Revolutionizing Drug Development and Translation Research

Creating a paradigm shift in drug discovery for better & longer lives

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Rapid Human PoC and Development Path Focused on neuroscience and immuno-oncology

Program Product Candidate Phase 1/2 Phase 2/3 Anticipated Milestones Worldwide Rights
Treatment of Acute Agitation BXCL501
(Selective α2a Adrenergic Receptor Agonist)

Geriatric Dementia
  • BA study initiated with BXCL501 (4Q 2018)
  • BA study data readout (2Q 2019)
  • Launch registration trials (2019)

Immuno-oncology BXCL701
(DPP 8/9 & FAP Inhibitor)
Neuroendocrine Prostate Cancer

Pancreatic Cancer
  • Initiated tNEPC phase 1b/2 trial(4Q 2018)
  • Initiate pancreatic trials(1H 2019)
  • Preliminary tNEPC readout(2H 2019)
  • Preliminary pancreatic readouts(2H 2019)

Pipeline Expansion BXCL501
Opioid Withdrawal, Delirium

Exploring Multiple Tumor Types
  • New indications & geography expansion (2019)

Future Programs
Additional Discovery Through an Exclusive AI Relationship with BioXcel Corporation (parent)



Clinical Pipeline

BXCL501 Treating the Symptoms of Neuropsychiatric and Neurodegenerative Diseases
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BXCL701 Revolutionizing Drug Development and Translation Research in Immuno-Oncology (IO)
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Emerging Pipeline

BXCL702BXCL702 is a first-in-class, intravenous compound that inhibits key negative regulators of pro-inflammatory cytokine receptors, one of the molecular causes for immune-cell exhaustion. Combining BXCL702 with immune checkpoint inhibition will optimize T cell anti-tumor activity by limiting immune-exhaustion. In addition, amplification and overexpression of BXCL702 targets were found in AML cells. BXCL702 has shown direct anti-tumor activity against AML patient derived cell and cell lines by inducing cell growth arrest and differentiation.

BTI plans to develop BXCL702 in hematological malignancies with a focus on AML as mono therapy and in combination with immune-checkpoint inhibitors.


BioXcel’s Neuroscience programs are focused on discovering and developing therapeutics for neurological disorders with high unmet medical need. These include developing disease modifying agents as well as those agents that address symptoms such as agitation that represent significant unmet need in treating neuropsychiatric and neurodegenerative disorders.

BXCL501 is our most advanced Neuroscience program, which is being developed initially for the treatment of acute agitation in patients with schizophrenia and bipolar disorders. It is a selective adrenergic agent with a sublingual route of administration, which provides a fast onset of action doesn’t produce excessive sedation and is free from any associated side effects. It is well differentiated from routinely used antipsychotics which produce unwanted symptoms such as tremors, neutropenia and extra-pyramidal motor effects. Managing patient agitation in neuropsychiatric and neurodegenerative disorders represents a significant challenge for physicians and caregivers, and BXCL501 has the potential to provide an efficient treatment regimen for these patients.

Treating the Symptoms of Neuropsychiatric and Neurodegenerative Diseases


BXCL701 is a first-in-class, clinically validated, oral, small molecule that targets the next wave of immune checkpoints, as recently shown by Okondo et al (Nature Chemical Biology Nov 2016). It represents a novel, and highly attractive approach to the modulation of the tumor microenvironment needed to switch ‘‘cold’’ tumors into ‘‘hot’’ immune-permissive tumors. BXCL701 has demonstrated the modulation of relevant cytokine and immune cells, as well as single agent efficacy in immune-responsive tumors like melanoma in humans.

BXCL701 has shown synergistic anti-tumor activity in-vivo with anti-PD1, fully supported by mechanistic explanation at the molecular level: synergistic induction of anti-tumor cytokines, and at the cellular level: synergistic activation of T-cell and NK-cells and inhibition of immunosuppressive cells like MDSC and CAF.

BXCL701’s molecular targets are over-expressed and amplified in castration resistant prostate cancer with neuroendocrine phenotype, progressing after Zytiga and Xtandi, a much more aggressive tumor that presently has no treatment. BXCL701's molecular signature appears to overlap with amplification and overexpression of PDL1, which opens a unique competitive development approach in combination with immune checkpoint inhibitors.

BTI plans to develop BXCL701 in this segment of prostate cancer as a mono therapy and in combination with immune-checkpoint inhibitors.

There are many papers describing the role of FAP+ CAF in immune suppression in pancreatic cancer, and BXCL701 has already received orphan drug designation in pancreatic cancer. BTI is planning to collaborate with academic investigators to initiate a study in pancreatic cancer to exploit this unique mechanism.

Revolutionizing drug development and translation research in Immuno-Oncology (IO)