Good morning, ladies and gentlemen. Welcome to the Capricor Therapeutics DMV program update call. A brief question-and-answer session will follow the formal presentation. As a reminder, this conference call is being recorded. I would now like to turn the conference over to Mr. AJ Bergman, Capricor's Chief Financial Officer. Please go ahead. Thank you and good morning, everyone. Before we start, I would like to state that we will be making certain forward-looking statements during today's presentation. These statements may include statements regarding, among other things, the efficacy, safety, and intended utilization of our product candidates, our future R&D plans, including our anticipated conduct and timing of preclinical and clinical studies, our enrollment of patients in clinical studies, our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory developments involving our product candidates, including the ability to obtain regulatory approvals or otherwise bring products to the market, revenue and reimbursement estimates, projected terms of definitive agreements, manufacturing capabilities, potential milestone payments, our financial position, and our possible uses of existing cash investment resources. These forward-looking statements are based on current information, assumptions, and expectations that are subject to change and involve a number of risks and uncertainties that may cause the actual results to differ materially from those contained in the forward-looking statements. These and other risks are described in our periodic filings made with the SEC, including our quarterly and annual reports. We are cautioned not to place undue reliance on these forward-looking statements, and we disclaim any obligation to update such statements. With that, I'll turn the call over to Linda Marbán, CEO. Good morning, AJ, and thank you so much, all of you, for joining. The purpose of today's call is to provide a regulatory and clinical update for our DMD program and outline next steps. As you know, following the CRL we received in July, we held a Type A meeting with the FDA. The meeting minutes took longer than the standard 30 days because we were going back and forth with the agency on some important clarifications, and so we thought it was best to align with them before we presented any public-facing opportunities. Overall, the dialogue with FDA was constructive and productive. The outcome was positive, and we continue to work closely together on the path forward to approval for DMD and DMD. Let me talk a little bit about HOPE-3, our pivotal trial, which, as you saw in the announcement this morning, FDA has agreed to accept the data and review it as part of our ongoing BLA. The trial was completed with 105 patients. All of them have passed the 12-month mark, and the database is currently undergoing cleaning with anticipated database lock before the end of the quarter with release of top-line data at that time. The data remain blinded, and the analyses are currently underway. We do expect top-line results in mid-November of this year. Importantly, about 70% of the boys and young men had cardiomyopathy at baseline, which means that the trial was well-designed to evaluate both skeletal muscle and cardiac outcomes. This was one of the questions that we had to answer and anticipate with the FDA, and, as you might have seen, was part of the CRL that was actually released publicly. Now, HOPE-3 is one of the largest placebo-controlled trials that has ever been conducted in Duchenne Muscular Dystrophy. With 105 patients, we believe that it is an adequate and well-controlled trial, and with each of the outcomes being both skeletal and cardiac, we're well-powered to a statistical significance. When combined with each of the cohorts, we believe that the dataset allows us the greatest opportunity to see what the evidence is of statistical significance as well as clinical efficacy in DMD. Now, I want to remind you that the data that we submitted as part of the original BLA was data that the FDA had encouraged us to apply on. That was the HOPE2 clinical trial data, as well as the HOPE2 open label extension compared to the external comparator control from Vanderbilt University. It was strong data. It showed that we showed preservation of left ventricular ejection fraction and measure of cardiac function, as well as volumes and other multiple parameters of cardiac function. When we submitted the BLA, as many of you have heard many times, we believe that this was the data that the FDA had requested, and we gave them everything in terms of 50 information requests that came through. We passed our pre-licensing inspection. We had a non-eventful mid-stage meeting, and we're looking forward to our adcom, which was canceled, as well as the late-stage meeting. However, the great part of the opportunity here for Capricor is that FDA knew that HOPE3 was coming. They understand that that data could be very important in demonstrating the efficacy of DMD, and so we had a productive Type A meeting, as I just mentioned, where they said they were not only willing to look at the data from HOPE-3, but they would continue to exercise regulatory flexibility. They were willing to look both at the skeletal muscle endpoint as well as the cardiac muscle endpoint. T his allows for two potential labeling opportunities. The way