Transcription
Welcome to the Molecules and Minerals podcast. I'm Steven Goldman. I'm a former trial lawyer, corporate director, executive, and a biotech investor. I'm here today to follow up with Frank Gleason, CEO, co-founder, and director of Satellos' BioScience, a Toronto-based biotech company advancing its first-in-class novel mode of action investigational drug SAT 3247, which aims to regenerate muscle function in Duchenne's muscular dystrophy and other degenerative muscle diseases and injuries and not merely delay muscle loss.
On February 10th, I had a lengthy podcast interview with Frank in which he discussed the Telos's Lee drug, the science behind the drug, and its very promising early clinical results, which were released last May from a one-month phase 1B clinical trial in five adult Duchenne's patients ages 20 to 27. They were administered SAT 3247 once daily just Monday to Friday for 28 days while remaining on their standard of care cortical steroids. These adult patients showed an average doubling of grip strength and nearly 6% improvement in forced vital capacity lung function. This was very promising albeit early results in a very small number of adult patients. For those interested in a deeper dive, I'll provide a link to my February 10th interview in the notes below.
By way of background and to put today's interview in perspective, currently Satellos has two ongoing clinical trials testing SAT 3247 in the treatment of Duchenne's. The first trial that we'll discuss in more detail is a phase 2 open-label extension trial in adult Duchenne's patients called Trailhead, which last fall reenrolled four of the five adult Duchenne patients which I just discussed and the plans are to enroll up to 10 adult Duchenne patients in Australia and subject to regulatory approval another 20 adult patients in the US and so hopefully by the end of this year there will be 30 adult patients that are being tested for a period of up to 12 months.
More importantly, Satellos has begun a phase 2 double-blind placebo-controlled trial called Basecamp, which plans to enroll 51 ambulatory Duchenne's boys ages 7 to 9 who will be dosed for a period of 3 months with either 60 mg of SAT 3247 per day, 120 mg, or a placebo group with all patients on standard of care cortical steroids. So the first boy was enrolled in the Basecamp trial in early February 2026 and hopefully, Frank will give us an update on that trial. So Satellos hopes to complete enrollment in the global Basecamp trial by Q3 of this year and if things go well, hopefully, we'll have some data by the end of this year. The Trailhead open-label extension trial in adults is expected to have quarterly updates including ongoing clinical readouts in the coming quarters.
Now since my February 10th interview, additional data has been released by Satellos at the 2026 Muscular Dystrophy Association Clinical and Scientific Conference on March 10th and 11th. Some of which appears to have been received negatively by the market. In fact, Satellos's share price has declined by approximately 50% since that March data release. In today's interview, Frank will provide a general update and also discuss that recent clinical data and why Satellos believes the market may have overreacted or misunderstood what was presented.
So, Frank, thank you very much for agreeing to be my guest today. A lot has happened since our last interview. And before we get into what was presented at the MDA conference, can you provide us an update on the global Basecamp trial? What's the interest in that trial and how is enrollment going?
Well, firstly, Stephen, it's nice to be back. Thank you very much for the opportunity to come back and speak with you. I'm glad you're still willing to have a chat with me about Satellos and I'm hoping that today we can maybe help the audience more clearly understand the messages we've tried to convey and how we interpret the data that we have presented and our plan going forward.
