Blueprint Medicines Corporation provided its 2025 corporate outlook, significantly updating the estimated peak revenue opportunity for its systemic mastocytosis (SM) franchise to $4 billion. The company also stated its expectation to achieve $2 billion in annual AYVAKIT revenue by 2030, driven by the global launch and new epidemiology data suggesting a larger SM prevalence.
The company announced positive results from the Phase 1 healthy volunteer study of BLU-808, an oral wild-type KIT inhibitor. BLU-808 was well-tolerated across all tested doses, with all treatment-emergent adverse events in multiple-ascending dose cohorts being Grade 1. The study demonstrated dose-dependent pharmacokinetics, with a half-life of approximately 40 hours supporting once-daily dosing, and rapid, robust, and sustained reductions in serum tryptase exceeding 80% at the highest dose.
Based on these positive data, Blueprint Medicines is initiating proof-of-concept studies for BLU-808 in multiple allergic and inflammatory diseases, including chronic urticaria, allergic asthma, allergic rhinitis, allergic conjunctivitis, and mast cell activation syndrome. This strategic move aims to characterize BLU-808's broad therapeutic potential. Additionally, the company is deprioritizing further internal investment in its CDK2 inhibitor, BLU-222, and is seeking a strategic partnership to advance this program.
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