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PTC Therapeutics Receives Department of Defense Clinical Translational Research Award

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

SOUTH PLAINFIELD, NJ UNITED STATES
   - $2.2 Million Grant Will Fund Preclinical and Clinical Development of
                         PTC299 in Breast Cancer -

    SOUTH PLAINFIELD, N.J., Sept. 26 /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 has received a
three-year, $2.2 million grant from the Department of Defense (DoD) to fund
preclinical and clinical development of the company's product candidate
PTC299 as a potential treatment for breast cancer.
    (Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
    PTC299 was discovered through PTC's proprietary Gene Expression
Modulation by Small-molecules (GEMS) technology. PTC299 is an
orally-administered small molecule designed to inhibit the production of
vascular endothelial growth factor, or VEGF. PTC299 functions by targeting
the post-transcriptional control processes that regulate VEGF formation.
PTC299 inhibits VEGF production through a mechanism that is distinct from
other VEGF inhibitors. VEGF plays a major role in the growth of most
tumors, therefore PTC299 may have applicability in many cancer types,
including breast cancer. PTC299 is currently in Phase 1 clinical trials in
healthy volunteers.
    "We are very pleased to receive this award from the DoD to assist us in
the Phase 1-2 development of PTC299 patients with breast cancer," stated
Langdon Miller, M.D., Chief Medical Officer, PTC Therapeutics, Inc. and
principal investigator for the grant. "The grant provides important
validation to PTC's innovative scientific approach of modulating post-
transcriptional control elements with a small molecule to treat human
diseases."
    PTC is collaborating with researchers at the New York University (NYU)
Medical Center in New York City. Dr. Robert Schneider, Director of
Translational Cancer Research, Co-Director of the Breast Cancer Research
Program and an Associate Director of the NYU Cancer Institute, who serves
as a co-principal investigator for the DoD award, commented, "This
collaboration combines the preclinical and clinical development expertise
of an experienced biopharmaceutical company and a DoD Breast Cancer Center
of Excellence. This funding will support translation of preclinical
pharmacological and biomarker data into the clinic while offering the
potential to address the high unmet medical need of patients with breast
cancer."
    About Department of Defense Clinical Translational Research Award
    The DoD Clinical Translational Research Award sponsors innovative
preclinical and clinical/translational research that may result in
substantial improvements over current approaches to breast cancer
chemoprevention and therapy. As part of the Congressionally Directed
Medical Research Programs, it administers funds for peer-reviewed research
toward specific diseases and supports research that positively affects the
health and well-being of Americans. For this grant (grant number
W81XWH-06-1-0629), the U.S. Army Medical Research Acquisition Activity, 820
Chandler Street, Fort Detrick, MD 21702-5014 is the awarding and
administering acquisitions office. The content of this press release does
not necessarily reflect the position or the policy of the Government, and
no official endorsement should be inferred.
    About NYU Medical Center Breast Cancer Research
    Located in the heart of New York City, NYU Medical Center is one of the
nation's premier centers of excellence in health care, biomedical research,
and medical education. The mission of the NYU Cancer Institute is to
decrease and eliminate cancer as a significant health problem throughout
New York, the nation, and the world, by developing and maintaining
excellent programs in patient care, research, education, and prevention.
    About GEMS
    Gene Expression Modulation by Small-molecules (GEMS) is PTC's novel and
proprietary screening technology for the identification of small-molecules
that modulate post-transcriptional control mechanisms. Compounds identified
through the GEMS technology modulate gene expression by targeting the post-
transcriptional control processes that act through the untranslated regions
(UTRs) of messenger RNA (mRNA) molecules. GEMS was the basis for the
discovery of PTC299 and is being used in multiple ongoing drug discovery
programs.
    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.


SOURCE PTC Therapeutics, Inc.




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Related links:
  • http://www.ptcbio.com
    Photo Notes:http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
    CONTACT:
    Jane Baj of PTC Therapeutics, Inc.,
    +1-908-222-7000, x167, jbaj@ptcbio.com; or Sheryl Seapy of Pure
    Communications, +1-949-608-0841, sheryl@purecommunicationsinc.com