Abeona Therapeutics Inc. Q1 FY2023 Earnings Call

Greetings. Welcome to the Abeona Therapeutics First Quarter '23 Portfolio Update Conference Call. Please note, this conference is being recorded. I will now turn the conference over to your host, Greg Gin, Vice President of Investor Relations and Corporate Communications. You may begin.

Thank you, Holly. Good morning, everyone. I would like to welcome and thank everyone for joining us on our portfolio update conference call. Our objective today is to share additional new positive data from our EB-101 and AAV ophthalmology programs recently presented at scientific congresses. The press release announcing the data at the ISID and ASGCT meetings is available on our website at www.abeonatherapeutics.com. Before we start, 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 section in our Form 10-K and periodic reports filed with the SEC. These documents are available on our website at www.abeonatherapeutics.com. On the call today are Dr. Vish Seshadri, Chief Executive Officer, who will give some opening remarks; Dr. Dmitriy Grachev, Chief Medical Officer, who will review the new VIITAL study data that we presented at ISID; and Dr. Brian Kevany, Chief Technical Officer, who will review the animal proof-of-concept data from our AAV ophthalmology program presented at ASGCT last week. After the prepared remarks, joining us for the Q&A session will be Joe Vazzano, Chief Financial Officer; and Dr. Madhav Vasanthavada, Vice President, Business Development. And with that, I will now turn the call over to Vish Seshadri to kick it off. Vish?

Thank you, Greg. Good morning, everybody, and thank you for joining us this morning. I want to start today by saying last week was a great advancement for the epidermolysis bullosa, or EB, community. I want to congratulate Krystal Biotech, the EB patient community, researchers, and regulators for bringing the first genetic therapeutic option to tackle this debilitating disease. As more therapies come to market, we anticipate increased awareness about therapeutic options in the EB space and also improved diagnosis and genotyping for this painful disease. Recessive dystrophic EB, or RDEB, is a connective tissue disorder in which both copies of the gene for collagen VII are mutated and dysfunctional, resulting in the lack of connectivity between the outer and inner layers of the skin. Patients with RDEB face a lifelong struggle with fragile skin that easily tears and blisters, with most patients developing large, painful wounds that remain unhealed, often covering a significant proportion of their body. Recently published natural history data in RDEB analyzing 251 RDEB wounds describes the two distinct types of RDEB wounds. On the top are chronic open RDEB wounds, while on the bottom are the recurrent wounds. On the right, the spider plot shows chronic wounds rarely show 50% or greater wound healing, while the recurrent wounds frequently heal completely and then reopen again. Furthermore, at ISID this year, a prospective study was presented by the team at Stanford that further corroborated this distinction between large chronic wounds and recurrent wounds. We are excited to advance EB-101 as a potential therapy that could deliver years of sustained wound healing and proven pain relief after a one-time treatment cycle for large chronic RDEB wounds. These are pictures of the patients in our VIITAL study with large chronic wounds. You can see the large area coverage and wound healing that were achieved with three EB-101 sheets applied in a meticulous fashion. On the far right, tattooed wounds were scored as greater than 75% healed. Although the wound looks nearly completely healed to the naked eye, they were scored at 75% plus healed because without picking the graft, the physician is unable to confirm complete healing. That speaks to the stringent criteria in our scoring for complete wound closure. Over the next two slides, I'll discuss the molecular basis for how EB-101 delivers wound healing in a sustained fashion. EB-101 works by durably restoring functional collagen VII expression to RDEB keratinocytes. Keratinocytes are sourced from patients' skin biopsy, grown in cell culture, and transduced with a corrected copy of the COL7A1 gene through a retroviral vector. The transduced keratinocytes are further cultured into epidermal sheets that are transplanted onto open wounds of patients to secure instantaneous closure. The scientific rationale for EB-101's durable benefit is that the transgene encoding function of collagen 7A1 integrates into the host genome and is therefore maintained stably as cells divide and form epidermal sheets in our manufacturing process. Furthermore, immunohistochemistry of skin biopsies from EB-101 treated sites demonstrates sustained collagen expression for up to 24 months. In fact, we have unpublished data for up to 36 months. Those green lines that you see indicate collagen 7A1 staining and the white arrows point to the epidermal junction. This is the rationale for the compelling durability with EB-101 as observed in our Phase 1/2a study at the mean follow-up of 5.9 years and a maximum of 8 years. The results show that the majority of EB-101 treated wounds showed sustained healing and pain reduction after a one-time treatment. I'd now like to turn the call over to our Chief Medical Officer, Dr. Dmitriy Grachev, who will review the results that were presented at ISID.

