Novartis revealed strong clinical data for its investigational chimeric antigen receptor (CAR) therapy CTL019 as treatment for relapsed/refractory acute lymphoblastic leukemia (r/r ALL).
CAR-T therapy reprograms the patient’s cells to hunt down cancer cells carrying the protein CD19. The reprogrammed T cell therapy, called CTL019, is re-introduced into the bloodstream to bind to and destroy the targeted cancer cells. Novartis has partnered with the University of Pennsylvania's Perelman School of Medicine (Penn) to develop CTL019, which has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) in July. The partners published results of the drug’s initial study in The New England Journal of Medicine (NEJM).
The company reported that the results of the long-term study show 92 percent, or 36 out of 39 patients with r/rALL, experienced complete remission of their disease following treatment with CTL019. In addition, remissions were sustained for up to one year or more with a six-month event-free survival of 70 percent and overall survival of 75 percent. The study, which involved children and young adult patients, showed that most of the cases needed no further therapy. All patients who responded developed cytokine release syndrome (CRS) at peak T cell expansion, which was managed with an IL-6 receptor antagonist and other required treatments.
“We're seeing pediatric patients who have not responded to any other therapy achieve complete remission as a result of treatment with CTL019. However, this is only the first step. Now that these patients have been followed for a longer period of time, we're seeing that a number of them remain in remission for one year or more. This leads me to believe the persistence and durability of CAR-modified cells may help protect against relapse,” said lead investigator Dr. Stephan Grupp, the Yetta Deitch Novotny Professor of Pediatrics at Penn.
Usman Azam, Global Head of Cell & Gene Therapies Unit at Novartis Pharmaceuticals, said, “When we see the response patients have to CTL019 when they have few options left, it's incredibly inspiring. Novartis will leverage our facility in Morris Plains… and the multi-center study for CTL019 in collaboration with the University of Pennsylvania, to broaden the reach of this therapy to additional patients in the clinical setting.”
Novartis will present the results at the upcoming 56th American Society of Hematology (ASH) annual meeting in San Francisco. The company said it will also include data on CTL019 as treatment for B cell cancers, chronic lymphocytic leukemia (CLL) and B cell non-Hodgkin lymphoma (NHL).