Rezolute (RZLT) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Programs and clinical progress

• Late-stage metabolic rare-disease programs target refractory hypoglycemia associated with congenital and tumor-associated hyperinsulinism; ersodetug is a fully human monoclonal antibody designed for all forms of the condition.

• Phase III for congenital hyperinsulinism is complete; top-line Phase III results for tumor-associated hyperinsulinism were expected soon.

• In tumor-associated disease, the FDA worked with the sponsor to streamline the Phase III design to a single-arm, open-label study; the stated primary threshold is a 50% reduction in glucose infusion requirements for at least 50% of patients.

• Expanded-access experience over three years included more than a dozen tumor patients in the U.S. and Europe; reported benefits included reduced hypoglycemia, hospital discharge, resumed cancer therapy, and return to normal activities.

• For congenital disease, CGM analyses were reported to show a 50%-75% difference between placebo and treatment arms in the worst-case scenario; after reviewing raw data, the FDA advised proceeding with the BLA process, without declaring the treatment approvable.

Regulatory plans and timing

• A congenital BLA filing was planned for the middle of next year; the FDA discussion on the path for both indications was expected in the first part of next year.

• The conservative regulatory sequence envisioned a congenital PDUFA date in Q1 2028, followed roughly nine months later by a tumor sBLA PDUFA date; timing could change after FDA discussions.

• The timing of disclosure after the pre-BLA meeting will depend on the meeting outcome; prior clear FDA feedback was announced before meeting minutes were received.

• A congenital pediatric rare-disease priority review voucher was a stated objective; the plan was to seek congenital approval first while discussing whether tumor review could proceed sooner given its unmet need.

Market and commercial outlook

• Estimated addressable populations were 1,500 pediatric congenital patients non-responsive to diazoxide, with additional patients potentially migrating from diazoxide; total congenital TAM was estimated at 2,000.

• Tumor estimates comprised 2,500 insulinoma patients with recurrent refractory hypoglycemia and hospitalization, plus 1,500 NICTH patients; total tumor TAM was ~4,000 and combined population ~6,000.

• Expanded-access experience included patients on treatment for more than two years; treatment duration was expected to be longer for insulinoma than NICTH.

• The stated total addressable market opportunity was well north of $5 billion; pricing was expected to be weight-based and linear from congenital to tumor, with ultra-rare pricing and minimal payer budget impact.

• A lean field footprint was envisioned: 10-15 salespeople for congenital, with a possible separate tumor team; patient support, market access, and commercial operations would support identification and reimbursement.