Thank you, Vish. First, I would like to express my excitement about the data presented at the International Societies for Investigative Dermatology meetings, or ISID. Dr. Jean Tang, Professor of Dermatology at Stanford and Principal Investigator of the VIITAL study, presented the data during an oral session at ISID, and the data were also featured in a separate poster presentation. The results presented at ISID show that EB-101 improved wound healing and pain reduction at 6, 12, and 24 weeks compared to control wounds following one-time application of EB-101. Furthermore, EB-101 demonstrated improvement in patient-reported and caregiver-reported outcomes for pain and blistering severity. Before reviewing the results, I want to provide a brief summary of the trial design for VIITAL. VIITAL was designed to investigate the efficacy, safety, and tolerability of EB-101 in approximately 36 large chronic wound pairs in 10 to 15 patients with a minimum age of 6 years. Our study was randomized and controlled. Each patient had a minimum of two large chronic wounds selected, with each undermined treated wound being paired with a controlled wound of similar size, that would remain chronically open, to the extent possible, within the same patients. In VIITAL, we define large chronic wounds as wounds that have greater than 20 square centimeters of surface area and remain open for a minimum of 6 months, although many were open for years. We aligned with the FDA on the study endpoints and statistical analysis plan. Given that EB-101 is targeting large chronic wounds, we considered these the hardest to treat wounds that remain open for years because they cannot self-heal. We aligned on the co-primary endpoint of greater than or equal to 50% wound healing and the second co-primary endpoint of pain reduction as an additional measure of clinical benefit. Specifically, the co-primary endpoints were the proportion of RDEB wound sites that showed greater than or equal to 50% of healing from baseline, comparing randomized treated with matched control wounds at the 6-month time point as determined by direct investigator assessment; and second, pain reduction associated with wound dressing change assessed by the mean difference in score of the Wong-Baker FACES scale between randomized treated and matched control wounds at the 6-month time point as reported by the patient. This is the first pivotal study to include patient-reported pain as a co-primary endpoint. The secondary endpoint was the proportion of RDEB wound sites with complete wound healing from baseline, comparing randomized treated with matched control wounds at weeks 12 and 24. Exploratory endpoints included additional assessments for wound healing and pain reduction as well as evaluation for itch severity and blistering. We reviewed the baseline characteristics during the VIITAL top line results call, and I won’t go into details here. I'm just to remind you of the large size and severe pain associated with the wounds included in VIITAL. The median body surface area of randomized wounds treated with EB-101 per patient was 160 square centimeters, with the range of 18 to 200 square centimeters. To clarify, areas of treated wounds were 40 square centimeters, exactly the size of the graft. For large chronic areas that exceed 40 square centimeters, multiple grafts could be applied in a layered fashion, in which case the area covered by each graft is considered a distinct wound. A wound area that was less than 40 square centimeters, debridement was required to prepare the wound bed to receive the graft. On the contrary, control wounds were not debrided. The median wound duration of randomized treated wounds that remained chronically open was 60 months, or 5 years. The control wounds had a similar wound duration. Now we can turn to the results. To help you follow the charts and graphs, EB-101 treated wounds are shown in blue, while control wounds are shown in gray. Wound healing was observed at the early time point assessed, which was 6 weeks, and was sustained at all subsequent time points, including 24 weeks. Consistent with wound healing, statistically significant pain reduction was seen at the early time point assessed, which was 6 weeks and sustained at all subsequent time points, including 24 weeks. Pain assessment by caregivers showed great improvement in pain scores for EB-101 treated wounds, with 46% of caregivers categorizing wounds as much improved or very much improved at week 24 from baseline for EB-101 treated wounds compared to 0% for the control wounds. In addition, at-home pain severity assessment using the Wong-Baker FACES scale showed significant pain reduction with EB-101 treatment as early as week 3. Pain was also assessed using the patient-reported outcomes measurement information system called PROMIS with a significantly greater improvement in pain quality sensory scores achieved with EB-101 treatment. In addition to significantly reducing pain, patient-reported and caregiver-reported outcomes related to each and blistering showed significantly greater improvement with EB-101 treatment. As a reminder, itch for RDEB patients is a significant factor that impacts quality of life and increases the risk of trauma, blistering, and infection. Now let's review safety and tolerability. EB-101 was shown to be safe and well-tolerated, with no serious treatment-related adverse events observed in VIITAL, consistent with past clinical trial experience. In conclusion, VIITAL demonstrated positive pivotal study results and a favorable risk-benefit profile for EB-101 in patients with RDEB. Back to you, Vish.

