Genzyme related Press Releases

Genzyme Announces FDA Approval of Framingham Manufacturing Plant

Tue, 24 Jan 2012 01:22:14 -0500

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today that the Food and Drug Administration (FDA) has approved its manufacturing plant in Framingham, Mass., for the production of Fabrazyme® (agalsidase beta). This follows the previously announced approval by the European Medicines Agency (EMA) last week.

“We are very pleased with the FDA approval of our Framingham plant as we continue our manufacturing recovery and path forward to serve the Fabry patient community,” said Genzyme’s President and CEO David Meeker. “With this approval, we continue upon our 2012 plan to restore unconstrained supply for all patients globally throughout the course of the year.”

Approval of the Framingham site allows Genzyme to begin the process of returning patients to full dosing (1 mg/kg) levels. Following the EMA approval, Genzyme will begin the process of moving the most severely affected patients in Europe to full dose of Fabrazyme in Q1 2012. Beginning in March, all patients in the U.S. currently on therapy will be returned to full dosing. In addition, the company will begin to transition new patients in the U.S. onto Fabrazyme, at full dosing levels. Globally, the complete return to normal supply levels of Fabrazyme will begin in the second quarter and continue throughout the year as planned, as Genzyme works to obtain all global regulatory approvals throughout the year and to build inventory.

About Genzyme, a Sanofi Company

Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme’s portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world’s largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Learn more at www.genzyme.com.

Forward Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labeling and other matters that could affect the availability or commercial potential of such products candidates, the absence of guarantee that the products candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, the Group’s ability to benefit from external growth opportunities as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2010. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

CONTACT:

Genzyme
Lori Gorski, 617-768-9344
Lori.gorski@genzyme.com

KEYWORDS: United States North America Massachusetts

INDUSTRY KEYWORDS: Health Biotechnology Clinical Trials Genetics Pharmaceutical FDA General Health

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Genzyme Announces European Approval of Framingham Manufacturing Plant

Wed, 18 Jan 2012 01:20:40 -0500

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today that the European Medicines Agency (EMA) has approved its manufacturing plant in Framingham, Mass., for the production of Fabrazyme^® (agalsidase beta).

“This approval by the EMA represents an important milestone in our manufacturing recovery and path toward unconstrained supply for all patients,” said Genzyme’s President and CEO David Meeker. “Providing the Fabry community with consistent access to treatment, increasing our inventory of Fabrazyme^® and working toward all regulatory approvals of our Framingham plant are our highest priorities, and we remain on track to achieve all of these crucial goals.”

The complete return to normal supply levels of Fabrazyme^® globally will not be immediate, as it will take time to obtain all global regulatory approvals throughout the year and due to production lead times.

About Genzyme, a Sanofi Company

About Sanofi

Sanofi, a global and diversified healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients’ needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, rare diseases, consumer healthcare, emerging markets and animal health. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).

Forward Looking Statements

CONTACT:

Genzyme Media Relations
Lori Gorski, +1 617-768-9344
Lori.gorski@genzyme.com
or
Sanofi Media Relations
Marisol Péron, +33 (0) 1 53 77 45 02
mr@sanofi.com
or
Sanofi Investor Relations
Sébastien Martel, +33 (0) 1 53 77 45 45
ir@sanofi.com

KEYWORDS: United States North America Massachusetts

INDUSTRY KEYWORDS: Health Biotechnology Clinical Trials Genetics Pharmaceutical FDA

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Inspiration Biopharmaceuticals Expands Leadership Team with Four New Hires

Thu, 22 Dec 2011 08:21:00 -0500

CAMBRIDGE, Mass., Dec. 22, 2011 /PRNewswire/ -- Inspiration Biopharmaceuticals, Inc. (Inspiration), the only biopharmaceutical company focused exclusively on the development and commercialization of new treatment options for people with hemophilia, announced today that the Company has expanded its leadership team with four key hires. Howard Levy, M.D., Ph.D. has joined Inspiration as Chief Medical Officer; Daniel Regan as Chief Commercial Officer; Nikki Hadas as General Counsel; and Karen Tubridy as Vice President and Factor IX Launch Team Leader.

Dr. Levy has over 22 years' experience in the pharmaceutical and medical industries, most recently serving as Chief Medical Officer at Sangart, Inc. Prior to Sangart, he was Associate Vice President, Clinical Research, Medical and Regulatory Affairs, at Novo Nordisk and was responsible for a number of clinical research programs, including the hemostasis platform. Earlier, Dr. Levy was Clinical Research Physician and Medical Director Acute Care in the U.S. Medical Division of Eli Lilly and Company, and Chief of Critical Care Medicine at the University of New Mexico in Albuquerque. Dr. Levy received his Ph.D. and MB BCh from University of Witwatersrand, Johannesburg, South Africa. Dr. Levy serves on the Scientific Advisory Boards of ZZ Biotech and Artisan.

