June 2021
Volume 62, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2021
Phase 1 Study of Intravitreal Gene Therapy with ADVM-022 for neovascular AMD (OPTIC Trial)
Author Affiliations & Notes
  • Brandon Busbee
    Tennessee Retina, Tennessee, United States
  • David S Boyer
    Retina-Vitreous Associates Medical Group, Los Angeles, California, United States
  • Arshad M. Khanani
    Sierra Eye Associates, Nevada, United States
  • Charles Clifton Wykoff
    Retina Consultants of Texas, Houston, Texas, United States
  • Dante Joseph Pieramici
    California Retina Consultants, California, United States
  • Carl Regillo
    Wills Eye Hospital, Philadelphia, Pennsylvania, United States
  • Carl J Danzig
    Rand Eye Institute, Florida, United States
  • Brian C Joondeph
    Colorado Retina Associates, Colorado, United States
  • James Major
    Retina Consultants of Texas, Houston, Texas, United States
  • Carol Hoang
    Adverum Biotechnologies Inc, Redwood City, California, United States
  • Adam Turpcu
    Adverum Biotechnologies Inc, Redwood City, California, United States
  • Carol Chung
    Adverum Biotechnologies Inc, Redwood City, California, United States
  • Szilard Kiss
    Weill Cornell Medicine, New York, New York, United States
  • Mehdi Gasmi
    Adverum Biotechnologies Inc, Redwood City, California, United States
  • Aaron Osborne
    Adverum Biotechnologies Inc, Redwood City, California, United States
  • Footnotes
    Commercial Relationships   Brandon Busbee, Adverum Biotechnologies (F); David Boyer, Adverum Biotechnologies (C), Adverum Biotechnologies (F); Arshad Khanani, Adverum Biotechnologies (C), Adverum Biotechnologies (F); Charles Wykoff, Adverum Biotechnologies (C), Adverum Biotechnologies (F); Dante Pieramici, Adverum Biotechnologies (F); Carl Regillo, Adverum Biotechnologies (C), Adverum Biotechnologies (F); Carl Danzig, Adverum Biotechnologies (F); Brian Joondeph, Adverum Biotechnologies (F); James Major, Adverum Biotechnologies (F); Carol Hoang, Adverum Biotechnologies (E); Adam Turpcu, Adverum Biotechnologies (E); Carol Chung, Adverum Biotechnologies (E); Szilard Kiss, Adverum Biotechnologies (F), Adverum Biotechnologies (C); Mehdi Gasmi, Adverum Biotechnologies (C); Aaron Osborne, Adverum Biotechnologies (E)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science June 2021, Vol.62, 352. doi:
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      Brandon Busbee, David S Boyer, Arshad M. Khanani, Charles Clifton Wykoff, Dante Joseph Pieramici, Carl Regillo, Carl J Danzig, Brian C Joondeph, James Major, Carol Hoang, Adam Turpcu, Carol Chung, Szilard Kiss, Mehdi Gasmi, Aaron Osborne; Phase 1 Study of Intravitreal Gene Therapy with ADVM-022 for neovascular AMD (OPTIC Trial). Invest. Ophthalmol. Vis. Sci. 2021;62(8):352.

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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 treatment burden and improve vision outcomes in patients with neovascular AMD (nAMD). OPTIC is a phase 1 study designed to assess the safety, tolerability and efficacy of a single intravitreal injection of ADVM-022 (AAV.7m8-aflibercept gene therapy) in patients with nAMD.

Methods : Open-label, multicenter, dose-ranging study in treatment-experienced patients with a previous confirmed response to anti-VEGF therapy. Patients were administered a single intravitreal injection of ADVM-022 at 6x10^11 vg/eye (Cohort 1: n=6, Cohort 4: n=9) or at 2x10^11 vg/eye (Cohort 2: n=6, Cohort 3: n=9). Incidence and severity of adverse events, change in visual acuity (BCVA), change in central retinal thickness (CST), need for and number of supplemental aflibercept injections were evaluated.

Results : As of October 15, 2020, median follow-up was 86 weeks (Cohort 1), 64 weeks (Cohort 2), 48 weeks (Cohort 3) and 16 weeks (Cohort 4). Patients in all 4 cohorts previously received frequent anti-VEGF injections (mean 7.1–9.2 injections in the prior 12 months) to maintain relatively good baseline BCVA (mean 65.0–65.9 ETDRS letters) prior to receiving ADVM-022. ADVM-022 continues to be well tolerated with a favorable safety profile. All ADVM-022-related ocular adverse events were mild (78%) to moderate (22%). Ocular inflammation predominantly affecting the anterior segment, when observed, has been responsive to steroid eye drops. No cases of retinal involvement or vasculitis were reported. A significant reduction in anti-VEGF injection burden was observed with both doses; 14/15 patients receiving high dose and 10/15 patients receiving low dose remained supplemental anti-VEGF injection free while mean annualized anti-VEGF injection frequency was reduced by 99% (high dose) and 85% (low dose) after ADVM-022. For Cohorts 1–3, data pending for cohort 4, BCVA was maintained with a mean change of -2.5 to +0.2 ETDRS letters, and CST improved with a mean change of -19.7 to -132.7 µm.

Conclusions : ADVM-022 is designed to provide continuous expression of aflibercept following a single intravitreal injection. Over 80% of patients with nAMD treated with a single injection of ADVM-022 in OPTIC have not needed any supplemental anti-VEGF injections up to 92 weeks follow-up. ADVM-022 has the potential to reduce treatment burden and improve patient vision outcomes.

This is a 2021 ARVO Annual Meeting abstract.

 

 

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