Rezolute Announces Positive Phase 2 Results for RZ402 in Diabetic Macular Edema Patients

Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company, recently announced promising topline results from its Phase 2 clinical study of RZ402 for treating Diabetic Macular Edema (DME). The study, which evaluated the safety and efficacy of RZ402, showed a positive outcome, meeting its primary endpoints of a good safety profile and a significant reduction in central subfield thickness (CST).

"The results are monumental for the DME community," said Dr. Quan Dong Nguyen, a Professor of Ophthalmology at Stanford University and a member of Rezolute’s Scientific Advisory Board. He highlighted the significant reduction in CST across all dosages, pointing out that retinal thickness is a crucial biomarker for determining the potential benefits of a therapy for patients.

The U.S. multi-center study enrolled 94 participants in a randomized, double-masked, placebo-controlled, parallel-arm format. Participants were either naïve to or had limited exposure to anti-vascular growth factor (anti-VEGF) injections. The trial aimed to assess the safety, efficacy, and pharmacokinetics of RZ402 as a monotherapy over a 12-week treatment period.

Dr. Arshad Khanani, the Primary Investigator of the study and Clinical Professor at the University of Nevada, expressed enthusiasm about the potential of an oral therapy for DME. He noted that oral therapy would enable treatment of both eyes simultaneously and allow for earlier intervention, potentially altering the long-term prognosis for DME patients.

Raj Agrawal, Vice President and Head of Ophthalmological Clinical Development at Rezolute, thanked the patients, retina specialists, and their staff for making the study possible. He emphasized the importance of the study results for future treatment options.

The study involved DME patients with mild to moderate non-proliferative diabetic retinopathy (DR). Eligible participants had received no more than three anti-VEGF injections previously and had specific CST measurements and visual acuity criteria. They were randomized equally into one of three RZ402 active treatment arms at doses of 50, 200, and 400 mg, or a placebo control arm, with the study drug administered once daily for 12 weeks.

Results showed a significant improvement in CST at all dose levels compared to placebo, with a continued downward trend over the course of the study. The most notable response was at the 200 mg dose, where patients experienced an improvement of approximately 75 microns in CST. Importantly, RZ402 was found to be safe and well-tolerated, with adverse events comparable to placebo and no significant ocular adverse effects typically seen with intravitreal injections.

The study's safety assessments, including electrocardiograms (ECGs), vitals, and safety labs, were unremarkable. Target concentrations were exceeded at all dose levels, supporting once-daily oral dosing.

While there were no significant improvements in Best Corrected Visual Acuity (BCVA) compared to placebo, the observed improvements in CST suggest potential visual benefits in a longer-duration study. Additionally, five participants treated with 200 mg of RZ402 experienced a 1-step improvement in the Diabetic Retinopathy Severity Score (DRSS) compared to one participant in the placebo group.

DME is a severe complication of diabetic retinopathy characterized by retinal swelling and macular thickening, which can severely affect vision. Current standard care involves invasive anti-VEGF injections, which require long-term administration and can result in compliance challenges and occasional serious side effects. RZ402, a selective and potent plasma kallikrein inhibitor, aims to address these issues by offering a non-invasive oral treatment option.

Rezolute is committed to developing transformative therapies for serious metabolic and rare diseases, aiming to significantly improve patient outcomes and reduce treatment burdens. The company integrates patient, clinician, and advocate perspectives into its drug development process to address a range of severe conditions effectively.