BioXcel Therapeutics’ stock hit an all-time high on Monday after the company reported final clinical trial results showing its lead drug successfully calmed agitation in patients with schizophrenia and bipolar disorder.
The positive data paves the way for the New Haven company to seek its first U.S. Food & Drug Administration approval in early 2021, the company said.
BioXcel’s stock hit a record $71.50 Monday on the news. It was trading at $63.80 as of noon Tuesday, bringing the company’s market capitalization to $1.3 billion.
BioXcel uses artificial intelligence to repurpose older drugs and discover new ones. Its lead drug, BXCL501, is a thin-film formulation of an older Pfizer drug known as “Dex” (dexmedetomidine). Similar to a mouthwash strip, BioXcel’s version tastes minty and dissolves under the tongue.
The company had been testing the drug against agitation in schizophrenia and bipolar disorder in two Phase 3 trials of nearly 400 patients each. Phase 3 is the last stage of human testing before regulatory approval.
In both studies, the drug met the primary goal of statistically significant and clinically meaningful improvements after two hours, as measured by a commonly used agitation score. The response lasted for four hours after treatment.
The drug also met the secondary goal of working as soon as 20 minutes, and was well tolerated by patients, BioXcel said.
“These compelling Phase 3 results show that BXCL501, if approved, has the potential to become an important new treatment option for patients suffering from acute agitation,” CEO Vimal Mehta said in a statement. “We are extremely pleased that rapid and robust reductions in agitation were demonstrated in both patient populations despite differing neuropsychiatric diagnoses.”
The studies found schizophrenia patients taking 180 mcgs. of the drug improved by 10.3 points on the “excitatory component” of the Positive and Negative Syndrome Scale (PEC), compared to 8.5 points for the 120 mcg. dose and 4.8 points for the placebo. Bipolar patients posted similar results.
Patients also improved using two additional measures of agitation, the Agitation and Calmness Scale (ACES) and Clinical Global Impression-Improvement Scale (CGI-I), BioXcel said.
In a statement, John Krystal, MD, Yale School of Medicine professor of psychiatry and neuroscience and a clinical advisor to BioXcel, said managing agitation has always been challenging for health care providers.
“We would welcome an oral treatment option that is safe, has a quick onset of action and reduces agitation with a minimum of sedation,” he said.
BioXcel is also conducting human testing on the drug for agitation associated with dementia and opioid withdrawal, and is planning another trial for patients with delirium.
Meanwhile, Massachusetts General Hospital has begun using the experimental drug to treat “ICU delirium” in COVID-19 patients, under an FDA compassionate use program.
Contact Natalie Missakian at news@newhavenbiz.com.
