Surrey Jul 20, 2020 (Thomson StreetEvents) -- Edited Transcript of ReNeuron Group PLC earnings conference call or presentation Monday, July 20, 2020 at 11:00:00am GMT

* Richard L. Beckman

Hello, and welcome to the ReNeuron Preliminary Results Conference Call. (Operator Instructions) And just to remind you, this conference call is being recorded.

Today, I'm pleased to present Olav Helleb, CEO. Please go ahead with your meeting.

Thank you very much. Good morning, and thanks, everyone, for joining this analyst meeting. I hope everyone is well, and we look forward to having one of these face-to-face again before too long.

Before we start digging into the presentation, if you do not have a copy of the slides, please go on to our website. There should be a link at the front page there or in the investor section where you can find the slides that will be easy to follow.

So let's start by turning to Slide #2. This is the disclaimer, so please note that. Then we'll jump to Slide #3. As you know already, ReNeuron is a leading cell therapy company. We're present in the U.K. and the U.S. And we have allogeneic, retinal and neural stem cell technology platforms. And we also have exosomes and pluripotent stem cells. Our lead program is in Phase II in retinitis pigmentosa, and we expect to partner that program when this Phase II study is completed.

There's now increasing focus on exosomes out there and also from our side and iPSCs as well. So we have already a number of research collaborations ongoing there, and we expect to have more of those going forward.

Let's have a look at the platform technologies on Page 4. So human retinal progenitor cells is our lead program. These are subretinal-delivered stem cell store, and we deliver them subretinally in order to enable engraftment, which has the best potential both in terms of efficacy and improved vision and also long-term efficacy. The cryopreserved formulation that we have developed and are currently using in the clinical trials allows for global ship and store. So these have a 9-month shelf life. We can ship them anywhere in the world. They're delivered in shipping containers that have 10 days dating on them so that we can really get them anywhere we need to get them. So that's obviously good for clinical trials, but even better for commercial launch.

The data we have so far is very positive in retinitis pigmentosa, which is our first indication. This program is unencumbered, except in China, where we have partnered up with Fosun Pharma. the exosome platform is -- comes from CTX. So it's a high-yielding platform. We have proven the ability to load this exosome with different modalities. And we also have proven that these exosomes are happy to go across the blood-brain barrier, so -- which is a big medical need. We are focusing on exosomes as a delivery vehicle. There are other potential uses as well. But for us, that is the focus.

The iPSC platform is also coming from CTX, so this is pluripotent stem cell platform, and we can engineer CTX stem cells into other forms of stem cells. And the potential here is -- are for new cell therapeutics based on this platform.

CTX cells you know well. We had positive Phase IIa results that was just -- was published in this financial year. Also published out there are potentials in other indications such as Huntington's. This program is partnered with Fosun for China, and it's available for licensing in other geographies, which is our strategy now for CTX.

The development pipeline is on Page 5. So you can see here that the retinitis pigmentosa program is on top of the list. We will have further readouts coming both this year and next year. This is an open-label study, so we're able to have a look continuously, and we'll update the market as and when required.

CTX, as I already mentioned, in stroke disability, the clinical development there is with Fosun for China the -- and other potential partnerships either for stroke or for Huntington's.

The exosome platform is in a preclinical phase with a number of different approaches. The next milestone there is proof-of-concept data. We have several shots on goal in order to deliver that over the next 6 to 12 months.

In iPSC, the focus is more about validation of the technology itself, and we're looking forward to preclinical proof-of-concept data also for that platform.

So with that, we'll skip to the operational highlights on Page 7. This is straight from today's RNS. So apologies for the slide. Busy slide, but yes, you have it in the RNS. You can read it at your convenience. These are the highlights for the last financial year.

For hRPC, we have sustained, efficacy throughout all-time points so far in the study that's ongoing. We have received regulatory approval to expand this study both in the U.S. and the U.K. And U.K. -- so far, the study has been in the U.S. only, so it's great that we can add the U.K. in here as well. We will have further readouts coming over the next 12 months. And the next step after this Phase IIa study is to agree with the regulatory agencies about a single pivotal trial that will lead to approval.

