Capricor Therapeutics, Inc.

I would now like to turn the conference over to our CFO, E.J. Bergman, 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 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 are cautioned 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.

Thanks, AJ. Good afternoon, everyone, and thank you for joining today's conference call. The mission of Capricor has always been to discover and develop transformative drug products for patients in need, and translating these biological medicines into commercial products. As many of you know, Capricor was founded over 20 years ago, originally in the laboratories at Johns Hopkins University, where we isolated, characterized, and harnessed a unique cell type derived from heart tissue. Even at that time of discovery, the cardiosphere-derived cells, which are now known as theramiocel, were conceptualized to potentially be a powerful cellular therapeutic which one day could treat diseases of the heart, for which there are many. Now let me remind you that Daromyosal has a defined mechanism of action, which is primarily immunomodulatory and antifibrotic, driven by the release of exosomes after the cells are infused. Our identity and potency assays, which are supported by the FDA, are based on those properties of bioactivity. Daromyosal is not a stem cell, let me remind you. Its active ingredient is a rarefied population of cardiac cells that Capricor isolates and expands using proprietary methods. Cells are infused in a simple, one-time-per-quarter intravenous infusion at a dose of 150 million cells. With over 700 infusions of the product over many years, Daramaya cells have been shown to be a safe and effective means for attenuating disease progression. As many of you know, we have been working for over eight years developing Daromyosal as a treatment for those impacted by Duchenne muscular dystrophy. And it is with great pride and joy that I can say we find ourselves potentially nearing approval. Today, I will walk you through the latest regulatory, DMC, and commercial updates as Capricor begins to transition from a translational medicines company to a potentially commercial stage company. We will continue to stay committed to our mission, developing transformational treatments for patients in need and driving value for our shareholders. Now turning first to our biologics license application, or BLA filing for DMD cardiomyopathy, which we announced in early March was accepted by FDA for priority review. The application is seeking full, not accelerated, approval of Daromyosal to treat DMD cardiomyopathy, an aspect of the disease that not only afflicts essentially all DMD patients, but also is the leading cause of mortality associated with DMD. As we look ahead to our PDUFA date set for August 31st, 2025, we are working with the FDA as they are actively reviewing our application. At this time, the FDA has not indicated to us whether an ADCOM will be necessary. But we are preparing for one, should that be needed. And I am pleased to inform you that we have officially scheduled our PLI, or pre-licensing inspection, of our manufacturing facility, which is set for the second quarter of this year. Our BLA is supported with data from two trials. Our Phase II, Hope II, placebo-controlled trial and our HOPE II open-label extension trial compared to patient-level natural history data from the DMD Cardiac Consortium led by Dr. Jonathan Soslow at Vanderbilt University. Many factors have given us confidence in our BLA submission pathway. First and foremost, it has a strong safety profile and has been administered to over 250 human subjects across several clinical trials over multiple years. Equally as important is that the data continues to show clinical and statistically significant efficacy in the treatment of DMD cardiomyopathy. This data is foundational to our BLA filing. I would also like to point out that it has become well known that the cardiac and skeletal aspects of the disease do not decline at the same rate. So therefore, because we see an impact on both cardiac and skeletal muscle function, we are confident that we are seeing a treatment effect of Daromyosal across multiple domains, further strengthening the opportunity to treat DMG with Daromyosal. Furthermore, as exhibited this week at the Muscular Dystrophy Association Conference, we see a year-over-year improvement in function, suggesting that long-term use of Daromyosal is warranted for the treatment of DMD. We also have continued to work in close collaboration with the FDA. We have been the beneficiaries of CBIR's approach to drug development and believe that with the combination of statistically and clinically significant data that addresses an unmet medical need, we are well poised to gain approval of Daromyosil for DMD cardiomyopathy. In addition, our RMAT designation allows us to work directly with FDA as we realize the true goal here is to bring the first treatment to market for DMD cardiomyopathy for which there are no approved therapies. Lastly, throughout 2024, we've met with FDA multiple times outlining our BLA filing strategies. of which we have discussed previously. And I want to reiterate that they have not requested the HOPE III data, and currently we do not believe it will be necessary to support our application. As you may recall, the HOPE III clinical trial has a primary efficacy endpoint of the performance of the upper limb version 2.0, which is an indicator of skeletal muscle function. We plan to use this data in the future for potential label expansion. To that end, we are currently evaluating the plans for HOPE III and will provide more updates as they become available. Now turning our attention to commercial planning. We are actively working with our commercial partner, NS Pharma, on launch readiness for the United States. Key areas of overlapping focus are market access and reimbursement. Their team in the United States is comprised of approximately 125 people with market access, reimbursement, medical affairs, and sales teams actively preparing for the launch of Daromyosil in the United States. We recently completed surveys with the top five payers in the U.S. with the response coming in very favorably from a reimbursement standpoint for Daromyosil. Since there are currently no approved therapies for DMD cardiomyopathy, and data suggests that standard cardiac medications do not have a significant impact on disease progression in most cases, we see daromyosal as a