BioXcel Therapeutics, Inc. ("BioXcel" or the "Company") (Nasdaq:
BTAI), a clinical-stage biopharmaceutical company utilizing
artificial intelligence approaches to develop transformative
medicines in neuroscience and immuno-oncology, today announced that
BXCL501, the Company's investigational, proprietary, orally
dissolving thin film formulation of dexmedetomidine ("Dex"), has
been granted Breakthrough Therapy designation from the U.S. Food
and Drug Administration ("FDA") for the acute treatment of
agitation associated with dementia. The Breakthrough Therapy
designation is intended to expedite the development and review of
certain product candidates designed to treat serious or
life-threatening diseases or conditions, and the designation
includes increased interaction and guidance from the FDA.
“Managing dementia related agitation,
specifically in elderly patients, represents a significant
challenge for physicians and caregivers, as there are currently no
FDA-approved therapies and off-label drugs come with black box
warnings," stated Vimal Mehta, Chief Executive Officer of BioXcel.
"The FDA's decision to grant Breakthrough Therapy designation
further underscores the significant unmet need for a new treatment
for this underserved patient population, as well as highlights
BXCL501’s potential in becoming the first therapeutic option, if
approved, to address this debilitating medical condition. We look
forward to working closely with the FDA to advance BXCL501 into a
pivotal dementia program, in hopes of quickly bringing this therapy
to the millions of patients across treatment settings that lack
alternative options.”
The Breakthrough Therapy designation for BXCL501
was supported by the positive topline data from the Phase 1b/2
TRANQUILITY study for the acute treatment of agitation associated
with dementia, including Alzheimer's disease. BXCL501 demonstrated
statistically significant reductions in agitation measures at 2
hours post-dose with both the 30 and 60 mcg doses as measured by
multiple scales. The dose dependent response observed has the
potential to support the Company’s plans to evaluate BXCL501 for
use across the full range of dementia care settings.
About FDA Breakthrough Therapy
Designation
Breakthrough Therapy designation is an FDA
program intended to expedite the development and regulatory review
of investigational therapies that are designed to address serious
or life-threatening conditions. The criteria for Breakthrough
Therapy designation requires preliminary clinical evidence that
indicates that the candidate may demonstrate substantial
improvement over existing therapies on one or more clinically
significant endpoints. This designation provides the Company
with more intensive FDA guidance on an efficient drug development
program, and eligibility for other actions to expedite the FDA
review, such as a rolling review of a New Drug Application (“NDA”),
where the FDA may review sections of the NDA before the complete
application is submitted. An NDA for a product candidate receiving
breakthrough designation may also be eligible for priority review
if the relevant criteria are met. Breakthrough Therapy designation
does not change the standards for approval. For more information,
please visit the FDA website at www.fda.gov.
About Dementia Related
Agitation
Dementia is a neurocognitive condition caused by
damage to brain cells that leads to a decline in cognitive
abilities and independent function. It affects approximately 6
million individuals in the United States, with Alzheimer's disease
accounting for up to 80% of these cases. During the course of the
disease, patients with dementia often suffer from psychological and
behavioral symptoms, such as agitation, which has been reported in
up to 70% of patients. Agitation associated with dementia can
negatively affect both the patient and caregiver's quality of life.
Caregiver burden can contribute significantly to burnout, which can
result in premature institutionalization of the patient. Treating
agitation associated with dementia has been a challenge for
providers as there are currently no FDA-approved therapies for the
treatment of dementia-related agitation, and off-label therapies
have black box warnings associated with their use.
About BXCL501
BXCL501 is an investigational, proprietary,
orally dissolving thin film formulation of dexmedetomidine, a
selective alpha-2a receptor agonist for the treatment of agitation
and opioid withdrawal symptoms. BioXcel believes that BXCL501
potentially targets a causal agitation mechanism, and the Company
has observed anti-agitation results in multiple clinical studies
across several neuropsychiatric disorders. BXCL501 has been granted
Fast Track Designation by the U.S. Food and Drug Administration for
the acute treatment of agitation in patients with schizophrenia,
bipolar disorders, and dementia. BXCL501 has been studied in two
Phase 3 trials (SERENITY I and II) for the acute treatment of
schizophrenia related agitation and bipolar disorder related
agitation, respectively, and in a Phase 1b/2 trial (TRANQUILITY)
for the acute treatment of dementia related agitation. This product
candidate is also currently being evaluated in a Phase 1b/2 trial
(RELEASE) for the treatment of opioid withdrawal symptoms and in a
Phase 2 trial (PLACIDITY) for the treatment of delirium related
agitation.
BioXcel Therapeutics, Inc.
BioXcel Therapeutics, Inc. is a clinical-stage
biopharmaceutical company utilizing artificial intelligence
approaches to develop transformative medicines in neuroscience and
immuno-oncology. BioXcel'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. BioXcel's two most
advanced clinical development programs are BXCL501, an
investigational, proprietary, orally dissolving thin film
formulation of dexmedetomidine for the treatment of agitation and
opioid withdrawal symptoms, and BXCL701, an investigational, orally
administered, systemic innate immunity activator in development for
the treatment of aggressive forms of prostate cancer and advanced
solid tumors that are refractory or treatment naïve 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 the potential for BXCL501 to
treat dementia-related agitation and the timing of the planned
pivotal Phase 3 trial of BXCL501 in dementia-related agitation.
When used herein, words including "anticipate," "being," "will,"
"plan," "may," "continue," 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 BioXcel's current expectations and various
assumptions. BioXcel believes there is a reasonable basis for its
expectations and beliefs, but they are inherently uncertain.
BioXcel 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 limited experience in
drug discovery and drug development; its dependence on the success
and commercialization of BXCL501 and BXCL701 and other product
candidates; 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 BioXcel's product candidates;
its approach to the discovery and development of product candidates
based on EvolverAI is novel and unproven; its exposure to patent
infringement lawsuits; its ability to comply with the extensive
regulations applicable to it; impacts from the COVID-19 pandemic;
its ability to commercialize its product candidates; and the other
important factors discussed under the caption "Risk Factors" in its
Annual Report on Form 10-K for the year ended December 31, 2020, 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 our 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 BioXcel 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 BioXcel's
views as of any date subsequent to the date of this press
release.
Contact Information:
BioXcel Therapeutics, Inc.www.bioxceltherapeutics.com
Investor Relations:Mary ColemanBioXcel Therapeutics, VP of
Investment
RelationsMColeman@bioxceltherapeutics.com1.475.238.6837
John GrazianoSolebury
Troutjgraziano@soleburytrout.com1.646.378.2942
Media:Julia DeutschSolebury
Troutjdeutsch@soleburytrout.com1.646.378.2967
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