Abeona Therapeutics Inc (ABEO) 2025 Q3 法說會逐字稿

Good morning, everyone, and welcome to the Abeona Therapeutics Third Quarter 2025 Conference Call.

At this time, all participants are in a listen-only mode, and the floor will be open for questions following the presentation.

If anyone should require operator assistance during this conference. (Operator Instructions)

I will now turn the conference over to your host, Gregory Gin, VP of Investor Relations and Corporate Communications at Abeona. Gregg, 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 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 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 Vishwas 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 in 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, 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 three. Furthermore, we've 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 launch 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. 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 trials and was an FDA requirement that was added during the BLA review. Retesting using established gold standard USP sterility methods confirmed the sterility of the product, but unfortunately, those test results were not available until after the lot's 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 were identified at the first two qualified treatment centers. Of these patients, we have already received ZEVASKYN Product Order Forms, or ZPOFs, for 12 patients. A ZPOF is an informed consent generated by the QTC physician after the patient has been consulted, a treatment decision has been made, and the patient 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+ 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 three, alongside Lurie Children's Hospital of Chicago and Lucile Packard Children's Hospital Stanford. We are also in active discussions with several EB centers across the U.S. to strategically expand the geographic footprint of ZEVASKYN even further. These centers are advancing through the various stages of site onboarding, and we 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 six 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 and 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 healthcare 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. 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 three and nine months ended September 30, 2025, 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 current and planned operations for over two years.

Turning to the statements of operations, research and development, or R&D spending for the three 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 expenses were $19.3 million for the three 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 -$0.10 per basic and diluted common share, compared to a net loss of $30.3 million in the third quarter of 2024, or -$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 two key updates.

First, our gene therapy program for X-linked retinoschisis, ABO-503, has been selected to participate in the FDA Rare Disease Endpoint Advancement, or 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. Gao as the Senior Vice President, Head of Clinical Development and Medical Affairs. Dr. Gao 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.

Thank you very much. At this time, we will be conducting our question-and-answer session. If you would like to ask a question (Operator Instructions)

Thank you. Our first question is coming from Maury Raycroft, Jefferies. Maury, your line is live.

Hi. Thank you for taking our questions. 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 timeline for these patients to receive treatment at this point? I have a follow-up.

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

Thanks, Amin, for the question. These patients, as I mentioned, the product order form is the first step. 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 the early part of 2026. We expect that if all paperwork goes through, administrative process in the coming months to treat these patients.

Helpful. Thanks.

So, Thanks, Madhav. The only point I want to add there, Amin, is that, as Madhav mentioned, these 12 patients are at various points in their journey. To generalize how much time it'll 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. What we believe is, as we start to treat patients, this is going to normalize. Metrics in terms of time taken from a ZPOF to various time points in the journey, we'll have a better idea having been through that process for a bunch of patients, which we should have in the first quarter of 2026.

Okay. Thanks. Of the 12 ZEVASKYN Product Order Forms, how many are from patients who referred to QTCs versus patients already being treated in these sites? What's your timeline estimate for achieving profitability at this point? Is that bumped by a quarter based on the current delay?

Yeah. On the topic of the first question. Yeah. For the first question, the vast majority are at the QTCs, Amin. There are patient referrals that already have been initiated, and those patients will go through the counsel process as well but for the 12 patients that we've talked about, the vast majority are the patients at the QTCs.

Yeah. Also, I mean to your second part question, which is, how does it impact the time to profitability? We do not see a significant impact. I think in the past, we have guided that in the first half of 2026, we should be a profitable business, and that continues to be our projection. We do not see the first patient treatment shifting to quarter four significantly impacting that timeframe.

Thanks.

Okay, thank you very much. Our next question is coming from KristenKlitska of Cantor Fitzgerald. Kristen, your line is live.

Hi. This is Rick on for Kristen. Thanks for taking our questions. To start out, are you still planning on shutting the plant down in December for the routine maintenance? If so, what's the timeline around reopening there?

Yeah. No, thanks for that question. 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 could add some color if I missed the timing or anything else to add there.