transformational treatment option for these boys and young men. We expect reimbursement would be consistent with other recently approved DMD therapies, such as exon skippers. If approved, We are anticipating that approximately 50% to 60% of the overall DMD population in the United States, or around 7,500 boys and young men with DMD, would be eligible for treatment with Daromyosal. If approved, we are anticipating entering the market with approximately 100 patients transferring from our open-label extension trial to commercial products. This would drive revenue to the bottom line. To remind you, in the U.S., we are entitled to between 30% to 50% of revenue share based on sales of the product, inclusive of cost of goods sold. I'd like to shift our attention to CMC, or Chemistry Manufacturing and Controls. Our current GMP compliance facility, located in San Diego, is fully operational and staffed, producing doses of Daromyosal. We built and carefully designed this facility to meet early market demand. The fully operational capacity in this facility can support approximately 250 to 500 patients per year, and we believe will be sufficient to support the anticipated first year of launch. As we expect a strong launch and rapid adoption of Daromyosal for DMD cardiomyopathy, if approved, I am pleased to announce that we have expanded our current San Diego facility where we have leased an additional 25,000 square feet for which we are going to build additional clean rooms, taking our manufacturing capacity for approximately 2,000 to 3,000 patients per year, once completed and fully operational. We have assembled a world-class team who is extremely sophisticated in this area, and I have high confidence we'll have this new expanded facility online by mid-2026, allowing us to bolster supply of the product for the next several years to meet demand. As we continue to expand our capabilities, we are already looking into product development endeavors to allow us to increase our yield further. On the corporate and commercial front, we are able to look at our balance sheet. Our cash balance of approximately $150 million is being deployed across the organization with our current runway into 2027 with no additional infusions of cash. If we receive FDA approval, we will be slated to receive an additional $80 million milestone payment from Nippon Shinnyaku. And in addition, we would receive a priority review voucher, which we have the full right to sell or transfer. These non-dilutive cash infusions could total well over $200 million, which would hit our balance sheet in 2025 alongside our existing cash and potential product revenue. This puts us in a strong position to deliver for our shareholders on multiple fronts. Our strong cash position will continue to allow us to strengthen our commercial organization, which includes enhancing our team with commercial and medical expertise in order to fuel future product development opportunities for Daramaya Cell and enable us to build Capricor into a world-class revenue-generating, cashflow-positive company with a commercial product on the market and a robust pipeline of expansion opportunities leveraging cell and exosome-based therapeutics. Last, for a brief update on our European partnering efforts, last year we entered into a term sheet with Nippon Shin'yaku for the marketing, sales, and distribution of Daromyosal in the European region, subject to finalization of a definitive agreement. Our commitment to Nippon Shinnyaku in the USA and Japan as our commercial partner is strong, but we have not yet come to final terms on the definitive agreement, which is still being negotiated. In the meantime, we have achieved important regulatory designations in Europe and are on track for meeting with EMA in the second quarter of 2025. We will provide additional color as our strategy for Europe continues to unfold. Now, I'd like to switch gears and give an update on our exosomes pipeline program. We continue to develop our StealthX exosome platform technology as part of a next-generation drug delivery platform. Our goal is to build the exosomes into a standardized drug delivery platform that has enhanced capabilities when compared to a lipid nanoparticle, including targeting and delivering contents across the cell membrane. While most of our focus has been on the commercialization of daromyosal, we have had a small team working on building the exosomes in the background. They have successfully designed a manufacturing method that is cost-effective and can be expanded to make large amounts necessary for therapeutic delivery. We have presented this proof-of-concept data at many scientific meetings and published these findings in peer-reviewed journals. Our approach is to concurrently demonstrate the utility of the exosome by developing a vaccine platform that is unique, using native proteins loaded in or coded on an exosome that could be made rapidly within the 100-day constraint developed by the U.S. government, but also able to generate a robust and long-lasting immune response. That program is part of the U.S. government's Project NextGen. which aims to test vaccine candidates for COVID-19 prevention in addition to prepare for future pandemics. Currently, our StealthX vaccine candidate is in the manufacturing phase. The NIAID, which is the National Institutes of Allergy and Infectious Disease, will then conduct and fully fund a phase one clinical trial. Currently, manufacturing is underway for our StealthX vaccine, And the NIAID is planning for regulatory approval in the second quarter of 2025 with the clinical study initiated soon thereafter. We will provide further updates on this program as they become available. Now that DERM-ISL is on a defined path towards potential commercialization, we are further evaluating the path forward for therapeutic exosome pathway and also evaluating other opportunities to expand our future pipelines. In conclusion, 2024 was a transformational year for Capricorn, and 2025 is the year in which we will hopefully transition into our next stages of development. I am proud of our progress and grateful to the patients, their families, our investors for their continued support. Capricorn's goal is to continue to meet its milestones as we continue to focus on our efforts on bringing Jeremiah Hill towards potential commercialization and are investing judiciously across the organization. to prepare for that endeavor. I will now turn the call over to A.J. to run through our financials. A.J.?