Precigen, Inc. · NASDAQ:PGEN
Precigen — Intrexon — JP Morgan Healthcare Conference 2017
23 slides from Precigen's conference presentation, Jan 11, 2017
Slide 1: JP Morgan Healthcare Conference – January 11th, 2017
Geno Germano, President JP Morgan Healthcare Conference – January 11th, 2017 Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements Safe Harbor Statement Some of the statements made in this presentation are forward-looking statements that involve a number of risks and uncertainties and are made pursuant to the Safe harbor Provisions of the Private Securities Litigation Reform Act of 1995. These forward
Slide 3: Leadership in Engineering Biology
Leadership in Engineering Biology FIRST-IN-CLASS ACHIEVEMENTS RheoSwitch®: 1st clinically validated transcriptional gene switch Sleeping Beauty: 1st in human CAR-T trials with this non-viral platform ExeGen® LDLR: 1st engineered miniswine research model cleared by FDA for commercial use AquAdvantage
Slide 4: Focus on Healthcare
Focus on Healthcare Intrexon’s approach is to engineer cells and microbes with gene programs to enable targeted delivery of potent bio-therapeutics in a cost-effective and controlled manner Design – UltraVector® Platform Control – RheoSwitch® technology and other platforms for gene expression and re
Slide 5: Controlled Expression of Therapeutic Effectors
Controlled Expression of Therapeutic Effectors Source: Phase 1 Study of intratumoral delivery of Ad-RTS-hIL-12 + oral veledimex is well tolerated and suggests survival benefit in recurrent high-grade glioma. 2016 Society for Neuro-Oncology Annual Scientific Meeting RHEOSWITCH THERAPEUTIC SYSTEM® Dat
Slide 6: Promising Overall Survival Data in Recurrent GBM Phase I Trial
Promising Overall Survival Data in Recurrent GBM Phase I Trial with Ad-RTS-hIL-12 + veledimex in subjects with recurrent glioblastoma (GBM) Based on tolerability and survival benefit (median OS=12.7 months, n=15), 20 mg was selected for an expansion cohort and we are following patients’ overall surv
Slide 7: Advancing Robust CAR-T Product Portfolio
Advancing Robust CAR-T Product Portfolio 2nd Generation CD19 Sleeping Beauty: Phase I trial underway. One ongoing CR (currently 90 days) in multiple-relapsed ALL patient after lymphodepleting chemotherapy. 3rd Generation CD19 Sleeping Beauty: Single low-dose of mbIL15-CAR-T cells infused less than 2
Slide 8: Sleeping Beauty with RTS Control of mbIL-15
Sleeping Beauty with RTS® Control of mbIL-15 Membrane bound IL15 (mbIL-15) expressed under RTS® control via Sleeping Beauty Oncology Veledimex ON Veledimex OFF CAR mbIL-15 Fold Change in mbIL-15 mRNA over CAR only control - Veledimex + Veledimex - Veledimex + Veledimex CAR 8 7 CAR mbIL-15 CAR 7 mbIL
Slide 9: Targeting Solid Tumors with Sleeping Beauty
Targeting Solid Tumors with Sleeping Beauty Oncology Intrexon and ZIOPHARM sign CRADA with the National Cancer Institute (NCI) Utilizing Sleeping Beauty System to Generate T cells Targeting Neoantigens – January 2017 Mol Ther. 2016 Jun;24(6):1078-89.
