Capricor Therapeutics, Inc.

Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics first quarter 2025 earnings call. At this time, all participants lines are in listen only mode. Following the presentation, we will conduct a question and answer session. If at any time during this call is require immediate assistance, please press star zero for the operator. This call is being recorded on Tuesday, May 13th of 2025. I would now like to turn the conference over to our CFO, H. A. Bergman for the forward looking statement. Please go ahead.

Thank you and good afternoon everyone. Before we start, I would like to state that we will be making certain forward looking statements during today's presentation. The statements may include statements regarding among other things, the efficacy safety and intended utilization of our product candidates, our future R and D plans, including our anticipated conduct and timing of preclinical and clinical studies. Our enrollment of patients in clinical studies are plans to present to report additional data, our plans regarding regulatory filing potential regulatory developments involving our product candidates, potential regulatory inspections, 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 risk and uncertainties that may cause actual results to defer materially from those contained in the forward looking statements. These and other risks are described in our periodic filing, the FCC, including our quarterly and annual reports. We're cautious not to place undue reliance on these forward looking statements. We disclaim any obligation to update such statements. With that, I'll turn the call over to Linda Marban, CEO.

Thanks AJ. Good afternoon everyone and thank you for joining today's first quarter conference call. Today, I will review the latest updates on the progress of our BLA seeking full approval for DERM-iCell and treating Duchenne muscular dystrophy cardiomyopathy as well as a brief update on our pipeline programs. While we understand that there have been some changes at the FDA, we remain confident that the strength of our data, which I will highlight in a few minutes, combined with the unmet need in treating DMD cardiomyopathy will potentially lead to approval. Our path with FDA to this point has been smooth and FDA has not fallen behind in any way. Our objectives, deliverables and timelines remain on track. There has also been concern expressed over the announcement of an FDA advisory committee meeting for CAPRICORP. I want to highlight that having the opportunity to participate in an ADCOM is a positive step for CAPRICORP for DERM-iCell and for the program as a whole, because it gives us the opportunity to showcase the strong scientific and clinical data that is the basis of our BLA. We do not believe nor has FDA signaled that the determination to hold an ADCOM has anything to do with weaknesses in the application. But rather, we believe the nature of a first in class therapy for a new indication warrants additional feedback from subject matter experts in the field, as well as giving the advocacy and patient community an opportunity to voice their opinion on DERM-iCell. The ADCOM also affords us the opportunity to highlight that DERM-iCell has a strong safety record demonstrated in over 700 infusions, treating over 250 patients with some subjects receiving DERM-iCell infusions for almost five years. We are asking for approval for a therapy that has been shown to be generally safe and effective for the treatment of DMD cardiomyopathy for which there are no approved therapies. For those new to the story, DERM-iCell is a cellular therapy. It is a biologic comprised of a rare population of cardiac cells that reside in the heart. We have spent almost 20 years developing, characterizing, and harnessing their potential. Our clinical focus for the past approximately eight years has been to shed muscular dystrophy, a rare, X-linked neurodegenerative disease with no cure, afflicting approximately 15,000 boys and young men across the United States and over 150,000 across the world. These young men aim to live life as any strong willed young adolescent boy would desire to, but throughout the course of their life, they suffer from devastating symptoms ranging from loss of ambulation, leading to full or part-time wheelchair use, deterioration of their upper limb function, leading to assistance needed to accomplish daily tasks such as feeding themselves or taking a drink of water, followed typically by the use of ventilatory support for breathing, and ultimately, DMD takes their life. I highlight these things to paint the picture of what these young men experience in their daily lives and why it is so important to develop therapies to attenuate the disease process. Now, what is our indication exactly? It is the heart disease that affects every patient with DMD at some point in their lives. Every day, silently, the hearts of the DMD boys are being damaged and no standard cardiac medication is good enough to combat that process. DMD cardiomyopathy is now the leading cause of death in DMD and Daramiya Cell is the only therapeutic that has been shown to be effective in slowing the decline in ejection fraction, which is a measure of how the heart is meeting the needs of the body. Well, there has been some progress made in treating the dystrophinopathy with several drug therapies now on the market in the US, specific to the genetic mutation associated with the disease. There are no therapies approved or on the market that aim to specifically treat the cardiomyopathy associated with DMD. Daramiya Cell's mechanism of action, which is immunomodulatory and anti-fibrodip, is directly targeted to treat the secondary effects of DMD. And we believe can be used with other therapeutics, which are currently approved or in development to treat the genetic mutation. Daramiya Cell is delivered by a simple intravenous infusion once a quarter at a dose of one hundred and fifteen million cells. I would now like to discuss the data that supports our BLM. The filing is based on our blinded, randomized, and placebo-controlled HOPE-2 study, and also by the HOPE-2 open label extension study compared to a robust FDA and NHLBI-funded natural history data set. While sample sizes are small, what is most relevant is not the size of the data set, but that the statistically and clinically significant differences are highly unlikely to be due to chance. We have worked with multiple internal and external statisticians, presented the data at meetings and to KOLs, and what we have heard, seen, and acted upon was that the likelihood is extremely low that the impact on the heart, or for that matter, the skeletal muscle, is due to chance. We have three clinical trials and approximately four years of open label extension