Abeona Therapeutics at Leerink’s Global Healthcare Conference: Launch Plans for PZcell

On Monday, 10 March 2025, Abeona Therapeutics (NASDAQ: ABEO) presented at Leerink’s Global Healthcare Conference 2025, outlining its strategic plans for the commercial launch of PZcell. The company is gearing up for a significant milestone with the anticipated PDUFA date for PZcell on April 29th. While the outlook is positive, challenges such as initial supply limitations were acknowledged.

Key Takeaways

• Abeona is preparing for the launch of PZcell, targeting a PDUFA date of April 29th.
• The company projects a total addressable market exceeding $2 billion for PZcell.
• Financial stability is secured into 2026, even without PZcell sales.
• Manufacturing capacity aims to increase to 10 patients per month by early 2026.
• Abeona is considering expanding PZcell’s indication to other DEB-related conditions.

Financial Results

• Abeona is financially stable into 2026, excluding potential PRV proceeds or PZcell sales.
• Gross margins are expected to reach 85%-90% due to in-house manufacturing efficiencies.
• Profitability is projected with the treatment of 3-5 patients monthly.
• The company anticipates receiving a priority review voucher upon PZcell’s approval.
• PZcell’s pricing will align with similar gene therapies, starting at $1.5 million.

Operational Updates

• A seasoned commercial team is in place to support the launch of PZcell.
• Engagement with five centers of excellence is ongoing, aiming for readiness by end of 2025.
• Initial manufacturing capacity at launch will support four patients monthly, with plans to expand.
• Manufacturing turnaround time for PZcell is set at 24 days.

Future Outlook

• Abeona plans to explore global expansion opportunities post U.S. launch.
• Updates on other AAV-based gene therapy platforms are expected in future quarters.

Q&A Highlights

• Approximately 750 patients in the U.S. are estimated to be eligible for PZcell treatment.
• Expansion to other DEB-related conditions is under consideration.
• All CMC items noted in the CRL have been addressed, paving the way for approval.

For further details, refer to the full transcript below.

Full transcript - Leerink’s Global Healthcare Conference 2025:

Lily Nesongo, Leerink Analyst, Leerink: All right. Welcome back to the Leerink twenty twenty five Global Healthcare Conference. So I’m Lily Nesongo within the genetic medicine team here at Leerink. And today, we have the pleasure of having the Abeona management team with us, and that includes Vish, CEO of Abeona. And we also have Madhav, who is the CCO as well as the Head of Business Development.

The floor is yours.

Vish Seshadri, President and CEO, Abeona Therapeutics: Thank you so much, Lily, and thanks for having us here. I’m Vish Seshadri, and the President and CEO of Aviono Therapeutics. And it’s my pleasure to talk on behalf of our company. Joining us, Doctor. Madhav Vasanthavada, who is the Chief Commercial Officer.

Avionna Therapeutics is a cell and gene therapy company based out of Cleveland, Ohio. And if you look at our there’ll be a lot of statements that I’ll be making through this presentation that are forward looking in nature. It’ll contain words like may, anticipate and expect. And please keep in mind that our business is biotech business, as usual, is wrought with these types of risks. So with that, I just wanted to introduce what our core capabilities are.

So as a cell and gene therapy company, we have capabilities in two different types of platforms. One is the ex vivo autologous cell therapy platform, which is where we have our lead asset, Pradimagen zamicarasil or PZcell. Most of the talk today is going to focus on this asset because this is the one that is most mature and we have a PDUFA date with the BLA review that is ongoing April twenty ninth of this year. So it’s less than two months away. We’re all very excited.

This is our first anticipated commercial launch. And this product, we have a rare pediatric designation and we anticipate a priority review voucher with it. And the second platform that we have is AAV based in vivo gene therapies. And we have assets in the preclinical stage of development for monogenic ophthalmic diseases. I’ll touch on that a little bit.

But the most, you know, the most advanced one there is X linked retinoschisis in the RS one gene. And other than our fully owned programs that I just mentioned, we also have out licensed gene therapies. One is UX 111, which is an MPS3A out licensed product for to Ultragenyx. And there is a PDUFA date of August 18 for that asset as well. So very exciting to see our assets reach these milestones and get to patients quickly.

And we also have another out licensed program. Our Rett Syndrome program is with Taysha Therapeutics. And we’re seeing very compelling early clinical data for that asset. Now moving on to our lead asset, which is pzcell. This is a product for patients with a disease called recessive dystrophic epidermolysis bullosa, or RDEB, in short.

