CAPR - NASDAQ

Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics Second (sic) [Fourth] Quarter 2024 Earnings Call. At this time, all participant lines are in listen-only mode. Following the presentation, we will conduct a question-and-answer session. [Operator Instructions] This call is being recorded on Wednesday, March 19, 2025. I would now like to turn the conference over to our CFO, A.J. Bergmann 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. 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, 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 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 Marban, CEO.

Thanks, A.J. Good afternoon, everyone, and thank you for joining today's conference call. The mission of Capricor has always been to discover and develop transformative drug products for patients in need and translating these biological medicines into commercial products. As many of you know, Capricor was founded over 20 years ago, originally in the laboratories at Johns Hopkins University where we isolated, characterized, and harnessed a unique cell type derived from heart tissue. Even at that time of discovery, the cardiosphere-derived cells, which are now known as deramiocel were conceptualized to potentially be a powerful cellular therapeutic, which one day could treat diseases of the heart for which there are many. Now let me remind you that deramiocel has a defined mechanism of action, which is primarily immunomodulatory and anti-fibrotic driven by the release of exosomes after the cells are infused. Our identity and potency assays, which are supported by the FDA, are based on those properties of bioactivity. Deramiocel is not a stem cell. Let me remind you. Its active ingredient is a rarefied population of cardiac cells that Capricor isolates and expands using proprietary methods. Cells are infused in a simple one time per quarter intravenous infusion at a dose of 150 million cells. With over 700 infusions of the product over many years, deramiocel has been shown to be a safe and effective means for attenuating disease progression. As many of you know, we have been working for over eight years developing deramiocel as a treatment for those impacted by Duchenne muscular dystrophy. And it is with great pride and joy that I can say we find ourselves potentially nearing approval. Today, I will walk you through the latest regulatory, DMC, and commercial updates as Capricor begins to transition from a translational medicines company to a potentially commercial stage company. We will continue to stay committed to our mission, developing transformational treatments for patients in need and driving value for our shareholders. Now turning first to our Biologics License Application or BLA filing for DMD cardiomyopathy, which we announced in early March was accepted by FDA for priority review. The application is seeking full, not accelerated approval of deramiocel to treat DMD cardiomyopathy, an aspect of the disease that not only afflicts essentially all DMD patients, but also is the leading cause of mortality associated with DMD. As we look ahead to our PDUFA date that for August 31st, 2025, we are working with the FDA as they are actively reviewing our application. At this time, the FDA has not indicated to us whether an AdCom will be necessary, but we are preparing for one should that be needed. And I am pleased to inform you that we have officially scheduled our PLI or prelicensing inspection of our manufacturing facility, which is set for the second quarter of this year. Our BLA is supported with data from two trials. Our Phase 2 HOPE-2 placebo-controlled trial and our HOPE-2 open label extension trial compared to patient level natural history data from the DMD Cardiac Consortium led by Dr. Jonathan Soslow at Vanderbilt University. Many factors have given us confidence in our BLA submission pathway. First and foremost, it has a strong safety profile and has been administered to over 250 human subjects across several clinical trials over multiple years. Equally as important is that the data continues to show clinical and statistically significant efficacy in the treatment of DMD cardiomyopathy. This data is foundational to our BLA filing. I would also like to point out that it has become well known that the cardiac and skeletal aspects of the disease do not decline at the same rate. So, therefore, because we see an impact on both cardiac and skeletal muscle function, we are confident that we are seeing a treatment effect of deramiocel across multiple domains, further strengthening the opportunity to treat DMD with deramiocel. Furthermore, as exhibited this week at the Muscular Dystrophy Association conference, we see a year-over-year improvement in function, suggesting that long-term use of deramiocel is warranted for the treatment of DMD. We also have continued to work in close collaboration with the FDA. We have been the beneficiaries of CBER's approach to drug development and believe that with the combination of statistically and clinically significant data that addresses an unmet medical need, we are well poised to gain approval of deramiocel for DMD cardiomyopathy. In addition, our RMAT designation allows us to work directly with FDA as we realize the true goal here is to bring the first treatment to market for DMD cardiomyopathy for which there are no approved therapies. Lastly, throughout 2024, we met with FDA multiple times outlining our BLA filing strategy, of which we have discussed previously. And I want to reiterate that they have not requested the HOPE-3 data, and currently, we do not believe it will be necessary to support our application. As you may recall, the HOPE-3 clinical trial has a primary efficacy endpoint of the performance of the upper limb version 2.0, which is an indicator of skeletal muscle function. We plan to use this data in the future for potential label extension. To that end, we are currently evaluating the plans for HOPE-3 and will provide more updates as they become available. Now turning our attention to commercial planning. We are actively working with our commercial partner, NS Pharma, on launch readiness for the United States. Key areas of overlapping focus are market access and reimbursement. Their team in the United States is comprised of approximately 125 people with market access, reimbursement, medical affairs, and sales teams actively preparing for the launch of deramiocel in The United States. We recently completed surveys with the top five payers in the US with the response coming in very favorably from a reimbursement standpoint for deramiocel. Since there are currently no approved therapies for