Mr. Regan joins Inspiration from Genzyme Corporation where he held positions of increasing responsibility since 1999, most recently serving as General Manager, Senior Vice President of the U.S. Personalized Genetic Health business unit. At Genzyme, he led multi-functional teams, including sales, marketing and commercialization, across a number of business units, and launched several new products into both large and small markets. In addition to his domestic experience, Mr. Regan worked extensively in Europe, Latin America, and Asian/Pacific markets. Prior to Genzyme, he held sales and marketing positions at Janssen Pharmaceutica, a pharmaceutical division of Johnson & Johnson. Mr. Regan began his career at Whitehall Laboratories. Mr. Regan received his B.A. from University of Massachusetts.

Ms. Hadas also comes to Inspiration from Genzyme where she spent 10 years, most recently as Senior Corporate Counsel. Ms. Hadas provided corporate legal support for Genzyme's Personalized Genetic Health, Renal, Endocrine and Cardiovascular business units. Prior to Genzyme, she was an Associate at Foley Hoag LLP. At Foley Hoag, Ms. Hadas represented pharmaceutical, biotechnology and medical device companies, as well as other healthcare entities. Earlier, she was a law clerk for Chief Justice David Brock of the New Hampshire Supreme Court. Ms. Hadas received her B.A. from the University of Michigan and her J.D. from Boston College Law School.

Ms. Tubridy has over 20 years' experience in directing global clinical programs, developing regulatory and clinical strategies, and leading development programs of novel and first in class biologics and monoclonal antibodies, with a particular focus on hematology/oncology. Ms. Tubridy most recently served as Executive Director, Clinical Operations and Regulatory Affairs, Translational Medicine at Alexion Pharmaceuticals. She served in this same role at Taligen until its acquisition by Alexion. Before Taligen, Ms. Tubridy was Senior Director, Clinical Operations at Biogen Idec Hemophilia and Vice President, Clinical Operations of Syntonix Pharmaceuticals. In these positions, she developed the clinical program strategy and provided operational support for the companies' hemophilia programs. Ms. Tubridy also held positions at AVANT Immunotherapeutics, Wyeth, Genetics Institute (where she was responsible for the hemophilia global medical affairs programs) and New England Medical Center. Ms. Tubridy received her B.Sc. and PharmD from Massachusetts College of Pharmacy and Allied Health Sciences.

John P. Butler, Chief Executive Officer of Inspiration, commented, "Howard, Dan, Nikki and Karen join Inspiration at a very exciting time in the Company. As Inspiration advances its lead programs, IB1001 and OBI-1, towards commercialization, we are focused on building a world-class company dedicated to improving the life of individuals with hemophilia. The new additions bring invaluable and diverse experiences to the Company and will undoubtedly build upon the established strengths and expertise of the leadership team. We expect our new Boston-area location will continue to attract great talent."

Dr. Levy, Mr. Regan, Ms. Hadas and Ms. Tubridy join Inspiration's executive leadership team which includes John Butler, Mike Griffith, Ph.D. (President and Chief Scientific Officer), Andrew Grethlein, Ph.D. (Executive Vice President and Chief Operating Officer) and Gordon H. Busenbark (Senior Vice President and Chief Financial Officer). Inspiration's team has broad expertise and experience in hemophilia product development, biologics manufacturing and the successful commercialization of products to treat hemophilia as well as chronic orphan diseases.

About Inspiration Biopharmaceuticals

Inspiration Biopharmaceuticals is dedicated exclusively to developing treatments for hemophilia, with a primary mission to broaden access to care to safe and effective recombinant therapies and advance innovation for people living with these conditions. Inspiration has a broad portfolio of recombinant hemophilia products, which includes two products in late-stage clinical development and two preclinical programs.

Inspiration's lead product candidates are IB1001, an intravenous recombinant factor IX (FIX) product for the treatment and prevention of bleeding in individuals with hemophilia B, and OBI-1, an intravenous recombinant porcine factor VIII (FVIII) product for the treatment of individuals with congenital hemophilia A who have developed inhibitors against human FVIII and for individuals with acquired hemophilia. Inspiration recently submitted its first marketing application for IB1001 in Europe, with a subsequent regulatory filing planned in the U.S. Earlier-stage preclinical programs at Inspiration are focused on human recombinant factor VIIa (FVIIa), for individuals with either hemophilia A or hemophilia B who have developed inhibitors, and for individuals with factor VII deficiency; and human recombinant FVIII, to treat individuals with hemophilia A.

Inspiration's senior management team has broad experience and expertise in hemophilia product development, biologics manufacturing and the successful commercialization of products to treat both hemophilia as well as chronic orphan diseases.

