Kira Pharmaceuticals Presents Updated Data of Phase 2 Study of KP104 in Paroxysmal Nocturnal Hemoglobinuria (PNH) at American Society of Hematology (ASH) 2024 Demonstrating Long-Term Safety and Efficacy

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Dec 10, 2024, 08:00 ET

CAMBRIDGE, Mass., Dec. 10, 2024 /PRNewswire/ -- Kira Pharmaceuticals, a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, today announced positive long-term results from its Phase 2 study of KP104 in complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH). KP104 is a first-in-class dual-targeting complement inhibitor (C5 & Factor H) designed to act on both the alternative and terminal pathways.

The updated data, presented at the 2024 American Society of Hematology (ASH) Annual Meeting in San Diego, continue to demonstrate long-term safety and transformative efficacy of KP104 as a novel monotherapy for PNH, addressing significant unmet medical needs in this rare and debilitating condition. Over a two-year treatment period, KP104 showed sustained clinical benefits by significantly improving hemoglobin levels and overall hematological outcomes in PNH patients.

"We are excited to share these promising long-term results with the hematology community," said Dr. Wenru Song, Head of R&D at Kira Pharmaceuticals. "PNH patients urgently need innovative therapies that offer lasting efficacy and safety. KP104's unique dual-targeting mechanism and this long-term safety and efficacy data position it as the best monotherapy for both naïve and treated PNH patients who are non-responsive or sub-responsive to current complement therapies".

The new data presented at ASH 2024 are from 18 patients treated with KP104 subcutaneously, with 56/58 weeks of the treatment time being on Optimal Biological Dose (OBD) for all patients. Key highlights include:

Patients continued to show sustained improvements in hemoglobin levels after switching to OBD (Figure 1): 100% (18/18) achieved a ≥2 g/dL increase from baseline, and 89% (16/18) attained hemoglobin normalization (≥12 g/dL) with an average (SD) of 13.6 (1.5) g/dL in the absence of RBC transfusions. The remaining two patients with co-existing conditions, one with aplastic anemia (AA) and the other with myeloproliferative neoplasms (MPN), also demonstrated hemoglobin improvement approaching near-normal levels.
Patients showed sustained control of LDH to near-normal level throughout the entire treatment period (Figure 2), demonstrating strong intravascular hemolysis (IVH) suppression. At 56/58 weeks post-OBD switch, 94% (17/18) patients achieved LDH <1.5x ULN.
All patients remained free from RBC transfusions between day 1 and week 84/85 (total duration) of KP104 treatment.
Significant clinical improvements were also observed in all other secondary endpoints: normalization of absolute reticulocyte counts, bilirubin levels, and FACIT-fatigue scores after switching to OBD.
KP104 was safe, well-tolerated and produced no treatment-emergent adverse events (TEAEs) at or above grade 3.

The positive long-term results from the Phase 2 study further demonstrate KP104's potential as an optimal first-line monotherapy, providing safe and effective control of both intravascular and extravascular hemolysis in PNH patients. Building on these promising findings, global Phase 3 studies are being planned to establish KP104 as a potential new standard of care for PNH.

Poster Details:

Title: KP104, A Bifunctional C5 Mab-Factor H Fusion Protein, Demonstrates Sustained Long-Term Efficacy and Safety in Complement Inhibitor-Naïve PNH Patients: Updated Results from A Phase 2 Study At 56/58 Weeks of OBD Treatment
Authors: Fengkui Zhang, Bing Han, 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: 508. Bone Marrow Failure: Acquired: Poster II
Time: Sunday, December 8, 2024: 6:00 PM-8:00 PM

The poster will be available on Kira's website at https://www.kirapharma.com/publications.

About KP104
KP104 is a first-in-class bifunctional biologic designed to simultaneously block both the alternative (Factor H) and terminal (C5) complement pathways, providing a powerful and synergistic method of targeting the 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