Kite Pharma Claims Success for Phase II CAR-T Study, Stock Soars

September 27, 2016
By Alex Keown, BioSpace.com Breaking News Staff

SANTA MONICA, Calif. – Shares of Kite Pharma are up nearly 12 percent in pre-market trading after the company’s CAR-T therapy KTE-C19 demonstrated a 39 percent response rate, including a complete response rate of one-third of patients with various forms of Non-Hodgkin’s Lymphoma after three months of treatment, the company announced Monday afternoon.

Kite believes the interim data of the Phase II trial will be strong enough to lead regulators at the U.S. Food and Drug Administration (FDA) to ultimately give its approval—making KTE-C19 the first CAR-T therapy to reach the market ahead of rivals Juno Therapeutics and Novartis . Kite said it plans to seek regulatory approval of KTE-C19 in diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL) and primary mediastinal B-cell lymphoma (PMBCL) based upon the combined data of multiple trial cohorts. Kite anticipates commercial launch of KTE-C19 in 2017. As a result, shares of Kite’s stock continue to rise, currently trading at $60.03 this morning.

But, before the FDA gives its nod, the company still has to overcome a few hurdles, including concerns about the small trial size, 62 patients, as well as two deaths associated with the experimental drug that occurred during the trial. There is also the lack of a placebo arm in the mid-stage trial that could raise an issue. Stat News reported that Kite pointed to historical data from other trials, particularly one in DLBCL. Kite noted that 26 percent of the 635 patients saw a reduction in their cancer, while 8 percent of patients showed a complete response.

Another question that may need to be clearly addressed is the sliding response rate of patients on KTE-C19. Initial data results, prior to the three-month follow-up, show that 76 percent of patients responded to the treatment, including a complete response from 47 percent. Seven patients who initially showed a complete response to the treatment relapsed, the company said.

Canaccord Genuity healthcare analyst John Newman said the FDA has full access to Kite’s data, far more than what is typically disclosed to Wall Street investors. In an email he said Kite should have “significant flexibility to update results at more than one time” before the end of the year. He said any updates the company could provide will help in dealing with the FDA.

"Also, we believe breakthrough status could enable a more seamless data update during the actual review, if desired by FDA, without resulting in any delay,” Newman said in an email to BioSpace.

The Phase II interim outcomes in ZUMA-1 are largely consistent with results from the Phase I portion of the study and the National Cancer Institute study based on the same CAR construct, a low-dose cyclophosphamide-fludaribine conditioning regimen, and Kite’s proprietary manufacturing process, the company said.

Chimeric Antigen Receptor T-Cell Therapies (CAR-T) are engineered in a laboratory to recognize a specific antigen in a cell and then administered into a cancer patient. The CAR-T cells should, if all goes as planned, multiply within the body and target the antigen and eliminate the threat. KTE-C19 is an investigational therapy in which a patient's T-cells are engineered to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T-cells to kill cancer cells. KTE-C19 has been granted Breakthrough Therapy Designation status for DLBCL, TFL, and PMBCL by the U.S. Food and Drug Administration and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

“Zuma-1 enrolled patients with chemorefractory aggressive NHL, a disease that is very difficult to treat. The combined CR rate of 39 percent at three months is very exciting as it represents nearly a five-fold increase from the CR rate of 8 percent seen in the Scholar-1 study in a similar patient population,” Jeff Wiezorek, Kite’s senior vice president of clinical development, said in a statement

CAR-T therapies, although promising, but complex and may not be seen as cost-effective, or have limited applications, by the industry. In late August, Novartis dissolved its Gene and Cell Therapy Unit. Novartis said it was committed to the continued development of CAR-T therapies, but was absorbing the unit’s work into other parts of the company—part of with the company’s integrated development model.

Juno has also seen its share of bumps in the CAR-T journey, including the death of three patients during the mid-stage trial of JCARO15, a CAR-T therapy for patients with relapsed or refractory B cell acute lymphoblastic leukemia.