0000950170-24-122804 | 10-Q | XBRL Viewer

Accrued Expenses and Other Current Liabilities

9 Months Ended

Sep. 30, 2024

Accrued Liabilities and Other Liabilities [Abstract]

Accrued Expenses and Other Current Liabilities

5. ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

Accrued expenses and other current liabilities consisted of the following:



	September 30,		December 31,
(in thousands)	2024		2023
Accrued pre-clinical and clinical expenses	$	4,386	$	10,142
Short-term insurance financing note		—		310
Deferred revenue		212		667
Accrued professional fees		1,166		1,307
Accrued compensation and benefits		2,013		2,066
Accrued commercial expenses		3,770		19
Accrued patent expenses		1,382		396
Other		560		379
Total accrued expenses & other current liabilities	$	13,489	$	15,286

Revenue	9 Months Ended
Sep. 30, 2024
Revenues [Abstract]
Revenue	14. REVENUE License Agreement with Advanz Pharma On January 3, 2023, the Company entered into an Exclusive License and Supply Agreement (the “Advanz Agreement”) with Mercury Pharma Group Limited (trading as Advanz Pharma Holdings) (“Advanz Pharma”). Pursuant to the Advanz Agreement, the Company granted Advanz Pharma the exclusive right and license to commercialize drug products containing AT-007 (also known as govorestat), our proprietary Aldose Reductase Inhibitor (ARI) (the “Licensed Product”), for use in treatment of Sorbitol Dehydrogenase Deficiency (“SORD”) and Galactosemia (each a “Licensed Indication”) in the European Economic Area, Switzerland and the United Kingdom (the “Territory”). The Agreement provides that the Company will perform research and development services prior to and subsequent to marketing authorization and manufacture and supply product for Advanz Pharma. The Company also granted Advanz Pharma a right of negotiation and “most-favored nation” rights with respect to acquiring the European commercialization rights for any additional indications for which the Licensed Product may be developed in the future (or any other products we may develop solely to the extent used for the Licensed Indications). Advanz Pharma is required to use commercially reasonable efforts to launch and commercialize the Licensed Products in the major markets in the Territory in each Licensed Indication following, and subject to, receipt of marketing authorization therein. Under the Advanz Agreement, Advanz Pharma agreed to pay the Company (i) an upfront payment of EUR 10.0 million (approximately USD $10.7 million), and certain development milestone payments upon clinical trial completion and receipt of marketing authorization in the territory, as well as certain commercial milestone payments, totaling EUR 134 million (approximately USD $142.2 million) in the aggregate, and (ii) royalties of 20% of net sales of the Licensed Product. Such royalty rate will be payable on a country-by-country basis until the later of (i) the expiration of the licensed patents covering the composition of matter of AT-007, or (ii) 10 years after the European Medicines Agency’s grant of marketing authorization for the Licensed Product. The royalties are subject to certain deductions, including certain secondary finishing costs, certain step-in establishment costs and a portion of fees for any potential third-party patent licenses if applicable in the future. Following the initial term of the license, as described above, the royalty rate will be reduced to 10% and shall continue in perpetuity unless the Advanz Agreement is terminated in various circumstances in accordance with its terms. In addition, the Company is entitled to receive cost sharing consideration for post-marketing authorization studies, if applicable. In accordance with the Company's ASC 606 assessment, Advanz Pharma is considered to be a customer. The Company identified three performance obligations, the exclusive license to commercialize the Licensed Product, the obligations to provide research and development for pre and post-marketing authorization and the obligation to manufacture and supply Advanz Pharma with the Product, at cost (a material right). The Company determined that the upfront payment of EUR 10 million (approximately USD $10.7 million) is the estimated transaction price at contract inception. The performance-based milestone payments, sales-based milestone payments, sales-based royalties, and post-marketing authorization study cost sharing are each determined to be variable consideration that are fully constrained due to the uncertainty of achievement. At inception of the Advanz Agreement, the Company determined the estimate of standalone selling price for the license performance obligation by using the adjusted market assessment approach. Under this method, the Company forecasted and analyzed Galactosemia and SORD in the European market, the probability of marketing authorization approval as well as considered recent similar license arrangements within the same phase of clinical development, therapeutic area, type of agreement, forecasted sales for the contract period, probability of success and a market discount rate. To estimate the standalone selling price of the research and development services, the Company forecasted its expected costs of satisfying that performance obligation and added an appropriate margin for that service. To estimate the standalone selling price for the manufacturing supply agreement material right, the Company utilized an adjusted market assessment approach. The Company analyzed the discount Advance Pharma is expected to obtain by estimating the material right the customer is receiving for the future purchase of manufacturing and supply services. This estimation utilized the estimated number of patients to be treated per year in the Territory; the estimated volume of bottles per patient required per year; typical margin; probability of success; and discounted at market rate. The Company allocated the total transaction price to each performance obligation on a relative standalone selling price basis and determined whether revenue should be recognized at a point in time or over time. Revenue should be recognized when, or as, an entity satisfies a performance obligation by transferring a promised good or service to a customer, i.e., when the customer obtains control of the good or service. The license granted to Advanz Pharma is being accounted for as a distinct performance obligation. The Advanz Pharma license relates to functional IP for which revenue is recognized at a point in time – in the case of this license agreement, the point in time is at inception of the contract because the customer obtained control of the license and was able to use and benefit from its right to use the intellectual property at that point. The Company recognized the transaction price allocated to the license obligation of $10.7 million as license revenue on its statements of operations for the nine months ended September 30, 2023. There were no amounts recognized for the three months ended September 30, 2023. As of December 31, 2023, the Company made certain immaterial adjustments, which resulted in the Company allocating $9.3 million to the license performance obligation; $1.3 million to the research and development performance obligation; and $0.1 million to the manufacturing and supply material right. The research and development services performance obligation under the Advanz Agreement represents a separate performance obligation. The research and development services were provided to Advanz Pharma by the Company from inception of the agreement in January 2023 and will continue through completion of post marketing authorization approval studies. Revenue related to the research and development services performance obligation was recognized as services were performed based on the costs incurred through September 30, 2024, as a percentage of the estimated total costs to be incurred for this performance obligation. The Company recognized $0.1 million and $0.5 million of revenue during the three and nine months ended September 30, 2024, respectively. The Company had deferred revenue of $0.1 million and $0.5 million related to the research and development performance obligation at September 30, 2024 and December 31, 2023, respectively. The Company expects to recognize the deferred revenue as of September 30, 2024, over the next six months. Operating expenses for costs incurred pursuant to this arrangement are reported in their respective expense line items in the Statements of Operations, net of any payments due to or reimbursements due from Advanz, with such reimbursements being recognized at the time the party becomes obligated to pay. The manufacturing supply agreement material right performance obligation provided to Advanz Pharma by the Company resulted in an allocation of the transaction price and resulting deferred revenue as of contract inception and September 30, 2024 and December 31, 2023, of approximately $0.1 million. Revenue associated with this performance obligation will be recognized when Advanz Pharma buys supply from the Company after regulatory approval or upon expiration of the material right.

Revenue

9 Months Ended

Sep. 30, 2024

Revenues [Abstract]

Revenue

14. REVENUE

License Agreement with Advanz Pharma

On January 3, 2023, the Company entered into an Exclusive License and Supply Agreement (the “Advanz Agreement”) with Mercury Pharma Group Limited (trading as Advanz Pharma Holdings) (“Advanz Pharma”). Pursuant to the Advanz Agreement, the Company granted Advanz Pharma the exclusive right and license to commercialize drug products containing AT-007 (also known as govorestat), our proprietary Aldose Reductase Inhibitor (ARI) (the “Licensed Product”), for use in treatment of Sorbitol Dehydrogenase Deficiency (“SORD”) and Galactosemia (each a “Licensed Indication”) in the European Economic Area, Switzerland and the United Kingdom (the “Territory”). The Agreement provides that the Company will perform research and development services prior to and subsequent to marketing authorization and manufacture and supply product for Advanz Pharma. The Company also granted Advanz Pharma a right of negotiation and “most-favored nation” rights with respect to acquiring the European commercialization rights for any additional indications for which the Licensed Product may be developed in the future (or any other products we may develop solely to the extent used for the Licensed Indications).

Advanz Pharma is required to use commercially reasonable efforts to launch and commercialize the Licensed Products in the major markets in the Territory in each Licensed Indication following, and subject to, receipt of marketing authorization therein. Under the Advanz Agreement, Advanz Pharma agreed to pay the Company (i) an upfront payment

of EUR 10.0 million (approximately USD $10.7 million), and certain development milestone payments upon clinical trial completion and receipt of marketing authorization in the territory, as well as certain commercial milestone payments, totaling EUR 134 million (approximately USD $142.2 million) in the aggregate, and (ii) royalties of 20% of net sales of the Licensed Product. Such royalty rate will be payable on a country-by-country basis until the later of (i) the expiration of the licensed patents covering the composition of matter of AT-007, or (ii) 10 years after the European Medicines Agency’s grant of marketing authorization for the Licensed Product. The royalties are subject to certain deductions, including certain secondary finishing costs, certain step-in establishment costs and a portion of fees for any potential third-party patent licenses if applicable in the future. Following the initial term of the license, as described above, the royalty rate will be reduced to 10% and shall continue in perpetuity unless the Advanz Agreement is terminated in various circumstances in accordance with its terms. In addition, the Company is entitled to receive cost sharing consideration for post-marketing authorization studies, if applicable.

In accordance with the Company's ASC 606 assessment, Advanz Pharma is considered to be a customer. The Company identified three performance obligations, the exclusive license to commercialize the Licensed Product, the obligations to provide research and development for pre and post-marketing authorization and the obligation to manufacture and supply Advanz Pharma with the Product, at cost (a material right). The Company determined that the upfront payment of EUR 10 million (approximately USD $10.7 million) is the estimated transaction price at contract inception. The performance-based milestone payments, sales-based milestone payments, sales-based royalties, and post-marketing authorization study cost sharing are each determined to be variable consideration that are fully constrained due to the uncertainty of achievement.

At inception of the Advanz Agreement, the Company determined the estimate of standalone selling price for the license performance obligation by using the adjusted market assessment approach. Under this method, the Company forecasted and analyzed Galactosemia and SORD in the European market, the probability of marketing authorization approval as well as considered recent similar license arrangements within the same phase of clinical development, therapeutic area, type of agreement, forecasted sales for the contract period, probability of success and a market discount rate. To estimate the standalone selling price of the research and development services, the Company forecasted its expected costs of satisfying that performance obligation and added an appropriate margin for that service. To estimate the standalone selling price for the manufacturing supply agreement material right, the Company utilized an adjusted market assessment approach. The Company analyzed the discount Advance Pharma is expected to obtain by estimating the material right the customer is receiving for the future purchase of manufacturing and supply services. This estimation utilized the estimated number of patients to be treated per year in the Territory; the estimated volume of bottles per patient required per year; typical margin; probability of success; and discounted at market rate.

The Company allocated the total transaction price to each performance obligation on a relative standalone selling price basis and determined whether revenue should be recognized at a point in time or over time.

Revenue should be recognized when, or as, an entity satisfies a performance obligation by transferring a promised good or service to a customer, i.e., when the customer obtains control of the good or service. The license granted to Advanz Pharma is being accounted for as a distinct performance obligation. The Advanz Pharma license relates to functional IP for which revenue is recognized at a point in time – in the case of this license agreement, the point in time is at inception of the contract because the customer obtained control of the license and was able to use and benefit from its right to use the intellectual property at that point. The Company recognized the transaction price allocated to the license obligation of $10.7 million as license revenue on its statements of operations for the nine months ended September 30, 2023. There were no amounts recognized for the three months ended September 30, 2023.

As of December 31, 2023, the Company made certain immaterial adjustments, which resulted in the Company allocating $9.3 million to the license performance obligation; $1.3 million to the research and development performance obligation; and $0.1 million to the manufacturing and supply material right.

The research and development services performance obligation under the Advanz Agreement represents a separate performance obligation. The research and development services were provided to Advanz Pharma by the Company from inception of the agreement in January 2023 and will continue through completion of post marketing authorization approval studies. Revenue related to the research and development services performance obligation was recognized as services were performed based on the costs incurred through September 30, 2024, as a percentage of the estimated total costs to be incurred for this performance obligation. The Company recognized $0.1

million and $0.5 million of revenue during the three and nine months ended September 30, 2024, respectively. The Company had deferred revenue of $0.1 million and $0.5 million related to the research and development performance obligation at September 30, 2024 and December 31, 2023, respectively. The Company expects to recognize the deferred revenue as of September 30, 2024, over the next six months. Operating expenses for costs incurred pursuant to this arrangement are reported in their respective expense line items in the Statements of Operations, net of any payments due to or reimbursements due from Advanz, with such reimbursements being recognized at the time the party becomes obligated to pay.

The manufacturing supply agreement material right performance obligation provided to Advanz Pharma by the Company resulted in an allocation of the transaction price and resulting deferred revenue as of contract inception and September 30, 2024 and December 31, 2023, of approximately $0.1 million. Revenue associated with this performance obligation will be recognized when Advanz Pharma buys supply from the Company after regulatory approval or upon expiration of the material right.

- Definition

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Stockholders' Equity - March 2024 Private Placement (Details) - USD ($) $ / shares in Units, $ in Millions		3 Months Ended	9 Months Ended
	Mar. 01, 2024	Sep. 30, 2023	Sep. 30, 2024
Common And Pre-Funded Warrants
Equity
Issuance of warrants (in shares)			2,000,000
Pre-Funded Warrants
Equity
Issuance of warrants (in shares)			2,000,000
Common Stock
Equity
Issuance of common stock (in shares)		4,284,344
March 2024 Private Placement
Equity
Proceeds from issuance of shares and warrants, gross	$ 92.3
Exercise price (in dollars per share)	$ 0.001
March 2024 Private Placement \| Pre-Funded Warrants
Equity
Issuance of warrants (in shares)	2,000,000
Purchase price, warrant (in dollars per share)	$ 6.999
Exercise price (in dollars per share)	$ 0.001
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants	9.99%
March 2024 Private Placement \| Common Stock
Equity
Issuance of common stock (in shares)	12,285,714
Purchase price, common stock (in dollars per share)	$ 7

Warrants - Warrants Issued with April 2023 Private Placement - Additional Information (Details) - $ / shares			3 Months Ended			9 Months Ended
	Apr. 26, 2023	Apr. 23, 2023	Sep. 30, 2024	Mar. 31, 2024	Jun. 30, 2023	Sep. 30, 2024	Dec. 31, 2023
Common Stock
Warrants
Exercise of pre-funded warrants for common stock (in shares)			1,385,525	4,206,285	4,250,000
Common And Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)						2,000,000
Warrants outstanding (in shares)			38,915,972			38,915,972	51,533,652
Warrants exercised (in shares)						14,617,680
Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)						2,000,000
Warrants outstanding (in shares)			28,190,972			28,190,972	31,783,652
Warrants exercised (in shares)						5,592,680
Common Warrants
Warrants
Warrants outstanding (in shares)			10,725,000			10,725,000	19,750,000
Warrants exercised (in shares)						9,025,000
April 2023 Offering, Private Placement \| Common Stock
Warrants
Purchase price, common stock (in dollars per share)	$ 0.946	$ 0.946
Exercise of pre-funded warrants for common stock (in shares)						3,091,810
April 2023 Offering, Private Placement \| Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)	22,000,000	22,000,000
Exercise price (in dollars per share)	$ 0.001	$ 0.001
Purchase price, warrant (in dollars per share)	$ 0.945	$ 0.945
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants	9.99%	9.99%
Warrants outstanding (in shares)			18,907,320			18,907,320
Warrants exercised (in shares)						3,092,680
April 2023 Offering, Private Placement \| Pre-Funded Warrants \| Weighted average
Warrants
Exercise price (in dollars per share)			$ 0.001			$ 0.001

