Good morning, everyone, and welcome to the NeuroScientific Biopharmaceuticals investor webinar. My name's Abbey Phillipps, investor and media relations manager. For those of you who are new to the company, NeuroScientific Biopharmaceuticals, ASX code NSB, is an innovative biotechnology company dedicated to developing novel technologies targeted at immune-mediated inflammatory diseases. NSB has been developing solutions to address complex medical needs where current treatment options are limited or ineffective. The company recently announced four out of five patients achieved a successful clinical response from patients treated with the StemSmart, under the Special Access Program. The format for today will include a presentation from the company, followed by Q&A. If you'd like to submit a question at any time during the presentation, please use the Q&A function at the bottom of your screen.
Presenting for the company today is Nathan Smith, CEO of NeuroScientific Biopharmaceuticals, as well as Dr. Marian Sturm, who is a Chief Scientific Officer, who pioneered the development of the StemSmart technology, who will join for Q&A. Nathan, I'll hand it over to you.
Great. Thanks, Abbey. As Abbey mentioned, my name's Nathan Smith. I'm the Chief Executive Officer at NeuroScientific Biopharmaceuticals, and I thank you all for joining us today while I get a chance to provide an update to the program and really talk about the Special Access Program results that Abbey mentioned, which are really encouraging and I think are going to lead to a number of different, fantastic things with the organization. NSB is a clinical stage. We're at phase II clinical stage program with a stem cell product that we are looking to treat for Crohn's disease. Yep. There we go. Just forward-looking statements, disclaimer, company snapshot. This slide deck was done a week or two ago, so it hasn't been updated for the current market shift that's ongoing now.
What I really wanted to focus on initially was living with Crohn's disease and the people who have to live with not just Crohn's disease, but severe Crohn's disease or even fistulizing Crohn's disease, which is an even more debilitating form of the disease. If any of you know somebody with Crohn's disease or irritable bowel disease, ulcerative colitis, things like that, you know how difficult their quality of life is when it comes to just everyday interactions, be it going to the store. If you're traveling anywhere outside of the house, a person with severe Crohn's disease needs to know where every bathroom is on their way to and from their destination. The sudden flare-ups can occur with extreme pain and nausea. The dietary constraints are unbelievable. I mean, my wife doesn't have Crohn's disease.
She has a gluten intolerance, just the dietary complications that she has there, let alone people with severe Crohn's disease and how they have to monitor their diet. People with fistulizing Crohn's disease, they have to wear a diaper, an adult diaper because of the complications of their disease. The social isolation and the anxiety, right, the inability to lead a normal life. These are the people that are dealing with severe Crohn's disease and are looking for solutions. Because of that, there is a significant market that has unmet medical need, right? The Crohn's market is over $13 billion USD globally, with over six to eight million patients worldwide. Of those patients, around 30% of those patients do not respond to the current standard of care treatment, because the current standard of care is antibiotics, anti-inflammatory drugs, steroids.
If it gets to past a certain point, then they actually move into the big blockbuster biologic drugs like HUMIRA, ENBREL, or STELARA, to treat their disease. Again, 30% of those patients don't respond to that or relapse from treatment with those biologic drugs. If those drugs aren't effective, if it continues to get worse and worse, then the next solution is actually surgery, where they remove actually part of the intestine and all the complications that come with that. Where our product looks to, I think, meet or provide a solution is in that refractory phase and that refractory stage, post biologics before you actually get into surgery, and there needs to be a solution for that for these severe Crohn's patients. A subset of refractory Crohn's patients start to form what they call fistulas.
These are where the inflammation gets so bad in the intestinal tract that there actually starts to form abscesses, and those abscesses start to become tunnels that lead from the intestine out to the skin, the external skin. Because of that, these patients that have fistulizing Crohn's actually can have fecal discharge that comes from their lower intestine, and their bowel to the outside. Which, again, makes their quality of life even more difficult. Out of the total population, we're probably looking at a subset of probably about 10% of the population, or even smaller, that has fistulizing Crohn's. Because it's such a debilitating disease that has no treatment option at this moment in time. What is NSB's approach, right? For the Q&A session, we'll have our Chief Scientific Officer, Dr. Marian Sturm, available to take questions as well.
