Q4 2024 Capricor Therapeutics Inc Earnings Call
Good afternoon, ladies and gentlemen, and welcome to the coffee courtyard boutiques, our second quarter 2024 earnings call.
At this time all participants lines are in listen only mode. Following the presentation. We will conduct a question and answer session. If at any time. During this call you require immediate assistance. Please press star Zero 40 operated.
Speaker Change: It's being recorded on Wednesday March 19, 2025, I would now like to turn the conference over to always CFO, Egypt, but I never forget forward looking statement. Please go ahead.
Speaker Change: Thank you and good afternoon, everyone.
Speaker Change: We start I would like to state that we will be making certain forward looking statements. During today's presentation. These statements may include statements regarding among other things the efficacy safety and intended utilization of our product candidates, our future R&D plan, including our anticipated conduct and timing of preclinical and clinical studies and enrollment of patients in clinical stuff.
Speaker Change: Our plans to present a report additional data our plans regarding regulatory filings potential regulatory developments.
Speaker Change: Our product candidate revenue and reimbursement estimates protracted terms of definitive agreement manufacturing capabilities potential milestone payments, our financial position and our possible uses of existing cash and investment resources. These.
Speaker Change: 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.
Speaker Change: Jim any obligation to update such statements with that I'll turn the call over to Linda Moore ban.
Speaker Change: Thanks, Hey, Jay Good afternoon, everyone and thank you for joining today's conference call.
Speaker Change: The mission of Capricorn has always been to discover and develop transformative drug products for patients in need and translating these biological and that extends into commercial products.
As many of you know Capricorn was founded over 20 years ago. Originally in the laboratories at Johns Hopkins University, where we isolated characterized and hardest a unique cell type derived from heart tissue.
Speaker Change: Even at that time of discovery, the cardio sphere derived cells, which are now known it there myself, where conceptualize to potentially be a powerful cellular therapeutic which one day could treat diseases of the heart for which there are many.
Speaker Change: Now, let me remind you that Darren myself has a defined mechanism of action, which is primarily immuno modular Tory and anti fibrotic driven by the release of accident after the cells aren't cute.
Our identity and potency assays, which are supported by the F. D. A R based on those properties of bio activity there.
Speaker Change: Sarah myself is not a stem cell, let me remind you. It's active ingredients is a rarefied population of cardiac cells, the capricorn isolates and expands using proprietary methods.
Speaker Change: Cells are infused in a simple one time per quarter intravenous infusion at a dose of 150 million cells.
Speaker Change: With over 700 infusions of the product over many years.
Speaker Change: From my yourselves had been shown to be a safe and effective means for attenuating disease progression.
Speaker Change: As many of you know we have been working for over eight years developing Darren myself as a treatment for those impacted by Duchenne muscular dystrophy.
Speaker Change: With great Pride and Joy that I can say, we find ourselves potentially nearing approval.
Speaker Change: Today, I will walk you through the latest regulatory.
Speaker Change: AMC and commercial updates as Capricorn begins to transition from a translational medicines company, but the casually commercial stage company.
Speaker Change: We will continue to stay committed to our mission developing transformational treatments for patients in need and driving value for our shareholders.
Speaker Change: Now turning first to our biologics license application or.
Speaker Change: Our BLA filing for D. M D cardiomyopathy, which we announced in early March was accepted by FDA for priority review.
Speaker Change: The application is seeking full not accelerated approval are very myself to treat DMD cardiomyopathy and aspect of the disease, the not only afflicts essentially all DMD patients.
Speaker Change: But also as the leading cause of mortality associated with the M D.
Speaker Change: As we look ahead to our producer date set for August 31 2025.
Speaker Change: We're working with the FDA as they are actively reviewing our application.
Speaker Change: At this time, the FDA has not indicated to us whether an AD com will be necessary, but we are preparing for what should that be needed and I'm pleased to inform you that would have officially scheduled or P. L. I, our pre licensing inspection.
Speaker Change: Our manufacturing facility, which is set for the second quarter of this year.
Speaker Change: Our BLA is supported with data from two trials are phase two hope to placebo controlled trial and our hope to open label extension trial compared to patient level natural history data from the D. M D. Cardiac consortium led by Dr. Jonathan <unk>.
Speaker Change: Flow at Vanderbilt University.
Speaker Change: Many factors have given us confidence in our BLA submission pathway.
Speaker Change: First and foremost it has a strong safety profile and has been administered to over 250 human subjects across several clinical trials over multiple years.
