07-08-2012: Novartis and the University of Pennsylvania (Penn) announced an exclusive global collaboration to research, develop and commercialize targeted chimeric antigen receptor (CAR) immunotherapies for the treatment of cancers. In addition, the parties will jointly establish a new research and development facility on the Penn campus, called the Center for Advanced Cellular Therapies (CACT).
In CAR immunotherapy, immune cells (T cells) are drawn from a patient's blood. Then, using CAR technology, the T cells are re-coded to identify and seek out cells that express proteins present on a patient's cancerous tumor. When the T cells are re-introduced into the patient's blood, they bind to the targeted cancer cells and destroy them.
As part of the transaction, Novartis acquired exclusive rights from Penn to CART-19, a novel investigational CAR therapy, currently being studied by Penn in a pilot clinical trial. CART-19 targets a protein called CD19 that is associated with a number of B-cell malignancies such as chronic lymphocytic leukemia (CLL), B-cell acute lymphocytic leukemia and diffuse large B-cell lymphoma.
To accelerate the discovery and development of additional therapies using CAR immunotherapy, Novartis and Penn will build the Center for Advanced Cellular Therapies on the Penn campus in Philadelphia. This will be a first-of-its-kind research and development center established specifically to develop and manufacture adoptive T-cell immunotherapies under the research collaboration guided by scientists and clinicians from Novartis and Penn.
Early results from a clinical trial of CART-19, conducted by Penn, showed potent antileukemic effects in three patients with advanced CLL who had previously undergone multiple courses of chemotherapy and biological therapy. Two of the patients were still in complete remission more than a year into the CART-19 trial, and the third patient maintained partial remission for more than seven months. An immune deficiency known as hypogammaglobulinemia, an expected chronic toxic effect, was corrected with infusions of intravenous immune globulin. Patients were also treated for symptoms associated with tumor lysis syndrome, an effect of tumor breakdown. Novartis expects to initiate a Phase II clinical trial with CART-19 in collaboration with Penn during the fourth quarter of 2012.
Under the terms of the agreement, Penn grants Novartis an exclusive worldwide license to CARs developed through the collaboration for all indications and CART-19. In addition Novartis will provide an up-front payment, research funding, funding for the establishment of the CACT and milestone payments for the achievement of certain clinical, regulatory and commercial milestones and royalty payments.