Everyone is familiar with how quickly a big idea can become a big business – just think of Uber, or Afterpay, or even A2 Milk.
Most of us use and consume these goods and services, so we know just how quickly they can become household names.
Big ideas can also become big businesses in biotech – the only difference is, they might not quite become household names.
Dr. Ian Dixon, an engineer and biotechnology entrepreneur, had a big idea back in 2013.
Dixon was looking at regenerative medicine, and the promise of stem-cells. In particular, he was looking at the exosomes that stem-cells secrete.
And this was his Eureka moment.
All cells produce exosomes, which are an essential component of cell-to-cell signalling: they are small particles that deliver therapeutic ‘cargoes’ – such as protein and nucleic acids – to other cells to reduce inflammation and promote regeneration. Exosomes (also known as extra-cellular vesicles, or EVs) are plentiful in our youth but decline with age: one of the most important roles of exosomes is in slowing the ageing process.
Dixon realised that the exosomes released by stem cells could have a powerful rejuvenating effect on the cells that take them up.
In fact, he realised that the EVs produced by stem cells could be a simpler product – and eclipse the use of stem cells as medicines – for many practical reasons.
Back in 2013, the promise of stem cells as a new form of regenerative medicine was still on the rise. Doctors were excited about placing stem cells in the patient’s body and waiting for the cells to secrete the therapeutic material into the patient’s body.
But Dixon’s breakthrough was to realise that the exosomes/EVs were the therapeutic material from the stem cells – and that this active material itself could be extracted and placed in the patient’s body, thus accelerating the therapeutic effect.
Dixon reasoned that these exosomes/EVs could represent a completely new type of medicine.
But first he had to solve the problem of how to purify EVs as large-scale proprietary therapeutic products, but using manufacturing equipment that is standard in the bioprocessing industry. Dixon defined the problem, and what success would look like, as the first critical step. He could envision success: it would be a chromatography process, of the kind already used in typical GMP (Good Manufacturing Practice) drug manufacturing – which would ensure that it was inexpensive and scalable – but modified to both capture EVs, and, with a slight change in conditions, release them.
Having defined the problem very specifically, Dixon and his team worked over three years, with many failures. Persistence is a virtue (if it leads to success), and in 2016 Dixon lodged a patent application on a purification technique that could mass-produce exosomes/EVs. This technique, known as ligand-based exosome affinity purification (LEAP), is the central IP behind the ASX-listed company Exopharm.
Since patenting the LEAP technology in December 2016, further patent applications have been lodged and the technology and know-how has been further refined and improved. Today, Exopharm is a leader in EV purification for therapeutic products and has already commenced clinical trials with EVs from platelets.
In 2020 Exopharm in-licensed (exclusive worldwide and all uses) two technologies (called LOAD and EVPS) that enable it to design and manufacture a range of “engineered” EVs (or EEVs). EEVs use the natural properties of EVs as a way of delivering a ‘payload’ of drug (small-molecule compounds, nucleic acids etc) into selected cells, while avoiding the limitations of liposomes and other ‘artificial’ carriers that people have tried in the past. Researchers can load the drugs into the EEVs and targeting the EVs to certain cell types. EEVs are an ideal way to implement ‘precision medicines’ – one of the hottest fields in medicine today
Through LEAP, Exopharm is now a world leader in the purification of EV medicines, and is a clinical-stage company. Through holding the exclusive rights to LOAD and EVPS, Exopharm can make advanced EEV medicines.
Over the past few years, several companies in the EEV field have struck billion-dollar deals (for example, Codiak, EVOX, and Carmine). In the same vein, Exopharm is positioning itself for deals in the EEV field, in areas such as neurology, inflammation, cancer and anti-virus. Exopharm can design and make custom EEVs for partners, and is talking to a range of potential partners.
EVs are a far better way to harness the regenerative power of stem cells as a regenerative medicine than stem cells themselves. This science can be applied to a range of conditions where medicine is desperate for a new approach – for example stroke, cardiac disease, neuro-degeneration, auto-immune disease, Type 2 diabetes, acute respiratory stress disorder and arthritis. EVs are poised to become important new medicines in both regenerative medicine and precision medicine – each multi-billion-dollar areas.
As a serial biotechnology entrepreneur, Dr Dixon doesn’t just develop technologies or products – he wants to build long-lasting businesses based upon big ideas. Over the past three years, the Exopharm team has been carefully curated to bring in people with diverse backgrounds, skills and experience – successful biotechnology is very much a team sport. Further, Exopharm has sought, and in-licensed, extra intellectual property to ring-fence the business in the EEV field. Other IP is being developed in-house – solving problems that others haven’t even thought about yet.
That’s definitely a big idea, and the only listed pure-play exposure to it on the ASX is Exopharm.
