 2016 Warrants for the purchase of Common Stock Options to purchase Common Stock Employee Stock Purchase Plan ​ ​ ​ ​ ​ ​ ​ ​ Total ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Stock Incentive Plans
Stock Incentive Plans | 12 Months Ended |
Dec. 31, 2017 | |
Stock Incentive Plans | |
Stock Incentive Plans | 13. Stock Incentive Plans Prior to the IPO, the Company granted awards to eligible participants under its 2008 Equity Incentive Plan ("2008 Plan"). In May 2015, the Company's board of directors adopted and, in June 2015, the Company's stockholders approved the 2015 Stock Incentive Plan ("2015 Plan"), which became effective immediately prior to the effectiveness of the IPO. Subsequent to the IPO, option grants are awarded to eligible participants only under the 2015 Plan. The 2015 Plan provides for the grant of incentive stock options, non-statutory stock options, restricted stock awards, restricted stock units, stock appreciation rights and other stock-based awards. The Company's employees, officers, directors and consultants and advisors are eligible to receive awards under the 2015 Plan. The maximum number of shares of Common Stock that may be delivered in satisfaction of awards under the 2015 Plan is 1,068,287 shares, plus (1) 25,942 shares that were available for grant under the 2008 Plan immediately prior to the closing of the IPO, (2) the number of shares of Common Stock subject to outstanding awards under the 2008 Plan upon closing of the IPO that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right and (3) an annual increase, to be added the first day of each fiscal year, beginning with the fiscal year ending December 31, 2016 and continuing until, and including, the fiscal year ending December 31, 2025, equal to the lowest of 1,297,334 shares of Common Stock, 4% of the number of shares of Common Stock outstanding on the first day of the fiscal year and an amount determined by the Company's board of directors. The January 1, 2018 and 2017 increases to the 2015 Plan added 945,810 and 752,702 authorized shares, respectively. As of December 31, 2017, the Company had reserved 832,092 shares of Common Stock under the 2008 Plan, of which none remained available for future issuance. As of December 31, 2017, the Company had reserved 2,837,699 shares of Common Stock under the 2015 Plan, of which 824,165 shares remained available for future issuance. Under the 2015 Plan, stock options may not be granted with exercise prices at less than fair value on the date of the grant. Terms of stock option agreements, including vesting requirements, are determined by the Company's board of directors, subject to the provisions of the applicable stock incentive plan. Options granted by the Company generally vest ratably over four years, with a one-year cliff, and options are exercisable from the date of grant for a period of ten years. For options granted through December 31, 2017, the exercise price or purchase price, as applicable, equaled the estimated fair value of the Common Stock as determined by the Company's board of directors on the date of grant. A summary of the Company's stock option activity and related information for employees and nonemployees follows: Shares Weighted- Weighted Aggregate Outstanding at December 31, 2016 $ $ Granted $ Exercised ) $ Cancelled or forfeited ) $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Outstanding at December 31, 2017 $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Vested and Exercisable at December 31, 2017 $ $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ The total intrinsic value of options exercised for the years ended December 31, 2017, 2016 and 2015 was $40 thousand, $0.5 million and $0.3 million, respectively. The total adjusted fair value of employee options vested for the years ended December 31, 2017, 2016 and 2015 was $2.2 million, $2.3 million and $1.4 million, respectively. The weighted-average grant date fair value of options granted to employees and non-employees for the years ended December 31, 2017, 2016, and 2015 was $0.85, $2.89 and $7.68, respectively. At December 31, 2017, the total unrecognized compensation expense related to unvested stock option awards was $3.2 million. The Company expects to recognize that cost over a weighted-average period of approximately 2.0 years. Stock-Based Compensation Expense The fair value of stock options granted to employees and non-employees was estimated using the Black-Scholes option-pricing model based on the following assumptions: Year Ended December 31, 2017 2016 2015 Weighted-average expected volatility 76.1 - 81.9 % 72.0 - 73.9 % 73.6 - 86.8 % Expected term (in years) 6.25 - 9.77 6.17 - 10.00 Risk-free interest rate 2.00 - 2.38 % 1.24 - 2.00 % 0.92 - 2.45 % Expected dividend yield % % % Volatility Due to the lack of company-specific historical and implied volatility data of its Common Stock, the Company does not have relevant historical data to support its expected volatility. As such, the Company has used a weighted average of expected volatility based on the volatilities of a representative group of publicly traded biopharmaceutical companies. For purposes of identifying representative companies, the Company considered characteristics such as number of product candidates in early stages of product development, area of therapeutic focus, and length of trading history. The expected volatility was determined using a weighted average of the historical volatilities of the representative group of companies for a period equal to the expected term of the option grant. The Company intends to continue to consistently apply this process using the same representative companies until a sufficient amount of historical information regarding the volatility of the Company's own share price becomes available or until circumstances change, such that the identified entities are no longer representative companies. In the latter case, more suitable, similar entities whose share prices are publicly available would be utilized in the calculation. Expected Term The Company uses the "simplified method" to estimate the expected term of stock option grants. Under this approach, the weighted-average expected life is presumed to be the average of the contractual term (ten years) and the vesting term (generally four years) of the Company's stock options, taking into consideration multiple vesting tranches. The Company utilizes this method due to lack of historical exercise data and the plain-vanilla nature of the Company's share-based awards. Risk-Free Rate The risk-free rate was based on the yield curve of U.S. Treasury securities with periods commensurate with the expected term of the options being valued. Employee Stock Purchase Plan In June 2015, the Company's board of directors adopted and the Company's stockholders approved the 2015 Employee Stock Purchase Plan (the "2015 ESPP") which became effective upon closing of the IPO. The 2015 ESPP initially authorized the issuance of up to a total of 182,352 shares of Common Stock to participating eligible employees. The number of authorized shares increases each January 1, commencing on January 1, 2016 and ending on December 31, 2026, by an amount equal to the lesser of one percent of the Company's outstanding shares as of the first day of the applicable year, 364,705 shares and any lower amount determined by the Company's board of directors. The January 1, 2018 and 2017 increases to the 2015 ESPP added 236,452 and 188,175 authorized shares, respectively. As of December 31, 2017, there had been no shares issued under the 2015 ESPP. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2017 | |
Income Taxes | |
