Abeona Therapeutics at H.C. Wainwright: Strategic Growth in Gene Therapy

On Tuesday, 09 September 2025, Abeona Therapeutics (NASDAQ:ABEO) presented its strategic initiatives at the H.C. Wainwright 27th Annual Global Investment Conference. The company highlighted its transition to a commercial-stage entity following FDA approval of its gene therapy ZevaSkin™. While Abeona is optimistic about its financial position and market potential, challenges remain in streamlining patient intake and expanding treatment capacity.

Key Takeaways

• Abeona has over $226 million in cash, providing more than two years of operational runway.
• ZevaSkin™ has been approved for treating recessive dystrophic epidermolysis bullosa (RDEB) and is priced at $3.1 million per treatment.
• The company aims for profitability by the first half of 2026, targeting over three patients treated per month.
• Two qualified treatment centers are operational, with plans to expand manufacturing capacity.
• Abeona estimates a $4.5 billion revenue opportunity from 750 eligible RDEB patients in the U.S.

Financial Results

• Abeona holds $226 million in cash as of June 30, 2025.
• The company anticipates profitability by mid-2026 by treating more than three patients monthly.
• ZevaSkin™ offers a potential cumulative revenue of over $4.5 billion, with 1,500 treatment opportunities priced at $3.1 million each.

Operational Updates

• FDA approval for ZevaSkin™ was granted in April 2025.
• Two treatment centers are active: Ann & Robert H. Lurie Children’s Hospital of Chicago and Stanford Medicine Children’s Health.
• Nearly 50 patients have been identified, with a treatment process taking three to four months.
• Manufacturing currently supports four patients per month, with a planned shutdown in December 2025 for GMP compliance.

Future Outlook

• Abeona plans to increase the number of activated treatment centers.
• The company is working to enhance manufacturing capacity to meet growing demand.
• Efforts are underway to secure more favorable coverage policies from payers.
• Abeona is developing additional gene therapies for inherited retinal diseases.

Q&A Highlights

• Discussions focused on patient identification and reimbursement momentum.
• Abeona can currently treat four patients monthly, with plans to expand.
• Approximately 40% of patients travel from out of state to receive treatment at the centers of excellence.

For further details, please refer to the full transcript below.

Full transcript - H.C. Wainwright 27th Annual Global Investment Conference:

Daniel Smith, HC Wainwright Equity Research Associate in Biotechnology, HC Wainwright: Good afternoon, everyone, and thank you for joining Day 2 of the HC Wainwright 27th Annual Global Investment Conference 2025. My name is Daniel Smith, and I’m an HC Wainwright Equity Research Associate in Biotechnology. With that said, let me introduce our presenters for the session. I’d like to welcome Madhav Vasanthavada, Chief Commercial Officer of Abeona Therapeutics Inc., and Joao Siffert, Chief Financial Officer of Abeona Therapeutics Inc., who develop and commercialize cell and gene therapies for serious diseases. Abeona is traded on the NASDAQ under the ticker ABEO. Gentlemen, the floor is yours.

Madhav Vasanthavada, Chief Commercial Officer, Abeona Therapeutics Inc.: Thank you. Thank you, Daniel, and thanks for hosting us here. I’m very excited to be presenting here today our story, Abeona story, and it’s the first time we are actually presenting here as a commercial stage company. Looking forward to sharing the updates as we are progressing towards our launch, and just wanted to go through some overlooking job.

Daniel Smith, HC Wainwright Equity Research Associate in Biotechnology, HC Wainwright: Yep, and this presentation will contain forward-looking statements. For further information, please reference our SEC filings.

Madhav Vasanthavada, Chief Commercial Officer, Abeona Therapeutics Inc.: All right, let me just start off by saying that at Abeona, we are really setting us up for growth with the recent FDA approval of ZevaSkin™, and from a cash position, we have never been in a stronger position in recent years with a strong balance sheet and the cash situation to get us going for more than two years of operations. With regards to ZevaSkin™, which is approved for the treatment of recessive dystrophic epidermolysis bullosa wounds in adult and pediatric patients, we are seeing a significant level of interest right out of the gates from the recessive dystrophic epidermolysis bullosa community.

As we announced in our recent earnings call, we have gotten approval for ZevaSkin™ in April of this year, and already we are available at two qualified treatment centers at Ann & Robert H. Lurie Children’s Hospital of Chicago and Stanford Medicine Children’s Health, and in the process of activating additional QTCs. We’ve already identified nearly 50 patients for ZevaSkin™, more than a dozen patients at the two QTCs that have been activated, and nearly three dozen patients from the referring community, just from 10 physicians, 10 referring physicians without even having a sales force in place. That is just a testament to the strong value proposition that ZevaSkin™ has got to offer and that the community is recognizing that. It’s a gene therapy, so obviously reimbursement, and it’s priced at around $3.1 million per treatment.

