BioMarin Announces Acceptance of Late Breaking Abstract for BMN 270 at the World Federation of Hemophilia (WFH) 2016 World Co...
June 20 2016 - 3:36PM
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced the
upcoming oral presentation summarizing a late breaking abstract
accepted for the XXXII International Congress of the World
Federation of Hemophilia. The presentation will report
interim results of an open-label, Phase 1/2 study of BMN 270, an
AAV5-FVIII Gene transfer in severe Hemophilia. BMN 270 has
received orphan drug designation from the European
Commission and U.S. Food and Drug Administration.
The data will be presented in the Late Breaking
Gene Therapy session between 2:15pm-3:45pm ET in Room Hall A 4 by
John Pasi, Professor of Haemostasis and Thrombosis, Barts and the
London School of Medicine, Honorary Consultant Haematologist, The
Royal London Hospital, a lead investigator on the study. The
data presented at the WFH congress will be updated since the
Company reported preliminary data April 20, 2016.
The WFH congress is being held July 24-28, 2016 in
Orlando, Florida. A reprint of the abstract will be available
following its presentation under the Investor Relations section of
the BioMarin website at http://www.BMRN.com.
Following the oral presentation at the WFH congress
on July 27 BioMarin management will host an investor event.
BMN 270 Study DesignThe current
Phase 1/2 study is evaluating the safety and efficacy of BMN 270
gene therapy in up to 12 patients with severe hemophilia A. The
primary endpoints are to assess the safety of a single intravenous
administration of a recombinant AAV vector coding for
human-coagulation factor VIII and to determine the change from
baseline of factor VIII expression level at 16 weeks after
infusion. The kinetics, duration and magnitude of AAV-mediated
factor VIII activity in individuals with hemophilia A will be
determined and correlated to an appropriate BMN 270 dose.
This is a dose escalation study with the goal of
observing an increase in factor VIII levels. Secondary endpoints
include assessing the impact of BMN 270 on the frequency of factor
VIII replacement therapy, the number of bleeding episodes requiring
treatment and any potential immune responses. Patients will be
monitored for safety and durability of effect for five years.
About Hemophilia AHemophilia A,
also called factor VIII (FVIII) deficiency or classic hemophilia,
is a 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.1 As an X-linked
disorder, hemophilia A mostly affects males, occurring in
approximately 1 in 5,000 male births.2 People living with the
disease are not able to form blood clots efficiently and are at
risk for excessive bleeding from modest injuries, potentially
endangering their life. People with severe hemophilia often bleed
spontaneously into their muscles or joints. The standard of care
for the 43 percent of hemophilia A patients who are severely
affected, is a prophylactic regimen of factor VIII infusions three
times per week.3 Even with prophylactic regimens, many
patients still experience microbleeds and spontaneous bleeding
events that result in progressive joint damage.
About Gene TherapyGene therapy is
a treatment designed to fix a genetic problem by adding a corrected
copy of the defective gene. The functional gene is inserted into a
vector - containing a small DNA sequence - that acts as a delivery
mechanism, providing the ability to deliver the functional gene to
cells. The cells can then use the information to build the
functional proteins that the body needs, potentially reducing or
eliminating the cause of the disease. Currently, gene therapy for
the treatment of hemophilia A is available only as part of a
clinical trial. The AAV approach to gene therapy has been
advanced at the University College London (UCL) in the
treatment of hemophilia B. At UCL, this technology has shown
evidence to be both safe and effective, correcting bleeding for
greater than four years in a continuing clinical trial.
About BioMarinBioMarin is
a global biotechnology company that develops and commercializes
innovative therapies for people with serious and life-threatening
rare disorders. The company's portfolio consists of five
commercialized products and multiple clinical and pre-clinical
product candidates.
For additional information, please
visit www.BMRN.com. Information
on BioMarin's website is not incorporated by reference
into this press release.
Forward-Looking StatementThis
press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about the development
of BioMarin's BMN 270 program generally and the timing
and results of the clinical trial of BMN 270. 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 pre-clinical studies and clinical
trials of BMN 270; our ability to successfully manufacture the
product candidate for the pre-clinical 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 2015 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.
1 Source: National Hemophilia Foundation
http://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A2 Source: CDC
http://www.cdc.gov/ncbddd/hemophilia/data.html3 Source: World
Federation of Hemophilia
http://www.wfh.org/en/resources/annual-global-survey
http://www.wfh.org/en/abd/prophylaxis/prophylaxis-administration-and-dosing-schedules
Contact:
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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