Speaker Change: Equally as important is that the data continues to show clinical and statistically significant efficacy in the treatment of DMD cardiomyopathy.
Speaker Change: This data is foundational to our BLA filing.
Speaker Change: I would also like to point out that it has become well known that the cardiac and skeletal aspects of the disease do not decline at the same rate. So therefore, because we see an impact on both cardiac and skeletal muscle function. We are confident that we are seeing a treatment effect of <unk>.
Speaker Change: Yourself across multiple domains further strengthening of the opportunity to treat D. M. D was dare myself.
Speaker Change: Furthermore, as exhibited this week at the muscular Dystrophy Association conference, we see a year over year improvement in function.
Speaker Change: I think that long term use of Darren myself is warranted for the treatment of D. M D.
Speaker Change: We also have continued to work in close collaboration with the FDA.
Speaker Change: Have been the beneficiaries of sea birds approach to drug development and believe that with the combination of statistically and clinically significant data that addresses an unmet medical need we are well poised to gain approval of Durham myself for DMD cardiomyopathy.
In addition, our RMR designation allows us to work directly with FDA as we realize the true goal here is to bring the first treatment to market for DMD cardiomyopathy.
Speaker Change: There are no approved therapies.
Speaker Change: Lastly throughout 'twenty 'twenty four we met with the F D. A multiple times outlining our BLA filing strategy.
Speaker Change: Which we have discussed previously.
Speaker Change: I want to reiterate that they have not requested the hope three data and currently we do not believe it will be necessary to support our application.
Speaker Change: As you May recall, the hope three clinical trial has a primary efficacy endpoint of the performance of the upper limb version two point out which is an indicator of skeletal muscle function. We plan to use this data in the future for potential label expansion.
Speaker Change: And we are currently evaluating the plans for hope three and we'll provide more updates as they become available.
Speaker Change: Now turning our attention to commercial planning.
Speaker Change: We are actively working with our commercial partner <unk> pharma on launch readiness for the United States.
He areas of overlapping focus our market access and reimbursement.
Speaker Change: There came in the United States is comprised of approximately 125 people with market access reimbursement medical affairs and sales teams actively preparing for the launch of Darren myself in the United States.
Speaker Change: We recently completed surveys with a top five payers in the U S. What's the response coming in very favorably from a reimbursement standpoint for Darren myself.
Speaker Change: Since there are currently no approved therapies for DMD cardiomyopathy, and data suggests that standard cardiac medications do not have a significant impact on disease progression in most cases we.
Speaker Change: We see Darren myself as a transformational treatment option for these boys and young men.
Speaker Change: We expect reimbursement would be consistent with other recently approved DMD therapies, such as exon skippers. If approved we were anticipating that approximately 50% to 60% of the overall G. M. D population in the United States or around 7500 boys and young men with G. M D.
Speaker Change: Would be eligible for treatment with Durham myself.
Speaker Change: If approved we are anticipating entering the market with approximately 100 patients transferring from our open label extension trial to commercial product. This would drive revenue to the bottom line.
Speaker Change: Remind you in the U S. We are entitled to between 30% to 50% of revenue share based on sales of the product inclusive of cost of goods sold.
Speaker Change: Now I'd like to shift our attention to CMT or chemistry manufacturing and controls.
Speaker Change: Our current GMP compliant facility located in San Diego is fully operational and stopped producing doses of Jeremy myself, we built and carefully designed this facility to meet early market demand.
Speaker Change: Fully operational capacity in this facility can support approximately 250 to 500 patients per year, and we believe will be sufficient to support the anticipated first year of launch.
Speaker Change: We expect a strong launch and rapid adoption of Durham myself for D. M. D. Cardiomyopathy, if approved I am pleased to announce that we have expanded our current San Diego facility, where we have leased an additional 25000 square feet for which we are going to build additional clean rooms, taking our manufacturing capacity.
Speaker Change: For approximately two to 3000 patients per year.
Speaker Change: Once completed and fully operational.
Speaker Change: [noise] assembled a world class team, who is extremely sophisticated in this area and I have high confidence we will have this new expanded facility online by mid 2026, allowing us to bolster supply of the product for the next several years to meet demand.
Speaker Change: As we continue to expand our capabilities, we are already looking into product development endeavors to allow us to increase our yield further.
Speaker Change: On the corporate and commercial fronts, we are able to look at our balance sheet, our cash balance of approximately $150 million is being deployed across the organization with our current runway into 2027 with no additional infusions of cash is.
