CAMBRIDGE, Mass., May 16, 2024 /PRNewswire/ -- Kira Pharmaceuticals, a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, today announced that it will present positive long-term safety and efficacy results from its Phase 2 study of KP104 in complement inhibitor-naïve patients with PNH in an oral session at the 2024 European Hematology Association (EHA) Hybrid Congress, to be held in Madrid, Spain, from June 13-16, 2024..

马萨诸塞州剑桥，2024年5月16日/PRNewswire/--Kira Pharmaceuticals是一家全球生物技术公司，开创了治疗免疫介导疾病的转化补体疗法，今天宣布，它将在2024年6月13日至16日在西班牙马德里举行的2024年欧洲血液学协会（EHA）混合大会上，在补体抑制剂初治PNH患者的口服会议上，展示KP104的长期安全性和有效性。。

'We are excited to share these promising Phase 2 results of KP104 at the upcoming EHA 2024 Hybrid Congress, which represents a significant advancement in addressing the unmet medical needs of patients with PNH,' said Dr. Wenru Song, Head of R&D at Kira Pharmaceuticals. 'The encouraging long-term safety and efficacy data demonstrate the potential of KP104 as an optimal and safe first-line monotherapy for PNH patients.

Kira Pharmaceuticals研发负责人宋文茹（Wenru Song）博士说：“我们很高兴在即将举行的EHA 2024混合大会上分享KP104的这些有希望的第二阶段成果，这在解决PNH患者未满足的医疗需求方面取得了重大进展。”令人鼓舞的长期安全性和有效性数据证明了KP104作为PNH患者最佳和安全的一线单一疗法的潜力。

We are actively preparing for global Phase 3 development and remain dedicated to bringing this innovative therapy to patients as quickly as possible.'.

我们正在积极准备全球第三阶段的开发，并致力于尽快将这种创新疗法带给患者。”。

Key findings from 18 patients who received 33-58 weeks of treatment of KP104, of which at least 16 weeks were under the optimal biological dose (OBD) regimen.

18名接受KP104治疗33-58周的患者的主要发现，其中至少16周处于最佳生物剂量（OBD）方案下。

Patients demonstrated continuous improvements in hemoglobin levels: 100% (18/18) of patients sustained an Hgb increase of ≥2 g/dL from baseline, with mean (SD) Hgb levels increasing by 7.0 (2.1) g/dL over baseline, and 88.9% (16/18) patients achieving Hgb normalization (≥12 g/dL).

患者表现出血红蛋白水平的持续改善：100%（18/18）的患者Hgb从基线水平持续增加≥2 g/dL，平均（SD）Hgb水平比基线水平增加7.0（2.1）g/dL，88.9%（16/18）患者达到Hgb正常化（≥12 g/dL）。

Patients showed sustained control of LDH levels: LDH <1.5xULN was achieved by 94.4% (17/18) patients, with a mean (SD) % LDH reduction of 84.3 (8.7) % from baseline.

患者表现出LDH水平的持续控制：94.4%（17/18）患者的LDH<1.5xULN，平均（SD）%LDH比基线降低84.3（8.7）%。

All patients remained free from RBC transfusion between Day 1 and Week 33-58 of KP104 treatment.

在KP104治疗的第1天和第33-58周之间，所有患者均无红细胞输注。

Sustained clinical improvements in all other secondary endpoints: normalization of absolute reticulocyte counts, bilirubin levels, and FACIT-fatigue scores were continuously observed across all three cohorts

所有其他次要终点的持续临床改善：在所有三个队列中连续观察到绝对网织红细胞计数，胆红素水平和FACIT疲劳评分的正常化

KP104 was safe and well-tolerated without treatment-emergent adverse events (TEAEs) at or above Grade 3.

KP104安全且耐受性良好，无3级或3级以上的治疗紧急不良事件（TEAE）。

Convenient dosing regimen with SC administration every 2 weeks.

方便的给药方案，每2周服用一次SC。

Details of the oral presentation are as follows:

口头陈述的细节如下：

Title: KP104, a bifunctional C5 mAb-factor H fusion protein, effectively controls intravascular and extravascular hemolysis in complement inhibitor-naïve PNH patients: long-term results from a phase 2 study

标题：KP104是一种双功能C5 mAb因子H融合蛋白，可有效控制未使用补体抑制剂的PNH患者的血管内和血管外溶血：2期研究的长期结果

Authors: Bing Han, Fengkui Zhang, Li Zhang, Chen Yang, Changhe Yue, Chunrong Wang, Jay Ma, Chaomei He, Ping Tsui, Jingtao Wu, Qing Yu Christina Weng, Richard Lee, Helen Fu, Hui Yan, Wenru Song

