FDA Grants Breakthrough Therapy Status to Rezolute's Ersodetug for Congenital Hyperinsulinism Hypoglycemia

Rezolute, Inc., a biopharmaceutical company specializing in rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to their drug candidate, ersodetug (RZ358), intended for treating hypoglycemia associated with congenital hyperinsulinism (HI). This designation is a significant achievement, aimed at accelerating the development and review process of treatments that promise considerable improvements over existing therapies for severe conditions.

The FDA's recognition of ersodetug is primarily based on the promising data from the Phase 2b RIZE study. This clinical trial illustrated that ersodetug effectively improved hypoglycemia in participants by 75% or more without causing significant hyperglycemia. This underscores the potential impact of ersodetug in the medical landscape, particularly for individuals with hyperinsulinism.

Nevan Charles Elam, CEO and Founder of Rezolute, highlighted 2024 as a pivotal year for the company, marked by significant clinical strides and advancements towards addressing all forms of hyperinsulinism. He emphasized the Breakthrough Therapy Designation as evidence of ersodetug's potential to help patients significantly. The company is focusing on completing the sunRIZE study and preparing for a Phase 3 trial for tumor HI this year.

Rezolute has made considerable clinical progress, with the FDA lifting partial clinical holds on ersodetug, allowing the inclusion of U.S. clinical sites in the ongoing global Phase 3 sunRIZE study. This study targets patients aged three months and older with congenital HI. The results are anticipated in the latter half of this year, and Rezolute has also received the Innovation Passport Designation from the U.K.'s Innovative License and Access Pathway (ILAP) for treating hypoglycemia due to congenital HI.

In addition to congenital HI, Rezolute is exploring the use of ersodetug for treating hypoglycemia due to non-islet cell tumors (NICTs) and islet cell tumors, such as insulinomas. This research potentially expands the patient population for ersodetug, more than doubling it. The company has received FDA clearance for an investigational new drug (IND) application for a Phase 3 registrational study on hypoglycemia due to tumor HI, with the study set to begin in early 2025 and results expected in late 2026. The FDA has also granted Orphan Drug Designation to ersodetug for treating tumor HI, with over ten patients already benefiting from the Expanded Access Program.

Financially, Rezolute is in a strong position, having raised $73 million in June. This funding ensures the advancement of their clinical programs and supports strategic corporate goals through to the second quarter of 2026.

Congenital hyperinsulinism is a prevalent cause of recurrent hypoglycemia in children and can lead to severe complications such as brain injury, seizures, and developmental issues if not properly managed. In severe cases, surgical intervention may be necessary. Ersodetug, a human monoclonal antibody, aims to normalize insulin receptor signaling and improve hypoglycemia across various forms of HI, offering a potentially universal treatment option.

Rezolute's mission centers on improving outcomes for those affected by hypoglycemia due to hyperinsulinism, with ersodetug showing considerable promise in clinical trials for both congenital and tumor HI.