- Kite Pharmaceuticals, one of the leading companies in the race to develop a CAR-T therapy, on Monday said it plans to file for U.S. approval of its leading treatment by the end of 2016, provided data the company will release at the end of September proves positive.
- The California biotech is testing the therapy, called KTE-C19, across a number of blood cancers and plans to read out top-line results from the first 50 patients with diffuse large B-cell lymphoma (DLBCL) "by the end of third quarter."
- This (slightly) faster-than expected timeline would put a potential filing and approval well ahead of rival Juno's plans for its CAR-T therapy, which was placed on a (now-resolved) clinical hold after several patients died during testing.
Kite quickly pressed its advantage in the wake of Juno's trial tragedy. Several hours after Juno disclosed the three patient deaths in its ROCKET study, Kite announced it had completed enrollment of the ZUMA-1 trial studying KTE-C19 in patients with DLBCL.
With enrollment in the pivotal study complete, Kite plans to press ahead and read out top-line data from the first 50 DLBCL patients with at least 3 months of follow-up by the end of third quarter. That interim analysis will reveal whether the study has met its pre-specified endpoint for objective response rate.
Kite then expects to present detailed data at the American Society of Hematology annual meeting in December. Provided all data points positive, Kite will then file with regulators by the end of the year.
"Subject to these interim results and discussions with the U.S. Food and Drug Administration, we expect to submit a Biologics License Application (BLA) for KTE-C19 by the end of 2016," said Arie Belldegrun, CEO of Kite.
Kite's application with the FDA would be for accelerated approval in patients with refractory DLBCL, including primary mediastinal B cell lymphoma and transformed follicular lymphoma.
The rapid ramp up towards an FDA filing could mean Kite beats Juno to market with a CAR-T therapy by a wide margin. Last week Juno said it now expects to win approval for its JCAR-015 in acute lymphoblastic leukemia (ALL) as early as the first half of 2018, delayed from 2017.
Although the FDA rapidly reversed the clinical hold on JCAR-015 in July, Juno has had to modify its trial to continue testing. The biotech removed a chemotherapy agent thought to be linked to the deaths from its pre-conditioning regimen and now will only treat one patient per week until six new patients have safely been treated.
Kite also uses that chemo drug, called fludarabine, in all of its ZUMA trials but doesn't think the fatal interaction seen in Juno's study will occur.
Speaking on a call with investors Monday, Kite's chief medical officer emphasized the breadth of experinece Kite has using fludarabine (flu) and another agent called cyclophosphamide (cy) in its pre-treatment regimen.
"Low dose Cy/Flu conditioning regimen using the KTE-C19 program is the culmination of nearly 10 years of cumulative clinical experience at [the National Cancer Institute] and Kite. More than 100 patients have received Cy/Flu conditioning in connection with studies conducted by NCI and Kite," said David Chang, the chief medical officer.
Another competitor, Novartis, has taken a more conservative approach to CAR-T development, and appears to be eyeing filing with the FDA next year. Although that now looks more timely given Juno's delay.