Transposon Announces Interim Results from a Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related ALS
TPN-101 showed a clinical effect on Vital Capacity, an objective respiratory measure that correlates with mortality in patients with ALS
Trial News & Events
National Institutes of Health (NIH) funds New Multi-Institutional ALS Clinical Research Consortium
The NIH has established a transformative national ALS clinical research consortium called Access for All in ALS (ALL ALS). This new consortium is a multi-institutional effort and aims to disrupt the ALS clinical research landscape in the U.S.
"FDA Grants Accelerated Approval for QALSODY™ (tofersen) for SOD1-ALS, a Major Scientific Advancement as the First Treatment to Target a Genetic Cause of ALS," published by Biogen
QALSODY is the first approved treatment to target a genetic cause of ALS.
"Biogen Provides Update on FDA Advisory Committee Meeting on Tofersen for SOD1-ALS," published by Biogen
FDA Advisory Committees provide non-binding recommendations for consideration by the FDA. The New Drug Application for tofersen for the treatment of SOD1-ALS was submitted to the FDA for consideration under accelerated approval. The FDA is continuing its review of tofersen with a Prescription Drug User Fee Act action date of April 25, 2023.
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.
"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).
