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spk02: This conference call is being recorded. I would now like to turn the conference call over to our host, Mr. A.J. Bergman, Cap Record's Chief Financial Officer. Please go ahead.
spk07: Thank you. Good afternoon, everyone. Before we start, I would like to state that we will be making certain forward-looking statements during today's call and presentation. These statements may include statements regarding, among other things, the efficacy, safety, and intended utilization of our product candidates. our future research and development plans, including our anticipated conduct and timing of 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, turn the call over to Linda Marban, CEO.
spk04: Thanks, AJ. Good afternoon and thank you for joining us today. I'm extremely pleased with the progress we are making towards the development of our lead asset, CAP 1002, for the treatment of Duchenne muscular dystrophy. As I begin the call today, I am delighted to inform you that we have reached our targeted enrollment goal for our HOPE III phase III pivotal study, which is designed to enroll approximately 58 patients across the United States. Additionally, we are planning on announcing the outcome of our interim analysis from the study prior to the end of this year. The purpose of this important milestone will be to determine if the trial is futile or should continue as planned. The analysis is based on the six-month results. Reminder, our primary endpoint is based on one year. This blinded data will be presented to our DSMB which will evaluate safety and efficacy and give us their decision as to whether the trial should continue as planned. Should the trial be determined not to be futile, this will trigger our first milestone payment under our U.S. agreement with Nippon Shinyaaku. There are also other potential milestone payments leading up to an approval of a BLA that will continue to support our balance sheet should we achieve them. As I have previously announced, the top line results from cohort A will be available in Q4 2024, and a subsequent BLA would be based on the full data set. However, we are now planning to reinvigorate our discussion with FDA to determine if there is an opportunity to speed up the approval pathway for CAP10A2 now that our enrollment target for HOPE3 cohort A has been reached. We owe it to the patients and the community to move CAP 1002 towards approval as quickly as possible, as everyone knows that time is muscle. Once a pull point is lost, it cannot be recovered. Now, this brings me to an overview of our recent Type B clinical meeting with the FDA, which was announced in late Q3, and why we believe the outcome was important for the program. As you may recall, earlier in 2023, FDA indicated that they wanted us to treat additional patients in the HOPE III study with the product manufactured at our San Diego GMP facility. There were two main outcomes from this meeting. Number one, the results of the HOPE III cohort A will support the filing of a BLA, and number two, FDA approved the design of an additional cohort of patients, now referred to as cohort B, to support the inclusion of our new San Diego facility for commercial manufacturing and enrollment is planned to begin imminently. It is very important to keep in mind that Cohort B and the data that we generate from it will not be required for initial registration of CAP 1002 for DMG. Over the last several years, Capricor has carefully and diligently invested resources in this program, bringing CAP-1002 through three successful clinical trials, building two manufacturing facilities, securing RMAT, orphan drugs, and rare pediatric designations from FDA, in addition to securing commercialization partnerships for the U.S. and Japan markets. These strategic deals were carefully negotiated and potentially bringing over $700 million in additional cash to Capricor, which would fuel future product potential expansion of PAP-10-02, and allow us to strategically invest in the further development of our exosome pipeline. This is the vision that I have for Capricor, which would transform our organization from a development stage therapeutic company into a world-class commercial and R&D operation. I realize much has to be done and accomplished before that time, but now that we have completed our target enrollment in the phase three, we are moving closer to that vision. To this point in our history, we have remained disciplined across the organization, not only in the management of cash and resources, but also with respect to our priorities as we have developed Cap10 or 2. We made the early decision to invest in the clinical development of Cap10 or 2 while managing our manufacturing operations in a just-in-time manner. Now that we have shown promising data in multiple clinical trials, we are focusing on scaling up