Abbvie puts first toe in gene therapy, courtesy of Regenxbio

Snippets

With safety concerns continuing to dog AAV-based gene therapies Regenxbio’s ability to bag Abbvie as partner for RGX-314, its gene therapy for eye diseases, is encouraging. Shares in the small biotech opened 23% higher, with a hefty $370m up front contributing to the rise. On a call executives declined to say whether Abbvie had seen any of the data due on RGX-314, though the assumption is presumably yes. Most importantly, investors are waiting for a first look at suprachoroidal delivery of RGX-314, requiring a simple in-office procedure rather than the surgery involved in subretinal administration, and success could substantially broaden commercial potential. Still, many groups are going after longer-acting VEGF agents, an area that is already very competitive; Regenxbio is arguably one of the most advanced with a gene therapy, however, so perhaps Abbvie thought the opportunity was worth a bet. The big pharma group has a large eye care division, though this will be its first step into gene therapy. The terms of the deal see the partners sharing future development costs, with Abbvie leading commercialisation; Regenxbio will co-commercialise and share US profits, and receive royalties in “late teens/early 20s” percent elsewhere.

Big events on the horizon for RGX-314
Project Indication Progress
RGX-314 suprachoroidal Wet AMD (phase 2) AAViate trial: 6mth data from Cohort 1 (lowest dose) due at Retina Society meeting (Sep 29- Oct 2), and time point data for other cohorts
Wet AMD (phase 2) AAViate trial: cohort 2 (higher dose) data to be released Q4 2021; cohort 3 (NAb positive) enrolling
Diabetic retinopathy (phase 2) Altitude trial: cohort 1 completed enrolling, data due Q4 2021; cohorts 2 (higher dose) and 3 (NAb positive) enrolling
RGX-314 subretinal Wet AMD (phase 3) Atmosphere trial enrolling (completes 2023); 2nd pivotal study to start H2 2021; BLA planned 2024
NAb=neutralising antibody. Source: company statements. 

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