November 13-15, 2018

Boston, MA, USA

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Day One
Wednesday 14th November

Day Two
Thursday 15th November

08.00
Registration & Morning Refreshments

09.00
Chair’s Opening Remarks

Explore Which Complement Target has the Most Clinical Success in Rare Disorders

09.30
Discovery and Development of Oral Complement Factor D Inhibitors

  • Mingjun Huang Senior Vice President & Head of Research, Achillion Pharmaceuticals

Synopsis

  • Rationales for discovery and development of small molecules targeting at complement factor D
  • Preclinical assessments of complement factor D inhibitors
  • Clinical assessments of complement factor D inhibitors

10.00
Developing a Pipeline of Complement Inhibitors; Lessons from Preclinical and Clinical Development

Synopsis

  • Clinical Development of RA101495: Update on Phase 1 and phase 2 studies
  • Developing a pipeline of macrocyclic Factor D inhibitors
  • Leveraging macrocyclic peptides for small molecule discovery, with a focus on the identification of orally bioavailable 1st in class, small molecule inhibitors of Complement C5

10.30
C3 Inhibition with APL-2 in Rare Diseases

  • Lukas Scheibler Executive Vice President, Research and Translatable Medicine, Apellis Pharmaceuticals

Synopsis

  • An update on the clinical development of APL-2, a potent C3 inhibitor, will be provided
  • ‘Proof of Concept’ has been demonstrated with APL-2 for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH) and Geographic Atrophy (GA)
  • APL-2 is developed in other indications such as complement dependent nephropathies, autoimmune hemolytic anemia among others

11.00
Morning Refreshments & Speed Networking

Improve Translational Biomarkers to Understand Complements’ Involvement in Any Given Disease State

12.00
Bioanalytical Approaches in Supporting Rare Disease Therapeutics

  • Mark Ma Senior Director, Bioanalytical and Biomarker Development, Alexion Research

Synopsis

  • Introduction of rare disease
  • Challenges in developing suitable bioanalytical strategy for support of rare disease R&D
  • Alexion’s experiences of bioanalytical innovation and quality enhancement

12.30
Significant Advances in Genetic Testing within Clinical Trials

  • Mike Ero Chief Executive Officer, Machaon Diagnostics

Synopsis

  • Presentation details to be confirmed

13.00
Lunch Time Refreshments

14.00
Total C5 in PNH Patients Treated with SKY59/RO7112689

  • Barbara Klughammer Senior Principal Scientist, Roche Pharma Research and Early Development

Synopsis

  • Sequential Monoclonal Antibody Recycling Technology (SMART)
  • Total C5 accumulation during treatment with C5 targeting antibodies

14.30
Panel Discussion: The Role of Translational Biomarker Activities in the Development of Complement Therapeutics

  • Simon Read Chief Scientific Officer, Ra Pharma
  • Mark Ma Senior Director, Bioanalytical and Biomarker Development, Alexion Research

Synopsis

  • Identify the current signatures that suggest complement is the primary driver of disease
  • Review current procedures of assay standardization and their deficiencies
  • Explore the next generation of translatable and standardized complement assays to allow for clinical comparisons

15.30
Afternoon Refreshments

16.30
Round Table Discussion Session

Synopsis

17.30
Chair’s Closing Remarks

17.35
End of Day One