HMB-001 is a novel bispecific antibody designed to be the first prophylactic treatment for Glanzmann Thrombasthenia (GT) and other debilitating bleeding disorders

HMB-001是一种新型双特异性抗体，旨在成为格兰兹曼血栓形成（GT）和其他衰弱性出血性疾病的首次预防性治疗

Phase 1 was successfully completed in the UK; Hemab plans additional sites in Europe and the U.S. for Phase 2

第一阶段在英国成功完成；Hemab计划在欧洲和美国为第二阶段增加更多站点

The U.S. FDA has cleared the Investigational New Drug (IND) application and granted Fast Track Designation to HMB-001 for the treatment of GT

美国FDA已批准研究性新药（IND）申请，并授予HMB-001治疗GT的快速通道指定

COPENHAGEN, Denmark and CAMBRIDGE, Mass., Dec. 11, 2023 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, announced today that it has completed Phase 1, the single ascending dose part, and transitioned to Phase 2, the multiple ascending dose part, of its Phase 1/2 clinical study of HMB-001 in Glanzmann Thrombasthenia (GT), a platelet disorder that causes severe, potentially life-threatening bleeding episodes..

丹麦哥本哈根和马萨诸塞州剑桥，2023年12月11日，临床阶段生物技术公司Hemab Therapeutics今天宣布，它已经完成了第一阶段的单次递增剂量部分，并过渡到第二阶段的多次递增剂量部分，HMB-001在格兰兹曼血栓形成（GT）中的1/2期临床研究，这是一种血小板疾病，可导致严重的，可能危及生命的出血发作。。

The company also announced that the U.S. Food and Drug Administration (FDA) has cleared Hemab's investigational new drug application (IND) for HMB-001 in GT, enabling enrollment in the U.S. Phase 1 of the clinical study was completed in the UK, and Phase 2 will include additional sites in Europe as well as the U.S..

该公司还宣布，美国食品和药物管理局（FDA）已批准Hemab在GT中针对HMB-001的研究性新药申请（IND），从而能够进入美国。临床研究的第一阶段已在英国完成，第二阶段将包括欧洲和美国的其他网站。。

In addition, the FDA granted Fast Track designation to HMB-001, emphasizing the seriousness and high unmet need for treatments for GT. The Fast Track program enables Hemab to have more frequent interactions with the FDA to facilitate the development of HMB-001.

此外，FDA授予HMB-001快速通道名称，强调GT治疗的严重性和高度未满足的需求。快速通道计划使Hemab能够与FDA进行更频繁的互动，以促进HMB-001的发展。

'People living with Glanzmann Thrombasthenia can experience frequent, sometimes severe bleeds that can be life-threatening and compromise their quality of life. Currently, there are no prophylactic treatment options that would reduce or prevent these bleeding episodes,' said Benny Sorensen, MD, PhD, CEO of Hemab.

“患有格兰兹曼血栓形成症的人可能会经历频繁的，有时是严重的出血，这可能危及生命并影响他们的生活质量。Hemab首席执行官Benny Sorensen博士说，目前还没有预防性治疗方案可以减少或预防这些出血事件。

'The completion of Phase 1 on time and transitioning to Phase 2 is an important milestone for HMB-001 and Hemab. Furthermore, expanding our clinical study into the U.S., following clearance of the IND, and Fast Track designation are a testament to the importance of advancing prophylactic treatment for people living with Glanzmann Thrombasthenia.'  .

“按时完成第一阶段并过渡到第二阶段是HMB-001和Hemab的重要里程碑。此外，在IND获得批准后，将我们的临床研究扩展到美国，并快速指定，这证明了推进预防性治疗对格兰兹曼血栓形成患者的重要性。”。

The Phase 1/2 clinical study evaluates the safety, tolerability, pharmacodynamics, and pharmacokinetics of HMB-001. Initial efficacy signals based on an assessment of changes in bleeding frequency will also be measured. The study is composed of three parts: Part A, single ascending dose, Part B, multiple ascending dose, and Part C, extended dosing. Hemab plans to report data from the Phase 1, single ascending dose portion, at an international scientific conference in early 2024..

