Sarepta Therapeutics and BioMarin Pharmaceutical Inc. Announce Execution of a Global Settlement and a License Agreement Resol...
July 18 2017 - 8:30AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage
biopharmaceutical company focused on the discovery and development
of unique RNA-targeted therapeutics for the treatment of rare
neuromuscular diseases, and BioMarin Pharmaceutical Inc.
(NASDAQ:BMRN), a leading biotechnology company in therapies for
rare genetic diseases, announced today that Sarepta and BioMarin
executed a license agreement that provides Sarepta Therapeutics
with global exclusive rights to BioMarin’s DMD patent estate for
EXONDYS 51 and all future exon-skipping products. BioMarin retains
the right to convert the license to a co-exclusive right in the
event it decides to proceed with an exon-skipping therapy for DMD.
In addition, Sarepta and BioMarin executed a settlement agreement,
resolving the ongoing worldwide patent proceedings related to the
use of EXONDYS 51 and all future exon-skipping products for the
treatment of DMD. The effectiveness of the agreements is subject to
closing conditions including execution of necessary approvals by
Academisch Ziekenhuis Leiden (AZL) by July 24, 2017.
Under the terms of the license and settlement
agreements, Sarepta will make a one-time payment of $35 million to
BioMarin and certain additional regulatory and commercial milestone
payments for exons 51, 45, 53 and possibly on future exon-skipping
products.
In addition, Sarepta will pay royalties to
BioMarin as follows:
- Exon-skipping compounds 51, 45, and 53 and possibly on future
exon-skipping products: Sarepta will pay BioMarin 5 percent of net
sales through the end of 2023 in the United States; and
- Exon-skipping compounds 51, 45, and 53 and possibly on future
exon-skipping products: Sarepta will pay BioMarin 8 percent of net
sales through September 30, 2024 in the European Union and in other
countries where certain BioMarin / AZL patents exist.
“Upon their effectiveness, these global license
and settlement agreements provide Sarepta worldwide freedom to
operate for EXONDYS 51 and our future exon-skipping products,” said
Douglas Ingram, Sarepta’s President and Chief Executive Officer.
“The resolution of these legal matters provides us with more
certainty to fully focus our resources and energy on our crucial
mission of developing innovative medicines to improve the lives of
those impacted by DMD around the world."
“We are pleased to reach a global settlement and
license agreement with Sarepta that fairly recognizes the important
innovation by the Leiden University Medical Center and allows
patients certainty that this issue will not create a barrier to
access,” said G. Eric Davis, BioMarin’s Executive Vice President
and General Counsel.
About EXONDYS 51EXONDYS 51 uses
Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO)
chemistry and exon-skipping technology to skip exon 51 of the
dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of
dystrophin pre-mRNA, resulting in exclusion of this exon during
mRNA processing in patients with genetic mutations that are
amenable to exon 51 skipping. Exon skipping is intended to allow
for production of an internally truncated dystrophin protein. Data
from clinical studies of EXONDYS 51 in a small number of DMD
patients have demonstrated a consistent safety and tolerability
profile. The pivotal trials were not designed to evaluate long-term
safety and a clinical benefit of EXONDYS 51 has not been
established.
Important Safety
InformationAdverse reactions in DMD patients (N=8) treated
with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion
with an incidence of at least 25% more than placebo (N=4) (Study 1,
24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%),
vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most
common adverse reactions were balance disorder and vomiting.
Because of the small numbers of patients, these represent crude
frequencies that may not reflect the frequencies observed in
practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is
not recommended.
In the 88 patients who received ≥30 mg/kg/week of
EXONDYS 51 for up to 208 weeks in clinical studies, the following
events were reported in ≥10% of patients and occurred more
frequently than on the same dose in Study 1: vomiting, contusion,
excoriation, arthralgia, rash, catheter site pain, and upper
respiratory tract infection.
There have been reports of transient erythema,
facial flushing, and elevated temperature occurring on the day of
EXONDYS 51 infusion.
