BioXcel Therapeutics (NASDAQ:BTAI) reported positive topline results from a Phase 2 investigator-sponsored trial evaluating its experimental therapy BXCL501 for the treatment of opioid withdrawal symptoms in adults undergoing methadone tapering.
The study, funded by the National Institute on Drug Abuse, enrolled 80 participants diagnosed with opioid use disorder. Researchers compared BXCL501 administered at doses of 180 µg and 240 µg twice daily with placebo and with lofexidine 0.54 mg given four times daily as an active comparator.
According to the company, the higher BXCL501 dose of 240 µg twice daily significantly reduced withdrawal symptoms compared with placebo during a seven-day methadone taper, as measured using the Short Opiate Withdrawal Scale-Gossop. Patients recorded a reduction in scores of more than 30%, with the strongest improvements observed on days three and four of treatment. The reduction in symptoms also numerically exceeded the improvement seen in patients receiving lofexidine.
The trial also reported lower rates of orthostatic hypotension in patients treated with BXCL501 compared with the active comparator. Orthostatic hypotension occurred in 18% of patients receiving BXCL501 at 180 µg twice daily and in 37% of those receiving the 240 µg dose, versus 50% among patients treated with lofexidine. No cases of sedation or somnolence were reported in the BXCL501 groups, compared with 5% in the lofexidine arm.
The data comes at a challenging time for BioXcel. The company’s shares have declined about 56% over the past six months, recently trading around $1.65 and giving the biotech a market capitalization of roughly $36 million.
The trial population included patients predominantly exposed to fentanyl, with a large share also exposed to fentanyl contaminated with xylazine.
Dr. Sandra Comer, Principal Investigator and Professor of Neurobiology at Columbia University, said the findings highlight the drug’s potential in treating opioid withdrawal. “The results demonstrate the potential of BXCL501 for reducing opioid withdrawal symptoms.”
BXCL501 is an investigational orally dissolving film formulation of dexmedetomidine. The company estimates that approximately 5.9 million people in the United States have been diagnosed with opioid use disorder.
BioXcel Therapeutics supplied the BXCL501 drug used in the NIDA-sponsored study.
Separately, the company continues to advance regulatory efforts for its drug IGALMI. BioXcel recently submitted a supplemental New Drug Application to the U.S. Food and Drug Administration seeking approval for the therapy to be used in at-home settings to treat acute agitation linked to bipolar disorder or schizophrenia.
IGALMI is currently approved for use under healthcare provider supervision. If the application is approved, the expanded indication allowing at-home treatment could be granted later in 2026.
BioXcel has also introduced milestone and retention bonus agreements for key executives following the filing, aiming to maintain leadership continuity during the regulatory review process as the company pursues broader use of the treatment.