Developability and Candidate Selection


The notion of “kill fast, kill early” has been a motto of the pharmaceutical companies for many years, and today, this same notion is becoming center-stage action for many biopharm companies looking to select the right biologic to move forward, decrease attrition rate in late-stage development and consequently bring down costs and timelines.

The Developability and Candidate Selection conference will look at analytical and profiling techniques to determine the feasibility of molecules for further development, such as evaluation of their physicochemical properties, analysis of their aggregation and immunogenicity risks, and optimization of their properties through engineering and design.

We invite analytical and discovery scientists working on developability, molecular assessment, profiling, candidate selection, early stage R&D, analytical method development, process development, pre-formulation and PKPD to join us and hear about the various techniques and efforts used at various companies to determine a molecule’s candidacy which helped move the right molecule forward.

MONDAY, MARCH 14

7:30 am Registration and Morning Coffee

8:30 Chairperson’s Opening Remarks

Pranay Khare, Ph.D., Independent Consultant


CANDIDATE SELECTION AT EARLY DISCOVERY


KEYNOTE PRESENTATION
8:40 Analytical Strategies to Guide Candidate Selection through Developability Assessment

Johnson VargheseJohnson Varghese, Ph.D., Senior Director, Head of Analytical Development, Shire

 

9:10 Integration of Developability Assessment and Protein Engineering for the Optimization of Therapeutic Antibodies

Chetan_PatelChetan N. Patel, Ph.D., Senior Research Scientist, Biotherapeutics Discovery & Research, Eli Lilly and Company

This presentation will discuss the analytical characterization of developability attributes of therapeutic molecules during discovery and provide case studies of engineering strategies to mitigate developability risks.


9:40 Early Discovery Developability and Molecular Assessments to Enable Optimal Candidate Selection

Amy Tam, MSc, Principal Scientist, Global Biotherapeutic Technologies, Pfizer WRD

The presentation will describe Pfizer’s three-tier molecular assessment approach for ranking, de-risking and selection of lead antibody candidate. A case study will be presented to illustrate the importance of early discovery developability and molecular assessment.

10:10 Networking Coffee Break

10:40 Streamlined in-vivo Fitness and FcRn Binding Assessment for Selection of Bio-Therapeutic Drug Candidates

Torsten_KuiperTorsten Kuiper, Dipl. Ing (FH), Principal Scientist, Integrated Biologics Profiling, Novartis Pharma AG

In vivo fitness is an important criterion for the developability risk assessment and selection of novel bio-therapeutic drug candidates during the pre-clinical development phase. The measurement of relevant parameters such as pharmacokinetics, stability in and ex vivo, FcRn binding properties and off-target binding activity is usually rather complex, laborious and time-consuming. In this talk we present our streamlined workflow and a case study outlining a novel method for screening FcRn binding interactions.

11:10 Application of High-Throughput Developability Assays during Early Antibody Discovery to Minimize Downstream Risks

Tingwan Sun, Ph.D., Scientist, Protein Analytics, Adimab

Without systematic and rigorous built-in QC for in vivo antibody discovery platforms, in vitro discovery is often challenged for selection of highly developable leads with minimum downstream issues. Here we implemented a few high throughput assays targeting detection of antibody self- and cross- interaction to predict the fate of an antibody during expression, purification, storage and serum clearance.

11:40 Selection of Novel Glycosylated Polypeptide Targets from Controlled Engineered Eukaryotic Display Library

Pranay_KharePranay D. Khare, Ph.D., Independent Consultant

Tumor antigen’s specificity, targeting capability, exclusivity and accessibility determine the therapeutic benefit and toxicity. This talk will discuss the use of novel patented eukaryotic display technology that can identify novel glycosylated polypeptides, proteins and/or receptors on tumor of your choice.


