Good. You guys all set? Yeah. We're all set. If you can hear us. Okay. Hi, everyone. My name is Maury Raycroft, and I'm one of the biotech analysts at Jefferies. It's with great pleasure that I'd like to welcome the team from Abeona. We've got the CEO, Vish Seshadri, and Madhav Vasanthavada, the Chief Commercial Officer, and then Joe Vazzano, CFO. Thanks so much for joining us today. Thank you for having us. We're going to do fireside chat format. For those who are new to the story, maybe give a one-minute intro to Abeona and your recently approved transformational therapy for RDEB. Sure. Even if you're not new to the story, we have a new story, which is we're a commercial-stage cell and gene therapy company with the approval of ZEVASKYN just over a month ago. This is for patients with recessive dystrophic epidermolysis bullosa and treating their wounds. With the approval, we also got a PRV grant, and we announced that we are in a sale agreement for $155 million. With those proceeds, we're funded for eight quarters of our operations without even accounting for the revenue generation. We do anticipate being a profitable company in the early half of 2026. Our partner programs with Ultragenyx and Taysha are also doing very well. Ultragenyx's asset, UX111, has a proof of data that is coming up in August as well. With all that, I can just say that we're a grossly undervalued company at a market cap of about $300 million. Great opportunity. Great overview for Abeona. I want to talk about the launch for RDEB. You mentioned Lurie Children's Hospital, now activated as the first qualified treatment center. What specific metrics are you tracking at this side, such as patient throughput and referral volume from other centers of excellence? I'll start off by saying that just about a month into launch, as Vish mentioned, and the feedback that we are hearing from multiple stakeholders has been very positive. I mean, the standard of therapy that exists right now for recessive DEB is just not bending the trajectory of this disease and the progression. With ZEVASKYN, what we are able to bring is, with a single treatment application, the ability to cover large areas of the body, regions of the body, with long-lasting efficacy and outcome. Because it's autologous, we are working with treatment centers to get them activated and Lurie's was one of our first treatment centers that has activated. Now, with that, we are able to track the number of patients. We are learning more about the patients that are getting referred into Lurie's from adjacent areas. As we learn more, we'll be able to communicate the actual numbers because there are steps that have to happen between identifying a patient and eventually placing them on therapy. Overall, the launch is off to a good start. Got it. Can you give an update on the latest number of QTCs that are online or expected to come online soon? When should we expect the next site activation, and what's your timeline to fully activate all five centers? Timeline to fully activate all five is by the end of this year. We are actively working with these various other centers, and every center is working at a different pace. We will keep everyone informed. It's hard to tell as to what's going to be the next center, but the site activation also maps up to our supply ramp capacity. As we get more centers onboarded, that also matches up with how many supply slots we have. That's a good cadence there. Got it. For these five centers, maybe just talk about how many total eligible patients are currently being treated at these centers, and how many do you expect will ultimately get the therapy? Across the five centers, we believe there is about an upper double-digit number of patients that are PGCL eligible. As we have more active centers, yes, they begin to start placing them on therapies. If you look at these double-digit number of patients, even if a fraction of them get on treatment, we have got enough demand to take us through this year and into the first quarters of next year before even triggering any referral activities from other centers into these QTCs. There is sufficient bolus of patients. Just to be clear, the numbers that Madhav is stating are those that are organically de novo patients being treated for other things in these centers already, so not specifically for referring to a ZEVASKYN site or anything. That should even ramp up the numbers a little bit. Got it. J ust from the early experience at Lurie’s specifically, how have patients responded when the new therapy is proposed, and roughly what percentage of eligible patients are open to getting ZEVASKYN? The reaction has been very positive. In speaking with some of the physicians, none of the patients that were approached for ZEVASKYN said they're not interested. It is overwhelmingly positive. Patients have reached out to physicians to ask more about this treatment and the process to getting this treatment. So far, it's been exactly as we had thought that it would play out. Got it. Last time we spoke, you talked about the Abeona Assist and that you've had 30 patient inquiries through that. Do you have an update on just the call volume and interest for ZEVASKYN? First, I'll say that the call volume, the 30 patients that we talked about, is just a sliver. There are other channels, and it's not the universe of patients that have called in. There are many other ways, which are the qualified treatment centers themselves and these physicians who are seeing those patients. We don't have the full visibility into that pool of patients yet. Even if you were to just look at the 30 patients, the initial interest, and you convert a fraction of those patients into treatment, again, going to our supply capacity and the ramp, which we have said about 10-14 patients this year, we will have treated and met our goals for this year and leading into next