BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical
company utilizing artificial intelligence to develop transformative
medicines in neuroscience and immuno-oncology, today announced the
completion of patient enrollment in the safety lead-in portion of
the investigator-sponsored Phase 2 trial of BXCL701 in combination
with KEYTRUDA® (pembrolizumab) in previously treated
metastatic pancreatic ductal adenocarcinoma (PDAC). BioXcel
Therapeutics, through its OnkosXcel Therapeutics immuno-oncology
subsidiary, is collaborating with Georgetown Lombardi’s Dr. Louis
M. Weiner, director of the cancer center, and Dr. Benjamin
Weinberg, the study’s principal investigator. BioXcel Therapeutics
and Merck & Co. are providing BXCL701 and KEYTRUDA for the
trial, respectively.
The trial is evaluating BXCL701, an investigational, oral innate
immune activator designed to inflame the tumor microenvironment and
thereby augment the activity of checkpoint inhibitors. As part of
the trial’s safety lead-in, the first six patients have been
enrolled and will be observed for a six-week safety window period.
The trial is then expected to enroll approximately 39 patients in
its efficacy phase in a Simon 2-stage single-arm, open-label design
[19 + 20 patients]. The primary objective is to determine the
18-week progression-free survival rate. Patients will be monitored
radiographically and by tumor markers for response assessment.
Tumor biopsies and blood samples will also be collected over the
course of treatment to better understand the mechanism of how the
drug combination works in humans. The human proof of concept
portion of the trial is expected to start in H1 2024.
“Pancreatic cancer represents a significant unmet medical need,
ranking as the third-leading cause of cancer deaths1, yet remains
an exceptionally difficult cancer to treat. No novel therapies have
emerged in decades, and overall survival for advanced disease has
not improved in over 10 years,” said Dr. Weiner. “Building on
results from the preclinical work conducted in our lab, we are
excited to evaluate BXCL701 in this important trial as a potential
treatment for advanced pancreatic cancer.”
The American Cancer Society estimates that, in 2024,
approximately 66,440 cases of pancreatic cancer will be diagnosed
in the United States.2 Few therapeutic options are available for
patients with this indication, which has a five-year survival rate
of 13%, among the lowest of all cancers.3 Preclinical xenograft
models of pancreatic cancer demonstrated strong synergy between
BXCL701 and checkpoint inhibitors and reduced tumor growth and
promoted an increase in intratumoral T cells, macrophages and NK
cells, with induction of host-protective immunity. In addition,
preclinical studies showed that BXCL701 has the potential to
provide a marked anti-fibrotic effect, as seen in the tumor stroma.
These findings have been published in the Journal for ImmunoTherapy
of Cancer4, and recently presented at the 2023 annual meeting of
the Society for Immunotherapy of Cancer5
“The major challenge in immuno-oncology is cold tumors and their
lack of sensitivity to checkpoint therapy,” said Vincent J.
O’Neill, M.D., Executive Vice President, Chief of Product
Development and Medical Officer of BioXcel Therapeutics. “We have
already demonstrated encouraging response rates and survival data
in two aggressive forms of prostate cancer: small cell
neuroendocrine cancer (SCNC) and adenocarcinoma, as highlighted in
our Key Opinion Leader Day presentation last year. This Phase 2
trial marks the third cold tumor setting where we are testing
BXCL701 in combination with KEYTRUDA. Based on encouraging
preclinical results, we look forward to the results from the human
proof of concept efficacy trial evaluating BXCL701 in combination
with pembrolizumab in patients with PDAC.”
About BXCL701BXCL701 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
anticancer 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 and 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. 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 (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, the Company’s expected timing, trial design and data
results for clinical trials of BXCL701 with pembrolizumab and
potential benefits from treatment with BXCL701. 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 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; and its novel approach
to the discovery and development of product candidates based on
EvolverAI , as well as the other 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,
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 © 2024, BioXcel
Therapeutics, Inc. All rights reserved.
References
- American Cancer Society: 2024 Estimated Cancer Deaths.
https://cancerstatisticscenter.cancer.org/, accessed February 5,
2024
- American Cancer Society: Key Statistics for Pancreatic Cancer.
https://www.cancer.org/cancer/types/pancreatic-cancer/about/key-statistics.html,
accessed February 5, 2024
- American Cancer Society: 5-year relative survival rates for
pancreatic
cancer.https://www.cancer.org/cancer/types/pancreatic-cancer/detection-diagnosis-staging/survival-rates.html,
accessed February 5, 2024
- Fitzgerald AA, Wang S, Agarwal V, et al. DPP inhibition alters
the CXCR3 axis and enhances NK and CD8+ T cell infiltration to
improve anti-PD1 efficacy in murine models of pancreatic ductal
adenocarcinoma. Journal for ImmunoTherapy of Cancer 2021;9:e002837.
doi:10.1136/jitc-2021-002837).https://jitc.bmj.com/content/9/11/e002837.info,
accessed February 5, 2024
- Dipeptidyl peptidase 4 (DPP4) inhibition is not solely
responsible for the antitumor effects of BXCL701, an inhibitor of
multiple DPPs, in a murine model of pancreatic ductal
adenocarcinoma (PDAC). Alexander Lekan, Rachael Maynard, Zoe X.
Malchiodi, Annie Zuo, Sandra A. Jablonski, Veena Agarwal, Moses
Donkor, Vincent O’Neill, and Louis M. Weine. Poster presented at
the 38th Annual Meeting of the Society for Immunotherapy of Cancer,
November 01-05, 2023, San Diego, CA.
chrome-extension://efaidnbmnnnibpcajpcglclefindmkaj/https:/ir.bioxceltherapeutics.com/static-files/d10d0294-9156-4fe5-8e2a-5f8f665f2e75,
accessed February 5, 2024
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