DMD cardiomyopathy and data suggests that standard cardiac medications do not have a significant impact on disease progression in most cases, we see deramiocel as a transformational treatment option for these boys and young men. We expect reimbursement would be consistent with other recently approved DMD therapies such as exon skippers. If approved, we are anticipating that approximately 50% to 60% of the overall DMD population in the United States or around 7,500 boys and young men with DMD would be eligible for treatment with deramiocel. If approved, we are anticipating entering the market with approximately 100 patients transferring from our open label extension trial to commercial products. This would drive revenue to the bottom line. To remind you, in the US, we are entitled to between 30% to 50% of revenue share based on sales of the product inclusive of cost of goods sold. Now I'd like to shift our attention to CMC or chemistry manufacturing and controls. Our current GMP compliant facility located in San Diego is fully operational and staffed, producing doses of deramiocel. We built and carefully designed this facility to meet early market demand. The fully operational capacity in this facility can support approximately 250 to 500 patients per year and we believe will be sufficient to support the anticipated first year of launch. As we expect a strong launch and rapid adoption of deramiocel for DMD cardiomyopathy, if approved, I am pleased to announce that we have expanded our current San Diego facility where we have leased an additional 25,000 square feet for which we are going to build additional clean rooms, taking our manufacturing capacity for approximately 2,000 to 3,000 patients per year once completed and fully operational. We have assembled a world class team who is extremely sophisticated in this area and I have high confidence we will have this new expanded facility online by mid-2026, allowing us to bolster supply of the product for the next several years to meet demand. As we continue to expand our capabilities, we are already looking into product development endeavors to allow us to increase our yield further. On the corporate and commercial front, we are able to look at our balance sheet. Our cash balance of approximately $150 million is being deployed across the organization with our current runway into 2027 with no additional infusions of cash. If we receive FDA approval, we will be slated to receive an additional $80 million milestone payment from Nippon Shinyaku. And in addition, we would receive a priority review voucher, which we have the full rights to sell or transfer. These non-dilutive cash infusions could total well over $200 million which would hit our balance sheet in 2025 alongside our existing cash and potential product revenue. This puts us in a strong position to deliver for our shareholders on multiple fronts. Our strong cash position will continue to allow us to strengthen our commercial organization, which includes enhancing our team with commercial and medical expertise in order to fuel future product development opportunities for deramiocel and enable us to build Capricor into a world class revenue generating, cash flow positive company with a commercial product on the market and a robust pipeline of expansion opportunities leveraging cell and exosome based therapeutics. Last, for a brief update on our European partnering efforts. Last year, we entered into a term sheet with Nippon Shinyaku for the marketing, sales and distribution of deramiocel in the European region, subject to finalization of a definitive agreement. Our commitment to Nippon Shinyaku in the USA and Japan as our commercial partner is strong, but we have not yet come to final terms on the definitive agreement, which is still being negotiated. In the meantime, we have achieved important regulatory designations in Europe and are on track for meeting with EMA in the second quarter of 2025. We will provide additional color as our strategy for Europe continues to unfold. Now I'd like to switch gears and give an update on our exosomes pipeline program. We continue to develop our StealthX exosome platform technology as part of a next generation drug delivery platform. Our goal is to build the exosomes into a standardized drug delivery platform that has enhanced capabilities when compared to a lipid nanoparticle, including targeting and delivering contents across the cell membrane. While most of our focus has been on the commercialization of deramiocel cells, we have had a small team working on building the exosomes in the background. They have successfully designed a manufacturing method that is cost effective and can be expanded to make large amounts necessary for therapeutic delivery. We have presented this proof-of-concept data at many scientific meetings and published these findings in peer-reviewed journals. Our approach is to concurrently demonstrate the utility of the exosomes by developing a vaccine platform that is unique using native proteins loaded in or coated on an exosome that could be made rapidly within the 100 day constraints developed by the US government, but also able to generate a robust and long lasting immune response. That program is part of the US government's project NextGen, which aims to test vaccine candidates for COVID-19 prevention in addition to prepare for future pandemics. Currently, our StealthX vaccine candidate is in the manufacturing phase. The NIAID, which is the National Institutes of Allergy and Infectious Disease will then conduct and fully fund a Phase 1 clinical trial. Currently, manufacturing is underway for our StealthX vaccine and the NIAID is planning for regulatory approval in the second quarter of 2025 with the clinical study initiated soon thereafter. We will provide further updates on this program as they become available. Now that, deramiocel is on a defined path towards potential commercialization, we are further evaluating the path forward for therapeutic exosome pathway and also evaluating other opportunities to expand our future pipeline. In conclusion, 2024 was a transformational year for Capricor and 2025 is the year in which we will hopefully transition into our next stages of development. I am proud of our progress. I'm grateful to the patients, their families, our investors for their continued support. Capricor's goal is to continue to meet its milestones as we continue to focus on our efforts on bringing deramiocel towards potential commercialization and are investing judiciously across the organization to prepare for that endeavor. I will now turn the call over to A.J. to run through our financials. A.J.?

