Abeona Therapeutics Inc. Q4 FY2024 Earnings Call

Greetings. Welcome to Abeona Therapeutics Full Year 2024 Results and Business Update Conference Call. At this time all participants are in a listen-only mode. Please note this conference is being recorded. I will now turn the conference over to your host, Greg Gin, VP of Investor Relations and Communications at Abeona. Greg, you may begin.

Thank you, Paul. Good morning, and thank you for joining us on our full year 2024 results and business update 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. Remarks made during today’s call may contain projections and forward-looking statements, 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. Various factors that could cause actual results to differ include, but are not limited to, those identified under the Risk Factors section 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. On the call today with prepared remarks are Dr. Vish Seshadri, Chief Executive Officer; Dr. Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development; Dr. Brian Kevany, Chief Technical Officer; and Joe Vazzano, Chief Financial Officer. And with that, I will now turn the call over to Vish Seshadri to lead us off. Vish?

Thank you, Greg. We appreciate everybody joining the call this morning. We’re less than six weeks away from our PDUFA date of April 29 for prademagene zamikeracel or pz-cel for recessive dystrophic epidermolysis bullosa or RDEB. Since resubmitting our pz-cel biologics license application in late October and receiving FDA acceptance in early November, we have been working with the FDA on the review process. As a reminder, the FDA has not indicated the need for additional site inspections or an outcome as part of this review. On March 14, 2025, we received from the FDA a marked-up version of the draft USPI to initiate label discussions. We have also received some post-marketing commitments and requirements from the FDA. The potential approval of pz-cel would be a significant moment for the RDEB community, a major milestone for our company, and would be the main driver of growth and profitability for Abeona for years to come. In anticipation of the PDUFA date, our launch preparations for pz-cel for RDEB are well underway. Five renowned EB treatment centers in the U.S. are undergoing onboarding and activation activities to become pz-cel qualified treatment centers or QTCs. We anticipate that these sites will be activated and ready to biopsy patients starting in the third quarter of this year, approximately three months after the PDUFA date. Similar to past autologous cell therapy launches, we anticipate launching pz-cel in the third quarter of this year, pending FDA approval. The RDEB community continues to highlight the unmet medical need for new therapies. Patients are seeking reliable and durable treatment options for their wounds. With the strength of our clinical data, manufacturing capabilities, commercial readiness efforts, and the significant cell and gene therapy launch experience of our commercial team, Abeona is poised to serve the U.S. RDEB community following an approval. We are confident that if approved, pz-cel and its differentiating clinical profile will transform the treatment paradigm for RDEB patients. I’ll end my remarks with a brief mention of our partnered program with Ultragenyx for Sanfilippo syndrome type A or MPS IIIA. In December, Ultragenyx submitted a BLA to the FDA seeking accelerated approval for UX111 for patients with MPS IIIA. Last month, Ultragenyx announced that the FDA granted the BLA a priority review with a PDUFA action date of August 18, 2025. So, 2025 is shaping up to be a very exciting and momentous year in our company’s history with the potential for two FDA approvals of our internal and partnered products. I’ll now hand the call to our Chief Commercial Officer, Madhav Vasanthavada, to highlight our commercial launch preparations for pz-cel. Madhav?

