Okay, hi, good morning, everybody. Welcome to day three of Cantor's Global Healthcare Conference. I'm Kristin Kluska, one of the biotech analysts. Really happy to be sitting with a company that I cover, Abeona Therapeutics. Big year for you guys. Joining me from the company is Dr. Vishwas Seshadri, the CEO and Director, and Dr. Madhav Vasanthavada, the Senior Vice President, CCO, and Head of Business Development. Great to have you both. Thanks so much for supporting us this year. Thank you so much, and pleasure to be here with you, and excited. Great. So Abeona is primarily centered around one near commercial asset, pz-cel and RDEB. Why do you believe that this is a product that could check off many key boxes for patients in the space? Thank you, Kristin, for that question. Before we talk about checking off the boxes, of course, just as a reminder, RDEB is one of the most debilitating dermatological conditions known to humankind. It not only reduces the quantity, but also the quality of life of the patients that live with this Collagen VII deficiency. And when we talk about checking the boxes, just in terms of what their needs are, unmet needs, these patients live with a lot of burden, which is not just clinical in nature, but also economic and humanistic. And the manifestations of this disease are in the form of large, chronic wounds that over time, not only inflict a lot of pain, but also the risks of cancers and ultimately taking the lives of these patients. When we talk about the biggest needs that these patients face, it's how do we heal these wounds in two dimensions? One is coverage, because their entire body is covered with these wounds, so how can we achieve that coverage? And the second, how can we meaningfully give them durability of a therapy, right? These are the two things that Abeona has always been focused on with pz-cel, and the evidence comes in two ways to us, two parts to it. One is the clinical evidence that we've seen with pz-cel, and that is two trials. One was the phase I/II-A study, which has up to eight years of follow-up of patients that is published in literature now, and also the VIITAL, which is the phase III randomized study, that is the pivotal study for our regulatory path. What we've learned through these studies is that pz-cel offers lasting relief for these large and chronic wounds, both in terms of pain as well as healing itself. The second part of that evidence of how we check those boxes is what patients tell us. Patients are telling us that their experience with VIITAL makes it worthwhile, and they would... You know, "This is the best piece of skin that I've had in my body." These are the types of testimonials that we're hearing from nearly half of the patients that have been through our studies. This is the first time in my career that I'm actually talking to half of the study population directly, right? This is something we've not seen. So this is very exciting, and we're working as hard as we can to get this therapy to patients as quickly as we can. Okay, great. So you had very successful phase III data. You filed for approval, and then back in April, you received a CRL that was based on CMC requirements. I have to give you guys a lot of props because the level of transparency that you've shared to the street has been extremely thoughtful. It's something we don't often see in biotech, so while I, while I can respect it wasn't the news you wanted, appreciate that you've been so open to us about everything going on. So maybe walk us through essentially what happened at this point, at that point, before we segue into where you are now. Sure. April, we received the CRL from our first BLA submission, and the good news is that the CRL actually de-risked our program in many ways. So there was no clinical observations- Mm-hmm. In the CRL. So there's, that validates our clinical data package. The CMC observations that were required for us to complete before we make a resubmission, we've spoken at length about what they are, and we're happy to share that we had a very successful Type A meeting with the agency. And if you take a look at all the list of items that they've asked, we've pretty much checked off everything- Mm-hmm. With the exception of one, where we had full alignment with the agency on what is the goalpost, what do we need to produce to actually get the resubmission done, and that clarity. With that clarity, we're finishing up the last few runs of that outstanding item, which is the rapid sterility testing. So once that is complete, we believe we're ready to put that package together and do a resubmission. We're on track for the second half of this year. Okay, and I know they haven't formally reviewed all of these, all this new work that you've done since April, but have they, you know, essentially, like, taken a peek at it quickly to say, like, "You know, this is essentially, like, on track of what we would be looking for"? Has any of that dialogue occurred? That's right. Okay. So, for the vast majority of items, we've shared the data and the reports that have come subsequent to doing the work to address these deficiencies. They've taken, they reviewed that. In fact, for the Type A meeting, we have to submit these things thirty days in advance. Mm. Okay, yeah. -to give them review time. So they have acknowledged that these appear adequate to address those items. With the exception of one thing that we did not have the data at the time of submitting- Sure ... that, we at least know the protocol has been reviewed, and we reached alignment on the protocol, so we're generating the data on that. Okay, and then, you've obviously are kind of in a creative position here, right? 