AIM


The specificity, efficacy and safety profiles of biologics make them good candidates for the treatment of autoimmune diseases, and the significant unmet medical needs in both large and orphan indications makes this an attractive field of research for industry companies. Scientists are striving to identify novel targets and mechanisms of action, and to apply exciting new product platforms and technologies in this space.  But major challenges remain in the areas of demonstrating improved efficacy over established standards of care, identifying quality preclinical research models and stratifying responder populations to improve clinical studies and applications. The 3rd Annual Biologics for Autoimmune Diseases presents a focused meeting that explores the application of new science and technology in the development of a next generation of effective and safe therapeutics in autoimmunity and inflammation. 

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MONDAY, APRIL 25

7:00 am Registration and Morning Coffee

8:30 Chairperson’s Remarks

Ali Zarrin, Ph.D., Scientist, Immunology, Genentech


8:40 KEYNOTE PRESENTATION:

Complement in Tolerance and Autoimmunity: Opportunities for Biotherapeutics Targeting Complement

Uday Kishore, Ph.D., Director, Center for Infection, Immunity and Disease Mechanisms, Brunel University

Complement system is a potent link between innate and adaptive immunity. Complement is a double-edged sword whose central role in immune tolerance as well as development and aggravation of inflammatory disorders including autoimmune diseases is well established. A number of antibody-dependent and independent strategies have been devised to manipulate and contain the complement activators and regulators. The lecture will give a holistic view of the field with a state-of-the-art critique.


EMERGING TARGETS AND MECHANISMS OF ACTION

9:10 Merging New and Old Paradigms to Understand Immunopathology of Smoker’s Lung Disease

Farrah Kheradmand, M.D., Professor, Pathology and Immunology, Baylor College of Medicine

The basis for cigarette smoke-induced autoimmune-mediated inflammation stems from our discovery that memory T cell responses directed against lung elastin peptides can be detected in smokers with emphysema. We have embarked on investigating the pathophysiology of chronic obstructive pulmonary disease using preclinical models that share common features with human emphysema. Our studies have elucidated the function of several upstream activators of autoimmune inflammation that could be harnessed for novel therapy.

9:40 “Accelerating” Discoveries in Autoimmunity

PJ Utz, M.D., Professor, Immunology and Rheumatology, Stanford University School of Medicine

Autoimmune diseases as a group share features including genetics, pathogenesis, and treatment choices. The field is now moving from murine studies and experiments using blood toward single cell methodologies for characterizing tissues. The speaker will describe NIH’s new Accelerating Medicines Partnership (AMP) program in rheumatoid arthritis and systemic lupus erythematosus in which synovium, bone marrow, skin, kidney and blood are being studied using RNA-Seq, ATAc-Seq, repertoire analysis, and CyTOF.

10:10 Coffee Break

10:45 Chairperson’s Remarks

10:50 Novel Mechanisms of Action for Inhibitory Receptors as Targets for Autoimmune Indications

Ali Zarrin, Ph.D., Scientist, Immunology, Genentech

Paired Ig-like type 2 receptor α (PILRα) inhibitory receptor and its counter-part PILRβ activating receptor, are both co-expressed on myeloid cells. PILRα is elevated in various inflammatory diseases such as rheumatoid arthritis. Pilrα−/− mice produce more pathogenic cytokines during inflammation and are prone to enhanced autoimmune arthritis. We report how modulation of this pathway has utility in inflammatory diseases.

11:20 Venom-Derived Peptides for the Treatment of Autoimmune Disease

Christine Beeton, Ph.D., Associate Professor, Molecular Physiology and Biophysics, Baylor College of Medicine

CCR7- effector memory T lymphocytes are involved in autoimmune diseases and up-regulate Kv1.3 channels upon activation. We have used ShK, a peptide isolated from a sea anemone venom, to design dalazatide (formerly ShK-186) as a selective and potent Kv1.3 blocker. Dalazatide has undergone preclinical testing in vitro and animal models. It was well tolerated in clinical trials with healthy volunteers and is currently being evaluated in patients with autoimmune diseases.

11:50 Investigation of Antibody-Drug Conjugates to Target Glucocorticoids to Immune Cells

Philip E. Brandish, Ph.D., Principal Scientist, Immunology & Oncology, Merck Research Laboratories

The magic bullet idea of using antibodies to target cytotoxic agents to tumor cells has proven feasible and we sought to build on those successes to enable dose-limited therapeutics beyond oncology. Using glucocorticoids as an example, we have used site-specific incorporation at a genetically encoded non-natural amino acid, novel linker chemistry, and a potent glucocorticoid receptor agonist to assess the feasibility of the general approach.

