Elamipretide Hydrochloride

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. Investor Contact Austin Murtagh Precision AQ [email protected] Media Contact Anna Stallmann Anna Stallmann Communications [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

Today, a brief rundown of news involving Merck & Co. and AstraZeneca, as well as updates from AbbVie, Stealth BioTherapeutics and Vigil Neuroscience that you may have missed.A regimen of cancer drugs Keytruda and Lenvima extended progression-free survival over standard chemotherapy in people with a type of gastroesophageal tumor, but did not show an overall survival benefit. The results, announced by Merck & Co. and Eisai Friday, are from a Phase 3 study called LEAP-015 that enrolled hundreds of people with locally advanced unresectable or metastatic HER2-negative gastroesophageal adenocarcinoma. The companies said they are continuing to evaluate the data, which theyll present at a future medical meeting. The findings do not affect Keytruda or Lenvimas current approvals, or other trials in the LEAP program. Ned PagliaruloAstraZeneca will invest $570 million in a new office facility in Ontario that will help grow the companys presence in Canada. The drugmaker already employs more than 2,100 people in Canada many of whom work on clinical trials and last year acquired Hamilton, Ontario-based Fusion Pharmaceuticals. The new facility will add more than 700 positions to its workforce there, across all areas of the business, AstraZeneca said Thursday. Ben FidlerAbbVie will work with San Diego-based biotech startup Neomorph to develop protein-degrading molecular glue drugs for cancer and immune diseases, the companies announced Thursday. Neomorph will get an unspecified upfront payment from AbbVie in the deal and stands to receive up to $1.64 billion more in option fees and milestones. Its the third industry collaboration since last February for Neomorph, which also has partnerships with Biogen and Novo Nordisk. Ben FidlerThe Food and Drug Administration will take another three months to decide whether it will approve Stealth BioTherapeutics drug elamipretide for Barth syndrome. The company on Thursday said the FDA determined it needs more time to review recent information submitted by Stealth following an October meeting of agency advisers. The FDA didnt cite any safety issues, nor did it request more testing, Stealth said. The new decision deadline is April 29. Ned PagliaruloShares in Vigil Neuroscience rose by double digits this week after the biotechnology company reported Phase 1 trial data for an experimental drug its developing for Alzheimers. The data, which detail the drugs safety and distribution through the body, support advancement into a Phase 2 study, Vigil said. Code-named VG-3927, the small molecule drug is designed to boost a neuroprotective response in the brain to accumulations of tau and amyloid, proteins associated with Alzheimers. Ned Pagliarulo '