Speaker Change: If we receive FDA approval, we will be slated to receive an additional $80 million milestone payment from Nippon sheet Yoki and in addition, we would receive a priority review voucher, which we have the full rights to sell or transfer. These.
Speaker Change: These non dilutive cash infusion could total well over $200 million, which was hit our balance sheet and 2025, alongside our existing cash and potential product revenue.
Speaker Change: This puts us in a strong position to deliver for our shareholders on multiple fronts. Our strong cash position will continue to allow us to strengthen our commercial organization, which includes enhancing our team with commercial and medical expertise and <unk>.
Speaker Change: Order to fuel future product development opportunities for Darren myself and enable us to build capricorn into a world class revenue generating cash flow positive company with a commercial product on the market and a robust pipeline of expansion opportunities leveraging sell an accident based therapeutics.
Speaker Change: Last for a brief update on our European partnering efforts last year, we entered into a term sheet with nib punched in Yahoo for the marketing sales and distribution of Jeremiah sell in the European region subject to Finalization of a definitive agreement or commitment to knit bunch and jaco in the USA.
Speaker Change: In Japan as our commercial partner is strong, but we have not yet come to final terms on the definitive agreement, which is still being negotiated in the meantime, we haven't achieved important regulatory designations in Europe and are on track for meeting with the MAA in the second quarter of 2025, we will provide additional.
Color is our strategy for Europe continues to unfold.
Speaker Change: Now I'd like to switch gears and give an update on our extra domes pipeline program.
Speaker Change: We continue to develop our self acts acts as one platform technology.
Speaker Change: As part of our next generation drug delivery platform.
Speaker Change: Our goal is to build the extra dogs into a standardized drug delivery platform that has enhanced capabilities will compare to a lipid nanoparticle, including targeting and delivering content across the cell membrane well.
Speaker Change: While most of our focus has been on the commercialization of Darren myself, we've had a small team working on building the accidents in the background. They have successfully designed our manufacturing method that is cost effective and can be expanded to make large amounts necessary for therapeutic delivery. We have presented this proof of.
Speaker Change: Sept data at many scientific meetings and published these findings in peer reviewed journals.
Speaker Change: Our approach is to concurrently demonstrate the utility of the X zone by developing a vaccine platform that is unique using native proteins loaded in our quoted on an extra them there could be made rapidly within the 100 day constraints developed by the U S government.
Speaker Change: But also able to generate a robust and long lasting immune response that program as part of the U S. Government's project Nexgen, which aims to test vaccine candidate for COVID-19 Prevention and addition to prepare for future Pandemics.
Speaker Change: Currently our stealth ex vaccine candidate is in the manufacturing phase the NAIAD, which is the national institutes of allergy and infectious disease will then conduct and fully fund a phase one clinical trial.
Speaker Change: Currently manufacturing is underway for our stealth ex vaccine and the NAIAD is planning for regulatory approval in the second quarter of 2025 with the clinical study initiated soon thereafter, we will provide further updates on this program as they become available.
Speaker Change: Now I'll, let jeremiah sellers on a defined path towards potential commercialization. We are further evaluating the password for a therapeutic exercise pathway and also evaluating other opportunities to expand our future pipeline.
Speaker Change: In conclusion 2024, it was a transformational year for Capricorn.
Speaker Change: <unk> 25 is the year in which we will hopefully transition into our next stages of development.
Speaker Change: I am proud of our progress.
Speaker Change: <unk> to the patients their families our investors for their continued support.
Speaker Change: <unk> goal is to continue to meet its milestones as we continue to focus on our efforts on bringing Darren myself towards potential commercialization.
Speaker Change: Our investing judiciously across the organization to prepare for that endeavor.
Speaker Change: I will now turn the call over to a J to run through our financials Hey, Jay.
Jay: Thanks Linda.
Jay: This afternoon's press release provided a summary of our fourth quarter and full year 2024 financials on a GAAP basis.
Jay: We may also refer to our annual report on Form 10-K, which we expect to become available in the next several days won't be accessible on the SEC website as well as the financial section of the company website.
Jay: Let me start with our cash position as of December 31, 2024 cash cash equivalents in marketable securities totaled approximately 151 $5 million.
Jay: Subsequent to December 31, 2024, we received a $10 million milestone payment from Nippon sheet Jaco pursuant to the terms of our U S distribution agreement.
On a pro forma basis, our cash cash equivalents marketable securities will total approximately $161 $5 million.
