Phio Pharmaceuticals Corp. · NASDAQ:PHIO
Phio Pharmaceuticals — RXi Pharmaceuticals — Investor Presentation (March 2013)
38 slides from Phio Pharmaceuticals's investor presentation, Mar 7, 2013
Slide 1: RXi Pharmaceuticals Corporation – Next Generation in RNAi
Exhibit 99.1 RXi Pharmaceuticals Corporation March 2013 OTC: RXII Next Generation in RNAi ®
Slide 2: Forward Looking Statements
Forward Looking “expects,” “Company”) associated estimates. presentation Company circumstances periodically The therewith), “indicates,” due current that inclusion toStatements occur risks beliefs the makes “will,” of and after expected forward-looking and uncertainties This with the “intends,” expe
Slide 3: Corporate Highlights
Corporate Experienced with prevention/revision an antisense Highlights scientific oligonucleotide (3)(1) and RXi RXi development has is broad a correlated leader development team in development with (2) RXI-109, improved opportunities of advanced RXi’s scars in based lead well-controlled RNAi-based
Slide 4: Corporate History
Corporate and ophthalmology, our lead History compound, neurology Spin-off RXI-109 and from liver Galena RXifibrosis received Biopharma, supported FDAInc. clearance by as research a public, to begin grants independent clinical and collaborative trials company with RXI-109 projects in April Located 2
Slide 5: RXi's Unique RNAi Platform
RXi’s Unique RNAi Platform Property of RXi Pharmaceuticals 5
Slide 6: Broad Intellectual Property Position in RNAi
Broad Intellectual rxRNAori development Modified todsRNA increase of RNAi-based Property Kreutzer-Limmer resistance Position drugs to nucleases (potency, in sd-rxRNA RNAiand selectivity/specificity, Compounds “self-delivering” to preventand off^target Delivery compounds half-life) effects, 1 3 9 rxR
Slide 7: sd-rxRNA: Combining Features of RNAi and Antisense
sd-rxRNA Potent, Structural long-lasting diversity Combines=activity Features novel intellectual Medicinal of RNAiChemistry property and Antisense Combining Improved Technologies cell many uptake positives sd-rxRNA and PK/PD of RNAi therapeutic O&sd-rxRNA antisense, compounds Single whilewith compou
Slide 8: sd-rxRNA: Robust Delivery and Cellular Uptake
sd-rxRNA: Liver vivo upon Spinal local Robust cord and Delivery Cellular systemic and Uptake administration silencing in vitro demonstrated and Alveolar in vivomacrophages in Hepatocytes many different primary cell mouse types Human, ARPE-19Primate, retinal Rat, pigment Mouse, epithelium Adherent, M
Slide 9: Area of Focus: Dermal Scarring
Selection ofantisense) (Excaliard Area of Focus: Large Dermal market in Scarring scar prevention/revision Attractive Therapeutic 42.3 million Opportunity surgical Unmet procedures need with perlimited year incompetition the U.S.* *US for anti-scarring truly effective market therapies – The NoNemetz
Slide 10: RXi's Lead Clinical Product Candidate: RXI-109
RXi’s Lead Clinical Product Candidate: RXI-109
Slide 11: RXI-109 Selection Rationale and Overview
improvedhas Preclinical RXI-109 Selection scarring data a clear demonstrate (vs. Rationale development placebo) potent, & Overview inpath 3,selective, well-controlled withNumerous early dose-dependent efficacy Phase studies end 2 implicate trials points and long-lasting byand another Connective ente
Slide 12: The Potential for an Effective Anti-Scarring Agent Is Large
The Potential for an Effective Anti- Scarring Agent Is Large Scarring Agent Is Large Scarring Agent Is Large US anti-scarring market – The Nemetz Group – December 2009
Slide 13: Connective Tissue Growth Factor: A Central Factor in the Pathway to Fibrosis
Connective Pathologic Dermal Anti-Scarring Effects Tissue Pulmonary Growth Property Factor Fibrosis of A RXI Central Acute Pharmaceuticals Factor SpinalinInjury the13Pathway Restenosis to Fibrosis Ocular RXI-109 Scarring Liver Connective Fibrosis Tissue Collagen Growth Deposition Factor Central Adhe
Slide 14: CTGF-Targeting Antisense Oligonucleotide Study Results