In addition, in January 2010, Inspiration entered into a strategic partnership with the Ipsen Group (EURONEXT: IPN; ADR: IPSEY), leveraging the combined expertise and resources of the two companies, to develop a broad portfolio of hemophilia products. As announced in late August 2011, Ipsen and Inspiration extended their partnership to create a hemophilia business unit structure that will act as the exclusive sales organization for all hemophilia products commercialized under the Inspiration brand in Europe. For further information on Inspiration, please visit http://www.inspirationbio.com.

Contacts:
Media	Company
Justin Jackson	Gordon H. Busenbark
Michelle Szwarcberg	Senior Vice President, Chief Financial Officer
Burns McClellan	Email: gbusenbark@inspirationbio.com
Tel: +1-212-213-0006
Email: jjackson@burnsmc.com
mszwarcberg@burnsmc.com

SOURCE Inspiration Biopharmaceuticals, Inc.

Genzyme Reports Top-line Results for TENERE Study of Oral Teriflunomide in Relapsing Multiple Sclerosis

Maureen Martino — Tue, 20 Dec 2011 12:15:12 -0500

Genzyme Reports Top-line Results for TENERE Study of Oral Teriflunomide in Relapsing Multiple Sclerosis

Paris, France - December 20, 2011 - Sanofi (EURONEXT: SAN and NYSE: SNY) and its subsidiary Genzyme today reported top-line results from TENERE, a Phase III clinical trial comparing the effectiveness, safety and tolerability of once-daily oral teriflunomide to interferon beta-1a (Rebif®), an approved injectable therapy, in people with relapsing forms of multiple sclerosis (RMS). The TENERE trial, which included 324 patients, is the second completed study of five efficacy studies of teriflunomide in MS, making the clinical program one of the largest and broadest of any multiple sclerosis agent under development.

No statistical superiority was observed between the Rebif and teriflunomide arms (7mg and 14mg) on risk of treatment failure, the primary composite endpoint of the study. Risk of treatment failure was defined as the occurrence of a confirmed relapse or permanent treatment discontinuation for any cause, whichever came first. In the study, 48.6 percent of patients receiving 7mg of oral teriflunomide (n=109) and 37.8 percent of patients receiving 14 mg of oral teriflunomide (n=111) reached the primary endpoint, versus 42.3 percent of patients receiving interferon beta 1-a (n=104).

The teriflunomide 14 mg daily dose (0.259) and Rebif (0.216) were not distinguishable on the endpoint of estimated annual relapse rate. The rate was higher in the 7mg arm (0.410). The percentage of patients experiencing any treatment emergent adverse events was similar across all arms of the study. The rate of permanent treatment discontinuation in the study due to a treatment emergent adverse event was higher in the Rebif arm (21.8 percent vs. 8.2 percent in the 7mg teriflunomide arm and 10.9 percent in the 14 mg teriflunomide arm).

Both the 7mg and 14mg doses of teriflunomide were safe and generally well tolerated. Most adverse events observed in the teriflunomide arms were mild in severity, including nasopharyngitis, diarrhea, hair thinning, and back pain. These occurred with a higher incidence than in the Rebif arm. The most common adverse events observed in the Rebif arm were increases in alanine aminotransferase levels, headache and flu-like symptoms. These occurred with a higher incidence than in the teriflunomide arms.

There were no deaths in the trial.

Genzyme anticipates presenting detailed TENERE study findings at a forthcoming medical meeting. The company will also include the results in its application with the EMA for marketing authorization in the European Union, along with results from its successful Phase lll TEMSO trial. The company expects to file an application for marketing authorization with the EMA in the first quarter of 2012. The U.S. FDA application for teriflunomide was accepted for review by the U.S. FDA in October 2011.

About the TENERE Trial TENERE was a two-year, randomized, rater-blinded comparator study that included 324 people with RMS from 53 centers in 13 countries. Trial participants were 18 years of age or older, with an Expanded Disability Status Scale (EDSS) of 5.5 or less at the initial screening visit. Trial participants were randomized to receive oral teriflunomide, 7 mg or 14 mg, once daily, or interferon beta-1a (Rebif® 44mcg tiw new formulation) and were followed for 48 weeks. The primary endpoint was risk of failure as defined by the first occurrence of relapse or permanent study treatment discontinuation for any cause, whichever came first. Secondary outcome measures included annualized relapse rate, subject-reported fatigue as assessed by the Fatigue Impact Scale (FIS), and subject satisfaction as assessed by the Treatment Satisfaction Questionnaire for Medication (TSQM). Safety and tolerability evaluations were based on adverse events, physical examinations, vital signs and laboratory investigations. A long-term extension of TENERE is ongoing.

About Teriflunomide Teriflunomide is an immunomodulatory, disease-modifying oral drug with anti-inflammatory properties, and is under investigation for the treatment of MS. Teriflunomide blocks the proliferation and functioning of activated T and B lymphocytes - which are thought to be especially damaging in MS - by selectively and reversibly inhibiting a critical mitochondrial enzyme. Slowly dividing or resting lymphocytes are unaffected by teriflunomide, leaving the immune system's response to infection uncompromised.

