(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 “ 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 presentation. Any such forward-looking statements represent management's estimates as of the date of this presentation. While BTI 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 BTI's views as of any date subsequent to the date of this presentation. 2
BioXcel Therapeutics Investment Highlights Neuro Symptoms Based Approach Innate Immunity Based Approach BXCL701 BXCL501 Targeting Rare Cancers Sublingual Thin Film for Acute Treatment of Agitation Clinical Partnerships Phase 3 Pivotal Trial Initiation • 3 Ongoing Trials • − 4Q 2019 − Pancreatic Cancer: ( 2 trials ) Phase 3 Data Readout • − Neuroendocrine Prostate Cancer ( tNEPC ) − 1H 2020 AI-powered Drug Development • Data Readouts Expected in 2H 2019 and 1H 2020 • Improves R&D Economics • Increases Development Efficiency • Maximizes Probability of Success 3 Proprietary & Confidential
Unleashing the Power of AI Across the Entire R&D Value Chain NDA 4-5 Year Development Cycle R E G U L A T O R Y T E A M 4 Registration Filings C L I N I C A L D E V E L O P M E N T T E A M 3 Human Proof Of Concept BXCL501 BXCL701 T R A N S L A T I O N A L T E A M 2 Candidate Validation M U L T I P L E C A N D I D A T E S 1 Selection of Best Candidates AI Powered Drug Development 4 Proprietary & Confidential
Pipeline To Rapid Human PoC and Development Path Anticipated Worldwide Program Phase 1/2 Product Candidate Phase 2/3 Milestones Rights • Phase 3 schizo/bipolar trial Phase 1b initiation ( 4Q 2019 ) Schizophrenia/Bipolar Completed BXCL501 • Phase 3 schizo/bipolar data Treatment of readout ( 1H 2020 ) Acute Agitation ( Selective α 2a Adrenergic • Schizo/bipolar NDA Proof of Concept submission ( 2H 2020 ) Receptor Agonist ) Geriatric Dementia Trial Initiating • Dementia trial initiation ( 2H 2019 ) Neuroendocrine • tNEPC data readouts ( 2H 2019 ) Prostate Cancer ( tNEPC ) • Pancreatic data readouts BXCL701 Immuno- ( double combination ) ( 1H 2020 ) Oncology ( DPP 8/9 & FAP Inhibitor ) Pancreatic Cancer ( triple combination ) BXCL501 Opioid Withdrawal, Delirium Pipeline • New indications & geography Expansion expansion ( 2019 ) BXCL701 Exploring Multiple Tumor Types Additional Discovery Through an Future Programs Exclusive AI Relationship with BioXcel Corporation ( parent ) *Bioavailability ( BA ) study for optimizing BXCL501 sublingual thin film dose for Phase 3 registration trials 5 Proprietary & Confidential
Clinical Programs BXCL501: First in Class Sublingual Thin Film Dexmedetomidine ( Dex ) for Acute Treatment of Agitation 6 Proprietary & Confidential
BXCL501 Agitation is an Unmet Medical Need • Common and expensive phenomenon associated ~19M At Risk with multiple psychiatric conditions − 8.3 million suffer each year in the US − $40 billion per year health care burden Suffer 8.3M Agitation • Rapid symptom relief with a non-invasive approach is desired Mild to Moderate 5M • Patients experience multiple episodes per year Agitation 7 Proprietary & Confidential
BXCL501 Proprietary Sublingual Thin Film of Dex # for Acute Treatment of Agitation Current Treatments are Suboptimal: Treatment • Dementia: Antipsychotic drugs ( black-box warning ) for elderly Options • Psychiatric: Invasive with severe side effects • Non-invasive • Non-traumatic / non-coercive • Calmness without sedation • Good safety profile Consensus Fast Track Opinion * • Easy to administer • Favorable tolerability Designation • Rapid onset • Patient preference BXCL501: Novel Mechanism of Action ( MoA ) ü Non-Invasive, easy to administer sublingual thin film designed for rapid onset of action #Dexmedetomidine *Martinez-Raga, J., et al. (2018). "1st International Experts' Meeting on Agitation: Conclusions Regarding the Current and Ideal Management Paradigm of Agitation." 8 Proprietary & Confidential
BXCL501 Proprietary Formulation and Scale Up Manufacturing 118 subjects dosed: Healthy volunteers (28) and Agitated Schizophrenia patients (90) • Transitioned to Automated Manufacturing for Scale Up − GMP manufacturing initiated − Scale up and supply phase 3 in 2H 2019 and commercial readiness in 2020 • Extensive Formulation Development Completed for Clinical Studies − Multiple dose strengths − Immediate release film with muco-adhesion properties − Proprietary technology delivers low dose ranges 9 Proprietary & Confidential
BXCL501 Predictable and Dose Proportional PK in Phase I Study Phase I clinical studies in 42 healthy volunteers 1000 Mean Concentration (ng/L) 100 40 µg 10 20 µg 10 µg 1 0.1 28 active : 14 placebo 0 1 2 3 4 5 6 7 8 Time (hours) 10 Proprietary & Confidential
BXCL501 Phase 1b Clinical Trial in Agitated Schizophrenia Patients Assessing Agitation Episodes in Schizophrenia Evaluation Period ( 6 hours ) 0 min 60 min 120 min 180 min 240 min 300 min 360 min * BXCL501 n=90 ( 20, 60, 80, 120, 180 mcg ) Agitated Screening R Schizophrenia 2:1 Randomization (n = 135) Period Patients Placebo n=45 Primary Endpoint: Change from baseline in PEC score ( PANSS-Excitatory Component ) ✔ Initiated May 2019 → ✔ Completed July 2019 * Patients Dosed 11 Proprietary & Confidential
BXCL501 Primary Endpoint: Change in PEC Score from Baseline Change in PEC Score from Baseline 0 -2 ( Least Squares means ) -4 Placebo -6 * * * 80 mcg -8 * * ** * * = p < 0.05 ** ** = p < 0.001 -10 180 mcg *** = p < 0.0001 *** *** *** *** -12 0 60 120 180 240 300 360 Time Post Dose (Minutes) % Responders ( Reduction in PEC of ≥ 40% ) Mean Change in PEC Score P-Value Drug/Dose # Placebo N=36 28% -4.5 BXCL501 ( 180 mcg ) N=18 89% -10.8 < 0.0001 Time = 120 Min BXCL501 ( 120 mcg ) N=18 67% -9.2 0.0003 ( Primary Endpoint ) BXCL501 ( 80 mcg ) N=18 56% 0.0152 -7.1 BXCL501 ( 60 mcg ) N=18 39% -6.0 0.1227 *The lowest dose tested, 20 mcg ( not shown ) was repeated in subjects who did not achieve response criterion 12 Proprietary & Confidential
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