0001711279-23-000073 | 10-Q | XBRL Viewer

Net Income (Loss) Per Share Attributable to Common Stockholders

9 Months Ended

Sep. 30, 2023

Net Income (Loss) Per Share Attributable to Common Stockholders

Basic net income (loss) per share attributable to common stockholders is calculated by dividing net income (loss) attributable to common stockholders by the weighted average shares outstanding during the period, without consideration for common stock equivalents. Diluted net income (loss) per share attributable to common stockholders is computed by dividing the net income (loss) by the weighted-average number of shares of common stock and common share equivalents outstanding for the period. Common share equivalents consist of common stock issuable upon exercise of stock options and vesting of restricted stock awards and restricted stock units.

There were 378,299 and 1,277,313 common share equivalents, in the form of stock options, that have been excluded of from the calculation of diluted net income (loss) per common share for the three and nine months ended September 30, 2023, respectively, as their effect would be anti-dilutive. There were 3,565,110 common share equivalents outstanding in the form of stock options and unvested restricted stock awards as of September 30, 2022 that were excluded from the calculation of diluted net income (loss) per common share as their effect would be anti-dilutive.


	Three Months Ended September 30,				Nine Months Ended September 30,
(In thousands, except share and per share data)	2023		2022		2023		2022

Numerator:
Net income (loss)	$	80,747	$	(29,850)	$	2,240	$	(107,923)
Denominator:
Weighted-average basic common shares	28,042,130		25,619,125		26,812,278		25,428,097
Dilutive effect of stock options and unvested restricted stock	850,096		—		572,261		—
Weighted-average diluted common shares	28,892,226		25,619,125		27,384,539		25,428,097

Net income (loss) per common share — Basic	$	2.88	$	(1.17)	$	0.08	$	(4.24)
Net income (loss) per common share — Diluted	$	2.79	$	(1.17)	$	0.08	$	(4.24)

Stock-Based Compensation - Summary of Stock Based Compensation Expense Related to Issuance of Stock Option Awards and Restricted Stock Awards (Detail) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Stock Options
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	$ 6,829	$ 8,617	$ 25,739	$ 22,338
Restricted Stock
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	437	441	1,305	1,340
Restricted Stock Units (RSUs)
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	673	0	1,835	0
Performance Shares
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	373	0	1,201	0
Research and development \| Stock Options
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	2,033	2,184	6,858	5,547
Research and development \| Restricted Stock Units (RSUs)
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	303	0	837	0
Selling, general, and administrative \| Stock Options
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	4,796	6,433	18,881	16,791
Selling, general, and administrative \| Restricted Stock
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	437	441	1,305	1,340
Selling, general, and administrative \| Restricted Stock Units (RSUs)
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	370	0	998	0
Selling, general, and administrative \| Performance Shares
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation	$ 373	$ 0	$ 1,201	$ 0

Balance Sheet Components - Schedule of Property and Equipment, Net (Detail) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Property, Plant and Equipment [Line Items]
Total property and equipment	$ 174,768	$ 168,477
Accumulated depreciation	(10,739)	(6,793)
Property and equipment, net	164,029	161,684
Building and building improvements
Property, Plant and Equipment [Line Items]
Total property and equipment	72,193	0
Construction in progress
Property, Plant and Equipment [Line Items]
Total property and equipment	56,074	131,331
Leasehold improvements
Property, Plant and Equipment [Line Items]
Total property and equipment	24,835	24,217
Manufacturing equipment
Property, Plant and Equipment [Line Items]
Total property and equipment	16,827	9,783
Laboratory equipment
Property, Plant and Equipment [Line Items]
Total property and equipment	2,339	2,089
Furniture and fixtures
Property, Plant and Equipment [Line Items]
Total property and equipment	1,518	957
Computer equipment and software
Property, Plant and Equipment [Line Items]
Total property and equipment	$ 982	$ 100

Balance Sheet Components

9 Months Ended

Sep. 30, 2023

Balance Sheet Related Disclosures [Abstract]

Balance Sheet Components

Property and Equipment, Net

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


	September 30, 2023		December 31, 2022

Building and building improvements	72,193		—
Construction in progress	$	56,074	$	131,331
Leasehold improvements	24,835		24,217
Manufacturing equipment	16,827		9,783
Laboratory equipment	2,339		2,089
Furniture and fixtures	1,518		957
Computer equipment and software	982		100
Total property and equipment	174,768		168,477
Accumulated depreciation	(10,739)		(6,793)
Property and equipment, net	$	164,029	$	161,684

Depreciation expense was $1.2 million and $3.5 million for the three and nine months ended September 30, 2023 and $669 thousand and $1.6 million for the three and nine months ended September 30, 2022, respectively.

On March 27, 2023, the Company received the permanent occupancy permit for its second commercial scale CGMP facility, ASTRA, which allowed the Company to begin utilizing certain portions of the building. As a result, certain assets relating to ASTRA were reclassified from construction in progress to leasehold improvements, manufacturing equipment, buildings and building improvements, furniture and fixtures, or computer equipment and software during the first half of 2023. The Company placed additional portions of ASTRA into service during the three months ended September 30, 2023 as it was determined that additional assets were ready for their intended use. As certain building improvements are not yet complete and certain qualification activities are still underway, the Company will continue to hold the remaining assets within construction in progress until validation has been completed and the assets are ready for their intended use. Validation of the facility is expected to be completed in 2023.

Accrued Expenses and Other Current Liabilities

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


	September 30, 2023		December 31, 2022



Accrued construction in progress	$	7,548	$	11,452
Accrued payroll and benefits	7,033		6,781
Accrued professional fees	2,786		3,397
Other current liabilities	2,315		267
Accrued preclinical and clinical expenses	1,908		1,365

Accrued taxes	152		43
Total	$	21,742	$	23,305

Revenue Recognition

9 Months Ended

Sep. 30, 2023

Revenue from Contract with Customer [Abstract]

Revenue Recognition

The Company began commercial marketing and sales of VYJUVEK throughout the United States and began recognizing revenue in 3Q 2023. For the three and nine months ended September 30, 2023 and 2022, the Company recognized net product revenues of $8.6 million and zero, respectively. Accounts receivable balances were $9.3 million and zero as of September 30, 2023 and December 31, 2022, respectively.

The following table summarizes changes in allowances and discounts for the three months ended September 30, 2023 (in thousands):


	Rebates		Prompt Pay		Other Accruals		Total
Balance as of June 30, 2023	$	—	$	—	$	—	$	—
Provision	920		195		78		1,192
Payments/Credits	—		(5)		—		(5)
Balance, as of September 30, 2023	$	920	$	190	$	78	$	1,187

- 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
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-9

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 10
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-10

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 15
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-15

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (b)(2)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-13

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
-Topic 606
-Publisher FASB
-URI https://asc.fasb.org//606/tableOfContent

+ Details

Balance Sheet Components - Schedule of Accrued Expenses And Other Current Liabilities (Detail) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Balance Sheet Related Disclosures [Abstract]
Accrued construction in progress	$ 7,548	$ 11,452
Accrued payroll and benefits	7,033	6,781
Accrued professional fees	2,786	3,397
Other current liabilities	2,315	267
Accrued preclinical and clinical expenses	1,908	1,365
Accrued taxes	152	43
Total	$ 21,742	$ 23,305

Condensed Consolidated Statements of Cash Flows (unaudited) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
Operating Activities
Net income (loss)	$ 2,240	$ (107,923)
Adjustments to reconcile net income (loss) to net cash (used in) operating activities
Gain from sale of priority review voucher	(100,000)	0
Depreciation and amortization	2,569	2,966
Stock-based compensation expense	30,080	23,678
Loss on disposals of fixed assets	0	22
Other, net	(4,290)	(224)
Changes in operating assets and liabilities
Accounts receivable	(9,316)	0
Inventory	(3,983)	0
Prepaid expenses and other current assets	(137)	1,031
Other non-current assets	(48)	(31)
Lease liability	(603)	(459)
Accounts payable	121	(316)
Accrued expenses and other current liabilities	1,795	3,016
Net cash (used in) operating activities	(81,572)	(78,240)
Investing Activities
Proceeds from sale of priority review voucher	100,000	0
Purchases of property and equipment	(9,952)	(47,762)
Purchases of investments	(425,870)	(214,712)
Proceeds from maturities of investments	428,620	153,599
Net cash provided by (used in) investing activities	92,798	(108,875)
Financing Activities
Issuance of common stock, net of issuance costs	200,880	32,927
Taxes paid related to settlement of restricted stock awards	(749)	(649)
Net cash provided by financing activities	200,131	32,278
Effect of exchange rate changes on cash and cash equivalents	(16)	0
Net increase (decrease) in cash and cash equivalents	211,341	(154,837)
Cash and cash equivalents at beginning of period	161,900	341,246
Cash and cash equivalents at end of period	373,241	186,409
Supplemental Disclosures of Non-Cash Investing and Financing Activities
Unpaid purchases of property and equipment included in accounts payable and accrued expenses	11,103	15,305
Initial recognition of right-of-use assets	$ 0	$ 1,556

Fair Value Instruments

9 Months Ended

Sep. 30, 2023

Fair Value Disclosures [Abstract]

Fair Value Instruments

The following tables show the Company’s cash, cash equivalents and available-for-sale securities by significant investment category as of September 30, 2023 and December 31, 2022, respectively (in thousands):


	September 30, 2023

	Amortized Cost		Gross Unrealized Gains		Gross Unrealized (Losses)		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash and cash equivalents	$	373,241	$	—	$	—	$	373,241	$	373,241	$	—	$	—
Subtotal	373,241		—		—		373,241		373,241		—		—
Level 2:

Commercial paper	43,496		1		(9)		43,488		—		43,488		—
Corporate bonds	70,465		4		(201)		70,268		—		56,463		13,805
U.S. government agency securities	111,635		225		(240)		111,620		—		88,877		22,743
Subtotal	225,596		230		(450)		225,376		—		188,828		36,548
Total	$	598,837	$	230	$	(450)	$	598,617	$	373,241	$	188,828	$	36,548


	December 31, 2022
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized (Losses)		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash and cash equivalents	$	161,900	$	—	$	—	$	161,900	$	161,900	$	—	$	—

Subtotal	161,900		—		—		161,900		161,900		—		—
Level 2:
Commercial paper	63,624		5		(23)		63,606		—		63,606		—
Corporate bonds	82,241		13		(419)		81,835		—		77,214		4,621
U.S. government agency securities	76,683		161		(393)		76,451		—		76,451		—



Subtotal	222,548		179		(835)		221,892		—		217,271		4,621
Total	$	384,448	$	179	$	(835)	$	383,792	$	161,900	$	217,271	$	4,621

(1)The Company’s short-term marketable securities mature in one year or less.

(2)The Company's long-term marketable securities mature between one year and two years.

