Avapritinib

-- 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

点击蓝字关注我们编者按：2025年4月18日-19日，由中国临床肿瘤学会（CSCO）和北京市希思科临床肿瘤学研究基金会联合主办的2025 CSCO指南会在济南山东大厦隆重召开。在本次大会上，北京大学人民医院许兰平教授，针对2025 CSCO造血干细胞移植治疗血液系统疾病指南的更新内容进行了深入解读。《肿瘤瞭望-血液时讯》特整理课件精粹，以飨读者。异基因造血干细胞移植（allo-HSCT）是治疗白血病、骨髓增生异常综合征（MDS）、多发性骨髓瘤（MM）、非霍奇金淋巴瘤（NHL）及重型再生障碍性贫血（SAA）等疾病的重要根治手段。随着单倍型移植“北京方案”的建立并在全国范围内推广，allo-HSCT进入“人人皆有供者”的新时代，推动我国移植病例数量持续快速增长。2025版指南立足中国本土的临床实践与科研成果，秉持与时俱进的前瞻视角，从多个方面进行了更新，充分体现持续优化与规范化的发展方向。异基因造血干细胞移植01适应证及时机在异基因造血干细胞移植治疗急性髓系白血病、急性淋巴细胞白血病、慢性髓系白血病、骨髓增生异常综合征、其他B细胞淋巴瘤、T细胞淋巴瘤的适应证中均删去了“≤65岁”的限制。新增“异基因造血干细胞移植治疗地中海贫血的适应证”，具体见下表。02患者及供者评估在患者评估中新增“地中海贫血内容”。03预处理方案在标准清髓性（MAC）预处理中删除了AA部分。新增“地中海贫血移植预处理内容”，见下表。04急性移植物抗宿主病（aGVHD）在aGVHD一线治疗的Ⅱ级推荐中，增加“也可考虑甲泼尼龙1 mg/(kg·d)联合低剂量MTX或低剂量芦可替尼(1B类)”。05慢性移植物抗宿主病（cGVHD）在cGVHD预防的Ⅱ级推荐中，删去了“ATG+PT/CY(2B类)”。在cGVHD的诊断中，新增“眼部GVHD诊断内容”，见下表。在cGVHD一线治疗的Ⅱ级推荐中，删去了“泼尼松+奥法妥木单抗(2B类)”和“泼尼松+伊布替尼(ibrutinib)(2B类)”。在cGVHD二线治疗的Ⅰ级推荐中增加“甲磺酸贝舒地尔(2A类)”，Ⅱ级推荐中增加“西罗莫司(2A类)”和“艾克利单抗(2A类)”，Ⅲ级推荐中增加“本外光分高置换术(ECP)（2C类）”，见下表。新增“cGVHD的患者报告结局评估内容”，见下表。06真菌感染防治在治疗药物的I级推荐中增加“艾沙康唑”，见下表。07病毒感染防治在HBV再激活/感染防治中，增加“干扰素治疗”的Ⅲ级推荐，见下表。08复发防治针对ALL移植后复发的预防，在预处理方案调整的Ⅲ级推荐中，增加“西达本胺联合BUCY预处理方案改善T-ALL/LBL的移植后生存（2B类）”；在靶向药物治疗中，将“Ph-ALL:移植前MRD阳性用贝林妥欧单抗桥接”由Ⅲ级（2B类）推荐调整为Ⅱ级（2A类）推荐，并在Ⅲ级推荐中增加“②移植前CAR-T桥接(MRD阳性)”，见下表。针对MDS移植后复发的预防，在预处理方案中，新增“地西他滨与其他药物(如去甲氧柔红霉素)联合使用可有效降低高危MDS患者移植后的复发率（2A类）”的Ⅱ级推荐，见下表。针对AML移植后复发的抢先治疗，在靶向药物的Ⅲ级推荐中新增“阿伐替尼（3类）”和“去甲基化药物（3类）”；在细胞免疫治疗的I级推荐中新增“改良DLI(2A类)”，见下表。针对ALL移植后复发的抢先治疗，在免疫治疗的Ⅱ级推荐中新增“贝林妥欧单抗（2A类）”，见下表。针对AML移植后复发的治疗，在免疫治疗的Ⅲ级推荐中新增“GO单抗(2A)”；在靶向治疗的Ⅱ级推荐中细化或新增“1.针对FLT3抑制剂吉瑞替尼（1类）和针对FLT3-ITD患者选择索拉非尼联合HMA（2A类）；2.IDH1抑制剂艾伏尼布、奥鲁替尼（2A类）；3.IDH2抑制剂恩西地平（2A类）”；Ⅲ级推荐中新增“2.其他如menin抑制剂(针对KMT2Ar或NPM1突变)（3类）”，见下表。09随访在移植后随访中新增“地中海贫血内容”，见下表。自体造血干细胞移植01适应证针对多发性骨髓瘤自体造血干细胞移植的适应证，将肾功能评估I级推荐中的“血30ml/min≤Cr<60ml/min且尿量正常时，预处理剂量减量(马法兰剂量减为140mg/M2)”和“血Cr<30ml/min，权衡移植利弊”分别调整为Ⅱ级、Ⅲ级推荐，见下表。专家简介许兰平 教授北京大学人民医院国家血液系统疾病临床医学研究中心北京大学人民医院血液科副主任、主任医师、博导海峡两岸医药卫生交流协会第三届、第四届理事会常务理事，第二届、第三届血液学专委会主任委员中华骨髓库北京分库第三届专家委员会主任委员中国研究型医院学会细胞研究与治疗专委会常务委员中国医疗保健国际交流促进会临床微生物与感染分会委员中国造血干细胞捐献者资料库第八届、第九届专家委员会委员《白血病.淋巴瘤》《中华老年多器官杂志》《中华血液学杂志》杂志编委2014、2017位两次列国家科学技术进步奖二等奖获奖者名单主要研究方向：造血干细胞移植治疗骨髓衰竭性疾病的研究责任作者或第一作者发表SCI文章50余篇，共同作者参与发表文章500余篇，参与编写教材或论著十余次（来源：《肿瘤瞭望–血液时讯》编辑部）声 明凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。