Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for
Pompe Disease
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 reserved.
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1 Zhu et al, Molecular Therapy, 2009.
Sanofi Genzyme
Media Relations
Lisa Clemence, +1-617-768-6699
lisa.clemence@genzyme.com
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