No, that's accurate. Yeah. 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. Yeah, mid-December to early January is the current schedule for the shutdown.

Okay. 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? If so, will you be able to just sort of move into manufacturing with these, or will you need to re-biopsy any patients?

Yeah. 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. Especially until we solved this problem, we did not know what exactly the problem was, how long it would take for us to resolve it. We did not take any chances there.

Okay, thank you.

Thank you very much. Our next question is coming from Ram-- Oop, apologies. Our next question's coming from Steven Willie of Steel. Stephen, your line is live.

Hey, good morning. Thanks for taking our question. 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? Do you anticipate this to maybe come down over time as patient demand continues to increase?

Yeah. I can take that, Josh. Yes, we do expect this will 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're already scheduled for biopsy collection starting next year, and we have resumed biopsies already. 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 a three-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. As more and more patients go through the queue, we should expect that process to come down.

Yeah. 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. That is 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 do not need exceptions for the patients.

I'll just say that this is nothing new about ZEVASKYN. I mean, right? I mean, any cell and gene therapy that has launched goes through these kinds of processes. The centers that we are working with are super experienced about working with cell and gene therapies. We've got a good team in place. We've got a good market access team on our side in place. 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. As more and more patients go through for a given payer, that time for agreements will also, we expect that to come down.

Okay, I appreciate the color. Thank you.

Thank you very much. Our next question is coming from Ram Salvaraju of HC Wainwright. Ram, your line is live.

Thanks so much for taking my questions. 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. Insurance clearances and those processes, we are working through those. 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. Our success rate has been, from clinical trials perspective, 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 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. 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.

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. If you add that, the last call we had mentioned close to 50. That number has gone way north, and we are not even talking about that right now. 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. These first 12 patients are earmarked as the highest priority. We do not anticipate attrition because for them, it's not easy to get the slot either. This is going to be just a matter of conversion.

Okay. That's very helpful. 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? 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?

Thank you.

Yeah. The prior auth process, Ram, essentially, I can break it into two 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 types of therapies, tend to follow the inclusion/exclusion criteria of the clinical trials. There are some payers who also cover it to the label, right? In terms of the requirements, it is 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, six years and above, but our FDA label is broader right from birth. Certain payers have those policies in place. When you do have a payer that, let's say, it's a five-year-old patient that requires the 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 are successfully overturned those initial PA denials that typically happen. That's the process that takes place. Once you have that clearance, 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.

Thank you.

Thank you very much. Just a reminder there, if you have any remaining questions (Operator Instructions)

Our next question is coming from Jeff Jones, Oppenheimer. Jeff, your line is live.

Good morning, guys, and thanks for taking the questions. 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, you want me to take that.

Sure. Yeah. 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, hasn't settled down. It is 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. 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. For reporting purposes, the revenue is recognized the day of administration.

Great. Appreciate it, guys. Thank you.

Thank you very much. Our next question's coming from James Malloy of Alliance Global Partners. James, your line is live.

Hi, guys. Thank you for taking our questions. Matt on for James 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. We would never have visibility into that, but we expect the 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. 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. We don't know the actual exact count.

Got it thank you guys for taking our question.

Thank you very much. And our next question is coming from David Bouts of Zach's Small Cap Research. David, your line is life.

Hey, good morning, everyone. Just one for me this morning. UMass was a center in the phase three study and, of course, noticed 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 driving signed on yet.

Yeah. I can take that question. Good morning, David. Different sites have different reasons. I do not 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 are willing to accept may not be fitting within their framework, right? There are multiple different reasons why a given site may not be onboarded yet with us as a ZEVASKYN site. We are not going to be discussing specific sites' hurdle on why they have not been activated despite the fact that they have experience with handling ZEVASKYN. I hope that gives some color to the types of reasons.

Yeah. Actually, that's great. I appreciate it.

Thank you very much. Well, we appear to have reached the end of our question-and-answer session, so I would now like to turn the call back over to Vish for closing comments.

Thank you very much, Jenny. I really appreciate everyone joining us today for the call. 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.