Phio Pharmaceuticals Corp. · NASDAQ:PHIO
Phio Pharmaceuticals — RXi Pharmaceuticals — 15th Annual Biotech in Europe Forum for Global Partnering and Investment Conference 2015
21 slides from Phio Pharmaceuticals's conference presentation, Sep 29, 2015
Slide 1: RXi Pharmaceuticals 15th Annual Biotech in Europe Forum
RXi Pharmaceuticals NASDAQ: RXII 15th Annual Biotech in Europe Forum for Global Partnering and Investment Conference September 29, 2015 RXi Pharmaceuticals NASDAQ: RXII 15th Annual Biotech in Europe Forum for Global Partnering and Investment Conference September 29, 2015 Exhibit 99.1 ®
Slide 2: Forward Looking Statements
Forward Looking Statements Forward Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “believes,” “anticipates,” “plans,” “expects,” “indicates,” “will,” “intends,” “potential,” “suggests”
Slide 3: Corporate Highlights
Corporate Highlights Corporate Highlights Leader in development of advanced RNAi-based therapeutics Novel, self-delivering RNAi (sd-rxRNA ® ) platform provides improved cellular uptake, safety, potency and selectivity over classic siRNA Developing innovative therapeutics in dermatology and ophthalmo
Slide 4: Leader in the Development of Advanced RNAi-based Therapeutics
Leader in the Development of Advanced RNAi-based Therapeutics Leader in the Development of Advanced RNAi-based Therapeutics The sd-rxRNA Advantage The sd-rxRNA Platform ‘Self-delivering’ therapeutic compounds with drug-like properties Single compound incorporates activity and delivery Structural div
Slide 5: Dermatology Franchise: Multiple Development Opportunities
Dermatology Franchise Dermatology Franchise Clinical Program Discovery Preclinical Multiple Development Opportunities RXI-109 (Hypertrophic Scars & Keloids) Samcyprone™ (Alopecia Areata, Warts, Cutaneous Metastases Melanoma) Anti-Collagenase (Aging, Chronic wounds) Anti-Tyrosinase (Melasma, PIH, Len
Slide 6: Dermal Scarring: RXI-109 Unmet Medical Need with Large Market Potential
Dermal Scarring: RXI-109 Unmet Medical Need with Large Market Potential Dermal Scarring: RXI-109 Unmet Medical Need with Large Market Potential Targeting Connective Tissue Growth Factor (CTGF) Unmet need with limited competition for truly effective therapies Trials initiated with RXi’s first clinica
Slide 7: RXI-109 Dermal Clinical Program
RXI-109 Dermal Clinical Program RXI-109 Dermal Clinical Program Phase 1 Phase 2a Phase 2b Phase 3 Dermatology Franchise RXI-109-1201 - Single dose Complete – n=15 RXI-109-1202 - Multi-dose Complete – n=15 RXI-109-1301 - Hypertrophic scars Enrollment and treatment complete. Three doses over a 2-week
Slide 8: Phase 2a: Scar Comparison at 3 Months
Phase 2a: Scar Comparison at 3 Months Treatment with RXI-109 Initiated Two Weeks Post Scar Revision Surgery Phase 2a: Scar Comparison at 3 Months Treatment with RXI-109 Initiated Two Weeks Post Scar Revision Surgery Pre-scar revision surgery 3 months post-scar revision surgery PLACEBO RXI-109 *White
Slide 9: Topical Immunotherapy: Samcyprone
Topical Immunotherapy: Samcyprone ™ Topical Immunotherapy: Samcyprone ™ Samcyprone ™ - RXi’s second clinical candidate A proprietary topical formulation of diphenylcyclopropenone (DPCP) DPCP is a topical immunomodulator that works by eliciting a T-cell response Efficacy in the three target indicatio
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Slide 14: Ophthalmology Franchise: Multiple Development Opportunities
Ophthalmology Franchise Ophthalmology Franchise Program Discovery Preclinical Clinical Phase 1 Phase 2 Phase 3 Multiple Development Opportunities Retinal scarring (Macular Degeneration) Corneal scarring (Topical) “Self-Delivering” (“sd”) Adaptation to acquired OPKO estate New targets
Slide 15: sd-rxRNA in Ophthalmology: Improved Delivery vs. Stabilized RNAi
sd-rxRNA: In Ophthalmology Improved Delivery vs. Stabilized RNAi sd-rxRNA chemistry is required for robust uptake to the cells of the eye No overt toxicity observed after sd-rxRNA treatment to the eye Property of RXi Pharmaceuticals sd-rxRNA Stabilized Placebo siRNA Mouse immediately post-dose Mouse
Slide 16: CTGF Protein Levels in NHP Retina and Cornea Decrease Following RXI-109
CTGF Protein Levels in the NHP Retina and Cornea Decrease Following Treatment with RXI-109 CTGF protein levels are reduced in a dose dependent manner 7-days following single intravitreal injection of RXI-109 Retina Cornea Treatment Dose Treatment Dose PBS 0.1 mg 0.33 mg 1 mg * ** * * p < 0.05, ** p
Slide 17: Research and Development Programs: New Targets for Potential Preclinical Development
Research and Development Programs New Targets for Potential Preclinical Development Collagenase (MMP1) Tyrosinase (TYR) OPKO RNAi Assets Market Potential MMP1 enzyme involved in breakdown of extracellular matrix • Potent MMP1 sd-rxRNAs identified for potential for preclinical development such as ski
Slide 18: Research and Development Programs: New Targets for Preclinical Development
Research and Development Programs New Targets for Potential Preclinical Development Research and Development Programs New Targets for Potential Preclinical Development MMP1 TYR VEGF DISCOVERY Target Selection In Progress Bioinformatics Pending Compound Synthesis Pending In vitro Screening Pending Fi
Slide 19: MMP1-Targeting sd-rxRNA Reduced Migration Rate of A549 Cells in vitro
NTC UTC MMP-26038 0 h 24 h 48 h 72 h MMP1 – Targeting sd-rxRNA Compounds Reduced Migration Rate of A549 Lung Carcinoma Cells in vitro • Scratch migration assay - A549 non-small cell lung carcinoma cell line • Cells in culture treated with MMP1 – targeting sd-rxRNA, non-targeting control (NTC), or un
Slide 20: TYR-Targeting sd-rxRNA Leads to Visible Reduction of Pigmentation in vitro
TYR – Targeting sd-rxRNA Compounds Lead to a Visible Reduction of Pigmentation in vitro TYR – Targeting sd-rxRNA Compounds Lead to a Visible Reduction of Pigmentation in vitro • MelanoDerm™ 3-dimensional epidermal culture model containing melanocytes • Fourteen day culture with TYR-targeting sd-rxRN
Slide 21: RXII: Driving Growth and Innovation
RXII: Driving Growth and Innovation RXII: Driving Growth and Innovation Research & Development Business Development Communicate preliminary read-outs for RXI-109-1402 Phase 2a for Hypertrophic Scars Q4 2015 Initiate Phase 1/2 trial in ophthalmology with RXI-109 Q4 2015 Initiate Phase 2 trial for cut