China-based rare disease firm CANbridge Pharmaceuticals Inc., (HKG: 1228) has published the preliminary results of a Phase Ib clinical study for its pipeline drug candidate omoprubart (CAN106), a long-acting monoclonal antibody (mAb) targeting C5 of the complement system. The drug candidate is under development as a potential treatment for paroxysmal nocturnal hemoglobinuria (PNH) in China.
Study Design and Patient Enrollment
The 26-week, multi-center, open-label, dosage escalation study enrolled 16 PNH patients not previously treated with complement inhibitors. Omoprubart was administered intravenously every 4 weeks at 3 different maintenance doses. The study aimed to assess the drug’s impact on lactate dehydrogenase (LDH) levels, a marker indicating the degree of hemolysis in PNH sufferers.
Dose-Dependent Reduction in LDH Levels
The study demonstrated a rapid reduction in LDH levels, with average reductions below the baseline ranging from 49% to 81% respectively in a dose-dependent manner. The percentage of subjects reaching an LDH level of less than 1.5 times the upper limit of normal, the therapeutic goal for hemolysis inhibition with an anti-C5 antibody, at least once during the study, was 25% in Cohort 1, 50% in Cohort 2, and 88% in Cohort 3.
Hemoglobin Level Increases Across All Study Cohorts
Hemoglobin levels increased across all study cohorts, with mean increases from baseline of approximately 2 g/dL in Cohorts 1 and 2 at Week 26, and 1 g/dL in Cohort 3 at Week 13. All subjects in Cohort 1 have been treated with omoprubart for over one year, with a mean hemoglobin increase of approximately 4 g/dL from baseline. This is significant for patients with PNH who often require frequent blood transfusions to treat severe anemia due to hemolysis.
Research Partnerships and Global Rights
Omoprubart emerged from research partnerships between CANbridge with Wuxi Biologics (signed in 2019) and Privus Biologics (signed in 2020). CANbridge holds exclusive development and commercialization rights to the molecule globally and initiated the ongoing Phase Ib/II study for the drug in PNH in March 2022.-Fineline Info & Tech