year. Got it. How do you triage the leads that come in, maybe through Abeona Assist or from elsewhere? Just how are you kind of setting up for these patients to get treated, given the limited treatment window and manufacturing capacity? Triaging the patients, it really depends on patients' preference. I mean, patients that are closer to Lurie's, of these patients who have called in, are more inclined towards going to Lurie's Children right now. There are some patients who want to see as to what other treatment centers are coming up online. The fact of the matter is that even though these are 30 initial inbounds, the level of engagement remains. These are not callers who have called us once and then hung up their phone. There is active dialogue back and forth that is going on with the patients who have called in already. Got it. Makes sense. Your treatment window appears to run from August when you expect to enroll the first and treat the first patient through November before the December shutdown. What operational metrics are in place to ensure that you meet your 10-14 patient treatment goal for 2025? The first and foremost is the number of slots, manufacturing slots that we have prepared for this year. It's more than sufficient to meet that 10-14. Initially, we're going to be operating at a ratio of many slots that we have released will be engineering runs and a couple, maybe in the first month, a couple of commercial runs, and then maybe four slots or so, maybe engineering runs. As we go closer to November, we'll phase out the engineering and ramp up more commercial slots. This is routine practice because we've trained a lot of new people. You don't want to put them all on a commercial run. If you really add up from a slots perspective, we feel that we have enough of a buffer above the 10-14 to be able to supply this year. Got it. So could go beyond 14 if need be or if it works out that way. Correct. Okay. How will the planned December manufacturing shutdown impact ability to meet first quarter 2026 treatment goals, and what mitigation strategies are in place to minimize disruption of patient care and revenue? From a timing perspective, if you look at when the facility shutdown is planned, it's from about the second week of December, and the facility will be back in process for the second week of January. It is very early in January that this site is actually up and running. We are ready to take any biopsies at that time. This is the natural holiday period where patients would also prefer to have their activities planned and everything. It is very minimal disruption for the patients themselves. It is just that you would not take a biopsy in the first week of December trying to treat them in the end of December or something like that. That is minimally disruptive. That is why we choose the December month, because that is when patients even have probably other things planned in. Got it. Makes sense. You plan to scale from four to six manufacturing slots per month by the end of this year to 10 per month in the first half of 2026. What do you have to do to enable to do that, and maybe just talk about the amount of support from staffing that you would need for that as well? To operate six a month, we already have the staff hired, and they're already under training. By the time August comes, we are going to have them fully trained. It's more to do with to ramp up from six to 10, we're going to be hiring more personnel that will be coming on board in the Q2, Q3 kind of time frame. When you're looking at the first half of next year, they should also be trained. From an FTE perspective, we're well ahead in our plan. This year, six a month, the facility is already up and running, and we should be operating at six a month technically, although a proportion of those are going to be engineering runs even by August, September kind of time frame. Got it. So basically, you've got enough in-house already to meet those goals. Correct. In your earnings call, you mentioned leveraging the RVV manufacturing site to expand ZEVASKYN capacity. What technical or regulatory considerations are involved with repurposing that space? That is the space within our GMP space, the compartment that has about four slots added to the six that will take us to 10 by the first half of next year. Right now, we use that space for RVV manufacturing. Once we have done three, four batches of RVV manufacturing, we have enough supply to take us through two to three years even. That area is going to be changed for drug product manufacturing. This is what is going to add the four slots. We have had the discussion during the inspection with the FDA that this is our intent. There is no roadblock per se, but we have to submit the change control protocol to the FDA, and that should be done by Q3 of this year. That gives us sufficient lead time to have that even approved. By 1Q of next year, we should anticipate that those regulatory processes should all be in place. Got it. Maybe for pricing, we thought it was a positive that you ended up with a $3.1 million price for this therapy. We think it's reasonable as well based on the improvement patients are getting. Just how do you anticipate any payer pushback or carve-outs in case a graft fails? How do you think about that? We have not had any pushbacks from the payers. I mean, the reaction has been exactly as expected. Also, the pricing as we launched, it was not just coming out of the blue and putting a price number out there. There is a lot of research that went behind the pricing because if you look at the standard of care, the wound bandage itself on a monthly basis can be $84,000-$85,000 a month for recessive DEB patients. You just mount that and cumulate that. That is an exorbitant amount. Some of the recently launched gene therapies are going into tens of millions of dollars in lifelong managing. It is