Data Published in Muscle and Nerve Validates the Six-Minute Walk Test as an Outcome Measure in Duchenne Muscular Dystrophy
SOUTH PLAINFIELD, N.J., Dec. 8 /PRNewswire/ -- Data published
online in the medical journal Muscle and Nerve support the use of
the six-minute walk test (6MWT) as an outcome measure in PTC
Therapeutics, Inc.'s ongoing registration-directed clinical trial
of ataluren in patients with nonsense mutation Duchenne/Becker
muscular dystrophy (nmDMD/BMD). The study results, which will also
be published in an upcoming print issue of the journal,
characterize the limitations on walking by patients with DMD
relative to healthy boys and indicate that young boys can
consistently and reliably perform the test. The data were obtained
from an observational study conducted at the University of
California Davis which was sponsored by PTC and supported by a
grant from Parent Project Muscular Dystrophy (PPMD).
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
)
The 6MWT is an accepted and standardized measure of muscle,
lung, and heart health in patients with other medical conditions.
However, the test had not previously been used to evaluate boys
with DMD. The observational study was designed to confirm that boys
with DMD would have the stamina and focus required to successfully
and consistently complete the test without injury.
"Advancing the validation of clinically meaningful endpoints for
therapeutic and natural history studies in DMD has been a focus of
the Duchenne clinical and advocacy communities for many years,"
said Craig McDonald, M.D., principal investigator of the
observational study and director of the Rehabilitation Research and
Training Center in Neuromuscular Diseases, University of California
Davis. "The publication of these findings demonstrates that the
6MWT can serve as a clinically meaningful outcome measure for
documenting the evolution of the disease and the treatment benefits
of new therapies for DMD. The new test has rapidly become the
favored primary outcome measure worldwide in clinical trials in
ambulatory boys with Duchenne muscular dystrophy. This is an
exciting moment for all of us involved in the care of boys with
this progressively disabling condition."
The study was designed to evaluate the feasibility, safety and
reproducibility of the 6MWT, comparing boys with DMD to healthy
boys of the same age. The study evaluated 21 ambulatory boys with
DMD and 34 healthy boys, ages four to 12 years old. Boys with DMD
were tested twice, one week apart and healthy boys were tested
once. This study demonstrated that ambulatory young boys can
complete the 6MWT safely and consistently.
"PPMD is proud to have sponsored research that sets the
foundation for developing drugs in DMD. The validation of the 6MWT
as a clinical endpoint has significant implications for the DMD
community," stated Patricia Furlong, Founding President & CEO
of Parent Project Muscular Dystrophy. "The ability to study the
efficacy of potential DMD treatments will facilitate the
advancement of new therapies and ultimately benefit patients. An
important part of what we do at PPMD is to help establish universal
standards for boys and young men with DMD. It is particularly
gratifying to have a validated clinical endpoint to expand the
development of therapeutic options."
The study found that boys with DMD walked profoundly shorter
distances in six minutes than healthy boys due to the muscular
deficits that are characteristic of DMD, including markedly reduced
stride length. The findings also suggest that the 6MWT is highly
reproducible, as evidenced by a high degree of correlation (r=0.91)
between the first and second test in boys with DMD. These findings
directly support the use of the 6MWT in PTC's ongoing Phase 2b,
double-blind, randomized trial of ataluren in boys with nmDMD/BMD,
for which the 6MWT is the primary outcome measure. The trial is
designed to determine whether ataluren taken orally can improve
walking, muscle function and strength in patients with nmDMD/BMD
and will also evaluate ataluren's long-term safety profile.
"We are encouraged that pretreatment data from 174 boys
participating in our pivotal Phase 2b study of ataluren are
consistent with the observational data and show that the 6MWT is
safe and reproducible when performed repeatedly at multiple trial
sites in many different countries," said Langdon Miller, M.D.,
Chief Medical Officer of PTC. "We look forward to sharing the data
from the ataluren clinical trial in nmDMD/BMD patients in early
2010."
About DMD/BMD
Duchenne and Becker muscular dystrophy (DMD/BMD) are progressive
muscle disorders that result in the loss of both muscle function
and independence. Patients with DMD/BMD have increasing problems
with walking as the disease progresses and eventually must depend
upon assistive devices to maintain mobility. DMD/BMD is perhaps the
most prevalent form of the muscular dystrophies and is the most
common lethal genetic disorder diagnosed during childhood today.
Each year, approximately 20,000 children worldwide are born with
DMD (one of every 3,500 male children). It is estimated that one in
ten DMD patients is likely to have a Becker presentation, a milder
form of the disease that is associated with later manifestation of
symptoms. Further information regarding DMD and BMD is available
through the Muscular Dystrophy Association (www.mdausa.org) and
Parent Project Muscular Dystrophy (www.parentprojectmd.org).
About Ataluren (PTC124®)
Ataluren is the first investigational new drug designed to
restore the formation of a functioning protein in patients with
genetic disorders due to a nonsense mutation. A nonsense mutation
is an alteration in the genetic code that prematurely halts the
synthesis of an essential protein. Ataluren is currently being
investigated for use in patients with nmDMD/BMD, nonsense mutation
cystic fibrosis (nmCF) and nonsense mutation hemophilia A and B
(nmHA/HB).
Ataluren has been granted orphan drug status for the treatment
of nmCF and nmDMD/BMD by the U.S. Food and Drug Administration
(FDA) and the European Commission. The FDA has also granted
ataluren Subpart E designation for expedited development,
evaluation, and marketing. The development of ataluren has been
supported by the Cystic Fibrosis Foundation Therapeutics Inc. (the
nonprofit affiliate of the Cystic Fibrosis Foundation), the FDA
Office of Orphan Products Development, the Muscular Dystrophy
Association, Parent Project Muscular Dystrophy, and the National
Center for Research Resources.
Collaboration With Genzyme
PTC Therapeutics has an exclusive collaboration with Genzyme
Corporation for the development and commercialization of ataluren.
PTC will commercialize ataluren in the United States and Canada,
while Genzyme will commercialize the product in other regions of
the world.
About PTC Therapeutics
PTC is a biopharmaceutical company focused on the discovery,
development and commercialization of orally administered,
proprietary, small-molecule drugs that target post-transcriptional
control processes. Post-transcriptional control processes regulate
the rate and timing of protein production and are of central
importance to proper cellular function. PTC's internally discovered
pipeline addresses multiple therapeutic areas, including genetic
disorders, oncology, and infectious diseases. PTC has developed
proprietary technologies that it applies in its drug discovery
activities and that are the basis for collaborations with leading
biopharmaceutical companies such as Celgene, Genzyme, Gilead,
Merck, Pfizer and Roche. For more information, visit the company's
website at www.ptcbio.com.
Photo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
Source: PTC Therapeutics, Inc.
CONTACT: Investors and Media: Jane Baj, PTC Therapeutics,
Inc.,
+1-908-912-9167, jbaj@ptcbio.com; Sheryl Seapy, Pure
Communications,
+1-949-608-0841, sheryl@purecommunicationsinc.com
Web Site: http://www.ptcbio.com/
Posted: December 2009
