An Interim analysis of 18 patients, completing 24/25 weeks of treatment in a phase 2 study in complement-naïve PNH patients demonstrates that KP104 is safe, well-tolerated, and has successfully met all primary and secondary endpoints.

对18名患者进行的中期分析表明，KP104是安全的，耐受性良好，并且成功地达到了所有主要和次要终点。

KP104 effectively managed both intravascular and extravascular hemolysis, with all 18 patients achieving a 2g/dL increase of hemoglobin from baseline in the absence of RBC transfusions and the majority of patients achieving normalization range of hemoglobin and LDH levels.

KP104有效地管理了血管内和血管外溶血，所有18例患者在没有红细胞输注的情况下血红蛋白从基线增加2g/dL，大多数患者血红蛋白和LDH水平达到正常范围。

The interim results support the advancement of KP104 to Phase 3 studies as an optimal and safe treatment for PNH patients to address currently unmet medical needs.

中期结果支持KP104进入3期研究，作为PNH患者的最佳和安全治疗方法，以解决目前未满足的医疗需求。

CAMBRIDGE, Mass., Dec. 14, 2023 /PRNewswire/ -- Kira Pharmaceuticals, a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, presented the interim safety and efficacy results from its Phase 2 study of KP104 in complement inhibitor-naïve patients with PNH at the 2023 American Society of Hematology (ASH) Annual Meeting held in San Diego, on December 10th in an oral presentation.

马萨诸塞州剑桥，2023年12月14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，在圣地亚哥举行的2023年美国血液学会（ASH）年会上，介绍了KP104在补体抑制剂初治PNH患者中的中期安全性和有效性研究结果，在12月10日的口头报告中。

The presentation has also been selected to be featured in the 2024 Highlights of ASH. KP104 is a first-in-class bifunctional biologic engineered to inhibit both alternative and terminal complement pathways..

该演讲也被选为2024年ASH的亮点。KP104是一流的双功能生物制剂，可抑制替代和末端补体途径。。

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This open-label Phase 2 clinical trial (NCT05476887) aims to assess the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of KP104 in patients diagnosed with PNH who have not received prior complement inhibitors. The primary objectives of the study are to evaluate key clinical markers including lactate dehydrogenase (LDH) and hemoglobin (Hgb) levels, transfusion requirements, and FACIT-fatigue scores.

这项开放标签的2期临床试验（NCT05476887）旨在评估KP104在未接受补体抑制剂治疗的PNH患者中的疗效，安全性，耐受性，药代动力学（PK）和药效学（PD）。该研究的主要目的是评估关键的临床标志物，包括乳酸脱氢酶（LDH）和血红蛋白（Hgb）水平，输血要求和FACIT疲劳评分。

The interim analysis of the Phase 2 study for KP104 demonstrated positive outcomes from 18 patients who have completed 24/25 weeks of treatment across three escalating doses:.

KP104第二阶段研究的中期分析显示，18名患者在三次递增剂量下完成了24/25周的治疗，取得了积极的结果：。

Patients showed rapid and sustained improvements in hemoglobin levels (Figures 1 and 2). At Week 24/25:

患者的血红蛋白水平迅速持续改善（图1和图2）。在第24周/第25周：

100% (18/18) achieved a ≥2g/dL increase from baseline;

100%（18/18）比基线增加≥2g/dL；

56% (10/18) attained hemoglobin normalization (≥12g/dL) in the absence of RBC transfusions, with the remaining patients approaching near-normal level;

56%（10/18）在没有红细胞输血的情况下达到血红蛋白正常化（≥12g/dL），其余患者接近正常水平；

Average Hgb levels for Cohort 1, 2 and 3 are 11.7 g/dL (SD 1.0), 14.2 g/dL (SD 1.9) and 12.5 g/dL (SD 1.9), respectively.

