AGTC Continues to Report Encouraging Data from its Phase 1/2 XLRP Gene Therapy Clinical Trial

Applied Genetic Technologies Corporation (AGTC), a developer of gene therapies for inherited retinal diseases, has reported additional, positive data from its ongoing Phase 1/2 clinical trial of a gene therapy for X-linked retinitis pigmentosa (XLRP) patients with mutations in the gene RPGR.

AGTC also reported that it is currently enrolling for an expanded Phase 2 XLRP gene therapy clinical trial known as Skyline, which is taking place at eight sites in the US. In addition, the company is planning to launch a Phase 2/3 XLRP gene therapy clinical trial later this year. Known as Vista, the Phase 2/3 trial will take place at sites in the US, Israel, and the Netherlands.

For the ongoing Phase 1/2 trial, AGTC reported 12-month data for patients from Groups 5 and 6 who met the inclusion criteria for the Skyline and Vista trials. Four of the eight patients responded to the gene therapy. Patient responses were captured using microperimetry, which measures retinal sensitivity at various points in the central retina. Responders were those who had improvements of at least 7 decibels in retinal sensitivity at five points or more in the central retina.

AGTC added that best corrected visual acuity data continue to provide evidence of biological response at 12 months.

Data from a subset of Group 4 patients available for analysis at 24 months also provide preliminary evidence of continued durable responses.

“A growing body of data supports the best-in-class potential of our XLRP therapy product candidate, and we are executing a robust plan to advance this program as rapidly as possible toward commercialization,” said Sue Washer, president, and chief executive officer at AGTC.

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 program to identify and recruit patients for its XLRP studies.