Millennium announces data on VELCADE in treatment of patients with amyloidosis

Millennium: The Takeda Oncology Company today announced data examining the use of VELCADE in the treatment of patients with relapsed or refractory light chain (AL) amyloidosis. Amyloidosis is a rare condition in which excessive proteins build up in organs or tissues, such as the heart, kidney or liver. These data were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) held in Chicago, Illinois, June 4-8, 2010.

“Single-agent VELCADE activity yielded impressive hematologic overall response rates in patients with relapsed or refractory disease. These data further support the rationale for Phase III studies of VELCADE in amyloidosis.”

"Amyloidosis is a rare disease for which there are currently no approved therapies, creating a large unmet medical need in the patient community," said Nancy Simonian, M.D., Chief Medical Officer, Millennium. "Single-agent VELCADE activity yielded impressive hematologic overall response rates in patients with relapsed or refractory disease. These data further support the rationale for Phase III studies of VELCADE in amyloidosis."

The Phase I/II international trial examined the use of single-agent VELCADE in patients with relapsed or refractory systemic AL amyloidosis who required further treatment. The trial was an open-label, non-randomized, dose-escalation study evaluating VELCADE in 70 patients. In the Phase II portion of the trial, 52 patients were treated at the maximum planned dose of 1.6 mg/m² weekly or 1.3 mg/m² twice-weekly. Primary and secondary objectives are safety and hematologic response rate, respectively. Donna Reece, M.D., Princess Margaret Hospital, Toronto, Canada, presented the following results:

• Hematologic overall response rates (CR+PR), as measured by serum and urine M-protein levels, in the weekly and twice-weekly groups were 69% and 67%; complete response rates were 37% and 24%, respectively
• More than 76% of patients had a duration of response of one year or longer
• The rate of serious adverse events (AE) was 44% and 41% and discontinuation due to AEs was 28% and 38% in the weekly and twice-weekly schedules, respectively. The most common AEs ≥ Grade 3 included fatigue, thrombocytopenia, vomiting, diarrhea and pneumonia. No peripheral neuropathy ≥ Grade 3 was seen

Fifty-two patients received single-agent VELCADE either weekly>2 on days 1, 8, 15 and 22 of 35-day cycles or twice-weekly>2 on days 1, 4, 8 and 11 of 21-day cycles.