Good morning, ladies and gentlemen. Welcome to the Capricor DMD 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. A.J. Bergmann, Capricor 's Chief Financial Officer. Please go ahead. Thank you and good morning. 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 our 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 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 and 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 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. You're 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 everyone and thank you, A.J., thank you all for joining us today. I would like to provide you an important update on the status of our Biologics License Application for Deramiocel, Capricor's lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne Muscular Dystrophy. As announced this morning, Capricor received a Complete Response Letter, otherwise known as a CRL, from the FDA, indicating that the agency is not able to approve the BLA in its current form, specifically citing that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and, in addition, the need for more clinical data. The CRL also referenced certain outstanding items in the Chemistry, Manufacturing, and Controls or CMC section of the application, most of which we believe have been addressed by Capricor in communications to the FDA in response to prior comments, but were not reviewed by the FDA due to the timing of the CRL issuance. At this point, the review clock has been stopped until we submit our complete response and we do not believe that a new BLA will be required. Capricor has already initiated the process to engage with the FDA and is seeking a meeting to discuss the CRL. In parallel, the company is preparing a written response to the CRL with all the next steps now actively in motion. To be clear, we are both surprised and disappointed by the FDA's decision. We have followed the guidance provided by the FDA at every stage from submission through inspection and review. We successfully completed our pre-licensure inspection, completed a mid-cycle review with no significant deficiencies noted, and responded to more than 50 information requests. During the review period, the public advisory committee meeting was canceled by the FDA without explanation. The company was not informed of that decision for more than a week and just one week before our scheduled late-cycle meeting we received the CRL for novel therapy and a new indication with a high unmet medical need. We believe this sequence of events is deeply concerning and, to our knowledge, unprecedented. Capricor's BLA for Deramiocel was granted priority review in March of 2025 and supported by data from the randomized, double-blind, placebo-controlled HOPE-2 Trial, our HOPE-2 Open-Label Extension, and natural history comparisons using FDA-funded data sets. The data submitted for this BLA demonstrated statistically significant, clinically relevant, and durable benefits in cardiac function. Specifically, in HOPE-2, Deramiocel met its primary efficacy endpoint with statistical significance when analyzed using appropriate statistical methodology. Furthermore, the secondary and exploratory cardiac endpoints were prespecified for hypothesis testing and demonstrated a high degree of internal consistency, yielding a composite p value of 0.0066 measuring the global statistical test of all cardiac measures, supporting the reliability and the reproducibility of the observed clinical benefit. In addition, the HOPE-2 Open-label Extension study showed statistically significant results in left ventricular ejection fraction as compared to a propensity-matched external comparator, resulting in a p value of 0.0079 favoring Deramiocel-treated patients after 2 years of continuous treatment. Similarly, when analyzing a global statistical test of multiple key cardiac parameters in the HOPE-2 Open-label Extension at 2 years, a statistically significant result of p equaling 0.0086 was observed. We believe that this is fundamental evidence of the consistency of our data, which highlights the benefit of treatment with Deramiocel across multiple studies and sustained durability. This data was presented to the FDA in August of 2024 and served as the foundation for the acceptance of the BLA, and as such, the CRL was unexpected. Importantly, Deramiocel has now been administered in over 700 infusions across more than 250 patients, many of whom have received multi-year treatments. Across all clinical studies, the therapy has demonstrated consistent and favorable safety, something particularly important in a population as medically fragile as those with Duchenne Muscular Dystrophy, where treatment toxicity tolerance is low and durable therapeutic benefit is rare. Following our review of the CRL, we view our ongoing Phase 3 HOPE-3 clinical trial as a key opportunity to address FDA's request for additional clinical data and to further support the potential efficacy and safety of Deramiocel for the treatment of cardiomyopathy in DMD patients. Capricor has completed the 12-month time point of the study evaluating 104 DMD patients in a randomized, double-blind, placebo-controlled study initially designed to assess upper limb function