data that supports that premise. There has also been an emphasis and written guidance from FDA encouraging the use of real-world evidence to support clinical trial data, especially in rare diseases. Daramiya Cell is a perfect case for using this type of data to validate the efficacy of a drug product. Turning to the HOPE-3 trial, our Phase 3 study, which is ongoing and fully enrolled in the United States. I want to be clear that at this time FDA has not requested the efficacy data from the HOPE-3 study to support our BLA application, although FDA has reviewed and will continue to review the safety data from the study. Our current plan is to use this data in the future for potential label expansion and are actively evaluating plans for HOPE-3 to be expanded internationally. We will provide more updates on this program as they become available. Now, as we are transitioning Capricor from a translational medicine company into a commercial stage entity, we continue to actively work with our commercial partner, NS Pharma, on launch readiness for the United States. As we announced earlier today, we also have appointed Dr. Michael Binks as our new Chief Medical Officer. I am extremely proud that Dr. Binks joined our team. He has over 25 years of experience leading global clinical development translational research efforts across the industry. Most recently as Vice President and head of rare disease clinical and translational research worldwide research development and medical advisor. And prior to that at GSK where he was instrumental and advancing multiple first in class therapies in early and late stage development. Based on our current plans, we aim to have over 100 patients transition from clinical to commercial product following potential BLA approval. Let me remind you that we have been providing DERM ISL to all open label extension patients for over 3 years. Nearly all HOPE-3 patients are an open label extension now and will transition to commercial product if it is their desire to continue on DERM ISL. We, along with NS Pharma, are now working with physicians to assist them in preparing to prescribe DERM ISL for DMG cardiomyopathy. We know it will be a partnership between treating neurologists and cardiologists and prescribing DERM ISL. And this is part of the reason we are enhancing our medical leadership with Dr. Binks who will guide the physicians through the prescribing process. Please remember that DERM ISL is not designed to compete with the medicines that address the dystrophinopathy such as gene or exome skipping therapies. But rather to address the secondary aspects of the disease, which is inflammation and fibrosis both in the heart and skeletal muscle and again as a very strong safety profile. It's important to note that a naturally derived cell therapy does not have the safety risk that is in any way similar to the gene therapies which do involve viral vectors. Now for an update on our commercial manufacturing preparations. As you know, we built our San Diego GMP manufacturing facility for the purpose of commercial manufacturing. So I have a high degree of confidence in our processes, procedures and facilities. To remind you, our San Diego GMP facility is fully staffed and operational and is currently producing doses of DERM ISL. In addition, we are underway with our previously announced manufacturing expansion to build additional clean rooms in the same facility. We plan on the expansion to be operational mid to late 2026, allowing us to bolster supply of the product to meet potential demands. Turning to an update on our European partnering opportunities, we remain in negotiations with Nipah and Shinyaqu with respect to the potential distribution of DERM ISL and the European region and have extended the period of negotiation of the definitive agreement through the end of the second quarter. Our strategy is emerging in regards to XUSA markets and we will continue to explore opportunities for our technology and other areas globally. We will add additional color as our strategy for Europe continues to unfold. And finally, for an update on our exosomes program, we continue to develop our stealth exosome platform technology as part of a next generation drug delivery platform. While this program has taken a backseat appropriately to DERM ISL, we are still working to develop exosomes as cellular delivery vehicles. We believe strongly in this opportunity and the exosomes ability to change the way we get biologics across the cell membrane. Our exosome development team is focused on advancing an efficient and cost effective way to manufacture them for scale for therapeutic utilization. Despite all the concerns regarding vaccines based on the newer ethos of the FDA, I am pleased to inform you that our program under project next gen, which aims to test vaccine candidates for COVID-19 prevention and to prepare for future pandemics remains underway. Our vaccine is very important because it is natural, made from the native proteins and contains no adjuvants, which has been one of the main concerns of the new HHS secretary. We continue to work in conjunction with the National Institutes of Allergy of Infectious disease, otherwise known as NIAID, which will conduct the clinical trial and provide the data to us. This vaccine could potentially be game changing as it meets all the criteria set forth by the US government of future vaccine technology. Phase one of the trial is set to start in Q3 and we will provide updates on this program as they become available. In conclusion, our program remains strong, both with our past dare myself potential approval and our exosome platform. Our cash balance totals approximately $145 million with our current runway taking us into 2027 with no additional infusions of cash. If we receive FDA approval, we will be slated to receive an $80 million milestone payment from Nipun Shinayaku. And we will also receive a priority review voucher, which we have the full rights to sell. These non dilutive cash infusions could potentially total well over $200 million. This would allow us to enhance our therapeutic pipeline for dare myself as well as expand other areas of our pipeline in an effort to deliver value for patients and for our shareholders. With over 250 publications on the CDC's, including the mechanism of action, dare myself and multiple statistically and significant and clinically relevant clinical trials. Demonstrating dare myself impact on patients. We look forward to presenting our data to the advisory committee at this time. We don't know the specific date for the meeting, but we will alert the market when a date is set. I want to thank you for joining today's call. We truly appreciate your continued support. I will now turn the call over to AJ to run through our finance.

