Q1 2025 Capricor Therapeutics Inc Earnings Call
Joseph Pantginis, Richard Miller, Edward Tenthoff, Leland Gershell,
Speaker Change: Good afternoon, ladies and gentlemen, and welcome to the Capricor third-of-futics' first quarter, 2020-25 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 with any time during this call is required immediate assistance, please press far-zero for the operator. Thank you very much for your later.
Speaker Change: Well there has been some progress made in treating the dystrophin apathy with several drug therapies now on the market in the U S.
Speaker Change: Civic to the generic genetic mutations associated with the disease. There are no therapies approved or on the market that aim to specifically treat the cardiomyopathy associated with G. M D M.
Speaker Change: Darren myself mechanism of action, which is immuno modulator Orient anti fibrotic is directly targeted to treat the secondary effects of D. M. D and we believe can be used with other therapeutics, which are currently approved or in development to treat the genetic mutation.
Speaker Change: Myself as delivered by a simple intravenous infusion once a quarter at a dose of 115 million cells.
Speaker Change: I would now like to discuss the data.
Speaker Change: Supports our BLA.
Speaker Change: Finally, as based on a blinded randomized and placebo controlled hope to study and also by the hope to open label extension study compared to a robust S. T E N H L. B I funded natural history dataset.
Speaker Change: Well the sample sizes are small what is most relevant is not the size of the dataset with the statistically and clinically significant differences are highly unlikely to be due to chance we.
Speaker Change: We have worked with multiple internal and external statisticians presented the data at meetings and to Kols and what we've heard seen an accurate upon was that the likelihood is extremely low but the impact on the heart or for that matter. The skeletal muscle is due to chance we have three clinical trials and approximately.
Speaker Change: Four years of open label extension data that supports that premise. There has also been an emphasis and written guidance from FDA encouraging the use of real world evidence to support clinical trial data, especially in rare diseases, Darren myself as a perfect case for using this type of data to validate the efficacy of a drug product.
Speaker Change: Turning to the hope three trial, our phase III study, which is ongoing and fully enrolled in the United States I wanted to be clear that at this time FDA has not requested the efficacy data from the hope three study to support our BLA application, although FDA has reviewed and will continue to.
Speaker Change: Review the safety data from the study.
Speaker Change: Our current plan is to use this data in the future for potential label expansion and are actively evaluating plans for hope three to be expanded internationally. We will provide more updates on this program as they become available.
Speaker Change: Now as we were transitioning copper core from a translational medicine company into a commercial stage entity. We continue to actively work with our commercial partner N S pharma launch readiness for the United States.
Speaker Change: As we announced earlier today, we also appointed Dr. Michael <unk> as our new Chief Medical Officer, I am extremely proud that Dr. Banks join our team. He has over 25 years of experience.
Speaker Change: Global clinical development translational research efforts across the industry. Most recently as vice President and head of rare disease clinical and translational research worldwide research development and medical adviser and prior to that at GSK, where he was instrumental in advancing multiple first in class therapies and earn.
Speaker Change: Early and late stage development.
Speaker Change: Based on our current plans, we aim to have over 100 patients transitioning from clinical to commercial product following potential BLA approval.
Speaker Change: Let me remind you that we had been providing care myself all open label extension patients for over three years nearly all hope three patients are in open label extension now I will transition to commercial product. If it is their desire to continue under myself.
Speaker Change: We along with that as pharma are now working with physicians to assist them in person pairing to prescribe there myself for DMD cardiomyopathy.
Speaker Change: We know it will be a partnership between treating neurologists and cardiologists and prescribing Gary myself and this is part of the reason we are enhancing our medical leadership with Doctor banks, who will guide the physicians through the prescribing process.
Speaker Change: Please remember that Darren myself is not designed to compete with the medicines that address the dystrophin empathy, such as gene or exon skipping therapies.
Speaker Change: Rather to address the secondary aspects of the disease, which is inflammation and fibrosis, both in the heart and skeletal muscle.
Speaker Change: <unk> is a very strong safety profile, it's important to note that a nationally derived cell therapy does not have the safety risk that is in any way similar to the gene therapies, which do involve viral vectors.
