Efficacy and safety of NTQ5082 monotherapy in complement-inhibitor-Naïve patients with paroxysmal nocturnal hemoglobinuria: A proof-of-concept, multicenter, randomized, open-label phase 2 study
Randomized trial demonstrated improvement in hemolysis and hemoglobin in patients with paroxysmal nocturnal hemoglobinuria, suggesting NTQ5082 is a promising complement factor B inhibitor.
Key Points
The study aimed to assess the efficacy and safety of NTQ5082 monotherapy in patients with paroxysmal nocturnal hemoglobinuria.
Multicenter, open-label, phase 2 study conducted in China
Included adult patients with complement inhibitor-naïve PNH
Patients were randomized to receive NTQ5082 at varying dosages for 12 weeks
All patients achieved an increase in Hb concentrations by ≥20 g/L compared to baseline
Treatment led to significant reductions in LDH levels by 85.9% in cohort A and 82.2% in cohort B
Most patients experienced clinically meaningful improvements in their FACIT-Fatigue scores