Bioxcel Therapeutics, Inc. · NASDAQ:BTAI
Bioxcel Therapeutics — Investor Presentation (March 2019)
35 slides from Bioxcel Therapeutics's investor presentation, Mar 12, 2019
Slide 1: Safe Harbor Statement
Safe Harbor Statement This document may contain forward-looking statements. Such forward-looking statements are characterized by future or conditional verbs such as “may,” “will,” “expect,” “intend,” “anticipate,” believe,” “estimate” and “continue” or similar words. You should read statements that
Slide 2: BioXcel Therapeutics Investment Highlights
BioXcel Therapeutics Investment Highlights Developing high value therapeutics in neuroscience and immuno-oncology utilizing a novel artificial intelligence platform BXCL501 First-in-Class Sublingual Thin Film for Acute Treatment of Agitation BXCL701 First-in-Class Targeting Rare Cancers First Clinic
Slide 3: BTI is Unleashing the Power of AI Across the Entire R&D Value Chain
BTI is Unleashing the Power of AI Across the Entire R&D Value Chain Opportunity to generate multiple NDAs N D A R E G U L A T O R YT E A M 4-5 Year Development Cycle 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
Slide 4: Pipeline: Rapid Human PoC and Development Path
BioXcel Therapeutics Pipeline: Rapid Human PoC and Development Path First-in-class neuroscience and immuno-oncology pipeline with multiple near-term milestones Program Worldwid Rights BA study initiated with BXCL501 (4Q 2018) • BA study data readout (1H 2019) • Launch registration trials (2019) Schi
Slide 5: Clinical Programs BXCL501: First in Class Sublingual Thin Film Dexmedetomidine
Clinical Programs BXCL501: First in Class Sublingual Thin Film Dexmedetomidine (Dex) for Acute Treatment of Agitation 6 Proprietary & Confidential
Slide 6: BXCL501: Sublingual Thin Film Dexmedetomidine for Acute Treatment of Agitation
BXCL501: Sublingual Thin Film Dexmedetomidine (Dex) for Acute Treatment of Agitation Rapid clinical development and regulatory approval path (505(b)(2)) Agitation: A growing global healthcare issue ($40B+) Safer, non-invasive anti-agitation treatment needed Current therapies sub-optimal: Dementia: A
Slide 7: BXCL501: Sublingual Thin Film Formulation of Dexmedetomidine
BXCL501: Sublingual Thin Film Formulation of Dexmedetomidine (Dex) Dex exerts calming effect at low exposures providing a broad therapeutic index Ideal Pharmaceutical Properties for a Non-invasive Sublingual Film Formulation Film manufacturing completed: • Multiple dose strengths ranging from 10µg t
Slide 8: Dexmedetomidine Mechanism of Action
Dexmedetomidine Mechanism of Action Reduction of hyper-arousal from overactive locus coeruleus neurons in response to stress Hyper-Arousal Physiology Dexmedetomidine MoA NE Locus Coeruleus (LC) Activation (+) Agitation (-) Agitation Norepinephrine (NE) Stress Induced Opiate Withdrawal Schizophrenia
Slide 9: Pre-Clinical Data to Support Clinical Development Plan
Pre-Clinical Data to Support Clinical Development Plan Properties of Dexmedetomidine in Cells, Brain Levels, and Efficacy Models De-Agitation without Sedation in Animal Models High Brain to Plasma Ratio Agitation reduction without sedation High Intrinsic Activity Translation of non-clinical data to
Slide 10: Positive Human Proof of Concept in Treating Agitation
Positive Human Proof of Concept in Treating Agitation IV Dex data from 105 patients: four disease pathologies (89) & healthy volunteers (16) SCHIZOPHRENIA ALZHEIMERS • • 14 patient study 14 patient study [10 treatment + 4 placebo] o [10 treatment + 4 placebo] o • • Clinical benefit observed in 9/10
Slide 11: Human Proof of Concept 1: IV Dex Reduces Agitation in Schizophrenia Patients
Human Proof of Concept 1: IV Dex Reduces Agitation in Schizophrenia Patients Study results announced Nov 2018: primary endpoint met Study Design 90% Response • • • • Randomized, placebo-controlled dose-ranging study 14 patients [10 treatment + 4 placebo] Primary endpoint: RASS of -1 Secondary endpoi
