BioMarin drops ENPP1 deficiency drug BMN 401

BioMarin has discontinued development of BMN 401, an investigational enzyme replacement therapy for ENPP1 deficiency, after its pivotal Phase III ENERGY 3 trial failed to meet one of its two co-primary endpoints and showed no meaningful benefits on secondary endpoints such as rickets severity and growth measures. The company announced the decision in its Q2 2026 financial results on August 6, 2026, and has removed BMN 401 from its pipeline across all indications with no plans for further development, partnerships, or out-licensing.

Arcturus Therapeutics and CSL Seqirus have terminated their self-amplifying mRNA (saRNA) vaccine collaboration via a settlement agreement that returns global rights for KOSTAIVE (ARCT-154), the world's first approved sa-mRNA vaccine, and Arcturus's broader infectious‑disease portfolio (seasonal flu, pandemic flu, RSV, EBV) to Arcturus. CSL Seqirus will pay Arcturus a one‑time $12 million cash payment, Arcturus will be released from about $16 million in R&D credit liabilities, and existing Japan rights for the 2026–2027 season remain with Meiji Seika Pharma.