Income Taxes | 14. Income Taxes For the years ended December 31, 2017, 2016 and 2015, the Company did not record a provision for federal or state income taxes as it has incurred cumulative net operating losses since inception. On December 22, 2017, the Tax Cuts and Jobs Act (the "Act") was enacted in the United States. The Act reduces the U.S. federal corporate tax rate from a graduated rate of 35% to a flat rate of 21%, requires companies to pay a one-time transition tax on earnings of certain foreign subsidiaries that were previously tax deferred and creates new taxes on certain foreign sourced earnings. At December 31, 2017, the Company has not completed its accounting for the tax effects of enactment of the Act, including the effects on its existing deferred tax balances and the one-time transition tax. For the year ended December 31, 2017, the Company recognized no transition tax as it has no foreign subsidiaries. As a result of the Act, the Company remeasured certain deferred tax assets and liabilities based on the rates at which they are anticipated to reverse in the future, which is generally 21%. This resulted in a decrease to the Company's tax effected deferred tax assets of $19.8 million. As the deferred tax asset is offset in full by a valuation allowance, this enacted legislation had no impact on the Company's financial position or results of operations. Any items reported are done so using provisional amounts until the initial accounting required by the Act is complete. Additional time and resources are needed to ensure the correct implementation of the Act. A reconciliation of the U.S. statutory income tax rate to the Company's effective tax rate is as follows for the years ended December 31, 2017, 2016 and 2015: Year Ended December 31, 2017 2016 2015 Federal income tax (benefit) at statutory rate % % % Permanent differences ) ) ) Federal research and development credits and adjustments State income tax, net of federal benefit Tax reform deferred rate change ) — — Other ) ) Change in valuation allowance ) ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Effective income tax rate — % — % — % ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ The Company's deferred tax assets consisted of the following (in thousands): Year Ended 2017 2016 Deferred tax assets Net operating loss carryforwards $ $ Tax credit carryforwards Capitalized research and development Capitalized legal expenses Other differences ​ ​ ​ ​ ​ ​ ​ ​ Total deferred tax assets Valuation allowance ) ) ​ ​ ​ ​ ​ ​ ​ ​ Net deferred tax assets $ — $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ The Company recorded a decrease to the valuation allowance of $8.4 million during the year ended December 31, 2017 primarily due to the federal rate reduction from 34% to 21% as a result of the Act. The Company recorded an increase to the valuation allowance of $14.7 million and $13.6 million during the years ended December 31, 2016 and 2015, respectively, due primarily to an increase in the net operating loss carryforwards and tax credits. In assessing the realizability of deferred tax assets, the Company considers whether it is more likely than not that some portion or all of the deferred tax assets will not be realized. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income during the periods in which the temporary differences representing net future deductible amounts become deductible. Due to the Company's history of losses and expectation of future losses, the deferred tax assets were fully offset by a valuation allowance at December 31, 2017 and 2016. As of December 31, 2017, the Company had approximately $152.1 million of federal and $150.4 million of state net operating loss carryforwards to offset future taxable income, if any. Such net operating loss carryforwards expire at varying times through the year 2037, if not utilized. The Company had approximately $4.5 million of federal and $1.8 million of state tax credit carryforwards available to reduce future tax liabilities as of December 31, 2017, which will expire at varying times through the year 2037. The Internal Revenue Code of 1986, as amended (the "Code"), provides for a limitation of the annual use of net operating losses and other tax attributes (such as research and development tax credit carryforwards) following certain ownership changes (as defined by the Code) that could limit the Company's ability to utilize these carryforwards. At this time, the Company has not completed a study to assess whether an ownership change under Section 382 of the Code has occurred, or whether there have been multiple ownership changes since the Company's formation, due to the costs and complexities associated with such a study. The Company may have experienced various ownership changes, as defined by the Code, as a result of past financing transactions. Accordingly, the Company's ability to utilize the aforementioned carryforwards may be limited. Additionally, U.S. tax laws limit the time during which these carryforwards may be applied against future taxes. Therefore, the Company may not be able to take full advantage of these carryforwards for federal or state income tax purposes. The Company's tax returns for the years ended December 31, 2009 through December 31, 2017 are still subject to examination by major tax jurisdictions. |
Defined Contribution Benefit Pl
Defined Contribution Benefit Plan | 12 Months Ended |
Dec. 31, 2017 | |
Defined Contribution Benefit Plan | |
Defined Contribution Benefit Plan | 15. Defined Contribution Benefit Plan The Company sponsors a 401(k) retirement plan, in which substantially all of its full-time employees are eligible to participate. Participants may contribute a percentage of their annual compensation to this plan, subject to statutory limitations. The Company did not provide any contributions to this plan during the years ended December 31, 2017, 2016 or 2015. |
Subsequent Events
Subsequent Events | 12 Months Ended |
Dec. 31, 2017 | |
Subsequent Events | |
Subsequent Events | 16. Subsequent Events The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the financial statements to provide additional evidence for certain estimates and to identify matters that require additional disclosure. Subsequent events have been evaluated as required. Sales Agreement Subsequent to December 31, 2017, the Company sold an aggregate of 5,390,255 shares of common stock pursuant to the Second ATM Program, at an average price of $1.59 per share, for gross proceeds of $8.6 million, resulting in net proceeds of $8.3 million after deducting sales commissions and offering expenses. The Second ATM Program was fully utilized as of the date of issuance of these financial statements. |
Quarterly Financial Information
Quarterly Financial Information (Unaudited) | 12 Months Ended |
Dec. 31, 2017 | |
Quarterly Financial Information (unaudited) | |
Quarterly Financial Information (unaudited) | 17. Quarterly Financial Information (unaudited, in thousands, except share and per share data) Three Months Ended March 31, June 30, September 30, December 31, Revenue $ — $ — $ $ Operating expenses Net loss ) ) ) ) Net loss per share: Basic and Diluted $ ) $ ) $ ) $ ) Weighted-average common shares outstanding used in net loss per share: Basic and Diluted Three Months Ended March 31, June 30, September 30, December 31, Operating expenses $ $ $ $ Net loss ) ) ) ) Net loss per share: Basic and Diluted $ ) $ ) $ ) $ ) Weighted-average common shares outstanding used in net loss per share: Basic and Diluted |
Summary of Significant Accoun26
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2017 | |
Summary of Significant Accounting Policies | |
Basis of Presentation and Principles of Consolidation | Basis of Presentation and Principles of Consolidation The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiary, Catabasis Securities Corporation. All intercompany balances and transactions have been eliminated in consolidation. These consolidated financial statements have been prepared in accordance with United States generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented. |
