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

Good morning, everyone, and welcome to the Abeona Therapeutics Third Quarter 2025 Conference Call. [Operator Instructions] Please note, this conference is being recorded. I will now turn the conference over to your host, Gregory Gin, VP of Investor Relations & Corporate Communications at Abeona. Greg, the floor is yours.

Thank you, Jenny. Good morning, and thank you for joining us on our third quarter 2025 results conference call. During this call, we will refer to the press release issued this morning announcing the financial results, which is available on our core 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 Securities and Exchange Commission. These documents are available on our website at www.abeonatherapeutics.com. Now joining me today with prepared remarks are Dr. Vish Seshadri, Chief Executive Officer; Dr. Brian Kevany, Chief Technical Officer; Dr. Madhav Vasanthavada, Chief Commercial Officer; and Joe Vazzano, Chief Financial Officer. After the prepared remarks, we will conduct a Q&A session. With that, I will now turn the call over to Vish Seshadri to lead us off. Vish?

Thank you, Greg. The third quarter of 2025 was marked by significant operational progress as we continue to scale the ZEVASKYN commercial launch to meet growing patient demand while our first patient treated has shifted to the fourth quarter of 2025 due to optimization of a product release assay, our conviction and our ability to achieve our 2026 launch goals remains steadfast based on trends in patient demand, treatment center expansion and market access. We're seeing growing patient demand for ZEVASKYN skin, the first and only autologous cell-based gene therapy for the treatment of adult and pediatric patients with recessive dystrophic EB or RDEB. We also continue to strategically expand our Qualified Treatment Center, or QTC network. The activation of a highly recognized EB Center, Children's Hospital Colorado brings our total activated centers to 3. Furthermore, we have established a strong foundation with broad market access, which is essential for sustained commercial success. In summary, despite the temporary delay in the first patient treatment, we are well positioned for long success in 2026. Before we dive deeper into our commercial launch progress and momentum, I now hand the call to our Chief Technical and Scientific Officer, Dr. Brian Kevany to briefly highlight the release assay optimization. Brian?

Thanks, Vish, and hello, everyone. As we continue the ZEVASKYN launch, we remain dedicated to maintaining the highest standards of quality in the manufacturing of personalized drug products for each patient. During the third quarter, a full batch of drug product was manufactured following a patient biopsy but could not be released due to a performance issue in one of our release assays. Specifically, a rapid sterility assay delivered false positive results, which required us to reject the lot. The rapid sterility assay was not part of our clinical trial and was an FDA requirement that was added during the BLA review. Retesting using established gold standard USP sterility methods confirm the sterility of the product, but unfortunately, those test results were not available until after the last expiration date, so they could not be used to release the lot. As a proactive measure to ensure product quality, we temporarily paused collecting additional patient biopsies so that we could conduct a thorough investigation, run additional tests and further optimize the new release assay. Following successful completion of optimization, validation and the necessary regulatory submissions, we resumed biopsy collection in November 2025. We now anticipate patient treatment starting in the fourth quarter of 2025. I will now hand the call over to Chief Commercial Officer, Madhav Vasanthavada to discuss our commercial launch progress. Madhav?