Exosomes. We have a number of collaborations ongoing using our exosomes as a delivery vehicle. We also presented new data on our induced pluripotent stem cells. This year, and we've signed collaborations on the exosome with major pharma/biotech companies, unnamed so far. These collaborations are mainly focused on delivering across the brain -- blood-brain barrier, whatever therapeutic these companies are interested in delivering.

We also have a COVID-19 vaccine delivery platform in development, which may come -- become very useful, depending on how the first vaccines -- how effective they are. And we hope to see them soon. But I think the common perception about COVID-19 vaccines is that the first ones will not be fully effective. And that's when delivery systems like this one can come in handy.

CTX cells. So yes, PISCES II was published this year. We have decided to continue this drug feasibility program through regional partnerships. Fosun Pharma in China is continuing their work on CTX. The PISCES III study in the U.S. was suspended due to COVID-19. This suspension was quite obvious since our patients there are disabled and had no business running in and out of hospitals during this time. So it was a natural suspension.

We have decided to remain in a suspended state for that study. It is both a great indication and an excellent product and a perfectly designed clinical trial. However, it is also very research -- resource-intensive for us. And we rather invest those resources in what we think are the 2 most promising programs in the mid -- short and medium term, which is in ophthalmology and exosomes. So for that reason, we decided to keep PISCES III suspended in the U.S., and we will not restart that unless it's funded by a partner.

So with that, I will hand over to Michael for the financials on Page 8. Michael?

--------------------------------------------------------------------------------

Michael E. Hunt, ReNeuron Group plc - CFO, Company Secretary & Executive Director [3]

--------------------------------------------------------------------------------

Thanks, Olav. Yes, I'm Michael Hunt. I'm Chief Financial Officer of ReNeuron, for those of you that don't already know me.

So Slide 8 just gives a summary of the preliminary results that we've announced this morning for the year ended March 2020. And we've shown the prior year comparatives here as well.

In terms of the underlying cost base, it's broadly similar to the prior year. As you can see, slight saving on G&A costs. And importantly, as Olav has just mentioned, the fact that we have now decided to make our stroke program essentially an outsourced endeavor under partnerships, that will have a consequent significant effect on our prospective cost base going forward. So we do expect to see our underlying cost base reduced significantly from what you see here in the numbers reported to March this year. The numbers you see here are also somewhat flatted by the upfront payment we received from Fosun when we signed that licensing deal for China in April last year. So that payment just made it into the numbers we're reporting on. And that's a large part, a very significant part actually, of the GBP 6 million -- or GBP 6.1 million you see on the top line.

Cash on hand at the end of March this year was GBP 12.6 million, as you see, and that gives us around about a year's cash from where we are now, maybe slightly less than 1 year. So we are reasonably well financed for the time being. And importantly, we do expect to garner further milestone payments from that Fosun deal and hopefully other deals that we sign going forward over the next year to 18 months. In the case of Fosun, that they continue to make progress with both our retinal and our CTX stroke program in China.

And with that, I'll hand back to Olav. Thank you.

--------------------------------------------------------------------------------

Olav Helleb, ReNeuron Group plc - CEO & Director [4]

--------------------------------------------------------------------------------

Thank you, Michael. Yes, I think I will now ask Rick Beckman, our Chief Medical Officer, to take you through the RP program. Rick happens to be an ophthalmologist and what he doesn't know about RP is probably not worth knowing. Over to you, Rick.

--------------------------------------------------------------------------------

Richard L. Beckman, ReNeuron Group plc - Chief Medical Officer [5]

--------------------------------------------------------------------------------

Thank you, Olav. If you turn to Page 10, we'll talk a little bit about our hRPCs, human retinal progenitor cells. hRPCs are no longer stem cells. They've already differentiated along the pathway. As such, they can only go on to become the cellular elements of the retina, particularly the photoreceptors, which are responsible for capturing a light impulse and turning it into an electric stimulus that goes on to the brain and that supports sight. We've shown in preclinical models that we have cells that integrate into the anatomic layers of the retina.

