Theriva Biologics, Inc. · AMEX:TOVX
Theriva Biologics — Synthetic Biologics — Investor Presentation (May 2015)
41 slides from Theriva Biologics's investor presentation, May 6, 2015
Slide 1: Synthetic Biologics
2 SYNTHETIC BIiouogics NYSE MKT: SYN Provoere 6
Slide 2: Forward-Looking Statements
Forward - Looking Statements This presentation includes forward - looking statements on Synthetic Biologics’ current expectations and projections about future events . In some cases forward - looking statements can be identified by terminology such as "may," "should," "potential," "continue," "expec
Slide 3: Investment Proposition
Investment Proposition • M icrobiome - focused , clinical - stage therapeutics to protect the microbiome while targeting pathogen - specific diseases • Addressing multi - billion dollar market opportunities addressing significant unmet medical needs • Multiple near - term and long - term clinical mi
Slide 4: Key Achievements
Mar 2015 C. difficile Positive PK data from Phase 1a and 1b clinical trials Feb 2015 IBS - C SYN - 010 modified - release formulation of Lovastatin Feb 2015 C. difficile Positive topline results from Phase 1b clinical trial Dec 2014 C. difficile Initiated Phase 1a and 1b clinical trials Dec 2014 C.
Slide 5: Upcoming Milestones
Upcoming Milestones 5 2H 2015 C. difficile Report topline data from Phase 2b clinical trial 2H 2015 IBS - C Report topline data from Phase 2 clinical trial 2Q 2015 IBS - C Initiate Phase 2 clinical trials 2Q 2015 C. difficile Initiate Phase 2b clinical trial 2Q 2015 C. difficile Report topline data
Slide 6: Product Pipeline
Product Pipeline C - Cedars - Sinai Medical Center collaboration I - Intrexon Corporation collaboration T - The University of Texas at Austin collaboration Completed Planned – 2015 6 Therapeutic Area Product Candidate Biologic Agent/ Drug Compound Discovery Preclinical Phase 1 Phase 2 Phase 3 C. dif
Slide 7: Human Microbiome
Human Microbiome The Body Has 10 Times as Many Microbe Cells as Human Cells 7 Source: http://commonfund.nih.gov/hmp/overview.aspx Human Microbiome > 1,000,000 Genes Human Genome 23,000 Genes 99% of your DNA Genes are in Microbe Cells (e.g., Bacteria) NOT Human Cells Leveraging the Microbiome Will Ra
Slide 8: Diseases Directly Influenced by the Gut Microbiome
Diseases Directly Influenced by the Gut Microbiome 8 Source: Genome Medicine 2011, 3 :14 http://genomemedicine.com/content/3/3/14
Slide 9: Human Microbiome Over Time
Human Microbiome Over Time Response to Environmental Conditions and Life Stages 9 Source: 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.
Slide 10: Collateral Damage Caused by Antibiotic Use
Collateral Damage Caused by Antibiotic Use 10 • Antibiotics ̶ Prevent/treat primary infections ̶ Carried to liver, transported to bile and excreted via large intestine ̶ May unintentionally upset natural balance of gut microbiome by killing off good bacteria • A microbial imbalance in the gut microb
Slide 11: C. difficile Infections (CDI)
C . difficile Infections (CDI) • CDI is currently the most prevalent hospital - acquired infection in the U.S., according to the CDC ̶ Surpassed Methicillin - resistant Staphylococcus aureus (MRSA) • CDI has been identified as an “urgent public health threat” by the CDC, FDA and EU health authoritie
Slide 12: Paradigm Shift
Paradigm Shift Fewer CDIs expected with co - administration of SYN - 004 Antibiotics Treatments • β - lactam • Fluoroquinolone • Clindamycin • Other • Metronidazole • Vancomycin • Fidaxomicin Current Paradigm SYN - 004 Paradigm C. difficile Infections (CDI) SYN - 004 + β - lactam Antibiotics* SYN -
Slide 13: SYN-004 Co-Administered with IV Antibiotics
SYN - 004 Co - Administered with IV Antibiotics Designed to neutralize β - lactam antibiotics in GI tract 13 SYN - 004 Antibiotic 1. SYN - 004 is designed as an oral enzyme tablet ( blue) to be co - administered with IV antibiotics (yellow ). 3. SYN - 004 is intended to remain in the GI tract and ne
Slide 14: Concept Validated by 1st Generation Candidate
Concept Validated by 1 st Generation Candidate Positive Phase 1 and 2 clinical data • 1 st generation candidate (P1A) was studied in four Phase 1 and one Phase 2 clinical trials conducted in Europe • 112 patients and 143 healthy normal subjects • Well tolerated with no safety concerns identified • A
Slide 15: SYN-004: Differentiators
