Adverum Biotechnologies, Inc. (Nasdaq: ADVM) today announced new interim clinical data from the OPTIC Phase 1 dose-ranging clinical trial of ADVM-022 intravitreal injection gene therapy. OPTIC includes treatment-experienced patients with wet age-related macular degeneration (AMD). The data are being presented today by David S. Boyer, M.D., senior partner, Retina-Vitreous Associates Medical Group and adjunct clinical professor of ophthalmology with the University of Southern California/Keck School of Medicine in Los Angeles, at the Angiogenesis, Exudation, and Degeneration 2020 Annual Meeting in Miami.
A copy of the presentation is available on the Adverum corporate website under Events and Presentations in the Investors section.
For the first time, data are being presented from patients in cohort 2 (n=6) at 24 weeks following treatment with a single intravitreal injection of a three-fold lower dose of ADVM-022 (2 x 10^11 vg/eye) compared to the cohort 1 dose (6 x 10^11 vg/eye). New data as detailed in the table below include:
ADVM-022 demonstrated a robust efficacy signal and evidence of a dose response:
Cohort 1: 6 of 6 patients remain rescue-injection-free at a median follow up of 50 weeks, with 3 patients at 52 weeks.
Cohort 2: 4 of 6 patients remain rescue-injection-free at 24 weeks at the lower dose.
In both cohorts combined, 10 of 12 (83%) patients remain rescue-injection-free. For these patients:
Vision was generally maintained as demonstrated by stable mean best corrected visual acuity (BCVA) compared to baseline.
Retinal anatomy improvements were achieved and maintained as demonstrated by mean central subfield thickness (CST) compared to baseline.
ADVM-022 continues to demonstrate a favorable safety profile and be well tolerated with no drug-related or procedure-related serious adverse events (SAEs), no drug-related systemic adverse events, and no adverse events meeting the criteria for dose-limiting toxicities (DLTs).
ADVM-022-related adverse events (AEs) have been mild (71%) to moderate (29%).
Low-grade ocular inflammation was commonly reported and was responsive to steroid eye drops.