Q3 2025 Capricor Therapeutics Inc Earnings Call

Good afternoon, ladies and gentlemen, and welcome to the Capricorn Therapeutics third quarter 2025 conference call.

At this time, all participants are in listening-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 *0 for the operator.

this call is being recorded on Monday, November 10th, 2025

I would now like to turn the conference over to our CFO, AJ Burgman, for the report. Good-looking statement. Please go ahead.

Thank you very much 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 pre-clinical and clinical stuff.

Studies, our enrollment of patients and our clinical studies, plans to present a report additional data plans regarding regulatory filings potential regulatory developments involving our product candidates potential regulatory, inspections, revenue and reimbursement, estimates projected. Terms of definitive agreements manufacturing capabilities potential. Milestone payments are financial position are possible uses of existing cash and investment resources.

These 4 looking statements are based on current information assumptions and expectations that our subject to change and evolve a number of risk and uncertainties that may cause our 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 and or reports your caution not to place undue Reliance on these forward-looking statements. And we disclaim any obligation to update such statements with that. Turn the call over to Linda Mar band CEO.

Good afternoon, and thank you for joining us on Capricor's third quarter 2025 conference call.

This has been a very busy time for Capricorn, as we are just weeks away from a major milestone. The top-line readout from our HOPE 3 Phase 3 clinical study of Jerusha cells, an investigational cell therapy for the treatment of Duchenne muscular dystrophy.

This, pivotal study represents the culmination of nearly a decade of scientific development, all aimed at helping, boys and young. Men, living with this devastating disease importantly, hope 3 focuses. Primarily on non ambulance individuals. A patient population that has historically had limited clinical research dedicated to it.

Function as measured by the performance of the Upper Limb version 2.0 and cardiac function as measured by left ventricular. Ejection fraction measured by cardiac MRI as well as several secondary and exploratory end points.

These 105 patients enrolled in. Hope 3 represent 2 cohorts.

Cohort A, which received the product manufactured at our Los Angeles clinical facility, and Cohort B, which received the product manufactured at our commercial GMP facility in San Diego,

As a reminder, the FDA required, the addition of cohort B, to evaluate the efficacy of the commercial scale product.

While we have demonstrated non-clinical comparability cohort, B provides, the opportunity to generate direct evidence of efficacy. For the commercial material, the San Diego facility was built to meet commercial manufacturing standards operating under elevated quality and compliance requirements to support commercial Jeremiah cell production.

The statistical analysis plan for Hope.3 includes analyses designed to evaluate efficacy, both across the combined cohorts and independently within Cohort B.

We believe in alignment with our biostatisticians and clinicians who designed our statistical analysis plan with us. That while the aggregated data are informative demonstrating efficacy of the commercial scale product represents, the most direct regulatory path to potential approval.

Jeremiah fell has been shown to help, preserve both cardiac and skeletal muscle function. And our goal will be to empathize to the FDA the life-limiting cardiovascular impact of this disease.

We are now in the final stages of data preparation. Our statistical analysis plan has been submitted to the FDA, and the comment period passed without additional feedback. We plan to unblind the study once all data management processes are finalized, which, as noted, will occur within the next several weeks.

The process has required review of more than 300 MRI by independent external readers who are fully blinded, both to treatment allocation and sequence. A process that requires additional time to collect and analyze the data set.

To remind you after our preb meeting with the FDA in. 2024, we submitted a bla based on existing data from our hope to and the hope to open label extension trials compared to an external control comparator from the cardiac Consortium at that time. The purpose of Hope 3 was to support potential X us expansion as well as label expansion. However, following receipt of the crl in July, the role of Hope 3 shifted.

The crl.

Primarily cited the need for additional substantial evidence of effectiveness and certain CMC. Clarifications importantly, most of the CMC issues had already been addressed in prior information request responses, and the remainder were resolved shortly after the receipt of the CRL.

While the crl was unexpected, we were well positioned with hope 3 to provide the additional safety and efficacy data requested by the FDA

During our type, a meeting in August of this year, the FDA indicated that the Hope 3 results could be submitted to address the issues raised in the crl. A key element of that meeting, was our request to keep the current bla open and maintain the indication for DMD Associated cardiomyopathy to advance that path. We proposed designating left ventricular, ejection fraction lvef as the primary efficacy endpoint. While the FDA did not allow this formal change. They agreed to exercise regulatory flexibility in reviewing the Hope 3 data.

