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CAMBRIDGE, Mass. - Sarepta Therapeutics, Inc. (NASDAQ:SRPT), currently trading near its 52-week low and down over 70% year-to-date according to InvestingPro data, announced the Japanese Ministry of Health, Labour, and Welfare’s approval of ELEVIDYS for Duchenne muscular dystrophy (DMD) treatment in children ages 3 to less than 8. This marks the first global approval for DMD therapy in patients younger than 4 years old. The approval, granted under Japan’s conditional and time-limited pathway, is contingent upon the demonstration of significant efficacy and safety in early clinical trials.
ELEVIDYS (delandistrogene moxeparvovec) is a gene therapy designed to address the genetic root of DMD by delivering a micro-dystrophin transgene to promote targeted protein production in muscle tissue. The approval was based on data from the EMBARK Phase 3 study, which indicated improved motor function in treated individuals over two years, with no new safety concerns. Despite recent market challenges, InvestingPro analysis shows the company maintains strong liquidity with a current ratio of 4.02, indicating solid ability to meet short-term obligations. These results were recently presented at the Muscular Dystrophy Association meeting and published in respected medical journals.
Sarepta’s collaboration with Roche aims to expand access to ELEVIDYS worldwide, with Sarepta managing U.S. regulatory approval and commercialization, and Roche, through Chugai Pharmaceuticals, covering other markets including Japan. The company is poised to receive up to $103.5 million in milestone payments linked to regulatory and commercial achievements. With revenue growth of 59% in the last twelve months and analysts projecting profitability this year, these developments could be crucial for the company’s financial trajectory. For detailed financial analysis and 13 additional ProTips, visit InvestingPro.
ELEVIDYS is also indicated in the U.S. for DMD patients at least 4 years old, both ambulatory and non-ambulatory, with a confirmed DMD gene mutation. The U.S. has granted accelerated approval for non-ambulatory patients based on micro-dystrophin expression in skeletal muscle, with continued approval dependent on confirmatory trials.
Safety information for ELEVIDYS highlights contraindications for patients with certain DMD gene deletions and precautions for potential infusion-related reactions, acute serious liver injury, immune-mediated myositis, myocarditis, and preexisting immunity against the AAVrh74 vector used in the therapy. The market currently values Sarepta at $3.5 billion, with analyst price targets ranging from $40 to $169, suggesting significant potential upside from current levels.
Sarepta emphasizes its commitment to advancing DMD treatment and its broader portfolio targeting muscle, central nervous system, and cardiac diseases. This news is based on a press release statement from Sarepta Therapeutics, Inc.
In other recent news, Sarepta Therapeutics has been the focus of several analyst adjustments and developments. The company reported lower-than-expected revenue for its gene therapy product, Elevidys, in the first quarter of 2025, which amounted to $375 million, falling short of the $444 million forecasted by analysts. This shortfall led Sarepta to revise its full-year revenue guidance for 2025 down to $2.3-$2.6 billion from the previous $2.9-$3.1 billion. In response, TD Cowen reduced its price target for Sarepta to $137, maintaining a Buy rating, while RBC Capital Markets lowered its target to $58, citing Elevidys-related concerns.
Evercore ISI downgraded Sarepta’s stock rating to "In Line" from "Outperform," slashing the price target to $50 due to competitive pressures and regulatory uncertainties. BMO Capital Markets also adjusted its price target to $120, maintaining an Outperform rating, reflecting cautious optimism despite recent challenges. Cantor Fitzgerald revised its price target to $81, maintaining an Overweight rating, citing safety concerns and competitive threats impacting Elevidys. These developments highlight the challenges Sarepta faces in navigating regulatory risks and market dynamics, with analysts expressing varied outlooks on the company’s future performance.
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