Regenxbio (RGNX) investor relations material

Strategic program updates and clinical progress

• Advanced gene therapy pipeline includes late-stage programs in retina, CNS, and Duchenne muscular dystrophy (DMD), with in-house manufacturing supporting rapid development.

• RGX-202 for DMD shows strong safety and efficacy, with robust microdystrophin expression and positive functional outcomes, especially in older patients; pivotal trial enrollment expected to complete by October.

• Regulatory pathway for DMD remains stable, with functional data included alongside microdystrophin as endpoints; commercial launch targeted for 2027, with growing market opportunity.

• RGX-121 for Hunter syndrome partnered with MS Pharma, with double-digit royalties and a monetization agreement; FDA requested 12-month data, which was provided, supporting the BLA review.

• Retinal disease program (RGX-314) with AbbVie restructured to accelerate pivotal studies, leveraging strong phase II data and flexible partnership milestones.

Commercial and operational outlook

• Commercial production underway in Rockville, Maryland, positioning for timely launches; payer discussions and manufacturing readiness support rapid post-approval patient access.

• Cash runway extends into early 2027, with potential to reach 2028 through PRV monetization and additional milestone payments; non-dilutive financing options remain available.

• No significant impact from tariffs; FDA interactions remain constructive, with positive feedback on rare disease development and ongoing BLA processes.

• AI is being leveraged for capsid discovery and optimizing clinical site selection, with ongoing exploration of further applications.

• Competitive threat from China in gene therapy seen as minimal in the near term due to strong internal capabilities and industry collaboration.

Market dynamics and unmet needs

• DMD program benefits from high patient and clinician interest, with next-generation therapy differentiation driving strong trial enrollment.

• Hunter syndrome launch readiness aligns with regulatory timelines, with MS Pharma prepared for commercialization and reimbursement discussions underway.

• Retinal disease programs address significant unmet needs in DR, DME, and wet AMD, with market expansion expected and gene therapy positioned as a transformative option.

• Superchoroidal delivery in retinal programs is scalable and well-accepted by clinicians, with rapid recruitment anticipated for upcoming pivotal studies.

• AbbVie partnership validates both subretinal and superchoroidal approaches, supporting broad clinical and commercial advancement.