NASDAQ:CAPR Capricor Therapeutics Q4 2023 Earnings Report $12.72 -0.56 (-4.18%) As of 01:25 PM Eastern This is a fair market value price provided by Polygon.io. Learn more. Earnings HistoryForecast Capricor Therapeutics EPS ResultsActual EPS-$0.02Consensus EPS -$0.23Beat/MissBeat by +$0.21One Year Ago EPSN/ACapricor Therapeutics Revenue ResultsActual Revenue$12.09 millionExpected Revenue$5.80 millionBeat/MissBeat by +$6.29 millionYoY Revenue GrowthN/ACapricor Therapeutics Announcement DetailsQuarterQ4 2023Date2/29/2024TimeN/AConference Call DateThursday, February 29, 2024Conference Call Time4:30PM ETUpcoming EarningsCapricor Therapeutics' Q1 2025 earnings is scheduled for Monday, May 12, 2025, with a conference call scheduled at 4:30 PM ET. Check back for transcripts, audio, and key financial metrics as they become available.Q1 2025 Earnings ReportConference Call ResourcesConference Call AudioConference Call TranscriptPress Release (8-K)Annual Report (10-K)Earnings HistoryCompany ProfilePowered by Capricor Therapeutics Q4 2023 Earnings Call TranscriptProvided by QuartrFebruary 29, 2024 ShareLink copied to clipboard.There are 6 speakers on the call. Operator00:00:00Good afternoon, ladies and gentlemen, and welcome to Capricorn Therapeutics 4th Quarter 2023 Earnings Call. At this time, all lines are in a listen only mode. Following the presentation, we will conduct a question and answer session. This call is being recorded on Thursday, February 29, 2024. I would now like to turn the conference over to A. Operator00:00:30J. Bergman, CFO of Capricor. Please go ahead. Speaker 100:00:36Thank you and thank you for joining today. Before we start, I would like to state that we will be making certain forward looking statements during today's presentation. These statements may include statements regarding among other things the efficacy, safety and intended utilization of our product candidates, our future R and D plans including our anticipated conduct and timing of preclinical 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, manufacturing capabilities, potential milestone payments, our financial position and our uses of existing cash and investment resources. These forward looking statements are based on current information, assumptions and expectations that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward looking statements. These and other risks are described in our periodic filings made with the SEC, including our quarterly and annual reports. Speaker 100:01:31You are cautioned not to place undue reliance on these forward looking statements, and we disclaim any obligation to update such statements. With that, I'll turn the call over to Linda Marban, CEO. Speaker 200:01:41Thanks, A. J. Good afternoon, and thank you for joining today's call. I'm encouraged with the progress we have made at Capricor in 2023 and into 2024. And today, I will outline our main priorities for our lead CAP-one thousand and two program as well as provide a brief update on our exosome platform technology. Speaker 200:02:022023 was a big year for Capricor as we are now gearing up for biologics license application and commercial authorization. To that end, Capricor has assembled a team primarily focused on executing in 4 main areas in order to be prepared to bring our lead product CAP-one thousand and two to market for the treatment of DMD as expeditiously as possible. These are clinical, manufacturing, CLA readiness and commercial preparation. I will provide an overview of each area today. First, let me provide a clinical update on our Phase III HOPE-three pivotal trial, enrolling late stage ambulance and non ambulant young men with DMD across the United States. Speaker 200:02:49Late last year, we announced completion of enrollment in our Phase 3 pivotal HOPE-three clinical trial where we enrolled 61 subjects randomized 1 to 1 to CAP-one thousand and two or placebo. In December, we conducted a pre specified interim futility analysis and we were very pleased to announce that the trial was successfully deemed to not be futile with a positive recommendation to continue the trial. This analysis was based on an assessment by the Data Safety and Monitoring Board otherwise known of course as the DSMB of 30 subjects who reached the 6 month time point and assessing their poll scores in a blinded fashion. This important positive outcome triggered our 1st milestone payment of $10,000,000 from Nippon Shinyaku further strengthening our balance sheet and extending our cash runway. Now as you know, 2024 is a pivotal year for Capricor as we will have data from Cohort A of our Phase 3 HOPE-three clinical study at the end of the year, as well as fully enroll Cohort B by the Q2 of this year. Speaker 200:04:07To remind you, Cohort B was designed at the request of FDA to demonstrate comparable efficacy of CAP-one thousand and two from our San Diego manufacturing facility to that produced in Los Angeles. This cohort which is designed to enroll approximately 44 subjects is enrolling very well. In fact, enrollment has proceeded even faster than predicted, partially based on the fact that there are no current therapeutics approved and very few in clinical trials for these later stage non ambulant patients. The primary endpoint of HOPE-three is the change from baseline and the performance of the upper limb version 2.0, of course commonly known as the pull at 1 year as well as various secondary skeletal and cardiac endpoints, including left ventricular ejection fraction. We have already seen efficacy in the POLL 2.0 and our Phase II HOPE-two study where the data showed a 1.8 point improvement relative to placebo and was statistically significant. Speaker 200:05:17Even more validating is that as shown in our Lancet paper, multiple whole endpoints, whether specific regions or in combination, showed improvements with statistically significant changes in multiple domains. Importantly, CAP-two zero two treated patients saw improvements in left ventricular ejection fraction, which is the gold standard measure of cardiac function. We saw 4% improvement in treated patients with a P value of 0.002. In addition, there were significant improvements in left ventricular and systolic and left ventricular end diastolic volume, further suggesting structural improvements in the heart. There are no approved therapeutics that we are aware of that directly address the cardiomyopathy associated with DMD. Speaker 200:06:09Therefore, the importance of CAP-ten zero two in this area of unmet medical need cannot be understated. We have long term safety and efficacy data in this patient population as we are continuing to follow the patients from the HOPE-two study in an open label extension study into their 4th year and we will plan to have the 3 year results available in the Q2 of 2024. The 2 year results shared last year continue to show statistically significant differences in the PULP 2.0 and the open label extension treatment group when compared to the original rate of decline of the placebo group from HOPE-two after 1 year. Further, while the natural history of DMD cardiomyopathy suggests a steady decline in cardiac function as measured by ejection fraction and the HOPE-two open label extension, we observed improvements in heart function in 66% of patients. The 2 year results underscore the potential long term benefits of CAP-one thousand and two treatment in DMD. Speaker 200:07:16As we envision CAP-one thousand and two as a multiyear treatment, this dataset will strengthen both our potential revenue modeling and payer discussions for long term reimbursement. DMD has rapidly become an orphan disease that has garnered a lot of attention, not only because of the terrible nature of a disease that robs children of the ability to use their muscles more, but also because of the promise of disease modification by gene therapies and exon skipping technologies to potentially allow modification of the dystrophinopathy. Many have thought that along with exon skipping technologies, if the gene therapies are approved, there would no longer be a need for other treatments for DMD, nothing to be further from the truth. The current gene therapy paradigm allows for a small, albeit potentially relevant amount of micro dystrophin protein to be made. Current clinical data suggests there is an attenuation of disease progression from treatment with the gene therapy. Speaker 200:08:18However, we believe