-- Breadth of data across 7 presentations, including 4 oral, reflects continued leadership in advancing the understanding and treatment of systemic mastocytosis --

--包括4次口服在内的7次演讲中的广泛数据反映了在促进对系统性肥大细胞增多症的理解和治疗方面的持续领导地位--

CAMBRIDGE, Mass., May 30, 2024 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced multiple upcoming datasets across two key conferences that reinforce the significant real-world burden of systemic mastocytosis (SM), and highlight the durable clinical outcomes of AYVAKIT®/AYVAKYT® (avapritinib) across the spectrum of the disease.

马萨诸塞州剑桥市，2024年5月30日/PRNewswire/--Blueprint Medicines Corporation（纳斯达克：BPMC）今天宣布了两次关键会议上即将发布的多个数据集，这些数据集加强了系统性肥大细胞增多症（SM）的显着现实负担，并强调了AYVAKIT®/AYVAKYT®（阿伐普利替尼）在整个疾病范围内的持久临床结果。

The presentations, which build on over a decade of pioneering research with clinical experts and patient advocates, will be reported at the following meetings:.

这些演讲建立在与临床专家和患者倡导者进行的十多年开创性研究的基础上，将在以下会议上报告：。

European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024, May 31 to June 3

2024年欧洲过敏与临床免疫学学会（EAACI）大会，5月31日至6月3日

European Hematology Association 2024 (EHA24) Hybrid Congress, June 13 to 16

欧洲血液学协会2024（EHA24）混合大会，6月13日至16日

'Our data reinforce the efficacy and safety of prolonged durations of AYVAKIT therapy, and highlight the urgency to diagnose and treat patients living with systemic mastocytosis,' said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. 'Based on unprecedented clinical datasets and the strength of our ongoing commercial launches in the U.S.

Blueprint Medicines首席医学官贝克尔·休斯（Becker Hewes）医学博士说：“我们的数据强化了延长AYVAKIT治疗时间的有效性和安全性，并突出了诊断和治疗全身性肥大细胞增多症患者的紧迫性。”基于前所未有的临床数据集和我们正在美国进行的商业发布的实力。

and Europe, we have made significant progress toward establishing AYVAKIT as the global standard of care. We look forward to continued collaboration with the SM community to redefine what 'well-controlled' means for patients across the spectrum of the disease.'.

在欧洲，我们在将AYVAKIT确立为全球护理标准方面取得了重大进展。我们期待着与SM社区继续合作，重新定义“控制良好”对各种疾病患者的意义。”。

PIONEER: With a median follow-up of more than two years, AYVAKIT showed durable efficacy and a favorable safety profile in patients with ISM. Safety data were consistent for the small number of patients (~10 percent) who dose escalated to 50 mg once daily.

先锋：AYVAKIT的中位随访时间超过两年，对ISM患者显示出持久的疗效和良好的安全性。对于每天一次剂量增加到50毫克的少数患者（〜10%），安全性数据是一致的。

AYVAKIT led to sustained improvements in total symptom score and all symptom domains (assessed by the Indolent Systemic Mastocytosis Symptom Assessment Form), as well as quality of life (assessed by the Mastocytosis Quality of Life Score).

AYVAKIT导致总症状评分和所有症状领域（通过惰性系统性肥大细胞增多症症状评估表评估）以及生活质量（通过肥大细胞增多症生活质量评分评估）的持续改善。

AYVAKIT had a favorable safety and tolerability profile, with patients receiving up to four-plus years of treatment. The most common treatment-related adverse event (AE) was edema, with the majority reported as Grade 1, and the rate of treatment-related AEs leading to discontinuations remained low (3 percent)..

AYVAKIT具有良好的安全性和耐受性，患者接受了长达四年以上的治疗。最常见的治疗相关不良事件（AE）是水肿，大多数报告为1级，导致停药的治疗相关不良事件发生率仍然很低（3%）。。

PATHFINDER: With a median follow-up of more than three years, the median overall survival (OS) for AYVAKIT has not been reached in patients with advanced SM.

