Good morning, everyone. The clock has just ticked past 11:30, so we will get started with today's quarterly investor briefing with Syntara CEO Gary Phillips, who's gonna provide an update on the clinical trial program. My name's Alfred, and I'm from Syntara's Investor Relations team, and I'll just be online to assist with running the briefing. Just a quick run through the formalities. After the presentation, Gary will be taking questions from attendees. If you do have any questions for Gary, there's a sidebar at the bottom of your screen. It's just got a little speech bubble with a question mark. If you click that, it'll come up with a box where you can enter your questions, and Gary will address them after the presentation.
If you're on a smart device, your Q&A button is at the bottom of your screen as well. Obviously, the last day of a quarterly, so it's very busy down the ASX, but Gary has been very generous with his time to offer this update and take any questions. Gary, I won't take up any more of your time. Let me hand over to you.
Okay. Thanks, Alfred. Thank you. I see we've got quite a few people online, so it's, thank you for the interest that you continue to show in Syntara. We have a busy few months ahead of us. You know, I think this update aims to both review some of the information that we provided in the last quarter and give an opportunity for questions around that. Also look forward to the next six months in particular and what's gonna be happening. The quarter was marked really by two big things. I mean, the first one was on our 5505 trial in myelofibrosis.
Now this trial is in open-label, so we put out a first of the data from that, from the first six patients, and also shared that data at the world's leading hematology conference in America, the American Society of Hematology, and also with industry, potential industry partners at JP Morgan in January. I'll give you a bit of feedback on the kind of reaction we got to the data that we have, and as I said, happy to answer questions on that. The second thing that happened was that we completed recruitment in the skin scarring study in Perth that's being run by Professor Wood. That means that we are now on track to deliver data from that in quarter two this year.
That's a, you know, a second exciting thing that's coming up. Then we ended the year with strengthening the Syntara board, and I'll talk a little bit about the rationale and the choice of the two appointees that we put onto the board. Then look really to the future and talk about what you should be following next if you do have an interest in us as a company. Next slide, Alfred. The interim data that came from PXS-5505. Now this study is being run in patients who are ineligible or intolerant for the current standard of care, which is a JAK inhibitor.
As you may remember, myelofibrosis patients who have a rare kind of bone marrow cancer, they are often treated with a first course of treatment is on a JAK inhibitor. Those drugs relieve the symptoms of myelofibrosis, but don't do too much about the life expectancy for those patients. They currently have a life expectancy of around about five years. Now, a lot of patients find the JAK inhibitor is very difficult to tolerate. Their side effect profile and they actually cause a lot of cytopenias, so drops in cell counts, make them quite difficult for patients to stay on. Once a patient comes off of those drugs, they typically have between 11 and 14 months left to live. These are patients that are going into our study.
They're quite rare, which is why it's been difficult to find them. You know, they are patients who are in a fairly serious way by the time they come onto our drug. With that as a, as a context, we've enrolled 18 patients into this study. Six of them have completed the 24 weeks of treatment, so six months. We've had six patients drop out, but pleasingly none of those dropouts have been due to adverse reactions to the drug. They've all been due to a lack of clinical response, which is the kind of thing that you'd expect to see in this patient group. A lot of them are actually progressing quite quickly at this stage, and some of them transfer to become leukemia patients in the first month or so they're on drug.
There's nothing really that the drug can do, so the physician has to take them off that and use a more suitable drug for them at that point. The endpoints that we presented last quarter and then we took to ASH were first of all, that this was a drug that was well-tolerated. It's interesting talking to the key opinion leaders at ASH and who came past and looked at our poster, looked at the data. We gave a further breakdown of the improvements in platelet scores and hemoglobin within the poster.
You know, despite the, you know, our interest in efficacy and what we're doing to blood cell counts, the first thing that all of them hone in on is the fact that this is a well-tolerated drug. They have, you know, got used to, in myelofibrosis, using drugs which are pretty toxic. Most of the drugs which are in the clinical development pipeline also cause cytopenia in patients as well. You know, these patients suffer because they haven't got adequate blood counts, and they're dropping because of the fibrosis in their bone marrow. At the same time, the drugs we use to treat them, in quite a high percentage of patients, are causing those blood cell counts to drop even further.
They really focus on this, first of all, that, you know, the drug is well-tolerated. It's kind of almost a free choice for them to add on because they know they're not gonna make the patient worse. That was the first thing. Second thing is the honing in on the, you know, the fact that we were showing across the board either patients who were stable or improving. We had two out of the six patients showed an important improvement in symptoms, and that's measured on a symptom score that these patients have, like sort of a composite score chart that they fill in. Five of the six patients had either stable or improved bone marrow fibrosis of at least one grade.
