PTC Therapeutics, Inc. Initiates Phase 1A Clinical Trial Of PTC299

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.

Photo: NewsCom: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGOAP Archive: http://photoarchive.ap.orgPRN Photo Desk, photodesk@prnewswire.comPTC Therapeutics, Inc.

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

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