Capricor reports $25.9 million net loss in Q2 2025; FDA CRL prompts strategic shift for Daramycel approval path.

OPERATOR - (00:00:00)

Good afternoon ladies and gentlemen and welcome to Capricor Therapeutics second quarter 2025 conference call. At this time all participant lines are in listen only mode. Following the presentation, we will conduct a question and answer session. If at any time during this call you require maintenance assistance, Please press the star zero for the operator. This call is being recorded on Monday, August 11, 2025. I would now like to turn the conference over to CFO AJ Burbank for the forward looking statements. Please go ahead.

AJ Burbank - Chief Financial Officer - (00:00:32)

Thank you and good afternoon everyone. Before we start, I would like to state that we will be making certain forward looking statements during today's presentation. These statements may include statements regarding, among other things, the efficacy, safety and intended utilization of our product candidates, our future research and development plans, including our anticipated conduct and timing of pre clinical and clinical studies, our enrollment of patients in our clinical studies, our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory developments involving our product candidates, potential regulatory inspections, revenue and reimbursement estimates, projected terms of definitive agreements, manufacturing capabilities, potential milestone payments, and our financial position and possible uses of existing cash and investment resources. Forward looking statements are based on current information, assumptions and expectations that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward looking statements. These and other risks are described in our periodic filings made with the sec, including our quarterly and annual reports. You are cautioned not to place undue reliance on these forward looking statements. We disclaim any obligation to update such statements. With that, I'll turn the call over to Linda Marban.

Linda Marban - Chief Executive Officer - (00:01:43)

Thank you AJ Good afternoon everyone and thank you for joining us on our second quarter conference call at Capricor Therapeutics. Our mission remains unchanged and clear to bring transformative therapies to patients with rare and life limiting diseases. While this past quarter has presented us with some unique challenges, it has also reinforced our conviction that we have in Daramycel for the treatment of DMD, the agility of our team and of course the promise of our pipeline, in particular for the Duchenne community. We remain unwavering in our commitment to deliver the first approved therapy aimed at specifically treating the cardiomyopathy that affects nearly every patient with Duchenne Muscular Dystrophy and remains the leading cause of death in this devastating disease. Now for the latest update on our Biologics License ApPLIcation or BLA. As previously disclosed, we received a complete response letter from the FDA in July. The CRL stated that the BLA in its current form does not meet the statutory requirements of for substantial evidence of effectiveness and also referenced certain CMC items, most of which we had already responded to but which were not reviewed by the FDA due to the issuance of the CRL. As a reminder, approximately one year ago in our pre BLA meeting with the FDA, we requested to switch the primary efficacy endpoint of our ongoing Phase 3 Hope 3 study to left ventricular ejection fraction or LVEF and the agency responded by encouraging us to submit on currently available Data from our Hope 2 and Hope 2 open label extension trials matched to an external natural history comparator and then use the data from our Hope3 trial to support potential label expansion and in the future this became the plan that we implemented. While the FDA response contained in the CRL was certainly disappointing, we stand behind the strength of our submission and the substantial progress made throughout the review process. From a successful pre license inspection or PLI to completion of our mid cycle review with no deficiencies noted and timely responses to more than 50 information requests from the FDA, we believe we consistently met the agency's expectations throughout the review process. The complete response letter was unexpected given the trajectory of positive interactions While the FDA continues to evolve under new leadership and its approach to novel therapies to treat rare diseases, we remain focused on working constructively with the FDA to define the clearest and most efficient path forward for Daramycel and the patients who need it. I would like to emphasize that our Hope 3 trial is still blinded and will not be unblinded until we have clarity on the path to potential approval from FDA. Let me take a minute to remind you of the features of the Hope 3 trial. The study is fully enrolled with the last patient last visit occurring in June of this year. HOPE three is a double blind placebo controlled clinical trial with a one to one randomization which enrolled 104 patients consisting of two arms, cohort A and Cohort B. The combined power of this trial using both cohorts is greater than 90%, with the original primary efficacy endpoint being the performance of the upper limb or the pull version 2.0. Based on a multitude of reasons, not the least of which is the tremendous unmet need of DMD cardiomyopathy. We have submitted a protocol amendment to designate left ventricular ejection fraction or LVEF as the primary efficacy endpoint and the skeletal muscle endpoint performance of the upper limb or pull as a prespecified secondary endpoint. This change is based on multiple factors. One the objectivity of LVEF as measured by cardiac mri. Remember, there is no volition in cardiac function as measured by mri as well as the relevance of left ventricular ejection fraction to the pathophysiology of DMD cardiomyopathy, which has only been recently elucidated by the work of Dr. Jonathan Soslo from Vanderbilt University and the DMD Cardiac Consortium in a study funded by the Office of Orphan Products of the FDA and the NHLBI. In addition, Hope3 is well powered to detect the treatment effect on cardiac function. I want to remind you that Capricorps developed Daramycel specifically to address heart disease, particularly the cardiomyopathy associated with DMD. However, until Dr. Soslow's study and its subsequent publication, there were no established efficacy benchmarks in DMD cardiomyopathy for the FDA to use in defining clinical benefit. Our entire regulatory path, including the current BLA, was built on the FDA's guidance on how efficacy should be defined in this patient population. We have always intended for ejection fraction to serve as our primary efficacy endpoint, so while this may appear to be a change in strategy, it is in fact a return to the original goals we set early in the development of Daramycel. To that end, with our Type A meeting with the FDA now scheduled, we have submitted a comprehensive briefing package that addresses the concerns raised in the CRL and outlines several potential paths to approval. These include, first and foremost, the continued review of our previously filed BLA, which we believe meets the apPLIcable regulatory requirements for approval, as well as supplementing the current BLA with additional data from Hope3 if needed. We believe the current handling of our submission is inconsistent with our interpretation of the FDA written guidances for cell and gene therapies as well as recent public statements addressing the approval of safe and effective therapies for rare disease populations. We are hopeful that FDA will exercise a patient focused and science driven approach in rare disease approvals in which they have been emphasizing in the media as well as highlighting in the FDA Direct podcasts. Based on the comments of Secretary Kennedy and Commissioner Makari, approving Daramycel for the treatment of DMD cardiomyopathy seems directly in. Line with their goals. In conclusion, about a year ago we received FDA feedback that shaped our decision to submit the BLA based on cardiac endpoints. We provided the requested data and analyses and fully expected any differences in interpretation to be addressed at an advisory committee meeting, one that was ultimately canceled by the FDA without explanation. We are concerned with how our file has been managed because we believe there were opportunities during the review period for the agency to raise the specific issues cited in the CRL before issuing the letter. We have long worked aside the DMD community and understand their calls both to continue treatment with Daramycel and to gain access if it becomes commercially available. We will continue to urge the FDA to recognize that cardiomyopathy is a leading cause of death in DMD and a far more severe consequence than the loss of arm function. Daramycel has demonstrated a strong safety profile and the data indicate it can help stabilize the inevitable decline in cardiac function for people living with dmd. With regard to the CMC and PRE commercial aspects of our program, I am pleased to announce that the FDA has now formally accepted all 483 items from our pre license inspection. This milestone further validates the strength of our quality systems, manufacturing capabilities and overall commercial readiness. In addition, the CMC related items noted in the CRL have either been addressed prior to the issuance of the CRL or have been internally addressed. Since we have prepared formal responses which we plan to submit with our response to the CRL. Our manufacturing facility in San Diego remains fully operational and in production and we are being disciplined in our commercial manufacturing investments to ensure we are fully prepared while managing resources wisely. In parallel, we are diligently and strategically investing in launch readiness activities including physician education, patient services, market access planning and reimbursement. We've also begun working closely with treating physicians across the field of neurology and cardiology who will ultimately collaborate in prescribing Daramycel to patients with DMD cardiomyopathy approved. Many of these clinicians are already familiar with the therapy through their participation in the Hope 2 and Hope 3 studies and we are committed to ensuring a smooth transition to commercial use if approved. At this time, we are focused on seeking approval for Daramycel in the US and with respect to our global expansion plans, we will provide updates as they become now turning to our Exazome program to remind you, in 2024 we were selected to participate in Project NextGen, an initiative led by the US Department of Health and Human Services aimed at advancing next generation vaccines for COVID 19 and other potential infectious diseases. Under this program, the National Institutes of Allergy and Infectious Disease, NIAID will be sponsoring the Phase 1 clinical trial of our Stealth X vaccine within the last several weeks. We reached a significant milestone for this program which was the clearance of the IND and initiation of the trial using Stealth X, our Exosome platform technology. The phase one study is being conducted and overseen by NIAID's Division of Microbiology and Infectious Disease, DMID and I am pleased to report we have already supplied them with our clinical material for use in the trial. The phase one study is assessing our COVID 19 vaccine product. The trial is divided into three arms comprised of three escalating doses of the spice spike or S antigen and a combined high dose S plus the nucleocapsid or N antigen. The multivalent vaccine we have been developing. NIAID is starting with the S first because previous Covid vaccines are mainly S based and they wanted to have a basis for comparison with our vaccine candidate using similar antigenic profiles. The end goal is for the adoption of the N S, which is our multivalent vaccine, and we will provide more updates on this developing program as they become available. We believe that Stealth Acts has the characteristics of a vaccine product that Secretary Kennedy would find acceptable. It contains no adjuvants, it is not MRNA based, uses a native protein antigen and can be rapidly produced if needed. We have long believed that this type of vaccine checks all the boxes for a safe and effective platform as supported by multiple preclinical studies and upcoming clinical data will allow us to confirm or challenge that hypothesis. This platform also has the potential to address multiple disease areas, including influenza and and rsv. While vaccines are not a core focus for us, if our candidate meets US Government criteria and demonstrates efficacy, it could potentially open meaningful business development opportunities. Just as importantly, it would serve as strategic proof for Exosome Platform, which we hope to advance as a versatile therapeutic engine for rare diseases and beyond. While a majority of our efforts this year have been focused on securing approval for Daramycel, an additional reason we recruited Dr. Michael Binks as our Chief Medical Officer was his expertise in translational science and medicine. He is now leading efforts to advance our Exosome pipeline with the goal of forging strategic partnerships to expand the platform into and beyond vaccines. We believe the differentiated features of Exosomes, including low immunogenicity, scalable manufacturing and targeted delivery, position Capricor for unique potential opportunities in the therapeutic delivery space. We look forward to sharing updates as they become available. Thank you. And with that I will now turn the call over to AJ to run through our financials. Thanks Linda.

