The Japanese pharmaceutical company Takeda has struck a deal worth up to €803M with UK biotech Evox Therapeutics to develop rare disease treatments delivered to cells via nanocapsules called exosomes.
As part of the deal, Evox will use its proprietary technology platform to modify and load RNA and protein drugs into exosomes and target the delivery of these exosomes to organs of interest.
Evox’s CEO, Tony de Fougerolles, explained to me that exosomes are nanoparticles that act as natural transporters in the human body’s communication machinery. It is this mechanism that can be exploited to create novel biotherapeutics that can cross natural barriers in the body such as the blood-brain barrier.
“Exosomes can transport various types of biomacromolecules – for instance, protein and RNA therapeutics – but also various types of small molecule drugs from one cell to another,” de Fougerolles said.
“Exosome-based drugs have the potential to address some of the limitations of protein, antibody, and nucleic acid-based therapies by enabling delivery to cells and tissues that are currently out of reach using other drug delivery technologies.”
Takeda has bought into Evox’s exosome-based treatment for Niemann-Pick disease type C. This is a rare, heritable condition where cells are unable to process waste molecules, and the molecules build up around the body, causing a wide range of symptoms including seizures and disability.
With the investment from Takeda, de Fougerolles hopes that the project will advance into phase I testing within the next couple of years.
As part of the agreement, Evox will receive up to €40M in upfront, near-term milestone payments and research funding. As well as the Niemann-Pick disease type C program and a second new program targeting an undisclosed rare disease, Takeda has the option to select up to three additional rare disease targets.
This takes the total value of the investment up to €803M, which will cover upfront, development, and commercial milestone payments. Evox will also receive tiered royalties on net sales of each product, which will be commercialized by Takeda.
In addition to the Takeda collaboration, Evox also has its own proprietary program targeting the rare disorder argininosuccinic aciduria, which can cause liver and brain damage. De Fougerolles said Evox is aiming to advance this product into phase I testing in 2021.
At present, there is only a handful of other companies involved in exosome therapeutics, including the Swiss biotech Anjurium Biosciences and US companies Codiak Biosciences, Aruna Bio, and Celltex. According to de Fougerolles, many of these companies rely on the natural contents of exosomes to create their therapeutics, but Evox differs from these because it engineers its exosomes to contain drug cargoes, which make them more potent.
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