Amicus announces publication of post-hoc analysis of data from PROPEL study

Amicus (FOLD) 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 in adults with late-onset Pompe disease in Muscle and Nerve. In this new publication, based on a within group effect-size analysis, subjects who switched from alglucosidase alfa to cipa+mig achieved improvements or stability in most of the outcomes measured. In PROPEL, 77% of patients received enzyme replacement therapy with alglucosidase alfa before study entry, with a median ERT duration of 7.4 years. In this new publication of post-hoc analysis from PROPEL, ERT-experienced patients switched to cipa+mig, showed improvement or stability, while those remaining on alglucosidase alfa + pbo generally showed within-group worsening or stability across most outcomes. Patients switched to cipa+mig did not demonstrate significant worsening for any outcomes and showed significant improvements for 6-minute walk distance; upper, lower, and overall manual muscle testing; Patient-Reported Outcomes Measurement Information System-Fatigue; Physician and Subject Global Impression of Change; and biomarker levels. Patients remaining on alglucosidase alfa + pbo demonstrated statistically significant worsening for several lung function outcomes, biomarker levels, and significant improvement for PROMIS-Dyspnea.