Regenxbio (RGNX) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic overview and pipeline progress

• Marked as a transformational year with multiple late-stage clinical readouts and a BLA under review for the Hunter program, with a PDUFA date set for February 8th.

• Top-line pivotal data for the Duchenne program (RGX-202) expected in Q2 2026, with BLA submission targeted for mid-2026.

• Wet AMD program partnered with AbbVie to deliver late-stage data by year-end, potentially the first non-rare gene therapy approval.

• Phase II-B/III study for diabetic retinopathy to begin in the first half of the year, with a $100 million milestone upon first patient dosing.

• Expansion plans include global studies and commercial readiness, leveraging in-house manufacturing capabilities.

Differentiation and technology advancements

• Proprietary AAV8 and AAV9-based NAV technology with over 5,000 patients dosed and five licensees.

• New capsids in preclinical development aim to improve tissue specificity and therapeutic window, especially for ocular and liver applications.

• Industry-leading manufacturing with high-purity capsids and scalable processes, validated by FDA inspection with no observations.

• Achieved 80% full capsid levels for Duchenne program, supporting safety and efficacy.

• End-to-end in-house capabilities enable rapid scale-up and global supply.

Duchenne muscular dystrophy (RGX-202)

• Novel construct includes C-terminus for improved muscle repair, showing durable and increasing functional benefit out to 18 months.

• All participants in phase I/II demonstrated microdystrophin levels above 10%, with no SAEs or AESIs observed.

• Functional improvements (NSAA) at 18 months reached a delta of 7.4, well above the clinically meaningful threshold.

• Proactive immune suppression regimen and high-purity manufacturing differentiate the program and have influenced competitors.

• Manufacturing capacity supports up to 2,500 doses per year, with global expansion and broad age inclusion criteria.