just not cutting it in terms of efficacy and durable healing. We have had dialogue with payers before coming into the marketplace with this kind of a price range. Right now, where we are in the process, we're obviously discussing with the payers around agreements, and we are standing behind the efficacy of our product, which is the outcomes agreement. It has been received very well. Of course, this is the commercial segment, and we have now also started discussing with the Medicaid channel to open up the access for these patients. Got it. Maybe just talk about your latest discussions with payers and what percentage of insured lives are now eligible for ZEVASKYN. What do insurance companies need to see in order to pre-authorize this treatment for a patient? Percent of lives covered, as we announced, we entered into agreement with a large conglomerate. That's 100 million-plus lives, commercially insured lives that's already covered. These are the agreements that we are having with commercial plans. You don't need to have an outcomes agreement for a payer to cover a patient. In terms of any prior authorization, a prescription really from the physician and then clinical criteria and the discussions that happen. It's a process that, as you have patients coming through the system, there's a letter of medical necessity in the absence of a policy. Payers will have a commercial policy placed in a few months. That's our goal. Got it. With both buy-and-bill and CVS specialty pharmacy models available, how are you guiding treatment centers and hospitals on choosing the optimal channel? What are the operational or reimbursement trade-offs between the two? So we have two channels, as you said, for a hospital to procure the product. One either through the buy-and-bill, and the other is through the CVS specialty pharmacy. We cannot guide a hospital one way or the other. It's really what the hospital will look into their mechanics. A lot of the hospitals have said they will go with the buy-and-bill model because they're very comfortable with it. They've treated many other patients with other cell and gene therapies and understand sort of the mechanics of it. There is an option for CVS specialty as well if they want to offload any of their reimbursement risk, for example. Got it. Maybe comment on what you expect for gross- to- net ratio and walk through some of the assumptions behind that, including the expected payer mix and Medicaid onboarding timelines. The gross- to- net overall at a steady state, we assume around mid to upper teens. That is when we get to in the next part of the year. Early on, we expect that to be on the lower side because most of the gross- to- net is adjusted based on the discounts that are rebates that are given to the payers. The majority of our payer lives are commercially insured lives where these are negotiated agreements. As we have Medicaid that opens up, you have the government 23.1% rebate that goes to the Medicaid channel. Net-net at a steady state we'll have around mid to upper teens. Got it. For financial outlook, you've guided a cash flow positivity in 2026 with just 36 patients treated. How confident are you in achieving this target, and do you still expect manufacturing to be a bottleneck in 2026? We're confident not only in achieving that 36, I think we're confident also in exceeding that expectation because, as I mentioned, by end of 2025, we should be operating at six patients a month capacity. By the first half of 2026, we should ramp that up to 10. You can already see why 36 is pretty kind of a low bar in terms of our supply capacity. It's just the point of neutrality. Anything north of the 36, you're looking at a profitable company. Both from a demand perspective as well as a supply perspective, we think that's quite a low-hanging fruit for 2026. Are you saying what the upside could be beyond 36 and what that could look like? I think we have bookends. We have scenarios. I think the middle of the range could be anywhere around 50 patients because if you're operating at six- a- month, even not counting the 10- a- month, which kicks in some point during the year, let's say six is what you assume, you should be well able to achieve that 50 patients treated. I'm giving you like a 14 patient excess here as a scenario case, which is not out of the realm of achievability. Got it. That's helpful. Once cash flow turns positive, how are you thinking about the capital allocation? Will you prioritize pipeline expansion, commercial infrastructure, or strengthening the balance sheet? This is not an either/or between the three, but there's a little bit of a prioritization. The prioritization is maximizing the U.S. opportunity for ZEVASKYN is our top priority. When we take care of that, it's going to fund us adequately to pursue both, the other two things, which is pipeline expansion. We'll do the pipeline expansion in a very thoughtful way and cadence that there is no distress on the strength of the balance sheet per se. Got it. You mentioned the PRV that you sold for $155 million. How does that factor into investment plans through 2026? What's the expected cost of the new facility you're planning to build for Abeona? So the $155 million plus our cash at the end of Q1 of $85 million puts us north of $200 million. Like Vish had mentioned earlier, that's sufficient to cover two years' worth of operating expenses before accounting for any ZEVASKYN revenues. With that, we're able to fund the capacity expansion, which we think it'll be about $25 million-$30 million spread over about a 24-month period. Got it. Just to be clear, the capacity expansion we're talking about here is beyond the 10- a- month that we're already securing by the first half of 2026. To add even more of that. Right. I s there more about that, just the additional capacity that you're adding that you want to mention? It's