This section highlights FDA-related milestones and regulatory updates for drugs developed by BioXcel Therapeutics (BTAI).
Over the past two years, BioXcel Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
BXCL501, BXCL501, BXCL501, BXCL701, and IGALMI™. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
BXCL501 (dexmedetomidine) - FDA Regulatory Timeline and Events
BXCL501 (dexmedetomidine) is a drug developed by BioXcel Therapeutics for the following indication: For dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BXCL501 (dexmedetomidine)
- Announced Date:
- September 19, 2024
- Indication:
- For dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist
Announcement
BioXcel Therapeutics, announced its clinical prioritization and an update on its late-stage development programs for the treatment of agitation with BXCL501.
AI Summary
BioXcel Therapeutics has announced a new clinical prioritization plan for BXCL501, its investigational treatment for agitation. The company is focusing on advancing its late-stage development programs to treat agitation associated with bipolar disorders, schizophrenia, and Alzheimer’s dementia. Central to this plan is the SERENITY At-Home pivotal Phase 3 trial, which has been initiated with 200 patients. This trial is designed to evaluate the safety of a 120 mcg dose of BXCL501 in an at-home setting, targeting agitation in patients with bipolar disorders or schizophrenia. Additionally, BioXcel submitted the protocol for the TRANQUILITY In-Care pivotal Phase 3 trial to the FDA. This trial will assess both the efficacy and safety of a 60 mcg dose for agitation related to Alzheimer’s dementia. The company believes that concentrating resources on these programs opens attractive growth opportunities in areas with significant patient needs.
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BXCL501 (SERENITY) - FDA Regulatory Timeline and Events
BXCL501 (SERENITY) is a drug developed by BioXcel Therapeutics for the following indication: Schizophrenia and bipolar disorders.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- July 1, 2025
- Estimated Event Date Range:
- July 1, 2025 - September 30, 2025
- Target Action Date:
- Q3 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, Inc announced that Topline data expected in Q3 2025.
AI Summary
BioXcel Therapeutics, Inc. announced that an independent Data Safety Monitoring Board (DSMB) has recommended continuing its SERENITY At-Home pivotal Phase 3 safety trial for BXCL501 without any changes. The review, based on unblinded safety data from 178 patients, supports the ongoing study of BXCL501 for the acute treatment of agitation in patients with bipolar disorders or schizophrenia in an at-home setting.
The trial, now fully enrolled and running over a 12-week period, is moving forward with promising safety observations. BioXcel expects to release the topline data in the third quarter of 2025, which will offer important insights into the safety and effectiveness of BXCL501. The company is focused on addressing an unmet medical need, as there are currently no FDA-approved therapies available for at-home treatment of agitation related to these conditions.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- July 1, 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics announced the second positive recommendation by an independent Data Safety Monitoring Board (DSMB) to continue, without modification, the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for acute treatment of agitation associated with bipolar disorders or schizophrenia.
AI Summary
BioXcel Therapeutics announced a second positive recommendation from an independent Data Safety Monitoring Board (DSMB) for its SERENITY At-Home Phase 3 trial. The DSMB reviewed unblinded safety data from 178 patients and advised that the study continue as planned without any changes. The trial is evaluating the safety of a 120 mcg dose of BXCL501 for the acute treatment of agitation in patients with bipolar disorders or schizophrenia, a significant unmet need, especially for at-home care. The study is double-blind and placebo-controlled, and it is fully enrolled with 200 patients participating over a 12-week period. BioXcel looks forward to the upcoming data readout, with topline results expected in the third quarter of 2025, as the company works to support a potential sNDA submission for expanding the at-home treatment option for agitation.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- July 1, 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, Inc. announced the second positive recommendation by an independent Data Safety Monitoring Board (DSMB) to continue, without modification, the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for acute treatment of agitation associated with bipolar disorders or schizophrenia.
AI Summary
BioXcel Therapeutics, Inc. announced that an independent Data Safety Monitoring Board (DSMB) has given a second positive recommendation to continue the SERENITY At-Home pivotal Phase 3 safety trial for BXCL501. This trial is focused on using BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia, and it will proceed without any modifications. The DSMB’s decision followed a review of unblinded safety data from 178 patients, confirming that the trial can safely continue. The study is fully enrolled, and data collection over a 12‑week period is ongoing, with topline results expected in the third quarter of 2025.
This favorable recommendation emphasizes the potential of BXCL501 to address an important, unmet need for patients experiencing agitation at home, and it supports the company’s efforts towards developing effective at‐home treatment options for bipolar disorders or schizophrenia.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- May 27, 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, announced that an independent Data Safety Monitoring Board (DSMB) recommended that the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for acute treatment of agitation associated with bipolar disorders or schizophrenia continue without modification.