Teriflunomide is being studied in a large clinical program that is expected to include more than 4,000 trial participants in 36 countries. Five efficacy clinical trials are either completed or underway with teriflunomide, making the clinical program one of the largest and broadest of any MS agent under development. In addition to the TEMSO and TENERE trials, the Phase III, placebo-controlled trial TOWER is ongoing in people with RMS. Another Phase III study, TOPIC, is underway in early MS or CIS (clinically isolated syndrome). Teriflunomide is also being evaluated as an adjunct therapy to interferon-β in the Phase III TERACLES trial. With up to 10 years of continuous use in a Phase II extension, teriflunomide has the longest clinical experience of any investigational oral MS therapy.

About Genzyme, a Sanofi Company Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme's portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world's largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Learn more at www.genzyme.com.

About Sanofi Sanofi, a global and diversified healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, rare diseases, consumer healthcare, emerging markets and animal health. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).

Forward Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forwardlooking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such 3/3 as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labeling and other matters that could affect the availability or commercial potential of such products candidates, the absence of guarantee that the products candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, the Group's ability to benefit from external growth opportunities as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2010. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements Genzyme® is a registered trademark.

Rebif® is a registered trademark of EMD Serono, Inc. or affiliates.

Contacts:
Genzyme Media Relations
Bo Piela
Tel: +1-617-768-6579
Mobile: +1-508-308-9783
E-mail: bo.piela@genzyme.com
Sanofi Media Relations
Marisol Péron
Tel: +33-(0)-1-53-77-45-02
E-mail: mr@sanofi.com
Sanofi Investor Relations
Sébastien Martel
Tel: +33-(0)-1-53-77-45-45
E-mail: ir@sanofi.com

Micromet Appoints Joseph Lobacki Senior Vice President and Chief Commercial Officer

Mon, 05 Dec 2011 08:20:47 -0500

ROCKVILLE, Md.--(BUSINESS WIRE)-- Micromet, Inc. (NASDAQ: MITI) today announced the appointment of Joseph Lobacki as Senior Vice President and Chief Commercial Officer.

Mr. Lobacki brings to Micromet over 25 years of commercial experience in the biopharmaceutical industry. From 2003 to 2011, Mr. Lobacki served as Senior Vice President and General Manager, Transplant and Oncology at Genzyme and as a member of the company’s senior management team. In this role, he was responsible for directing overall strategy for the company’s global hematology, oncology, and organ transplant business. Under his leadership Genzyme’s transplant/oncology franchise consistently produced double digit percent revenue growth through expansion of product market share, geographical growth, clinical market development and business acquisitions.

"Joe's proven track record in commercializing market leading oncology products makes him a valuable addition to the Micromet management team," said Christian Itin, Ph.D., Micromet’s President and Chief Executive Officer. "His extensive sales, marketing and medical affairs experience will be critical as we look to prepare the marketplace for blinatumomab’s potential launch."

“I am excited to join Micromet during a key growth phase for the company,” said Mr. Lobacki. “I look forward to providing the business direction for the planned launch of blinatumomab in acute lymphoblastic leukemia and helping to maximize the compound’s significant commercial potential across multiple indications.”

Prior to joining Genzyme, Mr. Lobacki served as Vice President, North American Marketing at SangStat Medical Corporation, where he was responsible for all marketing activities for the company’s lead product. Earlier in his career, he served in leadership roles at Cell Pathways and Rhone-Poulenc Rorer. Mr. Lobacki holds a B.S. in Biology from Boston College and a B.S. in Pharmacy from the Massachusetts College of Pharmacy.

About Micromet, Inc.

Micromet is a biopharmaceutical company focused on the discovery, development and commercialization of innovative antibody-based therapies for the treatment of cancer. The Company is advancing a robust pipeline of novel therapeutics based on its proprietary BiTE® technology. The Company's lead product candidate blinatumomab (MT103) is currently the subject of a European pivotal trial in patients with minimal residual disease positive acute lymphoblastic leukemia. Micromet has collaborations with a number of leading pharmaceutical and biotechnology companies, including Amgen, Bayer HealthCare Pharmaceuticals, Boehringer Ingelheim, MedImmune, Nycomed and Sanofi.