You know, confirmation that the drug mechanistically, is doing what we expected it to do and what it did in the animal models, that prompted us go into the clinic in patients. Importantly, I think, you know, five of the six had stable or improved platelet and hemoglobin scores. Two of those patients more than doubled their platelet scores. When in talking to the key opinion leaders, they really focused on the importance of that. There is a large unmet need still within myelofibrosis, and even when they look at the potential new drugs coming through, that nothing really addresses these patients in terms of their when they have low platelet counts. Low platelet counts are a double whammy for these patients.
It not only obviously causes them ongoing problems, but it also means that they have to drop the dose of the JAK inhibitor they're on very frequently, so they stop getting benefit from that as well. If you can increase the platelet count in these patients, then that really is something which is a very attractive thing. I think the overall feedback we got, both from the clinicians that we talked to at ASH, and then at JP Morgan in January, I spoke with a number of companies who are active in the myelofibrosis market and have drugs either in the market or in development or are interested in the market. You know, the data thus far is very encouraging.
We clearly need to see more data, and we do expect more data in the first half of this year. We expect to have a major data update before the middle of the year, which we hope will confirm what we saw in these first six patients, and that the story continues to be one of being well tolerated, which I'm very confident about. Also that we're still seeing those signs of efficacy in this very difficult to treat patient group. That's where we are with that study. You know, really pushing hard to get the recruitment to the end. And looking forward to seeing more data from this in the first before the middle of the year. Next slide, Alfred.
Moving on to the scarring study. Now, this again is a similar drug to PXS-5505, and it blocks all of the lysyl oxidase inhibitors. It's in a topical formulation, so it's applied to the skin. We're using it in a study of 50 patients. This is a placebo-controlled study of patients who've had a scar of 10 centimeters square or more, and they've had to have it for at least one year. These are patients with established scars. The theory here is that by changing the cross-linking of the collagen and reticulin fibers in the skin, we can change the ongoing scarring process and modify the appearance and the functionality of scars, even if they're already been in there for a considerable length of time.
This is the first of a number of studies planned with the group in Perth, and we've been thus far, very encouraged. You know, the great milestone that they reached just before Christmas was that they fully recruited the study. These patients are being followed for three months, which means the data will be out in quarter two, the top line results. We're working with Professor Fiona Wood and her team in Perth to fine-tune the way that we're looking at the data, the analysis procedures that we undertake, so that we're all aligned in terms of the data that we will produce in quarter two. Thus far, last quarter, we did announce that, you know, the first eight patients in this study who were, we know were on active drug.
We put them on active to check that we were getting the lysyl oxidase inhibition in these patients as that mirrored what we saw in healthy volunteers when we applied them in an earlier study. We know from that small subgroup that we are inhibiting the lysyl oxidase enzymes in the skin. We can measure that. We also measured changes in the structure of the skin and the amount of collagen that was in the skin biopsies as well. All things which line up, which suggest that the drug is actually working in changing the structure of the scar. The clinicians noted positive changes in both appearance and the pliability of the scars as well, which was encouraging.
We do now need to wait and see the results now of the other 42 patients which were randomized 1-to-1 to placebo and active, which, as I said, we aim to get them in quarter two. That's an important study. Again, quite a bit of commercial interest in this study. This drug is the only one, to my knowledge, that's in the clinic that's really dealing with this mechanism and dealing with changing the appearance of scars. We've had a lot of interest from clinicians as well as industry in terms of the other potential indications we could go into.
There's a lot of interest in keloid scarring as a potential commercial indication, where there's a really high unmet need, a really big clinical benefit that could be accrued from that, from treating patients with more serious scars over larger parts of their body area. Even indications such as Dupuytren's, which is a fibrosis in the tendon sheath in the hand, which causes a trigger finger or a finger that comes at a different angle and can't be extended. There's lots of potential future for this and a lot of interest in what we're doing because of the mechanism we have and the novelty of this program. Next slide, Alfred. There's just to conclude on the sort of trial front.
The company currently has five different studies that are either ongoing or planned to start in this year. They're all in significant markets with very large commercial opportunities. Two of them, we've already seen preliminary data which suggests the drug is working, and we're waiting for further data to corroborate that. We're gonna get this, that data in the first half of this year. This is a really exciting period when the fruits of many years of a scientific research program start delivering clinical results in patients that really will underline whether these are strong commercial assets that the company can further develop. Next slide, Alfred. I just wanted to wrap up this update with a mention of the board.
You would notice if you were following us that we appointed two new non-executive directors to the Syntara board in the last quarter. The first of those was Dr. Simon Green, the second one was Hashan De Silva. You know, when the board sat down and looked at its capabilities, we reviewed the experience and the skill sets of the board in the light of the untimely passing of Will Delaat last year, who had been a long-time board member, we felt that we needed to have additional commercial acumen on the board.