And we believe -- and we've also shown that they actually integrate and differentiate into photoreceptors. We believe that there's 2 modes of action: one is differentiation and performing photoreceptors and replacing cellular material; but the other mode of action is integration and providing trophic support. And by integrating, we believe that we can have a significantly durable source of trophic support, sort of a depot, if you may. We think that this type of technology is viable for a variety of eye diseases. Currently now, we're going after retinitis pigmentosa, and I'll talk a little bit more about that.

We have orphan drug designation in both the EU and the U.S. RP is a very large orphan population, but it is still an orphan disease. The proprietary manufacturing is what sets us apart from our competitors. This is from a collaboration between Schepens Eye Research Institute and University College of London. And we have a GMP manufacturing process. But the most important take-home point is that we have cryopreserved cells. Our competitor has not been able to develop cryopreserved cells. This is important clinically because, instead of having to fly your patients to be within 12 hours of one manufacturing site, we can ship these cells to anybody's surgical center throughout the world. They have a 9-month shelf life.

If you'll turn to Page #11, for those of you who are not familiar with retinitis pigmentosa, it's a very large unmet need. It has an incidence of about 1 in 4,000, which means, in the United States, there's some 80000-or-so patients and in EU somewhere between 130,000 and 150,000 patients, which makes it a large orphan disease. There have been over 100 genes which have been identified as causing mutations that could lead to this clinical syndrome. And you're probably aware that there is one treatment now available, that's Luxturna, for the single gene defect, RPE65.

The most important thing to understand about that is it's only a low single-digit number of patients that are able to be accessed, meaning in the range of 2% of patients will have one of these defects that people are working on right now. For Luxturna, it's probably about 2% of the population. There are multiple companies that are now working in this space going after one of these genetic defects. And that shows you that there's a very large commercial market potentially for this. But of all those defects, you're also talking about trying to access low-single-digit percentage of the patients. We believe that by using the cellular method that we are agnostic to the genetic type and therefore can go after the entire market in RP.

So the important thing to understand about retinitis pigmentosa is that patients start developing symptoms in their teens, sometimes a little bit later. And their first symptom is generally night blindness, meaning when they go into a dark room, they're not able to adapt to seeing well. This isn't that terrible. However, then it starts to rob them of their peripheral vision, which is very concerning to people. And the most important point is that these people, they know that they're slowly going blind. They get diagnosed. They look at the familial components. And aside from genetic counseling and future planning in the vast majority of these people, there's nothing that could be done. And by the time they reach their 40s and 50s and 60s, the most productive years of their life, their vision is being taken from them and they see it going away.

And so in terms of enrolling a clinical trial, clinical trials are easy to enroll because there's a list of patients who have been waiting for something available. And with their increased knowledge right now that there's a therapy available for some of them, everybody has a significant interest in being involved because there's no treatment available.

If you look at Slide #12, this is just to show you the landscape of companies that are participating in this disease. The only real competitor, and we actually think of them as somebody that's validating the principle, is a company called jCyte. jCyte uses a similar cell. However, they implanted it into the center of the eye, into the vitreous. And because it's not being implanted in the anatomic position where the cells are, we don't believe that integration and differentiation is possible. So the mechanism of action for them would just be providing trophic support, which is possible from the vitreous, but we haven't seen their data, but we're looking forward to seeing that relatively soon.

We, on the other hand, implant ourselves subretinally into the anatomically correct position such that the cells can integrate into the retina actually shown in preclinical models and differentiate and also potentially sit there and be a little biofactory for trophic factors that support the other cells.

The other players in the market are going after the gene therapy approach. And as I said, if you put all of them together, and a lot of them are going after the same disease because there's only certain RP variants that are amenable to gene therapy. Remember, you're not replacing cells. So what you have to be doing is stopping the degenerative process and potentially reviving cells that are not yet dead. They're going after the small, but there's a very large commercial value to that, as you can see from the Spark and the Nightstar acquisitions.

