Full Press Release Details
BioXcel Therapeutics Aligns with FDA Recommendation
for Phase 3 Trial for TRANQUILITY Program, Provides Update on Strategic Financing, and Reports Third Quarter 2023 Financial Results
Company plans to conduct Phase 3 trial of BXCL501
in the at-home setting for TRANQUILITY program to support potential sNDA for acute treatment of agitation associated with dementia due
to probable Alzheimer's disease
Potential market opportunity for BXCL501 to
include acute treatment of agitation across full spectrum of Alzheimer's-related dementia and agitation severity across all care
Based on FDA feedback, Company plans to conduct
Phase 3 trial with 120 mcg (IGALMI approved dose) in the at-home setting for SERENITY III program for acute treatment of agitation
in bipolar disorders or schizophrenia
Key financial terms agreed with Oaktree Capital
Management and Qatar Investment Authority to enhance operational and financial flexibility
Conference call and webcast set for 8:00 a.m. ET
HAVEN, Conn., Nov. 14, 2023 - BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing
artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced alignment
with the FDA's recommendation for a Phase 3 trial in the TRANQUILITY program. In addition, the Company provided an update on strategic
financing activities that would enable greater operational flexibility and reported its financial results for the third quarter ended
"This is a time of tremendous progress and excitement for our
company and stakeholders, especially as we report a clinical development path forward for our TRANQUILITY program," said Vimal Mehta,
Ph.D., CEO of BioXcel Therapeutics. "I am proud of the teamwork that enabled us to turn our focus to much larger potential
opportunities for BXCL501 in at-home settings, beginning with agitation associated with Alzheimer's dementia. Furthermore, we believe
FDA feedback in the recent meeting focused on the SERENITY III program provides a clear path to develop BXCL501 for at-home use in the
treatment of agitation associated with schizophrenia and bipolar disorders. When we couple these positive developments with revised terms
with our strategic financing partners, we believe we have a strong foundation to deliver success."
Progress on Two Late-Stage Clinical Programs
Evaluating potential use of BXCL501 for acute treatment of agitation
associated with Alzheimer's dementia
| Received meeting minutes from October 11, 2023 FDA meeting. | ||
| Company aligned with FDA's recommendation to conduct additional Phase 3 trial of BXCL501 to evaluate safety and collect additional efficacy data in at-home setting. |
| The long term safety requirement for submission will be discussed with the FDA based on the observed frequency of agitation | ||
| TRANQUILITY III trial is no longer required for potential sNDA submission; Company plans to explore potential separate clinical development program for chronic agitation. |
Evaluating potential at-home use of BXCL501 for agitation associated
with bipolar disorders or schizophrenia
| Completed meeting with FDA on November 8, 2023; meeting minutes expected by the first half of December 2023. | ||
| Based on FDA feedback, Company plans to conduct a Phase 3 trial with 120 mcg (IGALMI approved dose) to evaluate safety in at-home setting. |
Strategic Financing Update: Oaktree
Capital Management and Qatar Investment Authority
IGALMI Commercialization
OnkosXcel Therapeutics
Third Quarter 2023 Financial Results
Revenue: Net revenue of IGALMI was approximately $341,000 for the quarter.
and Development (R&D) Expenses: R&D expenses were $19.6 million for the third quarter of 2023, compared to $22.1 million
for the same period in 2022. The decreased expenses were primarily attributable to a
decrease in expenses associated with the BXCL501 SERENITY III and TRANQUILITY II clinical trials.
General and Administrative (SG&A) Expenses: SG&A expenses were $24.3 million for the third quarter of 2023, compared
to $17.1 million for the same period in 2022. The increased expenses were primarily
attributable to an increase in one-time legal and professional fees, costs associated with the OnkosXcel potential public offering as
well as in personnel, and related costs to support commercialization of IGALMI in the U.S. prior to the Reprioritization.
Loss: BioXcel Therapeutics had a net loss of $50.5 million for the third quarter of 2023, compared to a net loss of $41.8 million
for the same period in 2022. The Company used $37.6 million in operating cash during the third quarter.
Cash and cash equivalents totaled $90.0
million as of September 30, 2023. The Company has entered into a binding term sheet to restructure key financial terms in its
agreements with Oaktree Capital Management and Qatar Investment Authority to provide for enhanced operational and financial flexibility,
including the potential to access additional tranches of capital. 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
Conference Call and Webcast
Therapeutics will host a conference call and webcast on Nov. 14, 2023, at 8:00 a.m. ET to provide an update on recent operational
highlights and to discuss its third quarter 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 February 14, 2024.
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
(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. |
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.
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.
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.
see full Prescribing Information at igalmi.com.
indications other than those approved by the FDA as IGALMI 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 studies across several
neuropsychiatric disorders. BXCL501 is under investigation 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 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 BioXcel Therapeutics, Inc.
BioXcel Therapeutics, Inc. (Nasdaq:
BTAI) is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. 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,