2026 Outlook webinar and Q&A. All participants are in a listen-only mode. There'll be a short initial presentation provided by management, after which we'll focus on Q&A based on questions that have been submitted via email and live during the session. So if you have a question you'd like to submit, please do so using the Q&A function within Zoom. On the webinar from Syntara today, we have CEO Gary Phillips and is joined by Non-Executive Director Hashan De Silva, whose fund KP Rx is also one of Syntara's largest shareholders. To begin, I'll hand it over to Gary. Please go ahead.
Thanks, Matt. Welcome to all of you, and thank you for sparing some time this morning to listen to us. We thought about the year ahead, and I think Syntara stands out amongst its peer group as having a really busy 2026 with a pipeline of assets which are going to deliver a near-constant flow of news as we go through the year. We thought we'd start the year by actually giving a chance for shareholders to ask questions about that pipeline and for us to go into a bit more granularity about that news flow, what to expect and when as we head through the year. I'm delighted that Hashan's joined me today.
Hashan sits on the board with me at Syntara, so he's well across all of the operational issues of the company and the strategy going forward, as well as, Matt said, being one of our largest shareholders as well. So we go back, Hashan and I, go back a long way to the time when he was in Karst Peak, originally looking at us as an analyst. So he's been on the journey with me as well. I think having his perspective on the call gives an opportunity for shareholders to ask things from a different point of view as well and hear different opinions about the stock and the news flow that we've got. So just very briefly, I think many of you are familiar with us. We are a clinical-stage drug developer.
We've got a 43% institutional backing, which is quite unusual with a company with this level of market cap. We've got multiple shots on goal, and all of that comes from our own internal drug discovery group, which is funded a lot by the R&D tax credit from Australia. So it's a really efficient way of building pipeline in a small biotech in Australia. We've made the most of that and to the point where we've actually got five clinical trials which are delivering clinical trial data, efficacy, and safety data during this 12-month period. So that's an outstanding level of return, getting that number of opportunities to read out within a short period of time. As well as that, all of those assets have got pretty long intellectual property on them as well.
So they're valuable from a potential partnering commercial perspective because they've got a lot of patent life ahead of them. And on top of all that, a lot of those trials have been funded by non-dilutive cash. So we announced a pancreatic cancer grant earlier in the year. That brought us to over AUD 11.5 million in non-dilutive grant funding we've been awarded in the last three years, which is supporting that news flow and those trials going forward. So a really broad pipeline from a company which is delivering a lot of assets to value inflection points in a short period of time. That's just a reflection on where we are at the moment with the share price. So we're standing at about a AUD 50 million market cap, as I said, with that just over 40% institutional.
So there's smart healthcare specialist money invested in Syntara, and we've got a lot of coverage as well. So Canaccord, Bell Potter , Euroz , and Evolution Capital all issue coverage reports on the company. As we headed into 2026, we had a really strong last quarter. We presented amsulostat, our lead asset at the American Society of Hematology, ASH, in Orlando in December. And that was really the foundation for that asset, really building momentum as we entered into this year. On top of that myelofibrosis data presented at ASH, we had the second myelodysplastic syndrome study. It was a related hematological cancer initiated in Australia. So that's the MESSAGE Study. It goes along with the AZALOX Study, which was already started earlier in quarter four.
So both of those studies now recruiting, and you'll see in a minute when we're going to expect to get news out of those.
We broadened our regulatory interaction by going as well as to the FDA, to the European Medicines Agency. They granted us orphan drug designation. That is a slightly different process from the FDA, a very rigorous one where they have to see that the drug is going to add value in the indication that you're asking for before they grant it. So it's not just, "Is this a small group of patients?" This is a small group of patients, and we can see from the results of the studies that this drug is potentially going to add a lot of value for patients. And then obviously that announcement that we were getting granting for a pancreatic cancer study that's going to start later in the year as well. So the lead asset, amsulostat, really building momentum.
And then backing that up, our pipeline assets, PXS-4728 in a Parkinson's disease patient group who suffer from a sleep disorder, iRBD. We announced that was fully recruited with data expected later this year, also triggered a payment from Parkinson's UK of AUD 1.8 million, which we expect to get in this quarter. And also the skin scarring program with SNT-9465 progressing into a hypertrophic scarring study. And in quarter four, we had a great webinar. For those of you who caught it, those of you who haven't, I strongly recommend it as a good watch with Professor Maitz really digging into what it is about scarring that makes it problematic for patients, why there's so little in development at the moment, the hope he had for SNT-9465, and I think the very innovative design that we have now going forward.
