Regenxbio (RGNX) Morgan Stanley 24th Annual Global Healthcare Conference summary

Gene therapy pipeline and development progress

• Advanced AAV gene therapy platform with internalized programs in retina and rare diseases, including Duchenne muscular dystrophy (DMD) and Hunter syndrome.

• RGX-202 for DMD shows differentiated design with C-terminus addition, high purity, and modern manufacturing, aiming for commercialization in 2027.

• AFFINITY DUCHENNE study enrolled patients aged 1 and older, showing high and consistent microdystrophin expression, with 80% of patients achieving 40% or more of normal levels.

• Statistically significant correlation between microdystrophin expression and NSAA functional outcomes, with a correlation coefficient of 0.9.

• Safety profile of RGX-202 demonstrates less than one-tenth the liver injury rate compared to existing therapies, supported by proactive immune modulation regimen.

Regulatory strategy and commercialization plans

• Pre-BLA meeting planned for end of 2026, with BLA submission for RGX-202 targeted for early 2027 and potential U.S. launch in the second half of 2027.

• Approximately 63 patients' safety data and 30 pivotal study patients' biomarker data will support the filing, with half having 12-month functional data.

• Confirmatory study for RGX-202 completed enrollment, with primary endpoint at two years; placebo-controlled study planned for global submissions.

• Manufacturing capacity allows for 2,500 doses per year, with hundreds of doses expected to be available at launch.

• Cash runway extends to Q4 2027, with potential for additional non-dilutive capital from milestones and commercial revenue by 2028.

Retina franchise and future opportunities

• Phase III data for anti-VEGF gene therapy (314) in wet AMD expected in Q4 2026; program aims for significant reduction in injection burden and long-term durability.

• Largest gene therapy program in wet AMD, with two global pivotal studies powered for non-inferiority to Lucentis and EYLEA, using a 4.5-letter margin.

• Subretinal delivery chosen for safety, with no observed immune-mediated or inflammatory responses and five-year durability data.

• Commercial preparations underway with AbbVie, targeting both U.S. and global markets.

• Diabetic retinopathy program (DR NAAVIGATE) initiated with suprachoroidal delivery, robust early recruitment, and $100 million milestone achieved.