Obviously reimbursement is a factor, and we are already seeing strong momentum, a positive momentum from insurance companies who are willing to cover, and there is a lot of progress that we are making with regards to reimbursement and coverage for ZevaSkin™. With a demand that is building up and with the strong financial position, I just want to go over some of the launch progress, but before doing that, let’s spend a few minutes about the disease itself and what is ZevaSkin™ and how we differentiate ourselves from the rest of the products. If you look at recessive dystrophic epidermolysis bullosa, it’s a monogenic disease, and the largest organ in a human body is skin because skin protects us. It covers the entire body. It protects us from infection and from the metabolic aspects, regulates the body temperature, and it’s so very vital.

In these patients that have this particular disease, the skin has trouble staying on the body because the protein, the collagen 7 type protein, it acts as a velcro glue between the skin and the body and the anchoring fibrils that bind the skin to the body. In recessive DEB, this is severely compromised because the skin has trouble producing collagen 7 protein because the COL7A1 gene is defected. As a result of it, patients develop large wounds that continue to grow. They become chronic over time and extremely painful. Babies are born with skin missing on their bodies because the process of birthing itself is very traumatic. What ZevaSkin™ does eventually is to help address those large areas of the body.

As you can see, picture speaks a thousand words where the wounds that are there, they tend to become chronic over a period of time, and there is a very high risk of developing cancers, up to 90% risk of developing squamous cell carcinoma, significant infection on the body of these patients. These patients typically have more than 30% of their bodies wounded. These are just some select examples of patients that we have treated in our clinical trials with ZevaSkin™. In addition to the clinical burden, there is a significant economic burden. The cost of bandaging these patients on a weekly basis is enormous. You’re looking at north of $245,000 in annual bandage cost itself for young patients, and the existing treatments that are out in the market can range up to $15 million in the lifetime cost.

Given all of these factors, we are looking for a treatment that can offer coverage for large areas of the body, and that’s really where ZevaSkin™ has differentiated itself because it’s the first and only gene therapy that’s been approved that with a single treatment application, you have the ability to cover large areas of the body, not just small recurring wounds, but massive regions of the body. With the one-time application, provide multiple years of healing and pain relief. There used to be a debate as to what is the role of ZevaSkin™ that’s going to play when you’ve got existing products like Vijuex and Fulzuvist on the market. Do we really have a place? Right now, there’s not so much of a debate because you look at those types of images that you see in these recessive DEB patients.

There is a need for multiple treatment options, both topical gels as well as ZevaSkin™, which can cover significant areas. The process involves really taking two punch biopsies of the patients to 0.8 centimeters, and it’s a manufacturing process of 25 days where you can produce up to 12 ZevaSkin™ sheets. Each sheet is the size of a credit card, and you have then the ability to be able to treat either large contiguous areas that can be joined together and put in a tile-like fashion, as you can see here in this image, or put it in various parts of the body that are where the wounds can be located. With single surgical application, and based on the hospital procedures, you have massive wound coverage areas as well as the treatment duration and the durability that we were able to demonstrate in our clinical trials.

Some before and after examples of ZevaSkin™-treated wounds in our clinical trials. You can see that, needless to say, the picture speaks a thousand words, but here the observation of various anatomic body areas, whether it’s the thigh, the top left, the shin, the anatomic areas of the right arms and the legs, and the torsos. We have shown successful application of the product, and this is just six months after the treatment where there has been wound healing as well as pain reduction that was shown for all of the patients that we have treated. The identified patients in our current commercial setting, oftentimes we’ve gotten the questions that these are the types of patients that are being identified where physicians are looking at patients who have large wound areas and wounds that have never closed.

Those are the types of patients that are currently being prioritized in the commercial setting, and currently we are in the process of placing those patients on the treatment. In phase three VITAL study, we had shown both wound healing and pain reduction. There is no other, again, treatment option that has shown both co-primary endpoints of not just wound healing but also pain reduction. 81% of the treated wounds showed more than 50% wound healing at six months compared to 16% of the control wounds that were intra-patient randomized, as well as a mean pain reduction of more than threefold between the treated arm and the control arm. These wounds that were chosen in our clinical trials were the toughest to heal.

