Recent medical advances have led to new potential treatments for metastatic breast cancer.

Researchers have come to understand that breast cancer tumor cells make high levels of a protein called vascular endothelial growth factor (VEGF). VEGF stimulates formation of new blood vessels that feed tumors and help them grow. Inhibiting VEGF may slow new blood vessel formation and may slow tumor growth. Drugs targeting new blood vessel formation in tumors are currently available to treat breast cancer and other types of cancer, such as those of the brain lung, colon and rectum, kidney, and liver.

PTC Therapeutics has developed an oral investigational drug called PTC299. PTC299 is designed to block tumor-produced VEGF and not the VEGF necessary to maintain the growth and function of normal blood vessels.

In animal studies, PTC299 has been shown to decrease tumor VEGF and slow or stop the growth of tumors or cause them to shrink. Short duration, early phase clinical trials in healthy adult volunteers and patients with cancer have evaluated the safety of PTC299. Results from these trials show that adverse events observed in people receiving PTC299 at high doses have been infrequent, mostly mild to moderate, and not usually considered to be caused by PTC299.

New Clinical Trial of Oral PTC299 in Women with Metastatic Breast Cancer

PTC Therapeutics is now conducting a PTC299 clinical trial in adult women with metastatic breast cancer.

This study is testing the safety and activity of PTC299 in combination with an aromatase inhibitor (AI) such as Arimidex® (anastrozole), Femara® (letrozole), or Aromasin® (exemestane). It is evaluating the effect of PTC299 on VEGF levels in the blood, blood flow to the tumors, and tumor growth through noninvasive blood and laboratory tests.

Participants in this study take PTC299 by mouth twice per day. Each patient receives the drug in 6-week treatment cycles. Cycles of treatment can continue as long as the therapy appears to be safely offering tumor control.

Study participants will be able to maintain an all-oral regimen. They will be able to continue with their current hormonal therapy in combination with PTC299 or receive PTC299 in combination with a new hormonal therapy during the course of the trial.

Requirements for Participating in This Study

• To be eligible for the trial, participants must:
• be female
• be at least 18 years old
• have a diagnosis of metastatic breast cancer (note: patients with stable brain metastases may be eligible)
• currently receiving or eligible to receive one of the three FDA-approved hormonal therapies, i.e., Arimidex, Femara, or Aromasin
• not have been previously treated with chemotherapy regimens for metastatic disease
• be willing and able to participate in study procedures

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.