Thank you, Dmitriy. We've made significant progress towards BLA submission and are marching towards commercialization. We had previously mentioned that one of the key milestones was to complete three consecutive process performance qualification, or PPQ runs, to demonstrate our validated process and readiness for commercial production. We are excited to share that we have completed this important step for both the retroviral vector manufacturing and EB-101 drug product manufacturing, which was the last critical piece of data we had to generate to complete the CMC module for the BLA submission. We announced in our first quarter 2023 results press release that we had submitted a pre-BLA meeting request with the FDA. The FDA has since accepted our request and the pre-BLA meeting is scheduled for July 10, 2023, to discuss the format, content and acceptability of the anticipated BLA application. Based on this meeting date, we are on track for the anticipated BLA submission in early third quarter of 2023. Based on the anticipated timing of BLA submission, we expect potential BLA approvals in late first quarter or early second quarter of 2024. If the BLA is approved, we anticipate being granted the priority review voucher, which can be used to receive expedited review by the FDA of a subsequent marketing application for a different product or sold to another company. Prior PRVs have been sold to other biopharma companies for approximately $100 million. As part of our commercial planning for EB-101, we continue to engage with stakeholders across the health care system, including public and private payers and health care providers, to better understand market access and pricing for EB-101. We are encouraged by the initial feedback from these stakeholders and feedback that we have received from patient advocates and organizations, which collectively support positive coverage decisions and pricing in line with the value of a one-time treatment that delivers wound healing and pain reduction for years. Based on our initial discussions, payers view RDEB as a disease with very high unmet need and believe EB-101 has a well-differentiated profile with durable clinical benefit for the treated wounds. Also, given the ultra-rare prevalence of RDEB, payers view EB-101 as having a limited overall budget impact, and they have indicated a high willingness to cover EB-101 with favorable access policies, giving us confidence in its potential. Now let's turn to our preclinical ophthalmology programs. We presented animal proof-of-concept data at ASGCT for investigative AAV-based gene therapies for Stargardt disease, X-linked retinoschisis, and autosomal dominant optic atrophy. The preclinical proof-of-concept data provides early evidence of the potential of our proprietary AAV capsid and gene construct to express the recombinant protein in target tissues and rescue mutant phenotypes in mouse disease models. I'd now like to turn the call over to our Chief Technical Officer, Dr. Brian Kevany, who will review the results presented at ASGCT.

