Theriva Biologics, Inc. · AMEX:TOVX
Theriva Biologics — Synthetic Biologics — Investor Presentation (February 2015)
30 slides from Theriva Biologics's investor presentation, Feb 9, 2015
Slide 1: Synthetic Biologics February 2015
he NYSE MKT: SYN Sy NIENn February 2015
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: 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 3 Therapeutic Area Product Candidate Biologic Agent/ Drug Compound Discovery Preclinical Phase 1 Phase 2 Phase 3 Relaps
Slide 4: 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 5: 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 5 1 National Multiple Sclerosis Society. http://www.nationalmssociety.org 2 Credit Suisse. Multiple Sclerosis - Evolution or Rev
Slide 6: 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 7: 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 8: 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 9: 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 5 • Patents and pending patents licensed through UCLA: ̶ Issued method of treatment patents in the U.S. for MS therapy with est
Slide 10: What is the Microbiome?
What is the Microbiome ? • The GI tract is predominantly a bacterial ecosystem often called the gut microbiome that plays an essential role in supporting life and health ̶ Digests food ̶ Synthesizes essential nutrients ̶ Prevents disease - causing bacteria from invading the body • When the natural b
Slide 11: Collateral Damage Caused by Antibiotic Use
Collateral Damage Caused by Antibiotic Use 11 • Antibiotics ̶ Infused or ingested to prevent/treat primary infections ̶ Carried to the liver, transported to the bile and ultimately excreted via the large intestine ̶ May unintentionally upset the natural balance of the gut microbiome by killing off g
Slide 12: 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 13: 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 14: SYN-004 Co-Administered with IV Antibiotics
SYN - 004 Co - Administered with IV Antibiotics Designed to neutralize β - lactam antibiotics in GI tract 14 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 15: SYN-004: Market Potential
SYN - 004: Market Potential Intended to target certain IV β - lactam antibiotics 14.4M patients 16 26.5M prescriptions 15 117.6M doses of “SYN - 004 target” antibiotics purchased by U.S. hospitals to fill patient prescriptions 14 * Based on U.S. market data in 2012 ** Estimate based on the following
Slide 16: 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 (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 int
Slide 17: SYN-004 Reduces antibiotic resistance and protects microbiome
• Commonly utilized broad - spectrum antibiotics are cost - effective for many common infections • IV antibiotics excreted into GI tract can upset the natural balance of the microbiome • Prevention of antibiotic collateral damage to the gut microbiome may significantly reduce CDI, hospitalizations,
Slide 18: SYN-004 Clinical trial development
SYN - 004 Clinical trial development • Phase 1a and 1b trials (ongoing) • Phase 2a trial goals (initiation expected 1Q15) ̶ Characterize SYN - 004 activity on ceftriaxone in the small intestine ̶ Demonstrate SYN - 004 has no activity on ceftriaxone in the bloodstream • Phase 2b (Proof - of - Concept
Slide 19: Pathogen-Specific Microbiome Therapeutic
Pathogen - Specific Microbiome Therapeutic 19 Treating the underlying cause not symptoms
Slide 20: Constipation-Predominant Irritable Bowel Syndrome C-IBS
Constipation - Predominant Irritable Bowel Syndrome C - IBS 20 IBS is prevalent Up to 15% of the population has IBS^*+ • Up to 88.5 million patients (worldwide)^*+ • Up to 40 million patients (USA)^*+ IBS is under - diagnosed due to the lack of a definitive test or biomarker. An estimated 30% of pat
Slide 21: SYN-010: Antimethanogenic Therapy for C-IBS
SYN - 010: Antimethanogenic Therapy for C - IBS 21 • Intestinal methane production is an underlying cause of constipation ̶ Critical discovery by Mark Pimentel, MD, and collaborators at Cedars - Sinai ▪ Extensive clinical evidence in C - IBS and now chronic idiopathic constipation (CIC) • Reduction
Slide 22: 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 23: SYN-010: To Treat the Underlying Cause of Constipation
SYN - 010: To Treat the Underlying Cause of Constipation • Modified - release statin formulation targeting intestinal methane production ̶ Leveraging detailed pharmacokinetic and safety profiles from decades of prior clinical use • Patents and pending patents licensed through Cedars - Sinai ̶ An ext
Slide 24: SYN-010 for C-IBS Differentiators and clinical development
SYN - 010 for C - IBS Differentiators and clinical development • Differentiators ̶ Designed to t reat the underlying cause of constipation ̶ Patent protected delivery, technology and formulation ̶ Unique regulatory pathway – 505(b )(2 ) • Clinical trial development ̶ File IND (1H15) ̶ Phase 2 clinic
Slide 25: 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 26: SYN-005 mAb combination targeting 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 27: 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 28: Financial Snapshot
Financial Snapshot * As of 9/30/14 weighted average exercise price is $2.03 ** ~945,000 warrants as of 9/30/14 with a weighted average exercise price is $2.16; ~7 million warrants issued 10/16/14, with exercise price of $1.75 28 • Cash balance (as of 9/30/14) : ~$3.3 million • Capital raise (10/16/1
Slide 29: Upcoming Milestones
Upcoming Milestones Therapeutic Area (Product Candidate) 2015 2016 MS ( Trimesta TM ) 1H – MRI Data Review 1H – Partnering Deal Initiate Pivotal Phase 3 C. difficile (SYN - 004) 1H – Phase 2a Data 2H – Phase 2b Data Initiate Pivotal Phase 3 C - IBS (SYN - 010) 1H – IND 2H – Phase 2a/2b Data Initiate
Slide 30: February 2015 NYSE MKT: SYN SYN - Slide Deck - Feb 2015 (2.6.2015) -
February 2015 NYSE MKT: SYN SYN - Slide Deck - Feb 2015 (2.6.2015) - FINAL
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