Abeona Therapeutics (NASDAQ:ABEO), a biopharmaceutical company, provided updates on its financial status and the development of its lead gene therapy product, Pradimogene zamykerathal (PZ cell), during its third-quarter earnings call of 2024. The company reported a net loss of $30.3 million for the quarter, with a significant portion stemming from warrant liability remeasurement. Despite the loss, Abeona maintains a strong cash position with $110 million in reserves, which is estimated to fund operations into 2026. Regulatory progress was highlighted with the resubmission of the Biologics License Application for PZ cell and its acceptance by the FDA, setting a PDUFA date for April 2025. The company is preparing for commercial launch with five qualified treatment centers and is exploring pricing strategies for the one-time gene therapy.
Key Takeaways
- Abeona Therapeutics reported a net loss of $30.3 million in Q3 2024, with research and development expenses totaling $8.9 million.
- The company's cash reserves are projected to support its activities into 2026.
- The FDA has set a PDUFA date of April 29, 2025, for the company's lead product candidate, PZ cell.
- Abeona is advancing commercial preparations, including establishing treatment centers and a commercialization team.
- Manufacturing capacity is set for 10 patient runs per month, with plans for expansion due to anticipated demand.
Company Outlook
- Cash runway estimated to last into 2026, allowing continued investment in product development and commercial preparations.
- Two new patents issued, enhancing the company's intellectual property portfolio and protection until 2037 and 2040.
Bearish Highlights
- The company experienced a significant net loss, including a $15.2 million loss from warrant liability remeasurement.
Bullish Highlights
- No additional clinical trials or data requested by the FDA for PZ cell, indicating a smoother regulatory path.
- Positive outlook on the unique positioning of PZ cell for wound healing and pain reduction in RDEB patients.
Misses
- The company's financial performance was impacted by losses, particularly from warrant liability remeasurement.
Q&A Highlights
- CEO Vish Seshadri expressed confidence in PZ cell's potential as the first gene therapy using keratinocyte sheets for RDEB treatment.
- Chief Commercial Officer Madhav Vasanthavada emphasized the company's commitment to patient access and payer engagement for the pricing strategy.
Abeona Therapeutics (Ticker: ABEO) remains focused on advancing its gene therapy product, PZ cell, with a strong emphasis on regulatory success and commercial strategy. The company's financial health, with a substantial cash reserve, positions it to pursue its goals into the next couple of years. As the PDUFA date approaches, Abeona continues to engage with payers to ensure accessibility and reimbursement for what could be a pioneering treatment for RDEB patients.
Full transcript - Abeona Therapeutics Inc (ABEO) Q3 2024:
Jenny, Conference Call Operator: Good morning, and welcome to the Abeona Therapeutics Third Quarter 2024 Conference Call. At this time, all participants are in a listen only mode and we will open for questions following the presentation. Please note this conference is being recorded. I will now turn the conference over to your host, Greg Jin, VP of Investor Relations. Greg, the floor is yours.
Greg Jin, VP of Investor Relations, Abeona Therapeutics: Thank you, Jenny. Good morning, everyone, and thank you for joining us on our Q3 2024 conference call. During this call, we'll refer to the press release issued this morning announcing the Q3 results, which is available on our corporate website at www.abionatherapeutics.com. I would like to note that remarks made during today's call may contain projections and forward looking statements. Forward looking statements 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, and 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 sections in our Form 10 ks and periodic reports filed with the SEC. These documents are available on our website at www.abionatherapeutics.com. On the call today with prepared remarks are Doctor. Vish Seshadri, Chief Executive Officer Doctor.
Madhu Visanthavada, Chief Commercial Officer and Head of Business Development and Joe Vizzano, Chief Financial Officer. Also joining us for the Q and A session will be Doctor. Brian Kemeny, Chief Technical Officer. And with that, I will now turn the call over to Vish Sisadri to lead us off. Vish?
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: Thank you, Greg. We appreciate everybody joining this call this morning. We have continued to be productive since our last call and have made progress on multiple fronts toward bringing our lead product candidate, Pradimogene zamykerathal or PZ cell to patients with recessive dystrophic epidermolysis bullosa or RDEB. These patients continue to seek durable treatment options for their wounds. I'll start with the regulatory progress first.
