Core Insights - Rezolute, Inc. has aligned with the FDA on a streamlined clinical development path for its Phase 3 study (upLIFT) of ersodetug, aimed at treating hypoglycemia caused by tumor hyperinsulinism (HI) [1][2] - The FDA has approved modifications to the study design, allowing for a single-arm open-label trial with as few as 16 participants, eliminating the need for a double-blind randomized placebo-controlled trial [2][3] - The pivotal sunRIZE trial in congenital HI is expected to report topline results in December 2025 and will serve as confirmatory evidence for the efficacy of ersodetug [2] Company Overview - Rezolute is a late-stage rare disease company focused on treating hypoglycemia caused by hyperinsulinism, with its antibody therapy, ersodetug, showing meaningful benefits in clinical trials and real-world applications [8] - The company has treated over 10 patients with tumor HI under its Expanded Access Program, contributing to the favorable outcomes that influenced the FDA's decision [3] Study Details - The upLIFT study is a single-arm, open-label pivotal trial involving approximately 16 participants with insulinoma or non-islet cell tumors experiencing uncontrolled hypoglycemia due to tumor HI [4] - Participants will receive ersodetug at a dosage of 9 mg/kg per week for 8 weeks, with the primary endpoint being a 50% reduction in intravenous glucose requirements [4] Disease Context - Tumor hyperinsulinism is a rare disease caused by islet cell tumors (ICTs) and non-islet cell tumors (NICTs), leading to hypoglycemia through over-activation of the insulin receptor [5] - Insulinomas are the most common ICTs, while various NICTs, particularly hepatocellular carcinoma, can also cause hypoglycemia by secreting insulin-like substances [5] Mechanism of Action - Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor, reducing receptor over-activation by insulin and related substances, thereby improving hypoglycemia [7]