Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2019 | Aug. 02, 2019 | |
Document and Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2019 | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q2 | |
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 | true | |
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 | 102,929,257 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) | Jun. 30, 2019 | Dec. 31, 2018 |
Current Assets: | ||
Cash and cash equivalents | $ 23,417,512 | $ 16,559,400 |
Short-term investments | 36,503,442 | 36,922,213 |
Accounts receivable, net | 10,376,427 | |
Inventory | 269,879 | 56,012 |
Prepaid expenses and other current assets | 1,488,603 | 1,649,781 |
Total current assets | 72,055,863 | 55,187,406 |
Investments | 5,008,400 | 5,008,243 |
Operating lease right-of-use asset | 1,013,590 | |
Property and equipment, net | 147,619 | 245,425 |
Deposits | 8,888 | 8,888 |
Total assets | 78,234,360 | 60,449,962 |
Current Liabilities: | ||
Accounts payable | 3,328,726 | 2,337,367 |
Accrued expenses and other liabilities | 10,822,803 | 7,173,987 |
Total current liabilities | 14,151,529 | 9,511,354 |
Accrued expenses and other liabilities, non-current | 801,264 | 154,799 |
Operating lease liability, net of current portion | 801,264 | |
Total liabilities | 14,952,793 | 9,666,153 |
Commitments and contingencies | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at June 30, 2019 and December 31, 2018 | 0 | 0 |
Common stock, $0.001 par value, 150,000,000 shares authorized; 102,929,257 shares and 102,739,257 shares issued and outstanding at June 30, 2019 and December 31, 2018, respectively | 102,929 | 102,739 |
Additional paid-in capital | 213,405,396 | 211,265,279 |
Accumulated deficit | (150,248,516) | (160,563,961) |
Accumulated other comprehensive income (loss) | 21,758 | (20,248) |
Total stockholders' equity | 63,281,567 | 50,783,809 |
Total liabilities and stockholders' equity | $ 78,234,360 | $ 60,449,962 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Jun. 30, 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 | 102,929,257 | 102,739,257 |
Common stock, shares outstanding | 102,929,257 | 102,739,257 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Income (Loss) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Revenues: | ||||
Product revenue, net | $ 28,837,900 | $ 41,286,338 | ||
Type of Revenue [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | ||
Operating costs and expenses: | ||||
Cost of sales | $ 4,261,625 | $ 5,973,413 | ||
Type of Cost, Good or Service [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | ||
Research and development | $ 4,629,364 | $ 3,704,824 | $ 7,937,323 | $ 6,963,866 |
Selling, general and administrative | 8,987,722 | 2,631,031 | 17,404,182 | 5,305,429 |
Total operating costs and expenses | 17,878,711 | 6,335,855 | 31,314,918 | 12,269,295 |
Operating income (loss) | 10,959,189 | (6,335,855) | 9,971,420 | (12,269,295) |
Other income, net | 450,410 | 370,715 | 793,676 | 604,263 |
Net income (loss) before income taxes | 11,409,599 | (5,965,140) | 10,765,096 | (11,665,032) |
Provision for income taxes | 449,651 | 449,651 | ||
Net income (loss) | $ 10,959,948 | $ (5,965,140) | $ 10,315,445 | $ (11,665,032) |
Net income (loss) per share: | ||||
Basic | $ 0.11 | $ (0.06) | $ 0.10 | $ (0.11) |
Diluted | $ 0.10 | $ (0.06) | $ 0.10 | $ (0.11) |
Weighted average shares outstanding: | ||||
Basic | 102,869,202 | 102,596,446 | 102,808,897 | 102,577,005 |
Diluted | 105,928,970 | 102,596,446 | 105,098,930 | 102,577,005 |
Net income (loss) | $ 10,959,948 | $ (5,965,140) | $ 10,315,445 | $ (11,665,032) |
Other comprehensive income (loss): | ||||
Unrealized gain (loss) on available-for-sale securities | 28,446 | (14,672) | 42,006 | (36,498) |
Comprehensive income (loss) | $ 10,988,394 | $ (5,979,812) | $ 10,357,451 | $ (11,701,530) |
Consolidated Statement of Chang
Consolidated Statement of Changes in Stockholders' Equity - USD ($) | Total | Preferred Stock [Member] | 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 income (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, 2017 | 80,963,812 | 102,549 | 207,421,710 | (126,560,447) | ||
Net income (loss) | (11,665,032) | |||||
Ending Balance at Jun. 30, 2018 | 71,062,213 | 102,599 | 209,221,591 | (138,225,479) | (36,498) | |
Beginning Balance at Mar. 31, 2018 | 76,246,466 | 102,586 | 208,426,045 | (132,260,339) | (21,826) | |
Issuance of common stock, net | 10,549 | 3 | 10,546 | |||
Issuance of stock options for services | 776,510 | 776,510 | ||||
Exercise of stock options for common stock | 8,500 | 10 | 8,490 | |||
Other comprehensive gain (loss) | (14,672) | (14,672) | ||||
Net income (loss) | (5,965,140) | (5,965,140) | ||||
Ending Balance at Jun. 30, 2018 | 71,062,213 | 102,599 | 209,221,591 | (138,225,479) | (36,498) | |
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 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) | |
Beginning Balance at Dec. 31, 2018 | 50,783,809 | 102,739 | 211,265,279 | (160,563,961) | (20,248) | |
Net income (loss) | 10,315,445 | |||||
Ending Balance at Jun. 30, 2019 | 63,281,567 | 102,929 | 213,405,396 | (150,248,516) | 21,758 | |
Beginning Balance at Mar. 31, 2019 | 51,175,627 | 102,804 | 212,287,975 | (161,208,464) | (6,688) | |
Issuance of stock options for services | 924,996 | 924,996 | ||||
Exercise of stock options for common stock | 192,550 | 125 | 192,425 | |||
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 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Operating Activities: | ||
Net income (loss) | $ 10,315,445 | $ (11,665,032) |
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: | ||
Depreciation | 20,186 | 16,191 |
Non-cash change in right-of-use asset | 120,051 | |
Stock-based compensation | 1,858,407 | 1,762,847 |
Change in accrued interest and accretion of discount on investments | (77,547) | (255,210) |
(Increase) decrease in: | ||
Accounts receivable, net | (10,376,427) | |
Inventory | (213,867) | |
Prepaid expenses and other current assets and deposits | 161,178 | 496,403 |
Increase (decrease) in: | ||
Accounts payable | 991,359 | (1,022,982) |
Accrued expenses and other liabilities | 3,393,753 | (175,273) |
Operating lease liability | (135,123) | |
Net cash provided by (used in) operating activities | 6,057,415 | (10,843,056) |
Investing Activities: | ||
Purchases of property and equipment | (19,370) | (11,937) |
Purchases of investments | (29,772,428) | (36,790,854) |
Proceeds from sales/maturities of investments | 30,310,595 | |
Net cash provided by (used in) investing activities | 518,797 | (36,802,791) |
Financing Activities: | ||
Payment of employee withholding tax related to stock-based compensation | (4,448) | |
Proceeds from exercise of stock options | 281,900 | 41,532 |
Net cash provided by (used in) financing activities | 281,900 | 37,084 |
Net increase (decrease) in cash and cash equivalents | 6,858,112 | (47,608,763) |
Cash and cash equivalents - beginning of period | 16,559,400 | 57,496,702 |