AJ Burbank - Chief Financial Officer - (00:15:19)

This afternoon's press release provided a summary of our second quarter 2025 financials on a GAAP basis. You may also refer to our quarterly report on Form 10Q, which we expect to become available shortly and will be accessible on the SEC website as well as the financial section of our Website. Let me start with our cash position. As of June 30, 2025, our cash, cash equivalents and marketable securities totaled approximately $122.8 million. Turning into the financials, revenues for the second quarter of 2025 were 0, compared to approximately 4 million for the second quarter of 2024. Additionally, revenues for the first half of 2025 were 0, compared to Approximately 8.9 million for the first half of 2024. I'd like to point out that the source of revenue for 2024 was the ratable recognition of the $40 million we had received under our U.S. distribution agreement with Nippon Shinyaku, which has been fully recognized as of December 31, 2024. Moving to our operating expenses for the second quarter of 2025, excluding stock based compensation, our research and development expenses 20.1 million compared to approximately 11.7 million for Q2 2024. For the first half of 2025, excluding stock based compensation, our research and development expenses were approximately 36.3 million, compared to approximately 21.8 million for the first half of 2024. Moving into general and administrative expenses, excluding stock based compensation were approximately 4 million in Q2 2025 and approximately 1.8 million in Q2 2024 and for 1H25, also excluding stock based compensation, our general Administrative expenses were approximately 7 million for 1H25 and 3.6 million for the first half of 2024. Net loss for 2Q25 was approximately 25.9 million, compared to a net loss of approximately 11 million for 2Q24, and net loss for 1H25 was approximately 50.3 million, compared to a net loss of approximately 20.8 million for the first half of 2024. I will now turn the call back over to Linda for some closing remarks.

Linda Marban - Chief Executive Officer - (00:17:35)

Again, thank you, AJ. Just to reinforce AJ's point on our financial position, with over $120 million in cash, we are well positioned to support operations into late 2026 and continue to advance our key pipeline objectives. Additionally, if we receive approval, we would still be eligible to receive a priority review voucher as well as a milestone payment of $80 million from NIFON Shinyaku, representing additional significant non dilutive capital opportunities to further strengthen our balance sheet. This is an important moment for Capricorn. While we have faced recent regulatory headwinds, we are advancing deliberately, strategically and with confidence in our data, our team, and our path forward. We continue to believe that Daramycel represents a major step forward for patients with DMD cardiomyopathy and that our Exosome platform is well positioned to deliver value through continued innovation and partnerships to the Duchenne Muscular Dystrophy community. Thank you for your ongoing trust and support. We remain grounded in the science on execution and committed to building a company that delivers meaningful and lasting impact for all DMD patients. I will now open up the line for questions.

OPERATOR - (00:18:51)

Thank you, ladies and gentlemen. We will now begin the question and answer session. Should you have a question, please press Start followed by the number one. On your touchstone phone, you will hear a prompt that your hand has been raised. Should you wish to decline from the polling process, Please press the star followed by the numbers 2. If you are using a speakerphone, please leave the handset before pressing any keys. One moment please, for your first question. Your first question comes from Ted Tenhoff from Duiper Sandler. Please go ahead.

Ted Tenhoff - Equity Analyst - (00:19:28)

Great. Thank you so much for taking my question and I appreciate all of your hard work to keep fighting for these boys and get them a therapy that's going to help them with their heart function. I wanted to get a sense for sort of the plan for next steps in terms of unblinding Hope three is the plan to sort of get confirmation from the FDA on that first just maybe sort of reiterating what the plan is. Thanks.

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Now for the latest update on our biologics license application, or BLA.

Linda Marban - Chief Executive Officer - (00:20:11)

Thanks Ted. Always a pleasure. Even during these crazy, crazy times. Yeah. So, you know, we are waiting for adjudication from FDA as to what their requirements will be for Hope 3 and then we will submit a statistical analysis plan and proceed with unblinding only after that has been accepted. We just don't want to, you know, muddy or cloud the waters with any thoughts that we had unblinded early. So our plan is to stay quiet until we have plans from them. Great.

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As previously disclosed, we received a complete response letter from the FDA in July. The CRL stated that the BLA and its current form does not meet the statutory requirements for substantial evidence of effectiveness.

Ted Tenhoff - Equity Analyst - (00:20:44)

And when do you expect to hear back from the FDA or get that clarity?

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And also reference certain CMC items, most of which we had already responded to.

Linda Marban - Chief Executive Officer - (00:20:50)

Well, obviously from the rescheduling of our earnings call today, our type A meeting is this week. We anticipate to have a really good conversation with the fda. We're looking forward very much to meeting with them. We certainly August is our month, August of 2020, when this whole plan was put in place. So I'm very excited for this meeting and in terms of providing clarity and updates to the markets, that probably won't be until I get the official feedback in writing, especially with the liability of our current situation and times. So hopefully Adam Feuerstein won't report on it before I do.

Ted Tenhoff - Equity Analyst - (00:21:29)

Thanks so much, Linda. Good luck.

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Which were not reviewed by the FDA due to the issuance of the CRL.

Linda Marban - Chief Executive Officer - (00:21:32)

Thanks Ted.

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As a reminder, approximately one year ago.

Leland Gershel - Equity Analyst - (00:21:36)

Thank you. Your next question comes from Leland Gershel from Oppenheimer. Please go ahead. Thanks Linda, for the update and taking our questions. There's just a couple here, so just maybe further from Ted's inquiry. So if you go with the plan to not unblind Hope 3 but continue with the current bla as it is, would there then be supplemental OLE data that could go into what the FDA has on file versus what had been submitted? I guess if you could share just what incremental data except Hope 3 that could become available to them that would be different from what they had reviewed previously.

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In our pre bla meeting with the fda.

Linda Marban - Chief Executive Officer - (00:22:21)

Yeah, Leland. So actually I don't have really thought about submitting supplemental OLE data though of course it continues to support our safety profile and the efficacy. If you look at the long term efficacy of Daram isl, it's actually quite extraordinary and we're very proud of that record in our OLE patients. And I don't know of another clinical effort in DMD that has as long of a record post study as we do. Having said that, I think the meeting with FDA will define what they will want for data for either the reopening and resubmission of this bla. And so I don't have an answer on that. My current plan is to have everything ready. Then they have an opportunity to select what they think would be most efficient in determining efficacy for approval.

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We requested to switch the primary efficacy endpoint of our.

Leland Gershel - Equity Analyst - (00:23:09)

Okay. And I guess, I guess I have to ask, you know, with Vinay Prasad now back, you know, at cber, you know, how does that inform your thinking is, you know, are the people who you're interacting with at fda, is there a different team now with Nicole Verdun, you know, out of the picture since, you know, the last few weeks, how should we think about kind of who your kind of counterparties are at FDA at this point?

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Ongoing. Phase three hope three study to left ventricular rejection fraction, or LVEF and the agency responded by encouraging us to submit on currently available data from our hope to and hope to open label extension trials matched to an external. Natural history comparator and then use the data from our hope three trial to support.

Linda Marban - Chief Executive Officer - (00:23:40)

Thanks, Leland. You know, it's interesting. I think the last few years I've been thinking about this a lot. People have become much more focused on who the review team is and exercising political capital and regulatory flexibility and all of this lingo that has become very popular. I'm going back to old school. We have good clinical data, we have great safety data. We have guidance from the agency in writing as to what they wanted. We provided it. And now really, I think it's up to them to decide who is best suited within that agency to make the decision of adjudication and all of the factors that go into it. So I tell my team, I'll tell the markets, we are Proud of our data. We see it in terms of the long term efficacy in our patients and safety and we see, sincerely hope that the agency gives us a good path forward to get this to those patients as quickly as possible.