AI Summary
BioXcel Therapeutics announced that an independent Data Safety Monitoring Board (DSMB) recommended the SERENITY At-Home Phase 3 safety trial of BXCL501 continue as planned. The decision came after the DSMB reviewed unblinded safety data from the first 115 patients dosed as of May 2, 2025. This fully enrolled, double-blind, placebo-controlled study focuses on the acute treatment of agitation in patients with bipolar disorders or schizophrenia, conducted in an at-home setting.
CEO Vimal Mehta expressed enthusiasm over the DSMB’s favorable outcome and looks forward to the topline data expected in the third quarter of 2025. This data is seen as a key step toward a potential sNDA submission for expanding the label of IGALMI®, addressing an important unmet need in providing effective, at-home treatments for agitation.
Read Announcement - Drug:
- BXCL501 (SERENITY)
- Announced Date:
- March 27, 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, provided a business update.
AI Summary
BioXcel Therapeutics provided an encouraging business update, highlighting progress in its SERENITY At-Home pivotal Phase 3 trial. The study, designed to evaluate the safety of a 120 mcg dose of BXCL501 for acute treatment of agitation in patients with bipolar disorders or schizophrenia, has enrolled 127 out of 200 required patients, reaching about 63% of its target. Topline data from the trial is anticipated in the second half of 2025 and may support a supplemental NDA submission for IGALMI® label expansion in the at-home setting.
Additionally, the company announced a strengthened cash position following a $14 million equity financing, which now brings cash on hand to approximately $35 million. CEO Vimal Mehta highlighted the trial’s potential to address an unmet need, given the significant number of agitation episodes observed annually in the U.S., reinforcing the company’s commitment to advancing its neuroscience portfolio.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- March 11, 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, announced that, following the successful raise of $14 million gross proceeds in an equity financing that closed on March 4, 2025, it has approximately $35 million in cash. The strengthened cash position will support continued advancement of the Company's pivotal Phase 3 SERENITY At-Home trial. T
AI Summary
BioXcel Therapeutics announced it secured $14 million in equity financing that closed on March 4, 2025. Following this financing round, the company now has approximately $35 million in cash. This strengthened cash position is expected to support the advancement of its pivotal Phase 3 SERENITY At-Home trial. The trial is designed to study the safety of BXCL501, an investigational formulation of dexmedetomidine, for the acute treatment of agitation associated with bipolar disorders or schizophrenia in a home setting.
Topline data from the trial is anticipated in the second half of 2025. The company is hopeful that these results will support a potential supplemental New Drug Application (sNDA) to expand the label for IGALMI® in the at-home setting, potentially providing a new acute treatment option for patients suffering from agitation.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- March 7, 2025
- Estimated Event Date Range:
- July 1, 2025 - December 31, 2025
- Target Action Date:
- H2 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, announced that Topline data expected in second half of 2025
AI Summary
BioXcel Therapeutics announced that its SERENITY At-Home Phase 3 trial has reached 33% enrollment for evaluating BXCL501, a 120 mcg dose for the acute treatment of agitation in patients with bipolar disorders or schizophrenia in a home setting. With 23 clinical trial sites open, the study is focused on collecting safety data during a 12-week period. The company expects to share topline results in the second half of 2025. These results are intended to support a potential supplemental new drug application (sNDA) to expand the label of the FDA-approved IGALMI® sublingual film for treating agitation. BioXcel is working closely with its clinical research organization and principal investigators to optimize patient enrollment and ensure comprehensive data collection from the trial.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- March 7, 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, announced it has reached 33% enrollment in its 200-patient SERENITY At-Home trial.
AI Summary
BioXcel Therapeutics recently announced that it has reached 33% enrollment in its SERENITY At-Home trial, a pivotal Phase 3 study involving 200 patients. This trial is designed to evaluate the safety of BXCL501, an investigational orally dissolving film formulation containing dexmedetomidine, when used at home for the acute treatment of agitation in patients with bipolar disorder or schizophrenia.
The study focuses on testing the lowest approved dose of IGALMI® (120 mcg) with safety as the primary endpoint. With nearly all 23 clinical trial sites now open, the enrollment progress has reached 67 patients, and a Data Safety Monitoring Board is planned to assess safety data throughout the trial. Topline results are expected in the second half of 2025 and could support a supplemental new drug application for expanding the current label of IGALMI® for at-home use.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- February 5, 2025
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, today provided an update on the progress of its late-stage clinical programs for lead neuroscience asset BXCL501, as well as its recent steps to enhance operational and financial flexibility and strengthen its Board leadership.
