Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Presentation (February 2015)
54 slides from Assembly Biosciences's investor presentation, Feb 24, 2015
Slide 1: Assembly Biosciences February 2015
Assembly Biosciences February 2015 1
Slide 2: Forward-Looking Statements
Forward - Looking Statements This presentation contains forward - looking statements regarding future events. Forward - looking statements involve known and unknown risks that could cause actual results to differ materially from expected results. These risks and uncertainties include, among others:
Slide 3: Assembly Biosciences – Overview
Assembly Biosciences – Overview Building a world class infectious disease company Two Proprietary Technology Platforms, Best - In - Class Science and Novel Drugs for HBV and CDAD All current programs discovered and developed in - house • Multiple differentiated mechanisms and molecules • Core Protei
Slide 4: Assembly Biosciences - Organization
Assembly Biosciences - Organization Thought leadership in infectious disease and specifically HBV experience Derek A. Small – Pres , CEO - Founding CEO of multiple biotechs David Barrett – COO & CFO - CFO, director at multiple biotechs Uri Lopatin , MD – CMO, VP R&D - Led HBV programs at leading bio
Slide 5: Our Approach: Leverage HBc to Target cccDNA
Our Approach: Leverage HBc to Target cccDNA Our Core Competencies and Competitive Advantage Our Approach to an HBV Cure Prevent NEW cccDNA Inhibit Existing cccDNA Eliminate / Silence cccDNA HBV CURE Intellectual Property HBV Biology Proprietary Enabling Assays World Class Chemistry Clinical Expertis
Slide 6: Pipeline Progress – Building Momentum
Pipeline Progress – Building Momentum HBV Lifecycle Modulation Current and Planned Development Program “Downstream” Inhibit HBV Replication “Upstream” Modulating cccDNA Activity Research Hits Lead Optimization IND Enabling Phase I Phase II ASMB - 101 CpAM Capsid Targeted ASMB - 102 CpAM Upstream Mec
Slide 7: HBV Background & Assembly Approach
HBV Background & Assembly Approach 7
Slide 8: HBV Global Unmet Medical Need
HBV Global Unmet Medical Need • >350 million people worldwide have chronic hepatitis B infection • 10 - 30M new patients each year & >600K deaths/year WW sales of $3.2B in 2012 with only ~5% of chronic patients treated Source: WHO, Ferlay et al. Globocan (2002), Ministry of Health of the People's Re
Slide 9: Assembly's Approach to HBV Cure
Assembly’s Approach to HBV Cure 1. HBV is a DNA virus – unlike HCV, HBV viral reservoir ( cccDNA ) is in the nucleus 2. Curative therapy for HBV will require modulation and destabilization or silencing of cccDNA • Limited cure is seen on current therapies (3 - 10% of cases) (1) • Preliminary researc
Slide 10: HBV Lifecycle
HBV Lifecycle HBV is a DNA virus unlike HCV; Core Protein is involved in all aspects of the viral lifecycle 10
Slide 11: Capsid Programs Target Downstream
Capsid Programs Target Downstream Downstream therapies are unlikely to be curative as monotherapy “Capsid” Programs 11
Slide 12: Assembly's Core Protein Programs
Assembly’s Core Protein Programs Assembly’s CpAMs target upstream and downstream in the viral lifecycle “Capsid” Programs 12
Slide 13: Program and Pipeline Overview
Program and Pipeline Overview Core Protein Rationale 13
Slide 14: Core Protein Localizes to the Nucleus of Infected Cells
Core Protein Localizes to the Nucleus of Infected Cells Preclinical data (Cell culture) TET - TET+(24h) HBc DAPI HepaRG - TR - HBc (If anti - HBc ) Nuclear Extract Cytoplasmic Extract 0 8H 24H 48H 0 8H 24H 48H HBc PARP Tubulin HepaRG - TR - HBc (WB) Source: Durantel et al. AASLD. Presented at EASL.