So, in answer to your question specifically about Basecamp, this study is going extraordinarily well. Now, what does that mean when I say it's going extraordinarily well? Because what's posted on clinicaltrials.gov, of course, is five sites are initiated. And what we've publicly disclosed by press release is we treated the first patient in February. So, does that mean nothing else is happening? Does that mean we're not doing anything? No, it doesn't mean that at all. What it means is we are not going to report every time another patient comes into the trial to the market. What we have said to the market is that our objective and our current model is to have this study of 51 individuals fully enrolled by the end of Q3. That is already remarkable speed in the Duchenne muscular dystrophy space. Typically these trials enroll over very extended periods of time. We have identified 25 clinical sites across eight countries. All of these sites are engaged with us in the process of initiating the trial. There are multiple steps that go into this at each and every single site. You need to enter into multiple levels of contracts. In some cases, you have to enter into contracts with medical staff. You have to enter into contracts with the hospital administration. You have to go through ethics approvals, none of which can start prior to the FDA or whatever other country's host regulatory organizations sign off on the trials. And these processes can take several months. This is one of the reasons why we had so many sites that we went to because it's a numbers game. You want to have as many sites up and running as fast as possible as you can so that you telescope time. So we have multiple sites up and running. We already have demand well in excess of 51 patients for the 51 spots. In certain cases, we have caps on certain types of patient meaning patients that have been on certain drugs as background before entering our study, so that we don't overload the study statistically one way or the other. In some cases, we've already exceeded our quota, so to speak. At some sites, we have already received demand for multiple patients. We are basically overwhelmed with the level of response. Coming out of the MDA meeting, for example, more doctors became interested in what we're doing. More patients reached out to become involved in the study. So the response we've had from the medical and patient community and I must say also from our institutional investors, these are basically the people that put up the $57 million a couple of months ago has been very positive. They were pleased with the data. They were pleased with what we're seeing in terms of our speed at getting a study up and going. I mean this is actually quite a complicated study because we want to look at multiple variables that will increase the probability of accelerated approval. So, we've built into this study many different features that wouldn't classically be in a phase two and might not be typically involved in such a large study as well. So, we've tried to build as much in. We're doing well. We've got a great team of people. We have wonderful rapport with the Duchenne muscular dystrophy advocacy groups in multiple countries. We have lots of patient interest, lots of physician interest. This will play out over the next few months. This is one of the things about Satellos. We are months away from fully recruiting a study. We are months away from having data in that study. We are not years away. We're not conducting a year-long study that's going to take us two years to recruit. We're conducting a study that's taking us a matter of months to recruit. And you know that of course proof of the pudding of course is in the taste. We are working on this. And you know, largely speaking, I think over multiple years, Satellos has pretty much done what we said we would do, pretty much within the time frame, a little bit here and there sometimes, but overall, pretty high hit rate in terms of promises made, promises kept. I wish the stock price was different. Hurts me, too. But it is what it is right now, and we'll work to get it back to what we think it should be.
So Frank, one of the things that I've seen from your phase 1B data in adults and in the Trailhead study is there appears to be some relationship, albeit this is a very small number, between creatinine levels and or use that as a proxy for muscle and patients who perhaps respond better to the drug.
Sure. So this is something that is interesting. It makes sense to us. Hypothetically, it makes total sense to us for the very simple reason that the stem cells that we are attempting to rehabilitate, I guess you could say, to get them to divide properly, they need to live somewhere. And they live in muscle. They're in muscle. They're a component of muscle. And so if a patient has lost all their muscle, then they won't have very many stem cells for us to act on. So it stands to reason that a patient with more muscle will respond better than a patient with less muscle. It is also important for us to remember that that provides proof of concept because if they all responded equally then you might think, oh well, how do you know your drug is actually having an effect? So you have a differential population and you have a differential response to the same treatment. So it makes total sense to us.
Now what I sometimes feel uncomfortable to talk about is how difficult the lives are for these individuals in their 20s living with Duchenne muscular dystrophy. Their bodies have been ravaged by this disease and nobody really knows how much muscle they have left, how bad their situation is. And many of them, you know, the most simple and basic functions that we not only take for granted, but we would never even think of being an issue for them are hurdles to daily life. So when we're talking about individuals that have, you know, responded or not responded, we're talking about individuals that really, you know, are having a very, very tough day of their lives every day just with the simple things of rolling over, getting out of bed, getting into their wheelchair, taking a shower. For many of these functions, they need assistance. And so to see anything at all biologically, functionally is actually remarkable. Nobody has ever tried to treat these patients with a drug for Duchenne's. They're on many other medicines because they have multiple comorbidities, but these are not medicines designed to restore muscle or increase their functionality. They're muscles, they're drugs that might be cardiac medicines that attempt to deal with the congestive heart failure they're experiencing or steroids that attempt to deal with the massive levels of inflammation in their bodies. But they're not drugs that are designed to try to lessen the consequences of muscular dystrophy itself and help restore some function. So we are pioneers. We are out in front of everybody else. And you know, sometimes it doesn't end well when you're a pioneer. Sometimes it's not, you know, exactly going to deliver what you want.
