Sarepta Therapeutics, Inc. develops and commercializes precision genetic medicines for rare diseases, with a central focus on Duchenne muscular dystrophy and other neuromuscular conditions. Company news commonly covers product revenue and commercialization trends for ELEVIDYS and PMO therapies, including AMONDYS 45, VYONDYS 53 and EXONDYS 51.
Updates also address FDA interactions for Duchenne therapies, clinical and biomarker data from the siRNA pipeline, and programs such as SRP-1001 for FSHD1 and SRP-1003 for DM1. Other recurring announcements include quarterly financial results, Nasdaq inducement equity grants, and patient-community initiatives tied to Duchenne muscular dystrophy.
Sarepta (NASDAQ:SRPT) announced Medsafe approval of the clinical trial application for SRP-1005, enabling the Phase 1 INSIGHTT study to begin. The first-in-human, multi-center, dose-escalation trial will enroll ~24 participants and is expected to start in Q2 2026.
SRP-1005 is an siRNA designed with a TfR1 monovalent fAb delivery approach for subcutaneous dosing to target deep brain regions in Huntington’s disease.
Sarepta (NASDAQ:SRPT) reported positive topline three-year results from Part 1 of EMBARK showing durable, statistically significant motor benefit for ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory Duchenne patients treated at ages 4–7 and followed to a mean age of ~9. At Year 3 (n=52 treated), mean NSAA remained above baseline (+4.39 LSM vs external control, p=0.0002), with TTR and 10MWR showing sustained improvement versus a propensity-weighted external control (TTR LSM difference -6.05s; 10MWR -2.70s, p=0.0039) and ~70% or greater slowing of decline on timed tests. No new treatment-related safety signals were observed; analysis and publications are ongoing.
Sarepta Therapeutics (NASDAQ:SRPT) will present 3-year topline functional results from Part 1 of EMBARK (Study 9001-301) on Jan. 26, 2026 at 8:30 AM ET. EMBARK is a global, randomized, placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at treatment.
The company will host a live webcast and conference call under its investor relations site and will archive a replay for one year. Phone participants must register online to receive dial-in details and a personal PIN via auto-generated email.
Sarepta (NASDAQ:SRPT) reported preliminary, unaudited Q4 2025 net product revenue of $369.6M and full-year 2025 net product revenue of $1.86B. ELEVIDYS revenue was $110.4M in Q4 and $898.7M for 2025; PMO revenues were $259.2M in Q4 and $965.6M for 2025. Preliminary cash, cash equivalents, restricted cash and investments totaled approximately $953.8M at Dec 31, 2025. The company reconfirmed a yearly ELEVIDYS sales floor of $500.0M but will not provide detailed 2026 guidance until initiatives are evaluated. Results are preliminary and subject to adjustment; final 2025 results expected in late February 2026.
Sarepta Therapeutics (NASDAQ:SRPT) submitted a clinical trial application to Medsafe for SRP-1005 (INSIGHTT), an investigational subcutaneous siRNA therapy for Huntington’s disease.
Pending approval, the first-in-human Phase 1 INSIGHTT study is expected to start in Q2 2026 and will enroll ~24 participants in a multi-center, dose-escalation safety and tolerability trial. Preclinical data showed protein knockdown in deep brain regions including the putamen and caudate.
Sarepta Therapeutics (NASDAQ:SRPT) said senior management will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 12, 2026 at 12:00 p.m. ET / 9:00 a.m. PT, with a Q&A starting at 12:20 p.m. ET / 9:20 a.m. PT.
The presentation will be webcast live via the Events & Presentations section of Sarepta’s investor website and will be archived for 90 days. Attendees are advised to connect to the webcast several minutes early to allow time for any required software download.
Sarepta describes its mission as engineering precision genetic medicine for rare diseases, with leadership in Duchenne muscular dystrophy and programs across muscle, central nervous system, and cardiac diseases.
Sarepta Therapeutics (NASDAQ:SRPT) granted equity awards on Dec. 31, 2025 as material inducements to employment for 10 individuals hired in Q4 2025. The awards, approved under Sarepta’s 2024 Employment Commencement Incentive Plan and in accordance with Nasdaq Listing Rule 5635(c)(4), total 55,118 restricted stock units (RSUs) granted in the aggregate. The RSUs vest 25% annually on each anniversary of the grant date and will be fully vested on the fourth anniversary, subject to continued employment. No stock options were granted to these employees.
Sarepta Therapeutics (NASDAQ:SRPT) announced privately negotiated exchanges of approximately $291.4 million principal of its 1.25% convertible senior notes due 2027 for $291.4 million principal of new 4.875% convertible senior notes due 2030 plus $31.6 million in cash.
The Exchange is expected to close on or about December 18, 2025, subject to customary conditions. After closing, aggregate principal of the 4.875% notes due 2030 will be $893.4 million, and $158.6 million of the existing 2027 notes will remain outstanding.
Sarepta (NASDAQ:SRPT) said the FDA approved dosing for Cohort 8 of the ENDEAVOR study to test an enhanced immunosuppression regimen with sirolimus in ~25 non-ambulant Duchenne patients, with dosing expected to begin before year-end 2025. The regimen uses 14 days of peri-infusion sirolimus and continues for 12 weeks after ELEVIDYS infusion. Primary endpoints are incidence of acute liver injury (ALI) and ELEVIDYS-dystrophin expression at 12 weeks. Sarepta expects primary endpoint data collection in H2 2026 and will review results with the FDA to inform commercial dosing decisions for this population.
Sarepta (NASDAQ:SRPT) reported progress in the Phase 1/2 multiple ascending dose study of SRP-1003 for myotonic dystrophy type 1. Cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) are complete; cohort 3 (4.5 mg/kg) is fully enrolled and ongoing. Following a positive pre-specified drug safety committee review, the study will continue dose escalation with patients currently being dosed in cohort 4 (6 mg/kg) and planned initiation of cohort 5 (12 mg/kg) in early 2026. Having reached the enrollment target, a second milestone payment of $200 million to Arrowhead will be made within 60 days.