Biogen Discontinues Aducanumab, CHMP Issues Negative Opinion on Ataluren, SRP-5051 Shines in MOMENTUM Study

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

According to an announcement from Biogen, the company has decided to discontinue the development and commercialization of aducanumab (Aduhelm) 100 mg/mL injection for intravenous use and will terminate its postmarking confirmatory phase 4 ENVISION study. The decision was not based on efficacy or safety concerns, but rather to reallocate funds towards Biogen’s Alzheimer disease (AD) pipeline. Aducanumab, an antiamyloid therapy, was approved under the accelerated approval pathway in June 2021 as a treatment for patients with early-stage AD with a confirmed presence of amyloid-ß. In January 2023, Biogen began a strategic review of its research and development efforts, with the intention to gauge external financing and partnership opportunities. After considering the time and investment required for ENVISION, as well as the likely advancements by the time it would be complete, the company decided to change gears.

Months after the Committee for Medicinal Products for Human Use (CHMP) refused to convert ataluren’s (Translarna; PTC Therapeutics) conditional marketing authorization, the committee has now issued a negative opinion following its re-examination procedure. Ataluren, a protein restoration therapy for patients with nonsense mutation Duchenne muscular dystrophy (DMD), was originally approved in Europe under conditional circumstances in 2014. Per European regulations, the European Commission has 67 days to adopt the opinion. Ataluren is a small molecule treatment that allows for stop-codon read-through to produce dystrophin in patients with nonsense mutations, which affects up to 15% of DMD diagnoses.

Sarepta Therapeutics has announced new data from part B of its phase 2 MOMENTUM study with results showing that treatment with SRP-5051 resulted in increased dystrophin expression and exon skipping among patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. After 28 weeks of treatment, those on high-dose SRP-5051 (30 mg/kg every 4 weeks; n = 20) demonstrated a 5.17% mean dystrophin expression as measured by western blot and mean exon-skipping of 11.11%, as measured by digital drop polymerase chain reaction. Notably, there were a number of treatment-emergent cases of hypomagnesemia, which had been previously identified, and were subsequently managed and monitored through prophylactic magnesium supplementation as part of the study protocol.

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