BioXcel Therapeutics Announces FDA Clearance of IND Application

BioXcel Therapeutics recently announced an update of its immuno-oncology program for BXCL701, an orally available systemic innate-immune activator with dual mechanisms of action. BTI is a clinical-stage biopharmaceutical development company utilizing novel artificial intelligence approaches to identify and advance the next wave of medicines in neuroscience and immuno-oncology.

BTI is pleased to report that its Investigational New Drug (IND) application has received clearance from the US FDA to start a clinical trial to evaluate the triple combination of BXCL701, bempegaldesleukin and avelumab (BAVENCIO) for the treatment of pancreatic cancer as a second line therapy. BTI is collaborating with Nektar Therapeutics, Merck KGaA, Darmstadt, Germany and Pfizer to conduct this trial.

Louis M. Weiner, MD, Director of Georgetown Lombardi Comprehensive Cancer Center, a National Cancer Institute (NCI)-designated comprehensive cancer center, will serve as the Principal Investigator.

In addition, BTI also announced the acceptance of the company’s clinical trial authorization (CTA) by the UK’s MHRA of a trial evaluating the combination of BXCL701 and pembrolizumab (Keytruda) in tNEPC (treatment emergent neuroendocrine prostate cancer). Under the supervision of the European Principal Investigator, Professor Johann de Bono, MD, PhD, of The Royal Marsden NHS Foundation Trust and The Institute of Cancer Research, BTI plans to open its Phase 1b/2 study of BXCL701 and Keytruda in tNEPC, a hormone-refractory form of prostate cancer, in the UK. BTI currently believes that no viable treatment option exists for this type of cancer. The company has opened multiple sites in the US, and data from the open-label trial is expected to support the ongoing global clinical development of BXCL701.

Vincent O’Neill, MD, Senior Vice President & Chief Medical Officer, commented “We are excited to be able to proceed with site activation and enrollment for our triple combination study in second line pancreatic cancer. In addition, we are pleased with the progress made in expanding our global footprint for clinical sites for our tNEPC study. Both of these conditions sadly remain challenging, creating significant unmet medical needs. We eagerly await data that will help define the role of BXCL701 in these conditions.”

Chetan Lathia, PhD, Senior Vice President & Head, Translational Medicine, Clinical Pharmacology & Regulatory Affairs, added “We are very excited to receive FDA clearance of the IND application, for the combination of BXCL701, bempegaldesleukin and avelumab in pancreatic cancer. In addition, we are gratified with the MHRA approval of the CTA of a trial evaluating the combination of BXCL701 and Keytruda in tNEPC patients. This approval reflects BTI’s initial step in seeking regulatory approvals, to conduct clinical trials, around the world for products under development.”

Pancreatic cancer is a disease in which malignant (cancer) cells are found in the tissues of the pancreas. According to the American Cancer Society, pancreatic cancer accounts for approximately 3% of all cancers in the US and approximately 7% of all cancer deaths. Advanced pancreatic cancer is particularly aggressive, with a 5-year survival rate of less than 10%. Limited therapeutic options are currently available for this indication, further reinforcing the need to develop new therapeutic strategies and rational drug combinations aimed at improving overall patient outcomes and quality of life.

tNEPC is a hormone-refractory manifestation of prostate cancer occurring secondary to treatment with androgen deprivation therapies such as Zytiga (Johnson & Johnson) and Xtandi (Pfizer). This form of highly aggressive tumor, with no current treatment, is observed in approximately 20% to 30% of patients treated with androgen inhibitors and has a median survival time of less than 1 year. Single agent checkpoint inhibitor therapy produces very low response rates in hormone refractory prostate cancer, creating a major unmet medical need for tNEPC patients.

BXCL701 is an investigational orally available systemic innate-immune activator with dual mechanisms of action. It has demonstrated single agent activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients. Designed to stimulate both the innate and acquired immune systems, BXCL701 works by inhibiting dipeptidyl peptidase (DPP) 8/9 and blocking immune evasion by targeting Fibroblast Activation Protein (FAP). Preclinical combination data evaluating BXCL701, a checkpoint inhibitor and other immuno-oncology agents has demonstrated encouraging anti-tumor activity in multiple tumor types and formation of functional immunological memory. BXCL701’s primary mechanism of action has recently been highlighted in multiple peer reviewed journals, providing an important validation of the scientific rationale behind BXCL701.

BioXcel Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on drug development that utilizes novel artificial intelligence approaches to identify the next wave of medicines across neuroscience and immuno-oncology. BTI’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 indices. BTI’s two most advanced clinical development programs are BXCL501, a sublingual thin film formulation designed for acute treatment of agitation resulting from neurological and psychiatric disorders, and BXCL701, an immuno-oncology agent designed for the treatment of prostate cancer and for treatment of pancreatic cancer. For more information, visit