Gyroscope Therapeutics Ltd has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GT005 for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). The GT005 treatment is an investigational AAV-based gene therapy aimed to slowing the progression of GA. Clinical researchers will recruit GA patients with specific mutations in their Complement Factor I (CFI) gene and low levels of the CFI protein in their circulation. Enrolment in the Phase II “EXPLORE” study [NCT04437368] is to evaluate approximately 75 participants for five sites in the US.
The Phase II trial will be an interventional assessor-masked multicentre, randomised study to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with GA secondary to AMD. The overall objectives of the study are to evaluate the safety and efficacy (anatomical and functional visual outcomes) of two doses of GT005 in genetically defined subjects with GA due to AMD. The trial study includes a screening period of up to 8 weeks followed by a 48-week study period and subjects will be randomised to two groups; GT005 or the untreated control group. The primary outcome endpoint is the change from baseline to Week 48 in the GA area as measured by fundus fluorescence (FAF). The investigational gene therapy treatment is built on studies and scientific publications which links chronic local inflammation, activation of the complement system and AMD pathogenesis research. In addition, gene therapy is aimed to develop a sustained drug delivery modality within ophthalmic and non-ophthalmic indications which partner with surgical device technologies, also based with Syncona Ltd., a healthcare life sciences investment company.
According to the company’s Chief Medical Officer , Nadia Waheed, M.D., MPH, , “dry AMD is a life-altering diagnosis and there are currently no FDA-approved medicines available. Research suggests people with dry AMD who have certain CFI mutations that correlate with low CFI levels in the blood have a higher risk of developing AMD. We are pleased to receive Fast Track designation for our investigational gene therapy for this high-risk group. We look forward to working with the FDA as we advance our clinical programme evaluating the safety and effectiveness of GT005.”