BioMarin axes $270M rare disease asset after missing phase 3 endpoint

Biotech BioMarin axes $270M rare disease asset after missing phase 3 endpoint By Nick Paul Taylor Aug 7, 2026 9:10am BioMarin Pharmaceutical failed trials Failed drug program rare diseases BioMarin Pharmaceutical has ended development of BMN 401 in all indications months after reporting mixed phase 3 data on the rare disease prospect. In May, the biotech revealed that a late-phase study of the subcutaneous enzyme replacement therapy in ENPP1 deficiency missed one of its two co-primary endpoints. BioMarin opted against making a snap decision about the future of the drug candidate, choosing instead to take time to evaluate the data and determine next steps.

The company brought the ax down on the program after the stock market closed Thursday. As part of its second-quarter results, BioMarin disclosed that it has stopped development in all indications. While ENPP1 deficiency was the lead indication, Inozyme Pharma was studying the treatment in people with ABCC6 deficiency and end-stage kidney disease before being acquired by BioMarin.

Investors were braced for the termination of the program. In a June 2 note to investors, Guggenheim Securities analysts said that they believed “success of BMN 401 is highly unlikely in light of these data, and we are therefore removing it from our model.” Confirmation that BioMarin is pulling the plug completes BioMarin’s short, ill-fated attempt to expand into ENPP1 deficiency. The biotech completed its $270 million takeover of Inozyme in July 2025.

Less than one year later, BioMarin reported the mixed phase 3 data that signaled the beginning of the end of its interest in BMN 401. The molecule represented “substantially all” of the value of the acquired assets. BioMarin’s portfolio of commercial products could help the company recover from the setback.

Revenue rose 20% in the second quarter, encouraging the biotech’s executives to set intermediate- to long-term expectations that Guggenheim analysts said “should be viewed positively” in a note Friday. The company’s “ability to achieve these should be apparent within the next 12 to 18 months, allowing [management] to redeploy growth capital towards pipeline augmentation,” the analysts said. BioMarin Pharmaceutical failed trials Failed drug program rare diseases enzyme disorders Biotech