basically not a completely greenfield new building or anything. It's in the same floor where we currently have our 10- a- month. We're just taking additional space on that same floor. It is a very limited spend. We have already gotten the designs done, and we're also getting contract bids. We feel good about the later half of 2027 is about the time we would have that go online. Got it. For ZEVASKYN, wondering if there's plans to pursue regulatory submissions outside the U.S., and could this lead to new partnerships with potential upfronts? Also just wondering if you think that patients from ex-U.S. could come to the United States to get treated and what your thoughts are on that type of approach. To understand the opportunity outside of the United States, we've just kicked off a project as we speak to do a scour of at least the major markets, let's say Western Europe and Japan, on what the regulatory requirements would be, as well as what the reimbursement scenario looks like for those markets to inform our go-to-market strategies. We do know from preliminary research there are some centers of excellence like Austria, which is a big EB clinic, and countries in the vicinity actually get patients there to get treated. There could be some interesting models. We want to have this, we should have more of an update closer to late Q3 on this project that will inform if we do it ourselves, what the parameters should be, or if we are willing to partner, what can we accept. We should have more updates on that. Got it. Maybe talk about the long-term view on market penetration in RDEB and how you see the addressable patient pool evolving as awareness of this approach increases. I mean, long-term view-wise, right now, we have identified these 750 patients that are in the U.S., and about 30% of those 750 patients are in these seven centers of excellence that we are looking to target and bring them on board. We also see that some of the patients who are calling us are not genotyped. They have large wounds. They're adult patients, but they're not genotyped. That is also another opportunity to see, okay, how does the market improve? We have said that an average recessive DEB patient has significant areas of their body wounded that they'll require a repeat treatment with ZEVASKYN, which every time they come for a repeat treatment, that's a billing unit. There could be more treatment needs that will happen there. We are looking at recessive DEB, certainly right now with ZEVASKYN, but also life cycle. In addition to what Vish mentioned around ex-US opportunity, there's also life cycle opportunity that we are evaluating because these patients have their fingers and digits that get fused. There is an opportunity when you do debride and you open up, deglove these patients with applying a gene-modified skin sheet that can significantly impact their quality of life and trajectory. There is a lot of opportunity with ZEVASKYN. We are taking one step at a time, but the underlying feedback that we keep continuing to hear is very positive. Got it. I wanted to ask just on the competitive landscape, how you view that and how ZEVASKYN differentiates versus the others. As we've said, I mean, there is a constant burden of treatment, constant burden of wound care that these patients undergo. What ZEVASKYN does is a single application that covers not just small recurring wounds, but large regions of the body with a single application and keeps it long-lasting. We have followed these patients and published data for up to eight years. That is profound, not just wound healing, but also pain and itch reduction. Itch is a huge burden on these patients. Also, the risk of squamous cell carcinoma is so real. By mid to upper teens, there is a very high risk of developing squamous cell. We're not saying that by applying ZEVASKYN, you can avoid squamous cell because that was not our study endpoint. When we look retrospectively over the 11 years of follow-up across all the treated wound sites, none of the wound sites have shown squamous cell carcinoma, even though squamous cell was seen in other non-treated areas of the body. The opportunity to be able to close these wounds and improve their quality of life is profound. Got it. Vish, earlier you mentioned Ultragenyx with their MPS IIIA drug getting approved soon. It is an Abeona homegrown drug, and you guys would be eligible for royalty there. There is also TSHA-102 for Rett Syndrome, which is a few years behind this MPS IIIA program, which is with a different company, with Taysha. Maybe talk about both of those partnered programs and what Abeona would be eligible from those programs. Absolutely. Joe, I think you have this answer. Sure. Thanks. For the Taysha program, we're eligible for clinical, regulatory, and commercial milestones in addition to an upper single-digit royalty on net sales. For the Ultragenyx program, which has a PDUFA date coming up in August, we are eligible for tiered royalties up to 10% in addition to some commercial milestones as well. Got it. And these are not factored into your projections or guidance at all? No. It's all upside. Vish, you also mentioned just how you're focused on ZEVASKYN first, but then next, you may be able to develop the pipeline. You've talked about preclinical programs in the past and have disclosed some interesting proof- of- concept data there. Are there any immediate plans to ramp up investment and to focus on one of those programs? The RS1 program, we even presented data at ASGCT. We think this is going to be a uniquely differentiated asset. These patients, it affects boys basically and retinoschisis and inherited monogenic disease. We are seeing some very interesting properties of the proprietary capsid that we have in targeting certain compartments of the eye. We believe this is going to be differentiated. There are not very many options for these