0001193125-22-216148 | 10-Q | XBRL Viewer | Catalyst Pharmaceuticals INC

Cover Page - shares	6 Months Ended
Cover Page - shares	Jun. 30, 2022	Aug. 05, 2022
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Jun. 30, 2022
Document Fiscal Year Focus	2022
Document Fiscal Period Focus	Q2
Entity Registrant Name	CATALYST PHARMACEUTICALS, INC.
Entity Central Index Key	0001369568
Current Fiscal Year End Date	--12-31
Entity Current Reporting Status	Yes
Entity Shell Company	false
Entity Filer Category	Accelerated Filer
Trading Symbol	CPRX
Entity Emerging Growth Company	false
Entity Small Business	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		102,819,504
Entity Tax Identification Number	76-0837053
Entity Incorporation, State or Country Code	DE
Entity Address, Address Line One	355 Alhambra Circle
Entity Address, Address Line Two	Suite 801
Entity Address, City or Town	Coral Gables
Entity Address, Postal Zip Code	33134
City Area Code	305
Local Phone Number	420-3200
Document Quarterly Report	true
Document Transition Report	false
Entity File Number	001-33057

CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Current Assets:
Cash and cash equivalents	$ 210,912	$ 171,445
Short-term investments	9,876	19,821
Accounts receivable, net	9,587	6,619
Inventory	7,850	7,870
Prepaid expenses and other current assets	4,333	4,351
Total current assets	242,558	210,106
Operating lease right-of-use asset	2,895	3,017
Property and equipment, net	917	959
Deferred tax assets, net	20,930	23,697
Deposits	9	9
Total assets	267,309	237,788
Current Liabilities:
Accounts payable	2,315	2,768
Accrued expenses and other liabilities	20,173	24,295
Total current liabilities	22,488	27,063
Operating lease liability, net of current portion	3,729	3,894
Total liabilities	26,217	30,957
Commitments and contingencies (Note 11)
Stockholders' equity:
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at June 30, 2022 and December 31, 2021	0	0
Common stock, $0.001 par value, 200,000,000 shares authorized; 102,709,348 shares and 102,992,913 shares issued and outstanding at June 30, 2022 and December 31, 2021, respectively	103	103
Additional paid-in capital	239,476	233,186
Retained earnings (accumulated deficit)	1,643	(26,310)
Accumulated other comprehensive income (loss) (Note 4)	(130)	(148)
Total stockholders' equity	241,092	206,831
Total liabilities and stockholders' equity	$ 267,309	$ 237,788

CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Jun. 30, 2022	Dec. 31, 2021
Statement of Financial Position [Abstract]
Preferred stock, par value	$ 0.001	$ 0.001
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, shares issued	0	0
Preferred stock, shares outstanding	0	0
Common stock, par value	$ 0.001	$ 0.001
Common stock, shares authorized	200,000,000	200,000,000
Common stock, shares issued	102,709,348	102,992,913
Common stock, shares outstanding	102,709,348	102,992,913

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Revenues:
Total revenues	$ 53,113	$ 36,365	$ 96,202	$ 66,570
Operating costs and expenses:
Cost of sales	7,643	4,545	13,533	9,226
Research and development	3,983	4,450	7,386	7,457
Selling, general and administrative	12,918	11,532	29,348	24,248
Total operating costs and expenses	24,544	20,527	50,267	40,931
Operating income	28,569	15,838	45,935	25,639
Other income (expenses), net	(324)	62	(231)	143
Net income before income taxes	28,245	15,900	45,704	25,782
Income tax provision	6,626	3,719	10,844	5,938
Net income	$ 21,619	$ 12,181	$ 34,860	$ 19,844
Net income per share:
Basic	$ 0.21	$ 0.12	$ 0.34	$ 0.19
Diluted	$ 0.2	$ 0.11	$ 0.32	$ 0.18
Weighted average shares outstanding:
Basic	102,795,600	103,407,803	102,788,719	103,610,138
Diluted	109,264,730	107,734,924	109,149,185	107,299,262
Net income	$ 21,619	$ 12,181	$ 34,860	$ 19,844
Other comprehensive income (Note 4):
Unrealized gain (loss) on available-for-sale securities, net of tax of ($101), $0, (7) and $0, respectively	323	8	18	(68)
Comprehensive income	21,942	12,189	34,878	19,776
Product Revenue Net [Member]
Revenues:
Total revenues	53,049	33,636	96,082	63,841
License and other revenue [Member]
Revenues:
Total revenues	$ 64	$ 2,729	$ 120	$ 2,729

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (Parenthetical) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Statement of Comprehensive Income [Abstract]
Net of tax impact	$ 101	$ 0	$ 7	$ 0

CONSOLIDATED STATEMENTS OF CHANGES IN STOCKHOLDERS' EQUITY - USD ($) $ in Thousands	Total	Preferred Stock [Member]	Common Stock [Member]	Additional Paid-in Capital [Member]	Retained Earnings (Accumulated Deficit) [Member]	Accumulated Other Comprehensive Gain (Loss) [Member]
Beginning Balance at Dec. 31, 2020	$ 169,598	$ 0	$ 104	$ 223,168	$ (53,705)	$ 31
Beginning Balance (shares) at Dec. 31, 2020			103,782,000
Issuance of stock options for services	1,442			1,442
Exercise of stock options for common stock	188			188
Exercise of stock options for common stock (shares)			90,000
Amortization of restricted stock for services	129			129
Repurchase of common stock	(293)				(293)
Repurchase of common stock (shares)			(67,000)
Other comprehensive gain (loss)	(76)					(76)
Net income	7,663				7,663
Ending Balance at Mar. 31, 2021	178,651	0	$ 104	224,927	(46,335)	(45)
Ending Balance (shares) at Mar. 31, 2021			103,805,000
Beginning Balance at Dec. 31, 2020	169,598	0	$ 104	223,168	(53,705)	31
Beginning Balance (shares) at Dec. 31, 2020			103,782,000
Net income	19,844
Ending Balance at Jun. 30, 2021	188,944	0	$ 103	226,699	(37,821)	(37)
Ending Balance (shares) at Jun. 30, 2021			103,162,000
Beginning Balance at Mar. 31, 2021	178,651	0	$ 104	224,927	(46,335)	(45)
Beginning Balance (shares) at Mar. 31, 2021			103,805,000
Issuance of stock options for services	1,388			1,388
Exercise of stock options for common stock	271			271
Exercise of stock options for common stock (shares)			83,000
Amortization of restricted stock for services	130			130
Repurchase of common stock	(3,668)		$ (1)		(3,667)
Repurchase of common stock (shares)			(733,000)
Issuance of common stock upon vesting of restricted stock units, net	(17)			(17)
Issuance of common stock upon vesting of restricted stock units, net (shares)			7,000
Other comprehensive gain (loss)	8					8
Net income	12,181				12,181
Ending Balance at Jun. 30, 2021	188,944	0	$ 103	226,699	(37,821)	(37)
Ending Balance (shares) at Jun. 30, 2021			103,162,000
Beginning Balance at Dec. 31, 2021	206,831	0	$ 103	233,186	(26,310)	(148)
Beginning Balance (shares) at Dec. 31, 2021			102,993,000
Issuance of stock options for services	1,623			1,623
Exercise of stock options for common stock	1,102			1,102
Exercise of stock options for common stock (shares)			364,000
Amortization of restricted stock for services	280			280
Repurchase of common stock	(2,551)				(2,551)
Repurchase of common stock (shares)			(400,000)
Other comprehensive gain (loss)	(305)					(305)
Net income	13,241				13,241
Ending Balance at Mar. 31, 2022	220,221	0	$ 103	236,191	(15,620)	(453)
Ending Balance (shares) at Mar. 31, 2022			102,957,000
Beginning Balance at Dec. 31, 2021	206,831	0	$ 103	233,186	(26,310)	(148)
Beginning Balance (shares) at Dec. 31, 2021			102,993,000
Net income	34,860
Ending Balance at Jun. 30, 2022	241,092	0	$ 103	239,476	1,643	(130)
Ending Balance (shares) at Jun. 30, 2022			102,709,000
Beginning Balance at Mar. 31, 2022	220,221	0	$ 103	236,191	(15,620)	(453)
Beginning Balance (shares) at Mar. 31, 2022			102,957,000
Issuance of stock options for services	1,594			1,594
Exercise of stock options for common stock	1,282			1,282
Exercise of stock options for common stock (shares)			345,000
Amortization of restricted stock for services	429			429
Repurchase of common stock	(4,356)				(4,356)
Repurchase of common stock (shares)			(600,000)
Issuance of common stock upon vesting of restricted stock units, net	(20)			(20)
Issuance of common stock upon vesting of restricted stock units, net (shares)			7,000
Other comprehensive gain (loss)	323					323
Net income	21,619				21,619
Ending Balance at Jun. 30, 2022	$ 241,092	$ 0	$ 103	$ 239,476	$ 1,643	$ (130)
Ending Balance (shares) at Jun. 30, 2022			102,709,000

CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021
Operating Activities:
Net income	$ 34,860	$ 19,844
Adjustments to reconcile net income to net cash provided by (used in) operating activities:
Depreciation	71	128
Stock-based compensation	3,926	3,089
Deferred taxes	2,718	4,270
Change in accrued interest and accretion of discount on investments	9	(6)
Reduction in the carrying amount of right-of-use asset	122	108
Realized loss on sale of available-for-sale securities	633
(Increase) decrease in:
Accounts receivable, net	(2,968)	(305)
Inventory	20	(2,446)
Prepaid expenses and other current assets and deposits	18	687
Increase (decrease) in:
Accounts payable	(453)	(1,332)
Accrued expenses and other liabilities	(4,137)	(5,490)
Operating lease liability	(150)	942
Net cash provided by (used in) operating activities	34,669	19,489
Investing Activities:
Purchases of property and equipment	(29)	(912)
Purchases of investments		(10,000)
Proceeds from sale of available-for-sale securities	9,370
Net cash provided by (used in) investing activities	9,341	(10,912)
Financing Activities:
Payment of employee withholding tax related to stock-based compensation	(20)	(17)
Proceeds from exercise of stock options	2,384	459
Repurchase of common stock	(6,907)	(3,961)
Net cash provided by (used in) financing activities	(4,543)	(3,519)
Net increase (decrease) in cash and cash equivalents	39,467	5,058
Cash and cash equivalents – beginning of period	171,445	130,237
Cash and cash equivalents – end of period	210,912	135,295
Supplemental disclosures of cash flow information:
Cash paid for income taxes	$ 5,844	341
Non-cash investing and financing activities:
Operating lease liabilities arising from obtaining right-of-use assets		$ 3,309

Organization and Description of Business

6 Months Ended

Jun. 30, 2022

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Organization and Description of Business

1.	Organization and Description of Business.

Catalyst Pharmaceuticals, Inc. and subsidiary (collectively, the “Company”) is a commercial-stage biopharmaceutical company focused on

in-licensing,

developing, and commercializing novel medicines for patients living with rare diseases. With exceptional patient focus, Catalyst is committed to developing a robust pipeline of cutting-edge,

best-in-class

medicines for rare diseases.

Catalyst’s New Drug Application for FIRDAPSE

(amifampridine) Tablets 10 mg for the treatment of adults with Lambert-Eaton myasthenic syndrome (“LEMS”) was approved in 2018 by the U.S. Food & Drug Administration (“FDA”), and FIRDAPSE

is commercially available in the United States as a treatment for adults with LEMS. Further, Canada’s national healthcare regulatory agency, Health Canada, approved the use of FIRDAPSE

for the treatment of adult patients in Canada with LEMS in 2020 and FIRDAPSE

is commercially available in Canada for the treatment of patients with LEMS through a license and supply agreement with KYE Pharmaceuticals.

Since inception, the Company has devoted substantially all of its efforts to business planning, research and development, recruiting management and technical staff, acquiring operating assets, raising capital, and selling its product. The Company incurred operating losses in each period from inception and started reporting operating income during the year ended December 31, 2019. The Company has been able to fund its cash needs to date through offerings of its securities and from revenues from sales of its product. See Note 1

(Stockholders’ Equity).

Capital Resources

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

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

Risks and Uncertainties

There are numerous aspects of the coronavirus

(COVID-19)

pandemic that have adversely affected the Company’s business since the beginning of the pandemic. The Company closely monitors the impact of the pandemic on all aspects of its business and takes steps, wherever possible, to lessen those impacts. However, the Company is unable to predict the impact that the coronavirus pandemic will have on its business in future periods.

Basis of Presentation and Significant Accounting Policies

6 Months Ended

Jun. 30, 2022

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, 2021 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, 2021 included in the 2021 Annual Report on

Form 10-K

filed by the Company with the SEC. The results of operations for the six months ended June 30, 2022 are not necessarily indicative of the results to be expected for any future period or for the full 2022 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 consolidated financial statements and accompanying notes. Actual results could differ from those estimates.

CASH AND CASH EQUIVALENTS.

The Company considers all highly liquid instruments, purchased with an original maturity of three months or less, to be cash equivalents. Cash equivalents consist mainly of money market funds and U.S. Treasuries. The Company has substantially all of its cash and cash equivalents deposited with one financial institution. These amounts exceed federally insured limits.

INVESTMENTS.

The Company invests in high credit-quality instruments in order to obtain higher yields on its cash available for investments. At June 30, 2022 and December 31, 2021, investments consisted of short-term bond fund(s) and U.S. Treasuries. Such investments are not insured by the Federal Deposit Insurance Corporation.

The short-term bond fund and U.S. Treasuries held at June 30, 2022 are classified as available-for-sale securities. The short-term bond fund is classified as a current asset, which reflects management’s intention to use the proceeds from the sale of this investment to fund the Company’s operations, as necessary. The Company classifies

U.S. Treasuries with stated maturities of greater than three months and less than one year in short-

term investments. U.S Treasuries

with stated maturities greater than one year are classified as

non-current

investments in its consolidated balance sheets. There are no

non-current

investments as of June 30, 2022 and December 31, 2021.

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 (expense), 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 (expense), net in the consolidated statements of operations and comprehensive income. 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 as a result of a credit loss. The Company considers various factors in determining whether to recognize an allowance for credit losses 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. If the unrealized loss of an

available-for-sale

debt security is determined to be a result of a credit loss the Company would recognize an allowance and the corresponding credit loss would be included in the consolidated statements of operations and comprehensive income. The Company has not recorded an allowance for credit loss on its

available-for-sale

securities. See Note 3 (Investments).

ACCOUNTS RECEIVABLE, NET.

Accounts receivable is recorded net of customer allowance for distribution fees, trade discounts, prompt payment discounts, chargebacks and expected credit losses. Allowances for distribution fees, trade discounts, prompt payment discounts and chargebacks are based on contractual terms. The Company estimates the allowance for expected credit losses based on existing contractual payment terms, actual payment patterns of its customers and individual customer circumstances. At June 30, 2022 and December 31, 2021, the Company determined that an allowance for expected credit losses was not required. No accounts were written off during the periods presented.

INVENTORY

. Inventories are stated at the lower of cost or net realizable value. Inventories consist of raw materials,

work-in-process

and finished goods. Costs to be capitalized as inventories primarily include third party manufacturing costs and other overhead costs. Cost is determined using a standard cost method, which approximates actual cost, and assumes a

first-in,

first out (FIFO) flow of goods. The Company began capitalizing inventories post FDA approval of FIRDAPSE

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

were recorded as research and development expenses in prior years’ consolidated statements of operations and comprehensive income. If information becomes available that suggests that inventories may not be realizable, the Company may be required to expense a portion or all of the previously capitalized inventories.

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

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

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

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

PREPAID EXPENSES AND OTHER CURRENT ASSETS.

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

pre-clinical

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

PROPERTY AND EQUIPMENT,

NET.

Property and equipment are recorded at cost less accumulated depreciation. Depreciation is calculated to amortize the depreciable assets over their useful lives using the straight-line method and commences when the asset is placed in service. Leasehold improvements are amortized on a straight-line basis over the term of the lease or the estimated life of the improvement, whichever is shorter. Useful lives generally range from three to five years

for computer equipment, from

five

seven years for furniture and equipment, and from five to ten years for leasehold improvements. Expenditures for repairs and maintenance are charged to expenses as incurred.

FAIR VALUE OF FINANCIAL INSTRUMENTS.

The Company’s financial instruments consist of cash and cash equivalents, investments, accounts receivable, accounts payable, and accrued expenses and other liabilities. At June 30, 2022 and December 31, 2021, 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 (in thousands)

	Balances as of June 30, 2022		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	15,009	$	15,009	$	—	$	—

U.S. Treasuries	$	176,699	$	176,699	$	—	$	—

Short-term investments:
Short-term bond fund	$	9,876	$	9,876	$	—	$	—



	Balances as of December 31, 2021		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	$	10,990	$	10,990	$	—	$	—

U.S. Treasuries	$	140,995	$	140,995	$	—	$	—

Short-term investments:
Short-term bond funds	$	19,821	$	19,821	$	—	$	—

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 lease does 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 term includes options to extend or terminate the lease, however, these options are not considered in the lease term as the Company 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 a lease agreement with lease and

non-lease

components, which are accounted for separately.

SHARE REPURCHASES.

In March 2021, the Company’s Board of Directors approved a share repurchase program that authorizes the repurchase of up to $40 million of the Company’s common stock.

The Company accounts for share repurchases by charging the excess of the repurchase price over the repurchased common stock’s par value entirely to retained earnings (accumulated deficit). All repurchased shares are retired and become authorized but unissued shares. The Company accrues for the shares purchased under the share repurchase plan based on the trade date. The Company may terminate or modify its share repurchase program at any time.

REVENUE RECOGNITION.

Product Revenues:

The Company recognizes revenue when its customer obtains title of the promised goods, in an amount that reflects the consideration to which the Company expects to be entitled in exchange for these goods.

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

in the United States. The Customer subsequently resells FIRDAPSE

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

To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company assesses the goods or services promised within each contract and determines those that are performance obligations by assessing 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 collaborative and license agreements. Collaborative and license agreement payments may include nonrefundable fees at the inception of the agreements, contingent payments for specific achievements designated in the agreements, and/or net profit-sharing payments on sales of products resulting from the collaborative and license arrangements. For a complete discussion of accounting for collaborative and licensing arrangements, see Revenues from Collaboration and Licensing Arrangements below.

Product Revenue, Net:

The Company sells FIRDAPSE

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

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

The Company recognizes revenue on product sales when the Customer obtains control of the Company’s product, which occurs at a point in time (upon delivery or upon dispense to patient). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 15 and 30 days.

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

If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the three and six months ended June 30, 2022 and 2021.

During the three and six months ended June 30, 2022 and 2021, principally all of the Company’s sales of FIRDAPSE

in the United States were to its Customer.

Reserves for Variable Consideration:

Revenue from product sales is recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established. Components of variable consideration include trade discounts and allowances, prompt payment discounts, product returns, provider chargebacks and discounts, government rebates, and other incentives, such as voluntary patient assistance, and other allowances that are offered within contracts between the Company and its Customer, payors, and other indirect customers relating to the Company’s sale of its products. These reserves, as detailed below, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable (if the amount is payable to the Customer) or a current liability (if the amount is payable to a party other than a Customer).

