Home Tides Express | Argo Biopharma's siRNA Drug BW-40202 Achieves First Patient Dosing in Phase II Trials for Two Indications

Tides Express | Argo Biopharma's siRNA Drug BW-40202 Achieves First Patient Dosing in Phase II Trials for Two Indications

Apr 22, 2026 09:27 CST Updated 09:27
Argo

RNAi Drug Developer

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|Edited by the Content Team of Zhong肽 Biochemical
2026YearApril20DayArgo Biopharma Co., Ltd. (hereinafter referred to as "Argo Biopharma," or "the Company") announced that the first patient has been dosed in two Phase II clinical trials for its investigational small interfering RNA (siRNA) drug BW-40202, which is being developed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and immunoglobulin A nephropathy (IgA Nephropathy)。
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BW-40202 is an siRNA therapy targeting CFB mRNA. It inhibits the expression of CFB mRNA through the RNA interference (RNAi) mechanism, thereby reducing serum CFB protein levels and suppressing the activity of the complement alternative pathway (CAP). Preclinical studies have shown that BW-40202 exhibits excellent pharmacological activity, significantly and durably lowering serum CFB protein levels, effectively inhibiting CAP activity over the long term, and demonstrating favorable safety characteristics.

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare acquired blood disorder. The core pathogenesis stems from abnormal complement activation-induced hemolysis of red blood cells, accompanied by clinical manifestations such as high incidence of thrombosis and bone marrow failure. Epidemiological data show that the global prevalence of PNH is approximately 10 to 20 per million people. Patients often suffer from severe anemia, extreme fatigue, abnormal hemoglobinuria, kidney damage, and pulmonary hypertension, among other symptoms. Thromboembolism is the most life-threatening cause of mortality in this disease, accounting for 40% to 67% of PNH-related deaths, significantly reducing patients' survival time and greatly impairing their quality of life. siRNA-targeted therapy, with its precise mechanism, prolonged efficacy, reduced dosing frequency, and enhanced safety and compliance, holds promise to offer a new optimized treatment option for PNH patients.

Immunoglobulin A Nephropathy (IgA Nephropathy) is a chronic glomerular disease caused by the deposition of abnormal IgA complexes in the renal glomeruli, continuously triggering local inflammatory damage, which leads to progressive renal function decline. As the most prevalent primary glomerular disease globally, it carries a severe prognosis, with more than half of patients developing end-stage renal failure within 10 to 20 years of diagnosis. The current global patient population is substantial, and existing clinical intervention options are very limited. The industry urgently needs innovative therapeutic drugs capable of halting disease progression and improving long-term patient outcomes.

AboutArgo

Argo Biopharma is a clinical-stage biotechnology company dedicated to developing a new generation of RNAi therapies, providing better treatment options for patients worldwide. The company leverages its liver-targeting and extrahepatic delivery technology platforms to build a diversified RNAi drug R&D pipeline covering cardiometabolic diseases, specialty disorders, viral infections, central nervous system diseases, and rare diseases. Currently, Argo Biopharma has seven RNAi candidate drugs in clinical studies.


Source:

1.Argo