Condensed Statements of Stockholders Equity/(Deficit) - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-in Capital	Accumulated Deficit	Accumulated Other Comprehensive Income (Loss)
Balance at Dec. 31, 2022	$ 4,061	$ 5	$ 352,828	$ (348,823)	$ 51
Balance (in shares) at Dec. 31, 2022		48,063,358
Increase (Decrease) in Stockholders' Equity
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares)		50,203
Stock-based compensation expense	2,055		2,055
Net Income (Loss)	(10,137)			(10,137)
Other comprehensive loss	(51)				(51)
Balance at Mar. 31, 2023	(4,072)	$ 5	354,883	(358,960)
Balance (in shares) at Mar. 31, 2023		48,113,561
Balance at Dec. 31, 2022	4,061	$ 5	352,828	(348,823)	$ 51
Balance (in shares) at Dec. 31, 2022		48,063,358
Increase (Decrease) in Stockholders' Equity
Net Income (Loss)	(82,084)
Balance at Sep. 30, 2023	(11,044)	$ 7	419,856	(430,907)
Balance (in shares) at Sep. 30, 2023		77,133,516
Balance at Mar. 31, 2023	(4,072)	$ 5	354,883	(358,960)
Balance (in shares) at Mar. 31, 2023		48,113,561
Increase (Decrease) in Stockholders' Equity
Issuance of common stock and pre-funded warrants, net of issuance costs	27,450	$ 1	27,449
Issuance of common stock and pre-funded warrants, net of issuance costs (in shares)		9,735,731
Exercise of pre-funded warrants (in shares)		4,250,000
Exercise of stock options	22		22
Exercise of stock options (in shares)		20,174
Stock-based compensation expense	1,843		1,843
Net Income (Loss)	(29,577)			(29,577)
Balance at Jun. 30, 2023	(4,334)	$ 6	384,197	(388,537)
Balance (in shares) at Jun. 30, 2023		62,119,466
Increase (Decrease) in Stockholders' Equity
Issuance of common stock, net of issuance costs	7,004		7,004
Issuance of common stock, net of issuance costs (in shares)		4,284,344
Exercise of common warrants	26,755	$ 1	26,754
Exercise of common warrants (in shares)		10,250,000
Exercise of stock options	65		65
Exercise of stock options (in shares)		65,445
Exercise of stock options, not yet issued (in shares)		(3,804)
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares)		418,065
Stock-based compensation expense	1,836		1,836
Net Income (Loss)	(42,370)			(42,370)
Balance at Sep. 30, 2023	(11,044)	$ 7	419,856	(430,907)
Balance (in shares) at Sep. 30, 2023		77,133,516
Balance at Dec. 31, 2023	(17,146)	$ 8	451,432	(468,586)
Balance (in shares) at Dec. 31, 2023		84,869,832
Increase (Decrease) in Stockholders' Equity
Issuance of common stock and pre-funded warrants, net of issuance costs	104,748	$ 2	104,746
Issuance of common stock and pre-funded warrants, net of issuance costs (in shares)		15,285,714
Exercise of common warrants	61,218	$ 1	61,217
Exercise of common warrants (in shares)		9,025,000
Exercise of pre-funded warrants (in shares)		4,206,285
Exercise of stock options	33		33
Exercise of stock options (in shares)		32,290
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares)		822,682
Stock-based compensation expense	2,379		2,379
Net Income (Loss)	(83,938)			(83,938)
Balance at Mar. 31, 2024	67,294	$ 11	619,807	(552,524)
Balance (in shares) at Mar. 31, 2024		114,241,803
Balance at Dec. 31, 2023	$ (17,146)	$ 8	451,432	(468,586)
Balance (in shares) at Dec. 31, 2023		84,869,832
Increase (Decrease) in Stockholders' Equity
Exercise of stock options (in shares)	327,492
Net Income (Loss)	$ (149,631)
Balance at Sep. 30, 2024	5,892	$ 11	624,098	(618,217)
Balance (in shares) at Sep. 30, 2024		116,356,474
Balance at Mar. 31, 2024	67,294	$ 11	619,807	(552,524)
Balance (in shares) at Mar. 31, 2024		114,241,803
Increase (Decrease) in Stockholders' Equity
Exercise of stock options	310		310
Exercise of stock options (in shares)		295,202
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares)		309,266
Stock-based compensation expense	1,890		1,890
Net Income (Loss)	2,898			2,898
Balance at Jun. 30, 2024	72,392	$ 11	622,007	(549,626)
Balance (in shares) at Jun. 30, 2024		114,846,271
Increase (Decrease) in Stockholders' Equity
Exercise of pre-funded warrants (in shares)		1,385,525
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares)		124,678
Stock-based compensation expense	2,091		2,091
Net Income (Loss)	(68,591)			(68,591)
Balance at Sep. 30, 2024	$ 5,892	$ 11	$ 624,098	$ (618,217)
Balance (in shares) at Sep. 30, 2024		116,356,474

Benefit Plans

9 Months Ended

Sep. 30, 2024

Retirement Benefits [Abstract]

Benefit Plans

11. BENEFIT PLANS

The Company established a defined contribution savings plan under Section 401(k) of the Internal Revenue Code in 2018. This plan covers substantially all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre‑tax basis. Matching contributions to the plan may be made at the discretion of the Company’s board of directors. The Company made approximately $0.1 million and $0.3 million, respectively, in matching contributions to the plan during the three and nine months ended September 30, 2024. The Company made approximately $0.1 million and $0.3 million, respectively, in matching contributions to the plan during the three and nine months ended September 30, 2023.

- Definition

The entire disclosure for retirement benefits.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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-Subparagraph (d)(iii)
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-URI https://asc.fasb.org/1943274/2147480506/715-20-50-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (o)
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-URI https://asc.fasb.org/1943274/2147480506/715-20-50-1

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
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Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 715
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-Paragraph 2
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Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 715
-SubTopic 60
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480266/715-60-50-3

+ Details

Warrants - Warrants Issued with Series A Preferred Stock - Additional Information (Details) - 2017 Warrants - Warrants Issued with Series A Preferred Stock $ / shares in Units, $ in Millions	Jan. 26, 2017 USD ($) $ / shares shares
Warrants
Issuance of warrants (in shares) \| shares	309,389
Issuance of warrants (in dollars) \| $	$ 0.2
Exercise price (in dollars per share) \| $ / shares	$ 2.49

License Agreement - Columbia - Additional Information (Details) - USD ($)		1 Months Ended			3 Months Ended		9 Months Ended		96 Months Ended
	Oct. 03, 2019	Mar. 31, 2019	Jan. 31, 2019	Oct. 31, 2016	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Dec. 31, 2023
Agreements
Research and development					$ 14,828,000	$ 10,785,000	$ 37,049,000	$ 38,602,000
General and administrative					15,037,000	4,710,000	34,683,000	15,585,000
Operating expenses					29,865,000	15,495,000	71,732,000	54,187,000
Accrued expenses and other current liabilities					13,489,000		13,489,000		$ 13,489,000	$ 15,286,000
Accounts payable					2,837,000		2,837,000		2,837,000	1,742,000
License Agreements and Research Services Agreements \| Columbia University
Agreements
Operating expenses					100,000	$ 0	300,000	$ 100,000	3,200,000
Accrued expenses and other current liabilities					100,000		100,000		100,000	29,000
Accounts payable					$ 0		$ 0		$ 0	$ 0
2016 license agreement \| Columbia University
Agreements
Percentage of outstanding common stock issued				5.00%
Fair value of stock issued				$ 500,000
Written notice period for termination of agreement				90 days
Written notice period for termination of agreement, uncured material breach				30 days
Extended written notice period for termination of agreement, attempt to cure material breach				90 days
2016 license agreement \| Columbia University \| Minimum
Agreements
Percentage of sublicensing revenue, if sublicenses rights granted				10.00%
2016 license agreement \| Columbia University \| Maximum
Agreements
Payment on achievement of specified development and regulatory milestones				$ 1,300,000
Payment on achievement of specified level of aggregate annual net sales				$ 1,000,000
Percentage of sublicensing revenue, if sublicenses rights granted				20.00%
2019 license agreement \| Columbia University \| Maximum
Agreements
Payment on achievement of specified development and regulatory milestones			$ 1,300,000
Payment on achievement of specified level of aggregate annual net sales			$ 1,000,000
2019 research agreement \| Columbia University
Agreements
Agreement term		12 months
PI3k Research agreement \| Columbia University
Agreements
Agreement term	18 months

Net Loss Per Common Share - Schedule of Computation of Basic and Diluted Net Loss Per Common Share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023
Numerator:
Net loss	$ (68,591)	$ 2,898	$ (83,938)	$ (42,370)	$ (29,577)	$ (10,137)	$ (149,631)	$ (82,084)
Denominator:
Weighted-average common stock outstanding - basic (in shares)	144,345,781			90,669,969			137,893,249	75,482,234
Weighted-average common stock outstanding - diluted (in shares)	144,345,781			90,669,969			137,893,249	75,482,234
Net loss per share attributable to common stockholders - basic	$ (0.48)			$ (0.47)			$ (1.09)	$ (1.09)
Net loss per share attributable to common stockholders - diluted	$ (0.48)			$ (0.47)			$ (1.09)	$ (1.09)

- Definition

The amount of net income (loss) for the period per each share of common stock or unit outstanding during the reporting period.

+ References

+ Details

- Definition

The amount of net income (loss) for the period available to each share of common stock or common unit outstanding during the reporting period and to each share or unit that would have been outstanding assuming the issuance of common shares or units for all dilutive potential common shares or units outstanding during the reporting period.

+ References

+ Details

- Definition

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

+ References

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+ Details

Leases - Summary of Lease Terms and Discount Rates (Details)	Sep. 30, 2024	Sep. 30, 2023
Leases [Abstract]
Weighted-average remaining lease term - operating leases	5 years	1 year 1 month 6 days
Weighted-average discount rate - operating leases	15.45%	5.69%

Warrants

9 Months Ended

Sep. 30, 2024

Warrants and Rights Note Disclosure [Abstract]

Warrants

8. WARRANTS

Warrants Issued with Series A Preferred Stock

On January 26, 2017, in connection with the sale and issuance of the Series A Preferred Stock, the Company issued equity‑classified warrants to purchase 309,389 shares of common stock (the “2017 Warrants”), valued at $0.2 million, and included in the issuance costs of the Series A Preferred Stock. The warrants vested immediately and have an exercise price of $2.49 per share and expire on March 13, 2027.

The fair value of warrants issued was estimated using the Black‑Scholes option pricing model with the following assumptions for the 2017 Warrants.


Contractual term (in years)	10.0
Volatility	74.48	%
Risk-free interest rate	3.20	%
Dividend yield	0.00	%

Warrants Issued with the 2018 Notes

On January 18, 2018, the Company entered into a placement agent agreement through which it became obligated to issue common stock warrants in connection with the issuance of convertible promissory notes, issued on February 5, 2018 (the “2018 Notes”). The obligation to issue the 2018 Notes Warrants was recorded as a liability at its fair value, (see Note 3), which was initially $0.1 million, and was included in the issuance costs of the 2018 Notes. On November 5, 2018, in connection with the extinguishment of the 2018 Notes into shares of Series B Preferred Stock, the Company issued the 2018 Notes Warrants, which were equity‑classified warrants upon issuance, to purchase 76,847 shares of common stock, valued at $0.3 million. The exercise price of the 2018 Notes Warrants resets each time the Company issues common stock with an issue price less than the exercise price of the Warrants. The 2018 Notes Warrants vested immediately upon issuance and expire on November 4, 2028. The exercise price of the 2018 Notes Warrants was $3.98 and $3.99 per share as of September 30, 2024 and December 31, 2023, respectively.

Warrants Issued with Series B Preferred Stock

In November and December 2018, in connection with the sale and issuance of the Series B Preferred Stock, the Company was obligated to issue equity-classified warrants to purchase 72,261 shares of common stock (collectively the “2018 Warrants”), valued in the aggregate at $0.2 million, which was included in the issuance costs for the Series B Preferred Stock. The exercise price of the warrants resets each time the Company issues common stock with an issue price less than the exercise price of the warrants. The warrants vested immediately upon issuance and expire 10 years from the date of issuance. The exercise price of the warrants was $4.84 and $4.86 per share as of September 30, 2024, and December 31, 2023, respectively.

The fair value of the 2018 Warrants was estimated using the Black‑Scholes option pricing model with the following assumptions:


Contractual term (in years)	10.0
Volatility	73.22	%
Risk-free interest rate	2.70	%
Dividend yield	0.00	%

In February 2019, in connection with the sale and issuance of the Series B Preferred Stock, the Company was obligated to issue warrants to purchase 23,867 shares of common stock (collectively the “2019 Warrants”), valued in the aggregate at $0.1 million, which was included in the issuance costs for the Series B Preferred Stock. The exercise price of the warrants resets each time the Company issues common stock with an issue price less than the exercise price of the warrants. The warrants vested immediately upon issuance and expire 10 years from the date of issuance. The exercise price of the warrants was $4.84 and $4.86 per share as of September 30, 2024, and December 31, 2023, respectively.

The fair value of the 2019 Warrants was estimated using the Black-Scholes option pricing model with the following assumptions:


Contractual term (in years)	10.0
Volatility	73.22	%
Risk-free interest rate	2.70	%
Dividend yield	0.00	%

The inputs utilized by management to value the warrants are highly subjective. The assumptions used in calculating the fair value of the warrants represent the Company’s best estimates, but these estimates involve inherent uncertainties and the application of management judgment. As a result, if factors change and the Company uses different assumptions, the fair value of the warrants may be materially different in the future.

Warrants Issued with June 2022 Offering

On June 27, 2022, in connection with the sale and issuance common stock as part of the June Offering, the Company issued 10,000,000 Pre-Funded Warrants at an exercise price of $0.0001 per share, and 30,000,000 accompanying Common Warrants at an exercise price of $1.00 per share. Each share of common stock and accompanying Common Warrant was sold at a public offering price of $1.00, less underwriting discounts and commissions, and each Pre-Funded Warrant and accompanying Common Warrant was sold at a public offering price of $0.9999, less underwriting discounts and commissions, as described in the prospectus supplement, dated June 22, 2022, filed with the Securities and Exchange Commission on June 24, 2022.

The Pre-Funded Warrants and the Common Warrants are immediately exercisable and will expire five years from the date of issuance. Holders may not exercise any Pre-Funded Warrants or Common Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. Holders of the Pre-Funded Warrants and/or Common Warrants (together with affiliates) who immediately prior to June 27, 2022 beneficially owned more than 9.99% of the Company’s outstanding common stock may not exercise any portion of their Pre-Funded Warrants or Common Warrants if the holder (together with affiliates) would beneficially own more than 19.99% of the Company’s outstanding common stock after exercise. The Pre-Funded Warrants and Common Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants and/or Common Warrants will be entitled to receive, upon exercise, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants and/or Common Warrants immediately prior to such transaction. The Pre-Funded Warrants and Common Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which the Company’s stockholders are entitled.

The June 2022 Pre-Funded Warrants were initially recorded at fair value as a liability as the Company could be required to settle the Pre-Funded Warrants in cash in the event of an acquisition of the Company under certain circumstances. In December 2022, the Company amended the Pre-Funded Warrants to remove the potential requirement that they could be settled in cash under certain circumstances. Beginning December 31, 2023, the Pre-funded Warrants are recorded as equity, using their fair value as of the amendment date.

