The results were from 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%, including 14% complete responders, 29% partial responders, and 28% who demonstrated hematologic improvement.
Pharmion also announced interim results of a phase II 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. Hematologic improvement was demonstrated in 57% of patients in the study, with a range of 49 to 67% in the different arms.
Results of a further study into the safety and efficacy of the drug in AML patients also gave encouraging results. Patrick Mahaffy, Pharmion's president and CEO said: “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.
“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 US.