v3.26.1
SEGMENT INFORMATION
 6 Months Ended
Jun. 30, 2026
Segment Reporting [Abstract]  
SEGMENT INFORMATION

15. SEGMENT INFORMATION

The Company operates in one segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. As of June 30, 2026, there have not been material changes in significant expense categories or assets provided to the Company’s CEO, the Company’s chief operating decision maker. For further details regarding the Company's segment information, please read Note 21, Segment Information of the Annual Report on Form 10-K for the year ended December 31, 2025. The table below includes information about the Company’s segment, including significant segment expenses and a reconciliation to net (loss) income:

 

 

 

For the Three Months Ended
June 30,

 

 

For the Six Months Ended
June 30,

 

 

 

2026

 

 

2025

 

 

2026

 

 

2025

 

 

 

 

 

 

 

 

 

(in thousands)

 

 

 

 

Total revenues

 

$

401,251

 

 

$

611,091

 

 

$

1,132,054

 

 

$

1,355,947

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Segment expenses and other segment items:

 

 

 

 

 

 

 

 

 

 

 

 

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

 

 

149,385

 

 

 

152,558

 

 

 

258,153

 

 

 

290,122

 

Compensation and other personnel expenses

 

 

60,725

 

 

 

89,839

 

 

 

122,655

 

 

 

182,923

 

Up-front and collaboration license fees

 

 

—

 

 

 

—

 

 

 

50,000

 

 

 

583,787

 

Manufacturing expenses

 

 

1,837

 

 

 

80,685

 

 

 

21,595

 

 

 

148,150

 

Clinical trial expenses

 

 

23,102

 

 

 

33,511

 

 

 

41,406

 

 

 

66,658

 

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

 

 

23,890

 

 

 

28,580

 

 

 

46,832

 

 

 

56,237

 

Research and development- other (excluding non-cash items) (1)

 

 

19,847

 

 

 

31,843

 

 

 

38,518

 

 

 

60,314

 

Selling, general and administrative- other (excluding non-cash items) (2)

 

 

42,462

 

 

 

56,322

 

 

 

82,655

 

 

 

107,464

 

Roche collaboration reimbursement

 

 

(7,169

)

 

 

(25,689

)

 

 

(15,358

)

 

 

(54,170

)

Other segment items (3)

 

 

(709

)

 

 

(1,314

)

 

 

(2,515

)

 

 

(3,223

)

Loss (gain) on strategic investments

 

 

448

 

 

 

(36,721

)

 

 

2,160

 

 

 

54,007

 

Interest expense

 

 

21,508

 

 

 

5,228

 

 

 

43,221

 

 

 

9,731

 

Interest income

 

 

(6,207

)

 

 

(5,254

)

 

 

(12,567

)

 

 

(15,444

)

Income tax expense (benefit)

 

 

3,141

 

 

 

(43,254

)

 

 

15,356

 

 

 

20,736

 

Depreciation and amortization expense

 

 

10,409

 

 

 

10,840

 

 

 

21,003

 

 

 

20,818

 

Stock-based compensation expense

 

 

24,472

 

 

 

37,025

 

 

 

53,871

 

 

 

78,453

 

Litigation contingency charge

 

 

39,000

 

 

 

—

 

 

 

39,000

 

 

 

—

 

Segment net (loss) income

 

$

(4,890

)

 

$

196,892

 

 

$

326,069

 

 

$

(250,616

)

 

 

 

 

 

 

 

 

 

 

 

 

 

Reconciliation of profit or loss

 

 

 

 

 

 

 

 

 

 

 

 

Adjustments and reconciling items

 

 

—

 

 

 

—

 

 

 

—

 

 

 

—

 

Consolidated net (loss) income

 

$

(4,890

)

 

$

196,892

 

 

$

326,069

 

 

$

(250,616

)

 

(1)
Research and development-other includes professional services, pre-clinical expenses and research and other expenses.
(2)
Selling, general and administrative-other includes professional services and other expenses.
(3)
Other segment items included in segment net (loss) income include accretion of investment discount, net and other, net, as well as 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 collaboration license 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 loss (gain) on strategic investments, interest expense, interest income, income tax expense (benefit), depreciation and amortization, stock-based compensation expense and litigation contingency charge, 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 unaudited condensed consolidated balance sheets.