Abeona Therapeutics Inc.

Good morning, everybody, and welcome to the Abiona fourth quarter and fiscal year 2022 earnings conference call. At this time, all participants are in a listen-only mode. A question and answer session will follow the formal 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, Mr. Greg Ginn, Vice President of Investor Relations, and Corporate Communications at Abiona. Greg, over to you.

Thank you, Jenny. Good morning, everyone. I would like to welcome and thank everyone for joining us on our fourth quarter and full year 2022 conference call. The press release announcing the results is available on our website at www.abionatherapeutics.com. On the call today with prepared remarks are Vish Sasadri, Chief Executive Officer of Abiona, and Joe Vizzano, Chief Financial Officer. After the prepared remarks, we will host the Q&A session. For that session, we're also joined by Dr. Brian Keveny, Chief Technical Officer and Head of Research, and Dr. Madhav Vasanthabada, Vice President, Business Development. 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 other periodic reports 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 our CEO, Vish Seshadri. Vish?

Thank you, Greg. Good morning, everybody, and thank you all for joining us this morning. We finished 2022 with the biggest clinical milestone achievement in Abiona's history. In November, we successfully completed the pivotal phase three vital study for EB101, our autologous engineered cell therapy, for the treatment of patients with recessive dystrophic epidermolysis bullosa, or RDEP. The trial set a high bar for success, with one of the co-primary endpoints measuring patient-reported pain, which differentiates vital from any other pivotal study in RDEP. A patient's pain perception and scoring for a particular wound can be often influenced by changes in adjacent regions within the same anatomical area and create a halo effect, therefore a signal for pain improvement has to be pronounced enough to cut through the noise and yield a statistically significant result. The VITAL trial met its co-primary endpoints and all other endpoints, demonstrating statistically significant clinically meaningful improvements in wound healing and pain reduction of large chronic RdEV wounds in the EB101 group that exceeded those observed in the control group. In addition, the study also generated positive results and a post-hoc analysis of EB101-treated wounds with severe baseline pain, where we observed even more pronounced pain reduction. No serious treatment-related adverse events were observed, which is consistent with past clinical experience with EB101. The positive vital top-line results, together with compelling long-term Phase I-IIa results demonstrating multiple years of wound healing and pain reduction after an EB101 treatment, show the promise of EB101 as a one-time therapy for a given large chronic R-dev wound. We anticipate that an average of two cycles of treatment per patient will cover their large chronic wound surface area. We're looking forward to the presentation of additional data from VITAL by Dr. Jean Tang, professor of dermatology at Stanford University School of Medicine and principal investigator of the VITAL study during an oral session at the International Societies for Investigative Dermatology, ISID, meeting in May 2023. Since announcing the vital top-line results, we have been focused on preparations for submission of a BLA for EB101 to the FDA. We have had multiple recent collaborative interactions with the FDA through both Type B and informal meetings and communications. We continue to gain alignment with the FDA on various topics, especially those related to CMC, and continue to make progress towards a BLA submission. Toward that goal and as planned, we have started activities for the Process Performance Qualification or PPQ validation campaign for EB101 that is essential for the BLA. The PPQ campaign consists of three consecutive manufacturing runs that need to pass specific tests that attest to critical quality attributes. So that is on track, and we plan to submit the BLA for EB101 in late second quarter or early third quarter of 2023, depending on the scheduling of patient biopsies, which are critical for the PPQ runs. Based on the anticipated timing of BLA submission, we expect potential BLA approval in the late first quarter to early second quarter of 2024. Recall that EB101 has been granted a rare pediatric designation by the FDA, so concurrent with the PLA submission, we plan to submit the application for a Priority Review Voucher, or PRV, which can be used to receive an expedited review process for a subsequent marketing application for a different product and even by a different company. Prior PRVs have been sold to other biopharma companies for approximately $100 million. We have also commenced early commercial planning for EB101. working with Trinity Life Sciences, we initiated discussions in the first quarter of 2023 with stakeholders across the healthcare system, including public and private payers and healthcare providers. Through these engagements, we have shared our clinical data and begun evaluating the market access opportunity and potential pricing for EB-101. We are encouraged by the initial feedback from these stakeholders and feedback that we have received from patient advocates and organizations which collectively support positive coverage decisions and pricing in line with recently approved gene therapies. Based on our initial discussions, payers view R-DEP as a disease with a very high unmet need and believe EB101 has a well-differentiated profile with durable clinical benefits for the treated wounds. Also, given the ultra-rare prevalence of R-DEP, payers view EB101 will have a limited overall budget impact and have indicated a high willingness to cover EB101 with a favorable access policy, giving us confidence in its price potential. Let's turn to our preclinical ophthalmology programs. We are excited by the broad potential for treating serious eye diseases with both our novel AAV capsids and novel gene constructs. We have previously shared that AAV204, a novel AAV capsid, from our in-licensed AIM capsid library showed encouraging tropism and transduction efficiency in the macula and optic nerve in non-human primates with an administration route that is less invasive and safer than subretinal injection. Since then, we evaluated the ability of our gene constructs and capsids to deliver and express the recombinant protein of interest in target eye tissue and rescue mutant phenotypes in mouse disease models. Based on encouraging findings from the animal proof of concept experiments, we are looking forward to reporting new data from these programs at a scientific congress in the second quarter of 2023. Earlier this month, we announced three internally developed investigational preclinical gene therapy product candidates including ABO504 for Stargardt disease, ABO503 for X-linked retinoschisis, and ABO505 for autosomal dominant optic atrophy. We have started to submit pre-IND meeting requests to the FDA for these candidates and anticipate meetings in the second quarter of 2023. Turning to our people, we further strengthened our executive bench over the last year with the addition of three experienced biotechnology industry leaders to our senior management team. Dr. Dmitri Grashev joined us as chief medical officer and Dr. Madhav Vasanthavada as vice president business development. We also promoted Amanda Moore to the role of vice president program leadership and clinical operations. Dr. Grashev has over 20 years of industry experience across multiple therapeutic areas including dermatology, ophthalmology, and oncology, and has multiple global drug approvals while leading clinical development programs at pharmaceutical and biotech organizations. Dr. Vasanth Tavara, who has a Harvard MBA with patents to his name, has over 20 years of experience in the life sciences industry in a variety of R&D and commercial leadership roles at Novartis, Bayer, and Celgene in Bristol-Myers Squibb. He was most recently at Bristol-Myers Squibb, where he led global marketing for the global CAR-T cell therapy franchise. Prior to BMS, he spent nine years at Bayer in a variety of cross-functional leadership roles, including marketing, market access, and sales. Amanda has nearly 20 years of clinical operations and development experience in the biotech and pharmaceutical industry with Biogen, Rekit Benkinsa, Sage Therapeutic, and InterCell. She has a track record of leading innovation in clinical trials pioneering operating committee relationships and oversight of global development programs. The collective experience of these executives, combined with our experienced leaders of manufacturing, quality, and regulatory, position us well as we work towards completing and submitting the BLA for EB101 and preparing for a potential commercial launch. I'll now turn over the call to Joe Vazano to review the financial results.