Cover Page
Cover Page - shares | 3 Months Ended | |
Mar. 31, 2020 | May 07, 2020 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2020 | |
Document Fiscal Year Focus | 2020 | |
Document Fiscal Period Focus | Q1 | |
Entity Registrant Name | CATALYST PHARMACEUTICALS, INC. | |
Entity Central Index Key | 0001369568 | |
Current Fiscal Year End Date | --12-31 | |
Entity Current Reporting Status | Yes | |
Entity Shell Company | false | |
Entity Filer Category | Accelerated Filer | |
Trading Symbol | CPRX | |
Entity Emerging Growth Company | false | |
Entity Small Business | false | |
Entity Address, State or Province | FL | |
Entity Interactive Data Current | Yes | |
Title of 12(b) Security | Common Stock | |
Security Exchange Name | NASDAQ | |
Entity Common Stock, Shares Outstanding | 103,415,365 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) | Mar. 31, 2020 | Dec. 31, 2019 |
Current Assets: | ||
Cash and cash equivalents | $ 101,750,937 | $ 89,511,710 |
Short-term investments | 5,007,050 | |
Accounts receivable, net | 6,918,563 | 10,536,997 |
Inventory | 2,211,338 | 1,956,792 |
Prepaid expenses and other current assets | 6,062,715 | 4,351,074 |
Total current assets | 116,943,553 | 111,363,623 |
Operating lease right-of-use asset | 730,284 | 793,252 |
Property and equipment, net | 196,926 | 210,467 |
Deposits | 8,888 | 8,888 |
Total assets | 117,879,651 | 112,376,230 |
Current Liabilities: | ||
Accounts payable | 1,347,577 | 4,117,447 |
Accrued expenses and other liabilities | 16,287,936 | 19,981,295 |
Total current liabilities | 17,635,513 | 24,098,742 |
Operating lease liability, net of current portion | 568,421 | 647,532 |
Total liabilities | 18,203,934 | 24,746,274 |
Commitments and contingencies | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at March 31, 2020 and December 31, 2019 | 0 | |
Common stock, $0.001 par value, 150,000,000 shares authorized; 103,408,699 shares and 103,397,033 shares issued and outstanding at March 31, 2020 and December 31, 2019, respectively | 103,409 | 103,397 |
Additional paid-in capital | 217,751,166 | 216,205,678 |
Accumulated deficit | (118,262,609) | (128,688,624) |
Accumulated other comprehensive income (loss) | 83,751 | 9,505 |
Total stockholders' equity | 99,675,717 | 87,629,956 |
Total liabilities and stockholders' equity | $ 117,879,651 | $ 112,376,230 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2020 | Dec. 31, 2019 |
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 | 103,408,699 | 103,397,033 |
Common stock, shares outstanding | 103,408,699 | 103,397,033 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) (unaudited) - USD ($) | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Revenues: | ||
Product revenue, net | $ 29,136,472 | $ 12,448,438 |
Revenue, Product and Service [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember |
Operating costs and expenses: | ||
Cost of sales | $ 4,150,866 | $ 1,711,788 |
Cost, Product and Service [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember |
Research and development | $ 4,222,811 | $ 3,307,959 |
Selling, general and administrative | 10,063,048 | 8,416,460 |
Total operating costs and expenses | 18,436,725 | 13,436,207 |
Operating income (loss) | 10,699,747 | (987,769) |
Other income, net | 336,233 | 343,266 |
Net income (loss) before income taxes | 11,035,980 | (644,503) |
Provision for income taxes | 609,965 | |
Net income (loss) | $ 10,426,015 | $ (644,503) |
Net income (loss) per share: | ||
Basic | $ 0.10 | $ (0.01) |
Diluted | $ 0.10 | $ (0.01) |
Weighted average shares outstanding: | ||
Basic | 103,407,347 | 102,747,923 |
Diluted | 106,534,600 | 102,747,923 |
Net income (loss) | $ 10,426,015 | $ (644,503) |
Other comprehensive income (loss): | ||
Unrealized gain (loss) on available-for-sale securities | 74,246 | 13,560 |
Comprehensive income (loss) | $ 10,500,261 | $ (630,943) |
CONSOLIDATED STATEMENT OF CHANG
CONSOLIDATED STATEMENT OF CHANGES IN STOCKHOLDERS' EQUITY (unaudited) - USD ($) | Total | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Deficit [Member] | Accumulated Other Comprehensive Gain (Loss) [Member] |
Beginning Balance at Dec. 31, 2018 | $ 50,783,809 | $ 102,739 | $ 211,265,279 | $ (160,563,961) | $ (20,248) |
Beginning Balance (shares) at Dec. 31, 2018 | 102,739,257 | ||||
Issuance of stock options for services | 933,411 | 933,411 | |||
Exercise of stock options for common stock | 89,350 | $ 65 | 89,285 | ||
Exercise of stock options for common stock (shares) | 65,000 | ||||
Other comprehensive gain (loss) | 13,560 | 13,560 | |||
Net income (loss) | (644,503) | (644,503) | |||
Ending Balance at Mar. 31, 2019 | 51,175,627 | $ 102,804 | 212,287,975 | (161,208,464) | (6,688) |
Ending Balance (shares) at Mar. 31, 2019 | 102,804,257 | ||||
Beginning Balance at Dec. 31, 2019 | 87,629,956 | $ 103,397 | 216,205,678 | (128,688,624) | 9,505 |
Beginning Balance (shares) at Dec. 31, 2019 | 103,397,033 | ||||
Issuance of stock options for services | 1,383,672 | 1,383,672 | |||
Exercise of stock options for common stock | 26,149 | $ 12 | 26,137 | ||
Exercise of stock options for common stock (shares) | 11,666 | ||||
Amortization of restricted stock for services | 135,679 | 135,679 | |||
Other comprehensive gain (loss) | 74,246 | 74,246 | |||
Net income (loss) | 10,426,015 | 10,426,015 | |||
Ending Balance at Mar. 31, 2020 | $ 99,675,717 | $ 103,409 | $ 217,751,166 | $ (118,262,609) | $ 83,751 |
Ending Balance (shares) at Mar. 31, 2020 | 103,408,699 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited) - USD ($) | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Operating Activities: | ||
Net income (loss) | $ 10,426,015 | $ (644,503) |
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: | ||
Depreciation | 13,541 | 13,748 |
Amortization of right-of-use asset | 62,968 | 59,621 |
Stock-based compensation | 1,519,351 | 933,411 |
Change in accrued interest and accretion of discount on investments | 81,296 | (53,674) |
(Increase) decrease in: | ||
Accounts receivable, net | 3,618,434 | (7,251,381) |
Inventory | (254,546) | (40,575) |
Prepaid expenses and other current assets and deposits | (1,711,641) | (231,485) |
Increase (decrease) in: | ||
Accounts payable | (2,769,870) | 602,719 |
Accrued expenses and other liabilities | (3,699,518) | (1,337,155) |
Operating lease liability | (72,952) | (67,155) |
Net cash provided by (used in) operating activities | 7,213,078 | (8,016,429) |
Investing Activities: | ||
Purchases of property and equipment | (5,633) | |
Purchases of investments | (9,944,974) | |
Proceeds from maturities and sales of investments | 5,000,000 | 20,400,000 |