Thanks, Vish, and hello, everyone. With less than six weeks until our PDUFA date and label discussions started with the FDA, we are looking ahead to potential pz-cel approval and the prospect of positively impacting the lives of people living with recessive dystrophic EB. Before I share the status of our launch readiness, let me briefly recap how we see the commercial opportunity with pz-cel. In the U.S., based on medical claims data, we estimate there are approximately 1,300 dystrophic EB patients. Our research indicates that about 750 of those EB patients are RDEB patients with moderate to severe wounds who are potential candidates for pz-cel. An average RDEB patient has greater than 30% of their body wounded, and we estimate two treatment cycles of pz-cel would be needed to cover the vast majority of their existing wounds. In other words, with an average of two treatments per patient and about 750 patients, we foresee about 1,500 treatment opportunities for pz-cel in the U.S. At this time, we are not accounting for potential future RDEB wounds that patients may develop and other factors which could require additional rounds of pz-cel treatment for patients. Even with the conservative floor of $1.5 million per treatment, we believe pz-cel can have a cumulative revenue potential of more than $2 billion in the U.S. alone. Our confidence in realizing this opportunity stems from the continued enthusiasm we hear from patients, leading EB physicians, and payors. People with RDEB are desperate for therapeutic options that can reliably close multiple stubborn wounds in one treatment application and provide relief from the perpetual burden of chronic wound care. Pz-cel has the potential to be a groundbreaking therapy designed to deliver years of wound healing and pain reduction after a single treatment application, even in tough large chronic RDEB rebounds. Many of the EB physicians we spoke with at the recent American Association of Dermatologists Annual Conference indicated that they are looking forward to the approval of pz-cel. That said, pz-cel treatment, like other autologous cell and gene therapies, is an involved process that requires extensive treatment planning, travel, and care coordination. But despite this, patients are excited about the potential approval of pz-cel because of the robust clinical efficacy and durability seen in clinical trials. In a self-reported patient survey conducted two years after pz-cel treatment in a Phase 1/2a study, all seven participants reported that they would undergo pz-cel treatment again and would recommend the procedure to other patients with RDEB. In fact, in our ongoing Phase 3b trial, the majority of our patients are repeat patients who have chosen to receive pz-cel for their previously non-treated wound areas. One patient even returned for a third round. We want to make pz-cel available and accessible to this patient community as soon as possible following approval. As such, to talk about our launch readiness and expectations, we are in dialogue with five well-recognized epidermolysis bullosa EB centers to onboard them for pz-cel treatment. These centers are geographically dispersed, each with large catchment areas of patients and collectively can treat a sizable proportion of our estimated 750 pz-cel eligible patients. We expect the process of site activation to vary from site to site. Based on our experience with cell and gene therapy launches, site activation could take approximately three months post-FDA approval. During these initial months following approval, our teams will work with the centers to discuss eligible patients and secure the necessary payer authorizations. Our goal is to activate our target centers during this launch year, that being 2025, so that centers can begin treating patients with pz-cel in the third quarter of this year, as Vish indicated. We will announce the names of the pz-cel qualified treatment centers as they become activated. Once a site is activated, they can begin to schedule patients for biopsy based on available manufacturing slots and treat patients approximately 25 days from the time of biopsy collection. Revenue recognition will occur after the patient is treated with the product. In the early launch phase, we expect to see a gradual ramp-up at the QTCs, with centers treating one to two patients first as they get accustomed to the pz-cel treatment process, patient journey, and the reimbursement flow. As the sites become more experienced with pz-cel, we expect greater uptake of the product. This gradual ramp-up will happen in parallel with Abeona ramping up our manufacturing capacity to increase launch supply as we have always indicated we would, a topic that Brian will cover later on this call. From an overall demand standpoint, we are confident of pz-cel’s growth. Over time, with capacity ramped up in 2026, we intend to qualify additional treatment centers while also driving patient referrals into the QTCs from the community practices. Let me now turn to market access where our two main goals are to ensure favorable access policies for the patients and reimbursement for the treatment centers. We remain confident in meeting both of these objectives based on our payer interactions so far. Abeona will give QTCs the flexibility to procure pz-cel either as a buy-and-bill product or through a specialty pharmacy, with sites having the flexibility to choose. Since access and reimbursement are critical success factors for any high-cost gene therapy, we have been laser-focused on educating the payer community and have had over 40 pre-approval information exchanges communicating our clinical trial results. To remind you, in terms of the payer mix, our data shows us that 60% to 65% of RDEB lives are covered by commercial plans, about 30% to 35% by Medicaid, and the remaining less than 10% by Medicare. Payers recognize the persistent unmet need in the RDEB treatment landscape and the tremendous clinical value of pz-cel as they review pz-cel’s ability to cover large areas of the body and clinical data showing multiple years of wound healing and pain reduction after a single treatment application. Just last week, we were an emerging biotech sponsor at the PCMA Annual Meeting, one of the industry’s most important payer conferences, and we were pleased with the positive reactions we have received on the clinical and economic value of pz-cel during multiple private in-person engagements with the largest national payers covering the majority of RDEB commercial lives. At approval, we plan to file for the Medicaid Drug Rebate Application or MDRA and apply for a pz-cel-specific J-code so that state Medicaid programs will have an opportunity to carve out pz-cel from the bundled payment methodology with the goal of speeding up access and reimbursement. Finally, I want to mention Abeona Assist, our integrated support program designed to help eligible patients navigate their entire pz-cel treatment journey, including travel and logistics. With that, I would like to hand the call over to Dr. Brian Kevany, our Chief Technical Officer, to discuss commercial manufacturing for pz-cel. Brian?

