Q2 2025 Capricor Therapeutics Inc Earnings Call
<unk> will conduct a question and answer session.
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These qualities being recorded on Monday August 11th turning 25.
I will now like to turn the conference over to CFO AGM urban for the forward looking statements. Please go ahead.
Thank you and good afternoon, everyone before we start I would like to state that we will be making certain forward looking statements. During today's presentation. These statements may include.
Statements regarding among other things the efficacy safety and intended utilization of our product candidates, our future research and development plans, including our anticipated conduct and timing of preclinical and clinical study our enrollment of patients in our clinical studies are planned to present a report additional data our plans regarding regulatory filings potential regulatory developments involving our product.
Candidates potential regulatory inspections revenue and reimbursement estimates projected in terms of definitive agreements manufacturing capabilities potential milestone payments and our financial position and possible uses of existing cash and investment resources.
We're 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 within our quarterly and annual reports you are cautioned not to place undue reliance on these forward looking statements and we disclaim any.
<unk> to update such statements with that I'll turn the call over to Linda <unk> band.
Thank you Jay good afternoon, everyone and thank you for joining us on our second quarter conference call.
Our Capricorn, our mission remains unchanged and clear to bring transformative therapies to patients with rare and life limiting diseases.
While this past quarter has presented us with some unique challenges.
It has also reinforced our conviction that we havent Darren myself for the treatment of DMD, the agility agility of our team and of course, the promise of our pipeline.
In particular for the Duchenne community, we remain unwavering in our commitment to deliver the first approved therapy aimed at specifically treating the cardiomyopathy that affects nearly every patient with duchenne muscular dystrophy and remains the leading cause of death in this devastating disease.
Now for the latest update on our biologics license application or BLA.
As previously disclosed we received a complete response letter from the FDA in July.
<unk> stated that the BLA in its current form does not meet the statutory requirements for substantial evidence of effectiveness and also reference certain CMC items, most of which we have already responded to that.
Which were not reviewed by the FDA due to the issuance of the CRM.
As a reminder, approximately one year ago, and our pre BLA meeting with the FDA, we requested to switch the primary efficacy endpoint of our ongoing phase III <unk> III study to left ventricular ejection fraction or L V F.
And the agency responded by encouraging us to submit on currently available data from our hope to and hope to open label extension trials matched to an external natural history comparator and then use the data from our hope three trial to support potential label expansion in.
The future.
This became the plan that we implemented.
While the FDA response contained in the CRM was certainly disappointing we stand behind the strength of our submission and the substantial progress made throughout the review process from the successful pre license expansion or <unk> to completion of our mid cycle review with no deficiencies.
<unk> noted and timely responses to more than 50 information requests from the FDA. We believe we consistently met the agency's expectations throughout the review process.
The complete response letter was unexpected given the trajectory of positive interactions while the FDA continues to evolve under new leadership and its approach to novel therapies to treat rare diseases. We remained focus on working constructively with the FDA to define the clearest and most.
Efficient path forward for <unk>, and the patients who need it.
I would like to emphasize that our hope three trial is still blinded and will not be unblinded until we have clarity on the path to potential approval from FDA.
Let me take a minute to remind you of the features of the <unk> III trial. The study is fully enrolled with the last patient last visit occurring in June of this year.
<unk> three is a double blind placebo controlled clinical trial with a one to one randomization, which enrolled 104 patients consisting of two arms cohort a and cohort b.
The combined power of this trials using both cohorts is greater than 90% with the original primary efficacy endpoint being the performance of the upper limb or the poll version 2.0.
Based on a multitude of reasons not the least of which is the tremendous unmet need of DMD cardiomyopathy. We have submitted a protocol amendment to designate left ventricular ejection fraction or Lv app as the primary efficacy endpoint and the skeletal muscle endpoint performance of the upper limit.
Paul as a pre specified secondary endpoint.
This change is based on multiple factors one the objectives of Lvs as measured by cardiac MRI remember there is no volition and cardiac function.
As measured by MRI as well as the relevance of left ventricular ejection fraction to the pathophysiology of DMD cardiomyopathy, which has only been recently elucidated by the work of Dr. Jonathan Zaslow from Vanderbilt University, and the DMT cardiac consortium and a study funded by the office of orphan products of the <unk>.
