Capricor (CAPR) Q4 2025 Earnings Call Transcript

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DATE

Thursday, March 12, 2026 at 4:30 p.m. ET

CALL PARTICIPANTS

• Chief Executive Officer — Linda Marbán
• Chief Financial Officer — Anthony J. Bergmann

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TAKEAWAYS

• BLA Review Timeline -- The FDA accepted Capricor Therapeutics (NASDAQ:CAPR)'s Biologics License Application for deramycin, assigning a PDUFA target action date of August 22, 2026.
• Clinical Trial Data -- The pivotal HOPE-3 Phase 3 trial met its primary endpoint (Performance of the Upper Limb) and all Type 1 error-controlled secondary endpoints, with left ventricular ejection fraction showing a 91% reduction in disease progression among all evaluable patients and achieving statistical significance.
• Subgroup Results -- Among HOPE-3 participants with cardiomyopathy, the left ventricular ejection fraction endpoint achieved a p-value of 0.01.
• Duchenne Video Assessment (DVA) -- The trial showed statistically significant improvement in upper limb function, as measured by home-based DVA EAT 10 BITE, with approximately 50 treated and 50 placebo patients in the analysis.
• Cardiac Fibrosis Data -- Cardiac MRI with gadolinium demonstrated a significant reduction in fibrosis in deramycin-treated patients compared to placebo; this was measured in Cohort B only.
• Safety Profile -- More than 800 intravenous infusions of deramycin have been administered across studies, with consistent safety and evidence of long-term tolerability in open-label extension studies, including patients treated for up to five years.
• Manufacturing Capacity -- The San Diego manufacturing facility has completed FDA pre-license inspection, addresses all Form 483s, and can supply approximately 250 patients per year, with expansion underway to reach an approximate 2,500 patient annual capacity by late 2027.
• Financial Position -- As of December 31, 2025, Capricor Therapeutics held approximately $3.18 billion in cash, cash equivalents, and marketable securities, following a December public offering resulting in net proceeds of $162 million and an ATM drawdown of approximately $75 million.
• Operating Expenses -- Full-year 2025 operating expenses were approximately $108.1 million versus $64.8 million in 2024, reflecting increased investment in clinical, regulatory, manufacturing, and infrastructure for the Duchenne program.
• Revenue -- Reported revenue was $0 in 2025 compared to $22.3 million for 2024; prior-year revenue was fully recognized relating to the U.S. distribution agreement with Nippon Shinyaku.
• Net Loss -- Net loss for 2025 was approximately $105 million, up from $40.5 million in 2024, primarily due to R&D and commercial-readiness investment.
• Funding Outlook -- Management projects available capital is sufficient to fund operations into 2027, excluding potential milestone payments, commercialization revenue, or monetization of a priority review voucher.
• NDAQ Uplisting -- Capricor Therapeutics common stock was approved for transfer to the NASDAQ Global Select Market, the exchange's highest listing tier.
• Exosome Platform Clinical Update -- The StealthX exosome-based COVID-19 vaccine Phase 1 study reported favorable safety at all doses tested, but limited neutralization; final cellular response data is expected in 2026.
• Manufacturing Expansion Plan -- Management disclosed plans for an additional facility in San Diego County to increase capacity beyond 2,500 patients per year, to start after regulatory progress is confirmed.
• Labeling Discussions -- No FDA label negotiation has occurred; company aims to secure labeling for both skeletal and cardiac disease in Duchenne muscular dystrophy based on data strength.
• Priority Review Voucher (PRV) Prospect -- Upon approval, management expects potential receipt of a transferable PRV, which could provide significant additional capital through sale.

SUMMARY

Capricor Therapeutics reported that the FDA has accepted its resubmitted BLA for deramycin and assigned a PDUFA target action date, indicating a defined regulatory timeline and no outstanding review issues raised to date. Management described the HOPE-3 pivotal trial as demonstrating both efficacy and safety endpoints across cardiac and skeletal measures, including quantitative improvements in left ventricular ejection fraction and daily living activities. The San Diego manufacturing facility is operational, has addressed all regulatory observations, and is being expanded to scale supply for eventual commercial launch. Balance sheet strength was highlighted with a year-end cash position exceeding $3 billion, driven by recent financing activities, with operating runway extending into 2027 and no 2025 revenue recognized. Management is preparing for comprehensive commercial readiness and outlines global expansion and pipeline advancement plans beyond the lead indication.

• Management confirmed that the FDA classified the resubmission as Class II, and "has not identified any potential review issues in its communication to the company."
• Leadership stated that the HOPE-3 trial data, including MRI-based fibrosis reductions and DVA-based patient-reported outcomes, will be leveraged to support FDA labeling and payer discussions.
• Capricor Therapeutics plans to transfer all existing open-label extension patients to commercial supply upon approval, and is prioritizing access for non-trial eligible DMD patients as manufacturing scales.
• CEO Marbán said, "There is no way this would be a conditional approval," pointing to the randomized, double-blind, placebo-controlled dataset with statistically significant endpoints.
• Engagements for European and Japanese regulatory pathways with NS Pharma are planned for 2026, but remain subject to further negotiation and resource allocation as U.S. regulatory progress advances.
• The StealthX exosome delivery platform is advancing toward IND-enabling studies, with a target IND filing in 2027; early vaccine study data show safety but "limited neutralization" at tested doses.

