Capricor Therapeutics Inc Q3 2024 Earnings Call Transcript

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

Question: Ted Tenthoff - Piper Sandler - Analyst : Great. Thank you so much. And I really exciting to be on the call and congrats on the progress. Can you hear me okay?

Question: Ted Tenthoff - Piper Sandler - Analyst : Hello, are you there?

Question: Ted Tenthoff - Piper Sandler - Analyst : Great. Thank you very much. And congrats on all the progress. So I wanted to get a sense. Obviously, there's a lot to do behind the scenes here with the filings and with the regulatory approval that you're seeking with the FDA. What are you and Nippon Shinyaku doing to prepare this market and to get ready for launch? And I guess coupled in that question is: you mentioned some of the work that you're doing on the manufacturing front. How can you also sort of prepare to scale up to meet that potential commercial demand? Thanks. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 9:30PM, CAPR.OQ - Q3 2024 Capricor Therapeutics Inc Earnings Call

Question: Ted Tenthoff - Piper Sandler - Analyst : Yes. Awesome. Really exciting time. Thanks so much for taking my question.

Question: Leland Gershell - Oppenheimer - Analyst : Hey, great to see all the progress and few questions from us. Just teeing off from Ted's question about extending manufacturing, just wanted to drill in a bit further, particularly given the potential European option for commercialization, you could have many more patients who you could serve up there with deramiocel. So as we think about your capabilities for serving the market with your current manufacturing versus what you might be able to do as we build out, how should we think about that as it runs along in parallel to what could be Japan as well as European approvals in the not-too distant future?

Question: Leland Gershell - Oppenheimer - Analyst : Great. And once you have the cardio cardiomyopathy label in hand, and you're pursuing a label expansion with skeletal muscle improvement in the upper limb, I'm just wondering since these Children who were affected by DMD tend to get cardiomyopathy by the age of 10 or not that much beyond, you, Linda, have expected that 50% to 60% may be addressable by the product, what would be the incremental gain, I guess, to commercial size that you would get from having an expanded label? Or is that somewhat academic since presumably the cardiomyopathy label would be sort of the large factor here?

Question: Leland Gershell - Oppenheimer - Analyst : Great. And then lastly just a quick one off your second half '25 guidance for the approval, that captures what could be a priority review or standard review, but presumably given your RMAT designation, do you generally expect to have further review? Did you request it? Did you need to request it, or as you submit the BLA?

Question: Leland Gershell - Oppenheimer - Analyst : Great. Thanks so much for taking my question.

Question: Rick Miller - Cantor Fitzgerald - Analyst : Hello, team. This is Rick Miller on for Kristen. Thanks for taking our questions and maybe just first thinking about the world muscle data you presented what constitutes a clinically meaningful benefit on cardiac function like left ventricular ejection fraction, specifically in the more severe patients with a baseline LVF -- LVEF, excuse me, under 45%? And how does this relate to what you saw in this subset in that data set?

Question: Rick Miller - Cantor Fitzgerald - Analyst : Thanks. And maybe just a follow up from that. From a patient journey perspective, can you kind of help us to better understand how these patients are monitored for cardiac decline? Do they undergo cardiac MRI often? And what age are physicians starting to think about seriously monitoring cardiac function in DMD patients? And is this something that you think the field could do better if there is an approved therapy?

Question: Rick Miller - Cantor Fitzgerald - Analyst : That's great color. Thank you for sharing.

Question: Aydin Huseynov - Ladenburg - Analyst : Hi, good afternoon, Linda, AJ. Congratulations with the progress this quarter. I got a couple of questions that I want to ask. So first commercial question. So you still have several geographies that you haven't outlicensed: China, South America, rest of the world, Eastern Europe, Australia. So what happens if that -- if some patients from these regions would like to have a drug, so would you sell it directly or you would sell it through your partner?

Question: Aydin Huseynov - Ladenburg - Analyst : Okay. And another question I have is about potential label opportunities for deramiocel. So would you expect any limitations in terms of using it after gene therapies or exon skippers on the label or you would think that the FDA will give you open label and leave it to physicians, how they're going to use it?

Question: Aydin Huseynov - Ladenburg - Analyst : Would you expect adcoms based on your conversations and based on what you've seen with prior Duchenne drugs? Would you expect the advisory committee meeting?

Question: Aydin Huseynov - Ladenburg - Analyst : Got it. Very helpful. Another question I have is about HOPE-3 readout potential in the future. So just a sort of commercial question. Do you think the HOPE-3 readout a potential label change would add actual dollars to deramiocel sales given that, as you said, this 100% of DMD patients have cardiomyopathy, so everyone will be eligible. So do you think the readout would actually add dollar sales to potential future sales of deramiocel?

Question: Aydin Huseynov - Ladenburg - Analyst : Yeah, thank you. Very helpful. And the last one for me. So can we discuss your development plans for 2025? I know you're in a sort of different situation now. You've got the cash right now, $165 million or so. You'll fill your PRV, it's almost $150 million. Then you have a milestone payment from Nippon. So this does actually give you opportunity to develop something on your own. And I think we discussed in the past that you may have a trial in Becker muscular dystrophy. So could we talk a little bit about this, about potential trial design? How would you start? Would you design it as HOPE-2, et cetera, if you could elaborate a little bit on this?

Question: Aydin Huseynov - Ladenburg - Analyst : Okay. Thank you. Thanks. So much for taking questions and congrats on the progress. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 9:30PM, CAPR.OQ - Q3 2024 Capricor Therapeutics Inc Earnings Call