0001193125-21-081545 | 10-K | XBRL Viewer | Catalyst Pharmaceuticals INC

Cover Page - USD ($)	12 Months Ended
Cover Page - USD ($)	Dec. 31, 2020	Mar. 11, 2021	Jun. 30, 2020
Cover [Abstract]
Document Type	10-K
Amendment Flag	false
Document Period End Date	Dec. 31, 2020
Document Fiscal Year Focus	2020
Document Fiscal Period Focus	FY
Entity Registrant Name	CATALYST PHARMACEUTICALS, INC.
Entity Central Index Key	0001369568
Current Fiscal Year End Date	--12-31
Entity Well-known Seasoned Issuer	No
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Shell Company	false
Entity Filer Category	Accelerated Filer
Trading Symbol	CPRX
Entity Emerging Growth Company	false
Entity Small Business	false
Entity Address, State or Province	FL
Entity Interactive Data Current	Yes
Title of 12(b) Security	Common Stock
Security Exchange Name	NASDAQ
Entity Common Stock, Shares Outstanding		103,824,973
Entity Tax Identification Number	76-0837053
Entity Incorporation, State or Country Code	DE
Entity Address, Address Line One	355 Alhambra Circle
Entity Address, Address Line Two	Suite 801
Entity Address, City or Town	Coral Gables
Entity Address, Postal Zip Code	33134
City Area Code	305
Local Phone Number	420-3200
Document Annual Report	true
Document Transition Report	false
Entity File Number	001-33057
Entity Public Float			$ 444,887,344
ICFR Auditor Attestation Flag	true

Consolidated Balance Sheets - USD ($)	Dec. 31, 2020	Dec. 31, 2019
Current Assets:
Cash and cash equivalents	$ 130,237,109	$ 89,511,710
Short-term investments	10,041,068	5,007,050
Accounts receivable, net	5,987,426	10,536,997
Inventory	4,650,600	1,956,792
Prepaid expenses and other current assets	8,327,771	4,351,074
Total current assets	159,243,974	111,363,623
Operating lease right-of-use asset		793,252
Property and equipment, net	129,800	210,467
Deferred tax assets	32,971,264
Deposits	8,888	8,888
Total assets	192,353,926	112,376,230
Current Liabilities:
Accounts payable	4,256,020	4,117,447
Accrued expenses and other liabilities	18,500,267	19,981,295
Total current liabilities	22,756,287	24,098,742
Operating lease liability, net of current portion		647,532
Total liabilities	22,756,287	24,746,274
Commitments and contingencies (Note 8)
Stockholders' equity:
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at December 31, 2020 and 2019	0
Common stock, $0.001 par value, 200,000,000 and 150,000,000 shares authorized; 103,781,641 shares and 103,397,033 shares issued and outstanding at December 31, 2020 and 2019, respectively	103,782	103,397
Additional paid-in capital	223,168,149	216,205,678
Accumulated deficit	(53,705,624)	(128,688,624)
Accumulated other comprehensive income (loss)	31,332	9,505
Total stockholders' equity	169,597,639	87,629,956
Total liabilities and stockholders' equity	$ 192,353,926	$ 112,376,230

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2020	Dec. 31, 2019
Statement of Financial Position [Abstract]
Preferred stock, par value	$ 0.001	$ 0.001
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, shares issued	0	0
Preferred stock, shares outstanding	0	0
Common stock, par value	$ 0.001	$ 0.001
Common stock, shares authorized	200,000,000	150,000,000
Common stock, shares issued	103,781,641	103,397,033
Common stock, shares outstanding	103,781,641	103,397,033

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) (unaudited) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Revenues:
Product revenue, net	$ 118,740,617	$ 102,306,337
Revenue, Product and Service [Extensible List]	us-gaap:ProductMember	us-gaap:ProductMember	us-gaap:ProductMember
Revenues from collaborative arrangements	$ 332,186		$ 500,000
Total revenues	119,072,803	$ 102,306,337	$ 500,000
Operating costs and expenses:
Cost of sales	$ 17,039,157	$ 14,759,139
Cost, Product and Service [Extensible List]	us-gaap:ProductMember	us-gaap:ProductMember	us-gaap:ProductMember
Research and development	$ 16,496,715	$ 18,842,752	$ 19,919,204
Selling, general and administrative	44,233,754	36,881,187	15,875,961
Total operating costs and expenses	77,769,626	70,483,078	35,795,165
Operating income (loss)	41,303,177	31,823,259	(35,295,165)
Other income, net	586,897	1,585,774	1,291,651
Net income (loss) before income taxes	41,890,074	33,409,033	(34,003,514)
Income tax provision (benefit)	(33,092,926)	1,533,696	0
Net income (loss)	$ 74,983,000	$ 31,875,337	$ (34,003,514)
Net income (loss) per share:
Basic	$ 0.72	$ 0.31	$ (0.33)
Diluted	$ 0.71	$ 0.30	$ (0.33)
Weighted average shares outstanding:
Basic	103,512,913	102,944,316	102,633,884
Diluted	106,242,273	106,020,936	102,633,884
Net income (loss)	$ 74,983,000	$ 31,875,337	$ (34,003,514)
Other comprehensive income (loss):
Unrealized gain (loss) on available-for-sale securities	21,827	29,753	(20,248)
Comprehensive income (loss)	$ 75,004,827	$ 31,905,090	$ (34,023,762)

CONSOLIDATED STATEMENTS OF CHANGES IN STOCKHOLDERS' EQUITY (unaudited) - USD ($)	Total	Common Stock [Member]	Additional Paid-in Capital [Member]	Accumulated Deficit [Member]	Accumulated Other Comprehensive Gain (Loss) [Member]
Beginning Balance at Dec. 31, 2017	$ 80,963,812	$ 102,549	$ 207,421,710	$ (126,560,447)
Beginning Balance (shares) at Dec. 31, 2017		102,549,498
Issuance of common stock, net	10,549	$ 3	10,546
Issuance of common stock, net (shares)		3,094
Issuance of stock options for services	3,535,647		3,535,647
Exercise of stock options for common stock	297,563	$ 187	297,376
Exercise of stock options for common stock (shares)		186,665
Other comprehensive gain (loss)	(20,248)				$ (20,248)
Net income (loss)	(34,003,514)			(34,003,514)
Ending Balance at Dec. 31, 2018	50,783,809	$ 102,739	211,265,279	(160,563,961)	(20,248)
Ending Balance (shares) at Dec. 31, 2018		102,739,257
Issuance of stock options for services	3,780,086		3,780,086
Exercise of stock options for common stock	1,116,242	$ 658	1,115,584
Exercise of stock options for common stock (shares)		657,776
Amortization of restricted stock for services	44,729		44,729
Other comprehensive gain (loss)	29,753				29,753
Net income (loss)	31,875,337			31,875,337
Ending Balance at Dec. 31, 2019	87,629,956	$ 103,397	216,205,678	(128,688,624)	9,505
Ending Balance (shares) at Dec. 31, 2019		103,397,033
Issuance of stock options for services	5,694,120		5,694,120
Exercise of stock options for common stock	$ 758,130	$ 282	757,848
Exercise of stock options for common stock (shares)	281,762	281,762
Amortization of restricted stock for services	$ 566,803		566,803
Issuance of common stock upon vesting of restricted stock units, net	(56,197)	$ 103	(56,300)
Issuance of common stock upon vesting of restricted stock units, net (Share)		102,846
Other comprehensive gain (loss)	21,827				21,827
Net income (loss)	74,983,000			74,983,000
Ending Balance at Dec. 31, 2020	$ 169,597,639	$ 103,782	$ 223,168,149	$ (53,705,624)	$ 31,332
Ending Balance (shares) at Dec. 31, 2020		103,781,641

CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Operating Activities:
Net income (loss)	$ 74,983,000	$ 31,875,337	$ (34,003,514)
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities:
Depreciation	92,065	54,327	37,978
Amortization of right-of-use asset	793,252	243,399
Stock-based compensation	6,260,923	3,824,815	3,550,644
Deferred taxes	(32,971,264)
Change in accrued interest and accretion of discount on investments	(12,191)	(290,805)	(443,139)
(Increase) decrease in:
Accounts receivable, net	4,549,571	(10,536,997)
Inventory	(2,693,808)	(1,900,780)	(56,012)
Prepaid expenses and other current assets and deposits	(3,976,697)	(2,701,293)	(476,037)
Increase (decrease) in:
Accounts payable	138,573	1,780,080	391,792
Accrued expenses and other liabilities	(1,209,280)	12,540,197	4,850,743
Operating lease liability	(919,280)	(276,807)
Net cash provided by (used in) operating activities	45,034,864	34,611,473	(26,147,545)
Investing Activities:
Purchases of property and equipment	(11,398)	(19,369)	(92,018)
Purchases of investments	(10,000,000)	(34,725,401)	(36,790,854)
Proceeds from maturities and sales of investments	5,000,000	71,969,365	21,800,000
Net cash provided by (used in) investing activities	(5,011,398)	37,224,595	(15,082,872)
Financing Activities:
Payment of employee withholding tax related to stock-based compensation	(56,197)		(4,448)
Proceeds from exercise of stock options	758,130	1,116,242	297,563
Net cash provided by (used in) financing activities	701,933	1,116,242	293,115
Net increase (decrease) in cash and cash equivalents	40,725,399	72,952,310	(40,937,302)
Cash and cash equivalents – beginning of period	89,511,710	16,559,400	57,496,702
Cash and cash equivalents – end of period	130,237,109	89,511,710	16,559,400
Supplemental disclosures of cash flow information:
Cash paid for income taxes	2,785,497
Non-cash investing and financing activities:
Unrealized gain (loss) on available-for-sale securities	$ 21,827	$ 29,753	$ (20,248)

Organization and Description of Business

12 Months Ended

Dec. 31, 2020

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Organization and Description of Business

1.	Organization and Descript i on of Business.

Catalyst Pharmaceuticals, Inc. and subsidiary (collectively, the “Company”) is a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS) and Anti-MuSK antibody positive myasthenia gravis (MuSK-MG). The Company (f/k/a Catalyst Pharmaceutical Partners, Inc.) was incorporated in Delaware in July 2006. It is the successor by merger to Catalyst Pharmaceutical Partners, Inc., a Florida corporation, which commenced operations in January 2002.

On November 28, 2018, the U.S. Food and Drug Administration, or FDA, granted approval of Firdapse

for the treatment of adults with LEMS (ages 17 and above). On January 15, 2019, the Company launched its first product, Firdapse

, in the United States for the treatment of adults with LEMS.

On August 6, 2020, the Company announced that Canada’s national healthcare regulatory agency, Health Canada, has approved Firdapse

for the treatment of patients in Canada with LEMS. On October 28, 2020, the Company launched Firdapse

in Canada for the treatment of patients with LEMS through a license agreement with KYE Pharmaceuticals.

Since inception, the Company has devoted substantially all of its efforts to business planning, research and development, recruiting management and technical staff, acquiring operating assets, raising capital, and selling its product. The Company incurred operating losses in each period from inception and started reporting operating income during the year ended December 31, 2019. The Company has been able to fund its cash needs to date through several public and private offerings of its securities and from revenues from its product sales. See Note 11 (Stockholders’ Equity).

Capital Resources

While there can be no assurance, based on currently available information, the Company estimates that it has sufficient resources to support its operations for at least the next 12 months from the issuance date of this Form

10-K.

The Company may raise required funds in the future through public or private equity offerings, debt financings, corporate collaborations, governmental research grants or other means. The Company may also seek to raise new capital to fund additional drug development efforts, even if it has sufficient funds for its planned operations. Any sale by the Company of additional equity or convertible debt securities could result in dilution to the Company’s current stockholders. There can be no assurance that any required additional funding will be available to the Company at all or available on terms acceptable to the Company. Further, to the extent that the Company raises additional funds through collaborative arrangements, it may be necessary to relinquish some rights to the Company’s drug candidates or grant sublicenses on terms that are not favorable to the Company. If the Company is not able to secure additional funding when needed, the Company may have to delay, reduce the scope of, or eliminate one or more research and development programs, which could have an adverse effect on the Company’s business.

Risks and Uncertainties

There are many uncertainties regarding the novel coronavirus

(COVID-19)

pandemic, and the Company is closely monitoring the impact of the pandemic on all aspects of its business, including how the pandemic is impacting its patients, employees, suppliers, vendors, business partners, clinical trials, and distribution channels. The Company is unable to predict the impact that

COVID-19

will have on its financial position and operating results in future periods due to numerous uncertainties. The Company will continue to assess the evolving impact of the

COVID-19

pandemic and make adjustments to its operations as necessary.

Basis of Presentation and Significant Accounting Policies

12 Months Ended

Dec. 31, 2020

Accounting Policies [Abstract]

Basis of Presentation and Significant Accounting Policies

2.	Basis of Presentation and Significant Accounting Policies.

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.

The preparation of financial statements in conformity with accounting principles generally accepted in the U.S. (U.S. GAAP) requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. Actual results could differ from those estimates.

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 and U.S. Treasuries. The Company has substantially all of its cash and cash equivalents deposited with one financial institution. These amounts exceed federally insured limits.

INVESTMENTS.

The Company invests in high credit-quality instruments in order to obtain higher yields on its cash available for investments. At December 31, 2020, investments consisted of short-term bond funds and U.S. Treasuries. At December 31, 2019, investments consisted of U.S. Treasuries. Such investments are not insured by the Federal Deposit Insurance Corporation.

The short-term bond funds and U.S. Treasuries held at December 3

, 2020 are classified as

available-for-sale

securities. The short-term bond funds are classified as current assets, which reflects management’s intention to use the proceeds from the sale of these investments to fund the Company’s operations, as necessary. The Company classifies U.S. Treasuries with stated maturities of greater than three months and less than one year in short-term investments, U.S Treasuries with stated maturities greater than one year are classified as

non-current

investments in its 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 and comprehensive income (loss). 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 (Investments).

The Company previously owned a short-term bond fund that was classified as trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognized realized gains and losses and unrealized gains and losses to earnings. At December 31, 2020 and 2019, there were no

investments classified as trading securities, as the Company sold its interest in the short-term bond fund classified as trading securities in 2019

There was no realized or unrealized gain (loss) on trading securities for the year ended December 31, 2020. Realized losses on trading securities during the years ended December 31, 2019 and 2018 were $80,045 and $0, respectively. Unrealized gain (loss) on trading securities was $0 and ($29,430) for the years ended December 31, 2019 and 2018, respectively, and is included in other income, net in the accompanying consolidated statements of operations.

ACCOUNTS RECEIVABLE, NET.

Accounts receivable are recorded net of customer allowance for distribution fees, trade discounts, prompt payment discounts, chargebacks and expected credit losses. Allowances for distribution fees, trade discounts, prompt payment discounts and chargebacks are based on contractual terms. The Company estimates the allowance for expected credit losses based on existing contractual payment terms, actual payment patterns of its customer and customer circumstances. At December 31, 2020 and 2019, the Company determined that an allowance for expected credit losses was not required. No accounts were written off during the periods presented.

INVENTORY

. Inventories are stated at the lower of cost or net realizable value

Inventories consist of raw materials,

work-in-process

and finished goods. Costs to be capitalized as inventories primarily include third party manufacturing costs and other overhead costs.

Cost is determined using a standard cost method, which approximates actual cost, and assumes a first-in, first out (FIFO) flow of goods.

The Company began capitalizing inventories post FDA approval of Firdapse

on November 28, 2018 as the related costs were expected to be recoverable through the commercialization of the product. Costs incurred prior to the FDA approval of Firdapse

were recorded as research and development expenses in prior years’ consolidated statements of operations and comprehensive income (loss). If information becomes available that suggests that inventories may not be realizable, the Company may be required to expense a portion or all of the previously capitalized inventories. As of December 31, 2020 and 2019, inventory consisted mainly of

work-in-process

and finished goods.

Products that have been approved by the FDA or other regulatory authorities, such as Firdapse

, are also used in clinical programs to assess the safety and efficacy of the products for usage in treating diseases that have not been approved by the FDA or other regulatory authorities. The form of Firdapse

utilized for both commercial and clinical programs is identical and, as a result, the inventory has an “alternative future use” as defined in authoritative guidance. Raw materials associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes and, therefore, does not have an “alternative future use”.

The Company evaluates for potential excess inventory by analyzing current and future product demand relative to the remaining product shelf life. The Company builds demand forecasts by considering factors such as, but not limited to, overall market potential, market share, market acceptance, and patient usage.

PREPAID EXPENSES AND OTHER CURRENT ASSETS.

Prepaid expenses and other current assets consist primarily of prepaid manufacturing, prepaid tax, prepaid insurance, prepaid subscription fees, prepaid research fees, prepaid commercialization expenses, and amounts due from collaborative arrangements. Prepaid research fees consist of advances for the Company’s product development activities, including contracts for

pre-clinical

studies, clinical trials and studies, regulatory affairs and consulting. Prepaid manufacturing consists of advances for the Company’s drug manufacturing activities. Such advances are recorded as expense as the related goods are received or the related services are performed.

PROPERTY AND EQUIPMENT,

NET.

Property and equipment are recorded at cost. Depreciation is calculated to amortize the depreciable assets over their useful lives using the straight-line method and commences when the asset is placed in service. Leasehold improvements are amortized on a straight-line basis over the term of the lease or the estimated life of the improvement, whichever is shorter. Useful lives generally range from three to five years for computer equipment to five years for furniture and equipment, and from four to seven years for leasehold improvements. Expenditures for repairs and maintenance are charged to expenses as incurred.

FAIR VALUE OF FINANCIAL INSTRUMENTS.

The Company’s financial instruments consist of cash and cash equivalents, investments, accounts receivable, accounts payable, and accrued expenses and other liabilities. At December 31, 2020 and 2019, the fair value of these instruments approximated their carrying value.

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 December 31, 2020		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	15,673,626	$	15,673,626	$	—	$	—

U.S. Treasuries	$	104,994,400	$	—	$	104,994,400	$	—

Short-term investments:
Short-term bond funds	$	10,041,068	$	10,041,068	$	—	$	—


	Balances as of December 31, 2019		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	23,963,617	$	23,963,617	$	—	$	—

U.S. Treasuries	$	59,932,200	$	—	$	59,932,200	$	—

Short-term investments:
U.S. Treasuries	$	5,007,050	$	—	$	5,007,050	$	—

OPERATING LEASES.

The Company determines 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.

REVENUE RECOGNITION.

The Company recognizes revenue when its customer obtains title of the promised goods, in an amount that reflects the consideration to which the Company expects to be entitled in exchange for these goods. The Company had no contracts with customers until the FDA approved Firdapse

in November 2018. Subsequent to receiving FDA approval, the Company entered into an arrangement with one distributor (the “Customer”), which is the exclusive distributor of Firdapse

in the United States. The Customer subsequently resells Firdapse

to a small group of exclusive specialty pharmacies (“SPs”) whose dispensing activities for patients with specific payors may result in government-mandated or privately negotiated rebate obligations for the Company with respect to the purchase of Firdapse

To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to arrangements that meet the definition of a contract under Topic 606, including when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. For a complete discussion of accounting for product revenue, see Product Revenue, Net below.

The Company also may generate revenues from payments received under a collaborative agreement. Collaborative agreement payments may include nonrefundable fees at the inception of the agreements, contingent payments for specific achievements designated in the collaborative agreements, and/or net profit-sharing payments on sales of products resulting from a collaborative arrangement. For a complete discussion of accounting for collaborative arrangements, see Revenues from Collaborative Arrangements below.

Product Revenue, Net:

The Company sells Firdapse

to the Customer (its exclusive distributor) who subsequently resells Firdapse

to both a small group of SPs who have exclusive contracts with the Company to distribute the Company’s products to patients and potentially to medical centers or hospitals on an emergency basis. In addition to the distribution agreement with its Customer, the Company enters into arrangements with health care providers and payors that provide for government-mandated and/or privately negotiated rebates, chargebacks, and discounts with respect to the purchase of the Company’s products.

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 or upon dispense to patient). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 15 and 30 days.

Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods and are recorded in cost of sales.

If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred, if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the years ended December 31, 2020 and 2019.

During the years ended December 31, 2020 and 2019, all of the Company’s sales of Firdapse

in the United States were 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, prompt payment discounts, 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 contemplates 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 December 31, 2020 and, therefore, the transaction price was not reduced further during the years ended December 31, 2020 and 2019. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known.

Trade Discounts and Allowances:

The Company provides its Customer with a discount that is explicitly stated in its contract and is recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, the Company receives sales order management, data and distribution services from the Customer. To the extent the services received are distinct from the sale of Firdapse

to the Customer, these payments are classified in selling, general and administrative expenses in the Company’s consolidated statement of operations and comprehensive income (loss). However, if the Company has determined such services received to date are not distinct from the Company’s sale of products to the Customer, these payments have been recorded as a reduction of revenue within the consolidated statement of operations and comprehensive income (loss) through December 31, 2020 and 2019, as well as a reduction to accounts receivable, net on the consolidated balance sheets.

Prompt Payment Discounts:

The Company provides its customer with prompt payment discounts which may result in adjustments to the price that is invoiced for the product transferred, in the case that payments are made within a defined period. The prompt payment discount reserve is based on actual net sales and contractual discount rates. Reserves for prompt payment discounts are included in accounts receivable, net on the consolidated balance sheets.

Funded

Co-pay

Assistance Program:

The Company contracts with a third-party to manage the

co-pay

assistance program intended to provide financial assistance to qualified commercially-insured patients. The calculation of the accrual for

co-pay

assistance is based on an estimate of claims and the cost per claim that the Company expects to receive associated with Firdapse

that has been recognized as revenue, but remains in the distribution channel at the end of each reporting period. These payments are considered payable to the third-party vendor and the related reserve is 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 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. 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, net and accounts receivable, 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:

The Company provides free Firdapse

to uninsured patients who satisfy

pre-established

criteria for either the Bridge Program or the Patient Assistance Program. Patients who meet the Bridge Program eligibility criteria and are transitioning from investigational product while they are waiting for a coverage determination, or later, for patients whose access is threatened by the complications arising from a change of insurer may receive a temporary supply of free Firdapse

while the Company is determining the patient’s third-party insurance, prescription drug benefit or other third-party coverage for Firdapse

. The Patient Assistance Program provides free Firdapse

for longer periods of time for those who are uninsured or functionally uninsured with respect to Firdapse

because they are unable to obtain coverage from their payor despite having health insurance, to the extent allowed by applicable law. The Company does not recognize any revenue related to these free products and the associated costs are classified in selling, general and administrative expenses in the Company’s consolidated statements of operations and comprehensive income (loss).

Revenues from Collaborative Arrangements:

The Company has entered into collaboration agreements for the further development and commercialization of generic Sabril

(vigabatrin) tablets as well as the commercialization of Firdapse

in Canada. Pursuant to the terms of these agreements, collaborators could be required to make various payments to the Company, including upfront license fees, milestone payments based on achievement of regulatory approvals, and royalties or

net-profit

sharing payments on sales of products resulting from the collaborative agreement.

Nonrefundable upfront license fees are recognized upon receipt or over time based on a determination of whether access to the Company’s intellectual property is throughout the license period, or whether the right is provided as the intellectual property exists as the point in time in which the license is granted. In the third quarter of 2020, an upfront fee was earned under the collaboration agreement for the commercialization of Firdapse

in Canada.

The collaborative agreements provide for milestone payments upon achievement of development and regulatory events. The Company accounts for milestone payments as variable consideration in accordance with Topic 606. The Company recognizes

sales-based

royalties or net profit-sharing when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the

sales-based

royalty or net profit-sharing has been satisfied.

In arrangements where the Company does not deem the collaborator to be a customer, payments to and from the collaborator are presented in the statement of operations based on the nature of the Company’s business operations, the nature of the arrangement, including the contractual terms, and the nature of the payments.

