Business
Biogen and Ionis Announce Topline Phase 1 Study Results of Investigational Drug in C9orf72 Amyotrophic Lateral Sclerosis
BIIB078, an investigational antisense oligonucleotide for C9orf72-associated amyotrophic lateral sclerosis (ALS), did not show clinical benefit; clinical
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"BIIB078, an investigational antisense oligonucleotide for C9orf72-associated amyotrophic lateral sclerosis (ALS), did not show clinical benefit; clinical program will be discontinued Biogen and Ionis remain committed to their decade-long pursuit of advancing ALS research and developing therapies for all forms of this progressive and fatal neurodegenerative disease CAMBRIDGE, Mass. and CARLSBAD, Calif., March 28, 2022 /PRNewswire/ -- Biogen Inc. (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced topline results from the Phase 1 study of BIIB078 (IONIS-C9Rx), an investigational antisense oligonucleotide (ASO) for people with C9orf72-associated amyotrophic lateral sclerosis (ALS).\n\n \n \n \n \n \n \n\n \nIn this Phase 1 study, BIIB078 was generally well-tolerated. The adverse events (AEs) were mostly mild to moderate in severity and occurred at a similar rate across BIIB078 and placebo groups. The most common AEs were fall, procedural pain and headache.\nBIIB078 did not meet any secondary efficacy endpoints and it did not demonstrate clinical benefit. In the dose cohorts up to 60 mg there were no consistent differences between the BIIB078 group and the placebo group. Participants in the BIIB078 90 mg dose cohort trended toward a greater decline than those in the placebo group across secondary endpoints. Based on these results, the BIIB078 clinical development program will be discontinued, including its ongoing open-label extension study.\n\"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,\" said Toby Ferguson, M.D., Ph.D., Vice President and Head of the Neuromuscular Development Unit at Biogen. \"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. We remain focused on pioneering new treatments that will positively impact people living with this debilitating disease.\"\n\"C9orf72-associated ALS is a complex genetic form of ALS and there are multiple mechanisms by which the scientific community believes the C9orf72 gene causes disease. We designed BIIB078 to test the prevailing hypothesis that the mechanisms of disease for C9orf72-associated ALS were cau...