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PTC Therapeutics Presents Encouraging Phase 1 Results of its Novel VEGF Inhibitor, PTC299, at 30th Annual San Antonio Breast Cancer Symposium
Date:12/17/2007

rietary GEMS technology. This is an important milestone for the company," commented Stuart W. Peltz, Ph.D., President and CEO of PTC Therapeutics, Inc. "PTC299 is an example of the utilization of the GEMS technology to identify innovative oral small molecules with the opportunity to change the paradigm of treatment in areas of continued unmet medical need such as oncology and infectious diseases."

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.

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 Department of Defense Clinical Translational Research Award

In September 2006, the company received a $2.2 million award from the Department of the Army to fund development of PTC299 as a potential treatment for breast cancer. The Department of Defense Clinical Translational Research Award sponsors innovative preclinical and clinical/translational research that may result in substantial improvements over current approaches to breast cancer chemopreventi
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SOURCE PTC Therapeutics, Inc.
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