Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Mar. 14, 2019 | Jun. 30, 2018 | |
Document And Entity Information [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2018 | ||
Document Fiscal Year Focus | 2018 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | CPRX | ||
Entity Registrant Name | CATALYST PHARMACEUTICALS, INC. | ||
Entity Central Index Key | 0001369568 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Accelerated Filer | ||
Entity Shell Company | false | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | false | ||
Entity Common Stock, Shares Outstanding | 102,739,257 | ||
Entity Public Float | $ 298,397,093 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) | Dec. 31, 2018 | Dec. 31, 2017 |
Current Assets: | ||
Cash and cash equivalents | $ 16,559,400 | $ 57,496,702 |
Short-term investments | 36,922,213 | 26,516,711 |
Inventory | 56,012 | 0 |
Prepaid expenses and other current assets | 1,649,781 | 1,173,744 |
Total current assets | 55,187,406 | 85,187,157 |
Investments | 5,008,243 | 0 |
Property and equipment, net | 245,425 | 191,385 |
Deposits | 8,888 | 8,888 |
Total assets | 60,449,962 | 85,387,430 |
Current Liabilities: | ||
Accounts payable | 2,337,367 | 1,945,575 |
Accrued expenses and other liabilities | 7,173,987 | 2,320,587 |
Total current liabilities | 9,511,354 | 4,266,162 |
Accrued expenses and other liabilities, non-current | 154,799 | 157,456 |
Total liabilities | 9,666,153 | 4,423,618 |
Commitments and contingencies | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at December 31, 2018 and 2017 | 0 | 0 |
Common stock, $0.001 par value, 150,000,000 shares authorized; 102,739,257 shares and 102,549,498 shares issued and outstanding at December 31, 2018 and 2017, respectively | 102,739 | 102,549 |
Additional paid-in capital | 211,265,279 | 207,421,710 |
Accumulated deficit | (160,563,961) | (126,560,447) |
Accumulated other comprehensive loss | (20,248) | |
Total stockholders' equity | 50,783,809 | 80,963,812 |
Total liabilities and stockholders' equity | $ 60,449,962 | $ 85,387,430 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2018 | Dec. 31, 2017 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, shares issued | 102,739,257 | 102,549,498 |
Common stock, shares outstanding | 102,739,257 | 102,549,498 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Income Statement [Abstract] | ||
Revenues from collaborative arrangement | $ 500,000 | $ 0 |
Operating costs and expenses: | ||
Research and development | 19,919,204 | 11,375,237 |
General and administrative | 15,875,961 | 7,304,399 |
Total operating costs and expenses | 35,795,165 | 18,679,636 |
Loss from operations | (35,295,165) | (18,679,636) |
Other income, net | 1,291,651 | 454,163 |
Change in fair value of warrants liability | (186,904) | |
Loss before income taxes | (34,003,514) | (18,412,377) |
Provision for income taxes | 0 | 0 |
Net loss | $ (34,003,514) | $ (18,412,377) |
Net loss per share - basic and diluted | $ (0.33) | $ (0.21) |
Weighted average shares outstanding - basic and diluted | 102,633,884 | 85,802,487 |
Net loss | $ (34,003,514) | $ (18,412,377) |
Other comprehensive loss: | ||
Unrealized gain (loss) on available-for-sale securities | (20,248) | |
Comprehensive loss | $ (34,023,762) | $ (18,412,377) |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholders' Equity - USD ($) | Total | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Deficit [Member] | Accumulated Other Comprehensive Loss [Member] |
Beginning Balance at Dec. 31, 2016 | $ 39,308,930 | $ 82,972 | $ 147,374,028 | $ (108,148,070) | |
Issuance of common stock, net | 53,772,560 | 16,455 | 53,756,105 | ||
Issuance of stock options for services | 2,342,625 | 2,342,625 | |||
Amortization of restricted stock for services | 65,336 | 65,336 | |||
Exercise of warrants for common stock | 3,518,553 | 2,258 | 3,516,295 | ||
Exercise of stock options for common stock | 368,185 | 864 | 367,321 | ||
Net loss | (18,412,377) | (18,412,377) | |||
Ending Balance at Dec. 31, 2017 | 80,963,812 | 102,549 | 207,421,710 | (126,560,447) | |
Issuance of common stock, net | 10,549 | 3 | 10,546 | ||
Issuance of stock options for services | 3,535,647 | 3,535,647 | |||
Exercise of stock options for common stock | 297,563 | 187 | 297,376 | ||
Other comprehensive loss | (20,248) | $ (20,248) | |||
Net loss | (34,003,514) | (34,003,514) | |||
Ending Balance at Dec. 31, 2018 | $ 50,783,809 | $ 102,739 | $ 211,265,279 | $ (160,563,961) | $ (20,248) |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Operating Activities: | ||
Net loss | $ (34,003,514) | $ (18,412,377) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation | 37,978 | 45,689 |
Stock-based compensation | 3,550,644 | 2,407,961 |
Change in fair value of warrants liability | 186,904 | |
(Increase) decrease in: | ||
Inventory | (56,012) | |
Prepaid expenses and other current assets and deposits | (476,037) | (125,800) |
Increase (decrease) in: | ||
Accounts payable | 391,792 | 1,012,399 |
Accrued expenses and other liabilities | 4,850,743 | 1,142,652 |
Net cash used in operating activities | (25,704,406) | (13,742,572) |
Investing Activities: | ||
Purchases of property and equipment | (92,018) | |
Purchases of investments | (36,802,418) | (3,958) |
Proceeds from maturities of investments | 21,368,425 | |
Net cash provided by (used in) investing activities | (15,526,011) | (3,958) |
Financing Activities: | ||
Proceeds from issuance of common stock, net | 53,772,560 | |
Payment of employee withholding tax related to stock-based compensation | (4,448) | |
Proceeds from exercise of warrants | 3,209,423 | |
Proceeds from exercise of stock options | 297,563 | 368,185 |
Net cash provided by (used in) financing activities | 293,115 | 57,350,168 |
Net increase (decrease) in cash and cash equivalents | (40,937,302) | 43,603,638 |
Cash and cash equivalents - beginning of period | 57,496,702 | 13,893,064 |
Cash and cash equivalents - end of period | 16,559,400 | 57,496,702 |
Non-cash investing and financing activities: | ||
Unrealized gain (loss) on available-for-sale securities | $ (20,248) | |
Exercise of liability classified warrants for common stock | $ 309,130 |
Organization and Description of
Organization and Description of Business | 12 Months Ended |
Dec. 31, 2018 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization and Description of Business | 1. Organization and Description of Business. Catalyst Pharmaceuticals, Inc. and subsidiary (collectively, the “Company”), is a development-stage biopharmaceutical company focused on developing and commercializing innovating therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndromes (CMS), and MuSK antibody positive myasthenia gravis (MuSK-MG). On November 28, 2018, the U.S. Food and Drug Administration, or FDA, granted approval of Firdapse ® Since inception, the Company has devoted substantially all of its efforts to business planning, research and development, recruiting management and technical staff, acquiring operating assets and raising capital. The Company’s primary focus is on the development and commercialization of its drug candidates. The Company has incurred operating losses in each period from inception through December 31, 2018. The Company has been able to fund its cash needs to date primarily through public and private offerings of its securities. See Note 11. Capital Resources While there can be no assurance, based on currently available information, the Company estimates that it currently has sufficient resources to support its operations for at least the next 12 months from the issuance date of this Form 10-K. The Company may raise required funds in the future through public or private equity offerings, debt financings, corporate collaborations, governmental research grants or other means. The Company may also seek to raise new capital to fund additional product development efforts, even if it has sufficient funds for its planned operations. Any sale by the Company of additional equity or convertible debt securities could result in dilution to the Company’s current stockholders. There can be no assurance that any such required additional funding will be available to the Company at all or available on terms acceptable to the Company. Further, to the extent that the Company raises additional funds through collaborative arrangements, it may be necessary to relinquish some rights to the Company’s drug candidates or grant sublicenses on terms that are not favorable to the Company. If the Company is not able to secure additional funding when needed, the Company may have to delay, reduce the scope of, or eliminate one or more research and development programs, which could have an adverse effect on the Company’s business. |
Basis of Presentation and Signi
