BioXcel Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has designated as a Fast Track development program the investigation of BXCL701 in combination with a CPI for the treatment of patients with metastatic small cell neuroendocrine prostate cancer (SCNC) with progression on chemotherapy and no evidence of microsatellite instability. The FDA grants Fast Track designation to facilitate the development and expedite the review of medicines to treat serious conditions, fill unmet medical needs, and bring promising medicines to patients more quickly. Therapies granted this designation are given the opportunity for more frequent interactions with the FDA, a rolling review, and potential eligibility for accelerated approval and priority review. SCNC, classified as a ?cold?

tumor, represents an underserved, growing patient population, with cases increasing due to earlier and more widespread use of androgen receptor inhibitors. In 2024, the American Cancer Society estimates that 299,0101 men will be diagnosed with prostate cancer in the United States, with approximately 20% expected to progress to the more aggressive metastatic castration-resistant form, including an estimated 11,960 patients expected to progress to SCNC2. 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 five indications: SCNC, 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.