Jay: Turning now to the financials revenues for the fourth quarter of 24 were approximately $11 1 million compared to approximately $12 1 million for the fourth quarter of 2023. The source of revenue is the ratable recognition of the $40 million. We have received from our U S distribution agreement with the bunch of New York and the $10 million milestone payment we triggered.
Jay: In December 2024, after we submitted our BLA to the FDA.
Jay: Moving to operating expenses for the fourth quarter of 24, excluding stock based compensation.
Jay: Our research and development expense was approximately $13 6 million compared to approximately $9 4 million in Q4 2023.
Jay: And again, excluding stock based compensation, our G&A expense was approximately $3 million in Q4, 2024, and approximately $2 1 million in Q4 2023 net loss for the fourth quarter of 2024 was approximately $7 1 million compared to a net loss of approximately 800000 for the fourth quarter of 2023.
Jay: Net loss for the year ended December 31, 2024, it was approximately $40 5 million compared to a net loss of approximately $22 3 million for the year ended December 31 2023.
Jay: We will now open the lineup for questions operator.
Speaker Change: Thank you ladies and gentlemen, we will now begin the question and answer session should you have a question. Please press star followed by one on your telephone keypad should you wish to cancel your request this must styrofoam layer to do if you're using a speaker phone. Please lift the handset before pressing any DS one moment. Please for your first question.
Unidentified Operator: Your first question comes from the line of Ted <unk> from Piper Sandler. Please go ahead.
Ted: Great. Thank you very much and congratulations on all of the progress that's going to be an exciting year for you guys.
Speaker Change: Just looking at.
Speaker Change: The commercial preparation before Darren minus so what additional color can you tell us about the proppant.
Speaker Change: Division of labor between you and <unk> and how much of a benefit will it be with their existing commercial infrastructure. Thanks.
Yes.
Speaker Change: Thanks, Brad Yeah, it's great to hear from you as always so yeah. This has been a really exciting time for copper core and obviously <unk> is very excited as well, we just keep hitting our milestones as we told them, we would and so theyre at full scale preparation for commercial launch they have approximately 125.
Speaker Change: Employees are in the U S that had been dedicated to their real Pepto franchise. So there well ensconced in duchenne space, they're spending a significant amount of their time now.
Speaker Change: Now focusing on getting Jeremiah sell ready for commercialization, what that means sort of the in the trenches sort of way is that we are working with them on a day to day basis, I'm building out modeling or forecasting of market penetration first year launch activities working with physicians.
Speaker Change: And also potential infusion centers to make sure that that set up in an organized market access a meeting with payers now that's in full speed now that we have an accepted BLA and all of the pieces are in place. Our role is primarily to shepherd dare myself to market their role.
Speaker Change: Is to sell it and distribute it and obviously part of selling.
Speaker Change: Biologic like there myself is a deep understanding of the product, which we are providing to them. So we're very very excited about their energy and also working very closely with them on launch.
Speaker Change: Great. Thank you very much.
Speaker Change: Yeah take care Ted.
Speaker Change: Thank you.
Unidentified Operator: And your next question comes from the line of Blue Langer Shell from Oppenheimer. Please go ahead.
Speaker Change: Hey, good afternoon.
Speaker Change: Great to hear and all the progress and we're looking forward to Cleveland coming later this year for capital Corp.
Speaker Change: So a few questions.
Speaker Change: First Linda wanted to ask with respect to your discussions with payers.
Speaker Change: So far I'm wondering if that's part of those discussions if there was any contemplation of.
Speaker Change: For patients who may be on other premium priced drugs for DMD.
Speaker Change: And then may be also going on <unk>.
Speaker Change: Or even vice versa, just wondering given the expense burden of having to premium placed drugs for this condition.
Speaker Change: However, the players doing that if you have any intelligence there.
Speaker Change: Yeah. So we've had really good feedback leland from the payers. So far there are several reasons why I think this is going to be a great opportunity for reimbursement number one and first and foremost it is the only therapeutic that it will be treating the duchenne cardiomyopathy. This is cannot be highlighted enough you worked.
Speaker Change: Talking about a disease, where not only is cardiac disease. One of the primary features of the pathophysiology, but also has been indicated that to be more and more important as potentially treatments of the dystrophin novelty could become more relevant such as a gene therapy, we see long term benefit year over year, we've seen three years.
Speaker Change: Of improvement or stabilization in cardiac as well as skeletal muscle function, which we've shown not only to the FDA, but are also presenting to the payors. So overall, we think that you know when they have their you know sort of pharmacopeia to pick from and treating do said this will be a great opportunity to provide therapeutic benefits to them.