Use of Estimates | Use of Estimates The preparation of the Company's consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. Actual results could differ from such estimates. The Company utilizes certain estimates to record expenses relating to research and development contracts. These contract estimates, which are primarily related to the length each contract and the amount of service provided as of each measurement date, are determined by the Company based on input from internal project management, as well as from third-party service providers. |
Off-Balance Sheet Risk and Concentrations of Credit Risk | Off-Balance Sheet Risk and Concentrations of Credit Risk The Company has no off-balance sheet risk, such as foreign exchange contracts, option contracts or other foreign hedging arrangements. Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and restricted cash. The primary objectives for the Company's investment portfolio are the preservation of capital and the maintenance of liquidity. The Company's investment policy includes guidelines on the quality of the institutions and financial instruments and defines allowable investments that the Company believes minimizes the exposure to concentration of credit risk. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers highly liquid investments with a maturity of three months or less when purchased to be cash equivalents. Cash equivalents, which consist primarily of money market funds backed by U.S. government securities, are stated at fair value. Cash and cash equivalents consist of the following (in thousands): December 31, 2017 2016 Cash $ $ Money market fund ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Available-for-Sale Investments | Available-for-Sale Investments The Company classifies all short-term investments with a remaining maturity of greater than three months at the time of purchase as available-for-sale. Available-for-sale securities are recorded at fair value, with the unrealized gains and losses reported in other comprehensive loss. The amortized cost of debt securities is adjusted for the amortization of premiums and accretion of discounts to maturity. Such amortization is included in interest and investment income. Realized gains and losses, interest, dividends and declines in value judged to be other-than-temporary are included in interest and investment income. The cost of securities sold is based on the specific identification method for purposes of recording realized gains and losses. To determine whether an other-than-temporary impairment exists, the Company considers whether it has the ability and intent to hold the investment until a market price recovery, and whether evidence indicating the recoverability of the cost of the investment outweighs evidence to the contrary. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments The fair value hierarchy prioritizes the inputs to valuation techniques used to measure fair value into three broad levels as follows: Level 1 inputs are quoted prices (unadjusted) in active markets for identical assets or liabilities; Level 2 inputs are inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly; and Level 3 inputs are unobservable inputs that reflect the Company's own assumptions about the assumptions market participants would use in pricing the asset or liability. Financial assets and liabilities are classified in their entirety based on the lowest level of input that is significant to the fair value measurement. The carrying amounts reflected in the balance sheets for cash equivalents, restricted cash, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values at December 31, 2017 and 2016, due to their short-term nature. There have been no changes to the valuation methods during the years ended December 31, 2017 and 2016. The Company evaluates transfers between levels at the end of each reporting period. There were no transfers of assets or liabilities between levels during the year ended December 31, 2017 and 2016. At December 31, 2017, the carrying value of the Company's debt approximated fair value, which was determined using Level 3 inputs, including a quoted interest rate. The Company's investment portfolio may include fixed income securities that do not always trade on a daily basis. As a result, the pricing services used by the Company apply other available information as applicable through processes such as benchmark yields, benchmarking of like securities, sector groupings and matrix pricing to prepare valuations. The Company validates the prices provided by its third party pricing services by obtaining market values from other pricing sources and analyzing pricing data in certain instances. The Company determines the fair value of available-for-sale securities (Note 4) using Level 2 inputs. |
Deferred Financing Costs | Deferred Financing Costs Deferred financing costs include costs directly attributable to the Company's offerings of its equity securities and its debt financings. Costs attributable to equity offerings are charged against the proceeds of the offering once the offering is completed. Costs attributable to debt financings are deferred and amortized over the term of the debt using the effective interest rate method. |
Property and Equipment | Property and Equipment Property and equipment consist of laboratory equipment, computer equipment, leasehold improvements and furniture and fixtures. Property and equipment is stated at cost and depreciated using the straight-line method over the estimated useful lives of the respective assets. Costs of major additions and betterments are capitalized; maintenance and repairs, which do not improve or extend the life of the respective assets, are charged to expense as incurred. Upon retirement or sale, the cost of the disposed asset and the related accumulated depreciation are removed from the accounts and the resulting gain or loss is recognized. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets The Company continually evaluates whether events or circumstances have occurred that indicate that the estimated remaining useful life of its long-lived assets may warrant revision or that the carrying value of these assets may be impaired. The Company has not recognized any significant impairment charges from inception through December 31, 2017. |
Research and Development Expenses | Research and Development Expenses Research and development costs are expensed as incurred. Research and development costs include salaries and personnel-related costs, stock-based compensation, consulting fees, fees paid for contract research services, the costs of laboratory equipment and facilities and other external costs. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred. The deferred amounts are expensed as the related goods are delivered or the services are performed. |
Revenue Recognition | Revenue Recognition Effective January 1, 2017, the Company adopted Accounting Standards Codification ("ASC"), Revenue from Contracts with Customers, ("ASC 606"). This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, collaboration arrangements and financial instruments. Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. In determining the appropriate amount of revenue to be recognized as the Company fulfills its obligations under the agreement, the Company performs the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The Company only applies the five-step model to contracts when it is probable that it will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations, and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. |
