Stealth BioTherapeutics Announces Achievement of 50% Enrollment Target in Phase 3 ReNEW Study of Elamipretide in Patients with Dry Age-Related Macular Degeneration

Associated Press

13-03-2025

NEEDHAM, Mass., March 13, 2025 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the 'Company' or 'Stealth'), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that it has achieved its 50 percent enrollment target in the global Phase 3 ReNEW study ( NCT06373731) of elamipretide in patients with dry age-related macular degeneration (dry AMD). The total target enrollment is 360 participants. The ReNEW study is evaluating the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide – a first-in-class mitochondria-targeted investigational therapeutic – in participants with dry AMD. The primary efficacy endpoint (as agreed with the FDA) 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. Photoreceptors are specialized light-detecting cells in the retina that enable vision in both bright and dim light conditions, and loss of these cells is closely associated with worsening visual function.
'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,' said Charles Wykoff, MD, PhD from Retinal Consultants of Texas, and Stealth's ReNEW Trial Scientific Review Committee Chair. '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.'
'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,' said Reenie McCarthy, Chief Executive Officer of Stealth. '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 – a second global Phase 3 trial in dry AMD – in the coming months. Data from the ReNEW study is expected to be reported in 2026.
About ReNEW and ReGAIN
ReNEW and ReGAIN are Phase 3 global clinical trials evaluating the efficacy and safety of once-daily self-administered subcutaneous injections of elamipretide in participants with dry AMD. The primary endpoint for the trials is the rate of change in the macular area of photoreceptor loss assessed by spectral domain-optical coherence tomography and ellipsoid zone mapping at week 48. In the ReNEW and ReGAIN trials, 360 patients will be randomized 2:1 to either elamipretide or placebo for 96 weeks with the option for participants to enroll in the open-label extension trial, ReTAIN. Learn more about the studies at Dry-AMDClinicalTrials.com.
About Dry AMD
AMD is the leading cause of irreversible blindness and is estimated by the Vision and Eye Health Surveillance System to affect 19.8 million Americans aged 40 and older (Rein 2022), with 85% to 90% of cases being dry AMD (Schultz 2021). Dry AMD is a progressive retinal disease in which the photoreceptors, which are specialized neurons found in the retina that convert light into electrical signals required for normal visual function, suffer progressive damage and death, leading to loss of vision. Mitochondrial dysfunction, which has been associated with aging, smoking, obesity, and cardiovascular health, is known to precede clinical symptoms of AMD and increase commensurate with AMD disease progression (Feher 2006; Karunadharma 2010; Terluk 2015). Photoreceptor loss can be quantified by measuring the thickness between the EZ and retinal pigment epithelium (i.e., EZ-RPE thickness). Loss of photoreceptors cells, or EZ attenuation, has been shown to precede and predict the loss of visual function and areas of geographic atrophy in dry AMD.
About Stealth BioTherapeutics
Stealth BioTherapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel therapies for age-related and rare genetic diseases involving mitochondrial dysfunction. The Company is conducting Phase 3 clinical trials of elamipretide, its lead investigational product candidate, in dry age-related macular degeneration. Elamipretide is also being tested in a fully enrolled Phase 3 clinical trial in primary mitochondrial myopathy, a rare skeletal myopathic disease, and is under review by the Food and Drug Administration for Barth syndrome, an ultra-rare cardioskeletal disease. The Company is developing its second-generation clinical-stage candidate, bevemipretide (SBT-272), for ophthalmic and neurological disease indications. The Company has a deep pipeline of novel mitochondria-targeted compounds under evaluation as therapeutic product candidates.
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