Capricor Therapeutics - Earnings Call - Q4 2024

Executive Summary

• Q4 2024 delivered a revenue and EPS beat versus S&P Global consensus, driven by milestone recognition and steady ratable revenue from the Nippon Shinyaku agreement; revenue was $11.13M and diluted EPS was -$0.16, both ahead of consensus. Estimates marked with * retrieved from S&P Global.*
• The FDA accepted CAPR’s BLA for deramiocel with Priority Review and set a PDUFA target action date of August 31, 2025, sharpening the regulatory catalyst path laid out in Q3 (previously “2H25”).
• Cash, cash equivalents and marketable securities ended Q4 at $151.5M; with a $10M post-quarter milestone receipt and expected $80M on approval plus potential PRV monetization, management underscored runway into 2027 and robust financing options for commercialization and pipeline expansion.
• Manufacturing capacity expansion (additional 25,000 sq ft) targets 2,000–3,000 patients per year by mid-2026, supporting anticipated launch demand; current San Diego facility supports ~250–500 patients per year for early commercialization.
• Near-term stock reaction catalysts: AdCom determination (if any), PLI inspection timing, EMA engagement (with Orphan + ATMP designations), and Project NextGen milestones for StealthX vaccine; core narrative centers on first-in-class treatment for DMD cardiomyopathy and reimbursement readiness with NS Pharma.

What Went Well and What Went Wrong

• What Went Well

  • BLA accepted with Priority Review; PDUFA set for 8/31/2025, solidifying a clear regulatory timeline. Quote: “We continue to work diligently towards our August 31, 2025 action date…preparing for pre-approval licensure inspection and potential commercial launch with Nippon Shinyaku”.
  • Q4 revenue/EPS beat vs consensus, aided by recognition of the $10M second development milestone and ratable revenue from the NS agreement. Quote: “Revenues for the fourth quarter of 2024 were approximately $11.1 million… ratable recognition of the $40.0 million… and the recognition of the $10.0 million second development milestone payment”.
  • Strengthened balance sheet and expansion plan: $151.5M year-end cash; pro forma ~$161.5M after $10M milestone; capacity expansion to 2,000–3,000 patients by mid-2026.

• What Went Wrong

  • Operating expenses rose materially YoY as CAPR accelerated R&D and commercialization build-out: Q4 total OpEx $18.8M vs $13.4M in Q4 2023, expanding losses.
  • Q3 showed a notable revenue shortfall versus consensus ($2.26M actual vs ~$3.57M est) and a larger EPS loss (-$0.38 actual vs -$0.358 est), highlighting quarter-to-quarter variability prior to Q4 momentum. Estimates marked with * retrieved from S&P Global.*
  • Q2 cash runway had been guided only into Q1 2025 before subsequent capital raises and milestone timing extended runway; this earlier constraint underscored dependence on external financing and milestones prior to strengthening the balance sheet later in the year.

Transcript

Operator (participant)

Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics Second 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. If at any time during this call you require immediate assistance, please press * zero for the operator. 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.

A.J. Bergmann (CFO)

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 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. We are cautioned not to place undue reliance on these forward-looking statements, and we disclaim any obligation to update such statements. With that, I'll turn the call over to Linda Marbán, CEO.

Linda Marbán (CEO)

Thanks, A.J. Bergmann. 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 deramyocel, 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 deramyocel have 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. Theromyocyte 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, deramyocel have 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 deramyocel as a treatment for those impacted by Duchenne muscular dystrophy. 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, CMC, 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 the FDA for priority review. The application is seeking full, not accelerated, approval of deramyocel 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 set for August 31, 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 pre-licensing 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 II, 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. Therefore, because we see an impact on both cardiac and skeletal muscle function, we are confident that we are seeing a treatment effect of deramyocel across multiple domains, further strengthening the opportunity to treat DMD with deramyocel. 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 deramyocel is warranted for the treatment of DMD. We also have continued to work in close collaboration with the FDA. We have been the beneficiaries.

