The ALS Association Webinar: Tofersen and the FDA Approval Process
Click here to watch a recording of The ALS Association webinar on Tofersen and the FDA Approval Process.
Trial News & Events
"Amylyx Pharmaceuticals Announces Completion of Enrollment in Global Phase 3 PHOENIX Trial of AMX0035 in ALS," published by Amylyx Pharmaceuticals
Amylyx anticipates topline results in 2024. The study enrolled 664 participants living with ALS.
"BrainStorm Cell Therapeutics Partners with NEALS, The ALS Association, and I AM ALS to Provide Public Access to Biospecimens from NurOwn's Phase 3 ALS Study," published by BrainStorm Cell Therapeutics Inc.
Serum and cerebrospinal fluid samples from the Phase 3 study of NurOwn® in ALS to be donated to the NEALS biorepository for use by the research community
"Healey & AMG Center for ALS announces new drug regimen for testing DNL343 in HEALEY ALS Platform Trial," published by the Sean M. Healey & AMG Center for ALS
The HEALEY ALS Platform Trial is adding a new investigational drug, DNL343, to continue to research new treatments for ALS (Amyotrophic Lateral Sclerosis or Lou Gehrig’s disease).
Annual NEALS Meeting
The Annual Meeting of the Northeast Amyotrophic Lateral Sclerosis (NEALS) Consortium brings together clinicians, researchers, and members of the NEALS Consortium to share the latest data and findings in the care of ALS and discuss the hottest topics in neuromuscular medicine.
"Mitsubishi Tanabe Pharma America Presents Interim Data from REFINE-ALS Biomarker Study at 21st Annual NEALS Meeting," published by Mitsubishi Tanabe Pharma America
Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of an interim analysis of the Phase 4 REFINE-ALS study, designed to identify predictive and pharmacodynamic biomarkers to measure the effect of RADICAVA® (edaravone) in people with amyotrophic lateral sclerosis (ALS).
21st Annual NEALS Meeting Abstracts published in Muscle and Nerve
We are thrilled to report that this year’s abstracts will be published electronically in Muscle and Nerve.
"FDA Extends Review Date for ALS Treatment Tofersen," published by NeurologyLive
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
