Elamipretide Study Does Not Meet Primary Endpoint

Stealth BioTherapeutics announced top-line data from the Phase 3 MMPOWER-3 trial evaluating elamipretide for treatment of patients with primary mitochondrial myopathy (PMM). The study did not meet its primary endpoints assessing changes in the six-minute walk test and Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) Total Fatigue Score. Safety results showed that treatment with elamipretide was well tolerated with most adverse events mild to moderate in severity.

"We are deeply grateful to our patients and families, our investigators and their teams, and our advocacy partners for their support of this study, and share their disappointment that it did not meet the promise of our earlier trials in this indication," said Chief Executive Officer Reenie McCarthy. "We remain confident in the promise of our platform and committed to our mission of improving the lives of people living with diseases involving mitochondrial dysfunction. We plan to meet with the FDA in early 2020 regarding our Barth syndrome program, where we have observed significant improvement in cardiac stroke volume during open-label extension, and continue to enroll our Phase 2b clinical trial in geographic atrophy associated with dry age-related macular degeneration, in which we observed improvement in visual function during an earlier Phase 1 study. We are also progressing our pipeline of second-generation mitochondrial therapeutics, with lead pipeline compound SBT-272 entering Phase 1."

The Company plans to review its operational resources to align them with its near-term priorities of progressing its Barth, AMD and pipeline programs, and expects to provide further guidance next month.

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