Yeah. No, great. Very thoughtful. I have some updates I want to talk to you about. We'll chat later about- Okay. -other stuff. Yeah. Yep. Great. Thank you all for coming to the annual Morgan Stanley Global Healthcare Conference. My name is Ryuk Byun. I am the Head of West Coast Healthcare Investment Banking for Morgan Stanley. I have the pleasure of hosting the Abeona Therapeutics management team today. We'll go through a few questions, but recognizing that this is the first time Abeona's at our conference, would love to maybe start with a round of introductions. Thank you so much for having us, Ryuk. I'm Vish Seshadri. I'm the President and CEO of Abeona, and it's a pleasure to attend the Morgan Stanley Conference. This is a very important year for us, the first full year of launch since we got our first approval, so very excited to be here. Thank you, Ryuk. Madhav Vasanthavada, I'm the Chief Commercial Officer at Abeona. Great. So maybe just to start with some high-level questions about Abeona, just so that the audience here and those listening in can get a better sense and hear from you. Abeona has made the transition from being a development stage biotech now to a commercial stage company, and congratulations. What has been the most different or most surprising about operating a company on the other side of approval? Yeah, thank you for that question. In fact, just to level set, our approval happened last year, and we really launched this product in December when we treated the first patient with ZEVASKYN, which is an autologous cell sheet-based gene therapy for patients with recessive dystrophic EB, a connective tissue genetic disorder. If you look at the launch itself, a lot of the transition that you see from being an R&D company to a commercial stage is what we would expect in the natural course. Madhav and myself, we have a lot of launch experiences in our past, including cell therapies such as these. I think what you would normally expect is we've grown the company from being an 80, 90 people R&D stage company two, three years ago to now we're about 250 people with GMP manufacturing and supply. As you can imagine, that in itself is a little bit of a cultural shift because our day-to-day is focused on launch performance and such indicators moving from a data generation kind of remit. That comes with a little bit of a cultural shift. What has been the constant with our teams and our culture is the focus on the patient. Now that we have an approval, we feel that responsibility, making sure that the benefit from this drug is going to be. Every time there's a patient at our site or in a conference, all our employees watch with the same spellbound attention as we always do, and that's really some of the most motivating events for us. That hasn't changed. I think what is different when you talk about an ultra-rare disease, plus an autologous cell therapy with an 84-hour shelf life, with a qualified treatment center requirement of a new team, that multidisciplinary team that you need, which did not preexist. I think that brings with that some unique features of launch that you may not have seen very much with precedents. I think that's really what's unique, and there's a lot of learnings and we're very excited to talk about it. Given where we are with the third quarter really from launch, I think we're laying some important elements of foundation right now, and things are looking good. We're very happy where we are, and that is important. Great. Can you just tell us a little bit around the indication and the current treatment paradigm and the patient journey? The indication really is this is a cell sheet-based gene therapy. This is a connective tissue genetic disorder where patients are missing a functional gene for collagen VII. Collagen VII is that glue that basically keeps skin intact. The skin is two layers, and the two layers are tethered in normal skin, and you lose that integrity in patients that have the deficiency of collagen VII. So what happens over their lifetime is they have blistering and wounding of skin, so the integrity of the skin is lost, and that leads to a lot of complications. They have large wounds that become chronic over a lifetime, infections, sepsis. Nothing is normal in their lives, and it is there 24/7. This is not episodic. They have to live with these wounds all through their lives, and three, four times in a week, they have to undergo wound dressing changes that take six, seven hours, and they have to put bleach in the bathtub. There is so much procedural burden for patients and their families caring for these patients. Over their lifetime, they develop complications such as squamous cell carcinoma or sepsis. For these reasons, you do not see these patients often live beyond their 30s and 40s. So that is the kind of morbidity and mortality that the disease inflicts on these patients. Right. What treatment options are currently available for these patients? For the longest time, these patients have had to rely on antibiotics, bandage changes, really nothing that addressed the root cause of genetic deficiency. It's just in the last few years, you're seeing genetic medicine make an impact in this disease area, replenishing COL7A1. You've had topical applications where the gene is supplied as a temporary basis where as an episomal vector, which has to be reapplied, and such therapies have existed. ZEVASKYN is the first autologous cell sheet-based gene therapy, where we're actually converting a patient's own cells into collagen manufacturing factories. and putting it back on the patient as a one-time application for these wounds that provide multi-year wound healing and pain reduction. We have data that go as far as 12 years