Sarepta Therapeutics Inc Stock Price, News & Analysis

Arrowhead Pharmaceuticals (NASDAQ: ARWR) has achieved a significant milestone in its collaboration with Sarepta Therapeutics (NASDAQ: SRPT), earning a $100 million payment. The milestone was triggered by reaching the first enrollment target in a Phase 1/2 clinical study of ARO-DM1, an RNAi therapeutic targeting type 1 myotonic dystrophy (DM1).

The company anticipates reaching a second enrollment target by the end of 2025, which would trigger an additional $200 million milestone payment. The current payment is expected to be received within 60 days, as per the license and collaboration agreement terms.

Sarepta Therapeutics (NASDAQ:SRPT) has issued a clarifying statement regarding a reported death of an eight-year-old Duchenne muscular dystrophy patient who received ELEVIDYS gene therapy. The company emphasized that the death was deemed unrelated to the treatment, as confirmed by both Roche Holding AG and the reporting physician.

The incident was reported to the FDA on June 18, 2025, through the FDA's postmarketing electronic database (FAERS). ELEVIDYS remains the only approved gene therapy for Duchenne muscular dystrophy, indicated for both ambulatory and non-ambulatory patients aged 4 years and above.

Sarepta Therapeutics (NASDAQ:SRPT) received a negative opinion from the European Medicines Agency's CHMP regarding the conditional marketing authorization for ELEVIDYS in treating Duchenne muscular dystrophy (DMD) patients aged 3-7 years in the EU.

While ELEVIDYS, the first gene therapy targeting DMD's underlying cause, has gained approval in the US and other countries since June 2023, Sarepta acknowledges current FDA safety inquiries. Despite not meeting the primary endpoint in the EMBARK study's first year, the therapy demonstrated statistically significant improvements in secondary endpoints. The company submitted additional efficacy data, including two-year EMBARK results and a three-year pooled analysis showing improvements in motor function.

Partner Roche will continue discussions with EMA to explore potential pathways for making ELEVIDYS available in the EU, while maintaining responsibility for regulatory approvals outside the US.

Sarepta Therapeutics (NASDAQ:SRPT) announced a voluntary and temporary pause of ELEVIDYS shipments in the United States, effective July 22, 2025. The pause will allow the company to address FDA information requests and complete the safety labeling supplement process for their Duchenne muscular dystrophy treatment.

ELEVIDYS is a single-dose gene therapy designed to treat Duchenne muscular dystrophy in patients aged 4 and older through the delivery of a transgene that codes for micro-dystrophin production in skeletal muscle. CEO Doug Ingram emphasized this was a difficult but necessary decision to maintain a productive relationship with the FDA.

Sarepta Therapeutics (NASDAQ:SRPT) responded to an informal FDA request to voluntarily halt shipments of ELEVIDYS, their approved gene therapy for Duchenne muscular dystrophy. The company will continue shipping to ambulant patients while pausing shipments for non-ambulant patients pending label updates and enhanced immunosuppression protocols.

The statement addresses a recent death of a 51-year-old non-ambulant LGMD patient in a Phase 1 trial, clarifying this was related to a different investigational therapy (SRP-9004) for Limb-Girdle Muscular Dystrophy, not ELEVIDYS. The company reported the acute liver failure event to FDA on June 20, 2025, with follow-up notification of death on July 3, 2025.

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).

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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.