REYON Pharmaceutical(102460) announced 44-week results for the mid-dose cohort of NG101, a gene therapy for neovascular age-related macular degeneration it is co-developing with Elixigen. The trial showed that after the low dose, the mid dose also reduced injection frequency by 90%, and participants' vision improved.
REYON Pharmaceutical said on the 20th that at the world's largest retina specialists' meeting (ASRS), held in Montreal, Canada, from the 15th to the 18th, Elixigen presented the 44-week results for the mid-dose cohort. The presentation was given by Peter, a University of Toronto professor who is the principal investigator (PI) of the NG101 phase 1/2a clinical trial.
According to the presentation, even in the mid-dose cohort, the number of anti-VEGF injections decreased by 90% after NG101 administration compared with before administration. The additional injection reduction rate was assessed based on the 44-week data. If extended follow-up does not increase additional anti-VEGF dosing, the reduction rate in injections over the entire period could rise further.
In addition, the mid-dose cohort showed better efficacy than the low-dose cohort in vision improvement and retinal structure-related metrics. In terms of safety, despite the higher dose, all patient groups showed generally good tolerability, and there were no reports of drug-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs).
Previously, through the low-dose cohort results, Elixigen also demonstrated the possibility of significantly reducing the treatment burden for patients who would otherwise need about 8–10 anti-VEGF injections per year with a single administration.
Because most specialty medicines, including AAV gene therapies, can see increased adverse events as the dose rises, it is important to show sufficient medical efficacy at low doses. In this respect, Elixigen's gene therapy, at the low dose, is at or below 0.8%–3.3% of global competitors' dosing, and even at the mid dose is at 2.3%–10%, which the company said raises the likelihood that, once commercialized, market preference will concentrate on it.
Elixigen plans to present 24-week results for the high-dose cohort at the American Academy of Ophthalmology (AAO) in Oct. Kim Jong-muk, CEO of Elixigen, said, "It was an opportunity to widely publicize to the global ophthalmology community and business partners the clinical potential of NG101 to dramatically reduce the anti-VEGF injection burden with a single administration, consistently across both low- and mid-dose cohorts," and added, "Once we secure the high-dose cohort data in October this year, licensing talks are likely to gain momentum."
Elixigen's NG101 is a pipeline under development with partner REYON Pharmaceutical based on a co-development contract signed in 2020. REYON Pharmaceutical is responsible for global production and supply of NG101 and plans to manufacture the gene therapy at the newly built GMP facilities at its Chungju plant when commercial production begins.