Speaker Change: Now for an update on our commercial manufacturing preparations as you know we built our San Diego GMP manufacturing facility for the purpose of commercial manufacturing. So I have a high degree of confidence in our processes procedures and facilities.
Speaker Change: To remind you our San Diego GMP facility fully staffed and operational and is currently producing doses of Darren myself and.
Speaker Change: In addition, we are underway with our previously announced manufacturing expansion to build additional clean rooms in the same facility.
We plan on the expansion to be operational mid to late 2020, six, allowing us to bolster supply of the product to meet potential demand.
Speaker Change: Turning to an update on our European partnering opportunities, we remain in negotiations with knit bunch and yaacov with respect to the potential distribution of Jeremy myself in the European region and have extended the period of negotiation of the definitive agreement to the end of the second quarter.
Speaker Change: Our strategy is emerging in regards to ex USA markets and we will continue to explore opportunities for our technology in other areas globally, we will add additional color as our strategy for Europe continues to unfold.
Speaker Change: And finally for an update on our <unk> programs, we continue to develop ourself X X as a platform technology as part of a next generation drug delivery platform. While this program has taken a backseat appropriately. It's a dire myself. We are still working to develop exosomes are cellular did the liberty deal.
Speaker Change: It does.
Speaker Change: We believe strongly in this opportunity and the accident the ability to change the way, we got biologics across the cell membrane.
Speaker Change: Our accident on the team is focused on advancing an efficient and cost effective way to manufacture them for scale for therapeutic utilization.
Speaker Change: Despite all the concerns regarding vaccines based on the newer ethos of the F. D. A I am pleased to inform you that our program under project Nexgen, which aims to test vaccine candidate for COVID-19 prevention and to prepare for future pandemic remains underway. Our vaccine is very important because it is not.
Speaker Change: Natural made from a native proteins and contains no adjuvant, which has been one of the main concerns of the new H H as secretary.
Speaker Change: We continue to work in conjunction with the National institutes of allergy and infectious disease, otherwise known as <unk>, which will conduct the clinical trial and provide the data to us.
Speaker Change: This vaccine could potentially be game changing as it meets all the criteria set forth by the U S government a future vaccine technology.
Speaker Change: Phase one of the trial is set to start in Q3 I will provide updates on this program as they become available.
Speaker Change: In conclusion, our program remains strong both of their past Darren myself potential approval on our extra dough platforms, our cash balance totaled approximately $145 million with our current run rate taking us into 'twenty 27 with no additional infusions of cash if we receive FDA approval we will.
Speaker Change: Be slated to receive an $80 million milestone payment from there a bunch of gnocchi and we will also receive a priority review voucher, which we have the full rights to sell these non dilutive cash infusions could potentially total well over $200 million. This will allow us to enhance our therapeutic pipeline for Darren myself as well as expand.
Speaker Change: Other areas of our pipeline in an effort to deliver value for patients and for our shareholders.
Speaker Change: With over 250 publications on the C D CS, including the mechanism of action, there myself and multiple statistically significant and clinically relevant clinical trials, demonstrating jeremiah yourselves impact on patients. We look forward to presenting our data to the advisory Committee at this time, we don't know the spa.
Speaker Change: Civic date for the meeting, but we will alert the market when a dataset I want to thank you for joining today's call. We truly appreciate your continued support I will now turn the call over to a J to run through our financials.
Speaker Change: Thanks Linda.
Speaker Change: This afternoon's press release provided a summary of our first quarter 2025 financials on a GAAP basis and you may also refer to our quarterly report on Form 10-Q, which we expect to become available shortly it will be accessible on the FCC website as well as the financial section of our website.
Speaker Change: Let me start with our cash position as of March 31, 2025, our cash cash equivalents in marketable securities totaled approximately $144 $8 million.
Speaker Change: Turning briefly to the financials revenues for the first quarter of 2025 were zero compared to approximately $4 9 million for the first quarter of 2024, I'd like to point out that the source of our revenue for the first quarter of 'twenty four it was the ratable recognition of the $40 million, we received under our U S distribution agreement with the bunch and Jaco.
