BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical
company utilizing artificial intelligence to develop transformative
medicines in neuroscience and immuno-oncology, today provided an
update on its late-stage TRANQUILITY and SERENITY clinical
programs. In addition, the Company reported its financial results
for the fourth quarter and full year 2023.
“We believe our late-stage programs evaluating BXCL501 for acute
agitation associated with Alzheimer’s dementia, bipolar disorders,
and schizophrenia represent groundbreaking opportunities to bring
much-needed treatment options to large numbers of patients and
caregivers,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics.
“We are pleased to be advancing the clinical development paths for
TRANQUILITY and SERENITY, and plan to focus our resources on these
high-priority programs.”
TRANQUILITY Program:Evaluating 60 mcg
dose of BXCL501 for agitation associated with Alzheimer’s dementia
(AAD)
- On February 20,
2024, the Company held a Type B/Breakthrough Therapy designation
meeting with FDA.
- Based on the FDA’s feedback, the Company plans to generate
additional Phase 3 efficacy and safety data in care facilities to
expand the database beyond the 70 patients who have already been
treated with 60 mcg of BXCL501 in TRANQUILITY I and II to
date. The Company plans to generate these data in a variety of
relevant care settings and across severity of dementia using the
Positive and Negative Syndrome Scale-Excitatory Component
(PEC) as the primary efficacy measure, as used in the prior
TRANQUILITY II study.
- The Company
announced in November 2023 that it was planning to conduct a Phase
3 trial in the at-home setting, with safety as the primary
objective (TRANQUILITY At Home). Given the priority to expand
the database to generate additional efficacy data in care
facilities, the Company is re-evaluating the timing for initiating
TRANQUILITY At Home.
- Given no prior
regulatory precedent for episodic treatment of agitation in AD
patients, the Company plans to engage with the FDA regarding the
requirement for collection of long-term safety data.
- The Company expects
to provide further guidance regarding program plans following
receipt of final meeting minutes from FDA.
SERENITY Program: Evaluating potential
at-home use of 120 mcg of BXCL501 for agitation associated with
bipolar disorders or schizophrenia
- On March 6, 2024, the Company held
a Type C meeting with FDA.
- Based on FDA feedback, Company
plans to conduct a Phase 3 trial by amending the SERENITY III
protocol to evaluate the 120 mcg dose of BXCL501 (an approved dose
of IGALMI™) in the at-home setting, with safety as the primary
objective and efficacy measures as exploratory endpoints.
- The Company
expects to provide further guidance regarding program plans
following receipt of final meeting minutes from FDA.
Corporate Updates
IGALMI™ Commercialization
- Permanent J-Code for IGALMI™
(dexmedetomidine) sublingual film became effective Jan. 1, 2024;
code helps to standardize the reimbursement process, simplify
claims submission, and in turn streamline billing and reimbursement
for customers.
- Dedicated Corporate Account Director (CAD) team has secured and
continues to secure volume contracts, driving broader access to
IGALMI™.
Patent Portfolio
The Company continues to develop a broad global intellectual
property portfolio, with over 100 patent applications in
prosecution and multiple patents issued as of February 29,
2024.
- Neuroscience franchise (BXCL501 and pipeline): Company’s patent
portfolio includes 10 issued U.S. patents, with eight listed in the
FDA's Approved Drug Products with Therapeutic Equivalence
Evaluations (commonly known as the Orange Book), as well as three
patents from Japan and 11 patents from other countries.
Additionally, the portfolio has 19 utility patent applications and
seven provisional applications in the U.S. and 116 utility patent
applications in other countries, as well as two Patent Cooperation
Treaty (PCT) applications not yet in the national phase. The expiry
dates for the Orange Book listed patents covering Igalmi in the
U.S. run from December 29, 2037 to January 12, 2043.
- Immuno-oncology franchise (BXCL701 and pipeline): Company’s
patent portfolio includes two issued patents in the U.S., one in
Japan, and 12 in other countries, as well as one provisional
application and six utility patent applications in the U.S., and 31
utility patent applications in other countries.
OnkosXcel Therapeutics
- FDA designated as a Fast Track development program the
investigation of BXCL701 in combination with a checkpoint inhibitor
for treatment of patients with metastatic small cell neuroendocrine
prostate cancer (SCNC) with progression on chemotherapy and no
evidence of microsatellite instability.
- Reported completion of patient enrollment in safety portion of
Phase 2 relapsed pancreatic cancer trial of BXCL701 in combination
with KEYTRUDA® sponsored by Georgetown Lombardi Comprehensive
Cancer Center.
- Initiated formal process to assess strategic options.
