Eye On the Cure Research News

Jan 10, 2020

AGTC Reports Positive Six-Month Results for XLRP Phase 1/2 Gene Therapy Trial

The company is planning a Phase 3, pivotal trial for end of 2020

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Applied Genetic Technologies Corporation (AGTC), a biotech company developing gene therapies for retinal diseases and other conditions, has  reported a favorable safety profile and continued evidence of efficacy for participants in its Phase 1/2 gene therapy clinical trial for X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.

Twenty-five patients have received the treatment, including those in two new higher dose groups. Data from 17 of those patients was reported in September 2019. Of the 17, nine were treated centrally and eight were treated peripherally.

The nine patients treated centrally showed stable or improved visual acuity (as measured by an eye chart) through six months. Measurable improvements were observed in visual sensitivity (as measured by microperimetry) for four of the eight evaluable patients through six months.

The eight patients treated peripherally showed stable visual function through six months.

The company is enrolling additional Phase 1/2 patients in the first quarter of 2020 and plans to launch a Phase 3, pivotal trial by the end of 2020.

AGTC also has Phase 1/2 gene therapy clinical trials underway for achromatopsia (CNGA3 and CNGB3 mutations). The company has gene therapy preclinical programs for dry age-related macular degeneration and Stargardt disease. It also has a preclinical program for an optogenetic therapy.

XLRP affects approximately 20,000 people in the US and EU. The condition causes constriction of vision, reduced vision in dark settings, and central vision loss, especially in later stages. Most people with XLRP are legally blind by the age of 45.

The Foundation Fighting Blindness funded successful canine studies of XLRP gene therapy at the University of Pennsylvania School of Veterinary Medicine that helped make AGTC’s XLRP gene therapy clinical trial possible.

AGTC used the Foundation’s My Retina Tracker patient registry and genetic testing study to identify and recruit patients for the XLRP and achromatopsia studies.