8:15 am Coffee & Registration

9:15 am Chair’s Opening Remarks

Novel Approaches to Understand Complement in Physiology and Disease

9:30 am Classical Pathway Activation in Alzheimer’s Disease (AD)

Synopsis

• Expression of the classical pathway is induced in glial cells in both amyloidosis and tauopathy mouse models
• Knockout of C1q or C3 rescues plaque-associated synapse loss in amyloidosis mice, and ameliorates brain atrophy in tauopathy mice
• C1q and C3 are elevated at synapses in AD patient brains
• Elevated levels and activation of C4 and C3 are detected in AD patient cerebrospinal fluid

10:00 am A Diverse Pipeline of Targets and Modalities: Novartis’ Approach to Modulating the Alternative Complement Pathway

  • Thomas Smith Senior Research Investigator, Lead Discovery, Novartis
  • Christopher Adams Senior Investigator, Global Discovery Chemistry, Novartis

Synopsis

Research vignettes covering efforts to target Factor B (LMW), Factor D (LMW), Factor P (Ab) and C5 (Ab and LMW)

11:00 am Morning Refreshments and Speed Networking

12:00 pm CB 2782-PEG: A Complement Factor C3-Inactivating Protease and Potential Long-Acting Treatment for Dry AMD

Synopsis

• CB 2782-PEG is a 68 kDa engineered protease to specifically cleave C3 into inactive fragments. It has a ~500 kDa MW on SEC due to the large hydrodynamic radius
• The increased size of CB 2782-PEG resulted in enhanced ocular half-life in rabbits (3.9 vs 1.9 days for the unmodified CB 2782) and in non-human primates (3.7 vs. 1.7 days)
• Single intravitreal injection of 125 μg CB 2782-PEG achieved complete, rapid and sustained PD inhibition (>99%) of vitreous C3 for at least 28 days in African green monkeys
• The long pharmacodynamic effect is the result of the catalytic mechanism and a distinct advantage over traditional stochiometric binding inhibitors
• CB 2782-PEG has potential for anti-C3 best-in-class efficacy and convenience in geographic atrophy dry AMD with anticipated intravitreal human dosing three or four times a year

12:30 pm Development of Oral Complement Factor D inhibitors

Synopsis

• Update Achillion Development Pipeline
• Report Interim Danicopan Phase 2 Data in PNH Patients
• Provide Topline Summary on 2nd Generation of Oral Complement Factor Inhibitor

1:00 pm Update On the Retrospective MAC Plus Study of 1,000 aHUS Genetic Results and Other Laboratory News

1:30 pm Lunch & Networking

2:30 pm A Novel siRNA Approach for the Treatment of Complement-mediated Diseases

Synopsis

• Application of siRNAs in complement cascade interference – where in the complement cascade does use of a GalNAc siRNA make sense?
• Benefits of a GalNAc/liver specific treatment approach vs. other modalities
• Pre-clinical data from a new GalNAc siRNA therapy

3:00 pm Developing the First in Class Anti-C5a Antibody IFX-1: Lessons Learned from Clinical Trials

3:30 pm Afternoon Refreshments & Poster Session

4:30 pm Speed Learning: Exploring Complement Therapeutics in Various Indications & Hearing Novel Solutions

Synopsis

Short yet focused sessions allowing for multiple indications and challenges to be addressed in detail. Each table will be hosted by a leader who will give a 5 minute presentation, you then get the opportunity to discuss the topic and question the host for 10 minutes before moving on to your next table.
• Discover complement’s involvement in new indications
• Explore novel methods to evaluate complement targeting therapeutics
• Dive into discussion on groundbreaking complement research

2

6:00 pm Chair’s Closing Remarks

6:05 pm End of Day One

8:15 am Coffee & Registration

9:15 am Chair’s Opening Remarks

Novel Approaches to Understand Complement in Physiology and Disease

9:30 am Classical Pathway Activation in Alzheimer’s Disease (AD)

Synopsis

• Expression of the classical pathway is induced in glial cells in both amyloidosis and tauopathy mouse models
• Knockout of C1q or C3 rescues plaque-associated synapse loss in amyloidosis mice, and ameliorates brain atrophy in tauopathy mice
• C1q and C3 are elevated at synapses in AD patient brains
• Elevated levels and activation of C4 and C3 are detected in AD patient cerebrospinal fluid

10:00 am A Diverse Pipeline of Targets and Modalities: Novartis’ Approach to Modulating the Alternative Complement Pathway

  • Thomas Smith Senior Research Investigator, Lead Discovery, Novartis
  • Christopher Adams Senior Investigator, Global Discovery Chemistry, Novartis

Synopsis

Research vignettes covering efforts to target Factor B (LMW), Factor D (LMW), Factor P (Ab) and C5 (Ab and LMW)

11:00 am Morning Refreshments and Speed Networking

12:00 pm CB 2782-PEG: A Complement Factor C3-Inactivating Protease and Potential Long-Acting Treatment for Dry AMD

Synopsis

• CB 2782-PEG is a 68 kDa engineered protease to specifically cleave C3 into inactive fragments. It has a ~500 kDa MW on SEC due to the large hydrodynamic radius
• The increased size of CB 2782-PEG resulted in enhanced ocular half-life in rabbits (3.9 vs 1.9 days for the unmodified CB 2782) and in non-human primates (3.7 vs. 1.7 days)
• Single intravitreal injection of 125 μg CB 2782-PEG achieved complete, rapid and sustained PD inhibition (>99%) of vitreous C3 for at least 28 days in African green monkeys
• The long pharmacodynamic effect is the result of the catalytic mechanism and a distinct advantage over traditional stochiometric binding inhibitors
• CB 2782-PEG has potential for anti-C3 best-in-class efficacy and convenience in geographic atrophy dry AMD with anticipated intravitreal human dosing three or four times a year

12:30 pm Development of Oral Complement Factor D inhibitors

Synopsis

• Update Achillion Development Pipeline
• Report Interim Danicopan Phase 2 Data in PNH Patients
• Provide Topline Summary on 2nd Generation of Oral Complement Factor Inhibitor

1:00 pm Update On the Retrospective MAC Plus Study of 1,000 aHUS Genetic Results and Other Laboratory News

1:30 pm Lunch & Networking

2:30 pm A Novel siRNA Approach for the Treatment of Complement-mediated Diseases

Synopsis

• Application of siRNAs in complement cascade interference – where in the complement cascade does use of a GalNAc siRNA make sense?
• Benefits of a GalNAc/liver specific treatment approach vs. other modalities
• Pre-clinical data from a new GalNAc siRNA therapy

3:00 pm Developing the First in Class Anti-C5a Antibody IFX-1: Lessons Learned from Clinical Trials

3:30 pm Afternoon Refreshments & Poster Session

4:30 pm Speed Learning: Exploring Complement Therapeutics in Various Indications & Hearing Novel Solutions

Synopsis

Short yet focused sessions allowing for multiple indications and challenges to be addressed in detail. Each table will be hosted by a leader who will give a 5 minute presentation, you then get the opportunity to discuss the topic and question the host for 10 minutes before moving on to your next table.
• Discover complement’s involvement in new indications
• Explore novel methods to evaluate complement targeting therapeutics
• Dive into discussion on groundbreaking complement research

2

6:00 pm Chair’s Closing Remarks

6:05 pm End of Day One