Rezolute Announces Phase 3 sunRIZE Study Results in Congenital Hyperinsulinism

Core Insights - The Phase 3 sunRIZE study of ersodetug for congenital hyperinsulinism did not meet its primary or key secondary endpoints, showing a 45% reduction in hypoglycemia events at the highest dose compared to a 40% improvement in the placebo group, which was not statistically significant [1][2] - The study involved 63 participants across multiple countries and aimed to evaluate the efficacy and safety of ersodetug in patients experiencing hypoglycemia despite standard care [8] Study Results - The primary endpoint assessed the change in average weekly hypoglycemia events, which did not show significant improvement with ersodetug compared to placebo [2] - The key secondary endpoint, measuring average daily percent time in hypoglycemia, also did not demonstrate significant results, with a 25% reduction in time in hypoglycemia for the 10 mg/kg dose versus a 5% increase in the placebo group [2] - Dosing regimens included 5 and 10 mg/kg administered every other week for three doses, followed by every four weeks for the remaining 24-week treatment duration, achieving target drug concentrations across all age groups [3] Safety Observations - Safety data from the study were generally favorable, supporting the safe use of ersodetug in both pediatric and adult patients [4] - Two participants experienced serious hypersensitivity reactions leading to early discontinuation, but the incidence of serious allergic reactions was low compared to other biologic treatments [4] - The most common adverse event reported was hypertrichosis, which was mild and self-limiting [4] Company Statements and Future Plans - The company expressed disappointment over the study results but indicated that certain aspects warrant further investigation and a thorough evaluation to understand the outcomes better [5] - The company plans to meet with the FDA under the Breakthrough Therapy Designation to discuss next steps for the program [5] - An ongoing Phase 3 study for tumor hyperinsulinism (upLIFT) is expected to release topline results in the second half of 2026 [5] About Ersodetug - Ersodetug is a fully human monoclonal antibody designed to decrease insulin receptor over-activation, potentially treating hypoglycemia from any form of congenital or acquired hyperinsulinism [9] - The company focuses on rare diseases, particularly hypoglycemia caused by hyperinsulinism, and aims to provide meaningful treatment options for affected patients [10]