View Filing Data

Cover Page - shares	3 Months Ended
Cover Page - shares	Mar. 31, 2020	May 07, 2020
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Mar. 31, 2020
Document Fiscal Year Focus	2020
Document Fiscal Period Focus	Q1
Entity Registrant Name	CATALYST PHARMACEUTICALS, INC.
Entity Central Index Key	0001369568
Current Fiscal Year End Date	--12-31
Entity Current Reporting Status	Yes
Entity Shell Company	false
Entity Filer Category	Accelerated Filer
Trading Symbol	CPRX
Entity Emerging Growth Company	false
Entity Small Business	false
Entity Address, State or Province	FL
Entity Interactive Data Current	Yes
Title of 12(b) Security	Common Stock
Security Exchange Name	NASDAQ
Entity Common Stock, Shares Outstanding		103,415,365

Consolidated Balance Sheets - USD ($)	Mar. 31, 2020	Dec. 31, 2019
Current Assets:
Cash and cash equivalents	$ 101,750,937	$ 89,511,710
Short-term investments		5,007,050
Accounts receivable, net	6,918,563	10,536,997
Inventory	2,211,338	1,956,792
Prepaid expenses and other current assets	6,062,715	4,351,074
Total current assets	116,943,553	111,363,623
Operating lease right-of-use asset	730,284	793,252
Property and equipment, net	196,926	210,467
Deposits	8,888	8,888
Total assets	117,879,651	112,376,230
Current Liabilities:
Accounts payable	1,347,577	4,117,447
Accrued expenses and other liabilities	16,287,936	19,981,295
Total current liabilities	17,635,513	24,098,742
Operating lease liability, net of current portion	568,421	647,532
Total liabilities	18,203,934	24,746,274
Commitments and contingencies
Stockholders' equity:
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at March 31, 2020 and December 31, 2019	0
Common stock, $0.001 par value, 150,000,000 shares authorized; 103,408,699 shares and 103,397,033 shares issued and outstanding at March 31, 2020 and December 31, 2019, respectively	103,409	103,397
Additional paid-in capital	217,751,166	216,205,678
Accumulated deficit	(118,262,609)	(128,688,624)
Accumulated other comprehensive income (loss)	83,751	9,505
Total stockholders' equity	99,675,717	87,629,956
Total liabilities and stockholders' equity	$ 117,879,651	$ 112,376,230

Consolidated Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2020	Dec. 31, 2019
Statement of Financial Position [Abstract]
Preferred stock, par value	$ 0.001	$ 0.001
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, shares issued	0	0
Preferred stock, shares outstanding	0	0
Common stock, par value	$ 0.001	$ 0.001
Common stock, shares authorized	150,000,000	150,000,000
Common stock, shares issued	103,408,699	103,397,033
Common stock, shares outstanding	103,408,699	103,397,033

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) (unaudited) - USD ($)	3 Months Ended
	Mar. 31, 2020	Mar. 31, 2019
Revenues:
Product revenue, net	$ 29,136,472	$ 12,448,438
Revenue, Product and Service [Extensible List]	us-gaap:ProductMember	us-gaap:ProductMember
Operating costs and expenses:
Cost of sales	$ 4,150,866	$ 1,711,788
Cost, Product and Service [Extensible List]	us-gaap:ProductMember	us-gaap:ProductMember
Research and development	$ 4,222,811	$ 3,307,959
Selling, general and administrative	10,063,048	8,416,460
Total operating costs and expenses	18,436,725	13,436,207
Operating income (loss)	10,699,747	(987,769)
Other income, net	336,233	343,266
Net income (loss) before income taxes	11,035,980	(644,503)
Provision for income taxes	609,965
Net income (loss)	$ 10,426,015	$ (644,503)
Net income (loss) per share:
Basic	$ 0.10	$ (0.01)
Diluted	$ 0.10	$ (0.01)
Weighted average shares outstanding:
Basic	103,407,347	102,747,923
Diluted	106,534,600	102,747,923
Net income (loss)	$ 10,426,015	$ (644,503)
Other comprehensive income (loss):
Unrealized gain (loss) on available-for-sale securities	74,246	13,560
Comprehensive income (loss)	$ 10,500,261	$ (630,943)

CONSOLIDATED STATEMENT OF CHANGES IN STOCKHOLDERS' EQUITY (unaudited) - USD ($)	Total	Common Stock [Member]	Additional Paid-in Capital [Member]	Accumulated Deficit [Member]	Accumulated Other Comprehensive Gain (Loss) [Member]
Beginning Balance at Dec. 31, 2018	$ 50,783,809	$ 102,739	$ 211,265,279	$ (160,563,961)	$ (20,248)
Beginning Balance (shares) at Dec. 31, 2018		102,739,257
Issuance of stock options for services	933,411		933,411
Exercise of stock options for common stock	89,350	$ 65	89,285
Exercise of stock options for common stock (shares)		65,000
Other comprehensive gain (loss)	13,560				13,560
Net income (loss)	(644,503)			(644,503)
Ending Balance at Mar. 31, 2019	51,175,627	$ 102,804	212,287,975	(161,208,464)	(6,688)
Ending Balance (shares) at Mar. 31, 2019		102,804,257
Beginning Balance at Dec. 31, 2019	87,629,956	$ 103,397	216,205,678	(128,688,624)	9,505
Beginning Balance (shares) at Dec. 31, 2019		103,397,033
Issuance of stock options for services	1,383,672		1,383,672
Exercise of stock options for common stock	26,149	$ 12	26,137
Exercise of stock options for common stock (shares)		11,666
Amortization of restricted stock for services	135,679		135,679
Other comprehensive gain (loss)	74,246				74,246
Net income (loss)	10,426,015			10,426,015
Ending Balance at Mar. 31, 2020	$ 99,675,717	$ 103,409	$ 217,751,166	$ (118,262,609)	$ 83,751
Ending Balance (shares) at Mar. 31, 2020		103,408,699