Basis of Presentation and Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Significant Accounting Policies | 2. Basis of Presentation and Significant Accounting Policies. a. PRINCIPLES OF CONSOLIDATION b. USE OF ESTIMATES. c. CASH AND CASH EQUIVALENTS. d. INVESTMENTS. Short-term bond fund The short-term bond fund is classified as trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognizes realized gains and losses and unrealized gains and losses to earnings. At December 31, 2018 and 2017, the only investment classified as trading securities was the short-term bond fund. Unrealized gain (loss) on trading securities was ($29,430) and $29,430, respectively, for the years ended December 31, 2018, and 2017 and is included in other income, net in the accompanying consolidated statements of operations. U.S. Treasuries U.S. Treasuries are classified as available-for-sale available-for-sale Available-for-sale non-current available-for-sale available-for-sale available-for-sale e. INVENTORY. first-in-first-out ® ® Products that have been approved by the FDA or other regulatory authorities, such as Firdapse ® ® The Company evaluates for potential excess inventory by analyzing current and future product demand relative to the remaining product shelf life. The Company builds demand forecasts by considering factors such as, but not limited to, overall market potential, market share, market acceptance, and patient usage. f. PREPAID EXPENSES AND OTHER CURRENT ASSETS. pre-commercialization g PROPERTY AND EQUIPMENT. h OPERATING LEASES. i. FAIR VALUE OF FINANCIAL INSTRUMENTS. j. FAIR VALUE MEASUREMENTS. Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant (Level 2) Significant (Level 3) Cash and cash equivalents: Money market funds $ 14,462,087 $ 14,462,087 $ — $ — Short-term investments: Short-term bond fund $ 26,541,349 $ 26,541,349 $ — $ — U.S. Treasuries $ 10,380,864 $ — $ 10,380,864 $ — Investments: U.S. Treasuries $ 5,008,243 $ — $ 5,008,243 $ — Balances as of Quoted Prices in Significant Significant (Level 3) Cash and cash equivalents: Money market funds $ 56,820,688 $ 56,820,688 $ — $ — Short-term investments: Short-term bond fund $ 26,516,711 $ 26,516,711 $ — $ — k. WARRANTS LIABILITY. The following table rolls forward the fair value of the Company’s warrants liability activity for the year ended December 31, 2017. On May 2, 2017, all outstanding and unexercised 2011 warrants expired. 2017 Fair value, beginning of period $ 122,226 Issuance of warrants — Exercise of warrants (309,130 ) Change in fair value 186,904 Fair value, end of period $ — During the year ended December 31, 2017, 613,913 of the 2011 warrants were exercised, with proceeds of $798,087 to the Company. l. REVENUES FROM COLLABORATIVE ARRANGEMENT ® Nonrefundable upfront license fees are recognized upon receipt as persuasive evidence of an arrangement exists, the price to the collaborator is fixed or determinable and collectability is reasonably assured. An upfront license fee of $500,000 was recognized in the year ended December 31, 2018 when it was paid following execution of the collaborative arrangement for vigabatrin tablets. Refer to Note 7, Collaborative Arrangement, for further discussion on the Company’s collaborative arrangement. m. RESEARCH AND DEVELOPMENT. n. STOCK-BASED COMPENSATION o. CONCENTRATION OF CREDIT RISK. p. INCOME TAXES The Company recognizes the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the more-likely-than-not On December 22, 2017, the U.S. government enacted comprehensive tax legislation commonly referred to as the Tax Cuts and Jobs Act (the “Tax Act”). The Tax Act makes broad and complex changes to the U.S. tax code, including, but not limited to, (1) reducing the U.S. federal corporate tax rate from 35 to 21 percent; (2) requiring companies to pay a one-time q. COMPREHENSIVE INCOME (LOSS). available-for-sale r. NET LOSS PER SHARE 2018 2017 Options to purchase common stock 10,532,500 5,191,666 Potential equivalent common stock excluded 10,532,500 5,191,666 Potentially dilutive stock options to purchase common stock as of December 31, 2018, had exercise prices ranging from $0.79 to $4.64. Potentially dilutive stock options to purchase common stock as of December 31, 2017 had exercise prices ranging from $0.47 to $4.64. s. SEGMENT INFORMATION. t. RECLASSIFICATIONS. u. RECENTLY ISSUED ACCOUNTING STANDARDS. No. 2016-02, Leases (Topic 842), 2016-02 In May 2017, the FASB issued ASU No. 2017-09, Compensation – Stock Compensation (Topic 718): Scope of Modification Accounting 2017-09 In June 2018, the FASB issued ASU No. 2018-07, Compensation – Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting 2018-07 In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments |
Investments
Investments | 12 Months Ended |
Dec. 31, 2018 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | 3. Investments. Available-for-sale Amortized Gross Gross Estimated At December 31, 2018: U.S. Treasuries – ST $ 10,382,699 $ — $ (1,835 ) $ 10,380,864 U.S. Treasuries – LT 5,026,656 — (18,413 ) 5,008,243 Total $ 15,409,355 $ — $ (20,248 ) $ 15,389,107 At December 31, 2017, the Company did not have any available-for-sale In accordance with FASB ASC Topic 320, “Investments – Debt and Equity Securities” available-for-sale available-for-sale Certain U.S. Treasuries at December 31, 2018 had fair values less than their amortized costs and, therefore, contained unrealized losses. Given that the Company has no intent to sell the U.S. Treasuries until a recovery of the fair value, which may be at maturity, and there are no current requirements to sell any of these securities, as of December 31, 2018, the Company did not consider these investments to be other-than-temporarily impaired. The Company anticipates full recovery of amortized costs with respect to these investments at maturity. The duration of time the U.S. Treasuries have been in a continuous unrealized loss position as of December 31, 2018 was less than 12 months. The estimated fair values of available-for-sale 2018 Due in one year or less $ 10,380,864 Due after one year but within two years 5,008,243 $ 15,389,107 |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 12 Months Ended |
Dec. 31, 2018 | |
Text Block [Abstract] | |
Prepaid Expenses and Other Current Assets | 4. Prepaid Expenses and Other Current Assets. Prepaid expenses and other current assets consist of the following as of December 31: 2018 2017 Prepaid research fees $ 358,209 $ 388,977 Prepaid insurance 800,261 638,139 Prepaid pre-commercialization 17,030 65,000 Prepaid subscriptions fees 170,552 23,347 Prepaid rent 31,561 — Other 272,168 58,281 Total prepaid expenses and other current assets $ 1,649,781 $ 1,173,744 |
Property and Equipment, net
Property and Equipment, net | 12 Months Ended |
Dec. 31, 2018 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | 5. Property and Equipment, net. Property and equipment, net consists of the following as of December 31: 2018 2017 Computer equipment $ 52,704 $ 27,915 Furniture and equipment 212,451 169,931 Leasehold improvements 177,417 152,708 442,572 350,554 Less: Accumulated depreciation (197,147 ) (159,169 ) Total property and equipment, net $ 245,425 $ 191,385 Depreciation expense was $37,978 and $45,689, respectively, for the years ended December 31, 2018 and 2017. |
Accrued Expenses and Other Liab
Accrued Expenses and Other Liabilities | 12 Months Ended |
Dec. 31, 2018 | |
Payables and Accruals [Abstract] | |
Accrued Expenses and Other Liabilities | 6. Accrued Expenses and Other Liabilities. Accrued expenses and other liabilities consist of the following as of December 31: 2018 2017 Accrued preclinical and clinical trial expenses $ 821,633 $ 970,649 Accrued professional fees 1,311,061 227,457 Accrued compensation and benefits 1,941,449 821,935 Accrued license fees 3,000,000 252,500 Deferred rent and lease incentive 33,408 24,011 Other 66,436 24,035 Current accrued expenses and other liabilities 7,173,987 2,320,587 Deferred rent and lease incentive – non-current 154,799 157,456 Non-current 154,799 157,456 Total accrued expenses and other liabilities $ 7,328,786 $ 2,478,043 |
Collaborative Arrangement
Collaborative Arrangement | 12 Months Ended |
Dec. 31, 2018 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaborative Arrangement | 7. Collaborative Arrangement. In December 2018, the Company entered into a collaboration and license agreement with Endo International plsc’s subsidiary, Endo Ventures Limited (EVL), for the further development and commercialization of generic Sabril ® EVL assumes all development, manufacturing, clinical, regulatory, sales and marketing costs under the collaboration, while the Company is responsible for exercising commercially reasonable efforts to develop, or cause the development of, a final finished, stable dosage form of generic Sabril ® Under the terms of the Collaboration, the Company has received an up-front mid-double ® |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2018 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 8. Commitments and Contingencies. The Company has contracted with drug manufacturers and other vendors, including clinical research organizations (CRO) overseeing one or more of the clinical trials of the Company’s drug candidates, to assist in the execution of the Company’s preclinical and clinical trials, analysis, and the preparation of material necessary for the submission of new drug applications (NDAs) and abbreviated new drug applications (ANDAs) with the U.S. FDA. The contracts are cancelable at any time, but obligate the Company to reimburse the providers for any time or costs incurred through the date of termination. The Company has executed operating lease agreements for its corporate office. The leases have free and escalating rent payment provisions. The Company recognizes rent expense under such leases on a straight-line basis over the term of the lease. As of December 31, 2018, future minimum lease payments under the operating lease agreements are as follows: 2019 $ 329,725 2020 339,612 2021 349,792 2022 329,662 $ 1,348,791 In August 2018, the Company entered into a fourth amendment to the lease to its corporate offices to obtain additional space for its operations. The Company now leases approximately 7,800 square feet and the lease term expires in November 2022. In connection with the expansion, approximately $13,000 of tenant build-out There are no obligations under capital leases. On August 27, 2009, the Company entered into a license agreement with Northwestern University (Northwestern), under which it acquired worldwide rights to CPP-115. In 2018, the Company became aware that certain patents granted to Northwestern in 2018 (which patents have been licensed by Northwestern to a third-party) for a new GABA aminotransferase inhibitor were derived from CPP-115. On November 5, 2018, Northwestern advised the Company that in its view, Northwestern has a right to terminate the license agreement with the Company because the Company has allegedly breached the license agreement by failing to pay certain milestones and by allegedly failing to use commercially reasonable efforts to develop and commercialize any products. Northwestern has also advised the Company that, in its view, the Company has engaged in wrongful conduct and communications with the third party that licensed the new patents from Northwestern, and that such communications have damaged Northwestern’s relationship with that third party. The Company disputes Northwestern’s allegations and is vigorously defending itself against claims that Northwestern has brought against it in the arbitration proceeding. There can be no assurance as to the outcome of this matter. The Company has an employment agreement with its Chief Executive Officer. Under this agreement, the CEO received an annual base salary of approximately $525,000 in 2018. This agreement expires in November 2020. |