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Potential label expansion in the future.

Leland Gershel - Equity Analyst - (00:24:35)

Okay. All right. Thanks very much for taking the questions.

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This became the plan that we implemented.

Linda Marban - Chief Executive Officer - (00:24:38)

Thanks, Leland. Always a pleasure.

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While the FDA response contained in the CRL was certainly disappointing, we stand behind the strength of our submission and the substantial progress made throughout the review process from the successful prelicense inspection. Or pli?

OPERATOR - (00:24:41)

Thank you. The next question comes from Joe Pantkinis. From H.C. wainwright, please go ahead.

- (00:04:07)

To completion of our mid cycle review with no deficiencies noted and timely responses to more than 50 information requests from the FDA. We believe we consistently met the agency's expectations throughout the review process.

Joe Pantkinis - Equity Analyst - (00:24:48)

Hi Linda and A.J. thanks for taking the questions. Good afternoon. So just to sort of press the envelope a little bit, obviously things could change incrementally or dramatically, you know, this week and about a month after that when you provide the details from the minutes to the street. But you know, with language in the press release and what you talked about today, you know, you're talking about, you know, resubmitting in its current form, you're saying maybe not really having any incremental data from the ole that you just discussed in the last question. So what would you expect that could be potentially different?

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The complete response letter was unexpected given the trajectory of positive interactions. While the FDA continues to evolve under new leadership and its approach to novel therapies to treat rare diseases, we remain focused on working constructively with the FDA to define the clearest and.

Linda Marban - Chief Executive Officer - (00:25:25)

Well, again, you know, we have given the FDA a variety of opportunities in our briefing document. We remain open to resubmission of the BLA as it is. We didn't feel that the CRL was founded. The data that we submitted was exactly what they asked for. And the graphs themselves are interpretable as statistically and clinically significant. So that's of course our number one goal is, you know, explaining to them why perhaps their interpretation was wrong. There's then everything that follows from that accelerated approval with the submission of Hope3 data in support of that and a variety of other opportunities that they can help us adjudicate. And that's why we're looking forward to this type A meeting. So with that in mind, we go in with open hearts to meet with the agency and look forward to having them understand that developing and approving therapeutics for rare diseases, especially Pediatrics 1 does require you're looking at the data in a more holistic fashion. And we're hoping that that is exactly what happens this week.

Joe Pantkinis - Equity Analyst - (00:26:31)

All right, so that's fair. And so maybe just another question starting at the or from the back end of your comments around Stealth X. So it's great that it's getting into the clinic now. Any visibility with regard to what might be next with regard to an indication, Obviously you mentioned influenza as a potential. And how would you describe the early talks maturity levels of potential BD around Stealth X?

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Most efficient path forward for dermyophil and the patients who need it.

Linda Marban - Chief Executive Officer - (00:27:00)

Yeah, thanks Joe. So, you know, our Stealth X program is sort of our little engine that could, you know, we just kept on moving it forward. Our vaccine program is really exciting in the sense that, as I mentioned in my remarks, this type of vaccine, which we've always believed in, is exactly what Secretary Kennedy has advocated would be a, you know, a much better vaccine candidate. No adjuvants, native proteins, rapidly produced, no mRNA, that kind of thing. So we're very excited about that. We're looking forward to the NIAID data. They're very excited about it because it's a program that does fit that criteria and we'll see where that goes. In terms of Capricor's interest in developing vaccine technology, that's something that I've always said would be a business development opportunity. We think it's riper than ever based on the criteria I just put forth. In terms of therapeutic indications, we haven't disclosed some of the ones that we've been working on internally. As I've mentioned many times, we've focused most of our efforts and our capital on Daramycel and look forward to providing updates on where we're going to be taking the Exazone program as we further develop that therapeutic pipeline. Got it.

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I would like to emphasize that our hope three trial is still blinded and will not be unblinded until we have clarity on the path to potential approval from sea, let me take a minute to remind you of the features of the hope three trial. The study is fully enrolled. With the last patient. Last visit occurring in June of this year. Hope. Three is a double blind, placebo controlled clinical trial with a one to one randomization. Which enrolled 104 patients consisting of two arms, Cobra A and cohort b. The combined power of this trial using both cohorts is greater than 90%.

Joe Pantkinis - Equity Analyst - (00:28:08)

Looking forward to more visibility out of the program. And good luck with the meeting this week.

Linda Marban - Chief Executive Officer - (00:28:13)

Thanks, Joe. Always a pleasure.

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With the original primary efficacy endpoint being the performance of the upper limb or the pull version. 2.0.

OPERATOR - (00:28:17)

Thank you. Your next question comes from Kristin Kluska from Cantor. Please go ahead.

- (00:05:37)

Based on a multitude of reasons, not the least of which is the tremendous unmet need of DMD cardiomyopathy. We have submitted a protocol amendment to designate left ventricular ejection fraction, or LVEF, as the primary efficacy endpoint and the skeletal muscle endpoint performance of the upper lamb. Or pull as a prespecified secondary endpoint.

Kristin Kluska - Equity Analyst - (00:28:23)

Hi, Linda and AJ Also sending you. Best wishes for your meeting this week.

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This change is based on multiple factors. One, the objectivity of LVEF, as measured by cardiac mRI. Remember, there is no volition in cardiac function.

Kristin Kluska - Equity Analyst - (00:28:29)

Few questions for me.

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As measured by MRI, as well as the relevance of left ventricular ejection fractions. To the pathophysiology of DMD cardiomyopathy, which has only been recently elucidated by the work of Dr. Jonathan Saflow from Vanderbilt University and the DMD cardiac consortium in a study funded by the office of Orphan products of the F. Fda and the NHL, lbi, nhlbi.

Kristin Kluska - Equity Analyst - (00:28:30)

First, I was pleasantly surprised to see that you received the acceptances of responses related to the form 483 observations.

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In addition, hog three is well powered to detect a treatment effect on cardiac function. I want to remind you that Capricorn developed Garamycel specifically to address heart disease, particularly the cardiomyopathy associated with DMG. However, until Dr. Softbl's study and its subsequent public.

Kristin Kluska - Equity Analyst - (00:28:40)

I guess. Can you just comment on that?

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Publication. There were no established efficacy benchmarks in DMD cardiomyopathy for the FDA. To use in defining clinical benefit.

Kristin Kluska - Equity Analyst - (00:28:41)

Because typically after we see CRLs, the FDA isn't so much as engaged in responding to those things until you resubmit. So can you kind of explain that timeline for us?

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Our entire regulatory path, including the current BLA, was built on the FDA's guidance on how efficacy should be defined in this patient population. We have always intended for ejection fraction to serve as our primary efficacy endpoint, so while this may appear to be a change in strategy, it is in fact a return to the original goals we set early in the development of dermycel.

Linda Marban - Chief Executive Officer - (00:28:54)

Yeah. So, you know, this has been an unorthodox review process. As I've mentioned in my remarks, as we've talked about in our disclosures, and other companies have gone through similar situations. So we passed our PLI. They issued the 483s. We responded to the 483s, and that review team, independent of the CRL, provided feedback that we had cleared our 483s and we are on path for approval of our CMC and our manufacturing plant for GMP use. So that is where that situation is. As I mentioned in the CRL, there were several CRL issues related to cmc. Many of those had already been addressed in information request responses that we provided prior to the issuance of the. But they had stopped reviewing in anticipation of issuing the CRL. The rest of them have already been Addressed and we look forward to providing those in our response to the CRL.

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To that end, with our type A meeting with the FDA now scheduled, we have submitted a comprehensive briefing package that addresses the concerns raised in the CRL and outlined several potential paths to approval.

Kristin Kluska - Equity Analyst - (00:29:53)

Okay, thank you.

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These include, first and foremost.

Kristin Kluska - Equity Analyst - (00:29:54)

And just want to confirm that the new timeline guidance 4Q versus 3Q for Hope 3 is just solely driven by the fact that you haven't started the unblinding yet. Because again, you're waiting for this meeting and that clarity. Absolutely. Yes, yes. Everything's on time with Hope 3 and we just are waiting for feedback from FDA. Let's go. Okay, thank you. And then just lastly, I guess in a nutshell, what are the key things that you hope to align from after.

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The continued review of our previously filed Bla, which we believe meets the applicable regulatory requirements for approval.

Kristin Kluska - Equity Analyst - (00:30:23)

This meeting takes place this week?

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As well as supplementing the current BLA with additional data from hope, three if needed.

Kristin Kluska - Equity Analyst - (00:30:25)

Is it just understanding specifically what's required? If it is Hope three, do you expect to have full understanding of what.

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We believe the current handling of our submission is inconsistent with our interpretation of the FDA written guidances. For cell and gene therapies, as well as recent public statements addressing the approval of safe and effective therapies for rare disease populations.

Kristin Kluska - Equity Analyst - (00:30:33)

That is process, primary endpoint will be. And then, you know, even their kind of Blessing that if Hope 3 is successful on that endpoint, that could potentially be sufficient enough to support an approval.