Thanks, Brian. Hello, everyone. Our launch momentum continues to accelerate on multiple fronts. Patient demand continues to build. Our relationships and trust with qualified treatment centers have grown stronger and patient access to ZEVASKYN across all payer types has continued to broaden. On our second quarter call, we mentioned more than a dozen initial patients who were identified at the first 2 qualified treatment centers. Of these patients, we have already received ZEVASKYN product order forms or ZPOFs for 12 patients. ZPOF is an informed consent generated by the QTC physician after the patient has been consulted, a treatment decision has been made and the patients and their families have decided to move forward. Insurance prior authorizations have been obtained for several patients already, and we expect these patients to be biopsied over the coming months as and when full financial clearance is in place. We are happy to also report that demand for ZEVASKYN continues to grow. The number of identified eligible patients at our QTCs who are motivated to initiate the treatment process has now more than doubled to approximately 30 patients, up from the 12-plus mentioned on the second quarter call. At the same time, the broader pool of potential ZEVASKYN candidates at non-QTC referral sites continues to increase as our field force and promotional activities generate more ZEVASKYN awareness in the marketplace, and many of these referral sites have initiated patient referrals to the qualified treatment centers, which is exactly what we were hoping for. Regarding QTC activations, we are delighted that Children's Hospital Colorado is our newest ZEVASKYN qualified treatment center. Children's Hospital Colorado has an expert multidisciplinary team with years of EB experience and their commitment to onboarding ZEVASKYN speaks to their belief in the benefits this therapy can bring to RDEB patients. Activation of Children's Colorado brings the number of ZEVASKYN qualified treatment centers to 3 alongside Lurie Children's Hospital of Chicago and Lucile Packard Children's Hospital in Stanford. We are also in active discussions with several EB centers across the U.S. to strategically expand geographic footprint of ZEVASKYN even further. These centers are advancing through the various stages of site onboarding and will continue to announce new centers as they are activated. Finally, regarding market access, we have seen a steady cadence of positive coverage decisions from both national and regional commercial health plans in the 6 months since approval. Importantly, policies covering ZEVASKYN have been published by all major commercial payers, including UnitedHealthcare, Cigna, Aetna, Anthem and the majority of Blue Cross Blue Shield plans, all collectively covering more than 80% of all commercially insured lives. Now on the government payer front for Medicaid, we are happy to report that ZEVASKYN now has received baseline coverage across all 51 state Medicaid programs in Puerto Rico effective October 1, 2025. Moreover, multiple state Medicaid programs have already published policies covering ZEVASKYN signaling that payers recognize the value ZEVASKYN brings to their patients and the health care system. As another major highlight, CMS has established a permanent product J-code for ZEVASKYN that will go into effect on January 1, 2026. We believe that this product code will simplify claims and reimbursement processing between our QTCs and all payer types and will further support hospital adoption for ZEVASKYN. In summary, we are very encouraged by the growing patient demand, patients actively progressing toward treatment, continued growth of the QTC site network and a favorable market access landscape for ZEVASKYN, and we are looking forward to a strong start in 2026. With that, I'll now pass 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 could find additional details on our financial results for the 3 and 9 months ended September 30, 2025, and in our most recent Form 10-Q. Starting with our financial resources. We had cash, cash equivalents, restricted cash and short-term investments totaling $207.5 million as of September 30, 2025. This robust cash position provides us with significant financial flexibility as we execute on the ZEVASKYN commercial launch. The current cash position without accounting for anticipated revenue from ZEVASKYN is expected to be sufficient to fund the current and planned operations for over 2 years. Turning to the statements of operations. Research and development, or R&D spending for the 3 months ended September 30, 2025, was $4.2 million compared to $8.9 million for the same period of 2024. This reduction was primarily due to costs capitalized into inventory and the reclassification of selected costs such as engineering runs and other production costs to selling, general and administrative expense, or SG&A, following ZEVASKYN's FDA approval. SG&A expense were $19.3 million for the 3 months ended September 30, 2025, compared to $6.4 million for the same period of 2024. This increase reflects the reclassification of R&D expenses as noted, along with increased headcount and professional costs associated with the commercial launch of ZEVASKYN. Our net loss was $5.2 million for the third quarter of 2025 or negative $0.10 per basic and diluted common share compared to a net loss of $30.3 million in the third quarter of 2024 or negative $0.63 per basic and diluted common share. In terms of upcoming Investor Relations activities, we plan to participate in the Stifel 2025 Healthcare Conference tomorrow. With that, I'll pass the call back to Vish for additional remarks before opening the call for Q&A.

