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
topline results from its Phase 1b/2 proof-of-concept RELEASE study
of BXCL501, the Company’s proprietary, orally dissolving thin film
formulation of dexmedetomidine, for the treatment of opioid
withdrawal symptoms.
The
study met its primary safety endpoint across
multiple doses given twice-daily over seven
days. BXCL501 was generally well tolerated, with
no severe or serious adverse events reported, and
dose dependent exposures were observed across all doses evaluated
(30 mcg, 60 mcg, 90 mcg, 120 mcg, 180 mcg and 240 mcg). See
table below with focus on cardio-vascular and nervous system
treatment emergent adverse events.
System Organ Class Preferred Term |
BXCL501 30 mcg(N=17)n (%) |
BXCL501 60 mcg(N=17)n (%) |
BXCL501 90 mcg(N=21)n (%) |
BXCL501 120 mcg(N=19)n (%) |
BXCL501 180 mcg(N=21)n (%) |
BXCL501 240 mcg(N=15)n (%) |
Placebo(N=25)n (%) |
Cardiac
disorders |
0 |
0 |
0 |
0 |
0 |
1 (6.7) |
0 |
Bradycardia |
0 |
0 |
0 |
0 |
0 |
1 (6.7) |
0 |
|
|
|
|
|
|
|
|
Vascular
disorders |
0 |
1 (5.9) |
0 |
0 |
2 (9.5) |
6 (40.0) |
0 |
Hypotension |
0 |
1 (5.9) |
0 |
0 |
0 |
5 (33.3) |
0 |
Orthostatic hypotension |
0 |
0 |
0 |
0 |
2 (9.5) |
4 (26.7) |
0 |
|
|
|
|
|
|
|
|
Nervous
system disorders |
0 |
0 |
0 |
0 |
3 (14.3) |
7 (46.7) |
0 |
Dizziness |
0 |
0 |
0 |
0 |
1 (4.8) |
0 |
0 |
Presyncope |
0 |
0 |
0 |
0 |
0 |
1 (6.7) |
0 |
Somnolence |
0 |
0 |
0 |
0 |
2 (9.5) |
7 (46.7) |
0 |
Treatment Emergent Adverse Events ("TEAEs") are adverse events with an onset date (and time) equal to or later than the first dose date (and time).Subjects are counted once within each system organ class and preferred term.Includes number of any TEAEs, and number (%) of subjects with any TEAEs.BXCL501
doses were administered BID (twice a day).
With respect to retention, a secondary endpoint,
the study showed that patients in multiple dose cohorts treated
with BXCL501 had numerical improvements in retention rates, a key
goal of opioid withdrawal treatment. The 120 mcg and 180 mcg dose
groups showed 42% and 52% rates of retention at Day 6 of BXCL501
treatment, respectively, versus 24% for placebo, though
observations were not statistically significant. The results also
showed that of the 87% of patients who had fentanyl in their
systems upon entry, greater than 50% remained fentanyl positive
following the morphine stabilization phase of 5 days.
Consequently, withdrawal symptoms were not equivalent across
various dose cohorts indicating morphine did not normalize
withdrawal symptoms. Improvements were not observed in the severity
of opiate withdrawal as measured by the Short Opiate Withdrawal
Scale of Gossop (“SOWS-Gossop”) or the Clinical Opiate Withdrawal
Scale (“COWS”). The Company believes that the high fentanyl
prevalence and lack of normalization observed in study subjects
could have confounded these results and made them difficult to
interpret.
“We’re very pleased with the tolerability of
BXCL501 observed across multiple doses, twice-a-day and for
consecutive treatment days in this study, which we believe provides
valuable insights as we explore additional indications and
treatment settings that require multiple dosing regimens,”
commented Reina Benabou, M.D., Ph.D., Senior Vice President &
Chief Development Officer. “Treating opioid withdrawal is a
significant national challenge, complicated by the more recent high
rates of fentanyl addiction, which is significantly more potent and
more prevalent than other opioids. We’re encouraged that the
RELEASE study helped us to identify a dose range that was generally
well tolerated and resulted in numerical improvements in retention
in this patient population. We’ll continue to analyze these results
in collaboration with our advisors regarding potential next steps
for this important indication.”
