Blueprint Medicines Driving growth and innovation with operational excellence Fourth quarter and full-year 2024 financial results February 13, 2025 Fourth quarter and full-year 2024 financial results INTRODUCTION AYVAKIT PERFORMANCE Kate Haviland Chief Executive Officer Philina Lee, PhD Chief Commercial Officer Becker Hewes, MD Chief Medical Officer FINANCIAL RESULTS Mike Landsittel Chief Financial Officer 2 CLINICAL 3 2025 guidance progresses AYVAKIT toward $2 billion by 2030 $53 $111 $204 $479 2021 2022 2 ...

Financial Data and Key Metrics Changes - Blueprint Medicines achieved total revenues of $479 million in 2024, representing a robust year-over-year growth of 135% [7][52]. - The company expects AYVAKIT to generate revenue between $680 million and $710 million in 2025, indicating a 45% growth rate at the midpoint [8][38]. - Operating expenses were reduced in 2024 compared to 2023, contributing to a significant reduction in cash burn, which is expected to continue declining in 2025 [54][55]. Business Line Data and Key Metrics Changes - AYVAKIT's net product revenues reached $479 million in 2024, with fourth-quarter revenue of $144 million, comprising $124 million in the U.S. and $20 million internationally [25][52]. - The company has penetrated less than 10% of the diagnosed systemic mastocytosis (SM) patient pool, indicating substantial growth potential [10][72]. - The number of AYVAKIT prescribers has significantly increased, particularly among allergists, which is crucial for driving future growth [31][34]. Market Data and Key Metrics Changes - The diagnosed SM patient population in the U.S. is estimated to have increased from approximately 30,000 to 60,000, reflecting a twofold increase in prevalence [12][88]. - The international team has successfully obtained ISM pricing for AYVAKIT in Germany, with expectations for additional markets to come online throughout the year [28][111]. Company Strategy and Development Direction - The company aims to build a durable SM franchise with elenestinib and expand its commercial and medical field infrastructure to drive growth [16][21]. - Investments in education and market-building initiatives are planned for 2025 to support the growth of AYVAKIT and the broader SM franchise [15][35]. - The company is focused on leveraging its clinical development and commercial infrastructure to drive innovation while maintaining financial discipline [21][55]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving the $2 billion revenue target for AYVAKIT by 2030, citing a strong foundation and growth trajectory established in 2024 [8][40]. - The management highlighted the importance of early treatment and the long-term safety and efficacy data of AYVAKIT in motivating both providers and patients [42][43]. - The company anticipates continued growth in diagnosed SM patients and emphasizes the need for ongoing education and awareness among healthcare providers [11][90]. Other Important Information - The company is initiating proof-of-concept studies for BLU-808, which is positioned as a best-in-class oral KIT inhibitor for various mast cell-mediated allergic inflammatory diseases [20][47]. - The capital allocation strategy for 2025 is aligned with the company's growth plans, focusing on investments that drive near-term growth while solidifying its leadership in novel mast cell therapies [21][55]. Q&A Session Summary Question: How does the company plan to achieve the $2 billion revenue target by 2030? - Management believes the growth rate of 45% for AYVAKIT in 2025 is achievable, with a strong commercial foundation and increasing prescriber experience driving future growth [58][62]. Question: What is the safety profile of BLU-808 based on healthy volunteer data? - The healthy volunteer data indicates a wide therapeutic index, and various dosing strategies are being explored for different indications [66][68]. Question: How much of the expected growth in 2025 is driven by existing patients versus new patients? - The company sees significant potential for growth from both existing and new prescribers, with less than 10% of the diagnosed SM population currently on AYVAKIT [71][72]. Question: What are the assumptions built into the $2 billion guidance? - The guidance incorporates expectations for patient growth, market expansion, and the impact of potential competition, with a focus on increasing urgency to treat [85][90]. Question: How significant is the field team expansion for reaching more patients? - The field team expansion is expected to increase engagement with existing providers and broaden the reach to medical dermatologists and gastroenterologists, which will drive growth in the coming years [100][102].

-- Anticipate global AYVAKIT net product revenue of approximately $680 million to $710 million in 2025, representing a 45% percent year-over-year growth at the midpoint – -- Peak systemic mastocytosis franchise revenue opportunity updated to $4 billion, with AYVAKIT expected to achieve $2 billion in revenue by 2030 -- EXHIBIT 99.1 Blueprint Medicines Reports Fourth Quarter and Full Year 2024 Results -- Achieved $479.0 million in AYVAKIT® (avapritinib) global net product revenues in 2024, including $144.1 mi ...