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

Opportunities and Challenges in Rare Disease Drug Development

Jeffrey Siegel, M.D., Senior Group Medical Director, Product Development Immunology, Genentech, Inc.

  • Incentives for rare disease drug development
  • Orphan drug designation: how to qualify and advantages conferred
  • Special regulatory provisions relevant to rare diseases: Breakthrough Therapy Designation, accelerated approval, FastTrack designation
  • Biomarker-defined subsets of rare diseases
  • Key differences between US & EU

Targeting inhibitory Receptors in Autoimmune Diseases

Ali Zarrin, Ph.D., Scientist, Immunology, Genentech, Inc.

  • Various classes if inhibitory receptors and their utility as drug targets
  • What drug modalities are available to target inhibitory receptors?
  • What are the challenges to target inhibitory receptors?

Novel Pathways in Regulation of Systemic Autoimmune Diseases

Farrah Kheradmand, M.D., Professor, Pathology and Immunology, Baylor College of Medicine

  • Autoimmune diseases
  • Sterile inflammation
  • Selective inhibition of autoreactive T and/or B cells
  • Early detection of susceptibility to autoimmune diseases
 

3:20 Refreshment Break in the Exhibit Hall with Poster Viewing


PLENARY KEYNOTE SESSION

4:00 Chairperson’s Remarks

4:10 The Promise of Cancer Immunotherapy: An Overview of Recent Advances and Jounce’s Approach to Delivering the Right Therapy to the Right Patient

Deborah_LawDeborah Law, D. Phil. CSO Jounce Therapeutics, Inc.

As immunotherapies become an increasingly important component of cancer treatment the challenge will be to identify ways to provide the best therapy(s) to the individual. This presentation will provide an overview of current cancer immunotherapies as well as highlight some of the challenges ahead including selection of optimal combinations, moving outside of T cell-directed approaches, and will highlight how Jounce Therapeutics is using its Translational Science Platform as an approach to develop and deliver the right therapy to the right patient.

4:50 Antibody as Drugs: Then, Now and Tomorrow

Paul_CarterPaul J. Carter, Ph.D., Senior Director and Staff Scientist, Antibody Engineering, Genentech

Antibodies have grown into a clinically and commercially important drug class with more than >45 antibodies marketed for imaging or therapy in the USA and/or Europe and with ~$63 billion in worldwide sales in 2013.  This presentation will highlight progress in developing antibody drugs and consider opportunities for future innovation.  


5:30 Welcome Reception in the Exhibit Hall with Poster Viewing

6:45 End of Day

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TUESDAY, APRIL 26

8:00am Morning Coffee

BIOLOGICS FOR SMALL AND ORPHAN AUTOIMMUNE INDICATIONS

8:25 Chairperson’s Remarks

Tony Manning, Ph.D., Vice President, Research, Momenta Pharmaceuticals

8:30 New Diseases for Old Tools, Different Approaches to Known Targets: Expanding Indications for Approved Biologics into Rare Diseases

John H. Stone, M.D., MPH, The Edward Fox Chair in Medicine, Massachusetts General Hospital; Professor, Medicine, Harvard Medical School

Expanding indications into less common diseases requires the clinical insight to identify unmet need. This is facilitated by knowing the natural history of treated disease, but even more so by the ability to evaluate patients before they begin any treatment at all (not all conditions permit this). I will review the background to pivotal trials in vasculitis and the identification of a novel therapeutic for fibroinflammatory disease that grew out of studies in IgG4-related disease. The latter experience has broad implications for other fibrosing diseases.

9:00 Biologics for Orphan Autoimmune Indications

Jeffrey Siegel, M.D., Senior Group Medical Director, Product Development Immunology, Genentech

Orphan diseases represent an area of high unmet medical need. Though once a neglected area of drug development, legislation passed in the US and in other regions has provided incentives that have dramatically accelerated clinical development. In this talk I will explore opportunities and challenges in the development of targeted biologic therapies for orphan autoimmune conditions.

9:30 Deep Biocharacterization of Autoimmune Disease Patients Yields Unique Insights into Unmet Medical Need

Tony Manning, Ph.D., Vice President, Research, Momenta Pharmaceuticals

Development of novel therapeutics is confounded by our inability to understand the complex basis of disease, resulting in a high failure rate in development and unclear benefit for patients. We developed high-resolution analytics to identify novel drug targets and patient stratification markers that address unmet medical need in autoimmune disease. This presentation will describe the application of this technology to four different disease settings, and the actionable results it yielded.

