Blueprint Medicines Provides 2025 Outlook and Highlights Strategy for Continued Growth

BLU-808 Phase 1 Trial Results - BLU-808 demonstrated rapid, robust, and sustained reductions in serum tryptase, exceeding 80% in the Phase 1 healthy volunteer study [6] - Dose-dependent tryptase reductions were observed: 1 mg (-23%), 3 mg (-41%), 6 mg (-66%), and 12 mg (-87%) [2] - BLU-808 showed a wide therapeutic window and was well-tolerated at all doses tested, with no serious adverse events or discontinuations [7][16] Systemic Mastocytosis Franchise Opportunity - The company estimates the peak revenue opportunity for its systemic mastocytosis (SM) franchise at $4 billion, driven by AYVAKIT's strong launch and evolving SM prevalence estimates [6][15] - AYVAKIT revenue is expected to reach$2 billion by 2030 [6][15] - The company plans to achieve reimbursement for AYVAKYT in ≥20 countries by the end of 2025 [3] Pipeline and Strategic Updates - The company is advancing CDK2 and CDK4 targeted protein degraders, prioritizing further investment in its CDK franchise for breast cancer and other solid tumors [3] - The Phase 3 HARBOR trial of elenestinib, a next-generation KIT D816V inhibitor, has been initiated in patients with indolent systemic mastocytosis (ISM) [3] - Proof-of-concept trials for BLU-808 in chronic urticaria, allergic asthma, allergic rhinitis, allergic conjunctivitis, and mast cell activation syndrome are planned for 2025 [8][20] Financial and Corporate Goals - The company expects AYVAKIT product revenue for full-year 2024 to be $475 to$480 million, representing an increase of more than 130% over 2023 [12] - Corporate goals for 2025 include growing franchise leadership in SM and achieving clinical proof-of-concept for BLU-808 in allergic and inflammatory diseases [20] - The company aims to nominate two development candidates, including its first targeted protein degrader, in the second half of 2025 [17] J P Morgan Conference Presentation - Detailed data from the BLU-808 Phase 1 trial will be presented at the J P Morgan conference on January 13, 2025 [1][9] - The CEO will present a company overview and 2025 outlook at the conference, with a live webcast available on the company's website [9]