Safe Harbor Statement

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Any statements contained herein that do not describe historical facts are forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. These forward-looking statements include statements regarding the development and commercialization of blinatumomab. You are urged to consider statements that include the words "will," "believes," "potential," "plans," "intends," "may," "suggests," or the negative of those words or other similar words to be uncertain and forward-looking. Factors that may cause actual results to differ materially from any future results expressed or implied by any forward-looking statements include the risk that blinatumomab does not demonstrate safety and/or efficacy in on-going or future clinical trials, delays in development and testing, including the risk that we will not obtain approval to market blinatumomab, and the risks associated with reliance on outside financing to meet capital requirements. These factors and others are more fully discussed in Micromet's Annual Report on Form 10-K, as amended, for the fiscal year ended December 31, 2010, and Micromet's Quarterly Report on Form 10-Q for the fiscal quarter ended September 30, 2011, filed with the SEC on November 8, 2011 as well as other filings by Micromet with the SEC. Micromet cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Micromet also disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

CONTACT:

Micromet, Inc.
Jennifer Neiman, Director, Corporate Communications, 240-235-0246
jennifer.neiman@micromet.com

KEYWORDS: United States North America Maryland

INDUSTRY KEYWORDS: Health Biotechnology Clinical Trials Oncology Other Health Research Other Science Science

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Genzyme and Cystic Fibrosis Foundation Therapeutics Announce Collaboration to Discover New CF Drugs

Maureen Martino — Thu, 17 Nov 2011 12:38:32 -0500

Genzyme and Cystic Fibrosis Foundation Therapeutics Announce Collaboration to Discover New CF Drugs

New effort will search for potential therapies targeting the most common mutation of cystic fibrosis

CAMBRIDGE, Mass. & BETHESDA, Md., Nov 16, 2011 (BUSINESS WIRE) -- Genzyme, a Sanofi company , and Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, today announced a research agreement to support the discovery of new drugs to treat people with the most common mutation found in patients with CF, Delta F508.

People with cystic fibrosis, a genetic disease, experience a cascade of symptoms that can lead to life-threatening lung infections and premature death.

The program's focus is to identify compounds known as "correctors," which may aid in the ability of the malfunctioning CFTR protein found in CF patients to operate correctly. In the Delta F508 mutation, the CFTR protein does not move to its proper place at the cell surface, impeding the flow of fluids into the airways. Nearly 90 percent of people with CF have at least one copy of the Delta F508 mutation.

In this collaboration, researchers will evaluate different compound libraries for correctors for Delta F508, and will take advantage of the vast compound libraries of both Genzyme and Sanofi. The research will take place throughout several Genzyme and Sanofi R&D facilities globally. Genzyme brings to the collaboration more than 20 years' experience exploring treatments for people living with CF. The company's efforts have ranged from improved molecular diagnostics to clinical trials with a gene therapy, and have included past collaboration with the CF Foundation in the area of drug discovery.

"We are delighted to enter into a research collaboration with Genzyme, a company that has long dedicated itself to improving the lives of people with rare diseases," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. "Genzyme's capabilities and resources will help the CF Foundation accelerate its effort to find drugs to treat the most common mutation in CF and have the greatest impact on those with this disease."

"While there has been great momentum recently in cystic fibrosis research, there is still great unmet need," said Genzyme's president and CEO David Meeker, MD. "Together with the CF Foundation, we look forward to working to accelerate the pace of discovery on behalf of CF patients around the world."

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, go to www.cff.org .

About Genzyme, a Sanofi Company

Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme's portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world's largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Learn more at www.genzyme.com .

SOURCE: Genzyme

Genzyme
Lori Gorski, 617-768-9344
Lori.gorski@genzyme.com
or
CFF
Laurie Fink, 301-841-2602
lfink@cff.org

Genzyme and Cystic Fibrosis Foundation Therapeutics Announce Collaboration to Discover New CF Drugs

Wed, 16 Nov 2011 09:21:33 -0500

New effort will search for potential therapies targeting the most common mutation of cystic fibrosis

CAMBRIDGE, Mass. & BETHESDA, Md.--(BUSINESS WIRE)-- Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, today announced a research agreement to support the discovery of new drugs to treat people with the most common mutation found in patients with CF, Delta F508.

People with cystic fibrosis, a genetic disease, experience a cascade of symptoms that can lead to life-threatening lung infections and premature death.

The program’s focus is to identify compounds known as “correctors,” which may aid in the ability of the malfunctioning CFTR protein found in CF patients to operate correctly. In the Delta F508 mutation, the CFTR protein does not move to its proper place at the cell surface, impeding the flow of fluids into the airways. Nearly 90 percent of people with CF have at least one copy of the Delta F508 mutation.

In this collaboration, researchers will evaluate different compound libraries for correctors for Delta F508, and will take advantage of the vast compound libraries of both Genzyme and Sanofi. The research will take place throughout several Genzyme and Sanofi R&D facilities globally. Genzyme brings to the collaboration more than 20 years’ experience exploring treatments for people living with CF. The company’s efforts have ranged from improved molecular diagnostics to clinical trials with a gene therapy, and have included past collaboration with the CF Foundation in the area of drug discovery.

