Applied Genetic Slumps Despite Encouraging Data For Gene Therapy To Treat Rare Inherited Visual Disorder

Shares of Applied Genetic Technologies Corp AGTC are tumbling Thursday despite the company reporting encouraging results from an Phase 1/2 study.

The Cambridge, Massachusetts-based company released interim results from the dose-escalation cohorts of its ongoing Phase 1/2 clinical studies that evaluated its AAV-based gene therapy in patients with achromatopsia resulting from mutations in the ACHM CNGB3 or ACHM CNGA3 genes, which showed encouraging signs of biologic activity and a favorable safety profile, with no dose-limiting inflammatory responses observed.

Achromatopsia is a condition characterized by total or partial absence of color vision. It has no current treatment options.

"We are further encouraged by many patients' real-world positive anecdotes," the company said.

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The interim data were from 22 patients, 13 in the ACHM CNGB3 trial and 9 in the ACHM CNGA3 trial, Applied Genetic said.

Applied Genetic said it expects to report data from the additional dose groups, age groups and time points in the second half of 2020.

The company reported earlier this month positive results for its AAV-based gene therapy in X-linked retinitis pigmentosa, which triggered a 123% rally on Jan. 9.

Applied Genetic shares were slipping 24% to $7.48 at time of publication.