Update from Genzyme on ASMD Clinical Trials

Update on Clinical Trial of Enzyme Replacement Therapy (ERT) for Acid Sphingomyelinase Deficiency (ASMD)

The National Niemann-Pick Disease Foundation (NNPDF) has been staying in touch with representatives from Genzyme with regard to the status of Phase 2 of the Enzyme Replacement Therapy (ERT) clinical trial for Acid Sphingomyelinase Deficiency (ASMD) NPD Type B.

It seems there has been some confusion among members of the ASMD community; please note that we have contacted Genzyme and confirmed that the Phase 2 trial has not been cancelled and the Genzyme/Sanofi Company is committed to the ongoing support of our NPD Type A and B patients and families.

Genzyme has advised that they are still actively preparing for a Phase 2 clinical trial for enzyme replacement therapy in ASMD/NPD Type B. The trial is expected to evaluate the safety and efficacy of different doses of rhASM when administered once every two weeks.

The program remains a key priority for Sanofi. They are committed to the Niemann-Pick community and have provided the NNPDF with an update on the Genzyme-sponsored Acid Sphingomyelinase Deficiency (ASMD) Clinical Trials.

To read the update from Genzyme, visit the NNPDF's NewsLine or Enzyme Replacement Therapy Type B page.  To learn more about Niemann-Pick Disease of all types, visit http://www.nnpdf.org/.

Good Things Happening at Genzyme

Sanofi-Aventis Prioritizes Enzyme Replacement Therapy and
Genzyme to be Honored by NORD

Sanofi-Aventis CEO Chris Viehbacher recently outlined the research and development strategy that led to the purchase of Genzyme Corp., and which will lead the company to further expand its partnerships and acquisitions.

Good news for Niemann-Pick Disease Type B patients -- Viehbacher announced that three Genzyme programs would be priorities in particular, one being Genzyme's enzyme replacement therapy for NPB.

Genzyme is also in the news being honored by the National Organization for Rare Diseases (NORD), the nonprofit organization representing 30 million Americans with rare diseases, at NORD's annual Partners in Progress Celebration on May 17. Genzyme will receive the first-ever Power of Partnership Award.

Genzyme's Boston Marathon Team paired up with an NNPDF Type B family, and the runners dedicated their race in honor of their partners, wearing their partners' photos on their shirts. The Power of Partnership Award will now be presented annually to individuals, organizations, or companies for outstanding acts of volunteerism demonstrating partnership with the rare disease patient community.

Visit the NNPDF NewsLine page for more information and links.

Congratulations, and Thank You to Genzyme!

Sale of Genzyme to Sanofi-Aventis

Update from Jamie Manganello, Director, Patient Advocacy, at Genzyme:


Genzyme and sanofi-aventis have entered into an agreement under which sanofi-aventis will acquire Genzyme. Sanofi-aventis is a global, diversified healthcare company, headquartered in Paris, France and a leader in diabetes, oncology, innovative medicines, vaccines, consumer health care products and animal health. With this transaction, Genzyme will move into a new phase of our development, continuing our patient-focused mission and developing treatments that change the lives of people with rare diseases.

Sanofi-aventis and Genzyme have a shared vision for our future together and believe we will emerge even better prepared to serve you. The plan is for Genzyme to become a division of sanofi-aventis and global center of excellence for rare diseases. We will continue to serve the rare genetic disease community as we have done for the past 30 years, and I believe that sanofi-aventis will bring important new perspectives and new resources to this work.

Until the transaction closes, which is expected early in the second quarter of this year, both companies remain independent. Every aspect of how we work and our commitment to you remain the same.

In partnership with you, Genzyme’s mission has always begun and ended with the patient. Sanofi-aventis is making a significant investment in Genzyme because they value what we do, and our mission will remain unchanged.

As we work through the details, we will share our progress with you. Our future is dependent upon the support and involvement of patients and the treatment community, and we are eager to hear your thoughts and feedback as well.

Thank you for your continued support of Genzyme, and we look forward to updating you soon on our progress and path forward.

Visit the NNPDF's NewsLine page for a link to the press release from Sanofi-Aventis and Genzyme.

Genzyme Completes Sale of Diagnostics Business

Genzyme Completes Sale of Diagnostics Business to Sekisui Chemical Co.
Agrees to Sell Pharmaceuticals Business to International Chemical Investors Group

Genzyme announced that it has completed the sale of its diagnostic products business to Sekisui Chemical Co., Ltd.

Sekisui purchased substantially all of the assets of the business, including diagnostic product lines and technologies. Genzyme’s approximately 575 employees were offered similar positions with Sekisui, which plans to maintain operations in each of the business’s current locations. In connection with the sale, Sekisui also entered into a supply agreement to provide Genzyme with certain enzymes needed for the production of Cerezyme® (imiglucerase for injection).

Genzyme also announced that it has entered into a purchase agreement under which an affiliate of International Chemical Investors Group (ICIG) will acquire Genzyme’s pharmaceutical intermediates business.

For a link to the complete press release, visit the NNPDF's NewsLine page.

Update from Genzyme on Acid Sphingomyelinase Deficiency (ASMD) Clinical Trials

A note from Genzyme - January 2011:

Genzyme is continuing efforts to develop recombinant human acid sphingomyelinase (rhASM) for the potential treatment of the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease Types A and B). After completing the Phase 1 clinical trial in 2009, we engaged regulatory authorities in discussion about plans for a Phase 2 clinical trial and conducted additional preclinical research in 2010. This regulatory dialogue is ongoing. We remain committed to the development of a therapy for ASMD and will keep the community informed once our regulatory discussions are complete and we can confirm a start date for the Phase 2 clinical trial for Niemann-Pick B patients.

We are pleased to note that an abstract from the Phase 1 clinical trial was selected for a podium presentation by Dr. Margaret McGovern at the 7th annual lysosomal disease network's WORLD conference, to be held in Las Vegas, February 16-18, 2011.

Genzyme Corporation
500 Kendall Street
Cambridge, MA 02142
www.genzyme.com


(To learn more about Niemann-Pick Disease, visit www.nnpdf.org. To learn more about these clinical trials, please visit the National Niemann-Pick Disease Foundation's Enzyme Replacement Therapy page.)

Genzyme Marathon Team Runs for Rare Diseases

Genzyme Marathoners Raise Funds and Awareness for Rare Diseases
Two Run Boston Marathon in Honor of Niemann-Pick Disease Type B

A team of runners from Genzyme Corporation competed in the 2010 Boston Marathon this spring, raising approximately $25,000 for the National Organization of Rare Diseases (NORD).

Each runner or pair of runners raced in honor of someone affected by a rare disease, and two of the marathoners were matched up with Collin and Cohen Parody, both affected by Niemann-Pick Disease Type B. The runners wore shirts with a photo of the boys, and ran in their name.

The runners spoke of their excitement and sense of meaning in running in honor of a specific individual affected by a rare disease.

As one runner commented, "I just have to say that working on this has made the experience so much more significant for me. Besides putting a human connection on the whole venture, it puts what we’re doing in perspective.... The fundraising and awareness are as much a part of this – if not more so – than the running is now. And that’s saying a lot, considering we’re runners! "

For all the latest news from the National Niemann-Pick Disease Foundation, visit www.nnpdf.org.