Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — Investor Day (June 2018)
66 slides from Assembly Biosciences's investor day presentation, Jun 20, 2018
Slide 1: R&D Day Hepatitis B Program
1 R&D Day Hepatitis B Program June 20, 2018 NASDAQ: ASMB 2018 ©Assembly Biosciences, Inc.
Slide 2: Cautionary Note Regarding Forward-Looking Statements
2 2 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 program, the timing of the initiation
Slide 3: Assembly Biosciences Overview
3 3 ASSEMBLY BIOSCIENCES OVERVIEW HBV = hepatitis B virus. No cure for the > 250 million patients with chronic HBV Core inhibitors designed to break the life cycle of HBV The gut microbiome is essential to human health, yet there are no approved microbiome therapies Targeted delivery of oral, synthe
Slide 4: Development Programs Focused on Large Patient Populations With High Unmet Need
4 4 DEVELOPMENT PROGRAMS FOCUSED ON LARGE PATIENT POPULATIONS WITH HIGH UNMET NEED BLA = Biologic License Application; IND = Investigational New Drug Application; NASH = nonalcoholic steatohepatitis; NDA = Ne w D rug Application. Drug Candidate (Mechanism/Indication) Discovery & Lead Op/Selection IN
Slide 5: Significant Progress With Exciting New Developments
5 5 SIGNIFICANT PROGRESS WITH EXCITING NEW DEVELOPMENTS • Positive Phase 1b data presented at EASL on ABI - H0731 • These data and some of our preclinical data showing that Core Inhibitors are effective antivirals with distinct mechanisms from standard of care nucleoside analogs • On track to initia
Slide 6: Upcoming HBV Milestones
6 6 UPCOMING HBV MILESTONES EASL = European Association for the Study of the Liver; POC = proof of concept. INNOVATION ABI - H0731 2019 HBV 2018 ABI - H2158 Phase 2a (Cure POC) Phase 1b Initial POC data expected 1H19 Positive phase 1b data reported at EASL Phase 2a (Antiviral) Phase 1a Phase 1b 3 rd
Slide 7: HBV Disease Progression
7 7 HBV DISEASE PROGRESSION HCC = hepatocellular carcinoma. Source: World Health Organization; Hepatitis B Foundation. 20% - 30% Surgery, chemotherapy, and liver transplant Cirrhosis/HCC ~ 1 million people/year 2 people/ minute Death >250 million chronically infected worldwide 8% diagnosed <1% recei
Slide 8: More HBV Patients Than HCV Patients in the Major Geographies
8 8 MORE HBV PATIENTS THAN HCV PATIENTS IN THE MAJOR GEOGRAPHIES *US, Europe, South Korea, Japan, China. HCV = hepatitis C virus. WHO and ECDC. Patients with HCV 0 1M 2M 5M Prevalence 100 million * 23 million * Patients with HBV 0 50M 75M 100M 25M China 90M 15M 3M 4M South Korea 2.1M 0.2M Japan 1.3M
Slide 9: Assembly China Established to Rapidly Develop Programs in China
9 9 ASSEMBLY CHINA ESTABLISHED TO RAPIDLY DEVELOP PROGRAMS IN CHINA Beijing Shanghai 2018 ©Assembly Biosciences, Inc. HBV is a public health epidemic in China – the government has made HBV one of their highest public health priorities • China has made significant advances in regulatory, IP and busin
Slide 10: Developing a Potential Cure for Hepatitis B
10 10 DEVELOPING A POTENTIAL CURE FOR HEPATITIS B Assembly Biosciences has a deep pipeline of potent core inhibitors Novel mechanism designed to break the HBV life cycle We believe backbone of curative therapy Cure is achievable but currently at very low rates Core Inhibitors 2018 ©Assembly Bioscien
Slide 11: HBV Cure: Clinical Components
11 11 Expected Treatment Components to Achieve Cure HBV CURE: CLINICAL COMPONENTS 2018 ©Assembly Biosciences, Inc. Elimination of Viral Load and Inhibition of cccDNA Formation Decay of Existing cccDNA Pools and/or Infected Cells Treatment Continuation No Relapse Off Therapy Significant decreases in
Slide 12: Thought We Had a Potential Cure Over Years Ago
12 12 THOUGHT WE HAD A POTENTIAL CURE OVER 10 YEARS AGO 2018 ©Assembly Biosciences, Inc.