Since receiving the Complete Response Letter, CRL, in April, we have worked closely with the FDA in preparing the resubmission of our PV cells biologics license application and getting it back in the review process. As a reminder, the CRL highlighted the need for additional CMC data pertaining to the validation of certain manufacturing and release testing methods. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA and the FDA did not request any new clinical trials or clinical data to support the approval of P g Cell. In
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: August,
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: we had a Type A meeting where we aligned with the FDA on the content of the resubmission, including additional information to address all the deficiencies noted in BCRL. I'm pleased to report that we completed the resubmission of our BLA for PDcell at the end of October. Just last week, the FDA accepted the resubmission for review and set a PDUFA date of April 29, 2025. Meeting this regulatory milestone in Q4 as per our guidance was made possible by the tireless efforts and determination of our team, especially technical operations quality, regulatory and clinical, and I thank them for their dedication. The FDA's acceptance of our resubmission indicates that our package includes all the necessary data components, including those items identified in the CRL and observation from the pre license inspection to support review of the updated dossier.
The FDA has previously informed us that they do not intend to host an advisory committee or AdCom meeting. In addition, pre license inspections for all clinical sites and our manufacturing facility were completed earlier this year. Therefore, we do not anticipate additional site inspections as part of the resubmission review process. We will continue to work with the FDA to facilitate their timely review. We remain confident in our prospects for approval given the unmet medical need in RDEB to treat large chronic wounds, our strong clinical data, our comprehensiveness in addressing every CRO item and PLI observation and our collaborative interactions with and feedback to date from the FDA.
So now the focus for the rest of this year and into 2025 is obviously to drive through the regulatory process as we continue to ramp up our commercial readiness activities for potential launch next year, which Madhav will elaborate on in a moment. But before that, I'm excited to share that we have recently entered into a lease agreement for additional building space in Cleveland, Ohio, adjacent to our existing facility to enable capacity expansion beyond our current manufacturing footprint. I also want to briefly mention the recent issuance of a new U. S. Patent for the PV cell.
The patent is entitled Gene Therapy for Recessive Dystopic Epidermolysis Bullosa Using Genetically Corrected Autologous Keratinocytes and includes claims that cover the use of PV Cell for treatment of RDEK. The patent has an expiration date of Jan 3, 2037, subject to any applicable patent term extension. Another U. S. Patent will issue on November 19, 2024 relating to the packaging and transport system for PCcell sheets.
As in any patent term adjustment or extension that patent would expire in July 2040. The issuance of these patents by the USPTO acknowledges the groundbreaking nature of the technology underlying pzcell. If approved, pzcell will be the 1st product to use gene corrected keratinocyte sheets to treat RDEB. Based on pzcell's clinical trial experience, we are optimistic that the underlying technology for making pzcell and using it to treat a human patient that is claimed in this patent, which allows stable insertion of this functional collagen 7 gene into the genome of each patient's own skin cells could uniquely position PCcell to achieve wound healing and pain reduction RDEB wounds following a single administration. I'll now turn the call over to our Chief Commercial Officer, Doctor.
Madhav Vasanthavada to highlight our commercial launch preparations. Madhav?
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: Thanks, Vish, and hello, everyone. We are excited about the progress we are making as we continue to prepare for the potential launch of pzcell in the U. S. Next (LON:NXT) year. Let me start off with our progress on building the commercialization team and say that we have attracted a strong and passionate group of people with significant launch experience in the cell and gene therapy space and with deep expertise in market access, clinical site onboarding, cell therapy patient operations, supply chain, patient access, marketing and medical affairs.
At launch, we expect to have approximately 20 members on the PVcell commercialization team, and we feel very confident that this team will deliver a successful pzcell launch. Before going into other aspects of launch preparations, for those new to the PZcell story, let me remind you that PZcell is being developed for the treatment of RDEB, a recessive form of the genetic condition dystrophic epidermolysis bullosa. RDEB is a severe debilitating disease in which nearly 1 third of the patient's body can be covered in wounds. These wounds can be chronic, they can become chronic and do not heal by themselves and are at a high risk for several infections, including cancer called squamous cell carcinoma. Patients rarely live beyond 40 years of age.
