On March 11, 2021 BioXcel Therapeutics, Inc. ("BioXcel" or the "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its quarterly results for the fourth quarter and full year ended December 31, 2020 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAR 11, 2021, View Source [SID1234576503]).

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"Over the last 12 months, we have made significant progress with our leading neuroscience program, BXCL501, including reporting successful data readouts, submitting our first NDA, as well as advancing other potential indications," stated Vimal Mehta, Chief Executive Officer of BioXcel. "In addition to submitting our NDA for the acute treatment of schizophrenia and bipolar disorder related agitation, we also announced positive data from the TRANQUILITY Phase 1b/2 trial in dementia related agitation and will be discussing our plans for a pivotal Phase 3 program with the FDA in the second quarter of 2021. In parallel, we continue to evaluate BXCL501’s potential in a wide range of neuropsychiatric conditions with ongoing trials in opioid withdrawal symptoms and delirium related agitation. As we develop the commercial infrastructure needed to bring this potential candidate to market, we continue to execute on our clinical and corporate milestones, as well as leverage our unique AI platform to reach new patient populations where there are large unmet medical needs."

Dr. Mehta continued, "Moreover, our immuno-oncology candidate, BXCL701, has demonstrated encouraging signals of activity in difficult-to-treat tumors across both combination trials. We expect to report topline data from the ongoing trials in mid-2021, in hopes of further demonstrating this candidate’s potential at enhancing an individual’s innate immunity."

Fourth Quarter and Full Year 2020 and Recent Highlights

BXCL501-Neuroscience Program

BXCL501 is an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, designed for the treatment of agitation and opioid withdrawal symptoms. The Company believes BXCL501 may directly target a causal agitation mechanism.

BioXcel recently completed the rolling submission of its NDA to the FDA for BXCL501 for the acute treatment of schizophrenia and bipolar disorder related agitation. Leveraging the Company’s AI-based platform, BioXcel was able to develop this program from the first-in-human trial to NDA submission in just over 2 years.
The Company met the primary and secondary endpoints of the TRANQUILITY trial, a Phase 1b/2 trial of BXCL501 for the acute treatment of dementia related agitation. Topline results showed that BXCL501 was generally well tolerated, with statistically significant and clinically meaningful reductions in agitation achieved with both the 30 mcg and 60 mcg doses at 2 hours as measured by the PEC, PAS and Mod-CMAI scales. The end of Phase 2 meeting with the FDA is planned for Q2 2021, with the goal of initiating a registrational program in the H2 2021.
BioXcel has initiated a supplemental cohort investigating a 40 mcg dose of BXCL501 in 46 patients (1:1 randomization) as an expansion of TRANQUILITY to further inform its clinical development strategy.
The RELEASE study, a Phase 1b/2 trial of BXCL501 for the treatment of opioid withdrawal symptoms, is progressing on track, with the topline readout expected this month.
BioXcel has initiated the PLACIDITY trial, a Phase 2 trial of BXCL501 for the treatment of hospitalized patients with delirium related agitation. The Company expects to report topline results from this trial in Q1 2022.
BioXcel and its collaborators, the VA Connecticut Healthcare System and the Yale University Medical School, were awarded a grant by the U.S. Department of Defense’s ("DOD") Congressionally Directed Medical Research Programs ("CDMRP") to evaluate BXCL501 in patients suffering from post-traumatic stress disorder ("PTSD") related to alcohol and substance abuse disorder ("ASUD"). This will be the first time the Company is investigating BXCL501 as a potential chronic treatment.
The Company was issued U.S. patent No. 10,792,24 on October 6, 2020, which covers film formulations containing Dex and methods of treating agitation using such film formulations. The patent is expected to extend intellectual property ("IP") protection until 2039.
Commercial Highlights

