FDA Panel Votes Down On Amylyx's ALS Drug, Further Fading Its Hope For Regulatory Success

An advisory committee of the FDA voted against the approval of Amylyx Pharmaceuticals Inc's AMLX AMX0035, an investigational therapy for amyotrophic lateral sclerosis.

• On the question of "do the data from single-trial & open-label study establish a conclusion that the drug is effective?" four panelists voted "Yes," while six committee members voted "No."
• The FDA's advisory committees issue non-binding recommendations. However, the regulator usually follows them before finalizing authorizations, with an assigned PDUFA action date of June 29, 2022, for AMX0035.
• Related: Amylyx Shares Plummet On Skeptical FDA Adcomm Docs On ALS Drug.
• Amylyx's application is underpinned by a Phase 2/3 trial called Centaur and an open-label extension, which showed a slowing disease progression. 
• But the single study was "not exceptionally persuasive," according to the briefing document the FDA released ahead of the meeting. 
• Amylyx is already conducting a Phase 3 trial called Phoenix but is hoping for accelerated approval.
• The company held cash and equivalents of $96.1 million as of December 31, 2021.
• Price Action: Currently, Amylyx shares are on hold for trading.