Stealth BioTherapeutics Initiates Phase 1 Study of Elamipretide in Dry Age-Related Macular Degeneration

BOSTON, Nov. 14, 2016 /PRNewswire/ -- Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs to treat mitochondrial dysfunction, today announced the initiation of ReCLAIM, a Phase 1 study evaluating elamipretide in intermediate age-related macular degeneration (AMD). Top-line data are expected mid-year 2017.

"There are currently no FDA-approved treatment options for dry AMD, so we are eager to better understand the effect that elamipretide may have in treating these roughly 13 million patients," said Dr. Scott Cousins, the trial investigator, and Professor of Ophthalmology and Director of the Duke University Center for Macular Diseases. "Mitochondrial dysfunction that stems from various environmental toxins may be an important causative factor in dry AMD, and in laboratory models, elamipretide appears to prevent mitochondrial dysfunction in the retinal pigment epithelium."

ReCLAIM is an open-label study to evaluate the safety and tolerability of 12 weeks' treatment with daily subcutaneous injections of elamipretide in patients, age 55 and above who have at least one eye with intermediate AMD, and have either: a) high-risk protein deposits (drusen) on the retina without any geographic atrophy (GA), a characteristic of advanced AMD which can result in the loss of photoreceptor cells or b) GA with an unaffected central fovea (noncentral GA). The study's primary endpoints are safety and tolerability, and the secondary endpoints are changes from baseline in physical/ophthalmic examinations and feasibility of subcutaneous injections in this patient population.

"ReCLAIM will build upon our ongoing ophthalmic program to help us better understand the potential of elamipretide to treat back of the eye diseases as well as the effects of the treatment to slow the aging process in eye tissue," said Stealth Chief Executive Officer Reenie McCarthy. "We look forward to evaluating initial results of this study mid-year 2017."

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

About Age-Related Macular Degeneration
Age-related macular degeneration (AMD) is a progressive eye condition which affects approximately 15 million people in the U.S., and escalates with age. AMD causes severe vision loss because it attacks the macula of the eye, where the sharpest central vision occurs.

There are two types of AMD: wet and dry. Wet AMD affects only 10-15% of those with the condition and is characterized by the breaking of the membrane underlying the retina. Dry is the most common type of macular degeneration, but has no FDA-approved treatment options. Dry AMD is characterized by a breakdown or thinning of the retinal pigment epithelial cell layer in the macula – which supports the light sensitive photoreceptor cells critical to vision. There are three stages of AMD (early, intermediate and late) defined by the size and number of drusen under the retina, among other factors. Intermediate AMD currently affects up to eight million patients aged 40+ in the U.S.

About ReCLAIM
ReCLAIM is a Phase 1 open-label study to evaluate the safety and tolerability of twelve weeks' treatment with daily subcutaneous (SC) injections of elamipretide in patients with intermediate age-related macular degeneration.

Subjects eligible for the ReCLAIM study are age 55 and above, have at least one eye with intermediate AMD, and fall into one of the following two subgroups: a) a group with drusen on the retina but no GA or b) a group with noncentral GA. The study eye of eligible subjects must fall into one of the two subgroups and subjects must not have previously received antiangiogenic therapies.

The primary endpoints of this study are safety and tolerability. The secondary endpoints are changes from baseline in physical/ophthalmic assessments and feasibility of subcutaneous injections in this patient population.

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 diseases and common diseases of aging.

Stealth BioTherapeutics: Leading Mitochondrial Medicine
Stealth BioTherapeutics is a privately held biopharmaceutical company committed to bringing mitochondrial therapies to patients to treat both rare diseases and common diseases of aging. 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

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