Sarepta Therapeutics (SRPT) Stock News

Sarepta Therapeutics (NASDAQ:SRPT) announced a major strategic restructuring plan focused on cost reduction and pipeline prioritization. The company will implement a 36% workforce reduction affecting approximately 500 employees, targeting $400 million in annual cost savings. Preliminary Q2 2025 results show total net product revenue of $513 million, including $282 million from ELEVIDYS and $231 million from RNA-based PMOs.

The FDA has requested a black box warning for ELEVIDYS regarding acute liver injury. Sarepta is refocusing its pipeline on siRNA platform assets while pausing several gene therapy programs. The company will maintain its four Duchenne therapies while prioritizing development of treatments for FSHD, DM1, SCA2, IPF, and Huntington's disease.

The restructuring aims to reduce annual non-GAAP R&D and SG&A expenses to $800-900 million by 2026. The company reported $850 million in cash and investments as of June 30, 2025.

Sarepta Therapeutics (NASDAQ:SRPT), a precision genetic medicine company focused on rare diseases, announced the granting of equity awards to 53 new employees hired in Q2 2025. The awards, approved under the company's 2024 Employment Commencement Incentive Plan, include 22,016 stock options at an exercise price of $17.10 per share and 317,317 restricted stock units (RSUs).

The stock options will vest over four years, with 25% vesting after one year and the remainder vesting monthly. The RSUs will vest in four equal annual installments. Both awards are subject to continued employment with Sarepta. These inducement grants were made in accordance with Nasdaq Listing Rule 5635(c)(4).

[]

Sarepta Therapeutics (SRPT) has reported a second death from acute liver failure in non-ambulatory Duchenne patients treated with ELEVIDYS gene therapy. In response, the company is implementing critical safety measures including: suspending ELEVIDYS shipments for non-ambulatory patients, pausing the ENVISION confirmatory trial, and developing an enhanced immunosuppressive regimen that includes sirolimus. The company is consulting with clinical experts and regulators to strengthen safety protocols. The current treatment protocol remains unchanged for ambulatory patients. Over 900 patients have been treated with ELEVIDYS to date. The safety update follows liver enzyme elevations, a known side effect of AAV-based gene therapies, with evidence suggesting an adaptive immune response mechanism.

Sarepta Therapeutics announced that the FDA has granted platform technology designation to the rAAVrh74 viral vector used in SRP-9003, their investigational gene therapy for limb-girdle muscular dystrophy type 2E/R4. This designation, among the first of its kind, recognizes the technology's reproducibility and adaptability across multiple therapeutic programs. The platform technology designation allows Sarepta to leverage prior data to support future drug applications, streamlining development and review processes. SRP-9003 is designed to deliver a full-length beta-sarcoglycan transgene using the MHCK7 promoter, targeting skeletal, diaphragm, and cardiac muscle. This is particularly crucial for LGMD2E patients who often face pulmonary or cardiac complications.

Sarepta Therapeutics (NASDAQ:SRPT) announced that the UK's Medicines & Healthcare products Regulatory Agency (MHRA) has allowed continued uninterrupted dosing of ELEVIDYS in the ENVISION Phase 3 study. The study evaluates this gene therapy treatment in non-ambulatory and older ambulatory individuals with Duchenne muscular dystrophy (DMD). ELEVIDYS is a single-dose, AAV-based gene therapy designed to address DMD's genetic cause through micro-dystrophin production. The therapy is approved in the US for DMD patients aged 4 and older, both ambulatory and non-ambulatory, with the latter approved under accelerated approval pending confirmatory trials. The press release details important safety information, including potential risks such as infusion-related reactions, liver injury, immune-mediated myositis, and myocarditis.

Sarepta Therapeutics (NASDAQ:SRPT) reported new results from the ENDEAVOR study of ELEVIDYS for Duchenne muscular dystrophy treatment. In cohort 6 (n=6) with 2-year-old patients, treatment demonstrated mean protein expression of 93.87% measured by western blot and 79.9% dystrophin positive fibers. Safety profile aligned with previous studies, with common side effects including nausea and vomiting. Two patients experienced elevated liver enzymes, which resolved with steroid treatment. Previously reported results from Cohort 4 (3-year-olds, n=7) showed 99.64% mean protein levels. Over 25 patients under age 4 have been treated in clinical studies. The company plans to meet with FDA next month to discuss expanding ELEVIDYS label to include younger patients.

Sarepta Therapeutics (SRPT) presented new data from Part 2 of the EMBARK study for ELEVIDYS, their approved gene therapy for Duchenne muscular dystrophy. The analysis focused on 8-9 year old patients (n=14) who received treatment after placebo in Part 1. At one year post-treatment, patients showed statistically significant improvements across key endpoints compared to controls: • 4.75 points improvement in North Star Ambulatory Assessment (P=0.0026) • 6.87 seconds better in time-to-rise from floor (P=0.0010) • 4.76 seconds improvement in 10-meter walk/run (P=0.0097) These results are particularly notable as they demonstrate functional improvements at an age when motor decline typically occurs in Duchenne patients. The two-year study data showed sustained micro-dystrophin expression from Week 12 to Week 64 in a subset of patients, with no new safety signals observed.

Sarepta Therapeutics (NASDAQ:SRPT) has received approval from Japan's MHLW for ELEVIDYS, a gene therapy treatment for Duchenne muscular dystrophy (DMD). This marks the first global approval for treating patients younger than 4 years old, covering ages 3 to under 8 years. The approval is based on positive efficacy and safety data from clinical studies, including two-year results from the Phase 3 EMBARK trial, which demonstrated significantly better motor function outcomes compared to controls. Through a collaboration with Roche, Sarepta will receive up to $103.5M in milestone payments. The therapy will be commercialized in Japan by Chugai Pharmaceuticals via its Roche alliance. ELEVIDYS is a single-dose, AAV-based gene therapy designed to address DMD's genetic cause through micro-dystrophin production in skeletal muscle.

Sarepta Therapeutics (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced its senior management will participate in a fireside chat at the BofA Securities Health Care Conference. The event will take place at the Encore Hotel in Las Vegas on May 14, 2025, at 8:40 a.m. PT/11:40 a.m. ET.

The presentation will be available via webcast on Sarepta's investor relations website and will remain archived for 90 days. The company maintains leadership positions in Duchenne muscular dystrophy and limb-girdle muscular dystrophies, while developing a portfolio focused on muscle, central nervous system, and cardiac diseases.