See Note 2 to these unaudited condensed consolidated financial statements for additional discussion regarding the Company’s fair value measurements.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References + Details

Commitments and Contingencies	9 Months Ended
Commitments and Contingencies	Sep. 30, 2023
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	Commitments and Contingencies Agreements with Contract Research Organizations and Contract Manufacturing Organizations The Company enters into various agreements in the normal course of business with Contract Research Organizations ("CROs"), Contract Manufacturing Organizations ("CMOs") and other third parties for preclinical research studies, clinical trials and testing and manufacturing services. The agreements with CMOs primarily relate to the manufacturing of our cell and virus banks and for the manufacturing of our sterile gel that is mixed with in-house produced vectors as part of the final drug product for VYJUVEK. Agreements with third parties may also include research and development consulting activities, clinical-trial agreements, storage, packaging, labeling, and/or testing of our preclinical and clinical-stage or pre-commercial products. The Company is obligated to make milestone payments under certain of these contracts. The Company may also be responsible for the payment of a monthly service fee for project management services for the duration of any agreements. The estimated remaining commitment as of September 30, 2023 under these agreements is approximately $2.7 million. The Company has incurred research and development expenses under these agreements of $1.9 million and $5.0 million for the three and nine months ended September 30, 2023, respectively, and $2.1 million and $5.1 million for the three and nine months ended September 30, 2022, respectively. ASTRA Contractual Obligations The Company has contracted with various third parties to complete and qualify our second CGMP facility, ASTRA. These contracts typically call for the payment of fees for services or materials upon the achievement of certain milestones. The estimated remaining commitment as of September 30, 2023 is $11.1 million and primarily relates to the remaining building improvements and certain qualification activities of the facility. The Company has included costs incurred to-date associated with the ongoing build-out of ASTRA within construction in progress, except for the assets of the facility that have been placed in service. As of September 30, 2023, Substantial Completion, as defined in the Standard Form of Contract for Construction and the corresponding General Conditions of the Contract for Construction (the “Agreement”) with Whiting-Turner Contracting Company (“Whiting-Turner”), the construction manager for ASTRA, had not been achieved. Whiting-Turner’s work under the Agreement represents a portion of the work necessary to complete construction of the ASTRA facility and, therefore the date of Substantial Completion of Whiting-Turner’s work under the Agreement may not equate to the date of in-service for portions of ASTRA or the date of full facility completion of ASTRA. Legal Proceedings In May 2020, a complaint was filed against the Company in the United States District Court for the Western District of Pennsylvania by PeriphaGen, Inc. ("PeriphaGen") alleging breach of contract and misappropriation of trade secrets. On April 27, 2022, the Company and PeriphaGen entered into a final settlement agreement, and the Company paid PeriphaGen an upfront payment of $25.0 million on April 28, 2022 for: (i) the release of all claims in the trade secret litigation with PeriphaGen; (ii) the acquisition of certain PeriphaGen assets, and (iii) the grant of a license by PeriphaGen for dermatological applications. In accordance with the settlement agreement, on June 15, 2023, the Company paid PeriphaGen an additional $12.5 million following the FDA’s approval of VYJUVEK. The settlement agreement requires the Company to pay three additional $12.5 million contingent milestone payments upon reaching $100.0 million in total cumulative sales, $200.0 million in total cumulative sales and $300.0 million in total cumulative sales. As defined in the settlement agreement, cumulative sales shall include all revenue from sales of the Company products by the Company and its affiliates and licensees, as reported by the Company in its annual Form 10-K filings. If all milestones are achieved, the total consideration for settling the dispute, acquiring certain assets, and granting of a license from PeriphaGen will be $75.0 million, of which $37.5 million has been paid. The Company recorded the settlement payments of zero and $12.5 million for the three and nine months ended September 30, 2023, respectively, and zero and $25.0 million for the three and nine months ended September 30, 2022, respectively, under litigation settlement expense on the condensed consolidated statements of operations. As of September 30, 2023, the Company has not recorded an accrual for the remaining contingent milestone payments. The Company did not receive insurance proceeds during the three and nine months ended September 30, 2023 and received zero and $768 thousand during the three and nine months ended September 30, 2022, respectively. The reimbursements have been recorded as a reduction to our legal fees included in selling, general, and administrative expenses on the condensed consolidated statements of operations and within operating activities on the condensed consolidated statements of cash flows.

Condensed Consolidated Balance Sheets (unaudited) (Parenthetical) - $ / shares	Sep. 30, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Common stock, par value (in dollars per share)	$ 0.00001	$ 0.00001
Common stock, shares authorized	80,000,000	80,000,000
Common stock, shares issued	28,194,655	25,763,743
Common stock, shares outstanding	28,194,655	25,763,743

Summary of Significant Accounting Policies - Additional Information (Detail)	3 Months Ended		9 Months Ended
	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($) segment	Sep. 30, 2022 USD ($)	Dec. 31, 2022 USD ($)
Significant Accounting Policies [Line Items]
Number of operating segment \| segment			1
Product revenues, net	$ 8,556,000	$ 0	$ 8,556,000	$ 0
Inventory impairment adjustment	0		0		$ 0
Inventory under FIFO	$ 5,300,000		$ 5,300,000
Maximum
Significant Accounting Policies [Line Items]
Payment term threshold	60 days		60 days
Minimum
Significant Accounting Policies [Line Items]
Payment term threshold	30 days		30 days
Laboratory equipment
Significant Accounting Policies [Line Items]
Recognized impairment losses for long lived assets	$ 0	$ 0	$ 0	$ 0

Revenue Recognition - Allowances And Discounts (Details) $ in Thousands	3 Months Ended
	Sep. 30, 2023 USD ($)
Accounts Receivable, Allowance For Credit Loss [Roll Forward]
Balance as of June 30, 2023	$ 0
Provision	1,192
Payments/Credits	(5)
Balance, as of September 30, 2023	1,187
Rebates
Accounts Receivable, Allowance For Credit Loss [Roll Forward]
Balance as of June 30, 2023	0
Provision	920
Payments/Credits	0
Balance, as of September 30, 2023	920
Prompt Pay
Accounts Receivable, Allowance For Credit Loss [Roll Forward]
Balance as of June 30, 2023	0
Provision	195
Payments/Credits	(5)
Balance, as of September 30, 2023	190
Other Accruals
Accounts Receivable, Allowance For Credit Loss [Roll Forward]
Balance as of June 30, 2023	0
Provision	78
Payments/Credits	0
Balance, as of September 30, 2023	$ 78

Organization	9 Months Ended
Organization	Sep. 30, 2023
Accounting Policies [Abstract]
Organization	Organization Krystal Biotech, Inc. (the “Company,” or “we” or other similar pronouns) commenced operations in April 2016. In March 2017, the Company converted from a California limited liability company to a Delaware C-corporation, and changed its name from Krystal Biotech LLC to Krystal Biotech, Inc. In June 2018, the Company incorporated a wholly-owned subsidiary in Australia for the purpose of undertaking preclinical and clinical studies in Australia. In April 2019, the Company incorporated Jeune Aesthetics, Inc. (“Jeune Aesthetics”), in Delaware, a wholly-owned subsidiary, for the purpose of undertaking preclinical and clinical studies for aesthetic skin conditions. In January 2022, August 2022, December 2022, and August 2023, the Company incorporated wholly-owned subsidiaries in Switzerland, Netherlands, France, and Germany, respectively, for the purpose of establishing initial operations in Europe for the commercialization of the Company’s product pipeline. We are a commercial-stage biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat diseases with high unmet medical needs. Our approach leverages our patented platform that is based on an engineered Herpes Simplex Virus-1 (“HSV-1”) vector to deliver therapeutic transgenes to cells of interest in multiple organ systems. The cell’s own machinery then transcribes and translates the transgene to treat the disease. We formulate our vectors for non-invasive or minimally invasive routes of administration at a healthcare professional’s office or in the patient’s home by a healthcare professional. Our innovative technology platform is supported by two in-house, commercial scale Current Good Manufacturing Practices ("CGMP") manufacturing facilities. On May 19, 2023, the Company received U.S. Food and Drug Administration (“FDA”) approval for its first product, VYJUVEK® (“VYJUVEK”) for the treatment of Dystrophic Epidermolysis Bullosa (“DEB”) in patients six months or older. Additionally, the Company received a Rare Pediatric Disease Priority Review Voucher (“PRV”) in connection with the VYJUVEK approval. VYJUVEK became commercially available upon approval, and we began generating revenue from VYJUVEK product sales in 3Q 2023. Liquidity As of September 30, 2023, the Company had an accumulated deficit of $278.5 million. As the Company continues to incur operating losses, a transition to operating profitability is dependent upon the successful commercialization of VYJUVEK as well as successful development, approval, and commercialization of its other product candidates and the achievement of a level of revenues adequate to support the Company’s cost structure. The Company may never achieve operating profitability and unless and until it does, the Company will continue to need to raise additional capital or obtain financing from other sources. Management intends to fund future operations through its on hand cash and cash equivalents, revenue generated from the sale of VYJUVEK, the sale of equity, debt financings, and may also seek additional capital through arrangements with strategic partners or other sources. There can be no assurance that additional funding will be available on terms acceptable to the Company, if at all. The Company is subject to risks common to companies in the biotechnology industry, including but not limited to the failure of product candidates in clinical and preclinical studies, the development of competing product candidates or other technological innovations by competitors, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to commercialize product candidates. The Company expects to incur significant costs to further its pipeline and to expand its commercialization capabilities in advance of the potential global regulatory approvals of VYJUVEK. The Company believes that its cash, cash equivalents and short-term investments of approximately $562.1 million as of September 30, 2023 will be sufficient to allow the Company to fund its planned operations for at least the next 12 months from the date of this Quarterly Report on Form 10-Q.

Stock-Based Compensation

9 Months Ended

Sep. 30, 2023

Share-Based Payment Arrangement [Abstract]

Stock-Based Compensation

In 2017, the Company adopted the 2017 IPO Stock Plan (the “Plan”), which governs the issuance of stock options and restricted stock to employees, certain non-employee consultants, and directors. Initially, the Company reserved 900 thousand shares for issuance under the Plan with an initial sublimit for incentive stock options of 900 thousand shares. On an annual basis, the amount of shares available for issuance under the Plan increases by an amount equal to four percent of the total outstanding shares as of the last day of the preceding calendar year. The sublimit of incentive stock options is not subject to the increase. The Company has historically granted stock options and restricted stock awards to its employees. In February 2023, the Company began issuing restricted stock units and performance-based restricted stock units to certain employees.

Stock Options

Options granted to employees and non-employees vest ratably over a four-year period and stock options granted to directors of the Company vest ratably over one-year to three-year periods. Stock options have a life of ten years.

The Company granted 30,500 and 419,780 stock options to employees, non-employees, and directors of the Company during the three and nine months ended September 30, 2023 and 189,000 and 1,958,000 to employees, non-employees, and directors of the Company during the three and nine months ended September 30, 2022, respectively.

The following table summarizes the Company’s stock option activity:


	Stock Options Outstanding	Weighted- average Exercise Price		Weighted- average Remaining Contractual Life (Years)	Aggregate Intrinsic Value (In thousands) (1)
Outstanding at December 31, 2022	3,582,181	$	61.50	8.7	$	64,880
Granted	419,780	$	91.38
Exercised	(710,734)	$	57.98
Cancelled or forfeited	(623,967)	$	64.19
Expired	—	$	—
Outstanding at September 30, 2023	2,667,260	$	66.04	8.2	$	133,648
Exercisable at September 30, 2023	707,277	$	55.51	7.1	$	42,793

(1)Aggregate intrinsic value represents the difference between the closing stock price of our common stock on September 30, 2023 and the exercise price of outstanding in-the-money options.