So what we look at when we look at what we've seen with only, I mean it's only four individuals and it just so happens that two of these individuals by blood work have a greater level of muscle mass than the other two individuals. And what we see is that where there's a change, such as in grip strength or where there's a change in shoulder movement or a change in elbow flexion, consistently the two individuals that have more muscle mass show an improvement that's greater than what is seen in the two with much less muscle mass. What we see is basically a stabilization or no change in the two with little to no muscle mass and an upward improvement in the two with muscle mass. It's only four people and we think now they are all one year older than they were this time last year when we reported on them. They would typically all be expected to decline several percent in that one year, but they didn't. They didn't. The gains that they made last year, they kept. Now, we didn't see another doubling this year of grip strength. And that may just reflect we've seen the limits of the biology that we're attempting to modulate in people with very, very little muscle left. That's what it might mean, but it doesn't mean it's not important for the individuals living with their condition. And importantly, none of them want to leave the trial. They all want to stay in the trial and their physicians want them to stay in the trial.
Now, we did have an anomaly on the FVC and again, possibly we described it the wrong way. We did have an anomaly. As I've described, the individuals with the higher level of muscle mass did better on all of the functional measurements. But in one case, we had an anomalous result in FVC. We saw in three of the individuals, they maintained or improved the gains that they had made before. And we saw in one individual a stark decline in a short period of time since they came back in the study. And there's no evidence that this person has lost the ability to breathe. There's no evidence that this person has been harmed by our drug. We think it's entirely possible they just had a bad day. Maybe they had a cold. Maybe they had a little bit of difficulty with the procedure itself. Maybe they were fatigued. But the results are inconsistent with the positive results on everything else. Sometimes that happens. This just, that's why you try to get clinical trials with a lot more than four people because things like this happen and you know we've made a commitment to ourselves and to the market that the data is what the data is. We report the data and we did and in this case I think it's probably been overinterpreted. So as far as we can tell all four of these individuals are doing as well as can be expected, I guess you could say given their state of affairs and we think they're all quite pleased to be in the program and on the drug.
And in Australia, we will be recruiting additional patients. The hospital site is working on that now. And as you said, Stephen, we are bringing this study to the United States. We expect to have that up and running in Q2. As we have said, that's all on track. We have the sites identified. We're in discussions with the sites, with the physicians at the sites that will lead the study. We have a great deal of interest and we will be lowering the age to 16 for this study. So again, consistent with the idea that more muscle mass has the potential for more response. So therefore our thinking is let's work into this patients that are a little younger but still considered from a clinical point of view in that adult category. And then of course we have Basecamp, which is pediatric or childhood aged study.
When the 1B data had been released, there had been some proteomics data that had also been released. Will you be doing proteomic analysis as well for Trailhead?
Yes, absolutely. And again, what the proteomic data tells us is that the drug is biologically active on muscle. The markers that we're seeing affected are well-known, well-characterized markers of muscle degeneration, muscle regeneration, or muscle energy levels. So all of them are involved in the process of muscle metabolism and this is really, really significant because it does mean our drug is biologically active on muscle and we screened thousands of proteins in this proteomic analysis because that's what proteomics is all about is looking at massive numbers of molecular components of blood and of tissue all at the same time. And what we've reported on are those markers where there's been a dramatic change. So it's not as if everything changed and we picked five and said, "Okay, these are the ones we like." It's of the thousands, these are the ones that changed. That's really significant because it means the drug is precise.
So will you be reporting on ongoing proteomic changes?
Yes. Yes. So now that we know where to look, now that we know the ones to look for, now we have the beginnings of a biomarker panel and we've talked about this in the past as Satellos is working towards a biomarker panel. So this starts to give us now the building blocks for a biomarker blood-based panel and so yes, we will be doing blood draws and doing proteomic analysis and we will build that into all of our studies going forward.
Okay. Okay. Um, so the four patients that were reported in Trailhead, these were the four that were in your original phase 1B study. Yes. And then there had been, I guess a gap, some seven or eight months.
