BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical
company utilizing artificial intelligence to develop transformative
medicines in neuroscience and immuno-oncology, today announced
positive overall survival (OS) data from its Phase 2 trial of
BXCL701, the Company's investigational oral innate immune
activator, in combination with KEYTRUDA® (pembrolizumab) in
patients with metastatic castration-resistant prostate cancer
(mCRPC) of adenocarcinoma phenotype, the most common form of the
disease. As of a September 6, 2023 data cutoff, evaluable patients
with adenocarcinoma (n=29) showed a median OS of 15.5 months (95%
CI 9.6–NR), and a 12-month survival rate of 59%.
“Patients with mCRPC who have failed androgen deprivation and
taxane-based chemotherapy have few remaining treatment options and,
unfortunately, KEYTRUDA to date has not shown additional benefit in
this setting,” said Vincent J. O’Neill, M.D., Chief R&D
Officer, OnkosXcel Therapeutics, a wholly owned subsidiary of
BioXcel Therapeutics. “Therefore, we are highly encouraged by these
combination data bearing in mind historical data with checkpoint
inhibitor monotherapy. In addition, we now have a second positive
dataset in a separate cold tumor histology, further increasing our
belief that BXCL701 has the potential to inflame the tumor
microenvironment of cold tumors, thereby sensitizing them to
checkpoint inhibition. We believe the data warrant further
evaluation of BXCL701 in this setting and look forward to
determining the development path for this program following our end
of Phase 2 meeting with the FDA scheduled for December.”
In 2023 in the United States, there are expected to be an
estimated 288,3002 new patients with prostate cancer, which is
classified as a “cold” tumor. Of those, 20% are expected to advance
to mCRPC, a form of advanced prostate cancer that is no longer
responding to testosterone-lowering hormone treatments and has
spread to other areas of the body such as the lymph nodes, bones,
bladder, rectum, liver, or lungs. Approximately 80% of mCRPC cases
are of the adenocarcinoma phenotype, which represent approximately
46,128 patients.
In addition to the new OS data, the Company recently presented
an update on response rate data from the Phase 2 adenocarcinoma
cohort at the Prostate Cancer Foundation Annual Scientific Retreat.
The Company reported a RECIST partial response rate of 28% with a
median duration of response of 19 months. This is in contrast to a
RECIST response rate of 5% with a median duration of response of
16.8 months from the KEYNOTE-199 trial of pembrolizumab monotherapy
in a similar patient population.
The Company’s Phase 2 trial is an open-label, multicenter study
to evaluate the safety and efficacy of BXCL701 in combination with
pembrolizumab in men with mCRPC of adenocarcinoma phenotype as well
as in men with SCNC. Twenty-nine (29) evaluable adenocarcinoma
patients received 0.3 mg of BXCL701 twice daily (BID) on days 1
through 14 of a 21-day cycle (0.2 mg BID the first week of Cycle 1)
plus 200 mg of pembrolizumab administered intravenously on day 1
and every subsequent 21 days. The primary objective of the trial is
a composite response rate defined as either objective response by
RECIST 1.1 criteria and/or PSA50 and/or CTC count conversion.
Secondary objectives include duration of response, progression-free
survival, overall survival, and biomarker evaluation as measured by
changes in circulating cytokines and correlation of outcome with
baseline tumor characteristics.
The Company is continuing to actively evaluate strategic options
for OnkosXcel Therapeutics, including potential financing,
strategic partnership, or M&A.
About OnkosXcel Therapeutics, LLC and
BXCL701OnkosXcel Therapeutics, LLC is a wholly owned
subsidiary of BioXcel Therapeutics, Inc., focused on developing
transformative medicines in oncology utilizing artificial
intelligence. The subsidiary was formed in 2022 to develop
BXCL701.
BXCL701 is an investigational, oral innate immune activator
designed to initiate inflammation in the tumor microenvironment.