Under the arrangements, the Company will receive profit-sharing reports 60 days after quarter end from one collaborator, and within nine days after quarter end from the other collaborator. Since the Company will receive these reports 60 days after quarter end, profit-sharing revenue from sales of collaboration products by the Company’s collaborator will be recognized in the quarter following the quarter in which the corresponding sales occurred. In instances where profit-sharing reports are received within nine days after quarter end, revenue from sales of collaboration products by the Company’s collaborator will be recognized in the quarter in which the sales occurred. For the year ended December 31, 2020, there was profit-sharing revenue of approximately

33,000

from sales of the collaborative product in Canada. For the years ended December

2019

and

2018

, there was

profit-sharing revenue from sales of the collaborative product.

Refer to Note 7 (Collaborative Arrangements), for further discussion on the Company’s collaborative arrangements.

RESEARCH AND DEVELOPMENT.

Costs incurred in connection with research and development activities are expensed as incurred. These costs consist of direct and indirect costs associated with specific projects, as well as fees paid to various entities that perform research related services for the Company.

STOCK-BASED COMPENSATION.

The Company recognizes expense in the consolidated statements of operations and comprehensive income (loss) 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 three years. Forfeitures are recognized as a reduction of stock-based compensation expense as they occur.

CONCENTRATION OF RISK.

The financial instruments that potentially subject the Company to concentration of credit risk are cash equivalents (i.e., money market funds), investments and accounts receivable, net. The Company places its cash and cash equivalents with high-credit quality financial institutions. These amounts exceed federally insured limits. The Company has not experienced any credit losses in these accounts.

The Company sells its product in the United States through an exclusive distributor (its Customer) to specialty pharmacies. Therefore, its distributor and specialty pharmacies account for all of its trade receivables and net product revenues. The creditworthiness of its Customer is continuously monitored, and the Company has internal policies regarding customer credit limits. The Company estimates an allowance for expected credit loss primarily based on the credit worthiness of its Customer, historical payment patterns, aging of receivable balances and general economic conditions.

The Company currently has a single product with limited commercial sales experience, which makes it difficult to evaluate its current business, predict its future prospects and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, Firdapse

, and expects Firdapse

to constitute virtually all of product revenue for the foreseeable future. The Company’s success depends on its ability to continue to effectively commercialize Firdapse

The Company relies exclusively on third parties to formulate and manufacture Firdapse

and its drug candidates. The commercialization of Firdapse

and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and for the commercialization of Firdapse

. If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of its drugs.

ROYALTIES.

Royalties incurred in connection with the Company’s license agreement, as disclosed in Note 9 (Agreements), are expensed to cost of sales as revenue from product sales is recognized.

INCOME TAXES.

The Company utilizes the asset and liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on differences between the financial reporting and tax basis of assets and liabilities and are measured using enacted tax rates and laws that will be in effect when the differences are expected to reverse. A valuation allowance is provided when it is more likely than not that some portion or all of a deferred tax asset will not be realized.

The Company recognizes the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the

more-likely-than-not

threshold, the amount recognized in the financial statements is the largest benefit that has a greater than 50 percent likelihood of being realized upon ultimate settlement with the relevant tax authority. The Company is subject to income taxes in the U.S. federal jurisdiction and various state jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for years before 2017. If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense.

On March 27, 2020, the Coronavirus Aid, Relief, and Economic Security (CARES) Act was signed into law making several changes to the Internal Revenue Code. The changes include, but are not limited to: increasing the limitation on the amount of deductible interest expense, allowing companies to carryback certain net operating losses, and increasing the amount of net operating loss carryforwards that corporations can use to offset taxable income.

The tax law changes in the CARES Act did not have a material impact on the Company’s income tax provision.

COMPREHENSIVE INCOME (LOSS).

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 years ended December 31, 2020, 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.

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.

Diluted net income (loss) per common share is computed by dividing net income (loss) by the weighted average number of common shares outstanding, increased by the assumed conversion of other potentially dilutive securities during the period.

The following table reconciles basic and diluted weighted average common shares:


	For the Years Ended December 31,
	2020		2019		2018
Basic weighted average common shares outstanding		103,512,913		102,944,316		102,633,884
Effect of dilutive securities		2,729,360		3,076,620		—

Diluted weighted average common shares outstanding		106,242,273		106,020,936		102,633,884

Outstanding common stock equivalents totaling approximately 7.1 million and 4.6 million, were excluded from the calculation of diluted net income (loss) per common share for the years ended December 31, 2020 and 2019, respectively, as their effect would be anti-dilutive. For the year ended December 31, 2018, approximately 10.5 million shares of outstanding stock options were excluded from the calculation of diluted net loss per common share because a net loss was reported in this period and therefore their effect was anti-dilutive. Potentially dilutive options to purchase common stock as of December 31, 2020, 2019 and 2018 had exercise prices ranging from $0.79 to $3.95, $0.79 to $4.20 and $0.79 to $4.64, respectively.

SEGMENT INFORMATION.

Management has determined that the Company operates in one reportable segment, which is the development and commercialization of pharmaceutical products.

RECLASSIFICATIONS.

Certain prior year amounts in the consolidated financial statements have been reclassified to conform to the current year presentation.

RECENTLY ISSUED ACCOUNTING STANDARDS.

In November 2018, the FASB issued ASU

2018-18,

Collaborative Arrangements (Topic 808)

, which amends ASC 808 to clarify when transactions between participants in a collaborative arrangement under ASC 808 are within the scope of ASU

2014-09

(codified in ASC 606). The amendments require the application of ASC 606 existing guidance to determine the units of account that are distinct in a collaborative arrangement for purposes of identifying transactions with customers. If a unit of account within the collaborative arrangement is distinct and is with a customer, an entity shall apply the guidance in Topic 606 to that unit of account. In a transaction between collaborative participants, an entity is precluded by ASU

2018-18

from presenting a transaction together with “revenue from contracts with customers” unless the unit of account is within the scope of ASC 606 and the entity applies the guidance in ASC 606 to such unit of account. The Company adopted the new standard on January 1, 2020. The Company has a collaboration agreement with Endo Ventures Limited (Endo). See Note 7 (Collaborative Arrangement). However, these amendments did not have an impact on the Company’s consolidated financial statements, as Endo does not meet the definition of a customer.

In June 2016, the FASB issued ASU

No. 2016-13,

Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments

. The standard amends the impairment model by requiring entities to use a forward-looking approach based on expected losses to estimate credit losses for most financial assets and certain other instruments that aren’t measured at fair value through net income. For

available-for-sale

debt securities, entities will be required to recognize an allowance for credit losses rather than a reduction in carrying value of the asset. Entities will no longer be permitted to consider the length of time that fair value has been less than amortized cost when evaluating when credit losses should be recognized. The Company adopted the new standard on January 1, 2020. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements.

In August 2018, the FASB issued ASU

2018-15,

Intangibles – Goodwill and Other –

Internal-Use

Software (Subtopic

350-40),

Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract

. The amendments in this update align the requirements for capitalizing implementation costs incurred in a hosting arrangement that is a services contract with the requirements for capitalizing implementation costs incurred to develop or obtain

internal-use

software (and hosting arrangements that include an

internal-use

software license). Accordingly, the amendments in this update require an entity in a hosting arrangement that is a service contract to follow the guidance in Subtopic

350-40

to determine which implementation costs to capitalize as an asset related to the service contract and which costs to expense. The Company adopted the new standard on January 1, 2020 and applied prospectively to all implementation costs incurred after the date of adoption. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements.

The Securities and Exchange Commission, or SEC, issued a final rule on November 19, 2020, adopting amendments to Regulation

S-K

to modernize, simplify and enhance certain disclosure requirements. The amendments reflect a principles-based, registrant-specific approach to disclosure intended to enhance the focus of financial disclosures on material information for the benefit of investors, while simplifying compliance efforts for registrants. Registrants are required to comply with the rule beginning with the first fiscal year ending on or after August 9, 2021, or the mandatory compliance date. Although registrants are not required to apply the amended rules until the mandatory compliance date, they may comply with the final amendments any time after the effective date, so long as disclosure responsive to an amended item is provided in its entirety. The Company early adopted these amendments, effective for this report.

In December 2019, the FASB issued ASU 2019-12,

Income Taxes: Simplifying the Accounting for Income Taxes

, a new standard intended to simplify the accounting for income taxes by eliminating certain exceptions related to the approach for intraperiod tax allocation, the methodology for calculating income taxes in an interim period and the recognition of deferred tax liabilities for outside basis differences. The new standard also simplifies aspects of the accounting for franchise taxes and enacted changes in tax laws or rates and clarifies the accounting for transactions that result in a

step-up

in the tax basis of goodwill. The standard is effective for annual periods beginning after December 15, 2020 and interim periods within, with early adoption permitted. Adoption of the standard requires certain changes to be made prospectively, with some changes to be made retrospectively. The Company is evaluating the impact of the standard on its financial condition and results of operations.

Investments

12 Months Ended

Dec. 31, 2020

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 December 31, 2020:
U.S. Treasuries - Cash equivalents	$	104,994,400	$	2,709	$	—	$	104,991,691
Bond Funds - ST		10,041,068		28,623		—		10,012,445

Total	$	115,035,468	$	31,332	$	—	$	115,004,136

At December 31, 2019:
U.S. Treasuries - Cash equivalents	$	59,932,200	$	2,042	$	—	$	59,930,158
U.S. Treasuries - ST		5,007,050		7,463		—		4,999,587

Total	$	64,939,250	$	9,505	$	—	$	64,929,745

There were no realized gains or losses from

available-for-sale

securities for the years ended December 31, 2020, 2019 or 2018.

The Company did not hold any securities in an unrealized loss position for more than 12 months as of December 31, 2020.

The estimated fair values of

available-for-sale

securities at December 31, 2020, by contractual maturity, are summarized as follows:


	2020
Due in one year or less	$	115,035,468

Prepaid Expenses and Other Current Assets

12 Months Ended

Dec. 31, 2020

Text Block [Abstract]

Prepaid Expenses and Other Current Assets

4.	Prepaid Expenses and Other Current Assets.

Prepaid expenses and other current assets consist of the following as of December 31:


	2020		2019
Prepaid manufacturing costs	$	3,327,610	$	1,526,013
Prepaid tax		1,368,464		—
Prepaid insurance		1,285,104		1,263,129
Prepaid subscriptions fees		729,065		501,251
Prepaid research fees		453,034		481,057
Prepaid commercialization expenses		199,095		62,959
Due from collaborative arrangements		436,784		—
Other		528,615		516,665

Total prepaid expenses and other current assets	$	8,327,771	$	4,351,074

Operating Leases

12 Months Ended

Dec. 31, 2020

Disclosure of Operating Leases [Abstract]

Operating Leases

5.	Operating Leases.

The Company has operating lease agreements for its corporate office. The leases include options to extend the leases for up to 1 year and options to terminate the lease within 1 year. There are no obligations under finance leases.

The Company entered into an agreement in May 2020 that amended its lease for its office facilities. Under the amended lease, the Company’s leased space will increase from approximately 7,800 square feet of space to approximately 10,700

square feet of space. The amended lease commenced in early 2021 when construction of the asset was completed and space became available for use. Consequently, the Company will record the effects of the amended lease during Q1 2021

which will be material to the Company’s consolidated financial statements. The lease disclosures for the year ended December 31, 2020 in these financial statements have been adjusted for the modification of the current lease.

The components of lease expense for the old leased space were as follows:


	For the Year Ended December 31, 2020
Operating lease cost	$	261,736

Supplemental cash flow information related to leases was as follows:


	December 31, 2020
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$	339,605
Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$	55,801

Supplemental balance sheet information related to leases was as follows:


	December 31, 2020
Operating lease right-of-use assets	$	—

Other current liabilities	$	28,772
Operating lease liabilities, net of current portion		—

Total operating lease liabilities	$	28,772


Weighted average remaining lease term	0.1 years
Weighted average discount rate	3.68	%

Remaining payments of lease liabilities as o

December 31, 2020 were as follows:


2021	$	28,860
2022		—

Total lease payments		28,860
Less imputed interest		(88	)

Total	$	28,772

Rent expense was $278,753, $318,346 and $242,155 for the years ended December 31, 2020, 2019 and 2018.

