Capricor at Leerink’s Conference: Strategic Moves in Cell Therapy

On Wednesday, 12 March 2025, Capricor Therapeutics (NASDAQ: CAPR) presented at Leerink’s Global Healthcare Conference 2025, highlighting its strategic advancements in cell therapy. The company discussed both promising developments and challenges, including its allogeneic cell therapy, Deramiocell, aimed at treating Duchenne muscular dystrophy. Despite the positive outlook, the company faces hurdles such as regulatory approvals and market competition.

Key Takeaways

• Capricor’s cell therapy, Deramiocell, shows significant promise in slowing disease progression in Duchenne muscular dystrophy.
• The company expects milestone payments and sales-based revenues from its partnership with Nippon Shinyaku.
• Capricor has sufficient funds to operate until 2027, with plans for revenue generation before year-end.
• A new manufacturing facility is underway to meet increased demand.
• Expansion plans include targeting Becker muscular dystrophy and other orphan cardiomyopathies.

Financial Results

• Milestone Payments: $80 million anticipated from Nippon Shinyaku upon approval.
• Sales-Based Milestones: Up to $605 million expected.
• Priority Review Voucher: Potential sale valued between $100 million and $150 million in Q4.
• Revenue Model: Capricor to receive 30%-50% of net sales revenue share from Nippon Shinyaku.
• Cash Runway: Funds sufficient for independent operation until 2027.
• Manufacturing Agreement: Capricor sells to Nippon Shinyaku at about 10% of anticipated reimbursement cost, with the partner handling sales and marketing.

Operational Updates

• BLA Submission: Completed at the end of 2024 and accepted for review in March 2025.
• Priority Review: Granted with a PDUFA date set for 31 August 2025.
• Manufacturing: Existing facility in San Diego, with a new facility in development to increase capacity.
• Partnership: Collaboration with Nippon Shinyaku for sales, marketing, and distribution in the U.S. and Japan.
• Open Label Extension: 100 patients in the study, expected to transition to commercial product first.

Future Outlook

• Expansion Plans: Targeting Becker muscular dystrophy and other orphan cardiomyopathies.
• Revenue Generation: Expected to begin before the end of the year.
• Manufacturing Capacity: Expansion to meet the needs of thousands of patients.
• Pipeline Development: Stealth X exosome platform expected by the end of 2025 with trials beginning in 2026.

Q&A Highlights

• Trial Design: Full approval sought based on two pivotal studies, not accelerated approval.
• Patient Numbers: HOPE-2 study involved 20 patients; 10 patients with MRI data in the open-label extension.
• Side Effects: Common side effects include malaise and flu-like symptoms; 30% experience headaches or nausea.
• Safety & Dosing: Patients pre-treated with steroids and antihistamines; quarterly treatment for four years.
• FDA Feedback: Favorable due to objective measures and consistent dosing protocol.
• Revenue Share: Capricor to receive 30%-50% of net sales revenue from Nippon Shinyaku.

For more detailed insights, readers are encouraged to refer to the full transcript below.

Full transcript - Leerink’s Global Healthcare Conference 2025:

Unidentified speaker, Presenter, Capricor: the SEC. Please feel free to look at them should you have any questions. Capricor has been around for coming up on twenty years upon which we have been building our allogeneic cell therapy called Derimyocell. The technology was originally developed at Johns Hopkins University. And as I walk through my presentation today, we will hear about the mechanism of action, the publications that have been validated by the mechanism of action, the fact that we’re treating Duchenne muscular dystrophy, we manufacture our product in house and we have a commercial scale manufacturing facility and we have a commercial partnership in The United States and Japan with Nippon Shinyaku.

