NeuroScientific Biopharmaceuticals Investor Webinar. I'm Jane Morgan, Investor and Media Relations Manager, and today I am joined by our CEO, Nathan Smith. During the webinar, Nathan will be providing an overview of the previous reporting period in light of the recently released Quarterly Activities Report, as well as providing a company update and overview of their anticipated upcoming milestones for the company moving forward through to FY 2026. Today's webinar will be followed by a Q&A session, so to ask a question throughout today's webinar, please use the Q&A function, which can be found at the bottom of your screen. Nathan, I'll hand it over to you.
Great. Thanks, Jane. Much appreciated. Let me get the slides up. Excellent. All right, so welcome, everybody. It's great to be here, and thank you, Jane, for organizing this and the rest of Jane Morgan Management's fantastic team. My name is Nathan Smith, and I'm the CEO, the new CEO of NeuroScientific Biopharmaceuticals, ASX : NSB. I'll condense down to NSB for the rest of the webinar. What I'm really here to do is to share, and I have the privilege to be able to share the new chapter that we have at NSB. We've acquired a new technology that happened earlier this year, and because of that, we've brought in new leadership and expertise, me being one of them.
That pivot and that evolution that we've had of NSB is really, I think, where the news to the market and everything that you've—all the activity that you've seen over the last quarter and most recent announcements, that's what's fueling all of the activity that's there. That evolution has come with the acquisition of a patented stem cell platform called StemSmart. This stem cell technology is really focused on addressing unmet needs, and it hits a lot of large potential markets. It also has tailwinds because this stem cell technology is very similar to Mesoblast, their product, which received marketing authorization in the United States last year and has really elevated their share price in their organization in treating patients with pediatric graft versus host disease. That patented stem cell platform is a new opportunity.
Again, that technology has the capability to impact multiple high-value indications across inflammatory, degenerative, and autoimmune conditions. Most of these conditions are for patients with unmet medical needs, right? That target market is actually very huge, and I'll get into that later. The great thing about this asset and this stem cell technology platform is the fact that we have data. We already have data from multiple early-stage clinical trials. We know that the product works. We know the product works in a number of the indications that we're looking at. Our initial focus is in Crohn's disease. We've got some fantastic data around Crohn's disease.
The other reason why we're targeting Crohn's disease is that the current treatment options really are pretty limited once patients have failed the conventional treatments, and that includes antibiotics, steroids, and actual biologic drugs, these huge blockbuster biologic drugs like Humira and Stelara. We also have a large pipeline to come, and that includes our capability and our ability to expand on our IP platform for the stem cell technology. Just as background for NSB, we've got a robust company profile and a strong balance sheet, as you can see here on the slides. We talked about the STEMsmart technology platform and improving clinical efficacy. On top of that, we're also in the process of securing our supply chain.
I've talked about the STEMsmart MSCs, and one of the big great things to know about STEMsmart is the fact that, again, it's already been manufactured under TGA-accredited facilities, and the safety profile is fantastic. That's great for a stem cell product that is going to go from—is taken from a donor, and it's going to go into a number of patients out there. The safety profile has to be rock solid. That's very similar to Mesoblast's product, which has been approved by the FDA in the U.S. and has paved the way from a commercialization perspective. I think the next big piece to really talk through is really around our activities around the quarter. As you can see, I've captured here all of the announcements that we had over the last quarter.
The acquisition of the STEMsmart technology platform happened at the end of June, right? It has been a really busy four to five months. As you can see, we appointed both myself and Dr. Kathy Cole as the CEO and Chief Medical Officer. On top of that, we have been doing a number of different activities to help secure our strategy moving forward. That includes the evolution from bringing on Qiagen, our new manufacturing facility, to help de-risk the commercialization strategy and the clinical strategy moving forward, and to bringing on our Clinical and Scientific Advisory Board, which I will talk a lot more about later, which was announced last week. On top of that, we continue to do work. Once the acquisition was completed, we initiated a special access program for patients with fistulizing Crohn's disease here in Australia.
I'll get into some specific details there. The announcement around the manufacturing capacity, I think, and the relationship with Qiagen is really critical because one of the big things for us moving forward is not just being able to manufacture this product, but to be able to scale and to de-risk that process moving forward so that we can move into larger phase clinical trials and towards commercialization. That relationship and that announcement where we announced our partnership with Qiagen is huge because Qiagen is one of the largest contract manufacturing organizations of cell therapy products here in Australia. They have over 25 years of experience working with the TGA. We're working very heavily with them, leaning on their expertise.
