Full Press Release Details
BioXcel Therapeutics Reports Positive Overall
Survival Results from Single-Arm, Open-Label Phase 2
Trial of BXCL701 in Patients with Small Cell Neuroendocrine Prostate Cancer
overall survival of 13.6 months with BXCL701 + KEYTRUDA
(pembrolizumab), compared to 7.6 months with checkpoint inhibitor monotherapy (CPI) in late-line refractory patients
in separate, Phase 2 trial1
56% of studied patients alive at one year following
treatment with BXCL701 + KEYTRUDA , compared to ~33% with checkpoint inhibitor
monotherapy (CPI) in late-line refractory patients in separate, Phase 2 trial1
support potential development in small cell lung cancer (SCLC) and other neuroendocrine tumors
Company to host conference call and webcast
today at 8:00 a.m. ET
HAVEN, Conn., Oct. 10, 2023 - 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%.
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.
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.
BioXcel 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.
OnkosXcel Therapeutics, LLC and BXCL701
OnkosXcel 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 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 na ve 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
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