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, siRNA platform, 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, or the chief operating decision maker (the “CODM”), 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 the sale of commercial ELEVIDYS supply to Roche and royalty revenue from Roche. 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 measure of segment profit or loss that the CODM uses to allocate resources and assess performance is the Company’s consolidated net (loss) income. The table below includes information about the Company’s segment, including significant segment expenses, and a reconciliation to net (loss) income:

 

 

 

For the Year Ended December 31,

 

 

 

2025

 

 

2024

 

 

2023

 

 

 

(in thousands)

 

Total revenues

 

$

2,198,237

 

 

$

1,901,979

 

 

$

1,243,336

 

 

 

 

 

 

 

 

 

 

 

Segment expenses and other segment items

 

 

 

 

 

 

 

 

 

Cost of sales (excluding amortization of in-licensed rights)

 

 

839,605

 

 

 

319,099

 

 

 

150,343

 

Compensation and other personnel expenses

 

 

294,965

 

 

 

335,830

 

 

 

319,080

 

Up-front and milestone expenses

 

 

883,787

 

 

 

 

 

 

 

Manufacturing expenses

 

 

208,830

 

 

 

329,011

 

 

 

345,826

 

Clinical trial expenses

 

 

124,645

 

 

 

163,565

 

 

 

187,289

 

Facility- and technology-related expenses (excluding depreciation and amortization)

 

 

106,456

 

 

 

106,281

 

 

 

88,559

 

Restructuring charge

 

 

42,009

 

 

 

 

 

 

 

Research and development- other (excluding non-cash items) (a)

 

 

107,346

 

 

 

108,597

 

 

 

118,727

 

Selling, general and administrative- other (excluding non-cash items) (b)

 

 

207,189

 

 

 

226,598

 

 

 

181,310

 

Roche collaboration reimbursement

 

 

(84,731

)

 

 

(127,107

)

 

 

(106,885

)

Other segment items (c)

 

 

(3,985

)

 

 

(33,234

)

 

 

(47,602

)

(Gain) loss on debt extinguishment

 

 

(16,862

)

 

 

 

 

 

387,329

 

Loss (gain) on strategic investments, net

 

 

15,914

 

 

 

2,785

 

 

 

(1,527

)

Interest expense

 

 

38,140

 

 

 

18,391

 

 

 

22,010

 

Interest income

 

 

(30,763

)

 

 

(30,635

)

 

 

(36,257

)

Income tax expense

 

 

11,185

 

 

 

25,535

 

 

 

15,879

 

Depreciation and amortization expense

 

 

44,521

 

 

 

37,724

 

 

 

44,397

 

Stock-based compensation expense

 

 

123,396

 

 

 

184,300

 

 

 

182,514

 

Gain from sale of Priority Review Voucher

 

 

 

 

 

 

 

 

(102,000

)

Impairment of strategic investments

 

 

 

 

 

 

 

 

30,321

 

Segment net (loss) income

 

$

(713,410

)

 

$

235,239

 

 

$

(535,977

)

 

 

 

 

 

 

 

 

 

 

Reconciliation of profit or loss

 

 

 

 

 

 

 

 

 

Adjustments and reconciling items

 

 

 

 

 

 

 

 

 

Consolidated net (loss) income

 

$

(713,410

)

 

$

235,239

 

 

$

(535,977

)

 

(a) Research and development-other includes professional services, 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 (loss) income 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, up-front and milestone expenses, manufacturing expenses, clinical trial expenses, facility- and technology-related expenses, restructuring charge, 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 (gain) loss on debt extinguishment, loss (gain) on strategic investments, interest expense, interest income, income tax expense, depreciation and amortization and stock-based compensation, which are included in the measure of segment net (loss) income, 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.

Historical Timeline

Fiscal YearFiled
2025Mar 2, 2026Showing above
2024Feb 28, 2025

About Segments Disclosures

Segment disclosures break a company into its reportable operating units, revealing revenue, profit, and asset allocation that consolidated financial statements obscure. Under ASC 280, segments must match how the chief operating decision maker views the business, providing a window into internal management structure and resource allocation priorities.

Key signals: compare segment margins to identify which units drive profitability and which destroy value. Watch for changes in the number of reportable segments — segment aggregation or disaggregation often coincides with strategic shifts or attempts to obscure declining performance. Intersegment elimination patterns reveal internal pricing practices. The reconciliation between segment totals and consolidated figures exposes corporate overhead allocation and unallocated items. Geographic revenue concentration highlights regulatory and currency exposure. Compare segment-level capital expenditure against segment revenue to assess where management is investing for future growth versus harvesting existing assets.