Q2 2024 Capricor Therapeutics Inc Earnings Call
Speaker Change: .
Speaker Change: Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics second quarter 2024 earnings call. At this time, all participant lines are in the listen-only mode. Following the presentation, we will conduct a question and answer session.
AJ Bergmann: And if at any time during this call you require immediate assistance, please press star zero for the operator. Also note that this call is being recorded on Wednesday, August 7th, 2024. And I would like to turn the call over to our CFO , AJ Bergmann, for the forward looking statement.
AJ Bergmann: Thank you. Good afternoon, everyone.
AJ Bergmann: 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.
AJ Bergmann: 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 filings, potential regulatory developments involving our product candidates, revenue and reimbursement estimates, manufacturing capabilities, potential milestone payments, our financial position, and our possible uses of existing cash and investment resources.
AJ Bergmann: 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 the actual results to differ materially from those contained in the forward-looking statements.
AJ Bergmann: 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.
AJ Bergmann: With that, I'll turn the call over to Linda Marbn, CEO . Thanks, AJ. Good afternoon, and thank you for joining today's second quarter conference call.
Linda Marbn: Now, over halfway through 2024, Capricor continues to make significant progress on bringing our lead asset, Daramycel, which was previously referred to as CAP1002, closer to the submission of a Biologics License Application, or BLA.
Linda Marbn: for the treatment of Duchenne muscular dystrophy, as well as developing our pipeline of exosome products for clinical development.
Unknown Executive: Our published results in two independently conducted clinical trials and the recent long-term open-label extension results, we believe are some of the strongest data seen in the space. DMD will require a multi-drug paradigm to address all of the pathological consequences of this. I want to remind everyone that as DaraMyself moves towards potential approval and commercialization. In addition, greater improvements in cardiac function were observed in those patients that had ejection fractions greater than 45 percent at the beginning of the HOPE II randomized clinical trial.
Unknown Executive: Published results in two independently conducted clinical trials and the recent long-term open-label extension results, we believe are some of the strongest data seen in the space. As Daramiafel addresses the inflammation and fibrosis caused by cell death due to the lack of dystrophin, which typically protects muscle cells from damage, we believe it can serve as a potential backbone therapy for all boys and young men afflicted with DMG, with the product's ability to significantly slow disease progression from both a skeletal and cardiac standpoint.
Linda Marbn: Our published results in two independently conducted clinical trials and the recent long-term open label extension results, we believe are some of the strongest data seen in the space.
Linda Marbn: As Deremia cell addresses the inflammation and fibrosis caused by the cell death due to the lack of dystrophin, which typically protects muscle cells from damage.
Linda Marbn: We believe it can serve as a potential backbone therapy for all boys and young men afflicted with GMG, with the product's ability to significantly slow disease progression from both a skeletal and cardiac standpoint.
Unknown Executive: Additionally, as the KOLs across the DMG fields have continued to mutate, DMD will require a multi-drug paradigm to address all of the pathological consequences of this. I want to remind everyone that as DARE MySELF moves towards potential approval and commercialization. Our United States Distribution Agreement with Nipponshiniyaku provides for approximately $700 million in potential developmental and sales milestones that may become due capital, which would enable us to invest in our commercial launch and give us the resources to strategically expand our focus to other diseases of inflammation and fibrosis.
Linda Marbn: Additionally, as the KOLs across the DMT fields have continued to message, DMT will require a multi-drug paradigm to address all of the pathological consequences of this disease.
Linda Marbn: to
DaraMyself: So I want to remind everyone that as DaraMyself moves towards potential approval and commercialization.
Speaker Change: Our United States distribution agreement with Nippon Shinyaku provides for approximately $700 million in potential developmental and sales milestones that may become due to Capricor.
Speaker Change: Which would enable us to invest in our commercial launch and give us the resources to strategically expand our focus to other diseases of inflammation and fibrosis.
Unknown Executive: In fact, we were preparing to discuss opportunities to potentially expand darahmia cells into Becker muscular dystrophy with the FDA later this year. We believe in DeremiaCell's potential to become a transformational treatment for DMD and beyond. And we are positioning Capricor to become a world-class commercial and R&D operation with a robust pipeline of opportunities for growth, including the expansion of our StealthX exosome-based pod. My goal is to position the organization to deliver on that transformation, and I continue to be proud of the progress that we have made to date.
Speaker Change: In fact, we are preparing to discuss opportunities to potentially expand theramia cells into Becker muscular dystrophy with the FDA later this year.
Speaker Change: We believe in Jeremiah's health potential to become a transformational treatment for DMD and beyond.
Speaker Change: And we are positioning Capricor to become a world-class commercial and R&D operation with a robust pipeline of opportunities for growth, including the expansion of our StealthX exosome-based platform.
Speaker Change: My goal is to position the organization to deliver for that transformation, and I continue to be proud of the progress that we have made to date.
Unknown Executive: Now, I'd like to take a moment to update you on our recent three-year HOPE-II Open Label Extension, or OLE, study results, which were presented at this year's PPMD annual meeting. Treated subjects showed a statistically significant benefit with a p-value of 0.001 and an improvement of 3.7 pull points on the pull 2.0 when compared to an external comparator data set of similar DMD patients. This translates into an approximate 50% delay in disease progression in treated patients versus the external comparison. Drill, drill, drill, drill.
Speaker Change: Now I'd like to take a moment to update you on our recent three-year HOPE II Open Label Extension or OLE study results which were presented at this year's PPMD annual meeting.
Speaker Change: Treated subjects showed a statistically significant benefit with a p-value of 0.001 and an improvement in 3.7 pull points on the pull 2.0.
Speaker Change: When compared to an external comparator dataset of similar DMD patients, this translates into an approximate 50% delay in disease progression in treated patients versus the external comparator.
Unknown Executive: Data also showed improvements in multiple cardiac measures, including left ventricular ejection fraction, as well as indexed left ventricular and left ventricular end-systolic volume and end-diastolic volume. These are measures of cardiac function and are considered highly relevant in terms of predicting long-term outcomes. In addition, greater improvements in cardiac function were observed in those patients that had ejection fractions greater than 45 percent at the beginning of the HOPE II randomized clinical trial. Currently, as you know, there are no approved treatments specifically for DMD cardiomyopathy.
Speaker Change: Thank you everyone.
Speaker Change: Data also showed improvements in multiple cardiac measures, including left ventricular ejection fraction, as well as indexed left ventricular and left ventricular end systolic volume and end diastolic volume.
Speaker Change: These are measures of cardiac function and are considered highly relevant in terms of predicting long-term outcomes.
Speaker Change: In addition, greater improvements in cardiac function were observed in those patients that had ejection fractions greater than 45% at the beginning of the hoped-to randomized clinical trial.
Unknown Executive: Currently, as you know, there are no approved treatments specifically for DMD cardiomyopathy. Our HOPE-2 Open Label Extension Study continues to show a favorable safety profile, and we believe these three results continue to underscore the potential long-term and consistent benefits of daramysel for the treatment of DMD, which is the leading cause of death for those with DMT. Another option is a traditional full approval.
Speaker Change: Currently, as you know, there are no approved treatments specifically for DMD cardiomyopathy.
