September 12, 2019
2 min read

No anti-VEGF rescue injections needed in OPTIC phase 1 trial of ADVM-022

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An intravitreal dose of ADVM-022, a gene therapy designed for long-term VEGF suppression via the expression of aflibercept, resulted in no anti-VEGF rescue injections for patients with wet age-related macular degeneration, according to 24-week clinical data in the OPTIC phase 1 clinical trial.

Six patients with wet AMD first received Eylea (aflibercept, Regeneron) to ensure a response, followed by one intravitreal dose of ADVM-022 of 6 × 1011 vg per eye in the multicenter, open-label OPTIC trial.

The primary endpoint of the phase 1 clinical trial was safety and tolerability, with secondary endpoints related to changes in best corrected visual acuity, measurement of central retinal thickness, mean number of anti-VEGF rescue injections and percentage of patients needing anti-VEGF rescue injections. Rescue injections of aflibercept were permitted according to prespecified protocol criteria for vision, OCT or hemorrhage, Szilard Kiss, MD, said in an Adverum Biotechnologies teleconference.

“Today you saw data where there were zero rescue injections in six out of six patients. … If you look at the OCTs, five out of six patients had complete resolution for activity of wet AMD. The sixth patient has a polypoidal, something we all know is extremely difficult to treat, but the polypoidal responded very well,” Kiss told

Kiss presented the 24-week interim clinical data at the Retina Society 2019 annual meeting in London.

At 24 weeks, no patients in the trial required an anti-VEGF rescue injection, and the mean change in BCVA was –2 letters. Patients experienced a mean change of –52.7 µm in central retinal thickness, and no serious adverse events were recorded, according to Kiss.

However, 19 ocular adverse events possibly related to ADVM-022 occurred in the six patients. Fourteen events were mild and five were moderate, including two cases of intermediate uveitis, two cases of anterior chamber cells and one case of vitreous cells, according to an Adverum Biotechnologies press release.

Before receiving ADVM-022, the previously treated patients required a mean number of 35 anti-VEGF injections with an annualized treatment of 9.3 injections per year to maintain their functional vision of 20/50.

The 52-week data from the first cohort of patients, as well as 24-week data from the second cohort of patients, will be presented in the first half of 2020, the release said.

“Gene therapy is a reality, and it’s a reality for rare inherited retinal degeneration. The data we presented today is really pointing to the fact that it may become a reality for much more common non-inherited disorders, where the need for repeated injections is not only a huge burden for the patient, but their caregivers and to society in general and the health care system,” Kiss said.

Disclosure: Kiss reports he is a consultant for Adverum Biotechnologies, Regenxbio and Genentech/Roche.

Editor's Note: This article was updated to include quotes from Szilard Kiss, MD.