Speaker Change: Area that has largely been unaddressed.
Speaker Change: Can reduce hospitalization and mortality and will go along with any of the other treatments for the district monopolies.
Speaker Change: Alright. Thank you and then a question just sort of longer term.
Speaker Change: Got a healthy cash balance youre going to have the $80 million.
Speaker Change: Presumably on the FTA approval and then that.
Speaker Change: Which you could monetize them that doesn't even include other terms with them and as you know with Japan and potentially you're just curious given that the.
Speaker Change: The operating expense for textbook horrible remain relatively lean given that you wouldn't have to be building your own sales force and getting into commercial infrastructure themselves.
Speaker Change: Hum.
Speaker Change: Any thoughts on how you may invest that capital obviously exosomes.
Speaker Change: Very promising but given the stage of that development. It doesn't really seem like youll be expensive to be winning those initial studies. Just wondering if you have any thoughts on where to acquire.
Speaker Change: The much greater balance sheet that you may be coming into play.
Speaker Change: Thank you Leland, we really appreciate the question and it's actually been an area of exploration within the company right now we're looking at all types of opportunities not only in terms of label expansion for skeletal muscle myopathy of Duchenne and also Becker, which can further enhance our balance sheet as we move forward.
Speaker Change: Yes, we are focusing on our exits on pipeline I talked a little bit about that it is farther behind Darren myself and we'll be keeping you in the street updated is as we evaluate the opportunity to move this company forward, which we think will be plenty of them over the next few years.
Speaker Change: Alright, thanks for that color.
Unidentified Operator: Thanks Leland.
Unidentified Operator: Thank you and your next question comes from the line of Joe <unk> from H C. Wainwright. Please go ahead.
Speaker Change: Hey, Linda and a J. Thanks.
Speaker Change: Thanks for taking the questions. So first maybe for a J I mean this is fantastic news also that Linda you just announced about.
The new expanded 25 K square foot facility.
Speaker Change: Are you able to sort of define the cost and timeframe for this facility.
Speaker Change: Yeah. Thanks, Joe Yeah, we are excited about the expansion in our current footprint here in San Diego.
Basically the floor above where we're already operating in.
Speaker Change: We're kicking off all the planning right as we speak to expand out we havent put out the formal estimate, but I will say, we built our original clean room.
Speaker Change: Our commercial qualified clean room here in San Diego for just under a couple of million dollars. So we have the plans in place we have the team in place we already have the construction.
Speaker Change: Operations underway. So we envision that we can do this to a very reasonable number and we'll put more of that out there in the coming months as we move ahead.
Unidentified Operator: Got it and then I guess second question is obviously you have no indication that there's going to be an AD com right now.
Unidentified Operator: When do you think you might hear an answer and for example, if there were an AD com with that would be a place that they might want to not necessarily require but maybe force the discussion regarding hope three.
Speaker Change: Yeah. Thanks, Joe Yeah, So we're waiting everyday to hear from them.
Speaker Change: On whether they would want an AD com they will need some time to put it together and even though we're actively prepping for one as we speak.
Speaker Change: You know they they have to give us time to prepare as well so we expect to hear soon.
Think part of the delay is just based on some of the turmoil that's going on in the government right now and so I expected things are moving at a different pace than they might have even just a few months ago. So stay tuned when we know we will let everybody else know, we see an AD com, neither as a benefit nor risk.
Speaker Change: We believe very strongly in our application we are clinically and statistically significant data the data stands on its own. However, if we need to get out there and talk about it.
Speaker Change: We will absolutely do that in terms of hope three.
Speaker Change: What they have told US is that they are not considering poke three four this biologic license application that they understand that their primary efficacy endpoint of poultry as skeletal muscle, but that would be used for post approval label expansion. We plan on taking KOB three potentially outside the U S order expand our global footprint.
Speaker Change: And the focus of this application as we and they understand it is the data that we've talked about which is the hope to data to hope to open label extension data compared to the natural history dataset from the cardiac consortium and so I don't anticipate a discussion of KOB three at an AD com, but if it comes up we'll be ready to take those questions.
Speaker Change: As well.
Speaker Change: Also very helpful and thanks for the repetition there quick.
Speaker Change: Quickly just on the discussion about cash usage going forward, obviously, you talked about some of the obvious things.
Speaker Change: Since you guys that really have a specialty in say cellular therapy here.
Speaker Change: Would you consider in licensing number one and then second with regard to Europe I know you can't really even this or can't necessarily describe the first.
