Kira Pharmaceuticals to Present Positive Long-term Results of KP104 in Phase 2 Paroxysmal Nocturnal Hemoglobinuria (PNH) Study at the 2024 European Hematology Association (EHA) Hybrid Congress

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May 16, 2024, 09:00 ET

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.

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

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.

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).
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.
All patients remained free from RBC transfusion between Day 1 and Week 33-58 of KP104 treatment.
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
KP104 was safe and well-tolerated without treatment-emergent adverse events (TEAEs) at or above Grade 3.
Convenient dosing regimen with SC administration every 2 weeks.

Details of the oral presentation are as follows:

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

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

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

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

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

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 a genetic mutation that results 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: [email protected]

SOURCE Kira Pharmaceuticals