Assembly Biosciences, Inc. · NASDAQ:ASMB
Assembly Biosciences — RBC Capital Markets 2016 Healthcare Conference
22 slides from Assembly Biosciences's conference presentation, Feb 24, 2016
Slide 1: Assembly Biosciences RBC Capital Markets 2016 Healthcare Conference
Assembly Biosciences Nasdaq: ASMB February 2016 RBC Capital Markets 2016 Healthcare Conference
Slide 2: Cautionary Note Regarding Forward-Looking Statements
C autionary 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 cl
Slide 3: Investment Highlights
Investment Highlights An integrated i nfectious disease company Focus on infectious diseases Developing best - in - class small molecules for the treatment of chronic hepatitis B (HBV) Platform for developing drug - like oral microbiotic (MB) therapeutics World class team Proven leadership tea
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 President & CEO Richard Colonno, PhD Chief Science Officer Uri Lopatin , MD CMO & VP R&D Thomas Rollins Chief Development Officer Head of Microbiome Lee Arnold
Slide 5: HBV-Cure Program
Va an eer _ ——\ (A) HBV-Cure Program ZZ ASSSmme ly biosciences
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 s FDA ); > 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 signif
Slide 7: 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 i n the generation and maintenance of cccDNA – R equired for c
Slide 8: 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 9: Lead Candidate: AB-V101 to Prevent cccDNA
Lead Candidate: AB - 101 to Prevent cccDNA 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 • Reduces rcDNA delivery to nucleus – Prevents s
Slide 10: ASMB Hepatitis B Pipeline
ASMB Hepatitis B Pipeline 2016 2017 Hepatitis B program AB - V101 ( CpAM 1 st Generation) AB - V102 ( CpAM 2 nd Generation) AB - V201 (upstream) AB - V202 ( upstream) AB - V301 (novel target) Phase 1a/1b IND enabling s tudies Phase 1a/1b IND enabling studies Lead Optimization IND enabling studies Le
Slide 11: Microbiome Program
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Slide 12: Our Microbiome Program Vision
Our Microbiome Program Vision Strain Selection Development & Manufacturing Targeted Drug Delivery 12 Best - in - class microbiome program with a fully - integrated platform to deliver drug products across multiple indications and therapeutic categories
Slide 13: The ASMB Approach: 'Druggable' Microbes
The ASMB A pproach: ‘ Druggable ’ Microbes Problem: FMT is a procedure not a product Solution: Replace FMT with oral microbiotic therapy comprised of GMP mono - cultured bacteria Strain Selection Development & Manufacturing Targeted Drug Delivery Proprietary & scientifically rigorous strain s el
Slide 14: Gemicel: Providing Direct Delivery to Colon
Gemicel ™: Providing Direct Delivery to Colon Our proprietary targeted delivery technology enables direct treatment in the colon Controlled release p latform Targeted d elivery Second inner c oated for bolus release of drug A in right colon First outer c oated for bolus release of drug A in ileum •
Slide 15: Human Scintigraphy Study: Gemicel POC
Human Sc inti g r aphy Study: Gemicel TM POC ASMB presented the bolus release demonstrated in ileum and colon in Jan 2016 P atient Representative data: Sm - 153 • Presented at the 5th Drug Formulation, Solubility & Bioavailability Summit, January 25 - 27, 2016 15 Figure: images of human Gemicel ™ ga
Slide 16: ASMB Strain Selection: Any Relevant Phyla
ASMB Strain Selection: Any R elevant Phyla Non - Spore F ormers Spore Formers SILVA http://www.arb - silva.de/ rRNA gene database Broad capabilities provide more tools as options for complex diseases 16 Our d iscovery and manufacturing engine , combined with Gemicel ™ allows ASMB to use multiple phy
Slide 17: Potential Microbiome Opportunities
Strain Selection Development & Manufacturing Targeted Drug Delivery Potential Microbiome Opportunities Infectious Disease • CDI (FIRST INDICATION) • Antibiotic resistant organisms Gastro • IBD • Crohn's • Ulcerative Colitis • IBS Metabolic Disease • Obesity • Diabetes CNS • Psychiatric • Neurodegene
Slide 18: C. Difficile: #1 Hospital Acquired Infection in US
Dubberke 2012 Ghantoii 2010 National Action Plan for Combating R esistant B acteria: March 2015 Leffler DA, Lamont JT. N Engl J Med 2015;372:1539 - 1548 C. Difficile: #1 Hospital Acquired Infection in US Most common nosocomial infection in U.S D eaths annually E conomic impact in US alone C ost per
Slide 19: AB-M101 Oral Capsules for Recurrent CDI
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 therapy including both spore and non - spore forming bacteria, delivered specifically to the lower GI tract, can be b
Slide 20: ASMB Microbiome Pipeline
ASMB Microbiome Pipeline Lead program for recurrent rCDA projected to enter Phase 1 b in 2H 2016 2016 2017 Microbiome Program AB - M101 (Recurrent C.Diff ) AB - M102 (new indication) AB - M103 (new indication) Gemicel TM (oral delivery) Strain Library & Inventory IND enabling Phase 1b Phase 2/3 IND
Slide 21: Financial Summary
Financial Summary 21 Strong Base of Biotech Investors Baker Brothers Nasdaq ASMB Cash, cash equivalents & marketable securities ~$97M as of September 30, 2015 Shares outstanding ~17.2M Fully diluted ~20.4M
Slide 22: Investment Summary
Investment Summary Experienced team with proven track record HBV platform : D eveloping oral small molecules for HBV cure – L ead product anticipated to begin Phase 1 in 2H 2016 – O ther molecules to follow – Primary focus on modulating HBV Core Protein, provides an opportunity to: • Prevent new
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