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We are hopeful that FDA will exercise a patient focused and science driven approach and rare disease approvals. In which they have been emphasizing in the media, as well as highlighting in the FDA direct podcasts based on the comments of Secretary Kennedy and Commissioner Macari approving Jeremiah for.

Kristin Kluska - Equity Analyst - (00:30:45)

Thanks again.

- (00:08:43)

The treatment of DMD cardiomyopathy seems directly in line with their goals. In conclusion, about a year ago, we received FDA feedback that shaped our decision to submit the BLA based on cardiac endpoints. We provided the requested data and analyses and fully expected any differences in interpretation to be addressed at an advisory committee meeting.

Linda Marban - Chief Executive Officer - (00:30:47)

Yeah, so exactly what you hypothesized. So we're looking for feedback on exactly what it's going to take to get this approved. As I mentioned in a previous question, you know, we believed in the data that we submitted. We believed that the CRL was unfounded. We were going towards the PDUFA very directly because there were no issues raised in the mid cycle review meeting. We thought we were in a pretty good position when the ADCOM was canceled. I didn't really take too much issue with that because I felt like in the late stage meeting we would be able to address any concerns they might have. So my first plan is to try and understand what their resistance is to the currently available data and if that is maintained, then what it will take to get to approval. Thank you.

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One that was ultimately canceled by the FDA without explanation.

Kristin Kluska - Equity Analyst - (00:31:38)

Sending you the best.

Linda Marban - Chief Executive Officer - (00:31:40)

Thank you so much.

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We are concerned with how our file has been managed because we believe.

Madison Elsadi - Equity Analyst - (00:31:44)

Thank you. Your next question comes from Madison Elsadi from B. Riley Securities. Please go ahead. Hey Linda and aj, thanks for taking our question. I was just curious, has there been any informal agency communication? I believe post CRL you had noted there was an opportunity for an informal teleconference and just wondering what the takeaways were there and if that kind of contributed to your decision to resubmit. Thank you.

- (00:09:18)

There were opportunities during the review period for the agency to raise the specific. Issues cited in the CRL before issuing the letter.

Linda Marban - Chief Executive Officer - (00:32:18)

Yeah, so we did have a short informal meeting with them based on their guidance from their leadership. It was primarily to align on timelines of this type A meeting and what would potentially be submitted and then to clarify the CMC related issues. Neither clinical nor stats were part of that meeting. So this type A meeting is very important because it allows us to meet with the agency and really flesh it out in terms of our decision to reply or respond to the CRL. That's always been our intent. We'd like feedback from them so that we can keep it smooth and steady and work our way to the quickest date of a PDUFA as possible. But we will see what happens this week.

Madison Elsadi - Equity Analyst - (00:33:03)

Got it, thanks. And then do you expect to get an answer on the primary endpoint change at the August meeting? I believe you said you were expecting an answer on that.

- (00:09:27)

We have long worked aside the DMD community and understand their calls. Both the continued treatment with Daramyosil and to gain access if it becomes commercially available.

Linda Marban - Chief Executive Officer - (00:33:16)

Yeah. So that's been one of our primary questions. We are looking, looking to get that worked out during this meeting.

- (00:09:39)

We will continue to urge the FDA to recognize that cardiomyopathy is a leading cause. Of death in DMD and a far more severe consequence than the loss of arm function. Deromycel has demonstrated a strong safety profile and the data indicate it can help stabilize the inevitable decline in cardiac function for people living with DMD.

Madison Elsadi - Equity Analyst - (00:33:24)

Got it. Well, good luck this week at the meeting.

- (00:10:01)

With regard to the CMC and commercial precommercial aspects of our program, I am pleased to announce that the FDA has now formally accepted all 483 items from our pre license. Inspection. This milestone further validates the strength of our quality systems manufacturing cap.

Linda Marban - Chief Executive Officer - (00:33:27)

Thank you.

- (00:10:21)

Capabilities and overall commercial readiness.

Aydin Hosinoff - Equity Analyst - (00:33:31)

Thank you. Your next question comes from Aydin Hosinoff. From Vladimir Fama, please go ahead. Hi, good afternoon. Linda. AJ thanks for taking questions and appreciate all the work you're doing. A couple from us. So first I want to clarify and apologize if this has been addressed, but want to clarify the, the timeline of the upcoming events. So the fourth quarter you're going to read out talk three, resubmit the BLA and how do, how do we treat this bla? Is it, if it is the same bla, what is the review process typical for, for these kind of reapplications? Is it two months sort of acceptance, then review certain months. Just curious your thoughts on the timeline?

- (00:10:25)

In addition, the CMC related items noted in the CRL have either been addressed prior to the issuance of the CRL or have been internally addressed since we have prepared formal responses which we plan to submit with our response to the CRO. Our manufacturing facility in San Diego remains fully operational and in production, and we are being disciplined in our commercial manufacturing investments to ensure we are fully prepared while managing resources wisely.

Linda Marban - Chief Executive Officer - (00:34:18)

Yeah. So that's one of the issues that we're going to be taking on with the agency during this meeting. Obviously, you know, there's a lot of timeline issues that were predicated on how they want to update the bla, whether or not they would require a new bla, what that does to priority review. We are still eligible for the prv, the voucher that comes with approval for a pediatric indication independent of timeline. But and our RMAT allows us certain benefits in terms of, you know, submission and also return on feedback. But we'll have to figure that out during this meeting with them and we'll disclose that as soon as it becomes available to us.

Aydin Hosinoff - Equity Analyst - (00:35:01)

Okay, I appreciate that. And regarding the left ventricular rejection fraction as the primary efficacy endpoint for Hope3, could you walk us through your thought process as to why you chose this endpoint? I think the Hope 2 had multiple cardiac endpoints. I think 21 cardiac measures. And could you also remind us any prior successful or unsuccessful of left ventricular ejection fraction primary endpoint submissions so that we can model based on those precedents?

- (00:10:55)

In parallel, we are diligently and strategically investing in launch redness activities, including physician education. Patient services, market access planning and reimbursement.

Linda Marban - Chief Executive Officer - (00:35:33)

Yeah, really, really important question. So left ventricular ejection fraction is obviously one of the most important indicators of cardia Function and clinicians, cardiologists use it all the time to sort of define where their patients sit in terms of cardiac function and also what their likely outcomes are going to be. So, you know, cardiologists know that below and above certain points you're either, you know, at greater risk for morbidity and mortality or, you know, or in a reasonable position for stabilization of your cardiac function. We've known that for a long time. The reason that ejection fraction, and this answers your last question along with your, your first one, has not been used as a primary efficacy endpoint in clinical studies is because up until recently it's really been considered a surrogate. It has not been directly tied to clinically relevant events such as mortality, hospitalization, exercise performance, those types of things. The most amazing thing, and this is why the change in our submission occurred from a clinical endpoint of skeletal muscle to cardiac ejection fraction was in collaboration with the agency, because while everybody knew that cardiomyopathy was the leading cause of death currently for Duchenne muscular dystrophy, up until John Soslow's study with the Cardiac Consortium, there really were not general evidence of what would be the predicting facts for mortality, hospitalization, those kinds of things. And in Duchenne, it's super hard. You're dealing with a rare disease with a small patient population and a pediatric disease. So in order to do a mortality study, you'd probably have to do like a 20 year study globally in order to be able to gather enough information. So the Office of Orphan Products understood this paradigm or paradox, as did the NHLBI National Heart, Lung and Blood Institute. So they funded John's study which allowed them to look at what would be the predictive factors of either morbidity or mortality in Duchenne muscular dystrophy, cardiomyopathy. And along with what our data suggested, ejection fraction was the most important feature. So there's also left ventricular, end systolic and end diastolic volumes. We're measuring those, those are secondary endpoints, as well as some biomarkers that John had, which was like BNP and one or two other paradigms or one or two other endpoints that would suggest the paradigm of morbidity and mortality. And what we actually were able to demonstrate with the presentation of this data is that Daramycel attenuated and may have even reversed the path of that decline in ejection fraction, therefore predicting morbidity and mortality. Because again, highlighting what I just said, it's a pediatric disease and it's rare doing those types of large studies that sometimes require thousands of patients to look at mortality risk. This is good for rare disease. And so the agency at the time, and hopefully still is willing to understand that ejection fraction is probably the best way of predicting where this patient population could go should it not be stabilized.

Aydin Hosinoff - Equity Analyst - (00:38:34)

It is very helpful. Appreciate it. Linda, just to summarize this, maybe essentially LVEF was a surrogate endpoint and it requires a little bit of sort of innovative thinking on FDA side to make it like a primary endpoint going forward. Would that be sort of fair summarization here?

- (00:11:07)

We've also begun working closely with treating physicians across the field of neurology and cardiology, who will ultimately collaborate in prescribing jeremycil to patients with DMD cardiomyopathy if approved. Many of these clinicians are already familiar with the therapy. Through their participation in the hope two and hope three studies.