开拓者：中位随访时间超过三年，晚期SM患者尚未达到AYVAKIT的中位总生存期（OS）。

The median OS has not yet been reached regardless of disease subtype or prior therapy, reflecting the durable clinical benefits of AYVAKIT. In advanced SM, the historical median OS has ranged from less than six months to about 3.5 years.1

无论疾病亚型或先前的治疗方法如何，中位OS尚未达到，这反映了AYVAKIT的持久临床益处。在高级SM中，历史中位OS从不到六个月到大约3.5年不等。1

The overall response rate2 was 73 percent, and the rate of complete remission with full or partial hematologic recovery was 29 percent.

总体缓解率为73%，完全缓解率为29%，血液学完全或部分恢复率为29%。

AYVAKIT had a favorable benefit/risk profile consistent with previously reported data. Common treatment-related AEs were periorbital edema, thrombocytopenia, peripheral edema and anemia.

AYVAKIT具有与先前报道的数据一致的有利收益/风险概况。常见的治疗相关AE是眶周水肿，血小板减少症，外周水肿和贫血。

PRISM: The largest European study to evaluate patient perspectives in SM reinforces the significant burden of disease, highlighting the urgency to treat.

PRISM：欧洲最大规模的评估SM患者观点的研究强化了疾病的重大负担，突出了治疗的紧迫性。

The Perceptions, Realities & Insights on SM (PRISM) Survey – a collaborative research project led by Blueprint Medicines and involving disease experts and patient organizations across seven countries – further illustrates the substantial impact of SM on patients' quality of life.

关于SM（PRISM）调查的看法、现实和见解（这是一项由蓝图医学牵头的合作研究项目，涉及七个国家的疾病专家和患者组织），进一步说明了SM对患者生活质量的重大影响。

Patients reported debilitating symptoms, impaired physical and mental health functioning, meaningful impacts on the ability to work, and significant polypharmacy use.

患者报告有衰弱症状，身心健康功能受损，对工作能力产生有意义的影响，以及大量使用多种药物。

Healthcare providers broadly recognized the burden of SM; 65 percent reported the disease affected patients' lives 'quite a bit' or 'a great deal,' and 58 percent reported patients lost employment opportunities.

医疗保健提供者广泛认识到SM的负担；65%的人报告说，这种疾病对患者的生活影响“很大”或“很大”，58%的人报告说患者失去了就业机会。

At the start of their respective oral and poster sessions, data presentations will be made available in the 'Science―Publications and Presentations' section of Blueprint Medicines' website.

在他们各自的口头和海报会议开始时，数据演示将在Blueprint Medicines网站的“科学-出版物和演示”部分提供。

EAACI Congress 2024

EAACI大会2024

Oral Presentation: Safety of Avapritinib in Indolent Systemic Mastocytosis (ISM): Longer Term Follow-up from the PIONEER Study (Abstract 000223)

口头介绍：阿伐他尼在惰性系统性肥大细胞增多症（ISM）中的安全性：PIONEER研究的长期随访（摘要000223）

Oral Presentation: The Burden of Indolent Systemic Mastocytosis in Europe: Results from the PRISM Patient Survey (Abstract 000405)

口头介绍：欧洲惰性系统性肥大细胞增多症的负担：PRISM患者调查的结果（摘要000405）

Oral Presentation: Identifying KIT D816V Mutation in Patients with Evidence of Systemic Mast Cell Activation (MCA) and Enriched for Hereditary Alpha-Typtasemia (HaT): Results from the PROSPECTOR Clinical Trial (Abstract 000403)

口头介绍：在有全身肥大细胞活化（MCA）证据并富含遗传性α型斑疹伤寒（HaT）的患者中鉴定KIT D816V突变：PROSPECTOR临床试验的结果（摘要000403）

Poster Presentation: Healthcare Provider Perspectives on Management of European Patients with Systemic Mastocytosis (Abstract 000413)