Fourth Quarter and
Full Year 2023 Financial
Results
Net Revenue: Net revenue from
IGALMI was $376,000 for the fourth quarter of 2023, compared to
$238,000 for the same period in 2022.
Net revenue from IGALMI was $1.4 million for the full year of
2023, compared to $375,000 for 2022.
Research and Development (R&D) Expenses:
R&D expenses were $9.9 million for the fourth quarter of 2023,
compared to $32.5 million for the same period in 2022.
R&D expenses were $84.3 million for the full year of 2023,
compared to $91.2 million for the full year of 2022. The decreased
expenses for both the fourth quarter and the full year were
primarily attributable to a decrease in clinical trial activity
associated with the wind down of the SERENITY III and TRANQUILITY
II studies, a decrease in chemical, manufacturing, and control
(CMC) costs, and a decrease in personnel related to the
Reprioritization.
Selling, General and Administrative (SG&A)
Expenses: SG&A expenses were $9.6 million for the
fourth quarter of 2023, compared to $20.7 million for the same
period in 2022.
SG&A expenses were $83.4 million for the full year 2023,
compared to $68.8 million for 2022. The increased costs for the
full year were primarily attributable to an increase in legal and
professional fees, costs associated with increased personnel, and
related costs to support the commercialization of IGALMI in the
U.S. prior to the Reprioritization.
Net Loss: BioXcel Therapeutics
had a net loss of $22.3 million for the fourth quarter of 2023,
compared to a net loss of $54.8 million for the same period in
2022. For the full year of 2023, BioXcel Therapeutics reported a
net loss of $179.0 million, compared to a net loss of $165.8
million for the full year of 2022. The loss for the 2023 year
includes approximately $18.6 million in non-cash stock-based
compensation. Total cash expenditures for 2023 totaled
approximately $155.0 million.
Cash and cash equivalents totaled $65.2 million on December 31,
2023, compared to $193.7 million on December 31, 2022. The
Company estimates that its current cash and cash equivalents will
fund its operations through mid-2024. This estimated cash runway
does not include potential additional capital that may become
available under the amendments to the strategic financing
agreements or resulting from any potential financing activities
that may be undertaken by the Company.
Conference Call and
WebcastBioXcel Therapeutics will host a conference call
and webcast on March 12, 2024, at 8:00 a.m. ET to provide an update
on recent operational highlights and to discuss its fourth quarter
and full year 2023 financial results. To access the call, please
dial 877-407-5795 (domestic) or 201-689-8722 (international). A
live webcast will be available on the Investors section of the
corporate website, bioxceltherapeutics.com, and a replay will be
available through June 12, 2024.
BioXcel Therapeutics may use its website as a distribution
channel of material information about the Company. Financial and
other important information regarding the Company is routinely
posted on and accessible through the Investors sections of its
website at www.bioxceltherapeutics.com. In addition, you may sign
up to automatically receive email alerts and other information
about the Company by visiting the “Email Alerts” option under the
News/Events section of the Investors & Media website section
and submitting your email address.
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 BXCL501In indications other
than those approved by the U.S. Food and Drug Administration (FDA)
as IGALMI™ (dexmedetomidine) sublingual film, BXCL501 is an
investigational proprietary, orally dissolving film formulation of
dexmedetomidine, a selective alpha-2 adrenergic receptor agonist.
BioXcel Therapeutics believes that BXCL501 potentially targets an
important mediator of agitation, and the Company has observed
anti-agitation results in multiple clinical trials across several
neuropsychiatric disorders. BXCL501 is under investigation by
BioXcel Therapeutics for the acute treatment of agitation
associated with dementia due to probable Alzheimer’s disease and
for the acute treatment of agitation associated with bipolar I or
II disorder or schizophrenia in the at-home setting. The safety and
efficacy of BXCL501 for these investigational uses have not been
established. BXCL501 has been granted Breakthrough Therapy
designation by the FDA for the acute treatment of agitation
associated with dementia and Fast Track designation for the acute
treatment of agitation associated with schizophrenia, bipolar
disorders, and dementia.