The total intrinsic value (the amount by which the fair market value exceeds the exercise price) of stock options exercised during the three and nine months ended September 30, 2023 was $13.5 million and $41.4 million, respectively, and during the three and nine months ended September 30, 2022 was $1.3 million and $2.1 million, respectively.

The weighted-average grant-date fair value per share of options granted to employees, non-employees, and directors during the three and nine months ended September 30, 2023 was $85.93 and $62.87, respectively, and during the three and nine months ended September 30, 2022 was $49.59 and $43.66, respectively.

There was $77.5 million of unrecognized stock-based compensation expense related to employees', non-employees', and directors’ option awards that is expected to be recognized over a weighted-average period of 2.6 years as of September 30, 2023.

The Company has recorded stock-based compensation expense related to the issuance of stock option awards in the condensed consolidated statements of operations for the three and nine months ended September 30, 2023 and 2022, respectively, as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022

Research and development	$	2,033	$	2,184	$	6,858	$	5,547
Selling, general, and administrative	4,796		6,433		18,881		16,791
Total stock-based compensation	$	6,829	$	8,617	$	25,739	$	22,338

The fair value of options was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions for the three and nine months ended September 30, 2023 and 2022:


	Three Months Ended September 30,						Nine Months Ended September 30,
	2023			2022			2023			2022
Expected stock price volatility	74		%	78		%	73		%	78		%
Expected term of the award (years)	6.2			6.2			6.0			6.2
Risk-free interest rate	4.26		%	3.20		%	3.94		%	2.27		%
Weighted average exercise price	$	125.06		$	70.91		$	91.38		$	63.03
Forfeiture rate	—		%	—		%	—		%	—		%
Dividend yield	—		%	—		%	—		%	—		%

Restricted Stock Awards

Restricted stock awards ("RSAs") granted to employees vest ratably over a four-year period. The Company granted zero RSAs to employees of the Company during each of the three and nine months ended September 30, 2023 and September 30, 2022, respectively.


	Number of Shares		Weighted Average Grant Date Fair Value
Non-vested RSAs as of December 31, 2022	66,600		$	78.89
Granted	—		$	—
Vested	(12,649)		$	78.89
Surrendered for taxes	(9,551)		$	78.89
Non-vested RSAs as of September 30, 2023	$	44,400	$	78.89

There was $2.5 million of unrecognized stock-based compensation expense related to employees’ RSAs that is expected to be recognized over a weighted-average period of 1.4 years as of September 30, 2023.

The Company recorded stock-based compensation expense related to RSAs in the condensed consolidated statement of operations for the three and nine months ended September 30, 2023 and 2022, as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Selling, general, and administrative	$	437	$	441	$	1,305	$	1,340
Total stock-based compensation	$	437	$	441	$	1,305	$	1,340

Restricted Stock Units

Restricted stock units (“RSUs”) granted to employees vest ratably over a four-year period. The Company granted zero and 186,900 RSUs to employees of the Company during the three and nine months ended September 30, 2023, respectively, and zero RSUs during the three and nine months ended September 30, 2022, respectively.


	Number of Shares	Weighted Average Grant Date Fair Value
Non-vested RSUs as of December 31, 2022	—
Granted	186,900	$	81.91
Vested	—
Surrendered or forfeited	(24,700)	$	81.91
Non-vested RSUs as of September 30, 2023	162,200	$	81.91

There was $11.3 million of unrecognized stock-based compensation expense related to employees’ RSU awards that is expected to be recognized over a weighted-average period of 3.4 years as of September 30, 2023.

The Company recorded stock-based compensation expense related to RSUs in the condensed consolidated statement of operations for the three and nine months ended September 30, 2023 and 2022, as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Research and Development	$	303	$	—	$	837	$	—
Selling, general, and administrative	370		—		998		—
Total stock-based compensation	$	673	$	—	$	1,835	$	—

Performance-Based Restricted Stock Units

Performance-based restricted stock units (“PSUs”) granted to employees vest ratably over two years based upon continued service through the vesting date and the achievement of specific regulatory and commercial performance criteria as determined by the Compensation Committee of the Company’s Board of Directors. The performance criteria are to be completed by the end of the year in which the PSU awards were granted. Each PSU represents the right to receive one share of the Company's common stock upon vesting. The Company recognizes stock-based compensation expense for the fair value of the PSU awards relating to the portion of the awards that are probable of vesting over the service period. On a quarterly basis, management estimates the probable number of PSU’s that would vest until such time that the ultimate achievement of the performance criteria are known. As of September 30, 2023, the Company estimates that 100% of the PSUs granted will be eligible to vest.

The Company granted zero and 60,000 PSUs to employees of the Company during the three and nine months ended September 30, 2023 and zero PSUs during the three and nine months ended September 30, 2022.


	Number of Shares	Weighted Average Grant Date Fair Value
Non-vested PSUs as of December 31, 2022	—
Granted	60,000	$	81.91
Vested	—
Surrendered or forfeited	(10,000)	$	81.91
Non-vested PSUs as of September 30, 2023	50,000	$	81.91

There was $2.9 million of unrecognized stock-based compensation expense related to employees’ PSU awards that is expected to be recognized over a weighted-average period of 1.4 years as of September 30, 2023.

The Company recorded stock-based compensation expense related to PSUs in the condensed consolidated statement of operations for the three and nine months ended September 30, 2023 and 2022 as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Selling, general, and administrative	$	373	$	—	$	1,201	$	—
Total stock-based compensation	$	373	$	—	$	1,201	$	—

Shares remaining available for grant under the Plan were 1,489,488, with a sublimit for incentive stock options of 30,783, at September 30, 2023.

After the FDA approval of VYJUVEK on May 19, 2023, the Company began capitalizing stock-based compensation associated with the allocation of labor costs related to work performed to manufacture VYJUVEK. For the three and nine months ended September 30, 2023, the Company capitalized $410 thousand and $522 thousand, respectively, in inventory.

Historically, the Company also capitalized the portion of stock-based compensation related to work performed on the construction of our manufacturing facilities. There was zero and $162 thousand of stock-based compensation that was capitalized in property and equipment during the three and nine months ended September 30, 2023 and $137 thousand and $423 thousand of stock-based compensation that was capitalized in property and equipment during the three and nine months ended September 30, 2022, respectively.

Summary of Significant Accounting Policies - Summary of Estimated Useful Lives of Assets (Detail)	Sep. 30, 2023
Building and building improvements \| Minimum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	7 years
Building and building improvements \| Maximum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	47 years
Computer equipment and software \| Minimum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	3 years
Computer equipment and software \| Maximum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	7 years
Manufacturing equipment \| Minimum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	3 years
Manufacturing equipment \| Maximum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	20 years
Laboratory equipment \| Minimum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	3 years
Laboratory equipment \| Maximum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	15 years
Furniture and fixtures \| Minimum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	3 years
Furniture and fixtures \| Maximum
Property, Plant and Equipment [Line Items]
Estimated useful lives of assets	7 years

Net Income (Loss) Per Share Attributable to Common Stockholders - Additional Information (Detail) - shares	3 Months Ended	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2023	Sep. 30, 2022
Stock Options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Common share equivalents outstanding, excluded from the calculation of diluted net loss per common share	378,299	1,277,313
Stock Options and Unvested Restricted Stocks Awards
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Common share equivalents outstanding, excluded from the calculation of diluted net loss per common share			3,565,110

Cover - shares	9 Months Ended
Cover - shares	Sep. 30, 2023	Oct. 30, 2023
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2023
Document Transition Report	false
Entity File Number	001-38210
Entity Registrant Name	Krystal Biotech, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	82-1080209
Entity Address, Address Line One	2100 Wharton Street
Entity Address, Address Line Two	Suite 701
Entity Address, City or Town	Pittsburgh
Entity Address, State or Province	PA
Entity Address, Postal Zip Code	15203
City Area Code	412
Local Phone Number	586-5830
Title of 12(b) Security	Common Stock
Trading Symbol	KRYS
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		28,206,330
Amendment Flag	false
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q3
Entity Central Index Key	0001711279
Current Fiscal Year End Date	--12-31

Revenue Recognition (Tables)

9 Months Ended

Sep. 30, 2023

Revenue from Contract with Customer [Abstract]

Changes in Allowances and Discounts

The following table summarizes changes in allowances and discounts for the three months ended September 30, 2023 (in thousands):


	Rebates		Prompt Pay		Other Accruals		Total
Balance as of June 30, 2023	$	—	$	—	$	—	$	—
Provision	920		195		78		1,192
Payments/Credits	—		(5)		—		(5)
Balance, as of September 30, 2023	$	920	$	190	$	78	$	1,187

Balance Sheet Components - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Balance Sheet Related Disclosures [Abstract]
Depreciation expense	$ 1,200	$ 669	$ 3,500	$ 1,600

Gain from Sale of Priority Review Voucher	9 Months Ended
Gain from Sale of Priority Review Voucher	Sep. 30, 2023
Property, Plant and Equipment [Abstract]
Gain from Sale of Priority Review Voucher	Gain from Sale of Priority Review VoucherIn August 2023, the Company entered into an agreement to sell the rare pediatric disease voucher (“PRV”), which was awarded to the Company in connection with the FDA’s approval of VYJUVEK. The transaction closed in August 2023 and was not subject to any commissions or closing costs. The proceeds of $100.0 million from the sale of the PRV were recorded as a gain from sale of priority review voucher on the Company’s condensed consolidated statement of operations as it did not have a carrying value at the time of the sale.

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2022	Jun. 30, 2022	Mar. 31, 2022	Sep. 30, 2023	Sep. 30, 2022
Pay vs Performance Disclosure
Net income (loss)	$ 80,747	$ (33,210)	$ (45,297)	$ (29,850)	$ (28,108)	$ (49,965)	$ 2,240	$ (107,923)

- Definition

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

+ References

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-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-04(22))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483589/942-220-S99-1

+ Details

Revenue Recognition - Narrative (Details) - USD ($)	3 Months Ended		9 Months Ended
Revenue Recognition - Narrative (Details) - USD ($)	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022	Dec. 31, 2022
Revenue from Contract with Customer [Abstract]
Product revenues, net	$ 8,556,000	$ 0	$ 8,556,000	$ 0
Accounts receivable, net	$ 9,316,000		$ 9,316,000		$ 0

- Definition

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 924
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 11.L)
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-URI https://asc.fasb.org//1943274/2147479941/924-10-S99-1

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-Section 50
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org//1943274/2147479806/606-10-50-4

+ Details

Fair Value Instruments (Tables)

9 Months Ended

Sep. 30, 2023

Fair Value Disclosures [Abstract]

Summary of Cash, Cash Equivalents and Available-for-Sale Securities


	September 30, 2023

	Amortized Cost		Gross Unrealized Gains		Gross Unrealized (Losses)		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash and cash equivalents	$	373,241	$	—	$	—	$	373,241	$	373,241	$	—	$	—
Subtotal	373,241		—		—		373,241		373,241		—		—
Level 2:

Commercial paper	43,496		1		(9)		43,488		—		43,488		—
Corporate bonds	70,465		4		(201)		70,268		—		56,463		13,805
U.S. government agency securities	111,635		225		(240)		111,620		—		88,877		22,743
Subtotal	225,596		230		(450)		225,376		—		188,828		36,548
Total	$	598,837	$	230	$	(450)	$	598,617	$	373,241	$	188,828	$	36,548


	December 31, 2022
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized (Losses)		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash and cash equivalents	$	161,900	$	—	$	—	$	161,900	$	161,900	$	—	$	—

Subtotal	161,900		—		—		161,900		161,900		—		—
Level 2:
Commercial paper	63,624		5		(23)		63,606		—		63,606		—
Corporate bonds	82,241		13		(419)		81,835		—		77,214		4,621
U.S. government agency securities	76,683		161		(393)		76,451		—		76,451		—



Subtotal	222,548		179		(835)		221,892		—		217,271		4,621
Total	$	384,448	$	179	$	(835)	$	383,792	$	161,900	$	217,271	$	4,621

(1)The Company’s short-term marketable securities mature in one year or less.