海报展示：医疗保健提供者对欧洲系统性肥大细胞增多症患者管理的观点（摘要000413）

EHA2024 Congress

EHA2024大会

Oral Presentation: Avapritinib in Patients with Advanced Systemic Mastocytosis (AdvSM): Efficacy and Safety Analysis from the Phase 2 PATHFINDER Study with 3-Year Follow-up (Abstract S224)

口服介绍：阿伐他尼治疗晚期系统性肥大细胞增多症（AdvSM）：2期PATHFINDER研究的疗效和安全性分析，并进行了3年随访（摘要S224）

Poster Presentation: The Burden of Systemic Mastocytosis in Europe: Results from the PRISM Patient Survey (Abstract P1676)

海报介绍：欧洲系统性肥大细胞增多症的负担：PRISM患者调查的结果（摘要P1676）

ePoster Presentation: Patient Diagnostic Journey of Systemic Mastocytosis in Europe: Results from the PRISM Survey (Abstract P2292)

ePoster介绍：欧洲系统性肥大细胞增多症的患者诊断之旅：PRISM调查结果（摘要P2292）

About Systemic Mastocytosis

关于系统性肥大细胞增多症

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).

在大约95%的病例中，系统性肥大细胞增多症（SM）是由KIT D816V突变驱动的罕见疾病。肥大细胞不受控制的增殖和活化会导致多器官系统出现慢性，严重且通常不可预测的症状。绝大多数受影响的人患有惰性系统性肥大细胞增多症（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.

尽管采用多种症状导向疗法进行治疗，但ISM患者仍经常出现多种症状，包括过敏反应，斑丘疹，瘙痒，腹泻，脑雾，疲劳和骨痛。这种疾病负担可能会对生活质量产生深远的负面影响。

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..

患者通常生活在对严重，意外症状的恐惧中，工作或日常活动的能力有限，并将自己隔离以防止不可预测的触发因素。直到2023年，还没有批准用于治疗ISM的疗法。。

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..

少数患者患有晚期SM，其中包括一组高危SM亚型，包括侵袭性SM（ASM），伴有相关血液肿瘤（SM-AHN）的SM和肥大细胞白血病（MCL）。除了肥大细胞活化症状外，晚期SM还与肥大细胞浸润和生存率差引起的器官损伤有关。。

About AYVAKIT (avapritinib)

关于AYVAKIT（阿普替尼）

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.

AYVAKIT（avapritinib）经美国批准。S、 美国食品和药物管理局（FDA）用于治疗三种适应症：患有ISM的成年人，患有晚期SM的成年人，包括ASM，SM-AHN和MCL，以及患有PDGFRA外显子18突变（包括PDGFRA D842V突变）的不可切除或转移性胃肠道间质瘤（GIST）的成年人。该药物的商标名为AYVAKYT（avapritinib），经欧盟委员会批准，用于治疗症状控制不充分的中重度ISM成人，至少一次全身治疗后患有ASM，SM-AHN和MCL的成人，以及患有PDGFRA D842V突变的无法切除或转移性GIST的成人。

The therapy is not recommended for the treatment of patients with low platelet counts (less than 50,000/µL)..

不建议将该疗法用于治疗血小板计数低（低于50000/µL）的患者。。

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.

请单击此处查看完整的U。S、 为AYVAKIT处方信息，并单击此处查看AYVAKYT的欧洲产品特性摘要。

To learn about ongoing or planned clinical trials, contact Blueprint Medicines at medinfo@blueprintmedicines.com or 1-888-BLU-PRNT (1-888-258-7768). Additional information is available at blueprintclinicaltrials.com or clinicaltrials.gov.

要了解正在进行或计划进行的临床试验，请联系Blueprint Medicinesmedinfo@blueprintmedicines.com或1-888-BLU-PRNT（1-888-258-7768）。其他信息可在blueprintclinicaltrials.com或clinicaltrials.gov上获得。

Important Safety Information

重要安全信息

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.

颅内出血AYVAKIT治疗可能会发生严重的颅内出血（ICH）；少于1%的患者发生致命事件。总体而言，在临床试验中接受AYVAKIT治疗的749例患者中，有2.9%发生了ICH（例如硬膜下血肿，ICH和脑出血）。在接受每日200 mg AYVAKIT治疗的晚期SM患者中，75名患者中有2名（2.7%）在开始治疗前血小板计数≥50 x 109/L，80名患者中有3名（3.8%）发生ICH，无论血小板计数如何。

In ISM patients, no events of ICH occurred in the 246 patients who received any dose of AYVAKIT in the PIONEER study..

在ISM患者中，在PIONEER研究中接受任何剂量AYVAKIT的246名患者中均未发生ICH事件。。

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.

密切监测患者的ICH危险因素，包括前一年的血管瘤史，ICH或脑血管意外史，同时使用抗凝药物或血小板减少症。

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.

ICH的症状可能包括头痛，恶心，呕吐，视力改变或精神状态改变。建议患者立即就医以了解ICH的体征或症状。

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.

如果发生任何级别的ICH，则永久停用AYVAKIT。在晚期SM患者中，必须在开始治疗之前进行血小板计数。血小板计数<50 x 109/L的晚期SM患者不推荐使用AYVAKIT。开始治疗后，前8周必须每2周进行一次血小板计数。

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..

治疗8周后，每2周或根据血小板计数临床指示监测血小板计数。通过中断治疗或减少剂量来管理小于50 x 109/L的血小板计数。。

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).

认知效应接受AYVAKIT治疗的患者可能发生认知不良反应，995例接受AYVAKIT治疗的患者中有33%发生认知不良反应，包括：148例晚期SM患者中有28%（3%为≥3级），接受AYVAKIT+最佳支持治疗（BSC）的ISM患者中有7.8%，而接受安慰剂+BSC的患者中有7.0%（1%为3级）。

Depending on the severity and indication, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue..

根据严重程度和适应症，停用AYVAKIT，然后在改善后以相同剂量或减少剂量恢复，或永久停止。。

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..

光敏性AYVAKIT可能引起光敏反应。在临床试验中使用AYVAKIT治疗的所有患者（n=1049）中，2.5%的患者发生光敏反应。建议患者在使用AYVAKIT治疗期间以及停止治疗后一周内限制直接紫外线照射。。

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.

胚胎-胎儿毒性AYVAKIT给孕妇服用时会导致胎儿伤害。告知孕妇对胎儿的潜在风险。建议具有生殖潜力的女性和男性在使用AYVAKIT治疗期间以及服用AYVAKIT最终剂量后6周内使用有效的避孕方法。

Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks after the final dose..

建议女性在使用AYVAKIT治疗期间和最终剂量后2周内不要母乳喂养。。

Adverse Reactions—The most common adverse reactions (≥20%) in patients with Advanced SM were edema, diarrhea, nausea, and fatigue/asthenia.

不良反应晚期SM患者最常见的不良反应（≥20%）是水肿，腹泻，恶心和疲劳/虚弱。

The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing.

ISM患者最常见的不良反应（≥10%）是眼水肿，头晕，外周水肿和潮红。

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.

药物相互作用避免了AYVAKIT与强或中度CYP3A抑制剂的共同给药。如果晚期SM患者无法避免与中度CYP3A抑制剂共同给药，请减少AYVAKIT的剂量。避免将AYVAKIT与强或中度CYP3A诱导剂共同给药。

To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

要报告疑似不良反应，请联系Blueprint Medicines Corporation（电话：1-888-258-7768）或FDA（电话：1-800-FDA-1088）或http://www.fda.gov/medwatch.

Please click here to see the full Prescribing Information for AYVAKIT.

