Genzyme Initiates Phase 1/2 Clinical Trial to Evaluate Olipudase Alfa in Pediatric Patients

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Genzyme, a Sanofi company, announced today that the first pediatric patient has begun treatment in a Phase 1/2 clinical trial focused on evaluating the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B, as opposed to type A, which is characterized by neurological involvement. ASMD is a serious and life-threatening disorder caused by insufficient activity of the enzyme acid sphingomyelinase (ASM) resulting in toxic accumulation of sphingomyelin. There are currently no approved treatment options for patients with Niemann-Pick disease type B.

The Phase 1/2 trial is a multi-national, multi-center, open label, ascending dose trial to evaluate the safety, tolerability and pharmacokinetics of olipudase alfa administered intravenously once every 2 weeks for 52 weeks in pediatric patients with ASMD. Twelve pediatric patients will be enrolled into 3 age cohorts: an adolescent cohort (12 to <18 years of age); a child cohort (6 to <12 years of age); and an infant/early child cohort (birth to <6 years of age). The primary objective of the Phase1/2 trial is to assess the safety and tolerability of olipudase alfa. Upon completion of the 52-week trial, patients will have the option to enroll into an extension study. Genzyme is preparing for enrollment to begin in a Phase 2/3 trial involving adult patients with ASMD in the second half of 2015. For more information please visit http://clinicaltrials.gov.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olipudase alfa. Breakthrough Therapy designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions. The criteria for granting Breakthrough Therapy designation are preliminary clinical evidence of substantial improvement on a clinically significant endpoint over available therapies. The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review, and will allow for a close collaboration between Genzyme and the FDA on the olipudase alfa development program.

“With start of clinical studies both in pediatric and adult patients just a few months apart, we are confirming our commitment to the Niemann Pick disease patient community to advance the development of this novel therapy as quickly as possible and for both patient populations,” said Head of the Sanofi Genzyme R&D Center, Jim Burns, Ph.D. “We are very grateful to the patients for their engagement and their support in advancing this exciting program.”

About ASMD (Niemann-Pick Disease)

Traditionally called Niemann-Pick disease types A and B (NPD A and NPD B), Acid Sphingomyelinase Deficiency (ASMD) is one of a group of lysosomal storage diseases that affect the metabolism and that are caused by genetic mutations. ASMD is caused by the deficiency of a specific enzyme, acid sphingomyelinase (ASM). This enzyme is found in special compartments within cells called lysosomes and is required to metabolize a lipid called sphingomyelin. If ASM is absent or not functioning properly, sphingomyelin cannot be metabolized properly and is accumulated within the cell, eventually causing cell death and the malfunction of major organ systems. Niemann-Pick A and Niemann-Pick B are both caused by the same enzymatic deficiency and there is growing evidence that the two forms represent opposite ends of a continuum.

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.

Genzyme® is a registered trademark of Genzyme Corporation. All rights reserved.

About Sanofi

Sanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients’ needs. Sanofi has core strengths in diabetes solutions, human vaccines, innovative drugs, consumer healthcare, emerging markets, animal health and Genzyme. 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 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 labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product 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, trends in exchange rates and prevailing interest rates, the impact of cost containment policies and subsequent changes thereto, the average number of shares outstanding 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, 2014. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

Genzyme Media Relations
Lori Gorski +1 617-768-9344
lori.gorski@genzyme.com
or
Sanofi Investor Relations
Sébastien Martel, +33 (0) 1 53 77 45 43
ir@sanofi.com

This website uses cookies to track its audience and improve its content. By continuing to browse this website, you agree to the use of such cookies.

Click here for more information on cookies.
OK