The GMP Open Meeting - 21-23 October 2015 - Paris, France. Program at-a-glance



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The GMP Open Meeting - 21-23 October 2015 - Paris, France Program at-a-glance Wednesday 21 st October Thursday 22 nd October Friday 23 rd October 13:00 14:00 Delegates Arrival and Registration 08:30 10:00 Main Session 2 New advances in Bioassays: what and how to measure new biologic entities 10:00 10:30 Coffee and Poster Session 10:30 12:00 Main Session 3 PK/PD modelling, PBPK, Distribution of Targeted Biologics 12:00 14:00 Lunch (13:15 14:00 GMP general assembly) 09:15 11:15 Main Session 5 How to best combine drug therapy for optimal efficacy / safety 11:15 11:30 Coffee 11:30 12:50 Main Session 6 Drug discovery approach for de-risking drug development and lowering attrition rate 12:50 13:00 Closing Remarks 13 :00 14:00 Lunch Delegates Departure 14:00-14:15 Welcome and Introduction talk 14:00 15:30 14:15 15:00 Keynote Speaker - A. Deslandes Engineered protein scaffolds as emerging therapeutic proteins 15:00 17:00 Main Session 1 Optimizing PK profiles through nanoparticles delivery Main session 4 Immunotherapy: challenge and role of the PK and PD 15:30 16:00 Coffee and Poster Session 17:00 17:30 Coffee break or Cheese and Wine 16:00 18:00 Poster Blitz + Free Communication 17:30 18:15 Students presentations (Poster Blitz) 18:15 19:30 Posters Session 19:30 Dinner (Free Choice) 19:30 Conference Dinner & Entertainment

GMP Open Meeting 2015 21 st -23 rd October, Paris Meeting Theme: Personalized therapy: new advances in drug design, bioassay and PK/PD modelling Welcome to our invite for contributions to the GMP meeting. We have a great scientific program and debate for this year 2015! We hope that you will be inspired by both joining us and contributing with an oral presentation or a poster at this year s meeting, as well as contribute to the vivid discussions that will generate the topics. All meeting sessions, including posters, will be held in English. We invite you to contact the various session organizers (contact details are below) with your ideas for contributions of talks and posters. We are very much looking forward to meet you in Paris in October. Marion Dehez, Olivier Petricoul, Laurent Nguyen, Antonin Schmitt, Joseph Ciccolini, Vassili Aslanis, Charbel Azar and Marc Trellu.

Scientific Session Synopses Wednesday21 st October Keynote Speaker Antoine Deslandes, Sanofi, Paris, France Engineered protein scaffolds as emerging therapeutic proteins Engineered protein scaffolds have become attractive for many applications in biotechnology and biomedical research. We will review the current state of the art in this field, with special emphasis on biomolecular structure and function as well as on approaches toward clinical application. Several technologies are available to optimize the pharmacokinetics (PK) and pharmacodynamics (PD) of therapeutic peptides and proteins. Moreover, the bi- or multi-specific nature of some new constructs requires specific bioanalytical methods development. Main Session 1 - Optimizing PK profiles through nanoparticles delivery [J. Ciccolini/ ciccolini.joseph@gmail.com] Major recent breakthroughs in clinical medicine are all based upon the development of new chemical entities. However, redesigning old drugs as nanoparticles could help to stretch their efficacy/toxicity balance, because they are expected to display a higher selectivity towards target tissues. Improving drug delivery with nanoparticles can be achieved by designing a variety of supports encapsulating the active compounds (i.e., liposomes, nanocapsules, dendrimers, squalenes, to name but a few). Alternatively, direct conjugation of the active compounds to PEG chains, antibodies, or biotechnologically engineered proteins (i.e., ADC, nab-drugs ) proved to optimize as well the pharmacokinetics, thus the pharmacodynamics, of the drugs. The variety in supports and techniques made available today to drive active compounds can address numerous issues in pharmacokinetics such as liver uptake, trans-membrane passage, compound instability in biological fluids, or passive/active tissue targeting. This session will cover the current achievements, limits and perspectives of nanoparticles as tools for optimizing drug DMPK profile, including the emerging nano-safety issues. - Simona Mura, Institut Galien, Paris, France Optmizing drug delivery through novel scaffolds - Raphaelle Fanciullino, Aix-Marseille University, Marseille, France Nano-particles: pharmacokinetics and biodistribution - Jacques-Aurélien Sergent, Solvay Pharma, Bruxelles, Belgium Nano-safety and regulatory challenges Students Presentation [A. Schmitt/antonin.schmitt@u-bourgogne.fr] The best submitted posters will be selected for oral presentations. Thursday 22 nd October Main Session 2 - New advances in Bioassays: what and how to measure new biologic entities [M. Trellu/Marc.Trellu@sanofi.com] Biophysical analysis is now playing a significant role in the research and development for a new generation of complex therapeutic proteins. With the wave of new biotherapeutic formats, there is an increasing need for better and faster characterization tools and strategies, and improved

