Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q2 2023 Earnings Call Transcript

Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q2 2023 Earnings Call Transcript August 7, 2023

Operator: Good afternoon, ladies and gentlemen and welcome to the Capricor Therapeutics Second Quarter 2023 Financial Results and Corporate Update Call. At this time all lines are in a listen-only mode. Following the presentation we’ll conduct a question-and-answer session. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to our host, Mr. AJ Bergmann, Capricor’s Chief Financial Officer.

AJ Bergmann: 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 research and development plans, including our anticipated conduct and timing of preclinical and clinical studies, plans to present or report additional data, our plans regarding regulatory filings, potential regulatory developments regarding our product candidates, manufacturing capabilities, potential milestone payments and other 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. You 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, CEO.

Linda Marban: Thank you, AJ. Good afternoon, and thank you for joining our second quarter 2023 conference call. Today, I would like to begin by reiterating our deep commitment to optimizing patient focused medicine and in the first half of 2023, we continued to make steady progress across our pipeline and are well positioned to deliver on important clinical and regulatory milestones for our Duchenne Muscular Dystrophy program, as well as continue to advance our exosome platform technology. I am extremely pleased with the recent additions of two new members to our Board of Directors with the additions of doctors Philip Gotwals and Paul Auwaerter. Dr. Gotwals most recently served as the Global Head Vice President of Business Development and Licensing at Novartis Institutes for Biomedical Research and has nearly 30 years of experience in drug development, research, corporate strategy, and business development.

Dr. Auwaerter brings over 30 years of internal medicine and infectious disease experience as the Sherrilyn and Ken Fisher Professor of Medicine at the Johns Hopkins University School of Medicine, serving as the Clinical Director for the Division of Infectious Diseases and Director of the Sherrilyn and Ken Fisher Center for Environmental Infectious Diseases. Both Dr. Gotwals and Auwaerter deep industry experience will be invaluable as we continue to enhance our strategic and business development priorities across both of our programs. Our late stage clinical development program CAP-1002 in patients with DMD continues to advance through development and key priorities, including enrollment of our Phase 3 HOPE-3 clinical trial which I will give more details on in the next few minutes, as well as continuing discussions with the FDA regarding the proposed path towards submissions of a biologics license application.

Building on this momentum, we believe we are well positioned to execute our corporate executives throughout — objectives throughout the remainder of the year. I will now dive into our programs and provide an update on recent activity. Let me provide you an update on our recent FDA interactions. Earlier this year, as part of our RMAT designation, we have the Type-B CMC, or Chemistry Manufacturing and Controls related meeting with FDA, where we outlined our plans for production of commercial scale GMP CAP-1002. The outcome of this meeting was positive and that FDA clearly outlined for us to deliverables necessary for the CMC portion of our BLA. As we previously have reported, there also was a discussion concerning the possible inclusion of additional patients who would need to be treated with product from our San Diego GMP facility in order to support commercial product manufacturing at this site.

As this request would have likely extended the timeline for the completion of our pivotal trial and therefore the time to BLA. We requested a subsequent Type-B clinical meeting with FDA to determine if there was a more efficient and expeditious path to BLA due to the great unmet medical need of this patient population. The advocacy community believes, as we do, that CAP-1002 attenuate disease progression, and they have continued to raise awareness of our program within the FDA, and specifically [CBER] (ph) and as the FDA continues to highlight the importance of patient perspective, they have continued to pay close attention to our program. To that end, the FDA granted our request to discuss our program in more detail. And this meeting occurred in July of this year.

The goal was to further outline a plan for an expedited path to BLA for CAP-1002 for the treatment of later stage DMD. And I’m happy to share that FDA seemed generally supportive of this plan. Currently, we are awaiting the final minutes from FDA, which we expect this month. We have already had another informal meeting with FDA to further design certain aspects of our program, and we are greatly appreciated of — appreciative of the FDA’s guidance to optimize the HOPE-3 clinical trial design to support our path to potential approval. Next, I’ll provide an update on HOPE-3, our Phase 3 clinical trial. I am very pleased to inform you that as of today, we have randomized 48 subjects across 17 active sites. And based on the currently designed sample size of 68 patients, we are on track to be fully enrolled early in fourth quarter of this year.

Furthermore, we remain on track to conduct an interim analysis on HOPE-3 in the fourth quarter of this year, which will primarily focus on a futility analysis. I am very enthusiastic about the rate of enrollment which has been strong throughout the summer, I’m propelled by the positive feedback from patients and families, including the anecdotal reports of disease attenuation. Additionally, we are extremely pleased by the safety profile of CAP-1002 and the overall support of our investigators and families. Now for an update on our most recent HOPE-2 open label extension or OLE results presented at this year’s Parent Project for Muscular Dystrophy annual conference. To remind you, we presented positive statistically significant two year follow-up results from this study.

