Abeona Therapeutics Inc (ABEO) 2020 Q4 法說會逐字稿

Good day, and welcome to the Abeona Therapeutics Fourth Quarter and Full Year 2020 Conference Call. (Operator Instructions) As a reminder, today's conference call is being recorded.

I'll now introduce you to today's conference host, Greg gin, Vice President of Investor Relations and Corporate Communications at Abeona. Please go ahead.

Thank you, Holly. Good morning, everyone. I would like to welcome you, and thank you for joining us on our fourth quarter and full year 2020 conference call. Yesterday, after the close of financial markets, we issued a press release announcing the fourth quarter and full year results and recent operational progress. The press release is posted on our website at www.abeonatherapeutics.com.

On the call today with prepared remarks are Michael Amoroso, Chief Executive Officer of Abeona; and Ed Carr, Chief Accounting Officer. After the prepared remarks, we will host a Q&A session. And joining us for the Q&A today -- will be joined by Jay Bircher, Chief Technical Officer; and Dr. Juan Ruiz, Vice President of European Medical Affairs at Abeona and who also heads clinical development for our MPS programs.

Before we start, I will review our safe harbor statement. Remarks made during today's call may contain projections and forward-looking statements regarding future events. 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 section entitled Risk Factors in the company's annual report on Form 10-K and quarterly reports on Form 10-Q, filed by the company with the SEC. These documents are available on our website at www.abeonatherapeutics.com.

And with that, I will now turn the call over to Michael. Michael?

Thank you, Greg, and good morning to our investor community. It's my pleasure to be with you again today for our Q4 and full year call. We're excited at Abeona developing our novel and gene cell therapies for underserved patient populations, and we remain focused on execution. 2021 is a year of execution and delivery. On today's call, I'll start with a brief review of some of our key accomplishments over the past year 2020, and I'll talk to you about our fast start to 2021.

In '20 and '21, I'm very proud of the team. Despite the macro disruptions, caused by the pandemic, we continue to advance all 3 clinical programs toward bringing urgent-needed treatments to patients with recessive dystrophic epidermolysis bullosa, as I'll refer to as RDEB on our call, our lead program, as well as Sanfilippo syndrome types A and B, referred to as MPS IIIA and IIIB, respectively.

First, let's start with our lead pivotal program, EB-101, and look at a brief overview of 2020. Earlier in the year, we initiated our pivotal Phase III VITAL Study for EB-101, as you'll remember, and RDEB at the Stanford University, a world-renowned EB treatment center. In July of '20, we presented our Phase I/II data at the society for pediatric dermatology, showing that EB-101 treatment of large and chronic wounds resulted in substantial and durable wound healing for patients, also associated with long-term improvement in pain relief for up to 5 years.

As a reminder, large wounds characterized in our trial as 20 centimeters or greater, some going up to 100-plus centimeters, 200-centimeter wounds, and chronic wounds, wound that are open 6 months and longer and can no longer heal themselves like recurrent blistering, will start in EB or RDEB. These are the most problematic wounds for patients suffering from RDEB. They lead to significant morbidity issues, namely severe pain, opioid use, hours per day of dressing change. And ultimately, and most unfortunately, they lead to most patient demise by 30 to 40 years of age. This Phase I/II data was exciting and validating for us, also generated with Stanford University as it serves as the proof-of-concept for our pivotal VITAL Study.

Next, after accruing patients 2 and 3 in VITAL in the late summer, in December of 2020, as we've previously reported, we met with a formal type B meeting with the FDA. The result of the meeting was very positive. Our interaction resulted in shared alignment with the FDA on co-primary endpoints for the VITAL pivotal trial.

As a reminder, the endpoints are the proportion of RDEB wound sites with greater than 50% wound healing from baseline, comparing treated and untreated wounds at week 24 or month 6, determined by direct investigator assessment. And the second co-primary endpoint and pain reduction associated with wound dressing change assessed by mean difference in scores using the long baker validated faces scale, comparing treated and untreated wounds also at week 24 or month 6.