About Sarepta
TherapeuticsSarepta Therapeutics is a U.S.
commercial-stage biopharmaceutical company focused on the discovery
and development of unique RNA-targeted therapeutics for the
treatment of rare neuromuscular diseases. The company is primarily
focused on rapidly advancing the development of its potentially
disease-modifying Duchenne muscular dystrophy (DMD) drug
candidates. For more information, please visit www.sarepta.com.
About BioMarin Pharmaceutical
Inc.BioMarin 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 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.
Forward-Looking StatementsThis
press release contains statements that are forward-looking. Any
statements contained in this press release that are not statements
of historical fact may be deemed to be forward-looking statements.
Words such as "believes," "anticipates," "plans," "expects,"
"will," "intends," "potential," "possible" and similar expressions
are intended to identify forward-looking statements. These
forward-looking statements include statements about the license
agreement providing Sarepta with global exclusive rights to
BioMarin’s DMD patent estate for EXONDYS 51 and all future
exon-skipping products; the settlement agreement resolving the
ongoing worldwide patent proceedings related to the use of EXONDYS
51 and all future exon-skipping products for the treatment of DMD;
the payments and royalties that Sarepta will be making as part of
the settlement and license agreements; the settlement and license
agreements providing for Sarepta's worldwide freedom to operate for
EXONDYS 51 and Sarepta’s future exon-skipping products; the
settlement providing Sarepta with the certainty to fully focus its
resources and energy on its crucial mission of developing
innovative medicines to improve the lives of those impacted by DMD
around the world; and the statement that the patent proceedings
between the parties will not create for patients a barrier to
access to the innovation by the Leiden University Medical
Center.
These forward-looking statements involve risks and
uncertainties, many of which are beyond Sarepta's control. Known
risk factors include, among others: the settlement and license
agreements may not become effective if their conditions to
effectiveness are not met within the required deadline; the parties
may not be able to fulfill their commitments and obligations under
the settlement and license agreements; any future claims of
infringement by other third parties; the expected benefits and
opportunities related to the settlement and license agreements
between the parties may not be realized or may take longer to
realize than expected due to challenges and uncertainties regarding
the sales of EXONDYS 51 and the research and development of future
exon-skipping products; Sarepta may experience significant
fluctuations in sales of EXONDYS 51 from period to period and,
ultimately, Sarepta may never generate sufficient revenues from
EXONDYS 51 to reach or maintain profitability or sustain its
anticipated levels of operations; Sarepta may never receive
regulatory approval to its future exon-skipping products due to a
variety of reasons including that the results of additional
research may not be consistent with past results or may not be
positive or may otherwise fail to meet regulatory approval
requirements for the safety and efficacy of product candidates;
and even if Sarepta obtains regulatory approvals, it may not
achieve any significant revenues from the sale of such products;
Sarepta may not have worldwide freedom to operate for EXONDYS 51
and Sarepta’s future exon-skipping products due to future
proceedings brought by other parties.
Any of the foregoing risks could adversely affect
Sarepta's business, results of operations and the trading price of
Sarepta's common stock. For a detailed description of risks and
uncertainties Sarepta faces, you are encouraged to review Sarepta's
2016 Annual Report on Form 10-K and most recent Quarterly Report on
Form 10-Q for the quarter ended March 31, 2017 filed with the
Securities and Exchange Commission (SEC) as well as other SEC
filings made by Sarepta. We caution investors not to place
considerable reliance on the forward-looking statements contained
in this press release. Sarepta does not undertake any obligation to
publicly update its forward-looking statements based on events or
circumstances after the date hereof.
Internet Posting of InformationWe
routinely post information that may be important to investors in
the 'For Investors' section of our website at www.sarepta.com.
We encourage investors and potential investors to consult our
website regularly for important information about us.
Source: Sarepta Therapeutics, Inc.
Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan, 617-274-4052
iestepan@sarepta.com
or
W2O Group
Brian Reid, 212-257-6725
breid@w2ogroup.com
or
Investors:
BioMarin Pharmaceutical Inc.
Traci McCarty, 415-455-7558
Media:
BioMarin Pharmaceutical Inc.
Debra Charlesworth, 415-455-7451
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