管理層發言
Good morning, everyone, and welcome to Abeona Therapeutics 2Q 2026 Conference Call. Operator Instructions: Please note this conference is being recorded. I will now turn the conference over to your host, Gregory Gin, Vice President, Investor Relations and Corporate Development. Greg, the floor is yours.
Thank you, Jenny. Good morning, and thank you, everyone, for joining us on our second quarter 2026 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. Joining me on today's call are Dr. Vish Seshadri, Chief Executive Officer; Dr. Madhav Vasanthavada, Chief Commercial Officer; Joe Vazzano, Chief Financial Officer; and Dr. Brian Kevany, Chief Technical Officer. 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. And with that, I will now turn the call over to Vish Seshadri to please start. Vish?
Thank you, Greg, and good morning, everyone. I'll begin today with an overview of our commercial progress before turning the call over to Madhav for operational details. Our commercial experience to date reinforces our confidence in ZEVASKYN's substantial commercial opportunity. During the second quarter, we advanced our rollout by expanding our qualified treatment center network and progressing more patients through the treatment pathway. With the recent addition of Cincinnati Children's, which is one of the largest epidermolysis bullosa treatment centers in the country, we now have 7 activated QTCs nationwide. Importantly, CHOP and UTMB are biopsying patients and CHOP has completed its first treatment. We have treated 12 patients since launch, including 5 in the second quarter of 2026 and 3 additional patients in the third quarter to date. As Madhav will discuss further, a couple of these treatments did not generate revenue. As our commercial footprint expands, we're refining how we report progress to the investment community. Over the past quarter, we have seen that leading indicators such as scheduled biopsies or biopsies in manufacturing are subject to external variables outside our control and have limited utility in predicting revenue-generating treatments. Later on the call, Joe will outline the specific reporting updates we're making to eliminate this uncertainty and to align with standard practices of commercial stage companies. With that, I'll turn the call over to Madhav Vasanthavada, our Chief Commercial Officer, to detail our commercial execution and network expansion. Madhav?
Thank you, Vish, and good morning, everyone. We are making progress in executing the launch with clear priorities, advancing identified patients through the treatment journey, strategically expanding the QTC footprint by onboarding leading EB centers and raising ZEVASKYN awareness across the EB community. Let me start with EB community engagement since we just attended back-to-back meetings, the Debra Care Conference, which is a flagship meeting for EB patients hosted by Debra of America, a patient advocacy group, and the Society of Pediatric Dermatology, SPD, Annual Meeting, where we interacted with dozens of highly engaged patients, caregivers and physicians. Patient ambassadors from our Strong Together Network, which is a group of patients who received ZEVASKYN in clinical trials, engaged with families and physicians throughout these events, fostering meaningful dialogue, sharing the impact ZEVASKYN has had on their lives and helping connect patients and caregivers with ZEVASKYN resources and support. Following these interactions and the many that we have been having in recent months, we are energized by the opportunity and the fundamental role ZEVASKYN can play in healing RDEB wounds today and for the years to come. Based on our interactions with patients and caregivers, we continue to believe that the distinct value of ZEVASKYN is deeply resonating with the RDEB community. We see clinical conviction building across our QTC network and the community practices of other RDEB physicians. While we are thrilled with the patient community's interest in ZEVASKYN, our recent experience has revealed bottlenecks that we are working through in the journey from patient identification to ZEVASKYN treatment. Because ZEVASKYN is the first surgically applied autologous cell therapy in dermatology and is operationally very different from traditional topical therapies, QTCs have a steep learning curve to climb. As a result, the time from patient identification to patient treatment can vary considerably site to site and be influenced by a broad range of factors that are beyond the control of individual stakeholders. Executing the launch has provided us with several real-world learnings, of which I'd like to highlight three important ones. First, administering ZEVASKYN, which has an 84-hour shelf life, requires a QTC to execute seamless real-time coordination across multiple stakeholders. Well before requesting a biopsy slot, dermatologists, surgical specialists, anesthesiologists and hospital staff must lock in precise dates for biopsy appointments, for operating room reservations and for surgeon and medical teams. These unique operational dynamics require even greater planning, particularly when patient and physician availability can be limited with holidays and back-to-school planning, and we have seen this lead to scheduling disruptions for biopsy and treatment dates. Second, on the clinical side, we have observed that the health of harder patients can sometimes change unexpectedly, which can lead to unavoidable biopsy delays or cancellations. In the second quarter, patient health deterioration resulted in two last-minute cancellations of scheduled biopsies. Because of the amount of coordination required at the QTC with payer and patient schedules well before booking the treatment slot, a last-minute cancellation means that the slot cannot be filled by another patient. Lastly, on the supply side, as our patient sample size has continued to grow, we have learned that manufacturing yields can be influenced by variability in the incoming biopsy material. These factors resulted in one low-yield batch in Q2 and one out-of-specification batch in Q3, for which no revenue was recognized. Despite these challenges, we are gratified that both patients received treatment. While the launch has highlighted these complexities, they have provided valuable operational and commercial learnings that continue to strengthen execution by both Abeona and its QTC partners. Importantly, despite these complexities, we have maintained steady quarterly growth and 12 patients have now been treated with ZEVASKYN since launch. As we apply these launch learnings, our focus remains on ensuring more patients enter the top of the funnel to help offset patient attrition that can occur for reasons beyond our control. A key component of that strategy is continued expansion of our qualified treatment center network, continued engagement with the EB community and improving patient access. Towards that end, during the second quarter, we activated two leading institutions, New York-Presbyterian, Columbia University Irving Medical Center and Children's Hospital of Philadelphia, CHOP. More recently, Cincinnati Children's Hospital, one of the largest EB centers in the U.S., has come on board. Activating treatment sites has taken significant time and commitment from QTCs and Abeona teams, and I want to thank everyone involved who helped achieve our stated goal of activating seven QTCs by the end of this year. With our expanded QTC network, about 40% of our addressable market now has in-state access to a QTC based on claims analysis. In addition, our QTCs also provide specialized care for a sizable portion of patients traveling from out of state, which enables even broader patient access. That said, we are getting requests from additional EB centers to onboard ZEVASKYN, and we plan to work with those centers to further our expansion of the QTC network. While site activation is a critical milestone, it is only a first step that allows a QTC to initiate ZEVASKYN's treatment process, including consultation, patient workup and payer engagement. Our commercial and medical teams continue to communicate regularly with each activated center as they build treatment readiness and administrative planning, including pharmacy and therapeutics committee review, prior authorization and payer agreement processes and planning for surgery and logistics. As an example of exceptional operational efficiency, CHOP completed its first ZEVASKYN treatment in July, shortly after its activation in May. UTMB recently completed its first patient biopsy, representing another important step towards future treatments and overall reflecting growth in the number of QTCs that are treating patients. Next, as we think about the long-term adoption curve for ZEVASKYN, we know that physician confidence and learning builds over time. Our QTC physicians rely heavily on multicenter real-world experience shared through peer-to-peer dialogue before transitioning a new therapy like ZEVASKYN into standard practice. As we actively facilitate best practice sharing amongst QTCs and the early treaters observe positive post-treatment outcomes and share them with their peers, we believe that this growing clinical conviction will trigger the tipping point that bridges initial experience to broad clinical adoption and routine prescribing across our entire QTC network. Based on recent discussions with RDEB physicians, we expect that enthusiasm for ZEVASKYN will continue to build as RDEB physicians see and share even more examples of positive treatment outcomes. Equally important for adoption is ensuring economic alignment and reimbursement for our treatment centers across all payer channels. To that end, we achieved a significant milestone from CMS, granting new technology add-on payment or NTAP status for ZEVASKYN effective October 1, 2026, for fiscal year 2027. NTAP is a CMS program that provides hospitals with supplemental reimbursement for eligible new high-cost and innovative therapies during inpatient stays, helping to cover the costs beyond standard DRG payments. For fiscal year 2027, CMS had received 15 new applications under the traditional pathway, and ZEVASKYN was one of only three to achieve NTAP status. The other 12 either did not meet the requirements or withdrew their applications or were denied. We are pleased that CMS has granted ZEVASKYN a new technology add-on payment. This is a significant recognition that comes after months of rigorous clinical review and public commentary. And it is an external validation of the newness, cost criterion and substantial clinical improvement that ZEVASKYN offers over existing treatment options for RDEB. While Medicare represents about 10% of the RDEB payer mix, NTAP now provides a mechanism for hospitals to seek a substantial add-on reimbursement and facilitate patient access. In closing, we remain encouraged by the demand we see and are focused on ensuring eligible patients can receive ZEVASKYN. Our approach is to achieve this by building robust access, expanding our QTC networks and further improving patient and QTC treatment experiences, which is exactly what we are doing. With that, I'll now pass the call to our Chief Financial Officer, Joe Vazzano, to discuss our financial results.
Thanks, Madhav. Let me start by reviewing the reporting changes we're making to provide maximum transparency and align with standard commercial stage practices. We will anchor future quarterly disclosures around completed operational achievements, specifically patients treated during the quarter and net revenue recognized. Consequently, going forward, we will report treatment activity solely within the designated quarter. Before reviewing the financial results, I would like to remind everyone that you can find additional details for the quarter ended June 30, 2026, in our most recent Form 10-Q. Starting with the statements of operations. For the quarter ended June 30, 2026, Abeona reported net ZEVASKYN revenue of $11.4 million, representing a quarter-over-quarter increase of 31% or $2.7 million compared to $8.7 million in the first quarter of 2026. While five patients were treated with ZEVASKYN during the second quarter of 2026, we recognized revenue for four treatments as one batch had cell yield that was below the thresholds for revenue recognition. Research and development expenses were $5 million for the second quarter of 2026 compared to $9.6 million in the first quarter of 2026. R&D expenses in the first quarter of 2026 included a one-time upfront cost of $7 million for in-licensing ABO-701. Selling, general and administrative expenses were $15.8 million for the second quarter of 2026 compared to $19.5 million for the first quarter of 2026. The decrease primarily reflects fewer engineering runs and less manufacturing training costs in the second quarter of 2026. We reported a net loss of $20.2 million or a loss of $0.35 per basic and diluted common share for the quarter ended June 30, 2026. Net loss for the first quarter of 2026 was $17.1 million or $0.30 per basic and diluted common share. As of June 30, 2026, we maintained a strong balance sheet with cash, cash equivalents and short-term investments totaling $146.8 million. Our focus remains on disciplined capital allocation as we drive toward a sustainable cash flow positive business model, which we believe is achievable by maintaining a consistent cadence of patient treatments. And with that, I will pass the call back to Vish for additional remarks before opening the call for Q&A.
Thank you, Joe. In closing, we're proud of the dedication shown by our commercial, medical, manufacturing and quality teams, and we look forward to bringing ZEVASKYN to many more families. While we continue to learn to overcome the unique launch challenges associated with the logistically complex product delivery, each learning helps us lay a strong foundation to deliver sustained long-term value to both the RDEB community and our shareholders. With that, I will hand the call back to the operator to open the line for your questions.
分析師問答
Operator Instructions: Our first question is coming from Maury Raycroft of Jefferies.
This is Amin on for Maury. A couple of questions from us. First, you previously mentioned one patient per month per QTC is a reasonable near-term cadence. When do you expect your existing QTCs, particularly leading ones like Lurie and Stanford, to reach that? And I have a follow-up.
Thank you, Amin, for that question. Yes, I think Madhav is best positioned to answer this question.
Thanks, Amin. I think, yes, we continue to hear about one patient per month from QTCs on average. And this is something that we will have once all of these centers are reaching a steady state. As we now know, earlier, Lurie and Stanford were the ones treating. Now we have biopsy from UTMB and CHOP has treated a patient. So, we're just waiting for other centers also to open up to be able to say when we reach a steady state. But once we are in the steady state is when we believe that one patient per month cadence is something that we continue to hear from the QTCs.
Okay. And given you've now seen both a low-yield batch and an out-of-spec batch, how should we think about the long-term success rate for manufacturing? Do you view this as isolated incidents? Or do you think this could be something that we will see in future as well?
Thank you for that question. It's a little early. As you recall, our manufacturing experience in the clinical trials was a total of 11 patients treated; it's a very small data set. Between the clinical trial as well as the subsequent clinical studies of Phase IIIb and our manufacturing experience to date, the low-yield batch is the first time we've encountered this. Up until this point, it looks like a low probability event. There are several variables that cause such events that are related to variations in the incoming biopsy material. It could be related to the anatomic locations where biopsies are taken or a particular patient status or just the cellular yield and growth characteristics that we derive out of any given biopsy. Given the limited experience, this is a rare kind of event where the cell yield was low. Fortunately, whatever sheets were manufactured were used for patient treatment; it's just that it's below the threshold of a billable unit. Having said that, we continue to run a lot of process science on every manufacturing run that we conduct, and hopefully, with enough experience, we'll be able to point towards reasons why this may happen and how we can improve upon that. But it's very hard to predict what such ratios could be. Since you asked about the out-of-spec, I wanted to also mention what it was about. You may recall that there was one test that we never had in clinical development, which is the identity test that relates to the Pan-CK marker expression on keratinocytes. Since there was no clinical experience, the way in which thresholds or specifications were set for this test was based on six samples — five being healthy volunteers and one frozen RDEB sample that we had at the time of BLA review. This was not based on true GMP manufacturing run experience to set such specifications. That was the test that failed. This has nothing to do with either the safety of the product or the potency of the product. We are working with the agency to revisit whether the specifications that were set during the BLA review were appropriate or should be re-evaluated. Some of these things will take time and more experience to get concrete numbers to put on what should be our assumed rate of non-billable units. If you look at overall numbers to date for any autologous therapy that has been launched in the past, you will see such examples. We'll continue to keep refining numbers and probabilities as we gain more experience.
Our next question is coming from Stephen Willey of Stifel.
Can you remind us of the manufacturing yields that you're seeing in the commercial setting? I know you have the capacity for 12 sheets on a per-patient basis, but what's the average number of sheets you've been able to manufacture for the patients you've treated thus far? And I guess, how does this differ, if at all, from the prior clinical trial experience? And I just have a follow-up.
Steve, the manufacturing yields from our commercial experience are actually very favorable when you compare them to the clinical trial experience. You may recall that for VIITAL, our Phase III trial, the maximum number of sheets that were allowed to be put on patients was six per patient, and in reality, it was about five sheets across the trial. Right now, we are around nine sheets average per lot, which is a pretty healthy rate compared to our clinical trial experience. That is why we're calling the low-yield event anomalous or rare. Of course, 12 is the maximum that we can supply, but we're learning from every batch and making sure that any indicators that tell us about expected yield are integrated into our process to adopt best practices in real time. We're pleased by averaging nine sheets, which is a substantial body area coverage.
And then can you say what that low-yield number is that triggers your inability to recognize revenue?
Anything that is fewer than four sheets in a batch is considered a low-yield batch. So three, two or one sheet batches are still on-label per the NDC but, for billing purposes, we will not recognize revenue for those batches.
Okay. And then just with respect to the pan-CK marker assay that you mentioned on the keratinocyte side, where are you now in terms of engaging the agency around either changing that number with a larger sample size or widening the confidence intervals?
We have had some interactions with the agency. The first thing was to make sure that we could treat the patient, which is why we had to go through communications with the agency, and we were successful in treating the patient. We should have more updates on where we are with the revision of the specification by the next quarterly update because we're still gathering the data. We're confident that the data from our manufacturing runs in the GMP setting now justifies a revision of the specification based on real-world experience versus something that was set based on limited experience during BLA review. It's TBD how quickly this can be implemented because there are mechanisms that involve the FDA. We'll update you in subsequent quarters on this particular topic.
Our next question is coming from Ram Selvaraju of H.C. Wainwright.
Congrats on all the progress made this quarter. I wanted to ask about kind of last-minute cancellations, arbitrary withdrawals of patients from the process of ZEVASKYN treatment and how often you see that specifically occurring? So this has nothing to do with failures in manufacturing or inability to qualify. This specifically has to do with patients being unwilling to ultimately go through the treatment process, what we might call the arbitrary attrition rate. Just maybe you could give us some sense of how often that occurs based on current experience.
