Introduction
Nathalie Moll, Secretary General
EuropaBio
Dr. Philippe De Backer, MEP
2
Company highlights: Global Bioenergies
Jean-Babtiste Barbaroux, Head of
Corporate Development
3
Direct gaseous fermentation to isobutene, a
platform molecule for drop-in chemicals and fuels
Brussels,
Sept. 19th 2012
Global Bioenergies at a glance
• Established in 2008 by Marc Delcourt, a seasoned entrepreneur in
industrial biology and Philippe Marlière, a pioneer in synthetic biology
• Located close to Paris in Genopole, the number 1 biotech cluster in France
• A team of 30 people gathering multidisciplinary skills
• International Scientific Advisory Board
• Successful IPO on Alternext mid June 2011 and subsequent capital
increase of 3M€ in June 2012.
• Collaborations with Synthos, LanzaTech,
a German car manufacturer and a Major US industrialist
5
Mission statement
Converting renewable resources into light olefins
through direct fermentation
6
Why is it interesting ?
•
Because these molecules are the key building blocks of the
petrochemical industry
•
Huge markets – wide product trees – drop in
Existing
Market (b$)
Potential
Market (b$)
Main applications
Ethylene
144
Polyethylene (60%)
Propylene
88
Polypropylene (65%)
37-74
Co-monomers in various plastics
Linear butenes
Isobutene
29
Butadiene
14,6
Isoprene
2
>400
Tires, organic galss, pET, fuels
Tires, nylon, coating polymers
10
Tires, adhesives
7
Diverse feedstocks…
Sugar
Starch
170mT
2.070mT
Ligno-cellulose
To be industrialised in the near
future
CO (+H2)
Potential x10.000mT
Corn
Sugar beet
Steel mills
Forestry waste
Straw
Wheat
Municipal waste
Sugar cane
Energy crops
Rye, rice, barley,
Potatoes….
Biomass (all types)
8
…transformed into isobutene…

high purity
Isobutene

Low quality
Glucose
Genetically engineered
micro-organism
Reduced costs and improved environmental impact due to:
- No product toxicity, no feedback-inhibition no back-flux,
- No distillation nor liquid to liquid extraction
9
Issues with classical approaches yielding a liquid
intermediate molecule as the fermentation product
• Fermentation product = liquid (isobutanol, ethanol, butanediol..)
 accumulation in fermenter
 toxic for micro-organism at certain concentration
(e.g. 4% for iso-butanol)
 requires costly distillation or phase separation
• Fermentation product needs to be chemically transformed (e.g. through
dehydration) into final product
 cost in terms of OPEX and CAPEX
 possible source of isomer impurity generation
DIRECT fermentation to the final, GASEOUS end-product avoids those issues
10
Isobutene industrialization schedule
1
August 2012 : - more than half of the development has been accomplished
1
- start of industrialization: laboratory pilot, subsequently industrial pilot
11
The profitability question
• “ At what price for the oil barrel is your process going to be profitable ?”
• With current molasses prices and targeted process parameters, cost for
GBE bio-isobutene (including Capex depreciation) would be below
1500$/t compared to 1750$/t for fossil-based high-purity Isobutene.
• Cost structure says it also heavily depends on the cost of feedstock
 two dimensional analysis.
12
Commodity Chemicals
Bio-isobutene price = fossile high-purity isobutene price
600,0
Sugar beet
No profit area
Sugar cane
500,0
1996
2005
Cost of raw sugar ($/ton)
1995
400,0
1994
1993
1992
2003
2004
1991
2011
1997
1998
1999
300,0
2010
2009
2001
2000
2002
1995
2011
2008
2005
1994
1993
1992 2003
1991 2002
1999
100,0
2009
2006
2008
1997
1996
1998
200,0
2006
2007
2007 2010
2001
2000
PROFIT AREA
Commodity Chemicals
2004
0,0
0
20
40
60
80
100
120
140
160
180
200
Price of oil ($/baril)
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Fuels
• Fossil fuels have a price close to the price of oil (1$/kg)
• In the near future, cost-competitiveness of biofuels vs. fossil fuels will
be difficult to reach
• Therefore governments have installed biofuel quotas
• Within this “quota market”, Global Bioenergies’ biofuels
 are more cost- and energy efficient to produce due to the gaseous
fermentation process
 have a high energetic density due to the absence of oxygen atoms
 are 100% drop in (IsoOctane vs. Ethanol or Isobutanol)
 present a much better energy/hectare yield than vegetable oil based
Diesel/Jetfuel
14
General Business Model
• Out-licensing model combining upfront-fee and royalties
• Licenses are application specific, normally exclusive (for a determined
duration) and possibly geographically limited
Example: “Exclusive license for MMA derived from bio-Isobutene for
the APAC market (independent on production location)”
• Early collaboration in the form of license options allows to
- secure rights vs. competitors
- faster time to market due to involvement in purification requirements
- lower future upfront and royalty payments
15
Contact:
Jean-Baptiste Barbaroux
Head of Corporate Development
5 rue Henri Desbruères, 91000 Evry, France
Email: jean-baptiste.barbaroux@Global-bioenergies.com
Tel: +33 (0)1 64 98 20 50
Web : www.global-bioenergies.com
Company highlights: Green Biologics
Dr Edward Green, CEO
18
Company highlights: Promethera Biosciences
Eric Halioua, CEO
27
Cell therapy for genetic and orphan diseases:
Promethera Biosciences’ value proposition
E. Halioua (CEO)
EuropaBio’s Most Innovative European Biotech SME Award
September 19, 2012
eric.halioua@promethera.com
Tel: +32 474 05 78 66
1.