Cash and cash equivalents - end of period | 23,417,512 | 9,887,939 |
Non-cash investing and financing activities: | ||
Unrealized gain (loss) on available-for-sale securities | $ 42,006 | $ (36,498) |
Organization and Description of
Organization and Description of Business | 6 Months Ended |
Jun. 30, 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 ® ® 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 through March 31, 2019, and reported operating income in the three and six month periods ended June 30, 2019. The Company has been able to fund its cash needs to date through several public and private offerings of its securities and from revenues from its product sales. See Note 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 | 6 Months Ended |
Jun. 30, 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 six months ended June 30, 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 or less to be cash equivalents. Cash equivalents consist mainly of money market funds. The Company has substantially all of its cash and cash equivalents deposited with one financial institution. These amounts at times may exceed federally insured limits. e. INVESTMENTS . The Company invests in high credit-quality funds in order to obtain higher yields on its cash available for investments. At June 30, 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 June 30, 2019 and December 31, 2018, the only investment classified as trading securities was the short-term bond fund. Realized losses on trading securities were $0 and $4,980, respectively, for the three and six months ended June 30, 2019. There were no sales of trading securities for the three and six months ended June 30, 2018. Unrealized gain (loss) on trading securities was $36,664 and $89,405, respectively, for the three and six months ended June 30, 2019, and $29,431 and ($29,430) for the three and six months ended June 30, 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 June 30, 2019, the Company determined that an allowance for doubtful accounts was not required. No accounts were written off during the periods presented. g. INVENTORY. ® ® inventory consisted mainly of packaging, labeling costs, and indirect costs including compensation cost of personnel and supplies. 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. 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, investments, accounts receivable, accounts payables and accrued expenses and other liabilities. At June 30, 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 June 30, 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 $ 22,764,183 $ 22,764,183 $ — $ — Short-term investments: Short-term bond fund $ 16,607,242 $ 16,607,242 $ — $ — U.S. Treasuries $ 19,896,200 $ — $ 19,896,200 $ — Investments: U.S. Treasuries $ 5,008,400 $ — $ 5,008,400 $ — 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. Effective January 1, 2019, the Company determined if an arrangement is a lease at inception. Operating leases are included in operating lease right-of-use (“ROU”) assets, other current liabilities, and operating lease liabilities on its consolidated balance sheets. 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 on 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 Revenue , 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 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 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 and six months ended June 30, 2019. As of June 30, 2019, all of the Company’s sales are to its Customer. Reserves for Variable Consideration: Revenue from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established. Components of variable consideration include trade discounts and allowances, product returns, provider chargebacks and discounts, government rebates, and other incentives, such as voluntary patient assistance, and other allowances that are offered within contracts between the Company and its Customer, payors, and other indirect customers relating to the Company’s sale of its products. These reserves, as detailed below, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable (if the amount is payable to the Customer) or a current liability (if the amount is payable to a party other than a customer). 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 June 30, 2019 and, therefore, the transaction price was not reduced further during the three and six months ended June 30, 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: ® in the consolidated balance sheets. Product Returns: Consistent with industry practice, the Company offers the SPs and its distributor limited product return rights for damaged and expiring product, provided it is within a specified period around the product expiration date as set forth in the applicable individual distribution agreement. The Company estimates the amount of its product sales that may be returned by its Customer and records this estimate as a reduction of revenue in the period the related product revenue is recognized, as well as reductions to accounts receivable, net on the consolidated balance sheets. The Company currently estimates product return liabilities using available industry data and its own sales information, including its visibility into the inventory remaining in the distribution channel. The Company has an insignificant amount of returns to date and believes that returns of its products will continue to be minimal. Provider Chargebacks and Discounts: Chargebacks for fees and discounts to providers represent the estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to the Customer who directly purchases the product from the Company. The Customer charges the Company for the difference between what they pay for the product and the ultimate selling price to the qualified healthcare providers. These reserves are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue and accounts receivables, net. Chargeback amounts are generally determined at the time of resale to the qualified healthcare provider by the Customer, and the Company generally issues credits for such amounts within a few weeks of the Customer’s notification to the Company of the resale. Reserves for chargebacks consist principally of chargebacks that the Customer has claimed, but for which the Company has not yet issued a credit. Government Rebates: The Company is subject to discount obligations under state Medicaid programs and Medicare. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenses and other current liabilities on the consolidated balance sheets. For Medicare, the Company also estimates the number of patients in the prescription drug coverage gap for whom the Company will owe an additional liability under the Medicare Part D program. The Company’s liability for these rebates consists of invoices received for claims from prior quarters that have not been paid or for which an invoice has not yet been received, estimates of claims for the current quarter, and estimated future claims that will be made for product that has been recognized as revenue, but which remains in the distribution channel inventories at the end of each reporting period. 