Speaker Change: Which at this point has now been fully recognized as of December 31 2024.
Speaker Change: Moving to our operating expenses for the first quarter of 2025, excluding stock based compensation, our research and development expenses were approximately $16 2 million compared to approximately $10 1 million in Q1 2024.
Speaker Change: Again, excluding stock based compensation, our general and administrative expense was approximately $3 1 million in Q1 2025, approximately $1 8 million in Q1 2024 net loss for the first quarter of 2025 was approximately $24 4 million compared to a net loss of approximately $9 8 million for the first quarter of 2024.
Speaker Change: We will now open the lineup for questions.
Speaker Change: Thank you ladies and gentlemen, we will now begin the question and answer session. If you would like to ask a question. Please press Star then the number one on your telephone keypad, if you're using a speaker phone. Please make sure your mute function your stand off John allow your signal to reach our equipment again, Please press star.
Speaker Change: Followed by once you asked a question.
Speaker Change: Our first question comes from the line of Dan talk from Piper Sandler Your line is open.
Speaker Change: Okay.
Speaker Change: Great. Thank you very much can you hear me okay.
Speaker Change: Alright.
Ted: We can hear you fine thanks, Ted how are you.
Ted: Very well thanks.
Speaker Change: I just wanted to pick up on some of the comments that you have have you guys had your.
Speaker Change: It was actually up in San Diego and if not can you tell us one that's cause report.
Speaker Change: Yeah.
Speaker Change: Just with respect to preparation for the outcome walk us through maybe what you consider to be key features and I mean.
Speaker Change: Along the lines in terms of a particular problem. Thank you.
Speaker Change: Thanks, Ted Yeah, we haven't had or pre licensing inspection, it's coming up this quarter are within the next few weeks and so we'll update you guys. Once it's completed I will tell you. This places a buzz with preparation and we feel really good about it and we're looking forward to having that inspection done reminder, which I said in my prepared remarks.
Speaker Change: But this facility was built in anticipation of commercial manufacturing and so we we feel pretty ready for it.
Speaker Change: In terms of AD Com prep, we are actively working on that we had anticipated as we've said multiple times now that it was going to be requested by the agency typically as I also just stated when theres, a new indication or a new therapy first in class and they almost always conduct an AD com, we actually think it's an.
Speaker Change: Credibly good sign that they've asked for an outcome. One it shows that the agency is really moving forward with our application and I will tell you with I think we're over 20 information requests now a follow up meeting with them as most recently as tomorrow and several other meetings along the way they're actively in our file reviewing our file and working.
Speaker Change: With us they have told us in mid cycle review that there were no substantive issues, which gave us a good confidence in that in terms of AD com prep, we've already had to mark add comps and without speaking.
Speaker Change: About that because of confidentiality I can say that we passed with flying colors and the data looks really great. So we're looking forward to the future. We are waiting for a producer date and things here are hopping along as we get ready for approval.
Speaker Change: Great. Thank you so much.
Mylan Cushal: Our next question comes from the line of Mylan Cushal from Oppenheimer. Your line is open.
Speaker Change: Good afternoon, and thanks for taking the questions just two from US Linda first just wanted to ask with respect to Nippon Shin Young through you had signed the letter of intent or or what have you back in the fall and then I know you had updated us that it.
Mylan Cushal: We've extended the negotiation period.
Mylan Cushal: If we could also maybe you read into that that you may be considering an alternative mechanism for launching in Europe, perhaps on your own.
Mylan Cushal: Which which could be.
Mylan Cushal: More lucrative to two cap record in terms of economics preservation.
Mylan Cushal: Seem rare disease therapies. After you do even better over in Europe and in the States and then the second question is with respect to them and that's in the states. If you could just share to the extent that you can kind of how they're set up here in terms of their footprint.
Mylan Cushal: For marketing.
Mylan Cushal: During myself presenting.
Mylan Cushal: Yeah.
Speaker Change: Frank Leland, it's always great to hear your voice. So addressing your first question about our LOI with N S and the EU opportunity. Yeah. We have had really great experiences are building towards commercialization with that that's in the United States, We think that they're a great partner in the U S and and we're excited for the launch and I'll get into some of that.