On top of that, we ended the quarter with a cash balance of AUD 12.3 million. That cash balance takes us into 2027, so a strongly funded position with a lot of news flow to come. Now, just before we go into the Q&A, just to break down that news flow for you. So in the first half of the year, lead asset, amsulostat, we are in discussion with the FDA to get them to approve our development plan to go forward with the asset in myelofibrosis. That approval of that plan also triggers partnering engagement and potential deals with that asset at that point. So that is the trigger point that we're waiting to see for this lead asset. And those discussions are ongoing now, and we expect feedback from the FDA and announcements in the first half of this year.
Those two MDS studies, AZALOX in Germany, MESSAGE Study in Australia, the AZALOX Study is in high-risk MDS patients. The MESSAGE Study is in low-risk MDS patients. So two different patient groups. The AZALOX Study is running a bit ahead. We expect interim safety and efficacy data around the middle of the year, and then initiation of the phase II study on the back of that in the second half of the year. The MESSAGE Study, which is running slightly slower, we expect interim safety and efficacy data in the second half of the year. On top of that, the pancreatic cancer study, which we talked about earlier, trial initiation the second half. And just a reminder that both of those MDS studies and the pancreatic cancer studies funded by non-dilutive cash at this point of their development. Skin scarring program, two different assets.
The first generation scarring program, SNT-6302, is in the hands of Fiona Wood and the University of Western Australia in keloid scarring. We're expecting interim safety and efficacy data in that keloid scarring study around the middle of the year. The hypertrophic scarring study is open. It's recruiting. We expect top-line safety and efficacy data in the second half of the year with that. So those two studies, and in particular the one in hypertrophic scarring, are the ones that really unlock value in that asset. We think this is an enormously powerful study. It's going to give us clinical proof of concept and evidence that will allow us to really accelerate that program and potentially opens up immediate partnering as well for that asset. Finally, the study in iRBD Parkinson's, I mentioned just now, that study has completed its recruitment.
We're now on a schedule to release phase II data. We will have top-line data from that study in quarter two this year. With that, I will finish my presentation, and I'm happy to hand back to Matt to lead us through the Q&A. Matt, back to you.
Thanks, Gary. As you mentioned, we'll move on to the questions. And again, as a reminder, if anyone would like to submit one, use the Q&A panel within Zoom. Gary, the first question is, you've now got three separate blood cancer studies running in parallel. What have you found? What have you learned about where amsulostat's clinical sweet spot might ultimately be across risk categories and lines of therapy?
So we've completed studies in myelofibrosis, a number of them now, in patients who were not responsive to standard of care, the JAK inhibitors, and also those patients who were still on JAK inhibitors but really weren't being well treated. I think the things that we've learned about the sweet spot are, first of all, the safety seems to be really robust in a wide range of patients now. We've got patients who've come out the other end of ruxolitinib and not been treated on it at all, but 12 months really from end of life, and through to patients who have been three, four years on ruxolitinib and those patients who have just started on ruxolitinib as well. And throughout all of those, we're seeing a really clear picture, which is that the safety seems to be pretty robust.
The last study, I think, showed us that time to efficacy was actually a bit quicker than we thought. We extended that last study in suboptimal patients from the six months we looked at with the ruxolitinib failures through to a 12-month study because we thought from a mechanistic point of view, our drug would take a little bit longer to work. Actually, we saw really strong responses to symptoms inside three months. So that's really quite interesting in terms of unveiling where the sweet spot for this thing. And I think it's really important from a profile point of view. Patients who see benefit early on are much likely to stay on drug, are going to stick with the therapy because they immediately feel better. I think in myelodysplastic syndrome, it's a bit too early to tell.
The preclinical work that we did with the University of Heidelberg in Germany was in high-risk patients. So if you ask me to look at those two studies, one in low and intermediate risk, one in high risk, I think the one in the high risk is where we've got evidence in the preclinical study that we should see a fairly rapid response in numbers of red blood cells quite quickly in that study. But we need to wait and see the clinical data now, whether the preclinical work translates into the clinic at the middle of the year. In myelofibrosis, I think going back to that finally, I think suboptimal patients, mechanistically, we know now that the amsulostat blocks a pathway that bypasses the JAK inhibitor efficacy.
So having patients who are still on a reasonable dose of ruxolitinib, not at the end of their disease, and who are still on a reasonable dose, we think is probably that sweet spot that that question was trying to get to. So yeah, I think we've learned a lot in the studies we've got, and I think we've got a clear idea about where to go in the future in myelofibrosis. And in MDS, we need to wait and see.
Thank you. And then Hashan, what qualities of amsulostat do you think make it attractive as an asset?
Yeah, I think it has many features that make it quite attractive. If I start at high level, I think the safety that we've seen now across many different patients in many different stages of the disease gives amsulostat optionality, whether it can be used early stage or late stage. That's quite unique to a lot of drugs in development in oncology, where you see that the safety profile kind of limits the drug's use. Mechanistically, so if we're talking about just in myelofibrosis, it's quite unique. A lot of the drugs in development are kind of pseudo anti-inflammatory drugs. And so combining those with other anti-inflammatory drugs can have diminishing returns quite quickly. And then the side effect profile of those drugs means that you can't really combine them in patients who otherwise feel well.