Going to Slide #13. I'll just take us briefly through our clinical development to date. We started off about 3 years ago at Mass Eye and Ear Infirmary in Boston with a Phase I single-ascending dose study. So what we did is took 12 patients. We treated them. The first 3 patients received 250,000 fresh cells, then we went to 0.5 million fresh cells, then we converted to a cryopreserved formulation and treated an additional 6 patients such that they were getting 1 million cells.

Our data safety monitoring board, the regulatory authorities and our investigators felt comfortable at that point then moving into a different patient population. The first group of patients were all people who had very, very severe disease, very little remaining vision. None of them were really able to see letters on a chart. And this was done because of safety. We didn't want them to have a lot to lose. But consequently, they didn't really have much to gain. Because we believe there has to be some remaining in that and be present in order to have integration of the cells.

We next moved into a Phase IIa study, and I'll be showing you some of the data from that. That's 10 patients with established RP. They had better visual potential. And consequently, they also had some vision to lose. They received 1 million cells subretinally. Primary endpoint was safety, although we started looking at efficacy.

And just to summarize before I get into the data, I think what we've shown is that we've been able to achieve visual acuity results that would be consistent with an approval in the United States and the EU if we can replicate those in a much larger clinical study. But before we go into that larger clinical study, the decision was made to provide -- to go into 90 different patients in a study that's designed to really substantiate and further bolster the data that we have because we'll be going into a much larger clinical trial. Hopefully, we believe the pivotal clinical trial at the completion of this.

If you go into Slide 14, this shows you the data to date, and I want to show you a few things to separate out this data from what we presented back in February. The first thing is we now have 1 patient that has made it out to 18 months. And one of the questions that we've been getting from people all along is how long is it going to last? One of the values of having an open-label study is we get some readouts and some learnings as we move along. However, the -- this service provided by open-label studies, you don't get to look at the data at the end in aggregate. And so you see little things happening during the middle that can point you potentially in the wrong direction.

But what we've shown in this data now is a separation. If you look at the light blue line, that's the mean change in visual acuity in the treated eye. The dark blue line is the mean change in visual acuity in the non-treated eye. And this is all measured by ETDRS visual acuity. An ETDRS chart is very similar to the Snellen chart that you use when you go to the eye doctor to have your eyes checked, except it's been adapted for doing clinical trials, meaning it's been substantiated. All of the letters are the exact same size. All of the letters are equally difficult to see between each other.

There's a standard number of size at each line. Each line is a logarithmic distance from the line before. So all of the statistical things could be done more. Basically, as you read down the line, the letters get smaller and it takes better visual acuity to be able to differentiate the smaller letters. So when you go to additional letters, we consider that a letter game, meaning that each time you're able to read up smaller letters, you've improved in your visual acuity.

And what you see is that, at all-time points, we show a differentiation between the treatment eye and the untreated eye. And we're showing a very significant amount of visual gain in most patients. To get a drug approved in the United States, people will say that they consider clinical significance to be a 15-letter or 3-line change. That doesn't mean that you have to have a mean change of 15 letters to get a drug approved. Actually, all of the anti-VEGF drugs were approved, and their mean changes were between 8 and 10 letters, some even a little bit lower. What it does mean, though, is that you probably need to show a statistically significant difference between the percentage of patients in your treatment group that are seeing a clinically significant change versus that percentage in your control group. And in this particular disease process, control patients do not generally gain vision.

Now one of the things that people were concerned about early on in our data is we've shown that there is some improvement in vision in some of the patients in the untreated eye. And that's one of the things that we've worked on for the additional 9 patients because we had 1 particular patient which showed a very significant learning curve, up to 20 letters at one point. And with a small n, you can see that, that makes the mean very significant. We don't believe that, that's a true effect. We believe that that's part of the learning curve. And so when we design the additional 9 patients, we now are very careful with getting multiple baseline readings prior to treatment so that we can eliminate anybody with a learning beforehand.

Another thing that you're going to see with this data here is that we're excluding 1 patient with surgery-related vision loss. If some of you remember well, when we presented the data back in February, we excluded 2 patients with surgical-related vision loss because we didn't think that they showed the real effect of the drug therapy. This is a well-established surgical procedure, but it's still a very delicate surgical procedure. And these eyes are very thick eyes, meaning that they've had a lot of anatomic scar changes occurring to the retina.

