Stealth BioTherapeutics Announces Dosing Of First Patient In Phase 2 Study Of Elamipretide For The Treatment Of Dry AMD

BOSTON, March 28, 2019 /PRNewswire/ -- Stealth BioTherapeutics (NASDAQ: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that it has dosed the first patient in ReCLAIM-2, a Phase 2 study of elamipretide in patients with dry age-related macular degeneration (AMD) with geographic atrophy.

"People with dry AMD can ultimately lose central vision. However, long before that point, they suffer from very impaired vision under low light conditions and at night. Both types of vision loss have a very negative impact on mobility and quality of life," said Dr. Scott Cousins, trial investigator and Professor of Ophthalmology and Director of the Duke University Center for Macular Diseases. "Given there are no therapies approved for this debilitating disease, we are eager to learn more about the potential of elamipretide in this setting."

In November 2018, the U.S. Food and Drug Administration granted Fast Track designation for elamipretide for patients with dry AMD with geographic atrophy.

AMD is estimated to affect over 10 million individuals in the United States and is the leading cause of blindness among older adults in the developed world. There are no treatments approved for the dry form of the disease, which accounts for approximately 90 percent of all cases.

"Mitochondrial dysfunction has been implicated in the pathogenesis of dry AMD, leading to early failure of the highly energy-dependent photoreceptors responsible for night vision," said Chief Executive Officer Reenie McCarthy. "We were pleased to see gains in patients' low light vision during our Phase 1 trial, suggesting that elamipretide may help improve visual function for patients suffering from this blinding disease. The initiation of ReCLAIM-2 is an important milestone in our efforts to combat this devastating disease of aging."

ReCLAIM-2 is a double-masked, placebo-controlled, multi-center clinical trial involving approximately 180 subjects with non-central geographic atrophy in at least one eye. Subjects will be randomized 2 to 1 to either elamipretide or placebo, and will receive a 40 mg once-daily injection of elamipretide or placebo for a 48-week period. The study will evaluate safety, efficacy and pharmacokinetics of subcutaneous injections of elamipretide, and its primary endpoints are safety and tolerability, and changes from baseline in low-luminance best-corrected visual acuity.

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

About Age-Related Macular Degeneration

Age-related macular degeneration (AMD), a progressive eye condition that is the leading cause of blindness in adults, and is estimated to affect approximately 10 million people in the U.S. AMD affects the center portion of the retina, called the macula, which is responsible for central vision and color perception. Although there are FDA-approved treatments for wet AMD, which affects approximately 10 percent of those suffering from the disease, there are no approved therapies for dry AMD. Dry AMD with geographic atrophy, an advanced form of dry AMD, is characterized by central blind spots leading to permanent loss of vision.  The disease is a major contributor to loss of independence and diminished quality of life in older persons.

About Stealth

We are a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body's main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases, collectively known as primary mitochondrial diseases, and are also involved in many common age-related diseases. We believe our lead product candidate, elamipretide, has the potential to treat both rare genetic and common age-related mitochondrial diseases. We are studying elamipretide in the following primary mitochondrial diseases: primary mitochondrial myopathy, Barth syndrome and Leber's hereditary optic neuropathy. We are also studying elamipretide in dry age-related macular degeneration. Our other pipeline candidates include SBT-20, which we are evaluating for rare peripheral neuropathies, and SBT-272, which we are evaluating for rare neurodegenerative disease indications. We have optimized our discovery platform to identify novel mitochondrial-targeted compounds, which may be nominated as therapeutic product candidates or utilized as scaffolds to deliver other compounds to mitochondria. Our mission is to be the leader in mitochondrial medicine, and we have assembled a highly experienced management team, board of directors and group of scientific advisors to help us achieve this mission.

Forward-Looking Statements

This press release includes certain disclosures which contain "forward-looking statements," including, without limitation, statements regarding the anticipated development and commercialization of elamipretide. You can identify forward-looking statements because they contain words such as "believes," "goal" and "expects." Forward-looking statements are based on Stealth's current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Stealth's clinical trial protocol.

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SOURCE Stealth BioTherapeutics