Phio Pharmaceuticals Corp. · NASDAQ:PHIO
Phio Pharmaceuticals — RXi Pharmaceuticals — Investor Presentation (November 2016)
35 slides from Phio Pharmaceuticals's investor presentation, Nov 30, 2016
Slide 1: RXi Pharmaceuticals Corporation Investor Presentation
RXi Pharmaceuticals Corporation (NASDAQ:RXII) “The discovery of RNA interference (RNAi) is considered one of the greatest scientific breakthroughs since the discovery of DNA”1 Investor Presentation 1Wong, J. The Nobel for RNAi: From Petunias to Potential Cure. Oxbridge Biotech Roundtable Review; 201
Slide 2: 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” and similar expressions ar
Slide 3: Risk Factors
Risk Factors Investing in the Company involves a high degree of risk. Before investing, you should consider carefully the risks described below, together with the other information contained in the Company’s annual report 10-K. Risk factors include, but are not limited to: RXII is dependent on the s
Slide 4: Company Overview; Option to Acquire MirImmune; Pipeline; Upcoming Milestones
Company Overview Option to Acquire MirImmune RXi Pharmaceuticals’ Pipeline Upcoming Milestones
Slide 5: Company Overview: sd-rxRNA
Company Overview sd-rxRNA® RXi has developed a robust proprietary RNAi therapeutic platform that includes self-delivering RNAi (sd-rxRNA®) compounds. RNAi is a powerful molecular tool that has the ability to “silence” or down-regulate the expression of a specific gene that may be over-expressed in a
Slide 6: Company Overview (continued)
Company Overview (continued..) Selection of six lead sd-rxRNA compounds against six different extracellular and intracellular immune check points; Preclinical data that demonstrate silencing of all six targets in vitro, singly and in combinations; Efficient and long-lasting knockdown of immune check
Slide 7: Transaction: Option to Acquire MirImmune
Transaction: Option to Acquire MirImmune Dermatology (RXI-109, Samcyprone) Phase 2 Ophthalmology (RXI-109) Phase 1/2 Consumer health (RXI-231 / functional skin care): Pre-consumer testing 3 Ongoing Development Programs Targeting multiple immune checkpoints by ex-vivo treatment of therapeutic cells,
Slide 8: Terms of the Option to Acquire MirImmune
Terms of the Option to Acquire MirImmune Acquire all outstanding capital stock of MirImmune Consideration for a number of shares equal to 19.99% of the then outstanding common stock of RXi Additional potential consideration contingent on MirImmune reaching certain milestones
Slide 9: RXi Pharmaceuticals' Pipeline
Rx Discovery Preclinical Phase 1 Phase 2 RXI-109 Dermal Scarring *Samcyprone™ Warts RXI-109 Retinal Scarring RXI-109 Corneal Scarring Phase 3 sd-rxRNA / CAR T-cell Ovarian Cancer sd-rxRNA / TIL Melanoma Consumer Health Cosmetic Lead Identification Functional and Safety Testing Consumer / User Testin
Slide 10: Pipeline and News Flow
Pipeline and News Flow RXi Pharmaceuticals and MirImmune Anti-PD1 sd-rxRNA in CAR T-cells in mouse models for ovarian cancer P2 Samcyprone™ – cutaneous warts first read-out Study 1502 P2 RXI-109 (targeting CTGF) for dermal scarring (hypertrophic scarring) – preliminary read-out Study 1402 RXI-231: C
Slide 11: RXi's Proprietary sd-rxRNA Platform
RXi’s Proprietary sd-rxRNA Platform
Slide 12: RNAi Mechanism Results in Potent Gene Silencing
RNAi Mechanism Results in Potent Gene Silencing cell membrane RISC mRNA cleaved mRNA 1 2 3 4 RNAi compound (sd-rxRNA) administered sd-rxRNA enters cells sd-rxRNA loads into RNA-induced silencing complex (RISC) Target mRNA is cut & destroyed Disease protein expression is blocked sd-rxRNA RNAi is a na
Slide 13: The sd-rxRNA Advantage
The sd-rxRNA Advantage Novel, self-delivering RNAi therapeutic compounds Single compound incorporates gene silencing activity & cellular uptake Robust uptake & silencing in multiple preclinical models Demonstration of safety and clinical activity in human Best RNAi technology for enhancement of cell
Slide 14: sd-rxRNA: Robust Cellular Uptake
sd-rxRNA: Robust Cellular Uptake In vivo and in vitro T-cells ARPE-19 retinal pigment epithelium Hepatocytes primary mouse Keratinocytes human primary SH-SY5Y neuroblastoma Skin Eye Spinal cord Uptake and silencing demonstrated in many different cell types Human, Primate, Rat, Mouse, Adherent, Non-a
Slide 15: RXi's Advantages Over Competitors
RXi’s Advantages Over Competitors Proprietary Platform Delivery Efficacy / Safety Compatibility Single chemically-modified RNA compound with improved drug-like properties Potent, stable, specific Efficient cellular uptake and gene silencing Rapid lead identification and optimization (4 months) No de
Slide 16: Clinical Programs
a Clinical Programs
Slide 17: Dermal Clinical Program: RXI-109
Dermal Clinical Program: RXI-109 An sd-rxRNA in Development to Reduce Dermal Scarring Phase 2a: Current trial RXI-109-1402 3rd and 4th cohorts initiated Q4 2015 Objective: Reduce the formation of hypertrophic scars following scar revision surgery Market Potential: $1-3B Approximately 45M surgical pr
Slide 18: Dermal Clinical Program: RXI-109-1402
