0001558370-24-002541 | 10-K | XBRL Viewer

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Stock-based compensation (Details)	12 Months Ended
	Dec. 31, 2023
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
Dividend yield (as a percent)	0.00%

SUBSEQUENT EVENTS (Details) - USD ($) $ / shares in Units, $ in Thousands					1 Months Ended		2 Months Ended	12 Months Ended
	Mar. 01, 2024	Feb. 16, 2024	Jan. 31, 2024	Jun. 08, 2023	Feb. 29, 2024	Jan. 31, 2024	Feb. 29, 2024	Dec. 31, 2023	Dec. 31, 2022
Subsequent event
Proceeds from exercise of warrants								$ 10,250
Common And Pre-Funded Warrants
Subsequent event
Issuance of warrants (in shares)								22,000,000
Warrants exercised (in shares)								14,500,000
Pre-Funded Warrants
Subsequent event
Issuance of warrants (in shares)								22,000,000
Warrants exercised (in shares)								4,250,000
Common Warrants
Subsequent event
Warrants exercised (in shares)								10,250,000
Common Stock
Subsequent event
Stock issued in exercise of warrants (in shares)								10,250,000
Exercise of pre-funded warrants for common stock (in shares)				4,250,000				4,250,000	1,750,000
Leerink ATM Agreement
Subsequent event
Proceeds from stock issuance, net of cash issuance costs								$ 36,900
Leerink ATM Agreement \| Common Stock
Subsequent event
Issuance of common stock (in shares)								17,615,976
Leerink ATM Agreement \| Common Stock \| Average
Subsequent event
Purchase price, common stock (in dollars per share)								$ 2.04
Subsequent Event \| Pre-Funded Warrants
Subsequent event
Warrants exercised (in shares)						1,706,976
Subsequent Event \| Common Warrants
Subsequent event
Warrants exercised (in shares)					4,025,000
Proceeds from exercise of warrants					$ 4,000
Subsequent Event \| Common Stock
Subsequent event
Stock issued in exercise of warrants (in shares)					4,025,000
Exercise of pre-funded warrants for common stock (in shares)						1,706,285
Subsequent Event \| Leerink ATM Agreement
Subsequent event
Proceeds from stock issuance, net of cash issuance costs		$ 9,500	$ 3,000				$ 12,500
Proceeds from exercise of warrants							$ 4,000
Subsequent Event \| Leerink ATM Agreement \| Common Stock
Subsequent event
Issuance of common stock (in shares)		2,000,000	1,000,000
Subsequent Event \| Leerink ATM Agreement \| Common Stock \| Average
Subsequent event
Purchase price, common stock (in dollars per share)		$ 4.90	$ 3.13			$ 3.13
Subsequent Event \| March 2024 Offering, Private Placement
Subsequent event
Proceeds from issuance of shares and warrants, gross	$ 100,000
Subsequent Event \| March 2024 Offering, Private Placement \| Pre-Funded Warrants
Subsequent event
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%
Subsequent Event \| March 2024 Offering, Private Placement \| Common Stock
Subsequent event
Issuance of common stock (in shares)	12,285,714
Purchase price, common stock (in dollars per share)	$ 7.00

LEASES - Lease terms and discount rates (Details)	Dec. 31, 2023	Dec. 31, 2022
LEASES
Weighted-average remaining lease term - operating leases	1 year 1 month 6 days	1 year 9 months 18 days
Weighted-average discount rate - operating leases	6.86%	5.69%

Statements of Comprehensive Loss - USD ($) $ in Thousands	12 Months Ended
Statements of Comprehensive Loss - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Statements of Comprehensive Loss
Net Loss	$ (119,763)	$ (82,508)
Other comprehensive income (loss)
Unrealized gain (loss) on marketable securities	(51)	158
Other comprehensive gain (loss), net of tax	(51)	158
Comprehensive loss, net of tax	$ (119,814)	$ (82,350)

- Definition

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

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+ Details

ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

12 Months Ended

Dec. 31, 2023

ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

6. ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

Accrued expenses and other current liabilities consisted of the following (in thousands):


	December 31,		December 31,
(in thousands)	2023		2022
Accrued pre-clinical and clinical expenses	$	10,142	$	8,877
Short-term insurance financing note		310		622
Deferred revenue		667		—
Accrued professional fees		1,307		1,218
Accrued compensation and benefits		2,066		2,301
Accrued commercial expenses		19		896
Accrued patent expenses		396		361
Other		379		481
Total accrued expenses & other current liabilities	$	15,286	$	14,756

REVENUE - Performance Obligation (Details) - License and Supply Agreement - Advanz Pharma - USD ($) $ in Millions	Dec. 31, 2023	Jan. 03, 2023
Research and development services
Performance obligation
Amount of performance obligation		$ 1.3
Research and development services \| Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date [Axis]: 2024-01-01
Performance obligation
Amount of performance obligation	$ 0.5
Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Period	3 years
Manufacturing and supply material right
Performance obligation
Amount of performance obligation	$ 0.1	0.1
License
Performance obligation
Amount of performance obligation		$ 9.3

RELATED PARTIES (Details) - USD ($)	12 Months Ended
RELATED PARTIES (Details) - USD ($)	Dec. 31, 2023	Dec. 31, 2022
Related Party Transactions
Research and development expense	$ 53,905,000	$ 55,634,000
Accounts payable	1,742,000	4,534,000
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	100,000	100,000
Accounts payable	$ 8,000	$ 0

WARRANTS (Tables)

12 Months Ended

Dec. 31, 2023

WARRANTS

Schedule of warrants outstanding


	Equity Classified		Liability Classified		Total
	Warrants		Warrants		Warrants
Outstanding as of January 1, 2023		8,375,618		30,000,000		38,375,618
Warrants granted and issued		22,000,000		—		22,000,000
Warrants exercised		(4,250,000)		(10,250,000)		(14,500,000)
Common stock exchanged for prefunded warrants		5,658,034		—		5,658,034
Outstanding as of December 31, 2023		31,783,652		19,750,000		51,533,652

STOCK-BASED COMPENSATION - 2019 Employee Stock Purchase Plan (Details) - 2019 ESPP - shares	1 Months Ended	12 Months Ended
	May 31, 2019	Dec. 31, 2023
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.

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-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(2)(iii)(03)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 20: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (d)(1)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 21: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (d)(2)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 22: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (e)(1)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 23: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (e)(2)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 24: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (f)(2)(i)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 25: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (f)(2)(ii)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 26: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (f)(2)(iii)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 27: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (f)(2)(iv)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 28: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (f)(2)(v)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

+ Details

FAIR VALUE MEASUREMENTS - Warrants (Details) - shares		12 Months Ended
FAIR VALUE MEASUREMENTS - Warrants (Details) - shares	Jun. 27, 2022	Dec. 31, 2023
Common And Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)		22,000,000
Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)		22,000,000
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

PREPAID EXPENSES AND OTHER CURRENT ASSETS (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
PREPAID EXPENSES AND OTHER CURRENT ASSETS
Prepaid research and development expenses	$ 2,880	$ 4,272
Insurance premium asset	566	1,131
Prepaid rent expenses	47	99
Prepaid insurance expenses	72	71
Prepaid commercial and patient advocacy	284	206
Research and development tax credit receivable	262	252
Interest receivable	23	23
Other prepaid expenses and current assets	100	617
Total prepaid expenses & other current assets	$ 4,234	$ 6,671

STOCK-BASED COMPENSATION - Options activity (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Options Outstanding
Outstanding at beginning of period (in shares)	4,874,047
Options granted (in shares)	0
Options exercised (in shares)	(85,619)
Forfeited (in shares)	(84,639)
Expired (in shares)	(8,170)
Outstanding at end of period (in shares)	4,695,619	4,874,047
Exercisable at end of period (in shares)	4,334,312
Nonvested at end of period (in shares)	361,307
Weighted-Average Exercise Price
Outstanding at beginning of period (in dollars per share)	$ 1.97
Options exercised (in dollars per share)	1.05
Options forfeited (in dollars per share)	1.05
Options expired (in dollars per share)	1.05
Outstanding at end of period (in dollars per share)	2.01	$ 1.97
Exercisable at end of period (in dollars per share)	2.09
Nonvested at end of period (in dollars per share)	$ 1.05
Weighted-Average Remaining Contractual Term-in years
Outstanding	5 years 10 months 24 days	6 years 10 months 24 days
Exercisable	5 years 8 months 12 days
Nonvested	7 years 8 months 12 days
Aggregate Intrinsic Value
Options exercised (in dollars)	$ 100	$ 0
Outstanding (in dollars)	7,875
Exercisable (in dollars)	7,044
Nonvested (in dollars)	831
Grant-date fair value of stock options vested during the year	$ 400	$ 1,400

LEASES - Maturities of Lease Liabilities (Details) $ in Thousands	Dec. 31, 2023 USD ($)
LEASES
2024	$ 446
2025	23
Thereafter	20
Total lease payments	489
Less: interest	22
Total lease liabilities	$ 467
Operating Lease, Liability, Statement of Financial Position	Current portion of operating lease liabilities, Noncurrent portion of operating lease liabilities

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Clinical hold-back, long-term (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2023 USD ($)
Agreements
Clinical holdback, Balance as of beginning of period	$ 464
Clinical holdback retained	295
Clinical holdback, Balance as of end of period	$ 759
Minimum
Agreements
Clinical holdback, Invoiced costs withheld from payment (as a percent)	5.00%
Maximum
Agreements
Clinical holdback, Invoiced costs withheld from payment (as a percent)	10.00%

STOCKHOLDERS' EQUITY - June 2022 Offering (Details) - USD ($) $ / shares in Units, $ in Millions		12 Months Ended
	Jun. 27, 2022	Dec. 31, 2023
Common And Pre-Funded Warrants
Equity
Issuance of warrants (in shares)		22,000,000
Pre-Funded Warrants
Equity
Issuance of warrants (in shares)		22,000,000
June 2022 Offering
Equity
Offering price per share and accompanying warrant (in dollars per share)	$ 1.00
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
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

STOCK-BASED COMPENSATION - RSUs (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
STOCK-BASED COMPENSATION
Stock-based compensation expense	$ 7,360	$ 9,603
Restricted stock units
STOCK-BASED COMPENSATION
Stock-based compensation expense	2,400	$ 1,300
Unamortized stock-based compensation costs for unvested awards	$ 5,500
Summary of restricted stock units outstanding
Outstanding at beginning of period ( in shares)	815,509
Awarded (in shares)	6,814,362
Released (in shares)	(527,182)
Forfeited (in shares)	(185,267)
Outstanding at end of period ( in shares)	6,917,422	815,509
Nonvested at end of period (in shares)	6,917,422
Weighted-Average Grant Date Fair Value
Outstanding at beginning of period (in dollars per share)	$ 5.96
Awarded (in dollars per share)	2.09
Released (in dollars per share)	4.40
Forfeited (in dollars per share)	2.81
Outstanding at end of period (in dollars per share)	2.35	$ 5.96
Nonvested at end of period (in dollars per share)	$ 2.35
Weighted Average Remaining Recognition Period (in years)	3 years 3 months 18 days

STOCK-BASED COMPENSATION - Stock-based compensation expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Stock-based compensation
Total stock-based compensation expense	$ 7,360	$ 9,603
Research and development
Stock-based compensation
Total stock-based compensation expense	3,005	3,839
General and administrative
Stock-based compensation
Total stock-based compensation expense	$ 4,355	$ 5,764

STOCK-BASED COMPENSATION

12 Months Ended

Dec. 31, 2023

STOCK-BASED COMPENSATION

STOCK-BASED COMPENSATION

7. 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 is 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 shall be 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.

On August 2, 2022, the Board took action in accordance with its authority under the terms of the 2019 Plan to reset the per-share exercise price of all stock options previously granted under the 2019 Plan to $1.05 per share (the "Options Repricing"), which is equal to the closing price of a share of the Company’s common stock on August 1, 2022. The Options Repricing was deemed to be a Type I modification event under ASC 718, Compensation-Stock Compensation. No other terms of the repriced stock options were modified, and the repriced stock options will continue to vest according to their original vesting schedules and will retain their original expiration dates. As a result of the Options Repricing, 1,797,517 vested and 1,380,917 unvested stock options outstanding as of August 2, 2022, with

original exercise prices ranging from $1.22 to $49.60, were repriced. The Options Repricing resulted in incremental stock-based compensation expense of $1.4 million, of which $0.9 million related to vested stock option awards and was expensed on the repricing date, and $0.5 million of which related to unvested stock option awards and is being amortized on a ratable basis over the remaining weighted-average vesting period of those awards being approximately 2.4 years.

