Theriva Biologics, Inc. · AMEX:TOVX
Theriva Biologics — Synthetic Biologics — Investor Presentation (November 2014)
32 slides from Theriva Biologics's investor presentation, Nov 3, 2014
Slide 1: Synthetic Biologics November 2014
oe a NYSE MKT: SYN November 2014 SYNTHETIC
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 • Portfolio of novel therapeutics with large market opportunities ̶ Innovative, first - in - class product candidates for prevention and treatment ̶ Large markets with blockbuster potential and unmet need ̶ Shorter treatment period may reduce clinical trial duration • Several
Slide 4: Management Team
Management Team • Jeffrey Riley, CEO Pfizer, Nichols Institute (Quest), SmithKline Beecham, QIC • C. Evan Ballantyne, CFO Clinical Data, Inc., Avedro, ZymeQuest, ACNielsen, IMS • John Monahan, Ph.D., EVP R&D Avigen, Somatix, Triton Biosciences, Hoffman - LaRoche • Michael Kaleko, M.D., Ph.D., SVP R&
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 – 2014 / 2015 Therapeutic Area Product Candidate Biologic Agent/ Drug Compound Discovery Preclinical Phase 1 Phase 2 Phase 3 R
Slide 6: MS: Market Opportunity
MS: Market Opportunity • 400,000 MS patients in U.S. (~2.3 million worldwide) 1 • $17.8 billion forecasted worldwide sales in 2019 2 Trimesta TM – Oral add - on MS therapy Novartis/Gilenya $1.969 11% Teva/Copaxone $686 4% Biogen Idec/Avonex $2.206 12% Merck KGaA/Rebif $1.578 9% Biogen Idec/Tysabri $
Slide 7: Trimesta: Observed efficacy of estriol in MS
Trimesta™ Observed efficacy of estriol in MS • Pregnancy hormone linked to decreased MS relapse rate • Hormone produced by placenta during pregnancy, which peaks in third trimester • Landmark European observational study published in New England Journal of Medicine 3 − Relapse rates in 254 women dia
Slide 8: 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 9: Trimesta Phase 2 relapsing-remitting MS clinical data
Trimesta™ Phase 2 relapsing - remitting MS clinical data • C linical outcomes presented b y lead PI, Rhonda Voskuhl, M.D. • Annualized relapse rate declined relative to standard of care, Copaxone ® ̶ Statistically significant 47% decrease in annualized MS relapse rate at 12 months with Trimesta ™ +
Slide 10: Trimesta Phase 2 relapsing-remitting MS 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 11: Trimesta Phase 2 cognitive dysfunction clinical trial
Trimesta ™ Phase 2 cognitive dysfunction clinical trial • Randomized , double - blind, placebo - controlled clinical trial of female MS patients conducted at UCLA 6 − Enrollment ongoing − 64 patient study − Trimesta TM versus matching placebo over one year with all patients remaining on st andard FD
Slide 12: Trimesta: Planned next steps
Trimesta™ Planned next steps • Trimesta ™ for relapsing - remitting MS and cognitive dysfunction in MS ̶ Strategic partnering efforts supported by demonstrated therapeutic potential and safety profile of oral estriol (ongoing) ̶ MRI analyses ongoing to evaluate changes in the brain that correlate wi
Slide 13: Paradigm Shift
Paradigm Shift Fewer C. difficile infections 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 β - lactam Antibiotics* + SYN
Slide 14: SYN-004: Prophylactic treatment for C. difficile
• 24 million patients administered IV antibiotics annually in the U.S. 8 ̶ 1.1 million patients infected with C. difficile annually 9 ̶ Patients with C. difficile hospitalized approximately 4 - 7 extra days 10 ̶ $ 8.2 billion in costs associated with C. difficile - related stays in hospital 11 ̶ 30,
Slide 15: SYN-004: Market Potential
SYN - 004: Market Potential Intended to target certain IV β - lactam antibiotics 14.4M patients 15 26.5M prescriptions 14 117.6M doses of “SYN - 004 target” β - lactam antibiotics purchased by U.S. hospitals to fill patient prescriptions 13 * Based on U.S. market data in 2012 13 - 15 This informatio
Slide 16: SYN-004: Biologic for C. difficile infection
• Oral enzyme designed to be co - administered with certain IV beta - lactam antibiotics • Designed to neutralize certain IV beta - lactam antibiotics in the gut, while not disrupting the systemic antibiotic effect • Intended to protect and maintain the balance of bacterial flora in the GI tract, or
