biotechnology (biotec) - American Association of Pharmaceutical ...

BIOTECHNOLOGY (BIOTEC)
MONDAY TUESDAY WEDNESDAY THURSDAY
MONDAY MORNING ROUNDTABLES AND
MINI-SYMPOSIA ARE SUPPORTED BY A
GRANT FROM
8:00 AM–10:00 AM
Outcomes of the Global Bioanalysis
Consortium’s International Conference:
Review of the Proposals
ROUNDTABLE
MONDAY AFTERNOON SYMPOSIA ARE
SUPPORTED BY A GRANT FROM
1:30 PM–4:00 PM
Intracellular Signaling by Flow Cytometry:
A Critical New Flow Cytometry Application
for Drug Development
SYMPOSIUM
Manufacturing, Formulation and Analytical
Challenges and Strategies for Biosimilars
SYMPOSIUM
5:00 PM–7:30 PM
AAPS BIOTEC Section Joint Membership
Meeting and Reception
30
PRELIMINARY PROGRAM
TUESDAY MORNING SUNRISE SESSIONS ARE
SUPPORTED BY A GRANT FROM
7:00 AM–8:15 AM
Personalized Medicine 101
SUNRISE SESSION
8:30 AM–11:00 AM
Graduate Student Symposium in
Biotechnology (BIOTEC)
SUPPORTED BY A GRANT FROM
TUESDAY MORNING ROUNDTABLES
SUPPORTED BY A GRANT FROM
9:00 AM–11:00 AM
Emerging Biosimilar Strategy and
Challenges: Unique Aspects and Key
Contrasts to Comparability, other Follow
on Biotherapeutic Strategies, and Small
Molecule Generics
ROUNDTABLE
2:00 PM–4:00 PM
Biologics and Biosimilars Development and
Approval in Emerging Markets
ROUNDTABLE
7:00 AM–8:15 AM
PGx Today: What Do We Know?
SUNRISE SESSION
8:30 AM–11:00 AM
Next Generation Biologics
SYMPOSIUM
WEDNESDAY AFTERNOON SYMPOSIA ARE
SUPPORTED BY AN EDUCATIONAL GRANT
FROM
1:30 PM–4:00 PM
Biomarkers of CNS Function: From Mice
to Man
SYMPOSIUM
From a Monomer to an Aggregate: Models,
Challenges and Case Studies for Prediction
of Protein Aggregation In Solution
SYMPOSIUM
WEDNESDAY AFTERNOON ROUNDTABLES
ARE SUPPORTED BY A GRANT FROM
2:00 PM–4:00 PM
Emerging Platforms for Immunogenicity
and Quantitative Assays
ROUNDTABLE
AT-A-GLANCE
7:00 AM–8:15 AM
Personalized Medicine: Bring Your Birth
Weight
SUNRISE SESSION
Biosimilars Development: CMC, Preclinical,
and Clinical Considerations
SUNRISE SESSION
9:00 AM–11:00 AM
Problems and Solutions for Manufacturing
Therapeutic Proteins
ROUNDTABLE

MONDAY, OCTOBER 15
MONDAY MORNING ROUNDTABLE
SUPPORTED BY A GRANT FROM
8:00 AM–10:00 AM
Outcomes of the Global Bioanalysis
Consortium’s International
Conference: Review of the
Proposals
ROUNDTABLE
Currently, regulatory differences between regions
and countries requires additional work or are in
conflict; resulting with unneeded hurdles to the
bioanalytical community in using its work and
output in filings around the globe. To address this, in
September 2012, the Global Bioanalysis Consortium
(GBC) is holding an international conference where
proposals for harmonized bioanalytical regulations
will be presented to the scientific, quality assurance,
academic and regulatory communities. This
roundtable, occurring just three weeks later, will
present the significant outcomes of the meeting
and describe the content of the discussions so
that scientists who could not attend will be able
to understand the drivers that went into the final
proposals. An FDA representative will present some
early reflections on the recommendations. As it
writes the white paper outcome of the conference,
input from the audience will be sought as feedback
to the GBC.
