Good morning, everyone. The clock has now ticked past 11:00 A.M. We'll get started with today's special investor briefing with Syntara CEO, Gary Phillips, who will provide an update on the clinical trial program and the exciting FDA developments. My name's Alfred, and I'm part of the Syntara Investor Relations team, and I'll assist Gary with running the briefing today. I'll just quickly run through some of the formalities. After the presentation, Gary will be taking questions from attendees. If you do have any questions for Gary, there is a sidebar, at the bottom of your screen if you're on a desktop, and it's also at the bottom if you're on a smartphone.
Throughout the presentation, if you would like to enter any questions in, we'll read them out when Gary gets to the Q&A session at the end of the presentation. It's obviously a very busy day on the ASX today, especially being the short week. Gary has been very generous with his time to present the quarterly activities and plans going forward. I won't take up any more of your time, Gary. Let me hand over to you.
Thank you, Alfred, and morning, everybody. Yeah, I don't mind spending my time with you. This is a particular time in the life cycle of Pharmaxis where, we're ahead of some clinical trial results coming through. It's always a period when level of excitement grows. It's a period when we start to see the fruition of the investments that we've made, over a number of years to get to this point. An exciting point in time. Happy to take your questions at the end, in the meantime, just, you know, the usual forward-looking statement take note of. Really focusing this morning on two key areas.
The trial in myelofibrosis, which had a number of developments during the last quarter and some excitement coming up, and then the skin scarring study. Let's start with the myelofibrosis study. Those of you who've been following this know that it's a study that we're aiming to recruit 24 patients in. It's a monotherapy study, so we're looking at treating myelofibrosis patients who have failed on the existing standard of care, a JAK inhibitor. Only have, you know, about a year to live at this point, and then treating them with PXS-5505, our drug, as a monotherapy. Now that, you know that's proven to be quite difficult to recruit. These patients are very rare.
We've opened up 20 sites across the world in order to recruit these 24 patients, and we're currently standing at 21 out of the 24 patients recruited. Each patient stays on for six months. You know, when we discussed this with the FDA, I mean this is an IND approved study. We discussed it with the FDA at the beginning, and they'd always said to us that they wanted to see, you know, a minimum amount of data from these patients to check on the safety. As we were going through the study, we saw that the safety profile of the drug and certainly some of the early signs of efficacy were really promising, and particularly the safety profile was really clean.
We felt that even though we hadn't fully recruited the study, and certainly not finished the last patient, last study, I mean we expect to fully recruit it in this quarter, and therefore we're gonna have data from all of the patients by the end of the year. We felt that we had enough data to go back and justifiably ask the FDA whether they were comfortable with the profile of the drug they were seeing, and therefore, would they think of our plan to move forward immediately into the next stage of development. Which is where the drug's gonna be used as an add-on to the standard of care, the JAK inhibitors in myelofibrosis.
That's a really important clinical question, because we believe that the mechanism of our drug is ideally suited to be fitted with a JAK inhibitor. The JAK inhibitors tend to produce an immediate improvement in spleen size and some of the symptoms. Whereas our drug, tackling the fibrosis in the bone marrow, deals with an underlying cause of the disease and is likely to lead to a longer term improvement in these patients. Whereas patients who are on a JAK inhibitor are still gradually deteriorating, because the JAK inhibitors aren't curative, you know, our drug would be a real benefit. We were very keen to move on from a clinical sense with this add-on study.
From a commercial sense of, you know, in talking to companies that have JAK inhibitors that are active in this space, you know, they will be keen to see the data that we get with a JAK inhibitor as well. The question we posed to the FDA was, "Are you comfortable?" You know, we showed them all the data that we had to date. This is an open-label study, so we were able to show them all the data we have from all the patients so far, both the safety and the efficacy profile. You know, we reported in the last quarter of 2022 that we felt we were seeing encouraging signs of clinical efficacy and a very strong safety profile.
I, you know, and I was really pleased to report earlier this quarter that, you know, the FDA engagement that we had through requesting a Type C meeting with them, was very encouraging. They thought that we did have enough data to move on to the next study. Certainly to give them a protocol for the JAK inhibitor combination study. They gave us some guidance on what we might think about in preparing that protocol. One of the key things with this plan is that, you know, we're enabled to move on really quickly with the next phase. Two things that are behind that statement of being able to move quickly.
