Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2019 | May 06, 2019 | |
Document and Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2019 | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q1 | |
Entity Registrant Name | CATALYST PHARMACEUTICALS, INC. | |
Entity Central Index Key | 0001369568 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Accelerated Filer | |
Trading Symbol | CPRX | |
Entity Emerging Growth Company | false | |
Entity Small Business | true | |
Entity Common Stock, Shares Outstanding | 102,854,257 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) | Mar. 31, 2019 | Dec. 31, 2018 |
Current Assets: | ||
Cash and cash equivalents | $ 19,081,714 | $ 16,559,400 |
Short-term investments | 26,551,064 | 36,922,213 |
Accounts receivable, net | 7,251,381 | 0 |
Inventory | 96,587 | 56,012 |
Prepaid expenses and other current assets | 1,881,266 | 1,649,781 |
Total current assets | 54,862,012 | 55,187,406 |
Investments | 4,991,600 | 5,008,243 |
Operating lease right-of-use asset | 1,074,020 | 0 |
Property and equipment, net | 140,320 | 245,425 |
Deposits | 8,888 | 8,888 |
Total assets | 61,076,840 | 60,449,962 |
Current Liabilities: | ||
Accounts payable | 2,940,086 | 2,337,367 |
Accrued expenses and other liabilities | 6,086,029 | 7,173,987 |
Total current liabilities | 9,026,115 | 9,511,354 |
Accrued expenses and other liabilities, non-current | 0 | 154,799 |
Operating lease liability, net of current portion | 875,098 | 0 |
Total liabilities | 9,901,213 | 9,666,153 |
Commitments and contingencies | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at March 31, 2019 and December 31, 2018 | 0 | 0 |
Common stock, $0.001 par value, 150,000,000 shares authorized; 102,804,257 shares and 102,739,257 shares issued and outstanding at March 31, 2019 and December 31, 2018, respectively | 102,804 | 102,739 |
Additional paid-in capital | 212,287,975 | 211,265,279 |
Accumulated deficit | (161,208,464) | (160,563,961) |
Accumulated other comprehensive loss | (6,688) | (20,248) |
Total stockholders' equity | 51,175,627 | 50,783,809 |
Total liabilities and stockholders' equity | $ 61,076,840 | $ 60,449,962 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2019 | Dec. 31, 2018 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, shares issued | 102,804,257 | 102,739,257 |
Common stock, shares outstanding | 102,804,257 | 102,739,257 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Revenues: | ||
Product revenue, net | $ 12,448,438 | $ 0 |
Operating costs and expenses: | ||
Cost of sales | 1,711,788 | 0 |
Research and development | 3,307,959 | 3,259,042 |
Selling, general and administrative | 8,416,460 | 2,674,398 |
Total operating costs and expenses | 13,436,207 | 5,933,440 |
Loss from operations | (987,769) | (5,933,440) |
Other income, net | 343,266 | 233,548 |
Loss before income taxes | (644,503) | (5,699,892) |
Provision for income taxes | 0 | 0 |
Net loss | $ (644,503) | $ (5,699,892) |
Net loss per share - basic and diluted | $ (0.01) | $ (0.06) |
Weighted average shares outstanding - basic and diluted | 102,747,923 | 102,557,350 |
Net loss | $ (644,503) | $ (5,699,892) |
Other comprehensive loss: | ||
Unrealized gain (loss) on available-for-sale securities | 13,560 | (21,826) |
Comprehensive loss | $ (630,943) | $ (5,721,718) |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholders' Equity - USD ($) | Total | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Deficit [Member] | Accumulated Other Comprehensive Gain (Loss) [Member] |
Beginning Balance at Dec. 31, 2017 | $ 80,963,812 | $ 102,549 | $ 207,421,710 | $ (126,560,447) | |
Issuance of stock options for services | 971,340 | 971,340 | |||
Exercise of stock options for common stock | 33,032 | 37 | 32,995 | ||
Other comprehensive gain (loss) | (21,826) | $ (21,826) | |||
Net loss | (5,699,892) | (5,699,892) | |||
Ending Balance at Mar. 31, 2018 | 76,246,466 | 102,586 | 208,426,045 | (132,260,339) | (21,826) |
Beginning Balance at Dec. 31, 2018 | 50,783,809 | 102,739 | 211,265,279 | (160,563,961) | (20,248) |
Issuance of stock options for services | 933,411 | 933,411 | |||
Exercise of stock options for common stock | 89,350 | 65 | 89,285 | ||
Other comprehensive gain (loss) | 13,560 | 13,560 | |||
Net loss | (644,503) | (644,503) | |||
Ending Balance at Mar. 31, 2019 | $ 51,175,627 | $ 102,804 | $ 212,287,975 | $ (161,208,464) | $ (6,688) |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Operating Activities: | ||
Net loss | $ (644,503) | $ (5,699,892) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation | 13,748 | 8,015 |
Non-cash change in right-of-use asset | 59,621 | 0 |
Stock-based compensation | 933,411 | 971,340 |
(Increase) decrease in: | ||
Accounts receivable, net | (7,251,381) | 0 |
Inventory | (40,575) | 0 |
Prepaid expenses and other current assets and deposits | (231,485) | 180,916 |
Increase (decrease) in: | ||
Accounts payable | 602,719 | (1,140,552) |
Accrued expenses and other liabilities | (1,337,155) | (401,277) |
Operating lease liability | (67,155) | 0 |
Net cash used in operating activities | (7,962,755) | (6,081,450) |
Investing Activities: | ||
Purchases of property and equipment | (5,633) | 0 |
Purchases of investments | (9,944,974) | (31,847,693) |
Proceeds from sales/maturities of investments | 20,346,326 | 0 |
Net cash provided by (used in) investing activities | 10,395,719 | (31,847,693) |
Financing Activities: | ||
Proceeds from exercise of stock options | 89,350 | 33,032 |
Net cash provided by (used in) financing activities | 89,350 | 33,032 |
Net increase (decrease) in cash and cash equivalents | 2,522,314 | (37,896,111) |
Cash and cash equivalents - beginning of period | 16,559,400 | 57,496,702 |
Cash and cash equivalents - end of period | 19,081,714 | 19,600,591 |
Non-cash investing and financing activities: | ||
Unrealized gain (loss) on available-for-sale securities | $ 13,560 | $ (21,826) |
Organization and Description of
Organization and Description of Business | 3 Months Ended |
Mar. 31, 2019 | |
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 innovating 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), Congenital Myasthenic Syndromes (CMS), and Spinal Muscular Atrophy (SMA) Type 3. On November 28, 2018, the U.S. Food and Drug Administration, or FDA, granted approval of Firdapse® for the treatment of adults with LEMS. On January 15, 2019, the Company launched its first product, Firdapse®, in the United States for the treatment of adults with LEMS. 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 has incurred operating losses in each period from inception through March 31, 2019. The Company has been able to fund its cash needs to date through several public and private offerings of its securities and revenues from its product sales. See Note 12. 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. |
Basis of Presentation and Signi
