Mozobil Clinical Efficacy in Non-Hodgkin’s Lymphoma

The efficacy and safety of Mozobil in conjunction with granulocyte-colony stimulating factor (G-CSF) in non-Hodgkin’s lymphoma (NHL) patients were evaluated in a randomized, double-blind, placebo-controlled, multicenter phase 3 study (study 1).

Mozobil + G-CSF increased the number of patients achieving both minimum and target CD34+ cells/kg in fewer apheresis sessions compared with placebo + G-CSF.

Study 1 Endpoints

• Primary endpoint:

• Number of patients reaching a target of ≥ 5 × 10⁶ CD34+ cells/kg in ≤ 4 days of apheresis

• Secondary endpoints:

• Number of patients who achieved a minimum of ≥ 2 × 10⁶ CD34+ cells/kg in ≤ 4 days of apheresis
• Number of apheresis days required to reach the target of ≥ 5 × 10⁶ CD34+ cells/kg
• Time to neutrophil and platelet engraftment
• Graft durability at 100 days, 6 months, and 12 months
• Safety of Mozobil (0.24 mg/kg) + G-CSF

Patients

Study 1 randomized 298 patients with NHL, 150 Mozobil + G-CSF and 148 placebo + G-CSF. Mean age was 55.1 years for Mozobil patients and 57.5 years for placebo patients. Most (92.6%) subjects were Caucasian. More patients in the Mozobil group had late stage disease (stage IV), 48% versus 35%, respectively.

Ten patients treated with Mozobil + G-CSF and 52 patients treated with placebo + G-CSF failed to collect either 0.8 x 10⁶ cells/kg after 2 apheresis days or at least 2 x 10⁶ CD34+ cell/kg in 4 apheresis days. These patients entered the rescue protocol and went on to receive open label Mozobil + G-CSF. Among these rescue patients 37/62 (59.7%), 4/10 from the Mozobil group and 33/52 from the placebo group, achieved ≥ 2 x 10⁶ CD34+ cells/kg in 4 or fewer days of apheresis after a course of Mozobil + G-CSF; 84% proceeded to transplant.

Study 1 Outcomes

• Three times as many patients with NHL reached the primary endpoint of ≥ 5 million CD34+ cells within 4 days of apheresis with Mozobil + G-CSF vs. with G-CSF + placebo.
• 90% of NHL patients proceeded to transplant when treated with Mozobil + G-CSF vs. 55.4% treated with G-CSF + placebo.
• 87% of NHL patients collected ≥ 2 x 10⁶ CD34+ cells/kg within 4 apheresis days with Mozobil + G-CSF vs. 47% with G-CSF + placebo.

Kaplan-Meier Estimate of Required Apheresis Days to Collect ≥ 5 x 10⁶ CD34+ Cells/kg in NHL Patients

• NHL patients receiving Mozobil + G-CSF had a greater day 1 collection of CD34+ cells/kg than patients receiving placebo + G-CSF.

Kaplan-Meier Estimate of Percentage of NHL Patients Who Achieved ≥ 2 x 10⁶ CD34+ Cells/kg by Apheresis Day

Genzyme. Data on File.