Capricor Therapeutics, Inc.

Welcome to CapiCorps Fourth Quarter and Full Year 2022 Financial Results and Corporate Update Call. At this time, all participants are in a listen-only mode. A brief question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Mr. A.J. Bergman, Capricorn Chief Financial Officer. Mr. Bergman, you may begin.

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 call. 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 preclinical and 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, and our possible 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. Your caution 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, Chief Executive Officer.

Thank you, AJ. Good afternoon, and thank you for joining our fourth quarter and full year 2022 conference call today. We are pleased with the progress we have made over the past year and into the first few months of 2023. Today, I will provide important updates on our Duchenne muscular dystrophy program, our Exosome platform, as well as corporate updates. These past 15 months for Capricor and our CAP 1002 Duchenne program have seen many highlights, and we continue to execute on our three key priorities for our DMT program, which are as follows. First, advancing our HOPE III Phase III trial. Second, is continuing to engage with the FDA to bring CAP-1002 to patients as expeditiously as possible. And the third is securing commercial partnerships outside of the United States to ensure that CAP-1002 reaches patients with DMD around the world. I will begin with an update on our Phase III, HOPE III clinical trial, which continues to enroll very well. As of today, we have 11 active sites and have treated over 30% of the currently planned sample size of up to 68 patients. Our plans to conduct an interim analysis for sample size re-estimation and analysis of conditional power remain unchanged, and we anticipate that these results will be available in the fourth quarter of this year. In addition, we are continuing to bring on additional sites with the goal to have a majority of our targeted sites activated in the second quarter of this year. In parallel, we continue to treat patients in the Open Label Extension, or OLE, portion of the HOPE II Phase II clinical study. These patients are going into their fifth year of follow-up, and based on the positive results to date, at this time, we are continuing to dose these patients in a third year of OLE to further demonstrate the safety and potential long-term durability of the therapy. We continue to see a significant slowing of the disease for patients treated with CAP-1002 including for these patients who were initially on placebo. The data presented at both 12 and 18 months of HOPE II, we plan to report the 24-month data in the second quarter of this year. The data presented at both 12 and 18 months continue to show an average of 65% slowing of disease, similar to the 70% slowing we saw in the randomized portion of HOPE II. We plan to report the 24-month data of the HOPE II OLE study in the second quarter of this year. This data is fundamentally important in continuing to support the long-term safety and efficacy of CAP 1002 in DMD patients. We are thankful to the patients and their families for their continuous commitment to working with us on demonstrating the benefits of CAP 1002. To remind you, the 12-month data from the OLE was presented at the World Muscle Society last October, and the continuation data at 18 months were recently presented with supporting videos last month on a PPMD, or Parent Project for Muscular Dystrophy, hosted webinar. Both were presented by our lead investigator, Dr. Craig McDonald. I am also pleased to note that these 18-month results were recently accepted for a late-breaking presentation at this year's Muscular Dystrophy Association, or MDA, clinical and scientific conference being held in Dallas, Texas from March 19th to 22nd, 2023. Together with the long-term efficacy safety profile and the mechanistic effect of CAB 1002, we continue to believe that the therapy, if approved, could serve as a potential anchor therapy for the boys and young men impacted by this disease. With regard to our second priority, As you know, since we have been granted RMAT or Regenerative Medicines Advanced Therapy designation, we have access to more intensive and frequent guidance from FDA. Under the RMAT designation, we recently met with the FDA in a productive Type B CMC or Chemistry Manufacturing and Controls meeting to discuss our manufacturing plans for the HOPE III trial and production of commercial-scale CAP-1002 in anticipation of a potential BLA application. The meeting was constructive, and while we are awaiting the meeting minutes, we believe that we will be in a position to meet FDA's expectations for BLA with some adjustments to our planned clinical and manufacturing activities. we were able to outline our plans with FDA about our potency assay and other product release criteria to support commercialization. This is a great accomplishment and facilitates our potential path to BLA. Based on our initial assessment of the meeting, we believe that we will need to treat patients in HOPE III with products manufactured from our new San Diego facility to support a potential BLA application. In anticipation of this potential requirement, we designed our San Diego facility to produce commercial-scale GMP doses of CAB 1002, and we expect this facility to be functional in the third quarter of this year. We will provide more color on any potential impact to HOPE III as it becomes available. Furthermore, at the request of the FDA, we have submitted the interim results from our HOPE II open label extension study for their review as we continue to discuss our pathway towards potential registration. Now, turning to our commercial partnership strategy. In early 2022, we entered into a commercialization and distribution agreement with Nippon Shinnyaku, a leader in the field of Duchenne muscular dystrophy, with one of the few therapeutics for DMD approved in the United States. This agreement provided a significant upfront payment of $30 million, and there are potentially additional milestone payments of up to $705 million built into the agreement, As I have noted before, it is possible that we may start to trigger these milestones in 2023 should we continue to execute according to our plan. Also, as announced in February, I am very pleased to highlight that we have entered into a second agreement with Nippon Shinnyaku for the distribution rights