Indolent systemic mastocytosis

-- Achieved 61% year-over-year growth with $149.4 million in AYVAKIT net product revenues in the first quarter 2025 -- -- Raising AYVAKIT net product revenue guidance to $700 - $720 million for 2025 -- -- Initiated BLU-808 proof of concept studies in allergic rhinoconjunctivitis and chronic urticaria – CAMBRIDGE, Mass. , May 1, 2025 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today reported financial results, provided a business update for the first quarter ended March 31, 2025 , and provided corporate updates. " Blueprint Medicines strives to be a top-tier standout in biotech, with a core focus on innovation, commercial excellence, and a disciplined approach to global investment across our portfolio. Following strong performance in 2024, we have continued our executional momentum in 2025," said Kate Haviland , Chief Executive Officer of Blueprint Medicines . "AYVAKIT is well on its way to meeting our goal of $2 billion in revenue by 2030, as we continue to capture the substantial and growing multi-billion-dollar systemic mastocytosis opportunity that we anticipate will drive topline revenue growth into the next decade. We also advanced our prioritized pipeline programs, achieving significant portfolio milestones, including the initiation of two proof-of-concept studies for wild-type KIT inhibitor BLU-808 and advancing the HARBOR study of elenestinib in indolent systemic mastocytosis. The combination of our durable and growing commercial revenue, our strong cash position, and our disciplined capital allocation strategy enables us to focus on executing our business to plan and insulates us from broader market volatility." First Quarter 2025 Highlights and Recent Progress Achieved AYVAKIT net product revenues of $149.4 million for the first quarter of 2025, including $129.4 million in the US and $20 million ex-US, representing 61% percent growth year-over-year. Initiated two clinical proof-of-concept studies of BLU-808, a highly selective and potent investigational oral wild-type KIT inhibitor for the treatment of mast cell disorders, including: A randomized, double-blind, placebo-controlled Phase 2a challenge study of BLU-808 in patients with allergic rhinoconjunctivitis; and A Phase 2a proof-of-concept study in chronic urticaria. This study comprises an open-label portion in chronic inducible urticaria and a randomized, double-blind, placebo-controlled portion in chronic spontaneous urticaria. Presented 12 poster and two oral data presentations at the American Academy of Allergy , Asthma & Immunology (AAAAI)/ World Allergy Organization (WAO) conference. The breadth of data included three-year long-term follow-up data from the PIONEER study of AYVAKIT in patients with indolent systemic mastocytosis (ISM), data showing AYVAKIT-treated patients with ISM achieved improvements in bone health, and data from the healthy volunteer study of BLU-808. Read the presentations here. Strengthened cash balance with $78.7 million in connection with the sale of the company's equity investment in IDRx, Inc. following its acquisition by GSK plc. Hosting scientific seminar on mast cell activation syndrome (MCAS) with expert physician Dr. Matt Giannetti on Wednesday, June 4, 2025 at 1:00 p.m. A randomized, double-blind, placebo-controlled Phase 2a challenge study of BLU-808 in patients with allergic rhinoconjunctivitis; and A Phase 2a proof-of-concept study in chronic urticaria. This study comprises an open-label portion in chronic inducible urticaria and a randomized, double-blind, placebo-controlled portion in chronic spontaneous urticaria. 2025 Financial Guidance Blueprint Medicines is raising guidance and now anticipates approximately $700 million to $720 million in global AYVAKIT net product revenues in 2025, on the path to achieving $2 billion in global AYVAKIT net product revenues by 2030. This guidance increase reflects favorability observed in the free versus commercial mix of AYVAKIT sales in the first quarter and continued strength in underlying fundamentals of growth. Blueprint continues to expect a year-over-year reduction in cash burn in 2025, as it continues to invest in advancing its prioritized programs, balancing investments in innovation with financial discipline. Blueprint continues to anticipate that its existing cash, cash equivalents and investments, together with anticipated product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile. Key Upcoming Milestones The company plans to achieve the following remaining milestones in 2025: Mast cell disorders Deliver continued strong and steady AYVAKIT revenue growth. Achieve reimbursement of AYVAKYT in ≥ 20 countries overall. Activate sites and drive enrollment in HARBOR trial of elenestinib. Initiate proof of concept studies of BLU-808 in allergic asthma and MCAS. Discovery Nominate two development candidates, including the company's first protein degrader. First Quarter 2025 Results Revenues: Revenues were $149.4 million for the first quarter of 2025, generated by net product sales of AYVAKIT/AYVAKYT. Revenues were $96.1 million in the first quarter of 2024, including $92.5 million of net product revenues from sales of AYVAKIT/AYVAKYT and $3.6 million in collaboration revenues. Cost of Sales: Cost of sales was $2.8 million for the first quarter of 2025, as compared to $3.2 million for the first quarter of 2024. The decrease was primarily due to lower sales to our collaboration partner offset by an increase in product sales volume. R&D Expenses: Research and development expenses were $91.9 million for the first quarter of 2025, as compared to $88.2 million for the first quarter of 2024. This increase was primarily due to the increased investment in our priority programs to advance the associated clinical trials. Research and development expenses included $12.1 million in stock-based compensation expenses for the first quarter of 2025. SG&A Expenses: Selling, general and administrative expenses were $95.8 million for the first quarter of 2025, as compared to $83.6 million for the first quarter of 2024. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $16.9 million in stock-based compensation expenses for the first quarter of 2025. Net Income: Net income was $0.5 million for the first quarter of 2025, as compared to a net income of $89.1 million for the first quarter of 2024. The net income for the first quarter of 2025 was primarily driven by a one-time net gain of $50.0 million recorded in connection with the sale of the company's equity investment in IDRx, Inc. following its acquisition by GSK plc. The net income for the first quarter of 2024 was primarily driven by a one-time non-cash debt extinguishment gain of $173.7 million recorded in connection with the Royalty Pharma termination agreement. Cash Position: As of March 31, 2025 , cash, cash equivalents and investments were $899.8 million , as compared to $863.9 million as of December 31, 2024 . Conference Call Information Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss first quarter 2025 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 404-975-4839 (international) and referring conference ID 082088. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at http://ir.blueprintmedicines.com/. The archived webcast will be available on Blueprint Medicines' website approximately two hours after the conference call and will be available for 30 days following the call. Upcoming Investor Conferences Blueprint Medicines will participate in two upcoming investor conferences: Citizens JMP Life Science Conference on Wednesday, May 7, 2025 at 12:00 p.m. ET . Goldman Sachs 46th Annual Global Healthcare Conference on Wednesday, June 11, 2025 at 10:40 a.m. ET . Scientific Webinar Series Blueprint Medicines will host the third event in its scientific seminar series, focused on mast cell activation syndrome (MCAS), on Wednesday, June 4, 2025 at 1:00 p.m. ET . A live webcast of the above presentations and any related slides will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at http://ir.blueprintmedicines.com. A replay of the webcasts will be archived on the Blueprint Medicines website following the events. About Blueprint Medicines Blueprint Medicines is a global, fully integrated biopharmaceutical company that invents life-changing medicines. We seek to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. Our approach begins by targeting the root causes of disease, using deep scientific knowledge in our core focus areas and drug discovery expertise across multiple therapeutic modalities. We have a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib) which we are bringing to patients with systemic mastocytosis (SM) in the U.S. and Europe . Leveraging our established research, development, and commercial capability and infrastructure, we now aim to significantly scale our impact by advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases including SM and chronic urticaria, breast cancer and other solid tumors. For more information, visit www.BlueprintMedicines.com and follow us on X (formerly Twitter; @BlueprintMeds) and LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding plans, strategies, timelines and expectations for Blueprint Medicines' operations, including its expectations regarding growth and net product revenue in 2025 and its 2030 net product revenue goal; its goals related to global reimbursement of AYVAKIT/ AYVAKYT; statements regarding its current or future approved drugs and drug candidates and operations, including plans to drive enrollment of HARBOR trial of elenestinib and initiate proof of concept studies of BLU-808 in allergic asthma and MCAS; expectations related to the markets for current or future approved drugs and drug candidates; its planned milestones for 2025; the potential benefits of any of its current or future approved drugs or drug candidates in treating patients; statements related to liquidity and capital position, including expectations that its cash, cash equivalents and investments together with anticipated product revenues, will provide sufficient capital to enable it to reach a self-sustainable financial profile; its statements regarding broader market volatility; and its financial performance, strategy, goals and anticipated milestones, business plans and focus, including expectations regarding its reduction in cash burn. The words "aim," "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "opportunity," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation: the risk that the marketing and sale of AYVAKIT/ AYVAKYT or any future approved drugs may be unsuccessful or less successful than anticipated, or that AYVAKIT/ AYVAKYT may not gain market acceptance by physicians, patients, third-party payors and others in the medical community; the risk that the market opportunities for AYVAKIT/ AYVAKYT or Blueprint Medicines' drug candidates are smaller than we estimate or that any approval we obtain may be based on a narrower definition of the patient population that we anticipate; the risk of delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; risks