Thanks, Linda. This afternoon's press release provided a summary of our fourth quarter and full year 2024 financials on a GAAP basis. You may also refer to our annual report on Form 10-K, which we expect to become available in the next several days and will be accessible on the SEC website as well as the financial section of the company website. Let me start with our cash position. As of December 31st, 2024, our cash, cash equivalents and marketable securities totaled approximately $151.5 million. Subsequent to December 31st, 2024, we received a $10 million milestone payment from Nippon Shinyaku pursuant to the terms of our US distribution agreement. On a pro form a basis, our cash, cash equivalents, marketable securities would total approximately $161.5 million. Turning now to the financials. Revenues for the fourth quarter of 2024 were approximately $11.1 million compared to approximately $12.1 million for the fourth quarter of 2023. The source of revenue is the ratable recognition of the $40 million we have received from our US distribution agreement with Nippon Shinyaku and the $10 million milestone payment we triggered in December 2024 after we submitted our BLA to the FDA. Moving to operating expenses for the fourth quarter of 2024, excluding stock-based compensation, our research and development expense was approximately $13.6 million compared to approximately $9.4 million in Q4 2023. And again excluding stock-based compensation, our G&A expense was approximately $3 million in Q4 2024 and approximately $2.1 million in Q4 2023. Net loss for the fourth quarter 2024 was approximately $7.1 million compared to a net loss of approximately $800,000 for the fourth quarter of 2023. And net loss for the year ended December 31st, 2024 was approximately $40.5 million compared to a net loss of approximately $22.3 million for the year ended December 31st, 2023. We will now open the line-up for questions. Operator?

Thank you. Ladies and gentlemen we will now begin the question-and-answer session. [Operator Instructions] Your first question comes from the line of Ted Tenthoff from Piper Sandler. Please go ahead.

Great. Thank you very much and congratulations on all of the progress. It's going to be an exciting year for you guys. Just looking at the commercial preparation for deramiocel, what additional color can you tell us about the prep and sort of the division of labor between you and Nippon Shinyaku? And how much of a benefit will it be with their existing commercial infrastructure? Thanks.

Thanks, Ted. Yes, great to hear from you as always. So, yes, this has been a really exciting time for Capricor and, obviously, NS is very excited as well. We just keep getting our milestones, as we told them we would, and so they are in full scale preparation for commercial launch. They have approximately a 125 employees in the US that have been dedicated to their Viltepso franchise. So they're well in [indiscernible] the Duchenne space. They're spending a significant amount of their time, now focusing on getting, deramiocel ready for commercialization. What that means sort of in the trenches sort of way is that we are working with them on a day-to-day basis, building out modeling for forecasting of market penetration, first year launch activities, working with physicians, and also potential infusion centers to make sure that set up and organized, market access, meeting with payers now that's in full speed, now that we have an accepted BLA. And all of the pieces are in place. Our role is primarily to shepherd deramiocel to market. Their role is to sell it and distribute it. And, obviously, part of selling, a biologic like deramiocel is a deep understanding of the product, which we are providing to them. So we're very, very excited about their energy and also working very closely with them on launch.