now, that was presented at the SID Conference, from a single application. This is really game-changing for these patients because once you've grafted and they take, it's a multi-year effect. That's really the value proposition that's unique with ZEVASKYN. Great. The other product on market is marketed by a company called Krystal. They're about an $11 billion company. In terms of their application and the therapy, what are some of the, I guess to say, what is your ZEVASKYN's differentiation? Yeah. We, as well as the KOL community and patients, everybody that's in this disease space, pretty much unanimously will agree that the two therapies serve different types of purposes. It's not ZEVASKYN or VYJUVEK. We need these two as well as even more therapies to come into space because you have wounds that are of different nature. This is a weekly application, what you have with VYJUVEK. Week after week, you have to apply. Over time, you may be able to heal wounds, but then after you heal a wound, you can go to the next wound and treat that. It is a different way of looking at the problem, where you have large chronic wounded areas you cannot cover, you need a lot of coverage, then you need to go for something like ours, where every round of manufacturing, each sheet of ZEVASKYN gives you about 40 centimeter squared area, and our batch size is up to 12 sheets. You can imagine that you can cover a vast area in a one-time treatment. It is not a weekly application. After you are done, you are looking at all the data we have generated from our clinical studies are from a one-time application for any given particular wound. That is a very different type of value proposition. You can imagine that you are going to need both types of therapies for different types of wounds. But a lot of patients who have recessive dystrophic EB have contiguous and large areas of wound to be healed. It is not going to be one versus another. You need both therapies. Yeah. Yeah. It sounds like just based on the intuitively what the different applications, I would imagine that you have at the edge, as you mentioned, on the durability side of the equation. Absolutely. If you look at even our commercially treated patients, they are all pretty much prior treated with VYJUVEK. I do not even know if we have a VYJUVEK naive patient- No. Oh, yeah. -in our commercial setting. That unmet need basically is underscored by just that fact. Yeah, great. I think that's a point that people probably don't fully appreciate, so thank you for walking us through that. Now, shifting gears a little bit into the commercialization. What have you learned from the launch, and what will it take to really commercialize an individualized cell-based gene therapy, not just scientifically, but operationally? Yeah. I think this is a very important question because we're learning from the launch. I think the unique features that I mentioned, the qualified treatment centers that we need for a therapy like this, it's not only an autologous cell therapy in an ultra-rare disease, but it's also something that has an 84-hour shelf life. Qualified treatment centers, it's a finite universe of centers where multidisciplinary talents come together to form a team which did not pre-exist. We have had to create that team that has the surgeons, the anesthesiologists, the EB physicians, all knowing how to care for EB patients. When you put those two requirements together, basically what it necessitates is a very coordinated, ultra-coordinated patient treatment journey, where before even you undertake a patient and take a manufacturing slot, you need all these different actors to commit their times at certain time points and lock that in before you can take. It is almost like a solar eclipse needs to be aligned before we can take a manufacturing slot, and everybody understands. That is the one unique aspect of this launch that we all have to understand. The second aspect of it is our phase III study of 11 patients in one site, great results, Stanford. Every qualified treatment site that we activate basically wants to replicate that success in their site. So it is almost like the clinical evidence or burden thereof is only partially addressed by a phase III study. You have to replicate it in a different scale here, and that is part of laying the foundations, which is what we are going through right now. The third aspect is the patient-to-patient communication, because these patients have a very strong network, and patients rely on patient testimonials to see if this is the right therapy. So I think that is also another unique aspect of our launch, where we have a strong together network of patient ambassadors who have been through our therapy, either in the clinical studies or from a commercial setting, and they are sharing their experiences with other patients. When you look at all these types of factors, what it means is this launch is going to be atypical in that you need a pretty good timeframe for a foundation setting. It is not like an off-the-shelf therapy where you open the floodgates and everything is going to flow gushing out. It is going to be set the right foundations, activate as many sites as we need to, let every site accrue that experience, and let that be shared across. When you do that, you are really launching at that time, right? Yeah. That has to be appreciated. Got it. Thank you. You had a great announcement today. It looks like you have added a new treatment center, so congratulations on that. Can you talk about how many qualified treatment centers you have onboarded? As the network expands, where is the principal bottleneck shifting? Is it patient identification? Is it referrals, onboardings, hospital onboarding, scheduling, reimbursement, or