On Wednesday December 4th, 2024 PropThink hosted a live webinar with 3 incredible Key Opinion Leaders (“KOLs”) to discuss Duchenne Muscular Dystrophy (“DMD”) and learn about a small Canadian Company – Satellos Bioscience (OTC: MSCLF; TSX: MSCL.TO) – that is testing a novel oral small molecule drug targeting the muscle stem cell in order to regenerate muscle cells – a first-in-class approach to this terrible rare disease. Below is a recording of that live event, featuring DMD patient and patient-advocate Elijah Stacey, muscle stem cell expert Dr. Rudnicki and clinical & regulatory development expert Dr. Dubow.
PropThink DMD KOL Investor Call – TRANSCRIPT
PARTICIPANTS:
- Dmytry Shepsen (“PropThink Host”)
- Elijah J. Stacy (“Elijah”)
- Dr. Michael Rudnicki (“Dr. Rudnicki”)
- Dr. Jordan Dubow (“Dr. Dubow”)
DMD COMPANIES MENTIONED:
- Satellos Bioscience Inc. (TSX: MSCL.TO; OTC: MSCLF)
- Sarepta Therapeutics, Inc. (NASDAQ: SRPT)
- Capricor Therapeutics, Inc. (NASDAQ: CAPR)
- Destroy Duchenne, 501(c)(3) nonprofit (destroyduchenne.org)
- PTC Therapeutics, Inc. (NASDAQ: PTCT)
PropThink Host: Welcome all to this Duchenne Key Opinion Leader Investor Call. I’m Dmytry Shepsen, Founder of PropThink and the host of today’s discussion.
IMPORTANT DISCLAIMER: On this call, we will be making forward-looking statements that are based on assumptions. We are not registered investment advisors and nothing in this presentation should be construed as investment advice. Always consult a professional before making any decisions. You can read our full disclaimer at propthink.com/disclaimer.
PropThink Host: To help us understand what is going on in DMD, shorthand for Duchenne Muscular Dystrophy, we’ve invited three incredible guests, subject matter experts, each with deep professional and, in one specific case, very personal experience with this rare disease.
On this call are Elijah Stacey, a DMD patient, patient advocate, author of A Small If, and founder of Destroy Duchenne, a nonprofit that Elijah founded at 15 years old. Joining Elijah is Dr. Michael Rudnicki – a muscle stem cell and regenerative medicine expert whose research on the subject has been published in Nature, Cell, and other prestigious medical journals. Dr. Rudnicki is the Scientific Founder of Satellos Bioscience – traded on the TSX under the symbol MSCL.TO and over the counter as ‘MSCLF‘. Dr. Jordan Dubow, a neurologist with deep clinical and regulatory development experience across at least a dozen biotech companies including Abbvie (NYSE: ABBV), Cynapsus (which was acquired by Sunovion for $624 million in 2016), and Lexeo (NASDAQ: LXEO), joins Elijah and Dr. Rudnicki to give us his take on the regulatory and clinical development landscape in DMD. Dr. Dubow serves as the Chief Medical Officer of Satellos.
At the conclusion of our discussion, we will open the call to questions submitted to us. If you would like to submit a question, you may do so at any time using the chat box shown in the bottom right-hand corner of your screen.
PropThink Host: Elijah, thank you for joining us.
Elijah: Thank you for having us today.
PropThink Host: How are you?
Elijah: I’m doing good. I’ve been looking forward to this for the last couple of weeks, so it’s great to finally be doing it.
PropThink Host: That’s a great photo, the one on the screen. Who took it?
Elijah: So, this is actually a video that my little brother Kai, whom I’m sure we’ll talk about, took. He recorded this video, and so you could actually see I have like a necklace on. It was my badge. I was an intern for the Riverside District Attorney’s Office, and it was my last day there. And so I was 15 years old. I was in the backyard. My brother Kai, he took the picture in a video of me announcing I’d be starting a nonprofit. But Kai, he actually has Duchenne as well. So, Kai was walking at the time, and it’s common for patients to walk on their tippy toes. So, he’s actually on his toes filming me and taking that picture, and so that’s where this all started.
PropThink Host: Very nice. So, we took that photo from DestroyDuchenne.org. What is ‘Destroy Duchenne’? What do you do?
Elijah: Destroy Duchenne is the nonprofit I founded at 15 years old. Our mission is to destroy Duchenne, right? To cure Duchenne muscular dystrophy. But we also do a lot of everything. So, we have a giving program, which was inspired by my other brother Max, who unfortunately passed away when he was 14. He had Duchenne as well, but he had other health complications. But the thing that I learned is that there’s a lot of costs that are associated with disability that somebody that if they didn’t have it, would have to pay for. So, we have a giving program where we give back in trying to reduce those disability-associated costs that patients and families have. But Destroy Duchenne, it’s been a journey. Since I started, I’ve been giving public speeches to bring awareness and talking to many different people, many different scientists, and just bring awareness of this disease. It led to me writing my memoir, A Small If, which is now being turned into a movie, which is really, really exciting. So, in the Hollywood scene as well. It led me to meeting Jeff Bezos and sitting down and talking to him and telling him about Destroy Duchenne. And just recently, we collaborated with Mark Wahlberg, and we created really cool clothing that he wore on his Instagram and gave a shout-out on screen to raise awareness of Duchenne and Destroy Duchenne. So, that’s really what the nonprofit is. And we’ve had a lot of success in the short amount of time that we’ve been around.
PropThink Host: Very nice. So, we took that photo from Destroy Duchenne.org. What is ‘Destroy Duchenne’? What do you do?
Elijah: Destroy Duchenne is the nonprofit I founded at 15 years old. Our mission is to destroy Duchenne, right? To cure Duchenne muscular dystrophy. But we also do a lot of everything. So, we have a giving program, which was inspired by my other brother Max, who unfortunately passed away when he was 14. He had Duchenne as well, but he had other health complications. But the thing that I learned is that there’s a lot of costs that are associated with disability that somebody that if they didn’t have it, would have to pay for. So, we have a giving program where we give back in trying to reduce those disability-associated costs that patients and families have. But Destroy Duchenne, it’s been a journey. Since I started, I’ve been giving public speeches to bring awareness and talking to many different people, many different scientists, and just bring awareness of this disease. It led to me writing my memoir, A Small If, which is now being turned into a movie, which is really, really exciting. So, in the Hollywood scene as well. It led me to meeting Jeff Bezos and sitting down and talking to him and telling him about Destroy Duchenne. And just recently, we collaborated with Mark Wahlberg, and we created really cool clothing that he wore on his Instagram and gave a shout-out on screen to raise awareness of Duchenne and Destroy Duchenne. So, that’s really what the nonprofit is. And we’ve had a lot of success in the short amount of time that we’ve been around.
PropThink Host: Amazing. So, who would you like to play you in the movie that’s coming up?
