Study showed tolerability of dose-escalation regimen
Company Website:
http://www.genzyme.com
CAMBRIDGE, Mass. -- (Business Wire)
Genzyme,
a Sanofi company, today presented data from its Phase 1b clinical study
at the Lysosomal Disease Network’s WORLD Symposium 2015 in Orlando, Fla.
detailing the investigational use of enzyme replacement therapy in the
non-neurological manifestations of acid sphingomyelinase deficiency
(ASMD, also known as Niemann-Pick disease type B), a lysosomal storage
disease caused by genetic mutations that affect the metabolism of
sphingomyelin. The Genzyme study evaluated the tolerability and safety
of olipudase alfa (recombinant human acid sphingomyelinase) in five
adult patients with ASMD.
Melissa P. Wasserstein, MD,Director of the Program for Inherited
Metabolic Diseases; Medical Director of the International Center for
Types A and B Niemann Pick Disease, Mount Sinai School of Medicine,
presented: An open-label, multicenter, ascending-repeat-dose study of
the tolerability and safety of recombinant human acid sphingomyelinase
(rhASM) in patients with ASM deficiency (ASMD). In the trial, each
patient received a starting dose of intravenous olipudase alfa at 0.1
mg/kg and escalated dosing every two weeks according to a predetermined
schedule up to 3 mg/kg or their maximum tolerated dose. The secondary
objective was to study the pharmacokinetics, pharmacodynamics, and
exploratory efficacy of olipudase alfa administered every two weeks for
26 weeks. The study findings showed that the dose escalation regimen was
well tolerated, with all patients reaching the maximum dose of 3 mg/kg.
No serious or severe adverse events or deaths were reported. The data
presented on the repeat-dose safety, pharmacodynamics, and exploratory
efficacy of olipudase alfa support its continued development for the
investigational use in non-neurological manifestations of ASMD. All five
patients are participating in the Long-Term Study and will continue on
therapy.
"Though a small number of patients, the response we have observed to
date is an early indication that this ASM enzyme replacement therapy is
promising for this therapeutic area,” said Genzyme’s Acting Head of Rare
Diseases, Richard Peters, M.D., Ph.D. “We look forward to continuing
this program and learning more as we work toward advancing a treatment
option for patients that is both safe and well tolerated.” Genzyme plans
to begin enrolling patients in a Phase 2/3 program for Niemann-Pick Type
B in 2015.
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 the field of healthcare with seven growth platforms:
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).
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Contacts:
Genzyme Media Contact:
Lori Gorski, 617-768-9344
lori.gorski@genzyme.com
or
Sanofi
Investor Relations Contact:
Sebastien Martel, +33 (0) 1.53.77.45.43
IR@sanofi.com
Source: Genzyme
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