BioMarin Pharmaceutical is reassessing its BMN 401 program in ENPP1 deficiency after the investigational enzyme replacement therapy failed to demonstrate clinical benefit in a Phase 3 study, despite achieving its biomarker goals. In the ENERGY 3 trial, BMN 401 significantly reduced plasma inorganic pyrophosphate levels in children aged 1 to 12 years with ENPP1 deficiency, a rare disorder that damages blood vessels, bone, and soft tissue. However, the biomarker improvements did not translate into better outcomes on key clinical measures, including the Radiographic Global Impression of Change rickets scale, where BMN 401 did not separate from control. The therapy also missed additional secondary endpoints related to rickets severity and growth, with the company reporting “no positive trends” on these measures.
BioMarin said it is “disappointed that the significant increases in plasma PPi observed with BMN 401 did not translate into meaningful clinical improvements,” according to Chief R&D Officer Greg Friberg. The company indicated it will review the full data set from ENERGY 3 and engage with investigators and regulators before determining next steps for BMN 401 in ENPP1 deficiency. BioMarin’s broader rare disease portfolio includes approved therapies and other late‑stage programs, and management is expected to provide additional pipeline commentary in future investor updates.
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