Rezolute (RZLT) investor relations material

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.

Regulatory and commercialization strategy

• The company will submit the congenital HI dataset to FDA this month, with an agency review expected to conclude by fall, potentially clarifying the path to BLA submission.

• Both tumor and congenital HI programs could be ready for BLA filing in the first half of next year, depending on regulatory feedback.

• Pricing is expected to be robust, with pediatric rare disease pricing ranging from $400,000 to $600,000 per patient, and potentially higher for adult cancer patients due to weight-based dosing.

• Initial market opportunity is estimated at 3,000 patients across both insulinoma and non-islet cell tumor populations, with a focus on national cancer centers.

• Patient advocacy groups are actively engaged, supporting regulatory discussions and market awareness.

Forward-looking statements and growth plans

• The company is preparing for potential growth, including in-licensing new compounds in the ultra-rare disease space, but will prioritize current programs over the next year.

• If both tumor and congenital HI programs advance, a single BLA or broad label may be pursued, maximizing commercial potential.

• Significant catalysts are expected in the next six months, including pivotal data readouts and regulatory updates that could unlock further value.