Regenxbio (RGNX) investor relations material

Executive summary

• Reported a net loss of $90.1 million for Q1 2026, compared to net income of $6.1 million in Q1 2025, driven by lower revenues and a one-time $10 million settlement expense.

• Revenues declined sharply to $6.4 million from $89.0 million year-over-year, primarily due to lower license and royalty revenue and the absence of prior year milestone payments.

• Achieved positive pivotal data for RGX-202 in Duchenne muscular dystrophy, meeting primary endpoint with high statistical significance and favorable safety profile; confirmatory trial enrollment ongoing.

• Advanced late-stage pipeline with key milestones in gene therapies for Duchenne, wet AMD, diabetic retinopathy, and Hunter syndrome; clinical hold lifted for RGX-121.

• Manufacturing capabilities ramped up to support anticipated commercial needs.

Financial highlights

• License and royalty revenue fell to $5.1 million from $87.0 million, mainly due to a $70 million upfront payment from Nippon Shinyaku in Q1 2025 and lower Zolgensma royalties after U.S. patent expiry.

• Service revenue decreased to $1.3 million from $2.0 million, reflecting lower development and manufacturing services.

• Operating expenses rose to $89.8 million from $76.9 million, with a $10 million non-recurring GSK settlement charge and higher R&D and G&A costs.

• Cash, cash equivalents, and marketable securities totaled $150.5 million as of March 31, 2026, down from $240.9 million at year-end 2025, mainly due to operating activities and the settlement payment.

• R&D expenses increased to $57.3 million from $53.1 million year-over-year, driven by clinical trial and personnel costs.

Outlook and guidance

• Cash runway expected to fund operations into early 2027, but substantial doubt exists about the ability to continue as a going concern without additional capital.

• Plans to pursue accelerated approval and commercial launch of RGX-202 in 2027, with ongoing regulatory discussions for RGX-121 and ABBV-RGX-314.

• Topline data from pivotal wet AMD trials expected in Q4 2026; regulatory submissions planned for 2027.

• Completion of dosing in all RGX-202 pivotal and confirmatory trial patients anticipated by mid-year.