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References + Details

CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited) - USD ($)	3 Months Ended
	Mar. 31, 2020	Mar. 31, 2019
Operating Activities:
Net income (loss)	$ 10,426,015	$ (644,503)
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities:
Depreciation	13,541	13,748
Amortization of right-of-use asset	62,968	59,621
Stock-based compensation	1,519,351	933,411
Change in accrued interest and accretion of discount on investments	81,296	(53,674)
(Increase) decrease in:
Accounts receivable, net	3,618,434	(7,251,381)
Inventory	(254,546)	(40,575)
Prepaid expenses and other current assets and deposits	(1,711,641)	(231,485)
Increase (decrease) in:
Accounts payable	(2,769,870)	602,719
Accrued expenses and other liabilities	(3,699,518)	(1,337,155)
Operating lease liability	(72,952)	(67,155)
Net cash provided by (used in) operating activities	7,213,078	(8,016,429)
Investing Activities:
Purchases of property and equipment		(5,633)
Purchases of investments		(9,944,974)
Proceeds from maturities and sales of investments	5,000,000	20,400,000
Net cash provided by (used in) investing activities	5,000,000	10,449,393
Financing Activities:
Proceeds from exercise of stock options	26,149	89,350
Net cash provided by (used in) financing activities	26,149	89,350
Net increase (decrease) in cash and cash equivalents	12,239,227	2,522,314
Cash and cash equivalents—beginning of period	89,511,710	16,559,400
Cash and cash equivalents—end of period	101,750,937	19,081,714
Non-cash investing and financing activities:
Unrealized gain (loss) on available-for-sale securities	$ 74,246	$ 13,560

Organization and Description of Business

3 Months Ended

Mar. 31, 2020

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Organization and Description of Business

1.	Organization and Description of Business.

Catalyst Pharmaceuticals, Inc. and subsidiary (collectively, the “Company”) is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), Anti-MuSK antibody positive myasthenia gravis

(MuSK-MG),

and Spinal Muscular Atrophy (SMA) Type 3.

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

for the treatment of adults with LEMS (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.

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

Capital Resources

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

10-Q.

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

Risks and Uncertainties

There are many uncertainties regarding the novel coronavirus

(COVID-19)

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

3 Months Ended

Mar. 31, 2020

Accounting Policies [Abstract]

Basis of Presentation and Significant Accounting Policies

2.	Basis of Presentation and Significant Accounting Policies.

INTERIM FINANCIAL STATEMENTS.

The accompanying unaudited interim consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (U.S. GAAP), and pursuant to the rules and regulations of the Securities and Exchange Commission (SEC) for reporting of interim financial information. Pursuant to such rules and regulations, certain information and note disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been omitted. The consolidated balance sheet as of December 31, 2019 included in this Form

10-Q

was derived from the audited financial statements and does not include all disclosures required by U.S. GAAP.

In the opinion of management, the accompanying unaudited interim consolidated financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these consolidated statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2019 included in the 2019 Annual Report on Form

10-K

filed by the Company with the SEC. The results of operations for the three months ended March 31, 2020 are not necessarily indicative of the results to be expected for any future period or for the full 2020 fiscal year.

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 U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates.

CASH AND CASH EQUIVALENTS.

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 at times may exceed federally insured limits.

INVESTMENTS

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

Short-Term Bond Fund

The

Company previously owned a

short-term bond fund

hat

was classified

trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognizes realized gains and losses and unrealized gains and losses to earnings. At March 31, 20

and December 31, 201

, there were no investments classified as trading securities

as the Company sold its interest in the short-term bond fund in 2019.

There were no sales o

trading securities for the three months ended March 31,

2020. Realized losses on trading securities during the three months ended March 31, 2019 were $4,980

. Unrealized gain on trading securities was $0 and $52,741, respectively, for the three months ended March 31, 2020 and March 31, 2019, and is included in other income, net in the accompanying consolidated statements of operations.

U.S. Treasuries

U.S. Treasuries are classified as

available-for-sale

securities. The Company classifies

available-for-sale

securities with stated maturities of greater than three months and less than one year as short-term investments.

Available-for-sale

securities 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).

ACCOUNTS RECEIVABLE, NET.

Accounts receivable

recorded net of customer allowance for distribution fees, trade discounts, prompt payment discounts, chargebacks and doubtful accounts. Allowances for distribution fees, trade discounts, prompt payment discounts and chargebacks are based on contractual terms. The Company estimates the allowance for expected credit loss based on existing contractual payment terms, actual payment patterns of its Customer and individual Customer circumstances. At March 31, 2020

and December 31, 2019

, the Company determined that an allowance for expected credit loss was not required. No accounts were written off during the periods presented.