As of December 31, 2023, there were no shares of common stock available for issuance under the 2019 Plan. On January 1, 2024, there were 4,243,292, shares that became issuable for future grants subject to the adjustments provided in the 2019 Plan.

Stock-Based Compensation Expense

Total stock-based compensation expense recorded for employees, directors and non-employees (in thousands):


	Year Ended
	December 31,
(in thousands)	2023		2022
Research and development	$	3,005	$	3,839
General and administrative		4,355		5,764
Total stock-based compensation expense	$	7,360	$	9,603

Stock Option Activity

During the year ended December 31, 2023, the Company did not grant any stock options. For the years ended December 31, 2023 and 2022, amortization of stock compensation of options amounted to $5.0 million and $7.8 million, respectively. As of December 31, 2023, the total unrecognized stock-based compensation balance for unvested options was $2.6 million, which is expected to be recognized over 1.7 years. The weighted-average fair value of options granted during the year ended December 31, 2022 was $1.79 per share.

The following table summarizes the information about options outstanding at December 31, 2023:


				Weighted-Average
		Weighted-		Remaining	Aggregate
	Options	Average		Contractual	Intrinsic
(in thousands, except for share data)	Outstanding	Exercise Price		Term (in years)	Value
Outstanding at December 31, 2022	4,874,047	$	1.97	6.9	$	—
Options granted	—		—
Options exercised	(85,619)		1.05
Forfeited	(84,639)		1.05
Expired	(8,170)		1.05
Outstanding at December 31, 2023	4,695,619	$	2.01	5.9	$	7,875

Exercisable at December 31, 2023	4,334,312	$	2.09	5.7	$	7,044
Nonvested at December 31, 2023	361,307	$	1.05	7.7	$	831

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 December 31, 2023. The intrinsic value of stock options exercised during the year ended December 31, 2023 was $0.1 million. There was no intrinsic value on stock options exercised during the year ended December 31, 2022. The total grant-date fair value of stock options vested during the years ended December 31, 2023 and December 31, 2022 was $0.4 million and $1.4 million, respectively.

Valuation of Stock Options Granted to Employees that Contain Service Conditions Only

The fair value of each option award granted with service-based vesting is estimated on the date of the grant using the Black-Scholes option valuation model based on the weighted average assumptions noted in the table below for those options granted in the years ended December 31, 2023 and 2022.


	Year Ended
	December 31,
	2023			2022
Expected term (in years)		—			5.7
Volatility		—	%		75.83	%
Risk-free interest rate		—	%		2.13	%
Dividend yield		—	%		—	%

Restricted Stock Unit Activity

During the year ended December 31, 2023, the Company granted 6,814,362 restricted stock units (“RSUs”). For the years ended December 31, 2023 and 2022 amortization of stock compensation of RSUs amounted to $2.4 million and $1.3 million, respectively. As of December 31, 2023, the unamortized compensation costs associated with non-vested restricted stock awards was $5.5 million with a weighted-average remaining amortization period of 3.3 years.

The following table summarizes the information about restricted stock units outstanding at December 31, 2023:


		Weighted-Average
		Grant Date
(in thousands, except for share data)	Shares	Fair Value
Outstanding at December 31, 2022	815,509	$	5.96
Awarded	6,814,362		2.09
Released	(527,182)		4.40
Forfeited	(185,267)		2.81
Outstanding at December 31, 2023	6,917,422	$	2.35
Nonvested at December 31, 2023	6,917,422	$	2.35

Weighted Average Remaining Recognition Period (in years)	3.3

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, in each case subject to the approval of the Board, 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 December 31, 2023, no shares of common stock had been issued under the ESPP. The first offering period has not yet been decided by the Board.

FAIR VALUE MEASUREMENTS - Transfers (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
FAIR VALUE MEASUREMENTS
Assets, transfer from level 1 to level 2	$ 0	$ 0
Assets, transfer from level 2 to level 1	$ 0	$ 0

RELATED PARTIES

12 Months Ended

Dec. 31, 2023

RELATED PARTIES

RELATED PARTIES

14. 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 years ended December 31, 2023 and 2022, the Company made payments to Alexandria LaunchLabs of approximately $0.1 million under the LaunchLabs Agreement, which was recognized in research and development expenses. As of December 31, 2023 and 2022, there was $8,000 and $0 due to Alexandria LaunchLabs under the LaunchLabs Agreement, respectively, recorded in accounts payable.

- 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
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480990/946-20-50-2

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 5
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480990/946-20-50-5

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480990/946-20-50-6

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 235
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481062/946-235-50-2

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 235
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481062/946-235-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483326/850-10-50-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.6-07(2)(g)(3))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483575/946-220-S99-1

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.6-07(2)(c))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483575/946-220-S99-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.6-07(2)(e))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483575/946-220-S99-1

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org//850/tableOfContent

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483326/850-10-50-6

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483326/850-10-50-1

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483326/850-10-50-1

+ Details

Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
Statements of Cash Flows - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
OPERATING ACTIVITIES:
Net loss	$ (119,763)	$ (82,508)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation expense	7,360	9,603
Amortization of insurance premium	2,111	3,589
Amortization of operating lease right-of-use assets	353	441
Amortization of leasehold improvements	1	2
Change in operating lease liability	(367)	(442)
Change in fair value of warrant liabilities	56,573	66
Changes in operating assets and liabilities:
Financed insurance premium	(1,546)	(3,105)
Prepaid expenses	1,872	359
Accounts payable	(2,792)	(4,927)
Accrued expenses and other current liabilities	730	(1,635)
Other liabilities	295	464
Net cash used in operating activities	(55,173)	(78,093)
INVESTING ACTIVITIES:
Purchase of available-for-sale securities		(64,218)
Proceeds from sale of available-for-sale securities	4,944
Proceeds from maturities of available-for-sale securities	8,928	77,388
Net cash provided by investing activities	13,872	13,170
FINANCING ACTIVITIES:
Proceeds from issuance of common stock, pre-funded warrants and reissuance of treasury stock, net of issuance costs	64,514	27,811
Proceeds from financed insurance premium	1,546	3,105
Repayments of short-term borrowings	(1,858)	(3,273)
Exercise of stock options for common stock under Equity Incentive Plan	90	49
Exercise of Warrants	10,250
Net cash provided by financing activities	74,542	27,692
NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS	33,241	(37,231)
Cash and cash equivalents at beginning of period	16,657	53,888
Cash and cash equivalents at end of period	49,898	16,657
SUPPLEMENTAL DISCLOSURE OF CASH FLOW INFORMATION:
Operating lease right-of-use asset obtained in exchange for operating lease liabilities	(57)	21,835
Offering costs in accrued expenses	112
Conversion of warrant liabilities to equity for warrant exercises	16,505	1,417
Unrealized gain (loss) on marketable securities	$ (51)	158
Reclassification of prefunded warrant liability to equity		$ 6,827

ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES
Accrued pre-clinical and clinical expenses	$ 10,142	$ 8,877
Short-term insurance financing note	310	622
Deferred revenue	667
Accrued professional fees	1,307	1,218
Accrued compensation and benefits	2,066	2,301
Accrued commercial expenses	19	896
Accrued patent expenses	396	361
Other	379	481
Total accrued expenses & other current liabilities	$ 15,286	$ 14,756

INCOME TAXES (Tables)

12 Months Ended

Dec. 31, 2023

INCOME TAXES

Schedule of reconciliation of deferred taxes

A reconciliation of the Company's deferred taxes is as follows (in thousands):


	Year ended
	December 31,
	2023		2022
Deferred Tax Assets
Operating lease liability	$	146	$	282
Stock-based compensation		4,060		8,131
License fee		501		554
Net operating loss carryforwards		94,119		88,090
Research and development tax credits		25,145		21,397
Capitalized research and development expense		26,945		14,910
Other		445		1,085
Total deferred tax assets	$	151,361	$	134,449
Valuation allowance	$	(151,222)	$	(134,177)
Net deferred tax assets	$	139	$	272
Deferred tax liabilities
Right of use asset		(139)		(272)
Net deferred tax liabilities	$	(139)	$	(272)
Net deferred tax asset (liability)	$	—	$	—

Schedule of the U.S. federal statutory corporate tax rate reconciles to the Company's effective tax rate


	Year Ended
	December 31,
	2023		2022
Federal statutory rate	21.0	%	21.0	%
State and local taxes net of federal tax benefit	3.4		10.2
Credits	3.1		7.8
Permanent differences	(0.1)		(0.1)
Change in fair value of warrant liabilities	(9.9)		—
Change in valuation allowance	(14.2)		(37.9)
Stock-based compensation	(3.3)		(1.1)
Other	—		0.1
Total	0.0	%	0.0	%

INCOME TAXES - Provision (Details) - USD ($) $ in Thousands	12 Months Ended
INCOME TAXES - Provision (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
INCOME TAXES
Current tax provision	$ 0	$ 0
Deferred tax provision	$ 0	$ 0

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

12 Months Ended

Dec. 31, 2023

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 metabolic diseases such as Galactosemia, Sorbitol Dehydrogenase (“SORD”) deficiency, and diabetic complications including diabetic cardiomyopathy. The Company was incorporated in Delaware on January 20, 2016 and is headquartered in New York, New York.

Basis of Presentation

The Company’s fiscal year ends on December 31 and its first three quarters end on March 31, June 30 and September 30. The accompanying financial statements are prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Updates (“ASU”) of the Financial Accounting Standards Board (“FASB”).

The Company is an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act of 2012 (the “JOBS Act”). Section 107(b) of the JOBS Act provides that an emerging growth company can take advantage of an extended transition period for complying with new or revised accounting standards. Thus, an emerging growth company can delay the adoption of certain accounting standards until those standards would otherwise apply to private companies. The Company has irrevocably elected not to avail itself of this extended transition period, and, as a result, the Company will adopt new or revised accounting standards on the relevant dates on which adoption of such standards is required for other public companies.

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 December 31, 2023, financing through the Company’s April 2023 Private Placement, Leerink ATM Agreement and warrant exercises has resulted in net proceeds of $74.7 million, after deducting placement agent commissions and other offering expenses (see Note 8 and Note 9). Additionally, in January and February 2024, the Company issued additional shares under the Leerink ATM Agreement that resulted in net proceeds of $12.5 million, after deducting placement agent commissions and other offering expenses (see Note 16), and $4.0 million from the exercise of common warrants. Further, on March 1, 2024, we completed our March 2024 Private Placement (as defined herein) which resulted in gross proceeds of approximately $100.0 million, before deducting placement agent commissions and other offering expenses. The Company continues to actively pursue 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 $119.8 million for the year ended December 31, 2023 and has an accumulated deficit of $468.6 million as of December 31, 2023. 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 $49.9 million as of December 31, 2023 and $116.5 million received from the Leerink ATM, warrant exercises and private placement in January, February and March 2024, will fund its operating expenses and capital requirements for at least twelve months from the date of these audited financial statements are 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 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

Fair Value Measurements

Certain assets and liabilities are reported on a recurring basis at fair value. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable:

Level 1—Quoted prices in active markets for identical assets or liabilities.

Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data.

Level 3—Unobservable inputs that are supported by little or no market activity and that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques.

Cash and Cash Equivalents

The Company considers all short-term, highly liquid investments, with an original maturity of three months or less, to be cash equivalents. The Company maintains its cash in bank deposit accounts which, at times, may exceed federally insured limits. The Company has not experienced any losses in these accounts and does not believe it is exposed to any significant credit risk on cash and cash equivalents.

Investments

The Company determines the appropriate classification of its investments at the date of purchase and reevaluates the classifications at the balance sheet date. Marketable debt securities with maturities of 12 months or less are classified as short-term. Marketable debt securities with maturities greater than 12 months are classified as long-term. The Company’s marketable securities are accounted for as available for sale (“AFS”). AFS securities are reported at fair value. Unrealized gains and losses, after applicable income taxes, are reported in accumulated other comprehensive income/(loss). Realized gains or losses on the sale of marketable securities are determined using the specific identification method and are recorded as a component of other income (expense), net.

The Company conducts an other-than-temporary impairment (“OTTI”) analysis on a quarterly basis or more often if a potential loss-triggering event occurs. The Company considers factors such as the duration, severity and the reason for the decline in value, the potential recovery period and whether the Company intends to sell. For AFS securities, the Company also considers whether (i) it is more likely than not that the Company will be required to sell the debt securities before recovery of their amortized cost basis and (ii) the amortized cost basis cannot be recovered as a result of credit losses. As of December 31, 2023, the Company did not have an investment portfolio.