Net cash provided by (used in) investing activities | 5,000,000 | 10,449,393 |
Financing Activities: | ||
Proceeds from exercise of stock options | 26,149 | 89,350 |
Net cash provided by (used in) financing activities | 26,149 | 89,350 |
Net increase (decrease) in cash and cash equivalents | 12,239,227 | 2,522,314 |
Cash and cash equivalents—beginning of period | 89,511,710 | 16,559,400 |
Cash and cash equivalents—end of period | 101,750,937 | 19,081,714 |
Non-cash investing and financing activities: | ||
Unrealized gain (loss) on available-for-sale securities | $ 74,246 | $ 13,560 |
Organization and Description of
Organization and Description of Business | 3 Months Ended |
Mar. 31, 2020 | |
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 biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), Anti-MuSK antibody positive myasthenia gravis (MuSK-MG), 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, raising capital, and selling its product. The Company incurred operating losses in each period from inception, and started reporting operating income during the year ended December 31, 2019. The Company has been able to fund its cash needs to date through several public and private offerings of its securities and from revenues from its product sales. See Note 11 (Stockholders’ Equity). Capital Resources While there can be no assurance, based on currently available information, the Company estimates that it currently has sufficient resources to support its operations for at least the next 12 months from the issuance date of this Form 10-Q. The Company may raise required funds in the future through public or private equity offerings, debt financings, corporate collaborations, governmental research grants or other means. The Company may also seek to raise new capital to fund additional product development efforts, even if it has sufficient funds for its planned operations. Any sale by the Company of additional equity or convertible debt securities could result in dilution to the Company’s current stockholders. There can be no assurance that any required additional funding will be available to the Company at all or available on terms acceptable to the Company. Further, to the extent that the Company raises additional funds through collaborative arrangements, it may be necessary to relinquish some rights to the Company’s drug candidates or grant sublicenses on terms that are not favorable to the Company. If the Company is not able to secure additional funding when needed, the Company may have to delay, reduce the scope of, or eliminate one or more research and development programs, which could have an adverse effect on the Company’s business. Risks and Uncertainties There are many uncertainties regarding the novel coronavirus (COVID-19) COVID-19 COVID-19 |
Basis of Presentation and Signi
Basis of Presentation and Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2020 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Significant Accounting Policies | 2. Basis of Presentation and Significant Accounting Policies. a. INTERIM FINANCIAL STATEMENTS. 10-Q In the opinion of management, the accompanying unaudited interim consolidated financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these consolidated statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2019 included in the 2019 Annual Report on Form 10-K b. PRINCIPLES OF CONSOLIDATION c. USE OF ESTIMATES. d. CASH AND CASH EQUIVALENTS. e. INVESTMENTS Short-Term Bond Fund The Company previously owned a short-term bond fund t hat as 20 9 , as the Company sold its interest in the short-term bond fund in 2019. There were no sales o f 2020. Realized losses on trading securities during the three months ended March 31, 2019 were $4,980 . Unrealized gain on trading securities was $0 and $52,741, respectively, for the three months ended March 31, 2020 and March 31, 2019, 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 its available-for-sale available-for-sale available-for-sale f. ACCOUNTS RECEIVABLE, NET. is and December 31, 2019 , the Company determined that an allowance for expected credit loss was not required. No accounts were written off during the periods presented. g. INVENTORY. first-in-first-out ® ® o work-in-process 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. h. PREPAID EXPENSES AND OTHER CURRENT ASSETS. pre-clinical 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 $ 23,610,888 $ 23,610,888 $ — $ — U.S. Treasuries $ 69,997,200 $ — $ 69,997,200 $ — Balances as of Quoted Prices in Significant (Level 2) Significant (Level 3) Cash and cash equivalents: Money market funds $ 23,963,617 $ 23,963,617 $ — $ — U.S. Treasuries $ 59,932,200 $ — $ 59,932,200 $ — Short-term investments: U.S. Treasuries $ 5,007,050 $ — $ 5,007,050 $ — k. OPERATING LEASES. right-of-use non-lease l. REVENUE RECOGNITION. to ® ® ® ® To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to arrangements that meet the definition of a contract under Topic 606, including when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. For a complete discussion of accounting for product revenue, see Product Revenue, Net below. The Company also may generate revenues from payments received under a collaborative agreement. Collaborative agreement payments may include nonrefundable fees at the inception of the agreements, milestone and event-based payments for specific achievements designated in the collaborative agreements, and/or royalties on sales of products resulting from a collaborative arrangement. For a complete discussion of accounting for collaborative arrangements, see Revenues from Collaborative Arrangement below. Product Revenue ® ® The Company recognizes revenue on product sales when the Customer obtains control of the Company’s product, which occurs at a point in time (upon delivery). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 15 and 30 days. Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods, and are recorded in cost of sales. If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred, if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the three months ended March 31, 2020. During the three months ended March 31, 2020 and 2019 Reserves for Variable Consideration: These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the respective underlying contracts. The amount of variable consideration which is included in the transaction price may be constrained, and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplated application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of March 31, 2020 and, therefore, the transaction price was not reduced further during the three months ended March 31, 2020. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. Trade Discounts and Allowances: ® Funded Co-pay co-pay co-pay ® Product Returns: Provider Chargebacks and Discounts: Government Rebates: Bridge and Patient Assistance Programs: ® pre-established ® ® ® ® 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. The collaborative agreement provides for a milestone payment upon achievement of development and regulatory events. The Company accounts for milestone payments in accordance with the provisions of Accounting Standards Update (ASU) No. 2010-17, 1. The consideration is commensurate with either the entity’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone; 2. The consideration relates solely to past performance; and 3. The consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. A milestone is defined as an event (i) that can only be achieved based in whole or in part on either the entity’s performance or on the occurrence of a specific outcome resulting from the entity’s performance, (ii) for which there is substantive uncertainty at the date the arrangement is entered into that the event will be achieved, and (iii) that would result in additional payments being due to the vendor. The Company believes that achievement of the milestone will be substantive and there will be no substantive uncertainty once the milestone is achieved. Since the Company will receive royalty reports 60 days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter following the quarter in which the corresponding sales occurred. As of March 31, 2020 and 2019, there was no royalty revenue from sales of the collaborative product. Refer to Note 7 (Collaborative Arrangement), for further discussion on the Company’s collaborative arrangement. m. RESEARCH AND DEVELOPMENT. n. STOCK-BASED COMPENSATION. o. CONCENTRATION OF RISK. The Company sells its product in the United States through an exclusive distributor (its Customer) to specialty pharmacies. Therefore, its distributor and specialty pharmacies account for all of its trade receivables and net product revenues. The creditworthiness of its Customer is continuously monitored, and the Company has internal policies regarding customer credit limits. The Company estimates an allowance for expected credit loss primarily based on the credit worthiness of its Customer, historical payment patterns, aging of receivable balances and general economic conditions. The Company currently has a single product with limited commercial sales experience, which makes it difficult to evaluate its current business, predict its future prospects and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, Firdapse ® ® ® The Company relies exclusively on third parties to formulate and manufacture Firdapse ® ® ® p. ROYALTIES. q. 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 March 27, 2020, the Coronavirus Aid, Relief, and Economic Security (CARES) Act was signed into law making several changes to the Internal Revenue Code. The changes include, but are not limited to: increasing the limitation on the amount of deductible interest expense, allowing companies to carryback certain net operating losses, and increasing the amount of net operating loss carryforwards that corporations can use to offset taxable income. The tax law changes in the CARES r. COMPREHENSIVE INCOME (LOSS). available-for-sale s. NET INCOME (LOSS) PER COMMON SHARE. Diluted net income (loss) per common share is computed by dividing net income (loss) by the weighted average number of common shares outstanding, increased by the assumed conversion of other potentially dilutive securities during the period. The following table reconciles basic and diluted weighted average common shares: For the Three Months Ended 2020 2019 Basic weighted average common shares outstanding 103,407,347 102,747,923 Effect of dilutive securities: Unvested restricted stock units subject to vesting requirements issued to employees and non-employees 352,500 — Common stock issuable upon the exercise of stock options 2,774,753 — Diluted weighted average common shares outstanding 106,534,600 102,747,923 Outstanding common stock equivalents totaling approximately 5.4 million, were excluded from the calculation of diluted net income (loss) per common share for the three months ended March 31, 2020 as their effect would be anti-dilutive. For the three months ended March 31, 2019, approximately 10.6 million shares of outstanding stock options were excluded from the calculation of diluted net loss per common share because a net loss was reported in this period and therefore their effect was anti-dilutive. t. RECLASSIFICATIONS. u. RECENTLY ISSUED ACCOUNTING STANDARDS . 2018-18, 2014-09 2018-18 Ventures Limited (Endo). See Note 7 (Collaborative Arrangement). In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments. available-for-sale In August 2018, the FASB issued ASU 2018-15, Intangibles – Goodwill and Other – Internal-Use 350-40), u internal-use internal-use u 350-40 |
Investments
Investments | 3 Months Ended |
Mar. 31, 2020 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | 3. Investments. Available-for-sale Estimated Gross Gross Amortized At March 31, 2020: U.S. Treasuries – Cash equivalents $ 69,997,200 $ 83,751 $ — $ 69,913,449 At December 31, 2019: U.S. Treasuries – Cash equivalents $ 59,932,200 $ 2,042 $ — $ 59,930,158 U.S. Treasuries – ST 5,007,050 7,463 — 4,999,587 Total $ 64,939,250 $ 9,505 $ — $ 64,929,745 There were no realized gains or losses from available-for-sale March The Company did not hold any securities in an unrealized position for more than 12 months as of March 31, 2020. The estimated fair values of available-for-sale March 31, 2020 Due in one year or less $ 69,997,200 |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 3 Months Ended |
Mar. 31, 2020 | |
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: March 31, 2020 December 31, 2019 Prepaid manufacturing costs $ 3,471,078 $ 1,526,013 Prepaid insurance 995,146 1,263,129 Prepaid subscription fees 492,716 501,251 Prepaid research fees 490,102 481,057 Prepaid commercialization expenses 287,083 62,959 Other 326,590 516,665 Total prepaid expenses and other current assets $ 6,062,715 $ 4,351,074 |
Operating Leases
Operating Leases | 3 Months Ended |
Mar. 31, 2020 | |
Disclosure of Operating Leases [Abstract] | |
