Abeona Therapeutics Inc.

Good morning, ladies and gentlemen. Thank you for standing by. Welcome to the ABONA Q3 2021 earnings call. At this time, all participants are on a listen-only mode. After management's prepared remarks, there will be a question-and-answer session. I would now like to turn the call over to Greg Chin, Head of Investor Relations. Please go ahead.

Thank you, Kelly. Good morning, everyone. I would like to welcome and thank you for joining us on our third quarter 2021 conference call. The press release announcing the third quarter results and recent operational progress is available on our website at www.abionatherapeutics.com. On the call today with prepared remarks are Vish Seshadri, CEO of Abiona, and Ed Carr, CFO. After the prepared remarks, we'll host the Q&A session. We are also joined by Dr. Brian Keveny, our Chief Technical Officer. 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 End 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, I will now turn the call over to Vish.

Thank you, Greg. Thank you. And good morning, everyone. Thank you for joining us this morning. This is my first quarterly call since transitioning to CEO about four weeks ago, and I'm happy to update you on our substantial progress in the third quarter and since quarter end. I'm thrilled to be leading Ediona during this exciting and critical time in our life cycle. We are advancing two late-stage pivotal assets with rare pediatric designations, transformational potential, and key anticipated milestones in the coming months, including finishing patient accrual for the EB101 Phase III vital study in the first quarter of 2022 and top-line data readout in the third quarter of 2022. We are continuing to work toward bringing our gene therapies as safely, effectively, and quickly as possible to patients who suffer from these diseases that have no approved treatments. We have taken a big step toward that goal by recently enhancing our leadership and bench strength with gene therapy and biopharma industry veterans to prepare for two biologics license application submissions for our LEADS clinical programs in RDEB and MPS3A. These hires include John Voss as head of quality, Carl Denny as head of regulatory, and Kate Imhoff as senior director of regulatory. All three bring considerable gene therapy experience with late-stage clinical and commercialized products in companies like Avexis, Sarepta Therapeutics, and Selectus. Now let's take a closer look at EB101, our investigational autologous gene-corrected cell therapy that has demonstrated a high rate of instantaneous wound healing and pain reduction for six years, the latest follow-up time point reported, after treatment of large chronic wounds in R-DEP patients in the Phase I-II study. We are anticipating similar outstanding results from our ongoing Phase III vital study. As a reminder, the target for the vital study is the treatment of approximately 35 large chronic wounds, each treated wound being paired with an untreated control wound within the same patient. Large chronic wounds are the most severe and problematic wounds in our dead. Unlike small recurring wounds that can close spontaneously, these large chronic wounds have greater than 20 centimeters square of surface area and remain open for more than six months, very often for years, and cannot close themselves and are associated with severe pain that is often managed with opioids. I am very pleased to report that since the last earnings call, we have continued to treat patients and we are close to accruing the vital study. We have identified and are scheduling treatment for the final patients and anticipate treating them by first quarter of 2022. We therefore expect top line study results in the third quarter of 2022 upon completing the 24 week follow up for the last patient treated. The anticipated timing for BLA filing is year end 2022 to early 2023. As previously mentioned, UMass Memorial Medical Center, the second clinical site in the VITAL study, is now active and prescreening the patients. Turning to EB101 manufacturing, as a reminder, we are a non-CDMO-dependent company. We manufacture the drug products for the Phase III VITAL study at our Cleveland facility. We have made significant progress in updating the Module 3 of the IND with information pertaining to manufacture of EB101 in-house. We have also been advancing process characterization work and other CMC activities to support a BLA filing for EB101. We believe that bringing manufacturing and critical analytical assays for our assets in-house at Abiona is a key strategic advantage in the cell and gene therapy space. Let's turn to our adeno-associated virus platform and our second clinical program, AB0102, Investigational Therapy for San Salipo Syndrome Type A, or MPS3A. In the previous earnings call, we reported that we aligned with the FDA on the definition of the primary endpoint for the pivotal transfer age study to enable registration. The primary endpoint is a neurocognitive assessment using raw score of Bailey Scales