#China #Shanghai #PNH #Argo Biopharma #BW-40202

Argo Biopharma Receives IND Approval for Phase II Clinical Trial of BW-40202

Argo Biopharmaceutical Co., Ltd. (Argo Biopharma), a company specializing in small interfering RNA (siRNA) therapies, has achieved a significant milestone by obtaining Investigational New Drug (IND) approval from the National Medical Products Administration (NMPA) of China. This approval is for the Phase II clinical trial of their investigational drug, BW-40202, designed to target complement factor B (CFB) for patients suffering from paroxysmal nocturnal hemoglobinuria (PNH), as well as other diseases influenced by the complement system.

The anticipated start date for the clinical trial is January 2026, marking a critical step in the development of a targeted therapy for individuals impacted by this severe condition. Dr. Dongxu Shu, the co-founder and CEO of Argo Biopharma, expressed enthusiasm about this achievement, stating, "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."

BW-40202 represents a novel approach in treating PNH by directly targeting CFB mRNA in the liver. The drug employs an RNA interference (RNAi) mechanism, which effectively suppresses CFB expression, leading to a decrease in serum CFB protein levels and the inhibition of complement alternative pathway (CAP) activity. During preclinical evaluations, BW-40202 exhibited excellent stability and purity, along with significant and sustained suppression of serum CFB protein, demonstrating effective inhibition of CAP activity coupled with a favorable safety profile.

Understanding PNH and Its Complications

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by the destruction of red blood cells, blood clot formation, and dysfunctional bone marrow production. Approximately 10 to 20 individuals per million globally are estimated to be living with this condition. Patients frequently experience severe anemia, blood clots, hemoglobinuria, chronic kidney issues, and pulmonary hypertension, leading to a significant reduction in life expectancy and quality of life. The most critical threat remains thrombosis, accounting for 40% to 67% of deaths related to PNH.

The advent of siRNA therapies provides hope through precise targeting, longer-lasting efficacy, fewer required doses, and improved safety for patients. These options might lead to more effective treatments for managing PNH and other CAP-mediated disorders.

Argo Biopharma's Vision and Future Plans

Argo Biopharma is committed to paving the way for the next generation of RNAi therapeutics aimed at providing enhanced treatment options on a global scale. The company has a diverse pipeline, incorporating six RNAi candidates presently in clinical development across a spectrum of diseases, including cardiovascular issues, viral infections, metabolic conditions, and various rare diseases.

As their research progresses with BW-40202, Argo Biopharma aims to unlock its full potential as a groundbreaking treatment for PNH patients and beyond. The firm remains steadfast in their mission to deliver innovative solutions that can transform the lives of patients living with serious health conditions. For additional information regarding the company's initiatives and research, interested parties can visit www.argobiopharma.com.

In conclusion, these advancements in siRNA therapy showcase the dynamic potential of biotechnology in addressing unmet medical needs and offer a promise of hope for patients affected by PNH and similar disorders.