Stealth BioTherapeutics reaches 50 percent enrollment target in phase 3 ReNEW study

(Image Credit: AdobeStock/Fabio Principe)

Stealth BioTherapeutics Inc. has reached 50 percent of the enrollment target in the global Phase 3 ReNEW study (NCT06373731) of elamipretide as a potential treatment in patients with dry age-related macular degeneration (dry AMD). Complete target enrollment is set at 360 trial participants.

The ReNEW study for dry AMD is evaluating the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide, which is a first-in-class mitochondria-targeted investigational therapeutic. The primary efficacy endpoint will measure the rate of change in the macular area of photoreceptor loss, assessed by spectral domain-optical coherence tomography (SD-OCT) and ellipsoid zone (EZ) mapping.

Charles Wykoff, MD, PhD from Retinal Consultants of Texas, and Stealth's ReNEW Trial Scientific Review Committee Chair spoke to the importance treatment options for dry AMD. In the press release, he is quoted as saying, "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."

Reenie McCarthy, Chief Executive Officer of Stealth commented on the value the having an in-home dry AMD could bring to patients, saying, "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."

In addition to the ReNEW study, Stealth will initiate the ReGAIN study. This will be a second global Phase 3 trial in dry AMD set to begin in 2025. Data from the ReNEW study is expected to be reported in 2026.

Reference:

1. Stealth BioTherapeutics Announces Achievement of 50% Enrollment Target in Phase 3 ReNEW Study of Elamipretide in Patients with Dry Age-Related Macular Degeneration. Stealth Therapeutics. March 13, 2025. Accessed March 13, 2025. https://www.prnewswire.com/news-releases/stealth-biotherapeutics-announces-achievement-of-50-enrollment-target-in-phase-3-renew-study-of-elamipretide-in-patients-with-dry-age-related-macular-degeneration-302401062.html