Capricor Therapeutics, Inc. (CAPR) Earnings Call Transcript & Summary

Thank you for standing by. This is the conference operator. Welcome to the Capricor Therapeutics HOPE-2 top line 12-month data call. [Operator Instructions] And the conference is being recorded. [Operator Instructions] I would now like to turn the conference over to A.J. Bergmann. Please go ahead.

Thank you, and good morning, 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, among other things, regarding the efficacy, safety and intended utilization of our product candidates; our future research and development plans, including anticipated conduct and timing and preclinical and clinical studies; our plans to present or report additional data; our plans regarding regulatory filings; potential regulatory developments involving our product candidates and our possible uses of existing cash and investment resources. These forward-looking statements are based on current information, assumptions and expectations that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward-looking statements. These and other risks are described in our periodic filings made with the SEC, including our quarterly and annual reports. You are cautioned not to place undue reliance on these forward-looking statements, and we disclaim any obligation to update such statements. With that, I will turn it over to Linda Marbán, CEO.

Good morning, and thank you for joining us on this call to discuss the 12-month HOPE data. Shown here is our forward-looking statement slide. Please be aware that all of our filings are updated with the SEC. I hope we find you at home this morning and doing well during his most unprecedented times. Joining me this morning is Dr. Craig McDonald, our National PI from the University of California at Davis. Dr. McDonald really needs no introduction as he is a world-renowned leader in the neuromuscular disease space and has presented data for Capricor at the World Muscle Society as well as other venues over the past few years. However, to remind you, Dr. McDonald is Professor and Chair of the Department of Physical Medicine and Rehabilitation and Director of the Neuromuscular Disease Clinics at UC Davis, and of course, is an internationally recognized expert in the clinical management and rehabilitation of neuromuscular diseases, which includes Duchenne muscular dystrophy, otherwise known as DMD. Now before Dr. McDonald discusses the HOPE-2 data and its importance for patients with advanced DMD, I will take a few minutes of your time to put into context the importance of the data we have top line this morning. We have been working closely with the FDA for the last few years as we have been developing CAP-1002 for DMD. Along those lines, we have been granted RMAT, Regenerative Medicine Advanced Therapies designation, which is part of the 21st Century Cures Act and is similar to breakthrough designation for cell and gene therapies. We have also received work in drug designation and qualify for a rare pediatric disease coupon if our product is approved. All of these make the path forward a bit easier and collaborative with the regulators which is obviously critical in defining the path forward to approval. So let me remind you a little bit about CAP-1002. CAP-1002 is an allogeneic, which means off-the-shelf cell therapy product where we think the active ingredient is the exosomes that contain nucleic acids such as RNA and proteins. The cells release the exosomes, which in turn track to sites of injury. From experiments that we've done over the past many years on exosomes, we know they can obtain multiple RNAs, primarily a few micro RNAs and noncoding RNAs. These are very small sections of RNA that mediate protein expression. These are profoundly immunomodulatory and correlate with the potency of our product, CAP-1002. The cells have been in over 150 patients, and their safety profile is very good. So I know that this is a complicated graphic, but it's an important 1 because it shows the unifying mechanism of action of the cells, where they're treating COVID-19 or Duchenne muscular dystrophy. Now as you can see, the cells turn on macrophage and effector T cells who are mediators of the humoral or blood and cellular immune response and they modulate inflammation by controlling the release of cytokines. These are chemicals that stimulate either an inflammatory response or promote cellular repair. It is in this modulation of the immune response that we believe the cells can be effective in Duchenne muscular dystrophy as well as in the flagrant over-activation of the immune system, which is seen in COVID-19 as well as in other indications where inflammation is a major factor in the pathology. In fact, just yesterday, we published, along with our colleagues at Cedars-Sinai Medical Center in Los Angeles, a peer-reviewed publication in the journal called Basic Research in Cardiology, which is a peer-reviewed paper and highlights our experience using CAP-1002 and a series of critically ill patients with COVID-19-related ARDS, or acute respiratory distress syndrome. And the data from that was very promising and furthermore, promote -- suggest further clinical evaluation. Now a few years ago, some of the advocacy groups in Duchenne began speaking out about the fact that there was nothing available for the cardiomyopathy associated with DMD. And furthermore, the heart failure was found to be the #1 cause of death in patients with DMD. We were intrigued by this and embarked on a preclinical journey with the mdx mouse, a naturally occurring mouse model of Duchenne, and this preclinical work has ultimately redefined this company and our mission. Shown here is an overview of some of the preclinical work which reflects the potential of CDCs in Duchenne. What has been gratifying is the remarkable association between the preclinical data, which showed improvements and shown here in heart function, skeletal muscle function, both globally and an isolated muscle and repair of diaphragmatic muscle damage, which compared to the data, clinical data produced in both HOPE-Duchenne and our first clinical trial in DMD and now in HOPE-2, which is by far the best clinical data we have ever seen in the clinic. In HOPE-Duchenne, we focused on finding those patients that had advanced heart disease. It was a 25-patient randomized open-label study. Most of the patients were non ambulant, and we dosed them onetime with 75 million cells into their coronary arteries. We followed them for a year, and we studied some measures of skeletal muscle function based on the observations I just showed that were made preclinically. The data, which was published in the Journal of Neurology last year, showed an improvement in the scar in the heart in treated patients with a corresponding improvement in systolic wall thickening, which is a measure of how well a section of the heart is contracting. We also saw some intriguing data on the performance of the upper limb, the first version of such, which is called Version 1.2, which at the time was a relatively new way of measuring skeletal muscle function in non-ambulant patients. Again, we saw what we had seen in the mice, which was an improvement in the mid or arm plus distal, which is hand and wrist, upper limb function in the treated patients. While the control patients stayed the same over the course of the year. We also identified that our best improvements in the upper limb function occurred within the first 3 months of treatment. Again, exactly what we had observed in the mice. Now all of these observations coalesced beautifully with the discoveries that we -- were made in the lab that the exosomes are mediating the biology that the cells were immunomodulatory and that they stimulated muscle repair. We even have evidence that exosomes enter into the satellite muscle cells of skeletal muscle in the mice and stimulate repair. We also realized and perhaps this was one of the most important discoveries that we did not need to deliver the cells to the heart directly. We could deliver the cells by simple intravenous infusion which is, of course, safer and easier to administer. This change in the delivery method took us back to the lab and ultimately led to our ability to do two things: one was to IV infuse patients, and two was our ability to double the dose to 150 million cells. And of course, now we were able to dose quarterly, which the data suggested that we do. So this is where my narrative end and Dr. McDonald will take over to share with you the HOPE-2 story. I am beyond excited for the future of CAP-1002 and DMD. We at Capricor truly believe that these cells could markedly improve the lives of those patients for which limited options exist, and we plan to work hard and effectively in bringing this therapy to approval. I will stop there and ask Craig McDonald to take over. Dr. McDonald?

