ADARx Pharmaceuticals announced it will present initial Phase 1 clinical data for ADX-038, an investigational small interfering RNA (siRNA) therapeutic candidate, at the American Society of Nephrology (ASN) Kidney Week meeting scheduled for November 6-9, 2025, in Houston, Texas. The late-stage clinical biotechnology company is developing the therapy for the treatment of complement factor mediated diseases.
The poster presentation, titled "Interim Phase 1 Study Results of ADX-038: A Novel siRNA Against Complement Factor B, and Next Steps in IgAN and C3 Glomerulopathy (C3G)," will be presented on Friday, November 7 from 10:00 a.m. to 12:00 p.m. CST under poster number FR-PO0832.
Targeting the Complement Pathway
ADX-038 is designed to selectively cleave complement factor B (CFB), which ADARx describes as a validated target in the complement pathway. The complement system serves as a key component of the immune system that enhances the activity of both adaptive and innate immune responses. According to the company, dysregulation of this system has been implicated in a broad range of autoimmune and inflammatory diseases.
This mechanism of action positions ADX-038 as what the company calls a "pipeline-in-a-product," potentially addressing multiple indications through a single therapeutic approach. The company has initiated multiple Phase 2 clinical trials to evaluate the safety and efficacy of ADX-038 in patients with various complement-mediated diseases.
Broad Clinical Development Program
The Phase 2 program encompasses several complement-mediated conditions, including renal diseases such as immunoglobulin A nephropathy (IgAN) and complement 3 glomerulopathy (C3G). Additionally, the therapy is being evaluated for geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and paroxysmal nocturnal hemoglobinuria (PNH).
Company Profile and Strategic Partnerships
ADARx Pharmaceuticals positions itself as a late-stage clinical biotechnology company focused on transforming cutting-edge science into next-generation RNA medicines across multiple therapeutic areas. The company has developed proprietary technology to control the expression of specific disease drivers with highly selective RNA targeted therapies.
Beyond complement-mediated diseases, ADARx is developing product candidates for genetic, cardiovascular, thrombotic, central nervous system, and metabolic diseases, including obesity. The company has also entered into a collaboration and license option agreement with AbbVie to develop siRNA therapeutics across multiple disease areas, including neuroscience, immunology, and oncology.
