NEW HAVEN, Conn. - BioXcel Therapeutics, Inc. (NASDAQ:BTAI) has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for BXCL701, an investigational treatment for metastatic small cell neuroendocrine prostate cancer (SCNC), the company announced Monday. The FDA's Fast Track program aims to accelerate the development and review of drugs intended to treat serious conditions and fill unmet medical needs.
BXCL701, an oral innate immune activator, is designed to stimulate inflammation within the tumor microenvironment and enhance the activity of checkpoint inhibitors (CPIs). This designation follows promising clinical trial results for BXCL701 in treating SCNC, a subtype of prostate cancer with limited treatment options and poor prognosis.
The American Cancer Society estimates that in 2024, approximately 299,010 men will be diagnosed with prostate cancer in the U.S., and about 20% are expected to advance to a more aggressive metastatic form. SCNC accounts for an estimated 11,960 of these cases. The Fast Track status acknowledges BXCL701's potential to meet the significant need for new treatments in this patient population.
BioXcel Therapeutics plans to discuss the registration path for BXCL701 with the FDA in an upcoming meeting. The drug has already shown clinical proof of concept in SCNC and adenocarcinoma, with positive survival results from a Phase 2 trial reported at the end of last year.
The company's subsidiary, OnkosXcel Therapeutics, is also exploring strategic options in light of this development. BXCL701 has also received Orphan Drug Designation from the FDA for several indications, including SCNC, acute myelogenous leukemia, pancreatic cancer, melanoma, and soft tissue sarcoma.
BioXcel Therapeutics leverages artificial intelligence in its drug re-innovation approach, using approved drugs and clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications.
This news is based on a press release statement from BioXcel Therapeutics. The company's approach and the FDA's recognition may offer new hope for patients with aggressive forms of prostate cancer. However, as BXCL701 is still investigational, its efficacy and safety must be established in further clinical trials before it becomes available for widespread clinical use.
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