Great. Thank you very much.

Yes. Take care, Ted.

Thank you. And your next question comes from the line of Leland Gershell from Oppenheimer. Please go ahead.

Hey, good afternoon. Great to hear all the progress and we're looking forward to events coming later this year for Capricor. Just a few questions from us. First, Linda, I want to ask with respect to your discussions with payers so far, I'm wondering if as part of those discussions, there was any contemplation of -- for patients who may be on other premium priced drugs for DMD and then may be also going on deramiocel or even vice versa. Just wondering given the expense burden of having two premium priced drugs for this condition. How are the payers viewing that if you have any intelligence there?

Yes. So we've had really good feedback, Leland, from the payers so far. There are several reasons why I think this is going to be a great opportunity for reimbursement. Number one, and first and foremost, it is the only therapeutic that it will be treating the Duchenne cardiomyopathy. This cannot be highlighted enough. You are talking about a disease where not only is cardiac disease one of the primary features of the pathophysiology, but also has been indicated to be more and more important as potentially treatment for the dystrophinopathy could become more relevant such as a gene therapy. We see long-term benefit. Year-over-year, we've seen three years of improvement or stabilization in cardiac as well as skeletal muscle function, which we've shown not only to the FDA, but are also presenting to the payers. So overall, we think that, when they have their sort of pharmacopeia to pick from in treating Duchenne, this will be a great opportunity to provide therapeutic benefits to an area that has largely been unaddressed, can reduce hospitalization and mortality and will go along with any of the other treatments for the dystrophinopathies.

All right. Thank you. And then a question just sort of longer term, you've got a healthy cash balance. You're going to have the $80 million presumably on the FDA approval and then that and the PRV which you could monetize and that doesn't even include other terms with NS with Japan and potentially Europe. Just curious given that the operating expense for Capricor will remain relatively lean given that you wouldn't have to be building your own sales force and getting into commercial infrastructure yourselves. Any thoughts on how you may invest that capital? Obviously, exosomes are very promising, but given the stage of that development, it doesn't really seem like it would be expensive to be running those initial studies. Just wondering if you have any thoughts on where to apply the much greater balance sheet that you may be coming into? Thanks.

Yes. Thank you, Leland. We really appreciate the question. And it's actually been an area of exploration within the company right now. We're looking at all types of opportunities, not only in terms of label extension for skeletal muscle myopathy of Duchenne and also Becker, which can further enhance our balance sheet as we move forward. Yes, we are focusing on our exosome pipeline. I talked a little bit about that. It is farther behind deramiocel. And we'll be keeping you and The Street updated as we evaluate the opportunity to move this company forward, which we think will be plenty over the next few years.

All right. Thanks for your color.

Thanks, Leland.

Thank you. And your next question comes from the line of Joe Pantginis from H.C. Wainwright. Please go ahead.

Hey, Linda and A.J., thanks for taking the questions. So first, maybe for A.J, I mean, this is fantastic news also that Linda you just announced about the new expanded 25,000 square foot facility. Are you able to sort of define the costs and timeframe for this facility?

Yes. Thanks, Joe. Yes, we are excited about the expansion. It's in our current footprint here in San Diego, basically the floor above where we're already operating and we're kicking off all the planning right as we speak to expand out. We haven't put out the formal estimate, but I will say we built our original clean room, our commercial qualified clean room here in San Diego for just under a couple of million dollars. So we have the plans in place. We have the team in place. We already have the construction operations underway. So we envision that we can do this to a very reasonable number and we'll put more of that out there in the coming months as we move ahead.

Got it. And then I guess second question is obviously you have no indication that there's going to be an AdCom right now. When do you think you might hear an answer? And for example, if there were an AdCom, would that be a place that they might want to not necessarily would require, but maybe force a discussion regarding HOPE-3?