Accrued Expenses and Other Liabilities

12 Months Ended

Dec. 31, 2020

Payables and Accruals [Abstract]

Accrued Expenses and Other Liabilities

6.	Accrued Expenses and Other Liabilities.

Accrued expenses and other liabilities consist of the following as of December 31:


	2020		2019
Accrued preclinical and clinical trial expenses	$	584,502	$	1,183,513
Accrued professional fees		1,883,880		1,241,526
Accrued compensation and benefits		3,991,056		3,064,645
Accrued license fees		10,372,642		8,751,991
Accrued purchases		258,067		1,313,310
Accrued contributions		310,000		1,535,000
Operating lease liability		28,772		300,518
Accrued variable consideration		964,316		884,764
Accrued income tax		—		1,533,696
Other		107,032		172,332

Current accrued expenses and other liabilities		18,500,267		19,981,295

Lease liability – non-current		—		647,532

Non-current accrued expenses and other liabilities		—		647,532

Total accrued expenses and other liabilities	$	18,500,267	$	20,628,827

Collaborative Arrangements

12 Months Ended

Dec. 31, 2020

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Collaborative Arrangements

7.	Collaborative Arrangements .

In December 2018, the Company entered into a collaboration and license agreement (Collaboration) with Endo, for the further development and commercialization of generic Sabril

(vigabatrin) tablets through Endo’s U.S. Generic Pharmaceuticals segment, doing business as Par Pharmaceutical.

Under the Collaboration, Endo assumes all development, manufacturing, clinical, regulatory, sales and marketing costs under the collaboration, while the Company is responsible for exercising commercially reasonable efforts to develop, or cause the development of, a final finished, stable dosage form of generic Sabril

tablets.

Under the terms of the Collaboration, the Company has received an

up-front

payment, and will receive a milestone payment, and a sharing of defined net profits upon commercialization from Endo consisting of a

mid-double-digit

percent of net sales of generic Sabril

. The Company has also agreed to a sharing of certain development expenses. Unless terminated earlier in accordance with its terms, the collaboration continues in effect until the date that is

ten

years following the commercial launch of the product.

The collaborative agreement provides for a $2.0 million milestone payment on the commercial launch of the product by Par. As of December 31, 2020, 2019, and 2018, no milestone payments have been earned.

Revenues from collaborative arrangement with Endo for years ended December 31, 2020, 2019, and 2018 were $0, $0 and $500,000 (for upfront license fees). Total expenses incurred, net, in connection with the collaborative agreement with Endo for years ended December 31, 2020, 2019, and 2018 were approximately

$4,254, $65,061 and $0,

respectively. These expenses have been included in research and development expenses in the accompanying consolidated statements of operations and comprehensive income (loss).

In August 2020, the Company entered into a collaboration and license agreement with KYE Pharmaceuticals Inc (KYE), for the commercialization of Firdapse

in Canada.

Under the agreement, KYE assumes all selling and marketing costs under the collaboration, while the Company is responsible for supply of Firdapse

based on the collaboration partner’s purchase orders.

Under the terms of the agreement, the Company will receive an up-front payment, received payment upon transfer of Marketing Authorization and delivery of commercial product, received payment for supply of Firdapse®, will receive milestone payments, and a sharing of defined net profits upon commercialization from KYE consisting of a mid-double-digit percent of net sales of Firdapse®. The Company has also agreed to a sharing of certain development expenses. Unless terminated earlier in accordance with its terms, the collaboration continues in effect until the date that is

ten

years following the commercial launch of the product in Canada.

Revenues from arrangement with KYE for the year ended December 31, 2020 were approximately

$332,000

Total expenses incurred, net, in connection with the agreement with KYE for the year ended December 31, 2020 were approximately

$257,500.

These expenses have been included in selling, general and administrative expenses in the accompanying consolidated statements of operations and comprehensive income (loss).

The agreement provides for event-based payments subject to achievement of specified development, regulatory and sales-based milestones.

Commitments and Contingencies

12 Months Ended

Dec. 31, 2020

Commitments and Contingencies Disclosure [Abstract]

Commitments and Contingencies

8.	Commitments and Contingencies.

In 2018, the Company became aware that certain patents granted to Northwestern University (which patents have been licensed by Northwestern to a third party) for a new GABA aminotransferase inhibitor were developed from

CPP-115,

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. Under the settlement agreement, the Company received a $100,000

payment on May 21, 2019, which is reported as income in other income, net in the consolidated statement of operations and comprehensive income (loss). The Company is also entitled to receive certain contingent compensation that will be reported when and if received.

In May 2019, the FDA approved a New Drug Application (NDA) for Jacobus Pharmaceuticals for Ruzurgi

, their version of amifampridine (3,4-DAP), for the treatment of pediatric LEMS patients (ages 6 to under 17). The Company believes that Jacobus is offering Ruzurgi

at a lower price than the Company is offering Firdapse

. While the NDA for Ruzurgi

only covers pediatric patients, the Company believes that Ruzurgi

is being prescribed off label to adult LEMS patients. If Jacobus is able to successfully sell Ruzurgi

off-label to adult LEMS patients, it could have a material adverse effect on the Company’s business, financial condition and results of operations.

The Company believes that the FDA’s approval of Ruzurgi

violated its statutory rights and was in multiple other respects arbitrary, capricious and contrary to law. As a result, in June 2019 the Company filed suit against the FDA and several related parties challenging this approval and related drug labeling. The Company’s complaint, which was filed in the federal district court for the Southern District of Florida,

alleged

that the FDA’s approval of Ruzurgi

violated multiple provisions of FDA regulations regarding labeling, resulting in misbranding in violation of the Federal Food, Drug, and Cosmetic Act (FDCA); violated its statutory rights to Orphan Drug Exclusivity and New Chemical Entity Exclusivity under the FDCA; and was in multiple other respects arbitrary, capricious, and contrary to law, in violation of the Administrative Procedure Act. Among other remedies, the suit

sought

an order vacating the FDA’s approval of Ruzurgi

. Jacobus has intervened in the case.

On July 30, 2020, the Magistrate Judge considering the Company’s lawsuit against the FDA filed a Report and Recommendation in which she recommended to the District Judge handling the case that she grant the FDA’s and Jacobus’ motions for summary judgment and deny the Company’s motion for summary judgment. On September 29, 2020, the District Judge adopted the Report and Recommendation of the Magistrate Judge, granted the FDA’s and Jacobus’s motions for summary judgment, and dismissed the Company’s case. The Company has appealed the decision to the Eleventh Circuit Court of Appeals. There can be no assurance as to the outcome of this lawsuit.

On August 10, 2020, Health Canada issued a Notice of Compliance (NOC) to Medunik for Ruzurgi

for the treatment of LEMS. The Company has since initiated a legal proceeding in Canada seeking judicial review of Health Canada’s decision to issue the NOC for Ruzurgi

as incorrect and unreasonable under Canadian law. Data protection, per Health Canada regulations, is supposed to prevent Health Canada from issuing a NOC to a drug that directly or indirectly references an innovative drug’s data, for eight years from the date of the innovative drug’s approval. The Ruzurgi

Product

Monograph clearly references pivotal nonclinical carcinogenicity and reproductive toxicity data for amifampridine phosphate developed by the Company. As such, the Company believes that its data was relied upon to establish the nonclinical safety profile of Ruzurgi

needed to meet the standards of the Canadian Food and Drugs Act. There can be no assurance of the result of this proceeding.

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

12 Months Ended

Dec. 31, 2020

Text Block [Abstract]

Agreements

9.	Agreements.

LICENSE AGREEMENT WITH BIOMARIN (FIRDAPSE

On October 26, 2012, the Company entered into a license agreement with BioMarin Pharmaceutical, Inc. (BioMarin) for the North American rights to Firdapse

. Under the license agreement, the Company pays: (i) royalties to the licensor for seven years from the first commercial sale of Firdapse

equal to 7% of net sales (as defined in the license agreement) in North America for any calendar year for sales up to $100 million, and 10% of net sales in North America in any calendar year in excess of $100 million; and (ii) royalties to the third-party licensor of the rights sublicensed to the Company for seven years from the first commercial sale of Firdapse

equal to 7% of net sales (as defined in the license agreement between BioMarin and the third-party licensor) in any calendar year

for the duration of any regulatory exclusivity within a territory and 3.5% for territories in any calendar year in territories without regulatory exclusivity

On May 29, 2019, the Company entered into an amendment to its license agreement for Firdapse

. Under the amendment, the Company has expanded its commercial territory for Firdapse

, which originally was comprised of North America, to include Japan. Additionally, the Company has an option to further expand its territory under the license agreement to include most of Asia, as well as Central and South America, upon the achievement of certain milestones in Japan. Under the amendment, the Company will pay royalties on net sales in Japan of a similar percentage to the royalties that the Company is currently paying under its original license agreement for North America.

In January 2020, the Company was advised that BioMarin has transferred certain rights under the license agreement to SERB S.A.

AGREEMENTS FOR DRUG MANUFACTURING, DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES.

The

Company has entered into agreements with contract manufacturers for the manufacture of commercial drug and 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, most of these agreements are cancellable at any time, but obligate the Company to reimburse the providers for any time or costs incurred through the date of termination.

Income Taxes

12 Months Ended

Dec. 31, 2020

Income Tax Disclosure [Abstract]

Income Taxes

10.	Income Taxes.

The Company is subject to income taxes in the U.S. federal jurisdiction and various states jurisdictions.

For the year ended December 31, 2018, due to the Company’s historical operating losses and the inability to recognize any income tax benefit, there was no provision for income taxes presented in these financial statements for that respective period. Commencing in 2019, the Company has recorded income taxes on their pretax income using an effective tax rate.

The income tax expense (benefit) for the years ended December 31, 2020, 2019, and 2018 consists of:


	2020			2019		2018

Current	$	(121,662	)	$	1,533,696	$	—
Deferred		(32,971,264	)		—		—

	$	(33,092,926	)	$	1,533,696	$	—

The reconciliation of

ncome tax expense (benefit) computed at the statutory federal income tax rate of 21% to amounts included in the statements of operations is as follows:


	2020			2019			2018
Statutory rate		21.0	%		21.0	%		21.0	%
State tax		2.2	%		6.5	%		4.2	%
Valuation allowanc e		(99.4	)%		(20.9	)%		(25.9	)%
Tax credit		(2.4	)%		(2.5	)%		1.4	%
Othe r		(0.4	)%		0.5	%		(0.7	)%

		(79.0	)%		4.6	%		0.0	%

Deferred tax assets and liabilities reflect the net tax effects of net operating loss and tax credit carryovers and the temporary differences between the carrying amounts of assets and liabilities for financial reporting and the amounts used for income tax purposes. Significant components of the Company’s deferred tax assets as of December 31, 2020 and 2019 are as follows:


	2020			2019
Net operating loss	$	2,319,848		$	10,645,128
Start-up costs		11,203,034			12,894,926
Tax credits		15,615,681			14,320,860
Deferred compensation		3,889,133			3,183,767
Inventory		212,045			229,050
Prepaid expenses		(398,979	)		—
Other		130,502			355,023

Gross deferred tax asset		32,971,264			41,628,754

Valuation allowance		—			(41,628,754	)

Net deferred tax assets	$	32,971,264		$	—

The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. As of December 31, 2020, the Company determined that there is sufficient positive evidence to conclude that it is more likely than not that the above deferred taxes of approximately $33.0 million are realizable. Accordingly, the Company has released the full valuation allowance for deferred tax assets including

net operating loss

and tax credit carryover as of

December 31, 2020. The valuation allowance released over the course of the year ended December 31, 2020 was

$41.6 million.

At December 31, 2020 and 2019, respectively, the Company had federal net operating loss carryforwards of approximately

$3 million and $45 million available to reduce future taxable income.

