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 


7:00 AM–8:15 AM 

Personalized Medicine 101 

SUNRISE SESSION 

8:30 AM–11:00 AM 

Graduate Student Symposium in 

Biotechnology (BIOTEC) 


TUESDAY MORNING ROUNDTABLES 


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? 


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 


Biosimilars Development: CMC, Preclinical, 

and Clinical Considerations 


9:00 AM–11:00 AM 

Problems and Solutions for Manufacturing 

Therapeutic Proteins 

ROUNDTABLE

MONDAY, OCTOBER 15 

MONDAY MORNING ROUNDTABLE 


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. 


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 



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


TUESDAY, OCTOBER 16 

TUESDAY MORNING SUNRISE SESSION 


7:00 AM–8:15 AM 

Personalized Medicine 101 


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) 


32 


TUESDAY MORNING ROUNDTABLE 


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 


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 



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. 


Li Zhang, Ph.D., M.D. 



Shashi Amur, Ph.D. 


Progress in PGx and its Promise for 

Medicine 

Li Zhang, Ph.D., M.D. 


WEDNESDAY MORNING SYMPOSIUM 

8:30 AM–11:00 AM 

Next Generation Biologics 


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 


Pfizer Inc. 

Development of Therapeutic Monoclonal 

Antibodies and Antibody Alternatives: An 

FDA Perspective 

Barbara Rellahan, M.S., Ph.D. 


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 



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 


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 


Predicting Aggregation-prone Regions of 

Proteins through Molecular Modeling 

Naresh Chennamsetty, Ph.D. 


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 


WEDNESDAY AFTERNOON ROUNDTABLE 


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 


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 


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 



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

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