Approximately before they restarted the trial and it looked like they had sort of maintained or perhaps even improved their grip strength in the FVC. Was that your observation?
Yes. Yeah, it is our observation and you know this, I must say in all candor, this is kind of unusual that we did a study, we started a study, then there's a big gap, and then we, same patients go back into a study. It's this isn't what you want to structure from day one. So I'll take a moment to explain what transpired and why we have organized things in this manner. A year ago when we were conducting our phase 1 program, which was a safety and PK program, we had the opportunity because of the relationship we had with the clinical trial site in Australia and the comfort level that the regulators had with the safety of our drug to piggyback a small safety study in patients with DMD onto our safety study with the healthy volunteers. And so that's what led to us being able to treat five patients. We had hoped to get more patients, but that was the limit that we were able to enter into the trial at the time, not because of demand, but because of some of the preconditions of the trial. Not all of the patients qualified for the trial. And we were able to show the drug was safe in patients, which was important to get regulatory approval in other countries. And we also showed that the PK of the drug did not change even though these patients were on steroids, which of course was also important to be able to show because in Basecamp all these kids are on steroids. So we didn't want to get a big surprise, drug-drug interacts with steroids, big problem. That doesn't happen. That's not what's going on. And we were also able, just by the set of circumstances, to look at some functional measurements, but we only had 28 days of toxicology support. In other words, when you conduct a clinical trial, the regulators insist that if you have X number of days of preclinical evidence of safety in various systems, that's the maximum you can start with in your clinical trial. So we had 28 days of tox. That's the standard level. By the way, you build this in increments. You do one month, then you do three months, then you do six months, then you do nine months. And then that combination gives you long-term and you do it in different species and you're told what to do. It's all it's all mapped out. So it's not as if one company does it differently than another company. There's a there's a map you follow because it's all laid out by the regulators. You need to submit certain data in certain increments, so on. So we have all that now, but you can only do a 9-month study over a 9-month period of time. If you're going to do, you know, a toxicology study for 9 months, it takes 9 months, plus it takes a bunch of months to write the reports. So we have all that now. Well, last year we didn't have all that. So we did what we could and then we worked with the regulators in Australia to get the study started again as fast as possible when we had more longer-term toxicology and that's why there was a gap and for some of these individuals that gap was almost 12 months. So they were off drug between seven and 11 months, it turned out the four of them and when they came back, it did look as though they held the gains they made and there's probably sampling variability between a year ago and now that could explain a little bit of difference here and there, but at that time and then again another two months later on drug, they have held the gains. That's what it says to us on grip strength. They've held the gains on grip strength that they made a year ago. Maybe some a little bit up, some a little bit down, but it's four people. It's all within the margin of error. They've held the gains within the margin of error. As I explained on the FVC, there was one anomalous result, not a safety issue of the drug. Doesn't look like it's a health issue of the individual. Likely just sampling problem, bad day, whatever, but it happened. And these are people that are not as well as the rest of us. And sometimes things just go wrong.
Okay. With this anomalous individual, for lack of a better term, but that same person did well on the other measurements.
Yes. Yes. All of them. That's what we were trying to say in our press release. That actually we probably should have had a trial or a review of the press release before.
A great idea. I'm sure to recommend one. Okay. Well, that's terrific. So, I'll just stick to Trailhead for a moment with your adults. So, this first group, these four patients, because they were already in that phase 1B trial, this extension trial is only another 11 months for them, whereas the new patients will be a full 12 months.
Um, so we had kind of a stub period for them, if I may use that term. Exactly. Right. Initial 28 days and then this was only 56 days and now they start back into a full, I'll call it full quarter, full three months. Is that your understanding?
That's it. Exactly. And and all of this, by the way, I get it. All this is difficult to kind of get your head around, right? This is, yeah, it's tricky, but there are no games here. It's just it is just the way it played out because of a set of circumstances and we made a commitment to the four, well, it was five, the four individuals that if they participated in the 28 days when we got the thing up and running again, they could come back in.
Perfect. So just by mathematics, since you presented this data March 10th, I'm assuming another 3 months takes you into the end of May approximately. So, uh, next quarter, we could hear from you in end of May or June. Is that the time frame?
