On November 4, 2014 Exelixis reported positive top-line results from a randomized phase 2 trial of cabozantinib and erlotinib alone or in combination as second- or third-line therapy in patients with stage IV EGFR wild-type non-small cell lung cancer (NSCLC) (Press release Exelixis, NOV 4, 2014, View Source [SID:1234500919]). This trial (Study E1512) is sponsored by the U.S. National Cancer Institute (NCI) through a Cooperative Research and Development Agreement between the Cancer Therapy Evaluation Program (CTEP), Division of Cancer Treatment and Diagnosis, NCI and Exelixis. Study E1512 was designed and is being conducted by the ECOG-ACRIN Cancer Research Group as part of Exelixis’ collaboration with the NCI. Joel Neal, M.D., Ph.D., from ECOG-ACRIN member institution Stanford University/Stanford Cancer Institute, chairs the study.

In the E1512 trial, 125 patients were randomized to one of the three arms: erlotinib, cabozantinib, or the combination. During a pre-planned interim ECOG-ACRIN Data Safety Monitoring Committee analysis for futility, it was found that the trial met its primary endpoint of improving progression-free survival (PFS) with cabozantinib alone and also with the combination of cabozantinib plus erlotinib, as compared to erlotinib alone, and the results were highly statistically significant. Safety data were consistent with those observed in other trials of cabozantinib. At time of analysis, the median follow-up was 5.9 months and overall survival data were immature.

The results of the trial are the subject of ongoing analyses and will be submitted by the investigators for presentation at a future medical conference.

Exelixis President and CEO, Michael M. Morrissey, Ph.D., commented on the results: “This is one of the first substantial data sets from our collaboration with NCI-CTEP, which has enabled us to broaden the cabozantinib development program while focusing our internal resources on our pivotal trials. We are excited by these positive results and are looking forward to working with the trial investigators to support future development of cabozantinib in NSCLC and beyond, while we await top-line results from our pivotal phase 3 trial METEOR in metastatic renal cell carcinoma, now anticipated in the second quarter of 2015.”