manufacturing? Yeah, no. Definitely, it's great news for these patients, especially in the Florida area and the entire Southeastern United States, to have another center. University of Florida, it's a good institution. They have an EB clinic and well-coordinated. It's important to get these centers on board because these patients, as we talked earlier, Vish mentioned, they are living with so many comorbidities. They are looking for centers that they can trust to get a surgical application. That's really been on our ramp. In terms of the bottlenecks, as you talk about, it's not a bottleneck. We wanted to get seven centers onboarded this year. Now we have more centers that are coming on board, and to us, we look at that as a long-term leading indicator. The more centers you have, naturally, you'll have greater access to treatment. But for us right now, we are focused on getting the existing centers that are already activated to start treating more patients. It's always a little bit of a learning curve, institutional learning curve, to treat the very first patient in a setting. Once you have treated a first patient, and once you've gone through the motions of who's doing what kind of contracts do you need with insurance companies and the payer and process work, you're going to get better and better at it. In the first quarter, we talked predominantly most of our patients, we have reported so far three quarters of revenue growth, right? The bulk of those patients came from Chicago Lurie Children's and from Stanford. In the second quarter, we said we have Texas that has biopsied a patient, and CHOP has biopsied and treated a patient. That's exactly what we are focused on, is getting more centers to understand where the bottlenecks lie, and the bottlenecks lie in different places with different institutions due to just different payer mix that they have. Our role is we are trying to help cross-pollinate them and share the learnings so that we can do things differently and better. Our goal is to have some of these treatment centers that haven't treated yet treat as soon as feasible. Yep. Okay, great. Thank you. As you mentioned, it's an iterative process. You're constantly learning. What have you learned from the first commercially treated patients and their caregivers that has caused you to change or refine the launch strategy, if at all? Yeah, what we have learned, what we have reinforced really is the type of impact that this therapy has made. Not to repeat what we have said consistently, but it's sometimes important to remember the impact that this therapy has had. Recently, we've had patients from our clinical trials come to our company and share at the town hall multiple years later as to how the wounds have healed. We are arranging multiple sessions with these patient communities so that they can learn from each other, especially the initial set of patients that have gone through the process, having them talk to additional patients who may be wanting to learn more about what the process entails, who may be anxious about what does biopsy really mean, and what does the procedural component involve. That is one part of the learning. The other part of learning where we are realizing is that this is certainly a cellular therapy makeup. It's not a prescription and a pharmacy product that you can go and take the product. I think we've realized that very well. But what is new here is that it's an ultra-rare disease condition, so there are only a finite number of treatment centers, and you're trying to bring an EB medical dermatologist together with a surgical dermatologist, along with the cell and gene therapy expertise within an institution. So you're trying to mobilize three different parties along with the patients and the families. That particular learning curve, as we talked about more in detail at the previous earnings call, it requires time, especially since the expiration is only 84 hours. As the centers start treating more and more, then know as to, what can we do differently and better? We've also learned that some of these patients are fragile in nature, and so we just need to have more patients actively identified on top of the funnel so that even if there is any attrition that happens in the weeks due to factors beyond our control, we are able to keep the patient flow and the cadence going. Those are really the more important ones. We are pretty confident that this is just getting better, especially as more centers come on board, that patients will begin to flow through. Great. Thank you. How should we think about what the potential best leading indicators of launch momentum may be? Can you just try to give us a sense? Leading indicators for launch momentum, first and foremost, is just the interest that we are seeing from the community for this therapy, and the high unmet need that is existing. We've talked about the comorbidities, we've talked about the extent of wound area. About 30% of the body is wounded. Each percentage is roughly the size of the palm of the patient. So 30% is massive regions of the body, and the gels are just not physically enough to be able to cover that. We've said we have about 100+ patients that physicians out in the community and at the qualified centers believe are good candidates for ZEVASKYN. But now the challenge is, it's a multi-step process to bring them on, and the lead indicators can be different because there are so many stages that the patients have to go through. The best or the most definitive indicator is naturally a patient being treated, because when a patient gets treated is when we recognize revenue for that patient. Yeah. That's exactly what we have been reporting in the previous