Novartis Announces CTL019 Data Published in NEJM Demonstrating Efficacy in Certain Patients with ALL

Novartis and the University of Pennsylvania's Perelman School of Medicine (Penn) have announced preliminary results from two pilot clinical trials published in The New England Journal of Medicine (NEJM) evaluating the efficacy and safety of CTL019 in patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL). The studies, conducted by Penn, demonstrated that 27 of 30 pediatric and adult patients, or 90%, experienced complete remissions with the investigational chimeric antigen receptor (CAR) therapy CTL019.

"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," said Usman Azam, Global Head, Cell & Gene Therapies Unit, Novartis Pharmaceuticals. "These studies are another promising development in CTL019's history. With each new CTL019 milestone, we are one step closer to potentially offering these seriously ill patients an additional treatment option."

These data build on earlier research findings and are part of two pilot clinical studies that demonstrated sustained remissions of up to two years in pediatric and adult patients with r/r ALL. Median follow-up was just over six months, with event-free survival of 67% and overall survival of 78%. Probability of 6-month CTL019 persistence was 68% and CTL019-modified T cells were detectable in the blood by flow cytometry for up to 11 months. Sustained remissions were seen in 15 patients and were associated with CAR T cell persistence and B cell aplasia. Updated results have been submitted for presentation at a medical congress taking place later in 2014.

"We are excited by these results, which indicated how effective CTL019 may be in fighting ALL, a leading cause of childhood cancer deaths," said lead investigator Stephan Grupp, the Yetta Deitch Novotny Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania and director of Translational Research in the Center for Childhood Cancer Research at the Children's Hospital of Philadelphia (CHOP), where 25 pediatric patients were treated in the study cohort. "This represents the largest experience to date of CD19-CAR T cells and demonstrates the ability of this approach to achieve sustained complete responses in a patient population with few other treatment options. We are especially hopeful for those patients who remain in remission for 1–2 years without further therapy."