REGENXBIO Inc. Q2 FY2024 earnings call

• RGX-202: Next-generation microdystrophin gene therapy for Duchenne. Positive microdystrophin expression data across ages, well-tolerated with no SAEs. Completed end-of-Phase 2 meeting with FDA, plan to file BLA for accelerated approval. In-house manufacturing capacity to produce 2,500 doses of RGX-202 per year.
• AbbVie RGX-314: Collaboration for chronic retinal diseases. Accelerated end-of-Phase 2 meeting with FDA to Q4 2024 (previously Q1 2025), entitled to $200M milestone on first patient dose in DR. Expanding ALTITUDE trial to include diabetic macular edema (DME). Progress on wet AMD and RGX-121 programs.
• RGX-121: For MPS II. On track to initiate rolling BLA filing in Q3 2024, potential priority review voucher in 2025.

No detailed financial breakdown by product segments provided in terms of absolute revenue and contribution % as per the transcript.

• Cash, cash equivalents, and marketable securities of $327M as of June 30, 2024, expected to fund operations into 2026.
• RGX-202: Finalizing pivotal plans for accelerated approval.
• RGX-314: Accelerated end-of-Phase 2 meeting to Q4 2024, initiation of pivotal trial in H1 2025.
• RGX-121: On track for Q3 2024 rolling BLA filing, potential priority review voucher in 2025.

Forward-looking statements subject to risks and uncertainties described in REGENXBIO's annual report on Form 10-K for the full year ended December 31, 2023, and comparable risk factors sections of quarterly reports on Form 10-Q, available on the SEC's website.

Q: About RGX-202 protein level goal and FDA threshold, and RGX-314 dose level 4 and neutralizing antibodies.

A: Curran and Steve addressed microdystrophin level threshold above 10% and reasons for dose level 4.

Q: Functional data for RGX-202 and path to pivotal for RGX-314.

A: Curran and Steve discussed functional data timeline and pivotal path for 314.

Q: Interest in RGX-202 patient community and recruitment, and RGX-314 dose level 4 and rescue usage.

A: Curran and Steve talked about recruitment interest and potential reduction in rescue usage.

Q: Safety database for RGX-202 approval.

A: Curran mentioned ongoing discussions with FDA on sample size.

Q: RGX-202 strategy for younger patients and RGX-314 benchmarking.

A: Curran and Steve discussed age range expansion and benchmarking.

Q: Microdystrophin data beyond 3 months for RGX-202 and patient characteristics.

A: Curran and Steve talked about future data and no clear predictors.

Q: RGX-202 strategy in DMD and confirmatory trial endpoints.

A: Curran and Steve discussed broad approval strategy and use of North Star endpoint.

Q: Phase 3 plans for DR and DME cohort in RGX-314.

A: Curran and Steve discussed confidence in pivotal initiation and DME cohort details.

Q: Data from Fellow Eye study and long-term strategy for RGX-314 suprachoroidal.

A: Steve talked about Fellow Eye enrollment and confidence in suprachoroidal safety.