Ovid Therapeutics (OVID) 10-Q 13 Aug 24 2024 Q2 Quarterly report Financial data

Cover Page

Cover Page - shares	6 Months Ended
	Jun. 30, 2024	Aug. 10, 2024
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2024
Document Transition Report	false
Entity File Number	001-38085
Entity Registrant Name	Ovid Therapeutics Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	46-5270895
Entity Address, Address Line One	441 Ninth Avenue
Entity Address, Address Line Two	14th Floor
Entity Address, City or Town	New York
Entity Address, State or Province	NY
Entity Address, Postal Zip Code	10001
City Area Code	646
Local Phone Number	661-7661
Title of 12(b) Security	Common Stock, par value $0.001 per share
Trading Symbol	OVID
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Non-accelerated Filer
Entity Small Business	true
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		70,971,577
Entity Central Index Key	0001636651
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q2
Amendment Flag	false

Condensed Consolidated Balance

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 29,694	$ 27,042
Marketable securities	47,280	78,792
Prepaid expenses and other current assets	3,915	3,764
Total current assets	80,889	109,598
Long-term equity investments	21,052	17,626
Restricted cash	1,931	1,931
Right-of-use asset, net	13,357	13,894
Property and equipment, net	618	769
Other noncurrent assets	246	210
Total assets	118,093	144,027
Current liabilities:
Accounts payable	4,720	3,703
Accrued expenses	8,108	6,525
Current portion, lease liability	1,291	1,246
Total current liabilities	14,119	11,474
Long-term liabilities:
Lease liability	14,099	14,756
Royalty monetization liability	972	30,000
Total liabilities	29,190	56,230
Stockholders’ equity:
Preferred stock, $0.001 par value; 10,000,000 shares authorized; Series A convertible preferred stock, 10,000 shares designated, 1,250 shares issued and outstanding at June 30, 2024 and December 31, 2023	0	0
Common stock, $0.001 par value; 125,000,000 shares authorized; 70,971,577 and 70,691,992 shares issued and outstanding at June 30, 2024 and December 31, 2023, respectively	71	71
Additional paid-in-capital	369,883	365,591
Accumulated other comprehensive (loss) income	(12)	1
Accumulated deficit	(281,039)	(277,866)
Total stockholders’ equity	88,903	87,797
Total liabilities and stockholders’ equity	$ 118,093	$ 144,027

Condensed Consolidated Balanc_2

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Jun. 30, 2024	Dec. 31, 2023
Statement of Financial Position [Abstract]
Preferred stock, par value (in dollars per share)	$ 0.001	$ 0.001
Preferred stock, shares authorized (in shares)	10,000,000	10,000,000
Preferred stock, share designated (in shares)	10,000	10,000
Preferred stock, shares issued (in shares)	1,250	1,250
Preferred stock, shares outstanding (in shares)	1,250	1,250
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, shares authorized (in shares)	125,000,000	125,000,000
Common stock shares issued (in shares)	70,971,577	70,691,992
Common stock, shares outstanding (in shares)	70,971,577	70,691,992

Condensed Consolidated Statemen

Condensed Consolidated Statements of Operations (unaudited) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Revenue:
Total revenue	$ 169	$ 75	$ 317	$ 141
Operating expenses:
Research and development	12,582	5,999	22,984	12,613
General and administrative	8,104	8,248	15,267	16,592
Total operating expenses	20,686	14,247	38,251	29,205
Loss from operations	(20,517)	(14,172)	(37,934)	(29,064)
Other income (expense), net	29,038	1,764	34,760	3,300
Income (loss) before provision for income taxes	8,521	(12,408)	(3,174)	(25,765)
Provision for income taxes	0	0	0	0
Net income (loss)	$ 8,521	$ (12,408)	$ (3,174)	$ (25,765)
Net income (loss) per share, basic (in dollars per share)	$ 0.12	$ (0.18)	$ (0.04)	$ (0.37)
Net income (loss) per share, diluted (in dollars per share)	$ 0.12	$ (0.18)	$ (0.04)	$ (0.37)
Weighted-average common shares outstanding basic (in shares)	70,916,471	70,534,181	70,816,585	70,512,479
Weighted-average common shares outstanding diluted (in shares)	71,200,798	70,534,181	70,816,585	70,512,479

Condensed Consolidated Statem_2

Condensed Consolidated Statements of Comprehensive Income (Loss) (unaudited) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Statement of Comprehensive Income [Abstract]
Net income (loss)	$ 8,521	$ (12,408)	$ (3,174)	$ (25,765)
Other comprehensive income (loss):
Unrealized gain (loss) on marketable securities	7	0	(13)	47
Comprehensive income (loss)	$ 8,528	$ (12,408)	$ (3,187)	$ (25,717)

Condensed Consolidated Statem_3

Condensed Consolidated Statements of Stockholders' Equity (unaudited) - USD ($) $ in Thousands	Total	Preferred Stock Series A Convertible Preferred Stock	Common Stock	Additional Paid-In Capital	Accumulated Other Comprehensive Income (Loss)	Accumulated Deficit
Beginning balance (in shares) at Dec. 31, 2022		1,250
Beginning balance at Dec. 31, 2022	$ 132,273	$ 0	$ 70	$ 357,771	$ (42)	$ (225,527)
Beginning balance (in shares) at Dec. 31, 2022			70,466,885
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan (in shares)			24,625
Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan	67			67
Stock-based compensation expense	1,917			1,917
Other comprehensive income (loss)	48				48
Net income (loss)	(13,356)					(13,356)
Ending balance (in shares) at Mar. 31, 2023		1,250
Ending balance at Mar. 31, 2023	120,948	$ 0	$ 70	359,754	6	(238,883)
Ending balance (in shares) at Mar. 31, 2023			70,491,510
Beginning balance (in shares) at Dec. 31, 2022		1,250
Beginning balance at Dec. 31, 2022	132,273	$ 0	$ 70	357,771	(42)	(225,527)
Beginning balance (in shares) at Dec. 31, 2022			70,466,885
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net income (loss)	(25,765)
Ending balance (in shares) at Jun. 30, 2023		1,250
Ending balance at Jun. 30, 2023	110,699	$ 0	$ 71	361,914	5	(251,291)
Ending balance (in shares) at Jun. 30, 2023			70,603,793
Beginning balance (in shares) at Mar. 31, 2023		1,250
Beginning balance at Mar. 31, 2023	120,948	$ 0	$ 70	359,754	6	(238,883)
Beginning balance (in shares) at Mar. 31, 2023			70,491,510
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan (in shares)			112,283
Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan	211			211
Stock-based compensation expense	1,949			1,949
Other comprehensive income (loss)	0
Net income (loss)	(12,408)					(12,408)
Ending balance (in shares) at Jun. 30, 2023		1,250
Ending balance at Jun. 30, 2023	$ 110,699	$ 0	$ 71	361,914	5	(251,291)
Ending balance (in shares) at Jun. 30, 2023			70,603,793
Beginning balance (in shares) at Dec. 31, 2023	1,250	1,250
Beginning balance at Dec. 31, 2023	$ 87,797	$ 0	$ 71	365,591	1	(277,866)
Beginning balance (in shares) at Dec. 31, 2023	70,691,992		70,691,992
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan (in shares)			91,969
Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan	$ 228			228
Stock-based compensation expense	1,968			1,968
Other comprehensive income (loss)	(20)				(20)
Net income (loss)	(11,694)					(11,694)
Ending balance (in shares) at Mar. 31, 2024		1,250
Ending balance at Mar. 31, 2024	$ 78,279	$ 0	$ 71	367,787	(19)	(289,560)
Ending balance (in shares) at Mar. 31, 2024			70,783,961
Beginning balance (in shares) at Dec. 31, 2023	1,250	1,250
Beginning balance at Dec. 31, 2023	$ 87,797	$ 0	$ 71	365,591	1	(277,866)
Beginning balance (in shares) at Dec. 31, 2023	70,691,992		70,691,992
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net income (loss)	$ (3,174)
Ending balance (in shares) at Jun. 30, 2024	1,250	1,250
Ending balance at Jun. 30, 2024	$ 88,903	$ 0	$ 71	369,883	(12)	(281,039)
Ending balance (in shares) at Jun. 30, 2024	70,971,577		70,971,577
Beginning balance (in shares) at Mar. 31, 2024		1,250
Beginning balance at Mar. 31, 2024	$ 78,279	$ 0	$ 71	367,787	(19)	(289,560)
Beginning balance (in shares) at Mar. 31, 2024			70,783,961
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan (in shares)			187,616
Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan	356			356
Stock-based compensation expense	1,740			1,740
Other comprehensive income (loss)	7				7
Net income (loss)	$ 8,521					8,521
Ending balance (in shares) at Jun. 30, 2024	1,250	1,250
Ending balance at Jun. 30, 2024	$ 88,903	$ 0	$ 71	$ 369,883	$ (12)	$ (281,039)
Ending balance (in shares) at Jun. 30, 2024	70,971,577		70,971,577

Condensed Consolidated Statem_4

Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023
Cash flows from operating activities:
Net loss	$ (3,174)	$ (25,765)
Adjustments to reconcile net loss to cash used in operating activities:
Change in fair value of royalty monetization liability	(29,028)	0
Unrealized gain on equity investment	(3,427)	(848)
Change in accrued interest and accretion of discount on marketable securities	(1,678)	(1,139)
Stock-based compensation expense	3,708	3,865
Depreciation and amortization expense	284	282
Non-cash operating lease expense	538	511
Change in lease liability	(612)	573
Change in operating assets and liabilities:
Prepaid expenses and other current assets	(151)	(4,048)
Security deposit	0	12
Accounts payable	1,017	1,810
Accrued expenses	1,582	929
Net cash used in operating activities	(30,941)	(23,818)
Cash flows from investing activities:
Purchase of marketable securities	(46,821)	(24,574)
Sales/maturities of marketable securities	80,000	85,000
Purchase of long-term equity investment	0	(10,000)
Purchases of property and equipment	(53)	(21)
Software development and other costs	(116)	(110)
Net cash provided by investing activities	33,010	50,295
Cash flows from financing activities:
Proceeds from exercise of options and purchases from employee stock purchase plan	584	278
Net cash provided by financing activities	584	278
Net increase in cash, cash equivalents and restricted cash	2,653	26,755
Cash, cash equivalents and restricted cash, at beginning of period	28,973	46,799
Cash, cash equivalents and restricted cash, at end of period	$ 31,625	$ 73,554

NATURE OF OPERATIONS

NATURE OF OPERATIONS	6 Months Ended
	Jun. 30, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
NATURE OF OPERATIONS	NATURE OF OPERATIONS Ovid Therapeutics Inc. (the “Company”) was incorporated under the laws of the state of Delaware and commenced operations on April 1, 2014 and maintains its principal executive office in New York, New York. The Company is a biopharmaceutical company that is dedicated to meaningfully improving the lives of people affected by certain epilepsies and brain conditions with seizure symptoms. Since its inception, the Company has devoted substantially all of its efforts to business development, research and development, recruiting management and technical staff, and raising capital, and has financed its operations through the issuance of convertible preferred stock, common stock, other equity instruments, the sale and/or licensing of certain assets and the licensing of certain intellectual property. The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to, development and regulatory success, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations, and the ability to secure additional capital to fund operations. The Company’s major sources of cash have been licensing revenue, proceeds from various public and private offerings of its capital stock, option exercises and interest income. As of June 30, 2024, the Company had approximately $77.0 million in cash, cash equivalents and marketable securities. Since inception, the Company has generated $223.2 million in revenue, primarily from the Company’s royalty, license and termination agreement (“RLT Agreement”) with Takeda Pharmaceutical Company Limited (“Takeda”). For most periods, the Company has incurred recurring losses, has experienced negative operating cash flows and has required significant cash resources to execute its business plans, which the Company expects will continue for the foreseeable future. The Company has an accumulated deficit of $281.0 million as of June 30, 2024, working capital of $66.8 million and had cash used in operating activities of $30.9 million for the six months ended June 30, 2024. The Company recorded net income of $8.5 million due to an adjustment to a royalty monetization liability (see Note 2 “Summary of Significant Accounting Policies” below) and net loss of $3.2 million during the three and six months ended June 30, 2024, respectively, and expects to incur losses in subsequent periods for at least the next several years. The Company is highly dependent on its ability to find additional sources of funding through either equity offerings, debt financings, collaborations, strategic alliances, licensing agreements or a combination of any such transactions. Management believes that the Company’s existing cash, cash equivalents and marketable securities as of June 30, 2024 will be sufficient to fund its current operating plans through at least the next 12 months from the date of filing of this Quarterly Report on Form 10-Q. Adequate additional funding may not be available to the Company on acceptable terms or at all. The failure to raise capital as and when needed could have a negative impact on the Company’s financial condition and ability to pursue its business strategy. The Company may be required to delay, reduce the scope of or eliminate research and development programs, or obtain funds through arrangements with collaborators or others that may require the Company to relinquish rights to certain drug candidates that the Company might otherwise seek to develop or commercialize independently. The Company is subject to other challenges and risks specific to its business and its ability to execute on its strategy, as well as risks and uncertainties common to companies in the pharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: delays or problems in the supply of the Company’s product candidates, loss of single source suppliers or failure to comply with manufacturing regulations; identifying, acquiring or in-licensing additional products or product candidates; pharmaceutical product development and the inherent uncertainty of clinical success; the challenges of protecting and enhancing intellectual property rights; complying with applicable regulatory requirements; and obtaining regulatory approval of any of the Company’s product candidates.

