Abeona Therapeutics Inc.

Good morning, everyone, and welcome to AB Owner's fourth quarter and full year 2023 earnings conference call. At this time, all participants have been placed in a listen-only mode, and the floor will be open for questions after the presentation. If anyone should require operator assistance during the conference, please press star zero on your phone keypad. Please note this conference is being recorded. I will now turn the conference over to your host, Greg Gin, VP of Investor Relations. Greg, over to you.

Thank you, Jenny. And good morning, everyone. I would like to welcome and thank everyone for joining us on our fourth quarter and full year 2023 conference call. The press release announcing the full year 2023 results is available on our website at www.abostherapeutics.com. I would like to note that remarks made during today's call may contain projections and court-looking statements. 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 risk factor section in our Form 10-K and periodic reports filed with the SEC. These documents are available on our website at www.ablenttherapists.com. On the call today with prepared remarks are Dr. Vish Sasadri, Chief Executive Officer, Dr. Mata Vizantavada, Chief Commercial Officer and Head of Business Development, and Joe Giordano, Chief Financial Officer. Also joining us for the Q&A session will be Dr. Brian Keveny, Chief Technical Officer. And with that, I will now turn the call over to Vish Sasadri to lead us off. Vish? Thank you, Greg.

Hello, everybody, and thank you for joining us this morning. I'm pleased to highlight several important milestone achievements for Aviona since our last quarterly call. We continue to make significant progress towards U.S. commercialization. Our top priority is to secure FDA approval and launch Pradimogene Xamycarasso, or PZ cell, our investigational autologous Col7a1 gene-corrected epidermal sheet for recessive dystrophic epidermolysis bullosa, or RDEB. As a reminder, The FDA accepted and granted priority review in November for our PZ cell BLA and assigned a PDUFA date of May 25th, 2024. The BLA process is ongoing and we have several positive status updates to share with you today. The FDA has now completed a bioresearch monitoring BIMO inspection of Abiona, the BLA mid-cycle meeting, and a two-week pre-license inspection, or PLI, of our manufacturing sites. The BIMO inspection was conducted on-site at our headquarters in Cleveland, Ohio, from Jan 22nd through Jan 24th, 2024, and reviewed the conduct and practices that pertain to the clinical studies of PT cells and its data integrity. The FDA inspector did not issue any observations or form 483s during that inspection. The BLA mid-cycle review meeting took place on Jan 25th, 2024, following the BIMO inspection. At the meeting, the FDA indicated that there were no major safety issues and reaffirmed that they are not planning to hold an advisory committee meeting to discuss the BLA. In addition, the FDA advised that risk evaluation and mitigation strategies, or REMS, are not anticipated for PZ-Cell, although BLA review is still ongoing with a proof of target action date of May 25, 2024. In February, the FDA conducted a two-week PLI, or pre-license inspection, of Fabiona's manufacturing facility, which concluded on March 1st. During the inspection, which was conducted by five FDA inspectors, the FDA reviewed the facilities, systems, and processes at the Cleveland site. This comprehensive evaluation included observing the manufacturing process for PV cell, as well as the performance of various in-process and loss-relief assays. Upon completion of the inspection, the FDA inspector issued a Form 483 with observations related to process controls and recommended steps for resolution in a collaborative manner. On March 15, 2024, we submitted a response to the FDA outlining steps either already implemented or ongoing toward resolution based on the FDA's guidance. In addition, the agency has completed its site inspection of the two clinical study sites, Stanford University School of Medicine and University of Massachusetts Medical School, which treated subjects in the pivotal Phase III vital study supporting the PV cell BLA with no form 483 observations noted. We're looking forward to continuing to work with the FDA through the remainder of the BLA review process. Having reached these significant regulatory milestones, we continue to advance our preparations for the planned commercial launch of PV cell in the U.S. following FDA approval. and now turn the call over to our Chief Commercial Officer, Dr. Madhav Vasanthavada, to provide an update on our commercialization readiness activities. Madhav. Thanks, Vaish. If approved, PZ-Cell is expected to deliver unique value for patients with R-DEP. It would be the only therapy which, in clinical trials, address large body surface areas, including toughest-to-treat wounds, and demonstrated wound healing and pain reduction with years of durability after a single treatment. In terms of commercial opportunity and readiness, I want to highlight three important takeaways, all of which are very encouraging. First, dermatologists have indicated a high willingness to refer patients for PZ cell treatment. Following recent market research with 64 dermatologists across both the Centers of Excellence, or COEs, and community setting, we have gained compelling quantitative and qualitative insights. These insights have