Abeona Therapeutics(ABEO) - 2023 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of June 30, 2023, the company had cash, cash equivalents, restricted cash, and short-term investments of $37.1 million, down from $40.7 million as of March 31, 2023 [9] - License and other revenues for Q2 2023 were $3.5 million, compared to $1 million in Q2 2022, driven by a clinical milestone achieved under a sub-agreement [10] - The net loss attributable to common shareholders was $16.7 million for Q2 2023, or $0.92 loss per share, compared to a net loss of $7.9 million, or $1.36 loss per share, in Q2 2022 [10] Business Line Data and Key Metrics Changes - Significant progress was made in the EB-101 program, with completed process performance qualification (PPQ) manufacturing runs for both retroviral vector and EB-101 drug product [5] - Additional efficacy and safety data from the EB-101 pivotal Phase III VIITAL study showed improved wound healing and pain reduction at six, 12, and 24 weeks compared to control wounds [6] Market Data and Key Metrics Changes - The company anticipates a priority review based on the expected timing of the BLA submission, with potential approval in Q2 2024 [5] - The company is engaging with top EB treatment centers in the U.S. and has received positive feedback regarding the high unmet need for EB-101 [6][19] Company Strategy and Development Direction - The company plans to submit the EB-101 BLA in Q3 2023, with preparations for commercialization already underway [5][6] - The company is focusing on building a sales force and engaging with payer groups to ensure broad market access post-approval [19] - The company is also preparing for potential expansion into international markets, with interest from pharmaceutical companies in Europe and Japan [25] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress made with the FDA regarding the BLA submission and the comparability of retroviral vectors [14][15] - The management highlighted the importance of patient advocacy groups in raising awareness and facilitating self-referrals as the launch approaches [22] - The company is optimistic about the market opportunity, citing the potential for increased diagnosis and awareness of RDEB patients [24] Other Important Information - The company raised $25 million in a registered direct offering in July 2023 to fund pre-commercialization activities [6] - The company has mapped out key commercial and medical roles to be filled in the second half of the year to support a successful launch [19] Q&A Session Summary Question: Feedback on the briefing package and pre-BLA meeting agenda - Management indicated that they have had collaborative interactions with the FDA and are confident in the data submitted for RVV comparability [14][15] Question: Commercial launch rollout and CMC scale-up - Management confirmed that they are preparing for the commercial launch and will be careful about capacity expansion until after approval [16][17] Question: Awareness in the RDEB community - Management expects increased awareness and self-referrals as the launch approaches, aided by patient advocacy groups [22] Question: Market size and patient access - Management noted that it is early to speculate on market size based on competitor data but remains encouraged by the initial feedback [28] Question: CMC activities prior to BLA filing - Management confirmed that they have completed the necessary CMC activities and are on track for a Q3 submission [31]