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BioMarin drops BMN 401 following unsuccessful pivotal trial

BioMarin Discontinues BMN 401 Across All Indications After Pivotal ENPP1 Trial Fails Co-Primary Endpoints

Edited by Robert McTiernan··1 min read
BioMarin drops BMN 401 following unsuccessful pivotal trial

BioMarin Pharmaceutical (Nasdaq: BMRN) has discontinued development of BMN 401, its investigational therapy for ENPP1 deficiency, following the failure of the pivotal Phase III ENERGY 3 trial, removing the program from its pipeline across all indications.

The decision was disclosed in the company's Q2 2026 financial results on August 6, marking the end of BioMarin's efforts to develop BMN 401 for ENPP1 deficiency, a rare inherited metabolic disorder characterized by ectopic calcification and phosphate dysregulation. The company did not announce plans to pursue alternative indications, seek a development partner, or out-license the asset.

BioMarin announced in May 2026 that the Phase III ENERGY 3 trial failed to meet its co-primary endpoints, preventing the study from supporting a regulatory submission. In its latest earnings release, the company confirmed that development of BMN 401 (formerly INZ-701) has been terminated and the program has been removed from its active pipeline.

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BMN 401 represented one of BioMarin's most advanced investigational rare disease programs outside its marketed portfolio. Its discontinuation narrows the company's late-stage development pipeline while reinforcing the high clinical and regulatory risks associated with developing therapies for ultra-rare metabolic disorders.

BioMarin's commercial business remains anchored by therapies for rare genetic diseases, including enzyme replacement products and the hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec). The company did not disclose any restructuring or resource reallocation related to the termination of BMN 401 in its Q2 announcement.

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