Thank you, Vish. We are excited by the broad potential for treating serious eye diseases with the new AAV-based therapies using novel AAV capsids from our in-licensed AM capsid library and internal research. At ASGCT, we presented three posters highlighting encouraging findings from animal proof-of-concept experiments from our AAV ophthalmology program. The first poster presented featured ABO-504, a novel approach to treating Stargardt disease, the most common form of juvenile inherited macular dystrophy. Autosomal recessive Stargardt disease is caused by mutations in the ABCA4 gene, preventing the removal of toxic substances from photoreceptor cells that result in photoreceptor cell death and progressive vision loss. Our proprietary strategy is designed to efficiently reconstitute the full-length ABCA4 gene, which is too large to fit in a standard AAV genome, by implementing a dual AAV vector strategy utilizing the Cre-LoxP recombinase system. The data at ASGCT provides compelling evidence that two independent AAV vectors utilizing Cre recombinase can efficiently reconstitute the ABCA4 gene, leading to full-length ABCA4 protein expression. Key conclusions presented include that in vivo Cre-mediated recombination yields full-length ABCA4 using our dual AAV vector system, and recombinant human ABCA4 is detected and properly localized to photoreceptor outer segments within 1 month of treatment. Our results show that Cre-mediated recombination of dual AAV vectors is safe and effective in delivering an expression of full-length human ABCA4 in a knockout mouse model, paving the way for a novel therapeutic approach for treating Stargardt disease. Further studies will evaluate ABCA4 expression and accumulation of lipofuscin in a mouse model of Stargardt disease. Additionally, we believe our dual vector approach using Cre-LoxP can also be adapted for other therapies in which AAV-based delivery of large genes is desired. Our second poster presented at ASGCT featured ABO-503, a novel gene therapy approach for the treatment of X-linked retinoschisis, or XLRS, the most common form of inherited macular dystrophy in males. XLRS patients present in the first decade of life with cavities developing between retinal layers leading to discontinuity within the retinal circuitry, photoreceptor degeneration, and vision loss. ABO-503 is composed of a functional human RS1 packaged in the novel AIM capsid AAV204 that effectively targets photoreceptors and restores RS1 expression. ABO-503 will be administered via a para-retinal injection that will provide an improved safety profile for treatment of XLRS, in which targeting of photoreceptors is desired, but the disease renders the retinas susceptible to damage from more invasive approaches. The data presented at ASGCT demonstrated robust RS1 expression in the retina, improved cone photoreceptor density, and overall photoreceptor cell survival, as well as the restoration of the outer retinal architecture. Treatment with ABO-503 in mutant mice was associated with photoreceptor preservation and improved retinal function. These results support further development of ABO-503 for the treatment of XLRS. Our third poster presentation at ASGCT featured an investigative AAV gene therapy for autosomal dominant optic atrophy, or ADOA, a form of vision loss associated with the loss of retinal ganglion cells or RGCs, residing in the interretina caused by mutations in the Opa1 gene. The candidate vector is composed of the human Opa1 gene packaged in the AmCapsid-AAV204 discussed previously. The proximity of RGCs to the vitreous cavity makes them an attractive target for gene therapy delivered by a para-retinal dosing route. The data presented confirmed expression of Opa1 in both cell culture and retinas of dosed wild-type and disease model animals, improvements in photoreceptor outer nuclear layer thickness, and optokinetic responses were observed with our Opa1 gene therapy candidate in a 10-month proof-of-concept study designed to test the functional consequences of Opa1 restoration in mouse retinas following an intravitreal injection. Initial efficacy results suggest an improvement in retinal signaling to the brain and improved visual acuity in treated mutant mice. These benefits support further development of our Opa1 gene therapy candidate. Back to you, Vish.

Thank you, Brian. We are looking forward to pre-IND meetings with the FDA for two of our programs taking place this quarter. With that, I'll turn the call back over to Holly for the Q&A section.

Your first question for today is coming from Maury Raycroft at Jefferies.

Hi. Good morning and congrats on the updates and thanks for the presentation today. We saw Krystal's Vyjuvek was approved with a relatively broad label. Do you expect the same based on your conversations with the agency, including patient profile, type of wounds, et cetera? Maybe if you can talk a little bit about what you're expecting in the label.

