Argo planning 2026 trial in China to test PNH treatment BW-40202
Chinese regulators give green light to Phase 2 study of injection therapy
Chinese regulators have given the green light to Argo Biopharmaceutical to launch a Phase 2 clinical trial testing its treatment candidate BW-40202 in people with paroxysmal nocturnal hemoglobinuria (PNH).
The trial is expected to begin in January 2026, Argo said in a company press release announcing the decision by the National Medical Products Administration of China.
Dongxu Shu, PhD, Argo’s cofounder and CEO, called the trial clearance “an important milestone in our mission to redefine treatment for patients with PNH.”
According to Shu, “this achievement underscores our innovative approach … and moves us one step closer to delivering a potential new, targeted therapy to patients who need more treatment options.”
PNH is a rare disease in which the immune system mistakenly attacks and destroys the body’s own healthy red blood cells, leading to bloody urine, low blood cell counts, fatigue, and other symptoms. Uncontrolled activation of the immune system’s complement cascade is central to these attacks.
PNH treatment designed to reduce blood cell destruction, ease symptoms
BW-40202 aims to specifically inhibit a branch of the complement system known as the complement alternative pathway (CAP). It’s designed to do so by reducing the levels of complement factor B (CFB), a key complement protein.
Belonging to a class of therapies called small-interfering RNAs, or siRNAs, BW-40202 prevents CFB from being made by silencing the activity of the gene that encodes its production. It does this by binding to and causing the degradation of CFB messenger RNA, a template molecule that’s needed to transform the information in a gene’s DNA sequence into a working protein. In turn, that should inhibit CAP activation, reducing red blood cell destruction and easing disease symptoms.
The treatment is specifically targeted to the liver, where complement proteins are primarily produced, and is given via subcutaneous, or under-the-skin, injections.
We are excited to initiate clinical studies and unlock the full potential of BW-40202 for PNH.
In preclinical studies, BW-40202 led to lasting suppression of CFB and inhibition of the alternative complement pathway. It also had a favorable safety profile, according to Argo.
A first in-human study was launched this year to assess the safety, tolerability, and pharmacological properties of BW-40202 in healthy adults. Dosing in the Phase 1 trial (NCT06917482) was started at a site in Australia this summer. Regulators later cleared the trial’s expansion into Chinese clinical sites.
Data from this Phase 1 study may inform the upcoming Phase 2 trial in PNH. The company did not provide details on the planned PNH study, but did indicate that it should begin early next year.
Beyond PNH, Argo is also developing BW-40202 for other complement-mediated diseases.
Argo reported that Chinese regulators have also cleared a Phase 1/2a trial testing BW-40202 in people with IgA nephropathy, an autoimmune kidney disease in which the complement system is overly active.
“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,” Shu said.
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