Thanks, Madhav. We continue to prepare our current good manufacturing practices facility in Cleveland, Ohio, for manufacturing commercial-grade pz-cel drug products. As a reminder, our Cleveland site is a completely non-CDMO dependent facility for the production of pz-cel as well as our retroviral vector. As Vish mentioned, we anticipate launching pz-cel in the third quarter of this year, pending FDA approval, and we are on track from a manufacturing perspective to be prepared to begin receiving biopsy samples from qualified treatment centers and initiating the manufacturing process to support the planned launch. Our facility is led by a team of highly skilled production, process, assay development, and quality control scientists with expertise in cell and gene therapy, particularly in cell culture, analytical testing, and wet lab techniques. The maximum capacity at our CGMP facility supports pz-cel manufacturing for up to 10 patients or manufacturing runs per month. We have previously communicated that pz-cel will be a supply-measured launch, and there will be a ramp-up period to get to maximum capacity. We currently estimate our initial manufacturing capacity during the early launch phase to be about four treatments per month. We plan to gradually ramp that monthly capacity to six treatments starting roughly at the end of 2025 to early 2026, towards achieving a maximum capacity of 10 monthly treatments in the first half of 2026. We believe the planned manufacturing capacity ramp-up will sync up well with the ramp for QTCs treating our first patients during the early launch phase this year. It is important to note that we have a planned annual manufacturing shutdown for maintenance on equipment in the facility, which is standard for cell therapy facilities. This planned shutdown is crucial to maintain operational integrity, regulatory compliance, and product safety, and we schedule it during the December-January holiday time frame to minimize any temporary impact on our production capacity. We have already taken steps to enable capacity expansion beyond our current manufacturing footprint. During the fourth quarter of 2024, we leased additional building space in Cleveland adjacent to our existing facility, which will allow us to create additional manufacturing space to increase production capacity for pz-cel. In the coming months, we will be assessing design plans and we’ll share more on our increased manufacturing capacity on a future call. Our long-term plan is to build manufacturing capacity to support 200-plus annual pz-cel treatments by the second half of 2027. I’d like to hand the call over to our Chief Financial Officer, Joe Vazzano, to discuss our financial results.

Thank you, Brian. I would like to remind everyone that you can find additional details on our financial results for the full year ended December 31, 2024, in our most recent Form 10-K, which is available on our website. Starting with the financial resources on our balance sheet, we had cash, cash equivalents, short-term investments, and restricted cash of $98.1 million as of December 31, 2024. This compares to $52.6 million as of December 31, 2023. Based on our current operating plan and assumptions with our existing cash resources, we estimate we have sufficient financial resources to fund operations into 2026. Our cash runway assumptions do not account for any potential revenue from commercial sales of pz-cel or proceeds from the sale of a Priority Review Voucher, or PRV, if awarded by the FDA. I’ll remind you that pz-cel has been granted rare pediatric disease designation by the FDA. Upon its potential approval, we believe that we are eligible to receive a PRV. Research and development expenses were $34.4 million for the full year ended December 31, 2024, compared to $31.1 million for the full year ended December 31, 2023. Our spend on general and administrative activities was $29.9 million for the full year ended December 31, 2024, compared to $19 million for the full year ended December 31, 2023. The increase in general and administrative expenses is primarily due to commercial launch preparation costs, which included the hiring of a well-experienced commercial team to support the potential launch. Net loss was $63.7 million for the full year ended December 31, 2024, or $1.55 loss per common share, compared to a $54.2 million loss or $2.53 loss per common share for the full year of 2023. Lastly, we have received a question from investors about when we will start recognizing revenue after pz-cel’s commercially launched. As we stated earlier on this call, we are planning for a commercial launch in the third quarter of this year if pz-cel is approved. Revenue recognition occurs upon pz-cel administration, which is the surgical procedure. So that gives you a sense of the time it would take from a patient being biopsied through the 25-day pz-cel manufacturing process to when we recognize revenues. And with that, I will open the call for Q&A. Operator, can you please open the Q&A session of this call.

