20th-22nd March

London, UK

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Day One
Wednesday March 21st, 2018

Day Two
Thursday March 22nd, 2018

Breakfast & Networking

Chairman’s Opening Remarks

Expanding Complement Therapeutics Beyond Orphan Diseases

Advances in Understanding of Disease Pathogenesis in Rheumatoid Arthritis and Systemic Lupus Erythematosus Open Up New Opportunities for Complement Therapeutics

  • Michael Holers Scoville Professor & Head, Division of Rheumatology, University of Colorado


  • Investigating the processes by which Rheumatoid Arthritis begins as a chronic mucosal inflammatory process prior to the development of clinically apparent arthritis
  • Inhibition to mitigate the earliest phases of arthritis development, a process which exhibits several features of a complement system-engaging immune complex disease
  • The severe glomerulonephritis that is found in patients with Systemic Lupus Erythematosus (SLE) has been ameliorated in several case reports using a complement inhibitor
  • New imaging approaches to characterising and quantitating local complement activation in the lupus kidney have been developed
  • Complement receptor engagement likely plays an important role in the generation of an interferon-alpha signature in lupus patients

Therapeutically Harnessing the Role of Complement in Infection – Complement Evasion Strategies Developed by Bacterial Pathogens

  • Anna Blom Professor of Medical Protein Chemistry, Lund University


  • Analysing the crucial role of complement in antimicrobial defence
  •  Identifying mechanisms allowing bacterial pathogens such as Streptococci to establish infection despite surveillance of the complement system
  • Exploring bacterial complement evasion molecules as vaccine candidates
  • Developing fusion proteins composed of complement inhibitors and fragments of immunoglobulins for treatment of infections

Dual blockade of complement C3/C5 and TLR/CD14 as a Novel Treatment Approach for Sepsis and SIRS.

  • Tom Mollnes Professor of Immunology and Leader, Complement Research Group, University of Oslo


  • Discovery of the soluble terminal complement complex (TCC) in normal human biological fluids based on a novel antibody (aE11) against activated C9
  • aE11 antibody cross-reaction with pigs, paving the way for large animal studies
  • Establishing a human whole blood model with an anticoagulant not interfering with the inflammatory reaction, enabling investigation of cross-talk in the inflammatory network ex vivo
  • Identification of the upstream complement (C3/C5) and the TLR CD14 molecule as key “bottle neck” molecules responsible for the inflammatory reaction induced both by Gram-negative and Gram-positive bacteria, both in vitro and in vivo

Morning Refreshments & Networking

Applying Lessons Learned to the Next Generation of Complement Inhibitors

Coversin: The Role in the Treatment of Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome


  • Coversin- A subcutaneous medicine shows promising results in the treatment of
  • Paroxysmal Nocturnal Hemoglobinuria
  • Coversin has the advantage of being a patient self-administration medicine
  • Coversin begins pivotal Phase III trials in Q1 2018
  • Coversin also inhibits LTB4 allowing treatment of other orphan inflammatory diseases

Development of BIVV009 for the Treatment of Patients with Primary Cold Agglutinin Disease (CAgD)


  • For the treatment of CAgD and other CP mediated diseases, we developed antibody
    inhibitors of C1s, a CP specific serine protease
  • In a Ph1a/1b clinical study design, BIVV009 was tested for safety, tolerability, and
    pharmacokinetics and pharmacodynamics in healthy volunteers (Ph1a) and in 4
    cohorts of patients, including CAgD (Ph1b)
  • In primary CAgD patients, analyses of serum and plasma hemolytic markers
    demonstrated that one month of BIVV009 dosing immediately prevented hemolysis,
    leading to a median hemoglobin increase of ~4 g/dL and precluding the need for
    transfusions in the 5 patients who were previously transfusion dependent

The Power of Complement Therapeutics: A Patient Perspective


  • Our diagnostic journey into a world of rare disease
  • Research and development of therapeutic drugs creating a foundation of hope for patients and caregivers to rely on
  • The power of complement therapeutic drugs and the influence they have on a rare disease community

Lunch & Networking

Cytotopic Complement Inhibitors – Mechanisms and Insights

  • Richard Smith Director, Protein Therapeutics Laboratory, King’s College London


  • Complement regulation is primarily a cell-surface phenomenon yet complement inhibitors in current development act in bulk solution
  • The rise of Mirococept, an engineered fragment of CR1, Mr ~24kDa, which retains factor I cofactor and decay acceleration activities, binding to cells
  • Mirococept has been manufactured on an industrial scale to cGMP and has passed relevant safety and ADME tests
  • Tests in ~150 human subjects by several routes including ex-vivo perfusion of organs for transplantation and is well tolerated up to 100mg iv
  • A Phase II study in renal transplantation using ex-vivo perfusion has enrolled 80 patients so far – endpoints are DGF incidence and renal function
  • Several other types of agent have been cytotopically modified and are under investigation

Panel: Balancing Benefits and Risks by Assessing Optimum Inhibition Levels for Key Diseases

  • Michael Holers Scoville Professor & Head, Division of Rheumatology, University of Colorado
  • Michael Kirschfink Professor of Immunology, University of Heidelberg
  • Paul Morgan Professor of Immunology and Director, Systems Immunity Research Institute, Cardiff University


  • Identifying trends in the variance of optimum inhibition across different pathways
  • Determining which animal models companies have found most and least successful

Afternoon Refreshments & Networking

Safety Profiles, Risk and Managing Adverse Effects

Approaches to Computational Modelling of the Complement System


  • Comparison between computational models and traditional methods
  • Analysing Assumptions and limitations
  • Potential application in safety assessment, target selection, patient stratification, trial simulation

Details of this presentation aren’t available at this stage, please check back for further details.

Chairman’s Closing Remarks

  • Paul Morgan Professor of Immunology and Director, Systems Immunity Research Institute, Cardiff University

Close of Summit