Stealth BioTherapeutics Hits 50% Enrollment in AMD Trial

Stealth BioTherapeutics Inc. has achieved 50% of its enrollment target in the global Phase 3 ReNEW study (NCT06373731), evaluating elamipretide as a potential treatment for dry age-related macular degeneration (dry AMD). The study aims to enroll a total of 360 participants.

Evaluating a Novel Mitochondria-Targeted Therapy

The ReNEW study is assessing the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide, a first-in-class mitochondria-targeted investigational therapeutic. The trial’s primary efficacy endpoint measures the rate of change in the macular area of photoreceptor loss, evaluated through spectral domain-optical coherence tomography (SD-OCT) and ellipsoid zone (EZ) mapping.

Expert Insights on Dry AMD and Treatment Needs

Dr. Charles Wykoff, MD, PhD, from Retinal Consultants of Texas and Chair of Stealth’s ReNEW Trial Scientific Review Committee, emphasized the urgent need for treatment options.

"Dry AMD, and its late-stage manifestation, geographic atrophy, is a devastating disease process that leads to progressive visual dysfunction and irreversible vision loss. Our patients are in need of new and improved treatment options. A consistent and early hallmark of dry AMD is progressive thinning of the photoreceptors. It is exciting to consider the potential of elamipretide – the first mitochondria-targeted investigational therapy for dry AMD – to slow photoreceptor thinning and loss," he stated.

A Potentially Game-Changing At-Home Treatment

Stealth BioTherapeutics' CEO, Reenie McCarthy, highlighted the significance of an in-home dry AMD treatment.

"The rapid enrollment of the pivotal Phase 3 ReNEW study underscores this community’s desire for novel therapeutics that can be self-administered at home and offer the potential to improve visual function. By targeting retinal bioenergetics to reduce the loss of photoreceptors, we hope to disrupt the progressive vision loss that characterizes this devastating disease."

Next Steps: ReGAIN Study and Future Developments

Building on the ReNEW study, Stealth plans to launch ReGAIN, a second global Phase 3 trial in dry AMD, in 2025. Data from the ReNEW study is expected to be reported in 2026, marking a significant milestone in the search for effective treatments for dry AMD.