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Marque Todd, Ph.D., Regulatory Strategy Lead, Drug Safety Research & Development, Pfizer, Inc.



7:00am Registration and Morning Coffee


8:30 Chairperson’s Opening Remarks

Jeffrey A. Engelhardt, D.V.M., Ph.D., DACVP, President and Pathologist, Engelhardt Consulting, Inc., and SNBL USA, Ltd.


Revealing the “Magic” of Monoclonal Antibodies

Joy CavagnaroJoy Cavagnaro, Ph.D., President, Access BIO

Monoclonal antibodies have been used as experimental therapy and as essential research tools since the beginning of the 1980s. Initially considered as the ultimate realization of Paul Ehrlich’s concept of a “magic bullet,” monoclonal antibodies have evolved over the past three decades. This presentation will reveal the “tricks of the trade” that have enabled the successful development and approval of monoclonal antibodies for diagnosis and treatment and include a brief glimpse into the future.

9:10 Recommendations for Nonclinical PK Comparability Study Designs for Monoclonal Antibodies

Wendy Putnam, Ph.D., Scientist, Pharmacokinetics and Pharmacodynamics, Genentech, Inc.

Nonclinical pharmacokinetic studies may be required to demonstrate comparability of therapeutic antibodies following manufacturing changes during clinical development and post-approval. This presentation will discuss when and how to conduct nonclinical PK comparability studies. Factors to consider in designing comparability studies will be discussed, including selection of study species, dose/regimen, PK endpoint, acceptance criteria, and sample size. Case studies of the use of nonclinical PK comparability studies in the development of therapeutic monoclonal antibodies will be reviewed, together with lessons learned.

9:40 The Role of Pharmacokinetics in the Design, Conduct and Interpretation of Toxicology Studies with Antibody-Based Biotherapeutics

Stanley A. Roberts, Ph.D., D.A.B.T., Principal, SAR Safety Assessment LLC

Understanding the disposition of antibody-based therapeutics is critical for selecting the best new drug candidates. Principles and strategies will be reviewed that detail the studies necessary for assuring that compounds with the appropriate efficacy and safety attributes are selected for development. Specific case histories will be discussed that provide examples of how the pharmacokinetic characteristics are determined and their impact on toxicology studies. The potential impact on the development program, including human studies, will also be discussed.

10:10 Coffee Break, Poster and Exhibit Viewing

Tox - Safety

11:10 Nonclinical Reproductive and Developmental Toxicity Testing Strategies with Antibody Therapeutics

William J. Breslin, Ph.D., Senior Research Advisor, NSD Safety Assessment, Eli Lilly and Company

Because antibodies (Ab) therapeutics are highly target and species specific, they may not demonstrate active pharmacology in standard rat, mouse or rabbit models. As a result, nonhuman primate, homologous molecule, or transgenic models may be required for the evaluation of nonclinical reproductive and developmental toxicity. The application of these nontraditional strategies for reproductive and developmental toxicity using Ab therapeutics will be addressed using examples from previous and ongoing drug development programs.

11:40  Translational Safety for Therapeutic Antibodies: Protecting Subjects and Enabling Risk:Benefit Decisions During Early Clinical Development

Andrew Erdman, M.D., Global Safety Medical Director, Amgen

The transition from preclinical to clinical development is a critical milestone in drug development, with safety as the critical concern. This talk will discuss tactics, strategies and resources designed to ensure the safety of subjects in early clinical trials, identify and manage any risks to subjects and the development program as a whole, and provide the necessary safety information to enable early risk:benefit development decisions.

12:10pm Luncheon Presentations (Sponsorship Opportunities Available) or Lunch on Your Own

Tox - Testing Models

1:30 Chairperson’s Remarks

Stanley A. Roberts, Ph.D., D.A.B.T., Principal, SAR Safety Assessment LLC 

1:35 Species Selection: The Foundation of the Nonclinical Toxicology Program

Marque Todd, Ph.D., Regulatory Strategy Lead, Drug Safety Research & Development, Pfizer, Inc.

Selection of relevant species for the nonclinical development of biotherapeutics is foundational for the entire development program. Several aspects need to be addressed before the most relevant species can be selected and compared with the similar human data: target sequence homology, target distribution, target binding and affinity and biological activity.  Assessment of potential immunogenicity may also be important.  Several different techniques exist that can aid the nonclinical scientist in collecting and assessing data for species selection.  These various aspects of species selection will be addressed in the presentation.

2:05 Surrogates: Their Use, Qualification and Challenges

Janet Clarke, Ph.D., D.A.B.T., Senior Director, Pharmacotoxicology, Biogen Idec

Surrogates can serve a useful role in providing safety data when use of the clinical candidate is limited by lack of activity in animal models. Circumstances to consider when to use or not use a surrogate antibody, and how to “qualify” a surrogate for use to represent a safety assessment of the clinical candidate will be discussed with reference to specific examples. Finally, scientific, financial and regulatory challenges will be outlined.