Table of Contents _____________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 _____________________________ FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number 001-37359 _____________________________ BLUEPRINT MEDICINES CORPORATION (Exact Name of ...

Blueprint Medicines Corporation (NASDAQ:BPMC) Q3 2024 Earnings Conference Call October 30, 2024 8:00 AM ET Company Participants Jenna Cohen - Vice President-Investor Relations Kate Haviland - Chief Executive Officer Philina Lee - Chief Commercial Officer Christy Rossi - Chief Operating Officer Mike Landsittel - Chief Financial Officer Fouad Namouni - President-Research & Development Conference Call Participants Marc Frahm - TD Cowen Brad Canino - Stifel Reni Benjamin - Citizens JMP Paul Jeng - Guggenheim To ...

EXHIBIT 99.1 Blueprint Medicines Reports Third Quarter 2024 Results and Raises AYVAKIT®/AYVAKYT® (avapritinib) Full Year Revenue Guidance -- Achieved $128.2 million in AYVAKIT net product revenues in the third quarter 2024 -- -- Raising AYVAKIT net product revenue guidance to $475 million to $480 million for 2024 -- -- On track to initiate the registration-enabling HARBOR Part 2 study of elenestinib in ISM by year end -- CAMBRIDGE, Mass., October 30, 2024 – Blueprint Medicines Corporation (Nasdaq: BPMC) tod ...