I won’t be spending a lot of time today on our pipeline products, which is the Stealth X exosome platform, but look forward to that becoming available as we get through the end of twenty twenty five and into 2026. Shown here is our product pipeline and I’ll just highlight for a moment the fact that we’re going to be talking today primarily about Daramiosil, which is an allogeneic, which means an off the shelf cell therapy product for which we are using to treat Duchenne muscular dystrophy, primarily the cardiomyopathy. And again, shown here are some of the exosome based programs that are coming forward, anticipating to enter into the clinic in the therapeutic arena in 2026. So where are we in terms of our catalyst and development of dermael for Duchenne muscular dystrophy? The last twelve months have been really busy.

Actually, it’s more like fifteen months now, have been really busy ones for Capricor. We have been able to meet with FDA on a regular basis, starting with our pre BLA meeting in August of twenty four, where we decided along with the support of the agency to file on the cardiomyopathy associated with Duchenne muscular dystrophy. We then began submitting aspects of the BLA with the submission complete at the end of twenty twenty four. The application was accepted for review, most recently, just a few weeks ago in March of twenty five with priority review granted. Our PDUFA is the stated date of 08/31/2025.

We’re looking forward to that. On the economic side of the house, we have multiple milestones coming our way as we proceed to and get to approval, dollars 80,000,000 from our partner, Nippon Shinyaku, sales based milestones up to $6.00 5,000,000 and potential sale of the priority review voucher for which we are qualified and are in the Q4. And that has most recently been selling somewhere between $100,000,000 and $150,000,000 and we anticipate selling that when we anticipate receiving it. We have plenty of cash in the bank, did a fundraise in late twenty twenty four, which supported our balance sheet. We have money independent of those milestone payments I just discussed up into 2027, at which time, we will have been commercial for quite some time.

So let me tell you a little bit about dermisol, how it works, what it does, and why we think it’s an appropriate therapeutic for treating the cardiomyopathy associated with Duchenne. As I mentioned, it’s an off the shelf product. I’ll talk about manufacturing in the next few minutes. But what I’d like to highlight right now is that we have a stated mechanism of action which has been validated by our potency assays that have been approved by FDA as appropriate for this product and for approval. It is a multimodal mechanism of action started with immunomodulation.

There are over 200 patients that have been treated with dermael. As I mentioned in our first slide, there’s over 300 publications from 55 labs worldwide talking about the scientific designation of this product, which is called a Cardiosphere derived cell. And as I mentioned, we have potency assays identity criteria and other parameters with which to identify ourselves and validate that batch to batch consistency is achieved in terms of the therapeutic window. Because of the rare disease aspect as well as treating a pediatric disease and the fact that we’re cell and gene therapy, we have multiple designations that have helped to pave our way in dealing with the regulatory pathways, Orphan Drug Designation, RMAT, which is Regenerative Medicines Advanced Therapy, is like breakthrough for cell and gene therapy. We now have ATMP in Europe, similar to orphan designation and as I mentioned we have rare pediatric disease designation in The United States, which is how we qualify for the PRV.

So how do we make dermaicel? Dermaicel starts with an explanted human heart that is transplant qualified but cannot be used because of technical reasons. Now you might be scratching your head and saying, Wow, how often does that happen? There are way more people that need heart transplants that are getting them. Well, the answer is fortunately for, the people that can benefit from ourselves but unfortunate for those recipients, many times there are technical reasons for which those hearts cannot be transplanted safely into a human being, but are very appropriate for our use.

And that could be if there’s an accident or some type of physical damage to the heart or something that happens in the transport of the heart that we are able to get that heart into our labs. We then cut the heart up, we isolate out our cells of interest. It is a stromal cell population. It is not a stem cell. They do not retain and graft or become part of a functioning syncytium.

What they do is they release exosomes after they infused into the body. And I’ll talk a little bit about that over the next few slides. When we isolate out our cells, we then put them through some proprietary steps, which includes a spherical suspension step. We ultimately plate them on fibronectin coated dishes, gather them up into vials where then they are cryo preserved and ultimately deliver to patients the dose is one hundred and fifty million cells four times a year. So once a quarter, our guys get an infusion.

