Earnings Call: Q1 2026

Nov 6, 2025

Summary

Major clinical trial readouts in osteoarthritis and GVHD are expected in Q2 next year, with strong cash reserves and a unique manufacturing platform positioning the company for potential commercialization and partnerships. Ongoing trials and strategic initiatives target large unmet-need markets.

I just wanted to say good morning and thank you for joining Cynata Therapeutics Investor Webinar for the September 2025 quarter. Presenting this morning will be CEO and Managing Director, Dr. Kilian Kelly, and Chief Business Officer, Dr. Mathias Kroll. Both will provide us an update on Cynata's clinical programs, corporate developments, and financial position, followed by a Q&A session. If you'd like to ask a question, please use the Q&A button at the bottom of your screen. You can submit questions at any time, we will hold questions until the end. As a reminder, this webinar is being recorded and will be available on the company website in the coming days. I'll now hand over to Kilian to formally welcome you all and commence the presentation. Thanks very much, Lauren. Thanks to everybody for joining us this morning. As we usually do with these webinars, we'll go through a relatively brief presentation with some key points of our progress over the last quarter and the outlook ahead, and then we'll leave time at the end for questions. As Lauren said, you can submit questions through the webinar system, and we will get to those after we conclude the presentation. This really is an enormously exciting time for the company. It is no exaggeration at all to say it's the most important chapter in the company's history, and that's because we have two major clinical trial readouts within the next six to nine months. Obviously, we'll cover a bit more about those in a moment. Both of those trials are designed to show efficacy of our products in diseases with unmet needs, really serious diseases with unmet needs. We really do have the potential to change how these diseases are treated. These trials build on positive data that we have generated from prior studies over the years. I'll also remind everybody that we have not identified any material safety concerns with our products, and that's a statement that holds true for the type of therapies that we're developing, MSC-based therapies in general. There's a lot of very encouraging data out there already, and hopefully these trials will build on that and really put us into a position where these results really could transform our trajectory, both from a commercial perspective moving forward and of course, in terms of a company valuation perspective. During the quarter, we didn't release a lot of news during the quarter, but that's not to say that there wasn't a lot happening, because there really was. Importantly, we continued progress with those two key clinical trials and in addition, a third trial in kidney transplantation. In the GVHD trial, we continued to enroll patients, and we've got very close to completing that. Again, I'll expand on that in a moment. In the phase III osteoarthritis trial, we've had more patients reach their final two-year follow-up visit, again, we're reaching the end of that process now. There was also a lot of work going on behind the scenes in terms of strengthening our relationships with key opinion leaders in these key indications and also other investigators and members of site staff involved in our trials and potentially involved in future trials. We also did a lot of work to help put us in a position where we're going to be able to progress after these key readouts, progressing towards late-stage development of these products and ultimately commercialization. We've also been putting a lot of effort into building the company's profile. That includes with people in the industry sector, including potential partners and other stakeholders, as well as those key opinion leaders, but also with brokers, investment funds, and other investors, and that's an ongoing activity as well. In terms of our balance sheet, taking our balance of cash at the end of the quarter, which was AUD 3.2 million, and the AUD 1.7 million R&D Tax Incentive rebate, which we received yesterday, combined, of course, that's AUD 4.9 million. In addition, we have a AUD 7.5 million standby capital facility, which we put in place during the quarter with Acuity Capital. That gives us another potential option to help bring in working capital for the company, again, at the company's discretion as we move forward. In terms of where we're at, as I said, we have two big clinical trial readouts coming up. That is our phase III osteoarthritis trial and our phase II acute graft versus host disease trial. We now expect results of both of those trials to land around quarter 2 next calendar year, so probably less than six months from now. We also, during the quarter, moved forward with our kidney transplant trial, which is a phase I/II trial in patients who've received a kidney transplant, and we have completed the enrollment of the first cohort of patients in that trial. The readouts ahead, as I said, really are incredibly important for us. To elaborate on each of those trials. Our phase II trial in GVHD is a trial that's being conducted in Australia, the U.S., and Europe, more than 30 clinical centers around those various countries. We've now randomized 59 patients in this trial, and those of you who've been following this will perhaps remember that our original target was to randomize a total of 60 patients. The aim of doing that was to have at least 56 who