Stock-Based Compensation | Stock-Based Compensation The Company accounts for its stock-based compensation awards in accordance with ASC Topic 718, Compensation—Stock Compensation ( "ASC 718"). ASC 718 requires all share-based payments to employees, including grants of employee stock options, to be recognized in the statements of operations based on their grant date fair values. For stock options granted to employees and to members of the board of directors for their services on the board of directors, the Company estimates the grant date fair value of each option award using the Black-Scholes option-pricing model. The use of the Black-Scholes option-pricing model requires management to make assumptions with respect to the expected term of the option, the expected volatility of the Common Stock consistent with the expected term of the option, risk-free interest rates and expected dividend yields of the Common Stock. For awards subject to service-based vesting conditions, the Company recognizes stock-based compensation expense equal to the grant date fair value of stock options on a straight-line basis over the requisite service period. The Company expenses restricted stock awards based on the fair value of the award on a straight-line basis over the associated service period of the award. Share-based payments issued to non-employees are recorded at their fair values, and are periodically revalued as the equity instruments vest and are recognized as expense over the related service period in accordance with the provisions of ASC Topic 505, Equity . For equity instruments granted to non-employees, the Company recognizes stock-based compensation expense on a straight-line basis. During the years ended December 31, 2017, 2016 and 2015, the Company recorded stock-based compensation expense for employee and non-employee stock options and restricted stock, which was allocated as follows in the statements of operations (in thousands): Year Ended December 31, 2017 2016 2015 Research and development $ $ $ General and administrative ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ No related tax benefits were recognized for the years ended December 31, 2017, 2016 and 2015. |
Grant Awards | Grant Awards In the years ended December 31, 2017, 2016 and 2015, the Company received $124,000, $376,000, $100,000, respectively, in grants from the Muscular Dystrophy Association, Friedreich's Ataxia Research Alliance and Parent Project for Muscular Dystrophy. In the years ended December 31, 2017, 2016, 2015 the Company utilized $205,000, $224,000 and $100,000 of the grants received to offset related expenses incurred in the Company's statements of operations. |
Net Loss Per Share | Net Loss Per Share Basic net loss per share is calculated by dividing net loss by the weighted average shares outstanding during the period, without consideration for Common Stock equivalents. Diluted net loss per share is calculated by adjusting weighted average shares outstanding for the dilutive effect of Common Stock equivalents outstanding for the period, determined using the treasury-stock method. For purposes of the dilutive net loss per share calculation, Preferred Stock, stock options, warrants to purchase Common Stock and warrants to purchase Preferred Stock are considered to be Common Stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented. The following Common Stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect: Year Ended December 31, 2017 2016 2015 Stock options Common stock warrants ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Income Taxes | Income Taxes The Company provides deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the Company's financial statement carrying amounts and the tax basis of assets and liabilities using enacted tax rates expected to be in effect in the years in which the differences are expected to reverse. A valuation allowance is provided to reduce the deferred tax assets to the amount that will more likely than not be realized. The Company accounts for uncertain tax positions in accordance with the provisions of ASC Topic 740 , Expenses—Income Taxes . When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. The Company did not have any significant uncertain tax positions for any periods presented. |
Segment Information | Segment Information Operating segments are identified as components of an enterprise for which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision making group, in making decisions on how to allocate resources and assess performance. The Company views its operations and manages its business in one operating segment. The Company operates in one geographic segment. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss is defined as the change in equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. For the years ended December 31, 2017 and 2016, amounts in accumulated other comprehensive loss were comprised of unrealized gains and losses on available-for-sale securities. For the year ended December 31, 2015, comprehensive loss was equal to net loss. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements—Adopted In May 2014, the Financial Accounting Standards Board ("FASB") issued ASU 2014-09, Revenue from Contracts with Customers , ("ASU 2014-09"). The standard replaced existing revenue recognition standards and significantly expanded the disclosure requirements for revenue arrangements. The Company adopted the standard on January 1, 2017 using the full retrospective method. The adoption of the new standard did not have an effect on previously issued consolidated financial statements as the Company had not recognized any revenue prior to 2017. In March 2016, the FASB issued ASU 2016-09, Improvements to Employee Share-Based Payment Accounting , ("ASU 2016-09"). This standard amended the existing guidance in an attempt to simplify several aspects of accounting for employee share-based payment transactions, including the accounting for income taxes, forfeitures, and statutory tax withholding requirements, as well as classification in the statement of cash flows. The Company adopted the standard on January 1, 2017. The amended guidance eliminates the requirement that excess tax benefits be realized as a reduction in current taxes payable before the associated tax benefit can be recognized in additional paid-in capital. This created approximately $0.2 million of deferred tax assets relating to federal and state net operating losses that were fully offset by a corresponding increase in the valuation allowance. As a result, there was no cumulative effect adjustment to accumulated deficit. As part of the adoption of ASU 2016-09, the Company elected to record forfeitures as they occur, which did not have a material impact on the consolidated financial statements upon adoption. In May 2017, the FASB issued ASU 2017-09, Scope of Modification Accounting , ("ASU 2017-09"). This standard provides clarity on whether the change in existing terms and conditions of a share-based award require application of the guidance in ASC Topic 718, Compensation—Stock Compensation. The Company early adopted the standard in the period ending June 30, 2017. As of December 31, 2017, there was no material impact on the Company's consolidated financial statements, and any future impact from adoption will be dependent on future transactions involving modifications of share-based awards. Recent Accounting Pronouncements—Not Yet Adopted From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its financial position or results of operations upon adoption. In February 2016, the FASB issued ASU 2016-02, Leases , ("ASU 2016-02"). This standard amends the existing guidance to require lessees to present most leases on their balance sheets but recognize corresponding expenses on their statements of operations. It is effective for annual reporting periods beginning after December 15, 2018, but early adoption is permitted. The Company is currently evaluating the impact that this standard will have on its consolidated financial statements. |