that that will be manifest is that the Performance of the Upper Limb version 2.0 will stay as the primary efficacy endpoint. The agency was very uncomfortable with the concept of changing the primary endpoint. This is a legacy decision. It's the reason that the original BLA was based on the existing data of HOPE-2 and the HOPE-2 OLE because they were uncomfortable with the concept of changing the primary from HOPE-3. However, as HOPE-3 is well-powered to show skeletal, and they will allow us to continue to maintain and reopen the BLA that is currently under file using the skeletal muscle endpoint as well as the cardiac muscle endpoint for two labeling opportunities, we believe this is potentially the best possible outcome. And certainly, the tone of the meeting was supportive. They talked about the large unmet medical need. They appreciated the very complete presentation that we were able to give them, where we brought our key opinion leaders, Dr. Chet Villa of Cincinnati Children's and Dr. Jonathan Soslow of Vanderbilt, both pediatric cardiologists and perhaps the world's best adjudicators of cardiac function in Duchenne muscular dystrophy. Dr. Craig McDonald, not only our national PI, but probably the most visible Duchenne muscular dystrophy physician in the United States, as well as statisticians and members of our own team to talk not only about DMD, but also about treating the cardiomyopathy and why that is such an important indication. Because remember, it is the number one cause of loss of life in Duchenne muscular dystrophy. Beyond the clinical and regulatory front, we also brought Aidan Leffler, one of our patients, and his mom, Mindy Leffler, who not only is a Duchenne mom but has turned her personal journey into developing understandings of and measurements of clinical reported outcome measures in patients. Both of them presented to the FDA at the Type A meeting and talked about their experience with DMD and how it changed Aidan's life. There is a video available, which I believe the best way to access it is on YouTube, where Aidan talks about his experiences with deramiocel and how it changed his life. We also have the work of Elijah Stacey and an incredibly impassioned plea from his mom, who is also battling stage four cancer, that she is asking for deramiocel to be moved forward and approved so that her son has a chance to access deramiocel as his own cardiac function is beginning to deteriorate. We will continue to work with the patients, with the advocates, with the community, with the Food and Drug Administration, and with all of you to get deramiocel across the line for DMD because we see not only anecdotally but also from the perspective of data that deramiocel indeed attenuates the progression of Duchenne muscular dystrophy both from a cardiac and skeletal muscle viewpoint. With that in mind, the one question that remains a little bit unanswered is the timing of the PDUFA. I know that's going to be the first question that I get. Our plan is to submit the HOPE-3 data as a response to the CRL that we received in July. We are currently working with the agency on how we will deliver that data to them so that it can be best evaluated as quickly as possible. And to that end, we will provide updates as they become available. Financially, we remain in good shape. We have more than $120 million in cash and equivalents, which will fund operations well into 2026 and supports our launch readiness activity. We continue to work with Nippon Shinyaku, our partner, as we prepare for launch. We obviously now are gearing up for top-line data before the end of the year and then ultimately resubmission of our BLA and moving forward as rapidly as possible to PDUFA and therefore to launch. I'll stop there and take any questions as you have them. Again, thank you for your continued support. This has been a challenging time not only for Capricor but for all of biotechnology. As we negotiate the Food and Drug Administration and some of the changes that have been put in place there, we are delighted currently with our potential opportunity. Thank you so much for your time today. Thank you. Ladies and gentlemen, we will now begin the question and answer session. To ask a question, you may press star followed by the number one on your telephone keypad. If you're using a speakerphone, please pick up your handset before pressing the keys. To withdraw your question, please press star followed by the number two. Once again, please press star one to ask a question. Your first question comes from the line of Joseph Pantginis with H.C. Wainwright. Please go ahead. Hi, Linda and AJ. Good morning, everybody. Thanks for taking the questions, and especially for the updated visibility. I know everyone appreciates it across the street and the medical community. A couple of questions, if you don't mind. I know you talked about the PDUFA, but not necessarily the date, but one would consider this putting HOPE-3 data in there a major amendment, or do you feel that, as you said, you'd hope to get the data on a timely basis that might impact whether it's a major amendment or not? Yeah, Joe. T hank you so much for your question. This is one of the reasons why we have held off on some of the public announcements of the results of the Type A. It's just that we're not completely sure on how the FDA is going to ask