Abeona Therapeutics Inc. (ABEO) Earnings Call Transcript & Summary

Good day, and welcome to the Abeona Therapeutics First Quarter 2025 Conference Call. [Operator Instructions] Please note, this conference is being recorded. I will now turn the conference over to your host, Mr. Greg Gin. Sir, you may begin.

Thank you, Ali, and good morning, and thanks, everyone, for joining us on our first quarter 2025 results conference call. During this call, we will refer to the press release issued this morning announcing the financial results. It's available on our corporate website at www.abeonatherapeutics.com. We anticipate making projections and forward-looking statements during today's call, which are made pursuant to the safe harbor provisions of the federal securities laws. These forward-looking statements are based on current expectations and are subject to change. Actual results may differ materially from those expressed or implied in the forward-looking statements due to various factors, including, but not limited to, those outlined in our Form 10-K and periodic reports filed with the SEC. These documents are available on our website at www.abeonatherapeutics.com. Joining me today on today's call with prepared remarks are Dr. Vish Seshadri, Chief Executive Officer; Dr. Madhav Vasanthavada, Chief Commercial Officer; and Joe Vazzano, Chief Financial Officer. Also, Dr. Brian Kevany, Chief Technical Officer, will join us for the Q&A session. And with that, I will now turn the call over to Vish Seshadri to lead us off. Vish?

Thank you, Greg, and good morning. We have had a very exciting and momentous 2025 so far. We are incredibly proud that Abeona is now a commercial stage cell and gene therapy company following the U.S. FDA approval of our first product, ZEVASKYN, just a little over a couple of weeks ago. ZEVASKYN is the first and only autologous cell-based gene therapy for the treatment of adult and pediatric patients with recessive dystrophic epidermolysis bullosa, or RDEB. This is a persistent unmet need. To meaningfully heal RDEB wounds, especially those that are chronic and prone to infection through a single application, ZEVASKYN can provide the opportunity for significant closure and pain reduction in even the most severe RDEB wounds. The launch is underway, and we are quickly building positive momentum. Our marketing, medical affairs, market access, manufacturing and supply teams are all in place and engaging patients, physicians, payers and treatment centers. I want to touch upon some highlights on how the launch is going, keeping in mind that we're only about 2 weeks in, then Madhav will follow with details on the commercial progress in his remarks. We're pleased to announce that ahead of schedule, we have activated our first qualified treatment center or QTC and that ZEVASKYN is now commercially available. Lurie Children's Hospital of Chicago is our first QTC, enabling Lurie to begin identifying patients and our scheduling system is up and running. Lurie Children's is a top-ranked hospital and has been a center of excellence for genetic skin diseases for more than 30 years. As one of the largest North American centers for epidermolysis bullosa caring for more than 150 affected children and adults, Lurie Children's has been a member of the ED Clinical Research Consortium since its inception and continues to conduct cutting-edge bench and clinical research to better understand the disease and find new treatment options. Dr. Amy Paller, a world-renowned pediatric dermatologist is Head of the Epidermolysis Bullosa Research and Care Program at Lurie Children's and Chair of the Department of Dermatology at Northwestern University, Sainburg School of Medicine. And she was also the Chair of the Data Safety Monitoring Board for our pivotal VITAL study of ZEVASKYN. We look forward to a very collaborative relationship with Lurie Children's, which shares our conviction that ZEVASKYN is an important treatment option for people living with RDEB. We expect the first patient to be treated at Lurie in the third quarter of 2025. Last week, at the Society for Investigative Dermatology Annual Meeting, we presented 2 late-breaking abstracts that further underscore ZEVASKYN's efficacy and safety profile. One abstract details data showing the durable expression of functional collagen VII anchoring fibrils at ZEVASKYN-treated sites and long-term wound closure following a single treatment, both of which are hypothesized to be attributable to the presence and persistence of gene-modified progenitor cells within the ZEVASKYN gene-corrected cellular sheets. The second abstract confirmed the absence of squamous cell carcinoma or other cancer at any of the 144 ZEVASKYN-treated sites. In 7 instances where FCCs were observed in untreated areas, testing for proviral genome and replication competent retrovirus yielded negative results. These clinical results and nonclinical results of insertional oncogenesis that were reported at SID further support a favorable benefit risk profile for ZEVASKYN. Moving on, I want to briefly remind everyone that earlier this week, we announced that we entered into an agreement to sell for $155 million, the priority review voucher, or PRV, that we received with ZEVASKYN approval, subject to customary closing conditions, including antitrust review under the Hart-Scott-Rodino Act. The PRV proceeds will strengthen our balance sheet to fund our operations, as Joe will further elaborate in a bit. These efforts will be critical for both our near-term path to profitability and to drive our future growth. I'll now hand the call to our Chief Commercial Officer, Dr. Madhav Vasanthavada, to highlight our commercial launch preparations for ZEVASKYN. Madhav?

