Updates provided on navenibart, a potential HAE medicine currently being trialled in patients

A series of updates on navenibart, which is currently undergoing testing amongst HAE patients, were provided by the pharmaceutical company Astria Therapeutics. The key updates shared with HAEi were:

  • The ALPHA-ORBIT pivotal Phase 3 trial of navenibart in people with hereditary angioedema (HAE) is enrolling patients, with top-line results expected in early 2027. The ALPHA-ORBIT trial is a global, randomized, double-blind, placebo-controlled Phase 3 clinical trial to evaluate the efficacy and safety of navenibart over 6 months and includes both every 3-month (Q3M) and every 6-month (Q6M) treatment arms.
  • The navenibart Phase 3 program consists of the ALPHA-ORBIT Phase 3 trial and the ORBIT-EXPANSE long-term trial, which are designed to support registration globally.
  • Positive final top-line results from target enrollment in the Phase 1b/2 ALPHA-STAR trial of navenibart were announced in December 2024. These showed rapid onset of robust and durable efficacy, favorable safety and tolerability, and pharmacokinetics and pharmacodynamics consistent with sustained plasma kallikrein inhibition for both Q3M and Q6M administration. Final results included reduction in mean monthly attack rate of 90-95% and up to a 67% attack-free rate over 6 months.
  • All of the 16 target enrollment patients from ALPHA-STAR have entered the ALPHA-SOLAR long-term open-label trial. Initial safety and efficacy data from ALPHA-SOLAR, with long-term Q3M and Q6M administration, are expected mid-2025.

Jill C Milne PhD, Chief Executive Officer at Astria Therapeutics, said: “As a late-stage clinical company, we are focused on delivering on navenibart, a therapy with a trusted mechanism and modality, best-in-class profile, and the potential to change the way that people live with HAE. Our goal is to ensure that navenibart reaches its full potential as the market-leading HAE therapy. We are thrilled with patient and physician enthusiasm for the program as we enroll patients in our trial. With STAR-0310, we anticipate early proof-of-concept results from the Phase 1a trial in Q3, which we expect to be informative on differentiation regarding efficacy, safety, and low treatment burden and next steps.”

(Source: Astria)