quarters. We've kind of stopped reporting in the prior quarter about how many biopsies are planned or how many are in the manufacturing process, just because of certain things that are beyond our control. We have not really guided with regard to what's happening in the immediate upcoming quarter. But again, as more centers get on the map, that's going to be the long-term pull that's going to come from ZEVASKYN. With the current footprint of seven centers that we had, we have captured about 40% of the addressable market, is now living in the states where the qualified treatment center is located, so that's also going to help expedite the access for these patients. Thank you. How should we think about repeat treatment episodes over a patient's lifetime? Also, could patients return to treat additional wounds, and how might that influence the overall long-term utilization? I think for the foreseeable months to quarters, we should have new patients building up. Most of the patients are new patients that are going to get treated. I know this is usually a question we keep getting from investors. So when should we expect a repeat treatment to come in? If you look to our clinical trials, which is, if anything, that's a precursor we have, it was about a year and a half of gap when a patient came back again for subsequent clinical trials. But that also was a lag because of the clinical trial protocols, et cetera, that we had to write. When we talk to our doctors, they believe that six months is the least amount of time, maybe a year after the first treatment episode, that if the patient wants to come back, that we expect that recurrence to happen. Our initial is going to be the new patients treated, and then the recurrence should happen at some cadence. We anticipate about two treatments per patient in the lifetime of the patient, just because of the extent of coverage area and what ZEVASKYN can provide with each treatment. Each of the treatment is a billing unit, because we need to biopsy the patient every time they come back. Oh. In addition to these, we have talked about other areas of unmet needs, such as hand contractures is one area which is a pretty high unmet need for these patients because their fingers are fused and we are really looking into applications as to how we can potentially put ZEVASKYN over in these hands. There is precedence from our clinical trial experience- Yes -for the repeat treatment as well. Got it. Thank you. In terms of patient access and reimbursement, maybe starting with access question first, what are some of the challenges or barriers for a family currently considering ZEVASKYN? From an access standpoint, we are seeing most of these patients, a little over 50% patients are commercially insured patients. There are a lot of Medicaid patients also that we have, and about 10% are Medicare patients. We have coverage. We have more than 95% of commercial lives are covered by ZEVASKYN. We have baseline coverage for Medicaid states. For patients and caregivers, there is hardly much of a copay or any kind of financial obligations for them. It is primarily the centers that have to go through the process of clearance and authorization, and that really depends on the patient, the plan, and the contracting that needs to take place to arrange on a payment rate basis. That is nothing new about ZEVASKYN. It typically happens with every center. But once you have a particular plan, having a contract rate arrangement with the center, then the subsequent patients, things get better for those patients. In terms of the access itself, physical access, we have travel support programs. We have copay support programs for these patients, Abeona Assist, that we are going at great lengths, and we are getting good feedback from patients who have used these programs. Thank you. On manufacturing and execution, autologous therapies obviously start with material from the individual patient, so I would imagine that manufacturing reliability and consistency is probably pretty consequential. What have you learned about scaling this process commercially? A lot of our manufacturing processes and controls that we have in place have been learned and informed based on our clinical studies. We had the 11 patients we treated in the VIITAL study, which was really the training set to develop a lot of that, and that is what led to the BLA. The challenges that you face in scaling are less to do with the parameters that we have well-studied. I think they are more to do with tests and controls that have been introduced during commercial launch, where we had no prior experience. Despite that, I think our teams have been very nimble in learning. For example, I will give you, there are sterility tests that were introduced by the FDA during approval, which we did not have in clinical trials. Similarly, with the identity test, the pan-cytokeratin test that we talk about in the Q2 earnings call. There are things that are new in terms of how the product is released in the commercial setting, which is a little bit of more of the steeper learning curve. I think the parameters that we had a lot of experience have behaved rather very well. I would say even that the predictability of turnaround time is quite impressive. The range is about 23-26 days. Standard deviation, less than two days there. That is something that we will continue to monitor, but that helps us with the planning and logistics in a very predictable way. I would also say that with that one exception of the lot that had a low yield that wasn't billable, pretty much the overall experience with the robustness in yield output has also been quite encouraging. I think it's a continuous learning process. We have to be nimble, but the