And the NHL <unk> and <unk>.
In addition, <unk> III is well powered to detect a treatment effect on cardiac function.
I want to remind you the Capricorn developed Jarrod micelle, specifically to address heart disease.
Particularly the cardiomyopathy associated with TMT. However.
However, until Doctor's office study and its subsequent publication there were no established efficacy benchmarks and DMD cardiomyopathy.
F D a to use and defining clinical benefit.
Our entire regulatory path, including the current BLA was built on the Fda's guidance on how efficacy should be defined in this patient population. We've always intended for ejection fraction to serve as our primary efficacy end point. So while this may appear to be a change in strategy. It is in fact, a return to the original.
Goals, we set early in the development of Derm myself.
To that end with our type a meeting with the FDA now scheduled we have submitted a comprehensive briefing package that addresses the concerns raised in the CRM and outlined several potential paths to approval.
These include first and foremost the continued review of our previously filed BLA.
Which we believe makes the applicable regulatory requirements for approval.
As well as supplementing the current BLA with additional data from hope three if needed.
We believe the current handling of our submission is inconsistent with our interpretation of the FDA written guidance is for cell and gene therapies as well as recent public statements addressing the approval of safe and effective therapies for rare disease populations. We are hopeful that FDA will exercise.
The patient focused and science, driven approach and rare disease approvals and which they have been emphasizing in the media as well as highlighting in the FDA direct podcasts.
Just on the comments of Secretary Kennedy and Commissioner Macquarie.
Proven Darren myself for the treatment of DMD cardiomyopathy seems directly in line with their goals.
In conclusion about a year ago, we received FDA feedback that shaped our decision to submit the BLA based on cardiac endpoints.
We provided the requested data and analysis and fully expected any differences in interpretation to be addressed at an advisory Committee meeting Wanda was ultimately canceled by the FDA without explanation.
We're concerned with how our file has been manage because we believed there were opportunities during the review period for the agency to raise the specific issues cited in the CRO before issuing a letter.
We've long worked aside the DMD community and understand their calls both the continued treatment with Durham, ISO and to gain access to becomes commercially available.
We will continue to urge the F D. A to recognize a cardiomyopathy is a leading cause of death in DMD and a far more severe consequences from the loss of arm function.
<unk> has demonstrated a strong safety profile and the data indicate it can help stabilize the inevitable decline in cardiac function for people living with DMD.
With regard to the CMC and commercial App pre commercial aspects of our program I am pleased to announce that the FDA has now formally accepted all 483 items from our pre license inspection.
This milestone further validates the strength of our quality systems manufacturing capabilities and overall commercial readiness.
In addition, the CMC related items noted in our CRO have either been addressed prior to the issuance of the CRM or have been internally addressed since we are prepared formal responses, which we plan to submit with our response to the CRO.
Our manufacturing facility in San Diego remains fully operational and in production and we are being disciplined in our commercial manufacturing investments to ensure we are fully prepared while managing resources wisely.
In parallel we are diligently and strategically investing in launch readiness activities, including physician education patient services market access planning and reimbursement.
We've also begun working closely with treating physicians across the field of neurology and cardiology, who will ultimately collaborate and prescribing Jeremiah sell to patients with DMD cardiomyopathy if approved.
Many of these clinicians are already familiar with the therapy through their participation in the hope to and hope three studies and we are committed to ensuring a smooth transition to commercial use if approved at.
At this time, we are focused on seeking approval for <unk> in the U S and with respect to our global expansion plans, we will provide updates as they become available.
Now turning to our Exosomes program to remind you in 2024, we were selected to participate in project Nextgen and initiatives led by the U S Department of health and human services aimed at advancing next generation vaccines for COVID-19, and other potential infectious diseases under.
This program the national institutes of allergy and infectious disease and it.
It will be sponsoring the phase one clinical trial of our stealth ex vaccine.
Within the last several weeks, we reached a significant milestone for this program, which was the clearance of the IND.
And initiation of the trial using stealth ask our <unk> platform technology.
A phase one study is being conducted at and overseen by Nia Ids Division of micro biology, and infectious disease D. M D.
And I am pleased to report we have already supplied them with our clinical material for use in the trial.
The phase one study is assessing our COVID-19 vaccine product. The trial is divided into three arms comprised of three escalating doses of the spice bike or S antigen.