On top of that, we're also looking to establish a number of different advisory panels and clinical research organizations and partnerships so that we can carry out our strategy moving forward. I'll get into our timelines and milestones in a little bit. Moving forward, I did mention that we had opened up a special access program here in Australia, in Western Australia, for patients with Crohn's disease. Going a little bit more into Crohn's disease, why is Crohn's disease important? The fact of the matter is that—what is Crohn's disease? Crohn's is an inflammatory—it's an autoimmune disease where your own body attacks its own cells. That happens in the gastrointestinal tract and is really highlighted by chronic severe inflammation that happens over time and is something that just continues.
It affects patients of all ages, from young children all the way to the elderly. Clinically, about 30% of those patients do not respond to treatment, do not respond to the conventional therapies. They become refractory. This is out of a huge population, over 75,000 Australians, over a million in the United States, 68 million people globally live with Crohn's disease. 30% of that population is a huge population, and they really do not have strong options when it comes to treatment. What we are looking to do is to fill that gap. What we have done is we have actually gone to an even more difficult subset of patients. These are patients that actually form fistulas. They are refractory, they are not responding to treatments, and they are actually forming fistulas.
These are sores or ulcers in their gastrointestinal tract that really do not respond to any treatment. Surgeries are no more an option. How do we help these patients live with a better quality of life and avoid surgery? That is why we have actually opened up a special access program here in Australia. Background on special access, it is a program where we work very closely with the clinician to provide a product. That product has to be approved by the TGA. The patients that will receive that product have to be approved by the TGA under the special access scheme called a category B pathway. These patients do not have any options. There are not any options that are out there on the market at this time.
The clinician is looking to treat them with an experimental unapproved therapy like STEMsmart. The clinician will apply to the TGA. The patient will get approved by the TGA for treatment with the STEMsmart cells. They will get treated, and we have this treatment and are working with the clinician so that this treatment is under a protocol. Under that protocol, they'll get treated for a period of time, and then they'll have results that are analyzed, and the clinicians will share those results with us. The target here, and we just recently announced the fact that we've treated the first four patients under that special access program. We're looking to treat up to 12 patients. Whether or not we make it to 12 patients, that's to be determined if we can identify those patients.
At this point, we're treating the first four, and those first four patients, we should have results towards the end of the year, and we'll probably announce those results in January. We will continue to treat patients through Q1 of next year and finalize the results of all of those patients by mid-year at the latest of 2026, calendar year 2026. These patients, again, like I mentioned, they have very few treatment options, and getting the STEMsmart cells is a fantastic way for them to see if this experimental treatment will actually be able to—okay, there we go. Sorry about that, guys.
Just finalizing on the special access program, it's a great way to have these patients that have very few treatment options to give them some hope and potential that our cells actually can reproduce what they did previously in our earlier phase clinical trials, but for this patient population in need. Moving on from the special access program, we also announced recently that we've established our Clinical and Scientific Advisory Board, as you see the members here. That's being led by our Chief Medical Officer, Professor Catherine Cole, and then Dr. Marion Sturm, who's our Chief Scientific Officer. She's actually the inventor of the STEMsmart technology. She's also on the advisory board, advisory team, along with some clinicians and scientists, some scientist clinicians, including Dr. Ashley Irish, Dr. Lena Thin, Dr. Michael Musk, and Dr. Ewan Moodley.
All four of these doctors are very familiar with our product and mesenchymal stem cells in general. There is a diversity of strengths across the board. Dr. Lena Thin is actually the gastroenterologist we have been working very closely with for the special access program, and she has done an amazing job in identifying patients and working with clinicians all around Australia to bring patients, fistulizing Crohn's patients, into the special access program. Dr. Michael Musk has a lot of work in lung transplants, and Dr. Ewan Moodley has worked with development of MSCs and other technologies.
What we're really looking at in establishing this advisory board, again, since we've pivoted to and evolved our NSB's strategy towards stem cell technology, is really getting guidance not just on MSCs and the indications, but on different treatment options and the ability to further develop our product so that we can broaden our IP platform as well. One of the great things that—what we're looking for this advisory board to provide to us is really, I think, key touch points with key opinion leaders in the space, in each of these different spaces, be it kidney disease, obviously Crohn's and gastrointestinal lung disease, and other evolutions as well, help us with clinical trial and study design, and then also being able to, I think, broaden the credibility and help us with our future research.
That is really the guidance that we're looking at with this clinical advisory board. We actually have our first ad board meeting next week. That will be fantastic, and there will be a number of actions coming out of that as we progress our strategy moving forward. Finally, what I'd like to cover is our ongoing roadmap and milestones. As I mentioned earlier, we've got the special access program that we're progressing. We treated the first four patients with the intent to treat more patients throughout early next year. We will provide and be able to share the results of that study or of that program by mid-year next year.