As of now, fga has classified. The resubmission as type 2, which means the review period can be up to 6 months, but there is precedent for faster. Review times, we will make every effort to advance Jeremiah felt toward approval as efficiently as possible in 2026.

To remind you. We are eligible to receive a priority review. Voucher, if approved is a, if approval is attained prior to September 30th, 2026 and prvs, may become increasingly valuable as the program approaches, its statutory sunset.

This consistency demonstrated across multiple clinical studies, underscores Jeremiah's cell's potential to stabilize disease, progression and preserve both muscle and heart function. We now look forward to seeing whether the data from Hope 3 confirms these benefits in a larger rigorously controlled pivotal trial.

As we approach our quiet period. I will remind you that we expect to report Topline data within the next few weeks. And we will do everything we can to keep both the market and the DMD Community, informed of our further plans with respect to this program and the release of the data.

We also recently published a peer-reviewed paper and biome medicines detailing new mechanistic insights into Deer masels mechanism of action. The study described in the paper demonstrated that cardio fear. Derived cells cdc's the active component of Jeremiah release, exosomes and soluble factors that suppressed by broader gene expression, collagen, 1, and collagen 3, and human fibroblasts,

These findings were consistent across more than 100 manufacturing sites, further validating that Jeremiah sells antivirus and immunomodulatory properties. This evidence further supports its mechanism of action.

To complement this publication, we also released a scientific video illustrating Jeremiah's mechanism of action, which is available on our website. This video reinforces the biological rationale and consistency that underlie our entire development program for Jeremy My Cell.

Now, focusing for a moment on the CMC front.

Following acceptance by the FDA of all findings from our pre-licensed inspection (PLI), our San Diego commercial facility is fully operational and preparing for GMP production activities. Our manufacturing and quality systems are fully implemented, and all CMC-related items cited in the CRL have been addressed. This achievement reflects the strength of our operations and represents a critical milestone in ensuring readiness for commercialization and long-term product consistency.

In parallel, we continue to prepare for launch with advancing initiatives in physician education, patient services, market access, and reimbursement. We are engaging both neurology and cardiology specialists to ensure an integrated approach to patient care, should DARE MyCell receive approval.

While our immediate Focus remains on us approval, we are also laying the groundwork for potential Global expansion and will share updates as appropriate. We are closely monitoring evolving us and international pricing policies, including the current Administration stands on most favored nation Frameworks and will adapt our Global strategy accordingly.

We continue to advance our stealth X Program under Project NextGen, a U.S. government-funded initiative led by HHS and the National Institutes of Allergy and Infectious Disease, to develop next-generation vaccines for COVID-19 and other potential infectious threats.

Additional plant arms, that will utilize a multi-value. We expect initial data in the first quarter of 2026 subjects to completion of the trial by niaid. The goal is to validate stealth X. As a versatile non-mrna addivon, free platform capable of delivering native proteins, safely and efficiently, a model that could potentially extend to infections and rare diseases alike.

While vaccines are not our Core Business, this program serves as a critical proof of concept for the stealth X platform positive results. Could open the door to strategic collaborations and highlight the platform's potential for Target. Therapeutic delivery. Well beyond vaccinology.

With that, I will now turn the call over to AJ to run through the financials AJ.

Thanks Linda. This afternoon's press release. Provided a summary of our third quarter 2025 financials on a gap basis and you may also refer to our quarterly report on form 10q, which we expect to become available shortly and we'll be accessible on the FCC website, as well as the financial section of our website.

Let me start with our cash position.

As noted as of September 30th, 2025 our cash cash equivalent to Market will Securities totaled, approximately 98.6 million. We believe that based on our current operating plan and financial resources are available Cash, Cash equivalents and marketable, securities will be sufficient to cover anticipated expenses and capital requirements into the fourth quarter of 2026.

Earning briefly to the financials revenue for the third quarter of 25 or 0 compared to approximately 2.3 million for the third quarter of 2024, additionally revenue for the first 3 quarters of 2025 were zero compared to approximately 11.1 million for the first 3 quarters of 2024. I'd like to point out that the source of Revenue in 2024 was the rateable recognition of the 40 million dollars. We have received under our Us distribution agreement with Nippon shinyaku, which had been fully recognized as of December. 31st 2024

Turning to our operating expenses for the third quarter of 2025, excluding stock-based compensation, our research and development expense was approximately $18.1 million compared to approximately $11 million in Q3 2024. For the first three quarters of 2025, excluding stock-based compensation, our research and development expenses were approximately $54.4 million compared to approximately $32.8 million in the first three quarters of 2024.

