Bioxcel Therapeutics, Inc. · NASDAQ:BTAI
Bioxcel Therapeutics — Investor Presentation (December 2019)
26 slides from Bioxcel Therapeutics's investor presentation, December 2019
Slide 1: Next Wave of Medicines
(NASDAQ: BTAI) Next Wave of Medicines December 2019 BioXcel Therapeutics, 555 Long Wharf Drive, New1 Haven, CT 06511 | www.bioxceltherapeutics.com Proprietary & Confidential
Slide 2: Forward-Looking Statements
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
Slide 3: BioXcel Therapeutics Investment Highlights
BioXcel Therapeutics Investment Highlights Neuro Symptoms Based Approach Innate Immunity Based Approach BXCL501 BXCL701 Sublingual Thin Film for Acute Targeting Rare Cancers Treatment of Agitation • Phase 3 Pivotal Trial Initiation Expected • Phase 1b/2 double combination − 4Q 2019 trial in Neuroend
Slide 4: Unleashing the Power of AI Across the Entire R&D Value Chain
Unleashing the Power of AI Across the Entire R&D Value Chain 4-5 Year Development Cycle NDA R E G U L ATO R Y T E A M 4 Registration Filings CLINICAL DEVELOPMENT TEAM 3 Human Proof Of Concept BXCL501 BXCL701 T R A N S L AT I O N A L T E A M 2 Candidate Validation M U LT I P L E C A N D I D AT E S Se
Slide 5: Pipeline To Rapid Human PoC and Development Path
Pipeline To Rapid Human PoC and Development Path Anticipated Worldwide Program Product Candidate Phase 1/2 Phase 2/3 Milestones Rights • Phase 3 schizo/bipolar Phase 1b − Trial initiation (4Q 2019) Completed Schizophrenia/Bipolar Treatment of BXCL501 − Data readout (mid-2020) − NDA submission (2H 20
Slide 6: Clinical Programs
Clinical Programs BXCL501: Potential First in Class Sublingual Thin Film Dexmedetomidine (Dex) for Acute Treatment of Agitation Proprietary & Confidential 6
Slide 7: Agitation is an Unmet Medical Need
BXCL501 Agitation is an Unmet Medical Need • Common and expensive phenomenon ~19M associated with multiple psychiatric At Risk conditions – 8.3 million suffer each year in the US – $40 billion per year health care burden 8.3M Suffer Agitation • Rapid symptom relief with a non-invasive approach is de
Slide 8: Proprietary Sublingual Thin Film of Dex for Acute Treatment of Agitation
BXCL501 Proprietary Sublingual Thin Film of Dex* for Acute Treatment of Agitation Current Treatments are Suboptimal: Treatment Options • Dementia: Antipsychotic drugs have black-box warning for elderly • Psychiatric: Invasive with severe side effects Fast Track Designation BXCL501: Novel Mechanism o
Slide 9: Proprietary Formulation and Scale Up Manufacturing
BXCL501 Proprietary Formulation and Scale Up Manufacturing 118 subjects dosed: Healthy volunteers (28) and Agitated Schizophrenia patients (90) • Transitioned to Registrational Drug Product Process – Manufacturing phase 3 / registrational batches – Commercial scale-up planned for product launch • Pr
Slide 10: Phase 1b Clinical Trial in Agitated Schizophrenia Patients
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 * n=90 BXCL501 (20, 60, 80, 120, 180 mcg) Agitated ✔ Initiated May 2019 → Schizophrenia Screening R 2:1 Ra
Slide 11: Primary Endpoint: Change in PEC Score from Baseline
BXCL501 Primary Endpoint: Change in PEC Score from Baseline Clinically meaningful, rapid and durable responses Change in PEC Score from Baseline 0 -2 (Least Squares Mean) -4 Placebo -6 * * * -8 * * 80 mcg ** * * = p < 0.05 -10 ** ** = p < 0.001 *** 180 mcg *** = p < 0.0001 *** *** *** -12 0 60 120 1
Slide 12: Secondary Evaluation: Change in ACES from Baseline
BXCL501 Secondary Evaluation: Change in ACES from Baseline Consistent with primary endpoint Mean Change in Drug/Dose # ACES Score From P-Value 5 p = <0.0001 Baseline Change in ACES Score from Baseline Placebo N=36 1.20 4 p = 0.0156 (Least Squares Mean) BXCL501 (180 mcg) N=18 3.94 < 0.0001 3 BXCL501
Slide 13: Phase 1b: Safety and Tolerability
BXCL501 Phase 1b: Safety and Tolerability • Well-tolerated with no serious or severe adverse events across the entire dose range tested. • Most common treatment-related adverse events were mild somnolence and dry mouth. • A maximum tolerated dose was not reached. • All subjects (100%) were able to s
Slide 14: Phase 3 Pivotal Trial: Adaptable Design
