Cover Page
Cover Page - shares | 9 Months Ended | |
Sep. 30, 2020 | Nov. 05, 2020 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2020 | |
Document Fiscal Year Focus | 2020 | |
Document Fiscal Period Focus | Q3 | |
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 | 103,648,224 | |
Entity Tax Identification Number | 76-0837053 | |
Entity Incorporation, State or Country Code | DE | |
Entity Address, Address Line One | 355 Alhambra Circle | |
Entity Address, Address Line Two | Suite 1250 | |
Entity Address, City or Town | Coral Gables | |
Entity Address, Postal Zip Code | 33134 | |
City Area Code | 305 | |
Local Phone Number | 420-3200 | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity File Number | 001-33057 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) | Sep. 30, 2020 | Dec. 31, 2019 |
Current Assets: | ||
Cash and cash equivalents | $ 117,105,973 | $ 89,511,710 |
Short-term investments | 10,002,749 | 5,007,050 |
Accounts receivable, net | 5,871,893 | 10,536,997 |
Inventory | 4,747,538 | 1,956,792 |
Prepaid expenses and other current assets | 5,614,052 | 4,351,074 |
Total current assets | 143,342,205 | 111,363,623 |
Deferred tax assets | 31,347,442 | |
Operating lease right-of-use asset | 12,167 | 793,252 |
Property and equipment, net | 149,119 | 210,467 |
Deposits | 8,888 | 8,888 |
Total assets | 174,859,821 | 112,376,230 |
Current Liabilities: | ||
Accounts payable | 2,005,340 | 4,117,447 |
Accrued expenses and other liabilities | 16,226,609 | 19,981,295 |
Total current liabilities | 18,231,949 | 24,098,742 |
Operating lease liability, net of current portion | 647,532 | |
Total liabilities | 18,231,949 | 24,746,274 |
Commitments and contingencies | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at September 30, 2020 and December 31, 2019 | 0 | |
Common stock, $0.001 par value, 200,000,000 and 150,000,000 shares authorized; 103,648,224 shares and 103,397,033 shares issued and outstanding at September 30, 2020 and December 31, 2019, respectively | 103,648 | 103,397 |
Additional paid-in capital | 221,673,450 | 216,205,678 |
Accumulated deficit | (65,142,755) | (128,688,624) |
Accumulated other comprehensive income (loss) | (6,471) | 9,505 |
Total stockholders' equity | 156,627,872 | 87,629,956 |
Total liabilities and stockholders' equity | $ 174,859,821 | $ 112,376,230 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 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 | 200,000,000 | 150,000,000 |
Common stock, shares issued | 103,648,224 | 103,397,033 |
Common stock, shares outstanding | 103,648,224 | 103,397,033 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) (unaudited) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Revenues: | ||||
Product revenue, net | $ 29,166,658 | $ 30,897,444 | $ 87,907,894 | $ 72,183,782 |
Revenue, Product and Service [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Revenues from collaborative arrangements | $ 150,000 | $ 150,000 | ||
Total revenues | 29,316,658 | $ 30,897,444 | 88,057,894 | $ 72,183,782 |
Operating costs and expenses: | ||||
Cost of sales | $ 3,878,760 | $ 4,387,461 | $ 12,169,499 | $ 10,360,874 |
Cost, Product and Service [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Research and development | $ 3,749,233 | $ 4,597,039 | $ 12,321,687 | $ 12,534,362 |
Selling, general and administrative | 9,984,961 | 8,067,792 | 30,881,367 | 25,471,974 |
Total operating costs and expenses | 17,612,954 | 17,052,292 | 55,372,553 | 48,367,210 |
Operating income (loss) | 11,703,704 | 13,845,152 | 32,685,341 | 23,816,572 |
Other income, net | 33,567 | 393,415 | 481,069 | 1,187,091 |
Net income (loss) before income taxes | 11,737,271 | 14,238,567 | 33,166,410 | 25,003,663 |
Income tax provision (benefit) | (31,602,596) | 608,388 | (30,379,459) | 1,058,039 |
Net income (loss) | $ 43,339,867 | $ 13,630,179 | $ 63,545,869 | $ 23,945,624 |
Net income (loss) per share: | ||||
Basic | $ 0.42 | $ 0.13 | $ 0.61 | $ 0.23 |
Diluted | $ 0.41 | $ 0.13 | $ 0.60 | $ 0.23 |
Weighted average shares outstanding: | ||||
Basic | 103,535,431 | 102,974,105 | 103,452,025 | 102,864,571 |
Diluted | 106,316,241 | 107,045,234 | 106,386,617 | 105,821,609 |
Net income (loss) | $ 43,339,867 | $ 13,630,179 | $ 63,545,869 | $ 23,945,624 |
Other comprehensive income (loss): | ||||
Unrealized gain (loss) on available-for-sale securities | (5,280) | (2,330) | (15,976) | 39,676 |
Comprehensive income (loss) | $ 43,334,587 | $ 13,627,849 | $ 63,529,893 | $ 23,985,300 |
CONSOLIDATED STATEMENTS OF CHAN
CONSOLIDATED STATEMENTS 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, 2018 | 50,783,809 | $ 102,739 | 211,265,279 | (160,563,961) | (20,248) |
Beginning Balance (shares) at Dec. 31, 2018 | 102,739,257 | ||||
Net income (loss) | 23,945,624 | ||||
Ending Balance at Sep. 30, 2019 | 77,982,538 | $ 103,041 | 214,478,406 | (136,618,337) | 19,428 |
Ending Balance (shares) at Sep. 30, 2019 | 103,041,033 | ||||
Beginning Balance at Mar. 31, 2019 | 51,175,627 | $ 102,804 | 212,287,975 | (161,208,464) | (6,688) |
Beginning Balance (shares) at Mar. 31, 2019 | 102,804,257 | ||||
Issuance of stock options for services | 924,996 | 924,996 | |||
Exercise of stock options for common stock | 192,550 | $ 125 | 192,425 | ||
Exercise of stock options for common stock (shares) | 125,000 | ||||
Other comprehensive gain (loss) | 28,446 | 28,446 | |||
Net income (loss) | 10,959,948 | 10,959,948 | |||
Ending Balance at Jun. 30, 2019 | 63,281,567 | $ 102,929 | 213,405,396 | (150,248,516) | 21,758 |
Ending Balance (shares) at Jun. 30, 2019 | 102,929,257 | ||||
Issuance of stock options for services | 817,060 | 817,060 | |||
Exercise of stock options for common stock | 256,062 | $ 112 | 255,950 | ||
Exercise of stock options for common stock (shares) | 111,776 | ||||
Other comprehensive gain (loss) | (2,330) | (2,330) | |||
Net income (loss) | 13,630,179 | 13,630,179 | |||
Ending Balance at Sep. 30, 2019 | 77,982,538 | $ 103,041 | 214,478,406 | (136,618,337) | 19,428 |