(2)The Company's long-term marketable securities mature between one year and two years.

Balance Sheet Components (Tables)

9 Months Ended

Sep. 30, 2023

Balance Sheet Related Disclosures [Abstract]

Schedule of Property and Equipment, Net

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


	September 30, 2023		December 31, 2022

Building and building improvements	72,193		—
Construction in progress	$	56,074	$	131,331
Leasehold improvements	24,835		24,217
Manufacturing equipment	16,827		9,783
Laboratory equipment	2,339		2,089
Furniture and fixtures	1,518		957
Computer equipment and software	982		100
Total property and equipment	174,768		168,477
Accumulated depreciation	(10,739)		(6,793)
Property and equipment, net	$	164,029	$	161,684

Schedule of Accrued Expenses and Other Current Liabilities

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


	September 30, 2023		December 31, 2022



Accrued construction in progress	$	7,548	$	11,452
Accrued payroll and benefits	7,033		6,781
Accrued professional fees	2,786		3,397
Other current liabilities	2,315		267
Accrued preclinical and clinical expenses	1,908		1,365

Accrued taxes	152		43
Total	$	21,742	$	23,305

Capitalization	9 Months Ended
Capitalization	Sep. 30, 2023
Equity [Abstract]
Capitalization	Capitalization ATM Program On December 31, 2020, the Company entered into a sales agreement with Cowen and Company, LLC ("Cowen") with respect to an at-the-market equity offering program ("2020 ATM Program"), under which the Company issued and sold from time to time through Cowen, acting as agent and/or principal, shares of its common stock, par value $0.0001 per share, having an aggregate offering price up to $150.0 million ("Placement Shares"). The issuance and sale of the Placement Shares were made pursuant to the Company's effective "shelf" registration statement on Form S-3 that was filed with the Securities and Exchange Commission (the “SEC”) on May 4, 2020 (the “2020 Shelf Registration Statement”). During the nine months ended September 30, 2022, the Company issued and sold 434,782 Placement Shares at a weighted average price of $69.00 per share for net proceeds of $29.1 million after deducting selling commissions of approximately $900 thousand. The Company’s 2020 Shelf Registration Statement expired on May 4, 2023, and the Company put in place a new at-the-market equity offering program under substantially the same terms as the 2020 ATM Program (the “New ATM Program”). Accordingly, on May 8, 2023, the Company entered into a new sales agreement with Cowen to issue and sell shares of the Company’s common stock having an aggregate offering price of up to $150.0 million (the “New Placement Shares”) from time to time, under which Cowen will act as the Company’s agent and/or principal. The New Placement Shares will be offered and sold pursuant to the Company’s effective shelf registration statement on Form S-3 filed with the SEC on April 6, 2023, and a prospectus supplement relating to the New Placement Shares that was filed with the SEC on May 8, 2023. During the quarter ended September 30, 2023 no shares of common stock were issued pursuant to the New ATM Program, resulting in $150.0 million available for issuance under the New ATM Program. 2023 Private Placement Offering On May 22, 2023 and May 23, 2023, the Company sold 1,720,100 and 9,629 shares of common stock, respectively, in a private placement to certain institutional investors at a price of $92.50 per share for aggregate net proceeds of $160.0 million. In addition, the Company entered into a Registration Rights Agreement with the investors (“Registration Rights Agreement”) that required the Company to file a registration statement with the SEC within 60 days of the date of the Registration Rights Agreement registering the resale of the shares of common stock issued in the private placement. On July 18, 2023, the Company filed the resale registration statement on Form S-3ASR with the SEC, which became effective upon filing.

- Definition

The entire disclosure for equity.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481112/505-10-50-13

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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-Subparagraph (a)
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 815
-SubTopic 40
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Subparagraph (a)
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-URI https://asc.fasb.org//1943274/2147480237/815-40-50-6

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.3-04)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480008/505-10-S99-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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-Paragraph 1
-Subparagraph (SX 210.4-08(e)(1))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480678/235-10-S99-1

Reference 10: http://fasb.org/us-gaap/role/ref/legacyRef
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-URI https://asc.fasb.org//505/tableOfContent

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
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-Paragraph 13
-Subparagraph (g)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481112/505-10-50-13

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

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
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Reference 15: http://www.xbrl.org/2003/role/disclosureRef
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-Topic 505
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
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Reference 18: http://www.xbrl.org/2003/role/disclosureRef
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Reference 19: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org//1943274/2147481112/505-10-50-18

+ Details

Summary of Significant Accounting Policies

9 Months Ended

Sep. 30, 2023

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

Basis of Presentation

The accompanying interim condensed consolidated financial statements have been prepared in conformity with generally accepted accounting principles in the United States of America (“GAAP”). In the opinion of management, all adjustments, which consist of all normal recurring adjustments necessary for a fair presentation of the Company's financial position and results of operations for the interim periods presented, are reflected in the interim condensed consolidated financial statements. All intercompany balances and transactions have been eliminated in consolidation.

The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year. These unaudited interim condensed consolidated financial statements should be read in conjunction with the Company’s audited consolidated financial statements and the notes thereto included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022, as filed with the U.S. Securities and Exchange Commission (“SEC”) on February 27, 2023.

Use of Estimates

The preparation of condensed consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts in the condensed consolidated financial statements and accompanying notes. Actual results could materially differ from those estimates. Management considers many factors in developing the estimates and assumptions that are used in the preparation of these financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates in the period these variances become known. Estimates are used in the following areas, among others: variable consideration associated with revenue recognition, stock-based compensation expense, accrued expenses, and the valuation allowance included in the deferred income tax calculation.

Segment and Geographical Information

Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company and the Company’s chief operating decision maker view the Company’s operations and manage its business in one operating segment, which is the business of developing and commercializing pharmaceutical products.

Cash, Cash Equivalents and Investments

Cash and cash equivalents consist of money market funds and bank deposits. Cash equivalents are defined as short-term, highly liquid investments with original maturities of 90 days or less at the date of purchase.

Investments with maturities of less than one year are classified as short-term investments on the condensed consolidated balance sheets and consist of commercial paper, corporate bonds, and U.S. government agency securities. Investments with maturities of greater than one year are classified as long-term investments on the condensed consolidated balance sheets and consist of corporate bonds and government agency securities. Accrued interest on investments is also classified as short-term investments on the condensed consolidated balance sheets.

As our entire investment portfolio is considered available for use in current operations, we classify all investments as available-for-sale securities. Available-for-sale securities are carried at fair value, with unrealized gains and losses reported in accumulated other comprehensive loss, which is a separate component of stockholders’ equity in the condensed consolidated balance sheets. Any premium arising at purchase is amortized to the earliest call date and any discount arising at purchase is accreted to maturity. Amortization and accretion of premiums and discounts are recorded in interest and other income, net, in the condensed consolidated statements of operations and comprehensive income (loss).

Fair Value of Financial Instruments

Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. There is a three-level hierarchy that prioritizes the inputs used in determining fair value by their reliability and preferred use, as follows:

•Level 1— Valuations based on quoted prices in active markets for identical assets or liabilities.

•Level 2— Valuations based on quoted prices in active markets for similar assets and liabilities, quoted prices for identical or similar assets or liabilities in inactive markets, or other inputs that are observable or can be corroborated by observable market data.

•Level 3— Valuations based on inputs that are both significant to the fair value measurement and unobservable.

To the extent that a valuation is based on models or inputs that are less observable, or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized within Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.

There have been no significant changes to the valuation methods utilized by the Company during the periods presented. There have been no transfers between Level 1, Level 2, and Level 3 in any periods presented.

The carrying amounts of financial instruments consisting of cash and cash equivalents, investments, accounts receivable, net, prepaid expenses and other current assets, accounts payable, accrued expenses and other current liabilities included in the Company’s condensed consolidated financial statements, approximate fair value, primarily due to their short maturities. Our available-for-sale, short-term and long-term investments are considered to be Level 2 financial instruments.

Revenue Recognition

The Company sells VYJUVEK to a limited number of specialty pharmacies (“SPs”) that mix the medication and administer it to patients in the patient’s home by a healthcare professional and a single specialty distributor (“SD”), that distributes VYJUVEK to hospitals and outpatient clinics where patients are administered the medication at a healthcare professional’s office.

The Company recognizes product revenue under ASC Topic 606, Revenue from Contracts with Customers (“Topic 606”). Under Topic 606, the Company is required to complete the following five steps:

(i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price, including variable consideration, if any; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation.

The Company recognizes revenue when the customer obtains control of the product, which occurs at a point in time, upon delivery to the customer. Sales and other taxes collected concurrent with revenue-producing activities are excluded from revenue. Revenue is measured as the amount of consideration the Company expects to receive in exchange for transferring VYJUVEK and is generally based upon a list or fixed price less allowances for returns, rebates and discounts. The Company’s payment terms are generally 30 to 60 days from the invoice date.

Variable Consideration

Product revenues is recorded at the net sales price, or transaction price, upon delivery and transfer of control to the customer, and includes an estimate of variable consideration, which results from discounts, rebates, and returns that are offered within contracts between the Company and its customers.

–Prompt Pay Discounts: As an incentive for prompt payment, the Company offers a cash discount to customers. The Company estimates accrued prompt pay discounts using the most likely amount method. The Company expects that all eligible customers will comply with the contractual terms to earn the discount. The Company records the discount as an allowance against accounts receivable, net and a reduction of revenue.

–Government Rebates: The Company participates in certain government rebate programs including Medicaid. The Company estimates accrued government rebates using the expected value method. The Company accrues estimated rebates based on estimated percentages of VYJUVEK prescribed to qualified patients, estimated rebate percentages and estimated levels of inventory in the distribution channel that will be prescribed to qualified patients and records the rebates as a reduction of revenue. Accrued government rebates are recorded as a reduction of revenue and are included in other accrued liabilities on the condensed consolidated balance sheets.

–Commercial Rebates: The Company participates in certain commercial rebate programs. Under these rebate programs, the Company pays a rebate to the commercial entity or third-party administrator of the program. Accrued commercial rebates are estimated using the expected value method. The Company accrues estimated rebates based on contract prices, estimated percentages of VYJUVEK that will be prescribed to qualified patients and estimated levels of inventory in the distribution channel. Accrued commercial rebates are recorded as a reduction of revenue and are included in other accrued liabilities on the condensed consolidated balance sheets.

