BioMarin ended development of BMN 401 in all indications after the biotech reported mixed Phase 3 results and ultimately stopped the program across the board. The company previously described the therapy as a subcutaneous enzyme replacement approach intended for ENPP1 deficiency, with additional development efforts tied to other indications through acquired assets. BioMarin confirmed that it took the decision after assessing the data, following a prior mixed outcome on two co-primary endpoints in the late-stage study. The program’s valuation was central to BioMarin’s 2025 acquisition of Inozyme Pharma for $270 million, which the company later characterized as largely comprised of the acquired BMN 401 value. In its latest quarterly update, BioMarin also highlighted revenue growth of 20% in the second quarter, framing the halt as an action that could free capital for pipeline augmentation over the next 12 to 18 months.
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