Ending Balance (shares) at Sep. 30, 2019 | 103,041,033 | ||||
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 | ||||
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 | ||||
Net income (loss) | 63,545,869 | ||||
Ending Balance at Sep. 30, 2020 | 156,627,872 | $ 103,648 | 221,673,450 | (65,142,755) | (6,471) |
Ending Balance (shares) at Sep. 30, 2020 | 103,648,224 | ||||
Beginning Balance at Mar. 31, 2020 | 99,675,717 | $ 103,409 | 217,751,166 | (118,262,609) | 83,751 |
Beginning Balance (shares) at Mar. 31, 2020 | 103,408,699 | ||||
Issuance of stock options for services | 1,627,105 | 1,627,105 | |||
Exercise of stock options for common stock | 36,201 | $ 13 | 36,188 | ||
Exercise of stock options for common stock (shares) | 13,333 | ||||
Amortization of restricted stock for services | 167,357 | 167,357 | |||
Other comprehensive gain (loss) | (84,942) | (84,942) | |||
Net income (loss) | 9,779,987 | 9,779,987 | |||
Ending Balance at Jun. 30, 2020 | 111,201,425 | $ 103,422 | 219,581,816 | (108,482,622) | (1,191) |
Ending Balance (shares) at Jun. 30, 2020 | 103,422,032 | ||||
Issuance of stock options for services | 1,345,962 | 1,345,962 | |||
Exercise of stock options for common stock | 631,048 | $ 215 | 630,833 | ||
Exercise of stock options for common stock (shares) | 215,097 | ||||
Amortization of restricted stock for services | 131,884 | 131,884 | |||
Issuance of common stock upon vesting of restricted stock units, net | (17,034) | $ 11 | (17,045) | ||
Issuance of common stock upon vesting of restricted stock units, net (Share) | 11,095 | ||||
Other comprehensive gain (loss) | (5,280) | (5,280) | |||
Net income (loss) | 43,339,867 | 43,339,867 | |||
Ending Balance at Sep. 30, 2020 | $ 156,627,872 | $ 103,648 | $ 221,673,450 | $ (65,142,755) | $ (6,471) |
Ending Balance (shares) at Sep. 30, 2020 | 103,648,224 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited) - USD ($) | 9 Months Ended | |
Sep. 30, 2020 | Sep. 30, 2019 | |
Operating Activities: | ||
Net income (loss) | $ 63,545,869 | $ 23,945,624 |
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: | ||
Depreciation | 72,746 | 26,718 |
Amortization of right-of-use asset | 781,085 | 181,301 |
Stock-based compensation | 4,791,659 | 2,675,467 |
Change in accrued interest and accretion of discount on investments | (11,675) | (185,536) |
Deferred taxes | (31,347,442) | |
(Increase) decrease in: | ||
Accounts receivable, net | 4,665,104 | (10,095,352) |
Inventory | (2,790,746) | (543,789) |
Prepaid expenses and other current assets and deposits | (1,262,978) | (1,689,618) |
Increase (decrease) in: | ||
Accounts payable | (2,112,107) | 1,809,662 |
Accrued expenses and other liabilities | (3,568,729) | 6,400,279 |
Operating lease liability | (833,489) | (204,724) |
Net cash provided by (used in) operating activities | 31,929,297 | 22,320,032 |
Investing Activities: | ||
Purchases of property and equipment | (11,398) | (19,370) |
Purchases of investments | (10,000,000) | (34,725,401) |
Proceeds from maturities and sales of investments | 5,000,000 | 40,310,595 |
Net cash provided by (used in) investing activities | (5,011,398) | 5,565,824 |
Financing Activities: | ||
Payment of employee withholding tax related to stock-based compensation | (17,034) | |
Proceeds from exercise of stock options | 693,398 | 537,962 |
Net cash provided by (used in) financing activities | 676,364 | 537,962 |
Net increase (decrease) in cash and cash equivalents | 27,594,263 | 28,423,818 |
Cash and cash equivalents—beginning of period | 89,511,710 | 16,559,400 |
Cash and cash equivalents—end of period | 117,105,973 | 44,983,218 |
Supplemental disclosures of cash flow information: | ||
Cash paid for income taxes | 2,195,000 | |
Non-cash investing and financing activities: | ||
Unrealized gain (loss) on available-for-sale securities | $ (15,976) | $ 39,676 |
Organization and Description of
Organization and Description of Business | 9 Months Ended |
Sep. 30, 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 commercial-stage 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), and Spinal Muscular Atrophy (SMA) Type 3. On November 28, 2018, the U.S. Food and Drug Administration ( ) ® ® On August 6, 2020, the Company announced that Canada’s national healthcare regulatory agency, Health Canada, has approved 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 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 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 | 9 Months Ended |
Sep. 30, 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 The short-term bond funds and U.S. Treasuries held at September 30, 2020 are classified as available-for-sale non-current The Company records available-for-sale available-for-sale available-for-sale The Company previously owned a short-term bond fund that was 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 recognized classified as trading in 2019. There was no realized or unrealized gain (loss) on trading securities for the three and nine months ended September 30, 2020. Realized losses on trading securities during the three and nine months ended September 30, 2019 were $0 and $4,980, respectively. Unrealized gain (loss) on trading securities was $0 and $89,405 for the three and nine months ended September 30, 2019 , respectively, and is included in other income, net in the accompanying consolidated statements of operations. f. ACCOUNTS RECEIVABLE, NET. g. INVENTORY. first-in-first-out work-in-process ® ® 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 Significant Cash and cash equivalents: Money market funds $ 12,635,927 $ 12,635,927 $ — $ — U.S. Treasuries $ 94,992,100 $ — $ 94,992,100 $ — Short-term investments: Short-term bond funds $ 10,002,749 $ 10,002,749 $ — $ — Balances as of Quoted Prices in Significant Significant 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 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 Arrangements below. Product Revenue, Net: ® ® 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 and nine month periods ended September 30, 2020 and 2019. During the three and nine months ended September 30, 2020 and 2019, all of the Company’s sales were to its Customer. 