Exopharm (ASX: EX1) is a clinical-stage company at the forefront of developing transformative medicines using exosomes (EVs).
EVs (short for extracellular vesicles, also known as exosomes) can be used in two main ways as new medicines – as precision medicine delivery vehicles (‘engineered EVs’) and as regenerative medicines (‘naïve EVs’) to mimic the power of stem cells without the problems associated with cell-based therapies.
Exopharm was founded in 2013 by Dr Ian Dixon, a successful entrepreneur in the cell and cell-derived therapy space. Ian co-founded the ASX-listed stem cell therapy developer Cynata Therapeutics, and was a director of Cell Therapies, which produced adult stem cells for the ASX-listed stem cell company Mesoblast.
Listed on the ASX in December 2018, Exopharm was the first publicly traded pure play EV medicine company in any major capital market and now has a market cap of around A$98m [as at 12th February 2021]. Engineered EV company Codiak Biosciences recently listed on the NASDAQ and currently has a market capitalisation of US$470m (A$610m) [as at 12th February 2021]. Like Codiak, Exopharm is a platform technology company, not a single product company. Exopharm has invented and inlicensed multiple EV technologies. Using these technologies, Exopharm’s aim is to do multiple deals across technologies and products, enabling multiple shots at goal and the prospect of becoming cashflow positive in the near term
Extracellular vesicles (also known as exosomes) are nano-sized vesicles naturally released by virtually every cell in the body. They are a key way that cells communicate and co-ordinate regeneration and homeostasis (balance). EVs carry various types of molecules (e.g. mRNA, miRNA, proteins, enzymes, lipids, DNA) to local and remote sites in our bodies.
In 2007, seminal work from Dr Jan Lötvall’s group at University of Göthenburg provided the first hint that EVs were more than inert, cup-shaped particles containing cellular waste. The researchers demonstrated that these membranous structures can shuttle functional mRNA and microRNA (miRNA) between different cells and ‘reprogram’ recipient cells.
It is also now known that EVs can target certain cell or tissue types – a phenomenon called ‘tropism’. Thus, endogenous (from our body) EVs are nature’s way to deliver specific messages to certain cells in our bodies, cells in the brain for example.
Companies like Exopharm are developing EV medical products to harness these unique and powerful attributes inherent to EVs.
EVs can be produced outside of the body in a biomanufacturing facility by purifying the EVs from the secretome of stem cells, or other cells. These exogenous EVs can then be used as EV medicines to either ‘top up’ missing EVs in the aging body to promote regeneration or can be engineered (i.e. loaded, modified or enhanced) to deliver therapeutic payloads to specific tissues.
EVs offer a new paradigm for the treatment of diseases, with EV medicines able to be grouped into Engineered EVs (EEVs) or Naïve EVs (NEVs).
EEV products harness the powerful natural ability of EVs to efficiently target cells and transfer selected materials into cells and across barriers (e.g. the blood brain barrier). Such drug materials can be RNA (e.g. mRNA, siRNA, RNAi, miRNA), enzymes, large molecules or small molecules. Because of their cellular provenance, EEVs avoid critical limitations of other synthetic delivery vehicles such as liposomes and can be designed to treat a wide scope of medical problems including neurological diseases, infectious diseases, cancer and fibrosis.
Exopharm’s EEV products include Fortrexo™ for the treatment of viral infection, and Cognevo™ for neurological disease. These products use different engineering technologies that Exopharm licenses exclusively. Watch this webinar which explains, in laymans terms, how Exopharm has developed Fortrexo.
Naive EVs (NEVs), which are the unmodified, naturally secreted EVs collected from adult stem cells, particularly mesenchymal stromal cells (MSCs), have demonstrated immunomodulatory and regenerative effects. NEVs may replace MSC therapies in the regenerative medicine field in the future.
