November 14-16

Boston, MA, USA

Day One
Wednesday November 15 2017

Day Two
Thursday November 16 2017

08.00
Breakfast & Registration

09.00
Chairman’s Opening Remarks

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

Clinical Trial Design Optimization

09.10
Identifying the Right Candidates – Complement Testing in the Era of Complement Specifics Therapeutics

Synopsis

  • The current state of complement testing
  • Can we update the historic test to meet the current demands?
  • The challenges confounding quality complement results
  • A review of the international efforts to standardize complement analysis

09.40
Enabling Confident Decision Making by Generating Powerful Data in Rare Disease Trials

Synopsis

  • Determining effective methods for patient recruitment and experienced investigator acquisition
  • Streamlining clinical trials by ensuring patient suitability – optimal patient stratification practices
  • Altering clinical trial design to move beyond rare and orphan diseases into common disease areas

10.10
Panel: Assessing Optimum Dosage, Targeting, Endpoints and Sample Handling for Clinical Trials

Synopsis

  • Analyzing dosing requirements and site of targeting data
  • Clinical endpoints – identifying powerful biomarkers
  • Exploring methods for sample collection and handling to avoid ex-vivo activation

10.50
Morning Refreshments & Networking

Focus: Indication Selection and Target Identification

11.40
Therapeutic Insights from Studies of Complement Activation Mechanisms at Local Tissue Sites

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

Synopsis

  • Developing therapeutic strategies that minimize systemic inhibition of this pathway by targeting inhibition of the pathway to sites of complement activation
  • Understanding which of the complement activation and amplification mechanisms play essential roles in driving local injury in a disease-specific manner
  • Monitoring levels of in vivo complement activation in targeted tissues
  • Creating a method to image fixed complement C3d in tissue sites to provide essential insights into control of complement activation in tissue sites inaccessible to biomarker assessment

12.10
TNT009 Prevents the Deposition of Complement Split Products on Cells and Tissues from Patients with Classical Pathway Mediated Diseases

  • Graham Parry Senior Director of Translational Research, Bioverativ

Synopsis

  • Identifying biomarkers to assess the pharmacodynamic effect of candidate molecules in diseases where complement activation is thought to play a pathologic role.
  • Utilizing these markers of disease activity in a Phase 1 integrated trial design to evaluate monoclonal antibody TNT009’s ability to inhibit C1
  • Using TNT009 to treat patients with various classical pathway mediated diseases.
  • Evidence of the pharmacodynamic effect of TNT009 in these diverse patient populations and supporting the clinical development of the molecule in various disease settings

12.40
The Anaphylatoxins and their Receptors as Targets in Inflammatory Diseases.

  • Jörg Köhl Director of the Institute for Systemic Inflammation Research, University of Lübeck

Synopsis

  • Examining the roles of C3a and C5a as effector molecules in acute and chronic inflammatory diseases through activation of their G protein-coupled receptors
  • Analyzing anaphylatoxic regulation and control of innate and adaptive immune responses through intense cross-talk with immune receptor pathways.
  • Assessing evidence that C5a plays an important role for the development and severity of food allergy, and the pathophysiologic role of C5a in experimental models of skin
    blistering diseases.
  • The contribution of the C5a/C5aR1 axis for disease development, and discussing C5aR1 as an alternative or additional therapeutic option to block the inflammatory events in neurodegenerative diseases.

13.10
The Role of Complement Therapeutics in the Treatment of Geographic Atrophy, the Advanced Form of Dry Age-Related Macular Degeneration

Synopsis

  • Examining the role of complement in GA/AMD
  • Inhibiting the complement system to slow or halt AMD disease progression.
  • Success of the complement factor D inhibitor lampalizumab in GA phase II clinical trial.
  • Investigating the contribution of complement-independent pathways to GA disease pathogenesis.

13.40
Lunch & Networking

14.30
Terminal Complement Inhibition for Refractory Generalize Myasthenia Gravis, a Neurological Disease Driven by Classical Complement Pathway Activation

Synopsis

  • An overview of the classical pathway in complement therapeutics
  • Determining the role of complement in myasthenia gravis – examining animal model data
  • Phase II trial of eculizumab in gMG – POC data for moving into phase III
  • Phase III trial of eculizumab for refractory gMG: design, efficacy, safety results and conclusions
  • Overview of eculizumab safety

15.00
Therapeutic Targeting of C3 to treat PNH, GA & Macular Degeneration

Synopsis

  • Overcoming the challenges associated with inhibiting the most abundant complement protein
  • Realising the potential of C3 inhibition for disease control and modification
  • Lessons learned from phase I & II compound APL-2 clinical trials

15.30
Panel: Determining which Portion of the Complement Pathway is the Most Effective Intervention Point for Individual Diseases

  • Michael Holers President of the International Complement Society & Scoville, Professor & Head, Division of Rheumatology, University of Colorado
  • David Apelian CMO & EVP, Achillion Pharmaceuticals
  • Menno van Lookeren Principal Scientist, Immunology, Genentech

Synopsis

  • Understanding the pathophysiology of diseases through the relationships between pathway sub-components, and how this can be leveraged to treat individual conditions
  • Analyzing the advantages and disadvantages of targeting on different levels
  • Discussing trends in the complement therapeutics space between points of inhibition and disease areas

16.10
Afternoon Refreshments & Networking

17.00
Complement-based Drug Development Round Tables

Synopsis

  1. Exploring the Micro-Landscape of Complement Related Compounds

    -Which cells are responsible for the production of key compounds?
    -At what rate are components cleared from cells and replenished?
    -What implications do these areas have for the efficacy of complement-based drugs?

  2. The Future of Complement Therapeutics

    -What are the most interesting cutting edge developments in this area that could have great potential?
    -How can companies begin preparation to capitalize on emerging market trends in coming years?

  3. Expanding Complement Therapeutics into Common Diseases Areas

    -What alterations must be made to clinical trial design in order to move beyond rare and orphan diseases?
    -What implications will moving into common disease areas have for complement therapeutics – how can we begin to prepare for perceived challenges?

  4. Meeting Dosing and Efficacy Regulatory Requirements

    -What experiences have individuals had regarding PKPD and safety guidelines in the complement therapeutics space?
    -How can we ensure that drugs meet the required levels of therapeutic efficacy for market approval?
    -What are the biggest obstacles to getting complement-based drugs through the revelatory approval process?

  5. Preparing for Commercialization of Complement Therapies

    -How can drug production processes be upscaled to meet both initial market demand and subsequent growth?
    -How can logistical issues with manufacture, storage and transport of treatments be overcome?
    -What future methods for treatment delivery may solve the current patient access problem?

18.00
Chairman’s Closing Remarks