SOUTH PLAINFIELD, N.J.,
Oct. 6, 2016 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today announced new data
supporting the potential benefit of ataluren in preserving lung
function in non-ambulatory nonsense mutation Duchenne muscular
dystrophy patients (nmDMD). The results, which are being presented
today as part of a company-sponsored symposium, are based on PTC's
analyses of lung function data from one of PTC's ongoing open-label
extension studies (Study 019) versus natural history data from a
comparable non-ambulatory cohort.
"We are excited to share the first results available from our
long-term DMD extension study in non-ambulatory patients," said
Stuart W. Peltz, Ph.D., Chief
Executive Officer of PTC Therapeutics. "The potential for a therapy
to slow disease progression and to improve outcomes beyond
ambulation is significant for patients with nonsense mutation
DMD."
DMD patients experience progressive dysfunction of respiratory
muscles, in particular the diaphragm, leading to respiratory
failure which is the primary cause of death. Pulmonary function in
DMD patients has historically been assessed using forced vital
capacity (FVC), a measure of lung function that correlates with
disease progression and mortality.i FVC tends to peak at
an earlier age in DMD patients and then decline more rapidly than
in healthy individuals.
The analysis of ataluren's impact on preservation of lung
function is based on FVC, a marker of respiratory insufficiency
which progresses with age. The results presented are from a
preliminary assessment of pulmonary function in non-ambulatory
patients in Study 019 (n = 53). The data were compared with an
external control group of patients not receiving ataluren
("untreated") in a long-term DMD natural history study performed by
the Cooperative International Neuromuscular Research Group (CINRG)
(n = 114). The CINRG cohort was matched using the following
criteria: non-ambulatory (requiring wheelchair use), ≤ 25 years
old, with ≥ 24 months of corticosteroid use. To reflect the
standard of care available during Study 019, only CINRG data from
2012 onward were included.
In order to apply a linear regression model to the analysis, FVC
data were log-transformed to achieve a normal distribution. A
piecewise linear regression model, which accounts for peak lung
function, was utilized. In the untreated natural history cohort,
FVC peaked at a mean age of 12.5 years and then declined
thereafter. In comparison, ataluren-treated patients achieved peak
FVC at a mean age of 16.5 years, a difference of 4 years over the
untreated patients. In addition, absolute FVC was 13.8% higher in
ataluren-treated patients compared with untreated patients (nominal
p = 0.005), which suggests a slower progression of loss in lung
function in ataluren-treated patients.
These historically controlled, matched analyses provide insight
into the potential pulmonary benefits realized by non-ambulatory
ataluren-treated patients versus untreated DMD patients.
"The potential benefit of ataluren in preservation of lung
function highlights the long-term benefits of a protein restoration
treatment," stated Professor Francesco
Muntoni, M.D., Director of the Dubowitz Neuromuscular
Centre, University College, London and Great Ormond Street
Hospital. "Preserving respiratory function for patients with
Duchenne muscular dystrophy is critical."
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (DMD) 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 lose the ability to walk in their early teens and experience
life-threatening lung and heart complications in their late teens
and twenties. It is estimated that nonsense mutations account for
approximately 13% of DMD cases.
About Ataluren
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. 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 under the brand name Translarna.
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.
About PTC Therapeutics
PTC is a global biopharmaceutical company focused on the
discovery, development and commercialization of orally
administered, proprietary small molecule drugs targeting an area of
RNA biology we refer to as post-transcriptional control.
Post-transcriptional control processes are the regulatory events
that occur in cells during and after a messenger RNA, or mRNA,
molecule is copied from DNA through the transcription process.
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. PTC plans to continue to develop these
compounds both on its own and through selective collaboration
arrangements with leading pharmaceutical and biotechnology
companies. For more information on the company, please visit our
website www.ptcbio.com.
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 press release, are forward-looking statements, including
statements regarding: the future expectations, plans and prospects
for PTC; PTC's preliminary analyses of data, including data from
Study 019; the clinical utility and potential advantages of
ataluren (Translarna), including its potential benefit in
preserving lung function in non-ambulatory nmDMD patients; and the
objectives of management. Other forward-looking statements
may be identified by the words "potential," "preliminary,"
"implies," "plan," "anticipate," "believe," "estimate," "expect,"
"intend," "may," "possible," "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 those related to: the outcome of final
analyses of the Study 019 data, which may vary from PTC's
preliminary analyses and identify important information not
available at the time of this press release; the initiation,
conduct and availability of data from PTC's ongoing or future
clinical trials and studies and the outcome of such trials and
studies; the eligible nmDMD patient base; PTC's scientific approach
and general development progress; 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.
As with any pharmaceutical under development, there are
significant risks in the development, regulatory approval and
commercialization of new products. There are no guarantees that
ataluren will receive full regulatory approval in any territory or
maintain its current marketing authorization in the EEA, or prove
to be commercially successful in general, or specifically with
respect to the treatment of nmDMD.
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.
i Mayer, OH et al - Characterization of Pulmonary
Function in Duchenne Muscular Dystrophy Pulmonol. 2015;50:487–494.
And Philips, M et al - Changes in Spirometry Over Time as a
Prognostic Marker in Patients with Duchenne Muscular Dystrophy Am J
Respir Crit Care Med Vol 164. pp 2191–2194, 2001
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SOURCE PTC Therapeutics, Inc.