She is the inventor of the StemSmart technology. During her time at the Royal Perth Hospital, the StemSmart product that she developed was used in a number of different clinical trials and Special Access Programs under compassionate use, really focused on how can we treat the patient to ensure they get a better quality of life. What that ended up in was we had a previous clinical trial where we had a phase II clinical trial on Crohn's disease, where we saw some very strong clinical results, which I'll get into. That was kind of the basis for NSB's acquisition of StemSmart and the company Isopogen in June of last year.
Once that acquisition occurred, we had some product available that was going to expire, and we actually said, "Instead of discarding that product, let's see what this product does in patients with fistulizing Crohn's disease." That's where we opened up the Special Access Program towards the back half of last year. Those are the results that we announced a couple of weeks ago. That I'll get into details about today. Not only did we get a positive result in four or five patients, but we also got a lot of the data, and that's what I want to talk a little bit about, is how promising that data is. All of that's leading up to a planned phase II clinical trial that we plan to open at the end of this year. I'll talk about our clinical strategy as well. What is StemSmart?
StemSmart is a mesenchymal stem cell therapy. If you've ever heard of a company called Mesoblast or Cynata, they also have an MSC product, a stem cell product, that's very similar to our own. There was actually a write-up last week by The Prenzler Group that talked about Mesoblast, Cynata, and NSB when it comes to the stem cell market and what each of our companies are trying to achieve. StemSmart, the product that was developed by Marian, it separates itself in I think a very key feature. The fact is that the manufacturing process for StemSmart results in an activated stem cell that's very different than, say, what Mesoblast has or what Cynata has. That activated stem cell is actually what we think drives such strong clinical results and such great clinical effect in patients with Crohn's disease.
Just as background, these stem cells, mesenchymal stem cells, MSCs, they really hone in on inflammation. We give our cells to patients through intravenous delivery, so it's not a localized injection or anything like that. We feel like it's a systemic solution. When the stem cells get into the bloodstream, they know where to go because they're homing into the areas with severe inflammation. Obviously for Crohn's disease patients, these cells would hone into the gastrointestinal tract. This also allows it to be a platform technology, where there are other indications that have severe inflammation. A lot of times what we're looking at is severe inflammation that's caused by your body's immune reaction, right?
Crohn's is actually an autoimmune disease where your body's actually attacking itself, and that's causing the inflammation where the MSCs go to treat and actually allow the body to heal itself. Because of that, because the mechanism of action of these cells is very different than, say, the biologic drugs, where they're going after just a specific marker of inflammation. It's actually a more holistic approach to driving down inflammation and allowing your body to heal itself. That's why it's so effective in autoimmune diseases. These cells, like I mentioned before, have been used in a number of different clinical studies while Marian was at the Royal Perth Hospital.
We have tons of safety data. The fact that it does not need to be HLA type, it will not cause a graft-versus-host disease reaction within the body, won't be rejected by the body. That's a great thing, because what it allows us to do is to make a lot of these cells and then be able to provide them off the shelf to patients from one single donor of bone marrow. That's why we think it's a strong business model. This product has been manufactured to really strong regulatory standards over the years. It's held a TGA license for probably about 10 plus years. Marian can tell me the correct time. It's manufactured with very high quality, meets regulatory standards, not just here in Australia, but around the world.
Like I mentioned before, Marian's development at the Royal Perth Hospital led to a phase II clinical trial that occurred about 10 years ago. I think it was around 2016 to 2018 where there was a phase II clinical trial that was opened across Australia that treated patients that were resistant to biologic drugs, that had already failed treatment by these blockbuster biologic drugs. These patients then were treated with StemSmart because it was either they get treated with StemSmart or they go to surgery. In treating these patients, what we actually saw was we saw a 78% clinical response rate. The StemSmart product had a very strong clinical effect in a majority of the patients that were treated. 44 of those patients actually went into remission, so that they no longer exhibited the symptoms of Crohn's disease, even under endoscopy.