Agreements
Agreements | 12 Months Ended |
Dec. 31, 2018 | |
Text Block [Abstract] | |
Agreements | 9. Agreements. a. LICENSE AGREEMENT WITH BIOMARIN (FIRDAPSE ® ® Under the License Agreement, the Company has agreed to pay: (i) royalties to BioMarin for seven years from the first commercial sale of Firdapse ® ® Under the Company’s license agreement with BioMarin, the Company agreed to pay certain milestone payments that BioMarin was obligated to pay to both a third-party licensor of the rights that have been sublicensed to us and to the former stockholders of Huxley Pharmaceuticals (“Huxley”) under an earlier stock purchase agreement between BioMarin and the former Huxley stockholders. In full satisfaction of the milestone obligations, the Company has paid (i) $3,150,000 in milestone payments to the third party licensor of the rights that have been sublicensed to the Company ($3.0 million of which was paid in February 2019 and the balance of which was paid in 2018) and (ii) $2.0 million in milestone payments to the former Huxley Stockholders (all of which was paid in 2018). In the BioMarin License Agreement, the Company agreed to share in the cost of certain post-marketing studies of Firdapse ® b AGREEMENTS FOR DRUG DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 10. Income Taxes. Due to the ongoing operating losses and the inability to recognize any income tax benefit, there is no provision for income taxes in any period presented in these financial statements. Since inception, the Company has only generated pretax losses. The reconciliation of income tax expense (benefit) computed at the statutory federal income tax rate of 21% to amounts included in the statements of operations is as follows: 2018 2017 Statutory rate 21.0 % 34.0 % State tax 4.2 % 3.5 % Valuation allowance (25.9 )% 26.5 % Federal rate change 0.0 % (73.2 )% Tax credit 1.4 % 6.8 % Other (0.7 )% 2.4 % 0.0 % 0.0 % Deferred tax assets and liabilities reflect the net tax effects of net operating loss and tax credit carryovers and the temporary differences between the carrying amounts of assets and liabilities for financial reporting and the amounts used for income tax purposes. Significant components of the Company’s deferred tax assets as of December 31, 2018 and 2017 are as follows: 2018 2017 Net operating loss $ 19,867,591 $ 15,718,570 Start-up 13,861,147 10,508,487 Tax credits 12,625,275 11,582,134 Deferred compensation 1,919,434 1,326,189 Other 109,779 72,395 Gross deferred tax asset 48,383,226 39,207,775 Valuation allowance (48,383,226 ) (39,207,775 ) Net deferred tax assets $ — $ — The Company’s deferred tax assets have been fully offset by a valuation allowance at December 31, 2018 and 2017 because the Company believes that it is more likely than not that the deferred tax asset will not be realized. The increase and decrease in the valuation allowance on the deferred tax assets was $9,175,451 and $3,388,951 for the years ended December 31, 2018 and 2017, respectively. At December 31, 2018 and 2017, respectively, the Company had net operating loss carryforwards of approximately $79.0 million and $62.6 million available to reduce future taxable income, if any. The net operating loss carryforwards will expire at various dates beginning in 2023 and ending in 2037, the amount of net operating loss generated in 2018 will have an infinite life, but will be limited to utilization per year of 80% of taxable income. If an ownership change, as defined under Internal Revenue Code Section 382, occurs, the use of these carry-forwards may be subject to limitation. The effective tax rate of 0% in all periods presented differs from the statutory rate of 21% due to the valuation allowance and because the Company had no taxable income. Beginning in 2010, the Company has received several orphan drug designations by the FDA for products currently under development. The orphan drug designations allow the Company to claim increased federal tax credits for certain research and development activities. No interest or penalties were accrued through December 31, 2018. The Company’s policy is to recognize any related interest or penalties in income tax expense. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for any years before 2015. The Company is not currently under income tax examinations by any tax authorities. |
Stockholders' Equity
Stockholders' Equity | 12 Months Ended |
Dec. 31, 2018 | |
Equity [Abstract] | |
Stockholders' Equity | 11. Stockholders’ Equity. Preferred Stock The Company has 5,000,000 shares of authorized preferred stock, $0.001 par value per share at December 31, 2018 and 2017. No shares of preferred stock were outstanding at December 31, 2018 and 2017. Common Stock The Company has 150,000,000 shares of authorized common stock, par value $0.001 per share. At December 31, 2018 and 2017, 102,739,257 and 102,549,498 shares, respectively, of common stock were issued and outstanding. Each holder of common stock is entitled to one vote of each share of common stock held of record on all matters on which stockholders generally are entitled to vote. 2016 Shelf Registration Statement On December 23, 2016, the Company filed a shelf Registration Statement on Form S-3 No. 333-215315) 2017 Shelf Registration Statement On July 12, 2017, the Company filed a universal shelf Registration Statement on Form S-3 No. 333-219259) On November 28, 2017, the Company filed a prospectus supplement and offered for sale 16,428,572 shares of its common stock at a price of $3.50 per share in an underwritten public offering under the 2017 Shelf Registration. The Company received gross proceeds in the public offering of approximately $57.5 million before underwriting commission and incurred expenses of approximately $3.7 million. At December 31, 2018, there is approximately $92.5 million available for future sale under the 2017 Shelf Registration Statement. Warrant Exercises All unexercised warrants expired in 2017. For the year ended December 31, 2017, the Company issued 2,257,663 shares of its authorized but unissued common stock upon the exercise of previously issued common stock purchase warrants, with net proceeds to the Company of $3,209,423. Stockholder Rights Plan On September 20, 2011, the Board of Directors approved the Company’s adoption of a Stockholder Rights Plan. Under the Plan, a dividend of one preferred share purchase right (a Right) was declared for each share of common stock of the Company that was outstanding on October 7, 2011. Each Right entitles the holder to purchase from the Company one one-hundredth The Rights trade automatically with the common stock and will not be exercisable until a person or group has become an “acquiring person” by acquiring 17.5% or more of the Company’s outstanding common stock, or a person or group commences, or publicly announces a tender offer that will result in such a person or group owning 17.5% or more of the Company’s outstanding common stock. Upon announcement that any person or group has become an acquiring person, each Right will entitle all rightholders (other than the acquiring person) to purchase, for the exercise price of $7.80, a number of shares of the Company’s common stock having a market value equal to twice the exercise price. Rightholders would also be entitled to purchase common stock of the acquiring person having a value of twice the exercise price if, after a person had become an acquiring person, the Company were to enter into certain mergers or other transactions. If any person becomes an acquiring person, the Board of Directors may, at its option and subject to certain limitations, exchange one share of common stock for each Right. The Rights have certain anti-takeover effects, in that they would cause substantial dilution to a person or group that attempts to acquire a significant interest in the Company on terms not approved by the Board of Directors. In the event that the Board of Directors determines a transaction to be in the best interests of the Company and its stockholders, the Board of Directors may redeem the Rights for $0.001 per share at any time prior to a person or group becoming an acquiring person. On September 19, 2016, the Board of Directors unanimously approved, and on the same date the Company entered into Amendment No. 1 to the Stockholders Rights Plan (the “Amendment”). Under the terms of the Amendment, the outside expiration date of the rights plan has been extended from September 20, 2016 to September 20, 2019. Additionally, as part of the Amendment, the Board adopted a Certificate of Designation, Preferences and Rights of Series A Junior Participating Preferred Stock of the Company to increase the number of shares of Series A Junior Participating Preferred Stock of the Company available for issuance under the Rights Plan from 500,000 shares to 1.5 million shares. |
