Summit Fuels Discussion on New and Upcoming Hemophilia Treatments

For the second year, key pharmaceutical and academic leaders in hemophilia therapy development will gather at the Hemophilia Drug Development Summit — to be held in Boston Aug. 20–22 —to discuss and advance the next generation of safe and more effective therapies for bleeding disorders.

The recent approval of  Genentech’s Hemlibra (emicizumab-kxwh) in Europe and the United States created a new race to market more successful long-acting gene therapies for hemophilia and other bleeding disorders.

The summit was designed to help large pharmaceutical and biotech companies overcome translational research hurdles and be able to deliver more treatment strategies that can improve patients’ quality of life and clinical outcomes.

It is expected to bring together more than 100 of the world’s leading hemophilia experts and treatment developers. Based on last year’s audience numbers, half of the attendees are expected to be treatment developers. Equipment and service providers (14%), healthcare providers (14%), research institute representatives (10%), and other members of the bleeding disorders community will round out the summit.

Participants will have the opportunity to discuss the development and impact of non-factor products, and contribute to the development of better testing methods and assays to ensure adequate measurement of response to treatment and diagnosis.

Discussions also will focus on strategies for patient advocacy and engagement, and ways to overcome the hurdles in market access to new hemophilia treatments.

Organizers say the event aims to unite pharmaceutical, biotech, and academic figures on “working to translate and clinically develop longer-acting and more efficacious therapies” to overcome significant unmet medical needs of those affected by hemophilia and other bleeding disorders.

Several key aspects of the clinical-to-commercial pathway for bleeding disorder therapies will be addressed, including:

• Research and development, patient engagement, and payer challenges of gene therapies
• Development progression of novel subcutaneous prophylaxis strategies for inhibitor-positive patients
• Optimization of clinical development strategies
• Strategies to overcome the knowledge gap in the diagnosis and treatment of Von Willebrand disease
• Understanding of the needs of patients and insight from clinicians

The summit will have 20 speakers, including Howard Levy, chief medical officer of Catalyst Biosciences; Sander Van Deventer, chief scientific officer of uniQure; Dean Falb, chief scientific officer at LogicBio; Robert Peters, head of research of rare blood disorders at Sanofi Genzyme; Dawn Rotellini, senior vice president of program development of the National Hemophilia Foundation; and Didier Rouy, senior director of clinical sciences at Sangamo Therapeutics.

Pharmaceutical companies including Sanofi, CSL Behring, Expression Therapeutics, Catalyst Biosciences, Novo Nordisk, and Bayer, among others, will be represented at the summit.

Similar to the first meeting, this year’s participants will have the opportunity to register for two pre-conference workshops, set for Aug. 20.

• Workshop A will be led by Grant Blouse, vice president of translational research at Catalyst Biosciences, and Nisha Jain, global head, life cycle management of rare blood disorders at Sanofi Genzyme. The workshop will address immune reactions in next-generation factor replacement prophylaxis.
• Workshop B will be led by Michelle Rice, senior vice president of external affairs at the National Hemophilia Foundation; Brendan Hayes, director of external affairs at the National Hemophilia Foundation; and Edmund Pezalla, CEO and founder of Enlightenment Bioconsult. The workshop will address new payer and reimbursement strategies to improve patient access to gene therapies.
• The summit’s full event guide is available here.

Early registration discounts are offered through July 12; more information is available here. Pricing differs according to type of registration (industry or academic, for instance), and savings of up to $600 off regular prices are available.

The meeting’s venue is Wyndham Boston Beacon Hill, 5 Blossom St., Boston. Registration details can be found online, by email at [email protected], or by calling 617-455-4188.