Stealth BioTherapeutics to Participate in Upcoming Ultra-Rare Disease Events

BOSTON, Sept. 15, 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 announced that the company is participating in upcoming events to discuss challenges and opportunities in ultra-rare disease drug development.

Reenie McCarthy, Stealth's CEO, will be participating in "Rare Disease Research: A Prescription" hosted by STAT which will take place on Thursday, September 15th from 5:30 p.m.–9:00 p.m. ET at Convene located at 600 14th Street NW, Washington, DC.

Ms. McCarthy will also participate in a discussion on "Incentives, Investments & the Way Forward for Ultra Rare Disease Drug Development" at The Business of RARE Biotech Summit on Tuesday, September 20 from 9:00 a.m.–4:30 p.m. at the Hyatt Regency Capitol Hill located at 400 New Jersey Ave NW, Washington, DC.  A copy of the presentation will be available on the Investors & News section of Stealth's website at https://investor.stealthbt.com/.

These discussions are expected to include Stealth's clinical and regulatory development experience with Barth syndrome, an ultra-rare disease of cardiolipin deficiency. Stealth has recently met with the Division of Cardiology and Nephrology at the FDA to discuss data from Week 168 of its SPIBA-201 Part 2 open-label extension, which was the last visit completed by all SPIBA-201 Part 2 trial participants.  At that time point, after over 3.5 years of chronic elamipretide therapy, a >40% mean improvement from baseline in left ventricular stroke volume (p=0.007) and end diastolic volume (p=0.003) was observed. Also, subjects continued to show improved exercise tolerance on the six-minute walk test (>90-meter mean improvement from baseline; p=0.003), muscle strength (>60 newton mean improvement from baseline; p<0.0001) and other functional endpoints. The company has requested an additional meeting with the FDA to gain further clarity on a regulatory path forward. 

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 age-related macular degeneration, 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-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 clinical data and development efforts. 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 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

Kendall Investor Relations

Adam Bero, Ph.D.

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SOURCE Stealth BioTherapeutics Inc.