Thanks, Vich, and hello, everyone. It has been about 2 weeks since ZEVASKYN approval, and we are off to a very good start. The response from patients, caregivers and the physician community since gaining ZEVASKYN's FDA approval has been incredible. Already in just the last 2 weeks, our patient hub, Abeona Assist, has received phone calls and e-mail inquiries from about 30 patients and families across the country. We recognize that any new product approval comes with interest and excitement, but the volume and nature of these inbound inquiries are very encouraging. We are hearing from adult patients and parents of children who are all hopeful about the potential impact ZEVASKYN can have on them and their loved ones, and they want to know how soon they can access this treatment. These calls show the tremendously high unmet need and that patients are urgently seeking reliable and durable treatment options for their RDEB wounds. Many of the callers have told us that they are not satisfied with the currently available treatments, either because in their opinion, these treatments are not reliably healing their wounds or they are burdened by the continuous and potentially lifelong administration of the current options. ZEVASKYN is the only treatment proven to reliably close not just small wounds, but large wounded regions of the body with a single treatment application. With Lurie Children's activated as the first ZEVASKYN qualified treatment center, Dr. Amy Paller and her team at Lurie can now begin to schedule their identified patients for treatment with ZEVASKYN. Our teams will support Lurie as they identify eligible patients and secure the necessary payer authorizations, which we expect will take several weeks initially. We are currently anticipating the first patient to be biopsied in July 2025 and to receive this treatment in August 2025. In addition to Lurie, we are on track to activate other geographically dispersed QTCs by the end of 2025. Like Lurie, the other hospitals we are in discussions with are well-recognized institutions with cell and gene therapy infrastructure and experience in providing specialized care for EB patients. As we have previously mentioned, we expect to see a gradual ramp-up at QTCs with centers getting accustomed to ZEVASKYN treatment process and the patient journey through treatment of their first 1 or 2 patients. With this initial treatment experience, we then expect a greater uptake of ZEVASKYN at each QTC over time. With the already existing number of ZEVASKYN-eligible patients at these large QTCs and the strong inbound interest we have begun to see through Abeona Assist, we remain optimistic about our ability to achieve our 2025 goal of treating 10 to 14 patients as we previously guided and look forward to a robust start to 2026. Beyond the positive response from patients, we are equally excited to see the enthusiasm from the EB physician community on the news of ZEVASKYN approval. Last week, we had very successful physician engagements at the Society for Investigative Dermatology Annual Meeting. Our 2 late-breaking scientific abstracts that Vish mentioned were very well received. Also, Dr. Jean Gene Tang, one of the prominent EB experts and the lead investigator of our VITAL Phase III study gave an oral presentation on ZEVASKYN's clinical data to a highly engaged audience and generated interest. We also held an advisory Board meeting at the SID meeting with an interdisciplinary group of EB physicians from our potential QTCs. The group was excited about ZEVASKYN's approval and was eager to discuss the immediate next steps to putting their identified patients on ZEVASKYN treatment, and we had a productive discussion. Over the past 2 weeks, we have also made significant strides in ensuring that the value of ZEVASKYN is recognized by payers and that patients have timely and broad access to this therapy. We are actively progressing discussions around outcomes-based agreements, reinforcing our confidence in ZEVASKYN's real-world impact and supporting favorable access for patients. We are especially excited to announce that we have now executed outcomes-based agreements with 2 payer contracting organizations. The first with Anton Rx, a leading pharmacy solutions provider representing several regional commercial and managed Medicaid payers and the second with one of the largest medical connectives serving approximately 100 million commercially insured lives. These 2 agreements represent a significant milestone in our efforts to ensure ZEVASKYN reaches the patients who need it most. Additionally, we are continuing to actively engage with several well-recognized commercial payers and all state Medicaid programs to further expand ZEVASKYN access to eligible patients across the country, and we'll keep you updated on our progress. With that, I would like to hand the call over to our Chief Financial Officer, Joe Vazzano, to discuss our financial results. Joe?