–Copay Assistance: The Company provides copay assistance to qualified patients, helping them meet copay obligations to their insurance provider. The Company reimburses pharmacies for this discount through third-party vendors. The Company estimates copay assistance costs using the expected value method. The estimate is based on contract prices, estimated percentages of VYJUVEK that will be prescribed to qualified patients, average assistance paid based on reporting from third-party vendors and estimated levels of inventory in the distribution channel. Copay assistance costs are recorded as reductions to revenue and are accrued in other accrued liabilities on the condensed consolidated balance sheets.

–Returns: The Company offers SPs and SDs limited return rights relating to product damage or defect and based on these provisions, the Company believes that there will be minimal returns.

Variable consideration is estimated and reduces the transaction price to reflect the Company’s best estimate of the amount of consideration to which the Company is entitled based on the terms of the contracts and are recorded in the same period the related product revenues is recognized. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates in the period these variances become known.

Cost of Goods Sold

Cost of goods sold includes direct and indirect costs related to the manufacturing of VYJUVEK. These costs consist of manufacturing costs, personnel costs including stock-based compensation, facility costs, and other indirect overhead costs. Cost of goods sold may also include period costs related to certain manufacturing services and inventory adjustment charges.

Accounts Receivable

Accounts receivable is recorded net of allowances for prompt payment discounts, returns, and credit losses. The Company estimates an allowance for credit losses by considering factors such as credit quality, the age of the accounts receivable balances, and current economic conditions that may affect a customer’s ability to pay. As of September 30, 2023, the credit profiles for the Company’s customer was deemed to be in good standing, and as such an allowance for credit losses was not recorded.

Concentration of Credit Risk and Off-Balance Sheet Risk

Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents, short-term investments, long-term investments, and accounts receivable, net. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company is exposed to credit risk in the event of default by the financial institutions to the extent amounts recorded on the condensed consolidated balance sheets are in excess of insured limits. The Company has not experienced any credit losses in such accounts and does not believe it is exposed to any significant credit risk on these funds. The Company’s accounts receivable, net and marketable securities, which primarily consist of U.S. government agency securities and treasuries, equity securities, corporate bonds and commercial paper, potentially subject the Company to concentrations of credit risk. The Company had one customer for the three and nine months ended September 30, 2023 and no product revenues for the three and nine months ended September 30, 2022. The Company has no financial instruments with off-balance sheet risk of loss.

Inventories

The Company capitalizes inventory costs associated with products when future economic benefit is expected to be realized. These costs consist of raw materials, manufacturing-related costs, personnel costs including stock-based compensation, facility costs, and other indirect overhead costs. Prior to receiving FDA approval for VYJUVEK in May 2023, the Company expensed costs related to inventory for clinical and pre-commercial purposes directly to research and development expense. Following the FDA’s approval of VYJUVEK, the Company began capitalizing inventory related to commercialized products held for sale, in-process of production for sale, and raw materials to be used in the manufacturing of inventory.

The Company values its inventories at the lower-of-cost and net realizable value, on a first-in, first-out (“FIFO”) basis. The Company adjusts the net realizable value of any excess, obsolete or unsalable inventories in the period in which an impairment is identified. For the three and nine months ended September 30, 2023 and 2022, there were no inventory impairment adjustments. As of September 30, 2023, the Company recorded $5.3 million of inventory, consisting of raw materials, work-in-process, and finished goods within inventory on the Company’s condensed consolidated balance sheets.

Property and Equipment, net

Property and equipment, net, is stated at cost, less accumulated depreciation. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred, while costs of major additions and betterments are capitalized. Upon disposal, the related cost and accumulated depreciation is removed and any resulting gain or loss is included in the results of operations. Depreciation is recorded using the straight-line method over the estimated useful lives of the respective assets, which are as follows:


Buildings and building improvements			7 - 47 years
Computer equipment and software			3 - 7 years
Manufacturing equipment			3 - 20 years
Laboratory equipment			3 - 15 years
Furniture and fixtures			3 - 7 years
Leasehold improvements			lesser of useful life or remaining life of lease

The Company reviews the estimated useful lives of its property and equipment on a continuing basis. In evaluating the useful lives, the Company considers how long assets will remain functionally effective, whether the technology continues to be relevant and considers other competitive and economic factors. If the assessment indicates that the assets will be used for a shorter or longer period than previously anticipated, the useful life of the assets is adjusted, resulting in a change in estimate. Changes in estimates are accounted for on a prospective basis by depreciating the current carrying values of the assets over their revised remaining useful lives.

Construction in progress is not depreciated until the asset is placed in service.

Impairment of Long-Lived Assets

The Company evaluates long-lived assets for potential impairment when events or changes in circumstances indicate the carrying value of the assets may not be recoverable. We review the recoverability of the net book value of long-lived assets whenever events and circumstances indicate ("triggering events") that the net book value of an asset may not be recoverable. In cases where a triggering event occurs and undiscounted expected future cash flows are less than the net book value, we recognize an impairment loss equal to an amount by which the net book value exceeds the fair value of the asset. Long-lived assets to be disposed of are reported at the lower of carrying amount or fair value less cost to sell. The Company has not identified any triggering events or recognized any impairment losses for the three and nine months ended September 30, 2023 and 2022.

Leases

The Company accounts for its lease agreements in accordance with Financial Accounting Standards Board (“FASB”) Accounting Standards Codification, or ASC, Topic 842, Leases. Right-of-use lease assets represent the right to use an underlying asset during the lease term and the lease liabilities represent the commitment to make lease payments arising from the lease. Right-of-use lease assets and obligations are recognized based on the present value of remaining lease payments over the lease term. As the Company’s existing lease agreements do not provide an implicit rate and as the Company does not have any external borrowings, the Company has used an estimated incremental borrowing rate based on the information available at lease commencement in determining the present value of lease payments. Operating lease expense is recognized on a straight-line basis over the lease term. Variable lease expense is recognized in the period in which the obligation for the payment is incurred. In addition, the Company also has made an accounting policy election to exclude leases with an initial term of twelve months or less from its condensed consolidated balance sheets and to account for lease and non-lease components of its operating leases as a single component.

Research and Development Expenses

Research and development costs are charged to expense as incurred in performing research and development activities. These costs include employee compensation costs, facilities and overhead, preclinical and clinical activities, clinical manufacturing costs, contract management services, regulatory and other related costs.

The Company estimates contract research and manufacturing expenses based on the services performed pursuant to contracts with research organizations and manufacturing organizations that manufacture materials used in the Company’s ongoing preclinical and clinical studies. Non-refundable advanced payments for goods or services to be received in the future for use in research and development activities are capitalized within prepaid expenses and other current assets on the condensed consolidated balance sheets. The capitalized amounts are expensed as the related goods are delivered or the services are performed.

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. These estimates are based on communications with third-party service providers and the Company’s estimates of accrued expenses using information available at each balance sheet date. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company will adjust the accrual accordingly.

Stock-Based Compensation Expense

The Company applies the fair value recognition provisions of FASB, ASC Topic 718, Compensation—Stock Compensation ("ASC 718"), to account for stock-based compensation. Compensation costs related to equity awards granted are based on the estimated fair value of the awards on the date of grant.

ASC 718 requires all stock-based payments, including grants of stock options and restricted stock, to be recognized in the consolidated statements of operations based on their grant-date fair values. Compensation expense for stock options, restricted stock awards, and restricted stock units is recognized on a straight-line basis based on the grant-date fair value over the associated service period of the award, which is generally the vesting term. Compensation expense for performance-based restricted stock units is recognized for the awards that are probable of vesting over the service period of the award. On a quarterly basis, management estimates the probable number of performance-based restricted stock units that would vest until such time that the ultimate achievement of the performance criteria are known.

The Company estimates the fair value of its stock options using the Black-Scholes option pricing model, which requires the input of subjective assumptions, including: (i) the expected stock price volatility; (ii) the expected term of the award; (iii) the risk-free interest rate; and (iv) expected dividends.

The Company estimates stock price volatility by using its own historical data. The expected term of the Company’s stock options is estimated using the “simplified” method, whereby the expected term equals the arithmetic mean of the vesting term and the original contractual term of the option. The risk-free interest rates are based on U.S. Treasury securities with a maturity date commensurate with the expected term of the associated award. The Company has never paid and does not expect to pay dividends in the foreseeable future. The Company accounts for forfeitures as they occur. Stock-based compensation expense recognized in the financial statements is based on awards for which service conditions are expected to be satisfied.

Comprehensive Income (Loss)

Comprehensive income (loss) is defined as the change in equity during a period from transactions from non-owner sources. Unrealized gains or losses on available-for-sale securities and foreign currency translation are components of other comprehensive gains or losses and are presented net of taxes. We record reclassifications from other comprehensive gains or losses to interest and other income, net on the condensed consolidated statements of operations related to realized gains on sales of available-for-sale securities.

The Company reviews its securities quarterly to determine whether an other-than-temporary impairment has occurred. The Company determined that there were no other-than-temporary impairments during the three and nine months ended September 30, 2023 and 2022.

Recent Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the FASB that the Company adopts as of the specified effective date. There were no recently adopted accounting pronouncements that had a material impact on the Company’s condensed consolidated financial statements, and no recently issued accounting pronouncements that are expected to have a material impact on the Company’s condensed consolidated financial statements.

Leases - Lease expense (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Leases - Lease expense (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Leases [Abstract]
Operating lease expense	$ 379	$ 399	$ 1,282	$ 1,200
Variable lease expense	62	48	150	168
Total lease expense	$ 441	$ 447	$ 1,432	$ 1,368

Commitments and Contingencies - Additional Information (Detail)				3 Months Ended		9 Months Ended
	Jun. 15, 2023 USD ($)	Apr. 28, 2022 USD ($)	Mar. 12, 2022 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($) milestone	Sep. 30, 2022 USD ($)
Operating Leased Assets [Line Items]
Litigation settlement				$ 0	$ 0	$ 12,500,000	$ 25,000,000
Proceeds from insurance settlement, operating activities				0	0	0	768,000
PeriphaGen
Operating Leased Assets [Line Items]
Litigation settlement	$ 12,500,000	$ 25,000,000	$ 37,500,000	0	0	$ 12,500,000	25,000,000
Number of milestones \| milestone						3
Litigation settlement, total consideration			75,000,000
PeriphaGen \| Milestone Three
Operating Leased Assets [Line Items]
Litigation settlement, amount awarded to other party						$ 12,500,000
Litigation settlement, milestone payments, sales threshold			300,000,000
PeriphaGen \| Milestone One
Operating Leased Assets [Line Items]
Litigation settlement, amount awarded to other party						12,500,000
Litigation settlement, milestone payments, sales threshold			100,000,000
PeriphaGen \| Milestone Two
Operating Leased Assets [Line Items]
Litigation settlement, amount awarded to other party						12,500,000
Litigation settlement, milestone payments, sales threshold			$ 200,000,000
Clinical Supply Agreements
Operating Leased Assets [Line Items]
Estimated remaining commitment				2,700,000		2,700,000
Project management service fee				1,900,000	$ 2,100,000	5,000,000	$ 5,100,000
ASTRA Facility
Operating Leased Assets [Line Items]
Contractual obligation				$ 11,100,000		$ 11,100,000

