Emerging Indications for Therapeutic Antibodies Banner


Significant scientific advances in the fields of immunology and protein science are driving the development of biotherapeutic drugs in a growing range of therapeutic areas beyond oncology. These advances include the identification of new and unique targets, new approaches in drug delivery, methods of binding to illusive targets and translational science for patient stratification and drug development for niche indications. The new PEGS Emerging Indications for Therapeutic Antibodies meeting provides a forum for research organizations with diverse portfolios to explore new science and technology in the development of a next generation of safe and effective therapeutics in an important set of emerging indications.

Final Agenda

Sunday, April 30

Recommended Short Course(s)*

SC2: Translational Considerations for Development of Monoclonal Antibodies and Emerging Constructs: Part I: Focus on Construct Design

SC7: Translational Considerations for Development of Monoclonal Antibodies and Emerging Constructs: Part 2: Focus on Preclinical Development

*Separate registration required.


7:00 am Registration (Commonwealth Hall) and Morning Coffee  (Harbor Level)

Cambridge Complex

8:30 Chairperson’s Remarks

Joachim FeldwischJoachim Feldwisch, PhD, Director, Preclinical Development, Affibody AB, Sweden

8:40 New Approaches in CNS Drug Discovery; Challenges and Opportunities for Biologic Drugs

Leonard KaczmarekLeonard Kaczmarek, PhD, Professor, Pharmacology, Cellular and Molecular Physiology, Yale University School of Medicine

Basic research on all aspects of cellular physiology, including nervous system function, has always relied on small molecule inhibitors or activators of biochemical pathways linked to membrane receptors, second messengers and protein kinases. Typically, however, major breakthroughs have occurred with the development of specific biologic agents that perturb these pathways. This talk will give an account of such developments in basic research and potential applications to disease treatment.

9:10 Discovery and Development of PRX002 (Targeting α-synuclein) for Parkinson’s Disease

Robin BarbourRobin Barbour, Head, Antibody and Assay Development, Research, Prothena Biosciences

Millions of patients suffer from Parkinson’s Disease (PD) and currently no disease-modifying therapy is approved for this neurodegenerative disorder. PD brains contain neuronal inclusions (Lewy Bodies) composed mainly of α-synuclein, a protein also genetically linked to the disease. We will discuss the discovery and development of PRX002, an antibody that decreases α-synuclein pathology, protects synapses, and improves behavior in transgenic PD mouse models. We will also review the current findings from our Phase I studies.

9:40 Targets in the Complement Pathway

Gabriela Dos Santos Cruz De Matos, PhD, Senior Scientific Investigator, Biopharm Molecular Discovery, GlaxoSmithKline, United Kingdom

Complement plays a key role in the immunopathology of immune-mediated inflammatory diseases. Existing therapies only target a small proportion of known family members in the complement pathway. This talk will focus on trying to define the role in disease of these large families of molecules and how this knowledge may lead to the identification of tractable targets for the development of future transformational medicines.

10:10 Networking Coffee Break (Harbor & Mezzanine Level)

Cambridge Complex

10:50 Development and Clinical Update for an Anti-CGRP Antibody for Migraine

Dan AllisonDan Allison, PhD, Senior Director, Antibody Technologies, Alder Biopharmaceuticals

Approximately 1% of the world’s population suffers from debilitating migraine headaches. A link between migraines and calcitonin gene-related peptide (CGRP) is clearly established, and we have developed the monoclonal antibody Eptinezumab to antagonize the peptide. In this presentation, I will discuss some highlights of the discovery, development and pharmacology of this highly potent antibody, and provide an update on its clinical development.

11:20 Structure-Based Epitope Targeting for Engineering Functional Antibodies against Nav1.7 for Pain

Luke RobinsonLuke Robinson, PhD, Associate Director, Research, Visterra, Inc.

Ion channels remain a challenging class of targets to generate functional antibodies. Using our Heirotope® and AbMatch platforms for protein design, we have incorporated a structure-based approach to the discovery of antibodies against Nav1.7. We have engineered proteins to represent conformational and functionally relevant target epitopes of this channel. We will present how our approach facilitates engineering of antibodies to target epitopes that are obscured from conventional discovery methods.

11:50 KEYNOTE PRESENTATION: Trends and Challenges in the Development of Therapeutic Antibodies for Non-Oncology Indications

Janice M. ReichertJanice M. Reichert, PhD, Executive Director, The Antibody Society

Although antibodies for cancer garner substantial attention, those for non-cancer indications comprise nearly half of the 560 commercially sponsored antibody therapeutics currently in clinical studies. Antibody therapeutics in development are designed to prevent or treat a wide variety of diseases, including headaches, infections, diabetes and hemophilia, as well as immune-meditated disorders. In this presentation, the clinical pipeline and approval success rates of antibodies for non-cancer indications will be discussed.