Yes. Thanks, Joe. Yes. So we're waiting every day to hear from them on whether they would want an AdCom. They will need some time to put it together. And even though we're actively prepping for one as we speak, they have to give us time to prepare as well. So we expect to hear soon. I think part of the delay is just based on some of the turmoil that's going on in the government right now. And so I expect that things are moving at a different pace than they might have even just a few months ago. So stay tuned. When we know, we will let everybody else know. We see an AdCom neither as a benefit nor a risk. We believe very strongly in our application. We have clinically and statistically significant data. The data stands on its own. However, if we need to get up there and talk about it, we will absolutely do that. In terms of HOPE-3, what they have told us is that they are not considering HOPE-3 for this Biologic License Application that they understand that the primary efficacy endpoint of HOPE-3 is skeletal muscle that would be used for post approval label extension. We plan on taking HOPE-3 potentially outside the US to order expand our global footprint. And the focus of this application as we and they understand it is the data that we've talked about, which is the HOPE-2 data, the HOPE-2 open label extension data compared to the natural history data set from the Cardiac Consortium. And so I don't anticipate a discussion of HOPE-3 at an AdCom, but if it comes up, we'll be ready to take those questions as well.

Also very helpful and thanks for the repetition there. Quickly just on the discussion about cash usage going forward, obviously, you talked about some of the obvious things, since you guys really have a specialty in say cellular therapy here, would you consider in-licensing, number one? And then second, with regard to Europe, I know you can't really or can't necessarily describe the first part of the question, but what are the outstanding sort of things that you're still needing or the open questions with regard to potential signed NS partnership, and then also what are the outstanding questions you feel are important for your EMA discussions?

Yes. Thanks, Joe. That's a multi-pronged question. So in terms of in-licensing, we're evaluating opportunities as they become available. Right now, we are laser focusing on getting deramiocel approved and getting that ready to launch. That's going to obviously drive our cash value and our abilities to look at new opportunities. I will say this, we are experts now, in translational medicine and bringing things sort of from the colloquial bench to now commercial. So we would not rule out the right opportunity should it be presented to us. Having said that, shifting, to your second question about, the EMA. What we're doing and what we're really focusing on now is the ability to get deramiocel to Europe. We are working right now with EMA and with consultants outside the US in order to build that program. We will be meeting with the EMA hopefully, in the second quarter of this year to further understand what their requirements are for approval of deramiocel. Because we are working directly with Europe right now and because we are seeing some success by getting designations in Europe of ATMP and working, with EMA, we're slowing down a little bit our conversations to make sure that we have the best path forward for deramiocel in Europe. We are still negotiating with NS and have an active conversation going in terms of the definitive agreement and really feel very positive about their commitment to the therapeutic.

Really appreciate all the color. Thanks a lot.

Thanks, Joe. Take good care.

Thank you. And your next question comes from the line of Kristen Kluska from Cantor. Please go ahead.

Hi, everyone. Congrats on the data this week and the recent filing acceptance. So I know that your mechanism is complementary to a gene therapy, but after yesterday's unfortunate news regarding the patient on Elevidys, we are hearing doctors emphasize the need for treatment options for non-ambulatory patients in particular. So can you just remind us what percent of the later stage population in particular would have cardiomyopathy? And then while label extension could come later, could you reference some of your poll data if you have an approval as perhaps supportive evidence? Thank you.

Yes. Thanks, Kristen. Yes. I think the entire community is reeling from the death of that young man. It's a small patient community, and so, the loss of one life is tragic always, but especially in a community such as this. Most of our patients that we've treated have been non-ambulance. They are primarily the ones that have the cardiomyopathy manifestations, although we've been messaging that the scar that causes the ultimate decline in cardiac function is there when they are little. So we're going to try and get it as young as possible. But, yes, most of our patients are the later stage non-ambulant. The focus of this application is the cardiomyopathy. But as you just correctly stated, we have shown year-over-year improvement in performance of the upper limb in our open label extension guides and all of those guys are non-ambulant. So we do believe that deramiocel is, has been, and will continue to be a very good option for those that are later stages, non-ambulant of the disease and look forward to taking that across the line as well in the future.

