Sino-US biotech Kira Pharmaceuticals has announced receiving approval from the National Medical Products Administration (NMPA) to conduct a Phase II clinical study assessing the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of KP104, a first-in-class bifunctional biologic. The drug will be evaluated as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) in China.
PNH: Disease Overview and Treatment Landscape
PNH is a rare, life-threatening blood disease characterized by the production of aberrant red blood cells that are recognized as foreign entities within the body, leading to hyperactivation of the complement system and destruction of red blood cells. Patients with PNH may experience anemia, blood clots, and impairments to bone marrow function. Current treatments for PNH target single complement proteins and have shown reductions in hemolysis. However, recent studies suggest that treatments inhibiting both the alternative and terminal complement pathways may offer further improvements in patient outcomes.
KP104: Mechanism and Development
KP104 is a first-in-class bifunctional biologic designed to simultaneously block the alternative and terminal complement pathways. This unique mechanism of action has the potential to provide more comprehensive protection against complement-mediated damage in PNH patients. Phase I data from the SYNERGY-1 first-in-human (FIH) study of KP104 has demonstrated clinical proof-of-mechanism (POM) for the biologic. The drug received Orphan Drug Designation status from the US FDA for the treatment of PNH earlier this year. Kira Pharmaceuticals plans to present full data from the completed Phase 1 trial at the 2022 American Society of Nephrology meeting later this year.
Future Outlook
The approval for the Phase II clinical study of KP104 underscores Kira Pharmaceuticals’ commitment to advancing innovative treatments for rare diseases. By targeting both the alternative and terminal complement pathways, KP104 aims to improve patient outcomes and address significant unmet medical needs in PNH.-Fineline Info & Tech
