SOUTH PLAINFIELD, N.J.,
July 9, 2018 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ:PTCT) today announced the presentation
of data from the Translarna (ataluren) Phase II Study 030
demonstrating that the safety and pharmacokinetic profile of
Translarna in children from two to five years with nonsense
mutation Duchenne muscular dystrophy (nmDMD) was consistent with
that for older children.1 Importantly, the
data also showed that treatment with Translarna resulted in
improvements in timed function tests and the North Star Ambulatory
Assessment from baseline at weeks 28 and 52, with mean changes
showing as much as a 25 percent improvement after one
year.1 The data at 28 weeks formed the basis of the
recent positive opinion from the Committee for Medicinal Products
for Human Use (CHMP) of the European Medicines Agency (EMA) to
expand the current indication of Translarna to include nmDMD
ambulatory children from two to five years of
age.2 The data was presented at the
International Congress on Neuromuscular Diseases in Vienna.
Translarna is the only approved treatment to address the
underlying cause of nmDMD, a rare, genetic, muscle-wasting
disease,1 and is currently licensed in Europe for ambulatory patients aged five years
and older.3
"We are excited to demonstrate that Translarna showed an
improvement over one year of treatment in patients with nonsense
mutation Duchenne as young as two years of age," stated
Stuart W. Peltz, Ph.D., Chief
Executive Officer of PTC Therapeutics, Inc. "Irreversible muscle
damage starts before the age of five. Early intervention is
critical to maintain muscle function and delay disease
progression."
An interim analysis of Study 030 demonstrated that at week 28,
the safety and pharmacokinetic profile for Translarna in children
aged two to five years is consistent with that for older
children.1 Clinical benefits were also observed at 28
weeks with Translarna, with decreases versus baseline in the time
to run/walk 10 meters, climb four stairs, and stand from lying face
up (supine).1 The most common adverse events included
pyrexia, ear infection, and nasopharyngitis.1
Study 030 evaluated changes in timed function tests (TFTs) and
the 3-part, 8-part and full (16) items North Star Ambulatory
Assessment (NSAA) scales, adopted for children under five years of
age (N=12).1 Results summarized in the table below.
TFTs
|
Week
28
|
Week
52
|
|
Baseline
(s)
|
Time to
complete
(s) at week
28
|
Mean %
change
|
Baseline
(s)
|
Time to
complete
(s) at week
52
|
Mean %
change
|
Time to
descend 4
stairs
|
7.1
|
6.5
|
7.2%
|
7.5
|
5.3
|
24.2%
|
Time to
ascend 4
stairs
|
7.1
|
5.3
|
9.1%
|
7.5
|
4.5
|
23.3%
|
Rise from
floor
|
7.3
|
4.3
|
17.4%
|
7.1
|
4.1
|
19.6%
|
10 m
walk/run
|
6.7
|
5.9
|
8.4%
|
6.6
|
6.2
|
8.6%
|
NSAA
|
Week
28
|
Week
52
|
Item
|
Baseline
(s)
|
Score at week 28
|
Mean %
change
|
Baseline
(s)
|
Score at
week 52
|
Mean %
change
|
16
|
16.2
|
19.8
|
24.9%
|
16.0
|
21.5
|
36.6%
|
8
|
10.5
|
12.1
|
15.1%
|
10.5
|
12.8
|
23.3%
|
3
|
5.4
|
5.8
|
10.3%
|
5.4
|
5.6
|
6.3%
|
About Study 0301
Study 030 was an open-label, Phase 2 study designed to evaluate
the safety and pharmacokinetics (PK) of ataluren (10, 10, and 20
mg/kg) in patients aged ≥2 to <5 years with nmDMD. The study
includes a 4-week treatment period, a 48-week extension period, and
a 4-week follow-up period. Secondary objectives in Study 030
evaluated changes in timed function tests (TFTs) and the total,
3-part,8-part and full (16) items North Star Ambulatory Assessment
(NSAA) scales, adapted for children <5 years of age. All
patients were male (N=14) with genotypic confirmation of nmDMD. Two
patients were excluded from the current analysis: one patient did
not have reported functional assessment at Week 28; and one patient
did not have baseline measurement all for post-line evaluations,
resulting in N=12. Seven out of the fourteen patients in the safety
population (50%) reported ≥1 treatment-emergent adverse event
(TEAE) during the extension phase, all of which were deemed
unrelated to the study drug; there were no serious TEAEs or
discontinuations due to a TEAE. Pyrexia, ear infection, and
nasopharyngitis were the most common TEAEs, each occurring in 2
patients (14.3%).
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 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 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 the final determination by the European
Commission with respect to expanding the use of Translarna to
include children aged two to five years with nmDMD and with respect
to renewal of the marketing authorization in the European Economic
Area (EEA) for Translarna for the treatment of nmDMD; the clinical
utility and potential advantages of Translarna; PTC's ability to
continue to supply Translarna to patients across Europe and in other territories; 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: 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; the outcome of
pricing, coverage and reimbursement negotiations with third party
payors for Translarna; 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, Emflaza and PTC's other 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 or Emflaza.
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.
References
1 Tian C, et al. Ataluren
in patients aged ≥ 2 to < 5 years with nonsense mutation
Duchenne muscular dystrophy (nmDMD): 28-week results from a Phase 2
study. Poster presented at the International Congress on
Neuromuscular Diseases, July 6-10,
2018, Vienna, Austria.
Poster 807
2 PTC press release. CHMP Adopts Positive Opinion for
the Expansion of the Translarna™ (ataluren) Label to
Include Patients as Young as 2 Years of Age. Available at:
http://ir.ptcbio.com/news-releases/news-release-details/chmp-adopts-positive-opinion-expansion-translarnatm-ataluren.
Last accessed: June 2018.)
3 Translarna, Summary of Product Characteristics
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SOURCE PTC Therapeutics, Inc.