that it will require a multi drug paradigm to address all of the pathological consequences of DMD, primarily inflammation and fibrosis caused by the lack of dystrophin. CAP-one thousand and two is perfectly positioned to be a partner therapy for DMD as the stated mechanism of action is immunomodulation and reduction in fibrosis. In fact, some of the current subjects at HOPE-three are post gene therapy, but still qualify for CAP-one thousand and two based on the study's inclusion and exclusion criteria. CAP-one thousand and two has a strong safety profile and is a once a quarter infusion that has shown to be well tolerated. If CAP-one thousand and two delays the disease progression, which years of data and multiple clinical trials have demonstrated, it is our hope that CAP-one thousand and two would be a preferred treatment with gene or exon skipping therapies. Speaker 200:09:17Now, I would like to take a few minutes to update you on our recent FDA interactions and regulatory goals for the program over the next several quarters. As you may recall, we met with FDA last year and aligned on the design of our current Phase 3 program with Cohort A being the primary data set for the filing of the BLA and data from Cohort B to be used to transition to our San Diego manufacturing facility. Now that Cohort A has been fully enrolled and cohort B is heading towards full enrollment, we have continued to discuss with FDA any opportunity to expedite the filing of our BLA, Keeping in mind that in order to successfully achieve BLA acceptance, a critical aspect is to meet all CMC requirements as outlined by FDA. To that end, we have successfully established a potency assay for CAP-one thousand and two based on the mechanism of action of the product, which is acceptable to FDA for our 2b marketed product and critical to the establishment of comparability between each of our manufacturing sites. As many of you know, FDA leadership has taken a great interest in helping move the field of treating DMD forward, and we continue to believe that we can work with them on a strategy to move CAP-one thousand and two towards approval. Speaker 200:10:38Importantly, our San Diego manufacturing facility is now fully operational, staffed and producing doses for clinical use. Currently, we can produce enough CAP-one thousand and two in our San Diego facility to meet and exceed NS Pharma's forecast for year 1 of product launch, if approved by FDA. We also have plans in place to expand our San Diego facilities operations to support a larger demand as may be necessary, but further expansion or investment would be something we will look forward to do following potential BLA acceptance. I would like to highlight that we have expended a relatively small amount of capital to build our commercial manufacturing plant. This has also allowed us to strengthen our IP portfolio with additional process and method based patents filings and know how. Speaker 200:11:26We also are able to control COGS effectively to drive margins as high as possible on revenue and or revenue shares. Importantly, we can also potentially expand our CAP-one thousand and two program to other indications while replicating our manufacturing modules. All of this taken together puts Capricor in a good position as we prepare for our potential initial commercial product in DMD. A majority of the investment into our facility operations and personnel has gone into preparations for this endeavor and I feel confident that we can deliver according to the timelines we have set forth. Now for our update on our commercial partnership with NS Pharma, who is already actively preparing for the potential launch of CAP-one thousand and two, assuming the data is positive and we have an accepted BLA. Speaker 200:12:16We continue to work closely with them as we move closer to that goal. As we have stated, subjects are continuing to report slowing of disease progression on CAP-one thousand and two, which is supported by the pull data. This positive data combined with the strong safety profile has led to nearly full partition and open label extension studies. Therefore, by the time of a potential BLA acceptance, we would expect to have approximately 120 patients already on CAP-ten zero two on an ongoing basis. These patients would likely become our first commercial patients. Speaker 200:12:50This potential revenue stream will be very supportive of a strong launch and will provide an initial commercial market for this product. Additionally, we are in the early stages of establishing a strong commercial team to support our partners at NS Pharma. Now I'd like to briefly turn to provide an update on our exosome technology. Currently, we are pursuing 2 avenues of opportunity. One is our vaccine program using Stealth X, our proprietary platform that is useful for engineering select proteins either inside or on the surface of the exosome. Speaker 200:13:24And the other is using the same basic platform but for the development of therapeutics. One of our major achievements this year was being selected as part of the US government's Project NextGen, which is slated to text vaccine candidates for potential use in preventing COVID-nineteen as well as prepare for future pandemics. The structure of the arrangement with NIAID, which is otherwise known as the National Institute of Allergy and Infectious Diseases is that Capricor will provide them with manufactured vaccine, the campaign for which is underway now and they will conduct and fully fund a Phase 1 clinical trial. There will be 3 groups tested, a low dose S, a high dose S of the current strain of COVID-nineteen and then a bivalent candidate containing SNN, the nucleocapsid. I am pleased to inform you that we have submitted an IND to the FDA for our Stealth X vaccine, which is currently under review. Speaker 200:14:23And we anticipate that once the IND is approved, the NIAID will initiate the clinical trial in late 2024. I will provide more specific timelines on this program as we progress through the year. This will be the only multivalent candidate tested as far as we know and we have high hopes for success in terms of potential safety and efficacy. If NIA finds that the vaccine meets its criteria for safety and efficacy, they may consider our program for a fully funded Phase 2. This opportunity is very important for Capricor because it supports our exosome based vaccine. Speaker 200:15:01And while we don't have intention to become a vaccine focused company, it sets up the program nicely for partnering and other business development opportunities. As a reminder, the power of this technology is that it combines the speed of an mRNA vaccine with the potential efficacy of a recombinant protein based vaccine. Should it work in humans as well as in preclinical animal studies, it could be a very important improvement in vaccinology. Also on the exosome front, we are in discussions with several potential partners to develop the therapeutic arm of our engineered exosome technology. The strategy involves taking the same Stealth X platform and using it to target a specific tissue and then appropriately deliver a payload. Speaker 200:15:47Each early preclinical data suggests the strategy works and we are looking forward to sharing more color on this important program as data becomes available. And finally, on the corporate side, we raised approximately $23,000,000 late last year in an equity offering to support our balance sheet into 2025. This strategic financing was anchored by Nippon Sinyakou, further cementing our strong relationship and their commitment to Capricor. As we think about moving through 2024 and into 2025, I want to remind you that our U. S. Speaker 200:16:23Agreement with Nippon Shinyaku comes with it an additional $90,000,000 of potential milestone payments up to the time of approval, which are triggered upon certain regulatory based achievements. Following potential approval, there is an additional $605,000,000 in potential milestone payments, which will be payable to Capricor based on various sales based targets being met. Furthermore, if we receive FDA approval for CAP-one thousand and two for the treatment of DMD, we would be eligible to receive a priority review voucher or the PRB based on our previous receipt of a rare pediatric disease designation, which we retain full rights to and we'll look to sell to support our balance sheet. Lastly, we are in active