Trial’s controlled design was expected to quantify the original contribution of stem cells in the IMP. Credit: Petr Novák/Wikipedia.

Adverum Biotechnologies has advanced its ongoing Phase I OPTIC clinical trial of gene therapy candidate ADVM-022 for wet age-related macular degeneration (wet AMD) to the second cohort.

In the first cohort, patients were given a 6 x 10^11 vg/eye dose of the therapeutic, and the second cohort will involve a dose of 2 x 10^11 vg/eye based on the preliminary anatomical response seen in the first cohort subjects.

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The first patient in the second cohort has been dosed with a single intravitreal injection of ADVM-022.

ADVM-022 uses the company’s AAV.7m8 vector capsid to carry an aflibercept coding sequence. It is intended to deliver sustained therapeutic levels of aflibercept and reduce the frequency of anti-vascular endothelial growth factor (VEGF) injections.

In September last year, the therapeutic secured US Food and Drug Administration (FDA) fast track designation in wet AMD, a progressive retinal disease.

The multi-centre, open-label, dose-escalation Phase I trial is evaluating the safety and tolerability of a single intravitreal injection of ADVM-022 in patients responsive to anti-VEGF therapy.

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The primary endpoint of the study is safety and tolerability at 24 weeks after administration.

Secondary endpoints include changes in best-corrected visual acuity (BCVA), central retinal thickness (CRT) measurement, mean number of rescue anti-VEGF injections, and proportion of subjects requiring rescue anti-VEGF injections.

All participants in the OPTIC trial will undergo follow-up for a total of two years.

Adverum Biotechnologies chief medical officer Aaron Osborne said: “In the first cohort of patients, we have seen a robust preliminary anatomical response and no serious adverse events to date.

“We look forward to completing recruitment of the second cohort in OPTIC, and to presenting the 24 week primary and secondary outcomes from the first cohort of patients at a scientific meeting in the second half of 2019.”

The company expects to perform the trial at eight sites across the US.