“We are delighted to enter into a research collaboration with Genzyme, a company that has long dedicated itself to improving the lives of people with rare diseases,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Genzyme’s capabilities and resources will help the CF Foundation accelerate its effort to find drugs to treat the most common mutation in CF and have the greatest impact on those with this disease.”

“While there has been great momentum recently in cystic fibrosis research, there is still great unmet need,” said Genzyme’s president and CEO David Meeker, MD. “Together with the CF Foundation, we look forward to working to accelerate the pace of discovery on behalf of CF patients around the world.”

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, go to www.cff.org.

About Genzyme, a Sanofi Company

CONTACT:

Genzyme
Lori Gorski, 617-768-9344
Lori.gorski@genzyme.com
or
CFF
Laurie Fink, 301-841-2602
lfink@cff.org

KEYWORDS: United States North America Maryland Massachusetts

INDUSTRY KEYWORDS: Health Biotechnology Genetics Infectious Diseases Pharmaceutical

MEDIA:

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Genzyme Corporation (GENZ) MS Drug Significantly Reduces Relapse, Disability in Phase III Trial

Karl Hardy — Tue, 15 Nov 2011 07:48:21 -0500

Genzyme Corporation (GENZ) MS Drug Significantly Reduces Relapse, Disability in Phase III Trial

11/14/2011 7:03:18 AM

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), reports today that the Phase lll CARE-MS ll trial met both of its co-primary endpoints. Relapse rate and sustained accumulation (worsening) of disability (SAD) were significantly reduced in multiple sclerosis patients receiving alemtuzumab (LEMTRADA^TM) as compared with Rebif® (44 mcg subcutaneous interferon beta-1a). Results for both of these co-primary endpoints were highly statistically significant. CARE-MS II is the randomized Phase III clinical trial comparing the investigational drug alemtuzumab to interferon beta-1a in patients with relapsing-remitting multiple sclerosis (RRMS). Patients were required to have experienced a relapse while on a prior therapy to be eligible for CARE-MS II. Genzyme is developing alemtuzumab in MS in collaboration with Bayer HealthCare.

In this randomized trial involving 840 patients, a 49 percent reduction in relapse rate was observed in patients treated with alemtuzumab 12 mg compared to interferon beta-1a over two years of study (p<0.0001). Importantly, there was also a 42 percent reduction in the risk of sustained accumulation (worsening) of disability as measured by the Expanded Disability Status Scale (EDSS) (p=0.0084). Analysis of the full CARE-MS II data is ongoing and results will be presented at a forthcoming scientific meeting.

"CARE-MS ll represents the culmination of many years of clinical and laboratory research aimed at demonstrating the potential for alemtuzumab as a highly effective treatment for MS and understanding mechanisms involved in the complex natural history of the disease," said Professor Alastair Compston, Chair of the Steering Committee overseeing the conduct of the study and head of the Department of Clinical Neurosciences at the University of Cambridge, United Kingdom. "Taken together, the Phase ll and lll clinical trial data illustrate the promise that alemtuzumab holds as a transformative treatment for people with relapsing MS."

The CARE-MS II trial compared treatment with alemtuzumab 12 mg given daily as an IV administration for 5 days, and then again for 3 days one year later, to treatment with interferon beta-1a 44 mcg administered by injection three times per week throughout the two years of study.

"The superior efficacy results for alemtuzumab, particularly the slowing of disability, are very promising since this was a head-to-head comparison trial with high dose subcutaneous interferon beta-1a," said Dr. Jeffrey Cohen, Professor of Medicine (Neurology), Cleveland Clinic Lerner College of Medicine; Director of Experimental Therapeutics, Mellen Center for MS Treatment and Research; and a member of the Steering Committee overseeing the conduct of the study. "These results suggest alemtuzumab's potential to offer patients with MS a new and effective treatment option."

The safety profile observed in the trial was consistent with previous alemtuzumab use in MS and adverse events continued to be manageable. The most common types of adverse events associated with alemtuzumab in the CARE-MS II study were infusion-associated reactions, the symptoms of which most commonly included headache, rash, nausea, hives, fever, itching, insomnia, and fatigue. Infections were common in both groups with a higher incidence in the alemtuzumab group. The most common infections in patients receiving alemtuzumab included upper respiratory and urinary tract infections, sinusitis and herpes simplex infections. Infections were predominantly mild to moderate in severity and there were no treatment-related life-threatening or fatal infections.

Approximately 16 percent of alemtuzumab-treated patients developed an autoimmune thyroid-related adverse event and approximately one percent developed immune thrombocytopenia during the two-year study period. These cases were detected early through a monitoring program and managed using conventional therapies. Patient monitoring for immune cytopenias and thyroid or renal disorders is incorporated in all Genzyme-sponsored trials of alemtuzumab for the investigational treatment of MS.

