Abeona Therapeutics Inc.

morning ladies and gentlemen thank you for holding your conference call will begin in just a couple of minutes once again thank you for holding your conference call will begin in just a couple of minutes Thank you. Thank you. Thank you. Good day and welcome to the Abiona Therapeutics second quarter 2021 conference call. There will be a Q&A session after the presentation and instructions will follow. As a reminder, today's conference is being recorded. I'll now introduce your host for today's conference, Greg Ginn, Vice President of Investor Relations and Corporate Communications at Abiona. Please go ahead. Thank you, Paul.

Good morning, everyone. I would like to welcome and thank you for joining us on our second quarter 2021 conference call. The press release announcing the second quarter results and recent operational progress is available on our website at www.abionatherapeutics.com. On the call today with prepared remarks are Michael Amoroso, Chief Executive Officer of Abiona, and Ed Carr, Chief Accounting Officer. After the prepared remarks, we will host the Q&A session. We are also joined by Dr. Vish Seshadri, Head of Research and Clinical Development, and Dr. Brian Kavaney, a lead research scientist working on our preclinical eye 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 excuse me, 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 DSCC. These documents are available on our website at www.abionatherapeutics.com. And with that, I will now turn the call over to Michael. Michael?

Thank you, Greg. Good morning, everyone, and thanks for joining us. We are excited to be with you today, and I hope this call finds you and your loved ones safe in this still uncertain time. I'd like to begin this morning with a review of our three strategic priorities for this management team in 2021. First, bolstering our relevant operational experience, first and foremost for our management team, but also for our board members. delivering operational excellence, both timely and fiscally disciplined, to make sure we advance our clinical programs with meaningful data toward regulatory milestones. Third, prioritizing, executing, and advancing our preclinical pipeline toward the clinic, starting with a focus that we've discussed in our last call on the eye or ophthalmic gene therapy programs. Now that it's the midpoint of 2021, let's assess how we're doing. Priority one, accomplished. We have added specific operational experience to our management team and our board of directors. As noted on our Q1 call, we enhanced our board with the appointment of four new members. And most importantly, we strengthened our management team with the appointment of Dr. Vishwas Seshadri, who joined us from a senior level position at BMS to be our head of research and clinical development. In addition, we've added even more strength and experience to our clinical development program teams under Vish for ED and MPS. We've added relevant regulatory BLA experience, some of which has been paramount for our successful two Type B meetings over the last six months. Also, we continue to fortify our quality and technical operations teams in Cleveland as we move closer to the BLA readiness skill sets. All of these teams have been strengthened in 2021 thus far, and we will continue to be opportunistic about adding the best talent in the marketplace. Priority two, on target. We are delivering and have delivered operational excellence in achieving significant clinical milestones in a fiscally disciplined and timely manner. Let's start with our pivotal program in RDEM, EV101. I'm excited to tell you about the progress we made in patient enrollment, opening a second site for completing our registration trial, and ultimately all driving towards a BLA in 2022. I'm also excited to tell you about the progress made on our MPS3A program, which we had a successful and collaborative Type B meeting with the FDA last month, confirming that TRANSFER-A is the pivotal study for ABO102 in MPS3A. Our third clinical program, MPS3B, we are at the conclusion of enrollment at the high-dose cohort 3 level and will be closing the TRANSFER-B study for further accrual. We'll continue to follow these patients to assess long-term safety and efficacy, specifically neurocognitive development over time. And then priority three, also on target, we are advancing our preclinical eye programs toward the clinic with great speed and precision. During Q2, we reported the results of two non-human primate studies. We are on track to begin proof of concept studies in the second half of this year, and this will be toxicology study enabling into 2022 with some select ophthalmic indications. Now, let's take a deeper dive into the progress of our clinical programs and preclinical programs. Starting DB101, our lead pivotal phase three vital study for recessive dystrophic epidermolysis pilosa, or RDEM, we continue to build enrollment momentum in vital. There continues to be high and growing level of interest among patients and health care professionals, and we are very pleased to have recently activated a second clinical trial site at UMass Memorial Medical Center in Worcester, Mass., under principal investigator and world-renowned EB expert, Dr. Karen Wiss. Dr. Wiss is a professor of dermatology and pediatrics at UMass Medical School, and she's the director of pediatric dermatology. We are excited to have the UMass team join Aviona's mission of pursuing a standard of care for large and chronic R-dead wounds. This is a significant and major milestone, and I want to congratulate the team on this timely execution and thank the staff and team at UMass. Now, in addition to our lead trial site at Stanford Medical Center in Palo Alto, California, we can provide convenient treatment locations for study participants on both the West and East Coast, making travel logistics easier for patients and family. I'll remind our investment community, this was one of the major challenges we heard from the community in 2020 and thus far, In 21, it seems to be opening up with our pandemic. This second site is paramount to our success. At the same time, we're expanding necessary physician experience with EB101 as we plan for potential commercial launch in the U.S. We look forward to collaborating closely with the clinical team at UMass to screen and enroll subjects in vital as soon as possible. They are ready to go. Next, we continue our progress toward completing patient enrollment in the vital study. We have successfully administered EB101 to patient number five. As you may recall from our last call, this is the patient who opted to re-biopsy and make new product. We are extremely happy to report that the re-biopsy and manufacturing of EB101 product was successful for this patient and they have received their EB101 administration. The patient will be followed as per our vital protocol Additional patients continue to be identified and prescreened at both Stanford and now UMass to determine their eligibility for vital. As a reminder, the target for the pivotal trial is the treatment of