and out our manufacturing capabilities to produce larger quantities of CAB 1002 to meet FDA requirements and market demand, if approved. Our GMP manufacturing facility in San Diego cost approximately $3 million to build and equip. As is the case with all companies, launching a complex biologic such as CAB 1002 requires us to expand operations to include experts in cell manufacturing, quality, compliance, and regulatory affairs. which is reflected in the current structure of the organization. Capricorn has raised approximately $145 million in equity capital in totality throughout our company's history, both private and public, over the past 15 years. And with approximately $15 million in cash resources factoring in our most recent financing and Q3 financials, this gives us a runway into 2025. The dollars invested in CAP 1002 for DMD have strengthened many aspects of our program, most notably the ability to understand the mechanism of action of CAP 1002, recognizing that one of the hindrances to the approval of cell therapies has been an inadequate potency assay. We are pleased that the FDA supports our potency program, which is an important step towards approval. With the path paved by DMD, we are now planning product expansion into diseases with similar pathophysiology. We believe in CAP1002's potential to be a transformational treatment for patients with diseases of inflammation and fibrosis. Second to that, we are judiciously building our exosome platform technology to become a next-generation drug delivery platform. Now with that said, let's quickly turn to an update on our exosome technology. While our primary focus has been on advancing the development of Cap Center 2. We remain committed to our exosome technology as part of next generation drug delivery platform. Currently, we are pursuing two avenues of opportunity. One is our vaccine program using StealthX, our proprietary platform that is useful for engineering select proteins either inside or on the surface of the exosome. For our vaccine program, one of our aims is to secure either a partnership or a non-dilutive source of funding to speed clinical development. Currently, there is significant interest in an exosome-based vaccine platform, and we are in active discussions with several parties. We remain focused on that mission. The other aspect of our platform uses StealthX to develop therapeutics by harnessing exosomes as the delivery vehicles. The program requires loading of specific cargoes into exosomes with targeting moieties on the outside of the exosome, essentially to tell it where to go. Currently, in collaboration with an undisclosed pharma company, we are investigating the therapeutic perspective of this platform. The foundational work has the potential to set the stage for future therapeutic options with exosomes. Initial data from this ongoing study was presented recently, at this year's World Muscle Society Conference. Proof of concept results demonstrate that a muscle targeting moiety can be engineered on the surface of exosomes as demonstrated by the presence of a labeled ASO, which is antisense oligonucleotide, and used as representative cargo in the lower limbs of mice post intravenous injection. Repeated doses of the loaded exosome show enhanced targeting based on the initial PK study. This suggests that repeat dosing using our engineered exosomes has the potential to effectively enhance delivery, further differentiating our therapeutic approach from lipid nanoparticles. I look forward to providing more color on this important program as it becomes available. Now, finally, on the corporate side, we raised approximately $23 million this quarter to support our balance sheet into 2025. This strategic financing was anchored by Nippon Shinyaku, further cementing our strong relationship and their commitment to Capricorn. Part of the reason for the fundraise was we had to expand our team in order to prepare for the submission of a BLA. The work required to prepare a late-stage clinical asset is significant, and we are fortunate to have found a team that is experienced in driving programs through approval in the biologic space. Finally, in our effort to expand Capricorn's team of experts, we are pleased to announce the appointment of Michael Kelleher to our Board of Directors. Most recently, Michael served as Group Vice President of M&A and Business Development at Horizon Therapeutics, now part of Amgen. He brings to Capricorn expertise in business and strategic development. We look forward to Mike's contributions to our team. Overall, I want to thank you for your support for our program and our company. We are very much looking forward to the next several months as we will be announcing the outcome from the interim analysis, as well as continuing our interactions with FDA. I will now turn the call over to AJ to go through our financials. Thank you.
