The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping Duchenne muscular dystrophy drug Exondys 51.
After feedback from the FDA and an internal analysis, Sarepta Therapeutics will discontinue development of vesleteplirsen, an exon 51-skipping therapy for Duchenne muscular dystrophy that was a potential successor to Exondys 51.
The Cambridge, Mass.–based biotech made this announcement Wednesday during its third-quarter earnings call. The decision was based on feedback with the FDA, Sarepta’s risk-benefit analysis of the program and the evolving Duchenne muscular dystrophy (DMD) landscape, “including the approval of Elevidys,” Sarepta CEO Doug Ingram said on the call.
In January 2024, Sarepta announced positive data from Part B of the Phase II MOMENTUM study, which assessed vesleteplirsen in patients 8- to 21-years who are amenable to exon 51 skipping. The company noted at the time that cases of hypomagnesemia—or abnormally low magnesium—had previously been identified and that this was “managed and monitored” during the trial.
On Wednesday’s earnings call, Sarepta’s head of R&D Louise Rodino-Klapac noted that these cases persisted even after discontinuing treatment, Endpoints News reported. The FDA informed Sarepta that the accelerated approval pathway “was not open” for vesleteplirsen based on current information on the drug, Rodino-Klapac added.
In an investor note published Thursday, Jefferies analysts said they “see no meaningful stock impact” following the discontinuation of vesleteplirsen “since this implies pot’l sales upside to Elevidys (and/or Exondys).”
Elevidys, approved last year as the first gene therapy for DMD, brought in $181 million in net revenue in the third quarter, “exceeding prior guidance,” according to a Wednesday press release. Including non-U.S. sales from commercialization partner Roche, Elevidys generated $190.5 million for the quarter. This revenue represented 49% growth over Q3 2023, according to Jefferies.
Mizuho’s Uy Ear in an investor note called the result “undisputedly a strong start to commercializing the expanded Elevidys label.”
After winning an accelerated FDA nod for Elevidys in June 2023, Sarepta secured full approval and an expanded label for the gene therapy a year later, opening treatment to ambulatory DMD patients 4 years and older. The original label covered only ambulatory patients 4 to 5 years of age. The FDA also granted accelerated approval for Elevidys in non-ambulatory patients.
Elevidys’ original approval was controversial, as some experts questioned its efficacy—a concern that was heightened when the gene therapy missed the primary functional endpoint in its confirmatory trial in October 2023.
Last month, Sarepta presented new data on Elevidys at the World Muscle Society meeting in Prague, but experts told BioSpace more data, especially in these new patient populations, is needed.
Overall, Sarepta reported $467.2 million in the third quarter, up from $331.8 million in the corresponding 2023 timeframe. The company’s stock was up 3.46% in premarket trading Thursday.
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