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THE ACTOBIOTICS ® PLATFORMdesigning living microbe-based therapeutics
engineered to treat a wide set of diseases
Corporate presentation - Pieter Rottiers, PhD, Chief Executive Officer, Director - JANUARY 2020
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Some of the statements made in this presentation are forward-looking statements.These forward-looking statements are based upon our current expectations andprojections about future events and generally relate to our plans, objectives andexpectations for the development of our business.
Although management believes that the plans and objectives reflected in or suggestedby these forward-looking statements are reasonable, all forward-looking statementsinvolve risks and uncertainties and actual future results may be materially different fromthe plans, objectives and expectations expressed in this presentation. All informationin this presentation is as of the date marked on the cover page, and ActoBioTherapeutics™ undertakes no duty to update this information unless required by law.
© 2019 ActoBio Therapeutics, Inc. All rights reserved. ActoBio Therapeutics™ is sharing the following materials for informational purposes only. Such materials do not constitutean offer to sell or the solicitation of an offer to buy any securities of ActoBio Therapeutics™. Any offer and sale of Intrexon’s securities will be made, if at all, only upon theregistration and qualification of such securities under all applicable federal and state securities laws or pursuant to an exemption from such requirements. The attachedinformation has been prepared in good faith by ActoBio Therapeutics™. However, ActoBio Therapeutics™ makes no representations or warranties as to the completeness oraccuracy of any such information. Any representations or warranties as to ActoBio Therapeutics™ shall be limited exclusively to any agreements that may be entered into byActoBio Therapeutics™ and to such representations and warranties as may arise under law upon distribution of any prospectus or similar offering document by ActoBioTherapeutics™.
TrademarksIntrexon, ActoBio Therapeutics, ActoBiotics, Powering the Bioindustrial Revolution with Better DNA, and Better DNA are trademarks of Intrexon and/or its affiliates. Other names may be trademarks of their respective owners.
FORWARD-LOOKING STATEMENTS
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ACTOBIO THERAPEUTICS™ - KEY INVESTMENT HIGHLIGHTS
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• ActoBiotics® is a fully integrated, cost-effective & unique food microbe-based delivery platform for therapeutics, with potential for superior efficacy and safety through oral or local targeted delivery
• L. lactis is designed to perform specific biological interventions
• Accelerates development and validated regulatory path for new IND candidates: <2 yrs from inception to IND achieved with AG013 for oral mucositis
• Strong R&D engine and large collection of strains, with potential for further development
PLATFORMPRODUCTPORTFOLIO
EXPERIENCE
• Straightforward and reliable cGMP manufacturing process
• IP portfolio across the technology platform:• >250 granted patents • >50 pending patent applications
• Extensive regulatory acceptance of platform and applications
• Experienced leadership team with unique expertise in bacterial engineering and strong scientific, regulatory and clinical backgrounds
• Three clinical stage programs: One inPhase IIb and two programs in Phase Ib/IIa, with other programs close to clinical stage
• Broad therapeutic application across a wide set of diseases with strong growth dynamics
Topline datafrom 2 clinical programs
expected in 2020
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ACTOBIOTICS® - AN INTEGRATED MICROBIAL DRUG PLATFORM
L. lactis essential gene removed
Therapeutic gene inserted
¹ Steidler et al., 2003, Nature Biotechnology.
• L. lactis has a long history of safe use in human nutrition
• Non-colonizing, non-human commensal species allowing for control of dosage and timing of exposure
• No known pathogenicity• Oral delivery platform demonstrated safe
for use in humans
• Construction strategy allows for minimum genetic modifications
• Engineered through chromosomal integration of single or multiple genetic elements through precise, targeted double homologous recombination
• Host is engineered for environmental containment¹, preventing accumulation of bacteria outside the body
• Engineering process established in-house to allow for continual creation of new drug candidates
• Industrial microorganism used for large scale food production
• cGMP manufacturing process established
• Following fermentation, the modified bacteria (ActoBiotics™) are freeze-dried and packaged
GENETICENGINEERING
SCALABLEMANUFACTURING
SAFETY
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ACTOBIOTICS™
CLINICAL AND
DEVELOPMENT
PIPELINE
I M M U N O T H E R A P Y
INDICATION PRECLINICALDISCOVERY PHASE I PHASE IIEST 2019 GLOBAL
ADDRESSABLE POPULATION*
PRODUCTCANDIDATE
D E S E N S I T I Z A T I O N
pre-AGFood, seasonal, perennial and venom allergy
51 K
5The ActoBiotics® platform can be easily adapted to deliver a wide range of additional therapeutic candidates.