Really, the main intent is for that special access program to feed into a phase two clinical trial that we're planning to run here both in Australia and the United States. That's really key because taking data from the special access program and being able to take data from our earlier phase clinical trials will really support us being able to extend to the United States and to treat patients in the United States so that we can access that market as a broader strategy towards future commercialization and be able to access that market as we go down the pathway. We've already initiated startup activities for that phase two trial. We're in the process of vetting clinical research organizations and regulatory groups as we intend to file our IND next year.
We are already looking at setting up a pre-IMD meeting with the FDA in Q1 of next year. We are working through all of those startup activities with the intent to initiate that trial by the end of 2026. In parallel with those startup activities is also transfer of the manufacturing with Qiagen and being able to scale that manufacturing to support that phase 2 trial and future trials that we have in commercialization as well.
The intent is once we start to get data from that phase two trial is really looking at how we can partner with some of the other commercial organizations out there that might be interested, and that could potentially include Mesoblast or some other larger organization with the intent to go to move to a larger phase three study in refractory Crohn's and then submission with both Australia and the United States for marketing authorization in the future. If everything goes well, that may be 2030, that may be 2031, things like that. Really, what we're looking to do is to progress this treatment with these patients, get fantastic data, be able to partner and take that to market. In parallel, on top of that, we also are looking at our other indications.
This is why it's really important that we're working very closely with our Clinical and Scientific Advisory Board so that we can look at the other indications out there, what makes sense to the market, what is the next patient population that we need to be going after strategically. Then moving forward with starting up those activities, looking at phase one or phase two trial depending on the data that we have and being able to progress that moving forward. I think some of the big—and this is when we talked about some of those other targets, these are some of the key clinical targets that we're looking at. Obviously, Crohn's disease is what we're targeting right now. Kidney transplantation, which Dr.
Ashley Irish, who's part of our Clinical Advisory Board, has tons of past experience and has worked with us previously on the special access program. Lung disorders like COPD and lung transplantation, that's where Dr. Michael Musk comes in as well previously. We've also done some studies in GVHD. It really depends on what the indication here is and what the market access looks like for that indication. Moving forward, I think really what we're looking at is focusing on Crohn's disease and really meeting that unmet medical need there for patients that are in need, helping develop our technology such that we now have a very easy—and sorry, not an easy, but a very clear pathway to commercialization, being able to follow on the marketing authorization that Mesoblast achieved late last year.
Then being able to progress that moving forward using our patented platform, which is STEMsmart, obviously, and then taking and then continuing to build on that IP and further develop that with our clinical and scientific advisory board. Getting STEMsmart into that phase two trial and being able to take that towards commercialization is critical. Then being able to expand into other indications as we further develop our IP is really what we're looking at. That is what we're really focusing on. Coming up, the big announcements will really be—the key news items will really be the results for the first four patients, which we should have, I think, should be announced in January. Then the special access program finalizing by mid-year of next year. That will give a strong indication as to how STEMsmart's working, especially on this very difficult.
If we see some very positive results with these special access patients that have fistulizing Crohn's, that leads into some fantastic—not only will this help with enrolling patients in our future phase two, but it will also help us with getting clinicians on board across Australia and into the United States as well. That is really what we are looking at in the near term. In the longer term, we are establishing the manufacturing pathways and getting everything ready for the phase two clinical trial that we are starting late next year, really setting the foundation for commercialization of this new asset that we acquired in mid-June. I will leave it at that. I think that is really a great, a strong summary of what we have been doing over the last four to five months. I am happy to take questions.
Thank you for that, Nathan.
Again, webinar attendees can use the Q&A function at the bottom of the screen should you have any questions for Nathan. You actually just touched on this, but just to reiterate, obviously, the four patients are now being treated under the special access program. Maybe just if you can elaborate on why that's such a pivotal milestone for the company. Of course, you did say this, but results are expected to arrive in January 2026.
Yeah. The STEMsmart technology had been in earlier phase clinical trials, but they were back in, I think, early around 2014, 2015, when we got the results from the Crohn's, the refractory Crohn's trial. Those results were fantastic, absolutely fantastic.
