SOUTH PLAINFIELD, N.J.,
July 18, 2017 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the
publication of the results of the ACT DMD trial study ataluren, the
only treatment for the underlying cause of nonsense mutation
Duchene muscular dystrophy (nmDMD), in the current issue of The
Lancet.
"There is a critical need for disease-modifying therapies for
this devastating fatal disorder," stated Craig M. McDonald, M.D., lead author and an
investigator of ACT DMD and professor of Pediatrics and chair of
the Department of Physical Medicine and Rehabilitation at the
University of California Davis, School
of Medicine. "The data in the publication show that ataluren
provides benefit for nonsense mutation Duchenne patients who were
in the functional range where a treatment benefit can be seen in a
one-year trial. The slowing or stabilizing of disease progression
and motor function is a highly valuable effect of drug treatment
which likely translates to longer-term benefits."
ACT DMD is a multi-center, randomized, double-blind, Phase 3
clinical trial involving 228 patients in 53 sites across 18
countries. Patients with nmDMD were randomized to receive
either ataluren 40mg/kg per day (n=114) or placebo (n=114) over 48
weeks. The primary endpoint was change from baseline in the
six-minute walk test. Treatment effects are more likely to be
observed in patients in the transition stage of disease (baseline
six-minute walk distance of 300-400 meters). Analyses of data from
pre-specified subgroups, including the pre-specified subgroup of
patients with baseline six-minute walk distance of 300 - 400
meters, were also completed. Key secondary outcome measures
were timed-function tests, including time to run or walk 10 meters
and the time to ascend or descend four stairs. Exploratory
efficacy endpoints were change in physical function as assessed by
change in the North Star Ambulatory Assessment, parent-reported
health related quality of life, and activities of daily
living.
"We are proud to have the results of the ACT DMD study published
in The Lancet and highlighted in a commentary," said
Stuart W. Peltz, Ph.D., chief
executive officer, PTC Therapeutics, Inc.
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 its corporate
headquarters are located in South Plainfield,
New Jersey. For more information
on the company, please visit our website www.ptcbio.com.
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
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 Cystic Fibrosis Foundation Therapeutics Inc. (the
nonprofit affiliate of the Cystic Fibrosis Foundation); 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.
For More Information:
Investors:
Emily
Hill
+ 1 (908) 912-9327
ehill@ptcbio.com
Media:
Jane Baj
+1 (908) 912-9167
jbaj@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.
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SOURCE PTC Therapeutics, Inc.