Good day, ladies and gentlemen and welcome to the Abeona Therapeutics Fourth Quarter and Fiscal Year 2019 Conference Call. [Operator Instructions] At this time it is my pleasure to turn the call over to your host for today, Mr. Scott Santiamo. Sir, the floor is yours. .

Thank you. Good morning and welcome, everyone. Today's call will be led by João Siffert, our Chief Executive Officer. Following João, Christine Silverstein, our CFO, will review our financials. .

Before I turn the call over to them, I need to remind our listeners that remarks made during this call may contain forward-looking statements that involve risks and uncertainties. Forward-looking statements on this call are made pursuant to the safe harbor provisions of the federal securities laws..

Information contained in these statements is based on current expectations and is subject to change, and actual results may differ materially from forward-looking statements..

Some of the factors that could cause actual results to differ may be found in the company's annual reports on Form 10-K and quarterly reports on Form 10-Q, filed by the company with the Securities and Exchange Commission. These documents are available on our website, abeonatherapeutics.com. .

With that said, it is now my pleasure to introduce you to Dr. João Siffert. João, you have the floor. .

Thank you, Scott. And thank you all for joining us today for a call on fourth quarter and full year 2019 business highlights. For 2019, it was a year of tremendous progress across our gene and cell therapy programs, and I look forward to reviewing our accomplishments before turning the call over to Christine to review our financials..

Before proceeding, it's important to reflect upon the COVID-19 global pandemic. We are monitoring this rapidly evolving situation very closely, and recognize that may impact some of operations. Travel restrictions as well as concerns about viral exposure may interfere with patient screening, dosing and follow-up across our studies.

We have also conducted a risk assessment on our manufacturing operations. And so far, we have no immediate concerns..

We will continue to monitor the situation closely, while continuing to advance the development of our innovative medicines for patients in significant medical need..

With that said, I'd like to start with an update on the progress we've made with our lead clinical program, EB-101 for recessive dystrophic epidermolysis bullosa or RDEB.

As a reminder, RDEB is a highly debilitating rare genetic skin disorder caused by mutations in the Collagen VII gene that makes patients skin very fragile, easily blistering and tearing at the slightest contact. People with RDEB developed widespread wounds that are often large and chronic and can cover a significant portion of a patient's body.

Patients with higher wound burden are at a risk for systemic complications that may include infection, [ melt attrition ], anemia and even skin cancer.

There's currently no effective treatment options for these patients, and they rely on palliative care that primarily consists of painful, time-consuming bandaging and often requires prescription opioids to manage the pain..

EB-101 is an autologous gene therapy cell -- gene-corrected cell therapy that restores normal function of Collagen VII, is delivered through 40 square centimeter sheets comprised of gene-corrected keratinocytes in their progenitors and they are transplanted onto open wounds..

In January, we began what we expect to be a transformation year at Abeona with the initiation of the VIITAL study, our pivotal Phase III trial evaluating EB-101 in RDEB.

And today, I'm very pleased to share that just this morning, we announced that we have treated the first patient in the VIITAL study, which is our pivotal Phase III trial evaluating EB-101. The patient is doing well, following successful transplantation by the team at Stanford University Medical Center..

The successful dosing of the patient, the first patient in this study is an important milestone for this patient community that is the RDEB community for Abeona, and also for the Stanford team which began this effort over a decade ago.

Patients with RDEB can now look forward to potentially having effective treatment options in the foreseeable future with the Abeona's product uniquely positioned to address their significant unmet needs..

A concerted effort by the Abeona team has enabled us to successfully execute the multi-step EB-101 manufacturing process at our Cleveland facility. From transduction to cell growth and expansion to packaging, and transport to clinical site for transplantation, back on to the patient.

The Phase III study process closely parallels the potential real-world application of EB-101, and it's an important step towards commercialization of this product in the future..

The pivotal study is a randomized clinical trial assessing EB-101 in 10 to 15 RDEB patients with approximately 30 wound sites treated across all patients in total..

