SAN RAFAEL, Calif.,
May 3, 2020 /PRNewswire/ -- BioMarin
Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the company
has entered into a preclinical collaboration and license agreement
with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to
develop novel gene therapies to treat rare genetic
cardiomyopathies. DiNAQOR will receive an undisclosed upfront
payment and is eligible to receive development, regulatory and
commercial milestones on product sales in addition to tiered
royalties on worldwide sales. The company did not disclose
financial terms. BioMarin management reiterated its 2020 GAAP
net income guidance of $20 to
$80 million, inclusive of this
collaboration.
The license initially covers DiNAQOR's lead program, DiNA-001
for MYBPC3 hypertrophic cardiomyopathy (HCM). Additionally,
the companies will collaborate on several of DiNAQOR's other
pipeline programs, and BioMarin has the option to extend the
license to include these additional programs on similar
terms. Reflecting the long-term commitment to the
collaboration, BioMarin is simultaneously investing in DiNAQOR.
"With this agreement, BioMarin is continuing to apply its gene
therapy know-how and manufacturing expertise in new areas like
cardiology," said Jean-Jacques Bienaimé, Chairman and Chief
Executive Officer at BioMarin. "This collaboration extends
our global leadership position in gene therapy and boosts our
potential to transform the lives of patients worldwide with rare
genetic cardiomyopathies."
"We are thrilled to collaborate with the researchers at DiNAQOR
to conduct this pioneering work on the development of gene
therapies for inherited cardiomyophathies," said Lon Cardon, Chief Scientific Strategy Officer
and Senior Vice President at BioMarin. "We believe there is
tremendous potential in combining our experience in gene therapy
research and development with DiNAQOR's in-depth knowledge of
genetic heart diseases."
DiNAQOR was founded and is led by several leading pharmaceutical
and biotechnology executives and academics with deep cardiology and
gene therapy expertise. The company's holistic approach to
gene therapy is focused on gene therapies for the heart that
deliver a medical solution that can safely deliver gene therapies
to the heart muscle, ensure transduction of the cardiac cells, and
limit the exposure of the therapy to other organs.
"BioMarin is a global leader in rare disease research,
development and commercialization, and their commitment to DiNA-001
is a powerful validation of DiNAQOR's gene therapy platform," said
Dr. Johannes Holzmeister,
Co-Founder, Chairman and CEO at DiNAQOR. "We believe our platform
has many potential applications and this milestone agreement will
enable us to invest in expanding our genetic medicine
pipeline."
"Momentum for gene therapies continues to build, and BioMarin
has demonstrated tremendous scientific, clinical, and manufacturing
leadership and expertise in the space," said Thomas Voit, M.D., Ph.D., Co-Founder and Chief
Scientific Officer at DiNAQOR and Director of the Biomedical
Research Centre at the Great Ormond Street Hospital and the UCL
Institute of Child Health, University College London. "We are
looking forward to combining our strengths to expand the promise of
gene therapy treatments by targeting the heart muscle to treat rare
genetic cardiomyopathies."
About HCM and MYPBC3
Hypertrophic cardiomyopathy (HCM) is one of the most common
genetic heart diseases, with about 500,000 patients diagnosed with
HCM worldwide. Up to 60% of HCM cases have a genetic origin,
and it is estimated that 40% of those have mutations in MYBPC3, the
gene that encodes cardiac myosin-binding protein C (MyBP-C).
HCM affects the heart muscle, causing the muscle to
enlarge. HCM patients have an increased risk of developing heart
failure and life-threatening arrhythmias. There are no approved
pharmacological treatment options available that address the
underlying disease biology of HCM and invasive surgery or heart
transplantation may be the only options available for patients with
advanced disease.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for serious and
life-threatening 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. Information on BioMarin's website is
not incorporated by reference into this press release.
About DiNAQOR
Founded in 2019, DiNAQOR AG is a global gene therapy platform
company focused on advancing novel solutions for patients suffering
from heart disease. The company's lead preclinical program,
DiNA-001 is focused on the treatment of MYBPC3-linked
cardiomyopathy. DiNAQOR is headquartered in Pfäffikon, Switzerland, with additional presence in
London, England and Boston, Massachusetts (US). For more
information visit www.dinaqor.com.
Forward Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: BioMarin's expectations
regarding the announced collaboration, the prospects for the lead
and follow on pipeline products and it's 2020 GAAP
profitability. 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; the
content and timing of decisions by the U.S. Food and Drug
Administration, the European Commission and other regulatory
authorities concerning the programs; the ability to manufacture the
product candidates, BioMarin's revenue for 2020, especially with
the possible impact of COVID-19, 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, 2020, 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.
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SOURCE BioMarin Pharmaceutical Inc.