related to Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; preclinical and clinical results for Blueprint Medicines' drug candidates may not support further development of such drug candidates either as monotherapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions; the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and patient enrollment rates may be delayed or slower than anticipated; actions of regulatory agencies may affect its approved drugs or its current or future drug candidates, including affecting the initiation, timing and progress of clinical trials, as well as the pricing of its drug candidates; risks related to Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for its products and current or future drug candidates it is developing; the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing and other arrangements; risks related to its liquidity and financial position, including the risk that Blueprint Medicines may be unable to generate sufficient future product revenues to achieve and maintain a self-sustainable financial profile; and the accuracy of its estimates of revenues, expenses and capital requirements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements. Trademarks Blueprint Medicines , AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation. Blueprint Medicines Corporation Selected Condensed Consolidated Balance Sheet Data (in thousands) (unaudited) March 31 , December 31 , 2025 2024 Cash, cash equivalents and investments $ 899,784 863,937 Working capital (1) 451,485 481,882 Total assets 1,195,604 1,179,813 Deferred revenue (2) 10,316 10,198 Liability related to the sale of future royalties and revenues (2) 246,632 255,174 Term loan (2) 387,746 386,970 Total liabilities 853,473 881,148 Total stockholders' equity 342,131 298,665 (1) Blueprint Medicines defines working capital as current assets less current liabilities. (2) Includes both current and long-term portions of the balance. Blueprint Medicines Corporation Condensed Consolidated Statements of Operations Data (in thousands, except per share data) (unaudited) Three Months Ended March 31 , 2025 2024 Revenues: Product revenue, net $ 149,413 $ 92,525 Collaboration, license, and other revenue - 3,591 Total revenues 149,413 96,116 Cost and operating expenses: Cost of sales 2,802 3,191 Research and development 91,890 88,191 Selling, general and administrative 95,807 83,557 Total cost and operating expenses 190,499 174,939 Other income (expense): Interest expense, net (8,129) (5,895) Other income, net 461 376 Equity investment gain 50,039 - Debt extinguishment gain - 173,658 Total other income, net 42,371 168,139 Income before income taxes 1,285 89,316 Income tax expense 789 180 Net income $ 496 $ 89,136 Net income per share — basic $ 0.01 $ 1.45 Net income per share — diluted $ 0.01 $ 1.40 Weighted-average number of common shares used in net income per share — basic 64,096 61,580 Weighted-average number of common shares used in net income per share —diluted 66,526 63,802 View original content to download multimedia:https://www.prnewswire.com/news-releases/blueprint-medicines-reports-first-quarter-2025-results-and-raises-ayvakitayvakyt-avapritinib-full-year-revenue-guidance-302443541.html SOURCE Blueprint Medicines Corporation Media Contact: Andrew Law, 617-844-8205, media@blueprintmedicines.com, Investor Contact: Jenna Cohen, 857-209-3147, ir@blueprintmedicines.com

-- Updated data show AYVAKIT® (avapritinib) was safe and effective for long-term treatment of ISM, with sustained symptom and quality-of-life benefit and no new safety signals with multiple years of therapy -- -- AYVAKIT-treated patients achieved improvements in bone health based on an analysis of bone mineral density scans, underscoring the importance of treating patients early in the ISM disease course -- -- BLU-808 healthy volunteer trial results demonstrate wide therapeutic window for modulating mast cell activity, affirming potential as best- and first-in-class oral wild-type KIT inhibitor -- CAMBRIDGE, Mass., Feb. 28, 2025 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced data presentations reflecting its ongoing efforts to innovate the treatment of mast cell-driven diseases. The presentations include three-year median follow-up data from the registrational PIONEER trial demonstrating the strong long-term efficacy and safety of AYVAKIT® (avapritinib) in patients with indolent systemic mastocytosis (ISM), and real-world evidence further characterizing the substantial burden of the disease, highlighting the urgency to treat. In addition, the company will report positive data from the Phase 1 healthy volunteer trial evaluating BLU-808, an investigational, highly potent and selective oral inhibitor of wild-type KIT, consistent with top-line data previously announced in January 2025. A total of 14 data presentations, including two oral presentations, will be reported at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress being held February 28-March 3 in San Diego. "We are incredibly proud to showcase our mast cell therapy portfolio at AAAAI / WAO, with 14 presentations highlighting more than a decade of leadership and collaboration with the mast cell disease community, including renowned medical institutions pioneering innovative research and multi-disciplinary patient care," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "In ISM, new data have reinforced that AYVAKIT was very well-tolerated after multiple years of treatment, with a low discontinuation rate and safety results that have