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CAMBRIDGE, Mass. - Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a $5.7 billion market cap biotechnology company, announced that clinical trials for its gene therapy ELEVIDYS will resume following a favorable review of safety data by an independent committee. The trials had been temporarily halted due to concerns over acute liver failure in patients. According to InvestingPro data, the stock has declined over 50% in the past six months, with recent analysis suggesting the shares are currently trading near their Fair Value.
On April 3, the independent data monitoring committee (DMC) convened and supported the continuation of the paused clinical trials after examining the totality of evidence. The decision came after European Union (EU) reference member country authorities requested a review following a safety update issued on March 18. Sarepta, in collaboration with Roche, is expected to submit a response to the temporary halt within a week, as requested by EU regulators. The EU regulatory process will determine the subsequent lifting of the halt.
The clinical studies affected include Study SRP-9001-302 (ENVOL), Study SRP-9001-303 (ENVISION), and Study SRP-9001-104. Despite the pause, monitoring and data collection for already-enrolled participants continue, and Sarepta does not anticipate a material impact on the studies’ timelines.
ELEVIDYS (delandistrogene moxeparvovec-rokl) is the only approved gene therapy for Duchenne muscular dystrophy (DMD), designed to treat mutations in the DMD gene that prevent the production of dystrophin protein. It is indicated for patients at least 4 years old, both ambulatory and non-ambulatory, with a confirmed mutation in the DMD gene.
The therapy has been associated with certain risks, including infusion-related reactions and acute serious liver injury, prompting recommendations for close monitoring during and after administration. The most common adverse reactions reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.
The resumption of clinical trials marks a significant step for Sarepta, which is focused on developing precision genetic medicines for rare diseases. The company, which has demonstrated strong revenue growth of 53% over the last twelve months and maintains a healthy current ratio of 4.2, continues to monitor the safety and efficacy of ELEVIDYS as part of its commitment to addressing the needs of individuals with DMD. InvestingPro analysis reveals 12 additional investment insights for this stock, available to subscribers, along with a comprehensive Pro Research Report covering key financial metrics and growth prospects.
This news is based on a press release statement from Sarepta Therapeutics, Inc. With analysts maintaining a strong buy consensus and setting price targets significantly above current levels, investors seeking detailed analysis can access the full range of metrics and expert insights through InvestingPro’s extensive coverage of over 1,400 US stocks.
In other recent news, Sarepta Therapeutics has been in the spotlight due to several significant developments. The company is expected to report its first-quarter earnings for 2025 soon, with analysts anticipating strong demand for ELEVIDYS, a gene therapy for Duchenne muscular dystrophy. However, the European Medicines Agency (EMA) has placed a clinical hold on ELEVIDYS studies following a patient death, prompting Needham to lower its price target for Sarepta from $202 to $183 while maintaining a Buy rating. UBS also reaffirmed a Buy rating with a $188 target, viewing the market’s reaction to the incident as excessive and emphasizing continued demand for ELEVIDYS.
Conversely, H.C. Wainwright has downgraded its rating for Sarepta to Neutral, with a $75 price target, citing concerns over the drug’s adoption due to safety issues and skepticism about meeting revenue guidance. The FDA’s recent shakeup, including the resignation of Dr. Peter Marks, has added to the uncertainty, affecting the broader biotech sector, including Sarepta. Despite these challenges, Sarepta’s collaboration with Arrowhead Pharmaceuticals remains intact, with no changes to revenue forecasts for ELEVIDYS reported by UBS. The developments highlight the complexities and potential risks faced by Sarepta in the evolving regulatory and market landscape.
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