- Amylyx - founded by two Brown University students in 2013 - has developed a therapy for Amyotrophic Lateral Sclerosis, AMX-0035.
- Its Phase 2 CENTAUR trial data suggested the drug showed benefit on both function and survival - the first ALS drug to do so.
- ALS is an area of high unmet need, but the FDA has highlighted some potential issues with Amylyx's trial data, and an Advisory Committee recently voted against approval.
- Earlier this month, however, Health Canada opted to approve the drug based on the CENTAUR trial data - a surprising move that led to a 92% surge price surge.
- The FDA has since delayed its approval decision by three months to September. It's an emotive, complex decision, but ultimately Amylyx may need to conduct a full Phase 3 trial to prove its thesis.
For further details see:
Amylyx Pharmaceuticals: Share Price Bump On Canada Approval For ALS Drug May Not Last