MODERATORS
Mark E. Arnold, Ph.D.
Bristol-Myers Squibb Company
Fabio Garofolo, Ph.D.
Algorithme Pharma Inc.
Significant Outcomes for Immuno Assays,
Biomarkers and some Common Themes
Binodh S. DeSilva, Ph.D.
Bristol-Myers Squibb Company
Significant Outcomes for
Chromatographic Assays and some
Common Themes
Eric N. Fluhler, Ph.D.
Pfizer Inc.
Regulatory Perspectives: Where Do We
Go from Here
Brian P. Booth, Ph.D.
U.S. Food and Drug Administration
MONDAY AFTERNOON SYMPOSIA
SUPPORTED BY A GRANT FROM
BIOTECHNOLOGY (BIOTEC)
1:30 PM–4:00 PM
Intracellular Signaling by Flow
Cytometry: A Critical New Flow
Cytometry Application for Drug
Development
SYMPOSIUM
The detection of intracellular signaling events such
as protein phosphorylation by flow cytometry
(PhosphFlow) compared to gel techniques
has revolutionized signaling biology and drug
development. PhosphFlow allows for the detection
of specific protein phosphorylation in intact cells
combined with the detection of other intracellular
proteins and cell surface markers. This approach
holds enormous promise in a variety of therapeutic
areas, in particular, oncology, as pathway specific
mutations can be defined. Further the specific
effect of a drug or mutation on a pathway or
pathway “nodes” (areas where pathways converge
or diverge) can be identified. The purpose of
this session will be to discuss the challenges of
implementing robust PhosphFlow assays, such as
reagent verification, buffer optimization, specimen
stability, and analysis software. This symposium
would provide an open forum discussing not only
the challenges, but how people have been able to
overcome these challenges as well.
MODERATORS
Peter O’Brien, Ph.D.
Pfizer Inc.
Virginia M. Litwin, Ph.D.
Covance, Inc.
Intracellular Signaling by Flow
Cytometry: Overview
Omar D. Perez, Ph.D.
Tocagen Inc.
Steps to Success in Cell Signaling
Cytometry
Lisa J. Green, M.S.
Covance, Inc.
Thinking IN the Box: Development,
Optimization and Validation of Cell
Signaling Assay for Longitudinal
Clinical Trials
Leanne Flye-Blakemore, M.S.
LabCorp Clinical Trials
Utilization of Single Cell Network Profiling
to Build and Validate Multivariate Models
for the Prediction of Response to Therapy
for Pediatric Leukemia
Urte Gayko, Ph.D.
Nodality Inc.
1:30 PM–4:00 PM
Manufacturing, Formulation
and Analytical Challenges and
Strategies for Biosimilars
SYMPOSIUM
The patent expirations of biopharmaceutical
products have initiated the introduction of a new
generation of drugs, called ‘Biosimilars’ (followon
products to marketed biological medicines).
Biosimilars are highly similar structurally but not
identical to the original biopharmaceutical products.
Due to their large size and complex structure,
production of biopharmaceutical requires a
complex manufacturing and quality control process.
Also, they are highly sensitive to variations of the
production process. Unlike generic versions of
small-molecule-drugs, biosimilars require extensive
characterization and verifications to determine the
efficacy, immunogenicity and bioequivalence to the
original biopharmaceutical product. This symposium
will discuss the challenges the pharmaceutical
industry is facing in manufacturing, characterization
and demonstrating comparability.
MODERATORS
Arya P. Jayatilaka, Ph.D.
Pfizer Inc.
Ruta Waghmare, Ph.D.
EMD Millipore Corporation
Biosimilars: Key Aspects in Technical
Development
Carol Kirchhoff, Ph.D.
Pfizer Inc.