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

The amount of variable consideration which is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplates application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of June 30, 2022 and, therefore, the transaction price was not reduced further during the three and six months ended June 30, 2022 and 2021. 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, transactional 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. However, if the Company has determined such services received 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 through June 30, 2022 and 2021, as well as a reduction to accounts receivable, net on the consolidated balance sheets.

Prompt Payment Discounts:

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

Funded

Co-pay

Assistance Program:

The Company contracts with a third-party to manage the

co-pay

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

co-pay

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

that has been recognized as revenue, but remains in the distribution channel at the end of each reporting period. These payments are considered payable to the third-party vendor and the related reserve is recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenses and other current liabilities in the consolidated balance sheets.

Product Returns:

Consistent with industry practice, the Company offers the SPs and its distributor limited product return rights for damaged and expiring product, provided it is within a specified period around the product expiration date as set forth in the applicable individual distribution agreement. The Company estimates the amount of its product sales that may be returned by its Customer and records this estimate as a reduction of revenue in the period the related product revenue is recognized. The Company currently estimates product return liabilities using available industry data and its own sales information, including its visibility into the inventory remaining in the distribution channel. These payments are considered payable to the third-party vendor and the related reserve is recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenses and other current liabilities in the consolidated balance sheets. 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 paid 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 primarily 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 FIRDAPSE

free of charge 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 FIRDAPSE

free of charge for longer periods of time for those who are uninsured or functionally uninsured with respect to FIRDAPSE

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

Revenues from Collaboration and Licensing Arrangements:

The Company analyzes license and collaboration arrangements pursuant to FASB ASC Topic 808, Collaborative Arrangement Guidance and Consideration, (“Topic 808”) to assess whether such arrangements, or transactions between arrangement participants, involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities or are more akin to a vendor-customer relationship. In making this evaluation, the Company considers whether the activities of the collaboration are considered to be distinct and deemed to be within the scope of the collaborative arrangement guidance or if they are more reflective of a vendor-customer relationship and, therefore, within the scope of Topic 606. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement.

For elements of collaboration arrangements that are not accounted for pursuant to guidance in Topic 606, an appropriate recognition method is determined and applied consistently, generally by analogy to the revenue from contracts with customers guidance.

The Company evaluates the performance obligations promised in the contract that are based on goods and services that will be transferred to the customer and determines whether those obligations are both (i) capable of being distinct and (ii) distinct in the context of the contract. Goods or services that meet these criteria are considered distinct performance obligations. The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration.

The agreements provide for milestone payments upon achievement of development and regulatory events. The Company accounts for milestone payments as variable consideration in accordance with Topic 606. At the inception of each arrangement that includes variable consideration, the Company evaluates the amount of potential transaction price and the likelihood that the transaction price will be received. The Company utilizes either the most likely amount method or expected value method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Arrangements that include rights to additional goods or services that are exercisable at a customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the customer and, if so, these options are considered performance obligations.

After contract inception, the transaction price is reassessed at every period end and updated for changes such as resolution of uncertain events. Any change in the overall transaction price is allocated to the performance obligations based on the same methodology used at contract inception.

The Company recognizes sales-based royalties or net profit-sharing when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the sales-based royalty or net profit-sharing has been allocated has been satisfied.

Payments to and from the collaborator are presented in the statement of operations based on the nature of the Company’s business operations, the nature of the arrangement, including the contractual terms, and the nature of the payments.

Refer to Note

(Collaborative and Licensing Arrangements), for further discussion on the Company’s collaborative and licensing arrangements.

RESEARCH AND DEVELOPMENT.

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

ADVERTISING EXPENSE.

In connection with the FDA approval and commercial launch of FIRDAPSE

in 2019, the Company began to incur advertising costs. Advertising costs are expensed as incurred. The company incurred $0.8 million and $1.5 million in advertising costs during the three and six months ended June 30, 2022, respectively, and $0.5 million and $1.0 million during the three and six months ended June 30, 2021, respectively, which are included in selling, general and administrative expenses in the Company’s consolidated statement of operations and comprehensive income.

STOCK-BASED COMPENSATION.

The Company recognizes expense in the consolidated statements of operations for the grant date 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 SPs. Therefore, its distributor and SPs account for principally 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, and only limited commercial experience, which makes it difficult to evaluate its current business, predict its future prospects, and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, FIRDAPSE

, and expects FIRDAPSE

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

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

and its drug candidates. The commercialization of FIRDAPSE

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

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

ROYALTIES.

Royalties incurred in connection with the Company’s license agreement for FIRDAPSE

, as disclosed in Note 1

(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 2018. 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.

COMPREHENSIVE INCOME.

U.S. GAAP requires that all components of comprehensive income be reported in the financial statements in the period in which they are recognized. Comprehensive income is net income, plus certain other items that are recorded directly into stockholders’ equity. The Company’s comprehensive income is shown on the consolidated statements of operations and comprehensive income for the three and six months ended June 30, 2022 and 2021, and is comprised of net unrealized gains (losses) on the Company’s

available-for-sale

securities.

NET INCOME PER COMMON SHARE.

Basic net income per share is computed by dividing net income for the period by the weighted average number of common shares outstanding during the period. With regard to common stock subject to vesting requirements, the calculation includes only the vested portion of such stock and units.

Diluted net income per common share is computed by dividing net income 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 June 30,				For the Six Months Ended June 30,

	2022		2021		2022		2021
Basic weighted average common shares outstanding		102,795,600		103,407,803		102,788,719		103,610,138
Effect of dilutive securities		6,469,130		4,327,121		6,360,466		3,689,124

Diluted weighted average common shares outstanding		109,264,730		107,734,924		109,149,185		107,299,262

Outstanding

common stock equivalents totaling approximately

2.2

million were excluded from the calculation of

diluted

net income per common share for both the three and six months ended June 30, 2022, as their effect would be anti-dilutive. For the three and six months ended June 30, 2021, approximately

4.9

million and

5.1

million shares, respectively, of common stock were excluded from the calculation of diluted net income per common share as their effect would be anti-d

ilutive

Investments

6 Months Ended

Jun. 30, 2022

Investments, Debt and Equity Securities [Abstract]

Investments

3.	Investments.

Available-for-sale

investments by security type were as follows (in thousands):


	Estimated Fair Value		Gross Unrealized Gains		Gross Unrealized Losses			Amortized Cost
At June 30, 2022:
U.S. Treasuries - Cash equivalents	$	176,699	$	—	$	(39	)	$	176,738
Short-term bond fund		9,876		—		(130	)		10,006

Total	$	186,575	$	—	$	(169	)	$	186,744

At December 31, 2021:
U.S. Treasuries - Cash equivalents	$	140,995	$	2	$	—		$	140,993
Short-term bond funds		19,821		—		(196	)		20,017

Total	$	160,816	$	2	$	(196	)	$	161,010

There were realized losses from sale of

available-for-sale

securities of

633

thousand

during the three and six months ended June 30, 2022. There were no realized gains or losses from

available-for-sale

securities during the three and six months ended June 30, 2021.

The estimated fair values of

available-for-sale

securities at June 30, 2022, by contractual maturity, are summarized as follows (in thousands):


	June 30, 2022
Due in one year or less	$	186,575

Accumulated Other Comprehensive Income (Loss)

6 Months Ended

Jun. 30, 2022

Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract]

Accumulated Other Comprehensive Income (Loss)

4.	Accumulated Other Comprehensive Income (Loss).

The following table summarizes the changes in accumulated other comprehensive income (loss), net of tax from unrealized gains (losses) on available-for-sale securities, the Company’s only component of accumulated other comprehensive income (loss) for the three and six months ended June 30, 2022.

The amount reclassified out of accumulated other comprehensive income (loss), net of tax and into net income during the three and six months ended June 30, 2022, was solely due to a realized loss from sale of available-for-sale securities. There were no reclassifications out of accumulated other comprehensive income (loss) during the three and six months ended June 30, 2021.


	Total Accumulated Other Comprehensive Income (Loss)
Balance at March 31, 2022	$	(453	)

Other comprehensive loss before reclassifications		(310	)
Amount reclassified from accumulated other comprehensive income		633

Net current period other comprehensive gain (loss)		323

Balance at June 30, 2022	$	(130	)


Balance at December 31, 2021	$	(148	)

Other comprehensive loss before reclassifications		(615	)
Amount reclassified from accumulated other comprehensive income		633

Net current period other comprehensive gain (loss)		18

Balance at June 30, 2022	$	(130	)

Inventory

6 Months Ended

Jun. 30, 2022

Inventory Disclosure [Abstract]

Inventory

5.	Inventory.

Inventory consists of the following (in thousands):


	June 30, 2022		December 31, 2021
Raw materials	$	—	$	1,769
Work-in-process		6,592		5,172
Finished goods		1,258		929

Total inventory	$	7,850	$	7,870

Prepaid Expenses and Other Current Assets

6 Months Ended

Jun. 30, 2022

Text Block [Abstract]

Prepaid Expenses and Other Current Assets

6 .	Prepaid Expenses and Other Current Assets.

Prepaid expenses and other current assets consist of the following (in thousands):


	June 30, 2022		December 31, 2021
Prepaid manufacturing costs	$	970	$	307
Prepaid tax		96		564
Prepaid insurance		656		1,213
Prepaid subscriptions fees		697		909
Prepaid research fees		185		452
Prepaid commercialization expenses		914		195
Due from collaborative and licensing arrangements		217		105
Prepaid conference and travel expenses		204		279
Other		394		327

Total prepaid expenses and other current assets	$	4,333	$	4,351

Operating Leases

6 Months Ended

Jun. 30, 2022

Operating Lease, Lease Income [Abstract]

Operating Leases

7 .	Operating Leases.