Stock-Based Compensation - Fair Value of Stock Options, Valuation Assumptions (Detail) - Stock Options - $ / shares	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected stock price volatility	74.00%	78.00%	73.00%	78.00%
Expected term of the award (years)	6 years 2 months 12 days	6 years 2 months 12 days	6 years	6 years 2 months 12 days
Risk-free interest rate	4.26%	3.20%	3.94%	2.27%
Weighted average exercise price (in dollars per share)	$ 125.06	$ 70.91	$ 91.38	$ 63.03
Forfeiture rate	0.00%	0.00%	0.00%	0.00%
Dividend yield	0.00%	0.00%	0.00%	0.00%

- 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 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 35
-Paragraph 1D
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480483/718-10-35-1D

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 35
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480483/718-10-35-3

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
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-Subparagraph (a)(1)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

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

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)(3)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
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-Paragraph 2
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-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

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-Name Accounting Standards Codification
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-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

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-Paragraph 2
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-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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-Name Accounting Standards Codification
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-Paragraph 2
-Subparagraph (c)(1)(iv)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(1)(iv)(01)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(1)(iv)(02)
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-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(1)(iv)(03)
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-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(1)(iv)(04)
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-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(2)(i)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 50
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Reference 16: http://www.xbrl.org/2003/role/disclosureRef
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
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-Paragraph 2
-Subparagraph (c)(2)(iii)(01)
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Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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-Subparagraph (c)(2)(iii)(02)
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Reference 19: http://www.xbrl.org/2003/role/disclosureRef
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Reference 20: http://www.xbrl.org/2003/role/disclosureRef
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Reference 21: http://www.xbrl.org/2003/role/disclosureRef
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Reference 22: http://www.xbrl.org/2003/role/disclosureRef
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Reference 23: http://www.xbrl.org/2003/role/disclosureRef
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Reference 24: http://www.xbrl.org/2003/role/disclosureRef
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Reference 25: http://www.xbrl.org/2003/role/disclosureRef
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-Subparagraph (f)(2)(ii)
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Reference 26: http://www.xbrl.org/2003/role/disclosureRef
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Reference 27: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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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

Stock-Based Compensation - Schedule of Company's Stock Activity (Details) - $ / shares	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Restricted Stock
Number of Shares
Beginning balance (in shares)			66,600
Granted (in shares)			0	0
Vested (in shares)			(12,649)
Surrendered for taxes (in shares)			(9,551)
Ending balance (in shares)	44,400		44,400
Weighted Average Grant Date Fair Value
Beginning balance (in dollars per share)			$ 78.89
Granted (in dollars per share)			0
Surrendered for taxes (in dollars per share)			78.89
Ending balance (in dollars per share)	$ 78.89		$ 78.89
Restricted Stock Units (RSUs)
Number of Shares
Beginning balance (in shares)			0
Granted (in shares)	0	0	186,900	0
Vested (in shares)			0
Surrendered for taxes (in shares)			(24,700)
Ending balance (in shares)	162,200		162,200
Weighted Average Grant Date Fair Value
Beginning balance (in dollars per share)
Granted (in dollars per share)			81.91
Vested (in dollars per share)
Surrendered for taxes (in dollars per share)			81.91
Ending balance (in dollars per share)	$ 81.91		$ 81.91
Performance Shares
Number of Shares
Beginning balance (in shares)			0
Granted (in shares)	0	0	60,000	0
Vested (in shares)			0
Surrendered for taxes (in shares)			(10,000)
Ending balance (in shares)	50,000		50,000
Weighted Average Grant Date Fair Value
Beginning balance (in dollars per share)
Granted (in dollars per share)			81.91
Vested (in dollars per share)
Surrendered for taxes (in dollars per share)			81.91
Ending balance (in dollars per share)	$ 81.91		$ 81.91

Condensed Consolidated Balance Sheets (unaudited) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Current assets
Cash and cash equivalents	$ 373,241	$ 161,900
Short-term investments	188,828	217,271
Accounts receivable, net	9,316	0
Inventory	5,278	0
Prepaid expenses and other current assets	5,465	4,608
Total current assets	582,128	383,779
Property and equipment, net	164,029	161,684
Long-term investments	36,548	4,621
Right-of-use assets	7,360	8,042
Other non-current assets	285	324
Total assets	790,350	558,450
Current liabilities
Accounts payable	4,340	3,981
Current portion of lease liability	1,501	1,561
Accrued expenses and other current liabilities	21,742	23,305
Total current liabilities	27,583	28,847
Lease liability	6,819	7,372
Total liabilities	34,402	36,219
Commitments and contingencies (Note 7)
Stockholders' equity
Common stock; $0.00001 par value; 80,000,000 shares authorized at September 30, 2023 and December 31, 2022; 28,194,655 shares issued and outstanding at September 30, 2023; and 25,763,743 shares issued and outstanding at December 31, 2022	0	0
Additional paid-in capital	1,034,849	803,718
Accumulated other comprehensive loss	(382)	(728)
Accumulated deficit	(278,519)	(280,759)
Total stockholders' equity	755,948	522,231
Total liabilities and stockholders' equity	$ 790,350	$ 558,450

Leases - Schedule of Supplemental Condensed Consolidated Balance Sheet Information Related to Leases (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Leases [Abstract]
Right-of-use assets	$ 7,360	$ 8,042
Current portion of lease liability	1,501	1,561
Lease liability	6,819	7,372
Present value of lease liability	$ 8,320	$ 8,933
Weighted average remaining lease term, in years	12 years 3 months 18 days	12 years 6 months
Weighted average discount rate	9.40%	9.40%

Summary of Significant Accounting Policies (Policies)

9 Months Ended

Sep. 30, 2023

Accounting Policies [Abstract]

Basis of Presentation

Use of Estimates

Segment and Geographical Information

Segment and Geographical InformationOperating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company and the Company’s chief operating decision maker view the Company’s operations and manage its business in one operating segment, which is the business of developing and commercializing pharmaceutical products.

Cash, Cash Equivalents and Investments

Fair Value of Financial Instruments

•Level 1— Valuations based on quoted prices in active markets for identical assets or liabilities.

•Level 3— Valuations based on inputs that are both significant to the fair value measurement and unobservable.

Revenue Recognition

The Company recognizes product revenue under ASC Topic 606, Revenue from Contracts with Customers (“Topic 606”). Under Topic 606, the Company is required to complete the following five steps:

Variable Consideration

–Returns: The Company offers SPs and SDs limited return rights relating to product damage or defect and based on these provisions, the Company believes that there will be minimal returns.

Cost of Goods Sold

Cost of Goods SoldCost of goods sold includes direct and indirect costs related to the manufacturing of VYJUVEK. These costs consist of manufacturing costs, personnel costs including stock-based compensation, facility costs, and other indirect overhead costs. Cost of goods sold may also include period costs related to certain manufacturing services and inventory adjustment charges.

Accounts Receivable

Accounts ReceivableAccounts receivable is recorded net of allowances for prompt payment discounts, returns, and credit losses. The Company estimates an allowance for credit losses by considering factors such as credit quality, the age of the accounts receivable balances, and current economic conditions that may affect a customer’s ability to pay.

Concentration of Credit Risk and Off-Balance Sheet Risk

Concentration of Credit Risk and Off-Balance Sheet RiskFinancial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents, short-term investments, long-term investments, and accounts receivable, net. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company is exposed to credit risk in the event of default by the financial institutions to the extent amounts recorded on the condensed consolidated balance sheets are in excess of insured limits. The Company has not experienced any credit losses in such accounts and does not believe it is exposed to any significant credit risk on these funds. The Company’s accounts receivable, net and marketable securities, which primarily consist of U.S. government agency securities and treasuries, equity securities, corporate bonds and commercial paper, potentially subject the Company to concentrations of credit risk.

Inventories

Property and Equipment, net


Buildings and building improvements			7 - 47 years
Computer equipment and software			3 - 7 years
Manufacturing equipment			3 - 20 years
Laboratory equipment			3 - 15 years
Furniture and fixtures			3 - 7 years
Leasehold improvements			lesser of useful life or remaining life of lease

Construction in progress is not depreciated until the asset is placed in service.

Impairment of Long-Lived Assets

Leases

Research and Development Expenses

Stock-Based Compensation Expense

Comprehensive Income (Loss)

Recent Accounting Pronouncements

Condensed Consolidated Statements of Stockholders' Equity (unaudited) - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-in Capital	Accumulated Other Comprehensive (Loss)	Accumulated Deficit
Beginning balance (in shares) at Dec. 31, 2021		25,207,985
Beginning balance at Dec. 31, 2021	$ 593,576	$ 0	$ 734,523	$ (163)	$ (140,784)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock, net, (in shares)		1,475
Issuance of common stock, net	55		55
Shares surrendered for taxes and forfeitures (in shares)		(10,379)
Shares surrendered for taxes	(649)		(649)
Stock-based compensation expense	6,571		6,571
Unrealized gain (loss) on investments and other	(1,034)			(1,034)
Net income (loss)	(49,965)				(49,965)
Ending balance (in shares) at Mar. 31, 2022		25,199,081
Ending balance at Mar. 31, 2022	548,554	$ 0	740,500	(1,197)	(190,749)
Beginning balance (in shares) at Dec. 31, 2021		25,207,985
Beginning balance at Dec. 31, 2021	593,576	$ 0	734,523	(163)	(140,784)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on investments and other	(1,312)
Net income (loss)	(107,923)
Ending balance (in shares) at Sep. 30, 2022		25,709,664
Ending balance at Sep. 30, 2022	540,772	$ 0	790,954	(1,475)	(248,707)
Beginning balance (in shares) at Mar. 31, 2022		25,199,081
Beginning balance at Mar. 31, 2022	548,554	$ 0	740,500	(1,197)	(190,749)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock, net, (in shares)		472,706
Issuance of common stock, net	30,748		30,748
Shares surrendered for taxes and forfeitures (in shares)		(7,500)
Stock-based compensation expense	8,335		8,335
Unrealized gain (loss) on investments and other	(348)			(348)
Net income (loss)	(28,108)				(28,108)
Ending balance (in shares) at Jun. 30, 2022		25,664,287
Ending balance at Jun. 30, 2022	559,181	$ 0	779,583	(1,545)	(218,857)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock, net, (in shares)		45,377
Issuance of common stock, net	2,176		2,176
Stock-based compensation expense	9,195		9,195
Unrealized gain (loss) on investments and other	70			70
Net income (loss)	(29,850)				(29,850)
Ending balance (in shares) at Sep. 30, 2022		25,709,664
Ending balance at Sep. 30, 2022	540,772	$ 0	790,954	(1,475)	(248,707)
Beginning balance (in shares) at Dec. 31, 2022		25,763,743
Beginning balance at Dec. 31, 2022	522,231	$ 0	803,718	(728)	(280,759)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock, net, (in shares)		42,021
Issuance of common stock, net	2,208		2,208
Shares surrendered for taxes and forfeitures (in shares)		(9,551)
Shares surrendered for taxes	(749)		(749)
Stock-based compensation expense	10,599		10,599
Unrealized gain (loss) on investments and other	574			574
Net income (loss)	(45,297)				(45,297)
Ending balance (in shares) at Mar. 31, 2023		25,796,213
Ending balance at Mar. 31, 2023	489,566	$ 0	815,776	(154)	(326,056)
Beginning balance (in shares) at Dec. 31, 2022		25,763,743
Beginning balance at Dec. 31, 2022	522,231	$ 0	803,718	(728)	(280,759)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on investments and other	346
Net income (loss)	2,240
Ending balance (in shares) at Sep. 30, 2023		28,194,655
Ending balance at Sep. 30, 2023	755,948	$ 0	1,034,849	(382)	(278,519)
Beginning balance (in shares) at Mar. 31, 2023		25,796,213
Beginning balance at Mar. 31, 2023	489,566	$ 0	815,776	(154)	(326,056)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock, net, (in shares)		2,178,703
Issuance of common stock, net	185,397		185,397
Stock-based compensation expense	11,443		11,443
Unrealized gain (loss) on investments and other	(82)			(82)
Net income (loss)	(33,210)				(33,210)
Ending balance (in shares) at Jun. 30, 2023		27,974,916
Ending balance at Jun. 30, 2023	653,114	$ 0	1,012,616	(236)	(359,266)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock, net, (in shares)		219,739
Issuance of common stock, net	13,511		13,511
Stock-based compensation expense	8,722		8,722
Unrealized gain (loss) on investments and other	(146)			(146)
Net income (loss)	80,747				80,747
Ending balance (in shares) at Sep. 30, 2023		28,194,655
Ending balance at Sep. 30, 2023	$ 755,948	$ 0	$ 1,034,849	$ (382)	$ (278,519)

