Novartis will discontinue the development of a one-time gene therapy, picked up last year from Gyroscope Holdings, in geographic atrophy secondary to dry age-related macular degeneration.
The shuttering of the program was reported by London-based health care investment firm Syncona, who said the decision was based on a recommendation from the Independent Data Monitoring Committee following an overall benefit risk assessment of available data which concluded that data from the lead phase 2 HORIZON study did not support continuation of the program.
Novartis bought Gyroscope from Syncona back in February 2022 for an upfront cash payment of $800 million plus the promise of up to another $700 million in milestones. The deal included Gyroscope’s lead asset, GT005, an investigational one-time AAV-based gene therapy that is delivered under the retina and is intended to slow the progression of GA that can lead to blindness.
The drug, which had been fast tracked by the FDA in September 2020, was designed for the treatment of people with GA who have specific mutations in their Complement Factor I gene and low levels of the CFI protein in their blood.
GA is a progressive, irreversible outcome of advanced age-related macular degeneration, where lesions develop, harming vital retinal cells for vision. Apellis Pharmaceuticals' Syfvore, FDA-approved in May, stands as the lone remedy. The drug, however, must be administered once every 25-60 days. The company has recently run into issues with its injection kits and rare events of retinal vasculitis reported in real-world treatment.