discussions with several parties related to the European rights of CAP-one thousand and two for DMD. Our main goal is to continue to support our balance sheet, leveraging non dilutive partnerships to fuel CAP-one thousand and two towards potential approval and support the exosome program. Operator00:17:22Overall, I want to thank you Speaker 200:17:24for your support. We continue to diligently manage our resources, focus our efforts on bringing CAP-ten oh two towards potential commercialization in the most expeditious way possible. We are very much looking forward to the next several months as we will be continuing our interactions with FDA, announcing our 3 year open label extension data, completing enrollment for Cohort B and presenting at various medical, scientific and investor related conferences. Speaker 100:17:49I will now turn the call over to A. J. To run through our financials. A. J? Speaker 100:17:54Thank you, Linda. This afternoon's press release provided a summary of our Q4 and full year 2023 financials on a GAAP basis. And you may also refer to our annual report on Form 10 ks, which we expect to become available shortly, will be accessible on the SEC website as well as our website. Turning to the financials, let me start with our cash position. We ended December 31, 2023 with cash, cash equivalents and marketable securities of approximately 39,500,000 dollars This excludes the $10,000,000 milestone payment we received in January of 24 from Nippon Shinyaku under our distribution and commercialization agreement. Speaker 100:18:33Based on our recent operating results and projections, we expect our cash runway to extend into the Q1 of 2025, but this expectation excludes any additional potential milestone payments under our exclusive commercialization and distribution agreements with Nippon Shinyaka. In the Q4 of 'twenty three our revenue was approximately $12,100,000 compared to approximately $1,000,000 for the Q4 of 2022, which was primarily attributable to the ratable recognition of the $40,000,000 which includes the upfront and milestone payment we have received in accordance with our U. S. Commercialization and distribution agreement with Nippon Shinyaku. Excluding stock based compensation, our research and development expenses were approximately $9,400,000 for the Q4 of 2023 compared to approximately $6,000,000 for the Q4 of 2022. Speaker 100:19:22The increase in expenses of $3,400,000 was primarily due to increased clinical and manufacturing costs associated with our Phase 3 HOPE-three trial. Excluding stock based compensation, our general and administrative expenses were approximately $1,900,000 for both the Q4 of 2023 and 2022. Net loss for the Q4 of 'twenty three was approximately $800,000 compared to a net loss of 7 point $7,000,000 for the Q4 of 2022 and net loss for the full year 'twenty three was approximately $22,300,000 compared to a net loss of approximately $29,000,000 for the full year 2022. And with that, we will now open the line up for questions. Operator, go ahead. Operator00:20:07Thank you. Ladies and gentlemen, we will now conduct the question and answer session. Your first question comes from Joe Pantginis from H. C. Wainwright. Operator00:20:25Your line is now open. Speaker 300:20:27Hi, Linda and A. J. Thanks for taking the questions. Good afternoon. First off, so wanted to talk about your regulatory discussion. Speaker 300:20:36So last year you had some pretty clear frameworks that you shared with us again today about the need for Cohort A and giving some manufacturing comparability from Cohort B out of the San Diego facility. And you keep talking about ways to potentially expedite. So I wanted to explore that a little bit. So first question is, I mean, when you look at this, you have the RMAT status, is one of the potentials here rolling BLA because you'll be able to start submitting data quicker as part of the filing and other options you might be considering? Speaker 200:21:14Joe, always a pleasure. Thanks for the question. So, Operator00:21:18I'm going to start answering Speaker 200:21:19by saying, I've been working on this therapeutic for 19 years and where we are right now is just so staggeringly exciting to me. It's sometimes hard to express. But what has been the best part of the last few months has been the careful attention that FDA has been paying to Capricorn and to cap 102. They recognize the positive safety and efficacy data. They've looked at the open label extension data, the HOPE-two data, and they're working very closely with us. Speaker 200:21:48So yes, all options are on the table right now in terms of how to get this across the line as fast as possible. As I mentioned, leadership within CBER is aware of our program and really working very closely with us. We have RMAT. We have rare pediatric disease designation, orphan disease designation. So we have a lot of the bells and whistles that will carry our program as quickly as possible into the arms of DMD patients. Speaker 300:22:15Got it. And then just curiosity for Cohort B, are you strictly needing to show manufacturing comparability or do these patients need to be follow-up for a certain timeframe? Speaker 200:22:30So we belt and suspend the program by building in a full one to one randomized clinical trial with safety and efficacy built in. So it basically is a mirror image of Cohort A. We're working with FDA on what they're going to actually ask us for. What we know and what we can guarantee on is that they're willing to accept the license application on cohort A. What we're going to need from cohort B is still what we're working with them on, but it really is almost something that we've not only been prepared for, but something that comes along very naturally. Speaker 200:23:04So the trial cohort B, let me reemphasize, will be fully enrolled by Q2 of this year. And then we're going to be able to potentially position that as a post marketing commitment for the program. Speaker 300:23:17Got it. And then my last question, you don't mind and thanks for bearing with me. Absolutely. We've been talking about this for several years because we've been excited about the data. So I guess how would you portray the role of the cardiovascular data you've been accumulating through HOPE-two and beyond in the evolution of your regulatory discussions and how much that may or may not be coming into play to date? Speaker 300:23:43Yes. Speaker 200:23:43So it's obviously one of the most important cornerstones of our regulatory strategy, but also in the eagerness of the community to get approval for CAP-ten zero two. To remind you and I stated this, we saw a 4% improvement in ejection fraction, the gold standard of cardiac function in HOPE-two. In HOPE-two, open label extension, we didn't start measuring cardiac function until 2 years in. We'll have 3 years data in the Q2 of this year, so stay tuned for that on cardiac function. But we're pretty convinced that it's going to be one of the major parts of what we're going to look for on our label. Speaker 200:24:17And it's the primary secondary endpoints that has been built into hopefully both cohort A and cohort B. Speaker 300:24:25Got it. Thanks for the added details, Linda. Speaker 200:24:28Absolutely, Joe. Always a pleasure. Operator00:24:32Your next question comes from Kristin Tolska from Cantor Fitzgerald. Your line is now open. Speaker 400:24:39Hi, everyone. Good afternoon. Thanks so much for taking my question. Operator00:24:45Hi, Kristen. How are you? Speaker 400:24:47I'm well. How are you doing? Speaker 200:24:50Good, Sam. Speaker 400:24:52So thinking about the potential for combinations, I completely understand the need for this. But can you comment on what your expectations would be in terms of payer support? And then as gene therapy typically requires a lot more upfront timing to do the different testing, manufacturing, etcetera, Would it be your expectation that CAP-one thousand and two would be the first therapy essentially given in this cascade? And would that be an advantage in case there is some payer pushback? Speaker 200:25:25So we've had really positive feedback from payers, not just at Capricor, but also NS Pharma has done significant work in preparing for market launch. And the way that we're understanding it is that there's going to be therapy that would be necessary for sort of management of dystrophinopathies, right? So the exon skippers and the gene