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IBC’s 18th Annual
Well Characterized
Biologicals
15 Confirmed
Regulatory
Speakers
Innovative Strategies and Regulatory Guidance
for Characterizing Biological Products and
Identifying and Controlling Variants and Impurities
November 3-4, 2014
Washington Marriott at Metro Center
Washington, DC
Ensure Accurate CMC Submissions by:
Overcoming regulatory challenges by hearing guidance from FDA,
Health Canada, US Pharmacopeia, NIST, NIH and the International Serum
Industry Association
Achieving greater product characterization and lifecycle
management by hearing practical case studies and characterization
strategies from industry leaders
Accelerating your project performance by hearing in-depth updates on
recent characterization projects and emerging analytical technologies
Enhancing process and product quality by learning about new testing
methods to identify and control variants & impurities
www.IBCLifeSciences.com/WCB
MEDIA
PARTNERS:
PREMIER
PUBLICATION:
ORGANIZED
BY:
The Industry’s Leading Conference to Help You Characterize
Biological Products and Identify/Control Variants & Impurities
For nearly two decades, Well Characterized Biologicals has been
regarded as a must-attend event to get the latest updates on the most
novel bioanalytical methods for sound characterization of biologics,
and more recently, to identify and effectively control variants and
impurities for optimal processing and product quality.
At Well Characterized Biologicals, you can to interact with regulators
and hear their perspectives on guidance for industry and insights
on how it is best applied. You will also benefit from compelling case
studies that explore new protein characterization and detection
methods for variants and impurities.
And with two-days of presentations, many of which are chock full of
new data, you will leave prepared with applicable knowledge to meet
characterization expectations of increasingly complex modalities,
while improve your overall CMC, product and process bottom line.
Who Should Attend
Send a team to capitalize on this unique opportunity to meet faceto-face with multiple regulators. Benefit from hearing unpublished
data that will only be shared in person at this meeting. The practical
advice and industry case studies at this event will benefit Scientists,
Technicians, Managers, Lab Heads, Directors and other specialists in
the following departments:
• Analytical R&D
• Product Development
• CMC
• Protein Analytical Chemistry
• Technical Operations/
Manufacturing Sciences
• Bioassay Development
• Drug Product Manufacturing
• Regulatory Affairs and QA/QC
• Clinical Supply Chain
Procurement
• Process Development
Group rates available for companies registering 4+, see page 9 for details
Showcase Your Research ➜ Present a Poster
If you have new results/data on topics relevant to this
conference, we encourage you to submit a poster abstract for
consideration. Any registered conference attendee may register
to present a poster. The deadline to submit an abstract online
is Friday, Oct. 24, 2014. Full payment of conference registration
and poster fees must be received by this date for the abstract
to be included in the conference materials and a poster board
assignment to be made (see the registration page for details on
the poster fee). Posters should be PORTRAIT orientation, with
maximum dimensions of 36 inches wide (3 feet) x 48 inches high
(4 feet). Please note: Poster presentations may not be used as
exhibit displays or for marketing purposes, and all posters are
subject to approval by conference organizers. Only one poster
presentation is allowed per registered attendee/author.
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2
Monday, November 3, 2014
7:30 Registration and Coffee
8:25 Opening Remarks
Keynote Presentations
8:30 FDA PERSPECTIVE
New FDA Draft Guidance on Analytical Procedures and Analytical Method Validation
for Drugs and Biologics
CDER and CBER recently collaborated to create a new Guidance for Industry document entitled “Analytical Procedures and Methods Validation
for Drugs and Biologics” which is published as a draft in February, 2014. This guidance was designed to update agency thinking on requirements
for regulatory submission of analytical procedures and method validation data to support the documentation of the identity, strength, quality,
purity, and potency of drug substances and drug products. The draft guidance supersedes the 2000 draft guidance for industry on Analytical Procedures
and Methods Validation and, when finalized, will also replace the 1987 FDA guidance for industry on Submitting Samples and Analytical Data for Methods
Validation. The agency also wanted to ensure this draft guidance would complement the International Conference on Harmonization (ICH) guidance
documents, especially Q2 (R1) Validation of Analytical Procedures: Text and Methodology (Q2(R1)) for developing and validating analytical methods. In
addition to assuring that guidance documents are current, the need for an updated guidance stems from the need to ensure analytical methodologies are
consistent with changes to drug development and regulation seen with the concepts outlined in ICH Q8 (R2), Q9, Q10 and Q11.
Lokesh Bhattacharyya, Ph.D., Lab Chief, Laboratory of Analytical Chemistry and Blood Related Products, Division of Biological Standards and Quality
Control, CBER, U.S. FDA
9:00
NEW DATA
New USP Chapter for N-glycan Analysis: <212> Oligosaccharide Analysis
USP has proposed a new General Chapter <212> in Pharmacopeia Forum 40(5) to provide qualitative analysis primarily for N-glycan chains
with no or low levels of sialylated structures. In the meantime, an analytical procedure for analysis of multi-sialylated N-glycans is also
developed. Total of four reference standards will be used to assess the system suitability for these analytical procedures. Details of this
chapter are presented.
