Kira Pharmaceuticals Receives FDA Orphan Drug Designation for KP104, a Bifunctional Antibody Fusion Protein, for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

CAMBRIDGE,Mass. and SUZHOU,China,July 28,2022 --Kira Pharmaceuticals,a global clinical-stage biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases,today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to KP104 for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).

PNH is a rare,life-threatening blood disease that arises due to hyperactivity of the complement system. An intricate constellation of protein pathways,the complement system is a key component of innate immunity. Aberrant activity within this system can be a driver of diseases such as autoimmune and inflammatory conditions. KP104 is a bifunctional,first-in-class biologic with a unique dual-approach mechanism of action. Designed to selectively block the alternative and terminal pathways,KP104 provides a powerful and synergistic method of targeting validated drivers of disease in the complement system. KP104 has also been engineered to have an extended half-life and potency,with a formulation that can be used for both IV and subcutaneous administration.

"Receiving Orphan Drug Designation is a key milestone for Kira,validating KP104's differentiated potential to provide a safe and effective treatment for PNH patients," said Frederick Beddingfield,MD,PhD,CEO at Kira Pharmaceuticals. "As a bifunctional complement therapy targeting both the alternative and terminal pathways,KP104 has the potential to treat complement-mediated diseases where single-target therapies are not adequate. As we enter Phase 2 across multiple indications,we look forward to continued clinical evaluation of KP104 for patients in need."

TheFDA'sOrphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment,diagnosis,or prevention of rare diseases that affect fewer than 200,000 people inthe United States.

KP104 has completed a Phase 1 first-in-human (FIH) study and is entering Phase 2 trials across multiple indications. The Phase 1 clinical trial was a randomized,double blind,placebo-controlled study designed to evaluate the safety,tolerability,pharmacokinetics (PK),and pharmacodynamics (PD) of escalating single and multiple doses of KP104 in healthy volunteers. Kira plans to present this Phase 1 data at a medical conference later this year.

About KP104

KP104 is a bifunctional,KP104 provides a powerful and synergistic method of targeting validated drivers of disease in the complement system. KP104 has also been engineered to have an extended half-life and potency and has a formulation suitable for both IV and subcutaneous administrations. KP104 is entering Phase 2 trials across multiple indications,including IgA nephropathy (IgAN),C3 Glomerulopathy (C3G),Thrombotic microangiopathies secondary to systemic lupus erythematosus (SLE-TMA) and Paroxysmal Nocturnal Hemoglobinuria (PNH).Phase 2 trials will be conducted globally including in the U.S.,Australia,and South Korea.

About Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare,life-threatening blood disease that arises due to hyperactivity of the complement system,part of the innate immune system. 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. 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 Kira Pharmaceuticals

Kira Pharmaceuticals is a global clinical-stage biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases. Enabled by its LOGIC platform,the company has developed a pipeline of nine (9) novel assets spanning targets across the complement cascade. Kira is committed to advancing life-changing therapies that will transform the lives of patients globally with complement-driven diseases. Kira Pharmaceuticals is headquartered in Cambridge,Massachusetts with additional offices in Suzhou and Shanghai,China. More information on Kira can be found at www.kirapharma.com and on LinkedIn.