ADARx Pharmaceuticals Presents Positive Onvuzosiran Data

ADARx Pharmaceuticals, a late-stage clinical biotechnology company, announced positive Phase 1/2 clinical data for its investigational small interfering RNA (siRNA) therapeutic candidate, Onvuzosiran (ADX-324), for the treatment of hereditary angioedema (HAE). The data, presented at the 2026 Annual Meeting of the American Academy of Allergy, Asthma & Immunology (AAAAI), showed significant and durable dose-dependent reductions in plasma kallikrein levels with a favorable safety profile. The 300 mg dose achieved a 93% reduction at its lowest point, maintaining an 80% reduction through Day 169. Patients in the Phase 2a study experienced no HAE attacks when kallikrein levels were reduced by 80%, supporting the potential for twice-yearly dosing. The company also highlighted the design of its ongoing Phase 3 STOP-HAE clinical trial, which is a randomized, double-blind, placebo-controlled study enrolling approximately 90 patients. Onvuzosiran has received Orphan Drug Designation from the United States===Food and Drug Administration (FDA) for HAE.