Phase 2 BXCL701-Keytruda Trial: Preliminary Results for Metastatic Pancreatic Cancer at ASCO 2024

BioXcel Therapeutics, a company at the forefront of utilizing AI for developing breakthrough treatments in neuroscience and immuno-oncology, has announced the selection of a late-breaking abstract highlighting early findings from a Phase 2 trial of their drug BXCL701. The trial, in conjunction with KEYTRUDA® (pembrolizumab), is aimed at treating patients with previously treated metastatic pancreatic ductal adenocarcinoma (PDAC). The abstract will be unveiled at a poster session during the esteemed American Society of Clinical Oncology (ASCO) Annual Meeting, to be held in Chicago from May 31 to June 4, 2024.

The pivotal trial is spearheaded by the Lombardi Comprehensive Cancer Center at Georgetown University and is assessing the efficacy of BXCL701, an experimental oral innate immune activator. This drug is designed to stimulate the tumor microenvironment, enhancing the efficacy of checkpoint inhibitors. On February 6, 2024, BioXcel Therapeutics reported the completion of patient enrollment for the safety lead-in portion of the trial. The collaboration for this study involves Dr. Louis M. Weiner, the director of the Lombardi Comprehensive Cancer Center, and Dr. Benjamin Weinberg, the principal investigator, through BioXcel's immuno-oncology subsidiary, OnkosXcel Therapeutics. Both BXCL701 and KEYTRUDA are being supplied by BioXcel Therapeutics and Merck & Co., respectively, for the trial.

Dr. Benjamin Weinberg is slated to present the findings, which are encapsulated in a poster titled "Phase 2 Trial of BXCL701 and Pembrolizumab in Patients with Metastatic Pancreatic Ductal Adenocarcinoma (EXPEL-PANC): Preliminary Findings." The abstract, designated as LBA4132, will be displayed at poster number 112 during the session on gastrointestinal cancers. The unveiling of the poster abstract is scheduled for 7:00 AM CT / 8:00 AM ET on the day of the presentation, June 1, 2024.

BXCL701 is an innovative oral innate immune activator intended to initiate inflammation within the tumor microenvironment, addressing the challenge of "cold" tumors that often do not respond to standard immunotherapies. The drug is being evaluated for its potential to convert these "cold" tumors into "hot" ones, thereby making them more visible to the adaptive immune system and fostering a robust anticancer immune response. Preclinical data from OnkosXcel Therapeutics suggest that BXCL701 could synergize with existing checkpoint inhibitors and emerging T-cell activating immunotherapies. Currently, BXCL701 is in development for aggressive prostate cancer and advanced solid tumors that are either resistant to or have not been previously treated with checkpoint inhibitors. The drug has received Orphan Drug Designation from the U.S. Food and Drug Administration for several indications, including acute myelogenous leukemia, pancreatic cancer, melanoma, and soft tissue sarcoma. Additionally, the FDA has granted Fast Track designation for the investigation of BXCL701 in combination with a checkpoint inhibitor for the treatment of metastatic small cell neuroendocrine prostate cancer (SCNC) patients who have progressed on chemotherapy and do not exhibit microsatellite instability. An extensive clinical database supports the ongoing development of BXCL701.

BioXcel Therapeutics, Inc., listed on Nasdaq as BTAI, is a biopharmaceutical enterprise harnessing the power of AI to innovate in neuroscience. Its subsidiary, OnkosXcel Therapeutics, is dedicated to the advancement of immuno-oncology treatments. The company's approach to drug re-innovation involves the use of existing approved drugs and clinically validated candidates, combined with big data and advanced machine learning algorithms, to discover new therapeutic applications.