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Conference Day One

7:00 am Registration & Morning Coffee

7:45 am Chair’s Opening Remarks

Synopsis

Setting perspective with an overview of the current complement landscape, identifying the

gaps and the remaining unmet needs within the landscape

Correlation vs Causation: Does Complement Drive Disease Pathophysiology or is it a Biological Response?

8:00 am Exploring Complement Receptor C5aR2 as an Immunomodulatory Target to Treat Inflammatory Diseases

  • Xaria Li Postdoctoral researcher, University of Queensland

Synopsis

Recognising C5aR2 as a powerful regulator of the proinflammatory C5a-C5aR1 axis

  • Identifying the novel, multifaceted immunomodulatory functions of C5aR2
  • Past, present, and emerging tools for C5aR2 research

8:30 am Examining the Role of Complement in Age-Related Macular Degeneration Discovered Using Patient Stem Cell Derived 2D & 3D-Bioprinted Eye Tissues

Synopsis

How to move past animal models not being able to replicate multifactorial diseases

  • Navigating polymorphisms and complement not being the only driver of a diseases
  • How do we overcome the inability completely inhibit complement in humans to determine effect on disease

9:00 am Complement’s Role in the Pathogenesis of Diseases & Exploring Combination Therapy

Synopsis

  • Action vs. reaction: Is complement overactivity the driver of disease or is it an effect of disease?
  • Identifying drivers of disease to highlight new therapeutic opportunities
  • Does more than one pathway play a role in some diseases, does this have clinical relevance?

9:30 am Lessons Learned from C1q Inhibition in Neurodegenerative Conditions

Synopsis

  • Understanding the basic scientific mechanism behind C1q inhibition in neurodegenerative diseases
  • Explore how complement’s role in the pathophysiology of neurodegenerative diseases influenced the scientific rationale behind targeting C1q
  • Hear the lessons learned by Annexon in targeting C1q for Guillain-Barré syndrome, Huntington’s Disease and Amyotrophic Lateral Sclerosis

10:00 am Morning Break and Speed Networking

Exploring Sensitive & Specific Assays to Quantify Complement Levels in Tissues

11:00 am Navigating the Pitfalls in Complement Analysis

  • Erik Toonen Senior Scientist - Research, Hycult Biotech

Synopsis

  • Accurate analysis of complement components and complement activation is of utmost importance for diagnosing disease and/or therapy monitoring
  • Reliably determining the complement status of individual patients has proven to be challenging
  • To assess complement in a robust, reliable and standardized manner, guidelines are needed not only for the measurement itself, but also for sample collection, pre-analytical sample handling and storage

11:30 am Assessing PK/PD Correlation in Preclinical Models of Complement Activation

Synopsis

  • PD models that evaluate complement biomarkers
  • Efficacy model of monogenic disease with complement-mediated readouts for potential application in complex disease
  • Targeting key disease tissue for optimal therapeutic effect

12:00 pm The Complement System in Drug Development – Step by Step Towards the Bigger Picture

Synopsis

  • Quantitative evaluation of biomarkers
  • Functional detection of anaphylatoxins
  • Functional assessment of individual pathway- and convertase activity

12:10 pm Round Tables

Synopsis

This interactive session gives you the opportunity to be part of the discussion, share ideas and learn from your peers.

Amongst your tables, talking points to consider include:

  • Accurately assessing complement activation biomarkers with sensitive and specific assays
  • Developing biomarkers which accurately reflect complement levels in tissues
  • Measuring complement factors at different stages of disease to improve our understanding of complement’s role in disease

Closing this session with an audience-wide discussion on standardization of complement measures to better enable

comparison of results across the field. Why do we not currently have standardized protocol, is there really a need?

If so, what is needed to get there.

12:40 pm Clinically Actionable Diagnostics Assays for Complement Drug Discovery

Synopsis

  • Reliable and less reliable complement blood markers
  • Operationalizing difficult assays for 7-day-a-week performance
  • Innovative subject, site and sample finding solutions

12:50 pm Lunch Break & Networking

2:00 pm Complement Biomarker Assays Should Be Fit-for-Purpose

Synopsis

  • Adapting and developing assays in a fit-for-purpose setting is essential to obtain relevant answers
  • Knowledge from clinical diagnostic setting allows interpretation of results in the relevant context
  • Challenges in dilutional linearity, sample handling and multiplexing can be solved in discussion with the client
  • Combining cellular assays with serological assays gives more complete answers on which a clinical decision can be made

What is the Best Fragment to Target; Positioning Complement Inhibitors Against Complement Inhibitors

2:10 pm Advantages & Disadvantages of Upstream vs Downstream Complement Inhibition in Autoimmune Disorders

Synopsis

  • Assess the pros and cons of inhibiting upstream fragments compared to downstream fragments
  • How C1q inhibitors compare to C3 and C5 inhibitors
  • Hear how Annexon is tackling the decision behind which fragment is the best target in autoimmune disorders

2:40 pm Examining the Flexibility of RNAi-Based Therapy: Targeting C3, C5 & Complement Factor B

Synopsis

  • Sirnaomics’ approach of using oligonucleotides in hepatocytes for gene/protein targeting
  • Identify the benefits of a combination approach with STP247G’s ability to target complement Factor CFB/C5 simultaneously
  • Explore the scientific decisions behind which factor to target for certain diseases

3:10 pm Exploring Factor D: A Potential New Target to Modulate and Treat Complement-Mediated Disease

Synopsis

  • Identifying what makes Factor D an attractive target in complement disease
  • Exploring the diseases relevant to Factor D inhibition
  • Ongoing efforts in targeting Factor D

3:40 pm Afternoon Break

Synopsis

Connect with your peers in a relaxed atmosphere and continue to forge new and existing

relationships.

4:10 pm Targeting Complement Factor BB as a Selective Inhibitor of the Alternative Pathway

Synopsis

  • SAR443809 selectively binds to Factor Bb and inhibits the complement alternative pathway, but not classical or lectin pathways
  • SAR443809 inhibits AP activity by blocking the cleavage of C3 and Factor B
  • The selectivity and efficacy of SAR443809 confer a robust pharmacodynamic response for the treatment of AP mediated disorders.

4:40 pm Roundtable Discussion

Synopsis

In this interactive session, break away into smaller groups to share your ideas and discuss your thoughts on the most

valuable target when inhibiting the complement pathway. Topics to consider:

  • Positioning proximal inhibitors against terminal inhibitors for efficacy
  • Positioning fragments against fragments: which target is better to inhibit?
  • Do specific diseases respond better to inhibition of specific targets in the complement system, and why?

5:10 pm The Life-Saving Anti-Inflammatory Potential of Blocking the C5a / C5aR Signaling Pathway

Synopsis

  • Controlling the C5a/C5aR signaling pathway: what does it take?
  • The first-in-class monoclonal anti-C5a antibody vilobelimab (Gohibic): US EUA in critical COVID-19 space and development beyond
  • INF904: a new oral chemical inhibitor of C5aR with best in class potential

5:40 pm Chairs Closing Remarks

5:45 pm Drinks Reception & Poster Session

Synopsis

This is an informal session designed to connect you with your peers in a relaxed

atmosphere, continue to build and develop new and existing relationships.

Join complement enthusiasts, eager to explore the latest cutting-edge Complement-Based

research and development. Display your own poster showcasing your own developments

whilst reviewing other posters from your peers. Get ready to be impressed!

6:30 pm End of Day One