Four of six patients with neovascular age-related macular degeneration treated with a threefold lower dose of ADVM-022 in the OPTIC phase 1 trial did not require a rescue injection 24 weeks after treatment and demonstrated an efficacy signal with evidence of dose response.
David S. Boyer, MD, presented the first data from cohort two of the OPTIC trial at Bascom Palmer Eye Institute’s Angiogenesis, Exudation, and Degeneration 2020 symposium in Miami. The multicenter, open-label, dose-ranging trial is assessing the safety and tolerability of a single intravitreal injection of ADVM-022 (Adverum Biotechnologies), a gene therapy candidate that uses a propriety vector capsid to deliver sustained therapeutic levels of aflibercept in patients with wet AMD.
The first cohort of six participants was treated with a higher dose of ADVM-022 (6 × 1011 vg/eye) compared with six participants in the second cohort treated with a threefold lower dose (2 × 1011 vg/eye).
At 24 weeks’ follow-up, four of six participants in cohort two remained rescue injection-free compared with all six participants in cohort one who remained rescue injection-free at a median follow-up of 50 weeks.
In 10 of 12 patients who were rescue injection-free, the mean best corrected visual acuity was maintained, and the mean central subfield thickness improved.
Cohorts three and four will receive a 6-week prophylactic steroid eye drop regimen instead of the oral steroids used in the first two cohorts.
Boyer DS. Phase 1 study of intravitreal gene therapy with ADVM-022 for neovascular AMD (OPTIC Trial). Presented at: Angiogenesis, Exudation, and Degeneration 2020; Feb. 8, 2020; Miami.
Disclosure: Boyer reports he is a consultant with Acuela, Alimera Sciences, Allegro, Bayer, EyePoint, ONL Therapeutics, Oxurion and Takeda; is a consultant and researcher with Adverum, Aerpio, Apellis, Chengdu Kanghong, Clearside Biomedical, Genentech/Roche, Kodiak, Novartis, Recens Medical, Regeneron, Regenxbio, Roche and Santen; and is a researcher with Aerie Pharmaceuticals, Gemini Pharmaceuticals, Neurotech, Opthea and Outlook Therapeutics.