Edith Chang, Ph.D., Scientific Liaison, Biologics and Biotechnology, US Pharmacopeia
9:30
Technology Workshop
High-throughput and Validated Binding Kinetics Assay using Octet RED96 to Support Phase III
Drug Product Release and Stability in Quality Control
Potency is an critical quality attribute in monoclonal antibody manufacturing and quality control. Binding kinetics assay measuring antigen-antibody
interactions was originally evaluated in our facility using Surface Plasmon Resonance by Biacore. However, Biacore assay could not meet our needs with
increased testing volume from Manufacturing scale-up when we began FDA-approved Pivotal Phase III clinical trial for Cachexia. We developed an innovative
kinetics assay using ForteBio Octet RED96. Validation of this new method showed a precision of 17%, and it is also specific, accurate and robust enough to use
as a QC drug product release and stability assay. The number of samples one operator can test per day using the new Octet method is significantly increased
(8-fold) compared to the Biacore assay. Beside binding kinetics assay, we also use Octet RED96 to monitor cell culture titer in Manufacturing, which is also a
validated QC assay.
Qian Wu, Ph.D., Director of Quality Control, XBiotech USA, Inc.
10:00 Networking Refreshment Break in Poster and Exhibit Hall
TRACK A
10:40 Chairman’s Opening Remarks
Yves Aubin, Ph.D., Research Scientist, Centre for Vaccine Evaluation,
Biologics and Genetic Therapies Directorate, Health Canada
Comparability Strategies for Biosimilars and
Other Biotechnology Products
10:45 NEW DATA
Characterization Strategies for
Carbohydrate Biosimilars
The analysis of the glycoconjugates has become important for all
comparability studies and in the quality control of therapeutic recombinant
glycoproteins and polysaccharides. We will describe challenges in
chromatography and mass spectrometry methods and procedures that
are used for polysaccharide and glycoprotein biosimilar analysis in order to
obtain regulatory approval. Details will be given on the pros and cons of
certain methods from regulatory stand point. We will discuss procedures
necessary for the complete structural elucidation of heparin, low molecular
weight heparin and antibody products. Challenges in comparability study of
low molecular heparin will be discussed. Examples of the methodologies will
include data needed in different phases and currents analysis of biosimilars.
Parastoo Azadi, Ph.D., Technical Director, Complex Carbohydrate
Research Center, University of Georgia
TRACK B
10:40 Chairman’s Opening Remarks
Jonathan W. Cooper, Ph.D., Staff Scientist, Analytical Development,
Vaccine Production Program Laboratory, Vaccine Research Center,
NIAID, NIH
Assay Development Strategies for Vaccines
and Other Therapeutics
10:45 FDA PERSPECTIVE
NEW DATA Considerations for Use
of Serologic Assays in Vaccine Development for
Bacterial Diseases
The US Food and Drug Administration, Center for Biologics Evaluation
and Research, Office of Vaccines Research and Review (OVRR)
evaluates the safety and efficacy of vaccines for therapeutic and
vaccine preventable diseases in the U. S. Serologic assays may be
used as correlates of protection to clinically evaluate effectiveness
of a vaccine when disease cannot be measured. This presentation
will review why, when, and how it is appropriate to develop serologic
assays. Meningococcal vaccines (Neisseria meningitis) with use of the
serum bactericidal activity (SBA) assay, and pneumococcal vaccines
(Streptococcus pneumoniae) with the use of opsonophagocytic assay
(OPA) will be presented as examples.
Cara Fiore, Ph.D., Microbiologist, Master Reviewer, Division of
Vaccines and Related Products Applications OVRR, CBER, U.S. FDA
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3
Monday, November 3, 2014 (continued)
TRACK A
11:15 NEW DATA
Application of NMR Spectral
Fingerprinting for Structure Assessment of
Monoclonal Antibody Therapeutics
High-resolution NMR methods can provide spectral 'fingerprints' of the
higher order structure of protein therapeutics at atomic resolution.
This presentation will report on the application of NMR methods for
obtaining NMR 'fingerprints' of monoclonal antibody therapeutics and
how these spectral 'fingerprints' can be used to establish consistency
in drug manufacturing and for comparing a biosimilar to an innovator
reference product.
John P. Marino, Ph.D., Leader, Biomolecular Structure & Function
Group, NIST
TRACK B
11:15 Potency Assay Strategies for Bispecific Antibodies
Abstract Not Available. Please check www.IBCLife Sciences.com/WCB
for updates.