to CAP 1002 for Duchenne Muscular Dystrophy in Japan, pursuant to which we expect to receive a $12 million upfront payment and will potentially receive additional milestone payments of up to approximately $89 million and a meaningful double-digit share of net product revenues. As with the U.S. agreement, these milestones are based on clinical, regulatory, and sales-based achievements. This agreement is similar to the U.S. agreement with Nippon Shin'yaku in that Capricorn will be responsible for the clinical development and manufacturing of Cap-10-02, and Nippon Shin'yaku will be responsible for the distribution of Cap-10-02 in Japan if the product is approved. This has the potential to reach the estimated 3,500 patients that suffer from DMD in that region. In addition, Capricorn will hold the marketing authorization in Japan if the product is approved in that territory. Nippon Shinjaku will be able to leverage the expertise and infrastructure already established for Viltexo, which is their exon skipping drug that is already approved in Japan. Our plan is to engage pharmaceuticals and medical device agency in Japan, otherwise known as the PMDA, to gain an understanding of the regulatory process for approval of CAP10 or 2 for GMD in Japan. We will provide updates on this program as they become available. Lastly, we remain committed to securing additional partners in other markets around the world, with Europe being the main focus for us at this time. In addition, we are very proud to announce the results of HOPE II, our Phase II trial, and its publication in The Lancet was recently selected as one of 2022's Top 10 Clinical Studies in the World by the Clinical Research Forum. As you recall, the paper highlights the safety and efficacy of CAP-1002 in slowing the progression of both the skeletal and cardiac muscle myopathy in DMD. Overall, we are very pleased with the progress of our DMD program. And we look forward to sharing further updates from our interaction with FDA, our progress with HOPE III, and the development of potential additional partnerships in new territories. Now, let me turn to our exosome platform technology, which leverages the natural cell signaling communication system of the body. We are harnessing exosomes to serve as a novel drug delivery system with broad therapeutic applications. We continue to make progress on the manufacturing and production of exosomes with an emphasis on ensuring scalability, reproducibility, and quality control. Building on expertise and learnings from our core program for CAP 1002, for which the mechanism of action is mediated via exosomes, we have developed an extensive body of evidence and know-how on these three fronts. our strong foundation has supported further downstream efforts for innovative therapeutic payload loading methods and tissue-specific targeting. Our proprietary StealthX expression platform is at the core of our Exosome program, and we are focused on the development of two broad modalities, vaccinology and precision therapeutics. In January of this year, we published data on the online server BioRxIV.com, on our most advanced application of StealthX, which generated two vaccine candidates that independently and in combination induced a strong immune response against two SARS-CoV-2 proteins, Spike and nucleocapsin. Results show that this multivalent, protein-based vaccine candidate has the potential to achieve potent, longer-lasting immunization, broaden reactivity, and improve T cell responses with only nanograms of protein necessary without any added adjuvants. The data from the study suggests that StealthX could deliver a more potent vaccine with broader immunity than is currently available, all with an efficient development cycle that is competitive to the mRNA platform. The reason this data is so important is twofold. First, it allows us to use tiny amounts as in nanograms of protein to drive an immune response. suggesting that could be the case with any vaccine candidate. And second, it also highlights the ability to use a multivalent strategy. This technology opens the door for combination vaccines and, most importantly, pairs the strength of a recombinant protein vaccine with the ease of development of an mRNA vaccine. To that end, it takes approximately 12 weeks for our team to go from antigen characterization to vaccine candidate. Our current strategy is focused on securing partners who will provide capital and additional resources to bring this program into the clinic should we decide to do so. The other potential application of our Delta X platform is to develop precision therapeutics. The objective here is to more effectively deliver a payload to targeted cells or tissues, thus decreasing the overall systemic exposure to the payload while enhancing higher concentrations at the targeted site. We are targeting three tissues of interest for which we believe our exosomes can deliver a differentiated platform and potentially improve clinical outcomes for patients. To clarify, excuse me, the exosome technology, primarily StealthX, is a platform, but we are currently planning on internally developing the technology for specific indications using both of these approaches, vaccines and targeted therapeutics. We also remain open and, in fact, have initiated external discussions with potential partners to deliver therapeutic payloads from their portfolio for already established clinical programs. This approach allows us to optimize the clinical benefits of potential partners compounds with a preference for characterized therapeutic candidates. We continue to diligently invest in the future of Capricorn. We have assembled a world-class team and continue to maintain a lean profile that is capital efficient. With the 74 employees we have today across the organization, our hiring plan is nearly complete for 2023. Our financials announced prior to this call report over $41 million in cash, not including the addition of our $12 million upfront payment, which we expect to receive this quarter from Nippon Shin-yaki, and provides us with a runway to execute on our strategy. We are pleased with the advancements of our late-stage clinical development pipeline and the growing body of work with our Exosome platform technology. We look forward to continuing and accelerating this momentum and to executing towards our upcoming milestones. Now with that, I will turn the call over to our Chief Financial Officer, A.J. Bergman, to run through our financial results for the fourth quarter and full year 2022. A.J.?

