Theriva Biologics, Inc. · AMEX:TOVX
Theriva Biologics — Synthetic Biologics — Investor Presentation (January 2016)
38 slides from Theriva Biologics's investor presentation, Jan 11, 2016
Slide 1: Investor Slide Deck January 11, 2016
Investor Slide Deck January 11, 2016
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 Considerations
Investment Considerations NYSE MKT: SYN • Lead candidates in Phase 2 development focused on protecting the gut microbiome while targeting pathogen - specific diseases • SYN - 010 = innovative approach to target an underlying cause of irritable bowel syndrome with constipation (IBS - C), not just the
Slide 4: 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 5: Product Pipeline
Product Pipeline C - Cedars - Sinai Medical Center collaboration I - Intrexon Corporation collaboration T - The University of Texas at Austin collaboration Completed Planned – 2016 Therapeutic Area Proprietary Technology Product Candidate Discovery Preclinical Phase 1 Phase 2 Phase 3 IBS - C [Acute
Slide 6: Milestones: Achieved & Upcoming
Milestones: Achieved & Upcoming Therapeutic Area/Product Candidate Timeline IBS - C – SYN - 010: Phase 2 (1 st study; acute, placebo - controlled) 2Q 2015 – Initiated Phase 2 4Q 2015 – Reported Phase 2 topline data Phase 2 (2 nd study; extension, SYN - 010 42mg) 2H 2015 – Initiated Phase 2 1H 2016 –
Slide 7: Diseases Directly Influenced by the Gut Microbiome
Source: Genome Medicine 2011, 3 :14 http://genomemedicine.com/content/3/3/14 Diseases Directly Influenced by the Gut Microbiome 7
Slide 8: Human Microbiome
Human Microbiome The body has 10 times as many microbe cells as human cells Source: http://commonfund.nih.gov/hmp/overview.aspx Human Microbiome > 1,000,000 Genes Human Genome 23,000 Genes 99% of Genes in the body are Microbial, NOT Human Leveraging the microbiome could significantly change medicine
Slide 9: Human Microbiome Over Time
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. Human Microbiome Over Time Response to environmental conditions and life stages 9
Slide 10: Top Emerging Technologies
Top 10 Emerging Technologies Source: World Economic Forum, http://www3.weforum.org/docs/GAC/2014/WEF_GAC_EmergingTechnologies_TopTen_Brochure_2014.pdf 10 SYN Focus
Slide 11: SYN-010 Treatment of IBS-C
SYN-010 Treatment of IBS-C synthetic ()
Slide 12: Pathogen-Specific Microbiome Therapeutic
Pathogen - Specific Microbiome Therapeutic Treating the underlying cause not symptoms SYN APPROACH: Anti - Archaea specific therapeutic 12
Slide 13: Irritable Bowel Syndrome
Irritable Bowel Syndrome • IBS is a chronic GI disorder characterized as a group of symptoms • Diarrhea/constipation • Abdominal discomfort • Bloating • Severely impacts and reduces quality of life • Statistics • 10 - 15% of the global populatio n • Who is affected? • Women = 66.1%; Men = 33.9% • IB
Slide 14: SYN-010: Proprietary Modified-Release Lovastatin Lactone
SYN - 010: Proprietary Modified - Release Lovastatin Lactone Designed to reduce methane production by M. smithii in the intestine Bacteroides thetaiotaomicron is one of many bacterium that ferments carbohydrates in the gut which releases H 2 and CO 2 C arbohydrates Methanobrevibacter smithii archea
Slide 15: SYN-010: Differentiators
SYN - 010: Differentiators • 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 of intestinal methane ha
Slide 16: Anti-Methanogenic Therapy for IBS-C
