Regenxbio (RGNX) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Key business inflection points and leadership transition

• Advanced RGX-202 Duchenne program from first patient in phase 1 to readiness for pivotal study by year-end.

• Imminent first BLA filing for RGX-121 (Hunter disease), with potential approval and first sales next year.

• CEO transition leveraged long-term internal experience to maintain program continuity.

RGX-202 program differentiation and clinical progress

• RGX-202 uses AAV8 serotype and a microdystrophin construct with the C terminus, aiming for improved function.

• Advanced manufacturing enables high yield and purity, supporting pivotal dosing.

• Early clinical data show high microdystrophin expression, especially in older boys, a challenging group.

• Pivotal trial design agreed with FDA: no placebo control, use of historical/matched controls, and accelerated approval pathway.

• Market potential estimated at $7–9 billion, with significant opportunity in the prevalent population.

RGX-314 retinal programs and commercial strategy

• Phase 3 subretinal wet AMD trial expected in 2025, targeting sustained anti-VEGF effect and reduced injection burden.

• Subretinal delivery allows for fellow eye treatment and is considered the gold standard for safety and efficacy.

• Suprachoroidal delivery offers a simpler, scalable in-office procedure, with infrastructure already in place.

• Both subretinal and suprachoroidal approaches expected to coexist, tailored to patient needs.

• Diabetic retinopathy program advancing, with phase II meeting accelerated to Q4 this year due to strong phase II data.