received study treatment. To explain what I mean by that is, in this condition, in particular, and conditions like it, things change quite rapidly. We have a phenomenon where people can be enrolled and randomized in the trial, but for one reason or another, end up withdrawing before they even receive the first dose of study treatment. We had estimated that we might have up to about four patients where that would happen. As it turns out, we've actually had eight patients who've been randomized without receiving treatment, so four more than we expected. As a result, we've made a decision to add four patients to the trial. Our new target is 64. As I said, we hit 59 as of the date of the quarterly. We are very close. We do anticipate completing that by the end of this calendar year. Hopefully, before that even. It is quite unpredictable the enrollment rate for a trial like this. We, as I said, are very close, and we look forward to announcing that completion very soon. That still puts us on track to have the results after the 100-day follow-up during quarter 2 next calendar year. The phase III osteoarthritis trial, which is being conducted in Australia only in partnership with the University of Sydney. This is a trial that's being funded under an NH and MRC Australian government grant. It was fully enrolled two years ago, more than 320 patients. The final patients are now undergoing their last visits. It's a two-year follow-up from the first treatment, so we now have the final few patients reaching that two-year follow-up. We're expecting the final patient visits to occur within the next two to three weeks. That, of course, is a huge milestone. This is a trial that has been running for about five years. It takes a long time to get to this stage, but we really are almost there now. We again expect results from that trial in around quarter two next calendar year, so this and the GVHD results will both be out likely very close to each other. I'll just remind everybody that following an advisory meeting with the TGA, the Australian Regulatory Authority, we are optimistic that results from this trial could support marketing approval of this product in Australia. The kidney transplant trial. This is early stage. We've had the first cohort of 3 patients in this trial where the aim is to use the cells to help prevent kidney transplant rejection and to enable us to move away from the drugs that are currently used for that purpose, which have a very poor toxicity profile. The first 3 patients have been treated and followed up, and there's now a DSMB review, which we hope to be able to announce the results of very imminently. I'll just briefly pass over to Mathias, who'll say a few words about what next once we get these results. A few slides back, you've already seen that our clinical portfolio is targeting really quite sizable markets, and that goes as well for our preclinical portfolio. Obviously, we've continued going out to various organizations, telling our story, and soliciting and receiving indications of interest. We received indications of interest, both prompted and spontaneously, and that's very encouraging. Interestingly, also, when we work with collaboration partners and contractors, some of these will actually refer us further, and that can open new doors. On the other hand, of course, you've also seen that some of the companies that previously were strong in cell therapies have shifted their strategic focus. That means the list of go-to companies that we've established constantly changing. There are new additions, couple of erosions. I think overall, I'm very satisfied with the list we've got and happy to implement as soon as our results are out. Definitely within phase II and phase III results as we keep emphasizing, you've got a lot of credibility, you've got a lot of significance, and that actually puts you in a category of partners that are very relevant in any circumstances. I think we will continue engaging with partners. We will continue answering their questions. All of this is going to heat up early next year. Looking forward to a very busy early 2026. Back over to you, Kilian. Yeah. Thank you, Mathias. Again, for those of you who've been following the company, you'll be well aware that what is unique about Cynata is the manufacturing platform that we have. This really is unique, and it sets us apart from all of the other players in the MSC sector. We are the only company that can produce clinical-grade MSCs from iPSCs. The advantage of doing that is that we can make these MSC products in a very scalable manner and in a consistent way using cells from the same donor without needing new donors. We really believe that this puts us in a position where we can be the global standard for how MSC-based products are made. As Mathias alluded to, one of the opportunities for us then in terms of what next is this puts us in a position to enter into licensing deals and joint venture deals and so on, to enable these products to be taken forward towards commercialization. The importance of this manufacturing breakthrough really is that there has been a barrier here, in terms of how these products can be made scalably and consistently. That hasn't historically been possible, and our platform makes that possible. We're pursuing multiple multi-billion-dollar markets. We also have a very strong intellectual property portfolio to protect this. I would also remind people that even in the absence of patent protection, which we do have, but even in the absence of that, there is currently no generic or biosimilar pathway for cell-based