Speaker Change: The first part of the question, but what are the outstanding sort of things.
Speaker Change: Things that you are still meeting or the opening questions with regard to potential signed N S.
Speaker Change: Hartner ship and then also what are the outstanding questions. You feel are important for your EMEA discussions.
Speaker Change: Yeah.
Speaker Change: Yeah. Thanks, Joe that's a multi pronged question. So in terms of in licensing we're evaluating opportunities as they become available right. Now we are laser focusing on getting gara mitel approved and getting that ready to launch that's going to obviously drive our our cash value it and our abilities to to look at new opportunities I will say this we are expert.
Speaker Change: Now and translational medicine in and bringing things sort of from the colloquial a bench to now commercial so we would not rule out the right opportunity should it be presented to US having said that shifting to your second question about the M&A.
Speaker Change: What what we're doing and what we're really focusing on now is the ability to get there and myself to Europe. We are working right now with the MAA and with consultants and outside the U S. In order to build that program, we will be meeting with EMA hopefully in the second quarter of this year to further understand what they are.
Speaker Change: <unk> for approval of dairy myself because.
Speaker Change: We are working directly with our Europe, right now and because we are.
Speaker Change: Seeing some success by getting designations in Europe of AT&T, and and working with E. M. A were slowing down a little bit our conversations to make sure that we have the best path forward for Darren myself in Europe, we are still negotiating with them and have an active conversation go in terms of.
Speaker Change: The definitive agreement and and I really feel very positive about their commitment to the therapeutic.
Speaker Change: Really appreciate all the color thanks a lot.
Speaker Change: Thanks, Joe take care.
Speaker Change: Thank you and your next question comes from the line of Kristen <unk> from Cantor. Please go ahead.
Speaker Change: Hi, everyone. Congrats on the data this week and the recent filing acceptance. So I know that your mechanism is complementary to a gene therapy, but after yesterday's unfortunate news regarding the patient on a love. It as we are hearing doctors emphasize the need for treatment options for non Ami.
Speaker Change: Military patients in particular, so can you just remind us what percent of the later stage population in particular would have cardiomyopathy and then while label expansion could come later could you reference some of your pull data if you have and approval as perhaps supportive evidence. Thank you.
Speaker Change: Yeah, Thanks, Kristen yet I, thank the entire community.
Speaker Change: Is reeling from the death of that young man, it's a small patient community and so the loss of a one life is tragic always but especially in our community such as this.
Speaker Change: Most of our patients that we've treated or have been non ambulance. They are primarily the ones that have the cardiomyopathy manifestations, although we've been messaging that the scar that causes the ultimate decline in cardiac function is there when they are little so we're going to try and get it as young as possible, but again most of our.
Speaker Change: Patients are the later stage non ambulant the focus of this application is the cardiomyopathy, but as you correctly stated we have shown year over year improvement in performance of the upper limb and our open label extension guys.
Speaker Change: And all of those guys are not Andrew it. So we do believe that there am I S. L is has been and will continue to be a very good option for those that are in later stages non andrew into the disease.
Speaker Change: And look forward to taking that across the line as well in the future.
Speaker Change: Okay.
Unidentified Operator: Thank you for that and then another question I know you have robust safety data as well, but as we start to think about the potential to move to commercialization, where there will be the potential for more to get treatment. At once is there anything we should consider from a safety protocol will physicians want a tree a few patients.
Speaker Change: First or well doctors require.
Speaker Change: A more extensive testing and the reason why I ask is sometimes like we are seeing with the gene therapy that there's a plethora of different specialists that need to be involved because of some of the known safety risks of that.
Speaker Change: Yeah. Thanks, you know so I think the defense community.
Speaker Change: And I have been the beneficiary of participating in many of the symposia had been talking about the care teams necessary for a gene therapy for several years. The gene therapies are very complicated because they use viral vectors and as you know the immune system doesn't usually like it when the virus enters the system and so you have to do a lot of pre plan.
Speaker Change: And pre treatment to mitigate that immune response.
Speaker Change: In terms of Jeremiah cell to cell therapy, we are not doing any genetic manipulation of it they've performed over 700, infusions and not seen any serious safety events since the early stage hyper sensitivity, which has been mitigated.
Speaker Change: Pretreatment with anti Histamines and steroids I'm at the abundance of caution we will to our infusion set infusion centers or in care centers stood a hypersensitivity response ever occur we don't anticipating needing a care team. It's a very simple infusion and where the side effects, which are mild and sort of you know a little bit of.
