BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical
company utilizing artificial intelligence to develop transformative
medicines in neuroscience and immuno-oncology, today announced
details regarding the planned design of its upcoming TRANQUILITY
In-Care Phase 3 trial to evaluate BXCL501, the company’s
investigational proprietary, orally dissolving film formulation of
dexmedetomidine, as a potential acute treatment for agitation
associated with Alzheimer’s dementia (AAD) in the care setting. The
Company’s plan to conduct this trial is based on feedback received
from the U.S. Food and Drug Administration (FDA), following the
recent receipt of minutes from the Type B/Breakthrough Therapy
designation meeting held with the agency on February 20, 2024.
“The design of our upcoming TRANQUILITY In-Care trial largely
mirrors TRANQUILITY II, which demonstrated positive efficacy and
safety results with a 60 mcg dose of BXCL501,” said Vincent
O’Neill, M.D., Chief of Product Development and Medical Officer of
BioXcel Therapeutics. “We have completed two recent meetings with
the FDA on our TRANQUILITY program, and are pleased to have
obtained clarity on the next steps for our AAD development path.
This represents a major milestone since there is no U.S. regulatory
precedent for episodic treatment of AAD. We are now intensely
focused on advancing this important program.”
TRANQUILITY In-Care Pivotal Phase 3 Trial Design
Summary
- The TRANQUILITY In-Care trial is designed as a double blind,
placebo-controlled study to evaluate the efficacy and safety of a
60 mcg dose of BXCL501 over a 12-week period.
- The trial is expected to enroll a total of approximately 150
patients 55 years and older across the spectrum of Alzheimer’s
disease severity with mild, moderate, and severe dementia with
mini-mental state examination (MMSE) scores of 0 to 25 who reside
in skilled nursing facilities, memory care units, or assisted
living facilities.
- The trial is expected to enroll patients with episodic
agitation, with patients self-administering 60 mcg of BXCL501 or
placebo when agitation episodes occur over the trial period.
- The primary endpoint is expected to be a change from baseline
in the Positive and Negative Syndrome Scale-Excitatory Component
(PEC) total score at two hours post-first dose. This is the same
endpoint used in previous TRANQUILITY trials and in studies that
supported the FDA approval of IGALMI™ (dexmedetomidine) sublingual
film.
- Continued efficacy evaluations are expected to be conducted
through performing additional PEC and complementary efficacy
measures, including the global impression of change in
agitation.
- As part of the TRANQUILITY In-Care trial, the Company plans to
include a feasibility cohort of 20 patients that would be evaluated
in the home setting.
The Company expects to generate additional Phase 3 efficacy and
safety data in the TRANQUILITY In-Care trial to expand the database
beyond the 70 patients who have already been treated with 60 mcg of
BXCL501 in TRANQUILITY I and II to date. The Company also plans to
discuss the details of the requirement for long-term safety data at
a future meeting with the FDA.
A slide presentation on the TRANQUILITY program is available on
the Investors section of the Company’s website:
bioxceltherapeutics.com.
About IGALMI™
(dexmedetomidine) sublingual
film
INDICATION
IGALMI™ (dexmedetomidine) sublingual film is a prescription
medicine, administered under the supervision of a health care
provider, that is placed under the tongue or behind the lower lip
and is used for the acute treatment of agitation associated with
schizophrenia and bipolar disorder I or II in adults. The safety
and effectiveness of IGALMI has not been studied beyond 24 hours
from the first dose. It is not known if IGALMI is safe and
effective in children.
IMPORTANT SAFETY
INFORMATION
IGALMI can
cause serious
side effects,
including:
- Decreased blood pressure,
low blood pressure upon standing, and slower than normal heart
rate, which may be more likely in patients with low blood
volume, diabetes, chronic high blood pressure, and older patients.
IGALMI is taken under the supervision of a healthcare provider who
will monitor vital signs (like blood pressure and heart rate) and
alertness after IGALMI is administered to help prevent falling or
fainting. Patients should be adequately hydrated and sit or lie
down after taking IGALMI and instructed to tell their healthcare
provider if they feel dizzy, lightheaded, or faint.
- Heart rhythm changes (QT interval
prolongation). IGALMI should not be given to patients with
an abnormal heart rhythm, a history of an irregular heartbeat, slow
heart rate, low potassium, low magnesium, or taking other drugs
that could affect heart rhythm. Taking IGALMI with a history of
abnormal heart rhythm can increase the risk of torsades de pointes
and sudden death. Patients should be instructed to tell their
healthcare provider immediately if they feel faint or have heart
palpitations.
