BioMarin Pharmaceutical Reports No Functional Improvement in BMN 401 ENPP1 Deficiency Study
MT Newswires Live
May 18
BioMarin Pharmaceutical's (BMRN) Phase 3 ENERGY 3 trial of BMN 401 in children with ENPP1 deficiency met one of two co-primary endpoints but did not show improvement in key clinical outcomes, the company said Monday.
The study showed a statistically significant increase in plasma inorganic pyrophosphate levels but did not improve Radiographic Global Impression of Change scores, the primary measure of rickets severity.
The company added that no notable trends were observed in secondary endpoints, and the therapy was generally well tolerated with no new safety concerns reported.
Shares of BioMarin Pharmaceutical were down over 1% in Monday trading.
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