therapies. And because there's not an approved gene therapy theoretically yet, we don't really know how they're going to approach choosing those or both or whatever. But what we do know is that there needs to be a junk of therapy that would manage, and I've talked about this a lot, the immunomodulation or the inflammatory the immunomodulation or the inflammatory response caused by the constant breakdown of protein in the body due to the mutation as well as manage the fibrosis and help to support perhaps the framework laid down by a gene therapy or an exon skipper, which would be a healthier protein. Speaker 200:26:20So we are very confident that payers would find it beneficial to cover both the dystrophinopathy management strategy as well as CAP-ten zero two for the management of the inflammation and the fibrosis. Now in terms of the order in which the therapeutics are given, that we would sort of have to talk to some of the KOLs. Already starting to do some of that market research, obviously, because we're going for our initial label for some of the later stage patients, at least based on the HOPE-three data. We reserve the right to ask FDA to go as young as possible. Colloquially, we always say time is muscle. Speaker 200:26:55And the data has shown that once people get on CAPTAINERT to disease progression is significantly attenuated almost immediately. So in terms of the timing of how that's done, that remains to be seen, but we're very confident that it will be part of the overall treatment paradigm of Duchenne. Speaker 400:27:14Thank you for that. And then we have seen quite a bullish of the 4 to 5 year olds on the Sarepta therapy, which Operator00:27:21I think underscores the unmet need here. So I wanted to ask Speaker 400:27:21what your thoughts are patients wanting therapy, especially because you are going after that non ambulatory population? And then what capacity would you be able to help with given this is half of the patients with DMD? Thanks again. Operator00:27:48Yes. So thank you. So we plan on swinging Speaker 200:27:50the door wide open in terms of what we ask FDA for. The benefit here, unlike many therapeutics, we're going to come into BLA with 4 or 5 years' worth of safety data, tracking children from theoretically age 10 and beyond. So we're going to open the door and see what the opportunity is. Obviously, if I had a child with DMD, I want to get my child on CAP-one thousand and two as young as possible. There's really no downside. Speaker 200:28:16And potentially could even have impact on who knows things like steroid dosing, things that may have ultimate impacts and side effects. In terms of what we're looking for, I think that was your second question. We're looking for utilization of CAP-ten or 2 as early as it becomes available to the community. We're right there with it. Operator00:28:48Your next question comes from Aidan Husainov from Ladenburg. Your line is now open. Speaker 500:28:55Good afternoon, everyone. Good afternoon, Linda, Ajay. Congratulations with the progress this quarter and staying on track with the guidance with the top line in Q4 'twenty four. I have a couple of questions. First, I want to ask you about the potential expansion of indication. Speaker 500:29:15I think you mentioned something in your remarks. And we this the most natural expansion, I think we talked about it, it was the Becker dystrophy that we see tremendous increase in value in other Becker companies. And given your cardiomyopathy focus and focus on improving skeletal cardiac muscle function, could you expand a little bit on your any potential efforts that you're making regarding that expansion? Operator00:29:53Yes. Thanks, Aidan, and always good to talk with you. So we've been talking about this for Speaker 200:29:57a while. Obviously, there's tremendous opportunities for expansion. As I mentioned, the manufacturing paradigm is set. We have a potency assay that's been accepted by FDA. We understand the mechanism of action and how to make the cells and we certainly are poised to expand our efforts. Speaker 200:30:13Becker is certainly one that's of great interest, especially since the primary manifestation later in life is the cardiomyopathy, which as we've talked about is one of the main targets that CAPTAINR II seems to ameliorate. Having said that, we are right now focusing almost all of our efforts on getting CAP-ten zero two across the line for DMD. We're working on the BLA. We're working on a launch strategy, commercialization strategy. And so indication expansion will come behind that and we'll provide more color to you into the market as those opportunities become available. Speaker 500:30:49Okay, understood. Another question I have is regarding the HOPE TO open extension, open label extension trial results in the second quarter. So what are your expectations regarding this data? Speaker 200:31:06Well, we have 3 year data coming out in the Q2 of this year, as I said. The 2 year data was extraordinary. We presented that most recently at PPMD and then we'll be sharing some more data at the Muscular Dystrophy Association meetings next week in Orlando. We have every expectation based on the anecdotes that we hear from the subjects and their families that this trend of stabilization of disease and attenuation of disease progression will continue. We're really looking forward to seeing the MRI data because that will be 2 years of sequential cardiac function data. Speaker 200:31:40And we have every reason to believe that we should be able to stabilize heart function as well. So, we also have so many families that call us, that tell us that their sons are able to do what they weren't able to do before or they can't wait for their next infusion. Can we please get in sooner because we feel it wearing off after 3 months. So I'm very much looking forward to seeing that data and then ultimately sharing it with all of you. Speaker 500:32:10Understood. Thank you for that. And the last question is regarding the European discussions, discussions with potential European parties. So in your discussions with them, first of all, if you could give us a little bit of insight, are these like established players, are these new players? And what are the typical questions that he has there? Speaker 500:32:31What is the which part of CapTel or to value they are most Speaker 200:32:43community is as interested in getting therapies across the line for DMDS is the U. S. Authorities. They have a little bit different strategy in Europe. As you probably are aware, there's a little bit of different way of going about it. Speaker 200:32:56The parties that we're talking to are some new and some established. I think we've been on the radar for several larger companies for a long time now. And with the data coming around the corner, they were paying more attention. Perhaps one of the best things is that our San Diego manufacturing facility can be EMA qualified. So we can make doses here and ship them Europe, which is an added benefit. Speaker 200:33:20And in terms of the questions that we get, I think would be the typical ones, which is regulatory strategy, getting CAP2 across the line, what clinical trial work Speaker 100:33:29will be Speaker 200:33:29needed. From our standpoint, it's probably going to be fairly straightforward. And we definitely look forward to making Cap Tenet 2 available worldwide. Speaker 500:33:42Okay. Thank you so much and congratulations for the progress this quarter. Speaker 200:33:47Thanks, Aidan. Take care. Operator00:34:05There are no further questions at this time. I'm turning it over to the management for closing remarks. Speaker 200:34:12Before we conclude today's call, I want to extend my sincere gratitude to the patients, their families, the clinicians and our partners at Nippon Shinyaku and NS Pharma and of course at FDA who continue to work with us to bring CAP-one thousand and two closer to potential approval. Again, thank you to everyone who joined us this afternoon and I look forward to seeing you at meetings in the future. Operator00:34:37Ladies and gentlemen, this concludes today's conference. Thank you for joining. You may now disconnect.Read morePowered by Conference Call Audio Live Call not available Earnings Conference CallCapricor Therapeutics Q4 202300:00 / 00:00Speed:1x1.25x1.5x2x Earnings DocumentsPress Release(8-K)Annual report(10-K) Capricor Therapeutics Earnings HeadlinesCapricor