Incyte today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to INCA033989, a first-in-class mutant calreticulin (mutCALR)-targeted monoclonal antibody, for the treatment of patients with essential thrombocythemia (ET) harboring a Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy..

Incyte今天宣布，美国食品药品监督管理局（FDA）已授予INCA033989突破性疗法认定，这是一种首创的针对突变钙网蛋白（mutCALR）的单克隆抗体，用于治疗携带1型CALR突变且对至少一种细胞减灭疗法耐药或不耐受的原发性血小板增多症（ET）患者。

ET is a chronic myeloproliferative neoplasm (MPN) characterized by persistently elevated platelet counts due to abnormal blood cell production in the bone marrow. CALR mutations are the second most common oncogenic driver mutation and are observed in 25% of patients with ET. A 52-bp deletion, also known as a Type 1 mutation, occurs in 55% of patients with a CALR mutation, and is associated with the highest risk of transformation to myelofibrosis (MF) among all ET patients..

ET是一种慢性骨髓增殖性肿瘤（MPN），其特征是由于骨髓中异常的血细胞生成导致血小板计数持续升高。CALR突变是第二常见的致癌驱动突变，在25%的ET患者中可观察到。在携带CALR突变的患者中，55%会出现一种52个碱基对缺失的突变，也称为1型突变，这种突变在所有ET患者中与最高的骨髓纤维化（MF）转化风险相关。

“Incyte has long been committed to improving outcomes for patients with MPNs, and this Breakthrough Therapy designation underscores the potential of INCA033989 to be a novel therapy that could significantly transform the treatment of ET patients, who today have limited treatment options,” said Pablo J.

“Incyte长期以来一直致力于改善MPN患者的治疗效果，此次突破性疗法认定凸显了INCA033989作为一种新型疗法的潜力，有望显著改变ET患者的治疗现状，目前这些患者可选择的治疗方法十分有限，”巴勃罗·J说道。

Cagnoni, M.D., President and Head of Research and Development, Incyte. “The designation allows us to expedite the development pathway for INCA033989 in patients with Type 1 mutations. Looking ahead, we plan to initiate a Phase 3 program evaluating INCA033989 in ET patients with all types of CALR mutations in mid-2026, following alignment with regulators in the first half of next year.”.

卡尼奥尼医学博士，Incyte公司总裁兼研发部门负责人。“这一认定使我们能够加快INCA033989在1型突变患者中的开发进程。展望未来，我们计划在2026年年中启动一项三期项目，在与监管机构于明年上半年达成一致后，评估INCA033989在所有类型CALR突变的ET患者中的效果。”

The FDA Breakthrough Therapy Designation was supported by the early Phase 1 data evaluating INCA033989 in ET patients with a Type 1 CALR mutation available at the time of submission.

FDA的突破性疗法认定得到了在提交时可获得的评估INCA033989用于1型CALR突变ET患者的早期1期数据的支持。

The preliminary Phase 1 data were presented earlier this year at the 2025 European Hematology Association (EHA) Congress. In the study, INCA033989 was well-tolerated and demonstrated rapid and durable normalization of platelet counts across evaluated doses, with greater responses seen at higher doses across both mutation types.

今年早些时候，在2025年欧洲血液学协会（EHA）大会上公布了初步的第一阶段数据。在研究中，INCA033989表现出了良好的耐受性，并在评估的剂量范围内迅速且持久地使血小板计数恢复正常，且在两种突变类型中均观察到较高剂量带来更显著的反应。

Updated results from the Phase 1 dose escalation and expansion trial are planned for presentation at the 2025 ASH Annual Meeting in Orlando (Session 634; Publication #1024; December 8, 4:30-6:00 p.m. ET)..

计划在2025年奥兰多举行的美国血液学会（ASH）年会上公布1期剂量递增和扩展试验的最新结果（分会场634；发表编号#1024；12月8日，下午4:30-6:00，东部时间）。

Incyte plans to develop INCA033989 for patients with Type 1 and non-Type 1 CALR mutations and, following discussions with regulatory agencies, plans to initiate a registrational program evaluating patients with ET with a Type 1 or non-Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy in the first half of next year..

Incyte计划为1型和非1型CALR突变患者开发INCA033989，并在与监管机构讨论后，计划于明年上半年启动一项注册项目，评估携带1型或非1型CALR突变且对至少一种细胞减灭治疗耐药或不耐受的ET患者。

About Essential Thrombocythemia

关于原发性血小板增多症

Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by persistently elevated platelet counts due to abnormal blood cell production in the bone marrow. People living with ET are at increased risk for blood clots and bleeding and a proportion of patients may progress over time to myelofibrosis or acute leukemia..

