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Sanofi Genzyme commneces Phase three clinical trial for neoGAA for Pompe Disease.

M2 EQUITYBITES-November 8, 2016-Sanofi Genzyme commneces Phase three clinical trial for neoGAA for Pompe Disease


Sanofi Genzyme, Sanofi's specialty care global business unit, has commenced a pivotal Phase three clinical trial, named COMET, for the investigational therapy neoGAA for Pompe Disease, it was reported yesterday.

NeoGAA is a second-generation enzyme replacement therapy being studied for the treatment of Pompe disease, a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA). 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 three randomised, multi-centre, multi-national, double-blinded study to compare the efficacy and safety of repeated bi-weekly infusions of neoGAA and alglucosidase alfa in treatment-naive patients with late-onset Pompe disease. The primary endpoint of the Phase three 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 six-minute walk test, muscle strength, motor function, health-related quality of life, and patient reported outcomes. Approximately 96 patients, ages three and up, are expected to be enrolled in the study, which will last up to three years, including a 49-week blinded treatment period and a 96-week open-label treatment period.

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Publication:M2 EquityBites (EQB)
Date:Nov 8, 2016
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