NEW YORK – Syncona on Friday said it will write off $68.3 million (£54.5 million) in lost milestone payments because of Novartis' decision to stop developing GT005, a gene therapy it acquired from Syncona last year.
Novartis bought Syncona subsidiary Gyroscope Therapeutics in early 2022 in a deal worth $1.5 billion, including an $800 million upfront payment and up to $700 million in additional milestone-based payouts. In the deal, Novartis garnered Gyroscope's lead asset, GT005, which it took over developing as a treatment for geographic atrophy secondary to dry age-related macular degeneration (AMD).
GT005 is an adeno-associated viral vector-based gene therapy that encodes for human complement factor I. CFI regulates the complement system, the part of the immune system that controls the ability of antibodies and phagocytic cells to clear microbes and attack pathogens. Dysregulation of the complement system is associated with geographic atrophy, a form of late-stage AMD that causes vision loss.
In the Phase I/II FOCUS trial, GT005 appeared safe and well tolerated, and 11 out of 13 patients treated with the gene therapy had increased and sustained CFI levels. Based on this, researchers began the Phase II HORIZON trial to compare the disease progression of 250 patients on two different doses of GT005 or on no treatment in a control arm.
According to London-headquartered Syncona, however, Novartis decided to stop this development program after an independent data-monitoring committee conducted an overall risk-benefit analysis and concluded that the data didn't support continuing the HORIZON trial.
As part of the Gyroscope acquisition deal, Syncona received $442 million of the upfront cash proceeds, and was also eligible to receive certain clinical and commercial milestone payments that it valued at around $68.3 million, which it said it will now write off.
"We are naturally disappointed for patients following the decision to discontinue GT005, but we respect Novartis' decision," Syncona CEO Chris Hollowood said in a statement. "We remain focused on our strategy of building and maintaining our portfolio of companies providing significant opportunities to deliver strong risk-adjusted returns to our shareholders and to make a difference to the lives of patients with devastating diseases."