remained consistent with the safety profile from the placebo-controlled study as summarized in the FDA-approval label. This updated AYVAKIT data is particularly important for clinicians considering long-term treatment options for their ISM patients. Further, we are building on our foundational expertise in ISM with our oral wild-type KIT inhibitor BLU-808, with healthy volunteer data showing a differentiated clinical profile and wide therapeutic index that supports broad development across a range of mast cell diseases. Based on these data, we are initiating multiple proof-of-concept trials, with initial data expected later this year." AYVAKIT: PIONEER Three-Year Efficacy and Safety Data in ISM AYVAKIT 25 mg once daily (QD) was well-tolerated and no new safety signals were identified with a median of 3 years of exposure, with some patients out to five years of treatment. The most common treatment-related adverse events (AEs; ≥5 percent) were low-grade peripheral edema, periorbital edema, headache and nausea. AYVAKIT 25 mg QD led to robust and durable improvements in overall symptoms, individual symptom domains (skin, gastrointestinal, neurocognitive) and quality of life through 144 weeks of treatment. In patients who had high disease burden and escalated to AYVAKIT 50 mg QD (median follow-up: 10.6 months since escalation), treatment was well-tolerated, with side effects consistent with those reported at AYVAKIT 25 mg QD and no patients discontinuing due to AEs. Nearly all patients (93 percent) had improved or stable benefits in overall symptoms. Low Bone Density: Positive Clinical Impact of AYVAKIT and Real-World Prevalence in ISM In a single-site case series from the PIONEER trial, AYVAKIT showed clinically meaningful and durable improvements in bone density, including in the lumbar spine, femoral neck and total proximal femur. In real-world data generated from Mayo Clinic electronic health records, 67 percent of ISM patients had osteoporosis/osteopenia compared to 34 percent in a matched control cohort (p<0.0001). AYVAKIT: Clinical Benefits in Patients with Undetected KIT D816V by ddPCR Assay In the PIONEER trial, 15 percent of patients with ISM did not have KIT D816V mutations detected by the commercially available Droplet Digital Polymerase Chain Reaction (ddPCR) assay. Within this group, most patients had KIT mutations identified by the more sensitive duplex sequencing method, and these patients achieved similar reductions in serum tryptase and improvements in symptoms as those with detectable KIT mutations by the ddPCR assay. BLU-808: Positive Healthy Volunteer Data In the Phase 1 single-ascending dose (SAD) and multiple-ascending dose (MAD, 14-day dosing) trial, BLU-808 was well-tolerated at all doses tested. There were no serious AEs, no discontinuations or dose modifications due to AEs, and no significant changes in laboratory measures. BLU-808 achieved dose-dependent serum tryptase reductions exceeding 80 percent, reflecting evidence of mast cell target engagement. BLU-808 had a half-life supporting once-daily oral dosing and showed sustained target coverage across a range of doses. Blueprint Medicines' AAAAI / WAO data presentations are listed below. Copies of the data presentations will be available in the "Science—Publications and Presentations" section of the company's website at . Oral Presentation: Assessing Real-World Natural History of Indolent Systemic Mastocytosis: Retrospective Matched Cohort Study from Mayo Clinic Electronic Health Records (Abstract 955 – Monday, March 3) Oral Presentation: Responses to Avapritinib in Patients without Detectable KIT Mutations by ddPCR in Peripheral Blood Highlight Diagnostic Challenges and Opportunities in Indolent Systemic Mastocytosis (Abstract 965 – Monday, March 3) Poster Presentation: Patient Reported Burden of Indolent Systemic Mastocytosis in White vs. Non-White Patients (Abstract 220 – Friday, February 28) Poster Presentation: Identification of Clonal Mast Cell Disease in Patients with Anaphylaxis or Evidence of Systemic Mast Cell Activation: A Post Hoc Analysis from PROSPECTOR (Abstract 301 – Saturday, March 1) Poster Presentation: Avapritinib Impacts the Plasma Inflammatory Proteome in Patients with Indolent Systemic Mastocytosis (Abstract 517 – Saturday, March 1) Poster Presentation: Multiple Proteins Correlate with Tryptase Levels in Patients with Indolent Systemic Mastocytosis (ISM): Preliminary Results of Plasma Proteomic Analysis in PIONEER (Abstract 518 – Saturday, March 1) Poster Presentation: Comprehensive Analysis of Immunoglobulin E Levels in Healthy Donors and Patients with Indolent Systemic Mastocytosis Enrolled on the PIONEER Trial of Avapritinib (Abstract 521 – Saturday, March 1) Poster Presentation: Avapritinib and Bone Health in Indolent Systemic Mastocytosis: Learnings from the PIONEER Trial (Abstract 527– Saturday, March 1) Poster Presentation: PREDICT-SM: Development of Machine Learning Models to Support Screening for Undiagnosed Systemic Mastocytosis (Abstract 530 – Saturday, March 1) Poster Presentation (Trial in Progress): HARBOR: An Ongoing Phase 2/3 Study of Elenestinib in Patients with Indolent Systemic Mastocytosis (Abstract 533 – Saturday, March 1) Poster Presentation: Long-Term Quality of Life and Safety in Patients with Indolent Systemic Mastocytosis Treated with Avapritinib in the PIONEER Study (Abstract 534 – Saturday, March 1) Poster Presentation: BLU-808: A Potent and Selective Oral Small Molecule Wild-Type KIT Tyrosine Kinase Inhibitor for Allergic Conditions (Abstract 535 – Saturday, March 1) Poster Presentation: Utility of Large Language Models to Quantify Diagnostic Delays in Systemic Mastocytosis: A Multi-Center Real World Study (Abstract 544 – Saturday, March 1) Poster Presentation: Safety and Pharmacokinetics (PK) of BLU-808 Following Oral Dosing in Healthy Volunteers (Abstract 698 – Sunday, March 2) About Systemic Mastocytosis Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation in about 95 percent of cases. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms across multiple organ systems. The vast majority of those affected have indolent systemic mastocytosis (ISM). A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with ISM despite treatment with multiple symptom-directed therapies. This burden of disease can lead to a profound, negative impact on quality of life. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Until 2023, there were no approved therapies for the treatment of ISM. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival. About AYVAKIT (avapritinib) AYVAKIT (avapritinib) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adults with ISM, adults with advanced SM, including ASM, SM-AHN and MCL, and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Under the brand name AYVAKYT® (avapritinib), this medicine is approved by the European Commission for the treatment of adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, adults with ASM, SM-AHN and MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. The therapy is not recommended for the treatment of patients with low platelet counts (less than 50,000/µL). Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT. Important Safety Information Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC; <1% were Grade 3. Depending on the severity, withhold AYVAKIT and then resume at the same dose, or permanently discontinue AYVAKIT. Photosensitivity — AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment. Embryo-Fetal Toxicity — AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective contraception during treatment with AYVAKIT and for 6 weeks after the final dose. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose. Adverse Reactions — The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing. Drug Interactions — Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors or inducers. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary. To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or visit . AYVAKIT is available in 25-mg, 50-mg, 100-mg and 200-mg tablets. Please click here to see the full Prescribing Information for AYVAKIT. About Blueprint Medicines Blueprint Medicines is a global, fully integrated biopharmaceutical company that invents life-changing medicines. We seek to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. Our approach begins by targeting the root causes of disease, using deep scientific knowledge in our core focus areas and drug discovery expertise across multiple therapeutic modalities. We have a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib) which we are bringing to patients with systemic mastocytosis (SM) in the U.S. and Europe. Leveraging our established research, development, and commercial capability and infrastructure, we now aim to significantly scale our impact by advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases including SM and chronic urticaria, breast cancer and other solid tumors. For more information, visit and follow us on X (formerly Twitter; @BlueprintMeds) and LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding expectations for the potential benefits of AYVAKIT in treating patients with ISM; Blueprint Medicines' ability to transform treatment across the spectrum of SM; the development and potential benefits of BLU-808; statements regarding plans and expectations for Blueprint Medicines' current or future approved drugs and drug candidates; the potential benefits of any of Blueprint Medicines' current or future approved drugs or drug candidates in treating patients; and Blueprint Medicines' strategy, goals, business plans and focus. The words "aim," "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to Blueprint Medicines' ability and plans in continuing to build out and expand a commercial infrastructure, and successfully launching, marketing and selling current or future approved products; Blueprint Medicines' ability to successfully expand the approved indications for AYVAKIT/AYVAKYT or obtain marketing approval for AYVAKIT/AYVAKYT in additional geographies in the future; the delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the preclinical and clinical results for Blueprint Medicines' drug candidates, which may not support further development of such drug candidates either as monotherapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions; the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and patient enrollment rates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for AYVAKIT/AYVAKYT or any drug candidates it is developing; Blueprint Medicines' ability to successfully expand its operations, research platform and portfolio of therapeutic candidates, and the timing and costs thereof; and the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing arrangements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements. Trademarks Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation. 