Amylyx ALS drug fails crucial study, putting company’s future in doubt

 A large clinical trial meant to confirm the benefits of a new ALS medication has instead failed, dealing a major blow to the therapy’s developer as well as patients who had hung their hopes on it.

Amylyx Pharmaceuticals disclosed Friday morning that the drug, known as Relyvrio in the U.S., didn’t meet the trial’s main or secondary goals. Though well-tolerated by participants, Relyvrio was not significantly better than a placebo at changing the trajectory of their disease.

For Amylyx, the results are a monumental setback. The Massachusetts-based biotechnology company was built entirely around Relyvrio, which received approval from the Food and Drug Administration in the fall of 2022. Early sales made Amylyx profitable – a rare victory for any young drugmaker. Some Wall Street analysts have estimated Relyvrio could bring in $1 billion or more per year at its peak.

Now, the company’s future is unclear. Before Relyvrio’s approval, Amylyx co-founders Justin Klee and Joshua Cohen pledged, at the request of a top FDA official, to pull their drug from the market should confirmatory testing fail.

In a statement, Amylyx said that sometime in the next eight weeks, it will share its plans for Relyvrio, which “may include voluntarily withdrawing [the drug] from the market.” The company also intends to discuss the new results with regulators, doctors and the broader amyotrophic lateral sclerosis community.

In the meantime, Amylyx will pause promoting the product.

“Our main priority at the moment is sharing the information with people living with ALS
and their treating physicians,” said Klee and Cohen, who also serve as the company’s co-CEOs.

“We will be led in our decisions by two key principles: doing what is right for people living with ALS, informed by regulatory authorities and the ALS community, and by what the science tells us,” they added.

By the end of September, nearly 4,000 of the roughly 30,000 ALS patients in the U.S. were on Relyvrio, according to Amylyx’s estimates. The drug is also available in Canada, where it’s sold under the brand name Albrioza. Amylyx attempted to secure approval in the European Union, too, but wasn’t successful, as regulators there had concerns about the treatment’s overall effectiveness and the reliability of the supporting data.

https://finance.yahoo.com/m/5cbf4436-ed3e-3b98-820a-d29aa8ce6d7a/amylyx-als-drug-fails-crucial.html