BioXcel Therapeutics Provides Update on Recent Developments for Late-Stage Clinical Programs & Expansion of IP Portfolio for Sublingual Film

BioXcel Therapeutics, Inc. recently provided an update on recent developments with its late-stage clinical programs as well as its patent portfolio for IGALMI (dexmedetomidine) sublingual film. These developments include meetings scheduled with the US FDA to discuss the TRANQUILITY and SERENITY III clinical programs and the receipt of two Notices of Allowance (NOAs) from the US Patent and Trademark Office to extend method of use patent protection for sublingual dexmedetomidine.

“We believe that we have a substantial body of clinical, non-clinical, and pharmacokinetic (PK) data to discuss with the FDA with the goal of gaining alignment on a potential package required for supplemental new drug application (sNDA) submissions,” said Vimal Mehta, PhD, CEO of BioXcel Therapeutics. “The meetings with the FDA are critical components of our business strategy. We plan to seek alignment on a path for the potential approval to expand the market for BXCL501 for the at-home and assisted living facility treatment of mild to moderate agitation associated with dementia due to Alzheimer’s disease, and for the at-home acute treatment of agitation associated with bipolar I and II disorders and schizophrenia. Expanding BXCL501 into these large, underserved markets, while advancing IGALMI commercialization, are our top priorities.”

The company plans to review its TRANQUILITY clinical trial program with the FDA and to discuss the data package required to support submission of an sNDA for the approval of BXC501 for the acute treatment of agitation in mild to moderate dementia patients with probable Alzheimer’s disease in assisted living facilities and at-home settings.

The briefing book submitted to FDA for the meeting includes results from 11 double-blind, placebo-controlled Phase 2 and 3 clinical trials evaluating the safety and efficacy of BXCL501. Trials with BXCL501 have enrolled more than 1,100 patients across multiple neuropsychiatric conditions and in healthy volunteers, and have shown no unexpected safety signals, no reports of serious adverse events or falls related to the study drug, and no drug-related deaths, which is all consistent with the known pharmacological effects of BXCL501.

Of the subjects who received various doses of BXCL501, 273 were over 60 years of age, and 204 were over 65 years of age. The TRANQUILITY I and TRANQUILITY II trials were placebo-controlled, showed statistically significant separation from placebo in Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at two hours with the 60 mcg dose (primary endpoint), and had no unexpected safety findings. Data from TRANQUILITY I led the FDA to grant BXCL501 Breakthrough Therapy designation for the acute treatment of agitation associated with dementia. Additionally, the efficacy results seen in TRANQUILITY II after 12 weeks for all treated episodes was comparable to those observed after the first dose. Moreover, the adverse events (AEs) observed after the first dose were similar to the AEs observed after all doses in TRANQUILITY II (443 episodes treated) and comparable to the AEs observed in the single-dose TRANQUILITY I trial (100 episodes treated).

The company is continuing its previously disclosed investigation into protocol adherence and data integrity at a principal investigator’s trial site in connection with the TRANQUILITY II trial, and an independent third party is auditing the data collected at that site. For additional information regarding the TRANQUILITY II Phase 3 trial and related investigation and audit, see the company’s Current Report on Form 8-K filed with the Securities and Exchange Commission today October 4, 2023, which should be read in conjunction with this press release.

The company will review its SERENITY III program with the FDA on November 8, 2023 and plans to discuss the data package required to support submission of an sNDA seeking approval of BXCL501 for the acute treatment of agitation associated with bipolar disorders I and II and schizophrenia in the at-home setting. In addition, the company plans to discuss the evaluation of the 80 mcg dose of BXCL501 and several potential protocol amendments to the SERENITY III Part 2 trial. The company identified the 80 mcg dose as more favorable for further development based on pharmacokinetic-pharmacodynamic (PK-PD) modeling anchored by extensive data from studies that evaluated the 60 mcg dose of BXCL501 (half of the lower approved IGALMI dose) as well as studies that evaluated the 120 mcg and 180 mcg approved doses of IGALMI.

The primary objective of Part 2 of SERENITY III is to assess safety (the incidence of treatment-emergent adverse events), and the secondary objectives include various efficacy assessments.

BioXcel Therapeutics recently received two NOAs from the USPTO for patent applications related to the method of use of sublingual dexmedetomidine for the treatment of agitation associated with bipolar disorders and schizophrenia. When these patents are granted, the company plans to list them in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the Orange Book) for IGALMI.  The company now has four US patents claiming formulations of dexmedetomidine with exclusivity until 2039 currently listed in the Orange Book. The company expects that these two new patents will expire no earlier than Dec. 29, 2037, subject to the patent term adjustment, patent term extension, and terminal disclaimers. These patents further broaden the scope of intellectual property estate for IGALMI and for future potential indications.

IGALMI (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. The safety and effectiveness of IGALMI has not been studied beyond 24 hours from the first dose. It is not known if IGALMI is safe and effective in children.

BioXcel Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. The company’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 indications.

The company’s commercial product, IGALMI (dexmedetomidine) sublingual film (developed as BXCL501), is approved for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. The safety and effectiveness of IGALMI have not been established beyond 24 hours from the first dose. For more information, visit igalmi.com.

BXCL501 is under evaluation for at-home use for the acute treatment of agitation associated with Alzheimer’s dementia and for the acute treatment of agitation associated with bipolar disorders or schizophrenia. The safety and efficacy of BXCL501 for these uses have not been established. The company is also developing BXCL502 as a potential therapy for chronic agitation in dementia. The safety and efficacy of BXCL502 have not been established in any indication. For more information, visit bioxceltherapeutics.com.