The federal net operating loss carryforwards are expected to be utilized within the next year. Additionally, at December 31, 2020 and 2019, respectively, the Company had state net operating loss carryforwards of approximately $42 million and $52 million available to reduce future Florida taxable income. The state

net operating loss carryforwards will expire at various dates beginning in 2033.

During 2020, the Company completed an analysis to determine whether, as a result of prior ownership changes, the utilization of certain net operating loss and orphan drug tax credit carryforwards would be subject to annual limitations under Sections 382 and 383 of the Internal Revenue Code and similar state provisions. In this analysis, the Company determined that the total net operating loss and orphan drug tax credit carryforwards are fully utilizable. Thus, the deferred tax assets were adjusted accordingly.

Beginning in 2010, the Company has received several orphan drug designations by the FDA for products currently under development. The orphan drug designations allow the Company to claim increased federal tax credits for certain research and development activities. The orphan drug credit carryforwards will expire at various dates beginning in 2031.

interest or penalties were accrued through December 31, 2020. The Company’s policy is to recognize any related interest or penalties in income tax expense. The Company is not currently under income tax examinations by any tax authorities.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

+ References + Details

Stockholders' Equity

12 Months Ended

Dec. 31, 2020

Equity [Abstract]

Stockholders' Equity

11.	Stockholders’ Equity.

Preferred Stock

The Company has 5,000,000 shares of authorized preferred stock, $0.001 par value per share, at December 31, 2020 and 2019. No shares of preferred stock were outstanding at December 31, 2020 and 2019.

Common Stock

On August 20, 2020, the Company’s stockholders approved an increase in the Company’s authorized common stock par value $0.001 per share, from 150,000,000 shares to 200,000,000 shares. At December 31, 2020 and 2019, 103,781,641 and 103,397,033 shares, respectively, of common stock were issued and outstanding. Each holder of common stock is entitled to one vote of each share of common stock held of record on all matters on which stockholders generally are entitled to vote.

2020 Shelf Registration Statement

On July 23, 2020, the Company filed a shelf registration statement with the SEC to sell up to $200 million of common stock, preferred stock, warrants to purchase common stock, debt securities and units consisting of one or more of such securities (the “2020 Shelf Registration Statement”). The 2020 Shelf Registration Statement (file no.

333-240052)

was declared effective by the SEC on July 31, 2020. As of the date of this report, no offerings have been completed under the Company’s 2020 Shelf Registration Statement.

Stockholder Rights Plan

On September 20, 2011, the Board of Directors approved the Company’s adoption of a Stockholder Rights Plan. Under the Stockholders’ Rights Plan, a dividend of one preferred share purchase right (a Right) was declared for each share of common stock of the Company that was outstanding on October 7, 2011. Each Right entitles the holder to purchase from the Company one

one-hundredth

of a share of Series A Junior Preferred Stock at a purchase price of $7.80, subject to adjustment.

The Rights trade automatically with the common stock and will not be exercisable until a person or group has become an “acquiring person” by acquiring 17.5% or more of the Company’s outstanding common stock, or a person or group commences, or publicly announces a tender offer that will result in such a person or group owning 17.5% or more of the Company’s outstanding common stock. Upon announcement that any person or group has become an acquiring person, each Right will entitle all rightholders (other than the acquiring person) to purchase, for the exercise price of $7.80, a number of shares of the Company’s common stock having a market value equal to twice the exercise price. Rightholders would also be entitled to purchase common stock of the acquiring person having a value of twice the exercise price if, after a person had become an acquiring person, the Company were to enter into certain mergers or other transactions. If any person becomes an acquiring person, the Board of Directors may, at its option and subject to certain limitations, exchange one share of common stock for each Right.

The Rights have certain anti-takeover effects, in that they would cause substantial dilution to a person or group that attempts to acquire a significant interest in the Company on terms not approved by the Board of Directors. In the event that the Board of Directors determines a transaction to be in the best interests of the Company and its stockholders, the Board of Directors may redeem the Rights for $0.001 per share at any time prior to a person or group becoming an acquiring person.

On September 19, 2016, the Board of Directors unanimously approved, and on the same date the Company entered into Amendment No. 1 to the Stockholders Rights Plan (the “Amendment”). Under the terms of the Amendment, the outside expiration date of the rights plan

was

extended to September 20, 2019. Additionally, as part of the Amendment, the Board adopted a Certificate of Designation, Preferences and Rights of Series A Junior Participating Preferred Stock of the Company to increase the number of shares of Series A Junior Participating Preferred Stock of the Company available for issuance under the Rights Plan from 500,000 shares to 1.5 million shares.

On August 28, 2019, the Board of Directors unanimously adopted Amendment No. 2 to the Stockholders’ Rights Plan further extending the outside expiration date of the rights plan to September 20, 2022.

Stock Compensation

12 Months Ended

Dec. 31, 2020

Share-based Payment Arrangement [Abstract]

Stock Compensation

12.	Stock Compensation.

For the years ended December 31, 2020, 2019 and 2018, the Company recorded stock-based compensation expense as follows:


	2020		2019		2018
Research and development	$	1,585,202	$	1,137,596	$	1,079,230
Selling, general and administrative		4,675,721		2,687,219		2,471,414

Total stock-based compensation	$	6,260,923	$	3,824,815	$	3,550,644

The Company may issue stock options, restricted stock, stock appreciation rights and restricted stock units (collectively, the “Awards”) to employees, directors, and consultants of the Company under the 2014 and 2018 Stock Incentive Plans (the 2014 Plan and the 2018 Plan or collectively, the Plans). At December 31, 2020, no shares remain available for future issuance under the 2014 Plan. Under the 2018 Plan, 10,000,000 shares were reserved for issuance and as of December 31, 2020, 1,856,008 shares remain available for future issuance.

Stock Options

The Company has granted stock options to employees, officers, directors, and consultants generally at exercise prices equal to the market price of the common stock at grant date. Option awards generally vest over a period of 1 to 3 years of continuous service and have contractual terms of 7 years. Certain awards provide for accelerated vesting if there is a change in control. The Company issues new shares as shares are required to be delivered upon exercise of outstanding stock options.

During the years ended December 31, 2020, 2019, and 2018, options to purchase 281,762, 654,332 and 186,665 shares

, respectively,

of the Company’s common stock were exercised with gross proceeds to the Company of $758,130, $1,116,242, and $297,563, respectively.

During

the

years

ended December 31, 2020

and 2018,

no options to purchase shares of the Company’s common stock were exercised on a “cashless” basis. During the year ended December 31, 2019, options to purchase 6,666 shares of the Company’s common stock were exercised on a “cashless” basis, resulting in the issuance of an aggregate of 3,444 shares of the Company’s common stock.

During the years ended December 31, 2020, 2019, and 2018 the Company recorded

non-cash

stock-based compensation expense related to stock options totaling $5,694,120, $3,780,086, and $3,535,647, respectively.

During the years ended December 31, 2020, 2019, and 2018, the Company granted seven-year options to purchase an aggregate of 2,715,000, 2,183,500 and 5,882,500 shares, respectively, of the Company’s common stock to certain of the Company’s officers, employees, directors, and consultants.

Stock option activity under the Company’s Plans for the year ended December 31, 2020 is summarized as follows:


	Number of Options			Weighted Average Exercise Price		Weighted Average Remaining Contractual Term (in years)		Aggregate Intrinsic Value
Outstanding at beginning of year		11,478,334		$	2.95
Granted		2,715,000			3.75
Exercised or released		(281,762	)		2.69
Forfeited or cancelled		(160,668	)		3.84
Expired		(357,235	)		3.38

Outstanding at end of year		13,393,669		$	3.10		4.42	$	8,237,645

Exercisable at end of year		7,926,136		$	2.64		3.38	$	7,381,601

Other information pertaining to stock option activity during the years ended December 31, 2020, 2019, and 2018 was as follows:


	2020		2019		2018
Weighted–average fair value of granted stock options	$	2.33	$	2.69	$	1.93
Total fair value of vested stock options	$	5,311,616	$	3,864,995	$	2,193,294
Total intrinsic value of exercised stock options	$	325,102	$	1,899,862	$	274,864

As of December 31, 2020, there was approximately $10.02 million of unrecognized compensation expense related to

non-vested

stock option awards granted under the Plans. That cost is expected to be recognized over a weighted average period of approximately 2.35 years.

The Company utilizes the Black-Scholes option-pricing model to determine the fair value of stock options on the date of grant. This model derives the fair value of stock options based on certain assumptions related to the expected stock price volatility, expected option life, risk-free interest rate and dividend yield. Expected volatility is based on reviews of historical volatility of the Company’s common stock. The estimated expected option life is based upon estimated employee exercise patterns and considers whether and the extent to which the options are

in-the-money.

The Company estimates the expected option life for options granted to employees and directors based upon the simplified method. Under this method, the expected life is presumed to be the

mid-point

between the vesting date and the end of the contractual term. The Company will continue to use the simplified method until it has sufficient historical exercise data to estimate the expected life of the options. The risk-free interest rate assumption is based upon the U.S. Treasury yield curve appropriate for the estimated life of the stock options awards. The expected dividend rate is zero. Forfeitures are recognized as a reduction of stock-based compensation expense as they occur.

Assumptions used during the years were as follows:


	December 31, 2020			December 31, 2019			December 31, 2018
Risk free interest rate		0.24% to 1.64	%		1.51% to 2.53	%		2.09% to 2.88	%
Expected term		4.5 years			4.5 years			0 to 7 years
Expected volatility		80.5% to 83.7	%		75.5	%		82	%
Expected dividend yield		—	%		—	%		—	%
Expected forfeiture rate		—	%		—	%		—	%

Restricted Stock Units

Under the 2018 Plan, participants may be granted restricted stock units, each of which represents a conditional right to receive shares of common stock in the future. The restricted stock units granted under this plan generally vest ratably over a three-year period. Upon vesting, the restricted stock units will convert into an equivalent number of shares of common stock. The amount of expense relating to the restricted stock units is based on the closing market price of the Company’s common stock on the date of grant and is amortized on a straight-line basis over the requisite service period. Restricted stock unit activity during 2020 was as follows:


	2020
	Number of Restricted Stock Units			Weighted Average Grant Date Fair Value
Nonvested balance at beginning of year		352,500		$	4.64
Granted		30,000			4.70
Vested		(117,495	)		4.64
Forfeited		(29,334	)		4.64

Nonvested balance at end of year		235,671		$	4.65

During the year ended December 31, 2019, 352,500

estricted stock units were granted and outstanding and there were no vested or forfeited shares. There was no restricted stock unit activity during 2018.

During the year ended December 31, 2020 and 2019, the Company recorded

non-cash

stock-based compensation expense related to restricted stock units totaling $566,803 and $44,729, respectively. No stock-based compensation related to restricted stocks was recorded during 2018.

Common Stock

No shares of common stock were granted during the year ended December 31, 2020 or 2019. During the year ended December 31, 2018, the Company granted 3,094 net shares of common stock to an employee as compensation. The Company recorded stock-based compensation related to common stock issued to an employee totaling approximately $15,000, during the year ended December 31, 2018.

Benefit Plan

12 Months Ended

Dec. 31, 2020

Retirement Benefits [Abstract]

Benefit Plan

13.	Benefit Plan.

The Company maintains an employee savings plan pursuant to Section 401(k) of the Internal Revenue Code covering all eligible employees. Subject to certain dollar limits, eligible employees may contribute up to 15% of their

pre-tax

annual compensation to the plan. The Company has elected to make discretionary matching contributions of employee contributions up to 4% of an employee’s gross salary. For the years ended December 31, 2020, 2019, and 2018, the Company’s matching contributions were approximately $465,000, $268,000 and $123,000, respectively.

- Definition

The entire disclosure for retirement benefits.

