On November 14, 2019 BioXcel Therapeutics, Inc. ("BTI" or "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, reported its quarterly results for the third quarter ended September 30, 2019 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, NOV 14, 2019, View Source [SID1234551242]).

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"We are pleased with the progress made on both of our clinical development programs during the quarter," stated Vimal Mehta, Chief Executive Officer of BTAI. "BXCL501, our candidate for the acute treatment of agitation in patients with schizophrenia and bipolar disease and delivered in a sublingual thin film, is highly differentiated from the current standards of care, which can produce unwanted side effects and be difficult for caregivers to administer. BXCL501 has the potential to significantly improve care for patients, while providing healthcare providers with an important new option for treating their patients. We’re looking forward to reporting pivotal data during the first half of 2020."

Dr. Mehta added, "We are also pleased with the progress of our Phase 1b/2 double combination study of BXCL701 and Keytruda for tNEPC and anticipate additional safety data readouts from both cohorts in the fourth quarter of this year, followed by expected initial efficacy data in the first half of 2020."

Third Quarter 2019 and Recent Highlights

BXCL501-Neuroscience Program-

BXCL501 is an investigational sublingual thin film of dexmedetomidine, a selective alpha-2A adrenergic receptor agonist, designed for the treatment of acute agitation. The Company believes BXCL501 may directly target a causal agitation mechanism.

·BXCL501 met its primary endpoint and demonstrated statistically significant mean reduction in PEC (PANSS, or the Positive and Negative Syndrome Scale, Excitatory Component) in the Phase 1b trial with agitated schizophrenia patients. Pivotal studies for the acute treatment of agitation in schizophrenia and bipolar patients are expected to initiate in Q4 2019, with data readouts expected 1H 2020;

The Phase 1b/2 study of BXCL501 for acute treatment of agitation in geriatric dementia/Alzheimer’s disease is expected to begin in Q4 2019, with data expected in 1H 2020;

· Initiated BXCL501 strategic initiative to investigate the feasibility of development of digital device technology, such as the Apple Watch, that can be used in conjunction with BXCL501 to enhance the prevention and treatment of agitation, specifically in geriatric dementia patients;

·Awarded grant by CDMRP to expand clinical development of BXCL501 program for the treatment of alcohol and substance abuse disorders related to PTSD in collaboration with Yale University.

BXCL701-Immuno-Oncology Program-

BXCL701 is an orally-delivered small molecule, innate immunity activator designed to inhibit dipeptidyl peptidase (DPP) 8/9 and block immune evasion by targeting Fibroblast Activation Protein (FAP). It has shown single agent activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients.

· The Phase 1b/2 trial of BXCL701 and Keytruda for tNEPC is ongoing. The Company presented safety and tolerability data from the first patient cohort at the Annual Prostate Cancer Foundation Scientific Retreat and is currently enrolling a second patient cohort. BTI expects to report additional safety findings by year-end before advancing to the Phase 2 stage of the trial;

·BXCL701 received third orphan drug designation (ODD) from the FDA for the treatment of AML. Potential to expand clinical development of BXCL701 program into hematological malignancies;

· The BXCL701 phase of the triple combination study of BXCL701, bempegaldesleukin (NKTR-214, Nektar Therapeutics, Inc.) and BAVENCIO (avelumab, Merck KGaA, Darmstadt, Germany and Pfizer) in pancreatic cancer is expected to be initiated following Nektar and Pfizer’s safety run-in trial of a double combination of bempegaldesleukin and avelumab and the outcome of that trial.

Strengthened Balance Sheet

·BioXcel raised gross proceeds of $19.0 million in the quarter, the net of which, together with current reserves, provides sufficient capital to fund operations through key data readouts including Phase 3 and Phase 1b/2 studies with BXCL501.

Third Quarter 2019 Financial Results

BTI reported a net loss of $9.0 million for the third quarter of 2019, compared to a net loss of $4.9 million for the same period in 2018. The third quarter 2019 results include approximately $0.8 million in non-cash stock based compensation.

Research and development expenses were $7.1 million for the third quarter of 2019, as compared to $3.8 million for the same period in 2018. The increase was primarily due to an expansion of research and development activities, including increased personnel costs, clinical trials expenses, and professional fees, associated with BTI’s two lead product candidates.

General and administrative expenses were $2.0 million for the third quarter of 2019, as compared to $1.3 million for the same period in 2018. The increase was primarily due to additional payroll and payroll-related expenses, professional fees and costs associated with operating as a public company.

As of September 30, 2019, cash and cash equivalents totaled approximately $40.3 million which included proceeds from the Company’s follow-on-financing completed on September 30, 2019. BTI believes it is well positioned to execute on key milestones.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call, please dial 877-407-2985 (domestic) and 201-378-4915 (international). A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The replay will be available through November 28, 2019.