Thank you, Linda. It's really a pleasure to be with you this morning and share these exciting results. Just to remind people on the call, Duchenne muscular dystrophy is a relentlessly progressive disease. The natural history has been very well characterized, and patients go through a fairly typical stage of DMD progression from delayed and impaired acquisition of motor milestones to an ambulatory stage where they have fairly predictable loss of ambulatory skills. This CAP-1002 HOPE-2 trial has really focused on the early non-ambulatory stage where patients are actually losing overhead reach. They're losing the ability to get the hand to the mouth. And also losing hand function as well. And that was really the target population. This was really the first placebo-controlled trial in a non-ambulant Duchenne muscular dystrophy population. And the first to use the performance of upper limb measure as a primary outcome measure. So the target population, again, is the early non-ambulatory Duchenne population. The -- this actually shows the study design. It was a Phase II randomized double-blind placebo-controlled trial and the objective was really to evaluate both safety and efficacy. This was the first trial again to use intravenous systemic administration of CAP-1002. There were 9 really excellent trial sites nationally that participated in this population. The ITT population involved 20 subjects. Mean age of the patients was 14.3 years. All these patients were on corticosteroids, 80% of the patients were actually non-ambulatory. The remainder were in the last stages of the ambulatory stage of disease. This just shows a graphic on the entry items of the performance of upper limb measure, which is actually used as the primary outcome measure here. And just to demonstrate to you what the target population of Duchenne muscular dystrophy is. The entry items on the PUL actually range from a best score of 6 down to a score of 0, where you have complete loss of hand function. And the population that was a focus of our clinical trial here in Duchenne was those who had PUL entry scores of 5, 4, 3 and 2. These were patients that we believe we could actually demonstrate an improvement of arm function or a stabilization of arm function in the course of of a 12-month trial. Those who have complete overhead reach, those who have PUL entry scores of 6, would largely be difficult to show improved arm function in the course of a 12-month trial. If we go to the next slide here, this just demonstrates the -- really, the granularity of the PUL items. They are shoulder level items or tasks that are actually evaluated by the physical therapists, middle level or elbow function items and then distal level items as well. And the performance of upper limb is now really the choice end point that is being used in Duchenne muscular dystrophy to evaluate upper limb function. And this has been an evolving measure. The PUL version 2.0 had just come out at the initiation of this trial. We were fortunate enough to include both the PUL 2.0, and we evaluated function with a PUL 1.2 version. The PUL version 2.0 has a 3-point response scale, very similar to the North Star. It's much more robust and reproducible than the version 1.2. And it actually allows for compensatory strategies. So there's a focus on actual function. So it allows compensatory strategies to achieve a task. This is not allowed in version 1.2. And data that has emerged shows that it's really better able to detect a change at 12 months at all levels of ability in Duchenne muscular dystrophy. So to get into the data first, let's focus on the upper limb skeletal muscle function, measured both by the performance of upper limb version 2.0 and the version 1.2. So this is the data on the, really clinically meaningful changes that were observed on the PUL 2.0. This actually combines the shoulder plus the mid plus the distal item functions. The blue bars show really very minimal decline in the CAP-1002-treated patients in comparison to the placebo patients, who almost lost 4 points on their PUL 2.0 over the course of the 12 months. This represented a treatment effect of 2.4 points in the CAP-1002-treated patients versus placebo at 12 months. On the right, you can see the individual patient-level data. The p-value here was 0.053. So really built a statistically significant and also a clinically meaningful treatment effect. We believe that a 1 point change on the PUL is clinically meaningful over the course of a 12-month trial. And actually, in our discussions with the FDA, the FDA concurred that they were supportive that a 1 point change in the PUL 2.0 would be a clinically meaningful effect size. So we're really quite pleased to see a 2.4 point treatment effect here in the CAP-1002-treated patients. This, again, is the patient level data, showing the treated patients versus the placebo patients. There are a number of patients that have really nice stabilization even some improvement in arm function. The placebo patients actually had a very predictable decline over the course of 12 months. So again, this shows really the sensitivity of the performance of upper limb to actually demonstrate disease progression over the course of a 12-month period of time in