Operating Leases | 5. Operating Leases. The Company has operating lease agreements for its corporate office. The leases include options to extend the leases for up to 1 year and options to terminate the lease within 1 year. There are no obligations under finance leases. The components of lease expense were as follows: March 31, 2020 Operating lease cost $ 74,079 Supplemental cash flow information related to leases was as follows: March 31, 2020 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows $ 84,061 Right-of-use Operating leases $ 5,305 Supplemental balance sheet information related to leases was as follows: March 31, 2020 Operating lease right-of-use $ 730,284 Other current liabilities $ 306,677 Operating lease liabilities, net of current portion 568,421 Total operating lease liabilities $ 875,098 Weighted average remaining lease term 2.7 years Weighted average discount rate 4.81 % Remaining payments of lease liabilities as of March 31, 2020 were as follows: 2020 (remaining nine months) $ 255,544 2021 349,788 2022 329,662 Total lease payments 934,994 Less imputed interest (59,896 ) Total $ 875,098 |
Accrued Expenses and Other Liab
Accrued Expenses and Other Liabilities | 3 Months Ended |
Mar. 31, 2020 | |
Payables and Accruals [Abstract] | |
Accrued Expenses and Other Liabilities | 6. Accrued Expenses and Other Liabilities. Accrued expenses and other liabilities consist of the following: March 31, 2020 December 31, 2019 Accrued preclinical and clinical trial expenses $ 940,963 $ 1,183,513 Accrued professional fees 3,309,351 1,241,526 Accrued compensation and benefits 1,814,396 3,064,645 Accrued license fees 3,945,991 8,751,991 Accrued purchases 1,243,991 1,313,310 Accrued contributions 1,135,000 1,535,000 Operating lease liability 306,677 300,518 Accrued variable consideration 1,308,344 884,764 Income tax payable 2,143,490 1,533,696 Other 139,733 172,332 Current accrued expenses and other liabilities 16,287,936 19,981,295 Lease liability— non-current 568,421 647,532 Non-current 568,421 647,532 Total accrued expenses and other liabilities $ 16,856,357 $ 20,628,827 |
Collaborative Arrangement
Collaborative Arrangement | 3 Months Ended |
Mar. 31, 2020 | |
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 (Collaboration) with Endo, for the further development and commercialization of generic Sabril ® Under the Collaboration, Endo assumes all development, manufacturing, clinical, regulatory, sales and marketing costs under the collaboration, while the Company is responsible for exercising commercially reasonable efforts to develop, or cause the development of, a final finished, stable dosage form of generic Sabril ® Under the terms of the Collaboration, the Company has received an up-front mid-double ® The collaborative agreement provides for a $2.0 million milestone payment on the commercial launch of the product by Par. As of March 31, 2020 and 2019, no milestone payments have been earned. There were no revenues from collaborative arrangement for the three months ended March 31, 2020 and 2019. Total expenses incurred, net, in connection with the collaborati ve approximat e |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2020 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 8. Commitments and Contingencies. In 2018, the Company became aware that certain patents granted to Northwestern University (which patents have been licensed by Northwestern to a third party) for a new GABA aminotransferase inhibitor were developed from CPP-115, CPP-115 In May 2019, the FDA approved an NDA for Jacobus Pharmaceuticals for Ruzurgi ® (3,4-DAP), ® ® ® ® ® off-label The Company believes that the FDA’s approval of Ruzurgi ® ® ® Additionally, from time to time the Company may become involved in legal proceedings arising in the ordinary course of business. Except as set forth above, the Company believes that there is no other litigation pending at this time that could have, individually or in the aggregate, a material adverse effect on its results of operations, financial condition or cash flows. |
Agreements
Agreements | 3 Months Ended |
Mar. 31, 2020 | |
Text Block [Abstract] | |
Agreements | 9. Agreements. a. LICENSE AGREEMENT WITH BIOMARIN (FIRDAPSE ® ) ® ® ® On May 29, 2019, the Company entered into an amendment to its license agreement for Firdapse ® ® During January 2020, the Company was advised that BioMarin has transferred certain rights under the license agreement to SERB S.A. b. AGREEMENTS FOR DRUG MANUFACTURING, DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES. commercial drug and |
Income Taxes
Income Taxes | 3 Months Ended |
Mar. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 10. Income Taxes. The Company is subject to income taxes in the U.S. federal jurisdiction and various states jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for any years before 2016. If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense. The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. As of March 31, 2020 and December 31, 2019, based on the Company’s history of operating losses, the Company has concluded that it is more likely than not that the benefit of its deferred tax assets will not be realized. Accordingly, the Company has provided a full valuation allowance for deferred tax assets including NOL and tax credit carryovers as of March 31, 2020 and December 31, 2019. |
Stockholders' Equity
Stockholders' Equity | 3 Months Ended |
Mar. 31, 2020 | |
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 March 31, 2020 and December 31, 2019. No shares of preferred stock were outstanding at March 31, 20 2 Common Stock The Company has 150,000,000 shares of authorized common stock, par value $0.001 per share. At March 31, 2020 and December 31, 2019, 103,408,699 and 103,397,033 shares, respectively, of common stock 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. 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 March 31, 2020, there is approximately $92.5 million available for future sale under the 2017 Shelf Registration Statement. |
Stock Compensation
Stock Compensation | 3 Months Ended |
Mar. 31, 2020 | |
Share-based Payment Arrangement [Abstract] | |
