Speakers:
Markus Fido
Meridian Biopharmaceuticals
Andrea Berghold
Medical University Graz
Martin Imhof
General Public Teaching
Hospital Korneuburg
Markus Zeitlinger
Medical University, Vienna
Roland Wagner
Rentschler
Ralf Kircheis
Meridian
Andreas Nechansky
Vela Laboratories
Apollon Papadimitiou
Roche Diagnostics
Olwen Birch
Lonza
Hiltrud Horn
Horn Pharmaceutical
Consulting
Armin Franz
Auditreu, Vela Laboratories
Thomas Bauernhofer
Medical University, Graz
Günther Waxenecker
AGES
Thorsten Lemm
Roche Diagnostic
Development of
Biopharmaceuticals –
Preclinical and Clinical Phases
From Development to
Marketing Authorisation
With workshop about
financing biotech companies
30 March – 1 April 2009
Prague, Czech Republic
Objectives
During this course, dos and don’ts of all developing phases of Biopharmaceuticals will be discussed in detail. Industry experts will share their in-depth knowledge and experiences. During this conference a focus will be set on the special
topics.
 Preclinical Development
 Clinical Development
 Quality and Regulatory Aspects
 Marketing authorisation
 Financing
This course will bring together representatives of the biopharmaceutical industry
and regulatory authorities. It has been designed to answer your individual questions concerning all these topics.
Background
Early stage product development in the field of Biopharmaceuticals, Biosimilars
and Biologics is a complex, time-consuming and risky process, even for small Biotech companies. Even the strategy, that the pharmaceutical industry retrieves operational from this market segment, should give emphasis to small companies to
look for convincing and profitable product candidates. The product development
business is expected to provide long-term revenues, which could be significant
for blockbusters on the market, even for Biopharmaceuticals. On the other side
this business has a high demand of financial support from the starting phase (exploratory research, preclinical stages) to the late clinical stages (Phase IIb, Phase
III).
Even the time axis should not be underestimated, 8-12 years from the beginning
to product launch is a general rule. The exception proves the rule. For small Biotech companies this business scenario seems to be tightrope walk. Risk assessment, evaluation of projects, a strict asset-related cost planning and elaborate
project plans with sustainable milestones and decision points are of inherent purpose in the business. A profound knowledge of the research, preclinical and clinical facts and economics are essential. Well trained personal, a proved quality
management and clinical experts as well as a global network and partnerships are
basic requirement.
You have to build up a pipeline of product candidates, if one product fails in a
clinical phase. You have to deal with outsourcing items as well as contract companies. You have to be aware of the patent situation of your candidates and you
should publish experimental data in peer-reviewed journals to succeed hearing
of the scientific community. Additionally, you have to establish a Regulatory Affair-, Clinical Monitoring and Quality Control System and last not least a GMP-related laboratory. What else? Furthermore, a critical question – when it is time to
sell or out-licence the product or present it to the Big Pharma industry?
You have to sit down and ask the question:
 How to manage this complex situation?
 How to find people to spend money for this idea?
 How to fulfil all these guidelines and requirements?
 How to minimize the risks and deficits?
 How to prove the concept, how to make strategic decisions?
Target Group
This course is of interest to those who are involved in
 Product Development of Biopharmaceuticals
 Preclinical and Clinical Research
 Quality Control / Quality Assurance
 Regulatory Affairs
 CMO, CRO and service industry
 developing a pipeline of innovative drug candidates for the
biopharmaceutical market
Moderators
Dr Markus Fido, Meridian Biopharmaceuticals GmbH
Axel Schroeder, Concept Heidelberg
Introduction and Market Overview
A blockbuster is born
Preclinical aspects
Decision points during clinical development
Product launch – now or later
Dr Markus Fido
How to make sustainable
product development?
Biopharmaceuticals from
bench to market





Regulatory Aspects on
Biopharmaceuticals
 The clinical trial application procedure
 Scientific advice procedures – Why, When and How to prepare?