Duchenne dystrophy. This actually -- the next slide shows data on the mid-level PUL 1.2. This is a focus on elbow function. Going into the study, we believe that given our target population, we fully expected to see an improvement in elbow function. And again, here, you can see the data on the CAP-1002 patients versus the placebo-treated patients. Again, a 2.8 point difference in elbow function as measured by the PUL 1.2 in the CAP-1002 versus the placebo-treated patients. Over on the right, you can see there was one nonresponder patient treated with CAP-1002, but the rest of the patients really had a very nice stabilization in function. And then the placebo patients had a very predictable decline in their elbow function over the course of the 12-month trial. This, again, shows the patient-level data, the CAP-1002-treated patients in terms of their elbow mid-level function. Again, you can really see the stabilization function. And virtually all the CAP-1002-treated patients, except for the 1 nonresponder. And then on the right, you can see the very predictable decline in elbow function, mid-level function measured by the PUL 1.2 over the course of the 12-month trial. Next, I'd like to focus on cardiac function. This was actually measured by MRI. And so I'm going to present data on cardiac injection fraction measured by MRI. Left ventricular end-systolic volume as well as left ventricular end-diastolic volume. And then a biomarker evaluation of CK-MB fraction. The MRIs were -- I should mention the MRIs were actually evaluated in a blinded fashion by a pediatric cardiologist, who's really one of the leaders in cardiac MRI in Duchenne muscular dystrophy at Nationwide Children's, and this individual actually read all these MRIs in a blinded fashion. So the next slide actually shows the improvement and stabilization in left ventricular ejection fraction in the CAP-1002 patients. The placebo-treated patients actually had a rather significant decline in their ejection fractions over the course of the year. Versus the stabilization in the cardiac ejection fraction. So a downward value in this would show essentially worsening cardiac ejection fraction in the placebo-treated patients. And you can see here the stabilization of cardiac ejection fraction in the CAP-1002-treated patients. And I think this is really quite exciting for us. We haven't really seen a therapeutic like this that produces a stabilization in cardiac function as measured by cardiac MRI. This is the individual patient-level data. You can see the -- interestingly, the same nonresponder that did not respond to the cell-based therapy on the skeletal muscle function also showed continued decline in their cardiac MRI. The other patients showed actual stabilization and then the placebo-treated patients all showed a very predictable decline in their left ventricular ejection fraction over the course of the trial. Also, there were measurements of left ventricular end-systolic volume and left ventricular end-diastolic volume. That were observed in. And here, dilation would actually -- if you look at an indexed value, the end-systolic volume on an indexed basis has actually been used as a surrogate end point for drug approval in adult heart failure. And here, increased values are actually indicative of worsening heart functioning, worsening cardiomyopathy. And here, we actually see improvement in the CAP-1002 treated patients as measured by a mean decrease in their end-systolic volume. Really, which is very exciting to see the placebo patients continue to progress. And then not only systolic function appears to be improved, but also a diastolic function appears to be improved as well. As you can see, on the right, there was improvement in the left ventricular end-diastolic volume as measured by MRI. And again, these were really statistically significant. The end-systolic volume measure was statistically significant at the p 0.01 level. So we're really quite excited about this data. And then finally, in looking at the biomarker data, the creatine kinase MB fraction as a proportion or fraction of the total creatine kinase observed. This is an enzyme that's known to be associated with breakdown of cardiac muscle cells, it's specific to myocardial damage. And here, you can actually see an improvement in the CK-MB fraction, highly statistically significant here at the p 0.006 level. Increases in the CK-MB fraction, really quite consistent with the deterioration seen by cardiac MRI in the placebo-treated patients and actual improvement in the CK-MB fraction in the CAP-1002-treated patients. And then again, the next slide here shows the patient-level data. Virtually all of the CAP-1002 patients showing a stabilization in this biomarker, and you can see the trend towards worsening and increasing CK-MB fractions in the placebo-treated patients. The FDA is exceedingly interested in consistency of the treatment effect over multiple end points. And so we also looked at measures of respiratory function and we wanted to focus on both measures specific to inspiratory muscles, which is predominantly would be indicative of the diaphragmatic improvement the inventory flow reserve versus measures of expiratory function, the peak expiratory