Stock Compensation Plans
Stock Compensation Plans | 12 Months Ended |
Dec. 31, 2018 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock Compensation Plans | 12. Stock Compensation Plans. For the years ended December 31, 2018 and 2017, the Company recorded stock-based compensation expense as follows: 2018 2017 Research and development $ 1,079,230 $ 785,899 General and administrative 2,471,414 1,622,062 Total stock-based compensation $ 3,550,644 $ 2,407,961 The Company may issue stock options, restricted stock, stock appreciation rights and restricted stock units (collectively, the “Awards”) to employees, directors, and consultants of the Company under the 2014 and 2018 Stock Incentive Plans (the 2014 Plan and the 2018 Plan or collectively, the Plans). At December 31, 2018, no shares remain available for future issuance under the 2014 Plan. Under the 2018 Plan, 7,500,000 shares were reserved for issuance and as of December 31, 2018, 3,620,603 shares remain available for future issuance. Stock Options The Company has granted stock options to employees, officers, directors, and consultants generally at exercise prices equal to the market price of the common stock at grant date. Option awards generally vest over a period of 1 to 5 years of continuous service and have contractual terms from 5 to 7 years. Certain awards provide for accelerated vesting if there is a change in control. The Company issues new shares as shares are required to be delivered upon exercise of outstanding stock options. During the years ended December 31, 2018 and 2017, options to purchase 186,665 and 780,000 shares of the Company’s common stock were exercised with gross proceeds to the Company of $297,563 and $368,185, respectively. Further, during the year ended December 31, 2018, no options to purchase shares of the Company’s common stock were exercised on a “cashless” basis. During the year ended December 31, 2017, options to purchase 100,000 shares of the Company’s common stock were exercised on a “cashless” basis, resulting in the issuance of an aggregate of 84,280 shares of the Company’s common stock, respectively. During the years ended December 31, 2018 and 2017 the Company recorded non-cash During the years ended December 31, 2018 and 2017, the Company granted seven-year options to purchase an aggregate of 5,822,500 and 1,550,000 shares, respectively, of the Company’s common stock to certain of the Company’s officers, employees, directors, and consultants. Stock option activity under the Company’s Plans for the year ended December 31, 2018 is summarized as follows: Number of Weighted Weighted Aggregate Outstanding at beginning of year 5,191,666 $ 1.96 Granted 5,822,500 3.04 Exercised or released (186,665 ) 1.59 Forfeited or cancelled (270,001 ) 2.94 Expired (25,000 ) 0.47 Outstanding at end of year 10,532,500 $ 2.54 5.39 $ 2,239,500 Exercisable at end of year 4,349,996 $ 2.16 4.00 $ 1,679,297 Other information pertaining to stock option activity during the years ended December 31, 2018 and 2017 was as follows: 2018 2017 Weighted–average fair value of granted stock options $ 1.93 $ 0.91 Total fair value of vested stock options $ 2,193,294 $ 2,016,992 Total intrinsic value of exercised stock options $ 274,864 $ 2,296,100 The following table summarizes information about the Company’s options outstanding at December 31, 2018: Options Outstanding Options Exercisable Range of Exercise Prices Number Weighted Weighted Number Weighted Weighted $0.79 to $1.13 2,385,000 4.78 $ 0.99 1,686,664 4.69 $ 0.93 $1.14 to $2.28 2,440,000 6.87 $ 2.22 209,999 6.22 $ 2.07 $2.29 to $3.07 1,765,000 4.84 $ 2.60 1,098,333 3.72 $ 2.53 $3.08 to $3.75 2,005,000 4.59 $ 3.30 995,000 2.71 $ 3.14 $3.76 to $4.64 1,937,500 5.62 $ 4.03 360,000 3.92 $ 4.11 10,532,500 5.39 $ 2.54 4,349,996 4.00 $ 2.16 As of December 31, 2018, there was approximately $8.8 million of unrecognized compensation expense related to non-vested The Company utilizes the Black-Scholes option-pricing model to determine the fair value of stock options on the date of grant. This model derives the fair value of stock options based on certain assumptions related to the expected stock price volatility, expected option life, risk-free interest rate and dividend yield. Expected volatility is based on reviews of historical volatility of the Company’s common stock. The estimated expected option life is based upon estimated employee exercise patterns and considers whether and the extent to which the options are in-the-money. mid-point Assumptions used during the years were as follows: December 31, December 31, Risk free interest rate 2.09% to 2.88% 1.66% to 2.25% Expected term 0 to 7 years 4 to 7 years Expected volatility 82% 104% Expected dividend yield — % — % Expected forfeiture rate — % — % Restricted Stock Units Under the 2018 Plan, participants may be granted restricted stock units, each of which represents a conditional right to receive shares of common stock in the future. The restricted stock units granted under this plan generally vest ratably over a four-year period. Upon vesting, the restricted stock units will convert into an equivalent number of shares of common stock. The amount of expense relating to the restricted stock units is based on the closing market price of the Company’s common stock on the date of grant and is amortized on a straight-line basis over the requisite service period. No restricted stock units were granted during 2018. Restricted stock unit activity during 2017 was as follows: 2017 Number of Restricted Weighted Average Nonvested balance at beginning of year 26,667 $ 2.83 Granted — — Vested (26,667 ) 2.83 Forfeited — — Nonvested balance at end of year — $ — No stock-based compensation related to restricted stocks was recorded during 2018. During the year ended December 31, 2017, the Company recorded non-cash Common Stock During the year ended December 31, 2018, the Company granted 3,094 net shares of common stock to an employee as compensation. The Company recorded stock-based compensation related to common stock issued to an employee totaling approximately $15,000, during the year ended December 31, 2018. No shares of common stock were granted during the year ended December 31, 2017. |
Benefit Plan
Benefit Plan | 12 Months Ended |
Dec. 31, 2018 | |
Retirement Benefits [Abstract] | |
Benefit Plan | 13. Benefit Plan. The Company maintains an employee savings plan pursuant to Section 401(k) of the Internal Revenue Code covering all eligible employees. Subject to certain dollar limits, eligible employees may contribute up to 15% of their pre-tax |
Quarterly Financial Information
Quarterly Financial Information (unaudited) | 12 Months Ended |
Dec. 31, 2018 | |
Quarterly Financial Information Disclosure [Abstract] | |
Quarterly Financial Information (unaudited) | 14. Quarterly Financial Information (unaudited). The following table presents unaudited supplemental quarterly financial information for the years ended December 31, 2018 and 2017: Quarter Ended March 31, 2018 June 30, 2018 September 30, 2018 December 31, 2018 Revenues $ — $ — $ — $ 500,000 Loss from operations $ (5,933,440 ) $ (6,335,855 ) $ (8,182,603 ) $ (14,843,267 ) Net loss $ (5,699,892 ) $ (5,965,140 ) $ (7,838,873 ) $ (14,499,609 ) Net loss per share – basic and diluted $ (0.06 ) $ (0.06 ) $ (0.08 ) $ (0.14 ) Quarter Ended March 31, 2017 June 30, 2017 September 30, 2017 December 31, 2017 Revenues $ — $ — $ — $ — Loss from operations $ (4,679,871 ) $ (4,181,271 ) $ (4,306,708 ) $ (5,511,786 ) Change in fair value of warrants liability $ (397,235 ) $ 210,331 $ — $ — Net loss $ (4,967,129 ) $ (3,879,901 ) $ (4,177,649 ) $ (5,387,698 ) Net loss per share – basic and diluted $ (0.06 ) $ (0.05 ) $ (0.05 ) $ (0.06 ) Quarterly basic and diluted net loss per common share were computed independently for each quarter and do not necessarily total to the full year basic and diluted net loss per common share. |
Subsequent Events
Subsequent Events | 12 Months Ended |
Dec. 31, 2018 | |
Subsequent Events [Abstract] | |
Subsequent Events | 15. Subsequent Events. On January 15, 2019, the Company launched its first product, Firdapse ® |
Basis of Presentation and Sig_2
Basis of Presentation and Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
PRINCIPLES OF CONSOLIDATION. | a. PRINCIPLES OF CONSOLIDATION |
USE OF ESTIMATES | b. USE OF ESTIMATES. |
CASH AND CASH EQUIVALENTS | c. CASH AND CASH EQUIVALENTS. |
INVESTMENTS | d. INVESTMENTS. Short-term bond fund The short-term bond fund is classified as trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognizes realized gains and losses and unrealized gains and losses to earnings. At December 31, 2018 and 2017, the only investment classified as trading securities was the short-term bond fund. Unrealized gain (loss) on trading securities was ($29,430) and $29,430, respectively, for the years ended December 31, 2018, and 2017 and is included in other income, net in the accompanying consolidated statements of operations. U.S. Treasuries U.S. Treasuries are classified as available-for-sale available-for-sale Available-for-sale non-current available-for-sale available-for-sale available-for-sale |
