Stealth BioTherapeutics filed on Friday to raise up to $86 million in its Nasdaq IPO, funding that will push its lead asset through the clinic in multiple indications. The company is developing drugs that treat mitochondrial dysfunction linked to age-related diseases and genetic mitochondrial diseases.
Specifically, the funding will support the development of elamipretide for primary mitochondrial myopathy and dry age-related macular degeneration, both of which have no FDA-approved treatments. It will see the drug through a pivotal phase 3 trial in the former indication and start a phase 2b clinical trial in the latter, according to an SEC filing.
Elamipretide is a peptide that easily penetrates cell membranes to target cardiolipin in the inner mitochondrial membrane. The phospholipid is produced in this membrane and is “almost exclusively” found there, the company says. Preclinical and clinical studies have shown elamipretide to increase mitochondrial respiration and improve the production of adenosine triphosphate, which provides energy for cell function.
“This elamipretide-cardiolipin association has been shown to normalize the structure of the inner mitochondrial membrane, thereby improving mitochondrial function,” Stealth Bio says. The company is also developing elamipretide for Barth syndrome, a rare disease characterized by an enlarged and weakened heart and weakened muscles, and Leber’s hereditary optic neuropathy, an inherited form of blindness.
Stealth Bio said in its SEC filing that the funds will also advance the preclinical drug SBT-272, which is being developed for neurodegenerative diseases, into phase 1, and push forward its pipeline of discovery compounds.
“In addition, our internal discovery platform has generated a library of over 100 proprietary, differentiated compounds which could have clinical benefit for diseases related to mitochondrial dysfunction and from which we plan to designate potential product candidates. We may also utilize certain of these compounds as part of our carrier platform, in which they could potentially serve as scaffolds to deliver other beneficial compounds to the mitochondria,” the company said in the filing.
Stealth Bio is not alone in the mitochondrial dysfunction space. Just over a year ago, Astellas snapped up its partner, MitoBridge, in a deal potentially worth $450 million. The centerpiece of the acquisition was MA-2011, a PPAR-delta modulator that is designed to reverse the mitochondrial defects that contribute to the progression of Duchenne muscular dystrophy.