Excaliard’s surgical significant assessment necessarily incisions decrease indicative scales (Now in Pfizer’s) at in the both of scarring abdominal results 12CTGF-Targeting andwith with 24pannus weeks EXC RXI-109 001 demonstrated Physician Antisense in all 3Assessment scar Oligonucleotide a assessme
Slide 15: Positive Data from Phase 2 Studies of EXC 001
Positivewith Significant results Excaliard Improvement RXI-109 Data from in the 3 Phase Appearance 2 Studies of Scars of EXC from 001 Revision * Significant SurgeryImprovement Significant in Improvement the Appearance in theofAppearance Scars from of Revision Scars from Surgery Revision Significant
Slide 16: RXI-109 Efficiently Silences CTGF in vitro and in vivo
CTGF RXI-109 Phosphate ** *** Silencing Concentration Buffered in vitro Saline (mM) A549 (Vehicle mRNA cells Control) were levels treated were NTCwith quantified = Non-Targeting RXI-109 byand QPCRNTC Control onPassive Day sd-rxRNA 8, uptake normalized RXI-109 48 hours to the NTC EC50 housekeeping NT
Slide 17: CTGF Silencing Does Not Delay Early Wound Healing
CTGF Silencing Does Not Delay, and May Enhance, Early Wound Healing in a Rodent Model (CHART) (CHART) (CHART) ** * * *
Slide 18: RXI-109 Phase 1 Clinical Trials
RXI-109 Phase vehicle 1 Photographic single Phasecenter, 1 Clinical comparison randomized, Trials Study versus multi-dose 1201: vehicle Phase double-blind, Histological 1 single center, ascending comparison randomized, dose, of the within-subject scar single-dose, sites versus controlled double-blin
Slide 19: RXI-109-1201: Abdominal Incision Layout
RXI-109-1201: ‘columns’ pattern for were each Abdominal at subject. least 4Half cmIncision lateral of the sites to Layout thewere midline Wound treated ofAddresses the with abdomen. RXI^109, Column Rows half A(Right) were with placebo. spaced Column at least B(Left) 4 cm Row apart. 1 1ATreatment 1B
Slide 20: RXI-109-1201: Preliminary Blinded Histology Data
RXI-109-1201: injection of 7.5 mg Preliminary Images taken Blinded at 20X Histology magnification Data 12Full Weeks analysis Post-Treatment will provide Normal woundSkin area1A assessments 2A 1B 2B on Preliminary all subjects blinded data Trichrome staining of incision sites from one of three subjec
Slide 21: RXI-109-1201: Cohort 4 Blinded Scar Tissue Area
RXI-109-1201 from the lower (Single 2 sites on Dose) eachCohort subject4:(reported Area (µm2) as sum of Scar of area Tissue from(BLINDED) three sections A and per B site) sides Scar areArea treated (µm2) with01-18 RXI-109 01-19or01-02 placebo A B– A Blinded B A B data Average of 30% Difference betwe
Slide 22: RXI-109-1201: a-SMA and CTGF Images
RXI-109-1201:a-SMA magnification Subjectimage 01-18collected 2A Trichrome at 40XCTGF magnification a-SMA 2B andTreatment from adjacent per side sections is still blinded Smaller wound area appears to track with lower CTGF expression levels Images of CTGF and Trichrome taken at 20X
Slide 23: RXI-109-1202: Abdominal Incision Layout
RXI-109-1202: received intradermal 3 injections injection Abdominal over according a Incision given to time a predetermined Layout period. Wound The A randomization Addresses and B ‘columns’ Column pattern were A(Right) forateach least Column subject. 4 cm lateral B(Left) Half of toRow the midline s
Slide 24: RXI-109-1202: Clinical Pictures, Blinded Data
RXI-109-1202: not been brokenClinical Cohort 2Pictures Subjectof02-06 Subject in Cohort 2 (3 days after 3rd and last dose) R and L are on the right and left side of the abdomen, and are treated with either RXI-109 or Placebo—Blinded Data – Code has
Slide 25: RXI-109-1202: Clinical Pictures and CTGF mRNA Levels
RXI-109-1202: Placebo – Blinded Clinical Data –Pictures Code hasandnot CTGF beenmRNA broken Levels Cohortof 1 Subject 02-02 in Cohort 1 (3 days after 3rd and last dose) (CHART) (CHART) Right Side Left Side Right side and left side are treated with either RXI-109 or
Slide 26: RXI-109 Clinical Summary and Development Plan
RXI-109 clinical human from Phase and efficacy Summary animal 1 studies Tolerance exposure, oftoObservations be reported and intradermal safety in &to mid-2013 Clinical date injection considered Development Phase provides 2 excellent studies only Plan to 5% by Single-dose besystemic investigators st
Slide 27: Preliminary Plan for Phase 2 Program for RXI-109
Preliminary Cesarean section Plan scar for Phase revision 2 Program Bilateralfor keloid RXI-109 scar revision Two or three Studyrandomized, objectives include: double-blind, Determination within-subject of optimal controlled dose and studies schedule Potential Demonstration indications include: of s
Slide 28: Advancing the Product Pipeline
Advancing the Product Pipeline Program Ophthalmology(Retinoblastoma) CNS(ALS) Discovery strategic Preclinical interest projected Clinical Anti-Scarring(RXI-109) next steps Phase 1 PhaseOphthalmology(PVR) 2 Phase 3 Liver disease / Liver fibrosis(RXI-209) Ophthalmology(Macular Degeneration)
Slide 29: RXI-109 in Ocular Proliferative Vitreoretinopathy
RXI-109inocular Occurs potential minimal Orphan inapproximately Ophthalmology toxicity indication studies 8-10% with PVRofunmet (Proliferative patients needwho Collaboration Vitreoretinopathy) develop an with RD Preclinical Geoff Proliferative Lewis, dataPh.D. vitreoretinopathy strongly (UCSB) suppo
Slide 30: sd-rxRNA: Improved Retinal Delivery vs. Traditional RNAi Compounds
sd-rxRNA with overtDy547-labeled toxicity Conventional observed sd-after rxRNA, Placebo sd-rxRNA conventional siRNAtreatment MousesiRNA immediately to the or eye placebo post-dose sd-rxRNA: Mouse Improved at 24 hours Retinal post-dose Delivery Mouse vs. Traditional at 24 hoursRNAi post-dose Compound
Slide 31: sd-rxRNA: Extended Silencing in the Rodent Eye
sd-rxRNA: to NTCb-actin = Non-Targeting Data Extended assembled Silencing Control fromsd-rxRNA in 5 different vivo in the PPIB studies Rodent = Anti-cyclophilin to enable Eye 3 mg sufficient PPIB B sd-rxRNA or ‘n’NTC for each administered (CHART) data point ^51% by(n=5-8); intravitreal ** ^45% graph
Slide 32: Anti-SOD1 sd-rxRNA in Amyotrophic Lateral Sclerosis
Anti-SOD1 Current familial PhD. (top reimbursed cases neurologist sd-rxRNA of ALS patient Not ininthe Amyotrophic targetable care field) costswith upLateral tosmall $200,000 Sclerosis molecules; per(ALS) year no Misfolded effective ALS (Lou treatment SOD1 Gehrig’s accumulation Strong Disease) suppor
Slide 33: Preliminary Data: SOD1 Silencing in Spinal Cord
(CHART) pump SOD1 chemically in expression C57BL/6 52% optimized 83% in normal the 100% sd-rxRNA PBS (non- Preliminary group transgenic) compounds Silencing Data:mice pReduction observed = 0.077 60 µgpin of of <the SOD1 0.001 SOD1 lumbar SOD1 targeting mRNA region Silencing following orofnon-targeti
Slide 34: RNAi-Related Assets Acquired from OPKO
RNAi-Related prosecution disorders Cancer in several Assets Inflammatory to key Beterritories Acquired diseases including from OPKOUS OPKO and EUand Coverage collaborators/licensors of important biological among targets, early pioneers including: in RNAi Potential fieldindications Extensive intellec
Slide 35: Management Team
Management Therapeutics, Officer modified Assessment the Project onlyLead VP FDA-approved siRNA ofDirector for Team Inc. Pharmaceutical program to(acquired enter Geert Pharmacology leukotriene clinical Cauwenbergh, leading byDevelopment Stiefel trials to synthesis of discovery Ph.D. Laboratories, Ga
Slide 36: Governance and Scientific Advisory Board
Governance Life pharmaceutical 36 RNAi, National Research member years. Sciences, co-discovered for Academy Held Foundation a&number Inc., the Scientific andposition of abiologic wholly (ASERF) RNAi Sciences of cosmetic Leadership ofand life owned Metro Leroy Jeannette co-invented science and subsid
Slide 37: Expected 2013 Milestones
Expected 2013 Milestones Expected 2013 Milestones
Slide 38: Corporate Highlights
Corporate Experienced with prevention/revision an antisense Highlights scientific oligonucleotide (3)(1) and RXi RXi development has is broad a correlated leader development team in development with (2) RXI-109, improved opportunities of advanced RXi’s scars in based lead well-controlled RNAi-based
RXi Pharmaceuticals — Investor Presentation (March 2013) on 6ix