Novartis Books Gyroscope for $1.5 Billion in Pursuit of Gene Therapy for Blindness

Novartis is buying itself an early Christmas present. The Swiss drug giant has agreed to pay $800 million upfront and up to $700 million in milestones to buy Gyroscope Therapeutics, a UK-based gene therapy company. The deal gives Novartis control over Gyroscope’s lead pipeline candidate, GT005, a one-time gene therapy for geographic atrophy (GA), a leading cause of blindness and a part of dry age-related macular degeneration (AMD). 

Novartis has been eyeing gene therapies for ocular diseases for some time. In September, the Swiss drug giant acquired Arctos Medical for their treatments for diseases that involve photoreceptor loss. And last October, Novartis snapped up Vedere Bio for their pipeline therapies for inherited retinal dystrophies and GA. 

“With our own pioneering research in ocular gene therapies and our experience gained from bringing Luxturna to inherited retinal dystrophy patients outside of the US, Novartis has a well-established expertise in ocular gene therapies that will position us well to continue developing this promising one-time treatment” said Marie-France Tschudin, President of Novartis, in a statement.

Gyroscope’s Answer for GA

GA is a late-stage form of AMD, caused by an overactive complement system, a part of the immune system. Complement overactivation leads to inflammation at target sites, such as in the eyes. This causes degeneration of the  

Gyroscope’s lead candidate, GT005, is an AAV2 gene therapy intended as a one-time treatment that can be delivered under the retina to slow the progression of GA. GT005 increases the production of complement factor 1 (CFI), an anti-inflammatory protein that cools down the overactive complement system. 

GT005 is undergoing two clinical trials in GA secondary to dry AMD. The first of the studies, called Focus, is a Phase 1/2 trial that evaluates safety and dose response of the drug. The second, called Horizon, is a Phase 2 trial that studies the safety and efficacy of GT005 in a broader group of patients. 

A few months ago, Gyroscope presented updated interim data of the Focus trial at the Retina Society’s 54th Annual Scientific Meeting. The results showed that GT005 treatment led to increased CFI levels by an average of 122% from baseline in 11 of 13 patients. The increases were still seen at the time of the interim analysis (29 weeks and beyond), with one patient showing sustained increase nearly two years after being dosed. 

The drug has obtained Fast Track designation from the FDA for the treatment of GA.

Author

Joy Lin

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