Theriva Biologics, Inc. · AMEX:TOVX
Theriva Biologics — Adeona Pharmaceuticals — Investor Presentation (February 2012)
45 slides from Theriva Biologics's investor presentation, Feb 13, 2012
Slide 1: Adeona Pharmaceuticals d/b/a Synthetic Biologics
- SYNTHETIC LoGitie¢es February 13, 2012 Adeona Pharmaceuticals, Inc. (NYSE Amex: AEN) d/b/a Synthetic Biologics ~ >) SYNTHETIC Pending Name and Ticker Symbol Change
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation includes forward-looking statements on Adeona's current expectations and projections about future events. In some cases forward-looking statements can be identified by terminology such as “may,” "should,” "potential," “continue,” "expects," "anticipates,"
Slide 3: Corporate Overview
Corporate Overview ¢ Public Company (NYSE Amex: AEN) - Pending name change to Synthetic Biologics, Inc. (NYSE Amex: SYN) (anticipated February 15, 2012) * Synthetic DNA-based medicines for serious unmet medical needs - Exclusive collaboration with Intrexon Corporation (November 21, 2011) - Synthetic
Slide 4: Corporate Strategy
Corporate Strategy * Harness synthetic biology to treat human diseases * Design and develop synthetic DNA-based therapeutics * Forge and expand new and existing collaborations
Slide 5: Financial Snapshot
Financial Snapshot * Current Price: $2.69 * 52 Week Range: $0.42 - $2.75 * Average Volume (3 months): 504,992 ¢ Shares Outstanding: ~31.9 million * Market Capitalization: ~$86 million * Cap Structure: Straight Common. No Debt. No Preferred. * Cash: ~$7.3 million (pro forma January 31, 2012) Stock in
Slide 6: Product Pipeline
Product Pipeline Synthetic Biologics Division Medical indication Biologic Candidate Preclinical Phase Ill Pulmonary arterial | Synthetic DNA-based hypertension therapy Funded Clinical Programs Medical indication Drug Candidate Relapses in multiple sclerosis Trimesta™ pater Trimesta™ Fibromyalgia eff
Slide 7: Adeona Team
Adeona Team Management Collaborators * Jeffery L. Riley, CEO * PAH — Synthetic DNA-Based Therapy Pfizer, Nichols Institute (Quest), Intrexon Corporation SmithKline Beecham, QIC ; - - * Multiple Sclerosis — Trimesta™ * John Monahan, Sr. VP R&D Rhonda Voskuhl, MD, Professor, UCLA Avigen, Somatix, Trit
Slide 8: Gene Medicine 2.0
Gene Medicine 2.0 * 1990-2000: High hopes and promise * 2000-2004: Set-backs and disappointments — Safety concerns — Inability to control in vivo expression and protein production — Immunogenicity — Inability to optimize long-term expression * 2004-2011: Corporate abandonment of gene medicine * Gene
Slide 9: Intrexon Corporation
Intrexon Corporation San Francisco, CA Industrial Products Division Protein Production Division San Diego, CA Cell Engineering Unit oie, Fierce FierceBiotech Germantown, MD Human Therapeutics and Animal Science Divisions Blacksburg, VA UltraVector" poe Research Triangle Park, NC @ AsBio Division @ C
Slide 10: Synthetic Biologic Steps
Synthetic Biologic Steps Human Genome DNA Synthesizer —_ et Synthetic DNA Modules. Synthetic DNA Transgene DNA-based Therapeutic S SYNTHEMIG
Slide 11: Insertion of Synthetic DNA
Insertion of Synthetic DNA Image courtesy of Intrexon Corporation ), SYNTHETIC
Slide 12: Insertion of Synthetic DNA
Insertion of Synthetic DNA SyntheticDNA Segment of Virus DNA exchanged ee ) SYNTHETIC
Slide 13: Insertion of Synthetic DNA
Insertion of Synthetic DNA Synthetic DNA inserted image courtesy fntrexon Corporation 5) SYNTHETIC
Slide 14: Insertion of Synthetic DNA
Insertion of Synthetic DNA image courtesy of ntrexon Corporation 5) SYNTHETIC
Slide 15: Control of Gene Expression with RheoSwitch Therapeutic System
Control of Gene Expression with RheoSwitch Therapeutic System® Viral vector € youre RheoSwitch® Proteins RTS-GO! ‘OFF’ Nucleus cytoplasm Image courtesy of Intrexon Corporation. SYNTHETIC RheoSwitch® is a registered trademark of Intrexon Corporation.
