Strong new data for Novartis' investigational leukemia drug CTL019

Swiss pharma giant Novartis (NOVN: VX) and the US University of Pennsylvania's Perelman School of Medicine (Penn) has said that positive preliminary results from two pilot clinical trials were published in The New England Journal of Medicine (NEJM) evaluating the efficacy and safety of CTL019 in patients with relapsed/refractory acute lymphoblastic leukemia (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 [The Pharma Letter July 8], 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, at Novartis Pharmaceuticals, adding: "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."