SAN RAFAEL, Calif.,
June 16, 2017 /PRNewswire/ --
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that
the company will present data in a late breaking abstract session
at the International Society on Thrombosis and Haemostasis (ISTH)
2017 Congress being held July 8-13,
2017 in Berlin, Germany.
The presentation will report interim results from a Phase 1/2 study
of BMN 270, an AAV5-FVIII Gene transfer in severe hemophilia.
"We feel privileged to be presenting the only human data in gene
therapy for hemophilia A at ISTH," said Hank Fuchs, M.D., President Worldwide Research
and Development at BioMarin. "We are committed to further our
ongoing clinical program with BMN 270, which has the potential to
change the course of hemophilia A treatment, the most common form
of the disease, and look forward to sharing our latest
findings."
ISTH is a global not-for-profit membership organization
advancing the understanding, prevention, diagnosis and treatment of
thrombotic and bleeding disorders. The congress hosts the world's
leading experts on thrombosis, hemostasis and vascular biology,
presenting the most recent advances to improve patient care.
Oral Presentation – Late Breaking Abstract Session
Title
|
Authors
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Interim Results from
a Phase 1/2 AAV5-FVIII
Gene Transfer in Patients with Severe
Hemophilia
A
Presentation:
July 11 at 9:30-9:38 AM CEST
|
J. Pasi, S.
Rangarajan, L. Walsh, W.
Lester, D. Perry, B. Madan, H. Yu, G.F.
Pierce, W.Y. Wong
|
Conference Call to be held Tuesday, July 11th at
2:30pm CEST/8:30am ET
Interested parties may access a live webcast of the conference
call via the investor section of the BioMarin
website, www.biomarin.com. The Late Breaker slide presentation
will be available to download in advance of the call. A
replay of the call will be archived on the site for one week
following the call.
U.S. / Canada Dial-in Number: (866) 502-9859
International Dial-in Number: (574) 990-1362
Conference ID: 41581309
Replay Dial-in Number: (855) 859-2056
Replay International Dial-in Number: (404) 537-3406
Conference ID: 41581309
About Hemophilia A
Hemophilia A is a genetic disease caused by the deficiency of
clotting factor VIII. It is the most common type of hemophilia and
occurs much more frequently in males; incidence is estimated at 1
in 4,000-5,000 male births. People born with hemophilia produce
little or no clotting factors. The two main types of hemophilia are
A and B. People with hemophilia A are missing or have low levels of
clotting factor VIII.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for patients with serious and
life-threatening rare and ultra-rare genetic diseases. The
Company's portfolio consists of six commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.BioMarin.com.
Forward Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: our research on hemophilia A
treatment and our potential to change the course of hemophilia A
treatment. 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 BMN 270, including final
analysis of the Phase 1/2 data; any potential adverse events
observed in the continuing monitoring of the patients in the Phase
1/2 trial; the content and timing of decisions by the U.S. Food and
Drug Administration, the European Commission and other regulatory
authorities; a potential decision by the EMA to remove BMN 270 from
the PRIME scheme or the accelerated assessment framework; the
content and timing of decisions by local and central ethics
committees regarding the clinical trials; our ability to
successfully manufacture the product candidate for the preclinical
and clinical trials; and those factors detailed in BioMarin's
filings with the Securities and Exchange Commission, including,
without limitation, the factors contained under the caption "Risk
Factors" in BioMarin's 2016 Annual Report on Form 10-K, and the
factors contained in BioMarin's reports on Form 10-Q. Stockholders
are urged not to place undue reliance on forward-looking
statements, which speak only as of the date hereof. BioMarin is
under no obligation, and expressly disclaims any obligation to
update or alter any forward-looking statement, whether as a result
of new information, future events or otherwise.
BioMarin® is a registered trademark of BioMarin
Pharmaceutical Inc.
Contact:
|
|
Investors:
|
Media:
|
Traci
McCarty
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
BioMarin
Pharmaceutical Inc.
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(415)
455-7558
|
(415)
455-7451
|
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SOURCE BioMarin Pharmaceutical Inc.