-Company to Initiate Phase1b/2 Clinical Trial in Advanced Breast Cancer in
2007-
CHICAGO and SOUTH PLAINFIELD, N.J., June 3 /PRNewswire/ - PTC
Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the
discovery and development of small-molecule drugs targeting
post-transcriptional control mechanisms, today presented data from its
Phase 1 single- and multiple-dose studies in healthy volunteers evaluating
PTC299, its novel VEGF inhibitor. Data from these two safety and
pharmacokinetic studies confirmed that PTC299 is well tolerated up to the
maximum tested dose and, in the multiple-dose study, safely achieves the
desired target plasma concentrations at all tested dose levels. PTC299 is a
novel, orally bioavailable investigational drug discovered by PTC which
selectively inhibits production of vascular endothelial growth factor
(VEGF) in tumors.
(LOGO: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
"We are very encouraged by the results of these clinical studies which
show that PTC299 is well tolerated at plasma concentrations associated with
antiangiogenic and antitumor activity in preclinical models," stated
Langdon Miller, M.D., Chief Medical Officer at PTC Therapeutics, Inc.
"Furthermore, these Phase 1 data appear consistent with our preclinical
results that suggest PTC299 selectively inhibits pathological
tumor-associated VEGF production while not affecting physiological VEGF
production. It is notable that we did not see adverse events such as
bleeding, hypertension, or proteinuria that have been associated with other
VEGF inhibitors. Given the very high unmet medical need in oncology, we are
committed to rapidly advancing clinical trials of PTC299 in patients with
cancer."
Data from these clinical trials were detailed in a poster entitled,
"Phase 1 studies assessing the safety, PK, and VEGF-modulating effects of
PTC299, a novel VEGF expression inhibitor" (Poster Number H9). The poster
was presented on June 3rd at the Developmental Therapeutics: Molecular
Therapeutics session of the 43rd Annual ASCO Meeting, being held from June
1-5, 2007, in Chicago, Illinois.
In the initial clinical study, 52 healthy volunteers received single
doses up to 3 mg/kg of PTC299. In the subsequent multiple-dose study, 32
healthy volunteers received doses through 1.2 mg/kg/dose two times per day
and 1.6 mg/kg/dose three times per day. Among these healthy volunteers,
PTC299 was well tolerated, with no serious, dose-limiting, or definitively
drug-related adverse events. All clinical adverse events were mild (Grade
1) or moderate (Grade 2) in severity and did not require any intervention.
Pharmacokinetic data indicated dose-proportional increases in plasma
exposures and the target trough plasma concentration was achieved and
maintained at all dose levels in the multiple-dose study.
"The continued progress of PTC299 provides further validation of our
novel GEMS technology which targets post-transcriptional control
processes," commented Stuart W. Peltz, Ph.D., President and CEO of PTC
Therapeutics, Inc. "PTC299 selectively inhibited tumor VEGF production and
tumor growth in preclinical studies. With a new mechanism that acts
upstream of available anti-angiogenic agents, PTC299 has demonstrated
activity alone and in combination with hormonal, chemotherapeutic, and
other antiangiogenic agents. These results position PTC299 as a potential
therapy for a broad range of solid tumors."
PTC plans to initiate a Phase 1b/2 clinical trial in patients with
advanced breast cancer during the second half of 2007. This trial will be
funded in part through a three-year, $2.2 million grant from the Department
of Defense (DoD), which was awarded to PTC last year to fund both the
preclinical and clinical development of PTC299 in 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 PTC299
PTC299 is a novel, orally administered small-molecule compound that
inhibits the production of the protein vascular endothelial growth factor,
or VEGF, in tumors. PTC discovered PTC299 through PTC's proprietary GEMS
(Gene Expression Modulation by Small-Molecules) technology by targeting the
post- transcriptional processes that regulate VEGF formation.
Overexpression of VEGF plays a key role in the growth of many types of
cancers. PTC299 inhibits VEGF production through a mechanism that is
distinct from other VEGF inhibitors and has potential application in the
treatment of multiple solid tumor types. In September 2006, PTC announced
that it had received a three- year, $2.2 million grant from the Department
of Defense (DoD) to fund preclinical and translational clinical development
of PTC299 as a potential treatment for breast cancer.
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 target 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's internally-discovered
pipeline addresses multiple therapeutic areas, including genetic disorders,
oncology and infectious diseases. In addition, PTC has developed
proprietary technologies and extensive knowledge of post-transcriptional
control processes that it applies in its drug discovery and development
activities, including the Gene Expression Modulation by Small-molecules
(GEMS) technology platform, which has been the basis for collaborations
with leading pharmaceutical and biotechnology companies such as Pfizer, CV
Therapeutics and Schering-Plough. For more information, please visit the
Company's website at http://www.ptcbio.com.
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
http://www.asco.org/ac/1,1003,_12-002092,00.asp/
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, x167, jbaj@ptcbio.com; Andrea Johnston of Pure
Communications, +1-910-616-5858, purecommunicationsinc.com
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