Also a more current connection with the capital markets, from an investor relations point of view as well, to strengthen the overall roundness of the board and its ability to look after the interests of shareholders and work with management to make sure that our programs are focused on delivering that value. Obviously, Malcolm has been with us for a long time, a very established and experienced chairman that covers a wide range of industries, and across a wide range of with a strong financial background, that has brought an awful lot to us and guided us through some periods in the past. Kathleen Metters really brings the early research focus to the board, given her background in Merck.
Neil Graham comes with a very strong clinical development background. We felt we really, between the existing board, had the sort of early-stage research and clinical development covered. Was really as we moved towards commercializing our assets and looking at partnering, and maximizing the value of the company, we felt that, you know, we needed these two further talents. I, and I'm delighted with both the appointment of Simon Green and Hashan De Silva . I look forward to them joining the board if. Simon's had his first board meeting and Hashan will be joining us for the next one. Yeah, we look forward to for that coming forward. Next slide, Alfred. That just brings us to the future and the news flow.
What can you expect in this quarter? We are recruiting in the liver cancer study in Rochester. That's been difficult to get underway, but they're all set up in that clinic, and they are actively screening patients to find their first patient to go in. The myelofibrosis study, we expect to see fully recruited. In quarter two, because the PXS-5505 myelofibrosis study is open-label, we do anticipate putting out a significant data update before the middle of the year, so sometime in quarter two. That will depend on the number of patients going through, how many of them complete the six months, and when we feel there's enough data there to make a material announcement. We anticipate doing that before the end of quarter two.
From the LOX topical drug, we expect to see the data from that established scar study, also to start recruiting in a further study looking at one of those other indications that I talked about. As well as that, we've got the two major publications that are due from key opinion leaders in other cancers with PXS-5505. One of them is in pancreatic cancer and the other one is in myelodysplastic syndrome. Both cancers with a huge unmet need, where I think the data that they've generated using our compounds in preclinical studies is truly compelling data and has certainly moved the needle with a lot of the people that I've presented it to under a confidentiality agreement.
Finally, the PXS-4728, the study that's looking at treating patients with a serious sleep disorder, potentially preventing the onset of Parkinson's, that study will restart recruiting in 2023. As a reminder, that study is fully funded by Parkinson's UK. We're just going through the final stages of setup now with the finalization of the protocol and getting the centers signed up. That will start recruiting in quarter two. The second half of the year, of course, will be marked by the full data set from the myelofibrosis study, and reports on both the safety and efficacy data from that, which we're, you know, we're very excited about.
I think that brings to an end the sort of formal part of this moment. I'm happy to take any questions.
Excellent. Thank you very much, Gary, for that informative presentation. We have had a couple of questions come in already, which we'll address in a sec. If anyone does have any questions for Gary, please just type them into your Q&A box. If you're on a computer, that's the little speech bubble with the question mark at the bottom. Gary, the first question comes from Rhett. What are the differences between PXS and Constellation's Mylo drug? Is their $1.7 billion takeover achievable for Syntara?
Yes. Interesting question. The Constellation drug, I guess the main difference between their drug and our drug is that their drug is clinically more well-advanced. The takeover of Constellation by MorphoSys happened after they'd already completed a Phase 2b study, where they had data showing that if you combine their drug with a JAK inhibitor, then you see better outcomes for their patients. You know, they're in Phase III now. I think that the $1.7 billion, you know, that program was their lead asset, so it's not as though they've. The $1.7 billion is based on a huge valuation in some other drug they've got in another disease. It is based on that drug in myelofibrosis largely.
You know, it's been very encouraging to see the amount of commercial value that's put on a clinical benefit being accrued by a drug in development. Now, I think, you know, we are at an earlier stage, so we're certainly not gonna attract a $1.7 billion takeover just yet. The study that we've got ongoing and the preliminary data we've already presented suggests that we will have a profile which makes our drug attractive when we get to that stage. You know, the next, I think when we talk to companies, when I talk to companies at JP Morgan, clearly they can see that, despite the other drugs in development, the profile of our drug is still very attractive. It fulfills an unmet need.
It doesn't have some of the tolerability issues that the Constellation drug has, as well as others in the pipeline, and the drugs on the market at the moment. I wouldn't say that the Constellation deal precludes us doing a deal. I think it's highlighted that, you know, in one area, a further benefit as ascribed huge commercial value, and that if we continue to produce data along the lines of the first six patients that we had, that kind of opportunity would be open to us in the future as well. I find, you know, encouragement on two levels there.
One, on the commercial value that companies are ascribing to this, and secondly, on the profile that we've got and the fit that we have with the current unmet need that's in myelofibrosis, which was endorsed by the key opinion as we talked to at, around our poster.