There are no treatments on the market today that can heal RDEB wounds durably with a single treatment application. TZcell, if approved, and we are optimistic of its approval, will be the 1st in class autologous gene therapy to have shown game changing benefits to RDEB patients and their families. In clinical trials, tZcell has shown wound healing and pain reduction in large and chronic wounds over multiple years of follow-up after only a single surgical application and with a clean safety profile. We are therefore excited about the impact pzcell will have on RDEB patients and we continue to hear positive feedback about pzcell from physicians, payers, patients and caregivers we have spoken with. Now, in terms of commercialization strategy, our go to market approach is simple.
Upon launch, we intend to have onboarded and trained 5 EV centers that are very well recognized and are geographically dispersed across the U. S. To create the best possible PC cell patient experience. Over time, our focus will be to onboard additional Qualified Treatment Centers, QTCs, while driving patient referrals into the QTCs from other EV treatment centers and community settings. Based on our recently completed claims analysis, we estimate there are about 1300 dystrophic EB patients being treated in the U.
S. Of these patients, we can confirm there are about 750 PCcell eligible U. S. RDEB patients. And we have found that nearly 30% of these 7 50 patients are concentrated at a handful of EB centers and most of these centers we are targeting at launch.
This gives us confidence that we are working with the right treatment centers and are confident in expecting a high demand at launch. We've also learned from claims data that patients travel from far off distances into EV Centers of Excellence or COEs. Roughly 40% of patients currently being treated at the COEs come from out of state. Patients travel for specialized care with catchment areas spanning 300 to 400 miles from the COEs and an average referral distance of 250 to 300 miles. These data points continue to strengthen our confidence in PZ cell demand and our commercialization strategy.
With regards to progress on treatment site onboarding, we have conducted multiple interdisciplinary meetings between Abeona teams and the potential treatment sites, including executing formal in person meetings with members of the senior leadership at the hospitals. These meetings are often large group meetings with members from different functions of the hospitals, including contracting, medical that includes lead ED physicians and surgeons, cell therapy coordinators, pharmacy directors and revenue cycle managers. These sessions are helping us to align on an execution path towards site onboarding in connection with a potential PCell approval. The commitment of time and resources by these centers is a testimony to the significant value that PCcell may bring to RDEB patients if approved. In the coming months, we will continue with pre approval onboarding steps and training to ensure the multidisciplinary teams at each center are educated in all aspects of PD cell treatment and with processes to begin treating patients pending FDA approval.
And similar to other cell therapy launches, we would expect sites to be ready to start treating patients in about 2 to 3 months post approval. Now turning to market access. We are focused on ensuring timely and appropriate patient access upon approval. As a reminder, on payer mix, we believe approximately 60% of ARDA patients are covered by commercial plans, about 30% by Medicaid and the remaining 10% by Medicare. Our market access team continues to engage with the key national and regional commercial payers that represent more than 80% of RDAC commercial lives with a goal to remove barriers to access and ensure appropriate reimbursement for pbcell.
In the coming months, we will also engage with the state's Medicaid programs to support patient access and broaden reimbursement for pzcell. Earlier this August, the Centers For Medicare and Medicaid Services, CMS, granted our request for a product specific procedure code, ICD-ten PCS, which is now in effect for accurate hospital billing and timely reimbursement for PC cell procedures between healthcare facilities and all types of insurance providers, commercial and government. In addition, Medicare has mapped PCcell to a favorable MS DRG-eighteen assignment, recognizing the breakthrough nature of PCcell's technology for RDAP patients and supporting reimbursement and patient access for the relatively smaller pool of Medicare RDEB patients. MS DRG-eighteen is among the highest available inpatient hospital reimbursement levels for cell and gene therapies, and we are thrilled by CMS' favorable consideration. Commercial payers are also recognizing the clinical value of a treatment like pzcell as they review the durability of our clinical trials following a one time treatment application.
