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

CAMBRIDGE,Mass. and SUZHOU,China,Oct. 17,2022 -- Kira Pharmaceuticals,a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases,announced today that the Chinese National Medical Products Administration (NMPA) has cleared the Investigational New Drug (IND) application for evaluation of KP104,a first-in-class bifunctional biologic that selectively targets the alternative and terminal complement pathways,in paroxysmal nocturnal hemoglobinuria (PNH). This approval enables 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 advancement of lead asset,KP104,as a next-generation treatment for complement-mediated diseases like PNH. KP104 has been engineered to simultaneously block two key complement targets,an approach we believe will make it a groundbreaking therapeutic option for patients awaiting more effective treatments to address their conditions," said Angela Yan,President for China & Asia Development and Operations of Kira Pharmaceuticals.

PNH is a rare,life-threatening blood disease in which aberrant red blood cells are produced and recognized as foreign entities within the body,resulting in hyperactivation of the complement system and destruction of red blood cells. Patients with PNH may also experience anemia,blood clots,and impairments to bone marrow function. Current complement targeted treatments for PNH operate via inhibition of single complement proteins. While these drugs have resulted in 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 biologic that simultaneously blocks the alternative and terminal complement pathways by virtue of its bifunctional design. Phase 1 data from the SYNERGY-1 first-in-human (FIH) study of KP104 has demonstrated clinical proof-of-mechanism (POM) for the biologic,which received Orphan Drug Designation for treatment PNH from the US Food and Drug Administration (FDA) earlier this year. Kira will present full data from the completed Phase 1 trial at 2022 American Society of Nephrology annual meeting later this year.

About Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria (PNH) 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. Due to the complexity of complement biology and multiple pathways driving PNH pathology,there remains a significant unmet medical need for next-generation drugs with better efficacy and convenience of administration than offered by current therapies.

About KP104

KP104 is a first-in-class bifunctional biologic designed to simultaneously and selectively 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 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 (PNH). Phase 2 trials will be conducted globally,including in the U.S.,Australia,and South Korea.KP104 is an investigational agent not yet approved for any indication by any health authority.

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.