reserve or the peak expiratory flow rate, which actually is an indicator of chest wall function. And Santhera in their studies of idebenone have focused on the peak expiratory flow rate as a primary outcome measure in Duchenne muscular dystrophy. And in terms of the individual patient level data, we certainly would rarely expect these measures of the inspiratory flow reserve to improve over the course of 12 months. And here, you can see patient-level data a number of patients actually showed improvement in their inspiratory flow reserve, but whereas the placebo-treated patients quite predictably showed a trend towards decline in their inventory flow reserve. And then again, the peak expiratory flow rate, as a percent predicted value, you can see a number of patients here, treated with the CAP-1002 product show improvement in a percent predicted peak expiratory flow rate, where, again, the placebo patients all showed a predictable decline. I'd like to just comment on the safety data in using this cell-based product. We've probably treated more patients than anybody. We treated a number of patients at UC Davis with this product. And in terms of a total of 69 infusions were performed, either in the CAP-1002-treated patients or the placebo-treated patients. It was generally safe and well tolerated throughout the study. With the exception of 2 hypersensitivity reactions, there was really no permanent sequela from this, but transient hypersensitivity reactions. In December of 2018, Capricor put a voluntary hold on dosing after 2 patients in the trials had serious adverse event in the form of hypersensitivity reactions. These were possibly linked to excipients, namely DMSO, that's the current thinking in terms of the result of this hypersensitivity reaction that was seen. There were mitigation efforts to reduce the risk of such future events. We initiated a commonly used premedication strategy. We actually dose the patients with oral steroids ahead of time and an IV antihistamines to prevent or mitigate potential allergic reactions during the IV administration. And since the initiation of this pretreatment regimen, 42 infusions of the investigational drug have been administered with only one more mild hypersensitivity reaction that just required overnight observation of the patient. So just to conclude. The HOPE-2 trial, this was really the first placebo-controlled trial showing upper limb functional improvement in non-ambulant Duchenne muscular dystrophy patients. The non-ambulatory Duchenne population represents over half of the Duchenne dystrophy patients that we follow clinically. So we're really quite excited that we actually see results that demonstrate actual improvement in upper limb function that's so important to these patients' quality of life. They were also directionally consistent improvements in strength measures of upper limb function, respiratory measures and cardiac end points, both cardiac MRI and cardiac biomarker end points. And this is really the first ever study in Duchenne that correlates cardiac functional stabilization. With a reduction in a biomarker of myocardial cell damage. And also to myself as an investigator, I really am quite pleased to see really the consistent results showing in the preclinical animal data, the Phase I/II HOPE trial and the Phase II HOPE-2 trial. So I think moving forward, Capricor has requested an end of Phase II meeting with the FDA to discuss pathway to approval. They have engaged a global CML for scale up and manufacturing of CAP-1002. And also, we clinically believe this product is working and helping patients. So we are now expeditiously initiating an open-label extension trial of all the patients that participated in HOPE-2. Again, I think one of the advantages we have here, these patients have all continued to remain blinded, so we will actually have an off-treatment period of time where we can -- we have the ability to observe continued deterioration, and then we can then look at improvement in function with the initiation of the investigational product in the context of an open-label trial. So I think that will provide additional data demonstrating evidence of drug effectiveness as well as safety. I want to mention just the advisory Board to Capricor, really involved leaders in the field: Dr. Richard Finke, Pat Furlong, Kan Hor, actually did the cardiac MRI measures in a blinded fashion. John Jefferies, Henry Mayer, who's a pulmonologist, Eugenio Mercuri from Italy, who's really a worldwide leader in Duchenne muscular just therapeutic development as well as Francesco Muntoni, Thomas Voit, Lee Sweeney and Michael Taylor. So really, we had really phenomenal input from leaders throughout the world. I'd also like to just finally acknowledge all the patients and families who participated in the HOPE-2 study. It was supported by a number of advocacy groups: Parent Project Muscular Dystrophy, Coalition Duchenne, CareDuchenne and also the trial was funded with the support of the California Institute for Regenerative Medicine. And I'd like to also acknowledge the investigators throughout the U.S. who participated in the HOPE-2 trial. And with that, I'll conclude and pass the session back to you, Linda. Thank you very much.

