Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Presentation (September 2017)
37 slides from Assembly Biosciences's investor presentation, Sep 26, 2017
Slide 1: Assembly Biosciences, Inc. Corporate Presentation
Assembly Biosciences, Inc. Corporate Presentation September 26, 2017 Nasdaq: ASMB 1
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 clinical and therapeutic potential of our HBV - Cure and Microbiome programs, our ability to r
Slide 3: Two Innovative Drug Development Platforms
3 Assembly Biosciences: Two INNOVATIVE Drug Development Platforms HBV Microbiome • SOC does NOT CURE • Eliminating cccDNA (responsible for viral persistence) • FMT is inadequate as therapy • Establish CpAMs as critical component of HBV curative backbone • Develop oral, synthetic, live biotherapeutic
Slide 4: 4 Development Programs in Large Markets with High Unmet Need
Discovery Lead Optimization IND Enabling Phase 1a Phase 1b/2a Phase 2 Discovery Lead Op / Selection IND Enabling Phase 1 b 4 Development Programs in Large Markets with High Unmet Need Microbiome Hepatitis B ABI - H0731 ( CpAM ) ABI - H2158 ( CpAM ) 3 rd CpAM Novel Target Clostridium difficile ( C.di
Slide 5: Key Priorities Through 2018
Collaborations - Potential to achieve significant milestone - Continue to monetize platform capabilities Microbiome Assets - Advance AGN partnered programs in UC and Crohn’s, IBS - Development of additional portfolio assets (i.e. NASH, I/O) Next Generation CpAMs - ABI - H2158 selected as 2 nd CpAM c
Slide 6: Potential Value Drivers: 3+ Months
H2 2017 ►►► 2018 ►►► 6 Potential Value Drivers: 3+ Months ABI - H0731: Phase 1b Antiviral potency and decrease in viral DNA Third CpAM candidate selection ABI - H0731 : Phase 2a combo w/ nuc Clinical Data Pipeline Progress • KEY Multiple near term milestones AASLD • ABI - H0731: Phase 1a data • Next
Slide 7: Potential Value Drivers Over the Next 18 Months
H2 2017 ►►► 2018 ►►► 2019 7 Potential Value Drivers Over the Next 18 months ABI - H0731 : Phase 1b Antiviral potency and decrease in viral DNA ABI - H0731 : Phase 2 Longer term effects for cure Establish clinical candidate selection in NASH and I/O Advancement of partnered microbiome programs to sig
Slide 8: Hepatitis B: Cure Program
Hepatitis B: Cure Program Market overview and deficiencies of current treatment Assembly’s HBV Cure Program 8
Slide 9: Over 300 Million Chronically Infected Patients Globally
Low cure rates with current SOC /Vaccines are Preventative but do not cure those who are chronically infected HBV: Over 300 Million Chronically Infected Patients Globally China Prevalence: ~ 90 million Global Prevalence: >300 million U.S. Prevalence: ~1.5 million EU Prevalence: ~20 million Sources:
Slide 10: Putting the HBV Opportunity into Perspective
Number of HBV patients outnumber HCV + HIV combined with high unmet need for diagnosis and cure 10 Putting the HBV Opportunity into Perspective HCV HBV US Prevalence 3 M 1.2 - 3 M Worldwide Prevalence 160 M 350 M % Diagnosed in US 50% 30% % Diagnosed that are Treated in US 33% 6 - 10% Nature RNA vir
Slide 11: Current Treatment Paradigm is Inadequate to Cure HBV
11 Current Treatment Paradigm is Inadequate to CURE HBV Mechanism of Action Benefits Limitations Nucleo (t)side Analogs • Inhibit DNA polymerase • Knock out replication virus from RNA to DNA (reverse transcription) • 1x daily dosing • Well tolerated • High resistance barrier • Does NOT completely st
Slide 12: Clear Evidence that ETV Can Cure Woodchucks
Thought we had curative therapy in 2005 Clear evidence that ETV can cure woodchucks based on multiple parameters • ETV exhibited potent activity against Woodchuck HBV • Sustained suppression of viral DNA levels (≥ 8 logs) for 1 - 3 years with weekly treatment – N o rebounds or evidence of resistance
Slide 13: Aspirational Objectives for Clinical Cure in Humans
