SRPT Stock Up as Enrollment Begins in DMD Gene Therapy Safety Study

Shares of Sarepta Therapeutics SRPT rose about 2% yesterday after it announced that screening and enrollment are underway in cohort 8 of the phase Ib ENDEAVOR study.

This cohort is designed to improve the safety profile of Elevidys — its one-shot gene therapy for Duchenne muscular dystrophy (DMD) — in non-ambulatory patients by combining it with an enhanced sirolimus-based immunosuppressive regimen.

Data from this cohort will be used to determine whether administering sirolimus before or after Elevidys infusion can help reduce the risk of acute liver complications. Sarepta plans to enroll around 25 non-ambulatory patients with DMD in the United States who will receive the immunosuppressive regimen. This will include 14 days of sirolimus dosing before Elevidys infusion, followed by 12 weeks of treatment after infusion.

The company developed Elevidys in partnership with pharma giant Roche RHHBY. In 2019, Sarepta and Roche entered into a licensing agreement to develop Elevidys. Per the agreement, Roche has exclusive rights to launch and market the therapy in non-U.S. markets.

SRPT’s Stock Performance

In the past year, the stock has plunged nearly 84% against the industry’s 11% growth.

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More on Sarepta’s Elevidys

Sarepta has been facing a lot of heat from investors after three patient deaths were linked to its gene therapy treatments and attributed to acute liver failure (ALF). While two deaths occurred in patients after receiving Elevidys, one was caused by an experimental limb-girdle muscular dystrophy (LGMD) therapy. All deaths occurred in non-ambulatory patients and were linked to the AAVrh74 gene therapy vector used in both Elevidys and Sarepta’s experimental LGMD therapies.

Based on these events, the FDA approved significant changes to the label of Elevidys last November. Under the revised label, the gene therapy is now approved only for ambulatory patients aged four years and older with DMD. It is no longer authorized for use in non-ambulatory patients. Elevidys is mandated to carry a boxed warning — the most important safety warning issued by the FDA — highlighting the risks of ALF and acute liver injury (ALI).

The agency added additional limitations to the gene therapy’s label. It is no longer recommended for patients with pre-existing liver impairment, recent vaccinations or active/recent infections. The updated label introduces new monitoring requirements, including weekly liver function tests for at least three months post-treatment. Patients are advised to remain near an appropriate medical facility for at least two months after infusion to ensure rapid access to care if complications arise.

Following these setbacks, Sarepta paused development of most of its LGMD pipeline and shifted focus to the siRNA programs, which were acquired last year as part of a multi-billion-dollar licensing deal with Arrowhead Pharmaceuticals ARWR.

Sarepta Therapeutics, Inc. Price

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SRPT’s Zacks Rank

Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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