CBER 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 deramyocel for DMD cardiomyopathy. In addition, our RMAT designation allows us to work directly with the 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 the 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 expansion. 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 deramyocel in the United States.

We recently completed surveys with the top five payers in the U.S., with the response coming in very favorably from a reimbursement standpoint for deramyocel. 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 deramyocel 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 50-60% 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 deramyocel. 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 U.S., we are entitled to between 30-50% of revenue share based on sales of the product, inclusive of cost of goods sold. I'd like to shift our attention to CMC, or Chemistry, Manufacturing and Controls. Our current GMP compliance facility located in San Diego is fully operational and staffed, producing doses of deramyocel. We built and carefully designed this facility to meet early market demand. The fully operational capacity in this facility can support approximately 250-500 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 deramyocel 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 sq ft, for which we are going to build additional clean rooms, taking our manufacturing capacity to approximately 2,000-3,000 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 sheets.

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 deramyocel 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 deramyocel in the European region, subject to finalization of a definitive agreement. Our commitment to Nippon Shinyaku in the U.S.A. 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 the 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 Stealth-X 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 deramyocel, 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 coded on an exosome that could be made rapidly within the 100-day constraints developed by the U.S., government, but also able to generate a robust and long-lasting immune response. That program is part of the U.S., government's Project NextGen, which aims to test vaccine candidates for COVID-19 prevention in addition to prepare for future pandemics. Currently, our Stealth-X vaccine candidate is in the manufacturing phase.

The NIAID, which is the National Institute of Allergy and Infectious Diseases, will then conduct and fully fund a phase I clinical trial. Currently, manufacturing is underway for our Stealth-X 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 deramyocel are on a defined path towards potential commercialization, we are further evaluating the path forward for a 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 and grateful to the patients, their families, and 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 deramyocel 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. Bergmann to run through our financials. A.J. Bergmann?

A.J. Bergmann (CFO)

Thanks, Linda Marbán. 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 31, 2024, our cash, cash equivalents, and marketable securities totaled approximately $151.5 million.

Subsequent to December 31, 2024, we received a $10 million milestone payment from Nippon Shinyaku pursuant to the terms of our U.S. distribution agreement. On a pro forma basis, our cash, cash equivalents, and 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 U.S. 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.

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 of 2024 was approximately $7.1 million compared to a net loss of approximately $800,000 for the fourth quarter of 2023. The net loss for the year ended December 31, 2024, was approximately $40.5 million compared to a net loss of approximately $22.3 million for the year ended December 31, 2023. We will now open the lineup for questions. Operator?

Operator (participant)

Thank you. Ladies and gentlemen, we will now begin the question and answer session. Should you have a question, please press *4 or press 1 on your telephone keypad. Should you wish to cancel your request, please press *4 or press 2. If you are using a speakerphone, please lift the handset before pressing any keys. One moment, please, for your first question.

Your first question comes from the line of Ed Tenthoff from Piper Sandler. Please go ahead.

Edward Tenthoff (Analyst)

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 deramyocel, what additional color can you tell us about the prep and sort of the division of labor between you and Nippon Shinyaku? How much of a benefit will it be with their existing commercial infrastructure? Thanks.

Linda Marbán (CEO)

Thanks, Ed. Yeah, great to hear from you as always. This has been a really exciting time for Capricor, and obviously, NS is very excited as well. We just keep hitting our milestones as we told them we would, and they are in full-scale preparation for commercial launch. They have approximately 125 employees in the U.S.

that have been dedicated to their Viltepso franchise, so they're well ensconced in the Duchenne space. They're spending a significant amount of their time now focusing on getting deramyocel 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 that's 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 deramyocel to market. Their role is to sell it and distribute it. Obviously, part of selling a biologic like deramyocel is a deep understanding of the product, which we are providing to them.

We're very, very excited about their energy and also working very closely with them on launch.

Edward Tenthoff (Analyst)

Great. Thank you very much.

Linda Marbán (CEO)

Yep. Take care, Edward Tenthoff.

Operator (participant)

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

Leland Gershell (Analyst)

Hi, good afternoon. Great to hear all the progress, and we're looking forward to events coming later this year for Capricor. There's a few questions from us. First, Linda Marbán, 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 deramyocel 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?