Anti - Methanogenic Therapy for IBS - C • Studies demonstrated that statins in animal feed reduced methane gas in ruminant animals (4 - chambered stomach) • Dr. Pimentel translated the use of statins to reduce methane in humans ( single - chambered stomach) by evaluating commercial lovastatin formul
Slide 17: Phase 2 trial design for IBS-C
SYN - 010 Phase 2 trial design for IBS - C – multiple sites in U.S. 60 patients SYN - 010 High Dose 20 patients SYN - 010 Low Dose 20 patients Placebo 20 patients SYN - 010 High Dose 54 patients* Study #1 – 4 Week Acute Treatment Study #2 – 8 Week Extension Treatment Topline Analysis Study #1 Toplin
Slide 18: Improved Weekly Stool Frequency Response
16% 41% 22% 0% 10% 20% 30% 40% 50% 60% 70% 80% % Weeks Patients Had a Stool Response Placebo SYN - 010 21 mg SYN - 010 42 mg SYN - 010 Improved Weekly Stool Frequency Response Percentage of weeks subjects had a response ( mITT Population) *Statistically different from Placebo based an a Wilcoxon Man
Slide 19: Improved Weekly Abdominal Pain Response
11% 15% 29% 0% 10% 20% 30% 40% 50% 60% 70% 80% % Weeks Patients Had a Pain Response Placebo SYN - 010 21 mg SYN - 010 42 mg SYN - 010 Improved Weekly Abdominal Pain Response Percentage of weeks subjects has a response ( mITT Population) P=0.08† P=0.26† †P - value based an a Wilcoxon Mann - Whitney t
Slide 20: Effects on Combined Weekly Response
1% 7% 9% 0% 5% 10% 15% 20% 25% 30% % Weeks Patients Had a Weekly Response Placebo SYN - 010 21 mg SYN - 010 42 mg SYN - 010 Effects on Combined Weekly Response Trend to dose - dependent improvement ( mITT Population) †P - value based an a Wilcoxon Mann - Whitney test for the comparison of the SYN -
Slide 21: Effects on Global Assessment of Improvement
16% 32% 29% 21% 25% 35% 21% 31% 50% 0% 10% 20% 30% 40% 50% 60% Placebo SYN - 010 21 mg SYN - 010 42 mg % IBS - GAI Responders Week 2 Week 3 Week 4 SYN - 010 Effects on Global Assessment of Improvement Improvement in IBS - GAI over time at 42 mg dose ( mITT Population) †p - value based an a Chi - squ
Slide 22: SYN-010 Clinical trial development
SYN - 010 • Completed first Phase 2 trial (acute study) – 63 patients • Complete second Phase 2 trial (8 week extension study) • Topline data expected 1H 2016 • 54 patients from first Phase 2 trial continued into an 8 week, open - label extension study administering single, daily, doses of SYN - 010
Slide 23: IBS Market Overview
IBS Market Overview Source: IMS Audited Sales Data, Midas Global Sales (Analytics Link) 23 2015 IBS Global Therapeutic Sales Forecast ~$669.3M Global IBS Sales in 2023 are expected to be greater than $1.5B Market growth attributed to : • Increased u ptake of Linzess ® and label expansion of Xifaxan
Slide 24: IBS-C Market Overview
IBS - C Market Overview 1 SYN - 010 targets an underlying cause versus competition Product Candidate SYN - 010 * Linzess Amitiza OTC Laxatives Plecanatide Tenapanor Company Synthetic Allergan Takeda Various Synergy Ardelyx Phase/Status Phase 2 Marketed Marketed Marketed Phase 3 Phase 3 Treat underly
Slide 25: SYN-004 Prevention of C. difficile Infection and AAD
SYN - 004 Prevention of C. difficile Infection and AAD
Slide 26: Collateral Damage Caused by Antibiotic Use
Collateral Damage Caused by Antibiotic Use • 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 microbiom
Slide 27: C. difficile Infections (CDI)
