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BioXcel Therapeutics's acute myeloid leukemia drug gets US FDA orphan status

BioXcel Therapeutics Inc. said its experimental oral drug BXCL701 received an orphan drug designation in the U.S. to treat patients with acute myeloid leukemia.

Acute myeloid leukemia, or AML, is an aggressive type of cancer in which the bone marrow makes abnormal immature red blood cells, resulting in a decline in mature red blood cells, platelets and white blood cells.

The drug targets, attacks and kills the AML cells. It activates innate immune cells known as macrophages by inhibiting certain enzymes, and it also stimulates the immune system through a process known as pyroptosis, an inflammatory form of programmed cell death.

"BXCL701 has been observed to directly attack and kill AML cells in multiple preclinical studies, corroborating our belief that BXCL701 presents an opportunity to address this rare and deadly disease. We plan to further investigate BXCL701 as a single agent and/or in combination for specific patient segments with high unmet medical needs," Chief Medical Officer Vincent O'Neill said in the release.

The U.S. estimates about 21,450 new cases of AML in 2019.

BXCL701 previously received orphan-drug designations from the U.S. Food and Drug Administration for pancreatic cancer and melanoma and is also being studied to treat a form of prostate cancer.

The FDA's orphan drug program is meant for medicines that can treat rare diseases or disorders that affect fewer than 200,000 people in the U.S. With the designation, BioXcel Therapeutics's treatment will be eligible for certain incentives, including tax credits for covering clinical trial expenses, prescription drug user fee waivers and seven-year market exclusivity after approval.