patients. Even if you look at the current clinical trial landscape, there is probably one drug that is being tested. Even there, it is a very small sliver of patients that we need to choose because if you do a subretinal injection, there is this risk of retinal detachment. We have a novel route of administration as well where we are able to transduce these cells, photoreceptors. This is very exciting. We want to take this program to the clinic later half of 2026, and we're on track for that. The investments that are required now, given where we are with our funding and everything, the kind of investment that we need to take this program into clinic, it's a very small spend at a low rate. This is not going to pressure us financially, and we will still be able to sow seeds to see if there are any catalysts that come up in the next 2-3 year timeframe from clinical data. Got it. Madhav, earlier you mentioned just with some of the information that's coming in from the different centers and from the call volume as well, that you guys are quantifying that. Could you provide more specifics on that in the Q2 conference or your earnings update and just what you're seeing with the launch initially? We should be able to have a better handle on what the patient's identified patient looks like. We are hoping by then we will already have prior authorization and insurance discussions ongoing. Q2, maybe biopsy. We've guided Q3, for treating patients. We should have a lot more clarity by then. In terms of the demand and the expectation, it is very much like on track, which we have said that there are already patients that are being identified, and we'll be able to convey more in terms of how many actually there are. Because there are many steps in the funnel, right now, it's hard to predict what's that one good leading indicator for the next quarter sales or something like that. I think that's going to take some experience because patients have to go through prior authorization, and the slot has to be taken, and the biopsy is done. There are numerous steps that get to a patient from the first step to the treatment. As we learn this by the end of the year, we should have a pretty good idea of what's a good leading indicator for the forecast. Got it. So all this information you're collecting, that'll help you provide investors more clarity on which metrics are going to be important for tracking this. Maybe to wrap up, if you can just recap key milestones ahead over the next 6 to 12 months that investors should be focused on. I think the biggest milestone is the launch of ZEVASKYN and performance with 10-14 patients as our target and getting the QTCs up and running and initiated. I think these are the catalysts that are important for our launch of ZEVASKYN. Of course, on top of that, we have other PDUFA action dates for our partnered programs and everything. There is a lot of news flow coming up even just within 2025. Got it. Congrats again, and thanks. Hi, everyone. My name is Maury. And then Joe Vazzano, CFO. Thanks so much for joining us today. Thank you for having us. We're going to do fireside chat format. For those who are new to the story, maybe give a one-minute intro to Abeona and your recently approved transformational therapy for RDEB. Sure. Even if you're not new to the story, we have a new story, which is we are a commercial-stage cell and gene therapy company with the approval of ZEVASKYN just over a month ago. This is for patients with recessive dystrophic epidermolysis bullosa and treating their wounds. With the approval, we also got a PRV grant, and we announced that we are in a sale agreement for $155 million. With those proceeds, we're funded for eight quarters of our operations without even accounting for the revenue generation. We do anticipate being a profitable company early half of 2026. Our partner programs with Ultragenyx and Taysha are also doing very well. Ultragenyx's asset UX111 has a PDUFA date that is coming up in August as well. With all that, I can just say that we're a grossly undervalued company at a market cap of about $300 million. Great opportunity. Great overview for Abeona. I want to talk about the launch for RDEB. You mentioned Lurie Children's Hospital now activated as the first qualified treatment center. What specific metrics are you tracking at this side, such as patient throughput and referral volume from other centers of excellence? I'll start off by saying that just about a month into launch, as Vish mentioned, and the feedback that we are hearing from multiple stakeholders has been very positive. I mean, the standard of therapy that exists right now for recessive DEB is just not bending the trajectory of this disease and the progression. With ZEVASKYN, what we are able to bring is with a single treatment application, the ability to cover large areas of the body, regions of the body with long-lasting efficacy and outcome. Because it's autologous, we are working with treatment centers to get them activated and Lurie 's was one of our first treatment centers that has activated. Now with that, we are able to track the number of patients. We're learning more about the patients that are getting referred into Lurie's from adjacent areas. As we learn more, we'll be able to communicate the actual numbers because there are steps that have to happen between identifying a patient and eventually placing them on therapy. Overall, the launch is off to a good start. Got it. Can you give an update on the latest number of QTCs that are online or expected to come online soon? When should we expect the next site activation, and what's your timeline to fully activate all five centers? Timeline to fully activate all five is by the end of this year. We are actively working with these various other centers, and every center is working at a different pace. We'll keep everyone informed. It's hard to tell.