How Fast Can Kite Pharma File for an Approval of its Lead CAR-T?
October 19, 2016
By Alex Keown, BioSpace.com Breaking News Staff
NEW YORK – Shares of Kite Pharma jumped slightly in after-market and pre-market trading after Arie Belldegrun told investors the company aims to file for an approval of its lead CAR-T therapy by the end of the year.
Belldegrun shared the news about the company’s lead CAR-T product, KTE-C19 with investors at the company’s Investor Day meeting in New York. Belldegrun told the investors and members of the media that he has scheduled a pre-application meeting with the U.S. Food and Drug Administration, which will “provide the clarity Kite is looking for on submitting the data,” he said, according to an Endpoints report on the meeting.
Kite believes the interim data of the Phase II trial released in September will be strong enough to lead regulators at the U.S. Food and Drug Administration 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.
If the FDA does want more data, Belldegrun said the company will have an additional six months of data in February, which would push back plans to file a New Drug Application to the third quarter, Endpoints said. Even with the delay, Belldegrun said that would give Kite Pharma a lead of about one year over other CAR-T competitors, particularly Novartis, which recently shut down its Cell & Gene Therapy unit, and Juno, which had its own troubles with a clinical trial that saw the death of three patients.
There may be some hurdles though that Kite may have to address in its meeting with the FDA, 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. 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.
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.
But not only did Belldegrun and Kite share positive news about KCE-C19, the company unveiled four new near-term clinical development programs, along with projected timelines for Investigational New Drug Applications, including two planned for this year. Kite said it will file the IND for KITE-196 for the treatment of acute myeloid leukemia and KITE-718 for non-small cell lung cancer and bladder cancer this year. In 2017, the company plans to file an IND for KITE-585 targeting multiple myeloma and in 2018 will file an IND for KITE-439 for cervical cancer and head and neck cancer.
"While we prepare to manufacture and commercialize KTE-C19 upon approval, we believe this is just the beginning. The breadth of the pipeline we unveiled today, combined with our innovative T cells 2.0 programming, has the potential to deliver hope for a cure to thousands of people across 15 hematological and solid cancer indications,” Belldegrun said in a statement at the Investors Day conference.