Stealth Raises $100M to Advance Elamipretide as Treatment for Mitochondrial Diseases

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by Alice Melão |

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Entrada Therapeutics

Stealth BioTherapeutics announced that it has raised $100 million from investors to support the development of elamipretide and other compounds that might treat diseases involving mitochondria dysfunction.

“We are gratified by the support from these leading healthcare and venture capital investors,” Reenie McCarthy, CEO of Stealth, said in a press release.

Elamipretide is a small molecule that specifically targets mitochondria, restoring its capacity to produce energy while decreasing levels of damaging oxidative stress.

It has been designated an orphan drug by the U.S. Food and Drug Administration as a potential treatment of primary mitochondrial myopathy (PMM), and placed on fast track development for Barth syndrome.

Elamipretide is in clinical trials investigating its potential to treat Leber’s hereditary optic neuropathy (LHON), mitochondrial myopathy, Barth syndrome, and age-related macular degeneration.

“Data generated to date with elamipretide across a range of age-related and rare disease indications support our belief that mitochondrial medicine offers great promise to deliver novel and commercially successful treatments to address the unmet medical needs of patients worldwide,” McCarthy said.

With the money raised, Stealth plans to initiate a Phase 2b trial to further assess elamipretide as treatment for intermediate dry age-related macular degeneration.

The funds will also support further development of elamipretide’s Phase 2/3 clinical program in LHON (including NCT02693119), Stealth said in its release, and potential regulatory and commercial milestones that may follow the ongoing Phase 3 clinical trial in mitochondrial myopathy (NCT03323749). The therapy is also now in a Phase 2/3 study in patients with Barth syndrome (NCT03098797), and a Phase 1 study in age-related macular degeneration (NCT02848313).

The investors were led by Nan Fung Technology’s Pivotal Beta, and also included funds associated with Atlantis Investment Management, BVCF Management, CMBC Capital Holdings, Kingdon Capital, Ocean Equity Partners and Sagamore Investments, as well as existing investor Morningside Venture.

The managing partner of Pivotal, Vincent Cheung, will join Stealth’s board of directors, the release noted.

“Stealth’s novel mitochondrial therapeutic platform has the potential to not only address the unmet medical needs of many patients with rare mitochondrial disease, but also to disrupt the current treatment paradigms for age-related indications,” Cheung said. “We believe that Stealth has not just the talent, but also the vision to achieve great things.”