manufacturing science and technology that looks at trends and reacts quickly, I think that's really what distinguishes a successful manufacturing organization, and we're fully committed to continuing that. You mentioned the yield. What improvements or initiatives do you have internally on how to enhance or improve the yield side of the equation, and how should stakeholders assess manufacturing consistency as the launch continues to mature? Yeah. There are two categories really that relate to supply robustness. One is binary events, which can actually qualify or disqualify a lot. So you have nothing or everything kind of a thing. If you put that aside, if you look at purely the quantity of sheets or how many sheets can you make from a lot, in the VIITAL study, in the clinical study, we were only manufacturing six sheets maximum that can be applied to a patient. Since there, I think doubling from there to a commercial output of 12 sheets is a pretty big jump, and there are multiple parameters that influence how many sheets you can make in a lot because there's attrition of cells. So there's variability in how many cells you can get from a biopsied sample. Multiple factors, age of the patient or location of biopsy and all these things, or even the quality of biopsy. Once you have the cells, there's going to be attrition through the manufacturing process up to the last day and at various stages. The more cells we can have upstream the better the chances we will have the end product. What our teams have done rather well is for a product like this, which has primary cells, it's not even suspension cells that have to form gap junctions and make sheets. What they have done well is that you cannot have a process that is too rigid on day two, you have to go to that step. Day eight, you had to go to that step. Cells don't fit in a box. We have let the cells tell us what is the right appropriate timing to move from one step to another, and I think there's a lot of that optimization that happened in late-stage clinical development, even leading to the BLA submission, because that's where you have to lock in parameters. What's the flexibility that you're affording there? Beyond that, we have maximized many other steps, like how many cells or sheets that we lose in the process, what are the reasons why we may lose them, and are there trends that tell us that any small tweaks can actually improve that? That's a continuous process, and I would say having an average of nine sheets per lot, despite those outlier lots where you had the three sheets, I think it's a very good metric, and we're going to strive to only keep beating that. Great. As most folks here are equity investors, as they're thinking about Abeona as part of a potential investment, what are two to three proof points for your execution that investors should pay particular attention to over the next couple of years? Yeah. I think if you take the very near term, I'll start with 2026. A robust QTC network is the number one thing, and then as many QTC as we can have treated a patient and gained that- Receiving qualified treatment. Qualified treatment center. Yeah. Having treated a patient and starting that experience, I think that's the first base to achieve. The second is the outcomes from those treatments being shared in a peer-to-peer way. Has that happened? How many centers have had that positive experience? I think that's the second big base. After that, you can look at, okay, are we growing quarter-to-quarter and getting to a cadence to some of these sites, right? Because at least the early launch sites should get to a steady cadence, which we can expect in some point 2027 and beyond. I think when you look at the two-year window, what we should try to achieve by then is a free cash flow. Have we breached that three? I think anything greater than three patients a month is going to be free cash flows positive, right? That's something that we should look for, and it's very hard to predict when exactly we will hit that, but that's going to be an important inflection. Yep. What do you think the market still misunderstands about Abeona? The first thing is under-appreciation of the opportunity we have with ZEVASKYN. I think the speed of the launch does not define the peak opportunity. That is number one. This is foundation laying for, it's almost like an extended clinical trial phase through launch that we have to establish what we did with one site, has to happen with 10 or whatever the number of sites that we will eventually have. So that's for recognition of that. The second is, this is not a product that has an endpoint of loss of exclusivity or things like that because we don't see a generic entrant. This is something that we're going to own for a very long time. That is underappreciated. We're also not seeing emerging therapeutics that can achieve the type of product profile that we are seeing with ZEVASKYN. Again, these are features of ZEVASKYN that are very underappreciated. Then I think people have to also look at, we don't appreciate fully the programs that we've partnered. There is one PDUFA date that is within the next few days, and we have royalty streams coming from partnered programs, with Ultragenyx and Taysha. I don't think we have appreciated the value that's locked there. Some of our early programs, that is understandable because we have to reach clinical proof of concept, but when that happens, I think you're going to see a very different kind of company here. Great. Well, thank you very much for your time today, and good luck with the rest of the day and your meetings. Are there any questions from the audience? All right, well, thank you. Thank you very much for having me. Thank you. Yeah, of course.