SAN RAFAEL, Calif.,
Sept. 26, 2019 /PRNewswire/ --
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN), a pioneer in treatments
for rare disease Phenylketonuria (PKU) and in gene therapies, today
announced that it has submitted a Clinical Trial Application (CTA)
with the Medicines and Healthcare Products Regulatory Agency (MHRA)
in the U.K. for BMN 307, an investigational AAV5-phenylalanine
hydroxylase (PAH) gene therapy designed to reduce blood
phenylalanine (Phe) concentrations levels in patients with PKU. BMN
307 will be evaluated to determine whether a single dose of
treatment can restore Phe metabolism in patients with PKU,
normalize plasma Phe level, and enable a normal diet. The
company expects to start enrolling patients in a Phase 1/2 trial
early next year and is actively preparing regulatory submissions
for other countries. BMN 307 represents a potential third PKU
treatment option in BioMarin's PKU franchise and its second gene
therapy development program. BMN 307 follows BioMarin's first
investigational gene therapy program, valoctocogene roxaparvovec
for severe hemophilia A, currently in a Phase 3 study.
"This clinical trial application marks the latest milestone in
BioMarin's 15-plus year commitment to the PKU community.
BioMarin has brought the only two approved therapies for PKU to
patients around the world," said Hank
Fuchs, President, Worldwide Research and Development at
BioMarin. "Leveraging our expertise in gene therapy, we are
pleased to be adding a new gene therapy product, which is
potentially a transformative solution, to build on our current
achievements for the PKU community."
PKU is a rare genetic disease that manifests at birth and is
marked by an inability to break down Phe, an amino acid that is
found in most forms of protein. Left untreated, high levels of Phe
become toxic to the brain and may lead to serious neurological and
neuropsychiatric-related issues, affecting the way a person thinks,
feels, and acts. Due to the seriousness of these symptoms, in many
countries, infants are screened at birth to ensure early diagnosis
and treatment to avoid intellectual disability and other
complications. According to treatment guidelines, PKU patients
should maintain lifelong control of their Phe levels.
Phase 1/2 Study
The Phase 1/2 study will evaluate the safety efficacy and
tolerability of a single intravenous administration of BMN 307 in
patients with PKU. The Phase 1/2 study consists of a
dose-escalation phase, followed by a dose expansion phase. In
addition, there is also an observational study in PKU patients to
measure markers of disease and clinical outcomes over time
in patients.
BioMarin's 15-Plus Year Commitment to PKU Research
For more than 15 years, BioMarin has been a pioneer in ongoing
research to help improve the lives of PKU patients. BioMarin
has treated approximately 7,000 PKU patients around the
world. The company has two approved PKU therapies, in
addition to the investigational gene therapy BMN 307 in
development. BioMarin has conducted 40 clinical studies in
PKU and has sponsored 38 external clinical studies. BioMarin
researchers have authored 54 publications in medical and scientific
journals on PKU, and BioMarin has supported another 52 publications
by external researchers.
About Gene Therapy
Gene therapy is a form of treatment designed to address 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.
Gene Therapy Manufacturing
BioMarin has constructed, commissioned, and validated one of the
first gene therapy manufacturing facilities of its kind in the
world, which is located in Novato, California. This
facility can support up to 4,000 doses per year, and the production
process was developed in accordance with International Conference
on Harmonisation guidance to facilitate eventual worldwide
registration with health authorities. In 2018, the
International Society for Pharmaceutical Engineering (ISPE)
selected the Company's gene therapy manufacturing facility as the
Facility of the Year Category Winner for Project Execution. The BMN
307 PKU gene therapy clinical production occurred at commercial
scale in this facility. Production of material at commercial
scale in the state of the art facility significantly reduces risk
associated with making process changes later in development and can
speed overall development timelines.
About Phenylketonuria
PKU, or PAH deficiency, is a genetic disorder affecting
approximately 50,000 diagnosed patients in the regions of the world
where BioMarin operates and is caused by a deficiency of the enzyme
PAH. This enzyme is required for the metabolism of Phe, an
essential amino acid found in most protein-containing foods. If the
active enzyme is not present in sufficient quantities, Phe
accumulates to abnormally high levels in the blood and becomes
toxic to the brain, resulting in a variety of complications
including severe intellectual disability, seizures, tremors,
behavioral problems and psychiatric symptoms. As a result of
newborn screening efforts implemented in the 1960s and early 1970s,
virtually all individuals with PKU under the age of 40 in countries
with newborn screening programs are diagnosed at birth and
treatment is implemented soon after. PKU can be managed with a
Phe-restricted diet, which is supplemented by low-protein modified
foods and Phe-free medical foods; however, it is difficult for most
adult patients to adhere to the strict diet to the extent needed
for achieving adequate control of blood Phe levels.
To learn more about PKU and PAH deficiency, please visit
www.PKU.com. Information on this website is not incorporated by
reference into this press release.
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 seven commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.biomarin.com.
Information on such 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. (BioMarin),
including, without limitation, statements about: the development of
BioMarin's BMN 307 program generally, BioMarin's planned
submissions to regulatory authorities for BMN 307, BioMarin's gene
therapy manufacturing capabilities, and the timing and results of
BioMarin's planned Phase 1/2 trial of BMN 307. 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 307; the content and timing of
decisions by the U.S. Food and Drug Administration, the European
Commission and other regulatory authorities; uncertainties inherent
in research and development, including unfavorable new clinical
data and additional analyses of existing clinical data; the results
and timing of current and future clinical trials related to BMN
307; the risks related to commercialization of BMN 307 and our
ability to manufacture sufficient quantities of BMN 307; and those
factors detailed in BioMarin's filings with the Securities and
Exchange Commission (SEC), including, without limitation, the
factors contained under the caption "Risk Factors" in BioMarin's
Quarterly Report on Form 10-Q for the quarter ended June 30, 2019 as such factors may be updated by
any subsequent reports. 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.
Contacts:
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Investors
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Media
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Traci
McCarty
|
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.