Amicus announces additional results in Pompe disease study
Amicus Therapeutics announced additional positive results from a global Phase 1/2 clinical study to investigate ATB200/AT2221 in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen in cells. Patients treated with ATB200/AT2221 for up to 12 months showed improvements in six-minute walk test distance and other measures of motor function, stability or increases in forced vital capacity, and durable reductions in biomarkers of muscle damage and disease substrate. These clinical results are being featured at the 14th Annual WorldSymposium. John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics stated, "We continue to be impressed by the clinical data for our novel Pompe treatment paradigm ATB200/AT2221. These latest data, in more patients and over longer periods, have continued to show substantial improvements in functional outcomes in nearly all patients, which are aligned with the persistent and durable reductions in key biomarkers of muscle damage and disease substrate. On the heels of these data for ATB200/AT2221 we look forward to continuing our discussions with global regulators to define the best and fastest pathway to deliver this critically important medicine to people living with Pompe disease." Tahseen Mozaffar, MD, Director, Neuromuscular Program, Neurology School of Medicine at UC Irvine and Principal Investigator in the ATB200-02 study stated, "The results from this Phase 1/2 clinical study of ATB200/AT2221 show very meaningful improvements across functional measures in both ERT-switch and ERT-naive patients for up to 12 months, with remarkable consistency across the vast majority of patients, as well as across endpoints. This treatment regimen has also been well tolerated by the patients in this study. Overall, the safety and functional data, in addition to the biomarkers of muscle damage and disease substrate, suggest the potential for ATB200/AT2221 to become an important treatment paradigm for people living with Pompe disease." Amicus continues to engage in a series of collaborative discussions with regulators in the U.S. and EU, and expects to provide an update in the first half of 2018.