SUMMARY OF SIGNIFICANT ACCOUNTI

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES	6 Months Ended
	Jun. 30, 2024
Accounting Policies [Abstract]
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES	SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES The Company’s significant accounting policies are described in Note 2, “Summary of Significant Accounting Policies,” in the Company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (“SEC”) on March 8, 2024. (A) Unaudited Interim Condensed Consolidated Financial Statements The interim condensed consolidated balance sheet at June 30, 2024 and the condensed consolidated statements of operations, comprehensive income (loss), cash flows, and stockholders’ equity for the three and six months ended June 30, 2024 and 2023 are unaudited. The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) and following the requirements of the SEC for interim reporting. As permitted under those rules, certain notes or other financial information that are normally required by GAAP are condensed or omitted. These condensed consolidated financial statements have been prepared on the same basis as the Company’s annual financial statements and, in the opinion of management, reflect all adjustments, consisting only of normal recurring adjustments that are necessary for a fair statement of its financial information. The results of operations for the three and six month periods ended June 30, 2024 and 2023 are not necessarily indicative of the results to be expected for the year ending December 31, 2024 or for any other future annual or interim period. The balance sheet as of December 31, 2023 included herein was derived from the audited financial statements as of that date. These interim condensed consolidated financial statements should be read in conjunction with the Company’s audited financial statements as of and for the year ended December 31, 2023 included in the Company’s Annual Report on Form 10-K. (B) Basis of Presentation and Consolidation The accompanying condensed consolidated financial statements have been prepared in conformity with GAAP and include the accounts of Ovid Therapeutics Inc. and its wholly owned subsidiaries, Ovid Therapeutics Hong Kong Limited and Ovid Therapeutics Australia Pty Ltd. All intercompany transactions and balances have been eliminated in consolidation. (C) Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of income and expenses during the reporting period. Actual results could differ materially from those estimates. (D) Marketable Securities Marketable securities consist of investments in U.S. treasury instruments which are considered available-for-sale securities. The Company classifies its marketable securities with maturities of less than one year from the balance sheet date as current assets on its condensed consolidated balance sheets. The Company classifies its marketable securities with original maturities of less than three months as cash equivalents on its condensed consolidated balance sheets. Unrealized gains and losses on these securities that are determined to be temporary are reported as a separate component of accumulated other comprehensive (loss) income in stockholders’ equity. (E) Restricted Cash The Company classifies as restricted cash all cash pledged as collateral to secure long-term obligations and all cash whose use is otherwise limited by contractual provisions. Amounts are reported as non-current unless restrictions are expected to be released in the next 12 months. (F) Long-Term Equity Investments Long-term equity investments consist of equity investments in the preferred shares of Gensaic, Inc., formerly M13 Therapeutics, Inc. (“Gensaic”), and Graviton Bioscience Corporation (“Graviton”), both privately held corporations. The preferred shares are not considered in-substance common stock, and the investments are accounted for at cost, with adjustments for observable changes in prices or impairments, and are classified within long-term equity investments on the condensed consolidated balance sheets with adjustments recognized in other income (expense), net on the condensed consolidated statements of operations. The Company has determined that these equity investments do not have a readily determinable fair value and elected the measurement alternative. Therefore, the carrying amount of the equity investments will be adjusted to fair value at the time of the next observable price change for the identical or similar investment of the same issuer or when an impairment is recognized. Each reporting period, the Company performs a qualitative assessment to evaluate whether the investments are impaired. The assessment includes a review of recent operating results and trends, recent sales/acquisitions of the investees' securities, and other publicly available data. If an investment is determined to be impaired, the Company will then write it down to its estimated fair value. As of June 30, 2024 and December 31, 2023, the equity investment in Gensaic had a carrying value of $5.1 million. As of June 30, 2024 and December 31, 2023, the equity investment in Graviton had a carrying value of $15.8 million and $11.2 million, respectively. The initial investment in Graviton was $10.0 million, and cumulative measurement adjustments totaling $5.8 million have been recognized. The Company’s equity investments are assessed quarterly and increases have been based upon change in observable price. Long-term equity investments also consist of an equity investment in the common shares of Marinus Pharmaceuticals, Inc. (“Marinus”) that were received as non-cash consideration via the terms of a licensing agreement executed between the two companies effective March 2022. The equity shares are marked-to-market at each reporting date with changes in the fair value being reflected in the carrying value of the investment on the Company’s condensed consolidated balance sheets and other income (expense), net on the Company’s condensed consolidated statements of operations. As of June 30, 2024 and December 31, 2023, the equity investment in Marinus had a carrying value of $0.1 million and $1.3 million, respectively. (G) Fair Value of Financial Instruments Financial Accounting Standards Board (“FASB”) guidance specifies a hierarchy of valuation techniques based on whether the inputs to those valuation techniques are observable or unobservable. Observable inputs reflect market data obtained from independent sources, while unobservable inputs reflect market assumptions. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurement) and the lowest priority to unobservable inputs (Level 3 measurement). The three levels of the fair value hierarchy are as follows: • Level 1—Unadjusted quoted prices in active markets for identical assets or liabilities that the reporting entity has the ability to access at the measurement date. Level 1 primarily consists of financial instruments whose value is based on quoted market prices such as exchange-traded instruments and listed equities. The Company’s Level 1 assets consisted of investments in a U.S. treasury money market fund and equity securities totaling $18.9 million as of June 30, 2024 and $25.7 million as of December 31, 2023. • Level 2—Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly (e.g., quoted prices of similar assets or liabilities in active markets, or quoted prices for identical or similar assets or liabilities in markets that are not active). Level 2 includes financial instruments that are valued using models or other valuation methodologies. The Company’s Level 2 assets consisted of U.S. treasury bills, totaling $57.2 million as of June 30, 2024 and $78.8 million as of December 31, 2023. • Level 3—Unobservable inputs for the asset or liability. Financial instruments are considered Level 3 when their fair values are determined using pricing models, discounted cash flows or similar techniques and at least one significant model assumption or input is unobservable. The Company’s Level 3 liabilities consist of a royalty monetization liability that was valued at $972,000 as of June 30, 2024 and $30.0 million as of December 31, 2023. There were no Level 3 assets as of June 30, 2024 or December 31, 2023. The following table presents information about liabilities measured at fair value on a recurring basis and for which the Company utilizes Level 3 inputs to determine fair value. Royalty Monetization Liability (in thousands) June 30, 2024 Balance, beginning of period $ 30,000 Change in fair value of royalty monetization liability (29,028) Balance, end of period $ 972 The Company estimated the fair value of the royalty monetization liability using a probability-weighted discounted cash flow valuation based on the estimated future sales of soticlestat. Using this approach, the estimated future sales of soticlestat are calculated over the expected life of the agreement using certain unobservable inputs. The unobservable inputs include: the estimated probability of FDA approval for commercialization of soticlestat, the estimated future sales forecast for soticlestat, and the discount rate used to present value the probability-weighted estimated future sales of soticlestat. The royalty monetization liability is classified as a Level 3 liability as its valuation requires substantial judgment and estimation of factors that are not observable. If different input assumptions were used for the valuation, the estimated fair value could be significantly higher or lower than the fair value determined. See “Royalty Monetization Liability” in this Note 2 (L) below. The carrying amounts reported in the balance sheets for cash and cash equivalents, other current assets, accounts payable and accrued expenses approximate their fair values based on the short-term maturity of these instruments. (H) Leases The Company determines if an arrangement is a lease at inception and recognizes the lease in accordance with FASB Accounting Standards Codification (“ASC”) 842. Operating leases are included in right-of-use (“ROU”) assets, current liabilities, and long-term lease liability in the Company's condensed consolidated balance sheets. ROU assets represent the right to use an underlying asset for the lease term and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Operating lease ROU assets and liabilities are recognized at the lease commencement date based on the present value of the lease payments over the lease term. The Company determines the portion of the lease liability that is current as the difference between the calculated lease liability at the end of the current period and the lease liability that is projected 12 months from the current period. (I) Property and Equipment Property and equipment are stated at cost and depreciated over their estimated useful lives of three years using the straight-line method. Repair and maintenance costs are expensed. The Company reviews the recoverability of all long-lived assets, including the related useful life, whenever events or changes in circumstances indicate that the carrying amount of a long-lived asset might not be recoverable. (J) Research and Development Expenses The Company expenses the cost of research and development as incurred. Research and development expenses are comprised of costs incurred in performing research and development activities, including clinical trial costs, manufacturing costs for both clinical and preclinical materials as well as contracted services, license fees, and other external costs. Research and development expenses also include the cost of licensing agreements acquired from third parties. Nonrefundable advance payments for goods and services that will be used in future research and development activities are expensed when the activity is performed or when the goods have been received in accordance with ASC 730, Research and Development. (K) Stock-Based Compensation The Company accounts for its stock-based compensation in accordance with ASC 718, Compensation—Stock Compensation, which establishes accounting for stock-based awards granted to employees for services and requires companies to expense the estimated fair value of these awards over the requisite service period. The Company estimates the fair value of all awards granted using the Black-Scholes valuation model. Key inputs and assumptions include the expected term of the option, stock price volatility, risk-free interest rate, dividend yield, stock price and exercise price. Many of the assumptions require significant judgment and any changes could have an impact in the determination of stock-based compensation expense. The Company elected an accounting policy to record forfeitures as they occur. The Company recognizes employee stock-based compensation expense based on the fair value of the award on the date of the grant. The compensation expense is recognized over the vesting period under the straight-line method. The Company accounts for option awards granted to nonemployee consultants and directors in accordance with ASC 718. The fair value of the option issued or committed to be issued is used to measure the transaction, as this is more reliable than the fair value of the services received. The fair value is measured at the value of the Company’s common stock at the earlier of the date that the commitment for performance by the counterparty has been reached or the counterparty’s performance is complete. (L) Royalty Monetization Liability The Company accounted for its sale to Ligand Pharmaceuticals Incorporated (“Ligand”) under the purchase and sale agreement (the “Ligand Agreement”) of a 13% share of royalties and milestones owed to the Company pursuant to the RLT Agreement with Takeda related to the potential approval and commercialization of soticlestat in accordance with ASC 470, Debt, which addresses situations in which an entity receives cash from an investor in return for an agreement to pay the investor a specified percentage of the revenue from a contractual right. The Company classified the proceeds received from the sale to Ligand as debt as the Company determined that it had significant continuing involvement in the generation of the cash flows to Ligand. The Company further elected to account for the debt at fair value in accordance with ASC 825, Financial Instruments, which permits a company to elect the fair value option on an instrument specific basis for a recognized financial liability that is not specifically excluded. If commercialized, the Company will recognize 100% of the royalties and milestones received for sales of soticlestat as revenue and the 13% share of royalties payable to Ligand as a cash outflow from financing activities in the condensed consolidated statements of cash flows. Changes in the fair value of the debt will be classified as a component of other income (expense), net in the condensed consolidated statements of operations. In June 2024, Takeda reported Phase 3 topline study results for soticlestat, noting that soticlestat narrowly missed its primary endpoint and showed clinically meaningful and significant effects in multiple key secondary efficacy endpoints with respect to Dravet syndrome and missed its primary endpoint with respect to Lennox-Gastaut syndrome. Based on the study results, the Company’s management reassessed certain assumptions for soticlestat that factor into the valuation of the royalty monetization liability and determined the probability of potential commercialization related to Dravet syndrome to be 7.5% and the probability of potential commercialization related to Lennox-Gastaut syndrome to be 0%. A discount rate of 15% was utilized in calculating the change to the royalty monetization liability. The impact on the fair value resulted in a $29.0 million reduction in the royalty monetization liability which was recognized as other income (expense), net in the statement of operations for the period ended June 30, 2024. Given that the two Phase 3 Takeda trials evaluating soticlestat for the treatment of Dravet and Lennox-Gastaut syndromes did not meet their primary endpoints, it is uncertain whether Takeda will continue to progress, or elect to terminate the development of soticlestat as contemplated by the RLT Agreement, in which case we may not receive some or all of the royalty and milestone payments under the RLT Agreement. See Note 11 – Collaboration and License Agreements for a description of the RLT Agreement. (M) Income Taxes The Company accounts for income taxes under the asset and liability method, which requires deferred tax assets and liabilities to be recognized for the estimated future tax consequences attributable to differences between financial statement carrying amounts and respective tax bases of existing assets and liabilities, as well as for net operating loss carryforwards and research and development credits. Valuation allowances are provided if it is more likely than not that some portion or all of the deferred tax assets will not be realized. The impact of a change in the tax laws is recorded in the period in which the law is enacted. (N) Net Loss per Share Net loss per common share is determined by dividing net loss attributable to common stockholders by the basic and diluted weighted-average common shares outstanding during the period. The Company applies the two-class method to allocate earnings between common stock and participating securities. When applicable, net income per diluted share attributable to common stockholders adjusts the basic earnings per share attributable to common stockholders and the weighted-average number of shares of common stock outstanding for the potential dilutive impact of stock options using the treasury-stock method and the potential impact of preferred stock using the if-converted method. (O) Revenue Recognition Under ASC 606, Revenue from Contracts with Customers, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration that the entity expects to receive in exchange for those goods or services. In applying ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the promises and 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) it satisfies the performance obligations. The Company only applies the five-step model to contracts when it is probable that it will collect the consideration to which it is entitled in exchange for the goods or services the Company transfers to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract, determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. Prior to recognizing revenue, the Company makes estimates of the transaction price, including variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. If there are multiple distinct performance obligations, the Company allocates the transaction price to each distinct performance obligation based on its relative standalone selling price. The standalone selling price is generally determined using expected cost and comparable transactions. Revenue for performance obligations recognized over time is recognized by measuring the progress toward complete satisfaction of the performance obligations using an input measure. Non-refundable upfront fees allocated to licenses that are not contingent on any future performance and require no consequential continuing involvement by the Company, are recognized as revenue when the license term commences and the licensed data, technology or product is delivered. The Company defers recognition of upfront license fees if the performance obligations are not satisfied. (P) Recent Accounting Pronouncements The Company has reviewed recently issued accounting standards and plans to adopt those that are applicable. The Company will adopt ASU 2023-7, Segment Reporting (Topic 280): "Improvement to Reportable Segment Disclosures" ("ASU 2023-07"), beginning with its fiscal year ended December 31, 2024. ASU 2023-07 introduces a new requirement to disclose significant segment expenses regularly provided to the chief operating decision maker (“CODM”), extends certain annual disclosures to interim periods, clarifies that single reportable segment entities must apply ASC 280 in its entirety, permits more than one measure of segment profit or loss to be reported under certain conditions, and requires disclosure of the title and position of the CODM. The Company does not expect the adoption of these standards to have a material impact on its financial position, results of operations, or cash flows. The Company adopts new pronouncements relating to generally accepted accounting principles applicable to the Company as they are issued, which may be in advance of their effective date. Management does not believe that any recently issued, but not yet effective accounting standards, if currently adopted, would have a material effect on the accompanying financial statements.

