Positive Kite CAR-T data sees shares jump as it eyes FDA filing

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Kite will need longer-term data to convince people it truly has a cure on its hands, but it now has enough evidence to make its case to an FDA that has shown its willingness to grant early approvals of cancer drugs.

Kite Pharma’s pioneering CAR-T has shown sustained efficacy over six months as its shares jumped 16% on the news. The latest data drop from the pivotal trial of patients with aggressive non-Hodgkin lymphoma (NHL) shows most of the responses seen at three months persisted, diminishing concerns about the durability of response in the run-up to a filing for FDA approval.

After six months, 36% of the 77 patients with diffuse large B-cell lymphoma (DLBCL) had experienced an objective response (OR), down from 39% at the three month mark. The decline in complete responses (CR) was similarly slight, slipping from 33% to 31%. Data for the study as a whole, which includes patients with other forms of NHL, held up, too. The rate of ORs and CRs fell by three percentage points across the full 101-patient study.

The results allay concerns that the decline in responses seen between months one and three would continue out to month six. Back then, the drop in ORR in DLBCL from 76% to 39% in the early part of the study had observers fretting about the durability of the treatment.

That sharp initial drop-off has persisted as Kite has treated more patients. But importantly, the decline between three and six months is far gentler, suggesting that KTE-C19 can trigger long-term improvements in patients who weren’t responding to existing treatments. The safety results were solid, too, with Kite reporting no adverse event-related deaths or new side effect concerns.

Positive six-month data give Kite a stronger hand as it races to file a BLA with FDA by the end of the quarter, a timeline that Novartis is seeking to match stride for stride. And Kite SVP Jeff Wiezorek, M.D., was sufficiently emboldened to use the “C” word, despite the potential for response rates to fall away in the coming months.

“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,” Wiezorek said in a statement.

Kite will need longer-term data to convince people it truly has a cure on its hands, but it now has enough evidence to make its case to an FDA that has shown its willingness to grant early approvals of cancer drugs.

Analysts at Jefferies saw the update as positive, saying in a note to clients: "We believe these data points could increase how FDA views the overall benefit of axi-cel. As expected, safety was similar to what was reported at ASH with a 13% Grd 3/4 CRS rate, 28% Grd 3/4 neurotox, and only 3 patient deaths."

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