INVENTORY.

Inventories are stated at the lower of cost or net realizable value with cost determined under the

first-in-first-out

(FIFO) cost method. Inventories consist of raw materials and supplies, work in process and finished goods. Costs to be capitalized as inventories primarily include third party manufacturing costs and other overhead costs. 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 porti

n or all of the previously capitalized inventories. As of March 31, 2020 and December 31, 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 research fees, prepaid insurance, prepaid commercialization expenses, prepaid subscription fees and prepaid manufacturing. 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.

FAIR VALUE OF FINANCIAL INSTRUMENTS.

The Company’s financial instruments consist of cash and cash equivalents, investments, accounts receivable, accounts payables and accrued expenses and other liabilities. At March 31, 2020 and December 31, 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 March 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	$	23,610,888	$	23,610,888	$	—	$	—

U.S. Treasuries	$	69,997,200	$	—	$	69,997,200	$	—


	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 control of the promised goods or services, in an amount that reflects the consideration

which the entity expects to be entitled in exchange for these goods or services. 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”), who 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, milestone and event-based payments for specific achievements designated in the collaborative agreements, and/or royalties on sales of products resulting from a collaborative arrangement. For a complete discussion of accounting for collaborative arrangements, see Revenues from Collaborative Arrangement below.

Product

Revenue

, Net:

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

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

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

During the three months ended March 31, 2020

and 2019

, all of the Company’s sales 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, product returns, provider chargebacks and discounts, government rebates, and other incentives, such as voluntary patient assistance, and other allowances that are offered within contracts between the Company and its Customer, payors, and other indirect customers relating to the Company’s sale of its products. These reserves, as detailed below, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable (if the amount is payable to the Customer) or a current liability (if the amount is payable to a party other than a customer).

These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the respective underlying contracts.

The amount of variable consideration which is included in the transaction price may be constrained, and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplated application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of March 31, 2020 and, therefore, the transaction price was not reduced further during the three months ended March 31, 2020. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known.

Trade Discounts and Allowances:

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 March 31, 2020, as well as a reduction to 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 Customer 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 payer 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 Arrangement:

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

(vigabatrin) tablets. Pursuant to the terms of this agreement, collaborator could be required to make various payments to the Company, including upfront license fees, milestone payments based on achievement of regulatory approvals, and royalties on sales of products resulting from the collaborative agreement.

Nonrefundable upfront license fees are recognized upon receipt as persuasive evidence of an arrangement exists, the price to the collaborator is fixed or determinable and collectability is reasonably assured.

The collaborative agreement provides for a milestone payment upon achievement of development and regulatory events. The Company accounts for milestone payments in accordance with the provisions of Accounting Standards Update (ASU)

No. 2010-17,

Revenue Recognition – Milestone Method (“Milestone Method of Accounting”). The Company recognizes consideration that is contingent upon the achievement of a milestone in its entirety as revenue in the period in which the milestone is achieved only if the milestone is substantive in its entirety. A milestone is considered substantive when it meets all of the following criteria:

The consideration is commensurate with either the entity’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone;

The consideration relates solely to past performance; and

The consideration is reasonable relative to all of the deliverables and payment terms within the arrangement.

A milestone is defined as an event (i) that can only be achieved based in whole or in part on either the entity’s performance or on the occurrence of a specific outcome resulting from the entity’s performance, (ii) for which there is substantive uncertainty at the date the arrangement is entered into that the event will be achieved, and (iii) that would result in additional payments being due to the vendor.

The Company believes that achievement of the milestone will be substantive and there will be no substantive uncertainty once the milestone is achieved.

Since the Company will receive royalty reports 60 days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter following the quarter in which the corresponding sales occurred. As of March 31, 2020 and 2019, there was no royalty revenue from sales of the collaborative product.

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

RESEARCH AND DEVELOPMENT.

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 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 at times may 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 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 2016. If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense.

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 three months ended March 31, 2020 and 2019, 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 and units.

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

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


	For the Three Months Ended March 31,
	2020		2019
Basic weighted average common shares outstanding		103,407,347		102,747,923
Effect of dilutive securities:
Unvested restricted stock units subject to vesting requirements issued to employees and non-employees		352,500		—
Common stock issuable upon the exercise of stock options		2,774,753		—

Diluted weighted average common shares outstanding		106,534,600		102,747,923

Outstanding common stock equivalents totaling approximately 5.4 million, were excluded from the calculation of diluted net income (loss) per common share for the three months ended March 31, 2020 as their effect would be anti-dilutive. For the three months ended March 31, 2019, approximately 10.6 million shares of outstanding stock options were excluded from the calculation of diluted net loss per common share because a net loss was reported in this period and therefore their effect was anti-dilutive.

RECLASSIFICATIONS.

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 the FASB’s new revenue standard, 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

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

pdate 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

pdate 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.

Investments

3 Months Ended

Mar. 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 March 31, 2020:
U.S. Treasuries – Cash equivalents	$	69,997,200	$	83,751	$	—	$	69,913,449

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

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

There were no realized gains or losses from

available-for-sale

securities for the three months ended March 31, 2020 or

March

31, 2019.

