Rezolute (RZLT) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Disease background and unmet need

• Hyperinsulinism causes dangerous hypoglycemia, affecting both congenital and tumor-related patient groups.

• Congenital HI affects about 3,500 individuals in the U.S., with tumor HI estimated to be twice as prevalent.

• Current standard of care, diazoxide, is only effective in about 40% of patients and has significant side effects.

• There has been no universal therapy for all forms of hyperinsulinism until now.

Clinical development and regulatory progress

• Lead antibody therapy is in phase III for both congenital and tumor HI, aiming to modulate insulin binding in a dose-dependent manner.

• The UPLIFT study for tumor HI was streamlined with FDA alignment, reducing the trial size and expediting timelines.

• SUNRISE phase III study in congenital HI expects data in December, targeting a 35% difference between placebo and treatment arms.

• Breakthrough Therapy designation was granted for both indications, with BLA filing planned for mid-2026 and potential approval in early 2027.

Real-world impact and expanded access

• Expanded access program has shown the therapy can normalize glucose and improve quality of life, with some patients treated for up to three years.

• Real-world results have influenced regulatory flexibility, allowing for smaller, open-label studies.

• Patient and family testimonials highlight dramatic improvements from near end-of-life to normal activities.