Biotech Targeting Mitochondrial Dysfunction Research

Funding will more specifically support development of elamipretide for primary mitochondrial myopathy and dry age related macular degeneration, which do not have FDA approved treatments, and will see elamipretide through phase 3 trials in the former indication and start phase 2b clinical trials according to an SEC filing. The SEC filing also states that funding will advance preclinical drug SBT-272 being developed for neurodegenerative diseases into phase 1.

Their lead asset elamipretide is a peptide which penetrates cell membranes to target cardiolipin in inner mitochondrial membranes, the phospholipid is produced in this membrane; preclinical and clinical studies have shown elamipretide to increase mitochondrial respiration and improve production of adenosine triphosphate according to Stealth Bio.

Elamipretide cardiolipin association has been shown to normalize structure of the inner mitochondrial membrane improving function, and is also being developed for Barth syndrome and Leber’s hereditary optic neuropathy, according to the biotech.

According to Stealth Bio their internal discovery platform has generated a library of over 100 proprietary differentiated compounds that may have clinical benefits for disease which are related to mitochondrial dysfunction, certain compounds could also potentially serve as scaffolds to deliver other beneficial compounds to the mitochondria.

This biotech is not alone in the mitochondrial dysfunction space, Astellas acquired its partner MitoBridge with MA-2011 being the centerpiece of the acquisition which is a PPAR-delta modulator designed to reverse the mitochondrial defects that contribute to progression of Duchenne muscular dystrophy.