Unknown Executive: Our HOPE-2 Open Label Extension Study continues to show a favorable safety profile, and we believe these three results continue to underscore the potential long-term and consistent benefits of daramysel for the treatment of DMD. Based on the favorable data from our clinical trials, I want to reiterate that our primary goal is to file a BLA as soon as possible, and we are actively working with FDA to accomplish that goal. Darah Miasel is the only therapeutic, to our knowledge, that has shown statistically significant improvements in cardiac function and DMD-Associated Cardiomyopathy, which is the leading cause of death for those with DMT.
Speaker Change: Our HOPE II Open Label Extension Study continues to show a favorable safety profile, and we believe these three results continue to underscore the potential long-term and consistent benefits
Speaker Change: of Deremiasel for the treatment of DMD.
Speaker Change: Based on the favorable data from our clinical trials, I want to reiterate that our primary goal is to file a DLA as soon as possible, and we are actively working with FDA to accomplish that goal.
Speaker Change: Deremyosel is the only therapeutic, to our knowledge, that has shown statistically significant improvements in cardiac function and DMD-associated cardiomyopathy, which is the leading cause of death for those with DMD.
Unknown Executive: FDA is aware of the strength of Capricor's cardiac data and may allow for its inclusion in the label for daromyosal, which FDA acknowledges as important to the DMD patient community. We have just met with FDA in a very positive pre-BLA meeting. And while we are waiting for the final minutes from the meeting, I can share that we are discussing alternative paths to our BLA filing. One pathway would be filing our BLA with currently available data from the HOPE-2 and the HOPE-2 OLE studies to support an accelerated approval pathway with confirmatory data to be provided at a later date. Another option is traditional full approval.
Speaker Change: FDA is aware of the strength of Capricor's cardiac data, and may allow for its inclusion in the label for daromyosol, which FDA acknowledges as important to the DMD patient community.
Speaker Change: we have just met with f d a in a very positive preva meaneting and while we are waiting final minutes from the memeaneting i can share that we are discussing alternative paths to our balways violent
Speaker Change: One pathway would be filing our BLA with currently available data from the HOPE-2 and the HOPE-2 OLE studies to support an accelerated approval pathway with confirmatory data to be provided at a later date.
Unknown Executive: We are pleased to announce FDA's acceptance of our rolling BLA submission, and based on that, Capricor intends to initiate the filing of our BLA shortly. On the CMC front, as we reported last quarter, our San Diego manufacturing facility is fully operational and actively preparing for commercial runs necessary for the filing.
Speaker Change: Another option is a traditional full approval. We are pleased to announce FDA's acceptance of our rolling BLA submission, and based on that, Capricor intends to initiate the filing of our BLA shortly.
Speaker Change: On the CMC front, as we reported last quarter, our San Diego manufacturing facility is fully operational and actively preparing for commercial runs necessary for the filing.
Unknown Executive: Additionally, we have accelerated the pace of our preparations for the commercial market, and our partner, Nipun Sinyaku, is focused on their role in getting DERA Mycel ready for launch. We, and they, expect rapid adoption of Daramysel by payers and patients with DMD. We are actively engaged with them to ensure a smooth and successful launch. Capricor is focused on the areas of preparation for market access, reimbursement, as well as opportunities for expanded label use.
Speaker Change: Additionally, we have accelerated the pace of our preparations for commercial market entry.
Speaker Change: Our partner, Nippon Shinyaku, is focused on their role in getting DERA Myocell ready for launch. We, and they, expect rapid adoption of DERA Myocell by payers and patients with DMD.
Speaker Change: We are actively engaged with them to ensure a smooth and successful launch. Capricor is focused on the areas of preparation for market access, reimbursement, as well as opportunities for expanded label usage.
Unknown Executive: Now, for an update on our exosome program, while our primary focus is on advancing their might as well, we are committed to our stealth exosome platform technology as part of our next generation Drone Delivery Pop. We have been planning for the success of DERAMYCEL for several years and, to that end, have built a pipeline of engineered exosome products based on our foundational cell therapy experience. The goal has been to take advantage of our experience with scaling up and out cell therapies, as well as to continue to refine cell-based products such as, In terms of ability to be quantified based on mechanism of action, as well as targeting them for more strategic treatment paradigms.
Speaker Change: Now for an update on our exosomes program. While our primary focus is on advancing dermisal, we are committed to our stealth exosome platform technology as part of our next generation drug delivery platform.
Unknown Executive: We have been planning for the success of DERAMYCEL for several years and, to that end, have built a pipeline of engineered exosome products based on our foundational cell therapy experience. This program requires the loading of specific cargoes into the exosomes and then attaching targeting moieties on the outside of the exosome to direct it where to go. Regarding our vaccine candidate, we are actively collaborating with the United States government's Project NextGen, which aims to test vaccine candidates for COVID-19 prevention and also to prepare for future pandemics, which is being developed as a potential enzyme replacement therapy.
Speaker Change: We have been planning for the success of DERAMYCEL for several years and to that end have built a pipeline of engineered exosome products based on our foundational cell therapy experience.
Speaker Change: The goal has been to take advantage of our experience with scaling up and out cell therapies, as well as to continue to refine cell-based products such as exosomes.
Speaker Change: Thank you for joining us. Thank you. Thank you.
Speaker Change: in terms of their ability to be quantified based on mechanism of action as well as targeting them for more strategic treatment to paradigm
Unknown Executive: Our exosome platform focuses on the use of our proprietary Stealth-X technology to develop therapeutics by harnessing exosomes as delivery vehicles. This program requires the loading of specific cargoes into the exosome and then attaching targeting moieties on the outside of the exosome to direct it where to go.
Speaker Change: Our exosome platform focuses on the use of our proprietary Stealth-X technology to develop therapeutics by harnessing exosomes as delivery vehicles.
Speaker Change: This program requires the loading of specific cargoes into the exosomes and then attaching targeting moieties on the outside of the exosome to direct it where to go.
Unknown Executive: We are currently ramping up our business development efforts with our Exosome platform technology as we continue to develop the therapeutic opportunities of the platform. Regarding our vaccine candidate, we are actively collaborating with the United States government's Project NextGen, which aims to test vaccine candidates for COVID-19 prevention and also to prepare for future pandemics. Currently, our vaccine candidate is in the manufacturing phase, with plans to deliver it to the National Institutes of Allergy and Infectious Disease by the end of 2024. The NIAID will conduct and fully fund a phase one clinical trial.
Speaker Change: We are currently ramping up our business development efforts with our Exosome platform technology as we continue to develop the therapeutic opportunities of the platform.
Speaker Change: Regarding our vaccine candidate, we are actively collaborating with the United States government's Project NextGen, which aims to test vaccine candidates for COVID-19 prevention and also to prepare for future pandemics.
Speaker Change: Currently, our vaccine candidate is in the manufacturing phase, with plans to deliver to the National Institutes of Allergy and Infectious Disease by the end of 2024.
Speaker Change: Then NIAID will conduct and fully fund a Phase I clinical trial.
Unknown Executive: On the therapeutic front, we have recently made notable advancements with our exosome platform, which is in preclinical development. We presented data at the International Society for Cell and Gene Therapy in Vancouver, where we were selected for an oral presentation. Our preclinical data highlighted an exosome-based approach for targeted delivery to skeletal muscle in the mouth lower limb. Subsequently, our research demonstrated that, using an in-house developed proprietary exogenous loading technique, we could not only target exosomes to muscle cells but also deliver antisense oligonucleotides, one of our payloads, with exosomes carrying a muscle-targeting moiety. This work was featured in a poster presentation at the International Society for Extracellular Vesicles meeting in Melbourne, Australia.