We treated, I think, around 20 patients, and we had, I think, a 78% response rate, which is absolutely unheard of for patients that had failed previous lines of treatment. These were patients that were refractory. They had not responded to the conventional treatments that are out there. We got some great data, but that data was pretty old. I think from our side, what we wanted to do—and there were reasons for that. The asset had gone through a patent challenge previously and had gone through some tricky situations. The acquisition by NSB earlier this year was, I think, a godsend for the asset. From our side now, how do we get more recent data? How do we reinvigorate the market and the clinicians and the patients? That is why this special access program is pivotal for us.
The data that we capture from this is going to help us immensely. What it does is it's going to help us inform the phase two trial moving forward, right? What dosing do we need to do? What patient populations do we need to be looking at, right? How do these patients respond to this? Do we need to be looking at maintenance doses after the initial treatment that is done? I think that there are a number of things strategically from a clinical development perspective that are going to be—we're going to be able to take from the special access program. Treating these patients is fantastic, number one.
Number two, I think it's also going to be—if we can improve the quality of life for these patients, I think what that does for the phase two study is makes it very easy for us to build momentum and to be able to enroll patients. That's absolutely critical.
Thank you for that. That actually kind of answered the next question that came through just on that 78% response rate and how the efficacy profile positions STEMsmart hitting into that phase two for 2026. I'm not sure if you want to just elaborate a little bit more on that.
Yeah. It was a 78% overall response rate for, I think it was 18 patients that were actually part of that, but I think we actually treated more than that. Over 50% of the patients actually went into remission.
They no longer had Crohn's disease clinically, which is a fantastic result. What that does for us is it gives us confidence. It gives us confidence that these cells work. It's a patented—the manufacturing technology is patented, right? We know how to manufacture these cells. Why we think this product is kind of the next evolution of MSCs is the fact that these stem cells are activated prior to prior preservation, right, and prior to treatment in patients. That activation as the last step in the manufacturing process, which is what we have patented, actually provides a differentiated product than what Mesoblast and some of the other products that are out in the market or in clinical trials right now. That's what we see as being the driving clinical impact.
The fact that we know that these cells work now is really—and have proven this in prior clinical trials—is really helping us with now focusing on how do we design our phase two trial such that we give it the best chance for success, right? We look at what the patient population is, what the inclusion/exclusion criteria are so that we are identifying the right patients and enrolling the right patients and making sure that we give those patients the best chance for success with the STEMsmart technology. What that does is then if we get the—if we can recapitulate those results in an international phase two, including U.S. patients, what that does is it makes it very palatable for partnering for phase three and commercialization, right? I think starts to smooth that pathway, especially since commercialization of this type of product has already happened, right?
Especially since Mesoblast already has received their marketing authorization. I think what that does now is it gives confidence, not just that, number one, these cells work, but number two, that there is a viable commercialization pathway and that the patient population and the indication that we're targeting is one that I think is really important.
Wonderful. Thank you for that, Nathan. Just moving on, and again, you touched on this one. The manufacturing tech transfer to Qiagen is underway. How does this partnership accelerate your path to phase two and future commercial supply?
Yeah. I mean, I think what it does is it not only does it provide scale, so it's much—we're able to manufacture more of our product and be able to supply the volumes that are going to be needed, not just for the phase two, but for later phase clinical trials as well.
On top of that, what it does is because Qiagen has such a strong reputation in the industry and has been working with the TGA and internationally, I think what that does is that gives us—it de-risks the manufacturing from our perspective, right? I think once we transfer—we're in the process of transferring that technology, the manufacturing technology to Qiagen. Once that happens, that enables us—we know that that part is secure. We know the supply chain's managed. We know that we have the regulatory piece managed from a product perspective. Now it allows us to focus on the clinical trial, right? We know that the product's going to be there. Now it's about enrolling patients. Now it's about getting the sites up and running and making sure that the protocol is designed well, right?
That partnership really helps us and de-risks and helps mitigate any risk we have from the supply chain side. Obviously, we have to continue to manage and work closely with Qiagen as a partnership. Once we have that in place, that really helps us now take that off of kind of the huge radar screen that we have and now really allows us to focus on the clinical trials and the clinical trial and the special access program, which is what I think bringing on our advisory board is really critical for, right? It helps us focus on the next clinical trials, the next clinical trial for Crohn's, but then also what other indications are we looking at to be able to access all those markets for the different indications that we're looking at.
Thank you, Nathan.
Again, you covered this in the presentation, but we've obviously got this world-class clinical and scientific advisory board. The question is, so how does this strengthen your clinical strategy and de-risk development?
What it does is provide—the great thing about the advisory board, these are clinicians that have been there, right? They've done clinical trials across the board for the different areas of expertise that they bring as key opinion leaders in the market. What that does is that, again, it gives us the best chance for success, right? It helps us ensure that the protocol that we're putting together as we develop our phase two clinical trial, that it's taking into account a number of things that they have seen and have proven. It'll be battle-tested, right?