Thank you, Craig. That was absolutely fantastic. I would like to just say it has been an absolute pleasure working with you over the past 2 years on this trial. Your motivation, your commitment to patients and your strong intellect make every time we interact, a learning and growing experience for me. So thank you. Well, I'd like to close this call with a few personal comments. First of all, I'd like to thank the parents and the families of the children who participated in this trial. It's a huge lift for these people to travel to different sites. They have to take their children and wheelchairs and get on airplanes and go to different sites. And go through days of testing, and they're small children, so they don't want to be in a hospital, and they don't want to have their arms popped. And so we truly appreciate their efforts. And hope that we can provide the promise that we made to them, which is that we would work our hardest to get this approved. Finally, I'd like to say, we had 2 positive clinical trials in this space, both showing consistency of direction with improvement in upper limb function, cardiac structure and function and now in HOPE-2 respiratory function. We really believe that this product needs to get to these patients. And with that, we have brought into our inner circle, a new army of leaders, those that will help us work with the FDA to get this on an expeditious path to approval. We have already sent in a meeting request based on this data, as you'll see in the press release and as we've talked about today. Finally, I'd like to just close again with a personal comment. When I became a scientist, I dreamed that someday, I would be part of developing something that could make the lives of human beings better or longer. And today, I feel the fulfillment of that promise, looking at this data. Let's remember that p-values really represent the chance that your data is up to chance that you just saw this as a result of some kind of random opportunity. And what we can see here with the number of zeros before digits in many of these p-values is that it's highly unlikely that the data shown here has been as a result of chance. And so perhaps one day we can stand back and say we made a difference in the lives of those patients with Duchenne muscular dystrophy. Thank you, and please come forward with any of your questions.