Speaker Change: On the therapeutic front, we have recently made notable advancements with our exosome platform, which is in preclinical development.
Unknown Executive: The data from both conferences strongly suggest that accidents with targeting moieties can direct delivery to skeletal muscle and the mouth lower limb, presenting potential for targeted delivery applications, including the treatment of Duchenne muscle atrophy. When combined with earlier data on Arginase 1 deficiency, which is being developed as a potential enzyme replacement therapy. These findings reinforce the potential of our platform. We believe there are significant opportunities for broad applications, and our data from our SCELFx program continues to support this.
Speaker Change: We presented data at the International Society for Cell and Gene Therapy in Vancouver, where we were selected for an oral presentation. Our preclinical data highlighted an exosome-based approach for targeted delivery to skeletal muscle in the mouse lower limb.
Speaker Change: Subsequently, our research demonstrated that using an in-house developed proprietary exogenous loading technique
Speaker Change: We can not only target exosomes to muscle cells, but also deliver antisense oligonucleotides, one of our payloads, with exosomes carrying a muscle-targeting moiety.
Speaker Change: This work was featured in a poster presentation at the International Society for Extracellular Vesicles in Melbourne, Australia.
Speaker Change: The data from both conferences strongly suggest that accidents with targeting moieties can direct delivery to skeletal muscle and the mouse lower limb, presenting potential for targeted delivery applications, including the treatment of Duchenne muscular dystrophy.
Speaker Change: Combined with earlier data on Arginase 1 deficiency, which is being developed as a potential enzyme replacement therapy.
Unknown Executive: These findings reinforce the potential of our platform, based on our significant progress over the last quarter and now a potential expedited path to BLA. This clarity gives us confidence that we're well-positioned to bring additional capital into Capricor, to strengthen our balance sheet and extend our cash flow. While there are various ways to accomplish this goal, our primary mission is to secure capital in the best interest of our shareholders. I am very pleased to inform you that we are in late stage discussions about these rights, and we will share updates as they become available. We believe that we can achieve these milestones, details of which will be shared as they become available.
Speaker Change: These findings reinforce the potential of our platform. We believe there are significant opportunities for broad applications, and our data from our SkelpX program continues to support this belief.
Unknown Executive: Now I would like to take a few moments to provide our current corporate updates and priorities as we progress through 2024, based on our significant progress over the last quarter and now a potential expedited path to BLA. This clarity gives us confidence that we're well-positioned to bring additional capital into Capricor, to strengthen our balance sheet and extend our cash flow. While there are various ways to accomplish this goal, our primary mission is to secure capital in the best interest of our shareholders.
Speaker Change: Now I would like to take a few moments to provide our current corporate updates and priorities as we progress through 2024.
Speaker Change: Based on our significant progress over the last quarter, and now a potential expedited path to BLA, this clarity gives us confidence that we are well positioned to bring additional capital into Capricor to strengthen our balance sheet and extend our cash runway.
Speaker Change: While there are various ways to accomplish this goal, our primary mission is to secure capital in the best interest of our shareholders.
Unknown Executive: Therefore, we are pursuing multiple different business opportunities, business development opportunities, and we are in active discussions with several parties looking to potentially distribute DERMA IFL in Europe for the treatment of DMG. We have been judicious with respect to partnering for the rights in the EMA as we knew the asset would garner greater value as we progressed through clinical development and achieved clarity with. I am very pleased to inform you that we are in late stage discussions for these rights, and we will share updates as they become available.
Speaker Change: Therefore, we are pursuing multiple different business development opportunities and we are in active discussions with several parties looking to potentially distribute DERMA-ISL in Europe for the treatment of DMG.
Speaker Change: We have been judicious with respect to partnering for the rights in the EMA as we knew the asset would garner greater value as we progressed through clinical development and achieved clarity with FDA.
Speaker Change: I am very pleased to inform you that we are in late stage discussions for these rights and we will share updates as they become available.
Unknown Executive: We believe that Daramaisel is a highly valuable asset in the EU, as well as other regions around the world, and our partnering efforts remain focused on securing these relationships. Additionally, our U.S. agreement with Nippon Shinyaku, as I mentioned a moment ago, has approximately $700 million in potential milestone payments payable to Capricor. $90 million of which is up to the time of approval and is triggered upon certain regulatory-based achievements, some of them to be expected in the near term.
Speaker Change: We believe that Deromysel is a highly valuable asset in the EU, as well as other regions around the world, and our partnering efforts remain focused on securing these relationships.
Speaker Change: Additionally, our U.S. agreement with Nippon Shinyaku, as I mentioned a moment ago, has approximately $700 million in potential milestone payments payable to Capricor.
Speaker Change: $90 million of which is up to the time of approval and are triggered upon certain regulatory-based achievements.
Unknown Executive: We believe that we can achieve these milestones, as they will continue to support our balance sheet and further extend our cash flow. Furthermore, if we receive approval for deromysel, we will be eligible to receive a Priority Review Voucher, a PRV, based on our pediatric disease designation, to which we retain full right. And finally, we were pleased to announce earlier this quarter that we have been added to the Russell 2000 and Russell 3000, which should bring added visibility to our company as we continue to remain focused on bringing more institutional buy and sell side support to our stores. In conclusion, this has been an extraordinary year for Capricor thus far.
Speaker Change: Some of them to be expected in the near term.
Speaker Change: We believe that we can achieve these milestones.
Speaker Change: As they will continue to support our balance sheet and further extend our cash runway.
Speaker Change: Furthermore, if we receive approval for deromysel, we will be eligible to receive a Priority Review Voucher, a PRV, based on our pediatric disease designation, to which we retain full rights.
Speaker Change: And finally, we were pleased to announce earlier this quarter to be added to the RUSLE 2000 and RUSLE 3000 indices.
Speaker Change: We should bring added visibility to our company as we continue to remain focused on bringing more institutional buy and sell side support to our story.
Speaker Change: In conclusion, this has been an extraordinary year for Capricar thus far. We have made significant strides in our regulatory pathway, getting closer to potential approval with each FDA meeting.
Unknown Executive: We have made significant strides in our regulatory pathway, getting closer to potential approval with each FDA meeting. Patients and families are fully supportive of our efforts and see the lasting power of DERMAiFEL in helping those with DMD to feel and function better. We have built, equipped, and are now producing doses from our manufacturing facility in San Diego for a fraction of the cost of other biotech companies. We have some of the strongest and most consistent data in terms of efficacy and safety in the DMD community, with the HOPE-2 and the HOPE-2 Open Label Extension data.
Speaker Change: Patients and families are fully supportive of our efforts and see the lasting power of DERMAiFEL in helping those with GMD to feel and function better.
Speaker Change: We have built, equipped, and are now producing doses from our manufacturing facility in San Diego for a fraction of the cost of other biotechnology companies.
Speaker Change: We have some of the strongest and most consistent data in terms of efficacy and safety in the DMC community with the HOPE-2 and the HOPE-2 Open Label Extension datasets.
Unknown Executive: We have a strong pipeline in our engineered exosome technology, supported by StealthX. Capricor's goal is to continue to meet its milestones and deliverables as we have set forth. As we continue to focus our efforts on bringing Garamaya Salve towards potential commercialization, and we are investing judiciously across the organization to prepare for that endeavor. Over the next several months, we will be presenting at various medical, scientific, and investor-related conferences. Details of which will be shared as they become available.
