SOUTH PLAINFIELD, N.J.,
Oct. 6, 2018 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today announced preliminary data
from the first international drug registry for Duchenne patients
receiving Translarna™ (ataluren), underscoring the
long-term clinical benefit of Translarna when used in routine
clinical practice in delaying irreversible muscle loss in children
with Duchenne caused by a nonsense mutation, when compared with
published natural history. The data show that children and
adolescents receiving Translarna in the real-world setting are
continuing to walk years longer than untreated children, and are
staying more physically able. A time-to-event analysis for loss of
ambulation has shown that patients on Translarna had a median age
of loss of ambulation of 16.5 years of age – up to 5 years later
than seen with natural disease progression in untreated children.
The data were presented as a late breaker at the 23rd International
Annual Congress of the World Muscle Society in Argentina.
Experience the interactive Multichannel News Release here:
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"The results from the Registry data are exceptionally important
because they further substantiate the results observed in our
analyses of data from previous clinical trials studying Translarna
in Duchenne," said Stuart W. Peltz,
Ph.D., CEO, PTC Therapeutics. "Delaying the loss of
ambulation is critical to these patients, as it predicts the time
to loss of pulmonary function and mortality."
Patients who received Translarna in routine clinical practice
also experienced a slower decline in their physical function
compared with the placebo arm of Phase 3 Study 020, as measured by
a series of timed function tests. Safety outcomes for patients in
the STRIDE Registry were consistent with the known safety profile
of Translarna.
"These initial data are very encouraging because they provide
the first, real-world evidence for the impact of Translarna when
used routinely, longer term," said Dr. Eugenio Mercuri, Professor, Pediatric Neurology,
Catholic University. "We are
seeing children who have been exhibiting first clinical symptoms of
Duchenne at approximately 3 years of age and yet are still able to
walk years after they would normally be wheelchair-bound, which is
what matters most to patients and their families."
The analysis was based on data captured from 216 patients, the
majority of whom had not been previously enrolled in an ataluren
clinical trial, across 11 European countries and Israel. Patients had a mean age of 9.8 years
at first assessment and had been diagnosed with Duchenne caused by
a nonsense mutation at approximately five years of age. Almost 90%
had previously or were still receiving corticosteroids.
"Keeping a child with Duchenne walking for as long as possible
is crucial not only to maintain independence, but to delay the
rapid deterioration that usually follows loss of ambulation,
including loss of the use of arms, which are essential for
self-care, and respiratory and cardiac complications," said
Filippo Buccella, an author of the
study and Duchenne patient advocate. "As a father to a son with
Duchenne, slowing the progression of this devastating disease is
vital to maintaining quality of life for the patient as well as the
family."
About Translarna (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, the tradename of ataluren, is
licensed in the European Economic Area for the treatment of
nonsense mutation Duchenne muscular dystrophy in ambulatory
patients aged two 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 the STRIDE Registry
The STRIDE (Strategic Targeting of Registries and International
Database of Excellence) Registry is an ongoing, multicenter,
observational study of the safety and effectiveness of Translarna
in routine care. It is the first patient data repository to
provide real-world experience regarding the long-term use of
Translarna in routine clinical practice. Enrolled patients will be
followed for at least 5 years from the date of enrollment, or until
withdrawal from the study. As of 9 July 2018, 216 patients with a mean age of 9.8
years had been enrolled across 11 countries in Europe and Israel.
Effectiveness information may include neuromuscular function (as
measured for example by timed-function tests, the North Star
Ambulatory Assessment, and Performance of the Upper Limb (PUL)
measures, cardiac function (including echocardiogram where
available), pulmonary function (including spirometry measures), and
quality of life measures. Assessments of musculoskeletal health,
rehabilitation, orthopedic and gastrointestinal management, as well
as other measures of psychosocial management, will be collected to
allow for comparison of patient health-management activities in
routine clinical care to those of published treatment
guidelines.
STRIDE is a collaborative partnership between TREAT-NMD and PTC
Therapeutics, led by a Steering Committee comprised of leading
experts in Duchenne, patient advocates from around the world and
PTC representatives.
The Registry also fulfils a post-marketing commitment to the
Pharmacovigilance Risk Assessment Committee of the European
Medicines Agency.
About TREAT-NMD
TREAT-NMD is a network for the neuromuscular field that provides an
infrastructure to ensure that the most promising new therapies
reach patients as quickly as possible. Since its launch in
January 2007 the network's focus has
been on the development of tools that industry, clinicians and
scientists need to bring novel therapeutic approaches through
preclinical development and into the clinic, and on establishing
best-practice care for neuromuscular patients worldwide. The
network has developed from its European roots to become a global
organization that brings together leading specialists, patient
groups and industry representatives to ensure preparedness for the
trials and therapies of the future while promoting best practice
today.
Further information about TREAT-NMD can be found here:
http://www.treat-nmd.eu/
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (Duchenne)
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 all muscles, including skeletal, diaphragm, and heart
muscles. Patients with Duchenne can lose the ability to walk as
early as age ten, followed by loss of the use of their arms.
Duchenne patients subsequently experience life-threatening lung
complications, requiring the need for ventilation support, and
heart complications in their late teens and twenties.
More information on the signs and symptoms of Duchenne can be
found at: www.duchenneandyou.com
About PTC Therapeutics, Inc.
PTC is a science-led, global biopharmaceutical company focused on
the discovery, development and commercialization of
clinically-differentiated medicines that provide benefits to
patients with rare disorders. Founded 20 years ago, PTC
Therapeutics has successfully launched two rare disorder
products and has a global commercial footprint. This success is the
foundation that drives investment in a robust pipeline of
transformative medicines and our mission to provide access to
best-in-class treatments for patients who have an unmet medical
need.
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:
This press release contains forward-looking statements within the
meaning of The Private Securities Litigation Reform Act of 1995.
All statements contained in this release, other than statements of
historic fact, are forward-looking statements, including statements
regarding: the future expectations, plans and prospects for PTC;
the timing and outcome of PTC's regulatory process, including with
respect to Translarna; PTC's expansion of
commercialization of Translarna; PTC's strategy, future
operations, future financial position, future revenues, projected
costs; or intended use of proceeds from its public offering of
common stock; and the objectives of management. Other
forward-looking statements may be identified by
the words "guidance", "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 those related to: the outcome of
pricing, coverage and reimbursement negotiations with third party
payors for Translarna; PTC's ability to maintain its
marketing authorization of Translarna for the treatment of nmDMD in
the European Economic Area (EEA), 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, which is a specific obligation to continued
marketing authorization in the EEA; PTC's ability to complete
any dystrophin study necessary in order to resolve the matters set
forth in the denial to the Complete Response letter it received
from the FDA in connection with its NDA for Translarna for the
treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD),
and PTC's ability to perform additional clinical trials,
non-clinical studies, and CMC assessments or analyses at
significant cost; the eligible patient base and commercial
potential of Translarna and PTC's other products and
product candidates; and the factors discussed in the "Risk
Factors" section of PTC's most recent Quarterly Report on Form 10-Q
and Annual Report on Form 10-K 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 any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
Translarna.
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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