Healthcare Industry News: Azacitidine
News Release - December 11, 2006
New Data Validate Vidaza(R) Response Rates in MDS and Report Results in AMLAbstracts Presented at 2006 ASH Annual Meeting Highlight Alternate Dosing Schedules in MDS and Treatment of AML
ORLANDO, Fla., Dec. 11 (HSMN NewsFeed) -- Pharmion Corporation (Nasdaq: PHRM ) announced new data presented today at the 48th Annual Meeting and Exposition of the American Society of Hematology (ASH) in Orlando, December 9-12, 2006) on VidazaŽ (Azacitidine for injectable suspension) as single-agent therapy for the treatment of myelodysplastic syndromes (MDS) and acute myelogenous leukemia (AML).
Data on Vidaza Use in High Risk MDS and AML Post-MDS Demonstrate Response Rates of 74 Percent (Abstract 2664, Poster 842-II)
Claire Fabre, MD, of the Groupe Francophone des Myelodysplasies (GFM), Hopital Avicenne in Bobigny, France, delivered a poster presentation describing results of a study of high-risk MDS and AML post-MDS patients treated under the French ATU, which provides a temporary authorization for compassionate use of Vidaza prior to receipt of a marketing approval.
In this study of 78 patients evaluable for response after four cycles of Vidaza, overall response was 74 percent, including 14 percent complete responders, 29 percent partial responders, and 28 percent who demonstrated hematologic improvement.
Median overall response duration was six months, including 7.5 months for complete responders, nine months for partial responders and two months for patients who demonstrated hematologic improvement. Accrual in this study is ongoing.
Vidaza use in this study was associated with responses similar to those reported in the pivotal CALGB study as published in the Journal of Clinical Oncology in May 2002.
Data from Vidaza Alternative Dosing Study Show Hematologic Improvement of 49 to 67 Percent without Weekend Dosing (Abstract 2662, Poster #840-II)
Roger M. Lyons, MD, of US Oncology Research, delivered a poster presentation describing interim results of a Phase 2 study designed to evaluate the treatment response and safety of three alternative subcutaneous doses of Vidaza, each of which eliminates the need for weekend dosing.
Of the 130 patients randomized, 99 had received two or more cycles of Vidaza at the time of evaluation. Hematologic improvement was demonstrated in 57 percent of those patients, with a range of 49 to 67 percent in the different arms.
Also, of 46 baseline RBC transfusion-dependent patients, 70 percent became transfusion independent, with a range of 58 to 80 percent in the different arms.
Based on preliminary results, all three alternative doses provide clinical benefit, including transfusion independence and hematologic improvement.
Vidaza Demonstrates Hematologic Response in Patients with AML Refractory to or Not Eligible for Intensive Chemotherapy (Abstract 1953, Poster #131-II)
Haifa K. Al-Ali, MD, of the University of Leipzig, Germany, delivered a poster presentation describing interim results of a German study underway evaluating the safety and efficacy of Vidaza in AML patients.
This study, which utilizes a dose of 75 mg/m2 per day for five days every 28 days, had enrolled 18 patients at the time of analysis. Eight patients were ineligible for chemotherapy, and 10 had relapsed or refractory disease after standard chemotherapy.
After a median follow up of 23 weeks, 67 percent of patients responded after a median of four treatment cycles; this included 33 percent complete responders, 11 percent partial responders, 11 percent with hematologic improvement and 11 percent with stable disease.
Pharmion Corporation has several studies currently underway evaluating Vidaza in the treatment of AML.
"We are encouraged by these data presentations on Vidaza as single-agent therapy, as they further validate its clinical activity in the treatment of hematologic malignancies," said Patrick J. Mahaffy, Pharmion's president and chief executive officer. "We continue to expand our clinical development program for Vidaza, and look forward to final data from the Vidaza survival study and an increasing body of data on Vidaza in combination studies for higher-risk MDS, AML and other malignancies."
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.
In May 2004, Vidaza became the first drug approved by the FDA for the treatment of patients with Myelodysplastic Syndromes (MDS). The FDA approved Vidaza, the first in a new class of drugs called demethylation agents, for the treatment of all five MDS subtypes, which include both low-risk and high-risk patients. These subtypes include: refractory anemia (RA) or 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).
Vidaza is believed to exert its antienoplastic 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.
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, VidazaŽ, 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.
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