As of September 30, 2024, warrant holders exercised 8,500,000 Pre-Funded Warrants on a cash basis and received 8,500,000 shares of common stock. The Company received $850 in cash proceeds for the exercise of these Pre-Funded Warrants.

As of September 30, 2024, the Company had 1,500,000 Pre-Funded Warrants from the June 2022 offering outstanding with a weighted average exercise price of $0.0001 per share and an average contractual life of 5 years.

The Common Warrants were accounted for as liabilities under ASC 815-40, as these warrants provide for a settlement provision that does not meet the requirements of the indexation guidance under ASC 815-40. These warrant liabilities are measured at fair value at inception and on a recurring basis, with changes in fair value presented within the statement of operations.

As of September 30, 2024, warrant holders exercised 19,275,000 Common Warrants on a cash basis and received 19,275,000 shares of common stock. The Company received $19.3 million in cash proceeds for the exercise of these Common Warrants.

As of September 30, 2024, the Company had 10,725,000 Common Warrants outstanding with a weighted average exercise price of $1.00 per share and an average contractual life of 5 years.

The Common Warrants were remeasured using a Black-Scholes option pricing model with a range of assumptions included below as of September 30, 2024 and December 31, 2023.


	September 30,			December 31,
	2024			2023
Expected term (in years)		2.1			2.4
Volatility		99.92	%		109.46	%
Risk-free interest rate		3.77	%		4.32	%
Dividend yield		0.00	%		0.00	%

Warrants Issued with April 2023 Private Placement

On April 23, 2023, in connection with the sale and issuance of common stock as part of the April 2023 Private Placement, the Company issued 22,000,000 Pre-Funded Warrants at an exercise price of $0.001 per share. Each share of common stock was sold at a public offering price of $0.946, less underwriting discounts and commissions, and each Pre-Funded Warrant was sold at a public offering price of $0.945, less underwriting discounts and commissions, pursuant to a Securities Purchase Agreement, dated as of April 23, 2023, by and between the Company and the Purchasers.

The Pre-Funded Warrants are immediately exercisable from the date of issuance and do not have an expiration date. They have an exercise price of $0.001. Holders may not exercise any Pre-Funded Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. The Pre-Funded Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants will be entitled to receive, upon exercise of the Pre-Funded Warrants, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants immediately prior to such transaction. The Pre-Funded Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which holders of common stock are entitled. The Company intends to use the net proceeds to fund research and development and registration of its pipeline candidates, and for working capital and general corporate purposes. The April 2023 pre-funded warrants were classified as equity.

As of September 30, 2024, warrant holders exercised 3,092,680 Pre-Funded Warrants on a cashless basis and received 3,091,810 shares of common stock.

As of September 30, 2024, the Company had 18,907,320 Pre-Funded Warrants from the April 2023 private placement outstanding with a weighted average exercise price of $0.001 per share. The pre-funded warrants do not have an expiration date.

Warrants Issued with March 2024 Private Placement

On March 1, 2024, in connection with the sale and issuance of common stock as part of the March 2024 Private Placement, the Company issued 2,000,000 Pre-Funded Warrants at an exercise price of $0.001 per share. Each share of common stock was sold at a public offering price of $7.00, less underwriting discounts and commissions, and each Pre-Funded Warrant was sold at a public offering price of $6.999, less underwriting discounts and commissions, pursuant to a Securities Purchase Agreement, dated as of February 27, 2024, by and between the Company and the Purchasers.

The Pre-Funded Warrants are immediately exercisable from the date of issuance and do not have an expiration date. They have an exercise price of $0.001. Holders may not exercise any Pre-Funded Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. The Pre-Funded Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants will be entitled to receive, upon exercise of the Pre-Funded Warrants, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants immediately prior to such transaction. The Pre-Funded Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which holders of common stock are entitled. The March 2024 pre-funded warrants were classified as equity.

As of September 30, 2024, the Company had 2,000,000 Pre-Funded Warrants from the March 2024 private placement outstanding with a weighted average exercise price of $0.001 per share. The pre-funded warrants do not have an expiration date.

A summary of the Company’s outstanding pre-funded and common stock warrants as of September 30, 2024 is as follows:


	Equity Classified			Liability Classified			Total
	Warrants			Warrants			Warrants
Outstanding as of January 1, 2024		31,783,652			19,750,000			51,533,652
Warrants granted and issued		2,000,000			—			2,000,000
Warrants exercised		(5,592,680	)		(9,025,000	)		(14,617,680	)
Outstanding as of September 30, 2024		28,190,972			10,725,000			38,915,972

Prepaid Expenses and Other Current Assets - Summary of Prepaid Expenses and Other Current Assets (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Prepaid Expense and Other Assets, Current [Abstract]
Prepaid research and development expenses	$ 2,885	$ 2,880
Insurance premium asset		566
Prepaid manufacturing costs	327
Prepaid insurance	768
Prepaid commercial and patient advocacy	875	284
Research and development tax credit receivable	262	262
Other prepaid expenses and current assets	366	242
Total prepaid expenses & other current assets	$ 5,483	$ 4,234

Leases - Summary of Maturities of Lease Liabilities (Details) $ in Thousands	Sep. 30, 2024 USD ($)
LEASES
2024	$ 135
2025	546
2026	556
2027	550
2028	563
Thereafter	479
Total lease payments	2,829
Less: interest	858
Total lease liabilities	$ 1,971
Operating Lease, Liability, Statement of Financial Position	Current portion of operating lease liabilities, Noncurrent portion of operating lease liabilities

Prepaid Expenses and Other Current Assets

9 Months Ended

Sep. 30, 2024

Prepaid Expense and Other Assets, Current [Abstract]

Prepaid Expenses and Other Current Assets

4. PREPAID EXPENSES AND OTHER CURRENT ASSETS

Prepaid expenses and other current assets consisted of the following:


	September 30,		December 31,
(in thousands)	2024		2023
Prepaid research and development expenses	$	2,885	$	2,880
Insurance premium asset		—		566
Prepaid manufacturing costs		327		—
Prepaid insurance		768		—
Prepaid commercial and patient advocacy		875		284
Research and development tax credit receivable		262		262
Other prepaid expenses and current assets		366		242
Total prepaid expenses & other current assets	$	5,483	$	4,234

Stockholders' Equity - Venrock Warrant Exchange (Details) - Exchange Agreement $ / shares in Units, $ in Millions	Oct. 12, 2023 USD ($) $ / shares shares
Equity
Fair market value \| $	$ 12.9
Treasury Stock
Equity
Exchange of common stock for prefunded warrants (in shares)	5,658,034
Pre-Funded Warrants
Equity
Number of warrants issued in exchange of common stock (in shares)	5,658,034
Exercise price of warrants (in dollars per share) \| $ / shares	$ 0.001

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (d)
-Publisher FASB
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Reference 2: http://www.xbrl.org/2003/role/recommendedDisclosureRef
-Topic 272
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-Name Accounting Standards Codification
-Section 45
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483014/272-10-45-3

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 272
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Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
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-Section S99
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-Subparagraph (SX 210.4-08(d))
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-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (a)
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-URI https://asc.fasb.org/1943274/2147481112/505-10-50-13

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Paragraph 13
-Subparagraph (b)
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-URI https://asc.fasb.org/1943274/2147481112/505-10-50-13

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Section 50
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
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-Paragraph 13
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-13

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 14
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-14

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 18
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
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-Paragraph 1
-Subparagraph (SX 210.5-02(27)(b))
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 210
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(28))
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-URI https://asc.fasb.org/1943274/2147480566/210-10-S99-1

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-2

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 210
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(29))
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Reference 15: http://www.xbrl.org/2003/role/disclosureRef
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Reference 16: http://www.xbrl.org/2003/role/disclosureRef
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Subparagraph (SX 210.6-03(i)(2)(i))
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Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479886/946-10-S99-3

+ Details

Warrants (Tables)

9 Months Ended

Sep. 30, 2024

Warrants Issued with Series A Preferred Stock | 2017 Warrants

Warrants

Schedule of Fair Value of Warrants Using the Black Scholes Option Pricing Model

The fair value of warrants issued was estimated using the Black‑Scholes option pricing model with the following assumptions for the 2017 Warrants.


Contractual term (in years)	10.0
Volatility	74.48	%
Risk-free interest rate	3.20	%
Dividend yield	0.00	%

Warrants Issued with Series B Preferred Stock | 2018 Warrants

Warrants

Schedule of Fair Value of Warrants Using the Black Scholes Option Pricing Model

The fair value of the 2018 Warrants was estimated using the Black‑Scholes option pricing model with the following assumptions:


Contractual term (in years)	10.0
Volatility	73.22	%
Risk-free interest rate	2.70	%
Dividend yield	0.00	%

Warrants Issued with Series B Preferred Stock | 2019 Warrants

Warrants

Schedule of Fair Value of Warrants Using the Black Scholes Option Pricing Model

The fair value of the 2019 Warrants was estimated using the Black-Scholes option pricing model with the following assumptions:


Contractual term (in years)	10.0
Volatility	73.22	%
Risk-free interest rate	2.70	%
Dividend yield	0.00	%

Common And Pre-Funded Warrants

Warrants

Schedule of Warrants Outstanding

A summary of the Company’s outstanding pre-funded and common stock warrants as of September 30, 2024 is as follows:


	Equity Classified			Liability Classified			Total
	Warrants			Warrants			Warrants
Outstanding as of January 1, 2024		31,783,652			19,750,000			51,533,652
Warrants granted and issued		2,000,000			—			2,000,000
Warrants exercised		(5,592,680	)		(9,025,000	)		(14,617,680	)
Outstanding as of September 30, 2024		28,190,972			10,725,000			38,915,972

Common Warrants

Warrants

Schedule of Fair Value of Warrants Using the Black Scholes Option Pricing Model

The Common Warrants were remeasured using a Black-Scholes option pricing model with a range of assumptions included below as of September 30, 2024 and December 31, 2023.


	September 30,			December 31,
	2024			2023
Expected term (in years)		2.1			2.4
Volatility		99.92	%		109.46	%
Risk-free interest rate		3.77	%		4.32	%
Dividend yield		0.00	%		0.00	%

Prepaid Expenses and Other Current Assets (Tables)

9 Months Ended

Sep. 30, 2024

Prepaid Expense and Other Assets, Current [Abstract]

Summary of Prepaid Expenses and Other Current Assets

Prepaid expenses and other current assets consisted of the following:


	September 30,		December 31,
(in thousands)	2024		2023
Prepaid research and development expenses	$	2,885	$	2,880
Insurance premium asset		—		566
Prepaid manufacturing costs		327		—
Prepaid insurance		768		—
Prepaid commercial and patient advocacy		875		284
Research and development tax credit receivable		262		262
Other prepaid expenses and current assets		366		242
Total prepaid expenses & other current assets	$	5,483	$	4,234

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023
Pay vs Performance Disclosure
Net Income (Loss)	$ (68,591)	$ 2,898	$ (83,938)	$ (42,370)	$ (29,577)	$ (10,137)	$ (149,631)	$ (82,084)

- Definition

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

+ References

+ Details

Stock Based Compensation - Stock-based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Stock-based compensation
Total stock-based compensation expense	$ 2,091	$ 1,836	$ 6,360	$ 5,734
Research and development
Stock-based compensation
Total stock-based compensation expense	951	764	2,907	2,378
General and administrative
Stock-based compensation
Total stock-based compensation expense	$ 1,140	$ 1,072	$ 3,453	$ 3,356

Warrants - Warrants Issued with March 2024 Private Placement - Additional Information (Details) - $ / shares		9 Months Ended
	Mar. 01, 2024	Sep. 30, 2024	Dec. 31, 2023
Common And Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)		2,000,000
Warrants outstanding (in shares)		38,915,972	51,533,652
Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)		2,000,000
Warrants outstanding (in shares)		28,190,972	31,783,652
Common Warrants
Warrants
Warrants outstanding (in shares)		10,725,000	19,750,000
March 2024 Private Placement
Warrants
Exercise price (in dollars per share)	$ 0.001
March 2024 Private Placement \| Common Stock
Warrants
Purchase price, common stock (in dollars per share)	$ 7
March 2024 Private Placement \| Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)	2,000,000
Exercise price (in dollars per share)	$ 0.001
Purchase price, warrant (in dollars per share)	$ 6.999
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants	9.99%
Warrants outstanding (in shares)		2,000,000
March 2024 Private Placement \| Pre-Funded Warrants \| Weighted average
Warrants
Exercise price (in dollars per share)		$ 0.001

License Agreement - Miami University - Additional Information (Details) - USD ($) $ in Thousands			3 Months Ended		9 Months Ended
	Dec. 14, 2020	Oct. 28, 2020	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023
Agreements
Research and development			$ 14,828	$ 10,785	$ 37,049	$ 38,602
General and administrative			15,037	4,710	34,683	15,585
Operating expenses			29,865	15,495	71,732	54,187
Accrued expenses and other current liabilities			13,489		13,489		$ 15,286
2020 license agreement \| University of Miami
Agreements
Amount of first payment to be made for license issue fee		$ 1,100
Amount of second payment to be made for license issue fee, due on first anniversary of the date of license		500
Payment to be made on achievement of specified patenting and development milestones		2,200
Payment to be made on achievement of late stage regulatory milestones		$ 4,100
Agreement term		10 years
Written notice period for termination of agreement		60 days
Written notice period of material breach leading to termination		60 days
Research and development			1,100	200	1,300	200
Accrued expenses and other current liabilities			1,300		1,300		400
2020 license agreement \| University of Miami \| Minimum
Agreements
Royalties payment (as a percent)		0.88%
Non-royalty sublicensing revenue payment (as a percent)		15.00%
2020 license agreement \| University of Miami \| Maximum
Agreements
Royalties payment (as a percent)		5.00%
Non-royalty sublicensing revenue payment (as a percent)		25.00%
2020 research agreement and amendment \| University of Miami
Agreements
Consideration	$ 300
Research and development			0	$ 0	0	$ 0
Accrued expenses and other current liabilities			$ 0		$ 0		$ 100

Accrued Expenses and Other Current Liabilities - Summary of Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Accrued Liabilities and Other Liabilities [Abstract]
Accrued pre-clinical and clinical expenses	$ 4,386	$ 10,142
Short-term insurance financing note		310
Deferred revenue	212	667
Accrued professional fees	1,166	1,307
Accrued compensation and benefits	2,013	2,066
Accrued commercial expenses	3,770	19
Accrued patent expenses	1,382	396
Other	560	379
Total accrued expenses & other current liabilities	$ 13,489	$ 15,286

Accrued Expenses and Other Current Liabilities (Tables)

9 Months Ended

Sep. 30, 2024

Accrued Liabilities and Other Liabilities [Abstract]

Summary of Accrued Expenses and Other Current Liabilities

Accrued expenses and other current liabilities consisted of the following:



	September 30,		December 31,
(in thousands)	2024		2023
Accrued pre-clinical and clinical expenses	$	4,386	$	10,142
Short-term insurance financing note		—		310
Deferred revenue		212		667
Accrued professional fees		1,166		1,307
Accrued compensation and benefits		2,013		2,066
Accrued commercial expenses		3,770		19
Accrued patent expenses		1,382		396
Other		560		379
Total accrued expenses & other current liabilities	$	13,489	$	15,286

License Agreement

9 Months Ended

Sep. 30, 2024

LICENSE AGREEMENT

License Agreement

2. LICENSE AGREEMENT

Columbia University

In October 2016, the Company entered into a license agreement (the “2016 Columbia Agreement”) with the Trustees of Columbia University (“Columbia University”) to obtain an exclusive royalty-bearing sublicensable license in respect to certain patents. As part of the consideration for entering into the 2016 Columbia Agreement, the Company issued to Columbia University shares equal to 5% of its outstanding common stock on a fully diluted basis at the time of issue. The common stock had a fair value of $0.5 million at the time of issuance. The Company will be required to make further payments to Columbia University of up to an aggregate of $1.3 million for the achievement of specified development and regulatory milestones, and up to an aggregate of $1.0 million for the achievement of a specified level of aggregate annual net sales, in each case in connection with products covered by the 2016 Columbia Agreement. The Company will also be required to pay tiered royalties to Columbia University in the low‑ to mid‑single digit percentages on the Company’s, its affiliates’ and its sublicensees’ net sales of licensed products, subject to specified offsets and reductions. In addition, the Company is required to make specified annual minimum royalty payments to Columbia University, which is contingent upon the approval of the licensed products, in the mid-six figures beginning on the 10th anniversary of the effective date of the 2016 Columbia Agreement. When we grant sublicenses under the 2016 Columbia Agreement we are required to pay Columbia University a portion of the net sublicensing revenue received from such third parties, at percentages between 10% and 20%, depending on the stage of development at the time such revenue is received from such third parties. The Advanz Agreement includes a sublicense under the 2016 Columbia Agreement.