Approved and experimental immunotherapies often fail to address
cancers that appear “cold.” Therefore, BXCL701 is being evaluated
to determine if it can render “cold” tumors “hot,” making them more
detectable by the adaptive immune system and thereby facilitating
the development of a strong anti-cancer immune response. OnkosXcel
Therapeutics’ preclinical data support BXCL701’s potential synergy
with both current checkpoint inhibitors and emerging
immunotherapies directed to activate T-cells. BXCL701 is currently
being developed as a potential therapy for the treatment of
aggressive forms of prostate cancer and advanced solid tumors that
are refractory or treatment naïve to checkpoint inhibitors. BXCL701
has received Orphan Drug Designation from the U.S. Food & Drug
Administration in four indications: acute myelogenous leukemia,
pancreatic cancer, stage IIb to IV melanoma, and soft tissue
sarcoma. An 800+-subject clinical database, with data collected by
the Company and others, supports the ongoing development of
BXCL701.
About BioXcel Therapeutics, Inc.
BioXcel Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical
company utilizing artificial intelligence to develop transformative
medicines in neuroscience and 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 (the
“Securities Act”) and Section 21E of the Securities Exchange Act of
1934, as amended (the “Exchange Act”). All statements contained in
this press release other than statements of historical fact should
be considered forward-looking statements, including, without
limitation, statements regarding: the Company’s expected timing of,
trial design and data results from, future clinical trials of
BXCL701 with pembrolizumab, potential benefits from treatment with
BXCL701, the Company’s planned discussions with FDA, the future
clinical development of BXCL701, the Company’s plans to evaluate
strategic options for OnkosXcel Therapeutics and potential market
size and opportunity for product candidates. The words
“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; its ability to
successfully negotiate amended terms under the financing agreements
to be able to access funding and to obtain relief under financial
covenants; its significant indebtedness and potential payment
obligations related to such indebtedness and other contractual
obligations; risks associated with the strategic reprioritization;
its limited experience in drug discovery and drug development;
risks related to the TRANQUILITY II Phase 3 trial and related
audit; 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; 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; impacts from the COVID-19
pandemic; risks associated with the increased scrutiny relating to
environmental, social and governance (ESG) matters; 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 June 30, 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
Corporate
BioXcel TherapeuticsErik
Kopp1.203.494.7062ekopp@bioxceltherapeutics.com
Investor RelationsBioXcel TherapeuticsBrennan
Doyle1.475.355.8462bdoyle@bioxceltherapeutics.com
MediaRusso PartnersDavid
SchullT: 858-717-2310David.schull@russopartnersllc.comScott
StachowiakT: 646-942-5630Scott.stachowiak@russopartnersllc.com
Source: 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 © 2023, BioXcel Therapeutics,
Inc. All rights reserved.
1. FOR ILLUSTRATIVE PURPOSES ONLY: Analysis based on results
from Pembrolizumab KEYNOTE-199 study. Pembrolizumab for
Treatment-Refractory Metastatic Castration-Resistant Prostate
Cancer: Multicohort, Open-Label Phase II KEYNOTE-199 Study.
Emmanuel S. Antonarakis et al. Journal of Clinical Oncology;
published at ascopubs.org/journal/jco on November 27, 2019: DOI
https://doi.org/10.1200/JCO.19.01638. Pembrolizumab is not an
approved therapy for the treatment of any form of mCRPC, and no
head-to-head clinical trial has been conducted evaluating BXCL701
against pembrolizumab or other candidates or products. Notable
differences exist between the Company’s trial designs, conditions
under study and subject characteristics as compared to the
third-party results discussed above and caution should be exercised
when comparing data across these studies.
2. American Cancer Society. Key Statistics for Prostate Cancer.
Retrieved October 9, 2023.
https://www.cancer.org/cancer/types/prostate-cancer/about/key-statistics.html#:~:text=The%20American%20Cancer%20Society's%20estimates,34%2C700%20deaths%20from%20prostate%20cancer
BioXcel Therapeutics (NASDAQ:BTAI)
Historical Stock Chart
From Apr 2024 to May 2024
BioXcel Therapeutics (NASDAQ:BTAI)
Historical Stock Chart
From May 2023 to May 2024