Therapeutics Inc (CAPR) Q4 2024 Earnings Call Highlights: Navigating Financial ...April 21, 2025 | gurufocus.comCapricor Therapeutics Sees Unusually Large Options Volume (NASDAQ:CAPR)April 18, 2025 | americanbankingnews.comThe Trump Dump is starting; Get out of stocks now?The first 365 days of the Trump presidency… Will be the best time to get rich in American history.April 25, 2025 | Paradigm Press (Ad)Capricor Therapeutics to Present at the 2025 Piper Sandler Virtual Cardio DayApril 1, 2025 | markets.businessinsider.comCheck Out What Whales Are Doing With CAPRMarch 28, 2025 | benzinga.comCapricor Therapeutics Announces FDA Acceptance of Biologics License Application for Deramiocel in Duchenne Muscular Dystrophy CardiomyopathyMarch 21, 2025 | nasdaq.comSee More Capricor Therapeutics Headlines Get Earnings Announcements in your inboxWant to stay updated on the latest earnings announcements and upcoming reports for companies like Capricor Therapeutics? Sign up for Earnings360's daily newsletter to receive timely earnings updates on Capricor Therapeutics and other key companies, straight to your email. Email Address About Capricor TherapeuticsCapricor Therapeutics (NASDAQ:CAPR), Inc. (NASDAQ:CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor's lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. 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There are 6 speakers on the call. Operator00:00:00Good afternoon, ladies and gentlemen, and welcome to Capricorn Therapeutics 4th Quarter 2023 Earnings Call. At this time, all lines are in a listen only mode. Following the presentation, we will conduct a question and answer session. This call is being recorded on Thursday, February 29, 2024. I would now like to turn the conference over to A. Operator00:00:30J. Bergman, CFO of Capricor. Please go ahead. Speaker 100:00:36Thank you and thank you for joining today. Before we start, I would like to state that we will be making certain forward looking statements during today's presentation. These statements may include statements regarding among other things the efficacy, safety and intended utilization of our product candidates, our future R and D plans including our anticipated conduct and timing of preclinical 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, manufacturing capabilities, potential milestone payments, our financial position and our uses of existing cash and investment resources. These forward looking statements are based on current information, assumptions and expectations that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward looking statements. These and other risks are described in our periodic filings made with the SEC, including our quarterly and annual reports. Speaker 100:01:31You are cautioned not to place undue reliance on these forward looking statements, and we disclaim any obligation to update such statements. With that, I'll turn the call over to Linda Marban, CEO. Speaker 200:01:41Thanks, A. J. Good afternoon, and thank you for joining today's call. I'm encouraged with the progress we have made at Capricor in 2023 and into 2024. And today, I will outline our main priorities for our lead CAP-one thousand and two program as well as provide a brief update on our exosome platform technology. Speaker 200:02:022023 was a big year for Capricor as we are now gearing up for biologics license application and commercial authorization. To that end, Capricor has assembled a team primarily focused on executing in 4 main areas in order to be prepared to bring our lead product CAP-one thousand and two to market for the treatment of DMD as expeditiously as possible. These are clinical, manufacturing, CLA readiness and commercial preparation. I will provide an overview of each area today. First, let me provide a clinical update on our Phase III HOPE-three pivotal trial, enrolling late stage ambulance and non ambulant young men with DMD across the United States. Speaker 200:02:49Late last year, we announced completion of enrollment in our Phase 3 pivotal HOPE-three clinical trial where we enrolled 61 subjects randomized 1 to 1 to CAP-one thousand and two or placebo. In December, we conducted a pre specified interim futility analysis and we were very pleased to announce that the trial was successfully deemed to not be futile with a positive recommendation to continue the trial. This analysis was based on an assessment by the Data Safety and Monitoring Board otherwise known of course as the DSMB of 30 subjects who reached the 6 month time point and assessing their poll scores in a blinded fashion. This important positive outcome triggered our 1st milestone payment of $10,000,000 from Nippon Shinyaku further strengthening our balance sheet and extending our cash runway. Now as you know, 2024 is a pivotal year for Capricor as we will have data from Cohort A of our Phase 3 HOPE-three clinical study at the end of the year, as well as fully enroll Cohort B by the Q2 of this year. Speaker 200:04:07To remind you, Cohort B was designed at the request of FDA to demonstrate comparable efficacy of CAP-one thousand and two from our San Diego manufacturing facility to that produced in Los Angeles. This cohort which is designed to enroll approximately 44 subjects is enrolling very well. In fact, enrollment has proceeded even faster than predicted, partially based on the fact that there are no current therapeutics approved and very few in clinical trials for these later stage non ambulant patients. The primary endpoint of HOPE-three is the change from baseline and the performance of the upper limb version 2.0, of course commonly known as the pull at 1 year as well as various secondary skeletal and cardiac endpoints, including left ventricular ejection fraction. We have already seen efficacy in the POLL 2.0 and our Phase II HOPE-two study where the data showed a 1.8 point improvement relative to placebo and was statistically significant. Speaker 200:05:17Even more validating is that as shown in our Lancet paper, multiple whole endpoints, whether specific regions or in combination, showed improvements with statistically significant changes in multiple domains. Importantly, CAP-two zero two treated patients saw improvements in left ventricular ejection fraction, which is the gold standard measure of cardiac function. We saw 4% improvement in treated patients with a P value of 0.002. In addition, there were significant improvements in left ventricular and systolic and left ventricular end diastolic volume, further suggesting structural improvements in the heart. There are no approved therapeutics that we are aware of that directly address the cardiomyopathy associated with DMD. Speaker 200:06:09Therefore, the importance of CAP-ten zero two in this area of unmet medical need cannot be understated. We have long term safety and efficacy data in this patient population as we are continuing to follow the patients from the HOPE-two study in an open label extension study into their 4th year and we will plan to have the 3 year results available in the Q2 of 2024. The 2 year results shared last year continue to show statistically significant differences in the PULP 2.0 and the open label extension treatment group when compared to the original rate of decline of the placebo group from HOPE-two after 1 year. Further, while the natural history of DMD cardiomyopathy suggests a steady decline in cardiac function as measured by ejection fraction and the HOPE-two open label extension, we observed improvements in heart function in 66% of patients. The 2 year results underscore the potential long term benefits of CAP-one thousand and two treatment in DMD. Speaker 200:07:16As we envision CAP-one thousand and two as a multiyear treatment, this dataset will strengthen both our potential revenue modeling and payer discussions for long term reimbursement. DMD has rapidly become an orphan disease that has garnered a lot of attention, not only because of the terrible nature of a disease that robs children of the ability to use their muscles more, but also because of the promise of disease modification by gene therapies and exon skipping technologies to potentially allow modification of the dystrophinopathy. Many have thought that along with exon skipping technologies, if the gene therapies are approved, there would no longer be a need for other treatments for DMD, nothing to be further from the truth. The current gene therapy paradigm allows