Speaker Change: Color on your second question in terms of Europe, We've moved forward very rapidly in the United States. We were told and have now filed a BLA a four day on my iPhone in the U S. We have a great plan in the U S and we believe that that can be enacted in Europe. As you correctly stated therapies for rare diseases have a different.
Speaker Change: In Europe and I, there's nothing currently improved in Europe for D. M D and our data is very strong and new Sam a criteria. We are working directly with the European authorities ourselves right now as we prepare for our moving into Europe, and so we are evaluating on a regular basis the <unk>.
Speaker Change: Opportunity, we certainly remain in active negotiations with a bunch of Yahoo, and we'll provide more updates on that program as our thoughts and Theres evolve.
Speaker Change: In terms of their opportunity in the states they've done a really nice job of building a sales and marketing distribution team for built up so which is their exon skipper and so these seasoned executives who are U S. Based and has been a lot of time in the Duchenne space are preparing to launch there myself they have 125 F T.
Speaker Change: He's were told that nearly all of them are focusing on during my cell at this point, we work with their leadership both in the states and also in Japan. So that we remain aligned on the path forward, we are enhancing our own management team as you heard today, we appointed Dr. Michael <unk>, Chief Medical Officer, Dr. Banks is going to build some.
Speaker Change: Our med affairs support behind him, which we'll be talking about over the next month or two and we will continue to support our knit bunch and jaco and their education and commercialization for Darren myself to physicians in the community.
Speaker Change: Got it thanks very much.
Leland: Thanks Leland.
Speaker Change: Our next question comes from the line of Kristen <unk> from Cantor Fitzgerald. Your line is open.
Kristen: Hi, good afternoon, everyone. Thanks for taking the questions and I look forward should meaning in working with Doctor banks on your team. So you made a comment that when you talk to several key leaders. They they truly point out that these effects can't be by chance and that's something that we often hear is well when we speak to thought leaders.
Kristen: But I wanted to ask what are the biggest key drivers that these physicians say is that proof.
Kristen: I know the FDA certainly is no stranger to the natural history work, having funded it themselves, but maybe can you can you talk about what their viewing is truly the strongest signals when they look at that dataset.
Kristen: Yeah Christian. Thanks. So you have just hit on the most important part of our application and I want to highlight that the relevance even though theoretically in terms of new numbers of patients. The numbers are small sample size is small in reality. The reason that we were able to say very confidently that there is very.
Kristen: Little chance that the data is due to chance is because of the statistical significance now the statistical significance is actually a number that says how much likelihood is your data due to chance and well we were able to look at here is that MRI cardiac MRI, which is an objective measure of cardiac function.
Kristen: You can't what's your heart better you can't volition, only think Gee I'm going to perform better in the MRI today, and you can't do anything really to sustain or improve cardiac function in any relevant way in a short term basis and that is why MRI is such a clever and easy way of determining disease progression. So.
Speaker Change: Dr. Jonathan Zaslow, who also is the author lead author on the paper for the natural history study and is funded as I mentioned by F. D. A N N. They told me I have to collect that data has spoken very eloquently and pointed to literature that shows that very few patients are actually necessary to discern a treatment effect using cardiac M. A.
Kristen: Alright.
Kristen: So we are confident in the data and we are hopeful that the FDA will be confident into the data and that the outcome will also support the data because as I mentioned taken together mathematically as well as in an individual patient basis. The data is a very strong suggestion of not only improvement in ejection fraction, which is.
Kristen: As a measure of how the heart meets the needs of the body, but also the long term stabilization and even improvement in cardiac function. Our open label extension guys are out to four years now and some of them have dropped literally no ejection fraction points, which are non ambulant later stage Duchenne patient is almost unheard of.
Speaker Change: Thank you for that and yeah that was literally going to be my next question last summer is when you presented the three year data and well I know, we don't have a date yet for this odd calling them I'm wondering if you will have any four year data to share at that point with what the agency yours you've been disclosed.
Kristen: Early data as you've had these ongoing review meetings.