Kendall Carey, Ph.D., Scientist, Biological Technologies, Genentech, Inc.
Regulatory Considerations in the
Safety Assessment of Vaccine Adjuvants and
Adjuvanted Vaccines
11:45 FDA PERSPECTIVE
The Office of Vaccines Research and Review (OVRR) is responsible
for regulatory review of new Investigational New Drug Applications
(INDs) and Biologics License Applications (BLAs) for preventive vaccines
and therapeutic vaccines for infectious disease indications. Through
this review process, OVRR ensures that vaccines are safe, effective,
pure, and potent, as specified in Title 21 of the Code of Federal
Regulations, Sections 312, 600, and 610. This presentation will review
the key components in nonclinical and clinical safety assessment of
investigational vaccines regulated by OVRR, with a focus on product
testing and characterization and pharmacology and toxicology study
design considerations for vaccines with novel adjuvants. In addition,
updates on clinical trial design considerations, including demonstration
of the “added benefit” of the adjuvant, adjuvant dose selection and
safety monitoring will be discussed briefly.
Kirk C. Prutzman, Ph.D., Office of Vaccines Research and Review,
Division of Vaccines and Related Products Applications, Regulatory
Review Branch 3, CBER, U.S. FDA
11:45 CASE STUDY
NEW DATA Monitoring Effects of Excipients,
Formulation Parameters and Mutations on the High
Order Structure of Filgrastim by NMR
Filgrastim is the generic name for recombinant methionyl human
granulocyte colony-stimulating factor (r-metHuG-CSF). It is produced in
E. coli in a non-glycosylated form. Here we show that NMR spectroscopy
can be used to assess the three-dimensional structure of the active
ingredient of formulated biosimilars. Recombinant metHuG-CSF was
prepared in E. coli and isotopically enriched with 13C and 15N isotopes
to study the effects of excipients such as pH, sorbitol, polysorbate-80,
and ionic strength, on the conformation. Also, similar? Identical?
NMR spectra were recorded for Neupogen®, a commercially available
r-metHuG-CSF product purchased at a local pharmacy, thus showing
that our labelled filgrastim has an identical conformation to this
product. Therefore, NMR does provide residue specific information
of the structure of the active ingredient of a product. In addition to
current methods, the ability to assess the conformation with a high
degree of resolution can greatly facilitate the comparability exercise.
Yves Aubin, Ph.D., Research Scientist, Centre for Vaccine Evaluation,
Biologics and Genetic Therapies Directorate, Health Canada
12:15 Networking Luncheon in Poster and Exhibit Hall
1:25
1:25
Chairwoman’s Opening Remarks
Cara Fiore, Ph.D., Microbiologist, Master Reviewer, Division of
Vaccines and Related Products Applications OVRR, CBER, U.S. FDA
Assay Development Strategies for Vaccine and
Other Therapeutic Development
Chairman’s Opening Remarks
Featured Presentation
John P. Marino, Ph.D., Leader, Biomolecular Structure & Function
Group, NIST
1:30 CASE STUDY
Analytical Methods and
Characterization Strategies
1:30
12:15 Networking Luncheon in Poster and Exhibit Hall
Multi-Parameter Characterization of Biologics
as a Tool for Aiding Development and
Supporting Similarity
Characterization of proteins is essential to support claims of
comparability or superiority in the development of follow on biologics.
A fingerprinting approach that allows systematically building up
a library of biophysical stability, conformation and aggregation
information is demonstrated using a spectroscopic method typically
used in the application of high throughput screening. Using a variety
of orthogonal probes information can be determined about the way
different proteins interact with each other under different conditions.
In this presentation a family of data-rich instruments are used to
develop a characteristic fingerprint of a therapeutic monoclonal
antibody, providing the ability to gain detailed characterization
information that helps control protein folding and aggregate formation
while also providing a risk based assessment of the likelihood of
particular formation under various conditions.
Daniel Lund, Ph.D., Product Manager, Avacta Analytical
NEW DATA Fab-Arm Exchange of IgG4
Therapeutic Antibodies In Vitro and In Vivo: An
Industry Perspective
IgG4 isotype antibodies are prone to engage in Fab-arm exchange, a
structural rearrangement that involves the swapping of a heavy-light
chain unit between IgG4 molecules, resulting in bispecific hybrids in
vitro and in vivo. A simple point mutation (S228P) at the core hinge
region has been shown to be sufficient to stabilize the hinge disulfide
bridges and prevent Fab-arm exchange from occurring. Propensity to
engage in Fab-arm exchange is considered a potential critical quality
attribute and thus needs to be tightly monitored during manufacturing
and at the Drug Substance and Drug Product stages. This work will
describe a strategy to monitor the presence of Fab arm exchange using
multiple techniques both in vitro and in vivo.