The Company has an operating lease agreement for its corporate office. The lease includes an option to extend the lease for up to 5 years and options to terminate the lease within 6 and 7.6 years. There are no obligations under finance leases.

The Company entered into an agreement in May 2020 that amended its lease for its office facilities. Under the amended lease, the Company’s leased space increased from approximately 7,800 square feet of space to approximately 10,700 square feet of space. The amended lease commenced in March 2021 when construction of the asset was completed and space became available for use. Consequently, the Company recorded the effects of the amended lease during Q1 2021.

The components of lease expense were as follows (in thousands):


	For the Three Months Ended June 30,				For the Six Months Ended June 30,

	2022		2021		2022		2021
Operating lease cost	$	108	$	108	$	216	$	164

Supplemental cash flow information related to lease was as follows (in thousands):


	For the Six Months Ended June 30,
	2022		2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$	245	$	29
Right-of-use assets obtained in exchange for lease obligations:
Operating lease	$	45	$	36

Supplemental balance sheet information related to lease was as follows (in thousands):


	June 30, 2022		December 31, 2021
Operating lease right-of-use assets	$	2,895	$	3,017

Other current liabilities	$	322	$	308
Operating lease liabilities, net of current portion		3,729		3,894

Total operating lease liabilities	$	4,051	$	4,202

As of June 30, 2022 and December 31, 2021, the weighted average remaining lease term was 8.8 years and 9.3 years, respectively. The weighted average discount rate used to determine the operating lease liabilities was 4.51% as of June 30, 2022 and December 31, 2021.

Remaining payments of lease liabilities as of June 30, 2022 were as follows (in thousands):


2022 (remaining six months)	$	246
2023		506
2024		522
2025		537
2026		553
Thereafter		2,598

Total lease payments		4,962
Less: imputed interest		(911	)

Total	$	4,051

Rent expense was approximately $0.1 million and $0.2

million for both the three and six months ended June 30, 2022 and 2021, respectively.

Property and Equipment, Net

6 Months Ended

Jun. 30, 2022

Property, Plant and Equipment [Abstract]

Property and Equipment, Net

8 .	Property and Equipment, Net.

Property and equipment, net consists of the following (in thousands):


	June 30, 2022			December 31, 2021
Computer equipment	$	51		$	51
Furniture and equipment		223			203
Leasehold improvements		980			980
Less: Accumulated depreciation		(337	)		(275	)

Total property and equipment, net	$	917		$	959

Accrued Expenses and Other Liabilities

6 Months Ended

Jun. 30, 2022

Payables and Accruals [Abstract]

Accrued Expenses and Other Liabilities

9 .	Accrued Expenses and Other Liabilities.

Accrued expenses and other liabilities consist of the following (in

thousands):


	June 30, 2022		December 31, 2021
Accrued preclinical and clinical trial expenses	$	319	$	659
Accrued professional fees		1,533		2,391
Accrued compensation and benefits		2,853		4,035
Accrued license fees		9,923		12,819
Accrued purchases		525		2,045
Operating lease liability		322		308
Accrued variable consideration		2,613		1,716
Accrued income tax		1,855		79
Other		230		243

Current accrued expenses and other liabilities		20,173		24,295

Lease liability – non-current		3,729		3,894

Non-current accrued expenses and other liabilities		3,729		3,894

Total accrued expenses and other liabilities	$	23,902	$	28,189

Collaborative and Licensing Arrangements

6 Months Ended

Jun. 30, 2022

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Collaborative and Licensing Arrangements

10 .	Collaborative and Licensing Arrangements.

Endo

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 (Par). 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 Company evaluated the license agreement with Endo to determine whether it is a collaborative arrangement for purposes of Topic 808. As the Company shares in the significant risks and rewards, the Company has concluded that this is a collaborative arrangement. As developing a final finished dosage form of a generic product in exchange for consideration is not an output of the Company’s ongoing activities, Endo does not represent a contract with a customer. However, Topic 808 does not provide guidance on the recognition of consideration exchanged or accounting for the obligations that may arise between the parties. The Company concluded that ASC Topic 730,

Research and Development

, should be applied by analogy to payments between the parties during the development activities and Topic 606

for the milestone payment and sharing of defined net profits upon commercialization.

The collaborative agreement included a nonrefundable upfront license fee that was recognized upon receipt following execution of the collaborative arrangement for vigabatrin tablets.

The collaborative agreement provides for a $2.0 million milestone payment on the commercial launch of the product by Par. As of June 30, 2022 and 2021, no milestone payments have been earned.

There were no revenues from this collaborative arrangement for the three and six months ended June 30, 2022 or 2021. There were no expenses incurred, net, in connection with the collaborative arrangement for the three and six months ended June 30, 2022

. Total expenses incurred, net, in connection with the collaborative agreement for three and six months ended June 30, 2021 were approximately $45,000. These expenses have been included in research and development expenses in the accompanying consolidated statements of operations and comprehensive income.

KYE Pharmaceuticals Inc.

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

in Canada.

Under the agreement, Catalyst granted KYE an exclusive license to commercialize and market FIRDAPSE

in Canada. KYE assumes all selling and marketing costs under the collaboration, while the Company is responsible for supply of FIRDAPSE

based on the collaboration partner’s purchase orders.

Under the terms of the agreement, the Company will receive an

up-front

payment, received payment upon transfer of Marketing Authorization and delivery of commercial product, received payment for supply of FIRDAPSE

, will receive milestone payments, and a sharing of defined net profits upon commercialization from KYE consisting of a

mid-double-digit

percent of net sales of FIRDAPSE

This agreement is in form identified as a collaborative agreement and the Company has concluded for accounting purposes that it also represents a contract with a customer. This is because the Company grants to KYE a license and provides supply of FIRDAPSE

in exchange for consideration, which are outputs of the Company’s ongoing activities. Accordingly, the Company has concluded that this collaborative arrangement will be accounted for pursuant to Topic 606.

The collaborative agreement included a nonrefundable upfront license fee that was recognized upon transfer of the license based on a determination that the right is provided as the intellectual property exists at the point in time in which the license is granted.

Under the arrangement, the Company will receive profit-sharing reports within nine days after quarter end from KYE. Revenue from sales of FIRDAPSE

by KYE will be recognized in the quarter in which the sales occurred.

Revenues from the arrangement with KYE for the three and six months ended June 30, 2022 and 2021 were not material. Revenue from sales of FIRDAPSE

to KYE is included in product revenue, net and revenues from profit sharing and milestones is included in license and other revenue in the accompanying consolidated statements of operations and comprehensive income.

Total expenses incurred, net in connection with the agreement with KYE for the three and six months ended June 30, 2022 and 2021 were

t material and have been included in selling, general and administrative expenses in the accompanying consolidated statements of operations and comprehensive income.

DyDo Pharma, Inc.

On June 28, 2021, the Company entered into a license agreement with DyDo Pharma, Inc. (DyDo), for the development and commercialization of FIRDAPSE

in Japan.

Under the agreement, DyDo has joint rights to develop FIRDAPSE

, and exclusive rights to commercialize the product, in Japan. DyDo is responsible for funding all clinical, regulatory, marketing and commercialization activities in Japan, while the Company is responsible for clinical and commercial supply based on purchase orders, as well as providing support to DyDo in its efforts to obtain regulatory approval for the product from the Japanese regulatory authorities.

Under the terms of the agreement, the Company has earned an

up-front

payment and may earn further development and sales milestones for FIRDAPSE

, as well as revenue on product supplied to DyDo.

The Company has concluded that this license agreement will be accounted for pursuant to Topic 606. The agreement included a nonrefundable upfront license fee that was recognized upon the effective date of the agreement as the intellectual property exists at the point in time in which the right to the license is granted. The Company determined the granting of the right to the license is distinct from the supply of FIRDAPSE

and represents a separate performance obligation in the agreement.

The agreement includes milestones that are considered a sales-based royalty in which the license is deemed to be the predominant item to which these milestones relate. Revenue will be recognized when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the sales-based royalty has been allocated has been satisfied. Additionally, the agreement includes regulatory milestone payments which represent variable consideration, and due to uncertainty are fully constrained and only recognized when the uncertainty is subsequently resolved. For clinical and commercial supply of the product, the Company will recognize revenue when the Customer obtains control of the Company’s product, which will occur at a point in time which is generally at time of shipment.

There were revenues of $0.5 million from the arrangement with DyDo for the three and six months ended June 30, 2022

, which is included in product revenue, net in the accompanying consolidated statements of operations and comprehensive income.

Revenues from the arrangement with DyDo for the three and six months ended June 30, 2021

were

approximately $2.7 million relating to the nonrefundable upfront license fee

, which is included in licensing and other revenue in the accompanying consolidated statements of operations and comprehensive income

. As of June 30, 2022, no milestone payments have been earned.

Commitments and Contingencies

6 Months Ended

Jun. 30, 2022

Commitments and Contingencies Disclosure [Abstract]

Commitments and Contingencies

Commitments and Contingencies.

