Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced that Sarepta
and Roche have entered into a licensing agreement providing Roche
exclusive commercial rights to SRP-9001
(AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene
therapy for Duchenne muscular dystrophy (DMD), outside the United
States. Under the agreement, Sarepta will receive $1.15 billion in
an upfront payment and an equity investment; up to $1.7 billion in
regulatory and sales milestones; and royalties on net sales,
anticipated to be in the mid-teens. In addition, Roche and Sarepta
will equally share global development expenses. Sarepta retains all
rights to SRP-9001 in the United States.
The collaboration combines Sarepta’s leading
gene therapy candidate for DMD with Roche’s global reach,
commercial presence and regulatory expertise to accelerate access
to SRP-9001 for patients outside the United States. DMD is an
X-linked rare degenerative neuromuscular disorder causing severe
progressive muscle loss and premature death. SRP-9001, currently in
clinical development for DMD, is designed to deliver the
micro-dystrophin-encoding gene directly to the muscle tissue for
the targeted production of the micro-dystrophin protein.
“As a mission-driven organization, we are
inspired to partner with Roche with the goal of bringing SRP-9001
to patients outside the United States. This collaboration will not
only increase the speed with which SRP-9001 could benefit DMD
patients outside the United States, but will also greatly expand
the scope of territories within which we could potentially launch
SRP-9001 and improve and save lives,” said Doug Ingram, president
and chief executive officer, Sarepta. “In addition to the
validation that comes from joining forces with Roche, this
licensing agreement – one of the most significant ex-U.S. licensing
transactions in biopharma – will provide Sarepta with the resources
and focus to accelerate our gene therapy engine and, if
successful, bring SRP-9001 to patients as quickly as possible,
potentially transforming the lives of countless DMD patients across
the globe.”
Said James Sabry, Head of Roche Pharma
Partnering, “We are excited to enter this licensing agreement with
Sarepta. By working together to provide SRP-9001 to patients, we
hope to fundamentally transform the lives of patients and families
living with this devastating disorder for which there are currently
only limited treatment options.”
As part of the agreement, Sarepta will continue
to be responsible for the global development plan and manufacturing
build out for SRP-9001. Through its leading hybrid manufacturing
platform, Sarepta will remain responsible for manufacturing of
clinical and commercial supplies. Sarepta has also granted Roche an
option to acquire ex-U.S. rights to certain future DMD-specific
programs, in exchange for separate milestone and royalty
considerations, and cost sharing.
The closing of the transaction is subject to the
expiration or termination of the waiting period under the
Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other
customary conditions. The parties anticipate that the agreement
will close in the first quarter of 2020.
Goldman Sachs & Co. LLC is acting as the lead financial
advisor to Sarepta. Morgan Stanley & Co. LLC is also serving as
a financial advisor and Ropes & Gray LLP is serving as legal
advisor to Sarepta.
Conference Call InformationThe
conference call may be accessed by dialing (844) 534-7313 for
domestic callers and (574) 990-1451 for international callers. The
passcode for the call is 2077714. Please specify to the operator
that you would like to join the "Sarepta Therapeutics Conference
Call." The conference call will be webcast live under the investor
relations section of Sarepta's website at www.sarepta.com and will
be archived there following the call for 90 days. Please connect to
Sarepta's website several minutes prior to the start of the
broadcast to ensure adequate time for any software download that
may be necessary.
About Sarepta
TherapeuticsSarepta is at the forefront of precision
genetic medicine, having built an impressive and competitive
position in Duchenne muscular dystrophy (DMD) and more recently in
gene therapies for limb-girdle muscular dystrophy diseases (LGMD),
Charcot-Marie-Tooth (CMT), MPS IIIA and other CNS-related
disorders, totaling over 20 therapies in various stages of
development. The Company’s programs and research focus span several
therapeutic modalities, including RNA, gene therapy and gene
editing. Sarepta is fueled by an audacious but important mission:
to profoundly improve and extend the lives of patients with rare
genetic-based diseases. For more information, please visit
www.sarepta.com.
Sarepta Forward-Looking
StatementThis press release contains "forward-looking
statements." 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 but are not limited to statements regarding the closing of
the transaction; Sarepta’s right to receive any upfront payment or
equity investment from Roche pursuant to the agreement; Sarepta’s
right to receive regulatory and sales milestones, and royalty
payments from Roche pursuant to the agreement; Roche’s obligation
to share global development expenses pursuant to the agreement; the
continued development and manufacturing of SRP-9001; SRP-9001
expected delivery of micro-dystrophin-encoding gene directly to the
muscle tissue and the expected production of the micro-dystrophin
protein; the expected increased speed with which SRP-9001 could
benefit patients outside the United States and expansion of
territories within which Sarepta could launch SRP-9001; the
expectation that the licensing agreement will provide Sarepta with
the resources and focus to accelerate its gene therapy engine and
potentially bringing SRP-9001 to patients as quickly as possible
and transforming the lives of countless DMD patients across the
globe; potential regulatory approvals of SRP-9001; and the
potential launch and commercialization of SRP-9001.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta's control.
Known risk factors include, among others, market conditions, the
expected benefits and opportunities related to the licensing
agreement may not be realized or may take longer to realize than
expected due to a variety of reasons, including any inability of
the parties to perform their commitments and obligations under the
agreement, challenges and uncertainties inherent in product
research and development and manufacturing limitations; success in
preclinical testing and early clinical trials, especially if based
on a small patient sample, does not ensure that later clinical
trials will be successful, and early results from a clinical trial
do not necessarily predict final results; our data for SRP-9001 may
not be sufficient for obtaining regulatory approval; Sarepta may
not be able to execute on its business plans, including meeting its
expected or planned regulatory milestones and timelines, research
and clinical development plans, and bringing SRP-9001 to market,
for various reasons, some of which may be outside of Sarepta’s
control, including possible limitations of company financial and
other resources, manufacturing limitations that may not be
anticipated or resolved for in a timely manner, and regulatory,
court or agency decisions; and those risks identified under the
heading “Risk Factors” in Sarepta’s most recent Annual Report on
Form 10-K for the year ended December 31, 2018 and most recent
Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission (SEC) as well as other SEC filings made by the
Company which you are encouraged to review.
Any of the foregoing risks could materially and
adversely affect the Company’s business, results of operations and
the trading price of Sarepta’s common stock. 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
Information
We 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.
Sarepta Therapeutics, Inc.
Investors: Ian Estepan, 617-274-4052iestepan@sarepta.com
Media:Tracy Sorrentino,
617-301-8566tsorrentino@sarepta.com
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