and key cardiac outcomes, including the changes in left ventricular ejection fraction. One year data are expected in the third quarter of 2025 as we work diligently to finalize the data set for submission. We believe results from cardiac functional endpoints in HOPE-3, if positive, can meaningfully strengthen the current BLA, align with the FDA's guidance for an adequate and well controlled study, and serve as the foundation for a path to potential approval in DMD cardiomyopathy. Now switching to the CMC front, we view the FDA's deferral of review as an opportunity to efficiently clarify and resolve any remaining issues. Based on the feedback provided to us and the actions already taken, we believe these items are resolvable in a timely manner. We remain committed to ensuring the quality, consistency, and scalability of our manufacturing platform. We also want to acknowledge the broader regulatory landscape. While there is growing uncertainty across the sector, we continue to believe the FDA is committed to advancing innovation in rare diseases, particularly where unmet need is high and standard clinical trial models are not always feasible. The use of long term extension data, natural history comparisons, and real world evidence is not only supported by current guidance but essential in therapeutic areas like Duchenne Muscular Dystrophy. We urge the agency to consider the full context, complexity, and urgency of the disease as it evaluates our resubmission. After the cancellation of our advisory committee meeting, a group of the nation's leading DMD physician scientists, including our National Principal Investigator Dr. Craig McDonald, submitted a joint letter to the FDA expressing their support for the Deramiocel clinical program and the data submitted in our BLA. In order to give you context, I would like to share how this letter began. With this powerful statement. We are a group of physicians, scientists, academic collaborators, principal investigators, and biostatisticians who have collectively been involved in the treatment and study of thousands of children and adults with Duchenne Muscular Dystrophy in the clinic and in registration trials spanning four decades. We have over 100 years of treatment experience in this patient population, including expertise in DMD biology, pharmacological strategies, skeletal muscle and cardiac management, natural history, and the design, conduct, and interpretation of clinical trials for Duchenne Muscular Dystrophy. We are writing to share our clinical experiences treating DMD patients in clinical trials of Deramiocel for up to five years and our interpretation of the clinical trial data. These experts, many of whom are directly involved in the HOPE-2 Open-label Extension and the HOPE-3 clinical trials, express their collective belief that the totality of the data supports the efficacy and safety of Deramiocel in treating cardiomyopathy and Duchenne Muscular Dystrophy. Capricor remains committed to working collaboratively with the FDA and remains open to all regulatory pathways that can bring Deramiocel to patients. We will continue to act with urgency, transparency, and scientific discipline. Our goal remains the same, to bring this potentially life-changing therapy to those who need it the most. To our shareholders, collaborators, and most importantly, to the DMD community, we remain fully committed to this mission. We believe in the science, we believe in the patients, we believe in the families who inspire our work, and in the progress that we will still forge ahead to. This is not simply about advancing a therapy, it is about fulfilling a promise to a community that deserves better. We believe this application reflects the type of innovation the FDA has pledged to support. Data-driven, well-controlled, and consistent with regulatory precedent. We appreciate your continued support as we work to bring Deramiocel to the patients who need it most. Thank you. I will now open the line for questions. 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 any keys. To withdraw your question, please press star followed by the number two. One moment please for your first question. We do have our first question coming from the line of Ed Tenthoff with Piper Sandler. Please go ahead. Great. Thank you very much and sorry to hear this news, Linda. I know the CBER director has been vocal in his opposition, but we really think this is a therapy that needs to get approved for the patients. When it comes to the BLA, the response to the CRL as you laid out, when do you think you might be able to respond to the CRL? With the HOPE-3 data, would this still be for DMD cardiomyopathy or would it potentially be for the more expanded DMD label broadly? Yeah. Ted, thank you so much. Of course, while we're disappointed with this news, we are in a really terrific position having an adequate, well-controlled clinical trial in HOPE-3, which very few people in our position actually have. With the data slated to read out within a very short amount of time, really just a few months with last patient, last visit in June, we are working directly with the agency now. In the Complete Response Letter, they opened the door for an informal teleconference