So let’s talk now about the disease that we’re treating, the cardiomyopathy associated with Duchenne Duchenne muscular dystrophy. Highlighted in this quote here is some words taken from the publication from the paper published by John Soslow and his colleagues from the Action Network and the Cardiac Consortium to understand the pathogenesis of the cardiomyopathy associated with Duchenne muscular dystrophy. And what I want you to understand is that the cardiomyopathy is the leading cause of death in the Duchenne muscular dystrophy patient population. And while most of these boys and young men are on standard of care cardiac medications, it is not enough to ameliorate the progression of this disease process. And finally, with daromiosel, we have an opportunity to directly treat the cardiomyopathy associated with Duchenne.

So I’m going to walk you through this graphic here very quickly because I think it’s really important. So shown on the y axis is ejection fraction, which is how the heart meets the needs of the body. And it is the primary efficacy endpoint for which we have asked for approval. And on the y axis is age. The dotted blue line is an imaginary line that we’ve drawn through, at the ejection fraction level of 45%.

That’s because that’s what we and others know is a very relevant cutoff where the heart is sick enough in these boys and young men that they need to be treated or they’re going to continue towards death, which obviously is what we’re trying to avoid, or where we are able to start the treatment paradigm as early as possible to preserve cardiac muscle. So I want you to pay attention to the fact that ejection fractions continue to decline over the course of the lives of these boys and young men as is shown with the regression line shown here, but also that the pathogenesis of the cardiomyopathy is age independent. So you can have a young guy with a pretty sick heart and you can have an older guy with a pretty sick heart and they don’t necessarily go in correlation with the skeletal muscle myopathy. So it’s very important to treat the cardiomyopathy as a separate but very relevant implication of Duchenne muscular dystrophy. So along that line, I want to highlight the fact that dermaicel can be used in conjunction with any approved or along the way approved therapeutics for treating the cardiomyopathy associated with Duchenne or Duchenne muscular dystrophy itself.

So on the sort of left side of this diagrammatic you can see that there are the therapies that have been developed or are being developed to treat the dystrophinopathy. That is the lack of dystrophin caused by Duchenne muscular dystrophy. These therapies are gearing to try and create healthy muscle cells to try and sustain function in those muscles. On the right side of this graphic, you also see the drugs that are approved to treat the sequelae, as you will call it, associated with Duchenne muscular dystrophy, which is the inflammation and the fibrosis that go along with the constant breakdown of muscle cells, whether it be skeletal or cardiac. But what we have sort of in the middle is dermisol.

Dermisol is a good player in the sandbox to treat Duchenne muscular dystrophy because it can be used in conjunction with something to treat the dystrophinopathy and also because all of our patients are on every approved standard of care medication, including steroids, we know that what happens when the patient is treated with dermael means that they actually do better than anything else that they’re already on. So it’s very important to understand. We know that a lot of people are looking at the cardiomyopathy associated with Duchenne muscular dystrophy. It is a very serious component of Duchenne muscular dystrophy. We fully expect other advances to be made in the therapeutic paradigm.

We don’t think that anything will surpass what dermael cell can do because of its sustained benefit in preserving cardiac function and also skeletal muscle function, which I’m going to show you over the next few minutes. So I’d like to go through some of our clinical trial results. I think these results which I’ve talked about over time are part of the backbone of our application for approval from the FDA. What we showed in the HOPE two clinical trial, which was published in the journal The Lancet, as you know, a highly regarded peer reviewed journal. And what we showed is that we showed a seventy one percent slowing of the progression of the skeletal muscle aspect of the disease.

This was measured by a test called the performance of the upper limb. The boys and the young men that were in HOPE two were primarily non ambulant. That means that they couldn’t do a NorthStar ambulatory assessment. They couldn’t do a Time to Rise. So the assay that has been developed and utilized to measure upper limb function in people that can no longer ambulate is called performance of the upper limb and is a measure of shoulder, arm and hand function.

