Capricor Therapeutics Inc Q4 2024 Earnings Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Edward Tenthoff - Piper Sandler Companies - Analyst : Great. Thank you very much and congratulations on all of the progress. It's going to be an exciting year for you guys. Just looking at the commercial preparation for deramiocel, what additional color can you tell us about the prep and the division of labor between you and Nippon Shinyaku? And how much of a benefit will it be with their existing commercial infrastructure?

Question: Edward Tenthoff - Piper Sandler Companies - Analyst : Great. Thank you very much.

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Hi. Good afternoon. Great to hear all the progress and we're looking forward to events coming later this year for Capricor. Just a few questions from us. First, Linda, I want to ask with respect to your discussions with payers so far. I'm wondering, if as part of those discussions, there was any contemplation of -- for patients who may be on other premium-priced drugs for DMD and then may be also going on deramiocel or even vice versa. Just wondering, given the expense burden of having two premium-priced drugs for this condition, how are the payers viewing that, if you have any intelligence there?

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : All right. Thank you. And then a question just longer term, you've got a healthy cash balance. You're going to have the $80 million, presumably on the FDA approval and then that -- and the PRV, which you could monetize, and that doesn't even include other terms with NS, with Japan and potentially Europe. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 19, 2025 / 8:30PM, CAPR.OQ - Q4 2024 Capricor Therapeutics Inc Earnings Call Just curious, given that the operating expense for Capricor will remain relatively lean given that you wouldn't have to be building your own sales force and getting into commercial infrastructure yourselves. Any thoughts on how you may invest that capital? Obviously, exosomes are very promising. But given the stage of that development, it doesn't really seem like it would be expensive to be running those initial studies. Just wondering, if you have any thoughts on where to apply the much greater balance sheet that you may be coming into. Thanks.

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Alright. Thanks for the color.

Question: Joseph Pantginis - H.C. Wainwright & Co LLC - Analyst : Hey, Linda and AJ. Thanks for taking the questions. So first, maybe for AJ, I mean, this is fantastic news also that Linda, you just announced about the new expanded 25,000-square foot facility. Are you able to define the cost and time frame for this facility?

Question: Joseph Pantginis - H.C. Wainwright & Co LLC - Analyst : Got it. And then I guess second question is, obviously, you have no indication that there's going to be an ADCOM right now. When do you think you might hear an answer? And for example, if there were an ADCOM, would that be a place that they might want to not necessarily require but maybe force a discussion regarding HOPE-3?

Question: Joseph Pantginis - H.C. Wainwright & Co LLC - Analyst : Also very helpful, thanks for the repetition there. Quickly, just on the discussion about cash usage going forward. Obviously, you talked about some of the obvious things. Since you guys really have a specialty in, say, cellular therapy here, would you consider in-licensing, number one? And then second, with regard to Europe, I know you can't really -- or can't necessarily describe the first part of the question, but what are the outstanding things that you're still needing or the open questions with regard to potential signed NS partnership? And then also, what are the outstanding questions you feel are important for your EMEA discussions?

Question: Joseph Pantginis - H.C. Wainwright & Co LLC - Analyst : Really appreciate all the color. Thanks a lot.

Question: Kristen Kluska - Cantor Fitzgerald & Co - Analyst : Hi, everyone. Congrats on the data this week and the recent filing acceptance. So I know that your mechanism is complementary to a gene therapy, but after yesterday's unfortunate news regarding the patient on Elevidys, we are hearing doctors emphasize the need for treatment options for non-ambulatory patients in particular. So can you just remind us what percent of the later-stage population in particular, would have cardiomyopathy? And then while label expansion could come later, could you reference some of your pool data if you have an approval as perhaps supportive evidence? Thank you.

Question: Kristen Kluska - Cantor Fitzgerald & Co - Analyst : Thank you for that. And then another question. I know you have robust safety data as well. But as we start to think about the potential to move to commercialization where there will be the potential for more to get treatment at once, is there anything we should consider from a safety protocol? Will physicians want to treat a few patients first? Or will doctors require a more extensive testing? And the reason why I ask is sometimes like we are seeing with the gene therapy that there's a plethora of different specialists that need to be involved because of some of the known safety risks of that.

Question: Kristen Kluska - Cantor Fitzgerald & Co - Analyst : Thanks. And last question for me. I think you commented that with the additional manufacturing footprint, this could support 2,000 to 3,000 patients potentially as early as mid-2026. But should you get an approval later this year, how should we be thinking about what you can essentially handle until that time? Because I know you said there's already some patients that want to transfer on OLE as well as others that weren't involved in the trial that are aware of the potential of the drug. Thanks, again, everyone.

Question: Kristen Kluska - Cantor Fitzgerald & Co - Analyst : Thank you so much, again. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 19, 2025 / 8:30PM, CAPR.OQ - Q4 2024 Capricor Therapeutics Inc Earnings Call

Question: Catherine Novack - Jones Trading - Analyst : Hi. Good afternoon, everyone. Thanks for taking my question. I'm just wondering if you can talk a little bit about -- in the past, FDA had made comments about the issues of single-arm studies in DMD due to the disease heterogeneity. Can you just give us some color about in your discussions with FDA, what have they brought up regarding their view on cardiac outcomes shown in HOPE-2 OLE, why this might be -- why they might take a different approach on this?

Question: Catherine Novack - Jones Trading - Analyst : Correct.

Question: Catherine Novack - Jones Trading - Analyst : Got it. Thanks. Thanks so much. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 19, 2025 / 8:30PM, CAPR.OQ - Q4 2024 Capricor Therapeutics Inc Earnings Call

Question: Aydin Huseynov - Ladenburg Thalmann & Co Inc - Analyst : Hi. Good afternoon, everyone. Congratulations for the progress this quarter. So a couple of questions from us. So Linda, I think you mentioned heart diseases where deramiocel might be helpful. And obviously, the next indication, the next low-hanging fruit is Becker Muscular Dystrophy. It's in your corporate presentation. So when do you think we will have updates on the potential Becker Muscular Dystrophy trial design and whether it is going to look like HOPE-2? So could you give us a little bit of insights in terms of what is your clinical strategy as it comes to Becker Muscular Dystrophy?

Question: Aydin Huseynov - Ladenburg Thalmann & Co Inc - Analyst : Okay. Understood. Thank you. Thank you for the update. And what are other heart diseases where you think deramiocel can make a meaningful difference? I'm just trying to understand the whole commercial potential for deramiocel beyond DMD and beyond BMD.

Question: Aydin Huseynov - Ladenburg Thalmann & Co Inc - Analyst : Okay. Thank you. Thanks so much. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 19, 2025 / 8:30PM, CAPR.OQ - Q4 2024 Capricor Therapeutics Inc Earnings Call

Question: Madison El-Saadi - B. Riley Securities - Analyst : Hey, Linda and AJ. Thank you for taking our question and congrats on the progress. I'm coming away from MDA, so my question may be a bit academic, so forgive me. So based on what you've seen through the course of deramiocel's development, what do you think is the ideal baseline ejection fraction baseline where impact will be the highest? Is it in the, for example, less than 35% range? Or is it more the 35% to 45% range? And then secondly, I believe you mentioned that you would expect 7,500 boys to be eligible. Would this be based on a hard ejection fraction threshold? And I ask given that cardiac treatment in this population is often the result of shared decision-making. Thanks.

Question: Madison El-Saadi - B. Riley Securities - Analyst : Understood. Thanks.