AGTC outlines plans for phase 2/3 X-linked retinitis pigmentosa trial
Applied Genetic Technologies Corporation announced an update on the proposed design of an upcoming phase 2/3 trial for its X-linked retinitis pigmentosa clinical program, according to a press release.
The trial is expected to begin in the first quarter of 2021, with about 60 participants who will be randomly assigned across three arms: a group receiving a low dose of adeno-associated virus-based gene therapy, a group receiving a high dose of the therapy and an untreated control group.
The primary endpoint is based on visual sensitivity, “with a responder defined as having at least 7 decibel improvement in visual sensitivity in at least five loci at month 12, which is intended to represent a clinically meaningful benefit,” the release said. Responder rates in each arm will be compared with rates in the control arm.
AGTC is aiming to present 6-month interim results by the third quarter of 2022.
"We remain on track to initiate a phase 2/3 trial in 1Q 2021, which was designed based on feedback from the FDA earlier this summer and will utilize our highly productive manufacturing methods that also produce material with greater purity, in that the percent of vectors containing the desired gene of interest is nearly 90%, and process residuals are generally below the detectability of current test methods,” Sue Washer, president and CEO of AGTC, told Healio/OSN.
AGTC also announced its plan to include about 12 more patients in an ongoing phase 1/2 XLRP trial, with interim results expected in the fourth quarter of 2021.
The company’s fiscal year ended June 30, with no revenue reported for its fourth quarter and $2.5 million in revenue for the fiscal year, compared with $0.4 million and $41.7 million in the same periods in fiscal year 2019.