Please Note: Times Are Listed As ET

8:00 am Pre-Conference Virtual Networking

8:40 am Chair’s Opening Remarks

Reviewing the State of Play of Complement-Targeted Therapeutics

8:50 am SWOT Analysis Panel Discussion Topics to be Addressed

Synopsis

  • Where are we now with complement-based therapeutics?
  • What are the untapped clinical areas where complement-based therapeutics have not yet been investigated or reached their potential?
  • What is the therapeutic and commercial opportunity of shifting the market focus from rare to mainstream diseases?
  • Where is the greatest unmet patient clinical need that can be addressed by emerging complement-targeted therapies?
  • How have competing drug modalities reached late-stage clinical testing, particularly in the mainstream disease setting, and how must be done so that complement-targeted therapies can achieve the benchmarks set by standards of care in mainstream disease settings?

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9:20 am Overcoming Challenges for Accurate Real World Complement Analysis

Synopsis

  • Complement analytes are unstable
  • Current best practice for complement sample management is based on healthy normal donor samples – patient samples are different
  • Reliable testing is possible by tightly controlling or eliminating the clinical sample workflow from draw to analysis

Leveraging Complement Biomarkers to Evaluate Disease, Predict and Monitor Treatment Response

9:30 am A Precision Medicine Approach to Classical Complement Mediated Diseases: Identifying the Right Patients to Achieve Better Outcomes

Synopsis

  • Autoimmune diseases are clinically and biologically heterogeneous; in certain diseases complement drives pathology in the whole population
    while in others a subset of patients have complement-mediated disease
  • Complement proteins and activation products, especially upstream components of the classical cascade, can be leveraged as patient selection biomarkers
  • Normalization of complement activity is associated with reduced disease activity and can serve as a monitoring tool that is linked to treatment response

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10:00 am Update on the Machaon Study of 1,000 Patients’ aHUS Genetics, Clinical Outcomes, Clinical Presentation and Laboratory Results in the US (MAC Plus Study)

  • Jamey Kain VP of Genetic Innovation, Machaon Diagnostics, Inc.

Synopsis

  • Physicians have ordered >2000 aHUS/TMA genetic tests for their patients
  • We have initiated a retrospective study to further characterize this cohort and publish the clinical and genetic data together
  • Who is ordering this test and why?
  • Additionally, soluble C5b-9 is emerging as a marker of complement involvement

10:30 am Virtual Speed Networking & Break

Stream A – Revealing Complement Biology to Facilitate Discovery of Novel Targets

Chair: Scott Rottinghaus, VP Clinical Development Hematology/Nephrology, Alexion

Investigating the Intricacies of Complement Signaling to Identify Novel Targets in Neurodegenerative Diseases

11:30 am Complement in Neurological Diseases: Roles in Pathology and Target for Therapy

  • Paul Morgan Professor of Immunology, Cardiff University

Synopsis

  • Complement contributes to pathology in diverse brain diseases
  • Terminal pathway likely a critical contributor to damage
  • Strong case for testing brain penetrant anticomplement drugs in brain diseases

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12:00 pm Complement-Mediated Synapse Loss: A Viable Therapeutic Target in Multiple Sclerosis

  • Dorothy Schafer Associate Professor of Neurobiology, UMass Medical School

Synopsis

  • Synapse loss can occur early and as an independent pathology in multiple sclerosis (MS)
  • Synapse loss occurs as a consequence of complement C3 deposition at synapses and microglial synapse engulfment
  • Targeting C3 at synapses is a promising therapeutic target to preserve synapse structure and circuit function in MS

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12:30 pm Complement in the Central Nervous System During Normal Development and Diseases

  • Yingying Zhang Postdoctoral Fellow, Boston Children’s Hospital (Michael Carroll’s lab)

Synopsis

  • Complement components are locally produced in the brain by various cell types with spatial heterogeneity
  • The expression pattern of the complement system is developmentally regulated
  • The complement pathway affects neuronal development and its overactivation can induce Schizophrenia-like symptoms in a mouse model

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Stream B – Advancing Complement Inhibitors into the Clinic

Chair: Michael Storek, Head of Complement Cluster, Sanofi




Insights & Learnings from Clinical Trials Investigating Complement Inhibitors

11:30 am Roles of Systemic vs Local Complement Activation in Macular Degeneration

