- Kite Pharma shares soared Tuesday on news the clinical-stage immunotherapy developer plans to file its lead candidate for approval in the next month.
- Axicabtagene ciloleucel, also known as KTE-C19, achieved a 36% complete response rate after half a year of treatment in patients who had one of three types of aggressive B-cell non-Hodgkin lymphoma and didn't respond to chemotherapy. Overall response rates, which also factor in partial responses, were 82% after three months and 41% after six.
- The clinical-stage drugmaker said it will present additional data in April at American Association for Cancer Research, according to a Feb. 28 statement. Safety data will likely be at the forefront of stakeholders' minds, considering the trial had three patient deaths, two of which were related to the drug.
Top-line data in hand, Kite said it should have the Biologics License Application for axicabtagene ciloleucel submitted to the Food and Drug Administration by the end of the first quarter. Stock spiked almost 25% as a result, trading at $70.66 at Tuesday's market close.
The BLA submission will likely make Kite the first to file its application for the gene therapy, ahead of close competitor Novartis and well ahead of former front-runner Juno Therapeutics, which was forced to ditch its lead program due to safety concerns.
Particularly attractive for some analysts was how consistent the newest ZUMA-1 results were compared to Phase 1 data as well as other investigations of the drug.
"To us this indicates that this data is extremely consistent with previous data readouts," Jefferies analyst Biren Amin wrote in a note. Similar response rates at six months and after a three-month follow up are bright spots as well, reaffirming prior studies that showed "the large majority of responses seen at month 3 are durable out to 6 months."
Accelerated approval of the drug is likely by the end of 2017, Jefferies concluded.
"What is very clear is that these patients are driving significant clinical benefit, and at the end of the day that's what FDA will be looking at," Kite CEO Arie Belldegrun said in a Feb. 28 investor call.
Kite declined to comment on whether a panel would be necessary for an approval, but said it would be "more than happy to go to the panel and highlight the results coming from the ZUMA-1 study."
The Phase 2 portion of the ZUMA-1 trial tested 101 patients across two cohorts — 77 who had diffuse large B-cell lymphoma (DLBCL), and 24 who had either primary mediastinal B-cell lymphoma (PMBCL) or transformed follicular lymphoma (TFL). At six months, overall response rates for the cohorts was 36% and 54%, respectively, while complete response rates were 31% and 50%.
Kite noted six-month data for four of the participants across the two cohorts weren't recorded prior to the collection deadline, and correspondingly marked those patients as non-responders for that time frame. The company also acknowledged that overall survival is still unknown because the top-line data has a median follow-up of 8.7 months.
There were multiple severe adverse events seen in the trial. Investigators reported anemia in 43% of patients and abnormally low white blood cell count in 39%. Lower lymphocyte levels and encephalopathy were both seen in about 20% of participants, while 24% had blood platelet deficiencies. Compared to the interim analysis, grade 3 or higher cytokine release syndrome (CRS) decreased from 18% to 13%. There were no cases of cerebral edema.