Ionis Pharmaceuticals

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…

Ionis Pharmaceuticals is opening a Phase 3 safety and efficacy trial of ION363 (jacifusen) in amyotrophic lateral sclerosis (ALS) patients with confirmed mutations in the FUS gene, a known cause of juvenile-onset disease. The trial (NCT04768972) will enroll up to 64 people, ranging in age from 12 to 65,…

Biogen will not offer early access to tofersen, its experimental therapy for familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, at least until data from its pivotal clinical trial are analyzed. Now in its Phase 3 portion, the study (NCT02623699) is testing the…

A Phase 1/2 clinical trial investigating ION541, Ionis Pharmaceuticals‘ third antisense treatment and its first for sporadic, rather than familial, amyotrophic lateral sclerosis (ALS), has started dosing, the company announced. This ascending dose safety trial (NCT04494256) is recruiting up to 70 patients…

Eight patients will receive jacifusen, an experimental therapy for amyotrophic lateral sclerosis (ALS) caused by FUS gene mutations, under a joint effort from the ALS Association, Project ALS, and Columbia University’s Eleanor and Lou Gehrig ALS Center. The new clinical research program, which will be supported…

Biogen’s investigational therapy tofersen can significantly reduce toxic levels of SOD1 protein and may slow disability progression in people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations, interim study results show. The positive data from a Phase 1/2 clinical trial support advancing the experimental compound into Phase 3…

The Muscular Dystrophy Association (MDA) is financing eight new research projects aimed at developing new treatments and markers of disease progression for amyotrophic lateral sclerosis (ALS). The projects will investigate ALS mechanisms of disease, genetic causes, and pinpoint new therapy targets that can be used to develop better treatments. The…

Biogen has decided to exercise its option to acquire from Ionis Pharmaceuticals the exclusive rights to develop and commercialize BIIB067 as a potential treatment for amyotrophic lateral sclerosis (ALS). The decision to license the investigational therapy is covered under the terms of a collaborative agreement between the two companies…

IONIS-SOD1Rx (BIIB067), an investigational therapy for amyotrophic lateral sclerosis (ALS) developed by Biogen and Ionis Pharmaceuticals, was shown to prolong survival and reverse neuromuscular damage in mouse and rat models…