We want what we achieved in woodchucks! 13 Aspirational Objectives for Clinical Cure in Humans • Defined length of treatment to remission • Convenient dosing (QD?) and low pill burden • Excellent safety profile, with minimal side effects • Sustained remission off therapy • Viral DNA replication rema
Slide 14: CpAMs Are Only Modality that Stop cccDNA Generation
CpAMs are only modality that stop cccDNA generation HBV Life Cycle: A DNA Virus with Limited Targets cccDNA Generation 14 cccDNA Maintenance
Slide 15: Rationale for CpAM Treatment Resulting in Higher Cure Rates
15 Rationale for CpAM Treatment Resulting in Higher Cure Rates? • Inhibit HBV life cycle at several steps • Inhibit cccDNA generation in Primary Human Hepatocytes • cccDNA exhibit a reasonable half - life ( weeks to months) • ASMB CpAMs : Potent with Excellent Drug - Like Characteristics – In Vitro
Slide 16: CpAMs Inhibit Several Steps of HBV Life Cycle
CpAMs CpAMs Inhibit Several Steps of HBV Life Cycle 16 cccDNA Maintenance cccDNA Generation Nucs & Non - Nucleoside RNA Interference CpAMs cccDNA Maintenance Generate empty aberrant capsid formation – Bind to dimer - dimer interface disrupting formation of functional nucleocapsids Prevent encapsidat
Slide 17: Half-life of cccDNA May Be Shorter Than Previously Believed
New study shows that the half - life of cccDNA may be shorter than previously believed 17 Measuring the half - life of cccDNA All biological molecules have a half - life, including cccDNA • Longitudinal study conducted on samples (serum and biopsies) from patients with emerging resistance to LVD and
Slide 18: Hepatitis B: Cure Program
18 Hepatitis B: Cure Program Market overview and deficiencies of current treatment Assembly’s HBV Cure Program
Slide 19: ASMB CpAMs: Potent with Excellent Drug-Like Characteristics
19 ASMB CpAMs : Potent with Excellent Drug - Like Characteristics Potent and Predictable • Intrinsic potency to significantly inhibit new cccDNA as measured by surrogate markers such as pgRNA and HBsAg • EC 50 – 154 nanomolar potency in Primary Human Hepatocytes (PHH) • 10 - 30x exposure in liver ab
Slide 20: ABI-H0731: Lead Molecule from a Deep Pipeline of CpAMs
Lead molecule from a deep pipeline of CpAMs 20 ABI - H0731: Candidate Summary Phase 1b/2a currently enrolling Phase 1a Complete: Dose ranging well tolerated in all cohorts Single doses 100 - 1,000 mg, multiple doses 800 mg QD and 800 mg BID x 7 days • Favorable PK profile with a half - life consiste
Slide 21: CpAMs Inhibit cccDNA Generation in Primary Human Hepatocytes
Viral DNA, pgRNA , HBeAg and HBsAg in Primary Human Hepatocytes CpAMs Inhibit cccDNA Generation in Primary Human Hepatocytes 21 Compound Viral DNA HBeAg HBsAg pgRNA ABI - H0731 154 2,210 3,000 1,840 ETV <0.1 Incomplete Incomplete Incomplete • CpAMs reduced viral HBV DNA levels and known surrogate ma
Slide 22: ABI-H0731: General Clinical Investigational Plan
ABI - H0731: General Clinical Investigational Plan Phase 2/3 development will be determined on outcomes of Phase 2 studies • Possible scenarios are 6 to 12 month treatment with 6 to 12 month follow up to seek increased cure rate claims • Early approval scenarios may be feasible if significant viral
Slide 23: ABI-H2158: Next-Generation CpAM Candidate Summary
Next - generation CpAM selected from proprietary screening 23 ABI - H2158: Candidate Summary Highly Potent while maintaining favorable drug - like characteristics ABI - H2158 expected to enter clinic in 2018 ABI - H2158 Candidate profile to be presented at AASLD, October 2017 in Washington, DC Key P
Slide 24: Robust HBV Portfolio Focused on Potential Curative Therapies
Robust HBV Portfolio Focused on Potential Curative Therapies • Lead product ABI - H0731 completed Phase 1a earlier this year, and initiated Phase 1b/2a in HBV patients • Second CpAM , ABI - H2158 selected to advance to IND enabling studies • Third CpAM in research stage nearing candidate selection 2