Slide 12: Human Proof of Concept 2: IV Dex Reduces Agitation in Alzheimer's Patients
Human Proof of Concept 2: IV Dex Reduces Agitation in Alzheimer’s Patients Study results announced Jan 2019: primary endpoint met Study Design 70% Response • • • • Randomized, placebo-controlled individual dose-ranging study Infusion initiated at a low rate and increased by 0.1 mcg/kg/h 14 patients
Slide 13: Human Proof of Concept 3: IV Dex Reduces Symptoms in Opioid Withdrawal
Human Proof of Concept 3: IV Dex Reduces Symptoms in Opioid Withdrawal Study results announced Feb 2019: primary endpoint met Study Design Randomized, placebo-controlled individual dose-ranging study Infusion initiated at a low rate and increased by 0.1 mcg/kg/h 100% Response • • • • 15 patients [10
Slide 14: Human Proof of Concept 4: IV Dex Reduces Agitation in Haloperidol-Refractory Delirium
Human Proof of Concept 4: IV Dex Reduces Agitation in Haloperidol-Refractory Delirium Elderly hyperactive delirium patients refractory to haloperidol are difficult to treat 100% Response 5 p = .01 4 3 2 1 Titration 0 -1 -2 Minutes -3 0 15 30 45 60 120 240 360 480 600 Start IV Haloperidol IV Haloperi
Slide 15: Sublingual Film (BXCL501) Pharmacokinetic and Safety Study
Sublingual Film (BXCL501) Pharmacokinetic and Safety Study IND-opening study BXCL501 Sublingual Pharmacokinetic, Safety and Tolerability Study • Placebo-controlled, single ascending dose, pharmacokinetic (PK) study Safety & tolerability of BXCL501 (sublingual film) in healthy adult volunteers ages 1
Slide 16: BXCL501 Integrated Clinical Development Plan
BXCL501 Integrated Clinical Development Plan Acute agitation studies: short with easily measurable clinical endpoints [IND] 2018 PK and Safety Study Multiple Dose Strengths (N=60) Sublingual Thin Film Data readout 2Q19 2019 Registration Trial* (Phase 2/3 Trials) Agitated Schizophrenia and Bipolar Pa
Slide 17: Healthcare Costs Associated with Agitation are a Significant Economic Burden
Healthcare Costs Associated with Agitation are a Significant Economic Burden Cost of acute agitation treatment across neuroscience disorders Indication Expansion Schizophrenia Bipolar Alzheimer’s Opiate/Alcohol Withdrawal 1 Hyperactive Delirium 5.0* 2 12 – 24 episodes per patient 3 PTSD/Hyperarousal
Slide 18: Clinical Programs BXCL701: First-in-Class Oral IO Therapy
Clinical Programs BXCL701: First-in-Class Oral IO Therapy Targeting Pancreatic Cancer and tNEPC 19 Proprietary & Confidential
Slide 19: BXCL701: Potential First-in-Class Oral IO Therapy Targeting Pancreatic Cancer and tNEPC
BXCL701: Potential First-in-Class Oral IO Therapy Targeting Pancreatic Cancer and tNEPC Rare tumors with large market opportunity and limited competition Orally Administered Activator Innate Immunity Pathway of Systemic Dual MoA Inhibits DPP 8/9 & FAP Orphan Drug Designation Established clinical pro
Slide 20: BXCL701 Mechanism of Action
BXCL701 Mechanism of Action With overlapping factors and effects Activation of cytotoxic T and NK cells Cytokine Release, IL18, IL-1, Pyroptosis, IL-18 release IL-8, MCP1, GCSF, IL-5, IL-6 Caspase-1 Nlrp1b inflammasome DPP8/9 BXCL701 Depletion of CAF, MDSCs, immature dendritic, Treg cells FAP Breaks
Slide 21: BXCL701: Existing Clinical Evidence Enables Rapid Development Path
BXCL701: Existing Clinical Evidence Enables Rapid Development Path Data from >700 patients demonstrate well characterized PK/PD, target inhibition, & anti-tumor activity Dose Target Inhibition BXCL701 Human Proof of Concept Single Agent Efficacy ~10% CR/PR Long Duration Comparable to Yervoy (anti-CT
Slide 22: Triple Combination Achieved Complete Regression and Immunity in Pancreatic Tumors
Triple Combination Achieved Complete Regression and Immunity in Pancreatic Tumors BXCL701 combination with NKTR-214 and Anti-PD-1 400.0 2108 1917 1725 1533 1342 1150 4 6 8 10 12 14 16 18 20 958 767 575 Complete Tumor 383 192 0 5 10 15 20 25 303540 45 50 55 60 65 70 75 80 85 90 95 100 105 110 Days af