[Operator Instructions] Our first question comes from Joe Pantginis with H.C. Wainwright.

A heartfelt congratulations for the data. Very well-designed study, very pleased to see it. And thank you for all the details, Dr. McDonald. And my first question is for you, and I think the company might be able to add on to this also. When you look at the evolution of the discussion, with the FDA to this point. They've been that the FDA is amenable for the PUL 2.0 to be the primary end point going forward as of today. So ahead of the planned end of Phase II meeting, and I don't want to sound too exuberant about this, so I apologize for that. How could the FDA now really ignore the cardiac data have been generated to this point? I mean not ignore, but basically emphasize more.

Right, Joe, thank you. And always a pleasure to hear from you. We really appreciate the support that you've given us over the past few years as we've built towards this data. Your hunch is exactly correct. Even at our FDA meeting in October of 2019, when we shared with them the results of the interim analysis, they specifically spoke about the cardiac data and the fact that this would be the first time anybody has shown any relevant improvements in cardiac function. And just to be clear, the improvement in ejection fraction is over 1 year. And if we could continue this, if we can keep these boys at a basically healthy level of ejection fraction, even if gene therapy works, which, of course, we are hoping for and gets them up and out of their wheelchairs, they will need the strength of their heart to be able to sustain their greater activity. So we're hopeful that the FDA recognizes the unique and very relevant opportunity in improvement in cardiac function. And let me just say, although I don't want to be too long-winded here, the fact that we have a biomarker, a circulating biomarker in the CK-MB that suggests that there is a reduction in damage to the heart, actually is one of the most extraordinary findings I think I've seen in my career. Because what this basically says is that those overarching observations that we made using MRI that the function of the heart ejection fraction is better, that the structure of the heart through the volumes is better we also are seeing that there's actually less damage to the heart, which is what we were associating from the reduction in spacing and HOPE-Duchenne. So I think there's every reason to believe that this therapeutic is working, and I'm strongly hopeful for its approval.

Yes. And if I could just make an additional comment. That in Duchenne dystrophy, even with the initiation of mechanical ventilation, we are increasingly seeing that these boys and young men are coming to their cardiomyopathy, that's become one of the leading causes of death. And these patients were all treated at really centers of excellence throughout the United States. All these patients were already on really optimized medical cardiac management with the afterload reduction with ACE inhibitors or ARBs, they were on beta blockers and many of them we're also on eplerenone on or spironolactone as well. And despite those treatments, these patients were still demonstrating deterioration in their cardiac function over the course of a year, which is certainly what we see clinically. So to actually have a therapeutic stabilized cardiac function I think was really quite exciting to me. It should continue to be a focus as we move forward with this therapeutic.

That's helpful. And you actually did answer one of my questions. So I'm very happy to see the fact that you're going to go into open-label extension study to move the placebo patients over. So having that answered already, I will ask, and I guess this is a bit hypothetical. The nonresponding patients, do you have any hypotheses? Or is it just a function of the severity of the disease of patient or anything you might be looking into?

If I could just initially comment. I think that the interesting thing is that, that patient was on steroids. Despite being on steroids, seem to have pretty significant disease progression. Again, I've not been unblinded to this. But what I think is interesting is that really the nonresponder was also nonresponsive in terms of the skeletal muscle end point, the cardiac MRI end point and also the pulmonary end point. So sort of the nonresponse was very consistent across all the measures. Linda, can you just perhaps give some thoughts about the nonresponder?

