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Shares of Sarepta Therapeutics SRPT dropped more than 6% yesterday after the EMA placed a clinical hold on all studies evaluating Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD). The therapy has been developed in collaboration with pharma giant Roche RHHBY.
This hold has been placed in response to a patient’s death post-treatment with Elevidys, which was reported last month. Though the death was due to acute liver failure (a known risk of Elevidys), Sarepta believes that the patient’s recent CMV infection might have contributed to the outcome. Investigation is still ongoing.
In a letter to the World Duchenne Organization, Roche mentioned that the companies are pausing enrollment and dosing of study participants in three studies evaluating the therapy at the EU study sites. This hold will remain ‘until the analysis into the cause of death is complete.’
The paused studies include Sarepta-sponsored phase III ENVISION study (in boys aged 8-17 years), the Roche-sponsored phase II ENVOL study in infants and newborns, and Sarepta’s early-stage study in boys aged 4 to 9 years with pre-existing antibodies to a specific serotype. Roche has also paused enrolment and dosing for the ENVOL study being conducted at the U.K. study sites.
SRPT and RHHBY entered into a licensing agreement in 2019 to develop Elevidys. Per the agreement, Sarepta retains the rights to market the gene therapy in the United States while Roche has exclusive rights to launch and market the therapy outside the country.
SRPT Stock’s Performance
The downside in Sarepta stock was likely due to concerns that this latest development could make doctors even more hesitant to prescribe Elevidys, negatively impacting the therapy’s overall market adoption in the United States. Also, the clinical hold further delays the launch plans for the gene therapy in the European Union.
Elevidys is a major contributor to Sarepta’s topline. The therapy’s sales accounted for nearly 60% of the company’s total revenues in the fourth quarter of 2024. For full-year 2025, SRPT expects net product revenues in the range of $2.9-$3.1 billion, two-thirds of which are expected to come from Elevidys’ sales. Any further negative developments concerning the gene therapy could adversely impact both SRPT’s growth trajectory and stock performance.
Year to date, Sarepta’s shares have plunged nearly 52% against the industry’s 1% growth.
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Sarepta’s Strong Commercial DMD Portfolio
Sarepta’s portfolio consists of four approved therapies, all targeting DMD indications.