approximately 35 large chronic wounds across 10 to 15 patients. Today, I'm excited to report that we have treated more than half of the target number of randomized pairs of wounds thus far. Next, let's turn our attention to our AAV platform, our second in-clinic program, ABO102 for San Felipe syndrome type A or MPS3A disease. Yesterday, we announced some very exciting news about our type B meeting with the FDA for the MPS3A program. We had a successful collaborative type B meeting and aligned with the FDA that the current single-arm transfer A study will serve as the pivotal study for ABO102. and potentially support a biologic license application, of course, depending on the data set. Since 2016, we have now treated 21 patients in the TRANSFER-A trial. We're excited about the safety and the magnitude of benefit seen with our investigational ABO102 product in the younger children from the higher-dose cohort, as we reported at the World Symposium earlier this year. We remain hopeful that if the more recently dosed children in cohort three displayed similar treatment effects as those already presented, we could have an invaluable data set in 2022. The patients we serve have tremendous unmet need. They cannot wait. And we remain fully focused on operational excellence with the intent of now delivering two pivotal data packages in 2022, one for EB101 and one now for ABO102. At the Type B meeting, we also aligned with the FDA on the definition of the primary endpoint for Transfer A, which is neurocognitive assessment using the raw score from the Bailey Scales of Infant and Toddler Development and the CalcMint Assessment Battery for Children. These scales will be used in sequence. This is data we're already collecting as secondary endpoints. They will move to primary in Transfer A and Vish will tell you a little bit more about that shortly. We are grateful to the FDA for their guidance, and we look forward to continuing to work closely with the agency toward our goal of bringing potentially life-saving therapy to patients afflicted with MPS3A. In the near future, we intend to share the Type B meeting feedback with the EMA, followed by potentially other regulatory authorities around the world, to guide our development plans for NAA in ex-US markets. Of course, the United States is our first focus. In addition to the guidance and clarity from the FDA on the regulatory front, we also shared some very encouraging new clinical data from TransferA that had not before been published this past weekend during an oral presentation at the 16th International Symposium on MPS and Related Diseases, our new MRI data. The data indicates that ABL-102 increases gray matter, corpus callosum, and amygdala volumes in the brain in three young patients with MPS3A at 24 months post-treatment compared to an afflicted patient without treatment. Now, let's talk about this for a moment. Brain volume loss is characteristic in children with MPS3A, and it's associated with long-term cognitive and physical disabilities. Specifically, gray matter is important for cognitive development. Corpus callosum for motor function, and amygdala for fear learning, as well as social and emotional development. We're excited about the MRI data, as are our clinicians, and it's consistent with previously reported results of preservation of neurocognitive development in the three younger children from cohort three that we have presented from Transfer A earlier this year with the longest follow-up. Moving onward to our last in-clinic program, ABL101 in the Transfer B study for MPS3B. As noted on the last quarterly call, we reported results from Transfer B at the World Symposium in February. The results to date, Transfer B high-dose cohort, like the high-dose cohort from Transfer A, show the drug is safe and well-tolerated, eliciting a dose-dependent sustained reduction in disease-specific biomarkers, denoting clear biologic effects. As I've reminded you in the past, we absolutely want to see biomarkers moving in the right direction, but the gold standard will be the collection of neurocognitive development data, and we need to let that mature into 2022. Previously, we shared the accrual in Transfer B was paused after dosing four patients in the higher cohort because the drug product, again supplied by Nationwide Children's Hospital in Ohio, had reached its two-year shelf life expiration. Some of our additional stability testing results showed specifications were not met for all parameters of transfer B protocol. However, specifically physical titer. However, after reviewing the test results based on equivalent potency, infectious titer, as well as the purity profile of the drug, the German Health Regulatory Authority, along with our DSMB, deemed that the benefit of the drug outweighed the risk and endorsed the use of the product for those remaining patients with highest unmet need through the German Named Patient Program. The NPP is a compassionate use program in Germany that allows individuals to be treated at the request of the retrieving physician and families. We're thankful for the authority and the DSMB actions acting quickly to get drug to these patients. To date, three patients have been dosed in the German NPP and a fourth is pre-screened and getting ready for treatment. The total number of patients treated now at the higher dose, one times 10 to the 14th vector genome per kilogram, between transfer B cohort three and now the MPP program will soon be eight children collectively. In parallel, we're in the process of closing enrollment in the transfer B study, and patients from both the transfer B and the main patient use program will be monitored with the same rigor for ongoing safety and efficacy of the high-dose AB101 product as they would have been within the Transfer B study. While early biomarker data are promising, neurocognitive preservation and development is the gold standard as we've discussed. We look forward to seeing two-year neurocognitive data beginning in the first half of 22 for some of the first patients dosed in the higher-dose cohort from Transfer B. And subsequently, we'll determine next steps for the program once we have some of these important and essential data points. As we await these data, I'll remind you that out of our wholly owned Cleveland non-CDMO dependent manufacturing facility, AB0102 for NPS3A commercial grade product is being made. Our experience in AAV production from our fully owned Eliza Litton Center will be highly transferable if and when we decide to manufacture AB101 for MPS3B program, of course, data-dependent in 22. Finally, I'll give a brief update preclinical, and I'll open it up to the group of questions. As previously noted, we conducted preclinical research with novel AAV capsules, including our Abiona invented and partnered capsules in six undisclosed eye indications. These eye indications represent opportunities in the U.S. alone of about 5,000 to 15,000 patients with the highest unmet need. This continues our ebiona identity as a fully integrated end-to-end gene therapy company focused on high unmet needs.