BioMarin drops BMN 401 after Phase 3 trial misses clinical endpoints, ending $270M Inozyme asset

BioMarin announced it will stop development of BMN 401, an enzyme replacement therapy acquired in its $270 million purchase of Inozyme Pharma.

The decision follows the Phase 3 ENERGY trial in patients with ENPP1 deficiency, where the drug lowered a disease biomarker but did not improve clinical outcomes, meeting only one of two primary endpoints.

The failure leads BioMarin to discontinue BMN 401 across all studied rare diseases, including ENPP1 deficiency, ABCC6 deficiency and calcific uremic arteriopathy, as reported in its Q2 earnings release.