Summary of Significant Accoun27
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Summary of Significant Accounting Policies | |
Schedule of cash and cash equivalents | Cash and cash equivalents consist of the following (in thousands): December 31, 2017 2016 Cash $ $ Money market fund ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Schedule of stock-based compensation expense | During the years ended December 31, 2017, 2016 and 2015, the Company recorded stock-based compensation expense for employee and non-employee stock options and restricted stock, which was allocated as follows in the statements of operations (in thousands): Year Ended December 31, 2017 2016 2015 Research and development $ $ $ General and administrative ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Schedule of anti-dilutive common stock equivalents excluded from calculation of diluted net loss per share | Year Ended December 31, 2017 2016 2015 Stock options Common stock warrants ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Financial Instruments (Tables)
Financial Instruments (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Financial Instruments | |
Schedule of assets and liabilities measures at fair value on a recurring basis | Below is a summary of assets and liabilities measured at fair value on a recurring basis (in thousands): As of December 31, 2017 Quoted Prices Significant Significant Total Assets: Cash and cash equivalents: Money market funds $ $ — $ — $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total assets $ $ — $ — $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2016 Quoted Prices Significant Significant Total Assets: Cash and cash equivalents: Money market funds $ $ — $ — $ Available-for-sale securities: Corporate debt securities — — U.S. government-sponsored securities — — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total assets $ $ $ — $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Available-for-Sale Securities (
Available-for-Sale Securities (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Available-for-Sale Securities | |
Summary of available-for-sale securities | The following table summarizes the available-for-sale securities held at December 31, 2016 (in thousands): Amortized Gross Gross Fair Value December 31, 2016 Corporate debt securities $ $ — $ ) $ U.S. government-sponsored securities — — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ — $ ) $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Property and Equipment (Tables)
Property and Equipment (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Property and Equipment | |
Summary of property and equipment and related accumulated depreciation | Property and equipment and related accumulated depreciation were as follows (in thousands): December 31, Estimated Useful Life (Years) 2017 2016 Lab equipment 3 $ $ Computer equipment 3 Furniture and fixtures 5 Leasehold improvements Lesser of useful life or remaining lease term ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Less accumulated depreciation and amortization ) ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total property and equipment, net $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Accrued Expenses | |
Schedule of components of accrued expenses | Accrued expenses consisted of the following (in thousands): December 31, 2017 2016 Accrued compensation $ $ Accrued contracted research costs Accrued professional fees Accrued other ​ ​ ​ ​ ​ ​ ​ ​ Total $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Notes Payable (Tables)
Notes Payable (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Notes Payable | |
Schedule of estimated future principal payments | Estimated future principal payments at December 31, 2017 are as follows (in thousands): Year Ending December 31, Amount 2018 ​ ​ ​ ​ ​ Total $ Less: discount for warrants and costs paid to lenders ) Less: current portion ) ​ ​ ​ ​ ​ Note payable, net of current portion and discount $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Commitments (Tables)
Commitments (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Commitments | |
Schedule of future minimum payments under non-cancelable operating lease | Future minimum payments required under the non-cancelable operating lease as of December 31, 2017 are summarized as follows (in thousands): Period Ending December 31, Amount 2018 2019 2020 ​ ​ ​ ​ ​ Total minimum lease payments $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Common Stock (Tables)
Common Stock (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Common Stock | |
Schedule of Common Stock reserved for future issuance | As of December 31, 2017 2016 Warrants for the purchase of Common Stock Options to purchase Common Stock Employee Stock Purchase Plan ​ ​ ​ ​ ​ ​ ​ ​ Total ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Stock Incentive Plans (Tables)
Stock Incentive Plans (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Stock Incentive Plans | |
Summary of stock option activity | Shares Weighted- Weighted Aggregate Outstanding at December 31, 2016 $ $ Granted $ Exercised ) $ Cancelled or forfeited ) $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Outstanding at December 31, 2017 $ $ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Vested and Exercisable at December 31, 2017 $ $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Schedule of assumptions made based on the Black-Scholes option pricing model | Year Ended December 31, 2017 2016 2015 Weighted-average expected volatility 76.1 - 81.9 % 72.0 - 73.9 % 73.6 - 86.8 % Expected term (in years) 6.25 - 9.77 6.17 - 10.00 Risk-free interest rate 2.00 - 2.38 % 1.24 - 2.00 % 0.92 - 2.45 % Expected dividend yield % % % |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Income Taxes | |
Schedule of reconciliation of U.S. statutory income tax rate to effective tax rate | Year Ended December 31, 2017 2016 2015 Federal income tax (benefit) at statutory rate % % % Permanent differences ) ) ) Federal research and development credits and adjustments State income tax, net of federal benefit Tax reform deferred rate change ) — — Other ) ) Change in valuation allowance ) ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Effective income tax rate — % — % — % ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Schedule of components of deferred tax assets | The Company's deferred tax assets consisted of the following (in thousands): Year Ended 2017 2016 Deferred tax assets Net operating loss carryforwards $ $ Tax credit carryforwards Capitalized research and development Capitalized legal expenses Other differences ​ ​ ​ ​ ​ ​ ​ ​ Total deferred tax assets Valuation allowance ) ) ​ ​ ​ ​ ​ ​ ​ ​ Net deferred tax assets $ — $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ |
Quarterly Financial Informati37
Quarterly Financial Information (Unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Quarterly Financial Information (unaudited) | |
Summary of unaudited quarterly results of operations | Quarterly Financial Information (unaudited, in thousands, except share and per share data) Three Months Ended March 31, June 30, September 30, December 31, Revenue $ — $ — $ $ Operating expenses Net loss ) ) ) ) Net loss per share: Basic and Diluted $ ) $ ) $ ) $ ) Weighted-average common shares outstanding used in net loss per share: Basic and Diluted Three Months Ended March 31, June 30, September 30, December 31, Operating expenses $ $ $ $ Net loss ) ) ) ) Net loss per share: Basic and Diluted $ ) $ ) $ ) $ ) Weighted-average common shares outstanding used in net loss per share: Basic and Diluted |
Organization and Operations - L
Organization and Operations - Liquidity and Going Concern (Details) - USD ($) $ / shares in Units, $ in Thousands | Jan. 01, 2018 | Aug. 31, 2016 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | Oct. 31, 2017 | Mar. 31, 2017 | Dec. 31, 2014 |
