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06 May, 2025
Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, today announced its financial results for the first quarter ended March 31, 2025, and provided updates on recent corporate developments.
Our first-quarter net product revenue reached $611.5 million—a 70% increase year-over-year—driven by $375.0 million from ELEVIDYS, which grew 180% over the same quarter last year, and $236.5 million from our PMO franchise, said Doug Ingram, president and CEO. “While we encountered challenges that led us to revise our 2025 revenue guidance to $2.3–$2.6 billion, we remain confident in Sarepta’s trajectory. With four approved therapies, strong commercial performance, and a robust pipeline, we are well-positioned to weather current industry headwinds. We are particularly focused on advancing our siRNA programs and expect key readouts this year for both DM1 and FSHD1.
Q1 2025 Corporate and Pipeline Highlights
Arrowhead Collaboration Finalized: Closed a global licensing and collaboration agreement with Arrowhead Pharmaceuticals, securing rights to seven programs (four clinical-stage and three preclinical) targeting muscle, CNS, and rare pulmonary diseases. This includes siRNA-based candidates for DM1 and FSHD1.
Gene Therapy Programs for Limb-Girdle Muscular Dystrophy (LGMD):
SRP-9005 (LGMD 2C/R5): Cleared by the FDA to begin dosing in the U.S.-based COMPASS study.
SRP-9004 (LGMD 2D/R3): Enrollment and dosing completed in the phase 1 DISCOVERY trial.
SRP-9003 (LGMD 2E/R4): Enrollment and dosing completed in the global phase 3 EMERGENE study. Sarepta held a pre-BLA meeting with the FDA, which confirmed eligibility for accelerated approval. BLA submission is expected in H2 2025.
Scientific Presentations: At the 2025 MDA Clinical and Scientific Conference, Sarepta presented 11 abstracts and 2 oral presentations. Data from EMBARK demonstrated disease stabilization or delayed progression in Duchenne muscular dystrophy (DMD), supported by 2-year muscle MRI results.
Q1 2025 Financial Overview
Total Revenue: $744.9 million (up 80% YoY), driven by expanded ELEVIDYS sales and increased collaboration revenue from Roche.
GAAP Net Loss: $(447.5) million; Non-GAAP Net Loss: $(332.5) million.
Operating Loss: GAAP $(300.4) million; Non-GAAP $(249.6) million.
Cash, Equivalents & Investments: $647.5 million as of March 31, 2025 (down from $1.5 billion as of Dec 31, 2024), reflecting the Arrowhead transaction and continued R&D investment.
Financial Guidance Update
Revised 2025 Net Product Revenue Guidance: $2.3 to $2.6 billion (previously $2.9 to $3.1 billion), reflecting an updated outlook for ELEVIDYS.
Combined Non-GAAP R&D and SG&A Expenses: $1.78 to $2.18 billion, now inclusive of Arrowhead-related expenses.
Sarepta continues to prioritize pipeline advancement, with a focus on delivering multiple LGMD gene therapy programs, progressing its siRNA platform, and maintaining strong commercial momentum.