+ References

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 55
-Paragraph 17
-URI http://asc.fasb.org/extlink&oid=118257860&loc=d3e4179-114921

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section S99
-Paragraph 2
-URI http://asc.fasb.org/extlink&oid=21916913&loc=d3e273930-122802

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (d)(i)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (o)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (p)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 70
-Section 50
-Paragraph 1
-URI http://asc.fasb.org/extlink&oid=49170846&loc=d3e28014-114942

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-URI http://asc.fasb.org/topic&trid=2235017

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 60
-Section 50
-Paragraph 3
-Subparagraph (c)
-URI http://asc.fasb.org/extlink&oid=6414203&loc=d3e39689-114964

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (d)(iv)(03)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (q)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (r)(1)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (d)(iii)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (l)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 715
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (r)(2)
-URI http://asc.fasb.org/extlink&oid=118255775&loc=d3e1928-114920

+ Details

Basis of Presentation and Significant Accounting Policies (Policies)

12 Months Ended

Dec. 31, 2020

Accounting Policies [Abstract]

PRINCIPLES OF CONSOLIDATION.

USE OF ESTIMATES

USE OF ESTIMATES.

CASH AND CASH EQUIVALENTS

CASH AND CASH EQUIVALENTS.

INVESTMENTS

INVESTMENTS.

The short-term bond funds and U.S. Treasuries held at December 3

, 2020 are classified as

available-for-sale

non-current

investments in its consolidated balance sheets.

The Company records

available-for-sale

securities. See Note 3 (Investments).

investments classified as trading securities, as the Company sold its interest in the short-term bond fund classified as trading securities in 2019

ACCOUNTS RECEIVABLE, NET

ACCOUNTS RECEIVABLE, NET.

INVENTORY

. Inventories are stated at the lower of cost or net realizable value

Inventories consist of raw materials,

work-in-process

and finished goods. Costs to be capitalized as inventories primarily include third party manufacturing costs and other overhead costs.

Cost is determined using a standard cost method, which approximates actual cost, and assumes a first-in, first out (FIFO) flow of goods.

The Company began capitalizing inventories post FDA approval of Firdapse

on November 28, 2018 as the related costs were expected to be recoverable through the commercialization of the product. Costs incurred prior to the FDA approval of Firdapse

work-in-process

and finished goods.

Products that have been approved by the FDA or other regulatory authorities, such as Firdapse

PREPAID EXPENSES AND OTHER CURRENT ASSETS

PREPAID EXPENSES AND OTHER CURRENT ASSETS.

pre-clinical

PROPERTY AND EQUIPMENT, NET

PROPERTY AND EQUIPMENT,

NET.

FAIR VALUE OF FINANCIAL INSTRUMENTS

FAIR VALUE OF FINANCIAL INSTRUMENTS.

FAIR VALUE MEASUREMENTS

FAIR VALUE MEASUREMENTS.


	Fair Value Measurements at Reporting Date Using
	Balances as of December 31, 2020		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	15,673,626	$	15,673,626	$	—	$	—

U.S. Treasuries	$	104,994,400	$	—	$	104,994,400	$	—

Short-term investments:
Short-term bond funds	$	10,041,068	$	10,041,068	$	—	$	—


	Balances as of December 31, 2019		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	23,963,617	$	23,963,617	$	—	$	—

U.S. Treasuries	$	59,932,200	$	—	$	59,932,200	$	—

Short-term investments:
U.S. Treasuries	$	5,007,050	$	—	$	5,007,050	$	—

OPERATING LEASES

OPERATING LEASES.

The Company determines if an arrangement is a lease at inception. Operating leases are included in operating lease

right-of-use

non-lease

components, which are generally accounted for separately.

REVENUE RECOGNITION

REVENUE RECOGNITION.

in November 2018. Subsequent to receiving FDA approval, the Company entered into an arrangement with one distributor (the “Customer”), which is the exclusive distributor of Firdapse

in the United States. The Customer subsequently resells Firdapse

Product Revenue, Net:

The Company sells Firdapse

to the Customer (its exclusive distributor) who subsequently resells Firdapse

Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods and are recorded in cost of sales.

During the years ended December 31, 2020 and 2019, all of the Company’s sales of Firdapse

in the United States were to its Customer.

Reserves for Variable Consideration:

Trade Discounts and Allowances:

Prompt Payment Discounts:

Funded

Co-pay

Assistance Program:

The Company contracts with a third-party to manage the

co-pay

assistance program intended to provide financial assistance to qualified commercially-insured patients. The calculation of the accrual for

co-pay

assistance is based on an estimate of claims and the cost per claim that the Company expects to receive associated with Firdapse

Product Returns:

Provider Chargebacks and Discounts:

Government Rebates:

Bridge and Patient Assistance Programs:

The Company provides free Firdapse

to uninsured patients who satisfy

pre-established

while the Company is determining the patient’s third-party insurance, prescription drug benefit or other third-party coverage for Firdapse

. The Patient Assistance Program provides free Firdapse

for longer periods of time for those who are uninsured or functionally uninsured with respect to Firdapse

Revenues from Collaborative Arrangements:

The Company has entered into collaboration agreements for the further development and commercialization of generic Sabril

(vigabatrin) tablets as well as the commercialization of Firdapse

net-profit

sharing payments on sales of products resulting from the collaborative agreement.

in Canada.

sales-based

royalties or net profit-sharing when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the

sales-based

royalty or net profit-sharing has been satisfied.

33,000

from sales of the collaborative product in Canada. For the years ended December

2019

and

2018

, there was

profit-sharing revenue from sales of the collaborative product.

Refer to Note 7 (Collaborative Arrangements), for further discussion on the Company’s collaborative arrangements.

RESEARCH AND DEVELOPMENT

RESEARCH AND DEVELOPMENT.

STOCK-BASED COMPENSATION

STOCK-BASED COMPENSATION.

CONCENTRATION OF CREDIT RISK

CONCENTRATION OF RISK.

, and expects Firdapse

to constitute virtually all of product revenue for the foreseeable future. The Company’s success depends on its ability to continue to effectively commercialize Firdapse

The Company relies exclusively on third parties to formulate and manufacture Firdapse

and its drug candidates. The commercialization of Firdapse

ROYALTIES

ROYALTIES.

Royalties incurred in connection with the Company’s license agreement, as disclosed in Note 9 (Agreements), are expensed to cost of sales as revenue from product sales is recognized.

INCOME TAXES

INCOME TAXES.

more-likely-than-not

The tax law changes in the CARES Act did not have a material impact on the Company’s income tax provision.

COMPREHENSIVE INCOME (LOSS)

COMPREHENSIVE INCOME (LOSS).

available-for-sale

securities.

NET INCOME (LOSS) PER COMMON SHARE

NET INCOME (LOSS) PER COMMON SHARE.

The following table reconciles basic and diluted weighted average common shares:


	For the Years Ended December 31,
	2020		2019		2018
Basic weighted average common shares outstanding		103,512,913		102,944,316		102,633,884
Effect of dilutive securities		2,729,360		3,076,620		—

Diluted weighted average common shares outstanding		106,242,273		106,020,936		102,633,884

SEGMENT INFORMATION

SEGMENT INFORMATION.

Management has determined that the Company operates in one reportable segment, which is the development and commercialization of pharmaceutical products.

RECLASSIFICATIONS

RECLASSIFICATIONS.

Certain prior year amounts in the consolidated financial statements have been reclassified to conform to the current year presentation.

RECENTLY ISSUED ACCOUNTING STANDARDS

RECENTLY ISSUED ACCOUNTING STANDARDS.

In November 2018, the FASB issued ASU

2018-18,

Collaborative Arrangements (Topic 808)

, which amends ASC 808 to clarify when transactions between participants in a collaborative arrangement under ASC 808 are within the scope of ASU

2014-09

2018-18

In June 2016, the FASB issued ASU

No. 2016-13,

Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments

available-for-sale

In August 2018, the FASB issued ASU

2018-15,

Intangibles – Goodwill and Other –

Internal-Use

Software (Subtopic

350-40),

Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract

internal-use

software (and hosting arrangements that include an

internal-use

software license). Accordingly, the amendments in this update require an entity in a hosting arrangement that is a service contract to follow the guidance in Subtopic

350-40

The Securities and Exchange Commission, or SEC, issued a final rule on November 19, 2020, adopting amendments to Regulation

S-K

In December 2019, the FASB issued ASU 2019-12,

Income Taxes: Simplifying the Accounting for Income Taxes

step-up

Basis of Presentation and Significant Accounting Policies (Tables)

12 Months Ended

Dec. 31, 2020

Accounting Policies [Abstract]

Fair Value Measurement Specific to Assets or Liability


	Fair Value Measurements at Reporting Date Using
	Balances as of December 31, 2020		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	15,673,626	$	15,673,626	$	—	$	—

U.S. Treasuries	$	104,994,400	$	—	$	104,994,400	$	—

Short-term investments:
Short-term bond funds	$	10,041,068	$	10,041,068	$	—	$	—


	Balances as of December 31, 2019		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	23,963,617	$	23,963,617	$	—	$	—

U.S. Treasuries	$	59,932,200	$	—	$	59,932,200	$	—

Short-term investments:
U.S. Treasuries	$	5,007,050	$	—	$	5,007,050	$	—

Basic and Dilutive Weighted Average Common Shares

The following table reconciles basic and diluted weighted average common shares:


	For the Years Ended December 31,
	2020		2019		2018
Basic weighted average common shares outstanding		103,512,913		102,944,316		102,633,884
Effect of dilutive securities		2,729,360		3,076,620		—

Diluted weighted average common shares outstanding		106,242,273		106,020,936		102,633,884

Investments (Tables)

12 Months Ended

Dec. 31, 2020

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 December 31, 2020:
U.S. Treasuries - Cash equivalents	$	104,994,400	$	2,709	$	—	$	104,991,691
Bond Funds - ST		10,041,068		28,623		—		10,012,445

Total	$	115,035,468	$	31,332	$	—	$	115,004,136

At December 31, 2019:
U.S. Treasuries - Cash equivalents	$	59,932,200	$	2,042	$	—	$	59,930,158
U.S. Treasuries - ST		5,007,050		7,463		—		4,999,587

Total	$	64,939,250	$	9,505	$	—	$	64,929,745

Estimated Fair Values of Available for Sale Securities

The estimated fair values of

available-for-sale

securities at December 31, 2020, by contractual maturity, are summarized as follows:


	2020
Due in one year or less	$	115,035,468

Prepaid Expenses and Other Current Assets (Tables)

12 Months Ended

Dec. 31, 2020

Text Block [Abstract]

Prepaid expenses and other current assets

Prepaid expenses and other current assets consist of the following as of December 31:


	2020		2019
Prepaid manufacturing costs	$	3,327,610	$	1,526,013
Prepaid tax		1,368,464		—
Prepaid insurance		1,285,104		1,263,129
Prepaid subscriptions fees		729,065		501,251
Prepaid research fees		453,034		481,057
Prepaid commercialization expenses		199,095		62,959
Due from collaborative arrangements		436,784		—
Other		528,615		516,665

Total prepaid expenses and other current assets	$	8,327,771	$	4,351,074

Operating Leases (Tables)

12 Months Ended

Dec. 31, 2020

Disclosure of Operating Leases [Abstract]

Lease, Cost

The components of lease expense for the old leased space were as follows:


	For the Year Ended December 31, 2020
Operating lease cost	$	261,736

Schedule of Supplemental Cash Flow Information Related To Lease

Supplemental cash flow information related to leases was as follows:


	December 31, 2020
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$	339,605
Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$	55,801

Schedule of Supplemental Balance Sheet related To Lease

Supplemental balance sheet information related to leases was as follows:


	December 31, 2020
Operating lease right-of-use assets	$	—

Other current liabilities	$	28,772
Operating lease liabilities, net of current portion		—

Total operating lease liabilities	$	28,772


Weighted average remaining lease term	0.1 years
Weighted average discount rate	3.68	%

Lessee, Operating Lease, Liability, Maturity

Remaining payments of lease liabilities as o

December 31, 2020 were as follows:


2021	$	28,860
2022		—

Total lease payments		28,860
Less imputed interest		(88	)

Total	$	28,772

Accrued Expenses and Other Liabilities (Tables)

12 Months Ended

Dec. 31, 2020

Payables and Accruals [Abstract]

Schedule of Accrued Expenses and Other Liabilities

Accrued expenses and other liabilities consist of the following as of December 31:


	2020		2019
Accrued preclinical and clinical trial expenses	$	584,502	$	1,183,513
Accrued professional fees		1,883,880		1,241,526
Accrued compensation and benefits		3,991,056		3,064,645
Accrued license fees		10,372,642		8,751,991
Accrued purchases		258,067		1,313,310
Accrued contributions		310,000		1,535,000
Operating lease liability		28,772		300,518
Accrued variable consideration		964,316		884,764
Accrued income tax		—		1,533,696
Other		107,032		172,332

Current accrued expenses and other liabilities		18,500,267		19,981,295

Lease liability – non-current		—		647,532

Non-current accrued expenses and other liabilities		—		647,532

Total accrued expenses and other liabilities	$	18,500,267	$	20,628,827

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2020

Income Tax Disclosure [Abstract]

Schedule of income tax expense

The income tax expense (benefit) for the years ended December 31, 2020, 2019, and 2018 consists of:


	2020			2019		2018

Current	$	(121,662	)	$	1,533,696	$	—
Deferred		(32,971,264	)		—		—

	$	(33,092,926	)	$	1,533,696	$	—

Reconciliation of income tax expense (Benefit) computed at statutory federal income tax rate

The reconciliation of

ncome tax expense (benefit) computed at the statutory federal income tax rate of 21% to amounts included in the statements of operations is as follows:


	2020			2019			2018
Statutory rate		21.0	%		21.0	%		21.0	%
State tax		2.2	%		6.5	%		4.2	%
Valuation allowanc e		(99.4	)%		(20.9	)%		(25.9	)%
Tax credit		(2.4	)%		(2.5	)%		1.4	%
Othe r		(0.4	)%		0.5	%		(0.7	)%

		(79.0	)%		4.6	%		0.0	%

Components of deferred tax assets

Significant components of the Company’s deferred tax assets as of December 31, 2020 and 2019 are as follows:


	2020			2019
Net operating loss	$	2,319,848		$	10,645,128
Start-up costs		11,203,034			12,894,926
Tax credits		15,615,681			14,320,860
Deferred compensation		3,889,133			3,183,767
Inventory		212,045			229,050
Prepaid expenses		(398,979	)		—
Other		130,502			355,023

Gross deferred tax asset		32,971,264			41,628,754

Valuation allowance		—			(41,628,754	)

Net deferred tax assets	$	32,971,264		$	—

Stock Compensation (Tables)

12 Months Ended

Dec. 31, 2020

Share-based Payment Arrangement [Abstract]

Stock-Based Compensation Expense

For the years ended December 31, 2020, 2019 and 2018, the Company recorded stock-based compensation expense as follows:


	2020		2019		2018
Research and development	$	1,585,202	$	1,137,596	$	1,079,230
Selling, general and administrative		4,675,721		2,687,219		2,471,414

Total stock-based compensation	$	6,260,923	$	3,824,815	$	3,550,644

Stock option activity under the Company's Plans

Stock option activity under the Company’s Plans for the year ended December 31, 2020 is summarized as follows:


	Number of Options			Weighted Average Exercise Price		Weighted Average Remaining Contractual Term (in years)		Aggregate Intrinsic Value
Outstanding at beginning of year		11,478,334		$	2.95
Granted		2,715,000			3.75
Exercised or released		(281,762	)		2.69
Forfeited or cancelled		(160,668	)		3.84
Expired		(357,235	)		3.38

Outstanding at end of year		13,393,669		$	3.10		4.42	$	8,237,645

Exercisable at end of year		7,926,136		$	2.64		3.38	$	7,381,601

Schedule of Other Information Pertaining to Stock Option Activity

Other information pertaining to stock option activity during the years ended December 31, 2020, 2019, and 2018 was as follows:


	2020		2019		2018
Weighted–average fair value of granted stock options	$	2.33	$	2.69	$	1.93
Total fair value of vested stock options	$	5,311,616	$	3,864,995	$	2,193,294
Total intrinsic value of exercised stock options	$	325,102	$	1,899,862	$	274,864

Summary of Stock Options Awards Based on Certain Assumptions

Assumptions used during the years were as follows:


	December 31, 2020			December 31, 2019			December 31, 2018
Risk free interest rate		0.24% to 1.64	%		1.51% to 2.53	%		2.09% to 2.88	%
Expected term		4.5 years			4.5 years			0 to 7 years
Expected volatility		80.5% to 83.7	%		75.5	%		82	%
Expected dividend yield		—	%		—	%		—	%
Expected forfeiture rate		—	%		—	%		—	%

Summary of Restricted Stock Unit Activity

Restricted stock unit activity during 2020 was as follows:


	2020
	Number of Restricted Stock Units			Weighted Average Grant Date Fair Value
Nonvested balance at beginning of year		352,500		$	4.64
Granted		30,000			4.70
Vested		(117,495	)		4.64
Forfeited		(29,334	)		4.64

Nonvested balance at end of year		235,671		$	4.65

Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) $ / shares in Units, shares in Millions	12 Months Ended
	Dec. 31, 2020 USD ($) Segment $ / shares shares	Dec. 31, 2019 USD ($) $ / shares shares	Dec. 31, 2018 USD ($) $ / shares shares
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Maximum maturity period of cash and cash equivalent	three months
Minimum amortization period of compensation cost on straight line basis	1 year
Maximum Amortization Period Of Compensation Cost On Straight Line Basis	3 years
Stock option exercise price range, Minimum \| $ / shares	$ 0.79	$ 0.79	$ 0.79
Stock option exercise price range, Maximum \| $ / shares	$ 3.95	$ 4.20	$ 4.64
Trading Securities, Realized Gain (Loss)		$ 80,045	$ 0
Trading securities, realized or unrealized gain (loss)	$ 0
Potential equivalent common stock excluded \| shares	7.1	4.6	10.5
Trading securities	$ 0	$ 0
Number of Reportable Segments \| Segment	1
Canada [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Revenue from collaborative product	$ 33,000	0	$ 0
Furniture and Equipment [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets	5 years
Other Income, Net [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Unrealized gain (loss), trading securities		$ 0	$ (29,430)
Minimum [Member] \| Computer Equipment [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets	3 years
Minimum [Member] \| Leasehold Improvements [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets	4 years
Maximum [Member] \| Computer Equipment [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets	5 years
Maximum [Member] \| Leasehold Improvements [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets	7 years

Basis of Presentation and Significant Accounting Policies - Fair Value Measurement Specific to Assets or Liability (Detail) - USD ($)	Dec. 31, 2020	Dec. 31, 2019
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Short-term investments	$ 10,041,068	$ 5,007,050
Short-Term Bond Fund [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Investments Fair Value Disclosure		5,007,050
Short-term investments	10,041,068
U.S. Treasuries [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	104,994,400	59,932,200
Money Market Funds [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	15,673,626	23,963,617
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] \| Short-Term Bond Fund [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Short-term investments	10,041,068
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] \| Money Market Funds [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	15,673,626	23,963,617
Significant Other Observable Inputs (Level 2) [Member] \| Short-Term Bond Fund [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Investments Fair Value Disclosure		5,007,050
Significant Other Observable Inputs (Level 2) [Member] \| U.S. Treasuries [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	$ 104,994,400	$ 59,932,200

Basis of Presentation and Significant Accounting Policies - Schedule Of Reconcile Basic And Dilutive Weighted Average Common Shares (Details) - shares	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Basic weighted average common shares outstanding	103,512,913	102,944,316	102,633,884
Effect of dilutive securities	2,729,360	3,076,620
Dilutive weighted average common shares outstanding	106,242,273	106,020,936	102,633,884

Investments - Additional Information (Detail) - USD ($)	12 Months Ended
Investments - Additional Information (Detail) - USD ($)	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Investments, Debt and Equity Securities [Abstract]
Realized gains losses from available for sale securities	$ 0	$ 0	$ 0

Investment - Summary of Available-for-Sale Investments by Security type (Detail) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019
Net Investment Income [Line Items]
Amortized cost	$ 115,004,136	$ 64,929,745
Gross Unrealized Gains	31,332	9,505
Gross Unrealized Losses
Estimated Fair Value	115,035,468	64,939,250
U.S. Treasuries - ST [Member]
Net Investment Income [Line Items]
Amortized cost		4,999,587
Gross Unrealized Gains		7,463
Estimated Fair Value		5,007,050
U.S Treasury Bond Securities Cash Equivalents [Member]
Net Investment Income [Line Items]
Amortized cost	104,991,691	59,930,158
Gross Unrealized Gains	2,709	2,042
Estimated Fair Value	104,994,400	$ 59,932,200
Bond Funds – ST [Member]
Net Investment Income [Line Items]
Amortized cost	10,012,445
Gross Unrealized Gains	28,623
Estimated Fair Value	$ 10,041,068

Investment - Estimated Fair Values of Available for Sale Securities (Detail) - USD ($)	Dec. 31, 2020	Dec. 31, 2019
Investments, Debt and Equity Securities [Abstract]
Estimated Fair Value	$ 115,035,468	$ 64,939,250

Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($)	Dec. 31, 2020	Dec. 31, 2019
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]
Prepaid manufacturing costs	$ 3,327,610	$ 1,526,013
Prepaid tax	1,368,464
Prepaid insurance	1,285,104	1,263,129
Prepaid subscriptions fees	729,065	501,251
Prepaid research fees	453,034	481,057
Prepaid commercialization expenses	199,095	62,959
Due from collaborative arrangements	436,784	0
Other	528,615	516,665
Total prepaid expenses and other current assets	$ 8,327,771	$ 4,351,074

Operating Leases - Operating Leases (Detail)	12 Months Ended
Operating Leases - Operating Leases (Detail)	Dec. 31, 2020 USD ($)
Disclosure of Operating Leases [Abstract]
Operating lease cost	$ 261,736

Operating Leases - Schedule of Supplemental Cash Flow Information Related To Lease (Detail)	12 Months Ended
	Dec. 31, 2020 USD ($)
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$ 339,605
Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$ 55,801

Operating Leases -Schedule of Supplemental Balance Sheet related To Lease (Detail) - USD ($)	Dec. 31, 2020	Dec. 31, 2019
Disclosure of Operating Leases [Line Items]
Operating lease right-of-use assets		$ 793,252
Other current liabilities	$ 28,772	300,518
Operating Lease, Liability, Current, Statement of Financial Position [Extensible List]	us-gaap:OtherAccruedLiabilitiesCurrent
Operating lease liabilities, net of current portion		$ 647,532
Total operating lease liabilities	$ 28,772
Weighted average remaining lease term	1 month 6 days
Weighted average discount rate	3.68%

Operating Leases -Lessee, Operating Lease, Liability, Maturity (Detail)	Dec. 31, 2020 USD ($)
Disclosure of Operating Leases [Line Items]
2021	$ 28,860
2022
Total lease payments	28,860
Less imputed interest	(88)
Total	$ 28,772

Operating Leases - Additional Information (Detail)	12 Months Ended
Operating Leases - Additional Information (Detail)	Dec. 31, 2020 USD ($) ft²	Dec. 31, 2019 USD ($)	Dec. 31, 2018 USD ($)
Disclosure of Operating Leases [Line Items]
Finance Lease Obligations \| $	$ 0
Lessor, Operating Lease, Option to Extend	1 year
Lessor, Operating Lease, Option to Terminate	1 year
Rent expense \| $	$ 278,753	$ 318,346	$ 242,155
Before agreement of company leased spaces \| ft²	7,800
After agreement of company leased spaces \| ft²	10,700

Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($)	Dec. 31, 2020	Dec. 31, 2019
Payables and Accruals [Abstract]
Accrued preclinical and clinical trial expenses	$ 584,502	$ 1,183,513
Accrued professional fees	1,883,880	1,241,526
Accrued compensation and benefits	3,991,056	3,064,645
Accrued license fees	10,372,642	8,751,991
Accrued purchases	258,067	1,313,310
Accrued contributions	310,000	1,535,000
Operating lease liability	28,772	300,518
Accrued variable consideration	964,316	884,764
Accrued income tax		1,533,696
Other	107,032	172,332
Current accrued expenses and other liabilities	18,500,267	19,981,295
Lease liability - non-current		647,532
Non-current accrued expenses and other liabilities		647,532
Total accrued expenses and other liabilities	$ 18,500,267	$ 20,628,827