validated a key assumption of our launch strategy, namely that death patients, in particular severe death patients, are co-managed between COEs and community derms. There's a high concentration of severe death patients in EB centers, and a high level of interest from community dermatologists to refer their severe patients to these EB centers of excellence for a treatment like PZ-Cell. The physicians we surveyed each treat, on average, seven to 10 RDEP patients on an ongoing basis. To hear about the strong enthusiasm from community DERMs and their willingness to refer patients for PZ-Cell, despite the logistical coordination required for surgical application, is very encouraging. The reason for such enthusiasm from both COEs and community practitioners is the clinical study data demonstrating PZ cells' ability to heal large wound areas and offer potential durability and significant pain reduction following a single application. They also noted that some patients currently travel three to four hours to a center of excellence for special procedures and would be willing to do so for a therapy like PZ Cell because their wounds could turn chronic over time and are at high risk of secondary infections and squamous cell carcinomas. We also hear from surveyed physicians as well as prominent physicians in the EB space about the need for using complementary treatment modalities for deaf patients. In addition to physicians, we surveyed patients and caregivers who stated their interest in PZ-Cell because of its ability to cover multiple wounds at once and they wanted to know specifics of the logistical aspects of getting PZ cell therapy. In phase three clinical vital trials, we had the ability to treat with six credit card-sized sheets that were applied either in a quilt-like manner for one large wound area or were discreetly applied to individual wounds as needed. In the commercial setting, we'll be able to supply up to 12 credit card-sized skin sheets, and this is viewed as a big deal by physicians as well as by patients whom we surveyed. The second takeaway is the continued engagement at the Centers of Excellence where we plan to make PZ Cell available. As stated in prior calls, our focus strategy is to partner with five to seven high-volume EV centers in the launch year and onboard them so that patients and caregivers get the best possible experience. We have a clear action plan and continue to gain buy-in from our target centers. Many of the identified centers treat both pediatric and adult EB patients and are geographically dispersed across the U.S., which would make help easy cell treatment accessible for patients. EB physicians at the target centers are in fact championing the onboarding process, including identifying a multidisciplinary team of surgeons, anesthesiologists, and coordinators. They're also helping us to navigate their institutional legal processes and working with their cell and gene therapy departments, which all helps expedite the process. We have signed CDAs with target centers and have initiated discussions on a master service agreement, which is often the most time-consuming process. Overall, we expect the site onboarding process to be like that of other autologous cell therapies, And speaking from personal experience, this typically takes around six to nine months of process. The initial target sites are at varying stages of onboarding, and we believe that they will be fully trained, buttoned up on the logistics, and ready to treat with PZ cells around three months after approval. In addition to the currently targeted sites, we are seeing interest from additional COEs to bring on PZ cells. We just wrapped up some productive meetings with physicians at the American Academy of Dermatology annual meeting in San Diego, and this direct physician feedback gives us reason to believe that the underlying medical need and the opportunity with PZ-Cell is strong. The third and final takeaway is the early encouraging dialogue we've had with payers to enable access due to the high unmet need and the transformational value proposition of PZ-Cell. We have initiated pre-approval information exchange with various commercial and managed Medicaid stakeholders to educate them on Rdap disease and PZ cells differentiated and meaningful clinical profile. We'll be speaking with many of the top payers in the coming weeks and months. From a pricing perspective, we believe PZ cells value is different from that of currently approved products in the EP space because PZ cells can cover much larger areas of a patient's body, multiple wounds at once, and does not rely on the need for a chronic application. We are currently working through our pricing models and expect pricing to be commensurate with the value of approved gene therapies that offer years of durable benefit following the single application. Lastly, we have engaged with the Centers of Medicare and Medicaid Services, CMS, to request a PZ Cell-specific procedure code that, if granted, would support efficient billing, access, and reimbursement of PZ Cell. In addition to the stated takeaways I would just like to note also that we are making rapid progress in building a highly experienced and nimble commercial team, and we are working with a strong network of collaborators to build capabilities for a successful launch and beyond. With that, I'll now hand the call over to our Chief Financial Officer, Joe Vizzano, to discuss our financial results.