BioXcel’s Phase 3 trial of BXCL501 continues as advised by DSMB

NEW HAVEN, Conn. - BioXcel Therapeutics, Inc. (NASDAQ:BTAI), a biopharmaceutical company focused on neuroscience drug development with a current market capitalization of $8.42 million, announced today that the independent Data Safety Monitoring Board (DSMB) has recommended the continuation of its SERENITY At-Home Phase 3 safety trial for BXCL501 without any modifications. According to InvestingPro analysis, the company currently shows weak financial health with a score of 1.44 out of 5, reflecting challenges in its development stage operations. This pivotal trial is investigating the efficacy of BXCL501, a sublingual film, for the acute treatment of agitation in patients with bipolar disorders or schizophrenia in a home setting.

The DSMB’s endorsement comes after a thorough review of unblinded safety data from the initial 115 patients who were dosed by May 2, 2025. The trial has reached full enrollment, with over 205 patients having received the treatment, and more than 150 of those patients have been administered multiple doses during the 12-week trial period. With trailing twelve-month revenue of $1.85 million and negative gross margins of -12.15%, the success of this trial could be crucial for the company’s financial trajectory. For deeper insights into BioXcel’s financial health and growth prospects, InvestingPro subscribers have access to over 30 additional financial metrics and expert analysis.

BioXcel Therapeutics’ CEO, Vimal Mehta, Ph.D., expressed optimism regarding the DSMB’s favorable decision and looks forward to the upcoming data readout. The company anticipates that the results could support a supplemental New Drug Application (sNDA) for expanding the label of IGALMI®, its FDA-approved medication for agitation associated with bipolar I or II disorder or schizophrenia, to include at-home treatment.

The SERENITY At-Home trial is designed as a double-blind, placebo-controlled study that assesses the safety of a 120 mcg dose of BXCL501, an investigational proprietary orally dissolving film of dexmedetomidine. While IGALMI® is already approved for acute treatment under medical supervision, BXCL501 aims to meet the significant unmet need for FDA-approved therapies for bipolar or schizophrenia-related agitation in an at-home environment.

Topline data from the trial are expected to be released in Q3 2025. BXCL501 has received both Breakthrough Therapy designation and Fast Track designation from the FDA for the acute treatment of agitation associated with dementia, schizophrenia, bipolar disorders, and dementia, though its safety and efficacy for these investigational uses have not been established.

This report is based on a press release statement from BioXcel Therapeutics. The company continues to leverage artificial intelligence in its drug re-innovation approach, aiming to transform the treatment landscape in neuroscience and immuno-oncology through its subsidiary, OnkosXcel Therapeutics. Despite the stock’s significant decline of over 95% in the past year, analyst price targets range from $16 to $80, suggesting potential upside opportunities. Get comprehensive analysis and Fair Value estimates for BTAI and 1,400+ other stocks with InvestingPro’s detailed research reports.

In other recent news, BioXcel Therapeutics, Inc. has made several noteworthy announcements. The company reported a successful equity financing round, raising $14 million and bringing its cash reserves to approximately $35 million. This capital is allocated for the ongoing Phase 3 SERENITY At-Home trial, which evaluates the safety of their investigational drug, BXCL501, for home use in treating agitation related to bipolar disorders or schizophrenia. Additionally, BioXcel has reached a milestone by enrolling 100 patients in this trial, with topline data expected in the second half of 2025.

In regulatory developments, BioXcel Therapeutics has been granted an extension by Nasdaq to regain compliance with the Minimum Value of Listed Securities Rule, with a new deadline set for September 16, 2025. This follows a previous notification from Nasdaq regarding potential delisting due to non-compliance with the market value requirement. Furthermore, BioXcel has initiated an at-the-market equity offering program, allowing for the sale of up to $8,135,000 of its common stock through Canaccord Genuity LLC. This program aims to provide flexible financing options to support the company’s operations and growth initiatives.

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