Cancer can be a disabling and life-threatening disease for patients with advanced solid tumors or metastatic disease. In the past decade, the development of chemotherapeutics agents and targeted drug therapies has demonstrated that cancer treatment can be improved by offering more efficacious, less toxic, and more convenient options to patients. However, there is still an urgent need for continuing efforts to develop safer and more effective therapies.

Tumors make high levels of a substance called vascular endothelial growth factor (VEGF). VEGF stimulates formation of blood vessels that feed tumors and help them grow. Blocking VEGF may slow blood vessel formation and may slow tumor growth.

PTC Therapeutics, Inc. has developed an orally administered investigational drug called PTC299. PTC299 is designed to block VEGF production by tumors. In studies using animal models of human cancer, PTC299 has been shown to decrease tumor VEGF and slow or halt tumor growth.

Phase 1a clinical trials in healthy adult volunteers have evaluated the safety of PTC299 when given by mouth for up to 7 days. Results from these short duration, early phase clinical trials indicate that PTC299 is generally well tolerated at dose levels higher than those currently being investigated in patients with cancer.

Working together with doctors at Memorial Sloan-Kettering Cancer Center in New York City, PTC Therapeutics is now conducting a PTC299 Phase 1b clinical trial in patients with metastatic cancer. The study is designed in 3 stages. In Stage 1, up to 18 patients will receive increasingly higher doses of PTC299 in a repeated on-off pattern (4 weeks on drug and 2 weeks off drug) to assess the drug’s safety. In Stage 2, a second group of up to 12 patients will receive PTC299 every day. In Stage 3, a third group of up to 12 patients will receive PTC299 together with the chemotherapy medication, docetaxel (Taxotere®).

Important Requirements for Taking Part in This Study

• be willing and able to participate in study procedures
• be willing to abstain from sexual intercourse or employ an effective barrier or medical method of contraception during the study
• be willing to undergo two tumor biopsies

Other eligibility requirements will be explained at the trial site.

Additional details about this PTC299 clinical trial can be found at www.clinicaltrials.gov or by contacting Patient and Professional Advocacy at (866) 282-5873 (toll free in the United States), (908) 912-9256 or PatientInfo@ptcbio.com.