Daisy Richardson, Ph.D., Director, Bioprocess Development, Merck
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4
Monday, November 3, 2014 (continued)
TRACK A
2:00
High Throughput and High Resolution Glycan
Analytics Using Multi-Capillary CE
Glycans represent a key quality attribute of biotherapeutics that requires
analysis during several points in development and manufacturing. Key
components of glycan analytics include sample preparation, glycan
separation and data analysis. Current glycan methods are hampered by a
laborious and time consuming sample preparation workflow coupled with a
low throughput and/or low resolution data generating system. To address
this analytical challenge, the sample preparation has been greatly simplified
through a reduction in hands on time, pipetting and manipulations.
Combined with analysis on a 24 capillary CE system, high quality relative
quantitation glycan data for 96 samples can be generated from start to
finish in less than 7 hours. This presentation describes the technology
behind this method, with time to result, ease-of-use, reproducibility,
sensitivity and benchmarking against existing glycan analysis platforms.
Wesley Straub, Senior Product Manager, BioProduction, Pharma
Analytics, Life Science Solutions, Thermo Fisher Scientific
2:30
TRACK B
Platform Assays to Support Process
Development, Release, and Characterization of
Virus-Like Particles for Western, Eastern, and
Venezuelan Equine Encephalitis Virus
2:00 NEW DATA
The Vaccine Research Center at NIAID is developing a virus-like particle
vaccine to protect from Western, Eastern, and Venezuelan equine
encephalitis viruses due to their possible use as biological agents.
Strategies for platform assays for CQAs, content, potency, purity, and
residuals to support process development, release, and characterization
for GMP production of the three VLPs will be discussed.
Jonathan W. Cooper, Ph.D., Staff Scientist, Analytical Development.
Vaccine Production Program Laboratory, Vaccine Research Center,
NIAID, NIH
2:30 FDA PERSPECTIVE
Strategies of Potency
Determination for New Generation Blood Products
Accurate and meaningful determination of potency is essential for
the safe and effective use of blood products. Although most blood
protein products are well characterized and international standards
of their biological activity are often available, recent investigations
have revealed discrepancies in potency assignment and post-infusion
monitoring for both licensed and investigational coagulation factor
products, especially the new generation products whose structures
are modified in an attempt to improve treatment regimen. This
lecture will discuss the issues related to potency assignment and
post-infusion monitoring of these products, and strategies to
address the problems.
NEW DATA Discovery and Characterization of a Novel
Post-Translational Modification in Recombinant
Monoclonal Antibodies
Abstract Not Available. Please check www.IBCLife Sciences.com/WCB
for updates.
Richard Beardsley, Scientist, Protein Analytical Chemistry, Genentech
3:00 Networking Refreshment Break in Poster and Exhibit Hall
3:40
Chairman’s Opening Remarks
Richard Beardsley, Scientist, Protein Analytical Chemistry, Genentech
Analytical Strategies for Diverse Products
Disclaimer: This is an informal communication and it represents authors’ own best
judgment. These comments do not bind or obligate FDA.
3:45 CASE STUDY
Mikhail V. Ovanesov, Ph.D., Visiting Scientist, Principal Investigator,
Laboratory of Hemostasis/Division of Hematology, Office of Blood
Research and Review, CBER, U.S. FDA
3
4:15
Monoclonal Antibody
Reference Material for the Advancement of
Characterization Technologies
NEW DATA
Therapeutic monoclonal antibodies harness the highly evolved specificity of
adaptive immunity to fight disease. Monoclonal antibody-based therapeutics
have grown exponentially with the advent of mammalian cell culture,
process, and formulation technology. At the same time, state-of-the-art and
emerging analytical and biophysical methodology provides very detailed
process and product information. Although such a battery of methodology
and wealth of information is critical to product understanding; the
accuracy, precision, robustness, and suitability of such techniques are also
of critical importance. A representative and widely available material along
with detailed historical data would greatly supplement characterization
efforts throughout the drug product lifecycle. To this end, a first-of-its
kind qualitative and quantitative biopharmaceutical Reference Material
to supplement drug substance/product characterization is described. The
NIST mAb IgG1κ is intended to provide a well characterized, longitudinally
available test material that is expected to greatly facilitate development
of originator and follow-on biologics for the foreseeable future. The RM
is intended for a variety of uses including, but not necessarily limited to:
system suitability tests, establishing method or instrument performance
and variability, comparing changing analytical methods, assisting in method
qualification, etc. Results from an ongoing round robin characterization
study and book project including over 65 contributors from industry,
academia, and regulatory agencies will also be discussed.