Kristen: Yeah, we plan on presenting the four year data at the P. P. M D meetings in June and you.
Kristen: No I'm delighted to say that the four year data is looking very promising I would give you that little bit of a preview.
Kristen: But it is really quite exciting to see this long term stabilization as I mentioned not only in the disease process, but remember our guys are the older guys. These are the guys that are in the later stage of the disease that they are losing function I'm in a measurable and very almost reliable sad to say way and so the fab.
Kristen: That there's long term stabilization and cardiac as well as the performance of the upper limb function is really important and data we plan to highlight a P. P. M D.
Speaker Change: Okay. Thanks, and then the last question that I had is obviously, there's been a lot of changes.
Speaker Change: The administration. So can you just talk from a high level if the people that you've been.
Speaker Change: King with over the course of the year and beyond when you first presented these data to the FDA has there been a lot of changes within that or for the most part are most of the people that you're speaking in working with the same people that it.
Speaker Change: And part of the process. Thanks again, everyone.
Speaker Change: Thanks, Chris and you know, it's funny I get asked that question I don't know eight to 10 times per day by investors and I think it's really important to say that we see the FDA really starting to calm down and stabilize the.
Speaker Change: The appointment of Dr. Mccarthy the appointment of Dr. Prasad, both of which have spoken about their commitment to rare disease their commitment to moving therapies forward and their commitment to making sure the medicines getting to patients as they need them based on good quality data, which copper of course, certainly has has given us great confidence taken together.
Speaker Change: Other the reviewers that we've worked with over time for the most part most of them are there we believe that Nicole Verdun, our it's still there and she has been working on our file really since the beginning of 2024, when we started having high visibility with the agency and several of the other reviewers I don't want to call out their names are definitely still there are definitely engaged and as I.
Speaker Change: I mentioned in my answer to my question to Ted.
Speaker Change: We are getting literally bombarded with questions and opportunities to continue the conversation with FDA on a weekly basis. So we know they're actively in our file.
Speaker Change: Yeah.
Speaker Change: Our next question comes from the line of Scott. So we know that some challenged research your line is open.
Speaker Change: Hi, good afternoon, Thanks for taking my question.
Speaker Change: Just wondering strategically suppose that the FDA issued a CIL for efficacy in August.
Speaker Change: You know, what's the plan, which you can read out hope three and submit for D. M D skeletal muscle function.
Speaker Change: Or do you see going from there.
Speaker Change: Yeah, that's exactly right hi, nice to chat with you again. So you know we're in a really unique and quite favorable position and that way, we have a fully enrolled phase III trial, the indication for which the skeletal muscle dysfunction, particularly in the non ambulant patients as measured by the performance of the upper limb so different indication on the cardiomyopathy.
Speaker Change: The unexpected circumstance.
Speaker Change: They issue a C or for whatever reason in August we just turned around and submit the data for the whole three trial, which we expect to be positive based on three positive clinical trials proceeding and just go after the skeletal muscle indication and the secondary endpoints for hope three are the same cardiac ones that we've applied for.
Speaker Change: So we would just apply for cardiac and skeletal based on that it's a randomized double blind placebo controlled trial and certainly would support any of the findings that we've already submitted on.
Speaker Change: Got it you would intend to submit for both.
If you were filing with hope three data.
Speaker Change: Yeah, I mean, I'm expecting that the data would be reflective of what we've seen so far we have done blinded assessments of the hope three data compared to the whole two data and the distribution charges look almost identical.
Speaker Change: Identical they can overlay each other so I feel very confident that we would see similar results in hope three that we saw and hope to and I haven't seen you know theres no way to to preview.
Speaker Change: Blinded data for cardiac and so we expect that to be positive as well based on the MRI data we've seen in all our trials.
Speaker Change: Great and then if you did.
Speaker Change: Any specific feedback FDA has given you about L D.
Speaker Change: A surrogate end point you know what if they had about this endpoint the ability to predict cardiac outcomes. How much of that are you supporting with your OLED data.
