Gyroscope Therapeutics announces first patient dosed in Phase II HORIZON Trial evaluating investigational gene therapy, GT005, in people with dry age-related macular degeneration

  • U.S. FDA grants second fast track designation to GT005 for Treatment of Geographic Atrophy

Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced the initiation of the Phase II HORIZON trial evaluating GT005 in people with geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). The first patient to receive GT005 in HORIZON was enrolled and dosed in the United States by Jeffrey Heier, M.D., at Ophthalmic Consultants of Boston. HORIZON is a randomised controlled Phase II trial evaluating the safety and effectiveness of GT005 in a broad population of people with GA secondary to dry AMD. GT005 is an investigational one-time AAV-based gene therapy that is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I (CFI) protein, and is intended to slow the progression of GA that can lead to blindness.

Gyroscope also announced that the U.S. Food and Drug Administration (FDA) has granted a second Fast Track designation to GT005. The new Fast Track designation is for the treatment of GA in people with variants in their complement genes. Approximately 95% of people with GA carry at least one genetic variant in a complement gene.[i] This patient population is being evaluated in the Phase II HORIZON trial. Gyroscope estimates that nearly 3.5 million people in the United States and EU5 European countries have GA.[ii],[iii]

Nadia Waheed, M.D., MPH, Chief Medical Officer, said

We are excited to get the HORIZON trial underway. We have designed a robust clinical development programme with two randomized controlled Phase II trials evaluating the safety and effectiveness of GT005 in two different populations of people with GA. These include a specific genetically defined subset of people with GA, as well as a broader group of GA patients. Ultimately, these two trials along with our Phase I/II FOCUS trial will enrol approximately 300 patients

 

Nadia continued: “We are also very pleased to receive a second Fast Track designation from the FDA for GT005. We look forward to continuing to work with the FDA as we advance our clinical programme with the goal of bringing a treatment option to the millions of people with GA.”

The FDA Fast Track programme streamlines the review of drugs for serious conditions without FDA-approved treatment options available. Fast Track designation gives applicants access to more frequent communication with the FDA throughout the review process. It also provides the potential to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete.

Read the full press release and learn more about Gyroscope Therapeutics here