Genzyme’s Second Mozobil Trial Meets Endpoint

Genzyme Corp. met its primary and secondary endpoints in a second Phase III trial of Mozobil in multiple myeloma (MM) similar to the recent trial in non-Hodgkin’s lymphoma. The combined results of these two trials—in which patients with two types of cancer achieved more rapid and effective mobilization of stem cells in preparation for transplant than patients treated with current therapies—will support the drug’s regulatory approval.

The randomized, double-blind, placebo-controlled trial included 302 patients undergoing a hematopoietic stem cell transplant (HSCT) for MM and examined the effectiveness of Mozobil in increasing the number of stem cells collected for a transplant. The trial then compared the stem cell yield from patients treated with Mozobil following G-CSF to patients treated with placebo following G-CSF.

In the primary efficacy endpoint, 72% of patients treated with Mozobil and G-CSF achieved the target threshold for collection, compared with 34% of patients in the G-CSF/placebo group. Like the previous trial, these results exceed the 20% absolute difference prospectively defined through the FDA’s SPA as a successful result.

More than half of the patients treated with Mozobil and G-CSF reached the target cells in the first day of apheresis. By comparison, it took four days for a similar percentage of the G-CSF/placebo group to reach this threshold.

Secondary outcomes were consistent with the primary endpoint, showing a statistically significant result in favor of Mozobil. The drug was well tolerated in the trial, with the most common adverse events being mild gastrointestinal effects and redness at the site of injection.