-- Fourth quarter 2017 EXONDYS 51 unaudited revenue of $57.3
million --
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicine to treat rare neuromuscular diseases,
today pre-announced its fourth quarter 2017 revenue and also
provided full-year 2018 revenue guidance for EXONDYS 51 during the
Company’s presentation at the 36th Annual J.P. Morgan Healthcare
Conference. Sarepta’s president and chief executive officer,
Douglas Ingram, who presented on behalf of the Company, stated that
revenue for the fourth quarter will total $57.3 million and $154.6
million for the full-year of 2017. EXONDYS 51 revenue guidance for
2018 will be in the range of $295 – $305 million.
“2017 was a remarkable year for Sarepta,” said
Douglas Ingram, Sarepta’s president and chief executive officer.
“Our full-year 2017 revenue, representing one of the most
successful rare disease launches in history, speaks to the value of
EXONDYS 51 and also to the ability of our talented colleagues at
Sarepta to execute on our plans and deliver on our commitments.”
Mr. Ingram continued, “In 2017, we set the stage
by successfully launching our first therapy, entering into
important collaborations and ensuring we have ample resources to
invest in our impressive pipeline. In 2018, we are accelerating our
plans, moving 16 programs through various stages of development,
and planning for multiple milestones and inflection
points. And we are doing all of this to serve our lofty but
achievable aspiration: to improve and extend the lives of the
thousands of children suffering from DMD, expand our platform to
other rare diseases, and in so doing, to become one of the most
important global leaders in precision genetic medicine to reduce
human suffering and treat disease.”
About EXONDYS 51
EXONDYS 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 Information About
EXONDYS 51
Adverse 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.
For further information, please see the full
Prescribing Information.
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicine to treat 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.
Forward-Looking Statements
This 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 statements regarding Sarepta’s revenue from EXONDYS 51 in
the fourth quarter of 2017 and in the year 2017; Sarepta’s
full-year 2018 EXONDYS 51 revenue guidance and approximately 100
percent year-over-year growth; Sarepta’s full year 2017 revenue
representing one of the most successful rare disease launches in
history, and speaking to the value of EXONDYS 51 and also to the
ability of Sarepta’s talented employees to execute on Sarepta’s
plans and deliver on its commitments; Sarepta accelerating its
plans in 2018, moving 16 programs through development, and planning
for multiple milestones and inflection points; and Sarepta’s lofty
but achievable aspiration to improve and extend the lives of the
thousands of children suffering from DMD, expand its platform to
other rare diseases and become one of the most important global
leaders in precision genetic medicine to reduce human suffering and
treat disease.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: the audit of Sarepta’s
financial statements for the year ended December 31, 2017 is
ongoing and could result in changes to the information; Sarepta may
not be able to meet expectations with respect to EXONDYS 51 sales
or attain the net revenues it anticipates for 2018, profitability
or positive cash‐flow from operations; Sarepta may not be able to
comply with all FDA post‐approval commitments/requirements with
respect to EXONDYS 51 in a timely manner or at all; Sarepta may not
be able to obtain regulatory approval for eteplirsen in
jurisdictions outside of the U.S., including from the EMA; the
results of Sarepta’s ongoing research and development efforts,
including those with strategic partners, and clinical trials for
Sarepta’s product candidates may not be positive or consistent with
prior results or demonstrate a safe treatment benefit which could
negatively impact its business; Sarepta may not be able to execute
on its business plans and goals, including meeting its expected or
planned regulatory milestones and timelines, clinical development
plans, and bringing its product candidates to market, for various
reasons including possible limitations of Sarepta’s financial and
other resources, manufacturing limitations that may not be
anticipated or resolved for in a timely manner, results of research
and development efforts and/or clinical trials may not be positive,
and regulatory, court or agency decisions, such as decisions by the
United States Patent and Trademark Office; 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,
2016 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. 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
September 30, 2017 filed with the Securities and Exchange
Commission (SEC) as well as other SEC filings made by Sarepta.
Sarepta cautions 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.
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
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