24.

Again, excluding stock-based compensation, our GNA expenses were approximately $4.1 million in Q3 2025 and approximately $2.2 million in Q3 2024. For the first three quarters of 2025, excluding stock-based compensation, our general and administrative expenses were approximately $11.1 million, compared to approximately $5.7 million for the first three quarters of 2024.

Net loss for the third quarter of 2525 was approximately 24.6 million compared to a net loss of approximately 12.6 million for the third quarter of 2024 and net loss. For the first 3 quarters of 25 was approximately 74.9 million compared to a net loss of approximately 33.4 million for the first 3 quarters of 2024 but that I'll turn the call back over to Linda. Uh thanks AJ. Um, as AJ just mentioned, we ended the quarter with approximately 100 million dollars in cash, providing a solid foundation to continue executing on our near-term objectives and advancing our Key Programs. And let me just remind you that if Jeremiah fell as approved, we remain eligible to receive 80 million Milestone payment from NS Pharma and a priority review voucher which represents significant non-violent Capital opportunities that would strengthen our balance sheet and our extend our Runway well into 2027.

And Beyond.

We will now open the line for questions.

Thank you, ladies and gentlemen, we will now begin the question and answer session. Should you have a question please? Press star. Followed by the number 1 on your touchtone phone. You will hear a prompt that your hand has been raised.

Great, thank you very much. Um, I excited for the upcoming hope 3 data. I just want to get a sense for what we should expect from that, in terms of what will be actually released in the um, initial data reporting in the Topline data. Thanks so much.

Hey, Ted, it's always great to hear your voice. Um, yeah. So, Topline.

Primary.

Uh, will we will release those as soon as we have them and, uh, we'll host a conference call to explain them and, and help the markets as well as um the key opinion, leaders help, explain the the ramifications of that data.

And just real quick, follow up if I met with the um consideration of left and Dracula rejection fraction as a key. Secondary are there any statistical um, changes in the study because of, um, sort of elevating lvef tanks

No, Ted, so thanks for that. So the great, excuse me, the great thing is, the study was, was always powered with the idea that we were, well overpowered to measure ejection fraction. We had such strong results from Hope to and hope to open label extension that um, it really wasn't an issue, we have a tremendous power in projection fraction. It was powered for pull but that the overarching power is is quite strong for for the cardiac as well.

Great. Well good luck and uh, break a leg.

Your next question comes from the line of vand. Gershel from oppenheim. Your line is now open.

All right, thank you, um, for this update and taking our questions, um, want to ask just a bit further on on the Hope 3 and, and the sap, uh, do you know, we're in this sort of unique circumstance of having a bla that's for the, um, cardio myopathy. But the primary endpoint of the of the trial is, um,

Is, um, the, uh, P, you know, 2.0. So so, so wondering how the sap designates treatment of the lve, secondary endpoint, um, in in the situation in which you miss

A significant on the primary.

Thanks, yeah, Leo. And thanks so much. So this is obviously been an issue that we've spent a lot of time thinking about working with the FDA. And we've brought in um, several uh, very well-respected statistical, Consultants to help us build an sap, that allows for both of those parameters, right? So, you know, the Food and Drug Administration said, we want the primary to remain the performance of the Upper Limb. We left it as a performance of the Upper Limb. The way that the primary is being analyzed as as I mentioned in my remarks. Um, we'll look at the combined cohorts, A and B. Um but we're going to focus then also on looking specifically at cohort B.

Because that's going to provide strength, um, in the manufacturing facility that we have built and passed PLI with.

The alpha is going to be used in the primary endpoint. As would be in any other situation, should we achieve statistical significance? The alpha passes through to the secondaries and continues along until you miss a secondary. The key secondary of ejection fraction. We did not reserve any specific alpha for, um, but we, uh, know from our type A meeting that the FDA was interested in looking at all of the data in its totality. So the SAP is a pretty traditional one and actually fairly simple, um, with which we think we have a lot of great opportunity to utilize the performance of the upper limb and then also ejection fraction. Now,

just to add a little bit of, you know, color to what you asked. Um, because our open bla is for cardiomyopathy. We are not going to act, at least in first iteration to expand the indication to skeletal muscle. Until we are assured that we're going to get the indication and label of cardiomyopathy. Once we have achieved that with the agency, we will then assuming that we have statistical significance. And in the primary we'll then asked to expand to skeletal muscle as well.