Thank you, Joe. Turning briefly to our pipeline, we have 2 key updates. First, our gene therapy program for X-linked retinoschisis, AB0503, has been selected to participate in the FDA Rare Disease Endpoint Advancement, RDEA, pilot program. This selection will provide opportunities for enhanced communication with the FDA to accelerate the development and validation of product-specific novel efficacy endpoints for the program. Second, we have strengthened our management team with the appointment of Dr. James A. Gow as the Senior Vice President, Head of Clinical Development and Medical Affairs. Dr. Gow brings over 20 years of industry experience and is a recognized expert in gene therapy, especially in ophthalmology, which will be valuable as we advance our pipeline. In closing, while the first patient treatment has shifted to the fourth quarter of 2025, we are encouraged by the doubling of identified patients, 10 product order forms -- 12 product order forms, the expansion to a third QTC and the broad and rapid payer coverage across commercial and government plans. This progress underscores the high value proposition of ZEVASKYN for the RDEB community. With that, I will now open the call for Q&A. Jenny, please open the Q&A session.

[Operator Instructions] Our first question is coming from Maurice Raycroft of Jefferies.

This is Amin on for Maury. A couple of questions from us. You mentioned receiving ZEVASKYN product order forms for 12 patients, what's the expected time line for these patients to receive treatment at this point? And I have a follow-up.

Yes. Thank you for that question, Amin. I'll request Madhav to take that one.

Thanks, Amin, for the question. So these patients, as I mentioned, the product order form is the first step. And after that, there are insurance discussions that have been ongoing between the qualified centers and the payers. For many of these patients already we have a prior auth. Some of them have already been scheduled for biopsy in November this year as well as in early part of 2026. So we expect that if all paperwork goes through administrative process in the coming months to treat these patients.

Thanks, Madhav. The only point I wanted to add there, I mean, is that as Madhav mentioned, these 12 patients are at various points in their journey to generalize how much time it will take for these 12 patients to come all the way through the funnel into a treatment, it's a hard thing to do at this point in time. But what we believe is as we start to treat patients, this is going to normalize. So metrics in terms of time taken from a ZPOF to various time points in the journey, we'll have better idea having been through that process for a bunch of patients, which we should have in the first quarter of 2026.

Okay. And of the 12 ZEVASKYN product order forms, how many are from patients who refer to QTCs versus patients already being treated in these sites? And what's your time line estimate for achieving profitability at this point? Is that bumped by quarter based on the current delay?

Yes. So the first question, the vast majority are at the QTCs, Amin, and there are patient referrals that already have been initiated, and those patients will go through the consult process as well. But for the 12 patients that we've talked about, the vast majority are the patients at the QTCs.

Yes. And also, Amin, to your second part question, which is how -- does it impact the time to profitability? We don't see a significant impact. I think in the past, we've guided that in the first half of 2026, we should be a profitable business, and that continues to be our projection. So we do not see the first patient treatment shifting to quarter 4 significantly impacting that time frame.

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

This is Rick on for Kristen. To start out, are you still planning on shutting the plant down in December for the routine maintenance? And if so, what's the time line around reopening there?

Yes, thanks for that question, and it's a great question. Yes, we do have a shutdown, which starts approximately second week or mid-December and takes about a month. Brian, you can add some color, if I missed the timing or anything else to add there?

No, Vish, that's accurate, yes. And this is really a mandated FDA requirement to have this type of shutdown at the end of the year for general maintenance and recalibrations of equipment. But yes, mid-December to early January is the current schedule for the shutdown.

Okay. And on the temporary pause while you were working on the optimization, were there any biopsies that were collected but not yet sent to manufacturing before the temporary pause and reoptimization? And if so, will you be able to just sort of move into manufacturing with these? Or will you need to rebiopsy any patients?

Yes. The answer is no. We paused on collecting any further biopsies when this happened, not from any regulatory action or anything, but our own abundance of caution to avoid patients giving their biopsies. And especially until we solved this problem we were not -- we didn't know what exactly the problem was, how long it will take for us to resolve it. So we didn't take any chances there.

Our next question is coming from Ram -- apologies. Our next question is coming from Stephen Willey of Stifel.

