NasdaqGS:BMRNBiotechs
BioMarin Trial Setback Puts Rare Disease Pipeline And Valuation In Focus
BioMarin Pharmaceutical (NasdaqGS:BMRN) reported that BMN 401 missed one of two co-primary endpoints in the Phase 3 ENERGY 3 trial for ENPP1 deficiency.
The therapy did not show improvement in skeletal health or key secondary outcomes, raising questions about its regulatory and commercial outlook.
The result affects BioMarin's rare disease pipeline at a time when the company is working to broaden its revenue base beyond existing treatments.
For investors watching rare disease drug...