INVENTORY | e. INVENTORY. first-in-first-out ® ® Products that have been approved by the FDA or other regulatory authorities, such as Firdapse ® ® The Company evaluates for potential excess inventory by analyzing current and future product demand relative to the remaining product shelf life. The Company builds demand forecasts by considering factors such as, but not limited to, overall market potential, market share, market acceptance, and patient usage. |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | f. PREPAID EXPENSES AND OTHER CURRENT ASSETS. pre-commercialization |
PROPERTY AND EQUIPMENT | g PROPERTY AND EQUIPMENT. |
OPERATING LEASES | h OPERATING LEASES. |
FAIR VALUE OF FINANCIAL INSTRUMENTS | i. FAIR VALUE OF FINANCIAL INSTRUMENTS. |
FAIR VALUE MEASUREMENTS | j. FAIR VALUE MEASUREMENTS. Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant (Level 2) Significant (Level 3) Cash and cash equivalents: Money market funds $ 14,462,087 $ 14,462,087 $ — $ — Short-term investments: Short-term bond fund $ 26,541,349 $ 26,541,349 $ — $ — U.S. Treasuries $ 10,380,864 $ — $ 10,380,864 $ — Investments: U.S. Treasuries $ 5,008,243 $ — $ 5,008,243 $ — Balances as of Quoted Prices in Significant Significant (Level 3) Cash and cash equivalents: Money market funds $ 56,820,688 $ 56,820,688 $ — $ — Short-term investments: Short-term bond fund $ 26,516,711 $ 26,516,711 $ — $ — |
WARRANTS LIABILITY | k. WARRANTS LIABILITY. The following table rolls forward the fair value of the Company’s warrants liability activity for the year ended December 31, 2017. On May 2, 2017, all outstanding and unexercised 2011 warrants expired. 2017 Fair value, beginning of period $ 122,226 Issuance of warrants — Exercise of warrants (309,130 ) Change in fair value 186,904 Fair value, end of period $ — During the year ended December 31, 2017, 613,913 of the 2011 warrants were exercised, with proceeds of $798,087 to the Company. |
REVENUES FROM COLLABORATIVE ARRANGEMENT | l. REVENUES FROM COLLABORATIVE ARRANGEMENT ® Nonrefundable upfront license fees are recognized upon receipt as persuasive evidence of an arrangement exists, the price to the collaborator is fixed or determinable and collectability is reasonably assured. An upfront license fee of $500,000 was recognized in the year ended December 31, 2018 when it was paid following execution of the collaborative arrangement for vigabatrin tablets. Refer to Note 7, Collaborative Arrangement, for further discussion on the Company’s collaborative arrangement. |
RESEARCH AND DEVELOPMENT | m. RESEARCH AND DEVELOPMENT. |
STOCK-BASED COMPENSATION | STOCK-BASED COMPENSATION |
CONCENTRATION OF CREDIT RISK | o. CONCENTRATION OF CREDIT RISK. |
INCOME TAXES | p. INCOME TAXES The Company recognizes the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the more-likely-than-not On December 22, 2017, the U.S. government enacted comprehensive tax legislation commonly referred to as the Tax Cuts and Jobs Act (the “Tax Act”). The Tax Act makes broad and complex changes to the U.S. tax code, including, but not limited to, (1) reducing the U.S. federal corporate tax rate from 35 to 21 percent; (2) requiring companies to pay a one-time |
COMPREHENSIVE INCOME (LOSS) | COMPREHENSIVE INCOME (LOSS). available-for-sale |
NET LOSS PER SHARE | r. NET LOSS PER SHARE 2018 2017 Options to purchase common stock 10,532,500 5,191,666 Potential equivalent common stock excluded 10,532,500 5,191,666 Potentially dilutive stock options to purchase common stock as of December 31, 2018, had exercise prices ranging from $0.79 to $4.64. Potentially dilutive stock options to purchase common stock as of December 31, 2017 had exercise prices ranging from $0.47 to $4.64. |
SEGMENT INFORMATION | s. SEGMENT INFORMATION. |
RECLASSIFICATIONS | t. RECLASSIFICATIONS. |
RECENTLY ISSUED ACCOUNTING STANDARDS | u. RECENTLY ISSUED ACCOUNTING STANDARDS. No. 2016-02, Leases (Topic 842), 2016-02 In May 2017, the FASB issued ASU No. 2017-09, Compensation – Stock Compensation (Topic 718): Scope of Modification Accounting 2017-09 In June 2018, the FASB issued ASU No. 2018-07, Compensation – Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting 2018-07 In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments |
Basis of Presentation and Sig_3
Basis of Presentation and Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Fair Value Measurement Specific to Assets or Liability | The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant (Level 2) Significant (Level 3) Cash and cash equivalents: Money market funds $ 14,462,087 $ 14,462,087 $ — $ — Short-term investments: Short-term bond fund $ 26,541,349 $ 26,541,349 $ — $ — U.S. Treasuries $ 10,380,864 $ — $ 10,380,864 $ — Investments: U.S. Treasuries $ 5,008,243 $ — $ 5,008,243 $ — Balances as of Quoted Prices in Significant Significant (Level 3) Cash and cash equivalents: Money market funds $ 56,820,688 $ 56,820,688 $ — $ — Short-term investments: Short-term bond fund $ 26,516,711 $ 26,516,711 $ — $ — |
Summary of Company's Warrants Liability Activity | The following table rolls forward the fair value of the Company’s warrants liability activity for the year ended December 31, 2017. On May 2, 2017, all outstanding and unexercised 2011 warrants expired. 2017 Fair value, beginning of period $ 122,226 Issuance of warrants — Exercise of warrants (309,130 ) Change in fair value 186,904 Fair value, end of period $ — |
Potential Shares Excluded from Determination of Basic and Diluted Net Loss Per Share | r. NET LOSS PER SHARE 2018 2017 Options to purchase common stock 10,532,500 5,191,666 Potential equivalent common stock excluded 10,532,500 5,191,666 |
Investments (Tables)
Investments (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available-for-Sale Investments by Security type | Available-for-sale Amortized Gross Gross Estimated At December 31, 2018: U.S. Treasuries – ST $ 10,382,699 $ — $ (1,835 ) $ 10,380,864 U.S. Treasuries – LT 5,026,656 — (18,413 ) 5,008,243 Total $ 15,409,355 $ — $ (20,248 ) $ 15,389,107 |
Estimated Fair Values of Available for Sale Securities | The estimated fair values of available-for-sale 2018 Due in one year or less $ 10,380,864 Due after one year but within two years 5,008,243 $ 15,389,107 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Text Block [Abstract] | |
Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consist of the following as of December 31: 2018 2017 Prepaid research fees $ 358,209 $ 388,977 Prepaid insurance 800,261 638,139 Prepaid pre-commercialization 17,030 65,000 Prepaid subscriptions fees 170,552 23,347 Prepaid rent 31,561 — Other 272,168 58,281 Total prepaid expenses and other current assets $ 1,649,781 $ 1,173,744 |
Property and Equipment, net (Ta
Property and Equipment, net (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | Property and equipment, net consists of the following as of December 31: 2018 2017 Computer equipment $ 52,704 $ 27,915 Furniture and equipment 212,451 169,931 Leasehold improvements 177,417 152,708 442,572 350,554 Less: Accumulated depreciation (197,147 ) (159,169 ) Total property and equipment, net $ 245,425 $ 191,385 |
Accrued Expenses and Other Li_2
Accrued Expenses and Other Liabilities (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Liabilities | Accrued expenses and other liabilities consist of the following as of December 31: 2018 2017 Accrued preclinical and clinical trial expenses $ 821,633 $ 970,649 Accrued professional fees 1,311,061 227,457 Accrued compensation and benefits 1,941,449 821,935 Accrued license fees 3,000,000 252,500 Deferred rent and lease incentive 33,408 24,011 Other 66,436 24,035 Current accrued expenses and other liabilities 7,173,987 2,320,587 Deferred rent and lease incentive – non-current 154,799 157,456 Non-current 154,799 157,456 Total accrued expenses and other liabilities $ 7,328,786 $ 2,478,043 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Commitments and Contingencies Disclosure [Abstract] | |
Future Minimum Lease Payments under Operating Lease Agreement | As of December 31, 2018, future minimum lease payments under the operating lease agreements are as follows: 2019 $ 329,725 2020 339,612 2021 349,792 2022 329,662 $ 1,348,791 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |
Reconciliation of Income Tax Expense (Benefit) Computed at Statutory Federal Income Tax Rate | The reconciliation of income tax expense (benefit) computed at the statutory federal income tax rate of 21% to amounts included in the statements of operations is as follows: 2018 2017 Statutory rate 21.0 % 34.0 % State tax 4.2 % 3.5 % Valuation allowance (25.9 )% 26.5 % Federal rate change 0.0 % (73.2 )% Tax credit 1.4 % 6.8 % Other (0.7 )% 2.4 % 0.0 % 0.0 % |
Components of Deferred Tax Assets | Significant components of the Company’s deferred tax assets as of December 31, 2018 and 2017 are as follows: 2018 2017 Net operating loss $ 19,867,591 $ 15,718,570 Start-up 13,861,147 10,508,487 Tax credits 12,625,275 11,582,134 Deferred compensation 1,919,434 1,326,189 Other 109,779 72,395 Gross deferred tax asset 48,383,226 39,207,775 Valuation allowance (48,383,226 ) (39,207,775 ) Net deferred tax assets $ — $ — |
Stock Compensation Plans (Table
Stock Compensation Plans (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock-Based Compensation Expense | For the years ended December 31, 2018 and 2017, the Company recorded stock-based compensation expense as follows: 2018 2017 Research and development $ 1,079,230 $ 785,899 General and administrative 2,471,414 1,622,062 Total stock-based compensation $ 3,550,644 $ 2,407,961 |