  • Stephen Poor Director External Opportunities & Translational Biomarkers, Novartis Institutes for Biomedical Research, Department of Ophthalmology

Synopsis

  • Implications for clinical trials
  • Further details to be announced

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12:00 pm Genetic Technology Applications in Complement Therapy Development

Synopsis

  • Further details to be announced

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12:30 pm Supplementing Complement Factor I as a Therapeutic Strategy in Age-Related Macular Degeneration

1:00 pm Lunch Networking Break

Exploring Complement Targets in the Cancer Setting


2:00 pm Complement Targeting Therapies: A Promising Approach for the control of Graft-versus-host Disease and Tumor Relapse

  • Hung D. Nguyen Assistant Professor of Medicine, University of Central Florida College of Medicine

Synopsis

  • Alternative Complement Pathway contributes to Graft-versus-host Disease (GVHD) but not graft-versusleukemia (GVL) function
  • Site-specific Targeting complement activation is critically to control GVHD and tumor relapse after allogeneic bone marrow transplant
  • Complement regulate GVHD and GVL effect via regulating immune cell metabolism

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2:30 pm Complement Factor H as a Novel Drug Target for Cancer

Synopsis

  • CFH is a novel tumor specific target
  • Human-derived antibodies against CFH cause complement activation and tumor specific cytotoxicity
  • Complement activation by the CFH Ab promotes an adaptive immune response

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Under Appreciated Areas of Complement Crosstalk & Interface with Innate Immunity & Other Systems

3:00 pm More Complement Crosstalk: The Ins and Outs of Sublytic MAC

Industry Developments of Complement Inhibition in the Neurodegenerative Disease Setting

2:00 pm Classical Pathway Activation in Neurodegenerative Disease

Synopsis

  • Activated glial cells express classical complement pathway components during neuroinflammation
  • C1Q or C3 knockout provide similar protection against neurodegeneration in Alzheimer’s disease and frontotemporal dementia mouse models
  • Elevated levels and increased activation of the classical pathway is found in the central nervous system of neurodegeneration patients

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2:30 pm Complement C5 Inhibition in Immune- Mediated Necrotizing Myopathy (IMNM)

  • Petra Duda Head of Development – Zilucoplan, UCB

Synopsis

  • IMNM is a disease with high complement levels in muscle
  • Non-clinical data supporting pathogenic role of complement in IMNM
  • Proof of concept trial results will be shared

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3:00 pm Terminal Pathway Inhibitors for Neurodegenerative Disorders

  • Frank Baas Chief Scientific Officer, Complement Pharma

Synopsis

  • Terminal pathway inhibition facilitates regeneration
  • CP010, a humanized C6 antibody, is an effective C6 blocker in vivo
  • Systemic administration of CP010 prevents relapse in experimental autoimmune encephalomyelitis

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3:30 pm Afternoon Networking Break

Understanding How Engineered Nanoparticles Can Activate the Complement System & Inform Drug Delivery

4:00 pm Use of Complement Inhibitors to Block Opsonization and Immune Uptake of Nanoparticles

Synopsis

  • Nanoparticles including those used for drug delivery and therapy and imaging activate complement
  • Complement activation leads to immune uptake in blood
  • Use of soluble complement regulators can effectively block the uptake

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Considering How to Use Complement Inhibitors as Part of Novel Combination Strategies to Improve Patient Outcomes

4:00 pm Validating Robust Combination Strategies Within the Context of the Patient Continuum of Care

  • Moshe Vardi VP, R&D Global Medicine Team Leader Hematology/Nephrology, Alexion

Synopsis

  • When is the right time to use a complement inhibitor?
  • Given that the complement system is a multifactorial system, should we be exploring multi-points of intervention?

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Breakout Roundtable Discussions

4:30 pm Uncover the story behind leaps and setbacks from the movers and shakers of complement research and drug development. Each roundtable will be hosted by a complement R&D leader who will share a challenge, question or case study to discuss.

Complement Pathophysiology – Targeting C5a Receptor
Roundtable Leader:

  • Thomas Schall President, Chief Executive Officer & Chairman, ChemoCentryx

Complement Biomarkers, the Utility of Complement Assays & Evaluating the Best Methods to Accurately Quantify Complement Activation
Roundtable Leader:

5:00 pm Chair’s Closing Remarks

5:10 pm End of Conference Day One