Speaker Change: A mild flu like symptoms and some of our patients.
Speaker Change: Our easily mitigated and understood by our investigators. So I don't think it's going to have the same complicated introduction or care paradigm needed as a gene therapy.
Speaker Change: Thanks, and last question for me I think you commented that with the additional manufacturing footprint just could support two to 3000 patients potentially as early as mid 2026, but should you got an approval later this year, how should we be thinking about well you can essentially.
Speaker Change: <unk> handle until that time, because I know you said, there's already some patients that want to transfer on OLED as well as others that weren't involved in the trial that are aware of the potential of the drug. Thanks again, everyone.
Speaker Change: Yeah. Thanks, Kristen Yeah, so our manufacturing facility here in San Diego, which we built in anticipation of commercial launch I can service between 200 5500 patients per year do you actually look at the timeline of watch. We're looking you know all things considered it a Q4 launch this year and by the time the new facility comes on it won't even be a fee.
Speaker Change: Full year into commercialization and so we believe that we'll have enough from our small scale commercial facility in San Diego to me you know early demand and then be able to switch right away and into the new facility as we mentioned, but I'll highlight the new facility is actually within the footprint of our currently existing manufacturing.
Speaker Change: Plant and so we're hopeful that the regulatory obligations required to bring that facility online will be light and therefore, we'll be able to get them on board as soon as possible after lunch.
Speaker Change: Thank you so much again.
Speaker Change: Thanks.
Speaker Change: Thank you.
Unidentified Operator: And your next question comes from the line of Catherine Novak from Jones trading. Please go ahead.
Catherine Novak: Hi, good afternoon, everyone. Thanks for taking my question I'm. Just wondering if you can talk a little bit about them you know in the past FDA had made comments about the issues of single arm studies and D. M D D.
Catherine Novak: Due to the disease heterogeneity can you just give us some color about you know in your discussions with FDA what have they brought up regarding their view on cardiac outcomes shy and hope to L. O N E.
Catherine Novak: This might be why they might take a different approach on that thanks.
Speaker Change: So I think what you're asking is because the hope to open label extension study is not placebo controlled how how is the FDA looking at that data at my understanding that correctly.
Catherine Novak: Correct.
Catherine Novak: Okay. Yeah. So there's a couple of very important point there. Thanks, so much for calling in.
Speaker Change: Number one is we have had access to a propensity matched H function medication mask set.
Speaker Change: Set of patients from the Vanderbilt natural history cardiac consortium study funded by the F. D. A to be able to use as an indicator of what would be considered a placebo group right. A group that has been untreated and we can therefore look at the natural history of the cardiomyopathy compared to the opening.
Speaker Change: Well extension guys on a year over year manner.
Speaker Change: Typically this would be an issue. If you were doing a type of volitional measurement like in Northstar ambulatory assessment of time to rise or in our case the performance of the upper limb because one might say well do you know what.
Speaker Change: Getting treated you know you're going to you know, maybe a function or perform better.
Speaker Change: Beauty of this dataset and the reason the FDA has been willing to look at it. So carefully is because nothing about cardiac MRI is volitional you cannot wish your heart better you cannot wake up and think I'm gonna get a cellular therapy and therefore, my heart's gonna function better so even though the datasets relatively small it's very concise.
Speaker Change: The ability to measure function are strong statistical significance is emblematic of the strong treatment effect that we are seeing that means there's very little opportunity for this data to be up to chance and so as a result of that.
Speaker Change: We remain confident that this is not a typical single arm study, but one that actually has a natural history real world evidence component that supports the data.
Speaker Change: Got it thanks, thanks, so much.
Thank you.
Unidentified Operator: Thank you and your next question comes from the line of eating Hussein off from leading break. Please go ahead.
Speaker Change: Hi, good afternoon, everyone. Congratulations on the progress.
Hussein: So a couple of questions from us.
Speaker Change: Maybe I think you mentioned the heart diseases, where are.
Hussein: Are there more so might be helpful.
Hussein: Obviously, then the next indication the next sort of low hanging fruit as becker's muscular dystrophy and <unk> corporate presentation. So.
Hussein: When when do you think we will we.
Hussein: We will have all dates on the potential Becker muscular dystrophy trial design and whether it is going to look like half two so could you give us a little bit of insights in terms of what is yours clinical strategy as it comes to Becker muscular dystrophy.
Hussein: Thanks Hayden Yeah. It's funny is that was that putting together my remarks.