Elijah: That’s a hard question. Perhaps I’ll have to play myself. I think who could play me better than myself?
PropThink Host: Yeah, good point. I read that you’re a consultant to two somewhat unorthodox companies in the DMD space – Capricor (NASDAQ: CAPR), and of course, Satellos (TSX: MSCL/OTC: MSCLF). Can you talk a little bit about why you chose to support Satellos – this small, unorthodox company, in particular?
Elijah: Yeah. So, one of the reasons for sure is that they are unconventional. I consider myself an unconventional guy, someone who likes to think different, right? And what happened was I was working at a different company at the time, and I was actually in the Boston Harbor, and I was talking to my colleagues about muscle regeneration. Is that possible? And a lot of them said, there’s nothing really being done with that. It doesn’t really seem to be possible. Once muscle’s gone, it’s gone. But I didn’t like that answer. I wanted to find some academic, someone that was working on this and go and fund them or do something, right? That was my idea. So I went home, did some research, and I found Dr. Rudnicki. And I learned that, okay, he’s a leading stem cell biologist at Muscle Biology, and I thought, okay, this is great. What is he doing with DMD? And it turns out that he’s been working on DMD his whole career and that he has published so many different papers and actually made a huge discovery, which I’m sure he’ll talk about here today. And he also has a company named Satellos (TSX: MSCL/OTC: MSCLF). So I found this company and started doing all the research on them, learning all the science, and it just made so much sense. And it’s really, really exciting stuff, super novel stuff, and really revolutionary, big ideas, and that’s what I love. And so I started to invest in them, started to accumulate tons of shares. I thought, this is a gold mine. Like, this is amazing stuff. I want to be part of this. And then as the months went by, I thought, let me reach out to management. Let me see what I can do because I feel like I can help them. And they met me with enthusiasm, and now I’m a consultant with them. And it’s been a journey. It’s been a lot of fun being here. And I’m excited to see what happens in our trials. But to give you a short answer to your question, it’s because of muscle regeneration, right? I think every patient with whatever drug they take, they want to do better. They want to feel better. And we’re the only company that is truly working on right now restoring muscle, trying to bring muscle back. And I think that’s what patients truly desire at the end of the day. And so that’s why I wanted to be a part of this.
PropThink Host: I wanted to ask you about something you mentioned that I found curious. You mentioned you’ve met [Amazon founder Jeff] Bezos. Did he know about Duchenne before he met you?
Elijah: No, he did not know about it. But he’s somebody that I always looked up to. And I was 17 at the time. And so, yeah, I got to sit down and talk to him, talk about Amazon, Blue Origin, all different things. But definitely talked about Duchenne. And I wanted to ask him, like, how would you approach this? And what are your thoughts on it? And, all that good stuff.
PropThink Host: Does Bezos know about Satellos (TSX: MSCL/OTC: MSCLF)?
Elijah: I’m not sure about that. He might, but I wouldn’t know.
PropThink Host: Maybe after this call, right?
Elijah: Yeah, maybe.
PropThink Host: Dr. Rudnicki, you are a Senior Scientist and Director of the Regenerative Medicine Program in the Sprott Center for Stem Cell Research at the Ottawa Hospital Research Institute. You are a professor at the Department of Medicine at the University of Ottawa. You are also a Scientific Director of the Canadian Stem Cell Network and have been published in the most prestigious medical journals, including Cell and Nature. Some have even called you the Arnold Schwarzenegger of Muscular Science.
Dr. Rudnicki, can you talk to us about what you discovered through your research on muscle that led to the thesis underpinning Satellos (TSX: MSCL/OTC: MSCLF)?
Dr. Rudnicki: So there was a couple of discoveries that were key, and they were serendipity, unexpected results that we followed up on. The first was that using some genetic models in mice, we discovered that muscle stem cells self-renew asymmetrically. This was quite a surprise at the time, and we also developed a set of tools that allowed us to examine that phenomenon. What is asymmetric division? A stem cell can divide in two ways. It can divide to make two copies of itself. That’s symmetric division, and that leads to an increase in the numbers of stem cells. You have two stem cells at the end of that, or it can divide asymmetrically. That occurs at a different orientation, and you end up with one stem cell and one stem cell that is going to become a progenitor that can divide multiple times, six or eight times before it adds itself to muscle. So that’s a big army of cells that can affect the repair or growth of that tissue.
So that was the first discovery. And then we were examining the phenomenon in MDX mice, a mouse model for Duchenne dystrophy. So that first discovery was published in Cell in 2007, I believe. That was an accidental discovery. The second discovery, we were looking at MDX mice, which are a mouse model of Duchenne muscular dystrophy. And we saw that the untreated muscle stem cells had a very low rate of asymmetric division. Their basal rate was in the basement, and that was unexpected. As we drilled down into that, and then we made the really surprising discovery that the dystrophin protein is highly upregulated when quiescent, quiet, resting muscle stem cells are then activated. And then over 30 hours get ready for a cell division.
The protein accumulates, but it accumulates not around the cell, but on one side of the cell, forming a cap. And that whole process is important to what’s called polarization of the stem cell. So it knows which side is up and which side is down, north and south. And without that understanding of what’s north and south, those cell divisions become disorganized. And a lot of them are lost. And so what they do is preferential symmetric expansion, making more stem cells rather than making progenitors. And this leads to a regeneration deficit because they just can’t make enough cells to effectively repair the tissue. And that was a total paradigm shift and change of our understanding of what DMD is. And the take-home message that came out of that work is that DMD is a disease of muscle stem cells. Repair is severely impaired.
PropThink Host: Fascinating. Is there a reason why you were looking at muscle stem cells specifically that long ago?
Dr. Rudnicki: Well, I’m a stem cell biologist. I’m trained as a molecular biologist. We’re interested in how molecules regulate the function of these cells at multiple levels, from signal transaction to control of gene expression, and so on. And we use a variety of different contexts to examine these issues. And one of them is a disease context. We’ve been doing this all along. So it’s an adult type of stem cell that is a great model to study stem cell function.
PropThink Host: Very interesting. Thank you.
Dr. Dubow: You were the Chief Medical Officer for over 10 companies, serving at Cynpasus Therapeutics – acquired by Sunovion for $624 million in 2016 – at Paragon Biosciences, which has invested upwards of $1B in novel drug research, and at Lexeo Therapeutics (NASDAQ: LXEO), a $250M CNS company. You were instrumental in over a dozen new drug applications (NDAs), including Emflaza® – a Duchenne Muscular Dystrophy drug approved in 2017 and now with annualized sales of over $100M. One might say you have something of a Midas Touch when it comes to drug development. How have you been able to find success in so many of the projects you’ve worked on?
