SAN RAFAEL, Calif.,
June 26, 2019 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today
that Professor John Pasi, M.B.,
Ch.B., Ph.D., from Barts and the London School of Medicine and
Dentistry and principal investigator for the valoctocogene
roxaparvovec Phase 1/2 study (BMN 270-201) and the Phase 3 study
(BMN 270-301) will present data in a late breaking abstract session
at the International Society on Thrombosis and Haemostasis (ISTH)
2019 Congress being held July 6-10,
2019 in Melbourne,
Australia. More than 5,000 experts from over 90 countries in
the field will convene at the Melbourne Convention Exhibition
Centre (MCEC) to present the latest basic, translational and
clinical research and discuss the diagnostic and therapeutic
advances in bleeding and clotting disorders.
"We are pleased to share clinical data in a late breaking
abstract on our novel investigational gene therapy treatment for
severe hemophilia A at the leading scientific event for thrombosis,
hemostasis and vascular biology," said Hank
Fuchs, M.D., President Worldwide Research and Development at
BioMarin. "Researching a gene therapy treatment for people with
severe hemophilia A has the potential to address a significant
unmet medical need and advance the standard of care."
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. ISTH is an international professional
membership organization with more than 5,000 clinicians,
researchers and educators working together to improve the lives of
patients in more than 100 countries around the world. Among its
highly regarded activities and initiatives are education and
standardization programs, research activities, yearly congresses,
peer-reviewed publications, expert committees and World Thrombosis
Day on 13 October.
Oral Presentation – Late Breaking Abstract Session
Title
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Presenter
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First-in-human
Evidence of Durable Therapeutic
Efficacy and Safety of AAV Gene Therapy Over
Three-years with Valoctocogene Roxaparvovec for
Severe Haemophilia A (BMN 270-201 Study)
Presentation:
Monday, July 8 10:45
– 12:00 (AEST or GMT +10)
Location: Plenary Hall
Presentation
Number: LB 01.2
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John Pasi, M.B.,
Ch.B., Ph.D., Barts
and the London School of Medicine
and Dentistry
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About Hemophilia A
People living with hemophilia A lack enough FVIII protein to
help their blood clot and are at risk for painful, potentially
life-threatening bleeds from even modest
injuries. Additionally, people with severe hemophilia A often
experience painful, spontaneous bleeds into their muscles or
joints. The standard of care for the 43 percent of individuals with
hemophilia A who are severely affected is a prophylactic regimen of
Factor VIII infusions administered intravenously two to three times
per week. Despite these regimens, many people continue to
experience bleeds, resulting in progressive and debilitating joint
damage which can have a major impact on their quality of
life. Hemophilia A, also called factor VIII (FVIII) deficiency
or classic hemophilia, is an x-linked genetic disorder caused by
missing or defective factor VIII, a clotting protein. Although it
is passed down from parents to children, about 1/3 of cases are
caused by a spontaneous mutation, a new mutation that was not
inherited. Approximately 1 in 10,000 people is born with Hemophilia
A.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for serious and
life-threatening rare and ultra-rare genetic diseases. The
Company's portfolio consists of seven commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.biomarin.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
Forward Looking Statements
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc.(BioMarin),
including without limitation, statements about the development of
BioMarin's valoctocogene roxaparvovec program generally; the Phase
1/2 study with valoctocogene roxaparvovec; and the impact of
valoctocogene roxaparvovec gene therapy for treating people with
severe hemophilia A;. These forward-looking statements are
predictions and involve risks and uncertainties such that actual
results may differ materially from these statements. These risks
and uncertainties include, among others: results and timing of
current and planned preclinical studies and clinical trials of
valoctocogene roxaparvovec, including final analysis of the above
interim data; additional data from the continuation of this Phase
1/2 trial, any potential adverse events observed in the continuing
monitoring of the participants in the clinical trials; the content
and timing of decisions by the U.S. Food and Drug Administration,
the European Commission and other regulatory authorities; the
content and timing of decisions by local and central ethics
committees regarding the clinical trials; errors and deficiencies
in our durability modeling; our ability to successfully manufacture
valoctocogene roxaparvovec for the clinical trials and
commercially, if approved; and those other risks detailed
from time to time under the caption "Risk Factors" and elsewhere in
BioMarin's Securities and Exchange Commission (SEC) filings,
including BioMarin's Quarterly Report on Form 10-Q for the quarter
ended March 31, 2019, and future filings and reports by
BioMarin. BioMarin undertakes no duty or obligation to update any
forward-looking statements contained in this press release as a
result of new information, future events or changes in its
expectations.
BioMarin® is a registered trademark of BioMarin Pharmaceutical
Inc.
Contact:
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Investors:
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Media:
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Traci
McCarty
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Debra
Charlesworth
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BioMarin
Pharmaceutical Inc.
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BioMarin
Pharmaceutical Inc.
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(415)
455-7558
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(415)
455-7451
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SOURCE BioMarin Pharmaceutical Inc.