They went in with a scope. Within the gastrointestinal tract, they no longer saw evidence of the severe inflammation that signifies Crohn's disease. That was a very positive and very strong phase II clinical result there. What the plan was at the time was Marian had her own company called Isopogen, and was planning to actually move that into larger phase clinical trials and was going to open it up to the market. As she was doing that, the Government of Western Australia went after the StemSmart patent. I think that's all very public. That's happened, and she won, which was absolutely fantastic, and it's free and clear. That was what led to NSB's acquisition of Isopogen and StemSmart in June of last year.
Now, the data and the clinical programs that were supposed to be driven by Marian about a number of years ago are now being driven through NSB for the phase II program. I mentioned this is for patients with what they call refractory Crohn's, right? Patients that have failed biologic drugs, and they still have this severe Crohn's disease. I did talk about fistulizing Crohn's previously. That's where the Crohn's got so bad that you actually form tunnels to your exterior skin. With the acquisition of StemSmart last year by NSB, we opened up a Special Access Program where we treated patients with fistulizing Crohn's disease with this drug. Fistulizing Crohn's disease is a much more disease-resistant form of the disease, just because there are these physical tunnels that are going.
It's not just severe inflammation, but it's actually severe inflammation plus a physical change to the body here. We thought this might be a good place, and because that physical change is due to severe inflammation, we still think that StemSmart can have a significant effect for patients with fistulizing Crohn's disease. The idea was when we opened up this Special Access Program, the intent was to treat up to 12 patients, with performance shares being triggered if we were able to reach a clinical result, or a clinical effect in at least four of those 12 patients that we were planning to treat. However, due to the expiration of the product and the difficulty in recruiting some of these patients across Australia, we were only able to treat five patients by the early part of this year, and when a bunch of the product expired.
However, we still met that four-patient threshold in order to trigger the issuing of performance shares, which occurred right after we announced this a couple of weeks ago. It was definitely a fantastic result. I know it's not a large number of patients, but the data that we got from these five patients was actually extremely promising. Of those four of five patients, in order to meet the threshold of a clinical response, the patients had to demonstrate either a reduction of at least 50% of their discharge from their fistula, or at least 50% of their fistulas had to close. What we found out during the study was that we were taking MRIs and doing imaging to see closure of the fistulas at around week 10 to 12. That was too early to actually show a complete closure of the fistulas.
What we did see, we did see very positive indicators towards healing and towards closure of fistulas. Those four of the five patients, their discharge reduction was significant, and significant enough for it to meet the threshold of a clinical response. The fifth patient also responded, that patient just didn't meet the threshold. What we were seeing was that the discharge was reducing because we were starting to see some healing of the fistulas. The biggest indicator that we saw for the healing of these fistulas is the fact that when a patient with fistulizing Crohn's gets treated, a lot of times they have this surgical implant that's put into the fistulas that keeps the fistula open in order to continue to allow it to drain. Right?
Because if it doesn't continue draining, then it can get caught up in there and actually form an abscess and go septic. Right? Which these complications can be really difficult and almost life-threatening. They put these implants called Setons into the fistulas. Well, what we found out was during the treatment with StemSmart, all five of these patients that were treated had their Setons removed. The only reason that the clinicians would remove those Setons is because the reduction in discharge was so significant, they did not feel that they needed to keep those fistula open. That was, from my perspective, a huge indicator of healing for these patients. I think we had such great results.
The clinician that we were working with, a woman named Dr. Lena Thin, out of the Fiona Stanley Hospital, I think saw the promise of this product and the positive effect it had on these fistulizing Crohn's patients that don't have really any treatment options. Because we had such a strong result, we are actually planning to open two phase II clinical trials in parallel. There was always the intent to open up the refractory Crohn's disease clinical trial, based on the earlier phase II data that we saw from about 10 years ago. We saw a 78% response rate in patients with refractory Crohn's disease. Again, these were patients, refractory Crohn's patients are patients that have failed biologic drugs.
However, fistulizing Crohn's disease is such a debilitating disease, and it has so few treatment options, and these patients don't respond to current treatments pretty much at all. That in our discussions with the TGA, there is the potential that we could use data from a small phase II clinical trial in fistulizing Crohn's patients to be able to potentially support marketing authorization in the future. The intent now is to run a small fistulizing Crohn's disease clinical trial that's open-label in Australia only to potentially, again, if we get a strong response from patients to potentially support an expedited marketing authorization in Australia, which is fantastic, right? Again, with it being open-label, that means that we can report data throughout the clinical trial.
