8:00 am Registration & Coffee

8:45 am Chair’s Opening Remarks

Evaluating the Impact of Non-factor Products in a Changing Hemophilia Treatment Paradigm

9:00 am Assessing the Impact of New Therapies on Hemophilia Patients

  • Aric Parnes Associate Director, Boston Hemophilia Center

Synopsis

  • Understanding patient preferences in the HAVEN trials
  • Evaluating surgical outcomes within emicizumab trials
  • Clarifying the role of new therapies in inhibitor patients

9:30 am Clinical Development of Concizumab, a Monoclonal Antibody for Subcutaneous Prophylaxis for all Hemophilia Patients

  • Jesper Haaning Vice President, Global Research Project & Biopharm Management, Novo Nordisk

Synopsis

  • Assessment and discussion of Concizumab phase II data
  • Treatment that improve clinical outcomes and improve quality of life
  • Future development plans

10:00 am American Thrombosis and Hemostasis Network – A National Collaborative

  • Crystal Watson President & Chief Executive Officer, American Thrombosis & Hemostasis Network

Synopsis

ATHN proudly collaborates with researchers to provide a variety of opportunities
through our national network of hemophilia treatment centers. Learn more about:

  • ATHN core services
  • Partnering with ATHN to conduct research
  • Current and future projects

10:30 am Speed Networking & Morning Refreshments

11:30 am Application of State-of-the-art Laboratory Techniques to an Ever-changing Hemophilia Drug Landscape

  • Michael Ero Chief Executive Officer & President, Machaon Diagnostics

Synopsis

  • New tests for new drugs
  • Appropriate test selection when evaluating new hemophilia therapies
  • Linkage between clinical trial outcomes and laboratory work-ups

12:00 pm Fitusiran, An RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in People with Hemophilia

Synopsis

  • Fitusiran is a monthly subcutaneous therapy that can treat Hemophilia A, B with and without inhibitors
  • Consistent level of protection without peaks or trough
  • Rebalance the hemostatic system

Advancing Novel Prophylaxis that Improves Quality of Life for Patients

12:30 pm A Tale of Two Subcutaneous Coagulation Factors

  • Howard Levy Chief Medical Officer, Catalyst Biosciences

Synopsis

  • High potency clotting factors are required for subcutaneous (SQ) delivery
  • Pharmacokinetics (PK) of SQ delivery results in prolonged half-life and summation of blood levels
  • SQ coagulation factors have very different PK: Cmax, Tmax, half-life, mean residence time, AUC and distribution across compartments
  • Activity levels are more sustained with SQ injection than intravenous (IV) infusion
  • Prophylaxis can be achieved with SQ delivery and is particularly convenient for pediatric patients

1:00 pm Lunch & Networking

2:00 pm Research and Development of Next Generation Prophylaxis

  • Robert Peters Rare Blood Disorders Therapeutic Area Head, Research, Sanofi Genzyme

Synopsis

  • BIVV001 phase 1/2a single dose and repeat dose studies
  • Improving the treatment paradigm for patients and physicians

2:30 pm Panel Discussion: Improving Current Standard of Care

  • Robert Peters Rare Blood Disorders Therapeutic Area Head, Research, Sanofi Genzyme
  • Jesper Haaning Vice President, Global Research Project & Biopharm Management, Novo Nordisk
  • Aric Parnes Associate Director, Boston Hemophilia Center

Synopsis

  • Moving towards personalised treatment options
  • What are the roles of each therapy at what stage
  • Is there a need for an extended factor half-life product?
  • Defining protection and goal for treatment
  • New product treatment paradigm
  • How are novel therapies impacting the market

3:30 pm Afternoon Refreshments & Scientific Poster Session

4:00 pm Roundtable Discussion

Synopsis

Be a part of this practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas. This is a valuable chance for you to unite with your peers around hot topics and debate best practice.

5:00 pm Chair’s Closing Remarks

5:15 pm End of Day 1

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