Thanks, Madhav. I would like to remind everyone that you can find additional details on our financial results for the 3 months ended March 31, 2025, in our most recent Form 10-Q, which is available on our website. Starting with the financial resources on our balance sheet. We had cash, cash equivalents, short-term investments and restricted cash of $84.5 million as of March 31, 2025, before accounting for the proceeds pending the closing of the PRV sale.This compares to $98.1 million as of December 31, 2024. The proceeds from our PRV sale, once the transaction has closed, will fully fund our operations for over 2 years, extending our runway through the point of projected ZEVASKYN-driven profitability in early 2026. This robust financial footing achieved even before ZEVASKYN revenues have begun, eliminates the need to raise additional capital to reach this crucial commercial milestone. Research and development expenses were $9.9 million for the quarter ended March 31, 2025, compared to $7.2 million for the quarter ended March 31, 2024. The increase was primarily due to increased headcount related to scale-up of manufacturing capacity in preparation for the planned ZEVASKYN commercial launch and preclinical development work. Our spend on general and administrative activities was $9.8 million for the quarter ended March 31, 2025 compared to $7.1 million for the quarter ended March 31, 2024. The increase was primarily due to increased headcount associated with the planned launch of ZEVASKYN. Net loss was $12 million for the first quarter of 2025 or $0.24 loss per common share as compared to $31.6 million or $1.16 loss per common share for the first quarter of 2024. In terms of upcoming Investor Relations activities, we will be participating in the Jefferies Global Healthcare Conference in early June. And with that, I will open the call for Q&A. Operator, please open the Q&A session.

[Operator Instructions] Our first question is coming From Maury Raycroft with Jefferies.

This is Amin on for Maury. Two from us. First, congrats on the activation of the first site. Related to that, what exactly do you mean when you say the site is activated? Does it include that the staff and surgeons are all trained at this point? And then the second question is related to -- well, you mentioned about the number of RDEB patients at Laurie Hospital, but specifically, how many RDEB patients are eligible for pz-cel at this treatment center? And in general, how about the other treatment centers? How many RDEB patients do you think they are eligible at these centers as well for pz-cel?

Go ahead, Madhav.

Thank you. Yes. So Amin, to your first question, the definition of activation and whether these sites are trained, yes, they are right now trained and every -- all of the processes within these institutions have been taken care of. They are in a position to have their patients identified for ZEVASKYN and begin the process to get payer authorizations. And once we have the payer approval for these patients, they can already start to slot their patients for a manufacturing schedule. So they have the ability to go ahead with patient identification and scheduling manufacturing. With regards to the patients, with Amy Paller -- with Dr. Amy Paller and her institution, there are -- we don't know the exact number of eligible patients, but from our prior discussions, there are at least a couple of dozen of patients that Dr. Amy Paller and her institutions treat within their own institutions. But then also, there are other physicians from the local community that we are hearing are in touch with her and referring their patients over to Lurie Children's. So we do think that over time, these patient count is going to build and there are going to be more patients identified from this center.

Our next question is coming from Kristen Kluska with Cantor Fitzgerald.

This is Rick Miller on for Kristen. We'll start with one and then maybe a follow-up. So for the Lurie Children's Center, were there any specific factors that led [Audio Gap]

Beyond just the clinical manifestation of these patients, also the insurance and the type of insurance that these patients have, we think that commercially insured patients would have faster access just because of the way that the insurance companies work relative to the Medicaid plans, which may take a few more extra months to open up the access. And so based on that factor and as well as the patient preference and how soon they can come up for treatment, we'll be able to slot these patients in manufacturing schedule. But that also all works out fine in our particular case just because given our manufacturing and the supply ramp that we had described in the past, where it will give us a chance with July as the first potential treatment slot, we have sufficient headroom to be able to slot these patients depending on how their access also works out.

