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 its
quarterly results for the fourth quarter and full year ended
December 31, 2020 and provided an update on key strategic and
operational initiatives.
“Over the last 12 months, we have made significant progress with
our leading neuroscience program, BXCL501, including reporting
successful data readouts, submitting our first NDA, as well as
advancing other potential indications,” stated Vimal Mehta, Chief
Executive Officer of BioXcel. “In addition to submitting our NDA
for the acute treatment of schizophrenia and bipolar disorder
related agitation, we also announced positive data from the
TRANQUILITY Phase 1b/2 trial in dementia related agitation and will
be discussing our plans for a pivotal Phase 3 program with the FDA
in the second quarter of 2021. In parallel, we continue to evaluate
BXCL501’s potential in a wide range of neuropsychiatric conditions
with ongoing trials in opioid withdrawal symptoms and delirium
related agitation. As we develop the commercial infrastructure
needed to bring this potential candidate to market, we continue to
execute on our clinical and corporate milestones, as well as
leverage our unique AI platform to reach new patient populations
where there are large unmet medical needs.”
Dr. Mehta continued, “Moreover, our immuno-oncology candidate,
BXCL701, has demonstrated encouraging signals of activity in
difficult-to-treat tumors across both combination trials. We expect
to report topline data from the ongoing trials in mid-2021, in
hopes of further demonstrating this candidate’s potential at
enhancing an individual’s innate immunity.”
Fourth Quarter and Full Year 2020 and Recent
Highlights
BXCL501-Neuroscience Program
BXCL501 is an investigational, proprietary, orally dissolving
thin film formulation of dexmedetomidine, a selective alpha-2
adrenergic receptor agonist, designed for the treatment of
agitation and opioid withdrawal symptoms. The Company believes
BXCL501 may directly target a causal agitation mechanism.
- BioXcel recently completed the rolling submission of its NDA to
the FDA for BXCL501 for the acute treatment of schizophrenia and
bipolar disorder related agitation. Leveraging the Company’s
AI-based platform, BioXcel was able to develop this program from
the first-in-human trial to NDA submission in just over 2
years.
- The Company met the primary and secondary endpoints of the
TRANQUILITY trial, a Phase 1b/2 trial of BXCL501 for the acute
treatment of dementia related agitation. Topline results showed
that BXCL501 was generally well tolerated, with statistically
significant and clinically meaningful reductions in agitation
achieved with both the 30 mcg and 60 mcg doses at 2 hours as
measured by the PEC, PAS and Mod-CMAI scales. The end of Phase 2
meeting with the FDA is planned for Q2 2021, with the goal of
initiating a registrational program in the H2 2021.
- BioXcel has initiated a supplemental cohort investigating a 40
mcg dose of BXCL501 in 46 patients (1:1 randomization) as an
expansion of TRANQUILITY to further inform its clinical development
strategy.
- The RELEASE study, a Phase 1b/2 trial of BXCL501 for the
treatment of opioid withdrawal symptoms, is progressing on track,
with the topline readout expected this month.
- BioXcel has initiated the PLACIDITY trial, a Phase 2 trial of
BXCL501 for the treatment of hospitalized patients with delirium
related agitation. The Company expects to report topline results
from this trial in Q1 2022.
- BioXcel and its collaborators, the VA Connecticut Healthcare
System and the Yale University Medical School, were awarded a grant
by the U.S. Department of Defense’s (“DOD”) Congressionally
Directed Medical Research Programs (“CDMRP”) to evaluate BXCL501 in
patients suffering from post-traumatic stress disorder (“PTSD”)
related to alcohol and substance abuse disorder (“ASUD”). This will
be the first time the Company is investigating BXCL501 as a
potential chronic treatment.
- The Company was issued U.S. patent No. 10,792,24 on October 6,
2020, which covers film formulations containing Dex and methods of
treating agitation using such film formulations. The patent is
expected to extend intellectual property (“IP”) protection until
2039.