About the Positive and Negative Syndrome
Scale-Excitatory Component Score (PEC or
PANSS-EC) The PEC total score is a validated endpoint
for use 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 BioXcel Therapeutics, Inc.BioXcel
Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical company
utilizing artificial intelligence to develop transformative
medicines in neuroscience. Its wholly owned subsidiary, OnkosXcel
Therapeutics, is focused on the development of medicines in
immuno-oncology. 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. 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 and Section
21E of the Securities Exchange Act of 1934, as amended. All
statements contained in this press release other than statements of
historical fact should be considered forward-looking statements,
including, without limitation, statements related to the Company’s
expected timing of, protocols for and data results from, trials and
clinical studies involving its product candidates; potential market
opportunity for BXCL501; the potential for the results from the
Company’s completed, ongoing and proposed clinical trials to
support regulatory approvals for its product candidates; its
ongoing strategy for IGALMI; the Company’s current patent
applications; expected cash runway and cash burn rates;
developments relating to the TRANQUILITY and SERENITY programs; and
the expected outcomes of further discussions with the FDA. When
used herein, words including “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; the impact of the reprioritization; its significant
indebtedness, ability to comply with covenant obligations and
potential payment obligations related to such indebtedness and
other contractual obligations; the Company has identified
conditions and events that raise substantial doubt about its
ability to continue as a going concern; its limited experience in
drug discovery and drug development; risks related to the
TRANQUILITY program; 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; our estimates of episodes of agitation and
total addressable market are subject to inherent challenges and
uncertainties; the Company still faces extensive and ongoing
regulatory requirements and obligations for IGALMI; 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; risks associated with the increased scrutiny relating to
environmental, social and governance (ESG) matters; risks
associated with federal, state or foreign health care “fraud and
abuse” laws; and its ability to commercialize its product
candidates, as well as the important factors discussed under the
caption “Risk Factors” in its Quarterly Report on Form 10-Q for the
quarterly period ended September 30, 2023, as such factors may be
updated from time to time in its other filings with the SEC,
including without limitation its Annual Report on Form 10-K for the
fiscal year ended December 31, 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
CorporateBioXcel Therapeutics Erik Kopp
1.203.494.7062ekopp@bioxceltherapeutics.com
Investor Relations BioXcel Therapeutics Brennan Doyle
1.475.355.8462bdoyle@bioxceltherapeutics.com
MediaRusso PartnersDavid Schull
1.858.717.2310David.schull@russopartnersllc.comScott
Stachowiak1.646.942.5630Scott.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 © 2024, BioXcel Therapeutics, Inc. All rights
reserved.
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BioXcel Therapeutics,
Inc. |
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Statements of
Operations |
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(Unaudited, in
thousands, except per share amounts) |
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Three months ended
December 31, |
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Year ended December
31, |
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2023 |
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2022 |
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2023 |
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2022 |
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Revenues |
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Product
revenues |
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$ |
376 |
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$ |
238 |
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$ |
1,380 |
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$ |
375 |
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Operating
expenses |
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|
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Cost of
goods sold |
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|
714 |
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|
9 |
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|
1,260 |
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|
20 |
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Research and
development |
|
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9,934 |
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|
32,459 |
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|
84,326 |
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|
|
91,239 |
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Selling,
general and administrative |
|
|
9,603 |
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|
|
20,664 |
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83,413 |
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68,761 |
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Restructuring costs |
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- |
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- |
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4,163 |
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- |
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Total
operating expenses |
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20,251 |
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|
53,132 |
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173,162 |
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|
160,020 |
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Loss from
operations |
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|
(19,875 |
) |
|
|
(52,894 |
) |
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|
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(171,782 |
) |
|
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(159,645 |
) |
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Other
(income) expense |
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Interest
expense, net |
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3,435 |
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|
2,921 |
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13,314 |
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|
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8,213 |
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Interest
income |
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|
(946 |
) |
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|
(1,487 |
) |
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|
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(5,649 |
) |
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|
(2,528 |
) |
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Other
(income) expense, net |
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|
(108 |
) |
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|
480 |
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|
|
|
(394 |
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|
|
427 |
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Net loss and
comprehensive loss |
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$ |
(22,256 |
) |
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$ |
(54,808 |
) |
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$ |
(179,053 |
) |
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$ |
(165,757 |
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Net loss per
share - basic and diluted |
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$ |
(0.76 |
) |
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$ |
(1.95 |
) |
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$ |
(6.15 |
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$ |
(5.92 |
) |
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Weighted average shares outstanding - basic and diluted |
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29,449 |
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28,068 |
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29,129 |
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28,015 |
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Condensed Balance
Sheets |
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(Unaudited, in
thousands) |
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December 31, |
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December 31, |
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2023 |
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2022 |
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Cash and
cash equivalents |
|
$ |
65,221 |
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$ |
193,725 |
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Total
assets |
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$ |
73,702 |
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$ |
205,853 |
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Total
liabilities |
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$ |
130,210 |
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$ |
129,078 |
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Total
stockholders' equity (deficit) |
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$ |
(56,508 |
) |
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$ |
76,775 |
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