This was a very important step forward, clarity with the FDA. We believe these are very appropriate and clinically meaningful endpoints, and we thank the FDA for their partnership in helping us advance the Phase III pivotal VITAL Study one step closer to patients.

Next, immediately upon alignment wI think the FDA and protocol amendment in January '21, I'm very excited to let you know that the fourth patient was identified, biopsied and treated in the VITAL study in February. In addition, as enrollment is ongoing and remains our top priority at Abeona for 2021, we are very pleased to report that patient 5 was biopsied earlier this week, and we anticipate their surgical transplantation at Stanford in mid-April.

Finally, with regard to patient accrual, our top priority, additional potential patients have been identified. They are being prescreened to determine their eligibility for study participation. As a reminder, the prescreening has a battery of test, but most importantly, the genetic screening to assure they meet the criteria of 2 confirmed C7 mutations, both with recessive inherited patterns causing RDEB.

Overall, we are very pleased with the progress against key milestones for the EB program, our lead program. The team's focus on operational excellence and laser focus, including Stanford, during a very difficult time, and we are on our way to meet our accrual goals for 2021.

Please recall that for the VITAL pivotal trial, target enrollment is anywhere between 10 to 15 patients. It is not patient number derived. But more importantly, it is the treatment of approximately 35 large chronic wounds in total. We believe the recent momentum of our type B meeting, patient 4 being treated in patient 5 now enrolled and on their weighted treatment in Q1, has us well on our way to complete our goal of accrual in 2021.

Last for EB-101 and even more exciting news, due to an increased interest from our KOL community from patients as the pandemic seems to hopefully slow and vaccines start to take hold across the world, we have selected a large academic EB medical center in the Northeast, that we are currently onboarding as site #2. As they are still going through their IRB approval, and we hope to have them up and running for the second half of this year and the second half of accrual of VITAL, I will not release their name just yet because I want to make sure they get through IRB approval, but we'll be speaking to you soon about that.

Even though, as I've stated before, we are on pace to finish VITAL in 2021 out of Stanford, we agreed to a second site for 2 main reasons. First, listening to our patients. The #1 barrier in 2020 for study participation was traveling due to COVID. We wanted to offer an option on the East Coast to make travel and logistics easier for some patients and families. Second, as we get closer to potentially commercializing EB-101, hopefully, in 2022, pending positive results, expanding HCP experience with EB-101 is necessary in meeting our goal as having EB-101 as the standard of care for all patients with recessive dystrophic epidermolysis bullosa in the United States.

Now I'll turn my attention to the other half of our gene therapy portfolio. Our adeno-associated virus or AAV-based gene therapy programs, namely our first program from an AAV standpoint is ABO-102, our program known as MPS IIIA. As a reminder, we completed target enrollment in ABO-102 to Transpher A study for MPS IIIA in the fourth quarter of 2020, as was laid out in our corporate goals and milestones for 2020. To date, 19 patients have been dosed in the Transpher A study, including 13 patients dosed in the higher dose Cohort 3, 3 times 10^13 vector genomes per kilogram, the dose that we believe is safe and efficacious, and we'll be speaking to the FDA about later this year.

We also communicated in our third quarter call that we'll continue to lead the Transpher A study open to patient enrollment, at least through our type B meeting with the FDA for 2 main reasons: one, the lack of treatment options for patients afflicted with MPS IIIA; two, based on the positive safety and efficacy results we've seen from the higher dose cohort, we thought it was the right thing to do to leave it open for patients.

In addition and just last month, we were very excited to share the latest interim data from Transpher A, which continues to provide hope for patients with MPS IIIA as well as their families. The updated results which were presented during a late-breaking platform oral session at the WORLDSymposium, and again, a few days later, with many of you at our subsequent investor webinar, continue to suggest that ABO-102 the potential to be a life-altering treatment option for children with MPS IIIA.