And a combined high dose S plus a <unk> <unk> or <unk> antigen.
The multivalent vaccine we have been developing.
<unk> is starting with the S. First because previous Covid vaccines are mainly app based and they wanted to have a basis for comparison with our vaccine candidate using similar antigenic profile.
The end goal is for the adoption of the N plus S, which is our multifamily vaccine and we will provide more updates on this developing program as they become available.
We believe that <unk> has the characteristics of a vaccine product that secretary Kennedy would find acceptable at kantar.
No adjuvant. It is not mrna based uses a native protein antigen and can be rapidly produce if needed. We have long believed that this type of vaccine checks all the boxes for a safe and effective platform as supported by multiple preclinical studies.
In upcoming clinical data will allow us to confirm or challenge that hypothesis.
This platform also has the potential to address multiple disease areas, including influenza and RSV.
While vaccines are not a core focus for us if our candidate meets U S government criteria and demonstrates efficacy it could potentially open meaningful business development opportunities.
Just as importantly, it would serve as strategic proof for our <unk> platform, which we hope to advance as a versatile therapeutic engine for rare diseases and beyond.
While the majority of our efforts this year have been focused on securing approval for Durham, ISO and additional reason we recruited Dr. Michael banks as our Chief Medical officer with his expertise in translational science and medicine.
He is now leading efforts to advance our <unk> pipeline with the goal of forming strategic partnerships to expand the platform into and beyond vaccines. We.
We believe the differentiated features of exosomes, including low Immunogenicity scalable manufacturing and targeted delivery physician capricorn for unique potential opportunities and the therapeutic delivery space.
We look forward to sharing updates as they become available.
Thank you and with that I will now turn the call over to a J to run through our financials.
Thanks, Linda this afternoon's press release provided a summary of our second quarter 2025 financials on a GAAP basis. We 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, let me start with our cash.
Position as of June 32025, our cash cash equivalents in marketable securities totaled approximately $122 8 million.
Turning into the financials revenues for the second quarter of 2025 years zero compared to approximately $4 million for the second quarter of 2024.
Additionally, revenues for the first half of 2025 or zero compared to approximately $8 9 million for the first half of 2024.
To point out that the source of revenue for 2024 was the ratable recognition of the $40 million. We have received under our U S distribution agreement with Nippon <unk>, which has been fully recognized as of December 31 2024.
Moving to our operating expenses for the second quarter of 2025, excluding stock based compensation, our research and development expenses were approximately $20 1 million compared to approximately $11 7 million for Q2 2024 for.
For the first half of 2025, excluding stock based compensation, our research and development expenses were approximately $36 3 million compared to approximately 21 8 million for the first half of 2024.
Moving into general and administrative expenses, excluding stock based compensation were approximately $4 million in Q2, 2025, and approximately $1 8 million in Q2 2024 and for the first half of 'twenty. Five also excluding stock based compensation, our general and administrative expenses were approximately $7 million for the first half.
25, and $3 6 million for the first half of 2024.
Net loss for the second quarter of 2005 was approximately $25 9 million compared to a net loss of approximately $11 million for the second quarter of 24, the net loss for the first half of 'twenty five was approximately $50 3 million compared to a net loss of approximately $28 million for the first half of 2024.
Now I'll turn the call back over to Linda for some closing remarks again, thank you AJ.
Just to reinforce ages points on our financial position with over $120 million in cash we are well positioned to support operations into late 2026 and continued to advance our key pipeline objectives. Additionally, if we receive approval we would still be eligible to receive a priority review voucher as well as.
The milestone payment of $80 million for <unk>, representing additional significant non dilutive capital opportunities to further strengthen our balance sheet.
This is an important moment for Capricorn, while we have faced recent regulatory headwinds, we are advancing deliberately strategically and with confidence in our data our team and our path forward. We continue to believe that <unk> represents a major step forward for patients with DMD cardiomyopathy and that our <unk> platform is well positioned to do.
Oliver value through continued innovation and partnerships.
So the Duchenne muscular dystrophy community. Thank you for your ongoing trust and support we remain grounded in the science is on execution and committed to building a company that delivers meaningful and lasting impact for all DMD patients I will now open up the line for questions.
Thank you.
Ladies and gentlemen, we will now begin the question and answer session.
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One moment. Please for your first question.
Your first question comes from Ben Hough from Piper Sandler. Please go ahead.
Great. Thank you so much for taking my question.
I appreciate all of your hard work.
The key part of it for these boys and get some money therapy, that's going to help them.
With their hearts heart function.
I wanted to get a sense for sort of.
The plan for <unk>.
The next steps in terms of.
Unblinded hope three is the plan to sort of just come from mutual from the FDA.
I'm not first.
Maybe sort of reiterate.
What's the plan there.
Thanks, Ted it's always a pleasure even during these crazy Crazy times, Yeah. So we are waiting for adjudication from FDA.
As to what their requirements will be for both three and then we will submit our statistical analysis plan and proceed with unblinded only after that has been accepted we just don't want to muddy our cloud the waters with any thoughts that we had unblinded early so our plan is to stay quiet until we have plans for.
Them.
Great.
And when do you expect to hear back from the FDA or Joe factory.
Well, obviously from the rescheduling of our earnings call today or type a meeting as this week.
We anticipate to have a really good conversation with the FDA. We're looking forward very much to meeting with them. We certainly August as our month August of 2024 was when this whole plan was put in place. So I'm very excited for this meeting.
And in terms of providing clarity and updates to the markets.
It won't be until I get the official feedback and writing, especially with the liability of our current situation and times.
So hopefully Adam <unk> will report on it before I do there.
Thanks, so much Glenda good luck.
Thanks, Tim.
Thank you. Your next question comes from Leland <unk> from Oppenheimer. Please go ahead.
Thanks, Linda for the update and taking our questions. Just a couple here. So just maybe further from from touching clients. So.
If you grow with the plan.
<unk> online training.
Let's continue with the current DLA as it is would there then be supplemental OLED.
That could go into <unk>.
The FDA has on file versus what had been submitted I guess, if you could share just what incremental data.
Ex <unk>.
Become available to them that would be.
The adjustments from what they had reviewed previously.
Yeah, Leland, so actually I haven't really thought about submitting supplemental data flow of course continues to support our safety profile and the efficacy. If you look at the long term efficacy of Durham, ISO is actually quite extraordinary and we're very proud of that record in our OLED patients and I don't know of another clinical effort India.
That has as long of a record post.
Study is as we do having said that.
The meeting with FDA will define what they want for data for either the reopening and resubmission of the BLA.
So I don't have an answer on that my current plan is to have everything ready then they have an opportunity to select what they think would be most efficient in determining efficacy for approval.
Okay.
I guess.
I guess I'll have to ask April sorry about that.
<unk> how does.
Does that does that inform your thinking is.
Are the people who you are interacting with the FDA was there a different team now.
Nicole overdone out of the picture.
The last few weeks, how should we think about kind of who you're kind of counterparties or with SG&A at this point.
Thanks, Leland you know it's interesting I think the last few years I've been thinking about this a lot people have become much more focus on who the review team is and exercising political capital and regulatory flexibility and all of this window that has become very popular.
I'm going back to old school, we have good clinical data we have great safety data, we have guidance from the agency in writing as to what they wanted we provided it.
And now really I think it's up to them to decide who is best suited within that agency to make the decision of adjudication and all of the factors that go into it. So I tell my team I'll tell the markets. We are proud of our data we see it in terms of the long term efficacy and our patients and safety and we sincerely.
That the agency gives us a good path forward to get this to those patients as quickly as possible.
Okay, alright, thanks, very much for taking my questions.
Thanks, Leland always a pleasure.
Thank you.
The next question comes from Joe <unk> from H C. Wainwright. Please go ahead.
Hi, Linda and a J thanks for taking the questions good afternoon.
So just to sort of press the envelope a little bit obviously things could change.
Mentally are dramatically this weekend about two months after that when you provide the details from the minutes to the street, but with language in the press release and what you talked about today you're talking about.
Resubmitting in its current form.
Maybe not really having any incremental data from the OLED that you just discussed and the last question. So what would you expect that could be potentially different.
Yeah.
Well again, you know we have given the FDA a variety of opportunities in our briefing documents.
We remain open to a resubmission of the BLA as it is and we didn't feel that the CRM was founded.
The data that we submitted was exactly what they ask for and the graphs themselves are interpretable as a statistically and clinically significant so thats of course, our number one goal is explaining to them why perhaps our interpretation was wrong. There was then everything that follows from that accelerated approval with the submission of of hope three data has supported that.
And a variety of other opportunities that they can help us adjudicated and that's why we're looking forward to this type a meeting so with that with that in mind.
We go in with open Hearts to meet with the agency and look forward to having them understand that developing and approving therapeutics for rare diseases, especially pediatrics one does require.
Youre looking at the data in a more holistic fashion and we're hoping that that is exactly what happened this week.
Alright, so that's it.
That's fair and so maybe just another question starting at the or.
From the back end of your comments around stealth X. So it's great that it's getting into the clinic now.
[laughter] any visibility with regard to.
What might be next with regard to an indication obviously, you mentioned influenza as a potential and.
How would you describe the.
Early talks maturity levels potential BD around stealth docs.
Yeah, Thanks, Joe so.
I felt like program is sort of our little engine that could you know we just kept on moving it forward our vaccine program is really exciting and the.
The sense that as I mentioned in my remarks, this type of vaccine, which we've always believed in is exactly what secretary. Kennedy has advocated is would be a much better vaccine candidate no AD events native proteins rapidly produce no mrna that kind of thing. So we're very excited about that our looking forward.
The NIAID data, they're very excited about it because it's a program that does fit that criteria and we will see where that goes in terms of copper cores interest in developing vaccine technology, that's something that I've always said would be a business development opportunity. We think it's wiper than ever based on the criteria I just put forth in terms of therapeutic.
<unk>, we haven't disclosed some of the ones that we've been working on internally as I've mentioned many times, we focus most of our efforts and our capital on Durham, ISO and look forward to providing updates on where we're going to be taking the <unk> program as.
As we further develop that therapeutic pipeline.
Got it looking forward to more visibility out of the program and good luck with the meeting this week.
Thanks, Joe always a pleasure.
Thank you. Your next question comes from Kristen <unk> from Cantor. Please go ahead.
Highlander and AJ also sending you best wishes for your meeting this week.
Questions for me first I was pleasantly surprised to see that you received the acceptance is of responses related to the form 43 observations I guess can you just comment on that because typically after we see <unk>.
Hey isn't so much as engaged in responding to those things until you resubmit. So can you.
Explain that timeline for us.
Yeah. So you know this has been an unorthodox review process of <unk>.
I mentioned in my remarks, as we've talked about in our disclosures and other companies have gone through similar situations. So we passed are they issued the 400 threes. We responded to the <unk> in that review team.
Independent of the CRO, providing feedback that we had cleared our 480 threes than we are on path for approval of our CMC and our manufacturing plant for <unk>. So.
That is where that situation.
Situation is as I mentioned in the CRO there were.
Several here several several issues related to CMC. Many of those had already been addressed an information request responses that we provided prior to the issuance of the chaparral, but they had stopped reviewing in anticipation of issuing the <unk>. The rest of them have already been addressed and we look forward to providing those in our response to the <unk>.
Ro.
Okay. Thank you and just want to confirm that the new timeline guidance for Q versus <unk>. Three is just solely driven by the fact that you haven't started the unwinding, yet because again youre waiting for this meeting and that clarity.
Absolutely, yes, yes, everything's on time with with hope III.
And we just are waiting for feedback from FDA.
Thank you and then just lastly, I guess in a nutshell what are the key things that you hope to align from after this meeting takes place. This week is it just understanding specifically what's required.
It is hope three do you expect to have full understanding of what that primary endpoint will be and then you know.
Even there kind of a blessing that it hoped there is successful on that endpoint that could potentially be sufficient enough to support an approval. Thanks again.
Yeah, So exactly what you hypothesize sewer, we're looking for feedback on exactly what it's going to take to get this approved as I mentioned in a previous question. We believed in the data that we submitted we believe that the <unk> was unfounded, we were going towards the <unk>.
Very directly.
Because there were no issues raised in the mid cycle review meeting we thought we were.
A pretty good position when the AD comm was canceled I didnt really take too much issue with that because I felt like in the late stage meeting we would be able to address any concerns. They might have so my first plan is to try and understand what their resistance is to the currently available data and if that is maintained then what it will take to get.
Two approval.
Thank you sending you the best.
Thank you so much.
Yeah.
Thank you.
Your next question comes from Matt <unk> from B Riley Securities. Please go ahead.
Okay.
Thanks for taking our question.
I was just curious has there been any informal communication I believe post <unk>.
Notice there was an opportunity for informal teleconference and just wondering.
What the takeaways were.
Are there.
Kind of contributed to your decision to resubmit. Thank.
Thank you.
So we did have.
Sure and formal meeting with them based on their guidance from their leadership. It was primarily to align on timelines of this type a meeting.
And what would potentially be submitted and then to clarify the CMC related issues, neither clinical north that.
We're part of that meeting so the type a meeting is very important because it allows us to meet with the agency and really flesh. It out in terms of our decision to reply or respond to the Sierra that's always been our intent.
Wed like feedback from them. So that we can keep it smooth and steady and work our way to the quickest date of a produce as possible.
<unk>.
We will see what happens this week.
Got it thanks, and then do you expect to get an answer on the primary endpoint change at the August meeting I believe you said you were expecting.
So Matt.
Yes, so thats been one of our primary questions. We are looking looking to get that.
Worked out during this meeting.
Got it good luck this week at the meeting.
Thank you.
Thank you. Your next question comes from Irene Haas from logging into pharma. Please go ahead.
Hi, good afternoon.
Hey, Jason Thanks for taking questions and push it all the work Youre doing.
A couple from us so first I want to clarify on apologize if this has been addressed.
Clarify the time line.
Of the upcoming events. So the fourth quarter or are you going to readout Humphrey resubmit, the BLA and how do we how do we treat <unk>.
If it is the same BLA and what does the review process typical awful for these kind of re applications two months.
Sort of shutdowns than review sort of months just curious your thoughts on the timeline.
Yes, so thats one of the issues that we're going to be taking on with the agency. During this meeting obviously.
There's a lot of timeline issues that were predicated on.
How they want to update the BLA, whether or not they would require a new BLA what that does to priority review we are still.
Eligible for the PR via the voucher that comes with approval for a pediatric indication independent of timeline, but in our our model allows us certain.
The benefits in terms of submission and also return on feedback, but we'll have to figure that out during this meeting with them and we'll disclose that as soon as it becomes available to us.
Okay I appreciate that.
Regarding the electrician corner ejection fraction as the primary efficacy endpoint for <unk> could you walk us through your thought process as to why you chose the standpoint, I think or.
We hope to have multiple cards okay.
One <unk> measures.
And could you also remind us of any prior successful or unsuccessful.
Ensure cola ejection fraction primary endpoints ambitions also of which a model based on those principles.
Yeah, really really important questions. So lessened tricolor ejection fraction is obviously one of the most important indicators of cardiac function and clinicians cardiologists use it all the time to sort of define where their patient sit in terms of cardiac function and also what theyre likely outcomes are going to be so cardiologist know that blow it.
Above certain points here either.
Greater risk for morbidity and mortality or.
Or in a reasonable position for stabilization of your cardiac function, we've known that for a long time.
The reason that ejection fraction in this answers your last question along with your your first one has not been used as a primary efficacy endpoint in clinical studies is because up until recently, it's really been considered a surrogate. It has not been directly tied to clinically relevant events such as mortality hospitalization exercise performance those types of things.
<unk>.
The most amazing thing and this is why the change in our submission occurred from a clinical endpoint of skeletal muscle to cardiac ejection fraction was in collaboration with the agency because.
Everybody knew the cardiomyopathy was the leading cause of death currently for Duchenne muscular dystrophy up until John Softwood study with the cardiac consortium there really.
We're not.
General evidence of what would be the predicting facts for mortality hospitalization those kinds of things and in Duchenne, It's super hard youre dealing with a rare disease with a small patient population and a pediatric disease. So in order to do a mortality study you'd probably have to do like a 20 year study globally in order to be able to gather enough information so the op.
A orphan products understood. This paradigm, where paradox has there been any NHL beyond national heart lung and blood Institute, So they've bundled John study, which allow them to look at what would be the predictive factors.
Either morbidity or mortality in Duchenne muscular dystrophy, cardiomyopathy, and along with what our data suggested ejection fraction was the most important features so theres also left ventricular end systolic and diastolic volumes, we're measuring those those are secondary endpoints as well as some biomarkers that John had which was like BNP and.
