Capricor Therapeutics, Inc.

This conference call is being recorded. I would now like to turn the conference call over to our host, Mr. A.J. Bergman, Cap Record's Chief Financial Officer. Please go ahead.

Thank you. Good afternoon, everyone. Before we start, I would like to state that we will be making certain forward-looking statements during today's call and presentation. These statements may include statements regarding, among other things, the efficacy, safety, and intended utilization of our product candidates. our future research and development plans, including our anticipated conduct and timing of preclinical and clinical studies. Our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory developments involving our product candidates, manufacturing capabilities, potential milestone payments, 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. Your caution not to place undue reliance on these forward-looking statements, and we disclaim any obligation to update such statements. With that, turn the call over to Linda Marban, CEO.

Thanks, AJ. Good afternoon and thank you for joining us today. I'm extremely pleased with the progress we are making towards the development of our lead asset, CAP 1002, for the treatment of Duchenne muscular dystrophy. As I begin the call today, I am delighted to inform you that we have reached our targeted enrollment goal for our HOPE III phase III pivotal study, which is designed to enroll approximately 58 patients across the United States. Additionally, we are planning on announcing the outcome of our interim analysis from the study prior to the end of this year. The purpose of this important milestone will be to determine if the trial is futile or should continue as planned. The analysis is based on the six-month results. Reminder, our primary endpoint is based on one year. This blinded data will be presented to our DSMB which will evaluate safety and efficacy and give us their decision as to whether the trial should continue as planned. Should the trial be determined not to be futile, this will trigger our first milestone payment under our U.S. agreement with Nippon Shinyaaku. There are also other potential milestone payments leading up to an approval of a BLA that will continue to support our balance sheet should we achieve them. As I have previously announced, the top line results from cohort A will be available in Q4 2024, and a subsequent BLA would be based on the full data set. However, we are now planning to reinvigorate our discussion with FDA to determine if there is an opportunity to speed up the approval pathway for CAP10A2 now that our enrollment target for HOPE3 cohort A has been reached. We owe it to the patients and the community to move CAP 1002 towards approval as quickly as possible, as everyone knows that time is muscle. Once a pull point is lost, it cannot be recovered. Now, this brings me to an overview of our recent Type B clinical meeting with the FDA, which was announced in late Q3, and why we believe the outcome was important for the program. As you may recall, earlier in 2023, FDA indicated that they wanted us to treat additional patients in the HOPE III study with the product manufactured at our San Diego GMP facility. There were two main outcomes from this meeting. Number one, the results of the HOPE III cohort A will support the filing of a BLA, and number two, FDA approved the design of an additional cohort of patients, now referred to as cohort B, to support the inclusion of our new San Diego facility for commercial manufacturing and enrollment is planned to begin imminently. It is very important to keep in mind that Cohort B and the data that we generate from it will not be required for initial registration of CAP 1002 for DMG. Over the last several years, Capricor has carefully and diligently invested resources in this program, bringing CAP-1002 through three successful clinical trials, building two manufacturing facilities, securing RMAT, orphan drugs, and rare pediatric designations from FDA, in addition to securing commercialization partnerships for the U.S. and Japan markets. These strategic deals were carefully negotiated and potentially bringing over $700 million in additional cash to Capricor, which would fuel future product potential expansion of PAP-10-02, and allow us to strategically invest in the further development of our exosome pipeline. This is the vision that I have for Capricor, which would transform our organization from a development stage therapeutic company into a world-class commercial and R&D operation. I realize much has to be done and accomplished before that time, but now that we have completed our target enrollment in the phase three, we are moving closer to that vision. To this point in our history, we have remained disciplined across the organization, not only in the management of cash and resources, but also with respect to our priorities as we have developed Cap10 or 2. We made the early decision to invest in the clinical development of Cap10 or 2 while managing our manufacturing operations in a just-in-time manner. Now that we have shown promising data in multiple clinical trials, we are focusing on scaling up and out our manufacturing capabilities to produce larger quantities of CAB 1002 to meet FDA requirements and market demand, if approved. Our GMP manufacturing facility in San Diego cost approximately $3 million to build and equip. As is the case with all companies, launching a complex biologic such as CAB 1002 requires us to expand operations to include experts in cell manufacturing, quality, compliance, and regulatory affairs. which is reflected in the current structure of the organization. Capricorn has raised approximately $145 million in equity capital in totality throughout our company's history, both private and public, over the past 15 years. And with approximately $15 million in cash resources factoring in our most recent financing and Q3 financials, this gives us a runway into 2025. The dollars invested in CAP 1002 for DMD have strengthened many aspects of our program, most notably the ability to understand the mechanism of action of CAP 1002, recognizing that one of the hindrances to the approval of cell therapies has been an inadequate potency assay. We are pleased that the FDA supports our potency program, which is an important step towards approval. With the path paved by DMD, we are now planning product expansion into diseases with similar pathophysiology. We believe in CAP1002's potential to be a transformational treatment for patients with diseases of inflammation and fibrosis. Second to that, we are judiciously building our exosome platform technology to become a next-generation drug delivery platform. Now with that said, let's quickly turn to an update on our exosome technology. While our primary focus has been on advancing the development of Cap Center 2. We remain committed to our exosome technology as part of next generation drug delivery platform. Currently, we are pursuing two avenues of opportunity. One is our vaccine program using StealthX, our proprietary platform that is useful for engineering select proteins either inside or on the surface of the exosome. For our vaccine program, one of our aims is to secure either a partnership or a non-dilutive source of funding to speed clinical development. Currently, there is significant interest in an exosome-based vaccine platform, and we are in active discussions with several parties. We remain focused on that mission. The other aspect of our platform uses StealthX to develop therapeutics by harnessing exosomes as the delivery vehicles. The program requires loading of specific cargoes into exosomes with targeting moieties on the outside of the exosome, essentially to tell it where to go. Currently, in collaboration with an undisclosed pharma company, we are investigating the therapeutic perspective of this platform. The foundational work has the potential to set the stage for future therapeutic options with exosomes. Initial data from this ongoing study was presented recently, at this year's World Muscle Society Conference. Proof of concept results demonstrate that a muscle targeting moiety can be engineered on the surface of exosomes as demonstrated by the presence of a labeled ASO, which is antisense oligonucleotide, and used as representative cargo in the lower limbs of mice post intravenous injection. Repeated doses of the loaded exosome show enhanced targeting based on the initial PK study. This suggests that repeat dosing using our engineered exosomes has the potential to effectively enhance delivery, further differentiating our therapeutic approach from lipid nanoparticles. I look forward to providing more color on this important program as it becomes available. Now, finally, on the corporate side, we raised approximately $23 million this quarter to support our balance sheet into 2025. This strategic financing was anchored by Nippon Shinyaku, further cementing our strong relationship and their commitment to Capricorn. Part of the reason for the fundraise was we had to expand our team in order to prepare for the submission of a BLA. The work required to prepare a late-stage clinical asset is significant, and we are fortunate to have found a team that is experienced in driving programs through approval in the biologic space. Finally, in our effort to expand Capricorn's team of experts, we are pleased to announce the appointment of Michael Kelleher to our Board of Directors. Most recently, Michael served as Group Vice President of M&A and Business Development at Horizon Therapeutics, now part of Amgen. He brings to Capricorn expertise in business and strategic development. We look forward to Mike's contributions to our team. Overall, I want to thank you for your support for our program and our company. We are very much looking forward to the next several months as we will be announcing the outcome from the interim analysis, as well as continuing our interactions with FDA. I will now turn the call over to AJ to go through our financials. Thank you.