Unlike the refractory Crohn's disease clinical trial, the one on the right, where that one is going to be a fully double-blinded, placebo-controlled, closed, two-arm clinical trial where we've got the treatment with StemSmart and patients being treated by placebo. That one's going to be under a U.S. IND, so we're actually going to open that trial in both the United States and Australia, and that's going to be a much larger study. That's treating a much larger patient population. Because that program and that clinical trial is going to be randomized, we're not going to be able to report the results out until the end of the clinical trial.
Having the two clinical trials going in parallel allows us to, number 1, fast-track StemSmart potentially for fistulizing Crohn's patients, to potentially get marketing authorization, but also provides us data to be able to continue to update the market while the refractory Crohn's disease clinical trial is moving in the background. The refractory Crohn's disease market is the big one. That's the 30% of $13 billion USD globally, right? That's really where we see the market driver to commercialization success down the line. Fistulizing Crohn's disease is a severe unmet medical need. I think we're looking into whether or not we can get orphan drug status or anything like that as we move forward with the fistulizing Crohn's disease study. Okay. The intent is to open both of those studies by the end of the year.
The reason why we can't open them earlier is that we're actually in the process of transferring the manufacturing of StemSmart to a large commercial CDMO, which is QGen. It's based in Brisbane. That's going well, and we're in engineering runs right now, which you see at the bottom. There's the tech transfer to QGen Cell Therapeutics. The engineering run and demo batches are ongoing right now with the intent of having the TGA come in to do their regulatory audit for licensure in September. That's really exciting. Once that's done, the plan and the intent is to have our license from the TGA and clinical product ready by the end of the year, so that we can open up both of the trials, the fistulizing Crohn's and refractory Crohn's studies.
In switching gears a little bit, the IP position is from our. We've gone, I know a lot of people have come to us and said, "Oh, Mesoblast has a ton of patents around MSC technology and things like that." We've looked very long at it and made sure that we are playing in a space that's open. The patent that we hold and that Marian was able to patent for the manufacturing process is for the cell culture method for the StemSmart technology. Right now, that patent goes out to 2034, obviously, there is the potential for us to apply for extension. On top of that, we're actually looking at further developing the manufacturing process. Any further developments there, again, obviously will broaden our IP.
We've also got some provisional patents that are in the works right now off of the great Special Access Program and some of the other things that we've been doing. Even more importantly is the intent is to get marketing authorization, not just in Australia, but in the U.S. for StemSmart to treat refractory Crohn's and Fistulizing Crohn's disease. Once we can gain marketing authorization, we have a 12-year exclusivity period within the U.S. That exclusivity period is different across the different regions, but the U.S. is the big market. That 12-year exclusivity period is fantastic once we get there. Moving on to key milestones.
Obviously, since the acquisition mid last year, almost a year ago now, there's been a lot of work that's been ongoing, including the Special Access Program, the tech transfer, getting everything started for the clinical programs, things like that. We're continuing to chug along in the background with all of the different operational pieces. We have a pre-IND meeting with the FDA coming up for the refractory Crohn's study. We are applying for that right now and with the intent, hopefully, to have that meeting towards the late July. Again, that will help inform the clinical design of the U.S., the refractory Crohn's study. Obviously, any learnings that we have there will obviously apply to the fistulizing Crohn's study as well. We have the TGA coming in for their audit in September of this year with the licensure to follow.
Regulatory submissions to happen in parallel. Those are regulatory submissions with the FDA for the IND, and with the TGA as well. Plan to commence the trial towards the end of this year. As we get further and further through the trial, we'll be talking with partnering and potential partners on advancing both the fistulizing Crohn's and the refractory Crohn's programs. Again, the refractory Crohn's program will be a phase III study for its global marketing authorization. Looking for a partner for that commercialization and further clinical development is I think from our perspective, the best pathway for commercialization. We'll be working on those partnering discussions. We've already had a couple discussions, and we're continuing to be open to those discussions as well.