We have prescreened most patient subjects for the study and are working closely with our collaborators at Stanford in expeditious enrollment. We have also began preparations for adding a second trial site on the East Coast and remain focused on advancing enrollment as soon as the third quarter that we plan to obviously provide updates before then..

Given the overwhelming unmet medical need of RDEB patients and based on data collected from a Phase I/II study, we continue to believe EB-101 has significant potential to improve the lives of patients with RDEB.

EB-101 is the only therapy in development that has published data confirming long-term healing of large wounds and also meaningful pain reduction.

Successful wound healing following EB-101 treatment may also provide a meaningful reduction in time and expense spent on wound care and lower reliance on opioids and other analgesics, potentially improving patients' quality of life. .

As we look ahead to bringing EB-101 to patients once we receive regulatory approval, it's important for us to briefly discuss our commercial plans.

Our manufacturing facility, The Elisa Linton Center for Rare Disease Therapies is a fully functional gene and cell therapy facility featuring cGMP capacity and laboratories to support CMC development and analytics.

Our flexible, scalable manufacturing process allow Abeona to meet the demand for our pivotal EB-101 trial and eventually, also meet the demands for a commercial launch without having to exclusively rely on CDMO. .

Moving on to -- moving on from EB-101, I'd like to next review our programs on MPS IIIA and IIIB, following the recent presentation of additional positive interim data from both studies of the WORLDSymposium in February.

MPS IIIA also known in Sanfilippo syndrome A is a rare lysosomal storage disease, primarily affecting the central nervous system, starting in early childhood..

The Transpher A studies are ongoing 2-year open-label dose escalation Phase I/II global clinical trial assessing ABO-102 for the treatment of patients with MPS IIIA..

ABO-102 is an AAV9 therapy clinically proven to deliver a functional copy of the SGSH gene to the CNS in peripheral organs by a single intravenous infusion, with the goal of correcting the underlying enzyme deficiency, thereby preventing accumulation of GAGs and preventing the rapid neurodevelopmental and physical decline pivotal of MPS IIIA patients..

Our most recent data update at WORLDSymposium showed that 3 patients in the highest dose cohort, who were treated early, continue to show improved neurocognitive skills up to 2 years post-treatment as compared to natural history of disease progression.

These results are important as they are consistent with a well-established knowledge, the early treatment provides the best chance to preserve neurodevelopment of children with a neurodegenerative disease..

We are encouraged to see clear and sustained dose-related reductions in disease-relevant biomarkers, including CSF heparan sulfate, demonstrating long-term biologic activity of ABO-102. .

Most importantly, the treatment remains well tolerated through long-term follow-up, ranging from 15 to 45 months post-dosing with no treatment-related serious adverse events reported to date..

This study has enrolled 14 patients to date across 3 dose escalating cohorts, and we have additional subjects in screening with others identified. Of course, as discussed, study enrollment may be disrupted by COVID-19. We expect to add sites in the U.S. and Germany to complement our existing sites in the U.S., Spain, France and Australia.

On the regulatory front, we're pleased to receive EMA PRIME designation for ABO-102 in December of 2019, adding to the RMAT, Fast Track, rare pediatric disease designations as well as orphan drug designations in both regions..

We continue to actively engage with the FDA and EMA and plan on providing a regulatory update in the second quarter with a discussion on the path forward for this program.

Transition now to the MPS IIIB program or Sanfilippo syndrome type B, and other rare devastating fatal lysosomal disease with no approved treatment, which leads to neuro developmental decline, other systemic manifestations and ultimately, early depth..

I'm pleased to report continued progress with our Transpher B study, an ongoing 2-year open-label dose escalation Phase I/II study evaluating ABO-101 gene therapy with the main objective of assessing safety and neurodevelopment. The treated -- we treated the first patient in the high-dose cohort in January for a total of 8 patients treated to date.

Screening across sites remain active and look forward to start sharing enrollment updates and additional interim data. From the study in the second half of this year, we of course, expect additional dosing of younger patients, but these, of course, are provided. There are no further disruptions from the COVID-19 pandemic..

