BioXcel’s two most advanced clinical development programs are BXCL501, an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine designed for the treatment of agitation and opioid withdrawal symptoms, and BXCL701, an investigational, orally-administered, systemic innate immunity activator for the treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to check point inhibitors.

Our Clinical Pipeline



Acute agitation in schizophrenia/bipolar
SERENITY I & II Trials Completed (PDUFA date – 4/5/22)
Acute agitation in dementia*
BTD Phase 3 Program Initiation planned
Major Depressive Disorder (MDD)
Ph1b/2 Trial Planned

KalmPenTM (Single-use IM)

Severe acute agitation
Formulation Development


Chronic agitation in dementia
Formulation Development

Wearable Device (+BXCL501)**

Pre & post-agitation in dementia
Feasibility Study Planned



Metastatic castration-resistant prostate cancer
(small cell neuroendocrine carcinoma and adenocarcinoma)
Phase 1b/2 (Combination with KEYTRUDA®)
Basket trial – hot & CPI resistant tumors (investigator-initiated study led by MD Anderson Cancer Center)
Phase 2 (Combination with KEYTRUDA®)
*Includes intermittent chronic
**Regulatory path to be determined; device + drug combination to be evaluated after further evaluation of predictive algorithm
Opioid withdrawal symptoms with BXCL501 pending NIDA grant decision
Agitation in delirium study with BXCL501 is on voluntary pause
Pipeline as of December 1, 2021


BXCL501 is an investigational, proprietary, orally dissolving, thin film formulation of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of agitation and opioid withdrawal symptoms.

BXCL501 is our most advanced neuroscience clinical program, currently being developed for the treatment of agitation associated with schizophrenia, bipolar disorders, dementia, depression and delirium and opioid withdrawal symptoms. As a selective adrenergic agent with a sublingual or buccal route of administration, BXCL501 is designed to be easy to administer and has demonstrated a rapid onset of action in clinical studies. BXCL501 is also designed to reduce agitation without producing excessive sedation or unarousable sleep. Managing a patient’s agitation or withdrawal symptoms represents a significant challenge for physicians and caregivers. BXCL501 has received Breakthrough Therapy designation for agitation associated with dementia and Fast Track designation by the FDA for agitation associated with schizophrenia, bipolar disorders and dementia.

The FDA has accepted for filing the New Drug Application for BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar I and II disorders, and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 5, 2022. The application is supported by data from the SERENITY I and II pivotal trials.

In early 2021, we announced positive topline results from the TRANQUILITY Phase 1b/2 trial for the acute treatment of agitation associated with dementia, including Alzheimer’s disease. Following discussions with the FDA in Q4 2021, we plan on advancing BXCL501 into a late-stage clinical development program for the acute treatment of agitation associated with dementia  . The Company also reported results from its RELEASE Phase 1b/2 trial for the treatment of opioid withdrawal symptoms.


BXCL701 (talabostat)is an investigational, oral small molecule inhibitor of dipeptidyl peptidases (DPP)—primarily DPP 8/9 and DPP 4—which triggers inflammasome to alert and prime immune cells, leading to induction of IL-18 and IL-1ß, bridging innate & adaptive immunity. BXCL701 is currently being developed for the treatment of aggressive forms of prostate cancer and advanced solid ”hot” tumors that are refractory or treatment naïve to checkpoint inhibitors.

BXCL701 currently has two ongoing combination therapy clinical trials. The Company is conducting a Phase 1b/2 trial of BXCL701 in combination with KEYTRUDA® (pembrolizumab) for metastatic castration-resistant prostate cancer (mCRPC), in patients with adenocarcinoma and in patients with the aggressive variant small-cell neuroendocrine carcinoma.. The trial will now continue to full enrollment and more complete efficacy data are expected to be presented at a scientific conference early next year.

The MD Anderson-led Phase 2 open-label basket trial was designed to evaluate the response rate of orallyadministered BXCL701, combined with KEYTRUDA, in two arms. Arm A is enrolling checkpoint naïve patients where checkpoint therapy is indicated (also referred to as “hot tumors”); and Arm B is enrolling patients who have progressed following checkpoint therapy alone. The Stage 1 efficacy bar was met for the checkpoint naïve patients, allowing Arm A of the trial to advance to completion. The Company plans to report an efficacy update from the trial in mid-2022.

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