Abeona Therapeutics (ABEO) Jefferies Global Healthcare Conference summary

Program overview and clinical progress

• Lead program pz-cel is a gene-corrected cell therapy for large wounds in RDEB, showing durable wound closure up to eight years in follow-up and meeting pivotal trial endpoints in 2022.

• BLA was filed in 2023 with an original PDUFA date of May 2024; a CMC-related CRL was received in April 2024, with no clinical efficacy or safety concerns raised.

• Resubmission to FDA is planned for the second half of 2024, aiming to address all CMC issues by Q3.

• Additional pipeline includes AAV-based therapies for inherited retinal dystrophies, but current focus is on pz-cel.

Regulatory and manufacturing updates

• CMC-related CRLs are generally more resolvable than clinical CRLs; 11 of 12 CMC CRLs reviewed were ultimately approved.

• Key CRL deliverables include rapid sterility assay (completion by mid-Q3), retroviral replication competency (completed), sterile filter validation (completion by June), and cell-based identity assay (targeting late July).

• Manufacturing is primarily in-house, with about 30% of testing at CROs; facility has been FDA-inspected.

• Type A meeting with FDA is planned for July to align on data package and outstanding items.

• All CMC issues in the CRL also address Form 483 observations.

Commercial strategy and market insights

• Launch planned at 5-7 high-volume, geographically dispersed centers, with active engagement and education of EB physicians and surgeons.

• Initial demand expected from 20-50 patients per site, with broader outreach to 23 centers of excellence and a tight-knit, well-informed patient community.

• Estimated eligible RDEB patient base is 750-1,200, with a conservative lower bound of 600; potential for 300-500 additional dominant DEB patients if label expands.

• Pricing will be per treatment cycle, aligned with recently launched gene therapies, and payers recognize the value of durability and large wound coverage.