Stock option activity under the Company's Plans | Stock option activity under the Company’s Plans for the year ended December 31, 2018 is summarized as follows: Number of Weighted Weighted Aggregate Outstanding at beginning of year 5,191,666 $ 1.96 Granted 5,822,500 3.04 Exercised or released (186,665 ) 1.59 Forfeited or cancelled (270,001 ) 2.94 Expired (25,000 ) 0.47 Outstanding at end of year 10,532,500 $ 2.54 5.39 $ 2,239,500 Exercisable at end of year 4,349,996 $ 2.16 4.00 $ 1,679,297 |
Schedule of Other Information Pertaining to Stock Option Activity | Other information pertaining to stock option activity during the years ended December 31, 2018 and 2017 was as follows: 2018 2017 Weighted–average fair value of granted stock options $ 1.93 $ 0.91 Total fair value of vested stock options $ 2,193,294 $ 2,016,992 Total intrinsic value of exercised stock options $ 274,864 $ 2,296,100 |
Summary of Company's Stock Options Outstanding | The following table summarizes information about the Company’s options outstanding at December 31, 2018: Options Outstanding Options Exercisable Range of Exercise Prices Number Weighted Weighted Number Weighted Weighted $0.79 to $1.13 2,385,000 4.78 $ 0.99 1,686,664 4.69 $ 0.93 $1.14 to $2.28 2,440,000 6.87 $ 2.22 209,999 6.22 $ 2.07 $2.29 to $3.07 1,765,000 4.84 $ 2.60 1,098,333 3.72 $ 2.53 $3.08 to $3.75 2,005,000 4.59 $ 3.30 995,000 2.71 $ 3.14 $3.76 to $4.64 1,937,500 5.62 $ 4.03 360,000 3.92 $ 4.11 10,532,500 5.39 $ 2.54 4,349,996 4.00 $ 2.16 |
Summary of Stock Options Awards Based on Certain Assumptions | Assumptions used during the years were as follows: December 31, December 31, Risk free interest rate 2.09% to 2.88% 1.66% to 2.25% Expected term 0 to 7 years 4 to 7 years Expected volatility 82% 104% Expected dividend yield — % — % Expected forfeiture rate — % — % |
Summary of Restricted Stock Unit Activity | Restricted stock unit activity during 2017 was as follows: 2017 Number of Restricted Weighted Average Nonvested balance at beginning of year 26,667 $ 2.83 Granted — — Vested (26,667 ) 2.83 Forfeited — — Nonvested balance at end of year — $ — |
Quarterly Financial Informati_2
Quarterly Financial Information (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Quarterly Financial Information Disclosure [Abstract] | |
Schedule of Quarterly Financial Information | The following table presents unaudited supplemental quarterly financial information for the years ended December 31, 2018 and 2017: Quarter Ended March 31, 2018 June 30, 2018 September 30, 2018 December 31, 2018 Revenues $ — $ — $ — $ 500,000 Loss from operations $ (5,933,440 ) $ (6,335,855 ) $ (8,182,603 ) $ (14,843,267 ) Net loss $ (5,699,892 ) $ (5,965,140 ) $ (7,838,873 ) $ (14,499,609 ) Net loss per share – basic and diluted $ (0.06 ) $ (0.06 ) $ (0.08 ) $ (0.14 ) Quarter Ended March 31, 2017 June 30, 2017 September 30, 2017 December 31, 2017 Revenues $ — $ — $ — $ — Loss from operations $ (4,679,871 ) $ (4,181,271 ) $ (4,306,708 ) $ (5,511,786 ) Change in fair value of warrants liability $ (397,235 ) $ 210,331 $ — $ — Net loss $ (4,967,129 ) $ (3,879,901 ) $ (4,177,649 ) $ (5,387,698 ) Net loss per share – basic and diluted $ (0.06 ) $ (0.05 ) $ (0.05 ) $ (0.06 ) |
Basis of Presentation and Sig_4
Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) | Jan. 01, 2019USD ($) | Oct. 31, 2011shares | Dec. 31, 2018USD ($) | Dec. 31, 2018USD ($)Segment$ / shares | Dec. 31, 2017USD ($)$ / sharesshares |
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Maximum maturity period of cash and cash equivalent | Three months | ||||
Inventory | $ 56,012 | $ 56,012 | $ 0 | ||
Deferred rent and lease incentive in accrued expenses and other liabilities | 188,207 | 188,207 | 181,467 | ||
Proceeds of Warrants | 3,209,423 | ||||
Upfront license fees | $ 500,000 | $ 500,000 | $ 0 | ||
Statutory rate | 21.00% | 34.00% | |||
Stock option exercise price range, Minimum | $ / shares | $ 0.79 | $ 0.47 | |||
Stock option exercise price range, Maximum | $ / shares | $ 4.64 | $ 4.64 | |||
Number of reportable segment | Segment | 1 | ||||
Lease Expiration Date | Nov. 30, 2022 | ||||
U.S. federal corporate tax rate [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Statutory rate | 35.00% | ||||
Furniture and Equipment [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 5 years | ||||
Subsequent Event [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Additional assets | $ 1,000,000 | ||||
Corresponding liability | $ 1,500,000 | ||||
October 28, 2011 Warrants [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Warrants issued in offering | shares | 1,523,370 | ||||
October 28, 2011 Warrants [Member] | Warrants Liability, at Fair Value [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Warrants expiration date | May 2, 2017 | ||||
Number of warrants exercised during the period | shares | 613,913 | ||||
Proceeds of Warrants | $ 798,087 | ||||
Other Income, Net [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Unrealized gain(loss),trading securities | $ (29,430) | $ 29,430 | |||
Minimum [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Minimum amortization period of compensation cost on straight line basis | 1 year | ||||
Minimum [Member] | Leasehold Improvements [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 4 years | ||||
Minimum [Member] | Computer Equipment [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 3 years | ||||
Maximum [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Minimum amortization period of compensation cost on straight line basis | 5 years | ||||
Maximum [Member] | Leasehold Improvements [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 7 years | ||||
Maximum [Member] | Computer Equipment [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 5 years |
Basis of Presentation and Sig_5
Basis of Presentation and Significant Accounting Policies - Fair Value Measurement Specific to Assets or Liability (Detail) - USD ($) | Dec. 31, 2018 | Dec. 31, 2017 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments | $ 5,008,243 | $ 0 |
Short-Term Bond Fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 26,541,349 | 26,516,711 |
U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 10,380,864 | |
Investments | 5,008,243 | |
Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Money market funds | 14,462,087 | 56,820,688 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | Short-Term Bond Fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 26,541,349 | 26,516,711 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Money market funds | 14,462,087 | $ 56,820,688 |
Significant Other Observable Inputs (Level 2) [Member] | U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 10,380,864 | |
Investments | $ 5,008,243 |
Basis of Presentation and Sig_6
Basis of Presentation and Significant Accounting Policies - Summary of Company's Warrants Liability Activity (Detail) | 12 Months Ended |
Dec. 31, 2018USD ($) | |
Fair Value Disclosures [Abstract] | |
Fair value, beginning of period | $ 122,226 |
Issuance of warrants | 0 |
Exercise of warrants | (309,130) |
Change in fair value | 186,904 |
Fair value, end of period | $ 0 |
Basis of Presentation and Sig_7
Basis of Presentation and Significant Accounting Policies - Potential Shares Excluded from Determination of Basic and Diluted Net Loss Per Share (Detail) - shares | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential equivalent common stock excluded | 10,532,500 | 5,191,666 |
Options to Purchase Common Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential equivalent common stock excluded | 10,532,500 | 5,191,666 |
Investment - Summary of Availab
Investment - Summary of Available-for-Sale Investments by Security type (Detail) | 12 Months Ended |
Dec. 31, 2018USD ($) | |
Net Investment Income [Line Items] | |
Amortized cost | $ 15,409,355 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (20,248) |
Estimated Fair Value | 15,389,107 |
U.S. Treasuries - ST [Member] | |
Net Investment Income [Line Items] | |
Amortized cost | 10,382,699 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (1,835) |
Estimated Fair Value | 10,380,864 |
U.S. Treasuries - LT [Member] | |
Net Investment Income [Line Items] | |
Amortized cost | 5,026,656 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (18,413) |
Estimated Fair Value | $ 5,008,243 |
Investments - Additional Inform
Investments - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Investments, Debt and Equity Securities [Abstract] | ||
Realized gains losses from available for sale securities | $ 0 | $ 0 |
Investment - Estimated Fair Val
Investment - Estimated Fair Values of Available for Sale Securities (Detail) | Dec. 31, 2018USD ($) |
Investments, Debt and Equity Securities [Abstract] | |
Due in one year or less | $ 10,380,864 |
Due after one year but within two years | 5,008,243 |
Estimated Fair Value | $ 15,389,107 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($) | Dec. 31, 2018 | Dec. 31, 2017 |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid research fees | $ 358,209 | $ 388,977 |
Prepaid insurance | 800,261 | 638,139 |
Prepaid pre-commercialization fees | 17,030 | 65,000 |
Prepaid subscriptions fees | 170,552 | 23,347 |
Prepaid rent | 31,561 | |
Other | 272,168 | 58,281 |
Total prepaid expenses and other current assets | $ 1,649,781 | $ 1,173,744 |
Property and Equipment, Net - P
Property and Equipment, Net - Property and Equipment, Net (Detail) - USD ($) | Dec. 31, 2018 | Dec. 31, 2017 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 442,572 | $ 350,554 |
