Bioxcel Therapeutics, Inc. · NASDAQ:BTAI
Bioxcel Therapeutics — Investor Presentation (May 2018)
27 slides from Bioxcel Therapeutics's investor presentation, May 21, 2018
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 First-in-Class IO Platform with Broad Applicability Targeting Rare Cancers Approach Improves Development Efficiency and Probability of Success BXCL501 Data Readouts; Initiation of BXCL701 Clinical Studies; Selection of New Candidate(s) Proven Track Record o
Slide 3: World-Class Leadership Team Supported By Strong Board 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 Peter Mueller Chairman of Board Vimal Mehta CEO & Member of Board Frank Yocca Chief Scientific Officer Krishnan Nandabala
Slide 4: BioXcel Therapeutics' Approach: Drug Re-Innovation
BioXcel Therapeutics’ Approach: Drug Re-Innovation Identification of new therapeutic index utilizing AI-powered R&D engine Developing the Next Wave of Medicines to Address Major Unmet Medical Needs BXCL501 BXCL702 BXCL701 Clinical PK/PD Target Engagement CMC Clinical Proof of Mechanism Regulatory Pa
Slide 5: BioXcel Therapeutics 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 Phase 1/2 Phase 3 Anticipated Milestones Program Emerging Programs Product Candidate BXCL502 Hematological Malignancies Selection of clinica
Slide 6: BioXcel Therapeutics 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 x x x : —— Baise Bisiie [ Program a 2088 sake Milestones Rights Geriatric Dementia + nat Phase ab as ai tan 208) pene | BXCL501 | IV Dexmede
Slide 7: Rapid Clinical Development and Regulatory Approval Path (505(b)(2))
Rapid Clinical Development and Regulatory Approval Path (505(b)(2)) Agitation resulting from Alzheimer’s and Schizophrenia / Bipolar disease PoC in Agitated Delirium NDA 2020 Established Clinical, Regulatory, Reimbursement Path BXCL501: Sublingual Thin Film Dexmedetomidine (Dex) for Acute Treatment
Slide 8: BXCL501: A Sublingual Thin Film Dexmedetomidine
Sold as sedative/anesthetic (approved in US as Precedex® 1999) Most selective alpha2a adrenergic agonist Well characterized safety and pharmacokinetic profile Produces an “arousable sedation” useful for treating agitation Anti-agitation effect demonstrated with IV dose of 0.5µg/kg BXCL501: A Subling
Slide 9: BXCL501 MoA Proven to Treat Agitated Delirium in Elderly
BXCL501 MoA Proven to Treat Agitated Delirium in Elderly Acute Agitation Clinical Study Shows Easily Measured Endpoints Hyperactive delirium patients refractory to haloperidol are difficult to treat All haloperidol-refractory agitated patients (n=46) were calmed by Dex treatment Carrasco et.al., Cri
Slide 10: BXCL501 Integrated Clinical Development Plan
BXCL501 Integrated Clinical Development Plan Acute agitation studies: short with easily measurable clinical endpoints IV Dex PK/PD Safety IV Dex Efficacy (Open Label) Agitated AD Patients (2 Studies) Agitated Schizophrenics and Bipolar Patients (1 Study/Indication) Sublingual Thin Film (BXCL501) Reg
Slide 11: 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 estimated >$40 billion Patients (mm) U.S. Addressable Market for Agitation Treatment Delirium Alcohol Withdrawal DTs PTSD/Hyperarousal Pre-MRI Anxiety Indicati
Slide 12: 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 estimated >$40 billion U.S. Addressable Market for Agitation Treatment ication Expansion 5.5" Se Delirium € = 2s | Alcohol withdrawal 2 id; 2 DTs = /adasuve s
Slide 13: BXCL701: Potential First-in-Class Oral IO Therapy Targeting tNEPC and Pancreatic Cancer
BXCL701: Potential First-in-Class Oral IO Therapy Targeting tNEPC and Pancreatic Cancer Disruptive immuno-oncology platform with potential to create transformative franchise Rare Tumors with Large Market Opportunity and Limited Competition Human PoC in Melanoma with BXCL701 established BXCL701 MoA,
Slide 14: BXCL701: Existing Clinical Evidence Enables Rapid Development Path
BXCL701: Existing Clinical Evidence Enables Rapid Development Path Data from >700 patients demonstrate well characterized human PK/Target Inhibition/PD, melanoma, and anti-tumor activity ----Daily BXCL701 Dose---- Stimulation Of Immune Cells Target Inhibition Induction of Pro-inflammatory Cytokine B
