Regenxbio (RGNX) investor relations material

Key milestones and program updates

• 2026 is highlighted as a pivotal year with several major milestones, including a February PDUFA date for the Hunter program and top-line data for the Duchenne program in early Q2.

• Wet AMD subretinal program is expected to deliver top-line data late next year, with pivotal readouts for RGX-314 in Q4.

• Manufacturing facility in Rockville has been modernized and inspected with no observations, supporting scalability and de-risking CMC aspects.

• Commercialization for the Hunter program will be handled by NS Pharma, leveraging newborn screening and established patient advocacy relationships.

• RGX-121 aims to be the first gene therapy in the ERT market, offering a one-time treatment alternative with strong adoption expected.

Strategic differentiation and competitive landscape

• One-time gene therapy is positioned as a major differentiator, especially for CNS manifestations where current treatments are lacking.

• Early treatment and strong data in younger patients are emphasized as key advantages, with newborn screening facilitating early identification.

• Duchenne program leverages unique construct design including the C-terminus, aiming for better functional benefit and safety profile.

• Phase 1/2 data for Duchenne shows improvement in older patients and no serious adverse events, supporting a compelling benefit-risk profile.

• Plans for initial BLA filing for Duchenne in mid-2026, with readiness for commercialization and openness to global partnerships.

Market opportunities and financial outlook

• Wet AMD market is estimated at $6–8 billion, potentially exceeding $10 billion by launch, with gene therapy offering a less burdensome, one-time solution.

• Partnership with AbbVie for commercialization in Wet AMD, with 50/50 profit sharing in the U.S. and royalties ex-U.S.

• Diabetic retinopathy program is advancing, with a $100 million milestone expected for first patient dosing in phase 2B, and another $100 million for the second pivotal.

• Cash position is sufficient to fund operations into early 2027, with potential to extend into late 2027 or early 2028 through non-dilutive financing and milestones.

• Institutional expertise in gene therapy, focus on safety, and transparent data sharing are highlighted as core strengths.