for a small, albeit potentially relevant amount of micro dystrophin protein to be made. Current clinical data suggests there is an attenuation of disease progression from treatment with the gene therapy. Speaker 200:08:18However, we believe that it will require a multi drug paradigm to address all of the pathological consequences of DMD, primarily inflammation and fibrosis caused by the lack of dystrophin. CAP-one thousand and two is perfectly positioned to be a partner therapy for DMD as the stated mechanism of action is immunomodulation and reduction in fibrosis. In fact, some of the current subjects at HOPE-three are post gene therapy, but still qualify for CAP-one thousand and two based on the study's inclusion and exclusion criteria. CAP-one thousand and two has a strong safety profile and is a once a quarter infusion that has shown to be well tolerated. If CAP-one thousand and two delays the disease progression, which years of data and multiple clinical trials have demonstrated, it is our hope that CAP-one thousand and two would be a preferred treatment with gene or exon skipping therapies. Speaker 200:09:17Now, I would like to take a few minutes to update you on our recent FDA interactions and regulatory goals for the program over the next several quarters. As you may recall, we met with FDA last year and aligned on the design of our current Phase 3 program with Cohort A being the primary data set for the filing of the BLA and data from Cohort B to be used to transition to our San Diego manufacturing facility. Now that Cohort A has been fully enrolled and cohort B is heading towards full enrollment, we have continued to discuss with FDA any opportunity to expedite the filing of our BLA, Keeping in mind that in order to successfully achieve BLA acceptance, a critical aspect is to meet all CMC requirements as outlined by FDA. To that end, we have successfully established a potency assay for CAP-one thousand and two based on the mechanism of action of the product, which is acceptable to FDA for our 2b marketed product and critical to the establishment of comparability between each of our manufacturing sites. As many of you know, FDA leadership has taken a great interest in helping move the field of treating DMD forward, and we continue to believe that we can work with them on a strategy to move CAP-one thousand and two towards approval. Speaker 200:10:38Importantly, our San Diego manufacturing facility is now fully operational, staffed and producing doses for clinical use. Currently, we can produce enough CAP-one thousand and two in our San Diego facility to meet and exceed NS Pharma's forecast for year 1 of product launch, if approved by FDA. We also have plans in place to expand our San Diego facilities operations to support a larger demand as may be necessary, but further expansion or investment would be something we will look forward to do following potential BLA acceptance. I would like to highlight that we have expended a relatively small amount of capital to build our commercial manufacturing plant. This has also allowed us to strengthen our IP portfolio with additional process and method based patents filings and know how. Speaker 200:11:26We also are able to control COGS effectively to drive margins as high as possible on revenue and or revenue shares. Importantly, we can also potentially expand our CAP-one thousand and two program to other indications while replicating our manufacturing modules. All of this taken together puts Capricor in a good position as we prepare for our potential initial commercial product in DMD. A majority of the investment into our facility operations and personnel has gone into preparations for this endeavor and I feel confident that we can deliver according to the timelines we have set forth. Now for our update on our commercial partnership with NS Pharma, who is already actively preparing for the potential launch of CAP-one thousand and two, assuming the data is positive and we have an accepted BLA. Speaker 200:12:16We continue to work closely with them as we move closer to that goal. As we have stated, subjects are continuing to report slowing of disease progression on CAP-one thousand and two, which is supported by the pull data. This positive data combined with the strong safety profile has led to nearly full partition and open label extension studies. Therefore, by the time of a potential BLA acceptance, we would expect to have approximately 120 patients already on CAP-ten zero two on an ongoing basis. These patients would likely become our first commercial patients. Speaker 200:12:50This potential revenue stream will be very supportive of a strong launch and will provide an initial commercial market for this product. Additionally, we are in the early stages of establishing a strong commercial team to support our partners at NS Pharma. Now I'd like to briefly turn to provide an update on our exosome technology. Currently, we are pursuing 2 avenues of opportunity. One is our vaccine program using Stealth X, our proprietary platform that is useful for engineering select proteins either inside or on the surface of the exosome. Speaker 200:13:24And the other is using the same basic platform but for the development of therapeutics. One of our major achievements this year was being selected as part of the US government's Project NextGen, which is slated to text vaccine candidates for potential use in preventing COVID-nineteen as well as prepare for future pandemics. The structure of the arrangement with NIAID, which is otherwise known as the National Institute of Allergy and Infectious Diseases is that Capricor will provide them with manufactured vaccine, the campaign for which is underway now and they will conduct and fully fund a Phase 1 clinical trial. There will be 3 groups tested, a low dose S, a high dose S of the current strain of COVID-nineteen and then a bivalent candidate containing SNN, the nucleocapsid. I am pleased to inform you that we have submitted an IND to the FDA for our Stealth X vaccine, which is currently under review. Speaker 200:14:23And we anticipate that once the IND is approved, the NIAID will initiate the clinical trial in late 2024. I will provide more specific timelines on this program as we progress through the year. This will be the only multivalent candidate tested as far as we know and we have high hopes for success in terms of potential safety and efficacy. If NIA finds that the vaccine meets its criteria for safety and efficacy, they may consider our program for a fully funded Phase 2. This opportunity is very important for Capricor because it supports our exosome based vaccine. Speaker 200:15:01And while we don't have intention to become a vaccine focused company, it sets up the program nicely for partnering and other business development opportunities. As a reminder, the power of this technology is that it combines the speed of an mRNA vaccine with the potential efficacy of a recombinant protein based vaccine. Should it work in humans as well as in preclinical animal studies, it could be a very important improvement in vaccinology. Also on the exosome front, we are in discussions with several potential partners to develop the therapeutic arm of our engineered exosome technology. The strategy involves taking the same Stealth X platform and using it to target a specific tissue and then appropriately deliver a payload. Speaker 200:15:47Each early preclinical data suggests the strategy works and we are looking forward to sharing more color on this important program as data becomes available. And finally, on the corporate side, we raised approximately $23,000,000 late last year in an equity offering to support our balance sheet into 2025. This strategic financing was anchored by Nippon Sinyakou, further cementing our strong relationship and their commitment to Capricor. As we think about moving through 2024 and into 2025, I want to remind you that our U. S. Speaker 200:16:23Agreement with Nippon Shinyaku comes with it an additional $90,000,000 of potential milestone payments up to the time of approval, which are triggered upon certain regulatory based achievements. Following potential approval, there is an additional $605,000,000 in potential milestone payments, which will be payable to Capricor based on various sales based targets being met. Furthermore, if we receive FDA approval for CAP-one thousand and two for the treatment of DMD, we