Bowave Pharmaceuticals Receives NMPA Approval for Phase II Clinical Trial of BW-40202 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Sep 16, 2025 08:00 CST Updated 08:00

Argo

RNAi Drug Developer

Argo Biopharma's New Drug BW-40202 for Treating PNH Receives Approval for Phase II Clinical Trial from China NMPA

Shanghai, China and Boston, USA — September 15, 2025— Argo Biopharma Co., Ltd. ("Argo Biopharma," "the Company") announced today that its investigational small interfering RNA (siRNA) drug BW-40202 has received clinical trial approval from the National Medical Products Administration (NMPA) of China, granting permission to initiate a Phase II clinical study for Paroxysmal Nocturnal Hemoglobinuria (PNH). This study is expected to commence in January 2026. BW-40202 is a drug targeting Complement Factor B (CFB) for the treatment of PNH and other complement-mediated diseases. BW-40202 is also being evaluated in clinical trials for IgA Nephropathy (IgAN), with the company having received NMPA approval in June 2025 to conduct a Phase I/IIa clinical trial for IgAN.

Dr. Dongxu Shu, Co-founder and CEO of Argo Biopharma, stated, "The approval of the BW-40202 new drug clinical trial application marks a key milestone in our mission to redefine treatment options for PNH patients. This not only validates the scientific value of our innovative strategy targeting the complement system but also represents an important step toward providing patients with a new targeted therapeutic option. We look forward to initiating clinical studies to fully realize the potential of this candidate drug."

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 impaired bone marrow function. It is estimated that approximately 10 to 20 people per million globally are affected by PNH¹. Patients may experience a range of symptoms, including severe anemia, thrombosis, hemoglobinuria, chronic kidney disease, and pulmonary hypertension. Thrombosis remains the most life-threatening complication, accounting for 40% to 67% of PNH-related deaths, significantly reducing patients' quality of life and life expectancy. siRNA therapy, with its specific targets, long-lasting effects, reduced dosing frequency, and enhanced safety and convenience, holds promise as a superior treatment option for PNH patients.

About Argo Biopharma

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 diseases, viral infections, central nervous system disorders, and rare diseases. Currently, Argo Biopharma has six RNAi candidate drugs in clinical studies.

For more information, please visitwww.argobiopharma.com。

1. Schrezenmeier, H. et al. Baseline Clinical Characteristics and Disease Burden in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): An Updated Analysis from the International PNH Registry. Annals of Hematology (2020). *99*: 1505-1514.