On March 10, 2022 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the fourth quarter and full year ended December 31, 2021 and provided an update on key strategic initiatives (Press release, BioXcel Therapeutics, MAR 10, 2022, View Source [SID1234609868]).
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"2021 was marked by significant achievement across the business, most notably with the NDA submission for BXCL501, which concluded an unprecedented, less than three-year journey from first-in-human trials to regulatory submission," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "As we near potential approval, we remain focused on executing our launch readiness plan. Matt Wiley, who recently joined as our Chief Commercial Officer, has, and will continue to play a pivotal role in developing our commercial and market access strategy and building out our field sales organization. In parallel, we continued to advance our ‘land and expand’ strategy by furthering our Alzheimer’s disease and MDD programs, both important components of our five-year vision to become the leading AI-enabled neuroscience Company."
Dr. Mehta added, "Our oncology franchise further progressed with encouraging composite response rates for BXCL701 in combination with KEYTRUDA for mCRPC patients with SCNC and adenocarcinoma phenotypes, both aggressive tumors with very few treatment options. We continue to develop BXCL701’s potential to convert cold tumors to hot as a novel systemic innate immune activator."
Company Highlights
Neuroscience Franchise
BXCL501 is an investigational, proprietary, orally dissolving, thin film formulation of the adrenergic receptor agonist dexmedetomidine for the treatment of agitation associated with neuropsychiatric disorders. BXCL501 has received U.S. Food & Drug Administration (FDA) Breakthrough Therapy designation for the acute treatment of agitation associated with dementia and FDA Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders I and Il, and dementia.
BXCL501 for the Acute Treatment of Agitation in Patients with Schizophrenia and Bipolar Disorders: The Company is gearing up to launch BXCL501 in the U.S. pending FDA approval by the April 5, 2022 PDUFA action date. This is a large addressable market, with over 7 million people in the U.S. diagnosed with schizophrenia or bipolar disorders1-3, totaling approximately 25 million annual agitation episodes.3
Data Published in Journal of the American Medical Association (JAMA) from SERENITY II Pivotal Phase 3 Trial Evaluating BXCL501 in Bipolar Disorders: Results demonstrate BXCL501 as a potential treatment for the millions of patients experiencing agitation, a difficult-to-manage symptom associated with many neuropsychiatric conditions.
Indication Expansion
BXCL501 for Acute Treatment of Agitation in Patients with Alzheimer’s Disease: Initiated pivotal Phase 3 program in Q4 2021 following alignment with FDA on key trial design features. Alzheimer’s disease is the most prevalent type of dementia in the U.S. and is expected to approximately double from 5.8 million patients in 2020 to 11.8 million patients by 2040.4
BXCL501 for Major Depressive Disorder (MDD): Submitted an Investigational New Drug (IND) application to evaluate BXCL501 as an adjunct treatment for MDD, the most common type of depression in the United States with approximately 27 million cases a year.5 There are over 300 million antidepressant prescriptions filled annually, with limitations of current treatment including slow onset of action and incomplete response.6
Geographic Expansion
International Growth: The Company expects to submit a Marketing Authorization Application to the European Medicines Agency (EMA) for BXCL501 in 1H 2022.
AI-driven Drug Discovery & Development
The Company continues its innovative approach to R&D by leveraging its proprietary artificial intelligence platform to expand its current neuroscience portfolio, including identification of the Company’s newest product candidate, BXCL502. The Company initiated formulation for this new product candidate for chronic treatment of agitation in dementia patients. BXCL502 is designed to be a potent and selective antagonist for a GPCR target affecting serotonergic signaling in the cerebral cortex.
BXCL501 Commercial Readiness Progress
Expanded Commercial Organization: Hired Chief Commercial Officer Matt Wiley to develop integrated commercial strategy for potential BXCL501 approval and beyond, and continue the buildout of the Company’s national sales force.
Medical Science Liaison and Medical Managed Care Teams Fully Deployed: Continued engagement with health care professionals (HCPs) and payers to provide key insights and support potential BXCL501 commercial launch, including planned participation at:
Academy of Managed Care Pharmacy annual meeting in March 2022
International Society of Pharmacoeconomics and Outcomes Research conference in May 2022
Immuno-Oncology Franchise
BXCL701 is an investigational, orally administered, systemic innate immune activator in development for treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors.
Metastatic Castration-Resistant Prostate Cancer (mCRPC) Program: Presented positive data from Phase 2 trial of BXCL701 in aggressive forms of prostate cancer at 2022 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium in February. The presentations showed that BXCL701 plus KEYTRUDA (pembrolizumab) demonstrated encouraging composite response rates in patients with either adenocarcinoma (21% response rate) or SCNC (33% response rate) phenotype, supporting further evaluation of BXCL701’s potential to extend checkpoint inhibitor therapy into cold tumor settings.
Solid Tumors Program (Checkpoint Naïve and Refractory): Additional efficacy data from MD Anderson-led open-label Phase 2 basket trial of BXCL701 and KEYTRUDA expected in 2H 2022.
Fourth Quarter and Full Year 2021 Financial Results
Research and Development Expenses: Research and development expenses were $12.5 million for the fourth quarter of 2021, compared to $11.4 million for the same period in 2020. The increased expenses were primarily attributable to increased headcount and related costs during the three-month period of 2021.
Research and development expenses were $52.7 million for the full year 2021, as compared to $58.0 million for the same period in 2020. The decrease for the year ended December 31, 2021 was primarily attributable to decreased clinical trial expenses related to BXCL501, partially offset by increased costs related to BXCL701 clinical trials.
General and Administrative Expenses: General and administrative expenses were $13.6 million for the fourth quarter of 2021, as compared to $9.7 million for the same period in 2020. The increase was primarily due to increased headcount and related costs, including higher stock-based compensation, increased marketing and commercial costs related to the potential launch of BXCL501 in the U.S., as well as increased legal and professional fees, and insurance costs.
General and administrative expenses were $54.2 million for the full year 2021, as compared to $24.3 million for the same period in 2020. The increase for the year ended December 31, 2021 was primarily attributable to increased headcount and associated costs, including non-cash stock-based compensation. The Company also incurred significant commercial costs in preparation for a potential U.S. launch of BXCL501.
Net Loss: BioXcel Therapeutics reported a net loss of $26.1 million for the fourth quarter of 2021, compared to a net loss of $21.1 million for the same period in 2020. For the full year, BioXcel reported a net loss of $106.9 million, compared to a net loss of $82.2 million for the same period in 2020.
As of December 31, 2021, cash and cash equivalents totaled approximately $233.0 million.
Conference Call
BioXcel Therapeutics will host a conference call and webcast March 10, 2022 at 8:30 a.m. ET, to discuss its fourth quarter and full year 2021 financial results and provide an update on recent operational highlights. To access the call, please dial 877-407-5795 (domestic) and 201-689-8722 (international). A live webcast of the call will be available on the Investors section of the BioXcel website, www.bioxceltherapeutics.com, and a replay of the call will be available through at least April 10, 2022.
BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the "Email Alerts" option under the News/Events menu of the Investors & Media section of its website.