Yeah. So we're doing more, we're doing additional work at the end of the 6 months cumulative 6 months of treatment, which includes MRI and we've added other physical measurements. So it may take a little bit longer to collect all the data, analyze all the data, put it all together. So sometime in the middle of the year timeframe, we'll update the market on where we are.
Terrific. And and we'll have at that time, because of what you're doing, you'll have what I'll call more objective data, the MRI data, proteomics, etc.
Right.
Okay. Um, that's how we look at it. It's not subjective.
Yes. And then with the enrollment of new patients in Australia, how is that coming? I know you can enroll another up to six more patients under your protocol.
Yeah. So we will enroll as many as we can up to the 10. Um, and you know, I think more importantly is getting the study launched in the US and to start with younger patients of age 16 and up. Um, you know, it's just a much larger country, so there's a lot more potential patients.
So, as I understand it, you're you're hoping to file your IND for that trial. Is it this quarter or this month or?
This month.
Okay. Okay. Um, great. And, uh, your what's your best guesstimate as to when you might start enrolling for that, assuming that you get approval in the US?
We'd like to have the trial up and running, as I said, in June. I'm careful now about the use of the word enroll because there are like three steps before a patient gets on drug. They have to be registered to sign and then they sign a consent form, then they get screened, then they get evaluated and then they get dosed. So, you've got multiple steps before they're dosed. But to me, once they're in that process, they're enrolled. But that's not apparently technically accurate. So yes, sometimes at the middle of the year, we'll have the study up and running and we'll get patients on drug in Q3.
For that, at least the first patient, once they're enrolled or or dosed, will that be something that Satellos would press release?
I think in the US that we will press release when that first patient is enrolled in Trailhead in the US. Yes.
That's a great summary and you've talked about Basecamp for the children, a very important trial and it sounds like you're on track to, if all goes well, to be enrolled by still by Q3.
Yeah.
All right. And are you still hoping to have topline results by the end of this year?
That's our goal. We want to have this study and we mean we want to know as well. So we want to know as soon as we can.
Sure. You know, so that's our goal and we believe we're on track for that goal.
Okay. So the other data that was presented, because you presented a number of posters at the MDA conference last month, was on in FSHD, there was some mouse data.
Yep. Perhaps you could briefly talk about that and then perhaps tell us where you are about filing for approval to begin a phase 2 trial with SAT 3247 in FSHD.
So FSHD is another form of muscular dystrophy. A different gene is mutated in FSHD than in Duchenne muscular dystrophy. It's a gene called DUX4. It's not dystrophin, it's DUX4. It's a disease that affects primarily the upper body. So F is facial, scapulo, the shoulders, humeral, the upper arms, FSHD. And it tends to be a later onset disease. Not in all cases, but it tends to be a later onset disease. FSHD has been on our radar for at least two years. We started doing preclinical work a couple of years ago. We had promising early results in an animal model of FSHD. We didn't have the bandwidth a couple of years ago to go any further than that because our focus was DMD and getting that right. And now that we have interest in working with us on FSHD from other parties, we have further conducted preclinical studies again showing the potential of this drug to have an effect in FSHD and we've learned more about the nature of the biology of FSHD. So there does appear to be a regenerative deficit in FSHD. It does appear to be kind of a side effect of the particular mutation that occurs in that disease that affects the stem cells differently than in DMD, but in a way that we think we can rescue. And so our plan before the end of Q2 is to submit a protocol to the FDA to conduct a trial in FSHD. And so we would want to get that trial started in the second half of 2026 and begin that process. I imagine you might ask, so in terms of the scale of the trial and so on, roughly similar numbers of patients to what we're doing in Basecamp. Likely something on the order of a 3-month treatment period as we're doing in Basecamp. These are details we're working out with our clinical advisors. But we're working on the protocol now.
Great. Would that be a global trial as well or just in the US?
We haven't made that decision yet. We will take that a little bit step by step because our primary focus is executing on Basecamp and Trailhead and not getting anything in the way of that. So at least the US and Canada as well in this particular case. And possibly more, but first those two jurisdictions.
