Bevemipretide

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

Barth Syndrome is an Ultra-rare, Progressive, Life-Shortening, Cardioskeletal Disease that Affects Approximately 150 Individuals in the United States If Approved, Elamipretide Would Become the First Approved Therapy for Barth Syndrome NEEDHAM, Mass., May 7, 2024 /PRNewswire/ -- Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration ("FDA") has now determined that its New Drug Application ("NDA") for elamipretide for the treatment of Barth syndrome is entitled to a Priority Review designation. Per FDA policy, the user fee goal date of January 29, 2025 is unchanged. The FDA grants Priority Review to applications for therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. Barth syndrome is an ultra-rare cardioskeletal disease which results in frequent pediatric deaths and imposes a significant disease burden on all affected individuals. There are no other therapies approved or known to be in clinical development for this disease. The FDA's decision to grant Priority Review designation follows its March 29, 2024 filing decision in which the FDA assigned a standard review designation. Elamipretide has previously received Orphan Drug, Fast Track and Rare Pediatric Designations for elamipretide for the treatment of Barth syndrome. "We are pleased that the FDA has recognized the serious nature of Barth syndrome, the urgency of the unmet medical need, and the potential for elamipretide, if approved, to significantly improve treatment of this devastating disease," said Reenie McCarthy, Chief Executive Officer. "We welcome our continued constructive engagement with the Agency as we complete the review process and plan for an anticipated advisory committee meeting later this year." If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondrial targeted therapeutic. Elamipretide is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024, and dry age-related macular degeneration, with pivotal Phase 3 clinical trials initiating this quarter. About Barth Syndrome Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age 5. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 1,000,000 males worldwide or around 150 individuals in the United States. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome. Elamipretide has Orphan Drug, Fast Track and Rare Pediatric Designation from the FDA and Orphan Drug Designation from the EMA for the treatment of Barth syndrome. About Stealth BioTherapeutics Stealth BioTherapeutics is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body's main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases and are involved in many common age-related diseases, typically involving organ systems with high energy demands such as the heart, the eye, and the brain. The Company believes its lead product candidate, elamipretide, has the potential to treat rare diseases including Barth syndrome and primary mitochondrial myopathy and common age-related diseases including dry age-related macular degeneration. The Company is also evaluating a topical ophthalmic formulation of its second-generation clinical-stage candidate, SBT-272, for dry age-related macular degeneration. The Company has a deep pipeline of novel compounds under evaluation for rare neurological and cardiac disease indications following promising preclinical data. Media Contact Anna Stallmann Communications Anna Stallmann [email protected] Investor Relations Contact Austin Murtagh Stern Investor Relations [email protected] SOURCE Stealth BioTherapeutics Inc.