原发性血小板增多症 (ET) 是一种慢性骨髓增殖性肿瘤 (MPN)，其特征是由于骨髓中异常的血细胞生成，导致血小板计数持续升高。ET患者发生血栓和出血的风险增加，并且部分患者可能随着时间的推移进展为骨髓纤维化或急性白血病。

About Mutations in Calreticulin (mutCALR)

关于钙网蛋白的突变 (mutCALR)

Calreticulin (CALR) is a protein involved in the regulation of cellular calcium levels and normal protein folding. Somatic, or non-inherited, DNA mutations in the CALR gene (mutCALR) can result in abnormal protein function and lead to the development of myeloproliferative neoplasms (MPNs),1 a closely related group of clonal blood cancers in which the bone marrow functions abnormally, overproducing blood cells.2,3 In Essential thrombocythemia (ET) and myelofibrosis (MF), CALR mutations occur in ~25-35% of patients.1,2.

钙网蛋白 (CALR) 是一种参与调节细胞钙水平和正常蛋白质折叠的蛋白质。CALR 基因 (mutCALR) 的体细胞（非遗传性）DNA 突变可能导致蛋白质功能异常，并引发骨髓增殖性肿瘤 (MPNs)，1 这是一组密切相关的克隆性血液癌症，其特征是骨髓功能异常，血细胞过度生成。2,3 在原发性血小板增多症 (ET) 和骨髓纤维化 (MF) 中，约 25-35% 的患者存在 CALR 突变。1,2

Incyte is at the forefront of developing novel therapies for patients with mutCALR ET or MF that target only malignant cells, sparing normal cells, including INCA033989, a first-in-class, mutCALR-specific therapy.

Incyte处于开发针对mutCALR ET或MF患者的新型疗法的前沿，这些疗法仅靶向恶性细胞，而不影响正常细胞，其中包括首创的、针对mutCALR的特异性疗法INCA033989。

About Incyte

关于Incyte

A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity.

一家全球生物制药公司，肩负着解决难题的使命。Incyte遵循科学，为有未满足医疗需求的患者寻找解决方案。通过专有疗法的发现、开发和商业化，Incyte已经建立了针对患者的首创药物组合，并在肿瘤学以及炎症与自身免疫领域拥有强大的产品管线。

Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia..

总部位于特拉华州威尔明顿，Incyte 在北美、欧洲和亚洲都有业务。

For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube.

有关Incyte的更多信息，请访问Incyte.com或在社交媒体上关注我们：LinkedIn、X、Instagram、Facebook、YouTube。

Incyte Forward-Looking Statements

Incyte前瞻性声明

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the presentation of data for INCA033989 studies, the potential this monoclonal antibody offers for patients, and expectations regarding ongoing and future clinical trials contain predictions, estimates, and other forward-looking statements..

本新闻稿中陈述的事项，除本文所载的历史信息外，包括关于INCA033989研究数据的展示、这种单克隆抗体为患者提供的潜力，以及对正在进行和未来的临床试验的预期，包含预测、估计和其他前瞻性陈述。

These forward-looking statements are based on Incyte’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA, EMA, and other regulatory authorities; the efficacy or safety of Incyte and its partners’ products; the acceptance of Incyte and its partners’ products in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in our reports filed with the U.S.

这些前瞻性陈述基于Incyte的当前预期，并受可能致使实际结果产生重大差异的风险和不确定性影响，包括与以下方面相关的意外发展和风险：意外延迟；进一步的研究与开发以及临床试验结果可能不成功或不足以满足适用监管标准或保证继续开发；在临床试验中招募足够数量受试者的能力；FDA、EMA及其他监管机构作出的决定；Incyte及其合作伙伴产品的功效或安全性；Incyte及其合作伙伴产品在市场中的接受度；市场竞争；销售、营销、制造和分销要求；以及我们不时向美国提交的报告中详细说明的其他风险。

Securities and Exchange Commission, including our annual report on Form 10-K and our quarterly report on Form 10-K for the quarter ended September 30, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements..

证券交易委员会，包括我们截至2025年9月30日的年度报告Form 10-K和季度报告Form 10-K。Incyte否认有任何意图或义务更新这些前瞻性声明。

Media

媒体

media@incyte.com

媒体@incyte.com

Investors

投资者

ir@incyte.com

ir@incyte.com

Source: incyte.com

来源：incyte.com