And Frank, when you say filing a protocol, is that an IND that you'll be filing in the US?
Yeah. Yeah. So yeah, so you develop a clinical protocol. It's sort of the code for this is how the trial is going to be conducted. So the docs can take that and at their site, they follow it. It's sort of the recipe book.
Perfect. Perfect. One of the other poster presentations that Satellos made at the MDA last month was a regenerative index and perhaps you could talk a little bit about what that is and why that may be important.
Well, this is really, I mean, to me, especially, I find this just super cool. So, you know, we have been focused on DMD for, you know, 8 years now since we started the company and until we conducted the preclinical work in the canine model. It's been very difficult for us to see right into the biology exactly what is going on in the muscle because when you're dealing in mice, they're really, really tiny. And so you're looking at really, really, really tiny slices of muscle under a microscope trying to figure out exactly what's changing and when. But when we got into a larger animal system with larger sections of muscle that we could look at and we could then use different types of tools to actually see what was going on and we could see the elements of the immune system that were acting into muscle, how they were destroying the muscle, how they were acting on it and basically causing muscle to disappear. It gave us this idea that wow, we can actually get markers for muscle, but more importantly, we can get markers for destruction. So, you know, the muscle is actually literally being consumed by elements of the immune system because the immune system thinks, and is correct in thinking, this muscle is dying and therefore the job of the muscle, of the immune system, is to get rid of the dying tissue to create room for new tissue to grow. That's how we recover from serious injury. That's how we get rid of infectious organisms is the immune system comes in and it basically helps clean out the system. And in the case of DMD, the system's not coming back. The immune system is doing its job. Maybe it goes into overdrive a little bit, but it's doing its job. It's the regeneration that's not keeping up. That's why these, that's why people with DMD lose muscle mass. It's literally disappearing. And so that gave us the idea for an index, a ratio between basically the immune system eating, consuming the damaged tissue and the new tissue coming back. And so then we took that idea which we first reduced to practice in the canine samples of muscle and then we started a relationship with one of the hospitals in the US that has basically a reservoir of human DMD samples. And we were able then to work with human DMD samples of different aged individuals that had been curated and take the same approach and look at at different ages and at different time points. What is the ratio of degeneration to regeneration? And that's how we came up with the regenerative index. So it's an index of the immune system on the one hand, you know, basically cleaning out the necrotic tissue, the dying tissue, and the muscle regeneration system on the other hand, creating new muscle cells. And so that's our ratio and you know, we've now published on this ratio, which means that we've generated a body of data from multiple samples which we could then put into publication for peer review. And so, you know, this really excites us because this provides sort of the QED of what we're doing.
So will this be something, an additional biomarker, if I can use that term, that you'll be using?
Totally. Now it requires biopsy.
So this is this is Basecamp. This is Basecamp.
Basecamp. Okay.
And and we feel that this is, this to us is the combination that I referred to earlier of safety.
Yes.
Really important. We show effect and then we show functional effect. And then we show the link to the biology. So if we can put those three together as kind of a trifecta in our studies, we're affecting the biology, we're affecting function, and it's safe. That becomes very, very compelling for the potential of accelerated approval. And then in the adults, if we show safety, we show through MRI that there's some effect on biology and we show some either ongoing benefit or sustained benefit, then we now have at two different age groups evidence that the drug could be effective and that becomes very, very compelling as well.
And that would be the basis upon which you could possibly file for accelerated approval, assuming everything went well and the data supported it, sometime perhaps even the first half of '27. Is that what you're thinking?
Exactly. That's our thinking. Now, I'll stress one point. We don't need both studies to shoot the lights out. One study or the other. Both studies together, you have the greatest potential for the broadest possible label on the drug.
Okay, let me just summarize where I think, and you can give me feedback on this, where I think you have upcoming catalysts or milestones in the next year or so.
Mhm. You've got your clinical trial results ongoing in your Trailhead study with your adult Duchenne patients and it sounds like the next quarter will include the MRI and some other important data and that sounds like it could be out sometime in the June timeframe. Is that?
Potentially. Do I have that right?
Potentially. Yep.