Thanks, Linda. This afternoon's press release provided a summary of our 1st quarter 2025 financials on a gap basis. And you may also refer to our quarterly report on form 10 Q, which we expect to become available shortly and will be accessible on the website as well as the financial section of our website. Let me start with our cash position as of March 31, 2025, our cash, cash equivalents and marketable securities totaled approximately 144.8M dollars. Turning briefly to the financials revenues for the 1st quarter of 2025 were 0 compared to approximately 4.9M for the 1st quarter of 2024. I'd like to point out that the source of our revenue for the 1st quarter of 24 was the ratable recognition of the 40M dollars we received under our US distribution agreement with the bunch of Yahoo, which at this point has now been fully recognized as of December 31st 2024. Moving to our operating expenses for the 1st quarter of 2025, excluding stock based compensation, our research and development expense for approximately 16.2M compared to approximately 10.1M in Q1 2024. Again, excluding stock based compensation, our general and administrative expense was approximately 3.1M in Q1 2025, approximately 1.8M in Q1 2024. Net loss for the 1st quarter of 2025 was approximately 24.4M compared to a net loss of approximately 9.8M for the 1st quarter of 2024.

We

will now open the line

up for questions.

Thank you. Ladies and gentlemen, we will now begin the question and answer session. If you would like to ask the question, please press start and the number one on your telephone keypad. If you're using a speakerphone, please make sure your mute function is turned off to allow your signal to reach our equipment. Again, please press star followed by one to ask the question. Our 1st question comes from the line of Ted from Piper Sandler. Your line is open.

Great. Thank you very much. Can you hear me?

Okay. We can hear you fine. Thanks, Ted. How are you?

Very well. Thanks. So, I just want to pick up on some of the comments that you had. Have you guys had your site inspection yet in San Diego? And if not, can you tell us when that's scheduled for? And then just with respect to preparation for the outcome, walk us through maybe what you consider to be key features and anything along the lines in terms of particular prep. Thank you.