And maybe just one quick follow-up, if I may. Thanks for the color on the calls and e-mails that you said you're receiving from the Abeona Assist reachouts. So is there anything you're getting from these conversations about what the wound profile here is starting to look like? Are these mostly patients with these kind of larger chronic wounds, along with a mix of other wounds? Or is this sort of a different kind of distribution than what you kind of saw in the clinical experience?

It's hard to comment exactly their wound profile or characteristics because it's really our Abeona Assist team that is engaging with them. But the context we can provide is these are patients, what we know, they are -- they all have large areas of their body. Typically, based on the natural history study, average patients has at least 1/3 of their body that is wounded. And some of the patients have said they have waited their entire life for having reliable treatment options as well as we are seeing patients across all age ranges from -- both across pediatric patients as well as adult patients. We've got children of adult patients who are calling in to see how soon they can access as well as caregivers of younger patients. So it seems to be across all of the age spectrum and across all of the age range across all of the -- in different regions of the country. So it seems to be people with multiple level of wound burden as well.

And just to add to that also, Rick, it's also somewhat challenging for us to know on a patient-by-patient basis because what we get is a consolidated view or kind of an update because this is managed by a separate team. And so we don't have patient-by-patient health information, but we just get kind of insights on what is the overarching patient types that are generally calling in. So that's why it's hard to place too much detail on the wound type, chronicity location, those types of details yet.

Our next question is coming from Stephen Willey with Stifel.

Congrats on the progress. I was just wondering if you could maybe provide some color around what the prospective QTCs that you're looking at now have intimated about patient throughput, I guess, both in terms of what patient volumes might look like at launch and maybe where that metric might go once logistics are optimized? And do you expect to see much variance in this throughput capacity across the QTCs that you intend to bring online? And then I just have a quick follow-up.

Yes, Stephen, thanks for that question. In terms of the patient throughput early on at the time of launch, we do think that -- there are patients, as we mentioned also at the ad board that we did last week, where we had physicians from basically all of our target qualified treatment centers around the table, and they were discussing about their identified patients in their mind, they already have queued up. So we think that the way this would work is they will have the first few patients going. In their mind, they want to treat patients who have large contiguous areas of their body that's wounded, first. And of course, with every production, you're getting up to 12 sheets that can be manufactured, right? So it's not just 1 or 2 wounds, you can pretty much cover large areas of the body. So these physicians are thinking about that to tackle those patients first. And then they're already thinking about their -- then the next sort of tier of patients will be those who have discrete wounds in various anatomical regions. So in terms of throughput, to put some numbers, even if when we post to them at 2 patients a month -- 2 patients every month per site because that's going to -- with our 10 patients a month capacity and 5 treatment sites at steady state, that's easily doable for these physicians. They are not like thinking twice about that. Nobody had actually put any resistance to that. Now this is from their respective QTCs, right? We will, over time, begin to also talk to other centers of excellence and have those patients referred into these QTCs. So at these kinds of throughput, 2 patients a month per treatment center is doable. As part of our onboarding process, as we speak with certain QTCs, other institutions have, as their pro forma also have indicated that they would like to go to 4 patients a month just because they have enough throughput capacity at their centers and sufficient bed capacity and things of that nature. So we are -- the type of numbers that we are talking about here is very much doable. It's really going to be the first couple of patients that they will put through the treatment and then ramp it up in '26. So we'll see how this goes, but we are optimistic.

Okay. That's very helpful color. And then maybe just wondering how we should be thinking about the prioritization of the earlier-stage pipeline now that you have improved visibility into the balance sheet. I mean you obviously have a lot on your plate right now -- with ZEVASKYN right now. But just curious if the sense of urgency at all around the pipeline has kind of changed at all following the PRV sale.

