Kite Pharma’s gene therapy trial has just delivered astonishing results: 41% of patients in response and 36% in complete response at month 6 after a single round of treatment. The news shook the bio and med industry last week, skyrocketing Kite Pharma’s shares by 25% on the day of the announcement according to Fortune. The company is committed to providing rapid, long-term durable response through innovative cancer immunotherapy, and has certainly delivered with their first re-engineered chimeric antigen receptor and t-cell receptor therapy, capable of boosting the patient’s immune system’s ability to recognize and kill tumors.
The patients that participated in the trial presented one of three types of non-Hodgkin lymphoma (NHL), a group of cancers that develop from the host’s lymphocytes that’s traditionally treated through chemotherapy. Radiation, immunotherapy and targeted therapy, according to the spread speed and areas affected. All patients in Kite Pharma’s trial were at advanced stages of their conditions, and were given only a few months to live prior to the trial’s start. And yet, following the first round of gene therapy (that took place nine months after the trial began) half the patients are still alive, and a third of them even present complete remission.
The treatment itself is based on reprogramming the patient’s T cells, redirecting them to kill cancer cells. This candidate therapy, known as axicabtagene ciloleucel, engineers the T cells to express a chimeric antigen receptor (CAR) that targets antigen CD19, a protein expressed on the surface of B-cell lymphomas and leukemias. According to Kite Pharma’s press release on the subject, the therapy has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.
Kite Pharma is developing engineered autologous T cells that express either a chimeric antigen receptor (CAR) or a T cell receptor (TCR), depending on the type of cancer. Their dual platform has the potential to address both hematological and solid tumor cancers. Source: http://kitepharma.com/technology/
“These results with axicabtagene ciloleucel are exceptional and suggest that more than a third of patients with refractory aggressive NHL could potentially be cured after a single infusion of axicabtagene ciloleucel,” states Jeff Wiezorek, M.D., Senior Vice President of Clinical Development in the release. “The study was built on a foundation of support and commitment from Dr. Steven Rosenberg and the National Cancer Institute and our clinical trial investigators who believed in the potential for CAR-T therapy to change the paradigm of cancer treatment.”
“We know as clinicians that patients with aggressive lymphoma who do not respond to their previous treatments have a very poor prognosis. In fact, we know from [previous studies], these patients have only an eight percent chance of achieving a complete response with current therapies,” said Frederick L. Locke, M.D., Co-Lead Investigator for the trial, and Director of Research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida, through the press release. “Several patients we treated at Moffitt Cancer Center experienced a rapid and durable complete response with this first-of-its kind therapy.”
Kite will present the full results of the trial in April, and is currently seeking approval both from European regulatory boards and in the U.S. If the latter works out, the company would win the race against Novartis AG and become the country’s first approved gene therapy. The company has stated they will complete their rolling submission of the Biologics License Application (BLA) by the end of the first quarter of 2017 based on the combined data from all 101 patients of their study. The company also plans to submit a marketing authorization application (MAA) for their therapy for the treatment of relapsed or refractory DLBCL, PMBCL and TFL with the European Medicines Agency during this year.