BioXcel Therapeutics (BTIAI) received a planning grant as part of its Alcohol and Substance Abuse Disorders Research Program (ADADRP) related to the development of the company’s investigational candidate BXCL501. The grant will support the development of a clinical study to evaluate the use of BXCL501 for the treatment of ASUD, particularly related to PTSD and TBI. The study will be led by principal investigator John Krystal, MD, the Robert L. McNeil, Jr. Professor of Translational Research, the Chairman of the Yale Department of Psychiatry, the Chief of Psychiatry at Yale-New Haven Hospital, and the Director of Clinical Neurosciences, National Center for PTSD.
The company recently announced positive top-line results from its adaptive Phase 1b, randomized, double-blind, placebo-controlled, multi-center, U.S. trial evaluating multiple doses of BXCL501, an investigational selective and potent alpha2-adrenergic receptor agonist, for acute treatment of agitation in 135 patients with schizophrenia. Based on these results, the company is planning to initiate Phase 3 pivotal trials in Q4 2019. The results from this study of BXCL501 suggest that it may merit further evaluation in patients with ASUD correlated with PTSD and TBI, experiencing hyper-arousal and high sympathetic nervous system activity. BXCL501 may be evaluated for use as an adjunct therapeutic before, during or after exposure therapy (ET), a gold standard therapy for PTSD, for treatment of treatment of ASUD patients comorbid with PTSD or TBI.
What is BXCL501?
BTIAI’s lead candidate, a potential first-in-class, proprietary sublingual thin film of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of acute agitation. BioExcel believes that the investigative product directly targets a causal agitation mechanism and using IV (intravenous) Dex has shown anti-agitation effects in multiple clinical studies.
About Alcohol and Substance Use Disorder (ASUD) comorbid with Post-traumatic Stress Disorder (PTSD) or Traumatic Brain Injury (TBI):
The relationship between ASUD and PTSD is well documented, where up to one third of people who survive traumatic accidents or disasters report drinking problems. These accidents may include traumatic brain injury, which results in physical, cognitive, and emotional symptoms that overlap with PTSD. Suffering from both ASUD and PTSD can make both issues worse, thus treatment for alcohol and substance abuse must often be part of PTSD treatment. Pharmacotherapies for ASUD can have significant side effects, including but not limited to, nausea, vomiting, dizziness, and abdominal pain. Therapies for PTSD include exposure therapy which involves re-experiencing the traumatic events and such therapy has high dropout rates. These issues limit the willingness of patients with ASUD or PTSD to seek treatment and limit compliance with treatment regiments. PTSD is a condition that is caused by either experiencing or witnessing a traumatic event, such as military combat, vehicle accidents, assault, abuse, natural disasters, and others. It is characterized by flashbacks, nightmares, severe anxiety, and also uncontrollable thoughts about the event. TBI is a form of acquired brain injury, which occurs when a sudden trauma causes damage to the brain either externally or internally.
Dr. Krystal is the Robert L. McNeil, Jr. Professor of Translational Research and Professor of Psychiatry, Psychology and Neuroscience; Chair of Department of Psychiatry; and Chief of Psychiatry at the Yale-New Haven Hospital. He also serves as the Director of National Alcohol Abuse and Alcoholism Advisory Council Center for the Translational Neuroscience of Alcoholism; Director, Clinical Neuroscience Division, VA National Center for PTSD. He is a leading expert in the areas of alcoholism, post-traumatic stress disorder, schizophrenia, and depression and has been crucial in the discovery of the rapid antidepressant effects of ketamine in depressed patients. Dr. Krystal is a clinical advisor to the Company.
About BioXcel Therapeutics, Inc.:
BioXcel Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on drug development that utilizes novel artificial intelligence approaches to identify and advance the next wave of medicines in neuroscience and immuno-oncology. BTI’s drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine-learning algorithms to identify new therapeutic indices. BTI’s two most advanced clinical development programs are BXCL501, an investigational sublingual thin film formulation in development for acute treatment of agitation resulting from neuropsychiatric disorders, and BXCL701, an investigational, orally administered, systemic, innate immunity activator in development for treatment of a rare form of prostate cancer and for treatment of pancreatic cancer in combination with other immuno-oncology agents. For more information, please visit www.bioxceltherapeutics.com.