Slide 15: Core Protein Localizes to the Nucleus of Patient Cells
Core Protein Localizes to the Nucleus of Patient Cells Representative clinical data : Histopathology of HBeAg +, High viral load patient Courtesy Patrick Kennedy MD 15
Slide 16: Core Protein has MULTIPLE Roles Modulating cccDNA
Core Protein has MULTIPLE Roles Modulating cccDNA HBc Regulates cccDNA Expression HBc Regulates cccDNA Persistence 16
Slide 17: HBc Modulates Immune Response
HBc Expressing Cell Line HBc Modulates Immune Response HBc expression interferes with hepatocyte innate response 0 50 100 PolyIC PolyIC + TET IFNB IL29 OAS1 IL6 T7 Expressing Cell Line TLR3Pathway RIGI/MDA5 Pathway 0 50 100 PolyIC PolyIC + TET IFNB IL29 OAS1 IL6 0 50 100 PolyIC PolyIC + TET IFNB IL2
Slide 18: Core Protein Provides ASMB Multiple Targets
Core Protein Provides ASMB Multiple Targets HBV core protein has multiple functions and conformations Free Cp Cp : C apsid Zlotnick, Tan, Selzer. (2013) Structure 21 , 6 - 8. Other Conformations* Assembly has a best in class team to understand core protein. Targeting Core PLEOTROPIC core functions b
Slide 19: HBV-Cure Pipeline – Clinical Strategy
HBV - Cure Pipeline – Clinical Strategy CpAMs classes show efficacy as monotherapy. Multiple classes allows exploration of CpAMs in combination across CpAM classes AND with other classes of HBV therapy. Planned clinical program: • Phase I (safety) studies as single agents • Phase IB studies in patie
Slide 20: HBV Program Milestones
HBV Program Milestones • Present “Upstream” mechanistic data in preclinical models on multiple CpAM classes • Select first lead candidate • Select additional clinical lead molecules • Completion of multiple IND directed safety programs • Initiation of clinical trials for multiple CpAMs • Initiation
Slide 21: Intellectual Property Portfolio: HBV & CpAMs
Intellectual Property Portfolio: HBV & CpAMs The patent applications fall broadly into two categories: 1. Platform patent applications – Core Protein • Multiple platform patent applications filed, with others in process • Patent applications cover novel mechanism of action, methods of treatment, nov
Slide 22: Microbiome Program: C. Difficile
Microbiome Program: C. D ifficile 22
Slide 23: Microbiome Therapeutics: Overview
Microbiome Therapeutics: Overview • We have in - licensed technology for targeted delivery of complex agents to select regions of the GI tract • Our delivery technology utilizes state of the art encapsulation technology and new coating technologies in conjunction to exploit specific pH gradients acr
Slide 24: Microbiome Therapeutics: Overview (cont'd)
2013 2014 2015 2016 MB - 101 — Targeted, Oral Delivery of Microbiome Therapeutics Multiple novel delivery patents filed 1) ID lead candidates from FMT patients 2) CMC development 3) ID strain leads IND submission Phase IB Clinical data 3Q of 2016 Microbiome Therapeutics: Overview (cont’d) Microbiome
Slide 25: Financials & Key Investment Highlights
Financials & Key Investment Highlights 25
Slide 26: Financial Summary
Financial Summary Ticker Nasdaq: ASMB Cash & Cash Equivalents ~$29M as of Dec 2014 Shares Outstanding ~10.6M (insiders own 3M, institutional investors own 4.6M) Fully Diluted Shares Outstanding ~13.8M Highlights • ASMB merger July 2014 • Capital raise of $15.8M in October 2014 (max under baby shelf
Slide 27: Investment Highlights
Experienced Management Team and Scientific Advisory Board with Leadership in HBV Core Protein Research Proven, In - House Discovery and Development Capabilities with Multiple Shots on Goal Novel Microbiome Program for C.Difficile Potential First - and Best - in - Class HBV Platform With Potential to
Slide 28: Assembly Biosciences
