Theriva Biologics, Inc. · AMEX:TOVX
Theriva Biologics — Synthetic Biologics — Investor Presentation (April 2012)
32 slides from Theriva Biologics's investor presentation, Apr 24, 2012
Slide 1: Synthetic Biologics
here ee NYSE Amex: SYN April 24,2012 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: Corporate Overview
Corporate Overview • Ticker symbol – NYSE Amex: SYN • Late - stage clinical programs ‒ Oral estriol for multiple sclerosis (MS) ▪ $ 8.75 million in grant funding ‒ Flupirtine for fibromyalgia ▪ $ 17.5 million partnership with Meda AB ‒ Oral zinc acetate for Lou Gehrig’s disease (ALS ) ▪ Phase II/III
Slide 4: Recent Milestones
Recent Milestones 4 Advancing late - stage and preclinical synthetic biologics programs x Completed planned enrollment in Phase II clinical trial for relapses in MS x Initiated Phase II clinical trial for cognitive dysfunction in MS x Strengthened Synthetic Biologics’ team: Jeff Riley, CEO; Evan Bal
Slide 5: Financial Snapshot
Financial Snapshot • Current Price : $1.57 • 52 Week Range: $0.42 - $ 2.95 • Average Volume (3 months) : 280,594 • Shares Outstanding: ~ 32.8 million • Market Capitalization: ~$51 million • Cap Structure: Straight Common. No Debt. No Preferred. • Cash: ~$6.9 million ( pro forma March 26, 2012) Stock
Slide 6: Synthetic Biologics' Team
6 Synthetic Biologics’ Team • Jeffrey Riley, CEO Pfizer, Nichols Institute (Quest), SmithKline Beecham, QIC • C. Evan Ballantyne, CFO Clinical Data, Inc., Avedro , ZymeQuest , ACNielsen, IMS • John Monahan, Sr. VP R&D Avigen, Somatix, Triton Biosciences, Hoffman - LaRoche
Slide 7: Product Pipeline
Product Pipeline Medical Indication | Drug Candidate Pre-IND Phase | Phase Il Phase Ill Relapsing-remitting mu multiple sclerosis ‘Temeste =————p Cognitive dysfunction om ‘nnmultiple sclerosis Trimesta —_—_——_> Fibromyalgia Effirma™ ee Lou Gehrig's disease AEN-100 Medical Indication Biologic Candida
Slide 8: MS: Market Opportunity
MS: Market Opportunity According to the National Multiple Sclerosis Society − 400,000 MS patients in U.S. − 200 people diagnosed per week in the U.S. − 2.5 million patients worldwide − 1.75 million patients worldwide are women 1 Current estimated sales of injectable disease - modifying therapies 2 C
Slide 9: MS: Historical Efficacy of Estriol
MS: Historical Efficacy of Estriol The “Pregnancy Hormone” – Linked to fetal immune privilege • Hormone produced by placenta during pregnancy • Approved in Europe/Asia for 40 years to treat symptoms of menopause • Never approved in the U.S. for any indication • Pr egnancy I n M ultiple S clerosis (P
Slide 10: MS: Relapsing-Remitting MS
MS: Relapsing - Remitting MS Drug candidate – Trimesta (oral estriol ) • Dr. Voskuhl completed an investigator - initiated, 10 - patient, 22 - month, single - agent, crossover clinical trial evaluating Trimesta – Over 6 months both the number and volume of brain lesions decreased by 82% (p - values
Slide 11: MS: Relapsing-Remitting MS Phase II Clinical Trial Status
MS: Relapsing - Remitting MS Phase II Clinical trial status – Trimesta • Randomized, double - blind, placebo - controlled clinical trial 1 of females with relapsing - remitting MS • Comparing relapse rates over 2 years between 8 mg of Trimesta taken daily versus matching placebo with Copaxone® as st
Slide 12: MS: Cognitive Dysfunction
MS: Cognitive Dysfunction • Dr. Voskuhl of UCLA completed investigator - initiated clinical trial evaluating Trimesta − Demonstrated a 14% improvement in P aced A uditory S erial A ddition T est (PASAT) cognitive testing scores (p = 0.04) in relapsing - remitting MS patients at 6 months of therapy –
Slide 13: MS: Cognitive Dysfunction Phase II Clinical Trial Status
MS: Cognitive Dysfunction in MS Phase II Clinical trial status – Trimesta • Initiated clinical trial in January 2012 1 − Randomized, double - blind, placebo - controlled clinical trial of female MS patients with cognitive dysfunction at UCLA − Enrolling 64 female MS patients − Patients randomized in
Slide 14: Fibromyalgia: Market Opportunity
Fibromyalgia: Market Opportunity Commercial potential for Effirma (flupirtine) • Condition characterized by widespread pain and stiffness throughout body, accompanied by severe fatigue, insomnia and mood symptoms – Affects 3 - 6% of worldwide population, including ~10 million people in the U.S. 1 •
Slide 15: Meda AB Corporate Partnership
Meda AB Corporate Partnership Effirma for fibromyalgia attracted large pharma collaboration • $17.5 million corporate partnership with Meda * – Meda assumes all future development costs – $2.5 million upfront payment (May 2010) – $15 million in future milestone payments – 3.5% future royalties based
