Good afternoon, and welcome to Capricor 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. [Operator Instructions] As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Mr. AJ Bergmann, Capricor's Chief Financial Officer. Mr. Bergmann, 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 have 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 reestimation 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…

Thank you, Linda. This afternoon's press release provided a summary of our fourth quarter and full year 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 be accessible at the SEC website, as well as the financial section of our company website. As of December 31, 2022, company’s cash, 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 2022, 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 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 the line for questions.

Thank you. We will now be conducting a question-and-answer session. [Operator Instructions] Our first question comes from Joe Pantginis with H.C. Wainwright. Please go ahead.

Hey, Linda and AJ. Good afternoon. Thanks for taking a 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 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 and 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 and on 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 and the CMC. And as you know, this really paves 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 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 [indiscernible] 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 -- 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. Thanks. 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 [Kaducia] (ph), 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 of 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 road map 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?

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 promising zone. And so, we don't have a futility built in it 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 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.

Next question comes from Michael Okunewitch with Maxim Group. Please go ahead.

Hi, 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 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 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 Shinyaku, since they're already in the DMD space with the exon skipping drug, does their partnering in Japan give further confidence for the strategy to position CAP-1002 as a back bone therapy in DMD?

Yes, absolutely. And we've been very strategic in selecting Nippon Shinyaku and 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 on 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] (ph) 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's still in late stage development and are 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 empowered 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-3 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 Aydin Huseynov with Ladenburg. Please go ahead.

Hi, Linda. Hi, AJ. Congratulations for the quarter and with the Nippon Shinyaku deal. 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 and DMD and other orphan diseases as well. What we are doing is positioning CAP-1002 worldwide as an anchor backbone therapy. We think that CAP-1002 will serve and great conjunct 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 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-3 trial?

Yes. 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 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 I didn't decide to make a deal now, but not after you hear back from HOPE-3 at the end of third and fourth quarter?

Yes. So we really had a long time ago identified the fact that Nippon Shinyaku 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, 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 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, 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.

So will we get from them while stones from the Japanese deal for the HOPE-3 readout as well on top of the U.S. deal milestones?

No, so the U.S. deal is constructed around milestones based on HOPE-3 and then the Japanese deal is based on milestones constructed around things that will -- or 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 dystrophy or would you invest in vaccine business? So how do you see the capital allocation going forward given that you collect all this 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 it's going exactly as we had planned, which is, we have 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 worked 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 as novel, it's innovative, it takes vaccinology to a new level, with combining the ease of an mRNA vaccine with the power or [indiscernible] protein vaccine and then this can also be used for protein replacement therapy. 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.

[Operator Instructions] Next question comes from Brian Corday with BullBear. Go ahead.

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

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.

Yes. So usually there's not -- this is not [indiscernible] call 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 is 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. It's 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. And 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 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 of weeks ago and the ground swell 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. Yes, 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 -- 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 sec, we have a cohort of kids even with gene therapy that seem to be doing better. They're staying on their feet longer and they're more active, but their hearts are worse. And this is unequivocal. So there's more and more focus on CAP-1002 will provide 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 had 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 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 [$6.50] (ph) 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 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 in 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 of 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. 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 they 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 a [indiscernible] 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. Alright. Well, I appreciate it. Thank you so much and keep up the great work.

Thanks, Brian. See you soon.

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 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 lines at this time. Thank you for your participation and have a great day.