Genzyme Corporation Announces Phase 3 Trial of Mozobil in non-Hodgkin's Lymphoma Meets Primary Endpoint

Genzyme Corp. (Nasdaq: GENZ) today announced that it has successfully completed its phase 3 trial of MozobilTM (plerixafor) in non-Hodgkin’s lymphoma (NHL), and that the trial has robustly met its primary and secondary endpoints.

The randomized, double-blind, placebo-controlled trial included 298 patients who were undergoing a hematopoietic stem cell transplant (HSCT) for NHL at medical centers in the United States and Canada. It examined the effectiveness of Mozobil in increasing the number of hematopoietic stem cells collected for a transplant. The study compared the hematopoietic stem cell yield from patients treated with Mozobil in combination with G-CSF to patients treated with G-CSF in combination with placebo. G-CSF is the standard of care for stimulating the mobilization of stem cells from the bone marrow; Mozobil is designed to allow for the more rapid and effective release of those stem cells from the marrow into the circulating blood for collection by apheresis.

In the primary efficacy endpoint, 59 percent of patients treated with a combination of Mozobil and G-CSF achieved the target threshold for collection of at least 5 million CD34+cells/kg from the peripheral blood with four or fewer days of apheresis sessions, compared with 20 percent of patients in the G-CSF/placebo group. The three-fold increase was highly statistically significant in favor of the Mozobil-treated patients (p<0.0001). The 40 percent absolute difference between the two treatment groups was nearly double the target that Genzyme prospectively defined in the protocol for the study, which was reviewed by FDA as part of the Special Protocol Assessment process.

In the secondary efficacy endpoint, nearly 87 percent of patients treated with Mozobil and G-CSF achieved the minimum level of stem cells generally associated with a successful transplant (2 million CD34+cells/kg) in four or fewer days of apheresis sessions, compared with approximately 47 percent in the placebo arm. This result was also highly statistically significant in favor of the Mozobil-treated patients (p<0.0001).

The other secondary efficacy endpoints were supportive of these findings, including analysis of the number of days needed to reach target ranges for stem cell mobilization, the success of engraftment, the number of days needed to engraft, and the durability of the engraftment for the first 100 days.

Mozobil was well tolerated in the trial, with the most common adverse events being mild gastrointestinal effects and redness at the site of injection. There were two related serious adverse events seen in the Mozobil plus G-CSF arm, and one in the G-CSF plus placebo arm.

“These are very impressive results with far-reaching clinical importance for patients undergoing a stem cell transplant for lymphoma,” said Principal Investigator John F. DiPersio, M.D., Ph.D., professor, Washington University, St. Louis. “Current literature suggests that increasing the number of stem cells in circulation and the number collected at the time of apheresis may improve the outcomes of patients undergoing a stem cell transplant, reduce the costs associated with stem cell collection and, more importantly, broaden the pool of patients for whom transplantation is an option.”

Based on these results Genzyme expects to file for US and European approval in lymphoma in the first half of 2008. In addition, Genzyme is completing a second phase 3 trial of Mozobil in multiple myeloma, and results are expected in the coming weeks.

About Mozobil

Mozobil, a novel small molecule CXCR4 chemokine antagonist, has been shown in multiple earlier studies to rapidly and effectively increase the number of stem cells in circulation in the blood. Once circulating in the blood, stem cells can be collected for use in a stem cell transplant. Mozobil has been granted special protocol assessment and orphan drug status in the United States and European Union and the pivotal trials have undergone Special Protocol Assessment by the FDA and Protocol Assistance by the EMEA. Genzyme intends to commercialize Mozobil through its existing global transplant business to hematologists and hematopoietic stem cell transplant centers in more than 50 countries throughout the world. Genzyme has been developing Mozobil since its acquisition of AnorMED, Inc. in 2006.

Approximately 55,000 stem cell transplants are performed each year for multiple myeloma, Hodgkin’s and non-Hodgkin’s lymphoma, and other conditions in markets where Genzyme has a commercial infrastructure, including the United States, Europe, Latin America and the Asian Pacific countries.

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 9,000 employees in locations spanning the globe and 2006 revenues of $3.2 billion. Genzyme has been selected by FORTUNE as one of the “100 Best Companies to Work for” in the United States.