Thanks for that question, Steve, as we've communicated earlier, we have 2 assets, ARVO, which is the big ophthalmology scientific congress that happened recently, we had some very interesting data from our RS1 program. We're all very excited about bringing this to clinic for -- in human studies second half of 2026. And that is still on track. Even though we haven't talked much about it, in the background, that work has been happening. We have clear instructions from our pre-IND meeting from the FDA on what's required to take these assets to clinical trials. So that at least one asset from our ophthalmology pipeline going into clinic in the second half of 2026 is very much on track. In terms of acceleration, I think there is -- things have to happen like, for example, the animal experiments are going on and that only happens at a certain pace. We have -- there's a follow-up required and all that is -- cannot be really sped up in any way. But this is a good natural cadence for our pipeline going into clinic because by then, we would have established our run rate for commercial performance as well. So it fits very nicely with our -- as we ramp up our ZEVASKYN commercial progress here. And of course, the other preclinical programs will follow on the heels of the first one, which is RS1. And what is very encouraging about these programs is with ophthalmology gene therapy, we're not -- the manufacturing and CMC is not as intense as it is for an autologous product. So this can -- and this is all managed through CDMOs. So it's not putting additional burden on our company per se and at manageable pace of spending. So that's really how we are looking at it. But from a developing data perspective, you're going to hear more and more in the upcoming scientific congresses as we generate these animal data. And what we see so far is very encouraging.

Our next question is coming from Ram Selvaraju with H.C. Wainwright.

So I just wanted to see if you could provide us with some additional granularity regarding how you expect to scale up commercial supply as and when demand progresses to that point and the degree to which you have sufficient commercial bandwidth now to meet the demand of X number of patients. Also, this question is for Joe. Can you just clarify for us what you expect the net proceeds to Abeona to be from the sale of the PRV? And if you can give us a sense of what you anticipate pro forma cash to be at the end of the second quarter?

Thank you, Ram. Maybe, Joe, you can take that question first, and then we'll move to Brian for the manufacturing scale up.

Sure. Yes. So the net proceeds from the PRV sale, once it closes the HSR will be about $152 million or so. And so I mean it's tough to project ending cash as of June. Not sure if we will clear that time period by then, but we can expect about $152 million net proceeds from the PRV. We don't owe anything -- any liabilities on that PRV. It's all ours.

Brian, the other question was...

Yes, about the manufacturing question. So yes, as we've previously communicated, we are at a capacity of roughly 4 patients per month at launch, which is where we are right now. As the year continues and we onboard additional QTCs, we will be ramping to 6 patients per month with an anticipation of being at 8 and 10 patients per month by early next year. For a little bit of context, we have -- within our overall facility, we have 2 independent facilities that supply both. One supplies the drug product, the other supplies the vector that's used within the drug product manufacturing. As we ramp to 10 patients a month, we will be converting the vector side of the facility to also be supplying ZEVASKYN for commercial manufacturing to allow us to get to that 10 patients per month. But we believe that, that will scale well with the onboarding of the QTCs and the anticipated ramp in patient availability.

And just to clarify -- sorry, I just wanted to clarify, the 10 patients per month run rate can readily be addressed with your existing facilities bandwidth, you would not need to expand the facility footprint in order to meet 10 patients per month demand level. Is that correct?

Correct. Correct. Yes, the current facility will support that 10 patients per month.

And just to give a little bit more context, Ram, what we talked about in terms of expansion is actually beyond the 10 patients a month for which additional GMP space is being designed. The design is almost complete now, and we're commissioning -- starting of building out that GMP space, which can add roughly about the same number of slots per month. The exact number of slots we just need to finalize and we'll communicate soon. But that part of the facility will be ready to -- for slots for commercial manufacturing somewhere in the second half of 2027.

And this would also be based in Cleveland. Is that correct?

Yes. Cleveland, same building, the same floor.

Okay. And then just very quickly for Madhav. I was wondering if at this point, you have some sense of the prior treatment history of certain RDEB patients who might be considered candidates for pz-cel. And if you are seeing at this juncture specific possibilities of treating patients with pz-cel who previously had been treated with VYJUVEK or even with Filsuvez and how that situation seems to be shaping up and how you expect it to evolve going forward, just in terms of prior treatment history with other approved RDEB products?