SYN - 004: Differentiators • Prevention of antibiotic collateral damage to the gut microbiome may significantly reduce CDI, hospitalizations, and associated cost and morbidity • A normal microbiome provides colonization resistance, reducing the chance of C. difficile colonization, leading to CDI • P
Slide 16: SYN-004: Market Potential
SYN - 004: Market Potential Intended to target certain IV β - lactam antibiotics ~14M patients 11 ~27M prescriptions 10 ~118M doses of “SYN - 004 target” antibiotics purchased by U.S. hospitals to fill patient prescriptions 9 * Based on U.S. market data in 2012 ** Estimate based on the following ass
Slide 17: SYN-004 Clinical Trial Development
SYN - 004 Clinical trial development • Completed Phase 1a (40 participants) and 1b (24 participants) trials ̶ PK data supports that SYN - 004 should have no effect on the IV antibiotic in the bloodstream ̶ No clinically significant safety events were observed; well tolerated by participants • Initia
Slide 18: Pathogen-Specific Microbiome Therapeutic
Pathogen - Specific Microbiome Therapeutic 18 Treating the underlying cause not symptoms SYN APPROACH: Anti - Archaea specific therapeutic
Slide 19: Irritable Bowel Syndrome with Constipation (IBS-C)
Irritable Bowel Syndrome with Constipation IBS - C 19 Source: GlobalData Publication Irritable Bowel Syndrome Global Drug Forecast and Market Analysis 2014. Statistics • IBS is the most common chronic disease • 10 - 15% of the global population Prevalent IBS Cases (age > 10) Chronic GI disorder char
Slide 20: SYN-010 Modified-Release Statin
SYN - 010 Modified - Release Statin Designed to reduce methane production by M. smithii in the intestine 20 Healthy GI System Patients with IBS typically suffer from abdominal pain, bloating and constipation without an obvious cause such as inflammation or damage
Slide 21: The Gut: Methane Gas Production
The Gut Methane gas production 21 Bacteroides thetaiotaomicron is one of many bacterium that ferments carbohydrates in the gut which releases H 2 and CO 2 C arbohydrates Methanobrevibacter smithii archeabacteria consumes hydrogen gas from Bacteroides and produces methane, which is lost from gut as “
Slide 22: Methane Production: Underlying Cause of IBS-C
Methane Production: Underlying Cause of IBS - C 22 • Intestinal methane production is an underlying cause of constipation ̶ Critical discovery by Mark Pimentel, MD, and collaborators at Cedars - Sinai ▪ Extensive clinical evidence in IBS - C and now chronic idiopathic constipation (CIC) • Reduction
Slide 23: Anti-Methanogenic Therapy for IBS-C
Anti - Methanogenic Therapy for IBS - C SYN - 010 demonstrated significant reduction in methane gas 23 - 80 - 60 - 40 - 20 0 20 40 60 80 100 120 Control Statin 1 Statin 2 Statin 3 Statin 4 Statin 5 ΔCH 4 0 - 270 min Normalized to Control • Early studies demonstrated that statins in animal feed reduc
Slide 24: SYN-010: Targeting Production of Methane Gas
SYN - 010: Targeting Production of Methane Gas Effect of SYN - 010 on levels of M. smithii and total bacteria in rat GI tract after 10 days oral gavage dosing M. smithii Total bacteria 0.0E+00 5.0E+05 1.0E+06 1.5E+06 2.0E+06 2.5E+06 3.0E+06 3.5E+06 Duodenum Jejunum Ileum Left Colon M. smithii (cfu/g
Slide 25: SYN-010: Differentiators
SYN - 010: Differentiators • Modified - release statin formulation targeting intestinal methane production ̶ Leveraging detailed pharmacokinetic and safety profiles from decades of prior clinical use • Designed to treat the underlying cause of constipation • Patents and pending patents licensed thro
Slide 26: IBS Market Overview
IBS Market Overview 26 $690 $344 $120 $65 $49 $34 rifaximin lubiprostone linaclotide alosetron ramosetron others 2013 IBS Global Therapeutics ($ million) 2013 Market Sales US $440.1M EU5 $88.8 M Japan $20.7M Total $589.6M IBS Market Sales (2013 and 2023) Global Sales in 2023 are expected to be great
Slide 27: The IBS Market has Significant Unmet Needs
The IBS Market has Significant Unmet Needs 27 Source: GlobalData PharmaPoint , IBS Global Drug Forecast & Market Analysis 2014 New products with improved efficacy Products that effectively address IBS symptoms, such as abdominal pain and bloating Improved diagnosis of IBS/definitive test or biomarke
Slide 28: SYN-010 for IBS-C Clinical Development