Hussein: Today, I was thinking a little bit about you and I knew that you'd have come to me with the Becker question. So we're working with the agency now and some of the initial steps of getting our program going with Becker. We've submitted some time requested them recently, our focus is to get their micelle approved for Duchenne and then I'll begin our our emphasis on building.
Hussein: Towards Becker for which by the way we hold all the economics.
Hussein: S has no rights to those.
Hussein: Our goal will be to convince the agency that the cardiomyopathy associated with Becker looks and functionally is the same as the Duchenne cardiomyopathy and see if we can potentially move towards an accelerated pathway and Becker, we are bringing and key opinion leaders are literally as we speak.
Hussein: And medical expertise to Capricorn to help us build out our Becker program and I expect to have more color for that program.
Hussein: Program over the next quarter or two.
Speaker Change: Okay understood. Thank you. Thank you for the update on.
Speaker Change: What are other heart diseases, where you think that Omar so can we.
Speaker Change: Make a meaningful difference I'm, just trying to understand the whole sort of commercial potential for thorough myself.
Speaker Change: Beyond DMD and beyond BMT.
Speaker Change: Well look you know this is this is an area of great interest to cap a core right now we have a very powerful therapeutic the defined mechanism of action, which is immuno modular tori that drives anti fibrotic activity were evaluating orphan cardiomyopathy is looking at where we might best dipped.
Speaker Change: Floyd him I think many of the physicians and are closer to a room, including yourself have ideas of where that could be and will provide some updates on pipeline expansion into other orphan cardiomyopathy is as we begin to evaluate them and determined that that would be the best path forward for <unk> for Darren myself.
Speaker Change: Okay. Thank you. Thanks, so much.
Speaker Change: Yep. Thank you have a great day.
Unidentified Operator: Thank you and your last question comes from the line of Madison <unk> from Bofa Securities. Please go ahead.
Madison: A J. Thank you for taking our questions and congrats on the progress.
Speaker Change: I'm coming away from MDA. So my question is maybe a bit academic so forgive me.
Speaker Change: Based on what you've seen through the course of <unk>.
Speaker Change: Termite sales development, what do you think is the ideal baseline.
Speaker Change: Ejection fraction baseline where impact would be the highest.
Speaker Change: Is it in the for example, less than 35% range or is it more than 35% to 45% range.
Speaker Change: And then secondly, I believe you mentioned that you expect 7500 boys be eligible.
Speaker Change: Would this be based only hard ejection fraction threshold and I ask given that cardiac treatment in this population as often as a result of sugar.
Speaker Change: Sure decision, making.
Speaker Change: <unk>.
Speaker Change: Yeah Madison. Thank you so much and hope you enjoyed the M. D. I. So you know what we understand about the cutoffs for ejection fraction is that if we can get in there early we believe that we will see the greatest long term benefit in terms of stabilization of disease progression. So.
Speaker Change: The patients with ejection fractions of 45% or greater we see sort of the greatest amount of long term stabilization in some of our patients haven't seen any decline in ejection fraction literally in years.
Speaker Change: That is because we believe that once you have the immuno modulation anti fibrotic youre actually preserving a healthy heart tissue that does exist, having said that we have patients that have had lower ejection fractions, where we're also seeing stabilization of the disease process, but I can say sort of overall is that early treatment is better.
Speaker Change: <unk> because you have a longer opportunity with we are a better opportunity than I see longer, but a better opportunity to treat the disease process early.
Speaker Change: Preserved cardiac function and also obviously, then hopefully sustain a life and a not only quantity, but quality of life. So we're still open to all comers in terms of ejection fractions, we do know the once they get below a certain point once they get to what would be considered an end stage heart failure.
Speaker Change: And it's harder and harder to bring them back from that edge and so that's what we're messaging you know to.
Speaker Change: To the agency as well as to physician leaders that we need to get in there early and I guess it will be a decision of the care providers. We're working now very actively with the cardiologists to treat you send patients both pediatric and adult as well as the neurologists so that they become educated and open to the idea of using dare myself.
Speaker Change: Part of the treatment paradigm for DMD cardiomyopathy.
Speaker Change: Understood. Thanks.
Speaker Change: Thank you for your question.
Speaker Change: Thank you I'll now turn the call back to management for final thoughts.
Speaker Change: Yeah. Thank you for joining today's call. We look forward to updating you on our continued progress over the coming months and I Hope you have a nice evening.
Unidentified Operator: Thank you and this concludes today's call. Thank you for participating you may all disconnect.
Unidentified Operator: [noise].