John Schiel, Ph.D., Research Chemist, National Institute of
Standards and Technology
Measuring Trisulfides in Biologics: Methods
for High Throughput Analysis of mAbs and Antibody
Drug Conjugation Processes
NEW DATA
A trisulfide bond is a previously known protein modification that was
only recently found in recombinant monoclonal antibodies using massspectrometry. This modification introduces heterogeneity into the interchain disulfide linkages and can impact the reduction step involved in the
manufacturing of cysteine-linked antibody-drug conjugates (ADCs). Several
analytical methods with higher throughput than the original MS strategy have
been developed to quantitate this product variant. These methods can be used
to study trisulfide formation and to mitigate its impact on ADC manufacturing.
Chris R. Cornell, MSc., Technical Development Senior Research
Associate, Protein Analytical Chemistry 3, Genentech Inc.
3:00 Networking Refreshment Break in Poster and Exhibit Hall
3:40
Chairman’s Opening Remarks
Dong Xu, Ph.D., Scientist II, Analytical Development, Technical
Development, Biogen Idec
Assay Development Strategies for Vaccines
and Other Therapeutics
Featured Presentation
3:45
Characterization of Serum: Possibilities
and Probabilities
This talk will introduce the International Serum Industry Association
and provide an overview of history and areas of focus. Current
programs around standardization of testing and traceability of origin
and serum type will be discussed. The impact of new testing methods
will be reviewed and the role going forward assessed.
Rosemary J. Versteegen, Ph.D., Chief Executive Officer,
International Serum Industry Association
4:15
Reagent Management for Bioassay Development
Group at Biogen Idec, Inc.
A reagent management system based on commercial software was
established for bioassay development. Efforts to minimize the risk of
mistaken information and provide users with secured and convenient
accessibility to the program are discussed. In addition, the procedures
and challenges associated with reagent production by CRO/CMOs are
summarized for improvement on cost and time efficiency.
Dong Xu, Ph.D., Scientist II, Analytical Development,
Technical Development, Biogen Idec
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5
Monday, November 3, 2014 (continued)
TRACK A
Predicting the Stability of
Lyophilized Protein Formulations by Fluorescence
Stokes Shift
4:45 CASE STUDY
NEW DATA
Disaccharides such as trehalose and sucrose are commonly used to
preserve therapeutic proteins in lyophilized formulations; however, there
is currently no systematic and rational approach to predict the stability of
protein formulations. We have shown firm evidence that protein stability
in sugar-based glasses is directly linked to the high-frequency, local
mobility of the sugar matrices, occurring on a timescale of nanosecond.
In this talk, we present a new fluorescent instrument to probe the
motions of lyophilized formulations. This technique will allow scientist
in early development stage to rapidly screen and select suitable protein
formulation candidates, and predict their long-term stabilities.
Ken K. Qian, Ph.D., Research Chemist, Biomaterials Group, NIST
5:15
Regulatory Challenges for Protein Therapeutics
Common regulatory challenges exist among protein therapeutics but
there are unique concerns for novel molecules. This talk will present
specific and general concerns for innovative non-mAb therapeutics which
are becoming increasingly more important and are now in preclinical
pipelines. Finally, the presentation will conclude with a case study
highlighting a fusion protein which utilizes a nonstandard manufacturing
process, illustrating the challenges with determining specifications,
comparability and process validation for a novel molecule.
John Alvino, Senior Manager, Global Regulatory Affairs,
AstraZeneca
5:45 Close of Day One
TRACK B
4:45 CASE STUDY
NEW DATA Improvement of a Sample
Pretreatment Procedure for Immunogenicity Testing
through Better Characterization of Cells, Positive
Control Antibodies and Residual Drug Level
Biological therapeutics can induce an undesirable immune response
resulting in the formation of anti-drug antibodies (ADAs), which can
include neutralizing antibodies (NAbs). Functional (usually cell-based)
assays are preferred to detect NAbs in patient serum samples, but
cell-based bioassays are subject to interference from numerous serum
factors such as growth factors, disease-related cytokines, as well as free
drug. In this case study, a biotin extraction and acid dissociation (BEAD)
procedure to isolate ADA was significantly improved through better
characterization at multiple steps. First, we show that the cells used
in the bioassay were extremely sensitive to a wide range of commonly
used buffers in the acid extraction procedure, namely, Tris, glycine,
acetic acid and citrate. Two different methods were identified to make
the final extraction buffer compatible with even the most sensitive
cell lines in the bioassay. We then discuss why some excellent NAb
positive controls (PCs) could go awry upon acid treatment and propose
a strategy for ADA or NAb PC characterization when they are intended
to be used in acid extraction. Last, we show how LC/MS-MS played
an instrumental role in the optimization of the extraction procedure
by monitoring residual human monoclonal Ab drug levels in the final
extraction, which contains an excess amount of endogenous human
Abs. Lessons learned through this case study enabled the development
and optimization of a universal ADA extraction method to minimize
both drug and matrix interference for downstream ADA assay or
functional cell-based NAb assays.
