Novartis CTL019 CAR T cell therapy demonstrates potential to treat B-cell lymphomas

Novartis is highlighting data from an ongoing Phase II clinical study of CTL019, an investigational chimeric antigen receptor (CAR) T cell therapy, that indicate its potential in the treatment of specific types of hard-to-treat non-Hodgkin lymphoma. Findings from the ongoing study conducted by the University of Pennsylvania's Perelman School of Medicine (Penn) in adults with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) found an overall response rate (ORR) of 100% in patients with FL and 50% in patients with DLBCL. Thirteen of 19 evaluable patients responded to the therapy. Eleven achieved a complete response (CR) and two experienced a partial response (PR) to treatment. These results will be presented in an oral session at the 51^st American Society of Clinical Oncology (ASCO) Annual Meeting on Monday, June 1 (Abstract #8516, June 1, 3:24 p.m. CDT).

"The results from this ongoing study of CTL019 are encouraging, as we now have data through six months showing that patients may have achieved durable overall response rates," said lead investigator Stephen Schuster, M.D., associate professor, division of Hematology/Oncology at the University of Pennsylvania, Abramson Cancer Center. "These data support our ongoing efforts to determine the potential role of CTL019 in improving outcomes for patients with certain types of B-cell lymphomas."

Study results include 19 adult patients (12 with DLBCL and seven with FL) who were evaluable for response. The study found that six patients with a PR to treatment at three months achieved a CR by six months. Two patients with a PR experienced disease progression at 6 and 12 months after treatment. Median patient follow-up is 274 days for the patients with DLBCL and 290 days for the patients with FL. In the study, two patients developed cytokine release syndrome (CRS) of grade 3 or higher at peak T cell expansion.

"These new CTL019 findings are inspiring as we continue clinical research with our collaborators at the University of Pennsylvania, with the goal of altering the course of cancer care and treating areas of critical unmet need," said Usman Azam, Global Head, Cell & Gene Therapies Unit, Novartis Pharmaceuticals. "As Novartis initiates the Phase II multi-center global study of CTL019, this reinforces our commitment to furthering the new frontier of cell and gene therapies."

Additional CTL019 data being presented at ASCO include the preliminary safety and efficacy findings of a Phase I study investigating the use of CTL019 in the treatment of multiple myeloma (Abstract #8517, June 1, 3:36 p.m. CDT)². This study adds to the growing body of data on CARTs and supports the advancement of the expanding pre-clinical and early clinical research pipeline at Novartis.

Novartis has initiated a Phase II multi-center global study of CTL019 in pediatric patients with r/r acute lymphoblastic leukemia (ALL). This study has opened in the United States, with the intention of expanding into other countries as soon as possible. Further, Novartis has begun to process patient cells at its cell processing facility in Morris Plains, N.J., and will utilize the facility in the Phase II multi-center global study. The facility is the first US Food and Drug Administration (FDA)-approved Good Manufacturing Practices quality site for a cell therapy.

Novartis and Penn have an exclusive global collaboration to research, develop and commercialize CAR T cell therapies for the investigational treatment of cancers. In July 2014, the FDA designated CTL019 as a Breakthrough Therapy for the treatment of pediatric and adult patients with r/r ALL under the Penn Investigational New Drug application (IND). Breakthrough Therapy designation is intended to expedite the development and review of drugs that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. Novartis holds the worldwide rights to CARs developed through the collaboration with Penn for all cancer indications, including the lead program, CTL019.