Financial Data and Key Metrics - Total revenues for Q2 2024 were $138.2 million, including $114.1 million in net product revenues from AYVAKIT and $24 million in collaboration, license, and other revenues [22] - AYVAKIT achieved $114.1 million in net product revenue, with $101.5 million in the US and $12.7 million ex-US, representing a year-over-year growth of more than 185% [9] - The company raised its AYVAKIT product revenue guidance to $435 million to $450 million for 2024, reflecting strong performance in the first half of the year [22] Business Line Performance - AYVAKIT's growth in the US was driven by new patient starts, low discontinuation rates, high compliance, and favorable commercial versus free goods mix [9] - The international team had an exceptional quarter, with the ISM launch underway in Germany and plans to expand to additional countries in 2025 [12] - The company expects continued strength in its international business, which is seen as an important contributor to future growth [12] Market Performance - The US market saw strong growth in AYVAKIT revenue, driven by expanding prescriber base and low discontinuation rates [9] - In Germany, the ISM launch is progressing well, with growing prescriber bases in both academic and community settings [12] - The company anticipates further international expansion, particularly in Europe, where pricing and reimbursement negotiations are ongoing [58] Company Strategy and Industry Competition - The company is focused on building a franchise in mast cell disorders with AYVAKIT, elenestinib, and BLU-808, targeting a multibillion-dollar market opportunity [18] - BLU-808, a wild-type KIT inhibitor, has entered clinical trials, with initial data expected early next year, potentially impacting a range of mast cell diseases [7][19] - The company is also advancing its cell cycle inhibition strategy, with BLU-222 and targeted protein degradation platforms, aiming to address unmet medical needs in oncology [20] Management Commentary on Operating Environment and Future Outlook - Management expressed strong confidence in AYVAKIT's long-term revenue potential, citing its unique value proposition and positive reception among physicians, patients, and payers [5][6] - The company highlighted the importance of patient compliance and long-term therapy duration as key drivers of future revenue growth [10] - Management noted that the chronic burden of ISM is often underappreciated and emphasized the need to redefine treatment expectations for patients and providers [15] Other Important Information - The company has $868.5 million in cash on hand, providing financial flexibility to invest in innovation and long-term growth [24] - Research and development expenses and SG&A expenses are expected to remain relatively flat for the remainder of the year [24] - The company is focused on driving long-term shareholder value through the successful launch of AYVAKIT and disciplined expense management [24] Q&A Session Summary Question: Quarterly dynamics and impact of Part D redesign on AYVAKIT launch [26] - Management noted seasonal dynamics in patient starts, with potential delays during vacation and holiday periods, but emphasized that the opportunity remains strong [28][29] - The proportion of free goods has stabilized at just under 20%, with no significant changes expected for the rest of the year [30] Question: Eligibility for AYVAKIT and long-term patient eligibility trends [33] - Providers are broadening their view of who is an appropriate patient for AYVAKIT, with a trend towards treating patients with fewer but impactful symptoms [34][35] - The company believes the prevalence of ISM may be underestimated, potentially leading to a larger patient population than initially thought [37] Question: Growth rate and potential for AYVAKIT to exceed $2 billion peak revenue [39] - Management reiterated confidence in AYVAKIT's peak revenue potential, citing strong year-over-year growth and expanding patient eligibility [41][43] - The company is monitoring factors such as broadening patient eligibility and increasing diagnosed patient numbers to assess potential for higher peak revenue [44] Question: Treatment duration and refill rates for AYVAKIT [47] - Patients on AYVAKIT are trending towards long-term therapy, with low discontinuation rates and high compliance, consistent with a multiyear duration of therapy [47][48] Question: Prescriber base expansion and strategy for AYVAKIT [50] - The prescriber base for AYVAKIT is expanding beyond the initial target of 400 physicians, with increasing adoption across hematologists, oncologists, and allergists [50][51] Question: Discontinuation rates and financial sustainability of BLU-808 development [53] - Discontinuation rates for AYVAKIT are very low, with patients staying on therapy for long durations [54] - The development of BLU-808 is factored into the company's financial guidance, with initial data expected to be a significant inflection point [56][57] Question: International expansion plans for AYVAKIT [58] - The company is focused on expanding AYVAKIT's presence in international markets, with ISM launches expected in additional countries in 2025 [58] Question: Spectrum of disease severity and patient eligibility for AYVAKIT [60] - The company is seeing a broadening of patient eligibility for AYVAKIT, with providers increasingly treating patients with less severe symptoms [63][64] Question: Biomarkers and metrics for BLU-808 healthy volunteer study [62] - The healthy volunteer study for BLU-808 will focus on safety, pharmacokinetics, and pharmacodynamics, with data expected to inform the breadth of its application [66] Question: Net pricing trends and revenue growth drivers for AYVAKIT [69] - There have been no quarter-over-quarter net pricing increases for AYVAKIT, with revenue growth driven by volume rather than price [70] Question: Prescriber trends in academic vs. community centers [72] - Prescribing trends for AYVAKIT are strong across both academic and community settings, with deepening adoption among experienced prescribers [73] Question: Differentiation of elenestinib and timeline for pivotal study [75] - Elenestinib, a next-generation KIT D816V inhibitor, is being developed with a focus on clinical differentiation in ISM, with Part 2 of the HARBOR study expected to begin by year-end [76] Question: Free drug dynamics and dose escalation trends for AYVAKIT [78] - The proportion of free goods has stabilized, with no significant changes expected for the rest of the year [81] - The majority of ISM patients start at 25 mg, with limited utilization of higher doses [82] Question: Business development strategy and challenges in HARBOR study enrollment [85] - The company is open to both inbound and outbound business development opportunities, with a focus on advancing its mast cell disorder franchise [86][87] - No major challenges are expected in enrolling patients for the HARBOR study, with international sites planned to support recruitment [89]

Table of Contents _____________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 _____________________________ FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number 001-37359 _____________________________ BLUEPRINT MEDICINES CORPORATION (Exact Name of Regi ...

Second Quarter 2024 Financial Results AUG 1, 2024 Agenda INTRODUCTION AYVAKIT PERFORMANCE Kate Haviland Chief Executive Officer Philina Lee, PhD Chief Commercial Officer CORPORATE PROGRESS Christy Rossi Chief Operating Officer | --- | --- | --- | |-------|-------------------------|-------| | | | | | | | | | | | | | | Q2 2024 FINANCIAL | | | | PERFORMANCE | | | | | | | | Mike Landsittel | | | | Chief Financial Officer | | Not for promotional use 2 Forward-looking statements 3 This presentation contains forwa ...

Exhibit 99.1 Blueprint Medicines Reports Second Quarter 2024 Results and Raises AYVAKIT/AYVAKYT (avapritinib) Full Year Revenue Guidance -- Achieved $114.1 million in AYVAKIT net product revenues in the second quarter 2024 -- -- Raising AYVAKIT net product revenue guidance to $435 million to $450 million for 2024 -- -- Initiated Phase 1 healthy volunteer trial of wild-type KIT inhibitor BLU-808 -- CAMBRIDGE, Mass., August 1, 2024 – Blueprint Medicines Corporation (Nasdaq: BPMC) today reported financial resu ...