NeoPharm, Inc. announced that an agreement with the US Food and Drug Administration (FDA) has been reached under the Special Protocol Assessment (SPA) process for the company's Precise trial. Precise is designed to be a pivotal Phase III study of NeoPharm's experimental tumour-targeting, anti-cancer drug IL13-PE38QQR, currently being investigated as a treatment for first recurrence of glioblastoma multiforme (GBM).

GBM is a deadly form of brain cancer for which there is presently few treatment options. The SPA process allows for a written agreement between NeoPharm and the FDA on the design of a study, including clinical drug supply, pivotal trial design, clinical endpoints, study conduct, data analysis, and other clinical trial issues, and is intended to provide assurance that if pre-specified trial results are achieved, they may serve as the primary basis for an efficacy claim in support of a Biologics Licensing Application (BLA) by the company for IL13-PE38QQR.

Precise, which stands for Phase III Randomized Evaluation of Convection Enhanced Delivery of IL13-PE38QQR with survival endpoint, is a randomized controlled Phase III clinical trial. It is designed to compare overall survival, drug safety and quality of life of patients receiving IL13-PE38QQR with patients receiving Gliadel Wafer in the treatment of first recurrent GBM following surgical tumour resection (removal). As one of the largest international Phase III trials of its kind, Precise will seek to enroll 300 patients in multiple centers across North America, Europe and Israel. Patients will be randomized so that 200 patients receive IL13-PE38QQR via Convection Enhanced Delivery (CED) using catheters placed following the resection, and 100 patients receive Gliadel Wafer placed at the time of resection. The company currently anticipates enrollment of the first patients in the Precise trial within the next four to six weeks.

"This is a very exciting time for NeoPharm. Precise will be one of the largest and most comprehensive clinical trials conducted to date in recurrent GBM," said James Hussey, NeoPharm's CEO. "We are hopeful that data we collect from Precise will support the findings from our previous Phase I/II studies of IL13-PE38QQR in recurrent GBM. The progression of this compound into Phase III further reinforces our commitment to finding more effective treatments for primary brain tumours-bringing new hope to patients and their families suffering from this devastating disease," said Hussey.

IL13-PE38QQR has received orphan drug designation in the US and Europe, and fast track drug development programme status from the FDA. NeoPharm has exclusively licensed IL13-PE38QQR from the National Cancer Institute and the FDA, and is developing the agent under a Cooperative Research and Development Agreement (CRADA) in collaboration with the laboratory of Raj Puri, MD at the FDA Center for Biologics Evaluation and Research (CBER).