10:00 Coffee Break in the Exhibit Hall with Poster Viewing and Poster Awards

10:50 TH17 Targets for Multiple Autoimmune Diseases

Andrea Itano, Ph.D., Vice President, Head of Tempero Discovery Performance Unit, Immuno-Inflammation, GlaxoSmithKline

The pathogenic role of the TH17/IL-23 axis in autoimmune and inflammatory diseases has been validated by recent the successes reported in late-stage clinical trials for the treatment of diseases including psoriasis, ankylosing spondylitis, and Crohn’s disease by antibodies targeting the IL-17, IL-17R, and IL-23. This talk will review the current status of TH17/IL-23-targeting therapeutics in clinical studies, and discuss the implications for new targets and new disease areas.

11:20 Regulatory T Cells-Based Cellular Immunotherapy of Inflammation and Autoimmunity

Arnaud Foussat, Ph.D., CSO, TxCell

Due to their natural function in inhibiting inflammation, Regulatory T (Treg) cells are seen as a promising therapeutic tool for patients with refractory chronic inflammatory and autoimmune diseases. As of today, several clinical trials have demonstrated the safety of Treg cell administration into patients and interesting signs of efficacy. Specific challenges of Treg cell therapy development have been described and new approaches to target Treg cells in specific diseases are in development.

11:50 Tolerogenic Nanoparticles for the Treatment of Autoimmune Diseases

Kei Kishimoto, Ph.D., CSO, Selecta Biosciences

We have developed novel nanoparticles carrying a tolerogenic immunomodulator to induce durable and antigen-specific immune tolerance. We illustrate two potential applications of tolerogenic nanoparticles for the treatment of autoimmune disease: 1) immune tolerance induction directed against a pathogenic autoantigen in a model of experimental autoimmune encephalomyelitis, and 2) tolerance to prevent the formation of anti-drug antibodies directed against an immunogenic biologic therapy, adalimumab, in a spontaneous mouse model of arthritis.

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

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


PRECLINICAL AND CLINICAL STUDIES

2:00 Chairperson’s Remarks

Rachel Ettinger, Ph.D. Senior Scientist Respiratory, Inflammatory, and Autoimmune Diseases, MedImmune

2:05 Investigation and Mitigation of Safety Issues in Autoimmune Disease Biologics

Meena Subramanyam, Ph.D., Vice President, Global Biomarker Product Safety, Biogen

A number of biological therapeutics have been approved for treating a diverse group of autoimmune diseases. The immune modulating properties of biological therapeutics for autoimmune diseases has prompted scrutiny and careful evaluation in post-marketing surveillance for increased incidence of cancer, central nervous system related events, and other opportunistic infections. This talk will discuss the safety events observed with the use of these agents in autoimmune disorders and strategies in consideration to manage the risk.

2:35 Early and Transient Blockade of CD40/CD40L Interactions Abolishes Sjögren’s Manifestations in Aged Autoimmune Mice

Rachel Ettinger, Ph.D. Senior Scientist Respiratory, Inflammatory, and Autoimmune Diseases, MedImmune

Autoantibodies can be present decades before the onset of autoimmune disease manifestations. This suggests that the initial trigger involves a peripheral component that drives disease in local tissue later in life. In an autoimmune mouse model of Sjögren’s we show that “pre-diseased” autoantibodies arise from early-life germinal centers; removal of which reverses disease pathology. These data suggest that early prophylactic intervention may prove efficacious for those diseases pre-dated by autoantibodies.

3:05 Sponsored Presentation (Opportunity Available)

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

4:25 Potent Neutralizing Anti-CD1d Antibody Reduces Lung Cytokine Release in Primate Asthma Model

Adam Clarke, Ph.D., Director, Lead Generation, Biologics, Teva Pharmaceuticals Australia

CD1d is a receptor on APCs that activate NKT cells. Targeting the CD1d/NKT pathway may have therapeutic potential in asthma. We developed a high affinity CD1d antibody (NIB.2) that has strong neutralizing activity in human primary cell-based assays. We characterize the epitope and specificity of NIB.2 for CD1d in complex with lipids. Anti-CD1d antibody blockade of the CD1d/NKT pathway modulates inflammatory parameters in vivo in an A.suum primate inflammation model.

4:55 Dissecting the Properties of Anti-CXCL10 Antibodies Having Anti-Inflammatory Activity In Vivo

Marie Kosco-Vilbois,Ph.D., CSO, Novimmune

We characterized three anti-murine CXCL10 mAbs targeting different epitopes, having different potencies and capacities to bind chemokines in the context of glycosaminoglycans (GAG). Binding to CXCL10 on GAG lead to rapid serum clearance and lower efficacy in mouse models of inflammation. In contrast, antibodies binding only to soluble chemokine persisted in circulation and showed superior efficacy. These results indicate mAb characteristics required for therapeutic intervention and suggest a revised model for chemokine function.

5:25 End of Biologics for Autoimmune Diseases

5:30 Registration for Dinner Short Courses



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