As you may recall, the HOPE-2 OLE study previously met its primary endpoint at the one year time points on the PUL version 2.0 scale. At the 24 month time point, the data continued to show statistically significant differences in the PUL performance of the upper limb version 2.0 in the open label extension treatment group when compared to the original rate of decline of the placebo group from HOPE-2 after one year. Even more profound were the cardiac findings we observe, while the natural history of DMD cardiomyopathy suggests a steady decline in cardiac function as measured by ejection fraction. In HOPE-2 OLE, we observed improvements in heart function in six of nine patients. Over time, we also observed an increasing correlation with the performance of the upper limb version 2.0 and ejection fraction results.

The two year results from this open label study are tremendously impactful for DMD patients, so in cardiac and skeletal functional benefits, which underscores the potential long term benefits of CAP-1002 treatment in DMD. The disease modifying potential, together with the favorable safety and tolerability profile, further positions CAP-1002 as a potential anchor therapy for DMD patients. We are thankful to the patients and their families for their continuous commitment to working with us on demonstrating the potential benefits of CAP-1002. Now, turning to our partnership strategy. Now that we have secured commercial and distribution rights in the U.S. and Japan, with our partner Nippon Shinyaku, we continue to focus on securing additional partners in other markets around the world, with Europe being a key priority and now have several companies evaluating the opportunity.

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Another focus I would like to briefly touch upon is the opportunity to evaluate new indications where CAP-1002 might provide benefits to additional patients. While we haven’t committed any resources to this development area yet, this opportunity would provide another potential avenue for partnering, licensing, or even soul development, leveraging on the capabilities we have built at Capricor for CAP-1002. Lastly, I would like to provide an update on our GMP manufacturing facility. We have designed this GMP manufacturing facility to produce commercial scale GMP CAP-1002 doses. We see this facility as a versatile and cost effective way to bring CAP-1002 to market efficiently. In anticipation of CAP-1002 obtaining market approval, we’ve built this facility because we believe having the ability to manufacture in-house will greatly increase our margins and will support the early launch of this product.

I am pleased to report that the San Diego facility is up and running, engineering runs are underway, and we remain on track to be able to release clinical doses in the third quarter of this year. Overall, I am very pleased with the progress of our DMD program. We look forward to sharing further updates from our interactions with FDA, our progress with HOPE-3 and the development of potential additional partnerships in new territories. We have two new additions to our senior leadership team enhancing our regulatory and CMC internal expertise. Dr. Yushi Feng has assumed his role as Vice President of Regulatory, overseeing all regulatory activities. Dr. Feng previously worked at the FDA, Waive Life Sciences, and Kodiak Biosciences. Each of these companies have overlapping areas with — overlapping with our areas of focus.

Additionally, Jonathan Tayco has joined us as Vice President of Program Management and Business Operations over seeing CMC Development. Prior experience for Mr. Tayco included Kite Pharma, where he played an integral role in the BLA approval of Yescarta, one of the first approved cell therapy treatments for large B-cell lymphoma. I am very pleased to welcome these two new members to our team. Now turning to our exosome platform technology, which leverages the natural cell-to-cell communication of the body. We are harnessing exosomes to serve as a novel drug delivery system with broad therapeutic applications and our proprietary StealthX expression platform is at the core of our exosome program, which is focused on the development of two broad modalities, infectious disease, and precision therapeutic.

As previously stated, we believe that exosomes are the future of drug delivery. They are able to deliver contents directly to the cell, avoid detection by the immune system and can be targeted. However, one of the issues that the field has been grappling with is the ability to manufacture a large volume of a relatively homogeneous formulation similar to lipid nano partials. We have focused a significant amount of time and energy into manufacturing and targeting exosomes, and I am delighted to share with you that we have made paradigm shifting progress on these objectives. This development step allows for rapid and efficient development of future exosome product. To that end, I’m very pleased to report that we have recently begun work with an undisclosed pharma company looking at enhancing delivery of ASOs using exosomes as the delivery vehicle.

I look forward to providing more details on this in the coming months as this is a very important step for Capricor as we begin to leverage our platform StealthX. We look further to exploring the applications of our StealthX platform as they expand into precision based therapeutic and are working in targets primarily in neuromuscular diseases at this time. Additionally, we recently published data in bioRxiv, a journal featuring two vaccine candidates that were generated with StealthX. Exosomes are engineered to express influenza H3 or SARS-CoV-2 spike. When administered individually, both StealthX, H3 and Stealth X spike introduced a strong immunization with the production of a potent humeral and cellular immune response. These effects were obtained with administration of nanograms of protein and without adjuvant or lipid nano particles to test the potential benefits of the [indiscernible] vaccine.

Hemagglutinin H3 and SARS-CoV-2 spike proteins were individually engineered on the exosome surface and mixed before injection. This combination approach also induced a strong immune response in mice, with both antibody and T-cell response. Additionally, there were no detectable immune competition between antigens by combining multiple targets in a single vaccine formulations, these results support the potential therapeutic utility and versatility of our StealthX platform and addressing a broad range of infectious diseases. Our current plans continue to focus on leveraging business development and partnering strategies as well as non-dilutive grant funding for our exosome platform technology. In closing, we are pleased with the advancements across our organization as we remain focused on becoming a commercial scale company with CAP-1002 as our lead asset and development of our exosome technology.