"We are very pleased with the results of the CARE-MS II study which are unprecedented," said David Meeker, M.D., President and Chief Executive Officer, Genzyme. "We believe that LEMTRADA^TM, with its impressive efficacy, novel dosing regimen and manageable safety profile, could make a very important contribution to the MS treatment landscape, where a significant unmet need still exists for many patients. Based on these positive results, we are on track to submit LEMTRADA^TM for review to US and EU regulatory authorities in the first quarter of 2012."

Alemtuzumab has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). The FDA's Fast Track program is designed to expedite the review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. Under Fast Track designation, alemtuzumab for MS is eligible for Priority Review. Since it is not yet approved for the treatment of MS, alemtuzumab must not be used in MS patients outside of a formal, regulated clinical trial setting in which appropriate patient monitoring measures are in place.

*LEMTRADA^TM is the proprietary name submitted to health authorities for the company's investigational multiple sclerosis agent alemtuzumab.

About the CARE-MS II Trial

CARE-MS II (The Comparison of Alemtuzumab and Rebif® Efficacy in Multiple Sclerosis) trial was designed to evaluate whether the investigational MS therapy alemtuzumab could achieve meaningful efficacy and safety improvements over the approved, active comparator interferon beta-1a, a standard treatment for relapsing MS.

CARE-MS II was a Phase III, global, randomized clinical trial comparing treatment with alemtuzumab to treatment with subcutaneous interferon beta-1a (44 mcg administered by injection three times per week) in 840 patients who relapsed despite receiving prior MS treatment. Patients enrolled in the trial had to have experienced at least two relapses within the two years prior to entering the trial, with at least one of these relapses occurring within one year prior to enrollment and at least one relapse occurring while on MS therapy.

The CARE-MS II trial had two co-primary endpoints: reduction in relapse rate and six months sustained accumulation of disability (SAD)**. Secondary outcome measures include: Percentage of relapse-free patients at year two; Expanded Disability Status Scale (EDSS) change from baseline; percent change in magnetic/resonance imaging (MRI)-T2-hyperintense lesion volume at year two; and Multiple Sclerosis Functional Composite (MSFC) change from baseline. Disability assessments were performed at regularly scheduled visits by independent, evaluating neurologists who were blinded to the patients' treatment assignments. Relapse was determined by a blinded committee.

**Sustained Accumulation of Disability - Clinical representation of the worsening of a patient's level of disability; CARE-MS ll monitored this endpoint over the course of six months.

About Alemtuzumab

Alemtuzumab is a humanized monoclonal antibody being studied as a potential therapy for relapsing MS. Alemtuzumab targets the cell-surface glycoprotein CD52, which is highly expressed on T- and B-lymphocytes. Preliminary research suggests that alemtuzumab initially depletes the T- and B-cells that may be responsible for the cellular damage in MS. This depletion of T- and B-cells is followed by a distinctive pattern of lymphocyte repopulation. Alemtuzumab appears to have little or no effect on other cells of the immune system. In addition to the completed CARE-MS II study, another Phase III trial, CARE-MS I, evaluated alemtuzumab against interferon beta-1a in relapsing-remitting MS patients naive to prior treatment and found a statistically significant reduction in relapse rate with alemtuzumab.

Genzyme has the worldwide rights to alemtuzumab and has primary responsibility for the development and commercialization of alemtuzumab in MS. Bayer HealthCare has been co-developing alemtuzumab in MS with Genzyme. Bayer HealthCare retains an option to co-promote alemtuzumab in MS and upon regulatory approval and commercialization would receive contingent payments based on sales revenue.

About Genzyme, a Sanofi Company

Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme's portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world's largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Learn more at www.genzyme.com.

About Sanofi

Sanofi, a global and diversified healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, rare diseases, consumer healthcare, emerging markets and animal health. Sanofi is listed in Paris (EURONEXT: SAN.PA - News) and in New York (NYSE: SNY - News).

About Bayer HealthCare

The Bayer Group is a global enterprise with core competencies in the fields of health care, nutrition and high-tech materials. Bayer HealthCare, a subgroup of Bayer AG with annual sales of more than EUR 16.913 billion (2010), is one of the world's leading, innovative companies in the healthcare and medical products industry and is based in Leverkusen, Germany. The company combines the global activities of the Animal Health, Consumer Care, Medical Care and Pharmaceuticals divisions. Bayer HealthCare's aim is to discover and manufacture products that will improve human and animal health worldwide. Bayer HealthCare has a global workforce of 55.700 employees and is represented in more than 100 countries. Find more information at www.bayerhealthcare.com.

Sanofi Forward Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labeling and other matters that could affect the availability or commercial potential of such products candidates, the absence of guarantee that the products candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, the Group's ability to benefit from external growth opportunities as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2010. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

Rebif® is a registered trademark of EMD Serono, Inc. or affiliates.