Dr. Dubow: I’ve been fortunate to align myself with good drugs and good teams. A lot of it depends on the team. We need to make sure that the study doesn’t fail the drug. And so that commonly happens. I’ve been very fortunate, especially at Satellos (OTC: MSCLF; TSX: MSCL.TO) to work with an outstanding clinical operations team and have great management teams around me. I think it’s really focusing on a drug that we think works and then providing operational and clinical excellence to make sure we run a study as perfect as possible to give a drug the best chance to win.
PropThink Host: What is the importance of having a dystrophin-independent approach for DMD therapy? And what can you do with that, that you can’t do with something that relies on targeting dystrophin specifically?
Dr. Dubow: I think the great thing with Duchenne is that we do have approved drugs, right? And we are finding ways to replace dystrophin. Granted, I don’t think we’re doing it as efficiently as we’d like. And I think the results have played that out. So although that targets somewhat the genetic underpinning of it, it doesn’t completely solve it. We’re not getting there. We need other methods to provide benefit. I think what Elijah just said really resonates and is right. People want drugs that make you better, not just less worse. And so I think some of these other approaches, although they maybe don’t target the genetic underpinning – this [Satellos’] drug can do that. And so I think that’s what’s really unique about what Satellos is doing. It really can make people better.
Dr. Dubow: And we’ve seen this for many other neuromuscular diseases. Right. You may not affect the underlying autoimmune of myasthenia, but people are trying to develop drugs to just make people stronger. And so I think there’s such a need for drugs that work outside of that, that still may provide, even if we say it’s somewhat symptomatic benefit, but regenerating muscle should have a long-term disease-modifying effect as well. So we need that. We’re not at the point where we’ve replaced dystrophin well enough. And so we need other options to help patients get better.
PropThink Host: It sounds like you’re saying there are solutions out there, but there’s so much more to be learned, to be done, which echoes what Elijah had said, what Dr. Rudnicki discovered through his research. And that there’s still a long way to go to treating this disease.
Dr. Dubow: There remains a large medical need. And that’s what you see in the development pipeline right now. And again, as I said, the great thing is, we have drugs. We have drugs that work; some work better than others. But there’s a huge need to develop drugs for obviously all aspects of Duchenne, not just the ambulatory component—non-ambulatory patients, the cardiac component, the pulmonary component as well.
PropThink Host: It’s interesting to us. You had mentioned there were several drugs approved for DMD. In fact, there are eight, to my knowledge, mainly focused on slowing disease progression, not altering the disease. But DMD is a small patient population relative to the patients out there with any skeletal muscular disease, something like 250,000 in total, DMD being something like 20,000 in the U.S. Why has there been so much focus on DMD specifically given that it’s a relatively small patient population?
Dr. Dubow: I think there’s really a number of factors. There still remains an unmet medical need. Unfortunately, we haven’t cured it yet. We haven’t solved all the issues. There are still a large number of patients that could derive benefit. Number two, when you look as a developer, there’s a pretty clear development and regulatory path forward. And what we’ve seen since 2015 with [Sarepta Therapeutics’ (NASDAQ: SRPT)] Exondys is the agency showed some regulatory flexibility, a willingness. This is an important condition. It’s a serious condition with an unmet medical need. We’ve seen companies get regulatory flexibility. So I think that makes people say, hey, listen, maybe the probability of success is a little higher. We don’t need the standard two adequate, well-controlled trials to generate substantial evidence of efficacy. And I think that helps developers. But also importantly, the commercial aspect. Right. We know these drugs are being reimbursed and these drugs are being used. And so from an investment standpoint, there’s been, as far as I can tell, good return on investment. So that cycle continues. We need more drugs. We have the ability to develop drugs. Unfortunately, we have patients that need the drugs and will go into the clinical trials. And then we have a clear regulatory path with the potential for some regulatory flexibility to get things on the market quicker. And so I think for all those reasons, that’s why it’s a tried and true pathway. And, people keep investing in it and people keep trying to get better drugs, which ultimately at the end of the day, we’re trying to benefit patients.
PropThink Host: That makes total sense. For those of you listening, as a reminder, you can submit any question you may have for Elijah, Dr. Rudnicki, or Dr. Dubow using the chat box in the bottom right-hand corner of your screen, and we will address those shortly. Elijah, this question is for you. On the screen, we have a list of approved drugs for DMD, ongoing trials in the clinic, and a list of companies in preclinical research. Just looking at it makes me feel a little overwhelmed. I can’t imagine what this is like for DMD patients. How do you think about which clinical study to choose to partake in? And can you give us some color on how others, other patients, might be thinking about this?
Elijah: Yeah, so you first have to start off with [the fact that] a lot of it is not up to you. You don’t have the option. And what I mean by that is if you look at the exon skippers, for example, sometimes your mutation isn’t on that exon that is amenable to skipping. For example, my mutation is on exon 71. And so exon skippers won’t work for me. That’s like trying to skip the end of the book where it might work in the middle of the book, but it certainly won’t work at the end of the book, like my mutation. So that would throw that out. Others, ambulatory status, whether you can walk or not walk. A lot of the trials tend to focus on patients that are still able to walk. And that makes sense because they’re like gene therapies that are trying to preserve muscle. So you have to have muscle there to preserve. You might have pre-existing antibodies, which if you do, you’re excluded from the trial. You can’t be in that as well. If your mutation happens to be on exon eight or nine, that would also be something that you’re excluded from when it comes to gene therapy.
To start with, you don’t have that many options if you’re somebody like me who’s a non-ambulatory patient. So it depends. Once you get past that, though, then I think it comes down to what is the reward-to-effort ratio, if you will. So what I mean by that is, it’s very difficult for patients to travel. They don’t have places for the wheelchair on the plane. A lot of people aren’t aware of this. If you have to go fly somewhere because these are small biotechs to begin with. They don’t have a lot of resources, so they can’t just put a trial site everywhere. And even if they had unlimited funds, there’s only so many experts that are qualified to actually conduct these trials. Right. And so you might have to go fly somewhere or drive really far. And they’re long, a lot of blood draws, MRIs, biopsies, all kinds of different things that are involved in these trials. And so it’s going to take a lot out of you. And so what are you going to be gaining from it? What do the side effects look like? What does the risk look like? Is it a phase three? Is it a phase two? There’s a lot of questions that I think families have to decide on what they’re willing to do and what they’re comfortable with. And so there’s a lot of factors at play.
For me, if it’s something that I think is really beneficial, then I’m going to make that sacrifice and make it happen. And, if you’re an older patient like myself, you have to ask yourself, where am I at? Where are my lungs at? Where is my heart at? Because perhaps maybe your heart’s not as well. So maybe you want to focus more on cardiac, or maybe it’s your pulmonary function, your lungs. They’re not doing as well. And so maybe you want to work on a treatment that potentially could help preserve that or maybe even improve it. There’s a lot going on with Duchenne. And so you really have to ask yourself where you’re at and all those factors. So it’s very complicated, but that’s the process that I think a lot of patients, including myself, go through.
