BioMarin’s investigational enzyme replacement therapy BMN 401 (INZ-701) for ENPP1 deficiency has delivered mixed results in the pivotal ENERGY 3 Phase III trial. While the study met one co-primary endpoint by significantly increasing plasma inorganic pyrophosphate (PPi) levels — a key biomarker — it failed to show improvement in the other co-primary endpoint, the Radiographic Global Impression of Change (RGI-C) score, which measures clinical bone healing. No positive trends were observed in key secondary endpoints, including rickets severity and growth.

The trial evaluated the subcutaneous therapy in children aged 1 to 12 with ENPP1 deficiency, a rare genetic disorder causing progressive vascular and bone damage. Despite the biomarker response, the lack of clinical benefit casts significant doubt on the regulatory path forward. BioMarin is currently evaluating the data to determine next steps for the program.

PharmCube's NextBiopharm® database shows that BMN 401 is being developed in five additional indications. Click here to request a free trial for NextBiopharm®.