The data from Transpher A demonstrated that the neurocognitive development was preserved with a normal range of nonafflicted children, 2.5 years to 3 years post-treatment with ABO-102 Cohort 3 dose in the 3 youngest patients treated. These patients, as a reminder, were treated at ages 27 months, 19 months and 12 months respectively, and they are now at the age ranges of 3.5- to 5-plus years chronologically.

Why this is so important? Their current age, 3.5 to 5 plus is a critical time point as we see the natural history of MPS IIIA disease, usually by about years neurocognitive development declines without a therapeutic intervention, which none being approved thus far today.

We are very excited about this data and our next steps are as follows: We requested a meeting, as we told you we would, with the FDA in January of '21, to discuss this data and next steps for a potential BLA path with the FDA. A meeting date has been set in June, we appreciate that from the FDA as they're quite inundated with COVID. We intend to discuss with the FDA whether the Transpher A dataset could serve as the backbone for a BLA submission with natural history data as a viable control arm. We believe this is most important for any development in MPS disease for all life science companies as these children are being demented by the day, and it is not feasible to give them a placebo. We're excited, and we're hopeful to review the data with the FDA at our upcoming meeting, and we believe we have a viable plan forward.

Also, a reminder, in mid-2020, we met with the EMA under the PRIME designation to discuss a viable path toward a marketing authorization in Europe for ABO-102. We expect the next steps from our FDA interaction in June to filter into our development plans, and we will again further refine our pathway with the EMA and have a discussion with them, probably targeted later in the second half of this year.

Moving on to our third in-clinic program, Transpher B. 11 patients -- I'm sorry, Transpher B in MPS IIIB. 11 patients have been dosed to date, including 4 in the higher dose cohort. A reminder here, the higher dose cohort here is 1 times 10^14.

In February of '21, also with the WORLDSymposium, new interim data was accepted as late-breaking oral presentation for Transpher B study. The updated results were encouraging. They show treatment with ABO-101 was associated with a dose-dependent and sustained improvement in CNS, cerebral spinal fluid heparan sulfate and systemic biomarkers, including plasma and urine heparan sulfate and urine like glycosaminoglycans, known as GAGs. We look forward to continued follow-up to assess ABO-101's potential to preserve neurocognitive development, the gold standard and need for MPS patients in IIIB later this year and also onward into 2022 as the data of neurocognitive assessments matures.

Finally, with regard to Transpher B, we will not be concluding patient accrual in Q1 as prior stated on our last quarterly call. We're currently working on the next product stability testing time point, for the clinical product being used in Transpher B, which, as I'll remind you, is the nationwide children's hospital product. After completion of this testing, we'll assess our options forward to potentially treat the remaining patients in need. We have plan to update you, the investment community, as soon as we have clarity on time lines and after we have discussed with our investigators, patients and the MPS community.

Next, turning to overall corporate and organizational progress in '20, we entered into 2 strategic partnerships with patient gene therapies that unlock near-term value in earlier stage noncore assets for Abeona and provide us opportunity to share in the future success and milestones of these programs. The deals provided patient gene therapies delights to ABO-202 and AAV-based gene therapy for CLN1 disease or infantile Batten disease. As well as certain IP rights, material and know-how relating to a potential AAV-based gene therapy for Rett syndrome. Most importantly, this deal -- this transaction put this asset in the hands of a partner that will expedite development much needed for these patients with, again, the highest of unmet need.

Next, as we look to add to our leadership and operational experience in the next phase of Abeona, our operational excellence phase, I am very excited to announce that this morning, we appointed 2 new independent members to our Board of Directors, further strengthening our leadership, helping us drive our path forward for future growth, enhancing our corporate governance and assisting in creating additional shareholder value. I want to comment on these Board members and their selection. It was based on their relevant and diversed experience, making them the right partners for my current management team oversight for both near and longer-term objectives of Abeona, specifically operational experiences in life science.