Common Stock | ||||||||
Share price (in dollars per share) | $ 0.001 | |||||||
Additional common stock issued | $ 8,036 | $ 11,994 | $ 61,744 | |||||
Proceeds from registered direct offering, net of issuance costs | 10,603 | |||||||
Accumulated deficit | (171,434) | (144,070) | ||||||
Cash and cash equivalents | $ 16,369 | $ 23,596 | $ 62,780 | $ 14,668 | ||||
Common Stock | ||||||||
Common Stock | ||||||||
Number of shares issued | 4,804,796 | 3,243,015 | 5,750,000 | |||||
Additional common stock issued | $ 5 | $ 3 | $ 5 | |||||
Common Stock | Cowen | ||||||||
Common Stock | ||||||||
Percentage of commission payable to underwriter based on proceeds from common stock | 3.00% | |||||||
Common Stock | First ATM Program | ||||||||
Common Stock | ||||||||
Number of shares issued | 4,804,796 | |||||||
Share price (in dollars per share) | $ 1.79 | |||||||
Additional common stock issued | $ 8,600 | |||||||
Net proceeds | 8,000 | |||||||
Common Stock | First ATM Program | Cowen | ||||||||
Common Stock | ||||||||
Aggregate maximum offering amount | $ 10,000 | $ 8,800 | ||||||
Common Stock | Second ATM Program | ||||||||
Common Stock | ||||||||
Remaining common stock available for sale under offering program | $ 8,600 | |||||||
Common Stock | Second ATM Program | Cowen | ||||||||
Common Stock | ||||||||
Aggregate maximum offering amount | $ 10,000 | |||||||
Common Stock | Second ATM Program | Subsequent Events | ||||||||
Common Stock | ||||||||
Number of shares issued | 5,390,255 | |||||||
Share price (in dollars per share) | $ 1.59 | |||||||
Additional common stock issued | $ 8,600 | |||||||
Net proceeds | $ 8,300 | |||||||
Common Stock | Second ATM Program | Subsequent Events | Cowen | ||||||||
Common Stock | ||||||||
Number of shares issued | 5,390,255 | |||||||
Share price (in dollars per share) | $ 1.59 | |||||||
Additional common stock issued | $ 8,600 | |||||||
Net proceeds | $ 8,300 | |||||||
Common Stock | Registered Direct Offering | ||||||||
Common Stock | ||||||||
Number of shares issued | 3,243,015 | |||||||
Share price (in dollars per share) | $ 4.04 | |||||||
Additional common stock issued | $ 13,100 | |||||||
Proceeds from registered direct offering, net of issuance costs | $ 12,000 |
Summary of Significant Accoun39
Summary of Significant Accounting Polices - Cash and Cash Equivalents and Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 |
Cash and Cash Equivalents | ||||
Cash | $ 2,781 | $ 1,173 | ||
Money market fund | 13,588 | 22,423 | ||
Total | 16,369 | 23,596 | $ 62,780 | $ 14,668 |
Fair Value of Financial Instruments | ||||
Transfers of assets or liabilities between levels | $ 0 | $ 0 |
Summary of Significant Accoun40
Summary of Significant Accounting Policies - Stock-Based Compensation (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Stock-Based Compensation | |||
Stock-based compensation expense | $ 2,007 | $ 2,160 | $ 1,658 |
Tax benefits recognized from stock-based compensation expense | 0 | 0 | 0 |
Research and development | |||
Stock-Based Compensation | |||
Stock-based compensation expense | 784 | 723 | 681 |
General and administrative | |||
Stock-Based Compensation | |||
Stock-based compensation expense | $ 1,223 | $ 1,437 | $ 977 |
Summary of Significant Accoun41
Summary of Significant Accounting Polices - Grant Awards (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Summary of Significant Accounting Policies | |||
Grants from Muscular Dystrophy Association | $ 124,000 | $ 376,000 | $ 100,000 |
Grants utilized to offset related expenses | $ 205,000 | $ 224,000 | $ 100,000 |
Summary of Significant Accoun42
Summary of Significant Accounting Policies - Net Loss Per Share and Segment Information (Details) | 12 Months Ended | ||
Dec. 31, 2017segmentshares | Dec. 31, 2016shares | Dec. 31, 2015shares | |
Antidilutive common stock equivalents excluded from computation of diluted net loss per share | |||
Anti-dilutive securities excluded from the calculation of diluted net loss per share (in shares) | 2,870,192 | 2,294,735 | 1,782,959 |
Segment Information | |||
Number of operating segments | segment | 1 | ||
Stock options | |||
Antidilutive common stock equivalents excluded from computation of diluted net loss per share | |||
Anti-dilutive securities excluded from the calculation of diluted net loss per share (in shares) | 2,845,626 | 2,270,169 | 1,723,554 |
Common stock warrants | |||
Antidilutive common stock equivalents excluded from computation of diluted net loss per share | |||
Anti-dilutive securities excluded from the calculation of diluted net loss per share (in shares) | 24,566 | 24,566 | 59,405 |
Summary of Significant Accoun43
Summary of Significant Accounting Policies - Recent Accounting Pronouncements (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
New Accounting Pronouncements | |||
Increase in valuation allowance | $ (8.4) | $ 14.7 | $ 13.6 |
Accounting Standards Update 2016-09 | |||
New Accounting Pronouncements | |||
Deferred tax assets related to federal and state net operating losses | 0.2 | ||
Increase in valuation allowance | $ 0.2 |
Financial Instruments (Details)
Financial Instruments (Details) - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 |
Cash and cash equivalents: | ||||
Cash and cash equivalents | $ 16,369 | $ 23,596 | $ 62,780 | $ 14,668 |
Available-for-sale securities: | ||||
Available-for-sale securities | 0 | 14,931 | ||
Corporate debt securities | ||||
Available-for-sale securities: | ||||
Available-for-sale securities | 13,930 | |||
U.S. government-sponsored securities | ||||
Available-for-sale securities: | ||||
Available-for-sale securities | 1,001 | |||
Recurring | ||||
Assets: | ||||
Total assets | 13,588 | 37,354 | ||
Recurring | Money market funds | ||||
Cash and cash equivalents: | ||||
Cash and cash equivalents | 13,588 | 22,423 | ||
Recurring | Corporate debt securities | ||||
Available-for-sale securities: | ||||
Available-for-sale securities | 13,930 | |||
Recurring | U.S. government-sponsored securities | ||||
Available-for-sale securities: | ||||
Available-for-sale securities | 1,001 | |||
Recurring | Level 1 | ||||
Assets: | ||||
Total assets | 13,588 | 22,423 | ||
Recurring | Level 1 | Money market funds | ||||
Cash and cash equivalents: | ||||
Cash and cash equivalents | $ 13,588 | 22,423 | ||
Recurring | Level 2 | ||||
Assets: | ||||
Total assets | 14,931 | |||
Recurring | Level 2 | Corporate debt securities | ||||
Available-for-sale securities: | ||||
Available-for-sale securities | 13,930 | |||
Recurring | Level 2 | U.S. government-sponsored securities | ||||
Available-for-sale securities: | ||||
Available-for-sale securities | $ 1,001 |
Available-for-Sale Securities45
Available-for-Sale Securities (Details) $ in Thousands | 12 Months Ended | |
Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($)item | |
Available-for-Sale Securities | ||
Amortized Cost | $ 14,935 | |
Gross Unrealized Losses | (4) | |
Fair Value | $ 0 | $ 14,931 |
Number of available-for-sale securities in an unrealized loss position | item | 15 | |
Number of available-for-sale securities in an unrealized loss position more than 12 months | item | 0 | |
Unrealized loss position, available-for-sale securities | $ 13,000 | |
Proceeds from sales of available-for-sale securities | $ 0 | 7,800 |
Corporate debt securities | ||
Available-for-Sale Securities | ||
Amortized Cost | 13,934 | |
Gross Unrealized Losses | (4) | |
Fair Value | 13,930 | |
U.S. government-sponsored securities | ||
Available-for-Sale Securities | ||
Amortized Cost | 1,001 | |
Fair Value | $ 1,001 |
Restricted Cash (Details)
Restricted Cash (Details) - USD ($) $ in Millions | Dec. 31, 2017 | Dec. 31, 2016 |
Restricted Cash | ||
Security deposit held for operating lease agreement | $ 0.1 | $ 0.1 |
Property and Equipment (Details