Looking at, again, we see StemSmart as a platform technology, we got a couple of candidates of the next indication that we will target and go after. We'll look at potential expansion into those other indications, what those clinical programs look like and further development. That's once we have the Crohn's studies up and running, we'll be looking at those other indications as well. Prepping for phase III studies and commercialization down the line. The team that we put together is a small, but I think a very talented and close-knit team, with obviously myself and I've got tons of experience in both the cell therapy and the biologic space. Dr. Marian Sturm, who's the inventor of the technology, and will be here for the Q&A shortly. Professor Kathy Cole, who's our Chief Medical Officer.
I want to take note, we've got our board of directors, and we've also got our clinical and scientific advisory board. All of the members of the clinical and scientific advisory board, along with our Chief Medical Officer, have actually used these cells in patients. I think it speaks to Marian and the StemSmart product in that these clinicians have used this product and they've come back and they have come on board to help guide us when it comes to how we move forward from a clinical and scientific perspective. I think they're a huge help in advising us as we move forward towards later stage clinical trials and other indications as well. Just to summarize, we've got strong leadership team with tons of experience to take us through the commercialization process.
The StemSmart technology platform is our IP is strong and the manufacturing is rock solid. We've got some very strong data within patients, which I think, I hear a lot about the hesitancy in investing in kind of pre-phase II clinical programs. I think some of that, I think our history and the experience that we've had to this point and what StemSmart has been through, de-risks that consideration. I think we've got proven clinical efficacy already in Crohn's disease. What we're showing now is actually how it compares to placebo and taking it further and getting a stronger data package for commercialization. I think the experience that we have, the safety of the product, the number of clinical trials and patients that we put in this product in, de-risks our program from your normal pre-phase II clinical program. We've got a commercialization pathway, right?
I didn't really talk much about it, Mesoblast getting the marketing authorization in the United States for their program, I think was a huge achievement, huge win. Kudos to Silviu and the Mesoblast team there. What it does is also paves that pathway for us. I think that's something that we can take those learnings and try to build upon them, right? Kind of follow their example. I'll leave it at that and open up the floor for Q&A. I appreciate you taking the time to join us today and helping us while I take you through the NSB program and the updates to the program. Thanks.
Thanks so much, Nathan, and thank you for the presentation. We do have a few questions coming through here. Marian, I do know you're on the call as well. We were just really hoping to break down how StemSmart works, if we could. Marian, I think you could be the person for this question.
I'll take it, I guess, until she's on. Oh, there she goes.
No. Hello. Yeah, sorry. I'm technologically challenged sometimes.
Yeah, these cells are remarkable things. Sometimes I can't believe that they work as well as they do, but we've seen great clinical outcomes for patients. Basically what they do is they home to inflammatory sites. That's what guides them to where they need to act. When they get there, the inflammation or the inflammatory markers are released by immune cells. They interact with the immune cells in a very complex manner. Some of the interaction is cell-to-cell contact, and some of it is the MSCs secrete all kinds of effectors, anti-inflammatory factors, to change the behavior of the immune cells. They basically inhibit the proliferation, activations, and the secretions of T and B cells and dendritic cells, NK cells, neutrophils, and macrophages. Then they support the generation of cells called Tregs, and these are the cells that are responsible for inducing immune tolerance.
In terms of the gut, they have a role in tissue repair as well, because they are actually a stem cell. In the lab, we can demonstrate this because we can turn them into bone, cartilage, and fat. They're known to induce the proliferation of intestinal epithelial cells and the intestinal stem cells, and this helps restore the intestinal barrier integrity. Basically repair the gut. Yeah, they're very complex immune actions.
Oh, sorry. Were you finished?
Yeah, no. Go ahead.
How safe is the product, what side effects are associated with it, and how does it compare to other treatments?