Thank you, Linda. This afternoon's press release provided a summary of our fourth quarter and full year of 2022 financials on a GAAP basis. You may also refer to our annual report on Form 10-K which we expect to become available shortly, will be accessible on the SEC website as well as the financial section of our company website. As of December 31, 2022, companies' cash, cash equivalents, marketable securities totaled approximately $41.4 million compared to approximately $34.9 million on December 31, 2021. Based on our current operating plan, the company's cash position is expected to be sufficient to support operations into the fourth quarter of 2024. Turning now to the financials. In the fourth quarter of 22, our net cash used in operating activities was approximately $6.9 million. For the fourth quarter of 2022, excluding stock-based compensation, our research and development expense was approximately $6 million compared to approximately $4.1 million in Q4 2021. Again, excluding stock-based compensation, our general and administrative expense was approximately $1.9 million in Q4 2022 and approximately $1.5 million in Q4 2021. Net loss for the fourth quarter 2022 was approximately $7.7 million compared to a net loss of approximately $6.2 million for the fourth quarter of 2021. Net loss for the full year 2022 was approximately $29 million compared to a net loss of approximately $20 million for the full year 2021. I would also like to note briefly that in light of the recent FDIC action and market volatility, Capricor holds no deposits at Silicon Valley Bank at this time.

We will now open up the line for questions. Thank you. We will now be conducting a question and answer session.

If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star 2 if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.

One moment, please, while we pull for questions.

Our first question comes from Joe Patchingens with HC Wainwright. Please go ahead.

Hey, Linda and AJ, good afternoon. Thanks for taking the question. A few questions, if you don't mind. First, I guess a two-parter with regard to your recent FDA interaction. So I know you don't have the minutes as of yet, so I know some of your comments might be a little more or might have less comments because of that. But anything you can talk to regarding, you know, the outstanding punch list, key things that the FDA have been looking at, And also with regard to the potency assay, can you talk to say the simplicity of that or is anything outstanding to be able to be in agreement with the potency assays? And then I'll go to part two.

