Stealth BioTherapeutics Secures MRC Grant to Advance SBT-589 for Leigh Syndrome Treatment
Key Insights
Stealth BioTherapeutics (search) received a Business Engagement Fund grant from the UK Medical Research Council to evaluate SBT-589 in Leigh syndrome (search) mouse models.
The collaborative research will assess SBT-589's effects on survival rates, motor function, and disease biomarkers in preclinical studies.
Previous studies demonstrated SBT-589 improved bioenergetics in Leigh syndrome (search) patient-derived cells and ameliorated mitochondrial dysfunction (search) pathology.
Stealth BioTherapeutics (search), a commercial-stage biotechnology company focused on mitochondrial dysfunction (search) therapies, has secured funding from the UK Research and Innovation Medical Research Council's Business Engagement Fund to advance preclinical evaluation of SBT-589 for Leigh syndrome (search) treatment.
The grant will support a collaborative research project among the Mary Lyon Centre at MRC Harwell (search), the MRC National Mouse Genetics Network (search), and academic leaders from University College London. The research will evaluate SBT-589's effects on survival rates, motor function, and biomarkers of disease in a mouse model of Leigh syndrome (search).
Targeting Mitochondrial Dysfunction in Leigh Syndrome
Mitochondrial dysfunction (search) is central to Leigh syndrome (search) pathology, affecting cells that supply energy to the brain, nerves, and muscles. Previous preclinical studies have demonstrated that SBT-589 improved bioenergetics in Leigh syndrome patient-derived cells and ameliorated pathology associated with mitochondrial dysfunction and neurodegeneration across animal models of disease.
"I'm excited to support the preclinical evaluation of SBT-589, leveraging our NMGN MitoCluster platform at the MLC to deeply characterize mitochondrial dysfunction (search) in disease models," said Professor Robert Pitceathly of UCL. "Combined with the extensive clinical and non-clinical expertise in mitochondrial pathology contained within our consortium, we aim to accelerate the development of therapeutics, such as SBT-589, towards clinical applications."
Addressing Development Barriers
The collaboration builds on Stealth's established partnership with investigators within the NMGN MitoCluster and prior clinical research in primary mitochondrial myopathy (search). David A. Brown, Ph.D., Stealth's Chief Scientific Officer, emphasized the project's significance in overcoming historical development challenges.
"Testing promising assets in disease-relevant animal models has historically been a barrier to progress in mitochondrial drug development," Brown stated. "This project helps address that gap and strengthens our ability to translate innovative science and advance potential therapies into the clinic."
Accelerating Translation to Clinical Development
Professor Owen Sansom, Director of the MRC National Mouse Genetics Network (search), highlighted the fund's strategic approach to fostering industry-academia partnerships. "The Business Engagement Fund is designed to bring the expertise of the MRC National Mouse Genetics Network together with the innovation capacity of industry partners," Sansom explained. "By supporting early-stage collaborations, we can help accelerate the translation of discoveries from advanced model systems into new approaches for understanding and treating disease, including in important areas such as mitochondrial biology."
The project is expected to accelerate the progression of promising mitochondrial therapies toward clinical development and future regulatory milestones, supporting shared goals to address significant unmet needs faced by individuals living with diseases of mitochondrial dysfunction (search).
Stealth BioTherapeutics (search)' first commercial product received accelerated approval from the U.S. Food & Drug Administration (search) in September 2025. The company is currently studying elamipretide in additional indications, including dry age-related macular degeneration (search) and primary mitochondrial disease (search) due to nuclear DNA mutations, while developing bevemipretide for ophthalmic and neurological disease indications. The preclinical pipeline includes SBT-589 and SBT-255 (search), both progressing through developmental milestones.