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BioMarin to Present Findings from Ongoing Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the World Federation of Hemophilia 2022 World Congress, May 8-11, 2022, Including Five Platform Presentations

Published: 2022-05-05 12:29:00 ET

Ongoing Clinical Development Program Represents Largest and Longest Development Program for any Gene Therapy in Hemophilia A, Demonstrates Commitment to Advancing Care for People with Hemophilia A

SAN RAFAEL, Calif., May 5, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced five platform presentations and one poster presentation on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the World Federation of Hemophilia (WFH) 2022 World Congress from May 8-11 in Montreal, Canada.

"We're pleased to provide updated data on durability of effect in patients treated over two years ago, on molecular contributors to variability, on hepatotoxicity and the role of immunosuppression, on the potential for integration-related oncogenicity, and on health-related quality of life. We continue to learn about the potential for investigational valoctocogene roxaparvovec to transform lives and the optimal way to manage patients through their journey," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We are progressing our regulatory efforts with the intent to deliver a therapy that may represent an important and valuable treatment choice for patients with severe Hemophilia A, as no presently available treatment offers such freedom from prophylaxis and reduced bleeding frequency at the same time."

"BioMarin is generating valuable data about investigational valoctocogene roxaparvovec that potentially could enable shared decision making between physicians and patients on what is the best therapy in each personal situation," said Professor Wolfgang Miesbach, Head of the Department of Coagulation Disorders and the Comprehensive Care Haemophilia Centre at the Goethe University Hospital in Frankfurt/Main, Germany.

Presentation of data at WFH follows positive two-year results from the ongoing, global phase 3 GENEr8-1 study of valoctocogene roxaparvovec presented at a medical meeting earlier in the year, as well as publication of one-year results from the pivotal clinical trial in the New England Journal of Medicine in March 2022.

BioMarin's presentations at WFH include:

Platform Presentations

Immune suppression following gene therapy in HemophiliaProfessor Wolfgang Miesbach, Head of the Department of Coagulation Disorders and the Comprehensive Care Haemophilia Centre, Goethe University Hospital, Frankfurt/Main, GermanyMonday, May 9, 2022, 1:30 -2:30 PM ET

Exploratory analyses of healthy liver biopsies and a single case of parotid acinar cell carcinoma do not identify a role for valoctocogene roxaparvovec vector insertion in altering cell growthKevin Eggan, Group Vice President, Head of Research and Early Development / BioMarinMonday, May 9, 2022, 1:30 -2:30 PM ET

Health-related quality of life over 2 years following valoctocogene roxaparvovec adeno-associated virus gene transfer for severe hemophilia A: Results from GENEr8-1Dr. Amy Dunn, Director of Pediatric Hematology, Nationwide Children's Hospital, Columbus, OhioTuesday, May 10, 2022, 1:30-2:30 PM ET

Human liver biopsy analysis showed interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A Sylvia Fong, Head of Hematology Research, BioMarinWednesday, May 11, 2022, 1:30 -2:30 PM ET

Interim 52-week analysis of immunogenicity to the vector capsid and transgene-expressed human FVIII in GENEr8-1, a phase 3 clinical study of valoctocogene roxaparvovec, an AAV5-medicated gene therapy for hemophilia A Brian Long, Principal Scientist, Clinical Immunology, BioMarinWednesday, May 11, 2022, 4-5 PM ET

Poster Presentation

Use of immunosuppressives in patients with hemophilia receiving gene therapy: Evidence generation using a mixed-methods approach Professor Wolfgang Miesbach, Head of the Department of Coagulation Disorders and the Comprehensive Care Haemophilia Centre, Goethe University Hospital, Frankfurt/Main, Germany

BioMarin-Sponsored Symposia

Gene Therapy Clinical Trial Patient Journey: A Look Into Shared Decision MakingMonday, May 9, 12:15 – 1:15 PM ET

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels

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