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Phase III Trial of Anti-Cancer Drug IL13-PE38QQR To Continue After FDA Protocol Review

LAKE FOREST, Ill.--(BUSINESS WIRE)--Feb. 5, 2004--NeoPharm, Inc. (Nasdaq/NM: NEOL) today announced that an agreement with the U.S. 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 tumor-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 are 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(R) Wafer in the treatment of first recurrent GBM following surgical tumor 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(R) 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.

For additional details regarding the PRECISE trial design, enrollment criteria and participating centers please visit www.clinicaltrials.gov (keyword: IL13-PE38QQR).

"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 M. Hussey, NeoPharm's President and Chief Executive Officer. "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 tumors-bringing new hope to patients and their families suffering from this devastating disease," said Hussey.

IL13-PE38QQR has received orphan drug designation in the U.S. and Europe, and fast track drug development program 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 K. Puri, MD, PhD, at the FDA Center for Biologics Evaluation and Research (CBER).

About Special Protocol Assessment
The Prescription Drug User Fee Act of 1992 (PDUFA) goals for Special Protocol Assessment and agreement provide that, upon request, FDA will evaluate within 45 days certain protocols, and issues relating to the protocols, to assess whether they are adequate to meet scientific and regulatory requirements identified by the sponsor. Three types of protocols related to PDUFA products are eligible for this protocol assessment under the PDUFA goals: (1) animal carcinogenicity protocols, (2) final product stability protocols, and (3) clinical protocols for Phase III trials whose data will form the primary basis for an efficacy claim if the trials had been the subject of discussion at an end-of-Phase II/pre-Phase III meeting with the review division, or in some cases, if the division agrees to such a review because the division is aware of the developmental context in which the protocols for Phase III trials can relate to efficacy claims that will be part of an original new drug application (NDA) or BLA or that will be part of an efficacy supplement to an approved NDA or BLA. For more information on Special Protocol Assessment, go to http://www.fda.gov.

About Glioblastoma Multiforme
Malignant glioma, which includes glioblastoma multiforme, anaplastic astrocytoma, and malignant mixed oligoastrocytoma tumors, is a highly malignant, rapidly infiltrating, primary brain tumor that penetrates surrounding brain tissue. GBM is the most common and aggressive form of malignant glioma. GBM, like most malignant gliomas, are known to recur in a location close to the resection cavity left by the removal of the tumor. As there are very limited treatment options to prevent rapid recurrence of the tumor once the tumor is surgically resected, most people usually survive for less than one year after initial diagnosis.

About IL13-PE38QQR
The key to making a useful tumor-targeting drug is the use of a targeting mechanism that is able to select and destroy tumor cells while bypassing healthy normal cells. IL13-PE38QQR is a recombinant protein consisting of a single molecule composed of two parts: a tumor-targeting molecule (IL13) and a cytotoxic agent (Pseudomonas exotoxin, or PE). In studies conducted by the Company, the cancer cell appears to latch onto and absorb the IL13, as well as the attached PE, which results in the death of the cancer cell. Healthy brain cells do not appear to express IL13 receptors, and thus, are not believed to internalize the PE.

About Convection Enhanced Delivery
Convection enhanced delivery (CED) involves the placement of one or more catheters into a brain tumor or nearby brain tissue to deliver a slow, continuous infusion of a drug or other substance over several days, directed against the tumor. This technique allows relatively large volumes of brain tissue to be treated. CED is designed to infuse IL13-PE38QQR directly to the tumor resection site and adjacent brain tissue to prevent recurrence of tumor cell growth.

About GLIADEL(R) Wafers
A GLIADEL(R) Wafer delivers BCNU (or carmustine), a commonly used chemotherapeutic anti-tumor agent for GBM, directly to the brain tumor resection cavity. Up to eight dime-sized wafers are placed in the space once occupied by the tumor at the time of resection. The wafers slowly dissolve over the next two to three weeks, bathing the surrounding cells with BCNU. GLIADEL(R) Wafer has been approved by the FDA for the treatment of recurrent and newly diagnosed GBM.

Source: NeoPharm

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