Linda Marban - Chief Executive Officer - (00:38:54)

I don't agree because again, with the understanding of the new data that has come forth and the again rare disease population, there really is no other opportunity for adjudication of a primary EPICA standpoint. So if you really want to hear, I'm passionate about this, as you can probably tell from my voice, but if you really want to understand the risk and benefit here, please listen to our parent Project Muscular Dystrophy webinar that we did about a week ago. Dr. Chet Villa talked about the unmet need in cardiomyopathy. And what Dr. Villa, who sees these patients all day every day at Cincinnati Children's talks about is there is no other way of measuring efficacy in this particular patient population that would be fair and safe for human beings.

- (00:11:28)

And we are committed to ensuring a smooth transition to commercial use. If approved at this time, we are focused on seeking approval for Dermycel in the US and with respect to our global expansion plans. We will provide updates as they become available.

Aydin Hosinoff - Equity Analyst - (00:39:40)

Very helpful. Thank you so much. And one final question on Becker smart school dystrophy, my favorite one because part of our modeling is based on that.

- (00:11:44)

Now turning to our exosome program to remind you, in 2024, we were selected to participate in project Nextgen, an initiative led by the US Department of Health and Human Services. Aimed at advancing next generation vaccines. For Covid-19.

Aydin Hosinoff - Equity Analyst - (00:39:51)

So. But all these discussions, what kind of takeaways it has for bmd? I mean, would you have to run sort of another sort of hope 3 style large trial in BMD to get a similar potential sort of label as in dmd? And would left ventricular ejection fraction serve also sort of a primary endpoint for bmg? Thank you.

- (00:12:02)

And other potential infectious diseases. Under this program, the National Institutes of Allergy and infectious disease, NIAID.

Linda Marban - Chief Executive Officer - (00:40:16)

So sideways answer to that because I don't know directly at this point. What the agency will require is it's very early in this administration to understand what they're actually going to do in moving rare disease approvals forward. Our plan previously, which I have discussed with you and discussed publicly as well, is that we were going to try and use diguchenne data to build the Becker program because the pathophysiology of the cardiomyopathy is identical, just somewhat slower progressing in the Becker patients. And in fact as the Becker patients get older, it becomes more and more of a risk factor for morbidity and mortality. I don't know my current plan with Becker because I need to get understanding from the agency of how they're going to view the current Duchenne data and then I'll be able to make more educated comments on it as I achieve clarity there.

Aydin Hosinoff - Equity Analyst - (00:41:06)

Thank you. Thanks so much for your comments and good luck with your meetings for dhea. Thank you.

- (00:12:11)

We'll be sponsoring the phase one clinical trial of our stealth x vaccine.

Linda Marban - Chief Executive Officer - (00:41:12)

Thank you so much. We really appreciate it.

- (00:12:16)

Within the last several weeks, we reached a significant milestone for this program, which was the clearance of the IND and initiation of the trial using Stealthx, our exosome platform technology.

OPERATOR - (00:41:16)

Thank you. Your next question comes from Bubalan Pakshayapan from Roth Capital. Please go ahead.

- (00:12:28)

The phase I study is being conducted and overseen by NIAIDS Division of Microbiology and infectious disease dmid.

Manasa - (00:41:23)

Yeah, thanks for taking the call. I'm Manasa dialing in for Bubalin. So we have a couple of questions. The first question is. So do you regard the upcoming Taipei meeting as an opportunity for the FDA to clarify their change of stance with respect to Desell's BLA or a bellwether for investors in predicting the future outcomes of potential resubmission with the Hope3 data? Also, we are curious to know whether you'll be open to sharing the type A meeting minutes to investors to the extent you can to be comprehensive and elaborate.

- (00:12:39)

And I am pleased to report we have already supplied them with our clinical material for use in the trial. The phase one study is assessing our Covid-19 vaccine product. The trial is divided into three arms, comprised of three escalating doses of the spice spike, or s adjudin. And it combined high dose s plus the nucleo capsid or n antigen, the multivalent vaccine we have been develop.

Linda Marban - Chief Executive Officer - (00:41:58)

Yeah, in terms of your first question. Yeah, we expect share the data as, as it becomes available and, you know, the information as it becomes available. In terms of providing meeting minutes to investors, that becomes a little bit of a, you know, as needed basis. I can tell you right now, as you heard, our cash position is very strong. We're not out raising money. We don't anticipate needing to raise money. We're focusing on approval of Daramycel and dmd and so, you know, we'll see if the situation calls for it. We would definitely discuss it directly with that investor. Okay, another question.

Manasa - (00:42:48)

So some investors are wondering about the scope and the pragmatic value of the early and the mid cycle FDA review processes, you know, in relation with the overall review process. So what are your general thoughts on that?

- (00:13:05)

Developing. NiaD is starting with the s first because previous Covid vaccines are mainly s based and they wanted to have a basis for comparison with our vaccine candidate using similar antigenic profiles. The end goal is for the adoption of the N plus S, which is our multivalent vaccine. And we will provide more updates on this developing program as they become available.

Linda Marban - Chief Executive Officer - (00:43:01)

I'm sorry, could you ask that question again? I'm not sure I understood the question.

Manasa - (00:43:06)

Okay. So some investors are wondering about the scope and the pragmatic value of the early and the mid cycle FDA review processes, you know, in relation with the overall review process. So what are your general thoughts on that?

- (00:13:31)

We believe that stealthax has the characteristics of a vaccine product that Secretary Kennedy would find acceptable. It contains no adjuvants, it is not mRNA based, uses a native protein antigen and can be rapidly produced if needed. We have long believed that this type of vaccine checks all the boxes. For a safe and effective platform.

Linda Marban - Chief Executive Officer - (00:43:21)

Well, we had a BLA, was accepted and we had a very successful mid cycle review. So I guess the takeaway for investors is in our situation anyway, it wasn't predictive of what was coming next.

Manasa - (00:43:35)

Okay, and one last question. So in terms of the EX us, you know, clinical pathway for development and for approval, particularly in the uk, we were wondering if Capricor, you know, could be eligible to take advantage of the IRP to seek UK authorization, You know, at some point, provided the future FDA decision was favorable.

- (00:13:53)

As supported by multiple preclinical studies. An upcoming clinical data will allow us to confirm or challenge that hypothesis. This platform also has the potential to address multiple disease areas, including influenza and rsV.

Linda Marban - Chief Executive Officer - (00:43:55)

So what was what? What decision?

- (00:14:09)

While vaccines are not a core focus for us, if our candidate meets us government criteria and demonstrates efficacy.

Manasa - (00:44:02)

So we were wondering if, in terms of the EX US clinical pathway, whether Capricor could be eligible to take the irp, which is the international recognition procedure, to seek the UK authorization at some point, given that the future FDA session is going to be favorable.

- (00:14:17)

It could potentially open meaningful business development opportunities.

Linda Marban - Chief Executive Officer - (00:44:21)

Yeah, thanks. So, as I mentioned in my prepared remarks, we're focusing on U.S. approval right now. Our global strategy is emerging. A lot of it will be based on some of the feedback we received from FDA and what our path forward is. And please stay tuned. We'll provide updates on our ex US strategies as they become available.

- (00:14:20)

Just as importantly, it would serve as strategic proof for exosome platform, which we hope to advance as a versatile therapeutic engine for rare diseases and beyond.

Manasa - (00:44:43)

Thank you so much, and best of luck.

- (00:14:31)

While a majority of our efforts this year have been focused on securing approval for Dermyosel, an additional reason we recruited Dr. Michael Binks as our chief medical officer was his expertise. In translational science and medicine.

Linda Marban - Chief Executive Officer - (00:44:45)

Yeah, thanks.

OPERATOR - (00:44:50)

Thank you. Your next question comes from Katherine Novak from Jones. Please go ahead.

- (00:14:46)

He is now leading efforts to advance our exosome pipeline with the goal of forging strategic partnerships to expand the platform.

Katherine Novak - (00:44:56)

Hi, Linda. Thanks for taking my question. So one question was, when the FDA responded to your request for a type A meeting, gave you the date, did they give any substantive replies to your meeting request, particularly around positive or negative wording around lves? No. So typically when they accept a meeting request, they just say they accept your meeting request and then they send over a date. We submitted a briefing package and we're awaiting feedback on that. And, you know, we'll discuss the ramifications of what we asked for and what they respond in the meeting. Got it. And then the R and D expense for 2Q, you know, what accounts for the increase? Does this build up of product inventory ahead of potential launch or is this due to ongoing clinical studies? We should expect to see continued growth on that line.

- (00:14:53)

Into and beyond vaccines. We believe the differentiated features of exosomes, including low immunogenicity scalable manufacturing and targeted delivery physician Capricorn for unique potential opportunities and the therapeutic delivery space. We look forward to sharing updates as they become available.

Linda Marban - Chief Executive Officer - (00:45:57)

Yeah, thanks, Katherine. I mean, it encompasses a little bit of both of what you said. We're obviously in the end stages of hope three, but those patients, 104 of them, are ongoing in that clinical development expense line item. We're also obviously preparing for the CMC endeavor. So that's where it's at. Obviously, when we get more clarity and feedback from FDA and announce more plans, I think we'll have more granular items on the burn rate moving forward. But it really encapsulates both of those areas. That's the main areas of spend.

