BioMarin discontinued development of BMN401, its enzyme replacement therapy for ENPP1 deficiency, after mixed Phase 3 results that included missing one of two co-primary endpoints. The company said it ended development across all indications, closing a short-lived expansion following BioMarin’s acquisition of Inozyme Pharma for $270 million in 2025. Investors had been positioned for a pullback given the clinical readout, and BioMarin’s decision effectively removes a major portion of acquired asset value. BioMarin reported rising Q2 revenue as it works to reallocate growth capital toward pipeline augmentation, even as it exits the ENPP1 effort.
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