This is Josh on for Steve. Is there maybe any color you can share related to the current lead time between receiving these ZEVASKYN product order forms following initial patient identification efforts? And do you anticipate this to maybe come down over time as patient demand continues to increase?

Yes. I can take that, Josh. So yes, we do expect this will reduce, decrease over a period of time. Like I mentioned earlier, some of these patients, even though the ZPOFs we received a couple of months ago, they are already scheduled for biopsy collection starting next year, and we have resumed biopsies already. So as more and more patients come through and the processes at the qualified centers and the payer policies with payer policies coming through nicely, we expect this overall time to reduce. At this point, when we mentioned on our second quarter call, it's about 3-month process is what it takes from the time that you have a patient identified, consulted, prior auth from a clinical standpoint and any agreements that take place. And as more and more patients go through the queue, we should expect that process to come down, and we'll guide more in terms of what we are seeing over a period of next quarter or so.

Yes. And the only other thing I would add there, Josh, is that the very first few patients at the time when their prior auth and letters of agreements were going through, the policies were not published by some of these payers. They have come in more recently. So that's the basis why we believe that for the future patients coming through the funnel, that time should reduce because the policy is already in place, and we don't need exceptions for the patients.

I'll just say that this is not new about ZEVASKYN. I mean, right -- I mean, any cell and gene therapy that has launched goes through these kinds of process and the centers that we are working with are super experienced about working with cell and gene therapies. So we've got a good team in place, we've got a good market access team on our side in place and the receptivity that we're getting from insurance companies and the willingness for the payers to work with centers to expedite this process is there. So as more and more patients go through for a given payer, that time for agreements will also -- we expect that to come down.

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

Firstly, I was wondering if you could give us some additional granularity on what you expect the attrition rate, if any, to be among those patients for whom ZPOFs have been received before you go through the entire biopsy, cell graft engineering and subsequently, administration of the graft? Just give us a sense of -- of those initial 12 patients, just taking that number as an example, how many do you anticipate are going to go successfully through the entire treatment process?

I would say it's a pretty high level of conversion, Ram, for these patients because these are the patients that the physicians -- obviously, these are motivated patients, they want to move forward, which is why we have the ZPOF already come through and insurance clearances and those processes are -- we are working through those. So we expect -- now with this release assay optimization also behind us, we expect as biopsies come through to be able to treat these patients. Of course, these are engineered cell therapies, right, that we are talking about. But our success rate has been -- from a clinical trials perspective has been pretty high. So for those reasons, we expect that these patients -- pretty high level of conversion rate now that we have these ZPOFs already in place for these patients. And the fact that when we mentioned a little over a dozen patients identified at these QTCs, for us to get 12 ZPOFs already shows that none of the patients that were identified, almost none of the patients that were identified early on actually said that, no, I'm not interested in getting ZPOF. I think that, to me, is a pretty strong metric. And as more patients get into this funnel and as patients get treated, you can already see the word of mouth and the data percolating, which only motivates additional patients to come through the process.

And I just wanted to add one more color to that, Ram. If you look at the number of patients that have been identified just organically within the QTCs that Madhav mentioned has more than doubled to about 30 patients or so, we're not even adding the referred patients. So if you add that, the last call, we had mentioned close to 50, and that number has gone way north, and we are not even talking about that right now. And in some ways, from that big pool of identified patients, the QTCs are acting as kind of gatekeepers in giving us the ZPOFs because they also want to regulate how much they can treat. And these -- the first 12 patients are earmarked as the highest priority. And so we do not anticipate attrition because it's -- for them, it's not easy to get the slot either. So this is going to be just a matter of conversion.

Okay. That's very helpful. And then with respect to the prior authorization process or prior authorization protocol that you are seeing with respect to payers, can you maybe describe for us what that looks like? And I'm in particular interested in situations involving RDEB patients with large chronic open wounds that have persisted for an extended period of time. What is the prior authorization requirement, if any, specifically in those types of patients that's being mandated by payers at this time?