This is a very rare, ultra rare and debilitating connective tissue disorder. Patients with this disease do not have functional collagen seven, which is encoded by the COL seven A one gene. And the product of this gene, really what it does is, it’s the anchoring mechanism by which the outer layer of the skin is well crowed onto the inner layer of skin. That’s how the intactness and integrity of the skin is maintained. In the absence of that function, skin blisters easily and forms wounds.

These patients are covered in large wounds that are quite life threatening because over time, they become very adamant and tough to treat. They do not self close. And these are the types of wounds that typically become chronic and then lead to cancers like squamous cell carcinoma over the lifetime of these patients. And, these patients very rarely see their forties and fifties of age. So that’s, that, that gives you the gravity of this disease.

And they’re constantly in need of wound dressing changes. Two to three times a week, they have to be bandaged again and again, and spend about six, seven hours every setting in every, you know, caring for their wounds. And, from a humanistic as well as economic perspective, there’s a lot of burden on these patients and their families. And what we have with PZcell is to address some of the still outstanding unmet needs for these patients, which is to provide multi year durable wound healing and also relief from their pain and itch. And, hopefully, if you address wound healing, that will reduce the risk of squamous cell carcinoma.

So that is really what we’re all trying to solve for for these patients. And, our first product, PZcell, or Pradimogene, Zami Caracel, is uniquely positioned to address many of these unmet needs that I just articulated. I’ll describe the product in a little bit. But before going there, the unique and differentiated factors of or properties of PZcell is, delivering long lasting wound healing as well as pain reduction for especially large and chronic wounds for ARDA patients for years following a one time therapy. And, as I mentioned, these wounds become chronic over time, which means that they do not have the property of healing by themselves.

So the studies that we have put, PZcell, we have investigated PZcell have the toughest to treat wounds in these studies, and we’re very happy to see that long lasting wound reduction wound healing accompanied by pain reduction and itch relief is something that we’ve been able to see in our Phase one to eight clinical studies as well as the Phase three study that has been completed to date. So, with that, I’m just going to talk about the pivotal study, that investigated p z cell in clinical trials. And this is unique in many ways. It’s an intra patient controlled randomized study where every treated wound with p z cell is paired with an untreated wound. And, we’re looking at co primary endpoints that straddle both in terms of physician assessed wound healing as well as patient reported pain reduction.

So it’s a very unique kind of a endpoint construct because usually you have, you don’t see patient reported outcomes in a primary endpoint for clinical studies. And 43 wound pairs across 11 patients were the primary dataset. And across both wound healing and pain reduction, we saw statistically significant results. And wound healing was studied. All of those endpoints were measured at six months post treatment.

Although we see treatment effect as early as six weeks post treatment, the primary measurement for the regulatory path was a six month time point, which is very standard for these types of assets. The first co primary endpoint, as I mentioned, you can see eighty one percent versus sixteen percent, very significant wound healing was observed at six months, and also pain reduction where patient reported, Wong Baker pain scale. That’s what was used. It’s a validated tool, and you can see that there’s a significant effect there as well. Picture speaks a thousand words.

Just to add a little bit more color and data to that, When we talked about wound healing, we are measuring wound healing at three different levels. What is the proportion of wounds treated with PZcell show at least fifty percent wound healing, which is fifty percent of the area that was treated, and seventy five percent or better, and then hundred percent. Right? So we have very stringent criteria for what would qualify as a hundred percent healed wound. I just wanted to give you a little bit of more detail on that.

So here you see a large thigh wound on in this picture, and there are three wound areas within that large wound that’s just designated because the sheets of pz cell come as 40 centimeters squared sheets, which are about the size of a credit card each. So in large contiguous wounds, you can see that we we can apply p z cell, more than one sheet in that large area. So you see three wounds here before and after at week twenty four, and you can see that, the wounds, show healing. All these three wounds are scored as greater than seventy five percent wound healing. They are not scored as 100% healed.

And the reason is you see some yellow crusting that’s, that’s visible on these wounds. You cannot pick on those crusts and verify if the skin underneath is healed or not. So the physician will give it a 75% plus and not a % wound healing. That is important context for, what would qualify for a % healed wound. Here you see an example where the bottom wound is scored as a % heal wound, whereas the top wound is, scored as a 75% plus because there is a small red speck there in the bottom right.