And so nobody in the ophthalmic community is concerned with the surgical complication rate in the rate of 5% to 10%. Because from a risk/benefit potential, there's no potential gain in these eyes. They're all going blind, and there is a very significant potential benefit. However, as you can see, right now, we're only excluding 1 patient with surgically related vision loss because one of the patients, the reason why their vision has declined is they developed a clouding of the lens following surgery, which is not uncommon with retinal surgery.

And the simple procedure removing their cataract, which is one of the most common procedures that we do in ophthalmology, probably the most common, resulted in their vision now crossing over, and that now they've gained vision. And so the effect that they have is, in the earlier months, they're bringing the visual acuity, the mean change down a little bit because they were a vision loser down to about 20 letters or so of vision loss. But as the data progresses and their vision has now improved, you'll start to see more of a separation of those lines. Again, that's the effect of looking at data day-to-day in an open label as opposed to just looking at it at the end when it's all in front of you.

So now we have a surgical complication rate of 1 in 22, which is about 4% to 5%. And nobody in the ophthalmic community is concerned about that. We'd rather have none, but it is a surgical procedure, and we are humans.

I'm going to go onto the next slide now, which is the design of the Phase IIa extension study, and I've already alluded to some of the components. One of the past criticisms that we've had from potential partners is, "Hey, we get it. You've got really good efficacy, and we believe that you can be approved on efficacy alone, but we'd like to see that corroborated by some other indicator of efficacy." And during conversations with our scientific advisory board and particularly Dr. [McFerran], who's now come on as one of the investigators from Oxford, who is involved in the formation and prosecution of Nightstar, it turns out that visual acuity was more difficult to achieve in those patients than the efficacy indicated that it was successful was microperimetry.

And microperimetry is a measure of retinal sensitivity and what you do is you focus the camera and light source at specific elements of the retina and you stimulate the same point at levels of illumination, which are too dim to be seen and then you ratchet it up until the eye can just barely see it. And by doing that, you can map out the sensitivity of the different retinal points.

Now what they did, which is very nice, and what we're doing in this study, is we're now only including patients that are capable of consistently repeating and performing the microperimetry test. And that will probably kick up the average visual acuity of our patients a little because, in general, with RP, it's the patients that still have more remaining vision that are able to fixate and take that test. So the study with the extra 9 a -- 9 additional subjects is designed to give us a -- another efficacy indicator to substantiate the visual acuity movements that we've already seen.

Finally, as I told you, we're doing additional baseline so that we're not going to establish a learning curve. And we've also eliminated a symmetry between the 2 eyes that they have more than a 20-letter difference between the 2 eyes we don't include them, just so people can't say that that's regression to the mean or anything like that.

Finally, what we've done is we've modified the surgical technique, and this is a really important minor detail to most people, but a major detail to the surgeons doing the surgery. We've doubled the dose to 2 million cells. The reason being is we've seen no dose-limiting toxicity. The regulatory agencies prefer it. And we also want to see if the reason why some patients have had a tremendous response and some have only had a moderate response could be a question of dose.

So we've increased the dose. We're now going to create 2 different surgical blebs underneath the retina. But the design now, unlike the previous patients who we treated. Previously, we treated patients, and we tried to deliver the cells right to the areas of the retina where we had remaining functional retinal tissue. And that's important because we want the cells in that area. But the difficult part of that is that, when you lift them off by creating a little blister underneath the retina for the fluid, which takes a few days to reabsorb, you're also separating them from their nutritional supply. So it's potential for a double-edged sword.

What we're doing now is we're going to be very carefully placing those blisters so that we have the edge of the blister in nonfunctional tissue, but getting very close to the functional tissue, with the idea being we'll get the cells very close to where we need them, but we're not going to be causing a separation of the retina. And we -- and potentially, that will give us a little better efficacy. So I believe and we believe, and our advisory board and investigators believe, that we've set up a very, very nice additional study to substantiate the data that we have from the earlier studies. And to use my own parlance, I think we have a very, very accurate shot on goal that is prepared.

