Stealth BioTherapeutics announced the initiation of a first-in-human Phase 1 trial evaluating its second generation pipeline compound, SBT-272, in healthy subjects. The Phase 1 trial is a double-blind, placebo-controlled, single-ascending dose study enrolling up to 40 healthy subjects across multiple cohorts. SBT-272 is being administered orally in the study. As a primary objective, the study will evaluate safety and tolerability of SBT-272. Secondary objectives include an analysis of the pharmacokinetic profile and appropriate dose range. SBT-272 is a novel peptidomimetic being developed for the treatment of neurodegenerative diseases involving mitochondrial dysfunction. SBT-272 has been shown to increase adenosine triphosphate (ATP) production and decrease levels of reactive oxygen species (ROS) in dysfunctional mitochondria in preclinical studies. SBT-272 demonstrates higher mitochondrial uptake, greater concentrations in the brain, and improved oral bioavailability relative to elamipretide, Stealth's first-in-class lead compound. 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 prolonged lifespan in a mouse model of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease characterized by motor neuron deterioration and muscle atrophy. The compound is currently being evaluated in another ALS preclinical model, as well as in a preclinical model indicative of activity in multiple system atrophy (MSA), a neurological disorder leading to parkinsonism, cerebellar ataxia, dysautonomia and other motor and non-motor symptoms. Mitochondrial dysfunction is believed to contribute to the progression of ALS and MSA, as well as other neurodegenerative diseases including Parkinson's, Huntington's and Alzheimer's.