Dermal Clinical Program: RXI-109-1402 RXI-109 Control Ex. 1 Ex. 3 Ex. 2 Representative Photos from P2 trial RXI-109-1402 5 mg/cm Cohort at 3 Months RXI-109: sd-rxRNA in Development to Reduce Dermal Scarring RXI-109 Treatment Scores Better than Control Demonstrates efficacy of sd-rxRNA approach in a
Slide 19: Ophthalmology Clinical Program: RXI-109-1501
Ophthalmology Clinical Program: RXI-109-1501 RXI-109: sd-rxRNA in Development to Reduce Retinal Scarring The objective is to block the formation of subretinal scarring in patients with advanced wet age-related macular degeneration (AMD) Goal: to maintain vision for a longer period of time than curre
Slide 20: RXI-109: Dose-dependent Reduction of CTGF in Retina and Cornea
CTGF protein levels are reduced in a dose dependent manner 7 days following a single intravitreal injection of RXI-109 in cynomolgus monkey Retina Cornea % Positive CTGF Staining % Positive CTGF Staining Treatment Dose Treatment Dose PBS 0.1 mg 0.33 mg 1 mg * ** * * p < 0.05, ** p < 0.01 Ophthalmolo
Slide 21: Overview of Phase 2a Clinical Trial RXI-SCP-1502
Overview of Phase 2 Clinical Trial RXI-SCP-1502 Phase 2a: Subjects with up to four cutaneous warts on the hands, feet, limbs or trunk Subjects are treated with a sensitization dose on the inner arm and on one preselected wart lesion After a confirmed sensitization response, subjects will be treated
Slide 22: Treatment with DPCP
Treatment with DPCP Cutaneous Metastases of Melanoma (refractory) Cotellessa, C et al. JAAD 2001;44:73-6 >200 patients treated for several months Response rates of 67-78% Damian, DL et al. J Surg Oncol 2014, 109:308-313 50 patients with mean of 15 months treatment 46% complete clearance and 38% part
Slide 23: Consumer Health / Functional Skin Care: RXI-231
Consumer Health / Functional Skin Care RXI-231: Cosmetic Product Development Potent sd-rxRNA targeting Tyrosinase (TYR) TYR: key enzyme in synthesis of melanin In development for uneven skin tone and pigmentation
Slide 24: MirImmune Overview
a Mirlmmune Overview
Slide 25: Cell Therapy can be Improved by Pre-treatment with sd-rxRNA
Cell Therapy can be Improved by Pre-treatment with sd-rxRNA sd-rxRNA pre-treatment of cells can be used to silence one or more immuno-suppressive genes (such as PD-1 and other checkpoints). This results in fewer suppressive receptors on the immune cell surface, which boosts their ability to detect a
Slide 26: Immuno-oncology Program
Immuno-oncology Program sd-rxRNA is the best RNAi technology for enhancing cellular immuno-oncology Nearly 100% transfection efficiency… sd-rxRNA is uniquely suited for checkpoint modulation in cellular immuno-oncology therapies, such as CAR T-cells 98.6% … combined with high cell viability sd-rxRNA
Slide 27: Targeting Multiple Immunosuppression Pathways in a Single Therapeutic Entity
Targeting Multiple Immunosuppression Pathways* in a Single Therapeutic Entity * Extracellular and Intracellular Simultaneous Silencing of Multiple Checkpoint Genes in T-cells is a Major Competitive Advantage of the Technology sd-rxRNAMIX sd-rxRNAPD1 sd-rxRNATarget 1 sd-rxRNALAG3 sd-rxRNATarget 2 NTC
Slide 28: sd-rxRNA in Cell Therapy: Reduced in vivo Tumor Growth Using CAR T-cells
sd-rxRNA in Cell Therapy Reduced in vivo Tumor Growth Using CAR T-cells Treated ex vivo with PD1 targeting sd-rxRNA CAR T-cells Against Solid Tumors (at one month) PD-1 Silencing by sd-rxRNA Increases Long-Term Efficacy in vivo Meso CAR T-cells: T-cells engineered to target mesothelin, overexpressed
Slide 29: sd-rxRNA in Cell Therapy: TILs Against Melanoma
sd-rxRNA in Cell Therapy Tumor Infiltrating Lymphocytes (TILs) Against Melanoma PD1 Silencing by sd-rxRNA in TILs Enhances Killing of Autologous Tumor Cells In Vitro TIL isolated from melanoma patient TILs treated with PD1 sd-rxRNA in a clinically used Rapid Expansion Protocol (REP) Tumor cell killi
Slide 30: Management Team
a Management Team 30
Slide 31: Management Team
Management Team Dr. Geert Cauwenbergh – President and Chief Executive Officer Served as Chief Executive Officer of Barrier Therapeutics, Inc. (formerly NASDAQ:BTRX) a biopharmaceutical company that he founded in 2001 and where he also served as Chairman. Barrier, which focused on dermatology drug de
Slide 32: Management Team
Management Team Pamela Pavco, PhD – Chief Development Officer Served as the Senior Vice President of Pharmaceutical Development of Galena Biopharma, Inc. from March 2007 until April 2012. Over 20 years of research and development experience in oligonucleotides. Senior Director, R & D Project Managem
Slide 33: Capitalization
a Capitalization 33
Slide 34: Capitalization Table
Capitalization Table Description Number of Common Shares Equivalent Common Shares Outstanding 6,599,846 Options (weighted average price $26.38) 390,969 Warrants (weighted average price $5.21) 1,300,464 Total Fully Diluted Shares Outstanding 8,291,279
Slide 35: RXi Pharmaceuticals: Developing Innovative Therapeutics For a Better Life
RXi Pharmaceuticals NASDAQ: RXII Developing Innovative Therapeutics For a Better Life www.rxipharma.com
RXi Pharmaceuticals — Investor Presentation (November 2016) on 6ix