Strategy and Development Plan
Promethera Biosciences is a cell therapy company dedicated to
the treatment of liver diseases.
ONE SINGLE MISSING ENZYME CAN HAVE A DRAMATIC CONSEQUENCE ON THE QUALITY OF LIFE
INBORN ERRORS OF LIVER METABOLISM (Crigler-Najjar Disease ) - Orphan diseases: < 1/5000 to
1<2000
Most young patients have only very limited therapeutic options and may die at an early age
Only a few % of enzymatic activities makes a drastic difference in the quality of life
29
1.
Strategy and Development Plan
With Promethera’s process, with one liver hundreds of patients
can be treated.
DRUG SUBSTANCE
Raw Material
Normal Adult
Human Liver
Human
Hepatocytes
Isolation of human
hepatocytes from
the liver
Enzymatic and
mechanical
dissociation of
parenchymal cells
Purification of
HHALPC from
mature liver
cells
HHALPC
in culture
Larger Volume of
HHALPC
In vitro
expansion
DRUG PRODUCT
INJECTION
HepaStem final
product
Intraportal
Injection into
Patient
Washing,
formulation and
final packaging of
HepaStem
Cell resistant to
cryopreservation
Optimized system of
cryopreservation /
storage and quality
control
Treatment with healthy liver cells can cure any single liver-based inborn error of metabolism, as each cell
contains all liver enzymes required for normal function
30
*Note: Method subject to an international patent application
(1) hHALPC = Heterologous Human Adult Liver-derived Progenitor Cells
1.
Strategy and Development Plan
Promethera Biosciences develops two products with the same
technology: HepaStem and HepaScreen.
• Innovative medicinal products to treat miscellaneous liver diseases
HepaStem
• Orphan status obtained from EMEA and FDA
• Proof of concept completed and three patients injected
• In vitro tool for toxicity and metabolic studies for new chemical entities
HepaScreen
• Unlimited access to the cell product
• Used by Pre-clinical departments of Pharmaceutical companies
• Spin-off from the UCL (Université Catholique de Louvain) lab of Professor Etienne Sokal, one of
the leading experts in hepatic cell transplantation
31
Promethera Biosciences’ Pipeline.
Acquired liver
disorders
HepaStem
Liver based Metabolic Diseases
Indications
32
Pre-clinical
Phase I/II
Phase II/III
Next steps
Crigler-Najjar
Start phase I/II
pediatrics in Q1 2012
Urea Cycle
diseases
Start phase I/II
pediatrics in Q1 2012
Phenylketonuria
Start exploratory trial in
adults in 2013
Glycogene
Storage Disease
Type-1
Start exploratory trial in
adults in 2013
Liver Fibrosis
Start animal tests Q2
2011
Product
HepaScreen
Research &
Development
Cell Based Assay
Development/
Characterization
Cell-based Assay
Optimisation
Market
Next steps
Full repertoire of
metabolic activities
Promethera Biosciences targets rare (ultra orphan) and very severe
diseases.
Patient pool for HepaStem for the 4 targeted orphan indications
Disease
Incidence (1 per…)
Europe
Crigler-Najjar
Urea cycle defects
Phenylketonuria
Glycogenosis Type-1
Total of 4 diseases in Europe
33
2008 Prevalence
1,000,000
40,000
19,600
50,000
100
2,841
10,750
3,090
16,781
1,000,000
40,000
19,600
50,000
-
United States
Crigler-Najjar
Urea cycle defects
Phenylketonuria (severs forms)
Glycogenosis Type-1
Total of 4 diseases in US
-
100
2,471
8,250
2,370
13,191
Total of 4 diseases in Europe + US
-
29,972
Assumptions
– Incidence of inborn errors of disease are equivalent across Europe and the United States
– All children born with an inborn error of disease live to be at least 19 years old
– In 2008, the total population of children ages 0 through 19 are 107,000,000 in Europe 82,000,000 in the U.S.