Slide 15: Differentiated MoA Induces Immune Activation and Blocks Immuno-evasion
ANTI-TUMOR ACTIVITY Differentiated MoA Induces Immune Activation and Blocks Immuno-evasion MoA inhibits DPP 8/9 & FAP and converts tumors from “cold” to “hot” Blocking of Immune Evasion Immune Activation Tumor Microenvironment BXCL701 Myeloid Derived Suppressor Cell FAP+ Fibroblast CXCL12-CXCR4 IDO
Slide 16: Triple Combination Achieved Complete Regression and Immunity in Pancreatic Tumors
Triple Combination Achieved Complete Regression and Immunity in Pancreatic Tumors Combo with anti-PD1 and NKTR-214 fully stimulates immune system, ”curing” mice, making them resistant to new tumors Mean Tumor Volume (mm3) Days After Tumor Re-challenge BXCL701 Dosing Vehicle BXCL701 Triple Combinatio
Slide 17: BXCL701+NKTR-214 +/- anti-PD-1 Show FAP Reduction, with CD8+ Infiltrates
BXCL701+NKTR-214 > +/- anti-PD-1 Show FAP Reduction, with CD8+ Infiltrates FAP reduction blocks immuno-evasion, neutrophil infiltration shows immuno-activation supported by existing clinical data BXCL701+NKTR-214 +/- Anti-PD1 Shows decrease in FAP 18 BXCL701+NKTR-214 +/- Anti-PD1 Shows increase in C
Slide 18: tNEPC and Pancreatic Cancer have Highest Level of DPP8/9 and FAP Expression
BXCL701: tNEPC and Pancreatic Cancer have Highest Level of DPP8/9 and FAP Expression Clinical activity in melanoma supported by genetic and functional data Deletion Amplification Initial Development Focus on tNEPC and Pancreatic Cancer 19
Slide 19: tNEPC Clinical Development Plan: Single Agent and Combination with Anti-PD1
tNEPC Clinical Development Plan: Single Agent and Combination with Anti-PD1 Biomarker driven development, breakthrough and fast track designation potential *Expect to commence global development planning (focus on EU and Japan) during Phase 2 Global* Pivotal Study Phase 2 Stage 1 (N=15) Phase 2 Stag
Slide 20: 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 Global* Pivotal Study YES Activity Stop NO 3 Weeks of BXCL701 Treatment Before Surgery Pre and Post Tissue Available (N=15)
Slide 21: Large Market Opportunity, Limited Competition
Large Market Opportunity Limited competition 30% progress to tNEPC: 30k tNEPC Zytiga and Xtandi Sales ~$4.5 billion Pancreatic Cancer Patients Eligible for BXCL701: 20k Abraxane Sales ~$1 billion Patients Eligible for Treatment with ADT ~180k US Prostate Cancer Patient Population ~3mm 50% Patients E
Slide 22: Offers Pipeline-in-a-Product Platform
Offers Pipeline-in-a-Product Platform Broad potential across multiple 10 modalities ===”: ST ot MoA complementary to 10 | , checkpoints by stimulation of innate and acquired immune system Increases efficacy in patients with “cold” tumors Cellular Therapies BXCL701 Stimulation of chemotaxix, ©} cytox
Slide 23: Key Milestones for Value Creation
Key Milestones for Value Creation Two mid-stage clinical trial candidates Drug Indication 1H’18 2H’18 1H’19 2H’19 2020 and Beyond BXCL501 Geriatric Dementia IV Dex Study Ongoing IV Dex Data Readout PK/PD PoC Trial Bio-Equivalence Film Study Initiation Registration Trial NDA Schizophrenia / Bipolar D
Slide 24: Optimally Positioned for Execution
Optimally Positioned for Execution MARCH 2018 Completed Initial Public Offering, generating gross proceeds of $60 million Major shareholders include Fidelity (10.7%) and Artemis (7.7%) Total cash and cash equivalents of $55.5 million as of March 31, 2018 Funded to Reach Multiple Inflection Points Su
Slide 25: BioXcel Therapeutics Investment Highlights
BioXcel Therapeutics Investment Highlights First-in-Class IO Platform with Broad Applicability Targeting Rare Cancers Approach Improves Development Efficiency and Probability of Success BXCL501 Data Readouts; Initiation of BXCL701 Clinical Studies; Selection of New Candidate(s) Proven Track Record o
Slide 26: Dr. Vimal Mehta, CEO
Dr. Vimal Mehta, CEO BioXcel Therapeutics, Branford, CT 06405 USA vmehta@bioxceltherapeutics.com
Slide 27: BioXcel Therapeutics contact
ct | bioxcel . Xr therapeutics Dr. Vimal Mehta, CEO BioXcel Therapeutics, Branford, CT 06405 USA vmehta@bioxceltherapeutics.com
BioXcel Therapeutics — Investor Presentation (May 2018) on 6ix