CASH, CASH EQUIVALENTS AND MARK

CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES	6 Months Ended
	Jun. 30, 2024
Cash and Cash Equivalents [Abstract]
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES	CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES The following tables summarize the fair value of cash, cash equivalents and marketable securities as well as gross unrealized holding gains and losses as of June 30, 2024 and December 31, 2023: June 30, 2024 (in thousands) Amortized Cost Gross Unrealized Holding Gains Gross Unrealized Holding Losses Fair Value Cash $ 1,037 $ — $ — $ 1,037 Cash equivalents 28,659 — (2) 28,657 Marketable securities 47,292 — (12) 47,280 Total cash, cash equivalents and marketable securities $ 76,988 $ — $ (14) $ 76,974 December 31, 2023 (in thousands) Amortized Cost Gross Unrealized Holding Gains Gross Unrealized Holding Losses Fair Value Cash $ 2,701 $ — $ — $ 2,701 Cash equivalents 24,340 — — 24,340 Marketable securities 78,791 1 — 78,792 Total cash, cash equivalents and marketable securities $ 105,832 $ 1 $ — $ 105,833 The Company did not hold any securities that were in an unrealized loss position for more than 12 months as of June 30, 2024 and December 31, 2023. There were no material realized gains or losses on available-for-sale securities during the six months ended June 30, 2024 and 2023.

PROPERTY AND EQUIPMENT AND INTA

PROPERTY AND EQUIPMENT AND INTANGIBLE ASSETS	6 Months Ended
	Jun. 30, 2024
Property, Plant and Equipment [Abstract]
PROPERTY AND EQUIPMENT AND INTANGIBLE ASSETS	PROPERTY AND EQUIPMENT AND INTANGIBLE ASSETS Property and equipment is summarized as follows: (in thousands) June 30, December 31, Furniture and equipment $ 1,517 $ 1,463 Leasehold improvements 306 306 Less accumulated depreciation (1,205) (1,001) Total property and equipment, net $ 618 $ 769 Depreciation expense was $103,000 and $105,000 for the three months ended June 30, 2024 and 2023, respectively. Depreciation expense was $204,000 and $214,000 for the six months ended June 30, 2024 and 2023, respectively. Intangible assets, net of accumulated amortization, were $219,000 and $186,000 as of June 30, 2024 and December 31, 2023, respectively, and are included in other assets. Amortization expense was $45,000 and $32,000 for the three months ended June 30, 2024 and 2023, respectively. Amortization expense was $80,000 and $63,000 for the six months ended June 30, 2024 and 2023, respectively.

LEASES

LEASES	6 Months Ended
	Jun. 30, 2024
Leases [Abstract]
LEASES	LEASES During September 2021, the Company entered into a 10-year lease agreement for its corporate headquarters with a term commencing March 10, 2022, for approximately 19,000 square feet of office space at Hudson Commons in New York, New York. The lease provides for monthly rental payments over the lease term. The base rent under the lease is currently $2.3 million per year. Rent payments commenced 10 months following the commencement date of the lease, or January 10, 2023, and continue for 10 years following the rent commencement date. The Company issued a letter of credit in the amount of $1.9 million in association with the execution of the lease agreement; the letter of credit is characterized as restricted cash on the Company’s condensed consolidated balance sheets. The Hudson Commons lease has a remaining lease term of approximately nine years and includes a single renewal option for an additional five years. The Company did not include the renewal option in the lease term when calculating the lease liability as the Company is not reasonably certain that it will exercise the renewal option. The present value of the lease payments was calculated using an incremental borrowing rate of 7.02%. Lease expense is included in general and administrative and research and development expenses in the condensed consolidated statements of operations. ROU asset and lease liabilities related to the Company’s operating lease are as follows: (in thousands) June 30, ROU asset, net $ 13,357 Current lease liability 1,291 Long-term lease liability $ 14,099 The components of operating lease cost for the six months ended June 30, 2024 were as follows: (in thousands) June 30, Operating lease cost $ 1,084 Variable lease cost — Short-term lease cost — Future minimum commitments under the non-cancelable operating lease are as follows: (in thousands) 2024 1,158 2025 2,316 2026 2,316 2027 2,316 2028 2,469 Thereafter 9,878 Total lease payments $ 20,453

ACCRUED EXPENSES

ACCRUED EXPENSES	6 Months Ended
	Jun. 30, 2024
Payables and Accruals [Abstract]
ACCRUED EXPENSES	ACCRUED EXPENSES Accrued expenses consist of the following: (in thousands) June 30, December 31, Payroll and bonus accrual $ 4,947 $ 4,277 Research and development accrual 2,170 1,396 Professional fees accrual 622 522 Other 369 331 Total $ 8,108 $ 6,525

STOCKHOLDERS_ EQUITY

STOCKHOLDERS’ EQUITY	6 Months Ended
	Jun. 30, 2024
Equity [Abstract]
STOCKHOLDERS’ EQUITY	STOCKHOLDERS’ EQUITY The Company’s capital structure consists of common stock and preferred stock. Pursuant to the Company’s amended and restated certificate of incorporation, as amended, the Company is authorized to issue up to 125,000,000 shares of common stock and 10,000,000 shares of preferred stock. The Company has designated 1,250 of the 10,000,000 authorized shares of preferred stock as non-voting Series A Convertible Preferred Stock (“Series A Preferred Stock”). The holders of common stock are entitled to one vote for each share held. The holders of common stock have no preemptive or other subscription rights, and there are no redemption or sinking fund provisions with respect to such shares. Subject to preferences that may apply to any outstanding series of preferred stock, holders of the common stock are entitled to receive ratably any dividends declared on a non-cumulative basis. The common stock is subordinate to all series of preferred stock with respect to rights upon liquidation, winding up and dissolution of the Company. The holders of common stock are entitled to liquidation proceeds after all liquidation preferences for the preferred stock are satisfied. There were 1,250 shares of Series A Preferred Stock outstanding as of June 30, 2024 and December 31, 2023. Each share of Series A Preferred Stock is convertible into 1,000 shares of common stock at any time at the holder’s option. However, the holder will be prohibited, subject to certain exceptions, from converting shares of Series A Preferred Stock into shares of common stock if, as a result of such conversion, the holder, together with its affiliates, would own more than, at the written election of the holder, either 9.99% or 14.99% of the total number of shares of common stock then issued and outstanding, which percentage may be changed at the holder’s election to any other number less than or equal to 19.99% upon 61 days’ notice to the Company; provided, however, that effective 61 days after delivery of such notice, such beneficial ownership limitations shall not be applicable to any holder that beneficially owns either 10.0% or 15.0%, as applicable based on the holder’s initial written election noted above, of the total number of shares of common stock issued and outstanding immediately prior to delivery of such notice. In the event of a liquidation, dissolution, or winding up of the Company, holders of Series A Preferred Stock will receive a payment equal to $0.001 per share of Series A Preferred Stock before any proceeds are distributed to the holders of common stock. Dividends Through June 30, 2024, the Company has not declared any dividends. No dividends on the common stock shall be declared and paid unless dividends on the preferred stock have been declared and paid.

STOCK-BASED COMPENSATION

STOCK-BASED COMPENSATION	6 Months Ended
	Jun. 30, 2024
Share-Based Payment Arrangement [Abstract]
STOCK-BASED COMPENSATION	STOCK-BASED COMPENSATION The Company’s Board of Directors adopted, and the Company’s stockholders approved, the 2017 Equity Incentive Plan (“2017 Plan”), which became effective on May 4, 2017. The initial reserve of shares of common stock issuable under the 2017 Plan was 3,052,059 shares. The 2017 Plan provides for the grant of incentive stock options, non-statutory stock options, restricted stock awards, restricted stock unit awards, stock appreciation rights, performance-based stock awards, and other forms of stock-based awards. Additionally, the 2017 Plan provides for the grant of performance cash awards. The Company’s employees, officers, directors, consultants and advisors are eligible to receive awards under the 2017 Plan. Following the adoption of the 2017 Plan, no further awards will be granted under the Company’s prior plan. Pursuant to the terms of the 2017 Plan, on each January 1st, the plan limit shall be increased by the lesser of (x) 5% of the number of shares of common stock outstanding as of the immediately preceding December 31 and (y) such lesser number as the Board of Directors may determine at its discretion. On January 1, 2024 and January 1, 2023 an additional 3,534,599 and 3,523,344 shares, respectively, were reserved for issuance under the 2017 Plan. As of June 30, 2024, there were 5,644,244 shares of the Company’s common stock reserved and available for issuance under the 2017 Plan. The Company’s Board of Directors adopted, and the Company’s stockholders approved, the 2017 Employee Stock Purchase Plan (“2017 ESPP”), which became effective on May 4, 2017. The initial reserve of shares of common stock issuable under the 2017 ESPP was 279,069 shares. The 2017 ESPP allows employees to purchase common stock of the Company at a 15% discount to the market price on designated semi-annual purchase dates. During the three months ended June 30, 2024 and 2023, no shares were purchased under the 2017 ESPP, and the Company recorded expense of $18,000 and $13,000, respectively. The number of shares of common stock reserved for issuance under the 2017 ESPP automatically increases on January 1 of each year, beginning on January 1, 2018 and continuing through and including January 1, 2027, by the lesser of (i) 1% of the total number of shares of the Company’s common stock outstanding on December 31 of the preceding calendar year, (ii) 550,000 shares or (iii) such lesser number of shares determined by the Board of Directors. The Board of Directors acted prior to each of January 1, 2024 and January 1, 2023 to provide that there be no increase in the number of shares reserved for issuance under the 2017 ESPP on either such date. As of June 30, 2024, there were 321,285 shares of the Company’s common stock reserved and available for issuance under the 2017 ESPP. The Company’s Board of Directors adopted and the Company’s stockholders approved the 2014 Equity Incentive Plan (“2014 Plan”), which authorized the Company to grant shares of common stock in the form of incentive stock options, non-statutory stock options, stock appreciation rights, restricted stock and restricted stock units. The 2014 Plan was terminated as to future awards in May 2017, although it continues to govern the terms of options that remain outstanding under the 2014 Plan. No additional stock awards will be granted under the 2014 Plan, and all outstanding stock awards granted under the 2014 Plan that are repurchased, forfeited, expire or are cancelled will become available for grant under the 2017 Plan in accordance with its terms. As of June 30, 2024, options to purchase 1,356,621 shares of common stock were outstanding under the 2014 Plan. Unless specified otherwise in an individual option agreement, stock options granted under the 2014 Plan and the 2017 Plan generally have a ten-year term and a four-year graded vesting period. The vesting requirement is generally conditioned upon the grantee’s continued service with the Company during the vesting period. Once vested, all options granted are exercisable from the date of grant until they expire. The option grants are non-transferable. Vested options generally remain exercisable for 90 days under the 2017 Plan and 30 days under the 2014 Plan subsequent to the termination of the option holder’s service with the Company. In the event of the option holder’s death or disability while employed by or providing service to the Company, the exercisable period extends to 18 months or 12 months, respectively, under the 2017 Plan and six months under the 2014 Plan. The fair value of options granted during the three and six months ended June 30, 2024 and 2023 was estimated using the Black-Scholes option valuation model. The inputs for the Black-Scholes option valuation model require significant assumptions that are detailed in the table below. The risk-free interest rates are based on the rate for U.S. Treasury securities at the date of grant with maturity dates approximately equal to the expected life at the grant date. The expected life is based on the simplified method in accordance with the SEC Staff Accounting Bulletin No. Topic 14D. Beginning January 1, 2023, the expected volatility is estimated based on the historical volatility of the Company since the Company’s initial public offering. The Company granted 80,000 and 270,000 stock options to employees during the three months ended June 30, 2024 and 2023, respectively. The Company granted 2,752,150 and 2,950,500 stock options to employees during the six months ended June 30, 2024 and 2023, respectively. There were 5,807,555 and 6,710,485 unvested employee options outstanding as of June 30, 2024, and 2023, respectively. Total expense recognized related to the employee stock options for the three months ended June 30, 2024 and 2023 was $1.6 million and $1.8 million, respectively. Total expense recognized related to the employee stock options for the six months ended June 30, 2024 and 2023 was $3.5 million and $3.6 million, respectively. Total unrecognized compensation expense related to employee stock options was $13.9 million as of June 30, 2024. The Company did not recognize any expense for employee performance-based option awards during the three months ended June 30, 2024 and 2023. The Company granted zero and 50,000 stock options to nonemployee consultants for services rendered during the three months ended June 30, 2024 and 2023, respectively. The Company granted 250,000 and 50,000 stock options to nonemployee consultants for services rendered during the six months ended June 30, 2024 and 2023, respectively. There were 268,959 and 130,834 unvested nonemployee options outstanding as of June 30, 2024 and 2023, respectively. Total expense recognized related to nonemployee stock options for the three months ended June 30, 2024 and 2023 was $98,000 and $163,000, respectively. Total expense recognized related to nonemployee stock options for the six months ended June 30, 2024 and 2023 was $157,000 and $272,000, respectively. Total unrecognized compensation expenses related to the nonemployee stock options was $0.1 million as of June 30, 2024. The Company did not recognize any expense for nonemployee performance-based option awards during the three and six months ended June 30, 2024 and 2023. The Company granted 348,575 restricted stock units to employees during the three and six months ended June 30, 2024. No restricted stock units were granted by the Company in prior periods. The restricted stock units granted will vest in equal installments over three years, beginning January 1, 2025 and otherwise have similar terms to the Company’s stock option grants. The Company’s stock-based compensation expense was recognized in operating expenses as follows: Three Months Ended Six Months Ended (in thousands) June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 Research and development $ 537 $ 580 $ 1,066 $ 1,090 General and administrative 1,203 1,369 2,642 2,775 Total $ 1,740 $ 1,948 $ 3,708 $ 3,865 Three Months Ended Six Months Ended (in thousands) June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 Stock options and restricted stock units $ 1,722 $ 1,935 $ 3,675 $ 3,837 Employee Stock Purchase Plan 18 13 33 29 Total $ 1,740 $ 1,948 $ 3,708 $ 3,865 The fair value of employee options granted during the three and six months ended June 30, 2024 and 2023 was estimated utilizing the following assumptions: Three Months Ended Six Months Ended June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 Weighted Weighted Weighted Weighted Volatility 79.01 % 84.11 % 79.82 % 84.56 % Expected term in years 6.08 6.08 6.05 6.07 Dividend rate 0.00 % 0.00 % 0.00 % 0.00 % Risk-free interest rate 4.22 % 3.63 % 4.32 % 3.97 % Fair value of option on grant date $ 1.75 $ 2.66 $ 2.60 $ 1.92 The fair value of nonemployee options granted during the three and six months ended June 30, 2024 and 2023 was estimated utilizing the following assumptions: Three Months Ended Six Months Ended June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 Weighted Weighted Weighted Weighted Volatility 80.59 % 83.00 % 80.59 % 83.00 % Expected term in years 6.50 5.25 6.50 5.25 Dividend rate 0.00 % 0.00 % 0.00 % 0.00 % Risk-free interest rate 4.35 % 3.89 % 4.35 % 3.89 % Fair value of option on grant date $ 2.71 $ 2.42 $ 2.71 $ 2.42 The following table summarizes the number of options outstanding and the weighted average exercise price: Number of Shares Weighted Weighted Aggregate Options outstanding December 31, 2023 15,124,546 $ 3.87 6.90 $ 5,212,286 Granted 2,752,150 3.66 6.96 Exercised (248,024) 2.05 Forfeited or expired (796,360) 6.48 Options outstanding June 30, 2024 16,832,312 $ 3.74 6.96 $ 12,790 Vested and exercisable at June 30, 2024 10,755,798 $ 4.06 5.92 $ 12,790 At June 30, 2024, there was approximately $13.9 million of unrecognized stock–based compensation expense related to employee and nonemployee grants, which is expected to be recognized over a remaining average vesting period of 2.51 years.

