November 14-16

Boston, MA, USA

Day One
Wednesday November 15 2017

Day Two
Thursday November 16 2017

Breakfast & Networking

Chairman’s Opening Remarks

  • Michael Holers President of the International Complement Society & Scoville, Professor & Head, Division of Rheumatology, University of Colorado

Focus: Therapeutic Approaches for Inhibition

Utilization of si-RNAs in Treatment of Complement-Mediated Diseases


  • Examining the role of siRNA and Alnylam technology to inhibit the complement pathway
  • Determining the efficacy and benefits of targeting C5 by siRNA in preclinical and clinical models
  • Benefits of at-source inhibition vs. peripheral inhibition

Developing a Highly Potent and Selective First-in-Class Anti-C5a Antibody for Treatment of Inflammatory Diseases


  • Overview of Terminal Complement Activation and illustration of the Extrinsic Pathway for C5a Generation
  • The InflaRx technology: Making of IFX-1, a targeted highly potent and selective anti- C5a antibody
  • Performance of IFX-1 in humans and in patients
  • Targeted C5a-inhibition in human disease: First data of IFX-1 treatment in patients suffering from Hidradenitis Suppurativa

Gene Therapy Targeting Complement for the Dry Form of Age-Related Macular Degeneration

  • Rajendra Kumar-Singh Founder & Managing Director, Hemera Biosciences │ Professor of , Developmental, Molecular and Chemical Biology, Tufts University


  • Investigating the role of complement in AMD
  • Developing an inhibitor of the membrane attack complex (MAC) that can be delivered via gene therapy
  • Proof of concept studies in mice, with preclinical dose and safety studies in mice and non-human primates
  • Determining safety of this approach with preliminary data from the phase I clinical trial
  • Examining proof of concept data for the role of complement in diabetic retinopathy

Morning Refreshments & Networking

The Role of MAC Inhibition in Treating Retinal Diseases

  • Kourous Rezaei Senior Vice President, Medical Strategy, Ophthotech Corporation


  • Why MAC inhibition makes sense
  • Examining MAC as a therapeutic approach for complement inhibition
  • The landscape of current unmet medical need in retinal diseases
  • Targeted indications for MAC Inhibition

Targeting Factor D of the Complement Alternative Pathway Using Small Molcules for the Treatment of C3 Glomerulopathy (C3G)


  • Targeting the critical trigger point of the alternative pathway with an oral complement inhibitor program
  • Benefits and Drawbacks of small molecules in complement therapeutics
  • Assessing pathophysiology of C3G as a disease of excessive AP activity and ideas for further development of AP complement inhibitors

Pharmacodynamic Efficacy of the Complement Factor B (FB) Antisense Oligonucleotide, IONIS-FB-LRX


  • Preclinical efficacy of FB antisense oligonucleotides (ASO)
  • Exploring pharmacodynamic responses to the FB ASO, IONIS-FB-LRX, in a Phase 1 clinical study
  • Assessing the impact of reducing plasma FB levels on systemic complement components
  • Identifying opportunities for future clinical development of a FB ASO

Panel: Comparing Current Approaches for Inhibition and Balancing Benefits and Risks by Assessing Optimum Inhibition Levels


  • Analyzing the strengths, limitations and potential of small molecules, monoclonal antibodies, siRNA and oligonucleotides in complement therapeutics
  • Assessing the current landscape of where each approach is experiencing particular success or difficulty
  • Identifying trends in the variance of optimum inhibition across different pathways
  • Determining which animal models companies have found most and least successful

Lunch & Networking

Focus: Safety Profiles, Risk and Managing Adverse Events

Approaches to Computational Modelling of the Complement System


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

Complement Activation as a Mediator of Anti-Complement Product Toxicities


  • Examining evidence showing that complement-directed therapies can actually activate certain complement pathways, resulting in adverse events and toxicity
  • Mechanisms of this undesired activation – direct actions, class effects and immunemediated mechanisms
  • Interpretation of these findings for the safety of complement-based drugs – functional assays and investigative method
  • Reviewing complement pathways in an applied manner – examples of anticomplement products activating complement pathways resulting in toxicity

Chairman’s Closing Remarks

  • Michael Holers President of the International Complement Society & Scoville, Professor & Head, Division of Rheumatology, University of Colorado

Close of Summit