Precigen, Inc. · NASDAQ:PGEN
Precigen — Intrexon — J.P. Morgan Healthcare Conference 2018
24 slides from Precigen's conference presentation, Jan 10, 2018
Slide 1: J.P. Morgan Healthcare Conference January 10, 2018
J.P. Morgan Healthcare Conference January 10, 2018 Exhibit 99.1
Slide 2: Forward-Looking Statements
as Statements Some of thi pursuant to the ‘sate arbor pros ofthe? Private Securities litigation Reform Act of 1995. These forward-looking statements are based upon generally relate to Intrexon's plans, objectives and expectations for the development of Intrexon's business though wnanagement believes
Slide 3: Enabling Targeted and Controlled Gene-Based Therapies
Construction of powerful gene expression programs drives efficacy Control of gene expression and regulation drives safety Delivery through cellular and non-viral based approaches increases applicability Enabling Targeted and Controlled Gene-Based Therapies Combinations of these three key variables e
Slide 4: Gene Expression in the DNA of Intrexon
Gene Expression in the DNA of Intrexon Control of Gene Expression L. lactis Insects Methanotrophs Plants Finfish Livestock Immune Cells Human
Slide 5: Intrexon's Cell and Gene Therapy is Now Precigen
Intrexon’s Cell and Gene Therapy is Now Precigen Precigen will utilize Intrexon’s leading platform for programming & engineering genetic code to build next-gen therapeutic products faster and cost effectively Management team in place Broad pipeline of internal & partnered programs Focus on expansion
Slide 6: CAR-T Therapy: Gene Delivery's Greatest Hit?
CAR-T Therapy: Gene Delivery’s Greatest Hit? Current Challenges Lack of control leads to: Cytokine Release Syndrome Tumor Lysis Syndrome Neurotoxicity Commercial application challenges: High cost of viral-based gene integration Lack of persistence limits targets To date therapy has been limited to c
Slide 7: The Point-Of-Care (POC) Revolution
The Point-Of-Care (POC) Revolution CAR-T therapies seem likely to be transformative approaches to the treatment of lymphoma, leukemia and most recently multiple myeloma. However, barriers to maximal adoption are emerging based on both high costs and the delays in treatment that result from a require
Slide 8: POC to Scale Genetically Modified T Cells with Distributed Manufacturing
POC to Scale Genetically Modified T Cells with Distributed Manufacturing Centralized Manufacturing Distributed Manufacturing Centralized production is necessary due to the use of viral-based gene transfer which is tied to the need for ex vivo numeric expansion. Distributed production, e.g., in blood
Slide 9: POC Cells Potentiate a New Business Model
POC Cells Potentiate a New Business Model No centralized manufacturing allows for a unique business model Partner with major medical centers. Hospitals hold exclusive regional license to Intrexon technology Intrexon receives per patient and yearly licensing fees and double digit royalties Expected s
Slide 10: POC Approach – Further Benefits
POC Approach – Further Benefits In hematological diseases durable complete responses (CRs) are the goal – likely representing cures Opportunity - currently 25% to 50% of patients get early CRs only to relapse in a few months Better - mbIL-15 is a strong candidate to reduce cell failure and raise the
Slide 11: Xogenex – Exploiting Precigen's Gene Expression Franchise
Xogenex – Exploiting Precigen’s Gene Expression Franchise Heart Disease is #1 cause of death in the US. 610,000 People in the US die of heart disease each year 1 in every 6 health care dollars is spent on cardiovascular disease $317 billion Health care costs and lost productivity in 2011 Xogenex* ha
Slide 12: Multigene Approach Utilizing Established Heart Failure Effectors
Multigene Approach Utilizing Established Heart Failure Effectors Favorable Pre-clinical Data: Demonstrated reversal of established ADM cardiomyopathy in rat model after a single pXoX treatment (** P<0.001) Mitotic marker confirmed regeneration of cardiomyocytes GLP toxicology study in pig models dem