therapies. We believe that we have a very strong moat really here once we move forward towards commercialization. Just to conclude, before we move on to questions. As I said, we have a unique manufacturing process that really puts us in a position to potentially dominate this MSC field, which has huge potential to treat a wide range of different clinical indications. We have orphan drug designation from the FDA, which potentially gives us a number of advantages moving forward. As you've heard several times already today, we have two huge clinical trial readouts coming up very soon. It's an incredibly exciting time ahead and we look forward to sharing those results in the near future. Lauren, I think I'll stop there, and then we can move over to questions. Yes. We have quite a high volume of questions, so we will attempt to get through as many as we possibly can in the next 15 minutes. Please keep sending them through. Anything that we don't answer, we'll try to get back to you as soon as possible. You're also welcome to email investors@cynata.com, and we can get back to you via email as well. The first few questions all relate to the osteoarthritis trial. The first question is: If the trial shows pain and function improvements that are clinically meaningful, but it doesn't demonstrate halting or modifying of the disease, would Cynata still consider that trial a success, and take the therapy forward? Yes, I guess is the short answer. To, I suppose, set the scene there, our trial has two co-primary endpoints. One of them is measuring the pain and symptoms, and the other one is measuring cartilage thickness by MRI. The latter is really a measure of the progress of the disease, and if we can show an effect on that's what would be considered a disease-modifying effect. Needless to say, we hope that we'll be successful with both of those endpoints. If we are successful in one but not the other, that would still very clearly show that our product has a meaningful effect. A reminder that this is a large randomized trial with a two-year follow-up period. If we show a clear benefit in terms of pain and function over that period of time, that is certainly something that is meaningful and would warrant further development. I think if we were to end up in that sort of situation where we have one success, one doesn't meet the endpoint, we'll really need to look into the details of the data and what exactly are we seeing, because there's a lot of sort of different ways that could come about. For example, it could be that there is a clear trend towards success in the other endpoint, just not quite statistically significant, and that might indicate that we needed more patients or something like that. There's a lot of different ways that that could come about, and we'd need to really look at exactly what we have. In short, if we are successful in either of those endpoints, we very clearly have a therapy that works. While disease-modifying effects is the holy grail in osteoarthritis, ultimately, what matters for the patient, of course, is pain and function. Both of them are important in their own right, and therefore a success in either is a success. That's not to change the fact that we are hoping to see success in both. Sticking with osteoarthritis, when is the last follow-up patient due? What happens between that last patient and obviously, the results? Yeah. The last patient visit, we believe is within the next 2-3 weeks. This trial, as mentioned, is being run by the University of Sydney, so we don't have full visibility of exactly when visits are happening and so on. The latest update we've had from them, which is very recent, is that there are just a few patients to go with that last visit, and it should all be done and dusted in the next 2-3 weeks. We look forward to confirming that once it has happened. In terms of what happens then. First and foremost, all of the data that has been collected needs to be entered and analyzed. When I say entered, what that means is it's all put into a validated database, which has appropriate audit trails and so on. Importantly, in this case, one of the key assessments at the end of the trial is an MRI scan of the knee, which is used to determine if there has been a change in cartilage thickness. Those MRI scans have to be read by two independent radiologists, so all of that has to happen. When all of the data has been collected and looked at and so on, and entered into the database, there's a process that goes on which we call monitoring, which means somebody actually checking that all of the data that's been entered actually is correct and corresponds to the medical records. This is all part of the rigorous QC process that goes on with clinical trials. Inevitably then, there'll be times when things have to be queried, something's not clear, there's a data point missing, et cetera. All of that, needless to say, that is already ongoing for data that's already in the database. Of course, it can't be completed on the final few visits until those visits happen. All of that happens. When all of that data is checked and monitored and queries are resolved. We have what is called a database lock, but from that point onwards, no data can be entered or changed, and so on. The data goes to the statisticians for formal statistical analysis. That will generate a whole lot of tables and figures and listings and so on. Finally, when all that has happened and that has been checked and everybody is happy that it's all been done correctly, we'll have the results. That process does take a few months, which is