Linda Marbán (CEO)

Yeah, so we've had really good feedback, Leland Gershell, from the payers so far. There's 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 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.

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.

Leland Gershell (Analyst)

All right. Thank you. 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 the PRV, which you could monetize. That does not 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 would not 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.

Linda Marbán (CEO)

Yeah, thank you, Leland Gershell. 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 expansion 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 deramyocel.

We will be keeping you and the street updated as we evaluate the opportunities to move this company forward, which we think will be plenty over the next few years.

Leland Gershell (Analyst)

All right. Thanks, Leland Gershell.

Linda Marbán (CEO)

Thanks, Leland Gershell.

Operator (participant)

Thank you. Your next question comes from the line of Joseph Pantginis from H.C. Wainwright. Please go ahead.

Joseph Pantginis (Analyst)

Hey, Leland Gershell and A.J. Bergmann. Thanks for taking the questions. First, maybe for A.J. Bergmann, I mean, this is fantastic news also that, Linda Marbán, 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?

A.J. Bergmann (CFO)

Yeah. Thanks, Joseph Pantginis. Yeah, we are excited about the expansion. It is in our current footprint here in San Diego, basically the floor above where we are already operating. We are kicking off all the planning right as we speak to expand out.

We have not put out the formal estimates, 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. We have the plans in place. We have the team in place. We already have the construction operations underway. We envision that we can do this to a very reasonable number, and we will put more of that out there in the coming months as we move ahead.

Joseph Pantginis (Analyst)

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

Linda Marbán (CEO)

Yeah. Thanks, Joseph Pantginis. Yeah.

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. Even though we're actively prepping for one as we speak, they have to give us time to prepare as well. 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. I expect that things are moving at a different pace than they might have even just a few months ago. 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 biologics license application, that they understand that the primary efficacy endpoint of HOPE-3 is skeletal muscle, that that would be used for post-approval label expansion. We plan on taking HOPE-3 potentially outside the U.S. to expand our global footprint. 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 sets from the cardiac consortium. 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.

Joseph Pantginis (Analyst)

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? 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? Also, what are the outstanding questions you feel are important for your EMEA discussions?

Linda Marbán (CEO)

Yeah. Thanks, Joseph Pantginis. That's a multi-pronged question. In terms of in-licensing, we're evaluating opportunities as they become available. Right now, we are laser-focusing on getting deramyocel 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. 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 deramyocel to Europe. We are working right now with EMA and with consultants outside the U.S. in order to build that program. We will be meeting with EMA, hopefully, in the second quarter of this year to further understand what their requirements are for approval of deramyocel.

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 deramyocel 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.

Joseph Pantginis (Analyst)

Really appreciate all the color. Thanks a lot.

Linda Marbán (CEO)

Thanks, Joseph Pantginis. Take good care.

Operator (participant)

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

Kristen Kluska (Analyst)

Hi, everyone. Congrats on the data this week and the recent filing acceptance.

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. Can you just remind us what % of the later-stage population in particular would have cardiomyopathy? While label expansion could come later, could you reference some of your poll data if you have an approval as perhaps supportive evidence? Thank you.

Linda Marbán (CEO)

Yeah. Thanks, Kristen. Yeah, I think the entire community is reeling from the death of that young man. It's a small patient community, and 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-ambulatory.

They are primarily the ones that have the cardiomyopathy manifestations, although we have been messaging that the scar that causes the ultimate decline in cardiac function is there when they are little. We are going to try and get in as young as possible. Yes, most of our patients are the later-stage non-ambulant. The focus of this application is the cardiomyopathy, as you just correctly stated, we have shown year-over-year improvement in performance of the upper limb in our open label extension guides. All of those guides are non-ambulant. We do believe that deramyocel has been and will continue to be a very good option for those that are at later stages non-ambulant of the disease and look forward to taking that across the line as well in the future.

Kristen Kluska (Analyst)

Thank you for that. 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? The reason why I ask is sometimes 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.