 Marketing Authorization for Biotech derived products – legal background,
formal procedures and experience from the assessors point of view
Dr Günther Waxenecker
Pharmacological &
Toxicology Studies






Method Development /
Validation of Biopharmaceuticals
 Relevant Guidelines
 Phases of Product Development / Testing requirements
 Method Portfolio/Method Development / Method Qualification /
Preclinical development (Overview)
Pharmacological models
Proof of concept
Pharmacokinetics/Pharmacodynamics
Routes of application/Bioavailability
Toxicological studies & Safety Pharmacology
Dr Ralf Kircheis
Method Validation
Dr Andreas Nechansky
Statistical issues in preclinical
and clinical studies




Development of Biopharmaceuticals ? Preclinical and
Clinical phases
 Regulation
 Authorities
 Arranging clinical studies
Study design, Statistical analysis plan (SAP)
Sample size estimation
Randomisation
Reporting
Prof Dr Andrea Berghold
Prof Dr Martin Imhof
Clinical StudiesPractical Aspects in
Design and Evaluation
 Learn about immune dysfunction in patients with advanced or meta-
static epithelial cancer with special focus on Treg cell and NK cells
 Learn about integrating aspects of immune dysfunction in design and
evaluation of clinical studies
 Are there surrogate parameters to evaluate immune response in patients
with advanced or metastatic epithelial cancer?
 Practical examples of study designs and evaluations in patients with advanced
or metastatic epithelial cancer
Univ. Prof Dr Thomas Bauernhofer
Clinical Pharmacology of
Biosimilars
 Pharmacokinetics of Biosimilars
 PK/PD of Biosimilars (including safety aspects if not covered by other talks)
 Biosimilars including regulatory aspects
Dr Markus Zeitlinger
Therapeutic protein production in CHO cells. Current
status and future challenges
 Facility design
 USP and DSP workflow
 Future manufacturing concepts
Dr Thorsten Lemm
The CMO Sense: Stadiumspecific Flexible Solutions for
Therapeutic Protein Manufacturing in Fed-batch and Continuous Cell Culture Processes
 Challenges and criteria in the development of processes at various
starting points
 Cutting the time for process development.
 Challenges and differences of fed-batch and con-
tinuous manufacturing processes.
 Clearing the track for successful IMPD profiling.
Prof Dr Roland Wagner
Structural and Functional
Characterization of Biotherapeutics – Analytical Method
Development




GMP Aspects of Biopharmaceutical API Development‘





Structure – Function Relationship for Biotherapeutics
Glyco-Analytics
Analysis of protein aggregation
Protein Degradation Pathways
Dr Apollon Papadimitiou
GMP requirements during process development
Current Initiatives in Pharmaceutical Development
Approaches to Process Development
Development and Validation
Case Study: Development of a GMP-compliant API
Manufacturing Process
Dr Olwen Birch
Marketing Authorisation
Dr Hiltrud Horn
Post-Conference Workshop
Financing a Biotech Company
How to Finance a young Biotech company in early stage phases
Mag. Armin Franz
Speakers
Dr Markus Fido, Meridian, Austria
Markus Fido is CEO of Meridian Biopharmaceuticals, responsible for Finance &
Controlling and Head of Regulatory Affairs & Quality Operations. He was previously CEO and Managing Director at Vela Laboratories GmbH. Before that he was
Head Quality Control at Igeneon/Aphton Biopharma where he was responsible
for all QC aspects of pre-clinical and clinical projects such as stability studies,
specifications, method validation, and product release. Prior he was Group Leader of Immunology and Product Development at Biomin GmbH, Head Biochemical Control at Baxter AG and Head Quality Operations at Octapharma GmbH.
Univ.-Prof. Dipl.-Ing. Dr Andrea Berghold, University Graz, Austria
Prof. Berghold is head of the Institute for Medical Informatics, Statistics and
Documentation and full professor for Biostatistics at the Medical University of
Graz since 2005. She received her PhD at the Technical University of Graz where
she studied Technical Mathematics. She is member of many national and international societies, being President of the Austrian-Swiss Region of the International
Biometric Society in 2004/05. Her major scientific interests are statistical modelling, meta-analytical methods, planning and analysis of clinical trials
Prof Dr Martin Imhof, General Public Teaching Hospital Korneuburg/Vienna.
