On Wednesday, Amylyx Pharmaceuticals announced that they are planning to file a New Drug Application—the formal submission for a drug to be approved by the FDA—for their investigational drug, AMX0035, for the treatment of ALS by the end of the year. This announcement reverses previous statements in which the company stated that the FDA would require a full Phase 3 clinical trial before Amylyx could submit for approval.
First and foremost, this is a meaningful step forward. A full Phase 3 clinical trial typically takes 3-4 years from start to finish, during which time thousands of Americans with ALS would not be able to access AMX0035. And people deserve to try AMX0035 now—in the completed Phase 2 CENTAUR trial and its open label extension, this treatment slowed ALS and extended life without serious side effects. Per the FDA’s own 2019 ALS guidance, regulatory flexibility should be applied so that AMX0035 can be approved for Americans with ALS as soon as possible.
But beyond this primary step forward achievement, this week’s news from Amylyx can—and should—have a massive ripple effect on how ALS drugs are developed. Amylyx took a novel approach to AMX0035’s development, partnering with ALS patients, non-profit organizations, researchers, and doctors from day 1—and AMX0035 went from idea to FDA submission in only 5 years because of this collaborative spirit. In fact, Amylyx was our first industry collaborator at the then-pilot Project ALS Therapeutics Core at Columbia in 2017, where we tested AMX0035 in our range of preclinical models. We founded the Core so that potential ALS drug candidates could be assessed, in tests designed by world-leading ALS experts, rapidly and comprehensively—to set a new blueprint for ALS drug discovery and development. Our early effort with with Amylyx validated our approach and opened the doors for collaborations with, now, dozens of industry partners who work with us to evaluate their potential ALS treatments.
The AMX0035 ALS development program has been a true ALS community effort—from ALS non-profit funding that allowed the Phase 2 trial to begin, to Amylyx’s early scientific collaboration with Project ALS, to thousands of patient advocates speaking out to hold FDA to the letter of its guidance this year—showing the power of collaboration toward faster, more humane, and more informative ALS drug development.
The AMX0035 program and all involved have set important precedents for expedited preclinical testing, patient-focused clinical development, and open data sharing, that all ALS drug developers can—must—now match and build upon.