/PRNewswire/ -- Sumitomo Pharma America, Inc. (SMPA) today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to nuvisertib (TP-3654) for the treatment of patients with intermediate or high-risk myelofibrosis (MF). The FDA Fast Track Designation is granted to investigational therapies being developed to treat serious or life-threatening conditions that demonstrate the potential to address unmet medical needs.

/PRNewswire/ -- 住友制药美洲公司（SMPA）今日宣布，美国食品药品监督管理局（FDA）已授予nuvisertib（TP-3654）快速通道资格，用于治疗中危或高危骨髓纤维化（MF）患者。FDA快速通道资格授予那些针对严重或危及生命疾病、且显示出满足未竟医疗需求潜力的在研疗法。

Nuvisertib is an oral, investigational, highly selective inhibitor of PIM1 kinase, which demonstrated clinical activity including symptom and spleen responses correlating with cytokine modulation in the updated preliminary Phase 1/2 data presented at the European Hematology Association (EHA) 2025 Congress in .

Nuvisertib 是一种口服的、研究性的、高度选择性的 PIM1 激酶抑制剂，在欧洲血液学协会 (EHA) 2025 大会上公布的更新初步 1/2 期数据显示，其临床活性包括与细胞因子调节相关的症状和脾脏反应。

Milan, Italy

意大利米兰

MF, a serious and rare type of blood cancer, is characterized by the buildup of fibrous tissues in the bone marrow which is caused by dysregulation in the Janus-associated kinase (JAK) signaling pathway. The clinical manifestations of MF include an enlarged spleen, debilitating symptoms and reduction in hemoglobin and/or platelets.

MF是一种严重且罕见的血癌，其特征是骨髓中纤维组织的堆积，这是由Janus相关激酶（JAK）信号通路失调引起的。MF的临床表现包括脾脏肿大、衰弱性症状以及血红蛋白和/或血小板减少。

MF affects 1 in 500,000 people worldwide..

MF影响全球每50万人中的1人。

'This positive momentum for nuvisertib signals strong promise in our pipeline and reflects our dedication to addressing unmet medical needs on behalf of patients with myelofibrosis and their families,' said

“nuvisertib的这一积极势头表明了我们研发管线的巨大潜力，并反映了我们致力于满足骨髓纤维化患者及其家属未被满足的医疗需求的决心，”

Tsutomu Nakagawa

中川勉

, Ph.D, President and Chief Executive Officer of SMPA. 'Receiving FDA Fast Track Designation for nuvisertib in the treatment of myelofibrosis reinforces our confidence in its potential as a treatment option for patients facing a poor prognosis with limited treatment options. We are committed to working closely with the FDA to progress the clinical development of nuvisertib and bring an alternative treatment option to patients with myelofibrosis.'.

博士，SMPA总裁兼首席执行官。“nuvisertib在治疗骨髓纤维化方面获得FDA快速通道资格，进一步增强了我们对其作为治疗选择潜力的信心，特别是对于那些预后较差且治疗选择有限的患者。我们致力于与FDA密切合作，推动nuvisertib的临床开发，为骨髓纤维化患者提供一种替代治疗方案。”

Updated data from the ongoing Phase 1/2 study of nuvisertib in patients with relapsed/refractory MF were presented at the EHA Congress on

在EHA大会上公布了nuvisertib在复发/难治性MF患者中正在进行的1/2期研究的更新数据

June 12, 2025

2025年6月12日

. Preliminary data showed that nuvisertib monotherapy appears to be well tolerated with no dose-limiting toxicities (DLTs). Evaluable patients showed clinical activity including a ≥25% spleen volume reduction (SVR25) in 22.2% of patients and a ≥50% reduction in total symptom score (TSS50) of 44.4% of patients, as well as improvement of bone marrow fibrosis (42.9% patients), hemoglobin (24% patients) and platelet count (26.7% patients).

初步数据显示，nuvisertib单药治疗似乎耐受性良好，未出现剂量限制性毒性（DLTs）。可评估的患者显示出临床活性，其中包括22.2%的患者脾脏体积减少≥25%（SVR25），44.4%的患者总症状评分减少≥50%（TSS50），同时骨髓纤维化改善（42.9%的患者）、血红蛋白改善（24%的患者）和血小板计数改善（26.7%的患者）。

Data also showed that nuvisertib treatment led to significant cytokine modulation [reduction of pro-inflammatory cytokines (e.g. EN-RAGE, MIP-1β) and increase of anti-inflammatory cytokines (e.g. adiponectin)], which demonstrated significant (p<0.001) correlation with symptom and spleen responses. Preclinical.

