Abeona Therapeutics Inc.

Good morning, and welcome to your Aviana Therapeutics third quarter 2020 financial results call. All lines have been placed in the listen-only mode, and the floor will be open for your questions and comments following the presentation. If you should require assistance throughout the conference, please press star zero on your telephone. I'll now introduce your host for today's conference, Greg Jin, Vice President of Investor Relations at Aviana. Please go ahead.

Thank you, Christy. Good morning, everyone. I'd like to welcome and thank you for joining us on Aviona Therapeutics' third quarter 2020 conference call. Yesterday, after the close of U.S. financial markets, we issued a press release announcing the third quarter results. The press release is posted on our website at www.avionatherapeutics.com. On the call today with prepared remarks are Michael Amoroso, Chief Operating Officer of Aviona Therapeutics, and Ed Carr, Chief Accounting Officer. After the prepared remarks, we will host the Q&A session. We are also joined by Jay Bircher, Chief Technical Officer. Before we start, I'll 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 Law. 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, followed by the company with the SDC. 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, and good morning to our investment community. The Abiona team is excited to be with you this morning. We at Abiona remain committed to pursuing the development of our portfolio of advanced and early-stage programs toward providing our novel gene and cell therapies to patients who currently have no approved treatment options. In the near term, we are focused on continuing our clinical programs in recessive dystrophic epidermolysis bullosa, or R-DEB, and Sanfilippo Syndrome Type A, also known as MPS3A, as well as Sanfilippo Syndrome Type B, also known as MPS3B, to drive ebiona's near and long-term growth and unlock shareholder value. First, starting with EB101 in our vital program. Enrollment is ongoing for the EB101 Pivotal Phase III Vital Study for RDEP. Three patients have been treated to date. As a reminder, target enrollment for the vital study is 10 to 15 R-dead patients with approximately 30 large chronic wound sites treated in total. We currently anticipate completing enrollment in the vital study in the first half of 2021, depending upon the impact of the COVID-19 pandemic, including travel restrictions and concerns about patient and or staff safety. Based on the current expectation for completing enrollment, we would anticipate top-line data for the vital study in late 2021. As a reminder, the pivotal endpoint will be six months post-last-treated patient. Let's move on to our Phase I-II clinical trials for our adeno-associated virus AAV-based investigational gene therapies, specifically AB0102 and AB0101 for MPS3A and 3B, respectively. We refer to these respective trials as Transfer A and Transfer B. I'm proud to report that target enrollment in the Transfer A study for MPA3A, which was 15 to 22 patients, has been achieved. Total enrollment to date in Transfer A is 18 patients, including 12 patients who were dosed in cohort three, the higher dose limit of three times 10 to the 13th vector genomes per kilogram. We at Abiona have made the decision to continue enrolling patients into the Transfer A study through the first quarter of 2021, given the lack of treatment options for patients afflicted with MPS3A, as well as on the back of our encouraging data from Cohort 3 from a safety and efficacy standpoint. Turning to the Transfer B study for MPS3B. To date, 11 patients have been dosed, including four patients dosed in Cohort 3. A reminder, the cohort 3 upper dose for transfer B is 1 times 10 to the 14th vector genomes per kilogram. We anticipate completing target enrollment in transfer B study, which is a range of 15 to 20 patients, in the first quarter of 2021, since COVID-19 has impacted some patients' ability to travel and undergo study treatment. Regarding treated patients in the transfer A study, we have previously reported data showing preservation of neurocognitive skills between 18 months and two years post-treatment among three young patients who had been treated in the dose cohort three of the study. We are gathering additional neurocognitive assessment data from the high-dose cohort three with follow-up for each patient more than two years, some up to three. These patients are between three and about five and a half years of age. and we plan to present these results at a very near-term future medical meeting. The new data could provide additional evidence of ABO102's potential to prevent further neurodegeneration and preserve a normalization in the acquisition of neurocognitive skills in young MPS3A patients versus the natural history of this disease which indicates patients with MPS3A between 30 and 36 months of age tend to plateau in terms of gaining skills, and they start to regress thereafter to minimal levels of neurocognitive and behavioral skills. This data will be a very important time point later in the year, as we will be looking at each of these patients two years plus post their dosing. In the third quarter, we presented our plan toward registration of ABL-102 for MPS III-A during a kickoff meeting under the EMA's prime program, which offers a path for accelerated review of promising therapies targeting unmet medical needs. Based on the meeting, along with our previous input from the Committee for Medicinal Products for Human Use, CHMP, and the Pediatric Committee of the EMA, PEDCO, we anticipate submitting a marketing authorization application ABO102 in MPS3A disease after the completion of two-year follow-up neurocognitive assessment of the last patient treated in the Transfer A study. With regard to U.S. filing, we have engaged the FDA to discuss the regulatory path for ABO102 in MPS3A. We're targeting a potential meeting to take place in the first quarter of 2021, of course, depending on the FDA's availability, as they've been quite bogged down with the COVID-19 pandemic. We look forward to providing an update early next year on our ongoing plan and timelines for ABO-102 in the United States. Next, moving onward to our manufacturing and technical operation activities, one of the great areas of strength for ABO and therapeutics. Process development at our GMP manufacturing facility in Cleveland, Ohio, is ongoing and is expected to enable production in-house of the retrovirus used for EB101 manufacturing. This will provide eBiona with increased control of our supply chain, product quality, while at the same time, of course, reducing costs. In addition, process development activities to enable in-house manufacturing of commercial supply for our gene therapy products, AB0102 and AB101, are ongoing. As Greg indicated, Jay Bircher, our Chief Technical Operations Officer, will join us during the question and answer portion if any questions arise around our manufacturing. Next, turning to corporate updates. We entered into two strategic partnerships with Keisha Gene Therapies for AB0202 for CLN1 disease also known as infantile Batten's disease, and for an AAV-based gene therapy for Rett syndrome. These partnerships are expected to allow us to unlock near-term value in earlier stage non-core assets, while also providing to Abiona the opportunity to share in future success of these programs through achievement-based clinical, regulatory, and sales milestones, plus royalty-based payments on net sales. We're excited to work with this partnership in order to propel these gene therapy products to patients faster. Before I turn the call over to Ed for the financial portion of today's discussion, I wanted to say a few words about what excites me about Abiona while I've assumed my new role as operating leadership. Abiona has a significant foundation in place which starts with our great people and their capability, our deep science and a robust pipeline, both near and longer term. In addition to our clinical programs, we are researching and developing next-generation AAV-based gene therapies using Abiona's novel capsids and capsids from the AIM technology platform. We intend to continue to develop chimeric AAV capsids capable of improved tissue targeting for various indications, including different monogeneic retinal disorders. Our board of directors and our senior management are fully committed to our company's future. It's an exciting time as we shape the future of Abiona, we shape the future for patients, and as we work toward addressing these unmet medical needs. With the organizational changes announced in October, I'm proud to report we have minimized distraction of the team. We have continued to stay poised to unlock the potential of our people in our pipeline, and I am privileged to lead this group going forward. I thank you for your time today. And with that, I'm going to turn this over to Ed Carr, our Chief Accounting Officer, for a financial update.