-- Achieved 61% year-over-year growth with

-- 实现了61%的同比增长

$149.4 million

1.494亿美元

in AYVAKIT net product revenues in the first quarter 2025 --

2025年第一季度AYVAKIT的净产品收入 --

-- Raising AYVAKIT net product revenue guidance to

—— 提高AYVAKIT净产品收入指导至

$700

700美元

-

-

$720 million

7.2亿美元

for 2025 --

对于2025年——

--  Initiated BLU-808 proof of concept studies in allergic rhinoconjunctivitis and chronic urticaria –

-- 启动了BLU-808在过敏性鼻结膜炎和慢性荨麻疹中的概念验证研究 --

CAMBRIDGE, Mass.

马萨诸塞州剑桥市

,

，

May 1, 2025

2025年5月1日

/PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq:

/PRNewswire/ -- Blueprint Medicines Corporation (纳斯达克:

BPMC

BPMC

) today reported financial results, provided a business update for the first quarter ended

）今天报告了财务结果，并提供了截至第一季度的业务更新

March 31, 2025

2025年3月31日

, and provided corporate updates.

，并提供了企业更新。

'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

“蓝图医药致力于成为生物技术领域的佼佼者，核心专注于创新、商业卓越以及对我们投资组合的全球投资采取严谨的方法。在2024年取得强劲表现后，我们在2025年继续保持执行的动力，”

Kate Haviland

凯特·哈维兰

, 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.

“Blueprint Medicines首席执行官。‘AYVAKIT正在朝着实现我们到2030年达到20亿美元收入的目标迈进，因为我们继续抓住巨大且不断增长的数十亿美元系统性肥大细胞增多症市场机遇，这将推动我们在下一个十年的营收增长。我们还推进了重点管线项目，取得了显著的组合里程碑，包括启动BLU-808野生型KIT抑制剂的两项概念验证研究，以及elenestinib在惰性系统性肥大细胞增多症中的HARBOR研究进展。”

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.'.

“我们持久且不断增长的商业收入、强劲的现金储备以及审慎的资本配置策略，使我们能够专注于按计划执行业务，同时免受更广泛市场波动的影响。”

First Quarter 2025 Highlights and Recent Progress

2025年第一季度亮点及近期进展

Achieved AYVAKIT net product revenues of

实现了AYVAKIT净产品收入

$149.4 million

1.494亿美元

for the first quarter of 2025, including

2025年第一季度，包括

$129.4 million

1.294亿美元

in the US and

在美国和

$20 million

2000万美元

ex-US, representing 61% percent growth year-over-year.

除美国外，同比增长61%。

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:

启动了两项BLU-808的临床概念验证研究，BLU-808是一种高选择性和强效的研究性口服野生型KIT抑制剂，用于治疗肥大细胞疾病，包括：

A randomized, double-blind, placebo-controlled Phase 2a challenge study of BLU-808 in patients with allergic rhinoconjunctivitis; and

一项针对过敏性鼻结膜炎患者的BLU-808的随机、双盲、安慰剂对照的2a期挑战研究；以及

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.

慢性荨麻疹的2a期概念验证研究。本研究包括慢性诱导性荨麻疹的开放标签部分和慢性自发性荨麻疹的随机、双盲、安慰剂对照部分。

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.

在美国过敏、哮喘和免疫学学会 (AAAAI)/世界过敏组织 (WAO) 会议上展示了12份海报和两份口头数据报告。广泛的数据包括来自AYVAKIT在惰性系统性肥大细胞增多症 (ISM) 患者中的PIONEER研究的三年长期随访数据、显示AYVAKIT治疗的ISM患者在骨骼健康方面取得改善的数据，以及BLU-808健康志愿者研究的数据。

Read the presentations .

阅读这些演示文稿。

here

这里

.

。

Strengthened cash balance with

加强现金余额

$78.7 million

7870万美元

in connection with the sale of the company's equity investment in IDRx, Inc. following its acquisition by GSK plc.

与公司出售其在IDRx, Inc.的股权投资有关，该公司已被GSK plc收购。

Hosting scientific seminar on mast cell activation syndrome (MCAS) with expert physician Dr.

与专业医生Dr.共同举办关于肥大细胞活化综合征（MCAS）的科学研讨会

Matt Giannetti

马特·詹内蒂

on

开启

Wednesday, June 4, 2025

2025年6月4日，星期三

at

在

1:00 p.m.