And what we showed in the HOPE clinical trial was that we had a seventy one percent slowing of disease progression, as I stated before. So that means that, the disease went much slower and that was treated than in the placebo, that were followed along at the same time. We showed a one hundred and seven percent slowing of the cardiac disease, which actually could translate into an improvement in cardiac function in these boys and young men. And that’s validated by the very, the very positive P value associated with the improvement in cardiac function. P equals 0.002, which means that there’s an almost infinitesimal chance that the data that we have seen is related to chance.

That means that it is most likely due to a treatment effect of dermaicel. And most importantly, and what is most important in our application to FDA, is that we show a very significant attenuation of disease progression year over year in our open label extension patients and a very safe and tolerable profile of the treatment of dermaiselle in these boys and young men with Duchenne muscular dystrophy. Now one point I’d like to highlight here on this slide, which is that remember I told you that the cardiomyopathy does not develop along the same lines as a skeletal muscle myopathy. You can have differential decline in function depending upon how your personal disease progresses. The fact that we see improvement in skeletal muscle function and cardiac muscle function really further supports the idea that dermisal is having a relevant treatment effect in these guys with Duchenne muscular dystrophy.

So let me highlight a little bit more the power of the cardiac data that we have shown. This is again taken from the HOPE-two clinical trial published in The Lancet. This is called a forest plot. A forest plot is the way of looking at multiple measures and whether the data is biasing towards a treatment effect to the right side of the centerline or towards basically no treatment effect, which would be to the left side of the centerline. And what you can see here on the left is in 21 out of 22 measures of cardiac function as measured by MRI, which is the gold standard of measurement of cardiac function.

We saw treatment effect of dermaicel in Duchenne muscular dystrophy and HOPE-two. In the pullout on the right side, so you can see it a little bit more clearly, are the measures by which we are able to assess the morbidity and mortality of Duchenne muscular dystrophy as published and assessed by what I showed a few slides ago, the John Soslow and Action Network data showing the relevant parameters that can predict progression of the disease. And I’ll just call those out very quickly in case you can’t see them very well, which is left ventricular ejection fraction. Remember the y axis on the graph I showed of the pathogenesis of the cardiomyopathy and diastolic and end systolic volumes. Basically those are measures of the dimensions of the heart and how the heart shape can predict its function.

Very important in sustaining the shape of the heart in order to meet the needs of the body. And then finally, an enzyme called CK MB, which is an enzyme that is typically housed within a cardiac muscle cell and is only measured in the blood if heart cells are being broken down pathogenically. Like for instance, if you go in and you think you’re having a heart attack, it’s one of the measures that they look for in your blood. If you have an elevated CKMB, it means heart cells are breaking down in a pathologic way. And what we see is a very significant attenuation of that cardiac muscle enzyme in our treated patients.

So these are the key measures from which we are using to identify the treatment effect, the efficacy and the possible long term outcomes of dermisol in treating Duchenne muscular dystrophy cardiomyopathy. So because of the power of this data, we did an open label extension study. The original plan for the open label extension was to treat the patients for one year, which was part of our protocol and is typically a way of showing our gratitude to patients that have entered into clinical trials. Because of multiple reasons, including trying to understand the efficacy of the product, The boys and young men were off all products, whether they were in a treated group or a placebo group for a year and then they came into the Open Label Extension Group. They’re coming around the fourth year of Open Label Extension now, even though we only promised them one.

They have done so well on it that we’ve kept it going. Some of them will be obviously starting their fifth year. Those Open Label Extension patients will be our first patients to come on to the commercial product and we’ll be really excited for the opportunity for all boys and young men to have access to dermaicel. The open label extension patients, there are 13 of them of our original 20. Six were the original dermaicel, seven were the original placebo, One patient withdrew from the study.

The age of our patients now are approximately 17 years. As I mentioned a few slides ago, but I want to highlight, all these guys are on stable corticosteroids, which means they’re getting the best medicine has to offer them and all of them were non ambulant. Because we studied these patients so far out after the end of the placebo controlled trial, we needed a benchmark. We needed to figure out like what are we seeing in relation to what happens in the natural course of this disease. So we’ve been very lucky on the cardiac side to work with John Soslow at Vanderbilt University, the Action Network and the Cardiac Consortium.