Leases

At the inception of an arrangement, the Company determines if an arrangement is, or contains, a lease based on the unique facts and circumstances present in that arrangement. Lease classification, recognition, and measurement are then determined at the lease commencement date. For arrangements that contain a lease the Company (i) identifies lease and non-lease components, (ii) determines the consideration in the contract, (iii) determines whether the lease is an operating or financing lease; and (iv) recognizes lease right-of-use (“ROU”) assets and liabilities. Lease liabilities and their corresponding ROU assets are recorded based on the present value of lease payments over the expected lease term. The interest rate implicit in lease contracts is typically not readily determinable and as such, the Company uses its incremental borrowing rate based on the information available at the lease commencement date, which represents an internally developed rate that would be incurred to borrow, on a collateralized basis, over a similar term, an amount equal to the lease payments in a similar economic environment.

Most leases include options to renew and, or, terminate the lease, which can impact the lease term. The exercise of these options is at the Company’s discretion and the Company does not include any of these options within the expected lease term as the Company is not reasonably certain it will exercise these options. The Company has elected to combine lease components (for example fixed payments including rent) with non-lease components (for example, non-dedicated parking and common-area maintenance costs) on our real estate asset classes.

Fixed, or in substance fixed, lease payments on operating leases are recognized over the expected term of the lease on a straight-line basis. Fixed lease expense on operating leases is recognized within operating expenses within the statements of operations. The Company has operating leases for our corporate offices. The Company has elected the short-term lease exemption and, therefore, do not recognize a ROU asset or corresponding liability for lease arrangements with an original term of 12 months or less. Leasehold improvements and assets under financing lease arrangements are amortized over the lesser of the asset’s estimated useful life or the term of the respective lease. Maintenance costs are expensed as incurred.

Operating leases are included in operating lease right-of-use asset, current portion of operating lease liabilities, and noncurrent portion of operating lease liabilities in the balance sheet as of December 31, 2023 and 2022.

Clinical hold-back, long-term

As part of the regulatory approval process for taking its products to market, the Company enters into certain Clinical Trial Agreements (CTAs) which include, among other things, the compensation and payment schedule the participating medical institutions and physicians will receive for all costs in connection with the clinical trial (or study) under the terms of the CTA. As individual patients are enrolled in the study by the participating medical institution or

physician, the Company pays certain per study fees according to the CTA for the duration of the trial. As invoices are received by the Company from the medical institution or physician, the Company retains an agreed upon percentage of total invoiced costs, generally ranging between 5% - 10%, that is withheld from payment until the end of the study.

These retained amounts are recorded as clinical holdback, a liability, on the accompanying balance sheets, and all expenses incurred in connection with these CTA activities are expensed as services are provided, which are included as research and development expenses on the accompanying statements of operations.

The following table shows the activity within the clinical holdback liability accounts for the year ended December 31, 2023:



(in thousands)
Balance as of December 31, 2022	$	464
Clinical holdback retained		295
Clinical holdback paid		—
Balance as of December 31, 2023	$	759

Deferred Offering Costs

The Company capitalizes certain legal, professional accounting and other third-party fees that are directly associated with in-process equity financings as deferred offering costs until such financings are consummated. After consummation of the equity financing, these costs are recorded in the statement of stockholders’ (deficit) equity as a reduction of proceeds generated as a result of the offering. Should a planned equity financing be abandoned, the deferred offering costs would be expensed immediately as a charge to operating expenses in the statement of operations.

As of December 31, 2023 and 2022, the Company did not have any deferred offering costs recorded.

Warrant Liabilities

The Company accounts for common warrant liabilities by measuring the fair value at inception and subsequently remeasures on a recurring basis, with changes in fair value recognized in other income (expense) within the Company’s statement of operations.

The Company utilized a Black-Scholes option pricing model to estimate the fair value of the warrant liabilities, which utilizes certain unobservable inputs and is therefore considered a Level 3 fair value measurement. Certain inputs used in the 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 consolidated statement of operations.

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

Treasury Stock

The Company records treasury stock activities under the cost method. Treasury stock is included in authorized and issued shares but excluded from outstanding shares. The re-issuance of treasury stock is accounted for on a first in, first-out basis and any differences between the cost of treasury stock and the re-issuance proceeds are charged or credited to additional paid-in capital.

Revenue Recognition

Under ASU 2014-09, Revenue from Contracts with Customers (Topic 606), or ASC 606, as amended by ASU 2016-08, 2016-10, 2016-12 and 2016-20, the Company recognizes revenue in an amount that reflects the consideration to which it expects to be entitled in exchange for the transfer of promised goods or services to customers. To determine revenue recognition for contracts with customers that are within the scope of ASC 606, the Company performs the following steps: (1) identifies the contract with the customer, (2) identifies the performance obligations in the contract, (3) determines the transaction price, (4) allocates the transaction price to the performance obligations in the contract, and (5) recognizes revenue when (or as) the entity satisfies a performance obligation.

The Company has entered into an agreement to license its intellectual property, or IP, related to Galactosemia and SORD to develop, manufacture and/or commercialize drug products with Mercury Pharma Group Limited, trading as Advanz Pharma Holdings, (“Advanz Pharma”). The agreement contains multiple performance obligations, including licenses of IP, research and development services, and the manufacturing and supply material right. Payments to the Company under this agreement may include nonrefundable fees, payments for research activities, payments based upon the achievement of certain milestones and royalties on any resulting net product sales.

The Company identifies agreements as contracts that create enforceable rights and obligations when the agreement is approved by the parties, the Company can identify the rights of the parties and the payment terms, the contract has commercial substance and it is probable that the Company will collect substantially all of the consideration to which it will be entitled in exchange for the goods and services that will be transferred to the customer. The counterparty is considered to be a customer when it has contracted with the Company to obtain goods and services that are the output of the Company’s ordinary activities (i.e., development of pharmaceutical products) in exchange for consideration.

A performance obligation is a promise to transfer distinct goods or services to a customer. Performance obligations that are both capable of being distinct and distinct within the context of the contract are considered to be separate performance obligations. Performance obligations are capable of being distinct if the counterparty is able to benefit from the good or service on its own or together with other resources that are readily available to it. Performance obligations are distinct within the context of the contract when each performance obligation is separately identifiable. In assessing whether the Company’s promises to transfer goods or services to the customer are separately identifiable, the Company considers factors in ASC 606, including whether the Company uses the goods or services as inputs to produce or deliver a combined output or outputs specified by the customer, whether one or more of the goods or services significantly modify or customize one of the other goods or services in the contract, and whether the goods or services are highly interdependent or highly interrelated. If a promised good or service is not distinct, it is combined with other promised goods or services until the Company identifies a bundle of goods or services that is distinct, which is accounted for as a single performance obligation. The determination of whether promised goods or services in a contract are distinct may require significant judgment.

The transaction price is the amount of consideration that the Company expects to be entitled to in exchange for transferring promised goods or services to the customer based on the contract terms at inception of a contract. The Company estimates an amount of variable consideration, if any, by using either the expected value or the most likely amount method depending on which method the Company expects to better predict the amount of consideration to which it will be entitled, and only to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur in the future when the uncertainty is resolved. The determination of whether it is probable that a significant reversal of revenue will occur in the future depends on the likelihood and magnitude of the reversal. Factors that could increase the likelihood or magnitude of a reversal of revenue include (a) the susceptibility of the amount of consideration to factors outside the entity’s influence, such as the outcome of clinical trials, the timing of initiation of clinical trials by the counterparty and the approval of drug product candidates by regulatory agencies, (b) situations in which the uncertainty is not expected to be resolved for a long period of time, and (c) the level of the Company’s predictive experience in the field with similar types of contracts. At the end of each reporting period, the Company will update the estimated transaction price and allocate the updated consideration to the separate performance obligations in the same manner as described below.

At inception of a contract, the Company allocates the transaction price to the distinct performance obligations based upon their relative standalone selling prices. Standalone selling price is the price at which an entity would sell a promised good or service separately to a customer. The best evidence of standalone selling price is an observable price of a good or service when sold separately by an entity in similar circumstances to similar customers. Since the Company typically does not have such evidence, it estimates standalone selling price so that the amount that is allocated to each performance obligation equals the amount that the Company expects to receive for transferring the promised goods or services. The methods that the Company uses to make such estimates include (1) the adjusted market assessment approach, under which the Company forecasts and analyzes Galactosemia and SORD sales in the appropriate market, the phase of clinical development as well as considering recent similar license arrangements within the same phase of clinical development, therapeutic area, type of agreement, etc. and (2) the expected cost of satisfying the performance obligations plus a margin, or the expected cost plus a margin approach.

The Company recognizes revenue when, or as, it satisfies a performance obligation by transferring a promised good or service to a customer and the customer obtains control of the good or service. Revenue related to the grant of a license of functional IP that is distinct from the other promised goods or services in the contract and therefore represents a performance obligation is recognized at the point in time that the Company has the right to payment for the license, the customer has legal title to the license and can direct the use of the license (for example, to grant sublicenses) to benefit from its right to use the intellectual property, the customer has the significant risks and rewards of ownership of the license and the customer has accepted the asset (license) by signing the license agreement.

Recognition of revenue related to research and development services that are a distinct performance obligation is deferred at inception of a contract and is recognized as the services are performed to satisfy the performance obligation based on the costs incurred as a percentage of the estimated total costs to be incurred to satisfy the performance obligation. The Agreement requires that Advanz Pharma receives regular updates and data on any research and development services as the services are performed. In accordance with ASC 606-10-25-27, the Company determined that Advanz Pharma simultaneously benefits from the research and development services that are satisfied over time. The Company believes this method most faithfully depicts its performance in transferring the promised services while research and development services are ongoing.

Milestone payments are considered to be variable consideration and are not included in the transaction price at inception of the contract if it is uncertain that the milestone will be achieved. Rather, when it becomes probable that the milestone will be achieved and, therefore, there will not be a significant reversal of revenue in future periods, the respective amount to be earned is included in the transaction price, allocated to the distinct performance obligations based on their relative standalone selling price and recognized as revenue, as described above. Sales based milestones and sales-based royalty payments related to a license of IP are recognized as revenue when the respective sales occur.

Research and Development

The Company expenses all costs incurred in performing research and development activities. Research and development expenses include salaries and other related costs, materials and supplies, preclinical expenses, manufacturing expenses, contract services and other outside expenses. As part of the process of preparing the financial statements, the Company is required to estimate accrued research and development expenses. The Company makes estimates of the accrued expenses as of each balance sheet date in the financial statements based on facts and circumstances known at that time. In addition, there may be instances in which payments made to the Company’s vendors will exceed the level of services provided and result in a prepayment of the expense in which case such amounts are reflected as prepaid expenses and other current assets. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company adjusts the accrual or the amount of prepaid expenses accordingly. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized in prepaid expenses and other current assets. The capitalized amounts are expensed as the related goods are delivered or the services are performed.

Research and development costs also include costs incurred in connection with certain licensing arrangements. Before a compound receives regulatory approval, the Company records upfront and milestone payments made to third

parties under licensing arrangements as expense. Upfront payments are recorded when incurred, and milestone payments are recorded when the specific milestone has been achieved. Once a compound receives regulatory approval, the Company will record any milestone payments in identifiable intangible assets, less accumulated amortization and, unless the asset is determined to have an indefinite life, the Company will amortize the payments on a straight-line basis over the remaining agreement term or the expected product life cycle, whichever is shorter.

General and Administrative

General and administrative expenses consist primarily of salaries and other related costs, including stock-based compensation, for personnel in the Company’s executive and finance functions. General and administrative expenses also include professional fees for legal, accounting, auditing, tax and consulting services; travel expenses; and facility-related expenses, which include allocated expenses for rent and maintenance of facilities and other operating costs.

Commercial expenses consist of payroll expense for commercial personnel, as well as marketing, market research, market access, and other focused investments to support launch of drug candidates, generate evidence of commercial potential and value proposition, and maximize potential business development deal leverage. Commercial expenses are included in general and administrative expenses.

Stock-Based Compensation

The Company accounts for its stock-based compensation as expense in the statements of operations based on the awards’ grant date fair values. The Company accounts for forfeitures as they occur by reversing any expense recognized for unvested awards.

Stock-Based Compensation–Stock Options

The Company estimates the fair value of options granted using the Black-Scholes option pricing model. The Black-Scholes option pricing model requires inputs based on certain subjective assumptions, including (a) the expected stock price volatility, (b) the calculation of expected term of the award, (c) the risk-free interest rate and (d) expected dividends. Due to a lack of company-specific historical and implied volatility data, the Company has based its estimate of expected volatility on the historical volatility of a group of similar companies that are publicly traded. The historical volatility is calculated based on a period of time commensurate with the expected term assumption. The computation of expected volatility is based on the historical volatility of a representative group of companies with similar characteristics to the Company, including stage of product development and life science industry focus. The Company uses the simplified method as allowed by the Securities and Exchange Commission (“SEC”) Staff Accounting Bulletin (“SAB”) No. 107, Share-Based Payment, to calculate the expected term for options granted to employees as it does not have sufficient historical exercise data to provide a reasonable basis upon which to estimate the expected term. The risk-free interest rate is based on a treasury instrument whose term is consistent with the expected term of the stock options. The expected dividend yield is assumed to be zero as the Company has never paid dividends and has no current plans to pay any dividends on its common stock.

