Amylyx Pharmaceuticals submits NDA for AMX 0035 to the FDA for the treatment of amyotrophic lateral sclerosis.

The NDA submission to the FDA is based on data from the CENTAUR trial, a placebo-controlled study evaluating 137 people with ALS. In this study, participants receiving AMX 0035 had statistically significant slowing of functional decline at the end of the 6-month randomized phase as measured by the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS. In an interim survival analysis conducted in all randomized participants from CENTAUR who were followed for up to three years, which included participants who continued to receive AMX 0035 in an open-label extension phase during the follow-up period, participants who started on AMX 0035 during the placebo-controlled phase of CENTAUR showed a 44% lower risk of death compared to those who started on placebo during the placebo-controlled phase (HR 0.56; 95% CI, 0.34-0.92). Median survival duration through the open-label long-term follow-up phase was 25.0 months (95% CI, 19.0-33.6 months) in the group that started on AMX 0035 and 18.5 months (95% CI, 13.5-23.2 months) in the group that started on placebo, a 6.5-month difference.

Overall, reported rates of adverse events and discontinuations were substantially similar between AMX 0035 and placebo groups during the 24-week randomized phase; however, gastrointestinal events occurred with greater frequency ( greater than 2%) in the AMX 0035 group. Detailed data from CENTAUR is published in the New England Journal of Medicine (NEJM) and Muscle and Nerve.

Amylyx has filed a New Drug Submission (NDS) for AMX 0035 for the treatment of ALS with Health Canada in June 2021.

Amylyx also intends to submit a Marketing Authorization Application (MAA) for AMX 0035 to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) by approximately the end of 2021 and to initiate a global Phase III clinical trial with sites in Europe and the United States in the fourth quarter of 2021. The Phase III PHOENIX trial of AMX 0035 for the treatment of people with ALS will assess the safety and efficacy of AMX 0035 in an international population of approximately 600 participants and build upon findings from the CENTAUR trial. Amylyx is currently exploring the possibility of an Expanded Access Program (EAP) in the United States. If implemented, the EAP would run in parallel with the ongoing Phase III PHOENIX trial and marketing application reviews. Further information about the EAP is expected in the fourth quarter of 2021.