Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease
November 04 2016 - 8:00AM
Business Wire
Sanofi Genzyme, the specialty care global business unit of
Sanofi, announced today that the first patient has been enrolled
and received an infusion in a pivotal Phase 3 clinical trial named
COMET for the investigational therapy neoGAA. NeoGAA is a
second-generation enzyme replacement therapy being studied for the
treatment of Pompe disease.
Pompe disease is a progressive, debilitating and often fatal
neuromuscular disease caused by a genetic deficiency or dysfunction
of the lysosomal enzyme acid alpha-glucosidase (GAA) affecting an
estimated 50,000 people worldwide. Patients often lose their
ability to walk and require wheelchairs to assist with mobility.
They also often experience difficulty breathing and may require
mechanical ventilation to breathe.
COMET is a Phase 3 randomized, multi-center, multi-national,
double-blinded study to compare the efficacy and safety of repeated
bi-weekly infusions of neoGAA and alglucosidase alfa in
treatment-naïve patients with late-onset Pompe disease. The primary
endpoint of the Phase 3 trial is the effect of neoGAA on
respiratory muscle strength as measured by percent predicted forced
vital capacity in the upright position. Other assessments include
functional endurance measured by the 6-minute walk test, muscle
strength, motor function, health-related quality of life, and
patient reported outcomes. Approximately 96 patients, ages 3 and
up, are expected to be enrolled in the study, which will last up to
3 years, including a 49-week blinded treatment period and a 96-week
open-label treatment period. For more information on the trial,
please visit https://www.clinicaltrials.gov/ or
https://www.clinicaltrialsregister.eu.
“The beginning of this pivotal trial is a critical milestone in
Sanofi Genzyme’s long history of advancing the understanding of
Pompe disease,” said Sanofi Genzyme Therapeutic Area Head for Rare
Diseases Development Rand Sutherland, M.D. “We are committed to
researching and developing novel treatment options to address the
unmet needs of Pompe patients.”
“Pompe disease is a serious and progressive condition,” said
Shafeeq S. Ladha, M.D., Ira A. and Mary Lou Fulton Chair in Motor
Neuron Diseases, Director, Gregory W. Fulton ALS and Neuromuscular
Disorders Center, Department of Neurology, Barrow Neurological
Institute. “My hope is that a second generation enzyme replacement
therapy with improved targeting to skeletal muscle may provide
additional benefit to patients with this debilitating disease.”
About neoGAANeoGAA is an investigational
second-generation alglucosidase alfa enzyme replacement therapy
that has been specifically designed for enhanced receptor targeting
and enzyme uptake through greater affinity for the M6P receptors on
muscle cells, with the aim of enhancing glycogen clearance and
improving on the clinical efficacy achieved with alglucosidase
alfa. In preclinical studies, neoGAA showed approximately five-fold
greater potency than alglucosidase alfa in terms of tissue glycogen
reduction compared to alglucosidase alfa. In the Pompe mouse model,
neoGAA reduced similar levels of substrate at one-fifth the dose of
alglucosidase alfa.1 The clinical significance of this data
requires further investigation.
Results from the Phase 1/2 proof of concept study were reported
at the WORLD Symposium earlier this year.
About SanofiSanofi, a global healthcare leader,
discovers, develops and distributes therapeutic solutions focused
on patients' needs. Sanofi is organized into five global business
units: Diabetes and Cardiovascular, General Medicines and Emerging
Markets, Sanofi Genzyme, Sanofi Pasteur and Merial. Sanofi is
listed in Paris (EURONEXT: SAN) and in New York
(NYSE: SNY).
Sanofi Genzyme focuses on developing specialty treatments for
debilitating diseases that are often difficult to diagnose and
treat, providing hope to patients and their families.
Genzyme® is a registered trademark of Genzyme Corporation.
Sanofi® is a registered trademark of Sanofi. All rights
reserved.
Forward-Looking StatementsThis 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,
Sanofi's ability to benefit from external growth opportunities
and/or obtain regulatory clearances, risks associated with
intellectual property and any related pending or future litigation
and the ultimate outcome of such litigation, trends in exchange
rates and prevailing interest rates, volatile economic conditions,
the impact of cost containment initiatives 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,
2015. Other than as required by applicable law, Sanofi does not
undertake any obligation to update or revise any forward-looking
information or statements.
1 Zhu et al, Molecular Therapy, 2009.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20161104005348/en/
Sanofi GenzymeMedia RelationsLisa Clemence,
+1-617-768-6699lisa.clemence@genzyme.com
Banco Santander (NYSE:SAN)
Historical Stock Chart
From Mar 2024 to Apr 2024
Banco Santander (NYSE:SAN)
Historical Stock Chart
From Apr 2023 to Apr 2024