Regenxbio's Q2 2025: Key Contradictions on RGX-121 Approval, Manufacturing Plans, and Pivotal Study Design

RGX-121 BLA submission and approval timeline, RGX-202 manufacturing plans, pivotal study design and efficacy, expected launch timeline, and rationale for dose level 4 in the pivotal study are the key contradictions discussed in REGENXBIO's latest 2025Q2 earnings call.



RGX-202 Progress and Market Positioning:
- REGENXBIO reported that enrollment in the AFFINITY DUCHENNE pivotal trial will be completed by October, ahead of schedule.
- The acceleration is due to strong interest from the Duchenne community and a robust enrollment pace.

RGX-121 BLA Acceptance:
- The FDA accepted REGENXBIO's BLA for RGX-121 for Hunter syndrome under the accelerated approval pathway with a target PDUFA date of November 9.
- This is a significant milestone as RGX-121 would be the company's first approved gene therapy.

Sura-vec Advancement for Diabetic Retinopathy:
- A new Phase IIb/III trial for surabgene lomparvovec (sura-vec) was announced, following positive 2-year data from the Phase II ALTITUDE trial.
- The advancement is due to the demonstrated durable efficacy and safety profile of the product.

Manufacturing Capabilities and Cash Runway:
- REGENXBIO initiated commercial manufacturing of RGX-202 at its Rockville facility, with the capacity to produce 2,500 doses per year.
- The cash runway is expected to support operations until early 2027, enabled by milestone payments and royalty monetization.