ABEONA THERAPEUTICS INC. Q2 FY2023 earnings call

• EB-101 progress: Completed process performance qualification manufacturing runs for retroviral vector and drug product, aligned with FDA on RVV comparability, submitted briefing package for pre-BLA meeting, and pivotal Phase III VIITAL study data showed wound healing and pain reduction. - Preclinical ophthalmology: Exciting potential for AAV-based therapies for eye diseases, presented posters at ASGCT, completed pre-IND meetings with FDA. - Financials: License and other revenues $3.5M in Q2 2023, R&D spend $8.5M, G&A $5.0M, net loss attributable to common shareholders $16.7M.

No distinct product segments with revenue contributions discussed. Focus is on lead program EB-101 and preclinical ophthalmology programs.

• Anticipate submitting EB-101 BLA in Q3 2023, potential approval in Q2 2024; plan to engage with payers and prepare for commercialization. - Preclinical ophthalmology: Gather additional proof-of-concept data, initiate IND-enabling tox studies in the second half of 2023.

Factors causing actual results to differ from forward-looking statements, as mentioned in the opening about Risk Factors in Form 10-K.

Q: Have you gotten recent feedback on the FDA briefing package submission for the pre-BLA meeting and details on the pre-BLA meeting agenda?

A: Vish Seshadri discussed ongoing FDA interactions, focus on BLA package format and CMC/clinical data sufficiency.

Q: Can you talk about the commercial launch rollout and CMC scale-up for EB-101?

A: Madhav Vasanthavada talked about engaging with providers, payers, CMS, and mapping commercial roles.

Q: Thoughts on RDEB community awareness and ex-U.S. markets?

A: Madhav and Vish discussed patient advocacy, self-referrals, and interest in ex-U.S. markets.

Q: Thoughts on Crystal Biotech's patient start forms and CMC for EB-101?

A: Vish and Madhav commented on market size and CMC PPQ runs.

Q: RVV data publication and ophthalmology study protocols?

A: Vish discussed RVV data in BLA and ophthalmology program timelines.