Rezolute Initiates Phase 3 Clinical Study for RZ358 in Congenital Hyperinsulinism

Company anticipates completing enrollment by the end of 2024 and reporting topline results in mid-year 2025

REDWOOD CITY, Calif., Dec. 14, 2023 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, today announced the initiation of sunRIZE, a pivotal Phase 3 clinical study of RZ358 in patients with congenital hyperinsulinism (cHI). Following country-level regulatory and ethics committee approvals, the first clinical site has now been activated outside the US, allowing for patient screening and enrollment to commence. Additional sites are being activated on a regular basis over the coming weeks and into the beginning of 2024.

The company's progression into Phase 3 was preceded and enabled by a successful Phase 2 trial (the RIZE study), which demonstrated promising results in the treatment of cHI, a serious condition with a tremendous burden on patients and families and a substantial unmet medical need. The current announcement of the Phase 3 start follows the receipt of a priority medicines (PRIME) designation of RZ358 for the treatment of cHI from the European Medicines Agency in October 2023.

“The start of the Phase 3 study marks a significant milestone in the development of RZ358 and exemplifies the tremendous progress of our Company as a whole. The unmet medical need for patients and their families living with cHI is unequivocal, particularly given that the only approved therapy, diazoxide, is unable to work in more than half of patients, or alternatively is associated with significant side effects in those that respond,” remarked Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute. “Better therapies are desperately needed, and we look forward to working closely with sites throughout the world to advance patient screening and enrollment in this pivotal study, so as to move one step closer to realizing that goal.”

The Phase 3 sunRIZE study is a multi-center, randomized, double-blind, placebo-controlled, parallel arm study designed to evaluate the efficacy and safety of RZ358 in patients with cHI who are experiencing poorly controlled hypoglycemia. Participants between the ages of 3 months to 45 years old are eligible to participate. The study will enroll up to 56 participants and be conducted at approximately 20 expert centers in more than a dozen countries around the world, many of which also participated in the Phase 2 RIZE study.

In the main comparator-controlled portion of the study, 48 participants in the age group of 1 year and older will be randomized (2:1) to receive RZ358 at doses of either 5mg/kg (n=16) or 10mg/kg (n=16), or matched placebo (n=16), in double-blind fashion and as add-on to standard of care. In parallel, an additional open-label arm will be conducted in approximately 8 additional participants in the age group of 3 months to 1 year old at a starting dose of 5mg/kg dose, which may be increased to 10 mg/kg as needed. All participants will receive study drug bi-weekly during a 6-week loading period, followed by every 4 weeks during the remainder of a 24-week total pivotal treatment period.

The primary efficacy endpoint over the 24-week pivotal period will be the change in average weekly hypoglycemia events (