Adverum Biotechnologies reports positive interim data from three cohorts of OPTIC phase 1 trial
New interim data for the OPTIC phase 1 dose-ranging clinical trial of ADVM-022 intravitreal injection gene therapy for the treatment of wet age-related macular degeneration showed robust efficacy in each cohort, long-term durability in cohort 1 and the potential for fewer adverse events in cohort 3, according to a press release from Adverum Biotechnologies.
Three cohorts of patients have been treated with a single injection of ADVM-022. The first cohort of six participants was treated with a higher dose of 6 × 1011 vg per eye compared with six participants in the second cohort treated with a lower dose of 2 × 1011 vg per eye. Nine participants in the third cohort were treated with the same lower dose but underwent a 6-week eye drop prophylactic steroid regimen, while cohorts 1 and 2 received a 13-day oral prophylactic steroid regimen.
Interim data showed all six patients in cohort 1 remained free of rescue injections beyond 1 year. Eight of 11 patients who received the lower dose remained free of rescue injections with less follow-up time compared with cohort 1.
Early data from cohort 3 showed the positive potential of the 6-week prophylactic regimen of steroid eye drops, with less inflammation and fewer adverse events when compared with the 13-day prophylactic regimen of oral steroids in cohorts 1 and 2. No patient in cohort 3 required treatment beyond steroid eye drops or had cellular inflammation above grade 1+.
Additionally, in the first five patients with 20 weeks of follow-up in cohort 3, central retinal thickness was reduced by a mean 137.8 µm, and the mean increase in best corrected visual acuity was 6.8 letters.
Cohort 4 is currently being enrolled, with patients receiving the higher dose of ADVM-022 and the same steroid regimen as cohort 3.