Abeona Therapeutics Inc.

Ladies and gentlemen, and welcome to the ABIONA first quarter earnings call. At this time, all participants are on a listen-only mode. We will open the floor for questions and comments after the presentation. It is now my pleasure to turn the floor over to your host, Gregory Jinn, Vice President of Investor Relations and Corporate Communications at ABIONA. Please go ahead.

Thank you, Holly. Good morning, everyone. And welcome and thank you for joining us on our first quarter 2021 conference call. Press release announcing the first 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. where we will be joined by Dr. Juan Ruiz, Vice President at Abiona, who heads up clinical development for our MPS programs, and Dr. Brian Cavani, a lead research scientist working on our preclinical I 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.abionatherapeutics.com. And with that said, I will now turn the call over to Michael. Michael?

Thank you, Greg. Good morning to our investor community. We're excited to spend some time with you this morning. Thank you for joining us. As I approach the midpoint of my first 100 days in the job as CEO, I wanted to start by reviewing three key strategic priorities for Abiona and our team. First priority being to bolster the relevant operational experience on our management team and board. making sure we have the right coaches in place, the right experience to meet our goals going forward. Second, delivering operational excellence, both timely and fiscally disciplined, to further advance our in-clinic programs toward meaningful milestones for patients in need. And third, to further prioritize, execute, and advance our preclinical I programs toward the clinic. We've made substantial progress on these priorities and are off to a fast start in 21. I want to thank the team for their efforts. First priority one, continuing forward under the right leadership. We continue to focus on building the right talent and experience on our team, which positions us well to execute against operational goals. I'm very excited just this morning to have announced the appointment of Dr. Vishwas Seshadri, a Senior Vice President and our new head of research and clinical development. Vish, as he goes by, brings more than 20 years of experience across academia, followed by various senior and executive-level leadership roles within the life sciences industry, overseeing product development, regulatory strategy and teams for submissions, and commercialization for novel therapies, including, most recently, personalized autologous cell therapies in the CAR-T space for Celgene and BMS. Vish has significant experience across early and late stage development, from first patient in all the way through successful commercial launches. He understands the trade-off decisions and necessary discipline for evidence-based drug development. Vish is also a proven coach in developing people across functions. Through his project leadership in working with clinical, regulatory, medical, and commercial teams at Celgene, We're thrilled to have him join our team in early June and we look forward to introducing Vish to you on future quarterly calls. Welcome Vish to the ABO and the team. Also in the first quarter, we have strengthened our board of directors with the appointment of four new independent members as well as myself. The new members have experience sitting on boards of public and private companies and bring relevant operational leadership experience within the life science industry. including areas such as clinical development, manufacturing of cell and gene therapy products, and corporate and financial compliance. The new members were selected based on their relevant and diverse experience, making them the right partners to the current management team in our pursuit of both bold, near, and long-term objectives. I'd like to take a minute to review the new members and their critical experiences for Abiona. First, Dr. Leila Aland, a pediatric hematologist oncologist and physician scientist. Leila has spent over 20 years in the biopharmaceutical industry, focusing on bringing novel therapies to patients with rare diseases, and is currently the chief medical officer at PMV Pharmaceuticals. We're delighted to have Leila, and she'll be partnering closely with Vish and team. Next, Don Werchtel, having over 29 years of experience in the life science industry, with senior roles in operations and CMC. Don is currently the Chief Manufacturing Officer at T-Knife Therapeutics. He has significant experience building and leading CGMP manufacturing organizations and facilities, so Don will provide essential feedback and guidance to our teams in Cleveland. Welcome, Don. We're really excited to have you. Next is Faith Charles, a Corporate Transaction and Security Partner at Thomson Hine with over 30 years of legal experience. and lead Thompson Heinz life science practice, providing valuable insights to companies on capital markets, corporate governance, and strategic development such as M&A, licensing transactions, and strategic collaborations. Welcome to Faith. And last but never least, Mark Alvino. Mark has provided leadership and experience in the areas of financial management and business strategy as a member of the board of directors of multiple life science companies, including previously, Abiona. Mark knows Aviona, our people, and our products well, and his capital market experience will be invaluable, as well as his longitudinal perspective on our organization. We're excited to have Mark back on board. Overall, I wanted to express my excitement to announce the addition of these respected leaders, experienced both to the management team and our board. I am confident that we have the right collective leadership in place that will help us to accomplish objectives at this critical point in AB owners lifecycle excuse me Mona let me take an additional minute to comment on our boards aim to enhance and evolve our governance at our annual meeting of stockholders to be held tomorrow our board recommends that stockholders vote for proposal to to approve the amendment of our restated certificate of incorporation and to declassify the board and eliminate three-year terms for directors in favor of one-year terms, thus further aligning with investor interest and increasing accountability of our directors to shareholders. Our board believes that annual director elections are in keeping with sound corporate governance practices and promotes additional accountability to shareholders. If you have questions or need help voting for this very important proposal, please contact Greg Ginn, our head of investor