With amsulostat, theoretically, you could combine it with the JAKs that are on market or any of the drugs that are in development. Mechanistically, it should be synergistic. And when you think about that mechanism of action, it is a pipeline in a pill in the sense that it can be used in these blood cancers. It can be used in solid tumors where fibrosis is an issue. It can be used even outside of oncology where fibrosis is a hallmark of disease. One of the things that I've spoken about before is in solid tumors, fibrosis is often key to the tumor spreading. And we've seen now, at least in breast cancer, that fibrosis predates the metastasis. So fibrosis is required in order to terraform the liver so that breast cancer can survive in it and other areas as well. So mechanistically, this is very, very exciting.
There are more things that we could explore than I think Syntara can afford to. The other kind of key benefit here is long IP, that it's also unencumbered by having to pay royalties to anyone else. If you look at some of the other drugs that are on development on the ASX, they might just have protection from the Orphan Drug Designation. They might have some kind of used patents, a manufacturing patent. amsulostat has a plethora of patents, but the composition of many patents that extend out decades. And that makes it very attractive to a potential acquirer, especially given the optionality of this drug across many disease states that you could do dozens of clinical trials and still have protection out well into late 2030 and potentially even into the 2040s.
Thank you. And what do you think the market is missing about the myelofibrosis clinical data?
I think a few things. I think first, when it comes to safety, well, I think the market's looking at the data maybe in isolation or thinking about how this data can be combined with other treatments in development or other JAK inhibitors given the safety profile of amsulostat. So I've heard some people focus on spleen size saying, "Look at the spleen size here. Look at the spleen size there. Why isn't amsulostat delivering a similar spleen size to XYZ drug?" That needs to be taken into consideration with the safety data, right? So you can reduce the spleen size in these patients really well by a drug called Navitoclax, right? But then the patient essentially suffering in every other aspect of disease. So it's not about just what is the headline results. It's about the headline results hand in hand with the safety data.
I think also the market got very confused or very distracted by the FDA feedback about going into a phase II before going into a phase III. I mean, that is normal drug development, right? The FDA is not asking us to do anything different than we would have done otherwise. Now, we were shooting for the stars. Maybe we're going straight into a phase III, but the FDA has brought us back to Earth. But from a shareholder perspective, there's not materially that different, right? So you are funding a phase II trial, which costs a whole lot less and significantly de-risks the asset before you have to raise for a much larger phase III trial. So from a risk-adjusted IRR perspective, there really isn't that much difference to an investor today than going straight into phase III, I believe.
The other aspect that I think that the investors are missing and something I've discussed extensively in the past is that we know now definitively that binding to an inhibiting LOX prevents fibrosis. We've seen that in the topical skin scarring trials where we saw a 30% reduction of myelofibrosis. Now, when I did that podcast a while ago, I said the question left to be answered is, "Does doing that in myelofibrosis cause modification of disease?" And I think we're even closer to answering that because we've got the phase II data that shows quite a rapid onset of symptomatic relief and then a reduction of spleen size. The results that we saw from that myelofibrosis trial, at least from my perspective, Karst Peak's perspective, far exceeded what we thought was possible in this patient subset. And so we are very excited about the future.
Another thing that I'll quickly mention in myelofibrosis specifically, the safety profile and the speed of action means that this drug could potentially be used even earlier in the disease course, potentially even as early as pre-JAK. And that would make amsulostat the backbone therapy for myelofibrosis where everything else gets added to amsulostat. No other treatment in development can be used in that patient subset because of its side effect profile. And so I think when I look at everything else in development for myelofibrosis, to me, this is the most exciting asset in myelofibrosis development.
Hashan, one for you again, Gary. Which aspects of the future trial strategy do you see as the most critical to get right to support successful late-stage development and then regulatory approval and commercialization, obviously?
Right. So yeah, if you're talking about development and regulatory approval and commercialization all in one bucket, I think I've spent a lot of time in the last 12 months talking to multiple potential partners. So the clear feedback we have from them is that we have a really attractive and well-differentiated asset for all the reasons that Hashan and I talked about a bit earlier. And what they want to see, as well as us, is the sort of trigger point to sort of going forward with both the development and potential commercialization is that clear guidance from the FDA in the first half of this year that will really unlock that value. And I think the thing that we are absolutely focused on now is demonstrating the safety and efficacy of this drug in a controlled trial.
