Amylyx Announces ALS Investigational Agent, FDA Approves Golodirsen for DMD, Wave Life Sciences Discontinues Suvodirsen, Discontinuation of Gosuranemab


Neurology News Network for the week ending December 21, 2019.

This week's Neurology News Network covered the investigational agent AMX0035 in patients with ALS, as well as the FDA approval of golodirsen in Duchenne muscular dystrophy. Additionally, Neurology News Network discusses the discontinuation of suvodirsen and the results from a phase 2 study of gosuranemab.

Marco: Welcome to Neurology News Network. I’m Marco Meglio. Let’s get into the news from this week.

Amylyx Pharmaceuticals has announced that treatment with its investigational agent AMX0035 resulted in a statistically significant slowing of disease progression in patients with ALS compared with placebo. The results were based on change from baseline of assessments on the ALS Functional Rating Scale-Revised, the primary end point of the CENTAUR clinical trial. Notably, nearly 90% of the trial participants who completed the 24-week study went on to enroll in the ongoing open-label extension, which will readout data in 2020. The company plans to provide updates on regulatory plans, as well as information on expanded access, in early 2020.

The FDA has approved Sarepta Therapeutics’ golodirsen injection for the treatment of Duchenne muscular dystrophy with genetic mutations subject to skipping exon 53 of the dystrophin gene. Additionally, Wave Life Sciences has announced that it will discontinue its development of suvodirsen, an investigational treatment for Duchenne muscular dystrophy in patients with mutations amenable to exon 51 skipping after an interim analysis of an open-label extension study demonstrated no change from baseline in dystrophin expression with either the 3.5 mg/kg or 5 mg/kg doses of suvodirsen. The phase 2/3 DYSTANCE 51 trial will be discontinued in addition to the open-label extension.

Top-line results from the phase 2 PASSPORT study of gosuranemab failed to meet the primary and secondary endpoints in patients with progressive supranuclear palsy, leading to discontinuation of the trial. The double-blind, placebo-controlled, parallel-group study of gosuranemab included 490 participants randomly assigned to either 50 mg of gosuranemab or placebo. Patients were administered 50 mg of gosuranemab through IV infusion once every 4 weeks for 48 weeks in the double-blind treatment period followed by a 50 mg dose of gosuranemab once every 4 weeks from weeks 52 to 208. Biogen still plans to continue its phase 2 TANGO study of gosuranemab for mild cognitive impairment due to Alzheimer disease.

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