Independent Radiology Assessment Panel Results Confirm Statistical Significance of Response and Progression Endpoints of Cell Therapeutics' Pixantrone PIX301 Pivotal Trial
-Analysis of data paves way for NDA resubmission in October 2011
SEATTLE, Sept. 28, 2011 /PRNewswire/ -- Cell Therapeutics, Inc. ("CTI") (NASDAQ and MTA: CTIC) today announced that a second independent radiology assessment of response and progression endpoint data from CTI's PIX301 clinical trial of pixantrone confirmed the statistical robustness of the PIX301 efficacy data that was previously submitted by CTI to the U.S. Food and Drug Administration ("FDA") in CTI's new drug application ("NDA") for pixantrone.
This second independent assessment was recommended by the FDA's Office of New Drugs ("OND") as part of CTI's appeal of the Complete Response Letter ("CRL") for the NDA, which CTI received from the FDA's Office of Oncology Drug Products.
"The review conducted by a second independent panel of three radiologists confirmed the statistically and clinically significant results the first independent panel reported for the PIX301 study response and progression endpoints," noted James A. Bianco, M.D., CEO of CTI. "We now have two separate independent response assessment panels reporting significant results for complete response/complete response unconfirmed, overall response rate and progression-free survival at both end of treatment and end of study assessment periods as well as among the histologically confirmed aggressive non-Hodgkin's lymphoma ("NHL") subset of patients. We look forward to sharing these confirmatory results with the Division of Oncology Drug Products ("DODP") in our resubmission of the pixantrone NDA."
The review and the review process was conducted based on the recommendations by OND in order to determine that efficacy had been established by confirming confidence in the endpoint ascertainment and reliability of the primary results.
Based on the results of this second independent assessment panel's review, CTI plans on resubmitting the pixantrone NDA as early as October 2011 for accelerated approval. The DODP previously confirmed that the review of the NDA would be completed within six months from the date of resubmission, which should provide CTI an FDA action date by April 2012.
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 in an effort 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 diffuse large lymphoma without unacceptable rates of cardiotoxicity.
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 market 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 for the treatment of relapsed or refractory NHL and/or other tumors as determined by the FDA and/or the European Medicines Agency, that accelerated approval by the FDA of pixantrone may not be possible or occur, that CTI may not be able to address satisfactorily the two key matters raised by the OND or other matters raised by the DODP, the OND and/or the FDA, that CTI's interpretation of the guidance provided by the OND, the DODP and/or the FDA may be different than the intent of the OND, the DODP and/or the FDA, that the OND, the DODP and/or the FDA may change its guidance, that the PIX301 study may not be deemed successful, that upon a re-review and/or resubmission of the NDA the FDA may find pixantrone to not be safe and/or effective, that the PIX301 study may still be deemed to be a failed study, that the FDA may require an additional clinical trial of pixantrone, that if CTI conducts an additional clinical trial, it may not demonstrate the safety and effectiveness of pixantrone, that CTI may not be able to provide satisfactory information in response to the FDA's Complete Response Letter, that CTI may not be able to re-submit the pixantrone NDA for accelerated approval as early as October 2011, that even if CTI resubmits the NDA for accelerated approval in October 2011 the FDA may not complete the review of the NDA within six months of resubmission, that CTI may not obtain an FDA action date by April 2012, that CTI may not obtain approval of the NDA from the FDA in the first half of 2012, 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.
Lindsey Jesch Logan
Medical Information Contact:
Posted: September 2011