INCOME TAXES

INCOME TAXES	6 Months Ended
	Jun. 30, 2024
Income Tax Disclosure [Abstract]
INCOME TAXES	INCOME TAXES The Company’s interim income tax provision consists of U.S. federal and state income taxes based on the estimated annual effective tax rate that the Company expects for the full year together with the tax effect of discrete items. Each quarter the Company updates its estimate of the annual effective tax rate and records cumulative adjustments as necessary. As of June 30, 2024, the Company was in a pre-tax loss position, and is anticipated to remain so throughout the year. For the six months ended June 30, 2024, the Company did not record any tax benefit or expense. In assessing the realizability of deferred tax assets, management evaluates whether it is more likely than not that some portion or all of the deferred tax assets will be realized. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income in those periods in which temporary differences become deductible and/or net operating losses can be utilized. Management assesses all positive and negative evidence when determining the amount of the net deferred tax assets that are more likely than not to be realized. This evidence includes, but is not limited to, prior earnings history, scheduled reversal of taxable temporary differences, tax planning strategies and projected future taxable income. Significant weight is given to positive and negative evidence that is objectively verifiable. Based on these factors, including cumulative losses in recent years, the Company continues to maintain a full valuation allowance against its net deferred tax assets as of June 30, 2024.

COMMITMENTS AND CONTINGENCIES

COMMITMENTS AND CONTINGENCIES	6 Months Ended
	Jun. 30, 2024
Commitments and Contingencies Disclosure [Abstract]
COMMITMENTS AND CONTINGENCIES	COMMITMENTS AND CONTINGENCIES License Agreements Northwestern University License Agreement In December 2016, the Company entered into a license agreement (“Northwestern Agreement”) with Northwestern University (“Northwestern”), pursuant to which Northwestern granted the Company an exclusive, worldwide license to patent rights of certain inventions (“Northwestern Patent Rights”) which relate to a specific compound and related methods of use for such compound, along with certain know-how related to the practice of the inventions claimed in the Northwestern Patent Rights. The Company is developing OV329 under this agreement. Under the Northwestern Agreement, the Company was granted exclusive rights to research, develop, manufacture and commercialize products utilizing the Northwestern Patent Rights for all uses. The Company has agreed that it will not use the Northwestern Patent Rights to develop any products for the treatment of cancer, but Northwestern may not grant rights in the technology to others for use in cancer. The Company also has an option, exercisable during the term of the agreement to an exclusive license under certain intellectual property rights covering novel compounds with the same or similar mechanism of action as the primary compound that is the subject of the license agreement. Northwestern has retained the right, on behalf of itself and other non-profit institutions, to use the Northwestern Patent Rights and practice the inventions claimed therein for educational and research purposes and to publish information about the inventions covered by the Northwestern Patent Rights. Upon entry into the Northwestern Agreement, the Company paid an upfront non-creditable one-time license issuance fee of $75,000, and is required to pay an annual license maintenance fee of $20,000, which will be creditable against any royalties payable to Northwestern following first commercial sale of licensed products under the agreement. The Company is responsible for all ongoing costs of filing, prosecuting and maintaining the Northwestern Patent Rights, but also has the right to control such activities using its own patent counsel. In consideration for the rights granted to the Company under the Northwestern Agreement, the Company is required to pay to Northwestern up to an aggregate of $5.3 million upon the achievement of certain development and regulatory milestones for the first product covered by the Northwestern Patent Rights, and upon commercialization of any such products, will be required to pay to Northwestern a tiered royalty on net sales of such products by the Company, its affiliates or sublicensees, at percentages in the low to mid-single-digits, subject to standard reductions and offsets. The Company’s royalty obligations continue on a product-by-product and country-by-country basis until the later of the expiration of the last-to-expire valid claim in a licensed patent covering the applicable product in such country and 10 years following the first commercial sale of such product in such country. If the Company sublicenses a Northwestern Patent Right, it will be obligated to pay to Northwestern a specified percentage of sublicense revenue received by the Company, ranging from the high single-digits to the low-teens. The Northwestern Agreement requires that the Company use commercially reasonable efforts to develop and commercialize at least one product that is covered by the Northwestern Patent Rights. Unless earlier terminated, the Northwestern Agreement will remain in force until the expiration of the Company’s payment obligations thereunder. The Company has the right to terminate the agreement for any reason upon prior written notice or for an uncured material breach by Northwestern. Northwestern may terminate the agreement for the Company’s uncured material breach or insolvency. AstraZeneca AB License Agreement In December 2021, the Company entered into an exclusive license agreement with AstraZeneca AB (“AstraZeneca”), for a library of early-stage small molecules targeting the KCC2 transporter, including lead candidate OV350. Upon execution of the agreement, the Company was obligated to pay an upfront cash payment of $5.0 million and issued shares of the Company’s common stock in an amount that equaled $7.3 million based on the volume-weighted average price of shares of the Company's common stock for the 30 business days immediately preceding the execution date of the transaction. Pursuant to the AstraZeneca license agreement, the Company agreed to potential milestone payments of up to $203.0 million upon the achievement of certain developmental, regulatory and sales milestones. The first payment of $3.0 million is due upon the successful completion of the first Phase 2 clinical study of a licensed product following a positive biomarker readout in a Phase 1 clinical study. Gensaic Collaboration and Option Agreement In August 2022, the Company entered into a collaboration and option agreement (“Gensaic Collaboration Agreement”) with Gensaic. The Gensaic Collaboration Agreement involves the research and development of phage-derived particle (“PDP”) products on Gensaic’s proprietary platform for certain rare central nervous system (“CNS”) disorder targets. Under the Gensaic Collaboration Agreement, Gensaic grants the Company an exclusive option to obtain an exclusive license with respect to certain identified lead PDP products, which are exercisable at any time prior to the expiration of the option period. Once a product is identified by the Company that demonstrates sufficient efficacy, the Company may exercise its option with respect to the specific research program for that PDP product. The Company shall reimburse Gensaic for Gensaic’s research costs related to the specific research plan for PDP products identified. The research plan and budget shall be mutually agreed upon by the parties and shall not exceed $3.0 million in any research year. The Company will record these reimbursement payments as research and development costs in the period the research costs are incurred. In May 2023, the Company identified a lead PDP candidate for further research and provided $3.5 million to Gensaic to support the approved research plan and budget. The amount was expensed as the research and development occurs with the remaining amount included in prepaid expenses and other current assets in the condensed consolidated balance sheets. If a product is ultimately commercialized under this agreement, the Company shall make tiered royalty payments to Gensaic in the mid-single to low double-digit range based on the net sales of all licensed PDP products during the royalty term. The Company is also responsible for potential tiered milestone payments of up to $452.0 million based upon the achievement of certain sales milestone events and developmental milestone approvals for three or more products. Gensaic also has the option to become a collaborative partner in the development and commercialization of PDP products in exchange for a fee based on a percentage of the costs incurred by the Company through the date Gensaic exercises its option. The Company would no longer be required to pay Gensaic royalty or milestone payments if Gensaic elects to exercise its option. The Company may terminate the Gensaic Collaboration Agreement by providing written notice to Gensaic 90 days in advance of the termination date. As of June 30, 2024, none of these contingent payments were considered probable. Contingencies Liabilities for loss contingencies arising from claims, assessments, litigation, fines, and penalties and other sources are recorded when it is probable that a liability has been incurred and the amount can be reasonably estimated. Legal costs incurred in connection with loss contingencies are expensed as incurred. The Company is not currently involved in any legal matters arising in the normal course of business that are material to the Company. Under the terms of their respective employment agreements, certain of our executive officers are eligible to receive severance payments and benefits upon a termination without “cause” or due to “permanent disability,” or upon “resignation for good reason,” contingent upon the executive officer’s delivery to the Company of a satisfactory release of claims, and subject to the executive officer’s compliance with non-competition and non-solicitation restrictive covenants.

COLLABORATION AND LICENSE AGREE

COLLABORATION AND LICENSE AGREEMENTS	6 Months Ended
	Jun. 30, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
COLLABORATION AND LICENSE AGREEMENTS	COLLABORATION AND LICENSE AGREEMENTS Takeda Collaboration In January 2017, the Company entered into a license and collaboration agreement with Takeda under which the Company licensed from Takeda certain exclusive rights to develop and commercialize soticlestat in certain territories. In March 2021, the Company entered into the RLT Agreement, pursuant to which Takeda secured rights to the Company’s 50% global share in soticlestat, and the Company granted to Takeda an exclusive worldwide license under the Company’s relevant intellectual property rights to develop and commercialize the investigational medicine soticlestat for the treatment of developmental and epileptic encephalopathies, including Dravet syndrome and Lennox-Gastaut syndrome. Under the RLT Agreement, all rights in soticlestat are owned by Takeda or exclusively licensed to Takeda by the Company. Takeda assumed all responsibility for, and costs of, both development and commercialization of soticlestat, and the Company no longer has any financial obligation to Takeda under the original collaboration agreement, including milestone payments or any future development and commercialization costs. In March 2021, upon the closing of the RLT Agreement, the Company received an upfront payment of $196.0 million and, if soticlestat is successfully developed, will be eligible to receive up to an additional $660.0 million upon Takeda achieving developmental, regulatory and sales milestones. In addition, the Company will be entitled to receive tiered royalties beginning in the low double-digits, and up to 20% on sales of soticlestat if regulatory approval is achieved. Royalties will be payable on a country-by-country and product-by-product basis for any indications that soticlestat is approved for and sold during the period beginning on the date of the first commercial sale of such product in such country and ending on the later to occur of the expiration of patent rights covering the product in such country and a specified anniversary of such first commercial sale. In October 2023, the Company sold a 13% stake in the royalty, regulatory and commercial milestone payments that the Company is eligible to receive under the RLT Agreement to Ligand for $30.0 million. The Company retained 87% of its interest in soticlestat’s potential royalties and milestones. In the event that soticlestat is not approved and commercialized, the Company has no continuing debt or other obligations to Ligand. In June 2024, Takeda reported the Phase 3 topline study results for soticlestat, noting that soticlestat missed its primary endpoints with respect to Dravet syndrome and Lennox-Gastaut syndromes. Based on the study results, management reassessed certain assumptions for soticlestat that factor into the valuation of the royalty monetization liability and determined the probability of potential commercialization related to Dravet syndrome to be 7.5% and the probability of potential commercialization related to Lennox-Gastaut syndrome to be 0%. The change in valuation assumptions resulted in a $29.0 million reduction in the royalty monetization liability which was recognized as other income in the condensed consolidated statement of operations for the three month period ended June 30, 2024. During the six months ended June 30, 2024 and 2023, no revenue or expense was recognized pursuant to the RLT Agreement. Healx License and Option Agreement In February 2022, the Company entered an exclusive license option agreement (“Healx License and Option Agreement”) with Healx, Ltd. (“Healx”). Under the terms of the Healx License and Option Agreement, Healx secured a one-year option to investigate gaboxadol (“OV101”) as part of a potential combination therapy for Fragile X syndrome in a Phase 1B/2A clinical trial, as well as a treatment for other indications, for an upfront payment of $0.5 million, and fees to support prosecution and maintenance of our relevant intellectual property rights. At the end of the one-year option period, Healx has the option to secure rights to an exclusive license under the Company’s relevant intellectual property rights, in exchange for an additional payment of $2.0 million, development and commercial milestone payments, and low to mid-tier double-digit royalties. On February 1, 2023, the Company granted an extension of the option period for up to four months for Healx to continue to investigate gaboxadol. Royalties are payable on a country-by-country and product-by-product basis during the period beginning on the date of the first commercial sale of such product in such country and ending on the later to occur of the expiration of patent rights covering the product in such country and a specified anniversary of such first commercial sale. In June 2023, the Company entered into an amendment to the Healx License and Option Agreement whereby revisions were made to terms regarding the timing of the option exercise fee payable by Healx to the Company, the clinical and regulatory milestone payment structure, and the royalty payment structure. Additionally, the parties agreed that following the exercise of the option, Healx would assume direct responsibility for patent maintenance and prosecution and that the Company would transfer to Healx all supply obligations with respect to the active pharmaceutical ingredient and finished gaboxadol products and any related licensed technology and know-how in the Company's possession that is relevant to the manufacture of such licensed products. Healx will assume all responsibility for, and costs of, both development and commercialization of gaboxadol following the exercise of the option. The Company will retain the option to co-develop and co-commercialize the program with Healx (“Ovid Opt-In Right”) at the end of a positive readout of clinical Phase 2B and would share net profits and losses in lieu of the milestones and royalty payments. If the Ovid Opt-In Right were exercised, the Company would be required to pay Healx 50% of development costs. The Company does not plan to conduct further trials of gaboxadol. The term of the Healx License and Option Agreement will continue until the later of (a) the expiration of all relevant royalty terms, or in the event that Healx does not exercise its option during the option period defined in the Healx License and Option Agreement, or the Option Period, the expiration of such period, or (b) in the event that Healx does exercise its option during the Option Period, and the Company does not exercise the Ovid Opt-In Right during the period of time it has to opt-in, or the Opt-In Period, or the opt-in terms are otherwise terminated, upon the expiration of all payment obligations, or (c) in the event that Healx does exercise the Option during the Option Period, and the Company does exercise the Ovid Opt-In Right during the Opt-In Period, such time as neither Healx nor the Company is continuing to exploit gaboxadol. Further, if the Company exercises the Ovid Opt-In Right to co-develop and co-commercialize the program, it will owe an equal share of the net profit share to a third party with which it previously established a licensing agreement. If the Company does not exercise the Ovid Opt-In Right, it will owe the third party a share of all milestone and royalty payments. No revenue was recognized relating to the Healx License and Option Agreement during the six months ended June 30, 2024 and 2023. Marinus Pharmaceuticals Out-License Agreement In March 2022, the Company entered into an exclusive patent license agreement with Marinus (“Marinus License Agreement”). Under the Marinus License Agreement, the Company granted Marinus an exclusive, non-transferable (except as expressly provided therein), royalty-bearing right and license under certain Ovid patents relating to ganaxolone to develop, make, have made, commercialize, promote, distribute, sell, offer for sale and import licensed products in the territory (which consists of the United States, the European Economic Area, United Kingdom and Switzerland) for the treatment of CDKL5 deficiency disorders. Following the date of regulatory approval by the FDA of the first licensed product in the territory which was received in March 2022, Marinus issued, at the Company’s option, 123,255 shares of Marinus common stock, par value $0.001 per share, as payment. The Marinus License Agreement also provides for payment of royalties from Marinus to the Company in single-digits on net sales of each such licensed product sold. The Company had unrealized losses on the Marinus common stock of $1.2 million and unrealized gains of $0.8 million for the six months ended June 30, 2024 and 2023, respectively, which were recorded as unrealized gains (losses) on equity securities and are reflected in other income (expense), net in the condensed consolidated statements of operations. Graviton License Agreement and Equity Purchase In April 2023, the Company entered into a collaboration and license agreement with Graviton (“Graviton Agreement”), whereby it secured from Graviton an exclusive license to develop and commercialize Graviton’s library of Rho-associated coiled-coil containing protein kinase 2 (“ROCK2”) inhibitors including their lead program OV888/GV101 in rare CNS disorders (excluding amyotrophic lateral sclerosis) worldwide (excluding China, Hong Kong, Macau and Taiwan). Under the Graviton Agreement, the Company and Graviton are investigating OV888/GV101 in cerebral cavernous malformations as well as Graviton’s library of ROCK2 inhibitors in other rare CNS disorders. The Company will be responsible for all development and commercialization costs of the products. Should the Company receive regulatory approval and commercialize any of Graviton’s ROCK2 inhibitors, it will pay Graviton tiered royalties on net sales ranging from the mid to high-teens. As part of the Graviton Agreement, the Company also purchased shares of Graviton’s preferred stock for $10.0 million. The Company recorded the purchase of the preferred stock as a long-term equity investment on its condensed consolidated balance sheets. In December 2023 and March 2024, the Company recognized unrealized gains on the investment due to observable changes in price and recorded the gains in other income (expense), net, in the condensed consolidated statements of operations.

