Kira Pharmaceuticals Announces IND Approval from China NMPA for Phase 2 Evaluation of KP104 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

CAMBRIDGE, Mass. and SUZHOU, China, October 17, 2022 /PRNewswire/ — Kira Pharmaceuticals, a global biotechnology company pioneering transformational complementary therapies to treat immune-mediated diseases, today announced that China’s National Medical Products Administration (NMPA) has cleared the application for new Investigational Drug (IND) for the evaluation of KP104, a first-in-class bifunctional biologic that selectively targets alternative and terminal complement pathways, in paroxysmal nocturnal hemoglobinuria (PNH). This approval enables the Phase 2 study of the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of KP104 in participants with PNH in China.

“This clearance represents an important milestone for Kira in our development of lead asset, KP104, as a next-generation treatment for complement-mediated diseases such as PNH. KP104 has been designed to simultaneously block two key complement targets, an approach that we believe will make it a breakthrough treatment option for patients awaiting more effective therapies to treat their conditions,” said Angela Yanpresident for China & Asia Development and operations of Kira Pharmaceuticals.

PNH is a rare and life-threatening blood disorder in which aberrant red blood cells are produced and recognized as foreign entities in the body, leading to overactivation of the complement system and destruction of red blood cells. Patients with PNH may also experience anemia, blood clots, and alterations in bone marrow function. Current complement-targeted treatments for PNH work via inhibition of single complement proteins. Although these drugs have shown reductions in hemolysis, recent studies suggest that treatments that inhibit both the alternative and terminal complement pathways may offer further improvements in patient outcomes.

KP104 is a biological agent that simultaneously blocks alternative and terminal complement pathways due to its bifunctional design. Phase 1 data from the first-in-human (FIH) SYNERGY-1 study of KP104 has demonstrated clinical proof of mechanism (POM) for the biologic drug, which has been granted orphan drug designation for the treatment of HPN by the United States Food and Drug Administration (FDA) earlier this year. Kira will present full data from the completed Phase 1 trial at the 2022 American Society of Nephrology Annual Meeting later this year.

About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening blood disorder characterized by destruction of red blood cells, formation of blood clots and impaired bone marrow function. PNH affects between 1 and 5 people per million and is almost always caused by a genetic mutation that results in the production of aberrant hematopoietic stem cells. These stem cells produce irregular red blood cells that are very susceptible to destruction by complement activation. Due to the complexity of complement biology and the multiple pathways driving PNH pathology, there remains a significant unmet medical need for next-generation drugs with superior efficacy and convenience of administration. those offered by current therapies.

About KP104
KP104 is a first-in-class bifunctional biologic designed to simultaneously and selectively block alternative and terminal complement pathways, providing a potent and synergistic method to target validated drivers of complement-mediated disease. This dual-target mechanism of action uniquely positions KP104 to treat complement-mediated diseases and potentially offers greater benefits than single-targeted complement agents. Designed to have an extended half-life and potency, KP104 has a formulation suitable for intravenous and subcutaneous administration. KP104 is entering Phase 2 POC trials for several kidney diseases and hematology indications and has received orphan drug designation from the FDA for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Phase 2 trials will be conducted around the world, including the United States, China, Australia, and South Korea. KP104 is an investigational agent that is not yet approved for any indication by any health authority.

About Kira Pharmaceuticals
Kira Pharmaceuticals is a clinical-stage biotechnology company pioneering targeted supplement therapies to treat immune-mediated diseases. Through its LOGIC platform, the company has developed a strong pipeline of new assets against validated complement targets. Based at Cambridge, MA and with facilities in China and Australia, Kira Pharmaceuticals has built a global team committed to advancing life-changing therapies for patients around the world. More information about Kira can be found at and on LinkedIn.

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