队列1、2和3的平均Hgb水平分别为11.7 g/dL（SD 1.0），14.2 g/dL（SD 1.9）和12.5 g/dL（SD 1.9）。

Patients demonstrated rapid reduction and sustained control of LDH levels (Figure 3). At Week 24/25:

患者表现出LDH水平的快速降低和持续控制（图3）。在第24周/第25周：

83% (15/18) sustained levels below 1.5x ULN;

83%（15/18）的持续水平低于1.5倍ULN；

72% (13/18) sustained levels below 1x ULN

72%（13/18）持续低于1x ULN

All patients remained free from RBC transfusion between Day 1 and Week 24/25 of KP104 treatment.

在KP104治疗的第1天和第24/25周之间，所有患者均无红细胞输血。

Rapid and clinically significant improvements in absolute reticulocyte counts, bilirubin levels, and FACIT-fatigue scores were consistently observed in all three cohorts through Week 24/25.

在第24/25周的所有三个队列中，始终观察到绝对网织红细胞计数，胆红素水平和FACIT疲劳评分的快速和临床显着改善。

KP104 was safe and well-tolerated without treatment-emergent adverse events (TEAEs) at or above Grade 3. Common AEs included transient injection site induration, headache, and COVID19 infection, all of which were promptly or duly resolved.

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）。常见的AE包括短暂的注射部位硬结，头痛和COVID19感染，所有这些都得到了及时或适当的解决。

Convenient dosing regimen with SC administration every 2 weeks.

方便的给药方案，每2周服用一次SC。

Kira Pharmaceuticals is committed to advancing KP104 as an innovative therapy for patients with PNH and other complement-mediated diseases. The interim results of the Phase 2 study in complement-naïve PNH patients study represent a significant step forward in developing KP104 as an optimal and safe treatment for PNH patients to address currently unmet medical needs.

Kira Pharmaceuticals致力于将KP104作为PNH和其他补体介导疾病患者的创新疗法。补体幼稚PNH患者研究第二阶段研究的中期结果代表了将KP104开发为PNH患者的最佳和安全治疗以解决目前未满足的医疗需求的重要一步。

These data also provide a proof-of concept and strong foundation for future clinical trials in other complement-mediated diseases, such as IgA nephropathy (IgAN), complement 3 glomerulopathy (C3G), and thrombotic microangiopathies secondary to systemic lupus erythematosus (SLE-TMA).Title: KP104, a bifunctional C5 antibody/factor H fusion protein, effectively controls both intravascular and extravascular hemolysis: interim results from a phase 2 study in complement inhibitor-naïve PNH patientsAuthors: Fengkui Zhang, Bing Han, Li Zhang, Chen Yang, Chunrong Wang, Changhe Yue, Hui Yan, Jay Ma, Helen Fu, Chaomei He, Ping Tsui, Jingtao Wu, Richard Lee, Wenru SongSession: 508.

这些数据还为其他补体介导的疾病（如IgA肾病（IgAN），补体3肾小球病（C3G）和继发于系统性红斑狼疮（SLE-TMA）的血栓性微血管病）的未来临床试验提供了概念验证和坚实基础。标题：KP104是一种双功能C5抗体/H因子融合蛋白，可有效控制血管内和血管外溶血：补体抑制剂初治PNH患者2期研究的中期结果作者：张凤奎，韩冰，张丽，杨晨，王春荣，岳长河，许燕，马杰，傅海伦，何朝美，徐萍，吴景涛，李泽楷，宋文如：508。

Bone Marrow Failure: Unraveling the Future of PNH Therapy from Clinical TrialsOral Presentation Time: December 10th, 2023, 4:45pm PTAbout 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.

骨髓衰竭：从临床试验中揭示PNH治疗的未来口腔介绍时间：2023年12月10日下午4:45关于KP104KP104是一种一流的双功能生物制剂，旨在同时阻断替代和终末补体途径，为靶向补体介导疾病的有效驱动因素提供了强大而协同的方法。

This dual-target mechanism of action uniquely positions KP104 to address complement-mediated diseases and potentially provide greater benefits than single-target complement agents. Engineered to have an extended half-life and enhanced potency, KP104 has a formulation suitable for both intravenous and subcutaneous administrations.

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。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 is entering Phase 2 POC tria.

KP104正在进入第二阶段POC试验。