for Infant and Toddler Development, VSITD, and the Kauffman Assessment Battery for Children, KABC2, for children who attain a developmental age of 42 months. We have since submitted an amended trial protocol reflecting the agreed-upon endpoints. We have collaborated with experts in the MPS3A to finalize the Statistical Analysis Plan, or SAP, which we are scheduled to discuss with the FDA in the first quarter of 2022 through the RMAT mechanism. As a reminder, we have already treated 10 patients with ABO102 in the therapeutic dose cohort. The preservation of neurocognitive development is evaluated over a period of up to five years. Therefore, depending on the primary analysis methodology and the neurocognitive performance observed in the more recently dosed patients, we anticipate top-line results from Transfer A anywhere between the fourth quarter of 2022 and second quarter of 2023. We are excited about the safety and magnitude of benefit seen with ABO102 in children treated early in age at the therapeutic dose for whom we have reported unprecedented trends in disease-specific biomarkers, preservation of neurocognitive development, as well as anatomical development using brain MRI, which was presented at the International Symposium on MPS and Related Diseases in the third quarter of 2021. The MRI data shows increased gray matter, corpus callosum, and amygdala volumes in the brain in three young patients with MPS3A treated at 24 months or before, and at two years post-treatment as compared to afflicted patients without treatment, which is consistent with the preservation of neurocognitive development that we have previously reported. While the triangulation of results from biomarker, neurocognitive development, and brain MRI builds clinical conviction about the treatment effect of ABO102, this will be important also from a regulatory approval standpoint. The FDA has indicated that they will consider all kinds of clinical data points holistically in regulatory decision-making. We have previously sourced the ABO-102 drug product from Nationwide Children's Hospital. Earlier this year we commenced activities to be able to manufacture ABO-102 at our Cleveland facility. We are on track to complete manufacturing of six GMP lots of ABO-102 this year using animal-free materials. We expect analytical comparability studies to establish equivalence of Aviona-produced drug product with that sourced from Nationwide Children's Hospital in the first half of 2022 using the battery of tests as instructed by the FDA for Avio-102. Additionally, in preparation for commercial supply of ABO-102, we have initiated the construction of a 12,000-square-foot commercial AAV manufacturing facility at our Cleveland site. This facility will also have the capacity to support other AAV programs, including our preclinical ocular programs in the future. Let's move on to a brief update on our third clinical program, the ABO101 Transfer B Study in Sanfilippo Type B, or MPS3B. As we first mentioned on the second quarter call, we have closed enrollment in the Transfer B Study and are following patients treated in the study and under the Compassionate Use Program in Germany for safety and efficacy of ABO101. As reported earlier, biomarker and safety data look encouraging. We look forward to seeing two-year neurocognitive data to assess efficacy of ABO101 in the second half of 2022. I will now briefly turn to our preclinical programs. While we are currently focused on rare diseases in our clinical programs, we intend to address larger areas of unmet medical need, and our preclinical programs are investigating novel AAV capsules in five undisclosed ophthalmic conditions with estimated U.S. prevalence ranging from 5,000 to 15,000 patients. We previously shared data from non-human primates to determine optimal routes of administration with different novel capsids. Subsequently, we have made significant progress in generating mouse models for three indications, prepared the genetic constructs, produced the vectors with appropriate capsids, and developed assays to measure efficacy in the preclinical setting. We are encouraged by gene expression levels with our constructs in in vitro studies. We're expanding the model mouse colonies and have already started dosing animals. We expect animal proof of concept data by mid-2022 and pre-IND meetings with the FDA in late 2022. Before I request Ed to review our financial results, I want to briefly comment on our formal settlement with Regenexx Bio, which resolves our previously disclosed dispute over an arbitration award. The settlement provides for payments by AB-ONA over a three-year period, and importantly, raises certain downside scenarios that could have made it challenging to make it to our next inflection points. With the arbitration uncertainty behind us, we are focused on completing the registration-enabling studies and submitting BLA filings for both EB-101 and AB-0102 to deliver these therapies to patients in need as quickly as we can. With that, I'll now turn over the call to Ed.