On November 13, 2026 Rezolute, Inc. (Nasdaq: RZLT) (Rezolute or the Company), a late-stage ultra-rare, metabolic disease company focused on treating refractory hypoglycemia caused by any form of hyperinsulinism (HI), reported positive results from the ongoing open-label Phase 3 upLIFT study of ersodetug in tumor HI, in which the primary efficacy endpoint has been achieved, based on preliminary results from a November 5th data evaluation.

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The primary efficacy endpoint in the upLIFT study is the proportion of patients able to achieve at least a 50% reduction from baseline in glucose infusion rate (GIR), a measure of intravenous glucose dependency for underlying hypoglycemia. To achieve statistical significance for the endpoint, the lower bound of the 95% confidence interval for percent of patients responding must exceed 30%, which equates to at least 9 responders (≥56%), based on enrollment of 16 participants. To date, 13 participants have met the responder criterion, including 12 who also achieved a complete discontinuation of IV glucose. Three participants are still in the pivotal period of the study, including two who have not yet met the responder criterion. As previously reported, one participant discontinued the study early and chose to enter home hospice care, and died one week later due to cancer progression. This participant has been classified as a non-responder for purposes of the primary endpoint. Results for the completed study, including statistical analysis of the primary endpoint, are expected by mid-December 2026.

"We are thrilled with this outcome and what it means for individuals living with tumor hyperinsulinism," said Nevan Charles Elam, Founder and Chief Executive Officer of Rezolute. "Notably, these results substantially mirror the powerful outcomes that we have observed over the last few years in our expanded access program. We believe that we are well positioned to advance both congenital HI and tumor HI into the approval process next year and we look forward to discussions with FDA in the first quarter of 2027."

The 16 enrolled participants include four with non-islet cell tumors and 12 with insulin-secreting malignant islet cell tumors (insulinomas). Ersodetug has been well-tolerated to date in the pivotal and extension phases of the study, with no drug-related safety findings attributed to ersodetug.

About upLIFT

The upLIFT Phase 3 registrational study is a single-arm, open-label, pivotal trial in approximately 16 participants with insulinoma or non-islet cell tumors who have uncontrolled hypoglycemia caused by tumor hyperinsulinism (HI). Eligible participants requiring continuous intravenous (IV) glucose receive ersodetug 9 mg/kg per week for 8 weeks, as an add-on to standard of care. Following this 8-week pivotal treatment period, all participants may receive ersodetug at a reduced dosing frequency in long-term extension. The primary endpoint is the proportion of participants able to achieve at least a 50 percent reduction from baseline in GIR, a measure of intravenous glucose dependency for underlying hypoglycemia. Additional endpoints include the number of participants achieving complete discontinuation of GIR and time to discontinuation, time to discharge from the hospital as applicable, extent of hypoglycemia events and hypoglycemia time in the outpatient setting by self-monitored blood glucose and continuous glucose monitor, respectively, and patient reported quality of life.

About Tumor Hyperinsulinism

Tumor hyperinsulinism (HI) is a rare disease that may be caused by two distinct types of tumors: islet cell tumors (ICTs) and non-islet cell tumors (NICTs), both of which lead to hypoglycemia because of over-activation of the insulin receptor. Insulinomas are the most common type of ICT and cause hypoglycemia via the overproduction of insulin. A variety of different NICTs, including hepatocellular carcinoma and soft tissue malignancies (e.g. sarcomas), can cause hypoglycemia by producing and secreting insulin-like paraneoplastic substances, such as variants of IGF-2, that bind to and activate the insulin receptor. With high morbidity and mortality rates within tumor HI, there remains a significant unmet need for new therapies directed at hypoglycemia treatment. Ersodetug has shown real-world benefit in patients with insulinoma and NICTs.

About Ersodetug

Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor to decrease receptor over-activation by insulin and related substances (such as IGF-2) in the setting of hyperinsulinism (HI), thereby improving hypoglycemia. Because ersodetug acts downstream from pancreatic insulin or paraneoplastic IGF-2 secretion, and from entero-incretin pathways, it has the potential to be universally effective at treating refractory hypoglycemia due to any form of HI, including congenital HI, tumor HI (insulinoma, non-islet cell tumors) or bariatric/non-bariatric gastrointestinal surgery hypoglycemia. Ersodetug for the treatment of HI is investigational. Statements about safety and efficacy have not been approved by any health authority.

(Press release, Rezolute, NOV 13, 2026, View Source [SID1234674752])