– On an annual basis, there are 5,000,000 live births in Europe and 4,250,000 in the U.S.
Source: Promethera Biosciences Business Plan 2009; US Census Bureau
1.
Strategy and Development Plan
Promethera Biosciences closed in 2012 a 2nd round of financing of
23,6M€: 17M€ in equity & 6.6M€ in loan (Walloon Region).
New investors: international pharmaceutical groups and investment funds
• Mitsui Global Investment, a subsidiary of Mitsui & Co. Ltd. which invests in
opportunities that have potential for growth.
• Shire Strategic Investment Group, Shire’s corporate venture vehicle.
• Boehringer Ingelheim Venture Fund , BI Corporate fund formed in March 2010.
• ATMI LifeSciences, a leader in single-use technologies, products and systems for
mixing, storage, bioreactors, cell culture technology.
• Sambrinvest, an actor in risk capital in the region of Charleroi in Belgium
34
1.
Strategy and Development Plan
This 2nd round of fundraising has been successful thanks to the
major achievements made by Promethera Biosciences.
Achievement of key milestones nearly a year ahead of the initial schedule
First clinical trial authorization by regulatory authorities in Belgium and the UK for HepaStem for
1 the treatment of Crigler-Najjar syndrome and urea cycle disorders in a paediatric setting
Between 2009 and 2011, injections of HHALPC 1 safely administered to three patients - a world
2 first - at Cliniques Universitaires St Luc by Professor Sokal and his team.
Proof of concept for the therapeutic use of the cells has been demonstrated in three different
3 animal models.
4
Successful technology transfer of manufacturing process from the University into Promethera
GMP facilities and manufacturing of 46 HepaStem batches.
1
5 The patent protecting the HHALPC cell and all of its applications officially granted in Europe in
February 2011.
35
1) hHALPC = Heterologous Human Adult Liver-derived Progenitor Cells
1.
Strategy and Development Plan
In just three years, Promethera Biosciences staff has grown from
2 to 46…
CEO
Eric Halioua
CSO
Etienne Sokal
Regulatory Affairs
Resources
VP: René Mignolet
VP: Carole Monterrat
Medical and Clinical
Affairs
Chief Medical Officer
Béatrice De Vos
Preclinical
Development
Manufacturing/QA/QC
Chief Operating Officer
Claude Dedry
VP: Eddy Rommel
QC/R&D
Senior Manager
Vinciane Wouters
IP Manager
Medical Officer
Luca Falciola
Joëlle Thonnard
Manager
Chantal Masungi
Logistic Manager
Senior Manager
Sarah Snykers
Philippe Ducarme
ProductionManager
Philippe Willemsen
Manager QC
Christophe Naômé
Accountancy
Assistant
Carole De Meester
Gessica Turi
Grant Project officer
Vanessa de Behr
Office Manager
Frédérique Ramon
Assistant
Andreea Manolescu
Welcome Desk
Anne
Clinical Project
Manager
Doreen Dekker
Clinical Research
Assistant
R&D Supervisors
Khuu Ngoc Dung
Christophe Gumy
R&D junior scientist
Sabrina Braibant
Elisa Bigaré
Clinical Trial assistant
Candice Hoste
R&D Technicians
Tuba Baran
Yan Greiling
QA/QC Supervisor
Valérie Sapin
ProductionSupervisors
Vanessa Codutti
Pascale Henneresse
QA Supervisors
Gabriela Vilchis
Mike Collodoro
Charlotte Vanhoorne
QC Seniortechnician
Hélène Mertens
QC Technicians
Aline Schepens
Thomas Laukes
Production Technicians
Laurence Antal
Sandrine Donkers
Isabelle Fery
Alain Foyte Kwette
Hélène Lescot
Sébastien Motte
Thao Phan
Duc Khanh tran Do
Ramazan Ay
1.
Strategy and Development Plan
… headed by an experienced management team.
Eric Halioua, MBA: Chief Executive Officer
• Master degrees in Pharmacology and Molecular
Biology, graduate from ESSEC business school
• co-founder of two biotechnology companies called
Myosix and Murigenetics
• principal of the international life sciences practice of
Arthur D. Little based in Paris and Boston
Professor Etienne Sokal, MD, PhD: Chief Scientific Officer
• Paediatrician specialized in gastroenterology with 20
years experience in the field of paediatric hepatology
and liver transplantation
• More than 200 peer-reviewed publications
• Leader of a research team actively working on liver
associated metabolic diseases in children
Beatrice De Vos, MD, PhD: Chief Medical Officer
• Belgian board certified medical doctor and specialist
in pharmaceutical medicine
• 19 years experience in leading positions of clinical
research and medical affairs departments of major
international pharmaceutical companies (WyethAyerst R&D, GSK Bio, Sanofi Pasteur).
