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 small cell neuroendocrine prostate cancer (SCNC). As
of a data cutoff of September 6, 2023, evaluable patients with SCNC
(n=28) showed a median OS of 13.6 months (95% CI 10.9–NR), and a
12-month survival rate of 56.5%.
“OS is the most meaningful measure by which the effectiveness of
an oncology treatment is evaluated. Though these results are based
on a non-randomized cohort of patients, observing a median OS of
this duration including patients with long-term survival at 12
months and beyond shows exceptional promise, bearing in mind
historic data with checkpoint inhibitor monotherapy in this
high-risk subset of prostate cancer,” said Rahul Aggarwal, M.D.,
Principal Investigator, Associate Director for Clinical Sciences,
Helen Diller Family Comprehensive Cancer Center, and Professor of
Medicine at the University of California San Francisco (UCSF).
“SCNC represents a major unmet medical need, with the majority of
patients unfortunately succumbing to their disease in less than one
year following chemotherapy. The results of this trial suggest that
BXCL701 has the potential to extend the lives of patients, and I
look forward to its continued clinical development.”
SCNC, classified as a “cold” tumor, represents an underserved,
growing patient population, with cases increasing due to earlier
and more widespread use of androgen receptor inhibitors. In 2023,
there will be an estimated 288,3002 new patients with prostate
cancer in the United States, with approximately 11,532 patients
progressing to SCNC.
“We believe our trial results are highly encouraging for
patients with this disease and have potential implications for our
evaluation of BXCL701 for the treatment of other high-grade
neuroendocrine tumors, such as small cell lung cancer, where
effective therapies are lacking,” said Vincent J. O’Neill, M.D.,
Chief R&D Officer, OnkosXcel Therapeutics, a wholly owned
subsidiary of BioXcel Therapeutics. “We intend to discuss these
data with the FDA to help determine our next steps with clinical
development.”
The Phase 2 trial is an open-label, multicenter study to
evaluate the safety and efficacy of BXCL701 in combination with
pembrolizumab in men with SCNC. Twenty-eight (28) evaluable SCNC
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.
Conference CallBioXcel
Therapeutics will host a conference call and webcast on October 10,
2023 at 8:00 a.m. ET to discuss the data results from the Phase 2
trial of BXCL701. To access the call, please dial 877-407-5795
(domestic) or 201-689-8722 (international). A live webcast and
presentation materials will be available on the Investors section
of the corporate website, bioxceltherapeutics.com, and a
webcast replay will be available through January 10, 2024.
BioXcel Therapeutics may use its website as a distribution
channel of material information about the Company. Financial and
other important information 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 Metastatic Castration-Resistant Prostate Cancer
(mCRPC) and Treatment-Emergent Small Cell Neuroendocrine Prostate
Cancer (SCNC)mCRPC is 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.
Treatment-emergent SCNC is a particularly difficult-to-treat
histologic subtype of mCRPC that emerges in approximately 20% of
mCRPC patients, though this number is increasing due to earlier and
more widespread use of androgen blockers.
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 (FDA) in four indications: acute myelogenous
leukemia, pancreatic cancer, stage IIb to IV melanoma, and soft
tissue sarcoma. An approximately 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 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
CorporateBioXcel TherapeuticsErik
Kopp1.203.494.7062ekopp@bioxceltherapeutics.com
Investor RelationsBioXcel TherapeuticsBrennan
Doyle1.475.355.8462bdoyle@bioxceltherapeutics.com
MediaRusso PartnersScott
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 © 2023, BioXcel Therapeutics,
Inc. All rights reserved.
1 FOR ILLUSTRATIVE PURPOSES ONLY: Analysis based on results from
avelumab PICK-NEPC study. Landon C. Brown et al. Prostate Cancer
and Prostatic Diseases; https://doi.org/10.1038/s41391-022-00524-7.
No head-to-head clinical trial has been conducted evaluating
BXCL701 against avelumab or other candidates or products. Notable
differences exist between the Company's trial designs, conditions
under study and subject characteristics as compared to the
evaluated third-party Phase 2 results discussed above and caution
should be exercised when comparing data across these studies. One
year survival rate extrapolated by the Company using Kaplan-Meier
estimates of overall survival curve from the PICK-NEPC study.
Please see the “Appendix” of the presentation materials for the
conference call being held today, October 9, 2023, for information
regarding the differences between the clinical trials.
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
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