The 2016 Columbia Agreement will terminate upon the expiration of all the Company’s royalty payment obligations in all countries. The Company may terminate the 2016 Columbia Agreement for convenience upon 90 days’ written notice to Columbia University. At its election, Columbia University may terminate the 2016 Columbia Agreement, or convert the licenses granted to the Company into non‑exclusive, non‑sublicensable licenses, in the case of (a) the Company’s uncured material breach upon 30 days’ written notice (which shall be extended to 90 days if the Company is diligently attempting to cure such material breach), (b) the Company’s failure to achieve the specified development and funding milestone events, or (c) the Company’s insolvency.

In January 2019, the Company entered into a second license agreement with Columbia University (the “2019 Columbia Agreement”). Pursuant to the 2019 Columbia Agreement, Columbia University granted the Company a royalty-bearing, sublicensable license that is exclusive with respect to certain patents, and non-exclusive with respect to certain know-how, in each case to develop, manufacture and commercialize PI3k inhibitor products. The license grant is worldwide. Under the 2019 Columbia Agreement, the Company is obligated to use commercially reasonable efforts to research, discover, develop and market licensed products for commercial sale in the licensed territory, and to comply with certain obligations to meet specified development and funding milestones within defined time periods. Columbia University retains the right to conduct, and grant third parties the right to conduct, non-clinical academic research using the licensed technology; provided that such research is not funded by a commercial entity or for-profit entity or results in rights granted to a commercial or for-profit entity. As consideration for entering into the 2019 Columbia Agreement, the Company made a nominal upfront payment to Columbia University. The Company will be required to make further payments to Columbia University of up to an aggregate of $1.3 million for the achievement of specified development and regulatory milestones, and up to an aggregate of $1.0 million for the achievement of a specified level of aggregate annual net sales, in each case in connection with products covered by the 2019 Columbia Agreement. The Company will also be required to pay tiered royalties to Columbia University in the low- to mid-single digit percentages on the Company’s, its affiliates’ and its sublicensees’ net sales of licensed products, subject to specified offsets and reductions. In addition, the Company is required to make specified annual minimum royalty payments to Columbia University, which is contingent upon the approval of the licensed products, in the mid-six figures beginning on the tenth anniversary of the effective date of the 2019 Columbia Agreement.

In July 2022, following regulatory changes impacting development of the class of PI3k inhibitors and the Company’s decision to discontinue its early stage preclinical PI3k program, the Company and Columbia entered into an agreement terminating the 2019 Columbia Agreement (the “2022 Columbia Termination Agreement”) as of July 25, 2022. Under the terms of the 2022 Columbia Termination Agreement, the Company assigned certain regulatory documents regarding the preclinical PI3k inhibitor AT-104 to Columbia and granted Columbia a non-exclusive royalty free license (with rights to sublicense any future Columbia licensee) under certain know-how, technical information and data relating to AT-104 that was developed by the Company during the term of the 2019 Columbia Agreement.

In March 2019, and in connection with the 2016 Columbia Agreement, the Company entered into a research services agreement (the “2019 Columbia Research Agreement”) with Columbia University with the purpose of analyzing structural and functional changes in brain tissue in an animal model of Galactosemia, and the effects of certain compounds whose intellectual property rights were licensed to the Company as part of the 2016 Columbia Agreement on any such structural and functional changes. The 2019 Columbia Research Agreement had a term of 12 months from its effective date and expired in accordance with its terms.

On October 3, 2019, and in connection with the 2019 Columbia Agreement, the Company entered into a research services agreement (the “PI3k Columbia Research Agreement” and collectively with the 2016 Columbia Agreement, 2019 Columbia Agreement and 2019 Columbia Research Agreement, the “Columbia Agreements”) with Columbia University with the purpose of analyzing PI3k inhibitors for the treatment of lymphoid malignancies. The PI3k Columbia Research Agreement had a term of 18 months from its effective date and expired in accordance with its terms.

During the three and nine months ended September 30, 2024, the Company recorded $0.1 and $0.3 million, respectively, in expense related to the Columbia Agreements. During the three and nine months ended September 30, 2023, the Company recorded zero and $0.1 million in expense, respectively, related to the Columbia Agreements. In aggregate, the Company has incurred $3.2 million in expense from the execution of the Columbia Agreements through September 30, 2024.

As of September 30, 2024, the Company had $0.1 million due to Columbia University included in accrued expenses and no amounts in accounts payable. As of December 31, 2023, the Company had $29,000 due to Columbia University included in accrued expenses and no amounts in accounts payable.

University of Miami

2020 Miami License Agreement

On October 28, 2020, the Company entered into a license agreement with the University of Miami (the “2020 Miami License Agreement”) relating to certain technology that is co-owned by the University of Miami (UM), the University of Rochester (UR) and University College London (UCL). UM was granted an exclusive agency from UR and UCL to license each of their rights in the technology. Pursuant to the 2020 Miami License Agreement, UM, on behalf of itself and UR and UCL, granted the Company a royalty-bearing, sublicensable license that is exclusive with respect to certain patent applications and patents that may grant from the applications, and non-exclusive with respect to certain know-how, in each case to research, develop, make, have made, use, sell and import products for use in treating and/or detecting certain inherited neuropathies, in particular those caused by mutation in the sorbitol dehydrogenase (SORD) gene. The license grant is worldwide. Under the 2020 Miami License Agreement, the Company is obligated to use commercially reasonable efforts to develop, manufacture, market and sell licensed products in the licensed territory, and to comply with certain obligations to meet specified development milestones within defined time periods. UM retains for itself, UR, and UCL the right to use the licensed patent rights and licensed technology for their internal non-commercial educational, research and clinical patient care purposes, including in sponsored research and collaboration with commercial entities.

Under the terms of the 2020 Miami License Agreement, the Company was obligated to pay UM an up-front non-refundable license fee of $1.1 million, and a second non-refundable license fee of $0.5 million due on the first anniversary of the date of the license. The Company will be required to make further payments to UM of up to an aggregate $2.2 million for the achievement of specified patenting and development milestones, and up to an aggregate of $4.1 million for achievement of late stage regulatory milestones. The Company will also be required to pay royalties ranging from 0.88% - 5% on the Company’s, the Company’s affiliates’ and the Company’s sublicensees’ net sales of licensed products. When the Company sublicenses the rights granted under the 2020 Miami License Agreement to one or more third parties, the Company will be required to pay to UM a portion of the non-royalty sublicensing revenue received from such third parties ranging from 15% – 25%. The Advanz Agreement includes a sublicense under the 2020 Miami License Agreement.

The 2020 Miami License Agreement terminates upon the expiration of all issued patents and filed patent applications or 10 years after the first commercial sale of the last product or process for which a royalty is due, unless earlier terminated. In addition, the 2020 Miami License Agreement may be terminated by the Company at any time upon 60 days prior written notice to UM, and may be terminated by either the Company or UM upon material breach of an obligation if action to cure the breach is not initiated within 60 days of receipt of written notice.

The Company recorded $1.1 million and $1.3 million, respectively, in research and development expense related to the Miami License Agreement during the three and nine months ended September 30, 2024. The Company recorded $0.2 million, respectively, in research and development expense related to the Miami License Agreement during each of the three and nine months ended September 30, 2023.

As of September 30, 2024, the Company had $1.3 million due to UM included in accrued expenses relating to the 2020 Miami License Agreement. As of December 31, 2023, the Company had $0.4 million due to UM included in accrued expenses relating to the 2020 Miami License Agreement.

2020 Miami Option Agreement

On October 28, 2020, the Company entered into an option agreement with the University of Miami (the “2020 Miami Option Agreement”) concerning certain research activities and technology relating to SORD neuropathy that may be pursued and developed by UM. Under the 2020 Miami Option Agreement, if UM conducts such research activities, then UM is obligated to grant us certain option rights to access and use the research results and to obtain licenses to any

associated patent rights upon us making specified payments to UM within specified time limits. If the Company elects to obtain option rights the Company will be required to make payments to UM in the low-six figures to the low-seven figures, depending upon the rights the Company elects to obtain, and the Company will be obligated to make certain milestone payments in the high-six figures to mid-seven figures if UM conducts and completes certain research activities within specified time periods and the Company elects to receive rights to use the results of that research.

2020 Miami Sponsored Research Agreement

On December 14, 2020, the Company entered into a research agreement with the University of Miami (the “2020 Miami Research Agreement”), under which the University of Miami will conduct a research study relating to SORD neuropathy and deliver a final report on the study to the Company. The term of the research agreement was from December 14, 2020 through December 30, 2021, and was extended through August 31, 2022, whereby the research study was completed. The total consideration for the 2020 Miami Research Agreement was $0.3 million.

During the three and nine months ended September 30, 2024 and 2023 , the Company recorded no research and development expense in relation to the 2020 Miami Research Agreement.

As of September 30, 2024, there are no amounts recorded as accrued expenses related to the 2020 Miami Research Agreement. As of December 31, 2023, the Company had $0.1 million in accrued expenses relating to the 2020 Miami Research Agreement.

Bayh-Dole Act

Some of the intellectual property rights the Company has licensed, including certain rights licensed in the agreements described above, may have been generated through the use of U.S. government funding. As a result, the U.S. government may have certain rights to intellectual property embodied in the Company’s current or future product candidates under the Bayh-Dole Act of 1980, or Bayh-Dole Act, including the grant to the government of a non-exclusive, worldwide, freedom to operate license under any patents, and the requirement, absent a waiver, to manufacture products substantially in the United States. To the extent any of the Company’s current or future intellectual property is generated through the use of U.S. government funding, the provisions of the Bayh-Dole Act may similarly apply.

Leases (Tables)

9 Months Ended

Sep. 30, 2024

Leases [Abstract]

Summary of Lease Related Costs

The following table summarizes the Company’s lease related costs for the three and nine months ended September 30, 2024 and 2023:


		Three Months Ended				Nine Months Ended
(in thousands)		September 30,				September 30,
Lease Cost	Statement of Operations Location	2024		2023		2024		2023
Operating Lease Cost	General and administrative	$	132	$	126	$	386	$	378
Total lease cost		$	132	$	126	$	386	$	378

Summary of Lease Terms and Discount Rates

Average lease terms and discount rates for the Company’s operating leases were as follows:


	Nine Months Ended
	September 30,			September 30,
Other Information	2024			2023
Weighted-average remaining lease term
Operating leases	5.0 years			1.1 years
Weighted-average discount rate
Operating leases		15.45	%		5.69	%

Summary of Maturities of Lease Liabilities

The following table summarizes the maturities of lease liabilities as of September 30, 2024:


	Operating
Year	(in thousands)
2024		135
2025		546
2026		556
2027		550
2028		563
Thereafter		479
Total lease payments		2,829
Less: interest		858
Total lease liabilities	$	1,971

Stock Based Compensation - Options Activity (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended		12 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023
Options Outstanding
Outstanding at beginning of period (in shares)			4,695,619
Options granted (in shares)			0
Options exercised (in shares)			(327,492)
Forfeited (in shares)			(58,123)
Outstanding at end of period (in shares)	4,310,004		4,310,004		4,695,619
Exercisable at end of period (in shares)	4,168,686		4,168,686
Nonvested at end of period (in shares)	141,318		141,318
Weighted-Average Exercise Price
Outstanding at beginning of period (in dollars per share)			$ 2.01
Options exercised (in dollars per share)			1.05
Options forfeited (in dollars per share)			1.05
Outstanding at end of period (in dollars per share)	$ 2.09		2.09		$ 2.01
Exercisable at end of period (in dollars per share)	2.13		2.13
Nonvested at end of period (in dollars per share)	$ 1.05		$ 1.05
Weighted-Average Remaining Contractual Term-in years
Outstanding			5 years		5 years 10 months 24 days
Exercisable			5 years
Nonvested			7 years 2 months 12 days
Aggregate Intrinsic Value
Options exercised (in dollars)	$ 0	$ 100	$ 1,100	$ 100
Outstanding (in dollars)	27,613		27,613		$ 7,875
Exercisable (in dollars)	26,561		26,561
Nonvested (in dollars)	1,052		1,052
Grant-date fair value of stock options vested during the period	$ 100	$ 100	$ 200	$ 400

Warrants - Warrants Issued with June 2022 Offering - Additional Information (Details) - USD ($)		3 Months Ended				9 Months Ended		27 Months Ended
	Jun. 27, 2022	Sep. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Dec. 31, 2023
Warrants
Proceeds from exercise of warrants						$ 9,025,000	$ 10,250,000
Common Stock
Warrants
Exercise of pre-funded warrants for common stock (in shares)		1,385,525	4,206,285		4,250,000
Exercise of common warrants (in shares)			9,025,000	10,250,000
Common And Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)						2,000,000
Warrants exercised (in shares)						14,617,680
Warrants outstanding (in shares)		38,915,972				38,915,972		38,915,972	51,533,652
Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)						2,000,000
Warrants exercised (in shares)						5,592,680
Warrants outstanding (in shares)		28,190,972				28,190,972		28,190,972	31,783,652
Common Warrants
Warrants
Warrants exercised (in shares)						9,025,000
Warrants outstanding (in shares)		10,725,000				10,725,000		10,725,000	19,750,000
June 2022 Offering
Warrants
Offering price per share and accompanying warrant (in dollars per share)	$ 1
Offering price per pre-funded warrant and accompanying warrant (in dollars per share)	$ 0.9999
Warrants term	5 years
June 2022 Offering \| Common Stock
Warrants
Exercise of pre-funded warrants for common stock (in shares)								8,500,000
Exercise of common warrants (in shares)								19,275,000
June 2022 Offering \| Common And Pre-Funded Warrants
Warrants
Warrants term	5 years
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants	9.99%
Threshold percentage of aggregate beneficial ownership of holder (together with affiliates) for exercising warrants	19.99%
June 2022 Offering \| Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)	10,000,000
Exercise price (in dollars per share)	$ 0.0001
Warrants exercised (in shares)								8,500,000
Proceeds from exercise of warrants								$ 850
Warrants outstanding (in shares)		1,500,000				1,500,000		1,500,000
June 2022 Offering \| Pre-Funded Warrants \| Weighted average
Warrants
Exercise price (in dollars per share)		$ 0.0001				$ 0.0001		$ 0.0001
June 2022 Offering \| Pre-Funded Warrants \| Average
Warrants
Warrants term						5 years
June 2022 Offering \| Common Warrants
Warrants
Issuance of warrants (in shares)	30,000,000
Exercise price (in dollars per share)	$ 1
Warrants exercised (in shares)								19,275,000
Proceeds from exercise of warrants								$ 19,300,000
Warrants outstanding (in shares)		10,725,000				10,725,000		10,725,000
June 2022 Offering \| Common Warrants \| Weighted average
Warrants
Exercise price (in dollars per share)		$ 1				$ 1		$ 1
June 2022 Offering \| Common Warrants \| Average
Warrants
Warrants term						5 years

Net Loss Per Common Share

9 Months Ended

Sep. 30, 2024

Earnings Per Share [Abstract]

Net Loss Per Common Share

12. NET LOSS PER COMMON SHARE

Basic net loss per common share is computed by dividing the net loss available to common stockholders by the weighted‑average number of shares of common stock outstanding during the period, which includes shares issuable under pre-funded warrants agreements for little consideration.

