NEEDHAM, Mass., Aug. 9, 2023 /PRNewswire/ — Stealth BioTherapeutics, (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 Michael J. Fox Foundation for Parkinson’s Research (MJFF) awarded a research grant to evaluate Stealth’s mitochondria-targeted molecule, SBT-272, for Parkinson’s disease (PD).
The preclinical study is expected to inform the development of SBT-272 for PD by accelerating identification of biomarkers of mitochondrial dysfunction. Stealth will also collaborate with Laurie Sanders, Ph.D., Associate Professor in Neurology and Pathology, at the Duke University School of Medicine, on this two-year research initiative.
“We are pleased that The Michael J. Fox Foundation recognizes the importance of targeting mitochondrial dysfunction in PD,” said Chief Executive Officer Reenie McCarthy. “We are thrilled to work with MJFF to expedite development of SBT-272 and, hopefully, expand therapeutic options for patients living with PD.”
About Parkinson’s Disease and Mitochondrial Dysfunction
Parkinson’s disease (PD) is a progressive neurodegenerative disease that primarily affects patients later in life. It is the second most common neurodegenerative disease in the world, with a reported prevalence in industrialized countries around 0.3% of the entire population and about 1% in people older than 60 years of age.1 PD has distinctive neuropathological brain changes including the misfolding and aggregation of α-synuclein, a presynaptic neuronal protein, into abnormal spherical bodies (Lewy bodies), and aberrant branching of neurites in affected nerve cells.2,3 Mitochondrial dysfunction is a hallmark feature of PD, where interactions between α-synuclein and the mitochondrial phospholipid cardiolipin and aberrant mitochondrial mitophagy may be key drivers of early disease progression.4 There are no approved therapies that address mitochondrial dysfunction although it is a primary etiology in PD. Research has indicated prominent interactions between misfolded α-synuclein and the mitochondrial phospholipid cardiolipin as the early driver of disease.
SBT-272 is a novel investigational small molecule that targets cardiolipin, a phospholipid within the inner mitochondrial membrane that is essential for mitochondrial structure and function. SBT-272 has demonstrated mitochondria- and neuro-protective effects across preclinical models of alpha-synucleinopathy, amyotrophic lateral sclerosis, fronto temporal dementia, Huntington’s disease, and ischemic stroke. SBT-272-mediated improvements in functional assessments, lifespan, inflammation, and reduction of protein aggregates have been observed in these preclinical models. Data from a Phase 1 study evaluating subcutaneous SBT-272 in healthy volunteers supports further clinical development.
Stealth is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body’s main source of energy production and are critical for normal organ function. Dysfunctional mitochondria are centrally involved in a number of rare genetic diseases and many common age-related diseases, typically involving organ systems with high energy demands such as the eye, the neuromuscular system, the heart and the brain. The Company believes that its lead product candidate, elamipretide, has the potential to treat ophthalmic diseases, such as dry AMD, rare neuromuscular disorders, such as primary mitochondrial myopathy, and rare cardiomyopathies, such as Barth syndrome. In addition to SBT-272, the Company has a deep pipeline of novel mitochondria-targeted compounds under evaluation as therapeutic product candidates.
- De Lau L. M. and Breteler M. M. (2006) Epidemiology of Parkinson’s disease. Lancet Neurol.2006; 5:525–535.
- Braak H., Ghebremedhin E., Rub U., et al. Stages in the development of Parkinson’s disease-related pathology. Cell Tissue Res.2004; 318:121–134. 4.
- BraakH., Del TrediciK., RubU. et al. Staging of brain pathology related to sporadic Parkinson’s disease. Neurobiol Aging.2003;24,197–211.
- Toomey, C.E., Heywood, W.E., Evans, J.R. et al. Mitochondrial dysfunction is a key pathological driver of early-stage Parkinson’s. acta neuropathol commun 10, 134 (2022). https://doi.org/10.1186/s40478-022-01424-6
Anna Stallmann Communications
SOURCE Stealth BioTherapeutics Inc.