We need to absolutely nail and get a very clear baseline of the patients coming into the study so we understand what their history's been, what their symptom score and spleen size is coming in, and then that really clear comparison with those baseline patients going into both a control arm on placebo and a JAK inhibitor and on our drug and a JAK inhibitor combination. Our open-label study had patients treated out for 12 months. And I think we saw improvements in patients who had been three years on ruxolitinib, sometimes even more. And those patients were still getting more than a 50% improvement in their symptoms and a reduced spleen size, which all of the key opinion leaders, when looking at our open-label data, thought was remarkable and could clearly say it was absolutely the drug working.
We now need to do that in that controlled trial setting, and that will give the confidence of both the regulator and potential partners that what we have is something which will get through regulatory approval and will bring real benefit to patients.
Thank you. The next question's for you again, Gary. What's the optionality the company has around partnering and how that might mitigate cash requirements phase II-B and then beyond?
Yeah. So I think Hashan already mentioned that we had looked to maybe take this straight to a phase III, and we're going to go through a two-step process rather than a one-step. So we're going to do phase II-B. the partnering after phase II-B is almost the same as partnering after a phase III, right? I mean, you're delivering safety and efficacy data in a controlled setting. So the value of the asset after phase II-B is still really, really, really strong. So I think when we talk about partnering at this point, I'd say we remain very open and flexible on deal structures. We're not trying to force this into one particular size hole. We've got a really strong pipeline. So any deal on amsulostat doesn't necessarily look like a company acquisition.
It looks more like a, in broad terms, it looks more like a licensing deal with significant cash upfront that gives value for the development that the company's done to this point. So I think you could see at this point, the board, including Hashan, has given me clear direction that we want to see optionality going into the first half of this year. So we want to explore what might be available from a partnering perspective, and we want to explore what might be possible in terms of raising the cash to do phase II-B. so I can imagine regional deals that trade territory rights for cash to support the study, as well as a global licensing deal, and probably everything in between as well. So yeah, at this point in time, we've put ourselves in a really good position.
We've got efficacy and safety data, which I think, as I've mentioned before, is compelling despite the fact that it doesn't have a control group. We're about to get that feedback from the FDA in the first half of this year in terms of agreement on the study design going forward. And then we'll see. We'll see what happens out. But yeah, the next few months are going to be really critical for the company and the drug, and we will maintain open dialogues with all interested parties at this point.
Thank you. And with your day-to-day roles with KP Rx, you're obviously looking across the market. So how have you seen Big Pharma's appetite for M&A and licensing changing over the last couple of years, and what sort of deals are getting done?
Yeah. So there's been quite an interesting evolution, particularly from the market to 2020, 2021, where we saw a lot of deals focused on platforms or kind of very early-stage deals looking at kind of groundbreaking technology that might generate more than one lead candidate. That has kind of done a complete 180, and we're seeing the deal focus really go to assets that are de-risked, that are late-stage, and with big pharma willing to pay out for those assets rather than take the risk of going early. In myelofibrosis, in particular, we've seen that in the last two to three years with essentially most assets that will finish a randomized controlled trial being acquired. That has positive trial data from a randomized controlled trial. And I think that's really been driven by the fact that the patent cliff has come right now.
2026-2027 is really where the patent cliff falls. We haven't seen Big Pharma launch anything, well, outside of the GLP-1s, launch anything blockbuster. So we're seeing much more activity. JPMorgan this year, the big healthcare conference, most of the conversation was around that patent cliff and finding assets that can generate revenue within two to three years. I think that's been a big shift that was not the focus of discussion kind of five to six years ago. On the kind of wider market as well, since 2021, really, we've been in a one-way trend for biotech, right? It was down and to the right where the XBI was testing new lows every year. 2026 feels different.
We saw the IPO markets kind of opening in the U.S. towards the end of last year and big deals, big IPOs, big secondaries getting done, and the secondary market, secondary liquidity for biotech in particular really coming back. There's definitely more optimism out in the market this year than I think there's been in the last kind of at least four years. And so we're quietly optimistic that 2026 is the turning point for this, well, 2025 was the lows, and 2026 is when the market starts to recover for biotech and medtech.
Yeah. It's probably worth adding to that, Matt, in terms of Hashan's comments on patent cliff, ruxolitinib, the AUD 4.5 billion drug, of which probably AUD 1.5 billion is in myelofibrosis, is off patent in 2028. So that's going to shake up the myelofibrosis market significantly at that point with, yeah, makes amsulostat really attractive going into that particular market with that dynamic going on.
Thank you. And then just changing tracks still on amsulostat, pancreatic cancer and the recent announcement there you alluded to, Gary, obviously a tough indication for drug development. What specifically gives you confidence that amsulostat's mechanism could translate into clinically meaningful benefit there?