for this data, how they'll adjudicate it, and how it will impact the timelines. My regulatory team tells me that we're going to submit it as a major amendment, that we're going to hope to weigh on the agency to use a quick turnaround of two months to PDUFA time, keeping our priority review alive. But it's also possible that they'll ask for a Class 2 resubmission, which really would slow down the clock and give them six months to review. We'll continue to keep the street updated as we become more aware ourselves of what the agency will accept and the timelines. Obviously, we'll work as hard as we can to get this across the line as fast as possible. No, that totally makes sense. A lso the question here, and this has been the open question. It's good to have the visibility of keeping the primary endpoint of PUL. But were there any real changes discussed with regard to the statistical analysis plan of how cardiomyopathy might play in? W hen you talk about two potential labeling opportunities, of course, it would be great to be a fly on the wall during your meetings. Do these labeling opportunities still, are they still only gated on PUL before you can, if that fails, for example, which I don't believe it will, negate the potential for cardiomyopathy? Just want to make sure we're going down the right roads here. Yeah. T hanks. A gain, these are really important and salient issues that we grappled with them as well. Our current understanding is that they are wanting to maintain regulatory. I was going to say rigidity, but let's say strength. T hey're going to be looking for the primary efficacy endpoint theoretically to be positive with a p-value of less than 0.05. We are too. W ith the key secondary being cardiac, that would then be adjudicated on its statistical significance as well, which we also have very high hopes of hitting to the level of less than p 0.05. The issue is whether they would look through a negative PUL to the primary cardiac or to the cardiac secondary is one of the issues we've raised with them and talked with them about in detail. As many others who have sat at my desk and other companies know, FDA never gives you firm language that you can absolutely bank upon. But what they did say is they would exercise regulatory flexibility and that they would continue to look at the cardiomyopathy as a separate indication and a separate opportunity. They understand that the current BLA was for the cardiomyopathy. They're not asking for a new BLA, as far as I know, at this point for the skeletal muscle myopathy. I don't know whether then the indication and the labeling would be gated on theoretically and practically the PUL scores, therefore the ambulatory or non-ambulatory status of the patients. Obviously, they don't do full labeling discussions until you get much later stage in the PDUFA process. I know it's a long-winded answer. The answer to the question is this study was well-powered to detect the statistically significant difference in the performance of the upper limb. We saw it in HOPE-2. We see it in the open label extension compared to the external comparators that we've used. C ertainly, this trial is powered for that. W e're looking forward to seeing that data and are expecting to see statistical significance in both the performance of the upper limb and cardiac function as measured by ejection fraction. No, I appreciate that very much. Do n't worry about your answers. My questions were long-winded as well, but it's nice to hear the positive tenor of the discussions. Thanks a lot. Thanks, Joe. Your next question comes from the line of Kristen Kluska with Cantor. Please go ahead. Good morning. Thanks for taking my questions. I'm very happy to see that this alignment is in place and you can focus on executing now. I wanted to ask for the HOPE-3. I know that it's very well-powered, but can you share with us how similar the patient population is relative to the previous trial experiences, which also helped with your powering assumptions? I believe for the original submission, you had included the safety findings from this study. Can you just confirm that's the case and that, again, the safety looks very clean? Yeah. L et's start with safety because obviously that's the most important issue always in a therapeutic. Yes, the safety was submitted with the original BLA. It's been updated, and it continues to be very safe with, thank goodness, no serious adverse events directly related to the product. W e are encouraged, of course, by the safety of the product. In terms of the powering of the study, HOPE-3 was powered on data assumptions from the HOPE-2 clinical study. Remember, the original powering was done based on a 60-patient study that was cohort A. Cohort B was added when FDA had originally asked us to validate the manufacturing of the product from our San Diego facility. T he 105 patients is actually way overpowered based on the original assumptions. The patient populations are very similar. Yes, because so many of those guys that are in the later stages of the skeletal muscle myopathy aspect of Duchenne also have cardiomyopathy with over 70% of them, as I said, having diagnosed cardiomyopathy and probably more of them on the verge of or starting to experience cardiac dysfunction. W e feel very confident on the powering of the study. Thank you so much. Thank you. Y