ABEONA THERAPEUTICS INC. Q1 FY2023 earnings call

Key Points

• Vishwas Seshadri highlighted progress in EB-101 for recessive dystrophic EB (RDEB), including completion of three consecutive process performance qualification (PPQ) runs for manufacturing, pre-BLA meeting with FDA scheduled for July 10, 2023, and anticipation of BLA submission in early third quarter 2023 with potential approval in late 2023 or early 2024.
• Dmitriy Grachev reviewed VIITAL study results showing EB-101 improved wound healing and pain reduction at 6, 12, and 24 weeks vs. control wounds, with sustained benefits and improvements in patient-reported outcomes.
• Brian Kevany presented preclinical ophthalmology data from ASGCT, including ABO-504 for Stargardt disease, ABO-503 for X-linked retinoschisis, and ADOA gene therapy, demonstrating proof-of-concept in mouse models with gene expression and phenotype rescue.

No specific financial performance data for product segments provided in the transcript.

Forward-Looking Statements

• Pre-BLA meeting with FDA scheduled for July 10, 2023, with anticipation of BLA submission in early third quarter 2023 and potential approval in late 2023 or early 2024.
• Anticipation of being granted a priority review voucher if BLA is approved, which could be sold for approximately $100 million.
• Ongoing engagement with stakeholders on market access and pricing for EB-101.

Risks

• Factors causing actual results to differ from forward-looking statements, including those in Form 10-K and SEC reports.
• Uncertainties in BLA approval timeline and regulatory decisions.
• Challenges in translating preclinical ophthalmology data to human studies and ensuring successful clinical trial outcomes.

Q: Regarding the label for EB-101 based on conversations with the agency, what can be expected?

A: Vishwas Seshadri mentioned it's early in the BLA process, focusing on large chronic RDEB wounds, with an ongoing dialogue with the agency.

Q: Thoughts on pricing informed by Vyjuvek's launch?

A: Madhav Vasanthavada discussed payer research and the value proposition of EB-101's one-time treatment with durable benefit.

Q: How are ophthalmology programs prioritized?

A: Vishwas Seshadri and Brian Kevany discussed high unmet need in all indications and ongoing dialogue with the agency.

Q: Differentiation of dual AAV vector strategy in ophthalmology?

A: Brian Kevany explained use of Cre-LoxP recombinase system for efficient reconstitution of the ABCA4 gene in Stargardt disease.

Q: Translation of ophthalmology preclinical data to human studies?

A: Brian Kevany discussed seamless translation due to sub-retinal injection, similar dosing between large animals and humans.

Q: Visual acuity assessments in ADOA study?

A: Brian Kevany explained prevention of visual acuity degradation in early intervention with the Opa1 gene therapy.

Q: Importance of early data points in RDEB ISID study?

A: Vishwas Seshadri highlighted early clinical benefit at 3 weeks and sustained effects across time points.

Q: Impact of Vyjuvek approval on pricing estimates?

A: Vishwas Seshadri discussed taking into account RDEB's narrower space and unique value proposition.

Q: Pre-BLA meeting and partnering plans?

A: Vishwas Seshadri discussed pre-BLA meeting as a final step to finalize dossier and openness to partnering for rare disease delivery.