Thank you for that. And then another question, I know you have robust safety data as well. But as we start to think about the potential to move to commercialization where there will be the potential for more to get treatment at once. Is there anything we should consider from a safety protocol? Will physicians want to treat a few patients first or will doctors require a more extensive testing? And the reason why I ask is sometimes like we are seeing with the gene therapy that there's a plethora of different specialists that need to be involved because of some of the known safety risks of that.

Yes. Thanks. So I think the Duchenne community and I've been the beneficiary of participating in many of this symposia, have been talking about the care teams necessary for gene therapy for several years. The gene therapies are very complicated because they use viral vectors. And as you know, the immune system doesn't usually like it when a virus enters the system and so you have to do a lot of preplanning and pretreatment to mitigate that immune response. In terms of deramiocel, it's a cell therapy. We are not doing any genetic manipulation of it. We've performed over 700 infusions and not seen any serious safety events since the early stage hypersensitivity, which has been mitigated by pretreatment with antihistamines and steroids. And the abundance of caution, we will do our infusions at infusion centers or in care centers should a hypersensitivity response ever occur. We don't anticipating needing a care team with a very simple infusion, and where the side effects, which are mild and sort of a little bit of mild flu like symptoms in some of our patients are easily mitigated and understood by our investigators. So I don't think it's going to have the same complicated introduction or care paradigm needed as a gene therapy.

Thanks. And last question from me. I think you commented that with the additional manufacturing footprint, this could support 2,000 to 3,000 patients potentially as early as mid-2026. But should you get an approval later this year, how should we be thinking about, what you can essentially handle until that time? Because I know you said there's already some patients that want to transfer on OLE as well as others that weren't involved in the trial that are aware of the potential of the drug. Thanks again, everyone.

Yes. Thanks, Kristen. Yes. So our manufacturing facility here in San Diego, which we built in anticipation of commercial launch can service between 250 to 500 patients per year. If you actually look at the timeline of launch, we're looking, all things considered at a Q4 launch this year. By the time the new facility comes on, it won't even be a full year into commercialization. And so we believe that we'll have enough from our small scale commercial facility in San Diego to meet early demand and then be able to swing right away into the new facility. As we mentioned, but I'll highlight, the new facility is actually within the footprint of our currently existing manufacturing plant. And so we're hopeful that the regulatory, obligations required to bring that facility online will be light and therefore we'll be able to get it on board as soon as possible after launch.

Thank you so much again.

Thanks.

Thank you. And your next question comes from the line of Catherine Novack from JonesTrading. Please go ahead.

Hi. Good afternoon, everyone. Thanks for taking my question. I'm just wondering if you can talk a little bit about, in the past, FDA had made comments about the issues of single arm studies in DMD, due to the disease heterogeneity. Can you give us some color about, in your discussions with FDA, what have they brought up regarding their view on cardiac outcomes shown in HOPE-2 OLE? Why this might be, why they might take a different approach on this? Thanks.

So I think what you're asking is because the HOPE-2 open label extension study is not placebo-controlled, how is FDA looking at that data? Am I understanding that correctly?

Correct.

Okay. Yes. So there's a couple of very important points there and thanks so much for calling in. Number one is, we have had access to propensity matched age function medication matched set of patients from the Vanderbilt Natural History, Cardiac Consortium Study, funded by the FDA to be able to use as an indicator of what would be considered a placebo group, right, a group that has been untreated. And we can therefore look at the natural history of the cardiomyopathy compared to the open label extension guys in a year-over-year manner. Typically, this would be an issue if you were doing a type of volitional measurement like a NorthStar Ambulatory Assessment, a time to rise, or in our case, a performance of the upper limb because one might say, well, if you know you're getting treated, you're going to maybe function or perform better. The beauty of this dataset and the reason the FDA has been willing to look at it so carefully is because nothing about cardiac MRI is volitional. You cannot wish your heart better. You cannot wake up and think I'm going to get a cellular therapy, and therefore, my heart's going to function better. So even though the dataset's relatively small, it's very concise, in its ability to measure function. Our strong statistical significance is emblematic of the strong treatment effect that we are seeing. That means that very little opportunity for this data to be up to chance. And so as a result of that, we remain confident that this is not a typical single arm study, but one that actually has a natural history, real world evidence component that supports the data.

Got it. Thanks. Thanks so much.

Thank you.

Thank you. And your next question comes from the line of Aydin Huseynov from Ladenburg. Please go ahead.

