British biotech Evox Therapeutics has signed a collaboration deal worth up to €1.1B with US big pharma Eli Lilly to develop RNA drugs that are delivered in exosomes to the brain to treat neurological diseases.

As part of the deal, Evox will get €17.6M upfront to fund research for the next three years and Lilly will also invest €8.8M into the company as a convertible bond. The UK biotech will also be eligible for more than €1B in future preclinical, regulatory and commercial milestone, and royalty payments. 

The agreement is for up to five targets to be developed by Evox, although the specific neurological indications were not disclosed by the two companies. Once the drug candidates reach the clinical stage, Lilly will take over subsequent development. 

Exosomes are small fat particles that transport proteins and RNA around the cell to where it is needed. Evox has discovered a way to use this natural cellular transport system to carry drugs to certain areas of the body. This, in turn, allows the company to target the treatment to certain tissues and does not need to go via the liver, which can prevent drugs from reaching their destination in the body. 

“As a field, the biotechnology industry has been trying to develop man-made solutions for extra-hepatic delivery of RNA drugs for a while with limited success,” Antonin de Fougerolles, CEO of Evox, told me. 

“In contrast, we have opted to use nature’s solution to this problem as a starting point, and are engineering exosomes to improve their drug-like properties.” 

This collaboration is the second big deal in the last three months for Evox — an impressive feat given the current global pandemic. The company signed an €800M deal with Japanese big pharma Takeda at the end of March to tackle rare diseases using its exosome technology.

Although the technology platform is the same for both partnerships, de Fougerolles explained that the collaboration with Lilly will be to develop small RNA drugs — specifically those based on RNA interference (RNAi) and antisense oligonucleotides — focused on neurological targets, whereas Takeda wants to develop messenger RNA or protein drugs for rare diseases. 

The brain is a particularly difficult target for drugs of all kinds due to the blood-brain barrier, which prevents molecules in the blood from entering the brain. Exosomes could be one way to circumvent this barrier.

“We and others have early preclinical data, some of it published, to suggest that exosomes can be engineered and directed to successfully deliver RNAi drugs to the brain,” said de Fougerolles. 

Only a small number of other companies are working in the exosome space. This includes Swiss biotech Anjurium Biosciences, and the US companies Codiak BioSciences and Aruna Bio, all of which are at the preclinical stage. 

“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,” emphasized de Fougerolles.

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Source: https://www.labiotech.eu/brain/evox-therapeutics-eli-lilly/