With a rapidly growing global interest in the superpower of exosomes, major pharmaceutical deals and foundational patents, Exopharm is poised to become an essential part of the modern medicine ecosystem.

As the only listed exosome company on the ASX, and one of a small number of exosome companies listed globally, Australian biopharma Exopharm (ASX:EX1) is focused on accelerating its lead in the burgeoning field of exosome-supported modern medicines.

Serial entrepreneur, biomedical engineer, and inventor Dr Ian Dixon founded Exopharm in 2013 to advance exosome medicines by harnessing the natural ability of exosomes to specifically, safely and reliably deliver drugs anywhere in the body.

Fast forward nearly a decade, and Dr Dixon and his team at Exopharm are leading this innovative new class of medicine.

Exopharm’s discoveries in science and engineering enable surface-engineering of exosomes for select-tissue targeting, cargo loading of exosomes for immune-silent drug delivery, and commercial-scale purification and manufacturing of exosomes. Together these exosome technologies fuel the conversion from promise to reality for a range of innovative medicines such as additive gene therapies, gene editing and mRNA therapies.

LEAP over industry bottleneck

Exosomes are naturally occurring nano-vesicles that are secreted in huge numbers by cells throughout the human body and are a natural and powerful way that cells communicate and co-ordinate.

Within the last 7 years, researchers and entrepreneurs like Dr Dixon envisaged using exosomes as delivery vehicles inside a person’s body – like programmable, targeted and safe nano-sized drones. Drugs that had delivery problems, like mRNA, DNA and some small molecules, could be packaged into exosomes at a bioprocessing facility to produce modern medicines which were safer, more tolerable, and more specific to the target tissue/destination than their cargo alone.

However, difficulty experienced to date in purifying therapeutic exosomes at large scale has limited the development of exosome medicines, leaving fully synthetic non-natural lipid nanoparticles (LNPs) to partially fill the gap in the interim.

Exopharm’s patented Ligand-based Exosome Affinity Purification (LEAP) technology solves this bottleneck by enabling commercial-scale, clinical-grade production of exosome medicines by Exopharm. LEAP bridges the manufacturing gap between medical needs and manufacturing-scale, so Exopharm’s LEAP technology could become an essential part of modern medicine development and complement the use of LNPs as drug delivery vehicles, especially in uses where LNPs are not suitable or have exhibited issues.

Armed with a commercial-scale LEAP technology platform, Exopharm is also developing a synergistic drug-loading capability (LOAD) that further enhances Exopharm’s essential value to the drug development industry. “Cargos can include modern medicines such as additive gene therapies, gene editing constructs, forms of RNA, proteins, and small and large molecule drugs” Dr. Dixon said. “With our LOAD technology platform and its growing capabilities, we aim to bring immune-silent, ‘Exo-drug’ delivery solutions to modern medicine development partners,” he added.

Rounding out Exopharm’s toolchest is its Extracellular Vesicle Positioning System (EVPS) technology platform. EVPS enables the addition of custom surface proteins to exosomes for targeting specific tissues. Such specificity can improve the safety and reduce the potential toxicity of the drug cargo.

Further strengthening the technology ‘economic moat’ of Exopharm, in December 2021 the Company announced its first and foundational US patent grant for the LEAP technology platform. The US patent came on the heels of a patent grant in Russia and is part of the broader IP strategy being implemented.

“Financial value in biotechnology comes from ring-fencing intellectual property (IP) and know-how and building an economic moat to protect the company’s competitive position.” Dr Dixon commented. “A global IP strategy built around multiple technologies is at the heart of Exopharm’s future value.” Dr. Dixon added.

Pharmaceutical deals & expanding technologies

Exopharm has been working with numerous pharmaceutical and biotechnology companies, each with ambitions of enabling or developing exosome medicines.

The most recent of these partnerships was announced in January with a division of Astellas Pharmaceutical Co. Ltd., from Japan. Exopharm announced a Master Collaboration Services Agreement (MSA) with the Astellas Institute for Regenerative Medicine (AIRM) to validate Exopharm’s LEAP, LOAD and EVPS technology platforms, with a view to the two companies potentially entering a commercial transaction to accelerate the inclusion of exosomes into the Astellas pipeline.

“Financial markets take notice of third-party validation, a potential sign of forthcoming value rerating,” Dr. Dixon said.

Exosome medicine pipeline

Owning and developing its own exosome medicine pipeline products has become a priority for Exopharm.

Dr Dixon said selection of Exopharm’s pipeline products is a strategic imperative for the Company and its Board. “It’s called Exopharm and not Exotech for a reason,” Dr Dixon observed. “Our pipeline products strategy will harness the ‘superpowers’ of exosomes in order to optimise the probability of technical and regulatory success when targeting disease areas of critical unmet medical need, rather than develop another set of me-too products,” Dr Dixon added. “We anticipate that implementing this strategy may result in a higher valuation commonly enjoyed by ‘product’ companies compared to ‘platform technology’ companies.”

As part of the Company’s product strategy, over 300 potential pipeline product opportunities have been assessed across major disease categories, such as genetic disorders, next-generation DNA products and mRNA therapeutics.

“The selected products will be a small number of strategically focused, high-value product candidates that Exopharm will pursue through clinical development and towards registration, either on our own undertaking or potentially in partnership, subject to stepwise success along the way” Dr Dixon added.

Dr Dixon said that Exopharm plans to take lead product candidates through the US FDA approval pathway, from proof-of-concept validation, pre-IND, and IND-enabled clinical trials and ultimately registration studies and market launch.

Exopharm – a drug delivery company

The past two years highlight the speed that modern medicines can become mainstream.

Modern medicines need smart delivery – and exosomes harness nature’s way to best deliver signals and materials into cells safely and naturally.

Many pharmaceutical companies are looking to use exosomes as an alternative to LNPs and AAVs, and have exosomes on their shopping list. “Commercial partnership discussions are progressing well, with the potential to embed our technologies into modern medicine development at leading pharma and biotech companies. The aim is to convert these relationships into income streams with the standard combination of upfront fees, development and sales milestones, and royalties,” Dr Dixon shared.

“Exopharm’s technologies, our growth strategy, and our team make EX1 an exciting company for investors looking for exposure to the future of modern medicine development,” Dr Dixon commented.

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.