ABEONA THERAPEUTICS INC. Q3 FY2023 earnings call

• Achieved submission of BLA for pz-cel (EB-101) to FDA in September 2023, a critical milestone. The BLA submission is a step toward potential approval, with a request for 6-month priority review potentially leading to Q2 2024 approval. • Planning for commercial launch includes onboarding 5-7 high-volume EB treatment centers and engaging with payers to ensure broad access. • Financials: As of September 30, 2023, cash, cash equivalents, restricted cash, and short-term investments totaled $54.1 million. R&D expenses for Q3 2023 were $7.1 million, and net loss attributable to common shareholders was $11.8 million. • Manufacturing team is preparing for preapproval site inspection and scaling up capabilities for commercial launch.

No specific product segments with financial performance details provided in the transcript.

• BLA submission with request for 6-month priority review, with potential approval in Q2 2024. • Financial resources are sufficient to fund commercial launch preparations and business operations into Q4 2024. • Initially plan to supply pz-cel for up to 120 patient treatments per year, with plans to scale capacity post-launch.

• Forward-looking statements are subject to change based on various factors, including those identified in the Risk Factors section of Form 10-K and periodic reports filed with the SEC.

Q: Elaborate on patient identification early on and involvement of prior trial participants.

A: Prior Phase I/II and Phase IIIb trial patients are involved, with an ad board indicating physicians believe patients may need multiple therapies. Patient identification is being formalized with analysis and claims data.

Q: How quickly will pz-cel be in the commercial field post-approval?

A: Post-approval, it is estimated to take 1-2 months to have processes in place for launch.

Q: Assumptions for peak sales of $500 million.

A: Projection is based on 500 patients per year over 5 years with 7-digit pricing, with capacity ramping up after launch based on initial demand.

Q: Reaching community doctors vs centers of excellence.

A: Focus on centers of excellence initially, then expand to community centers, working with patient advocacy groups for referrals.

Q: Phase III trial age limit.

A: Dialogue with FDA on age limit, currently 6+ but applicable to younger patients, with ongoing Phase IIIb study helping to address this.

Q: Hiring for commercial organization and challenges.

A: No issues hiring, focus on key account managers and medical teams, with no major sales team at launch.

Q: Takeaways from Krystal Biotech's launch.

A: Positive uptake helps raise awareness, pz-cel benefits from fast follower position, aiding in access clearance and awareness for patients and physicians.

Q: Additional indications for pz-cel.

A: Focus on launching pz-cel first, with plans to consider life cycle improvements for different products based on unmet needs identified.