Significant Difference in Primary Endpoint Could be Acceptable With Fewer
Patients Than Originally Planned
SEATTLE, Sept. 6 /PRNewswire-FirstCall/ -- Cell Therapeutics, Inc. (CTI) (Nasdaq: CTIC) (MTAX: CTIC) announced today that following discussions with the U.S. Food and Drug Administration (FDA) regarding its EXTEND (PIX301) trial for patients with aggressive non-Hodgkin's lymphoma (NHL) the Company has decided to conduct a full analysis of the trial instead of an interim analysis as previously planned. The decision was based in part on the Agency's guidance that a primary endpoint analysis of less than the initially-projected 320 eligible patients could be acceptable for New Drug Application (NDA) submission if it is able to demonstrate a statistically significant (p<=0.05) difference between treatment arms. In addition, the FDA agreed that randomized safety data from the RAPID (PIX203) study (CHOP-R vs. CPOP-R) could be used to support the EXTEND results in an NDA submission for pixantrone. Based on this guidance the Company currently anticipates completing enrollment in the EXTEND trial in the fourth quarter of this year with primary endpoint data and final results being reported in the first half of 2008. Pixantrone has received fast track status from the FDA for this indication.
"This was a very important outcome for CTI and the PIX301 trial timeline. If we achieve the primary endpoint of the study, even with fewer than 320 patients, we could submit our NDA" said James A. Bianco, M.D., President and CEO of CTI. "Going straight to a final analysis will allow us the potential to submit an NDA for pixantrone and receive approval in 2009. The inclusion of safety data from the randomized trial of pixantrone versus doxorubicin is also a very important recognition of the potential toxicity benefit of this novel agent."
The Company recently announced results of an interim analysis of the RAPID trial which showed that patients who received pixantrone had a reduction in severe (grade 3/4) side effects when compared to patients treated with standard doxorubicin-based therapy. Despite pixantrone patients receiving more treatment cycles, a three-fold reduction in the incidence of severe heart damage (LVEF decline >15 percent) was seen as well as clinically significant reductions in severe infections and febrile neutropenia.
The Data Safety Monitoring Committee, which met recently, recommended that the EXTEND study continue. The trial is expected to be completed by the end of 2007 with a full analysis during the first half of 2008. Since pixantrone has fast track status for relapsed, aggressive NHL, the drug could receive marketing approval in 2009.
About EXTEND (PIX301)
The EXTEND clinical trial is a phase III single agent trial of pixantrone for patients with relapsed, aggressive non-Hodgkin's lymphoma who have received two or more prior therapies and who are sensitive to treatment with anthracyclines. The trial is being conducted at 130 sites in 17 countries. Patients are randomized to receive either pixantrone or another single-agent drug currently used for the treatment of this patient population and selected by the physician. The trial is designed to examine the complete response (CR) or unconfirmed complete response (uCR) rate, time to tumor progression, and overall survival. The study was powered based on a CR rate assumption of less than 5 percent for the control arm and a 10 percent improvement in CR rate for the pixantrone arm. The study initially anticipated the requirement of 320 patients to meet the statistical assumptions of the trial. The study is being conducted under a Special Protocol Assessment from the U.S. Food and Drug Administration (FDA) and pixantrone has received fast track designation for this indication.
About RAPID (PIX203)
The RAPID trial is a first-line randomized phase II study of the CHOP-R versus CPOP-R in previously untreated aggressive NHL patients. The study is evaluating replacing doxorubicin in the standard CHOP-R combination regimen (cyclophosphamide, doxorubicin, vincristine, prednisone and rituximab) with pixantrone to part of the CPOP-R regimen (cyclophosphamide, pixantrone, vincristine, prednisone and rituximab). The objective of the study is to demonstrate non-inferior complete response rates as the standard doxorubicin-based therapy with significantly less severe cardiac toxicities and other doxorubicin-related toxicities on the CPOP-R arm of the study. 280 patients are expected to be enrolled.
Pixantrone is an investigational agent under development for the potential treatment of various hematological malignancies, solid tumors and immunological disorders. It was developed to improve the activity and safety of the anthracycline family of anti-cancer agents. Anthracyclines have been shown to be very active clinically in a number of tumor types. However, they are usually associated with cumulative heart damage that prevents them from being used in a large proportion of patients. Pixantrone has been designed to reduce the potential for these severe cardiotoxicities, as well as to potentially increase activity and simplify administration compared to the currently marketed anthracyclines.
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 http://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 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 treatment of non-Hodgkin's lymphoma, our ability and time needed to enroll, treat and evaluate eligible patients in our trials, the limitations inherent in having a smaller enrollment size, determinations by regulatory, patent and administrative governmental authorities, competitive factors, technological developments, costs of developing, producing and selling pixantrone, 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. Except as may be required by Italian law, 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.
Cell Therapeutics, Inc.
Cell Therapeutics, Inc.
|SOURCE Cell Therapeutics, Inc.|
Copyright©2007 PR Newswire.
All rights reserved