Slide 1: Agenda
Agenda 01 02 BXCL501 PROGRAM OVERVIEW CLINICAL TRIALS RESULTS WITH IV DEXMEDETOMIDINE ALZHEIMER’S | SCHIZOPHRENIA 03 04 BXCL501 DEVELOPMENT PLAN IN 2019 SUMMARY ATTENDEES Vimal Mehta, CEO & Member of Board Vincent O'Neill, Chief Medical Officer Frank Yocca, Chief Scientific Officer Robert Risinger,
Slide 2: 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 3: Dementia: Symptoms of Alzheimer's Disease
Dementia: Symptoms of Alzheimer’s Disease Nearly half of the Alzheimer’s patients have Agitation symptoms every month1 NEUROPSYCHIATRIC5 • • • • Agitation Aggression Mood disturbances Wandering COGNITIVE2-4 FUNCTIONAL6 • • Memory loss Impaired spatial and temporal orientation Language disturbance Ag
Slide 4: 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 5: 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 Locus Coeruleus (LC) Activation (+) Agitation (-) Agitation Norepinephrine Stress Induced Bipolar Disorder Opiate Withdrawal Schiz
Slide 6: Dexmedetomidine: High Potency and Intrinsic Activity at Alpha2 Receptors
Dexmedetomidine: High Potency and Intrinsic Activity at Alpha2 Receptors Data generated using transfected human Alpha2 receptor Human Alpha2 Receptor Response [Compound] Molar Dexmedetomidine: Pharmacology may translate into greater clinical efficacy 7 Proprietary & Confidential Functional Receptor
Slide 7: Human Biology Studies for BXCL501
Human Biology Studies for BXCL501 Differentiate central nervous system (CNS) from peripheral effects EEG Measurements • Dex elicits a characteristic change in electroencephalographs (EEG) • Plan to use EEG to demonstrate CNS activity that determines therapeutic window • • Lowest dose with CNS activi
Slide 8: IV Dex: Positive Human Proof of Concept in Treating Agitation
IV Dex: Positive Human Proof of Concept in Treating Agitation from Alzheimer's Disease, Schizophrenia and Delirium IV Dex data from 90 subjects: healthy volunteers and three disease pathologies 1 2 ALZHEIMER’S DISEASE 14 patient study [10 treatment + 4 placebo} Clinical benefit observed in 7/10 trea
Slide 9: 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 • • • • Randomized, placebo-controlled dose-ranging study 14 patients [10 treatment + 4 placebo] Primary endpoint: RASS of -1 Secondary endpoint: PEC score
Slide 10: 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 • • • • Randomized, placebo-controlled individual dose-ranging study Infusion initiated at a low rate and increased by 0.1 mcg/kg/h 14 patients [10 treatment
Slide 11: Human Proof of Concept 3: IV Dex Reduces Agitation in Haloperidol-Refractory Delirium
Human Proof of Concept 3: IV Dex Reduces Agitation in Haloperidol-Refractory Delirium Elderly hyperactive delirium patients refractory to haloperidol are difficult to treat 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 Haloperidol + IV Dex I
Slide 12: Sublingual Film (BXCL501) Pharmacokinetic and Safety Study
Sublingual Film (BXCL501) Pharmacokinetic and Safety Study IND-opening study BXCL501-101 Sublingual Pharmacokinetic, Safety and Tolerability Study • Placebo-controlled, single ascending dose, pharmacokinetic (PK) study, safety & tolerability of BXCL501 (sublingual film) in healthy adult volunteers a
Slide 13: 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 1H2019 2019 Registration Trial* (Phase 2/3 Trials) Agitated Schizophrenia and Bipolar
Slide 14: BXCL501 Program Summary
BXCL501 Program Summary Broad market potential • Low doses produce clinically measurable anti-agitation effects • On track to initiate a registration trial for an indication for BXCL501 in 2019 with potential NDA filing in 2020 • Potential for multiple indications due to BXCL501’s anti-agitation pro
Slide 15: Dr. Vimal Mehta, CEO
Dr. Vimal Mehta, CEO BioXcel Therapeutics, New Haven, CT 06511 vmehta@bioxceltherapeutics.com Proprietary & Confidential
Slide 16: BioXcel Therapeutics contact
ct bioxcel - Of therapeutics Dr. Vimal Mehta, CEO BioXcel Therapeutics, New Haven, CT 06511 vmehta@bioxceltherapeutics.com