Finally, let's review the key anticipated milestones for 2021 and what you can expect from us. The accomplishments over the past year and into our fast start Q1 2021 give us very positive momentum as we focus on execution and continue to move our programs in clinic closer to patients.

Number one, in 2021, we plan to complete enrollment in the EB-101 Phase III pivotal VITAL Study, assuming no more COVID-related delays, and if you could see me, my fingers across. Based on the current expectation for completing patient enrollment in '21, we anticipate -- I'm sorry, we anticipate top line data for the VITAL Study will be available in mid-2022, which will be followed immediately by a BLA filing, assuming positive results.

Milestone 2, regarding our program in MPS IIIA, ABO-102. We have already put a check in the box of our first objective, assuring a type B meeting with the FDA to discuss our path forward. As a reminder, that will be in June of '21. In addition, we expect the first lot of Abeona produced clinical-grade product for ABO-102 to be released in the second half of this year, a very exciting milestone for our group. Under Jay Bircher's leadership and our tech ops group in Cleveland, Ohio. I often say we believe this is the lifeblood of our organization.

Finally, and later this year, we expect additional follow-up visits and neurocognitive assessments of both the patients, the younger patients who have been treated in Cohort 3, the first 3 patients as well as patients 4, 5 and 6. As a reminder, in Cohort 3, we have dosed up to 8 patients and the 2-year neurocognitive assessment is a very significant time point because these patients will be chronologically past that 3-year time point, where we can compare to the natural history of the progression of the disease in an untreated patient. We'll let you know more about our exact data dissemination plan when we're clear on exactly what congresses will target.

Number three, regarding our last in-clinic program, ABO-101 and MPS IIIB. We will work timely through our product stability testing time points for clinical product nationwide. Once we have the completion of our stability test, we'll assess our options for treating the patients remaining from Transpher B. Also, it's important to note that the FDA feedback that we will receive for MPS IIIA program in June will be a significant learning for the path forward for all MPS programs and will absolutely filter immediately into our MPS IIIB plans as well as will continue to guide our EMA plans for both IIIA and IIIB. Finally, we can expect to see additional clinical data and updates from Transpher B later this year, some early neurocognitive data, and we will let you know as our congresses are designated when that data dissemination will occur.

Let's now shift briefly to our work on adding new in-clinic programs from our rich preclinical pipeline. We're focusing resources, and we have started to begin to prioritize our ophthalmic indications, following a strategic prioritization and review. On the back of the progress from our AAV programs in MPS IIIA and IIIB, we're conducting preclinical research assessing some of our in-house and partnered AAV capsids in fixed undisclosed eye disorders.

We are executing toward IND-enabling studies in 2 to 3 indications in '22. We'll pick 2 to 3 of the 6. Just to give you an idea, while these indications are undisclosed right now as these are very, very busy spaces, and I want to make sure we have more concrete path forward. Each of the indications in the U.S. alone represent about 5,000 to 15,000 patients. So we're starting to parlay our success in AAV programs to some bigger patient markets with tremendous need. We expect to have results from some nonhuman primate studies in Q2, Q3 '21, and we plan to examine these results and have them guide us into proof-of-concept studies in the H2 of this year. Ultimately, these will lead us into pre-IND meetings and toxicology studies in 2022.

I thank you for your time today. I look forward to a substantive conversation in our Q&A. And before that, I'm going to turn it over to our Chief Accounting Officer, my right hand, Ed Carr, so he could take you through the full year financial results. Ed, please take it away.

Thank you, Michael. I would like to remind everyone that we have recently filed the Form 10-K, which is available on our website and where you can get the details on our financial results.

In summary, our cash, cash equivalents and short-term investments, as of December 31, 2020, totaled $95 million compared to $129.3 million as of December 31, 2019. We did not use our $150 million at-the-market or ATM program during the fourth quarter of 2020. We believe we have sufficient cash resources to build on our momentum and fund our current development and operating plan through the achievement of key anticipated milestones, including multiple potential regulatory submissions. For the full year of 2020, net cash used in operating activities was $35 million compared to $62.8 million for the full year of 2019.

