Novartis And Penn Post Positive Data For ALL Drug

Novartis and its partner the University of Pennsylvania's Perelman School of Medicine (Penn) announced positive preliminary results from two pilot clinical trials evaluating CTL019 in patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL). The partners published the data in The New England Journal of Medicine (NEJM).

CTL019 harnesses chimeric antigen receptor (CAR) technology to reprogram the body’s immune T cells to recognize and seek cancer cells that express a specific protein called CD19. The reprogrammed T cells bind to the targeted CD19+ cancer cells and kill them. CTL019 received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) in July this year.

Novartis reported that initial study results show 90 percent of patients with r/r ALL experienced total remission of their disease following treatment with CTL019. The therapy kept patients in remittance for up to two years. Overall survival reached 78 percent. The study was conducted by Penn.

Usman Azam, Global Head of Cell & Gene Therapies Unit at Novartis Pharmaceuticals, said, “These interim results, which supported the recent FDA Breakthrough Therapy designation, reinforce the potential CTL019 has as a life-saving therapy for patients with relapsed/refractory ALL. …With each new CTL019 milestone, we are one step closer to potentially offering these seriously ill patients an additional treatment option.”

Novartis has an ongoing exclusive global collaboration with Penn for the research, development, and marketing of CAR T cell therapies targeting cancer. Noelle Frey, oncologist at Penn’s Abramson’s Cancer Center, co-first author of the study, said, “Our results support that CTL019 can produce long-lasting remissions for certain heavily pre-treated ALL patients without further therapy. For our patients who have already relapsed after stem cell transplants, or don’t have any options for donors, this option has provided new hope.”

The partners will present updated results of the trial at an upcoming medical congress taking place this year.