ABEO - NASDAQ

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. The floor will be opened for questions following the presentation. If anyone should require operator assistance during this conference, please press 0 on your phone keypad. Please note this conference is being recorded. I will now turn the conference over to your host, Gregory Gin, VP of Investor Relations and Corporate Communications at Abeona Therapeutics. 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 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. 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-Ks 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. Vishwas Seshadri, Chief Executive Officer; Dr. Brian Kevany, Chief Technical Officer; Dr. Madhav Vasanthavada, Chief Commercial Officer; and Joseph Walter 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.

Thank you, Greg. The 2025 was marked by significant operational progress as we continue to scale the

Thanks, Vish. And hello, everyone. As we continue the

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

Thanks, Madhav. I would like to remind everyone you could find additional details on our financial results for the three and nine months ended 09/30/2025, 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 09/30/2025. This robust cash position provides us with significant financial flexibility as we execute on the

Thank you, Joe. Turning briefly to our pipeline, we have two key updates. First, our gene therapy program for X-linked retinoschisis, ABO503, 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. Gao as the Senior Vice President, Head of Clinical Development and Medical Affairs. Dr. Gao brings over twenty 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 2025, we are encouraged by the doubling of identified patients from 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

Thank you very much. At this time, we will be conducting our question and answer session. If you would like to ask a question, please press star 1 on your phone keypad. A confirmation tone will indicate that your line is in the queue. You may press star 2 if you would like to remove your question from the queue. For anyone using speaker equipment, it might be necessary to pick up your handset before you press the key. Please wait a moment whilst we poll for questions.

Thank you.

Our first question is coming from Maury Raycroft of 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

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

Hey. 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 authorization. Some of them have already been scheduled for biopsy in November as well as in 2026. So we expect that if all paperwork goes through the administrative process in the coming months, we will treat these patients.

Okay. 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 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

Okay. Thanks. And of the 12

Yeah. On the topic of 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 homegrown, you know, the patients at the QTCs. Yeah. 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 have 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 four significantly impacting that time frame. Thanks.

Okay. Thank you very much. Our next question is coming from Kristen Kluska 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? And if so, what's the timeline around reopening there?

Yeah. No. Thanks for that question, and it's a great question. Yes. We do have a shutdown which starts approximately, you know, the 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. That's accurate. Yeah. And this is really a mandated FDA requirement to have this type of shutdown at the end of the year for general maintenance and recalibration of equipment. But, yeah, 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 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, you know, 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'll take for us to resolve it. So we didn't take any chances there.

Okay. Thank you.

Thank you very much. Our next question is coming from Stephen Willey of Stifel. 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

Yeah. I can take that, Josh. So, yes, we do expect this will reduce over a period of time. Like I mentioned earlier, some of these patients, even though the

Yeah. And the only other thing I would add there, Josh, is that the very first few patients at the time when their prior authorization 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.

Right. I'll just say that this is nothing new about

Okay. Appreciate the color. Thank you.

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

Thanks so much for taking my question. Firstly, I was wondering if you could give us some additional granularity on what you the attrition rate, if any, to be among those patients for whom

I would say it's a pretty high level of conversion, Ram. These patients, because these are the patients that the physicians obviously, you know, these are motivated patients, they want to move forward, which is why we have the

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 QTC 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. 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 and giving us the

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 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. So the prior authorization process, Ram, essentially it's I can break it into two steps. One is a clinical prior authorization, and then the other is the financial, you know, discussion that takes place after a patient is clinically given a green signal from the insurance company. The prior authorization most payers, especially for these types of therapies, tend to follow the inclusion exclusion criteria of the clinical trials. And then there are some payers who also cover it, you know, 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 seven a one gene that they have recessive dystrophic EB. In our clinical trial, there were certain wound size requirements. We are seeing most of the insurance companies now, for example, UnitedHealthcare, covering the 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. So certain payers have those policies in place. But when you do have a payer that, let's say, it's a five-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

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, you can still join the queue by pressing star 1 on your keypad now. Our next question is coming from Jeffrey Michael Jones of 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?

Which 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. But 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 that hasn't settled down. So it's too early. We're gonna 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 these two accounts payable that's involved. That's different for different, you know, it's governed more by trade policies with each site. But for reporting purposes, the revenue is recognized the day of administration.

Great. Appreciate it, guys. Thank you.

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

Hi, guys. Thank you for taking our questions. Matt on for Jim today. Just one from us. Of the 30 patients that are slated to receive

We don't have visibility into that, Matt. I don't we would never have visibility into that, but we expect the vast majority would be on VYJUVEC and or on FIL

Got it. Thank you guys for taking our question.

Thank you very much. And our next question is coming from David Bautz of

Hey. Good morning, everyone. Just one for me this morning. So 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 if they haven't signed on yet.

Yeah. I can take that question. Good morning, David. Different sites have different reasons. I don't want to call out specific reasons for 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, you know, 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

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 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.