The first thing is that we can now do this as an additional arm to the existing study. That means we don't have to start up a new study. We can keep the existing sites going. We've already talked to our investigators in many of these sites. They're enthusiastic about going on to the next arm. There's no long, protracted period at the beginning where we're contracting sites, getting the CRO set up, getting the protocols and everything else worked out. You know, we'll be able to move on really quickly. That's an enormous benefit. The other thing is that, you know, in discussion with the FDA centered around which dose we might use in the add-on study as well. This first study that we did in monotherapy, we went through a dose-finding period first.
We started with a low dose and then built it up into the highest dose, which is 200 mg twice a day. That's what the patients are staying on. The conversation with the FDA was a positive one, we believe that they're comfortable with us going in straight away with that high dose of 200 mg because of the safety profile that we're showing. Means we don't have to go through a dose escalation phase, which again chops, you know, a long period of time off the start of the study and getting it going. We aim to get this study and start recruiting this before the end of the year, so in the second half with a really fast start.
In the immediate future, towards the end of this quarter, we will have a further major update from the data on the monotherapy study as well. Some really key elements of this clinical development plan, which really expand both the clinical utility of the drug going forward and also its commercial value as well. Add-ons to JAK inhibitors are valued by both the clinicians and potential partners and have driven very large acquisition deals in the space in the last couple of years. In fact, we're with a couple of companies going for more than $1 billion with phase II data showing effectiveness in a combination setting. That's really exciting.
The other study to talk about briefly is the scarring study that we're doing with Professor Fiona Wood over in the University of Western Australia in Perth. That's an established scar study. We've recruited patients who have a scar of at least 10 centimeters squared in size and have had that scar for at least one year. It's over a period of one to five years. The treatment is a cream which is being applied to the scar. We're looking for signs of improvement in both the structure of the scar. We are looking at the taking skin biopsies from these scars before and after. We're also looking at the appearance of the scars as well. This is a three months study, and it's placebo-controlled.
We finished recruitment in Q4 last year. I'm pleased to say that the last of those 50 patients which were enrolled in this study was dosed in March. Which means that we are now in the period of cleaning the database. The data is currently still with the sites, going through it, analyzing the data. They're looking at the images of the scars before and after still in a blinded sense, and assessing how they're scoring the scars. We expect that later this quarter, you know, that data will be unblinded, and we'll be able to announce the top line results from that scar. It's been a really exciting period.
You know, talking to Professor Wood, last year, you know, she felt that the first few patients that came into this study, that were on active treatment, that she was seeing some changes in the scar. We need to see that relative to the placebo effect. We're excited to see the results, and we're looking forward to announcing that and talking in much more detail about the potential for this treatment in scars. Not only in changing established scars but also looking at how we might use our pan-LOX inhibitors to treat the scars that occur after surgery as well.
Looking at this in a prevention setting, and this is an ongoing dialogue that we have, with Professor Wood and her team, on what other indications we can go after with this drug and where it might be best suited. Looking forward to announcing that data later. Really those are the highlights of the of both the quarter that's just gone and the excitement leading ahead. I'll just remind you know, Syntara is a company with several irons in the fire in terms of clinical studies. We have five, four or five trials going on at the moment. The fifth one being the, the JAK inhibitor combination study that's coming up, which will deliver near-term value.
you know as a small biotech, with a pipeline of assets, all of them in the stage where we're looking to show clinical proof of concept and looking at real, valuation events in the future of the company coming up. That goes all the way from the myelofibrosis studies that we've talked about where, you know, I think I've got an addressable market here of AUD 1 billion. But we know that the lead product in this, in this particular cancer is already selling more than that. That's a conservative estimate of the commercial value of the market. If we can demonstrate that the drug works as an add-on to the JAK inhibitor, then, you know, there's a, there's a real possibility of a really significant commercial opportunity there.
Then in scarring, as I said before, you know, there are very few drugs in the market that can be used. The standard of care is laser therapy or steroid creams or injections into the scars which don't always produce a good outcome. Our drug is, I think, one of the only drugs in clinical development that's a pharmacological treatment of scarring. We've got a lot of interest from potential partners in this as well, and the market for this is absolutely enormous. Looking at keloid scarring globally, and hypertrophic scarring that comes from surgery after burns and other injuries. You know, that in its own is $3.5 billion a year. A huge opportunity if we can demonstrate some efficacy there.
