As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s de­ci­sion on Amy­lyx’s ALS drug is set to come out some­time Thurs­day. In a space with few drugs, any ap­proval would be a ma­jor land­mark.

But else­where in the ALS field, things are a bit more tepid.

Thurs­day morn­ing, Bio­haven an­nounced that its drug verdiper­stat failed its arm of an ALS plat­form tri­al led by Mass­a­chu­setts Gen­er­al Hos­pi­tal. Ac­cord­ing to a press re­lease, the drug did not meet its pri­ma­ry end­point — im­prove­ment on an ALS func­tion­al sta­tus test — or any key sec­ondary end­points at 24 weeks. The tri­al had en­rolled 167 pa­tients, giv­ing them ei­ther verdiper­stat or place­bo twice a day.

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