INDUSTRY GLOSSARY

• BLA (Biologics License Application): Regulatory submission to the FDA seeking commercial approval for a biologic drug in the U.S.
• PDUFA (Prescription Drug User Fee Act) Date: The deadline by which the FDA must respond to a drug approval application.
• HOPE-3 Study: A pivotal, multicenter, randomized, double-blind, placebo-controlled Phase 3 trial of deramycin in Duchenne muscular dystrophy cardiomyopathy.
• DVA (Duchenne Video Assessment): A validated, home-based patient-reported outcome measure for upper limb function in DMD.
• OLE (Open-Label Extension): A study phase where trial participants continue receiving treatment after the formal trial, typically to assess long-term safety and efficacy.
• Form 483: An FDA-issued notice listing observations from an inspection of a facility.
• PRV (Priority Review Voucher): A transferable incentive granted by the FDA upon approval of certain rare pediatric drugs, allowing expedited review of a future application.
• PPQ (Process Performance Qualification) Runs: Biotech manufacturing validation batches used to demonstrate consistent product quality for regulatory approval.
• Cohort A / Cohort B: Distinct manufacturing-site-based groups within the HOPE-3 trial, examined for comparability of product and results.
• LGE (Late Gadolinium Enhancement): An MRI imaging technique using gadolinium to detect cardiac fibrosis.

Full Conference Call Transcript

Linda Marbán: Good afternoon, everyone, and thank you for joining us on Capricor Therapeutics, Inc.’s quarterly conference call. For our investors, collaborators, the team here at Capricor Therapeutics, Inc., and especially the Duchenne muscular dystrophy patient community, thank you for your continued support and belief in our mission. We entered 2026 with a clear focus as we work to advance Garamia cell for potential approval for Duchenne muscular dystrophy in the United States. As we announced earlier this week, we were very pleased to report that the U.S. Food and Drug Administration has stated that our response to our complete response letter is complete and has therefore been accepted, our previously submitted biologics license application, or BLA, for review.

The agency assigned a PDUFA target action date of 08/22/2026. Clearly, this represents a significant regulatory milestone for Capricor Therapeutics, Inc., of course, for all of those who have DMD. The BLA seeks full approval of deramycin. While we have not yet had detailed label discussions with the FDA, our goal will be to position deramycin to treat as many eligible patients as possible, consistent with the clinical data generated over more than a decade of development where both skeletal and cardiac muscle function have shown stabilization. If approved, deramycin has the potential to become the first therapy designed to address both skeletal and cardiac disease manifestations of Duchenne muscular dystrophy.

We believe that distinction is highly meaningful, particularly given that cardiomyopathy remains one of the most serious and life-limiting aspects of this disease. Our highest priority as an organization is execution, working closely with the FDA, preparing for a potential commercial launch, and continuing to build the capabilities required of a world-class commercial-stage biotechnology company. We believe the strength of our data, our manufacturing readiness, as well as strong balance sheet position us well for this next phase of growth. Our current corporate mission is to build an infrastructure to launch and commercialization of deramycin as well as to expand our pipeline to treat other indications.

Now let me turn to a brief summary of the HOPE-3 trial, the top-line results of which were released in late 2025 and is one of the strongest data sets generated in this disease to date. The entire HOPE-3 data set was submitted to the FDA as the response to our CRL and was contained in our CSR. These data will now serve as the foundation for potential approval as well as for the preparation for commercial launch. For those of you who have not been following our story, here is a brief recap of the HOPE-3 clinical trial. HOPE-3 is a pivotal Phase 3 multicenter, randomized, double-blind, placebo-controlled study evaluating deramycin in the treatment of Duchenne muscular dystrophy cardiomyopathy.

The study enrolled 106 patients and met its primary efficacy endpoint on the Performance of the Upper Limb, otherwise known as the PUL, as well as all Type 1 error-controlled secondary endpoints. The key secondary endpoint of left ventricular ejection fraction showed a 91% slowing of disease progression in all evaluable patients regardless of cardiac disease status and importantly achieved statistically significant results. Furthermore, the results were even stronger in specific patients with a diagnosis of cardiomyopathy, achieving a p-value of 0.01.

Over the last decade, it has become apparent that cardiomyopathy is one of the leading causes of mortality in Duchenne, and stabilizing cardiac function has remained a major unmet need with current guideline-directed care to include standard cardiac medications which are somewhat effective but do not work long term and certainly are not addressing some of the root causes of the cardiac dysfunction. The statistically and clinically significant preservation of left ventricular ejection fraction in patients treated with deramycin observed in HOPE-3 underscores the potential of deramycin to address the DMD-associated cardiomyopathy.