RELATED PARTY TRANSACTIONS

RELATED PARTY TRANSACTIONS	6 Months Ended
	Jun. 30, 2024
Related Party Transactions [Abstract]
RELATED PARTY TRANSACTIONS	RELATED PARTY TRANSACTIONS In March 2021, the Company entered into the RLT Agreement with Takeda. For a description of the RLT Agreement, see Note 11 – Collaboration and License Agreements.

NET INCOME (LOSS) PER SHARE

NET INCOME (LOSS) PER SHARE	6 Months Ended
	Jun. 30, 2024
Earnings Per Share [Abstract]
NET INCOME (LOSS) PER SHARE	NET INCOME (LOSS) PER SHARE Basic net income (loss) per share is calculated based upon the weighted-average number of common shares outstanding during the period, excluding outstanding stock options that have not yet vested. For any period in which the Company records net income, diluted net income per share is calculated based upon the weighted-average number of common shares outstanding during the period plus the dilutive impact of weighted-average common equivalent shares outstanding during the period resulting from the assumed exercise of outstanding stock options determined under the treasury stock method and the assumed conversion of preferred stock into common shares determined using the if-converted method. Diluted net loss per share is equivalent to the basic net loss per share due to the exclusion of outstanding stock options and convertible preferred stock because the inclusion of these securities would result in an anti-dilutive effect on per share amounts. The basic and diluted net loss per common share is presented in conformity with the two-class method required for participating securities and multiple classes of shares. The Company considers its preferred stock to be participating securities. For any period in which the Company records net income, undistributed earnings allocated to the participating securities are subtracted from net income in determining net income attributable to common stockholders. The undistributed earnings have been allocated based on the participation rights of preferred stock and common shares as if the earnings for the year have been distributed. For periods in which the Company recognizes a net loss, undistributed losses are allocated only to common shares as the participating securities do not contractually participate in the Company’s losses. Basic net loss per share is computed by dividing the net loss attributable to common stockholders by the weighted-average number of common shares outstanding during the period. Participating securities are excluded from basic weighted-average common shares outstanding. The following table summarizes the calculation of basic and diluted net income (loss) per share: For the Three Months Ended June 30, For the Six Months Ended June 30, (in thousands) 2024 2023 2024 2023 Net income (loss) $ 8,521 $ (12,408) $ (3,174) $ (25,765) Net income attributable to participating securities 150 — — — Net income (loss) attributable to common stockholders $ 8,371 $ (12,408) $ (3,174) $ (25,765) For the Three Months Ended June 30, For the Six Months Ended June 30, (in thousands, except share and per share data) 2024 2023 2024 2023 Net income (loss) attributable to common stockholders $ 8,371 $ (12,408) $ (3,174) $ (25,765) Weighted average common shares outstanding used in computing net income (loss) per share - basic 70,916,471 70,534,181 70,816,585 70,512,479 Weighted average common shares outstanding used in computing net income (loss) per share - diluted 71,200,798 70,534,181 70,816,585 70,512,479 Net income (loss) per share, basic $ 0.12 $ (0.18) $ (0.04) $ (0.37) Net income (loss) per share, diluted $ 0.12 $ (0.18) $ (0.04) $ (0.37) The following potentially dilutive securities have been excluded from the computations of diluted weighted-average shares outstanding as they would be anti-dilutive: For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 Stock options to purchase common stock 16,547,985 15,448,835 16,547,985 15,448,835 Common stock issuable upon conversion of Series A convertible preferred stock 1,250,000 1,250,000 1,250,000 1,250,000

ORGANIZATIONAL RESTRUCTURING

ORGANIZATIONAL RESTRUCTURING	6 Months Ended
	Jun. 30, 2024
Restructuring and Related Activities [Abstract]
ORGANIZATIONAL RESTRUCTURING	ORGANIZATIONAL RESTRUCTURING In June 2024, the Company initiated a reduction of its workforce to prioritize its programs and extend its cash runway. The decision, which was approved by the Company’s Board of Directors, was precipitated by Takeda’s report of Phase 3 topline study results for soticlestat, which are described in Note 11 – Collaboration and License Agreements. The workforce reduction included 17 impacted individuals, or approximately 43% of the Company’s headcount, and was recognized in the three month period ended June 30, 2024. Severance costs of approximately $3.4 million related to the organizational restructuring were recognized during the period, and included in operating income (loss) in the line items of the employees’ regular compensation in the condensed consolidated statements of operations. Accrued severance costs of $3.4 million are included in accrued expenses within the condensed consolidated balance sheet.

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended				6 Months Ended
	Jun. 30, 2024	Mar. 31, 2024	Jun. 30, 2023	Mar. 31, 2023	Jun. 30, 2024	Jun. 30, 2023
Pay vs Performance Disclosure
Net income (loss)	$ 8,521	$ (11,694)	$ (12,408)	$ (13,356)	$ (3,174)	$ (25,765)

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended
	Jun. 30, 2024
Trading Arrangements, by Individual
Rule 10b5-1 Arrangement Adopted	false
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false

