SEGMENT INFORMATION

12 Months Ended

Dec. 31, 2024

Segment Reporting [Abstract]

Segment Information

21. SEGMENT INFORMATION

The Company, together with its wholly-owned subsidiaries, is a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. The Company’s research and development organization is responsible for the research and discovery of new product candidates and supports development and registration efforts for potential future products. The Company’s supply chain organization manages the development of the manufacturing processes, clinical trial supply and commercial product supply. The Company’s commercial organization is responsible for worldwide commercialization of EXONDYS 51, VYONDYS 53 and AMONDYS 45 and domestic commercialization of ELEVIDYS. The Company is supported by other back-office general and administration functions. Consistent with this decision-making process, the Company’s CEO uses consolidated, single-segment financial information for purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets.

The Company operates in one segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. The Company’s reportable segment derives its revenues from sales of its products, which include the PMO Products and ELEVIDYS, as well as through collaboration and other revenues primarily generated from its collaboration arrangement with Roche and other revenues related to commercial ELEVIDYS supply to Roche and royalty revenue from Roche. The Company’s CEO, as the CODM, manages and allocates resources to the operations of the Company on a total company basis by assessing the overall level of resources available and how to best deploy these resources across functions and in line with the Company's strategic goals. The Company’s accounting policies associated with segment information are described in Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements to the consolidated financial statements.

The measure of segment profit or loss that the CODM uses to allocate resources and assess performance is the Company’s consolidated net income (loss). The CODM uses consolidated net income (loss) to assess the segment’s overall profitability. The table below includes information about the Company’s segment, including significant segment expenses, and a reconciliation to net income (loss):

	For the Year Ended December 31,
	2024			2023			2022
	(in thousands)
Total revenues	$	1,901,979		$	1,243,336		$	933,013
Segment expenses and other segment items
Cost of sales (excluding amortization of in-licensed rights)		319,099			150,343			139,989
Compensation and other personnel expenses		335,830			319,080			256,382
Manufacturing expenses		329,011			345,826			466,111
Clinical trial expenses		163,565			187,289			135,838
Facility- and technology-related expenses (excluding depreciation and amortization)		106,281			88,559			71,700
Research and development- other (excluding non-cash items) (a)		108,597			118,727			117,171
Selling, general and administrative- other (excluding non-cash items) (b)		226,598			181,310			124,948
Roche collaboration reimbursement		(127,107	)		(106,885	)		(117,807	)
Other segment items (c)		(30,449	)		(49,129	)		(11,014	)
Interest expense		18,391			22,010			53,248
Interest income		(30,635	)		(36,257	)		(16,488	)
Income tax expense		25,535			15,879			13,525
Depreciation and amortization expense		37,724			44,397			41,864
Stock-based compensation expense		184,300			182,514			233,018
Gain from sale of Priority Review Voucher		—			(102,000	)		—
Impairment of strategic investments		—			30,321			2,575
Loss on debt extinguishment		—			387,329			125,441
Segment net income (loss)	$	235,239		$	(535,977	)	$	(703,488	)
Reconciliation of profit or loss
Adjustments and reconciling items		—			—			—
Consolidated net income (loss)	$	235,239		$	(535,977	)	$	(703,488	)

(a) Research and development-other includes professional services, up-front, milestone, and other expenses, pre-clinical expenses and research and other expenses.

(b) Selling, general and administrative-other includes professional services and other expenses.

(c) Other segment items included in segment net income (loss) include accretion of investment discount, net, change in fair value of derivatives and other, net, as well as the items separately presented and not defined as significant expenses below.

Significant expense categories that are regularly provided to the CODM include cost of sales (excluding amortization of in-licensed rights), compensation and other personnel expenses, manufacturing expenses, clinical trial expenses, facility- and technology-related expenses, research and development- other and selling, general and administrative- other. The other expense or income information are other segment items and include separate presentation of interest expense, income tax expense, depreciation and amortization, stock-based compensation, which are included in the measure of segment income (loss) but are not significant segment expenses.

Assets provided to the CODM for the single segment are consistent with those reported on the consolidated balance sheets.