REGENXBIO Inc. Q4 FY2024 earnings call

• Submitted first BLA for RGX-121 (Hunter syndrome treatment) and expect FDA approval in Q4 2025.
• Advancing diabetic retinopathy program to pivotal stage in 2025, with potential BLA filings for RGX-202 (Duchenne) and ABBV-RGX-314 (wet AMD) in 2026.
• RGX-202 pivotal study for Duchenne is progressing rapidly, with a mid-2026 BLA filing expected.
• ABBV-RGX-314 retinal franchise in late-stage studies; pivotal trials for wet AMD to complete enrollment in 2025 and diabetic retinopathy pivotal study planned with AbbVie in 2025.
• MPS program: BLA for RGX-121 submitted, based on campsite trial data meeting primary endpoint with high statistical significance.
• Clinical progress: RGX-202 phase one/two data showed positive safety, efficacy, and biomarker results; 314 suprachoroidal program has a differentiated safety profile; MPS program's BLA is based on a surrogate endpoint reasonably likely to predict clinical benefit.

No detailed segment performance data by product segments provided in the transcript, as it does not break down financial performance by absolute revenue and contribution % for product segments.

• Expect FDA approval of RGX-121 in Q4 2025.
• Advance diabetic retinopathy program to pivotal stage in 2025, with potential BLA filings for RGX-202 and ABBV-RGX-314 in 2026.
• RGX-202 pivotal study to complete enrollment in 2025 and share top-line data in H1 2026.
• ABBV-RGX-314: complete enrollment of wet AMD pivotal trials in 2025 and plan pivotal study for diabetic retinopathy in 2025.

• Regulatory risks associated with BLA reviews for RGX-121, RGX-202, and ABBV-RGX-314.
• Timing risks for non-dilutive financing events like DR milestone, PRV monetization, and Zolgensma royalty reverts.
• Competition risks in wet AMD and Duchenne markets.

Q: Delve deeper into components of non-dilutive financing and probabilities of realizing them, including PRV and AbbVie DR milestone.

A: Mitchell Chan discussed DR milestone gated on first patient dose, PRV monetization upon regulatory approval, and Zolgensma royalty revert once HGR cap is hit.

Q: NDA update on RGX-202, timing of data update, and enrollment including younger patients.

A: Curran and Steve discussed updating functional data in H1 2025, enrolling across age ranges including 1-3, and higher microdystrophin levels in younger patients.

Q: Cardiac endpoints in RGX-202 study and wet AMD subretinal trial enrollment.

A: Steve commented on measuring cardiac endpoints and no plan to cut short wet AMD subretinal trial enrollment.

Q: Regulatory discussions for DR, data disclosure in DMD, and enrollment in pivotal.

A: Steve discussed clear regulatory path for DR, DMD data disclosure including new patients, and pivotal enrollment including new and phase one/two patients.

Q: Continuity in regulatory discussions, screening in RGX-202, and post-trial commitments.

A: Curran and Steve noted stable regulatory discussions, broad screening in RGX-202, and plans for confirmatory studies post-pivotal.

Q: Functional data in RGX-202, treatment durability in DR, and market dynamics in DMD.

A: Curran and Steve discussed functional data updates, DR treatment durability, and market plans for RGX-202 commercial launch.

Q: Sales team for RGX-121 and sales-related milestones.

A: Curran discussed sales team alignment with NS Pharma and potential sales milestones in first year of launch.