Basis of Presentation and Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2019 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Significant Accounting Policies | 2. Basis of Presentation and Significant Accounting Policies. a. INTERIM FINANCIAL STATEMENTS . The accompanying unaudited interim consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (U.S. GAAP), and pursuant to the rules and regulations of the Securities and Exchange Commission (SEC) for reporting of interim financial information. Pursuant to such rules and regulations, certain information and note disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been omitted. The consolidated balance sheet as of December 31, 2018 included in this Form 10-Q was derived from the audited financial statements and does not include all disclosures required by U.S. GAAP. 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, 2018 included in the 2018 Annual Report on Form 10-K filed by the Company with the SEC. The results of operations for the three months ended March 31, 2019 are not necessarily indicative of the results to be expected for any future period or for the full 2019 fiscal year. b. PRINCIPLES OF CONSOLIDATION . The consolidated financial statements include the Company’s accounts and those of its wholly-owned subsidiary Catalyst Pharmaceuticals Ireland, Ltd. (“Catalyst Ireland”). All intercompany accounts and transactions have been eliminated in consolidation. Catalyst Ireland was organized in 2017. c. USE OF ESTIMATES. The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. d. CASH AND CASH EQUIVALENTS. The Company considers all highly liquid instruments purchased with an original maturity of three months e. INVESTMENTS . The Company invests in high credit-quality funds in order to obtain higher yields on its cash available for investments. At March 31, 2019 and December 31, 2018, investments consisted of a short-term bond fund and U.S. Treasuries. Such investments are not insured by the Federal Deposit Insurance Corporation. Short-Term Bond Fund The short-term bond fund is classified in trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognizes realized gains and losses and unrealized gains and losses to earnings. At March 31, 2019 and December 31, 2018, the only investment classified as trading securities was the short-term bond fund. Realized losses on trading securities during the three months ended March 31, 2019 were $4,980. There were no sales on trading securities for the three months ended March 31, 2018. Unrealized gain (loss) on trading securities was $52,741 and ($58,861), respectively, for the three months ended March 31, 2019 and March 31, 2018 , 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 securities. The Company classifies available-for-sale securities with stated maturities of greater than three months and less than one year from the date of purchase as short-term investments. Available-for-sale securities with stated maturities greater than one year are classified as non-current investments in the accompanying consolidated balance sheets. The Company records available-for-sale securities at fair value with unrealized gains and losses reported in accumulated other comprehensive income (loss) in stockholders’ equity. Realized gains and losses are included in other income, net and are derived using the specific identification method for determining the cost of securities sold. Interest income is recognized when earned and is included in other income, net in the consolidated statements of operations. The Company recognizes a charge when the declines in the fair value below the amortized cost basis of its available-for-sale securities are judged to be other-than-temporary. The Company considers various factors in determining whether to recognize an other-than-temporary charge, including whether the Company intends to sell the security or whether it is more likely than not that the Company would be required to sell the security before recovery of the amortized cost basis. The Company has not recorded any other-than-temporary impairment charges on its available-for-sale securities. See Note 3. f. ACCOUNTS RECEIVABLE, NET. Accounts receivable are recorded net of customer allowance for distribution fees, trade discounts, prompt payment discounts, chargebacks and doubtful accounts. Allowances for distribution fees, prompt payment discounts and chargebacks are based on contractual terms. The Company estimates the allowance for doubtful accounts based on existing contractual payment terms, actual payment patterns of its Customer and individual Customer circumstances. At March 31, 2019, the Company determined that an allowance for doubtful accounts was not required. No accounts were written off during the periods presented. g. INVENTORY. Inventories are stated at the lower of cost or net realizable value with cost determined under the first-in-first-out (FIFO) cost method. Inventories consist of raw materials and supplies, work in process and finished goods. Costs to be capitalized as inventories include third party manufacturing costs, associated compensation related costs of personnel indirectly involved in the manufacturing process and other overhead costs such as ancillary supplies. The Company began capitalizing inventories post FDA approval of Firdapse® on November 28, 2018 as the related costs were expected to be recoverable through the commercialization of the product. Costs incurred prior to the FDA approval of Firdapse® had been recorded as research and development expenses in the consolidated statements of operations. If information becomes available that suggests that inventories may not be realizable, the Company may be required to expense a portion or all of the previously capitalized inventories. The costs of inventories during the quarter ended March 31, 2019 consisted mainly of packaging and labeling costs, indirect costs including compensation cost of personnel and supplies. As of March 31, 2019, inventory was approximately $97,000. As of December 31, 2018, inventory was approximately $56,000, and consisted mainly of packaging and labeling costs, indirect costs including compensation cost of personnel and supplies. Products that have been approved by the FDA or other regulatory authorities, such as Firdapse®, are also used in clinical programs to assess the safety and efficacy of the products for usage in treating diseases that have not been approved by the FDA or other regulatory authorities. The form of Firdapse® utilized for both commercial and clinical programs is identical and, as a result, the inventory has an “alternative future use” as defined in authoritative guidance. Raw materials and purchased drug product associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes and, therefore, does not have an “alternative future use”. 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 . Prepaid expenses and other current assets consist primarily of prepaid research fees, prepaid commercialization expenses, prepaid insurance and prepaid subscription fees. Prepaid research fees consist of advances for the Company’s product development activities, including drug manufacturing, contracts for pre-clinical studies, clinical trials and studies, regulatory affairs and consulting. Such advances are recorded as expense as the related goods are received or the related services are performed. i. FAIR VALUE OF FINANCIAL INSTRUMENTS. The Company’s financial instruments consist of cash and cash equivalents, short-term investments, accounts receivable, accounts payables and accrued expenses and other liabilities. At March 31, 2019 and December 31, 2018, the fair value of these instruments approximated their carrying value. j. FAIR VALUE MEASUREMENTS. Current Financial Accounting Standards Board (FASB) fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity’s own assumptions that it believes market participants would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). 