Sanofi Genzyme Begins Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy

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 neoGAA
NeoGAA 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 Sanofi
Sanofi, 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
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¹ Zhu et al, Molecular Therapy, 2009.