Thanks, Ted. Yeah, we haven't had our pre-licensing inspection. It's coming up this quarter within the next few weeks. And so we'll update you guys once it's completed. I will tell you this place is a buzz with preparation. It would feel really good about it. We're looking forward to having that inspection done. Reminder, which I said in my prepared remarks, but this facility was built in anticipation of commercial manufacturing. And so we, we feel pretty ready for it. In terms of ad comp prep, we are actively working on that. We had anticipated, as we said multiple times now, that it was going to be requested by the agency. Typically, as I also just stated, when there's a new indication or a new therapy first in class, they almost always conduct an ad comp. We actually think it's an incredibly good sign that they've asked for an ad comp. One, it shows that the agency is really moving forward with our application. And I will tell you with, I think we're over 20 information requests now, a follow up meeting with them as most recently as tomorrow and several other meetings along the way. They are actively in our file, reviewing our file and working with us. They have told us in mid cycle review that there were no substantive issues, which gave us good confidence in that in terms of ad comp prep. We've already had to mock ad comps. And without speaking about that, because of confidentiality, I can say that we pass the flying colors and the data looks really great. So we're looking forward to the future. We're waiting for our produce a date and things here are hopping along as we get ready for approval.

Great. Thank you so much.

Our next question comes from the line of Laila and Gershaw from Oppenheimer. Your line is open.

Good afternoon and thanks for taking their questions. Just two from us. Linda first, just want to ask with respect to Nipan and Shinyaqu, you had signed kind of that letter of intent or what have you back in the fall and I know you'd updated us that you've extended that negotiation period. Just wondering if we could also maybe read into that, that you may be considering an alternative mechanism for launching in your perhaps on your own, which could be more lucrative to Capricor in terms of economics preservation. As you've seen rare disease therapy is often to be even better over in Europe than in the States. And then the second question is with respect to NS in the States, if you could just share to the extent you can kind of how they're set up here in terms of their footprint for marketing, DeraMaisel presenting approval. Thanks.

Thank you. And it's always great to hear your voice. So addressing your first question about our LOI with NS and the EU opportunity, we have had really great experiences building towards commercialization with NS in the United States. We think that they're a great partner in the US and we're excited for the launch. And I'll get into some of that color in your second question. In terms of Europe, we've moved forward very rapidly in the United States. We were told and have now filed a BLA for DeraMaisel in the US. We have a great plan in the US and we believe that that can be enacted in Europe as you correctly stated. Therapies for rare diseases have a different path in Europe and there is nothing currently improved in Europe for GMD and our data is very strong and meets AMA criteria. We are working directly with the European authorities ourselves right now as we prepare for moving into Europe. And so we are evaluating on a regular basis the opportunity. We certainly remain in active negotiations with Nipah and Shinyaqu and we'll provide more updates on that program as our thoughts and theirs evolve. In terms of their opportunity in the States, they've done a really nice job of building a sales and marketing distribution team for Viltepso, which is their ex-unskipper. And so these seasoned executives who are US based and have spent a lot of time in the Duchenne space are preparing to launch DeraMaisel. They have 125 FTEs. We're told that nearly all of them are focusing on DeraMaisel at this point. We work with their leadership both in the States and also in Japan so that we remain aligned on the path forward. We are enhancing our own management team. As you heard today, we appointed Dr. Michael Banks, a chief medical officer. Dr. Banks is going to build some med-affair support behind him, which we'll be talking about over the next month or two. And we will continue to support Nipah and Shinyaqu in their education and commercialization for DeraMaisel to physicians in the community.

Thanks

very much. Thanks Leland.

Our next question comes from the line of Kristen Kloska from Canter Fitcherill. Your line is open.

Hi, good afternoon, everyone. Thanks for taking the questions. I look forward to meeting and working with Dr. Banks on your team. So you made a comment that when you talk to several key leaders, they truly point out that these effects can't be by chance. And that's something that we often hear as well when we speak to thought leaders. But I wanted to ask, what are the biggest key drivers that these physicians say is that proof? And I know the FDA certainly is no stranger to the natural history work having funded it themselves. But maybe can you talk about what they're viewing as truly the strongest signals when they look at that data set?

