Sarepta reports promising ELEVIDYS study results for Duchenne

CAMBRIDGE, Mass. - Sarepta Therapeutics, Inc. (NASDAQ:SRPT) has released new data from its ENDEAVOR study, showing encouraging results for its Duchenne muscular dystrophy treatment, ELEVIDYS. The latest cohort of the study, involving six participants aged 2, demonstrated a mean protein expression of 93.87% of normal, with a safety profile consistent with previous findings. Despite the positive clinical developments, InvestingPro data shows the stock has faced significant headwinds, trading near its 52-week low of $34.10, though analysis suggests the company is currently undervalued.

The ENDEAVOR study, formally known as Study 9001-103, is an ongoing Phase 1b trial assessing the expression and safety of ELEVIDYS in male patients with Duchenne across various cohorts. The recent results, which were observed 12 weeks post-treatment, also indicated 79.9 percent dystrophin positive fibers in biopsies from the participants. Common adverse events included nausea and vomiting, while elevated liver enzymes were resolved with steroids in two patients.

These findings build on earlier reported results from Cohort 4, which included participants aged 3 and showed a mean protein level of 99.64 percent. To date, the study has treated more than 25 patients under the age of 4. According to InvestingPro data, Sarepta maintains strong liquidity with a current ratio of 4.02, while revenue grew by 59% in the last twelve months. The company’s market capitalization stands at $3.47 billion, reflecting investor interest in its innovative pipeline despite recent market challenges.

Louise Rodino-Klapac, Ph.D., Sarepta’s chief scientific officer, expressed optimism about the biomarker outcomes in younger patients and mentioned an upcoming meeting with the U.S. FDA to discuss potential label expansion to include this age group.

ELEVIDYS is a single-dose gene therapy for Duchenne muscular dystrophy, a condition caused by mutations in the DMD gene leading to a lack of dystrophin protein. The treatment is designed to deliver a transgene for targeted production of micro-dystrophin in skeletal muscle. Currently, ELEVIDYS is indicated for patients at least 4 years old who are ambulatory or non-ambulatory with a confirmed DMD gene mutation.

The press release also outlined important safety information, including contraindications for patients with certain DMD gene deletions, precautions for infusion-related reactions, liver injury, immune-mediated myositis, myocarditis, and preexisting immunity against AAVrh74, the virus vector used in the treatment.

This news is based on a press release statement and provides an update on Sarepta Therapeutics’ progress in developing treatments for rare genetic diseases. The company continues to focus on advancing its portfolio of programs across muscle, central nervous system, and cardiac diseases. For investors seeking deeper insights, InvestingPro offers comprehensive analysis through its Pro Research Report, including 12 additional ProTips and detailed financial metrics that help evaluate the company’s growth potential and market position.

In other recent news, Sarepta Therapeutics announced the approval of its gene therapy, Elevidys, by the Japanese Ministry of Health, Labour, and Welfare for treating Duchenne muscular dystrophy in young children. This approval marks a significant milestone as it is the first global approval for DMD therapy in patients younger than four years old. The approval in Japan, achieved under a conditional and time-limited pathway, comes amid challenges in Europe, where Elevidys faced a partial clinical hold due to safety concerns. Morgan Stanley has maintained its Overweight rating on Sarepta with a $113 price target, indicating confidence in the drug’s market potential following the Japanese approval. However, Evercore ISI downgraded Sarepta’s stock to "In Line" and slashed the price target to $50, citing competitive pressures and regulatory uncertainties. BMO Capital also adjusted its price target from $160 to $120, maintaining an Outperform rating, reflecting cautious optimism about Sarepta’s future growth prospects. Meanwhile, RBC Capital reduced its price target to $58, maintaining a Sector Perform rating, due to concerns surrounding Elevidys, including a patient death and production challenges. These developments highlight the mixed sentiment among analysts regarding Sarepta’s future prospects in the gene therapy market.

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