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 September 30, 2020 and, therefore, the transaction price was not reduced further during the three and nine months ended September 30, 2020 and 2019. 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 determination, or later, for patients whose access is threatened by the complications arising from a change of insurer may receive a temporary supply of free Firdapse ® ® ® ® Revenues from Collaborative Arrangements: ® ® 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. In the third quarter of 2020, an upfront fee was earned under the collaboration agreement for the commercialization of Firdapse ® The collaborative agreements provide for milestone payments 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 milestones will be substantive and there will be no substantive uncertainty once the milestones are achieved. No milestones were achieved in the three and nine months ended September 30, 2020 and 2019. In arrangements where the Company does not deem the collaborator to be a customer, payments to and from the collaborator are presented in the statement of operations based on the nature of the Company’s business operations, the nature of the arrangement, including the contractual terms, and the nature of the payments. Under the arrangements, the Company will receive royalty reports 60 days after quarter end from one collaborator, and within nine days after quarter end from the other collaborator. 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. In instances where royalty reports are received within nine days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter in which the sales occurred. For the three and nine months ended September 30, 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. one 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 2017 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 Act did not have a material impact on the Company’s income tax provision. 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 September 30, For the Nine Months Ended September 30, 2020 2019 2020 2019 Basic weighted average common shares outstanding 103,535,431 102,974,105 103,452,025 102,864,571 Effect of dilutive securities 2,780,810 4,071,129 2,934,592 2,957,038 Dilutive weighted average common shares outstanding 106,316,241 107,045,234 106,386,617 105,821,609 Outstanding common stock equivalents totaling approximately 5.4 million and 4.8 million, were excluded from the calculation of diluted net income (loss) per common share for the three and nine months ended September 30, 2020 as their effect would be anti-dilutive. For the three and nine months ended September 30, 2019, approximately 0.4 million and 2.9 million shares of outstanding stock options were excluded from the calculation of diluted net income (loss) per common share as their effect would be anti-dilutive. t. RECLASSIFICATIONS. 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 2018-15, Intangibles – Goodwill and Other – Internal-Use 350-40), internal-use internal-use 350-40 |
Investments
Investments | 9 Months Ended |
Sep. 30, 2020 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | 3. Investments. Available-for-sale Estimated Fair Value Gross Gross Amortized Cost At September 30, 2020: U.S. Treasuries – Cash equivalents $ 94,992,100 $ — $ (1,195 ) $ 94,993,295 Bond Funds – ST 10,002,749 — (5,276 ) 10,008,025 Total $ 104,994,849 $ — $ (6,471 ) $ 105,001,320 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 The estimated fair values of available-for-sale September September 30, 2020 Due in one year or less $ 104,994,849 |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 9 Months Ended |
Sep. 30, 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: September 30, 2020 December 31, 2019 Prepaid manufacturing costs $ 3,268,032 $ 1,526,013 Prepaid insurance 247,610 1,263,129 Prepaid subscription fees 561,430 501,251 Prepaid research fees 472,093 481,057 Prepaid commercialization expenses 168,611 62,959 Due from collaborative arrangements 306,678 — Other 589,598 516,665 Total prepaid expenses and other current assets $ 5,614,052 $ 4,351,074 |
Operating Leases
Operating Leases | 9 Months Ended |
Sep. 30, 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 Company entered into an agreement in May 2020 that amended its lease for its office facilities. Under the amended lease, the Company’s leased space will increase from approximately 7,800 square feet of space to approximately 10,700 square feet of space. The lessor is currently building this space. The lease is expected to commence in early 2021 when construction of the asset is completed and available for use. The lease disclosures for the three and nine months periods ended September 30, 2020 in these financial statements have been adjusted for the modification of the current lease. The components of lease expense were as follows: For the Three Months Ended September 30, 2020 For the Nine Months Ended September 30, 2020 Operating lease cost $ 61,111 $ 200,624 Supplemental cash flow information related to leases was as follows: September 30, 2020 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows $ 253,024 Right-of-use Operating leases $ 37,528 Supplemental balance sheet information related to leases was as follows: September 30, 2020 Operating lease right-of-use $ 12,167 Other current liabilities $ 114,563 Operating lease liabilities, net of current portion — Total operating lease liabilities $ 114,563 Weighted average remaining lease term 0.3 years Weighted average discount rate 3.68 % Remaining payments of lease liabilities as of September 30, 2020 were as follows: 2020 (remaining three months) $ 86,582 2021 28,860 2022 — Total lease payments 115,442 Less imputed interest (879 ) Total $ 114,563 |
