BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical
company utilizing artificial intelligence approaches to develop
transformative medicines in neuroscience, today provided an update
on recent developments with its late-stage clinical programs as
well as its patent portfolio for IGALMI™ (dexmedetomidine)
sublingual film. These developments include meetings scheduled with
the U.S. Food and Drug Administration (FDA) to discuss the
TRANQUILITY and SERENITY III clinical programs and the receipt of
two Notices of Allowance (NOAs) from the U.S. Patent and
Trademark Office to extend method of use patent protection for
sublingual dexmedetomidine.
“We believe that we have a substantial body of clinical,
non-clinical, and pharmacokinetic (PK) data to discuss with
the FDA with the goal of gaining alignment on a potential package
required for supplemental new drug application (sNDA) submissions,”
said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “The meetings
with the FDA are critical components of our business strategy. We
plan to seek alignment on a path for the potential approval to
expand the market for BXCL501 for the at-home and assisted
living facility treatment of mild to moderate agitation
associated with dementia due to Alzheimer’s disease, and for
the at-home acute treatment of agitation associated with bipolar I
and II disorders and schizophrenia. Expanding BXCL501 into these
large, underserved markets, while advancing IGALMI™
commercialization, are our top priorities.”
Late-Stage Clinical Programs: TRANQUILITY and SERENITY
III
Agitation Associated with Mild to Moderate Dementia due
to Probable Alzheimer’s Disease (AAD): Type
B/Breakthrough Meeting on October 11The Company plans to
review its TRANQUILITY clinical trial program with the FDA and to
discuss the data package required to support submission of an sNDA
for the approval of BXC501 for the acute treatment of agitation in
mild to moderate dementia patients with probable Alzheimer’s
disease in assisted living facilities and at-home settings.
The briefing book submitted to FDA for the meeting includes
results from 11 double-blind, placebo-controlled Phase 2 and 3
clinical trials evaluating the safety and efficacy of BXCL501.
Trials with BXCL501 have enrolled more than 1,100 patients across
multiple neuropsychiatric conditions and in healthy volunteers, and
have shown no unexpected safety signals, no reports of serious
adverse events or falls related to the study drug, and no
drug-related deaths, which is all consistent with the known
pharmacological effects of BXCL501.
Of the subjects who received various doses of BXCL501, 273 were
over 60 years of age, and 204 were over 65 years of age. The
TRANQUILITY I and TRANQUILITY II trials were placebo-controlled,
showed statistically significant separation from placebo in
Positive and Negative Syndrome Scale-Excitatory Component (PEC)
total score at two hours with the 60 mcg dose (primary endpoint),
and had no unexpected safety findings. Data from TRANQUILITY I led
the FDA to grant BXCL501 Breakthrough Therapy designation for the
acute treatment of agitation associated with dementia.
Additionally, the efficacy results seen in TRANQUILITY II after 12
weeks for all treated episodes was comparable to those observed
after the first dose. Moreover, the adverse events (AEs) observed
after the first dose were similar to the AEs observed after all
doses in TRANQUILITY II (443 episodes treated) and comparable to
the AEs observed in the single-dose TRANQUILITY I trial (100
episodes treated).
The Company is continuing its previously disclosed investigation
into protocol adherence and data integrity at a principal
investigator’s trial site in connection with the TRANQUILITY II
trial, and an independent third party is auditing the data
collected at that site. For additional information regarding the
TRANQUILITY II Phase 3 trial and related investigation and audit,
see the Company’s Current Report on Form 8-K filed with the
Securities and Exchange Commission today October 4, 2023, which
should be read in conjunction with this press release.
At-Home Setting in Agitation Associated with Bipolar
Disorders I and II and Schizophrenia:Type C
Meeting on November 8 The Company will review its SERENITY
III program with the FDA on November 8, 2023 and plans to discuss
the data package required to support submission of an sNDA seeking
approval of BXCL501 for the acute treatment of agitation associated
with bipolar disorders I and II and schizophrenia in the at-home
setting. In addition, the Company plans to discuss the evaluation
of the 80 mcg dose of BXCL501 and several potential protocol
amendments to the SERENITY III Part 2 trial. The Company identified
the 80 mcg dose as more favorable for further development based on
pharmacokinetic-pharmacodynamic (PK-PD) modeling anchored by
extensive data from studies that evaluated the 60 mcg dose of
BXCL501 (half of the lower approved IGALMI™ dose) as well as
studies that evaluated the 120 mcg and 180 mcg approved doses of
IGALMI™.