The fair value of stock-based payments is recognized as expense over the requisite service period which is generally the vesting period.

Stock-Based Compensation–Restricted Stock Units

The Company accounts for restricted stock units in accordance with the authoritative guidance for stock-based compensation. The fair value of restricted stock units is measured at the grant date based on the closing market price of the Company’s common stock on the date of grant and is recognized as expense on a straight-line basis over the period of vesting. Forfeitures are recognized as a reduction of stock-based compensation expense as they occur.

Income Taxes

The Company uses the asset and liability method of accounting for deferred income taxes. Under this method, deferred tax assets and liabilities are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities at currently enacted tax rates. These temporary differences primarily relate to net operating loss carryforwards available to offset future taxable income. Valuation allowances are established, if necessary, to reduce a deferred tax asset to the amount that will more likely than not be realized.

The Company recognizes tax liabilities from an uncertain tax position only if it is more likely than not that the tax position will not be sustained upon examination by the taxing authorities, based on the technical merits of the tax position. There are no uncertain tax positions that have been recognized in the accompanying financial statements. The Company is required to file tax returns in the U.S. federal jurisdiction and in the state of New York. The Company’s policy is to recognize interest and penalties related to uncertain tax benefits, if any, as part of income tax expense. No such interest and penalties have been accrued as of December 31, 2023 and 2022.

Net Loss per Share

Basic net loss per share is calculated by dividing net loss available to common stockholders by the weighted-average common stock outstanding. Diluted net loss per share is calculated similarly, except that it includes the dilutive effect of the assumed exercise of securities, including outstanding warrants and the effect of shares issuable under the Company’s stock-based compensation plan, if such effect is dilutive.

Segment Information

Operating segments are defined as components of an enterprise for which separate discrete information is available for evaluation by the chief operating decision-maker in deciding how to allocate resources and assess performance. The Company and the Company’s chief operating decision-maker, the Company’s chief executive officer, views the Company’s operations and manages its business as a single operating segment, which is the business of discovering and developing its product candidates.

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.

FAIR VALUE MEASUREMENTS

12 Months Ended

Dec. 31, 2023

FAIR VALUE MEASUREMENTS

FAIR VALUE MEASUREMENTS

3. FAIR VALUE MEASUREMENTS

The following tables summarize, as of December 31, 2023 and 2022, 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 section.


	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


	As of December 31, 2022
(in thousands)	Level 1		Level 2		Level 3		Total
Cash	$	1,337	$	—	$	—	$	1,337
Money market funds		15,320		—		—		15,320
Total cash and cash equivalents	$	16,657	$	—	$	—	$	16,657
U.S. government agency debt securities		—		13,923		—		13,923
Total marketable securities	$	—	$	13,923	$	—	$	13,923
Total financial assets measured at fair value on a recurring basis	$	16,657	$	13,923	$	—	$	30,580
Warrant liabilities - Common Warrants		—		—		13,657		13,657
Total financial liabilities measured at fair value on a recurring basis	$	—	$	—	$	13,657	$	13,657

Investments in U.S. government agency debt securities have been classified as Level 2 as they are valued using quoted prices in less active markets or other directly or indirectly observable inputs. Fair values of U.S. government agency debt securities were derived from a consensus or weighted average price based on input of market prices from multiple sources at each reporting period. During the period ended December 31, 2023 and 2022, there were no transfers of financial assets between Level 1 and Level 2.

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 1 and Note 8 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 consolidated statement of operations.

The Common Warrants were remeasured using a Black-Scholes option pricing model with a range of assumptions included below as of December 31, 2023 and 2022. The Pre-Funded warrants were remeasured and

reclassified to equity using the Black-Scholes option pricing model with a range of assumptions included below as of December 31, 2022:


	December 31,		December 31,
	2023		2022
Expected term (in years)	2.4		3.7
Volatility	109.46	%	91.53	%
Risk-free interest rate	4.32	%	4.11	%
Dividend yield	0.00	%	0.00	%

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.

As of December 31, 2022, 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 10.0% probability of change in control was used for each of the remaining years 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, 2023	$	13,657
Warrants exercised		(16,505)
Change in fair value		56,573
Balance as of December 31, 2023	$	53,725

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.

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables)

12 Months Ended

Dec. 31, 2023

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

Schedule of activity within the clinical holdback liability accounts



(in thousands)
Balance as of December 31, 2022	$	464
Clinical holdback retained		295
Clinical holdback paid		—
Balance as of December 31, 2023	$	759

LEASES (Tables)

12 Months Ended

Dec. 31, 2023

LEASES

Summary of lease related costs


		Year Ended
(in thousands)		December 31,
Lease Cost	Statement of Operations Location	2023		2022
Operating Lease Cost	General and administrative	$	505	$	489
Total lease cost		$	505	$	489

Summary of lease terms and discount rates


	Year Ended
	December 31,		December 31,
Other Information	2023		2022
Weighted-average remaining lease term
Operating leases		1.1 years		1.8 years
Weighted-average discount rate
Operating leases		6.86%		5.69%

Summary of maturities of lease liabilities


	Operating
Year	(in thousands)
2024		446
2025		23
Thereafter		20
Total lease payments		489
Less: interest		22
Total lease liabilities	$	467

INCOME TAXES - Valuation allowance (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
INCOME TAXES
Net deferred tax asset (liability)	$ 0	$ 0
Valuation allowance	$ (151,222)	$ (134,177)

FAIR VALUE MEASUREMENTS - FV hierarchy (Details) - Fair Value, Recurring - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	$ 49,898	$ 16,657
Marketable securities		13,923
Total financial assets measured at fair value on a recurring basis	49,898	30,580
Warrant liabilities - Common Warrants	53,725	13,657
Total financial liabilities measured at fair value on a recurring basis	53,725	13,657
Level 1
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	49,898	16,657
Total financial assets measured at fair value on a recurring basis	49,898	16,657
Level 2
Financial assets or liabilities measured at fair value on a recurring basis
Marketable securities		13,923
Total financial assets measured at fair value on a recurring basis		13,923
Level 3
Financial assets or liabilities measured at fair value on a recurring basis
Warrant liabilities - Common Warrants	53,725	13,657
Total financial liabilities measured at fair value on a recurring basis	53,725	13,657
Cash
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	24,887	1,337
Cash \| Level 1
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	24,887	1,337
Money market funds
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	25,011	15,320
Money market funds \| Level 1
Financial assets or liabilities measured at fair value on a recurring basis
Cash and cash equivalents	$ 25,011	15,320
U.S. government agency debt securities
Financial assets or liabilities measured at fair value on a recurring basis
Marketable securities		13,923
U.S. government agency debt securities \| Level 2
Financial assets or liabilities measured at fair value on a recurring basis
Marketable securities		$ 13,923

WARRANTS

12 Months Ended

Dec. 31, 2023

WARRANTS

WARRANTS

9. 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.

On December 5, 2019, an optionholder exercised 20,000 options in a cashless exercise at net, and the Company issued 17,026 shares of common stock.

On February 21, 2020, two warrantholders exercised 185,634 options in a cashless exercise at net, and the Company issued 176,092 shares of common stock. On February 24, 2020, a warrantholder exercised 72,818 options in a cashless exercise at net, and the Company issued 69,094 shares of common stock.

On February 5, 2021, a warrantholder exercised 27,855 warrants on a cash basis and received 27,855 shares of common stock. The Company received $69,000 in cash proceeds for the exercise of these warrants.

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 the Company’s 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 2018 Notes Warrants vested immediately upon issuance and expire on November 4, 2028. The exercise price of the 2018 Notes Warrants was $3.99 and $6.59 per share as of December 31, 2023 and 2022, respectively.

On February 24, 2020, a warrantholder exercised 386 options in a cashless exercise at net, and the Company issued 333 shares of common stock. On June 24, 2020, a warrantholder exercised 20,331 options in a cashless exercise at net, and the Company issued 17,369 shares of common stock.

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 warrants vested immediately upon issuance and expire 10 years from the date of issuance. The exercise price of the warrants was $4.86 and $8.24 per share as of December 31, 2023 and 2022, respectively.

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 warrants vested immediately upon issuance and expire 10 years from the date of issuance. The exercise price of the warrants was $4.86 and $8.24 per share as of December 31, 2023 and 2022, respectively.

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 2022 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 June22, 2022, filed with the Securities and Exchange Commission on June 24, 2022 (See Note 1 and Note 8).

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, 2022, the Pre-funded Warrants are recorded as equity, using their fair value as of the amendment date.

On June 28, 2022, a warrant holder exercised 1,750,000 Pre-Funded Warrants on a cash basis and received 1,750,000 shares of common stock. The Company received $175 in cash proceeds for the exercise of these Pre-Funded Warrants.

On April 26, 2023, in connection with the sale and issuance common of stock as part of the April Offering, the Company issued 22,000,000 Pre-Funded Warrants at an exercise price of $0.0001 per share. Each share of common stock was sold at a public offering price of $0.946 and each accompanying Pre-Funded Warrant was sold at a public offering price of $0.945, pursuant to the Securities Purchase Agreement, dated as of April 23, 2023, by and between the

Company and the 2023 Purchasers (See Note 1 and Note 8). The Pre-Funded Warrants are recorded as equity, using fair value at issuance date, which was equivalent to the fair value of the common stock.

On June 8, 2023, a warrant holder exercised 4,250,000 Pre-Funded Warrants on a cash basis and received 4,250,000 shares of common stock. The Company received $425 in cash proceeds for the exercise of these Pre-Funded Warrants.

As of December 31, 2023, the Company had 4,000,000 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.

On August 1, 2023, a warrant holder exercised 1,000,000 Common Warrants on a cash basis and received 1,000,000 shares of common stock. The Company received $1.0 million in cash proceeds for the exercise of these Common Warrants.

On September 11, 2023, a warrant holder exercised 2,500,000 Common Warrants on a cash basis and received 2,500,000 shares of common stock. The Company received $2.5 million in cash proceeds for the exercise of these Common Warrants.

On September 14, 2023, a warrant holder exercised 750,000 Common Warrants on a cash basis and received 750,000 shares of common stock. The Company received $0.8 million in cash proceeds for the exercise of these Common Warrants.

On September 19, 2023, a warrant holder exercised 6,000,000 Common Warrants on a cash basis and received 6,000,000 shares of common stock. The Company received $6.0 million in cash proceeds for the exercise of these Common Warrants.

As of December 31, 2023, the Company had 19,750,000 Common Warrants outstanding with a weighted average exercise price of $1.00 per share and an average contractual life of 5 years.

On October 12, 2023, the Company entered into an exchange agreement with entities affiliated with Venrock Healthcare Capital Partners 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. As of December 31, 2023, our treasury stock consisted of no common stock.