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

The estimated fair values of

available-for-sale

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


	March 31, 2020
Due in one year or less	$	69,997,200

Prepaid Expenses and Other Current Assets

3 Months Ended

Mar. 31, 2020

Text Block [Abstract]

Prepaid Expenses and Other Current Assets

4.	Prepaid Expenses and Other Current Assets.

Prepaid expenses and other current assets consist of the following:


	March 31, 2020		December 31, 2019
Prepaid manufacturing costs	$	3,471,078	$	1,526,013
Prepaid insurance		995,146		1,263,129
Prepaid subscription fees		492,716		501,251
Prepaid research fees		490,102		481,057
Prepaid commercialization expenses		287,083		62,959
Other		326,590		516,665

Total prepaid expenses and other current assets	$	6,062,715	$	4,351,074

Operating Leases

3 Months Ended

Mar. 31, 2020

Disclosure of Operating Leases [Abstract]

Operating Leases

5.	Operating Leases.

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

The components of lease expense were as follows:


	March 31, 2020
Operating lease cost	$	74,079

Supplemental cash flow information related to leases was as follows:


	March 31, 2020
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$	84,061
Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$	5,305

Supplemental balance sheet information related to leases was as follows:


	March 31, 2020
Operating lease right-of-use assets	$	730,284

Other current liabilities	$	306,677
Operating lease liabilities, net of current portion		568,421

Total operating lease liabilities	$	875,098


Weighted average remaining lease term	2.7 years
Weighted average discount rate	4.81	%

Remaining payments of lease liabilities as of March 31, 2020 were as follows:


2020 (remaining nine months)	$	255,544
2021		349,788
2022		329,662

Total lease payments		934,994
Less imputed interest		(59,896	)

Total	$	875,098

Accrued Expenses and Other Liabilities

3 Months Ended

Mar. 31, 2020

Payables and Accruals [Abstract]

Accrued Expenses and Other Liabilities

6.	Accrued Expenses and Other Liabilities.

Accrued expenses and other liabilities consist of the following:


	March 31, 2020		December 31, 2019
Accrued preclinical and clinical trial expenses	$	940,963	$	1,183,513
Accrued professional fees		3,309,351		1,241,526
Accrued compensation and benefits		1,814,396		3,064,645
Accrued license fees		3,945,991		8,751,991
Accrued purchases		1,243,991		1,313,310
Accrued contributions		1,135,000		1,535,000
Operating lease liability		306,677		300,518
Accrued variable consideration		1,308,344		884,764
Income tax payable		2,143,490		1,533,696
Other		139,733		172,332

Current accrued expenses and other liabilities		16,287,936		19,981,295

Lease liability— non-current		568,421		647,532

Non-current accrued expenses and other liabilities		568,421		647,532

Total accrued expenses and other liabilities	$	16,856,357	$	20,628,827

Collaborative Arrangement

3 Months Ended

Mar. 31, 2020

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Collaborative Arrangement

7.	Collaborative Arrangement.

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

(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 March 31, 2020 and 2019, no milestone payments have been earned.

There were no revenues from collaborative arrangement for the three months ended March 31, 2020 and 2019. Total expenses incurred, net, in connection with the collaborati

agreement for three months ended March 31, 2020 and March 31, 2019 were

approximat

$5,700 and $11,952, and have been included in research and development expenses in the accompanying consolidated statements of operations.

Commitments and Contingencies

3 Months Ended

Mar. 31, 2020

Commitments and Contingencies Disclosure [Abstract]

Commitments and Contingencies

8.	Commitments and Contingencies.

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

CPP-115,

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. The Company is also entitled to receive certain contingent compensation that will be reported when and if received.

In May 2019, the FDA approved an 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

. In addition, while the NDA for Ruzurgi

only covers pediatric patients, the Company believes 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, alleges 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 seeks an order vacating the FDA’s approval of Ruzurgi

. Jacobus has intervened in the case. Each party has filed a cross motion for summary judgement. There can be no assurance as to the outcome of this lawsuit.

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

3 Months Ended

Mar. 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.

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.

During 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, 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

3 Months Ended

Mar. 31, 2020

Income Tax Disclosure [Abstract]

Income Taxes

10.	Income Taxes.

The Company is subject to income taxes in the U.S. federal jurisdiction and various states jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for any years before 2016. If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense.

The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. As of March 31, 2020 and December 31, 2019, based on the Company’s history of operating losses, the Company has concluded that it is more likely than not that the benefit of its deferred tax assets will not be realized. Accordingly, the Company has provided a full valuation allowance for deferred tax assets including NOL and tax credit carryovers as of March 31, 2020 and December 31, 2019.

Stockholders' Equity

3 Months Ended

Mar. 31, 2020

Equity [Abstract]

Stockholders' Equity

11.	Stockholders’ Equity.

Preferred Stock

The Company has 5,000,000 shares of authorized preferred stock, $0.001 par value per share, at March 31, 2020 and December 31, 2019. No shares of preferred stock were outstanding at March 31, 20

0 and December 31, 2019.

Common Stock

The Company has 150,000,000 shares of authorized common stock, par value $0.001 per share. At March 31, 2020 and December 31, 2019, 103,408,699 and 103,397,033 shares, respectively, of common stock were issued and outstanding. Each holder of common stock is entitled to one vote of each share of common stock held of record on all matters on which stockholders generally are entitled to vote.