37
Claude Dedry: Chief Operating Officer
• Industrial Pharmacist and graduate in business
administration (EPM, Belgium),
• Searle-Monsanto in the department of quality
control of the center of European development
• 15 years experience with Glaxo SmithKline
Biologicals.
Carole Monterrat, PhD, MBA: VP Resources
• PhD in molecular and cellular biology from the
University of Bordeaux
• executive master in finance from the Solvay Business
School
• Several research positions within the Rhône-Poulenc
Rorer research development team at Dagenham
(Essex, UK),
Eddy Rommel: VP of Preclinical Development
• Doctor in Veterinary Medicine and Master of
Sciences in Animal Productions from the University
of Liège (Belgium),
• 15 years experience with GlaxoSmithKline
Biologicals: in-vivo testing of vaccines and
regulatory affairs
Thank you !!
38
Company highlights: ProtAffin AG
Jason Slingsby, CEO
40
ProtAffin AG
Jason H Slingsby, CEO
EuropaBio’s Most Innovative EU Biotech SME Award 2012
Lung Inflammation and COPD
• COPD is chronic inflammatory disease of the lungs
• 4th leading cause of death in EU
• 1 in 10 people >45 have COPD
• 85% are smokers or ex-smokers
• Air pollution also a trigger
• 1 in 4 patients with COPD also develop lung cancer
• Current therapies have limited effectiveness
• One COPD patient dies every 10 seconds
• €10 billion market opportunity
NON- CONFIDENTIAL
Cystic Fibrosis and Lung inflammation
• Approx. 37,000 CF patients in 27 EU states
• Most common life threatening genetic disease
• Median survival is ~35 years of age 1
• Standard of care focusses on managing multiple
disease symptoms and infections
• New hope for CF patients with small molecules
correcting chloride channel function (i.e. Kalydeco)
• Urgent need for potent anti-inflammatory for lungs
1. The Cystic Fibrosis Foundation: www.cff.org/AboutCF
NON- CONFIDENTIAL
ProtAffin Summary
•
Biotechnology Company based in Graz, Austria & London, UK
•
Lead product PA401 is anti-inflammatory protein in clinical development
– Phase I study in UK started June 2012, completing Q1 2013
– Outstanding preclinical data package in COPD and cystic fibrosis
– Broader anti-inflammatory activity than benchmark competitor compounds
•
ProtAffin‘s CellJammer® discovery technology
– Novel therapeutic platform of proteins targeting sugar molecules
•
Pipeline of follow-up products active in models of:
– Tumour metastasis, other inflammatory diseases
•
Raised €19m from international venture capital investors, €6m in grants
NON- CONFIDENTIAL
ProtAffin‘s Innovation vs. Competitors
•
Successful but crowded
field of antibody companies,
market now with sales ca.
€100 bn
•
Heparins are established
drug category of glycans
with €3 billion market
•
ProtAffin is unique in
developing protein
therapeutics targeting
glycans
● IL8 decoy PA401 in
Ph.1
Glycan
● Focus on COPD & CF
● Pipeline of earlier
products in inflammation
and oncology
TARGET
Micromet
Top 20 Pharma
F-Star
Adimab
Protein
● Unfractionated Heparin
● LMW Heparin
Abgenix
● €4 bn market
Medarex
Glycans
Biologics
– Highly innovative
– Unique approach
– Broad IP granted and claimed
DRUG
NON- CONFIDENTIAL
ProtAffin Summary
Novel COPD/CF product PA401
New class of biopharmaceuticals
• Outstanding preclinical data
• New way to target 40+ chemokines
• Differentiated to competitors
• Application to 440 other targets in db
• Flexibility of delivery
• Broad IP granted in EU
• Patent granted US & EU
• Dominating a new field
Pipeline of Follow-ups
• MCP-1 in oncology, MS, CVM
• SDF-1α in oncology, others
• Expanding into other target classes
with Partners
Financing
• Strong VC syndicate
• €19m equity raised to date
• €6m development grants,
• High exit potential: new modality
NON- CONFIDENTIAL
Company highlights: to-BBB
Willem van Werpen, CEO
48
54
55
Title

First level

Second level
•
Third level
56
57
58
59
Key note remarks from host:
Dr Kay Swinburne, MEP
61
Key note address:
G.Steven Burrill, Founder and CEO
of Burrill & CO.
62
Key note address & announcement of winner
Commission Vice-President Tajani
63
Closing remarks
Tom Saylor, Chair of the
EuropaBio SME Platform & CEO of
Arecor Ltd
64
NETWORKING COCKTAIL IN
MEMBERS SALON!
65