In addition to the earlier reporting of the positive top-line results which I just highlighted, yesterday, we presented additional data from the HOPE-3 trial in a late-breaking oral presentation at the 2026 Muscular Dystrophy Association Clinical and Scientific Conference. This data was of great significance not only from a clinical trial perspective, but to the patient community because it highlights the effectiveness of deramycin in multiple endpoints, all pointing to the direction of stabilization of the disease process associated with DMD. I would like to provide a few highlights here. One of the most important was an improvement in a direct activity of daily living and one that is also correlated with quality of life.

We show that there was a statistically significant improvement in a measure of upper limb function analyzed in a home-based setting using a validated and published patient-reported outcome measure, the DVA, or Duchenne Video Assessment. The DVA was developed by frustrated caregivers and professionals who were concerned that clinic-based assessments did not tell the whole story, especially in a pediatric population. So they developed a DVA to track their sons at home. The measure we specifically used was called EAT 10 BITE, and it is manifest exactly as it sounds and represents not only the ability to self-feed, but also to move one's arm between table and mouth.

Caregivers would video their sons during the task at prescribed times post-infusion of daratumumab and then the videos were analyzed by a core lab and scored based on ability and compensatory measures. The DVA assessment of E10 BITE supports the clinic-based measure of the Performance of the Upper Limb and is supportive of the observed efficacy of deramycin that we have seen clinically. These data will also support payer discussions as it is a measure of feels, functions, survives.

We also showed images of the hearts of a treated patient as opposed to a placebo patient in the analysis of cardiac fibrosis. This is measured by MRI, using a dye called gadolinium that can distinguish between healthy tissue and scar tissue. The data showed that there was significant reduction in fibrosis in the hearts of those that were treated with deramycin compared to placebo. For cardiologists, this is one of the most encouraging aspects of the HOPE-3 data because there is the aggregation of scar that ultimately leads the heart to fail and life to end for those with DMD. These data will also be used in our labeling negotiations.

It is important to begin treating the fibrosis as soon as it is evident, which can be many years before there are functional implications. Remember, the heart is a terminally differentiated organ, so once a cardiomyocyte is lost, it cannot be easily replaced. Therefore, preservation of functional muscle and attenuation of fibrosis is one of the main goals in treating Duchenne cardiomyopathy. We were delighted to share these results with the Duchenne community as one of only four late-breaking presentations at the Muscular Dystrophy Association Conference yesterday in Orlando. The full HOPE-3 dataset has now been submitted for publication in a major peer-reviewed academic journal.

One of the most important features of deramycin is, of course, its safety profile. To date, we have completed more than 800 intravenous infusions of deramycin across multiple clinical studies, and the therapy continues to demonstrate a consistent safety profile. There is evidence of long-term safety in our open-label extension studies. Some of our young men participating in our HOPE-2 open-label extension study have been receiving continuous infusions for up to five years, and with over 100 patients in our collective open-label extension studies at the time. Deramycin offers the potential opportunity for functional stabilization and also a well-tolerated safety profile. Taken together, we believe deramycin will become an important and foundational therapy for the treatment of Duchenne muscular dystrophy.

We believe the HOPE-3 results provide compelling evidence supporting deramycin's potential benefit in Duchenne and further strengthen our confidence in the therapeutic profile of this product candidate. The consistency of the data across both cardiac and skeletal muscle-related measures supports our view that deramycin may offer a differentiated and meaningful therapeutic approach for patients living with this devastating disease.

Turning now to the regulatory pathway, following receipt of the complete response letter in July 2025, we were able to complete our response based on the results from the already completed HOPE-3 trial. Through both formal and informal interactions with the FDA, we aligned that the HOPE-3 data would be sufficient to support resubmission and we have now submitted that dataset in its entirety. The FDA classified the submission as a Class II resubmission and assigned a PDUFA target action date of August 22, 2026. Importantly, at this stage, the FDA has not identified any potential review issues in its communication to the company, which we view as encouraging.

We also expect to be eligible to receive a priority review voucher upon approval of daratumumab. As these vouchers are transferable and can be monetized through sale, they represent a potential source of meaningful capital that could further strengthen our financial position as we execute on our strategy.

At the same time, we continue to make meaningful progress operationally. Our in-house GMP manufacturing facility located in San Diego successfully completed its FDA pre-license inspection in connection with the BLA review process last year. All Form 483 observations were addressed, and the facility is operational and positioned to support a potential initial commercial launch. That facility can meet the commercial demand of approximately 250 patients per year. However, our current plan is to begin stockpiling commercial doses as soon as we finalize our label with the FDA. In addition, we are now well underway with an expansion to the second floor of that same facility, which will add approximately six additional clean rooms.

At full capacity, this expansion is expected to support treatment of approximately 2,500 patients per year or roughly 10,000 doses annually. Our current projections are that the new facility will come online and be able to support commercial manufacturing in late 2027.

Commercial readiness activities are also continuing to advance. We are cognizant that the DMD community is anxiously waiting for approval and launch. Due to the unmet need and our desire to have product to those who need it, our hiring plan is based on preparing across key areas relevant to launch including patient support, market access, reimbursement planning, and physician education. Capricor Therapeutics, Inc. is at a transformational point, and as a result, we are not simply preparing for only the launch of deramycin for DMD, we are building to operate as a world-class commercial biotech company.

