RE: In Vivo11 Oct 2021 14:24
Easier to let the CEO explain it in his own words from the July 2021 preliminary results call:
https://www.youtube.com/embed/jg6EQaxyr1E
Skip to around 30m for main discussion on exosomes (whole video is worth watching for overview of all the programmes and company itself).
Some of the transcript below for exosomes:
'So exosomes are nano-sized vesicles that are excreted by all cells in the body, and they're part of how cells communicate with each other. And so it's basically a natural liposomal delivery system. It's possible to hijack this system, either to use it as a drug delivery system or even as a therapeutic in itself, or as a diagnostic tool. Our current focus is on the drug delivery bit, and we have collaborations ongoing on that.
On Page 21, you can see some of the advantages of what we are working on. The first is that we've noticed a favorable distribution across the blood brain barrier. That's one of the biggest hurdles in drug therapy is how to get drugs into the brain efficiently. So we are exploring that now to see if our exosomes can answer that. We've shown ability to load micro RNAs and proteins, we've shown stable and high yields with a clinical-grade product. Obviously, our starting point is a GMP product, which is [GP] and CTX. We've been working on this some time and so we have established analytics, and we have seen a free scalable process already established.
On Page 22, a little bit more on the collaborations we have ongoing. So the 3 partners we're working with, their names are undisclosed. We would wait on disclosing that until we actually have a licensing deal, which should follow from a positive proof of concept. It's all about -- it's focused on delivering our partners' payload, and they are siRNAs and mRNAs for the moment. And the way this is done is that a partner sends the payload to us. We load it into our exosomes. They pay us for that work so that we can cover our costs. We send them the new construct back to the partner, and they run animal studies in the chosen indication to see if we can knock down the target with an exosome-delivered gene therapy. Now if the animal data comes back positive, we would expect then that our partner would like to take it forward. We own this new contract 50-50, so they would then obviously need to license rights from us to go forward.'
So the main point here is that they've been waiting to publish proof of concept, and now they have it, they expect licencing deals with the major pharmas they have been collaborating with. The other party will pay upfront fees along with milestone payments as it progresses through clinical trials. The scope if huge, and this isn't even the lead programme for Reneuron, the main programme is for retinitis pigmentosa, where they already have some industry leading data and now evaluating the treatment at a higher dose, before starting a pivotal trial H2 2022. News flow is looking great for the foreseeable future.