Stealth BioTherapeutics Completes Enrollment of Phase 2 Study in Dry Age-Related Macular Degeneration with Geographic Atrophy

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Feb 23, 2021, 07:30 ET

BOSTON, Feb. 23, 2021 /PRNewswire/ -- Stealth BioTherapeutics Corp (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 the company completed enrollment for ReCLAIM-2 (SPIAM-202), and expects top-line data in the first half of 2022. ReCLAIM-2 is a phase 2 randomized, double-masked, placebo-controlled study to evaluate the efficacy and pharmacokinetics of elamipretide in patients with dry age-related macular degeneration (AMD) with geographic atrophy (GA).

"Distorted vision, reduction in low luminance visual acuity, reduced visual acuity, and blurred vision are symptoms of dry AMD with geographic atrophy and have a negative impact on the quality of life of affected patients," said Dr. Jeff Heier, director of the retina service and retina research at Ophthalmic Consultants of Boston and Lead Investigator. "Data demonstrated in prior trials suggests elamipretide may be able to help our patients with dry AMD, and I was pleased to be able to offer our patients the opportunity to participate in this trial."

"We believe that mitochondria-targeted therapeutics offer a differentiated approach to treat diseases of aging, such as dry AMD, which significantly affects the independence of over a million elderly patients," said Reenie McCarthy, Stealth's CEO. "By targeting the bioenergetic deficits that drive pathology in this disease, we hope to improve visual function for affected patients. Completing enrollment of this trial represents a meaningful milestone in our mission to improve the health-span of patients suffering from diseases of aging and also fulfills the condition for additional funding ($10m) under the Development Funding Agreement announced in October 2020."

The ReCLAIM-2 study completed enrollment with 176 patients. The primary endpoint of the 48-week study will measure the low-luminance best-corrected visual acuity, which assesses visual function under low light conditions meant to represent dusk or indoor (artificial) lighting. Secondary functional endpoints are change in low-luminance reading acuity, best-corrected visual acuity (BCVA). Secondary imaging endpoints assessing the rate of progression of the disease include GA area as measured by fundus autofluorescence and optical coherence tomography.

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

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

About Age-Related Macular Degeneration

AMD, a progressive eye condition that is the leading cause of blindness in adults, 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 GA, 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 and are involved in many common age-related diseases, typically involving organ systems with high energy demands such as the heart, the eye, and the brain. We believe our lead product candidate, elamipretide, has the potential to treat both rare metabolic cardiomyopathies, such as Barth, Duchenne muscular dystrophy and Friedreich's ataxia, rare mitochondrial diseases entailing nuclear DNA mutations, such as POLG-related disorders, as well as ophthalmic diseases entailing mitochondrial dysfunction, such as dry AMD and Leber's hereditary optic neuropathy. We are evaluating our second-generation clinical-stage candidate, SBT-272, and our new series of small molecules, SBT-550, for rare neurological disease indications following promising preclinical data. We have optimized our discovery platform to identify novel mitochondria-targeted compounds which may be nominated as therapeutic product candidates or utilized as scaffolds to deliver other compounds to mitochondria.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding Stealth BioTherapeutics' plans, strategies and expectations for its preclinical and clinical advancement of its drug development programs, including its ongoing clinical trials of elamipretide and planned clinical trial of SBT-272; the potential benefits of Stealth BioTherapeutics' product candidates; its key milestones for 2021; its plans regarding future data presentations; its interactions with regulatory authorities; and its financial guidance regarding the period in which it will have capital available to fund its operations. Statements that are not historical facts, including statements about Stealth BioTherapeutics' beliefs, plans and expectations, are forward-looking statements. The words "anticipate," "expect," "hope," "plan," "potential," "possible," "will," "believe," "estimate," "intend," "may," "predict," "project," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Stealth BioTherapeutics may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of known and unknown risks, uncertainties and other important factors, including: Stealth BioTherapeutics' ability to obtain additional funding; the ability to successfully demonstrate the efficacy and safety of Stealth BioTherapeutics' product candidates and future product candidates; the preclinical and clinical results for Stealth BioTherapeutics' product candidates, which may not support further development and marketing approval; the potential advantages of Stealth BioTherapeutics' product candidates; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, which may affect the initiation, timing and progress of preclinical studies and clinical trials of Stealth BioTherapeutics product candidates; Stealth BioTherapeutics' ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Stealth BioTherapeutics' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in the Stealth BioTherapeutics' most recent Annual Report on Form 20-F filed with the Securities and Exchange Commission ("SEC"), as well as in any future filings with the SEC. Forward-looking statements represent management's current expectations and are inherently uncertain. Except as required by law, Stealth BioTherapeutics does not undertake any obligation to update forward-looking statements made by us to reflect subsequent events or circumstances.

Investor Relations
Stern Investor Relations
Janhavi Mohite, 212-362-1200
[email protected]

SOURCE Stealth BioTherapeutics Inc.