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 and six months ended June 30, 2019 and June 30, 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 sells its product in the United States through an exclusive distributor 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 doubtful accounts 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 ® ® ® n. ROYALTIES. Royalties incurred in connection with the Company’s license agreement with BioMarin, as disclosed in Note 10 – Agreements, are expensed to cost of sales as revenue from product sales is recognized. o. STOCK-BASED COMPENSATION. The Company recognizes expense in the consolidated statements of operations for the fair value of all stock-based payments to employees, directors and consultants, including grants of stock options and other share-based awards. For stock options, the Company uses the Black-Scholes option valuation model, the single-option award approach, and the straight-line attribution method. Using this approach, compensation cost is amortized on a straight-line basis over the vesting period of each respective stock option, generally one to five years. Forfeitures are recognized as a reduction of stock-based compensation expense as they occur. p. COMPREHENSIVE INCOME (LOSS). U.S. GAAP requires that all components of comprehensive income (loss) be reported in the financial statements in the period in which they are recognized. Comprehensive income (loss) is net income (loss), plus certain other items that are recorded directly into stockholders’ equity. The Company’s comprehensive income (loss) is shown on the consolidated statements of operations and comprehensive income (loss) for the three and six months ended June 30, 2019 and 2018, and is comprised of net unrealized gains (losses) on the Company’s available-for-sale securities. q. NET INCOME (LOSS) PER COMMON SHARE. Basic net income (loss) per share is computed by dividing net income (loss) for the period by the weighted average number of common shares outstanding during the period. With regard to common stock subject to vesting requirements, the calculation includes only the vested portion of such stock and units. Diluted net income 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 weighted average common shares: For the Three Months Ended June 30, For the Six Months Ended June 30, 2019 2018 2019 2018 Basic weighted average common shares outstanding 102,869,202 102,596,446 102,808,897 102,577,005 Effect of dilutive securities: Common stock issuable upon the exercise of stock options 3,059,768 — 2,290,033 — Diluted weighted average common shares outstanding 105,928,970 102,596,446 105,098,930 102,577,005 Outstanding common stock equivalents totaling approximately 3.0 million and 3.6 million, respectively, were excluded from the calculation of diluted net income (loss) per common share for the three and six months ended June 30, 2019 . For the three and six months ended June 30, 2018, approximately 7.7 million shares of outstanding stock options were excluded from the calculation of diluted net loss per common share because a net loss was reported in each of these periods and therefore their effect was anti-dilutive. Potentially dilutive options to purchase common stock at both June 30, 2019 and 2018, had exercise prices ranging from $0.79 to $4.64. r. RECENTLY ISSUED ACCOUNTING STANDARDS. In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) , which requires an entity to recognize assets and liabilities arising from a lease for both financing and operating leases. The ASU also requires new qualitative and quantitative disclosures to help investors and other financial statement users better understand the amount, timing, and uncertainty of cash flows arising from leases. ASU 2016-02 is effective for fiscal years beginning after December 15, 2018. The Company adopted the standard as of January 1, 2019, using the modified retrospective approach in which prior comparative periods are not adjusted. The Company elected the package of practical expedients permitted under the transition guidance within the new standard, which among other things, allows the Company to carry forward historical lease classification. The Company has operating leases for its office facilities, which expire on 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. on January 1, 2020. The Company does not anticipate the adoption will have a material impact on its consolidated financial position or results of operations. s. RECLASSIFICATIONS. Certain prior year amounts in the consolidated financial statements have been reclassified to conform to the current year presentation. |
Investments
Investments | 6 Months Ended |
Jun. 30, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | 3. Investments. Available-for-sale investments by security type were as follows: Estimated Fair Value Gross Unrealized Gains Gross Unrealized Losses Amortized Cost At June 30, 2019: U.S. Treasuries - ST $ 19,896,200 $ 12,102 $ — $ 19,884,098 U.S. Treasuries - LT 5,008,400 9,656 — 4,998,744 Total $ 24,904,600 $ 21,758 $ — $ 24,882,842 At December 31, 2018: U.S. Treasuries - ST $ 10,380,864 $ — $ (1,835 ) $ 10,382,699 U.S. Treasuries - LT 5,008,243 — (18,413 ) 5,026,656 Total $ 15,389,107 $ — $ (20,248 ) $ 15,409,355 There were no realized gains or losses from available-for-sale securities for the three or six months ended June 30, 2019 or 2018. The Company did not hold any securities in an unrealized loss position for more than 12 months as of June 30, 2019. The estimated fair values of available-for-sale securities at June 30, 2019, by contractual maturity, are summarized as follows: June 30, 2019 Due in one year or less $ 24,904,600 Due after one year — $ 24,904,600 |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 6 Months Ended |
Jun. 30, 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: June 30, 2019 December 31, 2018 Prepaid research fees $ 371,743 $ 358,209 Prepaid insurance 399,939 800,261 Prepaid commercialization fees 149,785 17,030 Prepaid subscription fees 184,851 170,552 Other 382,285 303,729 Total prepaid expenses and other current assets $ 1,488,603 $ 1,649,781 |
Operating Leases
Operating Leases | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure of Operating Leases [Abstract] | |