In May 2019, the FDA approved a New Drug Application (NDA) filed by Jacobus Pharmaceutical Company, Inc., or Jacobus, for Ruzurgi

, another formulation of amifampridine for the treatment of pediatric LEMS patients (ages 6 to under 17). As a result of the approval of Ruzurgi

, based on the Company’s belief that the approval violated its statutory rights to exclusivity under the Orphan Drug Act, in June 2019 the Company filed suit against the FDA in the U.S. District Court for the Southern District of Florida challenging this approval, and Jacobus intervened in the Company’s case. While the District Court granted summary judgement in favor of the FDA and Jacobus in 2020, on September 30, 2021, a three-judge panel of the U.S. Court of Appeals for the 11th Circuit issued a unanimous decision overturning the District Court’s decision. The appellate court adopted the Company’s argument that the FDA’s approval of Ruzurgi

violated the Company’s rights to Orphan Drug Exclusivity. On January 31, 2022, after Jacobus’ motions for stay were denied by the 11th Circuit and the U.S. Supreme Court, the U.S. District Court for the Southern District of Florida, based on a mandate issued by the 11th Circuit, entered summary judgement in the Company’s favor. On February 1, 2022, the FDA informed Jacobus that, consistent with the 11th Circuit decision, the final approval of the Ruzurgi

NDA had been switched to a tentative approval until the

7-year

orphan-drug exclusivity, or ODE, for FIRDAPSE

has expired, and Ruzurgi

was no longer available on the U.S. market. Subsequently, Jacobus filed a petition for Writ of Certiorari seeking U.S. Supreme Court review of the 11th Circuit’s decision.

In October 2020, the Company filed lawsuits against Jacobus and the specialty pharmacy marketing Ruzurgi

, PANTHERx Rare LLC, for infringement of the ‘893 patent. The lawsuits alleged that the Ruzurgi

product infringes the ‘893 patent when administered in accordance with its product labeling. Further, in August 2021, the lawsuits were amended to include alleged infringement of the ‘128 patent. The lawsuit sought damages and injunctive relief to prevent further marketing of Ruzurgi

in violation of the Company’s patent rights.

On July 11, 2022, the Company settled certain of its disputes with Jacobus. In connection with the settlement, the Company licensed the rights to develop and commercialize Ruzurgi

in the United States and Mexico (the “Territory”). Simultaneously, the Company purchased, among other intellectual property rights, Jacobus’ U.S. patents for Ruzurgi

, its new drug applications in the United States for Ruzurgi

, and certain Ruzurgi

inventory previously manufactured by Jacobus. At the same time, the Company received a license from Jacobus for use of its

know-how

related to the manufacture of Ruzurgi

. Further, the Company settled the patent case, which has been dismissed without prejudice. Additionally, Jacobus has withdrawn its petition for Writ of Certiorari seeking review of the 11th Circuit’s decision. Finally, Jacobus agreed that until the later of (i) the expiration of the royalty term or (ii) December 31, 2034, Jacobus and its affiliates, will not, directly or indirectly, research, develop, manufacture, commercialize, distribute, use or otherwise exploit any product competitive to FIRDAPSE

or Ruzurgi

in the Territory, and Laura Jacobus, the sole shareholder of Jacobus, and two of Jacobus’ other officers, also signed individual

non-competition

agreements containing the same terms.

The Company’s New Drug Submission filing for FIRDAPSE

for the symptomatic treatment of LEMS was approved when Health Canada issued a Notice of Compliance, or NOC, on July 31, 2020. In August 2020, the Company entered into a license agreement with KYE Pharmaceuticals, or KYE, pursuant to which the Company licensed to KYE the Canadian rights for FIRDAPSE

for the treatment of LEMS. On August 10, 2020, Health Canada issued a NOC to Medunik (Jacobus’ licensee in Canada for Ruzurgi

) for the treatment of LEMS. Shortly thereafter, the Company initiated a legal proceeding in Canada seeking judicial review of Health Canada’s decision to issue the NOC for Ruzurgi

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

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

needed to meet the standards of the Canadian Food and Drugs Act.

On June 3, 2021, the Company announced a positive decision in this proceeding that quashed the NOC previously issued for Ruzurgi

and remanded the matter to the Minister of Health to redetermine its decision to grant marketing authorization to Ruzurgi

in spite of FIRDAPSE

’s data protection rights. However, on June 28, 2021, the Company announced that Health Canada had

re-issued

an NOC for Ruzurgi

, once again allowing the product to be marketed in Canada for patients with LEMS. As a result, in July 2021 the Company, along with its partner in Canada, KYE, filed a second suit against Health Canada to overturn this decision.

On March 11, 2022, the Company announced that the Company had received a favorable decision from the Canadian court setting aside, for the second time, the decision of Health Canada approving Ruzurgi

for the treatment of LEMS patients. In its ruling, the court determined that the Minister of Health’s approach to evaluating whether FIRDAPSE

’s data deserved protection based on FIRDAPSE

’s status as an innovative drug, which protects by regulation the use of such data as part of a submission seeking an NOC for eight years from approval of the innovative drug, was legally flawed and not supported by the evidence. As a result, the matter has, once again, been remanded to the Minister of Health to redetermine its decision in light of the court’s ruling, and, in that regard, the Minister of Health recently sought appellate review of the Court’s decision (which appeal is ongoing). As a result, Ruzurgi

is no longer currently available for sale in Canada. The settlement between Catalyst and Jacobus described above did not resolve the outstanding issues between the parties in Canada, and this dispute continues. There can be no assurance as to the outcome of this proceeding.

Additionally, from time to time the Company may become involved in legal proceedings arising in the ordinary course of business. Except as set forth above, the Company believes that there is no other litigation pending at this time that could have, individually or in the aggregate, a material adverse effect on its results of operations, financial condition, or cash flows.

Agreements

6 Months Ended

Jun. 30, 2022

Text Block [Abstract]

Agreements

Agreements.

LICENSE AGREEMENT FOR FIRDAPSE

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

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

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

equal to 7% of net sales (as defined in the license agreement between BioMarin and the third-party licensor) in any calendar year for the duration of any regulatory exclusivity within a territory and 3.5% for territories in any calendar year in territories without regulatory exclusivity.

On May 29, 2019, the Company and BioMarin entered into an amendment to the Company’s 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 to our licensor on net sales in Japan of a similar percentage to the royalties that the Company is currently paying under its original license agreement for North America.

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

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

The Company has entered into agreements with contract manufacturers for the manufacture of commercial drug and drug and study placebo for the Company’s trials and studies, with contract research organizations (CRO) to conduct and monitor the Company’s trials and studies and with various entities for laboratories and other testing related to the Company’s trials and studies. The contractual terms of the agreements vary, but most require certain advances as well as payments based on the achievement of milestones. Further, 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

6 Months Ended

Jun. 30, 2022

Income Tax Disclosure [Abstract]

Income Taxes

Income Taxes.

The Company’s effective income tax rate was 23.7% and 23.0% for the six months ended June 30, 2022 and 2021, respectively. Differences in the effective tax and the statutory federal income tax rate of 21% are driven by state income taxes and anticipated annual permanent differences and offset by the orphan drug credit claimed.

The Company had no uncertain tax positions as of June 30, 2022 and December 31, 2021.

- Definition

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

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+ Details

Stockholders' Equity

6 Months Ended

Jun. 30, 2022

Equity [Abstract]

Stockholders' Equity

Stockholders’ Equity.

Preferred Stock

The Company has 5,000,000 shares of authorized preferred stock, $0.001 par value per share, at June 30, 2022 and December 31, 2021. No shares of preferred stock were outstanding at June 30, 2022 and December 31, 2021.

Common Stock

The Company has 200,000,000 shares of authorized common stock, par value $0.001 per share. At June 30, 2022 and December 31, 2021, 102,709,348 and 102,992,913 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.

Share Repurchases

In March 2021, the Company’s Board of Directors approved a share repurchase program that authorizes the repurchase of up to $40 million of the Company’s common stock, pursuant to a repurchase plan under Rule

10b-18

of the Securities Act. The share repurchase program commenced on March 22, 2021. During the three and six months ended June 30, 2022, 600

thousand

shares and 1.0

million

shares were repurchased for an aggregate purchase price of approximately $4.4 million and $6.9 million, respectively ($7.26 and $6.91 average price per share, respectively). During the three and six months ended June 30, 2021,

approxim

atel

733

thousand

shares and 800

thousand

shares were repurchased for an aggregate purchase price of approximately $3.7 million and $4.0 million, respectively ($5.00 and $4.95 average price per share, respectively).

2020 Shelf Registration Statement

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

333-240052)

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

- Definition

The entire disclosure for shareholders' equity comprised of portions attributable to the parent entity and noncontrolling interest, including other comprehensive income. Includes, but is not limited to, balances of common stock, preferred stock, additional paid-in capital, other capital and retained earnings, accumulated balance for each classification of other comprehensive income and amount of comprehensive income.

+ References

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-Publisher FASB
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+ Details

Stock Compensation

6 Months Ended

Jun. 30, 2022

Share-based Payment Arrangement [Abstract]

Stock Compensation

Stock Compensation.

For the three and six months ended June 30, 2022 and 2021, the Company recorded stock-based compensation expense as follows (in

thousands):


	Three months ended June 30,				Six months ended June 30,
	2022		2021		2022		2021


Research and development	$	425	$	376	$	857	$	765
Selling, general and administrative		1,598		1,142		3,069		2,324

Total stock-based compensation	$	2,023	$	1,518	$	3,926	$	3,089

Stock Options

As of June 30, 2022, there were outstanding stock options to purchase 13,859,919 shares of common stock, of which stock options to purchase 9,284,480

shares of common stock were exercisable.

During the three and six months ended June 30, 2022, the Company granted seven-year term options to purchase an aggregate of 33,000 and 443,000 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $1.6 million and $3.2 million, respectively, during the three and six months ended June 30, 2022.

During the three and six months ended June 30, 2021, the Company granted seven-year term options to purchase an aggregate of 70,000 and 630,000 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $1.4 million and $2.8 million, respectively, during the three and six months ended June 30, 2021.

During the three and six months ended June 30, 2022, options to purchase 345,593 shares and 709,365 shares, respectively, of the Company’s common stock were exercised, with proceeds of $1.3 million and $2.4 million respectively, to the Company.