Stock Compensation | 12. Stock Compensation. For the three-month periods ended March 31, 2020 and 2019, the Company recorded stock-based compensation expense as follo w Three months ended March 31, 2020 2019 Research and development $ 418,053 $ 287,721 Selling, general and administrative 1,101,298 645,690 Total stock-based compensation $ 1,519,351 $ 933,411 Stock Options As of March 31, 2020, there were outstanding stock options to purchase 12,201,668 shares of common stock, of which stock options to purchase 6,687,980 shares of common stock were exercisable as of March 31, 2020. During the three-month periods ended March 31, 2020 and 2019, the Company granted seven-year term options to purchase an aggregate of 735,000 and 207,000 shares, respectively, of the Company’s common stock to employees and directors. The Company recorded stock-based compensation related to stock options totaling $1,383,672 and $933,411, respectively, during the three-month periods ended March 31, 2020 and 2019. During the three-month periods ended March 31, 2020 and 2019, respectively, 854,831 and 984,164 options vested. During the three-month periods ended March 31, 2020 and 2019, options to purchase 11,666 shares and 65,000 shares, respectively, of the Company’s common stock were exercised, with proceeds of $26,149 and $89,350 respectively, to the Company. As of March 31, 2020, there was approximately $10.4 million of unrecognized compensation expense related to non-vested Restricted Stock Units There were no grants of restricted stock units to employees or directors during the three-month periods ended March 31, 2020 and March 31, 2019. During the three-month period ended March 31, 2020, the Company recorded non-cash As of March 31, 2020, there was approximately $1.5 million of unrecognized compensation expense related to non-vested |
Subsequent Events
Subsequent Events | 3 Months Ended |
Mar. 31, 2020 | |
Subsequent Events [Abstract] | |
Subsequent Events | 13. Subsequent Events. On May 7, 2020, the Company amended the lease for its . Under the amended lease, will increase |
Basis of Presentation and Sig_2
Basis of Presentation and Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2020 | |
Accounting Policies [Abstract] | |
INTERIM FINANCIAL STATEMENTS | a. INTERIM FINANCIAL STATEMENTS. 10-Q In the opinion of management, the accompanying unaudited interim consolidated financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these consolidated statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2019 included in the 2019 Annual Report on Form 10-K |
PRINCIPLES OF CONSOLIDATION. | b. PRINCIPLES OF CONSOLIDATION |
USE OF ESTIMATES | c. USE OF ESTIMATES. |
CASH AND CASH EQUIVALENTS | d. CASH AND CASH EQUIVALENTS. |
INVESTMENTS | e. INVESTMENTS Short-Term Bond Fund The Company previously owned a short-term bond fund t hat as 20 9 , as the Company sold its interest in the short-term bond fund in 2019. There were no sales o f 2020. Realized losses on trading securities during the three months ended March 31, 2019 were $4,980 . Unrealized gain on trading securities was $0 and $52,741, respectively, for the three months ended March 31, 2020 and March 31, 2019, 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 its available-for-sale available-for-sale available-for-sale |
ACCOUNTS RECEIVABLE, NET | f. ACCOUNTS RECEIVABLE, NET. is and December 31, 2019 , the Company determined that an allowance for expected credit loss was not required. No accounts were written off during the periods presented. |
INVENTORY | g. INVENTORY. first-in-first-out ® ® o work-in-process 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 | h. PREPAID EXPENSES AND OTHER CURRENT ASSETS. pre-clinical |
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 $ 23,610,888 $ 23,610,888 $ — $ — U.S. Treasuries $ 69,997,200 $ — $ 69,997,200 $ — Balances as of Quoted Prices in Significant (Level 2) Significant (Level 3) Cash and cash equivalents: Money market funds $ 23,963,617 $ 23,963,617 $ — $ — U.S. Treasuries $ 59,932,200 $ — $ 59,932,200 $ — Short-term investments: U.S. Treasuries $ 5,007,050 $ — $ 5,007,050 $ — |
OPERATING LEASES | k. OPERATING LEASES. right-of-use non-lease |
REVENUE RECOGNITION | l. REVENUE RECOGNITION. to ® ® ® ® To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to arrangements that meet the definition of a contract under Topic 606, including when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. For a complete discussion of accounting for product revenue, see Product Revenue, Net below. The Company also may generate revenues from payments received under a collaborative agreement. Collaborative agreement payments may include nonrefundable fees at the inception of the agreements, milestone and event-based payments for specific achievements designated in the collaborative agreements, and/or royalties on sales of products resulting from a collaborative arrangement. For a complete discussion of accounting for collaborative arrangements, see Revenues from Collaborative Arrangement below. Product Revenue ® ® The Company recognizes revenue on product sales when the Customer obtains control of the Company’s product, which occurs at a point in time (upon delivery). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 15 and 30 days. Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods, and are recorded in cost of sales. If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred, if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the three months ended March 31, 2020. During the three months ended March 31, 2020 and 2019 Reserves for Variable Consideration: These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the respective underlying contracts. The amount of variable consideration which is included in the transaction price may be constrained, and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplated application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of March 31, 2020 and, therefore, the transaction price was not reduced further during the three months ended March 31, 2020. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. Trade Discounts and Allowances: ® Funded Co-pay co-pay co-pay ® Product Returns: Provider Chargebacks and Discounts: Government Rebates: Bridge and Patient Assistance Programs: ® pre-established ® ® ® ® 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. The collaborative agreement provides for a milestone payment upon achievement of development and regulatory events. The Company accounts for milestone payments in accordance with the provisions of Accounting Standards Update (ASU) No. 2010-17, 1. The consideration is commensurate with either the entity’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone; 2. The consideration relates solely to past performance; and 3. The consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. A milestone is defined as an event (i) that can only be achieved based in whole or in part on either the entity’s performance or on the occurrence of a specific outcome resulting from the entity’s performance, (ii) for which there is substantive uncertainty at the date the arrangement is entered into that the event will be achieved, and (iii) that would result in additional payments being due to the vendor. The Company believes that achievement of the milestone will be substantive and there will be no substantive uncertainty once the milestone is achieved. Since the Company will receive royalty reports 60 days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter following the quarter in which the corresponding sales occurred. As of March 31, 2020 and 2019, there was no royalty revenue from sales of the collaborative product. Refer to Note 7 (Collaborative Arrangement), for further discussion on the Company’s collaborative arrangement. |
