ADARx Pharmaceuticals to Present Phase 1/2 Data for Onvuzosiran siRNA Therapy at AAAAI 2026 Meeting
Key Insights
ADARx Pharmaceuticals (search) will present Phase 1/2 clinical data and Phase 3 design for onvuzosiran (search) (ADX-324), an investigational siRNA therapy for hereditary angioedema (search), at the 2026 AAAAI Annual Meeting.
Onvuzosiran (search) targets prekallikrein (search) at the mRNA level to reduce bradykinin production and prevent HAE (search) attacks with semi-annual subcutaneous dosing.
The therapy is currently being evaluated in the Phase 3 STOP-HAE (search) clinical trial and has received FDA Orphan Drug Designation for HAE treatment.
ADARx Pharmaceuticals (search) announced it will present Phase 1/2 clinical data and Phase 3 design for onvuzosiran (search) (ADX-324), an investigational small interfering RNA (siRNA) therapeutic candidate for hereditary angioedema (search) (HAE (search)), at the 2026 Annual Meeting of the American Academy of Allergy, Asthma & Immunology (AAAAI). The poster presentation is scheduled for February 27, 2026, from 2:45-3:45 pm EST at the Pennsylvania Convention Center in Philadelphia.
Novel siRNA Approach Targets Prekallikrein Pathway
Onvuzosiran (search) represents a next-generation RNA therapeutic designed to inhibit prekallikrein (search) (PKK (search)) generation at the mRNA level. The therapy aims to reduce plasma PKK production, thereby preventing bradykinin generation and potentially preventing HAE (search) attacks. The investigational treatment is administered via semi-annual subcutaneous injection, offering a less frequent dosing regimen compared to current prophylactic options.
HAE (search) is a rare genetic disorder characterized by recurrent, unpredictable attacks of swelling that can be painful, disabling, and life-threatening. These attacks result from dysregulation of the kallikrein-kinin system (KKS), which regulates blood pressure, inflammation, coagulation and pain. In HAE patients, a dysfunctional KKS leads to excessive release of bradykinin, a potent vasodilator that causes the characteristic swelling attacks.
Enhanced Therapeutic Profile Expected
According to ADARx, onvuzosiran (search) is expected to decrease PKK (search) to a greater degree compared to currently approved prophylactic treatments. This enhanced reduction is anticipated to offer greater and more durable control of kallikrein activity, potentially resulting in a higher proportion of patients remaining attack-free with the less frequent dosing schedule.
The therapy targets prekallikrein (search), a critical protein in the plasma kallikrein pathway that activates kallikrein, which produces bradykinin when present. By intervening at the mRNA level, onvuzosiran (search) aims to prevent the cascade that leads to HAE (search) attacks.
Phase 3 Trial and Regulatory Status
Onvuzosiran (search) is currently being evaluated in the Phase 3 STOP-HAE (search) clinical trial. The investigational therapy has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with HAE, recognizing both the rarity of the condition and the potential therapeutic benefit of the treatment.
ADARx Pharmaceuticals (search) positions itself as a late-stage biotechnology company dedicated to developing next-generation RNA medicines across multiple therapeutic areas. The company has developed technology to control the expression of specific disease drivers with highly selective RNA targeted therapies, focusing on delivering treatments for patients with urgent unmet medical needs.