our next question comes from the line of Madison El-Saadi with B. Riley Securities. Please go ahead. Hi guys. Thanks for providing the update today. Could you remind us, is there a correlation between pull and ejection fraction? And then to follow up on a prior comment, given that 70% of patients do have cardiomyopathy, does this tell us anything about what we could expect and how does that align with the HOPE-2 dataset? Thank you. Yeah. H i Madison. Thanks for the questions. In terms of the cardiomyopathy, the HOPE-2 dataset suggests that when you have a, especially if this was very evident in the HOPE-2 open label extension, if you have an ejection fraction that's maintained above 45%, you have a really great chance of seeing stabilization. Once they start dropping below 45%, they have a lot of scar in their heart, a lot of disease. It's harder to stabilize and bring them back. W e're looking to treat primarily those patients. Well, any patient that has diagnosed cardiomyopathy or scar in the heart will be what we ask for on the label. W e're encouraging physicians to consider starting early because the data is stronger the earlier that the therapy is started. I'm sorry, I lost the second part of your question. What was the second part of your question? You mentioned that 70% have cardiomyopathy. I was just curious how that aligned with the phase 2. T he second part, I'll go ahead and squeeze that second part. Will you have ejection fraction or PUL measurements beyond 12 months? I know every patient has gone 12 months, but just curious if you all have some datasets at 18 months, etc. Thanks. Yeah. Sorry. T he part that I missed originally was you asked about a correlation between PUL and cardiomyopathy. And well, they tend to coexist in terms of losing skeletal muscle function. And it's very, very common as the disease progresses to start seeing the cardiac implications. They don't directly correlate and i t's been one of the conundrums in developing therapeutics for DMD because the heart muscle disease develops on its own continuum compared to the skeletal muscle disease. Y ou can have a patient that has pretty advanced heart disease but might be still ambulant, or you can have a patient that is non-ambulant, losing a lot of upper limb function, but has a pretty strong heart. T hey don't correlate, but they both need treating. As Mindy Leffler, Aidan's mom, says so eloquently, "Treat this cardiac, treat the skeletal, whatever you treat will be grateful." We're taking that attitude as well. I thought I answered, but I'll reiterate. The patient population in HOPE-3 in terms of cardiomyopathy is very similar to the HOPE-2 patient population. We were not using inclusion-exclusion criteria in HOPE-2 specifically for cardiac, but if you actually bucket them, they're very similar in terms of the manifestations and representations of ejection fraction, especially as we move into the open-label extension aspect of HOPE-2. Got it. Thanks a lot, Linda. Yep. You're welcome. Your next question comes from the line of Ted Tenthoff with Piper Sandler. Please go ahead. Great. Thank you and very exciting update. I know it's been a long and winding road here, but I appreciate your persistence on behalf of the boys and investors, so thank you for the update. My question really has to do sort of twofold. Firstly, just with respect to Nippon Shinyaku, any updates? What's the latest there? Obviously, that potential milestone for regulatory approval of $80 million could be pretty relevant, and then also when it comes to the potential for pediatric voucher, just remind me sort of what the latest is there and how that could come into play. Thanks. Thanks, Ted. Yeah. I'll take the last one first. In terms of the pediatric voucher, that program currently remains active, as I'm aware, until September of 2026. We expect to have PDUFA before that. That's obviously going to be one of our goals. We do continue to qualify for that. We have not lost that. T hat will be one of the opportunities that will remain front and center as we negotiate with the agency for our PDUFA dates. Plus, we just want to get it out there. The other question that you had was regarding Nippon Shinyaku. Nippon Shinyaku. Nippon Shinyaku. Nippon Shinyaku. Yeah. We continue to work very closely with them. They're doing the work that needs to be done to get ready for launch. In fact, we have a day meeting with them today in order to continue to work on launch activities. Obviously, this news is relieving to everybody that the FDA is willing to take HOPE-3, that we have the opportunity for dual labeling. W e're all now sitting on the edge of our seats, excitedly waiting for the top-line data and to really move this program towards approval very rapidly. Great. Excellent. Thank you so much. Thank you, Ted. Your next question comes from the line of Aydin Huseynov with Ladenburg Thalmann. Please go ahead. Hi, good morning, Linda, AJ. Thanks for providing this update. A couple of questions on our end. D o you think that there may be some subpopulations in the HOPE-3 trial where more advanced patients may have sort of more profound benefits from deramiocel? Like more advanced patients may have more cardiomyopathy. And do you think the FDA will be flexible enough to take sort of a holistic approach and look at both HOPE-2 and HOPE-3 data and give