BioXcel Therapeutics, Inc. announced the completion of patient enrollment in the safety lead-in portion of the investigator-sponsored Phase 2 trial of BXCL701 in combination with KEYTRUDA® (pembrolizumab) in previously treated metastatic pancreatic ductal adenocarcinoma (PDAC). BioXcel Therapeutics, through its OnkosXcel Therapeutics immuno-oncology subsidiary, is collaborating with Georgetown Lombardi?s Dr. Louis M. Weiner, director of the cancer center, and Dr. Benjamin Weinberg, the study?s principal investigator. BioXcel Therapeutics and Merck & Co.

are providing BXCL701 and KEYTRUDA for the trial, respectively. The trial is evaluating BXCL701, an investigational, oral innate immune activator designed to inflame the tumor microenvironment and thereby augment the activity of checkpoint inhibitors. As part of the trial?s safety lead-in, the first six patients have been enrolled and will be observed for a six-week safety window period.

The trial is then expected to enroll approximately 39 patients in its efficacy phase in a Simon 2-stage single-arm, open-label design [19 + 20 patients]. The primary objective is to determine the 18-week progression-free survival rate. Patients will be monitored radiographically and by tumor markers for response assessment.

Tumor biopsies and blood samples will also be collected over the course of treatment to better understand the mechanism of how the drug combination works in humans. The human proof of concept portion of the trial is expected to start in H1 2024.