Primary Endpoint of Phase 3 Trial Met: BXCL501 for Agitation Associated With Bipolar Disorders or Schizophrenia

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BioXcel Therapeutics today announced that the SERENITY At-Home pivotal phase 3 trial evaluating the safety of BXCL501, an acute treatment for agitation associated with bipolar disorders or schizophrenia in the at-home setting, met its primary endpoint.¹

Outside of the FDA-approved indication, dexmedetomidine (Igalmi) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, which is a selective alpha-2 adrenergic receptor agonist. The SERENITY data will form the basis of the sNDA submission for the label expansion of Igalmi in the at-home setting.

“The SERENITY At-Home results are transformative in our journey toward outpatient use of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia,” said Vimal Mehta, PhD, the CEO of BioXcel Therapeutics. “By meeting its primary endpoint, the SERENITY At-Home pivotal phase 3 trial reinforces BXCL501’s potential to be safely used at home like it is already used in the previously FDA approved institutional setting. The unmet medical need in the at-home setting is significant with no FDA approved treatments. We are committed to changing the treatment paradigm as we prepare for our planned sNDA submission intended to provide patients with access to IGALMI® in the home setting. We believe the total addressable market is significantly larger than previously reported.”

The SERENITY At-Home pivotal phase 3 trial is a double-blind, placebo-controlled 12-week trial designed to evaluate the safety of a 120 mcg dose of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting. BXCL501 achieved SERENITY At-Home’s primary endpoint of being well tolerated during treatment of agitation episodes in patients with bipolar disorders or schizophrenia in the at-home setting. There were approximately 246 participants enrolled, and 45% were diagnosed with bipolar disorders and 55% diagnosed with schizophrenia. Investigators collected data from 2628 agitation episodes in 215 participants and treated 2437 episodes in 208 participants. Of these participants, 168 (81%) completed the full 12-week trial. An average of 11.7 agitation episodes were recorded per treated participant. All participants were able to successfully self-administer BXCL501.

As to safety and tolerability profile, the 120 mcg dose of BXCL501 was well-tolerated in patients with episodes of agitation in the outpatient setting, meeting the primary objective. This tolerability outcome was observed across repeat dosing and through the duration of the 12-week trial. There were no discontinuations for tolerability in the BXCL501 arm. The adverse event profile was consistent with FDA-approved Igalmi’s profile. There were no treatment-related serious adverse events, syncopes, or falls reported, and no new or unexpected treatment emergent adverse events.

“The management of agitation associated with bipolar disorder and schizophrenia in the home setting is an important clinical challenge where we currently have few optimal options,” said John Krystal, MD, the Robert L. McNeil, Jr. Professor of Translational Research and chair of the Department of Psychiatry at Yale School of Medicine. “Timely intervention has the potential to mitigate patient distress, decrease emergency room visits, and enhance overall patient safety while reducing healthcare costs, and I am pleased that this promising data could pave the way for a potential first approval of BXCL501 for at-home treatment.”

Based on positive feedback from the FDA’s earlier this month,² BioXcel believes that the planned sNDA regulatory package for BXCL501 will be sufficient to support the sNDA submission, which is on track for the first quarter of 2026.

References

1. BioXcel Therapeutics announces SERENITY At-Home pivotal phase 3 safety trial met its primary endpoint in support of sNDA submission for label expansion of IGALMI®. News release. August 27, 2025. Accessed August 27, 2025. https://www.globenewswire.com/news-release/2025/08/27/3139874/0/en/BioXcel-Therapeutics-Announces-SERENITY-At-Home-Pivotal-Phase-3-Safety-Trial-Met-its-Primary-Endpoint-in-Support-of-sNDA-Submission-for-Label-Expansion-of-IGALMI.html

2. Kuntz L. BXCL501 for agitation associated with bipolar disorders or schizophrenia: positive Pre-sNDA meeting with FDA. Psychiatric Times. August 18, 2025. https://www.psychiatrictimes.com/view/bxcl501-for-agitation-associated-with-bipolar-disorders-or-schizophrenia-positive-pre-snda-meeting-with-fda