That means maintaining a disciplined approach to execution, investing in our pipeline, and ensuring that our infrastructure can support both potential commercialization for DMD and beyond.

On the scientific front, we continue to strengthen the foundation supporting geromycel. In the fourth quarter of last year, we published a peer-reviewed paper in Biomedicine describing germany cells' anti-fibrotic and immunomodulatory mechanisms of action including the release of exosomes and soluble factors that suppress fibrotic gene expression. These findings were reproduced across more than 100 manufacturing labs supporting the biologic, consistency, and potency of the product. As we move toward approval in Duchenne, we are also beginning to lay the groundwork for potential expansion into other diseases, focusing initially on Becker dystrophy while engaging with regulatory authorities in Europe and Japan with the goal of bringing deromyosil to as many patients as possible globally.

Please stay tuned for more updates on this as we move through 2026.

Now let me turn briefly to our exosome platform. The Phase 1 COVID vaccine study under Project NextGen with the National Institutes of Allergy and Infectious Diseases remains underway. Preliminary results indicate the Stealth X vaccine has been well tolerated and demonstrated a favorable safety profile across all doses tested thus far. However, limited neutralization was observed in early results at the tested dose levels, which may reflect prior vaccination or infection in trial participants. Preclinical data in naive and primed animal models continue to support the of the Stealth X COVID vaccine. Final results from the trial, the cellular response data, are expected in 2026.

NIAID has requested exploration of expanded dosing range at higher dose levels and the potential use of adjuvants. At this time, we are evaluating how these options may fit with our broader pipeline development strategy and will provide additional updates as they become available. Importantly, this program demonstrated the safety of Stealthex exosomes and supported the continued development of our broader engineered exosome delivery platform. It also enables us to expand our manufacturing capabilities to support future exosome programs. We are continuously advancing our StealthX platform, focusing on muscle targeting and capable of delivering multiple payloads including siRNA, proteins, small molecules.

The platform is being applied across several therapeutic programs currently progressing toward IND-enabling studies with a target IND filing in 2027.

From a financial perspective, we ended last year in a very strong position. As of 12/31/2025, our cash position was approximately $318,000,000. This balance was significantly strengthened in the fourth quarter through a successful financing completed in late December, which included participation from dozens of new institutional healthcare-focused investors who we believe share our long-term vision for the company. Based on our current operating plan, we believe this capital is sufficient to support the business into 2027. Importantly, this outlook does not include any additional sources of capital including potential product revenue, or the potential monetization of a priority review voucher should we receive one upon approval.

Earlier this week, Capricor Therapeutics, Inc.’s common stock was approved for uplisting to the NASDAQ Global Select Market, NASDAQ's highest listing tier. We believe this milestone further enhances our visibility within the institutional investment community as we move into what we believe could be a transformational period for the company. Overall, we believe Capricor Therapeutics, Inc. enters this next chapter from a position of strength with our BLA under review, positive pivotal clinical trial data, manufacturing commercial readiness underway, additional pipeline opportunities beyond geramycin, and the capital required to execute on our priorities. Most of all, we remain focused on what matters most, bringing forward a potentially transformative therapy for patients and families affected by Duchenne muscular dystrophy.

With that, I will now turn the call over to AJ to review the financial results. AJ?

Anthony J. Bergmann: Thanks, Linda. For a brief overview of our financial position, which Linda summarized somewhat a moment ago, cash, cash and marketable securities totaled approximately $3,181,000,000 as of 12/31/2025, compared to approximately $151,500,000 as of 12/31/2024. In December 2025, we completed a public offering resulting in net proceeds of $162,000,000, and in addition, the company drew down approximately $75,000,000 under our ATM program in December 2025. Revenue for 2025 was $0 compared to approximately $11,100,000 for 2024. Revenue for the full year ended 12/31/2025 was also $0 compared to approximately $22,300,000 for the full year ended 12/31/2024.

As a reminder, our prior year revenue was primarily derived from the ratable recognition of our upfront and developmental milestone payments under our U.S. distribution agreement with Nippon Shinyaku, all of which has now been fully recognized and was recognized as of 12/31/2024. Total operating expenses for 2025 were $29,200,000 compared to approximately $18,800,000 for 2024. Total operating expenses for the full year ended December 2025 were approximately $108,100,000 compared to approximately $64,800,000 for the full year ended 12/31/2024. The year-over-year increase was primarily driven by continued investment in clinical, regulatory, and manufacturing activities as well as infrastructure expenditures supporting our Duchenne program.

Net loss for 2025 is approximately $30,200,000 compared to a net loss of approximately $7,100,000 for 2024, and net loss for the full year ended December 2025 was approximately $105,000,000 compared to a net loss of approximately $40,500,000 for the full year ended December 2024. Based on our current operating plan, as Linda mentioned a moment ago, our financial resource—we believe our available cash, cash equivalents, and marketable securities—will be sufficient to fund anticipated operating expenses and capital expenditures into 2027, and this expectation does exclude potential milestone payments under our agreements with Nippon Shinyaku as well as any strategic uses of capital that are not included in our current base case assumptions.

And with that, we are ready to open the line up for questions.