Abzena12:10 pm Analytical Challenges for Antibodies and Antibody Drug Conjugates

Martin PabstMartin Pabst, Ph.D., Abzena



12:40 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:55 Chairperson’s Remarks

Chetan N. Patel, Ph.D., Senior Research Scientist, Biotherapeutics Discovery & Research, Eli Lilly and Company


HIGH THROUGHPUT SCREENING AND COMPUTATIONAL TECHNIQUES FOR PREDICTING DEVELOPABILITY

2:05 Selected Poster Presentation: Accurate and Sensitive Detection of Free Thiols in Proteins for Early Developability of Biological Therapeutics

Steven Orcutt, Research Scientist, Janssen R&D

Accurate determination of disulfides in antibodies ensures appropriate developability profiles for production. Free thiol determination of antibodies by Ellman's Reagent is limited to accurate detection of 1-2% and requires milligram amounts. Commercial fluorescent thiol reactive probes promise greater sensitivity, yet relative signals require care in quantitation. The probe N-(7-Dimethylamino-4-methyl-coumarinyl) maleimide robustly reports antibody free thiol content of <2%, is in good agreement with Ellman’s, and requires 200µg or less.

2:35 Assessing Developability-Related Issues with Computational and Experimental Methods

Daniel_SeeligerDaniel Seeliger, Ph.D., Research Scientist, Discovery Research, Boehringer-Ingelheim Pharma

Minor changes on the sequences of antibodies can have dramatic effects on stability. We use a complementary approach of computational and experimental methods to detect potential liabilities at an early stage and to map undesired properties to distinct sequence patterns. The presentation will cover the basic concepts of the approach and present a case study from BI research.


3:05 ROUNDTABLE DISCUSSIONS

Table 1
• Protein Engineering for Mitigation of Immunogenicity Risk

Moderator: Chetan N. Patel, Ph.D., Senior Research Scientist, Biotherapeutic Discovery & Research, Eli Lilly and Company

Table 2
• Eukaryotic Target Discovery Platforms

Moderator: Pranay D. Khare, Ph.D., Independent Consultant

Table 3
• Orthogonal Analytical Assays: Friend or Foe?

Moderator: Liangyi Zhang, Ph.D., Senior Scientist, Analytical Development, AbbVie Biotherapeutics, Inc.

 

  • How often is orthogonal methods used in product characterization?
  • How to leverage the results from orthogonal methods?
  • How often are the results from two methods not consistent with each other?
  • Are orthogonal methods really worthwhile?

 

Table 4
• Aggregation: Factors that Give Rise to Aggregation and Means of Characterizing Aggregates

Dean Ripple, Ph.D., Supervisory Physicist, Bioprocess Measurements Group, National Institute of Standards and Technology


4:05 Networking Refreshment Break

4:35 Developability Screening of Protein Therapeutics: HT Solubility and Physical Stability Studies

Aaron Yamniuk, Ph.D., Senior Research Investigator II, Protein Science, Molecular Discovery Technologies, Bristol-Myers Squibb


KEYNOTE PRESENTATION
5:05 Revealing the Complicated Relationships between Antibody Fragment Stability, Structural Flexibility, and Allosteric Response

Dennis LivesayDennis R. Livesay, Ph.D., Professor, Bioinformatics and Genomics, University of North Carolina at Charlotte

Recombinant antibody fragments have emerged as credible alternatives to full therapeutic antibodies. Unfortunately, reduced thermostability is frequently observed, limiting their broad utility. In response, screening for mutants that increase stability without compromising affinity is commonly employed. Little is known about how the uncovered mutations affect dynamic properties. In this talk, I will discuss the frequency and scale of changes in structural flexibility and allostery across a number of different antibody fragment systems.


5:35 Welcome Reception in the Exhibit Hall with Poster Viewing

6:45 Close of Day One

TUESDAY, MARCH 15

8:00 am Morning Coffee

8:30 Chairperson’s Opening Remarks

Liangyi Zhang, Ph.D., Senior Scientist, Analytical Development, AbbVie Biotherapeutics, Inc.


DEVELOPABILITY AND MANUFACTURABILITY ASSESSMENT OF NOVEL AND COMPLEX BIOLOGICS

8:40 Developability and Manufacturability of κλ-bodies

Sylvain Raimondi, MSc, Head of Analytics Unit, Bioprocess R&D, Novimmune SA

κλ-bodies are a novel bispecific format with properties indistinguishable from an IgG. This talk will cover early expression studies in CHOK1 stable pools, and analytical profiling techniques and forced degradation studies to assess the physical properties and mechanisms of degradation in order to select the most appropriate molecule for development with the aim of producing gram per liter quantities with ≥99% purity and low aggregates.