数据显示，nuvisertib治疗可显著调节细胞因子[减少促炎细胞因子（例如EN-RAGE、MIP-1β）并增加抗炎细胞因子（例如脂联素）]，这与症状和脾脏反应显著相关（p<0.001）。临床前研究。

and emerging clinical data support the development of nuvisertib in combination with JAK inhibitors for the treatment of patients with MF.

新兴的临床数据支持将nuvisertib与JAK抑制剂联合使用，用于治疗MF患者。

'The data observed to date demonstrate promising clinical activity for nuvisertib and the strong potential for selective PIM1 inhibition to slow the progression of myelofibrosis,' said

“迄今为止观察到的数据表明，nuvisertib 具有令人鼓舞的临床活性，且选择性 PIM1 抑制在减缓骨髓纤维化进展方面具有强大潜力，”

Jatin Shah

贾丁·沙阿

, MD, Chief Medical Officer, Oncology. 'Patients with myelofibrosis are in need of new therapeutic approaches, including combination treatment options, that can provide increased and durable response rates with limited hematologic adverse events. The FDA Fast Track Designation reinforces the potential of nuvisertib to provide clinical benefits for patients with myelofibrosis, an unmet medical need.'.

医学博士，肿瘤学首席医学官。‘骨髓纤维化患者需要新的治疗方法，包括组合治疗方案，这些方法可以提高反应率并具有持久的疗效，同时减少血液系统不良反应。FDA快速通道资格进一步证实了nuvisertib为骨髓纤维化患者提供临床益处的潜力，这是一种未被满足的医疗需求。’

About Nuvisertib (TP-3654)

关于Nuvisertib (TP-3654)

Nuvisertib (TP-3654) is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.

Nuvisertib (TP-3654) 是一种口服的在研选择性 PIM1 激酶抑制剂，已在多种通路中显示出潜在的抗肿瘤和抗纤维化活性，包括在临床前模型中诱导细胞凋亡。

Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.

Nuvisertib 被观察到在表达临床上重要的 JAK2 V617F 突变的小鼠和人类造血细胞中抑制增殖并增加凋亡。

Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization, and also reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.

Nuvisertib单独使用以及与ruxolitinib联合使用，在JAK2 V617F和MPLW515L骨髓纤维化小鼠模型中显示出白细胞和中性粒细胞计数正常化，并减少了脾脏大小和骨髓纤维化。

The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate and high-risk myelofibrosis (

nuvisertib 的安全性和有效性目前正在一项针对中高危骨髓纤维化患者的 1/2 期临床研究中进行评估 (

NCT04176198

NCT04176198

). The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in

). 美国食品药品监督管理局（FDA）授予nuvisertib用于骨髓纤维化适应症的孤儿药资格。

May 2022

2022年5月

. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in

日本厚生劳动省（MHLW）授予nuvisertib治疗骨髓纤维化的孤儿药资格。

About Sumitomo Pharma

关于住友制药

Sumitomo Pharma Co., Ltd., is a global pharmaceutical company based in

住友制药株式会社是一家全球性的制药公司，总部位于

Japan

日本

with key operations in the U.S. (Sumitomo Pharma America, Inc.),

在美国有关键业务（住友制药美国公司），

Canada

加拿大

(Sumitomo Pharma Canada, Inc.), and

（住友制药加拿大公司），以及

Europe

欧洲

(Sumitomo Pharma Switzerland GmbH) focused on addressing patient needs in oncology, urology, women's health, rare diseases, psychiatry & neurology, and cell & gene therapies. With several marketed products in the U.S.,

（住友制药瑞士有限公司）专注于满足肿瘤学、泌尿学、妇科学、罕见病、精神病学与神经学以及细胞和基因疗法领域中的患者需求。在美国拥有多种已上市产品，

Canada

加拿大

, and

，以及

Europe

欧洲

, a diverse pipeline of early- to late-stage assets, we aim to accelerate discovery, research, and development to bring novel therapies to patients sooner. For more information on SMPA, visit our website

，我们拥有多样化的早期到晚期资产管道，我们的目标是加速发现、研究和开发，以便更快地将新颖的治疗方法带给患者。有关SMPA的更多信息，请访问我们的网站。