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 March 31, 2019 Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Cash and cash equivalents: Money market funds $ 15,137,632 $ 15,137,632 $ — $ — Short-term investments: Short-term bond fund $ 16,571,264 $ 16,571,264 $ — $ — U.S. Treasuries $ 9,979,800 $ — $ 9,979,800 $ — Investments: U.S. Treasuries $ 4,991,600 $ — $ 4,991,600 $ — Balances as of December 31, 2018 Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Cash and cash equivalents: Money market funds $ 14,462,087 $ 14,462,087 $ — $ — Short-term investments: Short-term bond fund $ 26,541,349 $ 26,541,349 $ — $ — U.S. Treasuries $ 10,380,864 $ — $ 10,380,864 $ — Investments: U.S. Treasuries $ 5,008,243 $ — $ 5,008,243 $ — k. OPERATING LEASES. Operating lease ROU assets and operating lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term at commencement date. As the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of future payments. The operating lease ROU asset also includes any lease payments made and excludes lease incentives and initial direct costs incurred. The Company’s lease terms do not include options to extend or terminate the lease as it is not reasonably certain that it will exercise these options. Lease expense for minimum lease payments is recognized on a straight-line basis over the lease term. The Company has lease agreements with lease and non-lease components, which are generally accounted for separately. Refer to Note 2.r. for discussion of adoption method . l. REVENUE RECOGNITION. To determine revenue recognition for arrangements that are within the scope of 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 Sales, Net The Company also generates 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 Sales, 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 10 and 60 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, 2019. As of March 31, 2019 all of the Company’s sales are to its distributor. Reserves for Variable Consideration: 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, 2019 and, therefore, the transaction price was not reduced further during the three months ended March 31, 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 Assistance Program: Product Returns: Provider Chargebacks and Discounts: Government Rebates: Bridge and Patient Assistance Programs: 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. For the three months ended March 31, 2019 and March 31, 2018, no revenue was recognized. Refer to Note 8, Collaborative Arrangement, for further discussion on the Company’s collaborative arrangement. m. CONCENTRATION OF RISK. The Company relies exclusively on third parties to formulate and manufacture Firdapse® and its drug candidates. The commercialization of Firdapse® and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and the commercialization of Firdapse®. If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of drugs. n. ROYALTIES. o. STOCK-BASED COMPENSATION. p. COMPREHENSIVE INCOME (LOSS). q. NET LOSS PER SHARE. March 31, 2019 2018 Options to purchase common stock 10,604,500 6,902,500 Potential equivalent common stock excluded 10,604,500 6,902,500 Potentially dilutive options to purchase common stock as of March 31, 2019 and 2018 had exercise prices ranging from $ 0.79 4.64 r. RECENTLY ISSUED ACCOUNTING STANDARDS. Leases (Topic 842) November 30, 2022 In June 2018, the FASB issued ASU No. 2018-07, Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting that largely aligns the accounting for share-based payment awards issued to employees and nonemployees. Under this ASU, most of the guidance on such payments to nonemployees would be aligned with the requirements for share-based payments granted to employees. ASU 2018-07 is effective for all entities for annual reporting periods beginning after December 15, 2018, including interim reporting periods within each annual reporting period, with early adoption permitted. The Company has adopted this standard as of January 1, 2019. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments s. RECLASSIFICATIONS. Certain prior year amounts in the consolidated financial statements have been reclassified to conform to the current year presentation. |
Investments
Investments | 3 Months Ended |
Mar. 31, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | 3. Investments. Available-for-sale investments by security type were as follows: Amortized cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value At March 31, 2019: U.S. Treasuries - ST $ 9,979,760 $ 40 $ — $ 9,979,800 U.S. Treasuries - LT 4,998,328 — (6,728 ) 4,991,600 Total $ 14,978,088 $ 40 $ (6,728 ) $ 14,971,400 At December 31, 2018: U.S. Treasuries - ST $ 10,382,699 $ — $ (1,835 ) $ 10,380,864 U.S. Treasuries - LT 5,026,656 — (18,413 ) 5,008,243 Total $ 15,409,355 $ — $ (20,248 ) $ 15,389,107 In accordance with FASB ASC Topic 320, “Investments – Debt and Equity Securities”, or ASC 320, the Company has classified its U.S. Treasuries as available-for-sale securities with secondary or resale markets, and, as such, they are reported at fair value with unrealized gains and losses included in comprehensive loss in stockholders’ equity and realized gain and losses, included in other income, net. There were no realized gains or losses from available-for-sale securities for the three months ended March 31, 2019 or 2018. Certain U.S. Treasuries at March 31, 2019 had fair values greater or lower than their amortized costs and, therefore, contained unrealized gains (losses). Given that the Company has no intent to sell the U.S. Treasuries until a recovery of the fair value, which may be at maturity, and there are no current requirements to sell any of these securities, the Company did not consider these investments to be other-than-temporarily impaired as of March 31, 2019. The Company anticipates full recovery of amortized costs with respect to these investments at maturity. The duration of time the U.S. Treasuries have been in a continuous unrealized loss position as of March 31, 2019 was more than 12 months. The estimated fair values of available-for-sale securities at March 31, 2019, by contractual maturity, are summarized as follows: March 31, 2019 Due in one year or less $ 9,979,800 Due after one year but within two years 4,991,600 $ 14,971,400 |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 3 Months Ended |
Mar. 31, 2019 | |
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, 2019 December 31, 2018 Prepaid research fees $ 240,377 $ 358,209 Prepaid insurance 616,761 800,261 Prepaid commercialization fees 430,573 17,030 Prepaid subscription fees 287,760 170,552 Other 305,795 303,729 Total prepaid expenses and other current assets $ 1,881,266 $ 1,649,781 |
Operating Leases
Operating Leases | 3 Months Ended |
Mar. 31, 2019 | |
Disclosure of Operating Leases [Abstract] | |
Operating Leases | 5. Operating Leases. The Company has operating lease agreements for its corporate office. The leases have free and escalating payment provisions. The leases have remaining lease terms of approximately 3.7 years, and 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, 2019 Operating lease cost $ 74,079 Supplemental cash flow information related to leases was as follows: March 31, 2019 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 81,616 Right-of-use assets obtained in exchange for lease obligations: Operating leases $ 5,305 Supplemental balance sheet information related to leases was as follows: March 31, 2019 Operating lease right-of-use assets $ 1,074,020 Other current liabilities $ 282,605 Operating lease liabilities 875,098 Total operating lease liabilities $ 1,157,703 Weighted Average Remaining Lease Term 3.7 years Weighted Average Discount Rate 4.81 % Payments of lease liabilities as of March 31, 2019 were as follows: 2019 (remaining nine months) $ 248,108 2020 339,605 2021 349,788 2022 329,662 Total lease payments 1,267,163 Less imputed interest (109,460 ) Total $ 1,157,703 |
