Sanofi completes study of potential Pompe disease therapy
NeoGAA is an enzyme replacement therapy, which was tested on 24 patients with the rare, progressive neuromuscular condition Pompe disease. NEO1 was an open-label, multicenter, multinational study. Most subjects tolerated the drug well at all dosing levels; no patients died during the study or experienced any life-threatening side effects.
“Respiratory function measures are important clinical indicators for Pompe patients,” Loren Pena, the coordinating investigator for the study, said. “The positive trending of exploratory data across all three respiratory endpoints -- percent predicted FVC, MEP and MIP -- suggests improvement or stabilization of pulmonary function in late-onset Pompe patients. This combined with the safety data indicates the potential for further development of neoGAA.”
The illness -- which often robs patients of their ability to walk, causes respiratory difficulty and is fatal -- is rooted in a genetic deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). It is estimated that 50,000 patients are living with Pompe disease around the world.
Sanofi researchers are now enrolling patients for the phase-three trial of neoGAA, which is expected to commence in the second quarter of this year.
“Sanofi Genzyme has a long history of investing in research and development to further the understanding of Pompe disease and advance treatment options,” Jorge Insuasty, head of global development, said. “We are highly encouraged that the safety profile and exploratory efficacy assessments provide positive proof of concept to continue the clinical development of neoGAA, which we plan to undertake later this year with a phase-three clinical trial.”
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