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Associated Press
29-05-2025
- Business
- Associated Press
Telomir Pharmaceuticals Confirms Telomir-1 Restores Vision and Retinal Structure in Age-Related Macular Degeneration (AMD) Animal Model Using FDA-Recognized Surrogate Endpoints
A unique oral drug candidate that is shown to restore vision and regenerate the retina in an animal model, addressing a major unmet need in ocular therapeutics MIAMI, FLORIDA / ACCESS Newswire / May 29, 2025 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) ('Telomir' or the 'Company'), an emerging leader in age-reversal science, today announced compelling preclinical results from a study evaluating its novel oral therapeutic, Telomir-1, in a genetically modified zebrafish model of age-related macular degeneration (AMD). Following a 14-day oral dosing regimen, Telomir-1 reversed central vision response and vision acuity, restored retinal degeneration and architecture, and significantly reduced oxidative stress-achieving improvements across several FDA-recognized surrogate endpoints relevant to AMD. The study utilized the Sen57wrn-/-ND6-/+ zebrafish model, which combines genetic mutations associated with premature aging (WRN), mitochondrial dysfunction (ND6), and chronic senescence (Sen57). These animals exhibit progressive retinal degeneration, visual impairment, and oxidative stress-closely modeling dry AMD and geographic atrophy in humans. Model Transformation: From Degeneration to Recovery Before treatment, the aged (18-month-old) zebrafish demonstrated clear signs of neurodegeneration and visual impairment. Mutant animals showed sluggish, uncoordinated swimming behavior and delayed responses to visual stimuli such as light and movement-evidence of significant vision loss. Microscopic analysis of their retinas revealed approximately 15% total retinal degeneration, affecting several critical layers: In addition to retinal damage, the diseased animals exhibited reactive oxygen species (ROS) levels nearly four times higher than healthy controls-indicating intense oxidative stress-and suffered a 15% mortality rate during the two-week study window. After receiving Telomir-1, treated animals demonstrated marked recovery: Histological cross-sections confirmed Telomir-1's ability to regenerate not only the inner retinal layers, but also additional retinal structures-supporting improved-laminar retinal restoration and function. Collectively, these results demonstrate Telomir-1's ability to restore visual function, reverse retinal degeneration, reduce oxidative stress, and improve survival-all from a short oral treatment regimen. 'This breakthrough reinforces our vision at Telomir: to redefine how we treat age-related diseases by going beyond symptom management and targeting the root mechanisms of degeneration,' said Erez Aminov, Chief Executive Officer of Telomir. 'To our knowledge, no oral drug has ever demonstrated this level of retinal restoration and vision recovery in any AMD model-this is a meaningful leap forward for patients and the field.' 'The preclinical success achieved in this AMD model is truly remarkable,' added Dr. Angel, Chief Scientific Advisor of Telomir. 'Telomir-1 when studied orally, restored both structure and function in the retina, demonstrating not just neuroprotection, but true regenerative capacity-a property rarely seen in ophthalmic drug development.' Cautionary Note Regarding Forward-Looking Statements This press release, statements of Telomir's management or advisors related thereto, and the statements contained in the news story linked in this release contain 'forward-looking statements,' which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications, and the safety of Telomir-1. Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications and safety of Telomir-1. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which is on file with the SEC. Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Contact Information Helga Moya [email protected] (786) 396-6723 SOURCE: Telomir Pharmaceuticals, Inc press release
Yahoo
29-05-2025
- Business
- Yahoo
Stealth BioTherapeutics Announces "Path Forward" Despite Disappointing Delay for Ultra-rare Barth Syndrome