Okay, thank you. And, and also just a question with respect to the, uh, cardiac MRI review procedure. Is there could you just run us through? That is are all of these reviewed by an external Reviewer? Is there an adjudication process? If you wouldn't mind just summarizing that thank you.

Yeah, so that's obvious.

Important.

Charter in place.

MRI their first quality controls. So everybody looks at them um, at the cro to make sure that the image meets the standards of being able to be reviewed and analyzed once that happens, then they're read by a primary reader, a secondary reader. And then anywhere the primary reader in the secondary reader disagree by, um, a certain number, which is of relevance, that could not possibly change that much over a certain time period. There's a third reader that comes in for adjudication and then the 3 of them look at it together to decide uh which in fact would be the appropriate read so there's a

Try level measurement procedure and they are, um, independent to time point. So they don't know which time point it is and also to patient ID and obviously to treatment group.

That's helpful. And thanks very much.

Absolutely. And great to hear your voice.

Your next question comes from the line of Joseph penis from hcw, right? The Line is now open.

Hey, Linda and AJ, thanks for taking the questions. Uh, Linda. I just wanted to clarify something quickly before my questions, so, um, depending on the primary endpoint, you said you're going to um continue to look for cardiomyopathy and then if statistically significant expand to skeletal, if I heard you correctly, would that be in the form of an SBA?

Yeah, so interesting that you asked that we don't really know exactly how we're going to. Um go about that yet. It's going to involve conversations with the agency. So right now we have a crl the crl said we want to see more data. We're going to give them more data as a response to the crl. We're going to provide all the data which includes the primary and the key secondary endpoints which we've um also agreed that we would um provide publicly. So you'll get to see them too. And then in those conversations we'll decide how we're going to do the label expansion. The skeletal should that be appropriate?

Understood. Okay. Um, and I just wanted to make sure I understood because it looks like um, with regard to the analyses, you're going to be looking at the 2 manual uh cohorts of A and B versus B Alone. Um does that include any Alpha spend at all? Or how should we view those um in general um, impacting the sap or not?

Yeah. So the way that the analysis is built is, uh, uh, statistical analysis. That's very commonly used called the hawk Berg analysis which basically does not um, utilize Alpha spend if you prespecified going into your secondary, but if you actually directed

A pre-specified, you save all your Alpha, and therefore you have it to use in your secondaries.

Understood. Okay. And maybe a question for AJ, and if you'd like to fill in, that'd be great. I wanted to get a sense now, with regard to your burn going forward. You have a couple of things coming down and a few things potentially going up. You'll have Hope 3 wrapping up in the clinical trials, expenses around that. I wanted to see about, you know, discussing manufacturing expenses that might be increasing, personnel, and then maybe a gradual increase in exosomes. Perhaps some views on how the expenses might be going forward.

Yeah. Thanks Joe. I mean, obviously our expenses in the third quarter were were higher than they had been but we were moving towards pretty much a pufa date. As we moved. Got our received our crl in July. A lot of the expenses. You just said, uh, correctly have gone into the um, the execution of the Hope 3 trial, which is winding down. So, we'll see those expenses. Hopefully, continue to wind down, but they're going into the manufacturing and then development of the commercial product as we prepare and continue to prepare for a commercial launch. So we're maintaining and cautiously watching our burn in every area we can. Um, we're building out our team in areas that are absolutely necessary and then obviously following the results of the data and our next steps with FDA will continue to put the dollars to work where they need to go. So we feel very comfortable with where we're at. And we're putting, uh, putting diligently investing in where, uh,

We hope to drive value exosomes, you know, same type of answer, you know, just to point out, niad is obviously funding that study. We've said that many times, we've already made the doses necessary for that, so that's well off our balance sheet, which hopefully will be a nice, um, value driver, and Catalyst for us in the early part of 2026.

Got it. Thank you.

Thanks Joe.

And I'm sending you all my best in the next few weeks ahead for the company.

So, on the statistical analysis plan, my understanding was when you...

originally designed,

Study you powered it based off of cohort a in terms of patient, size, number and now that you will be using cohort B. It's essentially the same size. Anyway, is, is my understanding correct here.