They were large wounds that had remained open for more than six months on a median of five years, and some wounds that had never closed for even 21 years. We had chosen the toughest to treat wounds, but in the commercial setting and right now FDA-approved setting, the label is for regardless of the wound sizes and the wound chronicity, and those are really the types of patients that we are currently in the process of bringing them on. Beyond the vital study, which was a phase three randomized clinical trial, we also had in phase one 2A with a single treatment application, wound healing demonstrated for multiple years. Here you can see the examples of a back wound of a patient with a median follow-up of 6.9 years and wounds followed for as long as eight years.

That’s massive because these types of wounds, especially on the back of the wound, back of the body where there are very high friction areas and that severely limits their ability to move, to be able to demonstrate with a single surgical application is obviously a big deal for these patients. Patients who have gone through our clinical trials are demonstrating these kinds of outcomes and are showing their present-day images having treated almost seven years after the treatment application. We have shown that the impact that we are seeing is because of the sustained collagen 7 production of the treated areas.

These are the punch biopsies that we had taken after the application of the product that with a single treatment, you are able to show the formation of anchoring fibrils and the collagen 7 production, which leads to a continuous, it’s almost like putting a collagen factory, protein factory on your body because of an ability to produce constant anchoring fibrils leading to sustained wound healing. What’s the process like in terms of the application right from the start of a patient intake in a commercial setting to application of the product and discharge? At the moment, we are seeing this to be about a three to four-month process from the initial consult to the treatment. The first step is the initial consult of the patient, sorry, initial consult that happens. Before a patient biopsy happens, there are multiple steps that need to take place, including insurance clearance.

There’s a payer prior auth. After that, after a clinical prior auth is in place, then there is a financial agreement that needs to happen between the hospital site and the payer. After that, the patient is scheduled for a biopsy. That process overall right now is taking three to four months, and we expect this process to get better over time. Right now, it’s the initial stages of clearance and getting from step one to step two. Once you have biopsied a patient, and we have indicated we have biopsied a patient already, despite all of these long lead times, it’s a 25-day process to go from biopsy to treating a patient. Despite all of these steps, we’ve had numerous patients who have gone through this procedure more than once and have stepped forward to be a part of our Strong Together network.

These are our clinical trial patients who have seen the benefit of ZevaSkin™ in the real world and have elected to come back again to treat different parts of their body through the clinical trial process and are stepping forward to talk to remaining patients out in the community. This is the part of the Strong Together network of both patients and caregivers. As we speak, we have had numerous meetings, regional meetings, patient conferences where some of these patients have stepped forward to share what their stories are because there are a lot of patients out in the community. As I mentioned, there are more than three dozen that we have identified. Oftentimes, a question we get is, are patients really willing to travel these far distances?

This is an example of a claims analysis that we had done before these products were approved to showcase that the patients already travel significant distances for specialized care because in the status quo for infections, to treat their infections, to treat their hand surgeries, squamous cell carcinoma, and complex procedures, patients travel multiple far-off distances. Nearly 40% of the patients at these 23 centers of excellence that we have tend to come from out of state. The 70% of the referrals that are coming from out of state are coming from 300 to 400 miles away, and 30% of them are traveling from nearly 1,000 miles. When we talk to these patients, they express much greater willingness to travel for a one-time application, these kinds of significant distances to the qualified treatment centers because they see the benefit and the impact that it can have.

To support these kinds of travels, we have in place patient support programs, Abeona Assist™, which is our patient support program, ready to offer them travel support for all the qualified patients, as well as a hotel and logistics stay to make it easier for these patients because the patients have to travel for biopsy as well as for the actual surgery itself. We are in the process of activating centers. ZevaSkin™ can be administered only at qualified treatment centers. As we mentioned, we’ve announced the opening of two centers. One is Ann & Robert H. Lurie Children’s Hospital of Chicago and Stanford Medicine Children’s Health in California. Both of these very reputed institutions, they’ve got infrastructure, they’ve got patients that they care under in their own treatment paradigm. In addition, these are the centers that patients have trusted and they traveled in the past.

We are in the process of activating additional centers. Even though we are already seeing a significant demand with the 50 patients that we have identified, the work is pretty much cut out for us to get these 50 patients treated at these centers. It’s going to take us several months and well into 2026, given where we are with our supply and we are in the process of ramping up our manufacturing capacity. For us to clear that kind of a demand, and that demand is just growing and that demand is just building because as patients get identified, as these hospitals have more EB clinics, they are coming in contact with new patients. As those patient consults happen, we expect this demand to grow while we in parallel ramp our manufacturing capacity to be able to treat these patients. These two treatment centers are already positioning as well.