Operating Leases | 5. Operating Leases. The Company has operating lease agreements for its corporate office. The leases include options to extend the leases for up to 1 year and options to terminate the lease within 1 year. There are no obligations under finance leases. The components of lease expense were as follows: For the Three Months Ended June 30, 2019 For the Six Months Ended June 30, 2019 Operating lease cost $ 74,079 $ 148,158 Supplemental cash flow information related to leases was as follows: June 30, 2019 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows $ 163,232 Right-of-use assets obtained in exchange for lease obligations: Operating leases $ 10,610 Supplemental balance sheet information related to leases was as follows: June 30, 2019 Operating lease right-of-use assets $ 1,013,590 Other current liabilities $ 288,470 Operating lease liabilities 801,264 Total operating lease liabilities $ 1,089,734 Weighted Average Remaining Lease Term 3.4 years Weighted Average Discount Rate 4.81 % Payments of lease liabilities as of June 30, 2019 were as follows: 2019 (remaining six months) $ 166,492 2020 339,605 2021 349,788 2022 329,662 Total lease payments 1,185,547 Less imputed interest (95,813 ) Total $ 1,089,734 |
Property and Equipment, net
Property and Equipment, net | 6 Months Ended |
Jun. 30, 2019 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | 6. Property and Equipment, Net. Property and equipment, net consists of the following: June 30, 2019 December 31, 2018 Computer equipment $ 50,904 $ 52,704 Furniture and equipment 230,741 212,451 Leasehold improvements — 177,417 281,645 442,572 Less: Accumulated depreciation (134,026 ) (197,147 ) Total property and equipment, net $ 147,619 $ 245,425 Depreciation expense was $6,438 and $20,186, respectively, for the three and six-month periods ended June 30, 2019 and $8,176 and $16,191, respectively for the three and six-month periods ended June 30, 2018. |
Accrued Expenses and Other Liab
Accrued Expenses and Other Liabilities | 6 Months Ended |
Jun. 30, 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: June 30, 2019 December 31, 2018 Accrued preclinical and clinical trial expenses $ 706,951 $ 821,633 Accrued professional fees 2,046,433 1,311,061 Accrued compensation and benefits 1,262,446 1,941,449 Accrued license fees 4,673,939 3,000,000 Lease liability 288,470 — Accrued variable consideration 1,242,055 — Deferred rent and lease incentive — 33,408 Accrued income tax 449,651 — Other 152,858 66,436 Current accrued expenses and other liabilities 10,822,803 7,173,987 Lease liability - non-current 801,264 — Deferred rent and lease incentive – non-current — 154,799 Non-current accrued expenses and other liabilities 801,264 154,799 Total accrued expenses and other liabilities $ 11,624,067 $ 7,328,786 |
Collaborative Arrangement
Collaborative Arrangement | 6 Months Ended |
Jun. 30, 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 (Endo), for the further development and commercialization of generic Sabril ® Endo 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 ® 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 Endo consisting of a mid-double digit percent of net sales of generic Sabril ® |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 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 University (which patents have been licensed by Northwestern to a third party) for a new GABA aminotransferase inhibitor were developed from CPP-115, which had previously been licensed to the Company by Northwestern. As a result, on October 26, 2018, the Company terminated the license agreement for CPP-115 and commenced an arbitration proceeding against Northwestern seeking damages for alleged breaches of the license agreement. Shortly thereafter, Northwestern filed counterclaims against the Company in the arbitration action seeking damages for alleged breaches by the Company of the license agreement. On May 21, 2019, the Company entered into a settlement agreement with Northwestern that resolved all pending disputes between the parties with no admission of liability by either party, released all claims of liability or wrongdoing between the Company and Northwestern, and dismissed the pending arbitration. In May 2019, the Company became aware that the FDA had approved an NDA for Jacobus Pharmaceuticals 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 | 6 Months Ended |
Jun. 30, 2019 | |
Text Block [Abstract] | |
Agreements | 10. Agreements. a. LICENSE AGREEMENT WITH BIOMARIN (FIRDAPSE ® ) October 26, 2012 ® , the Company pays: (i) royalties to BioMarin for seven years from the first commercial sale of Firdapse ® ® On May 29, 2019, the Company entered into an amendment to its License Agreement with BioMarin with respect to its license for Firdapse ® ® b. AGREEMENTS FOR DRUG DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES. The Company has entered into agreements with contract manufacturers for the manufacture of drug and study placebo for the Company’s trials and studies, with contract research organizations (CRO) to conduct and monitor the Company’s trials and studies and with various entities for laboratories and other testing related to the Company’s trials and studies. The contractual terms of the agreements vary, but most require certain advances as well as payments based on the achievement of milestones. Further, these agreements are cancellable at anytime, but obligate the Company to reimburse the providers for any time or costs incurred through the date of termination. |
Income Taxes
Income Taxes | 6 Months Ended |
Jun. 30, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 11. Income Taxes. The Company’s effective income tax rate is the ratio of income tax expense (benefit) over its income (loss) before income taxes. The effective income tax rate was 4.21% and 0.00% for the six months ended June 30, 2019 and 2018, respectively. Differences in the effective tax and the statutory Federal income tax rate of 21% is driven by state income taxes and anticipated annual permanent differences, including orphan drug credit expense limitations and other items. The Company had no uncertain tax positions as of June 30, 2019 and December 31, 2018. The Company has a 100% valuation allowance at June 30, 2019 and December 31, 2018. |
Stockholders' Equity
Stockholders' Equity | 6 Months Ended |
Jun. 30, 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 June 30, 2019, there is approximately $92.5 million available for future sale under the 2017 Shelf Registration Statement. |
Stock Compensation
