Full Press Release Details
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
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
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
HAVEN, Conn., Aug. 9, 2022 -- 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."
Neuroscience Franchise
IGALMI (dexmedetomidine) sublingual film
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
SERENITY III will consist of two parts:
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.
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
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
Second Quarter 2022 Financial Results
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.
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.
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.
Therapeutics will host a conference call and webcast August 9, 2022, at 8:30 a.m., ET, to discuss its second quarter 2022 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 November 9, 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.
About TRANQUILITY II and III
Initiated in December of 2021, TRANQUILITY II and III are pivotal
Phase 3 trials evaluating BXCL501 for the acute treatment of agitation in patients with probable Alzheimer's disease (AD). The trials
expand the evaluation of patients who experience agitation across diverse medical settings and across the range of dementia severity.
TRANQUILITY II and III are designed to maximize the opportunity of BXCL501 for the potential treatment of the full spectrum of agitation
associated with AD. Each trial will enroll approximately 150 dementia patients 65 years and older who will self-administer 40 mcg or 60
mcg of BXCL501 or placebo whenever agitation episodes occur over a three-month period. TRANQUILITY II will assess patients in assisted
living or residential facilities requiring minimal assistance with activities of daily living. TRANQUILITY III will assess patients residing
in nursing homes with moderate to severe dementia and require moderate or greater assistance with activities of daily living. The studies
will assess agitation as measured by the changes from baseline in the Positive and Negative Syndrome Scale-Excitatory Component (PEC)
and Pittsburgh Agitation Scale (PAS) total scores. The primary efficacy endpoint for both studies is change in PEC score from baseline
measured at two hours after the initial dose and subsequent doses.
About BioXcel Therapeutics, Inc.
Therapeutics, Inc. is a commercial-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative
medicines in neuroscience and immuno-oncology. The Company's drug re-innovation approach leverages existing approved drugs and/or
clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic
indices. The Company's commercial product, IGALMI (developed as BXCL501), is a proprietary, sublingual film formulation
of dexmedetomidine approved for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.
The safety and effectiveness of IGALMI has not been established beyond 24 hours from the first dose. For more information, please visit
www.IGALMIhcp.com and also see the IGALMI full Prescribing Information. BXCL501 is under investigation for the acute
treatment of agitation associated with probable Alzheimer's disease, and as an adjunctive treatment for major depressive disorder.
The Company is also developing BXCL502 as a potential therapy for chronic agitation in dementia. Under its subsidiary, OnkosXcel Therapeutics,
the Company is developing BXCL701, an investigational, orally administered, systemic innate immunity activator, for the treatment of
aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment na ve to checkpoint inhibitors. For
more information, please visit www.bioxceltherapeutics.com.
Forward-Looking Statements
This press release includes "forward-looking statements"
within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include but
are not limited to: statements regarding the Company's expected timing of, and data results from, trials and clinical studies involving
its product candidates; planned discussions with regulators; its commercial plan and strategy for IGALMI and strategic options for OnkosXcel;
and the Company's future financial and operational results. When used herein, words including "anticipate," "will,"
"plan," "may," "continue," "intend," "designed," "goal" and similar
expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations,
beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying
assumptions, are forward-looking. All forward-looking statements are based upon the Company's current expectations and various assumptions.
The Company believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. The Company may
not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied
by such forward-looking statements as a result of various important factors, including, without limitation: its limited operating history;
its incurrence of significant losses; its need for substantial additional funding and ability to raise capital when needed; its significant
indebtedness and other contractual obligations; its limited experience in drug discovery and drug development; its dependence on the success