BioMarin Enrolls First Patient in Phase 1/2 Trial of Gene Therapy Drug Candidate BMN 270 for the Treatment of Hemophilia A
September 28 2015 - 4:09PM
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that it
has enrolled the first patient in a Phase 1/2 trial for BMN 270, an
investigational gene therapy for the treatment of patients with
hemophilia A. BMN 270 is an AAV-factor VIII vector, designed to
restore factor VIII plasma concentrations, essential for blood
clotting in patients with hemophilia A. The gene therapy program
for Hemophilia A was originally licensed from University College
London and St. Jude Children's Research Hospital in February 2013
and has since been developed at BioMarin's facilities.
"Hemophilia A results from mutations at the genetic level,
making gene therapy a potentially powerful technique to treat
patients with a single dose," stated Hank Fuchs, M.D., Executive
Vice President, Chief Medical Officer of BioMarin. "For the first
clinical trial of BMN 270, we are looking to demonstrate that
treatment with BMN 270 increases the expression of the factor VIII
protein, necessary for blood clotting."
"The global bleeding disorders community greatly benefits from a
wide range of support to help advance our vision of Treatment for
All," said Alain Weill, World Federation of Hemophilia (WFH)
President. "We welcome BioMarin as a new WFH Corporate Partner and
greatly appreciate their commitment to support people with
hemophilia A through their innovative gene therapy research."
Study Design
The Phase 1/2 study will evaluate 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, human-coagulation Factor VIII
vector 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 for five
years.
About Hemophilia A
Hemophilia 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
Approximately 1 in 10,000 people is born with Hemophilia
A.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 spontaneous bleeding
events that result in progressive and debilitating joint
damage.
About Gene Therapy
Gene therapy is a form of 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, in the United States, gene therapy is available only as
part of a clinical trial.
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 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 Statement
This 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 planned 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 preclinical
studies and clinical trials of BMN 270; the content and timing of
decisions by the U.S. Food and Drug Administration, the European
Commission and other regulatory authorities; 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 2013 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.
Vimizim™ is our trademark, and BioMarin®,
Naglazyme®, Kuvan®, Firdapse® are registered trademarks of BioMarin
Pharmaceutical Inc.
Aldurazyme® is a registered trademark of
BioMarin/Genzyme LLC.
1 Source: National Hemophilia Foundation
http://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A
2 Source: World Federation of Hemophilia
http://www.wfh.org/en/page.aspx?pid=637#How_common
3 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
BioMarin Pharmaceutical (NASDAQ:BMRN)
Historical Stock Chart
From Mar 2024 to Apr 2024
BioMarin Pharmaceutical (NASDAQ:BMRN)
Historical Stock Chart
From Apr 2023 to Apr 2024