Accrued Expenses and Other Liab
Accrued Expenses and Other Liabilities | 9 Months Ended |
Sep. 30, 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: September 30, 2020 December 31, 2019 Accrued preclinical and clinical trial expenses $ 643,578 $ 1,183,513 Accrued professional fees 1,923,164 1,241,526 Accrued compensation and benefits 2,877,077 3,064,645 Accrued license fees 7,840,955 8,751,991 Accrued purchases 1,332,190 1,313,310 Accrued contributions — 1,535,000 Operating lease liability 114,563 300,518 Accrued variable consideration 859,399 884,764 Accrued income tax 566,075 1,533,696 Other 69,608 172,332 Current accrued expenses and other liabilities 16,226,609 19,981,295 Lease liability—non-current — 647,532 Non-current — 647,532 Total accrued expenses and other liabilities $ 16,226,609 $ 20,628,827 |
Collaborative Arrangements
Collaborative Arrangements | 9 Months Ended |
Sep. 30, 2020 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaborative Arrangements | 7. Collaborative Arrangements. 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-digit ® The collaborative agreement provides for a $2.0 million milestone payment on the commercial launch of the product by Par. As of September 30, 2020 and 2019, no milestone payments have been earned. There were no revenues from collaborative arrangement with Endo for the three or nine months ended September 30, 2020 and 2019. Total expenses incurred, net, in connection with the collaborative agreement with Endo for three and nine months ended September 30, 2020 were approximately $0 and $4,200, respectively. Total expenses incurred, net, in connection with the collaborative agreement with Endo for three and nine months ended September 30, 2019 were approximately $32,000 and $70,000, respectively. These expenses have been included in research and development expenses in the accompanying consolidated statements of operations. In August 2020, the Company entered into a collaboration and license agreement with KYE Pharmaceuticals Inc (KYE), for the commercialization of Firdapse ® Under the agreement, KYE assumes all selling and marketing costs under the collaboration, while the Company is responsible for supply of Firdapse ® Under the terms of the agreement, the Company has earned an up-front mid-double-digit ® As of September 30, 2020, a $150,000 upfront fee was recognized in connection with the collaborative agreement with KYE. Total expenses incurred, net, in connection with the collaborative agreement with KYE for three and nine months ended September 30, 2020 were approximately $161,000. These expenses have been included in selling, general and administrative expenses in the accompanying consolidated statements of operations. The collaborative agreement provides for event-based payments subject to achievement of specified development, regulatory and sales-based milestones. As of September 30, 2020, no milestone payments have been earned. |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 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 CPP-115, CPP-115 In May 2019, the FDA approved an NDA for Jacobus Pharmaceuticals (“Jacobus”) for Ruzurgi ® (3,4-DAP), ® ® ® ® ® off-label The Company believes that the FDA’s approval of Ruzurgi ® ® ® On July 30, 2020, the Magistrate Judge considering the Company’s lawsuit against the FDA filed a Report and Recommendation in which she recommended to the District Judge handling the case that she grant the FDA’s and Jacobus’ motions for summary judgement and deny the Company’s motion for summary judgement. On September 29, 2020, the District Judge adopted the Report and Recommendation of the Magistrate Judge, granted the FDA’s and Jacobus’s motions for summary judgement, and dismissed the Company’s case. The Company has appealed the decision to the Eleventh Circuit Court of Appeals. There can be no assurance as to the outcome of this lawsuit. On August 10, 2020, Health Canada issued a Notice of Compliance (NOC) to Medunik for 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 | 9 Months Ended |
Sep. 30, 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. |
Income Taxes
Income Taxes | 9 Months Ended |
Sep. 30, 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 2017 The Company has evaluated the positive and negative e v |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 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 September 30, 2020 and December 31, 2019. No shares of preferred stock were outstanding at September 30, 2020 and December 31, 2019. Common Stock On August 20, 2020, the Company’s stockholders approved an increase in the Company’s authorized common stock par value $0.001 per share, from 150,000,000 shares to 2020 Shelf Registration Statement On July 23, 2020, the Company filed a shelf registration statement with the SEC to sell up to $200 million of common stock, preferred stock, warrants to purchase common stock, debt securities and units consisting of one or more of such securities (the “2020 Shelf Registration Statement”). The 2020 Shelf Registration Statement (file no. 333-240052) |
Stock Compensation
Stock Compensation | 9 Months Ended |
Sep. 30, 2020 | |
Share-based Payment Arrangement [Abstract] | |