Last month, investigator from Nationwide Children's Hospital presented encouraged data on this Transpher B study at WORLDSymposium, highlighting the results from the first 2 cohorts, which demonstrated initial improvement in disease-specific biomarkers.

Specifically, there was a decrease in heparan sulfate in cerebrospinal fluid, reductions in plasma in urine heparan sulfate and GAGs and reduction in patient liver volume, all reported at the symposium..

Together, these data suggest that treatment with ABO-101 may prevent the accumulation of GAGs, which are directly associated with the NAGLU enzyme deficiency..

The biological activity of ABO-101 observed to date is consistent with that observed for the ABO-102, which is for the Sanfilippo A. We're hopeful that improvement in disease-specific biomarkers may translate to improved patient outcomes in this devastating disease and look forward to providing updated data later this year. .

Now shifting on to our preclinical assets. I'm pleased to report that we have made progress in our AAV9 program for CLN1 or infantile Batten disease, a rare fatal inherited nervous system disorder, generally presenting childhood with seizures, visual loss and neurodevelopmental delay..

In 2019, we had announced FDA clearance of our IND for ABO-202 in CLN1 as well as FDA Fast Track designation for this program. We have since successfully developed a process to manufacture this gene therapy for the Phase I/II clinical trial at Abeona in Cleveland.

This comes in the wake of a CDMO manufacturing challenges that precluded us from moving forward into the clinic..

I'd also like to briefly share an update on our novel proprietary AIM AAV capsids library. In January, we announced the issuance of the U.S. patents for AAV204, both for the capsid itself as -- in composition of matter, and also methods of use.

This achievement strengthens our licensed IP and recognizes the unique qualities of this gene therapy delivery method..

We're excited by the potential of our AIM capsids to more efficiently target various tissues such as the CNS, including the neuroretina, muscle and other tissues to deliver payloads aimed at addressing variety of devastating diseases..

Additionally, we're exploring how our next-generation AAV technology could treat patients who have existing antibodies to wild AAV serotypes and potentially permit retreatment of patients who received prior gene therapy with certain AAV vectors..

We continue to characterize additionally in capsids, in nonhuman primate experiments and continue to receive interest from potential partners..

Finally, from a corporate standpoint, in December, we announced the closing of the underwritten public offering of $103.5 million gross proceeds, strengthening our financial position. We expect this capital to provide us with the necessary resources to complete the VIITAL study for ABO-101 and continue with other clinical and preclinical programs..

A conclusion from the strategic review completed in 2019, that is in the best interest of Abeona to continue to develop our core pipeline products in the near term, while continuing to engage with prospective business partners..

With this additional funding, we believe we're well positioned to realize the potential of our innovative gene therapy pipeline, facilitated by a talented group of researchers, clinical developers and the staff, who were fully functioning in an independent manufacturing facility under the leadership of Jay Bircher, who was recently promoted to Chief Technical Officer.

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Before I turn the call over to Christine, I'd like to thank the entire Abeona team whose hard work and dedication enabled a remarkable progress this year. In addition, we remain deeply grateful to the patients, families, clinicians, researchers and patient organizations who have made our work possible.

We look forward to continued partnerships and remain committed to delivering approved therapies for patients who most desperately need them..

With that, I'll now turn over to Christine.

Christine?.

Thank you, João. I'd like to remind everyone that we have recently filed our Form 10-K, where you can find all the specific details on our financial results. But in summary, our cash, cash equivalents and marketable securities as of December 31, 2019, were $129.3 million compared to $47.9 million as of September 30, 2019.

The increase in cash, as João mentioned, of $81.4 million was driven primarily by the $103.5 million gross underwritten public offering..

Net loss was $0.30 per share for the fourth quarter of 2019 compared to $0.36 per share in the comparable period of 2018. For the 12 months ended December 31, 2019, net loss was $1.51 per share compared to $1.19 per share in the same period in 2018. That's the summary of financials..

Before I turn it over back to João, I want to address my pending departure from the company. As you may have read in the filing last night, it is with the very heavy heart that I have tendered my resignation as CFO, effective March 31, 2020.

