Kite Pharmaceuticals $KITE has lined up a fresh batch of positive efficacy data for its lead CAR-T for advanced cases of acute lymphoblastic leukemia at ASH. And then later Sunday evening the biotech added that it has launched its rolling submission for a first approval.
Investigators turned up at the big ASH meeting to say that the drug spurred a complete remission in 9 of 11 evaluable patients — 82% — enrolled in the ZUMA-3 and ZUMA-4 Phase I studies for ALL in a preliminary review.
But the treatment was not without danger. Five of the 13 had serious bouts of cytokine release syndrome as well as 5 who had grade three or above cases of neurological events. There were no cases of cerebral edema, which have bedeviled rival Juno, but one of the patients in the trials died from CRS.
Kite had long hoped to file its first application for KTE-C19 before the end of the year, but was forced to delay until Q1 2017 after some interactions with the FDA, presumably so it would have time to include data on the 6-month responses of patients in ZUMA-1. Some analysts have been skeptical of the durability of the treatment after seeing the effect decline over a few months of treatment, but company execs expect to see that response level plateau at a significant level.
Soon after Kite posted the latest data on their therapy Sunday, the company announced that it had launched the rolling submission and would get it completed by the end of the first quarter of 2017. But Novartis now may have an edge in filing first, and looking for the first ever approval of a CAR-T therapy.
The drug remains one of the leading CAR-T therapies heading to the FDA. Novartis is now hustling along its own CAR-T after registering an impressive 82% complete remission rate among the 50 children and young adults with B-cell acute lymphoblastic leukemia.
“In the Phase I portion of these studies, we only enrolled patients with high disease burden to rigorously evaluate safety and efficacy of KTE-C19,” said Kite R&D director David Chang. “We look forward to initiating the Phase II portions of these studies in 2017.”
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