2017 Shelf Registration Statement

On July 12, 2017, the Company filed a universal shelf Registration Statement on Form

S-3

(the 2017 Shelf Registration Statement) with the SEC to sell up to $150 million of common stock, preferred stock, warrants to purchase common stock, or debt securities (including debt securities that may be convertible or exchangeable for common stock or other securities), which securities may be offered separately or together in units or multiple series. The 2017 Shelf Registration Statement (file

No. 333-219259)

was declared effective by the SEC on July 26, 2017.

On November 28, 2017, the Company filed a prospectus supplement and offered for sale 16,428,572 shares of its common stock at a price of $3.50 per share in an underwritten public offering under the 2017 Shelf Registration. The Company received gross proceeds in the public offering of approximately $57.5 million before underwriting commission and incurred expenses of approximately $3.7 million.

At March 31, 2020, there is approximately $92.5 million available for future sale under the 2017 Shelf Registration Statement.

Stock Compensation

3 Months Ended

Mar. 31, 2020

Share-based Payment Arrangement [Abstract]

Stock Compensation

12.	Stock Compensation.

For the three-month periods ended March 31, 2020 and 2019, the Company recorded stock-based compensation expense as follo


	Three months ended March 31,
	2020		2019
Research and development	$	418,053	$	287,721
Selling, general and administrative		1,101,298		645,690

Total stock-based compensation	$	1,519,351	$	933,411

Stock Options

As of March 31, 2020, there were outstanding stock options to purchase 12,201,668 shares of common stock, of which stock options to purchase 6,687,980 shares of common stock were exercisable as of March 31, 2020.

During the three-month periods ended March 31, 2020 and 2019, the Company granted seven-year term options to purchase an aggregate of 735,000 and 207,000 shares, respectively, of the Company’s common stock to employees and directors. The Company recorded stock-based compensation related to stock options totaling $1,383,672 and $933,411, respectively, during the three-month periods ended March 31, 2020 and 2019. During the three-month periods ended March 31, 2020 and 2019, respectively, 854,831 and 984,164 options vested.

During the three-month periods ended March 31, 2020 and 2019, options to purchase 11,666 shares and 65,000 shares, respectively, of the Company’s common stock were exercised, with proceeds of $26,149 and $89,350 respectively, to the Company.

As of March 31, 2020, there was approximately $10.4 million of unrecognized compensation expense related to

non-vested

stock option awards granted under the 2014 and 2018 Stock Incentive Plans. The cost is expected to be recognized over a weighted average period of approximately 2.23 years.

Restricted Stock Units

There were no grants of restricted stock units to employees or directors during the three-month periods ended March 31, 2020 and March 31, 2019. During the three-month period ended March 31, 2020, the Company recorded

non-cash

stock-based compensation expense related to restricted stock units totaling $135,679. No stock-based compensation related to restricted stocks was recorded during the three-month period ended March 31, 2019.

As of March 31, 2020, there was approximately $1.5 million of unrecognized compensation expense related to

non-vested

restricted stock units granted under the 2018 Stock Incentive Plan. The cost is expected to be recognized over a weighted average period of approximately 2.67 years.

Subsequent Events

3 Months Ended

Mar. 31, 2020

Subsequent Events [Abstract]

Subsequent Events

13.	Subsequent Events.

On May 7, 2020, the Company

amended the lease for

its

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.

Basis of Presentation and Significant Accounting Policies (Policies)

3 Months Ended

Mar. 31, 2020

Accounting Policies [Abstract]

INTERIM FINANCIAL STATEMENTS

INTERIM FINANCIAL STATEMENTS.

10-Q

was derived from the audited financial statements and does not include all disclosures required by U.S. GAAP.

10-K

PRINCIPLES OF CONSOLIDATION.

PRINCIPLES OF CONSOLIDATION

USE OF ESTIMATES

USE OF ESTIMATES.

CASH AND CASH EQUIVALENTS

CASH AND CASH EQUIVALENTS.

INVESTMENTS

Short-Term Bond Fund

The

Company previously owned a

short-term bond fund

hat

was classified

and December 31, 201

, there were no investments classified as trading securities

as the Company sold its interest in the short-term bond fund in 2019.

There were no sales o

trading securities for the three months ended March 31,

2020. Realized losses on trading securities during the three months ended March 31, 2019 were $4,980

U.S. Treasuries

U.S. Treasuries are classified as

available-for-sale

securities. The Company classifies

available-for-sale

securities with stated maturities of greater than three months and less than one year as short-term investments.

Available-for-sale

securities 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. See Note 3 (Investments).

ACCOUNTS RECEIVABLE, NET

ACCOUNTS RECEIVABLE, NET.

Accounts receivable

and December 31, 2019

, the Company determined that an allowance for expected credit loss was not required. No accounts were written off during the periods presented.

INVENTORY

INVENTORY.

Inventories are stated at the lower of cost or net realizable value with cost determined under the

first-in-first-out

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

n or all of the previously capitalized inventories. As of March 31, 2020 and December 31, 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

PREPAID EXPENSES AND OTHER CURRENT ASSETS

PREPAID EXPENSES AND OTHER CURRENT ASSETS.

pre-clinical

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 March 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	$	23,610,888	$	23,610,888	$	—	$	—

U.S. Treasuries	$	69,997,200	$	—	$	69,997,200	$	—


	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.