Similarities and Differences in the Process
Development Cycle
Neil Schauer, Ph.D.
Hospira, Inc.
Challenges in Demonstrating Biosimilarity
to a Reference Product
Speaker to be Determined
Analytical Characterization of
Biopharmaceuticals in the Determination
of Biosimilarity
Graham Jones, D.Sc.
Northeastern University
JOINT MEMBERSHIP MEETING
AND RECEPTION
5:00 PM–7:30 PM
AAPS Biotechnology (BIOTEC)
Section Joint Membership Meeting
and Reception
2012 AAPS Annual Meeting and Exposition 31

BIOTECHNOLOGY (BIOTEC)
TUESDAY, OCTOBER 16
TUESDAY MORNING SUNRISE SESSION
SUPPORTED BY A GRANT FROM
7:00 AM–8:15 AM
Personalized Medicine 101
SUNRISE SESSION
Personalized Medicine is the tailoring of therapies
to individuals or sub-populations of individuals
and is gaining popularity with the advances in the
fields of diagnostics, biomarkers and genomics.
Examples of personalized medicine include but
are not limited to Warfarin (small molecule —
anticoagulant), Herceptin ® (biologic — cancer
monoclonal antibody), and the more recently
approved Provenge ® (cancer vaccine for prostate
cancer). The advancement of personalized
medicine is dependent on a number of players —
pharmaceutical and biotech companies, diagnostic
companies, researchers, clinicians, information
technology managers, healthcare providers,
laboratories, policymakers and payers. This sunrise
session will cater to the science, promise and
challenges in the field of personalized medicine.
MODERATORS
Nisha Nanaware, M.Pharm.
University of Arkansas
Lavinia M. Lewis, Ph.D.
Pfizer Inc.
Personalized Medicine: An Overview
Wolfgang Sadee, Ph.D.
Ohio State University
Challenges in Manufacturing Personalized
Cellular Therapies
Tamara T. Monesmith, M.S.
Argos Therapeutics
GRADUATE STUDENT SYMPOSIUM
8:30 AM–11:00 AM
Graduate Student Symposium in
Biotechnology (BIOTEC)
SUPPORTED BY A GRANT FROM
32
PRELIMINARY PROGRAM
TUESDAY MORNING ROUNDTABLE
SUPPORTED BY A GRANT FROM
9:00 AM–11:00 AM
Emerging Biosimilar Strategy
and Challenges: Unique
Aspects and Key Contrasts to
Comparability, other Follow on
Biotherapeutic Strategies, and
Small Molecule Generics
ROUNDTABLE
This roundtable will focus on the unique aspects
of biosimilar strategy with a focus on key contrasts
to comparability, other follow on biotherapeutic
strategies (such as biobetters), and small
molecule generics. Biosimilarity is focused on the
development of a biosimilar product that is “similar”
to an externally marketed biotherapeutic drug
product with plans to license (bring to market)
the biosimilar when the original product loses
its patent exclusivity. There are several recent
biosimilar guidances in process and guidance
updates (especially in the emerging markets).
Comparability, due to manufacturing changes with
an internal product, has a large impact on internal
drug development programs resulting sometimes
in delays or in requirements to resubmit as a new
drug product. However, while biosimilarity and
comparability share a goal of comparing two (or
more) products the overall strategies and study
design optimizations are different. The aim of this
roundtable is to briefly recap some of the key
discussion points regarding biosimilarity issues over
the last several years (with a focus on nonclinical
and clinical assessments) and to bring forward
discussions on common issues and key differences
with comparability and biobetters. The key issues
of biosimilar strategy for a biotherapeutics product
will also be contrasted to that for a small molecule
generic. This is important as even the words
biosimilarity, comparability, biobetters, and generics
have become mingled in discussions on biosimilars.
