Gyroscope Therapeutics Limited has announced the launch of its Phase II programme to evaluate GT005, an investigational gene therapy for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). Gyroscope plans to conduct two Phase II trials evaluating GT005 and determine its potential to slow the progression of GA.
GT005 is a one-time AAV-based gene therapy that is delivered under the retina. It is designed to restore balance to an overactive complement system of the immune system by increasing production of the CFI protein. An overactive complement system has been implicated in the development of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production will dampen the system’s overactivity and reduce inflammation, with the goal of preserving a person’s eyesight.
The first of two trials, called EXPLORE (NCT04437368), is enrolling people who have a mutation in their Complement Factor I (CFI) gene. The first patient to receive GT005 in EXPLORE was enrolled and dosed by Dr. Arshad M. Khanani at Sierra Eye Associates in Reno, Nev., USA.
“Geographic atrophy is a devastating diagnosis, as there are no approved treatments for this gradual and irreversible loss of vision,” said Arshad M. Khanani, M.D., M.A., Director of Clinical Research at Sierra Eye Associates and Clinical Associate Professor at the University of Nevada, Reno School of Medicine, and an investigator in the EXPLORE trial. “We are excited to participate in this trial evaluating GT005 for the potential to slow progression of geographic atrophy. We believe one-time gene therapies could be a major advancement in the field of retinal disease.”
Research has found that approximately 3% of people with dry AMD have certain CFI mutations that correlate with low CFI levels in the blood and a higher risk of developing AMD. Gyroscope estimates that more than 100,000 people with GA in the United States and EU5 European countries may have these mutations. The EXPLORE trial will evaluate GT005 in this group of people with mutations in their CFI gene.
Research has also shown that a small supplementation of CFI can normalise complement activity in the blood, suggesting GT005 may also be applicable for a broader group of people with GA. It is estimated that approximately one million people in the United States alone have GA. Gyroscope therefore plans to initiate a second Phase II trial in 2020 that will evaluate GT005 in a broader GA population.
“We are excited about the potential of GT005 for people with dry AMD. Research suggests GT005 may be best suited for people with certain mutations in their CFI gene. However, evidence also suggests it may have potential for a broader population of people with geographic atrophy,” said Nadia Waheed, M.D., MPH, Chief Medical Officer of Gyroscope. “We have designed our clinical programme to evaluate these groups in two distinct Phase II trials, with the goal of determining which patients GT005 may be most appropriate for and to further our understanding of the role of the complement system in AMD.”
Full news release: https://www.businesswire.com/news/home/20200812005751/en/Gyroscope-Therapeutics-Announces-Initiation-Phase-II-Programme
Source: Gyroscope Therapeutics