Amicus Therapeutics Announced The Publication Of A Post-hoc Analysis Of Data From The Ert-experienced Cohort Of The PROPEL Study Of Cipaglucosidase Alfa-Atga + Miglustat (Cipa+Mig) In Adults With Late-onset Pompe Disease In Muscle And Nerve

Author: Benzinga Newsdesk | June 03, 2025 06:13am

PROPEL was a 52-week, double-blind randomized global study designed to assess the efficacy, safety, and tolerability of cipaglucosidase alfa-atga + miglustat compared to non-U.S. approved alglucosidase alfa + placebo (the comparator). The study enrolled 123 adult LOPD patients who still had the ability to walk and to breathe without mechanical ventilation.

Patients enrolled in PROPEL were randomized 2:1 so that for every two patients randomized to be treated with cipaglucosidase alfa-atga + miglustat, one was randomized to be treated with the comparator. Of the patients enrolled in PROPEL, 77% were being treated with alglucosidase alfa (n=95) for at least 2 years at study entry and 23% had never been treated with any ERT (n=28). 117 of the 123 patients (>95%) completed the PROPEL study.

Efficacy endpoints of the study included primary endpoint of change from baseline to week 52 in 6-minute walk distance (6MWD) for comparison of superiority and key secondary endpoint of change from baseline to week 52 in forced vital capacity (FVC). PROPEL did not achieve statistical significance for the primary endpoint of superiority in change from baseline to week 52 in 6MWD in the overall population. After 52 weeks, ERT-experienced patients treated with cipaglucosidase alfa-atga + miglustat (n=61) walked an estimated 17 meters (95% CI, 0.2, 33) farther than the comparator group (n=29). After 52 weeks, ERT-experienced patients treated with cipaglucosidase alfa-atga + miglustat (n=55) showed an estimated treatment difference of 3.5% (95% CI, 1.0, 6.0) in FVC compared with the comparator group (n=29).

More details about this post-hoc analysis

• This post-hoc analysis assessed the magnitude of within-group treatment effects across measures of motor function, muscle strength, pulmonary function, patient-reported outcomes/QoL measures, and biomarkers. Standardized effect size analysis describes the absolute effect size relative to the variability of the data. It transforms the effect size into a scale with no units of measurement (Cohen's d). The authors calculated standardized within-group effect sizes (Cohen's d for within-group comparisons) and corresponding 95% CIs for the change from baseline to week 52 for the primary, secondary, and pharmacodynamic endpoints of the PROPEL study. Standardized within-group effect sizes and corresponding 95% CIs were calculated by dividing the mean change from baseline values and CIs at week 52 by the standard deviation (SD) of the difference scores. Consistent with medical literature, effect sizes were defined as stable (−0.2 < d < 0.2), small (0.2 ≤ d < 0.5), medium (0.5 ≤ d < 0.8), or large (d ≥ 0.8) improvement, or as small (−0.5 < d ≤ −0.2), medium (−0.8 < d ≤ −0.5), or large (d ≤ −0.8) worsening, and were considered statistically significant if the standardized 95% CIs did not cross zero.

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