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BioMarin's ENPP1 Drug: Mixed Results in Pediatric Trial
20 May
Summary
- BMN 401 increased plasma inorganic pyrophosphate levels significantly.
- The drug did not show improvement in clinical impact assessments.
- No positive trends were observed in secondary disease severity scores.
BioMarin Pharmaceutical's BMN 401, a candidate enzyme replacement therapy, has shown mixed results in its Phase III ENERGY 3 trial for children with ENPP1 deficiency. The trial met one co-primary endpoint, demonstrating statistically significant increases in plasma inorganic pyrophosphate (PPi) levels by week 52 compared to conventional therapy.
Despite the rise in PPi, the study did not meet its second co-primary endpoint, failing to show a related improvement in Radiographic Global Impression of Change (RGI-C) scores, which assess clinical impact in pediatric rickets. Furthermore, no positive trends emerged for secondary endpoints like the Rickets Severity Score or growth Z-scores.
The drug candidate, BMN 401, was found to be generally well-tolerated with no new safety concerns identified. BioMarin is now evaluating the data to determine the path forward for treating this rare, progressive genetic condition.
Separately, in April 2025, BioMarin reported success with its Palynziq trial for adolescents with phenylketonuria, meeting its primary endpoint for reducing blood phenylalanine levels.
