BioXcel Therapeutics Announces Completion of Patient Enrollment in Safety Portion of Investigator-Sponsored Phase 2 Relapsed Pancreatic Cancer Trial of BXCL701 in Combination with KEYTRUDA - Seite 2

The American Cancer Society estimates that, in 2024, approximately 66,440 cases of pancreatic cancer will be diagnosed in the United States.² 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.³ Preclinical xenograft models of pancreatic cancer demonstrated strong synergy between BXCL701 and checkpoint inhibitors and reduced tumor growth and promoted an increase in intratumoral 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 tumor stroma. These findings have been published in the Journal for ImmunoTherapy of Cancer⁴, and recently presented at the 2023 annual meeting of the Society for Immunotherapy of Cancer⁵

“The major challenge in immuno-oncology is cold tumors 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 tumor 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.”

About BXCL701
BXCL701 is an investigational, oral innate immune activator designed to initiate inflammation in the tumor 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” tumors “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 U.S. Food and Drug Administration in four indications: acute myelogenous leukemia, 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.