RESEARCH AND DEVELOPMENT | m. RESEARCH AND DEVELOPMENT. |
STOCK-BASED COMPENSATION | n. STOCK-BASED COMPENSATION. |
CONCENTRATION OF CREDIT RISK | o. CONCENTRATION OF RISK. The Company sells its product in the United States through an exclusive distributor (its Customer) to specialty pharmacies. Therefore, its distributor and specialty pharmacies account for all of its trade receivables and net product revenues. The creditworthiness of its Customer is continuously monitored, and the Company has internal policies regarding customer credit limits. The Company estimates an allowance for expected credit loss primarily based on the credit worthiness of its Customer, historical payment patterns, aging of receivable balances and general economic conditions. The Company currently has a single product with limited commercial sales experience, which makes it difficult to evaluate its current business, predict its future prospects and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, Firdapse ® ® ® The Company relies exclusively on third parties to formulate and manufacture Firdapse ® ® ® |
ROYALTIES | p. ROYALTIES. |
INCOME TAXES | q. 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 March 27, 2020, the Coronavirus Aid, Relief, and Economic Security (CARES) Act was signed into law making several changes to the Internal Revenue Code. The changes include, but are not limited to: increasing the limitation on the amount of deductible interest expense, allowing companies to carryback certain net operating losses, and increasing the amount of net operating loss carryforwards that corporations can use to offset taxable income. The tax law changes in the Act did not have a material impact on the Company’s income tax provision. |
COMPREHENSIVE INCOME (LOSS) | r. COMPREHENSIVE INCOME (LOSS). available-for-sale |
NET INCOME (LOSS) PER COMMON SHARE | s. NET INCOME (LOSS) PER COMMON SHARE. Diluted net income (loss) per common share is computed by dividing net income (loss) by the weighted average number of common shares outstanding, increased by the assumed conversion of other potentially dilutive securities during the period. The following table reconciles basic and diluted weighted average common shares: For the Three Months Ended 2020 2019 Basic weighted average common shares outstanding 103,407,347 102,747,923 Effect of dilutive securities: Unvested restricted stock units subject to vesting requirements issued to employees and non-employees 352,500 — Common stock issuable upon the exercise of stock options 2,774,753 — Diluted weighted average common shares outstanding 106,534,600 102,747,923 Outstanding common stock equivalents totaling approximately 5.4 million, were excluded from the calculation of diluted net income (loss) per common share for the three months ended March 31, 2020 as their effect would be anti-dilutive. For the three months ended March 31, 2019, approximately 10.6 million shares of outstanding stock options were excluded from the calculation of diluted net loss per common share because a net loss was reported in this period and therefore their effect was anti-dilutive. |
RECLASSIFICATIONS | t. RECLASSIFICATIONS. |
RECENTLY ISSUED ACCOUNTING STANDARDS | u. RECENTLY ISSUED ACCOUNTING STANDARDS . 2018-18, 2014-09 2018-18 In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments. available-for-sale In August 2018, the FASB issued ASU 2018-15, Intangibles – Goodwill and Other – Internal-Use 350-40), u internal-use internal-use u 350-40 |
Basis of Presentation and Sig_3
Basis of Presentation and Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Accounting Policies [Abstract] | |
Fair Value Measurement Specific to Assets 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 $ 23,610,888 $ 23,610,888 $ — $ — U.S. Treasuries $ 69,997,200 $ — $ 69,997,200 $ — Balances as of Quoted Prices in Significant (Level 2) Significant (Level 3) Cash and cash equivalents: Money market funds $ 23,963,617 $ 23,963,617 $ — $ — U.S. Treasuries $ 59,932,200 $ — $ 59,932,200 $ — Short-term investments: U.S. Treasuries $ 5,007,050 $ — $ 5,007,050 $ — |
Basic and Dilutive Weighted Average Common Shares | The following table reconciles basic and diluted weighted average common shares: For the Three Months Ended 2020 2019 Basic weighted average common shares outstanding 103,407,347 102,747,923 Effect of dilutive securities: Unvested restricted stock units subject to vesting requirements issued to employees and non-employees 352,500 — Common stock issuable upon the exercise of stock options 2,774,753 — Diluted weighted average common shares outstanding 106,534,600 102,747,923 |
Investments (Tables)
Investments (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available-for-Sale Investments by Security type | Available-for-sale Estimated Gross Gross Amortized At March 31, 2020: U.S. Treasuries – Cash equivalents $ 69,997,200 $ 83,751 $ — $ 69,913,449 At December 31, 2019: U.S. Treasuries – Cash equivalents $ 59,932,200 $ 2,042 $ — $ 59,930,158 U.S. Treasuries – ST 5,007,050 7,463 — 4,999,587 Total $ 64,939,250 $ 9,505 $ — $ 64,929,745 |
Estimated Fair Values of Available for Sale Securities | The estimated fair values of available-for-sale March 31, 2020 Due in one year or less $ 69,997,200 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Text Block [Abstract] | |
Prepaid expenses and other current assets | Prepaid expenses and other current assets consist of the following: March 31, 2020 December 31, 2019 Prepaid manufacturing costs $ 3,471,078 $ 1,526,013 Prepaid insurance 995,146 1,263,129 Prepaid subscription fees 492,716 501,251 Prepaid research fees 490,102 481,057 Prepaid commercialization expenses 287,083 62,959 Other 326,590 516,665 Total prepaid expenses and other current assets $ 6,062,715 $ 4,351,074 |
Operating Leases (Tables)
Operating Leases (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Disclosure of Operating Leases [Abstract] | |
Lease, Cost | The components of lease expense were as follows: March 31, 2020 Operating lease cost $ 74,079 |