And with that, I will now turn it over to the operator for Q&A. Holly?

(Operator Instructions) Your first question for today is coming from Maury Raycroft.

Congrats on the progress. This is Maury from Jefferies. First question is just on MPS IIIB. So just wondering if you can bookend, how long will it take to do the product stability testing and wondering if that was requested by FDA? Was it something observed with the product? Or was it just the right time to do the stability test?

Yes, Maury, I'll take that one. And if we need any smart science talk, I'll definitely turn it over to Jay. No. IIIB, as planned, nationwide product has expiry on it. So we have to make sure that we're doing our stability planning. This has not been asked for by the regulators, specifically. But of course, it's part of our plans, part of our stability protocols and our IND. So we -- the nationwide product, our last lot late last year, hit expiry. So what we're doing is we're testing stability, making sure our product still has the appropriate integrity, and we're looking at some final patients for Transpher B. So that's the impetus of that.

As far as timing, that's more imminent than long term. I didn't put timing out exactly today because I don't want to have to take it back anymore. Right now, we're looking at some test in-house versus out. And we should have some time lines very, very soon here. Jay is working quickly on that because we know we've got some patients waiting. So I predict that will be more imminent than longer term. But as soon as we know, we'll update you.

Got it. Very helpful. And then second question is just checking for EB-101. If that second site comes on board second half could that -- could adding that site accelerate time line some? And would any patients or data from that site be included in the filing submission? Or do you want all the data to come from Stanford? I guess how are you guys thinking about that?

Yes, Maury, great question. So absolutely on both. The answer is, as we've said all along, really excited. We're almost at -- I don't want to say that loud, right? I'm a superstitious guy. But we almost, in quarter 1, have as many patients on board as we've had in all of 2020. And to be fair to the team, we know '20 was a tough year with the pandemic.

So we think we can finish out of Stanford. Remember, we could be done as early as 10 patients. And we'll think about leaving it open a little bit if there's someone sitting on the bench. We want to get all patients in need. Right now, we think we're projecting somewhere -- it depends on how many wounds we get for patient. So I won't project, right away, but we could be done as early as 10 patients. So 5 takes us halfway there.

No doubt that the second site, which I'll announce as soon as they're through IRB for disclosure purposes of University, that will get us -- definitely expedite time to fulfill accrual. And yes, those results will be as important part of the BLA filing, any patient under VITAL-treated, they're part of VITAL, will be part of the BLA. All correct.

Your next question is coming from Ram Selvaraju.

This is Ram Selvaraju from H.C. Wainwright. Firstly, relating to the EB-101 program, I was wondering if you could confirm that you are effectively going to stop enrollment once you have the 35 chronic wounds to assess. In other words, it's not specifically dependent on patient number. And once you have 35 accessible chronic wounds, you would help enrollment at that time or if there's a possibility that you could continue to enroll additional patients after getting that number of wounds?

Yes. I'll take that one, Ram. Great question. So the answer is, we use the word approximate for a reason, 35 wounds important for our stats plan, right, as part of our pivotal. So yes, that's the target. Give or take, probably in the direction, we think we've got to hit 35, but we could go a little more.

What we want to be mindful, Ram, is to make sure if they have a patient in the queue, we're not going to refuse them. But it is -- you're exactly right. It's not a specific number of patients. The stats plan is based on number of wounds.

So that's why we have a range of patients because knowing what our rate is, we'll be able to get there between 10 and 15. So you're right, it is 35 wounds, it's approximate. I won't say we'll definitively stop at 35. It depends if we have a patient in the queue, but we won't do 50, for example. We'll be close to that number.

Okay. And just to clarify, I think this is following up on Maury's question earlier. Can you kind of give us some more granularity on how many patients are kind of in screening or in prescreening meeting that (inaudible) know where they are and started the process, but they're not all the way through to being able to actually enter the study? Just give us a sense of how many patients we're looking at there.