“With more than 81,000 drug overdose deaths in
the U.S. last year, there is an urgent need for improved strategies
to help transition patients off opioids. Over the past five years,
the epidemic has grown more challenging due to the widespread
emergence of counterfeit fentanyl,” said Tom Kosten, M.D., Waggoner
Professor in Psychiatry, Pharmacology, Neuroscience and Immunology
at Baylor College of Medicine. “The results from the RELEASE study,
which identified dosing regimens for BXCL501 that were well
tolerated, showed that more patients receiving BXCL501 were able to
complete treatment, suggesting that BXCL501 may have potential as a
non-opioid based treatment option to address this unmet need.”
About RELEASE
The multicenter, randomized, double-blind,
placebo-controlled, ascending dose Phase 1b/2 study was designed to
evaluate the safety, pharmacokinetics, tolerability, and efficacy
of escalating doses of BXCL501 versus placebo, following
discontinuation of morphine maintenance in patients (n=125) with
opioid use disorder who are physically dependent on opioids.
Throughout the 7-day treatment phase, BXCL501 was evaluated in
sequential, ascending dose cohorts and patients received BXCL501 at
either the 30 mcg, 60 mcg, 90 mcg, 120 mcg, 180 mcg and 240 mcg or
placebo, administered twice daily, approximately 12 hours apart.
Following the completion of each dose cohort, a safety and
tolerability review was performed to determine the next tested
dose. The study was designed to assess patients’ symptoms of acute
opioid withdrawal, following the morphine maintenance phase with
the Clinical Opiate Withdrawal Scale ("COWS") and the Short Opiate
Withdrawal Scale of Gossop ("SOWS-Gossop").
About Opioid Drug
Withdrawal
According to the Centers for Disease Control and
Prevention (“CDC”), the misuse of and addiction to opioids is a
serious national crisis and is the leading cause of death in the
U.S. for those under 50 years old. Between 1999-2019, almost
450,000 people died from an overdose involving an opioid, with
approximately 36,000 deaths occurring in 2019 alone involving
synthetic opioids, including fentanyl. The surge in fentanyl
availability began in 2013. Synthetic opioid-involved death rates
increased by over 15% from 2018 to 2019 and accounted for nearly
73% of all opioid-involved deaths in 2019. The rate of overdose
deaths involving synthetic opioids were more than 11 times higher
in 2019 than in 2013. The CDC estimates the total "economic burden"
of prescription opioid misuse alone in the U.S. is $78.5 billion a
year, including the costs of healthcare, lost productivity,
addiction treatment and criminal justice involvement. Opioid
withdrawal is a condition characterized by symptoms such as
anxiety, agitation, sleep problems, muscle aches, runny nose,
sweating, nausea, vomiting, diarrhea, and drug craving — that occur
after stopping or reducing the use of opioids in anyone with
physical dependence on opioids.
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
Breakthrough Therapy designation for the acute treatment of
agitation in dementia and Fast Track designation for the acute
treatment of agitation in 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, a
Phase 1b/2 trial (TRANQUILITY) for the acute treatment of dementia
related agitation, as well as a Phase 1b/2 trial (RELEASE) for the
treatment of opioid withdrawal symptoms. This product candidate is
also being evaluated 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 opioid withdrawal symptoms, the possible impact of the
presence of fentanyl on treatment with BXCL501 and the Company’s
future strategy for BXCL501 for the treatment of opioid withdrawal
symptoms and other indications. 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.
BioXcel Therapeutics, Inc.www.bioxceltherapeutics.com
Contact InformationMary ColemanBioXcel Therapeutics, VP of
Investment Relations
MColeman@bioxceltherapeutics.com1.475.238.6837
Investor Relations:John
Grazianojgraziano@troutgroup.com1.646.378.2942
Media:Julia Deutschjdeutsch@troutgroup.com1.646.378.2967
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