C. difficile Infections (CDI) Preventing CDIs is a national priority 1 Leffler DA et al. N Engl J Med 2015; 372:1539 - 1548. 2 (APIC) National Prevalence Study of Clostridium difficile in U.S. Healthcare Facilities. November 11, 2008. 3 Louie TJ, et al. N Engl J Med 2011;364:422 – 31. 4 Cornely OA,
Slide 28: Paradigm Shift
Paradigm Shift Fewer CDIs expected with co - administration of SYN - 004 Treatments • β - lactam • Fluoroquinolone • Clindamycin • Other • Metronidazole • Vancomycin • Fidaxomicin SYN - 004 Paradigm SYN - 004 + beta - lactam antibiotics* SYN - 004 designed to protect the natural balance of the gut m
Slide 29: SYN-004 Broad patent estate
SYN - 004 • ~30 U.S. and foreign patents; ~30 U.S. and foreign patents pending • Composition of matter claims and pharmaceutical compositions of beta - lactamases, including SYN - 004, was issued in November 2014 (U.S. Patent 8,894,994) • Carries a term to at least 2031 • An extensive portfolio of g
Slide 30: SYN-004 Co-Administered with Certain IV Antibiotics
SYN - 004 Co - Administered with Certain IV Antibiotics Designed to neutralize beta - lactam antibiotics in GI tract SYN - 004 Antibiotic To view the SYN - 004 mechanism of action video , please visit: http:// www.syntheticbiologics.com/SYN - 004 1. SYN - 004 is an oral enzyme tablet ( blue) to be c
Slide 31: Clinical trials completed to date
SYN - 004 • First generation candidate, P1A, was evaluated in four Phase 1 and one Phase 2 clinical trials conducted in Europe • In total, 112 patients and 143 healthy normal subjects participated in the studies • Completed Phase 1a (40 participants) and 1b (24 participants) trials • PK data support
Slide 32: SYN-004 Ongoing and planned trials
• Second Phase 2a trial (ongoing) • Characterize SYN - 004 activity on ceftriaxone in the small intestine in the presence of esomeprazole, an approved, OTC proton pump inhibitor • Phase 2b Proof - of - Concept trial (ongoing) • Evaluate the ability of SYN - 004 to prevent CDI and AAD in patients adm
Slide 33: Phase 2b trial design for CDI prevention
33 ~75 Global Clinical Sites 370 patients SYN - 004 + Ceftriaxone Placebo + Ceftriaxone Primary Endpoint: • Prevention of CDI and C. difficile associated diarrhea (CDAD) Secondary Endpoints: • Prevention of AAD • Limiting disruption of gut microbiome diversity 1:1 SYN - 004 Phase 2b trial design for
Slide 34: SYN-004: Market Potential
SYN - 004: Market Potential Intended to target certain IV beta - lactam antibiotics * Estimate based on the following assumptions: Cost of SYN - 004/day of $100 x 117M days of “SYN - 004 target” β - lactam antibiotics in 2014 1 - Arlington Medical Resources (AMR), a Decision Resources Group Company
Slide 35: C. difficile Market Overview
C. difficile Market Overview 1 SYN - 004 is a prophylactic approach versus treatment Currently the only methods for preventing primary C. difficile infection are through antibiotic stewardship and infection control 1 N ot a comprehensive list of pipeline products; representative of compounds that ar
Slide 36: Financial Highlights
Financial Highlights
Slide 37: Financial Snapshot
Financial Snapshot • Cash balance (as of 9/30/15): ~$31.8 million • Current Price: $ 2.27 (as of 1/5/16) • 52 Week Range: $ 1.48 - $4.32 • Average Volume (3 months): 933,995 • Shares Outstanding: ~91 million (as of 11/2/15 ) • Options Outstanding: ~7.5 million* • Warrants Outstanding: ~7.9 million**
Slide 38: Investor Slide Deck January 11, 2016
Investor Slide Deck January 11, 2016 SYN – Slide Deck (1.11.2016) - FINAL
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