Yes. So the prior auth process, Ram, essentially, it's -- I can break it into 2 steps. One is a clinical prior authorization and then the other is the financial discussion that takes place after a patient is clinically given a green signal from the insurance company. The prior auth, most payers, especially for these type of therapies tend to follow the inclusion/exclusion criteria of the clinical trials. And then there are some payers who also cover it to the label, right? So in terms of the requirements, it's often pretty straightforward, make sure that the patient has recessive dystrophic EB, which means the genetic testing or confirmation of the mutation of the collagen 7A1 gene that they have recessive wounds. In our clinical trial, there were certain wound size requirements, but we are seeing most of the insurance companies now, for example, UnitedHealthcare covering to label, meaning no real requirement on the size of the wound for these patients. Some payers have age 6 years and above, but our FDA label is broader, right, from birth. So certain payers have those policies in place. But when you do have a payer that -- let's say, it's a 5-year-old patient that requires a treatment, we are seeing that with the letter of medical necessity, you're able to overturn that because you're able to explain as to what the impact ZEVASKYN is bringing, and we successfully overturned those initial PA denials that typically happen. So that's the process that take place. And once you have that clearance, then the next step is the financial agreement between the payer and the qualified center.

And no meaningful step edits, correct?

No, we have not seen any step edits.

[Operator Instructions] Our next question is from Jeff Jones of Oppenheimer.

Can you comment on of the 12 patients in process how many have had their biopsies done today? And have any of those doses passed the revised sterility release criteria or with the new assay rather?

Vish, do you want me to take that?

Sure. Yes. I think as of this point, we announced that we have resumed biopsy, right? We have biopsied a patient. We're not guiding how many are going to be biopsied within the time window for this year versus how many will spill over because that's an ongoing process, it hasn't settled down. So it's too early. We're going to be talking about that in our next quarterly call.

Okay. Can you remind us then in terms of revenue recognition from the time that you dose these patients how long until revenue recognition?

The revenue is recognized when the product is applied on the patient from an accounting standpoint. And obviously, the cash flow has days to accounts payable that's involved. That's different for different -- it's governed more by trade policies with each site. But for reporting purposes, the revenue is recognized at the day of administration.

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

Matt on for Jim today. Just one from us. Of the 30 patients that are slated to receive ZEVASKYN, how many are on background VYJUVEK currently or have failed VYJUVEK?

We don't have visibility into that, Matt. I don't -- we would never have visibility into that, but we expect vast majority would be on VYJUVEK and/or on FILSUVEZ because that's what we hear from the patient community that these patients require -- there's an unmet need for multiple treatment options. We hear that from patients as well as from physicians across the board. So we would expect those patients. And from an access standpoint, that only helps us, right, because these patients have their copies of genetic records and other letters and background already in place so that physicians can provide that information to the payer because they have received prior other gene therapies. But we don't know the actual -- exact count.

And our next question is coming from David Bautz of Zacks Small-Cap Research.

Just one from me this morning. So UMass was a center in the Phase III study and of course, notice that they are not signed up as a QTC yet. I'm curious if there's any hold up there or why they are not listed as a QTC or haven't signed on yet?

Yes, I can take that question. David. Certain different sites have different reasons. I don't want to call out specific reasons, specific sites. Sometimes sites would intend to onboard and activate with us, but there could be financial constraints on how the site is faring. Sometimes the types of trade policies that we're willing to accept may not be fitting within their framework, right? So there's multiple different reasons why a given site may not be onboarded yet with us as a ZEVASKYN site. But we're not going to be discussing specific sites hurdle on why they haven't been activated despite the fact that they've got experience with handling ZEVASKYN. I hope that gives some color to the types of reasons.

Well, we appear to have reached the end of our question-and-answer session. So I will now like to turn the call back over to Vish for closing comments.

Thank you very much, Jenny, and I really appreciate everyone joining us today for the call, and we look forward to talking to you soon. Bye-bye.

Thank you very much. This does conclude today's conference call. You may disconnect your phone lines at this time, and have a wonderful day. We thank you for your participation. Goodbye.