Investors Show Growing Enthusiasm in Exosome Biotech Stocks

Biotech and Exosome Investment Growth

Recent financial transactions highlight increasing biotech investment activity in the field of exosomes and extracellular vesicles. These naturally occurring packets of biomolecules could represent a new paradigm in healthcare.

Exopharm Limited (ASX-EX1) is one of a small number of Australian biotech stocks currently attracting investors in regenerative medicine, and its LEAP purification technology overcomes the main problem limiting commercialisation of therapeutic exosome products.

Exopharm is focused on the use of exosomes as a new form of regenerative medicine. The field is growing rapidly. The global Regenerative Medicine Market, valued at US$5,444 million in 2016, is estimated to reach US$39,325 million by 2023, registering a CAGR of 32.2% from 2017 to 2023. Asia-Pacific is expected to grow with a highest CAGR during the forecast period ¹.

While a number of the other companies are seeking to use exosomes as a delivery vehicle for active ingredients, Exopharm’s core exosome products are therapeutically active in their own right. Exopharm’s experimental products Plexaris and Exomeres, produced by human platelets or stem cells, are natural vesicles with inherent regenerative medicine properties.

Regenerative exosomes potentially offer a paradigm shift for the way we treat age-related diseases, explains Dr Ian Dixon, the founder of Exopharm Ltd.

“Most drugs only treat the symptoms and cannot resolve the underlying causes of medical conditions that adversely affect health span, or reverse the damage that has already occurred,” Dixon says.

“Research in the exosome field points to the potential of natural exosomes to harness and improve natural regenerative biological processes inside the body.”

 Biotech and Exosome Investment Around the World

A number of companies are progressing ‘engineered’ exosomes – using exosomes to deliver a cargo of active ingredient into cells.

Significant funding is being invested in the emerging exosome sector. Examples include the following:

• May ’19 – Codiak BioSciences Inc. lodged documentation to list on NASDAQ and reportedly to raise around US$80 million
• January ’19 – Codiak BioSciences expects to receive US$56 million upfront, up to US$200 million in milestone payments Jazz Pharmaceuticals
• September ’18 – Evox Therapeutics Ltd announced that it had raised £35.5 million in a Series B financing round
• September ’18 – ExoCoBio Inc. announced that it raised about US $27 million in its Series B funding
• July ‘18 – PureTech Health PLC entered into a collaboration with Roche to receive up to US$36 million and will be eligible to potentially receive development milestone payments of over US$1 billion

More details on the above transactions are provided here:

In May 2019, Codiak BioSciences Inc. lodged documentation to list on NASDAQ and reportedly to raise around US$80 million to commercialise their engEx Platform – a modular and flexible therapeutic exosome that can be engineered to include and carry specific biologically active entities, including small molecules, proteins, antibodies, peptides and nucleic acids.

The company’s two lead engEx product candidates are exoSTING and exoIL-12. Codiak has previously raised around US$168 million in capital from prominent investors². The IPO valuation has not yet been announced.

In January 2019, Jazz Pharmaceuticals and Codiak BioSciences announced a strategic collaboration to commercialize engineered exosomes to treat five currently-undruggable cancer targets³.

Codiak expects to receive US$56 million upfront, up to US$200 million in milestone payments for each of the five targets, additional preclinical development milestones of up to US$20 million, a co-commercialisation option on two products, and royalties on future net sales.

In September 2018 Evox Therapeutics Ltd announced that it had raised £35.5 million in a Series B financing round⁴ led by Redmile Group and significant contributions from GV (Google Ventures), Cowen Healthcare Investments, Panacea Healthcare Venture, Borealis Ventures, and some private investors.

The company is using the funding to advance its preclinical-stage exosome therapeutics pipeline towards the clinic. Evox is focused on developing exosomes for targeted delivery of a wide range of therapeutic molecules, including mRNA, peptides and monoclonal antibodies, leveraging exosomes’ ability to reach cells in the brain and other body compartments usually not accessible to drugs.

Also, in September 2018, ExoCoBio Inc. announced that it raised about US $27 million in its Series B funding⁵. Since it was established in January 2017, ExoCoBio has raised a total of around $46 million in investor funding. Series B money will be used to set up GMP-compliant exosome manufacturing facilities to produce exosome pharmaceuticals, to attract high-quality talents, and to secure additional business opportunities for commercialization in the cosmetics industry.

In July 2018 PureTech Health PLC announced that it had entered into a collaboration with Roche to advance PureTech’s milk-derived exosome platform technology for the oral administration of Roche’s antisense oligonucleotide platform⁶.

Under the terms of the agreement, PureTech Health will receive up to US$36 million and will be eligible to potentially receive development milestone payments of over US$1 billion and additional sales milestones and royalties for an undisclosed number of products.

Exopharm’s non-core Development Activities

In addition to its core programs, Exopharm is progressing other uses of its LEAP Technology in applications including exosome diagnostics and engineered exosomes. The aim is to add value and then seek partners for these non-core activities.

Exopharm in the News:

• Exosomes Heads to Wall Street
• Exopharm listed details: ASX information

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