Capricor Therapeutics Inc DMD Program Update Call Transcript

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

Question: Joseph Pantginis, Ph.D. - H C Wainwright & Co LLC (Pre-Merger) - Analyst : Thanks for taking the questions, and especially for the updated visibility. I know everyone appreciates it across the Street and the medical community. A couple of questions, if you don't mind. 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 25, 2025 / 12:30PM, CAPR.OQ - Capricor Therapeutics Inc DMD Program Update Call I know you talked about the PDUFA but I think not necessarily the date but one would consider this putting HOPE-3 data in there a major amendment or do you feel that, as you said you'd hope to get the data on a timely basis, that might impact whether it's a major amendment or not?

Question: Joseph Pantginis, Ph.D. - H C Wainwright & Co LLC (Pre-Merger) - Analyst : No. That totally makes sense. And I guess also, the question here, and this has been the open question, it's good to have the visibility of keeping the primary endpoint of pull. But were there any real changes discussed with regard to the statistical analysis plan of how cardiomyopathy might play in? And then, when you talk about two potential labeling opportunities, of course, it would be great to be a fly on the wall during your meetings. Is it -- do these labeling opportunities still -- are they still only gated on pull before you can -- if that fails, for example, which I don't believe it will, negate the potential for cardiomyopathy? Just want to make sure we're going down the right roads here.

Question: Joseph Pantginis, Ph.D. - H C Wainwright & Co LLC (Pre-Merger) - Analyst : No. I appreciate that very much. And don't worry about your answer because my questions were long-winded as well. But it's nice to hear the positive tenor of the discussions.

Question: Madison El-Saadi - B. Riley Securities Holdings Inc - Analyst : Thanks for providing the update today. Can you remind us, is there a correlation between pulled and ejection fraction? And then to follow up on a prior comment, given that 70% of patients do have cardiomyopathy, does this tell us anything about what we could expect? And how does that align with the HOPE-2 data set? 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 25, 2025 / 12:30PM, CAPR.OQ - Capricor Therapeutics Inc DMD Program Update Call

Question: Madison El-Saadi - B. Riley Securities Holdings Inc - Analyst : You mentioned that 70% have cardiomyopathy. I was just curious how that aligned with the Phase 2? And then the second part, I'll go ahead and squeeze that second part. Will you have ejection fraction or pull measurements beyond 12 months? I know every patient has gone 12 months but just curious if you will have some data sets at 18 months, et cetera.

Question: Edward Tenthoff - Piper Jaffray Inc - Analyst : Great. And a very exciting update. I know it's been a long and winding road here but I appreciate your persistence on behalf of the boys and investors. So thank you for the update. My question really has to do sort of twofold. Firstly, just with respect to Nippon Shinyaku, any update? What's the latest there? Obviously, that potential milestone for regulatory approval of, I think, $80 million could be pretty relevant. And then, also, when it comes to the potential for pediatric voucher, just remind me what the latest is there and how that could come into play? 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 25, 2025 / 12:30PM, CAPR.OQ - Capricor Therapeutics Inc DMD Program Update Call

Question: Aydin Huseynov - Ladenburg Thalmann & Co Inc - Analyst : Linda, AJ, thanks for providing this update. A couple of questions on our end. So do you think that there may be some sub-populations in the HOPE-3 trial, where more advanced patients may have more profound benefits from deramiocel, like, more advanced patients may have more cardiomyopathy? Do you think the FDA will be flexible enough to take a holistic approach and look at both HOPE-2 and HOPE-3 data and give you some more narrow label, if needed?

Question: Aydin Huseynov - Ladenburg Thalmann & Co Inc - Analyst : Makes sense. Makes sense. Another question I have, during your discussions with the FDA, was there any other additional cardiac endpoint beyond LVEF that the FDA was particularly interested in? 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 25, 2025 / 12:30PM, CAPR.OQ - Capricor Therapeutics Inc DMD Program Update Call

Question: Catherine Novack - Jonestrading Institutional Services LLC - Analyst : Just wanted to get a clarity on something. So after a month of discussion, ultimately, didn't get to change the primary endpoint. So where exactly was FDA flexible? What were the requests that you had that FDA did comply with? Was it the dual labeling? Was it LVEF as a key secondary? Where were they flexible with you?

Question: Catherine Novack - Jonestrading Institutional Services LLC - Analyst : Yeah. Okay. And then, I remember at the time of the CRL, there were CMC items that the FDA had not yet addressed. Are these items still outstanding? Or has FDA addressed your responses to those outstanding issues?

Question: Rohan Mathur - Oppenheimer & Co Inc - Analyst : This is Rohan on for Leland Gershell. Just a couple from me. When do you expect to submit the final statistical analysis plan? And if HOPE-3 were to miss the primary, would the (technical difficulty) statistically significant, if a p-value of under 0.05 is achieved? And in that scenario, what would be the likely (technical difficulty)?

Question: Rohan Mathur - Oppenheimer & Co Inc - Analyst : Got it. Just one more. With respect to discussions with the [EU's] CHMP, can you give any updates there? How are those discussions being guided by what's been happening with FDA?

Question: Boobalan Pachaiyappan - Roth Capital Partners LLC - Analyst : Thanks for taking our question. So a couple from us. SO firstly, with respect to HOPE-3, what is your primary -- when you release the data, what if the primary endpoint was not met but you still achieved statistical significance in your secondary endpoint? What could be the potential next steps? That's the first one. And then, in terms of LVEF, I understand that the FDA doesn't want to go against its own regulatory dogma but what if in the future there's a new drug developer who wants to approach only cardiomyopathy and they wanted to use only LVEF as an endpoint because with all the publications from Soslow and others, do you think the agency will be open to discussing LVEF as a primary endpoint for someone else? I know it doesn't concern you but just curious. Your answer will provide some clarity to future drug developers.

Question: Rohan Mathur - Oppenheimer & Co Inc - Analyst : All right. Thanks very much.