Speaker Change: We have a strong pipeline in our engineered exosome technology, supported by Stealth-X.
Speaker Change: Capricor's goal is to continue to meet its milestones and deliverables as we have set forth, as we continue to focus our efforts on bringing Garamayasal towards potential commercialization and are investing judiciously across the organization to prepare for that endeavor.
Speaker Change: Over the next several months, we will be presenting at various medical, scientific, and investor-related conferences.
Unknown Executive: And, of course, finally, I want to thank the patients, their families, and all of our investors for their continued support. Later this quarter, we plan to announce further details from our pre-BLA meeting, at which time we will be able to articulate more formal guidance on the upcoming pivotal milestones of our DMD program. I will now turn the call over to AJ to run through our finances.
Speaker Change: Details of which will be shared as they become available.
Speaker Change: And of course, finally, I want to thank the patients, their families, and all of our investors for their continued support.
Speaker Change: Later this quarter, we plan to announce further details from our pre-BLA meeting, at which time we will be able to articulate more formal guidance on the upcoming pivotal milestones of our DMD program.
Speaker Change: I will now turn the call over to AJ to run through our financials.
Unknown Executive: This afternoon's press release provided a summary of our second quarter 2024 financials on a gap 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.
AJ Bergmann: Thanks, Linda.
AJ Bergmann: This afternoon's press release provided a summary of our second quarter 2024 financials on a gap 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.
AJ: Let me start with our cash position on June 30th, 2024. Companies' cash, cash equivalents, and marketable securities totaled approximately 29.5 million compared to approximately 39.5 million on December 31st, 2023. Based on our current operating plan and projections, our cash runway is expected to be sufficient to support operations into the first quarter of 2025. This expectation excludes any potential additional milestone payments under our commercialization and distribution agreements with Nibon Shinyako. Turning now to the financials, revenue for the second quarter of 2024 was approximately $4 million compared with approximately $3.9 million for the second quarter of 2023.
Speaker Change: Let me start with our cash position.
Speaker Change: june tieth two thousand and twenty four companies cash cash equivalent to marketable securities total approximately twenty nine point five million compared to approximatelythirtynine point five million on december thirty first twothousand andtwenty three
Speaker Change: Based on our current operating plan and projections, the company's cash runway is expected to be sufficient to support operations into the first quarter of 2025. This expectation excludes any potential additional milestone payments under our commercialization and distribution agreements with Nippon Shinyaku.
AJ: The source of revenue is the ratable recognition of the $40 million we've received from our U.S. exclusive commercialization and distribution agreement with Nippon Shinya. Moving to our operating expenses for the second quarter of 2024, excluding stock-based compensation, our R&D expense was approximately $11.7 million, compared to approximately $8.4 million in Q2 2023. The increase in expenses of $3.3 million was primarily due to increased clinical and manufacturing costs associated with our Duchenne muscular dystrophy program.
Unknown Executive: revenue for the second quarter of 2024 was approximately $4 million compared with approximately $3.9 million for the second quarter of 2023. The source of revenue is the routable recognition of the $40 million we have received from our U.S. exclusive commercialization and distribution agreement with Nippon Shinya.
Speaker Change: Turning now to the financials, revenue.
Speaker Change: for the second quarter of 2024 were approximately $4 million compared with approximately $3.9 million for the second quarter of 2023. The source of revenue is the ratable recognition of the $40 million we have received from our U.S. exclusive Commercialization and Distribution Agreement with Nippon Shinyaku.
Speaker Change: Moving to our operating expenses for the second quarter of 2024, excluding stock-based compensation, our R&D expense was approximately $11.7 million, compared to approximately $8.4 million in Q2 2023.
Speaker Change: The increase in expenses of $3.3 million was primarily due to increased clinical and manufacturing costs associated with our Duchenne muscular dystrophy program.
AJ: Again, excluding stock-based compensation, our general and administrative expense was approximately $1.8 million in Q2 2024 and approximately $1.7 million in Q2 2023. The net loss for the second quarter of 2024 was approximately $11 million, compared to a net loss of approximately $7.4 million for the second quarter of 2023. And the net loss for the first half of 2024 was approximately $20.8 million, compared to a net loss of approximately $15.1 million for the first half of 2023. We will now open the line up for questions.
Speaker Change: Again, excluding stock-based compensation, our general and administrative expense was approximately $1.8 million.
Speaker Change: in Q2 2024 and approximately 1.7 million in Q2 2023.
Speaker Change: Net loss for the second quarter of 2024 was approximately $11 million, compared to a net loss of approximately $7.4 million for the second quarter of 2023.
Speaker Change: And net loss for the first half of 2024 was approximately $20.8 million, compared to a net loss of approximately $15.1 million for the first half of 2023. We will now open the line up for questions.
Operator: Thank you. Ladies and gentlemen, if you do have a question, please press star followed by one on your touchtone phone; you will then hear a prompt that your hand has been raised. And if you wish to withdraw from the polling questions, please press star followed by two. And if you're using a speakerphone, we do ask that you please lift the handset before pressing any keys. Please go ahead and press star one now if you do have a question.
Operator: Thank you. Ladies and gentlemen, if you do have a question, please press star followed by one on your touch-tone phone. You will then hear a prompt that your hand has been raised. And if you wish to withdraw from the polling questions, please press star followed by two. And if you're using a speakerphone, we do ask that you please lift the handset before pressing any keys. Please go ahead and press star one now if you do have a question. And your first question will be from Joseph Pantginis at HC Wainwright. Please go ahead.
Speaker Change: butnot
Speaker Change: Thank you. Ladies and gentlemen, if you do have a question, please press star followed by one on your touchtone phone.
Speaker Change: You will then hear a prompt that your hand has been raised.
Speaker Change: And if you wish to withdraw from the polling questions, please press star followed by 2. And if you're using a speakerphone, we do ask that you please lift a handset before pressing any keys. Please go ahead and press star 1 now if you do have a question.
Joseph Pantinis: And your first question will be from Joseph Pantinis at HC Wainwright. Please go ahead.
Joseph Pantginis: Hi, good afternoon, Linda and AJ, and thanks for taking the questions. Two questions, if you don't mind. So, Linda, in your prepared comments, you talked about you just met with the FDA, and it sounds like the key aspects were, you know, deciding on the two alternate paths for filing. I guess I wanted to get the latest update, and, of course, I know this is before you get the minutes, but to what extent did the cardiovascular play into this latest meeting, and what do you consider the key outstanding questions or issues still?
Unknown Attendee: Hi, good afternoon, Linda and AJ, and thanks for taking the questions. Two questions, if you don't mind. So, Linda, in your prepared comments, you talked about you just met with the FDA, and it sounds like the key aspects were, you know, deciding on the two alternate paths for filing. I guess I wanted to get the latest update, and, of course, I know this is before you get the minutes, but to what extent did the cardiovascular play into this latest meeting, and what do you consider the key outstanding questions or issues still?
Speaker Change: Hi, good afternoon, Linda and AJ, and thanks for taking the question.
Joseph Pantinis: Two questions, if you don't mind. So Linda, in your prepared comments, you're talking about you just met with the FDA and it sounds like the key aspects were, you know, deciding on the two alternate paths for filing. I guess I wanted to get the latest update. And of course, I know this is before you get the minutes, is, you know, to what extent did the cardiovascular play into this latest meeting? And what do you consider the key outstanding questions or issues still?