Weifeng Xu, Ph.D., Research Investigator, Bioanalytic and Analytic
Science - Biologics (BAS-B), Bristol-Myers Squibb
5:15 Systematic Evaluation of In Vitro and In Vivo
Adventitious Virus Assays for the Detection of Viral
Contamination of Cell Banks and Biological Products
“Always well worth the
ticket price. Excellent industry and
agency participation.”
- Jackie McGourty, Emergent BIosolutions
Viral vaccines and the cell substrates used to manufacture them
are subjected to tests for adventitious agents, including viruses,
contaminate. Some of the compendial methods (in vivo and in vitro in
cell culture) were established in the mid-20th century. These methods
have not been subjected to current assay validation, as new methods
would need to be. This study was undertaken to provide insight into the
breadth (selectivity) and sensitivity (limit of detection) of the routine
methods, two such validation parameters. Sixteen viral stocks were
prepared and characterized. These stocks were tested in serial dilutions
by the routine methods to establish which viruses were detected by
which methods and above what limit of detection. Sixteen out of
sixteen viruses were detected in vitro, though one (bovine viral diarrhea
virus) required special conditions to detect and another (rubella virus)
was detected with low sensitivity. Many were detected at levels below
1 TCID50 or PFU (titers were established on the production cell line in
most cases). In contrast, in vivo, only 6/11 viruses were detected, and
4 of these were detected only at amounts one or more logs above
1 TCID50 or PFU. Only influenza virus and vesicular stomatitis virus
were detected at lower amounts in vivo than in vitro. Given the call to
reduce, refine, or replace (3Rs) the use of animals in product safety
testing and the emergence of new technologies for the detection
of viruses, a re-examination of the current adventitious virus testing
strategies seems warranted. Suggested pathways forward are offered.
Jim Gombold, Senior Director, Charles River Laboratories
5:45 Close of Day One
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6
Tuesday, November 4, 2014
7:30 Registration and Coffee
8:25
Opening Remarks
Keynote Presentations
Regulatory Considerations
for Adventitious Agents and Host
Contaminants in Vaccines
8:30 FDA PERSPECTIVE
This talk covers ensuring vaccine safety; mitigation of
adventitious agent risk; reduction of residual host cell
material in final product: Cellular DNA and Protein;
manufacturing challenges and control measures; designing product
safety; and regulatory challenges.
Santosh Nanda, Ph.D., Interdisciplinary Scientist, CBER, U.S. FDA
9:00 FDA PERSPECTIVE
Protein Aggregates and Other
Particulates: Testing Methodologies and
Regulatory Issues
Abstract Not Available. Please check www.IBCLife Sciences.com/WCB
for updates.
Joseph Kotarek, Ph.D., ORISE Fellow, Office of Blood Research and
Review, Division of Hematology, Laboratory of Plasma Derivatives,
CBER, U.S., FDA
9:30 FDA PERSPECTIVE
Characterization of Conjugate Vaccines
from a Regulatory Perspective
Abstract Not Available. Please check www.IBCLife Sciences.com/WCB
for updates.
Tina Roecklein, M.S., Consumer Safety Officer, OVRR, DBPAP, CBER U.S. FDA
10:00 Networking Refreshment Break in Poster and Exhibit Hall
TRACK A
TRACK B
Identifying and Controlling Variants & Impurities
QbD for Development
10:40 Chairman’s Opening Remarks
Justin Sperry, Ph.D., Senior Principal Scientist, Analytical Research
and Development, Senior Scientist, Pfizer Inc.
10:40 Chairman’s Opening Remarks
Thomas A. Little, Ph.D., President, Thomas A. Little Consulting
NEW DATA In-depth Examination of
Interferon A-2 Products Using High-Resolution
Separation Techniques
10:45 CASE STUDY
Interferons (IFNs) are some of the most frequently prescribed therapeutic
proteins for the treatment of diseases such as hepatitis, multiple
sclerosis and leukemia. Like other therapeutic proteins, however, IFNs are
susceptible to a number of process and product-related modifications
during preparation, formulation or storage. Such modifications may result
in the loss of therapeutic efficacy or unwanted immune reactions. We have
examined IFNα-2a and IFNα-2b products using methods based on high
resolution separation techniques, in particular, capillary electrophoresis
(CE) and high performance liquid chromatography (HPLC). Size-exclusion
HPLC was used to assess the formation of dimers, aggregates and
denatured IFNα-2. Ion-exchange chromatography as well as a CE enabled
monitoring charge variants while reversed-phase HPLC enabled detection
of oxidized products and other variants. In addition, a CE method was
developed to directly assess the integrity of the active ingredient in
finished products containing excipients such as human serum albumin or
polysorbate. A brief overview of these methods will be presented.