The Company recognizes revenue when its customer obtains control of the promised goods or services, in an amount that reflects the consideration

which the entity expects to be entitled in exchange for these goods or services. 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”), who 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 three months ended March 31, 2020

and 2019

, all of the Company’s sales were to its Customer.

Reserves for Variable Consideration:

Trade Discounts and Allowances:

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 Arrangement:

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

No. 2010-17,

The consideration relates solely to past performance; and

The consideration is reasonable relative to all of the deliverables and payment terms within the arrangement.

The Company believes that achievement of the milestone will be substantive and there will be no substantive uncertainty once the milestone is achieved.

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

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 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 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 Three Months Ended March 31,
	2020		2019
Basic weighted average common shares outstanding		103,407,347		102,747,923
Effect of dilutive securities:
Unvested restricted stock units subject to vesting requirements issued to employees and non-employees		352,500		—
Common stock issuable upon the exercise of stock options		2,774,753		—

Diluted weighted average common shares outstanding		106,534,600		102,747,923

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

In November 2018, the FASB issued ASU

2018-18,

2014-09

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. 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.

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.

The amendments in this

internal-use

software (and hosting arrangements that include an

internal-use

software license). Accordingly, the amendments in this

pdate require an entity in a hosting arrangement that is a service contract to follow the guidance in Subtopic

350-40

Basis of Presentation and Significant Accounting Policies (Tables)

3 Months Ended

Mar. 31, 2020

Accounting Policies [Abstract]

Fair Value Measurement Specific to Assets or Liability


	Fair Value Measurements at Reporting Date Using
	Balances as of March 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	$	23,610,888	$	23,610,888	$	—	$	—

U.S. Treasuries	$	69,997,200	$	—	$	69,997,200	$	—


	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 Three Months Ended March 31,
	2020		2019
Basic weighted average common shares outstanding		103,407,347		102,747,923
Effect of dilutive securities:
Unvested restricted stock units subject to vesting requirements issued to employees and non-employees		352,500		—
Common stock issuable upon the exercise of stock options		2,774,753		—

Diluted weighted average common shares outstanding		106,534,600		102,747,923

Investments (Tables)

3 Months Ended

Mar. 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 March 31, 2020:
U.S. Treasuries – Cash equivalents	$	69,997,200	$	83,751	$	—	$	69,913,449

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

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

Estimated Fair Values of Available for Sale Securities

The estimated fair values of

available-for-sale

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


	March 31, 2020
Due in one year or less	$	69,997,200

Prepaid Expenses and Other Current Assets (Tables)

3 Months Ended

Mar. 31, 2020

Text Block [Abstract]

Prepaid expenses and other current assets

Prepaid expenses and other current assets consist of the following:


	March 31, 2020		December 31, 2019
Prepaid manufacturing costs	$	3,471,078	$	1,526,013
Prepaid insurance		995,146		1,263,129
Prepaid subscription fees		492,716		501,251
Prepaid research fees		490,102		481,057
Prepaid commercialization expenses		287,083		62,959
Other		326,590		516,665

Total prepaid expenses and other current assets	$	6,062,715	$	4,351,074

Operating Leases (Tables)

3 Months Ended

Mar. 31, 2020

Disclosure of Operating Leases [Abstract]

Lease, Cost

The components of lease expense were as follows:


	March 31, 2020
Operating lease cost	$	74,079

Schedule of Supplemental Cash Flow Information Related To Lease

Supplemental cash flow information related to leases was as follows:


	March 31, 2020
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$	84,061
Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$	5,305

Schedule of Supplemental Balance Sheet related To Lease

Supplemental balance sheet information related to leases was as follows:


	March 31, 2020
Operating lease right-of-use assets	$	730,284

Other current liabilities	$	306,677
Operating lease liabilities, net of current portion		568,421

Total operating lease liabilities	$	875,098


Weighted average remaining lease term	2.7 years
Weighted average discount rate	4.81	%

Lessee, Operating Lease, Liability, Maturity

Remaining payments of lease liabilities as of March 31, 2020 were as follows:


2020 (remaining nine months)	$	255,544
2021		349,788
2022		329,662

Total lease payments		934,994
Less imputed interest		(59,896	)

Total	$	875,098

Accrued Expenses and Other Liabilities (Tables)

3 Months Ended

Mar. 31, 2020

Payables and Accruals [Abstract]

Schedule of Accrued Expenses and Other Liabilities

Accrued expenses and other liabilities consist of the following:


	March 31, 2020		December 31, 2019
Accrued preclinical and clinical trial expenses	$	940,963	$	1,183,513
Accrued professional fees		3,309,351		1,241,526
Accrued compensation and benefits		1,814,396		3,064,645
Accrued license fees		3,945,991		8,751,991
Accrued purchases		1,243,991		1,313,310
Accrued contributions		1,135,000		1,535,000
Operating lease liability		306,677		300,518
Accrued variable consideration		1,308,344		884,764
Income tax payable		2,143,490		1,533,696
Other		139,733		172,332

Current accrued expenses and other liabilities		16,287,936		19,981,295

Lease liability— non-current		568,421		647,532

Non-current accrued expenses and other liabilities		568,421		647,532

Total accrued expenses and other liabilities	$	16,856,357	$	20,628,827

Stock Compensation (Tables)

3 Months Ended

Mar. 31, 2020

Share-based Payment Arrangement [Abstract]