Yes, sure, guys. And I know this came up in the past, and I appreciate you're asking that, Ram. I'm going to just address this to the team, to be fair to the team prior to me. Stanford has a prescreening study open where it filters many patients into different studies they have opened based on genetic testing, okay? So we did have some patients on the bench guys in 2020 in fairness to our prior team, who, based on pandemic were unwilling to travel, moved on to different studies that potentially are open. So we did lose some people along that line.

Right now, Ram, we've got -- I get nervous saying the numbers because I don't like to jinx, but we do have a handful of patients that could potentially be patient 6. I mean we look at the next patient. Right now, we're screening potentially 2 to 3, if we can get them to the site. Whether they're all willing to travel, we've contacted them. We're in communication. We've got 5 biopsies. So I like to talk about definitive, 5 is on their way to treatment. But right now, patient 6, we're looking at 3 different individuals. And that's obviously, again, at a Stanford before we have the second site up and running.

But we're always adding every month, patient seminars. I hired a Chief Patient Officer and Jodie Gillon. We're full-court pressed. We're doing this all the time. So next week, we could find 2 or 3 more we can vet. So there's even some international allowances now since Stanford IRB has opened up on that. So we're looking at patients even out of Mexico, potentially Canada, Germany. So exact numbers tough to give you, but I could tell you right now in the U.S., there's 2 to 3 we're assessing. We could go 3 for 3. We could go 0 for 3. I don't know what their prescreening will look like. I hope that gives you clarity, Ram.

No, that's very helpful. And then lastly, on EB-101. I was wondering if you could provide some commentary regarding the correlation between wound closure, degree of wound healing and the extent of pain relief that you anticipate seeing based on the prior data. How tight is that correlation? Is complete wound closure accompanied by like a 50% reduction in pain relief, significantly greater than 50% reduction in pain relief? Just give us a sense of what you're expecting to see with regard to the correlation between wound closure, wound healing and pain release?

Yes. Another good question, Ram. So look, I think they're highly correlative. Can I give you a perfect number? The answer is no, but I'll use data to tell you what was the backbone of why we drove such endpoints with the FDA and had these conversations. First and foremost, I commend the FDA on saying, hey, look, we understand that Abeona, you're not treating the same wounds as maybe non-EB diseases or even other EB diseases. You're treating the most problematic wounds, large and chronic, wounds that can no longer heal themselves, lead to significant opioid use, quality-of-life issues and, ultimately, mortality.

So if you look at our Phase I/II, I'll be evidence-based in my decision, Ram. Look at our Phase I/II, that's not exactly how we're measuring pain in the Phase III. In the Phase III, we're measuring it in a much more specific, objective way. Remember, the Phase I/II study was an academic study.

But if you look just at patients at baseline, I'll tell you the difference. From the Phase I/II, 59% -- I think the number was in the 50%. At the top of my head, and Greg can correct me, 59% or 53% had pain at baseline, but that was a global impression of pain, not specific to just one wound being treated. What we're testing is an actual wound that we treat versus the control wound in VITAL. So when the patients were enrolled in the Phase I/II, 50% to 75% wound closure, which we had in a lot of patients, we were over 90% of 50% wound closure, right?

It was highly correlative. If you look at the results, you can see it in our investor deck, we're taking pain down to 0 in a lot of people. Some had some trickle back pain. But again, the Phase I/II was looking at global pain. So you couldn't delineate that maybe that wound felt great, but maybe they had an esophageal stricture or the dilation surgery a week before, and that could be muddied in the Phase I/II result.

That's not a possibility in the Phase III. In the Phase III, the endpoint is specific wound being treated at time of dressing change, 6 months, onetime point, versus the exact same pain scale self-administered by the patient in front of the physician on the untreated wound.