Well, I guess first thing you say is it's a free shot for us, right? So we didn't have to make an investment decision on this because the MRFF, the Australian government grant for this to the Garvan Institute, has made this possible. So for Syntara shareholders, this is a free shot, which is great. The preclinical work that we did with the Garvan over a number of years, which resulted in a Nature Cancer publication in 2023, was an outstanding piece of work. It really showed that when you add amsulostat to standard of care chemotherapy, what you see is amsulostat breaking down the fibrotic nature of a pancreatic cancer tumor, reducing intratumoral pressure by doing that, which allows in the body's own killer cells, T cells, to go in there and attack the cancer, as well as chemotherapy. And the result of that was the tumor shrunk.
You've got longer survival. It was really a wonderful piece of preclinical work. So this trial is looking at translating that. I think the trial design itself is really, really helpful in that, as you mentioned at the outset, the clinical development in pancreatic cancer is notoriously difficult because patients tend to be diagnosed quite late. Often, they've metastasized before they come in, and they haven't got very long to live. So if you're going to address the fibrotic nature of the tumor, the question you have is, how long do these patients have? How much can we change the nature of the tumor? How much benefit can it bring as the disease is progressing rapidly in the background? So I think the great thing about this study is that they've got a huge amount of experience in imaging and genetics within the Garvan Institute and The Kinghorn.
So they will be looking at each individual patient that comes in. They'll be looking at what specifically does the treatment at a micro level within the tumor. So even if these patients are still progressing for whatever reason, we will learn a lot about the combination of amsulostat and what it does with chemotherapy on a cellular and mechanistic level with these patients, which will allow the treatment regimen to evolve and to pick the right patients where we can bring benefit. So I think it's a free shot for the company, but I think the study design is brilliant, and I'm really looking forward to seeing that translation of what was a tremendous bit of preclinical work into the clinic.
Thank you. And we've frequently described the pipeline as being multiple shots on the goal. So how do you internally decide what programs deserve the incremental capital versus being partnered or even paused?
Well, you can see from our record, I think it's an easy question to answer. Amsulostat is our first priority, absolutely. It's already got clinical proof of concept. It's already got lots of interest from potential partners, and there's ongoing regulatory dialogue with already a clear pathway forward to commercialization. So that asset has all that you would look for in a biotech company to say that is an investable asset which deserves to be prioritized and accelerated as fast as possible. Beneath that, the scarring program that we have, which came out of the overall look at fibrosis, and it was a long development, co-development with the Fiona Wood Foundation and UWA. That's been a really cost-effective program to develop in conjunction with the Fiona Wood Foundation. But it's now on track to get proof of concept by the end of this year.
It's a relatively small study, but extremely innovative, I think. So it's funded. It hasn't cost us a lot of money. It hasn't distracted at all away from amsulostat. And I think the trial design is right. We just need now to wait and see. The bet has been placed. The trial is funded. The commercial potential in scarring is huge, and we've got a lot of interest partnering incoming when people see what we're doing. And all they're waiting to see actually is, can you change a scar? And I think this study, we will see very clearly now whether we're going to get that photograph almost as a before and after, what a scar looks like before and what it looks after three months with this drug. So it has a bit of investment but hasn't detracted away from amsulostat.
And then there's PXS-4728, which is absolutely a free shot. It's fully funded by Parkinson's UK. If it doesn't come off, it doesn't impact us at all. But if it's positive, then it's a huge upside for shareholders. And we'll again seek probably to use partners or non-dilutive cash to advance it. So I don't see PXS-4728 being a distraction. It's ongoing. The study's been paid for, and now we're just waiting to see what the result is.
Thanks, Gary. And then for you, Hashan, if you look at Syntara's portfolio as a whole, as Gary's just spoken to, which program do you think is most understood by the market, and why do you think that disconnection might exist?
If I take a step back from an asset perspective, but look at the whole portfolio, I think what the market is, or what Syntara's suffering from, is almost a conglomerate effect that you see in the likes of where Berkshire Hathaway, in some of the parts, is worth more than the whole. Where it's difficult with Syntara, given how many programs are being run, unless you do this for a living, to look at each of the assets and come up with the probability of success and a value for each of the assets.
And when you try to kind of back-solve Syntara's valuation at an AUD 42 million-ish EV, AUD 40 million EV, versus all of the programs that they've got running, randomized controlled trial in Parkinson's, a randomized controlled trial in skin scarring, a phase II trial in pancreatic phase II-B trial of amsulostat in myelofibrosis, it doesn't make any sense, right? You've got single-asset companies which arguably have the same, if not a lower probability of success, as one of Syntara's programs having a substantially higher valuation than the company as a whole. So I think investors need to take a step back and look at all the shots on goal that Syntara has. And to kind of pat Gary and the team on the back a little bit, how that's been done so capital-efficiently, right?