A summary of the Company’s outstanding common stock warrants as of December 31, 2023 is as follows:


	Equity Classified		Liability Classified		Total
	Warrants		Warrants		Warrants
Outstanding as of January 1, 2023		8,375,618		30,000,000		38,375,618
Warrants granted and issued		22,000,000		—		22,000,000
Warrants exercised		(4,250,000)		(10,250,000)		(14,500,000)
Common stock exchanged for prefunded warrants		5,658,034		—		5,658,034
Outstanding as of December 31, 2023		31,783,652		19,750,000		51,533,652

Pay vs Performance Disclosure - USD ($) $ in Thousands	12 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Pay vs Performance Disclosure
Net Income (Loss)	$ (119,763)	$ (82,508)

- Definition

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

+ References

+ Details

LICENSE AGREEMENTS - Miami University (Details) - USD ($) $ in Thousands			12 Months Ended
	Dec. 14, 2020	Oct. 28, 2020	Dec. 31, 2023	Dec. 31, 2022
Agreements
Research and development			$ 53,905	$ 55,634
General and administrative			20,623	27,316
Operating expenses			74,528	82,950
Accrued expenses and other current liabilities			15,286	14,756
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			100	100
Accrued expenses and other current liabilities			400	300
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	100
Accrued expenses and other current liabilities			$ 100	$ 1,000

STOCKHOLDERS' EQUITY - Authorized stock (Details)	12 Months Ended
STOCKHOLDERS' EQUITY - Authorized stock (Details)	Dec. 31, 2023 Vote $ / shares shares	Dec. 31, 2022 $ / shares shares
STOCKHOLDERS' EQUITY
Common Stock, authorized (in shares) \| shares	200,000,000	200,000,000
Common Stock, par value (in dollars per share) \| $ / shares	$ 0.0001	$ 0.0001
Preferred Stock, authorized (in shares) \| shares	10,000,000	10,000,000
Preferred Stock, par value (in dollars per share) \| $ / shares	$ 0.0001	$ 0.0001
Number of votes per common stock \| Vote	1

INCOME TAXES - Deferred taxes (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Deferred Tax Assets
Operating lease liability	$ 146	$ 282
Stock-based compensation	4,060	8,131
License fee	501	554
Net operating loss carryforwards	94,119	88,090
Research and development tax credits	25,145	21,397
Capitalized research and development expense	26,945	14,910
Other	445	1,085
Total deferred tax assets	151,361	134,449
Valuation allowance	(151,222)	(134,177)
Net deferred tax assets	139	272
Deferred tax liabilities
Right-of use asset	(139)	(272)
Net deferred tax liabilities	(139)	(272)
Net deferred tax asset (liability)	$ 0	$ 0

Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
CURRENT ASSETS:
Cash and cash equivalents	$ 49,898	$ 16,657
Investments		13,923
Current portion of security deposits and leasehold improvements	254	57
Prepaid expenses and other current assets	4,234	6,671
Total current assets	54,386	37,308
Noncurrent portion of security deposits and leasehold improvements		198
Operating lease right-of-use asset	447	857
TOTAL ASSETS	54,833	38,363
CURRENT LIABILITIES:
Current portion of operating lease liabilities	429	477
Accounts payable	1,742	4,534
Accrued expenses and other current liabilities	15,286	14,756
Warrant liabilities	53,725	13,657
Total current liabilities	71,182	33,424
NONCURRENT LIABILITIES:
Noncurrent portion of operating lease liabilities	38	414
Clinical holdback - long-term portion	759	464
Total noncurrent liabilities	797	878
Total liabilities	71,979	34,302
STOCKHOLDERS' (DEFICIT)/EQUITY:
Common stock, $0.0001 par value; 200,000,000 shares authorized as of December 31, 2023 and December 31, 2022; 84,869,832 shares issued and outstanding as of December 31, 2023 and 48,063,358 shares issued and outstanding as of December 31, 2022	8	5
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of December 31, 2023 and December 31, 2022; 0 shares issued and outstanding as of December 31, 2023 and December 31, 2022
Additional paid-in capital	451,432	352,828
Accumulated other comprehensive income		51
Accumulated deficit	(468,586)	(348,823)
Total stockholders' (deficit)/equity	(17,146)	4,061
TOTAL LIABILITIES AND STOCKHOLDERS' (DEFICIT)/EQUITY	$ 54,833	$ 38,363

LEASES

12 Months Ended

Dec. 31, 2023

LEASES

LEASES

10. LEASES

The following table summarizes our lease related costs for the years ended December 31, 2023 and 2022:


		Year Ended
(in thousands)		December 31,
Lease Cost	Statement of Operations Location	2023		2022
Operating Lease Cost	General and administrative	$	505	$	489
Total lease cost		$	505	$	489

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


	Year Ended
	December 31,		December 31,
Other Information	2023		2022
Weighted-average remaining lease term
Operating leases		1.1 years		1.8 years
Weighted-average discount rate
Operating leases		6.86%		5.69%

The following table summarizes the maturities of lease liabilities as of December 31, 2023:


	Operating
Year	(in thousands)
2024		446
2025		23
Thereafter		20
Total lease payments		489
Less: interest		22
Total lease liabilities	$	467

BENEFIT PLANS (Details) - USD ($) $ in Millions	12 Months Ended
BENEFIT PLANS (Details) - USD ($) $ in Millions	Dec. 31, 2023	Dec. 31, 2022
BENEFIT PLANS
Employer matching contributions to the plan	$ 0.4	$ 0.4

WARRANTS - Issued with the 2018 Notes (Details) - Warrants Issued with the 2018 Notes - USD ($) $ / shares in Units, $ in Millions	Jun. 24, 2020	Feb. 24, 2020	Nov. 05, 2018	Dec. 31, 2023	Dec. 31, 2022	Jan. 18, 2018
Warrants
Initial fair value recorded as debt discount (in dollars)						$ 0.1
Issuance of warrants (in dollars)			$ 0.3
Warrants to purchase common stock (in shares)			76,847
Exercise price (in dollars per share)				$ 3.99	$ 6.59
Warrants exercised (in shares)	20,331	386
Stock issued in exercise of warrants (in shares)	17,369	333

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Dec. 31, 2023
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

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies)

12 Months Ended

Dec. 31, 2023

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

Basis of Presentation

Basis of Presentation

Liquidity and Going Concern

Liquidity and Going Concern

Risk and Uncertainties

Risks and Uncertainties

Use of Estimates

Fair Value Measurements

Fair Value Measurements

Level 1—Quoted prices in active markets for identical assets or liabilities.

Cash and Cash Equivalents

Cash and Cash Equivalents

Investments

Investments

Leases

Leases

Clinical hold-back, long-term

Clinical hold-back, long-term

The following table shows the activity within the clinical holdback liability accounts for the year ended December 31, 2023:



(in thousands)
Balance as of December 31, 2022	$	464
Clinical holdback retained		295
Clinical holdback paid		—
Balance as of December 31, 2023	$	759

Deferred Offering Costs

Deferred Offering Costs

As of December 31, 2023 and 2022, the Company did not have any deferred offering costs recorded.

Warrant Liabilities

Warrant Liabilities

Treasury Stock

Treasury Stock

Revenue Recognition

Revenue Recognition

Research and Development

Research and Development

General and Administrative

General and Administrative

Stock Based Compensation

Stock-Based Compensation

Stock-Based Compensation–Stock Options

The fair value of stock-based payments is recognized as expense over the requisite service period which is generally the vesting period.

Stock-Based Compensation–Restricted Stock Units

Income Taxes

Income Taxes

Net Loss per Share

Net Loss per Share

Segment Information

Segment Information

Recent Accounting Pronouncements

Statements of Stockholders' Equity (Deficit) - USD ($) $ in Thousands	Common Stock	Treasury Stock	Additional Paid-in Capital	Accumulated Deficit	Accumulated Other Comprehensive Income (Loss)	Total
Balance at Dec. 31, 2021	$ 3		$ 328,958	$ (266,315)	$ (107)	$ 62,539
Balance (in shares) at Dec. 31, 2021	26,215,514
Increase (Decrease) in Stockholders' Equity
Issuance of common stock, pre-funded warrants, and common warrants sold for cash, net of issuance costs of $96	$ 2		12,801			12,803
Issuance of common stock, pre-funded warrants, and common warrants sold for cash, net of issuance costs of $96 (in shares)	20,000,000
Exercise of pre-funded warrants			1,417			1,417
Exercise of pre-funded warrants (in shares)	1,750,000
Exercise of stock options			49			49
Exercise of stock options (in shares)	47,602
Restricted stock units released for common issued under Equity Incentive Plan (in shares)	50,242
Stock-based compensation expense			9,162			9,162
Issuance of options in-lieu of bonus			441			441
Net Income (Loss)				(82,508)		(82,508)
Other comprehensive income (loss)					158	158
Balance at Dec. 31, 2022	$ 5		352,828	(348,823)	51	4,061
Balance (in shares) at Dec. 31, 2022	48,063,358
Increase (Decrease) in Stockholders' Equity
Exchange of common stock for prefunded warrants		$ (12,900)	12,900
Exchange of common stock for prefunded warrants (in shares)		5,658,034
Issuance of common stock, prefunded warrants and reissuance of treasury stock, net of issuance costs of $4.0 million	$ 2	$ 12,900	51,500			64,402
Issuance of common stock, prefunded warrants and reissuance of treasury stock, net of issuance costs of $4.0 million (in shares)	21,693,673	(5,658,034)
Exercise of common warrants	$ 1		26,754			26,755
Exercise of common warrants (in shares)	10,250,000
Exercise of pre-funded warrants (in shares)	4,250,000
Exercise of stock options			90			$ 90
Exercise of stock options (in shares)	85,619					85,619
Restricted stock units released for common issued under Equity Incentive Plan (in shares)	527,182
Stock-based compensation expense			7,360			$ 7,360
Net Income (Loss)				(119,763)		(119,763)
Other comprehensive income (loss)					$ (51)	(51)
Balance at Dec. 31, 2023	$ 8		$ 451,432	$ (468,586)		$ (17,146)
Balance (in shares) at Dec. 31, 2023	84,869,832
Balances, Treasury stock (in shares) at Dec. 31, 2023						0

WARRANTS - Issued with Series B Preferred Stock (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	Dec. 31, 2023	Dec. 31, 2022
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)			$ 4.86	$ 8.24
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)			$ 4.86	$ 8.24

Statements of Operations - USD ($) $ in Thousands	12 Months Ended
Statements of Operations - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
REVENUE:
Total Revenue	$ 9,993
COSTS AND EXPENSES:
Research and development	53,905	$ 55,634
General and administrative	20,623	27,316
Total costs and expenses	74,528	82,950
LOSS FROM OPERATIONS	(64,535)	(82,950)
OTHER INCOME (EXPENSE), NET:
Interest income	1,372	685
Change in fair value of warrant liabilities	(56,573)	(66)
Other expense	(27)	(177)
Total other income (expense), net	(55,228)	442
Net loss	$ (119,763)	$ (82,508)
Net loss per share attributable to common stockholders-basic (in dollars per share)	$ (1.42)	$ (2.18)
Net loss per share attributable to common stockholders-diluted (in dollars per share)	$ (1.42)	$ (2.18)
Weighted-average common stock outstanding-basic (in shares)	84,244,494	37,825,431
Weighted-average common stock outstanding-diluted (in shares)	84,244,494	37,825,431
License
REVENUE:
Total Revenue	$ 9,219
Research and development services
REVENUE:
Total Revenue	$ 774

NET LOSS PER COMMON SHARE (Tables)

12 Months Ended

Dec. 31, 2023

NET LOSS PER COMMON SHARE

Schedule of computation of basic and diluted net loss per common share

The following table sets forth the computation of basic and diluted net loss per common share (in thousands, except share and per share data):


	Year Ended
	December 31,
(in thousands, except for share data)	2023		2022
Numerator:
Net loss	$	(119,763)	$	(82,508)
Denominator:
Weighted-average common stock outstanding		84,244,494		37,825,431
Net loss per share attributable to common stockholders - basic and diluted	$	(1.42)	$	(2.18)

Schedule of potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders


	As of
	December 31,
	2023	2022
Options to purchase common stock	4,695,619	4,874,047
Restricted stock units	3,867,422	815,509
Warrants to purchase common stock	19,875,618	30,125,618

NET LOSS PER COMMON SHARE

12 Months Ended

Dec. 31, 2023

NET LOSS PER COMMON SHARE

NET LOSS PER COMMON SHARE

13. 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 years ended December 2023 and 2022, as the inclusion of all potential common shares outstanding would have been anti-dilutive.

The following table sets forth the computation of basic and diluted net loss per common share (in thousands, except share and per share data):


	Year Ended
	December 31,
(in thousands, except for share data)	2023		2022
Numerator:
Net loss	$	(119,763)	$	(82,508)
Denominator:
Weighted-average common stock outstanding		84,244,494		37,825,431
Net loss per share attributable to common stockholders - basic and diluted	$	(1.42)	$	(2.18)

The Company’s potentially dilutive securities, which include restricted stock units, stock options and common warrants, have been excluded from the computation of diluted net loss per share as the effect would be to reduce the net loss per share. Therefore, the weighted-average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The Company excluded the following potential common shares, presented based on amounts outstanding at December 31, 2023 and 2022, from the computation of diluted net loss per share attributable to common stockholders.