Slide 13: ETV Monotherapy Cured Chronically Infected Woodchucks
13 13 Unfortunately, this is not what happens in HBV patients, despite prolonged therapy ETV MONOTHERAPY CURED CHRONICALLY INFECTED WOODCHUCKS CLEAR EVIDENCE OF CURE WITH ETV (0.5 mg/kg) daily/weekly ETV = entecavir; HBcAg = hepatitis B core antigen; WHBsAg = woodchuck hepatitis B surface antigen; W
Slide 14: So What Happened in Patients? Virus Not Fully Suppressed
14 14 SO WHAT HAPPENED IN PATIENTS? VIRUS NOT FULLY SUPPRESSED 2018 ©Assembly Biosciences, Inc.
Slide 15: SOC Nucs Are Potent Antivirals and Reduce Viral Load
15 15 Patients Treated With Entecavir (%) HBeAg+ (n=354) HBeAg - (n=325) 0 20 40 60 80 100 HBV DNA undetectable 90 67 21 78 ALT normalization 68 2.0 0.3 HBsAg loss Viral Markers at 48 Weeks SOC NUCS ARE POTENT ANTIVIRALS AND REDUCE VIRAL LOAD ALT = alanine aminotransferase; HBeAg = hepatitis B e ant
Slide 16: But They Fail to Fully Suppress Viral Replication
16 16 …BUT THEY FAIL TO FULLY SUPPRESS VIRAL REPLICATION ADV = adefovir dipivoxil ; IFN = interferon; LVD = ledipasvir; TDF = tenofovir disoproxil fumarate. 1. Marcellin P, et al. AASLD Poster 1861, 2014. 2. Huang, Q, et al. collaborative study in progress. 2018. Treatment Years 0 10 20 30 40 50 60
Slide 17: Lack of Full Viral Suppression May Be Reason for Low HBV Cure Rates
17 17 HCV = hepatitis C virus; LLQ = lower limit of quantification. LACK OF FULL VIRAL SUPPRESION MAY BE REASON FOR LOW HBV CURE RATES 12 Weeks HCV Viral Load Time on Treatment LLQ ~ 100% cure rates HCV To improve cure rates…must eliminate residual virus Years HBV Viral Load Time on Treatment LLQ <1
Slide 18: How Can Therapy Be Improved?
18 18 HOW CAN THERAPY BE IMPROVED? 2018 ©Assembly Biosciences, Inc.
Slide 19: Critical Elements of HBV Life Cycle
19 19 CRITICAL ELEMENTS OF HBV LIFE CYCLE 2018 ©Assembly Biosciences, Inc. Conversion to cccDNA Covalently Closed Circular DNA (cccDNA) pgRNA packaging into nucleocapsid Core Relaxed Circular DNA (rcDNA) Polymerase maturation & release of virus Core and polymerase proteins play critical roles in HBV
Slide 20: Nucs Reduce Virus Levels but Fail to Prevent cccDNA Establishment
20 20 NUCS REDUCE VIRUS LEVELS BUT FAIL TO PREVENT cccDNA ESTABLISHMENT 2018 ©Assembly Biosciences, Inc. Covalently Closed Circular DNA (cccDNA) Core Relaxed Circular DNA (rcDNA) Polymerase maturation & release of virus Pregenomic RNA (pgRNA) pgRNA conversion into rcDNA Polymerase inhibition • Preve
Slide 21: Core Inhibitors Block Viral Replication and cccDNA Establishment