Linda Marbn: Yeah, Joe, thank you. And thank you for the support over these many years. I really think we're going to get this across the line, and I'm super excited.
Linda Marbn: Yeah, Joe, thank you. Thank you for the support over these many years. I really think we're going to get this across the line.
Linda Marbn: The cardiovascular aspect of the program is finally getting the attention that it deserves from the FDA. I think it's really due to the combination of efforts of the KOLs and the Duchenne advocacy community, which have highlighted that this is the number one cause of death in these boys and young men. And yet, frankly, all the therapies that are available to them are ones that might go to, you know, some person who has adult-based cardiac disease would be kind of a joke, you know, something your grandma might get.
Speaker Change: And I'm super excited. The cardiovascular aspect of the program is finally getting the attention that it deserves from the FDA.
Speaker Change: I think it's really due to the combination of efforts of the KOLs and the Duchenne Advocacy Community that have highlighted that this is the number one cause of death in these boys and young men, and yet, frankly, all the therapies that are available to them are ones that might go to, you know, some person who has adult-based cardiac disease, the kind of joke, you know, something your grandma might get.
Linda Marbn: This would be the first therapeutic to treat the cardiomyopathy associated with Duchenne muscular dystrophy, whose pathogenesis is extraordinarily different from others. So, yes, it has become a key focus of the program. They are willing to consider daramia cell as part of the label for cardiac. They are also, at least in writing thus far, willing to consider expanding the label to all patients diagnosed with Duchenne muscular dystrophy. And so we're really excited to see the results of these minutes and continue to push this towards BLA very quickly.
Joseph Pantginis: That's very helpful. Thanks.
Speaker Change: This would be the first therapeutic to treat a cardiomyopathy disease.
Speaker Change: Associated with Duchenne Muscular Dystrophy, which pathogenesis is extraordinarily different from others.
Speaker Change: So, yes, it has become a key focus of the program. They are willing to consider daramysel as part of the label for cardiac. They are also, at least in writing thus far, willing to consider expanding the label to all patients diagnosed with Duchenne muscular dystrophy. And so we're really excited to see the results of these minutes and continue to push this towards BLA very quickly.
Linda Marbn: And if I could just switch swiftly to the Exosomes Program. Obviously, it's a platform that has broad-reaching capabilities. So you mentioned your BD efforts. How would you describe the proportion of, say, inbound versus outbound, you know, reach with regard to BD?
Speaker Change: That's very helpful. Thanks. And if I could just switch quickly, swiftly, excuse me, to the exosomes program. Obviously, it's a platform that has broad-reaching capabilities. So you mentioned your BD efforts, I guess. How would you describe sort of the the proportion of say inbound versus outbound
Linda Marbn: Yeah, so, you know, the exosome program is really coming to light. I think you, and others that follow our story, know that my goal is to launch DERM-ISL and expand that into other indications as appropriate.
Speaker Change: You know, reaching with regard to BD.
Speaker Change: Yeah, so, you know, the Existome program is really coming to light, I think.
Speaker Change: You know, and others that follow our story know, that my goal is to launch JeremiahCell and expand that into other indications as appropriate. But because of our long experience with cell therapy and making cells and using cells, expanding into an exosome-based program was critical. The first thing that we had to do was make sure that we could make exosomes in large numbers easily and relatively cheaply because their main competitor would be a lipid nanoparticle. So exosomes are way better than lipid nanoparticles because you can target them and load them and so there's a lot more opportunities, plus they're very safe, they're not clogging up your liver.
Linda Marbn: But because of our long experience with cell therapy and making cells and using cells, expanding into an exosome-based program was critical. The first thing that we had to do was make sure that we could make exosomes in large numbers easily and relatively cheaply because their main competitor would be a lipid nanoparticle. Now exosomes are way better than lipid nanoparticles because you can target them and load them, and so there's a lot more opportunities. Plus, they're very safe; they're not clogging up your liver.
Linda Marbn: So that was our first goal. We achieved that. I know people thought we were quiet for a long time.
Linda Marbn: Now that we're getting out and about with some of these opportunities, including potentially loading, for instance, a Richen-based antisense and exon skipper into the exosome, targeting it to skeletal muscle using our targeting moiety, this becomes a very tangible product. And so, yes, we have more incoming interest than we've had before. We're also planning in 2025 a pretty extensive business development external outreach effort.
Speaker Change: So, that was our first goal. We achieved that. I know people thought we were quiet for a long time. Now that we're getting out and about with some of these opportunities, including potentially loading, for instance, a Richette-based antisense and exon skipper into the exosome, targeting it to skeletal muscle using our targeting moiety, this becomes now a very tangible product. And so, yes, we have more incoming interest than we've had before. We're also planning in 2025 a pretty extensive business development external reaching effort.
Joseph Pantginis: Thank you very much for the color, Linda.
Linda Marbn: Absolutely, Joe. I look forward to seeing you soon.
Speaker Change: Thank you very much for the color, Linda.
Leland Gershell: Thank you. The next question will be from Leland Gershell at Oppenheimer. Please go ahead.
Speaker Change: Absolutely Joe, look forward to seeing you soon.
Speaker Change: Thank you. Next question will be from Leland Gershel at Oppenheimer. Please go ahead.
Leland Gershell: Hi, thanks for taking the questions. Just looking to get some clarity on the timelines here. You mentioned the pre-VLA meeting and plans that will be coming up around the data and thereafter. I wanted to understand, have you begun any aspects of the rolling submission? If not, when do you expect to begin that? And how long after the top-line data from the HOPE-3 study do you expect to complete the rolling VLA submission?
Leland Gershel: Yeah, hi, thanks for taking the questions. Just looking to get some clarity on the timelines here. Linda, you mentioned the, you know, pre-BLA meeting and...
Leland Gershel: I wanted to understand, have you begun any aspects of the rolling submission, if not when do you expect to begin that, and how long after the top line data from the HOPE-3 study do you expect to complete the rolling BLA submission?
Unknown Executive: Yeah, so our current plan is to begin submitting the rolling BLA within the next 60 days. FDA has seen our timelines and has approved it as part of our communication plan in our most recent meeting. Should the HOPE-3 data be necessary for the BLA, we would anticipate that the submission would be complete at the end of the first quarter. We are also exploring, however, with the agency, the opportunity to complete the file without the HOPE-3 data, and then that would obviously accelerate submission to the end of 2020.
Speaker Change: Yeah, so our current plan is to begin submitting in the rolling BLA within the next 60 days. FGA has seen our timelines and have approved it as part of our communication plan in our most recent meeting.
Speaker Change: Should the HOPE-3 data be necessary for the VLA, we would anticipate that the submission would be complete at the end of the first quarter. We are also exploring, however, with the agency, the opportunity to complete the file without the HOPE-3 data, and then that would obviously accelerate submission to the end of 2024.
Leland Gershell: Okay, thank you. And then a question on the potential XUS partnership. You know, wondering if there's any, you know, based on where you may be in your discussions, if you can make any comment as to the view of having one or more partnerships in place by your end, to sort of want to understand kind of what the evolution of those discussions is like. Yeah, our goal
Speaker Change: okay thank you and then a question on on the x two fidentential partnership you know you wondering if if there's any even basing where you may be in your discussion that you can make any comment as to
Speaker Change: The view of having one or more partnerships in place by year end to sort of want to understand kind of what the evolution of those discussions are like at this time.