Okay. Right. And I appreciate there'll be some more analysis. Um, you're going to increase enrollment in Trailhead in Australia and hopefully, I think that'll be something that we'll probably see it sounds like this quarter.
Possibly. Yep. We're hoping that's what we're hoping for.
When that happens, will you either be press releasing it or updating data in clinicaltrials.gov?
Yeah. Um, we will. We always report what is required in clinicaltrials.gov. Other than, you know, press releasing the beginning of Trailhead in the US, which is a new study because it's a new country. I don't think we will press release every individual patient in different countries.
Of course, yes, I understand that. And as you mentioned, one of, I consider it a catalyst, you're filing an IND in the US for Trailhead there and dosing the first patient and that sounds like that could be in the coming months if all goes well.
Yeah. So, Trailhead, our plan is to file with the FDA this month, April. So we hope that study is approved. We hope that study can get up and running in Q2.
Okay. And with the very important phase 2 Basecamp, the global trial with the boys, Q3 of this year, if all goes well, you'll complete enrollment.
Correct.
And if that happens, then you could have topline results by the end of this year.
Correct.
As well, you're hoping to file an IND to begin a trial in the US, phase 2 trial with SAT 3247 in FSHD, possibly this Q2, this quarter.
Um, to get the filing this quarter. Yes.
Yeah. And then if all goes well, you could actually start that trial in or about Q3.
Yes. Yeah.
And that's about a 50 patient trial, more or less.
More or less.
And and I assume that the readout will be, you're talking about the first half of next year.
Um, that study likely will take a little longer than that. It's a, it tends to take a little longer to basically recruit in that particular population of patients for some reason. So more likely that that would read out in the second half of 2027.
Okay. And then if you do have a successful trial in Basecamp, and also hopefully Trailhead, but Basecamp in particular, there is the potential to apply for accelerated approval in the first half of '27.
Correct.
Okay. That's very important. Of course, accelerated approval could lead to commercial approval sometime a year later or so. So that within a couple years potentially you could be on the market. I mean, I know that's best-case scenario, but that would be interesting.
Yeah. So I mean, in the world of wild and crazy possibilities.
Yes.
Once we cross that threshold of an accelerated approval designation, then you're entitled within a matter of months to apply for approval. So this could be much sooner than two years from now.
Wouldn't that be wonderful for patients, for investors? Um, yeah, that would be that would be great. But first, let's get the clinical.
Exactly. First things first, get the trial, get the trial finished. Get the data.
Perfect. So Frank, is there something that I've missed that you'd like to address? I think we've covered a lot of ground and I appreciate the opportunity to speak with you as I said at the outset and I would I would simply like to maybe reiterate that drug development is a process and we have built a team of people that understand the drug development process and have had great success in developing novel medicines in the neuromuscular world and in also in cystic fibrosis with our new head of regulatory development. So, we're very familiar with breakthrough designations. We're very familiar with accelerated approval. We're very familiar with complex diseases and we have attempted and I believe that we're succeeding in building the data set in a sort of a block kind of building format that we started the foundation and now we're building on top of the foundation. Every time we do a study, every time we attempt to develop a data package, it's meant to build on what's gone before. So we can meet this sort of trifecta that I described that at the end of Basecamp, the end of Trailhead, we want to be able to demonstrate the drug is unambiguously safe, the drug is having functional effect, and the drug is linked directly to muscle biology. And that combination in either of those two studies is what we believe provides the basis for an accelerated approval opportunity. That's what we're aiming for. That's the eye on the prize that we have. That's why we went out and raised additional money. That's why we went to NASDAQ because when that happens, we want to be on the best exchange in the world for recognizing the value of a drug that could get approved.
That's that's great. So for full disclosure, I've been an investor in Satellos since 2023. And I've actually published an article about Satellos back last September on Seeking Alpha. And I also want to indicate that this podcast is for information purposes only and should not be considered investment advice. And with that, Frank, I want to thank you once again. That was terrific that you're able to share all that information with our audience.
Thank you so much, Stephen, and thank you for your support and look forward to speaking with you again in the future. Maybe maybe 3 months from now or so.
I would like that.
Okay, super. Thanks so much. Okay, bye. Bye now.