Readiness initiatives for the potential launch of BXCL501 in schizophrenia and bipolar disorder related agitation continue to progress, including the infrastructure needed to support commercialization, the design of our sales force structure and size, as well as market research with both healthcare professionals and payers to inform launch strategy, communications and the BXCL501 value proposition.
The launch of our educational campaign on schizophrenia and bipolar disorder related agitation is planned for May 2021, in sync with Mental Health Awareness Month.
Medical Affairs Initiatives

Our Medical Science Liaison ("MSL") and Medical Manage Care ("MMC") teams are completing their training and will be deployed this month to begin scientific medical to medical exchange with healthcare professionals and payers, respectively.
Two abstracts on SERENITY I and II have been accepted for presentation at the American Psychiatric Association ("APA") Annual Meeting in May 2021.
BXCL701-Immuno-Oncology Program

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

The Phase 2 efficacy portion of the Phase 1b/2 trial of BXCL701 in combination with pembrolizumab (KEYTRUDA) for treatment emergent Neuroendocrine Prostate Cancer ("tNEPC") and castrate-resistant prostate cancer ("CRPC") is advancing, with topline results expected in mid-2021. In November 2020, interim data from this trial was presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 35th Anniversary Annual Meeting ("SITC"), with an efficacy update presented last month at the 2021 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium.
Preliminary efficacy data from the MD Anderson-led Phase 2 open label basket trial evaluating the combination of BXCL701 and KEYTRUDA in patients with advanced solid tumors was presented at SITC (Free SITC Whitepaper). Topline results from the trial are expected in mid-2021.
In January, the FDA granted orphan drug designation to BXCL701 for the treatment of soft tissue sarcoma.
Corporate Highlights

BioXcel continues to integrate and evolve its neuroscience AI machine learning and drug development platform, focusing on symptoms resulting from stress-related behaviors. This platform led to the identification and rapid development of BXCL501, leading to the submission of its first NDA. The Company continues to leverage the platform to identify and develop new neuroscience programs.
In March 2021, June Bray was appointed to the Company’s Board of Directors. Ms. Bray brings over forty years of extensive U.S. and global regulatory experience across all therapeutics areas, including psychiatry and neurology.
In February 2021, Javier Rodriguez was appointed as Chief Legal Officer and Corporate Secretary of BioXcel. Mr. Rodriguez joins the Company with over 20 years of extensive strategic and legal experience within the biopharmaceutical industry.
Fourth Quarter and Full Year 2020 Financial Results

BioXcel reported a net loss of $21.1 million for the fourth quarter of 2020, compared to a net loss of $8.3 million for the same period in 2019. For the full year, BioXcel reported a net loss of $82.2 million, compared to a net loss of $33.0 million for the same period in 2019.

For the fourth quarter of 2020, research and development expenses were $11.4 million as compared to $6.5 million for the same period in 2019. The increase was primarily attributable to increased personnel, clinical trial and professional research costs primarily related to our BXCL501 studies.

Research and development expenses were $58.0 million for the full year 2020, as compared to $25.8 million for the same period in 2019. The increase for the year ended December 31, 2020 was generally attributable to increased clinical trial costs, compensation costs and professional research costs associated with BXCL501, as the Company continues to expand its clinical programs.

General and administrative expenses were $9.7 million for the fourth quarter of 2020, as compared to $1.9 million for the same period in 2019. The increase was primarily due to an increase in compensation costs related to the growth of BioXcel’s operations. Professional fees also increased primarily related to costs associated with preparation for the potential commercial launch of BXCL501.

General and administrative expenses were $24.3 million for full year 2020, as compared to $7.8 million for the same period in 2019. The increase was primarily due to the growth of BioXcel’s operations. Professional and consulting fees also increased due to the expansion of our corporate activities.

The fourth quarter 2020 results include approximately $6.6 million in non-cash stock-based compensation costs. For the full year, non-cash stock-based compensation costs totaled $14.6 million.

As of December 31, 2020, cash and cash equivalents totaled approximately $213.1 million.

Conference Call:

BioXcel 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 BioXcel website at www.bioxceltherapeutics.com. The replay will be available through at least March 25, 2021.