PropThink Host: Do you feel that many Duchenne patients almost become Duchenne scientists in a way, trying to figure out all these things that you mentioned? What works for me? What doesn’t? Where am I excluded? Where can I participate? Who helps them with all this information? What goes on in the community? Do you have some color?
Elijah: Yeah, well, I think patients really do pay attention to a lot of these events or a lot of presentations at advocacy groups. They’re definitely really involved because this disease is terrible. Patients want to get better. Patients want to live. This is a fatal disease where the average life expectancy is in the second decade or third decade, if you’re lucky. And patients want to live their life. They want to be here. They want to get better. They want to feel better. So they’re constantly looking for answers. They’re constantly looking for things. And yes, they want to understand it. They want to know what can help them in particular. Something else that should be said is Duchenne is really heterogeneous. No two people are really the same with the disease. They have to look at every single different point for themselves. The patient population here is definitely really involved in constantly trying to educate themselves on what’s taking place in the industry.
PropThink Host: Do patients sometimes come to you for advice on how to navigate, what to do based on having some experience now?
Elijah: Well, as I would say, I’m not a medical doctor, right? But we definitely can have conversations and talk about it. And that’s something I’m always trying to inform and educate when we do our giving program. I always ask parents and patients, hey, are you involved in any clinical trials? What are you guys looking at? Are you doing any medication? Just trying to get that information out because I believe that knowledge truly is power. And typically, the earlier you start with a therapy with this disease, the better the outcome will be. So I’m constantly trying to circulate information and put it on my social media, put it on Destroy Duchenne social media, talk to people and all that. So, yeah, there are conversations that we have.
PropThink Host: Incredible. Dr. Rudnicki, this next question is for you. In your view, why is it that others may have missed looking at the muscle stem cell in the context of muscle diseases or Duchenne specifically?
Dr. Rudnicki: I think that’s because it’s the discipline of scientists who are working on Duchenne were originally the histopathologists and then the gene therapists, physiologists. And so they were concerned with function of the entire muscle tissue and differentiated muscle. Also, the understanding, the dogma was that dystrophin was not expressed in muscle stem cells and not in the progenitors. It’s one of the largest, if not the largest gene in our body. It takes a very long time for it, for the RNA to be made and a long time for the protein to be made. And in cells that are dividing, that doesn’t occur. And so it’s not expressed in progenitors, for example. So it wasn’t thought to be an issue, so no one looked. And I’m a stem cell biologist. We work on muscle stem cells, and we looked. So the type of scientist I like is that when we see something anomalous, we drill down and try to learn something from that. So, scientists are human beings, and we tend to look under the street lamp where the light is. And I think that’s part of the explanation. So it took 30 years for that insight to emerge. But it really is, in my view, a paradigm shift in the understanding of the disease.
We know from gene deletion studies in mice that if you delete dystrophin or its transmembrane portion of the complex DAG1, only in muscle fibers, you have a mild muscular dystrophy because the robust regeneration is intact. And if you delete those in both satellite cells and muscle fibers, you have the progressive muscle muscular dystrophy with poor regeneration. So you need both the ongoing damage, structural damage to the muscle fibers, and the reduced ability to repair it to have the full-blown disease. So, the Satellos approach is addressing a mechanism of disease. We’re not treating a symptom. We’re treating something that’s contributing to disease progression. And we really believe we’re going to be able to make an impact with this approach.
PropThink Host: It’s interesting that you use the word symptom because it reminds me of the longstanding consensus in Alzheimer’s, of the plaque accumulation being thought of as the cause of Alzheimer’s and then someone coming out and all of a sudden saying, actually, it may just be a symptom and there’s a deeper pathology.
Dr. Rudnicki: Yes, precisely. It’s an important distinction.
PropThink Host: I want to talk to you a little bit about this study that you recently reported. Satellos recently reported data on lead compound SAT-3247. The chart is up on the screen. What did you find? What did the results suggest for the potential for this drug in terms of functional benefit?
Dr. Rudnicki: Yeah, so we treated two dogs. So this is a pilot study. It’s not a full-blown scientific study because we can’t do statistics. Two dogs, one male and one female, because those were the only dogs that were made available to us. And female dogs are not the same as male dogs because of testosterone. There’s less regeneration. Muscle is not as strong and so on. So dog two is a female dog. That’s why they don’t get as strong. But we saw really a remarkable effect on the ability to generate force for the muscle that after two months of treatment, they markedly increased, and they continued to increase out to four months in quite a dramatic way. And we think those levels are about the same as a disease-free dog, a healthy dog, in the same ballpark. We don’t have those controls, so we can’t say that for sure. That’s benchmarking it against the published data. These muscles are being regenerated. Histologically, when I first looked at those sections from those animals, I thought there had been some mistake. They looked almost normal. It was quite dramatic. And the changes and the other characteristics of dystrophic muscle, they’re being made better. They’re regenerating. So really, I was floored when I was looking at this data. I have to be honest. It was quite exciting.
PropThink Host: How long will these two dogs be followed? Is this study concluded at four months?
Dr. Rudnicki: The study was concluded at four months. That’s all we were allowed to do. We would have liked to have continued, but the center that does the study would not allow it to continue.
PropThink Host: Okay. Based on the study itself, did you gain any insights into potential surrogate endpoints that you might use in clinical development?
Dr. Rudnicki: Yeah, absolutely. So, these are older dogs. I mean, normally these dogs are put down around two years of age because the disease becomes so bad. And at this age, they’re at a plateau phase before they start a real decline. They don’t get better, which in my mind, means this is a difficult test of any therapy, right? We’re not looking at preservation of function. You have to make it better. Like, a young dog, they’re continuing to improve. At this age, they’re not. So histologically, we developed a measure that we think provides good insight into our mechanism of action. We measure the proportion of necrotic fibers that are dying. And we measure the proportion of fibers that are newly regenerating. And we divide the new remade fibers or repaired fibers by the numbers of fibers that are dying. And we call that the regeneration index. If regeneration is outpacing death of fibers, you have a high number. And dystrophic dogs typically have a very low number. And we saw in these animals that that was elevated in every muscle that we looked at. So repair is ongoing everywhere.
PropThink Host: To your knowledge, other companies in any muscular disease that have run animal studies, have they seen similar results with canines? Or is this unusual, maybe best in class?
Dr. Rudnicki: I think in dogs treated at young ages with gene therapy, they preserve the strength of the fiber. And we appear to be in the same ballpark, although we haven’t done a head-to-head comparison, with gene therapy-treated dogs. But they’re preserving strength rather than making and repairing and adding to strength. If you follow me, it’s a little bit different.
PropThink Host: So harking back to functional benefit, that being the hope here.
Dr. Rudnicki: That’s right. We think that as long as that muscle has been preserved, that we can make it better. Or we hope that’s the case.
PropThink Host: Right. Hopefully. Dr. Dubow, this is a question for you. Can this lead compound be used potentially in combination with other DMD treatments like exon skipping or gene therapy?