Commercial Highlights
- Readiness initiatives for the potential launch of BXCL501 in
schizophrenia and bipolar disorder related agitation continue to
progress, including the infrastructure needed to support
commercialization, the design of our sales force structure and
size, as well as market research with both healthcare professionals
and payers to inform launch strategy, communications and the
BXCL501 value proposition.
- The launch of our educational campaign on schizophrenia and
bipolar disorder related agitation is planned for May 2021, in sync
with Mental Health Awareness Month.
Medical Affairs Initiatives
- Our Medical Science Liaison (“MSL”) and Medical Manage Care
(“MMC”) teams are completing their training and will be deployed
this month to begin scientific medical to medical exchange with
healthcare professionals and payers, respectively.
- Two abstracts on SERENITY I and II have been accepted for
presentation at the American Psychiatric Association (“APA”) Annual
Meeting in May 2021.
BXCL701-Immuno-Oncology Program
BXCL701 is an orally-delivered small molecule, immunomodulator
designed to inhibit dipeptidyl peptidase (DPP) 8/9 and block immune
evasion by targeting Fibroblast Activation Protein (FAP). It has
shown single agent anti-tumor activity in melanoma and safety has
been evaluated in more than 700 healthy subjects and cancer
patients.
- The Phase 2 efficacy portion of the Phase 1b/2 trial of BXCL701
in combination with pembrolizumab (KEYTRUDA®) for treatment
emergent Neuroendocrine Prostate Cancer (“tNEPC”) and
castrate-resistant prostate cancer (“CRPC”) is advancing, with
topline results expected in mid-2021. In November 2020, interim
data from this trial was presented at the Society for Immunotherapy
of Cancer’s 35th Anniversary Annual Meeting (“SITC”), with an
efficacy update presented last month at the 2021 ASCO Genitourinary
Cancers Symposium.
- Preliminary efficacy data from the MD Anderson-led Phase 2 open
label basket trial evaluating the combination of BXCL701 and
KEYTRUDA® in patients with advanced solid tumors was presented at
SITC. Topline results from the trial are expected in mid-2021.
- In January, the FDA granted orphan drug designation to BXCL701
for the treatment of soft tissue sarcoma.
Corporate Highlights
- BioXcel continues to integrate and evolve its neuroscience AI
machine learning and drug development platform, focusing on
symptoms resulting from stress-related behaviors. This
platform led to the identification and rapid development of
BXCL501, leading to the submission of its first NDA. The Company
continues to leverage the platform to identify and develop new
neuroscience programs.
- In March 2021, June Bray was appointed to the Company’s Board
of Directors. Ms. Bray brings over forty years of extensive U.S.
and global regulatory experience across all therapeutics areas,
including psychiatry and neurology.
- In February 2021, Javier Rodriguez was appointed as Chief Legal
Officer and Corporate Secretary of BioXcel. Mr. Rodriguez joins the
Company with over 20 years of extensive strategic and legal
experience within the biopharmaceutical industry.
Fourth Quarter and Full Year 2020 Financial
Results
BioXcel reported a net loss of $21.1 million for the fourth
quarter of 2020, compared to a net loss of $8.3 million for the
same period in 2019. For the full year, BioXcel reported a net loss
of $82.2 million, compared to a net loss of $33.0 million for the
same period in 2019.
For the fourth quarter of 2020, research and development
expenses were $11.4 million as compared to $6.5 million for the
same period in 2019. The increase was primarily attributable to
increased personnel, clinical trial and professional research costs
primarily related to our BXCL501 studies.
Research and development expenses were $58.0 million for the
full year 2020, as compared to $25.8 million for the same period in
2019. The increase for the year ended December 31, 2020 was
generally attributable to increased clinical trial costs,
compensation costs and professional research costs associated with
BXCL501, as the Company continues to expand its clinical
programs.
General and administrative expenses were $9.7 million for the
fourth quarter of 2020, as compared to $1.9 million for the same
period in 2019. The increase was primarily due to an increase in
compensation costs related to the growth of BioXcel’s operations.
