BioXcel Therapeutics Reports Second Quarter 2022 Financial Results and Recent Operational Highlights

• Commercially launched IGALMI™ (dexmedetomidine) sublingual film for the acute treatment of agitation in bipolar I or II disorder and schizophrenia in adult patients
• SERENITY III pivotal trial evaluating at-home use of BXCL501 for the acute treatment of agitation in bipolar and schizophrenia patients planned to initiate in 2H 2022 following recent Type B meeting with FDA
• Top-line data from TRANQUILITY II pivotal trial for agitation associated with Alzheimer’s disease expected in 1H 2023; TRANQUILITY III enrollment initiating in 2H 2022
• Top-line results from ongoing Phase 1 Major Depressive Disorder dose-selection trial in healthy volunteers expected in 1H 2023; Completed 30mcg and 60mcg seven-day daily dosing regimen cohorts
• Well-funded with cash runway into 20251; received $100 million in total from previously announced $260 million of strategic financing
• To host conference call today, August 9, 2022, at 8:30 a.m. ET

NEW HAVEN, Conn., Aug. 09, 2022 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a commercial-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced its financial results for the second quarter ended June 30, 2022 and provided an update on key strategic initiatives.

“BioXcel Therapeutics made tremendous progress in its journey to becoming a fully integrated AI-driven commercial-stage company with the potential to transform the agitation treatment landscape,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “This transformation is being driven by the continued execution of our land and expand strategy within our neuroscience franchise. We are focused on the commercial launch for our recently FDA approved drug IGALMI™ while significantly increasing the opportunity for BXCL501 through at-home, medical setting expansion and the pursuit of multiple additional indications for our BXCL501 franchise. We believe we are well-positioned and have laid a strong foundation to drive long-term sustainable growth.” 

Company Highlights

Neuroscience Franchise

IGALMI™ (dexmedetomidine) sublingual film

IGALMI was approved by the U.S. Food and Drug Administration (FDA) on April 5, 2022 for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.² Within these two patient populations in the U.S., up to 25 million agitation episodes occur each year.^3-5

• Trade and Market Access: Key focus on unlocking access and supporting unencumbered demand generation for IGALMI.
• IGALMI made available in 120mcg and 180mcg doses through the Company’s third-party logistics provider and for order through wholesalers as of early July.
• Engagement underway with 59 high-value Integrated Delivery Networks (IDNs) and their affiliated hospitals.
• Discussions and negotiations ongoing with major national Group Purchasing Organizations (GPOs), representing over 90% of beds in target hospitals.

• Institutional Sales Force: Deployed first phase of our national sales force to target high-priority accounts in late May, with expansion of commercial footprint in process. Engaging multiple stakeholders in target hospitals and progressing P&T formulary review discussions. Gathered field level market intelligence which further reinforces and augments current and future commercial strategy.
• Early response to the IGALMI value proposition exceeding expectations across full spectrum of institutional and healthcare stakeholders.
• Market dynamics are rapidly evolving, favoring a less invasive and voluntary approach to treating agitation.
• Marketing: Strategic marketing focused on branded campaigns to support sales force activities.
  
  
  
• Peer-to-peer speaker programs educating healthcare providers (HCPs) of the importance of early intervention to mitigate agitation episodes.
• Digital marketing efforts driving significant engagement resulting in over 100,000 visits to IGALMI HCP website.
• Investigating Potential Expansion of BXCL501 for At-Home Use for Agitation Associated with Bipolar Disorders and Schizophrenia: Planning SERENITY III double-blinded, placebo-controlled, pivotal study designed to evaluate BXCL501 60mcg dose for at-home use. This strategic trial decision follows successful completion of Type B meeting with FDA and observed dose-dependent responses in a prior Phase 1/2b study assessing 60mcg, 80mcg, 120mcg, and 180mcg doses. Of the approximately 25 million agitation episodes that occur in the U.S. each year related to schizophrenia and bipolar disorders, approximately one-third occur outside of the institutional setting.⁶

SERENITY III will consist of two parts:

• First part of SERENITY III is similar to the SERENITY I and II pivotal trials and designed to assess efficacy and safety in acutely agitated bipolar and schizophrenia patients.
• Primary efficacy endpoint is change from baseline in Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at two hours after dosing compared to placebo.
• Second part of SERENITY III designed to assess safety compared to placebo when self-administered at home.

