stemvac_consortium management case_dr. sandra huygen
Post on 15-Jul-2015
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• Breast cancer– 1.3 million new cases worldwide (second cause of cancer death)
– + 3.2% yearly
• Early stage of disease represent the majority of cases
• In addition to surgery, 3 treatments types– Chemotherapy
– Hormone therapy
– Selective therapies (Her2/neu)
• Treatments Limitations– Toxicity
– Efficacy: 60 % of patients die after disease recurrence
Clear medical need for treatments associated withimproved efficacy and safety
Immunotherapies and targeted therapies have thepotential to address this need
Market needs
3
Introduction to program
FRAMEWORK • Grant (5 partners - 3.053.260 € - 4Y)
• Developpement of innovative therapies against breast cancer
OBJECTIVES • Identify antigens specifically expressed by breast cancer stem cells.
• Followed by development into targets for cancer immunotherapies
Therapeutic vaccines: deliverable is a product ready to enter afirst-time-in-human clinical trial
Targeted therapies (monoclonal antibodies): deliverable is aproduct ready to enter preclinical development
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Consortium
Partner Expertise
UNamurUnité de Recherches en Physiologie Moléculaire
AcademiaStem cell cultureCancer stem cells
UCL & De Duve Institute AcademiaGene expression
analysis
ULG & GIGA-CancerMetastases Research Laboratory
AcademiaInnovative proteomics, Biomarkers discovery
Targetome (TGM) Spin-offAntibody based
targeted therapies and in vivo imaging
GSK Vaccines CompanyVaccine R&D, production,
commercialization
4STEMVAC Kick-off
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Consortium & program management
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Legal framework – 2 agreements Partner rights & obligations IP Access rights Publications Confidentiality Gantt chart & budget
Organization Steering committee Business development Valorization committee Executive committee Task forces Biowin Rw
Design control R&D technical review Marketing Business development
Regulatory affairs Clinical affairs IP QA
• Academic level
– UCL: Televie, FRSM, Actions de recherches concertées
– ULg: Televie, FRSM, Actions de recherche concertées, projet européen FP7 (ADAMANT, Marie-Curie)
– FUNDP: Televie, FNRS
• Industrial level
– Feed the pipeline with more innovative candidates
– Multiple interactions foreseen with other development projects
Synergies and multiplier effects
Innovation
Stem cell primary culture from patients’ samples
Breast cancer stem cells antigen identification
Based on both transcriptomic and proteomic data
Biomarker validation on large human tissue collections
Cancer immunotherapy development
Double translational research concept
• From the patient to the laboratory and back to the patient
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Innovation
Highly innovative immunotherapeutic products to targetcancer via newly discovered cancer stem cell specificantigens.
Most important characteristics of cancer stem cell antigens
• Identified based on their specific expression by cancerstem cells.
• Identified at the protein level, thus taking into accountany post-translational expression regulation.
• Rigorously validated for tumor specific expression in aseries of tumoral and normal tissues.
Identification of antigens specifically expressed by breastcancer stem cells would be a major breakthrough.
At this stage, there are no existing patents that wouldprevent us from protecting our findings and products.
CSC SC
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• Very few companies engaged on cancer stem cell research
– Macrogenics (formerly Raven Biotechnology)
– Oncomed
– Focus on pancreas, colon and lung cancer.
• No clinical trial (ongoing or planned) to test immunotherapies targeting cancer
stem cell associated antigens
• No patent with broad claims that would prevent us from patenting our findings
• Patenting strategy will be designed based on earlier patent application history
• Freedom to operate has to be re-evaluated for each proposed antigen
Competitors and FTO
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Program flow details
Breast Cancer Cell lines Human Breast Tissue Primary Culturescancer
normalCSC
Proteomics
Total proteome Membranome (after cell surface biotinylation)
Mass spectrometry identification of a repertoire of biomarker specific for breast CSC
Data analysis
• PCR and IHC on human normal tissues
• PCR and IHC on collections of human breast cancer
Vaccine Develop
ment
mAbDevelop
ment
1
ULg
-FU
ND
P
• Transcriptomic analysis (microarrays)
• Bioinformatic treatment of data for antigen selection
CSC
SC (stem cells)
2
3
5 6 • Selection of the most promising antigens• Preclinical PoC and validation• Production of a clinical grade product
• Selection of accessible biomarkers• Antibody generation• Preclinical evaluation
ULg
UC
LG
SK
TGM
Target Validation
4
ULg
-FU
ND
P-
UC
L-G
SK
• Functional assays using siRNA and xenografts
Breast Cancer Stem Cells (CSC)
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Risk & mitigation
Cell culture steps could introduce biological biases
• Analysis based on short term cultured primary cells
Need to enrich the culture in cancer stem cells
• Use of short-term culture, in normoxic vs hypoxic conditions
• Sampling of cells after chemotherapy whenever possible
False positives in specifically expressed genes
• Integration of transcriptomic and proteomic data
• Rigorous validation on tumor tissue banks
Cancer therapeutic vaccine development is a high risk activity
• First cancer vaccine approved in 2010 in the US
• GSK’s approach is supported by two ongoing phase III clinical trials
• Rigorous project management which includes regulatory experts
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Risk & mitigation
Choice of antigens to be used for development is critical
Go/No Go decision point is proposed after the research phase
Decision will involve all partners and take into account 5 pre-specified criteria
– Commercial
– Competition
– IP– Expression
– Biology
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Valorization & marketing
The results of the research phase of the project, i.e. newtargets for cancer immunotherapy, will be patented. Based onthese results, GSK and Targetome will develop new therapiesfor breast cancer
Targetome is granted an exclusive option on the IP generatedduring the project in the field of monoclonal antibodies
Targetome will develop monoclonal antibodies forimmunotargeted therapies
GSK is granted an exclusive option on the IP generated duringthe project in the field of cancer vaccines / active cancerimmunotherapies
The proposed products will be commercialized within theexisting worldwide GSK commercial structure
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Social economic impact
• We estimate that the performance of this project (2013-2017)will require the creation of about 10 qualified and highlyqualified stable positions in Wallonia, mostly in Targetome andGSK.
• If successful, after 2017 the project will create and maintain
Worldwide early clinical trials: several hundreds ofpositions, out of which around 40 in Wallonia
Worldwide late clinical trials: several hundreds of positions,out of which around 150 in Wallonia
For vaccine production and commercialization, severalhundreds of positions in Wallonia
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Summary
Integrated research and development project to createinnovative new therapies against cancer
Breast cancer: a worldwide unmet medical need
Synergy between the partners’ expertise
Innovation lies in the nature of the proposed therapies
• Immunotherapies
• Targeting novel antigens
• Targeting Stem Cells
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Opportunity for the Walloon region
Strengtening wallonia expertise (genomics, proteomics, bioinformatics, cancer stem cells)
Cutting-edge cancer stem cell research
With a input from Biotech and Pharma from the research phase onwards
For a successful technology transfer and to create value
Thank you for your attention!
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For more information
Dr. Sandra HUYGEN, PhD, MBA
Consortium management & synergies expert
sandra.huygen@allianses.bewww.allianses.be
0032/486.467.191
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