Rezolute, Inc. (Nasdaq: RZLT), a leader in the development of novel therapies for serious metabolic and rare diseases, today disclosed promising topline results from its Phase 2 clinical trial of RZ402, aimed at treating Diabetic Macular Edema (DME). The study highlighted significant improvements in central subfield thickness (CST), a critical biomarker in DME management, across all dosages tested.

Findings in DME Treatment

The clinical trial involved 94 participants across various U.S. centers in a randomized, double-masked, placebo-controlled format, focusing on the efficacy, safety, and pharmacokinetics of RZ402 administered as monotherapy over a three-month period. Patients participating in the study showed a notable reduction in CST by up to 50 microns, confirming RZ402’s potential as a non-invasive first-line treatment for diabetic macular edema.

Quan Dong Nguyen, MD, a member of Rezolute’s Scientific Advisory Board and a distinguished Professor at Stanford University, stated, “These data are very encouraging and support the potential for a new, non-invasive treatment for DME.” The study outcomes are particularly significant given that current standard treatments involve invasive anti-VEGF injections, which can be burdensome and lead to delayed treatment and suboptimal outcomes.

Study Insights and Future Prospects

Participants in the study were limited to those with mild to moderate non-proliferative diabetic retinopathy and had received a maximum of three anti-VEGF injections prior to the study, with none within eight weeks of randomization. The study drug was administered once daily at doses of 50, 200, and 400 mg, or a placebo, followed by a four-week follow-up.

While the primary endpoints concerning CST reduction were met convincingly, the secondary endpoints related to Best Corrected Visual Acuity (BCVA) showed no significant improvements over the placebo during the short duration of the study. However, the observed improvements in CST suggest potential visual enhancements in longer-duration studies.

Raj Agrawal, MD, Vice President and Head of Ophthalmological Clinical Development at Rezolute, expressed gratitude towards the participants and medical staff involved, emphasizing the collaborative effort that made this study possible.

Industry Impact

As an oral therapy, RZ402 offers a significant shift from the current treatment paradigm by potentially allowing earlier intervention and treatment of both eyes simultaneously, which could fundamentally alter the long-term management of diabetic macular edema. The favorable safety profile and the absence of any significant ocular adverse effects further enhance its profile as a compelling treatment option.

This breakthrough could represent a substantial shift in how DME is treated, reducing reliance on invasive procedures and improving patient compliance and outcomes. As the study progresses to longer trials, the medical community eagerly anticipates further validation of RZ402’s efficacy and safety.

Rezolute’s success in this Phase 2 trial marks a pivotal step towards introducing an effective oral treatment for diabetic macular edema, which could significantly improve quality of life for patients worldwide. With these encouraging results, RZ402 is poised to become a key player in the future of DME treatment strategies.

  CST reduction, diabetic macular edema, DME treatment, non-invasive DME therapy, Phase 2 clinical trial, Rezolute, Rezolute RZ402, RZ402