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Kira Pharmaceuticals Showcase Promising Data on KP104 for PNH at ASH 2024

Kira Pharmaceuticals, a leading global biotechnology firm, has made a significant announcement regarding its innovative drug, KP104, at the American Society of Hematology (ASH) Annual Meeting 2024. The company revealed positive long-term outcomes from a Phase 2 study involving KP104, specifically targeting patients suffering from paroxysmal nocturnal hemoglobinuria (PNH). This information underscores Kira's commitment to addressing urgent medical needs in immune-mediated diseases.

A Groundbreaking Therapy

KP104 is designed as a dual-targeting complement inhibitor, focusing on both the C5 and Factor H components of the complement system. This approach aims to inhibit two critical pathways responsible for hemolysis—both the alternative and terminal pathways. The updated data shared during the conference has proven KP104 to be a promising treatment option for naïve PNH patients, marking it as a first-in-class medication.

The trial results showed that over a two-year treatment period, KP104 significantly enhanced hemoglobin levels and improved overall hematological parameters among participants. This outcome is vital, given that PNH patients often experience severe anemia and blood-related complications.

Key Findings from the Study

During the ASH event held in San Diego, Dr. Wenru Song, the Head of R&D at Kira Pharmaceuticals, commented on the data, expressing excitement about the findings. The Phase 2 study included 18 patients who were treated with KP104. The pivotal results included:

• - Sustained Hemoglobin Improvement: All patients experienced an increase of 2 g/dL or more in hemoglobin from baseline. Notably, 89% achieved levels above 12 g/dL without the need for red blood cell transfusions.
• - Control of LDH Levels: Patients maintained near-normal levels of lactate dehydrogenase (LDH), indicating effective suppression of intravascular hemolysis throughout the treatment.
• - No Need for Transfusions: Remarkably, no patient required red blood cell transfusions during the entire 84 to 85 weeks of treatment with KP104.

Additionally, other secondary endpoints showed significant improvements, including normalization of absolute reticulocyte counts, bilirubin levels, and FACIT-fatigue scores after transitioning to the Optimal Biological Dose (OBD).

An Exciting Future Ahead

The absence of treatment-emergent adverse events at or above grade 3 during the study suggests that KP104 is not only effective but also remarkably well-tolerated. Such promising results position KP104 as an optimal monotherapy for both naïve and previously treated PNH patients, especially those who have not responded adequately to existing therapies.

Looking ahead, the company plans to initiate global Phase 3 trials to further establish KP104 as a potential new standard of care for PNH, building on the encouraging outcomes already noted. Kira Pharmaceuticals is committed to advancing its pioneering therapies and fostering better health outcomes for patients around the world.

Understanding Paroxysmal Nocturnal Hemoglobinuria

PNH is a rare but life-threatening blood disorder characterized by the destruction of red blood cells, which may lead to various complications, including blood clots and compromised bone marrow function. Caused primarily by genetic mutations, PNH affects approximately 1 to 5 individuals per million. Current therapeutic options primarily focus on C5 inhibitors, but many do not adequately target the complex mechanisms of this condition.

Kira Pharmaceuticals’ Mission

Based in Cambridge, Massachusetts, Kira Pharmaceuticals is leveraging its LOGIC platform to lead in complement-targeted therapies aimed at immune-mediated diseases. With a robust pipeline and an international footprint, Kira’s aim is to provide breakthrough treatments that can significantly improve the quality of life for patients needing advanced therapy options.

For more information about KP104 and Kira Pharmaceuticals, visit Kira's website.