Phase III Clinical Trial in Newly Diagnosed Multiple Myeloma Stopped Early Due to Highly Significant Efficacy Advantage of VELCADE for Injection Based Therapy Across All Endpoints
- Millennium on track to file sNDA in first quarter 2008 -
September 18, 2007: 07:01 AM EST
CAMBRIDGE, Mass., Sept. 18 -- Millennium Pharmaceuticals, Inc. today announced that the interim analysis results of the large, international Phase III VISTA(1) trial in patients with newly diagnosed multiple myeloma showed that the therapy of VELCADE, melphalan and prednisone (VMP) demonstrated a highly statistically significant improvement in all efficacy measures, including time-to-disease progression, complete remission rate, progression-free survival and overall survival, compared to melphalan and prednisone (MP) alone. Based on the recommendation of an independent data monitoring committee (IDMC), which conducted the planned interim, the control arm of the trial was stopped early to allow patients still being treated with MP to have VELCADE added to their therapy.
"These results position VELCADE based therapy as a new standard of care for newly diagnosed multiple myeloma patients," said Paul Richardson, M.D., Associate Professor of Medicine, Harvard Medical School; Clinical Director, Jerome Lipper Multiple Myeloma Center Dana-Farber Cancer Institute Boston; and a lead investigator of the VISTA trial. "The combination of VELCADE with melphalan and prednisone surpassed all efficacy endpoints, including time-to-disease progression, complete remission rate, progression-free survival and overall survival, and did so, much earlier than expected. We are excited at the possibility this new therapy could be available to our patients sooner."
In the first quarter of 2008, the Company plans to file a supplemental new drug application (sNDA) in the U.S. for use of VELCADE in patients with newly diagnosed multiple myeloma, based on the data from this trial, which was conducted under the special protocol assessment (SPA) process with the Food and Drug Administration (FDA). The Company also expects that these data will be presented at the December 2007 meeting of the American Society of Hematology (ASH) in Atlanta, Georgia.
"An approval in front-line multiple myeloma would double the number of patients eligible to receive the benefit of VELCADE," said Nancy Simonian, M.D., Chief Medical Officer, Millennium. "We are confident that VELCADE based therapies will become standard of care, since physicians typically use the most active agents in the front-line setting to improve long-term outcomes for patients."
The Phase III VISTA trial is being conducted by the Company and its co-development partner Johnson & Johnson Pharmaceutical Research & Development, L.L.C. (JJPRD). The trial randomized 682 patients with newly diagnosed multiple myeloma, ineligible for stem cell transplantation, to receive either VMP or MP, a recognized standard of care in this treatment setting. Patients were enrolled at 151 clinical trial sites in 22 countries. The primary endpoint of the trial is time-to-disease progression with secondary endpoints, including overall survival, progression-free survival, complete remission rate and safety. Side effects of the VELCADE based therapy were manageable and included those seen in previous VELCADE clinical trials. Professor Jesus San Miguel, M.D., Hematology Department Head, University Hospital of Salamanca, Spain, is the principal investigator.
Conference Call Announcement
In conjunction with this news release, Millennium will host a webcast today, Tuesday, September 18, 2007 at 8:00 A.M. ET. This webcast can be accessed by visiting the Investors section of the Company's website, http://www.millennium.com . Following the webcast, an archived version of the call will be available at the same address for 30 days.
About Multiple Myeloma
Multiple myeloma is the second most common hematologic malignancy and although the disease is predominantly a cancer of the elderly (the average age of onset is 65 to 70 years of age), recent statistics indicate both increasing incidence and younger age of onset. In the U.S., more than 50,000 individuals have MM and 20,000 new cases are diagnosed each year. Worldwide there are approximately 74,000 new cases and over 45,000 deaths annually.
VELCADE is being co-developed by Millennium Pharmaceuticals, Inc. and JJPRD. Millennium is responsible for commercialization of VELCADE in the U.S., Janssen-Cilag is responsible for commercialization in Europe and the rest of the world. Janssen Pharmaceutical K.K. is responsible for commercialization in Japan. For a limited period of time, Millennium and Ortho Biotech Inc. are co-promoting VELCADE in the U.S. VELCADE is approved in more than 80 countries worldwide.
