NEEDHAM, Mass., Sept. 11, 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 and the Friedreich’s Ataxia Research Alliance (FARA), today jointly announced Stealth has been awarded the Kyle Bryant Translational Research Award from FARA to evaluate mitochondria-targeted molecule SBT-589 in Friedreich’s ataxia (FA).
The preclinical study is expected to inform the development of SBT-589 for FA neuropathy. Mitochondrial dysfunction is central to pathology in FA and causes impairments in iron regulation, bioenergetics, and oxidative stress responses. These perturbations could lower the threshold for iron-mediated cell death, leading to neuronal cell loss early in disease. Preclinical studies have shown that SBT-589 directly targets mitochondrial dysfunction to improve bioenergetics, ameliorate pathology associated with iron dysregulation and reduce reactive oxygen species (ROS) production. In the study, the impact of SBT-589 on neurological and biochemical aspects of disease will be assessed in a transgenic mouse model of FA neuropathy. The study will also examine the impact of co-treatment with omaveloxolone, a recently approved therapy for FA patients. Dr. Laura Kropp, Senior Manager of Discovery Biology at Stealth will serve as the Primary Investigator on the project, with Dr. Jordi Magrane, Associate Professor of Research in Neuroscience at Weill Cornell Medical College, serving as a consultant.
“We are grateful for our continued partnership with FARA to determine the therapeutic potential of SBT-589 for individuals living with Friedrich’s ataxia,” said David A. Brown, Senior Vice President of Discovery at Stealth. “This collaboration will enable further development of SBT-589 as a potential new treatment that directly targets mitochondrial pathology in FA. Stealth has a diverse pipeline of molecules designed to improve cellular energy production, and this award is expected to bolster the progression of one of our lead candidates, SBT-589, into clinical testing”.
SBT-589 is a promising novel molecule that acts on mitochondrial pathways essential for cellular health and energy production. SBT-589 has previously been studied in FA patient-derived cells, isolated heart mitochondria, and an aggressive mouse model of FA cardiomyopathy. SBT-589 improved bioenergetics in FA patient-derived cells and reduced ROS emissions in isolated mitochondria. In FA mice, treatment with SBT-589 significantly reduced parameters of cardiac hypertrophy and delayed the onset of mortality.
“FARA is proud to co-sponsor this research grant to Stealth BioTherapeutics with the Friedreich’s Ataxia Research Alliance- Ireland,” said Liz Soragni, Director of Research at FARA. “Collaboration with fellow advocacy organizations, industry researchers, and academic scientists is essential in the development of potential treatments for FA.”
“As someone living with FA, I’m honored that this named grant helps advance research for a potential therapy in the development pipeline,” added Kyle Bryant, FARA Spokesperson and rideATAXIA Director, “I’m also grateful to be part of a community where everyone contributes in some way to realizing treatments for FA. Some of us fundraise, some participate in or conduct research, and some advocate. Every action matters.”
FARA supports research through funding competitive grants across the spectrum from basic research through drug development and clinical research programs in FA. FARA facilitates connections among FA researchers both from academia and industry with the goal of advancing therapeutic development in FA.
About Friedreich’s Ataxia
Friedreich’s ataxia is a rare genetic disease affecting an estimated 1 in 50,000 individuals in the United States. The disease is caused by a defect in the frataxin gene resulting in a relative deficiency of frataxin, leading to mitochondrial iron accumulation and oxidative stress. More than 90% of patients with Friedreich’s ataxia experience progressive cardiomyopathy which usually becomes fatal by early adulthood and is the leading cause of early mortality.
About Stealth BioTherapeutics
Our 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 myopathic diseases.
About Friedreich’s Ataxia Research Alliance (FARA)
The Friedreich’s Ataxia Research Alliance (FARA) is a non-profit organization dedicated to curing Friedreich’s ataxia (FA) through research. FARA grants and activities provide support for basic and translational FA research, pharmaceutical/biotech drug development, clinical trials, and scientific conferences. FARA is also dedicated to bringing the patient voice to the drug development process by connecting FA families with the scientific community to create worldwide exchanges of information that drive medical advances. For more information about FARA, visit curefa.org.
Media Contacts
Anna Stallmann Communications
Anna Stallmann
anna@annacomms.com
FARA Media Contact
Laura Preby, FARA Communications Director
media@curefa.org
Stealth Investor Contact
Austin Murtagh
Precision AQ
Austin.Murtagh@precisionaq.com
SOURCE Stealth BioTherapeutics Inc.