Slide 16: Control of Gene Expression with RheoSwitch Therapeutic System
Control of Gene Expression with RheoSwitch Therapeutic System® RheoSwitch® Activator Ligand (AL) orally administered Nucleus Cytoplasm Image courtesy of Intrexon Corporation SYNTHETIC
Slide 17: Control of Gene Expression with RheoSwitch Therapeutic System
Control of Gene Expression with RheoSwitch Therapeutic System® Nucleus Cytoplasm Oral Activator Ligand (AL) Image courtesy of Intrexon Corporation >) SYNTHETIC
Slide 18: Control of Gene Expression with RheoSwitch Therapeutic System
Control of Gene Expression with RheoSwitch Therapeutic System® Cytoplasm Image courtesy of Intrexon Corporation >) SYNTHETIC
Slide 19: Control of Gene Expression with RheoSwitch Therapeutic System
Control of Gene Expression with RheoSwitch Therapeutic System® Translation Proteins Cytoplasm Image courtesy of Intrexon Corporation ) SYNTHETIC
Slide 20: Control of Gene Expression with RheoSwitch Therapeutic System
Control of Gene Expression with RheoSwitch Therapeutic System® RTS-GOl ‘on Nucleus Cytoplasm Image courtesy of Intrexon Corporation >) SYNTHETIC
Slide 21: RheoSwitch Technology
RheoSwitch® Technology On-Off switch with dose adjustment control ON-OFF | 40 | \ seri0se cette | \ InlL-42 expression (ngln! medium/6hes) 8 8 | ‘Time after viral transduction (hours) RheoSwitch® control design advantages include improved safety, reduced side effects and enhanced efficacy image cour
Slide 22: Pulmonary Arterial Hypertension (PAH)
Pulmonary Arterial Hypertension (PAH) Synthetic DNA-Based Therapy
Slide 23: Anatomy of PAH
Anatomy of PAH Heart and Lungs of Normal Patient Heart and Lungs of PAH Patient Constsiction of Pul Arteries Pulmonary (ota Lungs: Normal Artery Flow Constricted Artery Flow
Slide 24: PAH Market
PAH Market * The global PAH market is estimated to reach $3.6 billiont by 2015 * Current approved approaches for PAH include - Prostacyclin analogs — ET-1 receptor antagonists — PDES inhibitors * Prostacyclin analogs are most well established but have significant dose limiting systemic side effects
Slide 25: PAH Product Positioning and Clinical Need
PAH Product Positioning and Clinical Need Prostaglandin synthase (PGIS) is the final committed enzyme in the metabolic pathway of prostacyclin production PGIS is deficient in PAH pulmonary arteries Our intended synthetic DNA-based approach - Deliver PGIS gene to pulmonary arteries = Single procedure
Slide 26: Funded Clinical Programs
Funded Clinical Programs
Slide 27: Multiple Sclerosis: Trimesta
Multiple Sclerosis Trimesta™
Slide 28: Historical Efficacy of Estriol
Historical Efficacy of Estriol The “Pregnancy Hormone” — Linked to fetal immune privilege * Female hormone produced by placenta during pregnancy + Approved in Europe and Asia for 40 years to treat symptoms of menopause, but never approved in the U.S. for any indication * PRegnancy In Multiple Sclero
Slide 29: Multiple Sclerosis Patient Population and Market Size
Multiple Sclerosis Patient population and market size * According to the National Multiple Sclerosis Society — 400,000 patients in U.S. afflicted with MS — 200 people per week are diagnosed with MS in the U.S. — 2.5 million worldwide are affected by MS — ~85% of MS patients are originally diagnosed
Slide 30: Relapses in Multiple Sclerosis
Relapses in Multiple Sclerosis Drug candidate — Trimesta (estriol) * Trimesta developed by Rhonda Voskuhl, M.D., Professor, UCLA Department of Neurology — Jack H. Skirball Chair for MS Research and Director, UCLA MS Program * Completed an investigator-initiated, 10-patient, 22-month, single- agent,
Slide 31: Trimesta: Relapses in MS Trial
Trimesta Drug development — Relapses in MS trial * Randomized, double-blind, placebo-controlled Phase II clinical trial+ of female patients with relapsing-remitting MS underway at 15 U.S. centers * Comparing relapse rates over 2 years between 8 mg of Trimesta taken daily versus matching placebo with
Slide 32: Multiple Sclerosis Patient Population and Market Size