AI Summary
BioXcel Therapeutics provided an update on its late-stage clinical programs for its lead neuroscience asset, BXCL501. The company shared that the SERENITY At-Home Phase 3 trial for the acute treatment of agitation associated with bipolar disorder or schizophrenia has made significant progress, with most trial sites now open and patient enrollment advancing well. In addition, BioXcel has developed plans for the TRANQUILITY In-Care Phase 3 trial, targeting agitation associated with Alzheimer’s dementia. To enhance operational and financial flexibility, the company amended its existing credit agreement and raised additional equity funding. Furthermore, BioXcel strengthened its leadership by appointing new board members with expertise in clinical, financial, and legal areas. These actions underline the company’s commitment to advancing critical treatment options and creating value for patients and all stakeholders.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- September 5, 2024
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, announced the initiation of patient enrollment in its SERENITY At-Home pivotal Phase 3 trial.
AI Summary
BioXcel Therapeutics has initiated patient enrollment in its SERENITY At-Home pivotal Phase 3 trial. This study will evaluate the safety of BXCL501, a proprietary, orally dissolving film formulation of dexmedetomidine, for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting.
The trial is designed as a double-blind, placebo-controlled study and plans to enroll about 200 patients. Each participant, or their caregiver, will self-administer a 120 mcg dose of BXCL501 or placebo when experiencing an episode of agitation during the 12-week trial period. With a total trial duration expected to be between 9 to 12 months, the study aims to collect robust safety data and assess the treatment’s potential to reduce emergency room visits, enhance patient safety, and lower overall treatment costs.
Read Announcement- Drug:
- BXCL501 (SERENITY)
- Announced Date:
- April 22, 2024
- Indication:
- Schizophrenia and bipolar disorders
Announcement
BioXcel Therapeutics, announced additional details regarding the planned design of its SERENITY At-Home Phase 3 trial*to evaluate BXCL501, the Company's investigational, proprietary, orally dissolving film formulation of dexmedetomidine, as a potential acute treatment for agitation associated with bipolar disorders or schizophrenia in the home setting.
AI Summary
BioXcel Therapeutics announced new details about its SERENITY At-Home Phase 3 trial, which will evaluate BXCL501. BXCL501 is an investigational, orally dissolving film formulation of dexmedetomidine that could be used as an acute treatment for agitation in patients with bipolar disorders or schizophrenia at home. The trial will use a 120 mcg dose based on recent feedback from the FDA and is designed as a double-blind, placebo-controlled study lasting 12 weeks. Approximately 200 patients will participate by self-administering the film when experiencing episodes of agitation. In addition, the company plans to enroll about 30 patients in a separate study to compare patient-reported outcomes with assessments made by trained raters. BioXcel aims to collect vital data that might help expand the use of BXCL501 from clinical settings to outpatient care, where most agitation episodes happen.
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BXCL501 (TRANQUILITY) - FDA Regulatory Timeline and Events
BXCL501 (TRANQUILITY) is a drug developed by BioXcel Therapeutics for the following indication: Agitation Associated with Dementia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BXCL501 (TRANQUILITY)
- Announced Date:
- March 3, 2025
- Indication:
- Agitation Associated with Dementia
Announcement
BioXcel Therapeutics announced that the U.S. Food and Drug Administration (FDA) has concluded that the inspection of a single site in its TRANQUILITY II Phase 3 trial is closed under 21 C.F.R.20.64(d)(3) and released the Establishment Inspection Report.
AI Summary
BioXcel Therapeutics announced that the FDA has concluded the inspection of one site in its TRANQUILITY II Phase 3 trial is closed under 21 C.F.R.20.64(d)(3). The agency released the Establishment Inspection Report, assigning the site a "Voluntary Action Indicated" status. This decision supports the company’s assertions regarding the integrity and reliability of clinical data collected during the trial.
The closure of the inspection comes as part of the continued efforts to ensure that clinical processes meet regulatory standards. BioXcel’s CEO, Vimal Mehta, emphasized that this positive evaluation, along with the strong data evidence from the trial site, reinforces the potential clinical benefits of their lead treatment candidate, BXCL501. The company views this development as an important step in advancing their TRANQUILITY II trial and related clinical programs.
Read Announcement- Drug:
- BXCL501 (TRANQUILITY)
- Announced Date:
- April 10, 2024
- Indication:
- Agitation Associated with Dementia
Announcement
BioXcel Therapeutics announced details regarding the planned design of its upcoming TRANQUILITY In-Care Phase 3 trial to evaluate BXCL501, the company's investigational proprietary, orally dissolving film formulation of dexmedetomidine, as a potential acute treatment for agitation associated with Alzheimer's dementia (AAD) in the care setting.
AI Summary
BioXcel Therapeutics announced the planned design of its TRANQUILITY In-Care Phase 3 trial to test BXCL501, an investigational, orally dissolving film formulation of dexmedetomidine. The study will focus on evaluating BXCL501 as an acute treatment for agitation associated with Alzheimer’s dementia in care settings. The trial is designed as a double-blind, placebo-controlled study, mirroring the positive efficacy and safety outcomes observed previously with a 60 mcg dose during the TRANQUILITY II trial.