It's very safe. Worldwide, there's thousands of patients that have been treated with different types of MSCs. Ours are a bone marrow derived MSC, and they have probably the strongest safety profile with very few side effects. In our studies, we've treated over 200 patients in probably around about 1,000 infusion episodes across all kinds of immune inflammatory conditions and seen no serious adverse events attributed to the cells. Remember, the patients that have had these cells have been incredibly sick. The most common adverse event that you see, or is related to the solution that's put in the cells to cryopreserve them, it's called dimethyl sulfoxide. The patient might complain of a bad taste or a smell. It resolves within 24 or 48 hours. It's very mild.
It's the same cryoprotectant that we put in the products that we give to patients that undergo bone marrow transplant. Only the amount that's in an MSC product is much, much smaller than a bone marrow transplant patient would get. Occasionally, the patients have complained of a headache or a cough, that resolves very quickly, too. The headache can be related to the fact that the cells are often given a little bit cold, and that's what gives the patient the headache. The cells are generally well-tolerated. They're given as a small infusion through a peripheral vein, and the product's usually between 40 and 80 mils. The cells are given per kilo patient weight. A 100-kilo patient would receive effectively 80 mils of product. If you think a glass of wine is 120 mils, 80 mils is a very small amount.
It's given to the patients, they're usually in a day ward, sitting in a chair, and a nurse administers the cells over 15 minutes to a half an hour. Very easy to give.
Have MSC products been used in other groups for treatment in Crohn's disease or fistulizing Crohn's disease?
They have indeed. There's been very limited studies in what they call luminal disease, which is non-fistulizing Crohn's, and only very few patients have been treated. Most of the work in Crohn's disease has actually been in fistulizing disease, and there's been lots of study in fistulizing disease. All these studies, all of the work in fistulizing disease other than ours, actually injects the cells into the gut wall or the fistula tract. It involves a surgical procedure. They have seen some reasonable results with those cells. There was a commercial product, a fat-derived MSC, that actually came onto the market. It's subsequently been removed because the effects haven't been longstanding. As I said, our cells are different or our product's different because it's administered systemically, into a vein, so it doesn't involve a surgical procedure. It's much easy to give.
We're still working out what the therapy regime will look like, and the fistulizing study has given us some real guidelines to that, but it's quite easy when the patient needs a top-up of cells just to come in and get another infusion. Whereas if you're having to go to surgery, it's much more complex thing to do.
Has StemSmart been shown to be efficacious in other clinical disorders?
Yes. Why I developed this product was for patients with severe graft-versus-host disease. I've sort of got a background in hematology. I used to look after all the bone marrow grafts for the state, both pediatric and adult. I'm well exposed to severe graft-versus-host disease. The cells were developed for those particular patients because it was just so horrible for patients, clinicians, and their family, the patients' families. The rate of death was quite high from the disease. A lot of the features that you see in graft-versus-host disease, you see in other things. Patients with graft-versus-host disease can get it of the gut. It looks like Crohn's disease. I went from GvHD to Crohn's disease, and patients with GvHD can also get lung involvement, which looks a bit like lung transplant rejection.
We ended up in lung or other inflammatory disorders of lung like a COPD, chronic obstructive pulmonary disease, emphysema. When we sort of got into the solid organ transplant section and the first phase I study looked so good, we ended up in kidney transplants. We did a study in 16 patients that had acute renal rejection and got quite good results. It sort of everything grew from the features of graft-versus-host disease generally. The thing that I've learned over my time, which is over 20 years working with these cells, is that you have to have high levels of inflammation for the cells to work properly.
When we started to do other studies, we thought, well, if they work well when patients are so sick, why don't we try and use the cells when the patients aren't quite as sick? We went into things like as soon as they got GvHD, before they became resistant to standard therapies, would they work there? The answer is no, they didn't work because the levels of inflammation weren't high enough to elicit the good response of the cells. When we went into lung transplant and came in early before they got bad, they didn't work there either. We've learnt that you've got to have high levels of inflammation. They're going to work in any situation or any clinical condition where there's actually high levels of inflammatory markers.
They need inflammation to actually get them fully activated and to bring down immune responses.
Nathan touched on this in the presentation, but how does StemSmart help with closing or healing of fistulas?