Insider Trading Arrangements	3 Months Ended	9 Months Ended
Insider Trading Arrangements	Sep. 30, 2023 shares	Sep. 30, 2023 shares
Trading Arrangements, by Individual
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false
Kathryn Romano [Member]
Trading Arrangements, by Individual
Material Terms of Trading Arrangement		On August 29, 2023, Kathryn Romano, our Chief Accounting Officer, adopted a Rule 10b5-1 trading arrangement that is intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act for the sale of up to 25,000 shares of our Common Stock until June 28, 2024.
Name	Kathryn Romano
Title	Chief Accounting Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	August 29, 2023
Arrangement Duration	304 days
Aggregate Available	25,000	25,000

Gain from Sale of Priority Review Voucher (Details) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
Property, Plant and Equipment [Abstract]
Proceeds from sale of priority review voucher	$ 100,000	$ 0

Summary of Significant Accounting Policies (Tables)

9 Months Ended

Sep. 30, 2023

Accounting Policies [Abstract]

Summary of Estimated Useful Lives of Assets

Depreciation is recorded using the straight-line method over the estimated useful lives of the respective assets, which are as follows:


Buildings and building improvements			7 - 47 years
Computer equipment and software			3 - 7 years
Manufacturing equipment			3 - 20 years
Laboratory equipment			3 - 15 years
Furniture and fixtures			3 - 7 years
Leasehold improvements			lesser of useful life or remaining life of lease

Organization - Additional Information (Detail) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Accounting Policies [Abstract]
Accumulated deficit	$ (278,519)	$ (280,759)
Cash, cash equivalents and short-term investments	$ 562,100

- Definition

Amount of accumulated undistributed earnings (deficit).

+ References + Details

Subsequent Events	9 Months Ended
Subsequent Events	Sep. 30, 2023
Subsequent Events [Abstract]
Subsequent Events	Subsequent EventsThe Company evaluates events or transactions that occur after the balance sheet date, but prior to the issuance of the financial statements, to identify matters that require recognition or disclosure. The Company concluded that no subsequent events have occurred, that would require recognition or disclosure in the condensed consolidated financial statements.

Leases (Tables)

9 Months Ended

Sep. 30, 2023

Leases [Abstract]

Schedule of Future Minimum Operating Lease Payments

As of September 30, 2023, future minimum commitments under the Company’s operating leases with lease terms in excess of 12 months were as follows (in thousands):


	Operating Leases
2023 (remaining three months)	$	413
2024	1,539
2025	1,277
2026	1,277
2027	1,300
Thereafter	10,763
Future minimum operating lease payments	$	16,569
Less: Interest	(8,249)
Present value of lease liability	$	8,320

Schedule of Supplemental Condensed Consolidated Balance Sheet Information Related to Leases

Supplemental condensed consolidated balance sheet information related to leases is as follows:



	September 30, 2023			December 31, 2022
Operating leases:
Right-of-use assets	$	7,360		$	8,042
Current portion of lease liability	1,501			1,561
Lease liability	6,819			7,372
Total lease liability	$	8,320		$	8,933
Weighted average remaining lease term, in years	12.3			12.5
Weighted average discount rate	9.4		%	9.4		%

Lease, Cost

The components of the Company's lease expense are as follows:



	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Lease cost:
Operating lease expense	$	379	$	399	$	1,282	$	1,200
Variable lease expense	62		48		150		168
Total lease expense	$	441	$	447	$	1,432	$	1,368

Stock-Based Compensation - Additional Information (Detail)	3 Months Ended		9 Months Ended
	Sep. 30, 2023 USD ($) $ / shares shares	Sep. 30, 2022 USD ($) $ / shares shares	Sep. 30, 2023 USD ($) $ / shares shares	Sep. 30, 2022 USD ($) $ / shares shares	Dec. 31, 2017 shares
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Period increase in shares authorized			0.04
Stock option expiration period			10 years
Stock options granted (in shares) \| shares			419,780
Exercised	$ 13,500,000	$ 1,300,000	$ 41,400,000	$ 2,100,000
Inventories
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Amount capitalized	410,000		522,000
Property, Plant and Equipment
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Amount capitalized	$ 0	$ 137,000	$ 162,000	$ 423,000
Director
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock options granted (in shares) \| shares	30,500	189,000	419,780	1,958,000
Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares remaining available for grant \| shares	1,489,488		1,489,488
2017 IPO Stock Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares reserved (in shares) \| shares					900,000
Stock Options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option vesting period			4 years
Weighted-average grant-date fair value per share of options granted (in dollars per share) \| $ / shares	$ 85.93	$ 49.59	$ 62.87	$ 43.66
Stock Options \| Director
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized stock-based compensation expense	$ 77,500,000		$ 77,500,000
Estimated weighted average period			2 years 7 months 6 days
Non-Employee Stock Option \| Minimum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option vesting period			1 year
Non-Employee Stock Option \| Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option vesting period			3 years
Restricted Stock
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized stock-based compensation expense	$ 2,500,000		$ 2,500,000
Estimated weighted average period			1 year 4 months 24 days
Restricted Stock \| Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option vesting period			4 years
Incentive Stock Options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares remaining available for grant \| shares	30,783		30,783
Restricted Stock Units (RSUs)
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized stock-based compensation expense	$ 11,300,000		$ 11,300,000
Estimated weighted average period			3 years 4 months 24 days
Restricted Stock Units (RSUs) \| Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option vesting period			4 years
Performance Shares
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized stock-based compensation expense	$ 2,900,000		$ 2,900,000
Estimated weighted average period			1 year 4 months 24 days
Expected to vest	1		1

Stock-Based Compensation (Tables)

9 Months Ended

Sep. 30, 2023

Share-Based Payment Arrangement [Abstract]

Schedule of Company's Stock Option Activity

The following table summarizes the Company’s stock option activity:


	Stock Options Outstanding	Weighted- average Exercise Price		Weighted- average Remaining Contractual Life (Years)	Aggregate Intrinsic Value (In thousands) (1)
Outstanding at December 31, 2022	3,582,181	$	61.50	8.7	$	64,880
Granted	419,780	$	91.38
Exercised	(710,734)	$	57.98
Cancelled or forfeited	(623,967)	$	64.19
Expired	—	$	—
Outstanding at September 30, 2023	2,667,260	$	66.04	8.2	$	133,648
Exercisable at September 30, 2023	707,277	$	55.51	7.1	$	42,793

(1)Aggregate intrinsic value represents the difference between the closing stock price of our common stock on September 30, 2023 and the exercise price of outstanding in-the-money options.

Summary of Stock Based Compensation Expense Related to Issuance of Stock Option Awards and Restricted Stock Awards


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022

Research and development	$	2,033	$	2,184	$	6,858	$	5,547
Selling, general, and administrative	4,796		6,433		18,881		16,791
Total stock-based compensation	$	6,829	$	8,617	$	25,739	$	22,338


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Selling, general, and administrative	$	437	$	441	$	1,305	$	1,340
Total stock-based compensation	$	437	$	441	$	1,305	$	1,340


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Research and Development	$	303	$	—	$	837	$	—
Selling, general, and administrative	370		—		998		—
Total stock-based compensation	$	673	$	—	$	1,835	$	—


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Selling, general, and administrative	$	373	$	—	$	1,201	$	—
Total stock-based compensation	$	373	$	—	$	1,201	$	—

Fair Value of Stock Options, Valuation Assumptions


	Three Months Ended September 30,						Nine Months Ended September 30,
	2023			2022			2023			2022
Expected stock price volatility	74		%	78		%	73		%	78		%
Expected term of the award (years)	6.2			6.2			6.0			6.2
Risk-free interest rate	4.26		%	3.20		%	3.94		%	2.27		%
Weighted average exercise price	$	125.06		$	70.91		$	91.38		$	63.03
Forfeiture rate	—		%	—		%	—		%	—		%
Dividend yield	—		%	—		%	—		%	—		%

Share-based Payment Arrangement, Restricted Stock and Restricted Stock Unit, Activity


	Number of Shares		Weighted Average Grant Date Fair Value
Non-vested RSAs as of December 31, 2022	66,600		$	78.89
Granted	—		$	—
Vested	(12,649)		$	78.89
Surrendered for taxes	(9,551)		$	78.89
Non-vested RSAs as of September 30, 2023	$	44,400	$	78.89

The Company granted zero and 186,900 RSUs to employees of the Company during the three and nine months ended September 30, 2023, respectively, and zero RSUs during the three and nine months ended September 30, 2022, respectively.


	Number of Shares	Weighted Average Grant Date Fair Value
Non-vested RSUs as of December 31, 2022	—
Granted	186,900	$	81.91
Vested	—
Surrendered or forfeited	(24,700)	$	81.91
Non-vested RSUs as of September 30, 2023	162,200	$	81.91

Share-Based Payment Arrangement, Performance Shares, Activity

The Company granted zero and 60,000 PSUs to employees of the Company during the three and nine months ended September 30, 2023 and zero PSUs during the three and nine months ended September 30, 2022.