They were funded by the Food and Drug Administration, the Office of Orphan Products, to understand by doing imaging of hundreds of Duchenne patients to understand the pathogenesis of Duchenne cardiomyopathy, the morbidity and the mortality And that is sort of the quote that is shown here on this slide, is the actual specific aim of this study, which is to understand what measures we can use to understand are they getting worse or are they getting better. And so we were able to access that data both in a summary from the publication but then also at the patient level data for propensity matching, which we use in our Biologics License application. What you see here is the data that we have actually shown the Food and Drug Administration. The graph bar chart on the top is the ejection fraction, again how the heart meets the needs of the body. And the bottom bar chart is end systolic volumes, which as you remember from the forest plot I showed you a few slides ago, we were able to show conclusively that there is reverse remodeling or stabilization of structure and function of these hearts.

What we see is year over year, twenty four months into the Open Label Extension and then thirty six months into the Open Label Extension, stabilization of ejection fraction and improvement in volumes. And we also are seeing when you use that external comparator of Doctor. Soslow’s study, we see a 5% decline in twenty four months, with a delta of 6.9 ejection fraction points of improvement when you are on dermisal year over year and similar improvement in structure as measured by end systolic volume shown in the bottom. And then in conjunction with Cincinnati Children’s Hospital, we looked at the skeletal muscle function, the performance of the upper limb. This is same three years of data.

The blue line are dermisal treated boys and the red line, is the Cincinnati Children’s Natural History Study, where they just measure their performance of the upper limb when they come in for their annual visits. And what you can see is a very statistically significant as well as clinically relevant attenuation and disease. And this is after three years of treatment that we are still seeing slowing of the disease process. So showing long term treatment with dermael has beneficial effects both in cardiac and skeletal muscle function. So I’m just going to spend the last few slides very quickly running through the revenue opportunity here.

Let me just remind you that this would be a first in class product that there are fifteen thousand to twenty thousand Duchenne patients in The United States. Theoretically and practically every single one of them would qualify for dermaicel at some point in their life course because they all develop the cardiac disease as part of the pathogenesis of Duchenne. They get four doses a year. Reimbursement, we’re targeting right now at about the price of an Exxon Skipper, which as many of you know is quite significant. And then with our partnership with Nippon Shinnyaku, our revenue share is 30% to 50% and is quite an extraordinarily positive revenue model for Capricor.

And as I mentioned, I just want to highlight about 100 patients are already in open label extension. They’re likely to be the first to transition to commercial product. And all things being equal with PDUFA at the end of the summer, we anticipate being revenue generating before the end of the year. And finally, just very quickly, our partnership with Nifan Shin Yaku and S Pharma, we are doing the development, the manufacturing, regulatory activities, everything getting it ready to go into the arm of a patient. And then Nippon Shinayako takes care of sales, marketing and distribution with exclusive U.

S. Marketing and also Japanese rights as well. And so we are actively working with them now prepping for launch and looking forward to taking this forward commercially with them. And then finally, because this is such a powerful product, the pathogenesis of the muscular dystrophies in terms of the cardiomyopathy is very similar. So while we’re starting with Duchenne, our goals will be to be in vector muscular dystrophy before too long after approval with Duchenne, as well as other orphan cardiomyopathies.

We have a fully established manufacturing plant in our San Diego facility, which is commercial ready. And we’ve just recently started to work on a new manufacturing facility in our same building to be able to expand to the needs of thousands of patients as necessary. And then finally, I just want to thank the patients, the families, everybody that has made this possible. We have very strong relationships with the advocacy groups, as they know that the heart disease is the number one cause of loss of life in these boys and young men. And so we’re going to continue to be out there working hand in hand to get this across the line, with these patients and families.