would be eligible to receive a priority review voucher or the PRB based on our previous receipt of a rare pediatric disease designation, which we retain full rights to and we'll look to sell to support our balance sheet. Lastly, we are in active discussions with several parties related to the European rights of CAP-one thousand and two for DMD. Our main goal is to continue to support our balance sheet, leveraging non dilutive partnerships to fuel CAP-one thousand and two towards potential approval and support the exosome program. Operator00:17:22Overall, I want to thank you Speaker 200:17:24for your support. We continue to diligently manage our resources, focus our efforts on bringing CAP-ten oh two towards potential commercialization in the most expeditious way possible. We are very much looking forward to the next several months as we will be continuing our interactions with FDA, announcing our 3 year open label extension data, completing enrollment for Cohort B and presenting at various medical, scientific and investor related conferences. Speaker 100:17:49I will now turn the call over to A. J. To run through our financials. A. J? Speaker 100:17:54Thank you, Linda. This afternoon's press release provided a summary of our Q4 and full year 2023 financials on a GAAP basis. And you may also refer to our annual report on Form 10 ks, which we expect to become available shortly, will be accessible on the SEC website as well as our website. Turning to the financials, let me start with our cash position. We ended December 31, 2023 with cash, cash equivalents and marketable securities of approximately 39,500,000 dollars This excludes the $10,000,000 milestone payment we received in January of 24 from Nippon Shinyaku under our distribution and commercialization agreement. Speaker 100:18:33Based on our recent operating results and projections, we expect our cash runway to extend into the Q1 of 2025, but this expectation excludes any additional potential milestone payments under our exclusive commercialization and distribution agreements with Nippon Shinyaka. In the Q4 of 'twenty three our revenue was approximately $12,100,000 compared to approximately $1,000,000 for the Q4 of 2022, which was primarily attributable to the ratable recognition of the $40,000,000 which includes the upfront and milestone payment we have received in accordance with our U. S. Commercialization and distribution agreement with Nippon Shinyaku. Excluding stock based compensation, our research and development expenses were approximately $9,400,000 for the Q4 of 2023 compared to approximately $6,000,000 for the Q4 of 2022. Speaker 100:19:22The increase in expenses of $3,400,000 was primarily due to increased clinical and manufacturing costs associated with our Phase 3 HOPE-three trial. Excluding stock based compensation, our general and administrative expenses were approximately $1,900,000 for both the Q4 of 2023 and 2022. Net loss for the Q4 of 'twenty three was approximately $800,000 compared to a net loss of 7 point $7,000,000 for the Q4 of 2022 and net loss for the full year 'twenty three was approximately $22,300,000 compared to a net loss of approximately $29,000,000 for the full year 2022. And with that, we will now open the line up for questions. Operator, go ahead. Operator00:20:07Thank you. Ladies and gentlemen, we will now conduct the question and answer session. Your first question comes from Joe Pantginis from H. C. Wainwright. Operator00:20:25Your line is now open. Speaker 300:20:27Hi, Linda and A. J. Thanks for taking the questions. Good afternoon. First off, so wanted to talk about your regulatory discussion. Speaker 300:20:36So last year you had some pretty clear frameworks that you shared with us again today about the need for Cohort A and giving some manufacturing comparability from Cohort B out of the San Diego facility. And you keep talking about ways to potentially expedite. So I wanted to explore that a little bit. So first question is, I mean, when you look at this, you have the RMAT status, is one of the potentials here rolling BLA because you'll be able to start submitting data quicker as part of the filing and other options you might be considering? Speaker 200:21:14Joe, always a pleasure. Thanks for the question. So, Operator00:21:18I'm going to start answering Speaker 200:21:19by saying, I've been working on this therapeutic for 19 years and where we are right now is just so staggeringly exciting to me. It's sometimes hard to express. But what has been the best part of the last few months has been the careful attention that FDA has been paying to Capricorn and to cap 102. They recognize the positive safety and efficacy data. They've looked at the open label extension data, the HOPE-two data, and they're working very closely with us. Speaker 200:21:48So yes, all options are on the table right now in terms of how to get this across the line as fast as possible. As I mentioned, leadership within CBER is aware of our program and really working very closely with us. We have RMAT. We have rare pediatric disease designation, orphan disease designation. So we have a lot of the bells and whistles that will carry our program as quickly as possible into the arms of DMD patients. Speaker 300:22:15Got it. And then just curiosity for Cohort B, are you strictly needing to show manufacturing comparability or do these patients need to be follow-up for a certain timeframe? Speaker 200:22:30So we belt and suspend the program by building in a full one to one randomized clinical trial with safety and efficacy built in. So it basically is a mirror image of Cohort A. We're working with FDA on what they're going to actually ask us for. What we know and what we can guarantee on is that they're willing to accept the license application on cohort A. What we're going to need from cohort B is still what we're working with them on, but it really is almost something that we've not only been prepared for, but something that comes along very naturally. Speaker 200:23:04So the trial cohort B, let me reemphasize, will be fully enrolled by Q2 of this year. And then we're going to be able to potentially position that as a post marketing commitment for the program. Speaker 300:23:17Got it. And then my last question, you don't mind and thanks for bearing with me. Absolutely. We've been talking about this for several years because we've been excited about the data. So I guess how would you portray the role of the cardiovascular data you've been accumulating through HOPE-two and beyond in the evolution of your regulatory discussions and how much that may or may not be coming into play to date? Speaker 300:23:43Yes. Speaker 200:23:43So it's obviously one of the most important cornerstones of our regulatory strategy, but also in the eagerness of the community to get approval for CAP-ten zero two. To remind you and I stated this, we saw a 4% improvement in ejection fraction, the gold standard of cardiac function in HOPE-two. In HOPE-two, open label extension, we didn't start measuring cardiac function until 2 years in. We'll have 3 years data in the Q2 of this year, so stay tuned for that on cardiac function. But we're pretty convinced that it's going to be one of the major parts of what we're going to look for on our label. Speaker 200:24:17And it's the primary secondary endpoints that has been built into hopefully both cohort A and cohort B. Speaker 300:24:25Got it. Thanks for the added details, Linda. Speaker 200:24:28Absolutely, Joe. Always a pleasure. Operator00:24:32Your next question comes from Kristin Tolska from Cantor Fitzgerald. Your line is now open. Speaker 400:24:39Hi, everyone. Good afternoon. Thanks so much for taking my question. Operator00:24:45Hi, Kristen. How are you? Speaker 400:24:47I'm well. How are you doing? Speaker 200:24:50Good, Sam. Speaker 400:24:52So thinking about the potential for combinations, I completely understand the need for this. But can you comment on what your expectations would be in terms of payer support? And then as gene therapy typically requires a lot more upfront timing to do the different testing, manufacturing, etcetera, Would it be your expectation that CAP-one thousand and two would be the first therapy essentially given in this cascade? And would that be an advantage in case there is some payer pushback? Speaker 200:25:25So we've had really positive feedback from payers, not just at Capricor, but also NS Pharma has