I will further be available as an adviser to ensure a smooth transition for the company and further as a board member..

Ed Carr, our Chief Accounting Officer, has been an instrumental part of the financial operations built over the last year. And having worked closely with him, I strongly believe that Abeona and Abeona's finance team remains in very capable hands with his leadership. .

I'll now turn it back over to João.

João?.

Thanks, Christine. Over the past 3.5 years, Christine has made numerous contributions to the Abeona organization and has helped position us for successful 2020.

In her -- passion for Abeona, in its mission to transform the lives of patients with serious diseases, we're pleased to announce that Christine has been appointed a seat at our Board of Directors. Therefore, while Christine is no longer serving in her current capacity as CFO, her contributions to the Abeona and its mission will continue.

And I thank you, Christine. .

In summary, we've achieved interim milestones in the fourth quarter that have Abeona well positioned to bring long-term value to our shareholders and turn hope into reality for our patients and their families. .

Further, treating the first patient in the first -- in the Phase III trial, the study is a tremendous accomplishment for all involved, including the dedicated teams at Abeona, and our close collaborators at Stanford University. .

Most importantly, we're very thankful for patients who volunteered to participate in this pivotal study, into the RDEB community who has been support of this program for many years. .

Thank you. And I'll turn over to the operator for questions.

Operator?.

[Operator Instructions] We will go first to Maury Raycroft of Jefferies. .

Congrats on the milestone today. And best wishes on next steps, Christine..

First question is on the patient dose.

You're probably not going to provide a lot more details, but wondering, if you can say, when the patient was first treated?.

And if you can talk about the wound size, number of wounds, and the history of wounds that the patient had, I'll start there. .

We somewhat circumspect about the lot of the details, obviously, for privacy and obviously, we'll provide general updates, but not a blow-by-blow update. But suffice it to say, this is a young man who had been actually a volunteer for the first study. And unfortunately, at that time, he could not be treated after a successful biopsy.

Because he had developed fever and infection. So he had actually been waiting for this study for quite a while. So that goes to show the level of unmet need and sort of the promise of this treatment. Patient -- this patient had, as you can imagine, many of the patients with RDEB have a very large wound burden.

We're able to treat as many as possible within the sort of the compliance of the study. This patient, unfortunately, still has more open wounds that could not be addressed in the single sitting. .

So as we mentioned before, our intention is to deploy a second study, which is not a registration study, but rather is a Phase IIIb study where we plan to offer redosing for patients who participated in either the Phase I/II trial or in the Phase III trial. So where they can come back to have some of their other wounds treated..

The actual process itself, manufacturing and deployment of the EB-101 sheets, and ultimately, the surgery and postoperative have been successful to date. So we're pleased for the patient. And of course, pleased with the execution of this whole process. .

Got it.

And for the additional patients prescreened and in the queue, can you say, if that's closer to 5 or 10 or 15? And anything else you can say about the baseline characteristics for those patients?.

Yes. So we have -- we, meaning Stanford has been running a prescreening protocol for quite a while. In fact, they prescreened patients for all the open studies, including some of the other RDEB studies, which is actually a nice process because they have all this kind of mapped out..

For our program, specifically, they prescreened 11 patients, as we've discussed. Our intention is to enroll somewhere between 10 and 15 patients depending on the number of wounds treated. So we're well positioned on that.

Of course, the theme that may be repeated frequently from over the next, maybe weeks and months, a lot of the plans now may be disrupted based on the COVID-19. That did not happen with the patient who was treated.

But for subsequent patients will depend a lot on local regulations, vis-à-vis the medical centers, whether or not they will allow elective surgeries and participation in clinical trials. So the U.S. agencies have not yet issued anything formal.

This morning, we got communication from the German authorities as well as the Spanish authorities providing some guidance on that. And of course, locally, the hospitals and, of course, ultimately, the investigators will make a decision..

