Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Presentation (September 2017)
29 slides from Assembly Biosciences's investor presentation, Sep 7, 2017
Slide 1: Assembly Biosciences, Inc. Corporate Presentation
Assembly Biosciences, Inc. Corporate Presentation September 7, 2017 Nasdaq: ASMB
Slide 2: Cautionary note regarding forward-looking statements
Cautionary note r egarding forward - looking s tatements The information in this presentation contains estimates and other forward - looking statements regarding future events, including statements about the clinical and therapeutic potential of our HBV - Cure and Microbiome programs, our ability to
Slide 3: Assembly Biosciences: Overview
3 Delivering oral, synthetic live biotherapeutics to targeted location Allergan partnership for GI assets, rest of pipeline wholly owned HBV Deep pipeline of CpAMs with potential to achieve CURE Large market opportunity with high unmet need Microbiome Team Industry leaders w/ expertise in drug disco
Slide 4: Development Pipeline
Program Drug Candidate (Mech. / Indication) Discovery Lead Op / Selection IND Enabling Phase 1 Rights Hepatitis B ABI - H0731 ( CpAM ) CpAM 2 nd Generation CpAM 3 rd Generation Novel Target Microbiome ABI - M201 (Ulcerative Colitis ) ABI - M301 (Crohn’s Disease) IBS compounds NASH, I/O & Other Clost
Slide 5: Our Timeline Highlights Significant Progress to Date
Our Timeline Highlights Significant Progress to Date 5 x $2.8B Microbiome collaboration in GI with Allergan x ABI - H0731 Phase 1 a completed x Initiated ABI - H0731 Ph 1b o Advance 2 nd gen HBV compounds o Potential ABI - H0731 Ph 1b interim data x Expanded senior leadership team x Ph 1a/1b HBV tri
Slide 6: Hepatitis B— Cure Program
Hepatitis B— Cure Program @ assembly blosclences
Slide 7: Significant need for curative HBV therapies
Significant need for curative HBV therapies Market Opportunity ~ 240 million patients worldwide, ~90 million in China, ~2 million in US and >600,000 deaths/year globally CURE Eliminate (or Silence) Existing cccDNA Inhibit cccDNA Formation Prevent Virus Replication Limited to nucleos (t)ide analogs (
Slide 8: HBV Life Cycle: Complicated with Limited Targets
HBV Life Cycle: Complicated with Limited Targets cccDNA Maintenance 8 • HBV genome only encodes FOUR known genes: Core, X, Polymerase and S ( HBsAG )
Slide 9: HBV Life Cycle: Failure of Nucs to Inhibit cccDNA
cccDNA Maintenance HBV Life Cycle: Failure of Nucs to Inhibit cccDNA ? CpAMs CpAMs CpAMs CpAMs 9 cccDNA Maintenance Nucleos (t)ides
Slide 10: HBV Life Cycle: New Antiviral Targets Being Pursued
HBV Life Cycle: New Antiviral Targets Being Pursued cccDNA Maintenance Nucs & Non - Nucleoside Entry Inhibitor RNA Interference 10
Slide 11: HBV Core Protein Is Required Throughout Lifecycle
cccDNA Maintenance HBV Core Protein Is Required Throughout Lifecycle CpAMs ( C ore P rotein A llosteric M odulators) target Core protein and functions related to cccDNA generation ? CpAMs CpAMs CpAMs CpAMs 11
Slide 12: CpAMs and ETV inhibit HBV Replication by Distinct Mechanisms
CpAMs and ETV inhibit HBV Replication by Distinct Mechanisms 12 cccDNA CpAM AAA... AAA... AAA... ETV pgRNA 0 20 40 60 80 100 DMSO 100 nM ETV 1 uM ABI-H0731 1 uM GLS4 Percent of DMSO Control Total Intracellular Extracellular Encapsidated HBV pgRNA Levels 0 50 100 150 200 DMSO 100 nM ETV 1 uM ABI-H073
Slide 13: CpAMs inhibit cccDNA generation in primary human hepatocytes
CpAMs inhibit cccDNA generation in primary human hepatocytes Viral DNA, pgRNA, HBeAg and HBsAg in Primary Human Hepatocytes EC 50 ( nM ) Compound Viral DNA HBeAg HBsAg pgRNA ABI - H0731 154 2,210 3,000 1,840 Prototype ABI - H0808 80 196 310 305 ETV <0.1 Incomplete Incomplete Incomplete Viral DNA HBe
Slide 14: CpAMs block cccDNA formation in HBV infected cells
CpAMs block cccDNA formation in HBV infected cells Primary Human Hepatocytes (PHH) U p to ~ 125x EC 50 Entecavir DMSO 20 nM 100 nM DMSO 3.3 µM 10 µM ABI - H0731 >1,000x EC 50 Entecavir DMSO 100 nM ABI - H0731 DMSO 1.25 µM 2.5 µM 5.0 µM 10.0 µM cccDNA • Only CpAMs reduced cccDNA formation in HepG2 -
Slide 15: ABI-H0731: Candidate Summary
ABI - H0731: Candidate Summary • Differentiated mechanism(s) that target core protein and impact cccDNA – Exhibits activity not seen with current SOC therapies • Proprietary chemistry designed for optimal treatment regimen – Highly potent and selective – Good bioavailability and metabolically stable