done significant work in preparing for market launch. And the way that we're understanding it is that there's going to be therapy that would be necessary for sort of management of dystrophinopathies, right? So the exon skippers and the gene therapies. And because there's not an approved gene therapy theoretically yet, we don't really know how they're going to approach choosing those or both or whatever. But what we do know is that there needs to be a junk of therapy that would manage, and I've talked about this a lot, the immunomodulation or the inflammatory the immunomodulation or the inflammatory response caused by the constant breakdown of protein in the body due to the mutation as well as manage the fibrosis and help to support perhaps the framework laid down by a gene therapy or an exon skipper, which would be a healthier protein. Speaker 200:26:20So we are very confident that payers would find it beneficial to cover both the dystrophinopathy management strategy as well as CAP-ten zero two for the management of the inflammation and the fibrosis. Now in terms of the order in which the therapeutics are given, that we would sort of have to talk to some of the KOLs. Already starting to do some of that market research, obviously, because we're going for our initial label for some of the later stage patients, at least based on the HOPE-three data. We reserve the right to ask FDA to go as young as possible. Colloquially, we always say time is muscle. Speaker 200:26:55And the data has shown that once people get on CAPTAINERT to disease progression is significantly attenuated almost immediately. So in terms of the timing of how that's done, that remains to be seen, but we're very confident that it will be part of the overall treatment paradigm of Duchenne. Speaker 400:27:14Thank you for that. And then we have seen quite a bullish of the 4 to 5 year olds on the Sarepta therapy, which Operator00:27:21I think underscores the unmet need here. So I wanted to ask Speaker 400:27:21what your thoughts are patients wanting therapy, especially because you are going after that non ambulatory population? And then what capacity would you be able to help with given this is half of the patients with DMD? Thanks again. Operator00:27:48Yes. So thank you. So we plan on swinging Speaker 200:27:50the door wide open in terms of what we ask FDA for. The benefit here, unlike many therapeutics, we're going to come into BLA with 4 or 5 years' worth of safety data, tracking children from theoretically age 10 and beyond. So we're going to open the door and see what the opportunity is. Obviously, if I had a child with DMD, I want to get my child on CAP-one thousand and two as young as possible. There's really no downside. Speaker 200:28:16And potentially could even have impact on who knows things like steroid dosing, things that may have ultimate impacts and side effects. In terms of what we're looking for, I think that was your second question. We're looking for utilization of CAP-ten or 2 as early as it becomes available to the community. We're right there with it. Operator00:28:48Your next question comes from Aidan Husainov from Ladenburg. Your line is now open. Speaker 500:28:55Good afternoon, everyone. Good afternoon, Linda, Ajay. Congratulations with the progress this quarter and staying on track with the guidance with the top line in Q4 'twenty four. I have a couple of questions. First, I want to ask you about the potential expansion of indication. Speaker 500:29:15I think you mentioned something in your remarks. And we this the most natural expansion, I think we talked about it, it was the Becker dystrophy that we see tremendous increase in value in other Becker companies. And given your cardiomyopathy focus and focus on improving skeletal cardiac muscle function, could you expand a little bit on your any potential efforts that you're making regarding that expansion? Operator00:29:53Yes. Thanks, Aidan, and always good to talk with you. So we've been talking about this for Speaker 200:29:57a while. Obviously, there's tremendous opportunities for expansion. As I mentioned, the manufacturing paradigm is set. We have a potency assay that's been accepted by FDA. We understand the mechanism of action and how to make the cells and we certainly are poised to expand our efforts. Speaker 200:30:13Becker is certainly one that's of great interest, especially since the primary manifestation later in life is the cardiomyopathy, which as we've talked about is one of the main targets that CAPTAINR II seems to ameliorate. Having said that, we are right now focusing almost all of our efforts on getting CAP-ten zero two across the line for DMD. We're working on the BLA. We're working on a launch strategy, commercialization strategy. And so indication expansion will come behind that and we'll provide more color to you into the market as those opportunities become available. Speaker 500:30:49Okay, understood. Another question I have is regarding the HOPE TO open extension, open label extension trial results in the second quarter. So what are your expectations regarding this data? Speaker 200:31:06Well, we have 3 year data coming out in the Q2 of this year, as I said. The 2 year data was extraordinary. We presented that most recently at PPMD and then we'll be sharing some more data at the Muscular Dystrophy Association meetings next week in Orlando. We have every expectation based on the anecdotes that we hear from the subjects and their families that this trend of stabilization of disease and attenuation of disease progression will continue. We're really looking forward to seeing the MRI data because that will be 2 years of sequential cardiac function data. Speaker 200:31:40And we have every reason to believe that we should be able to stabilize heart function as well. So, we also have so many families that call us, that tell us that their sons are able to do what they weren't able to do before or they can't wait for their next infusion. Can we please get in sooner because we feel it wearing off after 3 months. So I'm very much looking forward to seeing that data and then ultimately sharing it with all of you. Speaker 500:32:10Understood. Thank you for that. And the last question is regarding the European discussions, discussions with potential European parties. So in your discussions with them, first of all, if you could give us a little bit of insight, are these like established players, are these new players? And what are the typical questions that he has there? Speaker 500:32:31What is the which part of CapTel or to value they are most Speaker 200:32:43community is as interested in getting therapies across the line for DMDS is the U. S. Authorities. They have a little bit different strategy in Europe. As you probably are aware, there's a little bit of different way of going about it. Speaker 200:32:56The parties that we're talking to are some new and some established. I think we've been on the radar for several larger companies for a long time now. And with the data coming around the corner, they were paying more attention. Perhaps one of the best things is that our San Diego manufacturing facility can be EMA qualified. So we can make doses here and ship them Europe, which is an added benefit. Speaker 200:33:20And in terms of the questions that we get, I think would be the typical ones, which is regulatory strategy, getting CAP2 across the line, what clinical trial work Speaker 100:33:29will be Speaker 200:33:29needed. From our standpoint, it's probably going to be fairly straightforward. And we definitely look forward to making Cap Tenet 2 available worldwide. Speaker 500:33:42Okay. Thank you so much and congratulations for the progress this quarter. Speaker 200:33:47Thanks, Aidan. Take care. Operator00:34:05There are no further questions at this time. I'm turning it over to the management for closing remarks. Speaker 200:34:12Before we conclude today's call, I want to extend my sincere gratitude to the patients, their families, the clinicians and our partners at Nippon Shinyaku and NS Pharma and of course at FDA who continue to work with us to bring CAP-one thousand and two closer to potential approval. Again, thank you to everyone who joined us this afternoon and I look forward to seeing you at meetings in the future. Operator00:34:37Ladies and gentlemen, this concludes today's conference. Thank you for joining. You may now disconnect.Read morePowered by