spk07: Thanks, Linda. This afternoon's press release provided a summary of our third quarter 2023 financials on a GAAP basis. And you may also refer to our quarterly report on Form 10-Q, which we expect to become available shortly, and will be accessible on the SEC website as well as the financial section of our website. Turning to the financials, let me start with our cash position. We ended September 30, 2023 with cash, cash equivalents, and marketable securities of approximately $28.5 million. This excludes the $23 million in gross proceeds from the registered direct offering we completed in October that bolstered our cash position. Based on our recent operating results and current projections, we now expect our cash runway to extend into 2025, an extension from our prior guidance. In the third quarter of 2023, our commercialization revenue was approximately 6.2 million. That compares to approximately 1.6 million for the third quarter of 2022. Turning quickly to the expenses, excluding stock-based compensation, our research and development expenses were approximately $9.5 million for the third quarter of 2023, compared to approximately $5.4 million for the third quarter of 2022. The increase in expenses of $4.1 million was primarily due to increased clinical and manufacturing costs associated with our Phase III HOPE III trial. Excluding stock-based compensation, our general and administrative expenses were approximately $1.8 million for the third quarter of 2023, as compared to approximately $1.6 million for the third quarter of 2022. The increase of $200,000 was primarily due to increased facility and personnel costs. And our net loss was approximately $6.4 million for the third quarter of 2023 and 2022. And with that, let me turn it back over to Linda. Actually, open up the line for questions first before we do that. Go ahead, operator.
spk02: Thank you. Ladies and gentlemen, we'll now begin the question and answer session. Should you have a question, please press the star followed by the one on your touchtone phone. You will then hear a three-tone prompt acknowledging your request. Questions will be taken in the order that they are received, and should you wish to To withdraw your request, please press the star followed by the two. If you are using a speakerphone, please lift the handset before pressing any keys. One moment, please, for your first question. And your first question comes from Joe Pantgini from HC Wainwright. Please go ahead.
spk01: Hey, Linda and AJ. Thanks for taking the question. Good afternoon. My first question, I'm going to start at the back end of your comments regarding exosomes. So just wanted to get a sense of what potential news flow in preclinical data we might be getting over the next six to 12 months. And secondly, I know you said there's ongoing discussions right now, so there might be some confidentiality, but can you disclose what kind of vaccines you might be going after?
spk04: Yeah, so thanks, Joe. Thanks for your questions. And we are excited about the Exosome program. You know, it's been perking along in the background. We have devoted most of our time and effort to CAP 1002, as everybody knows. But the data from the exosome has been very positive and very exciting, including the ability to target, which has not really been shown before and was presented recently, as I mentioned, at World Muscle. So in terms of milestones coming up in 2024, we do plan on continuing to advance this program. We've had a vaccine for COVID that's been in development for quite some time. I think the world has come to realize that there's a lot to be left to understand about the vaccinology using mRNA. And so, the concept of using a protein-based vaccine that is easy to make, easy to manufacture, can be manufactured in just several months of time using native proteins and using a non-toxic molecule such as the exosome. is indeed something to be of great interest and certainly gives proof of concept for the program. So that's the type of vaccine we're working on. Same one, just looking for other opportunities for partnering for the therapeutic options as well as for the vaccine. The vaccine conversations are the farthest along at this point.
spk01: Got it. That's helpful. Thank you. And my main question has to do with the DMV program, obviously. I guess there's a few working parts here, but look, following your, you know, Type B meeting, you had very, very important visibility about the path forward to be able to file in Cohort A and Cohort B. So I guess my question is, you know, maybe a little bit of a scenario analysis of your comment about still further potentially being able to accelerate the programs. And I guess I would also focus my question around the upcoming interim analysis, would your disclosure to the public revolve around sort of continuous planned or any potential data and any potential stopping rules you might be able to share with us? Thanks.
