- A third of patients with an aggressive form of non-Hodgkin lymphoma remained in remission three months after receiving Kite Pharma's experimental CAR-T candidate, positioning the cell therapy company to file for approval with U.S. regulators in the coming months.
- Kite's drug, known as KTE-C19, met the primary endpoint of objective response rate in an interim analysis of the Phase 2 ZUMA-1 study, the company said late Monday. Results were collected from the first 51 patients with chemorefractory diffuse large B-cell lymphoma (DLBCL) who had at least 3 months of follow-up.
- Shares in Kite jumped by more than 8% Tuesday on news of the positive results. Some questions remained about the durability of responses, however. Kite plans to release primary analysis data early next year which will include six months of follow-up.
With positive interim results in hand, Kite retains its lead in the race to file for approval of a CAR-T therapy with the Food and Drug Administration. A tragic setback for Juno Therapeutics' lead candidate and Novartis recent decision to fold its dedicated CAR-T unit back into the larger company organization appear to have bolstered Kite's position in the still developing field.
Kite has previously said it plans to file for approval of KTE-C19 by the end of the year. On an investor call Monday evening, company executives were more cautious in discussing timing of a biologics license application, indicating Kite would finalize its submission plans after meeting with the FDA to discuss the interim results.
Juno, by comparison, anticipates submitting its leading JCAR-15 treatment for approval sometime in 2017, with a decision by the FDA possible in the first half of 2018. Novartis, which says its internal shake-up will not affect timing for its lead CTL-019 therapy, also plans to file sometime in 2017.
Additional data from this first look at Kite's data will be presented at an upcoming scientific meeting, the company said. A primary analysis including six months of follow-up data is expected in the first quarter of 2017.
Initially, an objective response rate (ORR) was recorded in 76% of the 51 DLBCL patients, with 47% experiencing a complete response (CR). Some patients relapsed in the three months following treatment however, leading to a 39% ORR and 33% CR at that time.
A similar drop-off in response rates was also seen in a Phase 1 portion of the Zuma-1 study, which tested KTE-C19 in 7 patients.
Still, Kite was excited by the data. "We believe these are impressive results given that the ZUMA-1 study enrolled patients with chemorefractory aggressive non-Hodgkin's lymphoma, whose outlook is dismal," said David Chang, chief medical officer at Kite.
Kite also reported data from 11 patients with transformed follicular lymphoma (TFL) and primary mediastinal B-cell lymphoma (PMBCL). After three months, 64% of patients saw a complete response, boosting the combined complete response rate for the 62 patients in the trial to 39%.
As in other CAR-T trials, Kite saw a large number of adverse events among the participating patients. Grade 3 or higher cytokine release syndrome (CRS) — a potentially deadly side effect seen in some CAR-T trials — was reported in 18% of patients, which 34% of patients experienced neurological toxicity.
Two patients died from adverse events related to KTE-C19, one from hemophagocytic lymphohistiocytosis and the other from cardiac arrest in the setting of CRS.
Given the advance nature of the cancers treated, Kite believes the side effect profile of KTE-C19 to be well-managed.