Cell Therapeutics, Inc. ("CTI") (Nasdaq and MTA: CTIC) announced that it has received notice that the European Medicines Agency ("EMEA") has validated the expanded Pediatric Investigation Plan ("PIP") that CTI filed in July for pixantrone for the treatment of relapsed or refractory, aggressive non-Hodgkin's lymphoma (NHL). Following the validation, the EMEA Pediatric Committee (the "PDCO") will review and comment or approve the content of pediatric plan. Once the PIP is approved, CTI will submit the Marketing Authorization Application ("MAA") for pixantrone in the E.U. later this year. The pediatric program will study pixantrone in pediatric patients aged 6 months to 18 years with the goal of determining the comparative safety and effectiveness of pixantrone compared to doxorubicin in pediatric lymphoid cancers.
"We are very pleased that the EMEA has validated the expanded PIP for pixantrone, as there is a real need for a less toxic, more effective anthracycline-like treatment option not only in lymphoma, but also possibly in other tumors," said Jack Singer, Chief Medical Officer of CTI. "We look forward to completing the MAA submission, and continuing to pursue our goal of providing pixantrone as a treatment option to the patients who need it most."
CTI submitted the updated, expanded PIP in July 2010 after the PDCO recommended CTI expand the original PIP of September 2009 to include pixantrone's potential, but unproven, clinical benefit to children in reducing long-term cardiotoxicity associated with current curative therapies. The recommendation from the PDCO came following discussions with CTI about the preclinical and clinical pixantrone data, including PIX301, and the desire to explore the potential benefits pixantrone may offer to children with hematologic cancer.
About Pixantrone
Pixantrone is a novel aza-anthracenedione that has distinct structural and physio-chemical properties that make its anti-tumor activity unique in this class of agents. Similar to anthracyclines, pixantrone inhibits Topo-isomerase II but unlike anthracyclines--rather than intercalation with DNA--pixantrone alkylates DNA--forming stable DNA adducts, with particular specificity for CpG rich, hyper-methylated sites. These structural differences resulted in significantly enhanced anti-lymphoma activity compared to doxorubicin in preclinical models. In addition, the structural motifs on anthracycline-like agents that are responsible for the generation of oxygen free radicals and the formation of toxic drug-metal complexes have also been modified in pixantrone to prevent the binding of iron and perpetuation of superoxide production--both of which are the putative mechanism for anthracycline induced acute cardiotoxicity. These novel pharmacologic differences may allow re-introduction of anthracycline like potency in the treatment of relapsed/refractory aggressive lymphoma without unacceptable rates of cardiotoxicity.
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 pixantrone include risks associated with preclinical and clinical developments in the biopharmaceutical industry in general, and with pixantrone in particular, including, without limitation, the potential failure of pixantrone to prove safe and effective and/or less toxic and effective for the treatment of relapsed or refractory, aggressive NHL and/or other tumors as determined by the EMEA, that the EMEA may not accept the PIP, that CTI may not file the MAA later this year, that CTI's MAA may not be approved by the EMEA by next year, that the current plans for the pediatric program may change, that the pediatric program may not determine the comparative safety and effectiveness of pixantrone compared to doxorubicin in pediatric lymphoid cancers, and CTI's ability to continue to raise capital as needed to fund its operations, competitive factors, technological developments, costs of developing, producing and selling pixantrone, and the risk factors listed or described from time to time in CTI's filings with the 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