SBT-272, in the pipeline at Stealth BioTherapeutics, is a new peptidomimetic being investigated as a potential therapy for neurodegenerative illnesses that are associated with mitochondrial dysfunction. In preclinical experiments, the administration of SBT-272 to defective mitochondria led to an increase in the generation of adenosine triphosphate (ATP) and a decrease in the levels of reactive oxygen species (ROS). In comparison to elamipretide, Stealth’s first-in-class lead chemical, SBT-272 exhibits much higher mitochondrial uptake, significantly higher concentrations in the brain, and significantly enhanced oral bioavailability.
Stealth BioTherapeutics ( Stealth ), announced May of 2018 that the US Food and Drug Administration (FDA) Office of Orphan Products Development granted Orphan Drug Designation to the investigational drug candidate, elamipretide, for the treatment of Leber’s hereditary optic neuropathy (LHON).
In a mouse model of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease characterized by the deterioration of motor neurons and the atrophy of muscle tissue, treatment with SBT-272 was associated with a dose-dependent delay in the onset of neurological disease, a reduction in systemic markers of neurodegeneration, and a prolonged lifespan. ALS is one of the most common forms of neurodegenerative disease. The compound is presently being investigated in a further preclinical model of amyotrophic lateral sclerosis (ALS), as well as in a preclinical model indicative of activity in multiple system atrophy (MSA). MSA is a neurological condition that can result in parkinsonism, cerebellar ataxia, dysautonomia, and other motor and non-motor symptoms. It is thought that mitochondrial dysfunction has a role in the course of neurodegenerative disorders such as ALS and MSA, as well as other conditions such as Parkinson’s disease, Huntington’s disease, and Alzheimer’s disease.