- Data from Phase 1a single-dose study to be presented at EORTC-NCI-AACR
Conference -
SOUTH PLAINFIELD, N.J., Nov. 7 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery and development
of small-molecule drugs targeting post-transcriptional control mechanisms,
today announced the initiation of a Phase 1 multiple-dose study to evaluate
escalating dose levels of PTC299 in healthy volunteers. PTC299 is a novel,
orally bioavailable drug designed to modulate RNA-mediated protein
expression to inhibit the production of vascular endothelial growth factor
(VEGF) in tumors.
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919 )
PTC has recently completed a Phase 1a escalating, single-dose safety
and pharmacokinetic study of PTC299 in healthy volunteers. Preliminary
results confirmed that the drug is orally bioavailable, well tolerated, and
safely achieves the desired target plasma concentrations. Data from this
trial will be presented in Poster Number 51, entitled, "Phase 1 Single-dose
Safety, PK, and Food-effect Study of PTC299, a Novel VEGF Expression
Inhibitor for Treatment of Solid Tumors" at the 18th Annual EORTC-NCI-AACR
Symposium "Molecular Targets and Cancer Therapeutics." The conference will
take place from November 7th to the 10th in Prague, Czech Republic.
"The favorable safety profile of PTC299 in preclinical toxicology
testing has allowed us to perform these initial Phase 1 single-dose and
multiple-dose studies in healthy volunteers," noted Langdon Miller, M.D.,
Chief Medical Officer at PTC Therapeutics, Inc. "The safety and
pharmacokinetic data obtained from these trials should allow a prompt
transition to Phase 1-2 clinical trials in cancer patients. The advantage
of this approach is that we have already established PTC299 dose levels
that are likely to be well- tolerated and achieve plasma concentrations
associated with preclinical testing."
"We are very pleased with the continued progress of PTC299," commented
Stuart W. Peltz, Ph.D., President and CEO of PTC Therapeutics, Inc. "PTC299
is the second compound that entered clinical development from our post-
transcriptional control technologies that identify small molecules which
affect RNA biology. In particular, PTC299 is an important validation of our
GEMS technology. Given the very high unmet medical need in oncology, we are
committed to rapidly advancing clinical trials of PTC299."
PTC will also present two preclinical PTC299 posters at the conference:
Poster Number 52, entitled "Preclinical Development of PTC299: An Orally
Bioavailable Small-molecule Drug That Selectively Inhibits VEGF Protein
Production, Tumor Growth, and Microvessel Density" and Poster Number 88,
entitled "PTC299 Inhibits VEGF Expression Through Its 5´-UTR." All PTC
posters will be on display on Wednesday, November 8th from noon to 3pm.
More information regarding the conference can be found at
http://www.fecs.be/emc.asp?pageId=973.
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. Over
expression 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 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 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.
 back to top
Related links:
http://www.ptcbio.com
Photo Notes:
NewsCom: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
PRN Photo Desk, photodesk@prnewswire.com
CONTACT:
Jane Baj, PTC Therapeutics, Inc.,
+1-908-222-7000, ext. 167, jbaj@ptcbio.com; or Sheryl Seapy, of
Pure Communications for PTC Therapeutics, Inc., +1-949-608-0841,
sheryl@purecommunicationsinc.com
|