1/2期临床研究评估HMB-001的安全性，耐受性，药效学和药代动力学。还将测量基于出血频率变化评估的初始疗效信号。该研究由三部分组成：A部分，单次递增剂量，B部分，多次递增剂量和C部分，延长剂量。Hemab计划在2024年初的一次国际科学会议上报告第一阶段单次递增剂量部分的数据。。

The Phase 1/2 study design was detailed in the company's poster presentation (number 1225), 'A Phase 1/2, First-in-Human, Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia,' at the 65th American Society of Hematology Annual Meeting and Exposition..

该公司的海报展示（编号1225）详细介绍了1/2期研究设计，“在第65届美国血液学会年会和博览会上，研究HMB-001在格兰兹曼血栓形成患者中的安全性，耐受性，药代动力学，药效学和有效性的1/2期人体首次研究”。。

About Glanzmann Thrombasthenia

关于格兰兹曼血栓形成

Glanzmann Thrombasthenia (GT) is a rare and severe bleeding disorder associated with debilitating and sometimes life-threatening bleeding episodes. Initial findings from the international Glanzmann's 360 (GT360) natural history study, Hemab's research initiative in partnership with UK specialist research consultancy Haemnet, found that 87% of the 104 respondents reported experiencing at least one bleed in the previous week, and 37% of those bleeds required medical treatment..

格兰兹曼血栓形成（GT）是一种罕见且严重的出血性疾病，与衰弱甚至有时危及生命的出血发作有关。国际格兰兹曼360（GT360）自然史研究（Hemab的研究计划与英国专业研究咨询公司Haemnet合作）的初步发现发现，104名受访者中有87%报告称上周至少有一次出血，其中37%的出血需要治疗。。

These bleeding episodes have a significant impact on the mental health and quality of life of people living with GT. Low mood, emotional problems, and social isolation were reported by participants (66%, 50%, and 44% respectively) and 80% reported that they missed school or work due to bruising or bleeding.

这些出血事件对GT患者的心理健康和生活质量产生重大影响。参与者报告情绪低落，情绪问题和社交孤立（分别为66%，50%和44%），80%的人报告他们因瘀伤或出血而错过了学校或工作。

To date, there are no effective prophylactic treatment options for people living with GT..

迄今为止，尚无针对GT患者的有效预防性治疗选择。。

About HMB-001

关于HMB-001

HMB-001 is bispecific antibody that binds and stabilizes endogenous factor VIIa (FVIIa) with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for accumulation of endogenous FVIIa in the body, recruitment of FVIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage.

HMB-001是双特异性抗体，其与一个抗体臂结合并稳定内源性因子VIIa（FVIIa），并与另一个抗体臂结合活化血小板上的TLT-1。这允许体内内源性FVIIa的积累，将FVIIa直接募集到已知有助于凝血的活化血小板表面，并在没有组织损伤的情况下避免凝血活性。

HMB-001 is designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia with the potential to treat other debilitating rare bleeding disorders..

HMB-001旨在成为格兰兹曼血栓形成的一流预防性治疗方法，有可能治疗其他使人衰弱的罕见出血性疾病。。

About Hemab Therapeutics

关于Hemab Therapeutics

Hemab is a clinical-stage biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA and Copenhagen, Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need.

Hemab是一家临床阶段的生物技术公司，正在开发针对严重，服务不足的出血和血栓性疾病的新型预防疗法。总部位于马萨诸塞州剑桥和丹麦哥本哈根的Hemab正在开发一系列基于单克隆和双特异性抗体的治疗方法，以改变未满足需求高的患者的治疗模式。

The company's strategic guidance, Hemab 1-2-5TM, targets the development of 5 independent assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, Bernard Soulier Syndrome, Von Willebrand Disease, and other serious disorders.

该公司的战略指导方针Hemab 1-2-5TM的目标是到2025年开发5项独立资产，为格兰兹曼血栓形成、VII因子缺乏、伯纳德·苏利尔综合征、Von Willebrand病和其他严重疾病等高度未满足需求的凝血障碍患者提供期待已久的创新。

Learn more at hemab.com..

更多信息，请访问hemab.com。。

SOURCE Hemab Therapeutics

来源Hemab Therapeutics