Diluted net loss per common share is computed by giving the effect of all potential shares of common stock, including stock options, preferred shares, warrants and instruments convertible into common stock, to the extent dilutive. Basic and diluted net loss per common share was the same for the three and nine months ended September 30, 2024 and 2023, as the inclusion of all potential common shares outstanding would have been anti‑dilutive.

The following tables set forth the computation of basic and diluted net loss per common share for the three and nine months ended September 30, 2024 and 2023:


	Three Months Ended						Nine Months Ended
	September 30,						September 30,
(in thousands, except for share data)	2024			2023			2024			2023
Numerator:
Net loss	$	(68,591	)	$	(42,370	)	$	(149,631	)	$	(82,084	)
Denominator:
Weighted-average common stock outstanding		144,345,781			90,669,969			137,893,249			75,482,234
Net loss per share attributable to common stockholders - basic and diluted	$	(0.48	)	$	(0.47	)	$	(1.09	)	$	(1.09	)

The Company excluded the following potential common shares, presented based on amounts outstanding for the three and nine months ended September 30, 2024 and 2023, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti‑dilutive effect:


	As of
	September 30,
	2024		2023
Options to purchase common stock		4,310,004		4,695,619
Restricted stock units		6,451,812		3,326,336
Warrants to purchase common stock		10,850,618		19,875,618

Condensed Balance Sheets (Parenthetical) - $ / shares	Sep. 30, 2024	Dec. 31, 2023
Condensed Balance Sheets
Common Stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Common Stock, authorized (in shares)	250,000,000	200,000,000
Common Stock, issued (in shares)	116,356,474	84,869,832
Common stock, outstanding (in shares)	116,356,474	84,869,832
Preferred Stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Preferred Stock, authorized (in shares)	10,000,000	10,000,000
Preferred Stock, issued (in shares)	0	0
Preferred Stock, outstanding (in shares)	0	0

Stockholders' Equity - April 2023 Offering (Details) - USD ($)			3 Months Ended	9 Months Ended
	Apr. 26, 2023	Apr. 23, 2023	Sep. 30, 2023	Sep. 30, 2024
Common And Pre-Funded Warrants
Equity
Issuance of warrants (in shares)				2,000,000
Pre-Funded Warrants
Equity
Issuance of warrants (in shares)				2,000,000
Common Stock
Equity
Issuance of common stock (in shares)			4,284,344
April 2023 Offering, Private Placement
Equity
Proceeds from issuance of shares and warrants, net	$ 27.5
April 2023 Offering, Private Placement \| Pre-Funded Warrants
Equity
Issuance of warrants (in shares)	22,000,000	22,000,000
Exercise price (in dollars per share)	$ 0.001	$ 0.001
Purchase price, warrant (in dollars per share)	$ 0.945	$ 0.945
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants	9.99%	9.99%
April 2023 Offering, Private Placement \| Common Stock
Equity
Issuance of common stock (in shares)	9,735,731
Purchase price, common stock (in dollars per share)	$ 0.946	$ 0.946

Condensed Statements of Operations - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Condensed Statements of Operations - USD ($) $ in Thousands	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
REVENUE:
Total revenue	$ 122		$ 455	$ 10,660
COSTS AND EXPENSES:
Research and development	14,828	$ 10,785	37,049	38,602
General and administrative	15,037	4,710	34,683	15,585
Total costs and expenses	29,865	15,495	71,732	54,187
LOSS FROM OPERATIONS	(29,743)	(15,495)	(71,277)	(43,527)
OTHER (EXPENSE) INCOME, NET:
Interest income	1,357	392	2,572	1,020
Change in fair value of warrant liabilities	(40,184)	(27,277)	(80,845)	(39,611)
Other (expense) income, net	(21)	10	(81)	34
Total other expense, net	(38,848)	(26,875)	(78,354)	(38,557)
Net loss	$ (68,591)	$ (42,370)	$ (149,631)	$ (82,084)
Net loss per share attributable to common stockholders - basic	$ (0.48)	$ (0.47)	$ (1.09)	$ (1.09)
Net loss per share attributable to common stockholders - diluted	$ (0.48)	$ (0.47)	$ (1.09)	$ (1.09)
Weighted-average common stock outstanding - basic (in shares)	144,345,781	90,669,969	137,893,249	75,482,234
Weighted-average common stock outstanding - diluted (in shares)	144,345,781	90,669,969	137,893,249	75,482,234
License
REVENUE:
Total revenue				$ 10,660
Research and development services
REVENUE:
Total revenue	$ 122		$ 455

Stock Based Compensation - 2019 Employee Stock Purchase Plan (Details) - 2019 ESPP - shares	1 Months Ended	9 Months Ended
	May 31, 2019	Sep. 30, 2024
STOCK-BASED COMPENSATION
Shares authorized (in shares)	180,000
Shares issued under ESPP		0
Maximum
STOCK-BASED COMPENSATION
Annual increase in number of shares reserved and available for issuance (as a percent)	1.00%
Increase in number of shares reserved and available for issuance (in shares)	360,000

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Net Loss Per Common Share (Tables)

9 Months Ended

Sep. 30, 2024

Earnings Per Share [Abstract]

Schedule of Computation of Basic and Diluted Net Loss Per Common Share

The following tables set forth the computation of basic and diluted net loss per common share for the three and nine months ended September 30, 2024 and 2023:


	Three Months Ended						Nine Months Ended
	September 30,						September 30,
(in thousands, except for share data)	2024			2023			2024			2023
Numerator:
Net loss	$	(68,591	)	$	(42,370	)	$	(149,631	)	$	(82,084	)
Denominator:
Weighted-average common stock outstanding		144,345,781			90,669,969			137,893,249			75,482,234
Net loss per share attributable to common stockholders - basic and diluted	$	(0.48	)	$	(0.47	)	$	(1.09	)	$	(1.09	)

Schedule of Potential Dilutive Securities Excluded From the Computation of Diluted Net Loss Per Share Attributable to Common Stockholders


	As of
	September 30,
	2024		2023
Options to purchase common stock		4,310,004		4,695,619
Restricted stock units		6,451,812		3,326,336
Warrants to purchase common stock		10,850,618		19,875,618

Leases - Summary of Lease Related Cost (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Leases
Operating Lease Cost	$ 132	$ 126	$ 386	$ 378
General and administrative
Leases
Operating Lease Cost	$ 132	$ 126	$ 386	$ 378

Fair Value Measurements - Warrants (Details) - shares		9 Months Ended
Fair Value Measurements - Warrants (Details) - shares	Jun. 27, 2022	Sep. 30, 2024
Common And Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)		2,000,000
Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)		2,000,000
June 2022 Offering \| Common Warrants
Warrants
Issuance of warrants (in shares)	30,000,000
June 2022 Offering \| Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)	10,000,000

Related Parties - Additional information (Details) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023
Related Party Transactions
Research and development expense	$ 14,828,000	$ 10,785,000	$ 37,049,000	$ 38,602,000
Due to related parties	100,674,000		100,674,000		$ 71,979,000
LaunchLabs Agreement \| Subsidiary whose founder and executive chairman of parent is a member of the Company's board of directors \| Alexandria LaunchLabs
Related Party Transactions
Research and development expense	25,000	$ 24,000	100,000	$ 100,000
Due to related parties	$ 0		$ 0		$ 8,000,000

- Definition

Amount of liability recognized for present obligation requiring transfer or otherwise providing economic benefit to others.

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+ Details

Stockholders' Equity - June 2022 Offering (Details) - USD ($) $ / shares in Units, $ in Millions		3 Months Ended	9 Months Ended
	Jun. 27, 2022	Sep. 30, 2023	Sep. 30, 2024
Common And Pre-Funded Warrants
Equity
Issuance of warrants (in shares)			2,000,000
Pre-Funded Warrants
Equity
Issuance of warrants (in shares)			2,000,000
Common Stock
Equity
Issuance of common stock (in shares)		4,284,344
June 2022 Offering
Equity
Offering price per share and accompanying warrant (in dollars per share)	$ 1
Offering price per pre-funded warrant and accompanying warrant (in dollars per share)	$ 0.9999
Proceeds from issuance of shares and warrants, net	$ 27.8
Warrants term	5 years
June 2022 Offering \| Common And Pre-Funded Warrants
Equity
Warrants term	5 years
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants	9.99%
Threshold percentage of aggregate beneficial ownership of holder (together with affiliates) for exercising warrants	19.99%
June 2022 Offering \| Pre-Funded Warrants
Equity
Issuance of warrants (in shares)	10,000,000
June 2022 Offering \| Common Warrants
Equity
Issuance of warrants (in shares)	30,000,000
June 2022 Offering \| Common Stock
Equity
Issuance of common stock (in shares)	20,000,000

Document and Entity Information - shares	9 Months Ended
Document and Entity Information - shares	Sep. 30, 2024	Nov. 06, 2024
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2024
Document Transition Report	false
Entity File Number	001-38898
Entity Registrant Name	Applied Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	81-3405262
Entity Address, Address Line One	545 Fifth Avenue
Entity Address, Address Line Two	Suite 1400
Entity Address, City or Town	New York
Entity Address, State or Province	NY
Entity Address, Postal Zip Code	10017
City Area Code	212
Local Phone Number	220-9226
Title of 12(b) Security	Common Stock, par value $0.0001
Trading Symbol	APLT
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	true
Entity Ex Transition Period	true
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		116,356,474
Entity Central Index Key	0001697532
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q3
Amendment Flag	false

Income Taxes

9 Months Ended

Sep. 30, 2024

Income Tax Disclosure [Abstract]

Income Taxes

10. INCOME TAXES

During the nine months ended September 30, 2024 and the year ended December 31, 2023, the Company recorded a full valuation allowance on federal and state deferred tax assets since management does not forecast the Company to be in a profitable position in the near future.

- Definition

The entire disclosure for income tax.

+ References

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+ Details

Revenue - Additional Information (Details) $ in Thousands, € in Millions			1 Months Ended	3 Months Ended		9 Months Ended
	Jan. 03, 2023 EUR (€) Item	Jan. 03, 2023 USD ($) Item	Jan. 31, 2023 USD ($)	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Dec. 31, 2023 USD ($)	Jan. 03, 2023 USD ($)
Revenue
Total revenue				$ 122		$ 455	$ 10,660
Deferred revenue				212		212		$ 667
License
Revenue
Total revenue							10,660
Research and development services
Revenue
Total revenue				122		455
License and Supply Agreement \| Advanz Pharma
Revenue
Upfront payment received	€ 10.0	$ 10,700	$ 10,700
Potential milestone payments to be received	€ 134.0								$ 142,200
Royalties as a percent of sales	20.00%								20.00%
Initial term	10 years	10 years
Royalties as a percent of sales following initial term	10.00%								10.00%
Number of performance obligations \| Item	3	3
License and Supply Agreement \| Advanz Pharma \| License
Revenue
Amount of performance obligation								9,300
Total revenue					$ 0		$ 10,700
License and Supply Agreement \| Advanz Pharma \| Research and development services
Revenue
Amount of performance obligation								1,300
Total revenue				100		500
Deferred revenue				100		100		500
License and Supply Agreement \| Advanz Pharma \| Manufacturing and supply material right
Revenue
Amount of performance obligation								100
Deferred revenue				$ 100		$ 100		$ 100

Stock Based Compensation

9 Months Ended

Sep. 30, 2024

Share-Based Payment Arrangement [Abstract]

Stock-Based Compensation

6. STOCK‑BASED COMPENSATION

Equity Incentive Plans

In May 2019, the Company’s board of directors (the “Board”) adopted its 2019 Equity Incentive Plan (“2019 Plan”), which was subsequently approved by its stockholders and became effective on May 13, 2019. As a result, no additional awards under the Company’s 2016 Equity Incentive Plan, as amended (the “2016 Plan”) will be granted and all outstanding stock awards granted under the 2016 Plan that are repurchased, forfeited, expired, or are cancelled will become available for grant under the 2019 Plan in accordance with its terms. The 2016 Plan will continue to govern outstanding equity awards granted thereunder.

The 2019 Plan provides for the issuance of incentive stock options (“ISOs”) to employees, and for the grant of nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock unit awards, performance stock awards, performance cash awards and other forms of stock awards to the Company’s employees, officers, and directors, as well as non- employees, consultants, and affiliates to the Company. Under the terms of the 2019 Plan, stock options may not be granted at an exercise price less than fair market value of the Company’s common stock on the date of the grant. The 2019 Plan is administered by the Compensation Committee of the Company’s Board.

Initially, subject to adjustments as provided in the 2019 Plan, the maximum number of the Company’s common stock that may be issued under the 2019 Plan was 4,530,000 shares, which is the sum of (i) 1,618,841 new shares, plus (ii) the number of shares (not to exceed 2,911,159 shares) that remained available for the issuance of awards under the 2016 Plan, at the time the 2019 Plan became effective, and (iii) any shares subject to outstanding stock options or other stock awards granted under the 2016 Plan that are forfeited, expired, or reacquired. The 2019 Plan provides that the number of shares reserved and available for issuance under the 2019 Plan will automatically increase each January 1, beginning on January 1, 2020, by 5% of the outstanding number of shares of common stock on the immediately preceding December 31 or such lesser number of shares as determined by the Board. Subject to certain changes in capitalization of the Company, the aggregate maximum number of shares of common stock that may be issued pursuant to the exercise of ISOs is equal to 13,000,000 shares of common stock. Stock options awarded under the 2019 Plan expire 10 years after grant and typically vest over four years.

As of September 30, 2024, there were 473,973 shares of common stock available for issuance under the 2019 Plan.

Stock-Based Compensation Expense

Total stock‑based compensation expense recorded for employees, directors and non‑employees:


	Three Months Ended				Nine Months Ended
	September 30,				September 30,
(in thousands)	2024		2023		2024		2023
Research and development	$	951	$	764	$	2,907	$	2,378
General and administrative		1,140		1,072		3,453		3,356
Total stock-based compensation expense	$	2,091	$	1,836	$	6,360	$	5,734

Stock Option Activity

During the nine months ended September 30, 2024, the Company did not grant any stock options. For the three months ended September 30, 2024 and 2023, amortization of stock compensation of options amounted to $0.4 million and $1.1 million, respectively, and for the nine months ended September 30, 2024 and 2023, amortization of stock compensation of options amounted to $1.6 million and $3.9 million, respectively. As of September 30, 2024 and 2023, the total unrecognized stock‑based compensation expense for unvested options was $0.7 million and $3.7 million, respectively, which is expected to be recognized over 1.2 years and 1.8 years, respectively.