SERENITY III will utilize many of the same investigators and clinical sites as SERENITY I and II and is expected to initiate in 2H 2022.

Clinical Pipeline

BXCL501, a proprietary, sublingual film formulation of dexmedetomidine, has received Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia.

Indication Expansion

• Alzheimer’s Disease-related Agitation: TRANQUILITY program is designed to capture Alzheimer’s-related agitation market opportunity. There are an estimated 100 million agitation episodes in Alzheimer’s patients occurring in the U.S. annually.⁷
• TRANQUILITY II: top-line data readout expected in 1H 2023.
• TRANQUILITY III: enrollment expected to begin in 2H 2022.
• Adjunctive Treatment for Major Depressive Disorder (MDD): Ongoing Phase 1 trial evaluating BXCL501 daily dosing designed to inform dose selection in future proof-of-concept study evaluating daily BXCL501 dosing in MDD patients. Over 300 million antidepressant prescriptions are filled annually in the U.S. and current treatments are limited by slow onsets of action and incomplete responses.⁸

1. Double-blind, placebo-controlled, multiple ascending dose (MAD) selection trial in healthy volunteers includes two cohorts of 18 patients, each having completed seven days of daily dosing of 30mcg or 60mcg compared to placebo.
• Dose escalation in current cohort also enrolling to evaluate 80mcg.
• Additional cohorts planned to evaluate safety and assess tolerability of a range of doses administered once daily and twice daily. 
• Top-line results expected in 1H 2023.

OnkosXcel Therapeutics

Established a wholly owned subsidiary to focus on the sustained expansion and optimization of the Company’s immuno-oncology (I-O) franchise, including its most advanced I-O program, BXCL701. BXCL701 is an investigational orally administered, systemic innate immune activator in development for the treatment of aggressive forms of prostate cancer.

• Strategic Advancements: Fully functioning subsidiary dedicated to executing focused strategy and developing innovative oncology pipeline. Evaluating strategic options, which may include third party investments, aimed to fully capture unique value creation opportunity in areas of high unmet medical need.
  
  
• Metastatic Castration-Resistant Prostate Cancer (mCRPC) Program: Continued ongoing Phase 2 trial for BXCL701 in combination with KEYTRUDA^® (pembrolizumab) in mCRPC patients with small cell neuroendocrine carcinoma (SCNC) or adenocarcinoma phenotype.
  
  
• Expect to complete enrollment of 28-patient SCNC cohort in 2H 2022.
• Continued enrollment in adenocarcinoma randomized trial expansion evaluating BXCL701 monotherapy vs. BXCL701-KEYTRUDA combination therapy.

Corporate Updates

• Strengthened Board of Directors: Appointed Michael P. Miller to the Company’s Board of Directors, to provide strategic leadership and commercial growth expertise.
• Enhanced Intellectual Property: Received two Notices of Allowance for patents (issued from patent application numbers 17/560,392 and 17/560,423) related to IGALMI to cover film formulations containing dexmedetomidine and methods of treating agitation using the films.

Second Quarter 2022 Financial Results

Research and Development Expenses: Research and development expenses were $17.9 million for the second quarter of 2022, compared to $13.5 million for the same period in 2021. The increased expenses were primarily attributable to clinical trial costs related to the Company’s TRANQUILITY program.

Selling, General and Administrative Expenses: Selling, general and administrative expenses were $18.4 million for the second quarter of 2022, as compared to $14.1 million for the same period in 2021. The increase was primarily due to personnel and costs related to the launch of IGALMI in the U.S.

Net Loss: BioXcel Therapeutics reported a net loss of $37.7 million for the second quarter of 2022, compared to a net loss of $27.6 million for the same period in 2021.

As of June 30, 2022, cash and cash equivalents totaled approximately $233.5 million. This excludes $30 million of contributions from the $260 million strategic financing announced in April. To date the Company has met the milestones and has received $100 million from the agreement.