展品99.1
Blueprint Medicines Reports Third Quarter 2024 Results and Raises AYVAKIT®/AYVAKYT® (avapritinib) Full Year Revenue Guidance
-- Achieved $12820万 in AYVAKIT 2024年第三季度净产品收入区间
调升2024年AYVAKIt净产品收入指引至47500万至48000万美元
预计按计划在年底前启动elenestinib在ISm中注册启动的HARBOR Part 2研究
2024年10月30日麻省剑桥─ Blueprint Medicines Corporation(纳斯达克代码:BPMC)今天报告了财务业绩,提供了截至2024年9月30日第三季度的业务更新,并提供了最新的财务指引。
“通过另一个强劲的AYVAKIt营收业绩季度,我们截至目前的业绩为我们在2025年及未来实现显著增长和创造长期股东价值奠定了基础。AYVAKIt的业绩增长势头使我们有信心再次提高我们今年的营收预期,估计我们将以47500万至48000万美元的收入结束本年度。在接近五亿美元的营收下,结束我们首个完整年度的推出,使AYVAKIt得以成为迄今为止最成功的罕见疾病推广之一,” Blueprint Medicines的执行长凯特·哈维兰说。“AYVAKIt已经在全球病人中显示出了具体好处,长期数据进一步强化了临床上具有意义的好处和耐受性良好的疾病修改治疗的轮廓。AYVAKIt的强劲营收增长加上对最具说服力的产品机会的纪律性投资,使蓝图药物处于实现今年现金燃烧显著下降的位置,同时保持我们专注于长期增长和价值创造。”
2024年第三季度亮点和最近进展
肥大细胞疾病
● | 实现AYVAKIt净产品营业收入为$12820万 2024年第三季度实现营业收入,包括美国营业收入$11310万和国外$1510万,年增长136% |
● | 爱文思控股进行BLU-808单次递增剂量/多次递增剂量健康志愿者研究的第1期,这是一种高度选择性和有效的口服野生型KIt抑制剂,在慢性荨麻疹和其他肥大细胞症有极大潜力。健康志愿者研究数据,包括安全性、药代动力学和药效动力学标志,预计将于2025年初公布。 |
● | 在美国过敏、哮喘和免疫学学会(ACAAI)会议上发表资料,展示AYVAKIt在基线时中、重度的惰性Sm(ISM)患者中的功效,并展示Sm的重大疾病负担,强调诊断和治疗的迫切性。阅读演示文稿 点此. |
● | 于2024年11月14日(星期四)举办关于肥大细胞专业领域发展的科学研讨会,由主要过敏和哮喘专家保罗·欧拜恩医生主持。 |
2024年财务展望
Blueprint Medicines now anticipates approximately $47500万 to $48000万 in global AYVAKIt net product revenues in 2024, an increase from the previous range of $43500万 to $45000万. This updated guidance is based on continued growth in patients on therapy, continued favorability in compliance and other performance factors, and stronger than expected performance outside of the U.S. The company continues to expect that full-year operating expenses and cash burn will decline in 2024 as compared to 2023, and that its existing cash, cash equivalents and investments, together with anticipated product revenues, will enable the company to maintain a
稳固的资本位置,以实现自给自足的财务状况。
重要即将达成的里程碑
该公司计划在2024年底前实现以下剩余里程碑:
肥大细胞疾病
● | 在年底前启动HARBOR试验电脑内腔多层亭隆中的第2部分注册。 |
细胞周期抑制
● | 继续进行战略业务发展讨论。 |
● | 完成BLU-222的第1阶段组合剂量递增,以便在年底前制定注册计划。 |
2024年第三季度成果
● | 收益2024年第三季度由AYVAKIT / AYVAKYt的净产品销售产生的收入为12820万美元。2023年第三季度的收入为5660万美元,包括AYVAKIT / AYVAKYt销售的净产品收入为5420万美元,以及合作、授权和其他收入为240万美元。 |
● | 销售成本: 2024年第三季度的销售成本为190万美元,较2023年第三季度的280万美元降低。这主要是由于向我们在大中华地区的合作伙伴销售较少所致。 |
● | 研发支出: 2024年第三季度的研究和开发支出为8530万美元,较2023年第三季度的11030万美元下降。这主要是由于我们在执行我们的首要计划中实现业务投入的营运效率和临床试验材料制造的时机所致。2024年第三季度的研究与发展支出中包括1260万美元的股票报酬费用。 |
● | 营销、一般及行政支出: 销售、总务及行政支出2024年第三季度为8990万美元,较2023年第三季度的7070万美元增加。此增加主要是由于支持AYVAKIT/AYVAKYt商品化的活动增加所致。销售、总务及行政支出2024年第三季度包括1570万美元的股票报酬费用。 |
● | 净亏损: 2024年第三季度亏损为5,630万美元,相较于2023年第三季度的亏损为13,370万美元。. |
● | 现金状况: 截至2024年9月30日,现金、现金等价物和投资总额为8,8240万美元,相较于2023年12月31日的76720万美元。Blueprint Medicines的现金和投资提供了稳固的资本基础,加上预期的产品收入,公司认为这将使其达到自我可持续的财务状况。. |
会议看涨信息
Blueprint Medicines将于今天上午8:00举行现场电话会议和网络直播,以讨论2024年第三季度的财务业绩和最近的业务活动。可以通过拨打833-470-1428(国内)或404-975-4839(国际),并参考会议ID 387547来参加电话会议。电话会议的网络直播也将在Blueprint Medicines网站的“投资者及媒体”部分的“活动和演示文稿”下提供。http://ir.blueprintmedicines.com/蓝图药物的网络研讨会存档将在会后大约两个小时发布在蓝图药物的网站,并在通话后的30天内提供。
即将举行的投资者会议
蓝图药物将参加三场即将举行的投资者会议:
● | 古根海姆首届医疗保健创新大会 2024年11月12日星期二下午3时30分东部时间。 |
● | Stifel 2024医疗保健会议 2024年11月18日星期一上午10时20分东部时间。 |
● | Jeffries London Healthcare Conference on Thursday, November 21, 2024 at 12:00 p.m. GMT. |
Scientific Webinar Series