21 21 CORE INHIBITORS BLOCK VIRAL REPLICATION AND cccDNA ESTABLISHMENT 2018 ©Assembly Biosciences, Inc. Conversion to cccDNA Covalently Closed Circular DNA (cccDNA) pgRNA packaging into nucleocapsid Core Relaxed Circular DNA (rcDNA) Polymerase Pregenomic RNA (pgRNA) trafficking to nucleus Core inhib
Slide 22: Core Inhibitors Block Formation of Functional Nucleocapsids
22 22 CORE INHIBITORS BLOCK FORMATION OF FUNCTIONAL NUCLEOCAPSIDS 2018 ©Assembly Biosciences, Inc. pgRNA packaging into nucleocapsid Addition of core inhibitor causes formation of aberrant capsids that are larger, cracked, and asymmetrical Control + Core inhibitor
Slide 23: Core Inhibitors Block Encapsidation of pgRNA
23 23 CORE INHIBITORS BLOCK ENCAPSIDATION OF pgRNA 2018 ©Assembly Biosciences, Inc. Huang Q, et al. AASLD Poster 922 2017. pgRNA packaging into nucleocapsid pgRNA DNA - 50 100 150 DMSO ETV (17x EC 50 ) Core inhibitor (10x EC 50 ) Nucs prevent synthesis of DNA from pgRNA in nucleocapsids Core inhibit
Slide 24: Core Inhibitors Cause Premature Melting of Nucleocapsids
24 24 CORE INHIBITORS CAUSE PREMATURE MELTING OF NUCLEOCAPSIDS 2018 ©Assembly Biosciences, Inc. 1. Cai, D, et al. Intl HBV Mtg Poster P - 140. 2017; 2. Huang Q, et al. AASLD Poster 922 2017. Conversion to cccDNA pgRNA packaging into nucleocapsid trafficking to nucleus Southern Blot of HBV DNA From H
Slide 25: Core Inhibitors Block Generation of cccDNA
25 25 CORE INHIBITORS BLOCK GENERATION OF cccDNA 2018 ©Assembly Biosciences, Inc. Conversion to cccDNA trafficking to nucleus Core Inhibitors Block cccDNA Generation (Southern Blot) Core inhibitors prevent cccDNA generation and formation Core (2158) Control Nuc (ETV) cccDNA ETV >10,000x EC 50 2158 5
Slide 26: Core Inhibitors Reduce Key Surrogate Markers for cccDNA
26 26 CORE INHIBITORS REDUCE KEY SURROGATE MARKERS FOR cccDNA 60 0 20 40 80 100 120 140 0.001 0.1 10 0.01 1 60 0 20 40 80 100 120 140 Concentration (µM) Control (%) Nuc (ETV) Treatment 3 Hours After Infection Concentration (µM) Control (%) 0.001 0.1 10 0.01 1 Core Inhibitor (2158) Treatment 3 Hours
Slide 27: Bottom Line: Core Inhibitors Are More Effective Antivirals
27 27 BOTTOM LINE…CORE INHIBITORS ARE MORE EFFECTIVE ANTIVIRALS 0.1 1 10 100 1000 0 25 50 75 100 HepAD38 Intracellular Viral Load Multiple of EC 50 Percentage of Control (Viral DNA) Nuc (ETV) Core Inhibitor (2158) Nuc+Core Inhibitor (ETV+2158) Cell Background Level 0.1 1 10 100 1000 10 2 HepAD38 Int
Slide 28: How Quickly Do cccDNA and Infected Cells Turn Over?
28 28 HOW QUICKLY DO cccDNA AND INFECTED CELLS TURN OVER? 2018 ©Assembly Biosciences, Inc.
