Gene therapy developers REGENXBIO (RGNX -11%) and Adverum Biotechnologies (ADVM +17.4%) have diverged after data presentations on their respective gene therapy candidates for wet age-related macular degeneration (wet AMD) at the American Academy of Ophthalmology Annual Meeting in San Francisco.

Additional results from the first cohort (median follow-up of 34 weeks) in the Phase 1 OPTIC study showed that treatment-experienced patients (n=6) previously requiring frequent anti-VEGF injections to maintain vision did not require any rescue injections after receiving a single administration of ADVM-022 while maintaining stable visual acuity.

Preliminary results from cohort 5 in a Phase 1/2a study of RGX-314 showed that 75% (n=9/12) of patients were anti-VEGF injection-free at months 5 or 6 while the remaining subjects still required injections. Across all 12 patients, the mean annualized anti-VEGF injection rate was 0.8, implying that three patients required an annualized average of more than three injections [(0.8 x 12)/3=3.2]. In cohort 4, 42% (n=5/12) were anti-VEGF injection-free at month 6 with an average of 2.2 injections over that time period, implying that seven patients received an average of almost four injections over six months [(2.2 x 12)/7=3.8].

Regeneron Pharmaceuticals (REGN +2.5%), maker of top wet AMD seller Eylea (aflibercept), an anti-VEGF therapy, is up on average volume.





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