Finally, but not last, is the drug, the repurposed drug that we have, an anti-inflammatory agent, and we've identified it as a potential treatment in neuroinflammation and in Parkinson's disease in particular. That study in a sleep disorder is about to start recruiting in the middle of the year. Again, another exciting going forward, and that's one that we're working in partnership with Parkinson's UK, who have fully funded this study. News flow. We've announced earlier this in the quarter already that we've strengthened our board, two new members.
We announced some new publication in Nature Communications, a really prestigious publication on the effect of PXS-5505 in another kind of hematological cancer, myelodysplastic syndrome. We've already announced the FDA feedback we've got on the ongoing myelofibrosis study. Still to come this quarter is the update on the myelofibrosis monotherapy study, the full recruitment of that study, with a looking forward to data by the end of the year. The more details we'll announce on the protocol for the add-on therapy to JAK inhibitor once we've had a further dialogue with the FDA.
Topical drug PXS-6302, top line data coming up before the end of the quarter in the established scars, and then hopefully kicking off studies in scar prevention, and the RBD, the sleep disorder study, Parkinson's disease study, kicking off around the middle of the year as well. A really exciting couple of quarters ahead of us, and I look forward to talking to you more about that. In the meantime, I'm very happy to take your questions on anything that we have in the quarterly report of what I've talked about this morning.
Excellent. Thank you very much to Gary for the informative presentation. We have had quite a few questions come in already, which we'll start answering in a moment. If anyone does have more questions, please type them into your Q&A box. Gary, quite a few questions have come in about the different drugs. I might start with PXS-6302 and the scarring trials. The first question, Gary, comes from Jawahar. Could you please elaborate on the preliminary findings of the ongoing human trials with the ointment, trials being conducted by Fiona Wood? Do the preliminary findings indicate whether the ointment is working on big scars versus small scars, and also old scars versus new scars?
Yes, a good question. The entry criteria for the study is that the patient has to have a scar of at least 10 centimeters squared. That actually doesn't count as a very big scar. And we're finding actually some of the patients in the study have much larger scars than that, and they're treating a small area of it. We're only treating a 10 centimeter square area, this whether the scar is only that size or it's much bigger. The scars have to be at least one year in age and up to five years. Talking to Fiona Wood and her team, we don't believe that once a scar gets to one year, it changes in its type after that point.
We were looking for a stable scar to see whether we could have an impact on that. The early results from that study were encouraging. In particular, I think we took skin biopsies from these patients, the first eight patients that were treated, and we were able to look at the, use our assays to look at the number of collagen crosslinks in the scar and the amount of collagen and the amount of LOX in the scar. We found that there were physiological changes within the scar which showed that the cream was doing something, it was changing the structure of the scar. That is the bedrock of our optimism going forward, that we are doing something.
When it comes to the appearance of the scars, we rely very heavily, of course, on the assessment of, Fiona Wood and her team. They're treating patients in the study who they've known sometimes for years, that Fiona herself would have operated on, you know, years ago, that resulted in the scar. She knows them well and, you know, she was, and I think it was taught publicly that she was, you know, she thought she was seeing signs of improvement in the appearance of the scar and also the feel of the scar. Of course, you know, these clinical trials in scarring are, I would say quite difficult. The endpoints are subjective.
That's why we've looked at this as a placebo-controlled study, so that we can try and tease apart the effect of the drug versus the effect of rubbing a cream on daily on these scars. You know, I think we take heart from the preliminary data that we saw, it will be good to see that matched up against the placebo arm so that we can start to really identify what's going on here and what's the best thing.
I think we'll learn an awful lot about the drug from this trial. We're very confident that we are doing something to the structure of the scar and we will change scars. I think the questions remain in terms of how long do you need to treat? What kind of scar will respond best? With Fiona Wood and her team, we're dissecting that and looking at what next. Yeah, very looking forward to seeing the data.
Thanks, Gary. just a follow-up to that one. Does the study also include keloid scarring?
