Favrille, Inc. Announces Data Cutoff for Phase 3 Registration Trial of Specifid; Data Analysis and Unblinding Expected in June
Median follow-up for ongoing patients is 31 months from randomization (range 21 to 41 months), or approximately 34 months from the initiation of Rituxan treatment (range 24 to 44 months). Based on the protocol's assumptions, this range of follow-up would provide sufficient power to detect a significant difference between the two arms for time to progression (TTP), the primary endpoint in the trial.
"We believe the design and execution of this registration trial have positioned us well for success," said John P. Longenecker, Ph.D., President and Chief Executive Officer of Favrille. "Based on the status of patients at data cutoff and our assumptions about the behavior of the control arm in this trial, we are very encouraged that the upcoming analysis will provide an outcome which is both clinically and statistically significant. Our clinical and biometrics team is working diligently to gather the required information and prepare for the analysis and unblinding of the trial in June."
Favrille initiated its Phase 3 randomized, placebo-controlled, double-blind registration trial of Specifid for follicular B-cell NHL in July 2004. The Company completed enrollment in January 2006 with 349 patients randomized into the trial in a span of 18 months. The trial was open to both treatment-naive and previously treated patients, ultimately enrolling a much larger treatment-naive population (78%). Patients were randomized at a one-to-one ratio to receive either Specifid or placebo following a standard course of Rituxan. A total of 45 patients came off study and were permanently censored for reasons other than disease progression, 28 (8%) because Specifid was not produced and 17 (5%) for a variety of other reasons. The trial was conducted at 67 centers in the U.S. under a Special Protocol Assessment granted by the U.S. Food and Drug Administration (FDA). Specifid was also granted Fast Track status by the FDA in January 2006.
In October 2007 an independent Data Monitoring Board (DMB) conducted an administrative analysis of the unblinded control arm in the Phase 3 trial. The objective of this analysis, conducted in agreement with the FDA, was to assess the behavior of disease progression in the control arm in order to help determine the duration of follow-up needed prior to unblinding the trial. The outcome of this DMB analysis supported the rationale for the timing of data cutoff.
About Specifid
Specifid (mitumprotimut-T, formerly FavId) is a personalized, active immunotherapy designed to stimulate a patient's immune system to mount a specific and sustained response to the patient's cancer. Specifid is based upon a recombinant protein, called idiotype (Id), which is derived from genetic material obtained from a patient's own tumor, then conjugated to keyhole limpet hemocyanin (KLH), a protein commonly used to boost immune responses. Favrille's unique and proprietary manufacturing process, which includes an insect cell (baculovirus) expression system, allows for the manufacture of Specifid for delivery to patients in as few as eight weeks. Data presented at the American Society of Hematology Annual Meeting in December 2007 showed that Id-KLH produced via insect cells results in a more immunogenic response compared to Id-KLH produced in a traditional mammalian cell manufacturing process. Also, unlike other Id-KLH active immunotherapies in development, Specifid is intended for use as a chronic therapy and is administered until the patient's disease progresses.
About Favrille, Inc.
Favrille, Inc. is a biopharmaceutical company focused on the development and commercialization of targeted immunotherapies for the treatment of cancer and other diseases of the immune system. The Company's lead product candidate, Specifid (mitumprotimut-T, formerly FavId), is a patient-specific, active immunotherapy based upon unique genetic information extracted from a patient's tumor. Specifid is currently under investigation in a Phase 3 registration trial for patients with follicular B-cell NHL and Phase 2 clinical trials in other B-cell NHL indications. The Company is developing additional applications based on its immunotherapy expertise and proprietary cost-effective manufacturing technology, including a second product candidate, FAV-201, for the treatment of cutaneous T-cell lymphoma.
The preliminary, blinded clinical data reported from time to time prior to the release of the final results of Favrille's Phase 3 registration trial have not been audited and have been taken from databases that have not been reconciled against medical records kept at the clinical sites or that may not include the most current information on patient disease progressions. The data released may not be indicative of the final results of Favrille's Phase 3 registration trial Failure can occur at any stage of testing. The Company does not know whether its Phase 3 registration trial or any future clinical trials will demonstrate safety and efficacy sufficient to result in marketable products. Favrille's failure to adequately demonstrate the safety and efficacy of Specifid will prevent the Company from obtaining regulatory approval for, or commercializing, Specifid.
Statements in this press release that are not strictly historical in nature constitute "forward-looking statements." Such statements include, but are not limited to, references to Favrille's product candidates, proprietary technologies and research and clinical development programs. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Favrille's actual results to be materially different from historical results or from any results expressed or implied by such forward-looking statements. These factors include, but are not limited to, risks and uncertainties related to progress and timing of clinical trials for Specifid, including difficulties or delays in development, testing, manufacturing and marketing Specifid or Favrille's other product candidates; delays in the availability of data from Favrille's Phase 3 clinical trial; Favrille's ability to obtain marketing approval for Specifid or Favrille's other product candidates and the timing of any such approvals, including whether it will receive expedited review as a result of the Fast Track designation; Favrille's ability to manufacture sufficient quantities of Specifid for use in clinical trials and, if Specifid receives marketing approval, for commercialization; risks associated with achieving projected operating metrics and financial performance or the anticipated number of patients using Specifid; Favrille's ability to obtain additional financing to support its operations; and additional risks discussed in Favrille's filings with the Securities and Exchange Commission. In addition, conclusions regarding the safety and efficacy of Favrille's product candidates cannot be made until the results of future clinical trials of longer duration in more patients are known. All forward-looking statements are qualified in their entirety by this cautionary statement. Favrille is providing this information as of the date of this release and, except as required by law, does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.
Source: Favrille, Inc.