Slide 16: HBV Program: ABI-H0731 Anticipated Updates and Milestones
HBV Program: ABI - H0731 Anticipated Updates and Milestones 16 H 2 2017 o Interim Phase 1b data o Select and advance 2 nd generation CpAM H2 2018 o Phase 2a initial POC data H1 2018 o Phase 2a initiation x ABI - H0731 was safe and well tolerated in Phase 1a study x Phase 1b initiated (preliminary an
Slide 17: Robust HBV Pipeline Focused on Targeting cccDNA
Robust HBV Pipeline F ocused on Targeting cccDNA 17 Program Drug Candidate Discovery Lead Op / Selection IND Enabling Phase 1 Phase 2 ABI - H0731 ( CpAM ) CpAM 2 nd Generation CpAM 3 rd Generation Novel Target Hepatitis B • Lead product ABI - H0731 completed Phase 1a earlier this year, and initiated
Slide 18: Microbiome Program
Microbiome Program assembly blosclences
Slide 19: The Microbiome: Realizing the Promise
The Microbiome : Realizing the Promise Modulating the gut microbiome has the potential to revolutionize the management of a broad range of therapy areas Infectious diseases GI disorders Oncology Metabolic diseases Neurosciences/CNS PoC data has demonstrated that fecal microbiota transplants (FMT) ca
Slide 20: The Microbiome: Challenges
The Microbiome : Challenges 20 Mechanisms of disease pathology must be considered in context with environment, genome and microbiota Lack of consistency, not scalable, not controlled, unreliable, poor route of administration Diversity is needed for optimal microbiotic therapeutics Unique microhabita
Slide 21: ASMB Solution: Proprietary Microbiome Platform
ASMB Solution: Proprietary Microbiome P latform Proprietary & scientifically rigorous, rational strain selection methodologies, including: • Human FMT studies • Sequencing and analysis protocols • Pathology - driven mechanisms • In vitro and in vivo models Differentiated Manufacturing Approach • Iso
Slide 22: GEMICEL: ASMB's proprietary targeted delivery technology
First/outer capsule: for bolus release of API in ileum Second/inner capsule: for bolus release of API in right colon GEMICEL®: ASMB’s proprietary targeted delivery technology 22 • GRAS ingredients • Manufacturing amenable to biologic products GEMICEL® Capsule The colon is an attractive site for drug
Slide 23: Assembly and Allergan Enter Microbiome Collaboration
23 Expertise in microbiome therapeutics, fully - integrated platform Expertise in gastrointestinal drug development and commercialization Collaboration Highlights: • Rights for GI development programs: ‒ ABI - M201 for Ulcerative Colitis (UC) ‒ ABI - M301 for Crohn’s Disease (CD) ‒ 2 compounds targe
Slide 24: Summary: AGN/ASMB Microbiome Collaboration
Summary: AGN/ASMB Microbiome Collaboration 1. Expedites our efforts into multiple GI indications 2. Leverages our end - to - end microbiome technology platform 3. Advances our ability to move microbiome candidates rapidly into clinical development with strategic partners 4. Aligns with our strategic
Slide 25: Microbiome Program pipeline
Microbiome Program Discovery Lead Selection IND Enabling Phase 1b ABI - M201 (Ulcerative Colitis ) ABI - M301 (Crohn’s Disease) IBS Compounds NASH, I/O & Other Clostridium difficile ( C.diff ) Gemicel ® (targeted oral delivery system) Other Indications for Our Microbiome Platform Gastrointestinal On
Slide 26: Financial Summary
Financial Summary 26 Nasdaq ASMB Cash, cash equivalents & marketable securities ~$78.6M as of June 30, 2017 Shares outstanding ~ 17.3M Fully diluted ~ 22M
Slide 27: 2017 ASMB Anticipated Milestones
2017 ASMB Anticipated Milestones 27 x Microbiome collaboration for GI indications x ABI - H0731 dose ranging Ph 1a portion complete x Initiate ABI - H0731 Ph 1b trial x AASLD: 3 abstracts accepted □ H2 : ABI - H0731 Ph 1a safety and PK profile □ H2: ABI - H0731 Phase 1b (interim) results □ H2: 2nd G
Slide 28: Assembly Biosciences: Overview
28 Delivering oral, synthetic live biotherapeutics to targeted location Allergan partnership for GI assets, rest of pipeline wholly owned HBV Deep pipeline of CpAMs with potential to achieve CURE Large market opportunity with high unmet need Microbiome Team Industry leaders w/ expertise in drug disc
Slide 29: Thank You
Thank You ( A) assembly losciences
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