Assoc. Prof. Martin Imhof (M.D., Ph.D.) Medical studies in Vienna (-1993). Residency at the Univ. Dep. for Heart- and Thoracic Surgery, General Surgrey and Gynaecology/Obstetric incl. In Vitro Fertilisation Team, 2004 Specialist for Obstetrics and Gynaecology, 2006 Venia Docendi, 2007 Senior Consultant at the 2002
Spin off for GMP production of cell therapies. Scientific activities: Umbilical Stem
Cells, Ovarian Tissue Banking, Tumor therapy with Dendritic Cells.
Dr Markus Zeitlinger, Medical University of Vienna, Austria
After his study and degree at the university of Vienna medical School and his
basic military service at the army hospital of Vienna, Dr Zeitlinger is employed at
Medical University of Vienna. His main current position is Section Head Molecular Pharmacokinetics and Imaging Department of Clinical Pharmacology. Furthermore he is European Expert of the European Medicines Agency (EMEA), Expert of
the Committee for Medicinal Products for Human Use (CHMP) and Assessor of
the Competent Authority of Austria “Bundesamt für Sicherheit im Gesundheitswesen” (BASG)
Prof Dr Roland Wagner, Rentschler Biotechnology, Germany
After his studies of Biology at the University of Braunschweig he was as scientist
at Department of Enzymology. From 1986 until 2004, he was head of the Cell
Culture Technology Department. 2004 he changed position to Miltenyi Biotec
GmbH, Teterow as head of Bioprocess science department. Since 2008 he is the
Senior Vice President Development at Rentschler Biotechnology GmbH, Laupheim, Germany. He is a member of different international organisations like European Society for Animal Cell Technology or European Federation of Biotechnology.
Dr Ralf Kircheis, Meridian, Austria
Following an academic carrier at leading institutions such as the Medical University in Moscow, the Max-Delbrück Center for Molecular Medicine in Berlin-Buch,
and the Biotechnology Research Center of Teikyo University in Tokyo/Kawasaki,
Ralf Kircheis has accumulated 15 years of experience in pharmaceutical industry
and biotech industry. From 2005 he became Head of Research & Development
at Igeneon/Aphton Biopharma. In 2006 Ralf Kircheis was one of the founders
and was the Chief Scientific Officer of Vela Laboratories until 2008. In 2008 Ralf
Kircheis co-founded Meridian Biopharmaceuticals and is currently Chief Scientific Officer / Business Development of Meridian Biopharmaceuticals.
Speakers, cont’d
Dr Andreas Nechansky, Vela Laboratories, Austria
Andreas Nechansky is Co-founder and CEO of Vela Laboratories and is also
responsible for the Department of Analytical Development & Validation
and for Business Development. He previously held the position of Head Assay Development at Igeneon / Aphton Biopharma where he was responsible for the establishment and qualification of analytical test systems that
support the pre-clinical and clinical product profiling. Before, he worked
at renowned institutes such as the Novartis Research Institute (NIBR) in Vienna and The Scripps Research Institute (TSRI) in La Jolla, USA. He received
his PhD in Molecular Genetics from the University Vienna, Austria.
Apollon Papadimitriou, Roche Diagnostics GmbH, Germany
Dr Apollon Papadimitriou is a biologist by training and received his degree from
the Julius Maximilians University Würzburg, Germany in 1992. He has earned his
PhD in Biochemistry at the Julius Maximilians University Würzburg in 1995. After a
post-doctorate at the Institute for Experimental Cancer Research in Freiburg, Germany, he joined Boehringer Mannheim in 1996 as a Senior Scientist in Biotechnology Research .After the integration of Boehringer Mannheim into Roche in 1997,
he held several management positions in Biotechnology Research. In his current
position in Roche Pharma Research Penzberg he is managing the Protein Analytics
unit within the Biotherapeutics R&D organisation.
Dr Olwen Birch, Lonza, Switzerland
Since 1999 Olwen has been involved in Quality Assurance, establishing and
developing a QA group to support the growing Biotechnology sector at Lonza in
Visp, and supporting the QA group at the Lonza Biotechnology plant in Kourim,
CZ. She is currently responsible for qualification/validation of the Biotechnology
plants at Lonza in Visp.