They also recognize patients may need to come back for additional treatments to cover their previously untreated wound areas. Based in part on our ongoing PZcell Phase 3b study, where 6 out of 7 patients we have treated so far have received PZcell at least twice, including one patient that has received PZcell three times. The willingness of returning patients to participate in the PC cell clinical trials is a testament to the impact on patients from these PC cell trials. 1 pediatric patient in the Phase 3b study received 12 PC cell sheets in a single application providing substantial coverage spanning 480 centimeters square on their back wounds. These data points are all exciting and give us increasing confidence about the role PCcell could play in treating RDEB wounds upon its potential approval.
With that, I would now like to hand the call over to our Chief Financial Officer, Joe Lozano, to discuss our financial results. Joe?
Joe Vizzano, Chief Financial Officer, Abeona Therapeutics: Thanks, Lana. I would like to remind everyone that you can find additional details on our financial results for the 3 months ended September 30, 2024 in our most recent Form 10 Q, 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 $110,000,000 as of September 30, 2024. This compares to $123,000,000 as of June 30, 2024. 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 PZcell or proceeds from the sale of a priority review voucher or PRV if awarded by the FDA. I'll remind you that pzcell has been granted rare pediatric disease designation by the FDA. So upon its potential approval, we believe that we are eligible to receive a PRV. The last two reported PRV sales were each completed at a value of above $150,000,000 indicating that PRVs could be increasing in value relative to the long time steady average of about 100,000,000 dollars Research and development expenses were $8,900,000 for the 3 months ended September 30, 2024 compared to $7,100,000 for the 3 months ended September 30, 2023. Our spend on general and administrative activities was $6,400,000 for the 3 months ended September 30, 2024 compared to $4,200,000 for the 3 months ended September 30, 2023.
The increase in general and administrative expenses is primarily due to commercial and launch preparation costs. Net loss was $30,300,000 for the Q3 of 2024. It is important to note that the net loss in the Q3 of 2024 included a $15,200,000 loss resulting from the quarterly remeasurement of the fair value of warrant and derivative liability. The warrants are required to be classified as a liability and remeasured at a fair market value each reporting period. Net loss in the Q3 of 2023 was $11,800,000 including a $1,100,000 loss resulting from the quarterly remeasurement of the fair value of warrant liabilities.
With that, I will open the call for Q and A. Operator, can you please open the Q and A session?
Jenny, Conference Call Operator: Thank you very much. At this time, we'll be conducting our question and answer session. Thank you. Your first question is coming from Dae Gon of Stifel. Dae Gon, your line is live.
Dae Gon, Analyst, Stifel: Good morning, guys. Thanks for taking our questions and congrats on all the progress. It's remarkable. Two questions from me. One is for Vish.
As we think about the resubmission and the acceptance, given that the CRL was primarily CMC related, I guess, which aspect at this point, since it is more of a review issue, which aspect do you think kind of keeps you up at night or do you find to be the most kind of question bearing going forward? And second for Madhav, as we think about the commercial rollout, thanks for the insights on your market research there. How should we think about or how are you guys thinking about the rollout strategy as we think Vyjuvek as well as Filsuvaz that's already available. Poly pharmacy wise, what's been your respective comment that you've received from payer discussions and how are you kind of strategizing around getting patients back into the centers? Thanks so much.
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: Thanks, Dae Gon. Good morning. This is Vish. I'll first address your CMC question and then I'll have Madhav talk about the VigelVectin filtulib. So, in terms of the as we had previously reported, the Type A meeting, we were quite comprehensive in discussing pretty much all outstanding CMC issues noted in the CRL as well as the PLI.
And the only ones we didn't discuss was probably because during the informal meeting leading up to the Type A meeting, we already got those pre reviewed, if you may, and kind of got a sense of we are in the right direction there. So in terms of tests or results that the FDA has not yet seen but agreed on, let's say, a protocol for validation, the only outstanding ones were identity testing and seriality, right? Those were the 2 topics. And even there, we had quite deep conversations on exactly what the expectation is. And so for identity testing, we actually had an informal meeting post the Type A meeting and shared a little bit of our preliminary data and we have the confidence based on that discussion that we have what we need to put that package together.
