Regenxbio (RGNX) Baird's 2024 Global Healthcare Conference summary

Pipeline and technology focus

• Fifteen years of AAV gene therapy innovation, with a shift from out-licensing to internal development and a current pipeline of four late-stage assets in retinal and rare disease franchises.

• Advancing RGX-202 for Duchenne muscular dystrophy (DMD), RGX-314 for ocular diseases, and RGX-121 for Hunter syndrome, with pivotal trials and BLA filings planned or underway.

• Partnership with AbbVie supports large-scale pivotal trials in wet AMD and diabetic retinopathy, using subretinal and suprachoroidal delivery methods.

Ophthalmology programs and clinical strategy

• RGX-314 aims to create a biofactory for anti-VEGF protein in the eye, targeting durability, efficacy, and disease progression in wet AMD.

• Two global phase III trials (ATMOSPHERE and ASCENT) compare RGX-314 to Lucentis and Eylea, with BLA filings planned for the first half of 2026.

• Subretinal and suprachoroidal routes offer immune privilege and safety advantages; phase I/II data show long-term vision gains and reduced need for injections.

• Suprachoroidal delivery in diabetic retinopathy halted disease progression in 90% of patients at one year, with pivotal trials expected to start next year.

Commercial and market positioning

• Single-injection gene therapy for diabetic retinopathy could overcome compliance issues seen with intravitreal injections, especially in working-age, non-compliant patients.

• Early adoption expected in patients with complications in one eye, with broader uptake over time.

• AbbVie partnership covers two-thirds of development costs and shares U.S. commercialization 50/50.