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 glioma /gli·o·ma/ (gli-o´mah) a tumor composed of neuroglia in any of its states of development; sometimes extended to include all intrinsic neoplasms of the brain and spinal cord, as astrocytomas, ependymomas, etc.
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 orphan drug, drug developed under the U.S. Orphan Drug Act (1983) to treat a disease that affects fewer than 200,000 people in the United States. The orphan drug law offers tax breaks and a seven-year monopoly on drug sales to induce companies to undertake the designation from the U.S. Food and Drug Administration (FDA FDA
Food and Drug Administration
n.pr See Food and Drug Administration.
n.pr the abbreviation for the Food and Drug Administration. ). Three abstracts were presented at the Sixth Annual Meeting of the World Federation of Neuro-Oncology/ Society for Neuro-Oncology (SNO SNO Sudbury Neutrino Observatory
SNO Second Network Operator
SNO Sakon Nakhon, Thailand (Airport Code)
SNO Società dei Neurologi, Neurochirurghi e Neuroradiologi Ospedalieri (Italy) ) 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 neurosurgery /neu·ro·sur·gery/ (noor´o-sur?jer-e) surgery of the nervous system.
Surgery on any part of the nervous system. and Oncology, Department of Neurosurgery, The Johns Hopkins University Johns Hopkins University, mainly at Baltimore, Md. Johns Hopkins in 1867 had a group of his associates incorporated as the trustees of a university and a hospital, endowing each with $3.5 million. Daniel C. ; and Michael Prados, MD, Director, Neuro-Oncology Service, Department of Neurosurgical Surgery, University of California The University of California has a combined student body of more than 191,000 students, over 1,340,000 living alumni, and a combined systemwide and campus endowment of just over $7.3 billion (8th largest in the United States). San Francisco.
Malignant glioma, which includes glioblastoma multiforme glioblastoma mul·ti·for·me
A virulent brain cancer that is usually fatal. and anaplastic an·a·plas·tic
1. Relating to the surgical restoration of a lost or absent part.
2. Of, relating to, or characterized by cells that have become less differentiated.
1. astrocytoma astrocytoma /as·tro·cy·to·ma/ (as?tro-si-to´mah) a tumor composed of astrocytes; the most common type of primary brain tumor and also found throughout the central nervous system, classified on the basis of histology or in order of 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 radiographic (rā´dēōgraf´ik),
adj relating to the process of radiography, the finished product, or its use. scans of the brain, as well as on histopathologic changes of tumor cell death upon microscopic evaluation of brain tumor Brain Tumor Definition
A brain tumor is an abnormal growth of tissue in the brain. Unlike other tumors, brain tumors spread by local extension and rarely metastasize (spread) outside the brain. 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” redirects here. For other uses, see CRADA (disambiguation).
A Cooperative Research and Development Agreement (CRADA) is an agreement between a government agency and a private company to work together. (CRADA CRADA Cooperative Research And Development Agreement ) 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 NABTT New Approaches to Brain Tumor Therapy (New Approaches to Brain Tumor Therapy) CNS See Continuous net settlement.
See continuous net settlement (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 malignant tumor
A tumor that invades surrounding tissues, is usually capable of producing metastases, may recur after attempted removal, and is likely to cause death unless adequately treated. 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 Tumor Removal Definition
Tumor removal is a surgical procedure to remove an abnormal growth.
A tumor can be either benign, like a wart, or malignant, in which case it is a cancer. , 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 dev·as·tate
tr.v. dev·as·tat·ed, dev·as·tat·ing, dev·as·tates
1. To lay waste; destroy.
2. To overwhelm; confound; stun: was devastated by the rude remark. 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.