'Cause I think a lot of companies, when this happens, sometimes the FDA will just say, "We'll look at it when it's all done," but clearly, you've been kind of working alongside them during this process. Is that because of all the designations you have in place? Was this something that was kind of agreed upon when that CRL came back in April? ... Yeah, I think, the FDA is certainly doing things a little atypically when it comes to- Yeah. -rare diseases with highest unmet need. We do have the designations like RMAT and Rare Pediatric Designation, Orphan Drug, Breakthrough, and all that. Is it because of those designations per se, or is it just a new way of, the FDA working with, companies when it comes to these types of ultra-rare diseases? I think, there seems to be that, you know, collaborative way in which- Mm-hmm. The agency works with us very responsive. If we want to have a question, we don't have to wait for a Type B or a Type C meeting to have that. It's a matter of reaching out to the agency, and the reviewer that's assigned to that particular question or part of that BLA review is giving us those points of feedback almost real time. So that's very encouraging. It looks like, you know, they're interested in seeing such products come through. Okay, thanks. So I have to say, a couple of years back, when everybody was still in clinical development, investors would ask all the time, "Do we really need multiple therapies for RDEB?" And now that some of these products are commercialized, it's more apparent than ever that, yes, that is 100% indeed the case. And, you know, you've always characterized yourself as not being their competitor, but rather using the term complementary, which I really like in this setting. So walk us through what a typical patient's journey is going to look like with an arsenal of treatments available, should you also be part of that? Absolutely. I will say that, you know, the conviction that our therapies work complementary to what's already approved is only building stronger and stronger as we talk to both the patient community as well as the physicians. I'm gonna turn it over to our Chief Commercial Officer, Madhav, here, because he is the one that's been in touch with both the patient caregiver, stakeholders, as well as the, you know, the, physicians. So the patient journey has been pretty well mapped now. So, Madhav, I'll- Yeah. We'll want you to take under. Thank you. Thank you. No, but, to your point, over the last couple of years to now, our excitement just continues to increase as we talk to the various stakeholders, especially the patients, advocacy groups, also directly with patients through market research, to position as to what is this new treatment going to look like, pz-cel, and the type of profile that we have had. We've been speaking with physicians, and it's very reassuring to know that there is interest and there is an unmet need that they see for, you know, pz-cel technology, and it's primarily because of the nature of the disease, right? Because the nature of the disease, it constantly wounds, closes, wounds open up again, closes again. So if you have a treatment option that can, with a single treatment episode, can provide multiple years of durability, pain reduction, and all of these types of outcomes, there is a strong interest for a treatment option like that. So that's what we continue to hear. Complementarity is definitely the way to go because these are non-systemic treatment options. If these were systemic treatment options, then yes, there would be an argument about sequencing these options or, you know, what happens when concomitantly... I will say, of course, we have not done clinical studies with giving both treatments to the same patient at the same time just because of the nature how it has been. But with that said, there is generally, when we talk to clinicians, the immediate gravitation is, yes, you have different wounds, you have different modalities of treatment options, you need different, you know, solutions for these patients. Okay, thanks. And while that point, again, is starting to be understood a lot more, with it comes the question of, well, is there going to be any difficulty around payers or reimbursement? And I know you guys are already doing a lot of work. You've issued press releases around this, so tell us what that might look like from their perspective. Yeah. I mean, to payers, we've obviously talked to the big boys, big payers - United, you know, the commercial insurance, which is about 60% of the payer mix. We've also spoken with Medicaid carriers, and there's willingness to defer the clinical