This roundtable will provide opportunities for
AAPS attendees to discuss/debate nonclinical and
clinical issues focused on the topics below. I.) To
briefly recap recent development strategies for
biosimilarity. II.) Key regulatory differences between
biosimilarity, comparability, and biobetters based
on guidances and overall study objectives (with a
brief overview of differences with small molecule
generics). III.) To understand the overlapping
scientific discussions that impact biosimilarity,
comparability, and biobetters such as: choice of
key parameters for assessment; incorporating ADA
considerations in the study design; considerations
for TMD; utilization of PD endpoints; incorporation
of Modeling and Simulation in study design setup;
utilization of historical data in assessments; setting
of guideposts (90% CI: 80 — 125 only?), and
power calculations; and optimization of the use of
preclinical species. IV.) To understanding the key
differences between biosimilarity, comparability,
and biobetter studies particularly around utilization,
design and interpretation of nonclinical studies
and the need for and design/optimization of
clinical studies.
MODERATORS
Susan I. Hurst, R.Ph., Ph.D.
Pfizer Inc.
Shefali Kakar, Ph.D.
Novartis Pharmaceuticals Corporation
Biosimilars, Biobetters, and Comparability
Assessments: Strategies and Challenges
Shefali Kakar, Ph.D.
Novartis Pharmaceuticals Corporation
Developing an Integrated Approach to
Global Biosimilar Development Programs
Deepa Deshpande, Ph.D., RAC
Universal Regulatory Inc.
Biosimilar Development: A Pharmaceutical
Industry Perspective
Michael Corbo, Ph.D.
Pfizer Inc.
TUESDAY AFTERNOON ROUNDTABLE
SUPPORTED BY A GRANT FROM
2:00 PM–4:00 PM
Biologics and Biosimilars
Development and Approval in
Emerging Markets
ROUNDTABLE
Biologics continue to attract increasing attention of
all major pharmaceutical companies globally year
after year since the new millennium. The global
biosimilars market has also continued to grow at
an impressive pace and is now predicted to reach
about $20 billion by 2014 (i.e. growing annually at
about 89% from 2009 to 2014). Asia in particular
has been a major growth region for biosimilars,
largely due to relatively early commercialization
and high acceptance rate of biosimilar products
in several populous Asian countries. While the
United States is the second-largest global market
are predicted to catch up soon and become the
dominant market by 2014 thanks to the recent
U.S. legislation and market opening for biosimilars
since 2010, Asia continues to dominate the
biosimilars market for now with about 34% share
of the global market in 2008 and continuing to
grow. In the meantime, the Asia biosimilars market
appears highly fragmented with a handful of major
biopharmaceutical companies making most of the
market share (e.g. Biocon, Dr. Reddy’s Laboratories,
LG LifeSciences, Green Cross, Ranbaxy, Kexing,
Celgen, Sandoz, Teva, Wockhardt). The regulatory
pathways and filing requirements for biosimilars also
vary from country to country. In this roundtable, an
overview of global biologics and biosimilars pipeline
and market trend will be presented, followed by
discussions of industry perspectives in several major
emerging markets, China, Korea, and India. A panel
discussion will also be held with all the invited
expert speakers.

MODERATORS
Qiang Ye, Ph.D.
Pharmaceutical Development
Yining Zhao, Ph.D.
Pfizer Inc.
Multi-national Companies’ Perspectives
on Developing Biosimilars in Emerging
Markets
Yining Zhao, Ph.D.
Pfizer Inc.
Opportunities and Obstacles to Develop
Biologics in China
Chris Chen, Ph.D.
WuXi AppTec Co.
Current Status and Future Outlook for
India’s Biosimilar Development and
Approval
Cartikeya Reddy, Ph.D.
Dr. Reddy’s Laboratories
WEDNESDAY,
OCTOBER 17
WEDNESDAY MORNING SUNRISE SESSION
7:00 AM–8:15 AM
PGx Today: What Do We Know?