	Number of Shares	Weighted Average Grant Date Fair Value
Non-vested PSUs as of December 31, 2022	—
Granted	60,000	$	81.91
Vested	—
Surrendered or forfeited	(10,000)	$	81.91
Non-vested PSUs as of September 30, 2023	50,000	$	81.91

Fair Value Instruments - Summary of Cash, Cash Equivalents and Available-for-Sale Securities (Detail) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	$ 373,241	$ 161,900
Gross Unrealized Gains	230	179
Gross Unrealized (Losses)	(450)	(835)
Amortized Cost	598,837	384,448
Aggregate fair value, total	598,617	383,792
Cash and Cash Equivalents
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate fair value, total	373,241	161,900
Short-term Marketable Securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate fair value, total	188,828	217,271
Long-term Marketable Securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate fair value, total	36,548	4,621
Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	373,241	161,900
Aggregate Fair Value	373,241	161,900
Level 1 \| Cash and Cash Equivalents
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	373,241	161,900
Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Amortized Cost	225,596	222,548
Gross Unrealized Gains	230	179
Gross Unrealized (Losses)	(450)	(835)
Aggregate Fair Value	225,376	221,892
Level 2 \| Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Amortized Cost	43,496	63,624
Gross Unrealized Gains	1	5
Gross Unrealized (Losses)	(9)	(23)
Aggregate Fair Value	43,488	63,606
Level 2 \| Corporate bonds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Amortized Cost	70,465	82,241
Gross Unrealized Gains	4	13
Gross Unrealized (Losses)	(201)	(419)
Aggregate Fair Value	70,268	81,835
Level 2 \| U.S. government agency securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Amortized Cost	111,635	76,683
Gross Unrealized Gains	225	161
Gross Unrealized (Losses)	(240)	(393)
Aggregate Fair Value	111,620	76,451
Level 2 \| Short-term Marketable Securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate Fair Value	$ 188,828	217,271
Debt securities, available-for-sale, term	1 year
Level 2 \| Short-term Marketable Securities \| Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate Fair Value	$ 43,488	63,606
Level 2 \| Short-term Marketable Securities \| Corporate bonds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate Fair Value	56,463	77,214
Level 2 \| Short-term Marketable Securities \| U.S. government agency securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate Fair Value	88,877	76,451
Level 2 \| Long-term Marketable Securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate Fair Value	36,548	4,621
Level 2 \| Long-term Marketable Securities \| Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate Fair Value	0	0
Level 2 \| Long-term Marketable Securities \| Corporate bonds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate Fair Value	13,805	4,621
Level 2 \| Long-term Marketable Securities \| U.S. government agency securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Aggregate Fair Value	$ 22,743	0
Level 2 \| Long-term Marketable Securities \| Minimum
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Debt securities, available-for-sale, term	1 year
Level 2 \| Long-term Marketable Securities \| Maximum
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Debt securities, available-for-sale, term	2 years
Cash and cash equivalents \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	$ 373,241	161,900
Aggregate Fair Value	373,241	161,900
Cash and cash equivalents \| Level 1 \| Cash and Cash Equivalents
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash and cash equivalents	$ 373,241	$ 161,900

Capitalization - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions		1 Months Ended	9 Months Ended
	May 22, 2023	May 31, 2023	Sep. 30, 2022	Sep. 30, 2023	May 08, 2023	Dec. 31, 2022	Dec. 31, 2020
Schedule of Capitalization, Equity [Line Items]
Common stock, par value (in dollars per share)				$ 0.00001		$ 0.00001	$ 0.0001
Shares issued (in shares)				28,194,655		25,763,743
Placement Shares
Schedule of Capitalization, Equity [Line Items]
Sale of stock, aggregate offering price				$ 150.0			$ 150.0
Public Offering
Schedule of Capitalization, Equity [Line Items]
Sale of stock, number of shares issued in transaction			434,782
Sale of stock, price per share (in dollars per share)			$ 69.00
Proceeds from initial offering of shares			$ 29.1
ATM Program
Schedule of Capitalization, Equity [Line Items]
Underwriting discounts and commissions			$ 0.9
Private Placement Offering
Schedule of Capitalization, Equity [Line Items]
Sale of stock, number of shares issued in transaction	1,720,100	9,629
Sale of stock, price per share (in dollars per share)	$ 92.50
Proceeds from initial offering of shares	$ 160.0
Registration Rights Agreement
Schedule of Capitalization, Equity [Line Items]
Shelf registration time frame	60 days
New ATM Program
Schedule of Capitalization, Equity [Line Items]
Sale of stock, aggregate offering price					$ 150.0
Shares issued (in shares)				0

Net Income (Loss) Per Share Attributable to Common Stockholders (Tables)

9 Months Ended

Sep. 30, 2023

Earnings Per Share [Abstract]

Schedule of Net Income (Loss) Per Share Attributable to Common Stockholders


	Three Months Ended September 30,				Nine Months Ended September 30,
(In thousands, except share and per share data)	2023		2022		2023		2022

Numerator:
Net income (loss)	$	80,747	$	(29,850)	$	2,240	$	(107,923)
Denominator:
Weighted-average basic common shares	28,042,130		25,619,125		26,812,278		25,428,097
Dilutive effect of stock options and unvested restricted stock	850,096		—		572,261		—
Weighted-average diluted common shares	28,892,226		25,619,125		27,384,539		25,428,097

Net income (loss) per common share — Basic	$	2.88	$	(1.17)	$	0.08	$	(4.24)
Net income (loss) per common share — Diluted	$	2.79	$	(1.17)	$	0.08	$	(4.24)

Stock-Based Compensation - Schedule of Company's Stock Option Activity (Detail) $ / shares in Units, $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2023 USD ($) $ / shares shares	Dec. 31, 2022 USD ($) $ / shares shares
Stock Options Outstanding
Beginning balance (in shares) \| shares	3,582,181
Granted (in shares) \| shares	419,780
Exercised (in shares) \| shares	(710,734)
Cancelled or forfeited (in shares) \| shares	(623,967)
Expired (in shares) \| shares	0
Ending balance (in shares) \| shares	2,667,260	3,582,181
Exercisable end of period (in shares) \| shares	707,277
Weighted- average Exercise Price
Beginning balance (in dollars per share) \| $ / shares	$ 61.50
Granted (in dollars per share) \| $ / shares	91.38
Exercised (in dollars per share) \| $ / shares	57.98
Cancelled or forfeited (in dollars per share) \| $ / shares	64.19
Exercised (in dollars per share) \| $ / shares	0
Ending balance (in dollars per share) \| $ / shares	66.04	$ 61.50
Exercisable end of period (in dollars per share) \| $ / shares	$ 55.51
Weighted- average Remaining Contractual Life (Years)
Weighted-average remaining contractual life (Years)	8 years 2 months 12 days	8 years 8 months 12 days
Weighted-average remaining contractual life (Years), exercisable	7 years 1 month 6 days
Aggregate Intrinsic Value
Aggregate intrinsic value, beginning balance, outstanding \| $	$ 64,880
Aggregate intrinsic value, ending balance, outstanding \| $	133,648	$ 64,880
Exercisable at September 30, 2023 \| $	$ 42,793

Condensed Consolidated Statements of Operations and Comprehensive Loss (unaudited) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Income Statement [Abstract]
Revenue from Contract with Customer, Product and Service [Extensible Enumeration]	Product [Member]	Product [Member]	Product [Member]	Product [Member]
Product revenues, net	$ 8,556,000	$ 0	$ 8,556,000	$ 0
Expenses:
Cost, Product and Service [Extensible Enumeration]	Product [Member]	Product [Member]	Product [Member]	Product [Member]
Cost of goods sold	$ 223,000	$ 0	$ 223,000	$ 0
Research and development	10,629,000	11,516,000	35,061,000	31,720,000
Selling, general, and administrative	23,697,000	19,935,000	73,637,000	53,705,000
Litigation settlement	0	0	12,500,000	25,000,000
Total operating expenses	34,549,000	31,451,000	121,421,000	110,425,000
(Loss) from operations	(25,993,000)	(31,451,000)	(112,865,000)	(110,425,000)
Other income:
Gain from sale of priority review voucher	100,000,000	0	100,000,000	0
Interest and other income, net	6,740,000	1,601,000	15,105,000	2,502,000
Net income (loss)	80,747,000	(29,850,000)	2,240,000	(107,923,000)
Unrealized (loss) gain on available-for-sale securities and currency translation adjustment	(146,000)	70,000	346,000	(1,312,000)
Comprehensive income (loss)	$ 80,601,000	$ (29,780,000)	$ 2,586,000	$ (109,235,000)
Net income (loss) per common share: basic (in dollars per share)	$ 2.88	$ (1.17)	$ 0.08	$ (4.24)
Net income (loss) per common share: diluted (in dollars per share)	$ 2.79	$ (1.17)	$ 0.08	$ (4.24)
Weighted-average common shares outstanding: basic (in shares)	28,042,130	25,619,125	26,812,278	25,428,097
Weighted-average common shares outstanding: diluted (in shares)	28,892,226	25,619,125	27,384,539	25,428,097

Leases - Minimum commitments (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Leases [Abstract]
2023 (remaining three months)	$ 413
2024	1,539
2025	1,277
2026	1,277
2027	1,300
Thereafter	10,763
Future minimum operating lease payments	16,569
Less: Interest	(8,249)
Present value of lease liability	$ 8,320	$ 8,933

Leases

9 Months Ended

Sep. 30, 2023

Leases [Abstract]

Leases

As of September 30, 2023, future minimum commitments under the Company’s operating leases with lease terms in excess of 12 months were as follows (in thousands):


	Operating Leases
2023 (remaining three months)	$	413
2024	1,539
2025	1,277
2026	1,277
2027	1,300
Thereafter	10,763
Future minimum operating lease payments	$	16,569
Less: Interest	(8,249)
Present value of lease liability	$	8,320

Supplemental condensed consolidated balance sheet information related to leases is as follows:



	September 30, 2023			December 31, 2022
Operating leases:
Right-of-use assets	$	7,360		$	8,042
Current portion of lease liability	1,501			1,561
Lease liability	6,819			7,372
Total lease liability	$	8,320		$	8,933
Weighted average remaining lease term, in years	12.3			12.5
Weighted average discount rate	9.4		%	9.4		%

The components of the Company's lease expense are as follows:



	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Lease cost:
Operating lease expense	$	379	$	399	$	1,282	$	1,200
Variable lease expense	62		48		150		168
Total lease expense	$	441	$	447	$	1,432	$	1,368

Net Income (Loss) Per Share Attributable to Common Stockholders - Schedule of Basic and Diluted Net Loss Per Share and Per Unit (Detail) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2022	Jun. 30, 2022	Mar. 31, 2022	Sep. 30, 2023	Sep. 30, 2022
Numerator:
Net income (loss)	$ 80,747	$ (33,210)	$ (45,297)	$ (29,850)	$ (28,108)	$ (49,965)	$ 2,240	$ (107,923)
Denominator:
Weighted-average common shares outstanding: basic (in shares)	28,042,130			25,619,125			26,812,278	25,428,097
Dilutive effect of stock options and unvested restricted stock (in shares)	850,096			0			572,261	0
Weighted-average common shares outstanding: diluted (in shares)	28,892,226			25,619,125			27,384,539	25,428,097
Net income (loss) per common share: basic (in dollars per share)	$ 2.88			$ (1.17)			$ 0.08	$ (4.24)
Net income (loss) per common share: diluted (in dollars per share)	$ 2.79			$ (1.17)			$ 0.08	$ (4.24)

- 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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- Definition

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

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+ Details

- 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