SYN - 010 for IBS - C Clinical development • Preclinical data ̶ Demonstrated reduction of methane gas production by M. smithii with minimal impact on microbiome • File IND (2Q15) • Phase 2 clinical trials (initiation expected 2Q15) ̶ Demonstrate reduction of breath methane in IBS - C patients using
Slide 29: MS: Market Opportunity
MS: Market Opportunity • 400,000 MS patients in U.S. (~2.3 million worldwide) 12 • $17.8 billion forecasted worldwide sales in 2019 13 Trimesta TM – Oral add - on MS therapy 29 12 National Multiple Sclerosis Society. http://www.nationalmssociety.org 13 Credit Suisse. Multiple Sclerosis - Evolution o
Slide 30: Trimesta Completed Phase 2 Trial
Trimesta™ Completed Phase 2 trial • Principal investigator evaluating Trimesta TM (oral estriol): Rhonda Voskuhl, M.D., Professor of Neurology, Director of UCLA MS Program • Pilot trial (n=6): demonstrated an 82% decrease in both the number and volume of brain lesions over 6 months (p - values < 0.0
Slide 31: Trimesta Phase 2 Relapsing-Remitting MS Clinical Data
Trimesta™ Phase 2 relapsing - remitting MS clinical data • C linical outcomes presented b y lead investigator • Annualized relapse rate declined relative to standard of care, Copaxone ® ̶ Statistically significant 47% decrease in annualized MS relapse rate at 12 months with Trimesta ™ + Copaxone ® c
Slide 32: Trimesta Phase 2 Clinical Data (continued)
Trimesta™ Phase 2 relapsing - remitting MS clinical data (continued) • Evidence of improvement of physical disabilities ̶ Expanded Disability Status Scale (EDSS) scores in the Trimesta™ arm significantly improved during 24 month follow - up by at least 0.5 point (p=0.03) compared to the active contr
Slide 33: Trimesta Other MS Assets
Trimesta ™ Other MS assets • A separate Phase 2, randomized, double - blind, placebo - controlled clinical trial of female MS patients with cognitive dysfunction is ongoing 16 • Patents and pending patents licensed through UCLA: ̶ Issued method of treatment patents in the U.S. for MS therapy with es
Slide 34: Whooping Cough (Pertussis)
Whooping Cough (Pertussis) Market opportunity: millions of cases globally per year • Highly contagious disease caused by the bacteria Bordetella pertussis ( B. pertussis ) • Symptoms include severe coughing and subsequent breathing difficulties • Pertussis toxin is a major cause of disease virulence
Slide 35: SYN-005 mAb Combination
SYN - 005 mAb combination designed to target and neutralize pertussis toxin • Combines two highly synergistic humanized monoclonal antibodies (mAbs) designed to target and neutralize pertussis toxin • U.S. Orphan Drug designation granted September 2014 for the treatment of Pertussis • Patents and pe
Slide 36: Non-Human Primate Pertussis Study
0 5 10 15 0 10 20 30 40 50 Study Day W B C / u L ( x 1 , 0 0 0 ) Control SYN-005(1) SYN-005(2) SYN-005 therapy Normal WBC count Pertussis infection Non - Human Primate Pertussis Study n=3 Favorable decreases in white blood cell count indicates that SYN - 005 neutralizes pertussis toxin, which in a p
Slide 37: Financial Snapshot
Financial Snapshot * As of 12/31/14 weighted average exercise price is $2.01 ** As of 12/31/14 weighted average exercise price is $1.80 37 • Cash balance (as of 12/31/14) : ~$17.5 million • Current Price : $2.30 (as of 5/4/15) • 52 Week Range: $1.16 - $3.18 • Average Volume (3 months): 1,375,060 • S
Slide 38: Management Team
Management Team • Jeffrey Riley, CEO Pfizer, Nichols Institute (Quest), SmithKline Beecham, QIC • C. Evan Ballantyne, CFO Clinical Data, Inc., Avedro, ACNielsen, IMS • John Monahan, Ph.D., EVP R&D Avigen, Somatix, Triton Biosciences, Hoffman - LaRoche • Lewis Barrett, SVP Commercial Strategy Pfizer
Slide 39: Investment Consideration
Investment Consideration NYSE MKT: SYN 39 • Microbiome - focused, clinical - stage therapeutics to protect the microbiome while targeting pathogen - specific diseases ̶ Innovative, first - in - class product candidates for prevention and treatment ̶ Large markets addressing significant unmet medical
Slide 40: Top Emerging Technologies in 2014
Top 10 Emerging Technologies in 2014 40 Source: World Economic Forum, http://www3.weforum.org/docs/GAC/2014/WEF_GAC_EmergingTechnologies_TopTen_Brochure_2014.pdf
Slide 41: May 6, 2015 Slide Deck
May 6, 2015 NYSE MKT: SYN SYN - Slide Deck - May 2015 (5.6.2015) - FINAL
Synthetic Biologics — Investor Presentation (May 2015) on 6ix