Yes. Thank you, Craig, and thank you, Joe. Yes. So the nonresponder paradoxically, although sometimes, you don't ever want a nonresponder, but I'll make a few comments about him. First of all, when we took them out of our analyses and reran the statistics. The p-value for the PUL at Level 1.2 was 0.004; and for the PUL 2.0, it was 0.006. So the nonresponder is clearly impacting the statistical relevance of the data. Having said all that, when I presented the nonresponders clinical history to several of our KOLs, excluding Dr. McDonald, who remains blinded. The one bit of information that I thought was very interesting is that the age of loss of ambulation prior to the use of steroids was between 8 and 10 years of age. And this young man went off his feet at 8-years stage, which is very uncommon now, typically, it's in the mid-teens, usually. And so -- and it has been suggested that he's not responding to steroids. He also has some preexisting respiratory disease and some other congenital issues, including some other types of muscle diseases that they don't understand. And during the study, he suffered several compression fractures of his spine. So it was a terrible pain throughout the course of the study. So taking together, this poor young man is really suffering tremendously, and we wish he and his family all the luck, and hoping that he feels better.

I understand. That is unfortunate. And I don't know if you can bear with me for one last question, it's a bit forward-looking, obviously. As you go into the FDA now with these data, what would you say is the best case and the worst-case scenarios?

Yes. So thank you for that. The best case is full approval with a confirmatory study on the backside, what we're going to propose. Is a longer confirmatory study, several years where we can really validate over the course of time the preservation of function and skeletal muscle or upper limb function, and then also the preservation of cardiac function, very, very important indicators. We also believe based on the data that we've seen, the pulmonary function should also track in a positive direction, all moving in the right direction. So in the best case scenario, I always say to people some version of approval because we're still working out those details with FDA. I'm very hopeful that we will be able to get this. This is not a Sarepta situation where the data does not support approval, and it has to be presented that this is a great opportunity. The data supports approval. And we expect to move that very quickly in that direction. Worst-case scenario is that FDA says that we need to do another confirmatory or a pivotal trial. And that would be -- potentially have to redo the power analysis based on the current data, but we're anticipating a 50- to 70-patient clinical trial that we would do very quickly. The interim solution and the one that we're banking on is that we'll present the data from the open-label extension. That will be right around the time that we would be preparing a BLA anyway and that, that would support approval independent of any more data collection. So our hope is no more clinical data collection.

Got it. Congratulations again, and thanks for indulging me.

Thank you, Joe. Have a wonderful day and stay well.

Our next question comes from Jason McCarthy with Maxim Group.

Linda, Dr. McDonald, congratulations again on the data, very impressive. I just want to follow-up a little bit on Joe's questions about FDA. In terms of positioning it with the FDA, I understand that the FDA looks at every drug individually. But what are your thoughts on with the challenges that gene therapy has faced in the last 12 to 18 months. And there's really not much out there. This is the first set of really hard data, certainly on the cardiac side. How do you think the FDA now positions CAP-1002? Because you had mentioned that we all know Exondys was approved, pretty much on the unmet need, not necessarily overwhelming data. How do they look at this now?

Well, of course, I can't jump into their head. Believe me, I want to. What I'm hoping is that they realize that this serves as a very strong indicator, as you said, of preservation of cardiac function, there's nothing there. So you have unmet need finally meeting and joining with positive clinical data suggesting improvements in cardiac structure and function. This particular segment of the patient population, these later-stage non-ambulant patients are largely not eligible for gene therapy yet, and there's a lot of questions in the space independent of whether they're eligible or not as to whether it would work, their muscles are so fibrosis. And I'll probably let Dr. McDonald comment on that in a moment. But we strongly believe that FDA will look at the strength of the data, the strength of the opportunity. And let's just not forget that our entire world has blown up in the past few months and traveling and participating in clinical trials is going to be virtually impossible, especially putting a boy in a wheelchair on an airplane. So we're hoping that the FDA takes all of that into consideration and lets these patients get infusions in their towns or wherever they are locally, so they don't have to travel. And then we'll show in a confirmatory study, the power of the therapeutic. Dr. McDonald, I don't know if you want to comment a little further on this.