Yeah, Kristen, thanks. So you have just hit on the most important part of our application, and I want to highlight that. The relevance, even though theoretically in terms of new numbers of patients, the numbers are small, the sample size is small. In reality, the reason that we were able to say very confidently that there is very little chance that the data is due to chance is because of the statistical significance. Now, the statistical significance is actually a number that says how much likelihood is your data due to chance. And what we were able to look at here is that MRI, cardiac MRI, which is an objective measure of cardiac function. You can't wish your heart better. You can't volitionally think, gee, I'm going to perform better in the MRI today. And you can't do anything really to sustain or improve cardiac function in any relevant way in a short term basis. And that is why MRI is such a clever and easy way of determining disease progression. So Dr. Jonathan Soslow, who also is the author, lead author on the paper for the natural history study and is funded, as I mentioned by FDA and NHLBI, to collect that data, has spoken very eloquently and pointed to literature that shows that very few patients are actually necessary to discern a treatment effect using cardiac MRI. So we are confident in the data. We are hopeful that the FDA will be confident in the data and that the adcom will also support the data because, as I mentioned, taken together mathematically as well as in an individual patient basis, the data is a very strong suggestion of not only improvement in ejection fraction, which is a measure of how the heart meets the needs of the body, but also the long term stabilization and even improvement in cardiac function. Our open label extension guides are out to four years now, and some of them have dropped literally no ejection fraction points, which, in a non-ambulant later stage, to send patient is almost unheard of.

Thank you for that. And yeah, that was literally going to be my next question. Last summer is when you presented the three year data. Well, I know we don't have a date yet for this adcom. I'm wondering if you will have any four year data to share at that point with the agency or if you've been disclosing early data as you've had these ongoing review meetings.

Yeah, we plan on presenting the four year data at the PPMD meetings in June. And I'm delighted to say that the four year data is looking very promising. I'll give you that little bit of a preview. So, we're going to be talking about the four year data, but it is really quite exciting to see this long term stabilization. As I mentioned, not only in the disease process, but remember our guys are the older guys. These are the guys that are in the later stage of the disease that they are losing function in a measurable and very almost reliable, bad to say way. And so the fact that there's long term stabilization in cardiac as well as performance of the upper limb function is really important and data we plan to highlight at PPMD.

Okay, thanks. And then the last question that I had is obviously there's been a lot of changes at the administration. So can you just talk from a high level? If the people that you've been speaking with over the course of the year and beyond when you first presented these data to the FDA, has there been a lot of changes within that? Or for the most part are most of the people that you're speaking and working with the same people that have been part of the process. Thanks again, everyone.

Thanks, Kristen. You know, it's funny, I get that ask that question now. I don't know, eight to ten times per day by investors. And I think it's really important to say that we see the FDA really starting to calm down and stabilize the appointment of Dr. McCarrie, the appointment of Dr. Prasad, both of which have spoken about their commitment to rare disease, their commitment to moving therapies forward and their commitment to making sure that medicines get to patients as they need them based on good quality data, which Capricor certainly has, has given us a lot of information. We've been given us great confidence taken together. The reviewers that we've worked with over time for the most part, most of them are there. We believe that Nicole Verdun is still there and she has been working on our file really since the beginning of 2024 when we started having high visibility with the agency. And several of the other reviewers, I don't want to call out their names, are definitely still there, definitely engaged. And as I mentioned in my answer to my question to Ted, you know, we are getting literally bombarded with questions. And opportunities to continue the conversation with FDA on a weekly basis. So we know they're actively in our file.

Our

next question comes from the line of Catherine Novak from Jones Research. Your line is open.

Hi, good afternoon. Thanks for taking my questions. I'm just wondering strategically suppose that the FDA issues a CRL for efficacy in August. You know, what's the plan? Would you then read out hope three and submit for DMD skeletal muscle function? You know, where do you see going from there?

