New Analysis Shows Correlation Between Ellipsoid Zone Dysfunction and Vision

BOSTON, May 23, 2022 /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, announced new data from ReCLAIM-2 Study of Elamipretide in Geographic Atrophy (GA) Secondary to Dry Age-Related Macular Degeneration (Dry AMD) in Clinical Trials at the Summit of Lake Tahoe, Nevada.

ReCLAIM-2 prespecified analyzes demonstrate that elamipretide ameliorated the progressive decline, or attenuation, of the mitochondria-rich ellipsoid zone (EZ) layer of photoreceptors (p 2 lines of low luminance visual acuity (LLVA) (p = 0.04) in patients with knee OA. Progressive LLVA dysfunction, which is one of the earliest symptoms of dry AMD, impairs patients’ ability to see in low light conditions such as at dusk, evening, or indoors using artificial light . Additional analyzes show that, consistent with previous results from the ReCLAIM Phase 1 study, changes in LLVA were correlated with baseline EZ attenuation in ReCLAIM-2. The new analyzes also show that the elamipretide-mediated reduction in EZ attenuation is correlated with improvements in LLVA. Although the primary endpoints of the trial, namely mean change in progression of LLVA and AG, were not met, these analyzes showed promise and supported and informed the development continuous use of elamipretide for dry AMD.

Dry AMD is a progressive retinal disease in which photoreceptors, which are specialized neurons found in the retina that convert light into electrical signals necessary for normal visual function, experience progressive damage and death, leading to progressive loss of vision. vision. One of the first signs of photoreceptor dysfunction is the progressive damage or attenuation of the ellipsoid zone (EZ), which is a mitochondria-rich layer of the photoreceptor. EZ attenuation has been shown to precede and predict visual dysfunction in dry AMD and other retinal diseases.

“Preserving the health of structures in the eye necessary for visual function, such as photoreceptors, is critical for patients with dry AMD,” said Jeffrey Heier, MD, Principal Investigator of the ReCLAIM-2 Study and Director of Retina Services and Director of Retina Research at Ophthalmic Consultants of Boston. “I am pleased to present these data highlighting for the first time the important relationship between mitochondrial health and vision in knee OA, which we hope will inform future efforts to improve outcomes for patients living with this disease. progressive and irreversible.

Elamipretide was generally well tolerated in ReCLAIM-2. The rate of recent onset exudations was 5.3% in the elamipretide-treated group versus 6.9% in the placebo group.

“We are delighted to confirm that targeting mitochondrial dysfunction in dry AMD can preserve and potentially improve visual function in some affected patients,” said Reenie McCarthy, CEO of Stealth BioTherapeutics. “We believe this data can help enrich and inform future development efforts for this devastating disease, and we are evaluating strategies to advance this agenda, including through potential partnership discussions.”

The company is also progressing the clinical development of elamipretide in several rare disease indications and is advancing a wide range of novel compounds targeting mitochondria.

About Dry AMD

Dry AMD is a leading cause of blindness affecting more than 5 million people worldwide and can seriously impair visual function, independence and quality of life.

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 almost every cell in the body, are the body’s main source of energy production and are essential for the normal functioning of organs. Dysfunctional mitochondria characterize a number of rare genetic diseases and are implicated in many common age-related diseases, typically involving energy-intensive organ systems such as the eye, neuromuscular system, heart, and brain. brain. We believe that our lead product candidate, elamipretide, has the potential to treat ophthalmic diseases resulting in mitochondrial dysfunction, such as dry AMD, rare neuromuscular disorders, such as primary mitochondrial myopathy and Duchenne muscular dystrophy, and rare cardiomyopathies, such as Barth syndrome. We are evaluating our second-generation clinical-stage candidate, SBT-272, for rare neurological disease indications, such as amyotrophic lateral sclerosis and frontotemporal dementia, following promising preclinical data. We have optimized our discovery platform to identify novel mitochondria-targeting compounds that can be designated as drug candidates or used as mitochondria-targeting vectors 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. These forward-looking statements include those regarding Stealth BioTherapeutics’ expectations regarding its clinical development of elamipretide for AG associated with dry AMD and other indications, the potential benefits of elamipretide and potential regulatory interactions. 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 such identifying words. Stealth BioTherapeutics may not achieve the plans, intentions or expectations disclosed in such forward-looking statements, and you should not place undue reliance on such forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements due to known and unknown risks, uncertainties and other important factors, including: Stealth BioTherapeutics’ ability to obtain additional financing and to continue a business as a going concern; the impact of the COVID-19 pandemic; the ability to successfully demonstrate the efficacy and safety of Stealth BioTherapeutics’ and future product candidates; preclinical and clinical results of Stealth BioTherapeutics’ product candidates, which may not support further development and marketing approval; the potential benefits of Stealth BioTherapeutics’ product candidates; the content and timing of decisions made by the FDA, EMA or other regulatory authorities, research review boards at clinical trial sites and publication review bodies, which may affect the launch , the timing and progress of preclinical studies and clinical trials of Stealth BioTherapeutics’ product candidates; Stealth BioTherapeutics’ ability to obtain and maintain required regulatory approvals and enroll patients in its planned clinical trials; unforeseen cash requirements and expenditures; competitive factors; Stealth BioTherapeutics’ ability to obtain, maintain and enforce patent and other intellectual property protections for any product candidates it develops; and general economic and market conditions. These and other risks are described in greater detail under the heading “Risk Factors” included in 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 undertakes no obligation to update any forward-looking statements we have made to reflect subsequent events or circumstances.

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SOURCE Stealth BioTherapeutics Inc.