So it's likely that some of the activities will need to be postponed. Don't have a lot of visibility yet on the EB-101 program. But for -- certainly for the European sites on the Sanfilippo syndrome, we anticipate a certain amount of delays in the treatment of patients..

Also for follow-up, the patients already enrolled in the trial, especially for the areas most affected by COVID-19, some of the immediate follow-ups will be, unless there's obviously a medical issue. But if it's a routine visit for the studies, a lot of these will be done online or by telephone. .

Got it. And one last question from me, just based on the cadence of treating patients in the Phase III.

So I guess, what's your plan for that, assuming that COVID-19 doesn't impact it too much? When will you dose the next patient? And then is there potential for you to try to ramp up and -- ramp up production and potentially, start treating 2 patients at a time?.

Yes. We have the capacity, and that was the plan, until now. So obviously, the exercise here is to be very mindful of -- because, just to backtrack a bit. So it takes about 4 weeks to process the biopsy into these gene-corrected cell therapy sheets. So the main decision here will be, if we were to biopsy a patient, because that's a procedure itself.

We want to make sure that the patient can then come back, when the sheets are ready for receiving the actual product. So that's basically the decision we'll have to make going forward. Obviously, patients who were biopsied, we are intending to treat, but patients who have not yet been biopsied.

This will have to be a very close discussion between Abeona, the investigators at Stanford, and of course, the medical center at Stanford, too, and the patients too. Families have to travel for this. So sometimes they may choose not to come for the visit. .

We'll go next to Mani Foroohar, SVB Leerink. .

And best wishes on you, Christine. Congratulations on getting a woman on the Board, although not exactly the mechanism we had thought..

You talked a little bit about expanding the IP and continuing to build your IP portfolio around your earlier stage preclinical pipeline opportunities, Batten disease, et cetera.

How do you think about putting resources to work in earlier stage, and earlier stage opportunities in developing preclinical assets, independently or for partnerships versus a pretty full docket of 3 programs already in the clinic?.

And then as a second question, how do you -- how should we think about the impact of -- or travel restrictions [ we have ] practical logistical issues in terms of the feedback you may get from the FDA and that conversation, has that in any way sort of slowed your progress on regulatory conversations? Or was that largely, a virtual non- in-person process to begin with?.

Yes. So 2 separate questions. So the first question related to the rank order of our pipeline priorities. So clearly, the clinical programs take precedence. That's where most of the effort and the impetus and the focus are currently deployed. EB-101 being the most active of them and certainly, the most advanced.

So there's -- the greatest priority is in EB-101, whereas, we obviously continue to be very focused on the 2 Sanfilippo programs, because these are enrolling patients. So we're obviously much more responsible for, for everything related to activities that involve patients. So these are the priorities.

At the same time, we believe that there's value in our preclinical pipeline, both in terms of medical value that is new programs that could address significant unmet disorders. But we are being very judicious of how advanced that.

So we're completing some of the characterization of the AIM capsids, so including the select nonhuman primate studies to round out the characterization of these capsids. And also advancing some of the preclinical programs within the confines of, obviously, Abeona here in some of our collaborators. So this is something that we have more control.

Doesn't require patient involvement, traveling, whatnot. These are much more of the scale of these investments and the effort obviously, commensurate to the level of development.

So whereas the bulk of the effort and resources are deployed to clinical programs, we have a very precise and focused, and circumscribed effort to keep these assets moving forward, whereas at the same time, not detracting from the clinical programs. This is important because there's value in these assets.

As I mentioned, both medical value, if you could pass forward on some of these programs that we're entertaining.

And also potentially, these could be parlayed into partnerships, which continue -- discussions are always ongoing and these, as you can imagine, in gene therapy, and there's a possibility that some of these partnerships may bring in resources to the programs that could be developed with other collaborators.

So we want to keep those active, as to enable us also to set these partnerships in bringing the most value out of this early pipeline. .

Separately, you asked about regulatory interactions. Much of the regulatory interactions with the FDA, in fact, over the past year have been virtual, either phone calls or correspondents.