Yeah, that's exactly right. Hi, nice to chat with you again. So, you know, we are in a really unique and quite favorable position in that way. We have a fully enrolled phase three trial, the indication for which is skeletal muscle dysfunction, particularly in the nonambulant patients as measured by the performance of the upper limb. So different indication than the cardiomyopathy. If in the unexpected circumstance, they are they issue a CR for whatever reason. In August, we just turn around and submit the data for the hope three trial, which we expect to be positive based on three positive clinical trials proceeding and just go after the skeletal muscle indication. And the secondary endpoints for hope three are the same cardiac ones that we've applied for. So we would just apply for cardiac and skeletal based on that. It's a randomized double blind placebo control trial and certainly would support any of the findings that we've already submitted on.

Got it. So you would intend to submit for both if if you were filing with help three data.

Yeah, I mean, I'm expecting that the data would be reflective of what we've seen so far. We have done blinded assessments of the hope three data compared to the hope to data and distribution charts look almost identical. They can overlay each other. So I feel very confident that we would see similar results in hope three that we saw in hope to and I haven't seen there's no way to preview blinded data for cardiac. And so we expect that to be positive as well based on the MRI data. We've seen in all our trials.

Great and then if you could share any specific feedback, I think, given you about LVF as a surrogate endpoint, what have they said about this endpoint, the ability to predict cardiac outcomes? How much of that are you supporting with your data? Thanks.

Yeah, so I actually read some of this in one of your notes, so I think it's an important point to highlight FDA has stated that they're not looking at ejection fraction as surrogate endpoint in this specific situation. Rare disease. That's why they funded the study with John Saucer by the office office of orphan products co-funded with the NHLBI, which to determine what measures they could conclude to be outcome measures in a rare disease in a rare pediatric disease. You cannot do an outcome measure like mortality. It's not going to happen. So you have to be able to find other measures that are analogous to those outcomes of mortality or some other type of hospitalization, other types of standard cardiac measurements of progress. And disease and use those and so Dr Saucer published in the journal circulation heart failure in 2023 this beautiful study with the natural history data that we were then able to use on a patient level in a propensity matched way to show that the ejection fraction and treated patients with Jeremiah cells was significantly improved over what would be expected in natural history. Real world evidence has been stated by FDA and has been put forth and guided says as well as in a new office and see that emphasizes looking at the actual progression of a disease real world evidence and able to use that analogous to a clinical trial data set to make sure that efficacy is effectively analyze. So we are in the really nice position of having good efficacy data of our own that we can compare to a natural history data set in an objective measure, which is cardiac MRI of disease progression. And so overall, we don't look at injection fraction, nor does the FDA in this situation as a surrogate measure, but rather an outcome measure.

Got

it. Thanks for

clarifying that for me. That was very helpful. No problem.

Our next question comes from the line of ID has enough from later than burger line is open.

Hi, good afternoon. Linda J. Congrats with the quarter. Thank you for taking questions. I got couple. So, given the sort of general pressure on all gene and cell therapy companies recently, and especially in those sort of treatments that were approved conditionally for almost a decade, never got conformity studies. Do you think a full approval there? Am I so we put it into some kind of, you know, specific position when it can actually be utilized more than excellent skippers or AV gene therapies?

Yeah, so I really can't comment on that specifically. You know, we certainly would be in a good position with full approval. We have highlighted in our biologic license application and the community is well aware that we see both skeletal and cardiac benefit. So it's possible that positions would use it with both in mind the patient population that typically have cardiac dysfunction that would be starting out on their myself probably already have implications and skeletal muscle dysfunction that would make them eligible to benefit from their myself. So, you know, that remains to be seen how FDA puts forth the label where we have applied for full approval and will provide updates when we have our label discussions later this summer. They're already scheduled.

Appreciate that. And for the hopes that it data, could you remind us again? When do you plan to to disclose that? I mean, irrespective what's going to happen in August. And if you get approved in based on hope to, how do you think the label is going to change hypothetically once you disclose hope that data and add for a label expansion?

