Good morning, everyone. Thanks for joining us to have a conversation with the management of Satellos. Here with us is Chief Financial Officer, Liz Williams, and Chief Medical Officer, Wildon Farwell. Thanks for coming down. Thank you for having us. Glad to be here. I guess, to kick off the conversation, could you guys walk us through how forazapadin could treat DMD without either replacing or patching the dystrophin, which is very different from the current mainstream treatment for the DMD? Yeah, absolutely. Well, thank you for having us here this morning. We're glad to be at the conference. Satellos has a very unique approach for treating DMD, and that approach is based on a discovery that was made by our Co-founder, Dr. Michael Rudnicki. As you pointed out, the current therapies in Duchenne are focused on restoring dystrophin, particularly in the muscle fiber. That has historically been accepted as the primary issue in Duchenne. Dystrophin is missing in the muscle fiber, and this leads to the fiber being weak and more susceptible to damage. What Michael could never reconcile as a stem cell biologist is why boys with Duchenne lose their muscle over time. Yes, the muscle is fragile and susceptible to damage, but a healthy individual has the ability to repair and regenerate the muscle. In individuals with Duchenne, that is not happening. They are actually losing their muscle over time. Michael is a stem cell biologist. He spent his career studying muscle stem cells in particular. When he started digging into the issue, what he identified is that dystrophin has a secondary role. Yes, it is a structural protein in the muscle fiber, but it also plays a signaling role in the muscle stem cells. In its absence, the stem cells are not able to polarize and create the progenitor cells that go on to create new muscle tissue. There are two problems, at least two problems in Duchenne, the structural issue, but also the regenerative deficit issue, which we believe is the more substantial issue in Duchenne. Michael and Frank Gleeson, our CEO, set about to come up with an alternative way to replace this missing signal in the stem cells. It was really important to them to not take a genetic approach. They wanted to take a small molecule approach to have a drug that could be dosed orally, making it easier for patients. That is the genesis of forazapadin. They were able to identify an alternative pathway upstream of dystrophin in the stem cells that regulates polarity in the stem cells. That pathway is AAK1. We determined by inhibiting AAK1 with a small molecule oral pill, we could reset the regenerative deficit in the stem cells to go on to create new progenitors and repair the damaged muscle. Dystrophin is still missing from that muscle, and a question that we get asked often is, "Well, okay, you are going to create new muscle, but it still does not have dystrophin. Is that muscle going to be any good?" What we see, we find this question interesting because boys are born with Duchenne, and they are born without dystrophin. But that muscle is functional at birth. The boys can grow. They learn to walk. They learn to ride a bike, often play sports, all of that on muscle that does not have dystrophin. The issue becomes over time, the damage to the muscle accumulates, leading to a tipping point where they start to lose function. We believe that if we can help the body reset the regenerative cycle in the stem cells, the body has the potential to repair and regenerate and keep up with that damage, and potentially, hopefully lead to a meaningful improvement in the progression of disease. Oh, thanks, Liz, for that. I guess the question I think naturally with these kind of dystrophin independent mechanism action, how you guys think about the development strategy for the drug? Yeah. I'll start, then you add on. One of the things that's really exciting about our approach is it's not an or, it's an and. Yes, dystrophin will continue to be missing in the muscle fiber, so there's potential for our drug to be used in combination with dystrophin restorative drugs, exon skippers, gene therapies. Our clinical trial, as Wildon will discuss, is treating patients concurrently with steroids, as well as patients who have previously had gene therapy, previously had exon skippers. We believe there is room for forazapadin on its own because it is not a genetic approach. It has the ability to treat all patients regardless of their exon status, regardless of their ambulation status, regardless of their antibody status. But it also could be used in combination with these dystrophin-focused approaches. Cool. Wildon, you want to add anything to that? I guess what I would add to that is that the exon skippers, the gene therapy, they've been available now for several years. There's still very significant unmet need. There's still patients who are not achieving much improvement in function. Some of the newer generation exon skippers are seeing some improvement in function, but still these boys and young men are profoundly weak, have lost lots of muscle, are not able to live their best lives with full function, and unfortunately, they're dying in their late 20s and 30s. So a very profound unmet need. We believe that a therapy targeting the satellite cells to improve muscle regeneration may be able to improve the life of these boys and young men to be able to restore muscle regeneration to a level to allow them to achieve even greater function than they've ever been able to achieve. That's what we've set out to do to really evaluate this very thoroughly within both an adult population as well as a younger population, to see what the overall benefit could be. Got you. Let's dig a little bit deeper into the pipeline here. You guys just updated very encouraging data for six months data from TRAILHEAD. Maybe Wildon, can you give us a big highlight about the update there? Yep, happy to. Satellos initiated their clinical program with a phase I study that was conducted in two basic parts, one in healthy volunteers and then five adults with Duchenne. The healthy volunteer included the single ascending dose, the multiple ascending dose, included overall PK. All of that was very reasonable, acceptable. No safety signals were identified. The PK was as predicted. The five DMD adults also were