The [indiscernible] division is overwhelmed with a lot of actually successful programs of gene and cell therapy have been quite successful over the past several years, and they're contending with a tsunami of new INDs and submissions and whatnot. So they've been quite judicious with granting live meetings.

So in that sense, in terms of the logistics, I don't anticipate necessarily, that this would delay any of the interactions.

But I do not have any visibility as to their internal demands now with COVID-19, which I'd assume, exist across the agency, but I don't have any -- I don't have direct visibility of this to, to how this will affect their operations. But we'll know more, or in the coming weeks. .

We'll go next to Kristen Kluska, Cantor Fitzgerald. .

Congratulations on the patient dosing announcement this morning. So now that your Phase III trial is up and running, how are you thinking about a commercial strategy at this point, both in the U.S. and ex U.S., I know the previous Chief Commercial Officer had laid out some initial potential plans for this.

And do you plan to reevaluate this, if you hire a new CCO?.

So a couple of questions there. Thanks for your questions. So yes, so obviously, yes, we start to Phase III. That's sort of -- the sort of set goal for deploying a commercial strategy.

And we have, have had, and this person continues, the consultant and commercial who had worked with our previous CCO, and we're actively recruiting for a new CCO to join the company. We have actually quite qualified candidates that we're very pleased with..

So this will be deployed. Obviously, the launch plan is of couple of years in the making. We have to start now. We have, in fact, started a few activities already, even late last year, we were already working on this. And at some point, we'll have additional hires in terms of the actual specific people in marketing and also health outcomes, et cetera.

So we're still very much focused on getting ready for launch, we'd be assuming the time lines that we disclosed, the completion of enrollment this year, data, first half of next year. So this is timing for commercial planning starts now. I hope that answers your question, specifically. .

Yes. It does. And just one follow-up here.

Could you characterize the primary endpoint of this trial? And how at 50% wound closure is going to translate for these patients? Meaning, to what extent does this reduce the risk of the comorbidities that lead to the serious adverse effects or mortality like the squamous cell carcinoma and infections? And how do you consider this, especially, since you are the only company that has the long-term data on hand from the initial trial?.

Yes. So I'll start with the first question. Obviously, the overall impact will depend on how many -- how much of the wound burden you can address. So I'll come back to that in a minute. But I'll answer by referring to the data that's been published. So the Stanford team published 2 papers, 3 papers, but 2 in the clinical trials of [ Phase I/II Trial I ].

Some time ago, I think, in 2017 or '16 in JAMA. And then most recently, they published a long-term follow-up paper at JCI. These are obviously 2 peer review papers of -- reputable journals. And what they described, and the data obviously, published, is that, first of all, patients have both durable wound healing.

A majority of patients had durable wound healing, and these were large wounds to start with. Most patients in the Phase I/II study received anywhere from 4 to 6 transplant. So I think 6 transplanted sheets, and each of these sheets at the time of the Stanford production were [ 35 centimeter square ].

So if you do the math, we're talking about over 200 centimeter squared off addressed wounds in the trial. So for the patients who continue to be followed, these patients have had durable wound healing, not all, but majority of the wounds were healed in the very long term.

And when we looked at patients who had a greater than 50% wound healing, so by wound size, these were clearly associated with absence of pain on those wound size. So we know that, wound healing is associated with pain.

In this case, in particular, for these large wounds, even a 50% reduction in the wound size was associated with the benefit in terms of pain..

Measurements of quality of life in this patient population is more complicated and that a lot of the things that affect their quality of life are results from years, long -- a very large wound burden and chronic wounds. So inasmuch as pain and, of course, wound care affects quality of life, which, of course, it does, significantly.

The more we can treat their wounds, the large is the area that I think the greatest, the overall impact will be for their quality of life. They have other limitations regarding ambulation. Some of these patients developed fusing of their fingers, meaning a pseudosyndactyly, that obviously limits their ability to use their hands.

They have joint contractors, they have issues with maintaining a good nutritional status, et cetera. So one could imagine that if you, over time, can treat a significant proportion of their wound burden, whereas you won't cure the disease, you could start to make a dent on some of the systemic manifestations.