Yeah, so we're in a really great position. As I said a minute ago with hope three, our current plans are somewhat fluid. We're making sure that everything proceeds as planned with this application for the cardiomyopathy. If it does, which we fully expect it to, then our plans are to take hope three to Europe and potentially to Japan to be able to expand our label globally. As we've talked about that would mean that we would keep hope three blinded add more patients from the appropriate geographic area and unblinded when those patients had reached their timeline for their primary efficacy endpoint, which would be a year after enrolling into the study. So that would change the unveiling of the hope three data. If for some reason, as the previous questioner just asked, there was some reason that we needed to move very quickly towards. I'm blinding the hope three data. It would be likely in the third quarter, early fourth quarter of this year, and we would then be able to expand the label in that way. Either in terms of skeletal muscle implications right now, the primary efficacy endpoint is the performance of the upper limb two point. Oh, we have had preliminary discussions with the FDA, although they were in twenty, twenty four that we would ask for approval for full DMD as opposed to all skeletal muscle myopathy's down to diagnosis. But we have not had full labeling discussions with them, nor have we finalized our plans on how that indication would look, but it would be for skeletal muscle myopathy.

Thank you. Thank you. And I hope when the final last question is regarding PRV voucher that you made eligible to receive in August or September. So would you plan to sell it right away and get whatever the last price is, I think, hundred fifty five million or would you like to keep it for possibly using for some indications on your own, such as vector Moscow district?

Yeah, our current plan is to sell it. It will really strengthen our balance sheet and we feel that any indications that have come along, we probably wouldn't need a PRV. We'd get probably priority review anyway for a rare disease. So I don't think that would be as helpful to us as the dollars in our bank account.

Okay, thank you. Thanks so much. Thanks.

Again, if you would like to ask the question, simply press star followed by one on your telephone keypad. Our next question comes from the line of public and papaya from Roth capital. Your line is open.

Hi, my name is and I'm dialing in for so congrats on your progress team and we have a few questions. First in your market research studies have the participants indicated the type of profile that, you know, would make the best indication.

I'm sorry. So are you asking which patients would do best on your myself?

Yeah, like, have the participants indicated the type of, you know, the profile that would like, what that preference would be. Yeah, that's what I'm trying to ask.

Yeah, yeah. So right now, what we are looking for on the label is either the presence of L. G. E., which is late gadolinium enhancement or scar in the heart as measured by MRI. Sometimes you can get that very, very young in age and the kids don't know it. They're sort of ignorance is bliss in terms of the damage to their heart, but they need to start on their myself so that we can attenuate that progression and or the presence of cardiac dysfunction, which would be measured by an ejection fraction of fifty five percent or less, which could be measured in any way, whether by MRI or by echo. So the presence of either or both of those situations would allow for the prescribing of their myself, at least in its first iterations.

Okay, and the next question is, like, can you provide some color on the progress achieved with the Japanese regulators?

Yeah, so we're just working with the Japanese regulators now. We've actually been in conversations with them for a couple of years. They knew that we were waiting for FDA approval before proceeding in Japan. We have some upcoming meetings with the CRO that we're working with in Japan. It's a very well known one called see. And then we're planning to have PMDA meetings either later this year or early next year as we plan on propelling their myself forward in Japan. While we have some ideas of what's going to be required, which does not look like it's going to be too difficult to achieve. I don't have specific information to disclose yet on Japan, but we're actively working on that now.

Okay, okay.

Thank

you. And the last question is, can you kind of speak about the ad coms? Like, can you speak to the ad coms committee composition and expertise? And do you regard the ad com as a positive indicator?

Yeah, so the components are the participants are panelists of the ad com is available online. I don't have it in front of me right now, but you can find it. It's the selling gene therapy advisory committee board. They select from there and then they're allowed to put ad hoc members onto the ad com. Obviously, we don't know who those are yet. We don't have a date yet for ad com, but we are practicing regularly. The company that we're working with brings in panelists that have curriculum details or resumes that are very similar to the panelists so that they can ask and answer questions for us and with us so that we are well prepared and we feel really good about it.

Okay, thank you so much and congratulations again on your progress.

Thank you so much.

I will now turn the call back to Capricorn management for final thoughts.

Thank you so much for joining today's quarterly call. We look forward to updating you on our progress. Obviously, there's going to be a lot of it over the coming months and we will definitely keep you informed as we move forward. Operator, you may now disconnect.

This concludes today's conference. Thank you for participating. You may now disconnect.