SUMMARY OF SIGNIFICANT ACCOUN_2

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies)	6 Months Ended
	Jun. 30, 2024
Accounting Policies [Abstract]
Unaudited Interim Condensed Consolidated Financial Statements	Unaudited Interim Condensed Consolidated Financial Statements The interim condensed consolidated balance sheet at June 30, 2024 and the condensed consolidated statements of operations, comprehensive income (loss), cash flows, and stockholders’ equity for the three and six months ended June 30, 2024 and 2023 are unaudited. The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) and following the requirements of the SEC for interim reporting. As permitted under those rules, certain notes or other financial information that are normally required by GAAP are condensed or omitted. These condensed consolidated financial statements have been prepared on the same basis as the Company’s annual financial statements and, in the opinion of management, reflect all adjustments, consisting only of normal recurring adjustments that are necessary for a fair statement of its financial information. The results of operations for the three and six month periods ended June 30, 2024 and 2023 are not necessarily indicative of the results to be expected for the year ending December 31, 2024 or for any other future annual or interim period. The balance sheet as of December 31, 2023 included herein was derived from the audited financial statements as of that date. These interim condensed consolidated financial statements should be read in conjunction with the Company’s audited financial statements as of and for the year ended December 31, 2023 included in the Company’s Annual Report on Form 10-K.
Basis of Presentation and Consolidation	Basis of Presentation and Consolidation The accompanying condensed consolidated financial statements have been prepared in conformity with GAAP and include the accounts of Ovid Therapeutics Inc. and its wholly owned subsidiaries, Ovid Therapeutics Hong Kong Limited and Ovid Therapeutics Australia Pty Ltd. All intercompany transactions and balances have been eliminated in consolidation.
Use of Estimates	Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of income and expenses during the reporting period. Actual results could differ materially from those estimates.
Marketable Securities	Marketable Securities Marketable securities consist of investments in U.S. treasury instruments which are considered available-for-sale securities. The Company classifies its marketable securities with maturities of less than one year from the balance sheet date as current assets on its condensed consolidated balance sheets. The Company classifies its marketable securities with original maturities of less than three months as cash equivalents on its condensed consolidated balance sheets. Unrealized gains and losses on these securities that are determined to be temporary are reported as a separate component of accumulated other comprehensive (loss) income in stockholders’ equity.
Restricted Cash	Restricted Cash The Company classifies as restricted cash all cash pledged as collateral to secure long-term obligations and all cash whose use is otherwise limited by contractual provisions. Amounts are reported as non-current unless restrictions are expected to be released in the next 12 months.
Long-Term Equity Investments	Long-Term Equity Investments Long-term equity investments consist of equity investments in the preferred shares of Gensaic, Inc., formerly M13 Therapeutics, Inc. (“Gensaic”), and Graviton Bioscience Corporation (“Graviton”), both privately held corporations. The preferred shares are not considered in-substance common stock, and the investments are accounted for at cost, with adjustments for observable changes in prices or impairments, and are classified within long-term equity investments on the condensed consolidated balance sheets with adjustments recognized in other income (expense), net on the condensed consolidated statements of operations. The Company has determined that these equity investments do not have a readily determinable fair value and elected the measurement alternative. Therefore, the carrying amount of the equity investments will be adjusted to fair value at the time of the next observable price change for the identical or similar investment of the same issuer or when an impairment is recognized. Each reporting period, the Company performs a qualitative assessment to evaluate whether the investments are impaired. The assessment includes a review of recent operating results and trends, recent sales/acquisitions of the investees' securities, and other publicly available data. If an investment is determined to be impaired, the Company will then write it down to its estimated fair value. As of June 30, 2024 and December 31, 2023, the equity investment in Gensaic had a carrying value of $5.1 million. As of June 30, 2024 and December 31, 2023, the equity investment in Graviton had a carrying value of $15.8 million and $11.2 million, respectively. The initial investment in Graviton was $10.0 million, and cumulative measurement adjustments totaling $5.8 million have been recognized. The Company’s equity investments are assessed quarterly and increases have been based upon change in observable price. Long-term equity investments also consist of an equity investment in the common shares of Marinus Pharmaceuticals, Inc. (“Marinus”) that were received as non-cash consideration via the terms of a licensing agreement executed between the two companies effective March 2022. The equity shares are marked-to-market at each reporting date with changes in the fair value being reflected in the carrying value of the investment on the Company’s condensed consolidated balance sheets and other income (expense), net on the Company’s condensed consolidated statements of operations. As of June 30, 2024 and December 31, 2023, the equity investment in Marinus had a carrying value of $0.1 million and $1.3 million, respectively.
Fair Value of Financial Instruments	Fair Value of Financial Instruments Financial Accounting Standards Board (“FASB”) guidance specifies a hierarchy of valuation techniques based on whether the inputs to those valuation techniques are observable or unobservable. Observable inputs reflect market data obtained from independent sources, while unobservable inputs reflect market assumptions. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurement) and the lowest priority to unobservable inputs (Level 3 measurement). The three levels of the fair value hierarchy are as follows: • Level 1—Unadjusted quoted prices in active markets for identical assets or liabilities that the reporting entity has the ability to access at the measurement date. Level 1 primarily consists of financial instruments whose value is based on quoted market prices such as exchange-traded instruments and listed equities. The Company’s Level 1 assets consisted of investments in a U.S. treasury money market fund and equity securities totaling $18.9 million as of June 30, 2024 and $25.7 million as of December 31, 2023. • Level 2—Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly (e.g., quoted prices of similar assets or liabilities in active markets, or quoted prices for identical or similar assets or liabilities in markets that are not active). Level 2 includes financial instruments that are valued using models or other valuation methodologies. The Company’s Level 2 assets consisted of U.S. treasury bills, totaling $57.2 million as of June 30, 2024 and $78.8 million as of December 31, 2023. • Level 3—Unobservable inputs for the asset or liability. Financial instruments are considered Level 3 when their fair values are determined using pricing models, discounted cash flows or similar techniques and at least one significant model assumption or input is unobservable. The Company’s Level 3 liabilities consist of a royalty monetization liability that was valued at $972,000 as of June 30, 2024 and $30.0 million as of December 31, 2023. There were no Level 3 assets as of June 30, 2024 or December 31, 2023. The following table presents information about liabilities measured at fair value on a recurring basis and for which the Company utilizes Level 3 inputs to determine fair value. Royalty Monetization Liability (in thousands) June 30, 2024 Balance, beginning of period $ 30,000 Change in fair value of royalty monetization liability (29,028) Balance, end of period $ 972 The Company estimated the fair value of the royalty monetization liability using a probability-weighted discounted cash flow valuation based on the estimated future sales of soticlestat. Using this approach, the estimated future sales of soticlestat are calculated over the expected life of the agreement using certain unobservable inputs. The unobservable inputs include: the estimated probability of FDA approval for commercialization of soticlestat, the estimated future sales forecast for soticlestat, and the discount rate used to present value the probability-weighted estimated future sales of soticlestat. The royalty monetization liability is classified as a Level 3 liability as its valuation requires substantial judgment and estimation of factors that are not observable. If different input assumptions were used for the valuation, the estimated fair value could be significantly higher or lower than the fair value determined. See “Royalty Monetization Liability” in this Note 2 (L) below. The carrying amounts reported in the balance sheets for cash and cash equivalents, other current assets, accounts payable and accrued expenses approximate their fair values based on the short-term maturity of these instruments.
Leases	Leases The Company determines if an arrangement is a lease at inception and recognizes the lease in accordance with FASB Accounting Standards Codification (“ASC”) 842. Operating leases are included in right-of-use (“ROU”) assets, current liabilities, and long-term lease liability in the Company's condensed consolidated balance sheets. ROU assets represent the right to use an underlying asset for the lease term and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Operating lease ROU assets and liabilities are recognized at the lease commencement date based on the present value of the lease payments over the lease term. The Company determines the portion of the lease liability that is current as the difference between the calculated lease liability at the end of the current period and the lease liability that is projected 12 months from the current period.
Property and Equipment	Property and Equipment Property and equipment are stated at cost and depreciated over their estimated useful lives of three years using the straight-line method. Repair and maintenance costs are expensed. The Company reviews the recoverability of all long-lived assets, including the related useful life, whenever events or changes in circumstances indicate that the carrying amount of a long-lived asset might not be recoverable.
Research and Development Expenses	Research and Development ExpensesThe Company expenses the cost of research and development as incurred. Research and development expenses are comprised of costs incurred in performing research and development activities, including clinical trial costs, manufacturing costs for both clinical and preclinical materials as well as contracted services, license fees, and other external costs. Research and development expenses also include the cost of licensing agreements acquired from third parties. Nonrefundable advance payments for goods and services that will be used in future research and development activities are expensed when the activity is performed or when the goods have been received in accordance with ASC 730, Research and Development
Stock-based Compensation	Stock-Based Compensation The Company accounts for its stock-based compensation in accordance with ASC 718, Compensation—Stock Compensation, which establishes accounting for stock-based awards granted to employees for services and requires companies to expense the estimated fair value of these awards over the requisite service period. The Company estimates the fair value of all awards granted using the Black-Scholes valuation model. Key inputs and assumptions include the expected term of the option, stock price volatility, risk-free interest rate, dividend yield, stock price and exercise price. Many of the assumptions require significant judgment and any changes could have an impact in the determination of stock-based compensation expense. The Company elected an accounting policy to record forfeitures as they occur. The Company recognizes employee stock-based compensation expense based on the fair value of the award on the date of the grant. The compensation expense is recognized over the vesting period under the straight-line method. The Company accounts for option awards granted to nonemployee consultants and directors in accordance with ASC 718. The fair value of the option issued or committed to be issued is used to measure the transaction, as this is more reliable than the fair value of the services received. The fair value is measured at the value of the Company’s common stock at the earlier of the date that the commitment for performance by the counterparty has been reached or the counterparty’s performance is complete.
Royalty Monetization Liability	Royalty Monetization Liability The Company accounted for its sale to Ligand Pharmaceuticals Incorporated (“Ligand”) under the purchase and sale agreement (the “Ligand Agreement”) of a 13% share of royalties and milestones owed to the Company pursuant to the RLT Agreement with Takeda related to the potential approval and commercialization of soticlestat in accordance with ASC 470, Debt, which addresses situations in which an entity receives cash from an investor in return for an agreement to pay the investor a specified percentage of the revenue from a contractual right. The Company classified the proceeds received from the sale to Ligand as debt as the Company determined that it had significant continuing involvement in the generation of the cash flows to Ligand. The Company further elected to account for the debt at fair value in accordance with ASC 825, Financial Instruments, which permits a company to elect the fair value option on an instrument specific basis for a recognized financial liability that is not specifically excluded. If commercialized, the Company will recognize 100% of the royalties and milestones received for sales of soticlestat as revenue and the 13% share of royalties payable to Ligand as a cash outflow from financing activities in the condensed consolidated statements of cash flows. Changes in the fair value of the debt will be classified as a component of other income (expense), net in the condensed consolidated statements of operations. In June 2024, Takeda reported Phase 3 topline study results for soticlestat, noting that soticlestat narrowly missed its primary endpoint and showed clinically meaningful and significant effects in multiple key secondary efficacy endpoints with respect to Dravet syndrome and missed its primary endpoint with respect to Lennox-Gastaut syndrome. Based on the study results, the Company’s management reassessed certain assumptions for soticlestat that factor into the valuation of the royalty monetization liability and determined the probability of potential commercialization related to Dravet syndrome to be 7.5% and the probability of potential commercialization related to Lennox-Gastaut syndrome to be 0%. A discount rate of 15% was utilized in calculating the change to the royalty monetization liability. The impact on the fair value resulted in a $29.0 million reduction in the royalty monetization liability which was recognized as other income (expense), net in the statement of operations for the period ended June 30, 2024. Given that the two Phase 3 Takeda trials evaluating soticlestat for the treatment of Dravet and Lennox-Gastaut syndromes did not meet their primary endpoints, it is uncertain whether Takeda will continue to progress, or elect to terminate the development of soticlestat as contemplated by the RLT Agreement, in which case we may not receive some or all of the royalty and milestone payments under the RLT Agreement. See Note 11 – Collaboration and License Agreements for a description of the RLT Agreement.
Income Taxes	Income Taxes The Company accounts for income taxes under the asset and liability method, which requires deferred tax assets and liabilities to be recognized for the estimated future tax consequences attributable to differences between financial statement carrying amounts and respective tax bases of existing assets and liabilities, as well as for net operating loss carryforwards and research and development credits. Valuation allowances are provided if it is more likely than not that some portion or all of the deferred tax assets will not be realized. The impact of a change in the tax laws is recorded in the period in which the law is enacted.
Net Loss Per Share	Net Loss per Share Net loss per common share is determined by dividing net loss attributable to common stockholders by the basic and diluted weighted-average common shares outstanding during the period. The Company applies the two-class method to allocate earnings between common stock and participating securities. When applicable, net income per diluted share attributable to common stockholders adjusts the basic earnings per share attributable to common stockholders and the weighted-average number of shares of common stock outstanding for the potential dilutive impact of stock options using the treasury-stock method and the potential impact of preferred stock using the if-converted method.
Revenue Recognition	Revenue Recognition Under ASC 606, Revenue from Contracts with Customers, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration that the entity expects to receive in exchange for those goods or services. In applying ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the promises and 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) it satisfies the performance obligations. The Company only applies the five-step model to contracts when it is probable that it will collect the consideration to which it is entitled in exchange for the goods or services the Company transfers to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract, determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. Prior to recognizing revenue, the Company makes estimates of the transaction price, including variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. If there are multiple distinct performance obligations, the Company allocates the transaction price to each distinct performance obligation based on its relative standalone selling price. The standalone selling price is generally determined using expected cost and comparable transactions. Revenue for performance obligations recognized over time is recognized by measuring the progress toward complete satisfaction of the performance obligations using an input measure. Non-refundable upfront fees allocated to licenses that are not contingent on any future performance and require no consequential continuing involvement by the Company, are recognized as revenue when the license term commences and the licensed data, technology or product is delivered. The Company defers recognition of upfront license fees if the performance obligations are not satisfied.
Recent Accounting Pronouncements	Recent Accounting Pronouncements The Company has reviewed recently issued accounting standards and plans to adopt those that are applicable. The Company will adopt ASU 2023-7, Segment Reporting (Topic 280): "Improvement to Reportable Segment Disclosures" ("ASU 2023-07"), beginning with its fiscal year ended December 31, 2024. ASU 2023-07 introduces a new requirement to disclose significant segment expenses regularly provided to the chief operating decision maker (“CODM”), extends certain annual disclosures to interim periods, clarifies that single reportable segment entities must apply ASC 280 in its entirety, permits more than one measure of segment profit or loss to be reported under certain conditions, and requires disclosure of the title and position of the CODM. The Company does not expect the adoption of these standards to have a material impact on its financial position, results of operations, or cash flows. The Company adopts new pronouncements relating to generally accepted accounting principles applicable to the Company as they are issued, which may be in advance of their effective date. Management does not believe that any recently issued, but not yet effective accounting standards, if currently adopted, would have a material effect on the accompanying financial statements.

SUMMARY OF SIGNIFICANT ACCOUN_3

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables)	6 Months Ended
	Jun. 30, 2024
Accounting Policies [Abstract]
Schedule of Fair Value on a Recurring	The following table presents information about liabilities measured at fair value on a recurring basis and for which the Company utilizes Level 3 inputs to determine fair value. Royalty Monetization Liability (in thousands) June 30, 2024 Balance, beginning of period $ 30,000 Change in fair value of royalty monetization liability (29,028) Balance, end of period $ 972

CASH, CASH EQUIVALENTS AND MA_2

CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES (Tables)	6 Months Ended
	Jun. 30, 2024
Cash and Cash Equivalents [Abstract]
Schedule of Debt Securities, Available-for-Sale	The following tables summarize the fair value of cash, cash equivalents and marketable securities as well as gross unrealized holding gains and losses as of June 30, 2024 and December 31, 2023: June 30, 2024 (in thousands) Amortized Cost Gross Unrealized Holding Gains Gross Unrealized Holding Losses Fair Value Cash $ 1,037 $ — $ — $ 1,037 Cash equivalents 28,659 — (2) 28,657 Marketable securities 47,292 — (12) 47,280 Total cash, cash equivalents and marketable securities $ 76,988 $ — $ (14) $ 76,974 December 31, 2023 (in thousands) Amortized Cost Gross Unrealized Holding Gains Gross Unrealized Holding Losses Fair Value Cash $ 2,701 $ — $ — $ 2,701 Cash equivalents 24,340 — — 24,340 Marketable securities 78,791 1 — 78,792 Total cash, cash equivalents and marketable securities $ 105,832 $ 1 $ — $ 105,833
Schedule of Cash and Cash Equivalents	The following tables summarize the fair value of cash, cash equivalents and marketable securities as well as gross unrealized holding gains and losses as of June 30, 2024 and December 31, 2023: June 30, 2024 (in thousands) Amortized Cost Gross Unrealized Holding Gains Gross Unrealized Holding Losses Fair Value Cash $ 1,037 $ — $ — $ 1,037 Cash equivalents 28,659 — (2) 28,657 Marketable securities 47,292 — (12) 47,280 Total cash, cash equivalents and marketable securities $ 76,988 $ — $ (14) $ 76,974 December 31, 2023 (in thousands) Amortized Cost Gross Unrealized Holding Gains Gross Unrealized Holding Losses Fair Value Cash $ 2,701 $ — $ — $ 2,701 Cash equivalents 24,340 — — 24,340 Marketable securities 78,791 1 — 78,792 Total cash, cash equivalents and marketable securities $ 105,832 $ 1 $ — $ 105,833

PROPERTY AND EQUIPMENT AND IN_2

PROPERTY AND EQUIPMENT AND INTANGIBLE ASSETS (Tables)	6 Months Ended
	Jun. 30, 2024
Property, Plant and Equipment [Abstract]
Schedule of Property and Equipment	Property and equipment is summarized as follows: (in thousands) June 30, December 31, Furniture and equipment $ 1,517 $ 1,463 Leasehold improvements 306 306 Less accumulated depreciation (1,205) (1,001) Total property and equipment, net $ 618 $ 769

LEASES (Tables)

LEASES (Tables)	6 Months Ended
	Jun. 30, 2024
Leases [Abstract]
Schedule of ROU Asset and Lease Liabilities Related to the Company Operating Lease	ROU asset and lease liabilities related to the Company’s operating lease are as follows: (in thousands) June 30, ROU asset, net $ 13,357 Current lease liability 1,291 Long-term lease liability $ 14,099
Schedule of Components of Operating Lease Cost	The components of operating lease cost for the six months ended June 30, 2024 were as follows: (in thousands) June 30, Operating lease cost $ 1,084 Variable lease cost — Short-term lease cost —
Schedule of Future Minimum Commitments Under the Non-Cancelable Operating Lease	Future minimum commitments under the non-cancelable operating lease are as follows: (in thousands) 2024 1,158 2025 2,316 2026 2,316 2027 2,316 2028 2,469 Thereafter 9,878 Total lease payments $ 20,453

ACCRUED EXPENSES (Tables)

ACCRUED EXPENSES (Tables)	6 Months Ended
	Jun. 30, 2024
Payables and Accruals [Abstract]
Schedule of Accrued Expenses	Accrued expenses consist of the following: (in thousands) June 30, December 31, Payroll and bonus accrual $ 4,947 $ 4,277 Research and development accrual 2,170 1,396 Professional fees accrual 622 522 Other 369 331 Total $ 8,108 $ 6,525