Certainly. The first question today is coming from Kristen Kluska from Cantor. Kristen, your line is live.

Hi, good morning everyone. Thanks for taking my questions, and I just want to say I very much appreciate how transparent you’ve been with us during this review process, including the last year. Considering where you are in discussions right now with the agency, do you feel that the FDA is satisfied with the work you’ve done on the CMC side that resulted in the CRL last year? Have you fielded more questions specific to that? Do you have a sense of any work that you’ll need to do on that side?

Good morning, Kristen, thank you for the question. I would say this: we have addressed all the asks of the FDA from our side that came out from the CRL. We did not negotiate any of those points, those 12 items that we articulated last year. We took the approach of comprehensively addressing each and every one of those, and we believe we’ve done that. The reason we believe we’ve done that is because of how many touch points we’ve had—like five informal meetings with them in the time between the CRL and the resubmission and also a formal Type A meeting where we got a lot of our questions answered. We feel like we have addressed all of those. Of course, technically, we cannot say the FDA has said, ‘You’ve satisfied all these items,’ because the review is still ongoing. But from our end, we have really checked those boxes. I hope that addresses your question?

Yes, it does. Thank you. And then of the five centers that you referenced, have you had conversations with them specifically just to get an early sense of maybe the percent of their patients that may be eligible for this therapy over time?

Great. For that question, I’ll actually turn over to Madhav to address it as the person that is directly in dialogue here.

Yes, Kristen, thanks for that question. These centers that we are talking to are large economic institutions with patients. We will get more concrete numbers closer to approval in terms of how many patients are actually eligible, but we continue to hear from them that there are patients they see pz-cel as a really good option for the moderate severe RDEB patients, primarily complementary to existing treatment options. So from that standpoint, they have patients that they are considering. Soon after we have approval, we’ll begin to clear all of the access-related topics that we discussed. In terms of numbers of patients, about 30% of the 750 patients primarily based on the claims analysis are in seven centers of excellence, five of which we are already in discussions with. So we are looking at at least a triple-digit number of patients that are currently housed within these institutions, and a sizable number of those triple-digit patients would at least be at the early phase of launch candidates, and then they will come on the product over time.

Okay. Thanks. And last question for me. I’ve seen a lot of new investor faces on the story over the last few weeks and months in particular. Can you just remind us what’s really going to be the top reason for patients wanting to seek this therapy? Is it simply the wound profile itself and having to deal with the advantages, or is it in particular because these wounds make patients more susceptible to things like squamous cell carcinoma and infections, which are leading causes of mortality here? What is really the key reason?

I think the key reason from what we are hearing is the need to have a durable, or longer-term closure of the wounds. Really, these patients have multiple wounds on their bodies, and if there is a treatment option that can minimize the chances of infections, reduce the need for bandage and wound dressing changes, these are significant quality-of-life impacts. Squamous cell carcinoma is certainly a significant factor that concerns not just patients and caregivers but also physicians. We know there is a high correlation between squamous cell carcinoma occurrences as wounds become more chronic, so they are always keeping an eye on that. Therefore, particularly for chronic wounds, if you can treat and heal these wounds, then that’s a significant impact for them. So that’s really the primary driver. Over time, I think we will learn many more quality-of-life impacts as patients get treated.

Thanks again, and wishing you all the best in the next six weeks.

Thank you, Kris.

Thank you. The next question will be from Maury Raycroft from Jefferies. Maury, your line is live.

Hi. This is Amin on for Maury. Thank you for taking our questions. Two from us. First, on the label draft that you mentioned, is the draft that you received from the FDA in line with your vision for pz-cel? Or do you anticipate further modifications and discussions with the FDA? The second question is probably for Madhav: upon the approval, do you expect a patient backlog until you reach maximum manufacturing capacity, or do you think patient interest and manufacturing capacity will increase gradually without creating that backlog?

Thank you for the question. I’ll take the first one and then pass it on to Madhav. Your question was whether the draft label is aligned with our expectations. The short answer is yes. As you can imagine, there are many details that will get refined over back and forth between the FDA and the sponsor. However, regarding the big-ticket items of how we look at the label, there are no big surprises here. Madhav?

Yes. I would add regarding the patient backlog; we do anticipate that happening. We mentioned it early on, and we continue to feel that this particular launch is supply gated for several months to come at the very least as we ramp up to 10 patients a month. In response to Kristen's earlier question, we have received interest from these physicians identifying patients for when we will work with them to start reserving manufacturing slots. So early on, at the time of launch, for several months as we release manufacturing slots, we want to ensure that these slots are filled for the foreseeable months. We will keep you updated on how that shapes up, but yes, we expect there will be a queue of patients.

Thank you very much.

Thank you. The next question is coming from François Brisebois from Oppenheimer. Your line is live.

Hi. Thanks for taking the question. Thanks for the updates on the developments here. I was just wondering, you mentioned your conferences recently. Can you help us understand maybe the physician education that’s necessary? The trials have been going on for a while, so I’m just wondering if this is a situation where the doctors are just waiting for it? Or is there still, based on the fact that it’s not approved yet, a lot of physician education and training that’s necessary at launch? Then I’ll have a follow-up.

Physician education is certainly needed. In terms of our focus areas, we really are discussing in greater detail with the qualified centers we are looking to onboard and qualify. For the rest of the physicians out in the community, there are about 23 centers of excellence that have coordinated practices for EB clinics. We are really targeting a subset of those at the time of launch. For the rest of the centers, as well as for the community physicians, we have not gone out and discussed really pz-cel with them because we are still waiting for the approval to happen. As we raise our promotional measures and efforts, we will engage with other patients who have gone through clinical trials and share their experiences to raise awareness better.

Yes. No, no, absolutely. The competitor having a product out there is probably helping with awareness. But I guess so on the physician side, and then on the patient angle: this RDEB community is so severe. I’m just wondering how tightly knit the patient community is? I’m not talking a full-blown DTC, but is there an angle on the digital side? Is there an online community that makes these patients very aware quickly of the opportunity? Or how do you market to that?

Absolutely. It’s a very tightly knit patient community. We have advocacy groups that have been great partners with us even through the approval process. We will have a digital presence, and we are engaging with patients to share their stories as well as our website presence and social media to raise awareness. Since we are coming in second in the market with a gene therapy, it could create advantages not only in terms of patient identification but also for other aspects of payer and access clearances. In terms of our digital presence, we will implement all of the right measures, and we’ll have field personnel out in the community to raise awareness and start helping with patient referrals to qualified treatment centers. That’s really going to be our two-pronged approach, but given our supply capacity, we are also trying to gauge how rigorously we want to implement these measures.

Thank you.

Thank you. The next question is coming from Ram Selvaraju from H.C. Wainwright. Ram, your line is live.

Hi, thanks for taking my questions. Firstly, on the PRV, I was just wondering if you could enumerate any potential factors that might preclude the PRV from being granted, or if at this juncture you fully expect it to be awarded? And also, if you could comment on the recent environment for PRV resales.

Good morning, Ram. Thank you for the question. We do not anticipate any potential factors that may preclude a PRV for us. In general, the big requirement items for PRV eligibility are you should have a rare pediatric designation, which we do. The second is we should have priority review in the current review process, which we do. Finally, this must be a new drug. It’s prademagene zamikeracel—our first-time launch. If you look at past examples, if the same drug were rebranded, then there was an instance where a PRV was not granted. We don’t believe that applies to us because it’s a first-time launch for us. Regarding the recent trend in PRV sale, we have seen that since the program has been put on hold or there’s uncertainty surrounding the future of the PRV program, that uncertainty has created a different pricing trend. The last four PRVs were sold for more than $150 million, and our goal is to optimize pricing. We’re not in a rush to sell the PRV; we have time to maximize pricing over speed.