Speaker Change: Yeah, So I actually read some of the sudden and one of your notes. So I think it's an important point to highlight them FDA has stated that they're not looking at ejection fraction as a surrogate endpoint in this specific situation rare disease D. M. D. That's why they funded study with jobs also by the office office of orphan products Copa.
Speaker Change: I knew what the NH lb I went to determine what measures they could conclude to be outcome measures in a rare disease and a rare pediatric disease, you cannot do an outcome measure like mortality not going to happen. So you have to be able to find other measures that are analogous to those outcomes of mortality or some other type of.
Speaker Change: Hospitalization and other types of standard cardiac measurements of progression of disease and use those and so doctors also published in the journal circulation heart failure in 2023. This beautiful study what the natural history data that we were then able to use on a patient level and a propensity matched way to show that the injection.
Speaker Change: <unk> and treated patients with Joe myself was significantly improved over what would be expected of natural history real World evidence has been stated by FDA and has been put forth in guidance as well as in a new office in Cedar.
Speaker Change: That's emphasize is looking at the actual progression of the disease real world evidence and able to use that analogous to a clinical trial dataset to make sure that efficacy is effectively analyze so we are in the really nice position of having good efficacy data of our own that we can compare to.
Speaker Change: Natural history dataset, and an objective measure, which is cardiac MRI of disease progression and so overall, we don't look at ejection fraction nor does the F. D. A in this situation as a surrogate measure, but rather an outcome measure.
Speaker Change: Got it thanks for clarifying that for me that was very helpful.
Speaker Change: No problem.
Our next question comes from the line of Deane as you know from Ladenburg. Your line is open.
Speaker Change: Hi, good afternoon.
Speaker Change: Congrats for the quarter. Thank you for taking the question. So I've got a couple.
Speaker Change: So given the sort of general pressure on all gene and cell therapy companies recently and.
Speaker Change: Actually in those.
Speaker Change: Sort of treatments that conditionally for almost a decade never got a confirmatory studies do.
Speaker Change: Do you think a full accrual or my so we'll put it into some kind of you know specific position when it can actually be utilized more than.
Speaker Change: Exon skippers, so or AAV gene therapies.
Speaker Change: Yeah.
Speaker Change: So I really can't comment on that specifically you know, we certainly would be in a good position with full approval. We have highlighted in our biologic license application in the community is well aware that we see both skeletal and cardiac benefit. So it's possible that physicians would use them with with both in mind the patient population there.
Speaker Change: Typically have cardiac dysfunction that would be starting out on their myself, probably already have implications on skeletal muscle dysfunction that would make them eligible to benefit from der myself. So you know that remains to be seen how F. D. A.
Speaker Change: Put sport the label, where we have applied for full approval and we'll provide updates when we have our label discussions later this summer there already scheduled.
Speaker Change: I appreciate that Oh, they hopefully data could you.
Speaker Change: Remind us again when do you plan to to disclose that I mean irrespective of what's going to happen in August.
Speaker Change: Yeah.
Speaker Change: And if you get approved and based on hopes to how do we think the label is going to change hypothetically. Once you disclose hope three data an AD for a label expansion.
Speaker Change: Yeah. So we're in a really great position as I said a minute ago with hope three our current plans are are somewhat fluid.
Speaker Change: We're making sure that everything proceeds as planned with this application for the cardiomyopathy, if it does which we fully expect it to then our plans are to take home three to Europe, and potentially to Japan to be able to expand our label globally. As we've talked about that would mean that we would keep hope three blinded add more patients from the.
Speaker Change: Appropriate geographic area and unblinded Windows Ah patients had reached their part the timeline for their primary efficacy endpoint, which would be a year after enrolling into the study so that would change the unveiling of the hope three data if for some reason as the previous questioner just asked there was some reason.
Speaker Change: We needed to move very quickly towards unblinded.
Speaker Change: Ending the hope three data it would be likely in the third quarter early fourth quarter of this year and we would then be able to expand the label in that way either in terms of skeletal muscle implications right now the primary efficacy endpoint is the performance of the upper limb 2.0, we.
Speaker Change: <unk> had preliminary discussions with the FDA, although they were in 2024 that we would ask for approval for a full D. M D as opposed to all skeletal muscle myopathy is down.