to discuss, and quoting the letter, path to approval. They've also suggested in follow-up to that we could actually request a Type A meeting in addition. They're anxious to work with us. I think they could not include HOPE-3 opportunity in the CLR because it was not part of this initial application. We're going to work closely with the agency to build HOPE-3 to be exactly what we need to get this BLA across the line. This BLA is for the cardiomyopathy. Therefore, the simplest path to approval is to see if they will take HOPE-3 for the cardiomyopathy to treat DMD. Of course, there's a broader application in terms of skeletal muscle as well, and we'll look forward to feedback from them. We do believe that based on a lot of the public presentations of Dr. Prasad and Dr. Makari and even Secretary Kennedy, this is exactly the type of therapeutic they want to see move forward. We're confident in our ability to work with them. Great, thanks. That's very helpful. Thanks. Good luck. Thank you. Your next question comes from the line of Leland Gershell with Oppenheimer. Please go ahead. Hi, good morning. Linda and team, also sharing my disappointment to hear the news. Wanted to just clarify with respect to dividing between the observations that have been made and the statistical analyses that have been applied. If you could just provide us some more color there and also how those may impact upon your statistical analysis plan for HOPE-3 with the understanding that the HOPE-3 primary endpoint is the skeletal muscle function analysis. Thank you. Thanks so much, Leland. This is the crux of the matter, right? This is where, you know, colloquially the rubber meets the road. Obviously, FDA had raised concerns about the initial analysis of the HOPE-2 clinical data. That conversation has been back and forth with the agency literally for years. Let me just explain for a minute exactly how we get to the statistical significance and what we've been going back and forth with the agency on and in fact got over the hump in August of 2024 in our meetings with the agency. That was that the original model that was designed for HOPE-2 was based on an 84 patient trial. Because of the early unblinding, there was an imbalance in the distribution. A normally distributed curve was what the original model was based on. You have to meet the assumption of a normal distribution in order to do that analysis. Once the violation of a normal distribution occurs, you must use a nonparametric analysis. Our statisticians have talked to the agency about this in great detail. When you apply the nonparametric analysis, you then have very robust statistical significance of the performance of the upper limb 1.2 mid in HOPE-2, which then leads to all of the other secondary endpoints achieving statistical significance, most importantly being left ventricular ejection fraction. The agency had accepted that. That was one of the topics that was discussed in the meeting in August of 2024, and now they've gone back to a little bit more of their traditional dogma of the fact that it did not meet the original model assumptions in terms of the analysis plan for the HOPE-3 clinical trial. This is where the story gets very beautiful because HOPE-3 was actually and is actually an adequate and well controlled study. Randomized, double-blind, placebo-controlled. We enrolled all the patients anticipated. We did an appropriate power analysis. The study is heavily powered for performance of the upper limb, but even more so for ejection fraction. We believe that the models that we have discussed in terms of the statistical analysis plan for HOPE-3 should be very easy to meet. We look forward to sharing that data with the agency when it becomes available. Thanks. Could you remind us or have you shared the specific powering of HOPE-3 for the primary endpoint based on prior data? Yeah, the pulp primary endpoints. Don't forget, HOPE-3 was originally designed as Cohort A. Based on Agency recommendation, we did Cohort B, which was a completely separate clinical trial of product made in our San Diego facility. Based on recommendations by the Agency, we were able to combine Cohort A and Cohort B. Each of the arms was independently powered. HOPE-3 was powered to a 80% power for a P value of 0.05. Those were the independent arms. Obviously, the combined arms are much more highly powered. All right, great. Thanks for the added color. Yeah, thanks, Leland. Your next question comes from the line of Kristen Kluska with Cantor. Please go ahead. Hi. Good morning, everybody. Sorry to hear about this. I know how much you genuinely care about this patient group. I had a few questions. First, was there anything that they said about the data itself being clinically or not clinically meaningful, or is the sense here that it was just more on the magnitude, the way the study was statistically analyzed based on what you just said for HOPE-2 and the number of patients? Yeah, Kristen, you hit right at, no pun intended, the heart of the matter. It seems mostly that the argument is around the statistical analysis. In HOPE-2, the concept was, as I just explained when Leland asked his question, in the model assumptions and whether to use a parametric normal