Decrease their metabolic demands, improve their nutritional status that obviously reduce pain and soften the burden of a wound care, which is quite significant. .

So I'm just thinking sort of longer-term here, and that will require, I think, a pretty extensive management of their wounds. This -- if you ask the patients, and I haven't asked them personally, because I don't know them, but the Stanford team has asked these patients who participated in the Phase I/II trial.

And they uniformly said, that they would like to be retreated, if they were given the option. .

So I think, whereas this is not a scientific research. It is I think, ultimately, the patients are the ones who will tell us whether or not they think this is meaningful.

And to the best of our ability to assess their quality of life from a distance, it seems to Stanford team and to us that this is quite a strong testament that this particular product has provided relief and benefits to these patients, who would, if given the choice volunteer to getting more of these EB-101 corrected-cell therapy sheets. .

[Operator Instructions] We will go next to Kennen MacKay at RBC Capital Markets. .

A couple here as well. First on RDEB.

I was wondering if there were additional centers you were planning to eventually open beyond Stanford and screen from?.

And if there were any estimates you could help us with around enrollment time lines, either prior to the COVID-19 impact, just to help us understand this? Or again, adjusted or what we're hearing or what we know so far, around COVID-19 impact. .

And then I was just hoping you could elaborate a little bit on the manufacturing challenges in MPS? Sort of where we are now? What next steps are? And again, if there's any pre or post COVID estimates on timelines to resolution there?.

And then lastly, Christine, sorry to hear about your departure. I was hoping, maybe you can elaborate a little bit more on this decision. It seems like, departure at the end of the month is about 2 weeks' notice here. .

Ken, thanks. So I'll go by step here and if I forget something, remind me. But second center, we are -- have identified a second center in the East Coast. We're in the process of going through contracts, IRB review, et cetera. Assuming there are no disruptions on their side, we should be able to announce the center in the coming months.

Obviously, a lot is up in the air now because people are worried about other things that perhaps, take precedence. But if no disruptions, that is the plan. .

We had announced that we expected enrollment to be completed by third quarter of this year. And we were geared up to do that and planning accordingly, including scheduling patients for biopsies at Stanford and getting all prepared for that. Obviously, with the COVID-19, a lot of these plans are being reassessed.

I don't have any definitive answer one way or the other, and I think a lot of it will depend on what happens in the coming weeks. We don't believe this to be a permanent setback, but it could be disruptive, temporarily. I just heard this morning the Bay Area is blocking all movement around and stuff.

So I'm not sure even how that -- how far it goes, whether it includes Palo Alto or not so -- even as early as this morning, there've been news on this. .

Switching gears to the MPS manufacturing. We -- as we announced before, we are now looking ahead for manufacturing MPS IIIA product in -- so that is ABO-102 at our Cleveland facility.

And we have begun already the efforts and activities to develop the process, which has been so far successful and also scaling up with additional equipment to be able to produce this in a larger scale. So this is underway.

It's -- obviously, these processes take time, until we can have a final release of the clinical material, but we are well underway on that.

And we're -- some of the effort deployed last year for the Batten disease product has equipped the team and the staff at Abeona to actually be more proficient with the process development, and that was an important sort of both step up for everyone in Cleveland, who successfully went through that process.

And then it actually, lock the process, in fact, for manufacturing. So that bodes well for the MPS IIIA too. So the same team is working on this now, a very qualified team. .

So I guess, that's the first questions for me. I'll let Christine speak for herself, since she's still here. .

Thank you, Kennen. As you mentioned, yes, effective at the end of the month, it's officially the resignation. I am staying on for a transitional period, until the end of June, to further oversee sort of the transitional efforts here.

And as João mentioned, I have accepted a nomination to the Board and look forward to assisting in the guidance of Abeona together. I care deeply about the team and various stakeholders, and look forward to that work ahead of us. .

We'll move next to Difei Yang at Mizuho. .

Just a couple here. The first one is EB-101.

João, if you could make comments to what is then, if the East Coast side doesn't get up and running, can you get away, just using the patients being treated from the Stanford site and for registrational purposes?.