Thank you, Maury, for the question, and good morning. Regarding the indication and the patient types, I want to reiterate that our VIITAL pivotal study has treated RDEB patients, recessive dystrophic EB. We have inclusion criteria that focus on large chronic wounds. It's very early in the BLA process to comment on how broad or restrictive the label would be, given the high unmet need, and this is an ongoing dialogue that will be maintained with the agency. So unfortunately, I cannot give you a definitive answer, but I can assure you that the types of wounds for which we have generated evidence are significant.

Got it. That makes sense. And you mentioned that initial feedback from stakeholders across the health care system has been positive. Maybe if you can talk a little bit about how you're anticipating pricing could work out with Vyjuvek having a list price of about $630,000. I guess how does that inform how you're thinking about pricing?

Certainly, we can talk about that. I will turn the call over to Madhav Vasanthavada, who is leading many of our discussions with crucial stakeholders like payers and hospital administrators, where there's a clear appreciation for the value proposition of a one-time treatment that can give many years of benefit without having to treat the same wound repeatedly. So Madhav, can you shed light on the differentiation and how we have discussed pricing?

Sure, Vish. Hey, Maury, thanks for the question. Yes, we find Vyjuvek's launch price now to be very insightful in how we will go about pricing. Earlier in the year, we conducted payer research, mostly blinded research with nearly a dozen payer groups across commercial, Medicare, and Medicaid. It was encouraging to hear that when we discussed the clinical profiles of all the investigational therapies, including our own EB-101, that the payers find the clinical profile for EB-101 to be transformational, given the wound healing profile, pain reduction, and durability. At that time, we had posited a few different pricing scenarios based on Vyjuvek’s expected entry price and how that could compare with our potential price and elasticity for EB-101, which was all very encouraging. Now that we have learned what the pricing for Vyjuvek is, we will be in a much better position to determine the launch price for EB-101, ensuring that there are no access hurdles for patients, while also capturing the value of the innovation and the durable effects that EB-101 can provide. Overall, I think the price elasticity remains favorable, and given the profile we now have, we will be well-positioned to establish a launch price for EB-101.

Got it.

Does that help clarify?

Yes, that's helpful. It makes sense with understanding the value proposition. And maybe last question, and then I'll hop back in the queue. The gene therapy data from ASGCT looks good. Just wondering if you could talk a little bit more about how you're prioritizing those three different opportunities. Should we think of Stargardt with the 504 program as being the lead? Or how should we think about that?

Sure. I can talk a little bit about that and then turn it over to Brian for any additional thoughts. Right now, we have compelling preclinical data from these disease models in mice that show the expression of the gene. As you may be aware, there's a high unmet need in all three of those indications. We have chosen those indications specifically based on how many other investigational therapies are able to make inroads and have hit some challenges along the way. We are encouraged to see that Stargardt, of course, the most talked about among the three, allows us to uniquely address the size of the ABCA4 gene and produce that and effectively correct tissue. In terms of prioritization and how we resource the programs, Maury, we have many dialogues ongoing. We are looking at different sources of non-diluted funding, as well as some potential government funds. These programs all present equally high unmet needs, and it's not really going to be a straightforward prioritization; it's more about timing. Such events will be discussed further in our upcoming calls as we make more progress and gain more clarity on nuances from our dialogue with the agency, that will inform our prioritization.

Got it. That makes sense. Thanks for taking my questions. I will hop back in the queue.

Thank you, Maury.

Your next question is coming from Kristen Kluska at Cantor Fitzgerald.

Hi. Good morning, everybody, and congrats on both of these recent data sets. The first question I had was related to the ophthalmology portfolio. So we haven't seen as many dual AAV vector strategies yet in gene therapy. I know it's something that others have discussed for some time. So maybe the first part of the question is just understanding what you think is differentiated about your construct that led to the successful findings at the preclinical stage. The latter half of that question is just looking at the dual AAV vector landscape. What are the expectations on how you would expect this to translate into human studies? And I guess the key things to look out for, given the difference between the models.

