Capricor Therapeutics Inc To Discuss DMD Program Update Call Transcript

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

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Thank you, and good morning, Linda and AJ. Congratulations on this terrific regulatory update and for walking us through the comparative data that showed what appears to be unprecedented therapeutic efficacy in this setting. A few questions from us here. Clearly treating earlier is better and as essentially all condition, patients develop cardiomyopathy as they age when would be the right time to come for deramiocel to start, will there be any sort of age information on the label or that you believe treating physicians will begin the therapy?

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Thanks. And following the last week's update with the partner in terms of the license to the EU geography, wanted to ask if you can share any thoughts on likelihood of EU approval on the same data using a similar strategy for the EMA?

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Thanks. And then in terms of manufacturing, I wanted to see if you could share kind of your current capacity, is you expect it to be available in the second half of next year, presuming approval and how that may change or increase maybe in subsequent years to serve the market more broadly?

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : And then finally, with HOPE-3 now to be used as a label expansion strategy, any view on when you may unblind the HOPE-3 cohorts at this point?

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Thanks for taking our questions. And again, congratulations.

Question: Joe Pantginis - H.C. Wainwright & Co - Analyst : Everybody. Good morning. This is great news and I want to congratulate you after this some very thoughtful path that you've been taking. So first, I wanted to -- I heard you say, Linda, a couple of times like I wanted to get a sense of how the role of cardiomyopathy has evolved with your discussions with the FDA? It appeared or I might have heard that it might have been somewhat more recent when you show them these data. This is something you and I and on the conference calls I've been talking about for quite some time. So I wanted to get a sense about the evolution of how important cardiomyopathy has become in your discussions till today?

Question: Joe Pantginis - H.C. Wainwright & Co - Analyst : No, thanks for that. And then my next question is short, but wanted to get your thoughts on as you're filing your BLA now, would you expect there to be an advisory committee?

Question: Joe Pantginis - H.C. Wainwright & Co - Analyst : Got it. And then I want to see if I heard you correctly, as we're looking at the population breakdown, this really doesn't appear to be a limited subpopulation on cardiomyopathy, especially since you said, it's the leading cause of mortality. So I mean, it really does apply to a broad population, if I heard you correctly.

Question: Joe Pantginis - H.C. Wainwright & Co - Analyst : No, great. And my last question is just taking a little bit of a turn very nice to see the ex US partnership with Nippon. Obviously, they seem to be the right one since you already have a great working relationship with them. Just curious if this was a competitive process.

Question: Joe Pantginis - H.C. Wainwright & Co - Analyst : Great. Appreciate all the comments, and this is great news. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 24, 2024 / 12:30PM, CAPR.OQ - Capricor Therapeutics Inc To Discuss DMD Program Update Call

Question: Kristen Kluska - Cantor Fitzgerald & Co - Analyst : Hi, good morning and congratulations, everyone, on these updates and appreciate all your commitment to helping these boys. Wanted to first drill down a little bit more in terms of the cardiomyopathy. So I understand in most patients, they develop this at some point or another. But looking at the addressable market today, what percent, roughly would you say present with this? I think most of us were modeling just the non-ambulatory patients about half of the patients being eligible. And then at what age do these patients typically see a cardiologist and in terms of building a sales force with your partners. Do you think this is something that their cardiologist excuse me -- are going to consider some treating with more than just other physicians they see in their practice. Thank you.

Question: Kristen Kluska - Cantor Fitzgerald & Co - Analyst : Appreciate that. And then based on the timelines you gave to us, it sounds like the approval decision is going to come before the HOPE-3 for data reads out next year. So has the FDA given any sense about what they would want to see from that full readout to ensure that this drug stays on the market and that confirmatory study. And obviously, the I understand that study's primary endpoint is on pole 2.0 to support the label expansion. But will there also be an increased emphasis on the cardiac endpoints, given that's what's supporting the approval process today?

Question: Kristen Kluska - Cantor Fitzgerald & Co - Analyst : Thank you. Appreciate that. And last question from me. I understand why you looked at the left ventricular ejection fraction above 45%, 55%, et cetera, in the trial. But did you happen to see any patients where it was even lower? I understand at that point, the literature suggests it could be really difficult to improve at that point, but like anything to assess there in terms of even just stabilization versus a further decline with what we'd expect from natural history. Thank you so much again.

Question: Kristen Kluska - Cantor Fitzgerald & Co - Analyst : Okay. Congrats again and all the best to you.

Question: Aydin Huseynov - Ladenburg Thalmann & Co. Inc - Analyst : Good morning, everyone. Linda, AG. Congrats with the decision to file BLA based on cardiomyopathy. And so given that Deramiocel cardiac drug in the first place. I'm curious what today's decision mean with the other for other cardiomyopathies end. Could you mention any other relevant indications where there milestone could be potentially helpful. And maybe a broader question is does the patient -- does the cardiomyopathy has to be associated with DMD in order to be treated at deramiocel?

Question: Aydin Huseynov - Ladenburg Thalmann & Co. Inc - Analyst : Thank you. And this is very helpful. On another question I have is about the potential drug-drug interaction if it's used in parallel with exon skippers also, what are your thoughts on using deramiocel after AAV gene therapy? And do you think this is sort of post gene therapy market. Is that expanding or is it shrinking in that regard? So in other words, do you see the DMD market resembling sort of SMA market after ZOLGENSMA was first approved?

Question: Aydin Huseynov - Ladenburg Thalmann & Co. Inc - Analyst : Yeah, makes sense. Makes sense. And the last question I have is about the potential pricing for deramiocel, given how close we are to the approval and whether you think there will be any payer sort of resistance of reimbursing to potential expensive DMD therapies at the same time, if they are on exon skippers or some other therapies?

Question: Aydin Huseynov - Ladenburg Thalmann & Co. Inc - Analyst : Okay. Thank you. Thanks so much. Very helpful, and congrats again, but the decision today.