Operator: Thank you. We will now open for questions. Ladies and gentlemen, we will now begin the question-and-answer session. You will hear a prompt that your hand has been raised. Should you wish to decline from the polling process, please press star followed by the number two. If you are using a speakerphone, please lift the handset before pressing any keys. Our first question comes from the line of Ted Tenthoff from Piper Sandler. Your line is now open.

Edward Andrew Tenthoff: Hi, guys. Sorry about that. I was on mute. So firstly, congrats on all the really exciting progress, you know, this year, this week. Great to see you down at MDA, excited about some new data coming out of that, and, obviously, the BLA acceptance. Are you guys anticipating any AdCom, anything along those lines? And what commercial prep are you doing in preparation for potential deramycin approval? Thank—

Linda Marbán: Hi, Ted. I have to say it was great to see you in Orlando and, you and I have been tracking each other for more than a decade on this. And so I am very proud of what we are accomplishing together. Thank you so much for your years of good support. To answer your question, in terms of an AdCom, I have been getting a lot of questions about that. Certainly, they have not made any moves towards that at this point. I do not think anybody has had an AdCom in about a year. And I do not know if they are going to be putting one in place.

I think with the departure of Vinay Prasad, it is a little bit up in the air as to what is happening within CBER and what their manifest will be. Either way, we will be prepared. Good news about the HOPE-3 data is it is so very strong. That I really would be delighted whether I presented at an AdCom or we directly to PDUFA without it. In terms of your second question, in terms of commercial readiness, look, deromyophil has been in development for a long time. This data is extraordinary.

Skeletal and cardiac disease attenuation and even improvement in those with cardiomyopathy and a product that is very safe and can be foundational and used with pretty much anything else that we can think of that is approved or coming along for DMD. So we are going to be building our own commercial program to support NS at this point. So that we make sure that everything from market access, payers, and all of the other aspects of commercial planning is done with the same precision that we have done the development of deromyosil to this point.

Edward Andrew Tenthoff: That is great. And one quick clarification, if I may. When it comes to the actual label, I know this is something that will be negotiated later in the review process. Do you believe the current label would be for the original cardiomyopathy, DMD cardiomyopathy submission, or would this be now for DMD more broadly? Just appreciate any clarity on that. Thanks.

Linda Marbán: Thanks, Ted. So I think this, again, is the biggest question that we all have. We have broached this with FDA both in formal and informal meetings, really since the issuance of the CRL last July. They have been relatively noncommittal, saying that they will discuss it during labeling. We certainly believe that the data supports a label both for DMD in terms of some the skeletal muscle ramifications related primarily, I would guess, to upper limb loss, which starts very young, and/or to the treatments and attenuation of Duchenne cardiomyopathy. So that is our plan internally. Obviously, we will keep the street updated as we enter into those conversations with FDA.

Currently, we believe that would be the best path forward both for the therapeutic and also for the regulatory pathway.

Edward Andrew Tenthoff: Great. Thanks. Well, either way, a big win. For the boys and for Capricor Therapeutics, Inc. Thanks so much.

Linda Marbán: Thanks, Ted.

Operator: Our next question comes from the line of Leland James Gershell from Oppenheimer. Your line is now open.

Leland James Gershell: Thank you for taking our question and appreciate the updates yesterday at MDA. Just wanted to ask, could you, Linda, refresh us on the import of the two different cohorts of HOPE-3 as you had material that was made at two different facilities. I think in the past, you had said that Cohort B had been more of a regulatory focus. Just wondered where we stand today in terms of how the FDA will consider those two different cohorts and, you know, in the context of the pooled analysis, as they go through their review? And also wanted to ask if you have any expectation around the timing that we should see a peer-reviewed publication of the HOPE-3 data?

Thank you.

Linda Marbán: Well, thanks, Leland. Yeah, I have not thought about the two cohorts in a while, so the FDA has not mentioned it in any conversation since probably 2024 when we decided to, under their recommendation, file the biologic license application for the cardiomyopathy based on the HOPE-2, HOPE-2 OLE and natural history data. You know, they then agreed that we would pair Cohort A and Cohort B and consider them as one trial because of the nonclinical comparability of the product. So they have not talked about it, and we have not talked about it. They have all of the raw data now.

The good news is, and what I feel very confident in is that Cohort B, independent of Cohort A, was statistically significant in both skeletal muscle performance, Performance of the Upper Limb, and in the cardiomyopathy ejection fraction. That would be the more important cohort to look at because that was what their question was originally—was that product comparable. It certainly is comparable in terms of its biologic activity. So we will keep everybody updated if there are any more questions on the cohorts, but as far as we know, they consider one clinical trial, one cohort, and the manufacturing facility here in San Diego passed PLI, so we are manufacturing ready.

In terms of an update on an academic publication, I know you are an academic scientist as well as I was, and we both know that one of the reasons people are in academia is because time is not of essence. So, you know, the academic review is ongoing and we will keep the community updated as soon as it is ready to be published or published.

Leland James Gershell: Great. Thanks very much.

Linda Marbán: Thanks, Leland.

Operator: Our next question comes from the line of Joseph Pantginis from H.C. Wainwright. Your line is now open.

