CLINICAL NEWS | First Cohort of Patients Dosed In Phase 2 Study Of KP104 In PNH
Kira Pharmaceuticals, a global biotechnology company headquartered in Cambridge, Massachusetts, US, announced that the first cohort of patients with paroxysmal nocturnal hemoglobinuria (PNH) has been dosed in a Phase 2 trial evaluating KP104 for the treatment of the disorder in China.
KP104 is a first-in-class, bifunctional biologic designed to modulate complement activity through selective targeting of key intervention points in the alternative and terminal complement pathways. On July 28, 2022, the experimental therapy was granted Orphan Drug Designation by the FDA for the treatment of PNH.
‘’Progression of KP104 into Phase 2 represents an important milestone for Kira in our pursuit of more effective treatments for complement-mediated diseases with few treatment options,’’ said Frederick Beddingfield, MD, PhD, CEO of Kira Pharmaceuticals. ‘’This advancement is supported by key Phase 1 biomarker and safety data that confirm KP104’s dual mechanism of action, demonstrate dose-dependent inhibition of complement activity, support development of KP104 for subcutaneous (SC) and intravenous (IV) administration, and indicate a favorable safety profile.’’
The Phase 2 study aims to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in patients with PNH that have not previously been treated with complement inhibitor therapies. The study is designed in two parts; the first is to assess escalating doses and varied dose intervals of KP104, while the second is to assess clinical proof of concept for treatment of PNH with KP104.
Phase 2 trials will be conducted globally including in the U.S., China, Australia, and South Korea.