Theriva Biologics, Inc. · AMEX:TOVX
Theriva Biologics — Synthetic Biologics — 2017 Biotech Showcase
30 slides from Theriva Biologics's conference presentation, Jan 9, 2017
Slide 1: 2017 Biotech Showcase
2017 Biotech Showcase Investor Presentation January 9, 2017
Slide 2: Forward-Looking Statements
Forward - Looking Statements This presentation includes forward - looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 , as amended, on Synthetic Biologics’ current expectations and projections about future events . In some cases forward - looking statements
Slide 3: Management Team
Management Team • Jeffrey Riley, CEO Pfizer, Nichols Institute (Quest), SmithKline Beecham, QIC • Steven Shallcross , CFO Vanda Pharmaceuticals, Inc., Empire Petroleum Partners, LLC, Innocoll AG (formerly privately held Innocoll Holdings, Inc.) • Joseph Sliman , MD, MPH, SVP Clinical/Regulatory Vand
Slide 4: Company Strategy
Company Strategy Pioneering and developing therapeutics focused on protecting the gut microbiome Core Advancing two lead microbiome - focused drugs with billion - dollar market potential poised for pivotal trials Future Growth Robust pipeline of preclinical therapeutic programs designed to enhance p
Slide 5: Product Pipeline
Product Pipeline C - Cedars - Sinai Medical Center collaboration I - Intrexon Corporation collaboration T - The University of Texas at Austin collaboration Therapeutic Area / Product Candidate Discovery Preclinical Phase 1 Phase 2 Phase 2b/3 IBS - C SYN - 010 C Oral modified - release lovastatin lac
Slide 6: Recent Highlights
Recent Highlights 6 SYN - 010 : IBS - C Milestone Phase 2 (1st study; acute, placebo - controlled) √ 4Q 2015 – Reported Supportive Phase 2 topline data Phase 2 (2nd study; extension, SYN - 010 42mg) √ 1Q 2016 – Reported Supportive Phase 2 topline data Completed end of Phase 2 Meeting with FDA √ Summ
Slide 7: The Human Microbiome
Source: http://commonfund.nih.gov/hmp/overview.aspx US National Library of Medicine. Image source: Ottman N, et al. (2012) The function of our microbiota: who is out there and what do they do? Front. Cell. Inf. Microbio . 2:104. Human Microbiome > 1,000,000 Genes Human Genome 23,000 Genes 99% of Gen
Slide 8: The Human Microbiome
Source: http://commonfund.nih.gov/hmp/overview.aspx US National Library of Medicine. Image source: Ottman N, et al. (2012) The function of our microbiota: who is out there and what do they do? Front. Cell. Inf. Microbio . 2:104. The Human Microbiome Helps digest food, keep the immune system operatin
Slide 9: Diseases Directly Influenced by the Gut Microbiome
Source: Belizário JE and Napolitano M (2015) Human microbiomes and their roles in dysbiosis , common diseases, and novel therapeutic approaches. Front. Microbiol . 6:1050. Diseases Directly Influenced by the Gut Microbiome Microbial communities provide ample therapeutic opportunities 9 Vagina Microb
Slide 10: SYN-004 (ribaxamase): Prevention of C. difficile Infection, AAD, and AMR
SYN - 004 (ribaxamase) Prevention of C. difficile Infection, AAD, and Antimicrobial Resistance (AMR)
Slide 11: C. difficile Infection (CDI): a Growing Epidemic
C. difficile Infection (CDI): a Growing Epidemic Support from CDC, Department of Health & Human Services and CMC to reduce CDI 11 #1 Hospital - acquired infection in the U.S. 453,000 Patients in the U.S. infected with C. difficile annually 1 4 ̶ 7 Extra days spent in hospital by CDI patients 2 25% P
Slide 12: Antibiotic Resistance: Public Policy Tailwind
Antibiotic Resistance: Public Policy Tailwind CDC BAA – 2016 • Microbiome assessment and intervention to address antibiotic - mediated antimicrobial resistance (AMR) SYN awarded CDC contract to evaluate ability of ribaxamase to prevent AMR WHO OSTP National Microbiome Initiative - 2016 • Integrated
Slide 13: SYN-004: A potential paradigm shift through prevention and protection
Gaming Displays & Accessories Services Core PC Business SYN - 004: A potential paradigm shift through prevention and protection of a diverse gut microbiome Ribaxamase Paradigm Current Paradigm Antibiotics • IV β - lactam antibiotic * AND • SYN - 004 (ribaxamase) Good microbes that protect against in
Slide 14: Piglet Model of Antibiotic-Mediated Dysbiosis
Feces collections Readouts: • Fecal DNA shotgun deep sequencing metagenomics analyses • Antibiotic blood levels Oral ribaxamase (75 mg QID) Days: - 7 - 4 IV Ceftriaxone (50 mg/kg, SID) 0 8 Piglet Model of Antibiotic - Mediated Dysbiosis 2 month old 20 kg piglets N=5 per cohort 7 4 1 2 Blood collecti
Slide 15: Ribaxamase Reduced Propagation of Antibiotic-Resistance Genes
Ribaxamase Reduced Propagation of Antibiotic - Resistance Genes Ceftriaxone (CRO) increased the frequency of genes conferring resistance to multiple classes of antibiotics, not just beta - lactams Ribaxamase mitigated this increase
