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Novel Drug Granted FDA Fast Track in Rare Prostate Cancer Subset

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The Food and Drug Administration has granted a Fast Track designation to the novel immune activator drug BXCL701 in combination with a checkpoint inhibitor for patients with metastatic small cell neuroendocrine prostate cancer (SCNC).

Light blue background with "FDA" in bold black letters.

The FDA granted a Fast Track designation for a novel drug to treat a rare form of prostate cancer.

The Food and Drug Administration (FDA) has granted a Fast Track designation to the novel immune activator drug BXCL701 in combination with a checkpoint inhibitor for the treatment of patients with metastatic small cell neuroendocrine prostate cancer (SCNC) whose disease has progressed on chemotherapy and has no evidence of microsatellite instability, according to biopharmaceutical company BioXcel Therapeutics.

The designation follows recent phase 2 trial findings which show that, among patients with SCNC, the average overall survival (OS; the time a patient lives following treatment, regardless of disease status) for those treated with BXCL701 and immunotherapy Keytruda (pembrolizumab) was 13.6 months, versus 7.6 months among patients treated with Keytruda alone. Researchers also found that 56.5% of patients treated with BXCL701 were still alive after one year.

SCNC, according to research published in Cureus, impacts less than 1% of the population “and is considered a lethal entity of prostate cancer,” with a median survival rate of one to two years following diagnosis.

“SCNC is characterized by poor prognosis and a low survival rate, and current treatment options are suboptimal,” said Dr. Vincent J. O’Neill, executive vice president, chief of product development and Medical Officer of BioXcel Therapeutics, in a news release issued by the company. “We are encouraged by the potential of BXCL701, which has demonstrated clinical proof of concept in both SCNC and adenocarcinoma.”

Fast Track designation is “designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need,” according to the FDA. “The purpose is to get important new drugs to the patient earlier.”

“The FDA’s Fast Track designation for the investigation of BXCL701 in SCNC is an important recognition of our most advanced immuno-oncology asset and an acknowledgment of its potential to address the considerable unmet medical need in these patients. At the same time, it further validates the unique AI-based drug re-innovation approach that we used to discover this asset,” said Vimal Mehta, CEO of BioXcel Therapeutics, in the news release. “BXCL701 has already demonstrated considerable potential in our clinical trials to date, and we plan to further define its development path while exploring strategic options for our OnkosXcel Therapeutics subsidiary.”

BXCL701 is designed to turn “cold” tumors “hot,” a way of describing the process of making cancer cells more deatectible by a patient’s immune system — in this case, by instigating inflammation in the tumor microenvironment. Likewise, Keytruda uses a patient’s immune system to fight cancer, by helping a patient’s T cells find and attack tumor cells.

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