Well, all the fistula patients had high levels of inflammation in that fistula. They were active fistulas. The cells home to that fistula, and there they bring the immune response under control, and they secrete anti-inflammatory mediators. One of the ways I know the cells work is they secrete something called TNF-alpha receptor, and one of the major inflammatory mediators is TNF. The receptor basically soaks up that inflammatory mediator, so that disappears. It is the actual inflammatory mediators that do all the damage to the gut. It basically brings that immune response locally under control. It absorbs a lot of those inflammatory mediators, and then it also encourages some of those epithelial cells and the gut stem cells to start the repair process of the gut, of the fistula.
Yeah, I always think of it as it brings the inflammation under control, right? Helps modulate your immune system and let your body heal, right?
Right.
Let your body do its natural healing. The fistula is an unnatural, is not something that you would normally see, and I think the body wants to get back and repair that. That is how I see it from a layman's term, right? Layman's perspective.
Nathan, we do have a question here on partnering options. You mentioned Mesoblast in the presentation. Have you spoken with them?
It's actually funny. I used to work at Mesoblast back in the day, so I know Silviu, Marian has had some definite interactions with Mesoblast and helped support them in their early clinical phase. We definitely have contacts with Mesoblast. At this time, I think we're trying to mature our program a little bit more. We're always open to those conversations, and we'll definitely be reaching out if they don't reach out to us first, right? Yes, they're on our calendar to contact when it comes to partnering. Through my past experience within the cell therapy industry, I've got a ton of contacts in a number of large pharma, BioNTech, J&J, Novartis, Gilead, a number of the large players within the cell and gene therapy space.
That we want to open conversation with I think a number of them as well, especially seeing as how Mesoblast has already paved the pathway for commercialization. Those companies don't have to invest and be the first to do that, right? Which is difficult for them to take on. Now that there's a commercialization pathway available for MSC programs, now it's just making sure that the data is strong enough. That's why we're opening the phase II studies.
How many patients are planned for the clinical trials? What is the total cost of the trials? With that, what is the cost of the treatment?
Sure. Obviously these are still being worked through, but preliminarily, with the small fistulizing Crohn's study, we're looking at probably about 25 patients, 20-25. We're still finalizing that number, but that's what it looks like. Again, that'll be open-label so that we can continue to, once we start to get data from those patients, we can publish that data to the market and keep them abreast of how the program's going. For the larger refractory study, we're looking at somewhere between 60-70 patients, probably around 65-66 patients is what we're looking at. Again, we're still waiting for that to be finalized, and we also have the pre-IND meeting with the FDA to validate the design of our study and everything there. With all of that, we're actually in active discussions with clinical research organizations.
These are the groups that run clinical trials and do that for all types of clinical trials all over the world. We're working through that budget, but the budget that we're looking at for both of those studies is somewhere between the AUD 15 million-AUD 25 million range, which actually for two phase II clinical trials is not that much. It's actually pretty reasonable. That's where we're looking at from the total cost of those programs, and that'll go out for the lifetime of the clinical trial. Spread that across a number of years as well. That's what we're looking at from the total cost of the actual clinical trial and those programs. The cost of the treatment itself, that's a tough one, right?
Because we've got to look at a number of different factors before we can calculate out what the actual cost of treatment will be. There's obviously the cost of goods, there's the hospital, the cost of the patient care, and follow-up that's going to be required, and that's going to be developed as we move through the clinical phases. What's the price for the product going to be? Well, that's a different question, right? Obviously, I think Mesoblast is charging AUD 1.5 million for their pediatric graft-versus-host disease product. Again, we'd have to take a look at what the actual health economics are going to be for these, what the ideal patient population looks like, and all of those things, to help us understand what the ideal price for this would be.
A big part of that is really going to be understanding what the overall cost of the product, cost of goods, and the cost of treatment is going to be. These are obviously a number of things that we're looking at as we move forward for this program. Right now, I think it's a little too early to say what the actual cost of the program or the cost of the product's going to be, or the price of the product at this point.
With that, thank you, Nathan and Marian, for your time today and for providing such valuable insights into StemSmart and the clinical development pathway. Also, thank you for everyone who joined the webinar today and submitted questions. For any questions that we were unable to get to, please feel free to reach out to the Jane Morgan Management team following the webinar. Thanks again.
Thank you.
Thank you.