Dr. Dubow: The good thing about this drug is it can be used with all current treatments. So obviously, we know there are steroids approved or not approved, prednisone but used. And vamorolone was approved. We have new [histone deacetylase] HDAC-inhibitors. And then the genetic targets. And the good news is this mechanism of action can work with all of those. When we study this in patients, this will be adjunctive to current standard of care. Patients in our trials are going to have to be on the standard of care for a certain period of time to ensure stability. We need to ensure there’s equal numbers between different groups. But given this unique mechanism, it can be used adjunctive to all current treatments and potentially other not-approved treatments who are in development as well.
PropThink Host: Has it been decided or discussed internally when that sort of decision might be made earlier on or later on in clinical development? Potentially partner or try it in combination?
Dr. Dubow: It’s going to be as part of the clinical trial. So it’s part of the inclusion criteria. We’re going to allow patients who are on stable doses of various steroids. We’re also going to allow stable doses of these other treatments as long as they’ve been on them for a long enough period of time. So it really won’t be, we don’t have to study it on a combination study, but it’ll just be base background standard of care. And just the most important thing from a development standpoint is that we ensure it’s consistent between groups. So we can do stratification, randomization, and to ensure that they’ve been on doses for a long enough period of time where it won’t need to either bias or make the results harder to interpret. And so that’s, again, the good of this drug is we want to make it available to regardless of your gene mutation or whatnot, regardless of your other background therapy, this drug we think can benefit all comers. And so we intend to study it that way.
PropThink Host: Fascinating. I think that’s incredible. It’s going to give patients some incredible options. Last year, the first gene therapy for DMD, Sarepta’s (NASDAQ: SRPT) Elevidys, was approved through the accelerated approval pathway. I’m wondering, does that affect your thinking on the regulatory pathway for your lead compound or affect your thinking in any way?
Dr. Dubow: It was first approved under the accelerated approval pathway, then [it] received full approval. That’s where, as a developer, oftentimes, it could affect things to your point once a drug is fully approved. What we’re seeing in Duchenne is that, as far as my understanding, the work that I do, it has not closed the pathway of accelerated approval. We do know that gene therapy has its limitations. As Elijah said, I think 30 percent of people have pre-existent antibodies and don’t qualify. Clearly, there are potential safety issues with it. It’s irreversible AAV, high, high doses being infused. As far as we can tell right now as developers, the path of accelerated approval is still open. Now, as more and more drugs become fully approved, that could become less so unless you can show your drug has either an efficacy advantage or a safety advantage. And I think even given that gene therapy has potential toxicities, high-dose AAV, [Liver functioning testing] LFT elevations, those types of situations, and not everyone can actually get the therapy, I think other therapies can prove that they can still be effective and have potential advantages over the currently approved gene therapy. As everyone’s moving forward, that [accelerated approval] pathway has not been shut as of yet.
PropThink Host: It’s probably had the intended effect that the FDA wanted, right? That there’s more and more money and R&D being focused on this terrible disease.
Dr. Dubow: Yeah. I mean, I think both divisions of FDA, both in CDER (Center for Drug Evaluation and Research) and CBER (Center for Biologics Evaluation and Research) for the biological side and the drug side, have shown a great interest in Duchenne [Muscular Dystrophy]. Well, lots of neurodegeneration in general. We’ve seen this with Alzheimer’s. We’re seeing it with ALS. But I think at Duchenne, since, again, starting in 2015 with Sarepta Therapeutics’ (NASDAQ: SRPT) Exondys, they’ve clearly acknowledged it’s an unmet medical need. It’s a serious condition. They needed to show some regulatory flexibility in certain cases. Clearly, the drugs need to meet a bar. They need to be safe. You need certain safety numbers. But they’ve clearly said that this is an unmet medical need and they want to work with companies to help get more drugs approved, which obviously is great for developers and, more importantly, great for patients.
PropThink Host: Yes, I would agree. Could you talk to us a little bit about the studies that are ongoing, the studies that are planned, and the clinical development pathway in general for Satellos (OTC: MSCLF; TSX: MSCL.TO), given the background that we went through, the mechanism, the opportunity there, the landscape, and what everyone else is doing? How is Satellos moving the program forward and what should we expect?
Dr. Dubow: The beauty of [Dr.] Michael Rudnicki’s many, many years of work is finally seen in the clinic, right? This year, the team, all the preclinical work, and then obviously all the clinical operations team getting this drug into the clinic was obviously such a milestone for decades of work from [Dr.] Michael Rudnicki et al. We quickly moved this into the clinic. Our phase one trial is going extraordinarily well, extraordinarily efficient. We’re already through the complete single ascending dose cohorts. We’ve dosed one of the multiple ascending dose cohorts. We’re soon to dose the second cohort and we’re very soon to dose the first adult DMD patient.
Phase one is going very well. I mean, that’s the challenge with it. You don’t know (a) is it going to be safe? And (b) what’s the PK [data] going to look like? Is the drug going to actually behave like you predicted based on your animal work? And the good thing we’re seeing is so far it is. The doses we predicted, the potential doses we think we need, we’re seeing all that in the phase one and the single dose, the multiple dose. It’s blinded data, but we see [the] blinded safety data. So far, we’re not seeing anything concerning from a safety standpoint, which has been consistent with our preclinical work and [the] two animal species.
Phase one is going quite well, quite efficient, with the hope to learn some important things from that. Obviously, we’ll look at the PK, understand the PK, which we’re doing right now, understand the safety, understand the tolerability. And we are dosing an adult DMD cohort. We’ll get to see some pharmacokinetics and patient population safety in a patient population, as well as potentially early signs for [whether] the drug is doing something [functional]. All of that plays into our phase two plan.
We really have a very robust phase two plan. It’s going to be a global phase two plan studying a broad range of ages, both ambulatory and non-ambulatory patients. It’s going to be able to show, number one, [most] importantly, proof of concept. As [Dr.] Michael Rudnicki alluded to, we have a regenerative index with the biopsy, which we’re going to test. Imaging, which with the recent givinostat (Italfarmaco’s HDAC-inhibitor Duvyzat) approval, imaging has become very predictive of function, as well as testing motor strength, muscle function. We’re going to do that in a wide range of patients to start broad because we think this [Satellos] drug could benefit a wide range of patients.
And then based on that, obviously, hopefully, we’ll see movement in the biopsy, imaging, as well as the functional or strength outcomes. Our goal with phase two is still to learn, to understand. But we are very confident the way the phase two study is designed that we look forward to having good data, talking to the FDA, and seeing if there’s an earlier path for us to get to market. Things are moving great, and we’re really excited to keep moving forward. Obviously, we need the help of the community, people like Elijah, the advocacy patients, and their families who have to enroll in these clinical trials. We’re obviously very gracious for all that they do because obviously without that, we can’t do anything.
