By Dhirendra Tripathi
Investing.com – Amylyx Pharmaceuticals (NASDAQ:AMLX) stock plunged more than 21% Thursday after an FDA panel voted against recommending the approval of an experimental drug for the treatment of amyotrophic lateral sclerosis.
ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death.
The AMX0035 is a proprietary oral fixed-dose combination of two small molecules.
The panel said study data failed to prove that the drug was effective in fighting the disease. The phase-2 trial comprised 137 participants.
The recommendations by the panel are non-binding and the final decision regarding approval of a pending new drug application is likely to be made by the FDA by June 29.
Amylyx is a clinical-stage biopharmaceutical company working on developing a novel therapeutic for ALS and other neurodegenerative diseases.