The following table summarizes the information about stock options outstanding at September 30, 2024:


						Weighted-Average
				Weighted-		Remaining
				Average		Contractual		Aggregate
(in thousands, except for share data)	Options Outstanding			Exercise Price		Term (in years)		Intrinsic Value
Outstanding at December 31, 2023		4,695,619		$	2.01		5.9	$	7,875
Options granted		—			—
Options exercised		(327,492	)		1.05
Forfeited		(58,123	)		1.05
Expired		—			—
Outstanding at September 30, 2024		4,310,004		$	2.09		5.0	$	27,613

Exercisable at September 30, 2024		4,168,686		$	2.13		5.0	$	26,561
Nonvested at September 30, 2024		141,318		$	1.05		7.2	$	1,052

The aggregate intrinsic value is calculated as the difference between the exercise price of all outstanding and exercisable stock options and the fair value of the Company’s common stock at September 30, 2024. The intrinsic value of stock options exercised during the three and nine months ended September 30, 2024, was zero and $1.1 million, respectively. The intrinsic value of stock options exercised during the three and nine months ended September 30, 2023, was $0.1 million. The total grant-date fair value of stock options vested during the three and nine months ended September 30, 2024 was $0.1 million and $0.2 million, respectively. The total grant-date fair value of stock options vested during the three and nine months ended September 30, 2023 was $0.1 million and $0.4 million, respectively.

Restricted Stock Unit Activity

During the nine months ended September 30, 2024, the Company granted 1,425,000 restricted stock units (“RSUs”). For the three months ended September 30, 2024 and 2023, amortization of stock compensation of RSUs amounted to $1.7 million and $0.7 million, respectively. For the nine months ended September 30, 2024 and 2023, amortization of stock compensation of RSUs amounted to $4.8 million and $1.8 million, respectively. As of September 30, 2024 and 2023, the unamortized compensation costs associated with non-vested restricted stock awards were $17.8 million and $4.5 million, respectively, with a weighted-average remaining amortization period of 2.6 years.

The following table summarizes the information about restricted stock units outstanding at September 30, 2024:


				Weighted-Average
				Grant Date
(in thousands, except for share data)	Shares			Fair Value
Outstanding at December 31, 2023		6,917,422		$	2.35
Awarded		1,425,000			5.71
Released		(1,256,626	)		1.86
Forfeited		(633,984	)		2.92
Outstanding at September 30, 2024		6,451,812		$	3.35
Nonvested at September 30, 2024		6,205,093		$	3.38

Weighted Average Remaining Recognition Period (in years)		2.6

2019 Employee Stock Purchase Plan

In May 2019, the Company’s Board and its stockholders approved the 2019 Employee Stock Purchase Plan (the “ESPP”), which became effective as of May 13, 2019. The ESPP is intended to qualify as an “employee stock purchase plan” within the meaning of Section 423 of the U.S. Internal Revenue Code of 1986, as amended. The number of shares

of common stock initially reserved for issuance under the ESPP was 180,000 shares. The ESPP provides for an annual increase on the first day of each year beginning in 2020 and ending in 2029, equal to the lesser of (i) 1% of the shares of common stock outstanding on the last day of the calendar month before the date of the automatic increase and (ii) 360,000 shares; provided that prior to the date of any such increase, the Board may determine that such increase will be less than the amount set forth in clauses (i) and (ii). As of September 30, 2024, no shares of common stock had been issued under the ESPP. The first offering period will commence on November 15, 2024.

Related Parties

9 Months Ended

Sep. 30, 2024

Related Parties

13. RELATED PARTIES

In December 2018, the Company entered into an agreement (the “LaunchLabs Agreement”) with ARE‑LaunchLabs NYC LLC (“Alexandria LaunchLabs”), a subsidiary of Alexandria Real Estate Equities, Inc. for use of specified premises within the Alexandria LaunchLabs space on a month-to-month basis. A member of the Company’s board of directors is the founder and executive chairman of Alexandria Real Estate Equities, Inc. During the three and nine months ended September 30, 2024, the Company made payments to Alexandria LaunchLabs of approximately $25,000 and $0.1 million. During the three and nine months ended September 30, 2023, the Company made payments to Alexandria LaunchLabs of approximately $24,000 and $0.1 million, respectively, under the LaunchLabs Agreement, which was recognized in research and development expenses. As of September 30, 2024, there was no amounts due to Alexandria LaunchLabs under the LaunchLabs Agreement. As of December 31, 2023, there was $8,000 due to Alexandria LaunchLabs under the LaunchLabs Agreement.

- Definition

The entire disclosure for related party transactions. Examples of related party transactions include transactions between (a) a parent company and its subsidiary; (b) subsidiaries of a common parent; (c) and entity and its principal owners; and (d) affiliates.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-URI https://asc.fasb.org/1943274/2147480990/946-20-50-2

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Warrants - Schedule of Fair Value of Warrants Using the Black Scholes Option Pricing Model (Details)	Sep. 30, 2024 yr	Dec. 31, 2023 yr	Feb. 28, 2019 yr	Dec. 31, 2018 yr	Jan. 26, 2017 yr
Warrants Issued with Series A Preferred Stock \| 2017 Warrants \| Expected term (in years)
Class of Warrant or Right [Line Items]
Measurement input					10
Warrants Issued with Series A Preferred Stock \| 2017 Warrants \| Volatility
Class of Warrant or Right [Line Items]
Measurement input					0.7448
Warrants Issued with Series A Preferred Stock \| 2017 Warrants \| Risk-free interest rate
Class of Warrant or Right [Line Items]
Measurement input					0.032
Warrants Issued with Series A Preferred Stock \| 2017 Warrants \| Measurement Input, Expected Dividend Rate [Member]
Class of Warrant or Right [Line Items]
Measurement input					0
Warrants Issued with Series A Preferred Stock \| 2018 Warrants \| Expected term (in years)
Class of Warrant or Right [Line Items]
Measurement input				10
Warrants Issued with Series A Preferred Stock \| 2018 Warrants \| Volatility
Class of Warrant or Right [Line Items]
Measurement input				0.7322
Warrants Issued with Series A Preferred Stock \| 2018 Warrants \| Risk-free interest rate
Class of Warrant or Right [Line Items]
Measurement input				0.027
Warrants Issued with Series A Preferred Stock \| 2018 Warrants \| Measurement Input, Expected Dividend Rate [Member]
Class of Warrant or Right [Line Items]
Measurement input				0
Warrants Issued with Series A Preferred Stock \| 2019 Warrants \| Expected term (in years)
Class of Warrant or Right [Line Items]
Measurement input			10
Warrants Issued with Series A Preferred Stock \| 2019 Warrants \| Volatility
Class of Warrant or Right [Line Items]
Measurement input			0.7322
Warrants Issued with Series A Preferred Stock \| 2019 Warrants \| Risk-free interest rate
Class of Warrant or Right [Line Items]
Measurement input			0.027
Warrants Issued with Series A Preferred Stock \| 2019 Warrants \| Measurement Input, Expected Dividend Rate [Member]
Class of Warrant or Right [Line Items]
Measurement input			0
Common Warrants \| Expected term (in years)
Class of Warrant or Right [Line Items]
Measurement input	2.1	2.4
Common Warrants \| Expected term (in years) \| June 2022 Offering
Class of Warrant or Right [Line Items]
Measurement input	2.1	2.4
Common Warrants \| Volatility
Class of Warrant or Right [Line Items]
Measurement input	0.9992	1.0946
Common Warrants \| Volatility \| June 2022 Offering
Class of Warrant or Right [Line Items]
Measurement input	0.9992	1.0946
Common Warrants \| Risk-free interest rate
Class of Warrant or Right [Line Items]
Measurement input	0.0377	0.0432
Common Warrants \| Risk-free interest rate \| June 2022 Offering
Class of Warrant or Right [Line Items]
Measurement input	0.0377	0.0432
Common Warrants \| Measurement Input, Expected Dividend Rate [Member]
Class of Warrant or Right [Line Items]
Measurement input	0	0
Common Warrants \| Measurement Input, Expected Dividend Rate [Member] \| June 2022 Offering
Class of Warrant or Right [Line Items]
Measurement input	0	0

Leases - Additional Information (Details) - New York Lease - USD ($) $ in Millions	Nov. 10, 2024	Sep. 11, 2024
Lessee, Lease, Description [Line Items]
Lease renewal term		5 years
Lease payments		$ 2.7
Scenario Forecast
Lessee, Lease, Description [Line Items]
Lease term	5 years
Lease payments	$ 1.3

Condensed Statements of Stockholders Equity/(Deficit) (Parenthetical) - USD ($) $ in Millions	3 Months Ended
	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Sep. 30, 2024
Increase (Decrease) in Stockholders' Equity
Issuance costs	$ 8.2		$ 2.5
Common Stock, par value (in dollars per share)				$ 0.0001
Common Stock
Increase (Decrease) in Stockholders' Equity
Issuance costs		$ 0.4
Common Stock, par value (in dollars per share)		$ 0.0001		$ 0.0001

Stockholders' Equity - Authorized stock (Details) - $ / shares	Sep. 30, 2024	Dec. 31, 2023
Equity [Abstract]
Common Stock, authorized (in shares)	250,000,000	200,000,000
Common Stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Preferred Stock, authorized (in shares)	10,000,000	10,000,000
Preferred Stock, par value (in dollars per share)	$ 0.0001	$ 0.0001

Warrants - Warrants Issued with the 2018 Notes - Additional Information (Details) - Warrants Issued with the 2018 Notes - USD ($) $ / shares in Units, $ in Millions	Nov. 05, 2018	Sep. 30, 2024	Dec. 31, 2023	Jan. 18, 2018
Warrants
Initial fair value recorded as debt discount (in dollars)				$ 0.1
Issuance of warrants (in shares)	76,847
Issuance of warrants (in dollars)	$ 0.3
Exercise price (in dollars per share)		$ 3.98	$ 3.99

Stock Based Compensation - Options (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
STOCK-BASED COMPENSATION
Options granted (in shares)			0
Stock-based compensation expense	$ 2,091	$ 1,836	$ 6,360	$ 5,734
Unrecognized stock-based compensation balance for unvested options	700	3,700	700	3,700
Employee Stock Option
STOCK-BASED COMPENSATION
Stock-based compensation expense	$ 400	$ 1,100	$ 1,600	$ 3,900
Weighted Average Remaining Recognition Period (in years)			1 year 2 months 12 days	1 year 9 months 18 days

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Fair Value Measurements - FV hierarchy (Details) - Fair Value, Recurring - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	$ 98,867	$ 49,898
Total financial assets measured at fair value on a recurring basis	98,867	49,898
Warrant liabilities - common warrants	82,377	53,725
Total financial liabilities measured at fair value on a recurring basis	82,377	53,725
Level 1
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	98,867	49,898
Total financial assets measured at fair value on a recurring basis	98,867	49,898
Level 3
Financial assets or liabilities measured at fair value on a recurring basis
Warrant liabilities - common warrants	82,377	53,725
Total financial liabilities measured at fair value on a recurring basis	82,377	53,725
Cash
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	13,757	24,887
Cash \| Level 1
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	13,757	24,887
Money market funds
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	85,110	25,011
Money market funds \| Level 1
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	$ 85,110	$ 25,011

Organization and Summary of Significant Accounting Policies - Liquidity and Going Concern - Additional Information (Details) $ in Thousands, € in Millions			1 Months Ended	9 Months Ended
	Jan. 03, 2023 EUR (€)	Jan. 03, 2023 USD ($)	Jan. 31, 2023 USD ($)	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Dec. 31, 2023 USD ($)
Cash and cash equivalents
Proceeds from issuance of shares and warrants and warrant exercises, net				$ 113,800
Net loss				(149,631)	$ (82,084)
Accumulated deficit				(618,217)		$ (468,586)
Cash and cash equivalents				$ 98,867		$ 49,898
Substantial Doubt about Going Concern, within One Year				false
Advanz Pharma \| License and Supply Agreement
Cash and cash equivalents
Upfront payment received	€ 10.0	$ 10,700	$ 10,700

- Definition

The consolidated profit or loss for the period, net of income taxes, including the portion attributable to the noncontrolling interest.

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-Section 45
-Paragraph 1A
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482790/220-10-45-1A

Reference 34: http://www.xbrl.org/2003/role/disclosureRef
-Topic 220
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 1B
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482790/220-10-45-1B

Reference 35: http://www.xbrl.org/2003/role/disclosureRef
-Topic 230
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482740/230-10-45-2

Reference 36: http://www.xbrl.org/2003/role/disclosureRef
-Topic 810
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1A
-Subparagraph (a)(1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481203/810-10-50-1A

Reference 37: http://www.xbrl.org/2003/role/disclosureRef
-Topic 810
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1A
-Subparagraph (c)(1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481203/810-10-50-1A

+ Details

- Definition

Amount of accumulated undistributed earnings (deficit).

+ References + Details

Condensed Balance Sheets - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
CURRENT ASSETS:
Cash and cash equivalents	$ 98,867	$ 49,898
Security deposits and leasehold improvements	253	254
Prepaid expenses and other current assets	5,483	4,234
Total current assets	104,603	54,386
Operating lease right-of-use asset	1,963	447
TOTAL ASSETS	106,566	54,833
CURRENT LIABILITIES:
Current portion of operating lease liabilities	264	429
Accounts payable	2,837	1,742
Accrued expenses and other current liabilities	13,489	15,286
Warrant liabilities	82,377	53,725
Total current liabilities	98,967	71,182
NONCURRENT LIABILITIES:
Noncurrent portion of operating lease liabilities	1,707	38
Clinical holdback - long-term portion		759
Total noncurrent liabilities	1,707	797
Total liabilities	100,674	71,979
STOCKHOLDERS' EQUITY/(DEFICIT):
Common stock, $0.0001 par value; 250,000,000 shares authorized as of September 30, 2024 and 200,000,000 shares authorized as of December 31, 2023; 116,356,474 shares issued and outstanding as of September 30, 2024 and 84,869,832 shares issued and outstanding as of December 31, 2023	11	8
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of September 30, 2024 and December 31, 2023; 0 shares issued and outstanding as of September 30, 2024 and December 31, 2023
Additional paid-in capital	624,098	451,432
Accumulated deficit	(618,217)	(468,586)
Total stockholders' (deficit)/equity	5,892	(17,146)
TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY/(DEFICIT)	$ 106,566	$ 54,833

Stock Based Compensation - Plans (Details) - shares	1 Months Ended	9 Months Ended
Stock Based Compensation - Plans (Details) - shares	May 31, 2019	Sep. 30, 2024	Sep. 30, 2023
Employee Stock Option
STOCK-BASED COMPENSATION
Weighted Average Remaining Recognition Period (in years)		1 year 2 months 12 days	1 year 9 months 18 days
2019 Plan
STOCK-BASED COMPENSATION
Shares reserved (in shares)	4,530,000
New shares reserved (in shares)	1,618,841
Reserved shares available from 2016 Plan (in shares)	2,911,159
Annual increase in number of shares reserved and available for issuance (as a percent)	5.00%
Number of shares available for future issuance		473,973
2019 Plan \| Employee Stock Option
STOCK-BASED COMPENSATION
Maximum number of shares authorized for issuance	13,000,000
Expiration period	10 years
Vesting period	4 years

Fair Value Measurements - Warrants Valuation (Details) - Common Warrants	Sep. 30, 2024 yr	Dec. 31, 2023 yr
Expected term (in years)
Fair value
Measurement input	2.1	2.4
Volatility
Fair value
Measurement input	0.9992	1.0946
Risk-free interest rate
Fair value
Measurement input	0.0377	0.0432
Dividend yield
Fair value
Measurement input	0	0

Benefit Plans - Additional Information (Details) - USD ($) $ in Millions	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Retirement Benefits [Abstract]
Employer matching contributions to the plan	$ 0.1	$ 0.1	$ 0.3	$ 0.3

Stock Based Compensation - RSUs (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
STOCK-BASED COMPENSATION
Stock-based compensation expense	$ 2,091	$ 1,836	$ 6,360	$ 5,734
Restricted stock units
STOCK-BASED COMPENSATION
Stock-based compensation expense	1,700	700	4,800	1,800
Unamortized stock-based compensation costs for unvested awards	$ 17,800	$ 4,500	$ 17,800	$ 4,500
Summary of restricted stock units outstanding
Outstanding at beginning of period ( in shares)			6,917,422
Awarded (in shares)			1,425,000
Released (in shares)			(1,256,626)
Forfeited (in shares)			(633,984)
Outstanding at end of period ( in shares)	6,451,812		6,451,812
Nonvested at end of period (in shares)	6,205,093		6,205,093
Weighted-Average Grant Date Fair Value
Outstanding at beginning of period (in dollars per share)			$ 2.35
Awarded (in dollars per share)			5.71
Released (in dollars per share)			1.86
Forfeited (in dollars per share)			2.92
Outstanding at end of period (in dollars per share)	$ 3.35		3.35
Nonvested at end of period (in dollars per share)	$ 3.38		$ 3.38
Weighted Average Remaining Recognition Period (in years)			2 years 7 months 6 days

Fair Value Measurements

9 Months Ended

Sep. 30, 2024

Fair Value Disclosures [Abstract]

Fair Value Measurements

3. FAIR VALUE MEASUREMENTS

The following tables summarize, as of September 30, 2024, the Company’s financial assets and liabilities that are measured at fair value on a recurring basis, according to the fair value hierarchy described in the significant accounting policies in the Company’s audited financial statements as of and for the year ended December 31, 2023, and the notes thereto, which are included in the Annual Report.