请单击此处查看AYVAKIT的完整处方信息。

About Blueprint Medicines

关于蓝图药物

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是一家全球全面整合的生物制药公司，致力于发明改变生命的药物。我们试图通过解决两个核心重点领域的重要医学问题来减轻人类的痛苦：过敏/炎症和肿瘤学/血液学。我们的方法首先针对疾病的根本原因，在我们的核心重点领域使用深入的科学知识，并在多种治疗方式中使用药物发现专业知识。

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.

我们有两种经批准的药物的成功记录，包括AYVAKIT®/AYVAKYT®（阿伐普利替尼），我们正在为美国和欧洲的系统性肥大细胞增多症（SM）患者提供这些药物。利用我们已建立的研究、开发、商业能力和基础设施，我们现在的目标是通过推进广泛的项目，从早期科学到肥大细胞疾病（包括SM和慢性荨麻疹、乳腺癌和其他实体瘤）的高级临床试验，显着扩大我们的影响力。

For more information, visit www.BlueprintMedicines.com and follow us on X (formerly Twitter; @BlueprintMeds) and LinkedIn..

有关更多信息，请访问www。BlueprintMedicines.com，并在X（以前的Twitter；@BlueprintMeds）和LinkedIn上关注我们。。

Cautionary Note Regarding Forward-Looking Statements

关于前瞻性声明的警示说明

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 for our current or future approved drugs and drug candidates; the potential benefits of AYVAKIT/AYVAKYT or any of our current or future drug candidates in treating patients; the strength of the AYVAKIT/AYVAKYT launches in the U.S.

本新闻稿包含1995年《私人证券诉讼改革法案》修订版意义下的前瞻性声明，包括但不限于：关于蓝图药物运营的计划、战略、时间表和期望的声明，包括对我们目前或未来批准的药物和候选药物的期望；AYVAKIT/AYVAKYT或我们目前或未来的任何候选药物在治疗患者方面的潜在益处；AYVAKIT/AYVAKYT在美国推出的实力。

and Europe; and Blueprint Medicines' ability to establish AYVAKIT/AYVAKYT as the global standard of care for the treatment of SM. The words 'aim,' 'may,' 'will,' 'could,' 'would,' 'should,' 'expect,' 'plan,' 'anticipate,' 'intend,' 'believe,' 'estimate,' 'predict,' 'project,' 'potential,' 'opportunity,' 'continue,' 'target' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

和欧洲；以及Blueprint Medicines将AYVAKIT/AYVAKYT确立为治疗SM的全球护理标准的能力。“目标”，“可能”，“意志”，“可能”，“应该”，“期望”，“计划”，“预期”，“打算”，“相信”，“估计”，“预测”，“项目”，“潜力”，“机会”，“继续”，“目标”和类似的表达都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 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 can.

本新闻稿中的任何前瞻性声明均基于管理层当前的期望和信念，并受到许多风险、不确定性和重要因素的影响，这些风险、不确定性和重要因素可能导致实际事件或结果与本新闻稿中任何前瞻性声明所明示或暗示的事件或结果存在重大差异，包括但不限于与以下相关的风险和不确定性：任何当前或计划的临床试验的延迟或公司当前或未来候选药物的开发；公司成功证明其候选药物的安全性和有效性并及时获得候选药物批准（如果有的话）的能力；该公司药物的临床前和临床结果可能。

Footnotes

脚注

1 Sperr WR, Kundi M, Alvarez-Twose I, et al. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. Lancet Haematol. 2019;6(12):e638-e649.

1 Sperr WR，Kundi M，Alvarez Twose I等。肥大细胞增多症国际预后评分系统（IPSM）：一项回顾性队列研究。柳叶刀血液。2019年；6（12）：e638-e649。

2 Treatment response was defined as complete remission with full or partial hematologic recovery, partial remission or clinical improvement.

2治疗反应定义为完全缓解，血液学完全或部分恢复，部分缓解或临床改善。

Trademarks

商标

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

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

SOURCE Blueprint Medicines Corporation

SOURCE Blueprint Medicines公司