下午1:00

2025 Financial Guidance

2025年财务指引

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

Blueprint Medicines 正在提高其预期，并预计2025年全球AYVAKIT净产品收入将达到约7亿至7.2亿美元，有望实现其目标。

$2 billion

20亿美元

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.

到2030年，全球AYVAKIT净产品收入预计将增长。这一指导值的上调反映了在第一季度中观察到的免费与商业销售组合的有利变化，以及增长基本面的持续强劲。Blueprint公司预计，随着其继续投资推进优先项目，在创新投资与财务纪律之间取得平衡，2025年的年度现金消耗将继续减少。

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..

蓝图公司继续预计其现有的现金、现金等价物和投资，加上预期的产品收入，将提供足够的资本，使公司能够实现自我可持续的财务状况。

Key Upcoming Milestones

关键的即将到来的里程碑

The company plans to achieve the following remaining milestones in 2025:

公司计划在2025年实现以下剩余里程碑：

Mast cell disorders

肥大细胞疾病

Deliver continued strong and steady AYVAKIT revenue growth.

持续强劲且稳定的AYVAKIT营收增长。

Achieve reimbursement of AYVAKYT in ≥ 20 countries overall.

在至少20个国家实现AYVAKYT的报销。

Activate sites and drive enrollment in HARBOR trial of elenestinib.

激活站点并推动elenestinib的HARBOR试验的注册。

Initiate proof of concept studies of BLU-808 in allergic asthma and MCAS.

启动BLU-808在过敏性哮喘和MCAS中的概念验证研究。

Discovery

发现

Nominate two development candidates, including the company's first protein degrader.

提名两名开发候选人，包括公司的首个蛋白质降解剂。

First Quarter 2025 Results

2025年第一季度业绩

Revenues

收入

: Revenues were

：收入为

$149.4 million

1.494亿美元

for the first quarter of 2025, generated by net product sales of AYVAKIT/AYVAKYT. Revenues were

2025年第一季度，由AYVAKIT/AYVAKYT的净产品销售收入产生的收入为。

$96.1 million

9610万美元

in the first quarter of 2024, including

2024年第一季度，包括

$92.5 million

9250万美元

of net product revenues from sales of AYVAKIT/AYVAKYT and

AYVAKIT/AYVAKYT 销售的净产品收入

$3.6 million

360万美元

in collaboration revenues.

合作收入。

Cost of Sales:

销售成本：

Cost of sales was

销售成本为

$2.8 million

280万美元

for the first quarter of 2025, as compared to

对于2025年第一季度，与

$3.2 million

320万美元

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.

2024年第一季度。减少的主要原因是向我们的合作夥伴销售减少，但产品销售量的增加部分抵销了这一影响。

R&D Expenses:

研发费用：

Research and development expenses were

研发费用为

$91.9 million

9190万美元

for the first quarter of 2025, as compared to

2025年第一季度，与之相比

$88.2 million

8820万美元

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

2024年第一季度。这一增长主要是由于我们优先项目投资的增加，以推进相关临床试验的进展。研发费用包括

$12.1 million

1210万美元

in stock-based compensation expenses for the first quarter of 2025.

2025年第一季度的基于股票的薪酬开支。

SG&A Expenses:

销售、管理及行政费用：

Selling, general and administrative expenses were

销售、一般和管理费用为

$95.8 million

9580万美元

for the first quarter of 2025, as compared to

2025年第一季度，与之相比

$83.6 million

8360万美元

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

2024年第一季度。这一增长主要是由于支持AYVAKIT/AYVAKYT商业化的活动增加。销售、一般和管理费用包括

$16.9 million

1690万美元

in stock-based compensation expenses for the first quarter of 2025.

2025年第一季度的基于股票的薪酬开支。

Net Income:

净利润：

Net income was

净利润为

$0.5 million

50万美元

for the first quarter of 2025, as compared to a net income of

2025年第一季度，相比之下，净收入为

$89.1 million

8910万美元

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

2024年第一季度。2025年第一季度的净收入主要由一次性净收益推动，

$50.0 million

5000万美元

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

与GSK plc收购IDRx, Inc.后公司出售其在IDRx, Inc.的股权相关的记录。2024年第一季度的净收入主要由一次性非现金债务清偿收益推动。

$173.7 million

1.737亿美元

recorded in connection with the Royalty Pharma termination agreement.