And if you turn to Slide #16, we believe that we're going to get started on this imminently. We do have approval from both the EU -- U.K. and the U.S. regulatory authorities. We're in the process of getting patients set up. And hopefully, we'll be doing something in August or early September. Remember, right now, a lot of centers are closed down because of COVID, which has caused a significant delay. But if our plans work out, and we're pretty confident they will, then we'll get started within the next month or 2. And we anticipate having top line data to present sometime around this time next summer. However, if there's something material that happens beforehand, we'll be presenting something earlier than that.

I think that I have -- oh, yes, and the idea would be to submit to the regulatory agencies to go into a potential pivotal clinical trial, submitting sometime at the end of 2021 and potentially getting started very early in 2022 at a pivotal trial. That's all I have. So I'll turn it back to Olav.

--------------------------------------------------------------------------------

Olav Helleb, ReNeuron Group plc - CEO & Director [6]

--------------------------------------------------------------------------------

Thank you, Rick. And yes, just to remind everyone that there will be an opportunity for Q&A at the end. Obviously, the RP portion is the key portion of this presentation. But let's have a look at exosomes and the iPSCs as well.

So on Page 18, just a quick primer on what exosomes are. So they're naturally occurring communication devices released by cells. We and others have demonstrated that these nanoparticles can be loaded with different cargoes to deliver, very efficiently, therapeutics to specific tissues. And this will overcome many hurdles that you see with the gene therapy, and I'm talking then about hurdles or disadvantages just really of the viral vector delivery system that gene therapies are using.

On Page 19, ReNeuron has years of experience around manipulation and culture of cell lines. The CTX cell line in particular, is easily to be modified so we can allow engineering and introduction of new payloads. And the c-mycER immortalization technology is key to that. It creates consistency of the product. And the stable cell line results in the stable exosome product. So we've shown that our exosomes will and can cross the blood-brain barrier. We've proven an ability to load micro RNAs and proteins. We've shown stable and consistent high-yield production, and we've shown that we can carry various payloads and also engineer to target specific tissues.

And that results in the pipeline on Page 20 of several exosome products and also some iPSC platform technologies. As examples of the exosome, so this is all about loading something into the exosome. The exosome is the delivery vehicle. And then we have a payload loaded into that.

ExoBDNF. As an example, this is an exosome that's engineered to express the neuroprotective growth factor, BDNF, brain-derived neurotrophic factor. That's been sitting on the surface of the exosome. And BDNF has been implicated in Parkinson's, Alzheimer's, but also hearing loss and glaucoma. The second here is ExoKRAS. this is an exosome engineered to deliver the sRNA against KRAS G12D gene mutation, which is found in a number of cancers, particularly glioblastoma and pancreatic cancer.

The third one down is exoSPIKE. This is an exosome that's engineered to express the SARS-CoV-2 spike protein on the surface of the exosome, and that aim -- that assists for the delivery of a prophylactic vaccine. It is a targeted delivery directed to the lymph nodes, and that stimulates strong immune response. So this has a great potential for delivery of -- sorry, COVID vaccines. Hopefully, Moderna or some of the other companies will take care of it all very quickly, but the chances are that that's not going to happen and improved delivery systems will be needed. In that case, this will be a very important program.

Well, the fourth one down is a collective of different approaches that we have. This is exosomes that are loaded with therapeutics of partners. So we have signed research collaborations with large biotechs. They have certain payloads they would have -- like to have delivered into the brain. That's obviously a very difficult thing to do. And what these partnerships are focused on is that our partner will send us payloads. And sRNA being a typical payload, we put them into our exosomes. We send them back to our partner. The partner then runs studies in the lab and then animal studies to see if this will solve the delivery issue. And if that -- if the payload is actually active on the inside, any proof-of-concept on any of these collaborations will be extremely valuable in validating the entire exosome platform.