- (00:15:14)

Thank you. And with that, I will now turn the call over to AJ to run through our financial.

Katherine Novak - (00:46:29)

Okay. All right, thanks. That's it for us.

- (00:15:19)

Thanks, Linda. This afternoon's press release provided a summary of our second quarter 2025 financials on a GAAP basis. We may also refer to our quarterly report from form ten q. Which we expect to become available shortly and will be accessible on the SEC website as well as the financial section of our website. Let me start with our cash.

Linda Marban - Chief Executive Officer - (00:46:34)

Thank you.

- (00:15:40)

Position. As of June 30, 2025, our cash, cash equivalents, and marketable securities totaled approximately $122.8 million.

OPERATOR - (00:46:35)

Thank you.

- (00:15:50)

Turning into the financials, revenues for the second quarter of 2025 were zero compared to approximately 4 million for the second quarter of 2024.

Matthew Venezia - Equity Analyst - (00:46:38)

Thank you. Ladies and gentlemen, as a reminder, should you have a question, please press star one. Your next question comes from Matthew Venezia from agp. Please go ahead. Hi, Linda. Hi, Ajay. Thank you for taking our questions. So just looking for a little clarity on the FDA review process to date.

- (00:15:59)

Additionally, revenues for the first half of 2025 were zero, compared to approximately 8.9 million. For the first half of 2024. Like to point out that the source of revenue for 2024 was the ratable recognition of the $40 million we had received under our us distribution. Agreement with Nippan Shinyaku, which has been fully recognized as of December 30.

Matthew Venezia - Equity Analyst - (00:47:04)

Has obviously there's been turnovers since the.

- (00:16:19)

1st 2024. Moving to our operating expenses for the second quarter of 2025, excluding stock based compensation, our research and development expenses were approximately 20.1 million, compared to approximately 11.7 million for Q two, 2024. For the first half of 2025, excluding stock based compensation, our research and development expenses.

Matthew Venezia - Equity Analyst - (00:47:06)

New administration, but has there been any. Turnover since Prasad's leaving the FDA and.

- (00:16:42)

Were approximately 36.3 million, compared to approximately 21.8 million for the first half of 2024.

Matthew Venezia - Equity Analyst - (00:47:12)

Then him coming back. And has the team that you've been engaging with at the FDA changed at all in that time?

- (00:16:49)

Moving into general and administrative expenses, excluding stock based compensation, were approximately 4 million. In Q two 2025 and approximately 1.8 million in Q two 2024 and for the first half to 25 also excluding stock based compensation or general administrative expenses, were approximately 7 million for the first half.

Linda Marban - Chief Executive Officer - (00:47:20)

Really don't know. You know, we'll know more this week. So, you know, the big change was Dr. Prasad leaving and now Dr. Prasad returning. We don't know the ramifications of his exit or return on our program, but what I can say for sure is that we're looking forward to working directly with him and with the team. And we do not think that the data should be interpreted differently by any. By any teammate that are different.

- (00:17:10)

Of 25 and 3.6 million for the first half of 2024. Net loss for the second quarter of 25 was approximately 25.9 million compared to a net loss of approximately 11 million for the second quarter of 24.

Matthew Venezia - Equity Analyst - (00:47:47)

Got it. And just a little bit on the run rate. Do you expect it to taper off in 2026 once you kind of get clarity regulatory wise and potential launch wise as Hope 3 winds down and should R and D come down and maybe.

- (00:17:25)

The net loss for the first half of 25 was approximately 50.3 million compared to a net loss of approximately 20.8 million for the first half of 2024. I will now turn the call back over to Linda for some closing remarks. Again, thank you, AJ.

Linda Marban - Chief Executive Officer - (00:48:07)

Gna go up a little bit? Yeah, I think that's fair.

- (00:17:39)

Just to reinforce AJ's points on our financial position. With over $120,000,000 in cash. We are well positioned to support operations into late 2026 and continue to advance. Our key pipeline objectives. Additionally, if we receive approval, we would still be eligible to receive a priority review voucher.

Matthew Venezia - Equity Analyst - (00:48:10)

Thanks, Matt. I mean, obviously, again, as I articulated the next steps in Hope three is a big aspect to that, but should we achieve approval, we'll have some pretty serious capital injections around the potential sale of the PRV and 80 million from Nippon Shinyaku that will allow us, of course, to invest in CMC expansion and everything we want to do around the launch for commercial endeavors. So that's kind of how we're looking at it. Obviously, more granular level can be discussed in the future, but we expect the capital to go right where it needs to be, which is preparing for the launch.

Linda Marban - Chief Executive Officer - (00:48:45)

All right, got it. Thank you guys for taking my questions and good luck at the Taipei meeting.

- (00:17:59)

As well as a milestone payment of $80 million from Nifon Shinyaku, representing additional significant nondilutive capital opportunities to further strengthen our balance sheet. This is an important moment for Capricorn. While we have faced recent regulatory headwinds, we are advancing deliberately. Strategically and with confidence in our.

OPERATOR - (00:48:50)

Thanks, Matt.

- (00:18:20)

Data, our team, and our path forward. We continue to believe that dermyosal represents a major step forward for patients with DMD cardiomyopathy and that our exozone platform is well positioned to deliver value.

Chris Lemos - (00:48:52)

Thank you.

- (00:18:32)

Through continued innovation and partnerships. To the Duchen Muscular Dystrophy community. Thank you for your ongoing trust and support. We remain grounded in the science.

Chris Lemos - (00:48:54)

Thank you. Your next question comes from Chris Lemos from Nada. Please go ahead. Do you know who you with at the Taipei meeting?

Linda Marban - Chief Executive Officer - (00:49:09)

I'm sorry. All right, well, thank you so much. I guess we lost the question. I want to thank you for joining today's call. We look forward to updating you on our progress over the coming months, although this is a big week for us. So we will update as soon as we get feedback from the FDA and look forward to a positive review of deramyocell. Thank you so much and have a great day.

- (00:18:42)

Execution and committed to building a company that delivers meaningful and lasting impact for all DMD patients. I will now open up the line for questions.

OPERATOR - (00:49:45)

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

- (00:18:52)