PropThink Host: In the study that’s currently ongoing with volunteers, that’s mainly a safety study?
Dr. Dubow: Correct. So the first human study, the single dose and the multiple single setting dose, we get one dose at higher and higher. And then multiple doses higher and higher is first with these healthy volunteers. And then the other component is we are actually testing it in adult patients with Duchenne who we’re currently screening. We should be dosing our first [Phase 1a] patient very soon. They’re all going along in parallel. We’re going higher in the healthy volunteers. Based on the dose we think might be the effective dose in patients, we’re studying that dose in a cohort of adults with Duchenne.
PropThink Host: Is there something that could read out from these two ongoing studies that would be sort of an aha moment, a de-risking of sorts?
Dr. Dubow: Clearly safety, [that’s] number one. And it’s funny how, when you develop a lot of drugs, no one even takes for granted the ability to A, get in the clinic, and then B, actually get in the clinic and have the pharmacokinetic profile you want and not see anything safety-related. And so like I don’t want to minimize the safety profile we’re seeing so far, the pharmacokinetics we’re seeing so far, although it may not be an aha moment. It’s a huge thing to check the box to de-risk the program moving forward. We obviously feel very good about [that]. From the patient cohort, we’re obviously going to look to ensure that the PK [profile] in the patient population would look very similar to the healthy volunteers. Then also looking for early signs of efficacy. We’re dosing patients for a long period, for a month. We think there is a chance we may start seeing some functional benefits in these adults. Granted, it’s a single cohort, smaller sample size. We’re optimistic we’ll start to learn very critical information as we then go into phase two and look at both adults, children, ambulatory, and non-ambulatory patients.
PropThink Host: And you’d be looking at imaging in the adult population in the phase one or would that be phase two?
Dr. Dubow: Yes, in the phase two is when we’ll be implementing the MRI.
PropThink Host: Ok, great. At this time, we’re going to open the call to questions from attendees and people that have emailed in questions and submitted them to us. If you’d like to ask a question, there’s still time to do so. Use the chat box in the bottom right-hand corner.
PropThink Host: Elijah, this question is for you. How do the patients you talk to find out about treatment options and new research taking place?
Elijah: Yeah, I think patients probably come across it through the different advocacy groups. When they present at these conferences, other patients are plugged in there, and that’s how they’ll hear about these options. And also, the community [is] very close together. These parents always hear about their own Facebook groups and are constantly communicating with each other. And so if something looks promising, the word gets out pretty quickly and they further investigate from there.
PropThink Host: Word of mouth.
Elijah: Definitely, word of mouth is a big part of it, for sure.
PropThink Host: Maybe all of you [Elijah, Dr. Rudnicki, Dr. Dubow] could take turns answering this next question. Which Duchenne or muscular disease studies are you tracking outside of Satellos, and why?
Dr. Dubow: I can start with that, if you want. As you said, there’s a lot going on, and we see and hear more from public companies than the non-public companies. There’s a second generation of gene therapy companies going into the space right now and we’ll see what happens there. I think we know [that] patients want gene therapy and the currently available gene therapy. There’s other ASOs (Antisense Oligonucleotide) that are trying to get higher expression. Unfortunately, most of the approved ASOs maybe have a few percent dystrophin. Recent [ASO] companies have put out potentially higher levels. That’s something that could be very beneficial to patients. There’s other drugs acting on skeletal muscle, another public company out there that’s doing that. Another looking at both Becker’s and Duchenne. The good news is we all want better treatments for patients. And the more drugs that make it further, the better for patients. We want to learn from other trials and get a better understanding of what goes well, what doesn’t go well. The good news is the pipeline is rich, and we’ll just continue to follow. And obviously, we root for success in this field. And, we hope to continue to move our drug [SAT-3247] forward and have success as well.
PropThink Host: Why follow those when you’re working on a novel unrelated approach? Is it to think about which drugs [Satellos’] compound could potentially be combined with or just to see what other mechanisms might be having an impact on the disease?
Dr. Dubow: Depending on where they are in development, you need to know how the landscape could change and how that could affect your trials down the road. More importantly, as a developer, understanding how placebo arms behave, understanding how active arms behave. One, developed drugs, you always want to study all drugs in your field on what’s the variability? What sites do people go to? What are placebo responses? What did active do? What did placebo do? Because that can help you in terms of designing your next phase of development in terms of powering. Are there better regions to go to, better sites to go to? For that reason, it’s very important for a developer to really study, educate [themselves], and follow what’s going on in the field because you need to learn the best way to conduct your clinical trial.
PropThink Host: How do you think about this, Elijah? Do you follow some specific studies that are ongoing right now or reading out that excite you?
Elijah: As a founder of a nonprofit [Destroy Duchenne] that works in this field, I try to be educated on everything that’s going on because I talk to patients, right? And I want to be able to answer their questions and help them out. I try to look at everything that could be beneficial. I think cardiac treatments are also very interesting and needed. You mentioned Capricor (NASDAQ: CAPR), [that] I’m a consultant with. I think that’s also very interesting for potential cardiac benefit and things like that. So just having that wide range, I think is important to see where we’re going as a community and to see what science is unlocking.
PropThink Host: Dr. Rudnicki, there’s a question here for you as well. As Satellos is conducting clinical trials with the lead compound, given all your experience with muscle dystrophy and the mechanism behind it, where do you see the biggest risk in terms of the phase of clinical trials and why?
Dr. Rudnicki: Where do I see the biggest risk? I guess this is a challenge for everybody. I think it’s the variability between patients and some of the force measurements or strength measurements are even more variable because they’re being done at different centers, like the ambulation test, and so on. I think that’s been a challenge for the field. Doing clinical trials in Duchenne [Muscular Dystrophy] is a new thing. This is recent, so we’re all learning from each other. There are some really interesting things happening, like the use of MRI and so on, that are evolving the field and evolving the trials in a really positive direction. Having too small a cohort could be a problem. I mean, [Dr.] Jordan Dubow is the expert on this, not me. I’m a lab scientist, not a trial expert. But that variance in the statistical analysis, if your sample size isn’t powered correctly, isn’t big enough. But I think we’re going to have a lot of patients, so we should be in good shape.
Dr. Dubow: And I’ll just add to that, [Dr.] Michael Rudnicki. We’re doing a lot of things to mitigate those risks. We are clearly defining age ranges or ambulatory status within different studies to make as little variability as possible, as [Dr.] Michael Rudnicki alluded to. We have multiple ways of looking at this biological effect, a pharmacodynamic surrogate we’re looking at with imaging, as well as, potentially, we can call it clinical intermediate muscle strength or muscle function. So that’s what I really like about how we’ve designed our future studies as it’s really looking at being precise with who you’re studying, why you’re studying, and how you’re studying it to have various ways we can show proof of concept. As [Dr.] Michael Rudnicki alluded to, that’s the challenge with the proof-of-concept study. You’re not powered, necessarily. You don’t have the sensitivity to always show an effect if there’s an effect. And so that’s why I think we’ve done a very good job designing our future studies to give us the best chance to see that effect.