	As of
	December 31,
	2023	2022
Options to purchase common stock	4,695,619	4,874,047
Restricted stock units	3,867,422	815,509
Warrants to purchase common stock	19,875,618	30,125,618

WARRANTS - Issued with June 2022 Offering (Details) - USD ($)										12 Months Ended
	Oct. 12, 2023	Sep. 19, 2023	Sep. 14, 2023	Sep. 11, 2023	Aug. 01, 2023	Jun. 08, 2023	Apr. 26, 2023	Jun. 28, 2022	Jun. 27, 2022	Dec. 31, 2023	Dec. 31, 2022
Warrants
Exercise of common warrants										$ 26,755,000
Proceeds from exercise of warrants										$ 10,250,000
Treasury stock (in shares)										0
Common Stock
Warrants
Exercise of pre-funded warrants for common stock (in shares)						4,250,000				4,250,000	1,750,000
Exercise of common warrants										$ 1,000
Exercise of common warrants (in shares)										10,250,000
Treasury Stock
Warrants
Exchange of common stock for prefunded warrants (in shares)										5,658,034
Common And Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)										22,000,000
Warrants exercised (in shares)										14,500,000
Warrants outstanding (in shares)										51,533,652	38,375,618
Number of warrants issued in exchange of common stock (in shares)										5,658,034
Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)										22,000,000
Warrants exercised (in shares)										4,250,000
Warrants outstanding (in shares)										31,783,652	8,375,618
Number of warrants issued in exchange of common stock (in shares)										5,658,034
Common Warrants
Warrants
Warrants exercised (in shares)										10,250,000
Warrants outstanding (in shares)										19,750,000	30,000,000
June 2022 Offering
Warrants
Offering price per share and accompanying warrant (in dollars per share)									$ 1.00
Offering price per pre-funded warrant and accompanying warrant (in dollars per share)									$ 0.9999
June 2022 Offering \| Common Stock
Warrants
Exercise of pre-funded warrants for common stock (in shares)								1,750,000
Exercise of common warrants (in shares)		6,000,000	750,000	2,500,000	1,000,000
June 2022 Offering \| Common And Pre-Funded Warrants
Warrants
Warrants term									5 years
June 2022 Offering \| Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)									10,000,000
Exercise price (in dollars per share)							$ 0.0001		$ 0.0001
Warrants exercised (in shares)						4,250,000		1,750,000
Proceeds from exercise of warrants						$ 425		$ 175
Warrants outstanding (in shares)										4,000,000
June 2022 Offering \| Pre-Funded Warrants \| Weighted average
Warrants
Exercise price (in dollars per share)										$ 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)									$ 1.00
Warrants exercised (in shares)		6,000,000	750,000	2,500,000	1,000,000
Proceeds from exercise of warrants		$ 6,000,000.0	$ 800,000	$ 2,500,000	$ 1,000,000.0
Warrants outstanding (in shares)										19,750,000
June 2022 Offering \| Common Warrants \| Weighted average
Warrants
Exercise price (in dollars per share)										$ 1.00
June 2022 Offering \| Common Warrants \| Average
Warrants
Warrants term										5 years
April 2023 Offering, Private Placement \| Common Stock
Warrants
Purchase price, common stock (in dollars per share)							$ 0.946
April 2023 Offering, Private Placement \| Pre-Funded Warrants
Warrants
Issuance of warrants (in shares)							22,000,000
Exercise price (in dollars per share)							$ 0.001
Purchase price, warrant (in dollars per share)							$ 0.945
Exchange Agreement
Warrants
Fair market value	$ 12,900,000									$ 12,900,000
Exchange Agreement \| Treasury Stock
Warrants
Exchange of common stock for prefunded warrants (in shares)	5,658,034
Exchange Agreement \| Pre-Funded Warrants
Warrants
Number of warrants issued in exchange of common stock (in shares)	5,658,034
Exercise price of warrants (in dollars per share)	$ 0.001

LICENSE AGREEMENTS

12 Months Ended

Dec. 31, 2023

LICENSE AGREEMENTS

LICENSE AGREEMENTS

2. LICENSE AGREEMENTS

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 years ended December 31, 2023 and 2022, the Company recorded $0.2 million in expense related to the Columbia Agreements. In aggregate, the Company has incurred $2.9 million in expense from the execution of the Columbia Agreements through December 31, 2023.

As of December 31, 2023 and 2022, the Company had $29,000 and $0.1 million, respectively, due to Columbia University included in accrued expenses and no amounts included 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 $0.1 million in research and development expense during the year-ended December 31, 2023, related to the Miami License Agreement. As of December 31, 2023, the Company had $0.4 million in accrued expenses.

The Company recorded $0.1 million in research and development expense during the year-ended December 31, 2022, related to the Miami License Agreement. As of December 31, 2022, the Company had $0.3 million in accrued expenses.

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 years ended December 31, 2023 and 2022, the Company recorded $0 and $0.1 million in research and development expenses, respectively, in relation to the 2020 Miami Research Agreement. As of December 31, 2023 and 2022, the Company had $0.1 million and $1.0 million, respectively, in accrued expenses.

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.

NET LOSS PER COMMON SHARE - EPS (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Numerator:
Net loss attributable to common stockholders-basic	$ (119,763)	$ (82,508)
Net loss attributable to common stockholders-diluted	$ (119,763)	$ (82,508)
Denominator:
Weighted-average common stock outstanding-basic (in shares)	84,244,494	37,825,431
Weighted-average common stock outstanding-diluted (in shares)	84,244,494	37,825,431
Net loss per share attributable to common stockholders-basic (in dollars per share)	$ (1.42)	$ (2.18)
Net loss per share attributable to common stockholders-diluted (in dollars per share)	$ (1.42)	$ (2.18)

- Definition

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

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+ Details

- Definition

Amount, after deduction of tax, noncontrolling interests, dividends on preferred stock and participating securities, and addition from assumption of issuance of common shares for dilutive potential common shares; of income (loss) available to common shareholders.

+ References + Details

STOCKHOLDERS' EQUITY - Venrock Warrant Exchange (Details) - USD ($) $ / shares in Units, $ in Millions		12 Months Ended
	Oct. 12, 2023	Dec. 31, 2023
Equity
Treasury stock (in shares)		0
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
Exchange Agreement
Equity
Fair market value	$ 12.9	$ 12.9
Exchange Agreement \| Treasury Stock
Equity
Exchange of common stock for prefunded warrants (in shares)	5,658,034
Exchange Agreement \| 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)	$ 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.

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+ Details

INCOME TAXES

12 Months Ended

Dec. 31, 2023

INCOME TAXES

INCOME TAXES

11. INCOME TAXES

The Company has not recorded a current or deferred tax provision for the years ended December 31, 2023 and 2022 due to net losses incurred.

A reconciliation of the Company's deferred taxes is as follows (in thousands):


	Year ended
	December 31,
	2023		2022
Deferred Tax Assets
Operating lease liability	$	146	$	282
Stock-based compensation		4,060		8,131
License fee		501		554
Net operating loss carryforwards		94,119		88,090
Research and development tax credits		25,145		21,397
Capitalized research and development expense		26,945		14,910
Other		445		1,085
Total deferred tax assets	$	151,361	$	134,449
Valuation allowance	$	(151,222)	$	(134,177)
Net deferred tax assets	$	139	$	272
Deferred tax liabilities
Right of use asset		(139)		(272)
Net deferred tax liabilities	$	(139)	$	(272)
Net deferred tax asset (liability)	$	—	$	—

Deferred tax assets result primarily from unutilized net operating losses, research tax credits, stock-based compensation, operating lease liability, and timing differences as a result of the Company reporting its income tax returns. As of December 31, 2023, the Company had approximately $299.8 million of federal operating loss (“NOLs”) carry forwards. Of this amount, approximately $3.3 million will begin to expire in 2037 and approximately $296.5 million are carried forward indefinitely. The Company had approximately $612.0 million in state and city net operating loss carryforwards available to offset future taxable income.

As of December 31, 2023, the Company had federal research tax credits (R&D) of approximately $6.1 million which may be used to offset future tax liabilities. Additionally, the Company had a federal orphan drug credit (“ODC”) related to qualifying research of $19.1 million. These tax credit carryforwards will begin to expire at various times beginning in 2037 for federal purposes.

The NOL, R&D and ODC credits carry forwards are subject to review and possible adjustment by the U.S. and state tax authorities. NOL carry forwards and credit carry forwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders, as defined under Sections 382 and 383 Internal Revenue Code. This could limit the amount of NOLs and credits that the Company can utilize annually to offset future taxable income or tax liabilities. As of December 31, 2023, the Company has not performed such an analysis. Subsequent ownership changes and proposed future changes to tax rules in respect of the utilization of losses carried forward may further affect the limitation in future years.

In assessing the realizability of the Company’s deferred tax assets, management considers whether or not it is more likely than not that some portion or all of the deferred tax assets will not be realized. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income. The Company’s assessment is based on the weight of available evidence, including cumulative losses since inception and expected future losses and, as such, the Company does not believe it is more likely than not that the deferred tax assets will be realized. Accordingly, a full valuation allowance has been established and no deferred tax assets and related tax benefit have been recognized in the accompanying financial statements. At December 31, 2023 and 2022, the Company recorded valuation allowances of

$151.2 million and $134.2 million, respectively. The increase in valuation allowance is primarily driven by additional net operating losses.

The Company files its income tax returns in the United States, California, Iowa, Massachusetts, Michigan, New Jersey, New York State, New York City, Pennsylvania, and Texas. All years remain subject to examination for federal and state purpose.

The U.S. federal statutory corporate tax rate reconciles to the Company’s effective tax rate for the years ended December 31, 2023 and 2022:


	Year Ended
	December 31,
	2023		2022
Federal statutory rate	21.0	%	21.0	%
State and local taxes net of federal tax benefit	3.4		10.2
Credits	3.1		7.8
Permanent differences	(0.1)		(0.1)
Change in fair value of warrant liabilities	(9.9)		—
Change in valuation allowance	(14.2)		(37.9)
Stock-based compensation	(3.3)		(1.1)
Other	—		0.1
Total	0.0	%	0.0	%

- Definition

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

+ References

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+ Details

SUBSEQUENT EVENTS

12 Months Ended

Dec. 31, 2023

SUBSEQUENT EVENTS

SUBSEQUENT EVENTS

16. SUBSEQUENT EVENTS

The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. The Company has evaluated all subsequent events and determined that there are no material recognized or unrecognized subsequent events requiring disclosure, other than as disclosed within the above notes to financial statements, and except as described below.

ATM Offering

On January 31, 2024, the Company sold an additional 1,000,000 shares of the company’s common stock pursuant to the Leerink ATM Agreement with an average sale price of $3.13 per share, resulting in net proceeds of $3.0 million, after deducting underwriting discounts, commissions and offering expenses.

On February 16, 2024, the Company sold an additional 2,000,000 shares of the company’s common stock pursuant to the Leerink ATM Agreement with an average sale price of $4.90 per share, resulting in net proceeds of $9.5 million, after deducting underwriting discounts, commissions and offering expenses.

Warrant Exercises

In February 2024, warrant holders exercised 4,025,000 Common Warrants on a cash basis and received 4,025,000 shares of common stock. The Company received $4.0 million in cash proceeds for the exercise of these Common Warrants.

In January 2024, warrant holders exercised 1,706,976 Pre-Funded Warrants on a cashless basis and received 1,706,285 shares of common stock.

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, 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 gross proceeds to the Company of approximately $100 million, before deducting placement agent commissions and other 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.

The Securities have the benefit of the Registration Rights Agreement (the “Registration Rights Agreement”), dated as of February 27, 2024, by and among the Company and the 2024 Purchasers, requiring the Company to prepare and file a registration statement with the SEC as soon as reasonably practicable, but in no event later than March 28, 2024. the Filing Deadline, and to use commercially reasonable efforts to have the registration statement declared effective within 30 days of the Filing Deadline, subject to extension under the terms of the Registration Rights Agreement. 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.

FAIR VALUE MEASUREMENTS - Warrants Valuation (Details)	Dec. 31, 2023 Y	Dec. 31, 2022 Y
Expected term (in years) \| Common And Pre-Funded Warrants
Fair value
Measurement input		3.7
Expected term (in years) \| Common Warrants
Fair value
Measurement input	2.4
Volatility \| Common And Pre-Funded Warrants
Fair value
Measurement input		0.9153
Volatility \| Common Warrants
Fair value
Measurement input	1.0946
Risk-free interest rate \| Common And Pre-Funded Warrants
Fair value
Measurement input		0.0411
Risk-free interest rate \| Common Warrants
Fair value
Measurement input	0.0432
Dividend yield \| Common And Pre-Funded Warrants
Fair value
Measurement input		0.0000
Dividend yield \| Common Warrants
Fair value
Measurement input	0.0000
Probability of change in control \| Common And Pre-Funded Warrants
Fair value
Measurement input		0.100
Probability of change in control \| Common Warrants \| Valuation input period, years two and three
Fair value
Measurement input	0.20
Probability of change in control \| Common Warrants \| Valuation input period, years four and five
Fair value
Measurement input	0.05

STOCKHOLDERS' EQUITY - April 2023 Offering (Details) - USD ($) $ / shares in Units, $ in Millions		12 Months Ended
	Apr. 26, 2023	Dec. 31, 2023
Common And Pre-Funded Warrants
Equity
Issuance of warrants (in shares)		22,000,000
Pre-Funded Warrants
Equity
Issuance of warrants (in shares)		22,000,000
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
Purchase price, warrant (in dollars per share)	$ 0.945
Exercise price (in dollars per share)	$ 0.001
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants	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

STOCKHOLDERS' EQUITY

12 Months Ended

Dec. 31, 2023

STOCKHOLDERS' EQUITY

STOCKHOLDERS' EQUITY

8. STOCKHOLDERS’ EQUITY

As of December 31, 2023 and 2022, the authorized capital stock of the Company consists of 200,000,000 shares of common stock, par value $0.0001 per share and 10,000,000 shares of preferred stock, par value $0.0001 per share.