Stock Compensation | 12. Stock Compensation. For the three and nine-month periods ended September 30, 2020 and 2019, the Company recorded stock-based compensation expense as follows: Three months ended September 30, Nine months ended September 30, 2020 2019 2020 2019 Research and development $ 399,247 $ 242,867 $ 1,238,521 $ 803,800 Selling, general and administrative 1,078,599 574,193 3,553,138 1,871,667 Total stock-based compensation $ 1,477,846 $ 817,060 $ 4,791,659 $ 2,675,467 Stock Options As of September 30, 2020, there were outstanding stock options to purchase 11,790,335 shares of common stock, of which stock options to purchase 6,833,809 shares of common stock were exercisable as of September 30, 2020. During the three and nine-month periods ended September 30, 2020, the Company granted seven-year term options to purchase an aggregate of 10,000 and 1,005,000 shares, respectively, of the Company’s common stock to employees and directors. The Company recorded stock-based compensation related to stock options totaling $1,345,962 and $4,356,739, respectively, during the three and nine-month periods ended September 30, 2020. During the three and nine-month periods ended September 30, 2020, respectively, 348,163 and 1,542,992 options vested. During the three and nine-month periods ended September 30, 2019, the Company granted seven-year term options to purchase an aggregate of 172,500 and 484,500 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $817,060 and $2,675,467, respectively, during the three and nine-month periods ended September 30, 2019. During the three and nine-month periods ended September 30, 2019, respectively, 74,998 and 1,365,827 options vested. During the three and nine-month periods ended September 30, 2020, options to purchase 215,097 shares and 240,096 shares, respectively, of the Company’s common stock were exercised, with proceeds of $631,048 and $693,398 respectively, to the Company. During the three and nine-month periods ended September 30, 2019, options to purchase 108,332 shares and 298,332 shares, respectively, of the Company’s common stock were exercised, with proceeds of $256,062 and $537,962 respectively, to the Company. During both the three and nine-month periods ended September 30, 2019, options to purchase 6,666 shares of the Company’s common stock were exercised on a “cashless” basis , resulting in the issuance of an aggregate of 3,444 As of September 30, 2020, there was approximately $7.8 million of unrecognized compensation expense related to non-vested Restricted Stock Units The Company granted zero and 30,000 restricted stock units during the three and nine-month periods ended September 30, 2020, respectively. There were no restricted stock units granted during the three and nine-month periods ended September 30, 2019. During the three and nine-month periods ended September 30, 2020, the Company recorded non-cash As of September 30, 2020, there was approximately $1.2 million of unrecognized compensation expense related to non-vested |
Subsequent Events
Subsequent Events | 9 Months Ended |
Sep. 30, 2020 | |
Subsequent Events [Abstract] | |
Subsequent Events | 13. Subsequent Events. On October 6, 2020, the United States Patent and Trademark Office issued U.S. Patent 10,793,893 (the ‘893 patent). The ’893 patent is exclusively licensed to the Company and covers certain methods for treating disease using amifampridine drug products, including Catalyst’s Firdapse ® On October 19, 2020, the Company announced that it has filed a lawsuit in the U.S. District Court for New Jersey against Jacobus, and a lawsuit in the U.S. District Court for the Western District of Pennsylvania against PantherRx Rare LLC (PantherRx) for infringement of the ’893 patent. The lawsuit arises from Jacobus’ and PantherRx’s sales and marketing of Ruzurgi ® ® ® There can be no assurance as to the outcome of this matter. |
Basis of Presentation and Sig_2
Basis of Presentation and Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 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 The short-term bond funds and U.S. Treasuries held at September 30, 2020 are classified as available-for-sale non-current The Company records available-for-sale available-for-sale available-for-sale The Company previously owned a short-term bond fund that was 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 recognized classified as trading in 2019. There was no realized or unrealized gain (loss) on trading securities for the three and nine months ended September 30, 2020. Realized losses on trading securities during the three and nine months ended September 30, 2019 were $0 and $4,980, respectively. Unrealized gain (loss) on trading securities was $0 and $89,405 for the three and nine months ended September 30, 2019 , respectively, and is included in other income, net in the accompanying consolidated statements of operations. |
ACCOUNTS RECEIVABLE, NET | f. ACCOUNTS RECEIVABLE, NET. |
INVENTORY | g. INVENTORY. first-in-first-out work-in-process ® ® 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 Significant Cash and cash equivalents: Money market funds $ 12,635,927 $ 12,635,927 $ — $ — U.S. Treasuries $ 94,992,100 $ — $ 94,992,100 $ — Short-term investments: Short-term bond funds $ 10,002,749 $ 10,002,749 $ — $ — Balances as of Quoted Prices in Significant Significant 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 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 Arrangements below. Product Revenue, Net: ® ® 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 and nine month periods ended September 30, 2020 and 2019. During the three and nine months ended September 30, 2020 and 2019, all of the Company’s sales were to its Customer. 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 September 30, 2020 and, therefore, the transaction price was not reduced further during the three and nine months ended September 30, 2020 and 2019. 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 determination, or later, for patients whose access is threatened by the complications arising from a change of insurer may receive a temporary supply of free Firdapse ® ® ® ® Revenues from Collaborative Arrangements: ® ® 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. In the third quarter of 2020, an upfront fee was earned under the collaboration agreement for the commercialization of Firdapse ® The collaborative agreements provide for milestone payments 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 milestones will be substantive and there will be no substantive uncertainty once the milestones are achieved. No milestones were achieved in the three and nine months ended September 30, 2020 and 2019. In arrangements where the Company does not deem the collaborator to be a customer, payments to and from the collaborator are presented in the statement of operations based on the nature of the Company’s business operations, the nature of the arrangement, including the contractual terms, and the nature of the payments. Under the arrangements, the Company will receive royalty reports 60 days after quarter end from one collaborator, and within nine days after quarter end from the other collaborator. 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. In instances where royalty reports are received within nine days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter in which the sales occurred. For the three and nine months ended September 30, 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. one |