2:35 Real Time Cell Surface Interaction Analysis: A Focus on Lectin-Cell Glycan Interactions
Staffan Grenklo, Ph.D, Cell Biologist, Attana AB
Sponsored by
attana small
In today’s quest for new therapeutic drugs, it is increasingly important to obtain early and informative data on potential agents in order to save time and costs. Combining biosensor technologies contributing to detailed information on molecular interactions with an experimental approach, as close as possible to the natural environment of action, increases the chances of selecting the optimal candidate already in the pre-clinical phase.

2:50 SBiomolecule Production Using PEI-Mediated Transient Transfection
Alain Cuzange, Ph.D., Polyplus-transfection
Transient gene expression in mammalian cells is an attractive alternative for recombinant protein & antibody production. Chemically-defined media supporting the growth of non-adherent cells are commonly used. The use of the cationic polymer, polyethylenimine (PEI), a well-known synthetic delivery reagent, for transfection is covered by a patent from which Polyplus-transfection™ is the worldwide exclusive licensee. Our latest generation of linear PEI is more efficient than the previous branched polymers for transfection into mammalian cells. An overview of the properties and different qualities of PEIs used in bioproduction will be presented.

3:05 Networking Refreshment Break, Poster and Exhibit Viewing

3:50 Problem Solving Break-Out Sessions

We present these one-hour moderated discussion groups to allow researchers the opportunity to network and exchange information with colleagues from around the world in a small-group setting. Each table will address a scientifi c topic that is related to the meeting to enhance indepth discussion and interactive problem solving with the potential for establishing collaborations. You will select a topic group, sit down at the selected table, and join the discussion.

Table 6: Toxicologists & Clinicians – We’re Talking, But Are We Communicating?

Moderator: Meredith Rocca, Ph.D., D.A.B.T., Director, Nonclinical Safety Evaluation, Elan Pharmaceuticals

  • Detailed toxicology summaries are included in the IB, but are they giving clinicians the information they need?  A non-adverse finding in a rat may be unacceptable in a clinical study
  • Changes in clinical chemistry over baseline may concern toxicologists, but not clinicians if they are within the normal range

Table 7: Cross Species Specificity – The Challenge Faced When Characterizing Targets

Moderator: Shane Olwill, Ph.D., Director, Research & Development, Fusion Antibodies Ltd.

  • How do you develop an antibody with cross species efficacy?
  • Designing antigens to conserved epitopes
  • Where do you go if your target is not conserved in small animal models?
  • Murine surrogates and benchmarking results

Table 8: Data Integration – Do You Need to Work Across Silos?

Moderator: Jeffrey A. Engelhardt, DVM, Ph.D., DACVP, President and Pathologist, Engelhardt Consulting, Inc., and SNBL USA, Ltd.

This discussion will address various ways to work across discipline “silos” to achieve an accurate gap analysis for the available study data.  There are many reasons for the formation of the silos with an equal or greater number of ways to tunnel under or break through them.  Without a quality gap analysis, the dossier is liable to have unintended holes that will raise questions during the validation and evaluation process by a regulatory agency.

 A quality nonclinical assessment requires input from pharmacology, PKDM, toxicology, manufacturing, and clinical.

  • How do you address different levels of understanding of drug development?
  • How do you address team leaders that need to be “in charge?”
  • How do you know what data to ask for?
  • What are the drivers for the silos in your organization?
  • What are some tactics to overcome these drivers?
  • Do you have any trump cards that you can play?
  • How much lead time do you have to prepare the documents?

Table 9: The ICH S6 Addendum – How Will It Change Your Nonclinical Safety Evaluation Strategy?

Moderator: Marque Todd, MS, DVM, DABT, Regulatory Strategy Lead, Drug Safety R&D, Pfizer, Inc.

  • The ICH S6 Addendum covers 5 key topics: species selection, study design, immunogenicity, reproductive and developmental toxicity and assessment of carcinogenic potential – do you understand the impact of the potential changes to your nonclinical development strategy?
  • Changes in the addendum can impact both early and late-stage nonclinical strategies
  • Changes can streamline your development plan and save resources and time

Table 10: What is Biologically Relevant?

Moderator: Staffan Grenklo, Ph.D., Cell Biologist & International Account Manager-Attana AB

  • Is measuring on pure samples good enough?
  • How to measure binding to complex structures?
  • How to bridge the gap between in vitro assays and bio assays?

Table 11: What Types of Non-Clinical Studies are Required for an IND?

Moderator: Stanley Roberts, Ph.D., DABT, Principal, SAR Safety Assessment, LLC

  • What are the non-clinical strategies for filing an IND as applied to a variety of biotherapeutic classes?
  • What types of non-clinical studies (i.e., efficacy, PK/disposition and toxicology) are needed?
  • Other issues to discuss include: the timing of studies, how to problem-solve scientifci challenges and how to select/manage collaborations with partners (e.g., universities and CRO's)

4:50 Networking Cocktail Reception in the Exhibit Hall

6:00 End of Day

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Program Navigation

Phage and Yeast Display of Antibodies and Proteins Engineering Antibodies Antibody Optimization Difficult to Express Proteins Pre-Clinical/Clinical Development Revival of Bispecific Antibodies Immunogenicity of Therapeutic Biologics Protein Aggregation in Biopharmaceutical Products Biotherapeutic Targets

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