Gene therapy fails to show clinical activity in X-linked retinoschisis

An investigational AAV-based gene therapy from Applied Genetic Technologies Corporation for X-linked retinoschisis due to mutations in the RS1 gene showed no signs of clinical activity at 6 months in a phase 1/2 trial, the company announced.

The rAAV2tYF-CB-hRS1 intravitreal injection was shown to be safe and well-tolerated in the dose-escalation study, but due to the lack of clinical activity, the company has decided not to move forward with further trials, Sue Washer, CEO of AGTC, said in a conference call regarding the study results.

“We will continue to analyze all of the XLRS data and complete all visits in the clinical protocol, but at this time we don’t plan to further develop the product,” she said. “We remain confident in the potential of our other ongoing clinical trials in X-linked retinitis pigmentosa, or XLRP, and achromatopsia to demonstrate clinical benefit.”

In addition, Biogen has terminated a collaboration agreement between the two companies as of March 8, 2019.

“Upon termination we will regain commercial rights to the XLRP clinical program, as well as three preclinical programs,” Washer said. “We believe regaining 100% control of these programs after investment from Biogen will create value to AGTC patients and shareholders as we move our programs forward with an even greater focus on gene therapy in ophthalmology.” - by Rebecca L. Forand

An investigational AAV-based gene therapy from Applied Genetic Technologies Corporation for X-linked retinoschisis due to mutations in the RS1 gene showed no signs of clinical activity at 6 months in a phase 1/2 trial, the company announced.

The rAAV2tYF-CB-hRS1 intravitreal injection was shown to be safe and well-tolerated in the dose-escalation study, but due to the lack of clinical activity, the company has decided not to move forward with further trials, Sue Washer, CEO of AGTC, said in a conference call regarding the study results.

“We will continue to analyze all of the XLRS data and complete all visits in the clinical protocol, but at this time we don’t plan to further develop the product,” she said. “We remain confident in the potential of our other ongoing clinical trials in X-linked retinitis pigmentosa, or XLRP, and achromatopsia to demonstrate clinical benefit.”

In addition, Biogen has terminated a collaboration agreement between the two companies as of March 8, 2019.

“Upon termination we will regain commercial rights to the XLRP clinical program, as well as three preclinical programs,” Washer said. “We believe regaining 100% control of these programs after investment from Biogen will create value to AGTC patients and shareholders as we move our programs forward with an even greater focus on gene therapy in ophthalmology.” - by Rebecca L. Forand