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1 BioXcel Therapeutics, 555 Long Wharf Drive, New Haven, CT 06511 | www.bioxceltherapeutics.com
Next Wave of Medicines August 2019 1 Proprietary & - - PowerPoint PPT Presentation
(NASDAQ: BTAI) Next Wave of Medicines August 2019 1 Proprietary & Confidential BioXcel Therapeutics, 555 Long Wharf Drive, New Haven, CT 06511 | www.bioxceltherapeutics.com Forward-Looking Statements This presentation includes
Proprietary & Confidential
1 BioXcel Therapeutics, 555 Long Wharf Drive, New Haven, CT 06511 | www.bioxceltherapeutics.com
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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 that relate to the advancement and development of BXCL501 and BXCL701, the commencement of clinical trials, the availability and results of data from clinical trials, BioXcel Therapeutic, Inc.'s (“BTI”) submission of its first New Drug Application with the FDA and other information that is not historical information. 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 BTI's current expectations and various assumptions. BTI believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. BTI 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 BXCL501 and BXCL701 and other product candidates; it ability to receive regulatory approval for its product candidates; its ability to enroll patients in its clinical trials; its approach to the discovery and development of product candidates based on EvolverAI is novel and unproven; its exposure to patent infringement lawsuits; its ability to comply with the extensive regulations applicable to it; 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 period ended March 31, 2019 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
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 BTI's views as of any date subsequent to the date of this presentation.
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Registration Filings Candidate Validation Human Proof Of Concept Selection of Best Candidates
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(DPP 8/9 & FAP Inhibitor)
(triple combination)
(Selective α2a Adrenergic Receptor Agonist)
initiation (4Q 2019)
readout (1H 2020)
submission (2H 2020)
(2H 2019)
(1H 2020)
*Bioavailability (BA) study for optimizing BXCL501 sublingual thin film dose for Phase 3 registration trials
Phase 1b Completed
expansion (2019)
Opioid Withdrawal, Delirium Exploring Multiple Tumor Types
Proof of Concept Trial Initiating
(double combination)
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Consensus Opinion* Treatment Options
#Dexmedetomidine *Martinez-Raga, J., et al. (2018). "1st International Experts' Meeting on Agitation: Conclusions Regarding the Current and Ideal Management Paradigm of Agitation."
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0 min 60 min 120 min 180 min 240 min 300 min 360 min
2:1 Randomization (n = 135)
n=90 n=45
* Patients Dosed
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Drug/Dose #
% Responders (Reduction in PEC of ≥ 40%) Mean Change in PEC Score P-Value Placebo N=36 28%
BXCL501 (180 mcg) N=18 89%
< 0.0001 BXCL501 (120 mcg) N=18 67%
0.0003 BXCL501 (80 mcg) N=18 56%
0.0152 BXCL501 (60 mcg) N=18 39%
0.1227
*The lowest dose tested, 20 mcg (not shown) was repeated in subjects who did not achieve response criterion
Time = 120 Min (Primary Endpoint)
60 120 180 240 300 360
Time Post Dose (Minutes)
** *** *** *** *** *
Placebo 180 mcg 80 mcg
* * * * ** *
* = p < 0.05 ** = p < 0.001 *** = p < 0.0001
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Drug/Dose # Mean Change in ACES Score From Baseline P-Value Placebo N=36 1.20 BXCL501 (180 mcg) N=18 3.94 < 0.0001 BXCL501 (120 mcg) N=18 3.11 0.0005 BXCL501 (80 mcg) N=18 2.33 0.0156 BXCL501 (60 mcg) N=18 2.11 0.0750
*The lowest dose tested, 20 mcg (not shown) was repeated in subjects who did not achieve response criterion The ACES consists of a single item that rates overall agitation and sedation at the time of evaluation, where 1 indicates marked agitation; 2, moderate agitation; 3, mild agitation; 4, normal behavior; 5, mild calmness; 6, moderate calmness; 7, marked calmness; 8, deep sleep; and 9, unarousable.
Placebo 80 mcg 180 mcg 1 2 3 4 5
Dose Change in ACES Score from Baseline (Least Squares Mean) p = 0.0156 p = <0.0001
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1:1 Randomization (n = 600-700)
0 min 60 min 120 min 180 min 240 min 300 min 360 min
* Patients Dosed
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2 4 6 8 10 12 14 16 18 20 AT RISK WITH AGITATION MILD TO MODERATE AGITATION
Schizophrenia Bipolar Disorder Dementia Opioid Use Disorder Delirium
Sources: Internal Company Estimates
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Phase 3: Schizophrenia & Bipolar Disorder
(Symptoms)
NDA Submission Phase 1b: Schizophrenia
(Acute Agitation)
Commercial Launch
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(1) Nature Chemical Biology, volume 13, pages 46–53 (2017)
Validation of AI Approach1
Pancreatic Cancer Melanoma
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YES Activity
Stop
NO
(N=30)
BXCL701 NKTR-214 Avelumab
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development planning during Phase 2 Focus on EU and Japan
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Combination Trial Initiated (BXCL701+Keytruda)
Mechanism Trial Initiated Triple Combination Trial Initiated
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Geoff Meacham (Barclays) Carter Gould (UBS) Do Kim (BMO Capital Markets) Sumant Kulkarni (Canaccord Genuity) Ram Selvaraju (H.C. Wainwright)
* As of July 2019 * As of March 2019
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CEO & Member of Board
Chief Scientific Officer
Chief Medical Officer
Chief Financial Officer
Senior Vice President, Head of Translational Medicine
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SHEILA GUJRATHI
CEO, Gossamer Bio
PETER MUELLER
Chairman of Board
STEVE LAUMAS
Member of Board
KRISHNAN NANDABALAN
Member of Board
MICHAL VOTRUBA
Member of Board
STEVEN PAUL
CEO, Karuna Therapeutics
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John Krystal, M.D. Chair, Department of Psychiatry Maurizio Fava, M.D. Director, Division of Clinical Research Thomas Laughren, M.D. Director, Regulatory Affairs Sheldon H. Preskorn, M.D. Professor of Psychiatry Thomas Kosten, M.D. Director, Division of Alcohol and Addiction Psychiatry Stephen R. Marder, M.D. Director, Section on Psychosis George Grossberg, M.D. Director, Geriatric Psychiatry Alan Breier, M.D. Professor of Psychiatry, Vice-Chair for Clinical Research
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Director, Georgetown Lombardi Comprehensive Cancer Center
Physician in Chief, Distinguished Professor at the TGen
Chief, Division of Hematology/Oncology
Associate Professor of Oncology and Urology
Head, Division of Clinical Studies
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