Excellent. Thank you, Gary. Question from Chris: How should we think about the potential increase in R&D spend with the ongoing clinical trial pipeline, and what does that mean for the available cash runway?
The, you know, the cash that we have, at the moment, I think it's, about AUD 21 million, as a pro forma number at the end of December. That takes us well into 2024. You know, clearly the next studies that we have to run in, myelofibrosis or myelodysplastic syndrome or another scarring study, will require us to have more money to do that.
Behind us, we will have, you know, a clear set of data which I anticipate, if it's positive, will see to a, you know, a dramatic increase in the valuation of the company, and allowing us to move forward confidently to either raise money to do the further studies or to partner one of the assets in order to raise non-dilutive funding to take the company forward. You know, the options for the company are certainly being kept open at the moment. We will see how the data develops in the first half of the year to decide how we want to fund the company going forward into the next.
Thanks, Gary. Kind of a follow-up question to that. Another one is, what are the options when Parkinson's UK funding is exhausted?
We, you know, we don't consider ourselves to be a CNS company. You know, I don't imagine sitting here talking to you in two years' time saying, you know, we are now into Alzheimer's and Parkinson's. The funding from Parkinson's UK, you know, has made it possible to look at the potential of this drug in both a sleep disorder, which is a commercial opportunity in itself. There are no treatments available for the particular sleep disorder that these patients we're treating in this trial have. showing the potential for slowing progression into Parkinson's. you know, we are already engaging with potential partners in the future.
It could be that we continue to receive, philanthropic, grant associated, albeit under commercial terms, money from funds like Parkinson's UK. I know that there are other funds, in the Parkinson's world, like Michael J. Fox Foundation, for example, that are, you know, following our program with interest. I think the data we get from this study, again, will give us the option of either continuing to do the next study with external funding or to find a partner to share that burden, with us and take the drug forward.
Thanks, Gary. Question from Dennis. Myelofibrosis trials is recruiting around one patient per month. What are you doing that will allow recruitment to complete this quarter?
We've, the sites in the US opened up in the last quarter. We spent quite a lot of time with the US investigators at ASH talking about it. They're, they are very enthusiastic and I, you know, although, you know, I can see the number of patients that are currently in screening that we anticipate, you know, some of them will get through into the study. That's what gives us the confidence to say that we'll finish recruitment this quarter.
I guess as well, you know, the number of patients that we need, you know, in the FDA and its guidance, when we receive the IND, you know, we sort of all agreed on a number of 24 based on a certain minimum number of patients they needed to see data from in order to sort of say, "Okay, that's enough." That assumed, a lot of this was a very sick group of patients. You'd see quite a lot of dropouts, and you'd see some tolerability issues. Now, the dropouts have been relatively low, and in those dropouts we've also collected data, sufficient data for the FDA still to assess those patients from a safety perspective. Also the drug's been very well-tolerated.
You know, I think when we say we'll complete recruitment, we'll certainly complete recruitment to the satisfaction we believe of what the FDA want to see from this study in the first quarter.
Thanks, Gary. We are just about right out of questions in the queue. If anyone does have any more, please pop them in. Another one here from Dennis. What are your distributors telling you about in-market demand for Bronchitol?
It's a bit of a mixed picture. I think in the U.S. is the market where, you know, we, I guess we've placed a lot of focus with the FDA approval and then the launch by Chiesi, our commercial partner and distributor there. We've been tracking that quite closely. We review, you know, their marketing program, the amount of resources they're pouring into it, and we've been very pleased to see what they're doing. They physically launched the product at the North American CF meeting in the last quarter. We're waiting to see this next quarter's data to see whether that's had an impact. I'd say that the response thus far before that had been disappointing.
I think Chiesi and ourselves were disappointed by it. There were a number of factors with COVID-affected markets and the difficulty they had of accessing prescribers in hospitals and getting CF patients into clinics in order to do it. You know, we sit there with a, with a caveat around that and think, "Well, yeah, not sure." On the other side of things, you know, we've seen continued demand and growth in Russia, where those patients don't have access to other medications and Bronchitol remains almost the only treatment that they can access for cystic fibrosis patients. There, the demand is growing still very strongly. It's a, it's a bit of a mixed, a mixed bag. It's, it's not easy, Dennis, to give you a very a one-size-fits-all answer to that.
Excellent. Thank you very much, Gary. All right. We are running a bit tight on time, so I'll wrap up the questions there, Gary. Thank you very much for your presentation, taking the time to answer shareholder questions today. If anyone does have any more questions, I've got Gary and David's email addresses there on the screen. They're always very generous with their time. A recording of this webcast will be made available shortly on the Syntara webcast. On behalf of all invested in attendance, Gary, thank you very much for your time today.
No, thank you for your time. Really appreciate it.
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