So I can't give you a perfect number, Ram, highly collative it's -- we think -- we know it's the most important morbidity condition today. Ultimately, we want to change mortality of this disease, and we think we've got to cover large chronic wounds as much as the body surface area for years, for long periods of time to do that. But again, that's not what VITAL is going to show us. We're going to see that years down the road of covering these wounds through, hopefully, registries once we're approved.

But highly correlative, can't give you a perfect number, but these wounds when unclosed, almost all patients, I'll quote Jean Tang here, almost all patients are in excruciating pain during dressing change with these types of wounds. I hope that gives you some color of the difference between the Phase I and the VITAL trial, the evidence from the Phase I/II and how we think about pain as really the most important morbidity to improve upon for these patients.

No, that's very, very helpful. Just 2 other very quick ones for me. With respect to the Transpher A study, can you comment on what you expect, ultimately, the average age of patients to be at time of enrollment in Cohort 3? And then lastly, do you have any updates on the situation with REGENXBIO?

Yes. What I'll do is I'll take the second question first on REGENXBIO, and then I'm going to turn it over to some of the muscle I have on the phone because I'm super proud of this management team. I'll turn the MPS question over to Juan. He can tell you about the age for the enrollment.

So from a REGENXBIO standpoint, guys, not a tremendous amount more I can give you. We will continue to update you. But the arbitration is ongoing. Obviously, it's an ongoing legal proceeding. The status is included in our 10-K. We had an arbitration hearing held earlier this month. The tribunal has not yet issued its opinion. For an overall overview of the proceeding and the extent to comment, please refer to the 10-K. Also, it will be on our website. So again, guys, still ongoing. I can't tell you about the results, but I can tell you that the arbitration hearing was held earlier this month.

Juan, why don't I turn the age question for MPS IIIA, over to you, please.

Yes. Thank you, Michael. Yes. In Cohort 3, we have enrolled so far 13 children. And since we modify amended protocol to enroll younger children with a younger than 2 years or with an IQ higher than 60, we have 8 patients with this criteria. And the ages range from 8 months to 33 months in that particular group. So we expect in the future, the next, I mean, the children that we are still -- that might still be enrolled and treated in the trial will be within this same range.

(Operator Instructions) Your next question is coming from Mani Foroohar.

This is Mani Foroohar from SVB Leerink. I'm not sure I missed the detail, I want to circle back on the Transpher B question. Can you give us a little sense of is this an assay development challenge? Is it like a question on data management, sort of SOP development sort of? Give us exactly sort of what needs to happen to put your foot back on the accelerator there.

Yes. So Mani, I'll remind you, the -- a couple of things. Transpher B, we are 4 patients shy of our Cohort 3 target. So we have patients in the queue. And the most important thing for us is to get those patients treated.

That being said, no, not an assay development. We have product -- the nationwide product is the product being used in Transpher B as well as Transpher A. Jay and team will be making our own Abeona product for MPS IIIA, second half of this year, another major milestone for us. But the product has patient -- I'm sorry, product expiry dates. And our last lot expired later into Q4. So what we're doing is we're doing a next stability test to look at our options to move forward. So it's not an assay development. We're just looking at a stability testing on a lot of product that was expired nationwide, and we're going to see what our options are for the final few patients to be treated.

Your next question is coming from Kristen Kluska.

This is Kristen from Cantor Fitzgerald. The first is, I know the data is hot off the press here from WORLD, but wondering if you could share any feedback that you heard from physicians or the different patient advocacy groups since your presentations, particularly from the youngest patients in the high-dose cohort and MPS IIIA.

Kristen, good to hear your voice, and there is no one better on this phone to do it than maybe the most knowledgeable guy known in MPS and neurocognitive programs. Juan, why don't you please give a little bit of maybe some verbatim and some feedback from some of the KOLs around the world and the reaction to the IIIA data and even the IIIB data, please?

