BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical
company utilizing artificial intelligence to develop transformative
medicines in neuroscience and immuno-oncology, today announced
additional details regarding the planned design of its SERENITY
At-Home 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 bipolar disorders or schizophrenia in the home
setting. The Company’s plan to conduct this trial using a 120 mcg
dose is based on feedback received from the U.S. Food and Drug
Administration (FDA) following the recent receipt of agency minutes
from the Type C meeting held on March 6, 2024.
“BXCL501 is already FDA-approved as IGALMI™ at the 120 mcg dose
for the acute treatment of agitation associated with schizophrenia
or bipolar I or II disorder in adults under healthcare
provider supervision, based on efficacy and safety data that we
previously generated in SERENITY I and II,” said Vincent O’Neill,
M.D., Chief of Product Development and Medical Officer of BioXcel
Therapeutics. “We are now striving to make BXCL501 available to
millions of additional patients by collecting the data necessary to
potentially expand its label for outpatient use at home, where
nearly 60% of the episode volume occurs.”
SERENITY At-Home Pivotal Phase 3 Trial Design
Summary
- The SERENITY At-Home Phase 3 trial is designed as a double
blind, placebo-controlled study to evaluate the safety and efficacy
of a 120 mcg dose of BXCL501 over a 12-week period.
- The outpatient trial is expected to enroll a total of
approximately 200 patients with agitation associated with bipolar
disorder or schizophrenia.
- Patients will self-administer 120 mcg of BXCL501 or placebo
when agitation episodes occur over the trial period.
- The primary objective is safety with efficacy measures as
exploratory endpoints to evaluate use in the outpatient
setting.
In addition, the Company expects to enroll approximately 30
patients in a separate study to evaluate the correlation between
patient-reported or informant-reported efficacy with trained
rater-reported efficacy using Positive and Negative Syndrome
Scale-Excitatory Component (PEC) measurements.
A corporate presentation, including information on the SERENITY
program, is available on the Investors section of the Company’s
website: bioxceltherapeutics.com.
*SERENITY At-Home represents the redesigned SERENITY III
trial
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 BXCL501
In 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 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 SERENITY At-Home Phase 3 trial; 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 Company’s 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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