I think sterility is the new data that they're going to see, but in terms of what they would expect, the statistical considerations and all the types of organisms that we have to demonstrate using our new method, these are all things that we had a pretty good agreement about, which is why I feel comfortable about how strong our package is. What I'm going to do is Brian Kemeny, our Chief Technical Officer, is actually online. So Brian, if you would like to add any more color on those two aspects, please do so.
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: Yes. Thanks, Vish, and thanks for the question. Yes, I think you've covered pretty much everything that we are aligning on internally. I think sterility was the biggest topic as part of the CRL. So I think as Vish mentioned, that's one area that we're interested in hearing the additional having them look at our additional data, especially considering that sterility seems to be like a topic that the agency is paying particularly close attention to.
But I can't speak more highly of the data that the team has collected and the package that they put together for the resubmission. So I would echo Mitch's sentiments around our confidence in the package that we put into the agency.
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: Thank you, Brian. Dae Gon, I hope that addressed your first question. I'll turn it over to Magoff to talk a little bit about how VIGUVECT, BILTOVAZ, existing treatment options and how our therapies will play there.
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: Yes. No, thanks, Egon, for the question. Yes, so certainly there are existing treatment options. And yes, patients are receiving treatment options in their home setting. When we present the market based on our market research that we have done, not just with physicians, but also with patients and caregivers, many of the respondents of our market research are on existing treatment options.
And they look at the profile of PD cell, especially the before and after wound images. And the fact that you're able to show how the wounds look like in a finite 6 month period of time has been really moving for them, emotionally moving and they are motivated to consider a treatment like PCcell just given the extent of wound burden that they have. So when a treatment like PZcell were to get approved and come to be offered to these patients, we are hearing testimonies that they will consider this treatment option given the effects that at least clinical trials have shown. Now, as we talk about rollout, because we are targeting high volume EV centers that are not just EV centers, but also clinical researchers from these centers, the fact that we are seeing this engagement, we know that there are patients, at least in our preliminary onboarding conversations as we are beginning to have those discussions. And as patients begin to come to these treatment centers and get their treatments, the referral strategy will kick in as well as other patient to patient and raising awareness through patient advocacy groups and multiple other channels.
I think the clinical benefit of this product is going to speak there. So that's how we feed with regards to keeping the demand going. With regards to the payers and the conversations, we have had no pushback with regards to the payer engagement so far with about PC cell vis a vis other treatment options. And again, that goes to, firstly, it's a rare disease part of it, but also goes to the fact that when you can cover large areas of the body with a single surgical application and multiple years of healing, that data again resonates very strongly with them. So we'll certainly keep you all posted, but so far we feel very confident about the uptake.
Dae Gon, Analyst, Stifel: Sounds good. Look forward to the next progress. Thanks guys.
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: Thanks, Tegan.
Jenny, Conference Call Operator: Thank you. Thank you very much. Your next question is coming from Maury Raycroft of Jefferies. Maury, your line is live.
Maury Raycroft, Analyst, Jefferies: Hi, good morning. Congrats on the progress and thank you for taking my questions. I was wondering if the favorable Medicare reimbursement designation, how that impacts conversations with payers related to commercial reimbursement plans? And can you talk more about payer conversations and what pricing strategy could look like? Maybe remind me if, the first treatment will be a flat price and then subsequent treatments would also be a flat price or variable price per number of sheets or how are you thinking about that?
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: Yes. Thanks, Maury. I think the Medicare question on the reimbursement. So the reimbursement, the MS DRG-eighteen is going to be applied only to the Medicare pool of patients. So the actual nature of reimbursement is not going to spill into the commercial side because the commercially insured patients, we expect will have a single or a case rate arrangement often between the provider side and the payer side.
But the fact that we have a DRG-eighteen is going to is reflected positively by commercial payers because they recognize that this is a sophisticated technology and not any other burn procedure kind of a graft related cost. So the value of the technology, the genetic nature of it has been established with this assignment. So that is 1. The ICD-ten PCS code is what is really positive because that procedure code, which is product specific procedure code, will make it the hospital billing even for the commercially insured patients seamless because now they don't have to rely on a multiple miscellaneous code. So I think that ICD-ten PCS code is going to be is going to help even with commercially insured patients.