9:10 Designing Bispecific Antibodies with Improved Developability Properties

Srinath_KasturiranganSrinath Kasturirangan, Ph.D., Scientist I, Antibody Discovery & Protein Engineering, MedImmune

While current scFv-based BiSAbs offer a variety of geometries between antigen binding sites, some spacing options are lacking. Using a structurally-motivated approach we designed additional variants with scFvs inserted into surface-exposed loops of an IgG1 Fc. The scFvs in these BiSAb variants are N- and C-terminally constrained, potentially preventing domain exchange and aggregate formation, thereby precluding the need for scFv engineering to stabilize the molecule.

9:40 Developability Considerations for Complex Biologics

Hubert_KettengergerHubert Kettenberger, Ph.D., Principal Scientist, Roche Pharma Research and Early Development (pRED)

Complex biologics such as bispecific or multispecific antibodies or fusion proteins play an increasingly important role as emerging new pharmaceuticals. In contrast to classic monoclonal antibodies, complex biologics can pose additional developability challenges. The talk will focus on our strategies to design and optimize stable, well-behaved drug candidates.

Caprion No Shadow10:10 Identification and Quantification of Host Cell Proteins by Mass Spectrometry

Laura McIntosh, Ph.D., Vice President, Translational Research, Caprion Biosciences Inc.

Mass spectrometry is a powerful tool for Host Cell Protein (HCP) characterization. A process for identification and quantitation of HCP will be described. Identification is performed using a 2D-RPLC-MS/MS proteomics workflow for which proteins are digested and the resulting peptides are submitted to 2 separation methods prior to RPLC-MS/MS analysis. Multiple reaction monitoring (MRM) is then utilized for absolute quantitation of individual HCP. HCP levels of 1 – 25 ppm can be quantified


10:40 Coffee Break in the Exhibit Hall with Poster Viewing

11:10 Analytical Characterization and Manufacturing Consistency Assessment of Antibody-Drug Conjugates for Early-Stage Clinical Development

Liangyi_ZhangLiangyi Zhang, Ph.D., Senior Scientist, Analytical Development, AbbVie Biotherapeutics, Inc.

An ADC molecule is typically comprised of a targeting antibody and a cytotoxic drug at various drug-to-antibody ratios. Because of its complex structure, an ADC should undergo extensive analytical characterization to ensure its purity, structure integrity and lot-to-lot comparability. Here we present a comprehensive analytical strategy for characterization and manufacturing consistency assessment of antibody-drug conjugates for early-stage clinical development. A case study will be presented to illustrate the challenges faced and lessons learned from this exercise.

11:40 Solution Behavior Analysis as Manufacturability Assessment for Subcutaneous Injectable Antibodies

Meiri_ShidaMeiri Shida, Ph.D., Researcher, Discovery Research Department, Chugai Pharmaceuticals Co. Ltd.

High concentration antibody solution is required to achieve subcutaneous injection. Although this can be managed to some extent by optimizing the formulation, intrinsic antibody property is critically important to achieve high solubility and low viscosity. Therefore, solubility and viscosity are now routinely evaluated during lead optimization. Even a single mutation can significantly affect the viscosity. Our experiences will give a better understanding of how these items can guide antibody selection.

 Unchained Labs
12:10 pm A Novel Automated System for Buffer Exchange and Concentration of Biopharmaceuticals

Russell Burge, Ph.D., Applications Scientist, Unchained Labs

Traditional approaches for buffer exchange include dialysis, desalting columns, and centrifugal UF/DF devices. While these are easy, they are also manual and don't allow for protein concentration. In this talk, we describe Unchained Labs' automated system for buffer exchange and concentration of biopharmaceuticals and its application to protein formulation preparation.

 

12:40 Close of Part One: Developability and Candidate Selection - Stay on for Part Two: Advances in Characterization Methods and Approaches