By Brad Sorensen, CFA

NASDAQ:MITO

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Stealth BioTherapeutics (NASDAQ:MITO) is clinical stage biopharmaceutical company that is attempting to repair mitochondria to treat previously untreatable conditions. Stealth BioTherapeutics announced that its signature treatment, elamipretide, was awarded Orphan Drug Designation for the treatment of Friedreich's Ataxia. The company also announce positive results from its preclinical trial of SBT-272 in a mouse model of Huntington's disease.

Friedrich's ataxia, the most common form of ataxia, affects about 1 in 40,000 people in the United States. Friedrich's ataxia is an inherited, genetic disease that damages the spinal cord, peripheral nerves and the cerebellum portion of the brain. The condition typically develops in children and gradually grows worse over time. Friedrich's is caused by a genetic defect in the gene labeled FXN, which carries the genetic code for a protein called frataxin. In preclinical studies, elamipretide has been shown to improved mitochondrial structure and function as well as increasing mature frataxin levels in Friedrich's patient lymphoblasts. As a result of these positive developments, the researcher involved in this study has submitted a Phase 2a clinical trial to the FDA and is currently waiting for Institutional Review Board approval. Stealth is supporting the study in the assessment of the impact of elamipretide in a cohort of patients affected by cardiomyopathy and/or visual decline associated with Friedrich's. As noted above, and we believe this to be a fairly significant development, Stealth has been granted Orphan Drug Designation for elamipretide for the treatment of Friedrich's ataxia, which provides various benefits including the potential for seven years of market exclusivity after approval.

Elamipretide is not the only compound that Stealth is developing. In fact, although it's early in the process, early indications from the compound known as SBT-272, in our view, may end up being the most consequential development from Stealth out of the current potential treatments—both in terms of impact on patients and the commercialization possibilities. Stealth's primary objective in designing SBT-272 was to increase brain exposure compared to elamipretide due to the potential that mitochondrial may be beneficial in neurological disorders such as amyotrophic lateral sclerosis (ALS), Parkinson's, Huntington's and Alzheimer's.

Preclinical data, according to Stealth, showed that SBT-272 demonstrated a three times greater concentration of the compound in the brains of rats compared to elamipretide and 25 times greater area under the concentration-time curve—both of which suggest that SBT-272 has both higher brain exposure and greater residence time.