During the three and six months ended June 30, 2021, options to purchase 83,332 shares and 173,330 shares, respectively, of the Company’s common stock were exercised, with proceeds of $0.3 million and $0.5 million respectively, to the Company.

As of June 30, 2022, there was approximately $9.8 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 2.15 years.

Restricted Stock Units

There were no grants of restricted stock units to employees or directors during the three months ended June 30, 2022. The Company granted 474,500 restricted stock units during the six months ended June 30, 2022. There were no grants of restricted stock units to employees or directors during the three or six months ended June 30, 2021. During the three and six months ended June 30, 2022, the Company recorded

non-cash

stock-based compensation expense related to restricted stock units totaling $0.4 million and $0.7 million, respectively. During the three and six months ended June 30, 2021, the Company recorded

non-cash

stock-based compensation expense related to restricted stock units totaling $0.1 million and $0.3 million, respectively.

As of June 30, 2022, there was approximately $3.4 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 2.48 years.

Subsequent Events

6 Months Ended

Jun. 30, 2022

Subsequent Events [Abstract]

Subsequent Events

Subsequent Events.

On July 11, 2022, the Company settled its ongoing patent infringement litigation with Jacobus Pharmaceutical Company, Inc., and PANTHERx Rare LLC. As part of the settlement, the Company has dismissed all claims related to the patent litigation between the companies and has acquired certain of Jacobus’s intellectual property rights, including rights to develop and commercialize Ruzurgi

in the U.S. and Mexico. See Note 1

In connection with the settlement, the Company has agreed to pay the following consideration to Jacobus:

•

$30 million of cash, of which $10 million was paid at the closing of the settlement on July 11, 2022 and the balance of which will be paid over the next two years;

•

an annual royalty on the Company’s net sales (as defined in the License and Asset Purchase Agreement between the Company and Jacobus) of amifampridine products in the United States equal to: (a) for calendar years 2022 through 2025, 1.5% (with a minimum annual royalty of $3.0 million per year), and (b) for calendar years 2026 through the expiration of the last to expire of the Company’s FIRDAPSE

patents in the United States, 2.5% (with a minimum annual royalty of $5 million per year); provided, however, that the royalty rate may be reduced and the minimum annual royalty may be eliminated under certain

circumstances; and

•

If the Company were to receive a priority review voucher for FIRDAPSE

or Ruzurgi

in the future, 50% of the consideration paid by a third party to acquire that voucher will be paid to Jacobus.

Basis of Presentation and Significant Accounting Policies (Policies)

6 Months Ended

Jun. 30, 2022

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.

Form 10-K

PRINCIPLES OF CONSOLIDATION.

USE OF ESTIMATES

USE OF ESTIMATES.

CASH AND CASH EQUIVALENTS

CASH AND CASH EQUIVALENTS.

INVESTMENTS

INVESTMENTS.

U.S. Treasuries with stated maturities of greater than three months and less than one year in short-

term investments. U.S Treasuries

with stated maturities greater than one year are classified as

non-current

investments in its consolidated balance sheets. There are no

non-current

investments as of June 30, 2022 and December 31, 2021.

The Company records

available-for-sale

securities. See Note 3 (Investments).

ACCOUNTS RECEIVABLE, NET

ACCOUNTS RECEIVABLE, NET.

INVENTORY

. Inventories are stated at the lower of cost or net realizable value. Inventories consist of raw materials,

work-in-process

first-in,

first out (FIFO) flow of goods. The Company began capitalizing inventories post FDA approval of FIRDAPSE

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

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

PREPAID EXPENSES AND OTHER CURRENT ASSETS

PREPAID EXPENSES AND OTHER CURRENT ASSETS.

pre-clinical

PROPERTY AND EQUIPMENT, NET

PROPERTY AND EQUIPMENT,

NET.

for computer equipment, from

five

seven years for furniture and equipment, and from five to ten years for leasehold improvements. Expenditures for repairs and maintenance are charged to expenses as incurred.

FAIR VALUE OF FINANCIAL INSTRUMENTS

FAIR VALUE OF FINANCIAL INSTRUMENTS.

FAIR VALUE MEASUREMENTS

FAIR VALUE MEASUREMENTS.

liability.

	Fair Value Measurements at Reporting Date Using (in thousands)

	Balances as of June 30, 2022		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	15,009	$	15,009	$	—	$	—

U.S. Treasuries	$	176,699	$	176,699	$	—	$	—

Short-term investments:
Short-term bond fund	$	9,876	$	9,876	$	—	$	—



	Balances as of December 31, 2021		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	$	10,990	$	10,990	$	—	$	—

U.S. Treasuries	$	140,995	$	140,995	$	—	$	—

Short-term investments:
Short-term bond funds	$	19,821	$	19,821	$	—	$	—

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 accounted for separately.

SHARE REPURCHASES

SHARE REPURCHASES.

In March 2021, the Company’s Board of Directors approved a share repurchase program that authorizes the repurchase of up to $40 million of the Company’s common stock.

REVENUE RECOGNITION

REVENUE RECOGNITION.

Product Revenues:

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

in the United States. The Customer subsequently resells FIRDAPSE

Product Revenue, Net:

The Company sells FIRDAPSE

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

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

During the three and six months ended June 30, 2022 and 2021, principally all of the Company’s sales of FIRDAPSE

in the United States were to its Customer.

Reserves for Variable Consideration:

Trade Discounts and Allowances:

Prompt Payment Discounts:

Funded

Co-pay

Assistance Program:

The Company contracts with a third-party to manage the

co-pay

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

co-pay

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

Product Returns:

Provider Chargebacks and Discounts:

Government Rebates:

Bridge and Patient Assistance Programs:

The Company provides FIRDAPSE

free of charge 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 FIRDAPSE

free of charge for longer periods of time for those who are uninsured or functionally uninsured with respect to FIRDAPSE

Revenues from Collaboration and Licensing Arrangements:

Refer to Note

(Collaborative and Licensing Arrangements), for further discussion on the Company’s collaborative and licensing arrangements.

RESEARCH AND DEVELOPMENT

RESEARCH AND DEVELOPMENT.

ADVERTISING EXPENSE

ADVERTISING EXPENSE.

In connection with the FDA approval and commercial launch of FIRDAPSE

STOCK-BASED COMPENSATION

STOCK-BASED COMPENSATION.

three

years. Forfeitures are recognized as a reduction of stock-based compensation expense as they occur.

CONCENTRATION OF CREDIT RISK

CONCENTRATION OF RISK.

, and expects FIRDAPSE

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

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

and its drug candidates. The commercialization of FIRDAPSE

ROYALTIES

ROYALTIES.

Royalties incurred in connection with the Company’s license agreement for FIRDAPSE

, as disclosed in Note 1

(Agreements), are expensed to cost of sales as revenue from product sales is recognized.

INCOME TAXES

INCOME TAXES.

more-likely-than-not

COMPREHENSIVE INCOME

COMPREHENSIVE INCOME.

available-for-sale

securities.

NET INCOME PER COMMON SHARE

NET INCOME PER COMMON SHARE.

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


	For the Three Months Ended June 30,				For the Six Months Ended June 30,

	2022		2021		2022		2021
Basic weighted average common shares outstanding		102,795,600		103,407,803		102,788,719		103,610,138
Effect of dilutive securities		6,469,130		4,327,121		6,360,466		3,689,124

Diluted weighted average common shares outstanding		109,264,730		107,734,924		109,149,185		107,299,262

Outstanding

common stock equivalents totaling approximately

2.2

million were excluded from the calculation of

diluted

net income per common share for both the three and six months ended June 30, 2022, as their effect would be anti-dilutive. For the three and six months ended June 30, 2021, approximately

4.9

million and

5.1

million shares, respectively, of common stock were excluded from the calculation of diluted net income per common share as their effect would be anti-d

ilutive

RECLASSIFICATIONS

RECLASSIFICATIONS.

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

RECENTLY ISSUED ACCOUNTING STANDARDS

RECENTLY ISSUED ACCOUNTING STANDARDS.

The Company did not adopt any accounting standards during the three and six months ended June 30, 2022.

Basis of Presentation and Significant Accounting Policies (Tables)

6 Months Ended

Jun. 30, 2022

Accounting Policies [Abstract]

Fair Value Measurement Specific to Assets or Liability

	Fair Value Measurements at Reporting Date Using (in thousands)

	Balances as of June 30, 2022		Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
Cash and cash equivalents:
Money market funds	$	15,009	$	15,009	$	—	$	—

U.S. Treasuries	$	176,699	$	176,699	$	—	$	—

Short-term investments:
Short-term bond fund	$	9,876	$	9,876	$	—	$	—



	Balances as of December 31, 2021		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	$	10,990	$	10,990	$	—	$	—

U.S. Treasuries	$	140,995	$	140,995	$	—	$	—

Short-term investments:
Short-term bond funds	$	19,821	$	19,821	$	—	$	—

Basic and Dilutive Weighted Average Common Shares

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


	For the Three Months Ended June 30,				For the Six Months Ended June 30,

	2022		2021		2022		2021
Basic weighted average common shares outstanding		102,795,600		103,407,803		102,788,719		103,610,138
Effect of dilutive securities		6,469,130		4,327,121		6,360,466		3,689,124

Diluted weighted average common shares outstanding		109,264,730		107,734,924		109,149,185		107,299,262

Investments (Tables)

6 Months Ended

Jun. 30, 2022

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 (in thousands):


	Estimated Fair Value		Gross Unrealized Gains		Gross Unrealized Losses			Amortized Cost
At June 30, 2022:
U.S. Treasuries - Cash equivalents	$	176,699	$	—	$	(39	)	$	176,738
Short-term bond fund		9,876		—		(130	)		10,006

Total	$	186,575	$	—	$	(169	)	$	186,744

At December 31, 2021:
U.S. Treasuries - Cash equivalents	$	140,995	$	2	$	—		$	140,993
Short-term bond funds		19,821		—		(196	)		20,017

Total	$	160,816	$	2	$	(196	)	$	161,010

Estimated Fair Values of Available for Sale Securities

The estimated fair values of

available-for-sale

securities at June 30, 2022, by contractual maturity, are summarized as follows (in thousands):


	June 30, 2022
Due in one year or less	$	186,575

Accumulated Other Comprehensive Income (Loss) (Tables)

6 Months Ended

Jun. 30, 2022

Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract]

Summary of Reclassifications out of Accumulated Other Comprehensive Income (loss)

There were no reclassifications out of accumulated other comprehensive income (loss) during the three and six months ended June 30, 2021.