Assembly Biosciences February 2015
Slide 29: Forward-Looking Statements
Forward-Looking Statements (m This presentation contains forward-looking statements regarding future events. Forward-looking statements involve known and unknown risks that could cause actual results to differ materially from expected results. These risks and uncertainties include, among others: ris
Slide 30: Assembly Biosciences — Overview
Assembly Biosciences — Overview (m Building a world class infectious disease company lwo Proprietary Technology Platforms, Bes’ Class Science and Novel Drugs for HBV and CDAD HBV Platform Microbiome Platform C. Difficile (CDAD) + Multiple differentiated mechanisms and lecul mICeceres. * Targeted, Or
Slide 31: Assembly Biosciences - Organization
—~ Assembly Biosciences - Organization @) Thought leadership in infectious disease and specifically HBV experience Derek A. Small - Pres, CEO - Founding CEO of multiple biotechs David Barrett - COO & CFO - CFO, director at multiple biotechs Poraure. Cafewm_— eran ig Deloitte. Uri Lopatin, MD — CMO,
Slide 32: Our Approach to an HBV Cure
Our Approach to an HBV Cure Our Approach: Leverage HBc to Target cccDNA Our Core Competencies and Competitive Advantage HBV Biology Prevent NEW cecDNA Eliminate| Silence cecDNA Inhibit Existing eceDNA Proprietary Enabling Assays World Class Chemistry Intellectual Property Clinical Expertise
Slide 33: Pipeline Progress — Building Momentum
Pipeline Progress — Building Momentum (m HBV Lifecycle Modulation ‘Current and Planned Development Lead cceDNA hetvity Research Hits Optimization INO Enabling Asme-101 cpaut Capsid Targeted eee e ‘ASMB-102 CpAM Upstream Mechanism ‘ASMe-103 CpaM Upstream Mechanism ‘ASMB CpAM (Gen 2) ToBe Selected ‘Se
Slide 34: HBV Background & Assembly Approach
Va SD Via HBV Background & Assembly Y +) Approach assembly, biosciences
Slide 35: HBV Global Unmet Medical Need
—~ HBV Global Unmet Medical Need @) + >350 million people worldwide have chronic hepatitis B infection * 10-30M new patients each year & >600K deaths/year ee SP 14 mition = 112 milion Diraillion.aoue- chronically infected —_chroni = in Europe in cted chronically infected In North America WW sales of
Slide 36: Assembly's Approach to HBV Cure
—~ Assembly’s Approach to HBV Cure @) 41. HBVis a DNA virus — unlike HCV, HBV viral reservoir (¢ccDNA) is in the nucleus 2. Curative therapy for HBV will require modulation and destabilization or silencing of eccDNA + Limited cure is seen on currenttherapies (3-10% of cases)” + Preliminary research
Slide 37: HBV Lifecycle
HBV Lifecycle HBV is a DNA virus unlike HCV; Core Protein is involved in all aspects of the viral lifecycle wuceus 10
Slide 38: Capsid Programs Target Downstream
Capsid Programs Target Downstream Downstream therapies are unlikely to be curative as monotherapy “Capsid” Programs "
Slide 39: Assembly's Core Protein Programs
Assembly’s Core Protein Programs Assembly’s CpAMs target upstream and downstream in the viral lifecycle 2
Slide 40: Program and Pipeline Overview
_ Program and Pipeline Overview Core Protein Rationale assem ley biosciens ay 2
Slide 41: Core Protein Localizes to the Nucleus of Infected Cells
Core Protein Localizes to the Nucleus of lah _ Infected Cells Sub-cellular Localization of HBc Protein in HBc Expressing Cell Line (HepaRG-TR-HBc) TeT- TET+(24n) ct ito cr papi HepaRG-TR-HBc (if anti-HBc) HepaRG-TR-HBc (WB) HBV core protein is mostly localized in the nucleus ‘Source: Durantelet al,
Slide 42: Core Protein Localizes to the Nucleus of Patient Cells
Core Protein Localizes to the Nucleus of —~ Patient Cells @) Representative clinical data: Histopathology of HBeAg +, High viral load patient Courtesy Patrick Kennedy MD. SAB. ss Cytoplasmic HBV core
