Amylyx to Remove ALS Drug from Market and Cut 70 Percent of Workforce

Rare Daily Staff

Amylyx Pharmaceuticals is voluntarily removing its ALS drug Relyvrio from the U.S and Canadian markets and cutting up to 70 percent of its workforce in a corporate restructuring to focus on upcoming clinical milestones and extend its cash runway into 2026.

The decision follows news that topline results from Amylyx’s phase 3 PHOENIX trial failed to meet pre-specified primary and secondary endpoints. The U.S. Food and Drug Administration approved Relyvrio in 2022 on the basis of a single, phase 2 trial with 137 patients.

Relyvrio, marketed as Albrioza in Canada, will no longer be available for new patients. Patients currently on therapy in the United States and Canada who, in consultation with their physician, wish to stay on treatment can be transitioned to a free drug program.

Amyotrophic lateral sclerosis (ALS) is a rare, progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. People living with ALS have a median survival of approximately two years from diagnosis.

“While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS and other neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “The decision to remove Relyvrio/Albrioza from the market and provide therapy free of charge for those who wish to continue was informed by the PHOENIX trial results, engagement with regulatory authorities, and discussions with the ALS community.”

Amylyx said it will continue to evaluate and share learnings from PHOENIX to help inform future ALS research and intends to continue to collect available data on survival at the encouragement of ALS specialists. The PHOENIX Open Label Extension is ongoing. Topline data from PHOENIX will be presented at the American Academy of Neurology Annual Meeting in Denver and online, taking place April 13-18, 2024.

Amylyx will continue to advance two key programs targeting neurodegenerative diseases, investigating its lead asset AMX0035 in Wolfram syndrome and progressive supranuclear palsy (PSP), and AMX0114, an antisense oligonucleotide targeting calpain-2, in ALS.

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.

The company plans to present interim data from the phase 2 HELIOS study of AMX0035 in Wolfram syndrome, a rare, genetic, fatal neurodegenerative disease with no FDA-approved treatment options, later this month. The phase 3 ORION study remains ongoing to evaluate AMX0035 for the treatment of progressive supranuclear palsy, a rare neurodegenerative disorder characterized as a tauopathy. Amylyx anticipates an interim analysis to evaluate the data from ORION in mid-2025. The company also expects to initiate a clinical trial studying AMX0114 in ALS in the second half of 2024.

Photo: Justin Klee, Co-CEO of Amylyx