dm+d

Unassigned

New Medicines

Information

New molecular entity
REGENXBIO
REGENXBIO

Development and Regulatory status

None
None
Phase II Clinical Trials
Sep 21AbbVie and REGENXBIO enters into a partnership to develop and commercialise RGX 314. AbbVie will lead the clinical development and commercialisation of RGX 314 globally and will lead manufacturing of RGX 314 for commercial supply outside the US. [2-4]
Jan 21REGENXBIO announces intention to submit Biologics license application (BLA) to the US FDA for wet AMD in 2024. [8]

Category

AAV8 vector carries gene encoding for monoclonal antibody fragment, ranibizumab, which binds to & neutralises activity of vascular endothelial growth factor (VEGF), modifying the pathway new leaky blood vessel formation and retinal fluid accumulation.
The estimated prevalence of AMD in the UK is 4.8% of those over 65 years of age and 12.2% of those aged 80 years or more. There are around 70,000 new diagnoses in the UK each year [1].
Choroidal neovascularization (CNV) secondary to wet age-related macular degeneration (AMD) in people aged ≥50 years
Subretinal injection
Parenteral

Trial or other data

Oct 21The pivotal PII/III RCT, ATMOSPHERE, (n=300, NCT04704921) is recruiting pts to assess the mean change in BCVA to 54 weeks in pts with wet AMD randomised to single dose of RGX 314 or monthly Lucentis. The estimated primary completion date is March 2023 with full data expected March 2024. Another pivotal trial is also planned in Q4 2021.[2-4,7]
Oct 21Interim data from PI/II AAVIATE trial (NCT04514653) showed that 14 pts dosed with RGX 314 in cohort 1 demonstrated stable visual acuity (VA) at 6 months with a mean Best Corrected Visual Acuity (BCVA) change of -2.8 letters (95% CI: -7.0 to 1.4) from day 1 and -0.6 letters (-5.2 to 4.0) from week 1. Stable central retinal thickness (CRT)at 6 months was reported. Five pts receiving monthly injections of ranibizumab demonstrated mean BCVA change at 6 months of +6.8 letters (-3.3 to 16.9) from day 1 and +3.0 letters (-4.7 to 10.7) from week 1. Pts on monthly injections of ranibizumab also demonstrated stable CRT at 6 months from day 1. A significant reduction in anti-vascular endothelial growth factor (anti-VEGF) treatment burden was reported with RGX 314 vs. monthly injections. The trial is due to complete in Feb 22. [2-4,6]
Feb 21REGENXBIO initiated an open label, PII interventional trial (n=60, NCT04832724) for the estimation of pharmacodynamics of two doses in two formulations of RGX 314 gene therapy administered via subretinal delivery in pts with wet AMD. Results are expected mid 2022. [2-5]