June 2020
Volume 61, Issue 7
Free
ARVO Annual Meeting Abstract  |   June 2020
Phase 1 Study of Intravitreal Gene Therapy ADVM-022 for neovascular AMD (OPTIC Trial)
Author Affiliations & Notes
  • Arshad M. Khanani
    Sierra Eye Associates, Reno, Nevada, United States
    Reno School of Medicine, University of Nevada, Reno, Nevada, United States
  • Szilard Kiss
    Weill Cornell Medical College, New York, New York, United States
  • Adam Turpcu
    Adverum Biotechnologies Inc, Menlo Park, California, United States
  • Carol Hoang
    Adverum Biotechnologies Inc, Menlo Park, California, United States
  • Aaron Osborne
    Adverum Biotechnologies Inc, Menlo Park, California, United States
  • Footnotes
    Commercial Relationships   Arshad Khanani, Adverum Biotechnologies Inc (R), Adverum Biotechnologies Inc (C), Allergan (R), Allergan (C), Allergan (S), Genentech (S), Genentech (R), Genentech (C), Grayburg (R), Grayburg (C), Kodiak (R), Kodiak (C), Novartis (R), Novartis (C), Novartis (S), Opthea (R), Opthea (C), Regenxbio (R), Regenxbio (C); Szilard Kiss, Adverum Biotechnologies Inc (I), Adverum Biotechnologies Inc (C), Fortress Bio (I), Fortress Bio (C), Novartis (C), Optos (R), Optos (C), Regenxbio (I), Regenxbio (C), Weill Cornell University (P); Adam Turpcu, Adverum Biotechnologies Inc (E); Carol Hoang, Adverum Biotechnologies Inc (E); Aaron Osborne, Adverum Biotechnologies Inc (E)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science June 2020, Vol.61, 1154. doi:
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      Arshad M. Khanani, Szilard Kiss, Adam Turpcu, Carol Hoang, Aaron Osborne; Phase 1 Study of Intravitreal Gene Therapy ADVM-022 for neovascular AMD (OPTIC Trial). Invest. Ophthalmol. Vis. Sci. 2020;61(7):1154.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Intravitreal gene therapy has the potential to significantly reduce anti-vascular endothelial growth factor (VEGF) treatment burden and improve real-world vision outcomes in neovascular AMD (nAMD). OPTIC (NCT03748784) assessed the safety, tolerability and efficacy of a single intravitreal injection of ADVM-022 (AAV.7m8-aflibercept gene therapy) in patients with nAMD.

Methods : OPTIC is an ongoing open-label, multicenter, dose-ranging study in treatment experienced patients with a confirmed response to anti-VEGF therapy. Patients were administered a single intravitreal injection of ADVM-022 at 6x10^11 vg/eye for cohort 1 (n=6) and at 2x10^11 vg/eye for cohort 2 (n=6). Incidence and severity of adverse events, change in visual acuity (BCVA), anatomical outcomes on OCT, and number of aflibercept rescue injections were evaluated.

Results : Patients in cohort 1 previously received frequent anti-VEGF injections (mean 6.2 injections in the prior 8 months) to maintain relatively good baseline BCVA (mean 65.8 ETDRS letters) prior to enrollment in OPTIC. Through October 1, 2019, with a median of 34 weeks follow-up (range 24, 44 weeks), there were no serious adverse events, no dose limiting toxicities and no non-ocular adverse events related to ADVM-022. Ocular inflammation occurred mainly in the anterior segment, and was generally mild and manageable with topical steroids. BCVA was maintained with a mean change of -1.5 (range -9,+5) ETDRS letters. On OCT, retinal anatomy improved with signs of exudation resolved or stabilized in all patients. No rescue injections were required for any patient. New data from the ongoing, open-label OPTIC study will be presented, including 52-week data from cohort 1. Baseline characteristics and October 1, 2019 data are in the table below.

Conclusions : ADVM-022 is designed to provide sustained therapeutic levels of aflibercept following a single intravitreal injection. Patients with nAMD treated with a single injection of ADVM-022 were able to maintain vision and improve anatomical outcomes without requiring any rescue anti-VEGF injections through a median of 34 weeks.

This is a 2020 ARVO Annual Meeting abstract.

 

 

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