The FDA will evaluate AMX0035, an investigational oral fixed-dose drug intended for people diagnosed with ALS.
From Left: Amylyx CEOs Justin Klee and Josh Cohen, Amylyx Pharmaceuticals/Courtesy Amylyx Pharmaceuticals
Amylyx Pharmaceuticals announced that it is meeting with the U.S. Food and Drug Administration on March 30, 2022, to review the New Drug Application (NDA) of its much-discussed treatment for amyotrophic lateral sclerosis (ALS).
At the virtual meeting, the regulator’s Peripheral and Central Nervous System Drugs Advisory Committee will evaluate AMX0035, an investigational oral fixed-dose drug intended for people diagnosed with ALS.
AMX0035 is composed of sodium phenylbutyrate (PB) and taurursodiol (TURSO, also known as ursodoxicoltaurine). PB is a small molecular chaperone that reduces the unfolded protein response, thereby preventing cell death, while TURSO is a Bax inhibitor that lowers cell death via apoptosis. Together, they may have the ability to reduce neuronal death and dysfunction.
The drug is engineered to target the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other similar diseases. The FDA accepted Amylyx’s application for NDA review in December 2021, granting it Priority Review and assigning a Prescription Drug User Fee Act date of June 29, 2022. The company is also reportedly planning to submit an Expanded Access Program to the regulator to launch in the U.S. for patients who were unable to participate in its Phase III PHOENIX clinical trial.
The FDA’s acceptance of the NDA for AMX0035 was based on the Phase II CENTAUR trial, which evaluated 137 ALS patients over the course of six months. Details were published in the New England Journal of Medicine and Muscle and Nerve.
“There is a lot of progress in ALS research; and now with AMX0035’s NDA acceptance, we believe we are one step closer to a potential new treatment option. We look forward to seeing AMX0035 potentially progress through the regulatory review process as we continue to investigate its therapeutic potential in the global Phase 3 PHOENIX clinical trial as part of the collaboration with the Northeast ALS (NEALS) Consortium and the Treatment Research Initiative to Cure ALS (TRICALS) in Europe,” said Merit Cudkowicz, M.D., co-principal investigator of the CENTAUR trial, in an earlier statement.
In other related ALS news, Aquinnah Pharmaceuticals announced it signed a deal with Roche to advance experimental treatments for the disease. Under terms of the agreement, the two firms will collaborate in research efforts for ALS and then Roche will carry them forward to development and global commercialization.
Aquinnah’s research integrates stress granule biology, bimolecular condensate formation and RNA binding proteins to create oral small molecule drug candidates to treat a range of neurodegenerative diseases, such as ALS, frontotemporal disease (FTD) and Alzheimer’s disease. Its approach involves eliminating pathologies caused by TDP-43 or tau proteins. The company is financially backed by AbbVie, Pfizer and Takeda Pharmaceuticals.