And, absent that closure, it’s scored as greater than 75%. Here, this example is an upper trunk. Here, there’s another example that you see upper back. They’re both scored as b three is a treated wound. It’s scored as a greater than seventy five percent wound heal.

Again, that’s because of the crusting. Another example of the left flank, you can see examples both of greater than seventy five percent heal and hundred percent heal wounds. Just to give you the idea that there are so many different anatomical areas that have been investigated in our pivotal study for wound healing. There was also prior to the pivotal study, there was a phase one to a study that was done. This was a Stanford investigator led study.

We have published the results, in 2022 with eight years of max follow-up and five point nine years of mean follow-up for the study. And you can see both fifty percent healed status as well as proportion that are greater than or equal to seventy five percent healed reported in the blue and green, histograms on the left. And you can see that wound healing is sustained after a one time application over years. And the pain reduction of these wounds here, the pain reduction was not measured on a Wong Baker scale, but it was a binary yes or a no, for a pain associated with the wound. And you can see that also significantly reducing and staying that way over time.

And that correlates with the wound healing status as well. So this is this is really the durability that we’ve studied. And some of these wounds that were treated in the, Phase one to a study were open for an average of like eleven years, which speaks to the chronicity of these wounds. They do not have the ability to self heal. So that’s very important.

It was also evident with the fact that if you looked at our pivotal study results, the completely healed wounds was zero. You don’t have any control wounds that showed complete wound healing. That’s another distinguishing feature that highlights what types of wounds we’ve really treated. Here’s another example from the phase one to a study, back wound treated with, PC cell sheets, upper back. And you can see what it looks like here at year two and also year five.

And the question always arises, why do you have this kind of durability of, PC cell treatment? And when, you know, we will we are doing some studies to look at progenitor phenotype cells, but at the molecular molecular level, the way the gene is introduced to the patient’s cells is through retroviral transduction. What that does is cause stable integration of the transduced COL seven a one gene, the genome of these cells. And as these cells divide and propagate, they are going to be maintained. So you don’t lose this gene as, over time.

That is really the reason for long term, gene expression. And what you see here is a biopsy of a patient treated with p z cell over various time points going all the way to year two. And you can see, even at year two, the green lines that you see in the bottom that is looking for COL7A1 expression. So that’s that’s kind of nice correlation to see continuous expression of collagen seven even two years post application of pZcell. The safety profile of PZcell is also very favorable.

We see across twenty one, PZcell patients, one hundred and twenty wounds treated. We do not have any serious adverse events that is treatment related, which is, which is an excellent safety profile in itself. And we’ve also paid specific attention to squamous cell carcinomas because squamous cell carcinomas are common in these patients. The cumulative risk of having a squamous cell carcinoma by the age of 55 is more than ninety five percent in these patients. So we’re almost definitely getting squamous cell carcinoma over that period of time.

And you can see that in our treated sites, we have not seen any occurrence of squamous cell carcinomas. And we also routinely check for replication competent retroviruses because our technology uses a retroviral vector, and we want to make sure that we haven’t introduced any replication competent retrovirus, and we’ve never seen such instances in any of these patients over more than 120 sample points. So with that, I’m going to turn this over to Madhav to take us through our commercial opportunity and preparations for launch.

Madhav Vasanthavada, Chief Commercial Officer, Abeona Therapeutics: Thank you, Vish. So as we get closer, we are getting excited and getting ready by the prospect of being able to treat many more patients, recessive death patients. We have done our homework with regards to finding out how many patients there are in terms of the prevalence pool. And based on our analysis of the claims dataset, we estimate there are about seven fifty patients in The US that are PZ cell eligible. There are about thirteen hundred dystrophic EB patients, seven fifty patients that are PZ cell eligible.

These are severe to moderately severe r dead patients. And based on the data that we shared and the natural history study where these patients have on average about thirty percent of their body is wounded, thirty percent. Each 1% of the body is roughly the size of the palm of the recessive dead patients. So that’s a significant area of the body that is wounded. We estimate a patient requiring roughly two treatments for their lifetime.

Could be more pediatric patients could be less, but two on average. So that equates roughly seven fifty patients time two treatments. So you’re talking about fifteen hundred north of 1,500 treatment opportunities. From a pricing standpoint, we have communicated, this is going to be priced similar to one time recently launched one time gene therapies. What we have said is a floor price of $1,500,000 It’s going to be north of that, but at least $1,500,000 So 1,500 treatment opportunities times $1,500,000 per treatment.