SUNRISE SESSION
Pharmacogenomics is the study of variations of
DNA and RNA characteristics as related to drug
response. This session will address the following
considerations: 1. how to relate phenotype to
genotype, 2. how to use PGx information to identify
novel drug targets for drug development, and 3. how
to apply PGx information to explain or predict the
role of genetic variability in drug efficacy and toxicity.
The session will also provide a number of examples
for the application of PGx in clinical practice.
MODERATORS
Shashi Amur, Ph.D.
U.S. Food and Drug Administration
Li Zhang, Ph.D., M.D.
U.S. Food and Drug Administration
PGx Today: What Do We Know?
Shashi Amur, Ph.D.
U.S. Food and Drug Administration
Progress in PGx and its Promise for
Medicine
Li Zhang, Ph.D., M.D.
U.S. Food and Drug Administration
WEDNESDAY MORNING SYMPOSIUM
8:30 AM–11:00 AM
Next Generation Biologics
BIOTECHNOLOGY (BIOTEC)
SYMPOSIUM
Antibody-based biotherapeutics currently enjoy
unprecedented success, growth and recognition of
their potential. Almost all FDA-approved therapeutic
antibodies and the vast majority of those in clinical
trials are full-size bivalent monoclonal antibodies
(mAbs) mostly in IgG1 format of about 150 kDa
size. This success and enthusiasm in the drug
development community can be largely explained
by the properties of antibodies: their exquisite
binding specificity and low intrinsic toxicity. A
fundamental problem for such large molecules
is their poor penetration into tissues and poor or
absent binding to regions on the surface of some
molecules that are only accessible by molecules
of smaller size. In addition, for certain applications
it is desirable to increase the potency of mAbs or
deliver them locally. Advances in protein engineering
have led to the generation of a number of antibody
derivatives and various scaffold proteins, which
due to their smaller size can be beneficial in various
aspects such as immunogenicity, biodistribution,
renal clearance, and tissue penetration. Several
approaches are being evaluated to improve the
potency by the linkage of mAbs to highly cytotoxic
drugs (antibody-drug conjugates, ADC), by
engineering antibodies with dual specificity (bispecific)
or by enhancing antibody effector function
by engaging T cells and effector cells using bispecific
T cell engagers (BiTE ® ) or glycoengineered
antibodies. Due to their smaller size (e.g.,
nanobodies), format (e.g., ADC), or pharmacology
(e.g., bi-specific, glycoengineered), the next
generation biologics present unusual nonclinical and
clinical challenges. Evaluation of the immunogenicity
responses to such molecules presents additional
challenges due to, for example, the need to identify
specificity of the anti-drug antibody responses to a
particular domain on the biotherapeutic molecule
and significance thereof. In addition, the new
antibody formats have biophysical features which
are different from the classical monoclonal full-size
antibodies or Fc fusion proteins, and thus constitute
new formulation challenges. This symposium will
provide overview of next generation biologics and
discuss general considerations for how to conduct
preclinical pharmacology and safety studies for
these novel unconventional biotherapeutics, how
to translate these findings into clinic, and how to
formulate and bioanalytically characterize these
molecules. Case examples and shared learnings will
be discussed.
MODERATORS
Vladimir Vexler, Ph.D.
Hoffmann-La Roche
Boris Gorovits, Ph.D.
Pfizer Inc.
Overview of Tandab Platform
Eugene Zhukovsky, Ph.D.
Affimed
In Vitro and In Vivo Evaluations to Aid
the Development of Novel Antibody Drug
Conjugates
Frank Barletta, Ph.D.
Pfizer Inc.
Overview and Comparison of
Immunogenicity Evaluation for a
Conjugated or Multi-domain vs.
Typical Biologic
Boris Gorovits, Ph.D.
Pfizer Inc.
Development of Therapeutic Monoclonal
Antibodies and Antibody Alternatives: An
FDA Perspective
Barbara Rellahan, M.S., Ph.D.
