Orphazyme's pipeline-in-a-product whiffs in second trial, though FDA decision still awaits
Orphazyme’s central molecule has whiffed in its second major test.
The Danish biotech announced Monday that arimoclomol, their “pipeline-in-a-product” molecule, failed in a Phase III trial for inclusion body myositis, a rare muscle-wasting disease. The drug couldn’t prevent disease progression in a 150-person study, missing on both primary and secondary endpoints.
Orphazyme’s stock $ORPH tumbled 31% on the news, from $12.39 to $8.50.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 107,000+ biopharma pros reading Endpoints daily — and it's free.