12:20 pm Sponsored Presentation (Opportunity Available)

12:50 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:50 Session Break

2:20 Problem-Solving Breakout Discussions (Commonwealth Hall)

3:20 Networking Refreshment Break (Harbor & Mezzanine Level)

PLENARY KEYNOTE SESSION               Amphitheater and Harborview Ballroom


4:00 Chairperson’s Remarks

Peter Fung, PhD, Senior Manager Product Marketing, NanoTemper Technologies

4:10 Challenges and Opportunities in Engineering Protein Biopharmaceuticals

Dane_WittrupK. Dane Wittrup, PhD, C.P. Dubbs Professor, Chemical Engineering and Biological Engineering; Associate Director, Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT)

Arthur C. Clarke’s First Law posits that “When a distinguished but elderly scientist states that something is possible, he is almost certainly right. When he states that something is impossible, he is very probably wrong.” Bearing this in mind, in this talk, I will highlight areas of protein drug development that appear poised for breakthroughs in the coming decade or so.

4:55 The Next Generation of Cancer Immunotherapy: Targeting Myeloid Immune Checkpoints

Kipp_WeiskopfKipp Weiskopf, MD, PhD, Resident Physician, Internal Medicine, Brigham and Women’s Hospital

Immune cells of the myeloid lineage hold tremendous potential as effectors of cancer immunotherapy. The CD47/SIRPα axis is a key molecular pathway that governs the interaction between myeloid cells and tumors. Therapies that target the interaction are effective across multiple preclinical models of cancer and are now under investigation in clinical trials. Further studies have revealed additional regulators of myeloid cell activation that can be exploited as myeloid immune checkpoints.

Precision for Medicine5:40 Welcome Reception in the Exhibit Hall with Poster Viewing (Commonwealth Hall)

7:15 End of Day


8:00 am Registration (Commonwealth Hall) and Morning Coffee (Harbor Level)

Cambridge Complex

8:25 Chairperson’s Remarks

Gregory C. IppolitoGregory C. Ippolito, PhD, Research Assistant Professor, Molecular Biosciences, Georgiou Lab, The University of Texas at Austin

8:30 Selection and Differentiation of High Affinity Antigen Specific B Cells in vivo

Ali ZarrinAli Zarrin, PhD, Senior Scientist, Immunology and Antibody Engineering, Genentech

B cells diversify their immunoglobulin light chain or heavy chain genes to produce high affinity antibodies. Antigen specific B cells are selectively differentiated in the germinal centers to seed short- or long-lived plasma cells, a process known as affinity maturation. It is not clear how B cells commit to short or long-lived plasma cell fate. Our study provides insights on how this decision might be made during normal immune response as well as autoimmune disease.

9:00 Discovery of PAD3/PAD4 Cross-Reactive mAbs, Potential Therapeutics for RA

Gregory C. IppolitoGregory C. Ippolito, PhD, Research Assistant Professor, Molecular Biosciences, Georgiou Lab, The University of Texas at Austin

PAD4 enzyme contributes to RA pathogenesis by providing a continual source of citrullinated autoantigens that induce autoimmune responses. Autoantibodies to PAD4 (and cross-reactive with PAD3) are associated with disease severity and severe erosive joint damage. We will present insights into the polyclonal anti-PAD serum immune response in RA (using a synergistic combination of IgG protein mass spectrometry and B-cell VH:VL NGS) which may facilitate improved patient stratification of RA patients and development of targeted therapeutics.

9:30 Treatment of Psoriasis Using the Engineered IL-17 Blocking Affibody Ligand Trap ABY-035: Interim Safety and Efficacy Results from Phase I/II

Joachim FeldwischJoachim Feldwisch, PhD, Director, Preclinical Development, Affibody AB, Sweden

ABY-035 consists of two small Affibody® domains for IL-17A inhibition and an albumin binding domain for half-life extension. In a Phase I/II study, dose escalation safety was assessed in 46 healthy volunteers (2-40mg) and safety and preliminary efficacy in 26 psoriasis patients (single IV or multiple SC administrations). ABY-035 was safe and well tolerated at all dose levels. Even low single doses of ABY-035 (0.03mg/kg) resulted in an early onset of effect and lower disease burden.

10:00 Coffee Break in the Exhibit Hall with Poster Viewing (Commonwealth Hall)

 Cambridge Complex

10:50 Antibacterial Monoclonal Antibodies: A Strategy to Prevent Serious Bacterial Infections

Christine TkaczykChristine Tkaczyk, PhD, Senior Scientist, Infectious Diseases/Vaccines, MedImmune

We have developed two human mAbs, MEDI4893 and SAR114, respectively against Staphylococcus aureus virulence factors alpha toxin and ClfA. mAb combination showed broad strain coverage in multiple animal models through complementary mechanism of action. MEDI4893 prophylaxis also demonstrated efficacy in a S. aureus + Gram-negative mixed lung infection model. Together our data hold some promise as an alternative therapy for the prevention of S. aureus diseases.