As in how we have additional treatment centers, those new treatment centers have their own pool of patients that they tend to follow on a periodic basis. Those additional treatment volume we have not even counted in our 50 count that we’ve talked about. We are looking pretty strong right now with regards to demand standpoint. It’s a matter of getting these patients queued up and going through the treatment process. It’s a significant opportunity at a macro level if you look at it just from a near-term optics to at a big picture level. Based on the opportunity at hand, we have about 1,300 dystrophic EB patients in the U.S. based on our claims analyses. Of the 1,300 patients, we estimate about 750 patients that are ZevaSkin™ eligible patients because these patients have moderate to severe wound areas on their body.

Given the clinical burden of disease, we expect that an average patient will require two treatment cycles during their disease journey to cover their impacted areas. So 750 patients times two treatment cycles, you’re looking at about 1,500 treatment opportunities. The price we have is for each treatment of ZevaSkin™. That’s about 1,500 times $3 million per treatment. You’re looking north of $4 billion in cumulative revenue for ZevaSkin™. That’s a sizable opportunity and we are encouraged by the payer coverage for this particular product. That’s kudos to the payers. For the last year, year and a half, we have been discussing with payers across national, regional plans as well as many other plans, and they are recognizing the value proposition that ZevaSkin™ has got to offer.

If we continue on this trajectory with the fiscal discipline, we expect to be profitable by treating north of three patients in a month. That’s putting us on the right track from a financial and profitability standpoint. A little bit on the payer front. As I said, we continue to engage with payers across all the channels. The total payer mix at a macro level, you’ve got about 60% of recessive DEB lives which are commercially insured, 30% are Medicaid, and about 10% are Medicare covered. Medicare not because they are 65 and older, but because they are on lifelong disability. So far, we have seen positive coverage for ZevaSkin™ across national and regional plans. We mentioned on our previous call, UnitedHealth issuing a policy to FDA label with no additional restrictions.

There’s no restrictions on the number of treatments, so a patient can get more than one treatment and there’s still coverage that’s being determined. There’s no restriction on the wound size or age or the chronicity of the wounds, which is again very favorable. Since then, Cigna also has come out with a policy similar to label. Anthem is covering it as a medically necessary product. There’s a good trend that’s already building up. To date, all of the patients that are currently in the queue, about four patients that we mentioned on the call that have submitted prior auth, have gotten prior auth approval, some within 48 hours, including Medicaid patients. That’s a pretty strong initial, again, indicators.

On the Medicaid side, which is about 30% of our business projected, we have already executed an agreement with CMS, the National Drug Rebate Agreement, which is the first step to initiate an expedited coverage from Medicaid individual states. Individual Medicaid states have also started issuing policy. Finally, on the Medicare side, as earlier announced, we have secured MSGDRG 18, which is the highest DRG payment for gene therapies, cell and gene therapies on the Medicare side. As the demand continues to build, as the qualified centers are coming on board, and the payer coverage continues to look promising, we’re also equally ramping up or in the process of ramping up our supply capacity. We currently are at a position to treat four patients a month, and our goal is to ramp that manufacturing capacity. We will plan to take a shutdown in the month of December, as indicated earlier.

It’s a GMP requirement for any cell and gene therapy. That’s what we’ll plan to do in the month of December. We are currently in the process of ramping up as well as thinking about long-term expansion for our capacity because right now demand certainly is exceeding our supply ability. We continue to focus again with patient support program in place. We have patients who have gone through our clinical trials who are stepping forward and are exchanging with other patients in the community through the Strong Together Network. We have a lot of educational materials on our website. We are focused on delivering really exceptional treatment experience for these patients. As more patients get treated at the QTCs, we have heard the centers then opening up to additional patients within their own treatment base.

We are in the process of getting that execution through the initial patients and building that positive momentum. Beyond ZevaSkin™, ZevaSkin™ in and of itself, there is opportunity to grow. If you look at our pipeline, beyond the current execution, we also have in-house programs of AAV-based gene therapies for inherited retinal diseases and partnered programs with Ultragenyx and Antasia. Just to wrap up, again, a significant level of interest for ZevaSkin™ out of the gates from the RDEB community. Two treatment centers, we’ve identified a sufficient number of patients, and we are in the process of getting 50 patients, 12, more than a dozen in the QTCs. We are in the process of getting them through treatment. $226 million in cash as of June 30th of 2025. That gives us more than two years of operating runway without accounting for revenue of the product.

We are projected profitability in the first half of 2026. The way we are progressing, we’ll continue to update you more as we make progress. Thanks again for giving us this opportunity.

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