Contact:

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Bo Piela

Tel: +1-617-768-6579

Mobile: +1-508-308-9783

E-mail: bo.piela@genzyme.com

Sanofi Media Relations

Marisol Péron

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E-mail: mr@sanofi.com

Sanofi Investor Relations

Sébastien Martel

Tel: +33-(0)-1-53-77-45-45

E-mail: ir@sanofi.com.

Genzyme Appoints Leaders for Multiple Sclerosis and Rare Disease Businesses

Thu, 10 Nov 2011 09:21:25 -0500

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today the appointment of William “Bill” Sibold as Head of Multiple Sclerosis and Rogério Vivaldi as Head of Rare Diseases. Both will report to David Meeker, President and Chief Executive Officer of Genzyme, and will join the Genzyme Executive Team. The MS and Rare Disease businesses constitute Genzyme’s core focus following its integration with Sanofi.

William "Bill" Sibold has been appointed Head of Multiple Sclerosis at Genzyme. Bill has more than 20 years of experience in the biopharmaceutical industry, primarily in commercial operations. (Photo: Business Wire)

“These appointments are a critical step in launching the new Genzyme,” said David Meeker. “Bill and Rogério are dynamic leaders with the experience, energy, vision and commitment to patients needed to move us forward.”

Bill Sibold has more than 20 years of experience in the biopharmaceutical industry, primarily in commercial operations, and has worked on multiple sclerosis products Avonex^® and Tysabri^®. He spent eight years in positions of increasing responsibility at Biogen Idec Inc., where, as Senior Vice President of U.S. Commercial, he was responsible for the company’s $2.5 billion neurology, oncology and rheumatology therapeutic areas consisting of approximately 800 employees. Prior to this, Bill served as managing director of Biogen Idec’s Asia Pacific region, where he was responsible for Australia, New Zealand and six Asian countries.

“Our goal is to build a world-leading multiple sclerosis franchise,” said David Meeker. “Bill’s substantial commercial experience and his deep knowledge of the MS field will be critical to the launch of Lemtrada™* and Aubagio™*, two investigational therapies with the potential to transform the lives of people living with MS.”

Bill joins Genzyme from Avanir Pharmaceuticals, where he was the Chief Commercial Officer, leading all of the company's commercial activities. Bill earned his MBA from Harvard Business School and his BA in Molecular Biophysics and Biochemistry from Yale University.

Rogério Vivaldi joined Genzyme in 1997 and has held positions of increasing responsibility over his career with the company. Prior to this appointment, Rogério was President of Genzyme’s Renal and Endocrinology Business. He previously served as Senior Vice President and President of Genzyme Latin America, responsible for the company’s significant growth and diversification in the region, after founding Genzyme in Brazil.

Rogério was the first Brazilian doctor to treat a patient with Gaucher disease with enzyme replacement therapy in 1992 and has authored several publications on Gaucher disease. He was the founding partner of the Latin American Group on Gaucher. Rogério received his medical degree from Universidade do Rio de Janeiro Medical School, and received his MBA degree from Copead – Universidade Federal do Rio de Janeiro.

“Rogério’s experience as a physician treating Gaucher patients in Brazil and his subsequent work in building our rare disease business in Latin America will provide both continuity and an energizing new beginning for our global rare disease business,” said David Meeker. “Our highest priority is to resolve our product shortages and consistently meet the needs of patients.”

About Genzyme, a Sanofi Company

* Lemtrada™ and Aubagio™ are the proprietary names submitted to health authorities for the company's investigational multiple sclerosis agent alemtuzumab and teriflunomide respectively.

Photos/Multimedia Gallery Available: http://www.businesswire.com/cgi-bin/mmg.cgi?eid=50064473&lang=en

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Genzyme Media Relations
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bo.piela@genzyme.com

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Rogerio Vivaldi has been appointed Head of Rare Diseases at Genzyme. Rogerio joined Genzyme in 1997 and has held positions of increasing responsibility over his career with the company. (Photo: Business Wire)

BioMarin to Present at the Credit Suisse Healthcare Conference

Thu, 03 Nov 2011 10:21:02 -0400

NOVATO, Calif., Nov. 3, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Dan Maher, Senior Vice President of Product Development at BioMarin, will present a company update at the Credit Suisse Healthcare Conference in Phoenix on Thursday, November 10, 2011 at 3:30 p.m. MST.

Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.BMRN.com. A replay of the call will be archived on the site for one week following the call.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA; PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU; BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease; and BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

BioMarin®, Naglazyme®, Kuvan® and Firdapse™ are registered trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

Contact:
Investors	Media
Eugenia Shen	Bob Purcell
BioMarin Pharmaceutical Inc.	BioMarin Pharmaceutical Inc.
(415) 506-6570	(415) 506-3267

SOURCE BioMarin Pharmaceutical Inc.