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The Sun
30-05-2025
- Business
- The Sun
Stealth Bio to cut jobs after FDA rejects therapy for rare disorder
PRIVATELY held Stealth BioTherapeutics said on Thursday the U.S. Food and Drug Administration had declined to approve its therapy for an ultra-rare condition called Barth Syndrome, prompting it to cut 30% of its workforce. Barth syndrome, which is estimated to affect around 150 people in the United States, has no approved treatments. It typically affects boys and causes heart and muscle weakness, as well as delayed growth. The FDA has asked the company to resubmit its application, which Stealth said would require more capital and prompted the job cuts. The FDA did not respond to a Reuters request for comment The therapy, elamipretide, has faced several setbacks, including the FDA's refusal to accept its marketing application in 2021. The company subsequently went private in 2022. The latest setback comes after a 16.5-month review, during which the FDA extended its review from January to April to assess additional data. Last month, the regulator missed the extended deadline. The company said the rejection came 'out of the blue'. 'We don't understand why it wasn't an approval, because there isn't really a request that we resubmit new data,' said CEO Reenie McCarthy. 'I think our frustration is: why didn't this come sooner?' McCarthy said. In October, a panel of advisers to the FDA voted 10-6 in favor of the therapy. Stealth had submitted data from a mid-stage trial that showed improvement in knee muscle strength by over 45%, which correlated with improvements on the six-minute walk test. FDA raised the possibility of an accelerated approval based on the findings, but is hesitant to extend the pathway to critically ill newborns, who make up nearly two-thirds of the therapy's expanded access program participants. The company said it will meet with the FDA next month.
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
Associated Press
13-03-2025
- Business
- Associated Press
Stealth BioTherapeutics Announces Achievement of 50% Enrollment Target in Phase 3 ReNEW Study of Elamipretide in Patients with Dry Age-Related Macular Degeneration
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 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 Media Contact Anna Stallmann Anna Stallmann Communications