With regards to the pricing strategy, our pricing, the way we are thinking about it is a flat price per treatment for the patients. And we expect for the NDC that we have, all available PC cell sheets will be supplied and that will all have one flat price per kit. So up to 12 sheets, one flat price. If the patient were to come back, let's say, later down the line in their lifetime, that will be a separate, its own standalone price point, flat price. We are still discussing with payers with regards to any there won't be any whole WACC price changes between the 1st and the second treatment cycles.
And we also have not had any pushback with regards to any subsequent treatment cycles from the payers. Does that answer Maury or any follow-up?
Maury Raycroft, Analyst, Jefferies: Yes. That's really helpful. And maybe one other question. Just wondering if you have line of sight into how many patients you could treat commercially potentially by the end of 2025, assuming approval and then the 2 to 3 months launching after the approval? And then can you talk more about what needs to be done to make the new facility operational that you're leasing and when that could happen as well?
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: Yes. In terms of how many patients we can expect to treat, I think that is something certainly we will continue to monitor and probably guide as we get closer. What our strategy is to have a centralized location to be able to funnel in all of the patients and get them queued up from a payer coverage and insurance benefit standpoint, because that's going to be a process that we will start soon after we get approval alongside which we will get the centers of excellence, qualified treatment centers activated and make them patient ready, right? So part of it is going to depend on how quickly we can bring the qualified treatment centers and make them patient ready to treat after we get the approval. So that is one.
And then the second is, of course, on the manufacturing side, we are going to ramp up that capacity. We have said and we continue to believe that the demand at the time of launch is going to exceed the supply and that is certainly our focus. As Vish alluded on the call, we have already started thinking about expanding our manufacturing footprint in the building where we are and how to go about that. So, Vishal, if you have anything else to add on that?
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: Yes. Thanks, Madhav, and thanks for that question, Maury. I think it's probably a good idea to remind people that at launch, we're going to have manufacturing capacity. The current manufacturing footprint can take us up to about 10 patient runs or 10 manufacturing runs in a month, but even that will have a certain ramp up speed, not because of the space per se, but because of hiring and training personnel. We're already doing some of that pre approval to train more manufacturing people.
However, I think the ramp up will continue post approval as well just to kind of balance out where we focus our efforts leading up to the launch, right? So the timing by which we will be operating at that full 10 a month kind of capacity is a little bit influx, but we think that by end of 2025, we should be achieving at least close to that kind of capacity. And the additional space that we talked about, it's a bit early. We have leased the space. We are engaging with design engineers who have experience in designing for such types of manufacturing GMP spaces for these types of autologous processing suite.
And we're trying to see how best to optimize that. I think there are multiple scenarios we are considering right now. So as we get closer and closer to the approval time, we'll give you more color as to how that ramp up will look like, how quickly we can do because there are multiple factors we are optimizing for. One is the speed at which we can add more additional suites and the other is how can we do that without disturbing existing manufacturing space because can't compromise on that. I think balancing these 2, as well as how much space do we really need to bring into additional GMP manufacturing, I think, these are all parts of the equation that will get clearer and clearer as we do our homework a little bit.
So hopefully by the next call, Maury, we should have a little bit more clarity on that. I hope at least conceptually it gives you an idea of how we're looking at capacity because as Madhav mentioned, clearly the demand is going to be a lot more than our supply at the time of approval.
Maury Raycroft, Analyst, Jefferies: Got it. Yes, all really helpful context. Thanks for taking my questions.
Jenny, Conference Call Operator: Thank you very much. Your next question is coming from James Molloy of Alliance Global Partners (NYSE:GLP). James, your line is live.
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: Hey, guys. Thank you very much for taking the questions. And actually a little more on the question from before or the answer from before. When you said the 10 patient runs per month, what does that translate into sort of per year and how about 2 25 patients in the target that you walk through the 30% of the 750 that are eligible upon should you get approval? What's how should we on the outside looking in be thinking about when you guys could be able to potentially supply that 225 or potentially even expand that going forward?
Thank you.
