Bowave Pharmaceuticals Announces First Patient Dosed in Phase II Trials of siRNA Drug BW-40202 for PNH and IgA Nephropathy

2026April 20,Argo Biopharma Co., Ltd. (hereinafter referred to as "Argo Biopharma," "the Company") announced that its investigational small interfering RNA (siRNA) drug BW-40202 has completed the first patient dosing in two Phase II clinical studies, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and immunoglobulin A nephropathy (IgA nephropathy), respectively.

Dr. Dongxu Shu, Co-founder and CEO of Argo Biopharma, stated, "The completion of the first patient dosing in these two Phase II clinical trials marks another significant step in our development of innovative complement-based therapies for PNH and IgA nephropathy. We will continue to advance the development of this therapy with the goal of providing patients with a new treatment option that offers meaningful clinical benefits as soon as possible."

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.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare acquired blood disorder characterized by complement-mediated red blood cell destruction (hemolysis), thrombosis, and bone marrow failure. It is estimated that approximately 10 to 20 people per million globally are affected by PNH.¹Patients may experience a range of symptoms, including severe anemia, thrombosis, fatigue, hemoglobinuria, chronic kidney disease, and pulmonary hypertension. Thrombosis is the most life-threatening complication of PNH, accounting for 40% to 67% of PNH-related deaths.²Significantly reducing patients' quality of life and life expectancy. siRNA therapy, with advantages such as specific targets, long-lasting effects, low dosing frequency, and safe and convenient administration, is expected to become a better treatment option for PNH patients.

About Immunoglobulin A Nephropathy (IgA Nephropathy)

Immunoglobulin A Nephropathy (IgA Nephropathy) is a chronic kidney disease characterized by the deposition of immunoglobulin A (IgA) in the glomeruli, triggering inflammatory responses and leading to progressive kidney function impairment. As the most common primary glomerulonephritis worldwide, up to 50% of patients with IgA nephropathy progress to renal failure within 10 to 20 years after diagnosis.^3-5。The disease has a wide global patient population, and currently available effective treatment options remain limited. There is an urgent need to develop innovative therapies that can effectively slow disease progression and improve long-term outcomes.

References:

1.Schrezenmeier, H., et al. (2020). Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry. Annals of Hematology, 99, 1505–1514.

2.Hill A, Kelly RJ, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 2013;121(25):4985-5105. doi:10.1182/blood-2012-09-311381.

3.Xie J, Kiryluk K, Wang W, et al. Predicting progression of IgA nephropathy: new clinical progression risk score. PLoS One 2012; 7(6): e38904.

4.Pitcher D, Braddon F, Hendry B, et al. Long-term outcomes in IgA nephropathy. Clin J Am Soc Nephrol 2023; 18: 727-38.

5.Kwon CS, Daniele P, Forsythe A, Ngai C. A systematic literature review of the epidemiology, health-related quality of life impact, and economic burden of immunoglobulin A nephropathy. J Health Econ Outcomes Res 2021; 8: 36-45.

About Argo Biopharma

Argo is a clinical-stage biotechnology company dedicated to developing a new generation of RNAi therapies to provide 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 has seven RNAi candidate drugs in clinical studies.