With many established products and services helping patients in nearly 90 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 diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as immune disease, infectious disease, and other areas of unmet medical need.

Genzyme to Build New Biomanufacturing Plant to Support Growth of Thymoglobulin

Genzyme Corp. announced today that it will build a new biomanufacturing plant in Lyon, France, for the production of Thymoglobulin® (Anti-thymocyte Globulin [Rabbit]), a treatment used in transplantation. The facility is needed to meet the anticipated long-term demand for Thymoglobulin, both for its current use and in potential new indications. The project represents one of a number of investments Genzyme is making in its global manufacturing infrastructure to support the growth of existing products and to prepare for the introduction of future products from its rich late-stage pipeline.

In addition to the Lyon project, Genzyme is completing the development of a new plant in Belgium for the production of monoclonal antibodies and proteins and expanding other facilities, including its Allston Landing protein manufacturing plant in Boston. Genzyme also recently received regulatory approvals for a new filling and packaging facility at its manufacturing site in Ireland, which has become the company’s major European center for the final production and distribution of a range of products, including Thymoglobulin. Genzyme operates fifteen GMP manufacturing plants around the world, seven of them in Europe.

Thymoglobulin’s growth is being driven by its launch in new geographic markets and by publications and clinical studies. Sales last year increased 17 percent compared with the previous year. The product is currently approved in more than 50 countries for various indications, including the treatment and the prevention of acute rejection of solid organ transplants. Thymoglobulin has encouraging potential beyond the transplantation field, and current studies are exploring its applicability to hematological disorders and autoimmune diseases such as diabetes.

The new 140,000-square-foot facility in Lyon will replace Genzyme’s smaller plant in nearby Marcy l’Etoile and will provide more than twice the manufacturing capacity. Approximately 165 people are employed at the existing Thymoglobulin plant, and Genzyme expects that approximately 50 new jobs will be created when the new facility operates at full capacity. The total cost of the project is expected to be €105 million.

The new plant will be located within the Lyon-Gerland Biopôle, an area dedicated to biotechnology. The facility will be situated on a nine-acre site, which will allow Genzyme to expand the plant if needed, as it has done at a number of its other manufacturing locations. Construction is expected to begin later this year, and regulatory approvals are expected starting in 2010. Routine manufacturing at the site is expected to commence in 2011.

“As with our other new facilities, we intend the Lyon plant to serve as a highly visible expression of Genzyme’s purpose, which is to innovate and set new standards in both the products that we develop for patients and in the way that we operate as a company,” said Mark Bamforth, senior vice president for Corporate Operations.

Consistent with Genzyme’s commitment to innovation and sustainable development, the facility will feature an attractive architectural design and innovative “green” features that will create a healthy and comfortable indoor environment for employees and reduce the plant’s impact on the external environment. Genzyme intends to develop the site using a High Environmental Quality (HEQ) approach and to obtain HEQ certification by meeting or exceeding standards set by the Center for Scientific and Technical Building. The plant would be one of the first manufacturing sites in France to gain HEQ certification, which is similar to the certification conferred by the U.S. Green Building Council through its LEED® (Leadership in Energy and Environmental Design) Green Building Rating System™.

In the United States, Genzyme is currently completing the construction of a new Science Center, which could become one of the first research laboratories to earn certification under the LEED system. In addition, the company’s landmark world headquarters building, Genzyme Center, has earned Platinum certification—the highest possible rating under the LEED system.

The Lyon facility will incorporate progressive design strategies similar to those in Genzyme Center and the Science Center. The initial design of the Lyon facility was completed by French architects Patriarche & Co. and engineers SNC Lavalin, in collaboration with Genzyme.

“This investment sends a clear message of support for French research and biotechnology and it illustrates Genzyme’s culture of establishing strong partnerships in the regions where we operate,“ said Frédéric Turner, Genzyme’s senior vice president and general manager for France and North Africa.

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 9,000 employees in locations spanning the globe and 2006 revenues of $3.2 billion. Genzyme has been selected by FORTUNE as one of the “100 Best Companies to Work for” in the United States.

With many established products and services helping patients in nearly 90 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 diagnostic testing. Genzyme’s commitment to innovation continues today with a substantial development program focused on these fields, as well as immune disease, infectious disease, and other areas of unmet medical need.