Rezolute Receives Breakthrough Therapy Designation from FDA for Ersodetug in the Treatment of Hypoglycemia Due to Tumor Hyperinsulinism

Core Insights - Rezolute, Inc. has received Breakthrough Therapy Designation (BTD) from the FDA for its investigational therapy, ersodetug, aimed at treating hypoglycemia caused by tumor hyperinsulinism (HI) [1][2][3] - The designation is based on clinical trial data and real-world experience demonstrating the potential of ersodetug to significantly improve treatment outcomes for patients with tumor HI [2][3] - A registrational study for ersodetug is expected to begin in mid-2025, with topline results anticipated in the second half of 2026 [3] Company Overview - Rezolute is a late-stage rare disease company focused on addressing hypoglycemia caused by hyperinsulinism [7] - The company's therapy, ersodetug, is designed to treat all forms of hyperinsulinism and has shown substantial benefits in both clinical trials and real-world applications for congenital and tumor HI [7] Treatment Context - Tumor hyperinsulinism is a rare disease caused by islet cell tumors (ICTs) and non-islet cell tumors (NICTs), leading to excessive insulin receptor activation and hypoglycemia [5] - Current standard treatments for persistent hypoglycemia in tumor HI are often inadequate, highlighting the need for new therapeutic options [3][5] Mechanism of Action - Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor, reducing over-activation by insulin and related substances, thereby improving hypoglycemia [6] - The therapy has shown real-world benefits in patients with insulinoma and has demonstrated potential in preclinical studies to blunt insulin-mediated signaling [5][6]