distribution or a nonparametric model, which was obviously acceptable to the statistical reviewers at The Lancet and to some of the previous reviewers at the Food and Drug Administration. In terms of the open-label extension, their concern is that there was not a concurrent control group. We use the natural history group funded by the FDA. This is a disease that is chronic and is progressive. The really amazing thing about MRI is that it cannot be interpreted right. There's no volitional aspect to measurement of cardiac function by MRI. Some of the concerns about post hoc analysis, for instance, we believe once we explain this clearly to the agency and give them clarity on the MRI, they should be able to understand that this is not necessarily statistically not as relevant as they might have thought. Okay, thanks. For the HOPE-3 study, we understand you're probably somewhere in the cleaning up and analyzing the data step. Can you just give us a sense of your confidence in this trial being successful? I don't know if you have seen any data from the trial yet or anything of that nature. Yeah. The data is being handled by the core labs, the statistical analysis groups. I have not had a chance to see data. We did do and presented to the FDA the safety data from HOPE-3 as part of this application. The safety data looks very good, continues to reflect the strong safety of the clinical trial based on the powering of the trial based on the HOPE-2 study, the HOPE-2 Open-label Extension study, and compared to with the natural history study. We believe that HOPE-3 will be positive, but we haven't looked at the data or had a chance to see it yet. Okay, thanks. Last one for me on why a BLA, a new BLA might not be required essentially. Do you get the sense there are? Certain modules in the application that are already going to be sufficient? They don't need to kind of look over from scratch? I guess I'm trying to understand why a new application might not be required. Is it just the case that you think they're going to just look at essentially what's going to be new to the package, primarily HOPE-3 data? Thanks again everyone. Yeah, thanks. This is what we'll be discussing with the agency. They were clear in their letter that they were going to look for a response to this Complete Response and that the clock would be restarted once we submitted a response to the CR. The whole crux of the matter will be in discussions with the agency. We do not believe that a new BLA will be necessary. We do believe that all of the other modules will be acceptable. They did say that they would re-review the CMC information that was submitted after they had stopped review once they restart the clock. Stay tuned for updates on this. This will be a very important conversation to have with the agency. Thanks again. Thank you. Your next question comes from the line of Madison El-Saadi with B. Riley Securities. Please go ahead. Hi. Thanks for taking our question this morning. I wanted to ask, you mentioned that the powering on HOPE-3 on pull is at 80%. Just wondering what that powering would look like if the ejection fraction was moved to the primary endpoint. If you could, could you clarify, are you expecting like a Class 2 resubmission or would this be a major amendment since it's not going to be an independent BLA? Lastly, could you mention if the FDA viewed the pull data point in HOPE-2 as statistically significant? Thanks. Yeah, thanks Madison. Good to hear from you. To take your questions in order, in terms of the powering of HOPE-3 for ejection fraction, it is highly overpowered. For ejection fraction, as you know, that was strongly statistically significant. In HOPE-2 and the HOPE-2 open like label extension study, very importantly, whether you use the parametric or the non-parametric distribution in HOPE-2, ejection fraction still was statistically significant in terms of the p value associated with it. We haven't done the actual calculations of power, but it's highly overpowered given that there's 104 patients. In terms of this sort of concept of a major amendment or a Class 2 submission, we do maintain our status and priority review due to our regulatory designations. The agency offered to talk with us. We have not had a chance to have a meeting with them yet. We're looking forward to that. We'll provide updates to the street as we have them. Of course, our goal will be that this will be a major amendment to this BLA that will not require a new BLA. They have not suggested that it would require a new BLA. They are asking for additional data. We believe that data can be provided with the HOPE-3 clinical trial data and, as I said before, but I'll reiterate, because the HOPE-3 data was not submitted as part of this BLA, they can't ask for it directly in this Complete Response Letter. What they can do is ask for new data and then we're going to offer them the HOPE-3 data. We remain really hopeful that this is going to be relatively easily resolved and with a little bit of a setback, but not a major setback. Understood. Thanks for taking your question and I'll keep it. Yes, it will. Your next question comes from the line of Catherine Novack with Jones. Please go ahead. Hi, good morning. Thanks