Yeah, thanks, Joe. That was two parts already, but you have as many questions as you like. So, first of all, let me just start off by saying that one of the big hurdles for any cell therapy company is to develop a potency assay that is reliable, repeatable, and can be validated by quality measurements, QA and QC, and we have done that. And we've now presented the plan to FDA. We think it is probably one of the most innovative ways of looking at potency in a cell therapy because it actually takes advantage of looking at positive clinical data and then relating it back to what was actually active in the cells at the time. This is a giant hurdle to overcome. We have, you know, some things to even out in terms of making sure that it's appropriately qualified and validated, but we're on the right path, and we have good interactions with the FDA and the CMC. So that's A very big win. We also had a big win in terms of identity criteria and some of the other supportive assays. So really effective and very collaborative meeting with FDA on the CMC. And as you know, this really paved the way for commercialization of CAP-1002, one of the big hurdles that all the companies have had to jump over. In terms of some of the other, you know, sort of tidbits and takeaways from the FDA meeting, you know, they did mention that they'd like to see doses from our San Diego facility, which is our planned commercial launch facility, to be used in patients prior to the VLA. And so we're waiting for feedback on that in terms of how that's going to be manifest. And then we're working with our statisticians, our own regulatory team, and our FDA consultants to to make sure that that path to commercialization is as smooth and easy as possible.

Got it. So actually that's a good segue to the last part of the FDA. So with regard to including samples or doses from the new facility, how does that apply to new – does that apply only to new patients, or will you be able to then, you know, change the doses to patients that have currently been enrolled? Or is there a fixed number of patients or a ratio that has to be treated by the new doses coming out of the San Diego facility?

Yeah, so thanks. So, you know, one of my real benefits has been our good working relationship with FDA, especially with the CMC group. And we've been working with the same group since we really started a long time ago with Caduceus, so they've followed our entire trajectory. And what they cautioned us about a while ago was not to mix doses from both facilities, that they are actually considered separate products and that they need to be validated independently. So they will be new patients that will be treated with doses from San Diego. And then those will be analyzed together and separately. We don't know the implications as yet. We obviously are going to plan further conversations with FDA. We're going to be reaching out to schedule a clinical meeting very soon so that we can get further clarity. And of course, as soon as we have. An idea of potential implications to the program, we'll let everybody know. But I want to highlight that this is an incredibly positive step. We have a roadmap now to a biologics license application for this product. We know exactly what we need to do. We still need to iron out the edges in terms of number of patients, but this is a really good sign for us.

Got it. And then I guess my last one is more of a logistical question. With regard to the expected, you're saying the interim analysis in the fourth quarter, I guess, is this more of a continuous plan or are we actually looking at real data that the street can take away from?

Yeah, so as of right now, we have built it as a sample size re-estimation, which is basically a blinded look at the data to determine whether or not we think it's going to hit the promising zone. And so we don't have a futility built in as yet, nor do we have a stop for success. It's just do we need to add more patients to potentially achieve success? Of course, we're working with our statistical team right now to determine if there's, you know, other ways that we might want to do an interim analysis to that. We have no further caller at this time, but we will if we do.

Perfect. Thanks, Linda.

Thanks, Joe. Be well.

Next question comes from Michael Onkiewicz with Maximum Group. Please go ahead.

Hey there. Thank you for taking my question, and congratulations on the great progress over the quarter and over 2022 as a whole. So I guess to start off, I'd like to just ask, in terms of the agreement with Nippon Shinnyaku in Japan, do you have any thoughts on your development plans for the Japanese market, in particular, if there are any thoughts on using the ASRM pathway to reach Japanese patients with minimal additional clinical work?

Yeah, so, exactly. Right now, the deal is, you know, freshly inked, and we're, you know, delighted to have them as our partner for Japan. We think they're our ideal Japanese partner, and this was, you know, in the makings for a while. We are the marketing authorization holder, so we will be responsible for the clinical work. We're glad for that because we have a very seasoned regulatory team that will be able to take advantage of our learnings in the U.S., apply them to our opportunities with the PMDA, and come up with a very efficacious plan to get CAP-1002 to market in Japan as well.

