Core Insights - The Phase III sunRIZE study for ersodetug in patients with congenital hyperinsulinism (HI) did not achieve statistical significance for its primary and secondary endpoints, which has caused disappointment within the company [3] - The company acknowledges the significant unmet need for new therapies in the congenital HI patient population and expresses gratitude for the participation of patients, families, and investigators in the study [2] Study Results - The study aimed to demonstrate the efficacy of ersodetug compared to placebo in an outpatient setting, where glucose levels were both the primary endpoint and a vital sign [3] - Despite the belief in the drug's potential activity, the results did not show a significant difference from the placebo group [3]

Core Insights - Rezolute, Inc. is focused on treating hypoglycemia caused by hyperinsulinism and has announced a late-breaking presentation at ENDO 2025 regarding its Phase 3 study of ersodetug [1][2] Group 1: Study Details - The Phase 3 sunRIZE study is a multi-center, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of ersodetug in patients aged 3 months to 45 years with congenital hyperinsulinism [3] - The study aims to enroll approximately 56 participants across more than a dozen countries, including the U.S., and has completed enrollment [4] - Key efficacy endpoints include the change from baseline in the average number of hypoglycemia events per week and the average percent time in hypoglycemia over six months of treatment [4] Group 2: Drug Information - Ersodetug is a fully human IgG2 monoclonal antibody that binds allosterically to the insulin receptor, aiming to decrease receptor over-activation and improve hypoglycemia [5] - The drug has shown substantial benefit in clinical trials and real-world use for treating both congenital and tumor-related hyperinsulinism [7] Group 3: Medical Context - Congenital hyperinsulinism is the most common cause of recurrent hypoglycemia in children, often presenting within the first month of life and can lead to severe complications if not managed properly [6] - More than half of children with congenital hyperinsulinism require long-term medical treatment for hypoglycemia that is not adequately addressed by existing therapies [6]

Core Viewpoint - The Independent Data Monitoring Committee (DMC) has recommended the continuation of the Phase 3 sunRIZE trial without increasing the sample size, indicating confidence in the study's design and initial assumptions [1][2][3] Company Overview - Rezolute, Inc. is a late-stage biopharmaceutical company focused on developing therapies for rare diseases, particularly congenital hyperinsulinism (HI) [2][7] - The company's lead product, ersodetug, is a fully human monoclonal antibody aimed at treating hypoglycemia caused by HI [5][8] Study Details - The Phase 3 sunRIZE study is a multi-center, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of ersodetug in patients with congenital HI [4] - The study is expected to enroll up to 56 participants aged 3 months to 45 years across more than a dozen countries, with enrollment anticipated to be completed by May 2025 [4][1] Clinical Insights - The DMC's interim analysis was based on a pre-specified evaluation of hypoglycemia events after approximately half of the enrolled patients completed primary assessments, confirming no need for a sample size increase [3] - The topline data from the study is expected to be announced in December 2025, with U.S. clinical sites actively enrolling patients [1][3] Treatment Potential - Ersodetug has the potential to be a best-in-class treatment option for individuals living with hyperinsulinism, as it acts downstream from the pancreas to improve hypoglycemia [5][3] - Congenital HI is a significant cause of recurrent hypoglycemia in children, and effective treatment options are limited, highlighting the importance of the ongoing study [6]