So PXS-4728 is a randomized controlled trial in a very difficult disease to treat, to recruit, a trial to run that is being fully funded by third-party money. I mean, I can't think of a single example of an ASX-listed biotech that is doing that or that has done that in the past. And that's just one of two randomized controlled trials that are being run and being funded that doesn't take away from the lead asset. I mean, that's quite amazing, really. In terms of the asset that is misunderstood the most, I still think that is amsulostat, right? People don't understand. I think what people are missing is that the safety profile makes amsulostat or gives amsulostat so much optionality to be used in all the way from first line all the way to end-stage patients. The mechanism of action is not disease-specific.
It's not specific to myelofibrosis, right? So if this works in myelofibrosis, it will de-risk its potential in myelodysplastic and other hematologic, but also de-risk its ability to work in other kind of solid tumors. And it is one of the rare drugs that I've seen in Australia that is a pipeline in a pill, right? So if we had access to unlimited capital, we could be running dozens of phase II trials with amsulostat. Obviously, that's not the world we live in. And I think that kind of concept of the safety profile and the mechanism of action makes this drug so unique and so versatile that it makes it a very attractive drug to a partner once we've proved it in a randomized control setting.
Matt, just to add to that, my discussions with potential partners for amsulostat, and when we talked earlier about what does a licensing deal look like, well, it looks like one where you also get value not just from the lead indication, myelofibrosis, but where you also get value from myelodysplastic syndrome and potentially solid tumors as well. We will be looking to make sure that the sort of valuation does take advantage of all those things that Hashan talked about and the optionality that a pharma company with deep pockets would be able to access almost immediately. So yeah, it very much drives our thinking in terms of how you try and get value from the broad range of indications that not only is it being developed in, but also it could extend to.
Thank you. It's only been touched on briefly so far, but the topical fibrosis programs, Gary, are structurally different from the oncology assets. So how does that change your thinking around development speed, partnering timelines, and ultimate ownership of that?
So yeah, I think we talked about this a bit earlier, but I think we've got a really innovative and efficient way to get to clinical proof of concept now with this program. It went through several iterations. We took a lot of advice. We talked to a lot of surgeons, plastic surgeons, dermatologists globally, and also companies globally about what they wanted to see in this area. There aren't any drugs approved for scar modification that have a pharmacological direct action on the scar. Professor Maitz, in his webinar, talked about unlocking the scar by breaking down the scaffolding, the fibrosis interlinks between the collagen fibers in order to change the scar structure. So we've now got that, and we've managed to get, we think, to get clinical proof of concept out of a phase I study.
So that study, which is ongoing now, the patients act as their own control. They'll get placebo and active looking at the scar. We will take tissue samples from that, so we'll be able to see structurally what's going on with the scar. We've got several different and again, quite innovative instruments which will look at the flexibility of the scar, the volume of the scar. We'll have really high-quality photography as well with independent and blinded ranking of the scars at the end of it. So we've done all of that with a phase I study in Australia, which is the most cost-effective way of getting through to something like that. So it's going to be fast. We hope it gives a definitive answer to the question of whether pan-LOX inhibition modifies the scarring process. Once we have that, then there are many routes forward open to us.
This could be a company of its own, right? A skin scarring program like this is. If you look at what the value of, I think at the moment, the global market for skin scarring modification, there's more than AUD 1 billion being spent on silicone products which have no real tangible effect on scarring longer term. So having the first drug which can pharmacologically change the structure of a scar and change the scarring process, it will open up a lot of possibilities for us that include, yeah, company-specific ones, investments, and partnering as well.
Thank you. There were a couple of questions across the board that came in around PXS-4728. Just wondering what the next steps are there, what it might look like if the results are positive from the current trial and the likely funding options going forward.
Well, as you know, that study is in patients that have a sleep disorder who are a very high probability of developing Parkinson's disease or other neurodegenerative diseases in the future. So there's two bits of inbound interest in the asset from pharma companies. One is from sleep companies, and we're seeing an emergence of interest in sleep as an indication. Narcolepsy, all other kinds of sleep disorders are beginning to see more investment going into them, and therefore that's driving more research and more partnering activity.
So we've got interest from companies that are focused on sleep saying, "Look, if you can produce some changes in the quality of life of these patients from a sleep score point of view, then that's of interest." We've also got interest from companies that are really focused on Parkinson's and other neurodegenerative diseases that would look very much at the PET scanning that we've got of these patients' brains and looking at whether we're reducing inflammation in these areas of the brain. Because again, that would be novel. It would be something that would be a clear indicator that you might be able to slow progression or stop progression of these diseases as they go forward. So the two different ways.
We know also from a global thing, and Hashan noted this as well, is that the uptick in deal values and interest in CNS has risen rapidly in the last two years. So there's money going back into CNS at the moment. So we've got partnering potential here if the data looks positive. But also, I want to pay respects here to the philanthropic organizations in Parkinson's who made this study possible, Parkinson's UK in particular, through their virtual biotech organization within their group. This was the biggest investment that that group had made, and it was the most advanced one. They'd not invested in a phase II study, blinded phase II study before. The Michael J. Fox Foundation, which also were very interested in what we were doing, are paying a very close watching brief on this as well.
