BioMarin’s BMN 401 Raises PPi Through 52 Weeks but Misses RGI-C Endpoint
BioMarin’s Phase 3 ENERGY 3 trial of BMN 401 in children aged 1–12 with ENPP1 deficiency showed a statistically significant increase in plasma inorganic pyrophosphate through week 52. The study failed to meet the Radiographic Global Impression of Change endpoint, with no improvement in RGI-C scores or secondary clinical measures.
1. ENERGY 3 Trial Results
The ENERGY 3 Phase 3 trial evaluated BMN 401 in 27 children aged 1–12 with ENPP1 deficiency over 52 weeks. Treatment achieved a statistically significant increase in plasma inorganic pyrophosphate compared to conventional therapy, meeting one of two co-primary endpoints.
2. Clinical Measures and Safety
Despite the PPi gains, there was no improvement in Radiographic Global Impression of Change scores, and secondary endpoints—including Rickets Severity Score and growth Z-scores—showed no positive trends. BMN 401 was generally well-tolerated with no new safety signals reported.
3. BioMarin’s Next Steps
BioMarin is reviewing the full dataset to determine next steps for the BMN 401 program. Detailed results will be shared at an upcoming medical meeting as the company evaluates potential regulatory strategies and further clinical development.