Vidaza

Researchers Present Data on Survival, Time to AML transformation and Safety

BOULDER, Colo., Dec. 11 /PRNewswire-FirstCall/ -- Pharmion Corporation reported today on several analyses of data from the Vidaza pivotal Phase III study. The results of these and several other Vidaza studies were presented at the American Society of Hematology 47th Annual Meeting and Exposition in Atlanta.

Data Analysis Highlights Survival, Time to AML Transformation (Abstract 2524, Poster #728)

A retrospective analysis of efficacy in high-risk MDS patients (RAEB or RAEB-T, age 65 or older) was presented on Sunday, December 11. Sixty-eight patients were included in the intent-to-treat (ITT) analysis of the pivotal data, including 31 patients randomized to Vidaza, and 37 patients randomized to supportive care. Patients who crossed over from supportive care to Vidaza were included as per the ITT analysis. Median overall survival was 19.5 months in the Vidaza arm, and 14.0 months in the supportive care arm, a difference of 5.5 months (p=0.04). Median time to AML transformation with Vidaza was 42.0 months, compared to 17.7 months on supportive care, representing a two-year difference (p=0.04). Median time to the combined endpoint of death or AML transformation was 19.1 months in the Vidaza arm compared to 9.2 months with supportive care, or 9.9 months longer on Vidaza therapy (p=0.008).

"We are encouraged by the data presentations on survival and transformation to AML with Vidaza treatment compared to supportive care in the high-risk MDS patient population," said Patrick J. Mahaffy, Pharmion's president and chief executive officer. "We look forward to the completion of our Phase III/IV prospectively-designed study to support these data."

Data Analysis Highlights Activity in AML (Abstract 1848, Poster #52)

A retrospective analysis of response rates in patients with Acute Myeloid Leukemia (AML) was presented on Sunday, December 11. Data from a total of 105 patients, who were reclassified as AML using the WHO criteria (>20 percent bone marrow blasts), treated with either Vidaza or supportive care demonstrates Vidaza's activity using the IWG response criteria. This analysis was based on three clinical studies (8421, 8921 and 9221) in which the French- American-British (FAB) criteria was originally used. Overall response rates (CR + PR +HI) based on IWG response criteria for MDS were 48 percent, 32 percent and 37 percent, for studies 8421, 8921 and 9221, respectively, for Vidaza patients with WHO AML. In study 9221, which was the only study to include a comparator arm, Vidaza patients (n=27) achieved a median survival of 19.3 months in comparison to the supportive care patients (n=25) who had a median survival of 12.9 months. In addition, the analysis demonstrated median transfusion independence of 411 days for red blood cells and 363 days for platelets.

Data Examines Rates of Infection and Bleeding in MDS Patients (Abstract 2525, Poster #729)

A retrospective analysis of the pivotal study which examined rates of infection and bleeding in all five FAB subtypes of MDS was presented on Sunday, December 11. The study compared Vidaza plus supportive care (n=99) versus supportive care alone (n=92) in patients with MDS. For this analysis, the Vidaza group included 150 patients, those randomized to the Vidaza arm (n=99) and patients who crossed over from supportive care to Vidaza treatment (n=51) as permitted in the pivotal study. A total of 147 of the 150 Vidaza-treated patients had pre-existing cytopenias. In addition, the analysis looked at 88 patients considered to be high-risk (RAEB or RAEB-T) and age 65 or older.

The Vidaza arm had a 0.64 rate of infection per patient-year of exposure. The supportive care arm had a rate of 0.95 per patient-year. Infections also occurred at a lower rate in the high-risk patient group, 0.38 in the Vidaza arm and 0.76 in the supportive care arm.

The overall rate (patient-years) of bleeding events was comparable, 0.56 in the Vidaza arm and 0.60 in the supportive care arm. Bleeding rates were also comparable between the high-risk patients treated with Vidaza (0.32) or supportive care (0.44).

"We continue to be encouraged by the safety and efficacy data we've seen with Vidaza in the MDS and AML patient populations," added Mahaffy. "These data are particularly important because patients with MDS, especially those with higher-risk forms of the disease, don't have many treatment options."

MDS are a group of diseases in which the bone marrow does not function normally, resulting in the production of malformed or immature blood cells. MDS affects approximately 40,000-50,000 people in the United States. The majority of patients with high-risk MDS eventually experience bone marrow failure, which can cause bleeding and infection that lead to death.

Vidaza is currently the only marketed treatment for all five subtypes of myelodysplastic syndromes (MDS), including refractory anemia (RA), refractory anemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL).

About Vidaza

Vidaza is believed to exert its antineoplastic effects by causing hypomethylation of DNA and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. The concentration of azacitidine required for maximum inhibition of DNA methylation in vitro does not cause major suppression of DNA synthesis. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation. The cytotoxic effects of azacitidine cause the death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanisms. Non-proliferating cells are relatively insensitive to Vidaza.

Important Safety Information

Vidaza is contraindicated in patients with a known hypersensitivity to azacitidine or mannitol and in patients with advanced malignant hepatic tumors.

In clinical studies, the most commonly occurring adverse reactions were nausea (70.5%), anemia (69.5%), thrombocytopenia (65.5%), vomiting (54.1%), pyrexia (51.8%), leukopenia (48.2%), diarrhea (36.4%), fatigue (35.9%), injection site erythema (35.0%), constipation (33.6%), neutropenia (32.3%) and ecchymosis (30.5%). Other adverse reactions included dizziness (18.6%), chest pain (16.4%), febrile neutropenia (16.4%), myalgia (15.9%), injection site reaction (13.6%), aggravated fatigue (12.7%) and malaise (10.9%).

Because treatment with Vidaza is associated with neutropenia and thrombocytopenia, complete blood counts should be performed as needed to monitor response and toxicity, but at a minimum, prior to each dosing cycle.

Because azacitidine is potentially hepatotoxic in patients with severe pre-existing hepatic impairment, caution is needed in patients with liver disease. In addition, azacitidine and its metabolites are substantially excreted by the kidneys and the risk of toxic reactions to this drug may be greater in patients with impaired renal function. Because elderly patients are more likely to have decreased renal function, it may be useful to monitor renal function.

Vidaza may cause fetal harm. While receiving treatment with Vidaza, women of childbearing potential should avoid becoming pregnant, and men should avoid fathering a child. In addition, women treated with Vidaza should not nurse.

About MDS

The highest prevalence of MDS is in patients over 60 years of age. According to the American Cancer Society and the Aplastic Anemia and MDS International Foundation, there are approximately 10,000-30,000 new cases of MDS in the United States each year. Survival ranges from six months to many years for the different subtypes of MDS.

About Pharmion

Pharmion is a pharmaceutical company focused on acquiring, developing and commercializing innovative products for the treatment of hematology and oncology patients in the U.S., Europe and additional international markets. For additional information about Pharmion, please visit the company's website at http://www.pharmion.com/.

For more information or complete prescribing information about Vidaza, please call 1-866-PHARMION.

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 differences include the status and timing or regulatory approvals for Thalidomide Pharmion 50mg and Vidaza; 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; acceptance and demand for new pharmaceutical products and new therapies, uncertainties regarding market acceptance of products newly launched, currently being sold or in development; Pharmion's ability to successfully acquire rights to, develop and commercialize additional pharmaceutical products; 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.

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