Thanks for the question. Madhav?
Yes. So far, we have had two such events, as we mentioned, that have happened. It's hard to predict, but if you look at the willingness for these patients to undergo the procedures, there's definitely very strong willingness. Some of these cases are situations where patients are unable to make it because of illness or some health deterioration reasons, and in those cases we are talking about moving the biopsy date to another date. It's not that the patients don't want to or are just backing off of the procedure itself. Coming out of the Debra conference and SPD that I mentioned, we were energized. Literally seeing the number of patients we engaged with at our booth who are talking about ZEVASKYN — some had concerns about what the biopsy and procedure look like — but our Strong Together Network members who went through the procedure in clinical trials shared their experiences. The product theater we presented was also packed; we had room for more than 160 people and there was a lot of interest to learn about the procedures and outcomes. So even if patients are dropping out for health deterioration reasons, we have not seen patients saying they don't want ZEVASKYN. It's a matter of rescheduling the biopsy to another date. When that happens, especially in a quarterly report like this, when we talk about the number of slots and the number of patients, we will have different numbers for that finite period of time. That's really how this current model is. We are not saying there is permanent patient attrition; we've only seen two such events so far.
And then with respect to maximizing patient accessibility and convenience, you said in your prepared remarks that at this point almost half of the addressable patient population has in-state access to a qualified treatment center. Could you elaborate from two perspectives: firstly, how necessary it needs to be for a patient to have in-state access to a QTC? And secondly, in order for the company to provide this to the majority of patients — say 80% of the addressable patient population — how large would the QTC network theoretically have to be?
Great question. On the first point, the importance of having a QTC in-state: when you have Medicaid, especially in-state Medicaid patients, access is much faster relative to a patient traveling from out of state because the physician needs to be enrolled in the host Medicaid state. So it helps to have a patient in the same state where you have a QTC from an access standpoint. When we say about 40% of our addressable patients are in-state, that's based on our claims data counting the number of claims in those states divided by the total number of claims across the country. It's not necessary to have a QTC in all states, and we will never have such a scenario. We'll have a limited number of QTCs because this is a rare community, and we know patients travel from out of state. In fact, about 40% roughly of the patient mix that a QTC has are coming from out of state, traveling 300 to 400 miles. We're dealing with leading EB centers. To get to an 80% coverage number, we can still achieve that through prioritization and by leveraging centers' ability to treat out-of-state patients. So far, many patients we've treated have traveled from out of state; that mechanism already exists for people to travel and get treated.
And then lastly, I was just wondering if you could give a sense of when you anticipate NTAP status to be reflected on two levels: first, the revenue cadence; and second, whether you expect it to show up on the margin front, and if so, how?
On the revenue cadence, it will depend on the payer of the patient. For Medicare beneficiaries, NTAP will apply primarily to those patients, whether they're pure Medicare or dual eligible. For those patients is where revenue will come in through NTAP. In the absence of NTAP, these patients would have had very limited access, if any. NTAP opens up important reimbursement for centers, which should facilitate access.
One more thing to add about NTAP status: it has two effects. The direct effect is for the approximately 10% of our patient mix that is dependent on Medicare reimbursement. However, the fact that we achieved NTAP after rigorous review is also going to have a halo effect with other payers in how they view the technology, because it's an external validation. That should make it easier for centers, even for other types of patients, to get paperwork done and aid payer negotiations. In terms of margins, it's more about ensuring hospitals are made whole — that's where NTAP plays a big role. For Medicare patients without NTAP, it's a significant P&L loss for a treating institution, and NTAP helps fill that hole and should de-bottleneck treatment for some of these patients in the centers.
Our next question is coming from Kristen Kluska of Cantor Fitzgerald.
So, you mentioned in your prepared remarks that you want to have more patients enter the top of the funnel in case some of these situations arise. Which parts or issues could having more patients at the top of the funnel potentially mitigate? And then which ones would this disruption still continue?