Joseph Pantginis: Hey there. Thanks for taking the question. So two questions, if you do not mind, Linda. So first, I know you have not had labeling discussions yet, but could you just sort of describe a before and after snapshot of—did you have prior labeling discussions ahead of the CRL? And how you think that may be similar or different. And then second, and this is strictly from a devil's advocate standpoint, could you envision any scenario where this might be a conditional approval?

Linda Marbán: So I can answer your second question first because it is easy. No. There is no way this would be a conditional approval. There would be no need for a confirmatory trial on a randomized, double-blind, placebo-controlled trial that has primary and key secondary and Type 1 error-controlled endpoints. I cannot imagine any scenario what they would make it conditional upon. But I will keep you updated on that. In terms of labeling conversations, we did not get that far before the CRL was issued. Last time was actually right before we would have begun them. So we do not have clarity there.

The only tea leaves I can read is that they knew that HOPE-3 was powered and primary efficacy endpoint was Performance of the Upper Limb. They knew that we had filed a cardiomyopathy BLA under the existing data. When they gave the CRL and then we had the Type A meeting, they wanted to see the HOPE-3 data unaltered in terms of its primary. So we believe that they will consider both the skeletal muscle aspects of the disease as well as the cardiomyopathy in the labeling. I suppose, you know, they can ask for anything. It is the FDA. And so we will keep the street informed as we get information ourselves.

My current belief is that the data is very strong. It supports labeling for both cardiomyopathy and skeletal muscle myopathy, and that is what we are planning for internally at this point.

Joseph Pantginis: Appreciate the comments, and here is to the end of the potential FDA drama.

Operator: Our next question comes from the line of Kristen Brianne Kluska from Cantor. Your line is now open.

Kristen Brianne Kluska: Hi, everyone. Thanks for taking the questions, and nice to spend time with the broad Capricor Therapeutics, Inc. team this week in Orlando. So with the Class II resubmission, can you just help us understand what parts of the review are going to be new versus what parts are already considered checked off with the first process—so, for example, like on manufacturing, mid and late-cycle review meetings, etc.? And then just on capacity, wanted to confirm in your prepared remarks you said it could support 250 patients per year, with potential stockpiling, but then you are expanding to reach 2,500 patients per year. What will you need to show to the FDA to get the expansion up and running?

Like, is there any comparability work or runs that you have to do to show them it is the same material, etc.? And then last question for me just on MDA. Obviously, a lot of doctors there. We talked to plenty ourselves, but curious what your takes were from these communications. This is really your big showing of the HOPE-3 data since it came out in December. So curious what the feedback is. Were there people even that were skeptical in the past that, now that you have this data, were willing to take a closer look? Anything you could share would be really helpful. Thanks again.

Linda Marbán: Yeah. So thanks. So this is obviously our first go-around with the CRL and a resubmission. What I can tell you is that we know the manufacturing facility passed pre-licensing inspection. All 483s were signed off on, and so we are good to go there. We anticipate there will be several CMC-related questions that come across as we go through this resubmission process just because there were a few loose ends, none of them that were areas that would be a major concern or slow things down. They just want clarification. We think that the nonclinical and other aspects of the BLA have already been signed off on, so we do not anticipate any changes there.

Obviously, the only thing that was really cited in this complete response letter that was now officially resubmitted was the HOPE-3 data. So we assume that clinical and stats will be the focus of the new review. Yeah. So, we very strategically built the new clean rooms in the same building as the 250-capacity clean room. So it does reduce the regulatory requirements if it is on the same street address as the original facility. You obviously have to demonstrate, you know, in PPQ runs that the product is the same and passes all of your requirements. I think they come and do another inspection, but they would be slated to do an inspection in early 2027 anyway.

As part of, you know, there is general maintenance on manufacturing plants. So I am not anticipating a long run-in terms of getting approval of the site based on sort of those components or that situation. But we will obviously keep you updated as to how that goes. I know Marty Makari has spoken publicly, and I know Vinay Prasad prior to his exit also spoke publicly that they were thinking they would reduce the number of PPQ runs that are necessary from three to one, which, obviously, if that actually is put into place, could significantly reduce the time that a manufacturing facility would need to come online.

So we are going very fast and anticipate getting those doses out to commercial community as quickly as the FDA will allow us. Yeah, thanks, Kristen. And let me just say it was wonderful to see you in Orlando, and I appreciate you turning out and spending some time with our team. The event we hosted was exactly what I had hoped for, which is that physicians and investors and patients and everybody could be together to learn about dirhamia cell. And you are correct.

You did speak to physicians who I think are echoing now what you just alluded to, which is that the HOPE-3 data has solidified belief in this product across physicians, across patients, really across the entire community. It is, you know, I have said now a few times, randomized, double-blind, placebo-controlled, hits primary endpoint, hits secondary endpoints, hits Duchenne video assessments, which went along with the Performance of the Upper Limb, as I said in prepared remarks. And so yes, I think physicians who before were hopeful are now convinced and looking forward to putting their patients on. We are getting a lot more questions about prescribing availability, launch than we ever have.