biomolecular and biophysical assays for the new biotherapeutics (ADCs, Bispecifics, ). The combination of improved understandings of the structure and function of proteins with advanced, higher resolution analytical methods helps researchers identify and fine-tune candidate molecules before advancing them into development as well as to have robust and reliable method in development able to quantify biologics with higher throughput and resolution. - Marie-Hélène Pascual, Sanofi, Paris, France Validation of an immunoassay to selectively quantify the naked antibody of a new antibody drug conjugate SAR566658 for pharmacokinetic interpretation improvement (Biological bioassay) - Elsa Wagner, Pierre Fabre, Saint-Julien-en-Genevois, France Antibody and ADC characterization by high resolution mass spectrometry: top, middle and bottom levels - Olivier Pasquier, Sanofi, Paris, France Determination of Drug to Antibody Ratio (DAR) in vivo by High Resolution Mass Spectrometry to support Antibody Drug Conjugates programs (Physico-Chemical bioassay) Main Session 3 Distribution and PK/PD modelling of Targeted Biologics? [O. Petricoul/olivier.petricoul@novartis.com] Understanding the disposition of large protein molecules, e.g. monoclonal antibodies (mabs), is critical for pharmaceutical development, as exposition at the site of action or at any organ/tissues of interest can have a significant impact on drug efficacy and safety. Although bioanalytics and imaging technics have evolved and are now more and more used to describe and quantify large therapeutic proteins in the whole body, modelling technics applied to physiologically based pharmacokinetic (PBPK) of mabs can be of help to predict drug level at the target site of action. This session will focus on the disposition of large molecules from discovery through development illustrated with case studies. - Ludivine Fronton, Roche, Basel, Switzerland PBPK modeling for mabs: more clarity on tissue clerances - Hans Peter Grimm, Roche, Basel, Switzerland Antibody Biodistribution = Imaging Modeling - Olivier Nicolas, Sanofi, Montpellier, France PBPK Simcyp models of biologics: three Sanofi examples Main Session 4 - Session Immuno-oncology: challenge and role of the PK and PD [L. Nguyen/laurent.nguyen@pierre-fabre.com] Immuno-oncology has emerged as a new promising and revolutionary area of cancer therapy. In just the past few years, the rapidly advancing field of cancer immunology has produced several new game changing strategies of treating cancer that increase the strength of immune responses or (re)activate the cellular immunity against tumors. Therapeutic approaches include for instance cytokines that enhance dendritic cell maturation and T-cell differentiation, checkpoint inhibitors that reactivate cytotoxic T-cell response, toll-like receptor agonists. Other innovative strategies include oncolytic viruses that induce tumor cell death and trigger systemic antitumor immunity, and adoptive transfer of native and genetically engineered T cells that are designed to recognize specific