	As of
	September 30, 2024
(in thousands)	Level 1		Level 2		Level 3		Total
Cash	$	13,757	$	—	$	—	$	13,757
Money market funds		85,110		—		—		85,110
Total cash and cash equivalents	$	98,867	$	—	$	—	$	98,867
Total financial assets measured at fair value on a recurring basis	$	98,867	$	—	$	—	$	98,867
Warrant liabilities - common warrants		—		—		82,377		82,377
Total financial liabilities measured at fair value on a recurring basis	$	—	$	—	$	82,377	$	82,377

The following tables summarize, as of December 31, 2023, the Company’s financial assets and liabilities that are measured at fair value on a recurring basis.


	As of
	December 31, 2023
(in thousands)	Level 1		Level 2		Level 3		Total
Cash	$	24,887	$	—	$	—	$	24,887
Money market funds		25,011		—		—		25,011
Total cash and cash equivalents	$	49,898	$	—	$	—	$	49,898
Total financial assets measured at fair value on a recurring basis	$	49,898	$	—	$	—	$	49,898
Warrant liabilities - common warrants		—		—		53,725		53,725
Total financial liabilities measured at fair value on a recurring basis	$	—	$	—	$	53,725	$	53,725

On June 27, 2022, the Company issued 30,000,000 warrants to purchase shares of common stock (the “Common Warrants”) and 10,000,000 pre-funded warrants to purchase common stock (the “Pre-Funded Warrants”) in connection with the June 2022 Offering (see Note 7 for more information on the June 2022 Offering). The Common Warrants were accounted for as liabilities under ASC 815-40, Derivatives and Hedging, Contracts in Entity’s Own Equity (“ASC 815-40”), as these warrants provide for a settlement provision that does not meet the requirements of the indexation guidance under ASC 815-40. The Pre-Funded Warrants were initially recorded at fair value as a liability as the Company could be required to settle the Pre-Funded Warrants in cash under certain circumstances. In December 2022, the Company amended the Pre-Funded Warrants to remove the potential requirement that they could be settled in cash under certain circumstances. Upon the amendment to the Pre-Funded Warrants, the Pre-funded Warrants liability was reclassified to equity, using their fair value as of the amendment date.

The Common Warrant liabilities were measured at fair value at inception and are then subsequently measured on a recurring basis, with changes in fair value recognized in other income (expense) within the Company’s statement of operations.

The Company uses a Black-Scholes option pricing model to estimate the fair value of the Common and Pre-Funded Warrants, which utilizes certain unobservable inputs and is therefore considered a Level 3 fair value measurement. Certain inputs used in this Black-Scholes pricing model may fluctuate in future periods based upon factors that are outside of the Company’s control, including a potential change in control outside of the Company’s control. A significant change in one or more of these inputs used in the calculation of the fair value may cause a significant change to the fair value of the Company’s warrant liabilities, which could also result in material non-cash gains or losses being reported in the Company’s condensed statement of operations.

The Common Warrants were remeasured using a Black-Scholes option pricing model with a range of assumptions included below as of September 30, 2024 and December 31, 2023.


	September 30,			December 31,
	2024			2023
Expected term (in years)		2.1			2.4
Volatility		99.92	%		109.46	%
Risk-free interest rate		3.77	%		4.32	%
Dividend yield		0.00	%		0.00	%

As of September 30, 2024, the Company utilized a probability-weighted approach that considered the probability of a change in control at the Company in the Black-Scholes option pricing model, whereby a 20% probability of change in control was used for years three and four and a 10% probability was used for year five in the term of the agreements.

As of December 31, 2023 the Company utilized a probability-weighted approach that considered the probability of a change in control at the Company in the Black-Scholes option pricing model, whereby a 20% probability of change

in control was used for years two and three and a 5% probability was used for years four and five in the term of the agreements.

The following table provides a roll forward of the aggregate fair values of the Company’s warrant liability, for which fair value is determined using Level 3 inputs (in thousands):


	Warrant Liabilities
Balance as of January 1, 2024	$	53,725
Warrants exercised		(52,193	)
Change in fair value		80,845
Balance as of September 30, 2024	$	82,377

The inputs utilized by management to value the warrant liabilities are highly subjective. The assumptions used in calculating the fair value of the warrant liabilities represent the Company’s best estimates, but these estimates involve inherent uncertainties and the application of management judgment. As a result, if factors change and the Company uses different assumptions, the fair value of the warrant liability for Common Warrants may be materially different in the future.

Stockholders' Equity - ATM Offering (Details) - USD ($) $ / shares in Units, $ in Millions		3 Months Ended	9 Months Ended
	Aug. 11, 2023	Sep. 30, 2023	Sep. 30, 2024
Common Stock
Equity
Issuance of common stock (in shares)		4,284,344
Leerink ATM Agreement
Equity
Maximum value of shares authorized to be sold in stock offering	$ 75.0
Maximum amount to be paid to sales agent (as a percent)	3.00%
Proceeds from stock issuance, net of cash issuance costs			$ 49.3
Leerink ATM Agreement \| Common Stock
Equity
Issuance of common stock (in shares)			20,615,976
Leerink ATM Agreement \| Common Stock \| Average
Equity
Purchase price, common stock (in dollars per share)			$ 3.36

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Warrants - Warrants Issued with Series B Preferred Stock - Additional Information (Details) - Warrants Issued with Series B Preferred Stock - USD ($) $ / shares in Units, $ in Millions	1 Months Ended	2 Months Ended
	Feb. 28, 2019	Dec. 31, 2018	Sep. 30, 2024	Dec. 31, 2023
2018 Warrants
Warrants
Number of warrants entity became obligated to issue (in shares)		72,261
Issuance of warrants (in dollars)		$ 0.2
Expiration term		10 years
Exercise price (in dollars per share)			$ 4.84	$ 4.86
2019 Warrants
Warrants
Number of warrants entity became obligated to issue (in shares)	23,867
Issuance of warrants (in dollars)	$ 0.1
Expiration term	10 years
Exercise price (in dollars per share)			$ 4.84	$ 4.86

Fair Value Measurements (Tables)

9 Months Ended

Sep. 30, 2024

Fair value

Schedule of financial assets or liabilities measured at fair value on a recurring basis


	As of
	September 30, 2024
(in thousands)	Level 1		Level 2		Level 3		Total
Cash	$	13,757	$	—	$	—	$	13,757
Money market funds		85,110		—		—		85,110
Total cash and cash equivalents	$	98,867	$	—	$	—	$	98,867
Total financial assets measured at fair value on a recurring basis	$	98,867	$	—	$	—	$	98,867
Warrant liabilities - common warrants		—		—		82,377		82,377
Total financial liabilities measured at fair value on a recurring basis	$	—	$	—	$	82,377	$	82,377


	As of
	December 31, 2023
(in thousands)	Level 1		Level 2		Level 3		Total
Cash	$	24,887	$	—	$	—	$	24,887
Money market funds		25,011		—		—		25,011
Total cash and cash equivalents	$	49,898	$	—	$	—	$	49,898
Total financial assets measured at fair value on a recurring basis	$	49,898	$	—	$	—	$	49,898
Warrant liabilities - common warrants		—		—		53,725		53,725
Total financial liabilities measured at fair value on a recurring basis	$	—	$	—	$	53,725	$	53,725

Schedule of aggregate fair values of the Company's warrant liability

The following table provides a roll forward of the aggregate fair values of the Company’s warrant liability, for which fair value is determined using Level 3 inputs (in thousands):


	Warrant Liabilities
Balance as of January 1, 2024	$	53,725
Warrants exercised		(52,193	)
Change in fair value		80,845
Balance as of September 30, 2024	$	82,377

Common Warrants

Fair value

Schedule of aggregate fair values of the Company's warrant liability


	September 30,			December 31,
	2024			2023
Expected term (in years)		2.1			2.4
Volatility		99.92	%		109.46	%
Risk-free interest rate		3.77	%		4.32	%
Dividend yield		0.00	%		0.00	%

Fair Value Measurements - Additional Information (Details) - Probability of change in control - Common Warrants	Sep. 30, 2024	Dec. 31, 2023
Valuation input period, years three and four
Fair value
Measurement input	20
Valuation input period, year five
Fair value
Measurement input	10
Valuation input period, years two and three
Fair value
Measurement input		20
Valuation input period, years four and five
Fair value
Measurement input		5

Fair Value Measurements - Fair Values of Company's Warrant Liability (Details) - Common Warrants $ in Thousands	9 Months Ended
	Sep. 30, 2024 USD ($)
Fair Value
Beginning Balance	$ 53,725
Warrants exercised	(52,193)
Change in fair value	80,845
Ending Balance	$ 82,377

Condensed Statements of Cash Flows - USD ($) $ in Thousands	9 Months Ended
Condensed Statements of Cash Flows - USD ($) $ in Thousands	Sep. 30, 2024	Sep. 30, 2023
OPERATING ACTIVITIES:
Net loss	$ (149,631)	$ (82,084)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation expense	6,360	5,734
Amortization of insurance premium	566	1,723
Amortization of operating lease right-of-use assets	345	347
Amortization of leasehold improvements	1	1
Change in operating lease liability	(357)	(356)
Change in fair value of warrant liabilities	80,845	39,611
Changes in operating assets and liabilities:
Financed insurance premium		(1,546)
Prepaid expenses	(1,815)	(480)
Accounts payable	1,095	1,471
Accrued expenses and other current liabilities	(1,486)	(2,816)
Other liabilities	(759)	227
Net cash used in operating activities	(64,836)	(38,168)
INVESTING ACTIVITIES:
Proceeds from sale of available-for-sale securities		4,944
Proceeds from maturities of available-for-sale securities		8,928
Net cash provided by investing activities		13,872
FINANCING ACTIVITIES:
Proceeds from issuance of common stock and pre-funded warrants, net of issuance costs	104,748	34,606
Proceeds from financed insurance premium		1,546
Repayments of short-term borrowings	(311)	(1,393)
Exercise of common warrants	9,025	10,250
Exercise of stock options	343	87
Net cash provided by financing activities	113,805	45,096
NET INCREASE IN CASH AND CASH EQUIVALENTS	48,969	20,800
Cash and cash equivalents at beginning of period	49,898	16,657
Cash and cash equivalents at end of period	98,867	37,457
SUPPLEMENTAL DISCLOSURE OF CASH FLOW INFORMATION:
Conversion of warrant liabilities to equity for warrant exercises	52,193	16,505
Operating lease right of use asset obtained in exchange for operating lease liability	$ 1,861
Unrealized loss on marketable securities		(51)
Offering costs still in accrued expenses		$ 152

Warrants - Schedule of Warrants Outstanding (Details)	9 Months Ended
Warrants - Schedule of Warrants Outstanding (Details)	Sep. 30, 2024 shares
Common And Pre-Funded Warrants
Warrants
Outstanding as of beginning of period (in shares)	51,533,652
Warrants granted and issued (in shares)	2,000,000
Warrants exercised (in shares)	(14,617,680)
Outstanding as of end of period (in shares)	38,915,972
Pre-Funded Warrants - Equity Classified Warrants
Warrants
Outstanding as of beginning of period (in shares)	31,783,652
Warrants granted and issued (in shares)	2,000,000
Warrants exercised (in shares)	(5,592,680)
Outstanding as of end of period (in shares)	28,190,972
Common Warrants - Liability Classified Warrants
Warrants
Outstanding as of beginning of period (in shares)	19,750,000
Warrants exercised (in shares)	(9,025,000)
Outstanding as of end of period (in shares)	10,725,000

Net Loss Per Common Share - Schedule of Potential Dilutive Securities Excluded From the Computation of Diluted Net Loss Per Share Attributable to Common Stockholders (Details) - shares	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Options to purchase common stock
Antidilutive securities excluded from computation of loss per share
Potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders	4,310,004	4,695,619
Restricted stock units
Antidilutive securities excluded from computation of loss per share
Potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders	6,451,812	3,326,336
Warrants to purchase common stock
Antidilutive securities excluded from computation of loss per share
Potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders	10,850,618	19,875,618

Organization and Summary of Significant Accounting Policies (Policies)	9 Months Ended
	Sep. 30, 2024
Organization and Summary of Significant Accounting Policies
Operations and Business	Operations and Business Applied Therapeutics, Inc. (the “Company”) is a clinical-stage biopharmaceutical company developing a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need. In particular, the Company is currently targeting treatments for rare diseases such as Galactosemia and Sorbitol Dehydrogenase (“SORD”) deficiency. The Company was incorporated in Delaware on January 20, 2016 and is headquartered in New York, New York. The accompanying unaudited condensed financial statements have been prepared by the Company in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”) for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted pursuant to such rules and regulations. These condensed financial statements should be read in conjunction with the Company’s audited financial statements and the notes thereto for the year ended December 31, 2023, included in the Annual Report, filed with the SEC on March 6, 2024 (the “Annual Report”). The unaudited condensed financial statements have been prepared on the same basis as the audited financial statements. In the opinion of management, the accompanying unaudited condensed financial statements contain all adjustments which are necessary for a fair presentation of the Company’s financial position as of September 30, 2024, results of operations for the three and nine months ended September 30, 2024 and 2023 and cash flows for the nine months ended September 30, 2024 and 2023. Such adjustments are of a normal and recurring nature. The results of operations for the three and nine months ended September 30, 2024, are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2024. Certain reclassifications have been made to prior period financial statements to conform to the current period presentation.
Liquidity and Going Concern	Liquidity and Going Concern Under ASC Topic 205-40, Presentation of Financial Statements - Going Concern, management is required at each reporting period to evaluate whether there are conditions and events, considered in the aggregate, that raise substantial doubt about an entity's ability to continue as a going concern within one year after the date that the financial statements are issued. As of September 30, 2024, financing through the Company’s March 2024 Private Placement, Leerink ATM Agreement and warrant exercises has resulted in net proceeds of $113.8 million, after deducting placement agent commissions and other offering expenses (see Note 7 and Note 8). The Company continues to evaluate several potential long-term financing options, including equity capital, debt, convertible debt, and synthetic royalty financing. Additionally, the Company is in active dialogue with several potential partners regarding business development opportunities related to one or more of its programs. There can be no assurances that the Company’s discussions with any of the current counterparties will be successful, and the Company expects to continue to pursue additional opportunities. As reflected in the accompanying financial statements, the Company incurred a net loss of $149.6 million for the nine months ended September 30, 2024, and has an accumulated deficit of $618.2 million as of September 30, 2024. The exclusive licensing agreement with Advanz Pharma for commercialization rights to AT-007 in Europe provides a source of capital to the Company based on clinical and regulatory milestones. The Company received a $10.7 million upfront payment from Advanz Pharma in January 2023 in conjunction with signing the agreement. If actualization of these milestones aligns with the projected timelines, and product approvals are received in the timeframes expected, this source of capital may be sufficient to cover operating expenses through expected product approvals and potential revenues. However, there are no guarantees that this will materialize timely or at all, and delays or unexpected data could disrupt this potential liquidity. Broadly, the Company has not yet established an ongoing source of revenues sufficient to cover its operating costs and is dependent on debt and equity financing to fund its operations. The Company currently expects that its existing cash and cash equivalents of $98.9 million as of September 30, 2024, will fund its operating expenses and capital requirements for at least twelve months from the date this Quarterly Report on Form 10-Q is issued.
Risk and Uncertainties	Risks and Uncertainties The Company is subject to risks common to companies in the biotechnology industry, including but not limited to, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for any product candidate that it may identify and develop, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary technology, compliance with government regulations, development by competitors of technological innovations and reliance on third-party manufacturers.
Use of Estimates	Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities and the Company's ability to continue as a going concern as of the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. In preparing the financial statements, management used significant estimates in the following areas, among others: prepaid and accrued expenses; warrant liabilities valuation; license revenue; research and development services revenue; stock-based compensation expense; the likelihood of realization of deferred tax assets; and the evaluation of the existence of conditions and events that raise substantial doubt regarding the Company’s ability to continue as a going concern. Actual results could differ from those estimates.
Recent Accounting Pronouncements	Recent Accounting Pronouncements Any recent pronouncements issued by the FASB or other authoritative standards groups with future effective dates are either not applicable or are not expected to be significant to the financial statements of the Company.

