Regenxbio (RGNX) investor relations material

Executive summary

• Achieved key milestones in late-stage gene therapy pipeline, including full enrollment in RGX-202 confirmatory study, FDA alignment for RGX-121 BLA resubmission, and positive Phase III results for Duchenne muscular dystrophy.

• Dosed first patient in NAAVIGATE trial for diabetic retinopathy, triggering a $100 million milestone payment from AbbVie in July 2026.

• Strengthened financial position with over $200 million in new capital and milestone payments, ending Q2 2026 pro forma with more than $310 million in cash and equivalents.

• Completed $107.8 million public offering in July 2026 to further bolster liquidity.

• Focused on executing pivotal readouts and regulatory submissions for multiple programs through 2027.

Financial highlights

• Q2 2026 revenues rose to $108.0 million from $21.4 million in Q2 2025, driven by the AbbVie milestone; net income was $22.7 million compared to a net loss of $70.9 million in Q2 2025.

• Cash, cash equivalents, and marketable securities were $105.5 million at June 30, 2026; pro forma liquidity exceeds $310 million after July events.

• R&D expenses for Q2 2026 were $56.1 million, down from $59.5 million in Q2 2025; G&A expenses rose to $21.6 million from $19.9 million.

• Gross margin for Q2 2026 was approximately 99% (cost of revenues $1.0 million on $108.0 million revenue).

• Diluted EPS for Q2 2026 was $0.43, compared to $(1.38) in Q2 2025.

Outlook and guidance

• Cash runway extends into Q4 2027, covering expected PDUFA date for RGX-202 and top-line data for wet AMD.

• BLA resubmission for RGX-121 planned for Q3 2026; U.S. approval for RGX-202 targeted for H2 2027.

• Topline data for pivotal wet AMD trials (ATMOSPHERE, ASCENT) expected Q4 2026; global regulatory submissions in 2027.

• Initiation of ex-U.S. AFFINITY RISE study for Duchenne planned for H1 2027.

• No additional studies required for RGX-121 BLA resubmission per FDA feedback.