why we have a final visit, hopefully this month, but results more likely to be a few months beyond that, database lock due next year. If the trial results allow for an approval, in the osteoarthritis in Australia, could a partner, via a sub-license from Cynata, also tap into additional markets via off-label use? First of all, there are certain markets where if we had an approval in Australia, there is essentially a very fast-track process to get approvals in other markets. They sort of recognize an Australian approval. That's one possibility. In terms of off-label use, the reality is, if the product was on the market in Australia only, that would only allow it to be used in Australia. For example, if somebody wanted to use the product in the U.S., not in the sense of off-label use, that wouldn't be feasible. There are other sort of compassionate use schemes and so on. I think the short answer is that we would only expect the product to be used in markets where it is actually approved. Yeah. Clearly, Cynata would not allow any off-label use to be promoted by any of our partners. We would very strictly control through our contractual agreements with any licensees what they're allowed to promote. Of course, off-label use can happen. That's in the physician's discretion, obviously. It's illegal for any company in any market, basically, to promote it. We would set up strict obligations and oversight, actually, in order to enforce that. Now, in terms of economic consequences, obviously, if some physicians in some countries decided to prescribe our product for similar indications outside of the exact approved indication and additional doses got sold, then obviously, Cynata would get the corresponding royalty or other agreed participation. Thank you. Still, this is partly osteoarthritis and then also trials in general, is that we've been told that we have enough cash to get us through until after the trial readouts. What does this mean for cash flow once the trial readouts have occurred? Yeah. Well, absolutely. It is correct. We have cash runway per current forecast beyond the end of this financial year. Into the middle of next calendar year. The trial readouts, as discussed, are expected before that. That is a fact, and that's a very good position to be in, of course. It's extremely important that we do have runway to get us past those readouts. In terms of what happens beyond then, needless to say, the business has to be funded beyond the middle of next calendar year. There are various ways that that can happen. As mentioned during the presentation, we have a standby capital facility in place that could potentially be used to bring in further working capital and to extend the runway. I'm not saying that is what's going to happen. It's an option we have. Of course, we have, as a listed company, there is the possibility at some point of raising capital through the more conventional means. Again, not saying that happens. There is certainly no plan to do that at the moment. Then the other important part of it is revenue through commercial partnerships, and that is something that we've been very actively working on and that we do aim to achieve. In terms of exactly what happens post results, that remains to be seen. Clearly, we will aim to bring further funding in through one or other of those means, and commercial partnerships is certainly a key focus for us. I should probably expand on that as well, which is as part of a commercial partnership, in addition to bringing actual cash into the company, in many cases, those partnerships would also remove the responsibility for funding further development for those projects. That, again, remains to be seen, depends on the deal, but that is often the case. For example, there could be a partnership related to GVHD, where the partner assumes responsibility for funding further costs related to that program. All of that remains to be seen, but of course, it's a very key focus for us in terms of exactly how we manage that. Thank you. We touched on use of stem cells for OA outside of Australia just a little bit earlier. Is the osteoarthritis trial targeting just TGA approval, or are you looking at FDA approval? Our aim is certainly to seek FDA approval and essentially global approval. It is important to explain there is a difference between the expectations in Australia versus overseas. This trial is taking place in Australia only, and the reason for that is it is an Australian government-funded trial through a grant, so that money can't be spent on conducting the trial overseas. Now, because it's an Australia only trial, but also because some regulatory authorities like the U.S. FDA typically expect two trials anyway, we do imagine that there will be the need for another trial to support approval in the U.S. We are optimistic that this Australian trial could serve as one of the two trials that the FDA would require. There is certainly a difference. We don't think this trial alone would support approval in the U.S. It hopefully will help to support approval in the U.S. in combination with another trial. It might be too early to speculate, but what would a further OA trial in the U.S. look like? We have done quite a lot of work on that, and the short answer is we're not sure yet. In part, it depends on what the results of this ongoing trial look like. There are some other questions. For example, exactly what the FDA will need to see from a second trial. There's a possibility that the trial would look reasonably similar to the trial that we've got going on at the moment, except that it would