Unknown Executive: By year end, we sort of want to understand kind of what the evolution of those discussions is like. Yeah, our goal
Unknown Executive: Yeah, our goal is to finalize those conversations before the end of this year. You know, as I said, and I know people have sort of wondered, we've been talking to EU partners for a while. The clarity with FDA has been critical, and also by beginning conversations with regulatory authorities and consultants in Europe, we now have a much clearer picture of what it's going to take to get it across the line there.
Speaker Change: Yeah, our goal is to finalize those conversations before the end of this year.
Speaker Change: You know, as I said, you know, and I know people have sort of wondered, you know, we've been talking to EU partners for a while.
Speaker Change: The clarity with FDA has been critical and also beginning conversations with regulatory authorities and consultants in Europe , we now have a much cleaner picture of what it's going to take to get it across the line there.
Unknown Executive: We think it's going to be much easier than we originally anticipated. Our partners think so also. So the goal would be to close these deals before the end of this calendar year to strengthen the balance sheet and to support the approval and expansion for DERMA ISL. Thanks. Yeah, great talking to you always, Leland.
Speaker Change: We think it's going to be actually much easier than we originally anticipated, our partners think so also. So the goal would be to close these deals, a deal, before the end of this calendar year to strengthen the balance sheet and to support the approval and expansion for DERMA ISL.
Unknown Attendee: Yeah, great talking to you always, Leland.
Speaker Change: Thank you.
Aydin Huseynov: Thank you. The next question will be from Aydin Huseynov at Leidenberg. Please go ahead.
Speaker Change: Yeah, great talking to you always, Leland.
Speaker Change: Thank you. Next question will be from Aydin Huseynov at Leidenberg. Please go ahead.
Aydin Huseynov: Hi, good afternoon, everyone. Congratulations on the progress.
Aidan Yusoff: All right, good afternoon everyone. Congrats with the progress.
Aydin Huseynov: Positive AFDA Meeting, Advanced EU Discussions, and final I&N name for CAP-10 Autority in the MISO. So I have a couple of questions regarding the FDA meeting. You mentioned that you had some discussions about expanding the indication, and I was just curious to see if there's any preliminary feedback or reaction from FDA to potential Baker dystrophy expansion and any thoughts that they have, and a hypothetical BMD trial design.
Aidan Yusoff: forthe positive d meeting turn to you discussions and fin it ison name for captain auto myself so a couple of questions regard it to be of the meeting so you mentioned that you had some discussions about
Aidan Yusoff: Expansion of the Indication, and I was just curious to see if there's any preliminary feedback or reaction of FDA to potential Baker dystrophy expansion and any thoughts that they have, and a hypothetical BMD trial design.
Linda Marbn: Yeah, Aydin, you know, thank you so much for the questions. I know you've been a really big supporter of us expanding into BMD for a while, so thank you for that guidance.
Aidan Yusoff: Yeah, Aydin, you know, thank you so much for the questions. I know you've been really a big supporter of us expanding into BMD for a while, so thank you for that guidance.
Linda Marbn: We have mentioned it as part of our conversations with FDA, but obviously, it hasn't been a main focus of the meeting. The really exciting part for us right now is that as FDA is beginning to understand the impact of cardiomyopathies in these muscular dystrophies, it really gives us a window into how we can move forward. And, for instance, Becker, where cardiomyopathy is also the leading cause of death. We haven't really talked about trial design yet because I want to get through this stage with FDA, see where it comes down in terms of cardiac implications, and then strategically build a program.
Speaker Change: We have mentioned it as part of our conversations with FDA. Obviously, it hasn't been a main focus on the meeting. The really exciting part for us right now is that as FDA is beginning to understand the impact of cardiomyopathies.
Aidan Yusoff: In these muscular dystrophies, it really gives us a window into how we can move forward and for instance, Becker, where the cardiomyopathy is also the leading cause of death.
Aidan Yusoff: We haven't really talked about trial design yet because I want to get through this stage with FDA.
Aidan Yusoff: See where it comes down in cardiac implications and then strategically build a program. We plan to meet with FDA on BMD before the end of the year so that we can get their guidance and we're putting together sort of our KOL panels and plans as we speak. So yeah, this is a great opportunity, obviously, for the therapy, but also for the patients with Becker's.
Linda Marbn: We plan to meet with FDA on BMD before the end of the year so that we can get their guidance. And we're putting together sort of our KOL panels and plans as we speak. So yeah, this is a great opportunity, obviously, for the therapy but also for the patients with Becker's.
Aydin Huseynov: And for potential DMD labels for dermyosel, we should think about the FDA label. Do you think it would be a pretty broad label that would include, you know, DMD patients, similar to the ones you recruited in the hope of and hope for the trial? Or do you think the FDA may sort of specify patients with cardiomyopathy only? And how do you think the FDA will approach this?
Speaker Change: Thank you. This is helpful. And for potential DMD label for their myosel,
Speaker Change: We should think about the FDA label. Do you think it it would be a pretty broad label that would include, you know,
Speaker Change: D&D patients similar to the ones you recruited in the HOPE-2 and HOPE-3 trial, or do you think the FDA may sort of specify patients with cardiomyopathy only, and how do you think the FDA will approach this?
Linda Marbn: Yeah, so the cardiomyopathy is going to be an andor, right? So that's how we've positioned it.
Unknown Executive: Yeah, so cardiomyopathy is going to be an and-or, right? So that's how we positioned it.
Unknown Executive: I think that's how the label will read, you know, it'll be for those with skeletal muscle and cardiac myopathy or, you know, or skeletal muscle or cardiomyopathy, because, as you know, and it has been well published, the disease does not progress similarly, both in cardiac and skeletal muscles. So some kid might have a bad heart and good skeletal muscle, or vice versa. So there will be an opportunity for the stabilization of disease, both in the skeletal and cardiac.
Speaker Change: Yeah, so the cardiomyopathy is going to be an and or, right? So that's how we positioned it. I think that's how the label will read, you know, it'll be for those with skeletal muscle and cardiac myopathy, or, you know, or skeletal muscle or cardiomyopathy, because as you know, and has been well published, the disease is not
Speaker Change: progressed similarly both in cardiac and skeletal muscle. So some kid might have a bad heart and good skeletal muscle or vice versa. So we will be an opportunity for stabilization of disease both in skeletal and cardiac.
Unknown Executive: In terms of labeling, our goal is all those diagnosed with Duchenne muscular dystrophy. Our preliminary feedback from FDA suggests that they are open to that opportunity. And we will certainly push that as hard as we can. Certainly, the precedents that have been set by, you know, some of the others that have gotten approval in the space with very broad labels give us a door open to that opportunity as well, which could double our market opportunity at launch.
Speaker Change: In terms of labeling, our goal is all those diagnosed with Duchenne muscular dystrophy. Our preliminary feedback from FDA suggests that they are open to that opportunity, and we will certainly push that as hard as we can. Thank you.
Speaker Change: Presidents that have been set by, you know, some of the others that have gotten approval in the space with very broad labels gives us a door open into that opportunity as well, which could double our market opportunity at launch.
Unknown Attendee: All right, right. It makes sense. It makes sense.
Aydin Huseynov: All right, right. It makes sense. It makes sense.
Speaker Change: Right, right. Makes sense. Makes sense. And regarding to the...
