On November 10, 2021 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 third quarter ended September 30, 2021 and provided an update on key strategic initiatives (Press release, BioXcel, NOV 10, 2021, View Source [SID1234595220]).

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"We have made tremendous progress advancing our neuroscience and immuno-oncology franchises," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "With our PDUFA date for BXCL501 less than two months away, we are excited that our commercial and launch readiness planning is progressing well. We are equally excited about initiating our Phase 3 program for BXCL501 for acute treatment of agitation associated with dementia in Alzheimer’s patients, the most prevalent type of dementia in the United States, which has a high unmet medical need and we believe would mark the first orally available FDA-approved therapy. Furthermore, adding major depressive disorder as a potential indication for BXCL501 and introducing BXCL502 as a candidate for chronic treatment of agitation in dementia strengthens our pipeline and expands our market opportunity."

Dr. Mehta added, "Our immuno-oncology program is also advancing, with positive interim results from our Phase 2 trial of BXCL701 in heavily pre-treated metastatic castration-resistant prostate cancer (mCRPC) demonstrating encouraging efficacy and a favorable safety profile. We believe BXCL701 shows considerable potential as an investigational, orally administered, 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 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 Acute Treatment of Agitation Associated with Schizophrenia and Bipolar Disorders I and II: On track with FDA review of BXCL501 NDA for acute treatment of agitation associated with schizophrenia and bipolar disorders I and II; Marketing Authorization Application to European Medicines Agency of BXCL501 expected to be submitted in 1H 2022.

·BXCL501 for Acute Treatment of Agitation in Patients with Alzheimer’s Disease: Following multiple meetings with FDA and alignment on key design features, on track to initiate Phase 3 program in Q4 2021. Alzheimer’s disease is the most prevalent type of dementia in the U.S. and is expected to double from 5.8 million patients in 2020 to 11.8 million patients by 2040.1 The Company remains interested in exploring BXCL501 for other dementia subtypes as part of future development.

·BXCL501 for Major Depressive Disorder (MDD): Held pre-Investigational New Drug (IND) meeting with FDA for use of BXCL501 as an adjunctive treatment for MDD, with Selective Serotonin Reuptake Inhibitors (SSRIs) and serotonin-norepinephrine reuptake Inhibitors (SNRIs), and to align on key design features; preparing to submit IND and expect to initiate a clinical trial in 1H 2022.

·BXCL502 for Chronic Treatment of Agitation in Patients with Dementia: Formulation and clinical development planning underway for BXCL502 as a potential monotherapy and in combination with BXCL501 for chronic treatment of agitation in patients with dementia; designed to be a potent and selective antagonist for a GPCR target affecting serotonergic signaling in the cerebral cortex.

AI-driven Drug Discovery & Development

·Hosted a Virtual R&D Day in September highlighting the Company’s innovative approaches to leveraging its proprietary artificial intelligence platform to expand current neuroscience portfolio, including identification of the Company’s newest product candidate, BXCL502, and to broaden the addressable market for lead program, BXCL501, in MDD.

1. Alzheimer’s Association.

Immuno-Oncology Franchise

BXCL701 is an investigational, orally administered, systemic innate immune activator in development for the 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 interim data from Phase 1b/2 trial of BXCL701 in combination with KEYTRUDA (pembrolizumab) for heavily pre-treated mCRPC patients with adenocarcinoma at the 2021 European Society for Medical Oncology Congress in September. Following this data, announced expansion of ongoing Phase 1b/2 trial of BXCL701 in mCRPC patients with either de novo or treatment-emergent small-cell neuroendocrine carcinoma (SCNC).

·Solid Tumors Program (Checkpoint Naïve and Refractory): Expect to present additional efficacy data from MD Anderson-led open-label Phase 2 basket trial of BXCL701 and KEYTRUDA in 1H 2022.

·Peer-Reviewed Journal Findings Published on BXCL701 Mechanism of Action: Journal of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) reported data findings, on November 4, 2021, suggesting BXCL701 may enhance immunotherapy efficacy in ‘cold’ tumor types such as pancreatic cancer. These findings also highlight the potential importance of natural killer (NK) cells along with T cells in regulating pancreatic cancer tumor growth.

Commercial and Launch Readiness Progress

·Expanded Sales Leadership: onboarded a Vice President of Sales and Regional Sales Directors; continuing to recruit sales force across key territories.

·Optimizing Market Access and Pricing Strategy for BXCL501: through evidence-based market research.

·Fully Launched Unbranded Disease Education Campaign (Including partnersincalm website): to promote awareness of acute agitation in schizophrenia and bipolar disorders.

Medical Affairs Progress

·Medical Science Liaison and Medical Managed Care Teams Fully Deployed: actively engaged with healthcare professionals and payers to provide key insights and support potential BXCL501 commercial launch, including participation and presentations at:

oNeuroscience Education Institute Congress in November

oPsych Congress, American College of Emergency Physicians conference and Academy of Managed Care Pharmacy conference in October

oEmergency Nurses Association Annual Meeting in September

Third Quarter 2021 Financial Results

Research and Development Expenses: Research and development expenses were $11.9 million during the third quarter of 2021, as compared to $16.3 million for the same period in 2020. The decreased expenses were primarily attributable to a reduction in BXCL501 clinical trial costs offset in part by increased BXCL701 trial costs. In addition, the Company experienced greater professional and consulting fees primarily related to BXCL501 development.

General and Administrative Expenses: General and administrative expenses were $14.9 million for the third quarter of 2021, as compared to $8.5 million for the same period in 2020. The increase was primarily due to higher stock-based compensation and personnel costs due to continued expansion of teams, 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.

Net Loss: BioXcel Therapeutics reported a net loss of $26.8 million for the third quarter of 2021, compared to a net loss of $24.8 million for the same period in 2020.

As of September 30, 2021, cash and cash equivalents totaled approximately $252.9 million.

Conference Call

BioXcel Therapeutics will host a conference call and webcast today at 8:30 a.m. EDT to discuss its third quarter 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 December 11, 2021.

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.