Profitability and Strong Operating Performance
Reported GAAP operating profit of $358 million and non-GAAP operating profit of $398 million in Q1 2026, reflecting a profitable base business and positive cash flow excluding Arrowhead-related payments.
Solid Cash Position and Funding Capability
Ended the quarter with approximately $748 million in cash and investments; management states they can fully fund pipeline programs (DM1, FSHD, Huntington's, SCA2, IPF and preclinical portfolio) without accessing the equity markets.
Revenue and Product Mix
Total revenues for Q1 were $731 million (down 2% year-over-year). Net product revenue was $331 million, comprised of $229 million from PMO therapies (EXONDYS, VYONDYS, AMONDYS) and $102 million from Alevitus.
Improved Gross Margins and Lower Cost of Sales
Total cost of sales decreased to $109 million (a 21% decline versus prior year) and unit gross margins were reported at 82%, reflecting favorable margin dynamics on product sales.
Advancing siRNA Portfolio with Encouraging Early Data
Early Phase 1/2 data for SRP-1001 (FSHD) and SRP-1003 (DM1) showed dose-dependent plasma exposure, superior muscle delivery via the alpha v beta 6 integrin-targeting ligand, robust target engagement (DUX4-related gene suppression and DMPK knockdown), rapid reduction in CK (FSHD), and favorable repeated-dosing safety (no anemia); MAD cohort readouts expected later this year.
Alevitus Clinical Evidence and Real-World Experience
Alevitus has been administered to more than 1,300 patients (clinical and commercial). EMBARK trial shows meaningful and growing benefit through year three on multiple measures; muscle MRI data support reduced muscle loss and fat/fibrotic replacement. ENDEAVOR real-world interim data and physician experience suggest sirolimus pretreatment may mitigate liver enzyme elevations.
Regulatory and Value-Building Milestones
Submitted sNDAs (April) seeking conversion of AMONDYS 45 and 53 (PMOs) from accelerated to traditional approval following ESSENCE confirmatory data and supporting real-world evidence; Roche-related non-cash collaboration revenue of $325 million recognized this quarter and $40 million milestone from first commercial sale of Alevitus in Japan also recorded.