Right. We think that at this point in time, majority will be previously exposed to prior treatments, both VYJUVEK as well as Filsuvez. And that's what we hear also not just from patients, but also from the physicians. And they're all pretty comfortable with that discussion also. In fact, that was also a point of our conversation. So over time, we -- with that said, there are patients that we hear who have not gotten any of the previous treatment, right? But that's relatively -- we don't know what percent, but it's minority is what we would think. Let's say, over time, we do expect -- we expect that pretty much to be the case because these patients will require as many treatment options that they can get just because of their wound modalities and such. So we'll continue to track that as we get into real treatments.

Our next question is coming from James Molloy with Alliance Global Partners.

Congrats on the prior approval as well. Any word on who you guys -- the Hart-Scott, the provision you're waiting for that. Any word on who will you sell it to? Is that something you won't be able to release? And then Joe had mentioned on the PRV, that profitable by second half '26. Is that EPS profitable, cash profitable?

Yes. So in regards to the buyer of the PRV, that's undisclosed. And yes, for 2026, yes, I'm anticipating positive EPS.

And then last question is, has the -- with the acquisition and potentially having $220 million or so on the books, does that increase any inbound calls for potential acquisitions?

Jim, I think it's premature to address that question. But when we have any updates, we will definitely bring that out to share. But at this time, we don't have any updates on that front.

[Operator Instructions] Our next question is coming from David Boutz with Zacks Small-Cap Research.

Congrats on the progress. I was wondering if you could just kind of remind us of how billing and reimbursement is going to work. For example, you've got a patient who is expected to be biopsied in July and treated in August. I'm just curious how long do you anticipate the time frame being from when that patient is treated or biopsied to when Abeona gets payment for that therapy? And is that going to be different for all the different treatment centers? Or will that kind of be uniform?

Madhav?

Yes. So -- thanks, Vish. So on the topic of payment, I mean, revenue recognition itself will happen right after the patient is treated, David. And so that's sort of as far as recognizing the revenue. In terms of the payment and the cash itself, that will really depend. We are still in discussions with the qualified treatment centers on that topic. But it will be on par with any other cell and gene therapy in terms of the payables. But these are large institutions who have experience doing other cell and gene therapy. So I mean, I think from that standpoint, it's looking like it will track along the same lines. But before any patient is placed on treatment, at least from the provider standpoint or the hospital standpoint, they will be prenegotiated or there will be discussions already with the insurance companies. So it's not like they're up for a surprise that they have treated the patient and they don't know whether they're going to get reimbursed or not on the back end. So we do anticipate a very high level of certainty that if a patient is treated and surgically treated then that's going to result in revenue recognition and cash incoming.

David, just to add to that, right, this explains why there is a little bit of a lead time even between approval and treating our first patients because even though we have a site activated now, and while the policies are being put in place by the various commercial insurers, in the meanwhile, these sites will be working on a case-by-case basis to make sure that reimbursement is kind of dealt with upfront before they take up a manufacturing slot because they don't want that risk on them, right? So that explains why there is a little bit of a lead time even to get the train started.

Right. Okay. Understood. So for the approximately 30 patients that you indicated you've received inbound interest on, are all these patients looking to go to Children's Hospital in Chicago? Are they just trying to get their name in the queue? Like what are they interested in doing there?

Yes. It's more of the latter at this point in time. These are -- we just wanted to give sort of an idea of the number of patients who have already called in, in the last 2 weeks. And at that point, we didn't have a qualified treatment center announced. So it's more about the queuing up of these patients. And now that we have announced Lurie, we will certainly let these patients know. The next step would be for these patients then to enroll in our Abeona Assist program so that they can continue to get more information and updates and/or directly reach out to Dr. Palmer and her staff. So that is certainly another option, plus also other patients who haven't called Abeona Assist also now are aware. So even if we think a fraction of these 30 patients do sign up and enroll in our program, we've got sufficient initial inbound interest.

As we have no further questions on the lines, I'd like to hand it back to Mr. Vish Seshadri for any closing remarks.

Thank you, Ali, and thank you, everyone, for joining us for today's business update. As you can see, a lot of progress has been made very quickly this year in 2025, and we look forward to bringing you more updates on the commercial launch progress very soon. So have a wonderful day, and we'll talk soon. Bye-bye.

Thank you. Ladies and gentlemen, this does conclude today's call. You may disconnect your lines at this time, and we thank you for your participation.