So we are on fire here getting this product ready for approval and for launch.

Kristen Brianne Kluska: Thanks, Linda.

Linda Marbán: Thanks, Kristen.

Operator: Our next question comes from the line of Madison El-Saadi from B. Riley Securities. Your line is now open.

Madison El-Saadi: Hi, Linda and team. Congratulations on the data, and thanks for taking our question. Your partner has previously said that they expect to transition all clinical trial patients to commercial drug within one quarter of launch. So should we think of these, call it, 100 patients as kind of the base case for how many patients may be treated with deromyosil in 2026, assuming approval? And then to follow that, as you know, there are 7,000 to 8,000 DMD boys, maybe more, with cardiomyopathy, and just given the data we saw in this subgroup, you know, it is hard to imagine there being a circumstance in which a patient does not go on this drug.

I guess, how do you scale beyond the 2,500 capacity? Is that something you guys are thinking about? What would it cost—do you have the cash? Maybe if you could just kind of help illustrate what that road map may look like. Thanks.

Linda Marbán: Yeah. Madison, thanks so much, and also great seeing you in Orlando. Thanks for making the trip. Always great to spend time together. So in terms of the OLE patients, yes, we have over 100 OLE patients on daratomycinol now. They all will be anxious to continue. We have seen that from the HOPE-2 OLE guys that have gone on for years. We anticipate all of them wanting to come over to commercial product. We have not figured out a launch date yet, we just got the PDUFA date. So I do not want to give a year or a timeline as to when, but yes, we will transfer all of them over as seamlessly as possible.

That is why we are focusing internally on access at this point so that can happen seamlessly. There are, Madison, a lot of young men that have been waiting in the wings for deramycin that did not qualify for our trials for whatever reason. And I am getting a lot of calls now from—so we will prioritize getting geromyosil to any and all of those that need or as quickly as possible. And I will say that is my mandate and why we are taking on manufacturing as aggressively as we are.

To that end, pertinent to your question, yes, we are now poised and, in fact, ready to go forward with another manufacturing build-out in San Diego County very close to our current footprint that will be able to accommodate many more thousands of patients per year. We wanted to make sure that we completed our response. We want to make sure that we are proceeding well towards PDUFA before we invest that capital. But we now have internal confidence that will happen. So we are actively planning to expand manufacturing to accommodate the needs of any and all of those that would like to have it. At diromycin.

Madison El-Saadi: Got it. Thanks.

Linda Marbán: Thanks, Madison.

Operator: Our next question comes from the line of Catherine Clare Novack from Jones Trading. Your line is now open.

Catherine Clare Novack: Hi. Good afternoon. Thanks for taking my question. One thing we heard over and over at the meeting was about how patients with DMD do better with earlier intervention. Just thinking about how you can make the case based on HOPE-III that it is a benefit to treat, you know, even before the development of cardiomyopathy, thinking that, you know, since virtually all DMD patients will eventually develop cardiomyopathy, and not thinking of it as then a separate indication, but as part of DMD as a whole. You know, what in the application supports that? And then can you remind us of the status of the European rights deal with NSF?

Linda Marbán: Yeah. Thanks. You know, we, of course, are laser focusing on those younger kids and those earlier in the disease process. As we have said and sort of have been stating for a long time, it is very safe. The infusion protocol is really easy. Even a little guy could sustain it very well. And, yes, the data that we have seen has been highly supportive of starting as young as possible. Getting a prevention label is very difficult until you can show prevention, which takes years. We are comfortable right now with the treatment of cardiomyopathy. The good news is because these kids now, most of them start getting MRI at a very young age.

As soon as they see one segment of scar, the cardiologists want to get them on deromyophil, and so that will be a way that we will get more and more active in sort of the younger kids and moving towards that prevention target. Of course, if they go on for the attenuation of skeletal muscle function myopathy, then we will be able to track their hearts and be able to ultimately—physicians will use it with skeletal muscle as well as cardiomuscle myopathy independent of progression of the disease. So we have been negotiating with NS Pharma for a while for rights to Europe. Honestly, we have not been focusing on it internally.

There was clearly a lot going on here with the CRL and getting the HOPE-3 data ready and submitted. And now that we have a PDUFA date, and I feel like we are on a good pathway there, we can take a little bit of a breath and start focusing on our outside-of-U.S. activities. They do have the rights to Japan, so we are going to start working with PMDA and getting that going in 2026. And then in terms of Europe, we are evaluating those now, and we will see sort of where the road map takes us. And we will provide updates as they become available.

Catherine Clare Novack: Got it. Thank you. It was great seeing you and great hearing all these updates. Looking forward to the year.

Linda Marbán: It was great to see you as well. Stay well, and see you soon.

Operator: Our next question comes from the line of Gubalan Pachayapan from ROTH Capital. Good afternoon, team, and thanks for taking our questions. So a couple from us. Your line is now open.

