Elamipretide Hydrochloride

NEEDHAM, Mass., Sept. 18, 2024 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced the presentation of new bevemipretide (SBT-272) preclinical data demonstrating topical ocular delivery to the retina with protective effects observed in models of AMD. The data were presented at the Keystone Symposium Targeting Dry Age-related Macular Degeneration: Pathophysiology and Emerging Therapies held September 17-19, 2024, in Pacific Grove, CA. In this study, the distribution profile of ocular topical bevemipretide and its protective effects in models of AMD were evaluated. In animal models, bevemipretide eyedrops were well-tolerated and demonstrated desirable ocular posterior segment and systemic exposure profiles with optimized delivery to the retina and low plasma concentrations. In a rat model of bright light-induced retinal degeneration, topical administration of bevemipretide significantly attenuated the loss of retinal thickness. Protective effects of bevemipretide were also investigated using iPSC-derived retinal pigment epithelial (RPE) cells from patients with AMD. Treatment with bevemipretide significantly improved viability of patient-derived RPE cells and reduced cellular and mitochondrial ROS production in ARPE-19 cells treated with hydroquinone, a model used to mimic cigarette smoke-induced oxidative stress in AMD. "In dry AMD, mitochondrial dysfunction ultimately leads to the degeneration and loss of photoreceptors and reduction in the EZ to RPE thickness within the macula, resulting in progressive loss of visual function," said Reenie McCarthy, CEO of Stealth BioTherapeutics. "We demonstrated in our ReCLAIM-2 study of elamipretide, our lead investigational compound, that our mitochondrial-targeted approach may reduce the progressive photoreceptor loss associated with declining visual function. We designed bevemipretide as a next-generation approach to offer a broader range of potential therapeutic options to patients living with this devastating disease. We plan to initiate clinical studies to support further development of topical bevemipretide as a potentially disease-modifying therapy in dry AMD." About Dry AMD AMD is a leading cause of irreversible blindness estimated by the Vision and Eye Health Surveillance System to affect 19.8 million Americans aged 40 and older (Rein 2022). Dry AMD, which accounts for 85% to 90% of these cases (Schultz 2021), is a progressive retinal disease in which the mitochondrial-rich photoreceptors (specialized neurons that convert light into electrical signals required for normal visual function) in the retina suffer progressive damage and death, leading to loss of vision. Mitochondrial dysfunction 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 using optical coherence tomography (OCT), a noninvasive imaging technique, to measure thickness between the ellipsoid zone (EZ), a mitochondrial-dense layer of the photoreceptors, and the RPE (i.e., EZ-RPE thickness). Loss of photoreceptors 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 studying elamipretide, its lead investigational product candidate, in a Phase 3 development effort in dry AMD. Elamipretide is also under evaluation for primary mitochondrial myopathy, a rare skeletal myopathic disease, with pivotal Phase 3 data expected by year-end, and is under review by the Food and Drug Administration for Barth syndrome, an ultra-rare cardioskeletal disease, with a Prescription Drug User Fee Act date in January 2025. 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. Media Contact Anna Stallmann Anna Stallmann Communications [email protected] Investor Contact Austin Murtagh Precision AQ [email protected] SOURCE Stealth BioTherapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED