Capricor Therapeutics (NASDAQ:CAPR) held its third-quarter earnings conference call on Tuesday. Below is the complete transcript from the call.
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Summary
Capricor Therapeutics is in the final stages of FDA review for its BLA for deramiocel, aimed at treating upper limb impairment in Duchenne muscular dystrophy (DMD) patients over nine years old.
The company submitted a major amendment to its BLA with additional data, including a 24-month open-label extension from the HOPE-3 trial, and believes this supports the efficacy of deramiocel.
Financially, the company reported a net loss of $42.7 million for Q3 2026, primarily due to pre-commercial activities and manufacturing scale-up, with $198.5 million in cash and equivalents.
Capricor is preparing for commercial launch, with a San Diego facility ready to support initial supply and plans for expansion if approved. The company is also managing a dispute with NS Pharma regarding distribution rights.
Management highlighted the robustness of the efficacy data across various analyses and emphasized their readiness for FDA approval and subsequent market launch.
Full Transcript
OPERATOR
Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics third quarter 2026 conference call. If at any time during this call you require immediate assistance, please press star zero for the operator. This call is being recorded today, Tuesday, November 10, 2026. I would like to turn the conference over to CFO AJ Bergman for the forward-looking statement. Please go ahead.
AJ Bergman (Chief Financial Officer)
Thank you. Good afternoon. Before we begin, I’d like to remind you that any statements made during today’s call are not historical and are considered to be forward-looking statements. Actual results may differ materially from those indicated by these statements as a result of various important factors including those discussed in the Risk Factors section of our most recent annual report on Form 10-K, our most recent quarterly reports on Form 10-Q, as well as other reports filed with the SEC.
Any forward-looking statements represent our views only as of today, November 10, 2026. Replay of the call will be available on our website following its completion. With that, I’ll turn the call over to Linda Marban, CEO.
Linda Marban (Chief Executive Officer)
Good afternoon everyone and thank you for joining us. Let me remind you that as Duchenne muscular dystrophy progresses, boys and young men lose the use of their arms and hands, the ability to feed themselves, use a phone or computer, or handle the everyday tasks that keep them independent. The loss of independence is what deramiocel is designed to slow and is the focus of the refined indication now under FDA review. This quarter we took important steps to strengthen that case and I will walk you through them today.
Our BLA for deramiocel is in the final stages of FDA review. Out of respect for that process, I will keep my comments on our interactions with the agency to what is already public. Today I will cover four areas, our regulatory status, our data, our commercial and manufacturing readiness, and our dispute with NS Pharma. And then we will close with a brief pipeline update before handing it off to AJ first let me talk about our regulatory status. Following the Advisory Committee meeting, we held discussions with the agency. FDA then agreed to review additional data and a major amendment to our BLA and extended the PDUFA target action date to November 22, 2026. In August we submitted that amendment which included approximately 30,000 pages of documents, reports, tables, listings and files and data sets. It included the 24 month open label extension data from HOPE-3 along with additional sensitivity and robustness analyses.
It also included an integrated summary of efficacy combining HOPE-2 and HOPE-3 efficacy data. Taken together, we believe these data support the refined indication the treatment of upper limb impairment in patients with DMD ages greater than nine years old, the population studied in HOPE-3 and the focus of its primary endpoint. At this time, all requested information has been submitted to FDA and the review remains ongoing. Now let me turn to our data. I would like to walk you through the full data set in chronological order. The one year randomized results, the two year data we provided in the amendment and what we believe the totality of the evidence tells us. The full HOPE-3 results published in The Lancet this past July following extensive peer review showed that the trial met its primary efficacy endpoint, a statistically significant slowing of upper limb disease progression as measured by the Performance of the Upper Limb 2.0, a validated measure of shoulder, arm and hand function with a p value of 0.029.
The trial was designed to detect a difference of approximately 1 point, a threshold that physicians who treat Duchenne and the FDA have described as clinically meaningful, and that is what we saw. The absolute difference between the deramiocel and placebo groups was 1.1 points, a result that was both clinically meaningful and statistically significant for a boy who can no longer walk. A single point on this scale can mean keeping the ability to feed himself, bring a hand to his mouth or operate his wheelchair or telephone.
Other secondary endpoints measuring upper limb function, such as the mid level Performance of the Upper Limb and the Duchenne Video Assessment, told the same story. HOPE-3 also evaluated cardiac outcomes this past weekend, Dr. Jonathan Soslo of Vanderbilt University presented the HOPE-3 cardiac data at the American Heart Association Scientific Sessions in Chicago. As we announced yesterday, the abstract received the American Heart Association’s Outstanding Research Award in Pediatric Cardiology. This is a significant achievement and to our knowledge, a first for research in DMD cardiomyopathy. As a reminder and the slides are available on our website.
Compared with placebo, deramiocel slowed the progression of heart muscle scarring as measured by LGE on cardiac MRI. Across all patients, the difference in ejection fraction did not reach statistical significance. However, in patients with existing cardiomyopathy, ejection fraction improved by approximately 2.8 percentage points relative to placebo. Of note, patients with preexisting cardiomyopathy were a prespecified subgroup in the HOPE-3 clinical trial.
The findings were also supported by a post hoc comparison to the natural history data set collected by the Duchenne Muscular Dystrophy Cardiac Care Consortium and led by Dr. Soslo. Now let me discuss the open label extension data from year two of our HOPE-3 clinical trial in October, we presented the 24 month HOPE-3 data at the World Muscle Society Congress and in Hiroshima, Japan. In the second year, all patients were eligible to be rolled over into an open label study and 82 patients received deramiocel for a full year, which led us to ask a simple question. What happens when patients on placebo start treatment? In their first year, patients on placebo lost about 2 points on the Performance of the Upper Limb, which is consistent with natural history.
However, when those same patients started on deramiocel, their decline in their second year slowed to about half a point, a difference of 1.56 points comparable to a clinically meaningful change observed in the randomized phase of HOPE-3. This also represents a 76% slowing of disease progression from year one on placebo to year two on deramiocel. In other words, disease progression slowed substantially once patients initiated deramiocel treatment.
Importantly, patients who started on deramiocel in the randomized portion of the trial lost less than one point in year one and maintained a similarly slow rate of decline in year two, supporting the durability of the treatment effect. We also compared both groups at two years to what natural history would predict for a similar patient population using two well known data sets. By month 24, both groups had declined less than predicted, 3.4 points against the predicted 5.9 points in the group treated from the start and 3.8 points against 5.4 points in the group that started on placebo.
The slide depicting this data is available on our website in the HOPE-3 OLE presentation from the World Muscle Society. Most importantly, the placebo group had a similar slope of decline to the natural history group until they went on deramiocel and then the difference was striking. While these open label results are descriptive, they are consistent with our randomized data and support the potential of deramiocel to slow Duchenne muscular dystrophy progression.
FDA has increasingly recognized the role of real world evidence and has recently approved several products where natural history data played an important role in establishing efficacy. That brings me to the full body of evidence and to its strength. Because we know the Advisory Committee left many people with questions, the concern we heard most was fragility, the worry that a result might depend on a few patients or a single analysis choice. So we put our data to the test and we shared that work with FDA. In terms of these robustness analyses, the answer was clear. The size and direction of the benefit held up across the analyses.
For instance, the robustness analyses submitted to FDA ran more than 900 different reanalyses using the HOPE-3 data. To answer one question, how fragile were the results? The analyses addressed multiple questions raised by the FDA and the Advisory Committee, including how missing data is handled, which patients are included, and how progression is defined. If the results were fragile, the benefit would shrink or disappear. However, in the large majority of these analyses, the result was or at or near statistical significance with a p value of p equals or less than 0.05.
Across the analyses, the estimated benefit stayed centered on about 1 point on PUL 2.0, a level that is clinically meaningful. And every analysis favored deramiocel. Not one favored placebo. We also included an integrated summary of efficacy or ISE in the major amendment which brings together data from HOPE-2 and HOPE-3. An ISE is generally included in BLA submissions. In the ISE, we analyze the data from our two placebo controlled trials, HOPE-2 and HOPE-3, which used the same dosing and the same assessments with more patients. The picture became clearer at 12 months. Measurement of skeletal muscle function using the same PUL measures showed that those patients on placebo lost about two points of upper limb function while the patients on deramiocel lost less than 1, a combined difference of 1.29 points and a p value of 0.0087.
All eight sensitivity analyses pointed the same way. On the cardiac side, the integrated analysis of ejection fraction across both trials favored deramiocel by about 2.2 percentage points and the direction held across the sensitivity analyses. Put simply, the data tell a consistent story one year, two years, two randomized trials and a decade of clinical experience all points in the same direction. We do not believe that this is a case that rests solely on unmet medical need, secondary endpoints, or regulatory flexibility.
HOPE-3 met its primary efficacy endpoint. The long term data show the same trajectory. The natural history comparison suggests deramiocel slows the progression of the disease. Safety continues to be a core strength of this program. Across three clinical trials, more than 125 patients with DMD have received over 1300 infusions of deramiocel. More than 80 patients continue in our open label extension studies, some for more than five years, and deramiocel has been generally well tolerated throughout.
Others have reached similar conclusions. The HOPE-3 results were published in The Lancet. The cardiac data received the American Heart Association’s Outstanding Research Award in Pediatric Cardiology. The data were presented at the World Muscle Society and at the Advisory Committee meeting. 26 speakers in the open public hearing urged that deramiocel be made available to people living with DMD. Finally, as many of you may have seen Dr. Craig McDonald and 27 other Duchenne clinicians and researchers, including the HOPE-3 investigators, sent a letter to FDA which they have since made public.
In it they explain why they believe FDA should approve deramiocel based on the strength of the data, not just the unmet medical needs. Now let me turn to our commercial readiness and manufacturing approximately 15,000 people in the US are living with DMD and we estimate that roughly 2/3 are ages 9 and older. The population our proposed refined indication would address Most boys lose the ability to walk by their early teens and from then on their arms and hands are what keep them independent that there is no therapy approved specifically to preserve upper limb function. HOPE-3 was designed for exactly this group, including those who are primarily non ambulant patients who have historically been underrepresented in Duchenne clinical trials.
We are continuing our commercial readiness activities in a disciplined way pacing spend as the regulatory timeline evolves. Launch planning is underway across all critical functions including distribution, patient support, market access, medical affairs and physician education. Payer discussions have begun and we will share more at the appropriate time. In terms of manufacturing, our San Diego GMP facility is up and running and ready to supply an initial launch if deramiocel is approved.
Build out of the second floor of our facility is progressing with validation targeted for the first half of 2027. Once complete, we expect this facility to support 2,000 to 2,500 patients or up to 10,000 doses per year, operating at full capacity, giving us room to grow based on demand. Now turning to our dispute with NS Pharma, we continue to pursue our contractual dispute with Nippon Shinyaku and NS Pharma regarding the deramiocel distribution agreement. As we have previously shared, we withdrew our motion for a preliminary injunction without prejudice to resolve the dispute in arbitration following the FDA’s decision and we expect the arbitration hearing to commence in mid-2027. Our position on the underlying dispute has not changed and we will share more updates on this as they become available.
Finally, our pipeline and organization to keep our resources focused on deramiocel, we have moderated the pace of pipeline work not directly tied to it until we have further regulatory clarity. As part of that same discipline, we reduced our workforce by approximately 10% in the third quarter to align our organization with our near term priorities. These were not easy decisions and we are grateful to the colleagues who contributed to Capricor and have gone in different directions.
Now outside the US we have initiated regulatory engagement in Europe and Japan evaluating deramiocel in younger boys with DMD and Becker muscular dystrophy remain an area of focus, with next steps to be informed by the outcome of the US review. We look forward to sharing more about our longer term pipeline vision as the deramiocel review progresses and I will now hand it over to AJ to walk through the financials.
AJ Bergman (Chief Financial Officer)
Thank you, Linda. As of September 30, 2026, Capricor Therapeutics’ cash, cash equivalents, restricted cash, and marketable securities totaled approximately $198.5 million. No shares were sold under our ATM program this year, and there’s been no revenue recognized for the third quarter of 2026 or 2025. Turning to operating expenses, for the third quarter of 2026, they were approximately $44.7 million compared to approximately $26.3 million for the third quarter of 2025.
The increase was primarily driven by pre-commercial activities and manufacturing scale-up. Net loss for the third quarter of 2026 was approximately $42.7 million, or $0.73 per share, compared to a net loss of approximately $24.6 million, or $0.54 per share, for the third quarter of 2025. For the nine months ended September 30, 2026, our net loss was approximately $117.3 million compared to approximately $74.9 million for the same period of 2025. We continue, as Linda mentioned, to pace commercial spending to the regulatory timeline, and our workforce reduction further aligns our cost structure with our priorities. Based on our current operating plan, we believe our existing cash, cash equivalents, and marketable securities are sufficient to fund operations for at least the, and with that, we’ll now open up the line for Q&A.
OPERATOR
Thank you, ladies and gentlemen. We will now begin the question-and-answer session. Should you have a question, please press star followed by the number one on your touchtone phone. You will hear a prompt that your hand has been raised. Should you wish to decline from the polling process, please press star followed by the number two. If you’re using a speakerphone, please lift the handset before pressing any keys. One moment, please, for your first question.
Our first question comes from the line of Leland Gershel from Oppenheimer. Please go ahead.
Leland Gershel (Analyst at Oppenheimer)
Great. Thank you very much for the update and taking our questions, and congrats on receiving the AHA award. Just actually a question on the cardiomyopathy data. Wondering, Linda, even though you didn’t show significance on the overall population as evaluated for LVEF on the secondary, but did on the cardiomyopathy subgroup, just wondering if from the data that you have today, comprehensively, if you can offer perspective on how long Daramycel could delay the onset of clinical cardiomyopathy in patients who don’t yet have it, should they begin treatment, perhaps before they actually have clinical cardiomyopathy.
Linda Marban (Chief Executive Officer)
Yeah, thanks, Leland. You raised some really important points. I’d like to highlight that in the intent-to-treat population of the HOPE-3 study, which was not designed specifically to evaluate cardiomyopathy—therefore inclusion/exclusion criteria were not designed around cardiomyopathy—approximately one third of the patients were healthier, had ejection fractions well within the normal range, and you can’t make a healthy heart healthier, so to speak.
So, in fact, the fact that we were nearing significance in the full ITT population portended the value of the therapeutic. And those with cardiomyopathy—unequivocally—which was two thirds of the population in the HOPE-3 study, unequivocally we saw clinical and statistically significant benefit. So that’s very important to note that in the patients with cardiomyopathy, Daramycel definitely shows a benefit and is similar to the benefit already shown in the HOPE-2 clinical trial.
In terms of your very important question, which is can we delay the progression of cardiomyopathy long term and prevent the development of long-term functional consequences, this will require longer-term evaluation of these patients in terms of ejection fraction. What we know from our clinical leaders in cardiology, including John Soslow, who presented the data, is that we had a reduction in the amount of scar in the heart, and that was evident from pretty early on.
What we know is that kids get scar pretty young. If we can get Daramycel to their hearts early, prevent the development of the scar, it’s likely that it will delay the progression of the onset or continuation of the cardiomyopathy. And we look forward to having that opportunity, both commercially and clinically, to continue that program of understanding the cardiomyopathy associated with Duchenne.
Leland Gershel (Analyst at Oppenheimer)
That’s very helpful. Thanks.
Linda Marban (Chief Executive Officer)
Thanks, Leland.
OPERATOR
Our next question comes from the line of Ted Dentoff from Piper Sandler. Please go ahead.
Ted Dentoff (Analyst at Piper Sandler)
Great. Thank you so much for taking the questions, and fingers crossed coming up with the PDUFA date. I really hope the FDA sees the value that everybody recognizes in this drug. I know you went through some details with respect to launch prep on the positive outcome. What can you tell us in terms of manufacturing supply, you know, now, and kind of how that will expand going forward? Thank you.
Linda Marban (Chief Executive Officer)
Yeah, thanks, Ted. Obviously, you know, it’s been really one of the important aspects of our clinical development program was to have manufacturing come along behind. And the development of our GMP facility here in San Diego was critical in that it’s passed PLI inspection, and the first-floor clean room is ready to go to be able to meet the needs of 250 patients per year, or, you know, numbers of doses that are equivalent to that—around 1,000 doses per year—which is important because remember, patients typically get a dose every quarter.
So you can actually have a lot more patients that are treated without hitting that year mark. Our second-floor facility is pretty much built out. We’re going to be finishing clean rooms sort of in sequential order as demand implies. And that facility, as I stated, can meet the needs of about 2,500 patients annually. And so we don’t anticipate that supply will be a problem either in early stages of launch or as we continue commercial development. If we are lucky enough to get approval in the U.S., we’re poised to continue the expansion of our manufacturing footprint into a new facility in San Diego, which we’ve already tentatively agreed to occupy and build out should the need prevail.
Ted Dentoff (Analyst at Piper Sandler)
Great. Thank you very much. Good luck.
Linda Marban (Chief Executive Officer)
Thank you, Ted. Take good care.
OPERATOR
Our next question comes from the line of Kristin Pliska from Cantor Fitzgerald. Please go ahead.
Ayan (Analyst at Cantor Fitzgerald)
Hi, this is Ayan on Kristin’s line. Thank you for taking our questions here. With the arbitration hearing expected to begin mid-2027 next year, I’m just wondering how you see this potentially impacting bringing Daramycel to market if approved later this month.
Linda Marban (Chief Executive Officer)
So regardless of how the dispute is resolved, we’re prepared. Our San Diego facility is operational, as I just mentioned. We’re building out patient support, market access, and medical affairs. We will share more on our specific commercialization plans following the FDA’s decision. Obviously, if it’s positive, commercialization will become our key focus, and then we’ll have updates to the Street. We don’t anticipate that the arbitration or its continuance or conduction will impact our ability to bring Daramycel to patients.
And that’s going to be our goal. We want to take care of those boys and young men with DMD.
Ayan (Analyst at Cantor Fitzgerald)
Sounds good. Thanks for that call, Linda. Just one more from us. With the July inspection, which you indicated that you’ve already submitted a response, I’m just wondering if the FDA could proceed with an approval before formally responding. Just wondering your thoughts on this.
Linda Marban (Chief Executive Officer)
Yeah, that’s in the hands of FDA. I don’t know exactly how they manage it. We have had no further follow-up or questions related to that inspection. We sent in all the requisite documents, and they closed out the actual inspection itself. So in terms of how FDA will manage that information in terms of the timelines of approval is really in the hands of FDA.
Ayan (Analyst at Cantor Fitzgerald)
All right, thank you, Linda. Oh, if I could just sneak in one—sorry. If I recall correctly, the FDA previously inspected the San Diego facility in 2025. I’m just wondering if you anticipate any additional inspections ahead of the PDUFA date here.
Linda Marban (Chief Executive Officer)
Well, the PDUFA date is in 12 days, so they better hurry up if they’re going to—no, no, no, I’m being a little silly. No, we don’t anticipate any more. I think what we’ve heard is that they’ve done the inspections that they need to. They have the data that they seem to need, and we haven’t heard anything. So obviously we’ll update the Street should anything become, you know, clear that they need to do any more. But right now we’re thinking we’re done.
Ayan (Analyst at Cantor Fitzgerald)
Sounds good. Thank you, Linda. And we’re voting for you.
Linda Marban (Chief Executive Officer)
Thank you. Tell Kristin we said hi.
OPERATOR
Our next question comes from the line of Madison Alsadi from B. Riley Securities. Please go ahead.
Madison Alsadi (Analyst at B. Riley Securities)
Hey guys, thanks for taking our question—one from us. Would you expect any of the cardiomyopathy subgroup data, the LGE results, to appear anywhere in a potential label, even though the focus now is on upper limb, or do you think this really even matters in terms of the potential launch? Maybe manufacturing is the greater constraint on scaling. Appreciate any color there. Thanks.
Linda Marban (Chief Executive Officer)
Thanks, Madison. Yeah, so we’re not disclosing the label information at this point. What I can tell you is I’m very bullish personally and professionally on the cardiomyopathy data, and whether it’s in the label now or will be a future label expansion is something that we’ll update people on later, but that data is good and the patients definitely deserve it.
Madison Alsadi (Analyst at B. Riley Securities)
Understood. Thanks for taking our questions and good luck.
Linda Marban (Chief Executive Officer)
Of course. Thanks, Madison.
OPERATOR
Our next question comes from the line of Rick Binkowski from Ladenburg. Please go ahead.
Rick Binkowski (Analyst at Ladenburg)
Hey, good afternoon, and thanks for taking the question. So the press release mentioned the list of preparations that were being made for the commercial launch, including distribution, patient support, etc., and it seemed like it was a pretty comprehensive list for launching a drug solo. So my question is, once we get to the other side of arbitration, what do you see as the role of NS Pharma here? And could any of these responsibilities eventually fall on NS?
Linda Marban (Chief Executive Officer)
Yeah, thanks for the question. Right now, because the dispute is ongoing and the agreement is written, by our interpretation of our lawyers, as non-transactable, we are going independently in preparation for launch and commercialization of Daramycel without the participation of NS Pharma at this point in any way. Having said that, you know, the arbitration panel obviously has important opportunities to direct how things go. And so in the future, if we work with NS, we will definitely disclose that and talk about that.
But, you know, there’s nothing better than being prepared, right? So we’d rather be prepared to go than unprepared.
Rick Binkowski (Analyst at Ladenburg)
All right, that’s it for me. Thank you.
Linda Marban (Chief Executive Officer)
Thank you.
OPERATOR
As a reminder, if you have any questions, please press star one. Our next question comes from the line of Catherine Novak from Jones. Please go ahead.
Catherine Novak (Analyst at Jones)
Hi. Afternoon. Thanks for taking my question. I’m just curious, in light of AHA’s response to the cardiac data from HOPE-3, can you give us a sense of what additional data they saw that maybe FDA didn’t have access to when they wrote the briefing documents going to the AdCom? You know, what do you think the most notable outcome was from AHA’s perspective? And then, you know, how many of the information requests that you got from FDA were related to anything cardiac?
Linda Marban (Chief Executive Officer)
Yeah, so thanks for that. You know, in terms of AHA, I think there was the totality of the data that drove them. The observation that DMD cardiomyopathy is an important problem and seems to be addressed by Jaramycel. I think AHA also, and this is all supposition, obviously, but just based on my own experience with AHA, thought that the combination of scar data, LGE data combined with function data was a really, really powerful tool. And so, you know, I think it was sort of all taken together in terms of the additional data.
I think what FDA now has, but was not part of the original review, is the open-label extension data compared to the natural history data, John Soslow’s data set, and then all the robustness analyses which weren’t really covered very much by the AHA data, but taken together it’s a much stronger data set showing long-term safety and efficacy. In terms of IRs from the FDA, which was the second part of your question, as I mentioned at the beginning, due to the sensitivity of the review process, we’re really not disclosing communication between Capricor Therapeutics and the FDA at this time.
Thank you. Take good care.
OPERATOR
Our next question comes from the line of Joe Pankinis from HC Wainwright. Please go ahead.
Joe Pankinis (Analyst at HC Wainwright)
Hey guys, good afternoon. Thanks for taking the question and all the details today. So first, with regard to NS, what are the terms to potentially part ways here, pending arbitration, versus being able to just move on to potentially find someone else, number one. And then obviously you made some thoughtful and required pipeline moves here. But are there any outstanding or ongoing ISTs with regard to the exosome programs that we can expect to hear some work or data from?
Linda Marban (Chief Executive Officer)
Yeah. So in terms of NS, we really are focusing now on independent launch and preparation for commercialization. That’s going very smoothly, and we stand poised and ready to do an effective launch should we be lucky enough to achieve approval. We’re not looking, obviously, for another partner. I think that if we’re lucky enough to achieve separation from NS completely, we’ll do it on our own. It’s a rare disease and we’re perfectly capable at this point in time doing that.
And I’m excited to have a good team in place to do so. In terms of exosome pipeline opportunities, you know, we are working diligently in the background. As you know, we’ve talked about this for a long time. We’ve de-emphasized the pipeline to conserve cash over time and focus on Jaramycel. We continue to do that, although we have some pretty exciting opportunities coming along. In terms of the exosomes, we’re successfully able to manufacture them to clinical and commercial scale now. We have GMP manufacturing of exosomes, and we have targeting and loading opportunities that we did not have before.
And so what I can tell you is that we’re still excited about them. We think that they are the future of drug delivery in many ways and look forward to sharing that as it moves forward.
Joe Pankinis (Analyst at HC Wainwright)
Great. Thanks, Linda.
Linda Marban (Chief Executive Officer)
Thanks, Joe.
OPERATOR
Our next question comes from the line of Bubalan Pachayapan from Roth Capital Partners. Please go ahead.
Bubalan Pachayapan (Analyst at Roth Capital Partners)
Hi. Thanks for taking your questions. So I wanted to revisit the July ADCOM which voted on cardiomyopathy rather than the upper limb indication you’re proposing now. So I’m just curious, you know, whether during your interactions with the FDA, did they indicate that they can reach a decision using their existing board or whoever they have advisors with? Or do you think there is a chance for a second ADCOM focusing on—
Linda Marban (Chief Executive Officer)
Well, they’d have 12 days to organize, conduct and get ADCOM together if they stick with the current PDUFA date. So I don’t think that’s very likely. You know, they use advisory committees in order to give them information or clarity in topics in which they don’t feel they have enough information to render a decision. I think they got a lot of information from the ADCOM. We tend to disagree with many of the ways that the ADCOM was organized, including the question that was asked, which was probably the worst part of the entire decision process for the advisors and was evidenced by their comments made post vote.
And so no, I don’t think that there’ll be another outcome within the next 12 days, but if there is, I suppose we’ll be ready.
Bubalan Pachayapan (Analyst at Roth Capital Partners)
All right, maybe just one more. Very quickly, let’s just say you receive a CRL and then the FDA wants you to do another trial. What would you need to see in the FDA’s feedback to justify additional investment and what would the likely timeline look like? Thank you.
Linda Marban (Chief Executive Officer)
Yeah, that is a really important question and it’s one that we’ve been debating internally. If the FDA issues us a CRL, obviously we will very carefully take in the information included in that CRL and what they ask for and then, you know, we’ll make the decisions prudently based on if we think that there’s a way to establish greater efficacy and or safety doing another clinical trial. What I can tell you is we believe that Jaramycel works to make Duchenne less impactful to these boys and young men in terms of upper limb function. We’ve seen it year over year and in multiple clinical studies, and many independent peer reviewers have seen it as well. So I’m reluctant to say goodbye to Jaramycel if it requires some simple clinical work that could further support it. Having said that, internally we are focusing primarily on approval.
We believe the FDA has enough data to make a positive decision and look forward to sharing that if and when it comes.
Bubalan Pachayapan (Analyst at Roth Capital Partners)
Great. Thanks for taking our questions and good luck.
Linda Marban (Chief Executive Officer)
Thanks, Bubalan.
OPERATOR
I will now turn the call over to management for closing remarks.
Linda Marban (Chief Executive Officer)
Thank you so much for joining us today. Since the advisory committee meeting in July, we have submitted the two-year open-label data from HOPE-3 to the FDA, presented new results at the World Muscle Society and the American Heart Association. The validation of work by our external review has continued to strengthen our confidence in the data. We recognize the urgent unmet need in Duchenne, but we are not asking for a pass because of it. We have run this program with rigor and transparency and we believe the evidence stands on its own.
We are encouraged by the FDA’s focus on rare disease, including the agency’s recently launched Rare Disease Framework Initiative, which recognizes the challenge of developing therapies for small, heterogeneous patient populations like Duchenne. We appreciate the FDA’s engagement throughout this review, and we respect this process. To the Duchenne community, the patients, families, clinicians and advocates: thank you. Thank you. Thank you. We hear you, and we are working tirelessly to bring Jaramycel to approval. You may now disconnect.
OPERATOR
Ladies and gentlemen, this concludes our conference call for today. Thank you for your participation. You may now disconnect.
Disclaimer: This transcript is provided for informational purposes only. While we strive for accuracy, there may be errors or omissions in this automated transcription. For official company statements and financial information, please refer to the company’s SEC filings and official press releases. Corporate participants’ and analysts’ statements reflect their views as of the date of this call and are subject to change without notice.
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