Thank you. Ladies and gentlemen, we will now begin the question and answer session. Should you have a question, please? Press start, followed by the number one. On your touchstone phone, you will hear a prompt that your hand has been raised. Should you wish to declare from the polling process, please purchase our follow by the numbers two. If you are using a speaker phone, please leave the handset before pressing any keys. One moment, please, for your first question. Your first question comes from. Diapers. Handler, please. Go ahead. Great. Thank you so much for taking my question, and I appreciate all of your hard work. To keep fighting for these boys and get them a therapy that's going to help them. With their heart function. I wanted to get a sense for sort of. The plan. For next steps in terms of. Unblinding hope. Three. Is the plan to sort of get confirmation from the FDA? On that first. Just maybe sort of reiterating. What the plan is. Thanks. Ted. Always a pleasure, even during these crazy, crazy times. Yeah, so. We are waiting for adjudication from FDA as to what their requirements will be for Hopetree. And then we will. Submit a statistical analysis plan and proceed with unblinding only after that has been accepted. We just don't want to muddy or cloud the waters with any thoughts that we had. Unblinded early. So our plan is to stay quiet until we have plans from them. Great. And when do you expect to hear back from the FDA or get that clarity? Well, obviously from the rescheduling of our earnings call today. Our type a meeting is this week. We anticipate to have a really good conversation with the FDA. We're looking forward very much to meeting with them. August is our month. August of 2024 was when this whole plan was put in place, so I'm very excited for this meeting. And in terms of providing clarity and updates to the markets, that probably won't be until I get the official feedback in writing, especially with the liability of our current. Situation and times. So hopefully Adam Pearson won't report on it before I do. Thanks so much, Linda. Good luck. Thanks. Thank you. Your next question comes from Leland Gershow. From Oppenheimer. Please go ahead. Thanks, Linda, for the update and taking our questions and just a couple here. So just to be further from Ted's inquiry. So. If you go with the plan to not unblind top three, but continue with the current bla as it is? Would there then be supplemental oled data that could go into. What the FDA has on file versus what had been submitted. I guess if you could share just what? Incremental. Data xhope three that could become available to them that would be different from what they had. Reviewed previously. Yeah, Leland. So actually, I don't have really thought about submitting supplemental alle data. Of course, it continues to support our safety profile and the efficacy. If you look at the long term efficacy of dheremia cell. It's actually quite extraordinary, and we're very proud of that record in our oled patients. And I don't know of another clinical effort in DM. DMD that has as long of a record post study as we do. Having said that, I think the meeting with FDA will define what they will want for data for either the reopening and resubmission of this bla. And so I don't have an answer on that. My current plan is to have everything ready. Then they have an opportunity to select. What they think would be most efficient in determining efficacy for approval. Okay. And I guess I have to ask whether the van a Prasad now back at Sebert. Does that inform your thinking? Are the people who you're interacting with? The FDA. Is there a different team now with Nicole verdon out of the picture. Since the last few weeks. How should we think about kind of who your kind of counterparties? Are at FGa at this point. Thanks, William. It's interesting. I think the last few years I've been thinking about. This a lot. People have become much more focused on who the review team is and exercising political capital and regulatory flexibility and all of this lingo that has become very popular. I'm going back to old school. We have good clinical data. We have. Great safety data. We have guidance from the agency in writing as to what they wanted. We provided. It. And now, really? I think it's up to them to decide who is best suited within that agency to make the decision of adjudication and all of the factors that go into it. So I tell my team. I'll tell the markets. We are proud of our data. We see it. In terms of the long term efficacy in our patients and safety, and we sincerely hope that the agency gives us a good. Path forward to get this to those patients as quickly as possible. Okay. All right. Thanks very much for taking the questions. Thanks, Leila. It always a pleasure. Thank you. The next question comes from you. Pankinis from AC went right. Please go ahead. Hi, Linda. And AJ, thanks for taking the questions. Good afternoon. So just to sort of press the envelope a little bit, obviously things could change. Incrementally or dramatically this week and about a month after that. When you provide the details from the minutes to the street, but with language in the press release and what you talked about today. You're talking about. Resubmitting in its current form. You're saying maybe not really having any incremental. Data from the ole that you just discussed in the last question. So what would you expect that could be potentially different. Well again. Given the FDA a variety of opportunities. In our briefing document. We remain open. To resubmission of the VLA as it is. We didn't feel that the CRL was founded. The data that we submitted was exactly what they asked for, and the graphs themselves are interpretable. Statistically and clinically significant. So that's, of course, our number one goal, is explaining to them. Why? Perhaps their interpretation was wrong. There's then everything that follows from the accelerated. Approval with the submission of hope, three data in support of that and a variety of other opportunities. That they can help us adjudicate, and that's why we're looking forward. To this type a. Meeting, so. With that in mind. We go in with open hearts to meet with the agency and look forward to having them understand. That developing and approving therapeutics for rare diseases, especially pediatrics, one does require. Looking at the data in a more holistic fashion, and we're hoping that that is. Exactly what happens this week. All right, so that's fair. So maybe just another question starting at the or from the back end of your comments around stealthex. So it's great that it's getting into the clinic now. Any visibility with regard to. What might be next with regard to an indication. Obviously. You mentioned influenza as a potential. And how would you describe. The early talks. Maturity levels of potential BD around stealthex. Yeah. Thanks, Joe. So our stealthex program is sort of our little engine that. Could we just. Kept on moving it forward. Our vaccine program is really exciting in the sense that, as I mentioned in my remarks, This type of vaccine, which we've always believed in, is exactly what Secretary Kennedy has advocated. Would be a much better vaccine candidate. No ADJ events. Native proteins rapidly produced no mRNA that kind of thing, so we're very excited about that. We're looking forward to Denya data. They're very excited about it because. It's a program that does fit that criteria, and we'll see where that goes. In terms of Capricorn's interest in developing vaccine technology, that's something that I've always said would be a business development opportunity. We think it's wiper. Than ever, based on the criteria I just put forth. In terms of therapeutic indications, we haven't. Disclosed some of the ones that we've been working on, intern. Internally, as I've mentioned many times, we've spoken most of our efforts and our capital on deromyosl and look forward to providing updates on where we're going. To be taking the exodome program. As we further develop that therapeutic pipeline. Got it. Looking forward to more visibility out of the program. And good luck with the meeting this week. Thanks, Joe. Always a pleasure. Thank you. Your next question comes from Kristen Kluska from canter. Please go ahead. Hi Linda and AJ. Also sending you best wishes for your meeting this week. Few questions for me. First, I was pleasantly surprised to see that. You received the acceptances of responses related to the form 483 observations? I guess. Can you just comment on that because typically after we see crls, the FDA isn't so much. As engaged in responding to those things until you resubmit. So can you kind of explain? That timeline for us. Yeah. So this has been an unorthodox review process. As I've mentioned in my remarks, as we've talked about in our disclosures, and other companies have gone through similar situations. So we passed our PLI. They issued the 483 we responded to the that review team. Independent of the crl. Providing feedback that we had cleared our four hundred and eighty three s and we are on path. For approval of our CMC and our manufacturing plan for GMP use. So that is. Where that situation is. As I mentioned in the CRL, There were several cr. CRO issues related to CMC. Many of those had already been addressed and information request responses that we provided prior to the issuance of the CRL, but they had stopped reviewing in anticipation of issuing the CRL. The rest of them have already been addressed, and we look forward. To providing those in our response to the CRL. Okay, thank you. And just want to confirm that. The new timeline guidance four q versus three q for hope. Three is just solely driven by the fact that you haven't started the unblinding yet, because again, you're waiting for this meeting and that clarity. Absolutely. Yes. Everything's on time with hope three, and we just are waiting for feedback from FDA. Let's go. Okay. Thank you. And then just lastly, I guess in a nutshell, What are the key things that you hope to align from after this meeting takes place this week? Is it just understanding specifically what's required? If it is hope three, do you expect to have full understanding of what that primary endpoint will be. And then even their kind of blessing that if hope three is successful on the endpoint, that could potentially be sufficient enough to support an approval. Thanks again. Yeah. So exactly what you hypothesize. So we're looking for feedback on exactly what it's going to take to get this approved. As I mentioned in a previous. Question. We believed in the data that we submitted. We believe that the CRL was unfounded. We were going towards the Padufa. Very directly. Because there were no issues raised in the mid cycle review meeting. We thought we were in a pretty good position. When the Adcom was canceled, I didn't really take too much issue. With that because I felt like in the late stage meeting, we would be able to address any concerns they might have. So my first plan is to try and understand what their resistance is to the currently available data. And. If that is maintained, then what it will take to get to approval? Thank you. Sending you the best. Thank you so much. Thank you. Your next question comes from Marison El Sadi from Briley securities, please. Go ahead. Hey, weather. Thanks for taking our question. I was just curious. Has there been any informal agency communication, I believe, post crl? Noted there was an opportunity for an informal teleconference and just wondering what the takeaways. Were there, and if that kind of contributed to your decision to resubmit. Thank you. Yeah. So. We did have a short, informal meeting with them. Based on their guidance from their leadership, it was primarily to align on timelines of this type A meeting and what would potentially be submitted and then to clarify the CMC related issues. Neither clinical nor stats were part of that meeting, so this type a meeting is very important because it allows us. To meet with the agency and really flesh it out in terms of our decision to reply or respond to the Crl. That's always been our intent. We'd like feedback from them so that we can keep it smooth and steady and work. Our way to the quickest date of a padufa as possible. But we will see what happens this week. Got it. Thanks. And then do you expect to get an answer? On the primary endpoint change. At the August meeting. I believe you said you were expecting. An answer on that. Yeah, so that's been one of our primary questions. We are looking to get that worked out during this meeting. Got it. Good luck this week at the meeting. Thank you. Thank you. Your next question comes from. Ivan has enough from Vladimir. Farma, please go out ahead. Hi. Good afternoon, Linda. AJ, thanks for taking questions and appreciate all the work you're doing. A couple from us. So first I want to clarify. And apologize if this has been addressed, but want to clarify the timeline of the upcoming event. So the fourth quarter, you're going to read out hog three, resubmit Bla and. How do we treat this bla? If it is the same VLA, what is the review process typical for this kind of reapplications is it? Two months sort of acceptance, then review certain amounts. Just curious your thoughts. On the timeline. Yeah. So that's one of the issues that we're going to be taking on. With the agency during this meeting, obviously. There's a