ADARx Pharmaceuticals dose first patient in phase 3 STOP-HAE clinical trial and orphan drug designation for ADX-324

ADARx Pharmaceuticals, Inc. (ADARx), a late-stage clinical biotechnology company developing next-generation RNA therapeutics, today announced that the first patient has been dosed in its Phase 3 STOP-HAE clinical trial evaluating ADX-324 in patients with hereditary angioedema (HAE). Additionally, ADARx announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ADX-324 for the treatment of patients with HAE.

Despite advances in prophylactic therapy, most HAE patients remain at risk of breakthrough attacks and do not achieve sustained, long-term attack-free control. In addition, the currently approved prophylaxis treatments require monthly or more frequent injections. We believe the FDA’s Orphan Drug Designation for ADX-324 underscores the continued need for additional treatment options and highlights the potential of ADX-324 to provide extended attack-free periods with substantially reduced dosing frequency. Dosing the first patient in our ADX-324 Phase 3 clinical trial is a significant milestone for ADARx and we look forward to advancing this program for the HAE community.

said Zhen Li, President and Chief Executive Officer of ADARx. 

The Phase 3 STOP-HAE clinical trial of ADX-324 is a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy of ADX-324 in preventing HAE attacks in adults with Type I and Type II HAE. The trial will also assess safety, pharmacokinetics (PK), pharmacodynamics (PD), and patient reported health-related quality of life (HRQoL). Approximately 90 patients will be enrolled and randomized to receive either ADX-324 300 mg every 6 months, ADX-324 240 mg every 3 months, or placebo during the study. Patients completing the trial will be eligible to enroll in a long-term-open-label extension study.

Enrollment for this Phase 3 program follows positive Phase 1/2 data demonstrating that ADX-324 achieved significant and durable suppression of prekallikrein (PKK) protein levels with a favorable safety profile.