Property and Equipment, net
Property and Equipment, net | 3 Months Ended |
Mar. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | 6. Property and Equipment, Net. Property and equipment, net consists of the following: March 31, 2019 December 31, 2018 Computer equipment $ 52,704 $ 52,704 Furniture and equipment 218,084 212,451 Leasehold improvements — 177,417 270,788 442,572 Less: Accumulated depreciation (130,468 ) (197,147 ) Total property and equipment, net $ 140,320 $ 245,425 Depreciation expense was $13,748 and $8,015, respectively, for the three-month periods ended March 31, 2019 and 2018. |
Accrued Expenses and Other Liab
Accrued Expenses and Other Liabilities | 3 Months Ended |
Mar. 31, 2019 | |
Payables and Accruals [Abstract] | |
Accrued Expenses and Other Liabilities | 7. Accrued Expenses and Other Liabilities. Accrued expenses and other liabilities consist of the following: March 31, 2019 December 31, 2018 Accrued preclinical and clinical trial expenses $ 943,742 $ 821,633 Accrued professional fees 1,651,181 1,311,061 Accrued compensation and benefits 764,362 1,941,449 Accrued license fees 1,573,642 3,000,000 Lease liability 282,605 — Accrued variable consideration 704,834 — Deferred rent and lease incentive — 33,408 Other 165,663 66,436 Current accrued expenses and other liabilities 6,086,029 7,173,987 Lease liability - non-current 875,098 — Deferred rent and lease incentive – non-current — 154,799 Non-current accrued expenses and other liabilities 875,098 154,799 Total accrued expenses and other liabilities $ 6,961,127 $ 7,328,786 |
Collaborative Arrangement
Collaborative Arrangement | 3 Months Ended |
Mar. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaborative Arrangement | 8. Collaborative Arrangement. In December 2018, the Company entered into a collaboration and license agreement (collaboration) with Endo International plc’s subsidiary, Endo Ventures Limited (EVL), for the further development and commercialization of generic Sabril® (vigabatrin) tablets through Endo’s U.S. Generic Pharmaceuticals segment, doing business as Par Pharmaceutical. EVL has assumed 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® tablets. Under the terms of the Collaboration, the Company has received an up-front payment, and will receive milestone payments based on achievement of regulatory approvals, and a sharing of defined net profits upon commercialization from EVL consisting of a mid-double digit percent of net sales of generic Sabril®. Unless terminated earlier in accordance with its terms, the collaboration continues in effect until the date that is ten years following the commercial launch. For the year ended December 31, 2018, a $500,000 upfront license fee was recognized. For the three-month period ending March 31, 2019, no collaborative arrangement revenue was recognized. |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2019 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 9. Commitments and Contingencies. In 2018, the Company became aware that certain patents granted to Northwestern in 2018 (which patents have been licensed by Northwestern to a third-party) for a new GABA aminotransferase inhibitor were derived from CPP-115. As a result, On November 5, 2018, Northwestern advised the Company that in its view, Northwestern has a right to terminate the license agreement with the Company because the Company has allegedly breached the license agreement by failing to pay certain milestones and by allegedly failing to use commercially reasonable efforts to develop and commercialize any products. Northwestern has also advised the Company that, in its view, the Company has engaged in wrongful conduct and communications with the third party that licensed the new patents from Northwestern, and that such communications have damaged Northwestern’s relationship with that third party. The Company disputes Northwestern’s allegations and is vigorously defending itself against claims that Northwestern has brought against it in the arbitration proceeding. There can be no assurance as to the outcome of this matter. |
Agreements
Agreements | 3 Months Ended |
Mar. 31, 2019 | |
Text Block [Abstract] | |
Agreements | 10. Agreements. a. LICENSE AGREEMENT WITH BIOMARIN (FIRDAPSE®) b. AGREEMENTS FOR DRUG DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES. |
Income Taxes
Income Taxes | 3 Months Ended |
Mar. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 11. 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 2015. 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’s net deferred tax asset has a 100% valuation allowance at March 31, 2019 and December 31, 2018 as the Company believes that it is more likely than not that the deferred tax asset will not be realized. |
Stockholders' Equity
Stockholders' Equity | 3 Months Ended |
Mar. 31, 2019 | |
Equity [Abstract] | |
Stockholders' Equity | 12. Stockholders’ Equity. 2016 Shelf Registration Statement On December 23, 2016, the Company filed a shelf Registration Statement on Form S-3 (the 2016 Shelf Registration Statement) with the SEC to sell up to approximately $33.8 million of common stock. The 2016 Shelf Registration Statement (file No. 333-215315) was declared effective by the SEC on January 9, 2017. No sales have been conducted to date under the 2016 Shelf Registration Statement. 2017 Shelf Registration Statement On July 12, 2017, the Company filed a universal shelf Registration Statement on Form S-3 (the 2017 Shelf Registration Statement) with the SEC to sell up to $150 million of common stock, preferred stock, warrants to purchase common stock, or debt securities (including debt securities that may be convertible or exchangeable for common stock or other securities), which securities may be offered separately or together in units or multiple series. The 2017 Shelf Registration Statement (file No. 333-219259) was declared effective by the SEC on July 26, 2017. 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, 2019, 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, 2019 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock Compensation | 13. Stock Compensation. For the three-month periods ended March 31, 2019 and 2018, the Company recorded stock-based compensation expense as follows: Three months ended March 31, 2019 2018 Research and development $ 287,721 $ 294,315 Selling, general and administrative 645,690 677,025 Total stock-based compensation $ 933,411 $ 971,340 Stock Options As of March 31, 2019, there were outstanding stock options to purchase 10,604,500 shares of common stock, of which stock options to purchase 5,259,156 shares of common stock were exercisable as of March 31, 2019. During the three-month periods ended March 31, 2019 and 2018, the Company granted seven-year term options to purchase an aggregate of 207,000 and 1,772,500 shares, respectively, of the Company’s common stock to employees and directors. The Company recorded stock-based compensation related to stock options totaling $933,411 and $971,340, respectively, during the three-month periods ended March 31, 2019 and 2018. During the three-month periods ended March 31, 2019 and 2018, respectively, 984,164 and 744,998 options vested. During the three-month periods ended March 31, 2019 and 2018, options to purchase 65,000 shares and 36,666 shares, respectively, of the Company’s common stock were exercised, with proceeds of $89,350 and $33,032, respectively, to the Company. As of March 31, 2019, there was approximately $8,071,744 of unrecognized compensation expense related to non-vested stock option awards granted under the 2014 and 2018 Stock Incentive Plans. The cost is expected to be recognized over a weighted average period of approximately 2.38 years. |
