Capricor Therapeutics Inc (CAPR) 2022 Q4 法說會逐字稿

Good afternoon and welcome to Capricor fourth-quarter and full-year 2022 financial results and corporate update call. (Operator Instructions) As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Mr. AJ Berjmann, Capricor Chief Financial Officer. Mr. Berjmann, 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. You 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, 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 has seen many highlights, and we continue to execute on our three key priorities for our DMD program, which are as follows. First, advancing our HOPE-3 Phase 3 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 3 HOPE-3 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 plan to conduct an interim analysis or 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-2 Phase 2 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 continue to show an average of 65% slowing of disease, similar to the 70% slowing we saw in the randomized portion of HOPE-2. We plan to report the 24-month data of the HOPE-2 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'm 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 19 to 22, 2023. Together with the long-term efficacy, safety profile, and the mechanism of -- mechanistic effect of CAP-1002, we continue to believe that the therapy, if approved, can 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 Medicine 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-3 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 a 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-3 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 CAP-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-3 as it becomes available. Furthermore, at the request of the FDA, we have submitted the interim results from our HOPE-2 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 Shinyaku, 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 Shinyaku 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 revenue. As with the US agreement, these milestones are based on clinical, regulatory, and sales-based achievements.

This agreement is similar to the US agreement with Nippon Shinyaku, in that Capricor will be responsible for the clinical development and manufacturing of CAP-1002, and Nippon Shinyaku will be responsible for the distribution of CAP-1002 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, Capricor will hold the marketing authorization in Japan if the product is approved in that territory.

Nippon Shinyaku will be able to leverage the expertise and infrastructure already established for Viltepso, 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 CAP-1002 for DMD 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-2, our Phase 2 trial, and its publication in The Lancet was recently selected as one of 2022's top ten 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-3, 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 on our most advanced application of StealthX, which generated two vaccine candidates that independently, and in combination, induce a strong immune response against two SARS-CoV-2 proteins, spike and nucleocapsid.

Results show that this multivalent, protein-based vaccine candidate has the potential to achieve potent, longer-lasting immunization, broadened reactivity, and improved T-cell responses with only nanograms of protein necessary without any added adjuvant. The data from this study suggest that StealthX could deliver a more potent vaccine with broader immunity that is currently available, all with an efficient development cycle that is competitive to the mRNA platforms.

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 StealthX 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, 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 payload 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 Capricor. 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 Shinyaku and provides us with the 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, AJ Bergmann, to run through our financial results for the fourth-quarter and full-year 2022. AJ?

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 and will be accessible on the SEC website as well as the financial section of our company website.

As of December 31, 2022, company's cash, cash equivalents, and 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.

(Operator Instructions) Joe Pantginis, H.C. Wainwright.

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 interactions. 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 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.

Yes, 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 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 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 on the CMC. So that's a very big win. We also had a big win in terms of identity criteria in some of the other supportive assay. So really effective and very collaborative meeting with FDA on the CMC. And as you know, this really paves the way for commercialization of CAP-1002, one of the big hurdles that all of the companies have had to jump over.

In terms of some of the other sort of tidbits and takeaways from the FDA meeting, 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 BLA. 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 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 patients -- does that apply only to new patients? Or will you be able to then 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?

Yes. So 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 [Kadusia]. So they 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 there 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, I will 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 -- 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?

Yes. 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 is 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 other ways that we might want to do an interim analysis. To that, we have no further color at this time, but we will if we do.

Perfect. Thanks, Linda.

Thanks, Joe. Be well.

Michael Okunewitch, Maxim Group.

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 Shinyaku 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?

Yes. So exactly. Right now, the deal is freshly inked, and we're delighted to have them as our partner for Japan; we think they're our ideal Japanese partner. And this was 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 US, apply them to our opportunities with the PMDA, and come up with a very efficacious plan to get that CAP-1002 to market in Japan as well.

All right. Thank you for that. And I'd like to follow-up again on Nippon Shinyaku. 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?

Yes, absolutely. And we've been very strategic in selecting Nippon Shinyaku, NS Pharma, as our partner. They're ideal. They have a commercial product in Duchenne Muscular Dystrophy. They have a sales and distribution network. Their Med Affairs group is focusing solely primarily on DMD. And so yes, it's an extension of our strategy to make sure that CAP-1002 will be available to every DMD patient ultimately.

All right. Thank you for that. And then just one last one from me. On the HOPE-3 trial, I'd like to see if you could just talk a little bit about what assumptions you currently have for effect size and what this -- and how the study is powered.

Yes. So we haven't really disclosed the full magnitude of our statistical analysis plan. It is still in late-stage development, and we'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-2, and we're powered to 90% power. So we have a really nice sort of benchmark of success and also we're able to take advantage of the positive HOPE-2 data to power the study.

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

Thanks so much. Have a great day.

Aydin Huseynov, Ladenburg.

Hi, Linda. Hey, AJ. Congratulations for the quarter and with the Nippon Shinyaku deal. So I have several questions. So first of all, I want to ask you, how do you assess the European market? So given that Europeans are not really receptive to exon skippers, but how do you think this market is going to evolve, the DMD market is going to evolve?

Yes. So obviously, EMA is taking somewhat of a different approach than FDA in DMD and other orphan diseases as well. What we are doing is positioning CAP-1002 worldwide as an anchor or backbone therapy. We think that CAP-1002 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, 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 HOPE-3 trial. So I think you mentioned 30% of 68 patients, which is around 2021. I think at 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 guide. 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-3 trial?

Yes. So we started off relatively slowly. Then we hit a really nice enrollment pace 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 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, understood. So that's the holidays. All right. I want to ask you a little bit about the Nippon Shinyaku deal, $12 million upfront. 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-3 at the end of third or fourth quarter?

Yes. So we really have, a long time ago, identified the fact that Nippon Shinyaku would be an ideal Japanese partner. They are 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, but felt that this would be the best one for us. In terms of the economics, the economics were very favorable, proportionately larger than the US deal, taking advantage of the fact that the product is more advanced and has more clarity towards the path towards commercialization in the US.

In terms of waiting, 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, and we could also strategize better as to how to take advantage of the HOPE-3 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.

But will we get from them milestones from the Japanese deal for the HOPE-3 readout as well on top of the US deal milestones?

No. So the US deal is constructed around milestones based on HOPE-3, 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-3.

Okay. 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?

Yes. Thanks for the question. So obviously, and we've been messaging for a while, that we are laser focusing on CAP-1002 and its development for DMD. Our number one goal is to get that product approved. So we've got money going into HOPE-3. Our manufacturing facility is getting up and running. We're going to make sure that the product is ready for commercialization.

Our exosome program is developing nicely, sort of behind the scenes is going exactly as we had planned, which is that we have a very promising preclinical moving towards clinical data both in vaccinology 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 works really successfully with CAP-1002 and HOPE-3. And so we're going to exercise the same strategy with the exosomes.

And by the way, 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 it's novel, it's 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 a protein replacement therapy. So we have a lot going on here at Capricor. 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.

(Operator Instructions) Brian Corday, BullBear.

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

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

Yes. So usually there's not -- this is not (inaudible) for a call. We don't expect any surprises. I kind of gave sort of our back-of-the-envelope assessment of the meeting. We will obviously adjust our plans 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 even the clinical reviewer who participated in our call was very positive about the progress CAP-1002 was making. And so, we expect to message this as data becomes available. But not expecting anything tumultuous.

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?

Yes. So I'm very proud to say that we're getting a lot of positive feedback from patients and families with DMD for CAP-1002. And I'll just highlight very briefly the reasons why. Number one, and perhaps most importantly, CAP-1002 has a long and established safety profile. So the kids don't get sick from it. There's no safety sequelae. They really don't mind. It is a 45-minute infusion, once every quarter. Super easy for them. And remember, 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 PPMD webinar we did a few weeks ago that kids seem to be doing better on CAP-1002. That's supported by calls that I'm regularly getting from families either asking to get CAP-1002 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 will go along with gene therapy or whatever else is approved, and continue to support the health and well-being of patients with DMD.

And 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 of weeks ago and the groundswell that you've had, 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. I think it was great.

Now in terms of the meeting in Dallas that you're going to. Were you -- 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. So 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 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 the investment community as well.

One of the things that we 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 second, 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 CAP-1002. We'll provide an addition to the treatment paradigm by hopefully supporting the wellness of their hearts.

Okay. Well, I'm really happy with 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 0.5 million at $6.16 when the stock was running. Where did the other 0.5 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 pricing structures that are out there now, you could definitely increase your $6, $6.50 that you projected for the regimen to at least 20% to 25% higher? Have you looked at that yet?

Yes 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 of 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 10, 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, obviously the reimbursement is critical for us and our success down the line. We have a really nice structure with NS Pharma and Nippon Shinyaku in terms our -- what we will receive as a meaningful double-digit split of net product revenue. We have early estimates of what we feel that this drug will be priced at. I thought 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 US have very high price tags, and hopefully CAP-1002 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?

Yes, this is something we haven't gotten into and disclosed publicly. We have real [fern], a real [fin], that's all we 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.

Yes.

At this time, I would like to turn the floor back over to Capricor's management for final thoughts.

Yes, thank you operator, and thank you to everyone who 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.