Yeah, pretty close. Uh, Kristen. That's exactly right. So we combined them, you know, it's it's been a long journey, right? So, um, we started with cohort, a then we were told by FDA, we needed to add cohort B for efficacy. Then when we, um, were filing the bla on the existing data, we combined A, and B into sort of 1 clinical trial, because they agreed to uh, non-clinical comparability between the sites but now especially with some of the things that's going on with the administration. The fact that um, our manufacturing plant in San Diego has passed pli. We've answered their CMC issues. We feel that it was important to be able to highlight, uh, the potential efficacy of cohort B. And yeah, the powering is is pretty much the same corby's a little smaller with an 80% power and cohort, a was a 90% powering, but we still feel that we're well within the range of of ability to achieve efficacy.

Okay, appreciate that. And I know in the past you've shared with us a little bit of the Baseline characteristics amongst these patients, including the percent that had cardiomyopathy as we now divide it between A and B. Would you say that that statement is still true or is there 1 cohorts or skewing a little bit differently?

Yeah, so the Baseline characteristics are pretty much identical across the cohorts. We didn't change inclusion exclusion criteria for either 1 and really it didn't, you know, work out in any specific way, that it was heavily weighted 1 way or the other we have seen. And obviously, I don't know the data from Hope 3, but we've seen from Hope 2. Open label extension from John sassa's, uh, natural history study. That those patients that, um, get treatment with a ejection fraction over, 45% seem to do specifically well, um, compared to those that are worse off. So we're, we're definitely trying to get to these guys as early in the pathogenesis of the cardiomyopathy as possible. Um, I think when we did the analysis of cohort, A and B together in preparation for the crl response, we had over 70, that would have had diagnosed cardiomyopathy 1 thing. That's really nice about cohort. B is we're also measuring scar as measured by late, Galilean enhancement.

So we'll also be able to do a correlation between the amount of damage that looks visible in the heart as well as ejection fraction and or volumes. Um, which is going to be very important for the field. Moving forward to understand, you know, sort of what the chipping points are in terms of Scar aggregation and function.

Okay, great. Thank you so much again. Wishing you all the best.

Thank you. Thank you. Thank you.

Your next question comes from the line of Katherine. Novak from Jones trading, your line is now open.

Hi um, good afternoon. Thanks for taking my question. Um I just have a question on, you know, when you mentioned FDA intended to exercise regulatory flexibility, what, at what point would you do, you intend to ask them to exercise this kind of flexibility? You know, there's a possibility that, um, you know, you don't see statistical significance on P, but there is some kind of

Uh, apparent benefit on lvef, you know, is this in a situation, which you would want the FDA to try to look at the totality of the data going forward.

Yeah, so you hit it, exactly. Um, you know, we are obviously anxiously awaiting the data, the easiest story will be if we hit Paul and we hit ejection fraction. Um, if for some reason we miss on pull and I think there's a lot of information that's being discussed. Both, you know, in the uh, cognoscente, um, Arenas, in terms of the performance of the Upper Limb, what its utility is, how good of a measure. It is, the liability of it, that kind of thing. If, for some reason we miss, um, pull, but we hit hard on cardiac, that would be where we would ask for that regulatory flexibility. Um, and we're hopeful that based on what we have in the type. A minutes what we have educated the FDA about with the performance of the Upper Limb and the key opinion leaders uh that we would still be able to succeed in getting the label for cardiomyopathy.

If there's anything you can share specifically.

What about what the FDA did say? When it comes to regulatory flexibility, we've obviously seen them be more stringent when it comes to statistics in recent, um, decision-making. If there is anything you can, you know, give us any more specific detail you can give us about what the FDA has said about, um, what it means to exercise regulatory flexibility.

Yeah, I think when we put out our press release on our type a meeting, we actually provided a quote in there that came directly from the minutes, which basically said, we want you to submit all of your data from Hope 3, we're not willing to change the primary 2 ejection fraction but we will um regard all of the data and uh make decisions based on um pretty much the preponderance of all the data. They did not give us specifics. Um we did have a hallway conversation in which 1 of the reviewers um assured. Um the mother uh Mindy lessler, that came with us to our type, a meeting that they would be, very sure to look at the cardiac data very carefully. Um, as they recognized that, this was the the unmet medical need with no approved Therapeutics. So that patient population,

Um, okay, that makes sense. Uh well.