tumor antigens through the T-cell receptor. New tumor vaccines either given alone or in combination with immuno-modulators are also currently investigated as therapeutic alternatives. These new therapies constitute a big challenge for integrating Pharmacokinetic and Pharmacodynamic framework during the pre-clinical and clinical phase of drug development. Amongst these challenges are the complex interplay between the immune system and the cancer cells rending difficult PD biomarkers selection and monitoring, the understanding of immunemediated tumor cell death mechanisms and the integration of PK/PD modelling. In addition, the translational relevance of pre-clinical models and how to deal with immune-mediated clinical efficacy response are questioned. In this session, speakers will present examples of immuno-oncology drug development in order to better understand how to deal with these new treatment options, with an emphasis in the PK/PD drug development activities. - Pierre Ferre, Pierre Fabre, Toulouse, France Overview of immuno-oncology strategy: PK/PD challenge - Franck Pages, HEGP, Paris, France Monitoring of immuno-therapy using PD biomarkers - Fabrice Barlesi, APHM, Marseille, France Check point inhibitors: clinical application and demonstration Free communication [A. Schmitt/antonin.schmitt@u-bourgogne.fr] - Bernard Maillère, CEA, Saclay, France How to predict Immunogenicity of therapeutics antibodies? - Adrien Tessier, Servier, Suresnes, France How to integrate phamacogenetics in population PK modeling - Nassim Djebli, Sanofi, Montpellier, France M&S to support BLA submission of a monoclonal antibody presenting TMDD elimination mechanism Friday 23rd October Main Session 5 - Development of drug combos: achievements, pitfalls and perspectives [J. Ciccolini/ciccolini.joseph@gmail.com] A rising number of diseases require several different drugs to be administered in a row to achieve some clinical efficacy. Oncology is paradigmatic of this new trend and the ever-increasing number of targets made druggable implies now multiple combinational strategies to circumvent possible crosstalks and rescue pathways associated with drug resistance. In addition, the complex interactions between MAP-Kinase or PI3-Kinase signaling pathways and immune checkpoints prompt several groups to study the benefits of multiple targeting in tumors. Although promising, this strategy presents several issues that remain to be addressed: - Increasing the number of drugs to be combined increases the probability of DDI. - Hitting several pathways can trigger unexpected pharmacodynamic responses, such as metastatic acceleration. - Drug delivery may be hampered by other drugs.

- Overlapping combined toxicities should not exceed the expected clinical benefit. To date, most combinational studies are based upon empirical designs, either during experimental non-clinical phases or early clinical phases of development. The above mentioned items call for a methodologically sound analysis of the previously described issues, i.e. by developing computeraided approaches to simulate in silico the PK/PD relationships of drugs when used in combination. This session will cover the current achievements and limits in the field of drug combination development in oncology. - Vincent Duval, Novartis, Basel, France Modeling combination therapy - Sébastien Benzékry, INRIA, Bordeaux, France Anti-angiogenic modeling - Pr. Benoit You, Hospices Civils de Lyon, France Clinical trial of combinational therapy Main Session 6 - Drug discovery approach for de-risking drug development and lowering attrition rate [C. Azar/charbel.azar@galderma.com and M. Dehez/ marion.dehez@ipsen.com] Drug discovery is plagued by high attrition rate. Developing a new drug is a complex process and requires an early appraisal of all factors impacting on the likely success of a drug candidate in the preclinical phase. It may take many years to build up a body of supporting evidence before selecting a target. Once a target has been chosen, the key preclinical stages are crucial for understanding the behaviour of the drug. It is well-known that efficacy of a drug is tightly intertwined with its interaction mechanism with the drug target as well as the mechanism is dependent on the physicochemical and structural characteristics of both target and drug molecule. Thus the pharmaceutical industries have streamlined a number of early processes to identify molecules that possess suitable characteristics to make acceptable drugs. One crucial preclinical process is to integrate and use the in-vivo PK data in order to support and optimized the drug design for increasing the successful of the selection of a candidate molecule for clinical development. Today, the relationship between the physicochemical drug properties (in particular lipophilicity) and the PK/PD/TOX is clearly encouraged for improving the quality of the lead compounds in the preclinical phase. Consequently, a deeper understanding of interaction mechanisms as well as the physicochemical properties and the classes of molecules is needed/mandatory for de-risking drug development and lowering attrition rate. - Cécile Bonnard & Ralph Eckenberg, 3DS, Dassault System, Vélizy, France In silico approaches for early assessment of drugs - Sebastien Tourlet, IPSEN, Les Ullis, France Prediction of toxicity using system s biology approaches on the biological target profile