ORLANDO, Fla., Dec. 12 /PRNewswire-FirstCall/ -- Pharmion Corporation today announced new data presented from two studies of thalidomide in combination therapy, including updated results from the pivotal MM-003 study in first-line multiple myeloma, and results from a study examining the effect of the addition of thalidomide to a post-transplant regimen. The results of these studies were presented at the 48th Annual Meeting and Exposition of the American Society of Hematology (ASH) in Orlando, December 9-12, 2006).
"These data continue to validate the use of thalidomide in a first-line setting," said Patrick J. Mahaffy, Pharmion's president and chief executive officer. "We look forward to submitting these data as a component of our filing with the EMEA in the first quarter of 2007 for thalidomide in the treatment of newly-diagnosed myeloma."
Statistically Significant Response Rates and Time to Progression Demonstrated in Randomized Trial of Thalidomide Plus Dexamethasone Versus Dexamethasone Alone for Newly Diagnosed Multiple Myeloma (Abstract 795, Oral Presentation)
S. Vincent Rajkumar, MD, of the Mayo Clinic in Rochester, Minnesota, delivered an oral presentation describing the final results of a randomized, double-blind placebo-controlled Phase 3 study evaluating the time to progression in newly-diagnosed multiple myeloma. The study compares the efficacy and safety of thalidomide plus dexamethasone versus placebo plus dexamethasone.
In the study, 470 patients were enrolled and randomized to the two study arms. Based on blinded adjudication of responses using the International Myeloma Working Group criteria, overall response was 69 percent in the thalidomide/dexamethasone arm, including eight percent complete responders, 36 percent very good partial responders, and 25 percent partial responders. This compares to 51 percent response on the placebo/dexamethasone arm, which includes three percent complete responders, 13 percent very good partial responders, and 35 percent partial responders. The p-value was 0.001.
Median time to progression was significantly higher in the thalidomide/dexamethasone arm, 22.4 months compared to 6.5 months in the placebo/dexamethasone arm. Median overall survival has not yet been reached in the thalidomide/dexamethasone arm, compared to 32 months in the placebo/dexamethasone arm.
The most common adverse events were constipation, peripheral edema and peripheral neuropathy. The most frequent thalidomide related severe toxicity was thromboembolism.
Data from this study suggest that treatment with thalidomide and dexamethasone is superior to placebo plus dexamethasone, with a significantly longer time to progression, with significantly higher response rates. These data will support the marketing authorization application for thalidomide in the treatment of first-line multiple myeloma that Pharmion intends to file in Europe during the first quarter of 2007.
Data Suggest that the Addition of Thalidomide to Standard Maintenance Therapy Following Stem Cell Transplant Prolongs Duration of Response (Abstract 58, Oral Presentation)
Andrew Spencer, MD, from the Alfred Hospital in Melbourne, Australia, delivered an oral presentation describing the first analysis of a study examining the effect of the addition of thalidomide to maintenance therapy on progression-free survival and overall survival in myeloma patients following autologous stem cell transplant.
At six weeks following autologous stem cell transplant, patients were evaluated and those with no evidence of disease progression were randomized to maintenance alternate day prednisolone, with or without thalidomide. In total, this included 243 patients from 29 centers.
The thalidomide plus prednisolone arm showed superior progression-free survival, with estimates at one, two and three years of 90 percent, 66 percent and 39 percent, respectively. This compares to the control arm, which showed 69 percent, 40 percent and 25 percent at one, two, and three years. The p-value was 0.0005.
The thalidomide plus prednisolone arm showed a trend toward improved overall survival to the control arm with estimates at one, two and three years, of 97 percent, 91 percent and 86 percent, respectively. This compares to the control arm, which showed 95 percent, 80 percent and 75 percent at one, two and three years. At this time the data are not mature, and do not reach statistical significance.
These data suggest that the addition of low-dose thalidomide to an alternate-day prednisolone maintenance regimen is an effective and tolerable therapeutic approach that prolongs the duration of disease response following stem cell transplant for multiple myeloma.
About Thalidomide Pharmion
Thalidomide Pharmion is approved in Australia, New Zealand, Turkey, Israel, Korea and Thailand for the treatment of multiple myeloma after the failure of standard therapies and the acute treatment of cutaneous manifestations of moderate to severe erythema nodosum leprosum (ENL). Thalidomide is not indicated as monotherapy for such ENL treatment in the presence of moderate to severe neuritis. Thalidomide is also indicated as maintenance therapy for prevention and suppression of the cutaneous manifestations of ENL recurrence.