The study aims to enroll approximately 150 patients aged 55 and older across different care facilities, including skilled nursing and memory care units. Patients will self-administer the film during episodes of agitation. The primary endpoint is the change in agitation scores two hours following the first dose, providing further insight into both the safety and effectiveness of BXCL501 for addressing agitation in Alzheimer’s dementia patients.
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BXCL701 + KEYTRUDA (pembrolizumab) - FDA Regulatory Timeline and Events
BXCL701 + KEYTRUDA (pembrolizumab) is a drug developed by BioXcel Therapeutics for the following indication: Neuroendocrine Prostate Cancer (NEPC).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BXCL701 + KEYTRUDA (pembrolizumab)
- Announced Date:
- April 24, 2024
- Indication:
- Neuroendocrine Prostate Cancer (NEPC)
Announcement
BioXcel Therapeutics announced that a late-breaking abstract with preliminary findings from the Phase 2 investigator-sponsored trial of BXCL701 in combination with KEYTRUDA® (pembrolizumab) in previously treated metastatic pancreatic ductal adenocarcinoma (PDAC) has been selected for presentation in a poster session at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. The meeting will take place from May 31 to June 4, 2024 in Chicago, IL.
AI Summary
BioXcel Therapeutics announced that a late-breaking abstract featuring preliminary findings from its Phase 2 investigator-sponsored trial of BXCL701 in combination with KEYTRUDA® (pembrolizumab) has been selected for presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. The trial, led by investigators at Georgetown University’s Lombardi Comprehensive Cancer Center, is exploring whether BXCL701—a novel oral innate immune activator—can help “heat up” normally “cold” tumors in patients with previously treated metastatic pancreatic ductal adenocarcinoma (PDAC). By inflaming the tumor microenvironment, the approach aims to enhance the effectiveness of checkpoint inhibitors such as KEYTRUDA® in generating a stronger anticancer immune response.
The ASCO meeting will take place from May 31 to June 4, 2024, in Chicago, Illinois. The dedicated poster session will highlight these promising early results and offer insights into new potential treatment strategies for aggressive pancreatic cancer.
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IGALMI™ - FDA Regulatory Timeline and Events
IGALMI™ is a drug developed by BioXcel Therapeutics for the following indication: Sublingual film.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- IGALMI™
- Announced Date:
- June 25, 2024
- Indication:
- Sublingual film
Announcement
BioXcel Therapeutics, announced positive topline results from a post-marketing requirement (PMR) study of IGALMI™ (dexmedetomidine) sublingual film that demonstrated no evidence of tachyphylaxis, tolerance, or withdrawal with the 180 mcg dose after seven days of PRN (as-needed) treatment.
AI Summary
BioXcel Therapeutics recently announced positive topline results from its post-marketing requirement study of IGALMI™ (dexmedetomidine) sublingual film. The study showed that a 180 mcg dose, used as needed over seven days, did not lead to tachyphylaxis, tolerance, or withdrawal effects. This finding is significant because it demonstrates that repeated doses remain effective without diminishing the drug’s ability to manage agitation.
The study involved adult patients experiencing episodes of agitation, and the results indicated a consistent reduction in agitation symptoms with each dose administered. Moreover, no serious adverse events were observed, which highlights IGALMI™’s favorable safety profile. These outcomes support the use of IGALMI™ in treating agitation and add valuable data for the ongoing Phase 3 SERENITY and TRANQUILITY trials, potentially paving the way for future updates to the product labeling.
Read Announcement- Drug:
- IGALMI™
- Announced Date:
- May 21, 2024
- Indication:
- Sublingual film
Announcement
BioXcel Therapeutics announced that Rob Risinger, M.D., Chief Medical Officer of Neuroscience, will deliver oral and poster presentations at the 2024 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting. The meeting will take place May 28 to 31, 2024 in Miami Beach, FL.
AI Summary
BioXcel Therapeutics announced that Rob Risinger, M.D., Chief Medical Officer of Neuroscience, will present both oral and poster presentations at the 2024 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting. This meeting is scheduled to be held from May 28 to 31, 2024, in Miami Beach, FL.
Dr. Risinger’s oral presentation, titled "An Exploratory Comparison of Sublingual Dexmedetomidine with Quetiapine in Healthy Elderly Subjects," is set for May 28 at Salon 3. Additionally, he will lead two poster presentations. The first, "Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of BXCL501 with Concomitant Treatment with Antidepressant in Healthy Volunteers," will be presented on May 29 at Salon 4. The second, focusing on a Phase Ib/II study in agitation associated with dementia, is scheduled on May 30 at the same location.
Read Announcement
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