Speaker Change: Down to diagnosis, but we have not had full labeling discussions with them nor have we finalized our plans on how that indication would look but it would be for skeletal muscle myopathy.
Speaker Change: Thank you very helpful and the final question is regarding CRV. Walter that you you made eligible to receive in August or September. So would you plan to sell it right away and get whatever the last prices I think $155 million or would you like to keep it for possibly.
Speaker Change: Using for some indications.
Speaker Change: All such us Becker muscular dystrophy.
Speaker Change: Yeah. Our current plan is to sell it you know it will really strengthen our balance sheet and we feel that any indications that would come along we probably would've need a P. R V. We'd got probably priority review anyway for a rare disease.
Speaker Change: So I don't think that would be as helpful to us as the dollars in our bank account.
Speaker Change: Okay. Thank you. Thanks, so much.
Adrian: Thanks Adrian.
Adrian: Again, if you would like to ask a question simply press star followed by one on your telephone keypad.
Speaker Change: Our next question comes from the line of Blue Bloods, Papa happens from Roth Capital. Your line is open.
Speaker Change: Hi, My name is monocyte and I'm dialing in for Bonnie So congrats on your progress Deane and we have a few questions.
Speaker Change: First in your market Research studies have built participants indicated the type of profile that you know would mean they don't mean.
Speaker Change: Best an indication.
Speaker Change: Okay.
Speaker Change: I'm sorry, so are you asking which patients would do best under myself.
Speaker Change: Yeah they have.
Speaker Change: Had the participants indicated the type of you know the profile that would like what my friends would be yeah, that's what I'm trying to ask.
Speaker Change: Yeah, Yeah. So right now what we are looking for in the label as either the presence of L. G, which is late gadolinium enhancement or scar in the heart as measured by MRI, sometimes you can get that I'm very very young in age and the kids don't know what their sort of ignorance is bliss in terms of the damage to their heart, but they need.
To start under myself, whether we can attenuate that progression and or the presence of cardiac dysfunction, which would be measured by an ejection fraction of 55% or less which can be measured in any way whether by MRI or by echo. So the presence of either or both of those situations would allow for the prescribing them.
Speaker Change: Dare myself at least in this first iterations.
Speaker Change: Okay and the next question is like can you provide some color on the progress achieved with the Japanese regulators.
Speaker Change: Yeah. So we're just working with the Japanese regulators now we've actually been in conversations with them for a couple of years. They knew that we were waiting for FDA approval before proceeding in Japan, we have some upcoming meetings with M. D C.
Speaker Change: CRO that we're working with in Japan is a very well known one called C. M. I see and then we're planning to have P. M. D. A meetings either later this year or early next year as we plan on propelling Jeremiah fell forward in Japan, while we have some ideas of what's gonna be required which does not look like it's going to be a too difficult to achieve.
Speaker Change: I don't have specific information does disclose yet on Japan, but we're actively working on that now.
Speaker Change: Okay. Okay. Thank you and the last question is can you kind of you know speak about the outcomes like can you speak to the outcomes Committee composition and expertise and do you regard the outcome is a positive indicator.
Speaker Change: Yeah, so the components or the participants our panelists are the odd comp as available online I don't have it in front of me right now, but you can find it if the cell and gene therapy and Advisory Committee Board. They select from there and then they're allowed to put AD hoc members onto the odd comp obviously, we don't know who those are.
We don't have a date yet for AD com, but we are practicing regularly are the company that we're working with brings and panelists that have curriculum V tasers or resumes that are very similar to the panelists. So that they can ask and answer questions for us and with us. So that we are well prepared and we feel.
Speaker Change: Really good about it.
Speaker Change: Okay. Thank you so much and congratulations again on your progress.
Speaker Change: So much.
I will now turn the call back to capital management for final thoughts.
Speaker Change: Thank you so much for joining today's quarterly call. We look forward to updating you on our progress obviously theres going to be a lot of it over the coming months and we will definitely keep you informed as we move forward operator, you may now disconnect.
Speaker Change: This concludes today's conference. Thank you for participating you may now disconnect.