Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Presentation (January 2017)
25 slides from Assembly Biosciences's investor presentation, Jan 9, 2017
Slide 1: Assembly Biosciences, Inc. Corporate Presentation January 2017
Assembly Biosciences, Inc. Corporate Presentation January 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
Assembly Biosciences 3 x Microbiome collaboration in GI with Allergan o ABI - H0731 Phase 1 a & 1b results o Initiate ABI - H0731 1b/2a trial and results o Initiate 1b c.diff trial and results Clinical - stage company focused on two key areas of drug development x Expansion of senior leadership team
Slide 4: Announcement Today: Assembly and Allergan enter Microbiome Collaboration
Announcement Today: Assembly and Allergan enter Microbiome Collaboration • January 9, 2017 Announcement • Allergan and Assembly collaboration to jointly develop Assembly’s microbiome compounds for IBD and IBS • Partnership includes license to: • ABI - M201 for Ulcerative Colitis • ABI - M301 for Cro
Slide 5: 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 - M101 (Recurrent C.diff. ) ABI - M201 (Ulcerative Colitis ) ABI - M301 (Crohn’s Disease) IBS com
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, >1 million in US CURE Eliminate (or Silence) Existing cccDNA Inhibit cccDNA Formation Prevent Virus Replication Limited to nucleos(t)ide analoges (Nucs) [entecavir, tenofovir] or pe
Slide 8: ASMB Virology Goal: Curative Therapy for HBV
ASMB Virology Goal : Curative Therapy for HBV • 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 maintenance of cccDNA and pgRNA enc
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
Microbiome Program la 4) a ssembly
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/C NS PoC data has demonstrated that fecal microbiota transplants (FMT) w
Slide 14: The Microbiome: Challenges
The Microbiome : Challenges 14 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 15: Solution: Proprietary Microbiome Platform
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 • Isolatio
Slide 16: GEMICEL: Targeted delivery to the colon
First/outer capsule: for bolus release of API in ileum Second/inner capsule: for bolus release of API in right colon GEMICEL®: Targeted delivery to the colon 16 • GRAS ingredients • Manufacturing amenable to biologic products GEMICEL® Capsule The colon is an attractive site for drug delivery, partic
Slide 17: ABI-M101 oral capsules for rCDI
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 18: Disclosed key terms for Allergan collaboration
Disclosed key terms for Allergan collaboration 18 Leader in microbiome therapeutics, fully - integrated platform Expertise in gastrointestinal drug development and commercialization Collaboration Highlights: • Rights for GI development programs: ‒ MBI - 201 for Ulcerative Colitis (UC) ‒ MBI - 301 fo
Slide 19: Summary: AGN/ASMB Microbiome Collaboration
Summary: AGN/ASMB Microbiome Collaboration Today’s announcement supports important aspects of Assembly’s microbiome technology platform moving forward: 1. Expedites our efforts into multiple GI indications 2. Leverages our end - to - end microbiome technology platform 3. Advances our ability to move
Slide 20: Microbiome Program
Microbiome Program Discovery Lead Selection IND Enabling Phase 1b ABI - M101 (Recurrent C.diff. ) ABI - M201 (Ulcerative Colitis ) ABI - M301 (Crohn’s Disease) IBS Compounds Other Indications Gemicel® (targeted oral delivery system) Other Indications for Our Microbiome Platform Clinical POC achieved
Slide 21: Financial Summary
Financial Summary 21 Nasdaq ASMB Cash, cash equivalents & marketable securities ~$66.5M as of Sept. 30, 2016 Q1 2017: ~$50 M upfront from Allergan collaboration Shares outstanding ~17.2M Fully diluted ~ 21.6M
Slide 22: 2016 ASMB Milestones and 2017 Plans
2016 ASMB Milestones 22 x Expansion of leadership team x ABI - H0731 candidate selection x Ph 1 HBV trial initiation x Increased inventory of microbiome drug substance x ABI - M101 candidate selection 2017 x Microbiome collaboration for GI indications □ H1: ABI - H0731 Ph 1a/b safety & PK profile □
Slide 23: 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 24: Thank You
Thank You Sf assembly blosclences
Slide 25: (Appendix Slide) HBV Lifecycle
(Appendix Slide) HBV Lifecycle • rcDNA: relaxed circular DNA • cccDNA: covalently closed circular DNA • pgRNA: pre - genomic RNA • RT: reverse transcription cccDNA Formation cccDNA Amplification cccDNA Maintenance pgRNA Encapsidation Polymerase Inhibition 25 • HBV Core Protein plays a critical role
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