Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)

We report data from the Phase I/II ATB200-02 study for up to 48 months of treatment. Four adult cohorts, including one non-ambulatory ERT-experienced (n = 6) and three ambulatory cohorts, (two enzyme replacement therapy [ERT]-experienced cohorts [2–6 years (n = 11) and ≥ 7 years (n = 6)]), one ERT-naïve cohort (n = 6), received 20 mg/kg intravenous-infused cipa plus 260 mg oral mig biweekly. Change from baseline (CFBL) for multiple efficacy endpoints at 12, 24, 36, and 48 months, pharmacodynamics, pharmacokinetics, safety, and immunogenicity data were assessed. Six-minute walking distance (% predicted) improved at 12, 24, 36, and 48 months: pooled ambulatory ERT-experienced cohorts, mean(± standard deviation [SD]) CFBL: 6.1(± 7.84),n = 16; 5.4(± 10.56),n = 13; 3.4(± 14.66),n = 12; 5.9(± 17.36),n = 9, respectively; ERT-naïve cohort: 10.7(± 3.93),n = 6; 11.0(± 5.06),n = 6; 9.0(± 7.98),n = 5; 11.7(± 7.69),n = 4, respectively. Percent predicted forced vital capacity was generally stable in ERT-experienced cohorts, mean(± SD) CFBL − 1.2(± 5.95),n = 16; 1.0(± 7.96),n = 13; − 0.3(± 6.68),n = 10; 1.0(± 6.42),n = 6, respectively, and improved in the ERT-naïve cohort: 3.2(± 8.42),n = 6; 4.7(± 5.09),n = 6; 6.2(± 3.35),n = 5; 8.3(± 4.50),n = 4, respectively. Over 48 months, CK and Hex4 biomarkers improved in ambulatory cohorts. O...

Source: Journal of Neurology - December 6, 2023 Category: Neurology Source Type: research