Slide 16: Lou Gehrig's Disease: Market Opportunity
Lou Gehrig’s Disease: Market Opportunity • Affects motor nerve cells in brain and spinal cord • Progressive loss of muscle strength and coordination • No cure for Lou Gehrig’s disease (ALS) • ALS Association facts − Up to 30,000 people have Lou Gehrig’s disease at any given time in the U.S . − Avera
Slide 17: Lou Gehrig's Disease: Prior Zinc Therapy Study
Lou Gehrig’s Disease: Prior Zinc Therapy Study • Top - line results from PNA Center for Neurological Research (PNA) Phase I/II study of oral high dose zinc therapy 1 • Top - line results − No safety issues related to zinc therapy − Disease progression slowed in study patients ▪ On average, patients
Slide 18: Lou Gehrig's Disease: Clinical Trial Status AEN-100
Lou Gehrig’s Disease • Planned double - blind, placebo - controlled adaptive Phase II/III trial − Enroll at least 65 patients at 6 clinical sites − Randomized into treatment or matching placebo arms − All patients remain on RILUTEK® (riluzole) as standard of care − Dosed for at least 6 months with p
Slide 19: Next Generation Gene Therapy
Next Generation Gene Therapy 19 2012 − Field demonstrating clinical successes − Next generation gene therapies - technology to regulate protein expression 1990 - 2000 − High hopes 2000 - 2004 − Set - backs − Safety concerns 2004 - 2011 − Corporate abandonment of gene therapy
Slide 20: Intrexon Corporation Antibody Discovery
Intrexon Corporation 20 Antibody Discovery More than 350 employees Image courtesy of Intrexon Corporation.
Slide 21: Modular Controllable Gene System
21 Modular Controllable Gene System (RheoSwitch Therapeutic System® + GOI) ECB Injection site RTS® - GOI Activator Ligand (oral dosing) Activator Ligand (small molecule) DNA Delivery GOI GOI GOI GOI RTS® Controlled Protein Expression RheoSwitch Therapeutic System® Protein of Interest GOI: Gene of In
Slide 22: RheoSwitch Therapeutic System: DNA delivered to pulmonary arteries
22 RheoSwitch Therapeutic System® Synthetic DNA delivered to pulmonary arteries RheoSwitch Therapeutic System® is a registered trademark of Intrexon Corporation.
Slide 23: RheoSwitch Therapeutic System: Gene production turned off
23 RheoSwitch Therapeutic System® Gene production is “turned off”
Slide 24: Oral Activator Ligand Turns On Gene Production
24 Oral Administration of RheoSwitch ® Activator Ligand “Turns on” the gene production in vivo
Slide 25: RheoSwitch Technology
RheoSwitch ® Technology RheoSwitch ® control designed to improve safety, Reduce side effects and enhance efficacy On - Off switch with dose adjustment control 25 Image courtesy of Intrexon Corporation. RheoSwitch ® is a registered trademark of Intrexon Corporation.
Slide 26: Anatomy of PAH
Anatomy of PAH 26 Normal Artery Flow in the Heart and Lungs of Normal Patient Constricted Artery Flow in the Heart and Lungs of PAH Patient
Slide 27: Current PAH Market
Current PAH Market 27 • Global PAH market expected to reach $3.6 billion 1 by 2015 • High morbidity and mortality • Three medical therapies − Prostacyclin analogs reserved for more severe stage of disease − Can slow the course and lower the mortality • Prostacyclin analogs have significant systemic
Slide 28: PAH Product Clinical Need/Positioning
PAH Product Clinical Need/Positioning • Gene therapy is intended to overcome dose - limiting toxicities by generating high levels of prostacyclin at the site of action – the pulmonary arteries • Synthetic DNA - based strategy − Via a single procedure, deliver a vector to the pulmonary arteries − Vec
Slide 29: Intellectual Property
Intellectual Property • Intrexon Corporation ‒ RheoSwitch Therapeutic System® and vector constructs • Synthetic Biologics ‒ Select issued patents ▪ Trimesta (oral estriol ) o U.S. Patent 6,936,599, issued August 30, 2005 ▪ Effirma (flupirtine) o U.S. Patent 6,610,324, issued August 26, 2003 ‒ Select
Slide 30: Coming Milestones
30 Coming Milestones • Initiate Phase II/III clinical trial for Lou Gehrig’s disease later in 2012 • Complete patient enrollment in Phase II clinical trial of females with relapsing - remitting MS • Establish and expand new and existing collaborations in the synthetic biologics area
Slide 31: Investment Summary
31 Investment Summary • Three drug candidates for treatment of MS, fibromyalgia and ALS that are either in Phase II clinical development or about to begin Phase II/III clinical development − Together, treatments for these serious conditions represent markets valued at approximately $18 billion − Cos
Slide 32: Synthetic Biologics
here ee NYSE Amex: SYN ‘SYNTHETIC April 24,2012
Synthetic Biologics — Investor Presentation (April 2012) on 6ix