Slide 17: P1A: First Generation Candidate Phase 1 and 2 clinical data
P1A: 1 st Generation Candidate Phase 1 and 2 clinical data • P1A was studied in four (4) Phase 1 and one (1) Phase 2 clinical trials conducted in Europe ‒ 112 patients and 143 healthy normal subjects exposed, generally: ▪ P reserved the normal intestinal microflora when co - administered with IV amp
Slide 18: SYN-004 Phase 1a and 1b clinical trials
SYN - 004 Phase 1a and 1b clinical trials • SYN - 004 in development for the prevention of C. difficile infections • Represents a 2 nd generation oral candidate intended to expand activity to include penicillins plus certain cephalosporins • Patents and pending patents: ̶ SYN - 004 patent pending on
Slide 19: SYN-010: Market Potential
• Up to $10 billion in direct costs to the U.S. healthcare system and up to $20 billion in indirect costs 16 • 40 million IBS patients in the U.S. 17 ̶ ~13.2 million C - IBS patients in the U.S. 18 • ~$2.7 billion market expected by 2020 19 • Major IBS players include ‒ Salix: XIFAXAN® ( rifaximin )
Slide 20: SYN-010: Antimethanogenic Therapy for C-IBS
SYN - 010: Antimethanogenic Therapy for C - IBS • Intestinal methane (CH 4 ) production is an underlying cause of constipation ̶ Critical discovery by Dr. Mark Pimentel and collaborators at Cedars - Sinai ̶ Extensive clinical evidence in C - IBS and now chronic idiopathic constipation (CIC) • Reduct
Slide 21: SYN-010: A Differentiated Statin Formulation
SYN - 010: A Differentiated Statin Formulation • Modified - release statin formulation targeting intestinal CH 4 production ̶ Treating the underlying cause of constipation ̶ Leveraging detailed pharmacokinetic and safety profiles from decades of prior clinical use • Patents and pending patents licen
Slide 22: SYN-010: Most Effective Statin CH4 Inhibitor In Vitro
- 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 SYN - 010: Most Effective Statin CH 4 Inhibitor In Vitro Figure 1: Effect of statins on CH 4 production in stool from C - IBS patients *5 mg statin/g wet stool. Data
Slide 23: SYN-010: NOT Microbicidal
SYN - 010: NOT Microbicidal Figure 2: Effect of Statin 1 on levels of M. smithii and total bacteria in the 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 tissu
Slide 24: SYN-010: Clinical Validation
SYN - 010: Clinical Validation 0 20 40 60 80 100 120 140 No Statin Statin 2 Statin 1 Form 1 Statin 1 Form 1 No Statin Statin 1 Form 2 Statin 1 Form 1 + Form 2 Breath CH 4 (ppm) Figure 3: Reduction of breath CH 4 in a C - IBS patient by Statin 1 but not Statin 2 Statin 1 Form 1 Modest efficacy No blo
Slide 25: SYN-010: Differentiated Product for C-IBS
SYN - 010: Differentiated Product for C - IBS SYN - 010 Property Differentiator x Designed to t reat the underlying cause of constipation • Not simple “mass movement”; no diarrhea x Patent protected technology • Issued patents, multiple patent applications x Uses a very potent statin CH 4 inhibitor
Slide 26: SYN-010: Planned next steps
SYN - 010 Planned next steps • SYN - 010 for C - IBS ̶ File IND (1Q15) ̶ Complete Phase 1 PK / PD clinical trial (1H15) ̶ Initiate Phase 2 clinical trial (1H15) ̶ Pursue Phase 3 clinical trial(s) (2016) 26
Slide 27: Whooping Cough (Pertussis) Market opportunity
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 28: SYN-005: mAb combination to neutralize pertussis toxin
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 29: 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 30: Financial Snapshot
Financial Snapshot * ~7 million warrants issued 10/16/14, with exercise price of $1.75 ** As of 6/30/14 weighted average exercise price is $2.05 ** As of 6/30/14 weighted average exercise price is $2.16 30 • Cash balance (as of 6/30/14) : ~$7.8 million • Capital raise (10/16/14 close) : ~$ 18.9 mill
Slide 31: Upcoming Milestones
Upcoming Milestones Therapeutic Area (Product Candidate) 2014 2015 2016 MS ( Trimesta TM ) 1H – MRI Data Review Partnering Deal Initiate Pivotal Phase 3 C. difficile (SYN - 004) 4Q – IND 4Q – Phase 1a/1b Data 1H – Phase 2a Data 2H – Phase 2b Data Initiate Pivotal Phase 3 C - IBS (SYN - 010) 1Q – IND
Slide 32: November 2014 NYSE MKT: SYN
November 2014 NYSE MKT: SYN SYN - Slide Deck - Nov 2014 (11.3.2014) - FINAL
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