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December 31, 2019 Bioscience Notebook

BioXcel launches Phase 3 trials for agitation drug; bladder cancer drug with Yale ties approved 


New Haven artificial intelligence biotech BioXcel Therapeutics Inc. on Monday announced that its drug to treat agitation is a step closer to FDA approval with the start of two Phase 3 clinical trials in patients with schizophrenia and bipolar disorder. 

Phase 3 trials are the final stage in the path to regulatory approval for new drugs.

The drug, BXCL501, is a new version of the older Pfizer drug dexmedetomidine. BioXcel’s formulation is a thin film that dissolves under the tongue, similar to a mouthwash strip. The drug was originally used in surgical units as an intravenous anaesthetic. 

BioXCel, which uses AI to discover new uses for existing drugs, touts BXCL501 as a less invasive way to calm agitated patients. Each year, 8.3 million people in the U.S. suffer from agitation, costing roughly $40 billion in treatment-related expenses, BioXcel said. 

“Managing agitation in patients with neuropsychiatric disorders is a major challenge for both physicians and caregivers,” said CEO Vimal Mehta in a statement. “BXCL501, designed as a non-invasive, fast-acting therapy, might be able to overcome the limitations associated with current standards of care, as we believe it provides calming without excessive sedation while protecting the caregiver-patient relationship.”

The studies, dubbed SERENITY I and II, are randomized, double-blinded, placebo-controlled trials of up to 750 patients ages 18 to 75.  In the first trial, schizophrenia patients suffering from agitation will receive doses of the drug at 120 or 180 micrograms, or a placebo. The second trial will test the same doses against a placebo in agitated patients with bipolar disorder. 

The drug has been awarded “Fast Track” status by the U.S. Food & Drug Administration, which can speed regulatory approval for drugs that treat serious or life-threatening illnesses and meet unmet medical needs. 

Results of the trials are expected in mid-2020. 


A drug with ties to Yale University and described by researchers as a “smart bomb” to treat an advanced form of bladder cancer has won FDA approval. 

Federal regulators approved the drug, enfortumab vedotin (EV), on Dec. 18 for the treatment of advanced urothelial cancer, a form of bladder cancer. The drug was developed by the U.S. affiliate of Tokyo-based Astellas Pharma Inc.

Yale Cancer Center and Smilow Cancer Hospital researchers led a multi-institution clinical trial for the drug, which demonstrated its efficacy in treating the disease. 

“This is very exciting progress as we haven’t had another therapy option for patients whose urothelial or bladder cancer has progressed after chemotherapy or immunotherapy,” said Daniel P. Petrylak, MD, professor of medical oncology and urology and co-director of the Cancer Signaling Research Program at Yale Cancer Center. “To my knowledge, this is the most active single drug in urothelial cancer.”

Petrylak was the senior investigator of a Phase 2 trial on the drug. 

EV is an antibody drug conjugate, which combines an antibody that targets a specific protein on the surface of tumor cells with powerful chemotherapy. 

The trial showed the drug produced responses in 44 percent of patients with locally advanced or metastatic bladder cancer who had been previously treated with chemotherapy and immunotherapy drugs known as checkpoint inhibitors, according to Yale.

Among 125 patients who had received the drug with standard chemotherapy plus a checkpoint inhibitor, 9 percent had no detectable sign of cancer following treatment, Yale said. Median overall survival was 11.7 months. 

The drug was also effective on 36 percent of patients whose cancer spread to the liver, a site that has been resistant to both standard chemotherapy and immunotherapy, Yale said.

Results of the trial were presented last June at the American Society of Clinical Oncology annual meeting.  

Contact Natalie Missakian at

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