Press Release


Proceeds will support advancement of an exosome therapy to treat a urea cycle disorder

OXFORD, United Kingdom, 22 March 2019 – Evox Therapeutics Ltd (‘Evox’ or the ‘Company’), a leading exosome therapeutics company, today announces that it has secured £1.5 million in funding from Innovate UK, the UK’s Innovation Agency, through the Biomedical Catalyst Early Stage Award. The funds will be used to support the Company’s pre-clinical development of an exosome-based therapy to treat argininosuccinic aciduria (ASA), a rare life-threatening metabolic disease, in collaboration with University College London (UCL).

ASA is a rare genetic disorder characterised by deficiency or lack of the enzyme argininosuccinate lyase (ASL). ASL is central to two metabolic pathways: i) the liver-based urea cycle, which detoxifies ammonia, and ii) the citrulline-nitric oxide cycle, which synthesises nitric oxide from L-arginine. Patients with ASL deficiency can present either shortly after birth or later in life and are characterised by hyperammonaemia and a multi-organ disease with a severe neurological phenotype. ASA is the second most common urea cycle disorder and with merely symptomatic treatment available today; there is currently a significant unmet medical need for these patients.

Evox is engineering exosomes, the body’s natural vesicular delivery system, to enable a wide variety of drugs to reach previously inaccessible tissues and compartments, such as crossing the blood-brain barrier to deliver drugs to the central nervous system, enabling intracellular delivery of biologics, and allowing for extra-hepatic delivery of RNA therapeutics. UCL has world-leading expertise in urea cycle disorders and will perform testing of Evox’s exosome-based therapy in an in vivo ASL deficiency model.

Dr Per Lundin, Chief Operating Officer and Co-Founder of Evox, commented: “We are delighted that Innovate UK have chosen to support our work to develop an exosome-based therapy to treat patients with ASA. We intend to leverage the broad tissue distribution pattern of exosomes to deliver the fully functional enzyme to the liver and other key tissues, such as the central nervous system, in order to treat all aspects of the disease. We look forward to working with our academic collaborators at UCL who are at the forefront of developing pre-clinical models of the disease and treating patients.”

Dr Paul Gissen from the UCL Great Ormond Street Institute of Child Health said: “We are very excited to receive this collaborative grant with Evox from Innovate UK. It will allow us to test the potential effect of this novel exosome-based therapy in a model of severe metabolic disease, argininosuccinic aciduria. Patients with this disorder suffer from dysfunction of the liver, brain and other organs and are representative of a larger group of inherited diseases in which there is a huge unmet need for disease-specific treatments. Therefore, if successful, the same approach may be used to treat many other severe illnesses.”

For enquiries, please contact:

Dr Antonin de Fougerolles, Chief Executive Officer, Evox Therapeutics Limited +44 (0) 1865 570210

Dr Per Lundin, Co-Founder & Chief Operating Officer, Evox Therapeutics Limited +44 (0) 1865 570210

Brett Pollard / Natalie Garland-Collins, FTI Consulting +44 (0) 203727 1000

About Evox Therapeutics

Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on harnessing and engineering the natural delivery capabilities of extracellular vesicles, known as exosomes, to develop an entirely new class of therapeutics. Backed by leading life sciences venture capital groups and supported by a comprehensive intellectual property portfolio, Evox’s mission is to positively impact human health by creating novel exosome-based therapeutics for the treatment of various severe diseases with limited options for patients and their families. Evox has created substantial proprietary technology to modify exosomes using various molecular engineering, drug loading, and targeting strategies to facilitate targeted drug delivery to organs of interest, including the brain and the central nervous system. Exosome-based drugs have the potential to address some of the limitations of protein, antibody and nucleic acid-based therapies by enabling delivery to cells and tissues that are currently out of reach using other drug delivery technologies, and Evox is leading the development within this emerging therapeutic space.

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About Innovate UK

Innovate UK drives productivity and economic growth by supporting businesses to develop and realise the potential of new ideas. We connect businesses to the partners, customers, and investors that can help them turn ideas into commercially successful products and services and business growth. We fund business investment into R&D. Our support is available to businesses across all economic sectors, value chains, and UK regions. Innovate UK is part of UK Research and Innovation.

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About UCL (University College London)

UCL was founded in 1826. UCL was the first English university established after Oxford and Cambridge, the first to open up university education to those previously excluded from it, and the first to provide systematic teaching of law, architecture and medicine. UCL is among the world’s top universities, as reflected by performance in a range of international rankings and tables. UCL currently has over 39,000 students from 150 countries and over 12,500 staff. Our annual income is more than £1 billion.