SOUTH PLAINFIELD, N.J.,
June 22, 2017 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the
presentation of new analyses utilizing alternative methods to
understand the progression of Duchenne muscular dystrophy and the
benefits of Translarna in nonsense mutation Duchenne muscular
dystrophy patients. A scientific presentation, "Slope analysis of
6-minute walk distance as an alternative method to determine
treatment effect in trials in Duchenne muscular dystrophy" will be
presented today during the Neuromuscular pathologies session. In
addition, a poster entitled, "Use of a ≥5-second threshold in
baseline time to stand from supine to predict disease progression
in Duchenne muscular dystrophy" will be presented during the poster
session on Friday. The 12th Annual European Pediatric
Neurology Society Congress is being held at the Cite Internationale
in Lyon, France.
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (DMD)
is a rare and fatal genetic disorder that results in progressive
muscle weakness from early childhood and leads to premature death
in the mid-twenties due to heart and respiratory failure. It is a
progressive muscle disorder caused by the lack of functional
dystrophin protein. Dystrophin is critical to the structural
stability of skeletal, diaphragm, and heart muscles. Patients with
DMD can lose the ability to walk as early as age ten, followed by
loss of the use of their arms. DMD patients subsequently experience
life-threatening lung complications, requiring the need for
ventilation support, and heart complications in their late teens
and twenties. It is estimated that a nonsense mutation is the cause
of DMD in approximately 13 percent of patients.
About ataluren
(Translarna™)
Ataluren, discovered and
developed by PTC Therapeutics, Inc., is a protein restoration
therapy designed to enable the formation of a functioning protein
in patients with genetic disorders caused by a nonsense mutation. A
nonsense mutation is an alteration in the genetic code that
prematurely halts the synthesis of an essential protein. The
resulting disorder is determined by which protein cannot be
expressed in its entirety and is no longer functional, such as
dystrophin in Duchenne muscular dystrophy. Translarna, tradename of
ataluren, is licensed in the European Economic Area for the
treatment of nonsense mutation Duchenne muscular dystrophy in
ambulatory patients aged five years and older. Ataluren is an
investigational new drug in the United
States. The development of ataluren has been supported by
grants from the Muscular Dystrophy Association; FDA's Office of
Orphan Products Development; National Center for Research
Resources; National Heart, Lung, and Blood Institute; and Parent
Project Muscular Dystrophy.
About PTC Therapeutics
PTC is a
global biopharmaceutical company focused on the discovery,
development, and commercialization of novel medicines using our
expertise in RNA biology. PTC's internally discovered pipeline
addresses multiple therapeutic areas, including rare disorders and
oncology. PTC has discovered all of its compounds currently under
development using its proprietary technologies. Since its founding
nearly 20 years ago, PTC's mission has focused on developing
treatments to fundamentally change the lives of patients living
with rare genetic disorders. The company was founded in 1998 and is
headquartered in South Plainfield, New
Jersey. For more information on the company, please visit
our website www.ptcbio.com.
Forward Looking Statements:
All
statements, other than those of historical fact, contained in this
release are forward-looking statements, including statements
regarding the future expectations, plans and prospects for PTC; the
clinical utility and potential advantages of Translarna (ataluren);
and the objectives of management. Other forward-looking
statements may be identified by the words "look forward", "plan,"
"anticipate," "believe," "estimate," "expect," "intend," "may,"
"target," "potential," "will," "would," "could," "should,"
"continue," and similar expressions.
PTC's actual results, performance or achievements could
differ materially from those expressed or implied by
forward-looking statements it makes as a result of a variety of
risks and uncertainties, including PTC's ability to resolve the
matters set forth in the Refuse to File letter it received from the
FDA in connection with its NDA for Translarna for the treatment of
nmDMD, including whether PTC's filing of the NDA over protest with
the FDA will result in a timely or successful review of the NDA,
and whether PTC will be required to perform additional clinical and
non-clinical trials or analyses at significant cost, which, if
successful, could potentially support the approval of the NDA filed
over protest or a new NDA submission; the recommendation the
advisory committee provides to the FDA for Translarna for the
treatment of nmDMD; delays in PTC's projected regulatory timeline
for the NDA; PTC's ability to maintain its marketing authorization
of Translarna for the treatment of nmDMD in the European Economic
Area, including whether the European Medicines Agency (EMA)
determines in future annual renewal cycles that the benefit-risk
balance of Translarna authorization supports renewal of such
authorization; PTC's ability to enroll, fund, complete and timely
submit to the EMA the results of Study 041, a randomized, 18-month,
placebo-controlled clinical trial of Translarna for the treatment
of nmDMD followed by an 18-month open label extension; the
eligible patient base and commercial potential of Translarna and
PTC's other product candidates; PTC's ability to commercialize and
commercially manufacture Translarna in general and specifically as
a treatment for nmDMD; the outcome of pricing and reimbursement
negotiations in those territories in which PTC is authorized to
sell Translarna for the treatment of nmDMD; the outcome of ongoing
or future clinical studies in Translarna; expectations for
regulatory approvals; PTC's ability to meet existing or future
regulatory standards with respect to Translarna; and the factors
discussed in the "Risk Factors" section of PTC's most recent
Quarterly Report on Form 10-Q as well as any updates to these risk
factors filed from time to time in PTC's other filings with the
SEC. You are urged to carefully consider all such
factors.
The forward-looking statements contained herein represent
PTC's views only as of the date of this press release and PTC does
not undertake or plan to update or revise any such forward-looking
statements to reflect actual results or changes in plans,
prospects, assumptions, estimates or projections, or other
circumstances occurring after the date of this press release except
as required by law.
For More Information:
Investors:
Emily Hill
+ 1 (908)
912-9327
ehill@ptcbio.com
Media:
Jane
Baj
+1 (908)
912-9167
jbaj@ptcbio.com
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SOURCE PTC Therapeutics, Inc.