I think that's really important and gives us the best chance to make sure that we got the right patient population, the right dosing schedule, all that, so that we've got the best chance for success, the best chance to—if the cells do what we expect, right? We've got the right patients and the right dosing schedule, then it can demonstrate an even greater impact to patients in need. I think make it much more appealing down the line as a commercial target and as a regulatory target, right? It makes it easier for us to get to access the marketing authorization both in Australia and the United States.
Thank you, Nathan. Jumping around here. This one's on the renal transplant data. Just saying how significant this is for the platform beyond Crohn's disease.
Perhaps you want to just give some background on what that data showed and then, yeah, why it is significant.
Yeah. This was a special access program that we did in the past, which gave us some fantastic data. I think having Dr. Ashley Irish on the team is fantastic. He was instrumental in putting that data together and reporting that out. What it does is it gives us—it demonstrates to us that we have some really strong targets beyond Crohn's disease that we can access. We know that these are, again, our patients in real need. I think what it does is it sets the stage for us. Obviously, we're targeting Crohn's first, but it sets the stage for the next indication that we have in line.
Again, these are patients with high to severe inflammation, patients in need, and being able to work with that, as the data shows from our earlier special access program, will really help us as we progress that forward and as we develop that clinical program moving forward.
Thank you. Quite a few coming through. Just bear with me. This is about STEMsmart addressing multi-billion-dollar markets. How do we plan to prioritize expansion into kidneys, lung, everything else that we are sort of targeting there?
I mean, look, from my perspective, I want to make sure that we are smart and strategic in how we progress things. Right now, the main target is going to be Crohn's and getting that phase two clinical trial of Crohn's.
We think that's closest to market, and we'll access a fantastic population there that's going to need this type of product. I think the strategy for the indications after, be that lung disease, be that kidney transplantation, I think is going to be—that's where we're going to be working very closely with our advisory team, right, the Clinical and Scientific Advisory Board, in looking at those indications and along with, obviously, looking at the overall markets, what does the competitive landscape look like, being able to put together a really strong business case as to what makes sense moving forward for the next indication. That is really kind of our strategy moving forward.
Again, this is a new asset, and we're working through the data that we have and looking at all the data along with where the market's at clinically and with products that are out on the market right now to make sure that we make the right—but I do want to focus on one at a time. We want to get the phase two for Crohn's disease up and running while we look at the other indications and do the work we need to do so that we can start developing the clinical program for the next indication once we have the phase two for Crohn's up and running. I just want to be strategic about it, right? Yes, there is—I think there's something to be said about kind of multiple shots on goal.
I think from our side, small organization, we want to make sure that we have all of our energies focused in the right directions, but we also want to make sure that we have our eyes on the next program that's going to take place.
Yeah, absolutely. Look, I think that there are quite a few double-ups in there, so that covers everything from the questions. I mean, the company holds just shy of $7 million in cash, 18 quarters of runway, obviously in a great position. What should shareholders be looking forward to from a news flow perspective now and for the next sort of three to six months?
Yeah. We've got—and thank you for highlighting that. I think we've got the cash in hand to take us through to the initiation of the phase two clinical trial, right? That includes tech transfer.
That includes all the clinical startup activities with the CRO, including filing of the IND and the regulatory piece on that side, right? So we've got the cash in hand to take us through. Obviously, we'll need to—for the phase two, we're actually probably going to be looking at a capital raise sometime in the first half of next year, Q1 or mid-Q1 or late Q1 to early Q2. That is something that we're still kind of starting to plan there as we progress. From a news flow perspective, it's really going to be, like I mentioned earlier, right, the results from the first four patients of the special access program. We will make decisions on regulatory partners and CRO, clinical research partners.
In that meantime, the manufacturing will continue to progress, and we'll probably announce some things from the manufacturing side to show that it's on target for delivery for the phase two clinical trial. Obviously, by mid-year next year, we'll have the results of all of the patients for the special access program. Also, at that point, the clinical development will be there. We'll have the pre-IND meeting probably towards the end of Q1 next year. There are a large number of pieces that are still in play and a lot of activity that is still to come, even though, again, we just acquired this asset in June of this year. There has been a lot of activity so far, but we're continuing to drive the strategy forward.
Wonderful. Nathan, I think that covers everything.
I want to thank you all for joining us today. If we have missed any of your questions, please feel free to reach out via the contact details on our ASX releases, and we look forward to hosting you again next time.
Great. Thanks, guys.