Stealth BioTherapeutics, a clinical-stage biotech focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, has 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. The value of the grant was not disclosed by Stealth.

Parkinson’s 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]. Parkinson’s has distinctive neuropathological brain changes and mitochondrial dysfunction is a hallmark feature of Parkinson’s, but there are no approved therapies that address mitochondrial dysfunction. However, research has indicated interactions between misfolded proteins 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 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 preclinical models, and according to Stealth, data from a Phase 1 study evaluating subcutaneous SBT-272 in healthy volunteers supports further clinical development.

Stealth says it expects the preclinical study to inform the development of SBT-272 for Parkinson’s by accelerating identification of biomarkers of mitochondrial dysfunction. Stealth will also collaborate with Laurie Sanders, PhD, 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 Parkinson’s,” 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 Parkinson’s.”

[1] https://pubmed.ncbi.nlm.nih.gov/16713924/