Slide 10: Transformational Fibroblast Gene Therapies
Transformational Fibroblast Gene Therapies Gene therapies centered on transformational autologous cell-based therapies for skin and connective tissue diseases developed in collaboration with Fibrocell Science, Inc. FCX-007 – Targets Recessive Dystrophic Epidermolysis Bullosa (RDEB) Initiated Phase I
Slide 11: Treating Underlying Pathology of RDEB
Treating Underlying Pathology of RDEB RDEB Patient’s Non-Transduced Fibroblasts RDEB patients lack COL7A gene that plays critical role forming anchoring fibrils & providing integral stability to skin In vitro studies show production of functional trimeric structure COL7A which RDEB patients lack cap
Slide 12: Xogenex: Multigene Approach to Cardiac Disease
Xogenex: Multigene Approach to Cardiac Disease Promoter Linker ORI Linker UTR CARDIO Gene 1 CARDIO Gene 2 CARDIO Gene 3 pXoX plasmid Cardiac Disease Xogenex’s pXoX plasmid expresses proteins from three cardiac effector genes involved in heart failure driven by a single promoter Angiogenesis Cell Hom
Slide 13: Genes Working Synergistically For Favorable Effect
Genes Working Synergistically For Favorable Effect Cardiac Disease Dilated Cardiomyopathy Patient iPSC-CM Healthy iPSC-CM Dilated Cardiomyopathy Patient iPSC-CM Healthy iPSC-CM Control Gene 1 Control Gene 1 Gene 1 & 2 Gene 1 & 2 pXoX pXoX Proprietary pXoX platform has demonstrated promising in vitro
Slide 14: Heart Failure Represents Large Unmet Clinical Need
Heart Failure Represents Large Unmet Clinical Need Cardiac Disease 825,000 new cases annually Disease costs US approximately $32 billion per year Experienced Scientific Advisory Board in retrograde biologics delivery Positive meetings to date with FDA Anticipate IND Filing in 2017
Slide 15: Ophthalmology: Wet AMD RTS Controlled Gene Therapy Approach
Ophthalmology Wet AMD: RTS® Controlled Gene Therapy Approach . Pre-clinical in vivo studies with lead therapeutic INTREXON/SUN PHARMACEUTICALS JOINT VENTURE candidate and RTS" activator ligand * Developing treatments for blinding ocular diseases; ‘90804 first target is Wet Age-related Macular e0E+08
Slide 16: ActoBiotics: Next-generation Oral Biotherapeutics
ActoBiotics®: Next-generation Oral Biotherapeutics 3 6 I kDa Trefoil Factor 1 Interleukin-10 Human Proinsulin Human Proinsulin + hIL10 Peptide ‘X’ AG014 Reference Control Bioactivity 6 14 I kDa 17 28 I kDa I control I PINS Anti-TNF-alpha Collaborations established for diabetes (T1D/T2D), oral mucosi
Slide 17: Upcoming Phase II Trial for Oral Mucositis
Upcoming Phase II Trial for Oral Mucositis 30% full responders in AG013-treated group AG013 reduces cumulative days with ulcerative mucositis versus placebo by ~35% Source: Phase 1b, multicenter, single blinded, placebo-controlled, sequential dose escalation study to assess the safety and tolerabili
Slide 18: Ongoing Expansion of Pipeline and Clinical Programs
Ongoing Expansion of Pipeline and Clinical Programs Neuropathic Pain & Cancer: Gene therapy, Exosomes Respiratory Infection: Chronic Rhinosinusitis Metabolic & GI Disorders: Type 2 Diabetes, Oral Mucositis, PKU Cartilage Repair: Off-The-Shelf Cells Cardiac Diseases: Heart Failure Autoimmune & Inflam
Slide 19: Collaborative Business Model
Collaborative Business Model Primary objective is to leverage Intrexon technology to create a broad range of successful products which, in turn, will result in a vast portfolio of significant backend economics in commercialized goods across multiple industries Collaborator Exclusive Commercial Right
Slide 20: Portfolio Expansion & Maturation
Portfolio Expansion & Maturation September 2016 Over 30 September 2016 $391 Million September 2016 $176 Million 1 January 2011 $17.5MM January 2011 $0 January 2011 Collaborations TAF and Received Milestones Cost Recovery Portfolio of Backend Economics Future Milestones & Royalties
Slide 21: Upcoming Events in Health Sector
Upcoming Events in Health Sector Oncology: Collaborator ZIOPHARM meeting with FDA in early Q1 regarding Ad-RTS-IL-12 Phase I trial for recurrent glioblastoma (GBM) ZIOPHARM anticipates multiple INDs and trials in 1H 2017: (1) CD33 CAR for refractory AML (2) off-the-shelf primary NK cells for AML (3)
Slide 22: Summary
Over 30 collaborations in place capitalizing on our leadership position in the engineering of biology Strong cash position with $280.7 million in cash, cash equivalents, and short and long-term investments, and equity securities and preferred stock valued at $163 million Developing high value bio-so
Slide 23: Appendix - Health Pipeline
Appendix - Health Pipeline* Field / Collaborators Indications Pre-Clinical Phase I Phase II Oncology (ZIOPHARM, Merck Serono, National Cancer Institute, Exotech Bio) Ad-RTS-IL-12: Breast cancer Malignant Glioma CAR/Cytokine Products: B-Cell Malignancies Myeloid Malignancies RTS-Controlled T Cell Sol