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PTC Therapeutics Initiates Phase 1a Clinical Trial of PTC299

   PTC Therapeutics logo. (PRNewsFoto/PTC Therapeutics, Inc.)

SOUTH PLAINFIELD, NJ UNITED STATES
    SOUTH PLAINFIELD, N.J., May 12 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery and development
of orally administered, proprietary small-molecule drugs that target
post-transcriptional control processes, today announced that it had
commenced a Phase 1a clinical trial of the company's product candidate
PTC299 in healthy volunteers. The trial is being conducted in Belgium.
PTC299 was discovered through PTC's proprietary Gene Expression Modulation
by Small-molecules (GEMS) technology.
    (Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
    This Phase 1a trial is a single-site, randomized, double-blind,
placebo-controlled escalating single-dose safety and pharmacokinetic study
in healthy volunteers between the ages of 18 and 55. The primary objective
of this trial is to determine a dose range for PTC299 that is well
tolerated, achieves pharmacologically active plasma concentrations and is
appropriate for use in a subsequent Phase 1b multiple-dose study. PTC
expects to complete this trial in the second quarter of 2006. If this trial
is successful, PTC plans to initiate further clinical trials of PTC299 in
solid tumor cancer patients.
    About PTC Therapeutics, Inc.
    PTC is a biopharmaceutical company focused on the discovery and
development 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 has assembled
proprietary technologies and extensive knowledge of post-transcriptional
control processes that it applies in its drug discovery and development
activities. PTC's current pipeline of clinical and preclinical product
candidates addresses multiple indications, including genetic disorders,
oncology, and infectious diseases.
    About PTC299
    PTC299 is a novel, orally administered small-molecule compound designed
to inhibit the production of the protein vascular endothelial growth
factor, or VEGF, in tumors. PTC has designed PTC299 to inhibit VEGF
production in tumors by targeting the post-transcriptional control
processes that regulate VEGF formation. Because PTC299 inhibits VEGF
production, its action occurs at a different point in the VEGF pathway than
other therapies that target the binding of VEGF to its receptors on the
surface of blood vessel cells.


SOURCE PTC Therapeutics, Inc.




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Related links:
  • http://www.ptcbio.com
    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:
    Jane Baj of PTC Therapeutics, Inc.,
    +1-908-222-7000 ext 167, jbaj@ptcbio.com, or Sheryl Seapy of Pure
    Communications, +1-949-608-0841, sheryl@purecommunicationsinc.com