Slide 25: Investment Summary for ASMB CpAMs
x CpAMs disrupt viral replication at multiple steps x CpAMs block the generation of new cccDNA molecules (SOC/ nuc’s do not effect cccDNA ) x Multiple distinct and proprietary chemical scaffolds x ASMB CpAMs exhibit a balance of potency AND favorable drug - like properties x ABI - H0731: • Favorable
Slide 26: Microbiome Program
Microbiome Program 26
Slide 27: The Microbiome: Realizing the Promise
27 The Microbiome: Realizing the Promise Infectious diseases GI disorders Oncology Metabolic diseases Neurosciences/CNS POC data has demonstrated that fecal microbiota transplants (FMT) can restore dysbiotic microbiome to health But, FMT is inadequate as a therapy: lack of consistency, not scalable,
Slide 28: The Microbiome: Challenges
28 The Microbiome: Challenges Lack of consistency, not scalable, not controlled, unreliable, poor route of administration Diversity is needed for optimal microbiotic therapeutics Unique microhabitats require targeted delivery • pH • Oxygen • Antimicrobials • Nutrients Strain selection Manufacturing
Slide 29: Fully-Integrated Platform to Deliver Synthetic Live Biotherapeutics
Our differentiated and fully - integrated platform to deliver synthetic live biotherapeutics (LBT) 29 ASMB Solution: Proprietary Microbiome Platform Strain Selection cGMP Manufacturing Targeted Drug Delivery Rapid Clinical Development Proprietary & scientifically rigorous, rational strain selection
Slide 30: GEMICEL: Proprietary Targeted Delivery Technology
The colon is an attractive site for drug delivery, particularly for microbiotic therapies if they can be delivered reliably. GEMICEL ® : ASMB’s proprietary targeted delivery technology • GRAS ingredients • Manufacturing amenable to biologic products GEMICEL® Capsule First/outer capsule Second/inner
Slide 31: Assembly and Allergan Enter Microbiome Collaboration
Significant microbiome platform collaboration with focus on GI disorders 31 Assembly and Allergan Enter Microbiome Collaboration Expertise in microbiome therapeutics, fully - integrated platform Expertise in gastrointestinal drug development and commercialization Rights for GI development programs •
Slide 32: Summary: AGN/ASMB Microbiome Collaboration
Summary: AGN/ASMB Microbiome Collaboration • Expedites our efforts into multiple GI indications • Leverages our end - to - end microbiome technology platform • Advances our ability to move microbiome candidates rapidly into clinical development with strategic partners • Aligns with our strategic goa
Slide 33: Capturing the Potential of the Human Microbiome
Capturing the potential of the human microbiome for development of novel therapeutics 33 Microbiome Platform Gastrointestinal Oncology • Immuno - oncology • Colorectal cancer Metabolic Disease • Obesity • Type 2 Diabetes Liver • NASH Leveraging our Microbiome Platform to Expand to Other High - ratio
Slide 34: Company Summary
Company Summary 34
Slide 35: ASMB Anticipated Milestones and Financial Summary
35 ASMB Anticipated Milestones and Financial Summary 2017 2018 x Up to $2.8B m icrobiome collaboration for GI indications x ABI - H0731 dose ranging Ph 1a portion complete x Initiated ABI - H0731 Ph 1b/2a trial x AASLD: 3 abstracts accepted x ABI - H2158 selected as 2 nd CpAM clinical candidate □ H2
Slide 36: Assembly Biosciences: Summary
Clinical stage company focused on two key INNOVATIVE areas of drug development 36 Assembly Biosciences: Summary Delivering oral, synthetic live biotherapeutics to targeted location Allergan partnership for GI assets, rest of pipeline wholly owned HBV Deep pipeline of CpAMs with potential to achieve
Slide 37: Thank You
Thank You 37
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