Dr Hiltrud Horn, Horn Pharmaceutical Consulting, Germany
Dr Horn is managing director of HORN PHARMACEUTICAL CONSULTING.
From 1990 to 1997, she was employed by Hoffmann-La Roche in Quality Assurance. From 1997 to 1999, she dealt with medical writing in the ‚International Drug
Regulatory Affairs and Project Management‘ department of the same company.
In 1999, she joined Knoll AG as head of the departments ‚Regulatory Compliance
and CMC Documentation‘ and ‚Dossier Production and Compliance‘ for international drug registration. In 2002, she started at Cap Gemini Ernst & Young, where
she was the responsible consultant for questions concerning biotechnology and
life sciences.
Dr Günther Waxenecker, AGES
Günter Waxenecker studied Food science and Biotechnology at the University of
Agricultural Sciences in Vienna; Started in Research at Novartis Research Institute
in Vienna, later in R&D for Igeneon and finally Intercell; Held various positions as
Postdoc, Project Leader and Program Manager; Now working as Assessor (Expert
Biologics) at AGES PharmMed (Austrian Competent Authority) involved in assessment of clinical trial applications and as EMEA expert assessing products submitted for marketing authorization under the EMEA centralized procedure. Additionally assessor for national and central (EMEA) Scientific Advice procedures.
Univ. Prof. Dr Thomas Bauernhofer, Department of Clinical Oncology, University Clinic of Internal Medicine, Medical University Graz, Austria
Thomas Bauernhofer, MD is associated professor of Internal Medicine, specialist
in Haematology and Oncology, head of the translational research unit and vice
director of the Department of Clinical Oncology, University Clinic of Internal
Medicine at the Medical University Graz (MUG), Austria. Dr Bauernhofer earned
his medical degree from the Karl-Franzens-University in Graz . Dr Bauernhofer got
insight in tumour immunology and endocrinology. He has been involved in conducting clinical Phase I-III trials using active and passive immunotherapy strategies
since 1989.
Speakers, cont’d
Dr Thorsten Lemm, Roche Diagnostics, Germany
Dr Thorsten Lemm is a Director of Recovery and Downstream Processing at
Roche Pharmaceutical Biotech Production and Development in Penzberg
near Munich. Dr Lemm received his PhD at the Rheinische Friedrich-Wilhelms-University and he came to Roche in 1999. In the Pharmaceutical Biotech Production department he successfully conducted a feasibility study for
the production of a recombinant human protein expressed in E. coli. After
a group leader position in the System Integration group of Roche Molecular
Diagnostics, optimizing processes for isolation and detection of DNA on fully
automated platforms, he again joined the Pharmaceutical division in 2003 in
the new implemented department of Biotech Production and Development.
Armin Franz, CFO, Vela Laboratories, Vienna, Austria
Armin Franz is currently CFO and Head of the Department Accounting, Tax
consulting and Payroll at Vela Laboratories GmbH and Meridian Biopharmaceuticals GmbH, where he is mainly responsible for Seed Financing and Capital
acquisition. At Meridian Bio-pharmaceuticals he is in charge for Networking and
global Business Development. He has 18 years experience in finance, controlling, tax consulting, accounting and consulting of different kind of enterprises,
especially start up companies and fast growing entities. He is also a consultant
partner of Auditreu tax consulting. He holds a master degree in business administration from the University Vienna of Business Studies and Economics.
Social Event
On 30 March 2009 you are cordially
invited to a social event. This is an excellent opportunity to share your experiences
with colleagues from other companies in a
relaxed atmosphere.
What Is ECA?
The European Compliance Academy (ECA)
is an independent educational organisation chaired by a Scientific Advisory Board
with members of the pharmaceutical industry and regulatory authorities. The ECA
will provide support to the Pharmaceutical
Industry and Regulators to promote the
move towards a harmonised set of GMP
and regulatory guidelines by providing information and interpretation of new or
updated guidances.
How Do You Become
a Member of ECA?
By participating in one of the European Compliance Conferences or Courses, you
will automatically become a member of ECA for two years - free of charge. More
information about ECA can be obtained on the Website www.gmp-compliance.
org
What Are the Benefits of
ECA?
During the membership, you enjoy a 10 % discount on the
regular participation fee of any European Conference organised by ECA in co-operation with CONCEPT HEIDELBERG.
