BioXcel advances BXCL501 formulation development

NEW HAVEN, Conn.—BioXcel Therapeutics, Inc. (BTI) announced today that the company has finalized the formulation development for BXCL501, and has transitioned to automated manufacturing for its sublingual thin film containing dexmedetomidine (Dex). The manufacturing process scale-up is on track to support the company’s Phase 3 clinical trial that is expected to be initiated in the second half of 2019.

BXCL501 is a sublingual thin film with dexmedetomidine, a selective alpha 2a receptor agonist for the treatment of acute agitation. BTI believes that BXCL501 directly targets a causal agitation mechanism, and using IV (intravenous) Dex has demonstrated anti-agitation effects in both preclinical and clinical studies.

BTI is currently assessing the intended commercial formulation of BXCL501 in a Phase 1 pharmacokinetic (bioavailability) and safety study of multiple dosing strengths, with data expected in the near term. BXCL501 has been designed for both robustness and stability. The production of the sublingual thin film has been transitioned to an automated, robot-aided platform that is scalable and provides flexibility to produce a wide range of dose strengths. The manufacturing process and proprietary film structure are designed to allow for dosing flexibility, and potential use in combination therapy.

“We believe that we have optimized BXCL501 thin film properties for acute treatment of agitation across multiple disease states. We are excited to have transitioned to automated manufacturing for scale-up and initiation of Phase 3 clinical study and commercial supply,” said Dr. David Hanley, vice president and head of Global Pharmaceutical Development and Operations, BTI.

The company has also recently appointed Dr. Pascal Borderies as vice president, Commercial Development and Medical Affairs, BTI. Borderies is responsible for designing and executing BTI’s global commercialization and medical affairs strategy, including sales and marketing. He has more than 25 years of experience in commercial development and medical affairs at companies, including Novartis Consumer Health and Schering Plough, among others. Borderies holds a medical degree from the Paris 12 Val de Marne University School of Medicine.

Noted Borderies, “I am thrilled to be joining the BioXcel Therapeutics leadership team to spearhead the company’s commercialization and medical affairs initiatives. The BTI team has made remarkable progress in developing an innovative drug portfolio. I look forward to supporting the team to maximize the potential of the entire pipeline and bringing these therapies to the market.”

BTI has completed dosing subjects in a Phase 1 placebo-controlled, single dose, dose-escalation study of BXCL501. The clinical trial data is currently undergoing analyses, with topline results expected to be announced shortly. The results are anticipated to encompass pharmacokinetics, safety, tolerability and therapeutic exposures. The company plans to initiate its first registration trial in the second half of 2019, based on findings from this study.

BTI also continues to explore a range of target indications for BXCL501 beyond its current focus areas of acute treatment of agitation in schizophrenia, bipolar disorder and Alzheimer’s disease. Treatment of agitation remains a significant global healthcare challenge in patients suffering from opioid and alcohol withdrawal, delirium and post-traumatic stress disorder, as the currently available treatment options are suboptimal, invasive and difficult to administer, and often pose safety issues. The manufacturing process scale-up is on track to support BTI’s Phase 3 clinical trial of BXCL501 for acute agitation, which is expected to be initiated in the second half of 2019.

“We are pleased with the rapid advancement of the BXCL501 program as a potential non-invasive treatment of acute agitation in patients suffering from schizophrenia, bipolar disorder and Alzheimer’s disease,” said Dr. Vimal Mehta, chief executive officer of BTI. “We anticipate that the upcoming results from our Phase 1 pharmacokinetic (bioavailability) and safety study will help establish a solid foundation as we work toward our goal of initiating Phase 2/3 trials and filing BTI’s first New Drug Application with the FDA in 2020.”