Amylyx Pharmaceuticals is withdrawing its drug for amyotrophic lateral sclerosis from the market. The decision comes a month after post-marketing clinical trial results showed the drug was no better than a placebo in treating the deadly neuromuscular disease.
Starting Thursday, Relyvrio (known as Albrioza in Canada) will no longer be available to new patients, Amylyx said. But patients who are currently receiving the drug in the U.S. and Canada and, in consultation with their doctors, wish to continue treatment can transition to a program that provides the drug free of charge.
Relyvrio’s withdrawal is voluntary. The drug received full FDA approval in 2022 based on the results of a small Phase 2 clinical trial. Although the FDA did not require a Phase 3 clinical trial for Relyvrio, Cambridge, Massachusetts-based Amylyx continued that trial in anticipation that the data would be needed for regulatory filings in other markets. Amylyx executives also vowed to pull Relyvrio from the market if the drug fails the larger, longer Phase 3 test.
Although Relyvrio will no longer be a commercial product, Amylyx intends for the drug’s clinical trials to serve as a foundation for future ALS research. At the suggestion of ALS specialists, Amylyx said it would continue to collect data on the drug’s impact on survival. Patients who completed the Phase 3 study had the option to continue receiving Relyvrio in an open-label extension study. This study is not yet completed. In the short term, phase 3 results are scheduled to be presented on April 16 during the annual meeting of the American Academy of Neurology in Denver.
While Amylyx is delisting Relyvrio, the company is conducting a corporate restructuring that will result in a reduction in headcount of approximately 70%. At the end of 2023, Amylyx employed 384 full-time employees, 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 balance at the end of last year was $371 million. Amylyx said Thursday that it expects the restructuring to leave enough cash for 2026, when the company could have key data for its other research programs.
Relyvrio, codenamed AMX0035 in development, consists of two small molecules, each targeting a different neurodegenerative pathway. By treating both factors at the same time, the aim is to reduce the cellular stress that leads to the death of neurons. Although ALS was the primary indication for AMX0035, the company also tested it in other neurodegenerative diseases. A Phase 3 study of AMX0035 is currently ongoing in progressive supranuclear palsy, a rare neurodegenerative disease characterized by the accumulation of tau protein in the brain. An interim analysis is expected in mid-2025. AMX0035 is also being evaluated in a Phase 2 trial in Wolfram syndrome, an inherited neurodegenerative disorder. The company said it will release preliminary data from the Wolfram Syndrome study during a webcast on April 10.
Amylyx has another ALS drug candidate, AMX0114. This drug is an antisense oligonucleotide that targets 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,” Camille Bedrosian, chief medical officer of Amylyx, said in a prepared statement. “In our preclinical studies of AMX0114 and in several independent published studies, inhibition of calpain-2 reduced cell death and degeneration and lowered neurofilament levels.”
Amylyx expects to begin clinical testing of AMX0114 in ALS in the second half of this year.
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