Michel Girard, Ph.D., Research Scientist, Centre for Vaccine Evaluation,
Biologics and Genetic Therapies Directorate, Health Canada
11:15 USP
Analytical Methods and Monograph Approaches
for Measuring Impurities in Biologics
USP’s USP-NF contains General Chapters and monographs that support
the quality, safety, and potency of drug substances and products. This
talk will explain the strategies used to prepare suitable monographs
containing impurity tests for biologics from multiple manufacturers. The
talk will also highlight new test Chapters containing validated methods
appropriate for many biologics. (PPVI)
Maura C. Kibbey, Ph.D., Senior Scientific Liaison, Biologics &
Biotechnology, U.S. Pharmacopeia
11:45 Assessment
of Small Molecule Impurities in
Antibody Drug Conjugates
10:45 FDA PERSPECTIVE
QbD Approaches to
Product Development
Summarizing case studies of where QbD filings have resulted in
reduction of regulatory burden; from a regulatory perspective, how
much data is needed to submit your filing.
Steve Kozlowski, MD, Director, Office of Biotechnology Products
(OBP), CDER, U.S. FDA (Invited)
11:15 Essentials
in Quality by Design for Modern
Biologics Development
QbD is a systematic approach to development that begins with
predefined development objectives and emphasizes product and
process understanding and process control, based on sound science,
data based decision making and quality risk management. Quality
by Design as introduced by the FDA and EU brings modern drug
development methodologies to CMC teams working on biologics,
pharmaceuticals and vaccines. The presentation will discuss the
application of QbD principles in the development, submission and
manufacturing of drug product and drug substance. Most CMC
development teams globally have little to no experience in generation
of design space, selection of PAR/NOR ranges and preparing for Stage I
Validation documentation and submission. This presentation will cover
basic and advanced principles for the practical application of QbD in
every phase of product development and control.
Thomas A. Little, Ph.D., President, Thomas A. Little Consulting
11:45 Practical
Application of QbD for Method
Development, Validation and Process Control
Abstract Not Available. Please check
www.IBCLife Sciences.com/WCB for updates.
Andrea Coombs, Director Validation and Technical Transfer,
Emergent Biosolutions
Currently there is little to no regulatory guidance for the treatment
of small molecule impurities in an antibody drug conjugates. The
International Consortium for Innovation and Quality in Pharmaceutical
Development (IQ) has developed a scientific rational that addresses this
gap. The IQ’s recommendation along with a case study will be presented.
Michael T. Jones, Ph.D., Research Fellow, Biotherapeutics
Pharmaceutical Sciences, Analytical R&D, Pfizer
12:15 Networking Luncheon Break in Poster and Exhibit Hall
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Tuesday, November 4, 2014 (continued)
TRACK A
1:30
NEW DATA Control of Process Variants in Novel
Vaccine Candidates
The insect cell-Baculovirus Expression Vector System (BEVS) is an
established platform for the production of biological products and
is used in the manufacture of commercial vaccines for human and
veterinary use. Using BEVS, new viral antigens can be produced rapidly,
without the need to produce live viral pathogens and without the
need to qualify new cell banks for production of recombinant antigens.
Applications of BEVS technology beyond the currently licensed products
along with next generation products will be reviewed. Process variants
and their control in the BEVS/insect cell system will discussed in the
context of new vaccine candidates.
Clifton E. McPherson, Ph.D., Vice President, Product Development,
Protein Sciences Corporation
TRACK B
1:25
Thomas A. Little, Ph.D., President, Thomas A. Little Consulting
QbD for Development Cont.
1:30 CASE STUDY
NEW DATA Establishing the Criticality of
the Quality Attributes of an Fc Fusion Protein
One of the first steps in the QbD approach consists in identifying
the Critical Quality Attributes (CQA), i.e. those quality attributes
of the product that have an impact on its clinical efficacy and/or
safety. According to ICH guidelines Q8/Q11, manufacturing process
development should include at a minimum a number of elements, of
one which is CQAs. CQAs are therefore expected in the submission for
a commercial pharmaceutical product.
The presentation describes how the potential CQAs of an Fc fusion
protein were selected and covers the following steps:
Host Cell Protein Detection and Characterization
2:00
2:30
• Overview of the quality attributes of recombinant proteins
expressed in mammalian cells
NEW DATA Characterization of Host Cell Proteins
Using Mass Spectrometry
Sensitive detection and quantitation of residual host cell proteins (HCPs)
during purification process development is critical in the design of robust
and well-controlled manufacturing processes that yield high quality
biotherapeutics. The enzyme-linked immunosorbent assay (ELISA) is the
current standard assay for determining residual HCP levels in the purified
biotherapeutic drug substance. Mass spectrometry-based methods are
emerging as a routine approach for HCP analysis where residual HCPs can
be detected, identified, and quantitated directly due to ever increasing
instrument performance. In this study, we have developed proteomic
approaches to identify and quantify residual HCPs in biotherapeutics
derived from both mammalian and bacterial expression systems in an
effort to complement ELISA results. Spiking studies were employed to
determine the limits of quantitation and detection for a wide variety of
possible HCPs. The proteomic method employs proteolytic digestion,
one dimensional chromatographic separation by RP-HPLC, ultrahighresolution mass spectrometry, and database searching to definitively
identify potential HCPs. A comparison of analytical approaches for HCP
detection and quantitation will be discussed.