Professional fees also increased primarily related to costs
associated with preparation for the potential commercial launch of
BXCL501.
General and administrative expenses were $24.3 million for full
year 2020, as compared to $7.8 million for the same period in 2019.
The increase was primarily due to the growth of BioXcel’s
operations. Professional and consulting fees also increased due to
the expansion of our corporate activities.
The fourth quarter 2020 results include approximately $6.6
million in non-cash stock-based compensation costs. For the full
year, non-cash stock-based compensation costs totaled $14.6
million.
As of December 31, 2020, cash and cash equivalents totaled
approximately $213.1 million.
Conference Call:
BioXcel will host a conference call and webcast today at 8:30
a.m. ET. To access the call, please dial 877-407-2985 (domestic)
and 201-378-4915 (international). A live webcast of the call will
be available on the Investors sections of the BioXcel website at
www.bioxceltherapeutics.com. The replay will be available through
at least March 25, 2021.
About 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 trials for
BXCL501 and BXCL701, the Company’s planned commercial structure,
the potential value of BXCL501 and BXCL701 as treatment options,
and the outcome of the Company’s new drug application. 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
Quarterly Report on Form 10-Q for the quarterly period ended
September 30, 2020 as will be updated by its Annual Report on Form
10-K for the year ended December 31 2020, as such factors may
further 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 BioXcel’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 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.
Statement of Operations
(Unaudited, in thousands, except
per share amounts)
|
Three Months Ended December
31, |
|
Year Ended December 31, |
|
|
2020 |
|
|
|
|
2019 |
|
|
|
|
2020 |
|
|
|
|
2019 |
|
|
Revenues |
$ |
– |
|
|
|
$ |
– |
|
|
|
$ |
– |
|
|
|
$ |
– |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating Expenses |
|
|
|
|
|
|
|
|
|
|
|
Research and Development |
$ |
11,401 |
|
|
|
$ |
6,495 |
|
|
|
$ |
|
57,995 |
|
|
|
$ |
25,797 |
|
|
General and administrative |
|
9,696 |
|
|
|
|
1,918 |
|
|
|
|
|
24,302 |
|
|
|
|
7,804 |
|
|
Total operating expenses |
|
21,097 |
|
|
|
|
8,413 |
|
|
|
|
|
82,297 |
|
|
|
|
33,601 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss from Operations |
|
(21,097 |
) |
|
|
|
(8,413 |
) |
|
|
|
|
(82,297 |
) |
|
|
|
(33,601 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Other Income (expense) |
|
|
|
|
|
|
|
|
|
|
|
Interest income, net |
|
11 |
|
|
|
|
138 |
|
|
|
|
|
128 |
|
|
|
|
633 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
$ |
(21,086 |
) |
|
|
$ |
(8,275 |
) |
|
|
$ |
|
(82,169 |
) |
|
|
$ |
(32,968 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share – basic and
diluted |
$ |
(0.87 |
) |
|
|
$ |
(0.45 |
) |
|
|
$ |
|
(3.79 |
) |
|
|
$ |
(2.02 |
) |
|
Weighted average shares
outstanding – basic and diluted |
|
24,375 |
|
|
|
|
18,051 |
|
|
|
|
|
21,683 |
|
|
|
|
16,289 |
|
|
BioXcel Therapeutics, Inc.
Condensed Balance Sheet
(Unaudited, in
thousands)
|
|
|
|
|
|
December 31, |
|
December 31, |
|
|
2020 |
|
2019 |
|
|
|
|
|
|
Cash and cash equivalents |
|
213,119 |
|
|
|
|
32,426 |
|
|
Working capital |
205,223 |
|
|
|
25,639 |
|
Total assets |
219,936 |
|
|
|
36,392 |
|
|
Long-term liabilities |
1,398 |
|
|
|
1,029 |
|
|
Total liabilities |
13,240 |
|
|
|
9,497 |
|
|
Total stockholders’
equity |
206,696 |
|
|
|
26,895 |
|
|
|
|
|
|
|
|
|
|
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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