U.S. Food and Drug Administration
WEDNESDAY AFTERNOON SYMPOSIA
SUPPORTED BY AN EDUCATIONAL GRANT FROM
1:30 PM–4:00 PM
Biomarkers of CNS Function:
From Mice to Man
SYMPOSIUM
More in-depth understanding the biological
mechanisms underlying the pharmacokinetic- and
pharmacodynamic (PK-PD) relationships of CNS
active drugs is of importance for the development
of treatments with improved safety and efficacy
in CNS diseases. To that end, a mechanism-based
approach should be used that includes specific
expressions to describe target site distribution,
target binding, target activation, and signal
transduction. As for a CNS drug the target site
kinetics may be distinctively different from plasma
kinetics, it is important to obtain specific data on
this. This directly points to the problem that such
information cannot be obtained from humans. It is
anticipated that the use of translational approaches
provides the way to go. CNS target site kinetics
can be measured in rats using microdialysis, while
plasma biomarkers of specific CNS activity can be
obtained from both rats and human blood sampling.
Subsequent mechanism-based PK-PD modeling
can be applied to the rat data. Using allometric
scaling and independent information on the values
of biological system specific parameters as prior
knowledge, predictions of the human plasma
biomarker kinetics can be made. The validity of such
predictions can be tested on the basis of the data
obtained from humans.
MODERATORS
Elizabeth C. De Lange, Ph.D.
Leiden/Amsterdam Center for Drug Research
Donald E. Mager, Pharm.D., Ph.D.
University of Buffalo
2012 AAPS Annual Meeting and Exposition 33

BIOTECHNOLOGY (BIOTEC)
Scaling Pharmacodynamics from In Vitro
and Preclinical Animal Studies to Humans
Donald Mager, Pharm.D., Ph.D.
University of Buffalo
Quantitative Microdialysis to Assess
Target Site Kinetics and CNS Functionality
Biomarkers
Thomas Cremers, Ph.D.
Brains-OnLine LLC
Model-based Translation of Pharmacology
in Support of Rational Drug Discovery:
Principles, Challenges and Case Examples
Patrick Trapa, Ph.D.
Pfizer Inc.
Mechanism-based PK-PD Model for the
Prolactin Biological System Response
following a Dopamine Inhibition
Challenge: Quantitative Extrapolation
to Humans
Elizabeth C. De Lange, Ph.D.
Leiden/Amsterdam Center for Drug Research
1:30 PM–4:00 PM
From a Monomer to an Aggregate:
Models, Challenges and Case
Studies for Prediction of Protein
Aggregation In Solution
SYMPOSIUM
In protein solutions, the assembly of the monomer
over time, even under non-denaturing and
physiologically relevant solution conditions, is
one of the major concerns especially when it
results in the formation of irreversible aggregates.
Ensuring long-term stability of the protein in
solution against aggregation for the duration of
its shelf-life is critical and requires an in-depth
understanding of the multitude of variables involved
viz. protein concentration, solution conditions
including temperature, pH and excipients, surface
and interfacial stresses, impurities, processing
variables etc. While real-time storage stability data
under intended storage conditions is necessary
to justify the storage shelf-life for the commercial
product, methodologies for the prediction of the
aggregation propensity of a protein monomer in
solution are indispensable during preclinical and
clinical formulation development. To this end,
recent advances in sequence based analysis of
aggregation propensity and an understanding of
weak intermolecular interaction in protein solutions,
especially at high protein concentrations, have
shown immense promise in improving the current
capabilities for predicting protein aggregation in
solution. Moreover, under circumstances wherein
multiple formulation and process attributes need to
be optimized along with protein aggregation (e.g.
chemical stability, solution viscosity, appearance,
manufacturing process etc.), it’s just not enough
to characterize a formulation qualitatively with
respect to aggregation propensity. Under these
circumstances, a decent quantitative estimate
of the rates of aggregation under refrigerated
storage conditions must be made from high
temperature, and relatively faster, stress studies.