As a condition of registration, the Pharmion Risk Management Program (PRMP) is mandatory in Australia, New Zealand, Turkey, Israel, Korea and Thailand. Prescribers and pharmacies are required to register with the PRMP in order to prescribe or dispense thalidomide, and patients are required to complete an informed consent process and to participate in a confidential surveillance registry. The PRMP is based on the S.T.E.P.S.(TM) program developed by Celgene Corporation in cooperation with the US Food and Drug Administration.
Safety Notice
If thalidomide is taken during pregnancy, it can cause severe birth defects or death to an unborn baby. Thalidomide should never be used by women who are pregnant or who could become pregnant while taking the drug. Even a single dose, one capsule (50 mg), taken by a pregnant woman can cause severe birth defects. Because thalidomide is present in the semen of male patients, males receiving thalidomide must always use a condom during sexual contact with women of childbearing potential even if he has undergone a successful vasectomy. Thalidomide Pharmion 50mg hard capsules will only be available under a special restricted distribution program. This program is called the Pharmion Risk Management Programme (PRMP). Under this program, only registered prescribers and pharmacists may dispense the drug. In addition, patients must be advised of, agree to and comply with the requirements of PRMP.
Thalidomide is known to cause nerve damage that may be permanent. Peripheral neuropathy is a common, potentially severe, side effect of treatment with thalidomide that may be irreversible. The most commonly observed adverse reactions associated with the use of thalidomide are constipation, somnolence and asthenia.
The other clinically most important adverse reactions associated with the use of thalidomide include orthostatic hypotension, decreased white blood cell counts including neutropenia, severe skin reactions including Stevens Johnson Syndrome and toxic epidermal necrolysis, headache, rash, eosinophilia, peripheral oedema, dyspnoea, dizziness, hypotension, bradycardia, symptomatic hypothyroidism, increase or decrease in platelet count, anaemia and, in HIV patients, an increase in HIV viral load.
Seizures, including grand mal convulsions, have been reported very rarely during the use of thalidomide in clinical practice. It has been suggested that thalidomide's anti-angiogenic properties may interfere with wound healing. Patients should be advised about associated adverse events and routinely monitored by a physician during treatment with thalidomide.
About ENL
ENL is a severe and painful complication of leprosy. Thalidomide has been used to treat ENL patients in the U.S. for more than twenty years through a U.S. Public Health Service compassionate use program.
About Multiple Myeloma
Multiple myeloma (also known as myeloma or plasma cell myeloma) is a cancer of the blood in which malignant plasma cells are overproduced in the bone marrow. Plasma cells are white blood cells that help produce antibodies called immunoglobulins that fight infection and disease. However, most patients with multiple myeloma have cells that produce a form of immunoglobulin called paraprotein (or M protein) that does not benefit the body. In addition, the malignant plasma cells replace normal plasma cells and other white blood cells important to the immune system. Multiple myeloma cells can also attach to other tissues of the body, such as bone, and produce tumors. The cause of the disease is unknown.
About Pharmion
Pharmion is a biotechnology company focused on acquiring, developing and commercializing innovation products for the treatment of hematology and oncology patients in the U.S., Europe and additional international markets. Pharmion has a number of products on the market including the world's first approved epigenetic drug, Vidaza(R), a DNA demethylating agent. For additional information about Pharmion, please visit the company's website at www.pharmion.com.
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995: This release contains forward-looking statements, which express the current beliefs and expectations of management. Such statements are based on current expectations and involve a number of known and unknown risks and uncertainties that could cause Pharmion's future results, performance or achievements to differ significantly from the results, performance or achievements expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such difference include the outcome of ongoing clinical trails, the status and timing or regulatory approvals; the impact of competition from other products under development by Pharmion's competitors; the regulatory environment and changes in the health policies and structure of various countries; uncertainties regarding market acceptance of products newly launched, currently being sold or in development; fluctuations in currency exchange rates, and other factors that are discussed in Pharmion's filings with the U.S. Securities and Exchange Commission. Forward-looking statements speak only as of the date on which they are made, and Pharmion undertakes no obligation to update publicly or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.
Pharmion CorporationCONTACT: Anna Sussman, Director, Investor Relations and CorporateCommunications of Pharmion Corporation, +1-720-564-9143
Web site: http://www.pharmion.com/