Pharmanovia Obtains Exclusive License to Commercialize Elamipretide for the Treatment of Barth Syndrome in the European Union, United Kingdom, Middle East, and Northern Africa

NEEDHAM, Mass., May 30, 2023 /PRNewswire/ — Stealth BioTherapeutics Inc. (the “Company” or “Stealth BioTherapeutics”), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced their entry into an exclusive licensing agreement whereby Pharmanovia will commercialize elamipretide, Stealth’s lead investigational product candidate, in Europe and other key global territories for the treatment of Barth syndrome, an ultra-rare life-threatening cardiac condition caused by mitochondrial cardiolipin deficiency for which there are currently no approved treatments. Elamipretide, an investigational mitochondrial protective agent which has been shown to improve mitochondrial structure and function through its interaction with cardiolipin, has completed Phase 3 development as a potential treatment for Barth syndrome.

Under the terms of the agreement, Pharmanovia receives exclusive rights to commercialize elamipretide for the treatment of Barth syndrome in the European Union and other European countries (including Iceland, Norway, Switzerland, and the United Kingdom), the Middle East and Northern Africa (MENA, including Algeria, Bahrain, Djibouti, Egypt, Iraq, Jordan, Kuwait, Lebanon, Libya, Malta, Morocco, Oman, Qatar, Saudi Arabia, Tunisia, the United Arab Emirates, Palestine, and Yemen). In exchange, Stealth BioTherapeutics will receive upfront and future development, regulatory, and commercial milestone payments, plus tiered royalties on net sales of elamipretide in the licensed territories.

“While we work with FDA toward a potential US approval pathway, we remain committed to expanding access to elamipretide to individuals living with Barth syndrome in Europe and beyond” said Reenie McCarthy, Chief Executive Officer of Stealth. “As a global pharmaceutical company with a strong footprint in Europe and a resolute commitment to patients with rare diseases, Pharmanovia is an ideal partner to work with regulatory authorities in Europe and MENA to progress our shared goal of facilitating access to a potential first treatment option for this ultra-rare genetic cardiac condition.”

James Burt, Chief Executive Officer of Pharmanovia, added “We are delighted to be Stealth BioTherapeutics’ partner across EU, Switzerland, Norway, UK, Iceland and MENA. Only 5% of rare diseases have an approved treatment option, therefore we share a common goal of wanting to meet unmet patient needs and we’re excited to have the opportunity to bring to market a potential first specific treatment option for those living with Barth Syndrome.”

About Barth Syndrome
Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities often leading to heart failure and reduced life expectancy, exercise intolerance, muscle weakness and severe fatigue, recurrent infections, and delayed growth. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 200,000 to 400,000 individuals worldwide. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome.

About Elamipretide and Barth syndrome
Elamipretide is an investigational mitochondrial protective agent that has been shown in preclinical studies to normalize mitochondrial structure and function and improve cell viability and organ function across a spectrum of disease models, including models of cardiovascular, renal, metabolic, skeletal muscle, neurodegenerative, and genetic mitochondrial disease. Elamipretide readily penetrates cell membranes and transiently localizes to the inner membrane of the mitochondria, where it interacts with cardiolipin, which is known to be depleted in Barth syndrome, and monolysocardiolipin, which is known to be elevated in Barth syndrome. Elamipretide has been assessed in a Phase 2 clinical trial and open-label extension and a Phase 3 retrospective natural history control trial in Barth syndrome. The Phase 3 natural history control trial met its primary and key secondary endpoints, demonstrating significant improvements in six-minute walk test, muscle strength and balance compared to prognostically matched natural history controls. Recent data from the Phase 2 open-label extension demonstrated nominally significant improvements from baseline in left ventricular stroke volume, distance walked on the six-minute walk test, muscle strength and balance.

About Stealth
The Company is 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. The Company believes that its lead product candidate, elamipretide, has the potential to treat ophthalmic diseases, such as dry AMD, rare neuromuscular disorders, such as primary mitochondrial myopathy, and rare cardiomyopathies, such as Barth syndrome. The Company has a deep pipeline of novel mitochondria-targeted compounds under evaluation as therapeutic product candidates.

About Pharmanovia
Pharmanovia is a global lifecycle management healthcare company. Its mission is to revitalize iconic medicines for the benefit of patients, prescribers, and payors, and utilize their capabilities to launch novel therapies. With a diverse and growing team in over 160 countries across the globe, they deliver high-quality solutions, ethically and sustainably, across four core therapeutic areas – Oncology, Endocrinology, Neurology and Cardiovascular.

Investor Relations
Kendall Investor Relations
Adam Bero, Ph.D.
abero@kendallir.com
IR@StealthBT.com

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Anna Stallman Communications
Anna Stallman
anna@annacomms.com