Yes. Thank you, Michael. Thank you, Kristen. Yes, the data has been -- the feedback has been very positive. I mean when we announced the data regarding the younger children, some of them were -- those with 30 months, 36 months after treatment, reaching the age where children in network history are declining significantly and getting these children is still gaining skill that's been received very positive by this by both the expert and the key opinion leaders and the patients community.

Yes. And that -- we've been reviewing this with some physicians that are recurring patients was all around the world. And yes, they are very happy and we are looking forward for new data coming this year in order to confirm that this positive development continues in these children and the others that were recently treated, and we are expecting additional data along the year.

And Kristen, I would be remised, and I'd get in a lot of trouble if I don't say this, my Chief Patient Officer is not on the phone with us today, Jodie Gillon, just texted me and said, patient groups are thrilled and encouraged, that's verbatim. So I'd be in a lot of trouble if I didn't share that with you. So thank you, Jodie.

So glad you won't be getting in trouble then. So as you think about the next potential chapter in ophthalmology, and I know you haven't disclosed these indications. Maybe could you speak and remind us about the differentiated aspects of your AIM capsid versus sort of the evidence and results we've seen across the field so far? And I do recall that you shared some initial data, I think, at ASGCT maybe 2 years ago back when we had conferences in person. So if you could remind us about some of the initial findings, and I know more work is ongoing here.

Yes, Kristen, I appreciate the enthusiasm on the question. I'm excited about it, too. And my preclinical team, (inaudible), are teaching me about this all the time. I think that the short answer is, I wish I could. I think we're a little early. I don't want to put the cart in front of the horse, which is why we haven't disclosed the indications. We'll have some nonhuman primate data, which my scientists tell me all the time is a very important proxy for -- when we're looking at the eye and the eye disorders. We'll have that in the second -- I'm expecting to have that second quarter, Kristen. So I can promise you a follow-up to talk about the NHP data as soon as we have it. But I think it's a little early to talk about the results.

What I can tell you is we are using some of our capsids from our partnerships we're also using some of our capsids from in house. And the goal here is to see -- we're also assessing administration, right, power retinal, subretinal endometrial. So we're trying to see which capsids find a home to the cell best, but I think it'd be a little early if I was to give you kind of what we're seeing because I think that nonhuman primate data is very important.

So we're enthusiastic. My scientists have some really good proof of concept, if you will. But I want to wait until we get the nonhuman primate data before we boast about it a little bit, if that's okay.

There are no more questions in queue.

Well, with that said, I want to take a moment here and first thank the investor community for their support, their interest. It's always nice to spend some time with you, and I appreciate the questions. They're very thoughtful and appropriate. So thank you very much.

I'll continue to ask you to hold us accountable. That's important. That's an important thing that we all take very seriously here at Abeona because we're accountable to delivering to these patients.

I want to take a moment to say how excited I am to be -- to have the privilege to be the next Chief Executive Officer here at Abeona. And I want to make sure I thank our stakeholders. The most important -- well, they're all important. So I'll take that back. But super important, our patients and the courage they have to be part of these trials.

But I want to take a moment and thank my team. People -- I won't name everybody because we'll be here for a while. But my management team, who has really been steadfast, our employees, steadfast. Through a lot of changes that you guys know occurred in 2020 with Abeona, but they stayed mission-focused. We had our most productive maybe quarter yet in Q4. We had an incredibly successful type B meeting. We have worked with the agency, brought increased talent in, and we've got our type B meeting on the books for MPS IIIA. We continue every day to make milestones. And I really model and I'm super impressed by a group of people and their commitment. And even the pandemic hasn't slowed them down. So again, please hold us accountable. It's a year of operational excellence. We need to bring this science to fruition for patients.

And I'll leave it at that. Just a sincere thank you to my team, to my employees, to my Board, to patients, stakeholders, our KOLs and investigators and to you, the investor community for your continued support and your open and honest dialogue. Thank you.

Thank you, ladies and gentlemen. This does conclude today's conference call. You may disconnect your phone lines at this time, and have a wonderful day. Thank you for your participation.