involve sites around the world. There's also a possibility that it could be a somewhat different and shorter trial. For example, if it wasn't necessary to have the same length of follow-up, because we've already, for example, hypothetically speaking here, but if we've already shown quite convincingly in one trial that we have the disease-modifying effect, it might be possible to argue that the second trial could have a shorter follow-up, for example. There's a few unknowns there, and those questions do actually potentially have quite a significant impact on how long that trial would take, for example. Okay. I might leave some of the osteoarthritis questions there because you could keep going. We might get back to some of these via email. A question on diabetic foot ulcers. It's almost been a year since the successful phase I results were announced. This investor is looking for an update on progress since then and what the next steps are. Yeah. When we announced the results, we said that at the time we were not going straight into another clinical trial, in part because we simply didn't have funds in place to fund that other trial, and of course, that remains the case. What we have been doing this year is working on what the next steps are and what that would look like, whether we move forward independently or with a partner. A key part of that has, of course, been engaging with potential partners. I'll also ask Mathias perhaps to expand a little bit on the partnering landscape in DFU, because it is a bit different in that the diabetic foot ulcers is really part of the wound management space. The companies involved in that space tend to be different to the companies that we're talking to for the other indications. I think where we're at now, before I hand over to Mathias to expand on this, where we're at now is that we have a decision to make about whether to go and fund a further trial ourselves to get us to a point where we add value to that program. If we look to do some sort of a co-development partnership deal in the near term and perhaps undertake a further trial with a partner. As I said, I'll let Mathias expand a little bit about the partnering landscape in this sector. Yeah. I think we've commented earlier in previous webinars that it's a particular space. It is certainly a great market with a huge unmet need. The types of companies that are first and foremost interested are obviously companies that are generally engaged in the complications of diabetes and wound care companies. Wound care companies tend to develop different types of products in different types of ways. There are lots of device-type approvals. Getting their heads around this type of living cell device combination takes time. There are some complexities, of course. It is a very rewarding and very significant indication, but it is also relatively complex and capital-intensive to develop. Development plans take some time to draft up, and it is a different type of development than what many of the potential partners are normally used to. That is why it means that these discussions can take time. Thank you. I know we've already gone over, but we do have several more questions. We'll just condense some of these down and move to the graft versus host disease, if everyone's happy to stick around for, say, five more minutes. There's a question around just the timing. When you said you could get 59 patients as of the quarterly, does that mean the September date, 30 September, or the date of the announcement of the quarterly? That 59 was at the date of the quarterly itself. Yeah. Can we expect interim 28-day results in this trial, as phase I interim data was published after each cohort? No. There is an important difference here in that this is a double-blind trial. The way it works here is that there will be a single analysis conducted at the end of the 100-day follow-up for all patients, and it's only then that the data will be unblinded. The difference in phase I was it was an open label study, and so we didn't have that blinding issue, so we were able to look at data as we went along. That's just simply not possible in this case. Yeah, unfortunately, we'll have to wait until the day 100 analysis. Thank you. I'd say this will probably be the last question that we can get to, and then we'll try to get back to people via email. Do you have any updates, or when can we expect an update from the SMART CRC? Mathias, do you want to take that one? Yeah. Yeah. I recently attended the official kickoff of the SMART CRC with a quite impressive delegation. There are many very interesting companies and labs and organizations for us to talk to, technology providers. All of these can help us open new programs, improve things about our technical operations, and so on. In fact, this afternoon, we're scheduled to have a very interesting discussions with a SMART CRC member. I, of course, can't put a time on when updates should be expected. SMART CRC is a great initiative. We are proud and happy to be part of it, and it'll help us. It'll help the entire industry. That's how we look at it. I think, yeah, updates will come as and when we enter into projects and collaborations that are ready to be announced. Thank you. Well, on that note, we'll take the last questions as raised and come back to that. Kilian, did you have any closing remarks for today? Yeah. I'd just, again, like to thank everybody for joining today and more broadly for your ongoing support. As I said, it is an incredibly exciting period ahead, and we really look forward to sharing these results as we get them.