ReNeuron upbeat on positive data from Spain collaboration

Exosome therapeutics developer ReNeuron announced positive data on Monday that it said provided “clear preclinical proof-of-concept” that its novel exosome drug delivery technology could effectively deliver therapeutic proteins to the specific region of the brain affected by several neurological diseases.

The AIM-traded firm said that in an ongoing collaboration with scientists at the University of Salamanca in Spain, it had sought to determine whether its stem cell-derived exosomes could deliver a therapeutic protein to the corpus striatum - a region of the brain affected by disabling neurological diseases including stroke, Parkinson's disease and Huntington's disease.

Major pharmaceutical companies had identified therapeutic proteins that are effective in treating a variety of neurological diseases, but there were major issues associated with the delivery of them, including the poor stability in living organisms given that proteins rapidly break down and do not last long in the body, as well as issues surrounding poor tissue distribution due to an inability to target specific tissues.

ReNeuron said that while the issues could not be overcome by simply administering more protein, as that could have unwanted side-effects, it believed that its proprietary exosomes had the potential to address both of those issues due to their natural tissue-targeting ability and superior stability characteristics.

Studies performed in collaboration with the University of Salamanca established that exosomes administered intrathecally were capable of delivering a therapeutic protein in an animal model to regions of the brain at a functional activity level much greater than that seen when simply supplying the protein alone.

Contrary to that, studies performed in the laboratory showed no difference in delivery between the exosomal-based protein and the protein alone.

That difference between the efficiency of in vivo delivery versus in vitro delivery suggested that the exosome-linked proteins were able to overcome the obstacle of protein breakdown and tissue targeting in the brain that was not possible with the native protein.

The company said the in vivo results were “key” in showing that ReNeuron's exosome delivery technology offered a striking higher stability, more targeted delivery, and an increase in potency, thus potentially solving the delivery issues that could be experienced with therapeutic proteins.

“I am delighted to see these extremely compelling data showing that exosomes efficiently deliver therapeutic proteins to targeted brain regions associated with severe neurological diseases,” said chief scientific officer Dr Stefano Pluchino.

“These data are the foundation of one of the main corporate programmes at ReNeuron and highlight the possibility to deliver other payloads using the same principles and technology platform, providing improved tissue distribution and specificity.

“This exciting strategy could potentially be used to deliver single therapies or multiple therapeutics with exosomes, enabling a number of exciting next generation precision medicine approaches.”

Chief executive officer Olav Hellebø added that the results were a “key milestone” for the firm’s exosome delivery technology, establishing a “clear” proof-of-concept in vivo, which more accurately imitated conditions for the targeted delivery of key neurological therapeutics into the human brain.

“In 2020 we refocussed the business on our exosome platform, as well as our retinal disease programme and iPSC platform, and these results show just how significant the potential is for exosomes to become a novel means of delivering third party biological drugs to the brain and other regions of the body,” Hellebø added.

“This is exciting news for the major pharmaceutical and biotechnology partners that we have collaboration agreements with and underpins the commercial opportunity that our platform provides.”

At 1131 BST, shares in ReNeuron Group were up 34.81% at 127.8p.