All right. Thank you for that. And I'd like to follow up again on Nippon Shin-Yaku. Since they're already in the DMD space with an exon-skipping drug, does their partnering in Japan give further confidence for the strategy to position CAP-1002 as a backbone therapy in DMD?

Yeah, absolutely. And, you know, we've been very strategic in selecting Nippon Shin-Yaku and Aspharma as our partner. They're ideal. They have a commercial product in Duchenne muscular dystrophy. They have a sales and distribution network. Their MedAffairs group is, you know, focusing solely primarily on DMD. And so, yes, it's an extension of our strategy to make sure that CAPTEDR2 will be available to every DMD patient ultimately.

All right. Thank you for that. And then just one last one from me on the HOPE3 trial. I'd like to see if you could just talk a little bit. about what assumptions you currently have for effect size and how the study is powered.

Yeah, so we haven't really disclosed the full magnitude of our statistical analysis plan. It's still in late-stage development. You're not required to submit that as yet. Currently, what I can tell you is that the effect size that we're looking for is less than what we saw in HOPE II, and we're a power to 90% power. So we have a really nice sort of benchmark of success and also potential we're able to take advantage of the positive HOPE II data to power the study.

All right. Thank you very much for that, Linda, and congratulations once again.

Thanks so much. Have a great day. Next question comes from Aiden Hewsonov with Leidenberg.

Please go ahead.

Hi, Linda. Hi, Eiji. Congratulations for the quarter and with the Nippon Shinya deal. I have several questions. First of all, I want to ask you, how do you assess the European market? Given that Europeans are not really receptive to excellence keepers, but how do you think this market is going to evolve, the DMD market is going to evolve?

Yeah, so obviously, you know, EMA is taking somewhat of a different approach than FDA and, you know, DMD and other often diseases as well. What we are doing is positioning CAP-10-02 worldwide as an anchor or backbone therapy. We think that CAP-10-02 will serve in great conjunction with gene therapies or any other approved therapies. The mechanism of action is supportive with its anti-inflammatory, pro-regenerative, anti-fibrotic mechanism of action. And so, you know, our goal right now is to continue to work with the worldwide regulatory agencies to get them to the same point and to get our therapy around the globe as fast as possible.

Okay, understood. Thank you. One question I have regarding the enrollment in the HOPE3 trial. So I think you mentioned 30% of 6-8 patients, which is around 2021. I think in the last quarter it was 18 patients. So is there any bottleneck in terms of the enrollment? And I also want to ask you what you think about the guidance. I think the last time guidance was mid-2023. Now I think the guidance is end of 2023, fourth quarter 2023. So could you comment on the enrollment process in the HOPE III trial?

Yeah, so we started off relatively slowly. Then we hit a really nice enrollment phase in the fall. And then as usual, and especially in pediatric trials, you see a significant slowdown around the holidays. Who wants to spend their holiday vacation getting an infusion? Enrollment has picked up beautifully in the first quarter. We're very excited with... with the enrollment and the infusions as they're going now. We did guide last quarter that we were going to the end of Q3 for full enrollment, and we are right on track for that, for enrolling the 68 patients currently planned for infusion. And so we look forward to providing more color on that as the year goes on.

Okay, I understood. So that's the holidays. All right. I want to ask you a little bit about the Nippon Shinnyaku deal, $12 million up front. So could you tell us, was it competitive? Did you have other contenders? And why did you decide to make a deal now but not after you hear back from Hope City at the end of third or fourth quarter?

Yeah, so... We really had a long time ago identified the fact that Nippon Shin'yaku would be an ideal Japanese partner. They're based in Japan. They've got an approved product for DMD in Japan. And it was an extension of a relationship that was already solid and strong. So, of course, we talked to other parties that felt that this would be the best one for us. In terms of the economics, the economics were very favorable, proportionally larger than the U.S. deal, taking advantage of the fact that the product is more advanced and has more clarity towards the path towards commercialization in the U.S., In terms of waiting, you know, there's really no reason to wait because we can get the party started, right? We can start thinking about getting the product into Japan and commercialize them. We can also strategize better as to how to take advantage of the HOPE III data to promote with PMDA. So we wanted to get all of those ducks in a row, and we felt very pleased not only with the partner but with the economics.