PropThink Host: What is the earliest age that a patient could receive your experimental therapy?
Dr. Dubow: Currently, we’re studying adults, and then in phase two, we’re going to step down to children as young as four.
PropThink Host: That’s pretty standard in the Duchenne space?
Dr. Dubow: That’s the way people are going. As Elijah said, to start the call, we know with the gene therapies, they started at four to eight, it was four to five versus six to seven. [Sarepta’s] givinostat went in [at] six or seven and higher, ambulatory, non-ambulatory. Some of the good news is younger boys are still gaining strength, which is what we want, versus as [patients] get older, you start to lose strength. You need to just ensure that you can design the right study and the right age group with the right endpoints to actually be able to detect a difference when at some ages, there’s still strength gains, then obviously after a certain age, you don’t see those gains anymore.
PropThink Host: Patients who have received gene therapy, would they be able to take part in your early trials?
Dr. Dubow: Yes, we will allow that [but] again, there’s going to be a period of time [that] they have to be on the drug, just to ensure [that] over time — we don’t know the rate of gain. In terms of our study, they will be allowed to be on it. We’re not going to have everyone on it. We’re going to ensure we can evaluate the drug both on top of it and, independent of it, because again, we do know there’s potentially 30% of people who can’t get it [gene therapy]. We just have to over time for what percent of people [gene therapy] becomes standard of care. It’s a little bit early to tell at this point.
PropThink Host: There’s a follow-up here on the pharmacology. Do you have PD measures to assess impact on notch signaling? How will you select the optimal dose?
Dr. Rudnicki: We have ways of doing that based on muscle biopsy. But it’s measuring regeneration rather than notch signaling. I should say that our drug does not alter notch signaling. What it does, if we look at activation of notch target genes, for example—sorry for getting technical—we don’t see an effect. But what’s happening is across a stem cell, there’s forming a gradient of notch signaling, high on one side, low on the other. And that’s what drives an asymmetric division. So we’re not affecting cell proliferation at all. We’re modulating a cell that’s already dividing and we’re stimulating asymmetric divisions, which leads to more cells being able to differentiate. So it’s quite an elegant mechanism of action to drive regeneration.
Dr. Dubow: I’d also add that based on the preclinical work, we know predicted exposures of what we think we need to get optimal efficacy. The dose prediction is tracking very well from the preclinical to the clinical. So it’ll be a combination of that as well. Predicted exposure with safety and tolerability to determine which doses we’re going to move forward into the first studies and besides the phase two in DMD.
PropThink Host: The selection of the dosing, is that informed just by the preclinical work or the volunteer [Phase 1] study and the Phase 1a as well?
Dr. Dubow: Yes, it’s going to be based on the translatability with the preclinical exposures where we saw a maximum effect. Looking at what level, what dose gives us those exposures in humans, and then determining going up or down from there. As I said, the really good thing is, oftentimes your PK in humans doesn’t go as planned, but so far it’s going really well, as predicted. So right now, even though we still have to complete the multiple ascending dose portion and our cohort and patients, we’re at a good place where we feel good about what we’re seeing and how to select the doses moving forward.
PropThink Host: Will you require a muscle biopsy in phase two [studies]?
Dr. Dubow: For some of the studies, we will. We want to look for a biological effect. Showing muscle regeneration, as [Dr.] Michael Rudnicki alluded to, is this regeneration index, which from a clinical trial standpoint, it’s not uncommon to do biopsies. Obviously, you’d love not to do an invasive procedure, even though it’s still minor, but it’s still an invasive procedure and a child in many cases. But that’s where we’ll get that true biological effect, looking at regeneration. We do intend to do biopsy in many of the studies we’re planning.
PropThink Host: When do you expect results from the Phase 1a in adult patients?
Dr. Dubow: Phase 1a in adult patients, we should get Q2-ish, but relatively first half of the year.
PropThink Host: OK, so we can say conservatively mid-year?
Dr. Dubow: I’ll defer to my other colleagues who aren’t on this call right now for absolute timelines, as I don’t want to be quoted [on this]. But yes, the first half of the year.
PropThink Host: For trials in children, when would you expect that that might begin? That would be your Phase 2 study, I imagine?
Dr. Dubow: The Phase 2 study is going to begin next year, in the first half of the year. We’re going to, based on as we go younger in age, there are certain requirements that you have to do to study kids younger. We’re still completing some of that work. We intend it to be an international study where it may start in other places before it starts in North America. We’re going to quickly move from the Phase 1 data to the Phase 2 studies early next year.
PropThink Host: What will be the inclusion criteria for [patient] age?
Dr. Dubow: We’re going to be looking at both ambulatory and non-ambulatory patients. In the ambulatory patients, we’re going to go down as young as four. And then in the non-ambulatory patients, generally, they’re older—12 and higher.
PropThink Host: Okay. Is that four to 18 or four to sort of adolescence?
Dr. Dubow: For the ambulatory study, we’re going to go to four until generally the age where patients start to lose ambulation. And then for the non-ambulatory study, it’s going to be 12 and higher.
PropThink Host: There’s another question for you [Dr. Dubow]. We covered it, but just in case. How do you optimize the dose and design of a Phase 1 so that the canine model data that we saw translates into humans?
Dr. Dubow: The good news from both the rodent and the canine data, and [Dr.] Michael Rudnicki could comment as well, is we see an effect quickly, right? And so obviously, you hope that translates to humans. Earlier in development, usually, you can only study for a shorter period of time until you’ve done longer toxicology studies to go out longer. We feel good based on that data. It’ll translate, and we can actually see effects sooner. I mean, we saw this with steroids, right? You actually see very rapid improvement in strength reduction and the inflammatory component. Based on what we’re seeing in that model, that’s where we’re optimistic. We can actually see biopsy movement, potentially imaging movement, as well as strength movement within our Phase 2 program of shorter duration trials. For Duchenne, standard trials go 48 weeks or longer as you move to Phase 2B and further. But based on the preclinical data, we feel very confident that we’ll see gains of strength and function very quickly.
Dr. Rudnicki: I think it’s also important to point out that we see a pretty broad therapeutic window and how the dose affects the speed of response. But all we’re seeing is a positive response. I think that’s fair to say, right, [Dr.] Jordan Dubow?
Dr. Dubow: Yes.
PropThink Host: There’s another question here on the rationale behind treating adult patients. I guess they’re referring to the Phase 1a versus younger patients.
Dr. Dubow: Our view was [that] getting into patients is important. Getting pharmacokinetics in patients is important. Getting safety in patients is important. We’re studying them for a month-ish, where we potentially can see gains. Our view is there’s still a lot to learn. It could help us as we then translate the pharmacokinetic effect to younger patients. Although we don’t think there are differences in PK between patients and controls, there may be. And so that’s why we felt it was important to do this [adult] cohort, to start to learn some of that information and [to] get some safety data in patients.