decisions to physicians. They are not looking to manage this treatment space, and the reason for that is because they appreciate when they look at these patients and the phenotypic presentation, they appreciate that these patients have such significant wound areas that current treatment options are just going to fall short of being able to treat such large areas of the body. So that's one part. The second part of it is the ultra-rare, again, nature of the disease. If you put this treatment paradigm versus the alternate, which is years of wound burden, you know, and the healing of these patients, it's almost $250,000, the current standard of care. There was a paper that DEBRA published, which talks about the burden of, you know, treating these patients with surgical procedures, squamous cell carcinoma, dilatation, surgery, et cetera. So if you do nothing, there is a significant amount of cost, and then they realize that, yes, you do want to be able to cover these kinds of treatment options. So payers have not really talked anything like a step edit, or you got to treat this first, try to close it, and then move on to another, just because they know that it's a non-systemic, again, disease. You have certain areas of the body that will require a much bigger guns, and then you have different areas of the body that caters itself to a more convenient kind of a treatment, you know, weekly dosing. Okay. Yeah, there's also another publication, I think, in JAMA, in terms of pricing models, in terms of thinking about lifelong treatment courses for these patients- Mm-hmm. And the different therapies. So I suspect you guys haven't announced any pricing yet, but can we just broadly talk about the value of the cost model relative to other therapies? I think the goal is patients would either require pz-cel once to twice in a lifetime. Yeah, I mean, if you look at the lifetime value, it's huge. Again, even if you keep the treatment options aside and just the cost-effectiveness of doing nothing and just trying to maintain these patients, there is a justification of having a pricing for a one treatment that can produce multiple years of benefit, priced comparable to recently launched one-time gene therapies. Now, on top of it, if you add the alternate, which are these continuous, you know, treatment options, as you point out, Kristin, and the paper that got published for B-VEC, where lifetime cost could mount to $15 million, assuming $300,000 cost per year, which we now know why you were pricing at close to $900,000 per year, it's just a, you know, huge, right? So we have done budget impact analysis, where we believe that with single episode of care, which can cover up to 480 square centimeter area, and you do the cost analysis to try and close that wound for multiple years with a treatment that has a continuous basis, and you can do the price comparison. And we have done that budget impact analysis, and we are currently, you know, discussing with payers to see what that would look like. I think the equation is going to pan out, you know, very, very favorably to be able to offset some of these costs of treatment options. So we want to come to market justifying the value and, you know, and showcasing the value that we are bringing to the system, and also to the, you know, the rest of the patients as well as the payers, I mean, the physicians. Sure. And you've guided that peak sales could be around $500 million plus. Give us a sense of how you kind of do a back of the envelope calculation to get to that. Yeah. So $500 million plus, the assumption is that you have to be manufacturing around 300-350, you know, treatment site patients per year. So, and currently, our manufacturing capacity is, you know, after we ramp up with our initial, you know, infrastructure we have at Cleveland, it's going to be around 120 patients per year. So we obviously, you know, will need to expand our manufacturing capacity, and we continue to see that the demand is going to outstrip the supply that we have right now. Okay, thanks. And then, assuming again that you get an approval, what is it gonna look like in terms of sites that are ready and able to do this procedure, and just patient willingness to travel and stay for that procedure, the follow-up? Yeah, in terms of sites and patient willingness to travel, our strategy is to, you know, there's already patients who are going to centers of excellence, not like weekly or monthly, but they do visit for procedures, special procedures that I was mentioning earlier. So there is trust that already is established with centers of excellence for these patients. So we are capitalizing on that. We are approaching these centers that have the experience in taking care of big number of recessive DEB patients, and some of these centers have dozens of recessive DEB patients that they continuously, you know, treat as part of their natural history study and ongoing care. The fact that we are getting a lot of enthusiasm from these five to seven treatment centers over the last 12 months, 18 months, because we are in the process of building these centers to be able to onboard them and give them pz-cel similar to