Multiple Sclerosis Patient population and market size * According to the National Multiple Sclerosis Society - 400,000 patients in U.S. afflicted with MS — 200 people per week are diagnosed with MS in the U.S. - 2.5 million worldwide are affected by MS — ~50% of MS patients will be affected by cogni
Slide 33: Cognitive Dysfunction in Multiple Sclerosis
Cognitive Dysfunction in Multiple Sclerosis Drug candidate — Trimesta (estriol) * Completed investigator-initiated clinical trial evaluating Trimesta - Demonstrated a 14% improvement in Paced Auditory Serial Addition Test (PASAT) cognitive testing scores (p = 0.04) in relapsing-remitting MS patients
Slide 34: Trimesta: Cognitive Dysfunction MS Trial
Trimesta Drug development — Cognitive dysfunction MS trial * Initiated second Phase II Trimesta clinical trial’ to be led by Dr. Voskuhl in MS patients (January 2012) - Randomized, double-blind, placebo-controlled clinical trial of female MS patients with cognitive dysfunction at UCLA - Enrolling 64
Slide 35: Fibromyalgia: Effirma
Fibromyalgia Effirma™
Slide 36: Fibromyalgia Market Opportunity
Fibromyalgia Market Opportunity Commercial potential for Effirma (flupirtine) * Chronic and debilitating condition characterized by widespread pain and stiffness throughout body, accompanied by severe fatigue, insomnia and mood symptoms — Affects 3-6% of the population worldwide, including an estima
Slide 37: Corporate Partnership with Meda AB
Corporate Partnership with Meda AB Effirma for fibromyalgia attracted large pharma collaboration * $17.5 million corporate partnership with Meda announced in May 2010 — Meda assumes all future development costs — $2.5 million upfront payment received — Potential to receive up to $15 million in futur
Slide 38: Amyotrophic Lateral Sclerosis (ALS): AEN-100
Amyotrophic Lateral Sclerosis (ALS) AEN-100
Slide 39: ALS or Lou Gehrig's Disease
ALS or Lou Gehrig’s Disease Devastating neurodegenerative disease * Affects the motor nerve cells in the brain and the spinal cord of both males and females usually over age 50 * Progressive loss of muscle strength and coordination eventually makes it impossible to get out of a chair, swallow or bre
Slide 40: Potential Role for Zinc in Pathogenesis of ALS
Potential Role for Zinc in Pathogenesis of ALS * Zinc is implicated in - All three forms of ALS mneane Soper — Multiple established disease pathways in ALS — Zine binds to BMA * Net result —= _ i inci ine sequestered Sequestration of zinc in the Tine sean central nervous system (CNS) * Therapeutic a
Slide 41: Zinc Therapy in ALS
Zinc Therapy in ALS Positive top-line results * PNA Center for Neurological Research (PNA) conducted a Phase I/II open label safety study? of oral high dose zinc therapy in ALS patients * 8 of the 10 patients enrolled in the study were dosed with 90 mg of zinc per day (and 2 mg of copper every other
Slide 42: ALS or Lou Gehrig's Disease: AEN-100 Development
ALS or Lou Gehrig’s Disease Drug development — AEN-100 (zinc acetate) * Planned double-blind, placebo-controlled Phase II/IlI clinical trial in ALS patients - Enroll at least 60 patients at 6 clinical sites Randomized into treatment or matching placebo arms All patients remain on RILUTEK® (riluzole)
Slide 43: Intellectual Property
Intellectual Property * Intrexon Corporation intellectual property — RheoSwitch Therapeutic System® and vector constructs * Synthetic Biologics intellectual property - Select issued patents = Trimesta (estriol) © USS. Patent 6,936,599, issued August 30, 2005 "= Effirma (flupirtine) © U.S. Patent 6,6
Slide 44: Recent Milestones
Recent Milestones Advancing synthetic biologics and funded clinical programs Y Worldwide exclusive channel collaboration with Intrexon Corporation for the development and commercialization of a synthetic DNA-based therapy to treat PAH v Proposed corporate name change to Synthetic Biologics, Inc., to
Slide 45: Synthetic Biologics
SYNTHETIC LoGitsie¢es Adeona Pharmaceuticals, Inc. (NYSE Amex: AEN) d/b/a Synthetic Biologics, Inc = © SYNTHETIC Pending Name and Ticker Symbol Change
Adeona Pharmaceuticals — Investor Presentation (February 2012) on 6ix