Unknown Executive: And regarding the sort of advanced discussions regarding partnerships in Europe, would you like to pursue a partnership deal before the top line readout in the fourth quarter 2024 or after the readout? So this is purely an economic question. Is it cash now or more cash later?
Aydin Huseynov: And regarding the sort of advanced discussions regarding partnerships in Europe, would you like to pursue a partnership deal before the top line readout in the fourth quarter 2024 or after the readout? So this is purely an economic question. Is it cash now or more cash later?
Speaker Change: sort of advanced discussions regarding partnerships in Europe . So would you like to pursue a partnership deal before the top line readout in the fourth quarter 2024 or after the readout? So this is purely economic question. Is it cash now or more cash later?
Linda Marbn: Well, you know, it's an interesting question. We're talking to partners right now, and we feel that the offers that are coming our way are reasonable and appropriate, almost assuming the approval of the BLA. So, you know, Europe has another regulatory structure. We're very confident now, based on some of our conversations with authorities over there, that we will meet their criteria, either with a small clinical trial or no clinical trial. We're not sure yet.
Speaker Change: Well, you know, it's, you know, an interesting question. We're talking to partners right now, we feel that the offers that are coming our way are reasonable and appropriate, almost assuming approval of the VLA. So, you know, Europe has another regulatory structure. We're very confident now based on some of our conversations with authorities over there, that we will meet their criteria either with
Linda Marbn: And so I guess, you know, the short answer to your question is we'll close the deal when we reach the best economics to support our balance sheet to drive the company forward and also to give credit to the asset as it is.
Speaker Change: Small clinical trial or no clinical trial, we're not sure yet. And so I guess, you know, the short answer to your question is we'll close the deal when we reach the best economics to support our balance sheet to drive the company forward and also to give credit to the asset as it is due.
Aydin Huseynov: And the last question I have is regarding the potential milestones from Nippon Shinyako. So we know there's $90 million awaiting until BLA approval, but should we expect anything before AFDA approval? Should we expect anything based on the top-line readout at the end of this year?
Speaker Change: vus
Speaker Change: And the last question I have is regarding...
Speaker Change: The potential milestones from Nippon Shinyako. So we know there's 90 million are waiting up until BLA approval.
Speaker Change: But should we expect anything before the AFDA approval? Should we expect anything based on the top-line readout at the end of this year?
Linda Marbn: So, as you know, we're not able to disclose the actual cadence of the milestone payments. We built them into our personal balance sheet estimations, and so, you know, we're aware of those milestones coming in, and that's really all I can say right now, but there is $90 million coming up to and including approval of the BLA.
Unknown Executive: So, as you know, we're not able to disclose the actual cadence of the milestone payments. We built them into our personal balance sheet estimations, and so, you know, we're aware of those milestones coming in, and that's really all I can say right now, but there is $90 million coming up to and including approval of the BLA.
Speaker Change: So as you know we're not able to disclose the actual cadence of the milestone payments. We've built them into our personal balance sheet estimations and so you know we're aware of those milestones coming in and that's really all I can say right now but there's 90 million coming up to and including approval of the BLA.
Aydin Huseynov: All right, thank you so much for taking questions and congratulations on the program.
Speaker Change: Got it, understood. All right, thank you so much for taking questions and congrats for the progress.
Linda Marbn: Thank you. I look forward to seeing you soon.
Operator: Thank you. Ladies and gentlemen, as a reminder, if you would like to ask a question, please press star followed by one on your touchtone phone.
Operator: Thank you. Ladies and gentlemen, as a reminder, if you would like to ask a question, please press star followed by one on your touchtone phone. And your next question will be from Kristen Kluska at Cantor. Please go ahead.
Speaker Change: Thank you. I look forward to seeing you soon.
Speaker Change: Thank you. Ladies and gentlemen, as a reminder, if you would like to ask a question, please press star followed by one on your touchtone phone.
Speaker Change: And your next question will be from Kristen Kluska at Kantor. Please go ahead.
Rick Miller: Hello, this is Rick Miller on behalf of Kristen. Thanks for taking our questions. We've got two for you here.
Unknown Attendee: Hello, this is Rick Miller on behalf of Kristen. Thanks for taking our questions. We've got two for you here.
Rick Miller: Hello, this is Rick Miller on for Kristen. Thanks for taking our questions. We've got two for you here. What are you hearing from physicians and caregivers about the potential for non-ambulant DMD patients now receiving gene therapy on an accelerated basis? How confident is the space in this, especially as it relates to these cardiac issues that you were talking about?
Rick Miller: What are you hearing from physicians and caregivers about the potential for non-ambulant DMD patients now receiving gene therapy on an accelerated basis? How confident is the space in this, especially as it relates to these cardiac issues that you were talking about?
Unknown Attendee: What are you hearing from physicians and caregivers about the potential for non-ambulant DMD patients now receiving gene therapy on an accelerated basis? How confident is the space in this, especially as it relates to these cardiac issues that you were talking about?
Unknown Executive: Yeah, really great question. So, yeah, I mean, everybody is laser focused on the implications of gene therapy for the non-ambulance and whether A, it will actually work, you know, are you knocking on a closed door, dead muscle is a dead muscle, or are you going to really, you know, preserve what's there? And I certainly hope for those guys with DMD, it preserves what's there. We actually presented to FDA a comparison of daramia cell and elevitis and its pathogenesis, or delay in the pathogenesis of the disease.
Unknown Executive: Yeah, really great question. So, yeah, I mean, everybody is laser focused on the implications of gene therapy for the non-ambulance and whether A, it will actually work, you know, are you knocking on a closed door, dead muscle is a dead muscle, or are you going to really, you know, preserve what's there? And I certainly hope for those guys with DMD, it preserves what's there. We actually presented to FDA a comparison of daramycel and alevitis and its pathogenesis, or delay in the pathogenesis of the disease.
Speaker Change: Yeah, really great question.
Speaker Change: Yeah, I mean, everybody is laser focused on the implications of gene therapy for the non-ambulance and whether, A, it will actually work, you know, are you knocking on a closed door, dead muscle is a dead muscle, or are you going to really, you know, preserve what's there? And I certainly hope for those guys with DMD, it preserves what's there. We actually presented to FDA a comparison of daramysal and alevitis and its pathogenesis.
Speaker Change: or delay in the pathogenesis of the disease, and we were able to show that
Unknown Executive: And we were able to show that Deremia Cell performs at least as well as gene therapy in year one and better than gene therapy in year two when compared with non-ambulant patients. However, cardiomyopathy becomes a real problem with post-gene therapy patients for multiple reasons. One, physicians are concerned about potential inflammatory processes as part of the gene therapy, and could that have negative implications on the heart? Two, if these guys are more active, is that going to put more strain on their hearts, and will their hearts fail faster?
Speaker Change: Derramycel performs at least as well as gene therapy in year one and better than gene therapy in year two when compared with non-ambulant patients.
Unknown Executive: And we were able to show that cardiomyopathy becomes a real problem with post-gene therapy patients for multiple reasons. One, physicians are concerned about potential inflammatory processes as part of the gene therapy, and could that have negative implications on the heart? Two, if these guys are more active, is that going to put more strain on their hearts, and will their hearts heal faster?