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 2017 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 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 September 30, For the Nine Months Ended September 30, 2020 2019 2020 2019 Basic weighted average common shares outstanding 103,535,431 102,974,105 103,452,025 102,864,571 Effect of dilutive securities 2,780,810 4,071,129 2,934,592 2,957,038 Dilutive weighted average common shares outstanding 106,316,241 107,045,234 106,386,617 105,821,609 Outstanding common stock equivalents totaling approximately 5.4 million and 4.8 million, were excluded from the calculation of diluted net income (loss) per common share for the three and nine months ended September 30, 2020 as their effect would be anti-dilutive. For the three and nine months ended September 30, 2019, approximately 0.4 million and 2.9 million shares of outstanding stock options were excluded from the calculation of diluted net income (loss) per common share as their effect would be 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 2018-15, Intangibles – Goodwill and Other – Internal-Use 350-40), internal-use internal-use 350-40 |
Basis of Presentation and Sig_3
Basis of Presentation and Significant Accounting Policies (Tables) | 9 Months Ended |
Sep. 30, 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 Significant Cash and cash equivalents: Money market funds $ 12,635,927 $ 12,635,927 $ — $ — U.S. Treasuries $ 94,992,100 $ — $ 94,992,100 $ — Short-term investments: Short-term bond funds $ 10,002,749 $ 10,002,749 $ — $ — Balances as of Quoted Prices in Significant Significant 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 September 30, For the Nine Months Ended September 30, 2020 2019 2020 2019 Basic weighted average common shares outstanding 103,535,431 102,974,105 103,452,025 102,864,571 Effect of dilutive securities 2,780,810 4,071,129 2,934,592 2,957,038 Dilutive weighted average common shares outstanding 106,316,241 107,045,234 106,386,617 105,821,609 |
Investments (Tables)
Investments (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available-for-Sale Investments by Security type | Available-for-sale Estimated Fair Value Gross Gross Amortized Cost At September 30, 2020: U.S. Treasuries – Cash equivalents $ 94,992,100 $ — $ (1,195 ) $ 94,993,295 Bond Funds – ST 10,002,749 — (5,276 ) 10,008,025 Total $ 104,994,849 $ — $ (6,471 ) $ 105,001,320 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 September September 30, 2020 Due in one year or less $ 104,994,849 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Text Block [Abstract] | |
Prepaid expenses and other current assets | Prepaid expenses and other current assets consist of the following: September 30, 2020 December 31, 2019 Prepaid manufacturing costs $ 3,268,032 $ 1,526,013 Prepaid insurance 247,610 1,263,129 Prepaid subscription fees 561,430 501,251 Prepaid research fees 472,093 481,057 Prepaid commercialization expenses 168,611 62,959 Due from collaborative arrangements 306,678 — Other 589,598 516,665 Total prepaid expenses and other current assets $ 5,614,052 $ 4,351,074 |
Operating Leases (Tables)
Operating Leases (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Disclosure of Operating Leases [Abstract] | |
Lease, Cost | The components of lease expense were as follows: For the Three Months Ended September 30, 2020 For the Nine Months Ended September 30, 2020 Operating lease cost $ 61,111 $ 200,624 |
Schedule of Supplemental Cash Flow Information Related To Lease | Supplemental cash flow information related to leases was as follows: September 30, 2020 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows $ 253,024 Right-of-use Operating leases $ 37,528 |
Schedule of Supplemental Balance Sheet related To Lease | Supplemental balance sheet information related to leases was as follows: September 30, 2020 Operating lease right-of-use $ 12,167 Other current liabilities $ 114,563 Operating lease liabilities, net of current portion — Total operating lease liabilities $ 114,563 Weighted average remaining lease term 0.3 years Weighted average discount rate 3.68 % |
Lessee, Operating Lease, Liability, Maturity | Remaining payments of lease liabilities as of September 30, 2020 were as follows: 2020 (remaining three months) $ 86,582 2021 28,860 2022 — Total lease payments 115,442 Less imputed interest (879 ) Total $ 114,563 |
Accrued Expenses and Other Li_2
Accrued Expenses and Other Liabilities (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Liabilities | Accrued expenses and other liabilities consist of the following: September 30, 2020 December 31, 2019 Accrued preclinical and clinical trial expenses $ 643,578 $ 1,183,513 Accrued professional fees 1,923,164 1,241,526 Accrued compensation and benefits 2,877,077 3,064,645 Accrued license fees 7,840,955 8,751,991 Accrued purchases 1,332,190 1,313,310 Accrued contributions — 1,535,000 Operating lease liability 114,563 300,518 Accrued variable consideration 859,399 884,764 Accrued income tax 566,075 1,533,696 Other 69,608 172,332 Current accrued expenses and other liabilities 16,226,609 19,981,295 Lease liability—non-current — 647,532 Non-current — 647,532 Total accrued expenses and other liabilities $ 16,226,609 $ 20,628,827 |
