NEW YORK – Blueprint Medicines unveiled top-line data from its Phase I/II trial on pralsetinib (BLU-667) for the treatment of RET fusion-positive non-small cell lung cancer on Wednesday.
The overall response rate among 80 patients previously treated with chemotherapy was 61 percent, and 95 percent of patients saw their tumors shrink via radiologic imaging. The median duration of response was not yet reached at the time of data collection in mid-November last year. Further, the 26 patients who had not been previously treated had an overall response rate of 73 percent, with 12 percent achieving a complete response to the drug.
The safety profile of the drug was consistent with previous studies, reporting mostly grade 1 or 2 adverse events. However, 4 percent of patients discontinued treatment due to adverse events.
Blueprint intends to use the data from the Phase I/II ARROW trial to support the new drug application submission for pralsetinib in the indication of RET fusion-positive NSCLC by the first quarter of this year. In the second quarter, the company plans to file an NDA for the medullary thyroid cancer indication.
RET fusions appear in approximately 1 to 2 percent of NSCLC patients. Pralsetinib, Blueprint's oral RET inhibitor, was granted breakthrough therapy designation by the US Food and Drug Administration.
In January, Blueprint intends to launch a Phase III trial to study whether pralsetinib extends progression-free survival in first-line RET fusion-positive NSCLC when pitted against chemotherapy or a chemotherapy and pembrolizumab combination.
Eli Lilly opened a Phase III trial for RET fusion-positive NSCLC for its competing RET inhibitor selpercatinib in early December last year.