Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Presentation (January 2016)
22 slides from Assembly Biosciences's investor presentation, January 2016
Slide 1: Assembly Biosciences
Assembly Biosciences Nasdaq: ASMB January 2016
Slide 2: Cautionary Note Regarding Forward-Looking Statements
Cautionary 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 clinica
Slide 3: Investment Highlights
Investment Highlights An integrated infectious disease company ¿ Developing best-in-class small molecules for the treatment of chronic Focus on hepatitis B (HBV) infectious diseases ¿ Developing drug-like oral microbiotic (MB) therapeutics for the prevention of recurrent C. difficile infection (rCDI
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 Richard Colonno, PhD Uri Lopatin, MD President & CEO Chief Science Officer CMO & VP R&D Thomas Rollins Chief Development David Barrett Lee Arnold, PhD Officer
Slide 5: HBV Opportunity
_ (4) HBV Opportunity wT assembly bioseTrences
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 sFDA); >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 significant
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 vir
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 in the generation and maintenance of cccDNA – Required for creatin
Slide 9: ASMB Progress: AB-101 to Prevent cccDNA
ASMB Progress: AB-101 to Prevent cccDNA Significant progress in 2015 – building R&D engine ¿ 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 •
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 Effect on Cp in HBV infected cells following treatment with CpAM vs ETV Untreated cells Entecavir treated cells CpAM treated cells ¿ Mechanisms by which altered CpAM localiza
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 Cp binds to cc
Slide 12: ASMB HBV Pipeline & Projected Timeline
ASMB HBV Pipeline & Projected Timeline Prevent Inhibit New cccDNA HBV Proteins CURE Eliminate (or Silence) Existing cccDNA 2015 2016 2017 Therapeutics 1st generation HBV Candidate selection IND enabling studies Ph 1a/1b Ph 2 CpAM 2nd generation HBV Research Candidate IND enabling Ph 1a/1b CpAM selec
Slide 13: Microbiome Opportunity
_ —N (4) Microbiome Opportunity NES) assembly bioseTrences
Slide 14: Microbiome Opportunity: FMT as a Starting Point
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 - Primary CDI - Irritable bowel syndrome (IBS) - Other in
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 healt
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 platform Targeted delivery GI tract has number of defenses that make colonization challenging Second inner coated 6 meters 1.5M for bolus release of drug A in right colon Small
Slide 18: Integrated Discovery & Manufacturing Engine
Integrated Discovery & Manufacturing Engine Rigorous scientific development engine for bacterial therapy selection Strain Selection Development / Manufacturing Proprietary selection methodologies Systems that assure proper design, ¿ Human FMT studies monitoring and control of selected strains requir
Slide 19: MB Anticipated Clinical Candidate For rCDI
MB Anticipated Clinical Candidate For rCDI Initial focus: AB-M101 as a best-in-class therapy for treatment of 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 thera
Slide 20: ASMB MB Pipeline & Projected Timelines
ASMB MB Pipeline & Projected Timelines 2015 2016 2017 Platform and therapeutics Microbiome strain Ongoing strain selection for library expansion of additional products library Candidate selection IND enabling Ph 2/3 Ph 1B AB-M101 – (rCDI) studies AB-M102 – New Candidate selection IND enabling Ph 1B
Slide 21: Financial Summary
Financial Summary Nasdaq ASMB Cash, cash equivalents & ~$97M as of September 30, 2015 marketable securities Shares outstanding ~17.2M Fully diluted ~20.4M Strong Base Of Biotech Investors Baker Brothers 21
Slide 22: Investment Summary
Investment Summary ¿ Experienced team with proven track record and deep industry experience ¿ HBV platform: Developing 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
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