This session will focus on the predictive approaches
34
PRELIMINARY PROGRAM
and models currently available as well as being
developed for protein aggregation prediction.
The challenges associated with these approaches,
given the complexity of the protein aggregation
process, and the case studies highlighting the
utility of the predictive approach will be presented.
Specifically, the session will focus on: theoretical/
sequence based prediction of protein aggregation
propensity; prediction of aggregation rate: modeling
and kinetics; weak molecular interactions in high
concentration protein solutions as predictors of
protein aggregation; and real-life examples on
application of predictive tools in aggregation from
industry perspective (e.g. setting specifications and
regulatory aspects). The talks will address multiple
aspects of the issue of protein aggregation and
approaches to better understand the mechanism of
and predicting protein aggregation in solution.
MODERATOR
Atul Saluja, Ph.D., M.Pharm
Bristol-Myers Squibb Company
Predicting Aggregation-prone Regions of
Proteins through Molecular Modeling
Naresh Chennamsetty, Ph.D.
Bristol-Myers Squibb Company
Assessing the Relative Contributions of
Repulsive and Attractive Intermolecular
Interactions to Protein Aggregation in
Concentrated Solution
Allen P. Minton, Ph.D.
National Institutes of Health
From Dimers to Particles: Kinetics and
Non-specific Interactions
Chris J. Roberts, Ph.D.
University of Delaware
Significance of Unfolding
Thermodynamics in Predicting Protein
Aggregation Kinetics: A Case Study at
High Protein Concentration
Atul Saluja, Ph.D., M.Pharm
Bristol-Myers Squibb Company
WEDNESDAY AFTERNOON ROUNDTABLE
SUPPORTED BY A GRANT FROM
2:00 PM–4:00 PM
Emerging Platforms for
Immunogenicity and Quantitative
Assays
ROUNDTABLE
Several new technical platforms have been
introduced to the analytical field recently. These
platforms include, but are not limited to Gyros, fortebio,
Singulex, MSD and Chimera. New platforms are
often developed to overcome technical challenges,
provide improved efficiency, improved sensitivity, or
to offer the end-user greater flexibility. Wide-range
adoption of new technology can be hampered by
the significant monetary investment required for a
relatively unknown entity. Without the support of
published data, it can be difficult to make such an
investment. The importance of improved technology
cannot be overlooked as it may hold a key to
driving the industry forward by gaining efficiency
and improving the level and specificity of analyte
detection. The objective of this roundtable is to
more deeply explore recently emerging technical
platforms. Four different technical platforms will
be presented and for each, the presenter will share
experiences as they relate to immunogenicity and/
or quantitative assays. Speakers will briefly discuss
the underlying platform principle and share data
generated in their laboratory. The presenters will
also discuss what they have found to be both
benefits and challenges of the platform associated
with particular assay types. A brief presentation
on strategies for advancing new technologies
to regulatory agencies will also be included. The
remaining time will be reserved for an interactive
discussion among audience members and panelists.
MODERATOR
Valerie Theobald
Genzyme, A Sanofi Company
Advancing New Technologies through to
Agency Approval
Marian Kelley, M.S.
MKelley Consulting LLC
Application of Singulex and Gyrlob
Technologies to Immunogenicity and
Quantitative Analysis
Boris Gorovits, Ph.D.
Pfizer Inc.
LC/MS and Immuno-PCR Technologies
for Immunogenicity and Biotherapeutic
Quantitation Applications
An Song, Ph.D.
Genentech, Inc.

THURSDAY, OCTOBER 18
THURSDAY MORNING SUNRISE SESSIONS
7:00 AM–8:15 AM
Personalized Medicine: Bring Your
Birth Weight
SUNRISE SESSION
The emerging science of ‘developmental origins of
health and disease’ inversely correlates birth weight
to the adulthood incidence of metabolic syndrome.
