Stealth BioTherapeutics Initiates Phase 2 Study of Elamipretide in Primary Mitochondrial Disease

BOSTON, Sept. 12, 2016 /PRNewswire/ -- Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs to treat mitochondrial dysfunction, today announced the initiation of a longitudinal extension trial for evaluating elamipretide in primary mitochondrial disease. The study, MMPOWER-2, will be limited to patients who completed the initial MMPOWER Phase 2 study. Positive data from MMPOWER were announced in June 2016 showing statistically significant improvement in distance walked in six minutes.

"Patients with rare primary mitochondrial diseases have no FDA-approved treatment options to address their needs," said Stealth Chief Executive Officer Reenie McCarthy. "We're committed to helping fill these significant gaps in care through our study of elamipretide. Following the promising results from our Phase 2 MMPOWER study, we're happy to announce the initiation of MMPOWER-2, which will help us better understand the effects of longer treatment with elamipretide for these patients, who often face severe challenges completing even simple daily activities."

MMPOWER-2 is a randomized, double-blind, placebo-controlled crossover study to evaluate the safety, tolerability and efficacy of four weeks' treatment with once-daily subcutaneous injections of elamipretide in patients with genetically confirmed mitochondrial disease. All patients in this study have previously completed the MMPOWER study.

"We look forward to the results of this follow-on study and hope to use these results, along with the positive data from MMPOWER, to engage in discussions with regulators to develop the design for a Phase 3 trial in primary mitochondrial disease," said Stealth Chief Medical Officer Doug Weaver.

For additional information on MMPOWER, MMPOWER-2 or elamipretide, please refer to Stealth's website and ClinicalTrials.gov.

About MMPOWER-2
MMPOWER-2 is a Phase 2 randomized, double-blind, placebo-controlled crossover study to evaluate the safety, tolerability and efficacy of four weeks' treatment with once-daily subcutaneous (SC) injections of elamipretide in patients with genetically confirmed mitochondrial disease previously treated in the MMPOWER study. Genetic mitochondrial diseases are a diverse group of rare inherited disorders characterized by systemic mitochondrial dysfunction that impairs patient health and well-being.

Subjects treated in the MMPOWER-2 study will be randomized (1:1) to receive either four weeks' treatment with 40 mg elamipretide administered once daily SC in Treatment Period 1 followed by four weeks of treatment with placebo administered once daily SC in Treatment Period 2 or vice versa. The two treatment periods will be separated by a four-week washout period.

The primary endpoint of this study is change in distance walked in six minutes after four weeks. Secondary endpoints include safety and tolerability assessments, patient-reported outcomes and improvements in mobility and movement patterns.

About Elamipretide
Stealth's lead candidate, elamipretide, is an investigational drug with the potential to modify disease by preserving energetics and restoring normal energy production in mitochondria, while decreasing oxidative stress. The energetics from mitochondria maintain healthy physiology and prevent disease. This mitochondria-targeted candidate is being developed for both rare and common diseases.

Stealth BioTherapeutics: Leading Mitochondrial Medicine
Stealth BioTherapeutics is a privately held biopharmaceutical company committed to bringing mitochondrial therapies to patients to treat both rare and common diseases. Stealth's clinical development program is focused along several core therapeutic areas, including primary mitochondrial diseases, cardiorenal diseases and ophthalmic disorders. More information regarding Stealth and its pipeline is available at StealthBT.com.

Contacts
Media Relations
dna Communications
Kate Contreras, 617-520-7088
[email protected]

Investor Relations
Stern IR
Beth DelGiacco, 212-362-1200
[email protected]

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SOURCE Stealth BioTherapeutics