Rezolute (RZLT) Jefferies Global Healthcare Conference 2026 summary

Key clinical updates and data insights

• Interim data from the tumor HI study show substantial drug activity, with six of eight patients meeting the primary endpoint, mirroring expanded access program results.

• Top-line data from the upLIFT phase III study in tumor hyperinsulinism is expected in the second half of the year, with enrollment completion anticipated within 2024.

• Quality of life improvements and reduction in hospitalization are being tracked as secondary outcomes, with strong evidence of clinical benefit and reduced need for background therapies.

• Open label extension data indicate a significant number of children remain on monotherapy, highlighting the drug's efficacy.

• The congenital HI phase III study missed its primary endpoint due to functional unblinding, but FDA is open to reviewing full data and CGM outcomes instead of requiring a new trial.

Background and disease overview

• Congenital hyperinsulinism is the leading cause of persistent, severe hypoglycemia in infants, with a birth incidence of 1:28,000 and over 30 genetic loci identified; 60% do not respond to diazoxide.

• Adverse neurodevelopmental outcomes occur in up to 50% of cases.

Study design and population

• The pivotal SUNRIZE study enrolled 63 participants aged 3 months to 15 years with persistent hypoglycemia despite standard care.

• Participants were randomized to receive ersodetug (5 or 10 mg/kg) or placebo, with dosing every 2–4 weeks and an open-label extension for long-term follow-up.

• Baseline characteristics included high rates of prior hospitalizations, multiple therapies, and significant hypoglycemia burden.