Regenxbio (RGNX) H.C. Wainwright 4th Annual Ophthalmology Virtual Conference summary

Panel composition and context

• Leading experts and executives from multiple ophthalmology companies and academia discussed the evolving therapeutic landscape for age-related macular degeneration (AMD).

• The panel included specialists with high-volume clinical experience and leaders from companies developing novel AMD therapies.

Current treatment paradigms and challenges

• Eylea, faricimab (Vabysmo), Avastin, Lucentis, and Cimerli are widely used, with insurance and socioeconomic factors influencing drug choice.

• Step therapy and insurance requirements often dictate initial use of Avastin before branded drugs.

• Durability and reducing treatment burden are key unmet needs, especially for patients traveling long distances.

Novel therapies and mechanisms in development

• RGX-314 (REGENXBIO): Subretinal gene therapy aiming for sustained anti-VEGF effect, with phase III data expected by end of 2025 and BLA submission in 2026.

• DURAVYU (EyePoint): Small molecule TKI in sustained-release implant, showing strong efficacy and safety in phase II, with phase III trials starting in 2024 and readout in 2026.

• CLS-AX (Clearside): Suprachoroidal TKI delivery, aiming for flexible dosing intervals and minimal endophthalmitis risk, with phase II results expected soon.

• AXT107 (AsclepiX): Integrin-regulating peptide in suprachoroidal suspension, targeting multiple angiogenic pathways, with phase I/2a safety data expected in 2025.

• VLTR-559 (Valitor): Preclinical anti-VEGF biopolymer conjugate designed for 6–8 month durability, leveraging hyaluronic acid for extended ocular retention.

• ORAI1 blocker (ForwardVue): Preclinical small molecule with pleiotropic anti-angiogenic action, delivered via PLGA implant for linear release over 100 days.