Abeona Therapeutics (ABEO) Stifel 2024 Healthcare Conference summary

Company overview and lead asset

• Focuses on cell and gene therapies for rare, debilitating diseases, with two platforms: ex vivo autologous cell therapy (lead asset pz-cel) and AAV-based in vivo gene therapies in preclinical ophthalmology.

• pz-cel is an autologous keratinocyte sheet for recessive dystrophic epidermolysis bullosa (RDEB), a severe connective tissue disorder.

• Clinical studies show pz-cel provides wound healing and pain reduction, with up to 10 years of follow-up from a single treatment.

• FDA review is ongoing, with a PDUFA date set for April 29th.

Regulatory and manufacturing updates

• Previous BLA received a CRL due to CMC issues, not clinical data; 12 items were addressed, with four major topics: sterility, replication competent retrovirus assays, cell-based identity assay, and sterile filtration controls.

• All FDA concerns have been comprehensively addressed, with robust validation and alignment on assay requirements.

• No changes to the manufacturing process since the phase 3 VIITAL trial; process has been preserved through tech transfer.

• Current GMP facility can ramp from 4–5 to 10 patients per month by end of 2025, with plans to expand further using leased space and modular construction.

Market opportunity and commercial strategy

• U.S. claims analysis estimates 1,300 dystrophic EB patients, with 750 moderate/severe RDEB patients as the core pz-cel market.

• Each patient may require two treatment cycles, equating to 1,500 treatment opportunities.

• Label is not expected to restrict by wound size or chronicity, potentially expanding eligible population.

• Market research indicates significant overlap with patients on Vyjuvek and Filsuvez; many patients may use multiple therapies.

• Launch will use a hub-and-spoke model, focusing on high-volume centers and leveraging existing patient records for access.