Stealth BioTherapeutics has achieved target enrolment in the Phase III NuPOWER (SPIMD-301) clinical trial of elamipretide to treat primary mitochondrial myopathy (PMM).
The company plans to continue enrolment until the end of the month to allow interested patients to participate in the study.
The placebo-controlled, parallel-group, double-blind, randomised study is designed to evaluate the safety and efficacy of elamipretide in subjects with PMM associated with nuclear DNA mutations (nPMM).
They will receive a single daily dose of elamipretide subcutaneously for 48 weeks.
Change from baseline in distance walked on the six-minute test, which evaluates exercise tolerance, is the primary efficacy outcome of the study.
Secondary outcome measures include triple-timed up-and-go tests and changes from baseline in the time to complete the five times sit-to-stand.
Stealth BioTherapeutics CEO Reenie McCarthy said: “We are thrilled to progress our PMM development efforts toward pivotal data.
“We have partnered closely with the patient community since initiating this programme in 2014, and deeply appreciate the over 350 individuals living with PMM and the many dedicated scientists and clinicians who have participated in and helped design and conduct our clinical trials.
“This important milestone brings us closer to the goal of addressing the unmet need of individuals living with PMM, as we look forward to topline data next year.”
The company is also advancing its lead product candidate, elamipretide, into Phase III clinical trials in dry age-related macular degeneration and Barth syndrome, an ultra-rare cardioskeletal disease.
