BXCL701 may improve patient response when combined with checkpoint inhibitors or re-sensitize patients who have failed checkpoint therapy

NEW HAVEN, Conn., Dec. 11, 2019 (GLOBE NEWSWIRE) — BioXcel Therapeutics, Inc. (“BTI” or the “Company”) (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to identify and advance the next wave of medicines in neuroscience and immuno-oncology, today announced that the Company will advance the clinical evaluation of BXCL701 into multiple advanced solid tumors. BXCL701 is an orally active, systemic innate immunity activator with dual mechanisms of action.

“This new study allows us to accelerate the evaluation of BXCL701 in combination with a checkpoint inhibitor in patients with a range of tumor types where checkpoint inhibitors are standard of care,” commented Vincent J. O’Neill, M.D., Senior Vice President and Chief Medical Officer of BTI. “We believe BXCL701 may have the potential to extend treatment responses to KEYTRUDA^®, a PD-1 inhibitor, when used together to treat advanced solid cancers. This trial expands on our studies of BXCL701 in prostate and pancreatic cancers as we explore its full potential.”

The open-label Phase 2 basket trial (NCT04171219) will take place at a leading cancer center in the U.S. The study is designed to evaluate the response rate of orally administered BXCL701, combined with Pembrolizumab (KEYTRUDA^®) in patients with advanced solid cancers. If successful, the study would provide an option for patients who have failed or are refractory to checkpoint therapy. Outcome measures will include progression-free-survival, overall survival, and duration of response, as well as the safety of the combined treatment.

To learn more about the trial, visit https://clinicaltrials.gov/

About BXCL701:

BXCL701 is an investigational orally administered innate immune activator designed to initiate inflammation in the tumor microenvironment. Approved and experimental immunotherapies often struggle to address cancers that appear “cold” or uninflamed. Therefore, BXCL701 may render “cold” tumors “hot,” making them more detectable by the adaptive immune system and thereby facilitating the development of a strong anti-cancer immune response. BTI’s data supports BXCL701’s synergy with both current checkpoint inhibitor-based therapies and emerging immunotherapies directed to activate T-cells, such as IL-2 and OX40 agonist antibodies.

This candidate is currently being developed as therapy for treatment emergent Neuroendocrine Prostate Cancer (tNEPC) and pancreatic cancer (both “cold” tumors) and other advanced solid cancers that are “hot” or have become resistant to checkpoint inhibitors. The safety escalation portion of the trial evaluating the double combination of BXCL701 and KEYTRUDA^® for tNEPC is ongoing with data read-out expected in the first half of 2020. The FDA has also accepted an IND application for the triple combination of BXCL701, bempegaldesleukin (produced by Nektar Therapeutics, Inc., or Nektar) and BAVENCIO^® (avelumab, Merck KGaA, Darmstadt, Germany and Pfizer) in pancreatic cancer. The triple combination trial is expected to begin following Nektar and Pfizer’s safety run-in study of a double combination of bempegaldesleukin and avelumab and the outcome of that trial.

About BioXcel Therapeutics, Inc.:

BioXcel Therapeutics, Inc. is a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology. BTI’s drug re-innovation approach leverages existing approved drugs and/or clinically evaluated 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, an investigational sublingual thin film formulation in development for acute treatment of agitation resulting from neuropsychiatric disorders, and BXCL701, an investigational orally administered systemic innate immunity activator in development for treatment of a rare form of prostate cancer, pancreatic cancer and advanced solid cancers in combination with other immuno-oncology agents. For more information, please visit www.bioxceltherapeutics.com.