evaluated for safety tolerability. PK, again, safety profile, very reasonable. No concerning safety signals identified. The PK, very similar to what was predicted based upon the preclinical, similar to what was seen in the healthy volunteer. All of that was very consistent with what was projected based upon the non-clinical data. In that first study, there was also the opportunity to begin to evaluate efficacy. What was observed in this 28-day study, and it was 28 days because that's what the tox profile allowed at the time. In that 28-day study, Satellos evaluated strength, they evaluated respiratory function, and they evaluated different biomarkers. What they saw was that in the first 14 days, there was an improvement in the biomarker profile. This is the proteomic SomaScan analysis. What was seen was a very significant decline in many different proteins that are known to be abnormal in people living with Duchenne. This was a very encouraging sign of biologic activity very soon after initiating the drug. What was also observed was a stabilization or improvement in respiratory function. The FVC improved by 5%-6% over this period of time. We know, especially in adults, respiratory function is a very significant aspect of the disease, and to be able to see the stabilization or improvement was very encouraging. What was also observed was a near doubling in handgrip strength. This was observed to be multiple fold in a few participants. When we looked deeper into that, what we saw was that improvement was associated with drug concentration. Those participants who had a higher drug concentration measured either by Cmax or AUC, those were the ones who were more likely to have an improvement in handgrip strength. It was also associated with baseline creatinine. Why that's important is because we know baseline creatinine is a surrogate for muscle mass. Those participants who had more muscle when they started on the drug were more likely to have the improvement in handgrip strength. Both of these factors bode well for our ongoing study now in boys between the ages of seven and 10 years of age. Because in that study, we're testing the 60 mg dose, which is what we have tested in that first phase I and continuing to test in an ongoing open-label study among adults with Duchenne. But we're also testing 120 mg. So we'll have the opportunity to see if higher drug concentrations can also improve function more in boys living with Duchenne. We also know that boys with Duchenne have more muscle mass than do adults. The two things that predicted a greater response in the adults are present in our study among boys, BASECAMP, which we'll speak to here in just a minute. Just to continue on the results that we saw in the adults. Later, after a 7 - 11 month break, we started what we're now calling the TRAILHEAD Study. Those five participants were invited back. Four of the five were enrolled, and we began to follow them now with muscle MRI, with assessment of their upper extremity function. What we reported at ICNMD earlier in the summer was that all four participants had a decline in muscle MRI, in fat fraction of their biceps. This is completely inconsistent with the natural history. The natural history consistently shows an increase in fat fraction over time across different muscles. What we saw was a 3.7% decline across the four participants, anywhere from 1% to over 6% decline. This is in the four months of observation within TRAILHEAD. In the natural history over a year, what's typically seen is about a 6% increase in fat fraction in this muscle. This is very encouraging. This, again, supports biologic activity and allows us to have confidence in the BASECAMP study. What we also saw was improvement in function, and this was measured by what's called a total effort at the 99th percentile, TE99C. This is a digital assessment where an Apple Watch-like device is placed on the wrist, are then able to assess movement, assess different strength during that movement. What we saw was an improvement of over 30% over the time on drug. Again, very encouraging. We saw improvement in all four participants. Again, this now gives us even more confidence as we're entering into the BASECAMP study, which is ongoing. This will be a study in 51 boys between the ages of seven and 10. We're on track to complete enrollment in that study this month. We can speak to more of what we're looking forward to seeing in that study. We believe that study, plus what we're seeing in TRAILHEAD, may allow us to speak with the agency about paths to accelerated approval for forazapadin in people living with Duchenne. Thanks, Wildon. I guess you guys are also guided, you're getting more updated from the TRAILHEAD by end of the year. Also we probably could look at see some data from new dosed patient. Could you give us a little bit of expectation about what the data readouts could be? Right. With the TRAILHEAD data, we are planning to have 12-month data on these four participants that are currently in the study. Our goal is to increase the participation in TRAILHEAD. We're opening sites in the U.S. We'll have additional sites in Australia. We'll be able to enroll participants between the ages of 16 and 25. It may be possible to also provide early data on some newly enrolled participants in TRAILHEAD by the end of the year. We believe this will then give us the opportunity to see what has happened with more time on drug for muscle MRI in the biceps in these four participants where we presented data earlier. See what's also happened with the upper extremity function, see how that has advanced with more time on drug, then potentially in new participants, begin to see how their strength is progressing as they initiate on drug, all while continuing to evaluate the safety profile in this older adult population. I see. At the same time, let's shift to the BASECAMP there. You're also going to present some data from the BASECAMP by the end of the year. But I believe last week you guys guided, you will spread the data update in two phases. Maybe could you elaborate, first of all, why you spread that? For the first part, what kind of data set we could expect? Sure. BASECAMP, this is our study, randomized placebo control in boys between the ages of seven and 10. It's a three-month placebo control