Yes. Again, this therapeutic is not targeting any specific gene deletion or mutation profile. It's agnostic to genetic profile of the patients. I think from my perspective, I think obviously, gene therapy is very exciting. The trials of all -- or the trials that are being conducted or focusing on 4- to 7-year-old patients, looking predominantly at ambulatory end points. I don't think the industry partners in the gene therapy space really have the dosing or the approach that you would take in trying to dose a 70- or 80-kilogram adult-size teenage or patient with gene therapy, I don't think that's been worked out to any degree. The focus is all on much smaller children that are 4 to 7 years of the age. And in addition, I think, obviously, the proportion of patients, even in the 4- to 7-year-old age range that have positive antibodies precluding them from participating in gene therapy is at least 15%. And my guess is in the non-ambulatory population, we're probably going to even see greater proportions of patients being antibody positive. So I think a therapeutic like this, I think, definitely will have a place in Duchenne dystrophy. And then keeping in mind the CK values that we've seen in the publicly discussed patients treated with gene therapy, they have very impressive decrease in CK values, but those CK values still didn't go to normal values. They go from 20,000 to 35,000 down to 2,000, 3,000 or 4,000, so there's still an active inflammatory component even after there's been some partial restoration of dystrophin. So I think even a modulatory treatment like this could still potentially have a place even in patients who perhaps in the distant past received gene therapy. So those are my thoughts.

So Linda, then how do you position the company now? Do you look for a partnership? Or is the data overwhelming enough that you try to move forward on your own? Or is this all dependent on the outcome with FDA?

Yes, Jason, thank you for that. It really is dependent upon the outcome with FDA. So we've been very aggressive behind the scenes and assembling the appropriate regulatory consultants, teeing up commercial scale manufacturing, getting the thought leaders, including Dr. McDonald and his colleague, Eric Hendrickson, who is an expert in understanding quality of life measures and its relationship in Duchenne, all lined up to go to FDA and present our best case. We are also, of course, interested in partnerships at this time, whether it be for future clinical work, which we're not expecting to have to do or for taking this forward through commercialization. But I think, ultimately, our current path will be defined by the response from FDA. We're hoping that FDA is supportive of the filing because it really is to the benefit of the patients. It's not just about Capricor. In fact, it's not about Capricor at all. My wishes for the boys and for their families. Because if I had a child with Duchenne muscular dystrophy, I would want them to get this therapeutic.

The last quick question for Dr. McDonald. So if you're non-ambulatory now, a little bit older boys, young men, is -- would you be treating -- or looking to treat earlier, younger kid, younger boys 4 to 7 or somewhere thereabout? Or do you need another trial to do that?

Well, I think I think, obviously, there's a lot more therapeutics that are under investigation in the ambulant population. So I think the trial design would be a little bit more complicated, I think, in an ambulatory population. Obviously, I think if you're preserving skeletal muscle function, this should have an impact in ambulant as well. In my experience, I think the FDA has actually been pretty reasonable at providing broad-based labeling in the rare disease space as long as there's sufficient safety data that supports usage in a younger population. So I do think since there have been 20% of the patients in this trial were in the late ambulatory phase. So there have been some ambulatory patients that have been treated, but my guess is the FDA would probably want to see a little bit more safety data to extend the label into younger patients.

And again, congratulations on the impressive data.

Thank you, Jason. Have a wonderful day and stay well.

Our next question comes from Mark Swaim with BioPub.

Linda, congratulations. Nothing succeeds like success. My question was already answered, and I didn't know how to withdraw it.

You're welcome, Mark. Thank you.

Our next question comes from Chen Lin with Lin S.S. Management.

Linda, A.J., I want to congratulate for this wonderful results. So as you said, not only for Capricor and also for the patients and very pleased with today's news release and the publication results. My question also mostly got answered mostly around your path forward -- but has been answered, but I just want to hear to congratulate you for the excellent results. I'm so happy for you.

Thank you so much. We look forward to working with you and continuing to deliver on the promise for the boys with Duchenne.

[Operator Instructions] This concludes the question-and-answer session. I would like to turn the conference back over to Linda Marbán for any closing remarks.

Thank you. Thank you for joining us today. Thank you again, Dr. McDonald. Thanks, patients and families. Please stay well during these most unprecedented times, and we look forward to providing updates on this program as they become available. Stay tuned, and thank you.

This concludes today's conference call. You may disconnect your lines. Thank you for participating, and have a pleasant day.