lot of timeline issues that were predicated on. How they want to update the bla, whether or not they would require a new bla. What that does to priority review. We are still eligible for the PRV. The voucher that comes with approval for a pediatric indication, independent of timeline. And our rmat allows us. Certain benefits in terms of submission and also return on feedback, but we'll have to figure that out during this meeting with them, and we'll disclose that as soon as it becomes available to us. Okay, I appreciate that. And regarding the electronic rejection fraction as the primary efficacy endpoint for hope. Three. Could you walk us through your thought process as to why you chose this endpoint? I think hope two had multiple cards at ports. I think 21 card measures. And could you also remind us, any prior successful or unsuccessful of left ventricular ejection fraction primary inputs. Ambitions so that we can model based on those precedents. Yeah, really important question. So, left ventricular ejection fraction is obviously one of the most important indicators of cardiac function, and clinicians, cardiologists use it all the time. To sort of define where their patients sit in terms of cardiac function and also what their likely outcomes are going to be. So cardiologists know that below and above certain points. You're either at greater risk for morbidity and mortality. Or in a reasonable position for stabilization of your cardiac function. We've known that for a long time. The reason that ejection fraction. And this answers your last question along with your first one. Has not been used as a primary efficacy endpoint in clinical studies is because up until recently it's really been considered a surrogate. It has not been directly tied to clinically. Relevant events such as mortality, hospitalization, Exercise performance, those types of things. The most amazing thing, and this is why the change in our submission occurred from. A clinical endpoint of skeletal muscle to cardiac ejection fraction was in collaboration with the agency, because while everybody knew that cardiomyopathy was the leading cause of death currently for Duchenne muscular dystrophy up until John Saslow's study with the cardiac consortium. There really were. Not general evidence of what would be the predicting facts for mortality, hospitalization, those kinds of things. And in Duchenne it's super hard. You're dealing with a rare disease. With a small patient population and a pediatric disease. So in order to do a mortality study, you'd probably have to do, like, a 20 year study globally in order to be able to gather enough information. So the office of Orphan Products understood this paradigm or paradox as did the NHLBI national heart. Lying and blood institute. So they funded John's study, which allowed them to look at what would be the predictive factors of either morbidity or mortality. And deshen muscular dystrophy cardiomyopathy. And along with what our data suggested, Ejection fraction was the most important feature, so there's also lapendricular and systolic and diastolic volumes. We're measuring those. Those are secondary endpoints. As well as some biomarkers that John had, which was like BNP. And one or two other paradigms or one arch, other endpoints that would suggest the paradigm of morbidity and mortality and what we actually were able to demonstrate with the presentation of this data is that Jeremiah cell attenuated and may have even reversed the path of that decline in ejection traction. Therefore, predicting morbidity and mor. Mortality because, again, Highlighting what I just said is a pediatric disease that is rare doing those types of large studies. That sometimes require thousands of patients to look at mortality risk. This is good for rare disease and so the agency at the time, and hopefully still is willing to understand that ejection fraction is probably the best way of predicting where. This patient population could go should it not be stabilized. It is very helpful. I appreciate. Linda, just to summarize this, maybe. Essentially, LDF was a survey endpoint, and it requires a little bit of sort of innovative. Thinking on FDA side to make it like a primary endpoint going forward. Would that be sort of fair summarization here. I don't agree. Because again, With the understanding of the new data that has come forth and the again, rare disease population. There really is no other opportunity for adjudication of a primary efficacy endpoint. So if you really want to hear? I'm passionate about this, as you can probably tell from my voice. But if you really want to understand. The risk and benefit here. Please listen to our parent project muscular dystrophy webinar that we did. About a week ago, Dr. Chetvilla talked about the unmet need in cardiomyopathy. And what Dr. Villa, who sees these patients all day, every day at Cincinnati children's talks about is there is no other way. Of measuring efficacy in this particular patient population that would be fair and safe for human beings. Very helpful. Thank you so much. And one final question. On Becker. Smart school dystrophy. My favorite one because. Part of our model is based on that. But all these discussions. What kind of takeaways it has for BMD. Would you have to run sort of another sort of hope city style large trial in BMD. To get. A similar. Potential sort of label as in DMV and would less than trickle injection fraction. Also sort of a primary endpoint for BNG. Thank you. So sideways answer to that, because I don't know directly at this point what the agency will require is. It's very early. In this administration to understand what they're actually going to do in moving rare disease approvals. Forward. Our plan previously, which I have discussed with you. And discuss publicly as well is that we were going to try and use digicen data. To build the Becker program because the pathophysiology of the cardiomyopathy is identical, just somewhat slower progressing in the Becker patients. And in fact, as a Becker patients get older, it becomes more and more of a risk factor for morbidity and mortality. I don't know my current plan with Becker because I need to get understanding from the agency of how they're going to view the current Duchen data, and then I'll be able to make more educated comments on it as I achieve clarity there. Thank you. Thanks so much for your comments and good luck. Good luck with your meetings. For DFA thank you. Thank you so much. We really appreciate it. Thank you. Your next question comes from Bubalam. Pacheyapan. From Roth capital, please. Go ahead. Yeah. Thanks for taking the call. I'm Manasar. Daling and for Bubalan. So we have a couple of questions. The first question is, so do you regard the upcoming Taipei? Meeting as an opportunity for the FDA to clarify their change of stance with respect to deromeo. Cells BLa or a bellwether for investors in predicting the future outcomes. Of potential resubmission with the hope three data. Also, we are curious to know whether you'll be open to sharing the type a meeting minutes to investors to the extent you can. To be comprehensive and elaborate. Yeah. In terms of your first question. Yeah. We expect to share the data. As it becomes available. And the information as it becomes available. In terms of providing meeting minutes to investors. That becomes a little bit of as needed basis, I can tell you right now, as you heard, our cash position is very strong. We're not outraising money we don't anticipate needing to raise. Money. We're focusing on approval of Dermyocell and DMD. And so. We'll see if the situation calls for it. We would definitely discuss it directly with that investor. Okay, another question. So some investors are wondering about the scope and the pragmatic value of the early and the mid cycle FDA review processes in relation with the overall review process. So what are your general thoughts on that? I'm sorry. Could you ask that question again? I'm not sure I understood the question. Okay, so some investors are wondering about the scope and the pragmatic value of the early and the mid cycle FDA review processes in relation with the overall review process. So what are your general thoughts on that? We had a bla was accepted, and we had a very successful mid cycle review. So I guess the takeaway for investors. Is. In our situation anyway. It wasn't predictive of what was coming next. Okay. And one last question. So in terms of the ex US clinical pathway for development and for approval, particularly in the UK. We were wondering if Capricorn could be eligible to take advantage. Of the IRP to seek uk authorization at some point, provided the future FD. Fda decision was favorable. What decision? So we were wondering if, in terms of the ex US clinical pathway, whether Capricorn could be eligible to take the IRP, which is the international recognition procedure to seek the UK authorization. At some point. Given that the future FDA session is going to be favorable. Yeah. Thanks. So, as I mentioned in my prepared remarks, We're focusing on us approval right now, our global strategy. Is emerging. A lot of it will be based on. Some of the feedback we received from FDA and what our path board is, and please stay tuned. We'll provide updates on our ex us strategies as they become available. Okay. Thank you so much and best of luck. Yeah, thanks. Thank you. Your next question comes from Katherine Novak from Jones. Please go ahead. Hi, Linda. Thanks for taking my questions. So one question was when the FDA responded to your request for a type A meeting, gave you the date. Did they give any substantive reply to your meeting request? Particularly around positive or negative wording around the LVEF. No. So typically when they accept a meeting request, they just say they accept your meeting request and. Then they send over a date. We submitted a briefing package, and we're waiting feedback on that. And we'll discuss the ramifications of what we asked for and what they respond. In the meeting. Got it. And then the R and D expense for two. Q what accounts for the increase was this build up of product inventory ahead of potential launch, or is this data ongoing clinical studies that we should expect to see continued growth on that line. Yeah. Thanks, Catherine. I mean. It encompasses a little bit of both of what you said. We're obviously, in the end, stages of hope? Three. But those patients, 104 of them, are ongoing in that clinical development expense line. Item. We're also obviously preparing for the CMC endeavor, so that's where it's. At. Obviously, when we get more clarity and feedback from FDA and announce more plans, I think we'll have more granular items on the burn rate moving forward, but it really encapsulates both of those areas. That's the main areas of spend. Okay. All right, thanks. That's it for us. Thank you. Thank you. Thank you. Ladies and gentlemen, as a reminder, should you have a question, please press star one. Your next question comes from Matthew Peninsia from AGP. Please go ahead. Hinda. Hi, AJ. Thank you for taking our questions. So just looking for a little clarity. On the FDA review process to date has obviously there's been turnovers since the new administration, but has there been any turnovers since. Prasad's leaving the FDA and then him coming back. And has the team that you've been engaging with at the FDA changed at all in that time. We don't know. We'll know more this week. So the big change. With Dr. Prasad leaving and now Dr. Prasad returning, we don't know. The ramifications of his exit or return on our program. But what I can say for sure is that. We're looking forward to working directly with him and with the team. And. We do not think that the data should be interpreted differently by any teammate that are different. Got it. And just a little bit on the run rate, do you expect it to taper off in 2026. Once you kind of get clarity. Regulatory wise and potential launch wise as hope three winds down. Should R and D come down and maybe gna go up a little bit. Yeah, I think that's fair. Thanks, Matt. I mean, obviously, again, as I articulated. What the next steps in hope. Three is a big aspect to that. But should we achieve approval? We'll have some pretty serious capital injections around the potential sale of the PRV. And 80 million from Nip on Shinyaku. That will allow us, of course, to invest. In CMC expansion and everything we want to do around the launch for commercial endeavors so that's kind of how we're looking. At it. Obviously, more granular level can be discussed in the future, but we expect the capital to go right where it needs to be, which is preparing for the launch. All right, got it. Thank you guys for taking my questions, and good luck at the type a. Meeting. Thanks, Matt. Thank you. Thank you. Your next question comes from Chris Lemos from Nada. Please go ahead. Hilan. You know who you. Pay meeting. I'm sorry. All right. Well, thank you so much. I guess we lost the question. I want to thank you. For joining today's call. We look forward to updating you on our progress over this. Coming months, although this is a big week for us, so we will update as soon as we get feedback from the FDA and look. Forward to a positive review of Jeremiah. Thank you so much and have a great day. Ladies and gentlemen, this concludes today's conference call. Thank you all for your participation. You may now disconnect.