Thank you so much for your time.

Your next question comes from the line of medicine elsaadi from B Riley Securities. Your line is now open.

Hey guys, thanks for taking our question a couple from us.

Um, it do you have a sense that from the fda's position? How did they view cohort B? Is it a I guess more important than the aggregate pools?

And then, secondly, maybe what if the bar to achieve a more rapid review time that you alluded to, and has that been a request that has made? Or is that something that you would? I guess, request alongside the submission of Hope 3 data.

Yeah, yeah. All really good questions. So, the reason that we're focusing a little bit more on cohort, B, which we might not have done, um, had the original plan of of the existing data, um, been accepted. And, and we had approval already for the cardio myopathy is because we knew that there was some question regarding the manufacturing of the product from San Diego. That it was a shift from a clinical facility to a commercial facility and we passed the pli. So we've seen um frankly um, a lot of crls.

Being issued in the last few months around, um, CMC related concerns. And so we wanted to obviate that by, um, targeting our efficacy data to our approved facility. We thought that would be the safest way to go about it. And since the power in was basically the same, we thought that would be 1 of the best ways to to assure the fastest path to approval in terms of timelines and speeding it up.

That again is going to be based on what we see in the data where we can convince the agency to move a little bit quicker. We have seen them do it. They did it with Calle Vista, they did it with stealth. Um they are typically falling back on as long, a review period as possible and part of that I think is just because they are so understaffed and going through so many changes themselves that speed is hard for them, but we certainly will work with them and, and try and get this to produce as quickly as we possibly can.

Understood. And if I may ask 1 more, uh, to clarify, I think you answered this a couple questions back, uh, but could you clarify the LGE stratification? If that was balanced across both cohorts, or is, is that a cohort be specific?

Um, stratification, that was used.

Corey end point but 1 is a cardiac physiologist. I'm very excited to see because um, you know, it could provide some very important answers um and developing treatment paradigms for duchan muscular distroy.

Got it. That makes a lot of sense. Um, and yeah, good luck on everything upcoming. Thanks. Yep. Thanks, Madison. Talk soon.

as a reminder, if you wish to ask a question, please press star 1 on your telephone keypad,

Your next question comes from the line of Bubala pan from Roth Capital Partners, your line is now open.

Hi, good afternoon, and thanks for taking my questions. Um, so I just have a couple starting from the type 2 classification. I think this is the first time you're articulating that a type 2 is likely. Um, and obviously this is, uh, going to Garner a review period of 6 months. So I wonder if there were any recent developments between you and the FDA that made you believe, or um, the class 1 resubmission is impossible. I just wanted to know when this uh, decision on Class Type 2 was sort of like you know, set on Stone.

Yeah, yeah, yeah. So, uh, thanks for your question. I think I pretty much answered it when Madison asked a similar question a moment ago. So we, um, know that most people are getting Class 2 resubmissions. That's what we're expecting. That's what we've been told. Um, we should expect, um, in terms of being able to speed it up, I think that's going to be

Incumbent upon the strength of the data, um, our conversations with the agency and sort of, you know, other imponderable factors that I cannot answer until I'm not only seen the data, but also met with the agency, but we'll go as fast as we can. We obviously are looking for a quick pufa as well.

And then, secondly, can you discuss whether the potency assay adequately fulfills, the fda's guidance or potency test for cellular products, expectations. Thank you.

Yeah, so we're very proud of of the way that we have developed the potency assay profile for Jeremiah. Sell 1 of our goals was always to make Jeremiah SLA drug product, and that sort of a hand waving Cell Therapy, it works. But we really don't know how it works. Um, and so, um, our science team did a methodical multi-year program in which they looked at the data from Hope 2. They then were able to identify the master cell banks that we use in order to treat the patients in hope to. So, we know it works and then they took those specific cell Banks and they put it through, um, some pretty rigorous, um, RNA seek assays, looking at 166 jeans and all of this, by the way, is in a very nice didactic. Uh, few minute overview on our website. Looking at those 166 genes, then use bioinformatics to basically quantify those, that identified cdc's as a completely unique cell type.