Stock Compensation (Tables)
Stock Compensation (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Share-based Payment Arrangement [Abstract] | |
Stock-Based Compensation Expense | For the three and nine-month periods ended September 30, 2020 and 2019, the Company recorded stock-based compensation expense as follows: Three months ended September 30, Nine months ended September 30, 2020 2019 2020 2019 Research and development $ 399,247 $ 242,867 $ 1,238,521 $ 803,800 Selling, general and administrative 1,078,599 574,193 3,553,138 1,871,667 Total stock-based compensation $ 1,477,846 $ 817,060 $ 4,791,659 $ 2,675,467 |
Basis of Presentation and Sig_4
Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($) shares in Millions | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Dec. 31, 2019 | |
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) | $ 0 | $ 4,980 | |||
Trading securities, realized or unrealized gain (loss) | $ 0 | $ 0 | |||
Potential equivalent common stock excluded | 5.4 | 0.4 | 4.8 | 2.9 | |
Trading securities | $ 0 | $ 0 | $ 0 | ||
Other Income, Net [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Unrealized gain (loss), trading securities | $ 0 | $ 89,405 | |||
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 | 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 ($) | Sep. 30, 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 | |
Investments | $ 10,002,749 | |
U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 94,992,100 | 59,932,200 |
Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 12,635,927 | 23,963,617 |
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 | 10,002,749 | |
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 | 12,635,927 | 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 | $ 94,992,100 | $ 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 | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | ||||
Basic weighted average common shares outstanding | 103,535,431 | 102,974,105 | 103,452,025 | 102,864,571 |
Effect of dilutive securities | 2,780,810 | 4,071,129 | 2,934,592 | 2,957,038 |
Dilutive weighted average common shares outstanding | 106,316,241 | 107,045,234 | 106,386,617 | 105,821,609 |
Investments - Additional Inform
Investments - Additional Information (Detail) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Investments, Debt and Equity Securities [Abstract] | ||||
Realized gains losses from available for sale securities | $ 0 | $ 0 | $ 0 | $ 0 |
Investment - Summary of Availab
Investment - Summary of Available-for-Sale Investments by Security type (Detail) - USD ($) | 9 Months Ended | 12 Months Ended |
Sep. 30, 2020 | Dec. 31, 2019 | |
Net Investment Income [Line Items] | ||
Amortized cost | $ 105,001,320 | $ 64,929,745 |
Gross Unrealized Gains | 9,505 | |
Gross Unrealized Losses | (6,471) | |
Estimated Fair Value | 104,994,849 | 64,939,250 |
U.S. Treasuries - ST [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 4,999,587 | |
Gross Unrealized Gains | 7,463 | |
Estimated Fair Value | 5,007,050 | |
U.S Treasury Bond Securities Cash Equivalents [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 94,993,295 | 59,930,158 |
Gross Unrealized Gains | 2,042 | |
Gross Unrealized Losses | (1,195) | |
Estimated Fair Value | 94,992,100 | $ 59,932,200 |
Bond Funds – ST [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 10,008,025 | |
Gross Unrealized Losses | (5,276) | |
Estimated Fair Value | $ 10,002,749 |
Investment - Estimated Fair Val
Investment - Estimated Fair Values of Available for Sale Securities (Detail) - USD ($) | Sep. 30, 2020 | Dec. 31, 2019 |
Investments, Debt and Equity Securities [Abstract] | ||
Estimated Fair Value | $ 104,994,849 | $ 64,939,250 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($) | Sep. 30, 2020 | Dec. 31, 2019 |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid manufacturing costs | $ 3,268,032 | $ 1,526,013 |
Prepaid insurance | 247,610 | 1,263,129 |
Prepaid subscriptions fees | 561,430 | 501,251 |
Prepaid research fees | 472,093 | 481,057 |
Prepaid commercialization expenses | 168,611 | 62,959 |
Due from collaborative arrangements | 306,678 | |
Other | 589,598 | 516,665 |
Total prepaid expenses and other current assets | $ 5,614,052 | $ 4,351,074 |
Operating Leases - Operating Le
Operating Leases - Operating Leases (Detail) - USD ($) | 3 Months Ended | 9 Months Ended |
Sep. 30, 2020 | Sep. 30, 2020 | |
Disclosure of Operating Leases [Abstract] | ||
Operating lease cost | $ 61,111 | $ 200,624 |
Operating Leases - Schedule of
Operating Leases - Schedule of Supplemental Cash Flow Information Related To Lease (Detail) | 9 Months Ended |
Sep. 30, 2020USD ($) | |
Cash paid for amounts included in the measurement of lease liabilities: | |
Operating cash flows | $ 253,024 |
Right-of-use assets obtained in exchange for lease obligations: | |
Operating leases | $ 37,528 |
Operating Leases -Schedule of S
Operating Leases -Schedule of Supplemental Balance Sheet related To Lease (Detail) - USD ($) | Sep. 30, 2020 | Dec. 31, 2019 |
Disclosure of Operating Leases [Line Items] | ||
Operating lease right-of-use assets | $ 12,167 | $ 793,252 |
Other current liabilities | 114,563 | 300,518 |
Operating lease liabilities, net of current portion | $ 647,532 | |
Total operating lease liabilities | $ 114,563 | |
Weighted average remaining lease term | 3 months 18 days | |
Weighted average discount rate | 3.68% |
Operating Leases -Lessee, Opera
Operating Leases -Lessee, Operating Lease, Liability, Maturity (Detail) | Sep. 30, 2020USD ($) |
Disclosure of Operating Leases [Line Items] | |
2020 (remaining three months) | $ 86,582 |
2021 | 28,860 |
2022 | |
Total lease payments | 115,442 |
Less imputed interest | (879) |
Total | $ 114,563 |
Operating Leases - Additional
Operating Leases - Additional Information (Detail) | 9 Months Ended |
Sep. 30, 2020USD ($)ft² | |
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 |
