Abeona Therapeutics Inc.

Good day and welcome to the Aviona Therapeutics second quarter 2023 update conference call. At this time, all participants are on a listen-only mode and 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 telephone keypad. As a reminder, today's conference is being recorded. I'll now introduce your host for today's conference, Greg Ginn, Vice President of Investor Relations and Corporate Communications at Abiona. Sir, please go ahead.

Thank you, Operator, and good morning, everyone. I would like to welcome and thank everyone for joining us on our second quarter 2023 update conference call, the press release announcing the results is available on our website at www.abionatherapeutics.com. Before we start, I would like to note that remarks made during today's call may contain projections and forward-looking statements. 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 risk factors section in our Form 10-K and periodic reports filed with the SEC. These documents are available on our website at www.abionatherapeutics.com. On the call today with prepared remarks are Dr. Vish Seshadri, Chief Executive Officer, and Joe Vizzano, Chief Financial Officer. Joining us for the Q&A session will be Dr. Madhav Vizantavada, Vice President, Business Development, and Dr. Brian Keveny, Chief Technical Officer. With that, I will now turn the call over to Vish Sosadri to lead us off. Vish?

Thank you, Greg. Hello, everybody, and thank you for joining us this morning. We're happy to update you on our continued progress, in particular with our lead program, DB101, our investigational autologous engineered cell therapy that could deliver years of sustained clinical benefit in patients with recessive dystrophic epidermolysis bullosa, or RDEM in short. Remember that all our clinical data pertain to treatment of large chronic wounds, which are the hardest to treat and have remained open for years because they cannot self heal and inflict the greatest length of burden to these patients. The durability of both investigator-assessed healing and patient-reported pain following a single administration observed with the toughest RDEM wounds paints a very compelling and unique value proposition for EB101 for patients. We have made significant progress during the last quarter toward the planned EB101 BLA submission. We have completed process performance qualification, or PPQ, manufacturing runs for both retroviral vector and EB101 drug product to demonstrate our validated process and readiness for commercial production. In June, we announced that we had gained alignment with the FDA on the data required to establish retroviral vector, or RVV, comparability which is a critical CMC component for the BLA. The FDA had requested additional assay data to establish comparability between RVV source from Indiana University and RVV manufactured in-house at Aviona, both of which have been used in the EB101 clinical study. We have since generated the additional data that we believe establishes comparability between the two vector sources and have included it in the briefing package that we submitted to the FDA in July for our pre-BLA meeting later this month. In that meeting, we will align the agency on the format, content, and overall acceptability of the anticipated BLA for EB101 based on discussion of critical components of the BLA package. Following the conclusion of the meeting and subject to supportive FDA feedback, we anticipate submitting the EB101 BLA in the third quarter of 2023. As we expect a priority review based on the anticipated timing of the BLA submission, We expect potential BLA approval in the second quarter of 2024. If the BLA is approved, we anticipate being granted a priority re-voucher, which is worth approximately $100 million based on recent PRV transactions. In parallel, with our BLA-related activities, we're looking ahead to EB101's potential FDA approval and launch next year. In anticipation of approval, we raised $25 million in the registered direct offering price at the market with select existing investors in July. With the proceeds from the offering, we're ready to invest in pre-commercialization activities and the planned launch of EV101 in the U.S. by being thoughtful about what we need to spend now versus later after a day approved. We have already initiated dialogue with top EB treatment centers in the U.S. about onboarding EB101 as a treatment option, and we're encouraged by the level of interest. Physicians indicate there is a high unmet need in Deb, and particularly for treatment like EB101 that has a compelling value proposition. The positive feedback we hear from KOLs is consistent with what we've heard from payers, hospital administrators, and patient advocacy groups. We plan to continue to engage with payers, both commercial and Medicaid, to ensure broad market access after launch. In addition to preparing the market for EB101, we're also preparing our organization for commercialization. We have mapped out key commercial and medical roles that we plan to fill in the second half of this year and the capabilities we will build to support a successful launch. During the second quarter, Additional efficacy and safety data from the EB101 Pivotal Phase III Vital Study were reported at the International Society for Investigative Dermatology, or ISID, and the Society for Pediatric Dermatology, SPD, meetings. The results presented at ISID and SPD showed that EB101 improved wound healing and pain reduction at 6, 12, and 24 weeks compared to control wounds following a one-time application of EB101. Furthermore, EB-101 demonstrated improvement in patient-reported and caregiver-reported outcomes for itch and blistering severity. Let's turn briefly to our preclinical ophthalmology programs. We're excited by the broad potential for treating serious eye diseases with new AAV-based therapies using novel AAV capsules from our in-licensed AIM capsule library and internal research. At ASGCT, we presented three posters highlighting encouraging findings from animal proof-of-concept experiments for investigative AAV-based gene therapies for Stargardt disease, X-linked retinoschisis, and autosomal dominant optic atrophy. The preclinical proof-of-concept data provides early evidence of the potential of our proprietary AAV capsules and gene constructs to express the recombinant protein and target tissues and rescue mutant phenotypes in mouse disease models. We completed pre-IND meetings with the FDA regarding preclinical development plans and regulatory requirements to support first-in-human trials for two preclinical gene therapy product candidates from our AAZ ophthalmology program. We're gathering additional proof-of-concept data before committing to IND-enabling tox studies, which we anticipate initiating in the second half of 2023. I'd now like to turn the call over to our Chief Financial Officer, Joe Hidalgo, who will review the second quarter financial results and recent capital rates. Joe?