Slide 43: Core Protein has MULTIPLE Roles
Core Protein has MULTIPLE Roles Modulating cccDNA @) HBc Regulates cccDNAExpression HBc binds to the CpG islands of HBV cccDNA and promotes an epigenetic permissive state HBc Regulates cccDNA Persistence ‘Science, 2014 Mar 14;349(6176):12218. dot 10.11284cionce. 1243462. Epub 2014 Fb 20. Specific an
Slide 44: HBc Modulates Immune Response
HBc Modulates Immune Response (m HBc expression interferes with hepatocyte innate response HBc — Cell Line T7 Expressing Cell Line 1 > one one 2 as wus =i 2 5 sorss worst 5 = aus ss ° ° Poy + TET Foyle Foye + TET =i 1% § z one sme ‘- a - “1 5 8 sonst s0Ast aus ary Hees eee ° @ Peye Poy + TET Pole Po
Slide 45: Core Protein Provides ASMB Multiple Targets
—~ Core Protein Provides ASMB Multiple Targets @) HBV core protein has multiple functions and conformations Free Cp propeptide ai, eZ 10 es Other a Conformations* Zlotnick, Tan, Selzer, (2012) Structure 21.68 Assembly has a best in class team to understand core protein. Targeting Core PLEOTROPIC cor
Slide 46: HBV-Cure Pipeline — Clinical Strategy
HBV-Cure Pipeline — Clinical Strategy (m HBV Lifecycle Modulation ‘Current and Planned Development “Downstream “Upstream” “InhibitHEY Modulating Lead Replication _eceDNA hetivity Research Hits Optimization IND Enabling Phase! Capsid Targeted ooo e Upstieam Mechanism ee ee aS Upstream Mechanism e eee
Slide 47: HBV Program Milestones
HBV Program Milestones A Anticipated 2015 Milestones + Present “Upstream” mechanistic data in preclinical models on multiple CpAM classes + Select first lead candidate Anticipated 2017 Milestones Anticipated 2016 Milestones —™ + Initiation of Phase 2 clinical + Select additional clinical lead Progra
Slide 48: Intellectual Property Portfolio: HBV & CpAMs
Intellectual Property Portfolio: HBV & CpAMs (m The patent applications fall broadly into two categories: 1. Platform patent applications — Core Protein * Multiple platform patent applications filed, with others in process * Patent applications cover novel mechanism of action, methods of treatment,
Slide 49: Microbiome Program: C. Difficile
Via Y Microbiome Program: C. Difficile iosciences
Slide 50: Microbiome Therapeutics: Overview
Microbiome Therapeutics: Overview @) + We have in-licensed technology for targeted delivery of complex agents to select regions of the Gl tract + Our delivery technology utilizes state of the art encapsulation technology and new coating technologies in conjunction to exploit specific pH gradients ac
Slide 51: Microbiome Therapeutics: Overview (cont'd)
Microbiome Therapeutics: Overview (cont'd) (m Microbiome CDAD Program — Planned Development 2014 2015 Mutipie novel delvery patents filed 1) lead candidates from FUT patients 2) CC development 2) 1D strain leads IND submission Phase Clinical data 30 of 2016 24
Slide 52: Financials & Key Investment Highlights
Va SD Via Financials & Key Investment Y +) Highlights assembly, biosciences 28
Slide 53: Financial Summary
—~ Financial Summary () 2 Ticker Nasdaq: ASMB Cash & Cash Equivalents ~$29M as of Dec 2014 ~10.6M (insiders own 3M, institutional investors own 4.6M) Shares Outstanding Fully Diluted Shares Outstanding ~13.8M + ASMB merger July 2014 + Capital raise of $15.8M in October 2014 (max Highlights under bab
Slide 54: Investment Highlights
—~ Investment Highlights () , a. KY assembly terete and Scientific Advisory Board with eadership in HBV Proven, In-House If Cors protein Discovery and Racer Development Capabilities with Multiple Shots on Goal Potential First-and Best-in-Class HBV Platform With Potential to Cure HBV World Class Infe
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