Blueprint Medicines (BPMC) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic priorities and growth outlook

• Focused on driving revenue growth and expanding patient reach with AYVAKIT in systemic mastocytosis (SM), while advancing next-generation KIT inhibitor elenestinib for future innovation.

• Establishing a new value pillar with BLU-808, a wild-type KIT inhibitor targeting allergy and inflammation diseases, with IND submission and healthy volunteer studies planned for the second half of the year.

• Maintaining financial discipline and a path to sustainability, leveraging AYVAKIT’s multibillion-dollar opportunity to support ongoing innovation.

• European expansion underway, with Germany showing strong initial results and other major markets targeted for growth.

• Total addressable population for SM estimated to be growing, with recent data suggesting prevalence may be higher than previously thought.

Commercial performance and market dynamics

• AYVAKIT launch in SM has exceeded expectations, with strong patient uptake, broad label coverage, and favorable benefit-risk profile supporting chronic use.

• Diagnosed SM patient population in the U.S. is growing at 20% year-over-year, with new prevalence estimates doubling previous figures.

• European launch, especially in Germany, mirrors U.S. success; Europe expected to contribute 10-15% of overall revenue this year.

• Access and reimbursement remain strong, with rapid time to fill and favorable dynamics due to younger ISM patient demographics and recent policy changes.

• Persistence and compliance trends are positive, with median therapy duration in advanced SM at 25 months and expectations for even longer persistence in ISM.

Pipeline and innovation strategy

• BLU-808 designed as a highly selective, oral wild-type KIT inhibitor for allergy and type 2 inflammation, with preclinical data supporting broad potential and flexible dosing.

• Healthy volunteer studies for BLU-808 to begin in the second half of the year, with Phase 1 data expected in 2025 as a major inflection point.

• Next-generation KIT inhibitor elenestinib aims to build on AYVAKIT’s success, with registration studies and strategic updates planned for the second half of the year.

• Oncology assets, including CDK2 inhibitor BLU-222, are being positioned for partnership, with recent ASCO data showing promising safety and efficacy in combination with CDK4/6 inhibitors.

• Business development remains strategic, with a focus on capital efficiency, leveraging AI-enabled drug discovery, and both sell-side and buy-side opportunities to support long-term growth.