FDA Sets Up Adcomm To Review Amylyx's ALS Drug Next Month

The FDA's Peripheral and Central Nervous System Drugs Advisory Committee will review Amylyx Pharmaceuticals Inc's AMLX marketing application for AMX0035 (sodium phenylbutyrate [PB] and taurursodiol) on March 30. 

• AMX0035 is an investigational therapy to treat amyotrophic lateral sclerosis (ALS). 
• The Company applied in December last year, and the FDA has granted Priority Review and assigned a Prescription Drug User Fee Act date for AMX0035 of June 29, 2022.
• The submission to the FDA was based on CENTAUR Phase 2 trial data in 137 people with ALS. Based on a functional rating scale, which Amylyx used to measure its primary endpoint, AMX0035 showed a statistically significant reduction in disease progression over about six months.
• A survival analysis conducted in all randomized participants who were followed for up to three years, AMX0035, demonstrated a 44% lower risk of death than those who started on placebo during the placebo-controlled phase. 
• Median survival duration through the open-label long-term follow-up phase was 25.0 months in the group that started on AMX0035 and 18.5 months in the group that began on placebo, a 6.5-month difference.
• Price Action: AMLX shares are down 0.46% at $21.72 during the market session on the last check Thursday.