	Total Accumulated Other Comprehensive Income (Loss)
Balance at March 31, 2022	$	(453	)

Other comprehensive loss before reclassifications		(310	)
Amount reclassified from accumulated other comprehensive income		633

Net current period other comprehensive gain (loss)		323

Balance at June 30, 2022	$	(130	)


Balance at December 31, 2021	$	(148	)

Other comprehensive loss before reclassifications		(615	)
Amount reclassified from accumulated other comprehensive income		633

Net current period other comprehensive gain (loss)		18

Balance at June 30, 2022	$	(130	)

Inventory (Tables)

6 Months Ended

Jun. 30, 2022

Inventory Disclosure [Abstract]

Summary of current inventory

Inventory consists of the following (in thousands):


	June 30, 2022		December 31, 2021
Raw materials	$	—	$	1,769
Work-in-process		6,592		5,172
Finished goods		1,258		929

Total inventory	$	7,850	$	7,870

Prepaid Expenses and Other Current Assets (Tables)

6 Months Ended

Jun. 30, 2022

Text Block [Abstract]

Prepaid expenses and other current assets

Prepaid expenses and other current assets consist of the following (in thousands):


	June 30, 2022		December 31, 2021
Prepaid manufacturing costs	$	970	$	307
Prepaid tax		96		564
Prepaid insurance		656		1,213
Prepaid subscriptions fees		697		909
Prepaid research fees		185		452
Prepaid commercialization expenses		914		195
Due from collaborative and licensing arrangements		217		105
Prepaid conference and travel expenses		204		279
Other		394		327

Total prepaid expenses and other current assets	$	4,333	$	4,351

Operating Leases (Tables)

6 Months Ended

Jun. 30, 2022

Disclosure of Operating Leases [Abstract]

Lease, Cost

The components of lease expense were as follows (in thousands):


	For the Three Months Ended June 30,				For the Six Months Ended June 30,

	2022		2021		2022		2021
Operating lease cost	$	108	$	108	$	216	$	164

Schedule of Supplemental Cash Flow Information Related To Lease

Supplemental cash flow information related to lease was as follows (in thousands):


	For the Six Months Ended June 30,
	2022		2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$	245	$	29
Right-of-use assets obtained in exchange for lease obligations:
Operating lease	$	45	$	36

Schedule of Supplemental Balance Sheet related To Lease

Supplemental balance sheet information related to lease was as follows (in thousands):


	June 30, 2022		December 31, 2021
Operating lease right-of-use assets	$	2,895	$	3,017

Other current liabilities	$	322	$	308
Operating lease liabilities, net of current portion		3,729		3,894

Total operating lease liabilities	$	4,051	$	4,202

Lessee, Operating Lease, Liability, Maturity

Remaining payments of lease liabilities as of June 30, 2022 were as follows (in thousands):


2022 (remaining six months)	$	246
2023		506
2024		522
2025		537
2026		553
Thereafter		2,598

Total lease payments		4,962
Less: imputed interest		(911	)

Total	$	4,051

Property and Equipment, Net (Tables)

6 Months Ended

Jun. 30, 2022

Property, Plant and Equipment [Abstract]

Property and Equipment, net

Property and equipment, net consists of the following (in thousands):


	June 30, 2022			December 31, 2021
Computer equipment	$	51		$	51
Furniture and equipment		223			203
Leasehold improvements		980			980
Less: Accumulated depreciation		(337	)		(275	)

Total property and equipment, net	$	917		$	959

Accrued Expenses and Other Liabilities (Tables)

6 Months Ended

Jun. 30, 2022

Payables and Accruals [Abstract]

Schedule of Accrued Expenses and Other Liabilities

Accrued expenses and other liabilities consist of the following (in

thousands):


	June 30, 2022		December 31, 2021
Accrued preclinical and clinical trial expenses	$	319	$	659
Accrued professional fees		1,533		2,391
Accrued compensation and benefits		2,853		4,035
Accrued license fees		9,923		12,819
Accrued purchases		525		2,045
Operating lease liability		322		308
Accrued variable consideration		2,613		1,716
Accrued income tax		1,855		79
Other		230		243

Current accrued expenses and other liabilities		20,173		24,295

Lease liability – non-current		3,729		3,894

Non-current accrued expenses and other liabilities		3,729		3,894

Total accrued expenses and other liabilities	$	23,902	$	28,189

Stock Compensation (Tables)

6 Months Ended

Jun. 30, 2022

Share-based Payment Arrangement [Abstract]

Stock-Based Compensation Expense

For the three and six months ended June 30, 2022 and 2021, the Company recorded stock-based compensation expense as follows (in

thousands):


	Three months ended June 30,				Six months ended June 30,
	2022		2021		2022		2021


Research and development	$	425	$	376	$	857	$	765
Selling, general and administrative		1,598		1,142		3,069		2,324

Total stock-based compensation	$	2,023	$	1,518	$	3,926	$	3,089

Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($) $ in Thousands, shares in Millions	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021	Dec. 31, 2021
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Maximum maturity period of cash and cash equivalent			three month
Non current investments	$ 0		$ 0		$ 0
Minimum amortization period of compensation cost on straight line basis			1 year
Maximum Amortization Period Of Compensation Cost On Straight Line Basis			3 years
Potential equivalent common stock excluded	2.2	4.9	2.2	5.1
Selling, General and Administrative Expenses [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Advertising Expense	$ 800	$ 500	$ 1,500	$ 1,000
Minimum [Member] \| Furniture and Equipment [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets			5 years
Minimum [Member] \| Computer Equipment [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets			3 years
Minimum [Member] \| Leasehold Improvements [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets			5 years
Maximum [Member] \| Furniture and Equipment [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets			7 years
Maximum [Member] \| Computer Equipment [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets			5 years
Maximum [Member] \| Leasehold Improvements [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Useful life of assets			10 years

Basis of Presentation and Significant Accounting Policies - Fair Value Measurement Specific to Assets or Liability (Detail) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Short-term investments	$ 9,876	$ 19,821
Short-term bond funds [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Short-term investments	9,876	19,821
U.S. Treasuries [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	176,699	140,995
Money market fund [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	15,009	10,990
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] \| Short-term bond funds [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Short-term investments	9,876	19,821
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] \| U.S. Treasuries [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	176,699	140,995
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] \| Money market fund [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	$ 15,009	10,990
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		$ 0

Basis of Presentation and Significant Accounting Policies - Schedule Of Reconcile Basic And Dilutive Weighted Average Common Shares (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Basic weighted average common shares outstanding	102,795,600	103,407,803	102,788,719	103,610,138
Effect of dilutive securities	6,469,130	4,327,121	6,360,466	3,689,124
Dilutive weighted average common shares outstanding	109,264,730	107,734,924	109,149,185	107,299,262

Investments - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2022
Investments, Debt and Equity Securities [Abstract]
Realized losses from sale of available-for-sale securities	$ 633	$ 633

Investment - Summary of Available-for-Sale Investments by Security type (Detail) - USD ($) $ in Thousands	6 Months Ended	12 Months Ended
	Jun. 30, 2022	Dec. 31, 2021
Net Investment Income [Line Items]
Estimated Fair Value	$ 186,575	$ 160,816
Gross Unrealized Gains	0	2
Gross Unrealized Losses	(169)	(196)
Amortized cost	186,744	161,010
U.S Treasury Bond Securities Cash Equivalents [Member]
Net Investment Income [Line Items]
Estimated Fair Value	176,699	140,995
Gross Unrealized Gains		2
Gross Unrealized Losses	(39)
Amortized cost	176,738	140,993
Short Term Bond Funds [Member]
Net Investment Income [Line Items]
Estimated Fair Value	9,876	19,821
Gross Unrealized Losses	(130)	(196)
Amortized cost	$ 10,006	$ 20,017

Investment - Estimated Fair Values of Available for Sale Securities (Detail) $ in Thousands	Jun. 30, 2022 USD ($)
Investments, Debt and Equity Securities [Abstract]
Due in one year or less	$ 186,575

Accumulated Other Comprehensive Income (Loss) - Summary of Reclassifications out of Accumulated Other Comprehensive Income (loss) (Detail) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Accumulated Other Comprehensive Income (Loss) [Line Items]
Beginning balance	$ (453)		$ (148)
Other comprehensive loss before reclassifications	(310)		(615)
Amount reclassified from accumulated other comprehensive income	633		633
Net current period other comprehensive gain (loss)	323	$ 8	18	$ (68)
Ending balance	$ (130)		$ (130)

- Definition

Accumulated change in equity from transactions and other events and circumstances from non-owner sources, net of tax effect, at period end. Excludes Net Income (Loss), and accumulated changes in equity from transactions resulting from investments by owners and distributions to owners. Includes foreign currency translation items, certain pension adjustments, unrealized gains and losses on certain investments in debt and equity securities, other than temporary impairment (OTTI) losses related to factors other than credit losses on available-for-sale and held-to-maturity debt securities that an entity does not intend to sell and it is not more likely than not that the entity will be required to sell before recovery of the amortized cost basis, as well as changes in the fair value of derivatives related to the effective portion of a designated cash flow hedge.