Hi, Kristen, thanks for the question. I'm going to turn it over to Brian Kevany, our expert on AAV and ophthalmology, but I want to say that thank you for the question specifically because there are certain mechanistic elements of why we believe we can be successful where other approaches to getting full-length ABCA4 have failed in the past. So Brian, can you please elaborate on our unique gene constructs and the dual AAV system?

Of course, yes. Thanks, Kristen. So I think there have been a number of strategies that have been tried, both in dual AAV vectors and mini genes for ABCA4. While those programs have been ongoing for a number of years, we haven't seen a lot of movement. We're encouraged by our ability to push into a field struggling to achieve constructs that provide full-length ABCA4. As far as how we differentiate from others, the Cre recombinase system is one of the most efficient recombination systems known. We're leveraging that technology as part of our therapy development. So I think the major differentiating point for us is less about the vectors and more about the actual mechanistic aspects of the therapy we're developing here. And Kristen, could you repeat your second part of your question?

The second part of the question was just essentially on how you think it's going to translate into human studies. What are the key things to consider, especially the risk of going from the differences in the models?

Sure. Yes. I don't anticipate any significant differences. The advantage here is we are approaching this with a sub-retinal delivery for the Stargardt program. For those who aren't aware, this is a relatively specific injection that occurs between the photoreceptors and the retinal pigment epithelium. This directed injection keeps the vector very close to the site of action and does not get diluted out, unlike intravenous or other types of injections. So this aspect keeps it somewhat similar in translation between mouse and larger animal models during IND-enabling studies, and then into human studies. The translation from large animal to human studies is likely to be relatively smooth, as the injection volume will be the same. We’ll use identical dosing techniques.

Thank you. And then for the autosomal dominant optic atrophy study that you presented on, you mentioned that visual acuity assessments demonstrate functional recovery. I think with a lot of AAV ophthalmology gene therapy trials, the goal is often to show the slowing down of disease progression rather than reversal. I'm interested in the context of that comment and how you think about this clinically, considering that a lot of it will depend on the age of intervention and the different rates at which these diseases progress.

Yes. Visual acuity in mice is something we’ve studied with this animal study, looking at preservation or improvement of visual acuity over time. The treatment was provided early on in the disease progression, prior to any degradation of that visual acuity. This treatment effectively prevented any further development of deterioration. Visual acuity is challenging to study in humans because it takes a long time for its development. We've seen numerous failures in the industry where visual acuity was the primary endpoint, and due to the slow progression of these diseases, it tends to be quite a blunt instrument for assessing therapeutic benefit. As part of our discussions with the agency, we will look more into how primary endpoint decisions are made to better encapsulate therapeutic benefits. This was a hot topic at ASGCT specifically in ocular space and the rare disease area regarding meaningful endpoints that adequately capture therapeutic benefit.

Yes. If I may add to that, Kristen, what we're aiming for in any monogenic disease with gene therapy is to treat early enough to ensure that the disease symptoms do not develop; we want to preserve eye function. However, in clinical development, the first priority will be to aim for prevention or cessation of deterioration of visual acuity. Ultimately, we are investigating whether it is possible to reverse the disease that has already occurred because there exists a prevalent pool of patients with ongoing disease. We will be seeking to test the various populations, potentially addressing visual function deterioration which could show reversibility. This will factor into our plans for clinical investigation.

Great. Thanks. Lastly, regarding the RDEB data presented at ISID, two key new findings included understanding the effects and earlier points of evaluation, as well as additional secondary endpoints. Since you've presented this data, how important are these two components in discussions with thought leaders? I know previously we discussed pain reductions correlated with wound healing, but now you have additional data points to support this idea?

