Scientific Committee of European Medicines Agency (EMEA) Agrees in Principle to Switch From Superiority to Non-Inferiority
SEATTLE, June 7 /PRNewswire-FirstCall/ -- Cell Therapeutics, Inc. (CTI) (Nasdaq: CTIC; MTAX) announced today that the EMEA’s Scientific Advice Working Party (SAWP) agreed in principle that a switch from the superiority endpoints in the STELLAR trials to non-inferiority endpoints could be justified as a pathway to approval. Their decision was based on the five key elements in the EMEA Points to Consider and the data presented at a recent meeting.
In addition, a shift in the non-inferiority margin may also be justified if the safety profile of XYOTAX(TM) (paclitaxel poliglumex) is superior to comparators in terms of important (serious) side effects and/or quality of life or use of rescue medications. CTI plans to file a marketing authorization application (MAA) in Europe in the first half of 2007 for XYOTAX as a single agent for first-line treatment of non-small cell lung cancer (NSCLC) in PS2 (ECOG performance status 2) patients.
“We had a very productive meeting with the scientific advisors for the EMEA,” noted Scott C. Stromatt, M.D., CTI’s Executive Vice President of Clinical Development and Regulatory Affairs. “The SAWP encouraged us to focus on all the clinically relevant XYOTAX safety data in our application to justify these changes to the statistical analyses, as well as to address the sensitivity of our assay when demonstrating non-inferiority. They agreed in principle with our position that it is clinically important to determine whether a drug like XYOTAX is non-inferior if it provides a more favorable risk/benefit profile for patients. This is a positive outcome given their willingness to review our application based solely on existing STELLAR trial data.”
Lung cancer remains the biggest cancer killer in Europe. The incidence of lung cancer in Europe is more than 13 percent of all cancers, and in 2000 resulted in nearly 350,000 deaths.
As announced earlier this month, CTI and the U.S. Food and Drug Administration agreed on a new drug application (NDA) route for XYOTAX for women with lung cancer in which the FDA agreed to review an NDA submission based on interim results of the PIONEER trial with the results of the STELLAR 3 and 4 trials to support the filing. If the PIONEER trial meets its pre- specified interim endpoint, CTI plans to submit an NDA in the first half of 2007.
“This is a very exciting outcome for CTI and more importantly for patients with lung cancer. We now have a clear route for potential approval in both the United States and Europe with both regulatory agencies willing to accept the STELLAR trial results as supportive or, in the case of the EMEA, as pivotal. This could have a significant positive impact on both the time and cost for bringing this drug to market,” commented James A. Bianco, M.D., President and CEO of CTI.
About the STELLAR Trials
The STELLAR trials are among the largest randomized, phase III trials to date in either second-line non-small cell lung cancer (NSCLC) or first-line PS2 NSCLC patients. STELLAR 2 tested XYOTAX versus docetaxel for the potential second-line treatment of NSCLC patients. STELLAR 3 tested carboplatin in combination with either XYOTAX or paclitaxel for the potential first-line treatment of poor performance status (PS2) patients with NSCLC. STELLAR 4 tested XYOTAX versus either gemcitabine or vinorelbine for the potential first-line treatment of poor performance status (PS2) patients with NSCLC.
About XYOTAX
XYOTAX(TM) (paclitaxel poliglumex) is a 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, the chemotherapy is rendered inactive, potentially sparing normal tissue’s exposure to high levels of unbound, active chemotherapy and its associated toxicities. Blood vessels in tumor tissue, unlike blood vessels in normal tissue, are porous to molecules like polyglutamate. Based on preclinical studies, it appears that XYOTAX 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 the paclitaxel chemotherapy. Preclinical and clinical studies support that XYOTAX metabolism by lung cancer cells may be influenced by estrogen, which could lead to enhanced release of paclitaxel and efficacy in women with lung cancer compared to standard therapies.
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.cticseattle.com.
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. Specifically, the risks and uncertainties that could affect the development of XYOTAX(TM) include risks associated with preclinical and clinical developments in the biopharmaceutical industry in general and with XYOTAX in particular including, without limitation, the potential failure of XYOTAX to prove safe and effective for treatment of lung and ovarian cancers, the potential failure of the PIONEER trial to accrue patients in a timely manner or meet the requirements of the interim analysis, the potential for the EMEA to not accept or approve the MAA in a timely manner or at all, determinations by regulatory, patent and administrative governmental authorities, competitive factors, technological developments, costs of developing, producing and selling XYOTAX, and the risk factors listed or described from time to time in the Company’s filings with the Securities and Exchange Commission including, without limitation, the Company’s most recent filings on Forms 10-K, 8-K, and 10-Q. CTI is under no obligation to (and expressly disclaims any such obligation to) update or alter its forward-looking statements whether as a result of new information, future events, or otherwise.
Source: Cell Therapeutics, Inc.
