BioXcel completes patient enrollment in safety portion

BioXcel completes patient enrollment in safety portion of investigator-sponsored phase 2 relapsed pancreatic cancer trial of BXCL701 in combo with Keytruda

BioXcel Therapeutics, Inc., a biopharmaceutical company, 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 tumour 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 tumour markers for response assessment. Tumour 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.

“Pancreatic cancer represents a significant unmet medical need, ranking as the third-leading cause of cancer deaths, yet remains an exceptionally difficult cancer to treat. No novel therapies have emerged in decades, and overall survival for advanced disease has not improved in over 10 years,” said Dr. Weiner. “Building on results from the preclinical work conducted in our lab, we are excited to evaluate BXCL701 in this important trial as a potential treatment for advanced pancreatic cancer.”

The American Cancer Society estimates that, in 2024, approximately 66,440 cases of pancreatic cancer will be diagnosed in the United States. Few therapeutic options are available for patients with this indication, which has a five-year survival rate of 13%, among the lowest of all cancers. Preclinical xenograft models of pancreatic cancer demonstrated strong synergy between BXCL701 and checkpoint inhibitors and reduced tumour growth and promoted an increase in intratumoural T cells, macrophages and NK cells, with induction of host-protective immunity. In addition, preclinical studies showed that BXCL701 has the potential to provide a marked anti-fibrotic effect, as seen in the tumour stroma. These findings have been published in the Journal for ImmunoTherapy of Cancer, and recently presented at the 2023 annual meeting of the Society for Immunotherapy of Cancer.

“The major challenge in immuno-oncology is cold tumours and their lack of sensitivity to checkpoint therapy,” said Vincent J. O’Neill, M.D., executive vice president, chief of product development and medical officer of BioXcel Therapeutics. “We have already demonstrated encouraging response rates and survival data in two aggressive forms of prostate cancer: small cell neuroendocrine cancer (SCNC) and adenocarcinoma, as highlighted in our Key Opinion Leader Day presentation last year. This phase 2 trial marks the third cold tumour setting where we are testing BXCL701 in combination with Keytruda. Based on encouraging preclinical results, we look forward to the results from the human proof of concept efficacy trial evaluating BXCL701 in combination with pembrolizumab in patients with PDAC.”

BXCL701 is an investigational, oral innate immune activator designed to initiate inflammation in the tumour microenvironment. Approved and experimental immunotherapies often fail to address cancers that appear “cold.” Therefore, BXCL701 is being evaluated to determine if it can render “cold” tumours “hot,” making them more detectable by the adaptive immune system and thereby facilitating the development of a strong anticancer immune response. OnkosXcel Therapeutics’ preclinical data support BXCL701’s potential synergy with both current checkpoint inhibitors and emerging immunotherapies directed to activate T-cells. BXCL701 is currently being developed as a potential therapy for the treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors. BXCL701 has received Orphan Drug Designation from the US Food and Drug Administration in four indications: acute myelogenous leukaemia, pancreatic cancer, stage IIb to IV melanoma, and soft tissue sarcoma. An 800+-subject clinical database, with data collected by the company and others, supports the ongoing development of BXCL701.

BioXcel Therapeutics, Inc. is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. Its wholly owned subsidiary, OnkosXcel Therapeutics, is focused on the development of medicines in immuno-oncology. The Company’s drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications.

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