One or two other paradigms.
Other end points that would suggest the paradigm of morbidity and mortality and what we actually were able to demonstrate with the presentation of this data is that Jeremiah sell attenuated and may have even reverse the path of that decline in ejection fraction, therefore, predicting morbidity and mortality because again high.
Highlighting what I just said is a pediatric disease that is rare during those types of large studies there were sometimes require thousands of patients to look at mortality risks. This is good for rare diseases. So the agency at the time and hopefully still is willing to understand that ejection fraction is probably the best way of predicting where this pace.
<unk> population could go should it not be stabilized.
It is very helpful. I appreciate it.
Just just to summarize this maybe.
Essentially LDF was a surrogate endpoint.
It requires a lot of sort of innovative thinking on <unk> to make it look a primary endpoint going forward would that be sort of a fair summarization.
I don't agree because again with the.
Understanding of the new data that has come forth Andy again rare disease population. There really is no other opportunity for adjudication of our primary efficacy endpoint. So if you really want to hear I'm passionate about this as you can probably tell from my voice, but if you really want to understand.
The risk and benefit here, please listen to our parent project muscular dystrophy webinar that we did about a week ago. Dr. <unk> talked about the unmet need in cardiomyopathy, and what Dr. Rolla, who see these patients all day everyday at Cincinnati Children's talks about is there is no other way of measuring efficacy in this particular patient population that would be fair and.
Safe for human beings.
Very helpful. Thank you so much and one final question on Becker's muscular dystrophy My favorite is one.
Part of our model a model that is based on that so with all of these discussions.
Arnaud takeaways.
For BMD.
Would you have to volumes sort of another some hope cities five large trial in DMD to get similar.
Potential sort of label us as in DMD, and what's left ventricular ejection fraction.
Also sort of a primary endpoint for <unk>. Thank.
Thank you.
So sideways to answer to that because I don't know directly at this point what the agency will require it's very early in this administration to understand what theyre actually going to do and moving rare disease approvals forward. Our planned previously which I have discussed with you.
And discuss publicly as well is that we were going to try and use the duchenne data to build the Becker program because the pathophysiology of the cardiomyopathy is identical just somewhat slower progressing in the Becker patients and in fact as a banker patients get older and becomes more and more of a risk factor for morbidity or mortality.
I don't know my current planning with Becker, because I need to get understanding from the agency of how theyre going to view the current Duchenne data and then I'll be able to make more educated comments on it as high as they are cheap clarity there.
Thank you. Thanks, so much for your comments on good luck. Good luck with go against what the FDA. Thank you.
Thank you so much we really appreciate it.
Thank you.
Your next question comes from Bob <unk> from Roth Capital. Please go ahead.
Yeah, Thanks for taking the call and I'm, a monoszon dialing in for Bob Dylan. So we have a couple of questions. The first question is so do you regard the upcoming Taipei meeting is an opportunity for the FDA to clarify data to change of stance with respect to that only ourselves BLA or a bellwether for the investors in predicting the future outcomes.
Of potential Resubmission with the hope that data, although we are curious to know whether you'd be open to sharing the diabetes meeting minutes to investors to the extent you can you know to be comprehensive and elaborate.
Yeah in terms of your first question.
Yeah.
<unk>.
We expect.
Sure the data is as good.
As it becomes available and.
The information as it becomes available.
In terms of provide.
Providing meeting minutes to investors.
That becomes a little bit of a.
As needed basis I can tell you right now as you heard our cash position is very strong we're not out raising money, we don't anticipate needing to raise money and focusing on approval of <unk> in DMD and so.
We'll see if the situation calls for we would we would definitely.
Discuss it directly with that Investor.
Okay.
Other question. So some investors are wondering about the scope and a pragmatic value of the only and the mid cycle EBITDA of your processes.
In relation with the overall review process. So what are your general thoughts on that.
I'm sorry could you ask that question again, I'm not sure I understood the question.
Okay. So some investors are wondering about the scoop and the pragmatic value of the early and the mid cycle F. D. A to have your processes you know in relation with the overall W. Process. So what are your general thoughts on that.
While we have a BLA was accepted and we had a very successful mid cycle review. So I guess the takeaway for investors is.
Our situation anyway, it wasn't predictive of what was coming next.
