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Adverum Shares Sink After Phase 1 Readout From Wet AMD Gene Therapy Trial

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Adverum Shares Sink After Phase 1 Readout From Wet AMD Gene Therapy Trial

Adverum Biotechnologies Inc (NASDAQ: ADVM) shares were plunging Thursday following the presentation of data from its gene therapy trial at a conference.

What Happened

Adverum, a gene therapy company focusing on ocular and rare diseases, announced 24-week, Phase 1 clinical data from the first cohort of patients treated with a one-time intravitreal dose of ADVM-022 for wet age-related macular degeneration, or wet AMD.

The actively traded, small-cap biotech said the patients achieved vision maintenance and improvements in retinal anatomy after a one-time intravitreal dose of ADVM-022 through week 24. More importantly, these patients did not require anti-VEGF rescue injections.

The mean change in best corrected visual acuity, or BCVA, was -2 letters and the mean change central subfield thickness, or CST, was -52.7 micrometers, the company said.

No serious adverse events were reported, with no adverse events meeting criteria for dose-limiting toxicity.

Yet 10 ocular adverse events were observed in six patients, with 14 showing mild adverse events and five experiencing moderate adverse events.

Adverum presented the data at the Retina Society 2019 Annual Meeting in London. 

Why It's Important

Wet AMD patients are burdened with frequent anti-VEGF injections to maintain vision, with undertreatment invariably resulting in vision loss.

"These data on ADVM-022 are compelling, as they demonstrate for the first time that a one-time gene therapy delivered by intravitreal injection has the potential to provide sustained efficacy and transform the treatment paradigm for patients with wet AMD," said Szilárd Kiss, a retinal specialist who presented the data at the Retina Society meeting.

Adverum shares were falling by 25.56% to $8.93 at the time of publication Thursday. 

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