Slide 29: cccDNA Biosynthesis Study
29 29 Strategy/Approach Resistance emerges rapidly in lamivudine - and telbivudine - treated HBV patients Obtain longitudinal clinical samples (paired plasma and biopsy) Establish and validate isolation methodologies Follow resistance signature mutations as genetic markers of cccDNA turnover, confir
Slide 30: Rapid Turnover of cccDNA in as Little as 12-16 Weeks
30 30 Virus, pgRNA and cccDNA populations can be completely replaced in as little as 12 weeks RAPID TURNOVER OF cccDNA IN AS LITTLE AS 12 - 16 WEEKS IFN = interferon; TBV = telbivudine. Huang, et al. AASLD Poster 1503 2017. IFN Treatment IFN Study ML18376 (Patient 50) Week 48 0 12 24 HBV DNA 100% HB
Slide 31: Summary Results From Ongoing cccDNA Biosynthesis Study
31 31 SUMMARY RESULTS FROM ONGOING cccDNA BIOSYNTHESIS STUDY Huang, et al. AASLD Poster 1503. 2017. 3. No evidence of an inactive subpopulation of cccDNA, as resistance mutations were observed in the entire population of cccDNA 1. Genetic source of resistance shown to be cccDNA cccDNA pgRNA virus 2.
Slide 32: Pipeline of Potent Core Inhibitors
32 32 PIPELINE OF POTENT CORE INHIBITORS 2018 ©Assembly Biosciences, Inc.
Slide 33: ASMB HBV Core Inhibitor Program Portfolio
33 33 ASMB HBV CORE INHIBITOR PROGRAM PORTFOLIO Novel Molecules With Distinct Chemical Scaffolds Discovered at Assembly Biosciences BLA = Biologic License Application; IND = Investigational New Drug Application; NDA = New Drug Application. Drug Candidate Discovery Lead Op/ Selection IND Enabling Pha
Slide 34: Lead Candidate From a Pipeline of Unique Core Inhibitors
34 34 ABI - H0731 IS THE LEAD CANDIDATE FROM A PIPELINE OF UNIQUE CORE INHIBITORS Proprietary chemical scaffold, distinct from other core inhibitors in development QD dosing in patients Excellent hepatocyte stability , minimal metabolism Clean safety profile in animal studies Potent and selective ac
Slide 35: Phase 1 Study Designs
35 35 400 mg (n=2) Follow up Treatment (28 days) Off Treatment (28 days) ABI - H0731 PHASE 1 STUDY DESIGNS PK = pharmacokinetics; PO = by mouth. Yuen, et al. EASL Poster LBP - 012 2018. Time (days) Treatment (14 days) Off Treatment (7 days) 100 mg PO QD (n=12) Follow up 200 mg PO QD (n=12) Follow up
Slide 36: Phase 1 Clinical Safety Summary
36 36 ABI - H0731 PHASE 1 – CLINICAL SAFETY SUMMARY AE = adverse event; SAE = serious adverse event; TEAE = treatment - emergent adverse event. Interim Data - Yuen, et al. EASL Poster LBP - 012 2018. Dose (n) 100 mg (20) 200 mg ( 20) 300 mg ( 20) 400 mg (2) Placebo (12) Grade 1 AEs 24 15 12 4 9 Grad
Slide 37: Phase 1b Study: HBV DNA Levels in Treated HBV Patients
37 37 PHASE 1 STUDY – HBV DNA LEVELS IN TREATED HBV PATIENTS Yuen, et al. EASL Poster LBP - 012 2018 . 300 - mg Cohort 1.0E+00 1.0E+01 1.0E+02 1.0E+03 1.0E+04 1.0E+05 1.0E+06 1.0E+07 1.0E+08 1.0E+09 0 5 10 15 20 25 30 Treatment (days) HBV DNA Level (IU/mL) HBeAg Neg patients HBeAg Pos patients • Ste
Slide 38: 731 Is as Potent as Any Therapy for HBV
38 38 0 0.5 1 1.5 2 2.5 3 Nucs QD Phase 1b 28 - Day Monotherapy Studies in HBV - Infected, HBeAg Positive Patients 731 IS AS POTENT AS ANY THERAPY FOR HBV *Estimated. BID = twice a day; POC = proof of concept; QD = once a day; RTV = ritonavir. 1. Yuen, et al. AASLD Poster LB - 10 2015. 2. Ding, et a