Exopharm’s NEV products include Plexaris™ (blood platelet-derived EVs) for the treatment of wounds, and Cevaris™ (adult stem cell-derived EVs) for mobility and sensory applications including osteoarthritis and hearing loss. Plexaris is Exopharm’s most advanced program, with a Phase I clinical safety trial underway and expected to be completed in Q1 of CY 2021. Exopharm was the first company in the world to commence an in-human trial using EVs and is a leader in the NEV space.
Despite the promise of administering exosomes as a new form of medicine, the development of therapeutic EV products has been held back by one key technological challenge: an efficient and proprietary EV purification technology that would enable the manufacture of EV-based medicines at clinical and then pharmaceutical grade and scale.
Exopharm’s LEAP technology solves this manufacturing problem with a key step in the downstream manufacturing process that has been demonstrated to isolate and purify EVs from a variety of cell sources. LEAP is a proprietary affinity-based purification method that works by selectively recognising and binding the surface of EVs. Affinity purification techniques are widely used by the biopharmaceutical industry and are demonstrably scalable.
As highlighted at the Exosome Based Therapeutic Development Summit in late 2020, Exopharm’s LEAP is the only technology globally which can purify EVs at scale using a fully proprietary step in an orthogonal flow-through process.
Exopharm’s LEAP technology is a leader in both operating cost and scalability
The LEAP technology, owned by Exopharm, is subject to multiple patent applications and is progressing through national phases in 13 countries.
LEAP technology has provided access to high-purity exosomes and has launched a virtuous circle of innovation at Exopharm, allowing it to solve other manufacturing and characterization challenges and to bring an exosome-based medicine into human clinical trials for the first time. This places Exopharm at the forefront of this emerging field worldwide.
For emerging EV medicines, Exopharm’s technologies and know-how provide a palette of potential for Exopharm and its partners.
|Exopharm’s Technology||Technology Enables||Status and Ownership|
|LEAP||Enables purification of EVs at scale||Patents going through national phase in USA and 12 other countries, 100% owned by Exopharm|
|LOAD||Loads Ribonucleic Acids (RNAs) into EVs to be delivered as a payload to cells as part of a treatment regime||Exclusive worldwide license of patent-applied-for technology|
|EVPS||Add proteins to EVs surface to customize which cells EVs enter – provides ‘tropism’ to EVs||Exclusive worldwide license of patent-applied-for technology|
|EXORIA||Proprietary fluorescent dye which tracks how EVs migrate and deliver their payload into cells. Can also be used in EV analytics||Patent application lodged in late 2020, 100% owned by Exopharm|
Exopharm also has other know-how and IP that it protects.
Why is Exopharm a Leader in the EV Medicine Field?
Exopharm is unique amongst the emerging companies in the EV field as it has combined:
Exopharm is unique in having each of proprietary EEV targeting technology, EEV protein loading technology, EEV RNA loading technology and proprietary scalable manufacturing (purification) technology
Exopharm is one of a handful of companies with comprehensive technologies in the EEV medicine field – and large deals with pharmaceutical companies are already being done for EEV technologies.
EEVs are interesting to pharmaceutical companies that have invested in potential products but have seen delivery problems with these assets, for example unable to cross the blood-brain barrier (‘stranded assets’). Partnering with an EEV company could be a way to rescue stranded assets – using EEVs to overcome limitations such as crossing the blood-brain barrier, stability in the body and the need for tissue targeting.
Exopharm is one of three companies that have announced proprietary platforms for EEV product development – and the other two companies have done significant partnering deals over the past 24 months
Codiak Biosciences Inc. listed on NASDAQ in 2020 (CDAK) and has a market capitalisation of around A$610m at 12th February 2021. Exopharm’s market capitalisation is around A$98m as of 12th February 2021. The other main EV company, EVOX, is unlisted.
Exopharm’s strategy is to enter into multiple partnership/out-licensing deals with partners – deals that typically involve upfront fees, milestone fees and royalties on product sales.
Exopharm is actively discussing product and technology deals.
