Beijing, China — Argo Biopharmaceutical Co., Ltd. (Argo Biopharma), a clinical-stage siRNA therapeutics company, announced that China’s National Medical Products Administration (NMPA) has granted Investigational New Drug (IND) approval for a Phase II trial of BW-40202. The therapy targets complement factor B (CFB) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and other complement-mediated diseases.
The Phase II study is expected to begin in January 2026. BW-40202 is also being tested in a Phase I trial for IgA Nephropathy (IgAN) following a June 2025 IND clearance.
CEO Statement
“The IND approval for BW-40202 is an important milestone in our mission to redefine treatment for patients with PNH. This achievement underscores our innovative approach in targeting the complement system and moves us one step closer to delivering a potential new, targeted therapy to patients who need more treatment options.”
- Dr. Dongxu Shu, co-founder and CEO of Argo Biopharma.
About BW-40202
- Therapeutic class: siRNA drug targeting CFB mRNA in the liver.
- Mechanism: Uses RNA interference (RNAi) to suppress CFB expression → reduces serum CFB protein levels → inhibits complement alternative pathway (CAP) activity.
- Preclinical findings:
- Strong purity and stability.
- Durable suppression of serum CFB protein.
- Effective CAP inhibition.
- Favorable safety and pharmacological profile.
Understanding PNH
- Prevalence: Affects 10–20 people per million globally.
- Key symptoms: Severe anemia, hemoglobinuria, blood clots, kidney disease, pulmonary hypertension.
- Risks: Thrombosis is the most life-threatening complication, linked to 40–67% of PNH-related deaths.
- Why siRNA matters: Offers precise targeting, long-lasting efficacy, fewer doses, and improved safety, potentially raising treatment standards in complement-driven diseases.
About Argo Biopharma
- Clinical-stage biotech focused on next-generation RNAi therapies.
- Pipeline covers cardiovascular, viral, metabolic, and rare diseases.
- Currently advancing six RNAi candidates in clinical development.