Common Stock

Voting

The holders of the common stock are entitled to one vote for each share of common stock held at all meetings of the stockholders. There is no cumulative voting.

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 Offering

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.

ATM Offering

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 December 31, 2023, the Company has sold an aggregate of 17,615,976 shares of the company’s common stock, pursuant to the Leerink ATM Agreement with an average sale price of $2.04 per share, resulting in net proceeds of $36.9 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. As of December 31, 2023, our treasury stock consisted of no common stock.

- Definition

The entire disclosure for equity.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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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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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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Reference 13: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

INCOME TAXES - Tax credit carryforward (Details) $ in Millions	Dec. 31, 2023 USD ($)
Research tax credit carryforward
Tax credit carryforward
Tax credit carryforward	$ 6.1
Orphan drug credit carryforward
Tax credit carryforward
Tax credit carryforward	$ 19.1

STOCK-BASED COMPENSATION - Valuation assumptions (Details)	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Valuation Assumptions
Dividend yield (as a percent)	0.00%
Stock options - Service conditions only
Valuation Assumptions
Expected term (in years)		5 years 8 months 12 days
Volatility (as a percent)		75.83%
Risk-free interest rate (as a percent)		2.13%

WARRANTS - Warrant Activity (Details)	12 Months Ended
WARRANTS - Warrant Activity (Details)	Dec. 31, 2023 shares
Common And Pre-Funded Warrants
Warrants
Outstanding as of beginning of period (in shares)	38,375,618
Warrants granted and issued (in shares)	22,000,000
Warrants exercised (in shares)	(14,500,000)
Common stock exchanged for prefunded warrants (in shares)	5,658,034
Outstanding as of end of period (in shares)	51,533,652
Pre-Funded Warrants - Equity Classified Warrants
Warrants
Outstanding as of beginning of period (in shares)	8,375,618
Warrants granted and issued (in shares)	22,000,000
Warrants exercised (in shares)	(4,250,000)
Common stock exchanged for prefunded warrants (in shares)	5,658,034
Outstanding as of end of period (in shares)	31,783,652
Common Warrants - Liability Classified Warrants
Warrants
Outstanding as of beginning of period (in shares)	30,000,000
Warrants exercised (in shares)	(10,250,000)
Outstanding as of end of period (in shares)	19,750,000

INCOME TAXES - Income tax rate reconciliation (Details)	12 Months Ended
INCOME TAXES - Income tax rate reconciliation (Details)	Dec. 31, 2023	Dec. 31, 2022
Tax rate reconciliation
Federal statutory rate (as a percent)	21.00%	21.00%
State and local taxes net of federal tax benefit (as a percent)	3.40%	10.20%
Credits (as a percent)	3.10%	7.80%
Permanent differences (as a percent)	(0.10%)	(0.10%)
Change in fair value of warrant liabilities (as a percent)	(9.90%)
Change in valuation allowance (as a percent)	(14.20%)	(37.90%)
Stock-based compensation (as a percent)	(3.30%)	(1.10%)
Other (as a percent)		0.10%
Total (as a percent)	0.00%	0.00%

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Liquidity and Going Concern (Details) $ in Thousands, € in Millions						1 Months Ended	2 Months Ended		12 Months Ended
	Mar. 01, 2024 USD ($)	Feb. 16, 2024 USD ($)	Jan. 31, 2024 USD ($)	Jan. 03, 2023 USD ($)	Jan. 03, 2023 EUR (€)	Jan. 31, 2023 USD ($)	Mar. 01, 2024 USD ($)	Feb. 29, 2024 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)
Cash and cash equivalents
Exercise of Warrants									$ 10,250
Net loss									(119,763)	$ (82,508)
Accumulated deficit									(468,586)	(348,823)
Cash and cash equivalents									$ 49,898	$ 16,657
Substantial Doubt about Going Concern, within One Year									false
Advanz Pharma \| License and Supply Agreement
Cash and cash equivalents
Upfront payment received				$ 10,700	€ 10.0	$ 10,700
Subsequent Event
Cash and cash equivalents
Proceeds from issuance of shares and warrants and warrant exercises, net							$ 116,500
Leerink ATM Agreement
Cash and cash equivalents
Proceeds from issuance of shares and warrants and warrant exercises, net									$ 74,700
Proceeds from stock issuance, net of cash issuance costs									$ 36,900
Leerink ATM Agreement \| Subsequent Event
Cash and cash equivalents
Exercise of Warrants								$ 4,000
Proceeds from stock issuance, net of cash issuance costs		$ 9,500	$ 3,000					$ 12,500
March 2024 Offering, Private Placement \| Subsequent Event
Cash and cash equivalents
Proceeds from issuance of shares and warrants, gross	$ 100,000

STOCK-BASED COMPENSATION - Options (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
STOCK-BASED COMPENSATION
Options granted (in shares)	0
Stock-based compensation expense	$ 7,360	$ 9,603
Unrecognized stock-based compensation balance for unvested options	2,600
Weighted-average fair value of options granted (in dollars per share)		$ 1.79
Employee Stock Option
STOCK-BASED COMPENSATION
Stock-based compensation expense	$ 5,000	$ 7,800
Weighted Average Remaining Recognition Period (in years)	1 year 8 months 12 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

REVENUE

12 Months Ended

Dec. 31, 2023

REVENUE

REVENUE

15. REVENUE

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 Applied 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. The Company allocated $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.

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 $9.3 million as license revenue on its statements of operations for the year ended December 31, 2023.

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 December 31, 2023, as a percentage of the estimated total costs to be incurred for this performance obligation. The Company recognized $0.7 million of revenue

during year ended December 31, 2023, and had deferred revenue of $0.5 million related to the research and development performance obligation at December 31, 2023. The Company expects to recognize this deferred revenue over the next three years. 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 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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+ Details

Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2023	Dec. 31, 2022
Balance Sheets
Common Stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Common Stock, authorized (in shares)	200,000,000	200,000,000
Common Stock, issued (in shares)	84,869,832	48,063,358
Common stock, outstanding (in shares)	84,869,832	48,063,358
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

Document and Entity Information - USD ($)	12 Months Ended
Document and Entity Information - USD ($)	Dec. 31, 2023	Mar. 05, 2024	Jun. 30, 2023
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2023
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
Trading Symbol	APLT
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	No
Entity Voluntary Filers	No
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
ICFR Auditor Attestation Flag	false
Document Financial Statement Error Correction	false
Entity Shell Company	false
Entity Public Float			$ 63,926,605
Entity Common Stock, Shares Outstanding		105,886,831
Auditor Name	Ernst & Young LLP
Auditor Location	New York, New York
Auditor Firm ID	42
Entity Central Index Key	0001697532
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	FY
Amendment Flag	false

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Deferred Offering Costs (Details) - USD ($) $ in Millions	Dec. 31, 2023	Dec. 31, 2022
Deferred offering cost
Deferred offering costs	$ 0.0	$ 0.0

REVENUE (Details) $ in Thousands, € in Millions			1 Months Ended	12 Months Ended
REVENUE (Details) $ in Thousands, € in Millions	Jan. 03, 2023 USD ($) item	Jan. 03, 2023 EUR (€) item	Jan. 31, 2023 USD ($)	Dec. 31, 2023 USD ($)	Jan. 03, 2023 EUR (€)
Revenue
Total Revenue				$ 9,993
Deferred revenue				667
Research and development services
Revenue
Total Revenue				774
License
Revenue
Total Revenue				9,219
License and Supply Agreement \| Advanz Pharma
Revenue
Upfront payment received	$ 10,700	€ 10.0	$ 10,700
Potential milestone payments to be received	$ 142,200				€ 134.0
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 \| Research and development services
Revenue
Amount of performance obligation	$ 1,300
Total Revenue				700
License and Supply Agreement \| Advanz Pharma \| Manufacturing and supply material right
Revenue
Amount of performance obligation	100			100
License and Supply Agreement \| Advanz Pharma \| License
Revenue
Amount of performance obligation	$ 9,300
Total Revenue				$ 9,300

PREPAID EXPENSES AND OTHER CURRENT ASSETS

12 Months Ended

Dec. 31, 2023

PREPAID EXPENSES AND OTHER CURRENT ASSETS

PREPAID EXPENSES AND OTHER CURRENT ASSETS

5. PREPAID EXPENSES AND OTHER CURRENT ASSETS

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


	December 31,		December 31,
(in thousands)	2023		2022
Prepaid research and development expenses	$	2,880	$	4,272
Insurance premium asset		566		1,131
Prepaid rent expenses		47		99
Prepaid insurance expenses		72		71
Prepaid commercial and patient advocacy		284		206
Research and development tax credit receivable		262		252
Interest receivable		23		23
Other prepaid expenses and current assets		100		617
Total prepaid expenses & other current assets	$	4,234	$	6,671

STOCK-BASED COMPENSATION - Plans (Details) - USD ($) $ / shares in Units, $ in Millions		1 Months Ended	12 Months Ended
	Aug. 02, 2022	May 31, 2019	Dec. 31, 2023	Jan. 01, 2024	Aug. 01, 2022
Employee Stock Option
STOCK-BASED COMPENSATION
Weighted Average Remaining Recognition Period (in years)			1 year 8 months 12 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%
Option exercise price upon option repricing (in dollars per share)	$ 1.05
Options vested upon option repricing (in Shares)	1,797,517
Options non-vested upon option repricing (in Shares)	1,380,917
Incremental stock based compensation expense	$ 1.4
Weighted Average Remaining Recognition Period (in years)	2 years 4 months 24 days
Number of shares available for future issuance			0
2019 Plan \| Subsequent Event
STOCK-BASED COMPENSATION
Number of shares available for future issuance				4,243,292
2019 Plan \| Minimum
STOCK-BASED COMPENSATION
Original exercise price (in dollars per share)					$ 1.22
2019 Plan \| Maximum
STOCK-BASED COMPENSATION
Original exercise price (in dollars per share)					$ 49.60
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
2019 Plan \| Vested stock option
STOCK-BASED COMPENSATION
Incremental stock based compensation expense	$ 0.9
2019 Plan \| Non-vested stock option
STOCK-BASED COMPENSATION
Incremental stock based compensation expense	$ 0.5

INVESTMENTS (Tables)

12 Months Ended

Dec. 31, 2023

INVESTMENTS

Schedule of marketable securities by security type

The following tables provide the Company’s marketable securities by security type (in thousands):


	As of December 31, 2023								As of December 31, 2022
			Gross		Gross						Gross		Gross
			Unrealized		Unrealized		Estimated				Unrealized		Unrealized		Estimated
(in thousands)	Cost		Gains		Losses		Fair Value		Cost		Gains		Losses		Fair Value
US government agency debt security	$	—	$	—	$	—	$	—	$	13,873	$	50	$	—	$	13,923
Total	$	—	$	—	$	—	$	—	$	13,873	$	50	$	—	$	13,923

Schedule of contractual maturities

Contractual maturities of the Company’s marketable securities are summarized as follows (in thousands):


	As of December 31, 2023								As of December 31, 2022
			Gross		Gross						Gross		Gross
			Unrealized		Unrealized		Estimated				Unrealized		Unrealized		Estimated
(in thousands)	Cost		Gains		Losses		Fair Value		Cost		Gains		Losses		Fair Value
Due in one year or less	$	—	$	—	$	—	$	—	$	13,873	$	50	$	—	$	13,923
Due in one through two years		—		—		—		—		—		—		—		—
Total	$	—	$	—	$	—	$	—	$	13,873	$	50	$	—	$	13,923

NET LOSS PER COMMON SHARE - Anti-dilutive securities (Details) - shares	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
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	4,695,619	4,874,047
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	3,867,422	815,509
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	19,875,618	30,125,618

Statements of Stockholders' Equity (Deficit) (Parenthetical) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Common Stock
Increase (Decrease) in Stockholders' Equity
Net of issuance costs		$ 96
Treasury Stock
Increase (Decrease) in Stockholders' Equity
Net of issuance costs	$ 4,000

INVESTMENTS

12 Months Ended

Dec. 31, 2023

INVESTMENTS

INVESTMENTS

4. INVESTMENTS

Marketable Securities

Marketable securities, which the Company classifies as available-for-sale securities, primarily consisted of U.S. government debt obligations as of December 31, 2022. Marketable securities with remaining effective maturities of twelve months or less from the balance sheet date are classified as short-term; otherwise, they are classified as long-term on the balance sheets.