Property and Equipment (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Property and Equipment | |||
Property and equipment, gross | $ 1,976 | $ 2,067 | |
Less accumulated depreciation and amortization | (1,655) | (1,499) | |
Total property and equipment, net | 321 | 568 | |
Depreciation and amortization expense | 304 | 395 | $ 202 |
Lab equipment | |||
Property and Equipment | |||
Property and equipment, gross | $ 1,470 | $ 1,565 | |
Estimated Useful Life | 3 years | 3 years | |
Computer equipment | |||
Property and Equipment | |||
Property and equipment, gross | $ 165 | $ 166 | |
Estimated Useful Life | 3 years | 3 years | |
Furniture and fixtures | |||
Property and Equipment | |||
Property and equipment, gross | $ 77 | $ 77 | |
Estimated Useful Life | 5 years | 5 years | |
Leasehold improvements | |||
Property and Equipment | |||
Property and equipment, gross | $ 264 | $ 259 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Accrued Expenses | ||
Accrued compensation | $ 632 | $ 1,252 |
Accrued contracted research costs | 1,357 | 1,850 |
Accrued professional fees | 298 | 215 |
Accrued other | 145 | 360 |
Total | $ 2,432 | $ 3,677 |
Notes Payable - (Details)
Notes Payable - (Details) - USD ($) | Mar. 31, 2015 | Aug. 27, 2014 | Jun. 30, 2015 | Dec. 31, 2017 | Dec. 31, 2015 |
Line of Credit Facility | |||||
Proceeds from issuance of promissory notes | $ 5,000,000 | ||||
Term Loan A | |||||
Line of Credit Facility | |||||
Initial borrowings | $ 5,000,000 | ||||
Proceeds from issuance of promissory notes | 5,000,000 | ||||
Term Loan B | |||||
Line of Credit Facility | |||||
Proceeds from issuance of promissory notes | $ 5,000,000 | ||||
Other Term Loan | |||||
Line of Credit Facility | |||||
Maximum borrowing capacity | 20,000,000 | ||||
Credit Facility | |||||
Line of Credit Facility | |||||
Initial borrowings | $ 10,000,000 | ||||
Maximum borrowing capacity | $ 25,000,000 | ||||
Number of monthly payments (in number of payments) | 36 | ||||
Interest rate (as a percent) | 7.49% | ||||
Percentage of amount outstanding due as a payment (as a percent) | 3.48% | ||||
Effective interest rate (as a percent) | 11.20% | ||||
Debt Instruments | |||||
Financial covenants | 0 | ||||
Final judgment amount that would trigger lender rights for remedy | $ 250,000 | ||||
Basis spread | 5.00% | ||||
Interest rate increase in the event of a default (as a percent) | 5.00% | ||||
Minimum | Credit Facility | |||||
Line of Credit Facility | |||||
Percentage of amount outstanding due as a payment (as a percent) | 1.00% | ||||
Maximum | Credit Facility | |||||
Line of Credit Facility | |||||
Percentage of amount outstanding due as a payment (as a percent) | 3.00% | ||||
Warrant Liability | |||||
Debt Instruments | |||||
Exercise period | 7 years | ||||
2014 and 2015 Warrants | |||||
Debt Instruments | |||||
Value of warrants | $ 200,000 | ||||
Debt discounts | $ 200,000 | ||||
2014 Warrants | Term Loan A | |||||
Debt Instruments | |||||
Number of shares after conversion of issued warrants (in shares) | 157,844 | ||||
2015 Warrants | Term Loan B | |||||
Debt Instruments | |||||
Number of shares after conversion of issued warrants (in shares) | 157,844 | ||||
Common stock warrants | Initial public offering | |||||
Debt Instruments | |||||
Number of shares after conversion of issued warrants (in shares) | 24,566 | ||||
Exercise price of warrants (in dollars per share) | $ 12.2100 |
Notes Payable - Future Principa
Notes Payable - Future Principal Payments (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Estimated future principal payments | |||
2,018 | $ 2,500 | ||
Total | 2,500 | ||
Less: discount for warrants and costs paid to lenders | (21) | ||
Less: current portion | (2,479) | ||
Notes payable, net of current portion and discount | $ 2,479 | ||
Credit Facility | |||
Estimated future principal payments | |||
Interest expense | $ 500 | $ 800 | $ 1,000 |
Commitments (Details)
Commitments (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Operating leases | |||
Rent expense | $ 1,300 | $ 900 | $ 800 |
Future minimum payments required under the non-cancelable operating lease | |||
2,018 | 1,379 | ||
2,019 | 1,421 | ||
2,020 | 721 | ||
Total minimum lease payments | $ 3,521 |
Revenue Recognition (Details)
Revenue Recognition (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||
Oct. 31, 2017 | Aug. 31, 2017 | Dec. 31, 2017 | Sep. 30, 2017 | Dec. 31, 2017 | |
Revenue recognition | |||||
Revenue recognized related to a Option agreement | $ 250 | $ 250 | $ 500 | ||
Option agreement | |||||
Revenue recognition | |||||
Non-refundable up-front payment | $ 250 | ||||
Quarterly payments | $ 250 | ||||
Quarterly payment received | $ 250 | ||||
Revenue recognized related to a Option agreement | $ 500 |
Preferred Stock (Details)
Preferred Stock (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | |||
Jun. 30, 2015shares | Mar. 31, 2015USD ($)$ / sharesshares | Dec. 31, 2017series$ / sharesshares | Dec. 31, 2015USD ($) | Dec. 31, 2016$ / sharesshares | |
Preferred Stock | |||||
Preferred stock, authorized (in Shares) | 5,000,000 | 5,000,000 | |||
Preferred stock, par value (in dollars per share) | $ / shares | $ 0.001 | $ 0.001 | |||
Preferred Stock, shares issued (in shares) | 0 | 0 | |||
Share price (in dollars per share) | $ / shares | $ 0.001 | ||||
Preferred stock, outstanding (in shares) | 0 | 0 | |||
Minimum number of series used to issue preferred stock | series | 1 | ||||
Net proceeds from issuance of preferred stock | $ | $ 12,331 | ||||
Initial public offering | |||||
Preferred Stock | |||||
Conversion of convertible preferred stock into common stock (in shares) | 9,029,549 | ||||
Series B Convertible Preferred Stock | |||||
Preferred Stock | |||||
Preferred Stock, shares issued (in shares) | 13,062,965 | ||||
Share price (in dollars per share) | $ / shares | $ 0.9503 | ||||
Net proceeds from issuance of preferred stock | $ | $ 12,300 |
Common Stock (Details)
Common Stock (Details) | 12 Months Ended | |
Dec. 31, 2017Vote$ / sharesshares | Dec. 31, 2016$ / sharesshares | |
Common Stock | ||
Common stock, authorized (in shares) | 150,000,000 | 150,000,000 |
Par value of common stock (in dollars per share) | $ / shares | $ 0.001 | $ 0.001 |
Common stock, issued (in shares) | 23,645,247 | 18,817,572 |
Common Stock, outstanding (in shares) | 23,645,247 | 18,817,572 |
Number of votes entitled to each common stock share | Vote | 1 | |
Common stock dividends, declared or paid (in dollars per share) | $ / shares | $ 0 | |
Shares reserved for future issuance (in shares) | 4,218,016 | 3,300,018 |
Common stock warrants | ||
Common Stock | ||
Shares reserved for future issuance (in shares) | 24,566 | 24,566 |
Stock options | ||
Common Stock | ||
Shares reserved for future issuance (in shares) | 3,669,791 | 2,939,968 |
Employee Stock Purchase Plan | ||
Common Stock | ||
Shares reserved for future issuance (in shares) | 523,659 | 335,484 |
Stock Incentive Plans - Plan In
Stock Incentive Plans - Plan Information and Valuation Assumptions (Details) - shares | Jan. 01, 2018 | Jan. 01, 2017 | Dec. 31, 2017 | Dec. 31, 2016 |
Stock-Based Compensation | ||||
Shares reserved for future issuance (in shares) | 4,218,016 | 3,300,018 | ||
2015 Plan | ||||
Stock-Based Compensation | ||||
Shares available for grant (in shares) | 1,068,287 | |||
Shares reserved for future issuance (in shares) | 2,837,699 | |||
Shares available for future issuance (in shares) | 824,165 | |||
2015 Plan | Annual minimum increase | ||||
Stock-Based Compensation | ||||
Shares available for grant (in shares) | 1,297,334 | |||
Percentage of outstanding Common Stock available for issuance (as a percent) | 4.00% | |||
Potential increase in shares available for issuance as determined by the Board of Directors (in shares) | 945,810 | 752,702 | ||
2008 Plan | ||||
Stock-Based Compensation | ||||
Shares available for grant (in shares) | 25,942 | |||