I’ll stop there, if anybody has any questions. Sure. So we’ve applied for full approval for So we’ve applied for full approval, for our biologics license application, not accelerated. That’s based on the data that I just showed you a little bit ago. We have priority review with our PDUFA date, at the August 2025.

Unidentified speaker, Questioner: So that means you don’t have to have large registration study on the other one?

Unidentified speaker, Presenter, Capricor: This is the register we’ve just presented two pivotal studies to FDA. This is the registration studies.

Unidentified speaker, Questioner: And the, the patient number, the end, on the active side this morning?

Unidentified speaker, Presenter, Capricor: On the active side, was 10 patients for the open label extension group and 20 patients for the HOPE-two study.

Unidentified speaker, Questioner: 20 patients for the HOPE-two?

Unidentified speaker, Presenter, Capricor: That’s correct.

Unidentified speaker, Questioner: 20 patients of ejection fraction

Unidentified speaker, Presenter, Capricor: in HOPE two. That’s correct. Yeah. So they take a very simple oral pretreatment regimen of some steroids and antihistamines to prevent, just a typical hypersensitivity reaction. They’re already on steroids.

So this would be this would be a bolus dose just given twenty four hours before, and it’s an oral dose. It’s pretty easy.

Unidentified speaker, Questioner: Okay. And what sort of side effects have you seen?

Unidentified speaker, Presenter, Capricor: We see sort of the typical, you know, malaise and and kind of light flu like symptoms and and some proportion of the patients, about thirty percent of the patients develop, you know, a little headache, a little nausea,

Unidentified speaker, Questioner: a little achy painy. Right.

Unidentified speaker, Presenter, Capricor: Now four years, yes. Every every quarter for four years now, they’ve been getting dosing. So that’s, our n of 10. For four years, yes.

Unidentified speaker, Questioner: Yes. N of 10 of four years. Yes. Okay. Thank you.

Unidentified speaker, Presenter, Capricor: Actually 12, so let me just be clear. So 12 have been dosed in the open label extension, because two of them cannot get into the magnet because of physical limitations. We have MRI data on 10, but 12 are dosed four times a year for now coming up on four years. And then in order to be clear, as I showed you, we did propensity matching of the natural history study from Vanderbilt. So there’s more of those patients, but they weren’t treated with daromiosol.

Unidentified speaker, Questioner: And is there an agreement with the FDA that, 20 patients

Unidentified speaker, Presenter, Capricor: Yeah. Yeah. So this was an active conversation with FDA. They’ve been following our data now for years. We had nine meetings with them in 2024, a variety of formal and informal meetings.

So they knew exactly how many patients we had studied. They’ve looked at the data. They look at the individual patient level data. The reason they’re so, or, you know, so favorable to the application was one, they’ve looked at patient level data. There’s not an outlier that’s kind of driving an average, which, of course, would be a concern to it’s an objective measure MRI, which is, able you know, you can’t wish your heart better, right?

You can maybe feel better if you’re you know that you have to lift your arms and you’ve gotten a drug, but you can’t wish your heart better. So even though it’s a small sample size, it’s actually very relevant data. It’s a very consistent dosing protocol. It’s very safe, very little variability and very statistically significant in terms of p values when we did the propensity matching. So even though it’s a small sample size, they’ve acknowledged that it’s a small sample size, but because the data appears so relevant for those reasons that I just highlighted, they are have been favorable to review the BLA.

Unidentified speaker, Questioner: So do they say this is acceptable for review versus acceptable for approval? Do they make a designation with respect to the end?

Unidentified speaker, Presenter, Capricor: Yeah. So when you get your BLA acceptance, they say they say that it is acceptable for review and they will review it. They haven’t found any deficiencies in the application thus far and they’ll let you know anything as it comes along the way. So far all good.

Unidentified speaker, Questioner: Post marketing commitments with respect to M?

