®

AI-Driven Transformative Medicines in Neuroscience and Immuno-oncology

41st Annual J.P. Morgan Healthcare Conference

January 11, 2023

BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | www.bioxceltherapeutics.com

NASDAQ: BTAI

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Forward-Looking Statements

This presentation includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995.Forward-looking statements in this presentation include but are not limited to: statements regarding BioXcel Therapeutics' expected timing of, and data results from, trials and clinical studies involving its product candidates; planned discussions with regulators; its commercial plan and strategy for IGALMI and strategic options for OnkosXcel; potential market size and opportunity for products and product candidates, and its future financial and operational results. When used herein, words including "anticipate," "being," "will," "plan," "may," "continue," and similar expressions are intended to identify forward-looking statements. 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 BioXcel Therapeutics' current expectations and various assumptions. BioXcel Therapeutics believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain.

BioXcel Therapeutics 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 limited experience in drug discovery and drug development; its dependence on the success and commercialization of IGALMI, BXCL501, BXCL502 and BXCL701 and other product candidates; the Company has no experience in marketing and selling drug products; 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; its exposure to patent infringement lawsuits; its ability to comply with the extensive regulations applicable to it; impacts from the COVID-19 pandemic; 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 September 30, 2022, 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.govand the Investors section of our website at www.bioxceltherapeutics.com.

These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this presentation. Any such forward-looking statements represent management's estimates as of the date of this presentation. While BioXcel Therapeutics 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 BioXcel Therapeutics' views as of any date subsequent to the date of this presentation.

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Indication and Important Safety Information

INDICATION

IGALMI is indicated for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. Limitations of Use: The safety and effectiveness of IGALMI have not been established beyond 24 hours from the first dose.

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.

The 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.

Patients 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.

Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatchor call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201-1088 or medinfo@bioxceltherapeutics.com.

Please see full Prescribing Information.

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Building a Unique Biopharmaceutical Business Model

Transformative Drug Re-innovation Approach Using AI

Targeting High Unmet Needs in Neuroscience and Immuno-oncology

  • Optimize R&D, accelerate development, increase probability of success

Neuroscience (BXCL501): FDA Approval

  • IGALMI™ (dexmedetomidine) sublingual film, acute treatment of agitation in schizophrenia and bipolar I and II disorder
  • Multiple indications for BXCL501, $15B market opportunity

Immuno-oncology (BXCL701): Human Proof of Concept

  • Unique innate immunity activator, turning cold tumors hot
  • Established OnkosXcel Therapeutics to maximize value

High-ValueNear-Term Catalysts

  • 2 pivotal readouts for BXCL501 in 1H23
  • Phase 2 readout for BXCL701 in 1Q23

Compelling Long-term Value

  • Integrated AI-drug development & commercialization capability to build a leading neuroscience company

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Disruption is in our DNA

Delivering

Disrupting

First oral film

$260m strategic

Poised to

potentially

innovation

drug development

treatment for

financing in

capture 139-

paradigm

BPD/SCZ agitation

April 2022

million-episode1

market

U.S. agitation

market

First public AI

IND to commercial

Built integrated

Advanced commercial

Bipolar disorders,

company focused on

launch of IGALMI™

commercial team and

schizophrenia

launch activities and clinical

neuroscience and

in under 4 years

implementing dynamic

& Alzheimer's-

pipeline development

Immuno-oncology

launch model

related agitation

(2018)

1 Data on file. BioXcel Therapeutics, Inc. New Haven, CT

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BioXcel Therapeutics Inc. published this content on 11 January 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 11 January 2023 17:46:11 UTC.