Okay and one last question. So in terms of the ex U S. You know clinical pathway for development on for approval, particularly in the U K. We were wondering if from category and what could be eligible to take advantage of the IRB to CPU gay authorization at some point provided though a future FDA decision.
Favorable so.
What was what decision.
So we were wondering if you know in terms of the ex U S clinical pathway, where they'll Catholic order could be eligible to date would be I'd be which is the international recognition procedure to seek though you'll get authorization at some point.
Given that you know the future FDA decision is going to be favorable.
Yeah. Thanks, So as I mentioned in my prepared remarks.
We're focusing on U S approval right now our global strategy is.
As emerging a lot of it will be based on some of the.
The feedback we received from FDA and what our path forward is and please stay tuned we'll provide updates on our ex U S strategies as they become available.
Okay.
So much and best of luck here.
Thanks.
Thank you. Your next question comes from Catherine Novack from Jones. Please go ahead.
Hi, and.
Thanks for taking my question.
So one question was when the FDA responded to your request for a type a meeting gave you. The date did they give any substantive reply to your meeting requests.
Particularly around positive or negative wording around the Lv app.
No. So it typically when they accept the meeting requests they just say sector meeting request and then they send over a day, we submitted a briefing package and we're awaiting feedback on that and.
Bill will discuss the ramifications of what we asked for and what they respond to the meeting.
Got it.
And then the <unk>.
R&D expense for two Q, what accounts for the increase was a buildup of product inventory ahead of potential launch or is that going clinical studies and we should expect to see continued growth on that line.
Yeah. Thanks Catherine.
It's both it encompasses a little bit of both of what you said, we're obviously in the end stages of hoped three but those patients of 104 of them or are ongoing in non clinical development expense line item. We're also obviously preparing for the CMC endeavor.
So thats, where it is that obviously, when we get more clarity and feedback from FDA and announce more plans I think we'll have more granular items on the burn rate moving forward, but it really encapsulates both both of those areas, but that's the main areas of spend.
Okay.
Alright. Thanks.
That's it for us.
Thank you. Thank you.
Thank you.
Ladies and gentlemen, as a reminder shows you have a question. Please press star one.
Your next question comes from Matthew financier from AGP. Please go ahead.
Hi, Linda Hi, AJ, Thank you for taking our questions.
So just looking for a little clarity on.
The FDA review process to date has obviously, there's been turnover since the new administration, but has there been any turnover since prasad.
Leaving the FDA and then him coming back.
And has the team that you have been engaging with the FDA changed at all in that time.
So we don't know we'll know more of this week so.
The big change with Doctor for Southern living and now Dr. Prasad returning we don't know the ramifications of his exit or a return on our program, but what I can say for sure is that we're looking forward to working directly with him and with the team and.
And we do not.
I think that the data should be interpreted differently by any by any teammate that are different.
Got it.
And just a little bit on the run rate do you expect it to taper off in 2026, once you kind of get clarity regulatory wise.
Potential launch wise as hoped III wind.
Wind down.
Sure.
Come down and maybe G&A go up a little bit.
Yes, I think Thats fair, Thanks, Matt I mean, obviously again as I articulated what the next steps would hope three is a big aspect to that but should we achieve approval, we'll have some some pretty serious capital injections around the potential sale of the <unk> and $80 million from the bunch and yaacov that will allow us of course to invest in CMC expansion.
Everything we want to do around the launch for commercial endeavors. So that's kind of how we're looking at it obviously more granular level can be discussed in the future, but we expect the capital to go right, where it needs to be which is preparing for the launch.
Alright got it. Thank you guys for taking my questions and good luck it sounds like that meeting.
Thanks, Matt.
Thank you.
Thank you. Your next question comes from Chris <unk> from Nomura. Please go ahead.
Yeah.
Oh.
No.
That's the type a meeting.
I'm sorry.
Okay.
Oh.
Yeah.
Alright, well. Thank you so much I guess, we lost the question I want to thank you for joining today's call. We look forward to updating you on our progress over the coming months. Although this is a big week for us. So we will update as soon as we get feedback from the FDA and look forward to a positive review of Durham ISO Thank you so much and.
Have a great day.
Ladies and gentlemen, this concludes today's conference call. Thank you all for your participation you may now disconnect.