Phase II study of BW-40202, a small interfering RNA (siRNA) therapy targeting complement factor B (CFB), is planned to commence in January 2026.
The therapy is also being examined in a Phase I trial for IgA nephropathy, following NMPA approval for Phase I/IIa in June 2025.
Argo Biopharma co-founder and CEO Dongxu Shu said: “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.
“We are excited to initiate clinical studies and unlock the full potential of BW-40202 for PNH and other complement alternative pathway (CAP)-mediated diseases.”
BW-40202 is designed to silence CFB mRNA in the liver by harnessing RNA interference, resulting in reduced serum CFB protein and inhibited CAP activity.
Preclinical evidence demonstrated a significant and sustained reduction in serum CFB protein, strong inhibition of CAP activity, and a favourable safety profile.
Furthermore, BW-40202 exhibited robust pharmacological activity and long-lasting effects in animal models.
PNH is a rare blood disorder marked by complement-mediated red blood cell destruction, blood clot formation, and bone marrow failure.
Worldwide, approximately 10 to 20 people per million are affected. Patients often experience severe anaemia, blood clots, kidney disease, and pulmonary complications, with thrombosis causing up to 67% of PNH-related deaths.
siRNA therapeutics are seen as offering precise, durable, and manageable treatment options for such rare complement-mediated diseases.