Stealth BioTherapeutics Receives Orphan Drug Designation from FDA for Elamipretide for the Treatment of Friedreich's Ataxia

Phase 2a clinical trial in Friedreich's ataxia has been initiated and is currently recruiting patients

BOSTON, March 28, 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 today that the US Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to elamipretide for the treatment of patients with Friedreich's ataxia. 

A phase 2a investigator-initiated trial evaluating elamipretide for the treatment of Friedreich's ataxia has been initiated at Children's Hospital of Philadelphia (CHOP) under the direction of Dr. David Lynch. The trial is evaluating two doses of elamipretide in patients with Friedreich's ataxia to assess safety, visual function, and cardiac function. 

Friedreich's ataxia is a rare genetic disease affecting an estimated 4,000 individuals in the United States for which there are no FDA-approved treatments. The disease is caused by a defect in the frataxin gene resulting in a relative deficiency of frataxin, leading to mitochondrial iron accumulation and oxidative stress. Most patients with Friedreich's ataxia experience a progressive decline in visual function which impairs their visual quality of life. Progressive cardiomyopathy, which affects more than 90% of individuals living with this disease, usually becomes fatal by early adulthood and is the leading cause of early mortality. Elamipretide has been shown to improve frataxin levels in Friedreich's ataxia patient-derived cells and to improve frataxin levels and motor and cardiac function in an animal model of Friedreich's ataxia. 

"We are pleased that the FDA has recognized the high unmet need for innovative treatments for Friedreich's ataxia," said Chief Executive Officer Reenie McCarthy. "We are delighted to be working closely with Dr. Lynch and the Children's Hospital of Pennsylvania to evaluate elamipretide as a potential treatment for the progressive visual dysfunction and cardiomyopathy that affects visual quality of life and lifespan for individuals affected with this disease."

The Orphan Drug Act was enacted in 1983 to encourage development of drugs for rare diseases, which are diseases that affect fewer than 200,000 persons in the United States. Once granted, Orphan Drug Designation provides various development benefits for an investigational drug, including seven-year exclusivity after marketing approval is received.

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 eye, the neuromuscular system, the heart and the brain. We believe our lead product candidate, elamipretide, has the potential to treat ophthalmic diseases entailing 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 lobar dementia, 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 mitochondria-targeted 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. Such forward-looking statements include those regarding Stealth BioTherapeutics' expectations for elamipretide preclinical data and clinical development efforts in Friedreich's ataxia. 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 and to continue as a going concern; the impact of the COVID-19 pandemic; 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 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
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SOURCE Stealth BioTherapeutics Inc.