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PRODUCT PIPELINE

BioXcel Therapeutics’ two most advanced clinical development programs are BXCL501, a proprietary, sublingual film formulation of dexmedetomidine being developed for the acute treatment of agitation associated with Alzheimer’s disease and as an adjunctive treatment for Major Depressive Disorder, 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 checkpoint inhibitors. BXCL501 has been granted Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia.

Our Clinical Pipeline

Neuroscience

BXCL501

Acute treatment of agitation associated with Alzheimer’s disease*

Pivotal Phase 3 Program Initiated
Major Depressive Disorder (MDD)
Ph1b/2 Trial Planned

KalmPenTM (Single-use IM)

Severe acute agitation
Formulation Development

BXCL502

Chronic agitation in dementia
Formulation Development

Wearable Device (+BXCL501)**

Pre & post-agitation in dementia
Feasibility Study Planned

Immuno-oncology

BXCL701

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®)

The safety and efficacy of investigational agents and/or investigational uses of approved products have not been established

*Includes intermittent chronic

**Regulatory path to be determined; device + drug combination to be evaluated after further evaluation of predictive algorithm

Pipeline as of April 5, 2022

BXCL501

BXCL501 is our most advanced neuroscience clinical program. BXCL501 is a proprietary, sublingual film formulation of dexmedetomidine, a selective alpha-2 receptor agonist. It is being investigated as an acute treatment for agitation in Alzheimer’s disease, and as an adjunctive treatment for major depressive disorder.

BXCL501 has received Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia. In 2021, the Company 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 initiated our pivotal phase 3 program of BXCL501 for acute treatment of agitation in patients with Alzheimer’s disease. The Company has also submitted an Investigational New Drug (IND) application to evaluate BXCL501 as adjunctive treatment for major depressive disorder (MDD).

BXCL701

pipeline-BXCL701-banner
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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