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.
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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.
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CONTACT: Jane Baj, PTC Therapeutics, Inc., +1-908-222-7000, ext. 167,jbaj@ptcbio.com; or Sheryl Seapy, of Pure Communications for PTCTherapeutics, Inc., +1-949-608-0841, sheryl@purecommunicationsinc.com
Web site: http://www.ptcbio.com/
