On March 9, 2020 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 fourth quarter and full year ended December 31, 2019 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAR 9, 2020, View Source [SID1234555298]).

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"2019 has been a tremendous year for BTI. We have made significant growth in our two programs – BXCL501 and BXCL701 – laying the groundwork to achieve key milestones in the coming years," stated Vimal Mehta, Chief Executive Officer of BTAI. "In neuroscience, we have made momentous advancements in the clinical development of BXCL501 and look forward to announcing topline results from our SERENITY program and our Phase 1b/2 TRANQUILITY trial in dementia-related agitation in mid-2020. Additionally, we have been dedicated to expanding the potential therapeutic use of BXCL501, announcing a fourth indication last month as well as examining biomarkers that may have relevance for a range of hyperarousal disorders. We believe these therapeutic opportunities, along with our plans to investigate BXCL501 for the treatment of all types of agitation, are crucial steps to building out a leading neuroscience franchise."

Dr. Mehta added, "In addition to our ongoing studies with BXCL701 in prostate and pancreatic cancers, we are also evaluating this immuno-oncology candidate, in combination with KEYTRUDA, in multiple advanced solid tumors with the goal of improving treatment responses to this PD-1 inhibitor. We believe this basket trial, led by researchers at MD Anderson, will help to accelerate the evaluation of BXCL701 and help to explore its full potential."

Fourth 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.

·We initiated pivotal Phase 3 trials for the acute treatment of agitation in patients with schizophrenia (SERENITY I) and bipolar disorder (SERENITY II). Enrollment of patients is on track and topline data readouts from both trials are expected mid-2020;
·In January, the first patient was enrolled in the TRANQUILITY study, a Phase 1b/2 trial of BXCL501 for the acute treatment of agitation associated with geriatric dementia, expanding potential therapeutic use of BXCL501 beyond current neuropsychiatric disorders. BTI expects to report data in mid-2020;
·We received clearance from the U.S. Food and Drug Administration for an Investigational New Drug application for the treatment of opioid withdrawal symptoms, a potential fourth indication for BXCL501;
·A Phase 2 study designed to measure biomarkers associated with agitation in patients with schizophrenia and their response to treatment with BXCL501 was initiated by researchers at Yale University earlier last month, with data expected in Q2 2020.

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.

•We advanced the clinical evaluation of BXCL701 via the initiation of an open-label Phase 2 basket trial, which is being conducted at MD Anderson. This study is evaluating the combination of BXCL701 and Pembrolizumab (KEYTRUDA) in patients with advanced solid cancers;
•The Company recently presented additional safety and tolerability data from the first and second patient cohorts of the Phase 1b/2 trial of BXCL701 and KEYTRUDA for tNEPC at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU). The trial is currently enrolling an expansion cohort to explore the use of BID dosing. After the successful optimization of BID dosing, the Company expects to advance to the efficacy stage of the trial;
•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

·In February 2020, the Company raised net proceeds of $60 million in connection with its common stock offering. We believe that these proceeds, together with current reserves, provide BTI cash runway to fund key clinical, regulatory and operational milestones into 2021.

Fourth Quarter and Full Year 2019 Financial Results

BTI reported a net loss of $8.3 million for the fourth quarter of 2019, compared to a net loss of $7.1 million for the same period in 2018. The fourth quarter 2019 results include approximately $0.7 million in non-cash stock based compensation.

Research and development expenses were $6.5 million for the fourth quarter of 2019, as compared to $6.0 million for the same period in 2018. The increase was primarily due to an increase in professional research and related project costs, salary, and related payroll costs, manufacturing costs offset in part by a decrease in clinical trial expenses.

General and administrative expenses were $1.9 million for the fourth quarter of 2019, as compared to $1.3 million for the same period in 2018. The increase was primarily due to increases in salary, and related payroll costs and professional fees.

BTI reported a net loss of $33.0 million for the full year 2019, compared to a net loss of $19.3 million for the same period in 2018.

Research and development expenses were $25.8 million for full year 2019, as compared to $14.6 million for the same period in 2018. The increase was primarily due to clinical trial costs, salary and related payroll costs, professional research and project costs and manufacturing costs.

General and administrative expenses were $7.8 million for full year 2019, as compared to $5.4 million for the same period in 2018. The increase was primarily due to salary and related payroll costs and professional fees.

As of December 31, 2019, cash and cash equivalents totaled approximately $32.4 million.

Please note that these numbers do not include our recent financing, which secured $60 million in net proceeds.

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 March 23, 2020.