作者：韩冰、张凤奎、张丽、陈阳、岳昌和、王春荣、马杰、何朝美、徐萍、吴景涛、邱庆宇、翁佳欣、李泽楷、傅海伦、许燕、宋文如

Session Name: s454 Clinical and Translational in Bone Marrow Failure and PNH

课程名称：s454骨髓衰竭和PNH的临床和转化

Oral Presentation Date and Time: June 15, 2024, 16:30 - 17:45 CEST

口头陈述日期和时间：2024年6月15日16:30-17:45 CEST

Additional information about the EHA 2024 and the abstract is available at: https://ehaweb.org/congress/eha2024-hybrid-congress/

有关EHA 2024和摘要的其他信息，请访问：https://ehaweb.org/congress/eha2024-hybrid-congress/

About KP104KP104 is a first-in-class bifunctional biologic designed to simultaneously block both the alternative and terminal complement pathways, providing a powerful and synergistic method of targeting validated drivers of complement-mediated disease. This dual-target mechanism of action uniquely positions KP104 to address complement-mediated diseases and potentially provide greater benefits than single-target complement agents.

关于KP104KP104是一流的双功能生物制剂，旨在同时阻断替代和末端补体途径，为靶向补体介导的疾病的有效驱动因素提供了一种强大而协同的方法。这种双靶点作用机制独特地定位KP104以解决补体介导的疾病，并可能比单靶点补体剂提供更大的益处。

Engineered to have an extended half-life and enhanced potency, KP104 has a formulation suitable for both intravenous and subcutaneous administrations. KP104 is entering Phase 2 POC trials across multiple renal disease and hematologic indications and has been granted Orphan Drug Designation by the FDA for the treatment of paroxysmal nocturnal hemoglobinuria.

KP104经过设计，具有延长的半衰期和增强的效力，其配方适用于静脉内和皮下给药。KP104正在进入多种肾脏疾病和血液学适应症的2期POC试验，并已被FDA批准用于治疗阵发性夜间血红蛋白尿的孤儿药。

Phase 2 trials will be conducted globally, including in the U.S., China, and Australia. KP104 is an investigational agent not yet approved for any indication by any health authority..

第二阶段试验将在全球范围内进行，包括在美国，中国和澳大利亚。KP104是一种尚未经任何卫生当局批准用于任何适应症的研究药物。。

About Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disease that is characterized by the destruction of red blood cells, formation of blood clots, and impairment of bone marrow function. PNH affects between 1 and 5 people per million and is almost always caused by a genetic mutation that results in production of aberrant hematopoietic stem cells.

关于阵发性夜间血红蛋白尿阵发性夜间血红蛋白尿是一种罕见的威胁生命的血液疾病，其特征是红细胞破坏，血栓形成和骨髓功能受损。PNH影响每百万人中有1至5人，几乎总是由导致异常造血干细胞产生的基因突变引起的。

These stem cells produce irregular red blood cells that are highly susceptible to destruction via complement activation. Current therapies include C5 inhibitors, which do not address extravascular hemolysis (EVH) related to the alternative pathway or a C3 inhibitor, which may address EVH but may not adequately block C5 downstream, leading to life-threatening breakthrough hemolysis (Breakthrough Hemolysis in PNH with Proximal or Terminal Complement Inhibition, N Engl J Med, July 14, 2022)..

这些干细胞产生不规则的红细胞，极易通过补体激活而被破坏。目前的治疗方法包括不解决与替代途径相关的血管外溶血（EVH）的C5抑制剂或C3抑制剂，其可能解决EVH但可能不足以阻断C5下游，导致危及生命的突破性溶血（PNH中具有近端或末端补体抑制的突破性溶血，N Engl J Med，2022年7月14日）。。

About Kira PharmaceuticalsKira Pharmaceuticals is a clinical-stage biotechnology company pioneering complement- targeted therapies to treat immune-mediated diseases. Enabled by its LOGIC platform, the company has developed a robust pipeline of novel assets against validated complement targets. Headquartered in Cambridge, Massachusetts and with facilities in China and Australia, Kira Pharmaceuticals has established a global team committed to advancing life-changing therapies to patients around the world.

关于Kira PharmaceuticalsKira Pharmaceuticals是一家临床阶段的生物技术公司，开创了补体靶向疗法治疗免疫介导疾病的先河。凭借其逻辑平台，该公司针对经过验证的补充目标开发了强大的新型资产管道。Kira制药公司总部位于马萨诸塞州剑桥市，在中国和澳大利亚设有工厂，成立了一支全球团队，致力于为世界各地的患者推进改变生命的疗法。

More information on Kira can be found at www.kirapharma.com and on LinkedIn..

有关Kira的更多信息，请访问www.kirapharma.com和LinkedIn。。

CONTACT: Kiramedia@kirapharma.com

联系人：Kiramedia@kirapharma.com

SOURCE Kira Pharmaceuticals

来源Kira Pharmaceuticals