Rare Daily Staff

Stealth BioTherapeutics, a clinical-stage biotechnology company focused discovering, developing, and commercializing novel therapies for diseases involving mitochondrial dysfunction, said it has reached an agreement with Morningside Ventures for Morningside to provide $30 million of additional payments to the company during 2021 under its existing Development Funding Agreement.

That agreement was struck in April 2020 with Morningside advancing $20 million to Stealth Bio. The additional payments, which will be paid in three tranches, were triggered by new positive analyses of clinical data from the ReCLAIM phase 1 trial, which associated baseline mitochondrial viability with improvements in vision for patients with geographic atrophy.

Stealth Bio also previously announced a new data analysis from its MMPOWER-3 phase 3 clinical trial, which identified responders among patients with primary mitochondrial disease due to nuclear DNA mutations (nPMD) and plans to initiate a phase 3 clinical trial in patients with nPMD based on these results.

“We are encouraged that Morningside shares our enthusiasm about these recent findings from our geographic atrophy and nPMD programs, which we believe will allow us to enrich our future studies in these indications,” said Reenie McCarthy, CEO of Stealth. “This additional funding will allow us to initiate a phase 3 clinical trial evaluating the impact of elamipretide in patients with nPMD as well as continue our work on an intravitreal formulation to inform phase 3 formulation decisions as we await data from our ongoing ReCLAIM-2 trial in geographic atrophy early next year.”

Stealth Bio’s lead experimental candidate elamipretide targets the inner mitochondrial membrane where it associates with cardiolipin—the signature phospholipid of the inner mitochondrial membrane, which plays a role in many mitochondrial processes, including respiration and energy conversion. In preclinical and clinical studies, elamipretide was shown to increase mitochondrial respiration, improve the electron transport chain function and ATP production, and reduce formation of pathogenic reactive oxygen species levels.

The company is advancing elamipretide as a potential treatment for both rare metabolic cardiomyopathies, such as Barth syndrome, Duchenne muscular dystrophy, and Friedreich’s ataxia, as well as ophthalmic diseases entailing mitochondrial dysfunction, such as dry age-related macular degeneration and Leber’s hereditary optic neuropathy.

Stealth has received Rare Pediatric Disease, Fast Track, and Orphan Drug designation for elamipretide as a treatment for the ultra-rare condition Barth.

Photo: Reenie McCarthy, CEO of Stealth Bio

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