PropThink Host: It’s arguably a more difficult cohort to treat, the adult population.
Dr. Dubow: Yeah, and I’ll let Michael speak to that, too. With this mechanism, we do think that there is an age range of patients we can treat. I think we feel more optimistic for younger patients than older, but we don’t know. And so that’s why we don’t want to just assume it’s not going to work at a certain age. And if there’s some muscle to be preserved that we can improve, even if it’s improving certain aspects of upper extremity strength, right, that can lead to functional benefits. We want to be able to see that. We want to be able to test that. That’s why early in development, we do want to test a wider range of patients and ages because we don’t know until we test it where it best works.
PropThink Host: Was there something you wanted to add, Dr. Rudnicki?
Dr. Rudnicki: Anything we can do to improve the quality of life of these patients from being able to eat or swallow, handle a mouse on your computer, that would all be positive. So treating older patients is important.
Elijah: Can I add something to that as well?
PropThink Host: Sure, go ahead.
Elijah: There’s not a lot of options for the non-ambulatory. Considering that this is just a pill [Satellos’ oral SAT-3247], it’s not something that’s an infusion or an injection. It’s straightforward. I think that it’s great that Satellos is offering it. As Satellos said, if muscle’s there, there should be an effect, right? So why not? Why not try and see if it helps, considering how straightforward the therapy is?
PropThink Host: There’s something interesting being asked here. It was sort of at the back of my mind as all three of you were speaking. Someone here asks, as there have been some negative results from other DMD experimental therapies, has that increased the amount of interest in Satellos as a result?
Dr. Rudnicki: Hard to say. Hard to say. Possibly. I mean, we’ve been out in left field from the very beginning, right? It took a long time for people to understand what we were trying to do. It’s a totally different approach to therapy. I think it’s an important shot on goal, if I can use the hockey metaphor.
PropThink Host: How do you feel about this, Elijah? Do you think there’s more interest now in these sort of out-of-the-box, unconventional approaches?
Elijah: I think the fact that we’re the one company working on regeneration, right, trying to restore [muscle], that is so unheard of. Every year, every company we’re talking about, could we just make the disease a little bit slower? How about a little bit more slower? But Satellos comes around and says, could we actually improve the situation? Could we make it better? And I think that once the word is starting to get out, I think that’s what’s driving a lot of interest to the company because let your imagination go wild, right? Let’s see what we can do with this. And that brings a lot of hope to patients like me, because, again, nobody wants to be disabled. Nobody wants to keep having negative milestones. I once was able to raise my arms. Now I can’t. I once was able to transfer myself. Now I can’t. How about we start to get positive milestones that, wow, I’m able to do this again. I’m able to do that again. This is a little bit easier. That’s what we all want and desire as a patient.
PropThink Host: There’s a question here. I’m going to paraphrase if I understood it correctly. This [question] is open for Dr. Rudnicki or Dr. Dubow. If you see functional benefit or what appears to be functional benefit from the Phase 1a in adults, would that then impact the study design or the thinking behind the Phase 2?
Dr. Dubow: We are planning on studying adults in Phase 2 anyway. If we see some motor benefit in that study, that’ll make us feel better. Some of those same outcomes we’re using in the longer Phase 2 study compared to the Phase 1 study. If for some reason, we don’t see something, it’s a small N [number of patients]. It’s a short [duration] study. It doesn’t affect it [Phase 2]. If we do see something, obviously, we’ll feel good about the dose we’re using [in Phase 1]. And then we’re going to study those same assessments in the Phase 2 study as well. And so that’s where, again, the N is going to be a little bit bigger in Phase 2. The duration is going to be longer in the Phase 2 than the Phase 1a right now. We obviously get safety. It’s still, first and foremost, PK. But if we see something, obviously, we’ll use that to our advantage.
PropThink Host: I think this is on study design as well. Somebody asks, boys who have received gene therapy as part of prior clinical trials have already had upwards of three muscle biopsies. Is there any consideration of using MRI as opposed to biopsies in the Phase 2 study?
Dr. Dubow: We are using MRI in the Phase 2 study. We’re going to be doing both [MRI and biopsy]. That’s important depending on, obviously, how many biopsies one could have. And we’ve thought about that carefully for our study. But the good news is we are going to be using the imaging component as well. We looked at it. Biological effect is the biopsy looking at regeneration—the biomarker, which is the MRI of muscle that we’re going to be doing. The other one is the muscle strength or function. Those are the three categories we’re going to be really looking at in our Phase 2 program. We’re hoping we move all of them and we’ll see what we get. We really do think this drug can have an effect, early, on regeneration, which should translate to a biomarker as well as muscle strength. And we’re going to be looking for all of those in our Phase 2 study.
PropThink Host: One final question for Dr. Rudnicki. Are you aware of any examples of companies using stem cells to treat other diseases where the role wasn’t previously well known? Are you tracking any such studies and any indication?
Dr. Rudnicki: Stem cells are targeted in a variety of different disease contexts. I guess one example would be cancer stem cells. So the discovery a number of years ago that tumors have a stem cell and that chemotherapy kills the bulk cells but not the stem cell led to the development of therapies that target the cancer stem cell. This is particularly true for diseases like leukemia and so on, blood cancers. So that’s one example that I can think of. But in terms of regenerative medicine, I think we’re very novel. I mean, what we’re doing is not regenerative medicine, which is cell therapy. We’re doing regenerative pharmacology, which is stimulating intrinsic repair by mobilizing the endogenous stem cell that’s already there, which is quite an innovative approach.
PropThink Host: Gentlemen, if there’s anything else you’d like to add before we conclude the call, the floor is open.
Elijah: I’d just like to say thank you again for having us. And I’m really fortunate, as a patient, to have people like Dr. Rudnicki and Dr. Dubow working on this. They’re very talented, very smart. And this could be a game changer if you’re successful for patients, considering that it’s just a pill, considering that it doesn’t matter what exon your mutation’s on, whether you have pre-existing antibodies, potentially your age won’t be a huge limiting factor, your ambulatory status. This could be a wide-ranging impact for a lot of patients. And so I’m very optimistic about it and just fortunate that there’s so many great people working on this.
PropThink Host: Thank you, Elijah, Dr. Rudnicki, Dr. Dubow, for making this discussion possible. Thank you to the sponsor of this call, Satellos Bioscience, traded in the U.S. under the symbol MSCLF and on the TSX in Canada under the symbol MSCL. If you’d like to learn more about Satellos, visit satellos.com or contact the company using the information provided on the screen in front of you. To learn more about PropThink, visit PropThink.com or follow us on Twitter/X @PropThinker. Thank you for attending, and we hope to see everyone again soon.
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