Collaborative Arrangement - Additional Information (Detail) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Collaborative Arrangements [Line Items]
Period of collaboration agreement	10 years
Expenses In Connection With Collaborative Arrangement	$ 16,496,715	$ 18,842,752	$ 19,919,204
Revenues	119,072,803	102,306,337	500,000
Collaborative Arrangement [Member]
Collaborative Arrangements [Line Items]
Milestone payments	2,000,000.0
Milestone payments income	0	0	0
Expenses In Connection With Collaborative Arrangement	4,254	65,061	0
Revenues	$ 0	$ 0	$ 500,000
Collaborative Arrangement [Member] \| KYE Pharmaceuticals [Member] \| CA [Member]
Collaborative Arrangements [Line Items]
Period of collaboration agreement	10 years
Revenues	$ 332,000
Collaborative Arrangement [Member] \| KYE Pharmaceuticals [Member] \| CA [Member] \| Selling, General and Administrative Expenses [Member]
Collaborative Arrangements [Line Items]
Expenses In Connection With Collaborative Arrangement	$ 257,500

Commitments and Contingencies - Additional Information (Detail)	1 Months Ended
	May 21, 2019 USD ($)
Settlement Agreement [Member] \| Other Income [Member]
Commitments [Line Items]
Litigation Settlement Received	$ 100,000

Agreements - Additional Information (Detail) $ in Millions	12 Months Ended
Agreements - Additional Information (Detail) $ in Millions	Dec. 31, 2020 USD ($)
License Agreement [Line Items]
Percentage of royalty on net sales	3.50%
License Agreement with BioMarin [Member]
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
License Agreement with BioMarin [Member] \| Minimum [Member]
License Agreement [Line Items]
Percentage of royalty on net sales	7.00%
License Agreement with BioMarin [Member] \| Maximum [Member]
License Agreement [Line Items]
Percentage of royalty on net sales	10.00%

Income Taxes - Schedule of income tax expense (Detail) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Income Tax Disclosure [Abstract]
Current	$ (121,662)	$ 1,533,696	$ 0
Deferred	(32,971,264)	0	0
Total income tax expense	$ (33,092,926)	$ 1,533,696	$ 0

- Definition

Amount of current income tax expense (benefit) and deferred income tax expense (benefit) pertaining to continuing operations.

+ References + Details

Income Taxes - Additional Information (Detail) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Income Tax Disclosure [Line Items]
Deferred tax assets recognised in income statement	$ (32,971,264)	$ 0	$ 0
Increase (decrease) in valuation allowance on deferred tax assets	41,600,000
Net operating loss carryforwards	$ 3,000,000	45,000,000
Net operating loss carryforwards expiration date	beginning in 2033
Interest or penalties accrued	$ 0
Tax credit carryforwards expiration date	2031 years
State and Local Jurisdiction [Member]
Income Tax Disclosure [Line Items]
Net operating loss carryforwards	$ 42,000,000	$ 52,000,000

Income Taxes - Schedule of Reconciliation of Income Tax Expense (Benefit) Computed at Statutory Federal Income Tax Rate (Detail)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Income Tax Disclosure [Abstract]
Statutory rate	21.00%	21.00%	21.00%
State tax	2.20%	6.50%	4.20%
Valuation allowance	(99.40%)	(20.90%)	(25.90%)
Tax credit	(2.40%)	(2.50%)	1.40%
Other	(0.40%)	0.50%	(0.70%)
Reconciliation of income tax expense (benefit)	(79.00%)	4.60%	0.00%

Income Taxes - Components of Deferred Tax Assets (Detail) - USD ($)	Dec. 31, 2020	Dec. 31, 2019
Components of Deferred Tax Assets [Abstract]
Net operating loss	$ 2,319,848	$ 10,645,128
Start-up costs	11,203,034	12,894,926
Tax credits	15,615,681	14,320,860
Deferred compensation	3,889,133	3,183,767
Inventory	212,045	229,050
Prepaid expenses	(398,979)	0
Other	130,502	355,023
Gross deferred tax asset	32,971,264	41,628,754
Valuation allowance	0	(41,628,754)
Net deferred tax assets	$ 32,971,264	$ 0

Stockholders' Equity (Preferred Stock and Common Stock) - Additional Information (Detail)	Dec. 31, 2020 Vote $ / shares shares	Dec. 31, 2019 $ / shares shares
Stockholders Equity [Line Items]
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, par value \| $ / shares	$ 0.001	$ 0.001
Preferred stock, shares outstanding	0	0
Common stock, shares authorized	200,000,000	150,000,000
Common stock, par value \| $ / shares	$ 0.001	$ 0.001
Common stock, shares issued	103,781,641	103,397,033
Common stock, shares outstanding	103,781,641	103,397,033
Number of votes entitled for each share of common stock \| Vote	1

Stockholders' Equity (2020 Shelf Registration Statement) - Additional Information (Detail) $ in Millions	Jul. 23, 2020 USD ($)
2020 Shelf Registration Statement [Member]
Stockholders' Equity [Line Items]
Maximum dollar amount of common stock to be issued under shelf registration statement	$ 200

Stockholders' Equity (Stockholder Rights Plan) - Additional Information (Detail) - $ / shares	Sep. 19, 2016	Sep. 20, 2011	Dec. 31, 2020
Series A Preferred Stock [Member]
Stockholders Equity [Line Items]
Preferred Stock Purchase Price		$ 7.80
Stockholder Rights Plan [Member]
Stockholders Equity [Line Items]
Warrants exercise price		$ 7.80
Minimum percentage of outstanding stock acquired by a person or group to trigger Shareholder Rights Plan		17.50%
Stockholders Rights Plan redemption price of right		$ 0.001
Class of warrant or right expiration date, Extended	Sep. 20, 2019
Certificate Of Designation [Member] \| Series A Preferred Stock [Member]
Stockholders Equity [Line Items]
Junior participating preferred stock shares issued available for issuance under rights agreement, initial			500,000
Junior participating preferred stock shares issued available for issuance under rights agreement, amended			1,500,000

Stock Compensation - Stock-Based Compensation Expense (Detail) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	$ 6,260,923	$ 3,824,815	$ 3,550,644
Research and Development [Member]
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	1,585,202	1,137,596	1,079,230
Selling, General and Administrative [Member]
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	$ 4,675,721	$ 2,687,219	$ 2,471,414

Stock Compensation - Additional Information (Detail) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of stock options exercised	281,762
Proceeds from exercise of stock options	$ 758,130	$ 1,116,242	$ 297,563
Common stock unit granted	2,715,000
Stock option contractual term	3 years 4 months 17 days
Expected dividend rate	0.00%
Non Vested Outstanding		352,500
Non Vested granted		352,500
Employee [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Common stock unit granted			3,094
Non-cash stock-based compensation expense			$ 15,000
Stock Option [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option contractual term	7 years
Stock Option [Member] \| Minimum [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation, vesting period	1 year
Stock Option [Member] \| Maximum [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation, vesting period	3 years
Restricted Stock Units (RSUs) [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Non-cash stock-based compensation expense	$ 566,803	$ 44,729
Non Vested Outstanding	235,671	352,500
Non Vested granted	30,000
Two Thousand Eighteen Stock Incentive Plan [Member] \| Common Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Common stock shares reserved for issuance under the Plan	10,000,000
Common stock shares available for future issuance under the Plan	1,856,008
Two Thousand Eighteen Stock Incentive Plan [Member] \| Restricted Stock Units (RSUs) [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation, vesting period	3 years
Options to Purchase Common Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of stock options exercised	281,762	654,332	186,665
Proceeds from exercise of stock options	$ 758,130	$ 1,116,242	$ 297,563
Stock option granted, contractual term	7 years	7 years	7 years
Common stock unit granted	2,715,000	2,183,500	5,882,500
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan	$ 10,020,000.00
Expected remaining weighted average vesting period	2 years 4 months 6 days
Non-cash stock-based compensation expense	$ 5,694,120	$ 3,780,086	$ 3,535,647
Expected dividend rate	0.00%
Options to Purchase Common Stock [Member] \| Cashless Basis [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of stock options exercised		6,666
Shares issued for cashless option exercise		3,444

Stock Compensation - Summary of Stock Option Activity under the Company's Plan (Detail)	12 Months Ended
	Dec. 31, 2020 USD ($) $ / shares shares
Share-based Payment Arrangement [Abstract]
Number of options, Outstanding at beginning of year \| shares	11,478,334
Number of options, Granted \| shares	2,715,000
Number of options, Exercised or released \| shares	(281,762)
Number of options, Forfeited or cancelled \| shares	(160,668)
Number of options, Expired \| shares	(357,235)
Number of options, Outstanding at end of year \| shares	13,393,669
Number of options, Exercisable at end of year \| shares	7,926,136
Weighted average exercise price, Outstanding at beginning of year \| $ / shares	$ 2.95
Weighted average exercise price, Granted \| $ / shares	3.75
Weighted average exercise price, Exercised or released \| $ / shares	2.69
Weighted average exercise price, Forfeited or cancelled \| $ / shares	3.84
Weighted average exercise price, Expired \| $ / shares	3.38
Weighted average exercise price, Outstanding at end of year \| $ / shares	3.10
Weighted average exercise price, Exercisable at end of year \| $ / shares	$ 2.64
Weighted Average Remaining Contractual Term (Years), Outstanding at end of year	4 years 5 months 1 day
Weighted Average Remaining Contractual Term (Years), Exercisable at end of year	3 years 4 months 17 days
Aggregate Intrinsic value, Outstanding at end of year \| $	$ 8,237,645
Aggregate Intrinsic value, Exercisable at end of year \| $	$ 7,381,601

Stock Compensation - Schedule of Other Information Pertaining to Stock Option Activity (Detail) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Share-based Payment Arrangement [Abstract]
Weighted-average fair value of granted stock options	$ 2.33	$ 2.69	$ 1.93
Total fair value of vested stock options	$ 5,311,616	$ 3,864,995	$ 2,193,294
Total intrinsic value of exercised stock options	$ 325,102	$ 1,899,862	$ 274,864

Stock Compensation - Summary of Stock Options Awards Based on Certain Assumptions (Detail)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Risk free interest rate, Minimum	0.24%	1.51%	2.09%
Risk free interest rate, Maximum	1.64%	2.53%	2.88%
Expected volatility		75.50%	82.00%
Expected dividend yield	0.00%
Expected forfeiture rate	0.00%
Expected term	4 years 6 months	4 years 6 months
Minimum [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected volatility	80.50%
Expected term			0 years
Maximum [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected volatility	83.70%
Expected term			7 years

Stock Compensation - Summary of Restricted Stock Unit Activity (Detail) - $ / shares	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019
Schedule Of Restricted Stock Activity [Line Items]
Nonvested balance at beginning of year	352,500
Granted		352,500
Nonvested balance at end of year		352,500
Restricted Stock Units (RSUs) [Member]
Schedule Of Restricted Stock Activity [Line Items]
Nonvested balance at beginning of year	352,500
Granted	30,000
Vested	117,495
Forfeited	29,334
Nonvested balance at end of year	235,671	352,500
Nonvested Weighted Average Grant Date Fair Value balance at beginning of year	$ 4.64
Weighted Average Grant Date Fair Value, Granted	4.70
Weighted Average Grant Date Fair Value, Vested	4.64
Weighted Average Grant Date Fair Value, Forfeited	4.64
Nonvested Weighted Average Grant Date Fair Value balance at end of year	$ 4.65	$ 4.64

Benefit Plan - Additional Information (Detail) - USD ($)	12 Months Ended
	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Retirement Benefits [Abstract]
Employees contribution of pre-tax annual compensation	15.00%
Discretionary matching contributions of employee contributions of an employee's gross salary	4.00%
Contributions	$ 465,000	$ 268,000	$ 123,000