Slide 13: Fibroblast-based Therapies: ERTs from Living Platforms
Fibroblast-based Therapies: ERTs from Living Platforms Developing transformational FCX-007 and FCX-013 autologous cell-based therapies in collaboration with Fibrocell Science, Inc. Synthetic Biology Atop Harnessed Biology. FCX-007 targets Recessive Dystrophic Epidermolysis Bullosa (RDEB) through COL
Slide 14: Snapshot of Precigen's Portfolio
Infectious diseases ImmunOncology Precigen Emerging Therapies Autoimmunity Snapshot of Precigen’s Portfolio Indication Gene/Cell Therapy Patient # Phase Recurrent Glioblastoma Ad-RTS-IL12 >10,000* Phase 3** Recessive Dystrophic EB FCX-007 2,500 Phase 1/2 Adv. Lymphoid Malignancies Sleeping Beauty CD
Slide 15: ActoBiotics® - Controlled Expression in a Human Commensal
ActoBiotics® - Controlled Expression in a Human Commensal L. lactis essential gene removed Therapeutic gene inserted Construction strategy allows for minimum genetic modifications In-house established engineering process allows for continual creation of new drug candidates Host is engineered for env
Slide 16: Delivery of ActoBiotics® Therapeutics
Delivery of ActoBiotics® Therapeutics 1 Genetically engineered bacterium freeze dried and inserted into enteric coated capsule 2 Capsule opens and releases the bacterium directly at the disease target 3 Bacterium releases the therapeutic protein locally at target site (e.g. in GI tract) ActoBiotics®
Slide 17: ActoBiotics® Stand Out Compared to Existing Treatments
ActoBiotics® Stand Out Compared to Existing Treatments ActoBiotics® Low cost of goods a.o. because purification is not required Can be delivered orally in a capsule or in a topical solution No known toxicity or immunogenicity Targeted delivery; optimal tissue availability Simultaneous delivery of mu
Slide 18: Non-Healthcare Programs Enjoy High Technical Synergies
Non-Healthcare Programs Enjoy High Technical Synergies
Slide 19: Methanotrophs: Not Your Grandmother's Expression System
Methanotrophs: Not Your Grandmother’s Expression System CH3OH Methanol Methane oxidization enzyme CH2O Formaldehyde Methanol dehydrogenation enzyme C6H13O9P fructose-6-phosphate C3H4O3 Pyruvate CxHxOx Feedstock Product Methanotroph cell CH4 Methane Intrexon’s Genetic Programming Intrexon scientists
Slide 20: Sizable Market Opportunity with Current MBP Pipeline
Sizable Market Opportunity with Current MBP Pipeline Market Size: c.$80bn ISOBUTANOL Market Size: c.$22bn 2,3 BDO (BUTADIENE) Market Size: c.$3bn 1,4 BDO Market Size: c.$7bn ISOBUTYRALDEHYDE (MMA) ISOPRENE MBP products actively under development represent a total addressable market that exceeds $100
Slide 21: Yield and Feedstock Position MBP for Success
Feedstock Pricing Advantage# Superior Yield Potential* Yield and Feedstock Position MBP for Success Products Methanotroph Yeast 2,3 BDO 93% 50% Isobutanol 77% 41% 1,4 BDO 93% 50% Farnesene 71% 25% 2015 2016 2017 *Yield based on weight/weight basis; # Pricing based on Henry hub natural gas and No. 11
Slide 22: Arctic® Apples – Simple Science
Current approaches using chemicals or anti-oxidants to stop browning impact flavor and cost of sliced apples 40% of apples are currently wasted, much from superficial bruising / browning Arctic® Apples – Simple Science Via RNAi technology, we reduced the levels of polyphenol oxidase (PPO), the enzym
Slide 23: Next Generation: Florian™ Controlled Gene Expression in Plants
Next Generation: Florian™ Controlled Gene Expression in Plants Non- or Delayed flowering with restorer gene controlled by Florian™ Ligand spray No Ligand Age at flowering (days) Biomass (Number of leaves at flowering) Seed Production (controlled flowering) Commercial (higher biomass) Florian™ plants
Slide 24: J.P. Morgan Healthcare Conference January 10, 2018
J.P. Morgan Healthcare Conference January 10, 2018