So will you get from the milestones from the Japanese deal for the HOPE III readout as well on top of the U.S.

deal milestones? No. So the U.S. deal is constructed around milestones based on HOPE III, and then the Japanese deal is based on milestones constructed around activities that will happen in Japan. But, of course, all of it was augmented by the promise of HOPE III.

Okay. Understood. Understood. Okay, so Linda, you have enough cash to invest in other programs. So what do you see as a priority going forward? Would you invest in other muscular dystrophies or would you invest in vaccine business? So how do you see the capital allocation going forward, given that you collect all these milestone payments this year and hopefully next year as well?

Yeah, thanks for the question. So obviously, and you know, we've been messaging for a while that we are laser focusing on cap 10 or two and its development for DMD. Our number one goal is to get that product approved. So we've got money going into hook three. Our manufacturing facility is, you know, getting up and running. We're going to, you know, make sure that the product is ready for, Commercialization program is developing nicely sort of behind the scenes. It's going exactly as we had planned, which is that we have very promising preclinical moving towards clinical data, both in and a potential therapeutic development. Just like with CAP 1002, we're going to make sure that we have the right partners identified and then potentially move the exosomes forward once we have some type of business development opportunity with the exosomes. And that worked really successfully with CAP 1002 and HOPE III, and so we're going to exercise the same strategy with the exosomes. I think our proprietary StealthX platform is extraordinary. I can't emphasize that enough, and you gave me a moment to speak about it, so I'm going to, which is as novel, as innovative, it takes vaccinology to a new level with combining the ease of an mRNA vaccine with the power of a recombinant protein vaccine, and then this can also be used for protein replacement therapies. So we have a lot going on here at Capricorn. We're really looking forward to continuing to deliver on our milestones.

Okay, thank you very much for taking my questions and congratulations for the quarter.

Thank you very much. Look forward to seeing you soon in New York. Again, if you would like to ask a question, please press star 1 on your telephone keypad. Next question comes from Brian Kordek with Bullbeer. Go ahead.

Hi, Linda. Hi, AJ. Great quarter. Lots of information to digest. I'm happy to, as an investor, to see how this is going.

We're happy to have you as an investor, Brian.

Oh, yeah, I'm listening.

As you know, I've been here for a while, and I'm looking forward to it. I just had a couple of questions. In terms of the FDA minutes when you get those, are you going to just give a summary or will you have another call to release it? How are you going to address that? Because you should have those relatively soon.

Yeah, yeah. So usually there's not, you know, this is not a situation that would call for a call. We don't expect any surprises. I kind of gave sort of our sort of back of the envelope assessment of the meeting. We will obviously, you know, adjust our plan based on feedback from FDA and our continued interaction. We really do need to get in front of them from a clinical perspective to make sure that we're all aligned there. But I can tell you that, you know, even the clinical reviewer who participated in our call was very positive about the progress CAP-10 and 2 is making. And so, you know, we expect to message this as data becomes available, but not expecting anything tumultuous.

All right. And in terms of the support you've been getting from the patient family advocacy groups, which I know has been growing quite a bit, and the last webinar you had was tremendous, how is that going along? Are you seeing a lot more? attention from them? Where is that leading you?

Yeah, so, you know, I'm very proud to say that we're getting a lot of positive feedback from patients and families with DMD for CAP10-02. And I'll just highlight very briefly the reasons why. Number one, and perhaps most importantly, CAP10-02 has a long and established safety profile. So, you know, the kids don't get sick from it. There's no safety sequelae. They really don't mind it. It's a 45-minute infusion once every quarter, super easy for them. And remember, you know, these are kids and parents, so the number one thing they're thinking of is the safety of their families. The other thing is they're seeing it work. So we're getting anecdotal feedback, and you also might have seen on the PPMT webinar we did a few weeks ago that, you know, kids seem to be doing better on CAP-1002. And that's supported by calls that I'm regularly getting from families either asking to get CAP-10 or 2 or to continue in the open-label extension. That is further validated by the fact that we're going into our third year of open-label extension because the families don't want to give it up. So we're positioning it as backbone therapy, as I've said a few times in this call and other places, that we think it'll go along with gene therapy or whatever else is approved and continue to support the health and well-being of patients with DMD.