Slide 16: SYN-004: Designed to Protect the Microbiome from Antibiotic Damage
SYN - 004: Designed to Protect the Microbiome from Antibiotic Damage Ribaxamase prevented ceftriaxone - mediated changes in pig fecal microflora 16 Heat map shows log relative abundance of microbial genes in pig feces. Each square represents a microbial species present in an individual microbiome. D
Slide 17: SYN-004 (ribaxamase): Phase 2b Proof of Concept Trial
SYN - 004 (ribaxamase): Phase 2b Proof of Concept Trial 84 Global Clinical Sites Ceftriaxone + Ribaxamase Ceftriaxone + Placebo Primary Endpoint: • Prevention of C. difficile associated diarrhea (CDAD) Secondary Endpoint: • Prevention of non - C. difficile AAD Exploratory Endpoints: • Evaluate abili
Slide 18: SYN-004: Phase 2b Proof of Concept Trial Study Design
SYN - 004: Phase 2b Proof of Concept Trial Study Design Fecal microbiome and fecal colonization samples taken 4 weeks Randomized 1:1, 150mg ribaxamase or placebo US Canada Romania Bulgaria Hungary Poland Serbia Diarrhea = 3 or more loose or watery stools in a 24 hour period, samples collected CDI =
Slide 19: SYN-004 (ribaxamase): Phase 2b Topline Results
New VRE Colonization Data consistent with MoA of protecting the gut microbiome from complications of antibiotic therapy SYN - 004 (ribaxamase): Phase 2b Topline Results P= 0.0002 • Achieved primary endpoint of statistically significant (P=0.045) reduction of CDI • 71.4% relative risk reduction in CD
Slide 20: SYN-004 (ribaxamase): Strong Patent Position
SYN - 004 (ribaxamase): Strong Patent Position 20 ~ 60 U.S. & Foreign Granted Patents ~25 U.S. & Foreign Pending Patents Patented ribaxamase Composition of Matter Expires 2031 Patented other beta - lactamase Composition of Matter & Uses Expires 2035 Pending Patent Applications • Methods of Manufactu
Slide 21: SYN-004 (ribaxamase): Market Potential
SYN - 004 (ribaxamase): Market Potential *Estimate based on the following assumptions: Cost of ribaxamase/day of $100 x 117M days of “ribaxamase target” β - lactam antibiotics in 2014 1 Arlington Medical Resources (AMR), a Decision Resources Group Company, 2014 Audits of Acute Care Hospital Antibiot
Slide 22: SYN-010 Treatment of IBS-C
SYN - 010 Treatment of IBS - C
Slide 23: Irritable Bowel Syndrome with Constipation (IBS-C)
Irritable Bowel Syndrome with Constipation (IBS - C) 23 Data confirmed that IBS is a long - term condition, with 1/3 of IBS sufferers having experienced IBS symptoms for over 10 years An estimated 10 - 15% of the population suffers from IBS IBS is a prevalent disorder that significantly impacts work
Slide 24: The Role of Methane in the Gut Microbiome
The Role of Methane in the Gut Microbiome SYN - 010: a proprietary modified - release formulation of lovastatin lactone 24 Bacteroides thetaiotaomicron is one such bacterium Carbohydrates Methanobrevibacter smithii archea consumes hydrogen gas from Bacteroides and produces methane, which is lost fro
Slide 25: SYN-010: Key Differentiators
SYN - 010: Key Differentiators No serious adverse events reported in clinical trials SYN - 010 is a proprietary, modified - release formulation of lovastatin lactone that is optimal for reducing methane - production by certain microorganisms ( M. smithii ) in the gut 25 Intended to minimize disrupti
Slide 26: SYN-010 Phase 2: Twelve Week Evaluation in IBS-C Patients
SYN - 010 Phase 2: Twelve Week Evaluation in IBS - C Patients SYN - 010 reduced breath methane and improved IBS - C symptoms 1 - Least - squares linear regression modeling 2 - Paired t - test vs Baseline (Day 1). A Monthly Responder is defined as a subject who has a weekly response in at least 50% o
Slide 27: IBS-C Market Overview
Product Candidate SYN - 010 * Linzess Amitiza OTC Laxatives Plecanatide Tenapanor Company Synthetic Allergan Takeda Various Synergy Ardelyx Phase/Status Planned pivotal 2b/3 Marketed Marketed Marketed Phase 3 Phase 3 Designed to treat underlying cause of IBS - C Designed to treat symptoms w/o causin
Slide 28: SYN-010's Strategic Patent Position
SYN - 010’s Strategic Patent Position 28 ~60 U.S. & Foreign Granted Patents ~20 U.S. & Foreign Pending Patents Patented Methods of Treatment (Broadest Indication) Expires 2023 Patented and Pending Methods of Treating Constipation with SYN - 010 in Screened Patients Expires 2034 Pending Patent Applic
Slide 29: Anticipated SYN-010 Phase 3 Pivotal Trials Design
Once daily, oral dosing Anticipated SYN - 010 Phase Pivotal Trials Design A randomized, 1:1:1, double - blind, placebo - controlled clinical study *An overall (12 - week) responder is defined as a subject who has a weekly response in at least 50% of the weeks in the 12 - weeks t reatment period. Pri
Slide 30: 2017 Biotech Showcase
2017 Biotech Showcase Investor Presentation January 9, 2017 SYN – January 7, 2017 Draft V4 VP