Genzyme Reports More Good News For Mozobil

Second Phase III trial shows twice the stem cell mobilization power called for in multiple myeloma protocol, adding to high efficacy shown in lymphoma study reported in July.

Based on the strength of results from a second Phase III trial, Genzyme will file an NDA for Mozobil (plerixafor) in patients undergoing autologous hematopoietic stem cell transplantation for both non-Hodgkin's lymphoma and multiple myeloma in the first half of 2008, the company said Aug. 2.

Genzyme said it anticipates receiving a priority review of its application, with marketing approval following at the end of 2008 and a launch "shortly after" in early 2009.

The company is working with the National Comprehensive Cancer Network "to look at standard of care guidelines with the goal of making Mozobil standard of care in stem cell transplant," Joe M. Lobacki, general manager of Genzyme's transplant business unit, said during a same-day conference call.

The Cambridge, Mass., biotech reported in July that Mozobil had demonstrated efficacy well beyond the parameters FDA agreed upon in a special protocol assessment for testing the granulocyte colony stimulating factor adjunct in NHL patients (¹ (Also see "Genzyme Reports Positive Phase III Results For Mozobil In Stem Cell Transplant" - Pink Sheet, 19 Jul, 2007.)).

As in the NHL trial, the MM study examined the effectiveness of Mozobil in increasing the number of hematopoietic stem cells collected for a transplant, comparing the stem cell yield from patients treated with Mozobil following G-CSF to patients treated with placebo following G-CSF.

In the MM trial, Mozobil met both primary and secondary endpoints, nearly doubling the 20 percent absolute difference prospectively defined in the SPA to 38 percent, P.K. Tandon, a VP-Biomedical Operations, said during the call.

Of the 140 patients randomized to receive 240 mcg/kg Mozobil plus the standard 10 mcg/kg G-CSF, 72 percent achieved the target threshold for collection of at least six million CD34+ cells/kg from the peripheral blood with two days or fewer of apheresis sessions, Tandon said. That is compared with 34 percent of 155 patients in the G-CSF/placebo group (p<0.0001).

The median number of days of apheresis needed was one for the Mozobil group and four for the placebo group. "Greater than 50 percent" of patients in the Mozobil group achieved their target cells in just one day," Tandon said.

"We plan on expanding the applications to include allogeneic transplantation, and not only the traditional uses of allotransplantation, but ... there are new applications of allotransplantation, such as nonmyeloablative transplants that help to make this type of therapy available to a broader array of patients, older patients and potentially some patients with solid tumors," Mark A. Goldberg, Sr VP-Clinical Research, said.

Genzyme is also exploring the use of Mozobil in chemosensitization and immunosensitization, Goldberg noted. The object is "to get refractory leukemic cells out of the bone marrow niche, where they're more refractory to attack by chemotherapy and immunotherapy, get them into the peripheral blood where they're more susceptible," he explained.

The firm currently has three chemosensitization studies lined up, one has begun patient screening and two others will start in September or October, Goldberg said. Another two studies are planned for the first half of 2008.

In addition, the firm anticipates investigating Mozobil against "solid tumors ... where we utilize the known ability of Mozobil to mobilize endothelial progenitor cells from the bone marrow in an attempt to improve angiogenesis," Goldberg said.

The firm now projects peak worldwide sales of Mozobil in the transplant setting at more than $400 million, assuming its approval in a range of HSCT therapies. "Approximately 55,000 autologous and allogeneic stem cell transplants are performed each year," according to Genzyme.

- Shirley Haley ([email protected])