CAR-T race heats up as Kite, Novartis complete filings to FDA
- CAR-T therapy took another step to becoming a commercial reality over the past week as both Kite Pharma and Novartis completed filings to the Food and Drug Administration for their respective treatments, setting the stage for potential approvals to come later this year.
- Strengthening Kite's chances were updated primary results from the ZUMA-1 trial of Kite's CAR-T therapy axi-cel. The data, presented Sunday, showed slightly improved response rates, including a complete response rate of 39% across non-Hodgkin lymphoma (NHL) types, compared to topline data released back in February.
- Notably, median overall survival still has not been reached, with 80% of patients alive after 6 months, a data point that analysts from Jefferies see as another puzzle piece supporting approval by the FDA later this year.
While Kite had previously released primary data on ZUMA-1, results presented Sunday at the annual meeting of the American Association for Cancer Research solidified a strong profile for the biotech's CAR-T therapy.
Across two NHL cohorts in the study, 44% of patients experienced an ongoing overall response, with 39% in complete response. Those response rates were a tick higher than the 41% and 36% overall and complete response rates reported in February, likely due to individuals in complete response who did not have 6 months of follow-up two months ago now being included.
Rates of cytokine release syndrome and adverse neurological events — two closely watched safety issues — had dropped between the initial interim analysis and the results released in February. At Sunday's AACR presentation, Frederick Locke, a ZUMA-1 co-lead investigator from the Moffitt Cancer Center, attributed the decline in adverse event rates to increased comfort with CAR-T treatment and its side effects among physicians managing these patients
The updated results come two days after Kite announced it had completed its filing for approval of axi-cel with the FDA, a milestone that puts the California biotech on track to win a potential landmark approval later this year.
Novartis' experimental therapy CTL019 might challenge axi-cel for claim to the title of first CAR-T therapy approved, however. After quietly submitting an application for approval of CTL019, the Swiss pharma revealed on Mar. 29 that the FDA had granted priority review for the CAR-T treatment, now referred to as tisagenlecleucel-T. With an expedited regulatory review in hand, Novartis could possibly secure approval by the end of the third quarter.
It's still unclear how much of an advantage being first to market will be, particularly since the two companies are driving towards approval in two different indications. Novartis is aiming for an approval in pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia, while Kite is seeking approval in patients with relapsed/refractory aggressive NHL who are ineligible for autologous stem cell transplant.
Approval in NHL would initially give Kite a larger market to target, although Novartis is expected to soon release data from its JULIET trial, which is studying CTL019 in diffuse large B-cell lymphoma (DLBCL) A primary analysis of 80 patients with at least 3 months of follow-up is planned for the second quarter, with a submission slated for the fourth quarter. Approval in DLBCL for Novartis would allow the pharma to challenge Kite.
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