Amylyx Withdraws ALS Drug From Market; Restructuring Will Slash 70% of Staff

Amylyx Pharmaceuticals is withdrawing its drug for amyotrophic lateral sclerosis from the market, a decision that comes a month after post-marketing clinical trial results showed the medication was no better than a placebo at treating the fatal neuromuscular disease.

Starting Thursday, Relyvrio (known as Albrioza in Canada) will no longer be available for new patients, Amylyx said. But patients currently receiving the drug in the U.S. and Canada who, in consultation with their physicians, wish to stay on treatment may transition to a program that will provide the drug for free.

The withdrawal of Relyvrio is voluntary. The drug received full FDA approval in 2022 based on results from a small Phase 2 clinical trial. Though the FDA did not require a Phase 3 clinical trial for Relyvrio, Cambridge, Massachusetts-based Amylyx continued this study with the expectation that the data would be required for regulatory submissions in other markets. Amylyx executives also pledged to remove Relyvrio from the market if the drug failed the larger and longer Phase 3 test.

Though Relyvrio will cease to be a commercial product, Amylyx intends for the drug’s clinical studies to inform future ALS research. At the encouragement of ALS specialists, Amylyx said it will continue to gather data on the drug’s effect on survival. Patients who completed the Phase 3 study had the option to continue to receive Relyvrio in an open-label extension study. That study is ongoing. In the nearer term, the Phase 3 results are scheduled to be presented on April 16 during the American Academy of Neurology annual meeting in Denver.

As Amylyx pulls Relyvrio from the market, the company is implementing a corporate restructuring that will slash headcount by about 70%. At the end of 2023, Amylyx employed 384 full-time workers, according to the company’s annual report. Severance payments and other related expenses will cost about $19 million, Amylyx said in a regulatory filing. The company’s cash position at the end of last year was $371 million. Amylyx said Thursday that it expects the restructuring leaves enough cash to last into 2026, when it could have key data for its other research programs.

Relyvrio, known in development by the code name AMX0035, is comprised of two small molecules, each one targeting a different neurodegenerative pathway. Addressing both of them together is intended to reduce the cellular stress that leads neurons to die. Though ALS was the lead indication for AMX0035, the company also tested it in other neurodegenerative diseases. A Phase 3 study of AMX0035 is ongoing in progressive supranuclear palsy, a rare neurodegenerative disorder characterized by the buildup of tau protein in the brain. An interim analysis is expected in mid-2025. AMX0035 is also being evaluated in a Phase 2 test in Wolfram syndrome, an inherited neurodegenerative disorder. The company said it will report interim data from the Wolfram syndrome study during an April 10 webcast.

Amylyx has another ALS drug candidate, AMX0114. This drug is an antisense oligonucleotide designed to target the gene that encodes calpain-2, an enzyme associated with neurodegeneration.

“Calpain-2 is considered an essential protein in the process of axonal degeneration and has been repeatedly linked to neurofilament biology in published studies,” Amylyx Chief Medical Officer Camille Bedrosian said in a prepared statement. “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.”

Amylyx expects to begin clinical testing of AMX0114 in ALS in the second half of this year.

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