Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Presentation (January 2016)
22 slides from Assembly Biosciences's investor presentation, Jan 12, 2016
Slide 1: Assembly Biosciences January 2016
Assembly Biosciences Nasdaq: ASMB January 2016
Slide 2: Cautionary Note Regarding Forward-Looking Statements
C autionary Note Regarding Forward - Looking Statements The information in this presentation contains estimates and other forward - looking statements regarding future events, including statements about the therapeutic potential of our HBV and CDI programs, timing of the initiation of our planned cl
Slide 3: Investment Highlights
Investment Highlights An integrated i nfectious disease company Focus on infectious diseases Developing best - in - class small molecules for the treatment of chronic hepatitis B (HBV) Developing drug - like oral microbiotic (MB) therapeutics for the prevention of recurrent C. difficile infection (
Slide 4: Experienced Scientific & Leadership Team
Experienced Scientific & Leadership Team Proven leadership team producing successful companies & drugs for the past 30+ years Derek A. Small President & CEO Richard Colonno, PhD Chief Science Officer Uri Lopatin , MD CMO & VP R&D Thomas Rollins Chief Development Officer Head of Microbiome Lee Arnold
Slide 5: HBV Opportunity
Va =. ZN a ) HBV Opportunity ZZ assembly biosciences
Slide 6: HBV Opportunity: More Patients Diagnosed/Treated
HBV Opportunity: More Patients Diagnosed/Treated ~ 240M chronically infected globally – 90M in China (top 5 health priority for s FDA ); > 10M EU; ~ 2M in USA (1) – Current standard of care is not curative in >90% of patients, yet represents >$1B/year market globally and is expected to grow signific
Slide 7: ASMB Goal: Curative Therapy for HBV
ASMB Goal: Curative Therapy for HBV Current therapies are inadequate – Interferons have poor tolerability, efficacy and are not an option for many patients – Polymerase inhibitors are highly effective in inhibiting viral replication and driving viral loads to undetectable levels, but low level viral
Slide 8: HBV Core Protein: Required Throughout Lifecycle
HBV Core Protein: Required Throughout Lifecycle HBV Core protein ( Cp ) is a non - enzymatic viral protein with no human homologue and is involved in entire HBV viral lifecycle (upstream & downstream) – Believed to play a critical role i n the generation and maintenance of cccDNA – R equired for cre
Slide 9: ASMB Progress: Prevent cccDNA
ASMB Progress: AB - 101 to Prevent cccDNA Strong patent position on CpAMs (Core protein Allosteric Modifiers ) compositions and uses for HBV Potent lead series – Alters Cp oligomer conformation • Reduces pgRNA packaging • Inhibits rcDNA formation • Reduces rcDNA delivery to nucleus – Prevents synthe
Slide 10: Ongoing Programs: Inhibit HBV Proteins
Ongoing Programs: Inhibit HBV Proteins CpAMs effect cytoplasmic and nuclear localization as well as intrinsic functions of Cp Untreated cells Entecavir t reated c ells CpAM t reated c ells Mechanisms by which altered CpAM localization can contribute to HBV cure – Potential to alter antigen presentat
Slide 11: Eliminate (or Silence) Existing cccDNA
Eliminate (or Silence) Existing cccDNA Persistent active cccDNA requires both host and viral proteins Only two viral proteins are associated with active cccDNA: Cp and HBx ASMB has initiated internal and external research to explore HBx and host related targets in addition to Cp Source: Bock , Ct et
Slide 12: ASMB HBV Pipeline & Projected Timeline
ASMB HBV Pipeline & Projected Timeline 2015 2016 2017 Therapeutics 1st generation HBV CpAM 2 nd generation HBV CpAM HBV upstream mechanism HBV upstream mechanism Novel target(s) Ph 1a/1b IND enabling s tudies Candidate selection Ph 1a/1b IND enabling studies Candidate selection IND enabling Candidat
Slide 13: Microbiome Opportunity
Va =. aN ¢ A) Microbiome Opportunity ZZ assembly biosciences
Slide 14: Microbiome Opportunity: FMT as a Starting Point
~500,000 cases of CDI yearly, 77% higher chance of being readmitted within 30 days, 55% longer hospital stay and 13% higher risk of mortality Dysbiotic microbiome linked to multiple diseases Microbiome Opportunity: FMT as a Starting Point 14 - Irritable b owel syndrome (IBS) - Metabolic disease - Ot
Slide 15: Fecal Microbiota Transplant Works, But...
Fecal Microbiota Transplant Works , But ….. Widespread adoption of fecal microbiota transplantation (FMT) is problematic FMT is a procedure not a product FMT is a true transplant of human - derived biological material Risks of infection transmission can’t be fully resolved Poor patient and health ca
Slide 16: ASMB's Proposed Solution: 'Druggable' Microbes
ASMB’s Proposed Solution : ‘Druggable’ Microbes Replace FMT with microbiotic therapy What success in replacing FMT looks like – An oral “biologic” pill – Efficacy that meets or exceeds FMT response Assembly program plans to combine two differentiated components – cGMP production of systematically -
Slide 17: GEMICEL Formulation
GEMICEL™ Formulation Our proprietary targeted delivery technology enables direct treatment in the colon Controlled release p latform Targeted d elivery Second inner c oated for bolus release of drug A in right colon First outer c oated for bolus release of drug A in ileum • GRAS ingredients • Manufa
Slide 18: Integrated Discovery & Manufacturing Engine
Rigorous scientific development engine for bacterial therapy selection Integrated Discovery & Manufacturing Engine Proprietary selection methodologies Human FMT studies Scientifically rigorous sequencing and analysis including machine based algorithms in vitro and in vivo models Systems that assure
Slide 19: AB-M101 Anticipated Clinical Candidate for rCDI
Efficacy : AB - M101 oral capsules will incorporate select strains of vegetative bacteria to achieve similar efficacy and safety as FMT in the treatment of rCDI – We believe that a therapy including both spore and non - spore forming bacteria, delivered specifically to the lower GI tract, can be bes
Slide 20: ASMB MB Pipeline & Projected Timelines
ASMB MB Pipeline & Projected Timelines 2015 2016 2017 Platform and therapeutics Microbiome strain library AB - M101 – ( rCDI ) AB - M102 – New indication AB - M103 – New indication GEMICEL™ d elivery technology Ongoing strain selection for library expansion of additional products IND enabling studie
Slide 21: Financial Summary
Financial Summary 21 21 Strong Base Of Biotech Investors Baker Brothers Nasdaq ASMB Cash, cash equivalents & marketable securities ~$97M as of September 30, 2015 Shares outstanding ~17.2M Fully diluted ~20.4M
Slide 22: Investment Summary
Investment Summary Experienced team with proven track record and deep industry experience HBV platform : D eveloping oral small molecules for HBV cure – Primary focus on Cp , as inhibitors have the potential to disrupt multiple aspects of viral life cycle – Continuing to explore other targets that m
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