Stock-Based Compensation Expense

For the three-month periods ended March 31, 2020 and 2019, the Company recorded stock-based compensation expense as follo


	Three months ended March 31,
	2020		2019
Research and development	$	418,053	$	287,721
Selling, general and administrative		1,101,298		645,690

Total stock-based compensation	$	1,519,351	$	933,411

Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($) shares in Millions	3 Months Ended
	Mar. 31, 2020	Mar. 31, 2019	Dec. 31, 2018
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Maximum maturity period of cash and cash equivalent	three months
Trading Securities, Realized Gain (Loss)		$ 4,980
Trading Securities Sales	$ 0
Potential equivalent common stock excluded	5.4	10.6
Trading securities		$ 0	$ 0
Other Income, Net [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Unrealized gain (loss), trading securities	$ 0	$ 52,741
Minimum [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Vesting period for stock options	1 year
Maximum [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Vesting period for stock options	3 years

Basis of Presentation and Significant Accounting Policies - Fair Value Measurement Specific to Assets or Liability (Detail) - USD ($)	Mar. 31, 2020	Dec. 31, 2019
Short-Term Bond Fund [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Investments Fair Value Disclosure		$ 5,007,050
U.S. Treasuries [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	$ 69,997,200	59,932,200
Money Market Funds [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	23,610,888	23,963,617
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] \| Money Market Funds [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	23,610,888	23,963,617
Significant Other Observable Inputs (Level 2) [Member] \| Short-Term Bond Fund [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Investments Fair Value Disclosure		5,007,050
Significant Other Observable Inputs (Level 2) [Member] \| U.S. Treasuries [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	$ 69,997,200	$ 59,932,200

Basis of Presentation and Significant Accounting Policies - Schedule Of Reconcile Basic And Dilutive Weighted Average Common Shares (Details) - shares	3 Months Ended
	Mar. 31, 2020	Mar. 31, 2019
Weighted Average Number Of Shares Outstanding Diluted Disclosure Items [Line Items]
Basic weighted average common shares outstanding	103,407,347	102,747,923
Effect of dilutive securities:
Common stock issuable upon the exercise of stock options	2,774,753
Diluted weighted average common shares outstanding	106,534,600	102,747,923
Restricted Stock Units (RSUs) [Member] \| Share Based Payment Arrangement Employee And Nonemployee [Member]
Effect of dilutive securities:
Unvested restricted stock units subject to vesting requirements issued to employees and non-employees	352,500

Investments - Additional Information (Detail) - USD ($)	3 Months Ended	12 Months Ended
Investments - Additional Information (Detail) - USD ($)	Mar. 31, 2020	Dec. 31, 2019
Investments, Debt and Equity Securities [Abstract]
Realized gains losses from available for sale securities	$ 0	$ 0

Investment - Summary of Available-for-Sale Investments by Security type (Detail) - USD ($)	3 Months Ended	12 Months Ended
	Mar. 31, 2020	Dec. 31, 2019
Net Investment Income [Line Items]
Amortized cost		$ 64,929,745
Gross Unrealized Gains		9,505
Gross Unrealized Losses		0
Estimated Fair Value	$ 69,997,200	64,939,250
U.S. Treasuries - ST [Member]
Net Investment Income [Line Items]
Amortized cost		4,999,587
Gross Unrealized Gains		7,463
Gross Unrealized Losses		0
Estimated Fair Value		5,007,050
US Treasury Bond Securities Cash Equivalents [Member]
Net Investment Income [Line Items]
Amortized cost	69,913,449	59,930,158
Gross Unrealized Gains	83,751	2,042
Gross Unrealized Losses	0	0
Estimated Fair Value	$ 69,997,200	$ 59,932,200

Investment - Estimated Fair Values of Available for Sale Securities (Detail) - USD ($)	Mar. 31, 2020	Dec. 31, 2019
Investments, Debt and Equity Securities [Abstract]
Estimated Fair Value	$ 69,997,200	$ 64,939,250

Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($)	Mar. 31, 2020	Dec. 31, 2019
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]
Prepaid manufacturing costs	$ 3,471,078	$ 1,526,013
Prepaid insurance	995,146	1,263,129
Prepaid subscriptions fees	492,716	501,251
Prepaid research fees	490,102	481,057
Prepaid commercialization expenses	287,083	62,959
Other	326,590	516,665
Total prepaid expenses and other current assets	$ 6,062,715	$ 4,351,074

Operating Leases - Operating Leases (Detail)	3 Months Ended
Operating Leases - Operating Leases (Detail)	Mar. 31, 2020 USD ($)
Disclosure of Operating Leases [Abstract]
Operating lease cost	$ 74,079

Operating Leases - Schedule of Supplemental Cash Flow Information Related To Lease (Detail)	3 Months Ended
	Mar. 31, 2020 USD ($)
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$ 84,061
Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$ 5,305

Operating Leases -Schedule of Supplemental Balance Sheet related To Lease (Detail) - USD ($)	Mar. 31, 2020	Dec. 31, 2019	Mar. 31, 2019
Disclosure of Operating Leases [Line Items]
Operating lease right-of-use assets	$ 730,284	$ 793,252
Other current liabilities	306,677	300,518
Operating lease liabilities, net of current portion	568,421	$ 647,532
Total operating lease liabilities	$ 875,098
Weighted average remaining lease term			2 years 8 months 12 days
Weighted average discount rate	4.81%