Hi. Good afternoon, everyone. Congratulations for the progress this quarter. So couple of questions from us. So Linda I think you mentioned heart diseases were or deramiocel might be helpful and obviously the next indication, the next sort of low hanging fruit is Becker muscular dystrophy, it's in your corporate presentation. So when do you think we will have updates on the potential Becker muscular dystrophy trial design and whether it is going to look like HOPE-2? So could you give us a little bit of insights in terms of what is your sort of clinical strategy as it comes to Becker muscular dystrophy?

Thanks, Aydin. Yes, it's funny. As I was putting together my remarks, today, I was thinking a little bit about you and I knew that you'd have come to me with the Becker question. So we're working with the agency now on some of the initial steps of getting our program going with Becker. We've submitted some request to them recently. Our focus is to get deramiocel approved for Duchenne and then begin our emphasis on building towards Becker for which, by the way, we hold all the economics. NS has no rights to those. Our goal will be to convince the agency that the cardiomyopathy associated with Becker looks and functionally is the same as the Duchenne cardiomyopathy and see if we can potentially move towards an accelerated pathway in Becker. We are bringing in key opinion leaders literally as we speak and medical expertise to Capricor to help us build out our Becker program and I expect to have more color for that program over the next quarter or two.

Okay. Understood. Thank you. Thank you for the update. And what are other heart diseases where you think deramiocel can make a meaningful difference? I'm just trying to understand the whole sort of commercial potential for deramiocel beyond DMD and beyond BMD.

Well, look, this is an area of great interest to Capricor right now. We have a very powerful therapeutic, a defined mechanism of action, which is immunomodulatory that drives anti-fibrotic activity. We're evaluating orphan cardiomyopathies, looking at where we might best deploy them. I think many of the physicians in the colloquial room, including yourself, have ideas of where that could be. And we'll provide some updates on pipeline expansion into other orphan cardiomyopathies as we begin to evaluate them and determine that would be the best path forward for Duchenne for deramiocel.

Okay. Thank you. Thanks so much.

Yes. Thank you. Have a great day.

Thank you. And your last question comes from the line of Madison El-Saadi from B. Riley Securities. Please go ahead.

Hey, Linda and A.J. Thank you for taking our question and congrats on the progress. I'm coming away from MDA, so my questions may be a bit academic, so forgive me. So based on what you've seen through the course of deramiocel development, what do you think is the ideal baseline ejection fraction baseline where impact will be the highest? Is it in the for example less than 35% range or is it more than 35% to 45% range? And then secondly, I believe you mentioned that you expect 7,500 boys to be eligible. Would this be based on a hard ejection fraction threshold? And I ask given that cardiac treatment in this population is often the result of shared decision making. Thanks.

Yes. Madison, thank you so much and hope you enjoyed the MDA. So what we understand about the cutoff for ejection fraction is that if we can get in there early, we believe that we will see the greatest long-term benefit in terms of stabilization of disease progression. So in the patients with ejection fractions of 45% or greater we see sort of the greatest amount of long-term stabilization and some of our patients haven't seen any decline in ejection fraction literally in years. And that is because we believe that once you have the immunomodulation anti-fibrotic you're actually preserving the healthy heart tissue that does exist. Having said that, we have patients that have had lower ejection fractions where we're also seeing stabilization of the disease process. What I can say sort of overall is that early treatment is better because you have a longer opportunity with or a better opportunity, not necessarily longer, but a better opportunity to treat the disease process early, preserve cardiac function, and also, obviously, then hopefully sustain life and, not only quantity, but quality of life. So we're still open to all comers in terms of ejection fractions. We do know that once they get below a certain point, once they get to what would be considered an end state heart failure, it's harder and harder to bring them back from that edge. And so that's what we're messaging to the agency as well as to physician leaders that we need to get in there early. And, yes, it will be a decision of the care providers. We're working now very actively with the cardiologists that treat Duchenne patients, both pediatric and adult, as well as the neurologists, so that they become educated and open to the idea of using deramiocel as part of the treatment paradigm for DMD cardiomyopathy.

Understood. Thanks.

Thank you for your question.

Thank you. I will now turn the call back to Capricor management for final thoughts.

Yes. Thank you for joining today's call. We look forward to updating you on our continued progress over the coming months and I hope you have a nice evening.