Stock Compensation | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock Compensation | 13. Stock Compensation. For the three and six-month periods ended June 30, 2019 and 2018, the Company recorded stock-based compensation expense as follows: Three months ended June 30, Six months ended June 30, 2019 2018 2019 2018 Research and development $ 273,212 $ 285,625 $ 560,933 $ 579,940 Selling, general and administrative 651,784 505,882 1,297,474 1,182,907 Total stock-based compensation $ 924,996 $ 791,507 $ 1,858,407 $ 1,762,847 Stock Options As of June 30, 2019, there were outstanding stock options to purchase 10,427,832 shares of common stock, of which stock options to purchase 5,385,825 shares of common stock were exercisable as of June 30, 2019. During the three and six-month periods ended June 30, 2019, the Company granted seven-year term options to purchase an aggregate of 105,000 and 312,000 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $924,996 and $1,858,407, respectively, during the three and six-month periods ended June 30, 2019. During the three and six-month periods ended June 30, 2019, respectively, 306,665 and 1,290,829 options vested. During the three and six-month periods ended June 30, 2018, the Company granted seven-year term options to purchase an aggregate of 945,000 and 2,717,500 shares, respectively, of the Company’s common stock to employees and directors. The Company recorded stock-based compensation related to stock options totaling $776,510 and $1,747,850, respectively, during the three and six-month periods ended June 30, 2018. During the three and six-month periods ended June 30, 2018, respectively, 565,000 and 1,309,998 options vested. During the three and six-month periods ended June 30, 2019, options to purchase 125,000 shares and 190,000 shares, respectively, of the Company’s common stock were exercised, with proceeds of $192,550 and $281,900, respectively, to the Company. During the three and six-month periods ended June 30, 2018, options to purchase 10,000 shares and 46,666 shares, respectively, of the Company’s common stock were exercised, with proceeds of $8,500 and $41,532, respectively, to the Company. As of June 30, 2019, there was approximately $7,234,145 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 2.20 years. Common Stock There were no grants of common stock to employees during the three and six-month periods ended June 30, 2019. During both the three and six-month periods ended June 30, 2018, the Company granted 3,094 net shares of common stock to employees as compensation. The Company recorded stock-based compensation related to common stock issued to employees totaling approximately $15,000 during both the three and six-month periods ended June 30, 2018. |
Basis of Presentation and Sig_2
Basis of Presentation and Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 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 six months ended June 30, 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 or less to be cash equivalents. Cash equivalents consist mainly of money market funds. The Company has substantially all of its cash and cash equivalents deposited with one financial institution. These amounts at times may exceed federally insured limits. |
INVESTMENTS | e. INVESTMENTS . The Company invests in high credit-quality funds in order to obtain higher yields on its cash available for investments. At June 30, 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 June 30, 2019 and December 31, 2018, the only investment classified as trading securities was the short-term bond fund. Realized losses on trading securities were $0 and $4,980, respectively, for the three and six months ended June 30, 2019. There were no sales of trading securities for the three and six months ended June 30, 2018. Unrealized gain (loss) on trading securities was $36,664 and $89,405, respectively, for the three and six months ended June 30, 2019, and $29,431 and ($29,430) for the three and six months ended June 30, 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 June 30, 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. ® ® inventory consisted mainly of packaging, labeling costs, and indirect costs including compensation cost of personnel and supplies. 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. 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, investments, accounts receivable, accounts payables and accrued expenses and other liabilities. At June 30, 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 June 30, 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 $ 22,764,183 $ 22,764,183 $ — $ — Short-term investments: Short-term bond fund $ 16,607,242 $ 16,607,242 $ — $ — U.S. Treasuries $ 19,896,200 $ — $ 19,896,200 $ — Investments: U.S. Treasuries $ 5,008,400 $ — $ 5,008,400 $ — 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. Effective January 1, 2019, the Company determined if an arrangement is a lease at inception. Operating leases are included in operating lease right-of-use (“ROU”) assets, other current liabilities, and operating lease liabilities on its consolidated balance sheets. 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 on 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 Revenue , 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 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 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 and six months ended June 30, 2019. As of June 30, 2019, all of the Company’s sales are to its Customer. Reserves for Variable Consideration: Revenue from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established. Components of variable consideration include trade discounts and allowances, product returns, provider chargebacks and discounts, government rebates, and other incentives, such as voluntary patient assistance, and other allowances that are offered within contracts between the Company and its Customer, payors, and other indirect customers relating to the Company’s sale of its products. These reserves, as detailed below, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable (if the amount is payable to the Customer) or a current liability (if the amount is payable to a party other than a customer). 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 June 30, 2019 and, therefore, the transaction price was not reduced further during the three and six months ended June 30, 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: ® in the consolidated balance sheets. Product Returns: Consistent with industry practice, the Company offers the SPs and its distributor limited product return rights for damaged and expiring product, provided it is within a specified period around the product expiration date as set forth in the applicable individual distribution agreement. The Company estimates the amount of its product sales that may be returned by its Customer and records this estimate as a reduction of revenue in the period the related product revenue is recognized, as well as reductions to accounts receivable, net on the consolidated balance sheets. The Company currently estimates product return liabilities using available industry data and its own sales information, including its visibility into the inventory remaining in the distribution channel. The Company has an insignificant amount of returns to date and believes that returns of its products will continue to be minimal. Provider Chargebacks and Discounts: Chargebacks for fees and discounts to providers represent the estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to the Customer who directly purchases the product from the Company. The Customer charges the Company for the difference between what they pay for the product and the ultimate selling price to the qualified healthcare providers. These reserves are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue and accounts receivables, net. Chargeback amounts are generally determined at the time of resale to the qualified healthcare provider by the Customer, and the Company generally issues credits for such amounts within a few weeks of the Customer’s notification to the Company of the resale. Reserves for chargebacks consist principally of chargebacks that the Customer has claimed, but for which the Company has not yet issued a credit. Government Rebates: The Company is subject to discount obligations under state Medicaid programs and Medicare. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenses and other current liabilities on the consolidated balance sheets. For Medicare, the Company also estimates the number of patients in the prescription drug coverage gap for whom the Company will owe an additional liability under the Medicare Part D program. The Company’s liability for these rebates consists of invoices received for claims from prior quarters that have not been paid or for which an invoice has not yet been received, estimates of claims for the current quarter, and estimated future claims that will be made for product that has been recognized as revenue, but which remains in the distribution channel inventories at the end of each reporting period. 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 and six months ended June 30, 2019 and June 30, 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 sells its product in the United States through an exclusive distributor 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 doubtful accounts 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 | n. ROYALTIES. Royalties incurred in connection with the Company’s license agreement with BioMarin, as disclosed in Note 10 – Agreements, are expensed to cost of sales as revenue from product sales is recognized. |
STOCK-BASED COMPENSATION | o. STOCK-BASED COMPENSATION. The Company recognizes expense in the consolidated statements of operations for the fair value of all stock-based payments to employees, directors and consultants, including grants of stock options and other share-based awards. For stock options, the Company uses the Black-Scholes option valuation model, the single-option award approach, and the straight-line attribution method. Using this approach, compensation cost is amortized on a straight-line basis over the vesting period of each respective stock option, generally one to five years. Forfeitures are recognized as a reduction of stock-based compensation expense as they occur. |
COMPREHENSIVE INCOME (LOSS) | p. COMPREHENSIVE INCOME (LOSS). U.S. GAAP requires that all components of comprehensive income (loss) be reported in the financial statements in the period in which they are recognized. Comprehensive income (loss) is net income (loss), plus certain other items that are recorded directly into stockholders’ equity. The Company’s comprehensive income (loss) is shown on the consolidated statements of operations and comprehensive income (loss) for the three and six months ended June 30, 2019 and 2018, and is comprised of net unrealized gains (losses) on the Company’s available-for-sale securities. |
NET INCOME (LOSS) PER COMMON SHARE | q. NET INCOME (LOSS) PER COMMON SHARE. Basic net income (loss) per share is computed by dividing net income (loss) for the period by the weighted average number of common shares outstanding during the period. With regard to common stock subject to vesting requirements, the calculation includes only the vested portion of such stock and units. Diluted net income 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 weighted average common shares: For the Three Months Ended June 30, For the Six Months Ended June 30, 2019 2018 2019 2018 Basic weighted average common shares outstanding 102,869,202 102,596,446 102,808,897 102,577,005 Effect of dilutive securities: Common stock issuable upon the exercise of stock options 3,059,768 — 2,290,033 — Diluted weighted average common shares outstanding 105,928,970 102,596,446 105,098,930 102,577,005 Outstanding common stock equivalents totaling approximately 3.0 million and 3.6 million, respectively, were excluded from the calculation of diluted net income (loss) per common share for the three and six months ended June 30, 2019 . For the three and six months ended June 30, 2018, approximately 7.7 million shares of outstanding stock options were excluded from the calculation of diluted net loss per common share because a net loss was reported in each of these periods and therefore their effect was anti-dilutive. Potentially dilutive options to purchase common stock at both June 30, 2019 and 2018, had exercise prices ranging from $0.79 to $4.64. |
RECENTLY ISSUED ACCOUNTING STANDARDS | r. RECENTLY ISSUED ACCOUNTING STANDARDS. In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) , which requires an entity to recognize assets and liabilities arising from a lease for both financing and operating leases. The ASU also requires new qualitative and quantitative disclosures to help investors and other financial statement users better understand the amount, timing, and uncertainty of cash flows arising from leases. ASU 2016-02 is effective for fiscal years beginning after December 15, 2018. The Company adopted the standard as of January 1, 2019, using the modified retrospective approach in which prior comparative periods are not adjusted. The Company elected the package of practical expedients permitted under the transition guidance within the new standard, which among other things, allows the Company to carry forward historical lease classification. The Company has operating leases for its office facilities, which expire on 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. on January 1, 2020. The Company does not anticipate the adoption will have a material impact on its consolidated financial position or results of operations. |
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) | 6 Months Ended |
Jun. 30, 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 June 30, 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 $ 22,764,183 $ 22,764,183 $ — $ — Short-term investments: Short-term bond fund $ 16,607,242 $ 16,607,242 $ — $ — U.S. Treasuries $ 19,896,200 $ — $ 19,896,200 $ — Investments: U.S. Treasuries $ 5,008,400 $ — $ 5,008,400 $ — 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 $ — |
Basic and Dilutive Weighted Average Common Shares | The following table reconciles basic and weighted average common shares: For the Three Months Ended June 30, For the Six Months Ended June 30, 2019 2018 2019 2018 Basic weighted average common shares outstanding 102,869,202 102,596,446 102,808,897 102,577,005 Effect of dilutive securities: Common stock issuable upon the exercise of stock options 3,059,768 — 2,290,033 — Diluted weighted average common shares outstanding 105,928,970 102,596,446 105,098,930 102,577,005 |
Investments (Tables)
Investments (Tables) | 6 Months Ended |