We are entering the second half of the year in a position of strength. I look forward to working with our new board members and capitalizing on their deep industry experience and forward-looking vision which we feel will be invaluable as we continue to execute on the key priorities for CAP-1002 and our exosome platform. Now with that, I will turn the call over to our Chief Financial Officer, AJ Bergmann to run through our financial results for the second quarter of 2023.

AJ Bergmann: Thank you, Linda. This afternoon’s press release provided a summary of our second quarter 2023 financials on a GAAP basis. You may also refer to our quarterly report on Form 10-Q, which we expect to become available shortly and will be accessible on the SEC website, as well as the financial section of our website. As of June 30, 2023, the company’s cash, cash equivalents and marketable securities totaled approximately $37.8 million compared to approximately $41.4 million on December 31, 2022. Based on our current operating plan, company’s cash position is expected to be sufficient to support operations through the third quarter of 2024. I would like to note that this expectation excludes any additional potential milestone payments under our exclusive commercialization and distribution agreements with Nippon Shinyaku that may become due, which would extend our cash runway.

Turning to the financials. Revenues for the second quarter of 2023 were approximately $3.9 million compared with zero for the second quarter of 2022. The primary source of revenue was from the ratable recognition of the $30 million upfront payment in accordance with our U.S. exclusive commercialization and distribution agreement we received from Nippon Shinyaku. Moving to our operating expenses for the second quarter of 2023, excluding stock based compensation, our research and development expense was approximately $8.4 million compared to approximately $4.7 million in Q2, 2022. Again, excluding stock based compensation, our general and administrative expenses were approximately $1.7 million in Q2, 2023, and approximately $1.4 million in Q2, 2022.

Net loss for the second quarter of 2023 was approximately $7.4 million compared to a net loss of approximately $7.1 million for the second quarter of 2022. Net loss for the first half of 2023 was approximately $15.1 million compared to a net loss of approximately $14.9 million for the first half of 2022. And with that, we will now open the line-up for questions. Thank you very much.

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Q&A Session

Operator: Thank you, sir. Ladies and gentlemen, we will now begin the question-and-answer session [Operator Instructions] Your first question comes from the line of Joe Pantginis from H. C. Wainwright. Please go ahead.

Joe Pantginis: Hi. Good afternoon, Linda and AJ. Thanks for taking the questions. So I know there’s a lot of working parts here. And I know my questions will revolve around some things that you might not be able to detail because the FDA minutes are not out yet, but I’m still going to ask them. So with that said, I guess, as San Diego comes online this quarter, if I heard you correctly, do you have a general range of the number of patients that would be required from that facility to start with?

Linda Marban: Yes, Joe. Hey. Good to talk to you. And, you’re correct. There’s very little we can say until we get the final minutes. But what I can tell you is that, once we get them, we plan on doing a public release in some way either via press release and/or a call to sort of update the market and the community as to exactly the path forward. What I can tell you is that, FDA has worked really closely with us. They’re very supportive of this program, several of our patients’ families have reached out to FDA in order to ask them to take a really careful look at Capricor, help move the program forward because they feel that their sons are feeling and focusing better. And so, we’re confident that the answers that we get back from FDA will be the best ones possible to move the products towards BLA.

Joe Pantginis: That makes sense. And, I guess for my next question, it’s sort of in the same realm, but I’ll ask it sort of scenario based. So, you do have the type — you do have the minutes that you’re waiting right now. You also have the upcoming interim analysis in the fourth quarter for HOPE-3, which you said primarily based on futility. However, in your — does one of the potential scenarios include the positive feedback from the minutes with regard to the data you might see in the interim that could allow for the acceleration or resetting of the timeline positively for HOPE-3.

Linda Marban: Yes. That was a pretty impressive question asked, Joe. Good work. I can tell you’ve been doing this a long time. So, the answer to that is, futility is the metric that we’re looking at for the interim analysis. FDA has been pretty clear with us that they want to keep this trial very tightly regulated, really carefully monitored, because they believe in the product too, we can tell from their feedback from us — for us. And so, we’re probably not going to use the interim for accelerated approval opportunity. I can tell you that I am laser focusing on opportunities where we may be able to take advantage of an earlier look to look for an accelerated approval. I cannot disclose now how we might do that, but that is something we’re definitely thinking about.

Joe Pantginis: Got it. And I guess my other questions are more — are strictly logistical. So in the conduct of HOPE-3, you’re obviously encouraged by the enrollment rates that you’ve been seeing, if I understand correctly. What are the prominent reasons for screening failure of patients or what does the patient and also what does the patient pipeline look like for potential enrollment?

Linda Marban: Yes, Joe. Great question. So screen fail rate, which has really come down a lot, thanks to our really very strong new clinical team that we put in place. And the primary reason that people fail are typically is that they don’t have inclusion criteria of the performance of the upper limb score two to five. And so, we typically see some of these guys whose scores are too low. And let me just say from a human level, it’s heartbreaking because they have to have the ability to use their hand and brand to their mouth unassisted. And we probably get two or three calls a week from people saying, well, you know, if they — what if they need help, what if they use their other hand to boost it? So people want CAP-1002 desperately.