Subsequent Events
Subsequent Events | 3 Months Ended |
Mar. 31, 2019 | |
Subsequent Events [Abstract] | |
Subsequent Events | 14. Subsequent Events. The Company became aware earlier this week from press reports that the FDA has approved an NDA for Jacobus Pharmaceuticals for their version of 3,4-DAP for pediatric LEMS patients. The Company is in the early stages of assessing both the impact of the FDA’s recent action on its business and its available options, and there can be no assurance as to the ultimate impact of the FDA’s recent action on the Company’s business and future results of operations. |
Basis of Presentation and Sig_2
Basis of Presentation and Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2019 | |
Accounting Policies [Abstract] | |
INTERIM FINANCIAL STATEMENTS | a. INTERIM FINANCIAL STATEMENTS . The accompanying unaudited interim consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (U.S. GAAP), and pursuant to the rules and regulations of the Securities and Exchange Commission (SEC) for reporting of interim financial information. Pursuant to such rules and regulations, certain information and note disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been omitted. The consolidated balance sheet as of December 31, 2018 included in this Form 10-Q was derived from the audited financial statements and does not include all disclosures required by U.S. GAAP. 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, 2018 included in the 2018 Annual Report on Form 10-K filed by the Company with the SEC. The results of operations for the three months ended March 31, 2019 are not necessarily indicative of the results to be expected for any future period or for the full 2019 fiscal year. |
PRINCIPLES OF CONSOLIDATION. | b. PRINCIPLES OF CONSOLIDATION . The consolidated financial statements include the Company’s accounts and those of its wholly-owned subsidiary Catalyst Pharmaceuticals Ireland, Ltd. (“Catalyst Ireland”). All intercompany accounts and transactions have been eliminated in consolidation. Catalyst Ireland was organized in 2017. |
USE OF ESTIMATES | c. USE OF ESTIMATES. The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. |
CASH AND CASH EQUIVALENTS | d. CASH AND CASH EQUIVALENTS. The Company considers all highly liquid instruments purchased with an original maturity of three months |
INVESTMENTS | e. INVESTMENTS . The Company invests in high credit-quality funds in order to obtain higher yields on its cash available for investments. At March 31, 2019 and December 31, 2018, investments consisted of a short-term bond fund and U.S. Treasuries. Such investments are not insured by the Federal Deposit Insurance Corporation. Short-Term Bond Fund The short-term bond fund is classified in trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognizes realized gains and losses and unrealized gains and losses to earnings. At March 31, 2019 and December 31, 2018, the only investment classified as trading securities was the short-term bond fund. Realized losses on trading securities during the three months ended March 31, 2019 were $4,980. There were no sales on trading securities for the three months ended March 31, 2018. Unrealized gain (loss) on trading securities was $52,741 and ($58,861), respectively, for the three months ended March 31, 2019 and March 31, 2018 , 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 securities. The Company classifies available-for-sale securities with stated maturities of greater than three months and less than one year from the date of purchase as short-term investments. Available-for-sale securities with stated maturities greater than one year are classified as non-current investments in the accompanying consolidated balance sheets. The Company records available-for-sale securities at fair value with unrealized gains and losses reported in accumulated other comprehensive income (loss) in stockholders’ equity. Realized gains and losses are included in other income, net and are derived using the specific identification method for determining the cost of securities sold. Interest income is recognized when earned and is included in other income, net in the consolidated statements of operations. The Company recognizes a charge when the declines in the fair value below the amortized cost basis of its available-for-sale securities are judged to be other-than-temporary. The Company considers various factors in determining whether to recognize an other-than-temporary charge, including whether the Company intends to sell the security or whether it is more likely than not that the Company would be required to sell the security before recovery of the amortized cost basis. The Company has not recorded any other-than-temporary impairment charges on its available-for-sale securities. See Note 3. |
ACCOUNTS RECEIVABLE, NET | f. ACCOUNTS RECEIVABLE, NET. Accounts receivable are recorded net of customer allowance for distribution fees, trade discounts, prompt payment discounts, chargebacks and doubtful accounts. Allowances for distribution fees, prompt payment discounts and chargebacks are based on contractual terms. The Company estimates the allowance for doubtful accounts based on existing contractual payment terms, actual payment patterns of its Customer and individual Customer circumstances. At March 31, 2019, the Company determined that an allowance for doubtful accounts was not required. No accounts were written off during the periods presented. |
INVENTORY | g. INVENTORY. Inventories are stated at the lower of cost or net realizable value with cost determined under the first-in-first-out (FIFO) cost method. Inventories consist of raw materials and supplies, work in process and finished goods. Costs to be capitalized as inventories include third party manufacturing costs, associated compensation related costs of personnel indirectly involved in the manufacturing process and other overhead costs such as ancillary supplies. The Company began capitalizing inventories post FDA approval of Firdapse® on November 28, 2018 as the related costs were expected to be recoverable through the commercialization of the product. Costs incurred prior to the FDA approval of Firdapse® had been recorded as research and development expenses in the consolidated statements of operations. If information becomes available that suggests that inventories may not be realizable, the Company may be required to expense a portion or all of the previously capitalized inventories. The costs of inventories during the quarter ended March 31, 2019 consisted mainly of packaging and labeling costs, indirect costs including compensation cost of personnel and supplies. As of March 31, 2019, inventory was approximately $97,000. As of December 31, 2018, inventory was approximately $56,000, and consisted mainly of packaging and labeling costs, indirect costs including compensation cost of personnel and supplies. Products that have been approved by the FDA or other regulatory authorities, such as Firdapse®, are also used in clinical programs to assess the safety and efficacy of the products for usage in treating diseases that have not been approved by the FDA or other regulatory authorities. The form of Firdapse® utilized for both commercial and clinical programs is identical and, as a result, the inventory has an “alternative future use” as defined in authoritative guidance. Raw materials and purchased drug product associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes and, therefore, does not have an “alternative future use”. 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 . Prepaid expenses and other current assets consist primarily of prepaid research fees, prepaid commercialization expenses, prepaid insurance and prepaid subscription fees. Prepaid research fees consist of advances for the Company’s product development activities, including drug manufacturing, contracts for pre-clinical studies, clinical trials and studies, regulatory affairs and consulting. Such advances are recorded as expense as the related goods are received or the related services are performed. |
