Rezolute Receives Positive Interim Analysis for Phase 3 sunRIZE Study on Ersodetug in Congenital Hyperinsulinism

Rezolute, Inc., a biopharmaceutical company concentrated on developing innovative treatments for rare diseases, has announced significant progress in its Phase 3 sunRIZE trial. The Independent Data Monitoring Committee (DMC) has recommended that the study proceed as designed, without the need to increase the number of participants. This recommendation suggests confidence in the trial's design for evaluating ersodetug, a promising treatment for congenital hyperinsulinism (HI).

The sunRIZE trial is a critical study targeting those with congenital HI, a condition characterized by recurrent hypoglycemia starting in infancy, which can lead to serious complications if not properly managed. This trial aims to assess the efficacy and safety of ersodetug, a fully human monoclonal antibody that mitigates receptor over-activation by insulin. This mechanism is particularly crucial as it operates downstream from the pancreas, potentially offering a treatment for various forms of HI.

Brian Roberts, M.D., the Chief Medical Officer at Rezolute, expressed enthusiasm over the DMC’s supportive recommendation. Although the company remains blinded to the interim data and specific statistical results, the DMC's endorsement is seen as a validation of the study's initial assumptions, based on prior positive outcomes from both the Phase 2 RIZE study and real-world observations. The global study is currently enrolling participants and is expected to complete enrollment by May 2025, with top-line results anticipated by December 2025.

The interim analysis conducted by the DMC involved a careful evaluation of the primary endpoint, namely hypoglycemia events, after about half of the participants had finished their primary assessments. This review was pivotal to determine whether the study might be futile or required a larger sample size to ensure robust final outcomes. The fact that the study will continue as initially planned underscores the potential effectiveness of ersodetug.

Ersodetug's mechanism involves allosterically binding to the insulin receptor, which helps manage hypoglycemia by reducing receptor over-activation. This is especially significant in the context of hyperinsulinism, where traditional treatments often fall short. The goal of the sunRIZE study is to enroll up to 56 participants across various countries, with eligible participants ranging from 3 months to 45 years old.

Congenital HI is the most prevalent cause of persistent hypoglycemia in children, and if untreated, it can lead to severe brain injuries, including developmental issues and seizures. While some cases require surgical intervention, many patients continue to rely on long-term medical therapies that do not fully address the hypoglycemia. Rezolute's development of ersodetug offers hope for a more effective and comprehensive treatment option.

Rezolute's commitment to addressing rare diseases extends beyond clinical trials. The company actively engages with real-world data and expanded access programs to ensure their therapies meet the urgent needs of patients. With the sunRIZE trial advancing as planned, Rezolute remains optimistic about the potential of ersodetug to become a leading treatment for those suffering from hyperinsulinism.

As the study progresses, Rezolute continues to advance its mission of providing transformative treatments for conditions with significant unmet needs. The ongoing support and recommendations from the DMC play a crucial role in this pursuit, allowing the company to maintain its focus on delivering innovative healthcare solutions.