8:00 am Registration & Morning Coffee

8:50 am Chair’s Opening Remarks

Deciphering Key Learnings & Principals from Approval Journeys of Trailblazing Complement Drugs

9:00 am Success Stories & Lessons Learned: TAVNEOS (Avacopan) – an Oral Inhibitor of the Complement C5a Receptor for the Treatment of ANCAAssociated Vasculitis

  • Thomas J. Schall President, Chief Executive Officer & Chairman of the Board, ChemoCentryx

Synopsis

  • Providing a disease and biology overview
  • Sharing experiences from non-clinical and clinical development
  • Addressing lessons learned from the journey

Exploring Complement Targets & Unique Complement Drug Development Considerations in the Cancer Setting

9:30 am Inside-Out of Complement in Cancer Immunotherapy

  • Martin Kolev Associate Director Complement Science, Apellis Pharmaceuticals

Synopsis

  • Examining why the role of complement in cancer is complex
  • Investigating how the location of complement proteins expression and activation might determine its effect on tumorigenesis
  • New anticancer therapeutic strategies need to take this complexity into account

10:00 am Finding Optimal Targets for Complement-Based Cancer Therapy

Synopsis

  • Despite strong preclinical evidence on a role of complement in promoting cancer, complement-based cancer therapies have not yet advanced to the clinic
  • Recent pre- and clinical studies suggest that progression of several subsets of renal cell carcinoma can be driven by complement-mediated mechanisms, therefore, these subsets are potential targets for complement-based intervention
  • Levels of complement fragments in plasma associate with the response to immune checkpoint inhibitors

10:30 am Morning Refreshments & Networking Break

11:30 am Expanding Horizons in Cancer Therapeutics Targeting the Complement Pathway

  • Madhumita Das Director, Immunology Discovery, Apellis Pharmaceuticals

Synopsis

  • Unravelling novel intricacies of complement cancer nexus will offer key insights
  • Are mechanistic models authentic predictors of clinical scenario?
  • Discussing why the selection of the right patient subsets for complement inhibition is the path forward

12:00 pm Complement as a Natural Adjuvant to Promote Anti-Tumor Immunity in Cancer Therapy

  • Edward F. Patz, Jr CEO & James and Alice Chen Distinguished Professor of Radiology, Pharmacology & Cancer Biology, Grid Therapeutics, Duke University School of Medicine

Synopsis

  • Exploring how the complement system interacts with the tumor microenvironment in driving anti-tumor immunity in cancer
  • Human-derived antibodies can cause complement activation, tumor-specific cytotoxicity, and anti-tumor immunity
  • Evaluating implications for metrics in measuring success of complement therapy in cancer, and subsequent novel trial design

12:30 pm Long-Term Graft Survival Achieved in NHP Recipients of Life- Sustaining, Endogenous Retrovirus-Free, Human-Compatible (Huco™) Porcine Renal Xenografts

Synopsis

  • Understand how genome-wide inactivation of porcine retroviruses greatly reduces the risk of zoonosis in xenotransplantation
  • Human immune-compatibility of retrovirus-free porcine organ donors was enhanced using multiplex, precision genome editing to produce a safe source of human-compatible (HuCo™) porcine organ donors for human transplantation
  • Demonstrating how human complement regulatory protein expressing HuCo™ donor kidneys show long-term survival in an NHP model of life-sustaining renal xenotransplantation

1:00 pm Networking Lunch

2:00 pm C5a Neutralization by Clinical-Stage AON-D21 Shows Promise in Animal Models from Pneumonia to Lung Cancer

  • Axel Vater Founder & Chief Scientific Officer, APTARION biotech AG

Synopsis

  • AON-D21 is a mirror-image (L-)RNA aptamer that binds C5a and inhibits its signaling on C5aR1 and C5aR2
  • Analyze preclinical data showing that C5a-inhibition with AON-D21 is efficacious in diseases such as pneumonia, sepsis and lung cancer
  • AON-D21 has passed Phase 1 First in Human studies and will enter a Phase 2 Proof of Concept trial shortly

Harnessing the Power of Locally Targeted Complement Inhibitors to Achieve Desired Therapeutic Safety & Efficacy Profiles

2:30 pm Developing Locally Targeted Complement Therapeutics

Synopsis

  • Reviewing strategies for locally targeted inhibitors
  • Addressing pharmacology challenges of characterizing locally targeted drugs
  • Discussing the advantages of local targeting – potency, durability, and safety

3:00 pm Targeted Delivery Platform of sC3, a Complement Depleting Protein, Using Viral Delivery, Conjugate Guidance, & Localized Application Modalities

Synopsis

  • Discover how specific cell types in the retina and kidney can be targeted to express sC3
  • Conjugate guidance and tissue specific local delivery are being developed to increase efficacy and decrease off target effects
  • Taken together, these technologies allow Cascade to optimize treatment based on the therapeutic target

Discussing Market Access Strategies for Complement Targeted Therapies in Different Geos

3:30 pm Opportunities for Complement Therapy in China

  • Keith Wilcoxen Senior Vice President, Global Research, Zenas BioPharma

Synopsis

  • Reflecting on the significant opportunities available for complement therapies in China
  • Examining how the considerations for development in China differ from US/EU
  • Outlining unique strategies that can be employed for approval of complement inhibitors in rare disease indications

4:00 pm Chair’s Closing Remarks & End of Summit