BioMarin halts development of BMN 401 after phase 3 miss

BioMarin Pharmaceutical announced it is ending all work on its experimental enzyme replacement therapy BMN 401 following a late‑stage trial that did not meet one of two primary goals in patients with ENPP1 deficiency.

The phase 3 study, presented in May, showed the drug failed to achieve the targeted endpoint, prompting the company to pause and review the data before deciding on the program’s future. After the review, BioMarin decided to discontinue the candidate across all planned indications.

The decision also ends work on related studies that Inozyme Pharma had begun on ABCC6 deficiency and end‑stage kidney disease before BioMarin acquired the firm. Analysts had already warned that the likelihood of success was low, and the termination aligns with those expectations.

The halted program represented a $270 million investment, and the news was disclosed after market close as part of the company’s second‑quarter results.