BioMarin's Phase 3 Trial of BMN 401 for ENPP1 Deficiency Meets One Co-Primary Endpoint
BioMarin Pharmaceutical (BMRN) reported that its Phase 3 ENERGY 3 trial met one co-primary endpoint, showing statistically significant increases in plasma PPi levels compared to conventional therapy. However, the drug failed to improve radiographic measures of rickets severity and showed no positive trends in secondary endpoints.
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Topics: biotech drug_development pharma rare_diseases