Benzinga | Jul 2, 2021 09:07AM EDT

BioMarin Announces 12 Presentations At International Society On Thrombosis And Haemostasis (ISTH) 2021 Virtual Congress

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced three oral presentations and nine poster presentations related to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress being held July 17-21, 2021. Notably, these presentations will include highlights from the Phase 3 GENEr8-1 trial, the largest gene therapy trial in Hemophilia A, and five years of clinical follow-up from the Phase 1/2 study, both of which continue to demonstrate prolonged hemostatic efficacy without the need for other treatment for hemophilia A.

"We are proud of the consistent and dramatic bleed control results to date, based on both long-term extension studies of at least five years, and the largest and most definitive gene therapy study in Hemophilia A. We look forward to the scientific presentations of the growing body of evidence for valoctocogene roxaparvovec and ensuing discussions at this important meeting," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin.

BioMarin's presentations at ISTH include:

Platform Presentations

Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results from the Phase 3 GENEr8-1 Trial

Professor Margareth C. Ozelo, Hematology and Transfusion Medicine, Internal Medicine Department - School of Medical Sciences of UNICAMP, University of Campinas-UNICAMP

Monday, July 19, 2021, 10-11 AM EDT

Hemostatic Response is Maintained for up to 5 Years Following Treatment with Valoctocogene Roxaparvovec, an AAV5-hFVIII-SQ Gene Therapy for Severe Hemophilia A

Professor Michael Laffan, faculty of Medicine, Department of Immunology and Inflammation at Imperial College London, Director of the Hammersmith Hospital Haemophilia Centre

Wednesday, July 21, 2021, 10-11 AM EDT

Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model

Dr. Paul Batty, Department of Pathology and Molecular Medicine, Queen's University

Wednesday, July 21, 2021, 1-2 PM EDT

Poster Presentations

Poster Title and Authors #

Global seroprevalence of pre-existing immunity against various AAV serotypes in people with haemophilia A LPB0022 Klamroth R, Hayes G, Andreeva T, Suzuki T, Hardesty B, Shima M, Pollock T, Slev P, Oldenburg J, Ozelo M, Castet S, Mahlangu J, Peyvandi F, Kazmi R, Leavitt A, Callaghan M, Pan-Petesch B, Quon D, Li M, Wong WY.

A savvy approach in clinical trial recruitment for the SAAVY (Seroprevalence of AAV AntibodY) study in the era of COVID-19: Designing for a prospective, observational study in the United States during a global pandemic PB0663 Valentino L, Vaghela M, Lauw M, Dela Cerda G, Jones M, Hinds D, Newman V, Leal-Padinas F, Rotellini D, Schafer K, Pipe S.

Exploring the level of congruence between patient- and physician-reported anxiety and depression in persons with haemophilia A PB0488 Burke T, Shaikh A, Pedra G, Hawes C, Camp C, O'Hara J.

Examination and validation of a patient-centric joint metric: "PROBLEM JOINT"; empirical evidence from the CHESS Paediatrics dataset PB0468 Burke T, Rodriguez-Santana I, O'Hara J, Chowdary P, Curtis R, Khair K, McLlaughlin P, Noone D, O'Mahoney B, Pasi J, Skinner M.

Real-world clinical and patient-centric outcomes in people with haemophilia A in France: Combined findings from the CHESS and CHESS II studies PB0452 Shaikh A, Burke T, Hawes C, Duport G, O'Hara J, Camp C.

Real-world clinical and patient-centric outcomes in people with haemophilia A in Germany: Combined findings from the CHESS and CHESS II studies PB0487 Shaikh A, Burke T, Hawes C, Becker T, Brandt S, O'Hara J, Camp C.

Real-world clinical and patient-centric outcomes in people with haemophilia A in Italy: Combined findings from the CHESS and CHESS II studies PB0464 Shaikh A, Burke T, Hawes C, Lupi A, O'Hara J, Camp C.

Real-world clinical and patient-centric outcomes in people with haemophilia A in Spain: Combined findings from the CHESS and CHESS II studies PB0456 Shaikh A, Burke T, Hawes C, O'Hara J, Camp C.

Real-world clinical and patient-centric outcomes in people with haemophilia A in the United Kingdom: Combined findings from the CHESS and CHESS II studies PB0479 Shaikh A, Burke T, Hawes C, McKeown W, Morgan D, O'Hara J, Camp C.

Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. The ISTH is an international professional membership organization with more than 7,700 clinicians, researchers and educators working together to improve the lives of patients in more than 110 countries around the world. Among its highly regarded activities and initiatives are education and standardization programs, research activities, meetings and congresses, peer-reviewed publications, expert committees and World Thrombosis Day on 13 October.