spk04: Yeah, thanks. So let me just start with the interim analysis since it's the most recent upcoming milestone, which would be before the end of the year. It is a futility analysis, so the DSMB gets the data, evaluates safety and efficacy, and determines based on preset metrics, which are not been disclosed, whether or not the trial should continue as planned. We are looking forward to the results of that interim analysis. We, of course, based on all of our previous clinical work, expect the trial to be not futile and to be continuous plan, and that's how we're building our story. But, of course, it will be a great day for celebration once we get that word from the DSMB that the trial is not futile and we can continue. In terms of the actual metrics, we have not disclosed those, but we can say that it is a standard futility analysis that gives good confidence that, you know, the trial should proceed as planned. And that's where we are with that. In terms of the FDA, you know, it's funny as you were asking your question, I was sort of thinking about how to respond, and I guess it's not unfair to say that dealing with FDA these days, not for Capricorn, but for everybody, is sort of like dealing with the wild, wild west, right? We never really know what's coming, what the opportunities are, what they are going to see as appropriate for making something available for approval or registration versus what they're going to hold back on. What I can tell you and what FDA knows is we have three positive clinical trials all showing improvement in the performance of the upper limb in young men with later stage Duchenne muscular dystrophy. We have families that are clamoring to receive CAT-10 or two because anecdotally they say their sons are feeling and functioning better. We have a fully enrolled phase three trial which FDA has been absolutely strong in the fact that they want to see the fully enrolled confirmatory trial before they would ever consider accelerated approval. So we consider that we check that box now. And we also have, you know, the ear of FDA with our RMED designation and also with the families from the community really speaking up to FDA. So, you know, the conversation gets much more involved as you get to this point in development. It's been a long time coming for us, so I am delighted. There's, you know, many opportunities and touch points. And so, at each of those touch points with FDA, we will continue to broach the idea that there may be a path to approval. But really, at this point, with a fully enrolled cohort A and top line data in just one year, we feel that we are poised for every level of success should the trial be positive.
spk01: Appreciate the color. Thanks, Linda.
spk03: Thanks, Joe.
spk02: Thank you. Ladies and gentlemen, as a reminder, should you have a question, please press the star followed by the one. And your next question comes from Aiden Huzinov from Leidenberg. Please go ahead.
spk05: Good afternoon, Linda, AJ. Congratulations with the progress this quarter and congratulations with completing the enrollment in cohort A. I have a couple of questions. First, I want to start from milestone, potential milestone payment from Ipon Shinyaku. So your total potential sort of receivable from NIPON for the next several years if the drug gets approved is $705 million. So can you give us a sense how much from that total amount NIPON may pay by the end of the year if the DSMB decision will be positive? Is it $10, $20, $30 million? Can you give us overall just a sense of it?
spk07: Yeah, thanks. Thanks, Aiden. Right now, we're not at liberty to state the exact dollar figure affixed to that interim utility analysis. Of course, as it hopefully is achieved, we will put more granular details out. So stay tuned for that. And so that's kind of where we're at now. In terms of the building milestones as we move forward towards approval, What we haven't disclosed up to this point, but we're now at liberty to say, is that up to and including the time of approval is $100 million in potential milestones to Capricor. So we're going to look to that to fuel, obviously, the expansion of Cap 1002 into Duchenne, as well as other efforts in terms of product expansion. So that will those milestones hopefully come due will total 100 million. Hopefully that's helpful.
spk05: Oh, yeah, this is super helpful. Appreciate that. Just to cover, it's 100 million including approval milestones. So imagine that you get approval. Okay.
spk07: Yeah, exactly. And look for our 10Q to become available shortly, which will articulate this basically the same way I'm saying it, but that should be put out there shortly.
spk05: Okay. I appreciate that. All right. Another question is on cohorts. So cohort A completed enrollment 58 patients. It seems like you're going to be starting cohort B enrollment within weeks from now. I think you mentioned by the end of the year. So given the holidays, do you think it is still realistic to sort of enroll, actually enroll patients in cohort B by the end of the year between now and January 1st?
spk04: So Aidan, were you on my team call yesterday? Because this is what I asked my clinical team. And they said that enrollment is going to be pretty robust before the end of the year. You know, there's nothing else in clinical trial right now for these later stage boys and young men with DMD, nothing with recent failures in the space and some of the other companies pulling back. And so there's a line out the door to get into cohort B, and we'll be delighted to welcome them and treat them even with the holidays coming up.
spk06: Oh, yeah, this is great to hear.
