Amylyx Pharmaceuticals is pulling from market one of the few approved treatments for ALS.
Rarely do drugmakers voluntarily withdraw products. In Amylyx’s case, the decision comes just weeks after a large clinical trial meant to confirm the benefits of its medicine instead found it no better than a placebo at slowing the fatal, nerve-destroying disease.
Starting Thursday, the medicine, which is sold as Relyvrio in the U.S. and Albrioza in Canada, will no longer be available for new patients. Those who are taking it and wish to continue may enter a free drug program. Additionally, a phase of that large trial that allows participants to continue on Relyvrio remains ongoing.
Relyvrio is Amylyx’s only approved product. The Massachusetts-based biotechnology company is now restructuring through a cost-savings plan that includes laying off approximately 70% of its workforce. That’s likely one of largest rounds of layoffs from a biotech so far this year, as Amylyx had 384 full-time employees as of Dec. 31, according to a regulatory filing.
Moving forward, Amylyx said it will to focus resources on several other drug programs. The company has been evaluating Relyvrio as a potential treatment for two other uncommon disorders, and has another drug for ALS, or amyotrophic lateral sclerosis, that hasn’t yet entered human testing.
Amylyx ended 2023 with $371 million worth of cash, cash equivalents and short-term investments. It expects these cash reserves to keep the company operational into 2026.
“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,” said Amylyx’s co-CEOs Joshua Cohen and Justin Klee, in a statement.
The withdrawal marks an abrupt end to Relyvrio’s highly unusual journey in ALS.
Dreamed up by Cohen and Klee when they were undergraduates at Brown University, the medicine offered a rare glimmer of hope for many ALS patients and caregivers. Results from a roughly 140-person clinical trial published in The New England Journal of Medicine showed those given Relyvrio as opposed to a placebo appeared to decline a little slower and live a median of five months longer.
The trial represented a rare win in a notoriously difficult area of drug development. Yet, the Food and Drug Administration wasn’t entirely convinced the results were persuasive enough to grant an approval. The FDA twice convened outside experts to weigh the pros and cons of Relyvrio. And, during the second of those meetings, a top agency official called on Amylyx to commit to a market withdrawal if it didn’t succeed in confirmatory testing.
Amylyx pledged to do so, and by the fall of 2022, had secured approval in the U.S. and Canada.
Since then, the drug has been met with high demand. Nearly 4,000 of the approximately 30,000 ALS patients in the U.S. were on Relyvrio by the end of last September.
Net revenue from the drug, which totaled $381 million last year, also surpassed Wall Street expectations. Before early March, when the confirmatory trial failed, Amylyx had a share price just shy of $19 and a market value of almost $1.3 billion. Shares traded at $2.80 Thursday morning, giving the company a market value of around $190 million.