Before agreement of company leased spaces | 7,800 |
After agreement of company leased spaces | 10,700 |
Accrued Expenses and Other Li_3
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($) | Sep. 30, 2020 | Dec. 31, 2019 |
Payables and Accruals [Abstract] | ||
Accrued preclinical and clinical trial expenses | $ 643,578 | $ 1,183,513 |
Accrued professional fees | 1,923,164 | 1,241,526 |
Accrued compensation and benefits | 2,877,077 | 3,064,645 |
Accrued license fees | 7,840,955 | 8,751,991 |
Accrued purchases | 1,332,190 | 1,313,310 |
Accrued contributions | 1,535,000 | |
Operating lease liability | 114,563 | 300,518 |
Accrued variable consideration | 859,399 | 884,764 |
Accrued income tax | 566,075 | 1,533,696 |
Other | 69,608 | 172,332 |
Current accrued expenses and other liabilities | 16,226,609 | 19,981,295 |
Lease liability - non-current | 647,532 | |
Non-current accrued expenses and other liabilities | 647,532 | |
Total accrued expenses and other liabilities | $ 16,226,609 | $ 20,628,827 |
Collaborative Arrangements - Ad
Collaborative Arrangements - Additional Information (Detail) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Collaborative Arrangements [Line Items] | ||||
Period of collaboration agreement | 10 years | |||
Upfront License Fee | $ 29,316,658 | $ 30,897,444 | $ 88,057,894 | $ 72,183,782 |
Expenses In Connection With Collaborative Arrangement | 3,749,233 | 4,597,039 | $ 12,321,687 | 12,534,362 |
Period of collaboration agreement | 10 years | |||
Upfront fee | 150,000 | $ 150,000 | ||
Collaborative Arrangement [Member] | ||||
Collaborative Arrangements [Line Items] | ||||
Milestone payments | 2,000,000 | |||
Milestone payments income | 0 | 0 | ||
Upfront License Fee | 0 | 0 | ||
Expenses In Connection With Collaborative Arrangement | 0 | $ 32,000 | $ 4,200 | $ 70,000 |
Collaborative Arrangement [Member] | KYE Pharmaceuticals [Member] | CA [Member] | ||||
Collaborative Arrangements [Line Items] | ||||
Period of collaboration agreement | 10 years | |||
Period of collaboration agreement | 10 years | |||
Upfront fee | $ 150,000 | |||
Collaborative Arrangement [Member] | KYE Pharmaceuticals [Member] | CA [Member] | Selling, General and Administrative Expenses [Member] | ||||
Collaborative Arrangements [Line Items] | ||||
Expenses In Connection With Collaborative Arrangement | $ 161,000 | $ 161,000 |
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 | 9 Months Ended |
Sep. 30, 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% |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) | 9 Months Ended |
Sep. 30, 2020USD ($) | |
Income Tax Disclosure [Abstract] | |
Deferred tax assets | $ 31,347,442 |
Deferred tax assets recognised in income statement | $ 31,300,000 |
Stockholders' Equity (Preferred
Stockholders' Equity (Preferred Stock and Common Stock) - Additional Information (Detail) | Sep. 30, 2020Vote$ / sharesshares | Aug. 20, 2020$ / sharesshares | Dec. 31, 2019$ / sharesshares |
Stockholders Equity [Line Items] | |||
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 | 200,000,000 | 150,000,000 | |
Common stock, par value | $ / shares | $ 0.001 | $ 0.001 | $ 0.001 |
Common stock, shares issued | 103,648,224 | 103,397,033 | |
Common stock, shares outstanding | 103,648,224 | 103,397,033 | |
Number of votes entitled for each share of common stock | Vote | 1 | ||
Maximum [Member] | |||
Stockholders Equity [Line Items] | |||
Common stock, shares authorized | 150,000,000 | ||
Minimum [Member] | |||
Stockholders Equity [Line Items] | |||
Common stock, shares authorized | 200,000,000 |
Stockholders' Equity (2020 Shel
Stockholders' Equity (2020 Shelf Registration Statement) - Additional Information (Detail) $ in Millions | Jul. 23, 2020USD ($) |
2020 Shelf Registration Statement [Member] | |
Stockholders' Equity [Line Items] | |
Maximum dollar amount of common stock to be issued under shelf registration statement | $ 200 |
Stock Compensation - Stock-Base
Stock Compensation - Stock-Based Compensation Expense (Detail) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | $ 1,477,846 | $ 817,060 | $ 4,791,659 | $ 2,675,467 |
Research and Development [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | 399,247 | 242,867 | 1,238,521 | 803,800 |
Selling, General and Administrative [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | $ 1,078,599 | $ 574,193 | $ 3,553,138 | $ 1,871,667 |
Stock Compensation - Additional
Stock Compensation - Additional Information (Detail) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Proceeds from exercise of stock options | $ 693,398 | $ 537,962 | ||
Stock options to purchase shares of common stock | 11,790,335 | 11,790,335 | ||
Stock Option [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Options granted, expiration Period | 7 years | 7 years | 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 | $ 1,200,000 | $ 1,200,000 | ||
Expected remaining weighted average vesting period | 2 years 2 months 12 days | |||
Non-cash stock-based compensation expense | $ 131,884 | $ 0 | $ 434,920 | $ 0 |
Common stock granted | 0 | 0 | 30,000 | 0 |
Options to Purchase Common Stock [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Number of stock options exercised | 215,097 | 108,332 | 240,096 | 298,332 |
Proceeds from exercise of stock options | $ 631,048 | $ 256,062 | $ 693,398 | $ 537,962 |
Common stock unit granted | 10,000 | 172,500 | 1,005,000 | 484,500 |
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan | $ 7,800,000 | $ 7,800,000 | ||
Expected remaining weighted average vesting period | 2 years 1 month 6 days | |||
Non-cash stock-based compensation expense | $ 1,345,962 | $ 817,060 | $ 4,356,739 | $ 2,675,467 |
Stock option vested during the period | 348,163 | 74,998 | 1,542,992 | 1,365,827 |
Stock options to purchase shares of common stock | 6,833,809 | 6,833,809 | ||
Common stock shares available for future issuance under the Plan | 3,444 | 3,444 | ||
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 on cashless basis | 6,666 |