other autologous processes, is pretty profound and quite telling in itself that there is enthusiasm. These physicians and faculty champions are the ones who are working with their legal team, the contract team, to bring these centers on board. You know, with that said, our centers are going to be geographically, you know, dispersed across the U.S., so that kind of minimizes sort of the burden. When we talk to the patients, the burden about their willingness to travel, they have said they already travel three to four hours. They prefer driving. Oftentimes, they prefer driving as opposed to flying, but also flights is not an option, not a problem. Sure. And when we talk to the physicians that are out in the community, they have shown willingness to refer these patients over to these qualified treatment centers. So far, all the data are pointing out to be a very strong underlying, you know, excitement, not just clinically, but also, yes, operationally, you know. And we are putting patient programs in place like any other, you know, cell and gene therapy company does. So we will have a really robust, you know, program in place. If I may just- Please. Add, also, when we look at what's the peak potential for pz-cel versus how we are launching, right? Mm-hmm. Madhav talked about five to seven centers that we will have ready and ready to go on launch. We are hearing from the patient volumes in these centers already organically, without even referrals coming in, appear to be sufficient to take up our manufacturing capacity that's going to be there at the Cleveland site, which is about 10 a month, kind of rate, that we can do right now. So in order to get to that peak potential that you were asking about, it's going to take an expansion in our capacity. So that will go hand in hand with when we expand beyond the first five centers. Mm-hmm. And when we are ready to build another line of manufacturing, I think you're going to see that happen, but such triggers will happen after launch, so we'll be having more updates on how we look at that, whether we have to ramp it up to another three hundred slots in a year, or is it more like, you know, is that even sufficient, or do we need to aim higher? Because we're learning about the epidemiology of the disease as every day, because this seems to be what we know from claims data and what we know as currently genotyped patients is one picture, whereas with more therapies coming to market, we're seeing that there's more patients that are actually getting appropriately labeled as DEB patients. So I think this is a little bit of a dynamic, and we will continue to evolve and learn with more patients, you know, coming out of the, you know, community practices into these centers. In terms of your earlier trial experiences, have you gathered any interest from patients that receive pz-cel that maybe want to go through that second cycle to target some wounds that weren't addressed the first time around? Yes. The currently ongoing clinical study- Yeah ... that we have open, it's called a 302 study, and we've treated four patients already in that study, and all of them are patients who came back from VIITAL. Okay ... or phase III study, who wanted their other controlled wounds or any previously untreated wounds to be treated. So that itself is testimony to the willingness and interest of these patients to get their wounds treated again. Yeah, I mean, we were at a DEBRA Care Conference this summer. A patient—when we presented Abeona upcoming, patients reached out to us, and this was the first time. They were introducing that I was part of your clinical trial. I came back for a second one, and this is the best piece of skin that I have on my back. And they were asking: "What else can you do? What else can I do? Can I infuse this into myself because I want a systemic treatment." And, you know, just the excitement from these patients for them to come in, and I want to talk to other patients about this product, you know, that really moves us, right? I mean, it's one thing to say that even for a single patient, that level of impact, they have no other option right now. You have the option as the topical application, Filsuvez and Vyjuvek. But to be able to cover vast areas of body durably for multiple years and through patient research, when they see the before and after, I think that really is another aspect that moves, because they are having a visual for what my wounds could look like within that, you know, six-month kind of a frame. That's pretty profound. Yeah, I mean, the power of advocacy in rare diseases is just incredible, and these communities just really get to know each other so well that one success story, they just- It breeds- ... want to tell everybody in the community, right? And it is really just incredible to see their success and to share it with the people that understand what they're going through more than anybody else. Yeah. Yeah. Yeah. Another thing we are hearing almost from every patient that has been through pz-cel treatment is: Can we somehow apply these for these hand surgeries? You know, they have pseudo-syndactyly, which is the mitten deformities. Mm-hmm. And