Basildon, UK, May 30, 2023 (GLOBE NEWSWIRE) -- PRESS RELEASE - FOR BUSINESS & TRADE MEDIA ONLY
- THE AGREEMENT COVERS RIGHTS FOR ELAMIPRETIDE FOR BARTH SYNDROME IN THE EU, NORWAY, SWITZERLAND, UK, ICELAND, AND MIDDLE EAST & NORTH AFRICA (MENA) TERRITORIES
- THERE ARE NO CURRENT APPROVED TREATMENTS FOR BARTH SYNDROME
- THIRD NEW CHEMICAL ENTITY DEAL FOR PHARMANOVIA, EXPANDING PORTFOLIO IN CARDIOLOGY AND RARE DISEASES
Pharmanovia, a global pharmaceutical company that commercialises novel medicines and revitalises, extends and expands the lifecycle of established medicines, has today announced the expansion of its cardiology portfolio through a new licensing agreement with US-based biopharmaceutical company, Stealth BioTherapeutics Inc.
The agreement gives Pharmanovia the licensing rights to elamipretide for Barth syndrome in Europe and MENA. This is a significant milestone as ongoing studies indicate that elamipretide has the potential to improve the quality of life for people with Barth syndrome, a serious and rare condition characterised by life-threatening cardiomyopathy.i
Pharmanovia will work with Stealth on the final analysis of studies for elamipretide’s potential in this disease area. A randomised, double-blind, placebo-controlled Phase II/III trial followed by an open-label study,ii exploring the safety and effectiveness of elamipretide, and a Phase III natural history control study,iii comparing the effect of elamipretide to prognostically matched natural history controls, have already shown promising results. Orphan Drug designations from the FDAiv and EMAv have been granted, demonstrating the unmet need in this condition.
It is estimated that Barth syndrome affects 1 in 300,000 to 400,000 births, making this an ultra-rare disease. The condition can have a huge impact on an individual’s quality of life and ability to function day-to-day, with common signs and symptoms including cardiomyopathy, undeveloped skeletal muscles and muscle weakness, delayed growth and fatigue.i
Pharmanovia CEO, James Burt commented; “This partnership is our third new chemical entity in as many months, further broadening our treatment portfolio, as we set our sights on improving the experience and lives of patients globally.”
“We are delighted to have been recognised by Stealth BioTherapeutics as the right overseas partner to launch and potentially bring this novel treatment to patients in the EU, Switzerland, Norway, UK, Iceland and MENA. Beyond revitalising and adding value to iconic brands, utilising our extensive technical and commercial platform to address unmet needs with novel medicines that complement our existing portfolio across our core therapeutic areas, in this instance – cardiology, is a key strategic priority for us.”
“Only 5% or fewer rare diseases are estimated to have an approved treatment option,vi therefore we’re especially excited to work with authorities and regulators on potentially bringing to market the first specific treatment optionvii for those living with Barth syndrome.“
Reenie McCarthy, CEO of Stealth Biotherapeutics, added: “We are committed to broadening global access to elamipretide for patients living with Barth syndrome. Partnering with Pharmanovia is a natural choice to achieve that goal given their experience in bringing medicines to patients across Europe and MENA and our mutual alignment on elamipretide’s potential to address the unmet medical needs of patients living with Barth syndrome.”
-Ends-
Notes to editors
For further information, please contact:
Alison Dyson, Director of Communications, Pharmanovia
07442 256310/ Alison.dyson@pharmanovia.com
About Pharmanovia
Pharmanovia is a global lifecycle management healthcare company. Our purpose is to make medicines fit for tomorrow, to improve the lives of patients globally.
We do this by rediscovering, repurposing or re-engineering established medicines to improve patient outcomes and experiences as well as identifying new chemical entities that complement our existing portfolio to address unmet need.
With a diverse and growing team in over 160 countries across the globe, we deliver high-quality solutions, ethically and sustainably, across our four core therapeutic areas – Endocrinology, Neurology, Cardiovascular and Oncology.
About Stealth BioTherapeutics
Stealth BioTherapeutics is a clinical-stage biotechnology company focused on the discovery, development, and commercialisation 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 characterise 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. In addition to Barth syndrome, the Company has clinical-stage neuromuscular and ophthalmology programs in development and a deep pipeline of novel mitochondria-targeted compounds under evaluation as therapeutic product candidates.
About elamipretide
Elamipretide is an investigational mitochondrial protective agent that has been shown to normalise 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.vii Elamipretide readily penetrates cell membranes and transiently localises 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.viii
About Barth Syndrome
Barth Syndrome is an ultra-rare disease. It is estimated that 1 in 300,000-400,000 people are born with Barth Syndrome, but evidence of misdiagnosis is growing, with estimates this figure could be as high as 1 in 140,000. It is an X-linked, multi-organ system disorder characterised by cardiolipin deficiency, cardiomyopathy, musculoskeletal weakness, neutropenia, debilitating fatigue, growth delay, and hypoglycaemia, along with clinical manifestations common in inborn errors of metabolism. The life-threatening disease most commonly affects males but has been reported in females and is associated with genetic mutations in the tafazzin gene causing abnormal cardiolipin remodelling and impaired mitochondrial structure and function.i
References
I Clarke et al., 2013. Barth syndrome. Orphanet journal of rare diseases, 8(1), 1-17. https://doi.org/10.1186/1750-1172-8-23
ii Thompson et al., 2021. Genetics in Medicine, 23(3), 471-478. https://doi.org/10.1038/s41436-020-01006-8
iii Hornby et al., 2022. Barth syndrome. Orphanet journal of rare diseases, 17(1), 1-9. https://doi.org/10.1186/s13023-022-02469-5
iv https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=607817 - FDA designations and approvals, last accessed 25.05.23
v https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2430 - EMA website, last accessed 26.5.23
vi IRDiRC research - https://ec.europa.eu/research-and-innovation/en/horizon-magazine/building-blocks-make-rare-disease-treatments-more-common#:~:text=Indeed%2C%20only%205%25%20or%20fewer,known%20as%20%E2%80%9Corphan%E2%80%9D%20therapies - last accessed 26.05.23
vii National Institute of Neurological Disorders and Stroke. Barth syndrome. Available at: https://www.ninds.nih.gov/health-information/disorders/barth-syndrome - last accessed 26.5.2023
Vii Siegel et al., 2013. Aging cell, 12(5), 763-771. https://doi.org/10.1111/acel.12102; Eirin et al., 2014. Journal of hypertension, 32(1), 154. https://doi.org/10.1097%2FHJH.0b013e3283658a53; Birk et al., 2013. Journal of the American Society of Nephrology, 24(8), 1250-1261. https://doi.org/10.1681/asn.2012121216; Manczak et al., 2010. Journal of Alzheimer's Disease, 20(s2), S609-S631. 10.3233/JAD-2010-100564; Dai et al., 2013. Circulation: Heart Failure, 6(5), 1067-1076. https://doi.org/10.1161/circheartfailure.113.000406
viii Mitchell, et al., 2020. Journal of Biological Chemistry, 295(21), 7452-7469. https://doi.org/10.1074%2Fjbc.RA119.012094
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