Schedule of Supplemental Cash Flow Information Related To Lease | Supplemental cash flow information related to leases was as follows: March 31, 2020 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows $ 84,061 Right-of-use Operating leases $ 5,305 |
Schedule of Supplemental Balance Sheet related To Lease | Supplemental balance sheet information related to leases was as follows: March 31, 2020 Operating lease right-of-use $ 730,284 Other current liabilities $ 306,677 Operating lease liabilities, net of current portion 568,421 Total operating lease liabilities $ 875,098 Weighted average remaining lease term 2.7 years Weighted average discount rate 4.81 % |
Lessee, Operating Lease, Liability, Maturity | Remaining payments of lease liabilities as of March 31, 2020 were as follows: 2020 (remaining nine months) $ 255,544 2021 349,788 2022 329,662 Total lease payments 934,994 Less imputed interest (59,896 ) Total $ 875,098 |
Accrued Expenses and Other Li_2
Accrued Expenses and Other Liabilities (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Liabilities | Accrued expenses and other liabilities consist of the following: March 31, 2020 December 31, 2019 Accrued preclinical and clinical trial expenses $ 940,963 $ 1,183,513 Accrued professional fees 3,309,351 1,241,526 Accrued compensation and benefits 1,814,396 3,064,645 Accrued license fees 3,945,991 8,751,991 Accrued purchases 1,243,991 1,313,310 Accrued contributions 1,135,000 1,535,000 Operating lease liability 306,677 300,518 Accrued variable consideration 1,308,344 884,764 Income tax payable 2,143,490 1,533,696 Other 139,733 172,332 Current accrued expenses and other liabilities 16,287,936 19,981,295 Lease liability— non-current 568,421 647,532 Non-current 568,421 647,532 Total accrued expenses and other liabilities $ 16,856,357 $ 20,628,827 |
Stock Compensation (Tables)
Stock Compensation (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Share-based Payment Arrangement [Abstract] | |
Stock-Based Compensation Expense | For the three-month periods ended March 31, 2020 and 2019, the Company recorded stock-based compensation expense as follo w Three months ended March 31, 2020 2019 Research and development $ 418,053 $ 287,721 Selling, general and administrative 1,101,298 645,690 Total stock-based compensation $ 1,519,351 $ 933,411 |
Basis of Presentation and Sig_4
Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($) shares in Millions | 3 Months Ended | ||
Mar. 31, 2020 | Mar. 31, 2019 | Dec. 31, 2018 | |
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Maximum maturity period of cash and cash equivalent | three months | ||
Trading Securities, Realized Gain (Loss) | $ 4,980 | ||
Trading Securities Sales | $ 0 | ||
Potential equivalent common stock excluded | 5.4 | 10.6 | |
Trading securities | $ 0 | $ 0 | |
Other Income, Net [Member] | |||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Unrealized gain (loss), trading securities | $ 0 | $ 52,741 | |
Minimum [Member] | |||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Vesting period for stock options | 1 year | ||
Maximum [Member] | |||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Vesting period for stock options | 3 years |
Basis of Presentation and Sig_5
Basis of Presentation and Significant Accounting Policies - Fair Value Measurement Specific to Assets or Liability (Detail) - USD ($) | Mar. 31, 2020 | Dec. 31, 2019 |
Short-Term Bond Fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | $ 5,007,050 | |
U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 69,997,200 | 59,932,200 |
Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 23,610,888 | 23,963,617 |
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] | ||
Cash and cash equivalents | 23,610,888 | 23,963,617 |
Significant Other Observable Inputs (Level 2) [Member] | Short-Term Bond Fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 5,007,050 | |
Significant Other Observable Inputs (Level 2) [Member] | U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 69,997,200 | $ 59,932,200 |
Basis of Presentation and Sig_6
Basis of Presentation and Significant Accounting Policies - Schedule Of Reconcile Basic And Dilutive Weighted Average Common Shares (Details) - shares | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Weighted Average Number Of Shares Outstanding Diluted Disclosure Items [Line Items] | ||
Basic weighted average common shares outstanding | 103,407,347 | 102,747,923 |
Effect of dilutive securities: | ||
Common stock issuable upon the exercise of stock options | 2,774,753 | |
Diluted weighted average common shares outstanding | 106,534,600 | 102,747,923 |
Restricted Stock Units (RSUs) [Member] | Share Based Payment Arrangement Employee And Nonemployee [Member] | ||
Effect of dilutive securities: | ||
Unvested restricted stock units subject to vesting requirements issued to employees and non-employees | 352,500 |
Investments - Additional Inform
Investments - Additional Information (Detail) - USD ($) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2020 | Dec. 31, 2019 | |
Investments, Debt and Equity Securities [Abstract] | ||
Realized gains losses from available for sale securities | $ 0 | $ 0 |
Investment - Summary of Availab
Investment - Summary of Available-for-Sale Investments by Security type (Detail) - USD ($) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2020 | Dec. 31, 2019 | |
Net Investment Income [Line Items] | ||
Amortized cost | $ 64,929,745 | |
Gross Unrealized Gains | 9,505 | |
Gross Unrealized Losses | 0 | |
Estimated Fair Value | $ 69,997,200 | 64,939,250 |
U.S. Treasuries - ST [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 4,999,587 | |
Gross Unrealized Gains | 7,463 | |
Gross Unrealized Losses | 0 | |
Estimated Fair Value | 5,007,050 | |
US Treasury Bond Securities Cash Equivalents [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 69,913,449 | 59,930,158 |
Gross Unrealized Gains | 83,751 | 2,042 |
Gross Unrealized Losses | 0 | 0 |
Estimated Fair Value | $ 69,997,200 | $ 59,932,200 |
Investment - Estimated Fair Val
Investment - Estimated Fair Values of Available for Sale Securities (Detail) - USD ($) | Mar. 31, 2020 | Dec. 31, 2019 |
Investments, Debt and Equity Securities [Abstract] | ||
Estimated Fair Value | $ 69,997,200 | $ 64,939,250 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($) | Mar. 31, 2020 | Dec. 31, 2019 |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid manufacturing costs | $ 3,471,078 | $ 1,526,013 |
Prepaid insurance | 995,146 | 1,263,129 |
Prepaid subscriptions fees | 492,716 | 501,251 |
Prepaid research fees | 490,102 | 481,057 |
Prepaid commercialization expenses | 287,083 | 62,959 |