Shares reserved for future issuance (in shares) | 832,092 | |||
Shares available for future issuance (in shares) | 0 | |||
Stock options | ||||
Stock-Based Compensation | ||||
Vesting period | 4 years | |||
Cliff period for vesting | 1 year | |||
Exercisable period | 10 years |
Stock Incentive Plans - Stock O
Stock Incentive Plans - Stock Option Plan Activity (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Stock options | |||
Outstanding at beginning of year (in shares) | 2,270,169 | ||
Granted (in shares) | 890,740 | ||
Exercised (in shares) | (22,879) | ||
Cancelled or forfeited (in shares) | (292,404) | ||
Outstanding at end of year (in shares) | 2,845,626 | 2,270,169 | |
Vested and Exercisable to vest at end of year (in shares) | 1,265,411 | ||
Weighted-Average Exercise Price | |||
Outstanding at beginning of year (in dollars per share) | $ 6.11 | ||
Granted (in dollars per share) | 1.26 | ||
Exercised (in dollars per share) | 1.01 | ||
Cancelled or forfeited (in dollars per share) | 7.13 | ||
Outstanding at end of year (in dollars per share) | 4.53 | $ 6.11 | |
Vested and Exercisable at end of year (in dollars per share) | $ 5.91 | ||
Weighted Average Remaining Contractual Term (years) | |||
Outstanding at end of year | 7 years 9 months | 8 years 1 month 21 days | |
Vested and Exercisable at end of year | 6 years 6 months 11 days | ||
Aggregate Intrinsic Value | |||
Outstanding at the beginning of the year | $ 613 | ||
Outstanding at the end of the year | $ 216 | $ 613 | |
Weighted average of the assumptions used to compute employee stock based compensation | |||
Expected term (in years) | 6 years 3 months | ||
Expected dividend yield (as a percent) | 0.00% | 0.00% | 0.00% |
Stock options | |||
Unrecognized stock-based compensation expense | |||
Intrinsic value of options exercised | $ 40 | $ 500 | $ 300 |
Adjusted fair value of options vested | $ 2,200 | $ 2,300 | $ 1,400 |
Weighted average grant date fair value of options granted (in dollars per share) | $ 0.85 | $ 2.89 | $ 7.68 |
Unrecognized compensation expense related to unvested stock option awards | $ 3,200 | ||
Weighted-average amortization period over which cost is expected to be recognized | 2 years | ||
Minimum | |||
Weighted average of the assumptions used to compute employee stock based compensation | |||
Weighted-average expected volatility (as a percent) | 76.10% | 72.00% | 73.60% |
Expected term (in years) | 6 years 3 months | 6 years 2 months 1 day | |
Risk-free interest rate (as a percent) | 2.00% | 1.24% | 0.92% |
Maximum | |||
Weighted average of the assumptions used to compute employee stock based compensation | |||
Weighted-average expected volatility (as a percent) | 81.90% | 73.90% | 86.80% |
Expected term (in years) | 9 years 9 months 7 days | 10 years | |
Risk-free interest rate (as a percent) | 2.38% | 2.00% | 2.45% |
Stock Incentive Plans - Employe
Stock Incentive Plans - Employee Stock Purchase Plan (Details) - shares | Jan. 01, 2018 | Jan. 01, 2017 | Jun. 30, 2015 | Dec. 31, 2017 | Dec. 31, 2016 |
Stock-Based Compensation | |||||
Common Stock, Issued, (in shares) | 23,645,247 | 18,817,572 | |||
2015 Plan | |||||
Stock-Based Compensation | |||||
Shares available for grant (in shares) | 1,068,287 | ||||
2015 Plan | Annual minimum increase | |||||
Stock-Based Compensation | |||||
Shares available for grant (in shares) | 1,297,334 | ||||
Percentage of outstanding Common Stock available for issuance (as a percent) | 4.00% | ||||
Potential increase in shares available for issuance as determined by the Board of Directors (in shares) | 945,810 | 752,702 | |||
Common Stock | 2015 ESPP | |||||
Stock-Based Compensation | |||||
Common Stock, Issued, (in shares) | 0 | ||||
Shares available for grant (in shares) | 182,352 | ||||
Common Stock | 2015 ESPP | Annual minimum increase | |||||
Stock-Based Compensation | |||||
Percentage of outstanding Common Stock available for issuance (as a percent) | 1.00% | ||||
Potential increase in shares available for issuance as determined by the Board of Directors (in shares) | 236,452 | 188,175 | 364,705 |
Income Taxes - Effective Tax Ra
Income Taxes - Effective Tax Rate Reconciliation (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Transition tax on foreign subsidiaries | $ 0 | |||
Amount of decrease in deferred tax assets | $ (19,800) | |||
Reconciliation of the U.S. statutory income tax rate to the effective income tax rate | ||||
Federal income tax (benefit) at statutory rate | 34.00% | 34.00% | 34.00% | |
Permanent differences | (1.16%) | (0.85%) | (0.86%) | |
Federal research and development credits and adjustments | 2.36% | 2.43% | 2.64% | |
State income tax benefit, net of federal benefit | 5.80% | 5.70% | 5.77% | |
Tax reform deferred rate change | (72.21) | |||
Other (as a percent) | (0.36%) | (0.40%) | 0.28% | |
Change in valuation allowance | 31.57% | (40.88%) | (41.82%) | |
Deferred tax assets | ||||
Net operating loss carryforwards | $ 41,436 | $ 48,682 | ||
Tax credit carryforwards | 5,896 | 4,833 | ||
Capitalized research and development | 1,879 | 3,394 | ||
Capitalized legal expenses | 1,156 | 1,597 | ||
Other differences | 1,048 | 1,335 | ||
Total deferred tax assets | 51,415 | 59,841 | ||
Valuation allowance | (51,415) | (59,841) | ||
Valuation allowance | ||||
Recorded increase (decrease) in valuation allowance | (8,400) | $ 14,700 | $ 13,600 | |
Federal | ||||
Valuation allowance | ||||
Net operating loss carryforwards | 152,100 | |||
Tax credit carryforwards | 4,500 | |||
State | ||||
Valuation allowance | ||||
Net operating loss carryforwards | 150,400 | |||
Tax credit carryforwards | 1,800 | |||
Accounting Standards Update 2016-09 | ||||
Valuation allowance | ||||
Recorded increase (decrease) in valuation allowance | $ 200 | |||
Forecast | ||||
Reconciliation of the U.S. statutory income tax rate to the effective income tax rate | ||||
Federal income tax (benefit) at statutory rate | 21.00% |
Subsequent Events (Details)
Subsequent Events (Details) - USD ($) $ / shares in Units, $ in Thousands | Jan. 01, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 |
Subsequent Events | ||||
Share price (in dollars per share) | $ 0.001 | |||
Additional common stock issued | $ 8,036 | $ 11,994 | $ 61,744 | |
Common Stock | ||||
Subsequent Events | ||||
Number of shares issued | 4,804,796 | 3,243,015 | 5,750,000 | |
Additional common stock issued | $ 5 | $ 3 | $ 5 | |
Second ATM Program | Common Stock | Subsequent Events | ||||
Subsequent Events | ||||
Number of shares issued | 5,390,255 | |||
Share price (in dollars per share) | $ 1.59 | |||
Additional common stock issued | $ 8,600 | |||
Net proceeds | $ 8,300 | |||
Second ATM Program | Cowen | Common Stock | Subsequent Events | ||||
Subsequent Events | ||||
Number of shares issued | 5,390,255 | |||
Share price (in dollars per share) | $ 1.59 | |||
Additional common stock issued | $ 8,600 | |||
Net proceeds | $ 8,300 |
Quarterly Financial Informati60
Quarterly Financial Information (Unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Quarterly Financial Information (Unaudited) | |||||||||||
Revenue | $ 250 | $ 250 | $ 500 | ||||||||
Operating expenses | 5,712 | 7,202 | $ 6,919 | $ 7,761 | $ 8,673 | $ 8,283 | $ 9,396 | $ 9,206 | 27,594 | $ 35,558 | $ 31,659 |
Net loss | $ (5,497) | $ (7,017) | $ (6,974) | $ (7,876) | $ (8,778) | $ (8,419) | $ (9,445) | $ (9,418) | $ (27,094) | $ (35,558) | $ (31,659) |
Net loss per share: | |||||||||||
Basic and Diluted (in dollars per share) | $ (0.24) | $ (0.31) | $ (0.32) | $ (0.41) | $ (0.47) | $ (0.54) | $ (0.61) | $ (0.61) | $ (1.26) | $ (2.22) | $ (4.06) |
Weighted-average common shares outstanding used in net loss per share: | |||||||||||
Basic and Diluted (in shares) | 23,218,476 | 22,563,174 | 21,796,194 | 19,093,273 | 18,699,480 | 15,512,608 | 15,373,964 | 15,335,516 | 21,681,534 | 16,230,190 | 8,041,948 |