Kite Pharma’s closely-watched lead CAR-T turned in positive six-month results for aggressive non-Hodgkin lymphoma, hitting a key goal as the biotech rolls up to the FDA with its application for marketing approval.
A group of skeptics had lashed the company after its three-month results, which demonstrated a cluster of relapses and a sharp drop in objective response rates and complete responses for axicabtagene ciloleucel (KTE-C19). But CEO Arie Belldegrun told me last fall that investigators believed that the six-month results would show that the efficacy plateaued, demonstrating its durability.
And that’s what happened.
At six months, the ORR in diffuse large B-cell lymphoma (DLBCL) hit 36%, down only three points from month three. The CR rate was 31%, down two points. Bigger drops were also recorded for primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL) enrolled in Cohort 2, but the six-month numbers posted were all clearly statistically significant.
“These results with axicabtagene ciloleucel are exceptional and suggest that more than a third of patients with refractory aggressive NHL could potentially be cured after a single infusion of axicabtagene ciloleucel,” said Jeff Wiezorek, MD, Senior Vice President of Clinical Development at Kite. “The ZUMA-1 study was built on a foundation of support and commitment from Dr. Steven Rosenberg and the National Cancer Institute and our ZUMA-1 clinical trial investigators who believed in the potential for CAR-T therapy to change the paradigm of cancer treatment.”
Its stock shot up more than 20% this morning.
Kite will now complete its rolling submission for this drug next month, before the end of Q1. That leaves it neck-and-neck with Novartis, which has its own pioneering application for its CAR-T headed to regulators as well.
The key point today is that Kite’s CAR-T looks durable, for now. As Leerink’s Michael Schmidt noted: “Importantly, the responses seem to be durable based on this landmark analysis…”
“Given the data, we see the chances of axi-cel accelerated approval as likely,” wrote Jefferies’ Biren Amin.
But some observers were rankled by Kite’s claim of a potential cure, noting that the therapy still has a long way to go before its durability can be fully assessed. Still, there’s plenty enough here for a quick OK.
CAR-T drugs burst on the biotech scene a few years ago as Kite, Juno and Novartis all angled for the lead spot in search of the first marketing approval. These first-generation drugs extract T cells from patients and then reengineer them to attack cancer cells.
It’s been an arduous and often dangerous process for the late-stage cancer patients enrolled in these studies. Kite notes that the deaths of three patients have been linked to their drug. Juno’s lead program is still in limbo, after it was forced to suspend work after a string of deaths caused by brain swelling.
The adverse events they recorded are well known to the field and the agency, Belldegrun told me last year. And investigators saw breakthrough benefits for their therapy. Based on SCHOLAR-1 data, these patients would normally expect to see an 8% complete response rate. Kite, he added, also proved that it could manufacture the personalized therapy for 22 centers, most of which had never handled the therapy before.
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