These organizations work together to advance treatments which are going to be helpful for Parkinson's. I think we will also get a lot of inbound interest from those philanthropic organizations as well if this study, which they have funded, comes up positive in quarter two this year.
If I can just add to that as well, Matt, there was a very insightful question that was put to us just now through the Q&A about what the BI trial that they did in NASH can provide some read-throughs or some learnings for its use in iRBD. I'll just hit a couple before I hand back to Gary. I think I want to hop on the safety again, right? We've got a safe compound that is now being trialed in pre-disease effectively. It's not in patients with Parkinson's. It's in patients with a sleep disorder that might develop into Parkinson's later. The safety profile means we can dose these patients even before they're symptomatic to hopefully prevent disease. That goes back to what I was saying with amsulostat.
The other key insight that I have from that trial is that we know that the drug crosses the blood-brain barrier. We know that it binds to and inhibits VAP-1. So we have a pretty good idea of the pharmacokinetics of the drug. Now, the question that we need to have answered is, does that mechanism of action cause a reduction in the microglial activation? But yeah, Gary, keen to hear your thoughts on that question as well because I thought that was quite an insightful question.
Yeah, no, I think that's actually true. And I think when we were talking to Parkinson's UK originally about the study, the thing that attracted them about this was just how well developed this drug was. Boehringer, despite the fact that they handed it back because they didn't feel in a NASH setting, which was very, very competitive, that they felt it had the right competitive profile for them to invest what would have been probably north of AUD 500 million in clinical development. It came back to us and they said, "Look, we think the drug's safe. It's just not got the right profile." And of course, when you now look at it, they did all of the studies that you need to do to go into phase III. They did all the GMP they developed a better manufacturing process.
We purchased GMP material from Boehringer back that now is being used in this study. So Boehringer paid us, I think, over AUD 80 million we got out of it from the sales to them. They handed it back. We got it back with not having to pay them anything, but it came back with them having invested probably, yeah, AUD 100 million-AUD 200 million in actually getting it forward to this point. So this is a really advanced asset, which if we show efficacy, is a really attractive one for a pharma again to step in and pick up for all those philanthropic organizations to drive through to something that will benefit patients.
Thank you. The next question's for you, Hashan. And stepping back from Syntara specifically, how do you see the current biotech market bifurcating between assets that get funded and those that don't? And what characteristics do you think investors and pharma are prioritizing right now?
Yeah, so like I was saying before, I think the focus has really shifted to drugs that are on market, that have a clear mechanism of action. Sorry, not on market, sorry, drugs that are in the clinic with a clear differentiated mechanism of action. Me-toos aren't getting funded like they were in 2020, 2021. It's much harder to raise capital for preclinical stage companies. So the focus really has been to, okay, so if this drug is at phase I, phase II, what is the mechanistic rationale for this drug working in this disease state? And what is a path to market and how do we get it to market?
We've already seen that translating in Australia now, actually, even very early on in 2026, where we saw a company, I believe it was yesterday, raise north of AUD 500 million for a drug entering a phase II trial with a very clear mechanistic rationale about why that drug will work in that disease state. The big investors were U.S. healthcare institutional investors. 10 years ago, that would have been unheard of in Australia, raising that kind of much, much, much money on the ASX, particularly from U.S. investors. So I think Syntara's asset, amsulostat in particular, but also the scarring assets, sit at that sweet spot where we've got substantial safety data, we've got early mechanistic data, we've got with amsulostat, we will have a clear path to market in this half, and their clinical stage assets. In amsulostat's case, it's a pipeline in a pill.
So I really think that amsulostat or Syntara is where the market is at today to be able to really kind of capitalize and exploit the assets that they have.
Thank you. One for you both, I think, is: are you seeing a shift in how Big Pharma values these clinical-stage assets? For example, is there more emphasis on durability or biomarker clarity or regulatory alignment? Just some thoughts on that.
Yeah, perhaps I'll just go first. I'm not seeing a huge shift there. I think that perhaps there has been a little bit of a change in that they're looking for things which have a broader applicability. So the days when you had sort of really niche drugs that might go forward in one small area are still there. But I think that probably with some of what we've seen with the GLP-1s is that you can pick them up and then they go into several different indications. They're broadening them out and they're getting differentiation between similar stage molecules and similar assets by taking them down different clinical development paths. And I think that is a real advantage for amsulostat in that it could go a number of different ways.