- Sleepiness/drowsiness. Patients should not
perform activities requiring mental alertness, such as driving or
operating hazardous machinery, for at least 8 hours after taking
IGALMI.
- Withdrawal reactions,
tolerance, and
decreased response/efficacy.
IGALMI was not studied for longer than 24 hours after the first
dose. Physical dependence, withdrawal symptoms (e.g., nausea,
vomiting, agitation), and decreased response to IGALMI may occur if
IGALMI is used longer than 24 hours.
The most
common side
effects of IGALMI in clinical studies were
sleepiness or drowsiness, a prickling or tingling sensation or
numbness of the mouth, dizziness, dry mouth, low blood pressure,
and low blood pressure upon standing.
These are not all the possible side effects of IGALMI. Patients
should speak with their healthcare provider for medical advice
about side effects.
Patients should tell their healthcare provider about
their medical history, including if they suffer from any
known heart problems, low potassium, low magnesium, low blood
pressure, low heart rate, diabetes, high blood pressure, history of
fainting, or liver impairment. They should also tell their
healthcare provider if they are pregnant or breastfeeding or take
any medicines, including prescription and over-the-counter
medicines, vitamins, and herbal supplements. Patients should
especially tell their healthcare provider if they take any drugs
that lower blood pressure, change heart rate, or take anesthetics,
sedatives, hypnotics, and opioids.
Everyone is encouraged to report negative side effects of
prescription drugs to the FDA. Visit www.fda.gov/medwatch or call
1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at
1-833-201- 1088 or medinfo@bioxceltherapeutics.com.
Please see full Prescribing Information at igalmi.com.
About BXCL501In indications
other than those approved by the U.S. Food and Drug Administration
(FDA) as IGALMI™ (dexmedetomidine) sublingual film, BXCL501 is an
investigational proprietary, orally dissolving film formulation of
dexmedetomidine, a selective alpha-2 adrenergic receptor agonist.
BioXcel Therapeutics believes that BXCL501 potentially targets an
important mediator of agitation, and the Company has observed
anti-agitation results in multiple clinical trials across several
neuropsychiatric disorders. BXCL501 is under investigation by
BioXcel Therapeutics for the acute treatment of agitation
associated with dementia due to probable Alzheimer’s disease and
for the acute treatment of agitation associated with bipolar I or
II disorder or schizophrenia in the at-home setting. The safety and
efficacy of BXCL501 for these investigational uses have not been
established. BXCL501 has been granted Breakthrough Therapy
designation by the FDA for the acute treatment of agitation
associated with dementia and Fast Track designation for the acute
treatment of agitation associated with schizophrenia, bipolar
disorders, and dementia.
About the Mini‐Mental State Examination
(MMSE)The MMSE is the best‐known and most frequently used
short screening tool for providing an overall measure of cognitive
impairment in clinical, research, and community settings. It is
used for the detection of Alzheimer's disease and other dementias
in people with mild cognitive impairment (MCI). The MMSE consists
of a simple pen‐and‐paper test of cognitive function based on a
total possible score of 30 points, and includes tests of
orientation, concentration, attention, verbal memory, naming, and
visuospatial skills.
About the Positive and Negative Syndrome
Scale-Excitatory Component Score (PEC or PANSS-EC)
The PEC total score is a validated endpoint for
use in clinical research to quantify the severity of a patient’s
acute agitation. The PEC rating evaluates 5 elements associated
with agitation: poor impulse control, tension, hostility,
uncooperativeness, and excitement; each scored 1 (minimum) to 7
(maximum). The PEC total score is the sum of these 5 elements and
thus ranges from 5 to 35.
About BioXcel Therapeutics, Inc.BioXcel
Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical company
utilizing artificial intelligence to develop transformative
medicines in neuroscience. Its wholly owned subsidiary, OnkosXcel
Therapeutics, is focused on the development of medicines in
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 indications. For
more information, please visit bioxceltherapeutics.com.
