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 an
update to its BXCL501 dementia program, including clinical updates
and the scheduling of its end of Phase 2 meeting with the FDA.
BXCL501 is the Company’s investigational, proprietary, orally
dissolving thin film formulation of dexmedetomidine (“Dex”).
Following the end of Phase 2 meeting with the FDA, the Company
plans to finalize study design, dosing, and endpoints for its Phase
3 registrational program that is expected to begin in the second
half of 2021.
As part of its overall clinical development
strategy aimed at addressing the broadest dementia related
agitation market, the Company is also providing an update on the
TRANQUILITY clinical results, as well as announcing initiation of a
supplemental study to evaluate a 40 mcg dose:
- In January 2021, BioXcel announced
that the 60 mcg dose of BXCL501 met the primary and all secondary
endpoints in the TRANQUILITY Phase 1b/2 study for the acute
treatment of dementia related agitation, demonstrating
statistically significant, clinically meaningful, rapid, and
durable reductions in agitation with no severe or serious adverse
events. The Company also reported that a lower 30 mcg dose showed
numerical improvements compared to placebo. Following a routine
quality control review of the Company’s TRANQUILITY study data, the
Company discovered that two patients were mis-categorized within
the 30 mcg cohort at the clinical site. After moving the two
patients into their appropriate placebo and 30 mcg groups, the data
from the 30 mcg cohort were re-analyzed, resulting in the 30 mcg
dose crossing over to statistical significance at the two hour time
point, as measured by PEC: p=0.0149; PAS: p=0.0195; and CMAI:
p=0.0364.
- Additionally, BioXcel has initiated
a 46 patient, multicenter, placebo-controlled study investigating a
40 mcg dose cohort of BXCL501. PK/PD modeling of BXCL501 data from
the TRANQUILITY trial was supportive of evaluating the efficacy of
a 40 mcg dose. Results are expected to provide additional insights
to support the Company’s clinical development strategy directed at
all segments of the dementia market, including assisted living
facilities and community or home care settings.
“We are extremely pleased with the overall
progress made with our BXCL501 program for the acute treatment of
dementia related agitation,” commented Vimal Mehta, Chief Executive
Officer of BioXcel. “We believe BXCL501, with its novel mechanism
of action, has the potential to provide a safe and effective
therapy for patients and caregivers to use across the full range of
dementia care settings. We look forward to discussing our
registrational development strategy with the FDA in the second
quarter of 2021 and initiating the pivotal BXCL501 dementia program
in the second half of 2021.”
The updated TRANQUILITY presentation is
available on the Investors section of the Company’s website at
www.bioxceltherapeutics.com.
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 the Positive and Negative Syndrome
Scale-Excitatory Component Score (PEC or PANSS-EC)
The PEC total score is a validated endpoint for
measuring acute agitation in schizophrenia and bipolar patients.
This scale is used 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 the Pittsburgh Agitation Scale
(PAS)
PAS is a validated instrument used to monitor
the severity of agitation associated with dementia. The PAS
measures 4 behavior groups: aberrant vocalization, motor agitation,
aggressiveness, and resisting to care. The groups are evaluated on
a scale from 0 to 4, with 0 defined as no agitation present and 4
defined as the highest form of agitation. The PAS total score
ranges from 0 to 16.
Modified Cohen-Mansfield Agitation
Inventory (Mod-CMAI)
The Modified Cohen-Mansfield Agitation
(Mod-CMAI) is an inventory consisting of 29 behaviors, each rated
on a 7-point scale of frequency with 1 defined as never occurring
and 7 defined as several times an hour. Only behaviors manifested
by the subject at baseline were assessed throughout the study.
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
directly 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 timing and data from
clinical development initiatives and trials for BXCL501 for the
treatment of dementia related agitation, the Company’s planned
discussions with the FDA and the future clinical development
strategy for BXCL501. 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
Quarterly Report on Form 10-Q for the quarterly period ended
September 30, 2020, as such factors may be updated from time to
time in its other filings with the SEC, 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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