+ References + Details

Inventory - Summary of current inventory (Detail) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Inventory Disclosure [Abstract]
Raw materials	$ 0	$ 1,769
Work-in-process	6,592	5,172
Finished goods	1,258	929
Total inventory, net	$ 7,850	$ 7,870

Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]
Prepaid manufacturing costs	$ 970	$ 307
Prepaid tax	96	564
Prepaid insurance	656	1,213
Prepaid subscriptions fees	697	909
Prepaid research fees	185	452
Prepaid commercialization expenses	914	195
Due from collaborative arrangements	217	105
Prepaid conference and travel expenses	204	279
Other	394	327
Total prepaid expenses and other current assets	$ 4,333	$ 4,351

Operating Leases - Operating Leases (Detail) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Disclosure of Operating Leases [Abstract]
Operating lease cost	$ 108	$ 108	$ 216	$ 164

Operating Leases - Schedule of Supplemental Cash Flow Information Related To Lease (Detail) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows	$ 245	$ 29
Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$ 45	$ 36

Operating Leases - Schedule of Supplemental Balance Sheet related To Lease (Detail) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Disclosure of Operating Leases [Line Items]
Operating lease right-of-use assets	$ 2,895	$ 3,017
Other current liabilities	322	308
Operating lease liabilities, net of current portion	3,729	3,894
Total operating lease liabilities	$ 4,051	$ 4,202

Operating Leases - Lessee, Operating Lease, Liability, Maturity (Detail) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Disclosure of Operating Leases [Line Items]
2022 (remaining nine months)	$ 246
2023	506
2024	522
2025	537
2026	553
Thereafter	2,598
Total lease payments	4,962
Less imputed interest	(911)
Total	$ 4,051	$ 4,202

Operating Leases - Additional Information (Detail) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022 USD ($)	Jun. 30, 2021 USD ($)	Jun. 30, 2022 USD ($) ft²	Jun. 30, 2021 USD ($)	Dec. 31, 2021
Disclosure of Operating Leases [Line Items]
Finance Lease Obligations \| $	$ 0		$ 0
Lessee, Operating Lease, Option to Extend			5 years
Rent expense \| $	$ 108	$ 108	$ 216	$ 164
Before agreement of company leased spaces \| ft²			7,800
After agreement of company leased spaces \| ft²			10,700
Weighted average remaining lease term	8 years 9 months 18 days		8 years 9 months 18 days		9 years 3 months 18 days
Weighted average discount rate	4.51%		4.51%		4.51%
Maximum [Member]
Disclosure of Operating Leases [Line Items]
Lessee, Operating Lease, Option to Terminate			7.6 years
Minimum [Member]
Disclosure of Operating Leases [Line Items]
Lessee, Operating Lease, Option to Terminate			6 years

Property and Equipment, Net - Property and Equipment, Net (Detail) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Property, Plant and Equipment [Line Items]
Less: Accumulated depreciation	$ (337)	$ (275)
Total property and equipment, net	917	959
Computer equipment [Member]
Property, Plant and Equipment [Line Items]
Property and equipment, gross	51	51
Furniture and equipment [Member]
Property, Plant and Equipment [Line Items]
Property and equipment, gross	223	203
Leasehold improvements [Member]
Property, Plant and Equipment [Line Items]
Property and equipment, gross	$ 980	$ 980

Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Payables and Accruals [Abstract]
Accrued preclinical and clinical trial expenses	$ 319	$ 659
Accrued professional fees	1,533	2,391
Accrued compensation and benefits	2,853	4,035
Accrued license fees	9,923	12,819
Accrued purchases	525	2,045
Operating lease liability	322	308
Accrued variable consideration	2,613	1,716
Accrued income tax	1,855	79
Other	230	243
Current accrued expenses and other liabilities	20,173	24,295
Lease liability – non-current	3,729	3,894
Non-current accrued expenses and other liabilities	3,729	3,894
Total accrued expenses and other liabilities	$ 23,902	$ 28,189

Collaborative and Licensing Arrangements - Additional Information (Detail) - USD ($)	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Collaborative Arrangements [Line Items]
Period of collaboration agreement			10 years
Total research and development expense	$ 3,983,000	$ 4,450,000	$ 7,386,000	$ 7,457,000
Milestone payments income			0	0
KYE Pharmaceuticals [Member] \| Selling, General and Administrative Expenses [Member]
Collaborative Arrangements [Line Items]
Expenses incurred in connection with collaboration agreement	0	0	0	0
Dydo Pharma Inc Member [Member]
Collaborative Arrangements [Line Items]
Milestone payments income			0
Dydo Pharma Inc Member [Member] \| Non Refundable Upfront License Fees [Member]
Collaborative Arrangements [Line Items]
Revenues		2,700,000		2,700,000
Collaborative Arrangement [Member]
Collaborative Arrangements [Line Items]
Total research and development expense	0		0
Milestone payments			2,000,000
Revenues	0	0	0	0
Collaborative Arrangement [Member] \| product revenue,net
Collaborative Arrangements [Line Items]
Revenues	$ 500,000		$ 500,000
Collaborative Arrangement [Member] \| Research and Development Expense [Member]
Collaborative Arrangements [Line Items]
Total research and development expense		$ 45,000		$ 45,000

Agreements - Additional Information (Detail) $ in Millions	6 Months Ended
Agreements - Additional Information (Detail) $ in Millions	Jun. 30, 2022 USD ($)
License Agreement [Line Items]
Percentage of royalty on net sales	3.50%
License Agreement with BioMarin [Member]
License Agreement [Line Items]
Date on which strategic collaboration is entered into	Oct. 26, 2012
Royalty agreement period	7 years
Net sales royalty threshold	$ 100
License Agreement with BioMarin [Member] \| Minimum [Member]
License Agreement [Line Items]
Percentage of royalty on net sales	7.00%
License Agreement with BioMarin [Member] \| Maximum [Member]
License Agreement [Line Items]
Percentage of royalty on net sales	10.00%

Income Taxes - Additional Information (Detail) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Dec. 31, 2021
Income Tax Disclosure [Line Items]
Effective Income Tax Rate Reconciliation, Percent	23.70%	23.00%
Unrecognized Tax Benefits that Would Impact Effective Tax Rate	$ 0		$ 0
Effective Income Tax Rate Reconciliation, at Federal Statutory Income Tax Rate, Percent	21.00%

Stockholders' Equity (Preferred Stock and Common Stock) - Additional Information (Detail)	Jun. 30, 2022 Vote $ / shares shares	Dec. 31, 2021 $ / shares shares
Stockholders Equity [Line Items]
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, par value \| $ / shares	$ 0.001	$ 0.001
Preferred stock, shares outstanding	0	0
Common stock, shares authorized	200,000,000	200,000,000
Common stock, par value \| $ / shares	$ 0.001	$ 0.001
Common stock, shares issued	102,709,348	102,992,913
Common stock, shares outstanding	102,709,348	102,992,913
Number of votes entitled for each share of common stock \| Vote	1

Stockholders' Equity ( Share Repurchases ) - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021	Mar. 31, 2021
Stockholders Equity [Line Items]
Aggregate purchase price	$ 4,400	$ 3,700	$ 6,907	$ 3,961
Average price per share	$ 7.26	$ 5	$ 6.91	$ 4.95
Common Stock [Member]
Stockholders Equity [Line Items]
Aggregate share repurchased	600,000	733,000	1,000,000	800,000
Share Purchase Program [Member] \| Common Stock [Member]
Stockholders Equity [Line Items]
Share repurchase program, authorised					$ 40,000

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

Stock Compensation - Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	$ 2,023	$ 1,518	$ 3,926	$ 3,089
Research and Development [Member]
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	425	376	857	765
Selling, General and Administrative [Member]
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Stock-based compensation	$ 1,598	$ 1,142	$ 3,069	$ 2,324

Stock Compensation - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Proceeds from exercise of stock options			$ 2,384	$ 459
Number of options, Outstanding	13,859,919		13,859,919
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	$ 3,400		$ 3,400
Expected remaining weighted average vesting period			2 years 5 months 23 days
Non-cash stock-based compensation expense	$ 400	$ 100	$ 700	$ 300
Common stock granted			474,500
Options to Purchase Common Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of stock options exercised	345,593	83,332	709,365	173,330
Proceeds from exercise of stock options	$ 1,300	$ 300	$ 2,400	$ 500
Stock option granted, contractual term	7 years	7 years	7 years	7 years
Common stock unit granted	33,000	70,000	443,000	630,000
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan	$ 9,800		$ 9,800
Expected remaining weighted average vesting period			2 years 1 month 24 days
Non-cash stock-based compensation expense	$ 1,600	$ 1,400	$ 3,200	$ 2,800
Stock options to purchase shares of common stock	9,284,480		9,284,480

Subsequent Events - Additional Information (Detail) - Subsequent Event [Member] - License and Asset Purchase Agreement [Member] $ in Millions	Jul. 11, 2022 USD ($)
Subsequent Event [Line Items]
Purchase price of an asset acquisition	$ 30.0
Upfront payment related to asset acquisition	$ 10.0
Percentage of the consideration paid by a third party to acquire that voucher	50.00%
Calendar Years 2022 through 2025 [Member]
Subsequent Event [Line Items]
Percentage of annual royalty on the Company's net sales	1.50%
Minimum royalty amount	$ 3.0
Calendar Year 2026 [Member]
Subsequent Event [Line Items]
Percentage of annual royalty on the Company's net sales	2.50%
Minimum royalty amount	$ 5.0