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-- ASH data reinforce survival benefits of front-line AYVAKIT® (avapritinib) use in patients with advanced SM -- -- Bone density analyses reported in patients with advanced SM underscore disease-modifying effects of AYVAKIT -- CAMBRIDGE, Mass., Dec. 7, 2024 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced data presentations that continue to demonstrate the long-term clinical benefits of AYVAKIT® (avapritinib) in advanced systemic mastocytosis (advanced SM), and reflect the company's ongoing partnership with the SM community to redefine the future of patient care – from improving diagnostic rates to raising the bar on treatment outcomes. The datasets, which include one oral presentation and three poster presentations, will be reported at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 7-10 in San Diego. "Systemic mastocytosis is associated with mast cell infiltration across multiple organ systems, chronic inflammation caused by immune dysregulation, unpredictable symptoms that may worsen over time, and serious comorbidities such as osteoporosis, highlighting the urgency to treat patients early in the course of their disease," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "AYVAKIT has fundamentally changed the treatment paradigm by targeting the disease at its source, showing prolonged survival outcomes for patients with advanced SM, as well as durable symptom control and quality-of-life improvements for patients with ISM. Based on the substantial datasets we have amassed over more than a decade, and our continued collaboration with the SM community, we have designed the HARBOR trial of elenestinib – a next-generation KIT D816V inhibitor – to rigorously assess a broad range of endpoints reflecting disease-modifying impact." Data reported at ASH highlight the survival benefits of front-line AYVAKIT use in patients with advanced SM, and support Blueprint Medicines' plans to evaluate novel clinical measures indicative of disease modification – such as bone density and biomarkers of chronic inflammation – in the registrational HARBOR trial. Key results include: In pooled analyses from the PATHFINDER and EXPLORER trials, treatment-naïve patients with advanced SM showed significant survival benefits with AYVAKIT, when indirectly compared to real-world outcomes for midostaurin. In the PATHFINDER trial, AYVAKIT led to sustained improvements in bone density for advanced SM patients who had low bone mass at baseline. In the PIONEER trial, patients with indolent SM (ISM) had significant baseline levels of immune dysregulation relative to healthy donors, reflecting the chronic inflammatory burden of the disease. Non-Invasive, Blood-Based Assay Identified KIT Mutations Not Previously Detectable by Existing Tests ASH data show that ultra-sensitive KIT testing in the peripheral blood – a novel tool in clinical development that is more sensitive than current commercially available methods – identified previously undetected KIT mutations in a number of PIONEER trial patients. Emerging clinical research with ultra-sensitive KIT assays suggest SM may be more prevalent than previously thought. An additional data presentation highlights the application of machine learning techniques to analyze baseline, blood-based parameters of Blueprint Medicines' clinical trial participants. Following these analyses, a predictive model was developed to distinguish between advanced SM and ISM, and its accuracy was validated by an independent dataset from Dana-Farber Cancer Institute. Collectively, these data build on Blueprint Medicines' collaborative efforts with clinical experts to improve and accelerate how SM is diagnosed and treated. Data presentations will be made available in the "Science―Publications and Presentations" section of Blueprint Medicines' website. Oral Presentation: Analysis of Avapritinib Clinical Trial Data Generates a Highly Accurate Predictive Model for Advanced Systemic Mastocytosis Versus Indolent Systemic Mastocytosis Based on Peripheral Blood Testing (Abstract 107 – Saturday, December 7) Poster Presentation: Overall Survival and Duration of Treatment in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib Versus Midostaurin or Best Available Therapy in a Real-World Setting (Abstract 1801 – Saturday, December 7) Poster Presentation: Ultra-Sensitive KIT Testing Uncovers Previously Undetected KIT Mutations in Patients with Indolent Systemic Mastocytosis: Results from the PIONEER Trial (Abstract 3164 – Sunday, December 8) Poster Presentation: Disease-Modifying Effects of Avapritinib in Patients with Advanced Systemic Mastocytosis: Improvements in Bone Density (Abstract 4544 – Monday, December 9) Publication-Only Abstract: Blood-Based Proteomics for Deeper Insights into Indolent Systemic Mastocytosis: the PIONEER Trial Experience (Abstract 6569) About Systemic Mastocytosis Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation in about 95 percent of cases. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms across multiple organ systems. The vast majority of those affected have indolent systemic mastocytosis (ISM). A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with ISM despite treatment with multiple symptom-directed therapies. This burden of disease can lead to a profound, negative impact on quality of life. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Until 2023, there were no approved therapies for the treatment of ISM. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival. About AYVAKIT (avapritinib) AYVAKIT (avapritinib) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adults with ISM, adults with advanced SM, including ASM, SM-AHN and MCL, and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Under the brand name AYVAKYT® (avapritinib), this medicine is approved by the European Commission for the treatment of adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, adults with ASM, SM-AHN and MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. The therapy is not recommended for the treatment of patients with low platelet counts (less than 50,000/µL). Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT. IMPORTANT SAFETY INFORMATION Intracranial Hemorrhage — Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT in clinical trials. In Advanced SM patients who received AYVAKIT at 200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts ≥50 x 109/L prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. In ISM patients, no events of ICH occurred in the 246 patients who received any dose of AYVAKIT in the PIONEER study. Monitor patients closely for risk factors of ICH which may include history of vascular aneurysm, ICH or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia. Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status. Advise patients to seek immediate medical attention for signs or symptoms of ICH. Permanently discontinue AYVAKIT if ICH of any grade occurs. In Advanced SM patients, a platelet count must be performed prior to initiating therapy. AYVAKIT is not recommended in Advanced SM patients with platelet counts <50 x 109/L. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of <50 x 109/L by treatment interruption or dose reduction. Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 33% of 995 patients overall in patients who received AYVAKIT in clinical trials including: 28% of 148 Advanced SM patients (3% were Grade ≥3), and 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC (<1% were Grade 3). Depending on the severity and indication, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue. Photosensitivity — AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment. Embryo-Fetal Toxicity — AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective method of contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks after the final dose. Adverse Reactions — The most common adverse reactions (≥20%) in patients with Advanced SM were edema, diarrhea, nausea, and fatigue/asthenia. The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing. Drug Interactions — Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided in patients with Advanced SM, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong or moderate CYP3A inducers. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary. To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or . AYVAKIT is available in 25-mg, 50-mg, 100-mg and 200-mg tablets. Please click here to see the full U.S. Prescribing Information for AYVAKIT. About Blueprint Medicines Blueprint Medicines is a global, fully integrated biopharmaceutical company that invents life-changing medicines. We seek to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. Our approach begins by targeting the root causes of disease, using deep scientific knowledge in our core focus areas and drug discovery expertise across multiple therapeutic modalities. We have a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib) which we are bringing to patients with systemic mastocytosis (SM) in the U.S. and Europe. Leveraging our established research, development, and commercial capability and infrastructure, we now aim to significantly scale our impact by advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases including SM and chronic urticaria, breast cancer and other solid tumors. For more information, visit and follow us on X (formerly Twitter; @BlueprintMeds) and LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the potential benefits of AYVAKIT for the treatment of patients with advanced SM and ISM; statements regarding the HARBOR trial for elenestinib; plans, strategies, timelines and expectations for Blueprint Medicines' current or future approved drugs and drug candidates; the potential benefits of any of Blueprint Medicines' current or future approved drugs or drug candidates in treating patients; and Blueprint Medicines' financial performance, strategy, goals and anticipated milestones, business plans and focus. 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Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to: the delay of any current or planned clinical trials or the development of the company's current or future drug candidates; the company's ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the possibility that preclinical and clinical results for the company's drug candidates may not support further development of such drug candidates either as monotherapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions; the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and the possibility patient enrollment rates may be delayed or slower than anticipated; the actions of regulatory agencies and how this may affect the initiation, timing and progress of clinical trials; the company's ability to obtain, maintain and enforce patent and other intellectual property protection for its products and current or future drug candidates it is developing; and the success of the company's current and future collaborations, partnerships or licensing arrangements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the company explicitly disclaims any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. 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