And then, um, identify which ones were up and which ones were down once we have identified, which ones are cdc's by their genotype. We then put them through an anti fibrosis. Assay 1 of the stated mechanisms of action, looking at the production of collagen, 1 and collagen 3, or in this case, the knockdown of collagen 1, collagen 3, um, the main product of fibrosis and each and every lot has to pass that by a certain quantification in order to be considered effective dermis cell. So we feel very confident in our potency assay profile. It's now been published. Um, and again, if you're interested in more details, uh Dr. Christy Elliott, our chief science and Operating Officer does a nice job explaining it in more detail on our website.

All right. Thank you very much.

Thank you.

Your next question comes from the line of Matthew Venezia from EGP Alliance Global Partners, your line is now open.

Hi Linda. Hi AJ. Thank you for taking my questions. Um, just 1 quick 1 on the potential label expansion. Is there any chance? Um at first launch, if an approval were to be granted that you could get as a cardiac and skeletal label or are you mostly looking at label expansion further down the line

No, I think we'll obviously enter into those conversations during our labeling discussions with the agency. Um, of course, it's all incumbent upon, you know, what the data shows, but certainly if we achieve statistical significance, um, in skeletal and cardiac in our labeling discussions, we will ask for for the label to have both parameters.

Got it. Okay and then just to switch gears a little bit to the exosomes platform um for the co vaccine program. Um,

Recently, selling their COVID-19 vaccine program to dine a vaccine, what are the partnership opportunities that you potentially see? Has there been any inbound? Is there anything you could share on that front for the exosomes platform?

Yeah so um we're all waiting with baited breath. For the data to come through from the niad study obviously

The government shutdown has not helped that um we are uh looking forward to getting that data. I think once we have that data, we'll be on a more directed shopping expedition for the appropriate partner. This vaccine is fantastic. Um, it's a native protein vaccine, we use, no ad events the um lipid that encompasses. It is a exosome, which is a natural product. So you don't have to worry about gumming up the liver or the spleen. Um, and if the antibody response and T Cell response, is anything similar to what we saw pre-clinical, it should have really tremendous Value Plus, because of the, um, little bit of protein that we need to evoke a strong immune response, we can do multivalent vaccines, not only with, you know, multi veillance per coid, for instance, but we could do a CO plus flu plus RSV and all of those are are um in the planning stages. So we're pretty, um, excited to see that data and we'll provide updates as the data becomes available.

Great. Thank you for taking our questions.

Yeah, more than welcome.

Your next question comes from the line of Joseph. Vanis from HC we write your line is now open. Hi. Um thanks for taking the follow-up. Um Linda I want to make sure I'm absolutely sure on this so forgive me if there's any repetition here. Um and this goes to the sap around hope 3 and I'm getting questions on this as well. So obviously you need the primary to hit to be able to trigger lvef. If I heard that correctly, number 1,

Is that is that a question or did you dismiss? Is that the like I just want to make sure that's correct. You have to hit the primary in order to trigger the analysis of lvef. And um, if you don't hit the primary, um, you know, how would lvef be analyzed and what would you say, would be the, um, hurdle for Success there?

Yeah, yeah, yeah yeah. So, you know, this is where sort of the colloquialism of regulatory flexibility becomes the open-ended question. It depends on how far we miss on the poll. Let's say if we did. Um and then what the P value independently of ejection fraction would be. I think we would go in there and find really hard if we missed the by a little, um, pull and achieved, uh, really nice P value on ejection fraction. It depends on how dogmatic the agency is going to want to be on statistical.

Um you know analyses which typically use up your Alpha and your primary. Um but in this situation because they have said they would be flexible that they would look through to the cardiac data that they know that the applications for cardiac and the bla they were looking for more cardiac data. There may be a lot more windows of opportunity than just sort of uh strict statistical Dogma.

Got it. Thanks for the clarification.

Absolutely.

There are no further questions that this time, I will not turn the call back to cap the card management for their closing remarks, please go ahead.

Thank you so much for all of your questions and also for participating today. Obviously, the coming weeks will be transformative for Capricorn as we prepare to announce the Hope 3 top-line results. These data will define the next chapter for Jeremiah Sell and for our company. As we advance toward our goal of delivering a life-changing therapy to patients and families affected by Duchenne muscular dystrophy, we remain steadfast in our mission and continue to execute with disciplined scientific rigor and readiness across every area of our business. To the DMD community, your courage and partnership continue to inspire everything we do. We look forward to sharing this important next step with you soon. Thank you so much for joining us today, and we will be in touch soon.

Ladies and gentlemen, this concludes today's conference call. Thank you for your participation. You may now disconnect.