与Royalty Pharma终止协议相关的记录。

Cash Position:

现金头寸：

As of

截至

March 31, 2025

2025年3月31日

, cash, cash equivalents and investments were

，现金、现金等价物和投资为

$899.8 million

8.998亿美元

, as compared to

，与...相比

$863.9 million

8.639亿美元

as of

截至

December 31, 2024

2024年12月31日

.

。

Conference Call Information

电话会议信息

Blueprint Medicines will host a live conference call and webcast at

Blueprint Medicines 将举办一场现场电话会议和网络直播。

8:00 a.m. ET

东部时间上午8点

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 .

今天讨论2025年第一季度的财务结果和最近的业务活动。可以通过拨打833-470-1428（国内）或404-975-4839（国际）并输入会议ID 082088来接入电话会议。此次电话会议的网络直播还将在Blueprint Medicines网站的“投资者与媒体”栏目下的“活动与演讲”部分提供。

http://ir.blueprintmedicines.com/

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.

网络直播存档将在电话会议结束后约两小时在Blueprint Medicines的网站上提供，并在电话会议结束后保留30天。

Upcoming Investor Conferences

即将举行的投资者会议

Blueprint Medicines will participate in two upcoming investor conferences:

Blueprint Medicines 将参与接下来的两场投资者会议：

Citizens JMP Life Science Conference

公民JMP生命科学会议

on

打开

Wednesday, May 7, 2025

2025年5月7日，星期三

at

在

12:00 p.m. ET

中午12点（东部时间）

.

。

Goldman Sachs 46

高盛 46

th

th

Annual Global Healthcare Conference

全球年度医疗保健会议

on

打开

Wednesday, June 11, 2025

2025年6月11日，星期三

at

在

10:40 a.m. ET

上午10点40分（东部时间）

.

。

Scientific Webinar Series

科学网络研讨会系列

Blueprint Medicines will host the third event in its scientific seminar series, focused on mast cell activation syndrome (MCAS), on

Blueprint Medicines 将举办其科学研讨会系列的第三场活动，聚焦肥大细胞活化综合征 (MCAS)，于

Wednesday, June 4, 2025

2025年6月4日，星期三

at

在

1:00 p.m. ET

下午1:00（东部时间）

.

。

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

上述演示文稿和任何相关幻灯片的实时网络直播将在Blueprint Medicines网站的投资者与媒体栏目下的“活动与演示”中提供。

http://ir.blueprintmedicines.com

http://ir.blueprintmedicines.com

. A replay of the webcasts will be archived on the Blueprint Medicines website following the events.

网络广播的重播将在活动结束后存档于Blueprint Medicines网站。

About Blueprint Medicines

关于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.

Blueprint Medicines是一家全球性的、完全整合的生物制药公司，致力于发明改变生命的药物。我们力求通过解决在过敏/炎症和肿瘤学/血液学两大核心领域的重大医学问题来减轻人类痛苦。我们的方法始于针对疾病的根本原因，利用我们在核心领域的深厚科学知识以及跨多种治疗方式的药物发现专长。

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 .

我们有两种获批的药物，包括AYVAKIT®/AYVAKYT®（阿伐普利尼），在美国我们将该药带给患有系统性肥大细胞增多症（SM）的患者。

Europe

欧洲

. 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.

利用我们已建立的研发和商业能力及基础设施，我们现在旨在通过推进涵盖从早期科学到晚期临床试验的广泛项目管道，显著扩大我们在肥大细胞疾病（包括系统性肥大细胞增多症和慢性荨麻疹）、乳腺癌及其他实体瘤领域的影响。

For more information, visit .

欲了解更多信息，请访问。

www.BlueprintMedicines.com

www.BlueprintMedicines.com

and follow us on

关注我们

X

X

(formerly Twitter; @BlueprintMeds) and

（原名Twitter；@BlueprintMeds）和

LinkedIn

领英

.

。

Cautionary Note Regarding Forward-Looking Statements

关于前瞻性陈述的警告声明

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.

本新闻稿包含1995年《私人证券诉讼改革法案》（经修订）所指的前瞻性陈述，包括但不限于关于Blueprint Medicines运营计划、战略、时间表及预期的声明，特别是其对2025年增长和净产品收入的预期，以及2030年净产品收入目标；关于AYVAKIT/AYVAKYT全球报销相关的目标；关于其当前或未来已获批药物及候选药物与运营的声明，包括推动elenestinib HARBOR试验入组的计划，以及启动BLU-808在过敏性哮喘和肥大细胞活化综合征（MCAS）中的概念验证研究；关于当前或未来已获批药物及候选药物市场预期的声明；2025年计划的里程碑；其当前或未来已获批药物或候选药物在治疗患者方面的潜在益处；关于流动性和资本状况的声明，包括其现金、现金等价物和投资连同预期产品收入将提供足够的资本以实现财务自给的预期；关于更广泛市场波动性的声明；及其财务表现、战略、目标和预期里程碑、业务计划和重点，包括减少现金消耗的预期。

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.

