Als treatment discontinued for now Based on data from a Phase 1 clinical trial, Biogen and Ionis Pharmaceuticals are discontinuing development of BIIB078, their experimental treatment candidate for amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene.
While the therapy was generally safe and well-tolerated in people with C9orf72-associated ALS, it resulted in no significant clinical benefit compared with a placebo.
The trial’s extension study (NCT04288856) — the only one currently active at this point — will be stopped. Full results of the therapy’s development will be presented at a future scientific meeting.
“We are incredibly grateful for the selfless commitment of the individuals with ALS who participated in the study, and the community’s dedication to advancing research for this devastating disease,” Toby Ferguson, MD, PhD, Biogen’s vice president and head of the neuromuscular development unit, said in a press release.
“While these were not the results we were hoping for, they are clear and will inform future research across our broad pipeline of investigational ALS therapies,” Ferguson said, adding that Biogen remains “focused on pioneering new treatments that will positively impact people living with this debilitating disease.”
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