STOCK-BASED COMPENSATION (Table

STOCK-BASED COMPENSATION (Tables)	6 Months Ended
	Jun. 30, 2024
Share-Based Payment Arrangement [Abstract]
Schedule of Recognized Stock-based Compensation Expense	The Company’s stock-based compensation expense was recognized in operating expenses as follows: Three Months Ended Six Months Ended (in thousands) June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 Research and development $ 537 $ 580 $ 1,066 $ 1,090 General and administrative 1,203 1,369 2,642 2,775 Total $ 1,740 $ 1,948 $ 3,708 $ 3,865
Schedule of Allocation of Stock-based Compensation Expense by Plan	Three Months Ended Six Months Ended (in thousands) June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 Stock options and restricted stock units $ 1,722 $ 1,935 $ 3,675 $ 3,837 Employee Stock Purchase Plan 18 13 33 29 Total $ 1,740 $ 1,948 $ 3,708 $ 3,865
Schedule of Assumptions Used to Compute Fair Value of Employee Option Granted	The fair value of employee options granted during the three and six months ended June 30, 2024 and 2023 was estimated utilizing the following assumptions: Three Months Ended Six Months Ended June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 Weighted Weighted Weighted Weighted Volatility 79.01 % 84.11 % 79.82 % 84.56 % Expected term in years 6.08 6.08 6.05 6.07 Dividend rate 0.00 % 0.00 % 0.00 % 0.00 % Risk-free interest rate 4.22 % 3.63 % 4.32 % 3.97 % Fair value of option on grant date $ 1.75 $ 2.66 $ 2.60 $ 1.92 The fair value of nonemployee options granted during the three and six months ended June 30, 2024 and 2023 was estimated utilizing the following assumptions: Three Months Ended Six Months Ended June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 Weighted Weighted Weighted Weighted Volatility 80.59 % 83.00 % 80.59 % 83.00 % Expected term in years 6.50 5.25 6.50 5.25 Dividend rate 0.00 % 0.00 % 0.00 % 0.00 % Risk-free interest rate 4.35 % 3.89 % 4.35 % 3.89 % Fair value of option on grant date $ 2.71 $ 2.42 $ 2.71 $ 2.42
Schedule of Options Outstanding and Weighted Average Exercise Price	The following table summarizes the number of options outstanding and the weighted average exercise price: Number of Shares Weighted Weighted Aggregate Options outstanding December 31, 2023 15,124,546 $ 3.87 6.90 $ 5,212,286 Granted 2,752,150 3.66 6.96 Exercised (248,024) 2.05 Forfeited or expired (796,360) 6.48 Options outstanding June 30, 2024 16,832,312 $ 3.74 6.96 $ 12,790 Vested and exercisable at June 30, 2024 10,755,798 $ 4.06 5.92 $ 12,790

NET INCOME (LOSS) PER SHARE (Ta

NET INCOME (LOSS) PER SHARE (Tables)	6 Months Ended
	Jun. 30, 2024
Earnings Per Share [Abstract]
Schedule of Basic and Diluted Net Income (Loss) Per Share	The following table summarizes the calculation of basic and diluted net income (loss) per share: For the Three Months Ended June 30, For the Six Months Ended June 30, (in thousands) 2024 2023 2024 2023 Net income (loss) $ 8,521 $ (12,408) $ (3,174) $ (25,765) Net income attributable to participating securities 150 — — — Net income (loss) attributable to common stockholders $ 8,371 $ (12,408) $ (3,174) $ (25,765) For the Three Months Ended June 30, For the Six Months Ended June 30, (in thousands, except share and per share data) 2024 2023 2024 2023 Net income (loss) attributable to common stockholders $ 8,371 $ (12,408) $ (3,174) $ (25,765) Weighted average common shares outstanding used in computing net income (loss) per share - basic 70,916,471 70,534,181 70,816,585 70,512,479 Weighted average common shares outstanding used in computing net income (loss) per share - diluted 71,200,798 70,534,181 70,816,585 70,512,479 Net income (loss) per share, basic $ 0.12 $ (0.18) $ (0.04) $ (0.37) Net income (loss) per share, diluted $ 0.12 $ (0.18) $ (0.04) $ (0.37)
Schedule of Potentially Dilutive Securities Excluded from Computations of Diluted Weighted Average Shares Outstanding	The following potentially dilutive securities have been excluded from the computations of diluted weighted-average shares outstanding as they would be anti-dilutive: For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 Stock options to purchase common stock 16,547,985 15,448,835 16,547,985 15,448,835 Common stock issuable upon conversion of Series A convertible preferred stock 1,250,000 1,250,000 1,250,000 1,250,000

NATURE OF OPERATIONS (Details)

NATURE OF OPERATIONS (Details) - USD ($) $ in Thousands	3 Months Ended				6 Months Ended		123 Months Ended
	Jun. 30, 2024	Mar. 31, 2024	Jun. 30, 2023	Mar. 31, 2023	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Dec. 31, 2023
Product Information [Line Items]
Cash, cash equivalents, and marketable securities	$ 76,974				$ 76,974		$ 76,974	$ 105,833
Total revenue	169		$ 75		317	$ 141	223,200
Accumulated deficit	281,039				281,039		281,039	$ 277,866
Working capital	66,800				66,800		$ 66,800
Cash outflows from operating activities					(30,941)	(23,818)
Net income (loss)	8,521	$ (11,694)	$ (12,408)	$ (13,356)	(3,174)	$ (25,765)
Royalty Monetization liability
Product Information [Line Items]
Net income (loss)	$ 8,500				$ (3,200)

SUMMARY OF SIGNIFICANT ACCOUN_4

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Additional Information (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023	Dec. 31, 2023
Summary of Significant Accounting Policy [Line Items]
Long-term equity investments	$ 21,052		$ 21,052		$ 17,626
Initial investment			0	$ 10,000
Marketable securities, Fair Value	47,280		47,280		78,792
Royalty monetization liability	$ 972		$ 972		30,000
Estimated useful life (in years)	3 years		3 years
Other income (expense), net	$ 29,038	$ 1,764	$ 34,760	$ 3,300
Measurement Input, Discount Rate
Summary of Significant Accounting Policy [Line Items]
Measurement input	15%		15%
Level 2
Summary of Significant Accounting Policy [Line Items]
Marketable securities, Fair Value	$ 57,200		$ 57,200		78,800
Level 3
Summary of Significant Accounting Policy [Line Items]
Royalty monetization liability	972		972		30,000
Money Market Funds and Short-term Investments | Level 1
Summary of Significant Accounting Policy [Line Items]
Fair value assets	18,900		18,900		25,700
Gensaic, Inc
Summary of Significant Accounting Policy [Line Items]
Long-term equity investments	5,100		5,100		5,100
Graviton Bioscience Corporation
Summary of Significant Accounting Policy [Line Items]
Long-term equity investments	15,800		15,800		11,200
Initial investment			10,000
Cumulative measurement adjustment of long term investment			5,800
Marinus Therapeutics, Inc
Summary of Significant Accounting Policy [Line Items]
Long-term equity investments	$ 100		100		$ 1,300
Ligand Pharmaceuticals Incorporated
Summary of Significant Accounting Policy [Line Items]
Other income (expense), net			$ 29,000
Ligand Pharmaceuticals Incorporated | Purchase Agreement
Summary of Significant Accounting Policy [Line Items]
Commercial interest rate	13%		13%
Ownership interest rate			100%
Dravet | Purchase Agreement
Summary of Significant Accounting Policy [Line Items]
Commercial interest rate	7.50%		7.50%
Lennox-Gastaut | Purchase Agreement
Summary of Significant Accounting Policy [Line Items]
Commercial interest rate	0%		0%

SUMMARY OF SIGNIFICANT ACCOUN_5

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Schedule of Fair Value on a Recurring (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023
Royalty Monetization Liability
Balance, beginning of period	$ 30,000
Change in fair value of royalty monetization liability	(29,028)	$ 0
Balance, end of period	$ 972

CASH, CASH EQUIVALENTS AND MA_3

CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES - Summary of Fair Value of Cash and Cash Equivalents and Gross Unrealized Holding Gains and Losses (Details) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Cash and Cash Equivalents [Abstract]
Cash Amortized Cost	$ 1,037	$ 2,701
Cash equivalents, Amortized Cost	28,659	24,340
Cash equivalents, Gross Unrealized Holding Gains	0	0
Cash equivalents, Gross Unrealized Holding Losses	(2)	0
Cash equivalents, Fair Value	28,657	24,340
Marketable securities, Amortized cost	47,292	78,791
Gross Unrealized Holding Gains	0	1
Gross Unrealized Holding Losses	(12)	0
Marketable securities, Fair Value	47,280	78,792
Total cash, cash equivalents and marketable securities, Amortized Cost	76,988	105,832
Total cash, cash equivalents and marketable securities, Gross Unrealized Holding Gains	0	1
Total cash, cash equivalents and marketable securities, Gross Unrealized Holding Losses	(14)	0
Total cash, cash equivalents and marketable securities, Fair Value	$ 76,974	$ 105,833

CASH, CASH EQUIVALENTS AND MA_4

CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES - Additional Information (Details)	6 Months Ended
	Jun. 30, 2024 USD ($) Investment	Jun. 30, 2023 USD ($)	Dec. 31, 2023 Investment
Cash and Cash Equivalents [Abstract]
Number of securities in an unrealized loss position for more than 12 months | Investment	0		0
Gains or losses on available-for-sale securities | $	$ 0	$ 0

PROPERTY AND EQUIPMENT AND IN_3

PROPERTY AND EQUIPMENT AND INTANGIBLE ASSETS - Schedule of Property and Equipment (Details) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Property, Plant and Equipment [Line Items]
Less accumulated depreciation	$ (1,205)	$ (1,001)
Property and equipment, net	618	769
Furniture and equipment
Property, Plant and Equipment [Line Items]
Property and equipment, gross	1,517	1,463
Leasehold improvements
Property, Plant and Equipment [Line Items]
Property and equipment, gross	$ 306	$ 306

PROPERTY AND EQUIPMENT AND IN_4

PROPERTY AND EQUIPMENT AND INTANGIBLE ASSETS - Additional Information (Details) - USD ($)	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023	Dec. 31, 2023
Property, Plant and Equipment [Abstract]
Depreciation expense	$ 103,000	$ 105,000	$ 204,000	$ 214,000
Intangible assets, net of accumulated amortization	219,000		219,000		$ 186,000
Amortization expense	$ 45,000	$ 32,000	$ 80,000	$ 63,000

LEASES - Additional Information

LEASES - Additional Information (Details) ft² in Thousands, $ in Thousands	Mar. 10, 2022 ft²	Jun. 30, 2024 USD ($)	Dec. 31, 2023 USD ($)
Short-Term Debt [Line Items]
Operating lease, term of contract	10 years
Land subject to ground leases (sqft) | ft²	19
Rent lease		$ 2,300
Rent payments commencement following this period	10 months
Restricted cash		$ 1,931	$ 1,931
Remaining lease terms		9 years
Renewal option term		5 years
Lease incremental borrowing rate		7.02%
Letter of Credit
Short-Term Debt [Line Items]
Restricted cash		$ 1,900

LEASES - Schedule of ROU Asset

LEASES - Schedule of ROU Asset and Lease Liabilities Related to the Company Operating Lease (Details) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Leases [Abstract]
ROU asset, net	$ 13,357	$ 13,894
Current lease liability	1,291	1,246
Long-term lease liability	$ 14,099	$ 14,756

LEASES - Schedule of Components

LEASES - Schedule of Components of Operating Lease Cost (Details) $ in Thousands	6 Months Ended
	Jun. 30, 2024 USD ($)
Leases [Abstract]
Operating lease cost	$ 1,084
Variable lease cost	0
Short-term lease cost	$ 0

LEASES - Schedule of Future Min

LEASES - Schedule of Future Minimum Commitments Under the Non-Cancelable Operating Lease (Details) $ in Thousands	Jun. 30, 2024 USD ($)
Leases [Abstract]
2024	$ 1,158
2025	2,316
2026	2,316
2027	2,316
2028	2,469
Thereafter	9,878
Total lease payments	$ 20,453

ACCRUED EXPENSES - Schedule of

ACCRUED EXPENSES - Schedule of Accrued Expenses (Details) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Payables and Accruals [Abstract]
Payroll and bonus accrual	$ 4,947	$ 4,277
Research and development accrual	2,170	1,396
Professional fees accrual	622	522
Other	369	331
Total	$ 8,108	$ 6,525

STOCKHOLDERS_ EQUITY (Details)

STOCKHOLDERS’ EQUITY (Details)	6 Months Ended
	Jun. 30, 2024 USD ($) vote $ / shares shares	Dec. 31, 2023 shares
Class of Stock [Line Items]
Common stock, shares authorized (in shares)	125,000,000	125,000,000
Preferred stock, shares authorized (in shares)	10,000,000	10,000,000
Preferred stock, share designated (in shares)	10,000	10,000
Number of votes per share | vote	1
Preferred stock, shares outstanding (in shares)	1,250	1,250
Dividends declared | $	$ 0
Series A Convertible Preferred Stock
Class of Stock [Line Items]
Preferred stock, share designated (in shares)	1,250
Preferred stock, shares outstanding (in shares)	1,250	1,250
Number of shares issued for each share of convertible preferred stock that is converted (in shares)	1,000
Maximum allowable owning percentage of outstanding common stock by associates or affiliates	9.99%
Maximum allowable voting right percentage of outstanding common stock holders	14.99%
Allowable voting right percentage of outstanding common stock holders	19.99%
Allowable voting right percentage of outstanding common stock holders upon notice of days	61 days
Preferred stock, liquidation preference per share (in usd per share) | $ / shares	$ 0.001
Series A Convertible Preferred Stock | Minimum
Class of Stock [Line Items]
Beneficial ownership limitations, percentage of issued and outstanding common stock	10%
Series A Convertible Preferred Stock | Maximum
Class of Stock [Line Items]
Beneficial ownership limitations, percentage of issued and outstanding common stock	15%