Exopharm’s technology platforms place the company in an ideal position to do multiple deals on multiple products with companies interested in developing EVs as new medicines. Its near term goal is to become revenue generating and then cash flow positive.
Exopharm has a team of 41 people dedicated to its programs and technologies. The team is based at the Baker Institute (Commercial Road, Melbourne) as a commercial tenant.
The team covers all aspects of biotechnology, including commercial (deals), finance, manufacture and process development, analytics, innovation and product innovation & operations.
In addition, Exopharm has:
Dr Ian E Dixon – Founder and Managing Director
PhD, MBA, MAICD
Dr Ian Dixon has a PhD in biomedical engineering from Monash University, an MBA from Swinburne University and professional engineering qualifications.
Ian has been successful in translating technology challenges into valuable businesses and respected intellectual property.
Ian is a co-inventor of Exopharm’s LEAP manufacturing technology, a co-inventor of Fortrexo and a co-inventor of Exoria.
Ian brings to Exopharm an extensive technical, commercial and entrepreneurial background.
Before founding Exopharm in 2013, Ian co-founded Cynata Inc, the owner of the Cymerus stem cell technology now being commercialised by ASX-listed clinical-stage Cynata Therapeutics Ltd (ASX:CYP).
He is also non-executive director of NYRADA Inc. (ASX:NYR), co-inventor of Nyrada PCSK9 inhibitor drug NYX-330, and founder and investor of Cardio Therapeutics Pty Ltd.
Ian was previously a founding director of Sydney-based Noxopharm Ltd (ASX:NOX) and non-executive director of Melbourne unlisted company Cell Therapies Pty Ltd.
Jason M Watson – Non Executive Chairman
LL.B, B. Comm
Mr Jason Watson has board and advisory experience acting with small and medium-sized enterprises, research institutes and listed companies in the life sciences and other sectors. In particular, he has assisted companies in developing, commercialising and transacting technologies through significant biotechnology licensing deals.
Jason is principal of Elementary Law, a legal practice based in Melbourne, Australia. His practice focuses on assisting clients to achieve the best outcomes for their patents and innovations, including through corporate fundraising, protection strategies, licensing and commercialisation. In this capacity, Jason has been recognised in the Intellectual Asset Magazine Patent 1000 independent list of The World’s 1000 Leading Patent Professionals.
Jason has expertise in relation to complex transactions, including establishing multi-party engagements, research and consultancy contracts and negotiating and implementing clinical trial, licensing, assignment, manufacturing, shareholding and other commercial arrangements.
Jason has a Bachelor of Laws with Honours and a Bachelor of Commerce.
Elizabeth McGregor – Non Executive Director and Company Secretary
BA (Hons), MBA, FGIA
Elizabeth works for the Automic Group and is Company Secretary for a number of ASX listed entities. She has experience in various industries including investment management, e-commerce and biotechnology.
She is a Fellow of the Governance Institute of Australia, a Member of the Australian Institute of Company Directors and a NSW Justice of the Peace.
Chris Baldwin – Chief Commercial Officer
Dr Chris Baldwin received his PhD in Chemical Engineering from the University of Cambridge, while matriculated at Trinity College. Chris joined Exopharm in November 2019, bringing a wide range of global commercial experience to the company.
Most recently, Chris was responsible for Haemonetics’ largest and most profitable business unit across Asia Pacific, with P&L responsibilities from Taiwan to Pakistan. Previously responsible for all sales and marketing in Southeast Asia, Chris provided strategy leadership across Asia Pacific, including China.
Chris’ strategic focus and approach to management comes from his time at McKinsey and Company, where he served banking, pharmaceutical and NGO clients.
Healthcare became the focus of Chris’ career at New York Blood Center, where he was Executive Director for 8 years and responsible for the blood supply across the greater New York metropolitan area. He founded and currently serves as Chairman of APAC Bioplasma Solutions Pte, a company that helps blood services and hospitals make productive use of their excess donations.