Stealth BioTherapeutics Showcases New Duchenne Muscular Dystrophy Nonclinical Data at the 2022 Muscular Dystrophy Association and Clinical and Scientific Conference
BOSTON, March 16, 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, today presented new data demonstrating the potential synergistic relationship between elamipretide and an exon skipping phosphorodiamidate morpholino oligomer (PMO) in the X-linked muscular dystrophy (mdx) mouse model. The data were presented at the 2022 Muscular Dystrophy Association and Clinical and Scientific Conference, held in Nashville, TN.
Mitochondrial dysfunction has been observed early in the progression of Duchenne muscular dystrophy (DMD) and contributes to impaired energy homeostasis and inflammatory signaling in DMD. As PMO efficacy has been shown to be energy-dependent, the study tested the hypothesis that improving mitochondrial function with the mitochondria-targeting investigational new product elamipretide would enhance PMO efficacy. Results from the 7-week study demonstrated that the combination of weekly PMO and daily elamipretide therapies produced more than double the mean level of dystrophin protein in muscles of affected mice compared to treatment with PMO alone. These data highlight combination therapy of a PMO and elamipretide as a potential treatment for patients with DMD.
"We believe that improved mitochondrial health may lead to better outcomes in DMD, where dystrophin deficiency and mitochondrial dysfunction contribute to the devastating and progressive decline in muscle and cardiac function for young patients," said Reenie McCarthy, Chief Executive Officer of Stealth. "These data demonstrating the promise of combined elamipretide and PMO therapies offer early support for this premise, energizing our efforts to develop improved therapeutic options for DMD patients."
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 age-related macular degeneration, rare neuromuscular disorders, such as primary mitochondrial myopathy and DMD, 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-targeted compounds which may be nominated as therapeutic product candidates or utilized as mitochondria-targeted vectors to deliver other compounds to mitochondria.
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 preclinical data in models of DMD. 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.
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Company Codes: NASDAQ-NMS:MITO