Rezolute, Inc. (NASDAQ: RZLT), a late-stage metabolic disease company focused on treating refractory hypoglycemia caused by hyperinsulinism, said preliminary results from a data evaluation show its open-label Phase 3 upLIFT study of ersodetug in tumour hyperinsulinism has met its primary efficacy endpoint.
The endpoint is the proportion of patients achieving at least a 50% reduction from baseline in glucose infusion rate, a measure of intravenous glucose dependency. Statistical significance requires the lower bound of the 95% confidence interval to exceed 30%, equivalent to at least nine responders (56% or more) among 16 enrolled participants.
To date, 13 participants have met the responder criterion, 12 of whom also stopped intravenous glucose entirely. Three remain in the pivotal period, two of whom have yet to respond. One participant left the study early, entered home hospice care and died a week later from cancer progression, and is classed as a non-responder.
Ersodetug has been well tolerated in the pivotal and extension phases, with no drug-related safety findings attributed to it.
Topline results and statistical analysis of the primary endpoint are expected by mid-December 2026. Rezolute aims to bring both congenital and tumour hyperinsulinism into the approval process next year, with discussions with the US Food and Drug Administration due in the first quarter of 2027.
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