As a little point to that, I don't think people are really understanding the gravity of how important it was for that webinar you had a couple weeks ago and the groundswell that you've had, the people that have been pushing for it from the families. And it would be really great if some of the analysts on this call could reach out to those people and understand how important that was. Yeah, I think it was great. Now, in terms of the meeting in Dallas that you're going to, were you, since it's such a last-minute addition, were you asked to come to that, or did you ask them to present?

No, so we did, you know, sort of the classic road of submitting an abstract of the data, and they responded in classic fashion by accepting it. We did it sort of just, you know, in time, and so we're really looking forward to that presentation. We were there last year. It's a wonderful meeting, great opportunity to present to patients. but also get in front of, you know, the investment community as well. You know, one of the things that we, you know, really are starting to highlight again in the backbone therapy arena is the cardiac implications of CAP-1002. And the families now are sitting up and saying, wait a sec, you know, we have a cohort of kids even with gene therapy that seem to be doing better. They're staying on their feet longer. They're more active. but their hearts are worse, and this is unequivocal. So, there's more and more focus on, you know, CAP-10 or 2 will provide, you know, an added, an addition to the treatment paradigm by hopefully supporting the wellness of their hearts. Okay.

Well, I'm really happy the way things have progressed. I have two questions for AJ. AJ, In terms of the shares that increased, I saw you sold about roughly a half a million at 616 when the stock was running. Where did the other half a million shares come from? And then final question for you is, have you adjusted your pricing model? Because I know we talked about this last quarter. You were going to think about it because I got to believe with the with the pricing structures that are out there now, you could definitely increase your 6,650 that you projected for the regimen to at least 20 to 25% higher. Have you looked at that yet?

Yeah, hey, Brian, thanks for the question. To your first point, just to be very clear, I laid this out in our earnings press release. Hopefully you caught it, but it was a couple minutes before the call. We sold shares under our ATM early on in the fourth quarter, predominantly October. All of those numbers and sales were disclosed in our last quarterly filing. And in this quarter, and really all of December following our last quarterly call, which I think occurred on November 10th, we have sold no shares under our ATM. So as you mentioned, we took advantage of our stock being in a nice position back in those days, but have sold no further shares. In terms of the number delta change in shares outstanding, most likely due to option exercises, et cetera, but nothing triggered right to the ATM. In terms of your next question, you know, obviously the reimbursement is critical for us and our success down the line. We have a really nice structure with NS Pharma and Nippon Shinnyaku in terms of what we will receive as a meaningful double-digit support. split of net product revenue. We have, you know, early estimates of what we feel that this drug will be priced at. It will ultimately come down to how good the data is both in the skeletal and cardiac arena. But it's something, of course, we're very focused on and will be with our partners. These exon skipping drugs that are approved in the U.S. have very high price tags, and hopefully CAP-10-02 will be right in that range as well.

Okay. And can you comment at all on Does Nippon have any kind of right of first refusal on any type of acquisition of the company outright?

Yeah, this is something we haven't gotten into and disclosed publicly. We have a roe fur and a roe fin. That's all we've basically stated. But now that Japan has been established, it's pretty clean. So it's just not something we've got into the details right now at this point. Okay.

All right, well, I appreciate it. Thank you so much, and keep up the great work.

Thanks, Brian. See you soon. Yep.

At this time, I would like to turn the floor back over to Tech Course Management for final thoughts.

Yeah, thank you, Operator, and thank you to everyone who had joined us this afternoon. Before we conclude today's call, I would like to thank the entire Capricor team, our investors, and the many people who have been supportive along the way, especially our patients and their families.

Once again, thank you for joining. This concludes today's teleconference. You may disconnect your line at this time.

Thank you for your participation, and have a great day.