Operating Leases -Lessee, Operating Lease, Liability, Maturity (Detail)	Mar. 31, 2020 USD ($)
Disclosure of Operating Leases [Line Items]
2020	$ 255,544
2021	349,788
2022	329,662
Total lease payments	934,994
Less imputed interest	(59,896)
Total	$ 875,098

Operating Leases - Additional Information (Detail)	3 Months Ended
Operating Leases - Additional Information (Detail)	Mar. 31, 2020 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

Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($)	Mar. 31, 2020	Dec. 31, 2019
Payables and Accruals [Abstract]
Accrued preclinical and clinical trial expenses	$ 940,963	$ 1,183,513
Accrued professional fees	3,309,351	1,241,526
Accrued compensation and benefits	1,814,396	3,064,645
Accrued license fees	3,945,991	8,751,991
Accrued purchases	1,243,991	1,313,310
Accrued contributions	1,135,000	1,535,000
Operating lease liability	306,677	300,518
Accrued variable consideration	1,308,344	884,764
Income tax payable	2,143,490	1,533,696
Other	139,733	172,332
Current accrued expenses and other liabilities	16,287,936	19,981,295
Lease liability - non-current	568,421	647,532
Non-current accrued expenses and other liabilities	568,421	647,532
Total accrued expenses and other liabilities	$ 16,856,357	$ 20,628,827

Collaborative Arrangement - Additional Information (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2020	Mar. 31, 2019
Collaborative Arrangement [Line Items]
Period of collaboration agreement	10 years
Expenses In Connection With Collaborative Arrangement	$ 4,222,811	$ 3,307,959
Collaborative Arrangement [Member]
Collaborative Arrangement [Line Items]
Milestone payments	2,000,000
Milestone payments income	0	0
Upfront License Fee	0	0
Expenses In Connection With Collaborative Arrangement	$ 5,700	$ 11,952

Commitments and Contingencies - 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) - License Agreement with BioMarin [Member] $ in Millions	3 Months Ended
	Mar. 31, 2020 USD ($)
License Agreement [Line Items]
Date on which strategic collaboration is entered into	Oct. 26, 2012
Royalty agreement period	7 years
Net sales royalty threshold	$ 100
Minimum [Member]
License Agreement [Line Items]
Percentage of royalty on net sales	7.00%
Maximum [Member]
License Agreement [Line Items]
Percentage of royalty on net sales	10.00%

Stockholders' Equity (Preferred Stock and Common Stock) - Additional Information (Detail)	Mar. 31, 2020 Vote $ / shares shares	Dec. 31, 2019 $ / shares shares
Equity [Abstract]
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, par value \| $ / shares	$ 0.001	$ 0.001
Preferred stock, shares outstanding	0	0
Common stock, shares authorized	150,000,000	150,000,000
Common stock, par value \| $ / shares	$ 0.001	$ 0.001
Common stock, shares issued	103,408,699	103,397,033
Common stock, shares outstanding	103,408,699	103,397,033
Number of votes entitled for each share of common stock \| Vote	1

Stockholders' Equity (2017 Shelf Registration Statement) - Additional Information (Detail) - USD ($)		3 Months Ended
	Nov. 28, 2017	Mar. 31, 2020	Jul. 12, 2017
Underwritten Public Offering [Member]
Stockholders' Equity [Line Items]
Number of common stock sold in offering	16,428,572
Common stock issued, price per share	$ 3.50
Gross proceeds from issuance of common stock	$ 57,500,000
Offering expenses	$ 3,700,000
2017 Shelf Registration Statement [Member]
Stockholders' Equity [Line Items]
Maximum dollar amount of securities to be issued under shelf registration statement			$ 150,000,000
Value of common stock available for future sale		$ 92,500,000

Stock Compensation - Stock-Based Compensation Expense (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2020	Mar. 31, 2019
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	$ 1,519,351	$ 933,411
Research and Development [Member]
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	418,053	287,721
Selling, General and Administrative [Member]
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	$ 1,101,298	$ 645,690

Stock Compensation - Additional Information (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2020	Mar. 31, 2019
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Proceeds from exercise of stock options	$ 26,149	$ 89,350
Stock options to purchase shares of common stock	12,201,668
Stock Option [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Common stock unit granted	0	0
Options granted, expiration Period	7 years	7 years
Restricted Stock Units (RSUs) [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan	$ 1,500,000
Expected remaining weighted average vesting period	2 years 8 months 1 day
Non-cash stock-based compensation expense	$ 135,679	$ 0
Common stock granted	0	0
Options to Purchase Common Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of stock options exercised	11,666	65,000
Proceeds from exercise of stock options	$ 26,149	$ 89,350
Common stock unit granted	735,000	207,000
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan	$ 10,400,000
Expected remaining weighted average vesting period	2 years 2 months 23 days
Non-cash stock-based compensation expense	$ 1,383,672	$ 933,411
Stock option vested during the period	854,831	984,164
Stock options to purchase shares of common stock	6,687,980

Subsequent Events - Additional Information (Detail) - Subsequent Event [Member]	May 07, 2020 ft²
Minimum [Member]
Subsequent Event [Line Items]
Operating lease, number of square feet \| ft²	7,800
Maximum [Member]
Subsequent Event [Line Items]
Operating lease, number of square feet \| ft²	10,700