BioXcel’s BXCL501 meets safety endpoint for at-home agitation treatment

Published 27/08/2025, 12:06
BioXcel’s BXCL501 meets safety endpoint for at-home agitation treatment

NEW HAVEN, Conn. - BioXcel Therapeutics, Inc. (NASDAQ:BTAI), whose stock has shown remarkable strength with a 203% surge over the past six months according to InvestingPro data, announced Wednesday that its SERENITY At-Home Phase 3 trial evaluating BXCL501 for the at-home treatment of agitation in patients with bipolar disorders or schizophrenia has met its primary safety endpoint.

The trial demonstrated that the 120 mcg dose of BXCL501, a sublingual film formulation of dexmedetomidine, was well-tolerated with no discontinuations due to tolerability issues in the treatment arm. The drug maintained a consistent safety profile across repeated doses throughout the 12-week study period. InvestingPro analysis reveals that two analysts have recently revised their earnings expectations upward for the upcoming period, suggesting growing confidence in the company’s prospects.

The company plans to submit a supplemental New Drug Application (sNDA) in the first quarter of 2026 to expand the use of IGALMI, the FDA-approved brand name for BXCL501, to the outpatient setting without healthcare provider supervision.

"The SERENITY At-Home results are transformative in our journey toward outpatient use of BXCL501," said Vimal Mehta, CEO of BioXcel Therapeutics, in a press release statement.

The double-blind, placebo-controlled trial involved 246 randomized patients who recorded over 2,600 agitation episodes, with more than 2,400 episodes treated across 208 patients. The study population consisted of 45% patients with bipolar disorders and 55% with schizophrenia.

While efficacy was not the primary objective, preliminary exploratory data indicated that BXCL501 reduced agitation symptoms throughout the trial, with a greater percentage of patients experiencing full resolution of symptoms compared to placebo.

IGALMI is currently approved for acute treatment of agitation associated with bipolar I or II disorder or schizophrenia, but only in medically supervised settings. If approved for at-home use, it would be the first FDA-approved treatment for agitation in the outpatient setting.

Based on data from the trial, BioXcel believes the potential market opportunity may be significantly larger than previously estimated, suggesting patients experience between 57 million and 77 million agitation episodes annually in the United States. With a current market capitalization of approximately $76 million and trading at a high revenue multiple, InvestingPro data indicates the company faces near-term challenges with short-term obligations exceeding liquid assets. For comprehensive analysis of BioXcel’s financial health and growth prospects, investors can access the detailed Pro Research Report, available exclusively to InvestingPro subscribers.

In other recent news, BioXcel Therapeutics has announced several key developments. The company is set to host an investor call to present topline data from its SERENITY At-Home Phase 3 trial, which evaluates the safety of BXCL501 for treating agitation in bipolar disorder and schizophrenia patients at home. The database lock for this trial has been completed, with data collected from over 2,600 episodes across 200 patients at 22 clinical sites. Additionally, BioXcel filed a prospectus supplement for an $80 million at-the-market equity offering program, with Canaccord Genuity designated as the sales agent. H.C. Wainwright has raised its price target for BioXcel Therapeutics from $8 to $10, maintaining a Buy rating in light of the upcoming clinical trial data. The firm highlighted the completion of the last patient visit in the pivotal Phase 3 trial, with top-line results expected soon. Furthermore, BioXcel received positive feedback from the FDA regarding the potential expansion of BXCL501 for at-home use, supporting the company’s planned supplemental New Drug Application submission in early 2026.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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