“目标”、“可能”、“将”、“能够”、“会”、“应该”、“预期”、“计划”、“预期”、“打算”、“相信”、“估计”、“预测”、“项目”、“机会”、“潜力”、“继续”、“针对”等词语及类似表达旨在识别前瞻性陈述，尽管并非所有前瞻性陈述都包含这些识别词。

Any forward-looking statements in this pre.

本前瞻性声明中的任何内容。

Trademarks

商标

Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation.

Blueprint Medicines、AYVAKIT、AYVAKYT 以及相关徽标是 Blueprint Medicines Corporation 的商标。

Blueprint Medicines Corporation

蓝图医药公司

Selected Condensed Consolidated Balance Sheet Data

精选简明综合资产负债表数据

(in thousands)

（单位：千）

(unaudited)

（未经审计）

March 31,

3月31日，

December 31,

12月31日，

2025

2025

2024

2024

Cash, cash equivalents and investments

现金、现金等价物和投资

$

$

899,784

899,784

863,937

863,937

Working capital (1)

营运资本（1）

451,485

451,485

481,882

481,882

Total assets

总资产

1,195,604

1,195,604

1,179,813

1,179,813

Deferred revenue (2)

递延收入（2）

10,316

10,316

10,198

10,198

Liability related to the sale of future royalties and revenues (2)

与未来版税和收入销售相关的责任 (2)

246,632

246,632

255,174

255,174

Term loan (2)

定期贷款 (2)

387,746

387,746

386,970

386,970

Total liabilities

总负债

853,473

853,473

881,148

881,148

Total stockholders' equity

股东权益总额

342,131

342,131

298,665

298,665

(1)

(1)

Blueprint Medicines defines working capital as current assets less current liabilities.

Blueprint Medicines 将营运资本定义为流动资产减去流动负债。

(2)

(2)

Includes both current and long-term portions of the balance.

包含余额的当前部分和长期部分。

Blueprint Medicines Corporation

蓝图医药公司

Condensed Consolidated Statements of Operations Data

合并简明损益表数据

(in thousands, except per share data)

（单位：千，每股数据除外）

(unaudited)

（未经审计）

Three Months Ended

截至三个月末

March 31,

3月31日，

2025

2025

2024

2024

Revenues:

收入：

Product revenue, net

产品收入，净额

$

$

149,413

149,413

$

$

92,525

92,525

Collaboration, license, and other revenue

协作、许可和其他收入

-

-

3,591

3,591

Total revenues

总收入

149,413

149,413

96,116

96,116

Cost and operating expenses:

成本和运营费用：

Cost of sales

销售成本

2,802

2,802

3,191

3,191

Research and development

研发

91,890

91,890

88,191

88,191

Selling, general and administrative

销售、管理和行政费用

95,807

95,807

83,557

83,557

Total cost and operating expenses

总成本与运营费用

190,499

190,499

174,939

174,939

Other income (expense):

其他收入（支出）：

Interest expense, net

净利息支出

(8,129)

(8,129)

(5,895)

(5,895)

Other income, net

其他收入，净值

461

461

376

376

Equity investment gain

股权投资收益

50,039

50,039

-

-

Debt extinguishment gain

债务清偿收益

-

-

173,658

173,658

Total other income, net

净其他总收入

42,371

42,371

168,139

168,139

Income before income taxes

税前收入

1,285

1,285

89,316

89,316

Income tax expense

所得税费用

789

789

180

180

Net income

净利润

$

$

496

496

$

$

89,136

89,136

Net income per share — basic

每股净收益——基本

$

$

0.01

0.01

$

$

1.45

1.45

Net income per share — diluted

每股净收益——稀释后

$

$

0.01

0.01

$

$

1.40

1.40

Weighted-average number of common shares used in net income per share — basic

用于每股净收益的加权平均普通股股数——基本

64,096

64,096

61,580

61,580

Weighted-average number of common shares used in net income per share —diluted

用于每股净收益的加权平均普通股股数——稀释后

66,526

66,526

63,802

63,802

SOURCE Blueprint Medicines Corporation

来源：Blueprint Medicines Corporation

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