Slide 39: ABI-H2158: Second-Generation Core Inhibitor Advancing to Phase 1 Studies
39 39 ABI - H2158 – SECOND - GENERATION CORE INHIBITOR ADVANCING TO PHASE 1 STUDIES Chemical scaffold is distinct from ABI - H0731 and other c ore i nhibitor s in development Favorable drug - like properties, with enhanced PK in animals Excellent hepatocyte stability , minimal metabolism Enhanced po
Slide 40: Enhanced Properties of ABI-H2158
40 40 ENHANCED PROPERTIES OF ABI - H2158 AUC inf = area under the curve from zero to infinity; C max = maximum concentration. Animal Species ABI - H0731 ABI - H2158 ABI - H0731 ABI - H2158 C max (µg/mL) C max (µg/mL) AUC inf (µg* hr /mL) AUC inf (µg*hr/mL) Rat 6.4 24.8 62.1 445 Monkey 3.2 45.4 45.2
Slide 41: HBV Cure: Clinical Components
41 41 Expected Treatment Components to Achieve Cure HBV CURE: CLINICAL COMPONENTS 2018 ©Assembly Biosciences, Inc. Elimination of Viral Load and Inhibition of cccDNA Formation Decay of Existing cccDNA Pools and/or Infected Cells Treatment Continuation No Relapse Off Therapy Significant decreases in
Slide 42: ASMB Core Inhibitor Program Summary
42 42 ASMB CORE INHIBITOR PROGRAM SUMMARY ABI - H0731 First - Generation Candidate ABI - H2158 Second - Generation Candidate • Exhibits enhanced potency and PK properties, while retaining favorable drug - like properties • Completing IND - enabling studies • Phase 1a clinical trial expected to initi
Slide 43: Core Inhibitor Roadmap for Functional Cure
43 43 ABI - H0731 WILL BE AMONG THE FIRST CORE INHIBITORS EXPLORED IN AN HBV CURE PROGRAM 2018 ©Assembly Biosciences, Inc. 1) Elimination of Viral Load 2) Decay of cccDNA / Infected Cells 3) Treatment Consolidation 4) No Relapse Off Therapy CORE INHIBITOR ROADMAP FOR FUNCTIONAL CURE cccDNA = covalen
Slide 44: Core Inhibitor Roadmap for Functional Cure
44 44 ABI - H0731 WILL BE AMONG THE FIRST CORE INHIBITORS EXPLORED IN AN HBV CURE PROGRAM 2018 ©Assembly Biosciences, Inc. Phase 2a 300 mg 731 + 0.5 mg ETV Goal: Significant improvement in viral load reduction over ETV - only arm 1) Elimination of Viral Load 2) Decay of cccDNA / Infected Cells 3) Tr
Slide 45: Core Inhibitor Roadmap for Functional Cure
45 45 ABI - H0731 WILL BE AMONG THE FIRST CORE INHIBITORS EXPLORED IN AN HBV CURE PROGRAM 2018 ©Assembly Biosciences, Inc. Phase 2a Phase 2a 300 mg 731 + 0.5 mg ETV 300 mg 731 + nuc Goal: Significant improvement in viral load reduction over ETV - only arm Goal: Significant decreases in cccDNA surrog
Slide 46: Core Inhibitor Roadmap for Functional Cure
46 46 ABI - H0731 WILL BE AMONG THE FIRST CORE INHIBITORS EXPLORED IN AN HBV CURE PROGRAM 2018 ©Assembly Biosciences, Inc. Phase 2a Phase 2a 300 mg 731 + 0.5 mg ETV 300 mg 731 + nuc Continue 300 mg 731 + nuc Goal: Significant improvement in viral load reduction over ETV - only arm Goal: Significant
Slide 47: Core Inhibitor Roadmap for Functional Cure
47 47 ABI - H0731 WILL BE AMONG THE FIRST CORE INHIBITORS EXPLORED IN AN HBV CURE PROGRAM 2018 ©Assembly Biosciences, Inc. Phase 2a Phase 2a 300 mg 731 + 0.5 mg ETV 300 mg 731 + nuc Continue 300 mg 731 + nuc Off therapy Goal: Significant improvement in viral load reduction over ETV - only arm Goal:
Slide 48: Phase 2a Strategy and Designs
48 48 Viral Load Study Patient population: nuc - naive, HBeAg+ Continued nuc + 300 mg 731 Continued nuc + placebo ABI - H0731 PHASE 2A STRATEGY AND DESIGNS 2018 ©Assembly Biosciences, Inc. Time (months) 0.5 mg ETV + 300 mg 731 0.5 mg ETV + placebo Viral Antigen POC Study Patient population: nuc - su
Slide 49: Viral Load Study: Combo Therapy Inhibits HBV Life Cycle More Completely
49 49 Primary endpoint ABI - H0731 VIRAL LOAD STUDY: COMBO THERAPY INHIBITS HBV LIFE CYCLE MORE COMPLETELY 2018 ©Assembly Biosciences, Inc. 0.5 mg ETV + 300 mg 731 0.5 mg ETV + placebo Screening Patient population: v iremic HBeAg+ n=24 Randomization 1:1 Time (months) 0 9 Open - label extension Open
Slide 50: Viral Antigen POC Study: Combo Therapy Drives cccDNA Loss
50 50 ABI - H0731 VIRAL ANTIGEN POC STUDY: COMBO THERAPY DRIVES cccDNA LOSS 2018 ©Assembly Biosciences, Inc. Screening Patient population: nuc - suppressed HBeAg+ n=45 Randomization 3:2 SOC (nuc) + 300 mg 731 SOC (nuc) + placebo 9 Open - label extension Open - label extension Initial data expected H
Slide 51: Open-Label Extension Study Test of Cure
51 51 ABI - H0731 OPEN - LABEL EXTENSION STUDY TEST OF CURE! 2018 ©Assembly Biosciences, Inc. Elimination of Viral Load Decay of cccDNA / Infected Cells Trea tm ent Consolidation No Relapse Off Therapy Goal: CONSOLIDATION = Eliminate the last vestiges of replicating virus while continuing to block n
Slide 52: Open-Label Extension Study Test of Cure
52 52 ABI - H0731 OPEN - LABEL EXTENSION STUDY TEST OF CURE! 2018 ©Assembly Biosciences, Inc. Elimination of Viral Load Decay of cccDNA / Infected Cells Trea tm ent Consolidation No Relapse Off Therapy HBV Viral Load Time on Treatment LLQ Open - label 300 mg 731 + nuc Viral Antigen POC Study Viral L
Slide 53: Open-Label Extension Study Test of Cure
53 53 ABI - H0731 OPEN - LABEL EXTENSION STUDY TEST OF CURE! 2018 ©Assembly Biosciences, Inc. Elimination of Viral Load Decay of cccDNA / Infected Cells Trea tm ent Consolidation No Relapse Off Therapy HBV Viral Load Time on Treatment LLQ ↑ cure rates Monitor off therapy for 6 months Complete respon
Slide 54: Core Inhibitor Roadmap for Functional Cure: Initial Data Expected H1 2019
54 54 INITIAL DATA EXPECTED H1 2019 CORE INHIBITOR ROADMAP FOR FUNCTIONAL CURE 2018 ©Assembly Biosciences, Inc. Phase 2a Phase 2a Open - Label Extension Study 300 mg 731 + 0.5 mg ETV 300 mg 731 + nuc Continue 300 mg 731 + nuc Off therapy Goal: Significant improvement in viral load reduction over ETV
Slide 55: Expected Clinical Timelines and Milestones
55 55 EXPECTED CLINICAL TIMELINES AND MILESTONES 2018 ©Assembly Biosciences, Inc. 2019 2018 Phase 2a (cure POC) Phase 1b Initial POC data expected H1 2019 Phase 2a (antiviral) Phase 1a Phase 1b/2a ABI - H0731 ABI - H2158 Positive phase 1b data reported at EASL
Slide 56: Expected Clinical Timelines and Milestones
56 56 Open - label extension EXPECTED CLINICAL TIMELINES AND MILESTONES 2018 ©Assembly Biosciences, Inc. 2019 2018 Phase 2a (cure POC) Phase 1b Initial POC data expected H1 2019 Phase 2a (antiviral) Phase 1a Phase 1b/2a ABI - H0731 ABI - H2158 Positive phase 1b data reported at EASL