Justin Sperry, Ph.D., Senior Principal Scientist, Analytical Research and
Development, Senior Scientist, Pfizer Inc.
Late-Breaking Presentation
Chairman’s Opening Remarks
• Risk assessment methodology to establish criticality
• Application of risk assessment to an Fc fusion protein case study
• Literature review of the impact of glycosylation on safety
and efficacy
Alex Eon-Duval, Associate Project Manager, Biotech Process
Sciences, Merck Serono S.A.
2:00
Technology Workshop
Case Studies in Biotherapeutic-Related
Workflows with the Latest in
Ultrahigh-Resolution Quadrupole
Time-of-Flight (UHR-qTOF) Instrumentation
Attendees of this technology seminar will hear about the latest
advancements in ultrahigh-resolution time-of-flight hardware and
their application to the analytical challenges facing innovators and
biosimilar's characterization labs. Several biotech-relevant case
studies will be presented to highlight: 1) The latest in high-mass
detection and native MS, 2) The latest advancements in resolution
and mass accuracy and, 3) Advancements in electron transfer
dissociation (ETD) capabilities.
Jason S. Wood, Ph.D., Market Manager, Bruker Daltonics
2:30
Interactive Panel Discussion:
QbD Successes, Lessons Learned and Adaptation
Moderator: Thomas A. Little, Ph.D., President, Thomas A. Little
Consulting
Panelist:
Steve Kozlowski, MD, Director, Office of Biotechnology Products
(OBP), CDER, U.S. FDA (Invited)
Andrea Coombs, Director Validation and Technical Transfer,
Emergent Biosolutions
Alex Eon-Duval, Associate Project Manager, Biotech Process
Sciences, Merck Serono S.A.
“Very informative conference, gave a reality check on
what others are doing for success and what regulators
expect from a successful following.”
- Kishain Rao, Alexion Pharmaceuticals
3:00 Networking Refreshment Break in Poster and Exhibit Hall
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Tuesday, November 4, 2014 (continued)
Analytical and Characterization Challenges for Marketed and High Protein Concentration Products
3:45 CASE STUDY
NEW DATA Analytical Control System
Lifecycle Management Strategies for Marketed
Protein Products
Although post-licensure control systems are often updated by modifying
specific assays or revising individual acceptance criterion, a periodic,
comprehensive and systematic evaluation and update is necessary to align
commercial control systems with contemporary standards. To address
this need, Genentech/Roche have developed a control system lifecycle
management program for application to the portfolio of current and
future licensed biological products. In this presentation, we will examine
strategies for lifecycle management of biopharmaceutical product
commercial control systems.
Minh Luu, Associate Program Director, Pharma, Technology and
Regulatory, Genentech
4:15
NEW DATA Development and Qualification
Strategy for Subvisible Particle Testing by Light
Obscuration Method
4:45 Characterization of Opalescence Behavior for High
Concentration Monoclonal Antibody Solutions and Its
Implications
Monoclonal antibody solutions are often opalescent at high protein
concentrations. Multiple analytical techniques, including measurement of
light scattering at 90° and transmission, Tyndall test, and microscopy, were
deployed to examine the opalescence behavior for a monoclonal antibody
in different conditions including temperature, protein concentration,
salt concentration and pH. The experimental findings suggest that
protein-protein interactions and phase diagram are key to understand the
opalescence behavior.
Jifeng Zhang, Ph. D., Head of Combination Product Analytical
Technology, Department of Analytical Biotechnology, MedImmune LLC
5:15 Close of Conference
Method development and qualification strategies will be discussed to
address the challenges of using a light obscuration method to quantify
subvisible particles in high protein concentration products in different
drug product presentations.
Yoen Joo Kim, Associate Scientist II, MedImmune LLC
“A great conference that puts the well underrated biological
processes on display for interested & engaged industry
professionals.” – Peter Jordan, SciLucent, LLC
“Well organized conference, wide variety
of topics covered by the speakers.”
– Suranjana Haldar, Dr. Reddy’s Lab Ltd.
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Did You Know? More than 250 attendees from 17 different countries attended last year’s meeting.
The Best Event Attracts the Best People ... ... and the Key Players in the Industry
22%
Director
14%
Manager
By Geography
United States 91%
Europe 6%
Asia 3%
4%
President/
Vice President
18%
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5%
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Teva Pharmaceuticals
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