Qirui Group’s First Innovative Drug, MY008211A Tablets (Lanocapam Hydrochloride Tablets), Successfully Achieves the Primary Efficacy Endpoint in Phase III Clinical Trials

February 13 On [date], the MY008211A tablet project team at Qirui Group announced that the company’s independently developed small-molecule complement factor B (CFB) inhibitor, MY008211A tablet, has entered the Phase III clinical trial (MY008211A-PNH-3-02) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The primary efficacy endpoint was successfully achieved. The research results show that, Compared with the positive control drug eculizumab, MY008211A The study demonstrated superiority in both the primary endpoint and multiple secondary endpoints. 。

• The MY008211A tablet treatment group totaled 50.0% The proportion of subjects achieving normal hemoglobin levels (Hb ≥ 120 g/L) was significantly higher than in the eculizumab-treated group, at 9.1%.
• MY008211A The study demonstrated significant efficacy across all secondary endpoints, including improvement in anemia, control of hemolysis, elimination of transfusion dependence, and enhancement of quality of life, with overall efficacy superior to that of eculizumab.
• MY008211A The formulation demonstrated good safety and tolerability in clinical trials, with overall risks remaining manageable.

This study was led by Professor Zhang Fengkui of the Tianjin Institute of Hematology and Professor Han Bing of Peking Union Medical College Hospital, in collaboration with seven PNH centers nationwide. The study employed a randomized, open-label, positive-control, parallel-group design, enrolling a total of 67 PNH patients who had not previously received complement inhibitor therapy. Patients were randomly assigned in a 1:1 ratio to the experimental group and the control group, receiving either oral MY008211A tablets (twice daily, 400 mg) or intravenous eculizumab, respectively, for a duration of 24 weeks. The primary objective was to evaluate the efficacy and safety of MY008211A tablets compared with eculizumab.

The study demonstrates that MY008211A tablets exhibit significant therapeutic superiority in the treatment of PNH. For the primary endpoint of hemoglobin normalization, 50.0% of patients in the MY008211A tablet group achieved normal hemoglobin levels (Hb ≥ 120 g/L), which was substantially higher than the 9.1% observed in the eculizumab group. Moreover, MY008211A tablets showed clear superiority across several secondary endpoints, including improvement in anemia, control of hemolysis, achievement of transfusion independence, and enhancement of quality of life. Subgroup analyses further indicated that a higher proportion of patients with more severe baseline disease also achieved hemoglobin normalization. In terms of safety, MY008211A tablets were well tolerated, with overall risks remaining manageable. During the trial, most adverse events were graded as mild to moderate (Grade 1–2), no cases of encapsulated bacterial infections were reported, and the risk of breakthrough hemolysis was controllable.