The following tables provide the Company’s marketable securities by security type (in thousands):


	As of December 31, 2023								As of December 31, 2022
			Gross		Gross						Gross		Gross
			Unrealized		Unrealized		Estimated				Unrealized		Unrealized		Estimated
(in thousands)	Cost		Gains		Losses		Fair Value		Cost		Gains		Losses		Fair Value
US government agency debt security	$	—	$	—	$	—	$	—	$	13,873	$	50	$	—	$	13,923
Total	$	—	$	—	$	—	$	—	$	13,873	$	50	$	—	$	13,923

As of December 31, 2023, the Company did not have an investment portfolio.

Contractual maturities of the Company’s marketable securities are summarized as follows (in thousands):


	As of December 31, 2023								As of December 31, 2022
			Gross		Gross						Gross		Gross
			Unrealized		Unrealized		Estimated				Unrealized		Unrealized		Estimated
(in thousands)	Cost		Gains		Losses		Fair Value		Cost		Gains		Losses		Fair Value
Due in one year or less	$	—	$	—	$	—	$	—	$	13,873	$	50	$	—	$	13,923
Due in one through two years		—		—		—		—		—		—		—		—
Total	$	—	$	—	$	—	$	—	$	13,873	$	50	$	—	$	13,923

At December 31 2023, the Company had no gross unrealized gains and gross unrealized losses.

During the year ended December 31, 2023, gross realized gains and losses from the sale of marketable securities, were immaterial.

There were no unrealized losses and fair values of available-for-sale securities that have been in an unrealized loss position for a period of less than or greater than 12 months as of December 31 2023 or 2022.

FAIR VALUE MEASUREMENTS (Tables)

12 Months Ended

Dec. 31, 2023

Fair value

Schedule of financial assets or liabilities 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


	As of December 31, 2022
(in thousands)	Level 1		Level 2		Level 3		Total
Cash	$	1,337	$	—	$	—	$	1,337
Money market funds		15,320		—		—		15,320
Total cash and cash equivalents	$	16,657	$	—	$	—	$	16,657
U.S. government agency debt securities		—		13,923		—		13,923
Total marketable securities	$	—	$	13,923	$	—	$	13,923
Total financial assets measured at fair value on a recurring basis	$	16,657	$	13,923	$	—	$	30,580
Warrant liabilities - Common Warrants		—		—		13,657		13,657
Total financial liabilities measured at fair value on a recurring basis	$	—	$	—	$	13,657	$	13,657

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, 2023	$	13,657
Warrants exercised		(16,505)
Change in fair value		56,573
Balance as of December 31, 2023	$	53,725

Common And Pre-Funded Warrants

Fair value

Schedule of fair value of warrants using the Black Scholes option pricing model


	December 31,		December 31,
	2023		2022
Expected term (in years)	2.4		3.7
Volatility	109.46	%	91.53	%
Risk-free interest rate	4.32	%	4.11	%
Dividend yield	0.00	%	0.00	%

LEASES - Lease Cost (Details) - USD ($) $ in Thousands	12 Months Ended
LEASES - Lease Cost (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Leases
Operating Lease Cost	$ 505	$ 489
General and administrative
Leases
Operating Lease Cost	$ 505	$ 489

STOCKHOLDERS' EQUITY - ATM Offering (Details) - Leerink ATM Agreement - USD ($) $ / shares in Units, $ in Millions				2 Months Ended	12 Months Ended
	Feb. 16, 2024	Jan. 31, 2024	Aug. 11, 2023	Feb. 29, 2024	Dec. 31, 2023
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					$ 36.9
Subsequent Event
Equity
Proceeds from stock issuance, net of cash issuance costs	$ 9.5	$ 3.0		$ 12.5
Common Stock
Equity
Issuance of common stock (in shares)					17,615,976
Common Stock \| Subsequent Event
Equity
Issuance of common stock (in shares)	2,000,000	1,000,000
Common Stock \| Average
Equity
Purchase price, common stock (in dollars per share)					$ 2.04
Common Stock \| Average \| Subsequent Event
Equity
Purchase price, common stock (in dollars per share)	$ 4.90	$ 3.13

FAIR VALUE MEASUREMENTS - Fair Values of Company's Warrant Liability (Details) - Common Warrants $ in Thousands	12 Months Ended
	Dec. 31, 2023 USD ($)
Fair Value
Beginning Balance	$ 13,657
Warrants exercised	(16,505)
Change in fair value	56,573
Ending Balance	$ 53,725

PREPAID EXPENSES AND OTHER CURRENT ASSETS (Tables)

12 Months Ended

Dec. 31, 2023

PREPAID EXPENSES AND OTHER CURRENT ASSETS

Summary of prepaid expenses and other current assets

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


	December 31,		December 31,
(in thousands)	2023		2022
Prepaid research and development expenses	$	2,880	$	4,272
Insurance premium asset		566		1,131
Prepaid rent expenses		47		99
Prepaid insurance expenses		72		71
Prepaid commercial and patient advocacy		284		206
Research and development tax credit receivable		262		252
Interest receivable		23		23
Other prepaid expenses and current assets		100		617
Total prepaid expenses & other current assets	$	4,234	$	6,671

BENEFIT PLANS

12 Months Ended

Dec. 31, 2023

BENEFIT PLANS

BENEFIT PLANS

12. 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. The Company made approximately $0.4 million in matching contributions to the plan during each of the years ended December 31, 2023 and 2022.

- Definition

The entire disclosure for retirement benefits.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 715
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-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480794/715-70-50-1

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 715
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-Name Accounting Standards Codification
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org//1943274/2147480506/715-20-50-1

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 715
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-Name Accounting Standards Codification
-Section S99
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480126/715-20-S99-2

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

+ Details

INVESTMENTS - By type (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Marketable securities by type
Cost		$ 13,873
Gross Unrealized Gains	$ 0	50
Gross Unrealized Losses	0
Estimated Fair Value	$ 0	13,923
U.S. government agency debt securities
Marketable securities by type
Cost		13,873
Gross Unrealized Gains		50
Estimated Fair Value		$ 13,923

- 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
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-2

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
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-Name Accounting Standards Codification
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-3

+ Details

INVESTMENTS - Unrealized losses and fair values of available-for-sale securities (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Securities in an unrealized loss position less than 12 months
Amount of unrealized losses in investment in available-for-sale securities in unrealized loss position for less than 12 months	$ 0	$ 0
Fair value of available-for-sale securities in unrealized loss position for less than 12 months	0	0
Securities in an unrealized loss position greater than 12 months
Amount of unrealized losses in investment in available-for-sale securities in unrealized loss position for greater than 12 months	0	0
Fair value of available-for-sale securities in unrealized loss position for greater than 12 months	$ 0	$ 0

ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Tables)

12 Months Ended

Dec. 31, 2023

ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

Summary of accrued expenses and other current liabilities

Accrued expenses and other current liabilities consisted of the following (in thousands):


	December 31,		December 31,
(in thousands)	2023		2022
Accrued pre-clinical and clinical expenses	$	10,142	$	8,877
Short-term insurance financing note		310		622
Deferred revenue		667		—
Accrued professional fees		1,307		1,218
Accrued compensation and benefits		2,066		2,301
Accrued commercial expenses		19		896
Accrued patent expenses		396		361
Other		379		481
Total accrued expenses & other current liabilities	$	15,286	$	14,756

LICENSE AGREEMENTS - Columbia (Details) - USD ($) $ in Thousands		1 Months Ended			12 Months Ended		87 Months Ended
	Oct. 03, 2019	Mar. 31, 2019	Jan. 31, 2019	Oct. 31, 2016	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2023
Agreements
Research and development					$ 53,905	$ 55,634
General and administrative					20,623	27,316
Operating expenses					74,528	82,950
Accrued expenses and other current liabilities					15,286	14,756	$ 15,286
Accounts payable					1,742	4,534	1,742
License Agreements and Research Services Agreements \| Columbia University
Agreements
Operating expenses					200	200	2,900
Accrued expenses and other current liabilities					29,000,000	$ 100	29,000,000
Accounts payable					$ 0		$ 0
2016 license agreement \| Columbia University
Agreements
Percentage of outstanding common stock issued				5.00%
Fair value of stock issued				$ 500
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
Payment on achievement of specified level of aggregate annual net sales				$ 1,000
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
Payment on achievement of specified level of aggregate annual net sales			$ 1,000
2019 research agreement \| Columbia University
Agreements
Agreement term		12 months
PI3k Research agreement \| Columbia University
Agreements
Agreement term	18 months

STOCK-BASED COMPENSATION (Tables)

12 Months Ended

Dec. 31, 2023

STOCK-BASED COMPENSATION

Schedule of stock-based compensation expenses

Total stock-based compensation expense recorded for employees, directors and non-employees (in thousands):


	Year Ended
	December 31,
(in thousands)	2023		2022
Research and development	$	3,005	$	3,839
General and administrative		4,355		5,764
Total stock-based compensation expense	$	7,360	$	9,603

Schedule of options outstanding


				Weighted-Average
		Weighted-		Remaining	Aggregate
	Options	Average		Contractual	Intrinsic
(in thousands, except for share data)	Outstanding	Exercise Price		Term (in years)	Value
Outstanding at December 31, 2022	4,874,047	$	1.97	6.9	$	—
Options granted	—		—
Options exercised	(85,619)		1.05
Forfeited	(84,639)		1.05
Expired	(8,170)		1.05
Outstanding at December 31, 2023	4,695,619	$	2.01	5.9	$	7,875

Exercisable at December 31, 2023	4,334,312	$	2.09	5.7	$	7,044
Nonvested at December 31, 2023	361,307	$	1.05	7.7	$	831

Schedule of restricted stock units outstanding


		Weighted-Average
		Grant Date
(in thousands, except for share data)	Shares	Fair Value
Outstanding at December 31, 2022	815,509	$	5.96
Awarded	6,814,362		2.09
Released	(527,182)		4.40
Forfeited	(185,267)		2.81
Outstanding at December 31, 2023	6,917,422	$	2.35
Nonvested at December 31, 2023	6,917,422	$	2.35

Weighted Average Remaining Recognition Period (in years)	3.3

Stock options - Service conditions only

STOCK-BASED COMPENSATION

Schedule of valuation assumptions


	Year Ended
	December 31,
	2023			2022
Expected term (in years)		—			5.7
Volatility		—	%		75.83	%
Risk-free interest rate		—	%		2.13	%
Dividend yield		—	%		—	%

- 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

INVESTMENTS - By contractual maturity (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
INVESTMENTS
Due in one year or less - Cost		$ 13,873
Due in on year or less - Gross Unrealized Gains		50
Due in one year or less - Estimated Fair Value		13,923
Cost		13,873
Gross Unrealized Gains	$ 0	50
Gross Unrealized Losses	0
Estimated Fair Value	$ 0	$ 13,923

ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Income Taxes (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
Uncertain tax positions	$ 0	$ 0
Accrued interest and penalties	$ 0	$ 0

WARRANTS - Issued with Series A Preferred Stock (Details) $ / shares in Units, $ in Thousands						12 Months Ended
	Feb. 05, 2021 USD ($) shares	Feb. 24, 2020 shares	Feb. 21, 2020 item shares	Dec. 05, 2019 shares	Jan. 26, 2017 USD ($) $ / shares shares	Dec. 31, 2023 USD ($)
Warrants
Proceeds from exercise of warrants \| $						$ 10,250
2017 Warrants \| Warrants Issued with Series A Preferred Stock
Warrants
Issuance of warrants (in shares) \| shares					309,389
Issuance of warrants (in dollars) \| $					$ 200
Exercise price (in dollars per share) \| $ / shares					$ 2.49
Number of warrant holders that exercised options \| item			2
Warrants exercised (in shares) \| shares	27,855	72,818	185,634	20,000
Stock issued in exercise of warrants (in shares) \| shares	27,855	69,094	176,092	17,026
Proceeds from exercise of warrants \| $	$ 69

INCOME TAXES - NOLs (Details) $ in Millions	Dec. 31, 2023 USD ($)
Federal
Income taxes
NOLs	$ 299.8
NOLs expiring through 2037	3.3
NOLs not subject expiration	296.5
State
Income taxes
NOLs	$ 612.0