This is why I said at the outset that when we talk to companies, we're not being restrictive in what we might consider because the value proposition for amsulostat is different depending on which company you talk to and what their particular strategic priorities are. We'll keep that open. I think you're seeing that broadly, that this idea of having optionality and more of a platform approach is really attractive for pharma at the moment.
Yeah, it's a good question. I was thinking, I mean, when you look at the podcast and the webinars, big pharma has been talking about precision medicine and smart clinical trial design for 10+ years. Have we really seen that translate into how they do M&A? I don't. I don't. I mean, to Gary's point, I think it's all about the data. They're looking later stage. They're looking at what the data is and their confidence in the ability to get this to market as soon as possible. And this whole kind of I think we all want to be focused on precision medicine and personalized medicine, but that shift has been more gradual than I think maybe people expected it to be 10, 20 years ago.
Thank you. Just coming back to the skin scarring, a question that's come through, Gary, was, is the company's strategy to license the skin scarring treatment or take it all the way to market? I think you touched on it a bit before, but if you can just return.
Yeah, I think we keep our options open. I think if that study comes back and shows that the drug really does make a difference to the way that these hypertrophic scars evolve against a placebo, then we will have a source of interest in terms of companies that want to. I can't tell you how many companies I've talked to who have been searching around and looking for something that works in scarring and they haven't found anything, and they keep on coming back to us and say, "Please let us know when you've got some results. Please let us know when you've got something that works." So I think that level of interest, inbound, makes immediate partnering possible, but it also suggests that there's enormous value for shareholders in hanging on and developing it as well.
We'll see how that trial goes and look and see what happens. Yeah.
You touched on the patients who stayed on treatment of amsulostat. Someone's asked, for those patients, after the end of the suboptimal study, are they still continuing on treatment?
Yes, as far as I know, yes. Which again is just testament to what Hashan emphasized earlier, that the safety of the compound, right? So these are patients who, on average, were on ruxolitinib for three years in a disease which has a five-year life expectancy from when they start on a JAK inhibitor. So it's really good to see those patients that reached 12 months staying on, still getting benefit. Symptom scores were across the board, almost 50% or more improved, and smaller spleens as well. So great to see those patients staying on. And we were delighted that all of them decided to do that. It just spoke volumes. We look at the data, obviously, and we dive in and we spend hours poring over the data.
At the end of the day, patients saying, "Please keep giving it to me," is the acid test for all drugs.
Just one final one again that I'll throw to you both is, how important do you see it right now for an emerging biotech to show optionality across various indications rather than just a single hero asset?
I think that's one to ask the investors. Matt, I think different investors have different profiles. There are those that want that black-and-white situation leading up to that, getting a clear answer. There are those that I think will value what we have, which is one lead asset with a very defined timeline, already with positive phase II clinical data, which has been substantially de-risked because of that. At the same time, a lot of other news flow, which can add value going on in the background as well, which they're not having to invest in. I think it depends on your investment profile as to whether you like that or not.
But I hope that we appeal to a broad section of the market and that as the news flow starts to emerge this year, more people will see the value in Syntara and the fact that there's a long way to go now this year upwards, I hope. Yeah.
Yeah, so that question, Matt, I'll frame it differently. It's, where is your marginal dollar better spent? It's really a capital allocation question, right? So if you've got AUD 100, where should that be spent? Should that all AUD 100 be spent on one asset, or should it be, or have you maxed out? So if you have more capital than you can spend on that one asset, then you can spend on others. But in Syntara, actually, it doesn't matter now because the other assets are being funded with non-dilutive capital, essentially from grant funding. So Syntara can still stay laser-focused on amsulostat and get that product developed and through the market. Whereas the optionality is being given now thanks to wonderful groups like Parkinson's UK, UWA, grant funding from the MRFF. It gives us free shots on goal.
I mean, like Gary mentioned, PXS-4728's trial results that will come out this half, that's a free kick on goal. And if it works, it's wonderful for Syntara and wonderful for the patients. But it hasn't taken away from the focus or the capital availability of Syntara. And I mean, I personally have never seen that for a biotech company. And that's where I think the pipeline really comes into its own.
All those excellent questions and answers from you both. Gary, I might just throw it back to you to provide a concluding comment.
Yeah, well, I hope that you all found this interesting. As I said at the outset, it was designed to give a bit more granularity on the news flow that's to come and what's going to drive value, what potential's there, and when it's going to happen. We'll keep you updated as we go through. But the team and I are really excited about the year ahead. We have made our choices. All of the programs now are well advanced and with studies recruiting or already recruited and coming to conclusion. I hope that for those of you that are not currently investors, you put us on your watchlist.
For those of the already investors, think about your position because I think at the current prices, Syntara still makes a lot of sense as an investment at the moment, given what else is going on in the market as a whole. Thanks very much for your time.
Thanks, Gary, Hashan. Thanks to everyone for joining.
Thank you.