I N B O R N E R R O R S O F M E T A B O L I S M
pre-AG Phenylketonuria
ANTICIPATEDMILESTONES
pre-AG Skin Disease
pre-AG Immuno-oncology
AG018 > 4 MChronic
Rhinosinusitis
AG020 12.3 MInflammatoryBowel Disease
AG013 850 KOral Mucositis Interim results1H 2020
T O L E R A N C E I N D U C T I O N
Type I Diabetes 132 KAG019 Interim resultsQ3 2020
Celiac Disease 1.9 MAG017 Start Phase Ib/IIa2020
*Total for US, EU, Japan. Sources on final slide.
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KEY TEAM MEMBERS
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PIETER ROTTIERS, PhD - CEO, Director• Extensive experience in drug discovery and development using the ActoBiotics platform, including over thirteen years of leadership and
managerial experience in biopharmaceuticals• Held several R&D and executive management functions at ActoGeniX and ActoBio Therapeutics, and progressed four ActoBiotics
products from discovery research into clinical trials• PhD in Biotechnology, University of Ghent, Belgium
LOTHAR STEIDLER, PhD - CTO• Leader of ActoBiotics technology, published author in Science and Nature Biotechnology, co-founder of ActoGeniX• Extensive experience of genetic engineering of L. lactis• Led and continues to lead all molecular biology at ActoGeniX and ActoBio Therapeutics.• PhD in Biotechnology, University of Ghent, Belgium
LUC VAN FRAEYENHOVEN - CFO• Finance executive with more than twenty years of experience in international finance functions• Held various senior finance positions including venture capital investor, CFO of the Ghent Volvo plant, and CFO of Volvo Europe• Commercial engineer and Master in Financial Management
STEFAN HERDINIUS, MSc, MBA - VP and Head of Strategic Drug Development• Accomplished leader with more than twenty years of experience in global early to late stage drug development• Instrumental in the development of new drugs in various therapeutic areas, including autoimmune diseases, neurology, virology and more,
at UCB Biopharma, Tibotec and Merck Sharp & Dohme• MSc in Pharmacy, Uppsala University, Sweden and MBA, Vlerick Business School, Belgium
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ACTOBIO THERAPEUTICS™: WORLD’S LEADING FOOD-MICROBE BASED VENTURE
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Well defined regulatory path
Scalable manufacturing
process
Multiple candidates ready for clinical development
Global IP Position
Experienced leadership team
Pioneering a new class of food-microbe
based genetically engineered
therapeutic agent
More than 20 years of scientific,
regulatory, and clinical experience
Designed to be safe & cost-
effective
Streamlined R&D engine allowing
continuous creation of new candidates
Strong pipeline with disease-modifying
potential and clinical stage programs
Fast path to IND &
clinic (<2yrs)
Therapeutic applications across
major diseaseswith high unmet
needs
ActoBiotics®A Unique and Superior
Delivery & Development Platform
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DELIVERY OF ACTOBIOTICS™ THERAPEUTICS
1. ActoBiotics™ freeze dried and inserted into enteric coated capsule
2. Capsule opens and releases ActoBiotics™ directly at the disease target
3. ActoBiotics™ produces therapeutic agent locally at target site (e.g. in GI tract)
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THE ACTOBIOTICS® PLATFORM COMPARED TO EXISTING TREATMENTS
IndicationTreatment withBiologicals
ROA (Biological) ActoBiotics™ ROA
Oral Mucositis Palifermin (HSCT) Injected Buccal rinse
IBD Anti-TNF Injected Oral capsule
Type 1 DiabetesInsulin (Replacement) Injected
Oral capsule (Immunotherapy)
Chronic Rhinosinusitis
Anti-IL5 / anti-IL4R InjectedNasal spray
Celiac Disease - - Oral capsule
ActoBiotics™ are as effective as injected biologicals
even at 1/1000th
the dose
32
50.000
1 1.000
ActoBiotics
Purified(topical)