To add to that, one of those four PRVs was sold within a week of receiving approval for the full $150 million price tag.

Okay. Very helpful. The only other follow-up was regarding potential positioning and deployment of pz-cel in markets outside of the U.S. In particular, I wanted to see if you had any comments regarding logistical potential deployment for treating European RDEB patients and what other high-priority markets you see as being viable for pz-cel outside the U.S.

Sure. I will start and then Madhav can add his thoughts. We have a lot of interest from Europe and some Asian markets for pz-cel. However, from a bandwidth perspective, we haven’t been able to engage in much depth as we wanted to get through the pz-cel approval in the U.S. first, then we will have more meaningful discussions. The interest exists, and we need to do more homework on the timing of supply. Our default assumption is that we will need to set up manufacturing in a European location. However, we are hearing options for product supply to European countries from a U.S. location depending on shelf life even for a transplant-like product that has limited shelf life. We will explore those options after the launch. Madhav, feel free to add anything.

Yes, you’ve addressed most. I would say within Europe, we are looking for markets where the reimbursement landscape also looks positive. We know that, for instance, Austria and Germany might be markets where there is a greater appetite. Austria, especially, is a big center for these kinds of surgeries. Overall, from a logistics standpoint, it’s not about venturing into multiple markets but rather maximizing existing infrastructures and patient pathways in those markets. If we can deploy in one or two centers and take advantage of that, it would be beneficial from both a cost and speed-to-market perspective. Other markets will require more analysis, but we see Japan as a potential, as well as Asia Pacific over time after we secure U.S. FDA approval.

Thank you.

Thank you. The next question is coming from David Bautz from Zacks Small Cap Research. David, your line is live.

Hey, good morning, everyone. Thanks for the overview this morning. You mentioned that you want to get five treatment centers online with launch, but what is the ultimate goal for the total number of treatment centers you’d like to see one day? Is it to get it up to 10, 15? What do you see in terms of that?

I think the total number, at this point in time, we don’t anticipate going more than 10. Yes, five this year and building it up slowly. Part of the reason is patients are already accustomed to traveling to specialty centers for various procedures for infections or surgical reasons, they are comfortable traveling up to 200 to 250 miles. We want to keep the number of centers tight and build the experience curve for these centers. As they treat more patients, they become better at it. That’s really our goal. This approach also matches with our supply since we’ve launched CAR T-cell therapies in the past and faced supply issues. So we want to be cautious in terms of managing that experience.

Okay. Great. Thanks for that. Most patients are probably going to require two treatments. What is the maximum service area that a patient can be treated at one time? And what dictates that number?

The maximum surface area that can be treated is we can supply up to 12 sheets in collagen-producing keratinocyte sheets, and one sheet is 40 square centimeters. So, 12 times 40 gives us up to 480 square centimeters. From natural history studies, patients have more than 30%, and some even up to 80% of their bodies wounded in recessive DEB. If we take the 30% and conduct a back-of-the-envelope body surface area analysis, we estimate around 1,000 square centimeters of the body covered by large chronic wounds on non-flexion areas. With that calculation, we estimate about two rounds would be needed since it could be 12 sheets for each patient. However, we cannot guarantee that there are going to be 12 sheets for each and every patient because it’s a cellular engineered cell therapy. For that reason, it could be eight or ten. The factors we see over time will dictate how many treatment cycles are required and how patients will return for treatment.

Okay. Great. I appreciate the information there. Thanks for taking the questions.

Thank you. There are no other questions in the queue at this time. I would now like to hand the call back to Vish Seshadri for closing remarks.

Thank you, Paul. Our top priorities continue to be to secure FDA approval for and launching pz-cel in the U.S. as a new treatment for RDEB patients. We believe we are well positioned for the significant opportunity ahead of Abeona, and we look forward to a potentially transformative 2025 for the company. Thank you, everyone, for joining us for today’s business update. We’ll talk to you again soon.

Thank you. This does conclude today’s conference. You may disconnect your lines at this time. Thank you for your participation.