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: Yes. Thanks, Jim. So yes, the 10 patients a month, which translates to about 120 a year, Our goal is to go beyond that, right? So that's why we are looking at additional space and lease agreements and things like that. How quickly will that come to effect?
I think it's a little premature to speak about it because there are multiple components in that and there is also regulatory review involved because once the additional space is created and we start to do engineering runs, we have to submit those designs and there's going to be a need for regulatory review as well. I think approximately the lead time is going to be anywhere between 18 to 24 months. That is just based on analogs we've seen in the past from these types of therapies. So can it be sooner or even later? I think that is something that we will have more clarity as these designs for new building space get crystallized a little bit and our dialogue with the FDA will ensue that step.
So we'll get more clarity, but I think a good ballpark is a year and a half to 2 years after we've made a clear design print on the paper. So that's kind of a very high level estimation.
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: Excellent. Thank you, Brian. Best you can do at this point, of course. Then given sort of the demand versus supply you guys will be facing and you guys are very good at giving ideas on pricing, obviously, see Bikubex pricing. What do you guys think on pricing power to raise pricing going forward as you're expanding capacity?
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: You mean pricing for PC cell treatment, Jim?
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: Yes. Yes, of course.
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: Yes. I mean, I think price for PC cell treatment, yes, definitely there is value here that we are bringing to the patients and the community and we will want to capture that value through pricing. Given the durability, etcetera, we have talked about it as comparable to one time gene therapy price that PC cells can secure. And that is really what our going in assumption is. We haven't guided in terms of the numbers.
I think in our last call, we said a floor of 1,500,000 per PD treatment. We are continuing to explore the upper bounds of this because we our main goal is not to limit patient access, right? We want to make sure payers are comfortable and we are exploring our options in terms of especially when you have a product like P gCell with its efficacy and the outcomes that we have, what options are there on the table. So we are being creative and want to of course make sure that we recognize the value that we are bringing and are able to capture it through pricing is certainly one
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: liver. And as I
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: mentioned, patients, we do expect them to come back for a repeat treatment for the previously untreated areas, which will be its own billing cycle and its own price there, right? So that's how we are thinking about it. And the fact that we in house, we are not relying on 3rd party to produce our vector, it's all done by Abeona. That in and of itself gives us a lot of control, autonomy as well as a profitable model to be able to exercise that, right? So it is helping us from both sides that we have the ability to control the speed and also manage the cost while we think about the manufacturing scale.
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: Great. Thank you very much. Yes, certainly seems pretty straightforward calculation to show the value of the treatment you're helping these patients avoid. Did I hear correctly $1,500,000 a year annual cost of therapy roughly? It's
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: not a year. It's for treatment, which is a flat price.
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: Right. Okay, great. Thank you.
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: Yes.
Jenny, Conference Call Operator: Thank you very much. Your next question is coming from Kristin Kluska of Cantor Fitzgerald. Kristin, your line is live.
Kristin Kluska, Analyst, Cantor Fitzgerald: Hi, good morning, everybody. Thanks for taking the questions and congrats on the resubmission and appreciate all the transparency you've shared with us over the last few months. It's been very helpful. So as you think about potential conversations with surgeons and doctors, if you are approved, I mean, I think the data and the durability really speak for themselves. But can you share with us how you're going to talk to them about the actual surgical procedure, including how the actual surgical procedure, including how the patients in the trial experience in the past have responded from it?
Do you see this as a potential barrier? Thank you.
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: Yes. Thanks for that question, Kristen. We don't see this as a barrier, but I can give you a high level answer. So the surgeons don't consider this as anything, especially new beyond what plastic surgery procedure would entail. We don't see the surgery aspects of applying TZ cell itself as a barrier.
As you know, 2 of the 5 centers potentially could be our clinical sites and our clinical sites have had a lot of that experience through our trials. And we've seen through the experience of onboarding the UMass site that was onboarded around the later half of our vital study that the surgical aspects were actually pretty smooth in the communications between the doctors that were at Stanford and at UMass. And we see the site onboarding path to be seamless that way, because we've been through that kind of an experience once. It's just replicating best practices that we need to achieve. I think from a patient perspective, having these additional sites should only be more helpful because our goal is to minimize their travel time and some of the logistic burden that comes with that.