relations. Next, moving on to priority two, advancing our clinical programs toward key milestones for patients. I'll start with an update on EB101, our lead pivotal program for recessive dystrophic epidermolysis bullosa, or as we refer to as RDEB. Regarding enrollment in the EB101 vital trial, we remain on track for activating a second study site in the northeast to complement Stanford on the west coast in the second half of 21 Patient and physician interest in the study continues to be high, and we're observing a greater willingness to travel on what we believe is the growing percentage of the population starting to receive vaccines for COVID-19. Next, as discussed on our last call, a fifth patient had been biopsied for the start of treatment. This patient's cells yielded product that didn't meet required release criteria as per the pivotal trial protocol. As a result, the clinical team and the patient have opted to re-biopsy, and begin a new cell therapy product generation ongoing. We are hopeful to treat this patient in the coming weeks. Also in parallel, additional patients have been identified and are being prescreened to determine their eligibility for the vital trial entry criteria. As a reminder, the target for this pivotal trial is the treatment of approximately 35 large chronic wounds across 10 to 15 patients. Next, the execution of our comparability plan is ongoing through our tech ops teams in Cleveland, as well as ongoing communication with the FDA. Thus far in 2021, overall progress for the EB101 program has been significant, and we continue toward our goal of completing enrollment in the vital study by year end. Thank you to the teams and our patients. Let us next turn our attention to our adeno-associated virus AAV-based gene therapy programs. starting with our ABO102 program in San Filippo Syndrome Type A or MPS3A. We are preparing for the FDA Type B meeting scheduled for next month in June. As a reminder, we plan to discuss the data to date from the ABO102 Transfer A study and next steps for a potential BLA path with the FDA. We intend to discuss with the FDA whether the Transfer A data set could serve as the basis for a BLA submission with natural history data as a viable control arm. We believe historical controls are critically important for MPS treatment and drug development globally in this disease, as these children are being irreversibly impaired and it's not feasible to give them a placebo. We are excited and hopeful to review the data with the FDA at our upcoming meeting and partner on a viable plan forward for patients. Also, the FDA feedback from our upcoming meeting will continue to guide and enhance our development plans and pathway to marketing authorization in Europe as well. Moving to our third in-clinic program, AB101, the Transfer B Study in San Felipe Syndrome Type B or MPS3B. Product stability testing as discussed on our last call for the clinical product from Nationwide Children Hospital is now ongoing. To expedite timing, we brought some assay testing in-house. I want to thank our teams in Cleveland for their adaptability there. After completion of stability testing, we'll assess our options forward for treating the additional patients remaining for Transfer B. During the first quarter and recent months, we also shared important clinical updates from both our Transfer A and B studies at multiple medical meetings and congresses. This is highlighted by positive data at the 17th Annual World Symposium in February 21. showing neurocognitive development of young MPS3A patients was preserved up to three years following treatment with ABL102. In addition, presented results from the transfer B study continued to show signals of biologic effect with reduction in disease-specific biomarkers in cerebrospinal fluid, plasma and urine, and reduction in liver volumes. We look forward to ongoing and additional updates of neurocognitive milestones. Moving on to our third priority, prioritizing and advancing our preclinical programs. We're focusing resources on ophthalmic indications within our AAV platform, following a strategic prioritization of our preclinical programs. On the back of our first AAV program success thus far in MPS, we're making significant progress toward adding new clinical programs to our pipe. We are conducting preclinical research with novel AAV capsids, including are wholly owned and partner capsids in six undisclosed eye indications. To give an idea to our investor team, the estimated prevalence for each of the eye indications ranges from five to 15,000 patients in the U.S. alone. This represents a significant market opportunity and more importantly, more patient lives we can help. And this stays very true to leverage our identity as a fully integrated end-to-end gene therapy company for the highest unmet needs in patients. During the first quarter, We presented the new data supporting the potential of pre-mediated dual AAV vector technology to enable delivery of large genes targeted for treatment of Stargardt's disease during an oral presentation at the Association for Research in Vision and Ophthalmology, otherwise known as ARBO, 21's annual meeting. In addition, we also recently completed non-human primate studies, comparing several capsids with AAV8, the current industry standard for intraocular administrations. I'm excited to say that the preclinical team delivered study results about four weeks early, showing that AAV204, our partnered capsid, in license from our AIM library, was superior to AAV8 using a recently developed novel route to ocular administration. In a separate non-human primate experiment, we tested our AAV214 and our AAV214D5, two wholly owned Aviona capsids. versus AAV8 by subretinal dosing administration. Both capsids demonstrated nearly identical levels of transduction compared with AAV8 of photoreceptor and retinal pigmented epithelium cells, which are the cell types most frequently affected in inherited retinal diseases. We are very excited about the findings of these experiments, which provide critical insight into the ability of our novel capsids to penetrate key cells and the optimal route of administration to accomplish this penetration. These results position us well to move rapidly into the proof of concept studies in the second half of this year, followed by toxicology studies in the first half of 22. One of our lead scientists, Brian Cavaney, has joined us this morning, and he can help us answer any questions we might have about some of this very significant preclinical progress. With that, I'll now turn the call over to Ed, our Chief Accounting Officer, to review our financial progress thus far in 21. Please, Ed.