Next Generation ERT for Pompe Begins Pivotal Trial
James Radke, PhD
The pivotal Phase 3 clinical trial testing neoGAA, a second-generation enzyme replacement therapy, for Pompe disease has begun. Genzyme announced the first patient has begun infusions.
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). Patients often lose their ability to walk and require wheelchairs to assist with mobility. They may experience difficulty breathing and require mechanical ventilation to breathe.
The phase 3 trial named COMET is a randomized, multi-center, multi-national, double-blinded study comparing bi-weekly infusions of neoGAA and alglucosidase alfa (Lumizyme) in treatment-naïve patients with late-onset Pompe disease.
The primary endpoint 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/ct2/show/NCT02782741
In a news release, Shafeeq S. Ladha, MD, 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 said “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.”
Late-Onset Pompe Disease
Last year we talked with Dr Ladha and Loren Del Mar Pena, MD, PhD, of Duke University, who describe the typical symptoms that appear in patients with late-onset Pompe disease and the difficulty in getting it diagnosed quickly.
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.