Another Australian first for Melbourne-based Exopharm as it finalized testing and lodged the complete specification for its disruptive LEAP technology – a further step in bringing extracellular vesicles as an acellular therapy to treat patients with degenerative conditions.

The medical need for innovative, safe and effective regenerative medicines have largely not been met by using cells such as adult stem cells – despite significant investment and decades of clinical trials.
Our aging population has problems such as osteoarthritis, chronic wounds, fibrosis, diabetes and vascular disease, and patient groups are pushing for new treatments to be brought forward. But meaningful progress has been too slow for thousands of patients in need.

Investors have also seen the promise in listed regenerative medicine companies – but have been mainly disappointed by progress in the field and have seen recent declines in share prices that reflect the poor progress being made by some promising companies.
Cellular therapies are where cells from the patient or a donor are injected into a patient to trigger regeneration, but these cellular treatments can have many problems such as rejection by the host, damaging fibrosis and high costs. More recently, researchers investigating the way that injected cells work inside the body have made discoveries that overturn the previous models – the cells themselves are not needed to trigger the regeneration, rather it is the secretions of the cells that have the main effects.

Dr Dixon was involved in the cell therapy field for more than a decade before taking on the challenge of acellular therapies in his new venture called Exopharm in 2013. “I saw that the promise of cell therapies just wasn’t being realized and the problems with cell therapies for regenerative medicine were escalating – it was time to think outside the square” said Dr Dixon.
Dr Ian Dixon and his team at Exopharm have been working on a disruptive technology that side-steps the problems and limitations of cellular therapy – harnessing extracellular vesicles produced by adult stem cells in a bioprocessing facility as an acellular (i.e. no cells) product.

Exopharm calls its acellular product Exomeres, and Exomeres are a patented form of extracellular vesicles produced by adult stem cells. Already extracellular vesicles have been shown to be safe and effective in studies across conditions such as kidney disease, fibrosis, wound healing, vascular disease and neurodegeneration.

“Even into 2018 many investors are puzzled by cellular therapies, how the product is made, how the cells might work and when products might shift from research and into sales and profit” said Dr Dixon. “Our work, and the recent work of academic researchers in the international extracellular vesicle field, shows that acellular vesicles as a medicine will disrupt the cell therapy field before it becomes mainstream, so smart investors need to spend some time to understand our story and decide which companies to support”.

Exopharm has worked for more than 3 years to solve the biggest problem in the field – how to isolate and purify in large-scale and pharmaceutical-grade the nano-sized Exomeres; this quest has resulted in the patent-applied-for LEAP technology.
“Solving this purification problem has taken our team over 3 years and was risky R&D – we have broken new ground. I was determined that we could solve this problem and our team has applied some really complex chemistry and innovative screening techniques – our first efforts failed big-time but we kept trying” said Dr Dixon. “We have been fortunate to have such dedicated and expert people in our biotechnology team, and access to equipment like electron microscopes located in Parkville precinct” said Dr Dixon.
Having further optimized and protected its LEAP technology, Exopharm is now moving to the next step in the commercialization of its product and preparing for clinical trials of Exomeres in age-related macular degeneration (AMD) in 2020 or earlier.
“We are seeing a number of long-established cellular therapy companies moving away from cellular products and into acellular vesicle products” said Dr Dixon. “However, each of these companies faces the challenge of how to isolate and purify vesicles secreted by their cells during cell culture – a problem our LEAP technology solves” said Dr Dixon.

Exopharm has also been approached by companies in China, Europe and USA wanting to use the LEAP technology under license, and discussions are underway. Exopharm aims to be a world-leader in the manufacture and use of extracellular vesicles derived from adult stem cells.

Exopharm is presently 94% owned by Dr Dixon and has been funded internally so far by his private investment company Altnia Group. “Exopharm is likely to become a listed company in 2018 and then transition to the USA once we have clinical trials underway.”

Dr Dixon is no stranger to the public markets. He is presently a director of anticancer company Noxopharm Ltd (ASX-NOX) and a director of medical device company Medigard Limited (ASX-MGZ); and previously a founder of Cynata Inc, a stem cell company which was acquired by Cynata Therapeutics Ltd (ASX-CYP) in 2013. Dr Dixon’s company Cardio Therapeutics Pty Ltd was acquired by Nyrada Ltd in December 2017 and aims to list in CY 2018.
This track record of innovation and commercialization is appealing to the investment community, because there are so many failures in the listed biotechnology sector.

Exopharm founder and CEO, Dr Ian Dixon, said: “Our mission is to bring therapeutic exosomes as a disruptive technology into large-scale clinical trials as soon as practicable; because there are thousands of patients with medical conditions awaiting improved treatments and we think stem cell derived exosomes promise a new paradigm in cell-free regenerative medicine that people are waiting for.”

About Exopharm Pty Ltd
Exopharm Pty Ltd is a privately held regenerative medicines company focused on bringing extracellular vesicle/exosome therapy to solve important unmet medical needs. Vesicles derived from adult stem cells and manufactured using Exopharm’s LEAP technology are called Exomeres. Exomere therapy is a completely new class of cell-free (acellular) drug. Exopharm has patented LEAP, a unique proprietary technology to isolate and purify intact Exomeres in large-scale in pharmaceutical-grade. Exopharm’s programs include pharma-grade (GMP) manufacturing and preclinical testing and then clinical trials in selected indications such as tendinopathy, age-related eye disease, fibrosis, joint disease, degeneration and ageing. Exopharm is partnering with thought-leaders in the field and is building strong relationships with researchers and clinicians in selected therapeutic areas.
For more information on Exopharm and Exomeres, please visit www.exopharm.com, follow on twitter (@Exopharm), or subscribe to our Newsletter at http://exo.ph/ExoMails.

For more information, please contact Dr Ian Dixon, CEO: +613 9894 4555, ian.dixon@exopharm.com