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PTC Therapeutics Receives $2.6 Million in Government Grants
Date:6/15/2009

ered small-molecule investigational new drug that selectively blocks the pathological, or disease-related, production of the protein vascular endothelial growth factor (VEGF) in tumors, while sparing physiological VEGF expression.

The grant awarded by the FDA Office of Orphan Products Development provides additional funding towards an ongoing pivotal, placebo-controlled study that is designed to determine whether ataluren can improve walking, muscle function and strength in patients with nmDMD/BMD. The study is also evaluating ataluren's long-term safety profile. The pivotal trial is being conducted at 37 sites across Australia, Canada, Europe, Israel and the United States. PTC announced in February that the study successfully completed enrollment two months ahead of schedule.

ABOUT PTC299

PTC299 is a novel, orally administered small-molecule investigational new drug 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 multiple tumor types.

In Phase 1 clinical studies, PTC299 has been generally well tolerated by healthy volunteers and patients with cancer. Adverse events have been mild or moderate in severity and have not usually been considered drug-related. In addition, adverse events commonly seen with other VEGF blockers, such as bleeding, hypertension and proteinuria, have not been observed. Clinical trials for PTC299 are also being conducted in patients with other solid tumors, metastatic breast cancer, and Kaposi sarcoma.

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SOURCE PTC Therapeutics, Inc.
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