TFF1 dose (ng) delivered for efficacy
ActoBiotics™
• Can be delivered in an oral capsule or in a topical solution
• No known toxicity or immunogenicity observed
• Targeted delivery to mucosa
• Simultaneous delivery of multiple proteins
Classic Protein Biologics
• Intravenous administration
• Systemic toxicity, immunogenicity observed
• Limited tissue penetration (notably in the gut)
• Single protein administration
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A unique delivery platform precisely tailored for specific disease modification
Specifically designed to target disease areas with high unmet need
Rapid development of new candidates
Targeted delivery via oral capsule, oral rinse or topical solution
Potential for agents with superior safety and efficacy
Robust and scalable manufacturing process
ACTOBIOTICS™MICROBE-BASED
BIOPHARMACEUTICALSfor expression and local
delivery of therapeutics at disease sites including the intestine, the mouth and
the nasopharynx
Feel free to visit our website www.actobio.com
the game changer
INDUSTRIEPARK 7C BUILDING D 9052 GHENT BELGIUM
PHONE +32 9 277 11 77 [email protected]
Janu
ary
20
, 20
20
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SOURCES AND REFERENCES
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Figures for total addressable population and peak sales are based on data from the following sources, for US, EU and Japan, with some assumptions applied internally.
Type 1 Diabeteshttps://www.cdc.gov/diabetes/pdfs/library/diabetesreportcard2014.pdf http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0011501 https://www.idf.org/e-library/epidemiology-research/diabetes-atlas/19-atlas-6th-edition.html https://link.springer.com/article/10.1007/s00125-006-0213-8#Tab1 https://data.worldbank.org/indicator/SP.POP.0014.TO.ZS https://data.oecd.org/pop/young-population.htm
Celiac disease http://www.cureceliacdisease.org/wp-content/uploads/FactSheet1_Overview-of-Celiac-Disease.pdf https://www.nature.com/ajg/journal/v108/n5/full/ajg201360a.html https://www.sciencedirect.com/science/article/abs/pii/S0198885998000081 http://www.thesuperallergycookbook.com/PDF/CeliacDiseaseFacts.pdf
Oral mucositishttp://oncolex.org/Head-and-Neck-cancer/Diagnoses/Oral-cavity/Background/Prognosis https://www.uptodate.com/contents/epidemiology-and-risk-factors-for-head-and-neck-cancer https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/cancer-treatment-and-survivorship-facts-and-figures/cancer-treatment-and-survivorship-facts-and-figures-2014-2015.pdf http://www.onclive.com/publications/oncology-live/2014/july-2014/study-finds-mouth-rinse-alleviates-oral-mucositis-symptoms-in-head-and-neck-cancers https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3662500/
Chronic Rhinosinusitishttps://www.cdc.gov/nchs/fastats/sinuses.htm https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4386276/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4702060/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4939220/ https://www.aaaai.org/global/latest-research-summaries/Current-JACI-Research/chronic-rhinosinusitis-US
Inflammatory Bowel Diseasehttps://www.cdc.gov/ibd/ibd-epidemiology.htm http://www.sciencedirect.com/science/article/pii/S1873994613000305 https://link.springer.com/article/10.1007/s00535-009-0057-3 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4863044/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4737871/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5127933/
Allergieshttps://web.archive.org/web/20160909210149/ http://www.niaid.nih.gov/topics/environmental-allergies/Pages/immunotherapy.aspxhttps://www.npr.org/sections/health-shots/2013/03/26/175348886/allergy-drops-under-the-tongue-may-be-fine-alternative-to-shots https://web.archive.org/web/20160909210149/http://www.niaid.nih.gov/topics/environmental-allergies/Pages/immunotherapy.aspx