I think having more patients at the top of the funnel helps mitigate issues that are outside of our control. For example, if a biopsy date moves or a patient needs to reschedule, if you have multiple patients across multiple centers aiming to have biopsies, that helps offset and increases the chance of filling manufacturing slots, which are finite. That's the primary purpose. Increasing the number of QTCs also helps with patient access, travel and distance concerns. Patients often trust certain institutions more than others, so increasing footprint helps. We're already seeing cross-pollination of best practices through physician engagement at meetings like SPD, which helps raise awareness and clinical conviction in ZEVASKYN. So increasing the funnel helps on multiple fronts, and that's exactly what we're doing.
Given that some of these windows are very limited, does it make sense to do patient screening when they come in for biopsies to ensure they're healthy? I know you can't prevent 100% of potential cancellations, but can this mitigate it at all?
Potentially. The two example cancellations we gave occurred very close to biopsy — one was on the day of biopsy where the patient was very sick and couldn't travel, and the other was a day or two before. When cancellations are that last-minute it's very hard to adjust. If you have information two to three weeks in advance, that's something you can act on. That's why having more patients on the top of the funnel gives you more flexibility and shots on goal to move patients and adjust dates. We'll continue to monitor and hopefully we see fewer last-minute cancellations as we learn mitigation strategies.
Our next question is coming from David Bautz of Zacks Small-Cap Research.
So, my first one is about clearing up the revenue recognition. If I understand correctly, you said that two of the patients you didn't record revenue for, but I believe those two patients were still treated. So, is this a case where the company is going to incur the full treatment manufacturing costs? Or is there going to be a chance to recognize revenue for those two patients at a later date?
We will not be recognizing revenue for those two treatments because that is our agreement — whether it's a low yield or an out-of-spec batch, we absorb the cost of goods sold.
Okay. The Q2 gross margins look like they were about 63%. As the manufacturing process becomes more predictable, where do you see normalized gross margin settling?
Our gross margins are heavily dependent on the number of patients treated in a given quarter, mainly because most of our manufacturing costs are fixed. With higher volumes, margins will improve. We think a steady state would be roughly about 85% to 90% once we reach full operating capacity.
Can you give any additional details on the patient funnel — where it stands today? How many patients were identified or even biopsied or treated in QTCs? Any of those numbers would be helpful.
David, patients are interested. On prior calls, we mentioned more than 100 patients that have been identified by their community physicians and QTCs as clinically eligible. There are multiple downstream steps, including consultation and payer processes. The rate-limiting step is at the QTC and advancing patients through the treatment process. As that continues to happen, we believe patients will continue to move through given recent interactions with patients and physicians. I can't provide a specific current funnel number today, but we see movement happening.
Our next question is coming from Fanyi Zhong of Oppenheimer.
This is Fanyi for Jeff Jones from Oppenheimer. Clarification question: when you indicated you had a low yield, so revenue was not recognized for two patients — does that mean the patient is unable to receive any treatment or is there sufficient material for partial treatment? What happens in that scenario? Second, do you have a view for how long new QTCs take to begin treating patients?
To clarify, the two cases where we did not recognize revenue were two different cases: one was low-yield and the other was out-of-spec. Both patients were treated. Whatever sheets we produced and provided to the treatment center were used, so patients received treatment. We will not recognize revenue for those two treatments. On the question of how long it takes to activate a QTC and get to patient treatment, we previously indicated an average of four to six months, but averages are not useful when variance is high. For example, CHOP was activated in May and treated a patient in July, a quick turnaround, while we have had other sites that have taken 12 months since activation and not treated a single patient. Reasons for variability include payer mix in certain states, enrollment in state Medicaid, and administrative paperwork. We're learning from these experiences and more recent activations are having sites talk to patients and pre-line up parts of the process before activation, which could accelerate timelines, but it's hard to predict precisely.
Thank you very much. We appear to have reached the end of our question-and-answer session. I will now turn the call back over to Vish for any closing comments.
Thank you, Jenny. I'd like to thank everyone for joining us for today's business update, and we'll talk to you again soon.
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.