so much for taking our questions. Just another question on through the timeline. You have just under two months left on the review. Would that, the new review period, be those two months plus whatever kind of PDUFA extension, assuming you can submit the data under the existing BLA? Why would the FDA stop the review so early prior to looking at any of your CMC responses? Yeah. Thanks for the questions. Again, I can't provide an update on timelines until I meet with the agency. It's going to be critical to have this meeting with them to get their feedback, to see exactly what they're going to want us to do and how that's going to impact timelines. Here's what I do know. HOPE-3 last patient, last visit was in June. We anticipate being able to have top-line data from that study before the end of the third quarter. All of the analyses are being undertaken right now. We remain, of course, completely blinded to anything that's going on. We will reveal top-line data as soon as we have it. I'm hoping that the agency will take this, as I just said in answer to the previous question, as a major amendment. I don't know the impact on the PDUFA clock at this point, but we'll let you know as soon as we know. In terms of their stopping the review, that remains somewhat of a mystery to us. We hit the deadline in terms of when they wanted the information request sent in. Our team worked diligently to make sure that every single one of the 50 information requests were handled and handled in a timely fashion. We did several, actually many, informal calls with the agency over the past six months. We successfully completed our pre-licensure inspection. We submitted our responses to the 483s. They did not give us a reason as to why they stopped review of these issues. What I can tell you is that most of them are easily resolved with the answers to the IRs in their hands or could be resolved with a simple follow-up IR. We remain confident that the CM package is on track whenever they reset the PDUFA clock too. Okay, thanks for clarifying that. I just want to ask about the partnership with NS Pharma. Obviously they're aware of the CRL. Have they said anything about their commitment to commercialization in the U.S.? Even Shinyaku have been an incredibly strong partner with us. They have been with us since we initiated HOPE-3. I think they were delighted, as we were when the agency was willing to review a cardiomyopathy Biologics License Application on the data from HOPE-2 and the HOPE-2 Open-label Extension compared to the propensity match controls from the Vanderbilt University Cardiac Consortium data. However, this is a setback. They remain completely committed. Of course, I've spoken with them, with their CEO and their leadership team, and they are on Team Capricor as we are on Team NS. All right, that's helpful. Thanks, Linda. Thank you. If you would like to ask a question, simply press star one on your telephone keypad. Your next question comes from the line of Boobalan Pachaiyappan with Roth Capital. Please go ahead. Hi team, I'm Maanasa, dialing in for Boobalan. Sorry to hear the news. We have a couple of questions. The first question is are you aware of regulatory precedence in which a drug application, you know, advanced mid cycle review without deficiencies and was issued a CRL at, you know, later stages? The second one is how does the CRL impact the ex U.S. clinical development, you know, particularly in terms of the timeline. Thanks. Yeah, thanks for those questions. No, I mean, I haven't spent a lot of time on regulatory precedents. I just sort of keep up with, you know, the companies that we follow in the space. We certainly don't think this is a traditional pathway. We were surprised. Our mid cycle review meeting was literally 10 minutes out of the allotted 60 because they had no major issues to discuss with us. This came as a complete surprise. To us as well. I can't speak for what happens with other companies in terms of our outside of the U.S. strategy. Obviously, we're going to continue to work with the MA. We're going to continue to work with PMDA. We had already thought that HOPE-3 might be sufficient for use outside of the United States, and our strategies have not changed. Obviously, right now we're going to focus on getting this across the line in the United States, working with the Food and Drug Administration. Thank you. We currently have 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 today. While this news obviously was disappointing, sets us back a little bit, upon reflection, both personally and with my team, I can say that the reason we show up at work here every day is because we want to make the difference in the lives of the young men and boys with Duchenne Muscular Dystrophy. We remain committed to this vision. We believe in the power of Deramiocel to address the consequences of this disease, and we look forward to providing you with updates as we achieve clarity with the FDA as to how to get this across the line. Thank you for your continued support and we look forward to happy outcomes in the future. Thank you, presenters and ladies and gentlemen. This concludes today's conference call. Thank you all for participating. You may now disconnect.