BioXcel Therapeutics Announces NIH NIDA Grant to Columbia University to Support Further Studies of BXCL501 (Sublingual Dexmedetomidine) for Treating Opioid Withdrawal

• Multi-year-funded award to support a 160-patient, randomized-controlled study in patients undergoing opioid withdrawal treatment
• Over 142 million opioid prescriptions dispensed in the U.S. in 2020¹
• More than 1.7 million people in the U.S. suffered from substance use disorders related to prescription opioid pain relievers in 2016¹

NEW HAVEN, Conn., Aug. 01, 2022 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a commercial-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced that the National Institute on Drug Abuse, part of the National Institutes of Health (NIH), has awarded Columbia University a multi-year NIDA grant to fund clinical testing of BXCL501 (sublingual dexmedetomidine) as a potential treatment for opioid withdrawal. The initial grant provides approximately $3.3 million for the project from August 1, 2022 through July 31, 2024 to support a 4-arm Phase 2b study comparing BXCL501 180 mcg and 240 mcg BID to placebo and Lucemyra™, to be followed by approximately $4.5 million to support a registrational study upon the completion of certain milestones.

Opioid abuse has long been recognized as a national health crisis. More than 142 million opioid prescriptions were dispensed in the U.S. in 2020, according to the Centers for Disease Control and Prevention (CDC), which also estimates that 1.7 million people in the U.S. suffered from substance use disorders related to prescription opioid pain relievers in 2016. In addition, the drug overdose epidemic claimed an estimated 104,000 lives in the 12-month period ending in September 20211 and a White House Council of Economic Advisers recently assessed the cost of the opioid crisis at $1 trillion2. 

Columbia University enrolled patients in the RELEASE trial — a multicenter, randomized, double-blind, placebo-controlled, ascending dose Phase 1b/2 trial designed to evaluate the safety, pharmacokinetics, tolerability, and efficacy of BXCL501 administered twice daily for seven days — in patients experiencing symptoms of opioid withdrawal. In March 2021, BioXcel Therapeutics announced RELEASE top line results. BXCL501 was generally well tolerated, with no severe or serious adverse events reported across all doses evaluated (30 mcg, 60 mcg, 90 mcg, 120 mcg, 180 mcg and 240 mcg).  After further post-hoc analysis, the 240 mcg BID dose significantly reduced both subjective ratings of insomnia and clinician ratings of anxiety or irritability among enrolled patients.

“We are pleased to continue testing BXCL501 in patients with opioid withdrawal with support from the NIH,” said Dr. Frances Levin, Professor of Psychiatry at Columbia University and Chief of the Division on Substance Use Disorders at New York State Psychiatric Institute/Columbia University, Multiple Principal Investigator of the study and Project Director for the grant.

Dr. Sandra Comer, Multiple Principal Investigator of the study and Professor of Neurobiology in the Department of Psychiatry at Columbia University, noted that “BXCL501 shows promise in treating opioid withdrawal symptoms, which may facilitate transition of patients with opioid use disorder to treatment medications such as buprenorphine and naltrexone.”

“The NIDA/NIH grant to Columbia University provides important support for BXCL501’s development as a potential for opioid treatment withdrawal, a significant national challenge,” said Frank Yocca, Ph.D., Chief Scientific Officer of BioXcel Therapeutics. “We believe the results observed thus far in multiple dose regimens within the RELEASE trial provide valuable insights to support investigation across additional indications and treatment settings.”

This research is supported by the National Institute on Drug Abuse of the National Institutes of Health under award number UG3DA056247. The content presented in this release is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.