Following five years of discussions and a positive advisory committee recommendation, FDA proposes a potential accelerated approval pathway requiring resubmission of the elamipretide NDA Barth syndrome, a progressive, lethal, pediatric cardioskeletal disease often diagnosed at birth and with no approved therapies, affects ~150 individuals in the U.S. NEEDHAM, Mass., May 29, 2025 /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 that the U.S. Food and Drug Administration ("FDA") has identified a potential path forward for elamipretide for the treatment of Barth syndrome following years of discussions with the Agency and a 2024 Cardiovascular and Renal Drugs Advisory Committee meeting ("Ad Com") that concluded that elamipretide is effective for the treatment of Barth syndrome. The Company submitted data in its January 2024 new drug application ("NDA") showing that knee extensor muscle strength, which improved by over 45 percent in the Company's Phase 2 clinical trial, was significantly correlated with improvements on the six-minute walk test, an FDA-recognized indicator of clinical benefit. The Company proposed in its NDA and the FDA has now agreed to consider knee extensor muscle strength, for which the Company previously submitted data, as a potential intermediate clinical endpoint to support accelerated approval. "We recognize that the FDA's recommendation for an accelerated approval for elamipretide in Barth syndrome offers a path forward for this incurable pediatric disease that affects an incredibly small number of individuals worldwide," said Reenie McCarthy, CEO. "Elamipretide, which targets the cardiolipin deficit central to Barth syndrome, is the only agent in clinical development to treat this ultra-rare disease for which the FDA has acknowledged that additional pre-approval randomized controlled trials are unfeasible. We hope the FDA will also prioritize ensuring rapid access for neonates affected by the disease subject to appropriate post-marketing safety monitoring. We are experiencing a sharp increase in emergency access requests for critically ill infants from medical experts worldwide following the recent publication of several positive case study reports." Stealth received the FDA's complete response letter after a 16.5-month priority review cycle during which the FDA extended its review from January to April 2025 then missed its April 29 extended prescription drug user fee action date. The FDA also cited observations arising from a May 2025 CGMP surveillance inspection of a third-party manufacturing facility for elamipretide, although no specific deficiencies related to the elamipretide manufacturing process were identified. The FDA did not raise concerns with the clinical safety data, requesting only that a resubmission include any additional safety data collected since NDA submission. The Company will meet with the FDA next month to discuss the proposed post-marketing study, with which the Agency previously expressed alignment when originally proposed by the Company in 2022. The regulatory pathway for this development effort has been complex, entailing four different FDA review divisions since data was first presented to the FDA in 2019. The ultra-rare nature of Barth syndrome, the FDA's reservations regarding the positive data from SPIBA-001, a Phase 3 natural history control study assessing the functional endpoints utilized in the TAZPOWER open-label extension, and the FDA's prior refusal to consider an accelerated approval pathway all contributed to the challenges of the review. In its NDA submission, the Company proposed accelerated approval on the basis of a muscle strength intermediate endpoint to address the FDA's desire, echoed by several members of the Ad Com, to see additional confirmatory data. The FDA's decision to now consider this pathway offers hope for access to treatment for the U.S. Barth syndrome patient community, approximately 20% of whom already receive elamipretide under the Company's expanded access program. However, the Agency has expressed reluctance to extend the accelerated pathway to critically ill neonates, who comprise nearly two-thirds of the Company's expanded access program. Half of early deaths reported in this lethal pediatric disease occur by age one. The FDA's recommendation to resubmit on the basis of this new advice will further delay the potential approval and launch of elamipretide. The Company has implemented a 30% reduction in its personnel to conserve resources to fund a potential NDA resubmission and avoid interrupting patients' access to elamipretide through the Company's expanded access program. About Barth SyndromeBarth 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 primarily 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, Priority Review, and Rare Pediatric Designation from the FDA and Orphan Drug Designation from the EMA for the treatment of Barth syndrome. About Stealth BioTherapeuticsOur mission is to develop novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction. Our lead product candidate, elamipretide, is under review for Barth syndrome and in late-stage development for primary mitochondrial myopathy and dry age-related macular degeneration. We are also evaluating a topical ophthalmic formulation of our second-generation clinical-stage candidate, bevemipretide (SBT-272), for dry age-related macular degeneration, and have a deep pipeline of novel compounds under evaluation for rare neurological and cardiac disease indications. Media ContactAnna Stallmann CommunicationsAnna Stallmannanna@ Investor ContactPrecision AQAustin View original content to download multimedia: SOURCE Stealth BioTherapeutics Inc. Sign in to access your portfolio