spk05: I mean, so given there's nothing, no other trials, it is feasible that you will enroll. Okay, understood. So, given the DMV landscape is changing, obviously, with different treatments, different approvals, so do you think the cohort B will be somehow different from cohort A, given that these are, this will be, different patients, so do you think any variability between two cohorts in the future?
spk04: Yeah, it's a really interesting and provocative question, again, one we've debated a lot internally, and I think the short answer is no. You know, we have the same inclusion-exclusion criteria, the same requirements in terms of their, you know, meds and all of that. The only potential difference is with the approval of the new De Slaza Court. We're also open up to taking patients on that regimen because many of them are switching due to a better safety profile. So that's really the only difference. We don't think that's going to mediate any difference in sort of the potential efficacy. We've talked at length to our KOLs and sort of the steroid is a steroid is a steroid in terms of what it does physiologically. So we're very hopeful that the enrollment criteria will be virtually the same and And cohort B will very rapidly allow us to transition from, you know, sort of clinical scale manufacturing to commercial scale manufacturing.
spk05: Appreciate that. This is very helpful. So another question I have is about expansion of potential label of CAP-1002. So other DMD companies may have certain limitations to expand into other stages of DMD or other dystrophies or other types of DMD patients. So we have a nice slide on your corporate presentation describing potential expansion into early-stage DMD patients and then into Becker muscular dystrophy or BMD. So could you help us understand how the potential endpoints with early-stage patients may look like with CAP-1002? So this likely won't be pooled because these patients are more active and more mobile. So just Give us a little bit of idea how these endpoints may look like.
spk04: Yeah, so, you know, it's interesting. All of these patient-reported outcome measures have their strengths and have their weaknesses. I think everybody really believed very strongly in the NSAA, but very recently with the Sarepta data, there's been a little question around the veracity of that measure. There's time to rise, which is an interesting one. We haven't designed that trial yet. Frankly, we're going to talk to FDA about label expansion potentially without more clinical work and then, you know, evaluate from there and use the advice of our key opinion leaders who are used to working with these younger boys to help find the one that's most effective in demonstrating the efficacy of CAP-10-02 in these younger kids.
spk05: Understood. Understood. Thank you, Linda. And the last question is in regarding Becker dystrophy. So for Becker dystrophy, cardiomyopathy is obviously one of the major causes of death. And it would make sense sort of starting from the late stage patient. So could you share with us how or why Capricorn is more advantageous, is in more advantageous position compared to other dystrophy companies when it comes to Becker dystrophy?
spk04: In terms of Becker dystrophy, you know, one of the major manifestations of Becker muscular dystrophy are the cardiac implications. And, of course, as you know, one of our major advantages over almost any other therapeutic that's in investigation or approved for DMD is that we have cardiac benefits. So we are highly enthusiastic, if you can sort of use that terminology when you're talking about treating a dread disease, about the opportunity for CAT10O2 in Becker muscular dystrophy and its cardiac implications.
spk06: Understood. Thanks so much, Linda and AJ. Congratulations again with the progress. Thank you so much.
spk02: Thank you. Ladies and gentlemen, just as another reminder, if you do have a question, please press the star followed by the one. There are no further questions at this time. I will be turning the call back over to Capricorn's management for final remarks. Please go ahead.
spk04: Thank you so much. Thank you so much, and thank you, AJ. Before we conclude today's call, I want to extend my sincere appreciation to the clinicians, patients, and their families who are working with us to bring Cap Center 2 closer to potential approval. I also just want to take a minute to thank our clinical operations team for doing an extraordinary job for getting this trial fully enrolled. Hopefully, once we continue to follow these patients and get the data, we'll be able to have an approved therapeutic for these later stage Duchenne patients. And I know that will be a day of celebration for the community as well as for us. We look forward to seeing you at upcoming meetings, and hopefully you'll have a happy and healthy holiday season. Thank you so much.
spk03: Ladies and gentlemen, this concludes your conference call for today.
spk02: We thank you very much for participating and ask that you please disconnect your lines. Have a great day.
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