FAIR VALUE OF FINANCIAL INSTRUMENTS | i. FAIR VALUE OF FINANCIAL INSTRUMENTS. The Company’s financial instruments consist of cash and cash equivalents, short-term investments, accounts receivable, accounts payables and accrued expenses and other liabilities. At March 31, 2019 and December 31, 2018, the fair value of these instruments approximated their carrying value. |
FAIR VALUE MEASUREMENTS | j. FAIR VALUE MEASUREMENTS. Current Financial Accounting Standards Board (FASB) fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity’s own assumptions that it believes market participants would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). 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 March 31, 2019 Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Cash and cash equivalents: Money market funds $ 15,137,632 $ 15,137,632 $ — $ — Short-term investments: Short-term bond fund $ 16,571,264 $ 16,571,264 $ — $ — U.S. Treasuries $ 9,979,800 $ — $ 9,979,800 $ — Investments: U.S. Treasuries $ 4,991,600 $ — $ 4,991,600 $ — Balances as of December 31, 2018 Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Cash and cash equivalents: Money market funds $ 14,462,087 $ 14,462,087 $ — $ — Short-term investments: Short-term bond fund $ 26,541,349 $ 26,541,349 $ — $ — U.S. Treasuries $ 10,380,864 $ — $ 10,380,864 $ — Investments: U.S. Treasuries $ 5,008,243 $ — $ 5,008,243 $ — |
OPERATING LEASES | k. OPERATING LEASES. Operating lease ROU assets and operating lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term at commencement date. As the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of future payments. The operating lease ROU asset also includes any lease payments made and excludes lease incentives and initial direct costs incurred. The Company’s lease terms do not include options to extend or terminate the lease as it is not reasonably certain that it will exercise these options. Lease expense for minimum lease payments is recognized on a straight-line basis over the lease term. The Company has lease agreements with lease and non-lease components, which are generally accounted for separately. Refer to Note 2.r. for discussion of adoption method . |
REVENUE RECOGNITION | l. REVENUE RECOGNITION. To determine revenue recognition for arrangements that are within the scope of 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 Sales, Net The Company also generates 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 Sales, 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 10 and 60 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, 2019. As of March 31, 2019 all of the Company’s sales are to its distributor. Reserves for Variable Consideration: 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, 2019 and, therefore, the transaction price was not reduced further during the three months ended March 31, 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 Assistance Program: Product Returns: Provider Chargebacks and Discounts: Government Rebates: Bridge and Patient Assistance Programs: 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. For the three months ended March 31, 2019 and March 31, 2018, no revenue was recognized. Refer to Note 8, Collaborative Arrangement, for further discussion on the Company’s collaborative arrangement. |
CONCENTRATION OF CREDIT RISK | m. CONCENTRATION OF RISK. The Company relies exclusively on third parties to formulate and manufacture Firdapse® and its drug candidates. The commercialization of Firdapse® and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and the commercialization of Firdapse®. If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of drugs. |
ROYALTIES | n. ROYALTIES. |
STOCK-BASED COMPENSATION | o. STOCK-BASED COMPENSATION. |
COMPREHENSIVE INCOME (LOSS) | p. COMPREHENSIVE INCOME (LOSS). |
NET LOSS PER SHARE | q. NET LOSS PER SHARE. March 31, 2019 2018 Options to purchase common stock 10,604,500 6,902,500 Potential equivalent common stock excluded 10,604,500 6,902,500 Potentially dilutive options to purchase common stock as of March 31, 2019 and 2018 had exercise prices ranging from $ 0.79 4.64 |
RECENTLY ISSUED ACCOUNTING STANDARDS | r. RECENTLY ISSUED ACCOUNTING STANDARDS. Leases (Topic 842) November 30, 2022 In June 2018, the FASB issued ASU No. 2018-07, Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting that largely aligns the accounting for share-based payment awards issued to employees and nonemployees. Under this ASU, most of the guidance on such payments to nonemployees would be aligned with the requirements for share-based payments granted to employees. ASU 2018-07 is effective for all entities for annual reporting periods beginning after December 15, 2018, including interim reporting periods within each annual reporting period, with early adoption permitted. The Company has adopted this standard as of January 1, 2019. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments |
RECLASSIFICATIONS | s. RECLASSIFICATIONS. Certain prior year amounts in the consolidated financial statements have been reclassified to conform to the current year presentation. |
Basis of Presentation and Sig_3
Basis of Presentation and Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2019 | |
Accounting Policies [Abstract] | |
Fair Value Measurement Specific to Assets or Liability | The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using Balances as of March 31, 2019 Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Cash and cash equivalents: Money market funds $ 15,137,632 $ 15,137,632 $ — $ — Short-term investments: Short-term bond fund $ 16,571,264 $ 16,571,264 $ — $ — U.S. Treasuries $ 9,979,800 $ — $ 9,979,800 $ — Investments: U.S. Treasuries $ 4,991,600 $ — $ 4,991,600 $ — Balances as of December 31, 2018 Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Cash and cash equivalents: Money market funds $ 14,462,087 $ 14,462,087 $ — $ — Short-term investments: Short-term bond fund $ 26,541,349 $ 26,541,349 $ — $ — U.S. Treasuries $ 10,380,864 $ — $ 10,380,864 $ — Investments: U.S. Treasuries $ 5,008,243 $ — $ 5,008,243 $ — |
Potential Shares Excluded from Determination of Basic and Diluted Net Loss Per Share | March 31, 2019 2018 Options to purchase common stock 10,604,500 6,902,500 Potential equivalent common stock excluded 10,604,500 6,902,500 |
Investments (Tables)
Investments (Tables) | 3 Months Ended |
