Cell Therapeutics OPAXIOTM Receives Orphan Drug Designation for Malignant Brain Cancer from FDA
SEATTLE, Oct. 2, 2012 /PRNewswire/ — Cell Therapeutics, Inc. (“CTI”) (NASDAQ and MTA: CTIC) announced that OPAXIO (paclitaxel poliglumex) has been granted orphan-drug designation by the U.S Food and Drug Administration (“FDA”) for the treatment of glioblastoma multiforme (“GBM”), a malignant brain cancer.
Orphan designation was granted based on preliminary activity seen from phase 2 results of OPAXIO when added to standard therapy (temozolamide (“TMZ”) plus radiation). In this study, progression-free and overall survival was encouraging among patients with GBM, including patients whose tumors expressed unmethylated MGMT. Current standard therapy is less effective in patients with tumors that have unmethylated MGMT, an important DNA repair enzyme. A randomized trial is now underway for patients with GBM with unmethylated MGMT comparing standard TMZ and radiation to OPAXIO and radiation.
According to the National Cancer Institute, GBM is the most common and deadliest type of primary brain tumor in adults. It is estimated that there will be 10,000-12,000 new cases of GBM diagnosed in the US this year alone. The standard of care for patients with GBM is a surgical resection, if possible, followed by radiation given with concurrent TMZ. The prognosis for the majority of patients with GBM is poor with less than 25% of patients surviving two years with current therapies. Survival is shorter for patients whose tumors have active (unmethylated) MGMT.
Orphan-drug designation is granted by the FDA to novel drugs that seek to treat a rare disease or condition. Orphan-drug designation provides substantial potential benefits to the drug developer, including seven years of market exclusivity for the product upon regulatory approval, fee waivers and tax incentives.
Under the leadership of Dr. Howard Safran at Brown University Medical Center, a multicenter Phase 2 study (BrUOG 244) has been initiated comparing the efficacy of OPAXIO plus radiation with that of TMZ plus radiation in newly-diagnosed patients with GBM and unmethylated MGMT. In approximately 55% of patients with GBM, MGMT is unmethylated, thereby decreasing the efficacy of standard therapy with TMZ plus radiation therapy (“RT”). The randomized study seeks to determine whether OPAXIO plus radiation will improve progression free survival and overall survival when compared to TMZ plus radiation, the current treatment standard in this disease.
“The current randomized trial is based on the encouraging results previously demonstrated with OPAXIO and radiation in patients with newly diagnosed malignant brain cancer and specifically targets GBM patients with a genomic marker, unmethylated MGMT, who are less likely to benefit from the current standard of care TMZ and radiation,” stated Howard Safran, M.D., Medical Director of the Brown University Oncology Group. “We are pleased OPAXIO has been granted orphan-drug designation as patients with this disease have a serious unmet medical need for improved long-term survival particularly when MGMT is unmethylated.”
About the Study
The study is expected to enroll up to 120 patients. Patients in the OPAXIO arm will receive OPAXIO once every week plus RT for six weeks. Patients in the TMZ arm will receive daily oral TMZ plus RT for six weeks. After completion of initial therapy, both arms will receive maintenance TMZ on day 1-5 and then every 28 days for up to 12 cycles for a total of 48 weeks. Participating institutions include Rhode Island Hospital of Brown University, Memorial Hospital of University of Massachusetts, Maine Medical Center, Upstate Medical Center of the State University of New York (Syracuse), the University of Washington, the University of California at San Diego and Penn State Hershey Medical Center.
OPAXIO(TM) (paclitaxel poliglumex, CT-2103), is an investigational, biologically enhanced, chemotherapeutic that links paclitaxel, the active ingredient in Taxol®, to a biodegradable polyglutamate polymer, which results in a new chemical entity. When bound to the polymer, paclitaxel is inactive, potentially sparing normal tissue’s exposure to high levels of paclitaxel and its associated toxicities. Blood vessels in tumor tissue, unlike blood vessels in normal tissue, are porous to macromolecules such as OPAXIO. Based on preclinical studies, it appears that OPAXIO is preferentially distributed to tumors due to their leaky blood vessels and trapped in the tumor bed, allowing significantly more of the dose of chemotherapy to localize in the tumor than with standard paclitaxel. Once inside the tumor cell, enzymes metabolize the protein polymer, releasing active paclitaxel. Unlike standard radiosensitizing agents, OPAXIO appears tumor selective and does not appear to enhance radiation toxicity to normal tissues.
About OPAXIO(TM) as a radiosensitizer
OPAXIO is a highly potent radiosensitizer that selectively enhances the anti-tumor potency of radiation therapy in preclinical animal models and has demonstrated excellent tumor sensitization during clinical trials of locally advanced lower esophageal cancer. In addition to the study in patients with GBM, is also being tested as a radiosensitizer in patients with locally advanced head and neck cancer in combination with cetuximab.
About Cell Therapeutics, Inc.
Headquartered in Seattle, CTI is a biopharmaceutical company committed to developing an integrated portfolio of oncology products aimed at making cancer more treatable. For additional information, please visit www.CellTherapeutics.com.
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This press release includes forward-looking statements that involve a number of risks and uncertainties, the outcome of which could materially and/or adversely affect actual future results and the trading price of CTI’s securities. Specifically, the risks and uncertainties that could affect the development of OPAXIO include risks associated with preclinical and clinical developments in the biopharmaceutical industry in general, and with OPAXIO in particular, including, without limitation, the potential failure of OPAXIO to receive regulatory approval from the FDA, the potential failure of OPAXIO to prove safe and effective and/or less toxic and effective for the treatment of newly diagnosed high-grade malignant brain tumors such as GBM, including when combined with TMZ and RT, and/or the treatment of locally advanced lower esophageal cancer, the potential failure of OPAXIO when combined with TMZ and/or RT to provide progression free survival and overall survival responses to newly-diagnosed high-grade malignant brain tumors such as GBM, that the results of the randomized OPAXIO study may not be positive or be used to plan a phase III study of OPAXIO for approval for use in treating newly diagnosed GBM, that CTI cannot predict or guarantee the pace of patient enrollment in the clinical trial comparing OPAXIO to TMZ, that CTI cannot guarantee or predict whether or not orphan-drug designation of OPAXIO will provide a substantial benefit to CTI, the CTI cannot guarantee that even if OPAXIO is approved by the FDA that CTI will receive seven years of market exclusivity for OPAXIO, fee waivers and/or tax incentives, CTI’s ability to continue to raise capital as needed to fund this study and its operations, competitive factors, technological developments, costs of developing, producing and selling CTI’s product candidates such as OPAXIO, and the risk factors listed or described from time to time in CTI’s filings with the U.S. Securities and Exchange Commission including, without limitation, CTI’s most recent filings on Forms 10-K, 10-Q and 8-K. Except as may be required by law, CTI does not intend to update or alter its forward-looking statements whether as a result of new information, future events, or otherwise
SOURCE Cell Therapeutics, Inc.