● | The second in our scientific seminar series, focused on mast cell diseases, to be held on Thursday, November 14, 2024 at 10:00 a.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 2024; statements regarding its current or future approved drugs and drug candidates and operations, including plans to initiate registration-enabling Part 2 of the HARBOR trial of elenestinib in ISM in 2024, complete Phase 1 combination dose escalation for BLU-222 to inform registration plans and continue strategic business development discussions by the end of 2024, and present healthy volunteer data from its Phase 1 trial for BLU-808 in early 2025; expectations related to the markets for current or future approved drugs and drug candidates, including expectations regarding the size or scale of patient opportunities that future approved drugs and drug candidates could address; 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 will provide a durable capital position which, together with anticipated product revenues, will enable Blueprint Medicines to reach a self-sustainable financial profile; and its financial performance, strategy, goals and anticipated milestones, business plans and focus, including expectations regarding its revenue ramp and continued decline in operating expenses and cash burn. 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: 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; 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)
| | September 30, | | December 31, | |
| | 2024 | | 2023 | |
Cash, cash equivalents and investments | $ | 882,353 | | $ | 767,171 |
Working capital (1) | | 597,187 | | | 593,470 |
Total assets | | 1,199,649 | | | 1,049,250 |
Deferred revenue (2) | | 12,034 | | | 5,604 |
Liability related to the sale of future royalties and revenues (2) | | 261,207 | | | 441,625 |
Term loan (2) | | 386,569 | | | 238,813 |
Total liabilities | | 886,501 | | | 918,641 |
Total stockholders’ equity | | 313,148 | | | 130,609 |
(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 | | Nine Months Ended | ||||||||
| | September 30, | | September 30, | ||||||||
| | 2024 | | 2023 | | 2024 | | 2023 | ||||
Revenues: | | | | | | | | | | | | |
Product revenue, net | | $ | 128,184 | | $ | 54,228 | | $ | 334,825 | | $ | 133,173 |
Collaboration, license and other revenue | | | — | | | 2,338 | | | 27,633 | | | 44,250 |
Total revenues | | | 128,184 | | | 56,566 | | | 362,458 | | | 177,423 |
Cost and operating expenses: | | | | | | | | | | | | |
Cost of sales | | | 1,932 | | | 2,782 | | | 12,716 | | | 8,280 |
Collaboration loss sharing | | | — | | | 1,771 | | | — | | | 4,301 |
Research and development | | | 85,300 | | | 110,252 | | | 257,761 | | | 330,184 |
Selling, general and administrative | | | 89,926 | | | 70,741 | | | 262,822 | | | 215,826 |
Total cost and operating expenses | | | 177,158 | | | 185,546 | | | 533,299 | | | 558,591 |
Other income (expense): | | | | | | | | | | | | |
Interest expense, net | | | (7,616) | | | (3,808) | | | (20,376) | | | (13,624) |
Other income (expense), net | | | 587 | | | (728) | | | 962 | | | (369) |
Debt extinguishment gain | | | — | | | — | | | 173,676 | | | — |
Total other income (expense), net | | | (7,029) | | | (4,536) | | | 154,262 | | | (13,993) |
Loss before income taxes | | | (56,003) | | | (133,516) | | | (16,579) | | | (395,161) |
Income tax expense | | | 273 | | | 197 | | | 554 | | | 907 |
Net Loss | | $ | (56,276) | | $ | (133,713) | | $ | (17,133) | | $ | (396,068) |
Net Loss per share — basic and diluted | | $ | (0.89) | | $ | (2.20) | | $ | (0.27) | | $ | (6.55) |
Weighted-average number of common shares used in net loss per share —basic and diluted | | | 63,381 | | | 60,688 | | | 62,608 | | | 60,445 |
Media Contact
Andrew Law
617-844-8205
media@blueprintmedicines.com
Investor Contact
Jenna Cohen
857-209-3147
ir@blueprintmedicines.com