The GMP Guideline Manager Software with a large number
of guidelines, e.g. EC Directives, FDA Guidelines, ICH
Guidelines, will be forwarded to you when you are using your membership for a conference registration.
About
CONCEPT HEIDELBERG
Founded in 1978, CONCEPT HEIDELBERG is the leading organiser of seminars on
pharmaceutical production, quality control, quality assurance and GMP in Europe.
This year more than 240 events will be organised by CONCEPT HEIDELBERG.
ECA has entrusted CONCEPT HEIDELBERG with the organisation of its events.
Easy Registration
Form:
 Reservation
CONCEPT HEIDELBERG

P.O. Box 10 17 64
69007 Heidelberg
Germany
Reservation Form:
+ 49 6221 84 44 34
Internet:
@ e-mail:
[email protected]  www.gmp-compliance.org
Accommodation
CONCEPT has reserved a limited number of rooms in the conference hotel. You will receive a room reservation form when you have
registered for the conference. Please use this form for your room reservation or be sure to mention “VA 5986 ECA Event” to receive the
specially negotiated rate for the duration of your stay. Reservation
should be made directly with the hotel not later than 28 February
2009. Early reservation is recommended.
Date
Monday, 30 March 2009, 09.00 – 17.00 h
(Registration and coffee 08.30 – 09.30 h)
Tuesday, 31 March 2009, 08.30 – 17.00 h
Wednesday, 1 April 2009, 08.30 – 16.30 h
Venue
Dorint Hotel Don Giovanni Prague
Vinohradská 157A
130 20 Prague 3
Phone +420 2 6703 1111
Fax +420 2 6703 6717
Conference language
The official conference language will be English.
Conference fees
Non-ECA Members EUR 1,890.- per delegate plus VAT
ECA Members EUR 1,605.- per delegate plus VAT
APIC Members EUR 1,795.- per delegate plus VAT
(does not include ECA Membership)
EU GMP Inspectorates EUR 945.- per delegate plus VAT
The conference fee is payable in advance after receipt of
invoice and includes conference documentation, dinner
on the first day, lunch on both days and all refreshments.
VAT is reclaimable.
Registration
Via attached reservation form, by mail or by fax message.
Or you register online at www.gmp-compliance.org.
Organisation and Contact
CONCEPT HEIDELBERG
P.O. Box 10 17 64
69007 Heidelberg, GERMANY
Phone +49 (0) 62 21/84 44-0
Fax +49 (0) 62 21/84 44 34
E-mail: [email protected]
www.concept-heidelberg.de
For questions regarding content:
Axel Schroeder (Operations Director) at +49-62 21/
84 44 10 or per e-mail at [email protected].
For questions regarding reservation, hotel, organisation etc.:
Marion Weidemaier (Organisation Manager) at +49-62 21/
84 44 46 or per e-mail at [email protected].
+49 6221 84 44 34
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Development of Biopharmaceuticals – Preclinical and Clinical Phases
30 March – 1 April 2009, Prague, Czech Republic
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General terms and conditions
If you cannot attend the conference you have two options:
1. We are happy to welcome a substitute colleague at any time.
2. If you have to cancel entirely we must charge the following processing fees: Cancellation
until 2 weeks prior to the conference 10 %,
until 1 weeks prior to the conference 50 %
within 1 week prior to the conference 100 %.
CONCEPT HEIDELBERG reserves the right to change the materials, instructors, or speakers without
notice or to cancel an event. If the event must be cancelled, registrants will be notified as soon as
possible and will receive a full refund of fees paid. CONCEPT HEIDELBERG will not be responsible for
discount airfare penalties or other costs incurred due to a cancellation.
Terms of payment: Payable without deductions within 10 days after receipt of invoice.
Important: This is a binding registration and above fees are due in case of cancellation or non-appearance. If you cannot take part, you have to inform us in writing. The cancellation fee will then
be calculated according to the point of time at which we receive your message. In case you do not
appear at the event without having informed us, you will have to pay the full registration fee, even if
you have not made the payment yet. Only after we have received your payment, you are entitled to
participate in the conference (receipt of payment will not be confirmed)!
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