Mar. 31, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available-for-Sale Investments by Security type | Available-for-sale investments by security type were as follows: Amortized cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value At March 31, 2019: U.S. Treasuries - ST $ 9,979,760 $ 40 $ — $ 9,979,800 U.S. Treasuries - LT 4,998,328 — (6,728 ) 4,991,600 Total $ 14,978,088 $ 40 $ (6,728 ) $ 14,971,400 At December 31, 2018: U.S. Treasuries - ST $ 10,382,699 $ — $ (1,835 ) $ 10,380,864 U.S. Treasuries - LT 5,026,656 — (18,413 ) 5,008,243 Total $ 15,409,355 $ — $ (20,248 ) $ 15,389,107 |
Estimated Fair Values of Available for Sale Securities | The estimated fair values of available-for-sale securities at March 31, 2019, by contractual maturity, are summarized as follows: March 31, 2019 Due in one year or less $ 9,979,800 Due after one year but within two years 4,991,600 $ 14,971,400 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 3 Months Ended |
Mar. 31, 2019 | |
Text Block [Abstract] | |
Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consist of the following: March 31, 2019 December 31, 2018 Prepaid research fees $ 240,377 $ 358,209 Prepaid insurance 616,761 800,261 Prepaid commercialization fees 430,573 17,030 Prepaid subscription fees 287,760 170,552 Other 305,795 303,729 Total prepaid expenses and other current assets $ 1,881,266 $ 1,649,781 |
Operating Leases (Tables)
Operating Leases (Tables) | 3 Months Ended |
Mar. 31, 2019 | |
Disclosure of Operating Leases [Abstract] | |
Lease, Cost | The components of lease expense were as follows: March 31, 2019 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, 2019 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 81,616 Right-of-use assets obtained in exchange for lease obligations: Operating leases $ 5,305 |
Schedule of Supplemental Balance Sheet related To Lease | Supplemental balance sheet information related to leases was as follows: March 31, 2019 Operating lease right-of-use assets $ 1,074,020 Other current liabilities $ 282,605 Operating lease liabilities 875,098 Total operating lease liabilities $ 1,157,703 Weighted Average Remaining Lease Term 3.7 years Weighted Average Discount Rate 4.81 % |
Lessee, Operating Lease, Liability, Maturity | Payments of lease liabilities as of March 31, 2019 were as follows: 2019 (remaining nine months) $ 248,108 2020 339,605 2021 349,788 2022 329,662 Total lease payments 1,267,163 Less imputed interest (109,460 ) Total $ 1,157,703 |
Property and Equipment, net (Ta
Property and Equipment, net (Tables) | 3 Months Ended |
Mar. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | Property and equipment, net consists of the following: March 31, 2019 December 31, 2018 Computer equipment $ 52,704 $ 52,704 Furniture and equipment 218,084 212,451 Leasehold improvements — 177,417 270,788 442,572 Less: Accumulated depreciation (130,468 ) (197,147 ) Total property and equipment, net $ 140,320 $ 245,425 |
Accrued Expenses and Other Li_2
Accrued Expenses and Other Liabilities (Tables) | 3 Months Ended |
Mar. 31, 2018 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Liabilities | Accrued expenses and other liabilities consist of the following: March 31, 2019 December 31, 2018 Accrued preclinical and clinical trial expenses $ 943,742 $ 821,633 Accrued professional fees 1,651,181 1,311,061 Accrued compensation and benefits 764,362 1,941,449 Accrued license fees 1,573,642 3,000,000 Lease liability 282,605 — Accrued variable consideration 704,834 — Deferred rent and lease incentive — 33,408 Other 165,663 66,436 Current accrued expenses and other liabilities 6,086,029 7,173,987 Lease liability - non-current 875,098 — Deferred rent and lease incentive – non-current — 154,799 Non-current accrued expenses and other liabilities 875,098 154,799 Total accrued expenses and other liabilities $ 6,961,127 $ 7,328,786 |
Stock Compensation (Tables)
Stock Compensation (Tables) | 3 Months Ended |
Mar. 31, 2019 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock-Based Compensation Expense | For the three-month periods ended March 31, 2019 and 2018, the Company recorded stock-based compensation expense as follows: Three months ended March 31, 2019 2018 Research and development $ 287,721 $ 294,315 Selling, general and administrative 645,690 677,025 Total stock-based compensation $ 933,411 $ 971,340 |
Basis of Presentation and Sig_4
Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($) | 3 Months Ended | ||
Mar. 31, 2019 | Dec. 31, 2018 | Mar. 31, 2018 | |
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Maximum maturity period of cash and cash equivalent | three months | ||
Inventory | $ 96,587 | $ 56,012 | |
Upfront license fees | $ 12,448,438 | $ 0 | |
Stock option exercise price range, Minimum | $ 0.79 | $ 0.79 | |
Stock option exercise price range, Maximum | $ 4.64 | $ 4.64 | |
Lease Expiration Date | Nov. 30, 2022 | ||
Additional assets | $ 1,000,000 | ||
Corresponding liability | 1,500,000 | ||
Trading Securities, Realized Gain (Loss) | 4,980 | ||
Trading Securities Sales | 0 | ||
Collaborative Arrangement, Product [Member] | |||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Upfront license fees | $ 500,000 | ||
Other Income, Net [Member] | |||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Unrealized gain(loss),trading securities | $ 52,741 | $ 58,861 | |
Minimum [Member] | |||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Minimum amortization period of compensation cost on straight line basis | 1 year | ||
Maximum [Member] | |||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||
Minimum amortization period of compensation cost on straight line basis | 5 years |
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, 2019 | Dec. 31, 2018 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments | $ 4,991,600 | $ 5,008,243 |
Short-Term Bond Fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 16,571,264 | 26,541,349 |
U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 9,979,800 | 10,380,864 |
Investments | 4,991,600 | 5,008,243 |
Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Money market funds | 15,137,632 | 14,462,087 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | Short-Term Bond Fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 16,571,264 | 26,541,349 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 0 | |
Investments | 0 | |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Money market funds | 15,137,632 | 14,462,087 |
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 | 0 | |
Significant Other Observable Inputs (Level 2) [Member] | U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 9,979,800 | 10,380,864 |
Investments | 4,991,600 | $ 5,008,243 |
Significant Other Observable Inputs (Level 2) [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Money market funds | 0 | |
Significant Unobservable Inputs (Level 3) [Member] | Short-Term Bond Fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 0 | |
Significant Unobservable Inputs (Level 3) [Member] | U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | ||
Investments | 0 | |
Significant Unobservable Inputs (Level 3) [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Money market funds | $ 0 |
Basis of Presentation and Sig_6
Basis of Presentation and Significant Accounting Policies - Potential Shares Excluded from Determination of Basic and Diluted Net Loss Per Share (Detail) - shares | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential equivalent common stock excluded | 10,604,500 | 6,902,500 |
Options to Purchase Common Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential equivalent common stock excluded | 10,604,500 | 6,902,500 |
Investment - Summary of Availab
Investment - Summary of Available-for-Sale Investments by Security type (Detail) - USD ($) | 3 Months Ended | |
Mar. 31, 2019 | Dec. 31, 2018 | |