BXCL501 Phase 3 Pivotal Trial: Adaptable Design SERENITY: Sublingual DExmedetomidine in Agitation Associated with SchizophRENIa and Bipolar Disorder STudY Evaluation Period (6 hours) 0 min 60 min 120 min 180 min 240 min 300 min 360 min * n=250 BXCL501 (120 & 180 mcg) SERENITY I: Agitated Schizophren
Slide 15: Potential To Capitalize on Large US Commercial Opportunity
BXCL501 Potential To Capitalize on Large US Commercial Opportunity • 5 Million Patients With Mild to Moderate Agitation 20 18.9M • Multiple Episodes Per Year 18 16 14 Patients (Millions) 0.53 Schizophrenia 12 10 1.34 Bipolar Disorder 8.3M 8 Dementia 6 5M 1.71 Delirium 4 0.94 2 Opioid Withdrawal 0.45
Slide 16: Care Centers for Neuropsychiatric Indications
BXCL501 Care Centers for Neuropsychiatric Indications Broad Potential for Use Across Multiple Clinical Environments Emergency Departments Urgent Care Centers General | Psychiatric BXCL501 Hospitals Mental Health Clinics Medical + Neuro + Psych Wards Crisis Care Psychiatric Hospitals Proprietary & Co
Slide 17: Key Targeted Milestones for Value Creation BXCL501
BXCL501 Key Targeted Milestones for Value Creation 3Q 2019 4Q 2019 mid-2020 2H 2020 2021 BXCL501 Anticipated Timeline Schizophrenia / Phase 1b Data Phase 3 Pivotal Phase 3 Data First NDA Commercial Bipolar Disorder Announced Trial Initiation Readout Submission Launch (July 22, 2019) Expected Proprie
Slide 18: Pipeline in a Product and Agitation Franchise Opportunity
BXCL501 Pipeline in a Product and Agitation Franchise Opportunity Severe Agitation/ Pre-Agitation Mild to Moderate Agitation Aggression BXCL501 (Sublingual Thin Film) BXCL501 + Wearable KalmPen™ Device Combo + Schizophrenia/Bipolar (NDA) Prophylaxis or IM Formulation Prevention of Agitation Dementia
Slide 19: Clinical Programs
Clinical Programs BXCL701: Potential First-in-Class Oral IO Therapy Targeting Pancreatic Cancer and tNEPC Proprietary & Confidential 19
Slide 20: BXCL701 Oral I/O Therapy
BXCL701 Oral I/O Therapy • Pancreatic Cancer Investigational Orally Administered Activator • Melanoma • Acute Myeloid of Systemic Innate Immunity Pathway Leukemia Dual MoA Designed to Inhibit DPP 8/9 & FAP Validation of AI Approach1 Observed Proof of Mechanism in Clinical Studies (1) Nature Chemical
Slide 21: BXCL701 Human Proof of Concept & Mechanism of Action
BXCL701 Human Proof of Concept & Mechanism of Action BXCL701 Human Proof of Concept ✓ Single Agent Activity in Melanoma >700 ✓ ~10% Response Rate (CR/PR) Patient Data Inflammasome Caspase-1 DPP 8/9 BXCL701 FAP Breaks Fibrotic Barrier Pyroptosis & IL-18 release Proprietary & Confidential 21
Slide 22: tNEPC Clinical Development Plan: Combination with Keytruda
BXCL701 tNEPC Clinical Development Plan: Combination with Keytruda Safety Run-in Safety/PD/Immune-phenotyping Phase 2 Expansion Global* (N=6) (N=30) Pivotal Study *Expect to commence global development planning during Phase 2 Focus on EU and Japan ✔ Multiple Patients Dosed Rahul Aggarwal, M.D. Divis
Slide 23: tNEPC Phase 1b – Safety Run-In Summary
BXCL701 tNEPC Phase 1b – Safety Run-In Summary • Cohort 1: Enrollment and DLT Evaluation Period Complete – BXCL701 (0.4 mg/day) + Keytruda® – Well-tolerated with no SAEs or DLTs – Preliminary pharmacokinetics of BXCL701 are within expectations based on prior data • Cohort 2: Enrollment Complete – BX
Slide 24: Pancreatic Cancer: Mechanistic and Triple Combination Trial
BXCL701 Pancreatic Cancer: Mechanistic and Triple Combination Trial 2 Weeks of BXCL701 Demonstration of Immune Cell Proof of Mechanism Trial Treatment Before Surgery Infiltration/Activation to (Pre and Post Tissue Available (N=10-15)) Validate MoA BXCL701 Stop NKTR-214 Avelumab NO Efficacy Trial in
Slide 25: Key Targeted Milestones for Value Creation BXCL701
BXCL701 Key Targeted Milestones for Value Creation 2H 2018 1H 2019 2H 2019 1H 2020 2H 2020 Beyond BXCL701 Anticipated Timeline Neuroendocrine Combination Sites Opening & Trial Initiated Registration Prostate Cancer (BXCL701+Keytruda) Recruitment tNEPC Data Readout Trial Ongoing (tNEPC) Triple Combin
Slide 26: Thank You
Dr. Vimal Mehta, CEO BioXcel Therapeutics, New Haven, CT 06511 [email protected] THANK YOU! Proprietary & Confidential 26
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