The opportunity to redefine the treatment of major diseases is what drives everyone at Vertex. Our development portfolio represents the fruit of these efforts. Spend some time here and look around. Click on the pipeline chart to get information on our development programs and links to selected published articles. Go further and see how, just maybe, disease challenges may be overcome with innovation.

AVN-944 (VX-944)
(Cancer)

AVN-944 (VX-944) is an oral IMPDH inhibitor in development for the treatment of cancer. Preclinical studies showed that AVN-944 is a highly specific inhibitor of IMPDH, suppresses pools of GTP, and in cultured cells has a selective growth inhibition effect on cancer cells compared to normal cells. In February 2005, Vertex entered into a licensing agreement for AVN-944 with Avalon Pharmaceuticals Incorporated.

Clinical Studies:

• Avalon completed a single-dose, dose escalation study of AVN-944 in healthy volunteers in the U.K., which indicated that AVN had a pharmacokinetic profile consistent with oral dosing in the oncology setting.
• Avalon is conducting a Phase 1, repeat-dose, dose escalation trial in patients with advanced hematologic malignancies. Patients are dosed for 21 days on a 28-day cycle. A minimum of three patients are treated at each dose level. The study is divided into two arms, one for treatment of leukemia patients and the other for treatment of patients with lymphoma and myeloma.
• In July 2007, Avalon announced the initiation of a Phase 2 study of AVN-944 in patients with pancreatic cancer. The trial will be conducted in two parts (Part A and Part B), both using an open-label, non-controlled design. Eligible patients will include adult patients with advanced newly diagnosed pancreatic cancer. Part A is a dose- escalation study with a primary objective to determine the maximum tolerated dose or effective biologic dose of AVN-944 in combination with gemcitabine. Approximately 15-20 patients will be enrolled in Part A. Part B is to evaluate the efficacy and safety of the AVN-944/gemcitabine combination and approximately 110-120 patients will be enrolled. Patients will receive an initial treatment cycle of AVN-944 and gemcitabine and may receive additional cycles of treatment every 28 days at the discretion of the Investigator and with the agreement of the patient. Information on the trial is available at the National Institutes of Health clinical trial database and www.clinicaltrials.gov