You’re talking about a total addressable market of north of $2,000,000,000 right? So even if you think that we’re going to get 50% of that that we accomplished, we’re talking about $1,000,000,000 at least, which is a big gap versus where we are right now, Aviona, as a company from a valuation standpoint. From a gross margin standpoint, we think it’s going to be 85%, ninety % at a steady state when we are able to manufacture consistently. And that is primarily because manufacturing for PZcell happens within Aviona. We are not relying on a third party to make the vector.

We make it ourselves in Cleveland, Ohio. And so based on our pricing potential and our COGS plus the sales general expenses, we think about three to five patients per month. We will be on a path to profitability. So that’s really where we are looking from a total opportunity standpoint. So the question is, how are we approaching?

What are we planning to do? With regards to our engagement, first off, the commercial commercialization team is in place. We’ve got a really good group of people experienced in having launched prior cell and gene therapies. We are very much in active discussions with, centers of excellence. There are about five centers of excellence that we look to onboard and make them patient ready by the end of this year, by the launch year, which is 2025.

And, what we hear from these centers that if they are able to treat one or two patients a month, that’s pretty sufficient for our capacity as to where we are right now. And so these are really good and long conversations we’ve been having with these centers, which involves usually the leadership within these institutions. We’ve had kickoff meetings with these centers, having visited them in person in some of these centers to understand how the patient flow is going to happen, how the product is going to be received, and all of the administrative work, as well as training these centers to be able to treat with with with the product. And the reason to believe that this is going to be met with is, success is, the enthusiasm that we hear not just from, the physicians and the payers, but also from the patients themselves. The feedback that really resonates here is the durability of the treatment.

Oftentimes, these patients that require, continuous application of other treatments are potential to be able to apply, single, although surgical application, it is a procedure. It’s a process that the patients have to go through. But the outcome and the durability of the outcome is quite, enthusiastic, feedback. That’s really what we hear from them. Also the excitement from the referring physician because our strategy is to get about five centers ready to treat.

And then we are also calling on and we will be educating other centers. There are about, 23 total centers of excellence within The US that treat recessive death patients. So the rest of the centers that are not the qualified treatment centers will be asking them to be able to refer their patients over to the QTCs for treatment. Along with the sites onboarding, we are in active discussions with various payers. The overall payer mix for RDEB, a little over 50% are commercially covered lives.

About 30% are Medicaid, and the rest is Medicare patients. Medicare, not because they are 65 and older, but because they are under lifelong disability. And the feedback that we hear from commercial payers is, is very encouraging. We’ve had more than 40, clinical presentations, to national payers, regional payers. And, and we are currently under, confidential CDA discussions with them discussing what is the price range and, you know, what the coverage status would look like.

And overall, given the ultra rare nature of the disease, the durability of the product and the ability to cover large areas of the body in one surgical application, is clearly, compelling, for these payers. Post approval, we will engage also with Medicaid. We’ll plan to apply for the Medicaid drug rebate application, and that will also incentivize some of these states to carve out the product from the bundle payment, which will potentially could make the reimbursement, favorable for these sites. As we do that, we are also in the process of ramping up our manufacturing capacity. We’ve always been communicating that this is going to be a supply gated, uptake.

And especially in the outer years, we look to ramp up significantly. I think in this year, 2025, we should expect the sites to, treat, one or two patients and get the confidence from a reimbursement and the payer flow. And as these sites begin to feel comfortable, then in 2026 onwards, we should expect a greater uptake of the product. And that syncs up very nicely with the planned ramp up of the manufacturing capacity. At launch, we will have, capacity to treat four patients a month, roughly just one a week.

And our goal will be to get to treat 10 patients a month, in first quarter or so of twenty twenty six. Now, of course, you know, like any other autologous process, there’s going to be certain requirements to shut down and maintenance of the manufacturing plant, so on, so forth that will need to be in place. So even with 10 patients a month kind of a capacity, we are looking around eighty, ninety plus patients, our ability to treat, once we are fully at steady state of manufacturing. And we will be supplying, with all of the resources we have to make sure that the patient journey is as seamless as possible. As as Wish explained earlier, the beauty of our product is that we are actually using patients’ own starting materials, and we are gene modifying the keratinocyte cells.