Less: Accumulated depreciation | (197,147) | (159,169) |
Total property and equipment, net | 245,425 | 191,385 |
Computer Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 52,704 | 27,915 |
Furniture and Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 212,451 | 169,931 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 177,417 | $ 152,708 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Property, Plant and Equipment [Abstract] | ||
Depreciation expenses | $ 37,978 | $ 45,689 |
Accrued Expenses and Other Li_3
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($) | Dec. 31, 2018 | Dec. 31, 2017 |
Payables and Accruals [Abstract] | ||
Accrued preclinical and clinical trial expenses | $ 821,633 | $ 970,649 |
Accrued professional fees | 1,311,061 | 227,457 |
Accrued compensation and benefits | 1,941,449 | 821,935 |
Accrued license fees | 3,000,000 | 252,500 |
Deferred rent and lease incentive | 33,408 | 24,011 |
Other | 66,436 | 24,035 |
Current accrued expenses and other liabilities | 7,173,987 | 2,320,587 |
Deferred rent and lease incentive - non-current | 154,799 | 157,456 |
Non-current accrued expenses and other liabilities | 154,799 | 157,456 |
Total accrued expenses and other liabilities | $ 7,328,786 | $ 2,478,043 |
Collaborative Arrangement - Add
Collaborative Arrangement - Additional Information (Detail) - USD ($) | 3 Months Ended | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2018 | Dec. 31, 2017 | |
Collaboration Arrangement Disclosure [Abstract] | |||
Upfront license fee | $ 500,000 | $ 500,000 | $ 0 |
Period of collaboration agreement | 10 years |
Commitments and Contingencies -
Commitments and Contingencies - Future Minimum Lease Payments under Operating Lease Agreement (Detail) | Dec. 31, 2018USD ($) |
Commitments and Contingencies Disclosure [Abstract] | |
2019 | $ 329,725 |
2020 | 339,612 |
2021 | 349,792 |
2022 | 329,662 |
Total | $ 1,348,791 |
Commitments and Contingencies_2
Commitments and Contingencies - Additional Information (Detail) | 1 Months Ended | 12 Months Ended | |
Aug. 31, 2018USD ($)ft² | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | |
Commitments [Line Items] | |||
Area of leased property | ft² | 7,800 | ||
Lease expiration date | 2022-11 | ||
Tenant build-out costs | $ 13,000 | ||
Rent expense | $ 242,155 | $ 204,170 | |
Obligations under capital leases | 0 | ||
Accrued license fees | 3,000,000 | $ 252,500 | |
CEO Employment Agreement [Member] | |||
Commitments [Line Items] | |||
Annual base salary of CEO | $ 525,000 | ||
Expiration date of employment agreement | 2020-11 | ||
Northwestern License Agreement [Member] | |||
Commitments [Line Items] | |||
License fee paid | $ 424,885 | ||
Accrued license fees | $ 0 |
Agreements - Additional Informa
Agreements - Additional Information (Detail) - License Agreement with BioMarin [Member] - USD ($) | 1 Months Ended | 12 Months Ended |
Feb. 28, 2019 | Dec. 31, 2018 | |
License Agreement [Line Items] | ||
Date on which strategic collaboration is entered into | Oct. 26, 2012 | |
Royalty agreement period | 7 years | |
Net sales royalty threshold | $ 100,000,000 | |
Third Party Licensor [Member] | ||
License Agreement [Line Items] | ||
Milestone payment due | 3,150,000 | |
Third Party Licensor [Member] | Subsequent Event [Member] | ||
License Agreement [Line Items] | ||
Milestone payment due | $ 3,000,000 | |
Huxley [Member] | ||
License Agreement [Line Items] | ||
Milestone payment due | $ 2,000,000 | |
Minimum [Member] | ||
License Agreement [Line Items] | ||
Percentage of royalty on net sales | 7.00% | |
Maximum [Member] | ||
License Agreement [Line Items] | ||
Percentage of royalty on net sales | 10.00% |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | ||
Statutory rate | 21.00% | 34.00% |
Increase (decrease) in valuation allowance on deferred tax assets | $ 9,175,451 | $ (3,388,951) |
Net operating loss carryforwards | $ 79,000,000 | $ 62,600,000 |
Net operating loss carryforwards expiration date | Beginning in 2023 and ending in 2037 | |
Effective tax rate | 0.00% | 0.00% |
Percentage of limited utilization of taxable income per year | 80.00% | |
Interest or penalties accrued | $ 0 |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of Income Tax Expense (Benefit) Computed at Statutory Federal Income Tax Rate (Detail) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | ||
Statutory rate | 21.00% | 34.00% |
State tax | 4.20% | 3.50% |
Valuation allowance | (25.90%) | 26.50% |
Federal rate change | 0.00% | (73.20%) |
Tax credit | 1.40% | 6.80% |
Other | (0.70%) | 2.40% |
Reconciliation of income tax expense (benefit) | 0.00% | 0.00% |
Income Taxes - Components of De
Income Taxes - Components of Deferred Tax Assets (Detail) - USD ($) | Dec. 31, 2018 | Dec. 31, 2017 |
Components of Deferred Tax Assets [Abstract] | ||
Net operating loss | $ 19,867,591 | $ 15,718,570 |
Start-up cost | 13,861,147 | 10,508,487 |
Tax credits | 12,625,275 | 11,582,134 |
Deferred compensation | 1,919,434 | 1,326,189 |
Other | 109,779 | 72,395 |
Gross deferred tax asset | 48,383,226 | 39,207,775 |
Valuation allowance | (48,383,226) | (39,207,775) |
Net deferred tax assets | $ 0 | $ 0 |
Stockholders' Equity (Preferred
Stockholders' Equity (Preferred Stock and Common Stock) - Additional Information (Detail) | Dec. 31, 2018Vote$ / sharesshares | Dec. 31, 2017$ / sharesshares |
Equity [Abstract] | ||
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, par value | $ / shares | $ 0.001 | $ 0.001 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, par value | $ / shares | $ 0.001 | $ 0.001 |
Common stock, shares issued | 102,739,257 | 102,549,498 |
Common stock, shares outstanding | 102,739,257 | 102,549,498 |
Number of votes entitled for each share of common stock | Vote | 1 |
Stockholders' Equity (2016 Shel
Stockholders' Equity (2016 Shelf Registration Statement) - Additional Information (Detail) | Dec. 23, 2016USD ($) |
2016 Shelf Registration Statement [Member] | |
Stockholders' Equity [Line Items] | |
Maximum dollar amount of common stock to be issued under shelf registration statement | $ 33,800,000 |
Stockholders' Equity (2017 Shel
Stockholders' Equity (2017 Shelf Registration Statement) - Additional Information (Detail) - USD ($) | Nov. 28, 2017 | Dec. 31, 2018 | Jul. 12, 2017 |
Underwritten Public Offering [Member] | |||
Stockholders' Equity [Line Items] | |||
Number of common stock sold in offering | 16,428,572 | ||
Common stock issued, price per share | $ 3.50 | ||
Gross proceeds from issuance of common stock | $ 57,500,000 | ||
Offering expenses | $ 3,700,000 | ||
2017 Shelf Registration Statement [Member] | |||
Stockholders' Equity [Line Items] | |||
Maximum dollar amount of securities to be issued under shelf registration statement | $ 150,000,000 | ||
Value of common stock available for future sale | $ 92,500,000 |
Stockholders' Equity (Warrant E
Stockholders' Equity (Warrant Exercises) - Additional Information (Detail) | 12 Months Ended |
Dec. 31, 2017USD ($)shares | |
Equity [Abstract] | |
Issuance of common stock for warrant exercises | shares | 2,257,663 |
Proceeds from exercise of warrants | $ | $ 3,209,423 |
Stockholders' Equity (Stockhold
Stockholders' Equity (Stockholder Rights Plan) - Additional Information (Detail) - $ / shares | Sep. 19, 2016 | Sep. 20, 2011 | Dec. 31, 2018 |
Series A Preferred Stock [Member] | |||
Stockholders' Equity [Line Items] | |||
Preferred share purchase right price | $ 7.80 | ||
Series A Preferred Stock [Member] | Certificate of Designation [Member] | |||
Stockholders' Equity [Line Items] | |||
Junior participating preferred stock shares issued available for issuance under rights agreement, initial | 500,000 | ||
Junior participating preferred stock shares issued available for issuance under rights agreement, amended | 1,500,000 | ||
Stockholder Rights Plan [Member] | |||
Stockholders' Equity [Line Items] | |||
Warrants exercise price | $ 7.80 | ||
Minimum percentage of outstanding stock acquired by a person or group to trigger Shareholder Rights Plan | 17.50% | ||
Stockholders Rights Plan redemption price of right | $ 0.001 | ||
Class of warrant or right expiration date | Sep. 20, 2016 | ||
Class of warrant or right expiration date, Extended | Sep. 20, 2019 |
Stock Compensation Plans - Stoc
Stock Compensation Plans - Stock-Based Compensation Expense (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | $ 3,550,644 | $ 2,407,961 |
Research and Development [Member] | ||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | 1,079,230 | 785,899 |
General and Administrative [Member] | ||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | $ 2,471,414 | $ 1,622,062 |
Stock Compensation Plans - Addi
Stock Compensation Plans - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Stock option contractual term | 4 years | |
Number of stock options exercised | 186,665 | |
Proceeds from exercise of stock options | $ 297,563 | $ 368,185 |
Common stock unit granted | 5,822,500 | 0 |
Expected dividend rate | 0.00% | 0.00% |
Employee [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Non-cash stock-based compensation expense | $ 15,000 | |
Common stock unit granted | 3,094 | |
Stock Option [Member] | Minimum [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Share based compensation, vesting period | 1 year | |
Stock option contractual term | 5 years | |
Stock Option [Member] | Maximum [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Share based compensation, vesting period | 5 years | |
Stock option contractual term | 7 years | |
Restricted Stock Units (RSUs) [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Non-cash stock-based compensation expense | $ 0 | $ 65,336 |
