Ulefnersen slows disease progression in Phase 3 ALS trial A win for autoimmune disease, a set back for Alzheimer’s disease, an FDA backing for a multi-cancer blood test, and more led the news this week. Brought to you byDDN editorial team

Ulefnersen slows disease progression in Phase 3 ALS trial A win for autoimmune disease, a set back for Alzheimer’s disease, an FDA backing for a multi-cancer blood test, and more led the news this week. Brought to you byDDN editorial team Otsuka and Ionis Pharmaceuticals’ investigational RNA-targeted therapy ulefnersen has met the primary endpoint in a Phase 3 trial of FUS-associated amyotrophic lateral sclerosis (FUS-ALS), a rare and rapidly progressive form of the disease caused by mutations in the FUS gene. The FUSION study enrolled 89 patients, with 73 included in the primary analysis, and found that ulefnersen significantly slowed the combined measure of functional decline and survival at week 72 compared with placebo. The therapy also outperformed placebo on secondary measures, including a biomarker of neurodegeneration and time to death, permanent ventilation, rescue treatment, or withdrawal due to disease progression. Ulefnersen is designed to reduce the production of FUS protein by binding FUS pre-messenger RNA, including mutant forms that contribute to motor neuron degeneration. Otsuka plans to discuss the results with regulatory authorities and has launched an early-access program for eligible patients with genetically confirmed FUS-ALS who cannot participate in a clinical trial. There are currently no approved therapies specifically targeting the underlying genetic cause of FUS-ALS. – Bree Foster https://www.drugdiscoverynews.com/ulefnersen-slows-disease-progression-in-phase-3-als-trial-17546