Madhav, let me ask you to add more color based on our recent interactions with potential sites that we're already working with in terms of the multidisciplinary aspects of how PCEL application can be cross pollinated for best practices across these centers?
Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development, Abeona Therapeutics: Right. Yes, no, absolutely. Everything what Vish mentioned, that's really the sentiment we are seeing from the other sites also, Kristen, that we are interacting with. Surgically, it's not this is not they are so comfortable because this is looking technologically difficult for them from the actual procedures standpoint. In fact, even dermatological surgeons have applied PD cell.
It's just that we say surgeons or plastic surgeons because they tend to have privileges to the OR. But these surgeons are part of the conversations we are having and they are seeing the technology and for them this is not at all a difficult thing. We are also working with the cell therapy coordinators at these centers. I think that's the beauty because these centers are have onboarded multiple cell therapies in their institutions. And from that standpoint, there is an infrastructure, there is a process flow.
Yes, of course, there are nuances for PDcell because PDcell, when approved, will be the 1st ex vivo autologous non systemic therapy, right, period, I mean across all of the technologies out there. So because of that, there are certain nuances of, okay, areas to be treated and mapping that out for the patient. And the fact that the clinical trial experience we have gained now with our Phase 3b study is also just helping build that experience curve for us. So no real issues that we are seeing from a procedural standpoint. And as Vish mentioned, yes, the patients, they are coming back for untreated areas that in and of itself is a pretty positive thing that they see the benefit.
Kristin Kluska, Analyst, Cantor Fitzgerald: Thank you so much for that. And then when you resubmitted the BLA and it was and the resubmission was accepted for review, did you get any sense from the agency whether the focus during the time from now to the PDUFA is solely going to be on those red line items that were new essentially from the first time? Or are they going to be looking at everything holistically? Thank you again.
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: Thank you, Chris. And the short answer is, no, we don't have a sense yet. We just had the 2 week period is they're going to just look at all the components of data that have been requested and is our application complete in many ways, right? So we do not have any sense to whether everything that has been previously reviewed is going to be opened up again versus focus on the delta, right? But our assumption is that majority of the review will focus on those additional items just based on our conversations leading up to the resubmission and also the nature of what a CRL really is by process.
A CRL is supposed to be a comprehensive list of things that having reviewed the entirety of the package, these are the things that you need to deliver for an approval. So in that spirit, our assumption is that, yes, the focus is going to be on those additional items. But very soon, we're going to have that dialogue pickup because it's just been about that 2 week period since we made the resubmission. So we'll definitely, as we've been transparent all these months, we will continue to do that, Christian.
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: Thank
Kristin Kluska, Analyst, Cantor Fitzgerald: you. Thank you
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: so much. Thank you
Brian Kemeny, Chief Technical Officer, Abeona Therapeutics: so much.
Jenny, Conference Call Operator: Well, we appear to have reached the end of our question and answer session. So I will now hand back over to Vish for his closing remarks.
Vish Seshadri, Chief Executive Officer, Abeona Therapeutics: Thank you so much. And before I give you the closing remarks, I just wanted to clarify one of the Q and A questions was about a pricing floor. We have not really disclosed any or even finalized pricing for pzcell at this point in time. Jim, the question that you had asked was about the $1,500,000 was just a floor that we had communicated. We do see from a lot of our dialogue so far that there could be an upside.
So we're still investigating that. So just wanted to clarify that, so people don't think that we have priced PD Cell at any given price point yet. So with that, I think, in closing, we're 1 step closer to a potential approval for PDCELL, and we believe that we're well positioned for this significant opportunity ahead at Abeona, and we look forward to finishing this year strong and to a potentially transformative 2025 for the company. And I know we did not talk much about our pipeline, which we will do in our future calls. We just wanted to focus today's time on the upcoming preparations for PDCELL.
So thank you everyone for joining us today for the business update and we'll talk to you again soon.
Jenny, Conference Call Operator: Thank you very much. This does conclude today's conference. You may disconnect your phone lines at this time and have a wonderful day. Thank you for your participation.
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