NeoPharm Announces New Data On Novel Tumor-Targeting Agent in Terminal Brain Cancer.

Business Editors

LAKE FOREST, Ill.--(BUSINESS WIRE)--Nov. 19, 2001

First Clinical Studies in IL13-PE38 for Malignant Glioma 

Show Early Potential for Serving Unmet Medical Need

NeoPharm, Inc. (Nasdaq National Market: NEOL) announced preliminary Phase I/II safety and efficacy results for its novel tumor-targeting agent, IL13-PE38, in the treatment of malignant glioma, a fatal form of brain cancer for which there is no known cure. IL13-PE38 was recently granted orphan drug designation from the U.S. Food and Drug Administration (FDA). Three abstracts were presented at the Sixth Annual Meeting of the World Federation of Neuro-Oncology/ Society for Neuro-Oncology (SNO) in Washington, D.C. on November 16. They represent the first clinical data reported in a scientific forum on IL13-PE38, which NeoPharm has exclusively licensed from the National Cancer Institute and the U.S. FDA for worldwide development. These preliminary findings suggest promising potential for IL13-PE38 in recurrent malignant glioma, which is diagnosed in approximately 17,500 people in the U.S. annually and claims approximately the same number of lives each year.

The complete abstracts are available online at www.soc-neuro-onc.org. Presenting the IL13-PE38 abstracts at the SNO meeting were study investigators Raj Puri, MD, PhD, Senior Investigator and Chief, Laboratory of Molecular Tumor Biology, Division of Cellular and Gene Therapies, U.S. FDA/CBER; Jon Weingart, MD, Assistant Professor of Neurosurgery and Oncology, Department of Neurosurgery, The Johns Hopkins University; and Michael Prados, MD, Director, Neuro-Oncology Service, Department of Neurosurgical Surgery, University of California San Francisco.

Malignant glioma, which includes glioblastoma multiforme and anaplastic astrocytoma tumors, is among the most deadly, invasive and rapidly growing forms of brain cancer. Most people diagnosed with malignant glioma usually live for less than one year after diagnosis, and there are currently very limited treatment options to prevent the rapid recurrence of the cancer once the tumor is surgically removed from the brain. Based on radiographic scans of the brain, as well as on histopathologic changes of tumor cell death upon microscopic evaluation of brain tumor biopsies prior to surgery and brain tumor tissue removed at surgery, the Phase I/II safety and efficacy studies to date indicate that IL13-PE38 may have potential as a treatment for malignant glioma. Additional patients are being enrolled in these studies to validate these early findings.

"This early clinical research marks an important milestone in NeoPharm's quest to fulfill an unmet medical need for people diagnosed with a severe and life-threatening form of brain cancer that has eluded most current therapeutic options," said James Hussey, President and Chief Executive Officer of NeoPharm. "In leading the research to unlock the potential of IL13-PE38 in targeting malignant glioma, we're encouraged by these initial data and plan to accelerate the clinical development into Phase II trials next year."

The Role of IL13 Receptors

IL13-PE8 is being developed by NeoPharm under a Cooperative Research and Development Agreement (CRADA) between NeoPharm and the U.S. FDA. Research at CBER/FDA has shown that malignant glioma cells, as compared to normal brain cells, express IL13 receptors at a high density. IL13-PE38 is designed to detect and bind to IL13 receptors on the surface of malignant glioma cancer cells. It then selectively delivers a potent cytotoxic agent called PE38 to destroy tumor cells while sparing healthy surrounding cells. According to the discoverer of IL13 receptors on tumor cells and IL13-PE38, Raj Puri, MD, PhD, "We're hopeful from the preliminary findings that additional research will demonstrate that patients with malignant glioma can receive a highly targeted, effective treatment at a clinically desirable dose level that kills potent tumor cells without damaging healthy brain tissue."

Phase I/II Study Results

Preliminary Phase I/II study data presented at the SNO meeting indicate that IL13-PE38 may be effective, safe and effectively administered by positive-pressure microinfusion. This convection-enhanced delivery, which uses catheters inserted in the brain before and/or following surgical removal of the tumor, is designed to infuse IL13-PE38 directly to the tumor site and adjacent brain tissue to prevent recurrence of tumor cell growth. The most effective dosage level of IL13-PE38 is being determined in these studies.

Fourteen patients have thus far entered the studies, and enrollment continues. In one study conducted by the NABTT (New Approaches to Brain Tumor Therapy) CNS Consortium, which is funded by the National Cancer Institute, noticeable response to IL13-PE38 was observed in two of six patients. One patient, who was treated with IL13-PE38, subsequently underwent surgical removal of the tumor mass. A histopathologic evaluation following surgery found IL13-PE38 effective in destroying approximately 95 percent of the malignant tumor cells. In a second patient, following IL13-PE38 administration, radiographic scans of the brain revealed a significant reduction in the size of the tumor, and the patient did not require a scheduled, additional, IL13-PE38 infusion.

A second dose escalation study is being conducted to determine the histologically effective concentration of IL13-PE38 to establish evidence of its ability to kill tumors. Patients undergo an initial tumor biopsy, followed by a continuous infusion of IL13-PE38 in the tumor and surrounding tissue before surgical resection. Following tumor removal, patients receive an additional continuous infusion of IL13-PE38 in the brain adjacent to the tumor resection cavity to treat glioma invading adjacent tissue. In one of the patients treated to date with a larger dose of IL13-PE38 in this dose escalation study, a marked histopathologic response was noted with IL13-PE38 as demonstrated by a large percent of necrotic, or killed, tumor cells in the post-surgical tumor specimen, as compared with the pre-infusion tumor biopsy.

"Malignant glioma is a devastating condition that urgently requires new treatment options. These clinical data point to evidence that we're making an important step forward in our ability to offer these patients hope where previously little existed. Further studies will ultimately determine the effectiveness of IL13-PE38 in treating this malignancy," said a lead investigator, Michael Prados, MD.

NeoPharm, Inc., based in Lake Forest, IL, is a publicly traded biopharmaceutical company dedicated to the discovery and commercialization of new and innovative cancer drugs for therapeutic applications. The Company has a broad portfolio of compounds in various stages of development.

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements include, but are not limited to, any statements relating to the Company's drug development program and any other statements that are not historical facts. Such statements involve risks and uncertainties, including, but not limited to, those risks and uncertainties relating to difficulties or delays in development, testing, regulatory approval, production and marketing of the Companies' drug candidates, unexpected adverse side effects or inadequate therapeutic efficacy of the Companies' drug candidates that could slow or prevent products coming to market, the uncertainty of patent protection for the Company's intellectual property or trade secrets and other risks detailed from time to time in filings the Company makes with Securities and Exchange Commission including their annual reports of Form 10-K and their quarterly reports on Forms 10-Q. Such statements are based on management's current expectations, but actual results may differ materially due to various factors, including those risks and uncertainties mentioned or referred to in this press release.