Fresh off ALS drug withdrawal, Amylyx touts interim rare disease data for the same therapy

A week af­ter vol­un­tar­i­ly pulling its ALS drug off the mar­ket fol­low­ing a failed Phase 3 tri­al, Amy­lyx Phar­ma­ceu­ti­cals out­lined a new di­rec­tion for the drug by pre­sent­ing in­ter­im da­ta in an­oth­er dis­ease.

On Wednes­day, the com­pa­ny shared new da­ta for AMX0035, the same chem­i­cal com­pound as the now-de­funct ALS drug Re­lyvrio, in a rare ge­net­ic con­di­tion called Wol­fram syn­drome. It’s a dis­ease that usu­al­ly man­i­fests in child­hood, with pa­tients ex­pe­ri­enc­ing symp­toms typ­i­cal­ly seen in di­a­betes (high blood sug­ar, high urine pro­duc­tion) that may progress to blind­ness and deaf­ness.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.