The primary objective of Part 2 of SERENITY III is to assess
safety (the incidence of treatment-emergent adverse events), and
the secondary objectives include various efficacy assessments.
IGALMI™ Market Exclusivity Strengthened: Two Notices of
Allowance (NOAs) ReceivedBioXcel
Therapeutics recently received two NOAs1 from
the U.S. Patent and Trademark Office (USPTO) for
patent applications related to the method of use of sublingual
dexmedetomidine for the treatment of agitation associated with
bipolar disorders and schizophrenia. When these patents are
granted, the Company plans to list them in the FDA’s
Approved Drug Products with Therapeutic Equivalence Evaluations
(commonly known as the Orange Book) for IGALMI™. The
Company now has four U.S. patents claiming formulations of
dexmedetomidine with exclusivity until 2039 currently listed in the
Orange Book. The Company expects that these two new patents will
expire no earlier than Dec. 29, 2037, subject to the patent term
adjustment, patent term extension, and terminal disclaimers. These
patents further broaden the scope of intellectual property estate
for IGALMI™ and for future potential indications.
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 BioXcel Therapeutics, Inc.
BioXcel Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical
company utilizing artificial intelligence to develop transformative
medicines in neuroscience. 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.
The Company’s commercial product, IGALMI™ (dexmedetomidine)
sublingual film (developed as BXCL501), is approved for the acute
treatment of agitation associated with schizophrenia or bipolar I
or II disorder in adults. The safety and effectiveness of IGALMI
have not been established beyond 24 hours from the first dose. For
more information, please visit igalmi.com and also see
the IGALMI full Prescribing Information.
BXCL501 is under evaluation for at-home use for the acute
treatment of agitation associated with Alzheimer’s dementia and for
the acute treatment of agitation associated with bipolar disorders
or schizophrenia. The safety and efficacy of BXCL501 for these uses
have not been established. The Company is also developing BXCL502
as a potential therapy for chronic agitation in dementia. The
safety and efficacy of BXCL502 have not been established in any
indication. 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 (the
“Securities Act”) and Section 21E of the Securities Exchange Act of
1934, as amended (the “Exchange Act”). 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 Company’s expected timing and
outcome of discussions with FDA; the potential outcomes of the
Company’s investigation and third-party audit of a principal
investigator’s site; the Company’s advancement of its product
candidates for regulatory approval; and the potential for the
results from the Company’s completed, ongoing and proposed clinical
trials to support regulatory approvals for its product candidates,
including the results from the TRANQUILITY II and SERENITY III
clinical trials. The words “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; its incurrence of significant losses; its need
for substantial additional funding and ability to raise capital
when needed; its ability to successfully negotiate amended terms
under the financing agreements to be able to access funding and to
obtain relief under financial covenants; its significant
indebtedness and potential payment obligations related to such
indebtedness and other contractual obligations; risks associated
with the strategic reprioritization; its limited experience in drug
discovery and drug development; risks related to the TRANQUILITY II
Phase 3 trial and related audit; its dependence on the success and
commercialization of IGALMI™, BXCL501, BXCL502 BXCL701 and BXCL702
and other product candidates; 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 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 receive regulatory approval for
its product candidates; 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; impacts from the COVID-19 pandemic; risks associated with the
increased scrutiny relating to environmental, social and governance
(ESG) matters; its ability to commercialize its product candidates;
and the other important factors discussed under the caption “Risk
Factors” in its Quarterly Report on Form 10-Q for the quarterly
period ended June 30, 2023, which are accessible on the SEC’s
website at www.sec.gov. 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.
Contact Information
Corporate
BioXcel TherapeuticsErik
Kopp1.203.494.7062ekopp@bioxceltherapeutics.com
Investor RelationsBioXcel TherapeuticsBrennan
Doyle1.475.355.8462bdoyle@bioxceltherapeutics.com
MediaRusso PartnersDavid Schull or Scott
Stachowiak1.858.717.2310David.schull@russopartnersllc.comScott.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 © 2023, BioXcel Therapeutics, Inc. All rights
reserved.
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