Gubalan Pachayapan: Firstly, you mentioned the Duchenne Video Assessment. Can you maybe tell us how many patients were included in the deramio cell arm and how many in the placebo arm? And also related to that, can you also comment on the inter-rater reliability of DVA? Because it is my understanding that it is rated by both caregivers and professionals. And then is there a reason why the sample size is low for the late gadolinium enhancement secondary endpoint? And maybe one last question from me. So the most recent PRV, as you probably know, was sold for a very high price of $205,000,000. And we are also aware that there is a new sunset date, which is 09/30/2029.

But do you think because the new sunset date is a little longer than three years from now, this is going to ease up some pressure from the buyers—maybe that could impact the price that you will be selling your PRV for? Any thoughts on that? Thank you.

Linda Marbán: That is right. So the DVA is actually a qualified and validated assessment tool that has been published, and not only been used by us but by others and not only by those with Duchenne muscular dystrophy but in other disease states. So it really is quite rigorous in its measurements. The recorders, or the video takers, are trained in how to do it, what to do, then they are sent to a facility where they are read by a blinded, trained reader and the data is then delivered blinded to the company and ultimately treated like any other data set. So it really is highly valuable data that is collected in a home-based setting.

In terms of the number of patients, that were in the DVA was about 50 patients in each group, so 50 in the treatment and 50 in the placebo group. Yeah. So, we added LGE measurement for Cohort B only. When we were designing Cohort A, there had been some press around gadolinium and the fact that it might aggregate in the brain, especially of young children, and could be a safety—so we decided not to look at scar in Cohort A. During the time between Cohort A and the initiation of Cohort B, that was considered not to be a safety risk.

And the value of the data collected would be highly necessary to sort of show the correlation between function and scar. The data is beautiful, and having been somebody that has worked in MRI for many decades, I am really excited by this data as are the cardiologists. So it is only those in Cohort B that were eligible for the gadolinium enhancer, and then they also had to have certain levels of kidney function. So that is why those numbers are relatively small. But that dataset is small but mighty. Yeah. If I had a crystal ball, I would be a really wealthy woman. But all that being aside, I do not know.

I certainly know that PRVs tend to be valuable. It really depends on how motivated the buyer is to get it when they come available, and we certainly are going to get the maximum price for our PRV should we be able to receive one.

Gubalan Pachayapan: Alright. Congratulations. Thank you. Thanks.

Operator: Our next question comes from the line of Matthew J. Venezia from Alliance Global Partners. Your line is now open.

Matthew J. Venezia: Hi, guys. Congrats on the progress, and thanks for my questions. First, I think I asked this question about six months ago, but how have the conversations at the FDA with you guys changed, if at all, since being—Prasad left the agency once again? And another talking about the age of the patient, it seems to be a drug where early intervention and a disease where early intervention is paramount to treatment. Do you expect a specific age on your label? Do you expect that to come up in labeling discussions with the FDA? I know, like, you guys had four plus on their label.

But is that something that you expect to be ironing out with the FDA in your labeling discussions? And just the final question, the StealthX platform—is there a specific time within second quarter when we can expect P1 trial results, or is that kind of just up to NIAID?

Linda Marbán: Thanks, Matthew. Good to talk to you. So far, nothing. We got our letter reopening the file, setting our PDUFA date, and that was almost in conjunction with his leaving. So, really, we have had no change in any interaction whatsoever at this point. Yeah. I do not know. We did not ever have a four plus. I do not know who that was. That was not us. Age is a possibility. We have treated down to age eight in our clinical trials. So we have not gone younger than that. We do not know, again. You know, I know everybody is hypothesizing about labeling discussions to build their models, and I certainly am doing the same myself.

I do not know if there will be an age cutoff or a function cutoff. As soon as we have clarity, though, we will let you know. I would say in building your models that the youngest we have actually treated to this point are those that are ambulant and down to age eight. Yeah. It is—I was just going to say you take the words from my mouth. It is an NIAID situation, so that data trickles in really slowly. We are super anxious to see the cellular response. We are really interested to see if there is a T-cell response.

COVID is kind of a crazy virus to try and get on top of these days because pretty much everybody is either had it or been vaccinated multiple times. And so we are looking forward to seeing that data to continue to work with NIAID. But most importantly, and I will take—thank you so much for asking me—we are very excited about our pipeline right now. We now have the opportunity to deploy on it. We were able to build out manufacturing for this vaccine. We know how to do it now. We know how to, you know, load the exosomes, target the exosomes.

And so while the vaccine program is important, it really was that learning experience that is now driving us towards a therapeutic exosome platform, and stay tuned for more information on that as we progress through 2026.

Matthew J. Venezia: Alright. Wonderful. Thank you, guys. Again, and congrats on the progress.

Linda Marbán: Thanks.

Operator: I will now turn the call back to Capricor Therapeutics, Inc. management for closing remarks. Please go ahead.

Linda Marbán: Thank you so much for joining us today. Thank you for those of you that attended the Muscular Dystrophy Association and took some time to be with Capricor Therapeutics, Inc. at that event. I will say that it gave me incredible joy and pride to be at that event and see how prominently Capricor Therapeutics, Inc. was featured at MDA, but also in the hearts and minds of those with DMD. We really feel like we have the opportunity now to meaningfully improve their lives and look forward to continue on that journey with them and with all of you. So we look forward to seeing you out in the community, and thank you so much.

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

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