Other | 326,590 | 516,665 |
Total prepaid expenses and other current assets | $ 6,062,715 | $ 4,351,074 |
Operating Leases - Operating Le
Operating Leases - Operating Leases (Detail) | 3 Months Ended |
Mar. 31, 2020USD ($) | |
Disclosure of Operating Leases [Abstract] | |
Operating lease cost | $ 74,079 |
Operating Leases - Schedule of
Operating Leases - Schedule of Supplemental Cash Flow Information Related To Lease (Detail) | 3 Months Ended |
Mar. 31, 2020USD ($) | |
Cash paid for amounts included in the measurement of lease liabilities: | |
Operating cash flows | $ 84,061 |
Right-of-use assets obtained in exchange for lease obligations: | |
Operating leases | $ 5,305 |
Operating Leases -Schedule of S
Operating Leases -Schedule of Supplemental Balance Sheet related To Lease (Detail) - USD ($) | Mar. 31, 2020 | Dec. 31, 2019 | Mar. 31, 2019 |
Disclosure of Operating Leases [Line Items] | |||
Operating lease right-of-use assets | $ 730,284 | $ 793,252 | |
Other current liabilities | 306,677 | 300,518 | |
Operating lease liabilities, net of current portion | 568,421 | $ 647,532 | |
Total operating lease liabilities | $ 875,098 | ||
Weighted average remaining lease term | 2 years 8 months 12 days | ||
Weighted average discount rate | 4.81% |
Operating Leases -Lessee, Opera
Operating Leases -Lessee, Operating Lease, Liability, Maturity (Detail) | Mar. 31, 2020USD ($) |
Disclosure of Operating Leases [Line Items] | |
2020 | $ 255,544 |
2021 | 349,788 |
2022 | 329,662 |
Total lease payments | 934,994 |
Less imputed interest | (59,896) |
Total | $ 875,098 |
Operating Leases - Additional
Operating Leases - Additional Information (Detail) | 3 Months Ended |
Mar. 31, 2020USD ($) | |
Disclosure of Operating Leases [Line Items] | |
Finance Lease Obligations | $ 0 |
Lessor, Operating Lease, Option to Extend | 1 year |
Lessor, Operating Lease, Option to Terminate | 1 year |
Accrued Expenses and Other Li_3
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($) | Mar. 31, 2020 | Dec. 31, 2019 |
Payables and Accruals [Abstract] | ||
Accrued preclinical and clinical trial expenses | $ 940,963 | $ 1,183,513 |
Accrued professional fees | 3,309,351 | 1,241,526 |
Accrued compensation and benefits | 1,814,396 | 3,064,645 |
Accrued license fees | 3,945,991 | 8,751,991 |
Accrued purchases | 1,243,991 | 1,313,310 |
Accrued contributions | 1,135,000 | 1,535,000 |
Operating lease liability | 306,677 | 300,518 |
Accrued variable consideration | 1,308,344 | 884,764 |
Income tax payable | 2,143,490 | 1,533,696 |
Other | 139,733 | 172,332 |
Current accrued expenses and other liabilities | 16,287,936 | 19,981,295 |
Lease liability - non-current | 568,421 | 647,532 |
Non-current accrued expenses and other liabilities | 568,421 | 647,532 |
Total accrued expenses and other liabilities | $ 16,856,357 | $ 20,628,827 |
Collaborative Arrangement - Add
Collaborative Arrangement - Additional Information (Detail) - USD ($) | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Collaborative Arrangement [Line Items] | ||
Period of collaboration agreement | 10 years | |
Expenses In Connection With Collaborative Arrangement | $ 4,222,811 | $ 3,307,959 |
Collaborative Arrangement [Member] | ||
Collaborative Arrangement [Line Items] | ||
Milestone payments | 2,000,000 | |
Milestone payments income | 0 | 0 |
Upfront License Fee | 0 | 0 |
Expenses In Connection With Collaborative Arrangement | $ 5,700 | $ 11,952 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) | 1 Months Ended |
May 21, 2019USD ($) | |
Settlement Agreement [Member] | Other Income [Member] | |
Commitments [Line Items] | |
Litigation Settlement Received | $ 100,000 |
Agreements - Additional Informa
Agreements - Additional Information (Detail) - License Agreement with BioMarin [Member] $ in Millions | 3 Months Ended |
Mar. 31, 2020USD ($) | |
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 |
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% |
Stockholders' Equity (Preferred
Stockholders' Equity (Preferred Stock and Common Stock) - Additional Information (Detail) | Mar. 31, 2020Vote$ / sharesshares | Dec. 31, 2019$ / 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 | 103,408,699 | 103,397,033 |
Common stock, shares outstanding | 103,408,699 | 103,397,033 |
Number of votes entitled for each share of common stock | Vote | 1 |
Stockholders' Equity (2017 Shel
Stockholders' Equity (2017 Shelf Registration Statement) - Additional Information (Detail) - USD ($) | Nov. 28, 2017 | Mar. 31, 2020 | 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 |
Stock Compensation - Stock-Base
Stock Compensation - Stock-Based Compensation Expense (Detail) - USD ($) | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | $ 1,519,351 | $ 933,411 |
Research and Development [Member] | ||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | 418,053 | 287,721 |
Selling, General and Administrative [Member] | ||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | $ 1,101,298 | $ 645,690 |
Stock Compensation - Additional
Stock Compensation - Additional Information (Detail) - USD ($) | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Proceeds from exercise of stock options | $ 26,149 | $ 89,350 |
Stock options to purchase shares of common stock | 12,201,668 | |
Stock Option [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock unit granted | 0 | 0 |
Options granted, expiration Period | 7 years | 7 years |
Restricted Stock Units (RSUs) [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan | $ 1,500,000 | |
Expected remaining weighted average vesting period | 2 years 8 months 1 day | |
Non-cash stock-based compensation expense | $ 135,679 | $ 0 |
Common stock granted | 0 | 0 |
Options to Purchase Common Stock [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of stock options exercised | 11,666 | 65,000 |
Proceeds from exercise of stock options | $ 26,149 | $ 89,350 |
Common stock unit granted | 735,000 | 207,000 |
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan | $ 10,400,000 | |
Expected remaining weighted average vesting period | 2 years 2 months 23 days | |
Non-cash stock-based compensation expense | $ 1,383,672 | $ 933,411 |
Stock option vested during the period | 854,831 | 984,164 |
Stock options to purchase shares of common stock | 6,687,980 |
Subsequent Events - Additional
Subsequent Events - Additional Information (Detail) - Subsequent Event [Member] | May 07, 2020ft² |
Minimum [Member] | |
Subsequent Event [Line Items] | |
Operating lease, number of square feet | ft² | 7,800 |
Maximum [Member] | |
Subsequent Event [Line Items] | |
Operating lease, number of square feet | ft² | 10,700 |