Amylyx Pulls ALS Drug, Reduces Workforce

Amylyx Pharmaceuticals has started a process with the FDA and Health Canada to voluntarily discontinue the marketing authorizations for its ALS drug and immediately remove the product from the market in both the U.S. and Canada. The drug is sold under the name Relyvrio in the U.S.and is also known as AMX0035. The decision is based on topline results from the Phase 3 trial. Patients currently on therapy in the U.S. and Canada who, in consultation with their physician, wish to stay on treatment can be transitioned to a free drug program.

Amylyx simultaneously announced a restructuring to focus the company’s financial resources on upcoming clinical milestones and plans to reduce its workforce by approximately 70% and decrease external financial commitments outside of its priority areas. With these changes, Amylyx expects to have cash runway into 2026, which will allow the organization to deliver on key upcoming milestones.

“Our pipeline is supported by compelling clinical and preclinical science demonstrating the potential of AMX0035 and AMX0114 in neurodegenerative diseases. AMX0035 was designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum stress and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration. We are investigating AMX0035 in diseases where these two pathways are implicated, which includes Wolfram syndrome and progressive supranuclear palsy,” said Camille Bedrosian, MD, Chief Medical Officer of Amylyx.

Dr. Bedrosian continued, “We also remain focused on ALS and believe AMX0114 has strong potential for the treatment of ALS and other diseases. Calpain-2 is considered an essential protein in the process of axonal degeneration and has been repeatedly linked to neurofilament biology in published studies. In our preclinical studies of AMX0114 and in multiple independent published studies, inhibition of calpain-2 has reduced cell death and degeneration and decreased neurofilament levels. We expect to initiate a clinical trial studying AMX0114 in ALS in the second half of this year.” (Source: Amylyx Website, 04 April,2024)