Genzyme’s Mozobil Shows Promise in Chemosensitization

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme Corporation (Nasdaq: GENZ) announced today that a Phase 1/2 trial provided early clinical data suggesting that Mozobil® (plerixafor injection) in combination with chemotherapy may offer a therapeutic impact on leukemic cells protected in bone marrow. The investigational trial, partially supported by Genzyme, was the subject of an oral presentation at the 2009 annual meeting of the American Society of Hematology in New Orleans, LA. Genzyme has initiated two clinical trials to further explore this potential new strategy to attack blood cancers.

“If we can begin to impact leukemic cells protected in bone marrow, we may be able to reduce residual disease and therefore provide better potential outcomes for patients,” said John F. DiPersio, M.D., Ph.D., professor, Washington University, St. Louis, the senior author of the study that was presented at ASH. “Our initial work in humans has produced some encouraging results.”

In the clinical trial, study director Geoffrey Uy, M.D. and colleagues from Washington University School of Medicine gave Mozobil as a pre-conditioning strategy prior to chemotherapy in forty patients with relapsed or refractory acute myeloid leukemia (AML). Many of the trial participants were either unresponsive to, or had short remissions, following prior treatments. The patients were given Mozobil, and four hours later received the AML combination chemotherapy regimen mitoxantrone, etoposide, and cytarabine (MEC). The Mozobil plus MEC regimen was repeated daily for five days, following the standard MEC treatment protocol.

Of the 32 patients available for the first follow-up evaluation, researchers observed a complete remission (CR or CRi) in 50 percent of patients. Thirteen of these patients had a CR with normal platelet recovery, and three patients had complete remission with incomplete platelet recovery (CRi). In a larger patient population, Washington University’s historical CR and CRi rate in relapsed and refractory AML patients receiving MEC alone ranges from 25 to 35 percent.

The study, developed primarily to determine optimal dosing and safety of Mozobil as a tumor sensitization agent, found no evidence of hyperleukocytosis, excessive release of white blood cells into the bloodstream. There was also no observed delay in the recovery of neutrophils or platelets important to immune system protection and prevention of bleeding, respectively. Treatment failure was considered to be due to persistent disease in 14 patients (44 percent) and death during aplasia (not producing enough new blood cells) in two patients (6 percent). Grade three or higher adverse events consisted primarily of cytopenias and infections, commonly seen in patients treated with the MEC combination alone.

Increasing the ability of chemotherapy to destroy cancerous blood cells in patients’ bone marrow has long been a goal of clinicians. Within the bone marrow environment, some studies suggest that certain cellular communication pathways and adhesion molecules work together to promote leukemic cell survival and poor response to chemotherapy. The ability of these cells to withstand chemotherapy contributes to minimal residual disease, small numbers of leukemic cells that remain in a patient during or after treatment, which is a cause of relapse in cancer.

Preclinical studies suggest that Mozobil may sensitize leukemic cells in the bone marrow to chemotherapy in several ways, including the disruption of a key signaling pathway that promotes leukemia cell survival, affecting interactions between stroma cells and leukemic cells, or by moving the cells out of the bone marrow entirely. Either separately or in combination, these potential actions may make cancer cells more susceptible to chemotherapy.

“We are excited by the interest that physicians have expressed in studying Mozobil for this potential new use,” said Mark Enyedy, president of Genzyme Oncology and Multiple Sclerosis. “The preclinical and early clinical data continue to support the development of Mozobil in this important area that could transform the treatment paradigm for hematologic cancers.”

Genzyme has also initiated company sponsored Phase 1 and Phase 1/2 Mozobil tumor sensitization trials in patients with blood cancers. The Phase 1 trial includes relapsed and refractory patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), and is designed to investigate Mozobil as a tumor sensitization agent in combination with the monoclonal antibody rituximab. The Phase 1/2 trial includes AML patients without prior treatment, and is evaluating Mozobil with the anti-cancer drug regimen cytarabine and daunorubicin (known as “7+3”). The primary objective of both trials is to evaluate safety and to define optimal dosing strategies. The company anticipates top-line data from these trials in 2011.

Mozobil is also being studied as a tumor sensitization agent in 10 additional investigator sponsored trials.

About Mozobil

Mozobil was approved in the United States in December 2008, where it is indicated for use in combination with granulocyte-colony stimulating factor (G-CSF) to mobilize hematopoietic stem cells (HSCTs) to the bloodstream for collection and subsequent autologous transplantation in patients with non-Hodgkin’s lymphoma and multiple myeloma.

Important Safety Information

Prescribing physicians and patients should be aware of the potential for tumor cell mobilization in leukemia patients, increased circulating leukocytes and decreased platelet counts, splenic enlargement, and fetal harm when administered to pregnant women. The most common adverse reactions (≥ 10%) reported in patients who received plerixafor in conjunction with G-CSF that were more frequent than in patients who received placebo were diarrhea, nausea, fatigue, injection site reactions, headache, arthralgia, dizziness and vomiting. For full prescribing information, please visit

About Genzyme

One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 11,000 employees in locations spanning the globe and 2008 revenues of $4.6 billion.

With many established products and services helping patients in nearly 100 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune disease, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as cardiovascular disease, neurodegenerative diseases, and other areas of unmet medical need.

This press release contains forward looking statements regarding the clinical development of Mozobil in a use other than its FDA approved indication. The forward looking statements include Genzyme’s estimates of: the therapeutic impact of Mozobil in chemosensitization, the potential for this treatment approach to reduce residual disease and provide better outcomes for patients; the ability of Mozobil to sensitize leukemic cells in the bone marrow by disrupting a key signaling pathway, affecting interactions between stroma cells and leukemic cells or moving those cells out of the bone marrow entirely; the possibility of transforming the treatment paradigm for hematologic cancers; and the anticipated timing for the availability of top-line data from the described trials. These forward looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those forecasted. These risks and uncertainties include: the risk that chemosensitization using Mozobil is not a viable therapeutic option for healthcare providers; the risk that the generation of data from these studies could negatively impact the approved use or other potential uses of Mozobil; and the uncertainty of the timing of data availability; and the risks and uncertainties described in reports filed by Genzyme with the Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended, including without limitation the information under the heading "Risk Factors" in Genzyme’s Quarterly Report on Form 10-Q for the quarter ending September 30, 2009. Genzyme cautions investors not to place substantial reliance on the forward-looking statements contained in this press release. These statements speak only as of the date of this press release, and Genzyme undertakes no obligation to update or revise these statements.

Genzyme® and Mozobil® are registered trademarks of Genzyme Corporation or its subsidiaries. All rights reserved.

Media Contact:
John Lacey, 617-768-6690; 857-222-8589 (mobile)
Investor Contact:
Emily Merchant, 617-768-6140

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