Linda Marbán (CEO)

Yeah. Thanks. I think the Duchenne community, and I've been the beneficiary of participating in many of these 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.

As you know, the immune system doesn't usually like it when a virus enters the system. You have to do a lot of pre-planning and pre-treatment to mitigate that immune response. In terms of deramyocel, 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 pre-treatment with antihistamines and steroids. Out of an abundance of caution, we will do our infusions at infusion centers or in care centers should a hypersensitivity response ever occur. We don't anticipate needing a care team. It's a very simple infusion. 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.

I do not think it is going to have the same complicated introduction or care paradigm needed as a gene therapy.

Kristen Kluska (Analyst)

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

Linda Marbán (CEO)

Yeah. Thanks, Kristen. Our manufacturing facility here in San Diego, which we built in anticipation of commercial launch, can service between 250-500 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. 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. 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.

Kristen Kluska (Analyst)

Thank you so much again.

Linda Marbán (CEO)

Thanks.

Operator (participant)

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

Catherine Novack (Analyst)

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.

Linda Marbán (CEO)

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?

Catherine Novack (Analyst)

Correct.

Linda Marbán (CEO)

Okay. Yeah. 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. We can therefore look at the natural history of the cardiomyopathy compared to the open label extension guides in a year-over-year manner.

Typically, this would be an issue if you were doing a type of volitional measurement like a North Star Ambulatory Assessment of 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 know you're going to maybe function or perform better." The beauty of this data set 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." Even though the data set'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. 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.

Catherine Novack (Analyst)

Got it. Thanks. Thanks so much.

Linda Marbán (CEO)

Thank you.

Operator (participant)

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

Aydin Huseynov (Analyst)

Hi. Good afternoon, everyone. Congratulations on the progress this quarter. A couple of questions from us. Linda Marbán, I think you mentioned heart diseases where deramyocel might be helpful. Obviously, the next indication, the next sort of low-hanging fruit is Becker muscular dystrophy. It's in your corporate presentation. 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?

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?

Linda Marbán (CEO)

Thanks, Eden. Yeah. As funny as I was putting together my remarks today, I was thinking a little bit about you, and I knew that you'd come to me with the Becker question. We're working with the agency now on some of the initial steps of getting our program going with Becker. We've submitted some requests to them recently. Our focus is to get deramyocel 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. I expect to have more color for that program over the next quarter or two.

Aydin Huseynov (Analyst)

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

Linda Marbán (CEO)

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. We'll provide some updates on pipeline expansion into other orphan cardiomyopathies as we begin to evaluate them and determine that that would be the best path forward for deramyocel.

Aydin Huseynov (Analyst)

Thank you. Thanks so much.

Linda Marbán (CEO)

Yep. Thank you. Have a great day.

Operator (participant)

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

Madison El-Saadi (Analyst)

Hey, Linda Marbán and A.J. Bergmann, 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.

Based on what you've seen through the course of deramyocel 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 the 35-45% range? Secondly, I believe you mentioned that you expect 7,500 boys to be eligible. Would this be based on a hard ejection fraction threshold? I ask, given that cardiac treatment in this population is often the result of shared decision-making. Thanks.

Linda Marbán (CEO)

Yeah, Madison, thank you so much, and hope you enjoyed the MDA. 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.

In the patients with ejection fractions of 45% or greater, we see sort of the greatest amount of long-term stabilization. Some of our patients have not seen any decline in ejection fraction literally in years. That is because we believe that once you have the immunomodulation, anti-fibrotic, you are actually preserving the healthy heart tissue that does exist. Having said that, we have patients that have had lower ejection fractions where we are 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. We are 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-stage heart failure, it's harder and harder to bring them back from that edge. That is what we're messaging to the agency as well as to physician leaders that we need to get in there early. 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 deramyocel as part of the treatment paradigm for DMD cardiomyopathy.

Madison El-Saadi (Analyst)

Understood. Thanks.

Linda Marbán (CEO)

Thank you for your question.

Operator (participant)

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

Linda Marbán (CEO)

Yeah. 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.