Blueprint Medicines Shares Surge 10% on $4B Mastocytosis Franchise Revenue Projection

Blueprint Medicines Corporation (BPMC) shares rose by 10% on Monday, January 13, following the company's announcement of a $4 billion peak revenue opportunity for its systemic mastocytosis (SM) franchise, catalyzed by the strong launch of AYVAKIT® (avapritinib) and evolving prevalence estimates. The company also expects to achieve $2 billion in AYVAKIT revenue by 2030.



The company's chief executive officer, Kate Haviland, presented at the J.P. Morgan conference on Monday, outlining the company's strategy for continued growth and its commitment to targeting the mast cell to fundamentally shift the treatment of allergic and inflammatory diseases. Blueprint Medicines' proven R&D and commercial capabilities have positioned the company to deliver significant near- and long-term value to both patients and shareholders.



Systemic Mastocytosis (SM) presents a large and growing opportunity for Blueprint Medicines. The company now estimates the peak revenue opportunity for its SM franchise at $4 billion, with $2 billion in annual revenues expected to be achieved by AYVAKIT by 2030. This projection is based on the strength of the global AYVAKIT launch, significant sustained growth in diagnosed SM patients, and new epidemiology data suggesting a greater prevalence of SM than previously thought.

Blueprint Medicines plans to report financial results for the fourth quarter and full-year 2024 in February 2025. The company previously provided guidance in October 2024 for AYVAKIT product revenue of $475 to $480 million for full-year 2024, representing an increase of more than 130 percent over 2023.



Positive data from the Phase 1 trial of BLU-808, a highly potent and selective oral wild type KIT inhibitor, further supports the company's growth prospects. BLU-808 demonstrated a wide therapeutic window with rapid, robust, and sustained tryptase reductions exceeding 80% in a healthy volunteer study. The drug was well-tolerated at all doses tested, with no serious adverse events or discontinuations due to adverse events. BLU-808 showed consistent, dose-dependent increases in drug exposure and sustained target coverage, with mean plasma concentrations exceeding predicted KIT IC50 levels at ≥1 mg QD and IC90 levels at ≥3 mg QD.

Based on these positive data, Blueprint Medicines is initiating proof-of-concept studies in chronic urticaria, allergic asthma, allergic rhinitis, allergic conjunctivitis, and mast cell activation syndrome to characterize BLU-808's broad therapeutic potential across multiple diseases where mast cells play a core role.

While Blueprint Medicines' ambitious revenue projections and positive clinical data are encouraging, investors should be aware of potential risks and challenges. Market acceptance and regulatory approvals for AYVAKIT and other drug candidates, achieving projected revenue targets and clinical trial timelines, and uncertainties in epidemiology data could impact the company's growth prospects. Additionally, competition in the biopharmaceutical industry, economic and market conditions, and operational challenges could pose further risks to the company's financial performance.

In conclusion, Blueprint Medicines' $4 billion peak revenue projection for its systemic mastocytosis franchise and positive data from the BLU-808 trial have driven a 10% increase in the company's shares. As the company continues to leverage its proven R&D and commercial capabilities, investors can expect significant near- and long-term value from Blueprint Medicines' mast cell-targeted therapies. However, potential risks and challenges should be carefully considered when evaluating the company's investment potential.