Unidentified speaker, Presenter, Capricor: Yeah. So they tell you that they will discuss post marketing requirements with you as you proceed along the PDUFA pathway. We haven’t had that conversation yet. They’ve highlighted that that will be sometime prior to PDUFA.

Unidentified speaker, Questioner: Last question. With the case studies, Exondis was approved, I think, at a small end as well. Could you remind us what that was?

Unidentified speaker, Presenter, Capricor: So I don’t remember the end for EXONDIS because it’s been for a while, but Alevitus, I know they had six non ambulant patients and I don’t know exactly how many they ended up qualifying for their phase three trial. But look, it’s a rare disease. It’s a terminal disease. There’s nothing out there for them. Safe Therapeutics, you know, Peter Marks and CBER, which is our regulatory body, has made the commitment to take safe and effective therapeutics and make them available.

And we we feel very committed to that same pathway. Thank you so much for your time. Any more questions?

Unidentified speaker, Questioner: Well, I just had a question. What have you told investors about the economic relationship if the drugs on the market with your partner?

Unidentified speaker, Presenter, Capricor: Yes. So, I had that on the one slide. I’ll just go back really quick so you can see. So ultimately, the revenue share that we will get is between 3050%. Our partner won’t let us disclose the exact number, but it’s not very hard, with that kind of a range to figure out exactly what the economics are.

So that is net sales revenue share for Capricor.

Unidentified speaker, Questioner: And who owns the cost of marketing?

Unidentified speaker, Presenter, Capricor: They do. So they handle all of SG and A. We just handle the development costs of the product. Okay.

Unidentified speaker, Questioner: Are you manufacturing the product at that point?

Unidentified speaker, Presenter, Capricor: We are manufacturing the product and we we sell it to them. Right.

Unidentified speaker, Questioner: So that 30 to 50% is is that’s what you sell the product for?

Unidentified speaker, Presenter, Capricor: It’s no. The 30 to 50% is what comes back to us when they sell it. So let’s just No, no, no, no, no. We functionally sell our partner for about a tenth of what we anticipate the reimbursement cost will be and then they sell it for whatever is going to be reimbursed and then we get 30 to 50% of that number. Yes.

Sorry if I wasn’t clear.

Unidentified speaker, Questioner: This is allo?

Unidentified speaker, Presenter, Capricor: It’s an allogeneic off the shelf product made from explanted hearts. It’s not a stem cell since you missed the first few minutes. It’s a stromal cell. No, it’s totally fine. It’s a stromal cell population that is isolated, expanded upon and delivered after being cryopreserved.

Unidentified speaker, Questioner: We need to take something out. Did you knock out something? This is naturally algal bites.

Unidentified speaker, Presenter, Capricor: Yeah. So we use explanted human hearts. These are transplant qualified human hearts that we get from organ procurement organization that we take back to our labs. We isolate out the cells of interest, grow them up and then use them as a product.

Unidentified speaker, Questioner: No, they

Unidentified speaker, Presenter, Capricor: do. They absolutely do. And our patients, yes. So they absolutely do. We don’t have to match.

There’s no immune consequences and you don’t have to worry about rejection because the only thing that could be rejected would be our cells, which actually they do die and go away on their own anyway. So there’s no safety risk. There’s no issues, regarding this this. And then we would probably not have seen an efficacy signal. And this is something we learned now, gosh, about ten years ago.

So we were we were able to discern the fact that, you know, when you’re infusing these allogeneic cells, if the immune system was going to kill them, it would kill the cells, it would kill the efficacy, but it would not do anything to the human being that was being treated. Allogeneic means it doesn’t come from you. Well, the cells do die over time, so probably the immune system does ultimately take them down or they die naturally. But they what that’s what I’m saying, like whatever what we know that the mechanism of action of the cells is mediated by the exosomes. Exosomes are released by the cells after they’ve been infused.

Exosomes track to the sites of injury, the cells can die and go away. So even if they are killed by the immune system at that point, it really has no impact on the efficacy signal. Yes. Do their work. That’s right.

They get released within minutes.

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