Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Presentation (November 2016)
23 slides from Assembly Biosciences's investor presentation, Nov 9, 2016
Slide 1: Assembly Biosciences, Inc. Corporate Presentation November 2016
Assembly Biosciences, Inc. Corporate Presentation November 2016
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 therapeutic potential of our HBV - Cure and Microbiome programs, timing of the initiation of
Slide 3: Company Highlights
CURE Eliminate (or Silence) Existing cccDNA Inhibit cccDNA Formation Preven t Virus Replication HBV - Cure Program Microbiome Program • A potent series of direct acting oral antivirals that selectively target HBV to increase the current low cure rate • ASMB lead series of CpAMs target HBV Core Prote
Slide 4: Experienced Leadership Team
“ Experienced Leadership Team Proven leadership team producing successful companies and drugs Previous companies Drugs discovered, developed, commercialized 4 Derek A. Small President & CEO David Barrett CFO & COO Uri Lopatin , MD Chief Medical Officer Richard Colonno , PhD Chief Science Officer, HB
Slide 5: Pipeline
ABI - H0731 (CpAM 1 st Generation) Q4 16: Initiated Ph 1 CpAM 2 nd Generation H1 17: Preclinical profile CpAM 3 rd Generation H2 17: Preclinical profile HBV Novel Target Development P ipeline HBV Program Discovery/Research Lead Selection/ Optimization IND Enabling Clinical Trials Near Term Milestone
Slide 6: Hepatitis B Cure Program
Hepatitis B - Cure Program ©) assembly blosclonces
Slide 7: Significant need for curative therapies for Hepatitis B
Significant need for curative therapies for Hepatitis B Market Opportunity ~ 240 million patients worldwide, ~90 million in China, >1 million in US CURE Eliminate (or Silence) Existing cccDNA Inhibit cccDNA Formation Prevent Virus Replication Limited to nucleos (t)ide analoges ( Nucs ) [ entecavir ,
Slide 8: ASMB Goal: Curative Therapy for HBV
ASMB Goal: Curative Therapy for HBV • HBV lifecycle is a complex process • Nucs only inhibit reverse transcription • HBV Cure requires inhibiting the formation of new cccDNA and/or silencing of existing cccDNA • HBV core protein is believed to be involved in the amplification, formation and maintena
Slide 9: CpAMs inhibit cccDNA formation in PHH cells
CpAMs inhibit cccDNA f ormation in PHH cells 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 ABI - H0808 80 196 310 305 ETV <0.1 Incomplete Incomplete Incomplete Viral DNA HBeAg HBsAg pgRNA CpAM - ABI
Slide 10: CpAMs block cccDNA formation in HBV infected cells
CpAMs block cccDNA formation in HBV infected c ells HepG2 - NTCP 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 formati
Slide 11: Lead Candidate ABI-H0731 and HBV Pipeline
Lead Candidate ABI - H0731 and HBV Pipeline • Unique mechanism(s) that target core protein and affect cccDNA – Not seen with current SOC • F avorable drug characteristics and PK profile in multiple species ‒ Balances potent antiviral effect with favorable drug properties • Initiated Phase 1 trial in
Slide 12: Microbiome Program
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Slide 13: 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) wi
Slide 14: Mechanisms of disease pathology must be considered in context
Mechanisms of disease pathology must be considered in context with environment, genome and microbiota Immune • Innate and adaptive immune • Inflammatory mediators • Oxidative stress Non - immune • Mucosal/epithelial barrier Strain selection guided by pathophysiological mechanism 14 • Immunopathogeni
Slide 15: Unique microhabitats require targeted delivery
Unique microhabitats require targeted delivery 15 Characteristics of Human Microbiota • Adult intestinal microbiota is partially stable • ~ 40 bacterial species • Consortia defined by • Founder effects • Immune system • Diet • Bacteria microhabitats • pH • Oxygen • Antimicrobials • Nutrient availabi
Slide 16: Diverse phyla require unique drug formulation
Diverse phyla require unique drug formulation Diversity is needed for optimal microbiotic therapeutics 16 Reference: SILVA. Available at: http://www.arb - silva.de /, rRNA gene database ASMB can deliver multiple phyla to optimize microbiotic therapeutics
Slide 17: Solution: Our best-in-class microbiome platform
Solution: Our best - in - class m icrobiome 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
Slide 18: ABI-M101 efficacy, regulatory, IP and patient preference
Efficacy: ABI - M101 oral capsules incorporate select strains of vegetative bacteria to achieve similar efficacy and safety profile as FMT in the treatment of rCDI • We believe that a therapy including both spore and non - spore forming vegetative bacteria, delivered specifically to the lower GI tra
Slide 19: Microbiome Pipeline and Platform
ABI - M101 (Recurrent CDI ) ABI - M201 (undisclosed indication) ABI - M301 (undisclosed indication) Other Indications Gemicel ® Platform for Additional Indications Microbiome Pipeline and Platform Multiple Clinical POC Programs planned in 2016 & 2017 Gastrointestinal UC Crohn’s IBS Oncology Immuno -
Slide 20: 2016/2017 Milestones
x Q3 2016 – File CTA (IND equivalent) for ABI - H0731 x Q4 2016 – Initiate ABI - H0731 Phase 1a/1b trial □ Q1 2017 – File IND for ABI - M101 □ H1 2017 – Initiate ABI - M101 Phase 1 trial in c.diff □ Q2 2017 – Initiate ABI - H0731 Phase 1b/2a trial □ H2 2017 – ABI - H0731 Phase 1 safety profile and r
Slide 21: Financial Summary
Financial Summary Nasdaq ASMB Cash, cash equivalents & marketable securities ~$75M as of June 30, 2016 Shares outstanding ~17.2M Fully diluted ~20.4M 21
Slide 22: Investment Summary
Investment Summary HBV platform: Developing direct acting oral antivirals for HBV cure – Lead product ABI - H0731 initiated Phase 1a study – Next generation molecules to follow – Primary focus on modulating HBV Core Protein, with affects on multiple parts of the viral cycle, including inhibition of
Slide 23: Thank You
Thank You ® assembly blosclonces
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