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. 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.
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. 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:
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. 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 patients
Authors: 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 Song
Session: 508. Bone Marrow Failure: Unraveling the Future of PNH Therapy from Clinical Trials
Oral Presentation Time: December 10th, 2023, 4:45pm PT
About KP104
KP104 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. 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. 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.
About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal 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 genetic mutations that result in production of aberrant hematopoietic stem cells. 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).
About Kira Pharmaceuticals
Kira 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. More information on Kira can be found at www.kirapharma.com and on LinkedIn.
Contact: Kiramedia@kirapharma.com
SOURCE Kira Pharmaceuticals