Kira Pharma to Present Positive Long-term KP104 Results in Phase 2 PNH Study at 2024 EHA Congress

Kira Pharmaceuticals, a leading biotechnology firm based in Cambridge, Massachusetts, has announced promising long-term safety and efficacy results from its Phase 2 study of KP104. KP104 is a novel treatment targeting complement inhibitor-naïve patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). The findings will be presented at the 2024 European Hematology Association (EHA) Hybrid Congress in Madrid, Spain in June.

Dr. Wenru Song, Head of Research and Development at Kira Pharmaceuticals, expressed enthusiasm over the Phase 2 results, emphasizing that KP104 represents a significant step forward in addressing the unmet medical needs of PNH patients. The data reveals KP104's potential as a safe and effective first-line monotherapy for PNH, with the company now gearing up for global Phase 3 trials to expedite the availability of this innovative therapy to patients.

The Phase 2 study involved 18 patients treated with KP104 for 33-58 weeks, with at least 16 weeks under the optimal biological dose (OBD) regimen. Key outcomes include:

- Hemoglobin Levels: All patients (100%) experienced an increase in hemoglobin levels of ≥2 g/dL from baseline, with an average increase of 7.0 g/dL. Additionally, 88.9% of patients achieved hemoglobin normalization (≥12 g/dL).
- LDH Levels: A substantial 94.4% of patients achieved LDH levels <1.5xULN, reflecting an average reduction of 84.3% from baseline.
- RBC Transfusions: None of the patients required red blood cell transfusions during the study period.
- Secondary Endpoints: Continuous improvements were seen in reticulocyte counts, bilirubin levels, and FACIT-fatigue scores across all patient cohorts.

KP104 was well-tolerated, with no treatment-emergent adverse events (TEAEs) at or above Grade 3. The dosing regimen, which involves subcutaneous administration every two weeks, was found to be convenient for patients.

The upcoming oral presentation titled, "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," is scheduled for June 15, 2024. The presentation will feature contributions from a diverse team of researchers, including Bing Han, Fengkui Zhang, Li Zhang, and several others.

KP104 is a pioneering bifunctional biologic that targets both the alternative and terminal complement pathways. This dual-target mechanism offers an advanced approach to treating complement-mediated diseases, potentially providing more comprehensive benefits than single-target therapies. KP104, designed for both intravenous and subcutaneous administration, is currently undergoing Phase 2 trials for various renal and hematologic conditions and has received Orphan Drug Designation from the FDA for treating PNH. The global Phase 2 trials are being conducted in the U.S., China, and Australia.

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, life-threatening blood disorder characterized by the destruction of red blood cells, blood clot formation, and impaired bone marrow function. Affecting between one to five people per million, PNH is typically caused by a genetic mutation leading to the production of abnormal hematopoietic stem cells. Current treatments, such as C5 inhibitors, do not address extravascular hemolysis (EVH) associated with the alternative pathway. KP104's dual-action mechanism aims to offer a superior alternative by effectively managing both intravascular and extravascular hemolysis.

Kira Pharmaceuticals is dedicated to developing complement-targeted therapies for immune-mediated diseases through its LOGIC platform. With facilities in the U.S., China, and Australia, the clinical-stage biotechnology firm is committed to advancing life-changing therapies globally.

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