Forward-Looking Statements
This press release includes “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995. We
intend such forward-looking statements to be covered by the safe
harbor provisions for forward-looking statements contained in
Section 27A of the Securities Act of 1933, as amended and Section
21E of the Securities Exchange Act of 1934, as amended. All
statements contained in this press release other than statements of
historical fact should be considered forward-looking statements,
including, without limitation, statements regarding the planned
trial design of the TRANQUILITY In-Care trial; expected discussions
with the FDA; and the potential for the results from the Company’s
completed, ongoing and proposed clinical trials to support
regulatory approvals for its product candidates in both the
care-facility and at-home settings. When used herein, words
including “anticipate,” “believe,” “can,” “continue,” “could,”
“designed,” “estimate,” “expect,” “forecast,” “goal,” “intend,”
“may,” “might,” “plan,” “possible,” “potential,” “predict,”
“project,” “should,” “target,” “will,” “would” and similar
expressions are intended to identify forward-looking statements,
though not all forward-looking statements use these words or
expressions. 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 and limited revenue generation; its incurrence of
significant losses; its strategic reprioritization and related
reduction in force may not achieve its intended outcome; its need
for substantial additional funding and ability to raise capital
when needed; its significant indebtedness, ability to comply with
covenant obligations and potential payment obligations related to
such indebtedness and other contractual obligations; the Company
has identified conditions and events that raise substantial doubt
about its ability to continue as a going concern; its limited
experience in drug discovery and drug development; risks related to
the TRANQUILITY program; risks related to the limited clinical data
supporting potential safety or efficacy of BXCL501 for use in the
at-home setting; its dependence on the success and
commercialization of IGALMI, BXCL501, BXCL502, BXCL701 and BXCL702
and other product candidates; interim “top-line” and preliminary
data from its clinical trials may change and result in material
changes in the final data; its ability to receive regulatory
approval from the FDA and comparable foreign authorities for its
product candidates; clinical trials are expensive, time-consuming,
difficult to design, difficult to conduct, and involve an uncertain
income; its lack of experience in marketing and selling drug
products; the risk that IGALMI or the Company’s product candidates
may not be accepted by physicians or the medical community in
general; the Company’s estimated number of episodes of agitation
and its corresponding estimated total addressable market are
subject to inherent challenges and uncertainties; the Company still
faces extensive and ongoing regulatory requirements and obligations
for IGALMI; the failure of preliminary data from its clinical
studies to predict final study results; failure of its early
clinical studies or preclinical studies to predict future clinical
studies; its ability to enroll patients in its clinical trials;
undesirable side effects caused by the Company’s product
candidates; its novel approach to the discovery and development of
product candidates based on EvolverAI; the significant influence of
and dependence on BioXcel LLC; its exposure to patent infringement
lawsuits; its reliance on third parties; its ability to comply with
the extensive regulations applicable to it; impacts from data
breaches or cyber-attacks, if any; the Company is and may in the
future be subject to legal proceedings, claims and investigations
in or outside the ordinary course of business, which could be
costly and time-consuming to defend and could result in unfavorable
outcomes; risks related to unfavorable global political or economic
events and conditions; risks associated with the increased scrutiny
relating to environmental, social and governance (ESG) matters;
risks associated with federal, state or foreign health care “fraud
and abuse” laws; and its ability to commercialize its product
candidates, as well as the important factors discussed under the
caption “Risk Factors” in its Annual Report on Form 10-K for the
fiscal year ended December 31, 2023, as such factors may be updated
from time to time in its other filings with the SEC, which are
accessible on the SEC’s website at www.sec.gov and the Investors
section of the Company’s website at www.bioxceltherapeutics.com.
These and other important factors could cause actual results to
differ materially from those indicated by the forward-looking
statements made in this press release. Any such forward-looking
statements represent management’s estimates as of the date of this
press release. While the Company may elect to update such
forward-looking statements at some point in the future, except as
required by law, it disclaims any obligation to do so, even if
subsequent events cause our views to change. These forward-looking
statements should not be relied upon as representing the Company’s
views as of any date subsequent to the date of this press
release.
Website DisclosureWe may use our 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 &
Media section 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 our website at
www.bioxceltherapeutics.com.
Contact Information
CorporateBioXcel Therapeutics Erik Kopp
1.203.494.7062ekopp@bioxceltherapeutics.com
Investor Relations BioXcel Therapeutics Brennan Doyle
1.475.355.8462bdoyle@bioxceltherapeutics.com
MediaRusso PartnersDavid Schull
1.858.717.2310David.schull@russopartnersllc.comScott
Stachowiak1.646.942.5630Scott.stachowiak@russopartnersllc.com
Source: BioXcel Therapeutics, Inc.
IGALMI™ is a trademark of BioXcel Therapeutics, Inc.BT BIOXCEL
THERAPEUTICS is a registered trademark of BioXcel Therapeutics,
Inc. All other trademarks are the properties of their respective
owners.Copyright © 2024, BioXcel Therapeutics, Inc. All rights
reserved.
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