SOUTH PLAINFIELD, N.J., July 7 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery, development, and
commercialization of small-molecule drugs targeting post-transcriptional
control mechanisms, today announced that PTC124 has been granted orphan drug
status for the treatment of Duchenne muscular dystrophy (DMD) and cystic
fibrosis (CF) by the Committee for Orphan Medicinal Products (COMP) of the
European Medicines Agency (EMEA). PTC124 is a novel, orally administered drug
that targets nonsense mutations and is being investigated initially as a
treatment for CF and DMD, with the potential to treat a number of other
genetic disorders. It is estimated that 10% of CF patients and 15% of DMD
patients have these diseases as a consequence of nonsense mutations.
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
Orphan drug designation is granted by the EMEA to promote the development
of products that may offer therapeutic benefits for diseases affecting less
than five in 10,000 people in the European Union (EU). The EMEA's orphan
medicinal product designations are based on several criteria that include the
rarity and seriousness of the condition, and the availability of other
effective therapies. Orphan drug designation provides opportunities for free
protocol assistance, fee reductions for access to the centralized community
procedures before and after marketing authorization, and 10 years of market
exclusivity following drug approval. The EMEA represents 25 EU countries,
including France, Germany, Italy, Spain, and the United Kingdom.
Results from Phase 1 studies have confirmed that PTC124 is orally
bioavailable, generally well tolerated, and achieves target plasma
concentrations that have been associated with activity in preclinical models.
Pending concurrence from regulatory authorities, PTC expects to advance PTC124
into Phase 2 studies in patients with nonsense-mutation-mediated CF and DMD
during the third quarter of 2005 in the US. PTC is working with patient
advocacy groups and other organizations to develop studies of PTC124 in other
regions of the world.
"Receiving orphan drug designation for PTC124 for the treatment of CF and
DMD from the EMEA is extremely gratifying. This designation is a significant
step towards the development of PTC124 in Europe," said Stuart Peltz, Ph.D.,
President and CEO of PTC Therapeutics.
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery, development,
and commercialization of small-molecule drugs targeting post-transcriptional
control mechanisms. Post-transcriptional control processes are the sequence
of events in the cell that ultimately regulate the rate and timing of all
protein production. PTC's compounds alter these processes by selectively
modulating how RNA is used to produce proteins. By applying this approach,
PTC has advanced its drug discovery programs rapidly from targets to
preclinical and clinical drug candidates, building a robust pipeline across
genetic disorders, oncology, and infectious diseases.
About PTC124
PTC124 represents a first-in-class, orally delivered investigational new
drug for the treatment of genetic disorders due to nonsense mutations.
Nonsense mutations are single-point alterations in the genetic code that
prematurely halt the translation process, producing a shortened,
non-functional protein. PTC124 allows the cellular machinery to bypass the
nonsense mutation and continue the translation process, restoring the
production of full-length, functional proteins. PTC124 has demonstrated the
ability to restore full-length functional protein in preclinical genetic
disease models harboring nonsense mutations. Phase 1 clinical studies confirm
that PTC124 is orally bioavailable, generally well tolerated, achieves target
plasma concentrations that have been associated with activity in preclinical
models, and does not induce ribosomal readthrough of normal stop codons.
Pharmacokinetic modeling of the Phase 1 results has allowed development of a
dosing regimen for the planned Phase 2 studies in cystic fibrosis (CF) and
Duchenne muscular dystrophy (DMD). It is estimated that 10% of the cases of
CF and 15% of the cases of DMD are due to nonsense mutations. PTC has
catalogued over 1,800 distinct rare disorders where nonsense mutations are the
cause of the disease in an appreciable percentage of patients. In addition to
CF and DMD, other potential indications include hemophilia, neurofibromatosis,
retinitis pigmentosa, epidermolysis bullosa, and lysosomal storage disorders.
PTC124 represents a unique opportunity to use a single small-molecule drug to
address chronic and life-threatening diseases of high unmet medical need.
Pending concurrence from regulatory authorities, Phase 2 studies in patients
with CF and DMD are planned for the third quarter of 2005. PTC is working
with patient advocacy and other organizations to develop studies of PTC124 in
the US and in other regions of the world.
The FDA has granted PTC124 fast track designation for the treatment of CF
and orphan drug designations for the treatment of CF and DMD due to nonsense
mutations.
About Duchenne Muscular Dystrophy (DMD)
DMD is a progressive muscle disorder that causes the loss of both muscle
function and independence. DMD is perhaps the most prevalent 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). More information regarding DMD
is available through the Muscular Dystrophy Association
(http://www.mdausa.org), and the Parent Project Muscular Dystrophy
(http://www.parentprojectmd.org). The Muscular Dystrophy Association (MDA)
has awarded PTC a grant of $1.5 million for the development of PTC124 for the
treatment of Duchenne muscular dystrophy (DMD) due to a nonsense mutation.
The Parent Project Muscular Dystrophy (PPMD) has awarded PTC a grant of $1
million for drug discovery efforts on other targets believed to be of
therapeutic relevance for DMD.
About Cystic Fibrosis (CF)
CF is a life-threatening, genetic disease affecting approximately 60,000
people worldwide. A defective gene causes the body to produce abnormally
thick, sticky mucus that leads to chronic lung-infections and impairs
digestion. More information regarding CF is available through the Cystic
Fibrosis Foundation (http://www.cff.org). The Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT) has awarded PTC $1.7 million for the development of
PTC124 for the treatment of CF due to a nonsense mutation in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene.
Other Rare Disorders:
Information on other rare disorders can be obtained through NORD: North
American Organization for Rare Disorders (http://www.rarediseases.org) or
Eurordis: European Organization for Rare Disorders (http://www.eurordis.org).
European Organizations Currently Involved in Planning for PTC124's
Development in Europe:
- Cystic Fibrosis: Vaincre La Mucoviscidose (French Cystic Fibrosis
Association) http://www.vaincrelamuco.org
- Duchenne Muscular Dystrophy: AFM (French Association against Muscular
Disorders) http://www.afm-france.org
For more information:
Patients, Patients' Families, Investigators and Patient Organizations
Kerri Donnelly
PTC Therapeutics, Inc.
(908) 222-7000, x112
kdonnelly@ptcbio.com
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
http://www.mdausa.org http://www.parentprojectmd.org
http://www.cff.org http://www.rarediseases.org
http://www.eurordis.org http://www.vaincrelamuco.org
http://www.afm-france.org
Photo Notes:
NewsCom:
http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
AP Archive: http://photoarchive.ap.org
PRN Photo Desk, photodesk@prnewswire.com
CONTACT:
Investors & Media - Jane Baj,
+1-908-222-7000, x167, jbaj@ptcbio.com, or Robert Stanislaro of
Noonan Russo, +1-212-845-4268, robert.stanislaro@eurorscg.com;
Patients, Patients' Families, Investigators and Patient
Organizations, Kerri Donnelly, PTC Therapeutics, Inc.,
kdonnelly@ptcbio.com, +1-908-222-7000, x112
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