you some sort of more narrow label if needed? Yeah, I don't think that's actually going to be needed, and I actually think it's the opposite, so our data and John's data, John Soslow's data as well, suggest that there's this tipping point around 45% ejection fraction. If you can think of the pathophysiology of Duchenne cardiomyopathy, it's like the skeletal muscle myopathy. You have current steady loss of muscle mass, and that can be measured by late gadolinium enhancement or scar, and the more scar the heart has, the harder it is to beat against that load, so we want to get in early while they still have cardiac muscle to preserve and function to preserve. We believe that's a much better treatment paradigm than trying to salvage late, which is much harder. There's really no way to turn the scar around once it's generated, especially in the heart. No, I don't think that there will be subpopulations that way. I f there were, it would be the greater than 45%. W e're not even talking or thinking about that. We've seen such statistically significant data across all ejection fractions that we've measured. The inclusion and exclusion criteria of HOPE-3 excluded those with very, very severe heart disease. What we know in Duchenne cardiomyopathy is once they cross a certain point where their function has dropped low, it really becomes a very rapid trajectory towards end of life, which is why the patients with Duchenne are so anxious to get on Deramiocel because they know that they're a ticking clock in terms of their cardiac function. Yeah. Makes sense. A nother question I have. During your discussions with the FDA, was there any other additional cardiac endpoint beyond LV, LVEF that FDA was particularly interested in? We didn't really discuss other cardiac endpoints. Obviously, we saw 23 out of 24 cardiac-related endpoints improve in HOPE-2. T hat was published in the Lancet study. We've always presented the totality of cardiac data with MRI. There's a lot. We'll present the volume, left ventricular and systolic index volume, which was very statistically significant and very clinically relevant in HOPE-2 to the FDA. And we'll also present to them a global statistical test of cardiac function, which will then allow them to see the totality of the evidence in terms of preservation of cardiac function. Thanks so much, Linda. Very helpful. Thank you. Thank you. Your next question comes from the line of Catherine Novack with JonesTrading. Please go ahead. Hi. Good morning. Thanks for taking my questions. Just wanted to get clarity on something. So after a month of discussion, ultimately didn't get to change the primary endpoint. So where exactly was FDA flexible? What were the requests that you had that FDA did comply with? Was it the dual labeling? Was it LVEF as a key secondary? Where were they flexible with you? Yeah. So that's kind of a complicated question. Th at they're trying to exercise regulatory flexibility across the program. Their lack of willingness to change the primary efficacy endpoint is really, as I mentioned from what they've explained, regulatory dogma. Our concern, the reason we were asking for the change, is we wanted to keep the current BLA open. The biggest win is that not only are they going to be keeping the current BLA open, they won't require us to submit a new BLA. So we can just submit this data under the current BLA. Our inspections are done. A lot of answers are done. CMC review is done. So this was a giant win for us. We're very pleased with that flexibility. And the dual labeling opportunity obviously is one that's presented by the fact that we'll have a primary of skeletal muscle and a key secondary of cardiac muscle function. So it was more the tone of the meeting, the idea to keep the current BLA open, to understand the unmet needs, to be able to look at the cardiac data. Technically, the study was not originally designed around cardiac function. And they understand that over 70% of the patients have the cardiomyopathy, so they're willing to look at that data. So it's a complicated question because everything is flexible in terms of the fact that they started with having an in-person meeting with us. So I feel very positive about our interactions with the agency right now. Yeah. Okay. And then so that I remember at the time of the CRL, there were CMC items that the FDA had not yet addressed. Are these items still outstanding, or has FDA kind of addressed your responses to those outstanding issues? Yeah. We've submitted them. They've acknowledged that they've received them and are ready to move forward with the next steps towards approval. We do not believe that there are any open CMC issues that will need to be addressed prior to PDUFA. All right. Well, thank you very much for taking my questions. Thank you. Once again, if you would like to ask a question, simply press the star one on your telephone keypad. Your next question comes from the line of Rohan Mathur with Oppenheimer. Please go ahead. Hey, this is Rohan. I'm from Oppenheimer. Thank you for the update. Just a couple from me. When do you expect to submit the final Statistical Analysis Plan? And if HOPE-3 were to miss the primary, would the SAP for LVEF be statistically significant if a p-value of under 0.05 is achieved? And in that scenario, what would be the likelihood? Yeah. So in terms of the