Yes. So thanks for your question. So Stanford, it has the capabilities to enroll the full trial, for sure. In fact, that could be the -- could have been the, the overall sort of the plan.

We felt that it would be important to add another site, and eventually, we want to qualify additional sites back to a question asked earlier, because at some point, we want to have multiple sites across the United States qualified to deploy this treatment, once it's approved.

So for the Phase III, we don't want to add too much sort of distraction and too much variability because we're trying essentially, as I see, this particular trial though, is a pivotal trial, it's ultimately a bridging trial for a product that was shown to be efficacious now for 3 to 5 years post treatment.

And we are doing this trial now, because we're manufacturing this product now, in compliance with a Phase III and to be commercial quality..

And obviously, the manufacturing site has transferred to Abeona. So for these reasons, we have to replicate the original trial, but the original trial, it surely works.

So we don't want to make too many variabilities in the conduct of the trial because I think that could add noise and disruption of the system, but we felt that adding one more site to qualified site was prudent, both in terms of the ability to enroll a little faster and start the process of equipping new sites, new centers, EB centers for treating EB-101 patients -- sorry, to treating our RDEB patients with EB-101.

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Okay. And then looking at MPS IIIA program, it seems like Abeona has also started ABT-003 trial for older patients, but also more cognitively challenge the patients.

Would you share your thoughts behind this trial, and whether this trial will be required for as part of the registration?.

Yes. So the second part of it, the answer is, no. Although, all safety data will be provided to the agencies. And of course, more safety data is relevant.

There is a -- Sanfilippo, obviously, is a disease that causes a fair amount of disruption in their development to manifest both by cognitive delay first and then cognitive decline, usually starting in the first few years of life, in fact. .

In addition to that, patients with Sanfilippo have a significant amount of behavioral problems, which often can be the presenting symptoms. Also sleep disturbances, some will develop seizures.

So there's a whole constellation of other consequences of the disease process that also affect the patient's quality of life and overall sort of life in general.

So we -- this has been a long-standing commitment to the community that -- whereas for a cognitive assessment, the earlier you treat, the better the chances of impacting this disease before it causes a more neurological damage.

It is unclear, and therefore, that this particular trial you mentioned, it's unclear whether or not treatments like ABO-102 in gene therapy, can help other aspects of the disease that has some of the behavioral manifestations, some of the sleep disturbances, some -- there are often patients who describe or the families who described their children is having sort of episodes -- it sounds like they are acutely in discomfort, as if he was in pain.

Although, it's hard to ascertain the nature of those, but these can be quite disruptive. So we've worked closely with the patient community and the clinicians to device also assessments that are more tailored to that population, who wouldn't qualify for the ABT-001 study, but who still have significant morbidity from the disease.

So it's conceivable, although it's too early to tell that some of this intervention could potentially mitigate some of these other symptoms, whereas it's less likely that we would reverse the cognitive decline, if there are too advanced. Again, there's not necessarily looking at the very advanced patients.

There are any -- it's essentially eligible patients are those who are not eligible for the ABT-001 study. And just to recap, the eligibility cutoff for patients coming into the ABT-001 studies, that they have a developmental quotient of at least 60% abnormal.

So if somebody has a developmental quotient of 50% abnormal, which it's not uncommon in the child, let's say, 4 years old or 5 years old, usually 4, around 4, they already have this much developmental delay. These would then be eligible for the ABT-003. .

With no other questions holding, that will conclude the portion for Q&A for today's call. I'll turn the conference back to management for any additional or closing comments. .

Anything? No. So I want to thank you, everyone, we see here. I'm sure I have some comments here, closing comments. But no real -- just other than, thank everyone for participating. And again, thank all of the collaborators and the patients who volunteered for these trials. And we continue to be committed, and we'll provide updates as we have those.

Thank you. Have a good day. .

Thank you. .

Thank you. Ladies and gentlemen, that will conclude today's call. We thank you for your participation. You may disconnect at this time, and have a great day..