Kira Pharmaceuticals, a global clinical-stage biotechnology company, announced that the US 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.
