- Swiss pharma Novartis plans to file for U.S. approval of its experimental CAR-T therapy in early 2017, keeping the pressure on the rest of the closely watched field even after an organizational shake-up earlier this year.
- Treatment with the therapy, known as CTL019, led to complete remission in 82% of pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia in a Phase 2 study.
- Interim results from the trial were presented over the weekend at the annual meeting of the American Society of Hematology, which also featured updates from some of Novartis' key competitors in CAR-T, such as Kite Pharma.
Novartis might steal Kite's thunder yet. Attention had shifted in recent months to the Santa Monica-based biotech as it forged ahead with its filing plans for its lead CAR-T, known as KTE-C19.
Biotech rival Juno Therapeutics has stumbled badly since the summer, recently reporting two more patient deaths in a key study which had been halted due to earlier deaths. And Novartis had seemingly de-emphasized its CAR-T pipeline when it decided to fold its dedicated cell therapy unit back into the broader company organization.
Yet Novartis could still beat Kite to the first approval of a CAR-T therapy, presenting safety and efficacy data for CTL019 which set up a filing with the Food and Drug Administration early next year.
Forty-one of 50 infused patients experienced either complete remission or complete remission with incomplete blood count recovery at three months post-treatment.
Notably, Novartis estimated 60% of responding patients were relapse-free six months after infusion. Durability of response is a key question for CAR-T treatments, which have shown remarkably high response rates early on only to lose some efficacy over time.
Safety is another important area to watch in CAR-T. On this front, Novartis' therapy did cause grade 3 or 4 cytokine release syndrome among nearly half of treated patients. And 15% of patients experienced grade 3 neurological and psychiatric events.
But no patient deaths were reported, nor were any grade 4 neurotoxicities, apparently giving Novartis enough confidence in CTL019's approvability.
Novartis also plans to file for approval of CTL019 with the European Medicines Agency later in 2017.