In the U.S., VELCADE is indicated for the treatment of patients with multiple myeloma who have received at least one prior therapy. VELCADE is indicated for the treatment of patients with mantle cell lymphoma who have received at least one prior therapy. VELCADE is contraindicated in patients with hypersensitivity to bortezomib, boron, or mannitol. VELCADE should be administered under the supervision of a physician experienced in the use of antineoplastic therapy. In the European Union and many other countries worldwide, VELCADE is approved for patients with multiple myeloma after first relapse.
Risks associated with VELCADE therapy include new or worsening peripheral neuropathy, hypotension observed throughout therapy, cardiac and pulmonary disorders, gastrointestinal adverse events, thrombocytopenia, neutropenia and tumor lysis syndrome. Women of childbearing potential should avoid becoming pregnant while being treated with VELCADE. Cases of severe sensory and motor peripheral neuropathy have been reported. The long-term outcome of peripheral neuropathy has not been studied in mantle cell lymphoma. Acute development or exacerbation of congestive heart failure, and/or new onset of decreased left ventricular ejection fraction has been reported, including reports in patients with few or no risk factors for decreased left ventricular ejection fraction. There have been rare reports of acute diffuse infiltrative pulmonary disease of unknown etiology such as pneumonitis, interstitial pneumonia, lung infiltration and Acute Respiratory Distress Syndrome in patients receiving VELCADE. Some of these events have been fatal. A higher proportion of these events have been reported in Japan. There have been rare reports of RPLS in patients receiving VELCADE. RPLS is a rare, reversible, neurological disorder which can present with seizure, hypertension, headache, lethargy, confusion, blindness, and other visual and neurological disturbances. VELCADE is associated with thrombocytopenia and neutropenia. There have been reports of gastrointestinal and intracerebral hemorrhage in association with VELCADE. Transfusions may be considered. Complete blood counts (CBC) should be frequently monitored during treatment with VELCADE. Rare cases of acute liver failure have been reported in patients receiving multiple concomitant medications and with serious underlying medical conditions.
Safety Data of VELCADE as a single-agent: In 1163 patients in multiple myeloma and mantle cell lymphoma studies, the most commonly reported adverse events were asthenic conditions (64%), nausea (55%), diarrhea (52%), constipation (41%), peripheral neuropathy (39%), thrombocytopenia (36%), appetite decrease, including reports of anorexia (36%), pyrexia (34%), vomiting (33%) and anemia (29%). Twenty percent of patients reported at least one episode of grade 4 toxicity; the most common grade 4 toxicities were thrombocytopenia (5%) and neutropenia (3%). Fifty percent of patients reported serious adverse events. The most commonly reported serious adverse events were pneumonia (7%), pyrexia (6%), diarrhea (5%), vomiting (4%), and nausea, dehydration, dyspnea and thrombocytopenia (each 3%).
For more information about VELCADE clinical trials, patients and physicians can contact the Millennium Medical Product Information Department at 1-866-VELCADE (1-866-835-2233).
Millennium Pharmaceuticals, Inc., a leading biopharmaceutical company based in Cambridge, Mass., markets VELCADE, a novel cancer product, and has a robust clinical development pipeline of product candidates. Millennium's research, development and commercialization activities are focused in two therapeutic areas: oncology and inflammation. By applying its knowledge of the human genome, understanding of disease mechanisms and industrialized drug discovery platform, Millennium is developing an exciting pipeline of innovative product candidates. Millennium's website is http://www.millennium.com .
This press release contains "forward-looking statements," including statements about the Company's growth and development of products. Various important risks may cause the Company's actual results to differ materially from the results indicated by these forward-looking statements, including: adverse results in its drug discovery and clinical development programs; failure to obtain patent protection for its discoveries; commercial limitations imposed by patents owned or controlled by third parties; the Company's dependence upon strategic alliance partners to develop and commercialize products and services based on its work; difficulties or delays in obtaining regulatory approvals to market products and services resulting from its development efforts; product withdrawals; competitive factors; difficulties or delays in manufacturing the Company's products; government and third-party reimbursement rates; the commercial success of VELCADE and INTEGRILIN(R) (eptifibatide) Injection; achieving revenue consistent with internal forecasts; and the requirement for substantial funding to conduct research and development and to expand commercialization activities. For a further list and description of the risks and uncertainties the Company faces, see the reports it has filed with the Securities and Exchange Commission. The Company disclaims any intention or obligation to update or revise any forward- looking statements, whether as a result of new information, future events or otherwise.