they undergo hand surgeries to separate the digits, which tend to fuse, and they're like: "Can we apply pz-cel here?" Obviously, our pz-cel comes in a very defined shape, which takes on for larger areas of the body, but in terms of their unmet need, they're looking at how else can we take this technology and apply it, so these are all giving us life cycle management ideas and opportunities, very excited to learn more about those as well. Yeah. Well, while I have you on stage for this platform, just wanted to mention that I actually cover two other companies that benefit from collaborating with you on some really great programs that you initiated on, brought in. So given that you're one of the pioneers in the rare disease space, what are your hopes and goals for the future, given some of the breakthroughs in science we've seen, and the FDA has been a lot more open these days to really try to understand these diseases better as well? Yeah, it's fascinating, right? I mean, we are a cell and gene therapy company. That is really- Yeah ... what our core competency is. We have this ex vivo cell therapy platform with pz-cel. We also have in vivo with our AAV platforms. Of course, you mentioned we have collaborations with some other companies. Of course, we're going to have the Taysha- Yeah, ... presentation next, and also Ultragenyx, they're- Yes ... advancing some of our other assets, and we're also in the preclinical setting. We have some proprietary AAV capsids that have demonstrated interesting tropism to specific compartments of the eye. We're very excited about the prospects, and we're not talking very much about it because pz-cel has to reach- Of course ... the finish line. Yeah. But we are preparing, in the, you know, in the meanwhile, to have these programs take off to human clinical trials, and we're just going... You know, it's waiting at the edge to go out of the gates once we have this, pz-cel to the finish line. So on that front, you will hear us, the FDA being more open to looking at endpoints differently. How do we have surrogate endpoints that may actually, have, you know, studies reach their goals earlier? I think those programs lend very well to some of the other indications that we're looking at in our preclinical pipeline. Yeah, it's one of the most exciting times, I think, to look at this space holistically, and I wish all these patients get the opportunity to have a treatment. So with everything we talked about in summary, your stock's trading roughly around cash and what the value of selling a PRV could look like. Of course, if there is a sunsetting, which we hope there isn't, you won't be part of that process. Mm-hmm ... given the timelines of when you actually got that designation. So what do you believe is the biggest misconception in the market here? We're talking about $500 million peak sales, yet essentially $0 valuation to it. So what's the misconception, and why invest in Abeona now? Yeah, I can think of a couple of, you know, points about why we're so undervalued. One is, of course, the company has gone through a turnaround in the last two to three years from where we were, and both from a financial perspective and the, you know, overhangs that we've had- Mm-hmm ... but also from the progression of our clinical program. I mean, pz-cel has really graduated through very important milestones in the last two, three years. I think you can say there is misconception along two dimensions. One is, can this company take this complex therapy all the way through regulatory approval, right? I think that's one part of the overhang, and you've- we've got a CRL this year, so it can definitely raise. But I think we have addressed a lot of those CRL items with the exception of one, which is already getting done. And so we feel very confident about making the resubmission, and this therapy will get approved. It's just a matter of when, it's not if. It's, it's gonna go there, right? So that's one dimension. The second dimension that we're trying to clarify is, what is the role of this therapy with other approvals that have happened in the same disease spaces? And I think this is where the conception of, okay, there is still an unmet need, and patients are gonna need all these therapies. I think that to really crystallize, it's gonna only happen with more and more patients and caregivers, as well as the physicians coming and talking about the unmet need. And you have about, you know, it's been a little over a year now, since the Vyjuvek approval. This is great because the adoption has been amazing. Yeah. So that tells a very good story for awareness about this disease, and I think you're going to see we'll soon bridge that gap between where we should be valued and where we are today. It's you know, it's only exciting. It's very motivating. Yeah. We're just focused on execution, and I think the results will come, right? Okay. Well, thank you so much. We're wishing you all the best during this filing process. Maybe this time next year, we'll be on stage, and you'll be a commercial stage company. Looking forward to that. Yes. Thank you so much. Thank you. Appreciate the support. Thanks, Kristin.