Jun. 30, 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: Estimated Fair Value Gross Unrealized Gains Gross Unrealized Losses Amortized Cost At June 30, 2019: U.S. Treasuries - ST $ 19,896,200 $ 12,102 $ — $ 19,884,098 U.S. Treasuries - LT 5,008,400 9,656 — 4,998,744 Total $ 24,904,600 $ 21,758 $ — $ 24,882,842 At December 31, 2018: U.S. Treasuries - ST $ 10,380,864 $ — $ (1,835 ) $ 10,382,699 U.S. Treasuries - LT 5,008,243 — (18,413 ) 5,026,656 Total $ 15,389,107 $ — $ (20,248 ) $ 15,409,355 |
Estimated Fair Values of Available for Sale Securities | The estimated fair values of available-for-sale securities at June 30, 2019, by contractual maturity, are summarized as follows: June 30, 2019 Due in one year or less $ 24,904,600 Due after one year — $ 24,904,600 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Text Block [Abstract] | |
Prepaid expenses and other current assets | Prepaid expenses and other current assets consist of the following: June 30, 2019 December 31, 2018 Prepaid research fees $ 371,743 $ 358,209 Prepaid insurance 399,939 800,261 Prepaid commercialization fees 149,785 17,030 Prepaid subscription fees 184,851 170,552 Other 382,285 303,729 Total prepaid expenses and other current assets $ 1,488,603 $ 1,649,781 |
Operating Leases (Tables)
Operating Leases (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure of Operating Leases [Abstract] | |
Lease, Cost | The components of lease expense were as follows: For the Three Months Ended June 30, 2019 For the Six Months Ended June 30, 2019 Operating lease cost $ 74,079 $ 148,158 |
Schedule of Supplemental Cash Flow Information Related To Lease | Supplemental cash flow information related to leases was as follows: June 30, 2019 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows $ 163,232 Right-of-use assets obtained in exchange for lease obligations: Operating leases $ 10,610 |
Schedule of Supplemental Balance Sheet related To Lease | Supplemental balance sheet information related to leases was as follows: June 30, 2019 Operating lease right-of-use assets $ 1,013,590 Other current liabilities $ 288,470 Operating lease liabilities 801,264 Total operating lease liabilities $ 1,089,734 Weighted Average Remaining Lease Term 3.4 years Weighted Average Discount Rate 4.81 % |
Lessee, Operating Lease, Liability, Maturity | Payments of lease liabilities as of June 30, 2019 were as follows: 2019 (remaining six months) $ 166,492 2020 339,605 2021 349,788 2022 329,662 Total lease payments 1,185,547 Less imputed interest (95,813 ) Total $ 1,089,734 |
Property and Equipment, net (Ta
Property and Equipment, net (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | Property and equipment, net consists of the following: June 30, 2019 December 31, 2018 Computer equipment $ 50,904 $ 52,704 Furniture and equipment 230,741 212,451 Leasehold improvements — 177,417 281,645 442,572 Less: Accumulated depreciation (134,026 ) (197,147 ) Total property and equipment, net $ 147,619 $ 245,425 |
Accrued Expenses and Other Li_2
Accrued Expenses and Other Liabilities (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Liabilities | Accrued expenses and other liabilities consist of the following: June 30, 2019 December 31, 2018 Accrued preclinical and clinical trial expenses $ 706,951 $ 821,633 Accrued professional fees 2,046,433 1,311,061 Accrued compensation and benefits 1,262,446 1,941,449 Accrued license fees 4,673,939 3,000,000 Lease liability 288,470 — Accrued variable consideration 1,242,055 — Deferred rent and lease incentive — 33,408 Accrued income tax 449,651 — Other 152,858 66,436 Current accrued expenses and other liabilities 10,822,803 7,173,987 Lease liability - non-current 801,264 — Deferred rent and lease incentive – non-current — 154,799 Non-current accrued expenses and other liabilities 801,264 154,799 Total accrued expenses and other liabilities $ 11,624,067 $ 7,328,786 |
Stock Compensation (Tables)
Stock Compensation (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock-Based Compensation Expense | For the three and six-month periods ended June 30, 2019 and 2018, the Company recorded stock-based compensation expense as follows: Three months ended June 30, Six months ended June 30, 2019 2018 2019 2018 Research and development $ 273,212 $ 285,625 $ 560,933 $ 579,940 Selling, general and administrative 651,784 505,882 1,297,474 1,182,907 Total stock-based compensation $ 924,996 $ 791,507 $ 1,858,407 $ 1,762,847 |
Basis of Presentation and Sig_4
Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | ||||
Maximum maturity period of cash and cash equivalent | three months | |||
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 | |||
Trading Securities, Realized Gain (Loss) | $ 0 | $ 4,980 | ||
Trading Securities Sales | $ 0 | $ 0 | ||
Potential equivalent common stock excluded | 3,000,000 | 7,700,000 | 3,600,000 | 7,700,000 |
Collaborative Arrangement, Product [Member] | ||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | ||||
Upfront license fees | $ 0 | $ 0 | $ 0 | $ 0 |
Other Income, Net [Member] | ||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | ||||
Unrealized gain(loss),trading securities | $ 36,664 | $ 29,431 | $ 89,405 | $ (29,430) |
Minimum [Member] | ||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | ||||
Minimum amortization period of compensation cost on straight line basis | 1 year | |||
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 ($) | Jun. 30, 2019 | Dec. 31, 2018 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments | $ 5,008,400 | $ 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 | 16,607,242 | 26,541,349 |
U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Investments Fair Value Disclosure | 19,896,200 | 10,380,864 |
Investments | 5,008,400 | 5,008,243 |
Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Money market funds | 22,764,183 | 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 | 16,607,242 | 26,541,349 |
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 | 22,764,183 | 14,462,087 |
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 | 19,896,200 | 10,380,864 |
Investments | 5,008,400 | $ 5,008,243 |
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 - Schedule Of Reconcile Basic And Dilutive Weighted Average Common Shares (Details) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Accounting Policies [Abstract] | ||||
Basic weighted average common shares outstanding | 102,869,202 | 102,596,446 | 102,808,897 | 102,577,005 |
Effect of dilutive securities: | ||||
Common stock issuable upon the exercise of stock options | 3,059,768 | 2,290,033 | ||
Diluted weighted average common shares outstanding | 105,928,970 | 102,596,446 | 105,098,930 | 102,577,005 |
Investments - Additional Inform
Investments - Additional Information (Detail) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
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 ($) | 6 Months Ended | 12 Months Ended |
Jun. 30, 2019 | Dec. 31, 2018 | |
Net Investment Income [Line Items] | ||
Amortized cost | $ 24,882,842 | $ 15,409,355 |
Gross Unrealized Gains | 21,758 | |
Gross Unrealized Losses | (20,248) | |
Estimated Fair Value | 24,904,600 | 15,389,107 |