Slide 23: Pancreatic Cancer Clinical Development Plan: Mechanistic and Anti-PD1 Combo Trial
Pancreatic Cancer Clinical Development Plan: Mechanistic and Anti-PD1 Combo Trial Biomarker driven development in advanced pancreatic cancer, potential breakthrough designation Demonstration of Immune Cell Infiltration/Activation to Validate MoA 2 Weeks of BXCL701 Treatment Before Surgery (Pre and P
Slide 24: tNEPC Clinical Development Plan: BXCL701 Combination with Keytruda
tNEPC Clinical Development Plan: BXCL701 Combination with Keytruda Biomarker driven development, breakthrough and fast track designation potential Global* Pivotal Study Safety Run-in Safety/PD/Immune-phenotyping (N=6) Phase 2 Expansion (N=30) *Expect to commence global development planning during Ph
Slide 25: Value Creation Catalysts
Value Creation Catalysts 26 Proprietary & Confidential
Slide 26: Milestones Accomplished Since IPO
Milestones Accomplished Since IPO Highlights as of 1Q19 Advisory Board Members 4 13 Regulatory 8 members for BXCL501 5 members for BXCL701 3 INDs approved: BXCL701 tNEPC BXCL501 PK (bioavailability) BXCL701 Pancreatic (MoA) 20 1 IND and 1 CTA planned in 1H19 Team 4 IV Dex Trials completed: 6 Schizop
Slide 27: Key Milestones for Value Creation
Key Milestones for Value Creation Two mid-stage clinical trial candidates 2H’18 1H’19 2H’19 1H’20 2H’20 Beyond BXCL501 (Schizophrenia / Bipolar Disorder / Dementia) Pharmacokinetic Study Initiated (healthy volunteers) Data Readout (PK Characterization/ Dose Range) Registration Trial Initiation (Phas
Slide 28: Optimally Positioned for Execution
Optimally Positioned for Execution Support from world-class investors Funded to Reach Multiple Inflection Points Total Cash and Cash Equivalents: 42.6 million as of December 31st, 2018 Major Shareholders: * * Artemis (7.4%) Fidelity (5.5%) DNCA Finance (5.11%) Do Kim (BMO Capital Markets) Sumant Kul
Slide 29: Appendix: Management Team, Board Profile
Appendix Management Team Board Profile 30 Proprietary & Confidential
Slide 30: World-Class Leadership Team Supported By Strong Board of Directors and Advisory Board
World-Class Leadership Team Supported By Strong Board of Directors and Advisory Board Combined experience of 150+ years in drug development with 15 approved drugs MANAGEMENT TEAM VIMAL MEHTA CEO & Member of Board FRANK YOCCA Chief Scientific Officer VINCENT J. O’NEILL Chief Medical Officer RICHARD I
Slide 31: World-Class Leadership Team: Board of Directors and Strategic Advisors
World-Class Leadership Team Supported By Strong Board of Directors and Advisory Board Combined experience of 150+ years in drug development with 15 approved drugs BOARD OF DIRECTORS STRATEGIC ADVISORS PETER MUELLER Chairman of Board STEVE LAUMAS Member of Board KRISHNAN NANDABALAN Member of Board ST
Slide 32: Neuroscience Clinical Advisory Board to Support Global Development of BXCL501
Neuroscience Clinical Advisory Board to Support Global Development of BXCL501 Prominent clinicians and neuroscientists to guide advancement of lead programs and emerging neuroscience pipeline Clinical Advisory Board Sheldon H. Preskorn, M.D. Professor of Psychiatry George Grossberg, M.D. Director, G
Slide 33: Immuno-Oncology Clinical Advisory Board to Advance BXCL701 Development
Immuno-Oncology Clinical Advisory Board to Advance BXCL701 Development Appointment of world renowned immuno-oncology clinicians and scientists Clinical Advisory Board Louis M. Weiner, M.D. Emmanuel S. Antonarakis, M.D. Daniel Von Hoff, M.D., F.A.C.P. Eric J. Small, M.D. Johann de Bono, M.D., Ph.D. A
Slide 34: Dr. Vimal Mehta, CEO, BioXcel Therapeutics
Dr. Vimal Mehta, CEO BioXcel Therapeutics, New Haven, CT 06511 vmehta@bioxceltherapeutics.com 35 Proprietary & Confidential
Slide 35: BioXcel Therapeutics contact
ct | bioxcel . xX therapeutics Dr. Vimal Mehta, CEO BioXcel Therapeutics, New Haven, CT 06511 vmehta@bioxceltherapeutics.com Proprietary & Conidenta
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