Leases

9 Months Ended

Sep. 30, 2024

Leases [Abstract]

Leases

9. LEASES

The following table summarizes the Company’s lease related costs for the three and nine months ended September 30, 2024 and 2023:


		Three Months Ended				Nine Months Ended
(in thousands)		September 30,				September 30,
Lease Cost	Statement of Operations Location	2024		2023		2024		2023
Operating Lease Cost	General and administrative	$	132	$	126	$	386	$	378
Total lease cost		$	132	$	126	$	386	$	378

Average lease terms and discount rates for the Company’s operating leases were as follows:


	Nine Months Ended
	September 30,			September 30,
Other Information	2024			2023
Weighted-average remaining lease term
Operating leases	5.0 years			1.1 years
Weighted-average discount rate
Operating leases		15.45	%		5.69	%

The following table summarizes the maturities of lease liabilities as of September 30, 2024:


	Operating
Year	(in thousands)
2024		135
2025		546
2026		556
2027		550
2028		563
Thereafter		479
Total lease payments		2,829
Less: interest		858
Total lease liabilities	$	1,971

On September 11, 2024, the Company entered into a lease renewal agreement for its New York office space located at 545 Fifth Avenue, suite 1400, New York, New York 10017 (the "New York Lease") for a five-year term with total lease payments totaling $2.7 million. In conjunction with the execution of the New York lease renewal, the Company leased additional space located in suite 1401 for a five-year term with lease payments totaling $1.3 million. The lease of suite 1401 is anticipated to commence on November 10, 2024, or such earlier date as the Company begins occupancy of the leased space for its business operations.

Stock Based Compensation (Tables)

9 Months Ended

Sep. 30, 2024

Share-Based Payment Arrangement [Abstract]

Schedule of Stock-based Compensation Expenses

Total stock‑based compensation expense recorded for employees, directors and non‑employees:


	Three Months Ended				Nine Months Ended
	September 30,				September 30,
(in thousands)	2024		2023		2024		2023
Research and development	$	951	$	764	$	2,907	$	2,378
General and administrative		1,140		1,072		3,453		3,356
Total stock-based compensation expense	$	2,091	$	1,836	$	6,360	$	5,734

Schedule of Options Outstanding

The following table summarizes the information about stock options outstanding at September 30, 2024:


						Weighted-Average
				Weighted-		Remaining
				Average		Contractual		Aggregate
(in thousands, except for share data)	Options Outstanding			Exercise Price		Term (in years)		Intrinsic Value
Outstanding at December 31, 2023		4,695,619		$	2.01		5.9	$	7,875
Options granted		—			—
Options exercised		(327,492	)		1.05
Forfeited		(58,123	)		1.05
Expired		—			—
Outstanding at September 30, 2024		4,310,004		$	2.09		5.0	$	27,613

Exercisable at September 30, 2024		4,168,686		$	2.13		5.0	$	26,561
Nonvested at September 30, 2024		141,318		$	1.05		7.2	$	1,052

Schedule of Restricted Stock Units Outstanding

The following table summarizes the information about restricted stock units outstanding at September 30, 2024:


				Weighted-Average
				Grant Date
(in thousands, except for share data)	Shares			Fair Value
Outstanding at December 31, 2023		6,917,422		$	2.35
Awarded		1,425,000			5.71
Released		(1,256,626	)		1.86
Forfeited		(633,984	)		2.92
Outstanding at September 30, 2024		6,451,812		$	3.35
Nonvested at September 30, 2024		6,205,093		$	3.38

Weighted Average Remaining Recognition Period (in years)		2.6

Condensed Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Condensed Statements of Comprehensive Income (Loss)
Net loss	$ (68,591)	$ (42,370)	$ (149,631)	$ (82,084)
Other comprehensive loss, net of tax
Unrealized loss on marketable securities				(51)
Other comprehensive loss, net of tax				(51)
Comprehensive loss, net of tax	$ (68,591)	$ (42,370)	$ (149,631)	$ (82,135)

- Definition

Amount after tax of increase (decrease) in equity from transactions and other events and circumstances from net income and other comprehensive income, attributable to parent entity. Excludes changes in equity resulting from investments by owners and distributions to owners.

+ References + Details

- Definition

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

+ References

+ Details

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Sep. 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

Stockholders' Equity

9 Months Ended

Sep. 30, 2024

Equity [Abstract]

Stockholders' Equity

7. STOCKHOLDERS’ EQUITY

As of September 30, 2024, and December 31, 2023, the authorized capital stock of the Company consisted of 250,000,000 and 200,000,000 shares of common stock, respectively, par value $0.0001 per share and 10,000,000 shares of preferred stock, par value $0.0001 per share, respectively.

Common Stock

June 2022 Offering

On June 27, 2022, the Company completed the June 2022 Offering, an underwritten public offering of 20,000,000 shares of common stock, 10,000,000 Pre-Funded Warrants, and accompanying Common Warrants to purchase up to 30,000,000 shares of common stock. The shares and accompanying Common Warrants were offered at a price to the public of $1.00 per share and warrant, and the Pre-Funded Warrants and accompanying Common Warrants were offered at a price to the public of $0.9999, resulting in aggregate net proceeds of approximately $27.8 million, after deducting underwriting discounts and commissions and offering expenses. The Pre-Funded Warrants and the Common Warrants are immediately exercisable and will expire five years from the date of issuance. Holders may not exercise any Pre-Funded Warrants or Common Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. Holders of the Pre-Funded Warrants and/or Common Warrants (together with affiliates) who immediately prior to June 27, 2022 beneficially owned more than 9.99% of the Company’s outstanding common stock may not exercise any portion of their Pre-Funded Warrants or Common Warrants if the holder (together with affiliates) would beneficially own more than 19.99% of the Company’s outstanding common stock after exercise. The Pre-Funded Warrants and Common Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants and/or Common Warrants will be entitled to receive, upon exercise, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants and/or Common Warrants immediately prior to such transaction. The Pre-Funded Warrants and Common Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which the Company’s stockholders are entitled.

April 2023 Private Placement

On April 26, 2023, the Company completed its sale of a total of 9,735,731 shares of the Company’s common stock, at a purchase price of $0.946 per share, and 22,000,000 Pre-Funded Warrants to purchase common stock, at a purchase price of $0.945 per Pre-Funded Warrant, in a private placement (the “April 2023 Private Placement”) to a select group of accredited investors, pursuant to the Securities Purchase Agreement, dated as of April 23, 2023, by and between the Company and the 2023 Purchasers. The April 2023 Private Placement resulted in net proceeds to the Company of approximately $27.5 million, after deducting underwriting discounts, commissions and offering expenses. The Pre-Funded Warrants are immediately exercisable from the date of issuance and do not have an expiration date. They have an exercise price of $0.001. Holders may not exercise any Pre-Funded Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. The Pre-Funded Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants will be entitled to receive, upon exercise of the Pre-Funded Warrants, the kind and amount of securities, cash or other property that the holders would have

received had they exercised the Pre-Funded Warrants immediately prior to such transaction. The Pre-Funded Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which holders of common stock are entitled.

Leerink ATM Agreement

On August 11, 2023, the Company entered into (the “Leerink ATM Agreement”) with Leerink Partners LLC, pursuant to which the Company may offer and sell, from time to time, shares of common stock having an aggregate offering price of up to $75.0 million through Leerink Partners LLC as sales agent. Under the Leerink ATM Agreement, the sales agents may sell shares of common stock by any method permitted by law deemed to be an “at the market offering” as defined in Rule 415(a)(4) of the Securities Act of 1933, as amended. The Company will pay the sales agent a commission rate of up to 3% of the gross offering proceeds of any shares sold and has agreed to provide the sales agent with indemnification and contribution against certain liabilities. The Leerink ATM Agreement contains customary representations and warranties.

As of September 30, 2024, the Company has sold an aggregate of 20,615,976 shares of the company’s common stock, pursuant to the Leerink ATM Agreement with an average sale price of $3.36 per share, resulting in net proceeds of $49.3 million, after deducting underwriting discounts, commissions and offering expenses. The Company intends to use the net proceeds from the Leerink ATM to fund research and development and registration of its pipeline candidates, and for working capital and general corporate purposes.

Venrock Warrant Exchange

On October 12, 2023, the Company entered into an exchange agreement (the “Exchange Agreement”) with entities affiliated with Venrock Healthcare Capital Partners (the “Exchanging Stockholders”), pursuant to which the Company exchanged an aggregate of 5,658,034 shares of common stock, owned by the Exchanging Stockholders for pre-funded warrants to purchase an aggregate of 5,658,034 shares of common stock (subject to adjustment in the event of stock splits, recapitalizations and other similar events affecting common stock), with an exercise price of $0.001 per share. The common stock exchanged, and the pre-funded warrants issued were recorded at fair market value of $12.9 million.

March 2024 Private Placement

On March 1, 2024, the Company completed its sale of a total of 12,285,714 shares of the Company’s common stock, at a purchase price of $7.00 per share, and 2,000,000 Pre-Funded Warrants to purchase common stock, at a purchase price of $6.999 per Pre-Funded Warrant, in a private placement to a select group of accredited investors (the “March 2024 Private Placement”), pursuant to a Securities Purchase Agreement, dated as of February 27, 2024, by and between the Company and the purchasers named therein (the “2024 Purchasers”). The Private Placement resulted in net proceeds to the Company of approximately $92.3 million, before deducting placement agent commissions and other offering expenses.

- Definition

The entire disclosure for equity.

+ References

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+ Details

Organization and Summary of Significant Accounting Policies

9 Months Ended

Sep. 30, 2024

Organization and Summary of Significant Accounting Policies

Organization and Summary of Significant Accounting Policies

1. ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

Operations and Business

Applied Therapeutics, Inc. (the “Company”) is a clinical-stage biopharmaceutical company developing a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need. In particular, the Company is currently targeting treatments for rare diseases such as Galactosemia and Sorbitol Dehydrogenase (“SORD”) deficiency. The Company was incorporated in Delaware on January 20, 2016 and is headquartered in New York, New York.

The accompanying unaudited condensed financial statements have been prepared by the Company in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”) for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted pursuant to such rules and regulations. These condensed financial statements should be read in conjunction with the Company’s audited financial statements and the notes thereto for the year ended December 31, 2023, included in the Annual Report, filed with the SEC on March 6, 2024 (the “Annual Report”).

The unaudited condensed financial statements have been prepared on the same basis as the audited financial statements. In the opinion of management, the accompanying unaudited condensed financial statements contain all adjustments which are necessary for a fair presentation of the Company’s financial position as of September 30, 2024, results of operations for the three and nine months ended September 30, 2024 and 2023 and cash flows for the nine months ended September 30, 2024 and 2023. Such adjustments are of a normal and recurring nature. The results of operations for the three and nine months ended September 30, 2024, are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2024. Certain reclassifications have been made to prior period financial statements to conform to the current period presentation.

Liquidity and Going Concern

Under ASC Topic 205-40, Presentation of Financial Statements - Going Concern, management is required at each reporting period to evaluate whether there are conditions and events, considered in the aggregate, that raise substantial doubt about an entity's ability to continue as a going concern within one year after the date that the financial statements are issued. As of September 30, 2024, financing through the Company’s March 2024 Private Placement, Leerink ATM Agreement and warrant exercises has resulted in net proceeds of $113.8 million, after deducting placement agent commissions and other offering expenses (see Note 7 and Note 8). The Company continues to evaluate several potential long-term financing options, including equity capital, debt, convertible debt, and synthetic royalty financing. Additionally, the Company is in active dialogue with several potential partners regarding business development opportunities related to one or more of its programs. There can be no assurances that the Company’s discussions with any of the current counterparties will be successful, and the Company expects to continue to pursue additional opportunities.

As reflected in the accompanying financial statements, the Company incurred a net loss of $149.6 million for the nine months ended September 30, 2024, and has an accumulated deficit of $618.2 million as of September 30, 2024. The exclusive licensing agreement with Advanz Pharma for commercialization rights to AT-007 in Europe provides a source of capital to the Company based on clinical and regulatory milestones. The Company received a $10.7 million upfront payment from Advanz Pharma in January 2023 in conjunction with signing the agreement. If actualization of these milestones aligns with the projected timelines, and product approvals are received in the timeframes expected, this source of capital may be sufficient to cover operating expenses through expected product approvals and potential revenues. However, there are no guarantees that this will materialize timely or at all, and delays or unexpected data could disrupt this potential liquidity. Broadly, the Company has not yet established an ongoing source of revenues sufficient to cover its operating costs and is dependent on debt and equity financing to fund its operations. The Company

currently expects that its existing cash and cash equivalents of $98.9 million as of September 30, 2024, will fund its operating expenses and capital requirements for at least twelve months from the date this Quarterly Report on Form 10-Q is issued.

Risks and Uncertainties

The Company is subject to risks common to companies in the biotechnology industry, including but not limited to, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for any product candidate that it may identify and develop, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary technology, compliance with government regulations, development by competitors of technological innovations and reliance on third-party manufacturers.

Use of Estimates

The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities and the Company's ability to continue as a going concern as of the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. In preparing the financial statements, management used significant estimates in the following areas, among others: prepaid and accrued expenses; warrant liabilities valuation; license revenue; research and development services revenue; stock-based compensation expense; the likelihood of realization of deferred tax assets; and the evaluation of the existence of conditions and events that raise substantial doubt regarding the Company’s ability to continue as a going concern. Actual results could differ from those estimates.

Significant Accounting Policies

The significant accounting policies and estimates used in preparation of the condensed financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2023, and the notes thereto, which are included in the Annual Report. There have been no material changes to the Company’s significant accounting policies during the three and nine months ended September 30, 2024.

Recent Accounting Pronouncements

Any recent pronouncements issued by the FASB or other authoritative standards groups with future effective dates are either not applicable or are not expected to be significant to the financial statements of the Company.