Specifically, low birth weight is a strong predictor
of incidence of hypertension, hyperlipidemia,
diabetes mellitus, and obesity during adulthood.
Studies carried out in animal models mechanistically
link this inverse correlation to morphological
and physiological alterations in various organs
including kidneys and liver. Pharmacotherapeutic
strategies to treat metabolic syndrome, however,
do not consider alterations in kidneys and liver
which play an important role in determining drug
pharmacokinetics. This sunrise session will focus on
presenting data from literature on morphological
and physiological changes in various organs and
how those changes could impact pharmacokinetic
processes and subsequently pharmacotherapies.
The first speaker will cover the existing literature
data from human subjects. The second speaker
will present animal model data from literature as
well as their own laboratory relevant to the field of
developmental origins of health and disease. Birth
weight is an economical and accessible marker. In
conjunction with other patient-specific data such as
body weight, height, BMI, ethnicity, race, genotype,
etc., birth weight will inform researchers in the
field and healthcare providers to design optimal
pharmacotherapeutic strategies.
MODERATOR
Ganesh Cherala, Ph.D.
Oregon State University
Fetal Programming, Health through Life
and Lifespan
David J. Barker, Ph.D., M.D.
University of Southampton
Fetal Programming and Clinical
Pharmacology
Julie R. Ingelfinger, M.D.
Harvard Medical School
Intrauterine Growth Restriction and Drug
Pharmacokinetics in Adulthood
Ganesh Cherala, Ph.D.
Oregon State University
7:00 AM–8:15 AM
Biosimilars Development:
CMC, Preclinical, and Clinical
Considerations
BIOTECHNOLOGY (BIOTEC)
SUNRISE SESSION
Biosimilars, also referred to as follow-on protein
products in the U.S., can be defined as biotech
drugs that have been shown to have comparable
quality, safety and efficacy to the original product.
Unlike traditional small molecule generics, the
task of developing and approving a follow-on
biologic is very challenging as a number of CMC,
preclinical and clinical considerations need to be
taken into account including: biosimilars are larger
and more complex molecules with associated
structural heterogeneities and therefore cannot
be completely characterized analytically; minor
changes in manufacturing process have been
known to cause significant change in efficacy or
immunogenicity of the drug in the clinic; and the
exact manner in which the numerous product
quality attributes of a biosimilar impact the
safety and efficacy of the product in the clinic
is generally not known completely. In particular,
immunogenicity assessments of novel and biosimilar
products still heavily depend on clinical studies.
Capital investments (including the operating costs)
associated with manufacturing of biosimilars along
with the risk of failure for biosimilars are significantly
higher than that for small molecule generics. The
result is a relatively smaller discount for biosimilars
compared to the small molecule generics. These and
other development challenges will be addressed in
this session.
MODERATOR
Kavitha Koushik, Ph.D.
Alvogen Inc.
Biosimilars: Regulatory Challenges
Vijay Tammara, Ph.D.
Nuron Biotech
Characterization of Biopharmaceuticals or
Follow-on Biologics
William Bakewell, Ph.D.
PPD Development, LLC
THURSDAY MORNING ROUNDTABLE
9:00 AM–11:00 AM
Problems and Solutions for
Manufacturing Therapeutic
Proteins
ROUNDTABLE
The talks and panel discussion in this session will be
focused on a variety of challenging problems and
solutions for practical production of therapeutic
proteins, including monoclonal antibodies and other
protein drugs. These will include, but not limited to,
the new production technologies to more efficiently
produce the proteins in large-scale, to thermally
stabilize the protein drugs, and to extend their
circulatory half-lives.
MODERATOR
Chang-Guo Zhan, Ph.D.
University of Kentucky
Challenges for Manufacturing Therapeutic
Proteins
Chang-Guo Zhan, Ph.D.
University of Kentucky
2012 AAPS Annual Meeting and Exposition 35