statistical analysis plan submission, that is imminent. We're planning on submitting that really within the next few days. It's finalized and under final review. So we're looking forward to getting that in. In terms of whether FDA would look past a failed primary to a key secondary for statistical significance, obviously, that would be something that we would work very hard for them to understand the value of. They'll never say, again, as somebody asked earlier, FDA would never write something like that down, but they did say they would continue to exercise regulatory flexibility, that they understand that the unmet need of cardiomyopathy is separate than the skeletal muscle myopathy, and that there is nothing currently approved to treat the cardiomyopathy associated with Duchenne. There is no guideline-directed medical therapy. Deramiocel certainly provides the very first opportunity. We are hopeful that based on the powering of HOPE-3, based on the data from HOPE-2, based on the open label extension studies compared to the external comparator, and all the other data that we've aggregated over now four years, that we will not have to deal with an issue of passing over the primary to look at the secondary. But please stay tuned as we update you further on our regulatory progress. Got it. And just one more. With respect to discussions with the EU CHMP, can you give any updates there? And how are those discussions being guided by what's been happening with FDA? Yeah. So we've made the strategic decision right now to focus primarily on getting across the line in the United States. There's a lot going on outside the United States. E verybody's aware of sort of President Trump's most favorite nations type of pricing paradigm. And also, the current administration is focusing hard on making things better here in the United States. W e have made the decision, as I mentioned, to focus on getting this across the line in the United States, getting this launch underway, and then we'll focus outside the U.S. more directly at that time. Got it. Thanks, Linda. Yeah. Thank you. And your next question comes from the line of Bhuvan Pachayappan with Roth Capital Partners. Please go ahead. Hi. Good morning. Thanks for taking our question. So a couple from us. So firstly, with respect to HOPE-3, what if your primary—I mean, when you released the data, what if the primary endpoint was not met, but you still achieved statistical significance in your secondary endpoint? What could be the potential next steps? That's the first one. And then in terms of LVEF, I understand that the FDA doesn't want to go against its own regulatory dogma, but what if in the future there's a new drug developer who wants to approach only cardiomyopathy and they wanted to use only LVEF as an endpoint? Because with all the publications from Soslow and others, do you think the agency will be open to discussing LVEF as a primary endpoint for someone else? I know it doesn't concern you, but just curious, your answer will provide some clarity to future drug developers. Thank you. Thanks. Well, your first question, I think I just answered from the Oppenheimer analyst, but I'll reiterate that we're expecting that based on the power in the study to hit the primary efficacy endpoint as well as the key secondary endpoint of left ventricular ejection fraction. If it misses on PUL, will they look at cardiac? That's what we're hoping for. They certainly said they would exercise regulatory flexibility, and they also said they understood that the pathophysiology of the skeletal muscle myopathy and the cardiomyopathy were similar but needed different treatments. There is no guideline-directed medical therapy for the cardiomyopathy associated with Duchenne. And so if that eventuality occurred, of course, we would continue to work with the agency to get it across the line for the cardiac indication independent of the skeletal muscle indication. In terms of other drug developers, I guess I'm going to be a little solipsistic here and say that that's not something I'm thinking a lot about. I'm a cardiac physiologist, so I've spent my entire career thinking about the heart, and I can tell you that I think left ventricular ejection fraction is a really great indicator of the progression of cardiac disease, and I certainly would be wanting to work with the agency and help them understand the value of using that as a clinically driven endpoint so they can predict morbidity and mortality. All right. Thanks so very much. You're welcome. Thank you. I'm showing no further questions at this time. I would like to turn it back to the Capricor management for closing remarks. Thank you so much for joining us this morning. We are delighted to provide this update on our Type A Meeting. We look forward to releasing top-line data before the end of the quarter. This is the culmination of many years of work, a lot of data, a very large clinical trial, and certainly a long and now storied tale with the Food and Drug Administration. Please do pay attention to some of the videos and letters that are out there from the patient community. They not only are quite emotional, but they also provide a really good understanding of how Deramiocel is working in the community. Again, thank you so much and have a great day. Thank you. Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.