LLY - An ALS Drug Fails Again

2024-03-09 00:20:00 ET

Summary

• The ALS drug (Relyvrio) had been put through a Phase II trial by its developer, Amylyx.
• Amylyx was already working on a Phase III trial, and they committed to withdrawing the drug if this trial showed no efficacy.
• Just like with the recent approvals in Alzheimer's, the patient advocacy groups took victory laps after Relyvrio was approved, and they were a big part of the pressure that made the FDA reverse its initial correct decision.

Two years ago, I wrote here about a new drug that was up for approval for ALS patients (who sadly have very few therapeutic options at all). The drug (Relyvrio, a mixture of sodium phenylbutyrate and taurursodiol) had been put through a Phase II trial by its developer (Amylyx), but as you'll see from that earlier post and the references therein, the data were not compelling. There were a number of statistical problems with the trial, and even as presented, it barely made the case for efficacy. When the FDA's own statisticians tried to correct for the gaps in the data, it seemed to make the readout worse every time. An uncharitable view would be that Amylyx ( AMLX ) ended up presenting the clinical package from the only angle that made it even possible to believe that the drug worked, and the FDA advisory committee ended up voting against approval....

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