ADARx Pharmaceuticals Presents Onvuzosiran Data Supporting Potential for Sustained Attack-Free Rates with Reduced Treatment Burden in Patients with HAE
– Onvuzosiran achieved significant and durable reductions of plasma kallikrein levels with a favorable safety profile in Phase 1/2 –
– Positive results support ongoing Phase 3 clinical trial –
Excerpt from the Press Release:
SAN DIEGO, Feb. 27, 2026 (GLOBE NEWSWIRE) — ADARx Pharmaceuticals, Inc. (ADARx), a late-stage clinical biotechnology company developing next-generation RNA therapeutics, today announced Phase 1/2 clinical data for onvuzosiran (ADX-324), an investigational small interfering RNA (siRNA) therapeutic candidate being developed for the treatment of hereditary angioedema (HAE). The Company also highlighted the clinical trial design of its ongoing Phase 3 STOP-HAE study. A poster presentation is being given at the 2026 Annual Meeting of the American Academy of Allergy, Asthma & Immunology (AAAAI) being held February 27 – March 2, 2026, in Philadelphia, PA.
In the Phase 1 study, onvuzosiran demonstrated deep and durable dose-dependent reductions in plasma kallikrein levels. The 300 mg dose showed a 93% reduction at the lowest point, with 80% reduction maintained through Day 169. In the ongoing Phase 2a study, patients achieved similarly deep kallikrein level reductions. Notably, HAE patients experienced no attacks when kallikrein levels were reduced by 80%, further supporting the potential of twice-yearly dosing to achieve high attack-free rates. In the Phase 1/2 study, onvuzosiran was well tolerated, and no serious adverse events were reported, nor were there any treatment-related discontinuations.
“While progress has been made in prophylactic HAE therapy, there remains significant need to further reduce attack frequency and ease the treatment burden that both impact quality of life for patients,” said Dr. Donald Fong, Chief Medical Officer of ADARx. “Plasma kallikrein is a clinically validated target in HAE, and onvuzosiran has demonstrated deep, sustained inhibition, a favorable safety profile, and the potential for less frequent dosing.
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