Slide 57: Expected Clinical Timelines and Milestones
57 57 EXPECTED CLINICAL TIMELINES AND MILESTONES 2018 ©Assembly Biosciences, Inc. ABI - H0731 Phase 2a (cure POC) Phase 1b Phase 2a (antiviral) 2019 2018 Phase 1a Phase 1b/2a ABI - H2158 Initial POC data expected H1 2019 Positive phase 1b data reported at EASL
Slide 58: Expected Clinical Timelines and Milestones
58 58 Open - label extension EXPECTED CLINICAL TIMELINES AND MILESTONES 2018 ©Assembly Biosciences, Inc. Phase 2a (cure POC) Phase 1b Phase 2a (antiviral) Phase 1a Phase 1b/2a Positive phase 1b data reported at EASL 2019 2018 ABI - H0731 ABI - H2158 Initial POC data expected H1 2019
Slide 59: Chronic Hepatitis B: Today
59 59 for chronic HBV: CHRONIC HEPATITIS B: TODAY HBV = hepatitis B virus . IQVIA. China US EU5 Japan Rest of EU South Korea $ 963 MILLION $ 621 MILLION $ 375 MILLION $ 237 MILLION $ 172 MILLION $ 157 MILLION SALES IN 2017 2018 ©Assembly Biosciences, Inc.
Slide 60: Chronic Hepatitis B: Tomorrow
60 60 CHRONIC HEPATITIS B: TOMORROW HCV = hepatitis C virus . Data on file. forecasted to grow dramatically WHY? The crucial role of improved cure rates The prevalence of chronic HBV • As cure rates improve, diagnoses and treatment rates expected to increase • The HCV experience as analog SALES IN H
Slide 61: The History of HCV Therapy
61 61 THE HISTORY OF HCV THERAPY 2018 ©Assembly Biosciences, Inc. DAA = direct acting antiviral; SVR = sustained viral response. Evaluate Ltd. 2017. 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 • In 2010, sales of the treatment for HCV (interferon and ribavirin) were $2.8 billion
Slide 62: The HCV Experience
62 62 Newly diagnosed Newly treated 0 50K 100K 150K 200K 250K People Improved cure rates in HCV: significantly increased diagnosis and treatment rates THE HCV EXPERIENCE... 2018 ©Assembly Biosciences, Inc. 1. Gilead Sciences, Inc. Q2 2017 earnings results. 2. Gilead Sciences, Inc. Q4 2016 earnings r
Slide 63: Lack of a Cure Has Limited the Uptake of Treatment
63 63 LACK OF A CURE HAS LIMITED THE UPTAKE OF TREATMENT 2018 ©Assembly Biosciences, Inc. CDC, ECDC, IMS, Evaluate Ltd. $54.7B 8.0M HCV $4.4B 10.0M HBV Sales, 2015 - 2017 Patients Although t here are more HBV patients than HCV patients in the developed countries, treatment has lagged
Slide 64: HBV Treatment Could Ultimately Look Similar
64 64 TBD 12 TREATMENT COULD ULTIMATELY LOOK SIMILAR 2018 ©Assembly Biosciences, Inc. Cure Tolerability Duration Pre - 2011 (pre - DAAs) HCV Today HBV ~50% Very poor 48 weeks Today ~100% Very good 12 weeks Goal Significantly better Very good Defined duration ~10% Very good Lifetime Cure Tolerability
Slide 65: HBV by the Numbers
65 65 HBV BY THE NUMBERS 2018 ©Assembly Biosciences, Inc. HCC = hepatocellular carcinoma ; nuc = nucleos (t)ide inhibitor. Data on file. $2.5 BILLION The market in 2017 for nucs used in HBV in the US, Europe, China, Japan, and Korea >250 million people with chronic HBV HBV is the cause of an enormou
Slide 66: Closing Remarks
66 2018 ©Assembly Biosciences, Inc. CLOSING REMARKS