We are thrilled to report that this year’s abstracts will be published electronically in Muscle and Nerve.
Trial News & Events
In an open-label extension trial, tofersen showed significant reductions in SOD1 protein and neurofilament light over a 12-month period. It is now expected to be reviewed by the FDA by early Q2 2023.
"Healey & AMG Center announces top line results in ALS platform trial with CMN-Au8," published by the Sean M. Healey & AMG Center for ALS
A secondary endpoint analysis of survival demonstrated a significant reduction in risk of death or permanently assisted ventilation when adjusting for baseline risk imbalances in the CNM-Au8 regimen for the 30 mg dose at 24 weeks
"Amylyx Pharmaceuticals Announces FDA Approval of RELYVRIO™ for the Treatment of ALS," published by Amylyx Pharmaceuticals
RELYVRIO (previously known as AMX0035 in the U.S.) is an oral, fixed-dose combination therapy for the treatment of adults with ALS
"Verdiperstat Fails to Differentiate From Placebo in Critical HEALEY ALS Platform Trial," published by NeurologyLive
Verdiperstat, Biohaven’s investigational first-in-class potent, selective, brain-penetrant, irreversible myeloperoxidase (MPO) enzyme inhibitor, failed to statistically differentiate from placebo in newly announced findings from the pivotal HEALEY ALS Platform Trial.
"The New England Journal of Medicine Publishes Pivotal Tofersen Data that Show Benefits in Rare, Genetic Form of ALS," published by Biogen
12-month data show earlier initiation of tofersen slowed decline across critical measures of function and strength in people diagnosed with SOD1-ALS
"Amylyx Pharmaceuticals Announces Donated CENTAUR Clinical Trial Data Now Available to Help Advance Science in ALS for Future Treatments and Discoveries," published by Amylyx Pharmaceuticals
Amylyx Pharmaceuticals announced that the donated clinical data from the placebo arm of the CENTAUR clinical trial that evaluated the safety, efficacy, and survival benefits of AMX0035 in adult participants with amyotrophic lateral sclerosis (ALS) are now available in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database.
"FDA Advisory Committee Votes to Recommend AMX0035 for Treatment of ALS," published by NeurologyLive
On September 7 the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, comprised of independent advisers to the FDA, voted 7—2 in favor of recommending AMX0035 for approval. The Advisory Committee’s recommendation is not binding. The FDA is expected to decide whether to approve AMX0035 by September 29.
"FDA Accepts Biogen’s New Drug Application and Grants Priority Review of Tofersen for a Rare, Genetic Form of ALS," published by Biogen
Biogen Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
"Investigational drug ABBV-CLS-7262 initiates design phase for entry into the HEALEY ALS Platform Trial," published by the Sean M. Healey & AMG Center for ALS
The HEALEY ALS Platform Trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in collaboration with the Northeast ALS Consortium (NEALS) is a trial in which multiple investigational products are tested and evaluated simultaneously to accelerate the development of potential new therapies for people with ALS.