STOCK-BASED COMPENSATION - Addi

STOCK-BASED COMPENSATION - Additional Information (Details) - USD ($)			3 Months Ended		6 Months Ended		12 Months Ended
	Jan. 01, 2024	Jan. 01, 2023	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023	Dec. 31, 2023	May 04, 2017
Stock-Based Compensation [Line Items]
Share based compensation expense			$ 1,740,000	$ 1,948,000	$ 3,708,000	$ 3,865,000
Options outstanding (in shares)			16,832,312		16,832,312		15,124,546
Granted (in shares)					2,752,150
Unrecognized compensation expenses			$ 13,900,000		$ 13,900,000
Unrecognized compensation not yet recognized, period for recognition					2 years 6 months 3 days
Stock options and restricted stock units
Stock-Based Compensation [Line Items]
Share based compensation expense			1,722,000	1,935,000	$ 3,675,000	3,837,000
Stock options and restricted stock units | Nonemployee Option
Stock-Based Compensation [Line Items]
Share based compensation expense			$ 98,000	$ 163,000	$ 157,000	$ 272,000
Granted (in shares)			0	50,000	250,000	50,000
Unvested stock options, outstanding (in shares)			268,959	130,834	268,959	130,834
Unrecognized compensation expenses			$ 100,000		$ 100,000
Stock options and restricted stock units | Employee Option
Stock-Based Compensation [Line Items]
Share based compensation expense			$ 1,600,000	$ 1,800,000	$ 3,500,000	$ 3,600,000
Granted (in shares)			80,000	270,000	2,752,150	2,950,500
Unvested stock options, outstanding (in shares)			5,807,555	6,710,485	5,807,555	6,710,485
Unrecognized compensation expenses			$ 13,900,000		$ 13,900,000
Performance-based Option Awards | Employee Option
Stock-Based Compensation [Line Items]
Share based compensation expense			0	$ 0
Non-employee Performance Based Option Awards | Nonemployee Option
Stock-Based Compensation [Line Items]
Share based compensation expense			$ 0	0
Restricted Stock Units | Employee Option
Stock-Based Compensation [Line Items]
Share based compensation, graded vesting period					3 years
Granted (in shares)					348,575
Restricted Stock Units | Employee Option | Share-Based Payment Arrangement, Tranche One
Stock-Based Compensation [Line Items]
Award vesting percentage					33%
Restricted Stock Units | Employee Option | Share-Based Payment Arrangement, Tranche Two
Stock-Based Compensation [Line Items]
Award vesting percentage					33%
Restricted Stock Units | Employee Option | Share-Based Payment Arrangement, Tranche Three
Stock-Based Compensation [Line Items]
Award vesting percentage					33%
2017 Equity Incentive Plan
Stock-Based Compensation [Line Items]
Number of company's common stock reserved for issuance under the plan (in shares)			5,644,244		5,644,244			3,052,059
Percentage of number of shares of common stock outstanding (percent)					5%
Number of additional shares reserved for issuance under the plan (in shares)	3,534,599	3,523,344
Share based compensation, term of plan					10 years
Share based compensation, graded vesting period					4 years
Share based compensation, exercisable period					90 days
2017 Equity Incentive Plan | Maximum
Stock-Based Compensation [Line Items]
Share based compensation, exercisable period					12 months
2017 ESPP
Stock-Based Compensation [Line Items]
Common stock, reserved for future issuance (in shares)								279,069
Share based compensation, percentage of discount from market price on purchase date			15%
Percentage increase, outstanding stock maximum							1%
2017 ESPP | Stock options and restricted stock units | Nonemployee Option
Stock-Based Compensation [Line Items]
Number of company's common stock reserved for issuance under the plan (in shares)			321,285		321,285
Number of additional shares reserved for issuance under the plan (in shares)	0	0
Share based compensation expense			$ 18,000	$ 13,000
Common stock, reserved for future issuance increase (in shares)							550,000
2014 Employee Stock Purchase Plan
Stock-Based Compensation [Line Items]
Options outstanding (in shares)			1,356,621		1,356,621

STOCK-BASED COMPENSATION - Sche

STOCK-BASED COMPENSATION - Schedule of Recognized Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Stock-Based Compensation [Line Items]
Stock-based compensation expense, Total	$ 1,740	$ 1,948	$ 3,708	$ 3,865
Research and development
Stock-Based Compensation [Line Items]
Stock-based compensation expense, Total	537	580	1,066	1,090
General and administrative
Stock-Based Compensation [Line Items]
Stock-based compensation expense, Total	$ 1,203	$ 1,369	$ 2,642	$ 2,775

STOCK-BASED COMPENSATION - Sc_2

STOCK-BASED COMPENSATION - Schedule of Allocation of Stock-Based Compensation Expense by Plan (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Stock-Based Compensation [Line Items]
Share based compensation expense	$ 1,740	$ 1,948	$ 3,708	$ 3,865
Stock options and restricted stock units
Stock-Based Compensation [Line Items]
Share based compensation expense	1,722	1,935	3,675	3,837
Employee Stock Purchase Plan
Stock-Based Compensation [Line Items]
Share based compensation expense	$ 18	$ 13	$ 33	$ 29

STOCK-BASED COMPENSATION - Summ

STOCK-BASED COMPENSATION - Summary of Assumptions Used to Compute Fair Value of Options Granted (Details) - Stock options and restricted stock units - $ / shares	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Employee Option
Stock-Based Compensation [Line Items]
Volatility	79.01%	84.11%	79.82%	84.56%
Expected term in years	6 years 29 days	6 years 29 days	6 years 18 days	6 years 25 days
Dividend rate	0%	0%	0%	0%
Risk-free interest rate	4.22%	3.63%	4.32%	3.97%
Fair value of option on grant date (in dollars per share)	$ 1.75	$ 2.66	$ 2.60	$ 1.92
Nonemployee Option
Stock-Based Compensation [Line Items]
Volatility	80.59%	83%	80.59%	83%
Expected term in years	6 years 6 months	5 years 3 months	6 years 6 months	5 years 3 months
Dividend rate	0%	0%	0%	0%
Risk-free interest rate	4.35%	3.89%	4.35%	3.89%
Fair value of option on grant date (in dollars per share)	$ 2.71	$ 2.42	$ 2.71	$ 2.42

STOCK-BASED COMPENSATION - Su_2

STOCK-BASED COMPENSATION - Summary of Options Outstanding and Weighted Average Exercise Price (Details)	6 Months Ended	12 Months Ended
	Jun. 30, 2024 USD ($) $ / shares shares	Dec. 31, 2023 USD ($) $ / shares shares
Number of Shares
Options outstanding, beginning balance (in shares) | shares	15,124,546
Granted (in shares) | shares	2,752,150
Exercised (in shares) | shares	(248,024)
Forfeited or expired (in shares) | shares	(796,360)
Options outstanding, ending balance (in shares) | shares	16,832,312	15,124,546
Vested and exercisable, ending balance (in shares) | shares	10,755,798
Weighted Average Exercise Price
Options outstanding, beginning balance (in dollars per share) | $ / shares	$ 3.87
Granted (in dollars per share) | $ / shares	3.66
Exercised (in dollars per share) | $ / shares	2.05
Forfeited or expired (in dollars per share) | $ / shares	6.48
Options outstanding, ending balance (in dollars per share) | $ / shares	3.74	$ 3.87
Vested and exercisable (in dollars per share) | $ / shares	$ 4.06
Weighted Average Remaining Contractual Life in Years and Aggregate Intrinsic Value
Weighted Average Remaining Contractual Life in Years, Options outstanding	6 years 11 months 15 days	6 years 10 months 24 days
Weighted Average Remaining Contractual Life in Years, Granted	6 years 11 months 15 days
Weighted Average Remaining Contractual Life in Years, Vested and exercisable	5 years 11 months 1 day
Aggregate intrinsic value, options outstanding | $	$ 12,790	$ 5,212,286
Aggregate intrinsic value, vested and exercisable | $	$ 12,790

INCOME TAXES (Details)

INCOME TAXES (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Income Tax Disclosure [Abstract]
(Benefit) provision for income taxes	$ 0	$ 0	$ 0	$ 0

COMMITMENTS AND CONTINGENCIES (

COMMITMENTS AND CONTINGENCIES (Details)	1 Months Ended				6 Months Ended
	May 31, 2023 USD ($)	Aug. 31, 2022 USD ($) product	Dec. 31, 2021 USD ($) shares	Dec. 31, 2016 USD ($) product	Jun. 30, 2024 shares	Dec. 31, 2023 shares
Loss Contingencies [Line Items]
Common stock shares issued (in shares) | shares					70,971,577	70,691,992
Research plan and budget, expected costs	$ 3,500,000	$ 3,000,000
License Agreement
Loss Contingencies [Line Items]
License agreement milestone payments (up to)		$ 452,000,000
Developmental milestone approvals, number of products (at least) | product		3
Advance written notice required to terminate agreement					90 days
Northwestern University | License Agreement
Loss Contingencies [Line Items]
Upfront non-creditable one-time license issuance fee payment				$ 75,000
Annual license maintenance fee payable				20,000
Consideration payable for rights grant				$ 5,300,000
Royalty obligation period				10 years
Minimum number of product covered under license agreement | product				1
AstraZeneca
Loss Contingencies [Line Items]
Upfront cash payment			$ 5,000,000
Common stock shares issued (in shares) | shares			7,300,000
Days immediately preceding the execution date			30 days
License agreement milestone payments (up to)			$ 203,000,000
Lundbeck
Loss Contingencies [Line Items]
First payment due upon completion of first phase			$ 3,000,000

COLLABORATION AND LICENSE AGR_2

COLLABORATION AND LICENSE AGREEMENTS (Details) - USD ($)			1 Months Ended			3 Months Ended		6 Months Ended
	Oct. 31, 2023	Feb. 01, 2023	Apr. 30, 2023	Feb. 28, 2022	Mar. 31, 2021	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023	Dec. 31, 2023	Mar. 31, 2022
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Other income (expense), net						$ 29,038,000	$ 1,764,000	$ 34,760,000	$ 3,300,000
Common stock shares issued (in shares)						70,971,577		70,971,577		70,691,992
Common stock, par value (in dollars per share)						$ 0.001		$ 0.001		$ 0.001
Unrealized gain (loss) on equity securities								$ 3,427,000	848,000
Collaborative Arrangement, Co-promotion | Takeda Pharmaceutical Company Limited
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Upfront payment under royalty and termination agreement					$ 196,000,000
Aggregate milestone payments					$ 660,000,000
Percentage of potential royalties	13%
License agreement milestone payments (up to)	$ 30,000,000
Ownership interest rate	87%
Collaborative Arrangement, Co-promotion | Takeda Pharmaceutical Company Limited | Maximum
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Additional payment on sales percentage (up to)					20%
Collaborative Arrangement, Co-promotion | Takeda Pharmaceutical Company Limited | Related Party
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Related party transaction expenses recognized								$ 0	0
Healx License and Option Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
License and option agreement, period				1 year
Upfront payment				$ 500,000
Development and commercial milestone payments				$ 2,000,000
Granted option extension period		4 months
Percentage of development costs								50%
Total revenue								$ 0	0
Marinus License Agreement | Marinus Pharmaceuticals, Inc
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Common stock shares issued (in shares)											123,255
Common stock, par value (in dollars per share)											$ 0.001
Takeda Pharmaceutical Company Limited | Collaborative Arrangement, Co-promotion
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Agreement ownership share					50%
Dravet | Purchase Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Commercial interest rate						7.50%		7.50%
Lennox-Gastaut | Purchase Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Commercial interest rate						0%		0%
Ligand Pharmaceuticals Incorporated
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Other income (expense), net								$ 29,000,000
Ligand Pharmaceuticals Incorporated | Purchase Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Ownership interest rate								100%
Commercial interest rate						13%		13%
Marinus Pharmaceuticals, Inc | Marinus License Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Unrealized gain (loss) on equity securities								$ 1,200,000	$ (800,000)
Graviton Bioscience Corporation | Series A Convertible Preferred Stock
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Purchase of preferred stock			$ 10,000,000

NET INCOME (LOSS) PER SHARE - S

NET INCOME (LOSS) PER SHARE - Schedule of Basic and Diluted Earnings (Loss) per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended				6 Months Ended
	Jun. 30, 2024	Mar. 31, 2024	Jun. 30, 2023	Mar. 31, 2023	Jun. 30, 2024	Jun. 30, 2023
Earnings Per Share [Abstract]
Net income (loss)	$ 8,521	$ (11,694)	$ (12,408)	$ (13,356)	$ (3,174)	$ (25,765)
Net income attributable to participating securities	150		0		0	0
Net income (loss) attributable to common stockholders	8,371		(12,408)		(3,174)	(25,765)
Net income (loss) attributable to common stockholders	$ 8,371		$ (12,408)		$ (3,174)	$ (25,765)
Weighted average common shares outstanding used in computing net income (loss) per share - basic (in shares)	70,916,471		70,534,181		70,816,585	70,512,479
Weighted average common shares outstanding used in computing net income (loss) per share - diluted (in shares)	71,200,798		70,534,181		70,816,585	70,512,479
Net income (loss) per share, basic (in dollars per share)	$ 0.12		$ (0.18)		$ (0.04)	$ (0.37)
Net income (loss) per share, diluted (in dollars per share)	$ 0.12		$ (0.18)		$ (0.04)	$ (0.37)

NET INCOME (LOSS) PER SHARE -_2

NET INCOME (LOSS) PER SHARE - Schedule of Potentially Dilutive Securities Excluded from Computations of Diluted Weighted Average Shares Outstanding (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Stock options to purchase common stock
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Dilutive securities excluded from computations of diluted weighted average shares outstanding (in shares)	16,547,985	15,448,835	16,547,985	15,448,835
Common stock issuable upon conversion of Series A convertible preferred stock
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Dilutive securities excluded from computations of diluted weighted average shares outstanding (in shares)	1,250,000	1,250,000	1,250,000	1,250,000

ORGANIZATIONAL RESTRUCTURING (D

ORGANIZATIONAL RESTRUCTURING (Details) $ in Millions	1 Months Ended	3 Months Ended
	Jun. 30, 2024 USD ($)	Jun. 30, 2024 USD ($) eliminated
Restructuring Cost and Reserve [Line Items]
Reduction in workforce number of positions eliminated | eliminated		17
Reduction in workforce		43%
Severance costs	$ 3.4
Employee Severance
Restructuring Cost and Reserve [Line Items]
Restructuring reserve	$ 3.4	$ 3.4