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- Net product revenues for the first quarter 2025 totaled $611.5 million, a 70% increase over the same quarter of the prior year<\/i><\/b><\/li>\n
- ELEVIDYS net product revenue for the first quarter totaled $375.0 million; Royalty revenue from the sales of ELEVIDYS by Roche for the quarter totaled $4.0 million<\/i><\/b><\/li>\n
- Revised 2025 total net product revenues guidance to $2.3 to $2.6 billion<\/i><\/b><\/li>\n
- Meaningfully advanced multiple clinical candidates in limb-girdle muscular dystrophy portfolio<\/i><\/b><\/li>\n<\/ul>\n
CAMBRIDGE, Mass.–(BUSINESS WIRE<\/a>)–
\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the first quarter 2025.\n<\/p>\n\n\u201cIn the first quarter, we achieved net product revenue of $611.5 million, a 70% increase over the same quarter prior year; our PMO franchise performed well at $236.5 million; and ELEVIDYS achieved $375.0 million, growing at 180% over the same quarter prior year. However, we also faced headwinds in the quarter. While we are taking a variety of actions to address and resolve these challenges, we have adjusted our guidance for 2025 to $2.3 billion to $2.6 billion,\u201d said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. \u201cThe broader biotech market has also faced significant pressure in the first quarter of 2025. Fortunately, as an organization with four life-changing therapies, significant revenue, and a deep pipeline, Sarepta is well-positioned to navigate these chaotic times. To that end, in addition to driving the launch of ELEVIDYS and the continued performance of our three approved PMO therapies, we continue to invest in our pipeline and look forward to a significant number of LGMD gene therapy approvals in the coming few years. We are also advancing our siRNA platform and look forward to proof of concept and proof of biology readouts later this year in both our DM1 and FSHD1 programs.\u201d\n<\/p>\n
Corporate Highlights:<\/i><\/b><\/p>\n
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- \nIn the first quarter Sarepta closed its global licensing and collaboration agreement with Arrowhead Pharmaceuticals, Inc. (\u201cArrowhead\u201d). The agreement includes global rights to four clinical-stage and three preclinical-stage programs in muscle, central nervous system, and rare pulmonary disorders, including potential best-in-class siRNA-based treatments for DM1 and FSHD1. The agreement adds meaningfully to Sarepta\u2019s mid- and early-stage pipeline, complementing the Company\u2019s existing leadership in Duchenne muscular dystrophy and limb-girdle muscular dystrophies and gene therapy, while adding new indications and expanding into adjacent therapeutic areas. Later this year the Company plans to share data from the candidates in development for FSHD1 and DM1.\n<\/li>\n
- Pipeline progress for multiple of Sarepta’s limb-girdle muscular dystrophy programs:<\/b>\n
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- SRP-9005 for LGMD type 2C\/R5: <\/b>Following input from the FDA\u2019s Office of Therapeutic Products (OTP), the Company is cleared to proceed with dosing in Study SRP-9005-101 (COMPASS) in the U.S. COMPASS is Sarepta\u2019s first-in-human clinical study of SRP-9005, an investigational gene therapy for LGMD type 2C\/R5, or gamma-sarcoglycanopathy.\n<\/li>\n
- SRP-9004 for LGMD type 2D\/R3:<\/b> Enrollment and dosing is complete in Study SRP-9004-102 (DISCOVERY). DISCOVERY is a phase 1, proof-of-concept study evaluating safety and expression of the alpha-sarcoglycan protein after treatment with SRP-9004, an investigational gene therapy for the treatment of LGMD type 2D\/R3, or alpha-sarcoglycanopathy.\n<\/li>\n
- SRP-9003 for LGMD type 2E\/R4:<\/b> Enrollment and dosing is complete in Study SRP-9003-301 (EMERGENE). EMERGENE is a phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of LGMD type 2E\/R4, or beta-sarcoglycanopathy. EMERGENE is a global study and the primary endpoint is the biomarker expression of beta-sarcoglycan protein. A pre-Biologics License Application (BLA) meeting has occurred and the OTP has confirmed eligibility for the accelerated approval pathway for the program. Sarepta remains on track to submit a BLA to the U.S. FDA in the second half of 2025.\n<\/li>\n<\/ul>\n<\/li>\n
- 2025 MDA Clinical and Scientific Conference:<\/b> Sarepta showcased progress across its portfolio of approved and pipeline therapies for Duchenne and limb-girdle muscular dystrophy with 11 abstracts and two oral presentations. The oral presentations included:\n
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- \nResults from the 2-year EMBARK and 3-year pooled analyses indicating stabilization or slowing of disease progression compared with well-matched external control assessed by functional outcomes predictive for delaying loss of ambulation.\n<\/li>\n
- \nTwo-year muscle MRI data, from a subset of patients in EMBARK Part 1, showing stabilization or minimal progression of underlying muscle pathology which remains consistent with functional benefits.\n<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n
\nAll of the posters and presentations are available on the Company\u2019s website, here<\/a>.\n<\/p>\n
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- \nSarepta remains on track to achieve robust revenue growth in 2025 and maintain a strong balance sheet.\n<\/li>\n<\/ul>\n
Conference Call<\/i><\/b><\/p>\n
\nThe event will be webcast live under the investor relations section of Sarepta’s website at https:\/\/investorrelations.sarepta.com\/events-presentations<\/b><\/a> and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form<\/b><\/a>. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.\n<\/p>\n
Q1 2025 Financial Highlights1<\/sup><\/i><\/b><\/p>\n
- \nSarepta remains on track to achieve robust revenue growth in 2025 and maintain a strong balance sheet.\n<\/li>\n<\/ul>\n
- SRP-9004 for LGMD type 2D\/R3:<\/b> Enrollment and dosing is complete in Study SRP-9004-102 (DISCOVERY). DISCOVERY is a phase 1, proof-of-concept study evaluating safety and expression of the alpha-sarcoglycan protein after treatment with SRP-9004, an investigational gene therapy for the treatment of LGMD type 2D\/R3, or alpha-sarcoglycanopathy.\n<\/li>\n
- SRP-9005 for LGMD type 2C\/R5: <\/b>Following input from the FDA\u2019s Office of Therapeutic Products (OTP), the Company is cleared to proceed with dosing in Study SRP-9005-101 (COMPASS) in the U.S. COMPASS is Sarepta\u2019s first-in-human clinical study of SRP-9005, an investigational gene therapy for LGMD type 2C\/R5, or gamma-sarcoglycanopathy.\n<\/li>\n
- ELEVIDYS net product revenue for the first quarter totaled $375.0 million; Royalty revenue from the sales of ELEVIDYS by Roche for the quarter totaled $4.0 million<\/i><\/b><\/li>\n
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