Thanks for that, Kristen. I’ll take that. To highlight the new data presented at ISID that wasn't included in the top line results, we focused on the 6-month endpoint as the regulatory endpoint. The new data demonstrates how quickly we see these effects; we observed benefits as early as 6 weeks, as well as results at 12 and 24 weeks, and even 3 weeks post-treatment where patients were self-reporting their pain levels significantly: caregivers were also recording these improvements. We've seen that clinical benefits manifest early, with strong effects lasting across all time points. The second key point concerns the correlation between pain reduction and wound healing, which is encouraging to see; notable improvements have been consistent throughout the time course post-treatment. This data will be important in building on the value proposition, and as Dr. Dmitriy Grachev presented, we have itch data demonstrating a significant improvement for treated versus untreated wounds. Itch is a risk factor for trauma and blistering, and this data is another important measure of success for EB-101, thus offering a holistic view of patient benefits. Encouragingly, patients from the VIITAL study are now following up and wanting their untreated wounds addressed, which further reinforces the value of our therapy.

Your next question is coming from Jim Molloy at Alliance Global Partners.

Hey, guys. Good morning. Thanks for taking my questions. I just want to follow up on a prior point regarding Vyjuvek's approval. On their call, they discussed a price of about $630,000 a year, near $485,000 a year after U.S. government discounts. I think they mentioned about 1,100 target patients. Not to hold due to a competitor's numbers, but how do those impact your thoughts on where pricing could land and the size of the market, given the publicly shared estimates?

Thank you for the question, Jim. We did take note of the pricing that was announced. At this point in time, as Madhav mentioned, we are encouraged by the value various stakeholders are attributing to gene therapies. I would point out that what we perceived a year ago regarding potential pricing has changed significantly in light of the positive VIITAL results reflecting a favorable clinical profile. What remains uncertain is many specifics about the label for Vyjuvek — #1, it’s very broad, covering all of dystrophic EB, whereas our clinical evidence is primarily focused on recessive dystrophic EB, which represents a narrower population. This difference means it's really an apples-to-oranges comparison; our value propositions are quite different because, on one side, you have a redoseable gene therapy; on the other, we have a one-time treatment that may bring many years of benefit. That being said, we will need to do our homework to understand how the estimated pricing plays out in reality, as we make initial considerations — it's crucial not to underestimate the real-world costs associated with these therapies—so we have some time ahead for that analysis. What I can promise is that we aim to be responsible corporate citizens and ensure patient access to our therapies as a guiding principle.

Outstanding. Thank you. On the pre-BLA, is there anything specific we should anticipate coming out of that meeting? I imagine it's a fairly straightforward meeting heading into a BLA. Also, given the competitor's approval, has this changed your thinking on self-launch or potential partnerships?

Thanks, Jim. To your first question on the pre-BLA, every BLA submission involves complexity regarding our therapies, the therapeutic niche, and our limited experience in these diseases, so the pre-BLA meeting is essential in confirming we included everything necessary for submission. This meeting will be a formative step, but I want to stress that this doesn’t imply total silence with the agency prior — we’ve engaged with them extensively, including informal meetings and exchanges. This meeting signifies the culmination step, affirming we are ready to present our dossier. We're confident about the timing because, as I mentioned, the major piece of work—the PPQ runs—has been successfully completed. And those runs not only needed to meet release criteria for EB-101 drug products but also all the various process parameters that must fall within pre-specified ranges, demonstrating process robustness. For your second question about partnering and commercialization, given the rare disease context, our focus will be delivering therapy in clinical centers that can administer it. We’re more focused on patient services than traditional sales and marketing demands. Hence, if we partner, we expect a very tailored approach rather than a conventional large pharmaceutical type of partnership. We're open to different structures while maintaining our goal of value retention. We're preparing for patient outreach and payer dialogues — these conversations will gain momentum following BLA submission, which will mark a crucial step for us.

Great. Thanks for taking the questions.

Of course, thank you.

We have reached the end of the question-and-answer session, and I will now turn the call over to Vishwas for closing remarks.

Thank you. In closing, I want to thank our shareholders and other stakeholders who have listened to this call, and we will talk to you again soon. Thank you.

This concludes today's conference, and you may disconnect your lines at this time. Thank you for your participation.