Net Investment Income [Line Items] | ||
Amortized cost | $ 14,978,088 | $ 15,409,355 |
Gross Unrealized Gains | 40 | |
Gross Unrealized Losses | (6,728) | (20,248) |
Estimated Fair Value | 14,971,400 | 15,389,107 |
U.S. Treasuries - ST [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 9,979,760 | 10,382,699 |
Gross Unrealized Gains | 40 | |
Gross Unrealized Losses | (1,835) | |
Estimated Fair Value | 9,979,800 | 10,380,864 |
U.S. Treasuries - LT [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 4,998,328 | 5,026,656 |
Gross Unrealized Losses | (6,728) | (18,413) |
Estimated Fair Value | $ 4,991,600 | $ 5,008,243 |
Investments - Additional Inform
Investments - Additional Information (Detail) - USD ($) | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Investments, Debt and Equity Securities [Abstract] | ||
Realized gains losses from available for sale securities | $ 0 | $ 0 |
Investment - Estimated Fair Val
Investment - Estimated Fair Values of Available for Sale Securities (Detail) - USD ($) | Mar. 31, 2019 | Dec. 31, 2018 |
Investments, Debt and Equity Securities [Abstract] | ||
Due in one year or less | $ 9,979,800 | |
Due after one year but within two years | 4,991,600 | |
Estimated Fair Value | $ 14,971,400 | $ 15,389,107 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($) | Mar. 31, 2019 | Dec. 31, 2018 |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid research fees | $ 240,377 | $ 358,209 |
Prepaid insurance | 616,761 | 800,261 |
Prepaid commercialization fees | 430,573 | 17,030 |
Prepaid subscriptions fees | 287,760 | 170,552 |
Other | 305,795 | 303,729 |
Total prepaid expenses and other current assets | $ 1,881,266 | $ 1,649,781 |
Operating Leases - Operating Le
Operating Leases - Operating Leases (Detail) | 3 Months Ended |
Mar. 31, 2019USD ($) | |
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, 2019USD ($) | |
Cash paid for amounts included in the measurement of lease liabilities: | |
Operating cash flows from operating leases | $ 81,616 |
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, 2019 | Dec. 31, 2018 |
Disclosure of Operating Leases [Line Items] | ||
Operating lease right-of-use assets | $ 1,074,020 | $ 0 |
Other current liabilities | 282,605 | 0 |
Operating lease liabilities | 875,098 | $ 0 |
Total operating lease liabilities | $ 1,157,703 | |
Weighted Average Remaining Lease Term | 3 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, 2019USD ($) |
Disclosure of Operating Leases [Line Items] | |
2019 (remaining nine months) | $ 248,108 |
2020 | 339,605 |
2021 | 349,788 |
2022 | 329,662 |
Total lease payments | 1,267,163 |
Less imputed interest | (109,460) |
Total | $ 1,157,703 |
Operating Leases - Additional
Operating Leases - Additional Information (Detail) | 3 Months Ended |
Mar. 31, 2019 | |
Disclosure of Operating Leases [Line Items] | |
Operating Lease, Weighted Average Remaining Lease Term | 3 years 8 months 12 days |
Lessor, Operating Lease, Option to Extend | 1 year |
Lessor, Operating Lease, Option to Terminate | 1 year |
Property and Equipment, Net - P
Property and Equipment, Net - Property and Equipment, Net (Detail) - USD ($) | Mar. 31, 2019 | Dec. 31, 2018 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 270,788 | $ 442,572 |
Less: Accumulated depreciation | (130,468) | (197,147) |
Total property and equipment, net | 140,320 | 245,425 |
Computer Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 52,704 | 52,704 |
Furniture and Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 218,084 | 212,451 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 177,417 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Property, Plant and Equipment [Abstract] | ||
Depreciation expenses | $ 13,748 | $ 8,015 |
Accrued Expenses and Other Li_3
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($) | Mar. 31, 2019 | Dec. 31, 2018 |
Payables and Accruals [Abstract] | ||
Accrued preclinical and clinical trial expenses | $ 943,742 | $ 821,633 |
Accrued professional fees | 1,651,181 | 1,311,061 |
Accrued compensation and benefits | 764,362 | 1,941,449 |
Accrued license fees | 1,573,642 | 3,000,000 |
Lease liability | 282,605 | 0 |
Accrued variable consideration | 704,834 | 0 |
Deferred rent and lease incentive | 0 | 33,408 |
Other | 165,663 | 66,436 |
Current accrued expenses and other liabilities | 6,086,029 | 7,173,987 |
Lease liability - non-current | 875,098 | 0 |
Deferred rent and lease incentive - non-current | 0 | 154,799 |
Non-current accrued expenses and other liabilities | 0 | 154,799 |
Total accrued expenses and other liabilities | $ 6,961,127 | $ 7,328,786 |
Collaborative Arrangement - Add
Collaborative Arrangement - Additional Information (Detail) - USD ($) | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | Dec. 31, 2018 | |
Collaborative Arrangement [Line Items] | |||
Upfront license fee | $ 12,448,438 | $ 0 | |
Collaborative Arrangement [Member] | |||
Collaborative Arrangement [Line Items] | |||
Upfront license fee | $ 0 | $ 500,000 | |
Period of collaboration agreement | 10 years |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2018 | Mar. 31, 2019 | |
Commitments [Line Items] | ||
Accrued license fees | $ 3,000,000 | $ 1,573,642 |
Northwestern License Agreement [Member] | ||
Commitments [Line Items] | ||
License fee paid | 424,885 | |
Accrued license fees | $ 0 |
Agreements - Additional Informa
Agreements - Additional Information (Detail) - License Agreement with BioMarin [Member] $ in Millions | 3 Months Ended |
Mar. 31, 2019USD ($) | |
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) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2019 | Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | ||
Valuation allowance | 100.00% | 100.00% |
Stockholders' Equity (2016 Shel
Stockholders' Equity (2016 Shelf Registration Statement) - Additional Information (Detail) | Dec. 23, 2016USD ($) |
2016 Shelf Registration Statement [Member] | |
Stockholders' Equity [Line Items] | |
Maximum dollar amount of common stock to be issued under shelf registration statement | $ 33,800,000 |
Stockholders' Equity (2017 Shel
Stockholders' Equity (2017 Shelf Registration Statement) - Additional Information (Detail) - USD ($) | Nov. 28, 2017 | Mar. 31, 2019 | 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, 2019 | Mar. 31, 2018 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | $ 933,411 | $ 971,340 |
Research and Development [Member] | ||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | 287,721 | 294,315 |
Selling, General and Administrative [Member] | ||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Stock-based compensation | $ 645,690 | $ 677,025 |
Stock Compensation - Additional
Stock Compensation - Additional Information (Detail) - USD ($) | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Proceeds from exercise of stock options | $ 89,350 | $ 33,032 |
Options to Purchase Common Stock [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock shares reserved for issuance under the Plan | 10,604,500 | |
Number of stock options exercised | 65,000 | 36,666 |
Proceeds from exercise of stock options | $ 89,350 | $ 33,032 |
Stock option granted, contractual term | 7 years | 7 years |
Common stock unit granted | 207,000 | 1,772,500 |
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan | $ 8,071,744 | |
Expected remaining weighted average vesting period | 2 years 4 months 17 days | |
Stock-based compensation | $ 933,411 | $ 971,340 |
Stock option vested during the period | 984,164 | 744,998 |
Stock options to purchase shares of common stock | 5,259,156 |