11:20 B-Cell Cloning and Screening to Improve the Potency of Antibodies against Infectious Diseases

Devin SokDevin Sok, PhD, Director, Antibody Discovery and Development, International AIDS Vaccine Initiative (IAVI)

Over 200 HIV broadly neutralizing antibodies (bnAbs) have been isolated and the most potent of these are being developed and evaluated for use as prophylaxis. The discovery of highly potent HIV bnAbs can be attributed to 1) the availability of donor samples that have been screened and selected for activity and 2) new technologies to isolate antibodies from memory B cells. These approaches are being explored with other infectious diseases.

11:50 Development of Next-Generation Antibody Therapeutics against Infectious Diseases

Tianlei YingTianlei Ying, PhD, Head, Antibody Engineering and Drug Discovery Group, School of Basic Medical Science, Fudan University, China

Therapeutic antibodies have shown clinical success in the treatment of many diseases. By using extraordinarily large human antibody libraries, we have identified a number of potent mAbs against emerging and chronic infectious diseases, including MERS, H7N9 influenza, Zika, HBV, HIV, etc. We have also been working on the development of novel antibody constructs (from 14 kDa to 180 kDa) as the next-generation safer, cheaper, and more potent antibody-based therapeutics.

12:20 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:20 Ice Cream Break in the Exhibit Hall with Poster Viewing (Commonwealth Hall)

 Cambridge Complex

2:00 Chairperson’s Remarks

Thomas MikitaThomas Mikita, PhD, Director, Centers for Therapeutic Innovation, Pfizer, Inc.

2:05 Clinical Update: PCSK9 Monoclonal Antibodies for Cholesterol Lowering

Robert P. GiuglianoRobert P. Giugliano, MD, Senior Investigator, TIMI Study Group, Staff Physician, Cardiovascular Medicine, Brigham and Women’s Hospital; Associate Professor, Medicine, Harvard Medical School

Monoclonal antibodies inhibiting circulating PCSK9 result in profound (50-70%) reduction in LDL-cholesterol. When added to standard therapy with a statin, PCSK9 inhibitors permit the majority of patients to achieve LDL-cholesterol levels that are below even the most stringent guideline recommendations, and significantly reduced myocardial infarction and stroke in randomized clinical trials. PCSK9 inhibitors have been added to recent practice cholesterol guideline updates, thereby solidifying their role in clinical practice.

2:35 High Affinity Factor XIa-specific IgGs and Reversal Agent as Potential Treatment for Thrombotic Disease

Thomas MikitaThomas Mikita, PhD, Director, Centers for Therapeutic Innovation, Pfizer, Inc.

We generated a high-affinity anti-FXIa mAb that does not bind the FXI zymogen or inhibit other coagulation proteases or plasma kallikrein and prevents thrombosis in two in vivo models at clinically relevant doses without detectable increases in spontaneous or induced bleeding. The co-crystal structure of this mAb bound to FXIa accounts for its high selectivity. We have also developed two reversal agents that quickly counter the effect of this mAb.

3:05 pm Engineering Cells at the Protein Level with Intracellular Antibodies

Andrea_MarschallAndrea Marschall, PhD, Postdoctoral Researcher, Biochemistry, Brandeis University 

Intracellular antibodies (intrabodies) can knock down functions by acting at the protein level and allows gradual quantitative tuning of membrane-receptors. We demonstrated gradual reductions of vascular adhesion molecule 1 (VCAM1) in vitro. In the world’s first transgenic intrabody mouse that we generated, ablation of VCAM1 by an intracellular antibody resulted in aberrant localization of B cells in adult animals. Intrabody mice were viable although genetic knockouts of VCAM1 are lethal.

3:35 Refreshment Break in the Exhibit Hall with Poster Viewing (Commonwealth Hall)

4:25 Humanized Anti-FIXa/FX Bispecific Antibody for the Treatment of Hemophilia A

Hikaru KogaHikaru Koga, Researcher, Biologics Discovery Department, Chugai Pharmaceutical Co., Ltd., Japan

Emicizumab is humanized anti-FIXa/FX bispecific antibody for the treatment of hemophilia A, which was filed for regulatory approval. In this presentation, I will introduce how emicizumab was created, its preclinical data, kinetics understanding of FVIII mimetic activity, and Phase III study data. In addition, novel antibody engineering to further improve the property of emicizumab will be presented.

4:55 Antibodies as Protease Inhibitors: Lanadelumab Inhibition of Plasma Kallikrein for Hereditary Angioedema Prophylaxis

Dan SextonDan Sexton, PhD, Director, Pharmacology, Shire

While there are several small molecule protease inhibitor therapies for hereditary angioedema, their off-target selectivity could be associated with associated side effects. Monoclonal antibody inhibitors of proteases can exhibit high potency and specificity combined with a long therapeutic half-life. Lanadelumab is one such antibody inhibitor of plasma kallikrein, discovered using phage display that is in clinical development for the prophylactic treatment of hereditary angioedema due to C1 inhibitor deficiency.

5:25 End of Emerging Indications for Therapeutic Antibodies

5:30 Registration for Dinner Short Courses (Commonwealth Hall)

Recommended Dinner Short Course(s)*

SC8: Introduction to Biophysical Analysis for Biotherapeutics: Discovery & Development Applications

*Separate registration required.

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