No. We've excluded keloid scar from this. A keloid scar is quite different in structure from the scar you get from some of the injuries like burns and things that we see. The keloid scar has a, you know, is prevalent in certain skin types, Asian and African skin types. It tends to be very heavy in, much heavier in collagen. A lot of that collagen isn't actually cross-linked, so it is a different kind of scar. We wanted to make sure that we. Again, we've got to be very careful. There's a lot of different skin types out there. There are a lot of different types of scars.
If we look at recruiting too broad a picture, we might end up with a very confusing mix and not understanding what the drug's doing. This first one is very much looking at that. We are looking at keloid scars as a potential future study. We've held with Professor Wood's help, national advisory boards with keloid scar experts from across the world, sitting down and looking at the mechanism of our drug and talking at the difficulties in looking at keloids. Nobody's done an effective study in keloid scarring. We really need to understand the genetic basis for the scar, what the current standard of care is, and how best to use our drug on it to make an impact.
Thanks, Gary. I'm gonna combine a question from George Gela and Dennis about PXS-6302. Based on success of the scarring trials, what is the proposed plan of action, and what further studies and approvals are proposed? Do you expect to present this at a conference presentation or would you announce it via the ASX as a standalone announcement?
Well, the second part of the question is easy. This will be a standalone. You know, this will be an ASX announcement. You know we always are faced with issues with publication and reserving data for conferences and the like. Clearly this is a through our normal channel to the ASX as soon as the data is unblinded and we've looked at the data and are able to say that we think we understand the data and there's nothing if there are questions in there we've been able to answer before we announce it, so it's not misleading. Yeah, that will be that.
In regards to the first part of the question, the next steps, you know, I think there's two parts to it, to that. One is, we recognize that dermatology is quite a specialist area and dealing with the regulators in this area and understanding the endpoints that regulators might see is quite a challenge. We have already started to engage with specialist dermatology companies who are looking for assets in this area. We're trying to learn from them in terms of what kind of data would they like to see.
I believe that, you know, this first study will be very encouraging for us, and will allow us to engage the regulators in a much more detailed discussion based on the data we're seeing to say, "Okay, this is the kind of endpoints we think might work." It will also enable us to talk to potential partners and say, "Okay, here's what we know. This is what we've learned. Now, you know, what is the study and which are the indications we're really gonna drive a big commercial valuation of this asset?" You know, that's something that we'll be doing around the middle of the year, fortunately. By this year, you know, the world's largest pharma partnering conference takes place in June.
We expect to have maybe just in time for that data from the study. It'll be a good time to be engaging with people and thinking about what do we do next. We are already, as I said, engaging with Professor Wood and her team on looking at keloid scarring as a potential option. We're looking at Dupuytren's, which is a scar which forms in the tendons on the hand and leads to a finger. That's a commercial opportunity. There's all kind of manner of things that you could use a treatment like this which deal with the skin, another example would be surgical adhesions.
You know, where the skin sticks together internally in the body after abdominal surgery, for example. That's an opportunity as well. There's cosmetic surgery and the impact that you can have on either preventing a scar emerging or dealing with people who already have scars from cosmetic surgery as well. There's a whole world out there. We're very keen to look at the, you know, exactly what is the best place to go in.
Thanks, Gary. I might move on to a couple of questions about 5505 and the myelofibrosis trials. Question from Rhett: How closely will you be working with the JAK companies in combo trial? Would you see merger potential if early data is encouraging?
Well, clearly the JAK inhibitors are, on their own, are not leading to a change in the outcome of myelofibrosis. I mean, these patients still, they have a life expectancy around about five years. Most of them drop off of JAK inhibitor before the end, and once they do, they have, you know, less than a year to live. You know, the market itself is very large. Clearly those companies that have a JAK inhibitor would like to be able to do more for the franchise and more for the patients that they're currently treating. There are a number of different approaches being trialed. Some of them are in the clinic ahead of us. A lot of them deal with a different aspect of myelofibrosis than we do.
We, you know, we believe that we're probably, you know, one of the few that's dealing with the fibrosis in the bone marrow directly in this particular approach, that's this advanced, and where we've already got, you know, FDA guidance on going ahead with a JAK inhibitor combination study. We believe that there's certainly commercial and clinical room for us in the myelofibrosis treatment area, despite us not being as well advanced as a couple of the assets that have come forward. The discussions that we've had to date with the companies with JAK inhibitors would seem to suggest that they are certainly interested in tracking us and seeing what we're doing.
