FDA Lifts Clinical Holds on Rezolute's Ersodetug (RZ358) for Congenital Hyperinsulinism

Key Insights

• The FDA has lifted partial clinical holds on Rezolute's ersodetug (RZ358), allowing clinical trials to proceed at all doses for patients older than three months in the U.S.
• Patient enrollment for the sunRIZE trial outside the U.S. is nearly complete, with over 90% of patients enrolled in Europe, Asia, and the Middle East, and completion expected by the end of 2024.
• Rezolute anticipates filing a New Drug Application (NDA) with the FDA in the second quarter of 2026, with a possible market introduction in 2027, following the inclusion of U.S. patients in trials.

The Food and Drug Administration (FDA) has lifted partial clinical holds on Rezolute's lead candidate, ersodetug (RZ358), a treatment for congenital hyperinsulinism (CHI), enabling the company to proceed with clinical trials across all doses for patients older than three months in the United States. This decision allows Rezolute to enroll U.S. patients in the trial, with topline data expected in the second half of 2025.

sunRIZE Trial Enrollment Progress

Rezolute's patient enrollment for its sunRIZE trial outside of the United States is progressing as planned, with completion expected by the end of 2024. The company has already enrolled over 90% of patients for this study in regions such as Europe, Asia, and the Middle East. The inclusion of U.S. patients is considered a strategic move to enhance the regulatory pathway in the United States, increase physician awareness, and facilitate patient identification, potentially accelerating the drug's launch in the U.S. market.

Regulatory and Market Outlook

Rezolute's management anticipates filing a New Drug Application (NDA) with the FDA in the second quarter of 2026, with a possible market introduction in 2027. According to an analyst from H.C. Wainwright, the global patient recruitment already ensures genetic diversity, and the addition of U.S. patients further solidifies the regulatory narrative. Investment firms BTIG, Craig-Hallum, Jones Trading, and H.C. Wainwright have all maintained a Buy rating on Rezolute. BTIG and H.C. Wainwright set price targets of $15.00 and $14.00 respectively.

Financial Position

Rezolute's fiscal fourth quarter of 2024 concluded with $127.1 million in cash and equivalents, which the company anticipates will support its operations into the second quarter of 2026. In addition, the company recently raised approximately $67 million in gross proceeds from an additional stock offering.

Additional FDA Clearance

The FDA also cleared Rezolute's phase 3 trial for RZ358, a therapy for hypoglycemia in patients with tumor hyperinsulinism, with patient enrollment in the U.S. expected to begin in the first half of 2025.