- Kite Pharma, fresh off positive interim data for its lead CAR-T candidate, remains confident it will be able to file an application for approval of the cell therapy by the end of the year, company executives said at an investor day presentation yesterday.
- Chief Medical Officer David Chang indicated Kite was readying for a pre-filing meeting with the Food and Drug Administration which would give greater clarity on timing. But Chang emphasized Kite believes it currently has fileable data, even without six month follow-up results on KTE-C19.
- Kite also gave further details on its plans for a potential launch, detailing a controlled, targeted approach if KTE-C19 is approved. Kite said it would target 72 institutions over the first 12 months following launch, which the company expects would give it access to much of the initially addressable market.
Thrust into the lead position in CAR-T, Kite Pharma hopes to capitalize on its first-mover advantage. The California cell therapy company used its investor day to underscore its readiness — both to file a biologics license application for KTE-C19 and to quickly launch if regulators give a green light.
Kite currently has positive data from the first 51 patients with diffuse large B-cell lymphoma (DLBCL) who are enrolled in its Phase 2 ZUMA-1 study and have at least 3 months of follow-up. According to those results, treatment with KTE-C19 led to a complete response in a third of DLBCL patients and an objective response rate in nearly 40%.
Durability of response is a major question mark, however, and Kite won't have 6 month follow-up data until a primary analysis set to read out in the first quarter of next year.
Still, the company says its ready to go with the data it has now. "I think you have to look at - do we have fileable data? In my mind, the answer is an absolute resounding yes," Chang said during the presentation.
In the meantime, Kite has been busy laying the groundwork of a potential launch. Shawn Tomasello, chief commercial officer, said the company has targeted 10 institutions which already have experience with CAR-T for an initial launch. Then, over the first 12 months post-launch, Kite plans to roll-out KTE-C19 to a total of 72 institutions, which would cover nearly 90% of DLBCL transplant patients.
This controlled approach, coupled with nature of CAR-T, means any launch would be much more lean than a typical pharmaceutical go-to-market strategy, Tomasello told BioPharma Dive in an interview.
"I don't think we need a huge commercial and medical affairs organization to execute on a therapy that is transformational and will be used largely, at least in the beginning, in some pre-targeted institutions," Tomasello said.
Kite anticipates KTE-C19 (if approved) would likely face an even split between government and commercial payers. While the company didn't tip its hand on price, it did point toward the wholesale acquisition cost for current blood cancer treatment options. Two years of treatment with Revlimid, for example, costs an estimated $300,000, while total charges for an allogeneic stem cell transplant weigh in at over $900,000, according to figures cited by Kite.
Still, Tomasello said it would be too early to draw any firm conclusions on pricing for KTE-C19, as Kite continues to conduct price research.
In order to support all of these commercial plans, Kite has readied a manufacturing process which it claims can turn around a finished CAR-T therapy in 14 to 16 days from apheresis. The company also expects to rapidly expand its technical operations headcount in preparation for a launch.
Much still hinges on Kite's conversations with the FDA, which could want to see 6-month follow-up data before reviewing KTE-C19. And in any case, Kite will need the primary analysis scheduled for early next year to show the initial responses seen in the interim analysis can persist.