The monotherapy data in itself, I think is important. It certainly gives an indicator of what the drug can do. I think the question specifically about merger opportunities, I think the more data we can get and the quicker we can get it in combination with the JAK inhibitor showing that both we don't make the safety of these. You know, the JAK inhibitors are poorly tolerated. They cause cytopenias in these patients.
Anything we can do in that perspective, which the preliminary data seemed to suggest that we were stabilizing or improving the cytopenia in these patients. If we can rip that out in combination with a JAK inhibitor and really synergize the effect of that, then I think that there's, yes, there's every chance that this would be a drug that would be partnered at that stage, acquired by one of these companies. The history of this is, has been. There have been a couple of companies that have been bought for over $1 billion. Morpho Sys being one, Constellation being another, which have driven very large bill values on the basis of phase IIb data.
Thanks, Gary. Still on PXS-5505. A question from Dennis. Have any subjects dropped out in the first month of treatment?
Very Yes. I think we've had one patient in the first month of treatment, the others have stayed. That's part of the, you know, why we went to the FDA, you know, they want to see at least one month of data from the patients that they wanted to see. We were able to present them with a lot of patients with significant amounts of data. Which is why I think they were in their response to us and encouraged us to submit a protocol for that one. They're being able to move it much more quickly than we had originally planned. That was all good news.
Thanks, Gary. Just one more question. If anyone does have any questions for Gary, please pop them in the Q&A box while he's available. A question from Michael. What is the current cash position to fund these trials? Was the decision to discontinue Rochester a function of cash constraints?
At the end of the quarter, AUD 15 million in the bank. We are currently looking at the feasibility and the budget for doing the combination study. As I said, you know, as well as being efficient in time, it's also very efficient in terms of cash as well to add an arm on to the existing study rather than to start a completely new study in stand-alone. We are currently working with our CRO on understanding the cost of that, and we will make our position a bit clearer on that later in the quarter once we've got more feedback on the protocol from the FDA and we understand exactly what we're going to do.
As with regard to the Rochester study, there were two sides to that. One is, you know, the study had not recruited, having been for some months, so we felt that, you know, it was an investigator-initiated study and, in talking with the investigators, they were still very enthusiastic. You know, they did the preclinical work in hepatocellular carcinoma and liver cancer as well. They recognized as well that based on the entry criteria that they'd agreed with the FDA for the study, that this was gonna be an extremely difficult study to recruit in a single center. We would need to add on more centers in order to recruit it in a reasonable time.
We didn't feel that, given the single center nature of it and the numbers of patients that were available to that center, that it was, you know, we weren't gonna see a result in a reasonable length of time. It was better to stop that study now and think again about that indication and how we might approach it in the future, rather than push ahead with a study that was just gonna tick over at one or two patients every quarter and then never come to a, you know, an answer, which is what we're trying to do. You know, we wanna use our cash to get answers to studies as quickly as we can. That was certainly one thing.
You know, cash was a consideration in thinking about that, not to, not to have that locked up and allocated to that study. The other one was thinking about profile of PXS-5505, and the interest that we're seeing in it, and the publication recently in myelodysplastic syndrome as well. All led us to believe that, you know, there is value in the strategic focus in hematological malignancies with this drug. That if we are gonna add on other indications going down the track and looking at the interest we've got in it and where other companies might wanna put this drug, then myelodysplastic syndrome might well be a better option than going down the solid tumor route, which as we know, is a difficult area to treat.
Lots of drugs fail in phase II. It's a real problem for, you know, clinicians and patients, with these ones where, you know, you're seeing liver cancer, I think with, you know, 12 months life expectancy in pancreatic cancer, the other one with, you know, less than six months. Regrettably, we felt it was better at this time to focus our resources in the current financial climate on blood cancers and myelofibrosis a priority, and then thinking about other ones we might add on later.
Excellent. Thank you very much, Gary. We are running a bit tight on time now, so I'll wrap up the questions there. Gary, thank you very much for your time today.
Pleasure.
If any investors do have any further questions, feel free to email them through by replying to an email you've received from Gary or David. Otherwise, Gary's contact details are available on the final slide of the presentation deck. A recording of this webcast will be made available shortly on the Pharmaxis website. Gary, on behalf of the investors in attendance and those that couldn't make it, thank you very much for your time today.
Pleasure. Thanks very much.