portion followed by a nine-month long-term extension. The boys will be randomized placebo, 60 mg, 120 mg. As I said, we're on track to complete enrollment in that study this month. That will then give us the opportunity to provide an update on that data this year. The full data set will be available early next year. That's when we'll have all the data evaluated and be able to present the full data set. This year, we may be able to provide an update on different aspects of the data, but the full data set will be available early next year. In this data, we will have the opportunity to evaluate the muscle biopsy. For the first time, look at the histology. In non-clinical studies, we see a very profound effect on what we call the regenerative index. The percentage of embryonic myosin heavy chain to IgG, really describing what is the biologic activity of the drug in the muscle. We'll also have muscle MRI. This time it'll be on the vastus lateralis which is a very well-characterized muscle in this age range. Fat fraction in this muscle predicts function as measured by NSAA, measured by ambulation. We'll be able to evaluate that. We'll be evaluating strength in the lower extremities as well as the upper extremities, and then functional assessments like stride velocity, 95th centile, NSAA, PROs. It'll be a very robust data set that we collect within the BASECAMP study. As I mentioned, we believe that because of this robust data, because of the high-quality data set that we're generating, that this may be an opportunity to have conversations with the agency about paths to accelerated approval, depending upon what the results of that data look like. I see. So speak of the endpoint-wise, you mentioned you have MRI, the fat fraction, you have the regenerative index, in combination with also the function test. For that, what is the reasonable expect we could see those change within the 12-week treatment window and what is the other expectation you think you could beyond that? Right. So within TRAILHEAD, what we saw was that the near doubling in handgrip strength occurred within the first month on treatment. In the non-clinical studies, we see a very profound effect in the animals very soon after the drug is initiated. So we do believe that it is possible that we may be able to see an increase in strength early. If you go back to the historical literature, the steroid trials showed an improvement in function early. So we know that with effective therapies, it is able to see change in these assessments early in, after starting treatment. So we are planning to have conversations with the agency, to talk about paths to accelerated approval, based upon what we have seen in TRAILHEAD. We will then adjust our analyses for BASECAMP to account for these conversations on, and dependent upon what we believe is the right path forward. Then, be able to conduct the analyses for BASECAMP to have the conversations with the agency once the full data set is available next year. I see. So when you are taking the data set to have a conversation with, say, FDA, what kind of the package do you expect to convince the agent to give you guys accelerated approval pathway there? The 2018 guidance for Duchenne called out myometry, which is the same thing as dynamometry, so strength, as an intermediate clinical endpoint. They already acknowledge that with improvement in strength, if one can show correlation to function, that that is potentially a path to accelerated approval. In that document, they also talk about MRI. They do not talk as explicitly about fat fraction as they do about dynamometry. Since that guidance document, there has been a lot of natural history showing correlation with function. There has been many sponsors that have been able to show a change in fat fraction and how that is associated with the function. With the TRAILHEAD data, we are planning to have the conversation with the agency about these paths to accelerated approval. We will then incorporate that into our analysis plan for BASECAMP, and we believe that is a potential package that could support accelerated approval depending upon the outcome of the study. Got you. Looking beyond DMD, you guys also guided, initially, the clinical trial in the FSHD. I guess the biggest question is why you picked that as a second indication you pursue and could any data from the TRAILHEAD or the BASECAMP can relatively de-risk the clinical risk for the FSHD? Yeah. FSHD is a very serious muscle disease. It is actually a larger population than is Duchenne. The mechanism that Michael discovered is completely independent of dystrophin. This is a mechanism that exists in all of us. What we know is that people living with FSHD, they have a decrease in muscle regeneration. Their muscle turnover is not able to keep up with all of the loss that is occurring. In preclinical models, when we administer forazapadin, we see an improvement in the strength in the FLExDUX4 mouse model. This is a standard model that everyone in the FSHD field uses to evaluate their drug. We believe that initiating a program in FSHD makes good sense. We know that there is a very significant unmet need. We know that there is a need for additional muscle regeneration. We have the preclinical data to support it. We do believe that there are learnings from TRAILHEAD that further de-risk that program. The ability to see the improvement in a number of different biologic markers, the ability to see improvement in fat fraction, both of these are also abnormal in people living with FSHD. That gives us additional confidence to move into this additional indication. And actually, we're thinking of other indications that this drug and this program could be applied to. I guess to close, Liz, could you remind us what's the cash position for the company and what's the runway, including and not included in? Sure, yeah. Our last report of June 30th, we had just over $60 million U.S. We are a very capital-efficient organization, so that takes us through 2027. When we build that forecast, it includes the operating the TRAILHEAD study, the BASECAMP study, as well as the FSHD clinical study. So funded well past our next set of significant milestones. Got you. Yeah. These are great. Thank for coming and- Thank you. Thanks for having us. Thanks, Arthur. Yeah, thank you.