So they're ongoing, and we expect to sign more. This is a very hot area for research. So I'll touch more on the iPSCs a bit later, but let's have a look at deals done in exosomes, first of all, on Page 21. So like I mentioned, the -- there's a lot more attention now for research around exosomes as an alternative to viral vectors. There are 2 pure-play exosome companies of note. One is Codiak Therapeutics in Boston. The other one is Evox Therapeutics in Oxford. Between them, this -- they have signed 4 major deals.

The Codiak/Sarepta one was just 2 weeks ago. That was a total of $72 million in research payments for Sarepta to -- that Sarepta paid in order to investigate 5 different neuromuscular targets. So -- and just in technology access and research payments, that's -- the $72 million, that's more than our market cap. So these deals are as significant when you consider that nobody is in the clinic yet. It's all in research.

Then the -- I guess the natural question is, what does Evox and Codiak have that ReNeuron doesn't? And the answer is proof-of-concept data in an animal model. And we're working feverishly on delivering that. You saw that on the previous page. We have 2 internal programs, one of them grant-funded. And we have several programs also with partners. So we have a number of shots on goal over the next 6 months or so to deliver on that.

A bit more on iPSCs on Page 22. So iPSCs are ideal material to produce themselves both the cell therapy and for -- to produce exosomes. They have the potential to differentiate into any of the 3 germ layers. However, they're unstable. Differentiation are -- is difficult and never 100% efficient. So for us to overcome these barriers, we have reprogrammed our CTX cell line to improve the potency. And what that has resulted is actually that the c-mycER immortalization technology is continued through this change in reprogramming. And that is very, very useful because that means that now we have been able to put that stability into the iPSCs and we can create off-the-shelf cell therapy products as a starting material for -- to produce cells such as T cells for CAR-T therapy, for example.

So there are 2 programs that we are developing right now. One is immortalized haematopoietic stem cells. So this is to provide a stable intermediate for these T-cells or NK cells, as I mentioned. And the other was the pancreatic progenitor cell for the generation, again, of a stable pancreatic progenitor that could be used in diabetes. So these are early research programs, and the goal is very much to validate the technology and have -- and create a cell line that we can take forward.

Lastly, let's go to CTX on Page 24. I just want to reiterate that our strategy for CTX going forward is a licensing strategy. So we, obviously, are committed to support Fosun for their work on CTX in China. And we're also looking at out-licensing for other potential territories as well as the Huntington disease potential indication where we have published some interesting animal data.

Last page, Page 26. Just a quick summary before we go to Q&A. So ReNeuron, as you know, is a global leader in cell-based therapeutics. Our platforms are allogeneic. They're patented and high-yielding. Ophthalmology is an area of great industry interest, and the RP Phase II study has produced excellent results so far, which has led to this study to be expanded. Another hot area is exosomes, and we are well positioned here to generate validating proof-of-concept data and then partnering deals following that.

So that concludes the presentation. I suggest we go to Q&A. I now hand over to the operator to take us through the -- how that works.

================================================================================

Questions and Answers

--------------------------------------------------------------------------------

Operator [1]

--------------------------------------------------------------------------------

(Operator Instructions) Our first question comes from the line of Christian Glennie from Stifel.

--------------------------------------------------------------------------------

Christian Glennie, Stifel, Nicolaus & Company, Incorporated, Research Division - Analyst [2]

--------------------------------------------------------------------------------

Three questions, if I may, and just take them in order, if that's okay. Firstly, on the expansion, just looking ahead to the RP trial and expansion of that trial. You obviously talked about the -- with COVID restrictions. But what's the potential timing of that -- starting that trial and actually getting those 9 patients treated? And potentially, you'll have sort of 4 sites, 2 -- [still] 2 in U.S. and an extra in the U.K., I think.

And as part of that expansion, did you consider on a higher dose, but did you consider a redosing at any point? Or are you thinking actually the longevity of the data you've seen so far suggests that maybe that may not be necessary?

--------------------------------------------------------------------------------

Originally posted here:
Edited Transcript of RQE.L earnings conference call or presentation 20-Jul-20 11:00am GMT - Yahoo Finance