Speaker Change: The cardiomyopathy becomes a real problem with post-gene therapy patients for multiple reasons. One, physicians are concerned about potential inflammatory processes as part of the gene therapy and could that have negative implications on the heart? Two, if these guys are more active, is that going to put more strain on their hearts and will their hearts fail faster? And three, there seems to be, you know, sort of a correlation between gene therapy and some worsening of the cardiomyopathy. So I think this is a great opportunity for dermiocel. We have shown pre-clinically that there is no negative impact of dermiocel treatment in conjunction with microdystrophin treatment. And so we believe that they'll be great partners together. Let the gene therapy restore the muscle and let dermiocel protect it.
Unknown Executive: And three, there seems to be, you know, sort of a correlation between gene therapy and some worsening of the cardiomyopathy. So I think this is a great opportunity for DERAMYCEL. We have shown pre-clinically that there is no negative impact of DERAMYCEL treatment in conjunction with microdystrophin treatment, and so we believe that they'll be great partners together. Let the gene therapy restore the muscle, and let DERAMYCEL protect it.
Rick Miller: And three, there seems to be, you know, sort of a correlation between gene therapy and some worsening of the cardiomyopathy. So I think this is a great opportunity for DaramayaCell. We have shown pre-clinically that there is no negative impact of DaramayaCell treatment in conjunction with microdystrophin treatment, and so we believe that they'll be great partners together. Let the gene therapy restore the muscle, and let DaramayaCell protect it.
Unknown Attendee: Okay, maybe just one more question then from us. Switching to Becker, can you help us kind of frame the cardiomyopathy opportunity that you're thinking about here? I think sometimes the field thinks sort of naively about Becker as being a less severe form of DMV. So how does the kind of cardiac burden and Becker break down as it relates to DMV? Is it sort of more proportional or more severe?
Unknown Executive: Okay, maybe just one more question then from us. Switching to Becker, can you help us kind of frame the cardiomyopathy opportunity that you're thinking about here? I think sometimes the field thinks sort of naively about Becker as being a less severe form of DMV. So how does the kind of cardiac burden and Becker break down as it relates to DMV? Is it sort of more proportional or more severe?
Unknown Attendee: Thoughts there?
Speaker Change: Okay, maybe just one more question then from us, switching to Becker, can you help us kind of frame the cardiomyopathy opportunity that you're thinking about here? I think sometimes the field thinks sort of naively about Becker as being a less severe form of DMV. So how does the kind of cardiac burden and Becker break down as it relates to DMV? Is it sort of more proportional or more severe?
Unknown Executive: Yeah, great question, and certainly one that the KOLs in the cardiology space are laser-focusing on. According to some of the key opinion leaders that we've talked to, we participate actively in PPMD's cardiomyopathy meetings, which they hold once a year for both Becker and Duchenne. You know, Becker cardiomyopathy actually begins at about age 14, not so dissimilar to Duchenne muscular dystrophy cardiomyopathy. However, the progression of skeletal muscle myopathy is slower. These guys end up with a pretty severe cardiomyopathy heart failure paradigm, and most of them die, you know, in their 40s and 50s as a result of heart disease.
Unknown Executive: Yeah, great question, and certainly one that the KOLs in the cardiology space are laser-focused on. According to some of the key opinion leaders that we've talked to, we participate actively in PPMD's cardiomyopathy meetings, which they hold once a year for both Becker and Duchenne. You know, Becker cardiomyopathy actually begins at about age 14, not so dissimilar to Duchenne muscular dystrophy cardiomyopathy. However, the progression of skeletal muscle myopathy is slower. These guys end up with a pretty severe cardiomyopathy heart failure paradigm, and most of them die, you know, in their 40s and 50s as a result of heart disease.
Rick Miller: Thoughts there? Unknown Speaker Yeah.
Speaker Change: Thoughts there.
Speaker Change: Yeah, great question, and certainly one that the KOL's cardiology space are laser focusing on. According to some of the key opinion leaders that we've talked to, we participate actively in PPMD's cardiomyopathy meetings, which they hold once a year for both Becker and Duchenne.
Speaker Change: You know, the Becker cardiomyopathy actually begins at about age 14, not so unsimilar.
Speaker Change: are dissimilar to the Duchenne muscular dystrophy cardiomyopathy. The progression of skeletal muscle myopathy is slower. These guys end up with a pretty severe cardiomyopathy heart failure paradigm, and most of them die, you know, 40s and 50s as a result of the heart disease.
Unknown Executive: I think now that the whole field is becoming more aware of cardiac dysfunction as a mediator of potential death, it'll open the door, as I mentioned in a question earlier, to treating the cardiomyopathy associated with Becker. And yes, you know, while it's less severe in terms of, you know, dying at 20 versus dying at 50, nobody wants to die at 50 either. So let's keep those guys going.
Unknown Executive: I think now that the whole field is becoming more aware of cardiac dysfunction as a mediator of potential death, it'll open the door, as I mentioned in a question earlier, to treating the cardiomyopathy associated with Becker. And yes, you know, while it's less severe in terms of, you know, dying at 20 versus dying at 50, nobody wants to die at 50 either. So let's keep those guys going. Okay, thank you so much.
Speaker Change: I think now that the...
Speaker Change: whole field is becoming more aware of the
Speaker Change: You know, cardiac dysfunction as a mediator of potential death.
Speaker Change: it'll open the door as i mentioned in a question earlier to treating the carding map of the associated with specker and yes you know while it's less of youryear in terms of dying at twenty versus dying a fifty nobody wants to die fifty either so i love to keep those guys go into
Speaker Change: Okay, thank you so much.
Operator: Thank you. And at this time, I would like to turn the call back over to Capricor Management for final thoughts.
Unknown Executive: Thank you. And at this time, I would like to turn the call back over to Capricor Management for final thoughts.
Speaker Change: Thank you so much.
Speaker Change: Thank you and at this time I would like to turn the call back over to Capricor Management for final thoughts.
Unknown Executive: Thank you very much for attending our earnings call today and thank you for your thoughtful questions. I look forward to updating all of you on our programs as we continue through 2024. Have a nice evening, and we'll talk to you soon.
Speaker Change: Thank you very much for attending our earnings call today and thank you for your thoughtful questions. I look forward to updating all of you on our programs as we continue through 2024. Have a nice evening and we'll talk to you soon.
Operator: Thank you. Ladies and gentlemen, this does indeed conclude the conference call for today.
Operator: Thank you. Ladies and gentlemen, this does indeed conclude your conference call for today. Once again, thank you for attending, and at this time, we do ask that you please disconnect your lines.
Speaker Change: Thank you. Ladies and gentlemen, this does indeed conclude your conference call for today. Once again, thank you for attending, and at this time we do ask that you please disconnect your lines.
Speaker Change: and uh... d hai kim hand tan pumpkin ف ت ت
Linda Marbn: I think that's how the label will read, you know, it'll be for those with skeletal muscle and cardiac myopathy or, you know, or skeletal muscle or cardiomyopathy because, as you know, and as has been well published, the disease does not progress similarly in both cardiac and skeletal muscle. So some kids might have a bad heart and good skeletal muscle or vice versa. So there will be an opportunity for stabilization of disease both in the skeletal and cardiac systems.
Linda Marbn: In terms of labeling, our goal is for all those diagnoses to be sent as muscular dystrophy. Our preliminary feedback from FDA suggests that they are open to that opportunity. And we will certainly push that as hard as we can. Certainly, the precedent that has been set by, you know, some of the others that have gone approval in the space with various broad labels gives us a door open to that opportunity as well, which could double a lot of market opportunity over the long term.