2014 Stock Incentive Plan [Member] | Common Stock [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock shares available for future issuance under the Plan | 0 | |
Two Thousand Eighteen Stock Incentive Plan [Member] | Common Stock [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock shares reserved for issuance under the Plan | 7,500,000 | |
Common stock shares available for future issuance under the Plan | 3,620,603 | |
Two Thousand Eighteen Stock Incentive Plan [Member] | Restricted Stock Units (RSUs) [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Share based compensation, vesting period | 4 years | |
Options to Purchase Common Stock [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of stock options exercised | 186,665 | 780,000 |
Proceeds from exercise of stock options | $ 297,563 | $ 368,185 |
Non-cash stock-based compensation expense | $ 3,535,647 | $ 2,342,625 |
Stock option granted, contractual term | 7 years | 7 years |
Common stock unit granted | 5,822,500 | 1,550,000 |
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan | $ 8,800,000 | |
Expected remaining weighted average vesting period | 2 years 6 months 29 days | |
Expected dividend rate | 0.00% | |
Options to Purchase Common Stock [Member] | Cashless Basis [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of stock options exercised | 0 | 100,000 |
Shares issued for cashless option exercise | 84,280 |
Stock Compensation Plans - Summ
Stock Compensation Plans - Summary of Stock Option Activity under the Company's Plan (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | ||
Number of options, Outstanding at beginning of year | 5,191,666 | |
Number of options, Granted | 5,822,500 | 0 |
Number of options, Exercised or released | (186,665) | |
Number of options, Forfeited or cancelled | (270,001) | |
Number of options, Expired | (25,000) | |
Number of options, Outstanding at end of year | 10,532,500 | 5,191,666 |
Number of options, Exercisable at end of year | 4,349,996 | |
Weighted average exercise price, Outstanding at beginning of year | $ 1.96 | |
Weighted average exercise price, Granted | 3.04 | |
Weighted average exercise price, Exercised or released | 1.59 | |
Weighted average exercise price, Forfeited or cancelled | 2.94 | |
Weighted average exercise price, Expired | 0.47 | |
Weighted average exercise price, Outstanding at end of year | 2.54 | $ 1.96 |
Weighted average exercise price, Exercisable at end of year | $ 2.16 | |
Weighted Average Remaining Contractual Term (Years), Outstanding at end of year | 5 years 4 months 20 days | |
Weighted Average Remaining Contractual Term (Years), Exercisable at end of year | 4 years | |
Aggregate Intrinsic value, Outstanding at end of year | $ 2,239,500 | |
Aggregate Intrinsic value, Exercisable at end of year | $ 1,679,297 |
Stock Compensation Plans - Sche
Stock Compensation Plans - Schedule of Other Information Pertaining to Stock Option Activity (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | ||
Weighted-average fair value of granted stock options | $ 1.93 | $ 0.91 |
Total fair value of vested stock options | $ 2,193,294 | $ 2,016,992 |
Total intrinsic value of exercised stock options | $ 274,864 | $ 2,296,100 |
Stock Compensation Plans - Su_2
Stock Compensation Plans - Summary of Company's Stock Options Outstanding (Detail) - $ / shares | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Range of Exercise Prices Lower Range | $ 0.79 | $ 0.47 |
Range of Exercise Prices Upper Range | $ 4.64 | $ 4.64 |
Number Outstanding, Options Outstanding | 10,532,500 | |
Weighted Average Remaining Contractual Life (Years), Options Outstanding | 5 years 4 months 20 days | |
Weighted Average Exercise Price, Options Outstanding | $ 2.54 | |
Number Exercisable Options Exercisable | 4,349,996 | |
Weighted Average Remaining Contractual Life (Years), Options Exercisable | 4 years | |
Weighted Average Exercise Price, Options Exercisable | $ 2.16 | |
$0.79 to $1.13 [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Range of Exercise Prices Lower Range | 0.79 | |
Range of Exercise Prices Upper Range | $ 1.13 | |
Number Outstanding, Options Outstanding | 2,385,000 | |
Weighted Average Remaining Contractual Life (Years), Options Outstanding | 4 years 9 months 10 days | |
Weighted Average Exercise Price, Options Outstanding | $ 0.99 | |
Number Exercisable Options Exercisable | 1,686,664 | |
Weighted Average Remaining Contractual Life (Years), Options Exercisable | 4 years 8 months 8 days | |
Weighted Average Exercise Price, Options Exercisable | $ 0.93 | |
$1.14 to $2.28 [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Range of Exercise Prices Lower Range | 1.14 | |
Range of Exercise Prices Upper Range | $ 2.28 | |
Number Outstanding, Options Outstanding | 2,440,000 | |
Weighted Average Remaining Contractual Life (Years), Options Outstanding | 6 years 10 months 13 days | |
Weighted Average Exercise Price, Options Outstanding | $ 2.22 | |
Number Exercisable Options Exercisable | 209,999 | |
Weighted Average Remaining Contractual Life (Years), Options Exercisable | 6 years 2 months 19 days | |
Weighted Average Exercise Price, Options Exercisable | $ 2.07 | |
$2.29 to $3.07 [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Range of Exercise Prices Lower Range | 2.29 | |
Range of Exercise Prices Upper Range | $ 3.07 | |
Number Outstanding, Options Outstanding | 1,765,000 | |
Weighted Average Remaining Contractual Life (Years), Options Outstanding | 4 years 10 months 2 days | |
Weighted Average Exercise Price, Options Outstanding | $ 2.60 | |
Number Exercisable Options Exercisable | 1,098,333 | |
Weighted Average Remaining Contractual Life (Years), Options Exercisable | 3 years 8 months 19 days | |
Weighted Average Exercise Price, Options Exercisable | $ 2.53 | |
$3.08 to $3.75 [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Range of Exercise Prices Lower Range | 3.08 | |
Range of Exercise Prices Upper Range | $ 3.75 | |
Number Outstanding, Options Outstanding | 2,005,000 | |
Weighted Average Remaining Contractual Life (Years), Options Outstanding | 4 years 7 months 2 days | |
Weighted Average Exercise Price, Options Outstanding | $ 3.30 | |
Number Exercisable Options Exercisable | 995,000 | |
Weighted Average Remaining Contractual Life (Years), Options Exercisable | 2 years 8 months 15 days | |
Weighted Average Exercise Price, Options Exercisable | $ 3.14 | |
$3.76 to $4.64 [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Range of Exercise Prices Lower Range | 3.76 | |
Range of Exercise Prices Upper Range | $ 4.64 | |
Number Outstanding, Options Outstanding | 1,937,500 | |
Weighted Average Remaining Contractual Life (Years), Options Outstanding | 5 years 7 months 13 days | |
Weighted Average Exercise Price, Options Outstanding | $ 4.03 | |
Number Exercisable Options Exercisable | 360,000 | |
Weighted Average Remaining Contractual Life (Years), Options Exercisable | 3 years 11 months 1 day | |
Weighted Average Exercise Price, Options Exercisable | $ 4.11 |
Stock Compensation Plans - Su_3
Stock Compensation Plans - Summary of Stock Options Awards Based on Certain Assumptions (Detail) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Risk free interest rate, Minimum | 2.09% | 1.66% |
Risk free interest rate, Maximum | 2.88% | 2.25% |
Expected volatility | 82.00% | 104.00% |
Expected dividend yield | 0.00% | 0.00% |
Expected forfeiture rate | 0.00% | 0.00% |
Minimum [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term | 0 years | 4 years |
Maximum [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term | 7 years | 7 years |
Stock Compensation Plans - Su_4
Stock Compensation Plans - Summary of Restricted Stock Unit Activity (Detail) - Restricted Stock Units (RSUs) [Member] | 12 Months Ended |
Dec. 31, 2017$ / sharesshares | |
Schedule Of Restricted Stock Activity [Line Items] | |
Nonvested balance at beginning of year | shares | 26,667 |
Granted | shares | 0 |
Vested | shares | (26,667) |
Forfeited | shares | 0 |
Nonvested Weighted Average Grant Date Fair Value balance at beginning of year | $ / shares | $ 2.83 |
Weighted Average Grant Date Fair Value, Granted | $ / shares | 0 |
Weighted Average Grant Date Fair Value, Vested | $ / shares | 2.83 |
Weighted Average Grant Date Fair Value, Forfeited | $ / shares | $ 0 |
Benefit Plan - Additional Infor
Benefit Plan - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Retirement Benefits [Abstract] | ||
Employees contribution of pre-tax annual compensation | 15.00% | |
Discretionary matching contributions of employee contributions of an employee's gross salary | 4.00% | |
Contributions | $ 123,000 | $ 84,000 |
Quarterly Financial Informati_3
Quarterly Financial Information (Unaudited) - Schedule of Quarterly Financial Information (Detail) - USD ($) | 3 Months Ended | 12 Months Ended | ||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | |
Quarterly Financial Information Disclosure [Abstract] | ||||||||||
Revenues | $ 500,000 | $ 500,000 | $ 0 | |||||||
Loss from operations | (14,843,267) | $ (8,182,603) | $ (6,335,855) | $ (5,933,440) | $ (5,511,786) | $ (4,306,708) | $ (4,181,271) | $ (4,679,871) | (35,295,165) | (18,679,636) |
Change in fair value of warrants liability | 210,331 | (397,235) | (186,904) | |||||||
Net loss | $ (14,499,609) | $ (7,838,873) | $ (5,965,140) | $ (5,699,892) | $ (5,387,698) | $ (4,177,649) | $ (3,879,901) | $ (4,967,129) | $ (34,003,514) | $ (18,412,377) |
Net loss per share - basic and diluted | $ (0.14) | $ (0.08) | $ (0.06) | $ (0.06) | $ (0.06) | $ (0.05) | $ (0.05) | $ (0.06) | $ (0.33) | $ (0.21) |