Developing the novel investigational mitochondrial therapeutic SBT-589 for Friedreich’s ataxia

This project intends to advance an investigational therapeutic developed by Stealth Biotherapeutics, SBT-589, that directly targets mitochondrial dysfunction and mitigates the pathology associated with iron dysregulation. In the laboratory, SBT-589 has shown promising effects in cells derived from FA patients. In a recent FARA-funded study (KMA Cardiac Research Award; Advancing novel mitochondrial therapies for FA cardiomyopathy: a pragmatic collaboration to move new therapies forward), SBT-589 showed efficacy in a mouse model of FA that recapitulates the FA cardiac disease. SBT-589 prevented cardiac hypertrophy and delayed the onset of mortality in mice. Building upon these encouraging results, Dr Kropp and her team will generate additional data in models that are relevant to the neurological manifestations and biochemical changes seen in FA to better the understanding of the effects of SBT-589 on neuronal tissues. The proposed work will test the hypotheses that the investigational compound SBT-589 can improve mitochondrial and biochemical parameters of FA neuropathy. Additionally, Dr Kropp will test the hypothesis that co-treatment with omaveloxolone will enhance the efficacy of SBT-589. Successful completion of these studies is expected to complement the encouraging data seen in cardiac-specific models, as well as patient-derived cells. This project is expected to help advance SBT-589 toward the clinic as a potential disease-modifying therapy for FA individuals.