Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Presentation (August 2014)
21 slides from Assembly Biosciences's investor presentation, Aug 13, 2014
Slide 1: Assembly Biosciences August 2014
Assembly Biosciences August 2014
Slide 2: 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: risks related to the costs, t
Slide 3: Assembly Biosciences – Overview
Russell Ellison, MD – CEO, Chair – Pegasys and multiple other drugs on market Derek A. Small – Pres , COO – Founding CEO of multiple biotechs David Barrett – CFO – CFO; director at multiple biotechs Uri Lopatin , MD – CMO, VP R&D - Led HBV programs at leading pharma Lee Arnold, PhD – CSO – Discovere
Slide 4: Why HBV?
4 Why HBV?
Slide 5: HBV by the Numbers
HBV by the Numbers Piper Jaffray est. $15B/yr. W/W RBC est. $10B/yr.+ W/W WW sales of $3.2B in 2012; most patients remain untreated or untreatable 2 million chronically infected In North America 14 million chronically infected in Europe 112 million chronically infected in Asia - Pacific More than 35
Slide 6: HBV is Both Underdiagnosed and Undertreated
* Wursthorn , K. et al. Kinetics of hepatitis B surface antigen decline during 3 years of telbivudine treatment in hepatitis B e antigen - positive patie nts. Hepatology 52 , 1611 – 1620 ( 2010) . ** 2012 EASL HBV Management Guidelines • Current Therapy Suppresses Virus, but Does Not Cure • Lack of
Slide 7: Current Targets in Development
Current Targets in Development • Immunomodulatory (Gilead, Gilead/Globimmune) • Mechanism: Adaptive and innate immune stimulation • Immune mediated suppression or clearance of infected cells, and/or • N on - specific, poorly understood mechanism of host mediated cccDNA clearance/suppression • Risk:
Slide 8: HBV Life Cycle & Current Approaches
8 HBV Life Cycle & Current Approaches Current therapies target downstream of intra - nuclear viral reservoir cccDNA directed therapy is required for “cure” cccDNA rcDNA ( capsid) rcDNA capsid Envelope (HBsAg) vRNA vRNA Viral Proteins: Cp (E) X Pol HBsAg (S,M,L) vRNA + Pol capsid Secreted HBsAg ( and
Slide 9: Why Core Protein?
Why Core Protein? 9
Slide 10: HBV Life Cycle – Assembly's CpAM Approach
10 cccDNA directed therapy is required for “cure” = Assembly Pharma CpAMs cccDNA rcDNA ( capsid) rcDNA capsid Envelope (HBsAg) vRNA vRNA Viral Proteins: Cp (E) X Pol HBsAg (S,M,L) vRNA + Pol capsid Secreted HBsAg ( and HBeAg ) Nuclear Membrane Cell Membrane HBV Life Cycle – Assembly’s CpAM Approach
Slide 11: Core Protein – One Target, Many Functions
HBV core protein is pleiotropic - it has multiple functions in the HBV lifecycle free capsid Zlotnick, Tan, Selzer. (2013) Structure 21 , 6 - 8. 11 Deep understanding of core protein is a differentiating advantage for Assembly Core Protein – One Target, Many Functions
Slide 12: Assembly Leadership on Cp is Based on Seminal Research
Assembly Leadership on Cp is Based on Seminal Research and Current Scientific Leadership of Founder Zlotnick 12 HBV Capsid Assembly Kinetics d escribed (Zlotnick et al.) • HBV Meeting “gedanke - experiment” • HBc binds to the CpG islands of HBV cccDNA and promotes an epigenetic permissive state. Zlo
Slide 13: Assembly's CpAMs – Potential Key Advantages
Assembly’s CpAMs – Potential Key Advantages • Convenience : Oral • Single Agent or Combination Opportunity : • Potential to be combined with other classes of compounds • Immunology : • CpAMs are not dependent on immune response, but patients can expect the same benefits as immune modulating approach
Slide 14: HBV Pipeline & Expected Upcoming Milestones
HBV Pipeline & Expected Upcoming Milestones 2014 Stage of Development Product Disease Area Lead ID IND Dev. Ph I Ph II Ph IIII CpAM Gen 1 HBV CpAM Gen 2 1H 2015 Initiate Phase I/ Ib Studies ( Aus /NZ) Select Gen 1 clinical c andidate Discovery Hits HBV Over Next ~18 Months Expected Upcoming Mileston
Slide 15: Intellectual Property Portfolio: HBV & CpAMs
Intellectual Property Portfolio: HBV & CpAMs The patent applications fall broadly into two categories: 1. Platform patent applications • Multiple platform patent applications filed, with others in process • Patent applications cover novel mechanism of action, methods of treatment, novel assays, and
Slide 16: Microbiome Program: C. difficile
Microbiome Program: C. difficile 16
Slide 17: Microbiome: Overview
Microbiome: Overview • We have in - licensed technology for targeted delivery of complex agents to select regions of the GI tract • Our delivery technology exploits specific pH gradients in the gut and new coating technologies in conjunction with state of the art encapsulation technology • This is d
Slide 18: Microbiome: Program Overview (Cont.)
Research Plan: • Identification of lead candidates from successful FMT patients (Brown, RI) by MIT, Roswell Park, Buffalo U. • Parallel completion of CMC development to be ready for IND, and then commercial supply for PH IIb /III Expected Milestones • Proof of Principle for coating fastidious anaero
Slide 19: ASMB Combined HBV & Microbiome Pipeline
ASMB Combined HBV & Microbiome Pipeline TBD Current & Planned Stage of Development Product Disease Area Lead ID IND Dev. Ph I Ph II Ph IIII CpAM Gen 1 HBV CpAM Gen 2 MB Gen 1 C. difficile Hits CpAM Other Other Virus (TBD) Selecting lead Rich science at the Core Protein and Microbiome Therapeutics pr
Slide 20: Financial Summary
20 Financial Summary • Nasdaq : ASMB • ~$20 mm on balance sheet (6/30/2014) • Cash runway enables multiple milestones into Q4 - 2015 under current development plan • Current S - 3 filing provides flexibility to opportunistically access the capital markets • Approximately 8.6 mm shares outstanding •
Slide 21: Assembly Biosciences
21 Assembly Biosciences Infectious Disease Focused Potential Curative Therapy for Hepatitis B and Clostridium difficile Two Proprietary Technology Platforms Pipeline Offers First - in - class and Best - in - class Product Opportunities Experienced Management and R&D Team Cash Allows Achievement of V
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