U.S. Treasuries - ST [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 19,884,098 | 10,382,699 |
Gross Unrealized Gains | 12,102 | |
Gross Unrealized Losses | (1,835) | |
Estimated Fair Value | 19,896,200 | 10,380,864 |
U.S. Treasuries - LT [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 4,998,744 | 5,026,656 |
Gross Unrealized Gains | 9,656 | |
Gross Unrealized Losses | (18,413) | |
Estimated Fair Value | $ 5,008,400 | $ 5,008,243 |
Investment - Estimated Fair Val
Investment - Estimated Fair Values of Available for Sale Securities (Detail) - USD ($) | Jun. 30, 2019 | Dec. 31, 2018 |
Investments, Debt and Equity Securities [Abstract] | ||
Due in one year or less | $ 24,904,600 | |
Due after one year but within two years | 0 | |
Estimated Fair Value | $ 24,904,600 | $ 15,389,107 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($) | Jun. 30, 2019 | Dec. 31, 2018 |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid research fees | $ 371,743 | $ 358,209 |
Prepaid insurance | 399,939 | 800,261 |
Prepaid commercialization fees | 149,785 | 17,030 |
Prepaid subscriptions fees | 184,851 | 170,552 |
Other | 382,285 | 303,729 |
Total prepaid expenses and other current assets | $ 1,488,603 | $ 1,649,781 |
Operating Leases - Operating Le
Operating Leases - Operating Leases (Detail) - USD ($) | 3 Months Ended | 6 Months Ended |
Jun. 30, 2019 | Jun. 30, 2019 | |
Disclosure of Operating Leases [Abstract] | ||
Operating lease cost | $ 74,079 | $ 148,158 |
Operating Leases - Schedule of
Operating Leases - Schedule of Supplemental Cash Flow Information Related To Lease (Detail) | 6 Months Ended |
Jun. 30, 2019USD ($) | |
Cash paid for amounts included in the measurement of lease liabilities: | |
Operating cash flows | $ 163,232 |
Right-of-use assets obtained in exchange for lease obligations: | |
Operating leases | $ 10,610 |
Operating Leases -Schedule of S
Operating Leases -Schedule of Supplemental Balance Sheet related To Lease (Detail) | Jun. 30, 2019USD ($) |
Disclosure of Operating Leases [Line Items] | |
Operating lease right-of-use assets | $ 1,013,590 |
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) | 288,470 |
Operating lease liabilities | 801,264 |
Total operating lease liabilities | $ 1,089,734 |
Weighted Average Remaining Lease Term | 3 years 4 months 24 days |
Weighted Average Discount Rate | 4.81% |
Operating Leases -Lessee, Opera
Operating Leases -Lessee, Operating Lease, Liability, Maturity (Detail) | Jun. 30, 2019USD ($) |
Disclosure of Operating Leases [Line Items] | |
2019 (remaining six months) | $ 166,492 |
2020 | 339,605 |
2021 | 349,788 |
2022 | 329,662 |
Total lease payments | 1,185,547 |
Less imputed interest | (95,813) |
Total | $ 1,089,734 |
Operating Leases - Additional
Operating Leases - Additional Information (Detail) | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure of Operating Leases [Line Items] | |
Operating Lease, Weighted Average Remaining Lease Term | 3 years 4 months 24 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 ($) | Jun. 30, 2019 | Dec. 31, 2018 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 281,645 | $ 442,572 |
Less: Accumulated depreciation | (134,026) | (197,147) |
Total property and equipment, net | 147,619 | 245,425 |
Computer Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 50,904 | 52,704 |
Furniture and Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 230,741 | 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 | 6 Months Ended | ||
Mar. 31, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Property, Plant and Equipment [Abstract] | ||||
Depreciation expenses | $ 6,438 | $ 8,176 | $ 20,186 | $ 16,191 |
Accrued Expenses and Other Li_3
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($) | Jun. 30, 2019 | Dec. 31, 2018 |
Payables and Accruals [Abstract] | ||
Accrued preclinical and clinical trial expenses | $ 706,951 | $ 821,633 |
Accrued professional fees | 2,046,433 | 1,311,061 |
Accrued compensation and benefits | 1,262,446 | 1,941,449 |
Accrued license fees | 4,673,939 | 3,000,000 |
Lease liability | 288,470 | |
Accrued variable consideration | 1,242,055 | |
Deferred rent and lease incentive | 33,408 | |
Accrued income tax | 449,651 | |
Other | 152,858 | 66,436 |
Current accrued expenses and other liabilities | 10,822,803 | 7,173,987 |
Lease liability - non-current | 801,264 | |
Deferred rent and lease incentive - non-current | 154,799 | |
Non-current accrued expenses and other liabilities | 801,264 | 154,799 |
Total accrued expenses and other liabilities | $ 11,624,067 | $ 7,328,786 |
Collaborative Arrangement - Add
Collaborative Arrangement - Additional Information (Detail) - Collaborative Arrangement [Member] - USD ($) | 3 Months Ended | 6 Months Ended | 12 Months Ended |
Jun. 30, 2019 | Jun. 30, 2019 | Dec. 31, 2018 | |
Collaborative Arrangement [Line Items] | |||
Upfront license fee | $ 0 | $ 0 | $ 500,000 |
Period of collaboration agreement | 10 years |
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 | 6 Months Ended |
Jun. 30, 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) - USD ($) | 6 Months Ended | 12 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |||
Effective Statutory Income Tax Rate | 4.21% | 0.00% | |
Change In Effective and Statutory Income Tax rate | 21.00% | ||
Uncertain tax Position | $ 0 | $ 0 | |
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 | Jun. 30, 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 | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | $ 924,996 | $ 791,507 | $ 1,858,407 | $ 1,762,847 |
Research and Development [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | 273,212 | 285,625 | 560,933 | 579,940 |
Selling, General and Administrative [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | $ 651,784 | $ 505,882 | $ 1,297,474 | $ 1,182,907 |
Stock Compensation - Additional
Stock Compensation - Additional Information (Detail) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Proceeds from exercise of stock options | $ 281,900 | $ 41,532 | ||
Employee [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Common stock unit granted | 0 | 3,094 | 0 | 3,094 |
Stock-based compensation | $ 15,000 | $ 15,000 | ||
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 | 10,427,832 | 10,427,832 | ||
Number of stock options exercised | 125,000 | 10,000 | 190,000 | 46,666 |
Proceeds from exercise of stock options | $ 192,550 | $ 8,500 | $ 281,900 | $ 41,532 |
Stock option granted, contractual term | 7 years | 7 years | 7 years | 7 years |
Common stock unit granted | 105,000 | 945,000 | 312,000 | 2,717,500 |
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan | $ 7,234,145 | $ 7,234,145 | ||
Expected remaining weighted average vesting period | 2 years 2 months 12 days | |||
Stock-based compensation | $ 924,996 | $ 776,510 | $ 1,858,407 | $ 1,747,850 |
Stock option vested during the period | 306,665 | 565,000 | 1,290,829 | 1,309,998 |
Stock options to purchase shares of common stock | 5,385,825 | 5,385,825 |