Founded less than a decade ago with a mission to harness the unique properties of exosomes as a new class of medicine, Aussie-listed Exopharm has become a trailblazer in the exosome regenerative medical field.
Australian technologist Dr Ian Dixon founded Exopharm (ASX:EX1) in 2013 with the foresight to recognise the exosome potential to revolutionise regenerative medicine, calling it the “secret sauce” to stem cells.
Exosomes are nano-vesicles secreted by cells for intercellular signaling and – wonderfully – they’ve been found to stimulate tissue regeneration.
First discovered in the 1980s, subsequent work has made it clear exosomes play a role across many aspects of physiology and disease, opening up the opportunity to explore intercellular communication.
The promise of these tiny vesicles to deliver target medicine and even combat age-related diseases, has scientists, governments and entrepreneurs around the globe a tad excited.
The challenge in 2022? Purifying these exosomes so they can be made in sufficient scale to allow for a variety – an almost infinite variety – of medical applications.
Exopharm takes a LEAP
Based out of its Melbourne labs, Exopharm’s team have been working day and night to solve the crucial bottleneck in the production of exosomes.
After a series of setbacks (and a lot of blood, sweat and tears) the Exopharm team discovered a way to load liquids containing exosomes through a chromatography column, a device used to separate chemical compounds.
Loaded with an inexpensive polymer charged to attract exosomes, they are retained while remaining biological detritus is simply washed away.
Pure LEAP
Exopharm named the purification process LEAP – Ligand-based Exosome Affinity Purification – and it now has a US patent for its key exosome manufacturing technology.
First-in-human clinical trials demonstrating the safety of its exosome products have also been successful.
The LEAP technology has four key advantages over other approaches:-
- it appears to capture the vesicles by binding to the membrane of the vesicle (and not specific molecules ‘decorating’ the vesicle);
- the ligand itself is not expensive to make;
- it gives Exopharm a proprietary step in the process; and,
- it uses affinity chromatography equipment – which is a standard technique in biomanufacturing.
The product is the process – LEAP gives Exopharm exclusivity
The LEAP technology is referred to as a ‘platform technology’ – because the one technology can be used to make a range of products. Platform technologies can be especially important and valuable once a few example products have derisked the platform.
Exopharm’s LEAP platform technology opens the way to manufacturing a range of products for treating a broad range of clinical indications.
Primed for growth
“In 2022, excitement around exosomes has converged on exosomes as an alternative to lipid nanoparticles (LNPs) and adeno-associated viral vectors (AAVs) for targeted multi-dose delivery of modern medicines and vaccines,” Dixon said.
Drug delivery accounts for a significant slice of the global pharmaceutical industry spend, around US$160 billion annually and growing strongly.
“Exosomes represent nature’s way to deliver a bioactive cargo into cells efficiently and safely,” Dixon said.
His establishment of a specialist exosome medicines company has been followed by US based Codiak Biosciences in 2015 (established by Arch Ventures) and Oxford-based Evox Therapeutics in 2016.
Today only two exosome-specific companies are publicly listed – Codiak (NASDAQ:CDAK) with a market cap of around US$200 million and Exopharm with a market cap of 73 million.
Financiers and pharmaceutical industry leaders are also starting to take notice of exosome medicine.
“In late 2021, multinational pharmaceutical manufacturing services company Lonza jumped into exosome manufacture, a sign that they see big things over the horizon,” Dixon said.
True to vision
Since its establishment, Exopharm has grown and evolved – now boasting a team of more than 40 of some of the best minds in biotech. In addition to LEAP the company has established exosome analytical tools (Exoria) and technologies to load exosomes with a specific therapeutic cargo (LOAD) and target exosomes to select cells (EVPS).
Exopharm is pursuing a pipeline driven platform technology strategy, on the way to generating a revenue stream from technology out-licensing while developing its own exosome medicines for selected markets.
The company is also becoming more international in focus, improving on messaging and communications with the wider pharmaceutical industry. Exopharm has signed a deal with the Astellas Institute for Regenerative Medicine (AIRM), a subsidiary of pharmaceutical heavyweight Astellas Pharma Inc.
The aim is to validate the company’s LEAP, LOAD and EVPS technology platforms to manufacture exosomes for Astellas.
In 2021 Dr Jenn King was appointed to the Exopharm board, bringing with her a wealth of big pharma and US biotech experience.
“In 2022 we look to engage with more experts, as members of our advisory panels, as consultants, executives or key opinion leaders,” Dixon said.
But through all the changes and developments the founder remains true to his vision for Exopharm – to harness the power of exosomes for the benefit of us all.
This article was developed in collaboration with Exopharm, a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.