Capricor Therapeutics, Inc.

Good afternoon, ladies and gentlemen, and welcome to the Capricors 3rd Quarter 2024 Financial Results Incorporate Update Call. At this time, all participant lines are in listen-only mode. Following the presentation, we will conduct a question-and-answer session. If at any time during this call you require immediate assistance, please press star 0 for the operator. This call is being recorded on Wednesday, November 13, 2024. I would now like to turn the conference over to our host, Mr. E.J. Bergman, CopyCourse Chief Financial Officer for the forward-looking statement. Please go ahead.

Thank you, and good afternoon, everyone. Before we start, I would like to state that we will be making certain forward-looking statements during today's presentation. These statements may include statements regarding, among other things, the efficacy, safety, and intended utilization of our product candidates, our future R&D plans, including our anticipated conduct and timing of preclinical and clinical studies, our enrollment of patients in our clinical studies, our plans to present or report additional data, our plans regarding regulatory filing, potential regulatory developments involving our product candidates, revenue and reimbursement estimates, projected terms of definitive agreements, manufacturing capabilities, potential milestone payments, our financial position, 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. We will caution not to place undue reliance on these forward-looking statements, and we disclaim any obligation to update such statements. With that, I'll turn the call over to Linda Marban, CEO.

Thanks, AJ. Good afternoon, everyone, and thank you for joining today's third quarter conference call. I am extremely proud of the progress we have made in the last quarter. As you know, we have been working to develop Daromyosal, formerly known as TAP10O2, for the treatment of DMD for the last eight years. We have shown in multiple clinical trials the salutary benefits of Daromyosal in attenuating the consequences of the skeletal muscle myopathy and improving the cardiomyopathy associated with this devastating disease. We have consistently presented the data as it has become available to the FDA, and the data has shown clinically meaningful as well as statistically significant improvements. Based on the strength of the data as well as the large unmet medical need of the cardiac implications, we have decided after conferring with the FDA to file a BLA for full approval for the cardiomyopathy associated with DMT. To say that we are proud and excited about our progress here is an understatement. For the new listeners and supporters of Capricor on this call, I will walk you through some of the developments that have brought us here today and outline the steps Capricor is taking as we position our organization to become a revenue-generating, commercial-stage company should we receive FDA approval, for which I am very optimistic. Now, first, I'd like to provide a regulatory update. has been an extraordinary year for Capricorn. We have taken the opportunity to work in collaboration with the FDA to prepare for the potential approval and commercialization of daromyosal to treat the cardiomyopathy associated with DMT. Our BLA will be based on existing cardiac data from our Phase II, HOPE II, and HOPE II open-label extension clinical studies compared to patient-level natural history data from the DMD Cardiac Consortium led by Dr. Jonathan Soslow at Vanderbilt University. I will now take a few minutes to explain how we got to this point from a regulatory perspective. As I have stated previously, we have been presenting clinical data as it becomes available to FDA, and as part of our pre-BLA meeting in August, when we showed them the cardiac data from the HOPE-2 open-label extension study compared to the natural history data set, it became clear that daromyosal was slowing the trajectory of cardiac dysfunction in DMD measured by ejection fraction and measurements of end-systolic and end-diastolic index volumes. FDA noted the strength of our data and also noted that there were no approved therapies for cardiomyopathy associated with DMD. Based on that meeting and subsequent meetings with FDA, we have decided to move forward to file for full approval. The opportunity here is to focus initially on the cardiomyopathy as it addresses a major unmet medical need of those with DMD, is supported by significant clinical data, and most importantly, it is de-risked in that that data is already available. No more clinical data is theoretically necessary, and the label for the cardiomyopathy, which I will provide details on the projected economics of in a few minutes, is broad and ultimately will encompass a large proportion of those with DMD. Now, let me explain the next steps for HOPE III, Cohort A, which was originally slated to be unblinded by year-end of 2024. HOPE III was designed to show that baromyosal attenuates upper limb skeletal muscle disease progression. And as you may recall, the primary efficacy endpoint of HOPE III is the pull or performance of the upper limb 2.0. Therefore, the approval based on HOPE III will be to treat the skeletal muscle myopathy, And while we are pleased thus far with the effect of Daromyosal on upper limb function, it is not as important for the first indication, which will now be cardiomyopathy, and which presents a greater initial market opportunity for Daromyosal. Therefore, we have decided after conferring with FDA to combine cohorts A and B and use that data to serve as a post-approval supplement and add skeletal muscle myopathy to the label in the future. Furthermore, Capricorn may elect to use this data set to support marketing authorizations outside of the USA, should that be necessary, which we'll have more clarity on in 2025.