ADARX PHARMACEUTICALS, INC. ADRX.US

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company focused on the discovery, development, and clinical advancement of RNA-targeted therapeutics using small interfering RNA (siRNA) and related technologies.

The company's lead candidate is ADX-324 (also known as Onvuzosiran), a long-acting siRNA therapy designed to inhibit prekallikrein (PKK) expression to prevent attacks in hereditary angioedema (HAE).

ADX-324 has progressed from Phase 1/2 clinical studies to a Phase 3 trial called STOP-HAE, where it is being evaluated in adults with Type I and II HAE for prophylactic treatment under dosing regimens that may require administration every three to six months.

The U.S. Food and Drug Administration has granted Orphan Drug Designation for ADX-324 for HAE. In addition to ADX-324, ADARx is developing ADX-038, an siRNA therapeutic targeting complement factor B (CFB) for treatment of diseases driven by alternative pathway activation of the complement system.

Indications under investigation include immunoglobulin A nephropathy (IgAN), complement 3 glomerulopathy (C3G), geographic atrophy (GA) secondary to age-related macular degeneration, and paroxysmal nocturnal hemoglobinuria (PNH).

Phase 1 data indicate that a single subcutaneous dose of ADX-038 delivered near-complete suppression of alternative pathway activity for up to six months while sparing classical pathway function, with ongoing Phase 2 trials assessing efficacy and safety in patients.

The company also has extrahepatic discovery programs including ADX-077 for obesity and ADX-199 for neurodegeneration. ADARx is headquartered in San Diego, California. Its technology platform includes proprietary approaches for oligonucleotide delivery (e.g., PLR™, "Preeminent Liver RNA") and for sequence-specific optimization (SPE™) to improve durability, selectivity and dosing convenience.

The company operates in multiple therapeutic areas beyond rare genetic disorders, including complement-mediated, cardiovascular, immunological, metabolic, and neurodegenerative diseases. All product candidates are internally developed and largely wholly owned, though the company has entered into at least one collaboration with AbbVie to jointly pursue siRNA therapeutics across certain disease areas.