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Promising results from ongoing Phase 1/2 clinical trial for X-linked retinitis pigmentosa (XLRP)

October 1, 2019

The Retina Foundation of the Southwest is one of four U.S. centers conducting a Phase 1/2 dose escalation, gene replacement trial for x-linked retinitis pigmentosa (XLRP). On September 26th, the clinical trial sponsor, Applied Genetic Technologies Corporation (AGTC) announced the initial results. Investigators from all four research sites have begun seeing encouraging signs of benefit and biologic activity.

A total of 22 patients are participating in this clinical trial where gene therapy is being administered to the peripheral or central region of their retinas. This report is from the data collected on 17 of those patients – seven have completed their three-month evaluation and ten have completed their six-month evaluation. The preliminary data shows evidence of improvement in visual acuity and sensitivity. Safety data from all 22 patients shows no dose-limiting inflammatory responses observed in patients dosed to date and no secondary inflammatory responses requiring re-administration of any steroid.

“The encouraging signs of stable or improved visual function, as measured by visual fields and visual acuity analysis in the XLRP trial, further increase our confidence in the promise of our therapy to make a difference to patients in need,” said Sue Washer, President and CEO of AGTC.

“Maintenance of peripheral vision and improvements in central vision are important and clinically meaningful. Reductions in these capabilities are hallmarks of this degenerative disease and negatively impact patients’ ability to engage in daily living and work activities,” said Dr. David Birch, Scientific Director, Retina Foundation of the Southwest. The biologic activity observed to date in this clinical trial, especially if sustained through the 12-month time point, suggest that sub-retinal gene therapy may be able to stabilize peripheral vision and improve central vision in patients.

The initial results were announced on September 26th by the sponsor, Applied Genetic Technologies Corporation (AGTC).