And in exchange, we are giving them, functional version of their own skin that has the ability to produce collagen. So with that kind of, process, it it involves, an intake process. There is a biopsy that is, required to collect. The biopsy is collected in a typically in an outpatient setting, in a hospital where which is a qualified treatment center. The manufacturing happens in Cleveland, Ohio.

It’s a twenty four day turnaround time. During that time, the patient is back at their own care at their home and residence. And then the patient comes back in for the surgical procedure, which the surgical procedure takes typically for three to four hours depending on how many sheets sheets that we have supplied in exchange And then, the patient stays in the hospital for about a week, to allow the wounds or the product to get taken up and then is discharged So this is a one time process. It is, it is a it is a process to get the product, but the outcome that what we are hearing from the patients, we’ve had patients who have gone through our clinical trials and have come back. We’ve had six patients who have come back again to go through this process to treat other parts of the body that were not previously treated.

We’ve had one patient who has come back three times to treat different areas of their body. So to us, those choices that these patients are making and these centers are making is quite telling in and of itself. And, before I give it back to Vish, like I said, we’ve gone through cellular therapy products before. Both Vish and I, as well as many members on our team, have launched CAR T cell therapies, and we understand what it takes. It takes a village to be able to get these patients to treatment.

But that’s really the beauty of these kinds of durable, long lasting treatment options where there is a high willingness to pay. We are also there to support these patients through their co pay assistance programs. We’ll have a patient navigator in place who is going to take care not only on the phone but even in at the site to be able to handle the logistics. The reimbursement support, the transportation support, and whatever it takes to get these patients the treatment because we know that it is going to be a game changing for them. So with that, Vish, back to you.

Vish Seshadri, President and CEO, Abeona Therapeutics: Thank you, Madhav. In closing, I just wanted to also add a note on our financial health. Without any accounting for any PRV proceeds or sales from PZcell this year, we’re still funded into 2026 with the capital that we have currently. So that’s a very healthy place to be in itself. And Madhav talked about capacity.

If you look at PZ cell capacity, even at three to five patients a month, this is going to be a profitable business. So we feel very confident that with the current cash and having a PRV in hand, sales starting from the later quarter three, quarter ’4 this year, we should this gives us a bridge to profitability for Abeona. We didn’t talk very much about our other platforms, but we do have goals for our AAV platforms, the first asset to get to clinic next year. But you will see that in the coming quarters and other updates, we’re going to talk more about those programs. We’ll be very excited.

We just want to, you know, pulse it as we get through important milestones with PV cell first. Thank you. Any questions? Yes. We have addressed all the CMC items that were noted in the CRL.

We have, had many meetings with the FDA before we made the resubmission. So we we feel we’re in a good place. Of course, the review is still ongoing, so I cannot say it’s done. But from our perspective, we are not negotiating that we’re going to give you 60% or 70%. We’re doing all those things that they asked for.

Lily Nesongo, Leerink Analyst, Leerink: I guess in the time we’ve left, maybe I’ll ask a few questions. The first one being, so you’ve mentioned in The U. S, in terms of patients that will be eligible, you estimate that it’s seven fifty patients. Are there

Vish Seshadri, President and CEO, Abeona Therapeutics: Yeah. That’s a great question. We’re looking at a lot of still unmet needs. For one one example is, of course, when you look at patient populations, we are focused on RDEB. Right?

I think, there is definitely DEB as a disease we are going to investigate, if we can help with PCcell. Some of these patients have, pseudosyndactylies. Right? So their digits fuse and then they undergo hand surgeries. So they have to undergo hand surgeries to separate the digits.

And then over a period of one or two years, you start they they start fusing again and they may need additional surgeries after that. So we will investigate if p z cell can be a potential, you know, solution to keep those digits separated. So these are some of the ideas in the RDEB related disease or on where we could potentially take our therapy to expand.

Lily Nesongo, Leerink Analyst, Leerink: Okay. And I guess in the last minute we have, how should we think about timing of global expansion beyond The U. S?

Vish Seshadri, President and CEO, Abeona Therapeutics: We have had a lot of interest come in, inbound interest on from, you know, third parties in Europe, in taking this technology ex U. S. We just haven’t had the bandwidth as of yet, but this is an important goal for us, whether it’s through a CDMO or through a partner. Once we get The U. S.

Launch underway, we’re going to be entertaining and exploring those avenues as well. So we should have future updates on that.

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